Neuroscience
Recent breakthroughs and three decades of progress in treating Huntington’s disease
Last month, the FDA declined to approve Sanofi’s tolebrutinib for a specific form of multiple sclerosis. In a recently published complete response letter, the agency detailed its reasoning behind the rejection.
After a strong open to the year, the public markets suffered a six-month drought that led to biotech’s tightest IPO window in years.
As 2026 begins, a slate of high-stakes clinical readouts—from a pivotal study of Novartis’ cardiovascular candidate pelacarsen to a Phase III test of Eli Lilly’s next-gen Alzheimer’s drug—are poised to reshape therapeutic landscapes.
The major depressive disorder failure for BHV-7000 is the drug’s second, after Biohaven’s spinocerebellar ataxia treatment troriluzole was rejected by the FDA in November 2025.
Despite the definitive failure of Novo Nordisk’s semaglutide in Alzheimer’s, biotech executives, analysts and other industry experts see potential in more testing of GLP-1s for the neurodegenerative disease, particularly in a combination approach.
Analysts said the outcome is disappointing because there are no approved treatments for dyskinetic cerebral palsy, but the setback had little impact on Neurocrine’s valuation.
A report from analysts at Jefferies suggested that new screenings for metachromatic leukodystrophy and Duchenne muscular dystrophy could bump sales of the gene therapy Libmeldy by more than $100 million.
The Massachusetts biotech will also be eligible to milestone payments, though the exact amount has yet to be disclosed.
The star of the agreement targets a specific type of tau protein, helping to prevent toxic accumulation in the brain while also preserving the function of healthy tau.
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