NEW YORK – UTR Therapeutics is planning to start a Phase I clinical trial of UTRxM1-18, its investigational mRNA destabilizer for treating c-MYC-driven cancers, in 2026 if the US Food and Drug Administration approves the investigational new drug application it submitted, the company said this week.
Overexpression of c-MYC, a transcription factor, occurs in more than 75 percent of cancers, the company said. To avoid off-target effects, UTRxM1-18 is designed to trigger degradation of cancer cells by overwriting the 3′UTR section of mRNA, which normally stabilizes mRNA in c-MYC genes. The drug involves a nanocage delivery system.
There aren't marketed treatments that target c-MYC, although other companies are also testing investigational products. In 2022, Omega Therapeutics launched a clinical trial of an mRNA therapeutic, called OTX-2002, that downregulates MYC by targeting it before it is transcribed. In late 2024, Omega paused the program in a reprioritization of its pipeline.
New York-based UTR said preclinical studies have shown promising safety and efficacy for UTRxM1-18, which leads the company's pipeline of drugs using its 3'UTR engineering platform. "The validation data supports the technological abilities of engineering mRNA stability elements on the 3'UTR of oncogenes for therapeutic use," Kevin Struhl, co-inventor of the technology, adviser to UTR, and professor of biological chemistry and molecular pharmacology at Harvard University, said in a statement.
UTRxM1-18 successfully inhibited primary and metastatic tumor growth and prolonged survival in mice with triple-negative breast cancer in a 2024 study. UTR said additional data show the treatment can target myriad tumor types, including pancreatic, colorectal, and ovarian cancer, which will also be included in the planned Phase I trial.