Archive


Category: R&D

  • Paul Stoffels swiftly resurfaces as Galapagos’ next CEO

    Paul Stoffels’ retirement at the end of last year after nine years as head of R&D at Johnson & Johnson was short lived – he’s just been named chef executive of Galapagos. Stoffels will take the helm of the troubled Belgian biotech on 1 April, when Galapagos’ long-serving CEO and co-founder Onno van de Stolpe […]

  • Moderna starts dosing patients with its Omicron COVID jab

    Moderna has started human trials of an Omicron version of its COVID-19 vaccine SpikeVax, just in case a modified version will be needed to boost protection against the new variant later this year. The biotech has also reported new data suggesting that may well be the case, with a six-fold reduction in neutralising antibody levels […]

  • US vs EU: How can Europe translate home-grown innovation into biotech success?

    We have seen a boom in biotech initial public offerings over the last two years, but many of Europe’s offerings have migrated to the United States. So, what can we do to keep home-grown science on European soil? From the Pfizer/BioNTech and Oxford/AstraZeneca vaccines to GSK’s lifesaving antibody treatments, European innovation has played a crucial […]

  • 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 […]

  • Pfizer starts dosing patients with its Omicron COVID shot

    Pfizer and partner BioNTech have started their clinical trial of a new version of their COVID-19 vaccine that has been specifically designed to protect against the Omicron variant. The trial in 1,420 adult patients aged 18 to 55 will include dosing with the partners’ existing Comirnaty mRNA-based vaccine with the Omicron-based jab in a mix-and-match […]

  • PolyPhotonix raises $10m to trial sleep mask for diabetic eye disease

    The UK company behind a sleep mask that aims to treat sight-robbing complications of diabetes has secured $10 million in funding that will be used to run trials of the medical device. PolyPhotonix secured the money in a first-round financing led by Prevail InfoWorks, a company specialising in management of clinical trial data, with the […]

  • Team behind RECOVERY trial of COVID drugs casts its net wider

    The UK scientists that ran one of the largest trials of experimental COVID-19 drugs have formed a non-profit company that will apply the methodology to other disease areas – with $6.8 million in funding from French drugmaker Sanofi. The non-profit – called Protas – is led by Sir Martin Landray,  professor of medicine and epidemiology […]

  • Chronic kidney disease: treating a silent killer

    In November, NICE backed NHS use of AstraZeneca’s diabetes and heart failure drug Forxiga for the treatment of chronic kidney disease. pharmaphorum caught up with AZ’s Joris Silon to discuss unmet needs in the condition. “Many times, I have talked to nephrologists who take care of patients with chronic kidney disease in the later stages, […]

  • Covid vaccine booster raises antibody levels in fight against Omicron

    A third dose of vaccine has been shown to effectively neutralise the Omicron variant of covid-19, according to researchers. Research led by UCLH, UCL and the Francis Crick Institute found that the booster dose successfully stimulates antibody levels that neutralise Omicron. Details of the study were published in The Lancet this week. UCLH researchers stated that antibodies […]

  • Biliary tract cancer drug granted orphan drug designation in US

    The US regulator has granted biliary tract cancer therapy Silmitasertib orphan drug designation. There are currently no treatments for this rare cancer, and the US food and Drug Administration (FDA) hopes that pharmaceutical firm Senhwa’s drug will meet the significant need for new biliary tract cancer options. First-line standard of care is currently a chemotherapy […]

  • BioTuring enters collaboration to enable detailed single-cell RNA research

    Research into single-cell sequencing has been boosted with Single Cell Discoveries and BioTuring joining forces to refine the process. The collaboration aims to “close the gap between single-cell RNA sequencing wet-lab services and single-cell data analysis solutions,” which will allow scientists to study the finest details that can be hard to access. A rapidly growing […]

  • Billionaire-backed “rejuvenation” start-up Altos Labs launches operations

    Just months after the venture was unveiled, controversial biotechnology company Altos Labs has officially launched with $3 billion in funding secured from investors. Dubbed an ‘anti-ageing’ company, Altos has gained mainstream attention for its focus on ‘rejuvenation’ and biological reprogramming technology. While the specific research goals have been kept a closely guarded secret, some scientists […]

  • Digital health companies enabling R&D topped 2021 US investment, Rock Health says

    According to Rock Health’s annual digital health funding report, US-based digital health companies enabling life sciences research and development raised $5.8 billion (£4.3 billion) in 2021, making it the top funded category of the sector. This is the first time R&D has topped the chart since Rock Health began tracking US digital health funding in […]

  • CVS Health and Microsoft alliance: an example of an extended partnership

    Microsoft and CVS Health have announced a new strategic alliance focusing on personalised care and accelerating digital transformation for the benefit of CVS customers and its employees. David Rhew, MD, global chief medical officer and VP of healthcare at Microsoft, tells us, “Using Microsoft technology, CVS Health will develop a more agile, digital-first technology foundation.” […]

  • Almirall and IRB Barcelona team up to tackle skin disease

    Two Spanish pharma companies are joining forces to identify new oral treatments for immune-inflammatory skin diseases with high unmet medical needs. Almirall and IRB Barcelona (the Institute for Research in Biomedicine), will conduct research using molecular glue degraders, a new approach to the skin diseases under scrutiny. The use of monovalent degraders, which enable the […]

  • Clinical trial begins of lung cancer immunotherapeutic

    Cancer Research UK’s Centre for Drug Development, working with the Ludwig Institute for Cancer Research and Vaccitech, has begun a phase I/IIa clinical trial of a new lung cancer vaccine. The novel immunotherapeutic, VTP-600, is not a preventative vaccine — it’s intended for patients who have recently been diagnosed with non-small cell lung cancer. The […]

  • Pre-Filled Syringes East Coast 2022

    SMi’s 9th Annual Conference Pre-Filled Syringes East Coast Main Conference: April 25 – 26, 2022 | Workshops: April 27, 2022 Sheraton Boston Hotel, Boston, MA, USA www.pfsamericas.com/pharmaphorum Sponsors are: SCHOTT, ZEON, Owen Mumford Pharmaceutical Services, PHC Corporation, POLYPLASTICS- TOPAS, Weiss-Aug Group, Baumann Medical, BD Medical, West Pharmaceutical Services Device innovations, connected delivery and regulatory guidance for […]

  • Decentralised trials: beyond the pandemic

    The adoption of decentralised activities was a major factor in allowing clinical trials to continue during the early stages of the pandemic. Ben Hargreaves examines whether this adoption looks set to become a permanent feature of clinical trials moving into the future, rather than just supplying the tools to a temporary need. Decentralised clinical trials […]

  • Novo Nordisk moves to strengthen obesity efforts

    After securing FDA approval for Wegovy last year, Novo Nordisk is strengthening its position in the obesity space through a collaboration with EraCal Therapeutics. Under the joint research plan, the Danish giant will work with EraCal to identify novel drug targets relevant for food intake regulation and additional metabolic phenotypes. Although specific details of the […]

  • Sanofi places $75m bet with ABL on popular Parkinson’s drug target

    Sanofi has joined a growing list of drugmakers going after alpha-synuclein targeting drugs for Parkinson’s disease, licensing a bispecific antibody from South Korea’s ABL Bio in a deal that could be worth more than $1 billion. The French pharma group is paying $75 million upfront for the preclinical-stage drug, codenamed ABL301, a double headed antibody […]

  • Daiichi Sankyo says Plexxikon R&D unit will close down

    Just over a decade after being acquired by Daiichi Sankyo, US biotech Plexxikon will be shuttered at the end of March, with its R&D projects transferred to other areas of the Japanese group. It’s the end of the road for the South San Francisco-based company, which became part of the Daiichi Sankyo in a $935 […]

  • Aldeyra builds case for troubled dry eye drug reproxalap

    Prospects for Aldeyra’s dry eye disease candidate reproxalap were hit last month after the drug failed to hit its primary objective in a phase 3 trial, but the company isn’t giving up, and has new data showing an improvement over Novartis rival therapy Xiidra. Patients treated with reproxalap reported significantly less eye discomfort and itching […]

  • Sarepta says early filing for DMD gene therapy may be back on

    The development of gene therapies for Duchenne muscular dystrophy has proved to be challenging, but one of the key players – Sarepta – thinks it may have the clinical data needed to file for regulator approval. The new data from the mid-stage Study 102 of SRP-9001 (delandistrogene moxeparvovec) comes from 21 patients who were initially […]

  • What HCPs think about mandatory vaccination

    What HCPs think about mandatory vaccination The practice of making vaccinations mandatory within a population has always been controversial. Historically, the first example of this was during the smallpox epidemic in the 1800s, when some European countries attempted to make vaccination against the disease compulsory. While it is hard to argue against the overall aim […]

  • Bayer trumpets $1bn CRISPR deal with Mammoth Bio

    Bayer has bolstered its cell and gene therapy platform by securing access to a CRISPR-based gene-editing platform developed by US biotech Mammoth Biosciences. The German group is paying $40 million upfront to kick off the alliance, which is focusing on the development of up to five CRISPR drugs that can be delivered in vivo for […]

  • After trial win, Novartis nabs COVID drug from Molecular Partners

    There are still licensing deals to be done in the COVID-19 category it seems, after Novartis paid around $163 million to license rights to an antiviral drug developed by Molecular Partners. The opt-in has been prompted by the results of a phase 2 trial of ensovibep (MPO420), which showed that a single intravenous dose of […]

  • Beyond the pandemic: the potential of mRNA technology

    The arrival of COVID-19 brought precious few positives, but one that emerged for the pharmaceutical industry was the emergence and validation of mRNA vaccine technology to prevent disease. Ben Hargreaves takes a look at how the space is currently progressing and in what direction it could be heading in future. The pandemic brought about an […]

  • Another big AI deal as Sanofi taps Exscientia for 15 new drugs

    If the last couple of days is an indication, 2022 is already shaping up to be the year of big-ticket alliances between big pharma companies and specialists in artificial intelligence-driven discovery. Shortly after Amgen unveiled its $1.9 billion alliance with Generate Biomedicines aimed initially at five drug molecules, Sanofi has announced a big expansion of […]

  • Putting patient perspectives at the heart of kidney cancer research

    Kidney cancer is the 14th most common cancer, with approximately 400,000 new cases worldwide every year. Renal cell carcinoma (RCC) is by far the most prevalent type of cancer originating from the kidney, making up nine out of ten kidney cancer diagnoses. As chief medical officer, head of global medical affairs, patient safety and patient […]

  • AstraZeneca goes bigger in amyloidosis with Neurimmune deal

    AstraZeneca has clearly made the rare disease transthyretin amyloidosis (ATTR) a key component of its rare disease pipeline, licensing a second drug candidate in the space of a couple of months. In the latest deal, the pharma group is paying $30 million upfront with another $730 million in milestone’s for global rights to Neurimmune’s NI006, […]

  • Amgen pledges $1.9bn to alliance with AI specialist Generate

    Fresh from a sizeable funding round that raised $370 million, artificial intelligence-based drug discovery firm Generate Biomedicines has just signed its first major partnering deal with Amgen. The machine learning specialist – founded by venture capital firm Flagship Pioneering in 2018 – is getting $50 million upfront to kick off the alliance, with another £370 […]

  • Otsuka dips its toe in psychedelic waters, backing Mindset Pharma

    Despite the growing interest in developing medicines based on psychedelic compounds, big pharma has seemed a bit wary of getting involved, leaving the task to small start-ups. Now, Japan’s Otsuka Pharmaceutical has become one of the first to gather a position in the sector via a small deal with Mindset Pharma that will provide funding […]

