TransCode Therapeutics Completes Initial Dosing of First Cohort in Phase 1 Trial With a microRNA-Targeted Technology

TransCode Therapeutics, Inc. recently announced it has dosed all patients in the first cohort of its Phase 1a dose-escalation clinical trial. The therapeutic candidate being evaluated, TTX-MC138, is TransCode’s lead candidate designed to inhibit microRNA-10b, a microRNA critical to the emergence and progression of metastatic cancer. All patients in the cohort received their first dose of TTX-MC138 and remain on study for continued treatment.  No significant safety or dose limiting toxicities have been reported.

“The rapid enrollment of the first cohort is extremely encouraging. The absence of dose limiting toxicities thus far supports continued dosing and assessment of TTX-MC138 for safety and tolerability,” said Sue Duggan, TransCode’s Senior Vice President of Operations. Duggan added, “We are excited to be in the clinic with TTX-MC138 on the heels of the recent announcement that this year’s Nobel Prize in Physiology and Medicine was awarded to the discoverers of microRNA. Their work informs the understanding of microRNA’s role in gene regulation, which we believe reflects the potential of TTX-MC138.”

MicroRNA is a regulatory molecule naturally occurring in cells that plays crucial roles in development, physiology, and disease. The 2024 Nobel Prize in Physiology and Medicine was awarded to US scientists Dr. Victor Ambros and Dr. Gary Ruvkun for their 1993 discovery of microRNA and its function in controlling the array of proteins that a cell produces and governing how cells in the body function.

TTX-MC138 is a first-in-class therapeutic candidate that targets microRNA-10b, a micro-RNA widely believed to be a driver of metastatic disease. TransCode’s 2023 Phase 0 clinical trial produced evidence of delivery of a radiolabeled version of TTX-MC138 to metastatic lesions and pharmacodynamic activity, even at a microdose of the drug candidate, suggesting a broad therapeutic window for TTX-MC138.

The Phase 1 clinical trial is a multicenter, open-label, dose-escalation and dose-expansion study, designed to generate critical data to support evaluation of the safety and tolerability of TTX-MC138 in patients with a variety of metastatic solid cancers. While not an endpoint, the trial may provide early evidence of clinical activity of TTX-MC138. The trial comprises an initial dose-escalation phase followed by a dose-expansion phase. The primary objective of the dose-escalation phase is to evaluate the safety and tolerability of escalating dose levels of TTX-MC138. In the dose-expansion phase, the safety, tolerability and anti-tumor activity of TTX-MC138 will be further evaluated in certain tumor types selected based on preliminary results from the dose-escalation phase. Further information is available at www.clinicaltrials.gov NCT Identifier: (NCT06260774).

TransCode is a clinical-stage oncology company focused on treating metastatic disease. The company is committed to defeating cancer through the intelligent design and effective delivery of RNA therapeutics based on its proprietary TTX nanoparticle platform. The company’s lead therapeutic candidate, TTX-MC138, is focused on treating metastatic tumors which overexpress microRNA-10b, a unique, well-documented biomarker of metastasis. In addition, TransCode is developing a portfolio of other first-in-class RNA therapeutic candidates designed to overcome the challenges of RNA delivery and thus unlock therapeutic access to a variety of novel genetic targets that could be relevant to treating a variety of cancers.