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 PK deficiency hemolytic anemia; alpha/beta-thalassemia anemia (sickle cell disease sNDA under review). Its next catalyst on record is AG-181: confirm proof of mechanism in PKU patients (H2 2026).
Upcoming catalysts
No catalyst is dated to the next 30 days or to an FDA action date.
Next further out: AG-181 — confirm proof of mechanism in PKU patients (H2 2026). 2 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 | sickle cell disease (sNDA, accelerated approval; Phase 3 REIGNITE confirmatory enrolling) | small molecule | Phase 3 |
| AG-236 | polycythemia vera | RNA (siRNA/ASO/mRNA) | Phase 1 |
| AG-181 | phenylketonuria | small molecule | Phase 1 |
| cevidoplenib | immune thrombocytopenia | small molecule | Pre-clinical |
Recent results and decisions
- 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 - Jul 30, 2026negative Phase 2b tebapivat LR-MDS trial showed biological activity but did not meet the predefined clinical benefit threshold; Agios will not advance it.
While tebapivat demonstrated evidence of biological activity, it did not demonstrate clinical benefit in a sufficient proportion of patients or any patient subgroup to meet the company's predefined threshold for advancement in LR-MDS.
Filing - Jul 30, 2026positive Phase 1 healthy-volunteer trial of AG-236 showed sustained hepcidin control and iron biomarker effects without titration, supporting up to every-six-month dosing.
Results from Agios’ Phase 1 trial of AG-236 in healthy volunteers demonstrated sustained hepcidin control and effects on iron regulation biomarkers without the need for titration, supporting iron pathway modulation that can potentially address excess red blood cell production in polycythemia vera.
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
- Incyte Corp INCY
- Grifols SA GRFS
- Disc Medicine, Inc. IRON
- Geron Corporation GERN
- Nuvectis Pharma, Inc. NVCT
- X4 Pharmaceuticals, Inc XFOR
The free Monday brief
Last week's readouts and FDA decisions and the dated catalysts in the week ahead, each with its filing sentence.