UniQure’s Phase I/II interim readout in Huntington’s disease showed improvements on functional and clinical measures, but mixed results on a pair of biomarkers.
UniQure N.V. said it is pleased with a new interim readout from its Phase I/II US trial of AMT-130, a gene therapy candidate for Huntington’s disease, but the response of its investors on 21 June suggests concern about a dataset that at least one analyst called “mixed.”
Data from the 26-patient study showed preserved function from baseline and clinical benefits compared to the natural history of Huntington’s...
Create an account to read this article
Already a subscriber?
CSO Jacob Thyssen tells Scrip that the monoclonal antibody which blocks the IL-22RA1 receptor subunit is a very safe and effective option to treat the chronic inflammatory skin disease.
With patent expiry for its blockbuster blood cancer drug Jakafi on the horizon, Incyte will need to translate these povorcitinib wins from clinic to market.
The company announced positive Phase III data for the drug in fibrodysplasia ossificans progressiva, as well as Phase II combination data among patients with obesity.
Positive Phase III data positions Roivant to launch the oral TYK2/JAK1 inhibitor for dermatomyositis, with an NDA filing planned in early 2026.
Positive Phase III data positions Roivant to launch the oral TYK2/JAK1 inhibitor for dermatomyositis, with an NDA filing planned in early 2026.
AstraZeneca’s IL-5 blocker has again missed in COPD, adding to a number of disappointments for biologics in this disease. Complex biology and overlapping inflammatory pathways seem to be at the heart of the problem.
The company’s cardiometabolic leader Ken Custer discusses the topline ACHIEVE-3 results in diabetes and just-published full results of ATTAIN-1 in obesity of orforglipron, with a pledge to maximize access to the oral GLP-1 therapy worldwide, once approved.
