On October 14, 2025 TransCode Therapeutics, Inc. (NASDAQ: RNAZ) reported completion of Phase 1a clinical trial with TTX-MC138, an investigational inhibitor of microRNA-10b, or miR-10b. By meeting its primary safety endpoint and defining a recommended Phase 2 dose, TTX-MC138 is moving forward into the next stage of clinical evaluation to assess its efficacy across selected metastatic diseases and for multiple indications.
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Primary objectives of the trial focused on safety, tolerability, pharmacokinetics ("PK") and establishment of a Phase 2 dose (RP2D). A total of sixteen patients were treated across four escalating dose levels. No significant treatment-related safety events or dose limiting toxicities were observed.
TTX-MC138 was administered to 16 patients (77 total doses to date) with positive pharmacodynamic effects over all four administered dose range levels. Currently three patients remain on trial receiving TTX-MC138. The median treatment duration was four months. Importantly, the duration of treatment for all patients ranged from two to 12 cycles, indicative of tolerability and disease control. Forty-four per cent or seven out of sixteen patients were classified as having stable disease lasting 4 months or longer. Preliminary data in 16 patients showed positive pharmacodynamic effects over a wide dose range, consistent with preclinical models and TransCode’s Phase 0 clinical trial. Of note, one patient diagnosed with thyroid cancer and historic evidence of an increase in thyroglobulin levels, demonstrated a reversal of the trend during treatment and presented at the most recent measurement with undetectable thyroglobulin levels.
Study investigator William McKean, MD, PhD (The START Center for Cancer Research) stated, "The clinical benefit we are observing in patients treated with TTX-MC138 is compelling given the pre-clinical data and length of time the drug remains in the tumor cells".
Data analysis and monitoring are ongoing. A final clinical study report, scientific presentations and publications are planned.
(Press release, TransCode Therapeutics, OCT 14, 2025, View Source [SID1234656647])