PTC Therapeutics, Inc.

Ladies and gentlemen, thank you for standing by. Welcome to PTC Therapeutics' fourth quarter and full year 2025 earnings conference call. All participants are in a 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 2025 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 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. 2025 was a year of many significant successes for PTC, positioning us well for 2026 and beyond. I'll start by reviewing the highlights in 2025 and then share our key objectives for 2026. The main highlight of 2025 was the initial global approvals and launch of Suffiance for children and adults living with PKU. As we have discussed, Suffiance will be the foundational product for PTC's near-term growth. In 2025, we gained approval for Suffiance in the U.S., EU, Japan, and other countries, all within several months. The global launch is off to a strong start with broad uptake across all key patient segments and age groups. In 2025, we also made a number of advances in our R&D pipeline, including achieving positive results from the Phase II PivotHD study of Vodoplan and progressing a number of early-stage programs, including those from our RNA splicing platform. And in 2025, we delivered another year of strong revenue performance. Fourth quarter net product and royalty revenue was $263 million, and full year 2025 total net product and royalty revenue was $831 million, exceeding guidance of $750 to $800 million. Total revenue includes $587 million of net product revenue with solid contributions from our mature products and from the early Suffiance launch. For Suffiance in the fourth quarter, we achieved $92 million in revenue, and for 2025 since launch, we achieved $111 million, a really impressive start. In addition to this strong revenue performance, non-GAAP R&D and SG&A OpEx for 2025 was $728 million, coming in below our guidance of $730 to $760 million. With our strong commercial execution, disciplined OpEx management, as well as the monetization of the remainder of the RISD royalty for $240 million in December 2025, we ended the year with $1.95 billion in cash. This financial strength will enable us to continue to support our commercial and R&D portfolios, as well as engage in strategic business development. For 2026, we are providing product revenue guidance of $700 to $800 million, with the majority coming from suffiants. This guidance represents 19 to 36% year-over-year product revenue growth. The 2026 revenue guidance excludes the VRISD royalty revenue, given the sale of the remaining portion of the VRISD royalty. We are providing expense guidance of 680 to 720 million. And based on our revenue and expense guidance, we have the potential to reach cash flow breakeven in 2026. a significant milestone for the company. As we move into 2026, our main focus will be continuing the strong momentum of the Safiance launch. To date, we have seen broad uptake across all disease severities and age groups. This year, we expect revenue growth through increased penetration in our current markets, as well as expansion into additional geographies. By the end of 2026, we expect to have patients on commercial drug in 20 to 30 countries across multiple regions, including Japan, where we gained approval in December and expect to launch in the coming weeks. Based on Sufiance's highly differentiated profile, the large unmet need for adults and children with PKU, and the early broad uptake into all patient segments, we believe Sufiance has a potential multi-billion dollar global revenue opportunity. In 2026, we also look forward to progress across our R&D portfolio. For the Vodoplam Huntington's Disease Program, an end-to-phase two meeting was held with FDA in the fourth quarter of 2025, where alignment was reached on the phase three study design. Novartis will be initiating the phase three trial, InvestHD, in the first half of this year. This trial could serve as the confirmatory study in the context of accelerated approval or as a registration trial. We also expect to have results from the Phase II Pivot-HD Extension Study in the first half of 2026, once all participants cross the 24-month time point. For vitiquinone, we had a Type C meeting with FDA last December, where we discussed potential next steps in the development program following the CRL for the vitiquinone NDA. FDA has indicated that an additional study will be necessary to support NDA resubmission and stated in the meeting minutes that this study could be a single-arm study with a natural history comparator group. We plan to meet with FDA in the second quarter to align on the protocol for this open-label study, including a matching strategy for the natural history control arm. In 2026, we expect to advance several programs from our innovative R&D platforms RNA slicing, and ferreptosis and inflammation. We expect to elect a development candidate for MSH3 program for HD and myotonic dystrophy, as well as to advance some of our earlier preclinical programs. From our inflammation platform, we look forward to initiating the phase one study for our NLRP3 program by mid-year and to potentially elect a development candidate for our ferreptosis Parkinson's disease program, as well as our NRF2 activator program. In summary, With our robust commercial engine, innovative R&D programs, and strong financial position, we look forward to a successful 2026. I'll now turn the call over to Eric to discuss our commercial performance, including details on the Suppliance launch. Eric?