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Agios Presents Updated PYRUKYND® (mitapivat) Long-term Extension Data Demonstrating Sustained Clinical Benefits in Adults with Pyruvate Kinase (PK) Deficiency at 64th ASH Annual Meeting and Exposition
– Data Suggest Long-term Treatment with PYRUKYND® in Adults with PK Deficiency is Associated with Improvements in Hemoglobin, Iron Overload, Transfusion
About this update from Agios Pharmaceuticals, Inc.
[{"type":"text","content":"– Data Suggest Long-term Treatment with PYRUKYND® in Adults with PK Deficiency is Associated with Improvements in Hemoglobin, Iron Overload, Transfusion Burden and Patient-reported Outcomes, Regardless of Transfusion Status – – Additional Data Presented at ASH Characterize Disease Complications and Co-morbidities of PK Deficiency in Pediatric Populations, Supporting Ongoing Pivotal ACTIVATE-Kids and ACTIVATE-KidsT Studies of PYRUKYND® – – Agios to Host Live and Webcast Investor Event on Dec. 12, 2022, at 7 a.m. CT – CAMBRIDGE, Mass., Dec. 11, 2022 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today reported new data from the ongoing extension study assessing the long-term efficacy and safety of PYRUKYND® (mitapivat) in adults with pyruvate kinase (PK) deficiency who had participated in one of the pivotal studies, ACTIVATE and ACTIVATE-T, conducted in not regularly transfused and regularly transfused adults with PK deficiency, respectively. Data from the studies were featured in multiple presentations at the 64th American Society of Hematology (ASH) Annual Meeting and Exposition, hosted Dec. 10-13, 2022, in New Orleans. PYRUKYND® is a first-in-class, oral PK activator and the first approved disease-modifying therapy for patients in the U.S. and EU with this rare, debilitating, lifelong hemolytic anemia. Long-term extension data (abstract # 2328) show that previously reported effects of PYRUKYND® on hemoglobin and transfusion burden were maintained over time. As of the March 27, 2022 data cut-off, the median duration of hemoglobin response among the 31 hemoglobin responders from ACTIVATE and the long-term extension study was 18.3 months, with responses ongoing up to 32.9 months. Hemoglobin response rate among patients who switched from placebo in ACTIVATE to PYRUKYND® in the extension study (39.5 percent Hb response rate) was similar to that observed in patients treated with PYRUKYND® in ACTIVATE. All regularly transfused patients who achieved transfusion-free status in ACTIVATE-T with PYRUKYND® treatment maintained transfusion-free status through the extension study for up to 38.3 months. PYRUKYND® was well tolerated, and the safety profile was consistent with that in ACTIVATE and ACTIVATE-T, as well as previous studies. “PYRUKY...