TransCode Therapeutics, Inc. (NASDAQ: RNAZ) has released additional positive results from its Phase 1a dose-escalation trial of TTX-MC138, an investigational microRNA-10b inhibitor for advanced cancer. The study 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, supporting advancement of TTX-MC138 into Phase 2a clinical development.
Among 14 evaluable patients, nine (64%) achieved stable disease lasting six months based on RECIST criteria. TransCode also reported pharmacokinetic data demonstrating drug bioavailability consistent with preclinical findings and highlighted a metastatic thyroid cancer patient who experienced a significant decline in thyroglobulin levels and has maintained stable disease for 12 months. The company said the results support selection of a recommended Phase 2a dose of 4.8 mg/kg and provide a rationale for continued clinical development in patients with limited treatment options.
These findings are significant because they address a critical unmet need in oncology: treating metastatic tumors that overexpress microRNA-10b, a unique biomarker of metastasis. For patients with advanced cancers who have exhausted standard therapies, TTX-MC138 offers a potential new option. The durable responses observed, including one patient maintaining stability for 12 months, suggest the drug may provide meaningful clinical benefit. The safety profile, with no dose-limiting toxicities across multiple cycles, is particularly encouraging for a patient population often vulnerable to treatment side effects.
TransCode Therapeutics is a clinical stage company pioneering immuno-oncology and RNA therapeutic treatments for high risk and advanced cancers. The company’s lead therapeutic candidate, TTX-MC138, is focused on treating metastatic tumors that overexpress microRNA-10b, a unique, well-documented biomarker of metastasis. In addition, TransCode has a portfolio of other first-in-class therapeutic candidates designed to mobilize the immune system to recognize and destroy cancer cells.
The implications of these results extend beyond TransCode. If TTX-MC138 proves effective in Phase 2a, it could establish microRNA-10b as a viable therapeutic target for metastasis, potentially opening a new class of cancer treatments. For the biotech industry, this validates RNA-based therapeutics in oncology, a field that has seen mixed success. For patients with metastatic cancer, particularly those with limited options, this development offers hope for a new treatment modality that specifically targets the mechanism of spread.
TransCode plans to proceed with Phase 2a trials, likely focusing on patient populations where microRNA-10b overexpression is prevalent, such as certain thyroid, breast, and pancreatic cancers. The company's progress will be closely watched by investors and clinicians alike, given the high unmet need in metastatic disease. For more information, visit the company's newsroom at https://ibn.fm/RNAZ and the full press release at https://ibn.fm/4WzoX.
