Ascendis Pharma A/S

Good day and welcome to the third quarter 2025 Ascendus Pharma earnings conference call. At this time, all participants are in listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question, please press star 1-1. If your question has been answered and you'd like to remove yourself from the queue, press star 1-1 again. We ask that you limit yourself to one question and one follow-up. I would now like to turn the call over to Chad Fugier, Vice President of Investor Relations at Ascendus Pharma. Please go ahead.

Thank you, Operator, and thank you, everyone, for joining our third quarter 2025 Financial Results Conference Call. I'm Chad Fugere, Vice President of Investor Relations at Ascendus Pharma. Joining me on the call today are Yen Mickelson, President and Chief Executive Officer, Scott Smith, Executive Vice President and Chief Financial Officer, Sherry Glass, Chief Business Officer, Jay Wu, EVP and President, U.S. Market, and Amy Hsu, EVP and Chief Medical Officer. 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, statements regarding our commercialization and continued development of Skytropha and YorviPath, as well as certain expectations regarding patient access and financial outcomes, our pipeline candidates, and expectations with respect to their continued progress and potential commercialization, our strategic plans, partnerships and investments, our goals regarding our clinical pipeline, including the timing of clinical results and trials, our ongoing and planned regulatory filings, and our expectations regarding the timing and the result of regulatory decisions. These statements are based on information that is available to us as of today. Actual results may 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 these 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 most recent annual report on Form 20F, filed with the SEC on February 12, 2025. Transcon Growth Hormone, or Transcon HGH, is now approved in the U.S. by the FDA for the replacement of endogenous growth hormone in adults with growth hormone deficiency. In addition to the treatment of pediatric growth hormone deficiency, and the EU has received MAA authorization from the European Commission for the treatment of pediatric growth hormone deficiency, Transcon TTH is approved in the U.S. by the FDA for the treatment of hypoparathyroidism in adults and European Commission and the United Kingdom'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 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 2025 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 Jan.

