Neuroscience
Acadia Pharmaceuticals was testing the drug, an intranasal formulation of the oxytocin analogue carbetocin, for its potential to ease hyperphagia in the rare neurological condition.
If approved, uniQure’s gene therapy AMT-130—which slowed disease progression by 75%—would be the first genetic treatment for Huntington’s disease. A BLA submission is planned for the first quarter of 2026.
The agency also pointed to the use of Tylenol and other acetaminophen products during pregnancy as being potentially linked to neurological and developmental defects in children, following a press conference Monday in which President Donald Trump did the same.
By improving gait stability, Ionis’ zilganersen could be “potentially disease modifying,” according to analysts at William Blair.
The company was expecting a decision from the FDA by Sept. 28 for its oral drug tolebrutinib, but an update to the drug’s application package convinced the agency to take more time to review.
MapLight Therapeutics, the second biotech to launch a Nasdaq bid this month, is a neuro-focused company advancing treatments for Alzheimer’s disease psychosis and schizophrenia.
With AbbVie’s $1.2 billion acquisition of Gilgamesh Pharmaceuticals’ lead depression drug, the psychedelic therapeutics space has soundly rebounded from Lykos’ rejection last year. There are now seven programs in Phase III trials across the sector, with multiple companies vying for that first approval.
VectorY Therapeutics will evaluate the use of SHP-DB1, a capsid developed by Shape Therapeutics, to deliver therapies to the brain, including VectorY’s developmental Huntington’s and Alzheimer’s disease treatments.
LB Pharma landed on the Nasdaq Thursday, with 3 million additional shares sold than expected.
Capsida has yet to disclose the exact cause of death. The patient had received the gene therapy CAP-002 for a type of epilepsy.
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