Agios Pharmaceuticals (NASDAQ:AGIO) said it has entered into a global license agreement with South Korea-based Oscotec for cevidoplenib, an oral next-generation Syk inhibitor being developed for immune thrombocytopenia, or ITP.
On a business update conference call, Agios executives described the transaction as an extension of the company’s strategy to build a broader rare hematology business beyond its existing work in hemolytic anemias with mitapivat and tebapivat.
Deal Terms and Development Plan
Chief Financial Officer Cecilia Jones said the agreement includes a $25 million upfront payment to Oscotec and up to $140 million in development and regulatory milestones. Oscotec is also eligible for tiered royalties and commercialization milestones if the product is successful.
Jones said the structure was designed to be “capital efficient,” with payments tied to development and regulatory milestones. Excluding the $25 million upfront payment, Agios still expects full-year 2026 operating expenses to be approximately flat compared with 2025, she said. In response to an analyst question, Jones said the upfront payment should be modeled in the second quarter as a research and development expense.
Agios plans to initiate a phase III trial of cevidoplenib in the first half of 2028, following additional chemistry, manufacturing and controls, or CMC, work. Executives said the immediate focus will be scaling up and optimizing manufacturing processes ahead of registrational studies.
Krishnan Viswanadhan, Agios’ Chief Strategy and Operations Officer, said the rate-limiting step before phase III is “CMC scale-up from a process development perspective,” which he described as typical for an oral molecule advancing into phase III trials.
Agios Sees ITP as an Adjacent Rare Hematology Opportunity
Chief Medical Officer and Head of Research and Development Dr. Sarah Gheuens described ITP as an autoimmune disorder in which antibody-driven platelet destruction increases bleeding risk. She said diagnosis is typically made by exclusion, often at platelet counts below 100,000, and patients can experience bruising, bleeding and fatigue.
Agios said approximately 200,000 patients are diagnosed with ITP globally, including about 90,000 adults in the United States. Goff said roughly 24,000 U.S. adults have failed a prior therapy, creating what the company views as a significant unmet need.
Gheuens said current ITP treatment options include corticosteroids in the first-line setting and thrombopoietin receptor agonists in the second-line setting. However, she said patients often cycle through multiple therapies without durable control.
Syk inhibition targets a central disease pathway in ITP by interrupting immune-mediated platelet clearance, Gheuens said. Agios executives argued that cevidoplenib may improve upon first-generation Syk inhibitors, which they said have been limited by tolerability and inconsistent durability.
Phase II Data and Safety Profile
Gheuens said cevidoplenib has shown dose-dependent activity in phase II, along with a favorable safety profile and no evident dose-limiting toxicities. She said the prior global randomized controlled phase II trial used a primary endpoint that was not aligned with endpoints typically used in ITP registrational trials.
When evaluated using platelet thresholds and durability-based measures more aligned with clinical practice and regulatory precedent, Agios observed separation versus placebo and sustained platelet control, particularly at the 400 milligram dose, Gheuens said.
Asked why Agios has confidence the phase II results can translate into phase III, Gheuens pointed to consistency across multiple secondary endpoints, including endpoints aligned with registrational trial designs. She also cited the favorable safety profile from the global phase II trial.
In response to a question about adverse events, Gheuens said gastrointestinal events such as nausea and diarrhea were limited in the phase II population. She also said ALT and AST increases were observed less frequently than with other agents, and that hypertension and neutropenia seen with some other compounds were “much less” with cevidoplenib.
Commercial Fit and Potential Combinations
Agios said ITP fits its existing rare hematology commercial model because patients are often treated by community hematologist-oncologists, similar to thalassemia. Chief Commercial Officer Tsveta Milanova said Agios sees significant overlap in the community treatment setting, although the company did not provide specific numbers.
Milanova said the company expects to leverage capabilities it has built for thalassemia and potentially sickle cell disease, including data-driven targeting, claims analytics, community engagement and patient services. She said near-term commercial investment will be minimal because phase III is not expected to begin until the first half of 2028.
Executives also discussed the potential for cevidoplenib to be used in combination regimens, noting that a well-tolerated agent could be important as ITP treatment evolves. Viswanadhan said cevidoplenib could maximize value as a monotherapy in second-line patients and potentially as part of combination therapy.
Other Pipeline Comments
During the question-and-answer session, Goff also addressed Agios’ recent decision to discontinue development of tebapivat in lower-risk myelodysplastic syndromes, or MDS. He said there is “no read-through to sickle cell disease,” describing MDS and sickle cell disease as very different conditions.
Gheuens said Agios discontinued the MDS program because the efficacy signal in a broad and more challenging patient population was not strong enough to support further development, although she said the safety profile was very good.
Goff closed the call by saying Agios remains focused on its core programs and upcoming data readouts. He said the company plans to share new mitapivat data in thalassemia and sickle cell disease at the 2026 European Hematology Association Congress in Stockholm, including an oral plenary presentation featuring the phase III RISE UP trial in sickle cell disease.
About Agios Pharmaceuticals (NASDAQ:AGIO)
Agios Pharmaceuticals, Inc is a biopharmaceutical company founded in 2008 as a spin-out from research at Dana-Farber Cancer Institute and the Broad Institute. Headquartered in Cambridge, Massachusetts, Agios focuses on understanding and targeting cellular metabolism to develop novel therapies for cancer and rare genetic diseases. The company’s scientific platform integrates genomic discovery, metabolic profiling and precision medicine approaches to identify and advance small-molecule candidates that correct or exploit metabolic dysfunction.
Agios’s lead products are IDH (isocitrate dehydrogenase) inhibitors that target specific cancer mutations.
