Agios Pharmaceuticals, Inc. (AGIO), a biopharmaceutical company focused on genetically defined diseases, has entered into an exclusive worldwide license agreement with Oscotec Inc to develop and commercialize, Cevidoplenib, a next-generation SYK inhibitor.
Cevidoplenib is designed to block signaling pathways that drive immune-mediated diseases, with a focus on immune thrombocytopenia (ITP), a rare autoimmune blood disorder where the immune system destroys platelets, increasing bleeding risk. The therapy has already shown clinically meaningful Phase 2 data meeting its primary endpoint improving platelet counts while maintaining a manageable safety profile. This positions Cevidoplenib as a potential best-in-class treatment option for ITP.
Under the terms of the agreement, Oscotec will receive an upfront payment of $25.0 million, plus $140.0 million in development, regulatory, and commercial milestone payments, as well as tiered royalties on net sales. Agios noted that it continues to expect its 2026 operating expense guidance to remain approximately flat compared to 2025, excluding the $25.0 million upfront payment to Oscotec.
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.
Cevidoplenib has received orphan drug designation from the FDA for ITP, underscoring its potential in a disease with limited treatment options. Agios plans to advance the drug into further clinical development, including Phase 2 trial, to expand its rare hematology portfolio.
Brian Goff, CEO of Agios, commented: "Cevidoplenib is a next-generation SYK inhibitor uniquely designed to potentially offer improved tolerability and durability compared to first-generation SYK inhibitors. Backed by Phase 2 data, we believe Cevidoplenib has the opportunity to become a best-in-class treatment option for ITP. Licensing this promising medicine expands our rare hematology portfolio while aligning with our disciplined approach to capital allocation."
AGIO has traded between $22.24 and $46.00 over the past year. The stock closed Friday's trading May 29, 2026, at $29.40, down 3.35%.
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