  • PureTech challenges Roche’s Esbriet in head-to-head IPF trial

    Roche’s idiopathic pulmonary fibrosis (IPF) therapy Esbriet is already being squeezed by its main rival in the market, and could come under renewed pressure if another competitor from PureTech Health gets approved. PureTech is developing a modified version of the active ingredient in Esbriet (pirfenidone) that has just been shown to be less likely to […]

  • Nine for 2022: innovation and opportunities in healthcare

    In the second part of our examination of issues that will shape the course of 2022, we’ll update the ongoing impacts of the pandemic, which have, as they did in 2021, a pervasive effect on both pharma and healthcare issues, with themes we highlighted at the beginning of 2021 continuing their impact. Big Tech makes […]

  • Look out GSK, Pfizer and BioNTech are coming for Shingrix

    GlaxoSmithKline places shingles vaccine Shingrix is among its top prospects, with peak sales potential of almost $6 billion. Now, Pfizer and BioNTech are joining forces to try to grab a slice of the market. Having already brought their mRNA-based CVID-19 vaccine Comirnaty to market at breakneck speed, Pfizer and BioNTech are hoping to fast-track a […]

  • Gritstone bio trumpets first data for ‘multivariant’ COVID jab

    A COVID-19 vaccine that could work against multiple variants of the coronavirus – developed by US biotech Gritstone bio – has generated encouraging immune response data in its first clinical trial. The new vaccine differs from the currently approved shots because it delivers antigens for both the spike protein and other proteins found in SARS-CoV-2. […]

  • Nine for 2022: International issues that are compelling, new or changed in 2022

    The year 2020 ended on a note of hope, with the 8th December vaccination of 91-year-old Margaret Keenan, the world’s first person to receive a COVID-19 vaccine outside clinical trials. Despite an estimated 9.25 billion doses administered globally by 4th January, with 25 different vaccines either approved for use or under emergency use authorisation, 2022 […]

  • Biogen looks beyond Spinraza in SMA with Ionis deal

    Spinal muscular atrophy (SMA) therapy Spinraza has been a key earner for Biogen in recent years, but with sales now heading into reverse, the company is looking to extend its franchise. Its latest move is a $60 million upfront licensing deal with longstanding partner Ionis to exercise an option on BIIB115 (ION306), a longer-acting antisense […]

  • Boehringer’s spesolimab data raises hope for serious skin disease

    Fresh from being awarded a fast-track review from the FDA, Boehringer Ingelheim’s spesolimab has shown encouraging efficacy in a phase 2 trial in generalised pustular psoriasis (GPP), a rare and life-threatening skin disorder. GPP is very different from the more common plaque psoriasis, not least because it causes episodes (flares) of widespread eruptions of painful […]

  • 2021 in review: Building the framework of a patient-centric industry

    Patient centricity and patient engagement have been hot topics in pharma for some time – but buzz words do not always translate into concrete action. However, we have seen a framework of guidelines and recommendations during the last year on everything from increasing diversity to producing lay summaries, start to take shape. As part of […]

  • Novartis adds gene therapy for blindness with Gyroscope buy

    Gyroscope Therapeutics and its gene therapy for the sight-robbing disease geographic atrophy (GA) has been acquired by a big pharma company – but likely not the one you would expect. Just a few weeks France’s Sanofi made a $40 million investment in Gyroscope for first refusal rights on the gene therapy – called GT005 – […]

  • Drug development and working with specialised Rare Disease networks

    There are currently an estimated 300 million people living with one or more Rare Diseases across the world. There are over 7,000 Rare Diseases that have been identified, yet only 5% have treatments. However, despite the work still left to be done, there are now a growing number of treatments in the pipeline and 2020 […]

  • Leveraging augmented analytics to drive better business decisions

    In early 2020, health officials were cautious about forecasting a COVID-19 vaccine timeline to the public, as vaccine discovery was a historically laborious process that took years (if not decades). Yet a successful COVID-19 vaccine gained emergency approval in just 11 months, a blistering pace that likely saved millions of lives. Such a fast turnaround […]

  • Blow for Novartis as ligelizumab can’t top Xolair in phase 3

    Novartis’ big-selling IgE inhibitor Xolair has been a mainstay of treatment for chronic spontaneous urticaria (CSU) for many years, but with biosimilars looming the company has been looking to follow-up ligelizumab to defend the franchise. That hope now look decidedly faint, as ligelizumab was able to match Xolair (omalizumab) but not improve on it in […]

  • Ipsen places €480m bet on Genfit’s liver drug elafibranor

    Genfit was hit hard last year when it abandoned a phase 3 programme for elafibranor in non-alcoholic steatohepatitis (NASH), but pledged to press on with the drug in other indications – and now has the help of fellow French pharma group Ipsen. Ipsen has taken rights to PPAR agonist elafibranor as a treatment for primary […]

  • Mesoblast pummelled as Novartis exits COVID deal

    Novartis as opted out of a collaboration with Mesoblast to develop its stem cell-based therapy remestemcel-L as a treatment for COVID-19, prompting a slump in the biotech’s share price. Novartis agreed to pay $25 million upfront for opt-in rights to remestemcel-L just over a year ago and to make a $25 million equity stake in […]

  • Flush with COVID cash, Pfizer tables $6.7bn bid for Arena

    Pfizer has found another use for the windfall profits it is making from it BioNTech-partnered COVID-19 vaccine, agreeing to buy Arena Pharma and its late-stage ulcerative colitis drug etrasimod. The $100-per-share deal values Arena at around $6.7 billion, and gives Pfizer an entry into the oral S1P receptor modulator category that puts it in contention […]

  • How IQVIA used real-time analytics to generate custom ads that drew thousands of diverse and highly engaged patients to a global vaccine trial

    If you want to accelerate recruiting, go directly to the source: patients. It is a lesson the industry has always known, but recruiting patients during the pandemic underscored just how important an effective direct-to-patient recruiting strategy can be when timelines are critical. Rather than waiting for patients to learn about a trial from their physicians, […]

  • 2021: the year mRNA therapeutics came of age

    It started with two Covid-19 vaccines – 2021 is the year interest in mRNA therapeutics boomed, reports Katrina Megget The Covid-19 pandemic showed what could be done with resources and collaboration to accelerate the development of vaccines. One of its other scientific legacies was the specific development and approval of mRNA vaccines, the first in […]

  • UCB chases after Argenx with FcRN drug for myasthenia gravis

    UCB has the positive result it was hoping for in its phase 3 trial of anti-neonatal FC receptor (FcRn) antibody rozanolixizumab for autoimmune disease generalised myasthenia gravis (gMG), setting up regulatory filings next year. The MycarinG study met its primary endpoint, demonstrating a “statistically significant and clinically meaningful” improvement from baseline in the myasthenia gravis-activities […]

  • Trial sets up filings for uniQure’s haemophilia gene therapy

    Dutch biotech uniQure and partner CSL Behring are planning to file for approval of their gene therapy for haemophilia B, armed with new trial data that they say answers earlier concerns about the programme. The results are from the HOPE-B study, which was temporarily placed on a clinical hold by the FDA earlier this year […]

  • Acadia closer to bringing first Rett syndrome drug to market

    Acadia Pharma’s trofinetide has hit its efficacy targets in a phase 3 trial involving girls with the devastating neurological disorder Rett syndrome, although safety has dampened enthusiasm a little. Top-line data from the LAVENDER study in 187 girls and young women with Rett have shown that trofinetide had a significant effect on two primary efficacy […]

  • AI specialist Recursion nabs multibillion-dollar Roche partnership

    Roche has formed a decade-long alliance with Recursion Pharma that will focus on using machine learning and “industrialised” drug discovery to find new neuroscience and cancer drugs. The Swiss drugmaker is paying $150 million upfront to kick off the strategic-level partnership, which could cover as many as 40 programmes and could be worth “several billion […]

  • AZ gets closer to Ionis, licensing amyloidosis drug in $3.6bn deal

    AstraZeneca has snapped up another Ionis-discovered drug for its pipeline, this time phase 3 candidate eplontersen for the disease transthyretin amyloidosis (ATTR). The deal is another sizeable one too, with an upfront fee of $200 million, another $485 million tied to regulatory approvals, and up to $2.9 billion in milestones depending on sales levels if […]

  • Merck halts more trials of HIV candidate islatravir

    Merck & Co has halted enrolment in a pair of phase 3 trials of its experimental HIV drug islatravir, raising another warning flag about the highly-touted therapy. The two studies – IMPOWER 22 and IMPOWER 24 – are testing islatravir as a once-monthly, oral therapy for pre-exposure prophylaxis or PrEP, which accounts for a sizeable […]

  • Viagra ‘should be tested as Alzheimer’s therapy’

    Scientists in the US have found that people who use Viagra for erectile dysfunction seem to have a lower risk of developing Alzheimer’s disease – a link that they say should be examined more closely in clinical trials. Viagra (sildenafil) – originally developed by Pfizer – is now widely available as a generic and can […]

  • Novartis takes aim at risky Parkinson’s target with $1.5bn UCB deal

    Novartis has licensed a potential drug for Parkinson’s from Belgium’s UCB that it thinks could be the first oral, disease-modifying drug for the disease – if it can avoid the fate of earlier drugs in the class. The drug – called UCB0599 – targets a protein called alpha synuclein that tends to get misfolded and […]

  • GSK pumps £30m into a new Oxford University partnership

    GlaxoSmithKline has formed a five-year partnership with Oxford University to set up a new institute that will apply machine learning and functional genomics to the discovery of new medicines. The Oxford-GSK Institute of Molecular and Computational Medicine – based at Oxford’s Nuffield Department of Medicine – is being set up with £30 million (around $40 […]

  • Dyno attracts $1.6bn gene therapy deal with Astellas

    Astellas has signed another bolt-on deal to build its gene therapy pipeline, agreeing a deal with Dyno Therapeutics to tap into its adeno-associated virus (AAV) vector platform for delivering genes to skeletal and cardiac muscle. The Japanese drugmaker is paying $18 million upfront to exercise an option on the AAV platform – which my help […]

  • Sanofi snaps up acne vaccine developer Origimm Bio

    Sanofi has added to its vaccine pipeline with an agreement to acquire Austria’s Origimm Biotechnology and its vaccine-based immunotherapy for people with acne. The French group said that ORI-001, a vaccine based on recombinant proteins from the  Cutibacterium acnes (formerly Propionibacterium acnes) bacterium that is often found in acne lesions, could be the first ever […]

  • GSK poaches Pfizer exec Dormitzer as new vaccines R&D chief

    GlaxoSmithKline has found a replacement for head of vaccines R&D Emmanuel Hanon, who left the company for a US biotech earlier this year, hiring Phil Dormitzer from Pfizer. Phil Dormitzer Dormitzer is due to take up his new role at GSK on Friday, and will report to chief scientific officer and group president of R&D […]

  • Report suggests UK’s national vaccine facility is on the block

    The UK’s Vaccines Manufacturing and Innovation Centre (VMIC) – unveiled with fanfare by the government in 2018 – is rumoured to be up for sale. The facility at the Harwell Science and Innovation Campus in Oxford has received £215 million in public money since it was announced, and several companies have submitted bids for the […]

  • Pharma pledges rapid response to Omicron COVID variant

    With governments around the world announcing measures to curb the new B.1.1.529 variant of COVID-19 – now christened Omicron – leading vaccine manufacturers have said they are poised to develop new shots if needed. News of the new variant emerged last week, and has resulted in travel bans being imposed on various African countries by […]

  • Fall in efficacy of Merck’s molnupiravir puts Pfizer in driver’s seat

    Merck & Co has reported new data with its oral antiviral molnupiravir that suggests it is not as effective against COVID-19 as originally hoped. An updated readout from the MOVE-OUT study of molnupiravir shows that the original 48% reduction in the risk of hospitalisation or death in people with mild or moderate COVID-19 at high […]

  • Could safety scupper Ionis, Pfizer’s cholesterol drug vupanorsen?

