Agios Pharmaceuticals, Inc. (AGIO) on Wednesday reported topline results from the RISE UP Phase 3 trial of mitapivat in patients aged 16 years and older with sickle cell disease. The trial failed to meet one of its two primary endpoints, sending the company's shares down more than 40% in pre-market trading.
Sickle cell disease is a rare, inherited blood disorder caused by the production of abnormal hemoglobin that disrupts the ability of red blood cells to carry oxygen throughout the body.
The 52-week RISE UP trial randomized participants to receive either mitapivat or placebo. The trial met the primary endpoint of hemoglobin response, with 40.6% of patients in the mitapivat arm achieving a response compared with 2.9% in the placebo arm. Hemoglobin response is defined as a 1 g/dL increase from baseline in average hemoglobin concentration from Week 24 through Week 52.
However, the study did not meet the second primary endpoint of annualized rate of sickle cell pain crises (SCPCs), which was 2.62 in the mitapivat group versus 3.05 in placebo. SCPCs were defined as acute pain requiring medical contact, acute chest syndrome, priapism, or hepatic or splenic sequestration.
Agios said it plans to submit a U.S. marketing application for mitapivat in sickle cell disease after a pre-sNDA meeting with the FDA in the first quarter of 2026, and remains focused on other near-term milestones, including potential U.S. approval of PYRUKYND for thalassemia, expected in early December 2025.
Agios shares closed at $45.49 on Tuesday, up 0.8%.
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