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Clinical Trial Updates (SCD)

Tebapivat

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  • Product Information

    Product Information

    Scientific name: Tebapivat
    Brand name: Ν/Α
    RESPONSIBLE: Agios Pharmaceuticals Inc

  • Clinical Trial/Study Information

    Clinical Trial/Study Information

    Trial Name: A Dose-Finding Study of Tebapivat to Assess Efficacy, and Safety in Participants With Sickle Cell Disease (SCD)
    Code: NCT06924970
    Phase: 2
    Eligible patient diagnosis: SCD (over 16 years)
    No. of Patients enrolled: 56 [anticipated] (Last update: 23/07/2025)
    Study Sites: 2 Sites per country

    Completion date: May 2027
    Scope of the Study / Aim: Hb response

  • Regulatory Information

    Regulatory Information

    Status: Not Authorised

     

    Additional notable points:

    • EMA: N/A
    • FDA: N/A
    • MHRA: N/A

Update: 18 October 2026

• Company has announced discontinuation following results from the Phase 2 trial. The data further reinforced PK activation as a clinically validated mechanism in sickle cell disease; however, they did not demonstrate a sufficiently differentiated profile relative to other PK activators to justify continued development of tebapivat.

• Topline results from the Phase 2 trial in patients 16 and older with SCD showed:
– Improvements in haemoglobin levels and hemolysis across all dose levels.
– The trial’s primary endpoint of hemoglobin response (≥1.0 g/dL increase in average hemoglobin concentration from Weeks 10 through 12 compared with baseline) was achieved by 43.8% (n=7/16), 47.1% (n=8/17), and 29.4% (n=5/17) in the 2.5 mg QD, 5.0 mg QD, and 7.5 mg QD tebapivat arms, respectively.
– The safety and tolerability of tebapivat in the Phase 2 trial were consistent with that observed in prior sickle cell disease trials.

Sources: https://investor.agios.com/news-releases/news-release-details/agios-reports-secondquarter-2026-financial-results-and-provides
https://investor.agios.com/news-releases/news-release-details/agios-providesupdate-phase-2-trial-tebapivat-sickle-cell

 

Update: 30 June 2026

No update available.

 

Update: 31 March 2026

  • Enrolment of phase 2 trial completed. The results are expected in the second half of 2026.
  • The double-blind, randomized, placebo-controlled trial is evaluating 3 tebapivat doses (2.5 mg, 5 mg, and 7.5 mg) versus a matched placebo over 12 weeks. The primary endpoint is hemoglobin response, defined as a ≥1.0 g/dL increase in average hemoglobin concentration from Week 10 through Week 12 compared with baseline.

Sources: https://investor.agios.com/news-releases/news-release-details/agios-reports-fourth-quarter-and-full-year-2025-financial
https://investor.agios.com/news-releases/news-release-details/agios-outlines-2026-strategic-priorities-and-key-milestones

 

Update: 22 December 2025

No update available.

 

Update: 30 September 2025

  • 1st patient dosed in clinical trial
  • The trial is enrolling across three dose cohorts (2.5mg, 5mg, 7.5mg) and placebo.
  • The primary endpoint will measure hemoglobin response, defined as a ≥1g/dL increase in hemoglobin concentration from week 10 to week 12, compared to baseline.

Source: Agios Reports Second Quarter 2025 Financial Results and Provides Business Update – Agios Pharmaceuticals, Inc.

 

Update: 30 June 2025

Improved RBC function and reduction of sickling tendency were observed in 6 patients.

Source: EX VIVO ACTIVATION OF PYRUVATE KINASE BY TEBAPIVAT REDUCES SICKLING… – Rab M – EHA-4147 – Jun 14 2025

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