ImCheck Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its lead program, ICT01, a humanized anti-butyrophilin 3A (BTN3A) monoclonal antibody designed to selectively activate γ9δ2 T cells, for the treatment of acute myeloid leukemia (AML).

AML remains a significant clinical challenge, particularly for older or unfit patients who are not eligible for intensive chemotherapy.

“Receiving FDA orphan drug designation for ICT01 is a significant recognition of ICT01’s innovative therapeutic potential to meet the urgent unmet medical needs of AML patients," said Stephan Braun, MD, PhD, Chief Medical Officer of ImCheck Therapeutics. "This important regulatory milestone reinforces our confidence that ICT01 will become the first immunotherapy for AML patients and supports our goal of rapidly advancing ICT01 into pivotal studies based on the unprecedented results observed in the clinic to date.”

In an oral presentation at the 2025 ASCO Annual Meeting, ImCheck reported results from the Phase I/II EVICTION study, evaluating ICT01 in combination with azacitidine and venetoclax (Aza-Ven) in newly diagnosed AML patients unfit for intensive chemotherapy.

“Orphan drug designation is a catalyst,” added Pierre d’Epenoux, Chief Executive Officer of ImCheck Therapeutics. “It validates our regulatory strategy, de-risks and supports clinical development acceleration, and sends a strong signal about the unique potential of ICT01 to transform AML treatment as well as other solid tumor indications.”

The FDA’s orphan drug designation is granted to drugs and biologics intended for the treatment, diagnosis, or prevention of rare diseases affecting fewer than 200,000 people in the United States.