Agios Secures $1B Sales Potential with $25M Cevidoplenib License for ITP
AGIO•Agios paid $25 million upfront for exclusive global rights to cevidoplenib, a late-stage oral SYK inhibitor targeting immune thrombocytopenia with $1 billion U.S. peak sales potential. The Phase 2 asset, which showed durable platelet responses and orphan drug designation, is slated for Phase 3 in H1 2028.
1. Agreement Details
Agios obtained exclusive global development and commercialization rights to cevidoplenib by paying a $25.0 million upfront fee to Oscotec and committing to up to $140.0 million in development and regulatory milestones plus tiered royalties ranging from high single digits to mid-teens on net sales. Oscotec retains an option for South Korean rights following Phase 3 data release.
2. Phase 2 Clinical Data
In a 12-week randomized trial, cevidoplenib demonstrated durable and clinically meaningful platelet responses compared to placebo across multiple secondary endpoints despite not meeting its novel primary endpoint, and it was well tolerated. The drug holds U.S. orphan drug designation for ITP based on its selective SYK inhibition mechanism.
3. Strategic Impact and Timeline
The agreement diversifies Agios’s rare hematology portfolio into immune thrombocytopenia while keeping 2026 operating expenses flat excluding the upfront payment. Agios plans to complete CMC work and initiate a Phase 3 trial in the first half of 2028 to support regulatory filings.
