Clinical Trials

BMS reports data that could give its cell therapy an edge over rival J&J product

Bristol Myers Squibb has data showing that its multiple myeloma treatment Abecma beat the standard of care, preliminary Phase 3 results that could support moving the FDA-approved CAR T-cell therapy into an earlier line of treatment—ahead of a rival cell therapy. In other BMS cell therapy news, the pharma giant started an R&D alliance focused …

BMS reports data that could give its cell therapy an edge over rival J&J product Read More »

GSK pays $100M to partner on a cancer drug with new twist on immunotherapy

GSK is partnering with Mersana Therapeutics on the development of an antibody-drug conjugate that is approaching Phase 1 testing. Unlike other ADCs that deliver a toxic drug payload, the Mersana drug is designed to activate the innate immune system to fight tumor cells.

Pfizer to buy sickle cell disease biotech Global Blood Therapeutics in $5.4B deal

Pfizer’s own R&D efforts in sickle cell disease have fallen short, so the pharmaceutical giant is turning to M&A to gain a presence in the blood disorder. The acquisition of Global Blood therapeutics brings the commercialized drug Oxbryta plus a pipeline of other therapeutic candidates.

ODM standard for CRF design

Over the last few years, our industry has become familiar with CDISC standards. This has largely been driven by regulation, with national regulators such as FDA and PMDA mandating the use of submission standards such as SEND, SDTM, and ADaM. This post shines a spotlight on the lesser-known Operational Data Model (ODM) standard, which is often overlooked as it’s …

ODM standard for CRF design Read More »

FDA turns down Acadia Pharma drug for Alzheimer’s psychosis; new trial needed

Acadia Pharmaceuticals drug Nuplazid failed to win FDA approval for the treatment of psychosis in Alzheimer’s disease patients. The regulator said that the data submitted were not from an adequate and well-controlled study and the company must run another clinical trial.

FDA approval of AstraZeneca drug is first for a new group of breast cancer patients

Enhertu, a breast cancer drug from AstraZeneca and Daiichi Sankyo, is now FDA approved for treating patients whose tumors express low levels of the protein HER2. The decision makes the infused therapy the first targeted treatment for this new category of breast cancer patients.

Alnylam drug succeeds in heart study, setting stage for FDA filing and showdown with Pfizer

Alnylam Pharmaceuticals drug Onpattro, an FDA-approved treatment for nerve pain caused by hereditary transthyretin amyloidosis, now has Phase 3 data showing it can also help the much larger group of patients suffering heart problems from the rare protein disease. An FDA submission is planned, which would pit the Alnylam drug in competition with a blockbuster …

Alnylam drug succeeds in heart study, setting stage for FDA filing and showdown with Pfizer Read More »

H1 & Lokavant Partner to Power Next-Gen Clinical Trials

What You Should Know: – H1and Lokavant today announced an extensive data-and-analytics collaboration to bring enhanced automated intelligence to drug development – As part of the bi-directional partnership, Lokavant will provide performance data on global trial sites and principal investigators from thousands of studies to supplement H1’s existing Trial Landscape clinical trial repository.  Integrating Clinical …

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Connecting EHR to clinical trials: How to embrace the promise of real-world data

We all know that real-world data (RWD) from electronic health records (EHR) could boost clinical trial efficiency and improve patient outcomes – so what’s holding us back? It’s been more than two decades since the ground-breaking potential of using electronic health records (EHR) to inform clinical research was first mooted. Since then, progress has been …

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Aiming to bring drug combos into earlier lines of cancer therapy, IDRx lands $122M

Taking inspiration from the success of drug cocktails that halt viral infections, startup IDRx aims to develop cancer drug combinations that stop tumor escape. The company, founded by serial entrepreneur Alexis Borisy, has set gastrointestinal stromal tumor (GIST) as its first disease target.

Digital transformation: Reimagining clinical trial management [Sponsored]

Decentralized clinical trial approaches helped the pharma industry navigate through Covid-19. Now it is becoming increasingly clear that there’s a need for a hybrid approach to decentralized clinical trials that considers the perspectives of patients and the impact to clinical trial sites.

With ‘bold experiment’ in drug pricing, Arcutis aims to stand out in plaque psoriasis

The FDA approved Arcutis Biotherapeutics drug Zoryve, a topical treatment for plaque psoriasis. With the decision, the company will proceed with a pricing strategy it believes will speed up insurance coverage and patient adoption of the new treatment for the chronic inflammatory skin disorder.

FDA hits pause on Beam Therapeutics’ off-the-shelf cell therapy for blood cancers

Beam Therapeutics’ application to begin human testing of a cell therapy made with base-editing technology is now under an FDA clinical hold. In other clinical hold news, the FDA lifted a hold on a Celyad Oncology cell therapy clinical trial that was halted in March.

Global comparator data signal “alarm bells” for UK Government, says APBI

Latest UK Life Sciences Competitive Indicators “ought to ring alarm bells across Government,” says ABPI. Despite the UK’s potential as an international life sciences leader, it lags many competitors in a number of key metrics, including access to new medicines and global share of clinical trial recruitment. Richard Torbett, chief executive at the Association of …

Global comparator data signal “alarm bells” for UK Government, says APBI Read More »

Report finds UK risks falling behind rivals in life sciences

A just-published report on the UK’s life sciences sector “ought to ring alarm bells across government,” according to the Association of the British Pharmaceutical Industry (SBPI). The Life Sciences Competitiveness Indicators report shows that spending on life sciences R&D was £2.7 billion in 2020 – accounting for 0.12% of GDP and ranked third among the …

Report finds UK risks falling behind rivals in life sciences Read More »

Medable Partners with Withings Health Solutions to Integrate Health Devices in Decentralized Clinical Trials

What You Should Know: – Medable Inc., the leading SaaS platform provider for patient-centered clinical trials, today announced a new partnership with Withings Health Solutions, the business-to-business division of Withings, one of the global leaders in at-home connected health – Withings’ devices will seamlessly connect to Medable’s decentralized clinical trial platform, reducing the burden on sponsors and …

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ForSight Robotics Raises $55M to Develop Robotic Cataract Surgery Procedure

What You Should Know: – Israeli-based ForSight Robotics, the pioneer in ophthalmic robotic surgery raises $55M in Series A funding to advance the world’s first surgical platform for fully robotic cataract surgery: ORYOM. The round was led by The Adani Group with existing investors Eclipse Ventures and Mithril Capital. A number of new investors, including …

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Practicing for the Next Pandemic with Real-World Data

Mac Bonafede, Ph.D., MPH, VP of Real World Evidence at Veradigm Data reporting challenges during the current pandemic relate to the lack of connectivity between public health and information technology (IT) reporting systems throughout the country. While access to real-world data (RWD) helps address this situation, the solution goes beyond simple accessibility. Real-world data represents …

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How to accelerate clinical study build

As you know, clinical trials can be complex. Industry standards are now required for clinical trial submission. This means even more associated costs, resources, time, and effort. The Formedix clinical metadata repository (MDR) can help! So what can it do for you? In a nutshell, it can help you:       How is this possible? Take studies …

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Wysa Secures $20M for AI-Powered Mental Health Chatbot

What You Should Know:  – Wysa, a Boston, MA-based AI digital platform for mental health, today announces it has secured $20M in financing led by HealthQuad, who along with British International Investment (BII), the UK’s development finance institution, joins earlier investors W Health Ventures, Kae Capital, Google Assistant Investments, and pi Ventures amongst others. – …

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FDA delay means BeiGene, Novartis must wait longer for cancer drug decision

The FDA has deferred a regulatory decision on an esophageal cancer drug from partners BeiGene and Novartis, citing the need to complete inspections of the China-based facilities where the drug is made. The agency said Covid-19 travel restrictions have prevented it from conducting those inspections.

EMA pilot will see if clinical trial data should be digested ‘raw’

A pilot study has been launched by the EMA to see whether it is helpful for regulators to look at the ‘raw data’ from clinical trials submitted in support of new marketing application along with the usual structured analyses from sponsors. The pilot will focus on individual patient data from clinical studies in electronic structured …

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Merck strikes a $290M deal for novel prostate cancer drug

Merck is partnering with Orion on the clinical development and potential commercialization of a new drug for advanced prostate cancer. Though Merck already has a piece of the prostate cancer drug market, Orion’s drug would offer a new approach for treating cancers that have become resistant to currently available hormone therapies.

Areteia gets $350M to turn failed ALS drug into a therapy for severe asthma

New company Areteia Therapeutics launched with up to $350 million in financing and an asthma drug candidate from Knopp Biosciences. That drug, dexpramipexole, previously failed a pivotal test in amyotrophic lateral sclerosis but more recent clinical testing found that the way the small molecule works has applications in eosinophilic asthma, a severe form of the …

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Roche pays $55M for late-stage drug for rare kidney disease with no approved treatment

An Ionis Pharmaceuticals drug has positive Phase 2 results in immunoglobulin A nephropathy, leading Roche to exercise its option to license the rights to the molecule. The pharmaceutical giant gets a contender in the chase to win the first regulatory approval of a therapy for this rare kidney disease.

