TransCode Therapeutics Completes First Cohort Dosing in TTX-MC138 Phase 1 Trial

TransCode Therapeutics, Inc., a company dedicated to advancing cancer treatment through RNA therapeutics, has announced the successful dosing of all patients in the initial cohort of its Phase 1a clinical trial. The trial is evaluating TTX-MC138, the company's leading therapeutic candidate, which is designed to inhibit microRNA-10b, a molecule that plays a significant role in the development and spread of metastatic cancer. Each patient in the cohort has received their first dose and continues to participate in the study for ongoing treatment. Importantly, no significant safety concerns or dose-limiting toxicities have been reported so far.

Sue Duggan, TransCode’s Senior Vice President of Operations, expressed optimism about the rapid enrollment of the first cohort. She emphasized that the absence of dose-limiting toxicities supports the continuation of dosing and further assessment of TTX-MC138’s safety and tolerability. Duggan also noted the excitement surrounding the trial, particularly following the recent awarding of the Nobel Prize in Physiology and Medicine to the discoverers of microRNA. This discovery has shed light on the critical role of microRNA in gene regulation, underlining the potential impact of TTX-MC138.

MicroRNA is a naturally occurring regulatory molecule in cells, significant for its role in development, physiology, and disease. The 2024 Nobel Prize in Physiology and Medicine was awarded to Dr. Victor Ambros and Dr. Gary Ruvkun for their discovery of microRNA's function in controlling protein production and cell function.

TTX-MC138 stands out as a first-in-class therapeutic candidate targeting microRNA-10b, widely recognized as a driver of metastatic disease. TransCode’s 2023 Phase 0 clinical trial demonstrated the delivery of a radiolabeled version of TTX-MC138 to metastatic lesions and pharmacodynamic activity, even at low doses, suggesting a broad therapeutic window for the drug.

The Phase 1 clinical trial is structured as a multicenter, open-label, dose-escalation and dose-expansion study. Its primary aim is to gather critical data on the safety and tolerability of TTX-MC138 in patients with various metastatic solid cancers. Although not a primary endpoint, the trial may provide initial evidence of TTX-MC138’s clinical activity. The initial phase of the trial focuses on escalating dose levels to evaluate safety and tolerability. In the subsequent dose-expansion phase, the study will further assess the drug's safety, tolerability, and anti-tumor activity in specific tumor types selected based on preliminary dose-escalation results.

TransCode Therapeutics is a clinical-stage oncology company focused on combating metastatic disease through the smart design and delivery of RNA therapeutics using its proprietary TTX nanoparticle platform. The company’s leading therapeutic candidate, TTX-MC138, targets metastatic tumors overexpressing microRNA-10b, a well-known biomarker of metastasis. TransCode is also developing a portfolio of other RNA therapeutic candidates aimed at overcoming RNA delivery challenges to target novel genetic markers relevant to various cancers.

In summary, TransCode Therapeutics is making significant strides with its Phase 1a clinical trial of TTX-MC138. The successful dosing of the first cohort and the absence of major safety concerns are promising indicators for the continued development of this potential cancer treatment. As the trial progresses, further evaluations will shed light on the efficacy and broader therapeutic implications of TTX-MC138 in treating metastatic cancers.