Tuesday, February 28, 2023
TransCode Therapeutics announced that it has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for its lead therapeutic candidate, TTX-MC138, in pancreatic cancer.
TTX-MC138 has the potential to dramatically improve clinical outcomes in patients with pancreatic cancer. TTX-MC138 is designed to inhibit the pro-metastatic RNA, microRNA-10b, described as the master regulator of metastasis in a number of advanced solid tumours. TTX-MC138 can be used as treatment for many of these cancers. In a preclinical model of pancreatic adenocarcinoma, administration of TTX-MC138 as monotherapy resulted in complete responses, manifested as regression without recurrence, in 40% of treated animals. This study comes on the heels of studies in breast cancer demonstrating complete regressions of metastatic disease. In addition to murine models of cancer, TTX-MC138 was successfully delivered and demonstrated preliminary efficacy in a case study of spontaneous feline mammary carcinoma.
ODD status provides several potential benefits including seven years of marketing exclusivity if the designated candidate subsequently receives FDA marketing approval, tax credits for qualified R&D expenses, and an exemption from payment of the Prescription Drug User Fee Amendment (PDUFA) filing fee, a savings estimated to be more than $3 million.
