#United States #Boston #TransCode #TTX-MC138 #microRNA-10b

TransCode Therapeutics Advances in Phase 1 Clinical Trial of TTX-MC138

TransCode Therapeutics, Inc., a pioneering RNA oncology company located in Boston, has recently received approval from the Safety Review Committee (SRC) to open a third cohort in its Phase 1 clinical trial for TTX-MC138, a promising therapeutic candidate aimed at treating metastatic cancer. Established to optimize treatment methods utilizing RNA therapeutics, the company reported encouraging findings pertaining to patient safety and treatment efficacy from earlier cohorts.

The SRC undertook a meticulous review of safety data from patients in Cohort 2 and concluded that there were no noteworthy safety concerns or dose-limiting toxicities reported. This green-light allows for the administration of nearly double the dose in the upcoming Cohort 3, which positions the trial for further exploration of TTX-MC138's potential.

Overview of the Clinical Trial

TransCode's Phase 1 trial represents a significant step in evaluating the safety and tolerability of TTX-MC138, particularly among patients suffering from a variety of solid metastatic cancers. While the primary aim is to rigorously analyze the safety and tolerability of escalating doses, early indications of clinical activity have also emerged as a potential outcome of the trial.

The trial's design includes an initial dose escalation phase which is now successfully transitioning into a dose expansion phase based on positive preliminary results. Current findings from the first cohort reveal that TTX-MC138 exhibited pharmacodynamic activity in the presence of high levels of microRNA-10b expression—this microRNA is significantly implicated in the emergence and advancement of metastatic cancers. In fact, a remarkable 66% inhibition of miR-10b was recorded just 24 hours after drug infusion.

Importance of MicroRNA-10b Targeting

Targeting microRNA-10b is fundamental in this therapeutic approach as this molecule has been recognized as a crucial player in driving metastatic processes across a variety of cancers. TTX-MC138 aims to obstruct this pathway, theoretically impeding the cancer's ability to spread, making the drug an attractive candidate in the evolving landscape of oncology treatments.

The promising pharmacokinetic (PK) profile observed in human trials further suggests favorable dosing strategies moving forward, as concentrations seen in blood plasma outperformed nonclinical study results. This information underpins TransCode’s optimism regarding TTX-MC138’s therapeutic window, which was previously indicated during preliminary animal studies and Phase 0 trials.

Next Steps and Future Implications

With the SRC’s approval, TransCode is now focusing on patient recruitment for the third cohort. Eligible patients are being screened and scheduled for the next treatment phase. The company's Senior Vice President of Operations, Sue Duggan, emphasized the importance of the SRC’s guidance in decision-making throughout this research process which safeguards patient welfare while advancing clinical objectives.

TransCode Therapeutics aims not only to contribute significantly to cancer research but also to fulfill an unmet medical need by developing RNA therapeutics designed to tackle metastatic diseases. As the trial progresses, the insights gained from these cohorts may pave the way for a transformative approach in the oncology field.

Conclusion

TransCode Therapeutics’ Phase 1 TTX-MC138 trial is making headway with promising safety data and a clear plan for advancing its investigation into the therapeutic benefits of targeting microRNA-10b. As further results unfold, this initiative could herald a new chapter in the treatment of metastatic cancers, offering hope to those affected by this challenging condition.

For more information about the clinical trials, visit ClinicalTrials.gov and search for the identifier NCT06260774.