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Agios Announces Updated Data from ACTIVATE and ACTIVATE-T Phase 3 Studies of Mitapivat in Pyruvate Kinase (PK) Deficiency at the European Hematology Association Virtual Congress
– Company Expects to File for Regulatory Approval for Mitapivat for the Treatment of Adults with PK Deficiency in the U.S. This Quarter and in the EU in
About this update from Agios Pharmaceuticals, Inc.
[{"type":"text","content":"– Company Expects to File for Regulatory Approval for Mitapivat for the Treatment of Adults with PK Deficiency in the U.S. This Quarter and in the EU in Mid-2021 – – Agios to Host Investor Webcast Today at 7:30 a.m. ET – CAMBRIDGE, Mass., June 11, 2021 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat genetically defined diseases, today reported a full analysis of updated data, including patient-reported outcome (PRO) measures, from its global Phase 3 ACTIVATE and ACTIVATE-T studies of mitapivat in adults with pyruvate kinase (PK) deficiency. Data from the studies will be featured in oral presentations on Tuesday, June 15, at the European Hematology Association (EHA) Virtual Congress. Consistent with previously announced topline data, the ACTIVATE and ACTIVATE-T studies met primary and secondary endpoints, including PRO outcomes that address symptom burden and quality-of-life impact of PK deficiency in adults. The safety profile observed in both studies was generally consistent with previously published data. Mitapivat is a first-in-class, investigational, oral, small molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase R (PKR) enzymes. “Results from the ACTIVATE and ACTIVATE-T Phase 3 studies underscore the potential of mitapivat to be the first disease-modifying therapy for individuals with pyruvate kinase deficiency, a disease characterized by chronic hemolysis and associated long-term complications affecting multiple organ systems, regardless of the degree of anemia or transfusion status. New disease-modifying treatment approaches are needed, as current management strategies are supportive and include regular blood transfusions, which can lead to iron overload and splenectomy, which is also associated with short- and long-term risks,” said Andreas Glenthøj, M.D., Ph.D., associate professor, Department of Hematology, Rigshospitalet; Copenhagen, Denmark. ACTIVATE Results Summary The Phase 3 ACTIVATE trial of mitapivat achieved its primary endpoint. Mitapivat demonstrated a sustained, statistically significant increase in hemoglobin in patients with PK deficiency who are not regularly transfused. 40 percent (n=16) of patients randomized to mitapivat achieved a hemoglobin response, compared to 0 patients randomized to placebo (2-si...