Neuroscience

The gene therapy world is in turmoil, but Arbor, armed with more than $1 billion in partnerships and raises, is going forward.
Ionis and Ultragenyx are competing to develop oligonucleotide treatments for Angelman syndrome, but will Neuren’s peptide catch up?
Days after suffering a rejection in Australia, the Alzheimer’s drug hit another roadblock in the U.K., which found the drug not cost-effective.
Analysts do not believe the Phase III stumble for aticaprant will derail J&J’s broader neuroscience strategy, particularly given its recent $14.6 billion acquisition of Intra-Cellular Therapeutics and the success of Spravato for treatment-resistant depression.
The new formulation of Keytruda, currently under FDA review, is sparking conflict with Halozyme, which makes enzymes that convert intravenous drugs into injectable versions.
Biohaven in recent months has reported a clinical stumble in spinal muscular atrophy, alongside a Phase I readout for its protein degrader candidate that investors found underwhelming.
After failing to hit the primary endpoint in a Phase III trial, Neumora is remixing study parameters in two replicate trials, with data expected in the first half of 2026.
Despite not differentiating itself from placebo, the Texas-based company said it plans to push pilavapadin into Phase III trials before long.
Leqembi’s application now moves forward to the European Commission, which will issue a formal verdict for the injection that will apply to all EU member states as well as Norway, Liechtenstein and Iceland.
Mission Therapeutics is down to its clinical assets MTX652 and MTX325, which work by disabling a key enzyme that interferes with the cell’s normal process of removing faulty or dysfunctional mitochondria.
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