TTX-MC138

In therapeutic studies in orthotopic models of GBM presented here we used MN-anti-miR10b as well as TTX-MC138, a clinically tested anti-miR10b nanotherapeutic now in Phase I trials in patients with solid (non-GBM) cancers. Both formulations showed efficient delivery, as demonstrated by imaging and improved survival, leading to target inhibition and increased apoptosis. This approach may offer a novel strategy for delivering therapeutics to GBM and improving patient outcomes in one of the most aggressive and treatment-resistant forms of brain cancer.

P1/2, N=16, Active, not recruiting, TransCode Therapeutics | Recruiting --> Active, not recruiting | Trial primary completion date: Feb 2026 --> Sep 2025

P1/2, N=20, Recruiting, TransCode Therapeutics | Not yet recruiting --> Recruiting

P1, N=1, Completed, TransCode Therapeutics | Recruiting --> Completed | N=12 --> 1 | Trial completion date: Jan 2025 --> Sep 2023 | Initiation date: Aug 2023 --> Apr 2023 | Trial primary completion date: Jan 2025 --> Aug 2023

P1/2, N=20, Not yet recruiting, TransCode Therapeutics | Initiation date: Mar 2024 --> May 2024

P1/2, N=20, Not yet recruiting, TransCode Therapeutics

The mean whole blood half-life was 12.2 ± 2.3 h (mean ± standard deviation) and the mean plasma blood half-life was 11.5 ± 1.8 h. RadioHPLC analysis of plasma samples showed that the drug is very stable with respect to metabolism. Combined, these studies support first-in-human testing of TTX-MC138 for the treatment of metastatic cancer and, by addressing the issue of drug delivery, enable the clinical development of a wide array of TTX-based therapeutics.

P1, N=12, Recruiting, TransCode Therapeutics

Animals treated with vehicle or gemcitabine served as controls. Gene expression analysis confirmed target engagement and molecular mechanism of action. Against the background of our earlier work, these studies provide further evidence of the potential of TTX-MC138 for cancer therapy and expand its relevance beyond metastatic breast cancer to also include pancreatic adenocarcinoma.