In Regeneron Pharmaceuticals Inc.’s phase I/II Chord study of 12 children with genetic hearing loss, 10 out of 11 have shown improvements after being treated with a gene therapy. “What is really remarkable about this type of therapeutic approach is that the first people who are going to see the impact are not actually the physicians – it’s the families,” Jonathon Whitton, vice president and Regeneron’s auditory global program head, told BioWorld.
Artificial intelligence (AI) technology developer Genesis Therapeutics Inc. has brought in another larger partner to go on a search for the right therapeutic small molecules. The company is getting an up-front $30 million payment from Incyte Corp. and could bring in as much as $295 million per target in development, regulatory and commercial milestones, bringing the deal to about $620 million total.
The phase IIb study results of Supernus Pharmaceuticals Inc.’s SPN-820 in treatment-resistant depression had pulled the company’s stock (NASDAQ:SUPN) down 15.6% on Feb. 19. The randomized, double-blind, placebo-controlled study of adults failed to show a statistically significant improvement on the primary endpoint of change from baseline on the Montgomery-Åsberg Depression rating scale. Shares closed at $33.52 apiece on Feb. 19.
With Gilead Sciences Inc. stepping away from its exclusive option rights to casdatifan in treating cancer, Arcus Biosciences Inc. has decided to move on by pricing a $150 million common stock offering. Arcus said Gilead’s rights to the HIF-2a inhibitor, which just produced positive early phase data, had expired.
With the U.S. FDA’s approval of Vimkunya, Bavarian Nordic A/S is now in the chikungunya virus infection mix along with Valneva SE’s Ixchiq. A single-dose, adjuvanted virus-like particle-based vaccine to protect against the chikungunya virus infection, Vimkunya is the first of its kind available in the U.S. for those age 12 and older.
A second pentavalent vaccine for preventing meningococcal disease has been approved by the U.S. FDA. GSL plc’s Penmenvy will now go up against Pfizer Inc.’s Penbraya, which had a two-year head start in the market.
The market took a strong liking to the latest U.S. FDA guidance for Moleculin Biotech Inc.’s upcoming Miracle phase III trial testing annamycin in cancer. So much so that the company’s stock (NASDAQ:MBRX) soared 202% on Feb. 13 to close at $1.27 a share.
Novartis AG is paying nearly $1 billion up front to buy privately held Anthos Therapeutics Inc. in a deal that eventually could top out around $3.1 billion. Novartis, coming back to where it started as it and Blackstone Life Sciences founded Anthos in 2019, is entering a crowded space.
4D Molecular Therapeutics Inc.’s gene therapy, 4D-150, in wet age-related macular degeneration (wet AMD) produced positive phase IIb data as the company preps two phase III studies set to begin this year.
