– Prespecified Statistical Criterion for the Primary Endpoint in ACTIVATE-KidsT Was Not Met; Results Were Clinically Meaningful, with Observed Response Rates Higher for Mitapivat than Placebo for the ...
Agios Pharmaceuticals' Rare Blood Disorder Drug Misses Primary Goal In Pediatric Study, Stock Slides
Mitapivat was generally safe and well-tolerated, with safety results consistent with the safety profile for mitapivat. Topline data from ACTIVATE-Kids study of mitapivat in children who are not ...
Findings showed 28.1% of patients treated with mitapivat achieved a TRR vs 11.8% of patients who received placebo. A phase 3 trial evaluating mitapivat in pediatric patients with pyruvate kinase ...
Rocket's clinical programs using lentiviral vector (LV) based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and ...
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, ...
(RTTNews) - Agios Pharmaceuticals, Inc. (AGIO) announced Friday that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion on ...
Agios Pharmaceuticals Inc.’s fourth-quarter and full-year 2023 financial report in mid-February highlighted further prospects with Pyrukynd (mitapivat), the pyruvate kinase activator for hemolytic ...
There are currently no FDA-approved oral treatments for non-transfusion-dependent thalassemia. Mitapivat was found to be an effective oral treatment for non-transfusion-dependent (NTD) alpha- or ...
– 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 ...
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