PTC Therapeutics, Inc.

Welcome to the PTC Third 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 Third 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, 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 the 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, Matthew Klein. Matt?

Thank you all for joining the call today. I'm happy to share the results of another quarter of outstanding commercial performance and pipeline execution. In the third quarter, we achieved total revenue of $197 million, including $124 million from the DMD franchise. I want to highlight the $52 million in IMPLASA revenue, which reflects our team's ability to effectively navigate a genericized and competitive marketplace in the U.S. With our strong third quarter revenue performance, we closed the quarter with over $1 billion in cash and are raising our 2024 total revenue guidance to $750 to $800 million. In the third quarter, we submitted two NDAs to the FDA, one for Cepiatarin and one for TransLarna. Both applications have been accepted for filing. The FDA-planned action date for Cepiatarin is July 29, 2025. An action date has not been provided for the TransLarna NDA due to the regulatory context of the filing. The Cepiatarin NDA includes the significant and clinically meaningful evidence of efficacy from the Phase III Affinity Study, as well as data from the Affinity Open Label Extension Study demonstrating durability of the C. pietarin treatment benefit and the ability of patients to increase their protein intake while maintaining control of feed levels. These data support that C. pietarin can provide significant benefit to PKU patients of all age groups and severity subtypes, including classical PKU. The impactful results from the INFINITI trial were recently published in the prestigious Lancet Journal, a testament to the importance of the study findings. In addition to the FDA submission, marketing authorization applications for Cpteran are currently under review in the EU and several other countries, including Brazil. We remain on schedule to submit the JNBA in Japan in December of this year. These submissions will support our planned global launch of Cpteran in 2025. Our launch plans are progressing well, as Eric will detail shortly. We remain on track to submit an NDA for vitiquinone for the treatment of children and adults with seizure cataxia in December. This NDA submission will be the fourth approval application submitted to the FDA this year, an impressive testament to our team's execution. The vaticlinone NDA will be based on the findings of significant benefit on the upright stability subscale of the mFARS disease rating scale in the MOVE-FA placebo-controlled trial, as well as results from two long-term extension studies. As we recently reported, both long-term extension studies met their primary endpoint with evidence of statistically significant long-term benefit in delaying disease progression. Given the extensive safety data collected to date in both children and adults, the tiquinone has the potential to fill the unmet need for pediatric and adolescent FA patients, as well as provide a promising therapy for adult FA patients. Moving to our PTC518 program for Huntington's disease, the program received fast-track designation from FDA in September. reflecting the potential of PTC518 to be the first-ever approved disease-modifying therapy for HD. In addition, we requested two Type C meetings with FDA. One meeting is to discuss the potential for Huntington lowering to serve as a surrogate endpoint supportive of accelerated approval, and the second meeting is to discuss the endpoint strategy for an FFC trial in HD patients, whether that trial is completed as a Phase III registration trial or as a confirmatory trial in the context of an accelerated approval. The FDA has asked to combine the two into a single type C meeting, which has now been scheduled for later in the fourth quarter. The ongoing phase two PIVOT-HD trial continues to progress well, and we remain on track to complete the study in the first quarter of 2025, with results expected in the second quarter. Turning to our utraloxostat program for ALS, we remain on schedule to share top-line results from the registration-directed Cardinals trial in the fourth quarter. Nutriloxistat is the first compound being developed for ALS patients that specifically targets ferruptosis, 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 uterloxacetab to address the significant unmet needs of ALS patients. Finally, we remain on schedule for the November 13 FDA action date for the AADC gene therapy BLA. If approved, this would be the first ever direct-to-brain administered gene therapy authorized by FDA and open the door to a whole new approach to treating brain diseases. I am very proud of our team's ability to bring this pioneering therapy to patients. In summary, we have had a very busy and productive quarter as we continue to execute on all of our planned objectives and achieve our many significant 2024 milestones. I will now turn the call over to Eric to discuss our commercial performance. Eric.