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Agios Pharmaceuticals announces initiation of Phase 3 ACTIVATE trial of AG-348.

Agios Pharmaceuticals announced the initiation of the Phase 3 ACTIVATE trial evaluating AG-348, a first-in-class, selective, small molecule activator of the pyruvate kinase-R enzyme. ACTIVATE is a randomized, placebo-controlled trial that will enroll approximately 80 adults with pyruvate kinase deficiency who do not receive regular blood transfusions. The trial is designed to demonstrate that treatment with AG-348 results in a sustained increase in hemoglobin in these patients. The purpose of this Phase 3, 1:1 randomized, double-blind, placebo-controlled global trial is to evaluate the efficacy and safety of treatment with AG-348 in approximately 80 adults with PK deficiency across approximately 35 sites in 15 countries. Key eligibility criteria include a hemoglobin less than or equal to 10 g/dL and at least one non-missense mutation. Patients homozygous for the R479H mutation will not be eligible for the trial. The study design has two parts. Part 1 is a dose optimization period where patients start at 5 mg of AG-348 or placebo twice daily, with the flexibility to titrate up to 20 mg or 50 mg twice daily over a three month period to establish their individual optimal dose, as measured by maximum increase in hemoglobin levels. After the dose optimization period, patients will receive their optimal dose for an additional three months in Part 2. The primary endpoint of the study is the proportion of patients who achieve at least a 1.5 g/dL increase in hemoglobin sustained over multiple visits in Part 2 of the trial.

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Publication:The Fly
Date:Jun 18, 2018
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