ICT01 Receives FDA Breakthrough Therapy in First Line for Unfit Patients With AML

The FDA has granted breakthrough therapy designation to ICT01 (IPN60340) in combination with venetoclax (Venclexta) and azacitidine (Vidaza) in the first-line setting for unfit patients with acute myeloid leukemia (AML).¹

ICT01 is a first-in-class BTN3A-targeted monoclonal antibody that selectively activates γ9δ2 T cells, which play a central role in immunosurveillance against malignancies and infections. The 3 isoforms of BTN3A are overexpressed across multiple solid tumors and hematologic malignancies and are also present on the surface of innate and adaptive immune cells.

This regulatory decision was supported by data from the phase 1/2 EVICTION trial (NCT04243499). Updated findings from cohort F of EVICTION were presented during the 2025 ASH Annual Meeting and showed high response rates with the triplet among newly diagnosed patients with AML (n = 57).² In the ICT01^low group (n = 38), the complete response (CR) rate was 71% (95% CI, 54%-85%) and the composite CR (CRc) rate was 84%. In the ICT01^high group (n = 16), the CR rate was 44% (95% CI, 20%-70%).

According to Ipsen, the drug’s developer, these CR rates were nearly doubled relative to those seen with historical standards of care, and responses were observed across all molecular subtypes, including less responsive subtypes.¹ The combination was also shown to be well tolerated and have a favorable risk-benefit profile.

“This breakthrough therapy designation recognizes both the urgent need for new treatment options for people living with AML and the promising data seen so far in the development program for [ICT01],” Christelle Huguet, PhD, executive vice president and head of Research and Development at Ipsen, stated in a news release. “We look forward to working closely with the FDA as we advance to the next stage of clinical development and continue to deliver medicines with the potential to be transformative to people living with cancer.”

Of note, IPN60340 was previously granted FDA fast track designation in combination with azacitidine and venetoclax as frontline treatment for patients 75 years of age or older with AML who are unfit for induction chemotherapy.³ The agent also received orphan drug designation in AML from both the FDA and European Medicines Agency in July of 2025.¹

What is the design of the EVICTION study?

EVICTION is a first-in-human, dose-escalation (part 1) and cohort-expansion (part 2) study evaluating IPN60340 in patients with advanced relapsed/refractory solid or hematologic cancers who have exhausted standard-of-care treatment options.⁴

Cohort F of EVICTION specifically included patients with newly diagnosed AML who were at least 75 years of age or ineligible for intensive chemotherapy.² Patients were excluded if they were eligible for intensive chemotherapy/hematopoietic stem cell transplantation; harbored translocations in 15;17, 8;21, or 16;16, or inversions in chromosome 16; had received a prior diagnosis of myeloproliferative neoplasm, polycythemia vera, chronic myeloid leukemia, or AML with BCR-ABL1 translocation; received prior venetoclax; had exposure to systemic corticosteroids in the past 28 days; or had active autoimmune disease.

Eligible patients enrolled onto the dose-finding phase (n = 35) were randomly assigned to 75 mg of ICT01 (ICT01^high) or 10 mg of ICT01 (ICT01^low), both in combination with venetoclax and azacitidine every 4 weeks. Patients proceeding to the dose-expansion phase (n = 22) received the recommended phase 2 dose of 10 mg.

The study’s primary end point was CR rate per European LeukemiaNet 2022 criteria. Secondary end points comprised composite CR rate, overall survival (OS), event-free survival, safety, pharmacokinetics, and pharmacodynamics.

What data were reported from EVICTION during ASH 2025?

At the October 6, 2025, data cutoff, 57 patients 51 to 87 years old were enrolled onto the trial. Of these, 41 received the ICT01^low dose and 16 received the ICT01^high dose.⁴

More than 90% of patients treated with the ICT01^low achieved a CRc as their best response by the end of cycle 2. At a median follow-up of 10.8 months (95% CI, 8.9-12.8), the median duration of response was not reached (NR) in the ICT01^low cohort and was 9.3 months in ICT01^high group.^2,4

The combination also showed an early trend toward favorable survival outcomes. The median OS in the ICT01^low cohort was NR (95% CI, 10.4–NR) vs 10.9 months (95% CI, 1.7-NR) in the ICT01^high group (HR, 0.5; 95% CI, 0.2-1.1; log-rank P = .07). The 12-month OS rates in these respective cohorts were 62% and 38%.

Regarding safety, hematological toxicities were as expected for a venetoclax/azacitidine–based regimen. The overall incidence of cytokine release syndrome was 10% and 13% in the ICT01^low and ICT01^high groups, respectively; most events were low grade. A lower incidence of grade 5 sepsis and pneumonia were also observed in the ICT01^low vs ICT01^high groups. The 30-day mortality rate was 4%, and no deaths were attributed to ICT01.⁴

References

1. U.S. FDA grants Ipsen’s IPN60340 (ICT01) breakthrough therapy designation in first line unfit acute myeloid leukemia. News Release. Ipsen. January 13, 2026. Accessed January 14, 2026. https://www.ipsen.com/press-release/u-s-fda-grants-ipsens-ipn60340-ict01-breakthrough-therapy-designation-in-first-line-unfit-acute-myeloid-leukemia-3218133/
2. Garciaz S, Dumas PY, Peterlin P, et al. γ9δ2 T-cell activation with ICT01 and azacitidine-venetoclax induces high rates of remission and overall survival in patients with newly diagnosed acute myeloid leukemia: results from the phase 1/2 study EVICTION. Blood. 2025;146(suppl 1): 652. doi:10.1182/blood-2025-652
3. ImCheck receives FDA fast track designation for ICT01 in combination with azacitidine and venetoclax in first-line acute myeloid leukemia for patients unfit for induction chemotherapy treatment. News release. ImCheck Therapeutics. September 18, 2024. Accessed January 14, 2026. https://www.imchecktherapeutics.com/all-press-release/news/imcheck-receives-fda-fast-track-designation-for-ict01-in-combination-with-azacitidine-and-venetoclax-in-first-line-acute-myeloid-leukemia-for-patients-unfit-for-induction-chemotherapy-treatment/
4. ImCheck reports durable responses and early overall survival signal with ICT01 in first-line AML at ASH 2025. News Release. ImCheck. December 7, 2025. Accessed January 14, 2026. https://www.imchecktherapeutics.com/all-press-release/news/imcheck-reports-durable-responses-and-early-overall-survival-signal-with-ict01-in-first-line-aml-at-ash-2025/