Ascendis Pharma A/S

Jay, and thank you for standing by. Welcome to the Ascendance Farmer Full Year 2022 Financial Results Conference Call. At this time, all participants are on 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 11 on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question, press star 11 again. Please be advised that today's conference call is being recorded. I would like to turn the call over to now your speaker for today, Tim Lee, Senior Director of Investor Relations. Please go ahead. The floor is yours.

Thank you, Operator, and thank you, everyone, for joining our full year 2022 Financial Results Conference Call. I'm Tim Lee, Senior Director of Investor Relations at Ascendance Pharma. Joining me on the call today is Jen Mickelson, President and Chief Executive Officer, Scott Smith, Executive Vice President and Chief Financial Officer, Dr. Stina 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 statements may include but are not limited to our U.S. commercialization and continued development of Skytrofo for the U.S. market, the commercialization of Transcon HGH for the EU market, statements regarding the expected timing of approval and launch of Transcon PTH in the U.S. market this year, statements regarding the expected timing of approval of Transcon PTH in Europe, statements regarding the potential size of the Transcon PTH, the market size for Transcon PTH, 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, including the timing of clinical results, statements regarding our pipeline product candidates, statements regarding our plan regulatory filings, our expansion into new therapeutic areas, and statements regarding the ability to create a sustainable leading global biopharma company. These statements are based on information that is available to us today. Actual results and events could differ materially from those in the forward-looking statements, and we may not be able to achieve our goals, carry out our plans, our intentions, our 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 our forward-looking statement section in today's press release and the risk factor sections of our most annual report on Form 20F, which is being filed today, February 16, 2023. Transcon Human Growth Hormone, or Transcon HGH, is approved by the FDA in the U.S. under the brand name Skytropha for the treatment of pediatric patients one year or older weighing at least 11.5 kilograms and have growth failure due to inadequate secretion of endogenous growth hormone. In addition, the European Commission has granted a marketing authorization for Skytropha to Ascendus Pharma developed under the name Transcon HGH It's a once-weekly subcutaneous injection for the treatment of children and adolescents aged 3 to 18 for growth failure due to insufficient secretion of endogenous growth hormone. In general, we refer to this product as Transcon growth hormone unless we're referring to the product in the context of particular jurisdictions such as the United States or the European Union. Otherwise, please note that our product candidates are investigational and are not 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 our product candidates shall be viewed as promotional. On the call today, we'll discuss our full year 2022 financial results. We'll provide further business updates. Following some prepared remarks, we'll then open up the call for questions. I'll now turn the call over to Jens Mikkelsen, President and Chief Executive Officer. Jens?

