TransCode Therapeutics Announces Safety Review Committee Approval to Open Fourth Cohort in Phase I/II Clinical Trial

• Clinical trial Safety Review Committee (SRC) approved opening of Cohort 4 based on favorable safety data from the three patients comprising Cohort 3
• No significant safety or dose limiting toxicities reported in Cohorts 1, 2, or 3
• Patients currently being evaluated for Cohort 4 eligibility
• PK and PD data from patients in Cohort 1 and Cohort 2 consistent with preclinical results and results from Phase 0 clinical trial

Excerpt from the Press Release:

BOSTON, March 13, 2025 /PRNewswire/ — TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology company committed to more effectively treating cancer using RNA therapeutics, today announced 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. 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.

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.

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