Ascendis Pharma A/S

Thank you for standing by and welcome to the Ascendance Pharma 4th Quarter 2025 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 during this session, you'll need to press star 1-1 on your telephone. If your question has been answered and you'd like to remove yourself from the queue, simply press star 1-1 again. We ask that you please limit yourself to one question. As a reminder, today's program is being recorded. And now I'd like to introduce your host for today's program, Chad Fugere, Vice President, Investor Relations.

Please go ahead, sir. Thank you, operator, and thank you, everyone, for joining our full year 2025 financial results conference call. I'm Chad Fugere, Vice President, Investor Relations at Ascendus Pharma. Joining me on the call today are Yen Mickelson, President and Chief Executive Officer, Scott Smith, Chief Financial Officer, Sherry Glass, Chief Business Officer, Jay Wu, Executive Vice President and President Ascendus U.S., and Amy Hsu, 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 YorbiPath, as well as certain expectations regarding patient access and financial outcomes, our pipeline candidates and our 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 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 section of our most recent annual report on Form 20F, filed with the SEC on February 11, 2026. 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 then 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 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 agencies. 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 2025 financial results and we'll provide further business updates. Following some prepared remarks, will then open up the call for your questions. With that, let me turn the call over to Jan.

Thanks, Chad. Good afternoon, everyone. With strong execution across our business and continued progress toward delivering on our vision 2030, Ascend is transforming into a leading global biopharma company. We believe this progression demonstrates the power of our Transcon platform and our R&D capabilities to deliver a sustainable pipeline. While our global commercial infrastructure and financial profile continue to shrink, we believe we are now at the base of a steep where we expect to achieve operating cash flow of around €500 million in 2026, and where we aspire to achieve at least €5 billion in annual product revenue by 2030. And at the same time, we are building an expanded pipeline of blockbuster product opportunities. In the fourth quarter, we saw multiple achievements across the organization, starting with EuroPATS. The fourth quarter was another period of strong execution for the global launch of EuroPATS. Revenue for the quarter was 187 million euros, bringing full year 2025 EuroPATS revenue to 477 million euros. In the U.S., access continues to expand. Two years in, more than 5,300 patients were prescribed UroPath by nearly 2,400 unique healthcare providers, highlighting continued strong and steady demand. To date, less than 5% of U.S. patients are currently on UroPath treatment, highlighting the significant long-term growth opportunity ahead. The overall insurance approval rate is about 70% of the total enrollment, and we continue to see this figure moving higher over time. In addition, we continue to see a majority of approvals within eight weeks. This provides a strong foundation for expected additional growth in 2026. and beyond as more patients initiate EUROPAT in line with treatment guidelines that support its use. Outside the US, we continue to reach more patients. As a reminder, EUROPAT is now available commercially or to name patient programs in more than 30 countries. We have full commercial reimbursement in four countries in our Europe direct markets and two countries in our international markets. In Japan, our partner Tachin launched Europass commercially last November. In 2026, we expect full commercial launches in 10 additional new countries. We also advance development activity to broaden UR-PATS label in a number of areas. In the U.S., we are working to expand the range of doses to our Partway 66 trial, and globally, we continue to advance clinical trials to expand UR-PATS to patients under the age of 18. Our work is progressing rapidly on once-weekly Transcon PTH. for patients who have been titrated with daily UroPaths or conventional therapy and have achieved a stable daily dose for a well-defined period. Last month at the annual J.P. Morgan Healthcare Conference, we shared preclinical data that support the target product profile for a once-weekly Transcon PTA candidate, matching the release PDAs seen with daily UoPath treatment over the entire week, thus providing a comparable efficacy and safety profile. Overall, we remain confident that UoPath has the potential to be a durable, long-term growth driver for Ascentis globally. Turning now to growth disorders. Combining of our once-weekly growth hormone Skytofa or Transcon growth hormone or once-weekly Transcon CMP. Skytofa delivered another solid quarter with Q4 revenue of €53 million, bringing full-year Skytofa revenue to €206 million. This performance reflects the strength and value of the brand. As a reminder, Skytofa is now approved in pediatric growth hormone deficiency in the US and adult growth hormone deficiency. Today, Skytofa has an overall market share of around 7% in the US. During the fourth quarter, we initiated our Phase