Trump fingers Robert F. Kennedy Jr. to lead the HHS, lupus and ATTR-CM dominate headlines this week, bluebird bio has a cash gap to leap and RegenxBio eyes Sarepta in Duchenne muscular dystrophy.
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President-elect Donald Trump announced last week that he would nominate prominent anti-vaxxer Robert F. Kennedy Jr. to lead the HHS. While this news caused a dip in biotech stocks, one analyst said the selloff was an overreaction.
Meanwhile, fall conference season continues with the American College of Rheumatology Convergence (ACR) and American Heart Association’s 2024 Scientific Sessions (AHA). Lupus was the indication du jour at ACR, where BMS, Kyverna, Fate Therapeutics and Cabaletta Bio presented data from early-stage trials of their CAR T candidates, and Biogen and UCB detailed data behind their unexpected late-stage victory for dapirolizumab pegol. This was a much-needed win for Biogen, which has seen its shares decline 36% this year.
Neurogene’s stock, meanwhile, tumbled 36% on Monday alone, following the release of details about an adverse event in a trial for its Rett syndrome gene therapy. This followed a 35% fall last week when the adverse event was first announced. And the company decided to drop its gene therapy in Batten disease after the FDA declined to grant Regenerative Medicine Advance Therapy designation to support the program.
We also took a look at bluebird bio’s cash problem, with the company slated to exhaust its runway to reach a breakeven point before the end of next year.
On a more positive note, RegenxBio announced this week it has aligned with the FDA on path to possible accelerated approval for its investigational gene therapy for Duchenne muscular dystrophy, following behind Sarepta’s Elevydis, which faced controversy after it received accelerated approval in June 2023, only to miss the primary functional endpoint in its confirmatory trial four months later. And speaking of accelerated approval, we released a special edition of ClinicaSpace Monday focused on this very topic. Sign up to receive it here.
