Agios Pharmaceuticals Inc. (NASDAQ: AGIO) announced an exclusive global licensing agreement with South Korea-based Oscotec to develop and commercialize cevidoplenib, an oral drug candidate for treating immune thrombocytopenia, a rare blood disorder.

Under the agreement, Agios will pay Oscotec $25 million upfront and up to $140 million in development and regulatory milestones across three indications in the U.S. and Europe. Oscotec will also receive commercial milestone payments and royalties ranging from high single digit to mid-teen percentages on future net sales.

Cevidoplenib is a spleen tyrosine kinase inhibitor designed to prevent the destruction of platelets in patients with immune thrombocytopenia. The condition affects approximately 200,000 individuals globally, including 90,000 adults in the U.S. The drug has received orphan drug designation from the FDA for treating the condition.

The drug candidate was evaluated in a global Phase 2 trial involving 60 patients with persistent or chronic immune thrombocytopenia. While the primary endpoint did not achieve statistical significance, Agios reported that durable platelet responses were observed across multiple secondary endpoints. The company expects to advance cevidoplenib into Phase 3 development in the first half of 2028.

Agios maintains its 2026 operating expense guidance will remain approximately flat compared to 2025, excluding the $25 million upfront payment. The Cambridge, Massachusetts-based company focuses on developing treatments for rare diseases, particularly in hematology.

Oscotec retains the option to secure exclusive development and commercialization rights to cevidoplenib in South Korea following the release of Phase 3 trial results.