AZ says Calquence is a safer drug than Imbruvica

AstraZeneca’s fast-growing BTK inhibitor Calquence is already challenging class leader Imbruvica from Johnson & Johnson/AbbVie in the treatment of chronic lymphocytic leukaemia (CLL). Now, AZ has new data suggesting its drug is less likely to cause a serious cardiac side effect.

The latest study in AZ’s ELEVATE trial series showed that Calquence (acalabrutinib) matched Imbruvica (ibrutinib) on keeping adults with previously treated, high-risk CLL alive without disease progression.

It also showed that AZ’s drug was significantly less likely to be associated with atrial fibrillation (AF), an irregular heart rate that can increase the risk of stroke, heart failure and other cardiovascular complications.

Two years ago, a study by the Cleveland Clinic in the US suggested that around 9% of patients treated with Imbruvica developed AF within a year of starting the drug, which was in the middle of other reviews suggesting the rate varied from around 4% to 16%. The authors suggested that it could generally be managed safely with drugs like beta blockers.

According to AZ, the ELEVATE-RR trial is the first  phase 3 study to compare two BTK inhibitors head to head in CLL, which is the most common form of leukaemia in adults. So far only the top-line outcome has been released, without any supporting data.

First approved for mantle cell lymphoma (MCL) in 2017, Calquence sales have rocketed since its approval for CLL towards the end of 2019, rising more than 200% to reach $340 million in the first nine months of 2020.

It is however still dwarfed by J&J and AbbVie’s blockbuster, which made around $5.6 billion in 2019 from half a dozen indications, including CLL and MCL. AZ has said is seeing swift growth for its brand in CLL thanks to its “outstanding efficacy and a favourable tolerability profile”.

That allowed the drug to capture around 35% of new patient starts in the US in the third quarter of 2020, according to the drugmaker.

That came ahead of the approval of Calquence for CLL in Europe, which was secured last November, as well registration of the drug in the last few days for relapsed or refractory CLL in Japan, based ono the results of the ASCEND trial.

“With over forty months of follow-up, today’s results confirm that Calquence…displays superior safety in atrial fibrillation without compromising efficacy,” said the company’s head of oncology José Baselga.

“The totality of the data confirm our confidence in the favourable benefit-risk profile of Calquence.”

AZ meanwhile is running trials to try to position Calquence as an alternative to Imbruvica in other indications, including diffuse large B-cell lymphoma, Waldenström’s macroglobulinaemia, and follicular lymphoma.

The post AZ says Calquence is a safer drug than Imbruvica appeared first on .

GSK/Merck & Co’s hopeful bintrafusp alfa fails in key lung cancer trial

GlaxoSmithKline’s big gamble on a cancer drug developed by Germany’s Merck KGaA looks unlikely to pay out, after bintrafusp alfa failed to outperform US-based Merck & Co’s Keytruda in a lung cancer trial.

GSK had high hopes in 2019 that Merck KGaA’s bintrafusp alfa could be a substantial addition to a pipeline that was in need of attention, paying more than $4.2 billion for the cancer immunotherapy in early 2019.

GSK secured co-development and co-marketing rights to the bifunction fusion protein, an anti-PD-L1/TGF beta trap for solid tumours including lung cancer.

The companies were so confident in the bispecific antibody that they began trialling it against Merck & Co’s blockbuster immunotherapy in first line lung cancer.

This is the most lucrative indication that has allowed Keytruda to establish a foothold as the most successful cancer immunotherapy on the market.

German Merck and GSK were testing the drug in patients with stage IV non-small cell lung cancer, with high expression of the PD-L1 biomarker, a mutation that tends to make tumours more susceptible to immunotherapy.

But an Independent Data Monitoring Committee has said the trial dubbed [email protected] Lung 037 should be halted.

Merck agreed, confirming the halt after admitting that the trial looks unlikely to meet its efficacy endpoint.

Although development is continuing in other forms of cancer, the results cast doubt on the future of the drug formerly known as M7824.

It also means that German Merck is set to miss out on some hefty “biobucks” milestone payments due from GSK under the terms of the deal, which began with a €300 million ($363 million) payment upfront.

There was another €500 million ($605m) tied to the lung cancer development programme, plus up to €2.9bn ($3.5bn) in development and commercial milestones.

The deal came off the back of phase 1 results announced at the 2018 European Society for Medical Oncology (ESMO) conference.

The data showed a much higher response rate with bintrafusp alfa in patients with NSCLC than would be expected with first-generation PD-1/PD-L1 inhibitors such as Keytruda (pembrolizumab).

The post GSK/Merck & Co’s hopeful bintrafusp alfa fails in key lung cancer trial appeared first on .

Lilly bolts on cancer bispecifics with $1.6bn Merus alliance

Always a big player in oncology, Eli Lilly has fallen behind some of its rivals when it comes to cancer immunotherapy, but a new R&D alliance with Dutch biotech Merus is further evidence of its fightback.

Lilly is tapping into Merus’ expertise in bispecific antibodies, specifically antibodies that target cancer cells with one end of the molecule and recruit immune cells with the other by binding to an activating receptor – in this case CD3.

Utrecht-based Merus is banking $40 million upfront from the deal, and getting a $20 million equity investment, but the total value could go north of $1.6 billion.

There is $540 million in milestones apiece on offer for each of three drug candidates covered by the partnership, and Merus is also in line for royalties in the “mid-single to low-double digits” if any of them reach the market.

It’s the first sizeable cancer deal for Lilly since the drugmaker acquired Loxo Oncology for $8 billion in 2019, although it has made a flurry of mid-size acquisitions and alliances in other areas like gene therapy (Prevail Therapeutics) and immunology/inflammation (Dermira) since then.

Shortly after buying Loxo, the big pharma revamped its cancer division under the leadership of Josh Bilenker, Loxo’s chief executive, and narrowed down its pipeline to a hit list of candidates from Loxo and Lilly’s pipelines.

The Merus deal comes under the umbrella of that new Loxo Oncology division, which has already brought one new drug – Retevmo (selpercatinib) for RET-positive lung and thyroid cancers – to the market.

Chief executive David Ricks previously said that Lilly would be actively pursuing deals in the oncology category, as it plays catch-up with early movers in immuno-oncology like Merck & Co, Bristol-Myers Squibb, Roche and AstraZeneca.

Merus will carry out discovery and early research on the bispecifics using its Biclonics development platform, which has already been used to generate a library of more than 175 anti-CD3 common light chain antibodies.

Thereafter, Lilly will take over for R&D on selected lead candidates and commercial activities should any of them reach the market.

“CD3-engaging bispecific antibodies are rapidly becoming one of the most transformative immune-modulating modalities used to treat cancer,” said Jacob Van Naarden, Loxo Oncology’s chief operating officer.

“Merus has built a differentiated platform and one that we believe can enable us to create bispecific antibody therapies with wider therapeutic indexes than those available today,” he added.

So far just one bispecific antibody has reached the market – Amgen’s Blincyto (blinatumomab) for B-cell precursor acute lymphoblastic leukaemia – but there are a host of others coming through the industry’s development pipeline.

Blincyto’s structure means it has a short half-life in the body and so needs daily dosing, but modifications to the structure of bispecifics means those coming through development are more like regular antibodies in their properties.

The post Lilly bolts on cancer bispecifics with $1.6bn Merus alliance appeared first on .

Careology joins forces with remote care support firm RedArc

Digital cancer care platform Careology has joined forces with RedArc, which provides long-term support for people with serious illnesses.

RedArc has a team of registered nurses that provide support and expert advice by phone to people after referral from insurers, trade unions and groups such as trade unions.

The new partnership provides RedArc nurses with digital tools that will help them care for cancer patients.

Using leading Bluetooth wearable and connected technology, the Careology platform allows RedArc nurses to quickly view real-time health and wellbeing data, including a patient’s temperature, heart rate, mood, journal entries and activity levels.

Access to this information can help provide consistent and effective practical advice and emotional support to patients remotely.

No financial details were disclosed about this latest deal.

In October, Careology linked with doctors at the private London General Practice, a private GP group with around 50,000 people on its books.

This allowed the doctors to remotely monitor patients.

Careology launched its remote monitoring service for cancer patients in the UK last summer, with Macmillan Cancer Support among early adopters of the tool.

The company points out that it offers a way to remotely manage or proactively support patients going through gruelling and often extremely toxic treatment regimes.

Patients themselves have to reach out if they have severe symptoms or side-effects at home.

It’s also a challenge for them to recall how they felt on particular days and assessing the impact and complications of treatment can be unreliable as results.

The system produces red flags and alerts in markers such as vital signs, systemic anti-cancer toxicities and medication adherence.

This allows early and proactive interventions to support the patient, with the potential of saving lives.

The company has its own dedicated NHS division headed by Dr Henry Carleton who has already established two software businesses that serve the health service.

The post Careology joins forces with remote care support firm RedArc appeared first on .

Conversa Health Expands Series B to $20M with Additional $8M in Funding

Backed by Northwell, Conversa Health Raises $12M for AI-Powered Chatbots

What You Should Know:

 – Conversa Health raises an additional
$8M, expanding its Series B round to $20M for its automated virtual care
platform, totaling $34M in funding to date.

– The Series B round, first announced at $12 million in
June, was co-led by Builders VC and Northwell Ventures, the corporate venture
arm of Northwell Health, New York’s largest healthcare provider with 23
hospitals and 800 outpatient facilities.

– Founded in 2014, Conversa enables health systems to
virtually engage, monitor and manage patients more effectively and efficiently
than ever before—for chronic care, acute discharge, perioperative, oncology,
OBGYN, prevention and wellness, and more. Conversa’s automated care platform
engages patients at high frequency and scale while triaging to
higher-touch/cost care venues when necessary, optimizing and improving the use
of telehealth e-visits, phone calls, and in-person consults.

– Conversa will use the additional Series B funds to
continue to scale its technology platform, expand its library of automated
virtual care digital pathways, and fuel growth with new and existing
customers. 


Scotland backs Roche’s Rozlytrek for ROS1 lung cancer

The Scottish Medicines Consortium (SMC) has given a green light to Roche’s Rozlytrek for a rare form of lung cancer, almost seven months after NICE backed the drug in England.

Rozlytrek (entrectinib) can now be used by the NHS in Scotland as a treatment option for ROS1-positive, advanced non-small cell lung cancer (NSCLC) not previously treated with ROS1 inhibitors.

ROS1 is a rare mutation found in fewer than 2% of NSCLC cases, and patients have few treatment options, especially when the disease has spread to the brain. NICE has previously estimated that roughly 412 patients across the UK are have NSCLC eligible for treatment with Rozlytrek.

The SMC cleared Rozlytrek via its Patient and Clinician Engagement (PACE) programme, a mechanism that brings agency reviewers, clinicians and patient group representatives together in order to discuss benefits of a medicine that may not be fully captured in the conventional appraisal process.

In the meeting, patients and clinicians said ROS1-positive NSCLC is often diagnosed at an advanced stage, so is associated with a short life expectancy, and there are limited treatment options available.

“This type of advanced NSCLC is a very rare and incurable lung cancer that often occurs in non-smokers and affects many under 60 years old,” commented Gemma Boni, head of lung cancer at Roche Products Ltd.

“Our commitment is to ensure that people in Scotland with lung cancer live longer and healthier lives, and today’s news shows how we are advancing science to achieve this,” she added.

In 2018, Pfizer’s ALK and ROS1 inhibitor Xalkori (crizotinib) was cleared by NICE in England – via the Cancer Drugs Fund (CDF) – as well as in Scotland by the SMC as a first-line option for treating ROS1-positive advanced NSCLC in adults.

Second-line treatment options include pemetrexed with carboplatin or other platinum doublet chemotherapy, with pemetrexed as a maintenance therapy.

Rozlytrek is also approved to treat people aged 12 years of age and older with solid tumours that have an NTRK gene fusion – regardless of where they appear in the body – and the SMC should decide whether that use is cost effective in the next couple of months. NICE has already approved Rozlytrek for that use in England via the CDF.

Other decisions

The SMC also backed NHS use for four other medicines in its January update, including Janssen’s Darzalex (daratumumab) for adults with newly-diagnosed multiple myeloma who are eligible for autologous stem cell transplant. NICE is due to deliver a verdict on this use for the drug in April.

Meanwhile, Rigel Pharmaceuticals’ Tavlesse (fostamatinib) – sold by Spain’s Grifols in Europe – can now be used in Scotland to treat chronic immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments. NICE is also conducting an appraisal of Tavlesse, but hasn’t yet set a timeframe for review.

Also backed were Takeda’s Adcetris (brentuximab vedotin) for systemic anaplastic large cell lymphoma (sALCL) – a use already approved by NICE in England – and Novartis’ Cosentyx (secukinumab) for non-radiographic axial spondyloarthritis which NICE expects to decide on in May.

The SMC rejected RAD Neurim Pharma’s Slenyto (melatonin) for the treatment of insomnia in children with autism spectrum disorder and rare neurogenetic disorder Smith-Magenis syndrome, ruling the company had not provided strong enough evidence to show it was cost-effective.

The post Scotland backs Roche’s Rozlytrek for ROS1 lung cancer appeared first on .

NHS and Kite sign access deal for Kite’s cancer cell therapy Tecartus

NHS patients in England will be among the first in the world to receive Gilead’s Tecartus cancer cell therapy for certain types of lymphoma, after the company’s specialist Kite unit struck a deal with NICE. 

Marketed as Tecartus (autologous anti-CD19-transduced CD3+) in Europe, the drug was approved in the EU in December for adults with relapsed or refractory mantle cell lymphoma after two or more lines of systemic therapy including a Bruton’s tyrosine kinase (BTK) inhibitor.

It has been approved in the US since July last year and like other CAR-Ts the one-time therapy is expensive at $373,000 a shot in the US.

NICE said in guidance that the treatment can be considered for those with relapsed or refractory mantle cell lymphoma, after treatment with drugs such as AbbVie/Janssen’s Imbruvica (ibrutinib).

Kite has signed a managed access agreement with NHS England that allows for funding via the Cancer Drugs Fund (CDF) at a commercially confidential discount, so more data can be collected for NICE’s cost-effectiveness calculations.

While in most cases reimbursement from the CDF results in regular NHS funding in the long run, manufacturers must usually produce convincing overall survival data before NICE gives this the go-ahead.

But as Bristol-Myers Squibb found out earlier this month with its Opdivo immunotherapy in head and neck cancer, NICE is prepared to say “no” in the absence of the required data after a period of funding on the CDF.

NICE said in a statement that it is looking for further data on progression-free survival, overall survival and age when treatment with Tecartus starts.

This will help reduce uncertainty in evidence while NHS is used on NHS patients.

The NHS has ten providers around the country which will be able to offer this treatment option.

Many parts of the country continue to experience pressures on critical care services, that are required for the administration of a CAR-T therapy and patients can travel to centres further afield to receive their treatment if necessary, NICE said.

There is no standard treatment for adults, who are usually in their 70s, with relapsed or refractory mantle cell lymphoma after a BTK inhibitor. A combination of rituximab, bendamustine and cytarabine (R BAC) is the most common treatment option.

Around 100 patients each year could be treated with this CAR-T therapy.

NHS England has been leading the way with funding of CAR-T (Chimeric Antigen Receptor T-cell) therapies after it became an early adopter of Novartis’ Kymriah the first approved drug from this class in September 2018 in acute lymphoblastic leukaemia (ALL).

CAR-T therapies are made by harvesting a patient’s T-cells, genetically engineering them to target cancer cells and reintroducing them into the body.

The post NHS and Kite sign access deal for Kite’s cancer cell therapy Tecartus appeared first on .

AZ/Daiichi Sankyo’s Enhertu gets second US cancer indication

AstraZeneca and Daiichi Sankyo’s Enhertu cancer drug has picked up a second indication, in patients who have stomach cancer and haven’t responded to Roche’s Herceptin (trastuzumab).

This latest indication adds to Enhertu first approval at the end of 2019 for advanced HER2-positive breast cancer after two or more HER2-targeted regimens.

Around one in five gastric cancers are HER2-positive, opening up a new market niche for AstraZeneca and Daiichi Sankyo, who have been working on Enhertu (trastuzumab deruxtecan) after signing a multi-billion partnership based on the drug in March 2019.

Under the terms of the companies’ partnership agreement, AZ is to pay Daiichi $115 million in a combined milestone payment covering second and third line HER-positive gastric cancer.

Sales of Enhertu in the US are recognised by Daiichi Sankyo. AstraZeneca reports its share of gross profit margin from Enhertu sales in the US as collaboration revenue in its financial statements.

The FDA approved the new use based on positive results from the randomised DESTINY-Gastric01 phase 2 trial conducted in Japan and South Korea.

In the trial, Enhertu demonstrated a statistically significant and clinically meaningful improvement in overall survival (OS) and objective response rate (ORR) versus chemotherapy (irinotecan or paclitaxel) in patients with advanced gastric cancer or gastroesophageal junction (GEJ) adenocarcinoma.

A pre-specified interim analysis showed patients treated with Enhertu had a 41% reduction in the risk of death versus patients treated with chemotherapy with a median OS of 12.5 months versus 8.4 months on chemotherapy.

Results showed a confirmed overall response rate (ORR) of 40.5% in 126 patients treated with Enhertu compared to 11.3% in patients treated with chemotherapy.

Patients treated with Enhertu had a 7.9% complete response rate and a 32.5% partial response rate compared to a complete response rate of 0% and a partial response rate of 11.3% for patients treated with chemotherapy.

It also showed a median progression-free survival (PFS) of 5.6 months compared to 3.5 months with chemotherapy.

Additionally, Enhertu showed a median duration of response (DoR) of 11.3 months versus 3.9 months with chemotherapy.

Enhertu is an antibody-drug conjugate, consisting of the trastuzumab antibody that targets HER2-positive cancers in Herceptin, with a cancer-killing payload.

The post AZ/Daiichi Sankyo’s Enhertu gets second US cancer indication appeared first on .

Ex-Merck & Co scientist accused of stealing trade secrets could face jail

A former Merck & Co scientist could face up to 10 years in prison after he was accused of stealing trade secrets relating to drugs including the cancer immunotherapy Keytruda by US authorities.

In a document outlining the charges against Shafat Quadri, of North Potomac, Maryland the US Department of Justice (DoJ) referred only to “one of the largest pharmaceutical companies in the world” based in New Jersey.

But supporting information refers to data from cancer drug Keytruda (pembrolizumab) and Quadri’s Linkedin profile shows he worked at New Jersey-based Merck & Co around the time of the incidents before taking a role at AstraZeneca.

Quadri, formerly Merck’s head of medical and scientific affairs, immune oncology, has been charged with one count of theft of trade secrets and one count of unauthorised transmission of trade secrets, some relating to the multi-billion dollar cancer immunotherapy Keytruda (pembrolizumab).

According to the DoJ, the company contacted the FBI in October 2019 to report suspicious activity by Quadri, who had been employed there since 2015 as director of medical and scientific affairs, immune oncology.

The company said an internal investigation found Quadri had copied and removed thousands of files containing proprietary information before he left in September 2019,

This included research protocols, data and strategic plans, using unauthorised USB devices and personal email accounts to transfer the information that he had access to as part of his job.

Some documents were copied and removed and the trade secrets were also sent to an email address controlled by his “subsequent employer” and one of Merck’s competitors, according to the document.

Quadri was not authorised to keep the documents, the DoJ noted, and the investigation showed the company regularly monitors its employees’ use of company-provided technology and systems.

The count of theft of trade secrets charge carries a maximum potential penalty of up to 10 years in prison and a fine of up to $250,000, or twice the gross pecuniary gain or loss.

The post Ex-Merck & Co scientist accused of stealing trade secrets could face jail appeared first on .

IO Biotech raises $155m to develop breakthrough cancer vaccine

IO Biotech, an oncology specialist formed and backed by Denmark’s Novo Holdings, has raised €127 million ($155 million) to further develop its cancer vaccine technology that has boosted efficacy of PD-1 immunotherapy in early trials.

The Series B fundraiser follows the FDA’s decision to grant Breakthrough Therapy designation last month for a combination of its lead immune-oncology therapies IO102 and IO103, with anti-PD-1 monoclonal antibodies for patients with metastatic melanoma.

IO Biotech intends to use the proceeds from the financing to advance clinical trials for its early and late-stage immune-oncology programs, including a large randomised trial for the IO102 and IO103 with anti-PD-1 combination in metastatic melanoma.

IO102 and IO103 are cancer first-in-class vaccines based on IO Biotech’s proprietary T-win technology platform.

The company said this enables the identification of compounds with a dual mechanism of action targeting and killing immunosuppressive cells and tumour cells while indirectly activating other T-effectors.

They are designed to engage and activate IDO and PD-L1 specific human T-cells, which leads to strong anti-tumour responses without adding additional safety concern.

The FDA’s decision to grant breakthrough therapy designation was based on data from the MM1636 phase 1/2 clinical trial of 30 patients with metastatic melanoma receiving IO102, IO103 and anti-PD-1 monoclonal antibodies.

According to the data recently presented in a late-breaking abstract at the European Society for Medical Oncology  Virtual Congress 2020, the combination of IO102 and IO103 vaccines and nivolumab was shown to be safe with encouraging early efficacy data.

An overall response rate (ORR) of 79% was reached and 45% of patients achieved a complete response (CR), or complete disappearance of their tumours. Vaccine specific T-cells were located in the peripheral blood mononuclear cells (PBMCs) and at the tumour site.

The financing round was led by HBM Healthcare Investments with the participation of existing investors Novo Seeds, Lundbeckfonden Emerge and Sunstone Life Science Ventures.

Other new investors joining the round included Vivo Capital, Kurma Partners, Avoro Capital, RA Capital, Samsara Biocapital, Idinvest Partners (Subsidiary of Eurazeo), PFM Health Sciences, Soleus Capital, Eir Ventures and Serrado Capital.

IO biotech was created and launched by Novo Seeds in 2015, which is the investment arm of Novo Holdings, a private company wholly owned by the Novo Nordisk Foundation.

The post IO Biotech raises $155m to develop breakthrough cancer vaccine appeared first on .

Boehringer to investigate ‘dark antigens’ with UK cancer specialist Enara

Boehringer Ingelheim has signed a strategic collaboration with UK biotech Enara Bio, focused on finding ways to fight cancer using “dark antigens” in a deal worth up to €876 million ($1.07bn).

Dark Antigens represent a new class of cancer-associated antigens that derive from the genomic “dark matter” – the portion of the human genome that is normally not expressed as protein.

These sequences are usually silenced in healthy cells but are activated and presented on tumour cells.

This makes them good targets for targeted therapies that will attack cancer cells while leaving healthy cells alone.

Dark antigens are associated with specific cancers and are shared across patients and since typically not visible to the immune system, they represent a large potential repertoire of novel antigens that can be developed as targets for new immunotherapies.

In the new collaboration, Boehringer wants to combine Enara’s expertise in antigen identification with its immune-oncology technology such as oncolytic viruses and cancer vaccines.

The discovery of shared antigens could lead to the development of vaccines that can be readily used to help a broader group of cancer patients.

Enara Bio’s proprietary technology will be used to discover and validate novel dark antigens in up to three tumour types in lung and gastrointestinal cancer.

Under the agreement, Boehringer Ingelheim has the option to license dark antigens discovered and validated by Enara Bio.

Boehringer will also be responsible for all non-clinical and clinical development, as well as marketing of associated cancer immunotherapies, including therapeutic vaccines and T-cell redirecting biologics.

Enara Bio retains rights to use any discovered antigens for use in cell therapy-based products.

Enara is eligible to receive an undisclosed upfront payment, together with research/preclinical milestones and licensing fees for each tumour type that is explored.

The Oxford-based biotech is also eligible to receive more than €876 million in clinical, regulatory and commercial milestones, in addition to royalties on future product sales.

Boehringer has been particularly active in cancer deal making, buying Swiss cancer biotech NBE-Therapeutics for €1.16bn ($1.43bn) in December, snapping up antibody-drug conjugate technology.

In the same week it bought cancer vaccine and oncolytic virus specialist Labor Dr. Merk & Kollegen.

The post Boehringer to investigate ‘dark antigens’ with UK cancer specialist Enara appeared first on .

bluebird bio to split into oncology and gene therapy specialists

US biotech bluebird bio has announced plans to split into two this year, with a separate oncology business spinning off as the company prepares to bring its products to market.

Under the plans the company’s rare disease drugs will remain under the aegis of bluebird with current genetic disease president Andrew Obenshain taking the reins as CEO.

Meanwhile the as-yet unnamed oncology company will spin off under the leadership of bluebird’s current chief executive Nick Leschly.

Leschly will also take the role of executive chair at bluebird, according to a company statement.

While bluebird has conducted pioneering work in gene therapy for blood disorders and in cancer cell therapy, products have been delayed by issues with filing data for the FDA.

Late last year, bluebird’s shares tanked after the FDA laid out additional manufacturing standards for its lead gene therapy product, Lentiglobin, in sickle cell anaemia that could hold up filing until late 2022.

Bluebird’s lead CAR-T cancer cell therapy idecabtagene vicleucel (ide-cel) was last year hit with an FDA refuse-to-file letter, which required additional data on chemistry, manufacturing and controls before reviewing the company’s dossier.

That made things difficult for development partner Bristol-Myers Squibb, which inherited the drug previously known as bb2121 through its acquisition of Celgene late in 2019.

The FDA is now due to make a decision on ide-cel as a treatment for multiple myeloma in late March.

Laying out the rationale for the spin-off, bluebird said that operating individually the two companies will be more effective at allocating capital.

The companies will be better equipped to deliver on goals and operations will be streamlined and simplified.

They will also be better at raising money with tailored investment theses and increased strategic flexibility.

The gene therapy firm will be focused on its most important therapies in beta-thalassemia, cerebral adrenoleukodystrophy and sickle cell disease in the US And Europe.

Zynteglo is already in beta-thalassemia in Europe, where the company will seek to expand access despite its hefty $1.8 million price tag.

On the oncology side, bluebird has also strengthened its board with the appointment of Dr Ramy Ibrahim, a high-profile leader in clinical development in immunotherapy and cell therapy.

Ibrahim is currently serving as a consultant for the Parker Institute for Cancer Immunotherapy (PICI) and built the clinical capabilities within the institute.

Ibrahim also worked as vice president and global therapeutic head for immuno-oncology at AstraZeneca and MedImmune, helping to develop the immunotherapy Yervoy (ipilimumab) at Bristol-Myers Squibb.

The post bluebird bio to split into oncology and gene therapy specialists appeared first on .

Frenova Begins Patient Enrollment to Build World’s Largest Genomics Registry for Kidney Disease

Frenova Begins Patient Enrollment to Build World’s Largest Genomics Registry for Kidney Disease

What You Should Know:

– Frenova Renal Research, a global division of Fresenius
Medical Care
, announced today that it has started to enroll patients in its
new endeavor to build the world’s largest genomics registry targeting kidney
disease.

– The registry will be used to help advance understanding
of the genetic drivers of kidney disease and shape more precise, individualized
therapies.


Fresenius
Medical Care,
the world’s leading provider of products and services for
people with chronic kidney failure, announced today that the company’s Frenova division has enrolled the first
participants in its new initiative to develop the largest renal-focused genomic
registry in the world. In addition, the company announced that Ali Gharavi, MD,
Chief of the Division of Nephrology at Columbia University Irving Medical
Center and Professor of Medicine at Columbia University Vagelos College of
Physicians and Surgeons, will lead the project and provide scientific guidance
as Principal Investigator.

Why It Matters

Nephrology has been under-represented in clinical research,
even as rapid progress in gene sequencing and analysis has led to advances in
precision medicine and individualized care in oncology, cardiology and other
medical areas. Frenova’s new genomic registry will contain genetic sequencing
data from chronic kidney disease patients worldwide, which will be used by
researchers to improve the understanding of kidney disease. Frenova developed
the registry after researchers identified the lack of a large-scale,
renal-focused registry of genomic and clinical data as a major impediment to
kidney disease research.

As a contract clinical development services company
dedicated exclusively to medicines and medical products in renal research,
Frenova orchestrates studies within the clinical footprint of Fresenius Medical
Care, which provides dialysis treatments to about 350,000 patients around the
globe. The renal-focused genomic registry represents a new business line within
Frenova, which is based in Fresenius Medical Care’s Global Medical Office. As
part of its growth strategy 2025, Fresenius Medical Care is using digital
technologies and the capability to analyze huge amounts of data to develop
new forms of renal therapy.

“The new Frenova registry will close this gap by generating data that adds a clinical and genetic backbone to help support and fuel scientific innovation,” said Franklin W. Maddux, MD, Global Chief Medical Officer of Fresenius Medical Care. “The evidence for genetic drivers in kidney diseases is substantial, but much larger data sets will be needed to untangle the complex interactions that lead to kidney injury. By combining clinical and genetic sequencing data from ethnically and pathologically diverse participants, this genomic and phenotypic research resource will help scientists better understand how genetic variations in patients can lead to more precise diagnoses and therapies that help improve outcomes by individualizing care.”

Chi-Med agrees R&D partnership with Inmagene

China’s Chi-Med and Inmagene Biopharmaceuticals have agreed a strategic partnership to develop preclinical drug candidates for potential treatment of several immunological diseases.

Funded by Inmagene, the companies will work together to move drug candidates towards regulatory filings with the FDA.

Chi-Med, full name Hutchison China MediTech, has granted Inmagene exclusive options to four drug candidates only for treatment of immunological diseases.

If Inmagene decides to exercise the option, it will have the right to further develop, manufacture and market that specific drug candidate globally.

Chi-Med will retain first rights to co-market in mainland China and for each drug candidate will be entitled to development milestone payments of up to $95 million, plus commercial milestones of up to $135 million, plus double-digit sales royalties.

Inmagene has wholly-owned subsidiaries in Shanghai, San Diego and Hangzhou, and is focused on building its drugs pipeline.

It’s lead candidate, IMG-020, is about to enter global clinical trials in several different indications that the company has not specified.

Chi-Med has been working on targeted therapies for immune diseases and cancer and has a portfolio of nine cancer drugs in clinical studies around the world.

Chi Med had been working with AstraZeneca on a late-stage oncology drug, savolitinib, for renal cell carcinoma.

However, the UK pharma opted to dump the MET inhibitor drug from its pipeline after the Chi-Med said that it looked unlikely to beat Pfizer’s Sutent in papillary renal cell carcinoma.

Chi-Med has also worked with Eli Lilly on another investigational cancer drug, fruquintinib.

Christian Hogg, chief executive officer of Chi-Med, said, “This partnership will enable Inmagene to investigate the immunological disease applications of these four drug candidates, discovered by our in-house discovery organisation.

“We believe that these four candidates have scope in multiple immunological diseases and we are pleased to see these opportunities investigated further by Inmagene.”

The post Chi-Med agrees R&D partnership with Inmagene appeared first on .

Oxford BioTherapeutics to research cell therapies for Gilead’s Kite

Gilead Sciences’ Kite unit has teamed up with the UK’s Oxford BioTherapeutics (OBT) to develop a new clutch of cell therapy products for solid tumours and blood cancers.

The partnership covers up to five oncology targets identified by OBT using its discovery platform, and the UK firm will try to develop antibodies against them. Kite and Gilead will then develop and commercialise therapies based on these targets or antibodies.

“Selecting the right target is fundamental for the successful development of first-in-class cell therapies,” said OBT chief executive Dr Christian Rohlff.

“We are delighted that Kite, the global leader in cell therapy, has recognised the potential of OBT’s OGAP discovery platform and antibody capabilities through this partnership.”

Under the agreement, OBT will receive an undisclosed upfront payment and may receive receive additional payments based on the achievement of discovery, clinical, and regulatory milestones, as well as royalties on any sales coming down the line.