Thank you so much, Tim. Ascend is built on the unique Transcon technology platform, which enables development of highly differentiated product candidates across multiple therapeutic areas. Combining the Transcon technology with our algorithm for product innovation has enabled us to create and develop product candidates with a higher likelihood of success than seen with conventional drug development. One of our key product selection criteria is to fulfill best-in-class potential on each of the four key pillars of drug development. efficacy, totability, and convenience. In addition, each product candidate must have the potential to achieve $1 billion or greater revenue in a single therapeutic indication. With this approach and driven by our values of patience, science, and passion, we have demonstrated our ability to continuously build out a robust pipeline, while taking product candidates from concept through approval and launch. With expected regulatory approvals of a new product or additional indication every one to two years, we are fulfilling our vision 3x3 goal of building a sustainable, profitable, leading biopharma company and creating long-term value for all stakeholders. This past year, we have advanced our pipeline as planned, entering 2023 with an April 30 PDUFA date and expected US launch of Transcom PTH for adult patients with hyperparathyroidism by the end of Q2. with an expected European Commission decision during Q4 as well. Transcon PTA is our second endocrinology rare disease product opportunity, representing a potential global opportunity greater than $5 billion. Turning to Transcon CMP, last November, we reported 12-month data from our first from the first ever randomized double-blinded placebo-controlled phase 2 trial in children diagnosed with achondroplasia. These results give me confidence that this third endocrinology rare disease product candidate may have its first approval by 2025 as target in our vision 3x3. Another component of Vision 3x3 is label and geographic expansion. We continue to build the value of our existing programs through additional clinical studies for label expansion and global commercial needs. Starting with our newly expanded European organization, which is preparing for launch of Skytropha in Germany this year, and if approved, Transcon PTA next year. With this great momentum across our pipeline, I would like to review additional details from our major programs. Turning to growth hormone. During the fourth quarter of this year, we plan to report top-line results from our global Phase III foresight trial in adult growth hormone deficiency. Our potential second indication for Transcon growth hormone. Adult growth hormone deficiency is a serious endocrine rare disease characteristic by abnormal body composition, dyslipidemia, insulin resistance, and impaired quality of life. Analysis has shown these consequences of adult growth hormone deficiency result in mean analyzed health care costs more than four times that of a non-growth hormone deficient population. Because Transcon growth hormone is the only once-weekly growth hormone product releasing unmodified somatropine, we expect it to be the first adult growth hormone treatment to meet or exceed the safety, efficacy, and solubility of daily growth hormone. Meanwhile, in the US, Skytropha is experienced the commercial success it deserved because of its unique product strengths. As we pre-announced during J.P. Morgan, fourth quarter 2022, U.S. Skytropha revenue growth to 17.1 million euro, providing a strong fundamental growth in 2023 and after. With our progress towards label expansion and plan commercial launch in market outside the US. We believe we can track, we are on track to build SCARTOFA into the leading growth hormone product in value by increasing the total market size. As we have predicted, we are seeing the consolidation of the data growth hormone market as other manufacturers begin to exit the US market. Turning to Transcon PTH, excitement continues to build among stakeholders around this potential treatment for adult patients with hyperpair ahead of the upcoming PDUFA date of April 30. Our expanded teams are hired, trained, and working to deepen physician and payer awareness of this serious health and quality of life issue that hyperparal causes. We have already made more than 2,000 calls to physicians related to disease awareness, and we are encouraged by their interest in learning more about the multi-organ impact of this disease and its negative effect on patient quality of life. Our commercial team, medical affairs, field reimbursement and manufacturing teams are ready to launch Transcon PTH in the US market as soon as possible after approval. Importantly, we are launching Transcon PTH, our second endocrinology rare disease product with the same commercial infrastructure that has proven its success with Chytropha. Coming back to CMP. As we did with Transcon Growth Termon and Transcon PTA, we ran a robust phase 2 trial to confirm Transcon CMP's target profile on all four key pillars, safety, efficacy, tolerability, and convenience, and de-risk it at the phase 2 level. This can only be done with a robust randomized placebo-controlled trial that will mimic the pivotal trial. We saw clear success in the complex trial with Transcon-CMP, demonstrating superiority over placebo at the 12-month primary endpoint in children aged 2 to 10. In addition, we saw clear dose response. 57 patients who started this trial remain in the OMAL label extension today. To extend and confirm this result, including positive treatment effect observed on acondoplasia-related comorbidities, we are running our Phase 2b approach trial. As investigators are aware of the Phase 2 results, we experienced very high interest in our approach trial. And we expect to complete target enrollment of around 80 patients in the next quarter. During our upcoming end of phase two meeting with FDA, we expect to collaborate on how to best achieve a broad treatment labeling rather than a linear labeling alone. Shifting to oncology. We are progressing with the developing of our two novel immuno-oncology programs, Transcon TLR78 Agonist and Transcon IL-2 Basagamma. With these two clinical programs, we are positioned this year to start evaluation of clinical efficacy in seven specific tumor types, nine different indications with four different combination therapies. including by combining our two Transcon oncology product candidates with each other. Clinical proof of concept phase 2 top line results are expected starting in 2024. In addition, this year we will initiate the randomized phase 2 trial BELIEVE201 using Transcon IL-2 beta gamma and TLR7 agonist combination therapy in head and neck cancer. As we successfully demonstrated with our endocrinology programs, we are building a solid phase two clinical proof of concept for our oncology products in multiple tumor types in the next one or two years. As you can see, we have and will always focus on achieving best in class product profile to benefit patients on the four key pillars of safety, efficacy, suitability and convenience. Errors in which we will not compromise. This development approach, including extremely robust clinical trial design, has positioned incentives to potentially launch a new product or indication every one to two years, building sustainability, long-term value for all stakeholders. Each successful clinical trial further confirms the power of the ISKCON technology platform and our product innovation algorithm and increases our confidence and likelihood of success for future product candidates. With an expanding pipeline and commercial successes, Ascentis remains on track to meet or exceed our goals outlined in our vision 3xT. I will now turn the call over to Scott