TransCode Therapeutics Reports Positive Data from First-in-Human Clinical Study Using Novel Lead Therapeutic Candidate, TTX-MC138
TransCode Therapeutics (NASDAQ: RNAZ) announced positive preliminary data from its Phase 0 clinical trial for TTX-MC138, their lead RNA-based therapeutic candidate. The trial showed significant inhibition of the molecular target miRNA-10b, with a 66% reduction in the patient's blood 24 hours post-administration. This inhibition is linked to complete regressions of metastatic disease in animal models. The study also revealed effective delivery of TTX-MC138 to metastatic tumors and no adverse events were observed, indicating the treatment's safety at a microdose. These findings support further clinical development, with a Phase 1 study planned to explore TTX-MC138's potential for treating multiple metastatic cancers.
- Significant 66% reduction in miRNA-10b levels 24 hours post-dosing.
- Evidence of TTX-MC138 accumulation in metastatic tumors.
- No adverse events observed, indicating good tolerance at microdose.
- Potential for broader therapeutic window than previously expected.
- Preliminary data; full data analysis is still ongoing.
- Results are based on a single patient's data.
The preliminary data from TransCode Therapeutics' Phase 0 clinical trial showcasing TTX-MC138's ability to significantly inhibit miRNA-10b in the blood is noteworthy. miRNA-10b is known to play a role in cancer metastasis, so its inhibition could potentially reduce or even eliminate metastatic tumors. This aligns with preclinical animal model studies where miRNA-10b inhibition led to complete regression of metastatic disease. The fact that the radiolabeled TTX-MC138 accumulates in metastatic lesions and is well tolerated with no adverse effects is a positive step forward. However, it's important to note that these results are preliminary and from a single patient. Comprehensive data from larger trials will be important to confirm these findings and understand the broader implications for patients with metastatic cancer.
TransCode Therapeutics' announcement is significant as it provides early indications of TTX-MC138's pharmacodynamic activity and its potential therapeutic window. The 66% reduction in miRNA-10b at 24 hours post-dosing is a strong indicator of the drug's immediate impact. Moreover, the accumulation of the drug in metastatic lesions suggests effective targeting and uptake by cancerous tissues, which could enhance treatment specificity and efficacy. From an investment perspective, these findings could bolster confidence in TransCode's pipeline and provide momentum for further clinical trials. However, investors should remain cautious until more comprehensive and peer-reviewed data is available, as early-stage trials often carry high variability and uncertainty.
The positive preliminary results from TransCode Therapeutics' Phase 0 trial are likely to have a beneficial impact on the company's stock, at least in the short term. Positive clinical trial results often drive investor sentiment and can lead to increased stock prices. The reported absence of adverse effects and the demonstrated anti-tumor activity could attract investor interest, particularly in the biotechnology sector, which is highly driven by innovation and breakthrough therapies. However, the ultimate financial impact will depend on the results of subsequent trials and the company's ability to successfully navigate regulatory approvals and commercialization challenges. Long-term investors should consider the inherent risks associated with early-stage biopharmaceutical developments.
BOSTON, May 29, 2024 (GLOBE NEWSWIRE) -- TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology company committed to more effectively treating cancer using RNA therapeutics, today announced new preliminary data from its Phase 0 clinical trial with radiolabeled TTX-MC138 suggesting anti-tumor activity.
New results from the patient dosed in this trial indicate that a microdose of radiolabeled TTX-MC138 resulted in significant inhibition of the drug candidate’s molecular target, miRNA-10b, in the patient’s blood. Specifically, after injection, the amount of miR-10b in the patient’s blood was significantly reduced compared to levels prior to administration of radiolabeled TTX-MC138, reaching a reduction of
In addition, the study also quantified the amount of drug candidate delivered to metastatic lesions, providing further evidence that TTX-MC138 accumulated in metastatic tumors. The increase of radioactive lesion-to-blood ratios suggests that circulating TTX-MC138 is actively taken up by the cancerous tissue.
The microdose of radiolabeled TTX-MC138 was well tolerated with no adverse events observed.
“These new data suggest that TTX-MC138 not only inhibits the miRNA-10b target but is pharmacodynamically active at a single microdose in the patient’s serum, supporting continued clinical development of TTX-MC138 for the treatment of multiple metastatic cancers in the planned Phase 1 clinical study. This could indicate a much broader therapeutic window than had previously been expected,” said Dr. Daniel Vlock, TransCode’s Chief Medical Officer.
Full data analysis is ongoing and will be included in the final study report.
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 initial results from the Phase 0 clinical study with our lead therapeutic candidate, TTX-MC138, in metastatic cancer, statements concerning expected preclinical and clinical results of TransCode’s other therapeutic candidates, statements concerning the potential for treating cancer with RNA therapeutics, statements concerning the timing and outcome of expected regulatory filings and clinical studies and statements concerning TransCode’s portfolio of drug candidates and TTX technology platform generally. Of note, a Phase 0 clinical study is an exploratory study, conducted under an exploratory Investigational New Drug application. Exploratory IND studies usually involve very limited human exposure to a therapeutic candidate to evaluate mechanism of action in order to inform potential clinical evaluation in future clinical studies, but otherwise have no therapeutic intent. 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 studies will not be consistent with our pre-clinical studies or expectations or with preceding clinical studies; risks associated with the timing and outcome of TransCode’s planned regulatory submissions; risks associated with TransCode’s conduct of clinical studies; 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
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