Gilead acquired Kite in 2017 for almost $12 billion, at a time when enthusiasm for cell therapies for cancer, and particularly CAR-T, was riding high on the back of unprecedented, life-saving efficacy in trials involving patients with few treatment options left.

The commercial reality for CAR-Ts has been more challenging, and as the pipeline has become more crowded Gilead has cut the value of the experimental assets it acquired along with Kite on more than one occasion to the tune of around $1.6 billion.

The main draw for the Kite takeover for Gilead was lead CAR-T Yescarta (axicabtagene ciloleucel), approved for large B-cell lymphoma shortly after the company made its move, which was joined by Tecartus (brexucabtagene autoleucel) for mantle cell lymphoma last July.

Problems with tolerability, reimbursement and a complex manufacturing process has pegged back sales of CAR-Ts from earlier expectations, but Yescarta reached $450 million in 2019, and nearly matched figure that in the first nine months of 2020, with Tecartus adding a modest $9 million to the pot.

Gilead and Kite remain convinced that cell therapy can become the cornerstone of cancer treatment, however, and the deal with OBT isn’t the first that has boosted Kite’s portfolio.

In April last year, for example, Kite licensed a suite of antibodies from Teneobio against BCMA – a key target for multiple myeloma therapy – that will be used to generate CAR-T treatments for the blood cancer.

Last September it forged a second alliance with HiFiBiO Therapeutics to find novel targets and antibodies for acute myeloid leukaemia (AML), adding to an earlier agreement focusing on T cell receptor (TCR) therapies for cancer.

In 2018 it also formed a partnership with Gadeta of the Netherlands to develop new forms of CAR-T with greater activity against solid tumours.

The post Oxford BioTherapeutics to research cell therapies for Gilead’s Kite appeared first on .

Why cell therapy manufacture is a team sport

Louis van de Wiel, Vice President, Site Head EU Manufacturing, Kite, a Gilead Company, reveals the complexity that sits behind the process of individualised cell therapy – and why team culture makes it work

This thought leadership series has been paid and developed by Kite, a Gilead Company.

In 2018, we were preparing to build a European facility to produce individualised cell therapies for the treatment of cancer.

Roll on two years and the team has achieved what at the time appeared a major challenge, putting 1,000 tonnes of steel, 1,800 solar panels and 176km of network cable into the creation of a centre of excellence for cell therapy near Amsterdam in the Netherlands.

In my experience, completing the design and build of a facility such as this, through to qualification, licence and becoming fully operational, would normally take four to five years. Our ambition was always to do this within two years, a goal we achieved despite the unprecedented challenge of a global coronavirus pandemic.

Indeed, while undoubtedly putting new hurdles in our path, the arrival of COVID-19 into our lives has transformed our business and operations by presenting an opportunity to be adaptable, flexible and responsive – and to continually evaluate and mitigate risk.

“There is huge complexity involved in cell therapy manufacture, with hundreds of personnel responsible for ensuring the quality and supply of an individual patient’s cells”

It’s been a complex process that’s required highly technical and skilled personnel. Not only did we build a specialised facility from the ground up, but we built an organisation, from 10-15 people two years ago to more than 400 now. We put energy and emphasis into creating the right team and a culture where everyone understands the values and drivers, allowing us to operate in a collaborative and cohesive way.

Now the new €130 million, 19,000m2 manufacturing facility near Amsterdam is able to support delivery of up to 4,000 cell therapies each year for eligible cancer patients across Europe.

But backtrack to August 2018 and the very first European patients were also receiving treatment, part of an expertly crafted operation that ran in parallel to the build. Our supply chain group worked with the existing US team to manage the shipment of patients’ cells to the US for modification and their return for treatment.

Having a fully operational site in Europe versus the US has several advantages; reducing transportation time, strengthening the chain of custody and, potentially, cutting lead time to the patient by approximately one week. This allows us to potentially provide the therapy quicker for eligible cancer patients who have stopped responding to or have progressed despite other treatments. At this stage of their disease, for patients who have no other options, a week can make a difference.

The journey of the cell

The very nature of cell therapy manufacture means employees work in tightly controlled environments to ensure adherence to good manufacturing practice standards and, ultimately, to ensure the quality and integrity of the product.

Ultimately, it’s a team sport between Kite and Gilead and the 100-plus qualifying hospitals across Europe, all of which have been individually trained and assessed to ensure they are fully compliant with the necessary procedures and meet exacting standards.

So, what does the journey of the cell look like?

To achieve consistent, timely delivery of a high-quality product requires a robust and efficient approach to engineering patient’s own T cells, which in itself encompasses apheresis, cell modification and final formulation – coupled with rigorous quality control testing throughout – reflecting the highly complex nature of the manufacturing process.

Understandably, teamwork is vital and requires an integrated network and seamless communication between Kite and the treating hospital. The journey starts with the hospital making a treatment reservation through KiteKonnect and shipping of the apheresis kit to enable the process of extracting the patient’s own white blood cells, kickstarting both the chain of identity and chain of custody.

Here, our quality and supply chain experts are integral to every stage of the cell therapy manufacturing continuum to ensure the product is returned to the patient in a timely manner.

As soon as apheresis has completed, the cells are shipped in temperature-controlled conditions to our facility near Amsterdam where they are assessed for quality and condition. One patient equals one individual treatment, so it is critical to preserve the chain of custody and chain of identity to ensure the product comes back to the same patient.

Why chain of custody and identity is critical

The chain of custody and chain of identity must, therefore, go hand-in-hand. In this way, not only do we know which cells belong to which patient, but we have precise location and up-to-the-minute feedback on storage conditions to ensure quality and safety is paramount at all times.

Once the cells have completed this first stage, the manufacturing process can begin, with T cell selection, activation, and genetic modification using viral vector technology to ensure the ability to recognise the patient’s cancer cells. Cell expansion follows to multiply the modified cells into their millions.

Further critical quality testing then takes place to ensure the cells are of a required standard and to create a finished purified product, which will be stored and returned to the originator hospital in temperature-controlled conditions (see diagram below).

Several quality attributes will be tested at this stage and the cells must meet these rigorous criteria and specifications. There is huge complexity involved in cell therapy manufacturing, with hundreds of personnel responsible for ensuring the quality and supply of an individual patient’s cells.

As part of this process, the supply chain team simultaneously coordinate with the hospital to prepare the individual so when the cells are infused back to the patient they are primed to potentially fight the cancer.

Individualising the approach

In stark contrast to basic biopharmaceutical products with a robust starting material, the cells of a patient with cancer who has already undergone multiple treatments will not have the same quality. Consequently, there can be unforeseen hurdles during the process and I am proud that the team has managed each situation to safeguard the patient’s cells and ensure they receive treatment in an efficient and timely way.

This is particularly important when you consider the turnaround for each individualised product from starting material to the patient is typically four weeks – versus months or even years for a standard biopharmaceutical product.

Additionally, each patient equals one product batch – we do not keep inventory – and the potential impact on the patient if something happens to that batch is why we are so passionate. From quality manufacturing, facility engineering, supply chain, we’re driven to make sure the batch is returned to the patient safely and effectively.

What of the future? For me, it’s all about leadership, clarity, direction, and commitment of the entire team. It’s about the opportunity to be involved in an innovative field of cancer therapy where the body is stimulated to fight cancer cells. It’s about optimising the manufacturing process to become more effective and efficient. But, most of all, it’s about the patients, their care partners and families

 

This was supported by Kite, a Gilead Company
UK-CTH-2020-11-0075 | Date of preparation: December 2020

The post Why cell therapy manufacture is a team sport appeared first on .

Roche nabs breakthrough tag for TIGIT cancer immunotherapy

Roche’s closely-watched combination of two checkpoint inhibitors – TIGIT-targeting tiragolumab and PD-L1 drug Tecentriq – has claimed breakthrough status from the FDA.

Like PD-L1, TIGIT is thought to act as a molecular brake that stops T cells from attacking tumours, and tiragolumab is currently leading the pack among drugs targeting the immune checkpoint.

The FDA’s breakthrough status is for tiragolumab plus Tecentriq is the first for a TIGIT drug, and covers the first-line treatment of patients with advanced-stage non-small cell lung cancer (NSCLC) that expresses high levels of PD-L1.

It’s been granted on the strength of data from the phase 2 CITYSCAPE study – reported at the ASCO congress last year – that showed the pairing shrank tumours in 31% of patients, twice the response seen with Tecentriq (atezolizumab) alone.

First-line immunotherapy for NSCLC is dominated by Merck & Co’s PD-1 inhibitor Keytruda (pembrolizumab), transforming the prospects of some patients and propelling the drug to multibillion-dollar sales.

PD-1 or PD-L1 targeting immunotherapy only works in around a third of cases, however, and Roche’s hope is that tiragolumab could boost that proportion and encourage greater use of Tecentriq in first-line NSCLC.

Roche’s drug is already FDA-approved as a monotherapy and in combination with Avastin (bevacizumab) and chemotherapy for previously-untreated NSCLC with high PD-L1 expression.

Main rival Keytruda however retains one advantage – a label that include the treatment of patients with low levels of the biomarker – which means that it can be used without chemotherapy in nearly all the first-line NSCLC population.

Ramping up the immune response to cancer with a second checkpoint inhibitor for use alongside the PD-1/PD-L1 inhibitors is on the wish list of many oncologists, although there have already been some big setbacks, such as the demise of Incyte’s IDO1 inhibitor epacadostat in 2018.

Bristol-Myers Squibb’s CLTA4 inhibitor Yervoy (ipilimumab) is already in use for some cancers, including NSCLC in combination with BMS’ PD-1 drug Opdivo (nivolumab), but has been held up by tolerability issues. Meanwhile, drugs targeting TIM-3, LAG3, VISTA and STING also coming through development.

TIGIT is very much among the front runners however, and Roche is being closely pursued by other drug developers including Merck & Co with MK-7684 and Gilead/Arcus, which are also in mid-stage testing, as well as iTeos Therapeutics and Mereo BioPharma.

Another rival – Astellas – fell out of the running last year when it abandoned its phase 1 candidate ASP8374, acquired as part of its $405 million Potenza takeover in 2018.

Roche has already started two phase 3 trials testing the tiragolumab with Tecentriq in lung cancer, and also has half a dozen earlier-stage studies running in other solid tumours as well as blood cancers.

“We have been researching TIGIT as a novel cancer immunotherapy target for almost 10 years and we are pleased that the FDA has acknowledged the potential of tiragolumab to substantially improve outcomes for people with certain types of lung cancer,” said Levi Garraway, Roche’s chief medical officer.

The post Roche nabs breakthrough tag for TIGIT cancer immunotherapy appeared first on .

2020’s Top 20 Digital Health M&A Deals Totaled $50B

Teladoc Health and Livongo Merge

2020’s Top 20 Digital Health M&A Deals Totaled $50B

The combination of Teladoc Health and Livongo creates a
global leader in consumer-centered virtual care. The combined company is
positioned to execute quantified opportunities to drive revenue synergies of
$100 million by the end of the second year following the close, reaching $500
million on a run-rate basis by 2025.

Price: $18.5B in value based on each share of Livongo
will be exchanged for 0.5920x shares of Teladoc Health plus cash consideration
of $11.33 for each Livongo share.


Siemens Healthineers Acquires Varian Medical

2020’s Top 20 Digital Health M&A Deals Totaled $50B

On August 2nd, Siemens Healthineers acquired
Varian Medical for $16.4B, with the deal expected to close in 2021. Varian is a
global specialist in the field of cancer care, providing solutions especially
in radiation oncology and related software, including technologies such as
artificial intelligence, machine learning and data analysis. In fiscal year 2019,
the company generated $3.2 billion in revenues with an adjusted operating
margin of about 17%. The company currently has about 10,000 employees
worldwide.

Price: $16.4 billion in an all-cash transaction.


Gainwell to Acquire HMS for $3.4B in Cash

2020’s Top 20 Digital Health M&A Deals Totaled $50B

Veritas Capital (“Veritas”)-backed Gainwell Technologies (“Gainwell”),
a leading provider of solutions that are vital to the administration and
operations of health and human services programs, today announced that they
have entered into a definitive agreement whereby Gainwell will acquire HMS, a technology, analytics and engagement
solutions provider helping organizations reduce costs and improve health
outcomes.

Price: $3.4 billion in cash.


Philips Acquires Remote Cardiac Monitoring BioTelemetry for $2.8B

2020’s Top 20 Digital Health M&A Deals Totaled $50B

Philips acquires BioTelemetry, a U.S. provider of remote
cardiac diagnostics and monitoring for $72.00 per share for an implied
enterprise value of $2.8 billion (approx. EUR 2.3 billion). With $439M in
revenue in 2019, BioTelemetry annually monitors over 1 million cardiac patients
remotely; its portfolio includes wearable heart monitors, AI-based data
analytics, and services.

Price: $2.8B ($72 per share), to be paid in cash upon
completion.


Hims & Hers Merges with Oaktree Acquisition Corp to Go Public on NYSE

Telehealth company Hims & Hers and Oaktree Acquisition Corp., a special purpose acquisition company (SPAC) merge to go public on the New York Stock Exchange (NYSE) under the symbol “HIMS.” The merger will enable further investment in growth and new product categories that will accelerate Hims & Hers’ plan to become the digital front door to the healthcare system

Price: The business combination values the combined
company at an enterprise value of approximately $1.6 billion and is expected to
deliver up to $280 million of cash to the combined company through the
contribution of up to $205 million of cash.


SPAC Merges with 2 Telehealth Companies to Form Public
Digital Health Company in $1.35B Deal

2020’s Top 20 Digital Health M&A Deals Totaled $50B

Blank check acquisition company GigCapital2 agreed to merge with Cloudbreak Health, LLC, a unified telemedicine and video medical interpretation solutions provider, and UpHealth Holdings, Inc., one of the largest national and international digital healthcare providers to form a combined digital health company. 

Price: The merger deal is worth $1.35 billion, including
debt.


WellSky Acquires CarePort Health from Allscripts for
$1.35B

2020’s Top 20 Digital Health M&A Deals Totaled $50B

WellSky, global health, and community care technology company, announced today that it has entered into a definitive agreement with Allscripts to acquire CarePort Health (“CarePort”), a Boston, MA-based care coordination software company that connects acute and post-acute providers and payers.

Price: $1.35 billion represents a multiple of greater
than 13 times CarePort’s revenue over the trailing 12 months, and approximately
21 times CarePort’s non-GAAP Adjusted EBITDA over the trailing 12 months.


Waystar Acquires Medicare RCM Company eSolutions

2020’s Top 20 Digital Health M&A Deals Totaled $50B

On September 13th, revenue cycle management
provider Waystar acquires eSolutions, a provider of Medicare and Multi-Payer revenue
cycle management, workflow automation, and data analytics tools. The
acquisition creates the first unified healthcare payments platform with both
commercial and government payer connectivity, resulting in greater value for
providers.

Price: $1.3 billion valuation


Radiology Partners Acquires MEDNAX Radiology Solutions

2020’s Top 20 Digital Health M&A Deals Totaled $50B

Radiology Partners (RP), a radiology practice in the U.S., announced a definitive agreement to acquire MEDNAX Radiology Solutions, a division of MEDNAX, Inc. for an enterprise value of approximately $885 million. The acquisition is expected to add more than 800 radiologists to RP’s existing practice of 1,600 radiologists. MEDNAX Radiology Solutions consists of more than 300 onsite radiologists, who primarily serve patients in Connecticut, Florida, Nevada, Tennessee, and Texas, and more than 500 teleradiologists, who serve patients in all 50 states.

Price: $885M


PointClickCare Acquires Collective Medical

2020’s Top 20 Digital Health M&A Deals Totaled $50B

PointClickCare Technologies, a leader in senior care technology with a network of more than 21,000 skilled nursing facilities, senior living communities, and home health agencies, today announced its intent to acquire Collective Medical, a Salt Lake City, a UT-based leading network-enabled platform for real-time cross-continuum care coordination for $650M. Together, PointClickCare and Collective Medical will provide diverse care teams across the continuum of acute, ambulatory, and post-acute care with point-of-care access to deep, real-time patient insights at any stage of a patient’s healthcare journey, enabling better decision making and improved clinical outcomes at a lower cost.

Price: $650M


Teladoc Health Acquires Virtual Care Platform InTouch
Health

2020’s Top 20 Digital Health M&A Deals Totaled $50B

Teladoc Health acquires InTouch Health, the leading provider of enterprise telehealth solutions for hospitals and health systems for $600M. The acquisition establishes Teladoc Health as the only virtual care provider covering the full range of acuity – from critical to chronic to everyday care – through a single solution across all sites of care including home, pharmacy, retail, physician office, ambulance, and more.

Price: $600M consisting of approximately $150 million
in cash and $450 million of Teladoc Health common stock.


AMN Healthcare Acquires VRI Provider Stratus Video

2020’s Top 20 Digital Health M&A Deals Totaled $50B

AMN Healthcare Services, Inc. acquires Stratus Video, a leading provider of video remote language interpretation services for the healthcare industry. The acquisition will help AMN Healthcare expand in the virtual workforce, patient care arena, and quality medical interpretation services delivered through a secure communications platform.

Price: $475M


CarepathRx Acquires Pharmacy Operations of Chartwell from
UPMC

2020’s Top 20 Digital Health M&A Deals Totaled $50B

CarepathRx, a leader in pharmacy and medication management
solutions for vulnerable and chronically ill patients, announced today a
partnership with UPMC’s Chartwell subsidiary that will expand patient access to
innovative specialty pharmacy and home infusion services. Under the $400M
landmark agreement, CarepathRx will acquire the
management services organization responsible for the operational and strategic
management of Chartwell while UPMC becomes a strategic investor in CarepathRx. 

Price: $400M


Cerner to Acquire Health Division of Kantar for $375M in
Cash

Cerner announces it will acquire Kantar Health, a leading
data, analytics, and real-world evidence and commercial research consultancy
serving the life science and health care industry.

This acquisition is expected to allow Cerner’s Learning
Health Network client consortium and health systems with more opportunities to
directly engage with life sciences for funded research studies. The acquisition
is expected to close during the first half of 2021.

Price: $375M


Cerner Sells Off Parts of Healthcare IT Business in
Germany and Spain

2020’s Top 20 Digital Health M&A Deals Totaled $50B

Cerner sells off parts of healthcare IT business in Germany and Spain to Germany company CompuGroup Medical, reflecting the company-wide transformation focused on improved operating efficiencies, enhanced client focus, a refined growth strategy, and a sharpened approach to portfolio management.

Price: EUR 225 million ($247.5M USD)


CompuGroup Medical Acquires eMDs for $240M

2020’s Top 20 Digital Health M&A Deals Totaled $50B

CompuGroup Medical (CGM) acquires eMDs, Inc. (eMDs), a
leading provider of healthcare IT with a focus on doctors’ practices in the US,
reaching an attractive size in the biggest healthcare market worldwide. With
this acquisition, the US subsidiary of CGM significantly broadens its position
and will become the top 4 providers in the market for Ambulatory Information
Systems in the US.

Price: $240M (equal to approx. EUR 203 million)


Change Healthcare Buys Back Pharmacy Network

2020’s Top 20 Digital Health M&A Deals Totaled $50B

Change
Healthcare
 buys
back
 pharmacy unit eRx Network
(“eRx”),
 a leading provider of comprehensive, innovative, and secure
data-driven solutions for pharmacies. eRx generated approximately $67M in
annual revenue for the twelve-month period ended February 29, 2020. The
transaction supports Change Healthcare’s commitment to focus on and invest in
core aspects of the business to fuel long-term growth and advance innovation.

Price: $212.9M plus cash on the balance sheet.


Walmart Acquires Medication Management Platform CareZone

2020’s Top 20 Digital Health M&A Deals Totaled $50B

Walmart acquires CareZone, a San Francisco, CA-based smartphone
service for managing chronic health conditions for reportedly $200M. By
working with a network of pharmacy partners, CareZone’s concierge services
assist consumers in getting their prescription medications organized and
delivered to their doorstep, making pharmacies more accessible to individuals
and families who may be homebound or reside in rural locations.

Price: $200M


Verisk Acquires MSP Compliance Provider Franco Signor

2020’s Top 20 Digital Health M&A Deals Totaled $50B

Verisk, a data
analytics provider, announced today that it has acquired Franco Signor, a Medicare Secondary Payer
(MSP) service provider to America’s largest insurance carriers and employers.
As part of the acquisition, Franco Signor will become part of Verisk’s Claims
Partners business, a leading provider of MSP compliance and other analytic
claim services. Claims Partners and Franco Signor will be combining forces to
provide the single best resource for Medicare compliance. 

Price: $160M


Rubicon Technology Partners Acquires Central Logic

2020’s Top 20 Digital Health M&A Deals Totaled $50B

Private equity firm Rubicon Technology Partners acquires
Central Logic, a provider of patient orchestration and tools to accelerate
access to care for healthcare organizations. Rubicon will be aggressively driving Central Logic’s
growth with additional cash investments into the business, with a focus
on product innovation, sales expansion, delivery and customer support, and
the pursuit of acquisition opportunities.

Price: $110M – $125 million, according to sources


Myovant Signs Agreement with Pfizer to Develop and Commercialize Relugolix in Oncology and Women’s Health

Shots:

  • Myovant & Pfizer will jointly develop & commercialize ORGOVYX (relugolix) in advanced prostate cancer & if approved, relugolix combination tablet in women’s health in the US & Canada. Myovant will receive ~$4.2B including $650M upfront, $200M in regulatory milestones for FDA approvals for relugolix combination tablet in women’s health, and tiered sales milestones upon reaching certain thresholds up to $2.5 billion in net sales for prostate cancer and women’s health indications
  • Additionally, Pfizer will receive an exclusive option to commercialize relugolix in oncology outside the US and Canada, excluding certain Asian countries and if Pfizer exercises the option to commercialize relugolix in oncology, Myovant will receive $50M and is eligible to receive double-digit royalties.
  • Relugolix is a GnRH receptor antagonist that reduces testicular testosterone, a hormone known to stimulate the growth of prostate cancer, and ovarian estradiol. Relugolix (120mg) is FDA approved as ORGOVYX for adult patients with advanced prostate cancer

Click here ­to­ read full press release/ article | Ref: GlobeNewswire | Image: Myovant

The post Myovant Signs Agreement with Pfizer to Develop and Commercialize Relugolix in Oncology and Women’s Health first appeared on PharmaShots.

BMS calls time on brain cancer trial as Opdivo misses second target

Bristol Myers-Squibb has put out another downbeat “update” announcement about its immunotherapy Opdivo in an aggressive form of brain cancer, saying that it looks unlikely to produce a survival benefit in the disease.

BMS said an independent committee had reviewed data from the phase 3 CheckMate-548 trial in newly diagnosed MGMT-methylated glioblastoma multiforme and concluded that it would be unlikely to meet its primary overall survival (OS) goal.

The company already announced in September last year that the trial had missed its other primary endpoint of progression-free survival in all randomised patients.

There were no safety concerns that warranted stopping of the study, which will be unblinded to investigators following the announcement.

Patients will be advised on their treatment options, and those currently deriving benefit from Opdivo (nivolumab) are permitted to continue treatment if agreed to with their doctor.

After a complete evaluation of trial results, investigators will share final results with the oncology community.

CheckMate-548 is testing Opdivo plus current standard of care (temozolomide and radiation therapy) versus placebo plus standard of care, in patients with newly diagnosed GBM with MGMT promoter methylation.

The secondary endpoints are investigator assessed PFS, and OS rate at 12 and 24 months.

The news comes as former Celgene shareholders wait to see whether the FDA will approve a cancer cell therapy ahead of a deadline on New Year’s Eve.

Celgene shareholders were given a tradeable three-drug bet known as a Contingent Value Right (CVR) for every share they owned when the companies merged in November last year.

Three drugs need to be approved before certain deadlines for each CVR to pay out $9 – the MS drug Zeposia (ozaonimod) has already been met earlier this year.

The cancer cell therapy for lymphoma known as liso-cel must be approved by the FDA New Year’s Eve, followed by another CAR-T ide-cel on March 31 next year.

Both of these deadlines are under threat, with the FDA waiting on results from an outstanding factory inspection after the regulator delayed its review by three months by three months in May.

That inspection happened a few weeks ago and there renewed hopes the FDA will okay the drug in time for the deadline.

It will also be a close-run thing with multiple myeloma drug ide-cel, which has a decision date just four days before the final 31st March deadline.

The post BMS calls time on brain cancer trial as Opdivo misses second target appeared first on .

Roche claims EU okay for Herceptin/Perjeta combination Phesgo

Roche has secured EU approval for Phesgo, a fixed-dose combination of its breast cancer drugs Herceptin and Perjeta that is easier and cheaper to administer to patients – and also provides a defence against biosimilar competition.

Phesgo combines the anti-HER2 antibodies in Herceptin (trastuzumab) and Perjeta (pertuzumab) in a subcutaneous injection that takes a few minutes to deliver.

At the moment, Herceptin is available as a subcutaneous injection but Perjeta needs to be delivered by intravenous infusion – a process that takes hours – and Roche says that means Phesgo is preferred by both patients and doctors and also reduces the cost of treatment.

Phesgo has been approved by the EU for same indications as Herceptin/Perjeta – in combination with chemotherapy in early HER2-positive breast cancer (pre- and post-surgery) as well as for front-line therapy of HER2-positive breast cancer that has already spread in the body.

The drug was cleared by the FDA in June for the US market and made CHF 7 million (almost $8 million) in preliminary sales in the third quarter, with Roche still in the process of securing formulary approvals.

That’s a drop in the ocean compared to the CHF 1.5 billion and CHF 910 million in Herceptin and Perjeta sales, respectively, in the same period, but with Phesgo Roche is looking to the future.

Herceptin sales dropped a third in the first nine months of the year due in part to the pandemic but mainly the impact of low-cost biosimilars, although Perjeta remains in-patent for the next few years and is growing fast – thanks largely to data from the APHINITY trial which showed that adding it to Herceptin can improve disease-free survival in early-stage breast cancer.

That makes Perjeta a particularly important part of Roche’s product range, particularly as two of its other big-selling antibodies – Avastin (bevacizumab) and Rituxan/MabThera (rituximab) – are also being hit by biosimilar competition.

As many patients with HER2-positive breast cancer received the drug in combination with Perjeta, the convenience of Phesgo is a big draw – patients can be in and out of clinic in 30 minutes rather than three hours or more.

It remains to be seen of course whether payers can be convinced to plump for the new drug over biosimilar trastuzumab plus Perjeta, allowing the drug to mitigate the impact of Herceptin biosimilars on Roche’s HER2 franchise.

“The innovation of Phesgo significantly reduces the time people spend receiving standard of care therapy with Perjeta and Herceptin, helping to minimise the impact of treatment on their everyday lives,” said Levi Garraway, Roche’s chief medical officer.

“It also addresses the increasing demand across healthcare systems for faster and more flexible treatment solutions.”

After the US approval, Piper Sandler analyst Joseph Catanzaro said that Phesgo could be an underappreciated blockbuster product that will be a growth driver for Halozyme, the company that provided the drug delivery technology that underpins the new product.

The post Roche claims EU okay for Herceptin/Perjeta combination Phesgo appeared first on .

Servier beefs up in cancer, buying Agios’ oncology business for $1.8bn

French pharmaceutical company Servier has swooped on Agios Pharma’s marketed and experimental cancer drugs, buying the entire franchise in a deal that leaves the US biopharma focused on genetic disorders.

It’s a major change of direction for Boston-based Agios, which has been concentrating on the oncology market since its inception in 2008, bringing the Bristol Myers Squibb-partnered Tibsovo (ivosidenib) for IDH1-mutant acute myeloid leukaemia to the US market.

It also shares commercial rights to BMS’ Idhifa (enasidenib), also for AML, and according to Bloomberg the two drugs should bring in around $244 million in revenues to Agios’ top line this year. Agios hit a setback for Tibsovo in Europe this year however after the EMA rejected the marketing application for the drug.

Servier is paying $1.8 billion upfront for the oncology assets, with the total value of the deal potentially rising to $2 billion if Agios’ key pipeline candidate vorasidenib – a dual inhibitor of mutated IDH1 and IDH2 in trials for a form of brain cancer called glioma – delivers as expected.

That will give Agios the financial resources it needs to develop mitapivat, a potential treatment for the inherited disease pyruvate kinase (PK) deficiency, as well as sickle cell disease and beta thalassaemia, that hit the mark in a phase 3 trial earlier this month. It’s hoping to launch mitapivat in the US and Europe in 2022.

With Tibsovo, Servier gets a drug that hasn’t made the headway expected in the marketplace since its launch two years ago, although the French company will be able to ramp up the commercial drive for the drug through its own oncology sales force – which in the US will now be bulked up by Agios reps.

Tibsovo also has potential in newly-diagnosed AML, which would expand the market for the drug significantly.

Cancer is a key area for the French company, which sells products like Pixuvri (pixantrone) for non-Hodgkin lymphoma (NHL) and colorectal cancer therapy Lonsurf (trifluridine/tipiracil) and devotes around half its R&D budget to oncology with around a dozen candidates in clinical development.

The Agios deal comes just a day after Servier announced a strategic alliance with Celsius Therapeutics to find new drug targets for colorectal cancer. This year it’s also doubled down on an alliance with off-the-shelf CAR-T specialist Cellectis, and bought Danish cancer biotech Symphogen.

Agios will retain a royalty stream on Tibsovo and vorasidenib of 5% and 15% respectively until the drugs lose their patent protection.

Agios CEO Jackie Fouse said in a statement: “The result of a deliberative strategic review, this decision reflects the progress we have made understanding and harnessing the science and promise of PK activation and captures the full value of our oncology assets.”

The post Servier beefs up in cancer, buying Agios’ oncology business for $1.8bn appeared first on .

Chasing BMS, J&J files BCMA CAR-T for multiple myeloma to FDA

Johnson & Johnson’s Janssen unit has filed a rolling submission for its multiple myeloma CAR-T ciltacabtagene autoleucel (cilta-cel) to the FDA, in hot pursuit of Bristol-Myers Squibb’s delayed rival therapy.

Cilta-cel – which targets B-cell maturation antigen (BCMA) – has been submitted as a treatment for patients with relapsed or refractory myeloma, an incurable form of blood cancer, specifically in heavily treated adults who currently have few therapeutic options.

BMS’ CAR-T idecabtagene vicleucel (ide-cel) is going after the same indication and is due for a verdict from the US regulator on 27 March 2021.

The rolling application allows portions of the dossier to be filed as they become available, shortening the review time, although BMS is still in pole position to bring a multiple myeloma cell therapy to the US market, despite a setback earlier this year when the FDA rejected the filing for the CAR-T, asking for more data.

Cilta-cel’s filing triggers a $75 million payment from J&J to its development partner Legend Biotech under the terms of a deal signed in December 2017. It is J&J’s first application for a cell therapy.

The submission is based on results from phase 1b/2 CARTITUDE-1 study, which showed an overall response rate of 97% with cilta-cel, with 67% of patients achieving a stringent complete response despite having received a median of five prior therapies.

The median duration of response and progression-free survival still have not been reached in the study, suggesting the effect is long-lasting, according to Janssen.

Like other CAR-Ts the therapy isn’t without risk however, and there were 14 deaths reported during the study, including six due to complications related to treatment itself.

With all the caveats about comparing different trials, cilta-cel’s results look a little better than those of ide-cel’s KarMMa study, which had an overall response rate of 73%, including 33% complete responses, among patients who had a median of six prior treatments.

BMS is also developing another BCMA directed CAR-T – orvacabtagene autoleucel (orva-cel) – which generated positive results in the EVOLVE trial this year.

