On March 13, 2025 TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology company committed to more effectively treating cancer using RNA therapeutics, reported that the Safety Review Committee (SRC) monitoring its Phase I clinical trial has unanimously approved opening of the fourth cohort of patients based on the SRC’s favorable review of Cohort 3 safety data (Press release, TransCode Therapeutics, MAR 13, 2025, View Source [SID1234651139]). The therapeutic candidate being evaluated, TTX-MC138, is a first-in-class therapeutic designed to treat multiple metastatic cancers using antisense technology. The dose administered to the fourth cohort, as originally planned in the clinical protocol, will be approximately fifty percent higher than the dose administered in the third cohort.
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Under the clinical protocol, patients may remain on study absent safety events or disease progression. Out of 9 patients treated with TTX-MC138 in the first three cohorts, 6 remain on study for continued treatment since there have been no dose limiting toxicities or disease progression with these patients. The patient that has remained on study the longest has, to date, received 7 doses each approximately 28 days apart over the approximately 7 months that this patient has been on study. In addition to approving opening the fourth cohort, the SRC approved enrollment of additional patients in Cohort 3 to build upon the safety profile of TTX-MC138. Further, Cohort 1 and 2 data analysis for both pharmacokinetic (PK) and pharmacodynamic (PD) activity 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. Preliminary results from Cohort 2 confirmed observations from the Phase 0 trial and from Cohort 1 that the drug candidate shows evidence of activity in patient blood. Preliminary PK analysis suggests that dose levels 0.8-1.6 mg/kg, the levels administered to Cohorts 1 and 2, could represent an efficacious range.
"SRC approval to open the fourth cohort and expand enrollment in Cohort 3 is an important advancement for the clinical trial. It will provide an opportunity to obtain additional safety and PK/PD data, inform the dose expansion stage of the clinical trial and may allow us to obtain initial evidence of clinical activity" commented Sue Duggan, TransCode’s Senior Vice President of Operations. Duggan added, "Enrollment into the study continues based on the cumulative safety data review. Eligible subjects may now be screened and scheduled in Cohort 4 for treatment with the next dose level of TTX-MC138 while preliminary data analysis continues."
About TTX-MC138
TTX-MC138 is a first-in-class therapeutic candidate that targets microRNA-10b, a micro-RNA widely believed to be a driver of metastatic disease. 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.
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 selected based on preliminary results from the dose-escalation stage.
Further information is available at www.clinicaltrials.gov NCT Identifier: (NCT06260774).