Agios Pharmaceuticals, Inc. AGIO
Agios Pharmaceuticals, Inc. (Nasdaq: AGIO) is a hematology drug developer. Its lead program, mitapivat (PYRUKYND®/AQVESME®), is in Phase 3 for hemolytic anemias (PKR deficiency, thalassemia, sickle cell disease). Its next catalyst on record is mitapivat (SCD): PDUFA goal date for sNDA in SCD (February 1, 2027).
Upcoming catalysts
- February 1, 2027mitapivat (SCD) — PDUFA goal date for sNDA in SCD
The PDUFA goal date was subsequently extended to February 1, 2027 following our response to an FDA information request to inform its evaluation as to whether a REMS is necessary for mitapivat in SCD.
Next further out: AG-236 — Initiation of phase 2 portion of phase 2/3 trial in polycythemia vera (H2 2026). 3 catalysts on record beyond the next 30 days; subscribers see each with its filing sentence.
Pipeline
| Program | Indication | Modality | Stage |
|---|---|---|---|
| mitapivat (PYRUKYND®/AQVESME®) | PK deficiency, thalassemia | small molecule | Approved |
| mitapivat in sickle cell disease | sickle cell disease | small molecule | Phase 3 |
| AG-236 | polycythemia vera | RNA (siRNA) | Phase 2 |
| AG-181 | phenylketonuria | small molecule | Phase 1 |
| cevidoplenib | immune thrombocytopenia | small molecule | Pre-clinical |
Recent results and decisions
- Nov 5, 2026positive New data from RISE UP Phase 3 trial shows mitapivat produced durable hemoglobin improvements for up to 115 weeks in sickle cell disease.
New clinical data from the RISE UP Phase 3 trial of mitapivat in sickle cell disease shows durable hemoglobin improvements for up to 115 weeks (over two years) with continued treatment.
Filing - Nov 5, 2026positive First clinical data from Phase 1 trial of AG-236 in healthy volunteers support every-six-month dosing in polycythemia vera.
The first clinical data for AG-236 from a Phase 1 trial in healthy volunteers support every-six-month dosing in polycythemia vera, supporting a potential best-in-class profile.
Filing - Jul 30, 2026accepted FDA accepted the mitapivat sNDA in sickle cell disease (accelerated approval pathway) with Priority Review; PDUFA goal date Nov 1, 2026.
The U.S. Food and Drug Administration (FDA) accepted Agios’ supplemental New Drug Application (sNDA) for mitapivat in sickle cell disease with a Priority Review.
Filing
5 more on record.
For subscribers
- Cash runway against each catalyst, with the company's own runway statement
- Track record: every Phase 3 readout, FDA decision and guidance slip since 2014
- Expected move for each dated catalyst, from comparable past events
- Phase 2 evidence behind each Phase 3 readout
- Companies on the same target or class
- Full catalyst list with filing sentences, deals, registered trials, Excel export
Other phase 3 hematology companies
The free Monday brief
Last week's readouts and FDA decisions and the dated catalysts in the week ahead, each with its filing sentence.