TransCode Therapeutics, Inc. reported successful completion of a preclinical study comprising four independent replicates with its lead therapeutic candidate, TTX-MC138, in pancreatic adenocarcinoma (PDAC). The study demonstrated that TTX-MC138 was effective against metastatic (stage IV) pancreatic cancer in animal models. There are currently no treatment options for patients with metastatic (stage IV) pancreatic cancer beyond palliative care.

The study involved weekly injection of TTX-MC138 into animals bearing human pancreatic tumors. Treatment was initiated after the tumors were established and continued for 8 weeks. Untreated animals and animals treated with the standard-of-care chemotherapeutic, gemcitabine, were used as controls.

The results of the study indicated that 38% of the animals treated with TTX-MC138 had evidence of metastasis at the end of the study, whereas 90% of untreated animals and 77% of animals treated with gemcitabine had metastases. The study showed that TTX-MC138 reduced metastatic burden by 50% compared to untreated animals and by 39% compared to animals treated with gemcitabine. Target engagement was demonstrated by measuring the expression of the target, miRNA-10b, in tumor tissue.

TTX-MC138, delivered via TransCode?s proprietary TTX delivery platform, demonstrated a nearly complete erasure (99.98%) of the miR-10b target in the tumors and successful engagement of multiple downstream oncogenes, many of which are currently undruggable using existing drugs but which could potentially be therapeutic targets using TTX-MC138. The company believes that these outcomes, which exceeded earlier in vitro observations, validate TTX-MC138?s targeting of miR-10b in tumors. Furthermore, the company believes that these results underscore the efficacy of its TTX platform for the systemic delivery of RNA-based therapeutics into solid tumors.