Ide-cel and cilta-cel aren’t the first BCMA-targeted drugs for myeloma, however. GlaxoSmithKline’s antibody-drug conjugate (ADC) Blenrep (belantamab mafodotin) has already been approved in the US and Europe, making almost $11 million in third-quarter sales in its first few weeks on the market.

There are other BCMA drugs coming through the pipeline as well, including a string of bispecific antibody therapies from Regeneron, Amgen, J&J and others that could sidestep the cumbersome and risky treatment and manufacturing process for CAR-T therapies.

The post Chasing BMS, J&J files BCMA CAR-T for multiple myeloma to FDA appeared first on .

Servier to Acquire Agios’ Oncology Business for ~$2B

Shots:

  • Agios to receive ~$1.8B upfront in cash & ~$200M as regulatory milestones for Vorasidenib along with 5% royalties on sales of Tibsovo in the US from transaction close through the loss of exclusivity and 15% royalties on sales of vorasidenib in the US from first commercial sale through the loss of exclusivity
  • The acquisition is expected to close in Q2’21. The acquisition allows Servier to strengthen its product portfolio and drug development pipeline in oncology
  • The transaction includes the transfer of Agios’ oncology portfolio and associated employees, including its Tibsovo and pipeline and clinical programs, including vorasidenib

Click here ­to­ read full press release/ article | Ref: PRNewswire  | Image: Enzo Life Sciences

The post Servier to Acquire Agios’ Oncology Business for ~B first appeared on PharmaShots.

The top 5 pharma M&A deals of 2020

2020’s M&A activity hasn’t quite reached the heights of last year’s, where two pharma mega-mergers – BMS’ buyout of Celegne and AbbVie’s acquisition of Allergan – accounted for almost 40% of total M&A deal values.

That said, there were still some interesting moves indicating new directions of travel for big pharma players – with most deals focused on specific drugs from biotechs, particularly in cancer (though we did get rumours of an AstraZeneca-Gilead merger, which would have been the biggest pharma M&A deal of all time).

Here we take a look at the biggest mergers and acquisitions of 2020 and what they might mean for the companies involved.

AstraZeneca & Alexion

By far the biggest pharma deal of the year is AstraZeneca’s late-breaking offer to buy Alexion for $39 billion.

Alexion has routinely featured among lists of top biopharma takeover prospects in the last couple of years, and with the purchase, AZ will bolster its immunology franchise with $4 billion blockbuster Soliris (eculizumab) and longer acting follow-up Ultomiris (ravulizumab), plus a pipeline of 11 drugs for rare and autoimmune diseases.

It marks something of a departure from AZ’s relentless focus on deal-making in oncology, its top product category, and also comes as Alexion has been locked in a battle with activist shareholders pushing for a sale.

Boston, US-based Alexion spent a lot of 2019 arguing the merits of remaining independent, saying that while Soliris is approaching the end of its patent life – with heavyweight competitors like Amgen already eyeing the biosimilar market for the drug – Ultomiris and its pipeline could help drives sales to $9 to $10 billion in 2025.

The threat of biosimilar competition to its cash cow has weakened Alexion’s share price, providing an opportunity for AZ, which has been rumoured to be angling for a large acquisition for several months.

While the first biosimilars to Soliris have already reached the market in some countries like Russia, Alexion cut a settlement deal with Amgen in the summer that prevents the latter’s biosimilar version of Soliris from entering the US market until 2025, avoiding a near-term cash cliff.

In the meantime, Ultomiris has been gathering momentum, fuelled by intravenous dosing every eight weeks, rather than every two weeks with Soliris. It racked up $340 million in sales last year, and added another $763 million in the first nine months of this year, backing up its blockbuster credentials.

Meanwhile, AZ will also pick up three other drugs – Strensiq (asfotase alfa) for hypophosphatasia, Kanuma (sebelipase alfa) for lysosomal acid lipase deficiency (LAL-D) and anticoagulant reversal agent Andexxa (andexanet alfa) – that collectively brought in almost $675 million in the first nine months of 2020.

Gilead & Immunomedics

The AZ-Alexion deal is likely to be the only big pharma merger this year, but Gilead’s purchase of US biotech Immunomedics and its potential cancer blockbuster Trodelvy isn’t far off it in terms of value, with the deal totalling $21 billion.

California-based Gilead announced its strong intentions in oncology in 2017 with its $11.9 billion buy of Kite Pharma and followed that earlier this year by acquiring immuno-oncology firm Forty Seven for $4.9 billion (see below). Shoring up its assets in a wider range of disease areas will help the company weather the storm as the pool of patients eligible to receive its hepatitis C drugs such as Sovaldi shrinks.

Trodelvy (sacituzumab govitecan) is a first-in-class TROP2 antibody-drug conjugate drug that was granted accelerated approval by the FDA in April for adults with metastatic triple-negative breast cancer (TNBC), who have received at least two previous therapies for metastatic disease.

Data from trials of the drug wowed ESMO in September – Trodelvy was shown to significantly extend overall survival (OS) and improved overall response rate (ORR) and clinical benefit rate (CBR), compared with standard chemotherapy in TNBC patients with brain metastases treated with at least two therapies.

The 500-plus patients in ASCENT had received a median of four previous anticancer treatments, but Trodelvy significantly improved OS with a median of 12.1 months, compared with 6.7 months in patients treated with chemotherapy.

Johnson and Johnson & Momenta

This $6.5 billion deal means that J&J has added potential inflammatory disease blockbuster nipocalimab to the pipeline at its Janssen pharmaceuticals unit.

J&J thinks that Momenta’s lead drug nipocalimab could be a kind of Swiss army knife drug that could be used across a range of inflammatory diseases including maternal-foetal disorders, neuro-inflammatory disorders, rheumatology, and autoimmune haematology.

The success of AbbVie’s Humira (adalimumab), which peaked at almost $20 billion in sales in 2018, demonstrates the potential of inflammatory diseases drugs to make mega-bucks.

Johnson & Johnson’s own Remicade (infliximab) was also a blockbuster several times over thanks to approvals in a range of inflammatory diseases including Crohn’s, rheumatoid arthritis and psoriasis.

But like the rest of this first generation of antibody-based drugs, Remicade has been hit by cheaper competition from biosimilars and the hunt is on for newer drugs that outperform standard therapy in terms of safety and efficacy.

Whether nipocalimab achieves the astronomical figures seen from Humira and Remicade remains to be seen – but the price J&J has paid shows the big pharma thinks it has considerable potential.

Momenta is best known for producing a generic version of Teva’s multiple sclerosis drug Copaxone (glatiramer), but nipocalimab is the company’s lead pipeline asset and the main rationale behind the acquisition.

“The first wave had disproportionate health, economic and social impacts on people in lower socioeconomic groups and those with black, Asian and minority ethnic backgrounds”

Gilead & Forty Seven

Further cementing Gilead’s ambitions in cancer, this $4.9 billion deal adds an antibody targeting several blood cancers to the company’s research pipeline.

Forty Seven is based in Menlo Park, a short drive away from Gilead’s base in Foster City, and is developing magrolimab, which is targeting myelodysplastic syndrome (MDS), acute myeloid leukaemia (AML), and diffuse large B-cell lymphoma (DLBCL).

A potential first-in-class therapy, magrolimab targets CD47, which produces a “do not eat me” signal that allows cancer cells to avoid destruction (an area AbbVie has almost invested significantly in).

By targeting CD47 it’s hoped that magrolimab will allow the patient’s own innate system to engulf and eradicate cancer cells.

The company presented promising results from a phase 1b study of magrolimab in patients with MDS and AML at the American Society of Hematology meeting in December.

Sanofi & Principia Biopharma

Sanofi added a potential multiple sclerosis drug to its pipeline when it bought Principia Biopharma for up to $3.68 billion in August.

The French pharma paid $100 per share in cash for San Francisco-based Principia, which specialises in Bruton’s kinase (BTK) inhibitor drugs, after the deal was unanimously agreed by both boards of directors.

Sanofi’s acquisition builds on a partnership to develop central nervous system drugs that began in late 2017.

In a statement Sanofi said that the acquisition will give it full control of the brain-penetrant BTK inhibitor SAR442168, making marketing more efficient and eliminating any royalty payments due under the 2017 agreement.

The drug known for short as ‘168 reduced multiple sclerosis brain lesions by 85% compared with placebo in a phase 2b trial.

Phase 3 development has begun and will comprise four pivotal trials across the MS disease spectrum.

Another of Principia’s BTK inhibitors, rilzabrutinib, is being tested in phase 3 for patients with moderate to severe pemphigus, a rare and debilitating autoimmune disease that causes blistering of the skin and mucous membranes.

Principia also has a topical BTK inhibitor, PRN473, which is in phase 1 development for immune diseases that could benefit from local application to the skin.

The deal follows Sanofi’s announcement late last year that it is rethinking its R&D operations, turning its back on diabetes and focusing on badly needed “transformative” therapies and maximising the potential of its asthma and eczema drug Dupixent.

CEO Paul Hudson, who was appointed to the role in June last year, has already acquired the oncology firm Synthorx and signed a potential $2 billion collaboration with Kymera Therapeutics to develop immune-inflammatory drugs.

The post The top 5 pharma M&A deals of 2020 appeared first on .

AZ’s Tagrisso gets new early lung cancer use in US

AstraZeneca’s Tagrisso (osimertinib) has been approved by the FDA in a new lung cancer indication that extends its use to a group of patients with early-stage disease.

The new use covers adjuvant treatment of adult patients with early-stage epidermal growth factor receptor-mutated (EGFRm) non-small cell lung cancer (NSCLC) after a potentially curative operation.

Patients must be tested to for the presence of the mutation to check they are eligible to receive the oral drug.

Up to 30% of all patients with NSCLC may be diagnosed early enough to have potentially curative surgery.

But disease recurrence is still common in early-stage disease and nearly half of patients diagnosed in Stage IB, and over three quarters of patients diagnosed in Stage IIIA, experience recurrence within five years.

Based on the findings of the phase 3 ADAURA study this could change, as Tagrisso demonstrated a statistically significant and clinically meaningful improvement in disease-free survival in a primary analysis of patients with Stage II and Stage IIIa NSCLC with EGFR mutations.

This was also seen in the overall trial population of patients with Stage 1B-IIIA disease, the secondary endpoint of the trial.

Findings showed that Tagrisso cut the risk of disease recurrence by 83% compared with placebo in the trial that was stopped early because of the high efficacy shown in the treatment arm.

The ADAURA results were showcased as the last set presentation at this year’s virtual American Society of Clinical Oncology conference, rather like the headline performer at a rock festival.

Results dazzled the oncologists who described the findings as a “home run” and the new indication will also have beneficial side-effect on AZ’s finances, adding substantially to the blockbuster revenues already generated by Tagrisso.

The results also suggest further uses for the drug in early disease and more revenues to come from one of the company’s biggest success stories from the last decade.

Tagrisso was first approved in 2015 to counter a single amino acid mutation known as T790M that nearly always occurs after about 10 months of treatment with tyrosine kinase inhibitor drugs, making tumours resistant.

But after approval in later stage disease, AZ found that the drug outperforms rival tyrosine kinase inhibitors as a first-line treatment in the FLAURA trial, leading to a second FDA indication two years ago.

Tagrisso is already a blockbuster bringing in revenues of more than $3.1 billion in the first nine months of this year, and the new indication will add further momentum to AZ’s biggest selling drug.

 

The post AZ’s Tagrisso gets new early lung cancer use in US appeared first on .

Merck cuts $1bn deal with Janux for cancer T-cell therapies

In a deal that could top $1 billion, Merck & Co has teamed up with US biotech Janux Therapeutics to bring T-cell immunotherapies to cancer patients.

The agreement sees San Diego-based Janux in line for around $500 million in milestones apiece for two T-cell engager therapies – which bind to a tumour cell and recruit a patient’s T cells to eradicate tumour cells – that will be developed using Janux’ TRACTr platform.

Each programme includes undisclosed upfront and milestone payments plus royalties on sales, with Merck indicating in a statement that it will be funding the research.

It’s the first major partnership for Janux, which was set up in 2017, and could be the last to be organised by Merck’s R&D head Roger Perlmutter who is retiring from the big pharma company at the end of the year.

Perlmutter has directed Merck to bolster its pipeline with a series of deals in the last few months of his tenure, as investors have started to ask whether the company has become a bit too reliant on checkpoint inhibitor Keytruda (pembrolizumab), which accounts for around a third of its sales.

Just a few weeks ago, Merck (known as MSD outside North America) paid $425 million to get hold of OncoImmune. The headline of the deal was an experimental COVID-19 drug, but it also claimed rights to a pipeline of early-stage oncology drugs.

Other recent deals include a $2.8 billion acquisition of VelosBio and its anti-ROR1 antibody-drug conjugate (ADC) VLS-101, as well as a partnership with Dragonfly Therapeutics for a cancer programme based on the biotech’s TriNKET natural killer (NK) cell engager platform.

Earlier in the year there was also $2.55 billion alliance with Taiho and Astex for cancer antibodies, including a KRAS drug, and a $4.2 billion deal with Seattle Genetics covering ADCs for  breast cancer and other solid tumours.

The deal-making activity signals Merck’s determination to stay at the forefront of cancer immunotherapy with new medicines that could complement Keytruda and broaden its portfolio.

Janux says its platform could allow it to succeed where other T-cell engager developers have failed due to “dose-limiting toxicities, poor pharmacokinetic profiles, and attenuated efficacy,”

In preclinical testing, its TRACTr drugs have been shown to offer the same anti-tumour activity as older T-cell engager drugs, but don’t cause the release of cytokines that can lead to a runaway immune response and damage to healthy tissues.

Those problems mean that so far only one T-cell engager has reached the market – Amgen’s Blincyto (blinatumomab) for leukaemia – although many others are coming through development.

The post Merck cuts $1bn deal with Janux for cancer T-cell therapies appeared first on .

Merck Signs a ~$1B Pact with Janux to Develop Cancer Therapies Using T Cell Engager Technology

Shots:

  • Janux to receive up to $500.5M/ target as upfront and milestones along with royalties on sales of product emerges from the collaboration, making a total deal value ~$1B. Merck will fund R&D performed under the agreement
  • Merck to get an exclusive WW license to products & IP developed from the collaboration
  • The focus of the collaboration is to leverage Janux’s TRACTr technology to engineer a novel, T cell engager candidates directed against two cancer targets selected by Merck

Click here ­to­ read full press release/ article | Ref: BusinessWire | Image: BusinessWire

The post Merck Signs a ~B Pact with Janux to Develop Cancer Therapies Using T Cell Engager Technology first appeared on PharmaShots.

MacroGenics’ HER2 breast cancer drug Margenza approved in US

MacroGenics’ HER2-targeted breast cancer drug Margenza has been approved by the FDA, challenging several recently approved drugs with a narrow efficacy edge over Roche’s Herceptin in data gathered so far in advanced disease.

Margenza (margetuximab) won FDA approval in combination with chemotherapy to treat metastatic HER2-positive breast cancer after at least two previous rounds of therapy.

One of the talking points at the American Society of Clinical Oncology (ASCO) in 2019, Margenza is a tweaked version of Roche’s Herceptin (trastuzumab), which dominated the HER2-mutated breast cancer market for years until the recent launch of cut-price biosimilar competitors.

MacroGenics has altered the “Fc” part of the antibody – the tail of the ‘Y’-shaped molecule – so that it interacts more efficiently with the immune system when engaged with a cancer cell.

This has produced a small but significant benefit in progression-free survival, with the phase 3 SOPHIA study showing a 24% reduction in the risk of disease progression or death with Margenza plus chemotherapy, compared with trastuzumab plus chemo.

The median progression-free survival (PFS) of patients treated with Margenza and chemotherapy was 5.8 months compared to 4.9 months in patients treated with trastuzumab and chemotherapy.

The difference was more marked in patients carrying a genetic variation called CD16A 158F, where PFS was prolonged by 1.8 months in the margetuximab arm compared to the trastuzumab arm (6.9 months versus 5.1 months).

Response rate was also improved with the Margenza regimen at 22%, compared with 16% in those treated with the Herceptin regimen.

A final overall survival analysis is expected in the second half of 2021, after a planned launch in March next year.

No price has been officially decided but the company said it plans to price it at the low end of the price range seen in other HER2 metastatic breast cancer therapies.

Those competing therapies include Seagen’s Tukysa (tucatinib), which was approved in May for advanced HER2-positive disease in combination with trastuzumab and capecitabine after treatment with at least one HER2-targeted drug.

AstraZeneca and Daiichi Sankyo’s Enhertu (trastuzumab deruxtecan) was approved a year ago for HER2-positive breast cancer after two or more previous HER2 regimens and Puma’s Nerlynx (neratinib) is another FDA-backed option in this indication.

The post MacroGenics’ HER2 breast cancer drug Margenza approved in US appeared first on .

Amgen files KRAS trailblazer sotorasib with FDA for lung cancer

Amgen has filed its groundbreaking KRAS inhibiting drug sotorasib with the FDA for a group of lung cancer patients with an aggressive form of the disease.

The drug was the first targeted at the mutation known as KRAS to show activity in the clinic and provided the biggest talking point at the American Society of Clinical Oncology (ASCO) conference in 2019.

Since then Amgen has been gathering evidence to support a filing in a group of patients with advanced or metastatic KRAS G12C mutated non-small cell lung cancer.

The FDA is reviewing sotorasib under its Real-Time Oncology Review (RTOR) programme and could be the first to be approved in this indication, which covers around 13% of NSCLC patients.

Amgen’s filing is on track with a schedule laid out at the beginning of the year, following a top-line read out from a phase 2 trial in October.

These results came from the CodeBreaK 100 clinical study, which tested the drug in patients whose cancer had progressed despite prior treatment with chemotherapy and/or immunotherapy.

In the study, treatment with sotorasib provided durable anticancer activity with a positive benefit-risk profile, Amgen said, although detailed results have yet to be announced.

Full results will be presented at the International Association for the Study of Lung Cancer (IASLC) 2020 World Conference on Lung Cancer (WCLC) Presidential Symposium next month.

KRAS is a target that has long evaded pharma companies but early trial results in solid tumours at ASCO led to a round of deal-making involving rivals.

Mirati, a biotech from California, specialises in drugs targeting KRAS and is a step behind Amgen with its rival adagrasib.

Novartis signed a deal to evaluate Mirati’s drug soon after ASCO and Merck & Co and Boehringer Ingelheim are among those who have signed KRAS deals.

Although it looks like the molecules developed so far will work only in lung cancer, rather than the wider range of cancers with KRAS mutations, there is hope the drug will provide a new treatment option for an aggressive and deadly form of the disease.

David Reese, executive vice president of Research and Development at Amgen, said: “Sotorasib was the first KRASG12C inhibitor to enter the clinic and now is on track to potentially be the first approved targeted therapy for patients with advanced NSCLC harbouring the KRAS G12C mutation.”

The post Amgen files KRAS trailblazer sotorasib with FDA for lung cancer appeared first on .

M&A: Cerner to Acquire Health Division of Kantar for $375M in Cash

M&A: Cerner to Acquire Health Division of Kantar for $375M in Cash

What You Should Know:

 – Cerner announces it will acquire Kantar Health, a leading data, analytics, and real-world evidence and commercial research consultancy serving the life science and health care industry.

– This acquisition is expected to allow Cerner’s Learning
Health Network client consortium and health systems with more opportunities to
directly engage with life sciences for funded research studies. The acquisition
is expected to close during the first half of 2021.


Cerner today announced an agreement to acquire Kantar Health, a division of Kantar Group, a leading data, analytics, and real-world evidence and commercial research consultancy serving the life science industry for $375 million in cash, subject to adjustment.

Acquisition Will Create Leading Data Insights and Clinical
Research Platform

With this acquisition, Cerner plans to harness data to improve the safety, efficiency, and efficacy of clinical research across life sciences, pharmaceuticals, and health care at large. This acquisition is expected to allow Cerner’s Learning Health Network client consortium to more directly engage with life sciences for funded research studies.

The Cerner Learning Health Network offers health systems complimentary access to a network of bi-directional, de-identified data resources. This access helps advance research efforts and provides opportunities to generate revenue with funded research studies from life science companies. Kantar Health’s proprietary syndicated data products including CancerMPact, Claritis, National Health & Wellness Survey, and its broader oncology, rare disease, and multi-therapeutic expertise are used today by all of the top 20 life science companies to further their real-world evidence, commercial and clinical research efforts.

The combination of Cerner and Kantar Health is expected to
enable a two-sided collaboration between providers and the pharmaceutical
industry, where researchers can generate insights and use differentiated
real-world data assets and expertise to address the most complex clinical
research questions.

“Cerner launched the Learning Health Network with our provider clients to advance a shared vision: treat global diseases more effectively through an acceleration of clinical research,” said Donald Trigg, president, Cerner. “Kantar Health has incredible health economics and medical affairs expertise, differentiated real-world data assets and strong relationships with the world’s leading life science companies. It offers us an amazing opportunity to drive cross-industry collaboration that can change health outcomes around the world.”

Acquisition Reflects Cerner’s Strategic Focus on Clinical
Research

This is the second announcement in this month expanding upon
Cerner’s commitment to improving the safety and efficiency of clinical research
in life sciences and health care. Last week we announced a relationship with
Elligo which broadens the clinical trial resources available to rural and
community hospitals and physician practices. This is significant to help
broaden the diversity of individuals involved in clinical research, including
those in minority populations and rural communities.

The acquisition is anticipated to close in the first half of 2021, subject to regulatory approval, employee consultations, and other conditions, and is not expected to have a material impact on Cerner’s earnings in 2021.

TG Therapeutics set for upsized IPO to develop cancer combination drug

TG Therapeutics provided one of the highlights of the American Society of Hematology (ASH) conference earlier this month after lifting the lid on phase 3 data from its potential challenger to Roche in chronic lymphocytic leukaemia (CLL).

The New York-based biotech requested a rolling approval from the FDA for its combination of ublituximab and umralisib ahead of the meeting and now it aims to raise $275 million with an IPO to fund further development.

This was up from the previously announced IPO size of $200,000 and the company has granted underwriters a 30-day option to buy an additional 948,000 shares of common stock.

The 6,320,000 shares of common stock will have a public offering price of $43.50 each on the IPO, which is expected to close on Thursday.

TG said it plans to use the proceedings from the offering to fund the continued development of ublituximab and umbralisib, as well as licensing in, buying, developing and marketing other drugs.

The combination therapy is based around the combination of the anti-CD20 antibody ulituximab and the oral inhibitor of PI3K-delta and CK-1 epsilon, umralisib.

TG expects the rolling data submission to be complete in the first half of next year.

The rolling filing is designed to speed up the review process for results from the UNITY-CLL trial, a phase 3 study comparing the combination therapy also known as U2, with an active control arm of Roche’s Gazyvaro (ofatumumab) plus chlorambucil.

Results announced at ASH showed U2 produced progression-free survival (PFS) of 38.5 months compared with the Gazyvaro combination, which produced a PFS figure of 26.1 months after a median follow-up period of 36 months.

Overall response rate was also higher with U2 on the 421-patient trial at around 83%, compared with just under 69% on the Gazyvaro combination.

TG has followed up the oncology data with two large studies of ublituximab in multiple sclerosis, which showed the drug outperformed Sanofi’s Aubagio in relapsing disease.

FDA decisions are also expected for ublituximab monotherapy in marginal zone lymphoma and follicular lymphoma next year.

The post TG Therapeutics set for upsized IPO to develop cancer combination drug appeared first on .

Daiichi Sankyo, AZ close on EU approval of Enhertu for breast cancer

Daiichi Sankyo and AstraZeneca could be just weeks away from an EU approval for their antibody-drug conjugate (ADC) for breast cancer – Enhertu – which is tipped to become a multibillion-dollar blockbuster.

At its meeting last week, the EMA’s Committee for Medicinal Products for Human Use (CHMP) recommended approval of Enhertu (trastuzumab deruxtecan) for patients with unresectable or metastatic HER2-positive breast cancer, who have been previously treated with other anti-HER2 drugs.

Enhertu has already been approved for third-line use in HER2-positive breast cancer in the US at the end of 2019 and in Japan earlier this year, based on the results of the phase 2 DESTINY-Breast01 trial in 184 patients, which revealed that the drug shrank tumours in 61% of recipients.

Revenues from the drug  in the first nine months of 2020 came in at $136 million – including $60 million in the third quarter.

Sales were recorded by Daiichi Sankyo, with AZ pocketing $63 million in profit sharing, and according to AZ the drug is now the most prescribed medicine in the third-line and fourth-line settings of HER2-positive metastatic breast cancer.

Enhertu consists of the antibody used in Roche’s blockbuster HER2 antibody Herceptin (trastuzumab), linked to a topoisomerase inhibitor that is toxic to cancer cells. Around one in five patients with breast cancer are considered HER2 positive, which is associated with aggressive disease, a high recurrence rate, and an increased risk of dying.

It works by latching on to HER2-positive cancer cells and delivering a payload to kill them, while ignoring healthy cells, in patients who have failed to respond to Roche’s HER2-targeting cancer drugs Herceptin, Perjeta (pertuzumab), and ADC Kadcyla (trastuzumab emtansine).

Kadcyla was once tipped to become the go-to treatment HER2-posiitve breast cancer when first line drugs like Herceptin/Perjeta had failed, but failed to meet the mark in pivotal trials, truncating its sales growth although it still managed to break into the $1 billion-plus bracket.

Daiichi Sankyo is confident Enhertu can top Roche’s ADC, and also expand the use of HER2 drugs into new cancers like HER2-positive gastric cancer – an indication that is under review by the FDA with a verdict due early next year – and HER2-positive non-small cell lung cancer (NSCLC).

The intention is to gradually position the drug for earlier-line use in breast, gastric and lung cancer, and eventually to try to expand its use into certain low HER2-expressing tumours.

If all the pieces fall into place it reckons peak sales could reach $4.5 billion, and there are plenty of analysts predicting that the drug could quickly breach the $2 billion-a-year threshold.

AZ’s confidence in the potential of Enhertu is evidence from the terms of its late 2019 licensing deal with Daiichi Sankyo, which included a hefty $1.35 billion upfronting a deal that could be worth up to $6.9 billion if all the ADC’s development and sales objectives are achieved.

The post Daiichi Sankyo, AZ close on EU approval of Enhertu for breast cancer appeared first on .

Boehringer buys NBE for €1.18bn, adding cancer ADC expertise

Boehringer Ingelheim has expanded its oncology focus for the second time this week by buying Swiss biotech NBE-Therapeutics, adding an antibody-drug conjugate (ADC) platform led by a drug for a hard-to-treat form of breast cancer.

The German pharma group is paying €1.18 billion ($1.43bn) for all the shares in privately-held NBE, based in Basel, and says the biotech will remain at its current location and operate as a new site within its R&D network.

Terms of the deal haven’t been disclosed, but some of the headline value is tied to clinical and regulatory milestones.

NBE’s lead therapeutic NBE-002is in phase 1 testing for triple-negative breast cancer (TNBC), so called because it lacks the biomarkers that other targeted therapies can latch on to. TNBC is more aggressive than other types of breast cancer, accounting for 15-20% of cases but causing 25% of deaths.

All the Swiss company’s other development programmes are currently at the preclinical stage.

Boehringer started its push into oncology in the mid-2000s, and initially focused on small-molecule, targeted therapies for lung and gastrointestinal cancer, leading to commercial products like Giotrif (afatinib) and Vargatef (nintedanib).

In the last few years it has been progressively adding to its capabilities, for example buying oncolytic virus specialist ViraTherapeutics in 2018 and cancer vaccine player AMAL Therapeutics last year.

It also added antibody firm Northern Biologics earlier in 2020, and – just this week – immuno-oncology company Labor Dr. Merk & Kollegen, which specialises in cancer vaccines and oncolytic viruses.

NBE represents its first foray into the ADC category, which consists of antibodies targeting tumour-associated molecules that are linked to a cell-killing payload.

While they have been around for a couple of decades, the number of ADCs on the market remains low, but latterly the class has been gathering momentum in biopharma drug development as early issues with stability and toxicity have been ironed out.

Just this year, the FDA approved the first ADC for TNBC – Immunomedics’ Trodelvy (sacituzumab govitecan) – although that targets a different molecular target than NBE’s candidate. Trodelvy is directed against Trop2, while NBE-002 targets ROR1, which along with TNBC is also seen in lung adenocarcinomas.

Boehringer’s head of innovation Michael Pairet said that NBE provides the drugmaker with “exceptional” tumour-targeting capabilities that sits well alongside its own immune cell-targeting platforms.

The post Boehringer buys NBE for €1.18bn, adding cancer ADC expertise appeared first on .

UK hospital deploys Microsoft AI to tackle cancer backlog

Addenbrooke’s Hospital in Cambridge will be the first in the world to use an artificial intelligence tool developed by Microsoft that promises to cut the time it takes to analyse computed tomography (CT) scans, and allow treatment to start sooner.

The Project InnerEye tool was developed just down the road from Addenbrooke’s at Microsoft’s Cambridge research labs, and uses AI to highlight tumours and healthy tissue on patient scans, guiding an individual treatment plan.

The AI has been shown to speed up clinicians’ ability to perform radiotherapy planning for head and neck as well as prostate cancers 13 times quicker than manual methods, without compromising accuracy, according to a JAMA Network Open research paper.

Microsoft is making the tool freely available as opensource software to speed up its use by hospitals, though of course clinical use of machine learning models is subject to regulatory approval.

Up to half of the population in the UK will be diagnosed with cancer at some point in their lives, and of these, half will be treated with radiotherapy, with delivery guided by a CT scan to reveal where the radiation beams should be directed to minimise damage to other tissues.

Stacks of 2D images generated during a CT scan have to be reviewed by a radiation oncologist, a time-consuming process, but using Project InnerEye the time to complete that process can be cut by 90%, according to studies.

The AI’s conclusions will be checked and confirmed by a clinical oncologist before the patient receives treatment.

With charity Cancer Research UK estimating that as many as three million people in the UK have missed out on cancer screening tests during the pandemic, the AI could help reduce a “mounting cancer treatment backlog” according to Microsoft.

Lightening the workload of oncologists could also help prevent clinician burnout, which Microsoft says is happening across the NHS as a result of COVID-19. The hope is that quicker treatment could also help improve survival rates for some cancers, although there’s no hard evidence for that yet.

Yvonne Rimmer, consultant clinical oncologist at Addenbrooke’s, said: “There is no doubt that InnerEye is saving me time. It’s very good at understanding where the prostate gland is and healthy organs surrounding it, such as the bladder. It’s speeding up the process so I can concentrate on looking at a patient’s diagnostic images and tailoring treatment to them.

“But it’s important for patients to know that the AI is helping me in my professional role; it’s not replacing me in the process. I double check everything the AI does and can change it if I need to. The key thing is that most of the time, I don’t need to change anything.”

The post UK hospital deploys Microsoft AI to tackle cancer backlog appeared first on .

What HCPs think about COVID-19’s impact on cancer

COVID-19’s knock-on effect on cancer patients is of concern by HCPs, with delayed or decreased diagnosis seen as a key worry. CREATION.co’s Lara Meyer explores why HCPs need more support.

The COVID-19 pandemic has been mainstream headline news throughout 2020 and continues to have a profound impact on all our lives. A key concern that has emerged from the crisis is how COVID-19 will affect other areas of healthcare, particularly cancer diagnosis and treatment.

To understand what healthcare professionals (HCPs) felt about this topic, CREATION.co investigated public social media, in collaboration with Sermo’s HCP survey platform, Sermo RealTime, which provides real time access to physician insights.

We looked at conversations that HCPs were having online to provide a full picture of their concerns and needs. By designing fast surveys based on insights from the online conversation we were able to get a powerful depth of insight. For the survey we recruited oncologists from the United States, United Kingdom and Spain.

