TransCode Therapeutics, Inc. (NASDAQ: RNAZ), a clinical-stage company developing immuno-oncology and RNA-based therapeutics for high-risk and advanced cancers, has announced the publication of a peer-reviewed article in Cancers detailing preclinical results for its lead therapeutic candidate, TTX-MC138, in a model of breast cancer bone metastasis. The study demonstrated that TTX-MC138 accumulated in metastatic bone lesions after systemic administration, reduced expression of microRNA-10b (miR-10b), increased expression of the downstream tumor-suppressor target HOXD10, and produced significant survival benefits compared with controls. Importantly, repeated dosing was well tolerated with no observed systemic toxicity. These findings further support TransCode's approach of inhibiting miR-10b using its proprietary oligonucleotide nanotechnology and suggest potential applicability across additional metastatic disease settings.
The research is a crucial step forward in addressing the unmet medical need for effective treatments for metastatic cancers, particularly those that have spread to the bone, which is a common site for breast cancer metastasis. Bone metastases can cause severe pain, fractures, and other complications, significantly impacting patients' quality of life. The current standard of care often involves palliative measures, and targeted therapies that can specifically address the underlying biology of metastasis are urgently needed.
TTX-MC138 is designed to target miR-10b, a well-documented biomarker of metastasis that is overexpressed in various aggressive tumors. By inhibiting this microRNA, the therapeutic aims to restore the expression of tumor-suppressor genes like HOXD10, thereby potentially preventing or reducing the spread of cancer. The positive results from this preclinical study provide strong evidence that this approach could be effective in a clinical setting, offering hope for patients with advanced cancers.
TransCode's proprietary oligonucleotide nanotechnology enables the targeted delivery of therapeutic agents to metastatic lesions, as demonstrated by the accumulation of TTX-MC138 in bone lesions in the study. This targeted delivery is critical for maximizing efficacy while minimizing systemic toxicity, which is a common challenge in cancer treatment. The observed safety profile in the study is particularly encouraging, as it suggests that the therapy could be well-tolerated in patients.
The publication of these findings in the peer-reviewed journal Cancers adds to the growing body of evidence supporting the potential of RNA-based therapeutics in oncology. It also underscores the importance of targeting specific molecular pathways involved in metastasis, a leading cause of cancer-related deaths. The implications of this research extend beyond breast cancer, as miR-10b is implicated in multiple cancer types, suggesting that TTX-MC138 could have broad applicability.
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The study's results are a significant milestone for TransCode as it progresses toward clinical development. With its lead candidate demonstrating survival benefits in a challenging metastatic model, the company is poised to advance TTX-MC138 into human trials, potentially offering a new therapeutic option for patients with metastatic cancers. The positive safety and efficacy data from this preclinical study lay a strong foundation for future clinical success.
