Ipsen Introduces Breakthrough IPN60340 (ICT01) for the Treatment of Acute Myeloid Leukaemia
Ipsen has introduced IPN60340 in combination with venetoclax and azacitidine (Ven-Aza) for the treatment of newly diagnosed acute myeloid leukaemia (AML) in patients considered unfit for intensive chemotherapy.
IPN60340 is an investigational first-in-class monoclonal antibody targeting BTN3A, an immune-regulatory molecule widely expressed across various cancers. The FDA’s Breakthrough Therapy Designation is intended to accelerate the development and review of medicines for serious or life-threatening conditions where early evidence indicates substantial clinical improvement.
EVICTION is a first-in-human clinical trial evaluating IPN60340 in patients with advanced relapsed or refractory solid and haematological cancers, as well as in newly diagnosed AML.
The Breakthrough Therapy Designation is based on data from the ongoing Phase I/II EVICTION trial. Clinical results presented at the American Society of Hematology included findings from patients treated with IPN60340 in combination with venetoclax and azacitidine. In the single-arm study involving newly diagnosed AML patients considered unfit for intensive chemotherapy, response rates were assessed against historical data from existing standard-of-care regimens. Safety observations indicated that the combination regimen was generally manageable within the studied population.
IPN60340 had previously received Orphan Drug Designations from both the U.S. Food and Drug Administration and the European Medicines Agency in July 2025. Following the recent designation, further regulatory discussions are expected regarding the next stage of clinical development.
IPN60340 is a humanised anti-BTN3A monoclonal antibody designed to activate γ9δ2 T cells, which play a role in immune surveillance against cancer and infections. By binding to BTN3A, the therapy promotes activation and migration of these immune cells into tumour tissue, triggering a broader anti-tumour immune response through the release of pro-inflammatory cytokines. The therapy is currently under investigation for patients aged 75 years or older with AML who are unable to receive intensive chemotherapy due to comorbidities.
IPN60340 is among the first investigational anti-BTN3A therapies to receive U.S. FDA Breakthrough Therapy Designation in first-line unfit acute myeloid leukaemia, offering a potential new immunotherapy option for older patients living with this aggressive blood cancer.
