A significant advancement in cancer treatment has emerged with the submission of an Investigational New Drug (IND) application to the U.S. FDA for a novel mRNA destabilizing therapy designed to target cancers driven by the c-MYC oncogene. Although the initial clinical trials will focus on triple-negative breast, pancreatic, colorectal, and ovarian cancers, the potential impact on prostate cancer, a disease where c-MYC overexpression is known to drive aggressive and treatment-resistant forms-is considerable.
This innovative drug employs a proprietary 3’UTR engineering technology that destabilizes c-MYC mRNA, leading to its degradation through molecular mechanisms found specifically in tumor cells. This strategy overcomes longstanding challenges associated with targeting c-MYC, a protein implicated in over 75% of human cancers but historically considered “undruggable” due to its structure and the risk of off-target effects. Preclinical studies have shown strong efficacy in reducing tumor growth and metastasis in animal models, without dose-limiting toxicities even at high doses.
In prostate cancer, where c-MYC amplification is a key driver of progression to lethal castration-resistant disease, this approach could address a critical unmet need. By targeting the mRNA blueprint of c-MYC, the drug disrupts the oncogene’s role in promoting cell proliferation, metabolism, and immune evasion-factors central to the disease’s aggressiveness. Although prostate cancer is not yet included in the upcoming Phase I trial scheduled for 2026, the broad applicability of the technology suggests future opportunities for this indication.
The developers of the therapy describe this IND submission as a transformative step in precision oncology, emphasizing that the ability to therapeutically degrade specific mRNA transcripts could redefine cancer treatment. The drug’s delivery system, which enhances targeting specificity, is designed to minimize side effects by sparing healthy cells, contrasting sharply with traditional chemotherapy approaches.

Source.