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Also known as [14C]-tebapivat, AG-946
Tebapivat · 3 trials · 4 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.
| 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. |
| Part 1: Single Ascending Dose (SAD) Phase | EXPERIMENTAL | Participants will receive a range of doses of AG-946 or placebo, orally, once on Day 1. AG-946 will be given under fasted or fed conditions. |
| Part 2: Multiple Ascending Dose (MAD) Phase | EXPERIMENTAL | Participants will receive a range of doses of AG-946 or placebo, orally, once daily (QD) for 14 days or using an alternative dosing regimen for up to 28 days under fasted conditions. |
| Part 3: Sickle Cell Disease (SCD) Phase | EXPERIMENTAL | Participants will receive a range of selected ascending doses of AG-946, orally, QD or using an alternative dosing regimen for 28 days. |
| Name | Type | Description |
|---|---|---|
| Tebapivat | DRUG | Oral tablets. |
| Tebapivat Matched Placebo | DRUG | Oral tablets. |
| AG-946 | DRUG | AG-946, oral encapsulated coated-granules or tablets. |
| AG-946 Matched Placebo | DRUG | AG-946 matched-placebo, oral encapsulated sugar spheres or tablets. |
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 sickle cell disease and for anemia due to lower-risk myelodysplastic syndromes. It is in Phase 2 clinical development and has not been approved by the FDA. It holds an orphan drug designation.
Tebapivat targets PKR, also known as PKM2. It is a small molecule designed to act on this pyruvate kinase target. Agios Pharmaceuticals is developing it as an oral therapy for hematologic conditions marked by red blood cell dysfunction.
Tebapivat is being developed by Agios Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker AGIO. Agios is running the clinical program for the drug in sickle cell disease and lower-risk myelodysplastic syndromes.
Tebapivat is in Phase 2 clinical development. It is investigational and has not received FDA approval. The program includes two Phase 2 studies, one in sickle cell disease and one in anemia due to lower-risk myelodysplastic syndromes, both currently active and not recruiting.
Tebapivat is being studied in NCT06924970, a Phase 2 dose-finding trial in sickle cell disease with 59 participants across the United States, Belgium, Canada, France, Ireland, Netherlands, United Kingdom, and in NCT05490446, a Phase 2 trial in anemia due to lower-risk myelodysplastic syndromes with 87 participants across multiple countries.
Yes, [14C]-tebapivat is an alternative name for tebapivat. It refers to the carbon-14 labeled form of the compound used in research settings. Both names refer to the same Agios Pharmaceuticals drug candidate being studied in Phase 2 trials.