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Pharming Group NV’s stock skyrocketed March 24 on news that the U.S. FDA approved Joenja (leniolisib) to treat activated phosphoinositide 3-kinase delta syndrome (APDS) for those ages 12 and older. The nod, which came a few days ahead of the March 29 PDUFA date, sent shares (NASDAQ:PHAR) up 33%, or $3.69, to end the day at $14.96.
Pharming Group NV’s stock skyrocketed March 24 on news that the U.S. FDA approved Joenja (leniolisib) to treat activated phosphoinositide 3-kinase delta syndrome (APDS) for those ages 12 and older. The nod, which came a few days ahead of the March 29 PDUFA date, sent shares (NASDAQ:PHAR) up 33%, or $3.69, to end the day at $14.96.
Incyte Corp.’s bid for a once-daily vs. twice-daily version of the Janus kinase inhibitor Jakafi (ruxolitinib) was foiled, at least temporarily, by a complete response letter (CRL) from the U.S. FDA.
Dupixent (dupilumab) continues to expand its scope, this time potentially leading it and its developers into a new, multibillion dollar blockbuster market. Dupixent hit the primary and all key secondary endpoints in a phase III study of treating chronic obstructive pulmonary disease (COPD).
Phase II data that rolled out from Merck KGaA with its Bruton's tyrosine kinase (BTK) inhibitor evobrutinib in relapsed multiple sclerosis (MS) put more eyeballs on the mechanism. It’s already well validated in oncology, but resistance has arisen there, and at least two firms – Beigene Ltd. and Nurix Therapeutics Inc. – are striving for solutions with degrader candidates.
After Novartis AG decided not to take it forward in-house, the U.S. National Institute on Drug Abuse is to fund phase III development of mavoglurant in the treatment of cocaine abuse disorder. The agreement to back a trial involving up to 330 participants is with Stalicla SA, which in-licensed the glutamate receptor antagonist from Novartis earlier this year.
The increased availability of capital, greater access to talent, strong local governmental support and more focused attention on IP issues have increased the complexity of deals taking place between biotech and big pharma companies in China, according to Michelle Chan, chief business officer of Insilico Medicine Inc, who spoke in a panel discussion on the topic of Asia-Pacific partnering at this year’s Bio-Europe Spring, in Basel, Switzerland on March 21.
Partially blocking a receptor that helps regulate the activity of the inflammatory cytokine molecule interleukin-6 (IL-6) seems to promote tissue regeneration and block degeneration in a model of osteoarthritis. As reported in the March 22, 2023, issue of Science Translational Medicine, the receptor, called glycoprotein 130 (gp130), regulates both positive and negative inflammatory responses that can help regenerate tissue, but also cause degeneration.
As James Peyer, the CEO of Cambrian Biopharma Inc., watched his grandfather fail every cancer treatment and pass away, he also saw the structure of his future company being born. Waiting until people became sick was the wrong way to treat disease, he found. Instead, Cambrian was created to develop anti-aging therapies, a unique business model that fits the new field of geroscience.
As broader markets struggled throughout 2022, the biopharma industry’s largest and most lucrative companies ended the year in a strong position, according to BioWorld’s Biopharmaceutical Index (BBI), which showed the 22 component stocks climbed by 17.3% throughout the year. Neither the Nasdaq...
The Biotechnology Innovation Organization (BIO) found in a new study that 77% of clinical programs focused on pain therapeutics five years ago are no longer active and that financings of companies working in the space are lackluster at best. Meanwhile, oncology companies, targeting an overall...
Starting out the year slow, biopharma financings are about 22% below where they were at the end of January last year, and they are well behind the first month of the previous two years as well, suggesting company executives may need to continue to prioritize costly programs as resources are...
While it made a sturdy effort as biopharma companies opted for licensing deals over M&As in 2022, the year did not surpass 2021 in deal values, falling about 3.5% short. Lackluster M&A values dropped to their lowest levels in five years and were down by 35.6% compared with 2021.
Mbiomics GmbH raised €13 million (US$14.2 million) in a first close of a series A round that will enable it to pivot from being a microbiome analytics firm to becoming a therapeutics developer.
A deficiency in fumarate metabolism could be behind a new mechanism of inflammation mediated by mitochondrial DNA and RNA. Two independent and simultaneous studies described how the accumulation of fumarate in the mitochondria released the genetic material of this organelle through vesicles,...
