TransCode reports positive phase 1 trial progress for cancer therapy

Published 06/02/2025, 14:23
TransCode reports positive phase 1 trial progress for cancer therapy

BOSTON - TransCode Therapeutics, Inc. (NASDAQ: RNAZ), a micro-cap RNA oncology company with a market capitalization of $6.22 million, has announced the progression of its Phase 1 clinical trial with the dosing of three patients in Cohort 3 with TTX-MC138, the company’s leading therapeutic candidate. According to InvestingPro data, the company maintains a current ratio of 1.58, indicating adequate short-term liquidity despite operating losses. The Safety Review Committee overseeing the trial has approved this new cohort following a review of safety and pharmacokinetic (PK) data from the first two cohorts and is currently monitoring the third cohort for preliminary data.

The trial’s nine patients, including several from the first two cohorts, have not reported significant safety issues or dose-limiting toxicities. These patients continue to receive TTX-MC138 in cycles of approximately 28 days. PK and pharmacodynamic (PD) analyses from Cohorts 1 and 2 are ongoing, with results so far indicating that TTX-MC138’s PK/PD profile aligns with preclinical and Phase 0 trial outcomes. Notably, Cohort 1 confirmed Phase 0 observations of pharmacodynamic activity in patients with high baseline expression of miR-10b, achieving a 66% inhibition at 24 hours post-infusion.

TTX-MC138, designed to inhibit microRNA-10b (miR-10b), is considered critical in the development and progression of various metastatic cancers. TransCode’s 2023 Phase 0 clinical trial indicated the drug candidate’s potential, showing delivery to metastatic lesions and pharmacodynamic activity even at microdoses.

The ongoing multicenter, open-label Phase 1 trial is structured in a dose-escalation and dose-expansion format to assess the safety, tolerability, and potential anti-tumor activity of TTX-MC138 across different metastatic solid cancers. With an EBITDA of -$14.55 million in the last twelve months, InvestingPro analysis indicates the company is quickly burning through cash, making this trial’s success crucial for future prospects. The primary goal of the dose-escalation stage is to determine the safety and tolerability of escalating doses of TTX-MC138, while the dose-expansion stage will further evaluate the safety, tolerability, and anti-tumor activity in selected tumor types based on preliminary results.

TransCode’s focus on RNA therapeutics for metastatic disease is underpinned by its proprietary TTX nanoparticle platform, with TTX-MC138 being the foremost candidate targeting metastatic tumors overexpressing miR-10b. The company also has a portfolio of RNA therapeutic candidates designed to address RNA delivery challenges and target a range of genetic factors relevant to cancer treatment.

The information provided is based on a press release statement and further details on the trial can be found under the NCT Identifier: NCT06260774 at www.clinicaltrials.gov. Despite the stock’s significant volatility and current trading price of $8.94, RNAZ has shown remarkable YTD returns of 165%. InvestingPro subscribers have access to 10+ additional investment tips and comprehensive financial metrics to better evaluate the company’s potential. Get the full picture with real-time updates and expert analysis.

In other recent news, TransCode Therapeutics Inc has made several significant advancements. H.C. Wainwright has increased its price target for the company, following the approval from the Safety Review Committee to progress to Cohort 3 in its Phase 1 study of TTX-MC138, a treatment for solid tumors. The company also announced a private placement agreement with institutional investors, raising approximately $8 million in gross proceeds for general corporate purposes and working capital.

TransCode Therapeutics also received stockholder approval on all matters during its Special Meeting of Stockholders, including ratification of its prior issuance and sale of securities in a public offering managed by ThinkEquity LLC. Another key approval was for the amendment to the company’s Amended and Restated Certificate of Incorporation to effect a reverse stock split of its common stock.

Furthermore, the Board of Directors of TransCode Therapeutics approved a 1-for-33 reverse stock split, aimed at satisfying Nasdaq’s minimum bid price requirement for continued listing. These are recent developments that are shaping the trajectory of TransCode Therapeutics.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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