FDA Grants Breakthrough Therapy Designation to Ipsen's Treatment for Acute Myeloid Leukemia
The U.S. regulator has awarded the 'Breakthrough Therapy' designation to Ipsen's IPN60340, an experimental monoclonal antibody for patients with acute myeloid leukemia who are ineligible for intensive chemotherapy, according to a press release dated January 13, 2026.
Accelerated Development for Promising AML Treatment
The U.S. Food and Drug Administration has granted the 'Breakthrough Therapy' designation to IPN60340, Ipsen reports. This status is intended to expedite the development and review of drugs aimed at serious diseases, based on evidence of substantial clinical improvement, the company specifies. The treatment is being developed in combination with venetoclax and azacitidine for first-line patients with acute myeloid leukemia who are ineligible for intensive chemotherapy or targeted treatments. IPN60340 is an experimental monoclonal antibody targeting BTN3A, an immune regulatory molecule widely expressed in cancers, the company emphasizes. The product had previously received 'Orphan Drug' designation from both the FDA and the European Medicines Agency in July 2025, the release recalls.
Promising Clinical Data from EVICTION Trial
This designation is based on data from the phase I/II EVICTION trial, Ipsen indicates. Updated clinical data, presented at the American Society of Hematology congress, involved 57 patients, the company specifies. According to the release, treatment with IPN60340 in combination with venetoclax and azacitidine, administered to 38 patients, resulted in nearly doubling the complete response rate compared to historical data from standard care. These results were observed across all molecular subtypes in newly diagnosed patients, including those typically less responsive to the standard of care, the company highlights. The combination also demonstrated good tolerance, Ipsen confirms. Based on these preliminary data, the group plans to discuss the design of phase II/III development plans with the FDA in the first half of 2026.
Mechanism and Potential of IPN60340
IPN60340 is a humanized monoclonal antibody directed against BTN3A, a protein overexpressed in many solid and hematological cancers, Ipsen explains. According to the company, this mechanism promotes the recognition and elimination of tumor cells by gamma-delta T lymphocytes, responsible for immune surveillance. By altering the conformation of BTN3A, IPN60340 selectively activates these circulating lymphocytes, leading to their migration to the tumor tissue and triggering an immunological cascade through the secretion of pro-inflammatory cytokines, the release details. This experimental therapy is being evaluated in individuals aged 75 years or older with acute myeloid leukemia who cannot receive intensive chemotherapy due to comorbidities, the company specifies.
