Ascendis Pharma A/S

Hello, thank you for standing by. Welcome to Ascended Farmer's second quarter 2024 earnings 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 the question during this session, you will need to press star 11 on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question, please press star 11 again. We ask that you limit yourself to one question and one follow-up. I would now like to turn the call over to Tim Lee, Senior Director of Investor Relations at Ascendus Pharma. Sir, you may begin.

Thank you, operator, and thank you, everyone, for joining our second quarter 2024 financial results conference call. We apologize for the delay due to some technical issues that we had here. I'm Tim Lee, Senior Director of Investor Relations at Ascendus Pharma. Joining me on the call today are Yen Mickelson, President and Chief Executive Officer of Scott Smith, Executive Vice President and Chief Financial Officer. Dr. Stina Singhal, Executive Vice President and Head of Clinical Development Oncology. Camilla Harder Hartvig, Executive Vice President and Global Chief Commercial Officer. And Joe Kelly, US General Manager. Before we begin, I'd like to remind you that this conference call will contain four linking statements that are intended to be covered from the safe harbor provided by the Private Securities Litigation Reform Act. Examples of such statement may include but are not limited to statements regarding our commercialization and continued development of Skytrolipha and Europath, the US 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, our goals regarding our clinical pipeline, including the timing of clinical results, are ongoing and planned regulatory filings for expectations regarding the timing and the results of regulatory decisions, and are exploration of market opportunities and therapeutic areas outside of endocrinology rare disease. These statements are based on information that is available to us as of today. Actual results may differ and could differ materially from those in our forward-looking statements and 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 can cause actual results to differ materially, please see our forward-looking statement section in today's press release and the risk factors section of our most recent annual report on Form 20F, filed with the SEC on February 7th, 2024. Kranskon Growth Hormone, or Kranskon HGH, is approved in the U.S. by FDA, and the EU has received MAA authorization from the European Commission for the Treatment of Pediatric Growth Hormone Deficiency. Kranskon PTH is approved in the U.S. by the FDA for the treatment of hypoparathyroidism in adults, and the European Commission and the United Kingdom's Medicines and Healthcare Products Regulatory Agency have granted marketing authorization for Kranskon PTH as a replacement therapy indicated for the treatment of adults with chronic hyperparathyroidism. Otherwise, please note that our product candidates are investigational and not approved for commercial use. As investigational products, the safety and effectiveness of 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 second quarter of 2024 financial results, and we'll provide further business updates. Following some prepared remarks, we will then open up the call for questions. With that, let me turn it over to Jens.

