Neurodegenerative disease
While taldefgrobep alfa failed to show improved motor function in spinal muscular atrophy, treated patients saw a marked reduction in body fat. Biohaven plans to launch a Phase II trial in obesity by the end of the year.
Cassava Sciences has revealed the late-stage clinical failure of controversial Alzheimer’s drug simulfilam. The company had pledged to share the results whether “good, bad or ambiguous.”
Sage Therapeutics discontinued development of its lead candidate dalzanemdor after a third clinical failure, leading analysts to question the biotech’s future profitability.
After previously failing studies in Parkinson’s and Alzheimer’s, dalzanemdor’s latest stumble in Huntington’s disease has pushed Sage Therapeutics to pull the plug on the NMDA receptor modulator.
Trump fingers Robert F. Kennedy Jr. to lead the HHS, lupus and ATTR-CM dominate headlines this week, bluebird bio has a cash gap to leap and RegenxBio eyes Sarepta in Duchenne muscular dystrophy.
Analysts did not seem very concerned by the treatment-related serious adverse event, noting that NGN-401 was well-tolerated at a lower dose and showed promising efficacy outcomes.
This year has seen several biopharma companies drop Alzheimer’s and Parkinson’s disease programs, but experts say plenty are still chasing these multi-billion-dollar markets.
Approved by the FDA in July to treat adults with early symptomatic Alzheimer’s disease, Eli Lilly’s Kisunla reduced the brain swelling of patients in a late-stage trial following an adjusted dosing regimen.
Researchers have linked Novo Nordisk’s semaglutide to a 40% to 70% reduction in the risk of Alzheimer’s disease diagnosis in a study of medical records from 1 million patients.
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.
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