On May 1, 2025 TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology company committed to more effectively treating cancer using RNA therapeutics, reported further progress in its Phase 1a clinical trial of TTX-MC138 in patients with metastatic cancer (Press release, TransCode Therapeutics, MAY 1, 2025, View Source [SID1234652459]). TTX-MC138 is a first-in-class therapeutic candidate designed to inhibit microRNA-10b, or miR-10b, a microRNA widely believed to be critical to the emergence and progression of many metastatic cancers. To date, 13 patients have received at least one dose of TTX-MC138 at 4 separate dose levels ranging from 0.8 mg/kg to 4.8 mg/kg. Two patients have been treated in the expanded enrollment.
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Eight patients remain on study for continued treatment, receiving an additional dose of TTX-MC138 during each 28 day treatment cycle and may remain on study absent any significant safety observations or disease progression. To date, the two patients who have remained on study the longest have received seven doses of TTX-MC138 over the course of approximately seven months and have demonstrated stable disease. No significant safety or dose limiting toxicities have been reported to date in any of the trial’s 13 patients. Ongoing analyses of PK activity from Cohorts 1, 2 and 3 suggest that TTX-MC138 demonstrates a PK/PD profile consistent with preclinical results and results from TransCode’s Phase 0 clinical trial. Specifically, the preliminary PK data follow a predictable dose-response relationship. Analysis of PD activity from cycle 1 treatments in Cohorts 1 and 2, treated with a dose of 0.8 mg/kg and 1.6 mg/kg respectively, demonstrates miR-10b target engagement at 24 hours post-infusion in 5 out of the 6 patients analyzed to date.
The observed tolerability profile and the available PK/PD results thus far supports advancement of the clinical trial to further evaluate safety and potential anti-tumor activity of TTX-MC138 in the planned dose expansion (Phase 1b) portion of the trial. At the highest dose administered, TTX-MC138 was well tolerated with no significant toxicities noted.
About TTX-MC138
TTX-MC138 is a first-in-class therapeutic candidate designed to inhibit microRNA-10b, or miR-10b, a microRNA widely believed to be critical to the emergence and progression of many metastatic cancers. 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.
About the Trial
TransCode’s 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 stage followed by a dose-expansion stage. The primary objective of the dose-escalation stage is to evaluate the safety and tolerability of escalating dose levels of TTX-MC138. In the dose-expansion stage, 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).