Vertex builds up pipeline of type 1 diabetes cell therapies with $320M buyout

Vertex Pharmaceuticals already has a cell therapy candidate for type 1 diabetes but the company says acquiring rival ViaCyte will provide it with complementary assets and technologies. The two companies already share a partner in common: both have therapeutic candidates that use the gene-editing capabilities of CRISPR Therapeutics.

Pfizer, BioNTech Covid-19 vaccine gets full FDA approval for adolescents

The FDA has granted the Pfizer and BioNTech Covid-19 full approval for use in adolescents. In other Covid-19 news, pharmacists were granted additional prescribing powers for an antiviral and Novavax’s European marketing authorization has been expanded to an additional age group.

AbbVie & Alector drop Alzheimer’s med from alliance, shifting focus to a 2nd drug

AbbVie paid Alector $205 million up front five years ago to begin an R&D partnership on two drugs intended to bring an immunotherapy approach to the treatment of Alzheimer’s disease. One of the drugs has reached Phase 2 testing but the pharmaceutical giant has terminated the alliance on a program in Phase 1.

Digital tools driving innovative clinical trials

The COVID-19 pandemic has catalysed significant changes in the way pharma develops drugs, particularly in the clinical trial space. Hybrid or decentralised clinical trials (DCTs) have gained traction as technology, infrastructure and knowledge have evolved to support their use. Preliminary research suggests that hybrid DCTs can reduce trial timing and resources, improve patient-centricity and  patient …

Digital tools driving innovative clinical trials Read More »

With new gene therapy data in hand, Sarepta talks with FDA about approval pathways

Sarepta Therapeutics has more clinical data showing the safety and efficacy of its experimental gene therapy for Duchenne muscular dystrophy. A pivotal Phase 3 test is already underway and could post data next year but the company is also talking with regulators about the possibility of a submission under the accelerated approval pathway.

CureVac sues BioNTech, claiming Covid-19 vaccine infringes key mRNA patents

Messenger RNA vaccine biotech CureVac is suing BioNTech, alleging that its rival’s Covid-19 vaccine infringes on intellectual property built on more than two decades of research. BioNTech responded that its work is original and the company will defend against the infringement allegations.

AstraZeneca broadens its scope in blood cancers with $100M TeneoTwo buyout

AstraZeneca is acquiring TeneoTwo in a $100 million bet on that biotech’s lymphoma drug candidate, which is designed to go after two targets. It’s the latest deal made by a big pharmaceutical company looking to get its hands on a new type of cancer therapy called a T cell engager.

Ensuring successful digital outreach and retention in atopic dermatitis trials

Trial organisers face intense competition to find and recruit eligible patients for atopic dermatitis studies. With more than 500 dermatology clinical trials currently underway, it is often heard that there is a ‘’competition for participants’’. Yet evidence from research studies and a survey with 4,000+ atopic dermatitis patients conducted by Clariness shows that this ‘race …

Ensuring successful digital outreach and retention in atopic dermatitis trials Read More »

UK adds £175m funding to clinical research recovery drive

The UK government has made an additional £175 million ($212 million) in funding available for its push to make the country a go-to location for clinical research, adding to £200 million promised in March to strengthen the national data infrastructure. The new money has been announced to coincide with the publication of a new three-year …

UK adds £175m funding to clinical research recovery drive Read More »

Liver injury leads FDA to pause Sanofi’s tests of MS drug acquired in $3.7B deal

The FDA placed a partial clinical hold on late-stage tests of Sanofi multiple sclerosis drug tolebrutinib after some patients developed drug-induced liver injury. While U.S. tests are paused, Sanofi said clinical testing in other countries is continuing with additional safety monitoring.

Feds commit $3.2B to lock in Covid-19 vax supply ahead of fall vaccination campaign

New Covid-19 vaccine booster shots are coming and they’ll likely include protection against the omicron variant. The U.S. government agreed to a purchasing contract covering 105 million doses of the Pfizer/BioNTech vaccine for a planned vaccination campaign in the fall.

From trial design for patients to trial design with patients: a key topic at DIA ’22

At the 2022 Drug Information Association (DIA) annual meeting last week, it was inspiring to connect with fellow research and development (R&D) stakeholders, regulators, health and digital data partners and more to navigate challenges in drug development that impact patients globally. As an industry, we recognise patients as partners in the development process, but if …

From trial design for patients to trial design with patients: a key topic at DIA ’22 Read More »

Care Access Acquires IBPClin to Expand Decentralized Clinical Trial Capabilities to Latin America

What You Should Know: – Today, Care Access announced the acquisition of IBPClin, a leading clinical trial center in Rio de Janeiro. As a result, Care Access will bring its innovative approach to decentralized clinical research to Latin America. – The acquisition demonstrates progress toward building diverse representation in clinical research and is a strategic first step following Reify Health’s $220 million investment round that was raised to …

Care Access Acquires IBPClin to Expand Decentralized Clinical Trial Capabilities to Latin America Read More »

Reimagining The Future of Pharmacovigilance (PV): Regulations, Tech, Public Perceptions

Barry Mulchrone, Senior Director& Head of Pharmacovigilance Oversight and Analytics, IQVIA Annette Williams, VP & Global Head, Lifecycle Safety, IQVIA Monitoring the safe and effective use of medicines has now been an essential part of public health policy for several decades. New advances in technology (e.g., ICH E2B for the electronic exchange of case reports) …

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Two for two: Sanofi snares twin European nods for enzyme replacement therapies

Sanofi won European Commission approval for two therapies that treat rare enzyme deficiencies. Xenpoyzme is the first treatment for acid sphingomyelinase deficiency (ASMD) while Nexviadyme is a successor to a blockbuster Sanofi drug for Pompe disease.

BMS cancer drug’s expanded approval gives it an edge over a Gilead cell therapy

The new FDA approval for Breyanzi moves the Bristol Myers Squibb cancer cell therapy into an earlier line of treatment for an aggressive type of blood cancer. The decision also gives the pharmaceutical giant access to an additional pool of patients untapped by a rival cell therapy from Gilead Sciences.

Ipsen’s $247M Epizyme acquisition brings approved cancer drug & more in pipeline

Ipsen is paying $247 million to acquire Epizyme, a cancer drug developer with one FDA-approved product: follicular lymphoma therapy Tazverik. The drug is a modest seller now, but more cash could be paid out if the small molecule hits sales targets in coming years.

Study exposes lack of diversity in US cancer trials

Almost half (48%) of cancer clinical trials in the US have no Hispanic or Latin American subjects, and 42% have no black patients, according to a diversity analysis focusing on thousands of studies conducted in the past 15 years. The analysis – from a dataset that spans more than half a million patients – reveals …

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As muscular dystrophy drug meets key goal, Italfarmaco plans march forward to regulators

A Duchenne muscular dystrophy drug candidate from Italfarmaco Group has encouraging preliminary data from a pivotal study. The Milan, Italy-based pharmaceutical company says it now plans to meet with U.S. and European authorities about seeking regulatory approval for the small molecule.

Analysis: Is Oracle Cerner Planning to Build A National EHR?

Earlier this month Oracle closed its $28.3B acquisition of Cerner and shortly after Oracle founder and CTO, Larry Ellison, outlined Oracle’s vision for Healthcare IT post-acquisition. The key elements of this vision included:   – Developing a solution for a national EHR database.  – Leveraging this solution, and similar smaller-scale connected EHR initiatives, to provide life …

Analysis: Is Oracle Cerner Planning to Build A National EHR? Read More »

New data for Intellia’s CRISPR therapy shows potential for one-time treatment of a rare disease

An Intellia Therapeutics therapy that uses CRISPR to perform in vivo genomic edits has clinical data showing that the therapeutic effects continue for a year in patients. The biotech now plans to speak with regulators about the design for a pivotal clinical trial testing this therapy, a potential one-time treatment for a rare inherited disease.

Which clinical trial innovation startups will present at INVEST PharmaTech Pitch Perfect?

The Pitch Perfect contest at MedCity INVEST PharmaTech, the free virtual conference scheduled for July 26, will spotlight five startups seeking to improve clinical trials.

