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Agios secures global rights to develop and commercialise cevidoplenib for rare blood disorders
Tuesday, June 02, 2026
Agios Pharmaceuticals has entered into an exclusive global licensing agreement with South Korea-based Oscotec to develop and commercialise cevidoplenib, an investigational next-generation oral spleen tyrosine kinase (SYK) inhibitor.
Under the agreement, Agios will obtain worldwide rights to develop and market cevidoplenib across all indications. The company plans to focus initially on immune thrombocytopenia (ITP), a rare autoimmune blood disorder in which the immune system destroys platelets, increasing the risk of bleeding. The disease is estimated to affect around 200,000 people globally, including approximately 90,000 adults in the United States.
Cevidoplenib is a highly selective SYK inhibitor designed to prevent the antibody-driven destruction of platelets, which is a major cause of ITP. The therapy has also been developed to overcome limitations associated with first-generation SYK inhibitors, with the aim of providing improved tolerability for long-term treatment. The drug has received orphan drug designation from the US Food and Drug Administration for the treatment of ITP.
The therapy has been evaluated in a global, randomised Phase 2 clinical trial involving adults with persistent or chronic ITP. Although the study’s primary endpoint did not achieve statistical significance, the treatment demonstrated durable and clinically meaningful platelet responses across several secondary endpoints when compared with placebo. The trial also showed that cevidoplenib was generally well tolerated.
Agios expects to begin Phase 3 development of cevidoplenib for ITP in the first half of 2028, following the completion of additional chemistry, manufacturing and controls (CMC) activities.
Under the terms of the agreement, Oscotec will receive an upfront payment of $25 million and could earn up to $140 million in development and regulatory milestone payments related to as many as three indications in the United States and Europe. The company is also eligible for commercial milestone payments and royalties on future net sales. Oscotec retains the option to secure exclusive development and commercialisation rights for cevidoplenib in South Korea after Phase 3 trial results become available.
Agios stated that its 2026 operating expense guidance remains unchanged from 2025 levels, excluding the upfront payment made under the agreement.
Source: globenewswire.com