    Ionis has reported phase 2b trial data for its Pfizer-partnered antisense drug vupanorsen for cholesterol lowering that showed it met its main efficacy targets, but raised a few safety concerns as well. The top-line results of the TRANSLATE-TIMI 70 study – in dyslipidaemia patients with elevated non-HDL cholesterol and triglycerides – showed that vupanorsen was […]

  • Trial of intranasal Alzheimer’s vaccine gets underway in US

    The first patients are now being treated as part of a first-in-human trial of a vaccine, administered as a nasal spray, that aims to slow down the progression of Alzheimer’s disease (AD). Researchers at Brigham and Women’s Hospital in Boston, US, hope that the vaccine will stimulate the immune system to clear the amyloid plaques […]

  • Buoyed by new data, Santhera preps filing for DMD drug vamorolone

    A year ago, Swiss biotech Santhera was slashing staff to cut costs after it abandoned lead Duchenne muscular dystrophy (DMD) therapy idebenone. Now, it’s ready to file replacement drug vamorolone, armed with new data from a pivotal trial, in what looks like a reversal of its fortunes. With prospects for idebenone looking shaky, Santhera snapped […]

  • AstraZeneca cuts the ribbon on its $1bn R&D lab in Cambridge

    After years of delays and rising costs, AstraZeneca has formally unveiled its $1 billion R&D facility in Cambridge, said to be the biggest science lab of its kind in the UK. The Discovery Centre (DISC) based in the Cambridge Biomedical Campus – first given the green light in 2015 – is being officially opened today […]

  • Chinese tech giant Baidu licenses mRNA algorithm to Sanofi

    Sanofi has bolstered its push into mRNA-based therapies with a new licensing deal – but not as might be expected with some up-and-coming biotech company. The French drugmaker has been enticed by an artificial intelligence (AI) algorithm developed by Chinese tech giant Baidu, which controls most of China’s search engine traffic and is sometimes referred […]

  • Next Generation Kinase Inhibitors Summit

    Turbocharge the Development of the Best-In-Class Kinase Inhibitor Products, to Enhance Specificity, Overcome Resistance to Deliver Safe & Durable Clinical Responses Celebrating 20 years of FDA approval and high efficacy profiles, kinase inhibitors have changed the face of oncology treatment. However, increasing resistance and safety issues limit the success of existing kinase inhibitor classes. The […]

  • 2nd Mitochondria-Targeted Drug Development Summit

    Overcome the Challenge of Treating Unmet Medical Needs Caused by Mitochondrial Dysfunction The 2nd annual Mitochondria-Targeted Drug Development Summit returns as the only industry-led meeting focused on overcoming the challenge of treating unmet medical needs caused by mitochondrial dysfunction. This forum will specifically focus on targeting mitochondrial dysfunction to develop therapies for chronic diseases caused by mitochondrial DNA […]

  • Gilead foregoes buyout, but pledges $725m to Arcus’ cancer drugs

    Gilead Sciences has forged closer ties to its cancer drug partner Arcus Biosciences , although a licensing deal doesn’t go as far as the takeover that some market commentators have been predicting. Gilead has exercised options on four drug candidates as part of its $2 billion, 10-year alliance that kicked off last year, with the […]

  • Novo Nordisk snaps up RNAi partner Dicerna in $3.3bn takeover deal

    Danish drugmaker Novo Nordisk must like what it has seen in its two-year-old alliance with gene-silencing specialist Dicerna Pharma – it has just agreed to acquire the biotech for $3.3 billion in cash. The two companies have been working together since 2019 to develop multiple RNA interference (RNAi) candidates for disorders such as non-alcoholic steatohepatitis […]

  • Novo Nordisk on seeking partnerships to expand its portfolio

    Its vast experience in diabetes and the amount of data collected has propelled Novo Nordisk into exploring new modalities and searching for valuable collaborators, according to the company’s senior VP of global drug discovery Karin Conde-Knape. She discusses which therapeutic areas the company is gaining momentum in and how it aims to build its presence […]

  • In shock move, Biogen chief medical officer Al Sandrock will stand down

    Biogen’s chief medical officer Al Sandrock has announced he will retire at the end of the year, ending a 23-year career at the biotech. The architect of many of Biogen’s biggest-selling drugs, Sandrock is bowing out as Biogen is still struggling with the rollout of its controversial new Alzheimer’s disease therapy Aduhelm (aducanumab). He will […]

  • Merck’s oral PCSK9 drug matches injectables in early trials

    Injectable antibodies targeting PCSK9 have failed to take off as expected in the cholesterol-lowering market, but Merck & Co hopes to do much better with an oral alternative. MK-0616 has been shown to reduce LDL cholesterol by a similar margin to injectables sold by Amgen and Sanofi/Regeneron – as well as Novartis’ longer-lasting RNAi therapy […]

  • Poor Glycaemic Control and Tooth Loss Study

    (World Diabetes Day, 14 November 2021) Largest tooth loss research study finds poor glycemic control in the over 30s results in fewer teeth In the largest study of its kind, 233,567 individuals’ insurance claims & check up data was analyzed to assess the relationship between glycemic control and natural tooth retention by age group The […]

  • AZ, Moderna say mRNA drug is promising in heart failure

    Moderna is already riding high on the strength of its mRNA-based COVID-19 vaccine, but the broader potential of its technology has been shown in a new AstraZeneca-partnered candidate for heart failure. At the American Heart Association (AHA) congress, the two partners presented results from the phase 2a EPICCURE trial that showed that injecting their AZD8601 […]

  • Legal requirement, ethical duty: How to develop compliant, useful lay summaries

    New lay summary guidelines walk sponsors through their obligations under EU regulation No. 536/2014, due to come into force in February next year. Patient involvement is key, they say. The sooner patients are involved in developing and publishing lay summaries, which will soon be a legal requirement in the European Union, the better. That was […]

  • Amid crowded migraine market, Lundbeck goes its own way

    The market for migraine treatment and prevention has been transformed by the launch of several new drugs targeting CGRP for many sufferers, but Lundbeck thinks there is still room for new approaches. The Danish drugmaker has started a phase 2 trial of a drug with a new mechanism of action, codenamed Lu AG09222, that could […]

  • One world, one health: The interconnected web of antimicrobial resistance

    Tackling antimicrobial resistance (AMR) requires a one health approach that joins the dots between the human, animal, and environmental drivers, consequences, and data. AMR threatens to undermine “almost a century of health gains” and marshalling a response to such a problem “requires cooperation at many levels”. That’s according to a newly published report from the […]

  • Compass points to positive psilocybin trial, but shares fall

    Compass Pathways has reported that its drug for treatment-resistant depression (TRD) based on magic mushroom ingredient psilocybin was effective in a phase 2b trial, although shares in the company fell afterwards. Treatment with COMP-360 delivered in a carefully controlled environment in which they listened to a curated music playlist, with two to four participants being […]

  • One dose of Regeneron’s COVID antibody protects for eight months

    A single dose of the COVID-19 monoclonal antibody therapy developing Regeneron reduced the risk of contracting the virus by around 82% for up to eight months, according to new trial data. The results suggest that the REGEN-COV (casirivimab/imdevimab) antibody could play a key role in protecting people who do not mount a sufficiently strong immune […]

  • Sanofi winds $40m into UK gene therapy firm Gyroscope

    French drugmaker Sanofi has made a $40 million investment in Gyroscope Therapeutics, with another $20 million potentially on offer as the UK biotech advances its clinical-stage gene therapies for ophthalmic diseases. The investment gives Sanofi rights of first refusal on GT005, Gyrocope’s lead gene therapy for geographic atrophy (GA) secondary to age-related macular degeneration (AMD), […]

  • Pfizer’s pill cuts COVID deaths and hospitalisations by 90%

    An oral antiviral drug developed by Pfizer has been shown to dramatically cut the risk of hospitalisation or death from COVID-19 when given in the early stages of infection. The phase 3 trial found that Paxlovid – which combines new protease inhibitor PF-07321332 with already-approved HIV drug ritonavir – reduced the risk of hospitalisation or […]

  • Novartis’ iptacopan hits the mark in rare kidney disease C3G

    Novartis has chalked up another successful trial for its targeted factor B inhibitor iptacopan, an orally-active drug trying to challenge antibody-based therapies for diseases associated with the complement pathway. A phase 2 trial of iptacopan in patients with the rare kidney disease C3 glomerulopathy (C3G) – which causes progressive kidney failure and has no approved […]

  • Talaris therapy ends need for immune drugs in transplant patients

    Two kidney transplant patients who received a stem cell therapy developed by Talaris Therapeutics were able to come off all immunosuppressant drugs within a year, without any evidence of graft rejection. The first findings from Talaris’ phase 3 trial of the cell therapy – called FCR001 – suggest it may be possible to eliminate the […]

  • Janssen on driving R&D innovation with data science

    The amount of patient data has skyrocketed, providing opportunities to gain a deeper understanding of diseases that are particularly complex or areas with a high level of unmet need, according to Janssen’s Najat Khan. She discussed the implications of this direction of travel for pharma, and some of the changes she’s seen in data science […]

  • Psychedelics startup Beckley attracts Janssen neuroscience VP

    Psychedelic medicines specialist Beckley Psytech has enticed a senior figure in Janssen’s neuroscience division to take on the role of its chief medical officer as it ushers its first drugs through clinical trials. Dr Frank Wiegand is joining Beckley after more than two decades at Janssen and parent company Johnson & Johnson, most recently as […]

  • UK biotech Dunad inks $1.3bn Novartis deal for its protein degraders

    In only its second year of operations, Cambridge, UK startup Dunad Therapeutics has already attracted a big pharma partner for its protein degradation platform. Swiss group Novartis has swooped in with a backloaded option deal that includes $24 million upfront in cash and equity – plus up to $1.3 billion in milestones – and covers […]

  • AbbVie preps filings after ABBV-951 tops oral Parkinson’s drugs in trial

    AbbVie has reported that a subcutaneous infusion of Parkinson’s disease candidate ABBV-951 was more effective than standard oral therapy in a phase 3 trial, paving the way for regulatory filings. ABBV-951 is based on foslevodopa and foscarbidopa – delivered via a subcutaneous device once a day – with the aim of providing full 24-hour control […]

  • Generic pill could be alternative to oral antivirals in COVID-19

    A generic pill has been shown to reduce hospitalisation rates in high-risk patients with early COVID-19, potentially offering a low-cost alternative to oral antivirals from Merck & Co and others, according to researchers in Brazil. The antidepressant fluvoxamine given as a twice-daily regimen for 10 days to acutely symptomatic patients with COVID-19 who also had […]

  • Takeda buys GammaDelta to boost cancer cell therapy credentials

    Japanese drugmaker Takeda has said it will exercise its option to buy UK-based GammaDelta Therapeutics, a specialist in ‘off-the-shelf’ cell therapies for solid tumours and blood cancers. The two companies have been working together since 2017, with Takeda pledging $100 million to help GammaDelta advance its cell therapy platform as part of a “build-to-buy” arrangement. […]