Thanks, Chad. Good afternoon, everyone. In the third quarter of 2025, we accelerated our momentum towards fulfilling our vision 2030 with key achievements in three areas. First, the global launch of EUROPAT continues to be strong with a steady increase in new unique patient prescriptions and prescribers as seen in Q1 and Q2 along with expansion in new geographic markets. We made great advancement towards leadership in growth disorders during the quarter. We saw the US approval of Skytropha in adult growth hormone deficiency. And following our late cycle meeting with FDA, we are progressing toward expected approval of Transcon-CMP in the US. Third, our strong operating fundamentals led to positive operating profit, signaling the beginning of sustained revenue and earnings growth for Ascendis. Now I will provide some specific comments on our commercial and day-to-day portfolio. Starting with EuroPath. EuroPath continues its strong global launch with revenue of 143 million euros in the third quarter. Nine months in the lungs in the biggest market, the U.S. Patient demand continues growing quarter by quarter. From lungs to the end of September, more than 4,250 patients have been prescribed UOPADS in the U.S. by over 2,000 unique healthcare providers, highlighting the strong, steady demand for UOPADS. even during the summer months. In October, the positive trend continued with EUAPET being prescribed for more than 400 new patients in the US alone. Positive physician and patient experience are driving a high rate of compliance. And we expect most patients will be on lifelong PTH therapy. We are expanding our physician weeks each quarter within the endocrinology community. And we are also expanding to other physician groups who manage hyperparapathies. An example, at last week's American Society of Nephrology meeting, we presented three years of kidney function data across our combined clinical trials, demonstrated sustained clinical meaningful improvement in kidney function in the UOPATH-treated patient. In addition, we continue working hard to expand patient access in the U.S. The overall insurance approval rate since the start of the launch is around 70% of total enrollment. And we believe this figure will continue to increase over time. We currently see approval across all payer types with a majority of approvals within eight weeks. We are pleased by the robust uptake of Europass. in our first three quarters of commercialization in the US. Today, less than 5% of US patients are currently on UroPath treatment. We see significant room to grow with around 80,000 to 90,000 patients already diagnosed with chronic hyperparalysis in the US and 3,000 to 4,000 new patients being diagnosed every year. Outside the US, EUROPATH is now available commercially or to name patient program in more than 30 countries. In Germany, Austria and Spain, we have now full commercial reimbursement. In Japan, our partner ChaiGen launched EUROPATH commercially last week following approval in August. We are looking forward to the commercial launch of UroPath in additional countries in the coming years. With a broad label covering hyperparathyroidism for all causes, international treatment guidelines that recommend PTH replacement therapy, and UroPath positioned as first-in-class therapy, we expect sustained patient growth and revenue growth for years to come. As we're building this global market, we're expanding our offerings to patients with hyperparam. We are conducting the part-way 60 trial to support doses up to 60 micrograms of UOPAD in the US. We plan to begin a clinical trial for people under 18 this quarter, and we are advancing our new one-weekly Transcon PTH product candidate, which we believe will be an attractive option for patients on stable doses of UroPads. In the new year, we will share more on our plans to maximize UroPads' value and reach even more patients. Let us now turn to growth disorder, which today compiles of our once-weekly growth hormone Skytropha approved for growth hormone deficiency and our once-weekly Transcon CP currently under review by FDA in the US and by EMEA in EU for children with achondroplasia. Skytropha is approved in the US and EU for treatment of pediatric growth hormone deficiency. With this single indication, Skytrofa is established as a high-value brand and treatment of choice for pediatric growth hormone deficiency. Q3 revenue for Skytrofa was 51 million euros. In July, we received our first label expansion with FDA approval for adult growth hormone deficiency, the first of multiple planned label expansion. In Q3, we initiated our Phase III basket trial of Scottsdorfer with a range of established growth disorder, including ISS, shock deficiency, Turner syndrome, and SDA. Turning to Transcon-CMP, we recently completed a late cycle meeting with the FDA and are in the final stage of the label discussion. Transcon-CMP is under priority review in the US with a due date of November 30 and is also under review in the EU where our MIA file was recently validated. Transcon-CMP once weekly is well positioned to become the leading treatment for children with acondoplasia. with the full degree of linear growth outcome that can be achieved with monotherapies addressing the overactive tyrosine-3 kinase. In addition, Transcon-CMP achieved statistical improvement in leg bowing compared to placebo, increasing spinal canal dimension, a safety and solubility profile compared to placebo, with a very low rate of injector site reaction. and no cases of symptomatic hypertension. We are confident in Transcon-CMP's ability to be a leading therapy. While we believe Transcon-CMP monotherapy is transformative by itself, we want to further enhance outcomes for people living with achondroplasia. Earlier this year, we presented 26-week results from the Phase II COACH trial of Transcon-CMP in combination with Transcon-Grotamo, which showed around three times improved linear growth compared to what had been observed with monotherapies over the same time period. This resulted in healthy linear growth in children with achondroplasia. higher than that observed with an average state of children, accommodated by improvement in body proportionality and without acceleration of bone age. This data has been recognized by key opinion leaders as groundbreaking. Based on this data, we believe, over time, the standards of care in achondroplasia will include combination therapy as a treatment option. building on the potential role of transcon-CFP as the backbone therapy. Following our recent FDA End-of-Phase II meeting related to our combination therapy, we plan to initiate a Phase II trial this quarter. We anticipate disclosing 52 RIG data from the close trial in early 2026. With one weekly growth hormone and one weekly CMP, two highly differentiated medicines, both as monotherapy and in combination, we believe Ascend is in a well-positioned to become the global leader in many different growth disorders. Our vision 2030 also includes creating value through partnership. And we see that being achieved through the rapid progress of Taijin in Japan, Vision in China, Iconis in ophthalmology, endovenolates in metabolic and cardiovascular diseases, where the once-monthly semi-glutide program is making fast progress towards the clinic. And finally, the commercial success of EUROCAT and SCARTOVA has already transformed the financial profile of attendees. In the third quarter, we achieved positive operating income. along with positive cash flows. For the near time, the building out of our commercial organization is largely completed in advance of future global launches. For the medium term, labor expansion, LCM activities have been indicated to maximize the value of our current products. At the same time, for long-term sustainability, our R&D organization continues to advance the Transcon technology platform to ensure a constant flow of new programs and potential new products. In summary, with Transcon CMP nearing potential approval, Ascendis is well positioned to get approval of its third Transcon-based product in a row. This highlights the uniqueness of Ascendis that continues to develop a highly differentiated product created by the Transcon Technology Platform and our unique low-risk drug development algorithm. Importantly, our current three rare disease endocrine products position us for doable future growth and give us confidence in our aspiration to achieve €5 billion or more in annual profit revenue in 2030. I will now turn it over to Scott