Solid Biosciences Inc. is preparing for a sit-down with the U.S. FDA this year to discuss the firm’s results with the next-generation gene therapy SGT-003 for Duchenne muscular dystrophy (DMD).
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.
The former head of the U.S. NIH has sounded a warning that the uncertainty caused by the Trump Administration’s funding cuts and layoffs is blocking “critical work” and “paralyzing” biomedical research. “Every time we launch a new program, every time we continue to commit resources to ongoing work, those are important decisions that we make every single day, and in times like this, that decision-making is paralyzed,” said Monica Bertagnolli, who stood down as director of the NIH on Jan. 17.
The first clinical data from an FDA-approved trial of a CAR T-cell therapy in the treatment of multiple sclerosis (MS) show the cells cause complete B-cell depletion, leading on to a reset of the immune system that is accompanied by an improvement in symptoms.
As part of a U.S. government-wide reduction in force aimed at restructuring and streamlining federal agencies, 5,200 Health and Human Services employees reportedly received their pink slips over the weekend, with 1,165, or 22%, of those at the NIH.
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.
Septerna Inc.’s stock plunged as much as 68% throughout the day Feb. 18 on news that the company was stopping a phase I trial of SEP-786 in healthy volunteers following two severe events of elevated unconjugated bilirubin in the highest dose cohort of the multiple ascending-dose portion of the study.
Becoming the second drug approved by the U.S. FDA for tenosynovial giant cell tumors, Deciphera Pharmaceuticals Inc.’s oral CSF1R inhibitor vimseltinib, newly branded Romvimza, could take significant market share from a competitor in the same class. A phase III trial showed a higher objective response rate with Romvimza than a separate phase III trial showed with Daiichi Sankyo Co. Ltd.’s Turalio (pexidartinib), and the latter’s label contains a boxed warning on rare but serious and sometimes life-threatening liver side effects.
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