AGIO News & Events

Agios Pharmaceuticals presented detailed results from the 52-week double-blind period of the global RISE UP Phase 3 trial of mitapivat. These efficacy and safety results, which include new transfusion burden and hemoglobin responder analyses reinforcing the strong anti-hemolytic profile of mitapivat, were presented during the distinguished Plenary Abstracts Session at the 31st European Hematology Association, EHA, Congress in Stockholm, Sweden. Key highlights: Mitapivat demonstrated statistically significant improvement in hemoglobin response compared with placebo, with rapid onset and durable effects; New analyses showed patients in mitapivat arm had clinically meaningful reduction in transfusion burden compared with placebo; Patients in mitapivat arm who achieved hemoglobin response had clinically meaningful benefits across measures of sickle cell pain crises, fatigue, and other patient-reported outcomes; Mitapivat was well-tolerated, with a safety profile consistent with previous trials of mitapivat in sickle cell disease

Agios Pharmaceuticals announced an agreement with Oscotec, headquartered in South Korea, to license the exclusive global rights to cevidoplenib, an oral spleen tyrosine kinase inhibitor. Agios will focus on advancing cevidoplenib for the treatment of immune thrombocytopenia, or ITP, an autoimmune blood disorder in which the immune system destroys platelets. The goal of treatment in ITP is to reduce the risk of bleeding events by safely achieving stable platelet levels while minimizing the burden of treatment-related toxicities. Cevidoplenib has received orphan drug designation from the FDA for the treatment of ITP. Under the terms of the agreement, Agios will obtain exclusive global rights to develop and commercialize cevidoplenib across all indications and will assume full responsibility for future development and commercialization costs. Oscotec will receive a $25M upfront payment and is eligible to receive up to $140.0M in development and regulatory milestones for up to three indications in the U.S. and Europe, as well as commercial milestone payments and royalties ranging from high single digit to mid-teen on future net sales. Oscotec retains the option to secure exclusive development and commercialization rights to cevidoplenib in South Korea following the release of Phase 3 trial results. Agios continues to expect its 2026 operating expense guidance to be approximately flat compared to 2025, excluding the $25M upfront payment to Oscotec.