UTRx Inc. submitted an Investigational New Drug (IND) application to the FDA for UTRxM1-18, a novel therapeutic targeting c-MYC driven cancers. This drug utilizes their proprietary 3’UTR engineering technology, which allows for precise targeting and degradation of specific mRNA transcripts. The first-in-human Phase 1 trial is anticipated in 2026, pending FDA approval.
This IND submission is potentially groundbreaking for cancer treatment, especially for cancers driven by c-MYC, a notoriously difficult-to-target oncogene overexpressed in a significant portion of human cancers. Previous attempts to target c-MYC have been hampered by off-target effects, leading to toxicity. UTRxM1-18 offers a novel approach that leverages molecular machinery specific to tumor cells, potentially minimizing harm to healthy cells and achieving higher efficacy with fewer side effects. This targeted approach may offer renewed hope for patients with difficult-to-treat cancers like triple-negative breast cancer, pancreatic cancer, colorectal cancer, and ovarian cancer, where effective therapies are limited.
UTRxM1-18 degrades oncogenic c-MYC by recognizing unique motifs and redirecting the ribosome’s function from protein synthesis to degradation. Preclinical studies have shown promising results with robust dose-dependent efficacy in various human-derived tumor models and no dose-limiting toxicities observed, even at high doses. This targeted approach offers a significant improvement over traditional chemotherapy, which often impacts both cancerous and healthy cells, leading to debilitating side effects.
This IND submission represents a pivotal step for UTRx Inc. and could significantly impact the oncology landscape. If clinical trials confirm preclinical findings, UTRxM1-18 has the potential to revolutionize the treatment of c-MYC driven cancers, offering a targeted and potentially more tolerable therapy for patients who currently have limited options. The success of this drug candidate could also validate the 3’UTR engineering platform, paving the way for the development of new therapies targeting other challenging oncogenes and diseases.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
