PTC Therapeutics, Inc.

are in listen-only mode. After the presentation, there will be a question and answer session. Today's conference is being recorded. I would now like to turn the conference over to Ellen Cavallari, Head of Investor Relations. Please go ahead.

Good afternoon, and thank you for joining us to discuss PTC Therapeutics' fourth quarter and year-end 2024 corporate update and financial results. I am joined today by our Chief Executive Officer, Dr. Matthew Klein, our Chief Business Officer, Eric Powles, and our Chief Financial Officer, Pierre Gravier. Today's call will include forward-looking statements based on our current expectations. These statements are subject to certain risks and uncertainties, and actual results may differ materially. Please review the slide posted on our Investor Relations website in conjunction with the call, which contains information about our forward-looking statements and our most recent annual report on Form 10-K filed with the SEC, as well as our other SEC filings, for a detailed description of applicable risks and uncertainties that could cause our actual performance and results to differ materially from those expressed or implied in these forward-looking statements. Additionally, 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 are available in today's earnings release. I will now pass the call over to our CEO, Dr. Matthew Klein. Matt?

Thank you all for joining the call today. 2024 was a year of outstanding execution across every part of the company. With our many 2024 accomplishments, we are well positioned for success in 2025 and beyond. I will now review some of our 2024 highlights and discuss some of our plans for 2025. In 2024, our customer-facing teams delivered another year of strong commercial performance. Fourth quarter revenue totaled $213 million, and full-year 2024 revenue was $807 million, exceeding guidance. These results are a testament to our commercial team's ability to effectively execute around the globe. even in genericized and competitive markets. With this strong commercial performance, effective OpEx management, and the rapid monetization for $150 million of the PRV received with the capability approval, we ended 2024 with over $1.1 billion in cash. And following the closing of the Novartis PTC 518 transaction, we received an additional $1 billion in January. This strong financial position enables several important things. It allows us to support our planned 2025 commercial launches, continue to invest in our innovative R&D platforms, and engage in business development activities to complement our existing commercial and R&D portfolios. In addition, in this time of uncertainty on a macro level, Our cash position provides the potential to reach cash flow breakeven without the need to raise additional capital. In 2024, we also achieved all clinical and regulatory milestones on schedule. We submitted four approval applications to the FDA, all of which were accepted for review. Capability for AADC deficiency, which was approved in November. Cepiacarin for PKU, which has a regulatory action date of July 29, 2025, Translarna for nonsense mutation DMD, and Vatiquinone for Friedreich's ataxia, which was accepted with priority review and has a regulatory action date of August 19, 2025. These submissions create the potential to have four commercial launches in the U.S. within 12 months. In addition, outside the U.S., We submitted a number of marketing authorization applications for CPI Taran to support the planned global launch. In December, we announced a global development and commercialization collaboration with Novartis for the PTC 518 Huntington's disease program. As part of the agreement, which closed in January, PTC received $1 billion upfront and is eligible to receive up to $1.9 billion in development and sales milestones. In addition, PTC will receive a 40% profit share in the U.S. and double-digit tiered royalties for ex-U.S. sales. Novartis will assume all development, manufacturing, and commercialization costs of PTC518 following the completion of the placebo-controlled portion of the PIVOT-HD trial. The strong economics of this deal are commensurate with the promise of PTC518 as potentially being the first disease-modifying therapy for HD. As we look forward to 2025, we anticipate several value-creating milestones, including the global launch of C. peterin, 12-month results from the PIVOT-HD study of PPC518, and a number of regulatory decisions in the U.S. and around the globe. Starting with the global launch of C. peterin, there's a great deal of excitement in the patient and physician communities for C. peterin, given the significant unmet need and the strong data package generated to date. we continue to collect data that support the potential for sepia tearing to address all key patient segments, including therapy naive patients, patients not well served by existing therapies, and patients who have failed existing therapies. Eric will provide more details on our global launch planning shortly. Also in 2025, we look forward to the potential launch of the tiquinone in the United States. If approved, the tiquinone would be the first therapy for pediatric phrygic ataxia patients and could provide an effective and well-tolerated treatment option for adults with FA. Turning to our PTC518 Huntington's Disease Program, in the second quarter, we plan to share 12-month results from all patients in the PIVOT-HD trial, approximately 140 in total, which includes Stage 2 and Stage 3 patients. These results will include safety and tolerability data, biomarker data, including Huntington protein levels, as well as data on clinical scales, including the total motor score and CUHDRS. Based on our discussions with FDA in December, the results of this study could support Huntington lowering as a surrogate endpoint for accelerated approval of PTC518. In summary, with our strong performance in 2024 and demonstrated ability to effectively execute across every part of the business, we look forward to an exciting and successful 2025. I'll now turn the call over to Eric to discuss our commercial performance. Eric? Thanks, Matt.