ImCheck Receives EMA Orphan Drug Designation for ICT01 in Acute Myeloid Leukaemia
Tuesday, July 22, 2025
ImCheck Therapeutics has received Orphan Drug Designation (ODD) from the European Medicines Agency (EMA) for its lead candidate ICT01, aimed at treating acute myeloid leukaemia (AML). ICT01 is a humanised monoclonal antibody targeting butyrophilin 3A (BTN3A), designed to selectively activate γ9δ2 T cells—an important component of the immune system involved in tumour surveillance.
This EMA designation follows a similar decision from the US FDA and further supports the therapeutic potential of ICT01 in AML, particularly for patients who are ineligible for intensive chemotherapy due to age or poor overall health. The designation also brings regulatory benefits such as reduced fees, scientific advice, and ten years of market exclusivity in the EU once the product is approved.
AML continues to pose a major treatment challenge, especially for older patients or those who cannot undergo aggressive therapies. Current standard non-intensive treatments, such as the combination of venetoclax and azacitidine, often fail to achieve long-term remission, and most patients are not considered suitable candidates for stem cell transplantation. As a result, effective new treatment options remain limited.
ICT01 offers a novel approach by targeting γ9δ2 T cells, which play a dual role in both innate and adaptive immunity. These cells have shown promise in controlling disease relapse and improving survival, particularly after transplantation. Unlike other immunotherapies that have not delivered meaningful clinical improvements in AML, ICT01 may enhance immune response through a different mechanism.
BTN3A, also known as CD277, is highly expressed in both solid tumours (such as melanoma, ovarian, lung, and colorectal cancers) and haematological cancers (such as leukaemia and lymphomas). It is also present on various immune cells, including T cells, B cells, NK cells, and γδ T cells. ICT01 activates γ9δ2 T cells by targeting all three isoforms of BTN3A, leading to their migration into tumour tissue and triggering the release of pro-inflammatory cytokines such as IFNγ and TNFα. This cascade strengthens the immune system’s anti-tumour activity.
Clinical data presented at major oncology conferences—including AACR, ASCO, ASH, ESMO, and SITC—have shown promising results for ICT01 across a range of cancers. These findings support its continued development as a potential immunotherapy for high-risk AML patients and other malignancies.
Source: globenewswire.com
