Ipsen Pipeline Boosted by Promising AML Data from ImCheck Unit
ICT01 drug, from the recently acquired ImCheck Therapeutics, shows durable responses and improved survival signals in hard-to-treat leukemia patients.
Ipsen’s significant investment in its oncology pipeline is showing early signs of paying off, as its pending acquisition target, ImCheck Therapeutics, reported encouraging new data for its lead cancer drug, ICT01, in treating Acute Myeloid Leukemia (AML).
Data presented at the 67th American Society of Hematology (ASH) Annual Meeting revealed that ICT01, when used in a triplet combination therapy, produced durable responses and an early signal of improved overall survival in newly diagnosed AML patients who are unable to tolerate intensive chemotherapy. The positive results provide a key validation for Ipsen's strategic move to acquire ImCheck, a deal valued at up to €1 billion, as it seeks to build out its portfolio of cancer treatments.
According to the press release issued by ImCheck, the updated findings from the Phase I/II EVICTION study are significant. In the trial, more than 90% of patients treated with a 10mg dose of ICT01 combined with the current standard-of-care agents azacitidine and venetoclax achieved complete remission by the end of the second treatment cycle.
More critically, the study demonstrated a promising survival signal. The 12-month overall survival rate for patients on the regimen was 62%, a figure that compares favorably to the roughly 54% survival rate seen in recent Phase 3 trials for the standard Aza-Ven combination alone. At a median follow-up of 10.8 months, the median duration of response had not yet been reached, suggesting the treatment's effects could be long-lasting.
This data provides an early de-risking event for Ipsen's acquisition of the French biotech company, which was announced in late October 2025. The deal was structured with a €350 million upfront payment and up to €650 million in additional payments contingent on clinical, regulatory, and commercial milestones. The strong performance of ICT01 in this difficult-to-treat patient population is a crucial first step toward unlocking that potential value.
The target patient group—older adults with AML who are ineligible for aggressive chemotherapy—represents a significant unmet medical need. While the introduction of venetoclax-based therapies has improved outcomes, long-term survival remains low, and many patients eventually relapse. ImCheck’s drug, a first-in-class monoclonal antibody, offers a novel mechanism of action that could provide a more effective and durable option.
The safety profile of the three-drug combination was also described as manageable, with a 30-day mortality rate of 4% and no deaths attributed to ICT01 itself. This is a critical factor in a frail patient population where treatment toxicity is a primary concern.
For Ipsen, a French pharmaceutical company heavily focused on oncology, specialty care, and neuroscience, the ImCheck acquisition is a strategic play to secure next-generation cancer assets. The positive ICT01 data reinforces this strategy, strengthening a pipeline that is central to the company’s future growth narrative.
The path forward includes further clinical development, with the 10mg dose of ICT01 having been endorsed by the U.S. Food and Drug Administration for future studies. Investors and analysts will be closely watching for the maturation of the survival data and the design of a pivotal Phase 3 trial, which will be the ultimate test of the drug's potential to become a new standard of care in AML.