Two weeks after Pfizer Inc.-Biontech SE’s mRNA-based COVID-19 vaccine received emergency use authorization (EUA) for adolescents ages 12 to 15, the first in that age group, Moderna Inc.’s mRNA vaccine has hit the primary immunogenicity endpoint in its phase II/III study of participants ages 12 through 17.
Missing the primary endpoint in a phase IIb study of aldafermin for treating nonalcoholic steatohepatitis (NASH) has caused NGM Biopharmaceuticals Inc. to halt the program’s development while rattling the company’s share value. But the failure didn’t stop there as other NASH therapy developers felt the reverberations. South Francisco-based NGM’s stock (NASDAQ:NGM) took a battering May 24 as shares closed down 40.77% at $16.81 each. Others in the NASH space rocked by NGM’s negative data on the day included San Francisco-based 89bio Inc., which is prepping BIO89-100, a glycopegylated FGF21 analogue for a phase IIb NASH study. 89bio’s stock (NASDAQ:ETNB) plunged 14.24% to close at $19.40 per share.
The EMA issued a positive scientific opinion on Glaxosmithkline plc (GSK) and Vir Biotechnology Inc.’s sotrovimab for early COVID-19 treatment. The Committee for Human Medicinal Products (CHMP)’s opinion concerns the monoclonal antibody’s use for adolescents ages 12 and older weighing at least 40 kg (88 pounds), plus adults. All must risk progressing to severe COVID-19 and not require oxygen supplementation.
Anixa Biosciences Inc. and the Moffitt Cancer Center now have details the FDA said it needs regarding their CAR T therapy development program. The collaborators said the information can be provided to the FDA in about 30 days. The agency then has 30 days to respond.
There’s been a change of plans. Following a preliminary review of briefing materials for a type C meeting, the FDA told Reata Pharmaceuticals Inc. that a pre-NDA meeting is instead the next best step in the development of omaveloxolone (RT-408) for treating Friedreich’s ataxia (FA).
Interius Biotherapeutics Inc., which just closed on an oversubscribed $76 million series A financing, is fixed on simplifying CAR T-cell creation by making the process less cumbersome, less expensive and time consuming, and all done in vivo.
Now that Apellis Pharmaceuticals Inc.’s Empaveli pegcetacoplan has won FDA approval as the first targeted C3 therapy for treating paroxysmal nocturnal hemoglobinuria (PNH), the cost of treating the rare blood disorder is hefty.
Little more than two years ago, Biogen Inc. plunked down about $800 million for gene therapy developer Nightstar Therapeutics plc, which had an X-linked retinitis pigmentosa drug in its pipeline. Top-line results from the phase II/III Xirius study of cotoretigene toliparvovec (BIIB-112), administered by subretinal injection, show the one-time therapy missed its primary endpoint of producing a statistically significant improvement in the proportion of treated study eyes.
It took a few years and three tries, but Heron Therapeutics Inc. finally got its pain drug, HTX-011, now branded Zynrelef, over the FDA finish line. Even so, the approval comes with a less broad label than Heron would have liked. Zynrelef (bupivacaine and meloxicam), which had a May 12 PDUFA date, is approved for use in adults for soft tissue or periarticular instillation to produce postsurgical analgesia for up to 72 hours after bunionectomy, open inguinal herniorrhaphy and total knee arthroplasty.
Bolstered by new and positive open-label extension data, Minerva Neurosciences Inc. won’t let a failed phase III of roluperidone in treating schizophrenia get in the way of considering an NDA filing.
