Pro-Metastatic RNA Inhibitor Gets Orphan Drug Status for Pancreatic Cancer

The Food and Drug Administration (FDA) has granted Orphan Drug designation to TTX-MC138 for the treatment of pancreatic cancer.

TTX-MC138 is designed to inhibit the pro-metastatic RNA, microRNA-10b, and eliminate metastasis. The Company believes that the investigational drug has the potential to produce regression without recurrence in a range of cancers. 

In preclinical models of pancreatic adenocarcinoma, 40% of animals treated with TTX-MC138 as monotherapy achieved complete responses, manifested as regression without recurrence. Additionally, TTX-MC138 showed positive preclinical evidence in animal models with triple-negative breast cancer.

The Company will be investigating TTX-MC138 in a first-in-human clinical trial in patients with advanced solid cancers.

“We are pleased to have received Orphan Drug Designation from FDA with our lead therapeutic candidate in pancreatic cancer,” said TransCode’s CEO and Co-founder, Michael Dudley. “This is the second drug in our pipeline to receive such status. In June 2022, FDA granted ODD status for our checkpoint inhibitor, TTX-siPDL1, also for treatment of pancreatic cancer,” added Dudley.

Reference

TransCode Therapeutics announces Orphan Drug designation status for TTX-MC138 for treatment of pancreatic cancer. News release. TransCode Therapeutics. Accessed February 28, 2023. https://www.globenewswire.com/news-release/2023/02/28/2616866/0/en/TransCode-Therapeutics-Announces-Orphan-Drug-Designation-Status-for-TTX-MC138-for-Treatment-of-Pancreatic-Cancer.html.