Phase II
The decision to stop the Phase IIb study was driven by “strategic business reasons,” according to a federal clinical trials database.
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.
Analysts at BMO Capital Markets expect the lack of other exon-44-skiping therapies to facilitate a “smooth” approval process for Avidity’s del-zota.
Truist Securities called pumitamig’s data on Monday “very reassuring,” given the consistency between its performance in Chinese and global patient populations.
Presenting at the World Sleep Congress 2025, the Dublin-based company’s Phase II study bested Takeda drug in both efficacy and safety.
Shares of Rapport Therapeutics popped Monday morning after Phase IIa data for RAP-219 exceeded analyst and Wall Street expectations, reducing seizures by almost 78% in patients with drug-resistant focal onset seizures.
According to analysts, the new data could present a path to accelerated approval for ifinatamab deruxtecan, a product of Merck and Daiichi Sankyo’s troubled ADC partnership.
The French giant is gaining access to darovasertib, a small molecule protein kinase C inhibitor already in Phase II/III trials, with rights for the whole world besides the U.S.
Closely watched data from Eli Lilly and Viking Therapeutics this month have reignited the discussion around oral weight-loss drugs—and their ultimate place within the anti-obesity medication market.
Vedanta is parting ways with 23 employees, or approximately 20% of its headcount, after Phase II data for microbiome therapy VE202 failed to demonstrate significant response rates in patients with ulcerative colitis.
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