PTC Therapeutics, Inc.

Ladies and gentlemen, thank you for standing by, and welcome to the PTC second quarter 2021 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 the question during this session, you will need to press star then 1 on your telephone. Please be advised that today's conference is being recorded. If you require any further assistance, please press star then 0. I will now like to hand the conference over to your speaker for today, Kyle E. O'Keefe, Senior Vice President of Global Commercial and Corporate Strategy. You may begin.

Good afternoon, and thank you for joining us today to discuss the PTC Therapeutics Second 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, 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 risks 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 10Q and annual report on Form 10K, 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 thanks for joining us today. As we close out the first half of 2021, I'm proud to say that a lot of progress has been made in all facets of our business. And I'll go into this in more detail shortly. However, before I do, I'd like to take a moment to reflect on our strategic plan at PTC and measure how we're doing. At PTC, our mission is to provide innovative treatments to patients with debilitating diseases that have few or no treatment options. The foundation of our strategy is to have a sustained pipeline so that we may continue to produce commercial treatments. that will drive revenue and create value for all stakeholders over both the short term and long term. We all know the importance of having a deep pipeline to balance the innate challenges of the drug discovery and development process. We're all working towards building a steady state number of therapeutic programs so that the successful programs move forward. This approach will allow us to have the same pipeline of potential new therapies that reach commercialization and substantially grow our revenue base. Though many of us have been fortunate to have access to COVID vaccines, we recognize that the pandemic is still very much a reality in many parts of the world. This brings additional challenges with potential impact across multiple aspects of our business. However, we'll continue to manage through these challenges as they arise. I want to emphasize the impressive revenue growth that we have seen this quarter, which is driven by the strength in our DMD franchise and the incredible uptake of IRISD. Based on this impressive growth, we'll be raising our 2021 DMD franchise revenue guidance to $370 to $390 million. And Emily will go into this in more detail later in the call. So let me start with the DMV franchise. Our commercial team has been working hard to make our therapies accessible to DMV patients around the globe. As a result, TransLinda saw an impressive 36% year-over-year growth. I'm very proud that the growth continues almost seven years post-launch, both in existing geographies and with continued geographic expansion. Impraza is also continuing to deliver strong revenue, with an increase of 36% over the same period of last year. Eric will discuss the success of the commercial franchise in more detail shortly. Now let me turn to the splicing platform, starting with Avrizzi. Avrizzi continues to see a strong uptake in the U.S. with 1,800 SMA patients now on treatment. representing almost 20% market share in less than a year post-launch. Evrizdy is also now approved in 53 markets outside of the U.S., and we are starting to see an early adoption in these markets and expect this growth to continue as we conclude on additional pricing and reimbursement discussion. As anticipated, Evrizdy was approved in Japan this quarter and and we will receive a $10 million milestone payment from Roche upon the first commercial sale. I also wanted to touch on the recent positive results from the SMA study, which I find to be quite remarkable. The Rainbow Fish study using Avrizzi in pre-symptomatic infants diagnosed with SMA demonstrated that Avrizzi-treated infants achieved the same developmental milestones, as healthy children. Interim data from the jewelfish study, which is assessing of RISD in SMA patients previously treated with other therapies, including Zolgentema and Spinraza, demonstrated overall stabilization and motor function after switching to a RISD. This shows the benefit of a RISD in the broad and heterogeneous real-world SMA population. Turning to PTC518 and our Huntington's disease program, in the second quarter of this year, we were excited to release the preliminary results from our phase one healthy volunteer study. As a reminder, the results from this study demonstrated a dose-dependent lowering of Huntington mRNA even after only a single dose of PTC518. The results also demonstrated a target mRNA lowering of 40 to 50% even in the lowest multiple ascending dose cohort. We are in the process of completing the phase one trial. This includes additional protein sampling, a food effect cohort, and a CSF pharmacology cohort. We plan to release the results of these additional cohorts in the third quarter of this year, as well as sheer details on our next clinical study, which we are planning to initiate before year end. I will now turn to our BioE platform. This is an exciting and novel science platform to identify new therapies that result as a consequence of excess electrons, usually produced from the mitochondria during electron transport. This triggers oxidative stress and causes havoc within the cell that results in and exacerbates multiple disease states. Drugs that can modulate this process would be valuable therapies to treat a wide variety of diseases. We have two important ongoing trials with vatiquinone. Our first compound from our BioE platform that is a 15-laboxygenase inhibitor, which is the key regulator of this pathway. The first is in mitochondrial epilepsy, and the second is in Friedreich ataxia. These two trials are registration-directed and therefore our potential near-term value drivers. We also have a second-generation 15-lipoxygenase inhibitor, PTC-857, with pharmacokinetic properties well-suited for a range of adult neurodegenerative diseases. We are pleased to have completed the Phase I Healthy Volunteer Study for PTC-857, and Matt will share the results later in the call. Turning now to our PKU program, which is another important near-term value driver. We're excited as we plan to initiate the Phase III Registration-Directed Study Affinity in September of this year. PKU is a large orphan indication within an estimated 58,000 patients globally. The vast majority of patients are not well-controlled with existing therapies, highlighting the substantial unmet medical needs. Study startup activities are well underway, and we are utilizing our global infrastructure to focus on sites both in the US and globally. PKU is a unique development and commercial opportunity in the rare disease world, as it has a well-defined patient population, well-known centers of excellence, and an expedited path to commercialization. We look forward to the potential of bringing PTC923 as a clinically differentiated therapy to the PKU community. Moving to our gene therapy platform and our AADC program, as a reminder, the CHMP imposed a clock stop to allow for the pre-approval inspection. This process is still ongoing, and we expect the CHMP opinion in the fourth quarter. Turning to the U.S., as we have previously shared, We were conducting additional surgeries in advance of the BLA submission. We're happy to announce that the third surgery has recently been completed and will now align with the FDA prior to the BLA submission, which we plan to submit by the end of this year. I want to take this moment to discuss our gene therapy manufacturing facility in Hopewell, New Jersey. We have a 220,000 square foot fully functional, well-equipped, and validated facility for the development and manufacture of the gene therapy products in our pipeline, thereby minimizing our reliance on external CROs. As we have excess capacity and retain the relevant manufacturing expertise, we have a unique opportunity to potentially create a revenue stream by entering into development and manufacturing service agreements with other companies. utilizing this facility and our expertise to produce high-quality plasma DNA and AAV vectors. Lastly, I wanted to highlight the potential upcoming milestones from our oncology platform. Unescolin, previously PTC-596, is in clinical trial for two niche solid tumors, DITG and LMS. DIPG is a rare pediatric brain tumor, and LMS is a rare adult solid tumor in muscle. Both have high medical needs with few beneficial to no treatment options. Results are anticipated in the second half of 2021 for the two clinical trials, and with positive results, we have the potential to initiate registration-directed trials. We look forward to sharing the results shortly. As you can see, we are continuing to make progress across our commercial and clinical efforts. I'm proud of our progress, driven by our people and their strong commitment to our mission to deliver therapy to patients in need. With that, I turn the call over to Matt for an update on development. Matt? Thanks, Stu.