Agios Licenses Oscotec Blood Disorder Drug Cevidoplenib in $165M Deal Despite Phase 2 Setback

Agios Pharmaceuticals has signed an exclusive global licensing agreement with South Korea's Oscotec for the investigational oral spleen tyrosine kinase (SYK) inhibitor cevidoplenib, a drug targeting immune thrombocytopenia (ITP), a rare autoimmune blood disorder that causes low platelet counts and increased bleeding risk.

The deal has a total potential value of up to $165 million, with Agios paying an upfront $25 million to Oscotec and up to $140 million in future development, regulatory, and commercial milestones, plus tiered royalties on net sales.

Cevidoplenib recently missed the primary endpoint in a Phase 2 ITP trial but showed clinically meaningful platelet responses and a favorable safety profile on secondary measures, leading Agios to plan a Phase 3 trial in the first half of 2028.

By the terms of the agreement, Agios will assume all further global development and commercialization costs, while Oscotec retains an option to regain exclusive rights in South Korea after Phase 3 results, and the deal is expected to have limited short‑term impact on Agios' operating expenses beyond the upfront payment.