Phase 2
The results are nevertheless “unprecedented,” UniQure executives said on an investor call Tuesday, emphasizing that the updated natural history database used as the control group for the Phase 1/2 study of AMT-130 underestimates disease progression.
Ten patients out of 20 who received Johnson & Johnson and Legend Biotech’s Carvykti remained alive at five years of follow up.
While Acadia Pharmaceuticals’ candidate has missed the main goal of a Phase 2 trial, the company is still moving forward with the anti-psychosis candidate in Alzheimer’s disease as well as Lewy body dementia.
Analysts were keen to see a new modality compete with Roche’s approved drug Lucentis in the late-stage trial but new safety issues could complicate the future for Merck’s candidate.
Kyverna Therapeutics’ miv-cel showed improved mobility and a favorable safety profile at one year in patients with stiff person syndrome, further derisking an upcoming FDA application, William Blair said on Thursday. The biotech plans to file for priority review this year.
After imeroprubart failed to demonstrate sufficient efficacy in lupus during a Phase 2b trial, Immunovant is moving on from the indication and looking to other high priority programs for the drug in neurology, endocrinology and rheumatology.
With just over $10 million in cash available to fuel operations through the end of the year, Longeveron is undertaking a strategic review after the failure of a cardiovascular study.
The TREM2 stabilizer failed to show efficacy in a Phase 2 trial of patients with early-stage ALS. This development follows two late-stage trial failures reported by Novartis last week, plus the news that the pharma has paused multiple CAR T cell trials following patient deaths.
The polycythemia vera field is heating up, with Disc Medicine and Silence Therapeutics each advancing next-generation therapies, playing catch-up to Takeda and Protagonist Therapeutics, who won the FDA’s approval last month for Mimrylo.
The enrollment pause comes after BHV-7000 failed to show efficacy in a midstage depression trial late last year. The news is another regulatory hit for Biohaven after the FDA declined to approve its spinocerebellar ataxia treatment in November 2025.
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