  • Industry welcomes boost to health research in UK budget

    Groups representing the biopharma industry have welcomed an increase in health-specific R&D to £5 billion in the UK autumn budget, despite a longer timeline for reaching the target of £22 billion in overall R&D spending. The Association of the British Pharmaceutical Industry (ABPI) and BioIndustry Association (BIA) both said that the increase acknowledges the enormous […]

  • GSK side-lines COVID drug otilimab to focus on Xevudy

    GlaxoSmithKline said today it would halt further development of its antibody for severe COVID-19 pneumonia – otilimab – in order to focus its resources on Xevudy for mild-to-moderate disease. The drugmaker said in its third-quarter results update this morning that it had decided to invest more in generating data on Vir BioTech-partnered anti-SARS-CoV-2 antibody Xevudy […]

  • Vifor, Angion organ damage drug fails phase 3 kidney transplant study

    Vifor Pharma and partner Angion Biomedica have suffered another setback with their drug to prevent organ damage, this time in high-risk kidney transplant patients. A phase 3 trial of ANG-3777 revealed that it was unable to improve eGFR – a measure of kidney function – compared to placebo in people who received a kidney transplant […]

  • 6th Microbiome Movement – Drug Development Summit Europe

    Decipher Mechanisms of Action, Understand Clinical Data & Ignite Partnership Opportunities to Accelerate Your Microbiome Research Whilst emerging phase 3 data is set to ignite investment and uncover the reality of microbiome-based therapeutics, there remain analytical challenges and translational bottlenecks that the community must come together to overcome, in order to redefine biopharmaceutical treatment and […]

  • Decentralized Clinical Research Strategies

    What strategies should your organization implement to better conduct decentralized research? Join colleagues from the research community at @Advarra’s free symposium to hear how leading research organizations  are adapting to a decentralized clinical landscape. https://info.advarra.com/decentralized-clinical-trials-symposium.html?utm_medium=advertisement&utm_source=pharmavoice&utm_campaign=october-pharmavoice-blog&utm_content=decentralized-clinical-trials-symposium The post Decentralized Clinical Research Strategies appeared first on PharmaVOICE.

  • Dupixent scores a first in rare skin disease prurigo nodularis

    Sanofi and Regeneron’s Dupixent has become the first biologic drug to show activity in prurigo nodularis (PN), raising the hope of a new treatment option for the rare and highly debilitating skin disease. The two companies are still waiting for the results of a second phase 3 trial, due in the first half of next […]

  • Blow to Biogen as ALS hope tofersen flunks phase 3 trial

    Already struggling to make headway with its recently launched Alzheimer’s drug, Biogen suffered a major late-stage pipeline setback after its tofersen drug for amyotrophic lateral sclerosis (ALS) missed the mark in a late-stage study. Tofersen missed its primary objective in the VALOR study, with patients on the drug failing to seen a statistically significant improvement […]

  • WHO plans pandemic prevention study of COVID’s origins

    The World Health Organization has set up a new scientific group to study the origins of COVID-19 in the hope that it will help prevent future pandemics. The new Scientific Advisory Group for the Origins of Novel Pathogens (SAGO) will bring together experts from areas such as epidemiology, animal health, clinical medicine, virology and genomics. […]

  • Positive safety data for long-term use of MS drug Kesimpta

    Multiple sclerosis drug Kesimpta has shown promising safety data for patients who are exposed to it for extended periods. Novartis announced the findings at the Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS), revealing that mean immunoglobulin G (IgG) levels in patients treated with Kesimpta (ofatumumab) remained stable, and there […]

  • Spark to use CombiGene therapy to target focal epilepsy

    Spark Therapeutics has secured the exclusive rights to CombiGene’s CG01 therapy, which aims to treat drug resistant focal epilepsy, in a deal worth $8.5m in advance with further payments due on reaching clinical milestones. The news sent Swedish firm CombiGene’s shares soaring – almost doubling from SEK6.29 to SEK14.10 the following day, when the development […]

  • J&J’s long-serving R&D chief Paul Stoffels announces retirement

    Paul Stoffels is bringing a nine-year tenure as head of R&D at Johnson & Johnson to a close, announcing plans to retire at the end of the year. Belgium-born Stoffels has been CSO at J&J since 2012, and was global head of pharma R&D for three years before that whilst also serving as chairman of […]

  • Failed IBD trial dents confidence in BMS’ deucravacitinib

    Bristol-Myers Squibb’s hopes of extending the use of its highly-anticipated psoriasis pill deucravacitinib into inflammatory bowel disease (IBD) have been hit by a mid-stage failure in ulcerative colitis. Confidence in the first-in-class tyrosine kinase 2 inhibitor has been riding high after a pair of phase 3 trials hit the mark in psoriasis, setting up regulatory […]

  • The power of R&D collaborations to improve outcomes in rare diseases

    Far more common than initially thought, rare disease treatments remain far too scarce. The answer to the problem, of course, lays in making medical research more effective. A key step towards this is great collaborative working, and partnerships with the likes of the European reference networks and French healthcare network G2M are crucial to success. […]

  • Pfizer agrees $630m deal to harness Voyager’s gene therapy tech

    Voyager Therapeutics has just landed a new gene therapy alliance with Pfizer that could go some way towards easing the pain of losing three other partners in the space of a couple of years. Pfizer is paying Voyager $30 million upfront to get access to its TRACER adeno-associated virus (AAV) vector technology, which aims to […]

  • Takeda halts trial of orexin drug in narcolepsy on safety grounds

    Japanese drugmaker Takeda has suspended dosing in phase 2 trials of its orexin agonist TAK-994, saying it had encountered a “safety signal” in the study. The future of the programme seems to be in jeopardy, as Takeda also indicated it decided to stop both studies early and said it is “committed to advancing its multi-asset […]

  • Could Pfizer’s Inlyta have a role in Alzheimer’s disease?

    Pfizer’s tyrosine kinase inhibitor Inlyta is already a cornerstone of treatment for some forms of cancer, but could have potential as a therapy for Alzheimer’s disease as well, according to scientists in Canada. The team – from the University of British Columbia – are particularly excited about their findings because the drug could represent an […]

  • Novartis’ Amit Nastik on COVID’s supply chain impact: the pharmaphorum podcast

    Episode 41 of the pharmaphorum podcast heard from Amit Nastik about the impact of COVID-19 on pharma supply chains and how he and his team mitigated disruption to Novartis’ own operations. Amit’s the global head strategy and operations and local markets manufacturing at Novartis Technical Operations and discussed how his company ensured an uninterrupted supply […]

  • Merck preps filings for oral COVID drug, but is it really a game changer?

    Merck & Co is on the brink of plugging a gap in the coronavirus treatment armamentarium after its oral drug molnupiravir cut deaths and hospitalisations in mild or moderate COVID-19 in a phase 3 trial. Merck and partner Ridgeback Therapeutics have said they will see emergency use authorisation from the FDA for molnupiravir on the […]

  • Key drug in Sanofi’s $1.45bn Kymab buyout shows promise in eczema

    Already a major player in atopic dermatitis with Dupixent, Sanofi looked to expand its position in the category earlier this year when it bought Kymab and its lead drug KY1005 for the skin disorder for $1.1 billion upfront. Now, it says the drug has delivered “exciting” results in a proof-of-concept trial. KY1005 – now renamed […]

  • Novartis drug raises hope of tackling malaria drug resistance

    More than two decades ago , Novartis made a big contribution to the fight against malaria when it won approval for Coartem, the first artemisinin-based combination therapy (ACT), which has transformed care for people with the parasitic infection. Now – over a billion doses of Coartem (artemether-lumefantrine) later – the drugmaker thinks it may have […]

  • UK’s clinical research recovering slower than Euro peers, says ABPI

    The pandemic had a negative effect on clinical research across all countries, but the UK seems to be taking longer than others to recover, according to a report from the Association of the British Pharmaceutical Industry (ABPI). The industry body maintains however that the UK can bounce back strongly if it adopts the innovative approaches […]

  • AZ claims full control of Caelum’s AL amyloidosis drug in $500m deal

    AstraZeneca’s newly acquired Alexion will buy the remaining equity in rare disease specialist Caelum Biosciences in a deal that could be worth up to $500 million. The deal gives the Anglo-Swedish drugmaker complete control of Caelum’s late-stage medicine for light chain (AL) amyloidosis, a potentially fatal disease that causes the rogue protein amyloid to build […]

  • Booster data backs Pfizer, Valneva Lyme disease vaccine

    Prospects for Valneva’s COVID-19 vaccine are up in the air, but another candidate partnered with Pfizer looks increasingly like it could fill the void of an effective shot for Lyme disease. New phase 2 data with their VLA15 vaccine shows that antibody responses from an initial three-dose regimen are initially high, although they start to […]

  • Sanofi abandons mRNA COVID jab, despite positive trial

    Sanofi has said that it won’t develop its mRNA-based vaccine for COVID-19 any further, even though it seemed to work in an early-stage trial, and will focus instead on another candidate partnered with GlaxoSmithKline. The French pharma group said it took the decision because mRNA vaccines from Pfizer/BioNTech and Moderna are already widely available today […]

  • Bad news for Biohaven as verdiperstat flunks first phase 3 test

    Biohaven’s key pipeline drug verdiperstat has failed to have any Therapeutic effect in patients with the rare neurological disorder multiple system atrophy (MSA) – its lead indication – but the biopharma company isn’t ready to abandon the drug yet. Myeloperoxidase (MPO) inhibitor verdiperstat was no better than placebo in the M-STAR trial in patients with […]

  • Treatment for all: the key steps to better representation in pharma

    As people have become more conscious and vocal about diversity and inclusion, Page & Page’s Lisa Lishman says pharma should reassess how it conducts clinical trials. The pharmaceutical industry is one of the most important industries in the world, providing lifesaving drugs and treatment to people who need it the most. This already significant industry […]

  • Capricor preps phase 3 for Duchenne cell therapy as it seeks a partner

    Capricor Therapeutics’ bid to find a partner for its Duchenne muscular dystrophy (DMD) cell therapy CAP-1002 could get a boost after final phase 2 data showed a strong effect on disease progression. Initial results from the 20-patient HOPE-2 trial reported last year showed signs of activity in older boys and young men with advanced DMD, […]

  • Early detection of cancers will require blood sweat and tears

    An NHS study of a new cancer blood test holds huge promise, but will not be without its challenges, says Snedden Campbell’s Ivor Campbell. The announcement that the NHS is taking part in a major new clinical trial for a blood test that could detect more than 50 cancers early has been welcomed as a […]

  • Roche builds evidence for Evrysdi’s benefits younger SMA patients

    Roche has highlighted new data with its oral treatment for spinal muscular atrophy (SMA) – Evrysdi – showing its benefits when given to pre-symptomatic babies with the rare disease. The updated results come from the open-label RAINBOWFISH study, which investigate the effects of Evrysdi (risdiplam) in children from birth to six weeks who had yet […]

  • 5th Antifibrotic Drug Development Summit -AFDD-

    The 5th Antifibrotic Drug Development Summit (AFDD) will provide you with a deeper understanding of how to progress early-stage candidates and avoid late-stage failures, know the studies that show early signs of efficacy and surrogate indication opportunities, and evaluate interacting mechanisms for new targets and biomarkers. This unique cross-organ, cross-disease discussion forum will host the transferrable insights […]

