PTC Therapeutics, Inc.

Good day, and thank you for standing by. Welcome to the PTC Second Quarter 2022 Financial Results and Corporate Update 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, you will need to press star 11 on your telephone. Please be advised that today's conference is being recorded. I would now like to hand the conference over to your speaker today, Kylie O'Keefe, Senior Vice President Head of Global Commercial and Corporate Strategy. Please go ahead.

Good afternoon, and thank you for joining us today to discuss the PTC Therapeutics Second Quarter 2022 Corporate Update and Financial Results. I am joined today by our Chief Executive Officer, Stuart Peltz, our Chief Operating Officer, Matthew Klein, our Chief Business Officer, Eric Powles, and our Chief Financial Officer, Emily Hill. 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 risk that can materially and adversely affect our business and results of operation. For a detailed description of applicable risk, and uncertainties, we encourage you to review the company's most recent annual report on Form 10-K and quarterly report on Form 10-Q 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 Pelt.

Stuart? Thanks, Kylie, and good afternoon, everyone, and thanks for joining us today. I'm excited to share PTC's second quarter results in what I expect to be a transformational year for the company. Our mission at PTC is to discover and develop innovative therapies and bring them to patients with rare disorders, and in doing so, to create significant value for all of our stakeholders. We've been very successful in pursuit of this mission. Today, PTC has five marketed products, seven development programs, and several scientific platforms which to generate future products. We continue to build a robust pipeline of potential new therapies that at steady state will deliver a new product every two to three years. We made important progress in the second quarter towards achieving our ambitious goals for 2022. We continue to achieve strong revenue growth and to advance towards multiple clinical milestones. I'd like to highlight several particularly significant achievements in the quarter. Let me begin with our strong revenue growth in the quarter in which we achieved $166 million in total revenue with a 42% increase over the second quarter in 2021. This is quite remarkable and puts us in good position to achieve our full-year revenue target of between $700 and $750 million. The DMV franchise is expected to continue a strong growth trajectory throughout the remainder of 2022. We also reported top-line results of study 41, which was an important step forward for Translarna. In study 041, we demonstrated a statistically significant benefit for Translina and NASF mutation DMD patients across a number of relevant functional outcomes in the broad intent to treat study population. Based on these results, we plan to request conversion of the EU conditional authorization to a standard marketing authorization. In addition to the EU, we look forward to discussing these results and a potential path forward for approval with the FDA and other international markets. In addition, these results will reinforce our strong value proposition with payers in the EU and international markets. Matt will go into these results in more detail shortly. We recently announced the marketing authorization for ABEZA by the European Commission. This approval expands our product portfolio to five commercialized products. Upstaza is the third gene therapy ever marketed and the first marketed gene therapy directly administered into the brain. Our goal at PPC is to bring Upstaza to AADC deficient patients and their families as quickly as possible. We are well poised for commercial launch and our planned commercial price is between $3 and $3.5 million. This reflects the exceptional value of Upstaza and takes into account the ultra-orphan AADC patient population, with a small budget impact to payers from this one-time durable treatment. Pricing of Upstazer is based on multiple factors, including that the disease is highly morbid and fatal, and the high medical needs of AADC deficient patients with no standard of care. Lastly, Upstazer brings substantial value to patients with transformative clinical results and durable effects with up to 10 years of follow-up. We have already treated our first patient on commercial drugs through the Early Access Program in France, and Eric will go into this in more detail shortly. Let me switch gears to RISD as we continue to achieve strong updates across all regions. The FDA recently approved the supplemental NDA of Evrizzi for pre-symptomatic infants with SMA under two months of age. Their approval is based on interim efficacy and safety data from the Rainbow Fish Study in Newport, which showed that pre-symptomatic babies treated with Evrizzi achieved key milestones, such as sitting, standing, and walking after 12 months of treatment, similar to that seen in normal babies. Turning now to our oncology program, with Unespalin, previously known as TTC596. We recently announced encouraging preliminary safety and efficacy results from our Phase 1B study in Unespalin, our tubulin binding agent, in advanced leomyelococcoma patients at a podium presentation at ASCO. Based on these preliminary results, TTC initiated the Sunrise LMS study, a placebo-controlled registration-directed study of the efficacy and safety of unescolin and dacarbazine in patients with advanced LMS. PTC has a broad and deep pipeline across a range of diseases, and we eagerly anticipate important results with several registration-directed studies during the next six to 12 months. We believe the positive results from these trials could be transformational for PTC. PTC is clearly having an exciting year and has executed on all of what we set out to achieve in the first half of 2022, which is truly remarkable. With that, let me hand it over to Matt for a development update. Matt?