Ascendis Pharma A/S

Good day and thank you for standing by. Welcome to Ascendis Pharma Q2 Earnings and SkyTOFA approval 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, press star 0. I would now like to hand the conference over to your speaker today, Scott Smith, Senior Vice President and Chief Financial Officer at Ascendus Pharma. Please go ahead.

Thank you, Operator. Thank you, everyone, for joining our conference call today. I'm Scott Smith, Chief Financial Officer of Ascendus. Joining me on today's call is Yen Mickelson, President and Chief Executive Officer, Jesper Hoyland, Global Chief Commercial Officer, Dr. Dana Pizzuti, Head of Development Operations and Chief Medical Officer, and Dr. Yuha Poonanen, Head of Oncology. Before we begin, I would like to remind you that this conference call will contain forward-looking statements that are intended to be covered under the safe harbor provided by the Private Securities Litigation Reform Act. Examples of such statements may include, but are not limited to, our commercialization and continued development of Skytrofa, our progress on our pipeline candidates and our expectations with respect to their continued progress, statements regarding our strategic plans, our goals regarding our clinical pipeline, statements regarding the market potential of Skytropha and our pipeline product candidates, and statements regarding our regulatory filings. These statements are based on the information that is available to us today. Actual results or events could differ materially from those in the forward-looking statements. And we may not achieve our goals, carry out our plans or intentions, or meet the expectations or projections disclosed in our forward-looking statements. And you should not place undue reliance on these statements. Our forward-looking statements do not reflect the potential impact of any licensing agreements, acquisitions, mergers, dispositions, joint ventures, or investments that we may enter into or terminate. We assume no obligation to update these statements as circumstances change, except as required by law. For additional information concerning the factors that could cause actual results to differ materially, please see the forward-looking statement section in today's press release and the risk factor section of our most recent annual report on Form 20F. Skytropa was approved by the FDA today for the treatment of pediatric patients one year and older who weigh at least 11.5 kilograms and have growth failure due to inadequate secretion of endogenous growth hormone. Otherwise, please note that our product candidates are investigational product candidates and have not been approved for commercial use. As investigational products, the safety and effectiveness of the product candidates have not been reviewed or approved by any regulatory agency. None of the statements made on the conference call regarding Skytropha and our product candidates shall be viewed as promotional. On today's call, we will walk through a presentation available on our website to discuss the FDA approval of Skytropha We'll discuss our second quarter 2021 financial results and provide further business updates. Following some prepared remarks, we will then open up the call to questions. I will now turn the call over to Yann Mickelson, our President and Chief Executive Officer.

Yann Mickelson Thanks, Scott, and good afternoon. Sometimes when you wake up in the morning, you some way think about what day will bring. But I have to say, today was one of the days where you someway had an agenda, you adapt to it, like Darwinism says, you need to adapt, this is the best way of survival. And I think we are in a great, great place today. And I have to say, if I can see you, you will see a very, very happy end, that basically are so happy what we have achieved today. But never forget, At Ascentis, the key element in our core values is to make a meaningful improvement in patients' lives. We feel strongly that with our focus on science and data, we can use our innovative Transcon technologies to develop new and potential best-in-class products that address major unmet medical needs. With the approval in the U.S. of Transcon Growth Hormone, or Skytropa, for pediatric growth hormone deficiency today. We are one step near to fulfill our vision three by three to become a leading biopharma company. Before I provide a general business update related to our advancing broad pipeline of now more and more product opportunities, I would like to give a short update related to the approval of Skytrooper. As Scott said, The slides can be found on our website and I would like to refer to this slide. Going to slide number three. GuideProver, our first product developed with the Transcon technology is now approved in the US. The Transcon technology is a technology we have taken basic from the design to preclinical, to clinical, and now the approval in the U.S. At the same time, it's the first approval of the Transcon technology, but it's also the first FDA-approved one-sweet product for pediatric growth hormone deficiency. And we're really proud about it. When we go to the next slide, and I actually think you can see the label different places, You basically will see what we have seen out from the label is basically what we basically could hope for, and it's basically reflecting the science, the clinical data we have received with it. Today, because of time, I will not go through the entire labeling discussion, but I hope you will have the opportunity to look at it, because everything starts with the mode of action, where basic are describing how we releasing and somatopene, the same as daily growth hormone, in going to the right efficacy, it go to the right safety. And we feeling really, really confident with the approval and the labeling we have. We have a strong fundament to build up Skytropa Transcon growth hormone to be the leading brand. But it has not been an easy journey for the patients. If you go to slide five, I often have given you this discussion. It has been possible to develop a long-acting product that basically providing and addressing the unmet medical needs specific you have in a pediatric population about a daily injection. And despite 30 years clinical development, trial development by different kinds of concepts. Currently today in the US, in Europe, in Japan, all patients with growth hormone deficiency is treated with the same entity that actually came into the market in the 80s. There has been advancement in pen devices. There has been advancement that one single product opportunity also could make room simple to a stability. What we see with Skytrover on the approval today, this is the first once weekly opportunity that basic are providing what we call the same entity that we see in daily growth hormone. But we also have built up what we call optimal element for the patient like an auto-injector, which facilitates that you have room temperature induced stability. When I talk about all clinical programs that support the approval on SCARTOFA, it's built on basic, the HIGH trial, the FLIGHT trial, and the ENLIGHTENMENT trial. The HIGH trial, just to sum up is where we really had clinical trials with new patients. We also had a switch trial in the flight trial, and we continue in the enlightenment trial to generate long-term data, how the benefit of our product are really developing on long-term basis, both related to efficacy and safety. That is our commitment really to ensure that we're building up the most comprehensive clinical program as support of this approval. The high trial, as you recall, was a direct comparison to a daily Glutamone at the same dose. And we randomized the patient for two to one. And it's basically the main element in the approval related to efficacy. In slide 8, you can see the result for it. And we saw and better analyzed high velocity at 52 weeks, illustrated from the statistic perspective on the confidence interval. Going to the safety, and when you talk about pediatric treatment, safety is extremely important, as in all other treatment. But when you think about that you have established a safety from a daily growth hormone, it's very hard to believe that you can compromise that. And what we saw in the summary of adverse events and everything what we looked from we basically had a similar profile like Daily Growth Thermal, including hemogenic profile. Now we're coming to the next stage. We don't want to develop Transcontinental Growth Thermal to a product addressing one single geographic region. We want to develop Transcon-Globetamone up from a global commercial strategy. We have already established how we really want to address the two largest commercial markets, the U.S. We are building up our own commercial organization. We do it because we have a pipeline. We have a pipeline of rare disease endocrinology products multiple products in the same therapeutic area. We are in a position that we are ready there and in the next few slides, we basically will have a presentation from Jesper that will go in and go more in detail. But before I start, I would like to give you the perspective of the growth hormone market. We see the growth hormone market in a global setting of around more than 4 billion. The U.S. market is around 1.3 billion, where the pediatric growth hormone estimated is about 700 million. But we've also seen that there's a market that's been highly fragmented with multiple products with basically the same product. And this is where we believe the market is really primed for disruption, primed for innovation that has been lacking for 30 years. And that is what we come with. And this is why we believe we have a possibility not only to improve the treatment, but we also have a possibility to improve and expand the market segment. Jesper, will you go over and talk more dedicated related to the U.S. commercial effort?