Ipsen Gains Breakthrough Therapy Status in the US for its Antibody IPN60340
French laboratory Ipsen announced that the US Food and Drug Administration has granted 'Breakthrough Therapy' designation to its drug candidate IPN60340, used in combination with venetoclax and azacitidine, for first-line treatment of acute myeloid leukemia in patients ineligible for intensive chemotherapy.
Encouraging Clinical Trial Results
According to the group, this designation is based on data from the phase I/II clinical trial named EVICTION. Updated results, presented at the American Society of Hematology congress involving 57 patients, showed encouraging response rates. In the treatment arm combining IPN60340 with venetoclax and azacitidine, the complete response rate almost doubled compared to historical data from standard care, Ipsen reports. This improvement was observed across all molecular subtypes of newly diagnosed patients, including those less sensitive to standard treatment. The group also noted that the combination demonstrated good tolerance. Based on these preliminary results, Ipsen plans to discuss the design of phase II/III studies with the FDA during the first half of 2026.
Mechanism and Potential of IPN60340
IPN60340 is an experimental monoclonal antibody targeting the BTN3A molecule, also known as CD277, a regulatory immune protein widely expressed in various types of cancers. According to the press release, this antibody works by altering the conformation of BTN3A, which enhances the activation of ?? T lymphocytes, a type of immune cell involved in tumor and infection surveillance. This activation leads to the migration of these lymphocytes to the tumor tissue and triggers a cascade of immune responses through the secretion of pro-inflammatory cytokines. The laboratory indicates that the three isoforms of BTN3A targeted by IPN60340 are overexpressed in many solid cancers, such as melanoma, urothelial, colorectal, ovarian, pancreatic, and lung carcinomas, as well as hematological cancers like leukemias and lymphomas. IPN60340 had previously received orphan drug designation from the FDA and the European Medicines Agency in July 2025.
About Acute Myeloid Leukemia
Acute myeloid leukemia is an aggressive blood cancer that primarily affects older individuals. According to the group, IPN60340 is being evaluated in patients aged 75 or older, ineligible for intensive chemotherapy due to comorbidities or other targeted treatments. The EVICTION trial, described as 'first-in-human', includes a dose escalation phase and a cohort expansion phase. It assesses the drug candidate in patients with advanced solid or hematological cancers, in relapse or refractory, who have exhausted standard therapeutic options, as well as in newly diagnosed patients with acute myeloid leukemia. The 'Breakthrough Therapy' designation aims to expedite the development and review of drugs intended to treat serious or potentially life-threatening diseases, based on evidence of substantial clinical improvement, the company reminds.
