Phase II
Several companies will head to the FDA seeking approval of new Duchenne muscular dystrophy treatments next year but the death of a patient taking Sarepta’s Elevidys raises important safety questions.
WVE-N531, an oligonucleotide, elicited significant functional benefit and reversal of muscle damage in the Phase II FORWARD-53 trial. Wave plans to file for accelerated approval of the candidate in 2026.
The company, launched with help from ex-Novartis executives, is targeting glutamate signaling in the brain to help treat alcohol- and cocaine-use disorders, among other indications.
Since 2016, the FDA has approved three disease-modifying treatments for spinal muscular atrophy, with several companies—including Novartis, Scholar Rock and Biogen—progressing novel candidates through clinical trials.
It’s early days for xenotransplantation, but eGenesis, Eledon, United Therapeutics and more are working to develop solutions to make this approach a viable option and help ease the organ shortage crisis.
Dyne is eyeing an accelerated approval filing for DYNE-251 in early 2026 that would pit the asset against Sarepta’s Exondys 51 in a patient population amenable to exon 51 skipping.
Having established success in cancer, biopharma is now looking to leverage CAR T therapies against a new target, autoimmune disorders, with several early- to mid-stage readouts expected this year.
Gilead plans to go straight to Phase III studies for once-yearly lenacapavir, while GSK and ViiV will push forward with their long-acting antivirals after touting positive early-stage results.
BEAM-302 “has set the bar for efficacy in this space,” William Blair analysts wrote in an investor note on Monday.
Mineralys’ shares got a more than 50% boost Monday morning as new data showed its hypertension drug reduced blood pressure in a pair of clinical trials.
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