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Agios Presents New Clinical Data Supporting the Benefits of PYRUKYND® (mitapivat) Treatment in Adults with PK Deficiency at European Hematology Association Annual Congress

– Treatment with PYRUKYND® Associated with Early and Robust Hemoglobin Responses in Phase 3 ACTIVATE and Extension Studies; Approximately One-Third of

articleAgios Pharmaceuticals, Inc.June 10, 20225/company/agios-pharm/news/agios-presents-new-clinical-data-supporting-the-benefits-of-pyrukyndr-mitapivat
Agios Presents New Clinical Data Supporting the Benefits of PYRUKYND® (mitapivat) Treatment in Adults with PK Deficiency at European Hematology Association Annual Congress

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[{"type":"text","content":"– Treatment with PYRUKYND® Associated with Early and Robust Hemoglobin Responses in Phase 3 ACTIVATE and Extension Studies; Approximately One-Third of Patients Achieved Normal Hemoglobin Levels at Least Once – – PYRUKYND®-Treated Patients Reported Significant Improvements in PK Deficiency Signs, Symptoms and Impacts, with Greater Improvements Evident in Hemoglobin Responders – – New Analysis from Peak Registry Demonstrates Wide Range of Comorbidities and Complications in PK Deficiency Patients Regardless of Genotype – – Agios to Host Investor Event on Monday, June 13 at 8 a.m. ET – CAMBRIDGE, Mass., June 10, 2022 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism pioneering therapies for genetically defined diseases, today reported new data further underscoring the significant burden of disease in adults with pyruvate kinase (PK) deficiency regardless of genotype and supporting the potential benefits of treatment with PYRUKYND® for these patients. Data are being presented at the European Hematology Association (EHA) Annual Congress, hosted virtually and in person in Vienna on June 9-12, 2022. “Following the FDA approval of PYRUKYND® for the treatment of hemolytic anemia in adults with PK deficiency in February of this year, the data presented at EHA add to the growing body of evidence underscoring the potential of PYRUKYND® to provide real-world benefits for patients with this rare blood disorder,” said Sarah Gheuens, M.D., Ph.D., chief medical officer at Agios. “Patients treated with PYRUKYND® had early and robust hemoglobin responses and improvements in hallmark signs and symptoms of PK deficiency, including jaundice, tiredness and shortness of breath. In addition, new data from the Peak Registry highlight the importance of appropriate disease management for all patients with PK deficiency, regardless of genotype. Agios continues to focus on maximizing the impact of PYRUKYND® as the first approved disease-modifying treatment for this community.” PYRUKYND® was approved in February 2022 by the U.S. Food and Drug Administration (FDA) for the treatment of hemolytic anemia in adults with PK deficiency. PYRUKYND® is also under review by the European Medicines Agency (EMA) as a potential treatment for adults with PK deficiency, and Agios expects a regulatory decision in the EU...

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