Agios Pharmaceuticals has started the Activate trial, a Phase lll study analysing the efficacy and safety of AG-348 for the treatment of patients with pyruvate kinase (PK) deficiency who do not receive regular blood transfusions.

The randomised, placebo-controlled trial intends to enrol about 80 adult subjects with haemoglobin less than or equal to 10g/dL and at least one non-missense mutation.

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Under the trial, the subjects will be randomised at 1:1 ratio at nearly 35 sites in 15 countries worldwide.

Part one of the trial will include a dose optimisation period where patients will receive 5mg of AG-348 or placebo twice daily, with the flexibility to titrate up to 20mg or 50mg twice daily over a three month period to establish their individual optimal dose, as measured by the maximum increase in haemoglobin levels.

Following the completion of the dose optimisation period, patients will receive their optimal dose for an additional three months in part two of the trial.

“We are executing a comprehensive pivotal development plan for adults with this lifelong anaemia who currently only have access to supportive care.”

The trial’s primary objective is the proportion of patients who achieve at least a 1.5g/dL increase in haemoglobin sustained over multiple visits in the second part of the trial.

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Agios Pharmaceuticals chief medical officer Chris Bowden said: “AG-348 is the first potential treatment for PK deficiency that targets the underlying cause of a patient’s anaemia and has demonstrated robust and sustained haemoglobin increases in adults with this disease who are not regularly transfused.

“With the initiation of Activate and the ongoing Activate-T study in patients who are regularly transfused, we are executing a comprehensive pivotal development plan for adults with this lifelong anaemia who currently only have access to supportive care.”

PK deficiency is a rare hereditary disease that presents as hemolytic anaemia, the accelerated destruction of red blood cells.