Oscotec, the original developer of the lung cancer drug "Leclaza," has scored another major technology transfer by sealing a global out-licensing deal for "cevidoplenib," a treatment candidate for autoimmune diseases that once faced the risk of halted development. Following last year's out-licensing of an Alzheimer's disease drug candidate, the company has clinched large-scale contracts for two consecutive years, drawing attention to the potential for additional transfers.
On the 4th, Oscotec CEO Yoon Tae-young said at the "Cevidoplenib technology transfer briefing" held at the Korea Association of KOSDAQ Listed Companies auditorium in Yeouido, Seoul, "Agios Pharmaceuticals in the United States, which received the license this time, is aiming to enter phase 3 for immune thrombocytopenia (ITP) within a year and a half," adding, "Because it is a rare disease, the trial size is not large, so we expect approval and commercialization before 2030, after which milestone receipts will also begin."
On the 1st of this month, Oscotec signed a technology transfer agreement with U.S. biotech Agios to transfer the global exclusive rights to develop and commercialize cevidoplenib, an autoimmune disease treatment candidate. The deal is worth up to 1 trillion won, with Oscotec to receive 37.5 billion won upfront. The company will also receive stepwise milestone payments tied to development, approval, and commercialization, as well as separate sales royalties.
This agreement marks Oscotec's third technology transfer deal, following "Lazertinib," the active ingredient in the lung cancer drug candidate Leclaza (Yuhan), and "ADEL-Y01" (Sanofi), an Alzheimer's disease drug candidate co-developed with Adel.
Cevidoplenib is an oral small-molecule drug candidate that selectively inhibits spleen tyrosine kinase (SYK), an enzyme involved in regulating immune responses. SYK mediates intracellular signaling in immune cells and is an enzyme that triggers inflammation and autoimmune diseases. Oscotec and its subsidiary Genosco have co-developed the drug since 2010, and 25% of the revenue generated will be allocated to Genosco.
Cevidoplenib was once touted as the "second Leclaza," but its development path has not been smooth.
Earlier, in 2021, it effectively halted after failing to meet the primary endpoint in a phase 2 trial for rheumatoid arthritis. Subsequently, in a global phase 2 trial in patients with immune thrombocytopenia (ITP), some efficacy signals were observed, but the failure to achieve statistical significance significantly dampened market expectations.
Agios, however, offered a different interpretation. Yoon said, "Agios judged that while the primary endpoint in phase 2 was not met, a durable platelet response was confirmed in secondary endpoints," adding, "It is targeting entry into phase 3 within a year and a half in ITP patients in the United States."
Agios plans to pursue phase 3 focused on immune thrombocytopenia while also considering a strategy to broaden the treatment's target indications. Beyond immune thrombocytopenia, development could expand to as many as three additional diseases.
ITP and rheumatoid arthritis are both autoimmune diseases caused by abnormalities in the immune system, and cevidoplenib was developed on the principle of inhibiting SYK, which is involved in this process.
However, which diseases will be selected for additional development has not yet been decided. Yoon said, "We do not know which indications Agios will expand to," adding, "It depends entirely on Agios' judgment."
Oscotec said it has received research proposals from investigators for chronic graft-versus-host disease and lupus nephritis. It also mentioned autoimmune hemolytic anemia, antibody-mediated rejection (AbMR), and antiphospholipid syndrome (APS) as diseases where a mechanism similar to ITP could apply.
Agios is a company with strengths in rare hematologic diseases. It already has experience commercializing two acute myeloid leukemia (AML) treatments approved by the U.S. Food and Drug Administration (FDA).
Yoon said, "Because rare diseases do not require large trials, we expect approval and commercialization before 2030," adding, "We anticipate that the process to actual approval and commercialization will proceed relatively quickly, with milestone receipts also beginning."
Oscotec plans to pursue additional technology transfers rather than being satisfied with this agreement.
In December last year, the company signed an out-licensing agreement with Sanofi for an Alzheimer's disease drug candidate and set a goal of achieving three or more technology transfers by 2030. With the Agios deal securing a second major contract, expectations for follow-up results are rising.
Yoon said, "We are discussing additional technology transfers for two follow-up pipeline assets by 2027."
Leading candidates include the idiopathic pulmonary fibrosis treatment "GNS-3545" and the chronic renal failure treatment "OCT-648." GNS-3545 is slated to begin business development after confirming phase 1 results within the year, while OCT-648 will start preclinical studies this month and aims for a technology transfer to a global pharmaceutical company before entering phase 1.
Yoon said, "We are ramping up development of the fibrosis-related pipeline, including anti-resistance anticancer agents and OCT-648," adding, "While development is currently centered on small molecules, we are also preparing to expand into various modalities going forward."
Meanwhile, regarding the possibility of fully consolidating Oscotec subsidiary Genosco, Executive Director Shin Dong-joon said, "The strategic direction of full subsidiary integration has not changed," adding, "We are in the process of fully communicating with shareholders and building consensus to ensure fair valuation and legitimacy."