  • 2nd Applied Biocatalysis Summit 2021

    Advancing Sustainable, Streamlined Small Molecules, API and Fine Chemical Production using Enzyme Engineering & Scalable Industrial Biotransformations Attending the 2nd Applied Biocatalysis Summit is the perfect opportunity to understand the most recent developments in enzyme-catalyzed processes, computational technologies, and cutting-edge methodologies in biocatalytic cascade reactions to help the API and chemical industries apply biocatalysis more […]

  • Theravance axes staff as lead pipeline drug flunks phase 3 trial

    Theravance has been forced to downsize its business after its lead pipeline drug ampreloxatine missed the mark in a phase 3 trial, cutting 75% of its workforce. The Irish-American biopharma said this morning that ampreloxetine did not meet its objectives in a trial involving patients with symptomatic neurogenic orthostatic hypotension (nOH) – a rare condition […]

  • Rome Therapeutics raises $77m for junk DNA-targeting drugs

    Startup Rome Therapeutics has raised $77 million in second-round financing to help mine sequences of DNA – which were dismissed for years as ‘junk’ – for hidden treasure. The Cambridge, Massachusetts biotech is focusing specifically on the 60% of DNA sequences that repeat themselves hundreds or thousands of times in the genome, known as the […]

  • First subject dosed in psilocybin trial for rare headache disorder

    The first patient has received a low dose of Beckley Psytech’s psychedelic medicine psilocybin in a clinical trial for short-lasting unilateral neuralgiform headache attacks (SUNHA), a rare disorder. The UK company says that SUNHA is a debilitating condition that is estimated to affect 40,000 patients in US and Europe. It is characterised by short, extremely […]

  • AbbVie spies potential in Regenxbio eye disease gene therapy

    AbbVie has expanded its ophthalmology pipeline via a licensing deal with Regenxbio, paying $370 million upfront to get its hands on a gene therapy for wet age-related macular degeneration (AMD) and other eye diseases. The partnership centres on RGX314, which delivers a gene coding for an antibody against VEGF – a well-established approach to treating […]

  • France’s biotech ecosystem is primed for growth – it’s time to feed it

    After some years of stagnant funding and growth France is now at an inflection point for health care innovation, says Jeito Capital’s Dr Rafaèle Tordjman. Prior to the pandemic, our homegrown entrepreneurs felt too often they had to go abroad to the United States to get the funding they needed, government support for clinical trials […]

  • Apellis reports mixed data with eye drug, but plans to file anyway

    Apellis Pharma’s has reported mixed results with its lead drug pegcetacoplan in geographic atrophy (GA) – a major cause of blindness – with one hit and one miss in its phase 3 programme. Shares in the company were hit hard after the news was announced, although Apellis insisted that the totality of the data would […]

  • Biotech Insight podcast: Overcoming planning challenges in oncology

    The first episode of Kantar Health’s new Biotech Insight podcast takes aim at the planning and forecasting challenges that biotechnology companies face, with a particular focus on oncology. Joining Dominic Tyer for this instalment of Biotech Insight are Prithu Somani, senior manager of forecasting and business analytics at Genmab, and Kantar Health’s vice president of […]

  • Server cans autism hope bumetanide after phase 3 fails

    Servier and Neurochlore have been forced to admit defeat in their bid to bring the first drug to market for people with autism spectrum disorder (ASD), after a comprehensive fail for their bumetanide candidate in two phase 3 trials. The French partners said today that there was “no sign of effectiveness” for bumetanide compared to […]

  • Hybrigenics says it wants a merger with Bone Therapeutics

    French biopharma company Hybrigenics has made another bid to merge with Belgium’s Bone Therapeutics, a specialist in regenerative medicine that is still recovering from a setback in its lead development programme. Hybrigenics said in a press release today that it had started discussions with Bone Therapeutics, but the Belgian company was quick to come out […]

  • Bayer’s eliapixant hits the right notes in PAGANINI cough study

    Bayer has released the data in its phase 2b trial of eliapixant in refractory chronic cough, as it tries to close the gap with Merck & Co’s rival drug gefapixant. The drugmaker said last month that the PAGANINI study hit its main objective, which was to reduce the average cough frequency compared to placebo after […]

  • FDA slaps clinical hold on BioMarin’s PKU gene therapy

    BioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA has placed a phase 1/2 trial of its candidate for phenylketonuria (PKU) on hold while it investigates a safety signal. The biotech said that the US regulator ordered the pause on the study after liver tumours were […]

  • Amgen builds case for severe asthma drug tezepelumab

    Amgen and partner AstraZeneca have said their recently filed severe asthma drug tezepelumab has shown impressive results in patients who also have nasal polyps, a common complication of the respiratory disease. Tezepelumab reduced the annualised asthma exacerbation rate (AAER) by 86% in a subgroup of people with severe, uncontrolled asthma and polyps in the NAVIGATOR […]

  • Novartis’ transplant drug hope iscalimab flunks mid-stage trial

    Novartis hopes of improving on tacrolimus as the standard drug for preventing organ rejection in transplant patients have been dashed after its iscalimab candidate failed a mid-phase trial. An interim analysis of data from the CIRRUS-1 trial suggested that iscalimab (CFZ533) was less effective than tacrolimus at preventing rejection in kidney transplant patients when both […]

  • Pfizer raises pressure on GSK, J&J with late-stage RSV vaccine trial

    Pfizer has vaulted ahead with its respiratory syncytial virus (RSV) vaccine, closing the gap with GlaxoSmithKline and Johnson & Johnson as the companies strive to bring the first licensed shot to market. The first of an anticipated 30,000 subjects have been vaccinated in Pfizer’s phase 3 RENOIR trial of its vaccine, just behind GSK and […]

  • Targeting COVID-19 and cancer with immunotherapy advancements

    Anna Biosciences’ Dr Anthony Rullo discusses the drug development company’s immunotherapy platform. Over the last 30 years, we have seen dramatic improvements in patient outcomes due to the emergence of biologics – particularly monoclonal antibodies – as therapies for a wide variety of diseases and infections. Monoclonal antibodies (mABs) are highly specific therapies associated with […]

  • J&J vaccine fails to protect women against HIV infection

    Johnson & Johnson’s HIV vaccine candidate has failed its first major efficacy test, as it was unable to protect women against infection with the virus in a phase 2b trial carried out in sub-Saharan Africa. The Ad26.Mos4.HIV vaccine – which uses the same adenoviral technology as J&J’s COVID-19 vaccine and targets four HIV antigens – […]

  • AC Immune claims half a win for tau drug in Alzheimer’s trial

    Shares in AC Immune leaped today after the company said its tau-targeting Alzheimer’s disease candidate semorinemab hit one of its objectives in a phase 2 trial, although it missed another. The Swiss biotech’s Nasdaq-listed shares were up around 66% shortly after the announcement of data from the LAURIET study in patients with mild-to-moderate Alzheimer’s disease, […]

  • External control arms and debunking real-world data myths

    For the healthcare ecosystem to drive research and care, clarity is needed about the opportunities and challenges of ECAs and their value in bringing therapies to patients faster, say COTA Viraj Narayanan and Karla Feghali from ConvergeHEALTH by Deloitte. External control arms (ECAs) – also known as synthetic control arms – are based on real […]

  • ‘Endless RNA’ startup Laronde raises $440m to fuel pipeline

    Just a few months after breaking cover with its new take on RNA therapeutics, Laronde has raised an impressive $440 million in second-round financing backed by Flagship Pioneering – which was behind the founding of mRNA specialist Moderna. Laronde is developing what it calls ‘endless’ RNA or eRNA, a circular form of the material that […]

  • GSK takes SK bio-partnered COVID jab into phase 3

    As a top vaccine maker, GlaxoSmithKline has been a laggard in the COVID-19 vaccine race, but has put on a late burst of speed with a start of phase 3 trials of a second candidate that will be compared directly to the AstraZeneca shot. The move follows phase 1/2 results with SK bio’s GBP510, which […]

  • Data points to big win for Jardiance in hard-to-treat heart failure

    The data is now in from the EMPEROR-Preserved study of Boehringer Ingelheim and Eli Lilly’s Jardiance in heart failure with preserved ejection fraction (HFpEF), and suggest the drug performs better than Novartis’ Entresto – currently the only approved option for these patients. SGLT2 inhibitor Jardiance (empagliflozin) reduced a composite of cardiovascular (CV) death or hospitalisations […]

  • After Ultomiris stumble, Alexion delivers a rare disease win for AZ

    A drug for Wilson disease developed by AstraZeneca’s Alexion rare disease unit has cleared a phase 3 trial, setting up regulatory filings in the coming months. The FoCus study of ALXN1840 showed that once-daily oral drug met its primary objective in improving a key biomarker in Wilson disease, a rare and progressive genetic condition in […]

  • Amplifying the patient voice for better engagement, recruitment and retention

    Everyone is familiar with the phrase ‘patient-centricity’ but, where it might once have been seen as a ‘checkbox exercise’, it has gained preeminent status in the era of COVID-19, says Prime Global’s Ross Jackson. The pandemic has transformed many aspects of our industry and one area that change has come to is patient engagement within […]

  • Merck raises pressure on Pfizer in pneumococcal vaccine tussle

    Just a month after getting approval for its new pneumococcal vaccine Vaxneuvance in adults, Merck & Co has reported positive trial results in children that will ramp up the pressure on Pfizer and its market-leading Prevnar franchise. The 15-valent conjugate vaccine was at least as effective as Pfizer’s 13-valent Prevnar 13 in the study, which […]

  • Lilly taps Lycia for protein degrader tech in $1.6bn deal

    Eli Lilly has bolstered its position the fast-emerging area of protein degrader drugs, agreeing a $1.6 billion-plus deal with Lycia Therapeutics spanning up to five drug targets. The drugmaker has looked close to home for its latest entry into the category, as Lycia’s founder Prof Carolyn Bertozzi has held a seat on its board of […]

  • Vertex builds in gene editing yet again, with $1.2bn Arbor deal

    Vertex Pharma has ramped up its involvement in gene-editing medicines for the third time in a matter of months, agreeing a partnership with CRISPR specialist Arbor Biotechnologies that could be worth up to $1.2 billion. The latest deal allows Vertex to tap into Arbor’s technology platform to develop cell therapies for diseases like type 1 […]

  • AZ’s Alexion takeover hits a snag, as Ultomiris flunks ALS trial

    It has only been a month since AstraZeneca’s $39 billion takeover of Alexion was completed, but the merger has already had a setback. Alexion has dropped development of its Ultomiris drug in amyotrophic lateral sclerosis (ALS), chalking up the first clinical failure for the drug since AZ took control. The phase 3 CHAMPION-ALS trial showed […]

  • PROVENT trial puts AZ COVID antibody combo back on track

    AstraZeneca’s AZD7442 has shown that it can prevent COVID-19 infection when given to healthy patients – the first time this has been demonstrated by a long-acting antibody-based drug. The results of the almost 5,200-patient PROVENT study show that pre-exposure prophylaxis using the antibody combination reduced the risk of developing symptomatic COVID-19 by 77% compared to […]

  • Does draft RCT guide set the scene for future trials?

