RNAZ TransCode Therapeutics Inc

TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology company committed to more effectively treating cancer using RNA therapeutics, today reported successful completion of a preclinical study comprising four independent replicates with its lead therapeutic candidate, TTX-MC138, in pancreatic adenocarcinoma (PDAC). The study demonstrated that TTX-MC138 was effective against metastatic (stage IV) pancreatic cancer in animal models. There are currently no treatment options for patients with metastatic (stage IV) pancreatic cancer beyond palliative care.

The study involved weekly injection of TTX-MC138 into animals bearing human pancreatic tumors. Treatment was initiated after the tumors were established and continued for 8 weeks. Untreated animals and animals treated with the standard-of-care chemotherapeutic, gemcitabine, were used as controls. The results of the study indicated that 38% of the animals treated with TTX-MC138 had evidence of metastasis at the end of the study, whereas 90% of untreated animals and 77% of animals treated with gemcitabine had metastases. The study showed that TTX-MC138 reduced metastatic burden by 50% compared to untreated animals and by 39% compared to animals treated with gemcitabine. Target engagement was demonstrated by measuring the expression of the target, miRNA-10b, in tumor tissue. TTX-MC138, delivered via TransCode’s proprietary TTX delivery platform, demonstrated a nearly complete erasure (99.98%) of the miR-10b target in the tumors and successful engagement of multiple downstream oncogenes, many of which are currently undruggable using existing drugs but which could potentially be therapeutic targets using TTX-MC138. The company believes that these outcomes, which exceeded earlier in vitro observations, validate TTX-MC138’s targeting of miR-10b in tumors. Furthermore, the company believes that these results underscore the efficacy of its TTX platform for the systemic delivery of RNA-based therapeutics into solid tumors.

“The survival rate of patients with pancreatic cancer is less than 10%. Exocrine PDAC has a 5-year survival rate of only 1% when diagnosed at an advanced inoperable stage, observed in 80% of PDAC cases. Despite overall research progress, the prognosis for people with this malignancy has not improved in over 40 years. A patient diagnosed with stage IV PDAC currently receives only palliative care. This preclinical data we announce today gives us confidence that TTX-MC138 has the potential to provide meaningful clinical benefit to these patients and address this significant unmet clinical need,” said Michael Dudley, co-founder and Chief Executive Officer of TransCode.

TransCode co-founder and Chief Technology Officer, Dr. Zdravka Medarova, commented, “Surgery, chemotherapy, and radiotherapy are currently used to extend survival and to relieve patients’ symptoms. However, no treatments have yet been found to be effective in the long term for patients with advanced disease. We believe that TTX-MC138, could represent an alternative that could shift the paradigm of PDAC treatment to help many patients survive long-term. Given that TTX-MC138 is already being tested in a Phase 0 clinical trial with the potential to enter Phase I clinical testing in 2024, the potential to dose patients with PDAC may be on the horizon.”

About TransCode Therapeutics

TransCode is an RNA oncology company created on the belief that cancer can be more effectively treated using RNA therapeutics. Using its proprietary iron oxide nanoparticle delivery platform, the company has created a portfolio of drug candidates designed to target a variety of tumor types with the objective of significantly improving patient outcomes. The company’s lead therapeutic candidate, TTX-MC138, is focused on treating metastatic cancer, which is believed to cause approximately 90% of all cancer deaths totaling over nine million per year worldwide. Another of the company’s drug candidates, TTX-siPDL1, focuses on treating tumors by targeting a protein called Programmed death-ligand 1 (PD-L1). TransCode also has three cancer-agnostic programs: TTX-RIGA, an RNA–based agonist of the retinoic acid-inducible gene I designed to drive an immune response in the tumor microenvironment; TTX-CRISPR, a CRISPR/Cas9–based therapy platform for the repair or elimination of cancer-causing genes inside tumor cells; and TTX-mRNA, an mRNA-based platform for the development of cancer vaccines designed to activate cytotoxic immune responses against tumor cells.

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 results of a preclinical study with its lead therapeutic candidate, TTX-MC138, in pancreatic adenocarcinoma, statements concerning expected clinical results of TransCode’s therapeutic candidates, statements concerning the results of RNA research, statements concerning the potential for treating cancer with RNA therapeutics, statements concerning the timing and outcome of expected regulatory filings and clinical trials, including the first-in-human study of TTX-MC138 currently underway and whether this study will demonstrate proof-of-mechanism, and statements concerning TransCode’s portfolio of drug candidates and TTX technology platform generally. Of note, a Phase 0 clinical trial is an exploratory study, conducted under an exploratory Investigational New Drug (eIND) 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 planned clinical trials will not be consistent with our pre-clinical studies or expectations; risks associated with the timing and outcome of TransCode’s planned regulatory submissions; risks associated with TransCode’s planned clinical trials for its product candidates; 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 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, 2022, 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.Alan Freidman, VP Investor Relationsalan.freidman@transcodetherapeutics.com