III basket trial, evaluating transcontinent growth hormone in additional established growth hormone indications, including ISS, shock deficiency, Turner syndrome, and SDA, which compromised up to half of the growth hormone market. Over the long term, This indication represents a meaningful opportunity to expand the role of scartofa as a treatment of choice in additional growth disorder. We also see an opportunity to potentially expand scartofa's use to novel indications where Croftamone has not previously been approved for use, such as acondoplasia in combination with TranscontCMP. Transcon CAP is expected to be the first and only once-weekly treatment for children with achondroplasia, providing the full linear growth outcome that can be achieved with monotherapies addressing the overactive FDRF3 tyrosine kinase. In addition, In our pivotal trial, Transcon-CMP achieved significant improvement in leg bowing compared to placebo, increasing spinal and calen dimension, and a similar safety and solubility profile compared to placebo with a very low rate of injection site reaction and no cases of symptomatic hypertension. In the US, Our NDA for children with achondroplasia remains under review with a PDUFA date of February 28. In the EU, the MAA review is underway following our submission last October with a regulatory decision expected in the fourth quarter of 2026. Recruitment of our ongoing trial in infants with achondroplasia 8 0 to 2 is going well, and we anticipate complete enrollment later this year. Turning to the combination therapy. Our 52-week coach data in acromtoplasia underscore the potential power of dual treatment with transcon-CMP and transcon-clotamol, where continuous exposure to CMP enables the benefit of sustained exposure to unmodified clotamol. In comparison, monotherapy trials of daily growth hormone in agonoplasia delivered only a limited effect on growth and no reported benefit beyond linear growth. Our 52-week data from the Phase II combination trial support our vision to significantly raise the bar for treatment of agonoplasia. with linear growth improvements in air contact pressure specific height score that were three to four times what has been shown with CMP or daily growth hormone monotherapies in the same time period. In addition, the combination trial demonstrated accelerated improvement in body proportionality. And for the first time, and meaningful improvement in arm span has been reported without compromising safety or toolability. Importantly, these benefits beyond height are meaningful to the ACONTOPLACER community and have been a core object of our patient-focused development program in both our monotherapy and combination therapy programs. Importantly, All children completed 52 weeks of treatment and remain in the trial, reinforcing the benefit of treatment and acceptable treatment burden of the once-weekly regime. These Phase II results demonstrate the effect of these complementary therapies, supporting that Transcon-CMP acts in synergy with the growth-promoting effect of Transcon-Growth Tremor. and has positive effect beyond linear growth. We believe over time the standard care in acromioplasia and other growth disorders long term will include dual therapy as a treatment option, building on the potential role of Transcon-CMP as an essential fundamental therapy. We recently held a successful end of phase 2 FDA meeting and scientific advice meeting in EU to align on our phase 3 trial fund. This novel combination approach for treatment children with acondyloplasia. We also remain on track for additional COATS trial updates including week 78 by mid-year and week 104 by year end. and plan to explore further opportunities in other growth disorders. To sustain durable long-term growth for ascendants well into the next decade, we plan to continue to invest in label expansion of our current products in rare endocrine diseases. In addition, we have a strong focus on the development of new blockbusters product opportunities, both inside and outside rare endocrine diseases, to food significant product revenue growth in the future. Looking at our partnerships, Transcon Technologies support a continuous flow of highly differentiated product opportunity across multiple therapeutic areas, more than we can develop and commercialize ourselves. For this reason, our vision 2030 includes a focus on creating additional value through partnership and collaboration. Our collaboration with Novo Nordisk for once-monthly Transcon semi-glutide continues to advance towards the clinic. At Arconis, Transcon NGVDF is on track to enter the clinic this year. In Japan, Japan received approval for UiPath in August 2025 and commercial launched it in November 2025. In addition, recent approval of Skytrover in China in late January 26. In summary, 2025 was another positive and transformative year for Centis. With two commercial Transcon products, continue to scale the potential approval of the third high-value Transcon product in the coming weeks. and a growing pipeline of highly differentiated programs, we believe we have the fundamentals in place to deliver doable long-term growth. A rapidly strengthening financial profile gives us confidence to achieve an expected operating cash flow of around €500 million in 2026 and our aspiration to achieve at least €5 billion in annual product revenue by 2023. all consistent with our Vision 2030 strategy. I will now turn the call over to Scott.