    A common set of standards for randomised clinical trials (RCTs) aims to help researchers develop the drugs and interventions of the future – no matter what they do or where they are based. The Good Clinical Trials Collaborative (GCTC) has published a draft guidance document, and is asking for the life sciences sector to make […]

  • Welcome to the COVIDome, a new tool for COVID-19 R&D

    Take a cohort of people hospitalised with COVID-19 and a group of negative controls, carry out extensive biological profiling of both groups and place all the information in a database. What do you get? A new R&D toolkit called the COVIDome, which has been shared by its developers in the hope that it could accelerate […]

  • Health Innovators: PureTech Health’s Daphne Zohar

    In latest episode of our Health Innovators video interview series, Dr Paul Tunnah speaks to Daphne Zohar, the co-founder and CEO of PureTech Health. They discuss the publicly-listed biotech’s pipeline projects and focus on the BIG (brain-immune-gut) axis as well as PureTech’s interest in digital therapeutics. Daphne explains how she built an impressive team of board […]

  • With Alzheimer’s market opening up, Lilly forms neuroscience unit

    Eli Lilly is one of the front runners in the bid to follow Biogen and Eisai onto the market with an anti-amyloid drug for Alzheimer’s disease, and its preparing the ground with a restructuring of its business units. As of 5 September, Lilly’s BioMedicines division will be split in two to form Lilly Neuroscience and […]

  • From start to finish – digitising clinical trials

    Over the past two years, the pharma industry’s demand for automated research solutions has increased significantly and, particularly when it comes to clinical trial design, the need has never been greater. This shift to digitised approaches has been triggered by some persistent challenges faced by those companies still reliant on applying manual processes. “A few […]

  • Beckley Psytech raises $80m for psychedelic meds development

    UK psychedelic medicine specialist Beckley Psytech has raised an impressive £58 million (around $80 million) in an oversubscribed second-round financing that will help fund clinical development of its psilocybin-based therapies. The Oxford-based company had originally hoped to raise $50 million from the series B, and the scale of the financing is further evidence that repurposing […]

  • Look out Dupixent, Lilly’s lebrikizumab is on the march

    Eli Lilly had a lot riding on its two phase 3 trials of IL-13 inhibitor lebrikizumab in atopic dermatitis (AD) – and it now looks like it has backed a winner. The company has claimed across-the-board wins against primary and secondary measures of efficacy in the studies, and while the data hasn’t been revealed yet, […]

  • Organicell on regenerative medicine: the pharmaphorum podcast

    In the latest episode of the podcast Dominic Tyer speaks with Dr Maria Ines ‘Mari’ Mitrani, chief science officer at the clinical-stage biopharmaceutical company Organicell Regenerative Medicine. They looked at the development of regenerative medicine and where Organicell hopes to add to that field. Then, with the company pivoting to try and help tackle Covid, they […]

  • More malaria hope as antibody protects against infection

    A single dose of a monoclonal antibody developed by scientists at the US National Institutes of Health (NIH) has provided protection for people exposed to malaria parasites for up to nine months. The results of the phase 1 trial suggest that antibodies could be used to protect travellers, military personnel and health care workers traveling […]

  • SK Bio’s COVID vaccine will start phase 3 study versus AZ jab

    South Korea’s SK Bioscience has been given the all-clear to start a phase 3 trial of its COVID-19 vaccine GBP510 that will compare the shot directly with AstraZeneca’s Vaxzevria. It is the first COVID-19 vaccine developed in South Korea to reach the pivotal trial stage, and from the earliest stages of its development has been […]

  • Predicting and improving clinical trial enrolment: A problem for psychiatry

    Patient enrollment is a common issue across many therapeutic areas in clinical research. Psychiatric disorders in particular represent a trial area with significantly high drop-out rates and poor patient recruitment. The reasons behind this problem, however, are rarely studied or understood. Despite this, new strategies devised over the last five years are beginning to reverse the […]

  • Kite takes the ‘Appian way’ to off-the-shelf cell therapies for cancer

    Gilead Sciences’ Kite Pharma has mapped out another route to the development of allogeneic or ‘off-the-shelf’ cell therapies for cancer, using technology developed by Appia Bio, a US startup focusing on haematopoietic stem cells (HSCs). The partnership will combine Kite’s experience in chimeric antigen receptor (CAR) therapies with Appia’s platform, which involves using HSCs to […]

  • Medicines Discovery Catapult relaunches Wolfson Radiochemistry facility in Manchester

    Medicines Discovery Catapult (MDC) today announces the relaunch of radiochemistry at the Wolfson Molecular Imaging Centre (WMIC) in Manchester. The facility, which was closed in 2020, includes the multi-million pound cyclotron, one of only a handful in the UK. It will supply hard-to-make radiochemicals to drug discovery biotechs and academic innovators – increasing the UK’s […]

  • Evaluating methods targeting Protein-Protein Interactions

    Protein-protein interactions (PPIs) are becoming increasingly relevant in the pathology of many diseases, including cancer. The issue, however, is developing an effective way of targeting them.  The latest advancements in methods targeting PPIs are designed to overcome the challenges limiting the conventional methods. Targeting PPIs offers another potential therapeutic target for diseases with complex biological […]

  • Bayer bolsters R&D platform with $2bn Vividion takeover

    Bayer headlined its second-quarter results this morning by unveiling a deal to buy Vividion Therapeutics, saying it will boost its ability to develop medicines against targets once considered “undruggable.” Bayer is paying $1.5 billion upfront to claim ownership of Vividion‘s drug discovery platform, which uses chemoproteomics to identify new binding sites on proteins and find […]

  • Lilly has a bad hair day as Pfizer rival hits the mark in alopecia

    Eli Lilly and Incyte have another rival in the rear-view mirror for Olumiant in alopecia areata after Pfizer reported that its ritlecitinib improved scalp hair regrowth in a late-stage trial. In the phase 2b/3 ALLEGRO trial, JAK inhibitor ritlecitinib hit its primary objective of improving the area of scalp covered by hair in people with […]

  • Chasing Merck & Co, Bayer builds case for cough drug eliapixant

    Bayer says its drug candidate for chronic cough – eliapixant – has hit the mark in a phase 2b trial, putting it in hot pursuit of Merck & Co’s gefapixant, which was filed for approval in March. The orally-active P2X3 receptor antagonist, also known as BAY1817080, hit the main objective of the study, which was […]

  • Novartis cleared to restart trials of new Zolgensma delivery route

    The FDA has relaxed a clinical hold on clinical trials of Novartis’ intrathecal formulation of spinal muscular atrophy (SMA) gene therapy Zolgensma, allowing a new phase 3 trial to get underway. Zolgensma (onasemnogene abeparvovec) is already approved as an intravenous treatment in babies and very young children for the rare muscle-wasting disease, but the new […]

  • Sanofi swoops on partner Translate, buying the mRNA firm for $3.2bn

    Sanofi has been forging ever-closer ties with its mRNA partner Translate Bio since the two started working together in 2018, and has now gone all in, offering $3.2 billion to buy the company outright. The $38-per-share deal – a 56% premium to Translate’s recent share price before an after-hours surge in the stock on rumours […]

  • Ipsen forges $1bn alliance with Exicure on neurodegenerative diseases

    Ipsen’s new chief executive David Loew pledged to ramp up spending on the company’s pipeline when he took the helm last year, and a flurry of licensing deals in the last few weeks shows that push is starting to gather momentum. The French company – which focusing on cancer, neuroscience and rare diseases – has […]

  • Takeda spins norovirus vaccine out into separate company

    Takeda has decided development of its norovirus vaccine candidate outside Japan will be more efficient if it is spun out into a dedicated company. The Japanese drugmaker has partnered with venture capital company Frazier Healthcare Partners to form HilleVax, which comes into being with full and exclusive ex-Japanese development and commercial rights to TAK-214 (now […]

  • Biogen adds real-world test for Aduhelm to its confirmatory trial

    Biogen and partner Eisai have said they will run a large-scale observational study of Alzheimer’s drug Aduhelm in the US to build evidence for the drug’s efficacy following its controversial approval. The ICARE AD-US study will provide information on the long-term effectiveness and safety of Aduhelm (aducanumab) as prescribed in routine clinical practice within the […]

  • AbbVie, Calico raise their age-related disease alliance funding to $3.5bn

    AbbVie and Alphabet spin-out Calico Life Sciences have agreed to fund their seven-year-old partnership seeking treatments for diseases of ageing with another $500 million apiece. The collaboration with Google sister company Calico – formed in 2014 with $1.5 billion in funding and extended in 2018 with another $1 billion top up – has already resulted […]

  • AbbVie, Calico raise their age-related disease alliance funding to $3.5bn

    AbbVie and Alphabet spin-out Calico Life Sciences have agreed to fund their seven-year-old partnership seeking treatments for diseases of ageing with another $500 million apiece. The collaboration with Google sister company Calico – formed in 2014 with $1.5 billion in funding and extended in 2018 with another $1 billion top up – has already resulted […]

  • Regeneron, AZ team up on drugs for obesity “superpower” gene

    AstraZeneca has formed a partnership with Regeneron to investigate the potential of treating obesity using drugs directed at GPR75, a protective gene identified by scientists at the Regeneron Genetics Centre. AZ’s buy into the programme comes a few weeks after Regeneron published findings in the journal Science suggesting that people with certain GPR75 mutations have […]

  • BioNTech’s mRNA vaccine drive now includes malaria, TB shots

    Flushed with the success of its COVID-19 vaccine, BioNTech has pressed the accelerator on the development of shots for other infectious diseases, and now plans to take malaria and tuberculosis candidates into the clinic next year. Human testing of the malaria shot should get underway by the end of 2022, according to the German biotech, […]

  • First published data backs CanSino’s inhaled COVID vaccine

    Early-stage clinical data with CanSino Biologics’ inhaled COVID-19 vaccine show that it was able to stimulate neutralising antibodies against SARS-CoV-2 at a dose well below that required with intramuscular administration. Published in The Lancet, a phase 1 study conducted in China showed that a two-dose regimen of aerosolised Ad5-nCoV was similar to that achieved with […]

  • Boehringer takes alteplase to phase 3 for COVID, but drops antibody

    Boehringer Ingelheim’s latecomer antibody therapy for COVID-19 has been side-lined, as the company focuses its attention on its thrombolytic drug alteplase, heading into a phase 3 programme later this year. The decision to discontinue development of BI 767551, an inhaled antibody, has been taken because of the “evolving landscape” in managing the pandemic including the […]

  • Oncology R&D: the patient insights we’re still missing, and how to catch up

    Imagine you are a pharma executive working in oncology R&D. The landscape is rapidly becoming more competitive and overcrowded. You are under pressure to bring solutions now, and for your entire career, you have been taught that things must be done a certain way. Patient engagement is viewed by many as a nice-to-have rather than […]

  • Allergen builds case for first drug therapy for presbyopia

    If you are in middle aged and find you have to hold your phone at arm’s length to read, you may have presbyopia – a difficulty focusing on close objects that is usually corrected using glasses, contact lenses or laser eye surgery. Now, there could be another alternative, in the form of eye drops administered […]

  • Virtual reality therapy: The future of chronic pain management?

