UTR Therapeutics Inc. Announces U.S. FDA Orphan Drug Designation and Rare Pediatric Disease Designation for UTRxMYCN M1-14

U.S. FDA Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) underscore UTRxMYCN M1-14's therapeutic targeting of the "undruggable" MYCN (N-MYC) in rare, aggressive soft tissue sarcomas, addressing a significant unmet clinical need in pediatric and adult sarcomas.

NEW YORK, Sept. 29, 2026 /PRNewswire/ -- UTR Therapeutics Inc., a biotech company with clinical trial-ready assets defining a new category of RNA therapeutics to conquer the undruggable proteome, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to 3′UTRMYCN M1-14 (UTRxMYCN M1-14) for the treatment of soft tissue sarcoma, including rhabdomyosarcoma (RMS), and Rare Pediatric Disease Designation (RPDD) for the treatment of rhabdomyosarcoma.

The company's core technology of ultra-targeted 3′UTR engineering, invented by Chidiebere U. Awah, M.D., Ph.D. (CEO, UTR Therapeutics Inc.), targets the oncogenic mRNA of undruggable genes, overwrites their endogenous oncogenic mRNA messages, and triggers them for degradation by the nonsense-mediated decay pathway, thus eliminating the diseased mRNA while sparing the healthy mRNA.

MYCN (N-MYC) is a basic helix-loop-helix transcription factor belonging to the MYC superfamily. It is an intrinsically disordered transcription factor with no binding pocket, which has rendered it "undruggable" and beyond the reach of conventional methods. MYCN is a defining oncogenic driver of aggressive soft tissue sarcomas, including fusion-positive rhabdomyosarcoma, the most common soft tissue sarcoma in children. MYCN contributes to a subset of the more than 80 soft tissue sarcoma subtypes affecting children and adults. There are currently no clinically approved therapies that directly target MYCN.

In preclinical studies, UTRxMYCN M1-14, invented by Dr. Awah at UTR Therapeutics Inc., reliably targeted and degraded the oncogenic MYCN in aggressive MYCN-driven rhabdomyosarcoma, including the deadly fusion-positive rhabdomyosarcoma. The drug achieved on-target specificity and therapeutic efficacy, demonstrating dose-dependent inhibition of MYCN, tumor inhibition, and inhibition of liver and lung metastasis while remaining safe.

"The ODD and RPDD designations granted to UTRxMYCN M1-14 by the U.S. FDA validate the UTR Therapeutics platform technology and the potential of UTRxMYCN M1-14 to address several aggressive sarcomas that represent serious unmet needs in the U.S. and worldwide," said David T. Asuzu, M.D., Chief Medical Officer.

About Orphan Drug Designation and Rare Pediatric Disease Designation

The FDA grants Orphan Drug Designation to drugs and biologics intended to treat diseases affecting fewer than 200,000 people in the United States. ODD provides seven years of U.S. market exclusivity upon approval, a tax credit for qualified clinical testing costs, and an exemption from prescription drug user fees.

The FDA grants Rare Pediatric Disease Designation to drugs for serious or life-threatening diseases that primarily affect individuals under 18. RPDD is a prerequisite for a Rare Pediatric Disease Priority Review Voucher, which may be awarded upon FDA approval of the asset, subject to the FDA's eligibility criteria at the time of approval. The Priority Review Voucher has been recorded to trade at values beyond $195M

About UTR Therapeutics Inc.

UTR Therapeutics Inc. is a biotech company headquartered in New York City, USA, advancing a first-in-class RNA Overwriting platform delivered by a proprietary Iron Oxide Nanocage (ION) delivery system. The platform is designed to identify and kill cancer-driving transcription factors that have resisted conventional drug modalities. UTR Therapeutics has clinical-ready assets spanning c-MYC, MYCN, TEAD1, YAP1, and HER2 (ERBB2), as well as assets for neurodegenerative disorders. These assets represent areas of high unmet medical need. For more information, visit www.utrtherapeutics.com

Forward-Looking Statements

This press release contains forward-looking statements regarding UTR's product candidates, development plans, market opportunity, and potential regulatory benefits. These statements are based on current expectations and are subject to risks. UTR undertakes no obligation to update these statements.

SOURCE UTR Therapeutics Inc.