TransCode Therapeutics (NASDAQ: RNAZ) has reported positive results from its Phase 1a dose-escalation trial of TTX-MC138, an investigational microRNA-10b inhibitor for advanced cancer, and is advancing the program into Phase 2a clinical development. The announcement provides new hope for patients with limited treatment options, particularly those with metastatic tumors that overexpress microRNA-10b, a biomarker associated with metastasis.
The Phase 1a trial met its primary safety endpoint, with no dose-limiting toxicities observed across 16 patients who received a total of 86 doses. The company reported durable disease stabilization in multiple patients, including three participants who remain on treatment after 14, 16, and 21 cycles. Among 14 evaluable patients, nine (64%) achieved stable disease lasting six months based on RECIST criteria, a standard measure of treatment response.
TransCode also highlighted a metastatic thyroid cancer patient who experienced a significant decline in thyroglobulin levels and has maintained stable disease for 12 months. Pharmacokinetic data demonstrated drug bioavailability consistent with preclinical findings, supporting the selection of a recommended Phase 2a dose of 4.8 mg/kg.
"The results support selection of a recommended Phase 2a dose and provide a rationale for continued clinical development in patients with limited treatment options," the company stated. The advancement of TTX-MC138 into Phase 2a is significant as it addresses a critical need for therapies targeting metastatic cancer, which is responsible for the majority of cancer-related deaths.
TransCode Therapeutics is a clinical-stage company focusing on immuno-oncology and RNA therapeutic treatments for high-risk and advanced cancers. Its lead candidate, TTX-MC138, is designed to treat metastatic tumors that overexpress microRNA-10b. The company also has a portfolio of other first-in-class therapeutic candidates aimed at mobilizing the immune system to recognize and destroy cancer cells.
For more information, visit the company's newsroom at https://ibn.fm/RNAZ and the full press release at https://ibn.fm/4WzoX.
