TransCode Therapeutics Advances Cancer Treatment with Successful Phase 1a Clinical Trial Dosing

TransCode Therapeutics Achieves Milestone in Clinical Trial

TransCode Therapeutics, Inc. (NASDAQ: RNAZ), an innovative RNA oncology company, has reported significant progress in its Phase 1a clinical trial for TTX-MC138, a novel RNA therapeutic designed to combat cancer. The company announced on May 8, 2025, that it has completed initial dosing for patients in Cohort 4 of the trial, with no dose-limiting toxicities reported thus far.

Clinical Trials Overview

The Phase 1a clinical trial is highly structured, comprising a dose-escalation and dose-expansion methodology. The primary purpose of this trial is to evaluate the safety, tolerability, and potential efficacy of TTX-MC138 in patients diagnosed with various metastatic solid cancers. As of this reporting, 15 patients across four escalating dose levels have been treated, ranging from 0.8 mg/kg to 4.8 mg/kg. The Safety Review Committee's assessment permitted the opening of Cohort 4, based on encouraging safety and pharmacokinetic data from previous cohorts.

Patient Progress and Safety Monitoring

Of the 15 patients treated, ten continue to receive doses of TTX-MC138 every 28 days. Remarkably, these patients have shown no signs of disease progression, and two long-term participants have undergone seven doses over approximately seven months with stable disease outcomes. This data is instrumental in highlighting the drug's potential efficacy and safety.

The ongoing analysis of pharmacokinetics (PK) and pharmacodynamics (PD) indicates that TTX-MC138 has a favorable PK/PD profile, consistent with preclinical findings. Notably, PD data from initial treatment cycles reflects significant engagement with the anticipated miR-10b target within 24 hours post-infusion, reinforcing the therapeutic's scientific premise.

A New Hope in Cancer Therapy

TTX-MC138 is designed to inhibit microRNA-10b, a crucial factor implicated in the development and progression of various metastatic cancers. The findings from TransCode's previous Phase 0 clinical trial indicated promising delivery mechanisms and pharmacological activity, affirming the drug's therapeutic potential even at microdose levels. This progress underlines TransCode’s commitment to developing effective interventions for metastatic disease.

Future Directions for TransCode

As the trial progresses into its anticipated Phase 1b, plans are in place for additional dose expansions to gather further insights into the safety and anti-tumor capabilities of TTX-MC138. The company aims to explore these effects within specific tumor types, thus paving the way for a tailored therapeutic approach.

About TransCode Therapeutics

TransCode Therapeutics continues to forge ahead in the battle against cancer, employing its proprietary TTX nanoparticle platform to enable smarter delivery of RNA therapies. Their flagship candidate, TTX-MC138, specifically targets tumors that overexpress microRNA-10b, a known biomarker of metastasis. The company’s unique portfolio also includes other first-in-class RNA candidates that promise to overcome existing obstacles in RNA delivery.

Overall, the completion of the initial dosing represents a notable achievement for TransCode Therapeutics, signaling potential advancements in cancer treatment methodologies. As they continue this groundbreaking work, the hope is that TTX-MC138 will contribute significantly to the future landscape of oncology care.

For further updates, visit clinicaltrials.gov and search for NCT Identifier NCT06260774.