Leukogene Therapeutics Inc. (LTI), a biopharmaceutical company developing next-generation immunotherapies for hematologic and other malignancies, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation 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 recognizes 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 U.S. market exclusivity upon approval.

“We are honored that the FDA has recognized the therapeutic promise of LTI-214 by granting Orphan Drug Designation,” said Dr. Sandeep Gupta, CEO of Leukogene. “AML remains one of the most challenging hematologic cancers, and outcomes for relapsed or refractory patients remain poor. The LTI-214 program 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.”

Nathan Dolloff, PhD, Founder and CSO of 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.”

About Leukogene and M2T™-Platform

Leukogene Therapeutics is a preclinical-stage biopharmaceutical company focused on developing next-generation immunotherapies for the treatment of cancer. LTI’s M2T™ platform is a novel, recombinant, high affinity MHCII binding protein conjugated to tumor associated antigens. M2T™ functions as an antigen presenting cell engager that directly stimulates MHCII and generates a powerful T and B cell response against the chosen antigen.

LTI-214 is Leukogene’s myeloid-targeted immunotherapy designed to selectively eliminate CD33-positive leukemic blasts and leukemic stem cells that drive disease progression in AML. Preclinical studies have demonstrated robust anti-leukemic efficacy in AML models, with significant reduction in leukemic burden and prolonged survival. LTI-214 has also shown a favorable safety profile, with minimal off-target toxicity and no evidence of cytokine related storm in preclinical studies. Leukogene expects to initiate first-in-human clinical evaluation of LTI-214 in AML in the near term.

LTI-002, targeting tumor antigen mesothelin, is Leukogene’s second M2T™ platform candidate. It is currently in preclinical development for treating pancreatic, ovarian, and other solid tumors.

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