    Could virtual reality tools be effective in helping patients ‘unlearn’ their chronic pain? pharmaphorum speaks to Professor Christopher Eccleston from the University of Bath’s Centre of Pain Research to find out how digital therapeutics are shaping the future of pain management. A digital software developed by Finnish drugmaker Orion is aiming to address chronic pain […]

  • COVID-19: Celltrion’s therapeutic option

    In just over a year, Celltrion Healthcare says it has identified and delivered a potential treatment for COVID-19. pharmaphorum spoke to Kwon Ki-sung, Head of Celltrion’s R&D unit, to learn more. Celltrion’s COVID-19 therapeutic, CT-P59, is a monoclonal antibody (mAb) with activity against COVID-19. It was identified as a potential treatment through the screening of […]

  • Look out BMS, Cytokinetics has its eye on mavacamten’s turf

    Shares in Cytokinetics surged after it reported positive mid-stage results for CK-274, a drug for hypertrophic cardiomyopathy (HCM) that could tread on the toes of Bristol-Myers Squibb’s mavacamten – acquired as part of its $13.1 billion takeover of MyoKardia last October. In HCM, the heart muscle become thickened and in some patients can obstruct blood […]

  • Complex Medicines: A multi-billion-pound addition to the UK life sciences industry

     A new report published by Medicines Discovery Catapult and CPI details the huge medical and market potential for complex medicines in the UK   ‘Shaping the UK into an epicentre for complex medicines’ is a report by the Medicines Discovery Catapult (MDC) in partnership with the CPI showcasing the extensive potential for the UK in […]

  • Ipsen tries again in Parkinson’s disease with $363m IRLAB deal

    French drugmaker Ipsen has made another foray into the Parkinson’s disease category, licensing rights to an oral dopamine D3 receptor antagonist from Sweden’s IRLAB for $28 million upfront.  The deal gives Ipsen worldwide rights to the mid-stage drug – called mesdopetam – and puts the company on the hook for another $335 million in development, […]

  • GSK unveils plan for life sciences cluster at Stevenage campus

    GlaxoSmithKline wants to sell more than a third of the space at its R&D campus in Stevenage, UK, to a new bioscience cluster that it says could become one of the largest in Europe. The drugmaker says it is seeking a development partner for the project, and hopes to unlock £400 million ($553 million) in […]

  • Virtual GP clinics – are they accessible as they need to be?

    As the the pandemic accelerates a move towards remote doctors appointments, work still needs to be done to ensure that digital healthcare is easy and accessible. Sam Jansen explores the practical steps needed to ensure people are comfortable with virtual clinics and the tech involved. Behind the swathe of headlines lauding the way in which […]

  • Trials leave Galapagos’ SIK programme looking under the weather

    Galapagos has had to send its SIK inhibitor programme back to the drawing board after its lead compound GLPG3970 has failed two of three clinical trials, adding to a string of disappointments for the Belgian biotech.  Results from a phase 1b study in psoriasis and two phase 2a studies in ulcerative colitis and rheumatoid arthritis […]

  • First US patient gets total heart replacement with CARMAT device

    An experimental artificial heart developed by French medical device specialist CARMAT has been implanted into the first of 10 subjects due to be enrolled in a US trial. The Aeson bioprosthetic device has already been implanted in a number of European patients with end-stage biventricular heart failure – affecting both sides of the heart – […]

  • Lilly grabs glucose-sensing insulin tech, buying Protomer in $1bn deal

    Eli Lilly bought a stake in Protomer Technologies and its glucose-sensing insulin platform last year, and it must like what it has seen in the programme since then – it has just agreed to buy the company outright.  Pasadena, California-based Protomer is working on proteins that can sense molecular activators in the body and only […]

  • AZ, J&J tweaking COVID shots to reduce clotting risks; report

    AstraZeneca and Johnson & Johnson are both exploring ways to modify their COVID-19 vaccines to minimise the risk of severe blood clotting reactions that are seen – albeit rarely – in some people receiving the jabs. At the same time, the UK’s National Institute for Health and Care excellence (NICE) is developing guidance to help […]

  • Shot in the arm for Affinivax as it challenges Pfizer’s Prevnar

    Already facing one rival to its blockbuster pneumococcal vaccine Prevnar, Pfizer could have another to contend with, after Affinivax reported positive phase 2 results with its ASP3772 candidate – plus a new breakthrough designation from the FDA. Last year, Pfizer made almost $6 billion from its Prevnar 13 vaccine, which covers 13 common serotypes of […]

  • Biogen builds MS pipeline via $125m deal with China’s InnoCare

    Biogen has moved to shore up its besieged multiple sclerosis franchise by licensing a BTK inhibitor from Chinese biotech InnoCare currently in mid-stage clinical testing. InnoCare gets $125 million upfront in return for sharing rights to orelabrutinib, an orally-active BTK drug that is able to cross the blood-brain barrier and penetrate the central nervous system. […]

  • Novo Nordisk bulks up in rare diseases with $1.2bn Prothena deal

    Diabetes giant Novo Nordisk has opened a new front in its rare disease R&D, thanks to an acquisition agreement with Prothena focusing on drugs for ATTR amyloidosis. The Danish drugmaker is paying $100 million upfront for rights to antibody drug PRX004, which is heading for phase 2 trials in cardiomyopathy associated with ATTR amyloidosis, a […]

  • Taking steps towards a data-driven future for health and social care

    A new policy paper promises to make it easier for innovators and developers to build and deploy tech and AI solution. So, will it help make the vision of a data-driven health service a reality? COVID-19 “revolutionised” the health and care data space, and we have a duty to apply what we have learnt to […]

  • Rare disease: industry, patients and tech join forces on diagnosis

    Takeda’s Adam Zaeske discusses how cross-sector collaboration is shaping a brighter future for rare disease diagnosis. Diagnosis can sometimes be the most difficult part of a rare disease patient’s journey, and it’s with good reason that the process is often referred to as an ‘odyssey’ in these communities. “We’re dealing with very small populations of […]

  • Accelerating the adoption of innovation in the NHS

    Spreading innovation in the NHS is as much about fostering collaboration and building relationships as it is about designing new interventions and launching new technologies. Scaling up the adoption of new interventions and technologies in the NHS has always been something of a challenge. But a review of approaches used by the Academic Health Science […]

  • Moderna plans triple vaccine targeting flu, COVID and RSV

    Buoyed by the success of its COVID-19 vaccine, Moderna has started a phase 1/2 trials of its mRNA-based shot for seasonal influenza, targeting four different flu strains.  The first patents have now been enrolled into the study of the shot – codenamed mRNA-1010 – which is designed to generate antibodies against influenza A H1N1, H3N2 […]

  • Dementia, cancer headline UK’s 10-year plan for life sciences sector

    The UK’s long-awaited Life Sciences Vision strategy has been published, setting out the government’s stall as it tries to deliver on its promise to keep the country at the forefront of the sector. The document published today lays out seven healthcare “missions” for the UK’s industry as it tries to double down on the success […]

  • A life sciences vision to deliver health, wealth and resilience for the UK

    The ABPI’s chief executive Dr Richard Torbett outlines a new ‘vision’ and 10-year strategy for UK life sciences, launched by the government today. After an extraordinary year of science versus disease, the UK has cemented itself at the forefront of the global response to COVID-19. What has been achieved in just 18 months is nothing […]

  • Boehringer, Lilly hail Jardiance data in hard-to-treat heart failure

    Boehringer Ingelheim and Eli Lilly have the results they were hoping for in a large-scale study of Jardiance therapy in heart failure with preserved ejection fraction (HFpEF), a form of chronic heart failure associated with high hospitalisation rates, poor quality of life and increased mortality. The results of the much-anticipated EMPEROR-Preserved trial found that SGLT2 […]

  • COVID-19 neutralising antibody tests – an integrated approach

    Nina Garrett, R&D director at Abingdon Health discusses the key role antibody testing is playing in the pandemic and how an integrated approach with vaccines could help life resume. Millions of tests for COVID-19 are taken worldwide every day, as they become an increasingly important tool for our return to normality. Testing programmes have been […]

  • UK biotech ‘booming’ as sector rakes in £1.6bn in Q2 financing

    The UK’s biotechnology sector is going through a purple patch, attracting almost £1.6 billion ($2.2 billion) in financing in the three months to end-May – which is a record for the industry. The new figures – from the BioIndustry Association and Clarivate – were dominated by more than £1 billion in venture capital funding for […]

  • Almirall kicks off another hunt for skin disease therapy partnerships

    Spanish drugmaker Almirall has launched another search for early-stage research projects that it can partner in dermatology, its core area of expertise. The latest edition of its AlmirallShare competition – which will take submissions until 31 October this year – is hunting for innovative therapies for skin diseases that are in the research or preclinical […]

  • Herd immunity is nice – but what about me?

    Shahrokh Shabahang discusses why we need a personalised approach to COVID-19 immunity testing. To-date, the battle against COVID-19 has been waged largely in the field of public health, using a series of binary, black-and-white thresholds to measure progress. The number of positive cases per 100,000 people, yes/no results on PCR tests, absence or presence of […]

  • Private equity firms play ‘pass the Parexel’ with $8.5bn handover deal

    Four years after being taken private, Parexel has been sold again, with Goldman Sachs’ private equity arm and EQT paying $8.5 billion to claim ownership of the contract research organisation. The handover comes four years after Pamplona Capital Management bought the company for around $5 billion, and just a few weeks after lab tech company […]

  • GSK backs CEO Walmsley, rejecting Elliott’s call to change board

    GlaxoSmithKline’s board had responded to the broadside attack delivered by activist investor Elliott Management yesterday, saying it stands firmly behind the leadership of chief executive Emma Walmsley. The drugmaker rejected calls by Elliott to change its board and accelerate the complete divestment of its consumer health unit which is due to be split from the […]

  • ABPI launches 2021 Code of Practice

    The Association of the British Pharmaceutical Industry (ABPI) has launched its 2021 Code of Practice to help companies operate to high ethical standards.   The ABPI Code of Practice is the industry’s commitment to operate in a professional, ethical, and transparent manner, for the benefit of patients and the public. It is independently administered by the […]

  • AZ drug for hard-to-treat heart failure has mixed results in trial

    AstraZeneca has reported the first phase 2 results with a drug for heart failure with preserved ejection fraction (HFpEF), showing it worked as expected but wasn’t able to provide any clinical benefit to patients.  Daily doses of AZD4831 were able to reduce the activity of myeloperoxidase (MPO), an enzyme linked to tissue and blood vessel […]

  • Health Innovators: Eliot Forster, F-Star

    In this episode of Health Innovators, pharmaphorum founder Paul Tunnah interviews Eliot Forster, CEO of F-Star – a clinical-stage, NASDAQ-listed biopharmaceutical company focused on transforming the lives of patients with cancer.     About the author Dr Paul Tunnah is chief content officer and UK managing director for Healthware Group, a next-generation integrated consulting group that […]

  • Pharma’s use of synthetic biology could deliver radical innovation

    Synthetic biology holds much promise for the life sciences sector as it continues to look for new ways of treating disease and accelerating drug development, says Debiopharm’s Tanja Dowe. Synthetic biology has grown exponentially in less than a decade, with CRISPR gene-editing as the key technology driving this through its use in areas like cancer, […]

  • Pharma’s use of synthetic biology could deliver radical innovation

    Synthetic biology holds much promise for the life sciences sector as it continues to look for new ways of treating disease and accelerating drug development, says Debiopharm’s Tanja Dowe. Synthetic biology has grown exponentially in less than a decade, with CRISPR gene-editing as the key technology driving this through its use in areas like cancer, […]

  • COVID-19 vaccine development: Safety monitoring continues revolution in RWD

    From cancer to COVID, real-world data is changing the way we research therapeutics, monitor safety, and collaborate on the development of treatments designed to improve outcomes for patients in need. In today’s feature, Dr. Miruna Sasu lays out how real-world data is evolving in the post COVID-19 vaccine reality.  Only a year after the emergence […]

  • COVID-19 vaccine development: Safety monitoring continues revolution in RWD

    From cancer to COVID, real-world data is changing the way we research therapeutics, monitor safety, and collaborate on the development of treatments designed to improve outcomes for patients in need. In today’s feature, Dr. Miruna Sasu lays out how real-world data is evolving in the post COVID-19 vaccine reality.  Only a year after the emergence […]

