Leukogene Therapeutics announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to the company’s lead product candidate, M2T-CD33 (LTI-214), for the treatment of Acute Myeloid Leukemia (AML).

This designation underscores the significant unmet medical need for AML patients and recognises the therapeutic potential of LTI-214 as a novel and differentiated approach for this aggressive blood cancer. The designation may provide LTI with a range of development incentives, including eligibility for tax credits on qualified clinical trial costs, waiver of certain FDA fees, and US market exclusivity upon approval.

“We are honoured that the FDA has recognised the therapeutic promise of LTI-214 by granting Orphan Drug Designation. AML remains one of the most challenging hematologic 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,” said Dr Sandeep Gupta, CEO, Leukogene.

AML remains one of the most challenging hematologic cancers, and outcomes for relapsed or refractory patients are poor. The M2T-CD33 programme embodies our commitment to advancing new immunotherapy approaches that are both potent and safer for patients.

Nathan Dolloff, PhD, Founder and CSO, Leukogene, 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 FDA is a testament to its high impact potential.”