Clinical Trial Updates (SCD)


  • Product Information

    Product Information

    Scientific name: Mitapivat
    Brand name: PYRUKYND®
    RESPONSIBLE: Agios Pharmaceuticals Inc.

  • Clinical Trial/Study Information

    Clinical Trial/Study Information

    Last update: 22/6/2023

    Extension of Phase 1 Pilot Study

    Trial Name: Extension of Phase 1 Pilot Study in SCD*
    Code: NCT04610866
    Phase: 2/3
    Eligible patient diagnosis: SCD (adults) (age 18 – 70)
    No. of Patients enrolled: 15 (Last update: 3/5/2023)
    Study Sites: 1 Sites per country

    Αnticipated completion date: February 2028
    Scope of the Study / Aim: Long-term effects of SCD in the frequency of VOCs
    *in collaboration with National Heart, Lung & Blood Institute (NHLBI)


    Trial Name: RISE UP
    Code: NCT05031780
    Phase: 2/3
    Eligible patient diagnosis: SCD (adults) (age 18 and above)
    No. of Patients enrolled: 267 [anticipated] [79 in phase 2 ; 198 – anticipated – in phase 3] (Last update: 23/6/2023)
    Study Sites: 57 Sites per country

    Anticipated completion date: November 2029
    Scope of the Study / Aim: Determine the recommended dose of mitapivat, evaluate efficacy & safety; decrease annual VOCs

  • Regulatory Information

    Regulatory Information

    Status: Not Authorised


    Additional notable points:

    • EMA: N/A
    • FDA: Orphan drug designation (2020)
    • MHRA: N/A

Update: 30 June 2023

Results from the RISE UP (phase 2) trial showed:

• Treatment with mitapivat demonstrated a statistically significant increase in hemoglobin response rate compared to placebo. Hemoglobin response was defined as an increase of ≥1 g/dL in average hemoglobin concentrations from Week 10 through Week 12 compared with baseline.
• 46.2% of patients (n=12) in the 50 mg twice daily (BID) mitapivat arm and 50.0% of patients (n=13) in the 100 mg BID mitapivat arm achieved a hemoglobin response.
• There were no adverse events (AEs) leading to discontinuation.

These results support proceeding with the Phase 3 part of the study. This is expected to enrol 198 patients, beginning in Oct-Dec 2023, reporting data in 2025 and receiving FDA approval in 2026.



Update: 31 March 2023

• Completed enrollment in the Phase 2 portion of the RISE UP study of PYRUKYND® in adults with sickle cell disease.
• Anticipate to announce data and decision to move forward with Phase 3 by mid-year.


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