On May 8, 2025 TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology company committed to more effectively treating cancer using RNA therapeutics, reported that the third patient in Cohort 4 of its Phase 1a clinical trial has received their initial dose of TTX-MC138 (Press release, TransCode Therapeutics, MAY 8, 2025, View Source [SID1234652781]). All cohorts have enrolled at least three patients who have been dosed with TTX-MC138 at least once. The Safety Review Committee monitoring the clinical trial unanimously approved opening the fourth cohort based on its review of available safety and pharmacokinetic (PK) data. Additionally, the Safety Review Committee approved expanded enrollment in Cohort 3 to obtain additional safety data. To date, 15 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. Three patients have been treated in the expanded enrollment.
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Ten patients remain on study for continued treatment, receiving an additional dose of TTX-MC138 during each treatment cycle every 28 days, and may remain on study absent any significant safety observations or disease progression. Two patients who have remained on study the longest have received so far seven doses of TTX-MC138 over approximately seven months and have demonstrated stable disease. The 10 patients currently on study have shown no disease progression. Further, no significant safety or dose limiting toxicities have been reported in any of the trial’s 15 patients. Analysis of PK activity from Cohorts 1, 2 and 3 is ongoing and suggests that TTX-MC138 demonstrates a PK/PD profile consistent with preclinical results and results from TransCode’s Phase 0 clinical trial. Specifically, preliminary PK and pharmacodynamic (PD) data follow a predictable dose-response relationship. Analysis of PD activity from cycle 1 treatments in Cohorts 1 and 2, treated with doses of 0.8 mg/kg and 1.6 mg/kg, respectively, demonstrates miR-10b target engagement at 24 hours post-infusion.
The observed tolerability profile and the available PK/PD results thus far support 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.
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 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.
About the Trial
TransCode’s Phase 1 clinical trial is a multicenter, open-label, dose-escalation and dose-expansion study designed to generate critical data to support evaluation of the safety and tolerability of TTX-MC138 in patients with a variety of metastatic solid cancers. While not an endpoint, the trial may provide early evidence of clinical activity of TTX-MC138. The trial comprises an initial dose-escalation stage followed by a dose-expansion stage. The primary objective of the dose-escalation stage is to evaluate the safety and tolerability of escalating dose levels of TTX-MC138. In the dose-expansion stage, the safety, tolerability and anti-tumor activity of TTX-MC138 will be further evaluated in certain tumor types and at a certain dose level selected based on preliminary results from the dose-escalation phase.
Further information is available at www.clinicaltrials.gov NCT Identifier: (NCT06260774).