PTC Therapeutics, Inc.

Ladies and gentlemen, thank you for standing by. Welcome to PTC Therapeutics' first quarter 2026 earnings conference call. All participants on a listen-only mode. After the speaker's presentation, there will be a Q&A session. Today's conference is being recorded. I would now like to send the call over to Ellen Cavallari, Head of Investillations. Please go ahead.

Good afternoon, and thank you for joining us to discuss PTC Therapeutics' first quarter 2026 corporate update and financial results. I'm 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 quarterly report on Form 10-Q and 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.

Thank you all for joining today. We are off to a terrific start to 2026. We had a record quarter of product revenue led by the continued strong momentum of the Suffiance launch, as well as contributions from our mature products. First quarter total revenue was $273 million, including $226 million of product revenue. With this revenue performance, we are raising our 2026 full-year product revenue guidance to $750 to $850 million, with expected total revenue of $1.08 to $1.18 billion. I'll begin by providing an update on the Safiance Global launch. In the first quarter, the launch continued at a strong pace, with all signs indicating sustained growth and breadth of update. First quarter suffiance global revenue was $125 million, representing 36% quarter-over-quarter growth, with U.S. revenue of $112 million. As of March 31st, we had 1,244 commercial patients globally, and in the U.S., we surpassed the 1,500 patient start form mark in the quarter, with a consistent cadence of prescription starts averaging 140 per month over the past few months. We see this robust cadence of U.S. starts continuing for the foreseeable future. In addition to the sustained momentum in the U.S., growth is accelerating internationally through both commercial access and paid early access programs. We had our first defiance sale in Japan in late March, ahead of schedule, and remain on plan to have commercial sales in up to 30 countries by year end. I'm incredibly proud of the execution of our global teams. Within nine months, we have gained marketing authorization in the U.S., Europe, Japan, Brazil, and several other countries, and are well positioned to serve the global addressable market of over 58,000 children and adults with PKU. making suffiance a blockbuster rare disease product. We continue to see broad adoption across age groups, disease severities, and treatment histories, including treatment-naive patients and those who have not responded to existing therapies. We are also seeing rapid penetration into centers of excellence in the US, with now over 90% of centers having prescribed suffiance. Persistence remains strong, supported by high refill rates underscoring the long-term commercial opportunity. Feedback from patients, their families, and healthcare providers continues to be positive. We have seen social media reports of meaningful reductions in fenylalanine and the ability to liberalize diet and enjoy certain foods for the first time. We also continue to gather, present, and publish real-world evidence on success with diet liberalization, as well as effects on other aspects of disease including mood and cognition. I'm also pleased to report that our manuscript describing Sufiance's novel differentiated dual mechanism of action has been accepted for publication. This manuscript nicely details how the dual mechanism of action supports the ability of Sufiance to provide greater benefit to those who have a response to BH4 as well as the potential for suffiance to deliver benefit to those individuals with more severe mutations not responsive to BH4, typically associated with classical PKU. Based on suffiance's highly differentiated efficacy and safety profile, the strong start to the launch, as well as our ability to maintain momentum in the U.S. and accelerate growth globally, we remain confident in the $2 billion-plus global commercial opportunity for suffiance. Turning to the Vodafone Huntington's disease program last week, we reported positive top line results from the 24 month interim analysis of the pivot HD long term extension study. At 24 months Vodafone demonstrated dose dependent slowing of disease progression on with an average slowing of 52% relative to a match natural history cohort at the 10 milligram dose level in participants with stage 2 disease. In addition, the safety profile continues to be favorable across doses and disease stages. These data support that the significant dose-dependent HTT lowering observed in the 12-month PivotHD study are manifesting in dose-dependent clinical benefit over long-term treatment. In addition, the interim study results give us increased confidence for the success of the now-enrolling global Phase III InvestHD study funded and led by our partner, Novartis. InvestHD has a target enrollment of approximately 770 individuals with early symptomatic disease who will be randomized three to two to receive Vodafone 10 milligrams or placebo. The study also includes an interim analysis. The PTC and Novartis teams are still reviewing the data from the phase two long-term extension interim readout and will discuss potential plans to meet with regulatory authorities. For vaticanone, we had a Type C meeting with FDA in April to discuss the design of a new trial to provide additional data to support NDA resubmission. Based both on written comments and meeting discussion, we are moving forward with an open-label study with a matched natural history control group from the robust FACOMS disease registry. The study will have a target enrollment of approximately 120 individuals with Friedreich's ataxia from age 7 to 21. The primary endpoint will be the change in MFARs from baseline to 24 months. We look forward to initiating this study within the next few months and believe the design of the study, along with our learnings from previous studies, significantly increases the probability of success. Turning to our earlier stage pipeline, in the second quarter, we expect to initiate the Phase I study of PTC612, our oral NLRP3 inhibitor. While the majority of the study will be conducted in healthy volunteers, we will look to include a dosing cohort of individuals with elevated inflammatory biomarkers to enable an early assessment of PKPD. As we have discussed, PTC612 benchmarks favorably to other NLRP3 inhibitors in terms of potency. We expect to develop PTC612 for inflammatory lung disorders where there is overlap between the NLRP3 inflammasome and disease pathology. We also continue to make good progress at our other pipeline programs, including our wholly owned MSH3 oral splicing program for HD and DM1. The MSH3 program for HD could complement the HDT reduction approach of the Vodafone program, as well as be particularly suited to target the juvenile HD population. We are also making good progress on several programs from our inflammation and theroptosis platform, including our phase two ready PTC-844-DHO-DH program, baroptosis Parkinson's disease program, and NRF2 activator program. Overall, we're off to a great start in 2026. We look forward to the sustained momentum of the CIFIENZ global launch over the course of 2026 and continued favorable developments in our R&D portfolio. Our strong cash position enables us to continue to support all current programs as well as to look for accretive business development opportunities that can leverage the strength of our global rare disease commercial engine to accelerate short and intermediate-term growth. I will now turn the call over to Eric to provide a commercial update, including more details on the Sophia's launch. Eric? Thanks, Matt.