Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Tebapivat · 3 trials · 3 indications
Hb response is defined as a ≥1.5-grams per deciliter (g/dL) increase from baseline in the average Hb concentration from Week 8 through Week 16.
Transfusion Independence is defined as transfusion-free for ≥8 consecutive weeks during the Core Period (participants With Low Transfusion Burden \[LTB\] only).
Transfusion independence, defined as transfusion-free for ≥8 consecutive weeks (TI8) during the Core Period.
Quantification of major metabolites of \[14C\]-tebapivat in plasma.
Quantification of major metabolites of \[14C\]-tebapivat in excreta.
Identification of the chemical structures of major metabolites of \[14C\]-tebapivat in plasma.
Identification of the chemical structures of major metabolites of \[14C\]-tebapivat in excreta.
| Arm | Type | Description |
|---|---|---|
| Tebapivat 2.5 milligrams (mg) | EXPERIMENTAL | Participants will receive 2.5 mg tebapivat orally, once daily (QD) for 12-weeks in the double-blind (DB) period. Participants who complete the DB Period will be eligible to receive the same dose in the Open-Label Extension (OLE) period for up to 52 weeks. |
| Tebapivat 5.0 mg | EXPERIMENTAL | Participants will receive 5.0 mg tebapivat orally, QD, for 12-weeks in the DB period. Participants who complete the DB Period will be eligible to receive the same dose in the OLE period for up to 52 weeks. |
| Tebapivat 7.5 mg | EXPERIMENTAL | Participants will receive 7.5 mg tebapivat orally, QD, for 12-weeks in the DB period. Participants who complete the DB Period will be eligible to receive the same dose in the OLE period for up to 52 weeks. |
| Tebapivat Matched Placebo | PLACEBO_COMPARATOR | Participants will receive a matched placebo, orally, QD, for 12-weeks in the DB period. Participants who complete the DB Period will be randomized in 1:1:1 to receive tebapivat 2.5 mg QD, tebapivat 5.0 mg QD, or tebapivat 7.5 mg QD in the OLE period for up to 52 weeks |
| Core Period: Phase 2a - Tebapivat 5 mg | EXPERIMENTAL | Participants will receive 5 milligrams (mg) tebapivat orally, once daily for up to 16 weeks. At the discretion of the investigator, participants who complete the Core Period will be eligible to receive the same dose in Extension Period for up to 156 weeks. |
| Core Period: Phase 2b - Tebapivat 10 mg | EXPERIMENTAL | Participants will receive 10 mg tebapivat, orally, once daily for up to 24 weeks. At the discretion of the investigator, participants who complete the Core Period will be eligible to receive the same dose in Extension Period for up to 156 weeks. |
| Core Period: Phase 2b - Tebapivat 15 mg | EXPERIMENTAL | Participants will receive 15 mg tebapivat, orally, once daily for up to 24 weeks. At the discretion of the investigator, participants who complete the Core Period will be eligible to receive the same dose in Extension Period for up to 156 weeks. |
| Core Period: Phase 2b - Tebapivat 20 mg | EXPERIMENTAL | Participants will receive 20 mg tebapivat, orally, once daily for up to 24 weeks. At the discretion of the investigator, participants who complete the Core Period will be eligible to receive the same dose in Extension Period for up to 156 weeks. |
| [14C]-tebapivat and [13C2,15N3]-tebapivat | EXPERIMENTAL | Participants will receive a single oral dose of 10 milligrams (mg) \[14C\]-tebapivat containing 200 microcurie \[μCi\] of radiocarbon in a capsule followed by a single intravenous (IV) microdose of 0.1 mg \[13C2,15N3\]-tebapivat on Day 1. |
| Name | Type | Description |
|---|---|---|
| Tebapivat | DRUG | Oral tablets. |
| Tebapivat Matched Placebo | DRUG | Oral tablets. |
| [14C]-tebapivat | DRUG | Oral Capsule |
| [13C2,15N3]-tebapivat | DRUG | Intravenous Solution |
Key Inclusion Criteria: * Documented diagnosis of SCD (HbSS, HbSC \[combined heterozygosity for hemoglobins S and C\], sickle hemoglobin \[HbS\]/β0-thalassemia, HbS/β+-thalassemia, or other sickle cell syndrome variants). * Hemoglobin ≥5.5 and ≤10.5 grams per decilitre (g/dL). Hemoglobin concentrat...
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Tebapivat is an investigational small molecule being studied for the treatment of anemia due to lower-risk myelodysplastic syndromes (LR-MDS) and for sickle cell disease. It is also being evaluated in healthy participants to understand how the drug is absorbed, broken down, and removed from the body.
Tebapivat is being developed by Agios Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol AGIO. The company is conducting clinical trials to evaluate the drug's safety and efficacy in multiple patient populations.
Tebapivat is currently in Phase 2 clinical development for lower-risk myelodysplastic syndromes and sickle cell disease. A Phase 1 study in healthy participants has been completed. The drug is investigational and has not been approved by the FDA.
Tebapivat is being studied in three clinical trials. NCT05490446 is a Phase 2 study in participants with anemia due to lower-risk myelodysplastic syndromes. NCT06745271 is a completed Phase 1 study in healthy participants. NCT06924970 is a Phase 2 dose-finding study in participants with sickle cell disease.
Tebapivat is a small molecule that targets pyruvate kinase, an enzyme involved in cellular energy production. By activating this enzyme, the drug aims to improve red blood cell function and reduce anemia in conditions like myelodysplastic syndromes and sickle cell disease.
Yes, Tebapivat has received orphan drug designation from the FDA. This designation is granted to drugs intended to treat rare diseases and provides certain development incentives, including market exclusivity upon approval.