Clinical Trial Updates (SCD)


Update: 30 September 2022

No update available.


Update: 30 June 2022

  • Initiated the sickle cell disease cohort of the Phase 1 study of AG-946.
  • Aiming to complete enrollment in the Phase 2 portion of the RISE UP study of PYRUKYND®in sickle cell disease by year-end.

Data from the  Phase 2 open-label study of mitapivat in SCD (ESTIMATE study; 9 patients) were presented at EHA2022 demonstrating the following:

  • Median treatment duration of 38 weeks (range 11-60 weeks).
  • 6/8 (75%) took 100 mg mitapivat twice daily in the fixed dose extension period and 2/8 (25%) took 50 mg mitapivat twice daily after a single dose reduction because of transaminase increase.
  • Hb level significantly increased, accompanied by a decrease in markers of haemolysis (absolute reticulocyte count, total bilirubin, and lactate dehydrogenase
  • Baseline characteristics were: median age 22 years (range 16-59 years), 6/9 (67%) female, and 6/9 (67%) used hydroxyurea. 7/9 (78%) had HbSS, 1/9 (11%) had HbS/β 0 , and 1/9 (11%) had HbS/β +.



Update: 25 April 2022

First patients in the Phase 2 RISE UP study of mitapivat in SCD have been dosed.



Update: 10 January 2022

Data from the Phase 2 open-label study of mitapivat in SCD (ESTIMATE study; 6 patients) were presented at ASH2021 demonstrating the following:

  • No serious adverse events (AEs) occurred, and all AEs were mild and mostly transient. One vaso-occlusive crisis (VOC) occurred without hospital admission and did not require dose reduction or discontinuation.
  • All six patients had improvements in point of sickling.
  • Observed changes in 2,3-DPG and ATP levels were consistent with proposed mechanism of action.
  • Mitapivat increased hemoglobin and decreased hemolysis and sickling parameters.
  • Five out of six patients (83.3%) achieved hemoglobin response (≥ 1g/dL increase from baseline).
  • Early improvements in albumin-to-creatinine ratio were observed.
  • Follow-up data from the ongoing study will be reported at a later stage.

Data from the Phase 1 study of mitapivat in SCD (in collaboration with NIH; 16 patients) were presented at ASH2021 demonstrating the following:

  • No AEs led to drug discontinuation.
  • No VOCs occurred during dose-escalation. Two VOCs occurred during drug taper, which were deemed possibly drug-related, and two VOCs occurred during the 28-day safety follow-up.
  • Treatment with mitapivat demonstrated improvement in anemia and decreases in markers of hemolysis, including lactate dehydrogenase, total bilirubin, absolute reticulocyte count, and aspartate aminotransferase.
  • Nine out of 16 patients (56.3%) achieved a hemoglobin response (≥1g/dL increase from baseline).
  • Mitapivat reduced 2,3-DPG and increased ATP, with expected increase in oxygen affinity and decreased sickling rate.
  • Long-term disease-modifying effects are being evaluated in an ongoing extension study.



Update: 27 October 2021

No update available


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

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