TransCode Therapeutics, Inc. announced positive results with its lead therapeutic candidate, TTX-MC138, in murine models bearing human glioblastoma multiforme (GBM) tumors. In this study, the therapeutic candidate was delivered to brain tumors and effectively engaged its target. GBM is the most common and aggressive form of brain cancer.

Its prognosis is poor despite advances in standard-of-care therapy. The 5-year survival rate has remained essentially unchanged over the past 30 years. TransCode believes there is an urgent need to develop more effective therapies.

In the study reported by TransCode, 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. In addition, the pharmacodynamic activity of TTX-MC138 was determined by measuring inhibition of the therapeutic target, miRNA-10b, using qRT-PCR. TTX-MC138 was injected intravenously and accumulated efficiently in the tumors.

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

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, TTX-MC138 was successfully delivered and demonstrated efficacy in spontaneous feline mammary carcinoma.

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

In this clinical trial, up to 12 patients will be given a single microdose of radiolabeled TTX-MC138 followed by noninvasive PET-MRI and other testing. 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 to expedite the path to success in later stage clinical trials.

This trial is not intended to evaluate therapeutic efficacy.