PTC Therapeutics: Building a durable company to benefit patients

Hello, and welcome to Pathfinders, podcast series from RBC Capital Markets where we uncover the key trends and catalysts shaping the fast moving world of biopharma and health care. I'm your host, Joe Colletti. Today, we're at RBC's global health care conference here in New York, and I'm joined by doctor Matt Klein, CEO of PTC Therapeutics, a global biotech advancing treatments for rare diseases. Under Matt's leadership, PTC is pushing forward high impact programs in PKU, Huntington's disease, Friedreich's ataxia, and more. Matt, thanks for joining us. Joe, great to be here. Thank you very much. So I wanna talk about the fact that you guys have really been a pioneer in RNA target therapies and rare disease treatments. Can you talk a little bit about your company's mission, how it's evolved under your leadership, and what really is kinda core to that mission so our audience learns a little bit more about you? Yeah. PTC was founded over twenty five years ago. We started, as you pointed out, with an initial focus on RNA. In fact, I think we're probably one of the first companies to drug RNA and understanding the incredible potential there is in targeting RNA to affect a number of different diseases. Our initial efforts were in something called nonsense suppression, which was specifically targeting RNA where there had been a nonsense mutation to allow for the production of a normal protein, which otherwise would not have been produced. And our early focus was in Duchenne Muscular Dystrophy. In fact, we were the first ever company to develop a drug for Duchenne, and we're very proud to have pioneered the field of therapies, not only RNA therapies, but therapies for Duchenne, and we've grown. We our RNA expertise had led us to founding a platform in small molecule splicing. In fact, we were the first company to develop a small molecule splicing therapy. The first product of our splicing efforts is a drug called Avisd, which we developed along with Roche and is now the leading therapy for spinal muscular atrophy worldwide. That platform now has gone on to develop and grow. We have a therapy for Huntington disease in development, PTC five one eight, which we partnered with Novartis, and we have a number of other splicing programs at various stages of preclinical development targeting neurology, metabolism, and other therapeutic areas. And we've migrated beyond RNA to continue to be that company that looks for innovative science, new ways of tackling challenging problems so that we can bring impactful therapies to patients with rare diseases. What defines a high impact program in your pipeline right now? You mentioned a lot of different therapies there and a lot a lot of drug development. Like, how do you focus your efforts accordingly across these? We focus on the things that we know we can do that others can't, and we have a long view. I mean, we understand that it may take several efforts. We understand …