Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Tebapivat
AG-946 · 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...
AG-946 is an investigational small molecule developed by Agios Pharmaceuticals for hematology indications, specifically anemia and sickle cell disease. It has been studied in a Phase 1 clinical trial in healthy volunteers and participants with sickle cell disease. AG-946 is not approved and remains in clinical development.
AG-946 is being developed for anemia and sickle cell disease. A Phase 1 study evaluated its safety, tolerability, pharmacokinetics, and pharmacodynamics in healthy volunteers and participants with sickle cell disease. The drug is investigational, and no approved use in these indications has been established.
AG-946 is developed by Agios Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker AGIO. Agios is the sponsor of the clinical program studying AG-946 in anemia and sickle cell disease.
AG-946 is in Phase 1 clinical development. Its single reported trial, NCT04536792, was a Phase 1 study that has been completed. The drug is investigational and has not been approved for any indication.
AG-946 has been studied in one clinical trial, NCT04536792, titled a study to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of AG-946 in healthy volunteers and in participants with sickle cell disease. The Phase 1 trial enrolled 122 participants across the United States, Denmark, and Spain and is completed.
Yes. The Phase 1 trial of AG-946, NCT04536792, was randomized, double blind, and controlled. It enrolled 122 participants aged 18 years and older, including healthy volunteers and participants with sickle cell disease, and was conducted in the United States, Denmark, and Spain.