UTR Therapeutics Publishes Landmark Preclinical Data in Frontiers in Pharmacology Supporting IND Submission for First-in-Class c-MYC RNA Therapeutic

NEW YORK, Sept. 16, 2025 (GLOBE NEWSWIRE) -- UTR Therapeutics Inc., a biotechnology company advancing a novel RNA Overwriting platform, today announced the peer-reviewed publication of preclinical data in Frontiers in Pharmacology demonstrating compelling efficacy of its lead candidate UTRxM1-18 in aggressive pancreatic cancer models. These findings are part of the company’s Investigational New Drug (IND) submission to the U.S. Food and Drug Administration, filed in May 2025.

A Breakthrough in Pancreatic Cancer and c-MYC Targeting

Pancreatic cancer is among the most aggressive and treatment-resistant malignancies, with a five-year survival rate below 12%. Central to its pathology is the overexpression of c-MYC, a transcription factor long deemed “undruggable.” UTRxM1-18 directly destabilizes c-MYC mRNA through engineered 3′UTR elements — representing a first-in-class approach to overwriting oncogenic drivers upstream of protein production.

In the published study, UTRxM1-18 produced dose-dependent tumor regression, 100% complete pathological responses at the highest tested dose, and significant inhibition of liver, lung, and brain metastases in preclinical pancreatic cancer models. These results were accompanied by marked dose dependent reductions in c-MYC expression and downstream markers of tumor proliferation and immune cells evasion, including PD-L1.

Delivery Innovation: Iron-Oxide Nanocages

A critical component of UTRxM1-18’s success is its delivery system. The therapeutic is formulated with proprietary iron-oxide nanocages, which enabled broad tumor biodistribution, penetration of metastatic sites, and a serum half-life exceeding 24 hours. This pharmacokinetic profile represents a significant advancement over conventional RNA-based therapeutics, which are often limited by rapid clearance and poor tumor uptake.

“The combination of RNA Overwriting with our nanocage delivery system achieves what was once thought impossible — drugging c-MYC in one of the deadliest cancers,” said Dr. Chidiebere U Awah MD PhD, CEO of UTR Therapeutics. “The peer-reviewed data provide compelling evidence of both efficacy and safety and directly support our IND submission as we advance into first-in-human trials.”

Safety and Translational Relevance

Across studies, UTRxM1-18 demonstrated a favorable safety profile, with treated animals maintaining stable body weight and showing no significant systemic toxicities to blood, liver, kidney, electrolytes & pancreatic functions. Together, these findings reinforce the therapeutic window for targeting c-MYC with this approach.

Platform Potential Beyond Pancreatic Cancer

c-MYC overexpression drives a wide range of malignancies, including breast, lung, and hematologic cancers. UTR Therapeutics’ RNA Overwriting platform is designed to be modular, enabling expansion into multiple disease indications.

“By directly destabilizing oncogenic RNA, UTRxM1-18 offers a new therapeutic modality that could transform how we approach cancers and diseases previously considered untreatable,” said Dr. David Asuzu, Chief Medical Officer of UTR Therapeutics.

Next Steps

UTR Therapeutics plans to initiate a Phase 1 trial of UTRxM1-18 in patients with advanced c-MYC–driven tumors pending regulatory approvals. The company is also exploring translational and commercial applications of its platform in global markets.

About UTR Therapeutics
UTR Therapeutics Inc. (New York, NY) is an IND-stage biotechnology company developing first-in-class RNA Overwriting therapeutics that reprogram gene expression by selectively destabilizing pathogenic mRNA transcripts. The company’s lead program, UTRxM1-18, targets c-MYC and is delivered via proprietary iron-oxide nanocage technology. UTR Therapeutics IND for UTRxM1-18 to the FDA is supported by peer-reviewed data demonstrating complete pathological responses and metastasis inhibition in aggressive pancreatic cancer models, triple negative breast cancer, metastatic ovarian cancers & invasive colorectal cancer.

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