Agios reports Q2 2026 results, sickle cell filing gets FDA priority review
Agios said its mitapivat sNDA in sickle cell disease has a PDUFA date of November 1, 2026, while it licensed cevidoplenib and dropped tebapivat from two programs.
Agios Pharmaceuticals reported second quarter 2026 results and business updates on July 30, 2026. The FDA accepted Agios' supplemental New Drug Application for mitapivat in sickle cell disease with Priority Review, setting a PDUFA goal date of November 1, 2026 under the accelerated approval pathway.1 Agios also dosed the first patient in the REIGNITE Phase 3 trial, the confirmatory study required under that pathway, designed to show mitapivat's effect on reducing transfusion burden in sickle cell disease patients aged 12 and older.1
In Europe, the European Commission granted marketing authorization for PYRUKYND in adults with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia, with orphan medicinal product designation, making it the only medicine approved across all EU member states for that patient population.1
On business development, Agios licensed exclusive global rights to cevidoplenib, an oral SYK inhibitor for immune thrombocytopenia, from Oscotec, a deal the company said could unlock up to $1.0 billion in peak U.S. sales potential.1 Agios said it expects to move cevidoplenib into Phase 3 development for ITP in the first half of 2028, pending additional manufacturing work.1 Separately, Agios plans to advance AG-236 into a Phase 2/3 program in polycythemia vera, with the Phase 2 portion expected to start in the second half of 2026.1
Agios discontinued two tebapivat programs: the company will not advance tebapivat in lower-risk myelodysplastic syndromes after Phase 2b results showed biological activity but insufficient clinical benefit to meet its threshold for advancement1, and it will also not advance tebapivat in sickle cell disease, saying Phase 2 data reinforced PK activation as a mechanism but did not show a sufficiently differentiated profile versus other PK activators.1
On finances, Agios held $964.8 million in cash, cash equivalents and marketable securities as of June 30, 2026, down from $1.2 billion at year-end 2025.1 The company said this position, along with expected product revenue and interest income, is intended to fund the AQVESME thalassemia launch, preparation for a possible sickle cell launch of mitapivat, and continued pipeline work.1
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