Athira Alzheimer’s drug fails mid-stage, but the biotech still eyes a Phase 3 path

An Alzheimer’s disease drug from Athira Pharma has failed a Phase 2 clinical trial and the biotech has a surprising theory why. Athira says standard of care drugs Alzheimer’s patients are already taking may diminish the effect of its small molecule and this finding could inform how the company proceeds with an ongoing Phase 3 …

Athira Alzheimer’s drug fails mid-stage, but the biotech still eyes a Phase 3 path Read More »

Moderna facility in UK confirmed, part of 10-year deal with gov’t

Moderna has said it will open a new R&D and manufacturing facility in the UK which could lead to improved access by NHS patients to mRNA-based vaccines and drug treatments. The facility is the centrepiece of a 10-year agreement-in-principle between the biotech and the UK government – which has been rumoured to be on the …

Moderna facility in UK confirmed, part of 10-year deal with gov’t Read More »

Aiming to catch Alnylam, AstraZeneca & Ionis plan FDA filing for rare disease drug

The planned FDA submission follows the report from AstraZeneca and Ionis Pharmaceuticals that their partnered drug, eplontersen, met the main goals of a pivotal study in treating nerve pain caused by hereditary transthyretin-mediated amyloidosis. The data come one week after rival Alnylam Pharmaceuticals won FDA approval for its second drug for this rare disease.

An introduction to case report forms

Different types of CRFs A Case Report Form (CRF) is a printed, optical, or electronic document designed to record all information required to be reported to the sponsor on each trial subject. CRFs play a crucial role in helping to assess the safety and efficacy of clinical products. An electronic CRF (eCRF) is an electronic …

An introduction to case report forms Read More »

Pfizer, Moderna Covid-19 vaccines are now authorized for kids as young as 6 months

Covid-19 vaccines from Pfizer and Moderna now have FDA emergency use authorization for children as young as 6 months old. If the Centers for Disease Control and Prevention signs off on these shots this weekend, these pediatric vaccines could become available for distribution early next week.

Pharmacy chain Walgreens plans to disrupt clinical trial sector

Walgreens is the latest US retail pharmacy giant to have a go at disrupting the clinical trials industry, with the launch of a new business unit that will tap into its reserve of patient data, as well as the ability to recruit patients across its retail network. The company says it plans to use a …

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Walgreens Launches New Clinical Trial Business to Address Access & Diversity

What You Should Know: – Walgreens today announced the launch of its clinical trial business to redefine the patient experience and increase access and retention in sponsor-led drug development research. – Walgreens flexible clinical trial model combines the company’s vast foundation of patient insights, partner-enabled health and technology capabilities and in-person and virtual care options …

Walgreens Launches New Clinical Trial Business to Address Access & Diversity Read More »

Why diverse clinical trials matter: the pharmaphorum podcast

In this episode of the pharmaphorum podcast, editor in chief Jonah Comstock welcomes Dr. Jennifer Jones-McMeans, divisional vice president for global affairs for Abbott’s vascular business to discuss diversity and equity in clinical trials. They discuss the nitty-gritty of clinical trial diversity – why it’s been so hard to achieve, why it’s important, and how …

Why diverse clinical trials matter: the pharmaphorum podcast Read More »

BIO panel: How Covid-19 is preparing us to better respond to the next pandemic

Biopharmaceutical companies and public health officials have learned a great deal about pandemic response in the last two years. A panel discussion during the annual meeting of the Biotechnology Innovation Organization explored some of the lessons from Covid-19 and how they can prepare us for the next pandemic.

ImCheck fetches €96M for antibodies that activate immune cells against cancer

ImCheck Therapeutics aims to broaden the reach of cancer immunotherapy with antibody drugs that activate a rare but powerful type of immune cell called gamma delta T cells. The biotech has unveiled €96 million to advance its pipeline, including a lead candidate in development for both liquid and solid tumors.

Tessa Therapeutics takes in $126M for cell therapies, and more biotech financings

Ten biotech companies were able to raise more than $500 million in combined financing in the past week. Here’s a recap of the funding activity, which spanned AI-based drug discovery, cell therapy clinical research, cancer drug development, and more.

4 Steps for Operationalizing the FDA’s Call To Improve Health Outcomes for All Patients

C.K. Wang, Chief Medical Officer of COTA, Inc. In April, FDA Commissioner Dr. Robert Califf laid out areas of challenge and opportunity when it comes to improving health outcomes, restoring public trust in science, and making the most of our ongoing investments in data analytics and clinical research. More specifically, Califf made clear that our current healthcare …

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Owkin bags $180m BMS alliance for cardiovascular clinical trials

Owkin has enticed another big pharma partner with its artificial intelligence-powered clinical development platform, Bristol-Myers Squibb, which is paying $80 million upfront to tap into its expertise. The deal is split between a Series B financing led by BMS and upfront fees, taking the total raised by the Paris, France-based start-up to $300 million, and …

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ASCO recap: Breast cancer breakthrough, Mirati’s moves, Arcellx ascends & more

Breast cancer drugs and cell therapies were prominent in this year’s annual meeting of the American Society of Clinical Oncology. Here’s a recap of some of the news highlights from the biggest cancer conference of the year.

PMV’s ASCO data show signs of drugging ‘undruggable’ p53 protein across tumor types

PMV Pharmaceuticals has its first clinical data showing that its lead therapeutic candidate can hit p53, a tumor-suppressing protein long thought to be undruggable. After reporting the preliminary data during ASCO, PMV said it plans to continue Phase 1 study with the goal of advancing to Phase 2 early next year.

Gilead offers more detail on breast cancer drug’s trial results. Is it enough?

Gilead Sciences has more detail about breast cancer drug Trodelvy, a blockbuster hopeful it acquired two years ago. Additional data from a pivotal study were presented during the annual meeting of the American Society of Clinical Oncology and they still leave many questions unanswered.

BMS bets $4.1B on lung cancer drug that could best products from Pfizer, Roche

Bristol Myers Squibb is splashing out $4.1 billion to acquire Turning Point Therapeutics, a clinical-stage biotech whose lead drug is currently in pivotal testing as a treatment for certain lung cancers. Recently reported preliminary data suggest the small molecule could have advantages over currently available drugs in the same class from rivals Pfizer and Roche.

Repare relinquishes cancer drug to Roche, gets $125M to finance growing pipeline

Roche’s deep oncology pipeline is adding a drug candidate from Repare Therapeutics that works by interfering with a DNA repair mechanism of cancer cells. Repare receives $125 million up front and it retains the right to share in the development and commercialization of the drug, camonsertib.

Akili + Roblox Form First of Its Kind Digital Treatment Partnership

What You Should Know: – Akili’s EndeavorRx, the first and only FDA-cleared video game ADHD treatment for kids, is uniting with Roblox’s global virtual universes forming a first-of-its-kind partnership.  – Initially, the partnership will establish an exclusive Roblox reward exchange for patients that complete their medically prescribed EndeavorRx treatment. The rewards will enable patients to redeem items via the exchange …

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ASCO 2022 ahead: Clinical trial updates and data readouts to watch for

The American Society of Clinical Oncology’s annual meeting begins this week, including the presentation of more than 2,800 abstracts. Amgen, Roche, and Sierra Oncology are among the companies with key presentations at this year’s gathering.

Using technology to reassess the clinical trial landscape

Josh Sackman, president and co-founder of AppliedVR, and Web Sun, president and co-founder of Komodo Health, relay how their newly formed collaboration reshapes the clinical trial process by using data, helping to cut costs and allowing for evaluation of broader patient populations.   AppliedVR and Komodo Health are collaborating to perform a clinical trial using the …

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A Balm for Psyches Scarred by War

MDMA-assisted treatment for post-traumatic stress disorder “represents real hope for long-term healing,” health experts say.

Covid-19 roundup: Boosters for kids, CAR-NK cell therapy, Paxlovid rebound & more

Pfizer and BioNTech reported positive clinical data for a Covid-19 booster in children younger than 5, results released ahead of a June FDA advisory committee meeting scheduled to discuss the use of that vaccine and Moderna’s in kids. In other Covid news this week, the CDC issued a health advisory for Pfizer’s antiviral Paxlovid and …

Covid-19 roundup: Boosters for kids, CAR-NK cell therapy, Paxlovid rebound & more Read More »

Can technology help diversify clinical trials?

This is the second in a two-part series examining how pharma can embrace the DEI agenda to create more effective medicines through more representative clinical trials. You can read part one of this article here. As the shift to home working proliferated during the pandemic, work became decentralised – and many businesses were able to …

Can technology help diversify clinical trials? Read More »

Why are clinical trials struggling with diversity?

In the diverse world we live in, the products we produce, the services we create and the places we work should be as equally representative. In recent years this has seen diversity, equity, and inclusion (DEI) become a big focus for industries to get right. Within pharma and healthcare, where greater representation has been proven …

Why are clinical trials struggling with diversity? Read More »

Heart trouble report & clinical hold spell end of Antios, Assembly Bio HBV alliance

A clinical hold on an Antios Therapeutics drug has led to the end of a collaboration with Assembly Biosciences less than a year after their alliance began. The partners had hoped pairing their drugs with an approved Gilead Sciences antiviral would produce a combination hepatitis B treatment with the potential for a cure.

