Ipsen’s IPN60340 (ICT01) Receives FDA Breakthrough Therapy Designation For First-Line Treatment Of Unfit Acute Myeloid Leukemia

Ipsen, announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for IPN60340 when used in combination with venetoclax and azacitidine (Ven-Aza) for first-line treatment of unfit patients with acute myeloid leukemia (AML). AML is an aggressive blood cancer that mainly affects older adults and often requires new therapeutic options for those who cannot tolerate intensive chemotherapy.

IPN60340 is an investigational, first-in-class monoclonal antibody that targets BTN3A, an important immune-regulatory molecule broadly present on cancer cells. The Breakthrough Therapy Designation is intended to accelerate the development and review of medicines that show strong potential to offer significant improvements for serious or life-threatening conditions. IPN60340 had previously been granted Orphan Drug Designation by both the FDA and the European Medicines Agency in July 2025.

Christelle Huguet, PhD, Executive Vice President and Head of R&D at Ipsen, stated that the designation reflects both the ongoing need for new AML treatments and the positive data emerging from the development of IPN60340. She noted that Ipsen looks forward to working closely with the FDA as the program advances into its next stages and as the company continues efforts to bring meaningful new therapies to people with cancer.

The Breakthrough Therapy Designation is supported by results from the Phase I/II EVICTION trial. Updated findings presented orally at the American Society of Hematology meeting, which included 57 patients, showed that IPN60340 combined with venetoclax and azacitidine delivered notably high response rates. Among the 38 patients treated with the combination, the complete response rate was nearly double that of historical standard-of-care outcomes across all molecular subtypes, including those typically considered less responsive to venetoclax and azacitidine alone. 

The combination was also well tolerated, further supporting the potential of IPN60340 as a promising immunotherapy approach for improving outcomes in AML. Based on these early but encouraging clinical results, Ipsen plans to discuss the design and progression of Phase II/III development with the FDA in the first half of 2026.