FDA Grants Orphan Drug Designation to M2T-CD33 in AML

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to M2T-CD33 (LTI-214) for the treatment of patients with acute myeloid leukemia (AML), according to a news release issued by Leukogene Therapeutics Inc., the biopharmaceutical company that is behind M2T-CD33.

The FDA may grant orphan drug designation to a drug or biological product intended to prevent, diagnose, or treat a rare disease or condition, the regulatory states on its website. This designation provides incentives for sponsors, including tax credits for qualified clinical trials, exemption from user fees, and the possibility of approximately seven years of market exclusivity after FDA approval.

Such a designation, according to the news release from Leukogene Therapeutics, underscores the significant unmet medical need for patients with AML, and recognizes the therapeutic potential of M2T-CD33.

“We are honored that the FDA has recognized the therapeutic promise of [M2T-CD33] by granting orphan drug designation,” said Dr. Sandeep Gupta, CEO of Leukogene, in a statement included in the news release. “AML remains one of the most challenging hematologic cancers, and outcomes for relapsed or refractory patients remain poor. The [M2T-CD33] 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, founder and chief scientific officer of Leukogene further stated in the news release, “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’s M2T Platform

The M2T platform, as Leukogene explained in the news release, 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.

M2T-CD33 is an immunotherapy specifically designed to selectively eliminate CD33-positive leukemic blasts and leukemic stem cells that drive disease progression in AML.

While preclinical studies, or those not using humans, have reportedly demonstrated robust anti-leukemic efficacy in AML models and a favorable safety profile with minimal off-target toxicity and no evidence of cytokine release storm, the company said it expects to initiate the first in-human clinical evaluation of the drug in patients with AML soon.

The drug, Leukogene’s second M2T platform candidate, is also in preclinical development for the treatment pancreatic, ovarian and other cancers.

More Information About AML

Leukemias, as explained by the American Cancer Society on its website, are blood cancers that start in the cells that would normally develop into different types of blood cells, and most often leukemia starts in early forms of white blood cells. AML, as the American Cancer Society explained, starts in the bone marrow, or the soft inner part of certain bones, where new blood cells are made, and most often quickly moves into the blood; it can sometimes spread to other body parts such as the lymph nodes, liver, spleen, central nervous system and testicles.

The American Cancer Society estimated that in 2025, approximately 22,010 people in the United States will receive a diagnosis of AML, and around 11,090 people will die from the disease, with AML accounting for a third of leukemias in adults and approximately 1% of all cancers.

References

1. “U.S. FDA Grants Orphan Drug Designation to Leukogene Therapeutics’ M2T-CD33 (LTI-214) for the Treatment of Acute Myeloid Leukemia,” news release; https://www.businesswire.com/news/home/20251105454890/en/U.S.-FDA-Grants-Orphan-Drug-Designation-to-Leukogene-Therapeutics-M2T-CD33-LTI-214-for-the-Treatment-of-Acute-Myeloid-Leukemia
2. “Designating an Orphan Product: Drugs and Biological Products,” FDA; https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/designating-orphan-product-drugs-and-biological-products
3. “What Is Acute Myeloid Leukemia (AML)?” American Cancer Society; https://www.cancer.org/cancer/types/acute-myeloid-leukemia/about/what-is-aml.html

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