Neuroscience
ITF, IntraBio and Orchard are among the companies that have won FDA nods in the past year for Duchenne muscular dystrophy, Niemann-Pick disease type C, metachromatic leukodystrophy and more.
Emalex is gearing up for a New Drug Application for ecopipam in Tourette syndrome later this year.
BridGene strikes another partnership with Takeda as the latter company continues its dealmaking streak, following high-ticket agreements with Keros Therapeutics, AC Immune and Degron Therapeutics in the past nine months.
The treatment, called DB-OTO, is one of several early-stage gene therapies being developed to treat relatively straight-forward causes of genetic deafness.
After SPN-820’s failure, Supernus is relying on its non-stimulant ADHD drug Qelbree and the recently approved Parkinson’s therapy Onapgo to sustain the company.
The partnership splits the rights to Stoke’s epilepsy antisense oligonucleotide, with up to $385 million in potential payments due to Stoke.
Helmed by Roche alums, Newleos Therapeutics is taking over four drugs dropped from the Swiss pharma’s pipeline in early 2024.
Biogen and Eisai have spent much of Leqembi’s launch convincing physicians and patients that it’s safe to treat Alzheimer’s disease. With patients now hitting the 18-month mark of treatment, the conversation is finally shifting to efficacy.
The approval comes days after Germany’s Merck KGaA confirmed it was in advanced talks to acquire SpringWorks.
Leqembi’s sales in the U.S. continue to underwhelm, overshadowed by its growth in international markets.
PRESS RELEASES