TransCode Therapeutics Completes Initial Dosing of First Cohort in Phase 1 Clinical Trial with TTX-MC138, a microRNA-Targeted Technology

Rhea-AI Summary

TransCode Therapeutics (NASDAQ: RNAZ) has completed initial dosing of the first cohort in its Phase 1a dose-escalation clinical trial for TTX-MC138, an antisense oligonucleotide designed to inhibit microRNA-10b, a driver of metastasis in multiple cancers. Key points:

• All patients in the cohort received their first dose and remain on study for continued treatment
• No significant safety or dose limiting toxicities reported
• TTX-MC138 uses TransCode's proprietary TTX delivery system
• TransCode is the only company with an ongoing US clinical trial targeting microRNA in cancer
• The trial's progress coincides with the 2024 Nobel Prize in Physiology and Medicine recognizing microRNA technology

The company views the rapid enrollment and absence of dose limiting toxicities as encouraging signs for the continued assessment of TTX-MC138's safety and tolerability.

Positive

• Successful completion of initial dosing for the first cohort in Phase 1a clinical trial
• No significant safety or dose limiting toxicities reported
• Rapid enrollment of the first cohort
• TransCode is the only company with an ongoing US clinical trial targeting microRNA in cancer
• Alignment with recent Nobel Prize recognition of microRNA technology

Negative

• None.

Insights

Medical Research Analyst

The completion of initial dosing for the first cohort in TransCode Therapeutics' Phase 1 clinical trial for TTX-MC138 is a significant milestone. This microRNA-targeted therapy aims to inhibit microRNA-10b, a key driver of metastatic cancer. The absence of significant safety issues or dose-limiting toxicities is encouraging, potentially supporting continued dosing and assessment.

The trial's progress is particularly noteworthy given the recent 2024 Nobel Prize in Physiology and Medicine recognition of microRNA technology. This validates the scientific foundation of TransCode's approach and could increase investor interest in microRNA-based therapies.

However, it's important to note that this is still an early-stage trial and the market cap of $9,338,994 suggests a small, speculative company. While the initial safety profile is promising, efficacy data and larger patient cohorts will be important for assessing the therapy's potential. Investors should monitor future cohorts and any signals of clinical benefit, as these will be critical for the company's prospects and potential partnerships or funding opportunities.

Financial Analyst

From a financial perspective, this news is positive for TransCode Therapeutics (NASDAQ: RNAZ) but should be viewed cautiously. The successful dosing of the first cohort without safety issues is a de-risking event, potentially increasing the company's value proposition. However, the company's micro-cap status ($9.3 million market cap) indicates high risk and likely financial resources.

Investors should consider:

• Cash runway and burn rate
• Potential for dilutive financing rounds
• Milestone payments or partnerships that could provide non-dilutive funding

The Nobel Prize connection may attract attention, potentially leading to increased trading volume or temporary stock price movements. Long-term value, however, will depend on clinical progress and the company's ability to advance its pipeline cost-effectively. Given the early stage and small size, RNAZ remains a highly speculative investment suited for risk-tolerant investors with a long-term horizon.

• Patients currently remain on study for continued treatment with TTX-MC138  
• No significant safety or dose limiting toxicities reported
• TTX-MC138, an antisense oligonucleotide conjugated to TransCode’s proprietary TTX delivery system, is designed to inhibit microRNA-10b, a known driver of metastasis in multiple cancers
• 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. 10, 2024 (GLOBE NEWSWIRE) -- TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology company committed to more effectively treating cancer using RNA therapeutics, today announced that it has dosed all patients in the first cohort of its Phase 1a dose-escalation clinical trial. 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 cohort received their first dose of TTX-MC138 and remain on study for continued treatment.  No significant safety or dose limiting toxicities have been reported.

“The rapid enrollment of the first cohort is extremely encouraging. The absence of dose limiting toxicities thus far supports continued dosing and assessment of TTX-MC138 for safety and tolerability,” commented Sue Duggan, TransCode’s Senior Vice President of Operations. Duggan added, “We are excited to be in the clinic with TTX-MC138 on the heels of the recent announcement that this year’s Nobel Prize in Physiology and Medicine was awarded to the discoverers of microRNA. Their work informs the understanding of microRNA’s role in gene regulation, which we believe reflects the potential of TTX-MC138.”

About microRNA

MicroRNA is a regulatory molecule naturally occurring in cells that plays crucial roles in development, physiology, and disease. The 2024 Nobel Prize in Physiology and Medicine was awarded to US scientists Dr. Victor Ambros and Dr. Gary Ruvkun for their 1993 discovery of microRNA and its function in controlling the array of proteins that a cell produces and governing how cells in the body function.

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  

The 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, any statement 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 is the purpose of TransCode Therapeutics' Phase 1 clinical trial for TTX-MC138?

The Phase 1a dose-escalation clinical trial aims to evaluate the safety, tolerability, and initial efficacy of TTX-MC138, an antisense oligonucleotide designed to inhibit microRNA-10b, which is involved in cancer metastasis.

Has TransCode Therapeutics (RNAZ) reported any safety concerns with TTX-MC138 in the initial dosing?

No, TransCode Therapeutics has reported no significant safety or dose limiting toxicities in the initial dosing of the first cohort in the Phase 1a clinical trial of TTX-MC138.

What is unique about TransCode Therapeutics' (RNAZ) clinical trial with TTX-MC138?

TransCode Therapeutics is currently the only company with an ongoing US clinical trial targeting microRNA in cancer, using their proprietary TTX delivery system for the antisense oligonucleotide TTX-MC138.

How does the 2024 Nobel Prize in Physiology and Medicine relate to TransCode Therapeutics' (RNAZ) work?

The 2024 Nobel Prize was awarded for discoveries in microRNA, which aligns with TransCode Therapeutics' focus on microRNA-targeted technology in their lead candidate TTX-MC138, potentially validating their approach in cancer treatment.