Tebapivat
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Product Information
Scientific name: Tebapivat
Brand name: Ν/Α
RESPONSIBLE: Agios Pharmaceuticals Inc -
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 countryCompletion date: May 2027
Scope of the Study / Aim: Hb response -
Regulatory Information
Status: Not Authorised
Additional notable points:
- EMA: N/A
- FDA: N/A
- MHRA: N/A
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.
Update: 30 June 2025
Improved RBC function and reduction of sickling tendency were observed in 6 patients.
