PTC Therapeutics, Inc.

Today's conference is scheduled to begin shortly. Please continue to standby. Thank you for your patience. Thank you. Thank you. Thank you. Thank you. Thank you. Good day, and thank you for standing by. Welcome to the PTC Fourth Quarter and Full Year 2021 Financial Results Conference Call. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question-and-answer session. To ask a question during the session, you'll need to press star, then 1 on your telephone keypad. Please be advised today's conference may be recorded. If you require operator assistance during the call, please press star, then 0. I'd now like to hand the conference over to your host today, Kylie O'Keefe, Senior Vice President of Global Commercial and Corporate Strategy. Please go ahead.

Good afternoon, and thank you for joining us today to discuss the PTC Therapeutics fourth quarter and full year 2021 corporate update and financial results. I am joined today by our Chief Executive Officer, Stuart Peltz, our Chief Operating Officer, Matthew Klein, our Chief Business Officer, Eric Powers, and our Chief Financial Officer, Emily Hill. Today's call will include forward-looking statements based on current expectations. Please take a moment to review the slide posted on our investor website in conjunction with the call, which contains our forward-looking statements. Our actual results could materially differ from these forward-looking statements, as such statements are subject to risks that can materially and adversely affect our business and results of operations. For a detailed description of applicable risks and uncertainties, we encourage you to review the company's most recent annual report on Form 10-K, filed with the Securities and Exchange Commission, as well as the company's other SEC filings. We will disclose certain non-GAAP information during this call. Information regarding our use of GAAP to non-GAAP financial measures and a reconciliation of GAAP to non-GAAP is available in today's earnings release. With that, let me pass the call over to our CEO, Stuart Peltz. Stuart?

Thanks, Kylie. I'm excited to share with you today the significant progress in 2021. Over the last few decades, we have advanced innovative therapies from ideas and discovery to commercializing and distributing them to patients around the globe. These efforts have positioned us to continue to grow to an enduring, innovative biopharmaceutical company with substantial revenues. Our vision is to build our pipeline, such that at steady state, we can commercialize a new product every two to three years. This will allow us to continue to create value for all of our stakeholders. Executing on our strategy over the last few years has led us to having a robust pipeline with five registration-directed studies ongoing, and we anticipate results for many of them this year. Along with the growth of our pipeline, I'm pleased to report that the commercial engine has performed exceedingly well. We have reached $539 million in total revenue in 2021. The Duchenne muscular dystrophy franchise continued to grow, contributing $423 million in net product revenue. These products demonstrate robust growth years after approval, and we expect this growth to continue based on the treatment benefit, maintaining treatment compliance, and continuing with geographic expansion. Our goal is to bring TransLiner to nonsense mutation to shed muscular dystrophy boys globally, and we seek continued geographic opportunity in regions like Asia Pacific, Central and Eastern Europe, Middle East and North Africa, and Latin America. In addition, with the potential of successful results, of study 041 mid-year, we look forward to pursuing the registration path to bring Translarna to patients in the United States. We anticipate that the commercial team will execute flawlessly and will have a stellar year. Based on this, we're confident that we'll continue to have a strong and growing revenue base. And our revenue guidance for 2022 is between $700 and $750 million. with $475 to $495 million from the Duchenne muscular dystrophy franchise. In addition to the DMD franchise, Avrizny has shown remarkable growth in 2021, just over a year after launch. Avrizny is now approved in 72 countries, capturing over 20% of the SMA market in the United States and is now the number one prescribed disease-modifying drug therapy in SMA. We also see continued growth in ex-United States markets with approximately 30% market share in Germany. This rapid and sustained update demonstrates the appeal of an effective, orally bioavailable therapy. Tecseti and Ray Libra are now well positioned for successful commercial launches in 2022. Both therapies received Category 1 pricing in Brazil, and this Category 1 designation allows pricing in line with international markets. Eric will go into more detail on this later in the call. In addition to PTC's commercial success, we're excited about the progress across our robust pipeline. By the end of 2022, we expect to have initiated three additional registration-directed studies, and a potential approval in Europe for PTC-AAGC, our first gene therapy. As alluded to previously, we have a number of results expected in 2022, and we believe this will be a transformative year for PTC. Let me share a few highlights. From our BioE platform, results from our registration-directed MITEI-E trial are expected in the fourth quarter for vertiquinone and mitochondrial disease-associated seizures. From our metabolic platform, the registration-directed PTC923 affinity trial for PKU is expected to be completed by the end of this year. As a reminder, there is substantial alumni medical need remaining in PKU, and with newborn screening, well-defined centers of excellence, and a clear path to registration, We're extremely excited about this program. We're moving forward with the next compound from our splicing platform, PTC518, for the treatment of Huntington's disease. As a reminder, the Phase I Healthy Volunteer Study demonstrated that PTC518 reduced HTT, mRNA, and protein levels to the target level of 50% reduction. PTC518 was also measured in the CSF. demonstrated that it crosses the blood brain barrier and has no efflux. The phase two study of the PTC518 in Huntington's disease patients is initiating this quarter. We know that the Huntington's disease community is eager for progress. We made significant achievements in 2021, despite the ongoing COVID-19 pandemic. I'm proud of the continued hard work of our people who have shown true dedication to our goal of bringing therapies to patients. PTC has consistently delivered results across research, development, and commercial business, and we're well-positioned to continue to deliver both short and long-term value to our stakeholders. I'll now turn it over to Matt for more on our development program. Matt?