Leukogene Therapeutics Inc. (LTI), a biopharmaceutical company focused on developing next-generation immunotherapies for blood and solid cancers, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its lead candidate M2T-CD33 (LTI-214) for the treatment of Acute Myeloid Leukemia (AML).
The designation highlights the significant unmet medical need for AML patients and recognizes LTI-214’s potential as a novel and differentiated immunotherapy for this aggressive blood cancer. It also provides Leukogene with several development incentives, including tax credits on clinical trial costs, waiver of certain FDA fees, and seven years of 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 are often poor. This milestone brings us closer to transforming the treatment paradigm for AML.”
Nathan Dolloff, PhD, Founder and Chief Scientific Officer of Leukogene, added that the designation validates the company’s Major Histocompatibility Complex Class II (MHCII) engager technology, which powers its proprietary M2T™ platform.
“The M2T platform represents a completely new approach to cancer immunotherapy,” Dolloff said. “The FDA’s recognition underscores its potential to make a high-impact difference for patients.”
The FDA’s decision marks a key regulatory milestone for Leukogene as it advances LTI-214 toward clinical development, reinforcing the company’s commitment to pioneering innovative, targeted immunotherapies for hematologic malignancies.