TransCode Therapeutics Announces Publication of Study with Lead Therapeutic Candidate Revealing Mechanisms Behind Candidate’s Preclinical Efficacy Against Metastatic Cancer

Rhea-AI Summary

TransCode Therapeutics (NASDAQ: RNAZ) has published a study in Oncotarget demonstrating the efficacy of its lead therapeutic candidate, TTX-MC138, against metastatic cancer. The research, conducted in collaboration with Michigan State University, reveals that TTX-MC138 reduces tumor cell capacity for self-renewal by targeting miR-10b, a molecule expressed in stem-like breast cancer cells.

Key findings include:

• Stem-like breast cancer cells increase expression of miR-10b
• TTX-MC138 treatment reduces cancer cell stemness
• Cancer cell stemness is important for metastasis and tumor formation

This study provides insights into TTX-MC138's mechanisms, potentially aiding in predicting clinical responses, defining molecular biomarkers, and improving patient stratification for future clinical trials.

Positive

• Publication of study demonstrating efficacy of lead candidate TTX-MC138 against metastatic cancer
• TTX-MC138 shown to reduce tumor cell capacity for self-renewal
• Potential for improved patient stratification in future clinical trials
• Ongoing Phase 1 clinical trial with TTX-MC138

Negative

• None.

Medical Research Analyst

This study reveals important insights into the mechanism of action of TransCode's lead candidate, TTX-MC138, in treating metastatic breast cancer. The research demonstrates that TTX-MC138 targets miR-10b, which is overexpressed in stem-like breast cancer cells. By reducing cancer cell "stemness" or self-renewal capacity, TTX-MC138 may inhibit metastasis formation, a critical factor in cancer progression.

The findings have significant implications for TransCode's ongoing Phase 1 clinical trial. Understanding this mechanism could lead to:

• Better patient selection based on tumor molecular profiles
• Development of predictive biomarkers for treatment efficacy
• Improved stratification of patients in future clinical trials

While these results are promising, it's important to note that preclinical efficacy doesn't always translate to clinical success. Investors should closely monitor the progress of the Phase 1 trial for further validation of TTX-MC138's potential in humans.

Oncology Doctor

The study's findings on TTX-MC138's ability to target cancer stem cells are particularly intriguing. Cancer stem cells are notoriously resistant to conventional therapies and play a important role in metastasis and recurrence. By demonstrating that TTX-MC138 can reduce stemness in breast cancer cells, this research suggests a potential breakthrough in treating metastatic disease.

The concept of using miR-10b inhibition to target cancer stemness is innovative and addresses a significant unmet need in oncology. If successful in clinical trials, this approach could represent a paradigm shift in metastatic cancer treatment. However, it's important to temper enthusiasm with caution. Many promising preclinical results fail to translate into clinical benefit. The ongoing Phase 1 trial will be critical in determining whether TTX-MC138 can maintain its efficacy and safety profile in human patients.

Financial Analyst

This publication represents a significant milestone for TransCode Therapeutics, potentially increasing investor confidence in their lead candidate, TTX-MC138. The study provides a strong scientific rationale for the ongoing Phase 1 clinical trial, which could positively impact the company's valuation if successful.

However, investors should consider that TransCode is still in the early stages of clinical development. The company reported $10.1 million in cash and cash equivalents as of June 30, 2023, which may not be sufficient to fund operations through the completion of the Phase 1 trial. Additional financing may be necessary, potentially leading to dilution for current shareholders.

While the scientific progress is encouraging, the path to market for novel cancer therapeutics is long and risky. Investors should closely monitor upcoming clinical milestones and the company's cash position. The potential market for an effective metastatic breast cancer treatment is substantial, but realizing this potential will require successful navigation of the clinical and regulatory landscape.

Study demonstrates that TransCode’s lead therapeutic candidate, TTX-MC138, reduces tumor cell capacity for self-renewal

BOSTON, Sept. 10, 2024 (GLOBE NEWSWIRE) -- TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology company committed to more effectively treating cancer using RNA therapeutics, in collaboration with Michigan State University, published an article in the journal Oncotarget titled, Inhibition of miR-10b treats metastatic breast cancer by targeting stem cell-like properties. The article was published on August 26, 2024. The study was led by Dr. Anna Moore, Professor, Director of the Precision Health Program, and Associate Dean of the School of Medicine at Michigan State University and scientific co-founder of TransCode.

In this study, the authors show that stem-like breast cancer cells increase expression of miR-10b, the molecule targeted by TTX-MC138. The study also demonstrates that treatment of breast cancer cells with TTX-MC138 reduces their stemness, confirming that these properties make metastatic cells susceptible to the therapeutic candidate’s actions.

Cancer cell stemness, or capacity for self-renewal, is a critical component of metastasis, since specialized cancer stem cells are those cells uniquely capable of creating new tumors and seeding metastatic dissemination. Stemness is a property of a distinct population of cancer cells that possess developmental plasticity allowing them to self-renew and adapt to new microenvironments found at the metastatic organ where they lead to creation of metastatic tumors.

“These new findings improve our understanding of the mechanisms behind TTX-MC138’s role in metastatic cancer,” commented Dr. Zdravka Medarova, Chief Scientific Officer of TransCode and an author of the publication. “This knowledge is essential as we conduct our ongoing Phase 1 clinical trial with this candidate because it may help better predict clinical response in individual patients based on the unique molecular fingerprint of their cancer. In addition, this new information may help us define potential molecular biomarkers of therapeutic efficacy that can serve as early surrogate indicators of clinical success. Finally, this knowledge may help us better stratify patients for enrollment in our later-stage clinical trials based on these predictive biomarkers,” added Dr. Medarova.

About TransCode Therapeutics

TransCode Therapeutics 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 therapeutic potential of TransCode’s TTX-MC138, statements concerning the conduct of and results from any of the company’s clinical trials, and statements concerning the results of pre-clinical research. 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 risk associated with drug discovery and development; the risk that the results of our clinical trials will not be consistent with our pre-clinical studies or expectations or with previous clinical trials; risks associated with the timing and outcome of TransCode’s planned regulatory submissions; risks associated with TransCode’s conduct of clinical trials; 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 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 TransCode’s dependence on third parties; and risks associated with geopolitical events and pandemics, including the COVID-19 coronavirus. 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 the 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 is the main finding of TransCode Therapeutics' recent study on TTX-MC138?

The study found that TransCode's lead therapeutic candidate, TTX-MC138, reduces tumor cell capacity for self-renewal by targeting miR-10b, a molecule expressed in stem-like breast cancer cells.

How might the findings of this study impact TransCode Therapeutics' (RNAZ) ongoing clinical trials?

The findings may help predict clinical responses in individual patients, define molecular biomarkers of therapeutic efficacy, and improve patient stratification for future clinical trials of TTX-MC138.

What is the significance of cancer cell stemness in metastatic cancer?

Cancer cell stemness, or capacity for self-renewal, is critical for metastasis as it allows specialized cancer stem cells to create new tumors and seed metastatic dissemination in new microenvironments.

Where was TransCode Therapeutics' (RNAZ) study on TTX-MC138 published?

The study was published in the journal Oncotarget on August 26, 2024, in collaboration with Michigan State University.