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Agios’ Phase III Data Move It Closer To First Therapy For Rare Anemia

 
• By Joseph Haas

Strong top-line efficacy in pyruvate kinase deficiency patients who don’t receive regular transfusions looks approvable, analysts say, but Agios will await data in regularly transfused patients.

Anemia medical diagram concept as normal and abnormal blood cell count and human circulation in an artery or vein as a 3D illustration isolated on a white background.
Agios hopes to bring the first drug therapy for PK deficiency to market

With two approved leukemia drugs that target mutated isocitrate dehydrogenase (IDH), Agios Pharmaceuticals, Inc. is moving closer to adding a major revenue driver with its first-in-class allosteric activator of mutated pyruvate kinase receptor enzymes. Top-line Phase III data for mitapivat reported on 1 December support submission for US and EU approval to treat a rare form of anemia, and the drug may be applicable in larger anemia indications.

Data from the 80-patient, pivotal Phase III ACTIVATE study of mitapivat in pyruvate kinase deficiency patients who are not regularly...

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