TransCode Therapeutics Announces Positive First-in-Human Trial Results for TTX-MC138

In an announcement on May 29, 2024, TransCode Therapeutics, Inc. (NASDAQ: RNAZ) reported encouraging preliminary data from its Phase 0 clinical trial involving the radiolabeled drug candidate TTX-MC138. The trial aims to explore the anti-tumor effects of this RNA therapeutic, which is designed to target metastatic cancers.

Significant Reduction in miRNA-10b Levels

Data from the trial indicated that a single microdose of radiolabeled TTX-MC138 led to a considerable decrease in the levels of the molecular target, miRNA-10b, in the patient's blood. Post-injection, the miRNA-10b levels dropped significantly—showing a 66% reduction within 24 hours compared to pre-treatment levels. This is a crucial finding, as previous animal models demonstrated that inhibiting miRNA-10b through TTX-MC138 can result in complete regression of metastatic diseases, according to TransCode’s Chief Scientific Officer, Dr. Zdravka Medarova. The data bolster the company’s belief that TTX-MC138 could offer substantial clinical benefits for patients with metastatic cancer.

Efficacy in Targeting Metastatic Lesions

The study also documented the delivery of the drug candidate to metastatic lesions, providing evidence that TTX-MC138 accumulates within tumor tissue. The increased radioactive lesion-to-blood ratios suggest active uptake of TTX-MC138 by the cancerous cells, reinforcing its potential effectiveness.

Safety and Tolerability

The microdose of the radiolabeled drug was well tolerated, with no adverse events reported. This suggests that TTX-MC138 not only targets miRNA-10b effectively but also demonstrates pharmacodynamic activity at a minimal dose. Dr. Daniel Vlock, TransCode’s Chief Medical Officer, remarked that these results support the ongoing clinical development of TTX-MC138 for treating various metastatic cancers. The findings might indicate a broader therapeutic window than previously anticipated.

Future Directions

Full data analysis is still in progress and will be included in the final report of the study. TransCode is preparing for a Phase 1 clinical trial to further investigate TTX-MC138 in a broader patient population and to potentially validate these initial findings.

About TransCode Therapeutics

TransCode Therapeutics is a clinical-stage oncology company focused on combating metastatic disease using RNA therapeutics. Utilizing their proprietary TTX nanoparticle platform, the company aims to develop innovative RNA-based treatments. Their lead candidate, TTX-MC138, targets metastatic tumors that overexpress microRNA-10b, a biomarker linked to metastasis. Besides TTX-MC138, TransCode is working on a portfolio of RNA therapeutic candidates designed to address RNA delivery challenges and unlock new genetic targets for cancer treatment.

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