Find your inner data and take precision marketing to the next level

Data is the powerhouse of the pharmaceutical sector. It powers drug discovery, clinical trials, and operational performance. But sizeable reservoirs of data remain unseen and untapped to commercial operations, allowing insights, opportunities, and efficiencies to leak away. Organisations are missing out on the ability to harness their data to energise precision marketing and transform commercial …

Find your inner data and take precision marketing to the next level Read More »

Biohaven drug fails another clinical test, this time in rare neuromuscular disorder

Biohaven’s migraine drug research led to an FDA-approved product with strong commercial uptake, but its other neuroscience efforts have yet to achieve the same outcomes. Drug candidate troriluzole has failed a Phase 3 test in neuromuscular disorder spinocerebellar ataxia, the molecule’s third failure in a pivotal study in the past two years.

Innoviva’s Entasis acquisition brings a novel antibiotic that’s ready for the FDA

Entasis Therapeutics is being acquired by Innoviva in a deal that brings an FDA-ready antibiotic developed to address drug-resistant strains of a bacterium that infects critically ill, immunocompromised patients. Innoviva has been steadily building up its stake in Entasis, an AstraZeneca spinout, over the past two years.

ePROs: Better Patient Input for Better Trial Output

Kris Gustafson, Vice President and Global Head Patient Centered Technologies, IQVIA During the pandemic, electronic clinical outcomes assessments (eCOAs) gained prominence as sponsors looked for more agile tools to help them capture patient data remotely through decentralized clinical trials (DCTs). From the adoption of virtual models like eCOAs and DCTs, a new opportunity emerged to …

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Cerner Collaboration to Increase Access to Cancer Clinical Trials

What You Should Know: – Cerner, Elligo Health Research® and Freenome are collaborating to enable a clinical trial through the Learning Health Network to help advance early cancer detection. – Together they will be using the research-activated network of health systems, real-world data (RWD) and multiomics technology, developed by Freenome, with the goal to help accelerate early cancer detection. Importance of Early Cancer Detection …

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INVEST Pitch Perfect winner: Modality.ai makes Tina the Alexa of clinical trials

Modality.ai has developed a conversational AI system that replaces in-person interviews that are part of neurological assessments for a range of disorders. The startup was judged co-winner in the pharmatech category during the recent MedCity INVEST conference in Chicago.

To focus on cancer, Chimerix sells rights to antiviral drug that defined the biotech

Chimerix is selling global rights to smallpox drug Tembexa, the biotech’s only FDA-approved product, as a way to fund clinical development of a therapy in pivotal testing for a rare type of brain cancer. The deal marks Chimerix’s nearly complete departure from the antiviral work that defined the company for most of its history.

UCB’s FDA setback delays competition in U.S. with blockbuster J&J psoriasis drug

The FDA cited manufacturing issues in turning down UCB’s application seeking approval for bimekizumab, an antibody drug developed to treat moderate-to-severe plaque psoriasis. Bimekizumab is already approved and available in other markets around world, but the FDA rejection delays UCB’s antibody drug from competing in the U.S. for now.

BMS builds on BridgeBio alliance, inking deal for rights to KRAS cancer drug

Bristol Myer Squibb is getting rights to BBP-398, a contender in the chase for drugs that address difficult-to-target KRAS mutations. Meanwhile, BridgeBio Pharma receives some needed cash as it restructures in the wake of the Phase 3 failure of its lead program late last year.

Moderna Vaccine Provokes Strong Immune Response in Children 6 to 11

Antibody levels rose in the children who received it, suggesting the vaccine protects against infection. But the data were gathered before the arrival of Omicron.

Moderna Vaccine Provokes Strong Immune Response in Children 6 to 11

Antibody levels rose in the children who received it, suggesting the vaccine protects against infection. But the data were gathered before the arrival of Omicron.

Moderna Vaccine Provokes Strong Immune Response in Children 6 to 11

Antibody levels rose in the children who received it, suggesting the vaccine protects against infection. But the data were gathered before the arrival of Omicron.

Closely watched Roche drug fails a second pivotal test in lung cancer

Roche reported that its drug tiragolumab failed to achieve one of two main goals of a Phase 3 study in non-small cell lung cancer. The antibody is designed to block TIGIT, a checkpoint protein that has been a hot target for pharmaceutical companies looking for a way to broaden the reach of cancer immunotherapy.

Osmind Secures $40M to Expand Mental Health EHR Platform

What You Should Know: – Osmind, a mental health EHR platform helping clinicians and researchers advance new life-saving mental health treatments, today announced a $40 million Series B investment led by DFJ Growth. The Series B round brings the total amount of funding for Osmind to $57M since the company’s founding in 2020. Justin Kao, …

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Pfizer buys its way into migraine with $11.6B Biohaven Pharma acquisition

Pfizer is turning to M&A to get its next blockbuster, buying Biohaven six months after the two companies began a commercialization pact centered on the oral migraine drug Nurtec. The acquisition will lead to the spinout of Biohaven’s other assets into a new, publicly traded company capitalized with $275 million.

Digital Documentation: How PDFs Can Help Providers Expedite Care

Deboshree Sarkar, Product Marketing Manager, PDF Editor Product line at Foxit We live in an increasingly digital world. Across industries, companies and consumers have come to rely on the rapid exchange of digital information. Healthcare, however, has been an outlier in this regard. While the healthcare digital transformation has been in play for years, it’s …

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Aiming to treat Parkinson’s by replacing neurons, Aspen Neuroscience nabs $147M

Aspen Neurosciences is developing a cell therapy that uses a patient’s own stem cells to develop a personalized treatment for Parkinson’s disease. The approach is slightly different than that of Bayer, whose experimental Parkinson’s cell therapy is made from stem cells sourced from healthy donors.

How cell & gene therapy startups can warm up to investors in this “biotech nuclear winter”

Cell and gene therapy research has been hot the past few years but the overall market has cooled down in recent months. During the World Medical Innovation Forum in Boston, a panel of venture capitalists discussed capital formation and what biotech firms need to do to win over investors and advance their research.

Regulatory recap: J&J vaccine limits, full Enhertu approval, a Vertex hold & more

The FDA amended emergency authorization of Johnson & Johnson’s Covid-19 vaccine. In other regulatory news this past week, drug approvals include a cancer drug that converted its accelerated regulatory approval to a full regulatory nod, while several companies dealt with rejections or clinical trial setbacks.

PepGen IPO nabs $108M for muscular dystrophy drug with potential edge over Sarepta med

PepGen’s technology improves the delivery of a therapy to more tissue types and the clinical-stage biotech plans to use its IPO cash to continue developing its lead program for Duchenne muscular dystrophy. Meanwhile, eye products giant Bausch + Lomb returned to the public markets as a standalone company. Both the PepGen and Bausch + Lomb …

PepGen IPO nabs $108M for muscular dystrophy drug with potential edge over Sarepta med Read More »

Are you a startup seeking to transform clinical trials? Here’s why you should apply to pitch at INVEST PharmaTech.

INVEST PharmaTech Pitch Perfect virtual conference July 26 will give startups the opportunity to present their approaches to clinical trial innovation to a team of investor judges. Apply today!

Curebase bags $40m to develop patient-centric trials platform

Curebase has raised $40 million in second-round financing that will help it run more complex interventional trials for its biopharma clients, according to chief executive Tom Lemberg. The Series B takes the total raised by Curebase to $59 million since it was set up five years ago to develop a patient-focused approach to clinical research, …

Curebase bags $40m to develop patient-centric trials platform Read More »

4 Reasons Why You Shouldn’t Ignore CDISC Data Standards

A quick recap: Who are CDISC and what do they do? The Clinical Data Interchange Standards Consortium (CDISC) is a non-profit organization that aims to maximise the impact of clinical research data by establishing and promoting the use of data standards. Basically, these standards make clinical data easier to understand and interpret. “We develop and advance data …

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BMS shows what’s NEX-T for CAR T, plus its strategy for next-gen cancer cell therapy

As an oncologist, Kristen Hege first encountered cancer cell therapy research in the mid-1990s. Now as a Bristol Myers Squibb executive, she oversees efforts to improve the pharma giant’s first generation of cell therapies while also building a pipeline of next-generation treatments with better features and properties.

How the Life Sciences Industry Can Boost Diversity and Inclusion Efforts

Although it is well established that diversity improves outcomes and drives excellence, implementing diversity lags behind in many industries, including life sciences and healthcare.  Diversity is lacking across the entire field, from academic research to clinical work, composition of the workforce, and medical practice. This leads to worse health outcomes and fewer professional opportunities for …

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HilleVax’s IPO hauls in $200M for clinical test of VLP norovirus vaccine from Takeda

Takeda Pharmaceutical spinout HilleVax broke the biotech IPO drought with a $200 million stock market debut for clinical development of a norovirus vaccine. Two eye health companies marked the other IPO activity: Belite Bio went public while Bausch + Lomb set financial terms for what could become one of the biggest IPOs of the year.

Ceryx Medical CEO said its technology could potentially cure heart failure, startup gets funding for first human trials

Ceryx’s device, Cysoni, is designed to pace the heart in rhythm with a person’s respiration, so that the heart rate and breathing are synced. This is typically the case for a healthy person, but for someone with heart failure, that link becomes severed, CEO Stuart Plant said.

