TransCode Therapeutics Moves Forward with Phase 1 Clinical Trial for TTX-MC138

TransCode Therapeutics, Inc., known for its focus on RNA oncology, has made an important stride in cancer treatment research by announcing that the first patient in its fourth cohort of the Phase 1 clinical trial has begun treatment with TTX-MC138. This innovative therapy targets microRNA-10b, which plays a significant role in the development and spread of metastatic cancer.

In total, ten patients have now been treated with TTX-MC138 at progressively escalating doses, marking a critical phase in this clinical study. The company is also evaluating additional patients for possible inclusion in this expanded cohort, as part of its goal to collect more safety and efficacy data.

Importantly, TransCode has reported no significant safety concerns or dose-limiting toxicities thus far. The dosing for this fourth cohort has been increased by approximately fifty percent compared to the previous cohort, reflecting the confidence in the data gathered so far.

Throughout this trial, patients can remain on study as long as they do not experience harmful side effects or disease progression. Currently, out of ten patients treated in the initial four cohorts, seven continue to participate without any observed dose-limiting toxicities or progression of their disease. Moreover, the Safety Review Committee has also greenlit the addition of more patients to Cohort 3, allowing for a more robust understanding of TTX-MC138’s safety profile.

The analysis of pharmacokinetic (PK) and pharmacodynamic (PD) activity for the first three cohorts is ongoing, and preliminary results are promising. They indicate that the profile of TTX-MC138 aligns with earlier preclinical findings as well as data from TransCode's Phase 0 clinical trial.

Sue Duggan, Senior Vice President of Operations at TransCode, expressed enthusiasm about this milestone. She stated, "Commencement of treatment in Cohort 4 has been our vision for evaluating the potential of TTX-MC138 at multiple dose levels. It builds upon our existing safety and exploratory data and is key to our clinical strategy moving forward. We anticipate that this milestone will inform the next stages of our trial and could yield initial evidence of clinical activity as we advance."

As it stands, continued enrollment for the study is proceeding in accordance with a thorough review of the cumulative safety data. Further treatments in Cohort 4 are scheduled, as the team continues to analyze preliminary data.

About TTX-MC138

TTX-MC138 is hailed as a groundbreaking therapeutic candidate designed specifically to inhibit the microRNA-10b pathway, known to drive metastatic disease. The encouraging outcomes from TransCode's Phase 0 clinical trial, which demonstrated the drug's potential even at low doses, suggest a wide therapeutic window for TTX-MC138.

About the Trial

The Phase 1 clinical trial for TTX-MC138 is a multi-center, open-label study that focuses on dose-escalation and expansion. This design aims to gather essential safety and tolerability information for TTX-MC138 among patients suffering from various forms of metastatic solid tumors. While establishing safety is a primary goal, the trial could also provide early indications of the drug’s clinical efficacy.

The trial structure includes an initial dose-escalation phase, followed by a dose-expansion phase which will evaluate the safety, tolerability, and potential anti-tumor activity of TTX-MC138 in selected tumor types based on initial findings.

More detailed information regarding this trial can be found at ClinicalTrials.gov with identifier NCT06260774.

About TransCode Therapeutics

TransCode Therapeutics is at the forefront of innovation in the field of oncology, focusing on advancing treatments for metastatic disease. Leveraging its proprietary TTX nanoparticle platform, the company aims to drive effective RNA therapeutics, including its flagship candidate, TTX-MC138, which targets tumors known to overexpress microRNA-10b. Additionally, TransCode's portfolio features several other pioneering RNA therapeutic candidates aimed at navigating the complexities of RNA delivery to unlock new treatment potentials across different cancer types.

Forward-Looking Statements

This announcement includes forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995. These statements reflect management’s current expectations and are subject to risks and uncertainties that could lead to materially different outcomes. Factors such as the challenges of clinical trials, varying results, financial conditions, regulatory submissions, and competitive pressures are among the uncertainties that may affect the actual results. For further information on these risks, refer to the comprehensive Risk Factors section in TransCode's Annual Report for the year ended December 31, 2023, as well as future filings with the SEC.

All details in this press release are current as of its release date, and TransCode has no obligation to update them unless required by law.