PTC Therapeutics, Inc.

Ladies and gentlemen, thank you for standing by. Welcome to PTC Therapeutics' third quarter 2025 earnings conference call. All participants are in listen-only mode. After the presentation, there will be a Q&A session. Today's call is being recorded. I would now like to turn the call over to Ellen Cavallari, Head of Investor Relations. Please go ahead.

Good afternoon, and thank you for joining us to discuss PTC Therapeutics' third quarter 2025 corporate update and financial results. I'm joined today by our Chief Executive Officer, Dr. Matthew Klein, our Chief Business Officer, Eric Powell, 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, our most recent quarterly report on Form 10Q, and annual report on Form 10K 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.

Thank you all for joining the call today. I'm excited to share our outstanding third quarter results. The highlight of the quarter was the initiation of the Suffiance Global launch in Europe and the United States. As we have discussed, we expect Suffiance to be the foundational product for PTC's sustainable growth and near-term path to profitability. Overall, third quarter revenue totaled $211 million, which includes the first revenue from the Suffiance launch, as well as continued contributions from our DMD franchise. With this strong quarter performance, we are narrowing our 2025 full-year revenue guidance to $750 to $800 million. the upper end of the initial revenue guidance range. The Suffiance launch is off to a great start. As of September 30, Suffiance generated $19.6 million in revenue. This includes $14.4 million in the U.S. and $5.2 million ex-U.S. We have seen a great deal of enthusiasm upon launch, with 521 patient start forms received from U.S. centers as of September 30. we have received SART forms for patients of all ages, including adults, all disease severities, including classical PKU, and various treatment histories, including patient switches, previous treatment failures, and treatment-naive patients. While these are still early days, the fact that we are seeing demand from all key patient segments underscores the significant unmet need for PKU patients and the potential for suffiance to become the standard of care providing a safe and effective therapy for both classical and non-classical PKU patients. While we are presenting results today through September 30, the results for the month of October show sustained momentum. We are encouraged by the strong initial numbers and broad interest from the physician and patient communities. To sustain broad uptake, our teams are continuing to gather data to help reinforce the highly differentiated suffiance profiles. At the International Metabolism Meeting in September, we presented the results from the Amplify head-to-head study comparing phenylalanine lowering between suffiance and BH4. In this crossover study, suffiance treatment resulted in an average 70% greater reduction in phenylalanine levels compared to BH4, demonstrating once again the robust and clinically differentiated benefits suffiance can provide. Additional presentations at the conference highlighted suffiance benefits on cognitive function and diet liberalization, including in the most severe patients with classical PKU or BH4 non-responsive mutations. We are also finalizing a publication on the suffiance mechanism of action, including in vitro, in vivo, and clinical efficacy data in the most severe PKU mutations, supporting the potential ability of suffiance to provide a safe well-tolerated and efficacious therapy for the full spectrum of PKU patients. Eric will provide additional details on the launch. I will now briefly provide an update on the development and regulatory status of our other programs. Starting with the Vodafone Huntington's Disease Program, a meeting with FDA is planned for the fourth quarter to align on the study design for the next efficacy study, as well as to discuss the data package that could potentially support and accelerated approval application. For the ptiquinone-fregix ataxia program, we are planning to meet with FDA this quarter to discuss potential next steps for the program. And for Translarna, the NDA remains under FDA review. Finally, we remain in a very strong financial position, ending the third quarter with approximately $1.68 billion in cash. As we have discussed, this enables us to reach cash flow breakeven as well as participate in strategic business development activities to complement our R&D and commercial portfolios. Additionally, as the company continues to build for future success, we will be hosting an R&D day on Tuesday, December 2nd to share progress on our research programs, including those from our splicing platform. I will now turn the call over to Eric to discuss details of the Safiance launch and our commercial performance.