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 anemia due to lower-risk myelodysplastic syndromes (LR-MDS) and for sickle cell disease (SCD). 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 Nasdaq under the ticker AGIO. The company is conducting clinical trials of Tebapivat in myelodysplastic syndromes, sickle cell disease, and healthy participants.
Tebapivat is in Phase 2 clinical development for lower-risk myelodysplastic syndromes and sickle cell disease. It has also completed a Phase 1 study in healthy participants. Tebapivat is investigational and not yet approved by the FDA.
Tebapivat is being studied in three clinical trials. NCT05490446 is a Phase 2 study in 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 sickle cell disease.
Yes, Tebapivat has received orphan drug designation from the FDA. Orphan drug designation is granted to drugs intended to treat rare diseases, which can provide certain development and commercial incentives to the sponsor.
Yes, Tebapivat is being studied in sickle cell disease. NCT06924970 is an active Phase 2 dose-finding study assessing the efficacy and safety of Tebapivat in participants with sickle cell disease, with enrollment of 59 participants across multiple countries.