Potential blockbuster BMS cardio drug gets FDA nod, first in rare heart disease

Bristol Myers Squibb drug Camzyos has FDA approval for treating obstructive hypertrophic cardiomyopathy, a rare and potentially fatal heart disorder. The drug is projected to become a blockbuster seller and its approval marks a payoff for BMS’s 2020 acquisition of the medicine’s developer, MyoKardia.

3 Keys to Eliminating Data Breakdowns in Life Sciences

Sudish Mogul, Chief Technology Officer of Healthcare Triangle Across life sciences, experts boldly predict that data is the new currency—and that mobilizing data from clinical trials, wearable devices and even social media will ignite innovation in 2022. But leveraging data for innovation depends not just on data analytics, but also access to the right data …

3 Keys to Eliminating Data Breakdowns in Life Sciences Read More »

Biohaven migraine drug partnered with Pfizer lands twin European approvals

The European Commission approved Biohaven Pharmaceuticals migraine drug rimegepant. While the regulatory decision marks another milestone for Biohaven, it’s also a win for Pfizer, which holds rights to commercialize the drug outside of the U.S.

3 Predictions for Post-COVID Clinical Trial Decentralization

Marc Leighton, VP of Product at Florence Healthcare Prior to COVID, major technological transformation initiatives were already underway at many clinical trial sites, sponsors, and contract research organizations (CROs.) But the move to decentralized trials accelerated dramatically in 2020 and 2021–and it’s not slowing down.  Survey data from WCG Avoca shows that research sites and …

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Biofourmis Secures $300M to Expand Virtual Care Offerings

Kuldeep Singh Rajput, Founder & Chief Executive Officer of Biofourmis What You Should Know: – Biofourmis, a global leader in virtual care and digital medicine raises $300 million in Series D investment led by leading global growth equity firm General Atlantic. CVS Health and existing investors also participated in the round, – Biofourmis also announced that …

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Nkarta’s natural killers flash potential as next-gen cell therapies for cancer

Two natural killer (NK)cell therapy candidates from Nkarta now have preliminary Phase 1 clinical data suggesting these therapies are helping patients in two types of blood cancers. Nkarta therapies based on engineered NK cells could offer both efficacy and safety advantages over the first generations of cell therapies, which are made from a patient’s own …

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Roche breast cancer drug fails in Phase 2 following similar stumble by rival Sanofi

Experimental Roche drug giredestrant failed to meet the main goal of a Phase 2 study in advanced breast cancer. The disappointing results allow competitors to gain ground in the field of companies developing oral selective estrogen receptor degraders (SERDs), drugs that block a receptor key to driving breast cancer growth.

Covid-19 vaccines & therapies helped push medicines spending to $407B in 2021

While Covid-19 has had a significant impact on drug spending, IQVIA projects that its effect will moderate in coming years. IQVIA’s new report on U.S. medicines spending projects that immunology, oncology, and neurology will continue to drive the growth in drug spending.

Biogen pulls Alzheimer’s drug application in Europe as EMA calls data insufficient

Biogen withdrew an application seeking approval in Europe for Alzheimer’s disease drug Aduhelm, a move that follows a negative opinion on the therapy issued by a European Medicines Commission committee last December. The company was unable to persuade European regulators to change their minds about the drug.

Financing pushes value of clinical trial tech firm Reify to $4.8bn

Reify Health has completed fourth-round financing that adds $220 million to its cash reserves and pushes its valuation up to a heady $4.8 billion, with plans to use the new cash to launch a new initiative to make sure clinical trials are inclusive and diverse. The need for diversity in clinical trial populations has been …

Financing pushes value of clinical trial tech firm Reify to $4.8bn Read More »

Reify Health Secures $220M to Support Clinical Trial Diversity at $4.8B Valuation

What You Should Know: – Reify Health, a leading provider of solutions that empower the clinical trial ecosystem, announced a $220M Series D funding round dedicated to improving diversity in clinical research. The investment comes less than a year after its Series C and brings the company’s valuation to $4.8B.  – The Series C found was co-led …

Reify Health Secures $220M to Support Clinical Trial Diversity at $4.8B Valuation Read More »

AstraZeneca’s long-acting Covid-19 drug keeps protecting high-risk patients at 6 months

AstraZeneca’s Covid-19 antibody drug Evusheld reduced the risk of infection by 83% after six months. These results published in the New England Journal of Medicine are the latest clinical data supporting the durability of Evusheld, which was designed to offer longer-lasting protection.

AI startup Unlearn adds $50M for better, faster, smaller & cheaper clinical trials

Unlearn.AI’s Series B round follows a positive European Medicines Agency draft opinion finding that the company’s artificial intelligence technology can be used in Phase 2 and Phase 3 clinical trials. The startup is now on the hunt for pharmaceutical partners that want to use the technology to speed up their clinical research.

Regeneron captures new piece for cancer with $250M Checkmate Pharma buyout

Regeneron Pharmaceuticals is paying $250 million cash to acquire Checkmate Pharmaceuticals, a clinical-stage biotech developing drugs that activate the innate immune system. The biotech’s lead drug candidate is currently in pivotal testing in melanoma.

Why Cheap, Older Drugs That Might Treat Covid Never Get Out of the Lab

In March 2020, Dr. Joseph Vinetz left the contemplative world of his Yale University infectious-disease laboratory and plunged into the covid ward at Yale New Haven Hospital, joining an army of health care workers who struggled to treat the deadly viral disease. There were no drugs against covid-19, and no way to predict which infected …

Why Cheap, Older Drugs That Might Treat Covid Never Get Out of the Lab Read More »

FDA’s tighter scrutiny on drug class leads TG Therapeutics to pull out of cancer

TG Therapeutics is pausing its cancer drug research and withdrawing the approved cancer drug Ukoniq from the market. The move comes as the FDA places greater scrutiny on Ukoniq’s drug class—concerns that will be discussed during an FDA advisory committee meeting scheduled for April 21.

Covid news roundup: Boosters for kids, breath diagnostic, a vax approval & more

Pfizer and BioNTech reported preliminary clinical data supporting use of their Covid-19 vaccine as a booster in children ages 5 to 11. In other Covid news, a vaccine developer won marketing authorization in Europe while another one faces a regulatory setback.

BMS and Nektar end cancer drug alliance after two more clinical trial failures

Bristol Myers Squibb and Nektar Therapeutics are ending a cancer drug research alliance after data showed the combination of their respective drugs failed clinical trials in kidney and bladder cancers. The disappointing results come one month after the drug combo failed a pivotal melanoma clinical trial.

Cell therapy biotech Aurion gets $120M to restore vision and revamp organ donor economics

Organ donation has long been a matter of demand outstripping supply, but Aurion Biotech aims to improve the supply part of the equation with a cell therapy. Aurion’s technology turns a single donated cornea into treatments for up to 100 patients and the startup now has $120 million for clinical trials.

FDA gives Gilead reprieve on partial hold for cancer drug studies

An FDA safety review has cleared Gilead Sciences to resume five clinical trials that were placed under a partial hold in January. The experimental Gilead drug, magrolimab, is part of a class of cancer immunotherapies that address a hot target for the field.

Microbiome biotech Kaleido Biosciences shutters amid cash crunch, drug setbacks

Kaleido Biosciences, formed by Flagship Pioneering as part of a wave of biotech companies developing microbiome therapies, has laid off its entire workforce and is winding down operations. The shutdown follows a strategic review that failed to turn up any deals to support its clinical-stage pipeline.

PathAI and GlaxoSmithKline Partner to Accelerate Research and Drug Development

What You Should Know: – PathAI and GSK announced a multi-year strategic artificial intelligence (AI) partnership today with a focus on accelerating research and- building drug development programs in oncology and non-alcoholic steatohepatitis (NASH). – As part of the partnership, GSK will leverage both PathAI’s expertise and models to advance GSK’s proprietary AI models and focus on designing better …

PathAI and GlaxoSmithKline Partner to Accelerate Research and Drug Development Read More »

Final CMS decision limits coverage of Biogen’s Aduhelm to those in clinical trials

Biogen’s Alzheimer’s disease drug Aduhelm will only be covered for Medicare beneficiaries participating in a clinical trial. The final coverage determination will have the effect of limiting use of the drug, but the agency said its decision provides clarity on what drug companies need to show in order to secure Medicare coverage.

Werewolf cancer drug strikes right note with Jazz Pharma, sparking licensing deal

Jazz Pharmaceuticals is licensing rights to a Werewolf Therapeutics cytokine therapy that’s on track to reach clinical testing next year. The deal gives Jazz a drug candidate that expands the company’s scope to immuno-oncology.

