AG-236

July 30, 2026: Agios Pharmaceuticals, Inc. (NASDAQ: AGIO) reported Q2 2026 results and provided pipeline/regulatory updates. Mitapivat (PYRUKYND/AQVESME) generated $44.7M worldwide net revenue ($40.9M U.S.; $3.8M ex-U.S.). The U.S. launch of AQVESME for thalassemia (launched late Jan 2026) reached 442 cumulative prescriptions as of June 30, 2026. Ex-U.S. revenue reflected Europe demand following May 2026 approval for thalassemia and early demand in GCC countries.

Regulatory: The European Commission granted marketing authorization for PYRUKYND in adults for anemia associated with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia (orphan designation). For sickle cell disease, FDA accepted Agios’ sNDA for mitapivat with Priority Review under accelerated approval; PDUFA goal date is November 1, 2026. Agios also filed for sickle cell disease approval in Saudi Arabia and dosed the first patient in the REIGNITE Phase 3 confirmatory trial (transfusion-burden endpoint; ages ≥12).

Pipeline/business development: Agios licensed global rights to cevidoplenib (oral SYK inhibitor) from Oscotec for immune thrombocytopenia, targeting Phase 3 start in 1H 2028 after additional CMC work. AG-236 (TMPRSS6 siRNA) Phase 1 data in healthy volunteers supported advancement into Phase 2/3 in polycythemia vera (Phase 2 expected 2H 2026). AG-181 (PAH stabilizer) dosed first patient in Phase 1b in PKU; data expected 2H 2026. Agios will not advance tebapivat in lower-risk MDS or sickle cell disease after Phase 2b/Phase 2 results did not meet predefined advancement criteria.