TransCode Therapeutics Opens Third Cohort and Shares Initial Phase 1 TTX-MC138 Trial Results

TransCode Therapeutics, Inc., an RNA oncology company focused on developing innovative RNA-based treatments for cancer, has reported a significant milestone in its Phase 1 clinical trial for its lead therapeutic candidate, TTX-MC138. The Safety Review Committee (SRC), which oversees patient safety during the trial, has unanimously approved the initiation of the third cohort of patients. This decision follows a favorable safety evaluation of data from the second cohort, which did not reveal any significant safety concerns or dose-limiting toxicities.

TTX-MC138 is designed to inhibit microRNA-10b (miR-10b), a microRNA implicated in the progression of various metastatic cancers. The dose for the third cohort will be approximately twice that of the second cohort. Several patients from the initial cohorts continue to receive treatment without notable safety issues.

The preliminary pharmacokinetic (PK) and pharmacodynamic (PD) data from the first cohort align with previous preclinical and Phase 0 trial results. Specifically, TTX-MC138 demonstrated a PK/PD profile indicative of its effective pharmacodynamic activity, achieving a 66% inhibition of miR-10b at 24 hours post-infusion. Moreover, the concentration of TTX-MC138 in blood plasma was higher than expected based on nonclinical studies, suggesting a favorable pharmacokinetic profile.

Sue Duggan, Senior Vice President of Operations at TransCode, explained that the SRC plays a critical role in determining the progression of clinical trials, including dose adjustments, based on safety data. The current approval allows for the enrollment and screening of eligible patients for the third cohort, where they will receive the next dose level of TTX-MC138 as preliminary data analysis continues.

TTX-MC138 represents a first-in-class therapeutic targeting miR-10b, a driver of metastatic disease. Evidence from a 2023 Phase 0 trial indicated successful delivery of a radiolabeled version of TTX-MC138 to metastatic lesions, even at low doses, suggesting a wide therapeutic window for the drug candidate.

The ongoing Phase 1 clinical trial is a multicenter, open-label study designed to evaluate the safety and tolerability of TTX-MC138 in patients with various metastatic solid tumors. The trial encompasses a dose-escalation phase, aimed at assessing safety and tolerability at increasing dose levels, followed by a dose-expansion phase to further evaluate these parameters and the anti-tumor activity of TTX-MC138 in selected tumor types.

TransCode Therapeutics is a clinical-stage oncology company dedicated to treating metastatic disease through advanced RNA therapeutics, leveraging its proprietary TTX nanoparticle platform. The company's efforts are focused on overcoming challenges in RNA delivery to unlock therapeutic potential for various cancers. TTX-MC138, their lead candidate, targets cancers that overexpress miR-10b, a well-documented biomarker of metastasis, with the aim of effectively combating metastatic tumors.

With the approval to proceed with Cohort 3 and promising preliminary data, TransCode continues its mission to transform cancer treatment through innovative RNA-based solutions.