Agios Pharmaceuticals, Inc. presented detailed data from the 52‑week double‑blind portion of its RISE UP Phase 3 trial of mitapivat in patients aged 16 years or older with sickle cell disease at the 31st European Hematology Association Congress in Stockholm. The global trial randomized 207 participants in a 2:1 ratio to receive oral mitapivat 100 mg twice daily or matched placebo; 176 participants completed the double‑blind period and 174 subsequently entered a 216‑week open‑label extension.
The primary hemoglobin response endpoint was met, with 40.6% of patients in the mitapivat arm achieving a ≥1.0 g/dL increase in average hemoglobin from baseline between Week 24 and Week 52, compared with 2.9% in the placebo arm. Among responders, the mean increase from baseline was 1.6 g/dL.
Transfusion metrics improved markedly: the proportion of patients requiring blood transfusions was reduced by 41.1% relative (23.9% on mitapivat versus 40.6% on placebo), and the average number of red blood cell units transfused per patient fell by 55.9% relative (0.70 units versus 1.59 units).
Patients who achieved the hemoglobin response experienced a 26% reduction in the annualized rate of sickle cell pain crises (2.20 versus 2.98 for non‑responders) and a 34% reduction in related hospitalizations (1.16 versus 1.76 for non‑responders). These responders also reported fatigue score improvements exceeding the predefined 4.1‑point threshold for clinical meaningfulness.
Despite these subgroup benefits, the trial did not meet its primary endpoint of reducing the overall annualized rate of pain crises compared with placebo, and there was no overall difference between mitapivat and placebo for the key secondary endpoint of patient‑reported fatigue.
Safety findings indicated that treatment‑emergent adverse events occurred in 97.1% of mitapivat‑treated patients versus 98.6% of placebo‑treated patients, with no treatment‑related deaths reported.
In May 2026, Agios submitted a supplemental New Drug Application to the U.S. Food and Drug Administration seeking accelerated approval of mitapivat for sickle cell disease. The company, valued at $1.75 billion, reported 78% revenue growth over the trailing twelve months as of Q1 2026. Agios shares were trading at $29.46, reflecting an 8% year‑to‑date gain, with analyst price targets ranging from $28 to $59 and a consensus recommendation leaning positive. The firm holds more cash than debt, though cash burn remains high, typical for biotech firms advancing clinical programs.
Separately, Agios entered a licensing agreement with Oscotec granting exclusive global rights to cevidoplenib, a spleen tyrosine kinase inhibitor for immune thrombocytopenia. Oscotec will receive $25 million upfront and may earn up to $140 million in development and regulatory milestones across up to three indications in the United States and Europe, together with commercial milestone payments and royalties on future net sales.
Agios also announced the termination of development of tebapivat for lower‑risk myelodysplastic syndromes after a Phase 2b trial in 65 patients failed to meet the company’s advancement criteria for transfusion independence over 24 weeks. The European Commission approved mitapivat for the treatment of anemia in adults with thalassemia, and Avanzanite Bioscience will commercialize the drug in Europe under an agreement with Agios.