Key Takeaways:
Agios Pharmaceuticals Inc. (Nasdaq: AGIO) will present detailed results from its 207-patient RISE UP Phase 3 trial of mitapivat in sickle cell disease at the 31st European Hematology Association (EHA) Congress, the company announced May 12. The data has been selected for the distinguished Plenary Abstracts Session on June 13.
"The submission of the mitapivat sNDA represents an important milestone for the sickle cell community, which urgently needs new treatments that can address key underlying aspects of this debilitating and deadly disease," Sarah Gheuens, M.D., Ph.D., Chief Medical Officer and Head of R&D at Agios, said in a statement. She noted the sNDA is supported by data showing the drug improved hemoglobin and reduced hemolysis.
The global, placebo-controlled RISE UP trial met one of its two primary endpoints. Mitapivat demonstrated a statistically significant improvement in hemoglobin response, defined as a 1.0 g/dL or greater increase from baseline. However, it did not achieve statistical significance on its other primary endpoint, the annualized rate of sickle cell pain crises, though a reduction was observed.
The data provides the foundation for a supplemental New Drug Application (sNDA) that Agios has already submitted to the U.S. Food and Drug Administration for accelerated approval. If approved, mitapivat could become the first oral pyruvate kinase (PK) activator for sickle cell disease, a condition affecting about 100,000 people in the U.S.
Mitapivat is an oral PK activator designed to improve red blood cell health by increasing ATP levels. In the RISE UP study, 207 patients were randomized 2:1 to receive either 100 mg of mitapivat twice daily or a placebo for 52 weeks. Patients who achieved a hemoglobin response also saw clinically meaningful benefits in pain crises, related hospitalizations, and fatigue, according to the company.
Agios has filed for approval under the FDA's accelerated pathway, which requires a confirmatory clinical trial to verify clinical benefit. The company has already agreed with the FDA on the design of this trial, which will assess the drug's ability to reduce transfusion burden in approximately 159 patients. Agios expects to receive notice on the sNDA's filing acceptance and review timeline in the third quarter of 2026.
The presentation at EHA is a key step for Agios to build clinical support ahead of a potential U.S. approval. Investors will be watching for the FDA's response to the sNDA filing in the third quarter of 2026 for the next major catalyst.
This article is for informational purposes only and does not constitute investment advice.
