Agios moves closer to filing oral thalassaemia drug after study success

03 Jan 2024
firstwordpharma.com
Clinical ResultPhase 3Drug Approval
Agios Pharmaceuticals said Wednesday that Pyrukynd (mitapivat) met the primary endpoint of haemoglobin response in a Phase III study in adults with non-transfusion-dependent alpha- or beta-thalassaemia, becoming the first oral drug to achieve this goal. The news drove the company’s shares up as much as nearly 20% in premarket trading.
The ENERGIZE trial randomised 194 patients with non-transfusion-dependent thalassaemia to receive Pyrukynd twice daily or matching placebo for 24 weeks. According to top-line results, 42.3% of those given Agios’ pyruvate kinase activator achieved a haemoglobin response - defined as an increase of ≥1 g/dL in average haemoglobin concentrations from week 12 through week 24 compared with baseline - which was significantly more than 1.6% of subjects on placebo.
“All subgroup analyses favoured the [Pyrukynd] treatment arm,” noted Sarah Gheuens, head of R&D at Agios. The drug also demonstrated statistically significant improvements compared to placebo for the key secondary endpoints of change from baseline in the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) subscale score and in average haemoglobin concentration.
Marketing applications by year end
Agios is also awaiting data from the Phase III ENERGIZE-T study of Pyrukynd in adults with transfusion-dependent alpha- or beta-thalassaemia, with a top-line readout expected in mid-2024. The company plans to use data from both trials to seek approval of the drug for thalassaemia by the end of the year.
In terms of safety, Agios noted that the incidence of adverse events was similar across the two study arms in ENERGIZE. However, four patients administered Pyrukynd experienced adverse events (AEs) leading to discontinuation, versus none in the placebo arm.
Pyrukynd is currently approved in the US for the treatment of haemolytic anaemia in adults with pyruvate kinase (PK) deficiencypyruvate kinase (PK) deficiency and for the treatment of PK deficiency in adults in the EU.
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