Agios' mitapivat succeeds in late-stage study in rare blood disorder

 

• A Phase 3 clinical trial, ACTIVATE, evaluating Agios Pharmaceuticals' (NASDAQ:AGIO) mitapivat in adults with pyruvate kinase (PK) deficiency who do not receive regular transfusions met the primary endpoint.
• Specifically, 40% of treated patients achieved a hemoglobin response at weeks 16, 20 and 24 compared to 0% in the control group (p<0.0001).
• Key secondary endpoints were also met.
• No new safety signals were observed.
• Complete results will be submitted for presentation at the European Hematology Association Virtual Congress in June 2021.
• The company plans to file marketing applications in the U.S. and Europe in 2021.
• Mitapivat is an orally available activator of wild-type and mutated pyruvate kinase-R enzymes.
• https://seekingalpha.com/news/3640320-agios-mitapivat-successful-in-late-stage-study-in-rare-blood-disorder