UTR Therapeutics Inc. announced the peer-reviewed publication of compelling preclinical data in Frontiers in Pharmacology for its lead candidate UTRxM1-18, a first-in-class RNA therapeutic targeting c-MYC in aggressive pancreatic cancer models. The findings support the company's Investigational New Drug (IND) submission filed with the U.S. Food and Drug Administration in May 2025.
Breakthrough Results in Pancreatic Cancer Models
UTRxM1-18 demonstrated remarkable efficacy in preclinical studies, achieving dose-dependent tumor regression and 100% complete pathological responses at the highest tested dose. The therapeutic also showed significant inhibition of liver, lung, and brain metastases in pancreatic cancer models, accompanied by marked dose-dependent reductions in c-MYC expression and downstream markers of tumor proliferation and immune cell evasion, including PD-L1.
These results address a critical unmet need in pancreatic cancer, one of the most aggressive and treatment-resistant malignancies with a five-year survival rate below 12%. Central to pancreatic cancer pathology is the overexpression of c-MYC, a transcription factor long considered "undruggable."
Novel RNA Overwriting Mechanism
UTRxM1-18 represents a first-in-class approach that directly destabilizes c-MYC mRNA through engineered 3'UTR elements, overwriting oncogenic drivers upstream of protein production. This RNA Overwriting platform offers a new therapeutic modality for targeting previously untreatable cancers.
"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.
Advanced Delivery System
A critical component of UTRxM1-18's success is its proprietary iron-oxide nanocage delivery system, 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.
Safety Profile and Clinical Translation
Across preclinical 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, and pancreatic functions. These findings reinforce the therapeutic window for targeting c-MYC with this approach.
"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," added Dr. Awah.
Platform Expansion Potential
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. The company's IND submission is supported by data demonstrating efficacy in triple negative breast cancer, metastatic ovarian cancers, and invasive colorectal cancer models.
"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.
Clinical Development Plans
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.
