TransCode Therapeutics Reports Further Progress on Phase 1a Clinical Trial with No Dose Limiting Toxicities Reported in Patients with Metastatic Cancer

• A total of 13 patients treated with four escalating doses of TTX-MC138
• No significant safety or dose limiting toxicities reported
• Two patients have so far maintained stable disease on treatment for at least seven months
• PD analysis at 24 hours post-dosing provides evidence of miR-10b target engagement

Excerpt from the Press Release:

BOSTON, May 1, 2025 /PRNewswire/ — TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology company committed to more effectively treating cancer using RNA therapeutics, today announced further progress in its Phase 1a clinical trial of TTX-MC138 in patients with metastatic cancer. 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. To date, 13 patients have received at least one dose of TTX-MC138 at 4 separate dose levels ranging from 0.8 mg/kg to 4.8 mg/kg. Two patients have been treated in the expanded enrollment.

Eight patients remain on study for continued treatment, receiving an additional dose of TTX-MC138 during each 28 day treatment cycle and may remain on study absent any significant safety observations or disease progression. To date, the two patients who have remained on study the longest have received seven doses of TTX-MC138 over the course of approximately seven months and have demonstrated stable disease. No significant safety or dose limiting toxicities have been reported to date in any of the trial’s 13 patients.   Ongoing analyses of PK activity from Cohorts 1, 2 and 3 suggest that TTX-MC138 demonstrates a PK/PD profile consistent with preclinical results and results from TransCode’s Phase 0 clinical trial. Specifically, the preliminary PK data follow a predictable dose-response relationship. Analysis of PD activity from cycle 1 treatments in Cohorts 1 and 2, treated with a dose of 0.8 mg/kg and 1.6 mg/kg respectively, demonstrates miR-10b target engagement at 24 hours post-infusion in 5 out of the 6 patients analyzed to date.

The observed tolerability profile and the available PK/PD results thus far supports advancement of the clinical trial to further evaluate safety and potential anti-tumor activity of TTX-MC138 in the planned dose expansion (Phase 1b) portion of the trial. 

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