HCPs concern for decreased cancer diagnosis

During the COVID-19 pandemic, delayed or decreased cancer diagnosis was highlighted by HCPs as a key concern. Prominent industry figures, such as oncologist and ex-director of the WHO Cancer Programme Professor Karol Sikora, shared news articles and utilised their networks to raise awareness of decreased cancer diagnosis.

However, only a fraction of HCPs explained why they experienced a decrease. Knowing the “why” can help to address the specific challenges that hospitals are facing to ensure patients are receiving the care they need.

Using Sermo RealTime, we asked physicians to rank why they believe there has been a decrease in diagnosis at their hospital or practice. We discovered that cancellation of appointments by hospitals was perceived to have caused the most impact. The reason ranked as the next factor, was hospital staff being diverted to COVID-19 efforts. Understanding these reasons could help with resource allocation and impact assessments.

On social media, HCPs chose to encourage their peers to continue supporting their oncology patients during the pandemic. Again, key online influencers, such as Dr Tatiana Prowell – a well-known medical oncologist specialising in breast cancer, led a call to prioritise patients and raise awareness of decreased diagnoses. We have seen many HCPs supporting their peers online throughout the pandemic.

Uncertainty around cancer treatment

A significant part of the HCP conversation online discussed treatment of oncology patients. Physicians shared their concerns about delaying or changing treatment approaches and the impact this would have in the long term. When surveying physicians using Sermo RealTime, 79% of HCPs shared that they had delayed their patients’ treatment, while 52% of HCPs opted to change their patients’ treatment approach either by switching the drug their patient is on, or changing the administration timing or dose.

For the pharmaceutical industry, this knowledge could help teams in their communication plans to support HCPs with updated information or guidelines about treatments.

We regularly see HCPs share treatment guidelines on social media to provide support when there is confusion around new or existing treatment approaches, often creating their own guidelines when none exist.

And in our survey of physicians, 58% of respondents shared that regulatory guidelines have been their go-to source for information and advice for treatment during the COVID-19 pandemic, alongside consulting their peers. However, even HCPs’ go-to source did not always provide as much support as they would have liked, with some HCPs sharing that they are still unsure of the correct treatment for patients during the COVID-19 pandemic or for cancer patients that have COVID-19.

After surveying HCPs about how confident they were about the information and advice they have received about continuing treatment for their COVID-19 positive or negative cancer patients, 70% of physicians shared that they were “somewhat confident but consulted with their peers”, showing how important peer support is during this time.

HCPs look to the future

As COVID-19 continues to affect countries around the world, HCPs are concerned about the short-term and long-term implications the pandemic will have on patient diagnosis and care. Despite having to respond reactively day-to-day, and the focus on the here and now, the future is still on HCPs’ minds.

Dr Stephanie Graff, a breast cancer oncologist, shared her concerns about “what this might mean long term—stage at diagnosis for example”, and how to bring patients safely back to care.

When physicians in our survey were asked what some of the key concerns are for them going forward, they shared the backlog of patient cases that will need manual review and further investigations, switching to less effective or immunosuppressive treatments, and patients’ hesitancy or distress preventing them getting treatment. Others also shared the same concern as Dr Graff, that cancer patients present at a later stage because of backlogs and hesitancy to come to hospitals having a much larger impact on the treatment approach for these patients.

Across both open and closed online networks HCPs are concerned about the future of patient care.

The online HCP conversation continues at a steady rate each day with oncologists, nurses and specialists continuing to share their concerns and needs online with peers. Throughout the year, HCPs continue to seek the answers they are looking for and share resources online.

These concerns all present opportunities for pharmaceutical companies, hospitals, advocacy groups and medical organisations to support HCPs in very specific areas. Listening to the voices of HCPs online, especially as they are more active during this time, can help uncover key areas for engagement and support.

About the author

Lara MeyerAt CREATION.co, Lara supports clients in scoping and delivering projects. Her pharmaceutical experience includes laboratory research, as well as in marketing and strategy and she recently completed an MSc in Global Management from the London School of Economics. Working with CREATION.co she leads a team of insight analysts compiling reports.

The post What HCPs think about COVID-19’s impact on cancer appeared first on .

Sharing experiences to level the healthcare playing field

Amanda Barrell speaks to Alfred Samuels on his experience of prostate cancer and diversifying clinical trials to include the Black, Asian and minority ethnic (BAME) community.

Alfred Samuels’ stage 4 metastatic prostate cancer brought his 30-year career, which had seen him travel the world with the likes of Beyoncé, Oasis, and Bob Dylan, to a shuddering halt.

Now, through his books and advocacy work, he is determined to help others prepare themselves for the “horrendous” cancer journey and help level the healthcare playing field for the BAME community.

In 2011, Alfred was a fit and healthy 54-year-old providing security for music’s A-listers on global tours. But then he started experiencing an excruciating pain which radiated from his lower back and into his leg.

“It’s really important for men, their partners and families to understand that there is much more to this disease than you think”

At A&E, he was told it was “probably sciatica”, but was advised to ask his GP to refer him for an MRI scan just in case.

“They messed me around for three months before agreeing to a scan. Then, two weeks after the MRI, the diagnosis came in. It was stage 4 metastatic prostate cancer and it had spread to six locations in my body,” said Alfred.

“I went for some tests and the prognosis came back – I was given six months.”

While he was fortunate enough to secure a place on the STAMPEDE clinical trial, during which he received abiraterone and hormone injections, the side effects were “not pleasant”.

“I was hospitalised on a couple of occasions because the pain was horrendous, and they hadn’t got the pain relief medications sorted out properly. I collapsed due to compression of the lower back. I had brain fog and fatigue. I went from someone who was able to run and sprint quite easily to being like a 90-year-old man. It was not a pleasant journey.

“It was untimely, unwanted, and as far as I’m concerned, undeserved,” he said. “My career was over, you’ve got all these mental issues, and then there’s the financial toxicity of it all.”

At the time of presentation, Alfred’s prostate-specific antigen (PSA) was more than 500 ng/ml, compared to the “normal” level for his age at the time, between 2 ng/ml and 4 ng/ml.

However, within months his PSA had dropped to less than 0.01 ng/ml, so Alfred stuck with the punishing protocol.

“One year became two years, two years became three years, and it’s probably then that I started to believe in myself, believe that I could beat it,” said Alfred.

Publishing the journey

Since then, he has published two books based on his experiences and the diaries he has kept of his journey. Invincibility in the Face of Prostate Cancer: Coming Out the Other Side charts his time in the clinical trial, and Motivated to Inspire focuses on the post-treatment period.

“I think it’s really important for men, their partners and families to understand that there is much more to this disease than you think.  You really have to work as a team to get through it,” said Alfred, adding that his wife, Grace, had been invaluable support throughout his journey.

“Interacting with the medical fraternity needs to be explained to people so they get a better understanding of what they’re going to go through. It’s a journey full of pitfalls and pain, and you’ve got to be able to condition yourself for that.”

Focus on inclusivity

Alfred is an ambassador for Cancer Research UK, which funded the STAMPEDE trial, and works with groups including The Urology Foundation and Orchid. He also sits on a patient consultation panel for pharmaceutical company Parexel.

Much of the work he does centres around raising awareness of the patient experience, and the issues of diversity and inclusion are always top of his mind.

“Black people are not getting the best deal when it comes to healthcare, and where prostate cancer is concerned, black men are disproportionately affected,” said Alfred, explaining that Caucasian males had a one in eight chance of developing the condition, while the figure is one in four among black men.

A big part of the problem, he went on, was a lack of BAME representation in clinical trials, and he urged anyone from underserved communities to take part in research if they could.

“There are medications that are just not being tested on people from the BAME community. We will react differently to Caucasian people, so without more people from BAME backgrounds getting involved, we can never be sure that the information we are getting is correct,” he said.

Asked how the industry could be more inclusive, he said it was about trust and understanding.

“If you don’t take the patient’s voice into consideration, you have no understanding of what’s going on for them out there and you end up in a sticky situation.

“I think pharmaceutical companies are cottoning on to that now, but there’s still a lot of mistrust. It is about building relationships and getting to know the communities you are working with.

“Are you speaking in a language that they understand? Are the concepts broken down so that they are digestible?

“It’s not rocket science,” said Alfred, adding that the industry is now starting to understand the importance of such issues, and put plans in place to address them.


Patient Insights is a monthly series that appears in partnership with Inspire, a company with an online support community of more than 2 million patients and caregivers worldwide.

The post Sharing experiences to level the healthcare playing field appeared first on .

KaliVir, Astellas licensing deal; AbCellera’s IPO; Bayer CAR-T Cell therapy collab with Atara; Aligos, Merck together against NASH

KaliVir, Astellas Pharma forms a licensing deal for VET2-L2 oncolytic virus

KaliVir Immunotherapeutics and Astellas Pharma entered into a worldwide exclusive licensing agreement for the development, research, and commercialization of VET2-L2 to widen the horizon of therapeutic approaches available in the Immuno-Oncology market. 

As its lead program, KaliVir is developing VET2-L2 leveraging its unique technology platform based on a genetically modified vaccinia virus. VET2-L2 is an oncolytic vaccinia virus that can be delivered intravenously to cancer patients. This will reduce the need for complicated procedures of the direct intra-tumoral administration and will be of huge convenience for patients. The therapy is in the preclinical stage.

The deal brings together KaliVir’s expertise in the development of oncolytic viruses with Astellas’ capabilities in advanced drug development. Under the terms of the agreement, Astellas is going to shell out an upfront sum up to USD 56 million in addition to the payments supporting the ongoing research, development, and pre-clinical activities surrounding VET2-L2 as well as the Second Product. KaliVir may also receive up to US$307 million and up to US$271 million for the development, regulatory, and commercialization of VET2-L2 and Second Product, respectively, and royalty payments on net sales of each licensed product.

AbCellera Biologics readies to raise up to USD 391 million in IPO

Canadian antibody-drug discovery platform AbCellera Biologics is setting the groundwork for a massive Initial public offering worth USD 391 million. 

The company made a mark when it struck a deal with the pharmaceutical engine, Eli Lilly, to work on its COVID 19 therapy – human antibody bamlanivimab – which managed to secure EUA with the USFDA. Not to lose sight of AbCellera’s AI-powered antibody discovery platform that speeds up the process of finding antibodies that can be developed into drugs. Further, the company has the support of PayPal founder and tech/life sciences investor Peter Thiel, German entrepreneur Christian Angermayer, the Bill & Melinda Gates Foundation, Viking Global Investors, and healthcare investment firm OrbiMed Advisors LLC in addition to a grant of USD 175.6 million from the Canadian government. 

The company is offering 23 million shares priced between USD 14 and USD 17 each. It is being estimated that this IPO if successful, will be the biggest debut on record for Canadian biotech.

Bayer collaborates with Atara Biotherapeutics for off-the-shelf CAR T-Cell Therapy 

Germany-based life sciences titan, Bayer, has set out on a collaborating spree to explore CAR-T cell gene therapy and oncology. Not long ago, the acquisition of an N.C.-based gene therapy company AskBio by Bayer made headlines. Last year, Bayer had got its hands on another cell and gene therapy company, BlueRock Therapeutics

And now the next step towards bolstering its strategic presence in the cell & gene therapy market, Bayer has collaborated with Bay Area-based Atara Biotherapeutics.

The doubleton through collaboration will focus on developing off-the-shelf T-cell immunotherapy for high mesothelin-expressing tumors. It covers Atara’s two developmental candidates, ATA3271, an armored allogeneic T-cell immunotherapy, and an autologous version, and ATA2271, for high mesothelin-expressing tumors such as malignant pleural mesothelioma and non-small-cell lung cancer.

While ATA3271, the allogeneic version of this CAR-T, leverages Atara’s EBV T-cell platform and is currently in IND-enabling studies, ATA2271, the autologous version has enrolled the first patient in an open-label, single-arm Phase 1 clinical study in November 2020.

Aligos Therapeutics, Merck team up to discover and develop an Oligonucleotide Therapy for NASH

Aligos Therapeutics has entered into an exclusive license and research collaboration agreement with Merck with a motive to get rid of the hassle due to non-alcoholic steatohepatitis (NASH) by developing oligonucleotide therapies. 

Under the terms of the deal, the duo plans to leverage Aligos’ oligonucleotide platform technology to discover, research, optimize and develop oligonucleotides directed against a NASH target as well as another additional target of interest in the cardiometabolic/fibrosis space. In exchange for its platform, Aligos will receive an upfront payment as well as additional milestone payments. 

Aligos will be committed to designing, preparing, and evaluating the oligonucleotide molecules and delivering optimized lead molecules, Merck, on the other hand, will lead in the research, clinical development, and commercialization efforts.

While the NASH market seems lucrative, the past failures and setbacks faced by the therapies and their pharma owners have nothing but a dejection. After Gilead, InterceptGenfit, and Albireo – all reported disappointing results in their respective NASH programs, the market picture was a bit grim. However, it seems the potential and opportunity the NASH market landscape has up its sleeves outweighs the risks. 

The post KaliVir, Astellas licensing deal; AbCellera’s IPO; Bayer CAR-T Cell therapy collab with Atara; Aligos, Merck together against NASH appeared first on DelveInsight Business Research.

FDA puts Bellicum’s cancer cell therapy trial on hold

The FDA has placed a clinical hold on a phase 1/2 trial of Bellicum’s cancer cell therapy BPX-601, following a patient death.

Houston, Texas-based Bellicum said that the regulator had placed recruitment and dosing on hold in the dose-escalation trial in patients with previously treated metastatic pancreatic cancer or prostate cancer.

Bellicum is hoping to develop a next-generation CAR-T that has the firepower to tackle solid tumours, something that approved therapies from companies such as Novartis and Gilead are unable to do.

The action was due to the death of a pancreatic cancer patient in the trial reported to the agency by the company and the clinical investigator. Bellicum classified the patient death as unrelated to BPX-601 and rimiducid.

Shares in the biotech were sharply down after the announcement, which came only a few weeks after the company cut development staff following a readout from the trial suggested BPX-601 was not capable of shrinking tumours.

The company had continued with the trial to further investigate how the drug was working and Bellicum said it would work with the FDA to resume the trial.

Recruitment on to a separate phase 1/2  clinical trial of BPX-603, described as a dual-switch cell therapy in patients with HER2+ solid tumours will still go ahead in the coming weeks.

BPX-601 is the company’s first potential drug candidate, a CAR-T cell therapy that is designed to enable production of cytokines and enable the drug to override cancer mechanisms that inhibit the immune system.

The company has dubbed the technology CoCAR-T, which activates two receptors – MyD88 stimulates functioning within immune cells, while stimulating CD40 promotes anti-tumour action from T-cells.

CD40 can also re-educate macrophages to destroy tumour stroma – dense thickets of cells that act as a physical barrier between the cancer and the rest of the body that cell therapies struggle to penetrate.

 

The post FDA puts Bellicum’s cancer cell therapy trial on hold appeared first on .

ASH: Lilly builds case for its BTK drug LOXO-305 in lymphoma

Eli Lilly’s buyout of Loxo Oncology last year has already yielded one approved drug, and it now has a path to market for a second after BTK inhibitor LOXO-305 after reporting promising data at the American Society of Haematology (ASH) meeting.

Updated results from the phase 1/2 BRUIN trial suggest that LOXO-305 could become a go-to therapy in B-cell non-Hodgkin’s lymphoma (NHL) patients whose cancer progresses despite earlier treatment with older BTK drugs.

Lilly’s drug is a non-covalent inhibitor, while already-approved BTK inhibitors like Johnson & Johnson and AbbVie’s Imbruvica (ibrutinib), AstraZeneca’s Calquence (acalabrutinib), and BeiGene’s Brukinsa (zanubrutinib) are covalent inhibitors.

According to its developers, that means it has efficacy in NHL that has developed resistance to first-line BTK drugs, mainly because it binds to its target reversibly. That means it can return to the circulation and continue to bind to BTK, without stimulating the resistance escape mechanism.

Imbruvica has become a mainstay of first-line treatment for NHL and other blood cancers like chronic lymphocytic leukaemia, driving it to sales of more than $6 billion collectively for J&J and AbbVie last year, but resistance can make its benefits short-lived.

It’s estimated that resistance can cause treatment failure of more than 40% of patients treated with first-line Imbruvica after five years, and more than 50% when the drug is used second-line for relapsed or refractory disease.

In BRUIN, LOXO-305 was tested in various types of NHL, including 61 patients with mantle cell lymphoma (MCL) and 19 with Waldenström’s macroglobulinemia (WM) who had previously been treated with a covalent BTK inhibitor.

In MCL, the drug had an overall response rate (ORR) of 52%, even though patients had received on average three prior lines of therapy, with around half of them seeing a complete response to the drug.

Most patients had been treated with a BTK drug, anti-CD20 antibody therapy with rituximab, and/or chemotherapy, with some also receiving Bristol-Myers Squibb/Celgene’s Revlimid (lenalidomide), a bone marrow transplant or CAR-T cell therapies.

Four out of five (83%) of the MCL patients were still responding to LOXO-305 at the data cutoff, after a median of six months of treatment.

For the WM group there was an ORR of 68%, with 13 of 19 patients responding including nine partial responses and four minor responses.

Ten of the 13 are still on the drug and responding after a median of 4.6 months, and there were also encouraging response data with other forms of NHL including follicular lymphoma (FL), diffuse large B-cell lymphoma (DLBCL), and marginal zone lymphoma (MZL).

Armed with the new data, Lilly has embarked on a bold strategy for LOXO-305 that will try to position it quickly as an alternative to the established drugs, rather than an option after they have failed.

The drugmaker is planning to start a head-to-head phase 3 trial of LOXO-305 versus the investigator’s choice of covalent BTK inhibitor in relapsed-refractory MCL in the first quarter of next year, gambling that its drug will show best-in-class efficacy.

If it eventually does get approved, LOXO-305 would join Lilly’s RET inhibitor Retevmo (selpercatinib, formerly LOXO-292) on the market, becoming the second product from Lilly’s $8 billion acquisition of Loxo in January 2019.

Analysts think LOXO-305 has greater sales potential than Retevmo, given the size of its target market and the fact that the RET inhibitor is in a marketing battle with rival drug Gavreto (pralsetinib) from Roche and Blueprint Medicine.

The post ASH: Lilly builds case for its BTK drug LOXO-305 in lymphoma appeared first on .

Exelixis takes $20m option Iconic/Zymeworks’ cancer ADC

Exelixis has taken a $20 million option to license in an antibody-drug conjugate cancer (ADC) drug from Iconic, with clinical trials expected to start next year.

The California biotech had reached an agreement with Iconic and Zymeworks giving the option on the drug in May last year.

Probably best known for the cancer drug Cabometyx (cabozantinib), which is marketed by Ipsen outside of the US and Japan, Exelixis already has four small-molecule drugs on the market.

But CEO Michael Morrissey has selected ADCs as an area of research focus, and announced two partnerships in this field with Swiss startup NBE Therapeutics and Catalent Pharma in September.

After taking the option Exelixis now has responsibility for clinical development, marketing and manufacturing of the Tissue Factor (TF) targeting drug known as XB002.

The drug consists of an antibody targeting TF, coupled with a linker and cancer-killing payload developed by Zymeworks.

Exelixis plans to file for clinical trial clearance with the FDA shortly and plans to take it into a phase 1 trial involving solid tumours early next year.

Exelixis gained an exclusive option to license XB002 (then ICON-2) in exchange for an upfront payment to Iconic of $7.5 million and a commitment of preclinical development funding.

Iconic is now also eligible for future development, regulatory and commercialisation milestone payments, as well as royalties on potential sales.

Zymeworks, based in Vancouver, will also get a share of the option fee and could also receive a share of all future revenue and any sales royalties under an agreement licensing the drug to Iconic.

Exelixis said it decided to take the option after preclinical studies showed that the drug binds to Tissue Factor without affecting the blood clotting cascade, which has held back development of other molecules aimed at the target.

Although it is an important part of the blood clotting process in healthy cells, tissue factor is also expressed by tumours.

It contributes to several of the processes that help cancers grow, including thrombosis, metastasis and growth of blood vessels that feed oxygen to the tumours.

 

The post Exelixis takes $20m option Iconic/Zymeworks’ cancer ADC appeared first on .

4th Antigen Specific Immune Tolerance Summit

The 4th Antigen Specific Immune Tolerance Digital Summit (ASIT) brings together industry representatives from the fields of autoimmunity, allergy, immuno-oncology and transplantation to tackle the complexities behind autoimmune disorders head on. With momentum building for antigen specific immunotherapies, ASIT 2021 will be the only antigen-specific summit offering thought-leading content to drive the field towards a more precise and ethical antigen-specific approach to drug development.

This is the industry’s definitive antigen specific drug development forum.

Across 3 packed days, thought leaders from NovartisImCyseHarvard Medical SchoolToleranziaToralgen will reveal insight, data, and lessons learned from the last 12 months to enable you to hurdle the roadblocks preventing the development of clinically safe and effective antigen-specific immunotherapies for autoimmune and immune mediated disorders.

Access the official agenda for more information.

The post 4th Antigen Specific Immune Tolerance Summit appeared first on .

J&J files lung cancer bispecific amivantamab for FDA approval

Johnson & Johnson has filed its bispecific antibody amivantamab to the FDA, hoping to muscle into the big market for drugs that are used to treat EGFR-positive non-small cell lung cancer (NSCLC).

J&J’s Janssen unit is seeking approval of the drug in a specific patient group – those with exon 20 insertion mutations whose disease has progressed despite first-line chemotherapy.

Janssen has ambitions to expand the use of the drug into the larger EGFR inhibitor market, which at the moment, is led by AstraZeneca’s fast-growing EGFR tyrosine kinase inhibitor (TKI) Tagrisso (osimertinib).

Amivantamab (formerly JNJ-6372) is a bispecific or double-headed antibody, combining an EGFR-binding domain at one end with one targeting mesenchymal epithelial transition (MET) factor, which is a common resistance mechanism that can reduce the activity of EGFR drugs.

That means it targets both the primary mutation in this form of NSCLC and the resistance mechanism at the same time, according to J&J, which has previously said it plans to expand use of the drug to “all EGFR-containing tumours”, challenging Tagrisso and other TKIs like AZ’s older Iressa (gefitinib) and Roche’s Tarceva (erlotinib).

Amivantamab is also the first drug to be filed specifically for patients with exon 20 mutations, who don’t tend to respond well to current therapies including EGFR inhibitors, and as a result picked up a breakthrough designation from the FDA for this use.

EGFR mutations are some of the most common mutations in NSCLC, and that has driven sales of Tagrisso – the standard of care for previously-untreated EGFR-positive NSCLC – to more than $3 billion last year.

The Exon 20-mutated population is fairly large in its own right, as these mutations are the third most prevalent primary EGFR mutation. That said, they often go undetected, so J&J will have to develop ways to identify suitable patients if amivantamab is to make headway in the market.

In the summer, the drugmaker signed partnered with Guardant Health to seek regulatory approval of a companion diagnostic for amivantamab that will be used to identify suitable patients.

Along with the marketing approval submission, J&J has also set up an expanded access programme, which would allow patients at need to use the drug while the FDA review is underway.

Patients with exon 20 insertion mutations have a median survival of less than 17 months, around half that of patients with other EGFR mutations.

Amivantamab has been filed for approval based on the phase 1 CHRYSALIS study which showed that the drug shrank tumours in 36% of all patients in the study, and 41% of those previously treated with chemo. The median durations of response were 10 months and seven months, respectively.

CHRYSALIS also included an arm that combined amivantamab with lazertinib, an experimental EGFR TKI that J&J licensed from South Korea’s Yuhan in 2018, which showed a 100% ORR with the combination in previously-untreated EGFR-mutant NSCLC.

That result prompted J&J to start a phase 3 trial comparing the duo to Tagrisso directly in treatment-naïve patients. Called MARIPOSA, it will compare Tagrisso to lazertinib alone as well as the combination.

Meanwhile, J&J is also exploring the use of amivantamab in patients who have failed TKI therapy and only have chemo as an option.

Both lazertinib and amivantamab – which was developed using technology from Danish biotech Genmab –  have blockbuster sales potential, according to the company.

The post J&J files lung cancer bispecific amivantamab for FDA approval appeared first on .

Cytokine-Based Cancer Immunotherapies Summit

The renewed interest in the anti-tumor properties of cytokines has led to an increase in the number of clinical trials that explore the safety and efficacy of cytokine-based drugs, not only as single agents, but also in combination with other immunomodulatory drugs.

Therapeutically targeting of soluble immune modulators in the tumor microenvironment (TME) has been shown to create a ‘hot’ tumor, increasing T cell infiltration and improving response rates to immune checkpoint blockade treatment. The immuno-oncology research community is pivoting towards engineering soluble factors and cytokines to transform the TME into an immunostimulatory state, acting as a complementary strategy to a suite of cancer immunotherapies approaches.

The virtual Cytokine-Based Cancer Immunotherapies Summit has been established to give drug developers insight into the latest clinical advancements and stimulate discussions around how to take immuno-oncology combinations forward to meet the vast unmet need of cancer patients.

Join us and leading experts from Roche, CytomX, Philogen, Bristol-Myers Squibb, Novartis and many more to discuss how to reduce toxicity and achieve an efficacious dose, determining the mechanisms of action and hear the latest data coming out of the clinic.

If you are a research team striving to advance your cytokine therapies, this is the best suited online networking forum which has been curated for you.

Access the official agenda for more information.

The post Cytokine-Based Cancer Immunotherapies Summit appeared first on .

PARP & DDR Inhibitors Summit 2021

Developing Inhibitors of Specific DDR Targets, in Monotherapy or in Combination, to Treat Predictable & Identifiable DDR-Defective Cancer Indications

PARP & DDR therapeutics are exploding thanks to numerous novel next generation synthetic lethal targets reaching an inflection point.

The PARP & DDR Inhibitors Summit is your only industry focused meeting dedicated to bringing DDR therapeutics to the clinic. The digital meeting will capitalize on the therapeutic application of PARP inhibitors by finding optimal treatment combinations and expanding its use in cancer indications, as well as exploring emerging targets such as Pol Theta.

Join us and the VPs, Heads and Directors of Translational Oncology, Target Discovery, and Early Drug Development to discuss how to develop novel inhibitors of specific DNA repair enzymes that can be used as either monotherapy or in combination with other agents within predictable, identifiable, DDR-defective tumor populations.

Gain insight into the latest techniques to decipher the entangled network of DNA damage response mechanisms, including multiple DNA repair pathways, damage tolerance processes, and cell-cycle checkpoints to safeguard genomic integrity.

Plan how to optimize PARP inhibitors in the clinic with treatment combinations to expand indications and deliver more selective and better tolerated medicines to improve survival rates in difficult-to-treat cancers.

Furthermore, there will be a unique pre-conference focus day looking at the rationales and approaches for enhancing replication stress in cancerous cells and inhibiting DNA repair mechanisms which prevent apoptosis.

If you are a research team striving to create best-in-class DDR therapeutics, this is the best suited online networking forum which has been curated for you.

Access the official agenda for more information.

The post PARP & DDR Inhibitors Summit 2021 appeared first on .

Merck KGaA Collaborates with Artios Pharma for DNA Damage Cancer Therapies

Shots:

  • Artios to receive $30M up front & near-term milestones, ~$860M/ target as option fee along with royalties on sales of each commercialized product. Additionally, Artios has opt-in rights for joint development & commercialization of the programs
  • Merck has the right to opt into exclusive development & commercialization of compounds on up to 8 targets
  • The collaboration leverages Artios’ nuclease targeting discovery platform to jointly identify multiple synthetic lethal targets for precision oncology drug candidates while Merck KGaA will utilize its expertise and resources in the field of DDR

Click here ­to­ read full press release/ article | Ref: PRNewswire | Image: PSD

The post Merck KGaA Collaborates with Artios Pharma for DNA Damage Cancer Therapies first appeared on PharmaShots.

Merck KGaA signs DNA damage response cancer deal with Artios Pharma

Germany’s Merck KGaA has joined with UK-based Artios Pharma in a potential multi-billion dollar deal to investigate novel DNA damage response targets in cancer.

The principle of DNA damage response is already being exploited by AstraZeneca and other companies with their poly (ADP-ribose) polymerase (PARP) inhibitor drugs.

These target the inherent genetic instability in certain cancer cells, which have switched to a backup mechanism to repair damage to their DNA code.

Turning off this mechanism causes tumour cells to die without affecting healthy tissue –  a concept scientists term “synthetic lethality”.

German Merck said it plans to work with Artios, a DNA damage response (DDR) collaboration involving Cancer Research UK and research partners worldwide, to find anticancer drugs that exploit a similar mechanism.

Artios will receive $30 million in up-front and near-term payments, plus double-digit option fees and up to $860 million in total milestones per target.

It will also receive up to double digit royalty payments on net sales of each product marketed by Merck KGaA.

Darmstadt-based Merck KGaA will contribute its expertise in DDR and will have exclusive worldwide rights to develop and market selected therapeutics discovered under the collaboration.

The partnership will focus on nucleases, which cancer cells depend on for their survival to repair DNA damage.

In certain cancer with mutations in DNA DDR pathways, inhibiting important nucleases can lead to cancer cell death.

The collaboration does not include Artios’ lead drugs, Pol theta and ATR inhibitors, for which Artios will retain rights.

Artios was founded by SV Health Investors in 2016 and has a partnership with Cancer Research UK, plus collaborations with DNA repair researchers worldwide such as the Institute of Cancer Research in London, the Netherlands Cancer Institute and the National Centre for Biomolecular Research at Masaryk University in the Czech Republic.

Backed by blue-chip investors including AbbVie Ventures, Novartis Venture Fund, Pfizer Ventures Artios is based at the Babraham Research Campus in Cambridge, UK, with offices in New York City.

The post Merck KGaA signs DNA damage response cancer deal with Artios Pharma appeared first on .

French startup lands $8.7M to advance microbiome therapies

money

Founded in 2014, MaaT Pharma is pushing two therapies through clinical trials that are designed improve survival for patients with blood cancers and other diseases. Despite high-profile hiccups, the field of microbiome therapy remains attractive to investors.

Accumulated genetic variations: What they are and why they matter to a complete health picture 

Unlike inherited genetic predispositions, accumulated genetic changes are the result of environmental influences, such as smoking, chemicals or ultra-violet radiation. A growing body of research links somatic changes to an increased likelihood of blood cancers and cardiovascular disease, both heart disease and stroke.

TG Therapeutics challenges Roche with FDA filing for CLL drug

US biotech TG Therapeutics has begun a rolling filing with the FDA for its combination therapy for chronic lymphocytic leukaemia, in a challenge to Roche.

The New York-based firm has requested approval for the combination of its anti-CD20 antibody ublituximab and umralisib, an oral drug that inhibits PI3K-delta and CK-1 epsilon.

The FDA had already granted fast-track designation to the combination therapy, allowing extra help during the development process on the basis of earlier clinical data.

Aside from Gazyvaro, CLL can be treated with older chemotherapy agents, or Roche/AbbVie’s Venclexta (venetoclax) in some instances, and the fast track tag showed the FDA considered the combination to be a promising new contender on the market.

TG said it expects the data submission to be complete in the first half of 2021.

The rolling filing is designed to speed up the review process for results from the UNITY-CLL trial, a phase 3 study comparing the combination therapy also known as U2, with an active control arm of Roche’s Gazyvaro (ofatumumab) plus chlorambucil.

The trial randomised patients into four treatment arms: ublituximab single agent, umbralisib single agent, ublituximab plus umbralisib, and an active control arm of obinutuzumab plus chlorambucil.

A prespecified analysis was conducted to assess the contribution of ublituximab and umbralisib in the U2 combination arm and allowed for the termination of the single agent arms.

