PTC Therapeutics, Inc.

Good day and thank you for standing by. Welcome to PTC's first quarter 2024 financial results conference call. 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'll 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, Jane Hanlon, Associate Director of Investor Relations. Please go ahead.

Good afternoon, and thank you for joining us today to discuss PTC Therapeutics' first quarter 2024 corporate update and financial results. I am joined today by our Chief Executive Officer, Dr. Matthew Klein, our Chief Business Officer, Eric Powell, our Chief Commercial 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 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 and 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, Dr. Matthew Klein.

Matt? Thank you, Jane. Good afternoon, and thank you all for joining the call. I'm pleased to share with you our first quarter 2024 financial results and to provide an update on the progress of our development programs. As we have discussed, 2024 will be a year of execution for PTC with a number of important and value-inflecting milestones throughout the year. We are off to a great start with strong revenue and achievement of all of the planned first quarter regulatory and clinical milestones. Starting with commercial performance, we had a great quarter driven by execution across the entire commercial portfolio. First quarter revenue totaled $210 million and DMD franchise revenue was $161 million. The first quarter DMD revenue resulted from our team's efforts to ensure that we optimize revenue during the time that Translarna remains authorized in Europe. At this time, the European Commission has not yet adopted the CHMP opinion to withdraw Translarna authorization, and thus, we will continue to commercialize Translarna across Europe. Eric and Kylie will provide additional details on our commercial performance shortly. We had a number of important regulatory achievements in the first quarter. As planned, we submitted the marketing authorization application for CPIaterin to the EMA, and we remain on schedule to submit the NDA for CPIaterin no later than the third quarter. The European MAA submission is an important step towards our planned global launch of Cpterin as we bring a potential new standard of care to PKU patients around the globe. As emphasized recently by Lisa Milberg, the Executive Director of the National PKU Alliance, the vast majority of PKU patients are not served by the currently available PKU therapies And there was a great deal of enthusiasm in the PKU community for sepiaterin based on the data generated to date, including the reported patient experience in diet liberalization. In the first quarter, we also submitted the BLA for Epsteza, and we aligned with the FDA on an NDA resubmission for Translarna and an NDA submission for vitiquinone for treatment of Friedreich ataxia. The NDA resubmission for Translarna will be based on the placebo-controlled results from Study 41 and data from the STRIDE registry, which confirm long-term benefits of Translarna in slowing time to loss of ambulation. We expect to submit the NDA in mid-2024. The vaticlinone NDA will include results from the 72-week placebo-controlled portion of the MOVE-FA trial, along with data from the trial's long-term open-label extensions. We expect to submit this NDA in late 2024. Turning to our development programs, we plan to share interim results from the PIVOT-HD study of PTC518 in Huntington's disease patients in the second quarter. This data update will include 12-month biomarker and clinical results from the initial cohort of approximately 30 subjects on whom we shared data last summer. Specifically, we will share data on blood Huntington protein lowering, CSF mutant Huntington protein lowering, volumetric MRI changes, and NFL levels, as well as data on several clinical scores, including the total motor score, total functional capacity, and the CUHDRS. These 12-month data will allow us to understand the longer-term safety and tolerability profile of PTC518, as well as to identify favorable early signals of CNS activity on disease biomarkers or clinical endpoints. The second quarter update will also include 12-week results for a larger number of Stage 2 and Stage 3 subjects, including blood Huntington protein changes. Lastly, we completed enrollment in the Cardinal Registration-Directed Trial of Eutroloxastat in ALS patients and remain on schedule to report top-line results in the fourth quarter of this year. Eutraloxistat 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 therapeutic landscape for ALS, positive results from the cardinal study could enable Eutraloxistat to address the significant unmet need of ALS patients. In closing, I am proud of our team's execution in the first quarter. We achieved all of our planned objectives and remain on schedule to achieve the many important expected milestones in 2024. I will now turn the call over to Eric and Kylie to discuss our commercial performance. Eric?