Agios Phase 3 Trial Shows Positive Results for Anemia Drug

Agios Pharmaceuticals has announced that its global Phase 3 ACTIVATE trial of mitapivat met its primary endpoint. ACTIVATE is a double-blind, placebo-controlled clinical trial evaluating the efficacy and safety of mitapivat as a potential treatment for adults with pyruvate kinase (PK) deficiency, a chronic, lifelong hemolytic anemia, who do not receive regular transfusions. Treatment with mitapivat demonstrated a statistically significant, sustained increase in hemoglobin compared to placebo.

Agios anticipates filing for U.S. and EU regulatory approval in adults with PK deficiency in 2021, with a potential 2022 commercial launch in both geographies. Mitapivat has been granted orphan drug designation for the treatment of PK deficiency by the FDA and the European Medicines Agency. Additionally, mitapivat has received orphan drug designation from the FDA for the treatment of thalassemia and sickle cell disease.

“The robust, clinically meaningful efficacy and safety results from the ACTIVATE study underscore mitapivat’s potential to be the first disease-modifying therapy for people with pyruvate kinase deficiency, a chronic, lifelong hemolytic anemia that often leads to serious physical and quality of life complications. With only supportive therapy currently available, there is tremendous unmet need in this community, and we are proud to advance a promising therapeutic candidate for these patients…,” said Chris Bowden, M.D., Agios Chief Medical Officer. (Source: Agios Website, 02 December, 2020)