CAMBRIDGE, Mass., June 13, 2026
Agios Pharmaceuticals, Inc. announced detailed results from the 52-week double-blind portion of the global RISE UP Phase 3 trial evaluating mitapivat, an oral pyruvate kinase (PK) activator, in patients aged 16 years and older with sickle cell disease (SCD). The findings were presented during the prestigious Plenary Abstracts Session at the European Hematology Association (EHA) 2026 Congress in Stockholm, Sweden. The study demonstrated a statistically significant improvement in hemoglobin response, rapid and durable anti-hemolytic activity, meaningful reductions in transfusion burden, and clinically important benefits across multiple patient-reported outcomes.
Mitapivat Demonstrates Strong Hemoglobin Response and Reduced Transfusion Burden
The RISE UP Phase 3 trial confirmed that 40.6% of patients receiving mitapivat achieved the primary endpoint of hemoglobin response, compared with only 2.9% in the placebo group. Patients treated with mitapivat experienced sustained improvements in hemoglobin levels and reductions in indirect bilirubin, a key marker of red blood cell destruction. New analyses presented at EHA 2026 showed that patients receiving mitapivat had a 41.1% relative reduction in the need for blood transfusions compared with placebo and a 55.9% reduction in average red blood cell units transfused per patient, highlighting the therapy’s potential to reduce dependence on supportive care interventions.
Clinical Benefits Extend Beyond Hemoglobin Improvement
Further analysis revealed that patients who achieved a hemoglobin response while receiving mitapivat experienced meaningful clinical improvements across several disease-related outcomes. Hemoglobin responders showed a 26% reduction in annualized sickle cell pain crises, a 34% reduction in pain crisis-related hospitalizations, and a 53% reduction in emergency room visits associated with pain crises. Additional benefits included improvements in fatigue, physical functioning, pain impact, sleep quality, and overall health-related quality of life. These findings reinforce the relationship between improved red blood cell health and better day-to-day outcomes for patients living with sickle cell disease.
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Favorable Safety Profile Supports Potential New Treatment Option
Mitapivat was generally well tolerated throughout the study, with a safety profile consistent with previous clinical trials. The incidence of treatment-emergent adverse events was similar between the mitapivat and placebo groups, and no treatment-related deaths were reported. Agios noted that the RISE UP study is the first pivotal Phase 3 trial to validate pyruvate kinase activation as a therapeutic approach in sickle cell disease. Building on more than a decade of clinical experience across hemolytic anemias, the company believes mitapivat has the potential to offer a differentiated treatment option for patients with sickle cell disease. Agios previously announced the submission of a supplemental New Drug Application (sNDA) to the U.S. FDA seeking accelerated approval of mitapivat for this indication.
Source: Agios Pharmaceuticals press release