Viz.ai Raises $100M for Care Coordination Platform at $1.2B Valuation

What You Should Know: – Viz.ai, a San Francisco-based AI-powered disease detection and care coordination platform raises $1000M in Series D funding led by Tiger Global and Insight Partners. The round also included participation from Scale Ventures, Kleiner Perkins, Threshold, GV (formerly Google Ventures), Sozo Ventures, CRV, and Susa.  – The new funds will be used to support Viz.ai’s significant growth trajectory, …

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Pfizer broadens its RSV reach with deal for ReViral and its clinical-stage antivirals

Pfizer is already in the mix of companies pursuing vaccines for respiratory syncytial virus. The pharma giant is now broadening its scope with a deal to acquire ReViral Therapeutics, a biotech with two clinical-stage antivirals, each taking a different approach to stopping the potentially deadly pathogen.

Novartis cancer drug is now first approved therapy for group of rare, genetic diseases

A Novartis drug that stops abnormal cell growth in breast cancer now has an additional FDA approval treating a group of rare disorders characterized by overgrowth in various types of tissue. The regulatory decision makes the drug, Vijoice, the first approved therapy for these disorders termed “PROS.”

Imara’s two trial flops in blood disorders dim drug’s prospects in heart failure

The lead drug candidate of Imara failed two separate mid-stage clinical trials evaluating it in rare, inherited hemoglobin disorders. Imara had been planning to advance the small molecule to yet another Phase 2 study in heart failure, but in light of the twin trial failures, the biotech is now weighing “strategic options.”

Study uncovers new Alzheimer’s genes, links to immune disruption

The largest-ever study of genetics in Alzheimer’s disease patients has identified 42 new genes that appear to be linked to the neurodegenerative disorder. The new genes takes the total number associated with Alzheimer’s to 75, opening up new avenues for research of ways to diagnose and treat the conditions, and suggest that immunological dysfunction may …

Study uncovers new Alzheimer’s genes, links to immune disruption Read More »

Labcorp & Medidata to Co-Develop Digital Biomarkers & Expand Decentralized Clinical Trials

What You Should Know: - Medidata, a Dassault Systèmes company, today announced that Labcorp, a leading global life sciences company, has selected Medidata’s technology platform to extend their 14-year partnership, the foundation of their initiative to co-develop digital biomarkers and expand the use and functionality of decentralized clinical trials. Labcorp Drug Development has developed its offerings on Medidata technologies, starting with Rave EDC (electronic data capture), Coder and …

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Safety concerns sink Akebia drug for anemia from chronic kidney disease

The FDA rejected Akebia Therapeutics drug vadadustat as a treatment for anemia caused by chronic kidney disease. According to the biotech, the agency cited safety concerns and asked for another clinical trial to show that the pill’s benefits outweigh its risks.

Sano raises $11m for patient and trial matchmaking platform

UK startup Sano Genetics has raised $11 million in first-round financing to develop its go-between service, which aims to overcome one of the big challenges in clinical research by connecting patients with trial sponsors. The Cambridge-based company said the new funding – which adds to around $4 million in seed financing – will be used …

Sano raises $11m for patient and trial matchmaking platform Read More »

VivoSense Raises $25M for Wearable Sensor Biomarkers to Advance Clinical Trials

What You Should Know: – VivoSense, an agile end-to-end scientific solutions company developing novel digital endpoints from wearable sensor data raises $25 Million in Series A funding. – The financing will accelerate the development of novel digital biomarkers and improve digital clinical outcome assessments. VivoSense will also use this financing to expand and refine its informatics platform …

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Vying to best bigger rivals in Parkinson’s, Neuron23 nabs $100M to reach clinic

Neuron23 is developing drugs that treat neurological conditions by penetrating the blood-brain barrier to reach disease targets in the central nervous system. The biotech’s lead program is a Parkinson’s disease drug candidate with features that could distinguish it from rival compounds that are aiming for the same target.

New blow for ALS pipeline as Biogen and Ionis candidate fails Phase 1 trial

Less than six months after the late-stage failure of tofersen, Biogen and Ionis have suffered further disappointment in the amyotrophic lateral sclerosis (ALS) pipeline after BIIB078 (IONIS-C9Rx) failed to demonstrate clinical benefit in a Phase 1 trial. In the randomised, placebo-controlled, dose-escalating trial, researchers evaluated the efficacy of the investigational antisense oligonucleotide BIIB078 as a …

New blow for ALS pipeline as Biogen and Ionis candidate fails Phase 1 trial Read More »

Data and regulatory roundup: Pfizer’s feat, an ADHD miss, Eli Lilly’s CRL & more

A pharmaceutical giant reported a Phase 3 success for a potential blockbuster drug, while four other companies reported disappointing data from their respective clinical trials. Our recap of data readouts and regulatory news also includes one FDA rejection.

AN2 Therapeutics’ IPO raises $69M to take antibiotic from Pfizer into pivotal test

AN2 is developing an antibiotic based on boron chemistry that could offer advantages over carbon-based molecules. The biotech plans to advance its drug candidate, licensed from Pfizer, to a pivotal test in a rare and potentially fatal lung infection caused by mycobacteria.

COTA Promotes Miruna Sasu to President and CEO

Miruna Sasu, Ph.D., MBA, President & CEO at COTA, Inc. What You Should Know: – COTA, Inc., an oncology real-world data and analytics company, announced today that Miruna Sasu, Ph.D., MBA, has been appointed president and chief executive officer.  – Since April 2021 when she was hired as the company’s chief strategy officer, Miruna has …

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Tempus Launches Pathology-Driven AI Platform

What You Should Know: – Tempus, a provider of artificial intelligence and precision medicine, today announced the launch of Edge, an innovative platform that allows pathologists to access developing AI models intended to identify specimens with potentially actionable biomarkers using a single hematoxylin and eosin stain (H&E) slide. – With access to over 50 petabytes of de-identified …

Tempus Launches Pathology-Driven AI Platform Read More »

Applied XL Raises $3.5M for Real-Time Data Tracking Platform

What You Should Know: – Applied XL, a real-time information company tracking disruptions to the health of people, places, and the planet, today announced it had raised $3.5 million in seed funding led by Hearst Ventures with the participation of the Boston Globe Media Partners and STAT, which are owned by the Henry family. – …

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FDA approves Marinus Pharma drug for rare, genetic form of epilepsy

Marinus Pharmaceuticals drug Ztalmy is now FDA approved for treating the seizures in patients who have CDKL5 deficiency, an inherited form of epilepsy. The drug is the first approved treatment for the rare disorder and it’s also Marinus’s first approved product.

BMS nabs FDA nod for first drug in new class of cancer immunotherapies

Bristol Myers Squibb’s Opdualag has been approved by the FDA, a first for a drug that goes after a new cancer target called LAG-3. The drug is approved for advanced melanoma as part of a combination treatment that includes the already approved BMS cancer immunotherapy Opdivo.

Apellis Pharma eyes a shot at getting first FDA approval in rare vision disorder

Apellis Pharmaceuticals has additional clinical data that show its drug having an effect on geographic atrophy, a rare vision-loss disorder with no FDA-approved treatment. The biotech plans to include the latest results in a new drug application being readied for submission in coming months.

Day 3 HIMSS Roundup: Glooko Acquires DIABNEXT, GE Healthcare/AliveCor Integration

Venture Capital, Private Equity, Mergers & Acquisitions (M&A) Activity Glooko Acquires DIABNEXT® to Expand Diabetes Offerings in France Glooko, a leading provider of remote patient monitoring and data management solutions for diabetes and related chronic conditions, today announced the acquisition of DIABNEXT, a Paris-based company whose digital health platform helps people with diabetes better manage …

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Diabetes data put Better Therapeutics’ digital offering on track for FDA filing

Better Therapeutics reported that its digital therapeutic for type 2 diabetes met the main goal of a pivotal study. An FDA submission is expected in the middle of this year, followed by additional clinical trials testing digital therapeutics developed for other cardiometabolic disorders.

Nektar drug key to muti-billion dollar BMS deal fails pivotal test in melanoma

Bristol Myers Squibb bet more than $1.8 billion that Nektar Therapeutics’ drug could be combined with the pharma giant’s Opdivo to expand the reach of that drug to more patients. The drug combination has failed its first Phase 3 in advanced melanoma; separate studies in kidney and bladder cancers are ongoing.

Modest activity, tolerability issues sink lead Harpoon cancer drug prospect

Harpoon Therapeutics is discontinuing work on its lead drug in prostate cancer following disappointing data in an early-stage clinical trial. Though Harpoon has other drugs in its pipeline, the decision is a setback to the company’s efforts to develop a new type of cancer immunotherapy.

Alliance aims to drive inclusivity in trials backed by digital devices

A consortium led by the Digital Medicine Society (DiMe) has been set up to try to end inequities in access to clinical trials, focusing on the increasing number of studies that use digital devices to allow participation. The organisation has teamed up with drugmakers GlaxoSmithKline and Amgen as well as partners in the clinical research, …

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MorphoSys cuts R&D from Constellation deal to focus on clinical-stage cancer drugs

MorphoSys is cutting the drug discovery and R&D work of Constellation Pharmaceuticals, a company it acquired lats year in a $1.7 billion deal. This consolidation of research operations will result in non-cash impairment charge on Constellation’s goodwill.

