Suggests potential to improve survival outcomes in patients with GBM

BOSTON, MA, USA I October 26, 2023 I TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology company committed to more effectively treating cancer using RNA therapeutics, today announced significantly improved survival in murine models bearing human glioblastoma multiforme (GBM) tumors treated with its lead therapeutic candidate, TTX-MC138. In this study, mice implanted with human GBM tumors treated with TTX-MC138 survived significantly longer than those in the control group.

GBM is the most common and aggressive primary brain tumor in adults and has the highest mortality rate among all brain malignancies. In this study reported by TransCode, one group of mice implanted with human GBM tumors was treated with TTX-MC138 as an alternative to the standard-of-care chemotherapy, temozolomide (TMZ). TMZ is the primary chemotherapy used for GBM. However, TMZ resistance is common in GBM and is a major contributor to the high rates of mortality with this disease. In the study, animals were treated weekly via systemic injection for six weeks with either TTX-MC138 or TMZ. Animals treated with TTX-MC138 survived significantly longer than the controls. Specifically, at 50 days after initiation of treatment, 75% of mice treated with TTX-MC138 were alive versus 25% of controls.

TransCode’s Chief Technology Officer, Zdravka Medarova, PhD, commented, “We believe these promising results with TTX-MC138 suggest the potential to improve survival outcomes in patients with GBM. Importantly, we believe that TTX-MC138 could be an alternative treatment in cases where resistance to standard-of-care chemotherapy has occurred.”

TTX-MC138 consists of an iron oxide nanocarrier conjugated to a nucleic acid designed to inhibit the oncogenic RNA, microRNA-10b. MiRNA-10b is described as the master regulator of cancer progression in a number of advanced solid tumors, including GBM. TransCode believes that TTX-MC138 could be used as a treatment for many of these cancers. Administration of TTX-MC138 has resulted in complete regression of metastatic disease in numerous mouse models of pancreatic and breast cancer. In addition to murine models of cancer, TTX-MC138 was successfully delivered and demonstrated preliminary efficacy in spontaneous feline mammary carcinoma.

“Given the lethal nature of GBM, adding a therapeutic such as TTX-MC138, which has a novel mechanism of action, to the armamentarium available to oncologists to fight the disease, could potentially have a high impact on the future of GBM treatment,” added Michael Dudley, Chief Executive Officer of TransCode.

The study was led by Dr. Anna Moore, Professor and Director of the Precision Health Program at Michigan State University, and a scientific co-founder of TransCode. This work will be presented at the 3d Annual Henry Ford + MSU Cancer Research Symposium, “Advancing Cancer Research Through Innovation and Collaboration,” November 9-10, 2023, East Lansing, MI.

TransCode is enrolling patients in a first-in-human clinical trial with TTX-MC138 in patients with advanced solid cancers ( In this clinical trial, up to 12 patients will be given a single microdose of radiolabeled TTX-MC138 followed by noninvasive PET-MRI. The trial is intended to quantify the amount of TTX-MC138 delivered to metastatic lesions and the pharmacokinetics of the therapeutic candidate in cancer patients. The trial could yield important data regarding TTX-MC138 delivery to clinical metastases that could inform dose selection, dosing frequency, and patient selection in later stage clinical trials. This trial is not intended to have therapeutic efficacy.

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 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. The Company believes that TTX-MC138 has the potential to dramatically improve clinical outcomes in a range of cancers, including breast, pancreatic, ovarian and colon, glioblastomas and others. 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.

SOURCE: TransCode Therapeutics