Agios Pharmaceuticals AGIO has inked an agreement with Oscotec, a South Korea-based biotech, in-licensing exclusive global rights to develop and commercialize cevidoplenib for immune thrombocytopenia (ITP).
Cevidoplenib: A Late-Stage SYK Inhibitor
Cevidoplenib is a next-generation, late-stage, highly selective oral spleen tyrosine kinase (SYK) inhibitor being developed for the treatment of patients with ITP, a rare autoimmune disorder characterized by low platelet counts and an elevated risk of bleeding. The disease affects approximately 200,000 individuals globally, including about 90,000 adults in the United States.
The FDA previously granted Orphan Drug designation to cevidoplenib for ITP.
The agreement expands and diversifies Agios’ rare hematology portfolio by adding a potential treatment for ITP, with an estimated peak annual U.S. sales potential of up to $1 billion.
Agios already has a growing commercial presence in rare blood disorders through Pyrukynd (mitapivat), which is approved for hemolytic anemia in adults with pyruvate kinase (PK) deficiency and is marketed as Aqvesme in the United States for anemia associated with alpha- and beta-thalassemia. The company is also seeking to expand mitapivat’s reach into sickle cell disease (SCD). Agios submitted a supplemental new drug application to the FDA seeking accelerated approval for the potential label expansion of mitapivat in SCD in May.
Beyond mitapivat, Agios is advancing tebapivat, a next-generation PK activator, in a phase II study for SCD, with top-line data expected in the second half of 2026. Positive data could further strengthen the company’s position in rare hematologic diseases and broaden its long-term growth opportunities.
Despite the strategic significance of the deal, AGIO’s shares declined nearly 5% on Tuesday. This likely reflects investor concerns over the recent discontinuation of tebapivat in lower-risk myelodysplastic syndromes. Year to date, Agios shares have risen 2.9% compared with the industry’s 0.1% growth.
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Path Toward Phase III Development
Oscotec has already completed a phase II study of cevidoplenib in ITP patients, in which the drug demonstrated durable platelet responses and a favorable safety profile, although the study did not meet its primary endpoint.
Based on these findings, Agios intends to advance cevidoplenib into a phase III study for ITP, with initiation expected in the first half of 2028 following additional chemistry, manufacturing and controls work. However, Oscotec retains the exclusive rights to develop and commercialize cevidoplenib in South Korea after the phase III study data become available.
Terms of the Deal
Per the agreement, Agios will fund all future development and commercialization costs associated with cevidoplenib. As part of the deal, Oscotec will receive a $25 million upfront payment and may earn up to $140 million in development and regulatory milestone payments across up to three indications in the United States and Europe. Oscotec will be entitled to receive commercial milestone payments and tiered royalties ranging from the high single digits to the mid-teens on future net sales.
AGIO’s Zacks Rank & Stocks to Consider
Agios currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Indivior Pharmaceuticals INDV, Liquidia Corporation LQDA, and Immunocore IMCR, each currently sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 30 days, estimates for Indivior Pharmaceuticals’ 2026 earnings per share have increased from $3.00 to $3.35. Over the same period, EPS estimates for 2027 have risen to $3.33 from $3.69. INDV shares have lost 2.8% year to date.
Indivior Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 65.44%.
Over the past 30 days, estimates for Liquidia’s 2026 earnings per share have increased to $2.97 from $1.50. Over the same period, EPS estimates for 2027 have risen to $4.81 from $2.91. LQDA shares have gained 70.3% year to date.
Liquidia’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 54.40%.
Over the past 30 days, loss per share estimates for Immunocore’s 2026 have improved from 88 cents to earnings per share of 6 cents. Over the same period, EPS estimates for 2027 have risen from 24 cents to 87 cents. IMCR shares have lost 16.1% year to date.
Immunocore’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 46.66%.
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