Thanks, Tim. Good afternoon, everyone. With the recent U.S. approval of UR-PET as the first and the only FDA-approved treatment of hypothyroidism in adults, Ascendis has successfully obtained approval for two out of three endocrinology rare disease product candidates in two major markets, the US and EU. And with pupil cell data from our third product candidate, Transcon-CMP in Akron, Malaysia, expected in the coming weeks, We are nearing our vision to achieve approval of all three product candidates by the end of 2025. Our algorithm for product innovation, combined with our Transcon technology, have enabled us to bring new, highly differentiated product candidates through the clinical development process faster and with a higher success rate compared to traditional drug development. We attempt to continue to develop new product candidates in endocrine disease and last therapeutic areas such as oncology, obesity, metabolic disease, and cardiovascular with best-in-class potential to make a meaningful difference for patients across the globe based on our strong scientific focus. Let us begin with UROPATS. In the US alone, an estimated 70,000 to 90,000 patients are living with hypoparathyroidism. For those who haven't seen it, please take time to watch the patient-arranged FDA hearing on the hypoparathyroidism associated website, providing an in-depth understanding of the serious consequence of having this disease. With the FDA approval of UROPAD, physicians and adult patients in the United States can now look forward to having a treatment option for treatment of hypopathy. We are preparing for EURAPATH's launch in the US, leveraging our established commercial infrastructure and expertise. We are expanding our dedicated team of sales reps and field medical personnel who will engage with around 6,000 physicians who treat 80% of patients with hyperparalysis in the U.S. Other key long-term initiatives are underway, including the roll-out of our patient support programs designed to support access to UroPaths. For example, eligible patients on commercial insurance will pay as little as $5 a month for the UOPAT prescription. In addition, following our goal to take care of all patients with this disease, we will also introduce a patient assistance program. We have also started engaging US payers and PBMs. We expect product availability in the US in the first quarter of 2025 or sooner if it's possible. Consisting with premium responsible pricing, we will launch with a VAT price corresponding to $285,000 annual per patient, reflecting the value of EUROPED to the U.S. healthcare system. In the U.S., there are around 140 patients currently active in the expanded SS program and about 50 more patients in the open-label extension of our clinical studies. Physicians will begin transplanting these patients over to commercial products as soon as it is available. In Europe, the number of patients and prescribing physicians initiating EUROPAT continues to increase, and we see a good mix of PTH experienced and new patients. This was the first full quarter of commercial loans in Germany and Austria, where sales momentum continues to build. We now have more than 250 patients on treatment and an estimated 125 prescribers in these two markets. UroPath patient retention rate is extremely strong and currently around 98%. As physicians gain more experience at UiPath, we expect them to bring more patients on therapy, including those finishing the remaining supplies of NADPAR. The interest for serving patients under named patient programs prior to full commercial loans is increasing. We now have patients in these programs in more than 10 countries and expect more by the end of the year. Moving to Skytrooper. We are proud to have more than 11,000 patients prescribed Skytropha in the first three years since launch, and to have achieved market value leadership while expanding the overall growth hormone market. Key components of our strategy to make Skytropha a blockbuster product in the U.S. include simplifying broadened market assets for both treatment-naive or switched patients, as well as expanding our labels. In the first half of the year, the reset to broader market access for Skytofa was largely completed. While this broader access to Skytofa will support long-term demand, in the short term it negatively impacted our first half net revenue. Scott will share more details. With our market assets transition largely completed, Skytrofa is now positioned as a premium product with a net value per patient of around three times compared to daily growth hormone. We are now focusing on using our new market assets coverage to drive further demand, continue to expand the overall growth hormone market, and are aiming to reach blockbuster status for Skytrofa in the US alone. Finally, to build on our market leadership position, we plan to submit and supplement BLA in adult growth hormone deficiency to the FDA in the third quarter of this year. Our first sclerotrophic label expansion. We also expect top-line data from our phase 2 trial in Turner syndrome in the fourth quarter of 2024. Switching to Transcon-CMP. I have always been extremely excited about our program in achondroplasia, and much more now as we are proposing the results from our February trial in the coming weeks. We have consistently in our messaging over the past eight years since we announced our product candidate that our aim is to develop a treatment that addresses both linear growth and the comorbidities that affect health and quality of life for people living with achondroplasia. Earlier this year, you saw a comprehensive result for our Phase 2 accomplished trial, demonstrating that our once-weekly Transcon-CMP increased analyzed growth velocity similar to Rosoratide about 5.6 cm after 12 months of treatment. In addition, for first-time error for a product in the setting of agronoplasia, We also demonstrated that compared to placebo, Transcon-CMP improved quality of life associated with the physical function of well-being in children with achondroplasia with a favorable side effect and tolerability profile. Now, we hope that we can replicate these results with more patients in our pivotal approach trial, and we are expecting top-line data in the next few weeks. one quarter earlier than guided. This trial enrolled 84 children aged 2 to 11 with achondroplasia. The mean age was 5.7 years, similar to our phase 2 trial. We also continued to enroll in COATS, a phase 2 trial of Transcon-CMP in combination with Transcon-Globetamol-Skytofe. The scientists show that adding scartofa to Transcon CMP could provide cash-up growth for patients who start CMP treatment late. We expect to complete enrollment in this combination trial during the first fourth quarter of 2024, with top-line 26 data expected in the second quarter of 2025. Turning now to oncology. We continue advancing three Phase II trials with multiple indication-specific cohort to study the best-in-class potential of our two product candidates, Transcon IL-2 Beta Gamma and Transcon TLR78 Agonist, in different combination scenarios. We plan to present initial result from our Transcon IL-2 beta gamma in combination with chemotherapy in plasma resistant ovarian cancer from the IL-BELIEVE trial later this month at the ESMO conference in Barcelona. I'm pleased how these programs are progressing. In closing, for a sentence, it's all about the patients. Patients tell us the US approval of UOPAT is transformative for them. We hear from parents that Skytrover has changed the life of children and parents both. With data expected in the next weeks for Transcon CMV, it is our goal that we are able to show that we also can transform the life of people living with achondroplasia. With our ongoing progress in our oncology program, and exploration of other areas of innovation in large market opportunities such as obesity. We continue to position incentives for sustainable growth with an expanded pipeline and transformative TransCon product candidates. I will now turn it over to Scott for a financial update.