Scenic Biotech scores $31M to take aim at genetic modifiers for cancer & rare disease

Targeting mutations isn’t the only way to treat genetically driven diseases. Scenic Biotech is developing molecules to target genes that suppress disease, and it has raised $31 million to advance its pipeline of potential therapies for cancer and rare diseases.

Which Companies Aren’t Exiting Russia? Big Pharma

Even as the war in Ukraine has prompted an exodus of international companies — from fast-food chains and oil producers to luxury retailers — from Russia, U.S. and global drug companies said they would continue manufacturing and selling their products there. Airlines, automakers, banks, and technology giants — at least 320 companies by one count …

Which Companies Aren’t Exiting Russia? Big Pharma Read More »

Sanofi’s hemophilia A data set stage for regulatory filings, matchup with Roche

An experimental hemophilia A therapy from Sanofi and partner Sobi has met the goals of a pivotal study, and the pharmaceutical giant is now planning global regulatory submissions for later this year. The drug could compete against a blockbuster hemophilia A treatment marketed by Roche.

With lung drug from Pfizer in hand, AN2 tests IPO waters in turbulent market

Two biotechs are braving the roiling financial markets to raise money for clinical trials. AN2 Therapeutics is preparing for a pivotal test of its boron-based drug candidate for a rare, chronic lung disease. Okyo Pharma also filed IPO paperwork as it looks to advance to the clinic with its dry eye disease drug.

Alpine Immune Sciences cancer drug trial placed on partial hold after patient death

The FDA placed a partial clinical hold on a Phase 1 study testing experimental Alpine Immune Sciences cancer drug davoceticept in combination with Merck cancer immunotherapy Keytruda. The partial hold does not affect a separate study testing the Alpine drug as a monotherapy.

Patients Divided Over Alzheimer’s Drug: Is It a ‘Risk I’m Willing to Take’ or Just a ‘Magic Pill’?

If you listen to the nation’s largest Alzheimer’s disease advocacy organizations, you might think everyone living with Alzheimer’s wants unfettered access to Aduhelm, a controversial new treatment. But you’d be wrong. Opinions about Aduhelm (also known as aducanumab) in the dementia community are diverse, ranging from “we want the government to cover this drug” to …

Patients Divided Over Alzheimer’s Drug: Is It a ‘Risk I’m Willing to Take’ or Just a ‘Magic Pill’? Read More »

Black Female Co-founders CancerIQ Raises $14M to Improve Cancer Early Detection and Prevention

CancerIQ Co-Founders Feyi Olopade Ayodele & Olufunmilayo (Funmi) Olopade, MD What You Should Know: – CancerIQ, a Chicago-based platform powering early cancer detection and prevention across broad patient populations, announced today it closed a $14M Series B financing round. – The Series B round was co-led by Merck Global Health Innovation Fund (Merck GHI) and Amgen Ventures …

Black Female Co-founders CancerIQ Raises $14M to Improve Cancer Early Detection and Prevention Read More »

How Support Automation Enhances Clinical Trial Management

David Karandish, CEO of Capacity The life of a clinical study relies on data from documentation, meetings, emails and calls; all of which can be overwhelming for patients, clinical trial teams and associates. Although mundane, documenting, executing and collecting data is crucial to move a trial from phase to phase. Clinical trial teams face a …

How Support Automation Enhances Clinical Trial Management Read More »

One year on: How is the UK’s vision of a future-proof research environment shaping up?

One year ago, a UK-wide vision for the country’s future research landscape promised streamlined processes, and a patient-centred, competitive environment. But how far have we come in achieving those aims? And what’s next on the system’s agenda? From Brexit and COVID-19 to the wholescale shift in the way research is designed, conducted, and reported, the …

One year on: How is the UK’s vision of a future-proof research environment shaping up? Read More »

How Support Automation Enhances Clinical Trial Management

David Karandish, CEO of Capacity The life of a clinical study relies on data from documentation, meetings, emails and calls; all of which can be overwhelming for patients, clinical trial teams and associates. Although mundane, documenting, executing and collecting data is crucial to move a trial from phase to phase. Clinical trial teams face a …

How Support Automation Enhances Clinical Trial Management Read More »

AbbVie makes neuro drug connection with $130M Syndesi Therapeutics acquisition

AbbVie has acquired Syndesi Therapeutics, an early clinical neuroscience startup developing drugs that have potential applications in a range of cognitive disorders. Syndesi is based on research from Belgian pharmaceutical company UCB.

FDA rejects Reata Pharma chronic kidney disease drug; new trial needed

The FDA told Reata that the clinical data do not show that the drug, bardoxolone, slows the progression of the loss of kidney function in patients who have the rare disease Alport syndrome. The regulatory decision follows an FDA advisory committee vote recommending against approval of the small molecule drug.

Biohaven pays $100M for epilepsy drug prospect; strikes deal for BMS muscle med

Neuroscience drug developer Biohaven Pharmaceuticals is expanding its pipeline to include epilepsy and spinal muscular atrophy. The drug developer is acquiring Channel Bioscience and licensing global rights to a Phase 3-ready drug from Bristol Myers Squibb.

Insilico’s AI-discovered, AI-designed IPF drug enters Phase 1 trials

Hong Kong-based Insilico Medicine has begun a Phase 1 trial of its AI-discovered and AI-designed anti-fibrotic small molecule inhibitor.  Currently referred to as ISM001-055, the drug is meant to treat ideopathic pulmonary fibrosis using a relatively novel mechanism of action. Insilico announced in February of last year that they had identified and developed the molecule …

Insilico’s AI-discovered, AI-designed IPF drug enters Phase 1 trials Read More »

Kodiak Sciences’ trial failure clouds eye drug’s prospects for challenging Eylea

A Kodiak Sciences drug failed to beat a Regeneron Pharmaceuticals product in a key test in the wet form of age-related macular degeneration. The biotech says the failure is due to a subset of undertreated patients, but the results raise the stakes for the drug, its lead product candidate, in other eye diseases.

Sanofi, GSK Covid-19 vax is now ready for regulators; its future may be as a booster

Sanofi and GlaxoSmithKline weathered clinical trial delays for their Covid-19 vaccine, but the partners now have data to support filings seeking regulatory authorizations. Key features of the vaccine may be able to persuade the vaccine hesitant; it may also be well-suited for use as a booster.

Helping Clinical Trial Sites Improve Study Recruitment and Retention

Tzvia Bader, CEO and co-founder, TrialJectory The clinical trial industry is, once again, at an uncertain crossroads. And while it might be assumed that the outsized impact of the COVID-19 pandemic has resulted in the inability to recruit patients for trials, it is instead, a challenge generated, over time, by the industry itself. Thankfully, there …

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EarliTec Diagnostics Raises $19.5M to Advance Autism Diagnosis and Treatment in Children

What You Should Know: – EarliTec Dx, a digital health company developing novel diagnostic and therapeutic products for children with Autism Spectrum Disorder (ASD) and early childhood vulnerabilities, today announced a $19.5 million financing led by Bernie Marcus, with participation from the Georgia Research Alliance (GRA). – This round of financing will be used to …

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Agios strategy shift pays off with FDA approval of first drug for rare anemia

Agios Pharmaceuticals has its third FDA-approved product, and the first under its new rare disease strategy. The regulatory decision also marks the first approved treatment for pyruvate kinase deficiency, a rare disease that leads to chronic anemia.

Cash woes at neuro biotech Yumanity lead to 60% staff cut and search for a buyer

Yumanity’s corporate restructuring means the biotech is now exploring “strategic alternatives” that could include a sale of the company or its assets. The biotech’s lead drug candidate is an experimental Parkinson’s disease drug currently in Phase 1 testing.

H1 Acquires London-Based Faculty Opinions to Broaden Global Doctor Network

What You Should Know: H1, a global healthcare professional data ecosystem to enable life sciences, academic medical institutions, health systems, and payors acquires London-based Faculty Opinions Ltd, formerly known as Faculty of 1000 and F1000 Prime.  Financial details of the acquisition were not disclosed. – A close partner of H1, Faculty Opinions fuses the comments …

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Backed by $100M, Star Therapeutics sets out to find constellations of rare disease

Star Therapeutics aims to develop drugs for rare diseases, many of which currently have no FDA-approved treatments. The company forms startups focused on particular areas of biology, and its first one, Electra Therapeutics, is going after a rare autoimmune condition that can become fatal in less than four years.

Roivant Sciences launches Hemavant to chase BMS in anemia from blood disease

An Eisai drug that produced disappointing data in a myelodysplastic syndrome study is getting another shot under a newly launched Roivant Sciences subsidiary, Hemavant. Roivant executives say the small molecule could be a safer, more effective alternative to MDS therapies marketed by Bristol Myers Squibb.

