Neuroscience

Ono Pharma’s ONO-1110 failed five Phase 2 trials, leading to the discontinuation of development in depression and social anxiety. Work will continue for three pain indications.
Novartis remains “very happy” with its $12 billion acquisition of Avidity Biosciences, despite a Phase 3 disappointment for the antibody-oligonucleotide conjugate del-desiran.
The disclosure—which came ahead of a presentation of data from the Phase 1 Breakfree-2 study at the ACTRIMS-ECTRIMS meeting—follows Bristol Myers Squibb’s late summer revelation that it had paused certain trials of zolacabtagene autoleucel.
RNAi specialist Alnylam Pharmaceuticals will advance the Huntington’s disease drug brezosiran into a placebo-controlled study in 2027 once regulatory alignment is achieved.
FDA
Troriluzole, in development for spinocerebellar ataxia, was rejected nearly a year ago when the FDA took issue with the design of Biohaven’s registrational study. The agency has now agreed to review a new application and render a decision in the first quarter of 2027.
Rapport Therapeutics clocked its second Phase 2 win in 14 months for RAP-219—which the company is positioning as a potential pipeline-in-a-product—this time showing improvement in symptoms of bipolar mania.
Arrowhead Pharmaceuticals’ siRNA candidate achieved “compelling” tau reductions in an early study of healthy volunteers, with a magnitude of effect that seems to outperformother blood-brain barrier platforms, according to Stifel.
While Oppenheimer doesn’t see the Phase 3 stumble of VQW-765 as highly detrimental to Vanda Pharmaceuticals, the analyst nevertheless believes the stakes are now higher for the company’s planned Q4 readout for Nereus in GLP-1-induced vomiting.
After one patient death prompted an FDA hold this spring, Newron Pharmaceuticals has now shared that a total of four fatalities have been recorded among participants receiving its schizophrenia candidate. A fifth death was recorded among those taking the placebo.
The Takeda spinout showed that a high dose of solengepras can shorten the periods during which difficult Parkinson’s disease symptoms like involuntary movements and sleepiness.
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