Clinical Trial Updates (SCD)


Update: 30 June 2021

Agios is initiating its pivotal Phase 2/3 study with mitapivat in sickle cell disease by year-end 2021.



Update: 08 January 2021

  • FDA Orphan Drug Designation Granted to Mitapivat for Treatment of Sickle Cell Disease
  • Data on safety and tolerability were presented at ASH2020:
    • 6/11 evaluable patients achieved a hemoglobin increase of ≥1.0 g/dL from baseline. The mean hemoglobin increase among all efficacy evaluable patients was 1.3 g/dL,
    • All 11 evaluable patients have received three ascending dose levels of mitapivat (5 mg BID, 20 mg BID, 50 mg BID) for two weeks’ duration, respectively, and of these, three patients have received an additional ascending dose of 100 mg BID for two All underwent a 12- or 15-day drug taper after completing the dosing.
    • Trial may enrol up to 25 patients.
  • A Phase 3, global, pivotal study of mitapivat in sickle cell disease is expected to initiate in 2021.

Sources: orphan-drug-designation-granted-mitapivat-0 updated-data-phase-1-study-mitapivat-first-class

Update: 05 November 2020

Safety and tolerability data will be presented at the 62nd ASH Meeting in December 2020.



Update: 25 August 2020

The ongoing Phase 1 study, which can enroll up to 25 patients, is evaluating the efficacy, safety, pharmacokinetics and pharmacodynamics of treatment with Mitapivat in adults with sickle cell disease.

6 patients have received three ascending dose levels of Mitapivat. 8 patients have completed all planned dose levels. 1 patient discontinued within the first week due to a pre-existing condition. The primary endpoint of the study is safety and tolerability as assessed by frequency and severity of adverse events and laboratory parameters. Secondary endpoints included changes in hemoglobin, markers of hemolysis, 2,3-DPG and ATP levels and HbS polymerization.

Clinical proof-of-concept has been established based on a preliminary analysis in the Phase 1 trial of Mitapivat (AG-348) in patients with sickle cell disease. The study is being conducted in collaboration with the National Institutes of Health (NIH) as part of a cooperative research and development agreement.

7/8 (88%) patients who completed all planned dose levels of Mitapivat experienced a Hb increase, with 5/8 patients (63%) achieving a hemoglobin increase of ≥1.0 g/dL and decreases in both sickling and HbS polymerization, further supporting the proposed mechanism of action.



Update: 31 May 2020

  • A phase II proof-of-concept trial of mitapivat in sickle cell disease is being run under a Cooperative Research and Development Agreement (CRADA) with the U.S. National Institutes of Health(NIH).
  • New enrollment is paused as a result of the COVID-19 pandemic but remains on track for mid-2020.
  • Data are expected to be submitted by NIH for presentation at the American Society of Hematology (ASH) Annual Meeting in December.


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