The Next Vaccine Debate: Immunize Young Children Now, or Wait?

It’s not clear whether three doses of the Pfizer-BioNTech vaccine will adequately protect young children. But the F.D.A. may authorize the first two doses anyway.

Outlook dims for ProQR’s RNA therapy for vision loss following failure in key test

The lead RNA therapy from ProQR Therapeutics has failed the main and secondary goals of a pivotal study testing it as a treatment for a rare, inherited vision disorder. Despite the disappointing preliminary data, the biotech said it remains confident in the technology that produced the experimental therapy, sepofarsen, and the Phase 2/3 study will …

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¿Vacunar a los niños pequeños o esperar?

No está claro si tres dosis de la vacuna Pfizer-BioNTech protegerán adecuadamente a los niños. Pero es posible que la FDA autorice las primeras dos dosis de cualquier manera.

¿Vacunar a los niños pequeños o esperar?

No está claro si tres dosis de la vacuna Pfizer-BioNTech protegerán adecuadamente a los niños. Pero es posible que la FDA autorice las primeras dos dosis de cualquier manera.

FDA Panel Rejects Lilly’s Cancer Drug Tested Only in China

The panel debated whether overseas trials could be applied to a more diverse U.S. population. The decision may affect other Chinese drug trials, and spotlights the high cost of immunotherapy.

FDA Panel Rejects Lilly’s Cancer Drug Tested Only in China

The panel debated whether overseas trials could be applied to a more diverse U.S. population. The decision may affect other Chinese drug trials, and spotlights the high cost of immunotherapy.

The Protein Degradation & Targeting Undruggables Congress

The 3rd Protein Degradation and Targeting Undruggables Summit is North America’s only in-person, industry-led conference covering clinical progress and commercial scale development of first-in-class degrader technology platforms. Our agenda will cover the key drug development challenges for this therapeutic modality including assessing diverse targets, improving selectivity and delivery to disease-relevant proteins. You will have the …

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CVS, Medable alliance aims to tackle diversity in clinical trials

CVS Health has said it will beef up its clinical trials operations, improving its ability to recruit patients from a broad demographic spectrum, via an agreement with digital health company Medable. CVS will harness Medable’s decentralised trials software, deploying it in thousands of CVS  MinuteClinics across the US, to create a network that could unlock …

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The Next Vaccine Debate: Immunize Young Children Now, or Wait?

It’s not clear whether three doses of the Pfizer-BioNTech vaccine will adequately protect young children. But the F.D.A. may authorize the first two doses anyway.

Medable Launches Decentralized Clinical Trials Partner Network

What You Should Know: – Medable Inc., a Palo Alto, CA-based provider for patient-centered clinical trials, today launched the Medable Partner Network – uniting a diverse ecosystem of technology, service, data, site, and direct-to-patient partners that work together to accelerate deployment of decentralized clinical trials. – The network includes new partnerships with Advanced Clinical, Cognizant, and CVS, …

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Importance of diversity in pharma and clinical trials

“Diversity.” It’s a word that is very commonly thrown around right now, not just in pharma and healthcare but across all industries. No matter which sector you work in, diversity and inclusion should not be treated as mere buzzwords designed to help improve the perception of a company, but as must-haves. We live in a …

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UBC, Seqster join forces on patient-centric, remote trials

The rise in the use of decentralised clinical trial (DCTs) designs forced by COVID-19 looks set to outlast the pandemic, and digital health company Seqster has formed another partnership to tap into the shift from traditional site-based studies. The latest agreement is with contract research organisation United BioSource (UBC), which will use Seqster’s expertise in …

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Endeavor Bio adds $101M to test ex-Eli Lilly, Roche drug in cancer & lung fibrosis

Endeavor BioMedicines aims to kill two birds with one stone—that stone being a small molecule drug. Taladegib targets a cell signaling pathway associated with both cancer and idiopathic pulmonary fibrosis, and the biotech will apply its Series B round toward clinical tests of the molecule on both fronts.

2 collaborations – CVS Health with Medable, and Thermo Fisher Scientific with Medidata Acorn AI – seek to improve clinical trials

CVS MinuteClinic providers will use Medable’s software platform to improve patient access, engagement and retention during clinical trials. Meanwhile, Thermo Fisher Scientific and Medidata Acorn AI announced a collaboration to develop an application that combines patient recruitment and site performance data for clinical trials.

CVS Health Taps Medable to Bring Decentralized Clinical Trials to Pharmacies Nationwide

What You Should Know: – Medable and CVS Health announced a strategic collaboration that will make clinical research more accessible to patients of all demographics. Using Medable’s decentralized trial platform available to CVS Health’s thousands of community MinuteClinics nationwide, most people will be within just five minutes of a decentralized clinical trial research site – …

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Clarify Health’s new offering shows one way tech can help improve clinical trial diversity

The last three years in pharma have seen a growing awareness of and dissatisfaction with a trend that has plagued the industry for its whole life: Nondiverse and unrepresentative clinical trials, which lead to incomplete data and compounded health disparity for minority populations. It’s been more than a year since the US Food and Drug …

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Bridging the pharma-startup gap with PharmStars, a new accelerator programme

From remote monitoring tech to enable clinical trials, to AI for drug discovery, to digital therapeutics and drugs with companion apps, digital innovation has the potential to touch every corner of the pharmaceutical sector. The startups know it. Pharma knows it. Yet, somehow, it’s not always easy to make the connection. The world of pharma …

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Arcellx’s IPO raises $124M to fuel chase of better CAR T-cell therapy for cancer

Clinical-stage Arcellx raised $123.8 million from its IPO, which the company will use to advance to a pivotal test for its lead program, a CAR T-cell therapy for multiple myeloma. Though Arcellx trails its large pharmaceutical rivals, the biotech contends its technology produces cell therapies with key advantages.

Unity Bio slashes headcount in half to save cash ahead of eye drug readouts

Unity Biotechnology’s corporate restructuring is a cash-saving move that allows the company to focus resources on its lead program, a drug in Phase 2 testing for diabetic macular edema and wet age-related macular degeneration. Unity previously restructured in 2020 following the Phase 2 failure of its osteoarthritis drug.

Bayer-led project on AI in trials eyes ‘virtual’ placebo groups

Last year, Bayer launched a project to explore the use of artificial intelligence in clinical trials, working with Finland’s Aalto University to find ways to use the technology to support decentralised trials and streamline the testing process. Now, an extension of the work is starting that will see if AI can be used to reduce …

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Assumptions to evidence: How NINR project is solidifying UK’s remote trial know how

Remote, virtual, or decentralised clinical trials are nothing new, but COVID-19 catapulted the model into the mainstream. Now, as the sector settles back into something approaching business as usual, how can we ensure new methodologies are delivering for participants, sites, and the sector alike? As the COVID-19 pandemic ricocheted around the world at the start …

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MHRA seeks comments on post-Brexit clinical trial legislation

The MHRA has kicked off an eight-week consultation on proposals for changes to laws governing the conduct of clinical trials following the UK’s departure from the EU. The proposals stem from the passage of the Medicines and Medical Devices Act 2021 a year ago, shortly after the post-Brexit transition period ended, which has given the …

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Philips Launches First At-Home, 12-Lead ECG Integrated Solution for Decentralized Clinical Trials

What You Should Know: – Philips introduced the industry’s first full-service, at-home, 12-lead electrocardiogram (ECG) solution for use in decentralized clinical trials. – The solution is the most advanced patient-centric ECG offering within the company’s cardiac monitoring portfolio, pairing data readings comparable to clinical, site-based ECGs with Philips leading cloud-based data collection and analysis services. …

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Gilead Sciences cancer drug hit with partial hold due to adverse event concern

Gilead Sciences did not disclose details about the safety concern that led to an FDA partial clinical hold on five studies evaluating its cancer drug, magrolimab. But this experimental cancer immunotherapy introduces a safety risk that’s a known problem for all drugs in this class.

Synthetic Control Arms – ambition to action

Clinical trials are involved, expensive, and have been done largely the same way for decades but with today’s technology all that can change. With drawn-out timelines and billions in investments, traditional clinical trial methods are increasingly a barrier to cost-efficient and timely drug development. Synthetic data is addressing this head-on by harnessing historical clinical trials …

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Sierra succeeds where Gilead missed; FDA filing now planned for blood cancer drug

Sierra Oncology myelofibrosis drug momelotinib achieved the main and secondary goals of a pivotal test and the biotech now plans to seek FDA approval. Sierra acquired momelotinib from Gilead Sciences after that company stopped work on the drug due to mixed results in Phase 3 testing.

What is inflation’s impact on clinical trials?

Basic organic chemicals used for the preparation of new medications cost over 54.8% more than they did 12 months ago. Further, with schools and daycare facilities either going fully remote and/or closed, many parents and caregivers had to stay at home, eliminating their availability for study participation.