CAMBRIDGE, Mass., June 1, 2026
Agios Pharmaceuticals has entered an exclusive global licensing agreement with South Korea-based Oscotec to develop and commercialize cevidoplenib, a next-generation oral spleen tyrosine kinase (SYK) inhibitor for immune thrombocytopenia (ITP) and other potential indications. Under the terms of the agreement, Agios gains worldwide rights to the late-stage therapy while Oscotec will receive a $25 million upfront payment, along with development, regulatory, commercial milestone payments, and tiered royalties on future sales. The deal strengthens Agios’ rare hematology pipeline and expands its reach into autoimmune blood disorders, a market the company estimates could represent up to $1 billion in peak U.S. annual sales potential.
Cevidoplenib Targets Significant Unmet Need in ITP
Immune thrombocytopenia (ITP) is a rare autoimmune disease in which the immune system destroys platelets, increasing the risk of serious bleeding complications. Agios plans to advance cevidoplenib as a potential treatment for patients who have relapsed after or become resistant to existing therapies. The drug is a highly selective SYK inhibitor designed to block autoantibody-mediated platelet destruction while potentially offering improved tolerability compared to first-generation SYK inhibitors. Globally, ITP affects approximately 200,000 people, including around 90,000 adults in the United States, with many patients requiring long-term treatment after failing first-line therapies. Cevidoplenib has already received FDA Orphan Drug Designation for the treatment of ITP.
Phase 2 Data Demonstrate Durable Platelet Responses
The licensing decision was supported by encouraging results from a global Phase 2 randomized, placebo-controlled study involving 60 heavily pretreated patients with persistent or chronic ITP. Although the trial’s novel primary endpoint did not achieve statistical significance, cevidoplenib demonstrated durable and clinically meaningful platelet responses across multiple key secondary endpoints commonly used in registrational ITP studies. Patients receiving cevidoplenib achieved sustained platelet count improvements compared with placebo, while maintaining a favorable safety profile. The most commonly reported treatment-related adverse events included transient liver enzyme elevations and gastrointestinal effects, with no new safety concerns identified. Agios expects to advance the program into Phase 3 development during the first half of 2028 following additional manufacturing and development activities.
Advertisement
Agreement Strengthens Agios’ Long-Term Growth Strategy
According to Agios Chief Executive Officer Brian Goff, the acquisition of cevidoplenib aligns with the company’s strategy of building a diversified portfolio focused on rare hematologic diseases. The company believes the therapy has the potential to become a best-in-class treatment option for ITP due to its selective mechanism of action and encouraging clinical profile. Under the agreement, Agios assumes full responsibility for future development and commercialization costs, while Oscotec retains an option to obtain exclusive development and commercialization rights in South Korea following Phase 3 results. The transaction enables Agios to expand beyond its existing hematology portfolio while maintaining financial discipline, with the company reaffirming that its 2026 operating expense guidance remains unchanged apart from the upfront licensing payment.
Source: Agios Pharmaceuticals press release
