TransCode Therapeutics Announces Completion of Cohort 3 Initial Dosing with Lead Candidate in Phase 1 Clinical Trial

• No significant safety or dose limiting toxicities reported
• PK data from Cohorts 1 and 2 consistent with preclinical and Phase 0 trial results

Excerpt from the Press Release:

BOSTON, Feb. 6, 2025 /PRNewswire/ — TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology company committed to more effectively treating cancer using RNA therapeutics, today announced that Cohort 3 of its Phase 1 clinical trial enrolled three patients and all have been dosed with TTX-MC138, its lead candidate.  The Safety Review Committee monitoring the clinical trial unanimously approved opening of the third cohort based on its review of Cohort 1 and 2 safety and pharmacokinetic (PK) data and is monitoring Cohort 3 as preliminary data becomes available. The dose administered to patients in the third cohort is approximately double the dose administered in the second cohort.

Several patients in the first and second cohort remain on study for continued treatment, receiving additional doses of TTX-MC138 over cycles of approximately 28 days each.  No significant safety or dose limiting toxicities have been reported in any of the trial’s nine patients.  Analyses of PK data and pharmacodynamic (PD) activity from Cohorts 1 and 2 is ongoing. To date, the analyses suggest that TTX-MC138 demonstrates a PK/PD profile consistent with preclinical results and results from TransCode’s previous Phase 0 clinical trial.  Specifically, results from Cohort 1 confirmed the Phase 0 observation that TTX-MC138 shows evidence of pharmacodynamic activity in the presence of high baseline expression of miR-10b, reaching a 66% inhibition at 24 hours after infusion, similar to that seen in the Phase 0 trial. Additionally, TTX-MC138 activity increased with the escalated dose administered in Cohort 2 and was consistent at subsequent administrations suggesting a favorable pharmacokinetic profile.

About TTX-MC138

TTX-MC138 is a first-in-class therapeutic candidate designed to inhibit microRNA-10b, or miR-10b, a microRNA widely believed to be critical to the emergence and progression of many metastatic cancers. TransCode’s 2023 Phase 0 clinical trial produced evidence of delivery of a radiolabeled version of TTX-MC138 to metastatic lesions and pharmacodynamic activity, even at a microdose of the drug candidate, suggesting a broad therapeutic window for TTX-MC138.  

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