Clinical Advisor

Aqvesme Approved for Anemia in Alpha- or Beta-Thalassemia

Approval was based on the phase 3 ENERGIZE and ENERGIZE-T studies evaluating the safety and efficacy of mitapivat in adults with alpha- or beta-thalassemia.
Seeking Alpha

Agios Pharmaceuticals: Potential Acquisition Target, Upcoming FDA Approvals, Promising Pipeline

Agios Pharmaceuticals develops therapies for rare diseases with a focus on Pyruvate Kinase activators like Pyrukynd for PK deficiency. AGIO is advancing its pipeline with research on Mitapivat for ...
Nasdaq

Agios Pharmaceuticals Receives Positive Orphan Designation for Mitapivat in Sickle Cell Disease from European Commission

Agios Pharmaceuticals has announced that the European Commission has granted orphan medicinal product designation to mitapivat, an oral small molecule pyruvate kinase (PK) activator, for the treatment ...
FOX8 Cleveland

Agios’ Phase 3 ACTIVATE-Kids Study of Mitapivat in Children with Pyruvate Kinase (PK) Deficiency Not Regularly Transfused Met Primary Endpoint

– 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 ...
BioWorld

N-Methylguanidine derivatives to inhibit pyruvate kinase M2 in lung cancer

Non-small-cell lung cancer (NSCLC) accounts for up to 85% of all cases of lung cancer, which is the most frequent cause of cancer-related deaths worldwide. The need for effective treatments against ...