TransCode Therapeutics Announces Initial Dosing in Fourth Cohort of Phase 1 Clinical Trial with TTX-MC138

• First patient in Cohort four has been treated with TTX-MC138
• Ten patients have been treated with TTX-MC138 at escalating dose levels
• Additional patients being evaluated for eligibility for expanded enrollment
• No significant safety or dose limiting toxicities have been reported

BOSTON, March 27, 2025 /PRNewswire/ -- TransCode Therapeutics, Inc. (NASDAQ:RNAZ), the RNA oncology company committed to more effectively treating cancer using RNA therapeutics, today announced that the first patient in Cohort 4 of its Phase I clinical trial has received their initial dose. TransCode also reported that two additional patients in Cohort 4 are scheduled to receive TTX-MC138. The therapeutic candidate being evaluated, TTX-MC138, is TransCode's lead candidate designed to inhibit microRNA-10b, a microRNA critical to the emergence and progression of metastatic cancer.   No significant safety or dose limiting toxicities have been reported.  The dose administered to the fourth cohort, as originally planned in the clinical protocol, is 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. Of the ten patients treated with TTX-MC138 in the first four cohorts, seven remain on study for continued treatment as there have been no dose limiting toxicities or disease progression in these patients. In addition to approving opening the fourth cohort, the Safety Review Committee approved enrollment of additional patients in Cohort 3 to build upon the safety profile of TTX-MC138.  Further, data analysis of both pharmacokinetic (PK) and pharmacodynamic (PD) 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.

"Commencement of treatment in Cohort 4 has been our vision for evaluating the potential of TTX-MC138 at multiple dose levels. Cohort 4 builds upon our safety, PK/PD and exploratory data and is an important element of our clinical development strategy. We believe this milestone will inform the dose expansion stage of the clinical trial and may allow us to obtain initial evidence of clinical activity as the program continues to advance," commented Sue Duggan, TransCode's Senior Vice President of Operations. Duggan added, "Enrollment into the study continues based on the cumulative safety data review. Additional Cohort 4 patients are scheduled for treatment in Cohort 4 for treatment with 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 phase followed by a dose-expansion phase. The primary objective of the dose-escalation phase is to evaluate the safety and tolerability of escalating dose levels of TTX-MC138. In the dose-expansion phase, 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 phase.

Further information is available at www.clinicaltrials.gov  NCT Identifier: (NCT06260774).

About TransCode Therapeutics 

TransCode is a clinical-stage oncology company focused on treating metastatic disease. The company is committed to defeating cancer through the intelligent design and effective delivery of RNA therapeutics based on its proprietary TTX nanoparticle platform. The company's lead therapeutic candidate, TTX-MC138, is focused on treating metastatic tumors which overexpress microRNA-10b, a unique, well-documented biomarker of metastasis. In addition, TransCode has a portfolio of other first-in-class RNA therapeutic candidates designed to overcome the challenges of RNA delivery and thus unlock therapeutic access to a variety of novel genetic targets that could be relevant to treating a variety of cancers. 

Forward-Looking Statements 

This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, without limitation, statements concerning the timing, conduct and results of the Phase 1 clinical trial, statements about microRNAs and their involvement in cancer, and statements concerning the therapeutic potential of TransCode's TTX-MC138. Any forward-looking statements in this press release are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: the risks associated with drug discovery and development; the risk that the results of clinical trials will not be consistent with TransCode's pre-clinical studies or expectations or with results from previous clinical trials; risks associated with the conduct of clinical trials; risks associated with TransCode's financial condition and its need to obtain additional funding to support its business activities, including TransCode's ability to continue as a going concern; risks associated with the timing and outcome of TransCode's planned regulatory submissions; risks associated with obtaining, maintaining and protecting intellectual property; risks associated with TransCode's ability to enforce its patents against infringers and defend its patent portfolio against challenges from third parties; risks of competition from other companies developing products for similar uses; risks associated with TransCode's dependence on third parties; and risks associated with geopolitical events and pandemics, including the COVID-19 coronavirus and military actions. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause TransCode's actual results to differ from those contained in or implied by the forward-looking statements, see the section entitled "Risk Factors" in TransCode's Annual Report on Form 10-K for the year ended December 31, 2023, as well as discussions of potential risks, uncertainties and other important factors in any subsequent TransCode filings with the Securities and Exchange Commission. All information in this press release is as of the date of this release; TransCode undertakes no duty to update this information unless required by law. 

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SOURCE TransCode Therapeutics, Inc.