TransCode Therapeutics Takes on Glioblastoma; Shares Rise 284%

TransCode Therapeutics, Inc. (Nasdaq: RNAZ) has received positive results in preclinical studies evaluating its lead therapeutic candidate, TTX-MC138, which was delivered to human glioblastoma multiforme (GBM) tumors in mice, and effectively engaged its target.

TransCode’s first-in-human clinical trial with TTX-MC138 is open for enrollment and has dosed its first patient.

TransCode’s Chief Technology Officer, Zdravka Medarova, said, “Given the lethal nature of this disease and the paucity of therapeutic options for patients with GBM, a therapeutic candidate with a novel mechanism of action, such as TTX-MC138, could have a dramatic impact on patient outcomes.”

Highlights

In TransCode’s study, mice implanted with tumors derived from human GBM patients were treated with TTX-MC138 and imaged by magnetic resonance imaging (MRI) to determine delivery of the therapeutic candidate to the tumors.

TTX-MC138 was injected intravenously and accumulated efficiently in the tumors.

The therapeutic candidate showed lasting activity and significantly inhibited miRNA-10b, known to be a driver of tumor progression in GBM.

TransCode has previously demonstrated that treatment with TTX-MC138 resulted in complete regression of metastatic disease in numerous mouse models of pancreatic and breast cancer. In addition, TTX-MC138 was successfully delivered and demonstrated efficacy in spontaneous feline mammary carcinoma.

GBM is the most common and aggressive form of brain cancer. Its prognosis is poor and the 5-year survival rate has remained unchanged over the past 30 years.

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 cancer, 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.