The trial continued enrolment in a 1:1 ratio into the two combination arms: the investigational arm of U2 and the control arm of obinutuzumab plus chlorambucil.

Around 420 people were recruited to the two combination arms and around 60% were untreated and 40% were relapsed or refractory.

Primary endpoint was superior progression-free survival in the control arm and topline results were announced in May, showing a statistically significant improvement in PFS in the combination therapy arm.

The company had hoped to file data from an interim analysis of the trial in late 2018, but suffered a setback when an independent assessment board said the data was not mature enough for an analysis of the overall response rate.

The FDA is also due to make a decision next year on umbralisib as a single agent for marginal zone lymphoma and follicular lymphoma.

The post TG Therapeutics challenges Roche with FDA filing for CLL drug appeared first on .

Roche, Blueprint raise pressure on Lilly with new Gavreto approval

The FDA has cleared Roche and Blueprint Medicine’s Gavreto for a new use in RET-mutated thyroid cancer, putting it on a level playing field with Eli Lilly’s rival therapy Retevmo.

The US regulator says Gavreto (pralsetinib) can be used to treat patient aged over 12 with advanced or metastatic RET-mutant thyroid tumours, adding to its earlier approval for adults with metastatic RET-positive non-small-cell lung cancer (NSCLC) in September

Retevmo (selpercatinib) was simultaneously approved by the agency for the lung and thyroid cancer in May, so has enjoyed an all-too brief period as the first and only drug for both RET-driven tumour types in the US – particularly as Gavreto’s latest green light has come three months ahead of schedule.

Retevmo is still in the early stages of rollout, adding a little under $12 million to Lilly’s top-line in the third quarter of this year, and will now have to contend with a rival drug backed by Roche’s considerable oncology marketing clout.

Roche clearly has high expectations for Gavreto, agreeing a licensing deal with Blueprint in June that could be worth up to $1.7 billion if it hits development and commercial targets. A hefty $675 million of that was in upfront cash, with another $100 million in equity.

Gavreto’s label says it can be used for patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require systemic therapy, or patients with RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate).

The latest Gavreto approval was based on results from the Phase 1/2 ARROW trial evaluating Gavreto in RET-altered cancers, including thyroid cancer, NSCLC and other solid tumours.

It had a 60% overall response rate in patients previously treated with Exelixis’ Cometriq (cabozantinib) or Sanofi’s Caprelsa (vandetanib), both kinase inhibitors that combine activity against RET with other targets.

The drug was also tested in patients with RET-mutant MTC who did not receive prior Cometriq or Caprelsa, with shrinking tumours in 64% of them, 84% of whom had responses that lasted six months or more. Among patients with RET fusion-positive thyroid cancer who were radioactive iodine-refractory, the ORR was 89%, all lasting at least six months.

The data – which is fairly closely matched with Lilly’s results with Retevmo (with the usual caveats about comparing different trials) – is also being evaluated by the EMA in Europe with a decision expected next year.

Both drugs are in pill form, with Gavreto given once daily while Retevmo is taken in two separate doses, and there are trade-offs when it comes to tolerability, according to some analysts.

Both drugs are linked to side effects including high blood pressure, liver toxicity and haemorrhage, but Retevmo’s label also includes a warning about cardiac effects (QT interval), and Gavreto’s mentions interstitial lung disease/pneumonitis.

Prostate cancer imaging agent also gets the nod

The FDA has also just approved Gallium 68 PSMA-11, the first PET imaging agent for prostate-specific membrane antigen (PSMA) positive lesions in men with prostate cancer.

Developed by the University of California, Los Angeles (UCLA) and the University of California, San Francisco (UCSF) – which hold the marketing license – the imaging agent is approved to identify if prostate cancer has spread to other parts of the body.

If identified, those metastases are potentially curable using surgery or radiotherapy, according to the FDA. At the moment metastases can be spotted with MRI or CT scanning but are not very effective at finding prostate cancer spread.

Two other PET drugs – F 18 fluciclovine and C 11 choline – are approved for prostate cancer imaging. However, they are only approved for use in patients with suspected cancer recurrence.

The post Roche, Blueprint raise pressure on Lilly with new Gavreto approval appeared first on .

A history of Pfizer

Few companies embody the term ‘pharma giant’ as much as Pfizer. Here we take a look at the colourful history of one of the biggest drugmakers in the world.

Pfizer was founded in 1849 by two recent German immigrants to the USA, Charles Pfizer and Charles Erhart. Both in their mid-twenties, the two men set up what was initially a fine chemicals business in a Brooklyn factory, using a loan from Pfizer’s father as capital. The company’s first product, a palatable anti-parasitic drug, made to taste like toffee, united Pfizer’s skills as a chemist with Erhart’s training as a confectioner. It was a success, and set the pattern for the company’s future development.

Pfizer’s Tokyo building

The convulsion of the American Civil War, which broke out soon after in 1862, had as much of an impact on the nascent pharmaceutical industry as on American society in general. The “first industrial war” involved drug producers as much as weapons manufacturers. Like their competitor Squibb, the sudden need for enormous quantities of painkillers and antiseptics for the Union armies provided a great scope to expand production. By 1868, Pfizer’s revenues had doubled since the start of the war, and their product lines had expanded greatly.

After the war, Pfizer continued to focus on industrial chemicals as much as medicines, producing the citric acid needed for the emerging soft drinks industry, fuelling brands like Coca Cola and Dr Pepper’s expansion in the 1880s. This became their mainstay for many years, laying the basis for their continued growth. Also, when supply of tartaric acid was disrupted due to the civil war and increased tariffs, Pfizer developed its production to become the leading supplier of chemicals in the US.

“The ‘first industrial war’ involved drug producers as much as weapons manufacturers”

Erhart died in 1891, and Pfizer in 1906, leaving a company of around 200 employees in the hands of Emil Pfizer, who served as president until the 1940s, the last member of the Pfizer family to be involved in managing the company. Under his stewardship, Pfizer’s expertise in scientific production methods developed greatly. In 1919 their scientists pioneered mould fermentation production of citric acid from molasses, freeing their citric acid business from European citrus fruit supplies, which had been disrupted by the First World War. They developed a deep tank fermentation process, the principles of which would later be applied to the production of penicillin. As a consequence of Pfizer’s innovation, the price of citric acid tumbled over the succeeding decades, with the value of the chemical falling by 5/6ths in 20 years. In 1936 the company discovered a fermentation free method of producing vitamin C, which they rapidly expanded into vitamins B2 and B12 amongst others, rapidly becoming a leading vitamin producer – chemicals that were very novel at the time.

This expertise in fermentation and large-scale pharmaceutical production put Pfizer in good stead when in 1941 the US government appealed to the pharma industry for support in producing penicillin for the war effort. In an unprecedented collaboration, Pfizer worked with government scientists, the researchers such as Frederick Banting who had been working on the drug before the war, and a plethora of other players in the industry to markedly improve the efficiency of drug production, as they proudly state “most of the penicillin that [went] ashore with Allied force on D-Day [was] made by Pfizer”.

Antibiotics marked the transition to the modern Pfizer. Their follow-up to penicillin, Terramycin, first marketed in 1950, was both their first proprietary drug, and the first for which the company used sales reps, their soon to be formidable force of salesmen starting with just eight members.

Pfizer initiated its first major internationalisation at this stage, moving into nine new countries in 1951. It was at this time they set their site at Sandwich in the UK, initially just to finish processing compounds imported from America, but due to tariffs on imported products the company rapidly expanded the plant to accommodate producing medicines from scratch. Pfizer’s international expansion put great trust in their local staff compared to other organisations, recruiting nationals and giving them a great deal of autonomy.

“Lipitor…became the biggest-selling prescription medicine ever, earning Pfizer $12 billion a year in 2007, one quarter of its total sales”

The areas that Pfizer directed its research into expanded in these years as well. In 1952, it established its Agricultural Division, beginning its foray into animal health, and in 1953 acquired Roerig, a nutritional supplement specialist, which became incorporated as a division in its own right. By the 1960s, Pfizer were at their “most diversified point in [its] history” – in its own words, its interests “stretched from pills to perfume, and petrochemicals to pet products”.

Throughout the 60s and 70s the company continued to bring out new drugs, such as the broad spectrum antibiotic Vibramycin, and broadening its research base, reorganising its R&D operations in 1971 into a Central Research Division, and increasing spend on this area of the company from 5% to 15% of revenue. This attention to innovation began to pay off in the 1980s, with a series of blockbusters, the first of which, the COX inhibitor Feldene, arrived in 1980 rapidly becoming one of the biggest-selling anti-inflammatories in the world. Others rapidly followed, including Glucotrol, aimed at diabetics, and Procardia, an anti-hypertensive. The 1990s and 2000s would soon take this blockbuster-based success to new levels.

The statin Lipitor, approved in 1997 for Warner-Lambert before their merger with Pfizer, became the biggest-selling prescription medicine ever, earning Pfizer $12 billion a year in 2007, one quarter of its total sales. It almost hadn’t made it through clinical development, facing problems with ineffective chiral isomers and limited efficacy in animal testing, but showed such impact in human trials that it blew the competition away.

But Pfizer’s almost Hollywood-level blockbuster of the 1990s was the little blue pill of Viagrar. Formulated initially at the Sandwich site in the UK as an anti-hypertensive, it was found have “unexpected” side effects that made the company rapidly change the indication to erectile dysfunction. But despite the cultural ubiquity, Viagra has recently faced the inevitable threat from competition and generics, dropping from 92% of the ED market in 2000 to around 50% in 2007, with vigorous competition from drugs such as Cialis and Levitra.

“Pfizer is the 6th largest lobbier in Washington, and spent $25 million on lobbying during the passing of Obama’s healthcare reform legislation alone”

Ups and downs

Like most pharma companies of its size, Pfizer has faced its fair share of controversy as one of the most well-known drugmakers in the world.

In 2009, Pfizer faced more than $2 billion in legal settlement payments over marketing practices for drugs, and around the same time announced it would close a large number of manufacturing and R&D sites worldwide, including its Sandwich facility, which at the time employed 2,400 people (though it ended up maintaining a reduced presence at the site).

In the late 2000s/early 2010s, Pfizer, like many other big pharma companies, was experiencing pipeline difficulties too, with drugs accounting for 40% of its sales coming off patent, and a series of high-profile failures of drugs in development, such as the anti-cholesterol drug torcetrapib that caused a marked increase in deaths compared with the control group in clinical trials. The news of this disastrous result came days after CEO Jeff Kindler had hailed the drug as potentially “one of the most important compounds of our generation”. Likewise, tanezumab, an anti-osteoarthritic, failed in trials.

However, these challenges in the core mission of drug discovery led Pfizer to focus on other means of keeping up its dominant position. One thing that highlighted this changed focus was the appointment of Kindler as CEO in 2006. Kindler was trained as a lawyer, and was a relatively new employee when he was given the top job in preference to others of much longer standing with scientific experience, highlighting the increasing importance of legal and marketing issues over traditional R&D. He was succeeded by Ian Read and later Albert Bourla.

Perhaps unsurprisingly for the biggest company in one of the biggest industries in the world, Pfizer has also been proficient in exerting its considerable political influence to preserve its interests, coming in as the 6th largest lobbier in Washington, and spending $US 25 million on lobbying during the passing of Obama’s healthcare reform legislation alone. It has been key in pushing counterfeit drugs up the political agenda, in part due to its ownership of that most counterfeited of drugs, Viagra. It has also been highly critical of parallel trade, and has been one of those militating for a pharmaceutical repackaging ban in the EU.

Despite this political clout, the company also tried to belay its image as a pharma monster, like many others in the industry, by spending generously on charity, donating AIDS drugs both to poor communities in the US, and to developing countries.

The era of mega-mergers

Since the turn of the millennium, Pfizer has embarked on a series of mega-mergers, gobbling up Warner-Lambert in 2000, Pharmacia and Upjohn in 2002, Wyeth in 2009, and Medivation in 2016.

In 2015 the company also paid $17 billion to acquire Hospira, a firm specialising in injectable drugs and biosimilars, at a time when copycat biologics were starting to make real waves in the market. The deal seemed to be a precursor to Pfizer’s plans to separate its patent-protected medicines business from its off-patent portfolio.

These plans were soon abandoned and Hospira has remained a key part of the core Pfizer organisation – but that didn’t put the idea of separate business units to rest completely.

In 2017/2018 Pfizer attempted to sell its consumer health unit, but buyers including Proctor & Gamble and GSK pulled out of negotiations.

This caused Pfizer to change its tactics, and instead the company ended up signing a deal with GSK to combine the two companies’ consumer health businesses and form a joint venture with combined annual sales of $12.7 billion.

Pfizer and GSK plan to divest the business completely in the long term and reap the rewards, while merging the two businesses is also expected to create cost savings for both partners.

Similarly, in 2019 Pfizer announced a deal to merge its Upjohn generics business with Mylan, creating a combined company called Viatris. The $12 billion deal was cleared in November 2020, creating a generics behemoth with annual sales of around $19 to $20 billion and operations in 165 markets around the word.

This era, though, was also marked by two major failed acquisitions, which both courted controversy due to Pfizer’s intention to exploit tax loopholes.

In 2014 the company made an offer of around $100 billion to acquire UK firm AstraZeneca (which at the time was going through a rough patch).

AZ seemed to have little interest in the idea, and the deal was instantly controversial in both Europe and the US. The merger would have created the biggest pharmaceutical company in the world – and would have given Pfizer a way to avoid paying costly US taxes on foreign earnings (a stance that president Barack Obama criticised heavily).

Indeed, critics feared this redomiciling was the main aim of the merger, and that Pfizer wouldn’t sustain investment in UK R&D in the long term.

Unusually, the UK parliament ended up getting involved, perhaps underlining the importance of AZ to the country’s life sciences sector, with both AZ and Pfizer asked to argue for the future of the company in parliamentary hearings. Pfizer seemed unable to allay the concerns of prime minister David Cameron and business secretary Vince Cable.

After numerous “friendly bids” and just as many rejections, Pfizer eventually made a final offer of £69.3 billion ($118 billion) – which was also turned down by AZ, with the company saying it was “inadequate”.

Leif Johansson, AZ’s chairman, did not mince his words, saying: “Pfizer’s approach throughout its pursuit of AstraZeneca appears to have been fundamentally driven by the corporate financial benefits to its shareholders of cost savings and tax minimisation.

“From our first meeting in January to our latest discussion yesterday, and in the numerous phone calls in between, Pfizer has failed to make a compelling strategic, business or value case. The Board is firm in its conviction as to the appropriate terms to recommend to shareholders.”

This did not stymie Pfizer’s desire to move its HQ out of the US, though. The next year it also attempted a ‘reverse takeover’ of Irish Pharma firm Allergan – where, technically, Allergan would acquire the US company and rename itself as Pfizer, allowing Pfizer to have its tax base in Ireland.

At the time $160 billion deal was the biggest ever seen in the pharma sector.

But soon the Obama administration came down hard on such ‘tax inversion’ deals, changing laws such that the deal was no longer attractive to Pfizer.

No signs of slowing down

Despite some setbacks, Pfizer remains one of the biggest pharma companies in the world today. The sheer size of the organisation is mindboggling, totalling well over 100,000 employees. One commentator compared the company’s 38,000 sales reps to “three army divisions”, a sales team that has been immortalised in a Hollywood rom-com of all things – Love and Other Drugs, starring Jake Gyllenhaal and Anne Hathaway.

And with the company becoming one of the first in the world to get a COVID-19 vaccine approved – via its collaboration with  BioNTech – it feels like we’re only on the cusp of seeing where the company could head in the future.

Pfizer’s sheer diversity and economies of scale likely mean it will have the power to shape the pharmaceutical industry well into the 21st century. With fingers in every pie, ranging from small molecules to biologics in every clinical area, to stem cells and consumer goods, Pfizer will surely celebrate its 200th anniversary in as strong a position as it spent the last 160 years.

For all the latest Pfizer news follow pharmaphorum’s Pfizer tag.

The post A history of Pfizer appeared first on .

Genmab axes development of AXL-targeting cancer drug enapotamab vedotin

Genmab has axed development of its pipeline cancer drug enapotamab vedotin after it failed to show enough activity in a proof-of-concept trial.

The drug is an antibody-drug conjugate where a monoclonal antibody is linked to monomethyl auristatin, a cancer-killing “payload”.

It is targeted against AXL, a signalling molecule that is overexpressed in several haematologic and solid malignancies.

Developing enapotamab vedotin formed a major part in the rationale behind the company’s $506 million Nasdaq IPO last year.

Genmab said it will not advance development of the drug after data from expansion cohorts showed it did not meet stringent criteria for proof-of-concept in the early trial.

There was some evidence of clinical activity but this was not “optimised by different dose schedules and/or predictive biomarkers” the company said in a statement.

The company will instead prioritise development of other drugs in its pipeline.

AXL overexpression is thought to drive several cancer processes, including metastasis, tumour angiogenesis, resistance to chemotherapy and targeted agents, and decreased antitumor immune response.

Enapotamab vedotin is fully owned by Genmab and the drug linker technology used for enapotamab vedotin was licensed from Seagen Inc, formerly known as Seattle Genetics.

Jan van de Winkel, CEO of Genmab, said: “We are committed to developing innovative antibody products for patients with cancer, however the data from the enapotamab vedotin expansion cohorts unfortunately does not support moving this product candidate forward.

“This decision will allow us to focus more of our resources and energy on other programs in our robust next-generation antibody therapeutics pipeline.”

AXL is also being targeted by the Norwegian biotech BerGenBio, which has also been looking at using the pathway to fight inflammation seen in COVID-19.

The company’s bemcentinib is under development as a combination and single agent therapy in lung cancer and leukaemia, as well as COVID-19.

It also has an anti-AXL antibody, tilvestamab, which is undergoing phase 1 testing in cancer.

The post Genmab axes development of AXL-targeting cancer drug enapotamab vedotin appeared first on .

Online Live Team Pharma Forecasting Training Now Available

Working remotely can have its challenges, why not get together for a fun and rewarding learning experience and refresh your forecasting skills?

Following the success of J+D Forecasting’s face to face training, the content has been redesigned to make it suitable for live, online audiences.  There are two types of courses, type one is tailored to your objectives and delivered by experienced forecasting professionals via a sharing platform. Your team is interviewed prior to the training and the focus of the training is agreed in advance with you and your colleagues.

The second type of training allows you to choose from a selection of pre-set courses that are completed when you choose. The most popular independent courses are the Fundamentals of Forecasting and Oncology Forecasting courses.

The training is suitable for anyone involved in pharmaceutical forecasting who wishes to refresh or learn new skills. It is particularly useful for Forecasters, Marketeers, Analysts and Market Researchers.

In addition, trainees gain access to FC+ software, case studies and quizzes, plus reminder cards.
For further information get in touch: [email protected] or visit jdforecasting.com

The post Online Live Team Pharma Forecasting Training Now Available appeared first on .

Y-mAbs claims FDA OK for neuroblastoma drug Danyelza

Y-mAbs Therapeutics has claimed its first product approval, getting a green light from the FDA for Danyelza for the rare cancer neuroblastoma.

Danyelza (naxitamab) is an antibody that targets GD2, a protein found on the surface of some tumour cells, and it has been cleared by the US regulator for use alongside GM-CSF – a cytokine drug – as a second-line neuroblastoma treatment after a priority review.

The drug can be used in in children aged over one and adults with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow, who have had a partial response, minor response, or stable disease on prior treatment.

Before now there was no approved treatment for these patients in the US. Y-mAbs has previously said it thinks there are around 300 children a year in the US who fall into this category.

Danyelza is one of two drugs for neuroblastoma that Y-mAbs has been pushing through late-stage development, but the biotech suffered a setback with its other candidate – B7-H3-targeting omburtamab – after the FDA refused to accept its marketing application last month.

Neuroblastoma underpins the R&D effort at Y-mAbs, which was set up by Thomas Gad after his daughter was treated for neuroblastoma at Memorial Sloan-Kettering Cancer Centre in the US. Both the antibodies stem from research carried out at the institution.

“It’s very exciting to see this treatment go from being an experimental therapy used at my daughter’s bedside to now being FDA approved,” said Gad, who is chairman and president of Y-mAbs.

“I want to thank all the patients and physicians who took part in our clinical trials and our scientific partner, Memorial Sloan Kettering, for helping us achieve this goal.”

Neuroblastoma is a cancer of nerve tissue that usually occurs in children and arises in the nervous system, outside the brain, although aggressive forms can spread quickly to the central nervous system as well as the bone and bone marrow.

Danyelza has been granted accelerated approval by the FDA on the strength of two open-label phase 2 studies. The first – study 201 – included 22 patients and showed an overall response rate of 45% with the antibody/GM-CSF combination, with 30% of responders seeing a benefit that lasted for at least six months.

The second, 38-patient trial (Study 12-230) had an overall response rate of 34%, with 23% of subjects having a response duration of six months or more. Progression-free survival and overall survival are secondary endpoints in long-term follow-up, with the data not yet available.

Y-mAbs said last month it is expecting to resubmit omburtamab for approval to the FDA in late 2020 or early 2021, and will also start clinical trials of a next-generation version of the drug before year-end in neuroblastoma as well as B7-H3-positive leptomeningeal tumours.

With Danyelza now approved as a second-line therapy, Y-mAbs’ aim is now to talk to the FDA about what would be needed to advance the antibody into front-line treatment, according to the company’s chief executive Claus Møller.

Two single-centre studies in previously-untreated neuroblastoma patients are already underway, with preliminary data from one available and the other due shortly. And combination trials with chemotherapy are also planned.

The post Y-mAbs claims FDA OK for neuroblastoma drug Danyelza appeared first on .

Evotec Collaborates with Rappta Therapeutics to Focus on Oncology Target

Shots:

  • Evotec to receive research funding & is eligible for milestones. Evotec will support Rappta’s program of developing small molecule activators of the enzyme PP2A
  • The collaboration leverages Evotec’s integrated platform for drug discovery and development and its oncology expertise coupled with state-of-the-art technologies
  • PP2A is a serine/threonine phosphatase that functions as a tumor suppressor by negatively regulating multiple oncogenic signaling pathways responsible for driving cancer progression

Click here ­to­ read full press release/ article | Ref: Evotech | Image: Personal Financial

The post Evotec Collaborates with Rappta Therapeutics to Focus on Oncology Target first appeared on PharmaShots.

Catamaran joins the CAR-NK fleet, raising $42m in first round

Catamaran Bio has weighed anchor with a $42 million first-round financing that will be used to pull its off-the-shelf natural killer (NK) cell therapies for cancer through early-stage development.

The Cambridge, Massachusetts-based biotech is the latest player in the emerging field of chimeric antigen receptor (CAR) NK cell therapies, which unlike the current generation of CAR-T therapies can be sourced from healthy donors, rather than patients themselves.

CAR-NK therapies also have the potential to be used to treat solid tumours, something that has so far eluded CAR-Ts, which to date have only been approved for blood cancers. Solid tumours tend to be a tougher treatment proposition for cell therapies, as they are harder to penetrate and are often swamped with immunosuppressive factors.

CAR-NK therapies consist of NK cells that have been modified with a CAR molecule, which allow them to bind to and attack a cancer cell.

Earlier this year, researchers at MD Anderson Cancer Centre in the US – a pioneer of CAR-NK – said they have achieved a 73% response rate with a CD19-targeting therapy derived from donated cells in patients with relapsed or refractory non-Hodgkin’s lymphoma and chronic lymphocytic leukaemia.

Catamaran’s cash injection comes just nine months after Catamaran was founded to build on research conducted by its scientific founders – George Washington University’s Catherine Bollard and University of Minnesota’s Branden Moriarty.

The other founders include Houman Ashrafian, Tim Harris and Kevin Pojasek of SV Health Investors, and Obsidian Therapeutics and Serien co-founder Vipin Suri, who will serve as Catamaran’s chief scientific officer.

Catamaran’s proprietary platform – called Tailwind – consists of a novel way to design and engineer improved CAR-NK cells using synthetic biology.

One element is the “architecture” or the CAR molecule, and Catamaran says it has novel structures in play that stimulate production of immune cytokine molecules by the CAR-NK cell, enhancing the cell-killing action.

Another is the delivery of molecule switches to CAR-NK cells, used to activate biological pathways that can help overcome the immunosuppressive microenvironment around tumours and improve recruitment of other elements of an immune response.

Finally, Tailwind includes a non-viral delivery system for engineering the CAR-NK cells based on transposons that make them easier and potentially cheaper to manufacture.

Viral vectors are limited in the size of genetic payloads they can deliver, but using transposons – a “jumping” DNA sequence that can change its position within a genome – sidesteps this issue and allow multiple CARs and switches to be delivered in one go.

Catamaran has two lead CAR-NK programmes at the lead optimisation stage, both of which target the same, undisclosed antigen that is found on both solid tumour and blood cancer cells. The lead programme is targeting a blood cancer.

“Catamaran is focused on expanding the frontier of cell therapies to treat solid tumours and provide transformative benefit to cancer patients,” said Suri.

“We are doing this by creating allogeneic cell therapies that harness the innate cancer-fighting power of NK cells and enhancing them with new biologically-powerful attributes from our leading-edge technologies,” he added.

Sofinnova Partners and Lightstone Ventures co-led the Series A, with SV Health Investors, Takeda Ventures and Astellas Venture Management also taking part.

Other groups developing CAR-NK therapies include Takeda, which has partnered with MD Anderson, as well as Artiva, Nkarta, Fate Therapeutics, oNKo-innate, Kuur Therapeutics, and ONK Therapeutics.

The post Catamaran joins the CAR-NK fleet, raising $42m in first round appeared first on .

$200M investment to advance clinical programs for Ambrx

The San Diego-based biopharma firm has several oncology therapies in development and has partnered in the past with the likes of Astellas, BeiGene and Bristol-Myers Squibb.

FDA sets May date for verdict on ADC’s lymphoma drug Lonca

ADC Therapeutics could claim its first product approval in the US next May, after the FDA started a priority review of lead drug Lonca for diffuse large B-cell lymphoma (DLBCL), an aggressive form of non-Hodgkin’s lymphoma.

Lonca – short for loncastuximab tesirine (formerly ADCT-402) – is an antibody-drug conjugate that combines an antibody against CD19 linked to a cell-killing drug. If approved, it would be a rival to CD19-targeting cell therapies from Novartis (Kymriah) and Gilead Sciences (Yescarta) in the third-line treatment of DLBCL.

The FDA has started a six-month review of Lonca for the treatment of relapsed or refractory DLBCL, with an action date of 21 May next year, and ADC says it hopes to have the drug on the market before the end of 2021.

Chief executive Chris Martin said the Swiss biotech has been working on building its salesforce in anticipation of the positive verdict from the FDA, and reckons it can now cover 90% of haematology and oncology specialists who treat DLBCL in the US.

The FDA is reviewing the ADC based mainly on the results of the LOTIS 2 trial, which Lonca in patients with relapsed or refractory DLBCL following two or more lines of prior systemic therapy. Around 40% to 50% of DLBCL patients are refractory to or relapse after front-line therapy.

The single-arm, 145-patient trial showed an overall response rate of 48.3% and a complete response rate of 24.1%, with Lonca as of a 6 April data cut-off point.

“Based on feedback from physicians on Lonca’s efficacy, tolerability protocol and ease of administration, we believe Lonca has the opportunity to become the standard of care in third-line, based on our competitive profile versus other available options,” Martin told analysts on a conference call earlier this month.

ADC Therapeutics is also testing Lonca in earlier lines of therapy. It is running a phase 3 trial (LOTIS 5) of the medicine in combination with rituximab in second-line DLBCL patients, and a phase 1/2 study (LOTIS 3) of Lonca paired with AbbVie/Johnson & Johnson’s Imbruvica (ibrutinib) with relapsed or refractory DLBCL or mantle cell lymphoma (MCL).

The Swiss company is also planning to begin a dose finding study of Lonca in combination with R-CHOP chemotherapy in previously untreated DLBCL patients in the first half of 2021, according to Jay Feingold, its chief medical officer.

In the third-line setting, the two approved CAR-T therapies – Yescarta and Kymriah – are making headway but like all autologous cell therapies require a complex treatment pathway that involves harvesting of immune, growth and modification of the cells outside the body, and the reintroduction of the modified cells to fight that cancer.

While the efficacy can be dramatic and CAR-T’s are potentially curative, the procedure itself can have significant side effects, and is labour-intensive and expensive to carry out.

Other competition is also emerging in relapsed/refractory DLBCL, and that is good news for patients desperately in need of new treatment options.

Among these is MorphoSys and Incyte’s CD19-targeting monoclonal antibody Monjuvi (tafasitamab), which was approved in combination with Bristol-Myers Squibb/Celgene’s Revlimid (lenalidomide) as a second-line treatment for DLBCL in August.

Roche’s ADC Polivy (polatuzumab) – which targets CD79b – was meanwhile approved for third-line DLBCL treatment last year in the US and earlier this year in Europe.

The post FDA sets May date for verdict on ADC’s lymphoma drug Lonca appeared first on .

COTA Lands $34M to Drive Innovation in Oncology Using Real-World Data

COTA Lands $34M to Drive Innovation in Oncology Using Real-World Data

What You Should Know:

– COTA, Inc., a healthcare technology company that uses
real-world data to bring clarity to cancer care, has secured $34M in funding.

– Access to over one million patient data records and additional
funding support enhanced real-world data and analytics services in oncology.


COTA, Inc., a Boston, MA-based healthcare technology company that uses real-world data to bring clarity to cancer care, today announced it has raised $34 million in Series D funding led by Baptist Health South Florida and ONC Capital with participation from EW Healthcare, Horizon BCBS and other existing investors. This also includes a $20M investment from Varian, who negotiated an option to acquire COTA at a later date.

Bringing Clarity to Cancer

COTA was founded in 2011 by a team of doctors, engineers,
and data scientists to create clarity from fragmented and often-inaccessible
real-world data. The company organizes fragmented, often hidden data from the
real world to provide clarity in cancer care. Combining clinical expertise in
cancer with proprietary technology and advanced analytics, COTA’s platform
helps inform decisions and action in oncology. COTA partners with
providers, payers, and life science companies to ensure that everyone touched
by cancer has a clear path to the right care.

COTA offerings include:

Providers: Curate EHR data that can be
used to drive research and standardization in order to improve patient outcomes
while also reducing costs at your institution.

Payers: Make clinical sense of claims data, providing
insight into performance and related outcomes across sites in order to support
value-based payment models.

Life Sciences: Deeplycurated real-world data accelerates drug development informing the decisions and actions that will deliver the best drugs to patients faster and at a lower cost.

Recent Traction/Milestones

The company will use the latest round of funding to expand
its data access by over 300%, which now well exceeds one million oncology
patient records. This growth will support the company’s commitment to
accelerating the use of real-world data to improve patient outcomes and
increase efficiency in oncology drug development. data helps life science
partners answer key research questions, and in 2020 alone, COTA is projected to
double its life science customers, growing from 8 to 16.

“COTA is proud to receive this validation from leading institutions across the oncology ecosystem.” said Michael Doyle, President and CEO of COTA. “The additional capital combined with our increased data access positions COTA for tremendous growth and enables us to drive innovation in oncology using real-world data. Our high-quality data and technology solutions will improve how cancer is treated and provide much needed clarity to patients as they navigate their cancer journey.”

TGFB for Immuno-Oncology Drug Development Summit

Are you interested in finding the next breakthrough in immuno-oncology preclinical or clinical development?

Recent scientific, clinical breakthroughs and high-profile industry deals have reignited the race to find the next blockbuster TGF-ß inhibitor for immuno-oncology applications.

As such, the TGFß for Immuno-Oncology Drug Development Summit (January 26-28) is the ONLY industry-focused meeting dedicated to pharma, biotech, and academia who will share their latest data, best practices, and top tips to expedite TGF-beta candidates into the clinic in a safe and efficacious manner.

