TransCode Therapeutics Announces Safety Review Committee Approval of Second Cohort Opening in Phase 1 TTX-MC138 Clinical Trial Following Favorable Review of Cohort 1 Safety Data

Rhea-AI Summary

TransCode Therapeutics (NASDAQ: RNAZ) announced that the Safety Review Committee (SRC) has unanimously approved opening the second cohort in its Phase 1 clinical trial of TTX-MC138, following a favorable safety review of Cohort 1 data. TTX-MC138, designed to inhibit microRNA-10b important in metastatic cancer progression, showed no significant safety or dose-limiting toxicities in the first cohort. All patients from Cohort 1 remain on study, and the second cohort will receive approximately double the initial dose. TransCode is notably the only company with an ongoing US clinical trial targeting microRNA in cancer, utilizing technology recognized by the 2024 Nobel Prize in Physiology and Medicine.

Positive

• No significant safety or dose-limiting toxicities reported in Cohort 1
• Unanimous SRC approval for proceeding to Cohort 2
• All patients from Cohort 1 continuing treatment
• Only company with ongoing US clinical trial targeting microRNA in cancer

Negative

• None.

Insights

Medical Research Analyst

The successful completion of Cohort 1 in the Phase 1 trial for TTX-MC138 with no significant safety concerns represents a important milestone for TransCode Therapeutics. The approval to proceed with Cohort 2 at double the dose level is particularly noteworthy given that this is the first US clinical trial targeting microRNA in cancer treatment.

The trial's progress gains additional significance considering the 2024 Nobel Prize recognition of microRNA technology. The absence of dose-limiting toxicities in Cohort 1 suggests a favorable safety profile for TTX-MC138, which targets microRNA-10b, a critical factor in metastatic cancer progression. This successful safety review opens the path for dose escalation studies, a critical step in determining optimal therapeutic dosing.

• The clinical trial Safety Review Committee (SRC) approved opening of Cohort 2 after review of safety data from the three patients comprising Cohort 1
• No significant safety or dose limiting toxicities have been reported in Cohort 1
• Patients are currently being evaluated for eligibility in Cohort 2
• TTX-MC138 is a therapeutic candidate based on microRNA
• microRNA technology was recognized with the 2024 Nobel Prize in Physiology and Medicine
• TransCode is the only company with an ongoing US clinical trial targeting microRNA in cancer

BOSTON, Oct. 23, 2024 (GLOBE NEWSWIRE) -- 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 1 clinical trial has unanimously approved opening of the second cohort of patients based on its favorable review of Cohort 1 safety data. 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. All patients in the first cohort remain on study for continued treatment.  No significant safety or dose limiting toxicities have been reported. The dose administered to the second cohort will be approximately double the dose administered to the first cohort.

“An SRC is a group of clinicians and experts that oversee the safety of subjects during the conduct of the trial. The SRC makes decisions on whether and how the study should proceed, including dose escalation and de-escalation decisions per the study design. The recommendations of the SRC are used to decide whether a clinical trial should be continued as designed, changed, or terminated,” commented Sue Duggan, TransCode’s Senior Vice President of Operations. Duggan added, “No dose-limiting toxicities were reported in the first cohort of patients treated with TTX-MC138. Eligible subjects may now be scheduled in Cohort 2 for treatment with the next dose level of TTX-MC138.”  

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 is developing 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. 

 For more information, please contact: 

TransCode Therapeutics, Inc. 
Tania Montgomery-Hammon, VP of Business Development 
tania.montgomery@transcodetherapeutics.com 

FAQ

What are the safety results of TransCode's (RNAZ) TTX-MC138 Phase 1 trial Cohort 1?

The Safety Review Committee reported no significant safety or dose-limiting toxicities in Cohort 1 of the TTX-MC138 Phase 1 trial.

What is the dosing plan for Cohort 2 in TransCode's (RNAZ) TTX-MC138 trial?

Cohort 2 will receive approximately double the dose that was administered to Cohort 1 patients.

How many patients completed Cohort 1 in TransCode's (RNAZ) Phase 1 trial?

Three patients comprised Cohort 1 in the Phase 1 trial of TTX-MC138.

What is unique about TransCode's (RNAZ) clinical trial in the microRNA space?

TransCode is the only company with an ongoing US clinical trial targeting microRNA in cancer treatment.