Ascendis Pharma A/S

Good day, and thank you for standing by. Welcome to the fourth quarter 2023 Ascendus Pharma 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 a question during the session, you will need to press star 101 on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question, please press star 101 again. Please be advised that today's conference is being recorded. I would now like to hand the conference over to your first speaker today, Tim Lee, Senior Director of Investor Relations. Please go ahead.

Thank you, Operator, and thank you, everyone, for joining our full-year 2023 Financial Results Conference call. I'm Tim Lee, Senior Director of Investor Relations at Ascendance Pharma. Joining me on the call today are Yen Mickelson, President and Chief Executive Officer, Scott Smith, Executive Vice President and Chief Financial Officer, Dr. Steena Singhal, Executive Vice President, Head of Clinical Development Oncology, and Joe Kelly, Senior Vice President, Head of U.S. Commercial Endocrinology. Before we begin, I'd 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 statement may include, but are not limited to statements regarding our commercialization and continued development of Skytrofa for the U.S. and European markets, as well as certain financial expectations for 2024, our commercialization and development of your path in the EU and expected timing of the FDA review and potential launch of Cranscom PTH in the US, 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 our ongoing and planned regulatory filings, and our expectations regarding the timing and the results of regulatory decisions, our ability to create value in multiple therapeutic areas outside of endocrinology rare disease, our progress towards Vision 2030, and the potential success of ICONIS. 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 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 20-F filed with the SEC later today, February 7, 2024. Kranskon Growth Hormone, or Kranskon HGH, is approved in the US by FDA, and the EU has received MAA authorization from the European Commission for the Treatment of Pediatric Growth Hormone Deficiency. The European Commission has granted marketing authorization for Kranskon 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 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 full year 2023 financial results and we'll provide further business updates. Following some prepared remarks, we'll then open up the call for questions. With that, let me turn it over to Yen.

Good afternoon, everyone. 2023 was a transformative year for Ascendis Pharma. We streamlined the company, including our structure, our processes, and operating expense allocation. And at the same time, we believe we are on track to achieve our vision 3x3 by 2025. Skytrover is the leading growth hormone product in value in the U.S. belongs in Europe direct, and international markets is underway, beginning with Germany and Austria. And our clinical program for transplant CMP is progressing to what people will data expected in Q4. We believe Ascendis is advancing to become a leading biopharma company with a strong focus on endocrinology rare diseases. and capabilities to also create value in other therapeutic areas, as we're doing in oncology, ophthalmology, and metabolic diseases. Because of the decisions we took in 2023, we believe Ascendis is a leaner, more efficient organization in 2024, well-positioned to fulfill vision 2030 with three independent endocrinology rare disease blockbuster products and expand our engine for future innovation. Now, let me provide an update on each program. From the beginning, we designed Skytover to be a best-in-class growth hormone product by releasing unmodified somatopoeia. By addressing the needs of the patient, caregivers, physicians and payers. Skytopa achieved U.S. market value leadership in 2023, just two years after launch. We estimated that Skytopa penetration in the U.S. pediatric growth hormone deficiency patient population was around 16% at the end of 2023. With Skytrofa expanding the U.S. growth hormone market to the potential of 3 billion U.S. dollars, we believe Skytrofa has the potential to be a blockbuster on U.S. revenue alone. Built on this value leadership, we expect our first label expansion to be adult growth hormone deficiency. For this, we plan to submit and supplement BLA to the FDA in the second quarter of this year. In addition, we expect top-line data from our Phase 2 trial internal syndrome in the fourth quarter of 2024. Now, turning to Transcon PTAs. Last week, we initiated the launch of Transcon PTAs in Europe direct and international market. Market as your VPATs. with full commercial availability in Germany and Austria at an initial list price of 105,000 euros per patient per year. An estimated 70,000 adult patients in Germany are living with chronic hyperparathyroidism, and our initial longs will target 22,000 of those. EURAPAD's EUROPE Direct Launch is off to a great start, with our first prescription and orders proceed at the first day of launch, demonstrating the serenity of the disease and the unmet medical need. We are continuing to expand our EU Direct infrastructure and expect availability of EURAPAD across Europe by the end of 2025. In parallel, to meet the needs of the patients, we plan to provide commercial reimbursed product to early access routes such as main patient programs. The US PDUVA date for Transcon PTH is May 14. If approved, we plan to launch our UR-PAT as quickly as possible thereafter to our established US commercial infrastructure. Moving now to Transcon CMP. Our clinic-level program is designed to support approval of Transcon-CMP as a treatment for patients of all ages with achondroplasia. Transcon-CMP dose at 100 micrograms per kilo per week has demonstrated superiority compared to placebo in two 52-week trials. In the open-label extension, we observed unexpected strong patient retention of 97% for now up to four years. As a result of this unexpected finding, we continue to review our own data and scientific literature. We believe now that acroendoplasia is both a growth disorder and a muscle disorder. As presented at the JPMorgan conference in January, we showed significant improvement in health and quality of life for children with aconthoplasia treated with Transcon-CMP compared to placebo. With this growing insight, we have designed our pivotal approach trial to evaluate meaningful benefits related to all aspects of aconthoplasia, including linear growth, and improvements in co-morbidity as measured by radiological endpoints, physical function, body composition, and quality of life. We expect to share top-line data from this trial, and we plan to submit an NDA in the fourth quarter of this year. In addition, we are pursuing additional treatment opportunities in aquantoplasia. Infant trial, age 0 to 2 years, first patient already had been enrolled. Combination trial with chytophthora, age 2 to 11. Week 26, top line, analyze growth velocity in the fourth quarter of 2024. Adult, plan to file an IMD or similar in the third quarter of this year. Switching now to oncology. Both Transcon IL-2 Beta Gamma and Transcon TR-78 Agonist had shown favorable safety profiles and single-agent clinical activity. We expect 2024 to be an extremely important year for oncology pipeline, with data readout in well-defined patient population in the fourth quarter. Also in the fourth quarter, we expect to have completed enrollment of our randomized phase two trial in neuroadjuvant head and neck cancer. Longer term, to maximize this potential reach and value of these oncology product candidate, we aspire to commercialize our oncology classes through partnership with companies with specialized oncology experience and capability in late-stage development and commercialization. Moving now to Ophthalmology, we recently announced the formation and launch of an independent company, ICONIS, to develop and commercialize Transcon Ophthalmology products globally. We have received an equity position in the new reform company, and we are eligible to receive development, regulatory, and sales milestones, plus single-digit royalties. In January, we introduced Vision 2030, our strategic roadmap to achieve blockbuster status for each of our three endocrinology rare disease products and expand our engine for future innovation. With the broad applicability of the Transcon technology platform, our goal, as laid out in Vision 2030, is to apply our algorithm for product innovation to establish opportunities in greater than US$5 billion, as we did with the NGVGF and GPL1 classes, where we believe we have designed the best in-class programs. We are pleased with the interest in our once-monthly GPL1 program and from our platform technologies we expect to continue to generate many more opportunities in the future. I will now turn the call over to Scott for financial review before we open for questions.