PTC Therapeutics, Inc.

Good day, and thank you for standing by. Welcome to the PTC Therapeutics Third Quarter 2021 Corporate Update and 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 will need to press star 1 on your telephone. Please be advised that today's conference is being recorded. If you require any further assistance, please press star 0. I would now like to hand the conference over to your speaker today, Kylie O'Keefe, Head of Investor Relations. Please go ahead.

Good afternoon, and thank you for joining us today to discuss the PTC Therapeutics Third Quarter 2021 Corporate Update and Financial Results. Joining me on today's call is our Chief Executive Officer, Stuart Peltz, our Chief Development Officer, Matthew Klein, and our Chief Business Officer, Eric Powers, and our Chief Financial Officer, Emily Hill. Before we start, let me remind you that today's call will include forward-looking statements based on 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 risk that can materially and adversely affect our business and results of operation. 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 is available in today's earnings release. With that, let me pass the call over to our CEO, Stuart Peltz. Stuart?

Thanks, Kylie, and thank you all for dialing in. I'm excited to tell you about our progress this quarter as we have continued to demonstrate remarkable commercial revenue and have been making good progress across a robust pipeline. At PTC, we are committed to creating both short and long-term value for all our stakeholders. To accomplish this, we have built a company that discovers, develops, and commercializes therapies to treat patients of higher medical needs. We are building a sustainable pipeline that at steady state will develop new therapies that reach commercialization on a continuous basis. This will allow us to substantially grow our revenue base so that it will continue to increase in the years to come. The results of all these efforts are bearing fruit. We see continuous and impressive commercial revenue growth of our products, and we now have five ongoing registration-directed studies with an increasing number of preclinical and clinical studies in progress. We are building a sustainable, enduring, biopharmaceutical companies. Turning to this quarter's DMV franchise commercial revenue, we continue to deliver impressive year-over-year growth. The franchise has grown by 39% compared to the third quarter of 2020, with $114 million in total revenue. This was primarily driven by new patient starts for both Inflaza and Translarna. and geographic expansion for Translarna. I'm pleased to announce that we are raising our 2021 revenue guidance for the DMD franchise to $400 to $420 million from the $370 to $390 million. Eric and Emily will go into more detail shortly. Now, I'd like to highlight the significant milestones achieved in Brazil with both Taxeti and Ray Lever this quarter. In August, we were pleased to announce the approval of Ray Lever in Brazil. This is the first approved treatment for familial chylomicronemia syndrome, or FCS, and we're excited to be able to bring this therapy to patients in need. This was not the only milestone for Brazil this quarter. I'm also happy to report that Taxeti, which is for the treatment of HATTR amyloidosis, received Category 1 pricing. Category 1 pricing is given to innovative treatments that show benefits over current standards of care. Due to the Portuguese descent, there is a higher prevalence of HATTR patients in the Brazilian population. Eric will go into more detail on the remarkable progress of the commercial team later in this call. Now let me turn to Evrizni, which is the first commercial product from our innovative splicing platform. Evrizni continues to show strong revenue this quarter, with approximately 20% total market share in just over a year after launch. Evrizni is the most prescribed SMA treatment in the United States, and Roche expects further growth in 2021, with approval in 63 countries and its continued focus on the pricing and reimbursement efforts outside of the U.S. The success of our commercial portfolio demonstrates our ability to utilize our commercial engine to grow revenues to further invest in the pipeline, which I'll now discuss in more detail. In particular, I'd like to start with our near-term value drivers. I'm excited to announce that we have initiated the registration-directed trial called Affinity for PTC923 in PKU. We view this as a significant opportunity as there is a well-defined patient population through newborn screening, known centers of excellence, and a clear pathway to approval with a blood-based biomarker as an endpoint, as well as having an enriched population through a run-in responder analysis that increases our chance of success. Let me talk about our next two registration-directed trials from our BioE platform. The BioE platform targets excess electrons and the oxidative stress that contributes to multiple disease states. The first compound from this platform, vatiquinone, inhibits 15-lipoxygenase, a key regulator that controls the oxidative stress response. The first of the registration-directed trials with vatiquinone is the MITEE study. in patients with mitochondrial disease associated with seizures. We expect results in the third quarter of 2022, and if positive, will be the basis of our NDA. Our second registration-directed vaticanone trial, MOVE-FA, is in patients with Friedreich ataxia, a mitochondrial disease that affects neuromuscular function. We're grateful for the collaboration and support from the FA community in helping us to drive enrollment. We anticipate results in 2023. We're also moving ahead with the next generation compound from the BioE platform, PTC 857. We have selected ALS as the first indication and are aiming to initiate the phase two trial for PTC 857 in the first quarter of next year. I'll now discuss our innovative splicing platform with a focus on PTC518 for the treatment of Huntington's disease. We recently reported results from PTC518 Healthy Volunteer Trial. We were able to show dose-dependent lowering of both HTT mRNA and protein and achieve the targeted 30% to 50% reduction. We also demonstrated that PTC518 passes the blood-brain barrier and has minimal efflux. These are key attributes to treat Huntington's disease. We remain very excited about this program and are moving as rapidly as possible into phase two. Moving into the phase two trial, we will replicate safety, tolerability, and pharmacology of PTC508, as well as decreases in Huntington mRNA and protein levels in the Huntington's disease patients. Matt will go into more detail on our phase two program shortly. now moving on to AADC. In Europe, we still expect the CHMP opinion by the end of this year and look forward to bringing this transformational therapy to AADC deficiency patients. I'm also pleased that we recently held a ribbon-cutting ceremony for our gene therapy manufacturing center of excellence in Hopewell, New Jersey. Our new state-of-the-art facility is well-equipped and staffed to produce high-quality plasmid and AAV vectors for gene therapy applications. I'm very proud of the progress we've made over the past quarter with exciting commercial achievement and steady state progress across our pipeline. This speaks to the dedication of our people who have worked incredibly hard in the face of the ongoing challenges brought by the pandemic. The past 18 months have required perseverance, and I'm proud to say that we've continued to execute across all fronts. We have all the ingredients, research, development, and commercialization in place that positions us to be a growing, revenue-generating, enduring biopharmaceutical company. I'll now turn the call over to Matt for an update on the development program. Matt?