With over 21 expert speakers presenting across 3 days packed full of content, this is your definitive guide to tame the TGF-beta double-edged sword and navigate a narrow therapeutic window, enhance tumor suppression, and maximize therapeutic potential in immuno-oncology – you won’t want to miss it!

Take a look at the full program here

Here’s a sneak peek into the world-class speaker faculty:

  • Shannon Turley, Senior Director, Cancer Immunology, Genentech
  • Rik Derynck, Professor – Departments of Cell, Tissue Biology & Anatomy, University of California San Francisco
  • Thomas Schurpf, Associate Director, Scholar Rock
  • Olaf Christensen, Vice President, Head of Bintrafusp alfa, Global Development, R&D, EMD Serono
  • Paul Rennert, President & Chief Scientific Officer, Aleta Biotherapeutics
  • Sam Shrivastava, Chairman & Chief Executive Officer, Venn Therapeutics
  • Vuong Trieu, Chairman & Chief Executive Officer, Oncotelic Inc
  • Christopher Heery, Chief Medical Officer, Precision BioSciences

Download your digital event guide here to access the full speaker faculty and conference agenda.

How can this get any better I hear you ask? Well, through our specialized digital platform, you can enjoy the full conference experience all from the comfort of your own home (without the bad conference coffee and awkward encounters at the buffet table). Using our algorithm get matched with fellow attendees and speakers for meaningful networking opportunities.

For more information visit www.tgf-beta-summit.com or email us at [email protected].

The post TGFB for Immuno-Oncology Drug Development Summit appeared first on .

Great creative is effective creative. And effective creative is affective creative

Our creative philosophy has always been simple. To produce works that works. Effective, impactful brand communications that deliver tangible results. As the great adman Bill Bernbach said: “If it doesn’t sell, it isn’t creative.”

And if you want to sell or change behavior, you need to reach your target audience with evocative creativity. That’s why we believe for creative to be truly effective, it needs to first be affective.

Our creative approach at Fishawack Health is Affective Creativity. Creative crafted to make deep, motivating connections with our target audience. We tap into psychology and combine that with creative craft and expertise and to deliver powerful, engaging work on a conscious as well as a subconscious level.

We’re learning more and more about the power of creativity itself every day. One study found that creativity can double the impact of every marketing dollar spent.  That’s powerful stuff.

Here’s what we now know for sure from the science:

  • Creativity sells.
  • Concepts and storytelling are the most effective ways of conveying information. (And the better the idea, the better the impact).
  • People are motivated by conscious and subconscious goals, which strongly influence choices.
  • 95% of decisions are made subconsciously.
  • Emotions drive action.
  • Craft matters. For example, that the active voice is more powerful than the passive; that something labelled ‘tender chicken’ is more desirable than just ‘chicken’.  Visually, we know that certain colours evoke certain emotions, and that shared cultural concepts can be powerful shortcuts to subconscious connections.

When it all comes together, and you hit that affective sweet spot, the result is strategically sound and effective creativity.

One shining example of this can be found in the Melanoma Likes Me campaign from WPP in Australia:

Melanoma Likes Me

Melanoma haunts outdoor, in-the-sun Australians. It’s the most lethal cancer for those between 15 and 30. To reach this elusive group, George Patterson Y&R created a mobile campaign for Melanoma Patients Australia, Melanoma Likes Me. An application searched popular sun-related hashtags and geolocations for posts from young Australians in the sun. Then, the creepy online persona ‘Melanoma’ – would ‘like’, ‘follow’ and comment, in real time.

It’s a brilliantly creative way to make an impact with the key target audience of young, sun-loving Australians, in an environment where they operate. Using social media itself as a vehicle to personify melanoma and physically demonstrate the way it sneaks up on you, unsuspected, is just inspired.

It’s affective and effective. So simple, so clever, so insightful.

And even though it’s utilizing the technology superbly, the idea is built around the audience first – where they are and what they’re doing and how to get their attention.

Technology changes but humans don’t

And that’s an important point about technology.  The creative industry tends to be obsessed with the new.  We love the latest tech, the latest ‘thing’. “This changes everything” is something we all hear ad nauseum.  And yet humans haven’t really changed since homo sapiens first emerged.

Again, as Bill Bernbach observed: “It took millions of years for man’s instincts to develop. It will take millions more for them to even vary. It is fashionable to talk about changing man. A communicator must be concerned with unchanging man, with his obsessive drive to survive, to be admired, to succeed, to love, to take care of his own.”

That’s why we should be obsessed with the customer not the technology. Provided your audience is at the heart of your idea, then technology is just a means to connect with them. Of course, you need to understand how they use the technology, why they use it, what they’re getting from it.  But the technology should never lead your creativity; that should always be your audience.

How to have great ideas. (It’s not a magic trick).  

Although creativity can be magical, coming up with ideas is anything but.

It all starts with a great brief. Creatives need is clarity, direction, and inspiration.  An inspirational brief, built upon deep insight, will ensure that your creatives are thinking of ideas before you’ve even finished the verbal briefing.

The other thing you really need, which seems like it is in shorter supply these days, is time.  Because in order to think of truly exceptional and breakthrough ideas, your brain needs permission to think and explore. James Webb Young’s seminal 1965 work, A Technique for Producing Ideas lays it out best.  He emphasises the point that the best ideas come when you stop consciously thinking about the brief.  That’s when your subconscious kicks in and starts making creative connections.

It’s a cliché that we have our best ideas in the shower, but it’s not far off. Some of our best ideas tend to come anywhere but at our desks (or nowadays our home office, kitchen table, or couch).

So, time to pause and switch to thinking about something else is vital process of great or even good ideas.

Do brainstorms work?

Of course we don’t always have the luxury of lots of time. Sometimes we need to move faster, and pool our resources and minds together. But the preparation work and the brief needs to be even more tightly screwed down.

We’ve developed various brainstorming formats designed to get initial ideas quickly and to a decent standard – including Pack Hack, Scamper and the 1-2-3 Interval technique.

But be careful, the solution to your problem will likely not come out of your brainstorming session.

The watchout is trying to solve your problem in the session. Brainstorming will reveal different avenues and undeveloped ideas that will help you get to the best solution. It will jump-start brains and fill minds with stimuli. It’s a push down the slope, but the road is still long ahead with more ups and downs, and a lot of pedaling to be done.

At the end of the day, ideas take shape and are crafted through the hands of individuals and dedicated teams. Someone needs to write it up, sketch it up, comp it up, deck it up. Check it. Prototype it. Present it. Sell it. And make it. But a good brainstorm can definitely help that get that process going if you have the right people working together.

To find out how we can help you to produce creative work that works, contact [email protected] 

The post Great creative is effective creative. And effective creative is affective creative appeared first on .

Roche’s Herceptin/Perjeta combo product nears EU approval

The EMA’s human medicines committee has recommended approval of a fixed-dose combination of Roche’s breast cancer drugs Herceptin and Perjeta – part of the company’s defense against Herceptin biosimilars.

Called Phesgo, the new product contains the active substances in Herceptin (trastuzumab) – already facing lower-cost competition in the US and Europe – and Perjeta (pertuzumab), which are both used to treat HER2-positive breast cancers.

Phesgo comes in a single-dose vial and can be dosed as a subcutaneous injection, while at the moment breast cancer patients the two anti-HER2 drugs separately receive Herceptin as a subcutaneous injection and Perjeta as an intravenous infusion, says the CHMP.

According to the committee, the combination offers “less invasive and faster administration as a single product” to patients. After a first eight-minute loading dose, each treatment takes about five minutes to administer and can be carried out at home.

In contrast, Herceptin/Perjeta administration needs to be done in a clinic and takes more than two hours for the first dose and an hour thereafter. Aside from the convenience, avoiding the need to go into a treatment centre is desirable during the coronavirus pandemic, according to Roche.

Phesgo has been recommended by the CHMP for use in combination with chemotherapy in early HER2-positive breast cancer – pre- and post-surgery – as well as for front-line therapy of HER2-positive breast cancer that has already spread in the body.

A CHMP recommendation is usually followed by EMA approval within a few weeks. Phesgo is already available in the US, having been approved by the FDA in the summer.

Perjeta has become one of Roche’s fastest-growing and top-selling drugs, thanks in no small part to data from the APHINITY trial which showed that adding it to Herceptin can improve disease-free survival in early-stage breast cancer.

Despite the impact of the pandemic on cancer care, Perjeta grew 17% to CHF 2.9 billion ($3.2 billion) in the first nine months of 2020, now just fractionally behind Herceptin which declined 31% to CHF 3.1 billion in the same period as biosimilars ate into its market share.

That makes Perjeta a particularly important component in Roche’s product range, particularly as two other big-selling antibodies – Avastin (bevacizumab) and Rituxan/MabThera (rituximab) – are also being hit by biosimilar competition.

Rolling out a subcutaneous formulation has already allowed Roche to mitigate the impact of copycat versions of Herceptin and Rituxan, and it is hoping it can repeat the trick with Phesgo, as Perjeta’s patents start to expire within the next four years.

It’s too early to say if that strategy will be successful. Roche revealed in its third quarter results statement that initial US sales of Phesgo were CHF 7 million, with the company still working on gaining formulary approvals in the US.

Xofluza also recommended

Roche also claimed a CHMP recommendation for Xofluza (baloxavir marboxil), a one-dose oral therapy for influenza that is a successor the company’s former blockbuster Tamiflu (oseltamivir), which has also lost patent protection.

Xofluza has been given a positive opinion by the CHMP as a treatment for uncomplicated influenza in people aged 12 and over, and to prevent infection in people exposed to someone with flu. The drug was approved by the US FDA last year.

The post Roche’s Herceptin/Perjeta combo product nears EU approval appeared first on .

NICE okays Roche liver cancer immunotherapy, Sanofi’s rare disease drug

Previously-untreated patients with hepatocellular carcinoma (HCC), a common form of liver cancer, will have an immunotherapy-based treatment option after NICE gave the nod to NHS funding of Roche’s Tecentriq and Avastin combination therapy.

The decision by NICE comes shortly after Tecentriq (atezolizumab) and Avastin (bevacizumab) was approved by the European Commission as a first-line treatment for adults with advanced or inoperable HCC, becoming the first and only immunotherapy regimen approved in Europe for this patient group.

Approval was based on findings of the phase 3 IMbrave 150 study, which showed an improvement in overall survival compared with Bayer’s Nexavar (sorafenib), one of the standard treatments for previously-untreated liver cancer along with Eisai’s Lenvima (lenvatinib).

NICE’s final appraisal document notes that patients receiving the dual regimen “live longer and have longer before their disease progresses than people who have sorafenib” and it is a cost-effective use of NHS resources.

It is the first treatment to be approved in Europe for over a decade that has improved overall survival for people with previously untreated advanced or unresectable HCC.

HCC accounts for 90% of all liver cancer cases and there are around 5,900 new cases in the UK every year. The disease is also becoming more common – Roche says the incidence of HCC is projected to rise by 38% to 15 cases per 100,000 people by 2035.

“Tecentriq, in combination with Avastin, is the only cancer immunotherapy treatment, approved by NICE for this indication,” said Gemma Boni, head of liver cancer at Roche Products Ltd.

“We hope that it can help further improve overall survival rates, while having a generally manageable safety profile, compared to what is already achieved,” she added.

The Tecentriq/Avastin combination is crucial to Roche’s immuno-oncology franchise, and along with HCC has also been approved to treat non-squamous non-small cell lung cancer (NSCLC).

Sales of Tecentriq sales rose 64% to around $2.2 billion in the first nine months of this year, but Avastin has started to succumb to biosimilar competition, shrinking 22% to a little over $4 billion in the same period.

Cablivi gets green light for aTTP

NICE’s recommendation that Sanofi’s Cablivi (caplacizumab) can be covered by the NHS, is also a big step forward for patients with acquired thrombotic thrombocytopenic purpura (aTTP), a rare blood-clotting disorder that can be fatal without prompt treatment.

Cablivi is the first treatment specifically approved for aTTP in 30 years, and the only therapy approved for people experiencing an acute aTTP episode. NICE has backed the drug after rejecting it earlier this year due to an “improved commercial arrangement” with Sanofi – in other words a price discount.

Cablivi was also recommended for NHS use by the Scottish Medicines Consortium (SMC) in September.

aTTP is a rare disease where blood clots form in small blood vessels throughout the body, limiting the flow of oxygen to organs, such as the brain, kidney and heart. NICE’s appraisal document says that Cablivi, combined with plasma exchange and immunosuppression, reduces time to bring blood platelet levels back to normal.

Sanofi welcomed the positive recommendation, but said access to Cablivi had been delayed by procedural issues at NICE.

As a treatment for an ultra-rare disease, Cablivi was initially supposed to be considered within the Highly Specialised Technology (HST) process, but was assessed under the Single Technology Appraisal (STA) process.

The STA does not consider the concerns around long-term follow-up and management, which are a well-known issue in evaluating medicines for ultra-rare diseases, says the company, which claims this delayed access to the drug.

“We now hope that the forthcoming NICE Methods Review and Innovative Drugs Fund consultation will fix the ongoing barriers for patients,” commented Deborah Lough, head of rare blood disorders UK & Ireland at Sanofi Genzyme.

NICE estimates that more than 100 people each year will be eligible for treatment with Cablivi.

Image by PublicDomainPictures from Pixabay

The post NICE okays Roche liver cancer immunotherapy, Sanofi’s rare disease drug appeared first on .

Janssen combines Darzalex with COVID drug in new multiple myeloma submission

Janssen has submitted a new combination regimen for its blockbuster multiple myeloma drug Darzalex for approval with the FDA and EMA, hoping to give the treatment another edge over emerging competitors.

Specifically, the combination utilises the subcutaneous formulation of the drug, Darzalex Faspro (daratumumab/hyaluronidase) with Celgene’s multiple myeloma drug Imnovid (pomalidomide) and dexamethasone – which has gained new fame this year as one of the few treatments available for COVID-19.

Darzalex Faspro was approved earlier this year and cuts the drug’s dosing time to just a few minutes from hours.

Janssen is seeking approval for use in the treatment of patients with relapsed or refractory multiple myeloma who have received at least one prior line of therapy. 

The submission is supported by data from the phase 3 APOLLO study, which showed significantly longer progression-free survival (PFS) in patients with relapsed or refractory multiple myeloma who received the combination compared with pomalidomide and dexamethasone alone. 

Analysts think the shorter treatment of Darzalex Faspro time could become a particularly important advantage over the established formulation during the coronavirus pandemic, when getting access to healthcare has become more difficult under lockdown, and this combination could further boost the drug’s profile.

In September Janssen also submitted the formulation for approval in the rare and potentially fatal disease light chain amyloidosis.

The original IV version of Darzalex has been a big earner for J&J ever since it was first approved in 2015 as a fourth-line myeloma therapy, growing rapidly on the back of successive approvals in earlier lines of therapy – including most recently in newly-diagnosed patients.

The product brought in almost $3 billion in sales last year, and shows no signs of slowing down, growing another 49% to reach $937 million in the first three months of 2020. J&J licensed the drug from Danish biotech Genmab in 2012 in a deal valued at $1.1 billion.

Last month however saw the approval of its first direct competitor – Sanofi’s anti-CD38 antibody Sarclisa (isatuximab) – which was given the nod by the FDA as a third-line therapy for myeloma.

Sanofi’s drug is delivered intravenously and – according to Sanofi – has simpler, two-hour dosing that improves on the current Darzalex formulation, although analysts think the availability of J&J’s new subcutaneous version will likely cancel out that advantage.

Last year, J&J licensed a follow-up to Darzalex from Genmab, HexaBody-CD38, that it says could be used in Darzalex-resistant patients as well as in other cancer indications beyond myeloma, in the hope of extending its anti-CD38 franchise.

 

The post Janssen combines Darzalex with COVID drug in new multiple myeloma submission appeared first on .

German biotech CatalYm raises €50 million for GDF-15 inhibitor immunotherapy

CatalYm has closed a €50 million ($59m) series B financing round to fund clinical studies of its immunotherapy targeting Growth Differentiation Factor 15 (GDF-15).

The round was led by Vesalius Biocapital III, with participation from Novartis Venture Fund (NVF), Wachstumsfonds Bayern, coparion and founding investors Forbion and BioGeneration Ventures.

Founded in 2016 as a spin-out from Wuerzburg University, CatalYm’s lead molecule CTL-002 is designed to neutralise the tumour-produced protein GDF-15. High concentrations of the GDF-15 in serum and tumour-micro-environment help cancers to evade the immune system and are associated with resistance to current therapies.

The therapy originated from research by company founder Professor Wischhusen, looking at similarities between how a tumour protects itself from attacks by the immune system and how foetuses grow during pregnancy with protection from the immune system of the mother.

“In both cases, the tissue is growing very rapidly and aggressively and there is a need for vascularisation, nutrients and to escape the immune system. In pregnancy, of course this is what is needed and allows a baby to grow. But if a tumour is doing the same then it is going to kill a patient,” CatalYm CEO Dr Manfred Ruediger told pharmaphorum. “A tumour often hijacks these mechanisms which benefit the foetus and that is how the whole story started.”

CTL-002 addresses three of the tumour’s immune suppressive mechanisms all involving the inhibitory effect of GDF-15 on the immunostimulatory LFA-1/ICAM-1 interaction. By neutralising GDF-15, CTL-002 is expected to enhance infiltration of immune cells into the tumour, improve priming of T-cells by dendritic cells and improve the tumour killing by T-cells and NK-cells.

The company is expecting to be at clinical stage by the end of the year. Proceeds from the series B raise will be used for a Phase I escalation trial. CatalYm will also test the compound in combination with approved checkpoint blockers. If shown to work with approved immuno-oncology drugs, it could benefit patients that relapse or are in refractory from current therapies.

According to Ruedinger, investors believed the clinical program had more potential compared to others in immunotherapy. “We are not another company activating immune cells or improving antigen exposure,” he said. “We are the only one which is working on the step where immune cells must leave the vessels which nourish the tumour and enter the tumour tissue properly. It’s fascinating for all of us and we are looking forward to the next stage.”

The post German biotech CatalYm raises €50 million for GDF-15 inhibitor immunotherapy appeared first on .

Five Prime eyes financing after positive results with stomach cancer drug

Shares in US biotech Five Prime Therapeutics more than tripled after it reported promising trial results with stomach cancer drug candidate bemarituzumab, returning the stock to levels not seen since 2018.

The phase 2 FIGHT trial testing bemarituzumab plus chemotherapy as a front-line therapy for advanced gastric and gastroesophageal junction cancer met all its efficacy objectives, with significant improvements in overall survival, progression-free survival and overall response rate compared to chemo alone.

Fuelled by the positive results, Five Prime promptly announced plans for a public offering to fund further clinical development of bemarituzumab and other pipeline programmes.

The scale of the stock recovery stems largely from the difficulties experienced by Five prime in the last few years, which have kept its shares pegged down.

In particular, the demise of immuno-oncology drug cabiralizumab – an anti-CSF-1R antibody previously partnered with Bristol-Myers Squibb that failed a pancreatic cancer trial earlier this year – has weighed heavy on its shares.

The FIGHT results raise the possibility that bemarituzumab – an antibody which targets fibroblast growth factor receptor 2b (FGFR2b) – could become the first new drug for previously-untreated advanced gastric cancer in over a decade, according to Five Prime’s chief medical officer Helen Collins.

FIGHT enrolled 153 patients with FGFR2b-positive, HER2-negative gastric tumours, and compared Five Prime’s antibody to the mFOLFOX6 chemotherapy regimen, a standard treatment for this type of cancer – the third leading cause of cancer death globally.

In the study, the median PFS improved from 7.4 months to 9.5 months. Median overall survival was 12.9 months with chemotherapy, but hadn’t been reached in the antibody group, which Five Prime said equated to a 42% risk reduction at the data cut-off point.

It’s worth noting however that Five prime used a p value threshold for statistical significance of 0.2, higher than the more usual 0.05, which the company said wasn’t unusual for a small trial. The PFS result came in at 0.073 so would have missed that tougher threshold, although for overall survival the p value was 0.027.

The tolerability of the antibody plus chemo regimen is also in question, as around a third of patients discontinued from that arm, compared to 5% with chemo alone.

Around a third of all HER2-negative stomach cancers are FGFR2b-positive, and overexpression of the biomarker tends to be associated with poorer survival. After binding to the receptor, bemarituzumab blocks a pathway that promotes tumour growth, and also stimulates the immune response to attack the malignant cells.

Five Prime reckons it is well ahead of any rivals as there are currently no other FGFR2b-specific antibodies in clinical development. FGFR2b overexpression is also seen in lung, colorectal, pancreatic, liver and breast cancer, amongst other tumour types.

Analysts have suggested that bemarituzumab’s potential could make Five Prime a buyout candidate, with BMS a possible candidate – despite the cabiralizumab fail – as it needs to build momentum in its immuno-oncology franchise.

The post Five Prime eyes financing after positive results with stomach cancer drug appeared first on .

Merck & Co/Eisai eye kidney cancer use for immunotherapy combination

Merck & Co and Eisai are to file results from a phase 3 trial testing a combination of the former’s immunotherapy Keytruda, combined with the latter’s cancer drug Lenvima in untreated patients.

Keytruda (pembrolizumab) and Lenvima (lenvatinib) outperformed standard care – Pfizer’s Sutent (sunitinib) – in untreated patients with advanced renal cell carcinoma when measured against progression-free survival (PFS) and secondary goals of overall survival (OS) and overall response rate (ORR).

The companies said the results in the intention to treat population would be shared with regulators across the world.

Merck & Co, known as MSD outside North America, gave no further details about the results but said that the improvements were “statistically significant and clinically meaningful” in the group that included across all risk group categories – favourable, intermediate and poor.

Results came from the 1,050 patient KEYNOTE-581/CLEAR trial, also known as Study 307, testing Lenvima in combination with Keytruda or in combination with everolimus, with patients taking Pfizer’s Sutent in the control group.

Patients were randomly assigned to three groups, either receiving Lenvima (18mg orally once daily) in combination with everolimus (5mg orally daily), Lenvima (20mg orally once daily) plus Keytuda (200mg intravenously every three weeks), or Sutent daily once daily for four weeks on treatment followed by two weeks off treatment.

The primary endpoint is PFS by independent review per RECIST v1.1 criteria.

Further details will be revealed at an upcoming medical meeting.

Keytruda was approved in combination with Pfizer’s Inlyta (axitinib) in first line kidney cancer last year, which is competing against the combination of BMS’ rival PD-1 inhibitor Opdivo (nivolumab) and CTLA4 inhibitor Yervoy (ipilimumab).

Merck KGaA/Pfizer’s Bavencio (avelumab), was approved for first-line RCC in combination with Inlyta in May last year.

US-based Merck and Eisai are continuing to study the Keytruda plus Lenvima combination through the LEAP (LEnvatinib And Pembrolizumab) clinical program across 19 trials in 13 different tumor types (endometrial carcinoma, hepatocellular carcinoma, melanoma, non-small cell lung cancer, RCC, squamous cell carcinoma of the head and neck, urothelial cancer, biliary tract cancer, colorectal cancer, gastric cancer, glioblastoma, ovarian cancer and triple-negative breast cancer).

 

The post Merck & Co/Eisai eye kidney cancer use for immunotherapy combination appeared first on .

M&A: RxVantage Acquires onPoint Oncology to Expand Offering to Oncology Practices

M&A: RxVantage Acquires onPoint Oncology to Expand Offering to Oncology Practices

What You Should Know:

– RxVantage has acquired onPoint Oncology to provide cancer care teams with on-demand access to educational resources, reimbursement data, and analytics.

– The acquisition of onPoint Oncology builds on
RxVantage’s rapidly expanding digital offerings for providers. In April,
RxVantage launched Virtual Meetings to help providers reestablish access to
life science experts amidst the disruptions caused by COVID-19.


RxVantage, the
leading digital platform connecting healthcare providers to educational
resources and experts from life science companies, today announced the acquisition
of onPoint Oncology,
the leader in oncology reimbursement data and analytics.

The partnership ensures that care teams will have access to
onPoint’s unique revenue cycle
data and reimbursement insights as well as one-click access to educational
resources and expertise from any life science company, all through the free RxVantage digital platform.

The acquisition of onPoint Oncology builds on RxVantage’s
rapidly expanding digital offerings for providers. In April, RxVantage launched
Virtual Meetings to help providers reestablish access to life science experts
amidst the disruptions caused by COVID-19.

onPoint will operate as a wholly-owned subsidiary of
RxVantage. As part of the acquisition, industry veterans Tracy Lewis and Bobbi
Buell will join RxVantage leadership team. Financial details of the acquisition
were not disclosed.

RxVantage Background

Founded in 2007, RxVantage’s mission is to ensure that every
provider has the most relevant information and data on medications and medical
technologies, in order to deliver the best care to every patient. More than
6,000 medical practices across the US utilize RxVantage to connect and engage
with over 50,000 experts from life science companies.

Merck & Co buys cancer biotech VelosBio for $2.75bn

Merck & Co has agreed to buy California biotech VelosBio and its experimental cancer drug for $2.75 billion in cash.

Known as MSD outside North America, the big pharma already has a firm foothold in cancer thanks to its immunotherapy Keytruda (pembrolizumab), which has been on the market for several years and has proven to be effective across a wide range of oncology indications.

But cancer is a highly competitive field and like rivals such as Bristol-Myers Squibb and Roche, Merck & Co is always looking for relatively small acquisitions that could bring in new technology and raise the bar in terms of safety and efficacy.

Based in San Diego, VelosBio is a privately held biotech specialising in a new class of drug that target receptor tyrosine kinase-like orphan receptor 1 (ROR1).

Its lead candidate in clinical development is VLS-101, an antibody-drug conjugate (ADC) targeting ROR1 that is being tested in a phase 1 and a phase 2 clinical trial for patients with blood cancer and solid tumours, respectively.

A phase 2 trial of VLS-101 in solid tumours began last month, including patients with hard to treat triple-negative breast cancer, hormone receptor-positive and/or HER2-positive breast cancer and non-small cell lung cancer (NSCLC).

VelosBio is also due to present detailed results from a phase 1 trial showing a complete response in seven out of a cohort 15 patients with mantle cell lymphoma and four out of five patients with diffuse large B-cell lymphoma.

Patients in that trial had been heavily pretreated and had failed to respond to other anticancer drugs.

The biotech also has a preclinical pipeline of next-generation ADCs and bispecific antibodies targeting ROR1, which could be used in tandem with VLS-101.

The transaction is expected to close at the end of 2020.

In separate announcements Merck said that Lynparza (olaparib), which it develops in partnership with AstraZeneca, had been approved in two new indications in Europe

The new label extensions cover BRCA1/2-mutated metastatic castration-resistant prostate cancer and first-line maintenance treatment for HRD-positive advanced ovarian cancer.

The post Merck & Co buys cancer biotech VelosBio for $2.75bn appeared first on .

Welcome to the CAR-TCR Summit Europe 2021

Accelerate the Bench to Bedside Development of Novel CAR Immunotherapies for Safe, Effective & Affordable Advanced Therapies

With CAR-TCR therapies on the cusp of achieving global approval, there are still many bottlenecks that are preventing this from becoming the ‘sell-out’ therapy that the field had hoped for.

The CAR-TCR Summit Europe (16th-18th February 2021) will unravel the technical challenges across R&D, translation, scale and delivery to provide your team with the platform to learn, collaborate and gain actionable insights to advance your therapy for clinical and commercial success.

This year’s focused agenda will delve into the technical bottlenecks encountered in every stage of the CAR-TCR drug development cycle, providing more comprehensive analysis from 55+ expert speakers across 3 streams of content including:

Research & Development Track:

  • Explore the emerging CAR-X landscape with Glycostem & Carisma Therapeutics
  • Breach the solid tumour microenvironment with Refuge Biotech Atara Biotherapeutics
  • Optimise target identification with Celyad Anocca AB
  • Investigate next-generation gene engineering techniques with Precision Biosciences

Translation Track:

  • Develop ‘off-the-shelf’ platforms with Leucid Bio
  • Provide a mechanistic rationale for combination therapies with Agenus & Ziopharm Oncology
  • Discuss ‘gold standards’ and innovations in assay development  GlaxoSmithKline & bluebird bio
  • Understand and minimise toxicity with Kite: A Gilead Company & The NHS

Manufacturing & Commercialisation Track:

  • Advance automation for cheaper, faster manufacturing with Gracell Biotechnologies & Glycostem
  • Gain regulatory guidance to initiate clinical trials in Europe from MHRA & Norwegian Medicines Authority
  • Maximise real-world evidence for value demonstration, and innovative reimbursement models with Janssen
  • Expedite batch release and maintain high-quality products with Novartis & Immatics

For agenda details and full speaker line up, find out more here

The post Welcome to the CAR-TCR Summit Europe 2021 appeared first on .

Merck paying $2.75B for cancer therapeutics firm VelosBio

The deal will broaden Merck’s oncology portfolio as it picks up an antibody-drug conjugate, a class of biological drugs that target tumor cells even as the pharma giant jettisons other parts of its business

Be in the room: Turning patient engagement to patient centricity

After three brushes with cancer, Robert Weker understands the patient journey more than many – and he is determined to put that experience to good use.

The avid blogger and passionate advocate retired from his R&D role four years ago to focus on his own health, and now works full time on making sure the patient voice is “in the room” when pharma companies make decisions.

Winding road

“My journey is a bit complicated, because I am a three-time cancer survivor,” said Robert, 60, who lives just outside of Philadelphia in the United States.

“First, it was testicular cancer, back in 1991, then I was diagnosed with liposarcoma in 2010, and pancreatic cancer in 2014.”

His first two cancers were discovered early, and, while he did experience treatment side effects, things were “manageable”, he said.

“My broad ambition is to get medicines and medical solutions that are more accessible, more affordable, and available in a more timely, more patient friendly way.”

“I don’t want to minimise cancer in any way, shape or form, but the testicular cancer was caught early, and the treatment success rate was well over 90%. It was more of an inconvenience. It was like entering a tunnel, but you could already see the light at the end.

“The liposarcoma was a little but different, but it was still caught pretty early and there was a high survival rate. Neither of the cancers significantly disrupted work.”

The pancreatic cancer, however, was a “completely different ball game”.

“At that time, the five-year survival rate was in the order of 7%. So, when I entered that tunnel, it was pretty dark.”

Robert decided to enrol on a clinical trial of chemotherapy plus high dose vitamin D at the University of Pennsylvania. Afterwards, he had to fight for the proton beam radiation therapy his doctor recommended as his insurance company considered it to be experimental.

“I was told my policy didn’t cover it. Even though my entire medical team was saying ‘we can’t do normal radiation because it’s too much exposure and his system as a whole won’t be able to handle it’.

“I am not a passive patient, so I kept calling and calling and finally they said there was a review panel,” said Robert, adding that he was shocked to find the panel did not include a radiation oncologist. “Even the voice of my doctor, who is probably one of the top three in his field worldwide, was being totally muted.”

After a number of reviews and appeals and countless phone calls, the treatment was finally approved but it had taken six weeks and many headaches.

Being in the room

His experiences have made him realise the importance of the patient voice being front and centre of healthcare decisions.

“I want to be in the room where it happens, where the decisions are made, so that it’s not just me, a passive patient sitting in the chemo suite,” he said.

“That means working with pharma companies to influence early decisions around R&D, and providing patient insights throughout the process.”

We also need to think about how other stakeholders – insurance companies, doctors, and hospitals, for example – fit into that, said Robert.

“My broad ambition is to get medicines and medical solutions that are more accessible, more affordable, and available in a more timely, more patient friendly way.”

