Pyruvate Kinase Deficiency Impact Assessment (PKDIA), a weekly measure of disease impacts, measures health-related quality of life, (e.g., daily activities, concentration, physical activity and the ...
– 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 ...
– 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 ...
– 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 ...
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 ...
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 ...
– Results from Phase 3 ENERGIZE-T Study of Mitapivat in Transfusion-dependent Thalassemia will be Presented in Oral Session – – Tebapivat Phase 1 Data in Sickle Cell Disease and Phase 2b ...
CAMBRIDGE, Mass., Feb. 17, 2022 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism pioneering therapies for genetically defined diseases, ...
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