TransCode Therapeutics Reports Positive Preclinical Results with its Lead Candidate, TTX-MC138, in Glioblastoma
Clinical Study
TransCode Therapeutics, Inc. (NASDAQ: RNAZ), a leading clinical-stage oncology company committed to using its proprietary delivery system platform to defeat cancer, today announced acceptance for publication by Frontiers in Molecular Biosciences of a preclinical study using TransCode’s lead therapeutic candidate, TTX-MC138, in glioblastoma multiforme (GBM).
The findings of this study support further development of TTX-MC138 for the treatment of GBM by identifying a potentially successful combination treatment protocol using TTX-MC138 with temozolomide (TMZ), currently the standard-of-care therapeutic. Treatment of human glioblastoma cells implanted in animals with the TTX-MC138 analogue, MN-anti-miR10b, inhibited tumor cell growth and induced cell death. When combined with a sub-therapeutic dose of TMZ in a sequence-specific protocol, MN-anti-miR10b enhanced the cell-killing ability of TMZ resulting in highly successful inhibition of tumor cell survival and invasiveness. GBM is the most common and aggressive form of brain tumors. The prognosis for patients with GBM is poor despite advances in standard-of-care therapy. The 5-year survival rate has remained unchanged over the past 30 years. There is an urgent unmet need to develop more effective therapies.
TTX-MC138 is designed to inhibit the oncogenic RNA, microRNA-10b, 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. In a preclinical model of pancreatic adenocarcinoma, administration of TTX-MC138 as monotherapy resulted in complete responses, manifested as regression without recurrence, in 40% of treated animals. This study comes on the heels of preclinical studies in breast cancer demonstrating complete regressions of metastatic disease. In addition to murine models of cancer, TTX-MC138 was successfully delivered and demonstrated preliminary efficacy in a case study of spontaneous feline mammary carcinoma.
Frontiers in Molecular Biosciences is a leading journal in its field with more than 22,000 citations and 11 million views across 3,200 articles. The study to appear in Frontiers in Molecular Biosciences was led by Dr. Anna Moore, Professor and Director of the Precision Health Program at Michigan State University, in collaboration with Dr. Zdravka Medarova, Chief Technology Officer at TransCode Therapeutics. Both Drs. Moore and Medarova are scientific co-founders of TransCode.
“We believe that demonstrating additional successful preclinical results with TTX-MC138 is an important step in the development process. Studies such as this further de-risks our pipeline that includes multiple RNA approaches including RNAi, PRR, mRNA vaccines and gene editing with CRISPR, all of which utilize our proprietary delivery platform,” added Michael Dudley, co-founder, president and CEO of TransCode.
TransCode Therapeutics recently received IRB approval for 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 dose of radiolabeled TTX-MC138 followed by noninvasive positron emission tomography-magnetic resonance imaging (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 critical data regarding therapeutic dose, timing, and potential safety that could inform later stage clinical trials and further advance TTX-MC138 as a therapeutic candidate against glioblastoma and other advanced malignancies. This trial is not intended to demonstrate any therapeutic effect.
About TransCode Therapeutics
TransCode is an RNA oncology company created on the belief that cancer can be more effectively treated using RNA therapeutics. The Company has created a platform 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. Two of the Company’s other drug candidates, TTX-siPDL1 and TTX-siLIN28B, focus on treating tumors by targeting PD-L1 and LIN28B, respectively. 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.
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