Leukogene Therapeutics has received orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for M2T-CD33 (LTI-214), its immunotherapy candidate targeting acute myeloid leukaemia (AML).
The designation highlights the considerable unmet medical needs of patients with AML and recognises LTI-214’s therapeutic potential as a new treatment for this aggressive form of blood cancer.
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Upon approval, it provides Leukogene with various development incentives, including market exclusivity in the US, eligibility for tax credits on qualifying clinical trial expenses and exemption from certain FDA fees.
Leukogene CEO Dr Sandeep Gupta stated: “AML remains one of the most challenging haematologic cancers, and outcomes for relapsed or refractory patients remain poor.
“The LTI-214 programme embodies our commitment to advancing new immunotherapy approaches that are both potent and safer for patients. This designation represents an important step toward our goal of transforming the treatment paradigm for AML.”
LTI-214 has been developed to selectively target and eliminate cluster of differentiation 33 (CD33)-positive leukaemic stem cells and leukaemic blasts that contribute to the progression of AML.
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By GlobalDataPreclinical trials have shown strong anti-leukaemic efficacy, resulting in a significant reduction in both leukaemic burden and prolonged survival.
The candidate exhibited a favourable safety profile, showing minimal off-target toxicity and no indications of a cytokine-related storm.
Leukogene founder and chief scientific officer Nathan Dolloff stated: “This is an important step forward for Leukogene and the company’s major histocompatibility complex class II (MHCII) engager technology.
“The M2T platform is a completely new approach to cancer immunotherapy and the endorsement from the FDA is a testament to its high impact potential.”
Leukogene’s M2T platform is a recombinant, high affinity MHCII binding protein conjugated to tumour-associated antigens, designed to function as an engager of antigen-presenting cells.
LTI-002, which targets the tumour antigen mesothelin, is the company’s second candidate from the M2T platform currently in preclinical development for ovarian, pancreatic and other solid tumours.
