PTC Therapeutics, Inc.

a day and thank you for standing by. Welcome to the PTC Therapeutics second quarter 2024 financial results. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you will need to press star 1 1 on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question, please press star 1 1 again. please be advised that today's conference is being recorded. I would now like to hand the conference over to your first speaker today, Ron Aubert, Senior Director of Investor Relations.

Good afternoon, and thank you for joining us today to discuss PTC Therapeutics' second quarter 2024 corporate update and financial results. I'm joined today by our Chief Executive Officer, Dr. Matthew Klein, our Chief Business Officer, Eric Powles, our Chief Strategy Officer, Kylie O'Keefe, and our Chief Financial Officer, Pierre Gravier. Today's call will include forward-looking statements based on our current expectations. Please take a moment to review the slide posted on our investor relations website in conjunction with the call, which contains information about our forward-looking statements. Our actual results could materially differ from these forward-looking statements, as such statements are subject to risks that can materially and adversely affect our business and results of operations. For a detailed description of applicable risks and uncertainties, we encourage you to review the company's most recent quarterly report on Form 10-Q, an annual report on Form 10-K filed with the Securities and Exchange Commission, as well as the company's other SEC filings. 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. With that, let me pass the call over to our CEO, Matthew Klein. Matt?

Thank you, Ron, and thank you all for joining the call. I'm pleased to share our second quarter 2024 financial results and to provide an update on the progress of our development programs. As we have discussed, our focus for 2024 is on execution. As we complete the first half of the year, I'm pleased to report that our teams have executed across all areas with solid revenue performance, on-schedule regulatory submissions, and effective management of our operating expenses. We had a very busy and productive second quarter, and I'll review some of the highlights. Starting with commercial performance, we had another great quarter across the portfolio. Second quarter revenue totaled $187 million, including DMV franchise revenue of $118 million. Based on this consistent, solid performance, we are updating 2024 total revenue guidance to $700 million to $750 million. In addition to strong revenue, we also continue to effectively manage our operating expenses and will thus maintain the 2024 OPTX guidance previously provided. In the second quarter, we also completed the sale of our gene therapy manufacturing business in Hopewell, New Jersey. As part of this transaction, PPC received an upfront cash payment of $27.5 million. In addition to the cash consideration, This transaction reduces expenses associated with operation of the facility, including employee costs. I'll now discuss the great progress our teams made across our development portfolio. As we shared last week, we submitted the NDA for CPI-Taren for the treatment of PKU to the FDA. This is an important step towards our planned global launch of CPI-Taren in 2025 as we bring a potential new standard of care to PKU patients around the globe. The CPS-herin NDA includes significant and clinically meaningful evidence of efficacy from the Phase III affinity study, as well as data from the open-label extension study demonstrating durability of the CPS-herin treatment benefit, as well as feed tolerance. Recent data from the feed tolerance sub-study indicate that approximately 60% of subjects are able to increase their daily protein intake beyond the recommended daily allowance while still maintaining control of seed levels. These data support that sepia-carin can provide significant benefit to PKU patients of all age groups and severity subtypes, including classical PKU. We plan to submit marketing authorization applications for sepia-carin in Japan and Brazil, as well as several additional countries in 2024 to support the global launch. Tylee will provide more details on our ongoing launch preparation. In July of planned, we also resubmitted the NDA for translarms. As discussed with the FDA, the submission is based on the results of study 41, in which significant benefit was recorded across key endpoints in the IPT population, as well as on the long-term evidence of meaningful treatment benefit from the strivers. In the second quarter, we also shared the positive results from the 12-month interim data readout of the PIVOT-HD study of PTC518 in Huntington's disease patients. The 12-month data demonstrated durable dose-dependent lowering of mutant HCT protein in blood cells, reaching 42% at the 10-milligram dose level, along with dose-dependent lowering of mutant Huntington's protein in the CSS at levels similar to the blood in stage 2 patients. In addition, at 12 months, there was dose-dependent favorable clinical effect on key disease measurements including the total motor score and a CUHDR scan. Importantly, the interim results also demonstrated PTC518 to be safe and well-tolerated. This combination of biomarker and clinical effect, along with favorable safety, positioned PTC518 as one of the most promising, if not the most promising therapy in development for Huntington's disease. We are in the process of preparing a meeting request to discuss with FDA the potential for accelerated approval based on the PIVOT-HC study results. In addition, we have begun work on the design of the Phase III efficacy trial of PTC518, which could serve as a confirmatory study in the context of an accelerated approval or as a registration trial. We look forward to continuing to advance PTC518 to the over 130,000 symptomatic Huntington's disease patients worldwide who desperately need a safe and effective disease-modifying therapy. As we move into the second half of 2024, we remain on schedule for our planned regulatory and clinical milestones. We are in the process of preparing the NDA submission for vaticinone for the treatment of Friedreich's ataxia, which we expect to submit by the end of the year. This submission will be based on the findings of significant treatment benefit in pediatric and young adult patients on the upright stability subscale of the MFARS disease rating scale from the MOVE-FA study, along with confirmatory evidence from the long-term open-label portion of the trial. As we have previously discussed, the upright stability scale is the most sensitive and relevant portion of the MFARS for pediatric and young adult ambulatory patients and has been shown to be predictive of risk of long-term loss of ambulation. Importantly, the tiquinone also has a strong safety profile, including in children under age 16, for whom there are no approved FA therapies. Finally, we remain on schedule to share top-line results from the registration-directed Cardinals trial of eutralaxastat in ALS patients in the fourth quarter. Eutroloquistat is the first compound being developed for ALS that specifically targets theroptosis, a pathway of oxidative stress and cell death demonstrated to be highly relevant to ALS pathology. Given the recent changes in the ALS therapeutic landscape, positive results from the cardinal study could enable Eutroloquistat to address the significant unmet needs of ALS patients. In closing, I am proud of our team's continued execution. We accomplished all of our objectives and remain on schedule to achieve the many milestones we have set for the remainder of 2022. I will now turn the call over to Eric and Kylie to discuss our commercial performance. Eric?