– 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 ...
– ACTIVATE-Kids is the First Study to Demonstrate Efficacy of an Oral Therapy for Children with PK Deficiency Who Are Not Regularly Transfused – – Safety Results Consistent with Safety Profile for ...
About Pyruvate Kinase Deficiency Pyruvate Kinase Deficiency (PKD) is a rare, monogenic red blood cell disorder resulting from a mutation in the PKLR gene encoding for the pyruvate kinase enzyme, a key ...
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, ...
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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