Neurodegenerative disease

Roche drops a third Alzheimer’s candidate this year, terminating a partnership with UCB just four years after agreeing to work together on new treatments for the neurological disease.
Wave Life Sciences in a Tuesday filing with the SEC said Takeda has elected to terminate its option to continue work on Wave’s WVE-003 clinical-stage Huntington’s disease program—a potential $5 billion commercial opportunity, according to the biotech.
Sage has decided to discontinue the development of dalzanemdor in Alzheimer’s disease. A study of the candidate in Huntington’s is ongoing, with early data expected later this year.
With Monday’s data from SAPPHIRE, Scholar Rock is building toward regulatory submissions for apitegromab in spinal muscular atrophy in the first quarter of 2025.
Results from TEMPO-1, which showed that tavapadon significantly improved motor symptoms in patients with Parkinson’s disease, will help AbbVie as it builds a regulatory case for the D1/D5 dopamine receptor agonist.
The FDA previously refused to review Biohaven’s candidate in the indication due to a failed late-stage trial. However, the company is now planning to file an NDA in the fourth quarter of 2024.
The positive readout in patients with non-relapsing secondary progressive multiple sclerosis comes on the heels of back-to-back failures in which tolebrutinib was unable to improve relapse rates in patients with relapsing MS.
FDA
The FDA has six target action dates ahead to round out September as drugs for gastroparesis, Niemann-Pick disease type C and more await decisions.
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.
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.”
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