Ascendis Pharma A/S

Good day, and thank you for standing by. Welcome to the Ascendance Farmer third quarter earnings 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 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 turn the conference over to your speaker for today, Scott Smith. Chief Financial Officer, please go ahead.

Thanks so much, operator. And thank you everyone for joining our third quarter 2024 Financial Results Conference Call. I'm Scott Smith, Executive Vice President and Chief Financial Officer at Ascendus Pharma. I'm joined today by Yen Mickelson, President and Chief Executive Officer. 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 statements regarding our commercialization and continued development of Skytropha and Yorvapath for the U.S. and European markets, as well as certain financial expectations, our pipeline candidates and our expectations with respect to their continued progress and potential commercialization, our strategic plans and partnerships, our goals regarding our clinical pipeline, including the timing of clinical results in trials, our ongoing and planned regulatory filings, and our expectations regarding the timing and the results of regulatory decisions, expected market developments, and our exploration of market opportunities in the therapeutic areas of endocrinology rare disease. These statements are based on information that is available to us as of today. Actual results could differ materially from those in our forward-looking statements, and you should not place undue reliance on these statements. 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 our forward-looking statements section in today's press release and the risk factors section of our prospective supplement filed on September 20, 2024, and our most recent annual report on Form 20F filed with the SEC on February 7, 2024. Transcon Growth Hormone, or Transcon HGH, is approved in the U.S. by the FDA and the EU has received MAA authorization from the European Commission for the Treatment of Pediatric Growth Hormone Deficiency. Transcon PTH is approved in the U.S. by the FDA for the treatment of hypoparathyroidism in adults, and the European Commission and the U.K.' 's Medicines and Healthcare Products Regulatory Agency have granted marketing authorization for Transcon PTH as a replacement therapy indicated for the treatment of adults with chronic hypoparathyroidism. Otherwise, please note that our product candidates are investigational and not approved for commercial use. As investigational products, the safety and effectiveness of these product candidates have not been reviewed or approved by any regulatory agency. None of the statements during this conference call regarding our product candidates shall be viewed as promotional. On the call today, we'll discuss our third quarter 2024 financial results and we'll provide further business updates. Following some prepared remarks, we'll then open it up for questions. With that, let me turn it over to Jan.

