TransCode Therapeutics CEO Letter to Shareholders - Seite 3

Our dedication to innovation and collaboration has also resulted in expanded strategic partnership conversations with esteemed academic institutions and industry leaders. By developing these collaborations, we aim to leverage their complementary expertise and resources, accelerating the development of our targeted therapies using our targeted therapeutic delivery platform.      

Our first alliance is with MD Anderson Cancer Center at the University of Texas Medical Center announced in August 2022. Through this alliance, TransCode and MD Anderson scientists are collaborating on preclinical studies to further validate TransCode’s therapeutic and diagnostic candidates and to expand the reach of TransCode’s discovery engine. The results of these studies will inform future clinical trials with these agents, including trials we intend to conduct at MD Anderson. This alliance could present an opportunity to access a wealth of knowledge and further fortify the scientific groundwork of our therapeutic programs.

In June 2022, we received Orphan Drug Designation (ODD) from the FDA for our TTX-siPDL1 checkpoint inhibitor, a candidate for treatment of pancreatic cancer. The designation was granted based on positive results achieved in in vivo studies treating human pancreatic tumors implanted in animals. In addition, we conducted preclinical in vivo studies with TTX-MC138 in a pancreatic cancer model and we received ODD status on February 27, 2023 from the FDA. We intend to conduct additional in vivo studies to support filings of other TTX-based therapeutic candidates in other orphan disease indications including osteosarcoma, glioblastoma, and small cell lung cancer. Along with the filing for ODD status in osteosarcoma, we also intend to file for Rare Pediatric Disease Designation (RPDD) with the goal of receiving a Priority Review Voucher (PRV) if the preclinical study results are positive and are accepted by FDA. A sponsor of a drug with RPDD may request a Rare Pediatric Disease Priority Review Voucher (PRV) at the time of a marketing application to FDA. In effect, the PRV has the potential to shorten the FDA review period for a future marketing application of any drug from 12 months to 6 months. When a company introduces a potential transformative therapy, the six-month acceleration in regulatory review can yield significant economic advantages. As a result, PRVs have been considered highly valued assets for companies, and we anticipate that PRVs will continue to be highly valued assets in the future due to their potential for significant economic benefits for companies.