FDA Grants Breakthrough Therapy Designation to Ipsen's IPN60340 for First-Line Acute Myeloid Leukemia
核心洞察
The U.S. FDA granted Breakthrough Therapy Designation to Ipsen's IPN60340 (搜索) in combination with venetoclax and azacitidine for first-line treatment of unfit acute myeloid leukemia patients.
Phase I/II EVICTION trial data showed IPN60340 (搜索) combination therapy nearly doubled complete response rates compared to historical standard of care across all molecular subtypes.
IPN60340 (搜索) is a first-in-class monoclonal antibody targeting BTN3A (搜索) that activates γδ T cells to enhance anti-tumor immune responses.
The U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to Ipsen's investigational immunotherapy IPN60340 (搜索) in combination with venetoclax and azacitidine for first-line treatment of unfit acute myeloid leukemia, marking a significant regulatory milestone for this aggressive blood cancer affecting older adults.
The designation, announced January 13, 2026, is intended to expedite development and review of medicines for serious or life-threatening conditions with evidence of substantial clinical improvement. IPN60340 (搜索) previously received Orphan Drug Designations from both the FDA and European Medicines Agency in July 2025.
"This Breakthrough Therapy Designation recognizes both the urgent need for new treatment options for people living with acute myeloid leukemia and the promising data seen so far in the development program for IPN60340 (搜索)," said Christelle Huguet, PhD, EVP and Head of R&D at Ipsen.
Clinical Trial Results Drive FDA Decision
The breakthrough designation is based on compelling data from the Phase I/II EVICTION trial presented at the American Society of Hematology. In the single-arm study of 38 patients, treatment with IPN60340 (搜索) combined with venetoclax and azacitidine resulted in nearly double the complete response rates compared to historical standard of care data across all molecular subtypes in newly diagnosed patients, including subtypes typically less responsive to standard venetoclax-azacitidine therapy.
The combination therapy demonstrated encouraging efficacy while maintaining a well-tolerated safety profile, underscoring IPN60340 (搜索)'s potential as a novel immunotherapy to improve outcomes for AML patients. The updated clinical data from 57 patients showed consistently high response rates across the study population.
Novel Mechanism of Action
IPN60340 (搜索) represents a first-in-class humanized monoclonal antibody targeting BTN3A (搜索) (also known as CD277 (搜索)), a key immune-regulatory molecule broadly expressed across cancers. The drug promotes recognition and elimination of tumor cells by γδ T cells, which are responsible for immunosurveillance of malignancy and infections.
The three isoforms of BTN3A (搜索) targeted by IPN60340 (搜索) are overexpressed on many solid tumors including melanoma, urothelial cell, colorectal, ovarian, pancreatic, and lung cancers, as well as hematologic malignancies such as leukemia and lymphomas. By altering BTN3A conformation, IPN60340 selectively activates circulating γδ T cells, leading to their migration into tumor tissue and triggering a downstream immunological cascade through secretion of pro-inflammatory cytokines including IFNγ and TNFα.
EVICTION Trial Design
EVICTION is a first-in-human, dose-escalation and cohort-expansion clinical trial evaluating IPN60340 (搜索) in patients with various advanced relapsed or refractory solid or hematologic cancers that have exhausted standard-of-care treatment options, as well as newly-diagnosed AML. The trial is registered at clinicaltrials.gov under NCT04243499.
Target Population and Next Steps
IPN60340 (搜索) is specifically being evaluated for patients 75 years or older living with acute myeloid leukemia who, due to comorbidities, are prevented from receiving treatment with intensive chemotherapy. This represents a significant unmet medical need in the AML treatment landscape.
Based on the preliminary data supporting the breakthrough designation, Ipsen plans to discuss Phase II/III development plans with the FDA in the first half of 2026. The company expects to advance the program to the next stage of clinical development while working closely with regulatory authorities.
The breakthrough designation provides Ipsen with enhanced FDA communication and guidance throughout the development process, potentially accelerating the path to approval for this novel immunotherapy approach in acute myeloid leukemia.