Thank you so much, Scott. 2024 has been another transformative year for Ascentis. 3 out of 3 of our rare endocrinology programs have delivered clinically differentiated pivotal data. Each product or program is demonstrating its potential to address major unmet medical needs and achieve blockbuster status, positioning us to be the market leader in each disease area. Our first approved product, Skytrofe, is a best-in-class once-a-weekly growth hormone and has achieved a leading position in a highly competitive US market with a single indication. Skytrofe's performance is a testament to its differentiated profile and the team's excellent execution against competition from multiple big pharma companies. We expect additional growth opportunity for Scythophora ahead. We are very excited about the upcoming launch of EUROPATH in the US. EUROPATH is the first and only product to be FDA approved for the treatment of hyperparathyroidism in adults. There are 72 90,000 adults with hypoparathyroidism in the U.S. Who would potentially benefit from this treatment? The highly positive pivotal data in AccraData that we announced in September for our third product candidate, Transcon-CMP, further strengthens our belief that it could if the crew becomes the treatment of choice in this multi-billion dollar market opportunity. Our new partnership with Novo Nordisk highlights our ability to extend the success of our transplant platform into large, high-volume therapeutic areas. By staying focused on our values of patient, science and passion, we believe we are creating extraordinary value for patients and stakeholders. I will now provide more detailed comments on each of these products and other key areas of progress. Starting with Skytofa. The fundamentals for Skytofa are strong. Demand volume in the third quarter increased more than 60 year over year. As physicians, caregivers and patients continue to recognize the benefit of Skytofa provides. We have achieved broader market assets for Skytover, but have not compromised on its value, maintaining a net value per patient of around three times that of daily growth hormone. Over time, we see an opportunity to drive Skytover growth beyond pediatric growth hormone deficiency with a number of label expansions. In September, we submitted and supplemented VLA for adult growth hormone deficiency. Next month, we expect top-line data from our phase 2 trial internal syndrome. And next year, we expect to initiate a scitrophage basket trial in established growth hormone indication, such as idiopathic short stature, small for gestational age, and the genetic condition shock, which include Turner's syndrome. Importantly, the daily growth hormone market is going to a consolidation with players such as Lilly and Genentech announcing plans to exit the market. Today, we repeatedly see that when giving the choice, Skytrover is the preferred product. The market converts to once-weekly treatment We therefore believe SCARTROFA is well positioned to compete for the 85% of prescriptions still being written for daily growth hormone. We are dedicated to make SCARTROFA into a blockbuster in the US alone. Our focus will stay on growing its market share with new patients, both treatment-naive and those switching from daily growth hormone. Increasing treatment adherence and duration and through label expansion. Supported by pricing that recognize the value of lung acting therapy. Now moving to UR-PET. We are preparing for the imminent launch of UR-PET in the US. Next month in December, we plan to begin accepting prescriptions and start the reimbursement process for the around 200 patients already being treated with UO-PATS in preparation for commercial product availability in mid-January 2025. From January 1st, we expect to begin accepting prescriptions for all adults with chronic hyperparathyroidism. To support our strong and successful U.S. loans, we have invested to expand our commercial infrastructure. including building a field organization for UiPath that is three times the number that covers Kiteofa. Since approval, our expanded UiPath field team has focused on engaging endocrinologists who treat adults, including among key opinion leaders and healthcare providers who are actively involved in the treatment of hyperparapacients. This market is driven by endocrinologists, a relatively concentrated specialty. And we estimate around 1,200 physicians have around 30,000 chronic hyperparalyzed patients in their care, or an average of about 25 patients each. We expect the initial uptake of UOPADs in the US to come from four segments. There are around 200 patients already on UOPADs, the 350 to 400 patients in the NAPA special use program that is ending soon, the last population of 4,000 to 5,000 PTH-experienced patients, and around 75,000 broader PTH treatment naive population. Outside the U.S., uptake of UroPath continues to be robust. As of today, There are now around 600 patients on commercial therapy in Germany and Austria, where the commercial launch began this year in January, and across named patient programs in multiple other countries. We believe EUROPASS is a truly unique product. We have designed it to have the same mode of action and distribution in the body as industrial speech aids, and to provide active PTA within physiological levels for 24 hours, 7 days a week. And we don't see any other compounds in the development that share these key attributes. We believe that EURAPAD will become the therapy for the majority of adults with hyperparathyroidism, which is aligned with the recently established guidelines for the treatment of hyperparathyroidism in adults. I will now provide some commentary on Transcon-CMP. Our value proposition for Transcon-CMP is simple. It is to establish a treatment for patients of all ages with achondroplasia. Our previous trial results showed that Transcon-CMP not only exceeded benchmark for growth that have been established in other randomized clinical trials, but also impacted other endpoints that are important for individuals with achondroplasia. We believe TransconCMP has a best-in-class efficacy profile with safety and tolerability similar to placebo, including an excellent injection site tolerability and once-weekly dosing. This sets the stage for its leadership in the achondroplasia market. We expect to submit an NDA to the FDA for Transcon-CMP for the treatment of children with acondyloplasia during the first quarter of 2025 and an MNA to the EMEA during the third quarter of 2025. The two once-weekly growth-promoting products in our portfolio, Skytropha and Transcon-CMP. We believe Ascendi is positioned to become the leader in treatment of growth disorder. We expect top-line week 26 data from COATS, our first combination trial of Transcon Growth Hormone and Transcon CMP in children with achondroplasia aged 2 to 11 years in the second quarter of 2025. Now moving to our recently announced Novo Nordisk collaboration. We believe this agreement is a strong validation of our ability to drive innovation, benefit patients and expand the Transcon technology platform to a larger therapeutic area as described in our vision 2030. The lead program in the collaboration is a once-monthly GPL-1 that will initially target obesity and type 2 diabetes. The global market for GPL-1s like Similutide is expected to exceed more than 50 billion this year and to double or triple in the next 10 years. And once monthly, GPL-1 could become the treatment of choice in this future market. And we are pleased to be working with NOVA Nordisk, a world leader with the manufacturing capacity and commercial infrastructure to realize the value of this opportunity. Financially, upon closing, we will receive an upfront payment of 100 million U.S. dollars and escalating tiered mid-single-digit royalties on global net sales of Transcon products, along with development, regulatory, and sales milestones. I will close with an update on our Oncology program. In September, we presented first results from the Plasm-Assisted OVM Cancer Cohort of the phase 1-2, I believe, trial of Transcon IL-2 beta gamma at ESMO, showing that anti-tumor clinical response was observed in 29 of efficacy-related patients treated with Transcon IL-2 beta in combination with chemotherapy. This was the second indication-specific cohort showing meaningful signs of anti-tumor activity in heavily pre-treated patients. Given these results, recently we closed enrollment to dose expansion cohort in the TRANSCOM IT101 and I believe trials of TRANSCOM TLR78 agonists to highly prioritize our effort on TRANSCOM IL-2 Beta Gamma. In summary, our progress and position are strong, and we believe the expected product revenue and strength of our balance sheet give us the ability to invest in global launches, label expansion, and lifecycle management for all of our three rare endocrinology programs to support each of them to reach blockbuster status. And at the same time, we will continue to invest in new product candidates created by our TransCon technology platform to build sustainable growth and profitability. I will now turn it over to Scott for a financial update.