Transcode Therapeutics’ TTX-MC138 shows no toxicities in Phase 1a cancer trial

TransCode Therapeutics (RNAZ) announced further progress in its Phase 1a clinical trial of TTX-MC138 in patients with metastatic cancer. 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. 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. 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 or Phase 1b portion of the trial. At the highest dose administered, TTX-MC138 was well tolerated with no significant toxicities noted.