PTC Therapeutics, Inc.

Ladies and gentlemen, thank you for standing by and welcome to the PTC first quarter 2022 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 1 on your telephone. If you require any further assistance, please press star 0. I would now like to turn the call over to your host, Kylie O'Keefe. You may begin.

Good afternoon, and thank you for joining us today to discuss the PTC Therapeutics First Quarter 2022 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 relations 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 risk that can materially and adversely affect our business and results of operation. For a detailed description of applicable risks and uncertainties, we encourage you to review the company's most recent annual reports. on Form 10-K and quarterly report on Form 10-Q 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. Good afternoon and thanks for joining today. I'm excited to share PTC's first quarter results, the first update in what I expect to be a transformative year for the company. Our mission at PTC is to develop therapeutics to help treat patients with rare disorders while producing revenue to provide value for all of our stakeholders. When I founded the company, it was built on the groundbreaking science of RNA biology. By regulating at the RNA level, we realized we could treat diseases of higher medical needs. The company has worked hard to turn these ideas into reality. Over the past 24 years, we have grown into an enduring biopharmaceutical company with a number of commercial products. We are continuing to build a robust pipeline of potential new therapeutics that at steady stage will deliver a new product every two to three years. This will allow us to continue to build into a company with a substantial revenues that bring growing value to all of our stakeholders. Let me start with our commercial portfolio. I'm proud to report that the net product revenue for the first quarter was $130 million, which represents 42% growth over the first quarter of 2021. Our Duchenne muscular dystrophy franchise Net product revenue was $128 million, demonstrating another strong quarter for Translarna and Inplaza. In addition, we recently had the first group purchase order for Tecseti in Brazil, which will be recognized in the second quarter. Eric will go into more detail on our substantial commercial progress later in the call. Evristi sales continue to show strong growth in all regions with substantial growth in Europe. Avrizzi continues to be the most prescribed disease-modifying therapy for SMA, with more than 20% market share in the United States and more than 30% market share in Germany. It is currently approved in 79 countries, and we're excited about the continued rapid update and sustained growth of Avrizzi, which demonstrates the demand across all SMA patients for an effective orally administered therapeutic. We have several exciting near-term value drivers that I'd like now to provide some updates on. For our first gene therapy for AAGC deficiency, we recently announced that we have completed the scientific advisory group and oral explanation meetings with the Committee for Advanced Therapies, or CAT. With the successful completion of these meetings, We now expect a CHMP opinion in May. If approved, PTCAADC would be the first marketed gene therapy administered directly into the brain. We're very proud to have gotten to this point in the European regulatory process and will now focus efforts on submission of the BLA. Turning now to development programs, where we have five ongoing registration-directed trials. The first of these is study 041, the placebo-controlled trial of TransLyna. We expect to report results of study 041 by the end of the second quarter. We also recently received a positive CHMP opinion for the eighth annual renewal for TransLyna in the EU. Turning now to PTC 923 for PKU and the registration-directed affinity study, We're excited by the opportunity in PKU. With a well-established patient population and a high medical need driven by the majority of PKU patients, either therapy naive or poorly controlled on existing therapies. In addition, the affinity study has an enriched population, a biomarker endpoint, and a defined path to registration. Results are expected from this study by the end of the year. For the BioE platform, we expect results from the registration-directed MiDE study of vaticinone in patients with mitochondrial disease-associated seizures in the fourth quarter of this year. We are also excited to announce that we initiated the Cardinal ALS study in PTC 857 in ALS. Moving to our validated splicing platform, we're excited to be following the successful pathway established by Evrizdi with PTC518 in Huntington's disease, or HD. HD is a debilitating disease with no disease-modifying treatments. For PTC518, an oral glycine modifier, we initiated the Phase II Pivot HD study in patients with Huntington's disease in the first quarter of this year, and we look forward to data from the first 12 weeks by the end of the year. From our ecology portfolio, we also recently initiated the Sunrise LMS study of Unespolin and Leomyosarcoma. We are excited to make progress with this platform and will provide additional updates in the next quarter. I'm proud that while PTC has demonstrated success with RNA science, we have worked to grow and diversify the business to increase the strength of the pipeline for continued success that will produce multiple therapies over the next decade. I'll now turn the call over to Matt for more detail on clinical development.