Gender bias in cardiac treatment guidelines is putting women at risk because guidelines are written based on clinical trials conducted mostly in men. As previously reported in BioWorld, nearly 70% of female patients are underdiagnosed for cardiovascular disease as women are grossly under-represented in clinical trials. Cardiac guidelines for treatments are binary numbers that don’t consider physiological differences between men and women, Echo IQ Chief Commercial Officer Deon Strydom told BioWorld.
Syndax’s Revuforj wins FDA go-ahead in leukemia
Ahead of its Dec. 26 PDUFA date, Syndax Pharmaceuticals Inc. scored U.S. FDA approval of Revuforj (revumenib) as the first and only menin inhibitor for relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene translocation in adult and pediatric patients one year and older. The FDA had previously tagged the drug as a breakthrough therapy and given it fast track designation as well as priority review. The NDA was cleared by way of the FDA's Real Time Oncology Review program. Shares of Waltham, Mass.-based Syndax (NASDAQ:SNDX) were trading at $16.11, up 28 cents.
Biosion sells antibody rights to Aclaris in $940M deal
Biosion Inc. is getting more than $40 million in cash up front from Aclaris Therapeutics Inc. for the rights to develop two antibodies. The regulatory and sales milestones could also bring Biosion another $900 million. The two assets are BSI-045B, a high affinity and high potency anti-TSLP monoclonal antibody, and BSI-502, a bispecific antibody directed against TSLP and IL4R. The deal is for global rights that exclude China. BSI-045B just wrapped up a single-arm, proof-of-concept phase IIa study in moderate to severe atopic dermatitis. Aclaris’ stock (NASDAQ:ACRS) got a solid boost at midday as shares were up 35% at $2.75 each.
Novartis continues push into radiopharma with $745M Ratio deal
With two approved radioligand therapies on its commercial roster and a recent plan to boost its manufacturing for radiotherapeutics, Novartis AG tapped Ratio Therapeutics Inc. in its latest deal. The companies entered an exclusive worldwide license and collaboration agreement to develop a somatostatin receptor 2 radiotherapeutic candidate for cancer, with Ratio eligible for up-front and potential milestone payments totaling $745 million, plus royalties. It also marks the latest collaboration for Ratio, which emerged from stealth only two and a half years ago, and has diagnostic-focused deals with Lantheus Holdings Inc. and Merck & Co. Inc.
The Liver Meeting: Arbutus’ cure for hepatitis B’s low-hanging fruit
At The Liver Meeting 2024, Arbutus Biopharma Corp. presented data from the phase IIa Im-prove study showing its RNAi drug, imdusiran, in combination with pegylated interferon alfa-2α and nucleos(t)ide analogue therapy, was able to cure patients of their chronic hepatitis B infection. The 50% cure rate is impressive compared to the approximately 5% cure rate with the current standard of care, but there were some caveats, including that the cure rate is based on just six patients and those patients started with levels of HBsAg levels less than 1,000 IU/mL. By contrast, none of the six patients with higher HBsAg levels were cured.
Neurogene slides on Rett syndrome gene therapy safety concern
Neurogene Inc.’s stock sank 43.5% in early trading Nov. 18 on news that its phase I/II gene therapy NGN-401 resulted in a serious adverse event in a Rett syndrome patient receiving the highest dose. The female patient received the therapy Nov. 5, subsequently experienced signs of a systemic hyperinflammatory syndrome, and is in critical condition. The New York-based company discontinued the high dose of 3E15 vg, but will continue the trial with the lower dose of 1E15 vg. Shares (NASDAQ:NGNE) were trading midday at $19.50, down $15.01. NGN-401 is the company’s lead program.
Keymed, Platina in $626M deal for bispecific antibody CM-336
Keymed Biosciences Co. Ltd. is out-licensing global rights, excluding China, for its bispecific antibody, CM-336, to Platina Medicines Ltd. (PML) in a deal worth up to $626 million plus sales royalties. Under terms of the deal, PML gains an exclusive global license for the research, development, registration, manufacturing, and commercialization of CM-336 excluding mainland China, Hong Kong, Macau and Taiwan. In return, Keymed will receive an up-front payment of $16 million, a minority equity interest in Ouro Medicines LLC (PML’s parent company), and up to $610 million for achieving certain clinical, regulatory and commercial milestones plus additional sales-based royalties.
Biopharma deal value down for year, but surges 72% in October
Biopharma deal activity in October totaled $22.31 billion, marking a 72% jump from $13.01 billion in September. Year-to-date deal values have decreased by 2%, from $175.29 billion during the first 10 months of 2023 to $171.77 billion in the same period this year, positioning 2024 as the second-highest year on record, according to BioWorld. The average monthly deal value in 2024 stands at $17.18 billion, slightly below the 2023 full-year monthly average of $18.14 billion.
‘A decade of innovation, a decade to come’ for China pharma
As China rises to second place, next only to the U.S., for innovative new drugs, a new report by Clarivate — A decade of innovation, a decade to come — outlines key policy reforms and regulatory, R&D and investment trends driving past and future growth of Chinese biopharmaceuticals. The 73-page report highlights innovation and challenges in China’s biopharmaceutical sector as the country rolls into its 10th year since launching a comprehensive drug review and approval reform in 2015.
BioWorld Insider podcast: Modifi gets creative on the rocky road to a multibillion-dollar buyout
For a small company, Modifi Biosciences has come a long way very quickly. In the newest episode of the BioWorld Insider podcast, founder Ranjit Bindra talks about how his company went from tiny to nearly extinct at least twice until it was acquired by Merck for $30 million up front.
Also in the news
89bio, Adaptimmune, Agomab, Akari, Alveogene, Aurinia, Beigene, Biogen, Bluebird, Bristol Myers Squibb, Cassava, Celltrion, Cyclacel, Diakonos, Evotec, Exevir, Eyenovia, Halozyme, Inflarx, Iqone Healthcare, Johnson & Johnson, Kaken, Kane, Levicept, Lixa, Medicus, Merck, Numab, Omega, Osteal, Pacira, Peak, Regeneron, Samsung Bioepis, Sandoz, Sanofi, Sea, TCG Greenchem, TFF, Theras, Vera, Zai Lab