Explaining why patients were an essential part of that mission, he said: “Everyone is driven by different goals and different stakeholders will have different drivers of what’s important.”

Levelling up

Robert, who works with several pharma companies on a variety of projects, believes that the industry is going in the right direction, but that there is still some way to go.

He described three levels of patient engagement, saying most organisations were currently “between one and two”.

“The first one is engaging the patient. That might call a patient or group of patients in to review a consent form or a trial protocol. They will ask them if it seems reasonable, feasible, or overly burdensome, then take those comments and say: ‘see you later’.”

The second level is more like a patient partnership, he said, using his position as member of one company’s Oncology Patient Council as an example.

“We meet once a month throughout the development cycle to provide feedback. We engage with them on challenges they might be facing, or on specific topics such as patient diversity. It’s a continuous process – a feedback loop – not a one-off interaction,” said Robert.

The next step is true patient centricity, which Robert described as “putting the patient at the centre of the wheel”.

“What that looks like in practice is ongoing involvement and engagement with the patient throughout,” he said, adding that it included making sure interventions were as easy to access geographically, logistically, and physically, as possible.

“It all goes back to this idea of being in the room. Of course, not all patients can be there, but if I can share the insights I have gained from my own journey, under the caveat that all patients are different, then at least they have heard what I have to say,” Robert concluded.


inspirePatient Insights is a monthly series that appears in partnership with Inspire, a company with an online support community of more than 2 million patients and caregivers worldwide.

The post Be in the room: Turning patient engagement to patient centricity appeared first on .

Genprex cancer gene therapy paired with AZ, Merck lung cancer drugs

An experimental gene therapy developed by Texas biotech Genprex will be paired with AstraZeneca’s Tagrisso and Merck & Co’s Keytruda – both leading their respective drug classes in the treatment of non-small cell lung cancer (NSCLC).

The two phase 1/2 trials are zeroing in on NSCLC patients with specific molecular biomarkers, to see if adding Genprex’ Reqorsa (quaratusugene ozeplasmid) – which delivers a gene that suppresses tumour growth – can enhance the activity of the AZ and Merck drugs.

The first trial, called Acclaim-1, will pair EGFR inhibitor Tagrisso (osimertinib) with Reqorsa as a second-line treatment for EGFR-mutated NSCLC patients whose cancer has progressed after first-line Tagrisso treatment.

The Acclaim-2 trial meanwhile will add Reqorsa to PD-1 inhibitor Keytruda (pembrolizumab) in NSCLC patients with PD-L1 expression levels of 1% to 49%, according to the partners. Both studies are due to start in the first half of next year.

Tagrisso is the top-selling EGFR inhibitor, with sales of almost $3.2 billion last year, while Keytruda dominates the market for cancer immunotherapies for NSCLC, accounting for a large chunk of its $11 billion-plus 2019 sales tally.

If positive, the trials would allow Reqorsa to piggyback on that success – assuming it makes it to market. So far, Genprex only has data for the therapy from two phase 1 trials, and part of a phase 2 study showing preliminary evidence of safety as well as efficacy in NSCLC.

Genprex’ therapy takes the form of a copy of the TUSC2 gene in a non-viral lipid nanoparticle formulation, delivered via an intravenous infusion, and was originally developed at the University of Texas’ MD Anderson Cancer Centre in the US.

After infusion, the nanoparticles are taken up by tumour cells, says Genprex, and leads to the expression of the TUSC2 gene into a protein that reprograms them to die.

In preclinical studies, the gene therapy also seems to block mechanisms involved in drug resistance, including TIM3, a bypass pathway that can lead to failure of PD-1-based therapy.

The FDA awarded fast-track status to the Tagrisso/Reqorsa for the treatment of EHGFR-mutated NSCLC earlier this year, as nearly all patients receiving AZ’s drug eventually experience disease progression.

According the company’s data, the median time to progression after first-line Tagrisso is about 18 months.

Austin-based Genprex has been preparing for the Reqorsa clinical trials programme, over the last few months, ramping up its manufacturing capacity for the gene therapy via an agreement with contract manufacturing organisation (CMO) Aldevron.

The post Genprex cancer gene therapy paired with AZ, Merck lung cancer drugs appeared first on .

FDA Extends RCA With COTA to Explore COVID’s Effect on Cancer

What You Should Know:

– The FDA just extended its research collaboration
agreement (RCA) with COTA, specifically looking at how COVID-19 is affecting
cancer.

– With this expanded agreement, COTA and the FDA will use real-world data to explore the impact of COVID-19 and the pandemic on cancer treatment, with the opportunity to expand into other oncology questions in the future.


COTA, Inc., a healthcare technology
company that uses real-world data to bring clarity to cancer care, today
announced it has extended its Research Collaboration Agreement (RCA) with the
U.S. Food and Drug Administration (FDA) for an additional three years. This
renewed RCA will expand on the objective to explore the applications of
real-world data in oncology, including the impact of COVID-19 and the
pandemic on cancer treatment. As the project advances, the research will
broaden to study other oncology care delivery questions.

Real-world data can provide critical insights into the
delivery of cancer treatment in the routine practice setting, as well as
potential long-term effects post-COVID-19 recovery. The expanded focus of this
RCA will enable the exploration of important research questions to help support
FDA’s understanding of how the COVID-19 pandemic continues to impact patients
with cancer.

Why It Matters

With over 8 million cases of COVID-19 in the United States,
there is a significant need to understand the pandemic’s impact on oncology
care. Additionally, an increasing body
of research
 has shown that oncology patients may be particularly
susceptible to harm during this pandemic – both in contracting the
condition itself
 or experiencing care delays.

Cancer patients are particularly at risk of severe complications with COVID-19, and there is currently no understanding of how this can affect their oncology care or progression. Through real-world data, we can begin to understand if COVID-19 should be considered as comorbidity – particularly around clinical trial criteria.

Europe approves Roche’s Tecentriq liver cancer combination

The European Commission has approved Roche’s Tecentriq immunotherapy, in combination with its established cancer drug Avastin, for patients with the most common form of liver cancer.

Tecentriq (atezolizumab) can be used with Avastin (bevacizumab) for adults with advanced or unresectable hepatocellular carcinoma (HCC) who have not received systemic therapy.

Approval is based on findings of the phase 3 IMbrave 150 study, which showed an improvement in overall survival compared with Bayer’s Nexavar (sorafenib), which has long been the standard of care in liver cancer.

The study showed that Tecentriq in combination with Avastin reduced risk of death by 42% and reduced risk of disease worsening or death by 41% compared with Nexavar.

This result was the first time that a phase 3 cancer immunotherapy showed an improvement over Nexavar in both overall survival and progression-free survival.

Grade 3–4 adverse events occurred in 57% of people receiving Tecentriq and Avastin and 55% of people receiving sorafenib.

The most frequent serious adverse reactions for the combination, occurring in 2% or more of patients, were bleeding in the gastrointestinal tract and fever.

The decision follows approval of the combination therapy from the FDA in May, and from China’s National Medical Products Administration last month, although the latter decision only applied to untreated patients with unresectable disease.

Tecentriq is becoming increasingly important to Roche as sales of its “big three” cancer drugs Avastin, Herceptin, and Rituxan/MabThera fall away because of competition from cheaper biosimilars.

Sales of Tecentriq were up 64% in the first nine months of this year to just over 2 billion swiss francs ($2.2 billion), making it one of the company’s fastest growing products, and the fastest growing excluding recently launched products.

A PD-L1 class checkpoint inhibitor, Tecentriq works by calling in an attack from the patient’s immune system and since hitting the market in 2016 has already picked up US approvals in a range of oncology indications including lung and bladder cancer.

The big Swiss pharma is also developing it for several other types of lung, genitourinary, skin, breast, gynaecological and head and neck cancers.

The post Europe approves Roche’s Tecentriq liver cancer combination appeared first on .

Jounce shares crash after it abandons immunotherapy combination trial

Shares in Jounce Therapeutics are down sharply after it emerged that its immunotherapy combination with Bristol-Myers Squibb’s Yervoy will likely fail to produce results in non-small cell lung cancer.

Jounce was combining its vopratelimab with BMS’ already-approved Yervoy, hoping that the experimental drug will bolster the T-cell attack called in by the big pharma’s immunotherapy.

But an early look at the data from the phase 2 EMERGE trial showed that the combination is unlikely to work in a group of patients already treated with a PD-1 or PD-L1 immunotherapy such as Merck & Co’s Keytruda.

Results from the 22 patients treated with the highest dose of vopratelimab in combination with Yervoy showed only one patient with a confirmed overall response based on standard criteria, although tumours did seem to shrink in 12 patients.

There were no confirmed responses in two other arms of 18 and 10 patients respectively treated with a lower dose of vopratelimab in combination with Yervoy.

Only nine patients remained on the study, including four patients continuing to benefit from vopratelimab alone with an overall survival across all groups of 11.6 months across all groups.

The data were short of the criteria to expand the trial requiring tumour reduction in at least 50% of patients, overall survival above 13 months and an overall response rate of 10% or above.

As a result the EMERGE trial will not be expanded, the company said, although the first patient has been dosed in the SELECT phase 2 trial, supporting vopratelimab in combination with the company’s own JTX-4014 in certain NSCLC patients untreated with immunotherapy.

EMERGE was testing the hypothesis that vopratelimab could swell the numbers of ICOS hi CD4 cells that are called in by Yervoy to attack tumours.

Shares in Jounce were down more than 18% on the Nasdaq following the announcement.

Jounce does have other strings to its bow however – in September it announced a deal with Gilead to develop another immunotherapy called JTX-1811.

This targets T-cells that have been taken over by tumours and suppress attacks from the immune system, and could be worth more than $800 million if trials work out.

The post Jounce shares crash after it abandons immunotherapy combination trial appeared first on .

Sanofi to acquire Kiadis and NK cell tech for $353 million

Sanofi is to acquire Kiadis, a biotech specialising in therapies based around ‘off the shelf’ natural killer (NK) cells, for 308 million euros ($353 million).

The French pharma is buying Kiadis for 5.45 euros per share in cash, an offer price representing a premium of 272% over the biotech’s closing price on Friday evening on Amsterdam’s Euronext market.

It’s a substantial price to pay for the small pharma. Although the company’s share price has been notoriously volatile, the NK-cell technology is still largely unproven in clinic.

Allogeneic, or ‘off the shelf’, cell therapies are derived from banks of cells, and potentially offer the therapeutic benefits seen with autologous cell therapies derived from a patient’s own bodies but without the complex, costly and lengthy manufacturing process.

Autologous CAR-T cancer therapies based on T-cells are already on the market from Novartis and Gilead, but have had a slow start in terms of sales as health systems struggled to come to terms with their high price and the logistics of manufacturing them and delivering them to patients.

While Kiadis’ technology is mainly intended for cancer, the biotech announced plans in August to develop a NK-cell therapy for COVID-19, sending its shares soaring before they fell back due to profit-taking.

This followed a separate deal with Sanofi in July, where the pharma licensed Kiadis’ pre-clinical drug K-NK004 for multiple myeloma.

Kiadis has K-NK002 in phase 2 development to prevent patients with acute myeloid leukaemia (AML) and myelodysplastic syndromes from relapsing after transplant.

Also in phase 1 development is K-NK003, for patients with relapsed or refractory AML.

Kiadis is drawing up plans to begin a phase 1/2a clinical trial of KNK-ID-101, its COVID-19 therapy in high risk patients, with funding from the French government.

Acquiring Kiadis outright makes sense for Sanofi, which already has a foothold in cancer immunotherapy thanks to its Libtayo (cemiplimab), developed in partnership with Regeneron and already approved in a form of skin cancer.

Shareholders in Kiadis will likely be pleased with the substantial premium agreed and the deal has the unanimous support of its board.

Funds managed by Life Sciences Partners have committed to support the offer, accounting for 18.3% of shares in the Netherlands-based biotech.

The post Sanofi to acquire Kiadis and NK cell tech for $353 million appeared first on .

ANGLE raises £20m, eyes opportunity in immunotherapy trials

Liquid biopsy company ANGLE has raised £20m through a conditional placing on the London Stock Exchange which will be used to expand commercial opportunities.

ANGLE’s Parsortix system detects and captures circulating tumour cells (CTCs) in blood for subsequent analysis. The company believes there is huge potential for its use in assessing patient response to immunotherapy drugs.

“This raise enables us to set up two clinical laboratories for pharma services and processing patient samples. It also enables us to do some development of new assays, particularly for PD-L1 checkpoint inhibitors,” ANGLE CEO, Andrew Newland told pharmaphorum.

A study by the Laboratory of Translational Oncology, School of Medicine, University of Crete, demonstrated the system can predict patient response to immunotherapy. Results showed the detection of Indolamine-2,3-dioxygenase IDO and CTCs, particularly the IDO+/PD-L1- CTC subpopulation harvested using Parsortix, was significantly associated with reduced progression-free survival and overall survival in NSCLC patients treated with anti-PD-1 agents.

“The immunotherapy market is currently generating revenue of $22bn and growing at 40% per year. With thousands of active clinical trials focused on PDL checkpoint 1 inhibitors, we believe every single one of those trials could benefit from using Parsortix for CTC analysis and PDL1 expression,” Newland said. “This is an exciting opportunity as this market is not dependent on us receiving clearance.”

ANGLE submitted a full De Novo FDA application for Parsortix in September. The system could be the first medical device to receive FDA clearance for harvesting intact CTCs from the blood of metastatic breast cancer patients.

According to Newland, the £20m raise attracted more US investors as the appetite for liquid biopsy increases. “There is a consensus amongst US investment bankers that the liquid biopsy market is going to be over $100 billion a year per year in the US alone. We are seeing much more interest from US investors because they see us as a very undervalued opportunity in this space. They see we have solutions that can expand what liquid biopsy companies are doing and we don’t have to compete with them.”

The liquid biopsy space has exploded this year as companies signed billion-dollar deals to strengthen their market positions. Illumina announced a $8 billion agreement to reacquire former spinout Grail and Exact Sciences entered a $2.15 billion deal to buy Thrive.

The post ANGLE raises £20m, eyes opportunity in immunotherapy trials appeared first on .

FDA sets Feb decision date for Libtayo lung cancer challenge to Keytruda

The FDA has started a priority review of Regeneron and Sanofi’s checkpoint inhibitor Libtayo in first-line non-small cell lung cancer (NSCLC), based on data they hope will carve out a niche for the drug in a highly-contested market.

PD-1 inhibitor Libtayo (cemiplimab) impressed oncologists earlier this year with results from a trial in previously-untreated NSCLC patients whose tumours had high levels of the PD-L1 biomarker, showing the drug reduced the risk of death by 32% compared to platinum chemotherapy.

Updated results from the study reported at the ESMO congress in September showed that among patients with confirmed PD-L1 expression of 50% or more – the patient group targeted in Sanofi and Regeneron’s marketing application – the mortality rate was reduced by 43%.

If the FDA cleared Libtayo by its deadline of 28 February, the drug will join the clutch of checkpoint inhibitors vying for a slice of the first-line NLSCLC market, nipping at the heels of Merck & Co/MSD’s mega-blockbuster Keytruda (pembrolizumab), the undisputed class leader in this form of lung cancer.

Libtayo is also under review by the EMA as a treatment for treatment-naïve NSCLC patients with PD-L1 expression of at least 50%, with a decision expected in the second quarter of 2021, according to Sanofi.

Libtayo was the sixth PD-L/PD-L1 inhibitor to reach the market, starting out as a therapy for an uncommon skin cancer called cutaneous squamous cell carcinoma (CSCC), but Sanofi and Regeneron are seeking to expand its use into first-line NSCLC as well as basal cell carcinoma (BCC), another form of skin cancer.

At the moment, the annual net sales of these drugs across all indications are around $25 billion, but about half of that comes from NSCLC, which means even a minor share of that market would be a big step up for Libtayo. Regeneron reported sales of the drug were around $155 million in the first half of 2020.

Keytruda is used mainly in combination with chemo in first-line NSCLC, based on  trial data showing a 51% reduction in death versus chemo alone – although it is also approved as a monotherapy – and has the advantage that it can be given to patients regardless of their PD-L1 status.

Sanofi and Regeneron are hoping that the impressive mortality data from their trial will allow Libtayo to vie for selection as treatment for higher PD-L1 expressors, but other rivals are also in the mix.

Bristol-Myers Squibb’s combination of PD-1 inhibitor Opdivo (nivolumab) and CTLA4 inhibitor Yervoy (ipilimumab) has now been approved by the FDA for two front-line NSCLC indications, both in May.

It was cleared as a monotherapy for patients with PD-L1 levels of 1% or more, and with “limited” chemo in all patients regardless of PD-L1 status, provided they have no EGFR or ALK mutations, which can be treated with targeted drugs.

Meanwhile, Roche’s PD-L1 drug Tecentriq (atezolizumab) has also picked up two approvals from the US regulator, one for the drug on its own in patients whose tumours express PD-L1, and a second alongside chemo in ‘all-comer’ patients with no EGFR or ALK mutations.

The post FDA sets Feb decision date for Libtayo lung cancer challenge to Keytruda appeared first on .

Northwestern to Deploy FDA-Cleared Deploy AI-Guided Cardiac Ultrasounds

Northwestern to Deploy FDA-Cleared Deploy AI-Guided Cardiac Ultrasounds

What You Should Know:

– Northwestern Memorial Hospital is the first in the
nation to deploy FDA-cleared AI-guided ultrasound by Caption Health, including
measurement of ejection fraction – the most widely used measurement to assess
cardiac function.

– Caption Health’s AI-guided cardiac ultrasound enables clinicians – including those without experience – to accurately perform diagnostic-quality exams — accelerating the availability of information and saving lives.

– Caption AI has been shown to produce assessments
similar to those of experienced sonographers in work presented to the American
Society of Anesthesiologists.


Northwestern Memorial
Hospital
is the first hospital in the United States to purchase Caption Health’s
artificial
intelligence (AI)
technology for ultrasound, Caption AI. The FDA cleared, AI-guided
ultrasound system enables healthcare providers to acquire and interpret quality
ultrasound images of the human heart, increasing access to timely and accurate
cardiac assessments at the point of care.

Performing an ultrasound exam is a complex skill that takes years to master. Caption AI enables clinicians—including those without prior ultrasound experience—to quickly and accurately perform diagnostic-quality ultrasound exams by providing expert turn-by-turn guidance, automated quality assessment, and intelligent interpretation capabilities. The systems are currently in the hospital’s emergency department, medical intensive care unit, cardio-oncology clinic, and in use by the hospital medicine group.

Democratize the Echocardiogram

Point-of-care ultrasound (POCUS) has a number of benefits. Increased usage of POCUS contributes to more timely and accurate diagnoses, more accurate monitoring, and has been shown to lead to changes in patient management in 47% of cases for critically ill patients. POCUS also allows patients to avoid additional visits to receive imaging, as well as providing real-time results that can be recorded into a patient’s electronic medical record.

“Through our partnership with Caption Health, we are looking to democratize the echocardiogram, a stalwart tool in the diagnosis and treatment of heart disease,” said Patrick McCarthy, MD, chief of cardiac surgery and executive director of the Northwestern Medicine Bluhm Cardiovascular Institute, a group involved in the early development of the technology. “Our ultimate goal is to improve cardiovascular health wherever we need to, and Caption AI is increasing access throughout the hospital to quality diagnostic images.” 

How Caption Health Works

Caption AI emulates the expertise of a sonographer by providing real-time guidance on how to position and manipulate the transducer, or ultrasound wand, on a patient’s body. The software shows clinicians in real-time how close they are to acquiring a quality ultrasound image, and automatically records the image when it reaches the diagnostic-quality threshold. Caption AI also automatically calculates ejection fraction, or the percentage of blood leaving the heart when it contracts, which is the most widely used measurement to assess cardiac function.

Northwestern Medicine has been a tremendous partner in helping us develop and validate Caption AI. We are thrilled that they are bringing Caption AI into key clinical settings as our first customer,” said Charles Cadieu, chief executive officer and co-founder of Caption Health. “The clinical, economic and operational advantages of using AI-guided ultrasound are clear. Most important, this solution increases access to a safe and effective diagnostic tool that can be life-saving for patients.”

Exact Sciences buys cancer detection company Thrive for $2.15 billion

Stocks for Exact Sciences leaped 33% after it announced the acquisition of liquid biopsy rival Thrive and UK start-up Base Genomics.

Exact Sciences said it has agreed to buy Thrive for a cash and stock deal worth up to $2.15 billion, positioning it as a leader in the cancer-screening market.

The Madison-based company has made no secret of its ambitions to become the top player in the liquid biopsy space.

Thrive’s technology has attracted excitement from investors since it spun out of Johns Hopkins University. In July 2020, the company closed a $257m series B funding round, led by Casdin Capital and Section 32.

The CancerSEEK blood test is designed to detect multiple cancers by analysing tumour specific genomic mutations in circulating tumor DNA (ctDNA) and cancer-associated protein biomarkers in plasma. These are then analysed by machine learning algorithms.

The company’s DETECT-A study was the first-ever prospective, interventional study of a multi-cancer blood test. Key findings from the study showed CancerSEEK had a specificity of 99.6% and identified cancer in individuals without a history of the disease. The study was hailed by Thrive as a “seminal moment” in cancer screening.

Exact Sciences, which is behind brands Cologuard and Oncotype DX, said it will combine CancerSEEK with its scientific platform, clinical organization, and commercial infrastructure. The company also announced the acquisition of Base Genomics, a UK epigenetics start-up for $410m. The start-up uses DNA methylation analysis to detect cancer in its early stages.

Exact Sciences Cologuard business also received a boost from updated guidelines released by the United States Preventive Services Task Force. The draft recommended colorectal cancer screening should now begin at age 45 and recommends Exact Sciences’ Cologuard as a screening method for all average-risk patients between the ages of 45 and 75.

The post Exact Sciences buys cancer detection company Thrive for $2.15 billion appeared first on .

Daiichi Sankyo Initiates P-I Study of DS-1055 for Relapsed/Refractory Advanced or Metastatic Solid Tumors

Shots:

  • The P-I dose-escalation study will assess the safety, tolerability, and preliminary efficacy of DS-1055 in adult patients with r/r advanced or metastatic head and neck, gastric and esophageal cancers, and other tumor types
  • The focus of the study is to determine the maximum tolerated dose and recommended dose of DS-1055 for further study and is expected to enroll ~40 patients across the US and Japan. The company will integrate novel technologies with expertise in Ab biology to develop clinical candidates
  • DS-1055 is a mAb designed to target GARP and to promote antitumor immunity through the depletion of GARP positive Tregs

Click here ­to­ read full press release/ article | Ref: Daiichi Sankyo | Image: Medical Dialogues

The post Daiichi Sankyo Initiates P-I Study of DS-1055 for Relapsed/Refractory Advanced or Metastatic Solid Tumors first appeared on PharmaShots.

NICE changes stance on Keytruda for first-line head and neck cancer

As recently as June, NICE was minded not to back routine NHS of MSD’s Keytruda as a first-line treatment for advanced head and neck cancer, but it has had a partial change of heart on the drug after the company submitted new data.

Just-published draft final guidance from the cost-effectiveness agency gives a green light for NHS use of Keytruda (pembrolizumab) as a monotherapy for adults with untreated metastatic or unresectable recurrent head and neck squamous cell carcinoma (HNSCC) whose tumours express the biomarker PD-L1.

The decision makes Keytruda the first checkpoint inhibitor to be recommended for this use by NICE, according to MSD, which is known as Merck & Co in North America.

NICE hasn’t backed use of the checkpoint inhibitor in combination with chemotherapy however, a decision that the Institute of Cancer Research (ICR) says is disappointing as its experts think there are patients who would benefit more from the combination rather than Keytruda on its own.

The verdict is also at odds with that of the Scottish Medicines Consortium (SMC), which backed both monotherapy and combination use of Keytruda in this setting last month.

ICR would like to see the Keytruda combination available via the Cancer Drugs Fund (CDF), which covers the cost of cancer drugs until confirmatory data is available, for a period of two years in order to allow new evidence to be collected.

NICE however says that while it can support use of Keytruda on its own as the drug is more effective than standard treatments, the cost of using it in combination with platinum chemotherapy and 5-FU “are higher than NICE normally considers an acceptable use of NHS resources”.

ICR’s position is that the decision highlights a major problem with cancer immunotherapies, namely a lack of good tests to determine who will benefit from them.

In this case, biomarker tests like PD-L1 alone “fail to give clinicians a clear-cut indication of who will benefit from immunotherapy – and, critically, who requires pembrolizumab alone and who needs the combination of pembrolizumab and chemotherapy,” it points out.

PD-L1 testing is a “good starting point”, according to ICR’s experts, but “does not allow for the more nuanced approach that is permitted in Scotland and in much of the rest of the world”.

“The partial approval leaves patients in England behind much of the world when it comes to accessing this game-changing treatment,” commented Prof Kevin Harrington of The Royal Marsden, who led the UK arm of the KEYNOTE-048 trial that supported approval of Keytruda in first-line HNSCC.

“The evidence for the benefit of pembrolizumab in combination with chemotherapy in recurrent head and neck cancer is clear – and I would urge NICE and the manufacturer to work together to find a way for patients to access the range of treatment options they deserve,” he added.

The post NICE changes stance on Keytruda for first-line head and neck cancer appeared first on .

Toxic positivity and grief: The reality of living through cancer

Cancer doesn’t end when treatment stops – it’s a lifelong journey and people need support throughout, says patient advocate Megan-Claire Chase.

No one gets through cancer unscathed. People are usually left with a mixture of treatment aftereffects, toxic positivity, and lingering anger to deal with.

Megan-Claire Chase, a fierce patient advocate who has been blogging about her experiences since she was diagnosed with breast cancer in 2015, said the disease never went away.

“Sometimes, people feel that once you finish your treatment, you are done, and you should stop talking about it. But really, it has only just begun. It is a lifelong journey.

“We’re going to need to have scans forever and we’ve been traumatised by the whole experience, so it’s hurtful when your family members and your friends think you should just move past it.”

Megan-Claire underwent 16 rounds of chemotherapy, nine operations, and 33 radiation treatments after developing invasive lobular breast cancer in her  30s.

As a result of the treatment, she is infertile, has chemo-induced fibromyalgia and neuropathy in her hands and feet. She goes to the cancer center for a diagnostic mammogram and rotates between getting an MRI and ultrasound every six months for 10 years.

“You know that people have good intentions and they are coming from a good place, but when your family and friends just want to see you smiling all the time, it can feel like you’re putting on an act”

Quality of life after cancer

There are a lot of quality of life issues, many of which were not understood outside of the cancer community, says Megan-Claire.

She says: “Sometimes I have to walk with a cane. Because of the neuropathy on my feet, I can’t walk very long distances because the numbness goes up my leg and I fall over and hurt myself.

“That’s what I want people to understand. Some people are on active treatment forever. For others, that treatment has a stopping point, but even then, there are all these other issues that can stem from the poison that goes into your body.”

News stories about cancer survivors going on to achieve great feats of endurance do nothing to help the public perception of what it means to live with the long-term effects of the disease and its treatments.

“Some people are able to climb mountains and do walks and all of that physical stuff. But for others that ability was taken away and it feels like yet another loss,” says Megan-Claire:

“We’re constantly grieving – grieving the loss of the body parts that are missing, the physical abilities we had, and, for many of us, grieving the jobs we used to have before we had to stop working.

“We also have to grieve the friends we have made and lost along the way. We’re constantly reminded of our mortality and have to live with the fear that it could come back at any time.”

Releasing the negative

Through her blog, Life on the Cancer Train, and her advocacy work, Megan-Claire meets many young people in a similar position to her. She believes it helps to give them the space to own their feelings and share their experiences with others who truly understand the nuances of life after cancer.

“When I write, I write what is on my mind, but I always find that resonates with people. A lot of people are hurting, and I think it helps that I post about the elephants in the room: the mental health issues, the anger, the post-traumatic stress disorder,” says Megan-Claire. “People need a place to release all that.”

‘Toxic positivity’ is another issue people who have been through cancer treatment commonly face and dealing with it head on can be extremely difficult, Megan-Claire explains.

“It’s impossible to be this tower of strength all the time. A lot of us get annoyed when we are told: ‘Oh, you’re so brave’, or ‘I don’t know how you do it’. When that happens, all I’m thinking is ‘Do I want to live or do I want to die. It’s not a fair choice.’

“You know that people have good intentions and they are coming from a good place, but when your family and friends just want to see you smiling all the time, it can feel like you’re putting on an act.”

Peer support

What people need, she went on, was a place where they could “take off the mask and just be vulnerable”. And that’s where peer support comes in.

“Cancer support groups are a safe place where people can meet and talk about their experiences with no judgement.“” says Megan-Claire, who belongs to several young adult cancer groups online.

“My advice to people is always: if you’re angry, be angry. If you’re sad, be sad. Just don’t wallow in it forever. We need to feel these emotions so we can move through them and then do what it is we need to do to move forward with our lives.”

To read Megan-Claire’s blog, click here. See our last interview with Megan-Claire here.


inspirePatient Insights is a monthly series that appears in partnership with Inspire, a company with an online support community of more than 1.5 million patients and caregivers worldwide.

The post Toxic positivity and grief: The reality of living through cancer appeared first on .

David Hung’s Nuvation Bio seals merger with blank cheque firm Panacea

Two years after quietly forming cancer start-up Nuvation Bio, ex-Axovant and Medivation CEO David Hung has agreed a big merger that will take it public – and provide more than $850 million in funding to boot.

Hung’s new company is merging with Panacea Acquisition Corp, a special purpose acquisition company or SPAC – sometimes referred to as a ‘blank cheque’ company – tapping into an emerging trend among biotech companies.

SPACs are set up with investor funding – generally aiming to bolt-on an R&D pipeline via M&A – and can provide a quicker route to a public market debut than the more common initial public offering (IPO).

The SPAC goes public itself, ahead of any acquisition or merger, and Panacea went down that route with a $150 million IPO earlier this year, along with a pledge to find suitable merger candidates in the oncology field.

That IPO adds to a sizeable $275 million first-round financing for Nuvation last year, while institutional investors led by EcoR1 Capital have committed $500 million to the company through concurrent equity investments associated with the Panacea merger.

That big war chest will be available to advance Nuvation’s pipeline of six-wholly-owned experimental cancer therapies that all scheduled to start clinical trials between now and 2026, headed by a drug for patients with high-grade gliomas, an aggressive form of brain cancer.

The deal catapults Hung back into the spotlight after a turbulent few years. He led Medivation to a big $14 billion takeover by Pfizer in 2016, and then joined Roivant group company Axovant, where things didn’t go so well.

Hung left Axovant in 2018, shortly after the biotech’s lead candidate for Alzheimer’s disease flunked a phase 3 programme and was abandoned, and founded Nuvation later that year to develop “novel oncology therapeutic agents for the most difficult-to-treat cancers, specifically targeting indications for which conventional therapies have failed.”

The first of these, CDK 2/4/6 inhibitor NUV-422, is due to start a phase 1/2 trial in gliomas, including a form called glioblastoma multiforme (GBM) with very few treatment options, in the first quarter of 2021. That’s also around the time the Panacea merger is expected to close.

There are also drugs targeting BET, WEE1 and A2A in preclinical development for leukaemia and solid tumours, and a series of PARP-targeting drugs for prostate, breast and ovarian cancer.

“We have demonstrated, in preclinical studies, the potential of those candidates to significantly improve outcomes over current standards of care,” said Hung, who will retain his position as CEO of the company after the merger closes.

Hung, together with the team of chemists from Medivation, invented all of the programs in Nuvation’s current pipeline, according to the company.