Somatic human genome editing has made huge strides in the past five years, but the likely extremely high prices will be unsustainable. A global commitment to affordable, equitable access is urgently needed because the costs and infrastructure needs of this form of treatment are not manageable for...
The researcher who pioneered prenatal surgery to correct neural tube defects has turned her attention to using CRISPR-edited gene therapies to correct severe monogenic diseases in utero. The availability of prenatal genetic diagnosis and advances in treating fetuses, and also in gene therapy/gene...
In Alzheimer’s, the amyloid beta hypothesis has proved most persistent in terms of drug development efforts to date, but aggregation of other pathogenic factors – phosphorylated tau (p-tau), APOE4, TREM2 and alpha-synuclein, for example – have also emerged as hallmarks of the disease. It’s that...
Newco Teitur Trophics ApS has raised €28 million (US$30.1 million) in a series A with which it will lay out a new route to targeting sortilin in the treatment of neurodegenerative diseases. The company is targeting the sortilin-related Vps10p domain containing receptor, which plays a role in...
In an ideal world, when a patient takes a medicine, it acts only at the specific site of disease in the human body whilst sparing healthy tissues. But it almost goes without saying that with many drug regimens, side effects or complications are part of the package. Working behind the scenes to...
Rapport Therapeutics Inc. launched with $100 million in series A funding and ambitious plans to bring a hitherto unprecedented level of precision to therapies for neurological disease. Although the Boston-based company is new to the world, its underlying platform has been a decade in the making,...
Newco Relation Therapeutics Ltd. is showing its colors after raising $25 million in a seed round to work on integrating single cell transcriptomics, functional genomics and machine learning – and cut through previously undecipherable combinatorial space – to find and validate drug targets in the...
Sarepta Therapeutics Inc. CEO Doug Ingram said the U.S. FDA has promised to schedule “expeditiously” an advisory committee meeting on the BLA related to SRP-9001 (delandistrogene moxeparvovec) for Duchenne muscular dystrophy (DMD).
Seamless Therapeutics GmbH raised $12.5 million in seed financing to take forward a novel gene editing technology based on reprogramming recombinase enzymes.
The U.K. arm of Silicon Valley Bank (SVB) is to be acquired by HSBC UK Bank plc for a symbolic £1, after senior ministers and the Bank of England worked all weekend on a rescue package. The acquisition is to complete immediately, averting what the U.K. government had acknowledged would be a crisis...
First the stick and now the carrots. The pharma industry in the U.K. on March 1 published its proposal for a new pricing scheme, under which it is offering to pay a fixed rebate of 6.88% across all eligible drug sales, an offer it said will deliver more than £1 billion (US$1.2 billion) per annum...
89bio Inc.’s top-line phase IIb data from the 216-subject study called Enliven testing pegozafermin in nonalcoholic steatohepatitis (NASH) pushed shares (NASDAQ:ETNB) to a closing price March 22 of $13.68, up $2.75, or 25%. The drug, due next for...
Astellas Pharma Inc.’s zolbetuximab, a monoclonal antibody targeting Claudin 18.2, met the primary endpoint for progression-free survival as well as secondary endpoints for overall survival in the phase III Glow trial in CLDN18.2-positive,...
Mereo Biopharma Group plc said its path to approval for alvelestat is a phase III study that would preclude an additional confirmatory trial. After meetings with the U.S. FDA and the EMA to discuss next steps, Mereo said it is designing a single,...
Selecta Biosciences Inc. and Swedish Orphan Biovitrum AB unveiled top-line phase III data from the Dissolve I and II trials testing SEL-212 in adults with chronic refractory gout (CRG) – results that position the companies for a regulatory filing...
Likang Life Sciences Holdings Ltd. received approval from China’s NMPA to start a clinical trial of its candidate, LK-101 injection, for advanced solid tumors. The company claims this is the first personalized neoantigen vaccine and mRNA editing...
With phase III data in hand from the Emergent-3 study of Karxt (xanomeline plus trospium) in adults with schizophrenia, Karuna Therapeutics Inc. continues to target mid-2023 for an NDA submission to the U.S. FDA. The 256-subject trial met its...
Breakthrough medicines, billion-dollar deals, spectacular clinical successes and crushing failures all play a part in biopharma’s dynamic story. Developers make scientific advancements with the potential to change everything, only to face regulatory conundrums and ever-fluctuating markets. BioWorld tracks key events in the fast-moving sector every business day. Now, the BioWorld Insider podcast lets you hear directly from the movers and shakers whose collective work is changing how we all live. Join us each week for a new conversation.