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Mitapivat (AG-348)


Update: 25 November 2019

No update available.


Update: 27 September 2019

DRIVE PK is an ongoing global, open-label, Phase 2, safety and efficacy study evaluating Mitapivat in adults with PK deficiency who do not receive regular transfusions. It is the first clinical trial in adults with PK deficiency.

Mitapivat is an investigational, first-in-class, oral, small molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase-R (PKR) enzymes that directly targets the underlying metabolic defect in PK deficiency, a rare, potentially debilitating, hemolytic anemia.

Methodology: Patients were randomly assigned to receive either 50 mg or 300 mg of Μitapivat twice daily for a 24-week core period and eligible patients could continue treatment in an ongoing extension phase.



  • Data from the study demonstrated rapid, clinically significant increases in hemoglobin in 50 percent of patients, and for patients in the extension phase, the response was sustained for up to 35 months
  • 26 of 52 patients (50%) achieved a clinically significant maximum hemoglobin increase of >1.0 g/dL


Based on these results the investigator industry has moved forward to conduct a proof-of-concept clinical trial of Μitapivat for the treatment of NTDT.

 DRIVE PK consists of two ongoing global trials in adults with PK deficiency that are on track to complete enrollment by year-end 2019:

  • ACTIVATE: A placebo-controlled trial with a 1:1 randomization, expected to enroll approximately 80 patients who do not receive regular transfusions, with an aim to achieve a sustained hemoglobin increase of ≥1.5 g/dL.
  • ACTIVATE-T: A single arm trial of up to 40 regularly transfused patients with an aim to reduce the transfusion burden over six months compared to individual historical transfusion burden over prior 12 months.