PTC Therapeutics, Inc.

Ladies and gentlemen, thank you for standing by. Welcome to PTC Therapeutics first quarter 2025 earnings conference call. All participants are in the 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 Cavalieri, head of investor relations. Please go ahead.

Good afternoon, and thank you for joining us to discuss PTC Therapeutics' first quarter 2025 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 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 today. Following the year of outstanding execution across every part of the company, we're off to a great start in 2025. We achieved $190 million of revenue in the first quarter, made great progress on our preparations for the anticipated global launch of Suffiance, and continued to work with regulatory authorities on our several pending approval applications. In addition, we closed the quarter with over $2 billion in cash, providing us the necessary resources to support all key commercial and R&D efforts as we continue to move towards becoming cash flow break-even. Let me begin with our revenue performance. We closed the quarter with $190 million in total product and royalty revenue with continued strong contributions from the DMD franchise. With this revenue performance, we are narrowing our 2025 full-year revenue guidance to $650 to $800 million. We expect a further narrow guidance pending regulatory actions and additional clarity on MFLASA performance for the remainder of 2025. We recently announced the positive CHMP opinion on the marketing authorization of Sufiance with an expected broad label, including the full spectrum of PKU patients of all ages. As we await European Commission adoption of the opinion, which is expected in June, we are preparing for our European launch. In terms of launch sequence, we are prioritizing Germany and other countries where we can achieve early access through named patient programs. Eric will provide more details on the suffiance launch plan in Europe, as well as in the U.S., ahead of the anticipated FDA decision. In the first quarter, we shared updated data from the ongoing suffiance long-term studies that continue to support the ability of suffiance to address all key patient market segments and provide patients the ability to liberalize their diet. The most recent analysis of affinity long-term extension data demonstrates that 97% of participants in the feed tolerance study were able to increase their dietary feed intake, with two-thirds of patients reaching or exceeding the recommended daily allowance of protein intake for an individual without PKU. Notably, these effects were also observed in classical PKU patients. We also shared results of the genetic variant analysis of the phase three affinity trial, which demonstrated meaningful treatment effect in classical PKU subjects with non-BH4 responsive genotypes, providing further evidence that Cefiance can provide benefit to all disease subtypes. Discussions with the FDA on the Cefiance NDA are progressing well. We are far along in labeling discussions, and I want to emphasize that we have seen no impact of recent FDA changes on the Suffiance NDA review. As we have discussed, we believe we can achieve over $1 billion in revenue from Suffiance, a significant revenue opportunity that will provide the foundation for PTC's future growth. Similar to the Suffiance NDA review, we have not seen any impact of recent FDA changes on our other approval applications. FDA review of the vitiquinone NDA for the treatment of children and adults with Friedreich's ataxia is progressing at the typical cadence for an application under priority review. The FDA is in the process of conducting inspections, and we have been told that FDA does not plan to hold an ad-com meeting. For the Translarna NDA, we have been receiving information requests, and clinical site inspections have already been conducted. Turning to the PTC518 Huntington's disease program, Yesterday, we announced positive top-line results for the PIVOT-HT Phase 2 study. The study met its primary endpoints of blood HCT lowering and safety, and the results on the full study population are consistent with the previously reported evidence of dose-dependent HCT lowering, favorable safety profile, and early signals of dose-dependent clinical effect at 12 months in Stage 2 patients. In addition, after 24 months of treatment, There were continued trends of dose-dependent favorable clinical effect relative to a propensity-matched natural history cohort, as well as dose-dependent NFL lowering, supporting that over a longer treatment period, we are seeing effects of HTT lowering on multiple aspects of disease. We plan to complete additional analyses and look forward to discussing next development and regulatory steps, including the potential for accelerated approval. Finally, I want to highlight our strong cash position. We closed Q1 with over $2 billion on our balance sheet. As we have discussed, this cash position enables us to support all planned commercial and R&D activities, participate in strategic business development activities, and reach cash flow breakeven without the need to access additional capital. The timing of cash flow breakeven will be determined by the ramp of PKU commercial sales, as well as the outcome of FDA approval applications for Vitiquinone and Translarna. In addition, this cash position provides us with insulation from global macro uncertainties. In summary, PTC is off to a strong start in 2025. I look forward to our team's continued execution as we build PTC for successful 2025 and beyond. I will now turn the call over to Eric to discuss our commercial performance. Eric? Thanks, Matt.