Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced an agreement with Oscotec, a clinical-stage drug discovery and development company focused on immunology and oncology, headquartered in South Korea, to license the exclusive global rights to cevidoplenib, a novel, next-generation, oral spleen tyrosine kinase (SYK) inhibitor.

Agios will focus on advancing cevidoplenib for the treatment of immune thrombocytopenia (ITP), a rare autoimmune blood disorder in which the immune system destroys platelets, leading to low platelet counts and an increased risk of bleeding. 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. Globally, ITP affects an estimated 200,000 individuals, including 90,000 adults diagnosed in the U.S.

Cevidoplenib is a highly selective SYK inhibitor designed to prevent the harmful autoantibody-mediated destruction of platelets – the key driver of ITP. It was also designed to address the limitations of first-generation SYK inhibitors, offering the potential for an improved tolerability profile that supports long-term, manageable care for patients. Cevidoplenib has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of ITP.

"Cevidoplenib is a next-generation SYK inhibitor uniquely designed to potentially offer improved tolerability and durability compared to first-generation SYK inhibitors. Backed by clinically meaningful Phase 2 data, we believe cevidoplenib has the opportunity to become a best-in-class treatment option for ITP," said Brian Goff, Chief Executive Officer, Agios. "Licensing this promising medicine is a natural extension of our therapeutic focus and expertise, expanding and diversifying our rare hematology portfolio into ITP – an autoimmune blood disorder with an urgent need for new treatment options. This agreement also aligns with our disciplined approach to capital allocation, enabling us to advance this novel medicine while remaining firmly focused on executing our 2026 strategic priorities."

Cevidoplenib has been evaluated in a global, randomized, 12-week Phase 2 trial assessing efficacy, safety, and dose response in adults with persistent or chronic ITP. The primary endpoint was platelet response, defined as platelet count ≥30,000/µL and doubling the platelet count compared to average platelet count during screening at any visit during the 12-week treatment period and without the use of rescue medication. While this novel primary endpoint did not achieve statistical significance, durable and clinically meaningful platelet responses were observed in the cevidoplenib arm compared with placebo across multiple secondary endpoints that align with primary endpoints used in ITP registrational trials. Additionally, cevidoplenib was well tolerated in the Phase 2 trial. Based on these results, Agios expects to advance cevidoplenib into Phase 3 development for ITP in the first half of 2028, following completion of additional chemistry, manufacturing, and controls (CMC) development work.

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 $25.0 million upfront payment and is eligible to receive up to $140.0 million 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 $25.0 million upfront payment to Oscotec.