Agios licenses SYK inhibitor cevidoplenib from Oscotec in $1bn ITP deal
Agios Pharmaceuticals has struck an exclusive global licence agreement with South Korean drug developer Oscotec to acquire rights to cevidoplenib, an oral spleen tyrosine kinase (SYK) inhibitor in development for immune thrombocytopenia (ITP). The Cambridge, Massachusetts-based company will pay $25 million upfront and could owe up to $140 million in development and regulatory milestones across up to three indications, plus tiered royalties of high single-digit to mid-teen percentages on net sales. Agios describes peak US sales potential at up to $1 billion.
ITP is a rare autoimmune blood disorder in which the immune system generates autoantibodies that mark platelets for destruction, leading to dangerously low platelet counts and elevated bleeding risk. The global patient population is estimated at around 200,000, with roughly 90,000 diagnosed adults in the US. Of that group, approximately 24,000 are refractory to initial therapies and progress to second-line treatment or beyond — the population Agios is primarily targeting.
Phase 2 data and the path to Phase 3
Cevidoplenib was evaluated in a 60-patient, randomised, double-blind, placebo-controlled Phase 2 trial (NCT04056195) in adults with persistent or chronic ITP, a heavily pretreated cohort in which 68% had received three or more prior lines of therapy. The study's novel primary endpoint — platelet count reaching at least 30,000/µL with doubling versus baseline, without rescue medication — did not achieve statistical significance. However, Agios points to durable and clinically meaningful responses across multiple secondary endpoints aligned with the primary endpoints used in ITP registrational trials, including consecutive platelet counts reaching thresholds of 30,000/µL and 50,000/µL. Tolerability was reported as favourable, with the most common treatment-related adverse events being transient liver enzyme elevations and gastrointestinal effects.
Chief executive Brian Goff described the asset as "backed by clinically meaningful Phase 2 data" and positioned cevidoplenib as a potential best-in-class option versus first-generation SYK inhibitors. The company intends to initiate Phase 3 development in the first half of 2028, subject to completion of additional chemistry, manufacturing, and controls work. Cevidoplenib holds FDA orphan drug designation for ITP. Oscotec retains an option to reacquire development and commercialisation rights for South Korea after Phase 3 results are available.
Market context and competitive landscape
The ITP treatment landscape has evolved considerably over the past decade. Approved options include thrombopoietin receptor agonists such as eltrombopag and romiplostim, the anti-CD20 agent rituximab used off-label, and more recently the neonatal Fc receptor blocker efgartigimod alfa, approved by the FDA in 2023. The SYK pathway has been explored previously: fostamatinib (marketed as Tavalisse by Rigel Pharmaceuticals) is an approved oral SYK inhibitor for chronic ITP, though its uptake has been limited in part by tolerability concerns — the precise competitive gap cevidoplenib's selectivity profile is designed to address.
The deal represents a portfolio diversification move for Agios, which built its reputation in rare haematology through pyruvate kinase activators and has been seeking to broaden beyond its existing commercial franchise. Licensing a late-stage, mechanistically differentiated asset with existing Phase 2 human data is consistent with that strategy, though investors will note the primary endpoint miss and will look for a robust Phase 3 design — including endpoint selection — before drawing firm conclusions about registration prospects. The company has indicated that 2026 operating expense guidance remains broadly unchanged, with the $25 million upfront treated as a one-off item outside its standing guidance.