  • New hybrid healthcare agency Stirred launches

    Healthcare communications specialists Jo Spadaccino and Stuart Hehir have launched a new agency named Stirred which will offer communications, marketing and creative consultancy to healthcare businesses. Spaduccino and Hehir previously worked together at health communications agency Pegasus (recently renamed Mind + Matter). “As the world repairs and responds to the needs of today, health has now […]

  • White House pledges $3 billion war chest for COVID antivirals

    The Biden Administration has said it will allocate $3 billion of its pandemic rescue plan to the development of a new generation of antiviral therapies for COVID-19, as well as future viral threats. The plan was announced today by National Institute of Allergy and Infectious Diseases (NIAID) director and chief medical advisor Anthony Fauci, who […]

  • Biogen abandons tau antibody for Alzheimer’s after phase 2 miss

    Biogen has four other Alzheimer’s candidates in its clinical pipeline behind recently-approved Aduhelm, but one of them – anti-tau antibody gosuranemab – has just been canned. The decision comes on the back of the phase 2 TANGO study of gosuranemab (also known as BIIB092), which showed that the antibody was unable to show a benefit […]

  • CureVac hit hard as its COVID jab fails to make the grade

    CureVac has just had a harsh reminder of the perilous nature of drug development, after reporting that its mRNA-based COVID-19 vaccine was just 47% effective in its 40,000-subject phase 3 trial. That is below the threshold efficacy level for a vaccine laid out by the World Health Organisation (WHO) in the early stages of the […]

  • Transforming pharma R&D with a scientist-centric approach to AI & automation

    As biopharmaceutical products have become increasingly complex, both in design and in regulatory requirements, we need a new way of working. Markus Gershater looks at how automation and artificial intelligence (AI) can transform pharma R&D. Due to increasing complexity in R&D, pharma teams are under pressure to drive down costs and speed up time to […]

  • Sage/Biogen’s zuranolone works in depression, but maybe not enough

    Sage Therapeutics and Biogen’s antidepressant candidate zuranolone has hit its objectives in a phase 3 trial, but a falloff in efficacy over time has raised concerns about the commercial potential of the drug. The WATERFALL trial of zuranolone showed that a two-week course of the oral GABAA receptor modulator significantly reduced symptoms of depression compared […]

  • Regeneron drug saves lives in severe COVID-19, but it’s not for all

    People who cannot generate an effective immune response against COVID-19 can be protected with an infusion of Regeneron’s antibody therapy REGN-COV, according to new data from the UK RECOVERY trial. The therapy significantly reduced the risk of death, the length of hospital stay and the need for a ventilator in the study, which involved around […]

  • Biogen’s ambitions in gene therapy hit by another failed pivotal trial

    When Biogen acquired Nightstar Therapeutics in 2019, it added a pair inherited retinal disorder (IRD) gene therapies that it hoped would accelerate a push into the fast-evolving category.  Just over two years later, both of those candidates have failed late-stage clinical trials, leaving Biogen’s $800 million investment in Nightstar looking like a poor deal. The […]

  • A roadmap for adopting New Science 

    The pandemic’s disruption galvanised the biopharma industry into unprecedented action, demonstrating what’s possible when the global scientific community collaborates in exceptional ways and public-private partnerships drive innovation to address a common need. Can the biopharma industry step up again to shatter industry orthodoxy and create new pathways for innovation, access, and affordability? The adoption of […]

  • Novavax unveils phase 3 COVID jab data, but has it missed the boat in US?

    The top-line phase 3 data for Novavax’ COVID-19 vaccine look good, with a 90% overall efficacy rate, but a dip in its share price indicates investors fear it could be too late for the programme. Novavax’ price spiked 9% after the data were announced today, but fell back just as quickly, despite the company’s assertions […]

  • EHA21: Study raises BMS’ hopes for Reblozyl in less severe disease

    Bristol-Myers Squibb has taken another step towards achieving its blockbuster hopes for beta thalassaemia-associated anaemia therapy Reblozyl, after hitting the target in a phase 2 trial in patients who don’t require regular blood transfusions.  Reblozyl (luspatercept) is already approved to treat thalassaemia patients who depend on transfusions, but getting approval in patients with milder disease […]

  • EHA21: Novartis trumpets iptacopan as oral alternative to antibodies in PNH

    The mainstay of therapy for the rare blood disease paroxysmal nocturnal hemoglobinuria (PNH) is Alexion’s infusion therapy Soliris, but Novartis’ iptacopan could provide an oral option after positive phase 2 trial results. The first-in-class therapy – a targeted factor B inhibitor – was able to “substantially” reduce the destruction of red blood cells (haemolysis) that […]

  • EHA21: Sanofi builds case for rare disease drug sutimlimab after FDA rejection

    Sanofi’s hopes of a speedy approval of first-in-class C1s inhibitor sutimlimab in rare disorder cold agglutinin disease (CAD) were dashed by the FDA, but new phase 3 data keep the drugmaker on track for a resubmission before year-end. The results of the 42-subject CADENZA study – reported at the ongoing European Haematology Association (EHA) – […]

  • Bayer’s two-pronged cell/gene therapy assault on Parkinson’s enters clinic

    Bayer subsidiary BlueRock Therapeutics has dosed the first Parkinson’s disease patient in a phase 1 trial of its cell-based therapy DA01, which aims to replenish the neurons lost in people with the neurodegenerative disorder. At the same time, the German group’s Asklepios BioPharma (AskBio) unit has started recruiting patients for a phase 1b trial of […]

  • J&J backs out of $1.6bn blood cancer antibody alliance with Argenx

    Belgian biotech Argenx has lost its development partner for acute myeloid leukaemia antibody cusatuzumab, after Johnson & Johnson backed away from the alliance after two and a half years. J&J’s Cilag unit – part of its Janssen pharma division – said this morning that it had decided not to continue the collaboration after a “review […]

  • How Microsoft Teams streamlined cross-country forecasting for biotech pioneers.

    J+D Forecasting has worked with one of Europe’s largest, leading biotechnology pioneers, helping to facilitate better, more collaborative forecasting for hundreds of colleagues across the continent. For the company’s most recent forecasting cycle, our experts have worked to incorporate Microsoft Teams as a core software, as well as extending Power BI, to enable regional users, and […]

  • The Bloc hires Stuart Goldstein as COO

    Stuart Goldstein has been hired as the chief operating officer at New-York-based healthcare creative agency The Bloc. In the newly-created role, Goldstein will oversee the execution of 4,500 projects per year across The Bloc’s US client business. He will be responsible for project management and identifying metrics to increase efficiency and profitability. Goldstein has over […]

  • MorphoSys offers $1.7bn for Constellation, but investors see stars

    Shares in German biotech MorphoSys have gone on the slide after it announced a $1.7 billion deal to buy its US rival Constellation Pharma and its pipeline of cancer and haematology therapies. MorphoSys said the $34 per share deal would accelerate growth by adding two clinical-stage drug candidates, namely BET inhibitor pelabresib (CPI-0610) in phase […]

  • What HCPs think about blood clots linked to COVID-19 vaccines

    In the latest edition of What HCPs Think, CREATION.co’s Laura Marsh looks at what healthcare professionals are saying about blood clots linked to COVID-19 vaccines. The formation of blood clots is a necessary process in order for the body to repair damage caused by injury and to slow down bleeding. However, when blood clots form inappropriately, […]

  • Amgen pays Kyowa $400m upfront for OX40 eczema drug

    Amgen has made a play in a hot area of immunology R&D, paying $400 million upfront to claim rights to an OX40-targeting drug from Kyowa Kirin that is ready for phase 3 testing for atopic dermatitis or eczema.  The deal gives Amgen rights to the KHK4083 antibody in all countries except Kyowa’s home market of […]

  • Sanofi’s venglustat bombs again, but isn’t dead yet

    Shortly after failing a Parkinson’s disease study, Sanofi’s venglustat has missed the mark in another trial in autosomal dominant polycystic kidney disease (ADPKD), whittling down the list of possible uses for the drug even further.  Sanofi is still running studies of the glycosphingolipid (GSL) inhibitor in rare lysosomal storage diseases like Gaucher disease type 3, […]

  • Are microtaggants the key to making medicines smarter?

    Pharmaceutical companies are accountable for ensuring that their medicine is safe when it comes into the hands of patients. The dramatic rise in online drug sales during the COVID-19 pandemic has increased the supply of counterfeit drugs, and many companies are now considering the addition of on-dose authentication for high-risk or high-value products. To implement […]

  • Novo Nordisk crowns cell therapy pipeline with $598m Heartseed deal

    Novo Nordisk is paying $55 million upfront to secure rights to a cell therapy for heart failure developed by Japanese startup Heartseed. The Danish drugmaker has acquired exclusive rights outside Japan to Heartseed’s HS-001 therapy based on heart muscle cells (cardiomyocytes) derived from human stem cells, and is due to start the phase 1/2 LAPiS […]

  • Chasing Lilly, Concert eyes 2023 filing for its JAK drug for alopecia

    Concert Pharmaceuticals has started the second of two planned phase 3 trials of its drug candidate for alopecia areata (AA), a common cause of hair loss, and says it hopes to report results in the second half of 2022. The two studies – THRIVE-AA1 and THRIVE-AA2 – are testing oral JAK1/2 inhibitor CTP-543 in adults […]

  • Novo Holdings leads $200m financing into Esco Lifesciences Group

    Life sciences tools company Esco Lifesciences Group has scored $200m in a series A and crossover round co-led by Novo Holdings and Vivo Capital. Funds will be used to expand the company’s presence across Asia and create an innovation hub in Boston for cell and gene therapy technologies. The deal was described by the companies […]

  • Now what? The future of clinical trials after the pandemic

    Sheila Rocchio highlights four key areas life sciences must improve on as an industry to drive real change in how clinical trials are conducted. This is definitely a time for the life sciences industry to be proud of our accomplishments. The way in which the industry came together to support the development of the COVID-19 […]

  • How diversity can help life sciences advance its lifesaving solutions

    Paula Parfitt discusses the importance of workplace diversity and how life science companies can build a thriving, diverse workforce. Diversity, equity and inclusion (D, E & I) is a sensible business choice with significant commercial benefits. A homogenous workforce has a smaller pool of collective experience, meaning that when problems arise, collective creativity and innovation […]

  • New Cell & Gene Therapy Collective seeks to boost UK adoption

    The UK has been a global leader in introducing cell and gene therapies that can potentially transform the lives of patients with debilitating diseases – but there are still many barriers to wider adoption. These challenges often arise because these therapies go through the same value assessment processes as less complex medicines, and the infrastructure […]

  • NGM craters after dropping mid-stage NASH candidate aldafermin

    Shares in NGM Biopharma have plummeted after the US biotech said it would abandon development of aldafermin in non-alcoholic steatohepatitis (NASH), adding to a lengthening list of failed candidates for the fatty liver disease.  The decision was taken after a phase 2b trial of NGM’s lead drug candidate missed its primary target of showing an […]

  • After Sanofi’s setbacks, Takeda eyes finish line with its dengue vaccine

    Takeda has revealed long-term clinical trial results with its dengue vaccine that reinforce its safety and efficacy – and help to differentiate it from Sanofi’s challenged Dengvaxia shot. Updated results from the large-scale TIDES trial of TAK-003 show that three years after dosing it was 62% effective at warding off dengue symptoms and prevented more […]