Agios licenses oral blood disorder drug from Oscotec in $165M deal

Agios Pharmaceuticals (NASDAQ:AGIO) has acquired an exclusive global license for Oscotec's cevidoplenib, an experimental oral treatment for a rare blood disorder, betting the asset can capture up to $1 billion in peak U.S. sales despite recently missing the main goal of a mid-stage trial.

Under the terms of the agreement, Agios will provide South Korea-based Oscotec with an upfront cash payment of $25 million.

Oscotec remains eligible to receive up to $140 million in additional development, regulatory, and commercial milestones, alongside tiered royalties on eventual net sales.

The therapy is a next-generation oral spleen tyrosine kinase (SYK) inhibitor targeting immune thrombocytopenia (ITP), an autoimmune disease that causes low blood platelet counts and increases bruising and bleeding risks.

The transaction comes after a Phase 2 clinical trial where cevidoplenib demonstrated clinically meaningful platelet responses and a favorable tolerability profile among patients.

However, the trial's primary endpoint did not reach statistical significance, representing a distinct clinical risk for Agios as it assumes full operational and financial control of the program.

Management remains confident in the asset's secondary efficacy signals and intends to initiate a pivotal Phase 3 clinical trial in the first half of 2028.

The licensing deal will not disrupt the company's near-term fiscal targets.

Agios confirmed that its full-year 2026 operating expense guidance remains roughly unchanged compared to 2025 spending levels, excluding the immediate impact of the $25 million upfront payment.