Neurodegenerative disease
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.
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.”
Sangamo Therapeutics announced Tuesday it secured an exclusive licensing agreement with Roche’s Genentech, which is paying $50 million in near-term upfront fees and milestone payments to develop novel genomic medicines for neurodegenerative diseases.
Longeveron and Lexeo Therapeutics are working on CGT therapies to treat Alzheimer’s disease, but it’s not clear whether they have a better chance of success than traditional approaches.
Eisai presented a plethora of data on the drug at the Alzheimer’s Association International Conference, including a study showing the consequences of pausing treatment.
Halia Therapeutics, NodThera and Gain Therapeutics target neuroinflammatory processes in hopes of modifying the course of Parkinson’s progression.
After a busy first half of 2024, several companies are expecting key data readouts in the neuropsychiatric and neurodegenerative disease spaces during the next six months.
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