Neuroscience
The investigational injection fosgonimeton appeared to have better efficacy in patients with more severe disease, according to post-hoc subgroup analyses, though none resulted in statistically significant effects.
Recursion’s oral drug candidate for cerebral cavernous malformation showed no improvements in patient- or physician-reported outcomes at 12 months. The biotech will engage with the FDA to determine the need for an additional study.
The BTK inhibitor showed promise in non-relapsing secondary progressive multiple sclerosis but not relapsing MS. The company said it plans to apply for approval for the former “as soon as possible.”
The FDA has three regulatory milestones in the next two weeks, including a decision on a subcutaneous formulation of an effective multiple sclerosis therapy.
Neurocrine Biosciences’ potential competitor to Bristol Myers Squibb’s KarXT improved symptoms of schizophrenia in a Phase II trial, but only at the low dose tested.
While approved by the Medicines and Healthcare products Regulatory Agency, the Alzheimer’s drug failed to win the backing of the U.K.’s National Institute for Health and Care Excellence, which said that its benefits were “too small to justify the cost.”
Psychedelic drug developers are undeterred by the FDA’s Complete Response Letter for the company’s MDMA therapy for PTSD, and experts expect Lykos will ultimately obtain approval.
On the heels of last week’s FDA rejection of Lykos’ MDMA-assisted PTSD therapy, Atai announced Tuesday positive preliminary results for its DMT-based treatment for depression from a Phase Ib study.
Crexont was previously rejected by the regulator in June 2023, citing insufficient safety data. However, Amneal Pharmaceuticals’ resubmission included findings from a healthy volunteer study.
Experts say the time is now to develop and provide widespread access to genetic medicines for the rarest diseases. What’s more, they say it is a moral imperative.
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