Agios Pharmaceuticals (Nasdaq: AGIO) announced a license deal to obtain exclusive global rights to cevidoplenib (SKI-O-703), a next-generation oral SYK inhibitor developed by South Korean biotech Oscotec (KOSDAQ: 039200), in a deal that could reach USD 165 million in disclosed payments plus undisclosed commercial milestones and tiered royalties. The Cambridge, Massachusetts-based rare disease company is betting on cevidoplenib's differentiated tolerability profile to carve out a position in immune thrombocytopenia, a rare autoimmune blood disorder affecting an estimated 90,000 adults in the US.
Under the terms, Oscotec receives a USD 25 million upfront payment and is eligible for up to USD 140 million in development and regulatory milestones tied to as many as three indications in the US and Europe. Agios will also pay tiered royalties ranging from high single-digit to mid-teen percentages on net sales. Oscotec retains an option to reacquire exclusive development and commercialization rights in South Korea following the release of Phase III trial results.
Deal context
Cevidoplenib is an oral small molecule that inhibits spleen tyrosine kinase, or SYK, a signaling enzyme involved in both Fc gamma receptor-mediated platelet destruction by macrophages and B cell receptor-driven autoantibody production. Both pathways are central to ITP pathophysiology, in which autoantibodies mark platelets for immune-mediated clearance, resulting in dangerously low platelet counts and elevated bleeding risk. SYK inhibition as a mechanism in ITP is clinically validated — Rigel Pharmaceuticals' fostamatinib (Tavalisse) was the first approved SYK inhibitor in the indication — but cevidoplenib is described by Oscotec as a next-generation compound with enhanced selectivity designed to reduce the off-target activity associated with first-generation agents.
The asset has been evaluated in a global, randomized, double-blind, placebo-controlled Phase II trial (NCT04056195) enrolling 60 adults with persistent or chronic ITP who had relapsed after or were refractory to at least one prior therapy. Patients were heavily pretreated: 68.3% had received three or more prior lines of therapy, and 68.3% had platelet counts below 15,000 per microliter at baseline. The trial's primary endpoint — platelet count at or above 30,000 per microliter with at least a doubling from baseline, without rescue medication — did not achieve statistical significance. However, Agios has pointed to durable responses across secondary endpoints aligned with primary endpoints used in ITP registrational trials, as well as a clean tolerability signal, with transient liver enzyme elevations and gastrointestinal events as the most commonly reported treatment-related adverse events.