TransCode Therapeutics Announces FDA Clearance To Initiate Phase 1/2 Clinical Trial with TTX-MC138 in Patients with Advanced Solid Tumors

Rhea-AI Summary

TransCode Therapeutics receives FDA clearance to begin Phase 1/2 clinical trial with TTX-MC138 in patients with advanced solid tumors, aiming to evaluate safety and anti-tumor activity.

Positive

• FDA clearance marks a significant milestone for TransCode Therapeutics.
• Phase 1/2 clinical trial to commence in mid-2024.
• Study to focus on safety and preliminary anti-tumor activity of TTX-MC138 in patients with advanced solid tumors.
• Trial includes dose-escalation and dose-expansion phases.
• TTX-MC138 shows potential in treating various cancers based on animal model results.

Negative

• None.

Insights

Oncology Doctor

The FDA's clearance for the Phase 1/2 clinical trial of TTX-MC138 in advanced solid tumors heralds a promising advancement in the field of oncology. As an Oncology Doctor, I can attest to the urgent need for innovative therapies, especially for metastatic cases where treatment options are often limited. The multi-phase structure of the trial, incorporating dose-escalation to assess safety followed by dose-expansion to evaluate tolerability and efficacy, is a prudent approach to determine the therapeutic's potential.

Given the preliminary animal model results showing reduction in metastatic burden, there is cautious optimism for TTX-MC138. However, it is critical to note that success in animal models does not guarantee human efficacy. The outcome of this clinical trial could significantly impact treatment protocols and patient survival rates for various cancers. The potential to positively affect outcomes in cancers such as breast, pancreatic and glioblastomas, which often have poor prognoses, should be of particular interest to the medical community.

Medical Research Analyst

In examining the implications of TransCode Therapeutics' IND clearance, the perspective of a Medical Research Analyst is crucial. The transition from preclinical to Phase 1/2 studies is a pivotal moment for any therapeutic candidate, with significant investment implications. For stakeholders, the long-term potential of TTX-MC138 lies not only in its clinical success but also in the broader portfolio validation of TransCode's RNA-based therapeutics platform.

Market responses to such announcements tend to be speculative until substantial clinical data is available. Nevertheless, if the trial demonstrates a favorable safety profile and early signs of efficacy, it could lead to increased investor confidence. Analysts will be watching closely for patient response rates and duration of response as indicators of the drug's market potential. Moreover, this clinical trial will provide insights into the viability of RNA therapeutics as a class, a domain that has gathered investor interest due to the success of mRNA vaccines in recent years.

Clinical trial expected to commence in mid-2024 to evaluate the safety and preliminary anti-tumor activity of TTX-MC138

BOSTON, April 15, 2024 (GLOBE NEWSWIRE) -- TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology company committed to more effectively treating cancer using RNA therapeutics, announced today that the U.S. Food and Drug Administration (FDA) has completed its review of the company’s Investigational New Drug (IND) application and concluded that the company may proceed with its Phase 1/2 multicenter, open-label, dose-escalation and dose-expansion study of its lead therapeutic candidate, TTX-MC138, in patients with advanced solid tumors.

“We are thrilled to have obtained FDA authorization to advance TTX-MC138 into the clinic. FDA’s clearance of our IND application represents a significant milestone for our company,” commented Sue Duggan, Senior Vice President, Operations, at TransCode. “Activation of the IND opens the door for continued development of TTX-MC138 in the clinical setting. The Phase 1/2 clinical trial is designed to generate critical data to support evaluation of the safety of TTX-MC138 and may provide early evidence of its clinical activity in patients with metastatic disease.”

The Phase 1/2 clinical trial includes an initial dose-escalation phase followed by a dose-expansion phase. The dose-escalation portion of the trial is designed to include patients with a variety of metastatic solid cancers. The primary objective of this phase is to evaluate the safety and tolerability of escalating dose levels of TTX-MC138. In the dose-expansion phase, certain tumor types will be evaluated based on preliminary results from the dose-escalation phase. The primary objective of the dose-expansion phase is to further evaluate safety as well as tolerability and anti-tumor activity of TTX-MC138 in cancer patients.

The company believes that TTX-MC138 has the potential to positively affect patient outcomes in a range of cancers, including breast, pancreatic, ovarian, colon, glioblastomas and others. In multiple animal models of cancer, treatment with TTX-MC138 led to statistically significant reductions of metastatic burden and longer animal survival relative to animals given control treatments. Successful clinical development of TTX-MC138 potentially represents a novel solution for patients with metastatic cancer.

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 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 and the timing, conduct and results of the planned Phase 1/2 clinical trial. 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, Business Development
tania.montgomery@transcodetherapeutics.com

 

FAQ

When will TransCode Therapeutics commence the Phase 1/2 clinical trial with TTX-MC138?

TransCode Therapeutics is expected to begin the Phase 1/2 clinical trial in mid-2024.

What is the primary objective of the dose-escalation phase in the clinical trial?

The primary objective of the dose-escalation phase is to evaluate the safety and tolerability of escalating dose levels of TTX-MC138.

Which types of cancers will be evaluated in the dose-expansion phase of the trial?

Certain tumor types will be evaluated based on preliminary results from the dose-escalation phase in the dose-expansion phase of the trial.

What potential does TTX-MC138 have in treating cancer according to TransCode Therapeutics?

TransCode Therapeutics believes that TTX-MC138 has the potential to positively affect patient outcomes in various cancers, including breast, pancreatic, ovarian, colon, glioblastomas, and others.

What were the results of treatment with TTX-MC138 in animal models of cancer?

Treatment with TTX-MC138 in animal models of cancer led to statistically significant reductions of metastatic burden and longer animal survival compared to control treatments.