Panacea CEO Oleg Nodelman said the SPAC was set up to partner with a company that had “an exceptional management team, a deep pipeline, and a platform technology that could enable success to be replicated over and over,” adding: “that is exactly what we saw in Nuvation.”

The post David Hung’s Nuvation Bio seals merger with blank cheque firm Panacea appeared first on .

FDA grants fast review for AstraZeneca’s Tagrisso in early-stage lung cancer

The FDA has granted a fast review for AstraZeneca’s Tagrisso oncology drug in certain patients with early-stage lung cancer.

Results from the phase 3 ADAURA trial were the talk of this year’s American Society of Clinical Oncology (ASCO) conference in summer and the regulator has granted a Priority Review for a label extension based on the results.

The FDA reserves these faster reviews, taking six months at the most instead of the standard ten-month timeframe, for medicines that demonstrate superior efficacy or safety for serious diseases.

ADAURA tested Tagrisso (osimertinib) in patients with early-stage (1B, II and III) epidermal growth factor receptor-mutated (EGFR) non-small cell lung cancer (NSCLC) after complete tumour removal.

Up to 30% of all patients diagnosed early with NSCLC in this patient group may be diagnosed early enough to have curative surgery, but disease recurrence is common and more than three quarters of patients diagnosed in Stage IIIA experience recurrence within five years.

ADAURA was halted early this year after Tagrisso showed “overwhelming efficacy” in this patient group.

In the trial Tagrisso reduced the risk of disease recurrence or death by 83% compared to placebo in patients with tumours that had spread locally but not to other parts of the body (stage II-IIIa), and who had surgery with the aim of completely removing the tumour and curing their cancer.

The drug also reduced disease-free survival by 79% in the overall trial population (stage 1b to IIIa), and after two years 89% of patients remained alive, compared to 53% of the placebo group.

It’s the first time that a targeted drug has shown an improvement in this group of patients in a large scale trial.

Approval will be another string in the bow for Tagrisso, which is already a blockbuster bringing in revenues of more than $1 billion per quarter for the UK pharma.

Tagrisso is a third-generation irreversible EGFR-tyrosine kinase inhibitor that was originally intended to treat patients with EGFR-mutated NSCLC after an older drug from that class has been rendered useless by a T790M mutation, which usually occurs after around 18 months of treatment.

But since this first FDA approval in 2015 it has also been licensed in first-line treatment in EGFR-mutated lung cancer, after outperforming AZ’s older EGFR drug Iressa (gefitinib) and Roche’s rival Tarceva (erlotinib) in the phase 3 FLAURA trial.

 

The post FDA grants fast review for AstraZeneca’s Tagrisso in early-stage lung cancer appeared first on .

FDA kicks off review of second kidney cancer combo based on BMS’ Opdivo

Bristol-Myers Squibb could be mere months away from claiming a second US approval for an Opdivo-based combination immunotherapy that will help defend its first-line kidney cancer franchise.

The FDA has started a priority review of PD-1 inhibitor Opdivo (nivolumab) alongside Exelixis’ tyrosine kinase inhibitor Cabometyx (cabozantinib) in previously-untreated advanced renal cell carcinoma (RCC) – the most common form of kidney cancer – and has set a date of 20 February to deliver a verdict on the marketing application.

If approved, the Opdivo/Cabometyx regimen would help BMS compete with two other combination therapies – Merck & Co’s Keytruda (pembrolizumab) and Pfizer’s Inlyta (axitinib), and Merck KGaA/Pfizer’s Bavencio (avelumab) plus Inlyta – which were approved by the FDA for first-line RCC treatment last year.

BMS was first to a first-line RCC approval, after getting a green light from the FDA for Opdivo alongside its CTLA4 inhibitor Yervoy (ipilimumab) in 2018.

The new combination would be an opportunity for BMS to shore up its position in this type of cancer, which is thought to have underpinned a lot of its recent sales growth, as the competition gathers strength.

Keytruda has already overtaken Opdivo in sales terms thanks to its dominant position in non-small cell lung cancer (NSCLC), and RCC has been an important therapeutic category for BMS as it tries to maintain its share of the immuno-oncology market.

The FDA is reviewing Opdivo/Cabometyx based on the results of the CheckMate-9ER trial, which was revealed to some fanfare at the virtual ESMO congress last month. In the study, the combination reduced the overall risk of death by 40% compared to Pfizer’s older drug Sutent (sunitinib), a standard first-line therapy for RCC.

That benefit applied to all patients, not just those with PD-L1-positive tumours who typically respond best to checkpoint inhibitors, and the duo also doubled the median progression-free survival (PFS) to 16.6 months versus 8.3 months for the Sutent group.

According to Exelixis, Cabometyx – a small-molecule inhibitor of RET, MET and VEGFR2 tyrosine kinases – creates a “more immune-permissive tumour environment that may allow for synergistic antitumor activity when combined with Opdivo.”

Approval alongside Opdivo in RCC would also be a big boost to Exelixis which currently makes the bulk of its Cabometyx sales from use of the drug as a second-line treatment for RCC patients previously treated with Sutent or other drugs like Bayer’s Nexavar (sorafenib) or Roche’s Avastin (bevacizumab).

When the CheckMate-9ER data was reported at ESMO, lead investigator Toni Choueiri of the Dana-Farber Cancer Institute and Harvard University said that tolerability could be a key differentiator for Opdivo/Cabometyx could be a better side-effect profile than its rivals.

“The various combination treatments will unlikely be compared head-to-head, but I think quality of life could differentiate this new therapy,” he suggested.

The post FDA kicks off review of second kidney cancer combo based on BMS’ Opdivo appeared first on .

Impact of COVID-19: 2020 State of Healthcare Performance Improvement Report

Impact of COVID-19: 2020 State of Healthcare Performance Improvement Report

What You Should Know:

– Nearly three-quarters of hospital leaders are either
moderately (52%) or extremely (22%) concerned about the financial viability of
their organizations without an effective treatment or vaccine for COVID-19,
according to a new report from Kaufman Hall entitled, “2020 State of Healthcare
Performance Improvement Report: The Impact of COVID-19”

– One-third of respondents saw operating margin declines
in excess of 100% in the second quarter of 2020 compared with the same period
of 2019.

– This year’s report findings were based on 64 responses
to a survey that Kaufman Hall fielded in August 2020.


Nearly three-quarters
of hospital leaders are either moderately (52%) or extremely (22%) concerned
about the financial viability of their organizations without an effective
treatment or vaccine for COVID-19, according to a new report from Kaufman Hall. One-third of respondents
(33%) saw operating margin declines in excess of 100% in the second quarter of
2020 compared with the same period of 2019.

2020 State of Healthcare Performance Improvement Report: The
Impact of COVID-19
” is Kaufman Hall’s fourth annual survey of hospitals
and health systems on their performance improvement and cost transformation
efforts.

“The challenges brought on by the COVID-19 pandemic have
affected nearly every aspect of hospital financial and clinical
operations,” said Lance Robinson, managing director, Kaufman Hall. “Organizations have
responded to the challenge by adjusting their operations and strengthening
important community relationships.”

Key findings from this
year’s report include the following:

Financial viabilityApproximately three fourths of survey respondents are either extremely (22%) or moderately (52%) concerned about the financial viability of their organization in the absence of an effective vaccine or treatment.

Operating margins. One-third of our respondents saw year-over-year operating margin declines in excess of 100% from Q2 2019 to Q2 2020.

Volumes. Volumes in most service areas are recovering slowly. In only one area—oncology—have a majority of our respondents seen volumes return to more than 90% of pre-pandemic levels.

Expenses. A majority of survey respondents have seen their greatest percentage expense increase in the costs of supplying personal protective equipment. Nursing staff labor is in second place, cited by 34% of respondents as their most significant area of expense increase.

Healthcare workforce. Three-fourths of survey respondents have increased monitoring and resources to address staff burnout and mental health concerns.

Telehealth. More than half of our respondents have seen the number of telehealth visits at their organization increase by more than 100% since the pandemic began. Payment disparities between telehealth and in-person visits are seen as the greatest obstacle to more widespread adoption of telehealth.

Competition. Approximately one-third of survey respondents believe the pandemic has affected competitive dynamics in their market by making consumers more likely to seek care at retail-based clinics.

To download a copy of the report, click on the download
now button below

Kite cues up first EMA okay for mantle cell lymphoma CAR-T

Gilead Sciences’ Kite Pharma unit is closing on approval of its second European approval for a CAR-T for cancer, after the CHMP backed its Tecartus therapy for mantle cell lymphoma.

The EMA’s human drugs advisory committee recommended approval of Tecartus (brexucabtagene autoleucel; formerly KTE-X19) for relapsed or refractory MCL setting up a formal approval by the regulator in the coming weeks.

Tecartus is the third CAR-T therapy to be recommended for approval in Europe and will be the first for MCL, an aggressive form of non-Hodgkin’s lymphoma (NHL).

MCL is generally treated first with chemotherapy, and patients whose disease progresses despite that can then receive stem cell transplantation or BTK inhibitor drugs like Johnson & Johnson/AbbVie’s Imbruvica (ibrutinib) and AstraZeneca’s Calquence (acalabrutinib).

Despite these therapies, relapsed/refractory MCL patients often relapse or stop responding to treatment, according to the EMA.

Tecartus was cleared by the US FDA in the summer, and was the first CAR-T from Kite to launch without direct competition, as its first therapy Yescarta (axicabtagene ciloleucel) is going head-to-head in the market with Novartis’ rival Kymriah (tisagenlecleucel).

Gilead will be hoping that Tecartus will inject some additional momentum into its CAR-T franchise, which underpinned its $11.9 billion acquisition of Kite in 2017.

So far, Yescarta – which is approved for other forms of NHL – has performed well below its blockbuster sales projection when it launched three years ago, bringing in $296 million in the first half of this year. Kymriah hasn’t delivered as hoped for Novartis either, which has been attributed to manufacturing problems.

Yescarta’s lacklustre performance caused Gilead to take an $800 million charge in the fourth quarter of 2019, and that came after an $820 million write-down a year earlier as the company downgraded the value of Yescarta and Kite’s CAR-T pipeline.

Tecartus has been recommended for approval in the EU on the strength of the ZUMA-2 trial, which involved 74 adult MCL patients who had previously been treated with at least two prior therapies, including BTK drugs.

Over 12 months of follow-up, 84% of patients treated with Tecartus responded to treatment, with 59% showing a complete response, a level of efficacy that goes further even than the impressive results achieved by Yescarta.

Tecartus employs the same T-cell construct as the previously approved CAR-T Yescarta but has a slightly modified manufacturing process to boost its activity in diseases where there is a large burden of circulating tumour cells.

The EMA designated Tecartus a Priority Medicine (PRIME), allowing for an expedited review.

The CAR-T is also being tested in acute lymphocytic lymphoma (ALL), and if approved that will place the CAR-T in competition with Kymriah, which has been approved to treat ALL since 2017.

The post Kite cues up first EMA okay for mantle cell lymphoma CAR-T appeared first on .

Thriving in a Value-Based Care Environment: Impacting Outcome-to-Cost Ratio

Thriving in a value-based care environment: impacting the outcome-to-cost ratio
Jerry Carlson, Product Support Manager, BG DI BU IC Sales, Dunlee

As the COVID-19 pandemic creates surges in acute care, many imaging departments are experiencing a decrease in volume, due to patients deferring or canceling non-urgent appointments and surgeries. The impact of this makes it painfully obvious that — because imaging departments rely on a fee-for-service model – when the volume is down, finances suffer. As an aspect of healthcare that has historically been hyper-focused on volume, adoption of a value-based care approach in radiology has evolved slowly, even before the pandemic. Despite the hurdles COVID-19 has presented, the rationale behind value-based care remains – there is a need to drive improved patient outcomes at a lower cost – and healthcare reimbursement will continue to shift, encouraging quality care and enhanced patient experiences. Radiology can take an important role in realizing this transformation, influencing the entire process of early diagnosis, efficient treatment, and follow-up care.

So how can imaging departments thrive when confronted with a value-based care model? One way is to make sure referrers value radiologists’ expertise as part of the care team. Active participation in care team discussions, as well as case study presentations, can demonstrate the extent to which imaging affects outcomes. Imaging departments can also invest in building referrals for areas where imaging intersects more directly with care, such as oncology. But perhaps the most direct way is to focus on an area over which the imaging department has the most control: the cost-effective use of resources. The strategies chosen today could help or hurt a practice in the future, and departments should look toward reliable technology that delivers consistent results and allows staff to focus less on technical issues and more on patient care.

An efficient department with the right mix of technology can thrive in a value-based healthcare environment. Answering these three questions can guide your technology strategy and help you weather the pandemic disruption and the continuing adoption of value-based care:

Are your imaging systems appropriate for your patient demand? 

As it relates to value-based care, you can expect the future to entail less “confirmation” imaging and more investigational, prevention-focused imaging. However, different imaging solutions have different purposes; while confirmation CT studies don’t demand as high performance, investigational imaging will often require more sophisticated systems with the image quality and performance to support complex studies and confident diagnoses, and potentially even spot incidental findings that circumvent health issues down the line. When purchasing new systems, consider the type of studies that make up the majority of your business, as well as new areas in which you’d like to increase expertise and referrals.

How cost-effective are your imaging technology operations? 

Consider every aspect of your operation to uncover opportunities to decrease costs without affecting quality. For best results, involve the entire imaging department team in these explorations. One possible budget drain is consumables. Sometimes the easiest way to service your car is by bringing it to the dealer, but that’s not always the most cost-effective option. The same goes for imaging technology; be sure to consider third-party options, in addition to Original Equipment Manufacturer (OEM) parts. Today, alternate parts are available for almost every piece of your organization, even technically sophisticated components such as x-ray tubes.

Is your technology reliable? 

Speed to diagnosis may impact patient outcomes.  Your referrers are looking for a quick turnaround of imaging studies. Highly advanced technology with reliable uptime can help you become a partner of choice, and reduce time spent maintaining equipment. For example, radiation oncology depends on CT for treatment planning, and oncologists need radiology partners who have CT systems that are dependable, integrate easily into their workflow, and do not distract from patient care. Even a small change, such as CT tubes that use highly reliable liquid metal bearings to eliminate the need to wait for tube cooling between studies, will impact your throughput and thus your ability to meet referrers’ needs. 

Are you putting unnecessary stress on your imaging systems? 

Educate all system users about manufacturer-recommended procedures for system use and upkeep to keep your systems running at high performance. For example, shutting the system down by turning off the power, rather than by following manufacturer-recommended procedures, places unnecessary stress on components that need time to cool.

While it’s important to take a measured approach as we navigate the repercussions of COVID-19, now is the time to begin adapting to value-based care. As the pandemic has taught us, a nimble imaging department can adapt to changing circumstances and create lasting value. Revisit these questions frequently, because consistent assessment and vigilance is key to a department’s success.

J+D FORECASTING LAUNCHES INTELLIGENT DATA

J+D Forecasting, known as the experts in pharmaceutical forecasting, has today announced the launch of EpiCube; the latest addition to its comprehensive suite of pharmaceutical forecasting products.

Unlike most standard epidemiological databases available, EpiCube is the first database where the user can choose how to build their own data set; be that by country, disease, or a specific attribute such as a bio marker.

Using Microsoft’s latest technology, data can be interrogated, analysed, and shared. Underpinned by J+D’s forecasting expertise, EpiCube aligns to pharmaceutical forecasting structures and helps pharmaceutical companies explore risk factors and underlying causes affecting the size of the patient pool for their new and existing products.

EpiCube is an epidemiology database for multiple therapy areas, including Oncology, multiple diseases, attributes, countries, age groups and genders. There are over 9,500 sub-groups and over 200 diseases across 50 countries. Click here for more information: https://jdforecasting.com/software/epicube/

David James, CEO, J+D Forecasting:

“We have developed EpiCube to help clients understand diseases better, so they have more time to think. To think about the forecast, to look at the assumptions, to test hypotheses better and to spend the time creating a good forecast.

We want to help pharmaceutical companies create maximum shareable insights in their forecasting approach, speed it up, visualise insights instantly and focus on what really matters.”

The post J+D FORECASTING LAUNCHES INTELLIGENT DATA appeared first on .

Shock as FDA rejects Mesoblast’s Ryoncil in paediatric graft versus host disease

The FDA has rejected Mesoblast’s Ryoncil cell therapy for children with graft versus host disease, despite a recommendation to approve from a panel of expert advisers.

In August the FDA’s Oncology Drugs Advisory Committee voted nine to one in favour of approving Ryoncil (remestemcel-L), making approval likely as the regulator usually follows the advice of such committees.

But the FDA does not have to follow the advice of its experts, and in this case it hasn’t.

The FDA issued a dreaded Complete Response Letter rejecting the therapy intended for paediatric steroid-refractory acute graft versus host disease.

In the letter the FDA asked for at least one more randomised controlled study in adults and/or children to provide further evidence of efficacy, prompting trading in the company to be halted on the Australian Securities Exchange.

Shares in the Melbourne-based firm lost more than a third of their value on the Nasdaq, falling to around $12 following the announcement that also weighed on other biotech stocks on Friday.

In the absence of any approved treatment for the potentially life-threatening condition in children aged under 12, Mesoblast has asked to meet with the FDA within 30 days to discuss an accelerated approval based on early data.

The company thinks it could ask for the approval pending confirmatory data from a late-stage trial.

The FDA needs further information showing the relationship of potency measurements to the product’s biologic activity.

Mesoblast is also testing remestemcel-L in a phase 3 trial in up to 300 ventilatory-dependent adults with moderate to severe acute respiratory distress (ARDS) due to COVID-19.

A second interim analysis by the trial’s independent Data Safety Monitoring Board is expected in early November, with completion of patient recruitment expected in December.

Like graft versus host disease, ARDS is caused by over-stimulation of inflammatory cytokines, which causes the immune system to attack the patient’s own body.

Graft versus host disease is caused when the body rejects stem cells that are transplanted to treat diseases such as leukaemia.

The post Shock as FDA rejects Mesoblast’s Ryoncil in paediatric graft versus host disease appeared first on .

FDA approves BMS’ Opdivo and Yervoy combination in first-line mesothelioma

Bristol-Myers Squibb’s immunotherapy combination of Opdivo and Yervoy has been approved in the US in a new indication for untreated mesothelioma, a rare but aggressive form of cancer that forms in the lining of the lungs.

The FDA has approved Opdivo (nivolumab) and Yervoy (ipilimumab), as the first and only immunotherapy for previously untreated unresectable malignant pleural mesothelioma.

While there are several immunotherapies in development for mesothelioma the Opdivo and Yervoy combination is the first to win FDA approval in this stage of the disease, based on findings of the CheckMate-743 trial.

In a pre-planned interim analysis the 303-patient trial showed the combination demonstrated superior overall survival versus the platinum-based standard of care chemotherapy.

After a follow-up period of 22.1 months those patients treated with the immunotherapy combination lived for a median of 18.1 months compared with a median of 14.1 months in the chemotherapy group.

In the longer term, the combination may face competition from AstraZeneca’s Imfinzi (durvalumab), which is being tested in combination with chemotherapy in first-line mesothelioma in the DREAM trial.

Imfinzi is also being tested as a salvage therapy, where Pfizer/Merck KGaA’s Bavencio (avelumab), Opdivo monotherapy and Merck & Co’s Keytruda (pembrolizumab) are also in clinical development.

Opdivo and Yervoy is also approved by the FDA in two other thoracic cancer indications: as a first-line treatment for patients with metastatic non-small cell lung cancer (NSCLC) whose tumours express PD-L1 and in combination with limited chemotherapy for the first-line treatment of adults with metastatic or recurrent NSCLC with no EGFR or ALK mutations regardless of PD-L1 expression.

However BMS said separately that a trial of the Opdivo and Yervoy combination that aimed to raise the bar in care standards for people recovering from surgical removal of melanoma lesions, missed its target.

CheckMate -915, a randomised phase 3 study tested Opdivo plus Yervoy, compared with Opdivo in patients who have had a complete surgical removal of stage IIIb/c/d or stage IV melanoma.

BMS said addition of Yervoy to Opdivo in this trial did not result in a statistically significant improvement in recurrence-free survival (RFS) in the all-comer (intent-to-treat) population.

But the company said CheckMate -915 reinforced the established benefit of Opdivo monotherapy as a standard of care in the adjuvant setting.

The post FDA approves BMS’ Opdivo and Yervoy combination in first-line mesothelioma appeared first on .

Roche pays $200m for Vaccibody’s cancer vaccine drug

Roche’s Genentech unit has paid $200 million to develop an individualised cancer vaccine with Norwegian biotech Vaccibody, which focuses on targets known as neoantigens that spring up as tumours grow and mutate.

The deal with the Oslo-based company is worth up to $715 million, with potential milestone payments worth up to $515 million on top of the $200 million in near-term payments.

Vaccibody, which is also entitled to tiered royalties on sales of any marketed products, will develop the DNA-based individualised neoantigen vaccines until the end of phase 1b.

Genentech, which is already well established as a player in cancer immunotherapy with its PD-L1 class drug Tecentriq (atezolizumab), will be responsible for development and marketing thereafter.

The Roche unit will also bear all costs for trials, filings, manufacturing and marketing after the completion of the phase 1b study.

Vaccibody has been working on a targeted DNA-based vaccine platform, in a bid to find a potential new treatment paradigm for individualised cancer vaccines.

The deal focuses on Vaccibody’s investigational product VB10.NEO, which is designed to be produced on-demand according to the neoantigen profile of an individual patient and produces an immune response that attacks the tumour.

Neoantigens are only found on tumours and are therefore an attractive target for cancer immunotherapy as they may be recognised as foreign by the immune system, while leaving healthy tissue untouched.

Vaccibody is not the only company that is trying to make immunotherapies that target neoantigens: in the UK, Cambridge-based NeoPhore is looking at ways to encourage cancers to produce these mutations to broaden the potential immune response against cancer.

Frame Therapeutics has also begun work on neoantigen-based cancer vaccines based on RNA technology.

Earlier this year it began a research collaboration with eTheRNA, which is developing cancer therapies based on its proprietary mRNA technology.

And last year Tokyo-based NEC Corporation and VAXIMM began a neoantigen collaboration using the former’s artificial intelligence technology and the latter’s T-cell immunotherapy technology.

The post Roche pays $200m for Vaccibody’s cancer vaccine drug appeared first on .

Triumvira Immunologics raises $55M in Series A funding round for new form of cancer cell therapy

The company hopes to use the money to bring its T-cell antigen coupler cell therapy technology – currently in preclinical development – into human clinical trials. German drugmaker Bayer’s venture capital arm led the round.

TIGIT Therapies Digital Summit

The TIGIT checkpoint has gained much attention in recent years due to the number of roles it plays in several malignancies. TIGIT’s diverse function in multiple cell types has made it a lucrative immunotherapy candidate, with an increasing number of candidates being investigated in pre-clinical and clinical settings.

As evidence of TIGIT’s clinical potential builds, the TIGIT Therapies Digital Summit brings you a comprehensive insight, from the fundamental biology to early clinical development, and the next wave of ICI.

Hear from leaders in TIGIT development from Merck, Compugen, iTeos Therapeutics, Agenus and more!

Core challenges must still be addressed by the community, such as:

Investigating the TIGIT pathway, ligands, and modes of action
Optimizing biomarker strategy to identify optimal patient populations and indications
Rationalizing combination strategy to leverage the TIGIT mechanism for maximum therapeutic benefit.

The TIGIT Therapies Digital Summit will gather the greatest minds in TIGIT research. Gain access to 1:1 time with leaders in TIGIT development, and take away tangible learnings with dedicated panel discussions and poster sessions throughout the event.

In such a fast-paced industry, this is a unique opportunity to provide an overview of the ‘knowns and unknowns’ in the field, and to accelerate the safe development of TIGIT therapies with maximum clinical impact.

For more details about the program, speaker faculty or pricing and discounts, download the brochure.

The post TIGIT Therapies Digital Summit appeared first on .

GSK moves further ahead in BCMA with EU okay for Blenrep

GlaxoSmithKline has approval on both sides of the Atlantic for its multiple myeloma drug Blenrep, after getting the nod from the European Commission.

Blenrep (belantamab mafodotin) becomes the first BCMA-targeted drug for myeloma to be approved in the EU, and according to its label can be used to treat the blood cancer in adult patients who have received at least four prior therapies.

Prior drugs have to include an immunomodulatory agent, a proteasome inhibitor and a CD-38 monoclonal antibody, and the patient must show progression of the disease since the last therapy.

Among a crowded field, GSK has now won the race to bring an anti-BCMA drug to market in both Europe and the US. The FDA backed Blenrep for the same indication three weeks ago, despite some reservations about eye toxicity, which set the drug on course for what some analysts think could be revenues of around $1.5 billion in 2026.

The EU and US approvals for Blenrep are based on two phase 2 trials – DREAMM-1 and DREAMM-2 – which showed that the antibody-drug conjugate (ADC) achieved responses in around a third of patients who had received an average of seven prior treatments for the cancer.

The conditional approvals mean that GSK may need survival data from a larger trial to keep the drug on the market in the longer term.

That data should come from an ongoing phase 3 study comparing Blenrep with BMS’ immunomodulatory drug Pomalyst (pomalidomide) plus low-dose dexamethasone, the standard treatment option for relapsed and refractory multiple myeloma.

It’s a key new launch for GSK as it tries to rebuild its oncology business under chief scientific officer Hal Barron, along with PD-1 inhibitor dostarlimab and PARP inhibitor Zejula (niraparib) – acquired as part of its $5.1 billion takeover of Tesaro.

Blenrep looks set to have a longer-than-expected period without direct BCMA competition after a BCMA-targeted cell therapy from Bristol-Myers Squibb and bluebird bio – ide-cel (bb2121) – was held up by an FDA request for more data.

Ide-cel was resubmitted to the FDA earlier this month, while other BCMA drugs coming through the pipeline include Amgen’s AMG-429 and Regeneron’s REGN5458 – both bispecific antibodies – as well as ADCs from AstraZeneca and BMS.

With approvals in hand for later-line therapy of myeloma, GSK’s attention will now switch to developing the drug for earlier use in the disease.

Its head-to-head trial with Pomalyst is recruiting patients with at least two prior lines of anti-myeloma treatments, and it also has a phase 3 trial running adding Blenrep to standard first-line treatment with Takeda’s proteasome inhibitor Velcade (bortezomib), BMS’ immunomodulator Revlimid (lenalidomide) and dexamethasone in newly-diagnosed patients ineligible for a bone marrow transplant.

The post GSK moves further ahead in BCMA with EU okay for Blenrep appeared first on .

FDA starts quick review of Merck’s MET-targeting lung cancer drug

Merck KGaA could be looking at an early 2021 approval for its MET inhibitor tepotinib in lung cancer from the FDA after the US regulator started a priority review of the drug, but could still be beaten to market by a rival drug.

Tepotinib has been developed to treat non-small cell lung cancer (NSCLC) with MET exon 14 (METex14) mutations, which occur in 3% to 5% of all cases of this form of lung cancer. It picked up its first world approval – as Tepmetko – in  Japan earlier this year.

The once-daily oral drug was awarded breakthrough status by the FDA last year for METex14-positive NSCLC patients who had progressed after first-line platinum-based chemotherapy, a week after the US regulator gave the same designation to another MET inhibitor – capmatinib – from Incyte/Novartis.

Capmatinib beat tepotinib to the US market, getting FDA approval in May under the Tabrecta trade name for all patients with METex14-positive NSCLC, regardless of whether or not they have received prior treatment. Merck is also seeking approval for a “line-agnostic” indication for its drug.

Merck’s filing comes on the back of a phase 2 study – called VISION – which enrolled patients with METex14-positive NSCLC diagnosed using an assay developed by ArcherDX that can detect the mutation from a blood test or tumour biopsy.

Tepotinib treatment achieved an objective response rate (ORR) of 48% among patients diagnosed using a blood test and 50% in the biopsy group, with a median duration of response of just over 11 months.

Tabrecta was approved based on the GEOMETRY mono-1 trial, which found a 68% ORR in patients treated with the drug first-line, with a 12.6 month median duration, and 41% among previously-treated patients with a duration of 9.7 months.

Novartis and Incyte’s drug was approved alongside a companion diagnostic developed by Foundation Medicine.

“METex14 skipping alterations drive a particularly aggressive form of NSCLC in a patient population that is generally elderly, facing poor clinical prognosis and in urgent need of new therapeutic options,” said Luciano Rossetti, Merck’s head of R&D.

Other MET inhibitors are also coming through development, including AstraZeneca’s savolitinib, which is being developed initially for NSCLC patients who have progressed after treatment with AZ’s EGFR inhibitor Tagrisso (osimertinib), and as a combination therapy for NSCLC.

MET is a recognised resistance pathway in EGFR-positive NSCLC, and earlier this year AZ reported preliminary data showing that the Tagrisso/savolitinib combination was well-tolerated in patients who had failed first-line EGFR drugs and was able to achieve partial responses in up to 48% of patients.

Merck is also exploring this application with tepotinib, and has a trial of its drug alongside Tagrisso.

The post FDA starts quick review of Merck’s MET-targeting lung cancer drug appeared first on .

Novartis eyes filings for leukaemia drug asciminib after phase 3 win

A first-in-class STAMP inhibitor developed by Novartis – asciminib – has outperformed a current drug for chronic myeloid leukaemia (CML) in a head-to-head trial, setting up regulatory filings.

The ASCEMBL trial compared asciminib (ABL001) to Pfizer’s Bosulif (bosutinib) in Philadelphia chromosome-positive CML patients who had previously been treated with two or more tyrosine kinase inhibitors (TKIs), the go-to treatment for this form of CML.

Current treatment for these patients relies on first-generation TKI imatinib – sold by Novartis as Glivec but also available as a generic – as well as second-generation drugs such as Bosulif, Bristol-Myers Squibb’s Sprycel (dasatinib) and Novartis’ Tasigna (nilotinib).

These drugs have transformed the prospects of people with CML, but most patients eventually see their cancer progress despite TKI therapy, and resistance to these drugs can lead to treatment failures. They can also be hard to tolerate, as they can affect healthy as well as leukaemic cells.

Asciminib’s novel mechanism of blocking STAMP – the ABL myristoyl pocket of the BCR-ABL tyrosine kinase – could sidestep conventional TKI resistance and side effects and provide a new third-line treatment option for CML patients, says Novartis.

In ASCEMBL, asciminib was better than Bosulif at achieving a major molecular response (MMR) at 24 weeks than Bosulif in Ph+ CML patients in chronic phase, the stage of the disease when the blood and bone marrow contains less than 10% malignant cells.

Molecular response is measured using a genetic probe to detect BCR-ABL gene mutations in blood or bone marrow based on the number of leukaemic cells present, and is considered to be MMR when the mutations are found at a rate of 1/1000th or less than would be expected in an untreated patient.

“Our ability to treat patients with TKIs changed CML care forever. However, the risk of disease progression is a reality for many patients,” said Novartis’ chief medical officer John Tsai.

“These results with asciminib are a testament to our commitment to further transform CML care – this time through STAMP inhibition, by exploiting a natural regulatory mechanism of the ABL kinase,” he added.

The FDA has awarded the drug fast-track status, given to new therapies that treat a serious or life-threatening condition and fill an unmet medical need, which means it could be eligible for a speedy review after filing.

Novartis originally planned to go after approval as a second-line treatment option as well on the back of another trial called ASC4MORE, but is sticking with third-line for the time being.

Data from the ASCEMBL trial will be submitted for presentation at an upcoming medical meeting, and results will be shared with regulatory authorities, said the drugmaker.

If approved, asciminib would beef up Novartis’ CML portfolio, which is a big earner for the company. Tasigna made sales of $1.8 billion last year, and despite generics Glivec is still a blockbuster brand for the company, adding almost $1.3 billion to its 2019 revenues.