U.S. FDA grants Fast Track designation to ICT01 for the treatment of newly diagnosed AML

On September 18, 2024, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to ICT01, a humanized anti-butyrophilin 3A monoclonal antibody, in combination with azacitidine and venetoclax, for the treatment of patients aged ≥75 years with newly diagnosed acute myeloid leukemia (AML) unfit for induction chemotherapy.¹ The decision is based on the encouraging results from the phase I dose-escalation portion of the EVICTION trial (NCT04243499).^1,2

The EVICTION trial

EVICTION is an ongoing open-label, dose-escalation, phase I/IIa trial evaluating ICT01 in patients with relapsed/refractory (R/R) hematological malignancies.¹ Interim findings were presented at the European Society for Medical Oncology (ESMO) Congress 2023.³ Overall, 26 patients with R/R hematological malignancies received ICT01 monotherapy (AML; n = 24, follicular lymphoma; n = 1, diffuse large B-cell lymphoma; n = 1), at doses ranging from 200 μg to 75 mg every 21 days. The key findings were as follows:³

• No dose-limiting toxicities were reported.
• Among 10 evaluable patients at Week 8, the primary endpoint of disease control rate was 30%.
• The treatment safely induced γ9δ2 T-cell activation and migration from the blood within hours of administration, showing effective target engagement.

A dose-optimizing and efficacy-estimating portion has been initiated to evaluate two doses of ICT01 in combination with azacitidine and venetoclax in newly diagnosed patients with AML who are older or ineligible for standard chemotherapy. To date, 29 patients have been enrolled.¹