Agios Lands Global Cevidoplenib Rights in Rare Hematology Push

Agios Pharmaceuticals has entered a global licensing agreement with South Korea-based Oscotec for cevidoplenib, a late-stage investigational spleen tyrosine kinase (SYK) inhibitor. The deal grants Agios exclusive worldwide rights to develop and commercialize the oral therapy across all indications, strengthening its rare hematology portfolio and expanding its presence into immune thrombocytopenia (ITP).

Under the agreement, Oscotec will receive a $25 million upfront payment and may earn up to $140 million in development and regulatory milestones across multiple indications in the United States and Europe. The company is also eligible for commercial milestone payments and tiered royalties on future net sales.

Disease Context

ITP is a rare autoimmune blood disorder characterized by immune-mediated platelet destruction, leading to low platelet counts and an increased risk of bleeding. The disease affects an estimated 200,000 people worldwide, including approximately 90,000 adults in the United States.

Although several treatment options are available, many patients relapse or fail existing therapies, highlighting the need for treatments that provide durable platelet responses while maintaining manageable long-term safety.

Mechanism of Action

Cevidoplenib is a highly selective, next-generation SYK inhibitor developed with the goal of improving tolerability compared with first-generation SYK inhibitors. By blocking SYK-mediated immune signaling, the therapy aims to reduce autoantibody-driven platelet destruction, a key mechanism underlying ITP.

The oral agent has received U.S. FDA orphan drug designation for ITP and enters a growing treatment landscape that includes thrombopoietin receptor agonists, FcRn antagonists, and BTK inhibitors.

Clinical Evidence

The licensing agreement is supported by data from a global Phase 2 randomized, double-blind, placebo-controlled study involving 60 adults with persistent or chronic ITP who had relapsed after, or were refractory to, at least one prior therapy. Patients received placebo or cevidoplenib at doses of 200 mg or 400 mg twice daily for 12 weeks.

The study enrolled a heavily pretreated population. More than two-thirds of participants had received three or more prior lines of therapy, while most entered the trial with severe thrombocytopenia.

Although the trial’s novel primary endpoint did not achieve statistical significance, cevidoplenib demonstrated durable and clinically meaningful platelet responses across multiple secondary endpoints commonly used in ITP registrational studies. Investigators observed sustained platelet counts of at least 30,000/µL and 50,000/µL in treated patients compared with placebo.

The therapy also showed a favorable safety profile. The most commonly reported treatment-related adverse events were transient elevations in liver enzymes and gastrointestinal events. No new safety signals emerged during the study.

Clinical Development Plans

Agios expects to advance cevidoplenib into Phase 3 development during the first half of 2028 following additional chemistry, manufacturing, and controls (CMC) activities.

The transaction broadens the company’s rare hematology franchise beyond its existing portfolio and provides entry into the ITP market, where significant unmet need remains among patients who relapse or become refractory to current therapies.

Future clinical development will focus on confirming the durability of platelet responses and establishing cevidoplenib’s potential role as a long-term treatment option in ITP.

Reference

Agios Enters Exclusive Global License Agreement with Oscotec to Develop and Commercialize Next-Generation SYK Inhibitor Cevidoplenib – Agios Pharmaceuticals, Inc.