U.S. FDA grants Breakthrough Therapy Designation for IPN60340 in first-line unfit acute myeloid leukemia

The U.S. Food and Drug Administration granted Breakthrough Therapy Designation to IPN60340 given with venetoclax and azacitidine for first-line unfit acute myeloid leukemia, based on early Phase I/II EVICTION trial data. Ipsen plans to discuss Phase II/III development plans with the FDA in the first half of 2026.

Ipsen announced that the U.S. Food and Drug Administration has granted Breakthrough Therapy Designation for IPN60340 in combination with venetoclax and azacitidine for first-line unfit acute myeloid leukemia. The designation is reported as based on data from the Phase I/II EVICTION trial, which included updated clinical results presented at the American Society of Hematology. IPN60340 is described as a first-in-class monoclonal antibody targeting BTN3A and under study for older adults with AML who are not candidates for intensive chemotherapy. Ipsen previously received Orphan Drug designations for IPN60340 in July 2025. Key facts: - The FDA granted Breakthrough Therapy Designation for IPN60340 plus venetoclax and azacitidine in first-line unfit acute myeloid leukemia, as announced by Ipsen. - The designation is based on Phase I/II EVICTION trial data; updated results presented at ASH included 57 patients overall and a subgroup of 38 treated with IPN60340 plus Ven-Aza. - In the single-arm cohort of 38 patients, the company reports a near doubling of complete response rates compared with historical standard-of-care data for venetoclax plus azacitidine. - IPN60340 is a humanized anti-BTN3A monoclonal antibody intended to activate γ9δ2 T cells and has shown anti-tumor activity across several cancer indications in early studies. - Ipsen expects to discuss Phase II/III study design with the FDA in the first half of 2026. Summary: Breakthrough Therapy Designation is intended to expedite development and review for medicines that show substantial clinical improvement over available therapies, and Ipsen reports that this designation for IPN60340 reflects promising early clinical data. Next procedural steps reported include planned discussions with the FDA on Phase II/III development in H1 2026.