AGIO Q2: Mitapivat SCD sNDA Priority Review, Nov 1

Agios Pharmaceuticals (AGIO) reported second-quarter 2026 earnings on July 30, confirming that the FDA accepted its supplemental NDA for mitapivat in sickle cell disease with Priority Review. The PDUFA goal date is November 1, 2026, filed under the accelerated approval pathway.

Mitapivat generated $44.7 million in Q2 net revenue, a 3.6x increase year over year, driven by the U.S. launch of AQVESME in thalassemia. The stock closed at $35.01 on July 29 and traded down roughly 10% intraday on July 30 as investors weighed the quarterly net loss and the long timeline to new pipeline revenue.

Price update August 2, 2026 (Polygon): AGIO closed at $30.02 on July 31, down 14.3% from the $35.01 reference price. The post-earnings sell-off reflects investor concerns about the Nov 1 PDUFA for the SCD sNDA, where the co-primary pain-crisis endpoint was missed, and the long runway to pipeline revenue from cevidoplenib and AG-236.

The SCD sNDA: what the data showed

The sNDA relies on the RISE UP Phase 2 and Phase 3 trials. Hemoglobin improvement was statistically significant, but the co-primary endpoint of reducing sickle cell pain crises was not met. Agios is seeking accelerated approval on the surrogate endpoint of hemoglobin response, a pathway the FDA accepted after a pre-sNDA meeting in March 2026. For a primer on this regulatory route, see our guide to accelerated approval.

The confirmatory trial, REIGNITE, is already enrolling. It randomizes approximately 159 patients 2:1 to mitapivat 100 mg twice daily or placebo, with transfusion-free status from Week 4 through Week 52 as the primary endpoint.

Sickle cell disease affects roughly 75,000 people in the U.S. If approved, mitapivat would be the first oral pyruvate kinase activator in SCD. The market has been volatile: GBT/PTC’s Oxbryta was pulled in September 2024 over safety concerns, leaving a treatment gap.

Pipeline expansion: two new assets

Agios licensed cevidoplenib, a next-generation oral SYK inhibitor, from Oscotec for immune thrombocytopenia (ITP). SYK inhibitors block the autoantibody-mediated platelet destruction that drives ITP. A Phase 3 trial is planned for the first half of 2028.

The company also advanced AG-236, an siRNA targeting TMPRSS6, into Phase 2/3 development for polycythemia vera. Phase 1 healthy-volunteer data showed sustained hepcidin control and iron pathway modulation, with potential for dosing as infrequently as every six months. The Phase 2 portion begins in the second half of 2026. AG-236 could compete with Takeda’s rusfertide, a hepcidin mimetic also in development for polycythemia vera.

Risk and what to watch

The near-term binary event is the November 1 PDUFA. The mixed pain crisis data could limit the label or trigger an advisory committee. On the competitive side, Novo Nordisk’s etavopivat met both co-primary endpoints in the Phase 3 HIBISCUS trial with a 27% reduction in vaso-occlusive crises, raising the efficacy bar. For context on Agios’s earlier SCD setback with tebapivat, see our coverage of the tebapivat SCD discontinuation.

Source: Agios Q2 2026 press release