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Ascendis Pharma A/S
8/13/2026
Ladies and gentlemen, thank you for standing by. Welcome to the second quarter 2026 Ascendis 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 would need to press star 1 1 on your telephone. You would then hear an automated message advising your hand is raised. We ask that you please limit to one question and return to the queue for additional questions. And to withdraw your question, please press star 11 again. Please be advised that today's conference is being recorded. I would like now to turn the conference over to Chad Fugure, Vice President of Investor Relations. Please go ahead.
Thank you, Operator, and thank you, everyone, for joining our second quarter 2026 Financial Results Conference Call. I'm Chad Fugure, Vice President, Ambassador Relations at Ascendis Pharma. Joining me on the call today are Jan Mikkelsen, President and Chief Executive Officer, Scott Smith, Chief Financial Officer, Sherrie Glass, Chief Business Officer, and Jay Wu, Executive Vice President and President, Ascendis U.S. Before we begin, I'd like to remind you that this conference call, including the Q&A session that follows our prepared remarks, will contain forward-looking statements that are intended to be covered under the safe harbor provided by the Private Securities Litigation Reform Act. All statements made on this call, other than the statements of historical fact, are forward-looking statements. Examples of such statements may include, but are not limited to, statements regarding our commercialization and continued development of Skytropha, URBPath, and UBWell, including label expansion and combination treatment, certain expectations regarding patient access and financial outcomes, our pipeline candidates and our expectation 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 results of regulatory decisions, and our financial outlook and Vision 2030 objectives. 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 under reliance on these statements. We assume no obligation to update these statements if circumstances change, except as required by law. For additional information concerning the factors that could cause actual results to differ materially, please see the forward-looking statements section of today's press release and the risk factors section for annual report on Form 20-F filed with the SEC on February 11, 2026. In addition, during this call, we will refer to certain non-IFRS financial measures These measures are not prepared in accordance with IFRS accounting standards and should not be considered in isolation from or as a substitute for our IFRS results. A reconciliation of each non-IFRS measure to the most directly comparable IFRS measure together with an explanation of why management believe these measures are useful to investors is included in today's press release. 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 in the EU has received MAA authorization for the European Commission for the treatment of pediatric growth hormone deficiency. Transcon PTH is approved in the US by the FDA for the treatment of hypoparathyroidism in adults and the European Commission in the United Kingdom's medicines and healthcare products Regulatory Agency have granted marketing authorization for Transcon PTH replacement therapy indicated for the treatment of adults with chronic hypoparathyroidism. Transcon CMP is approved in the U.S. by the FDA to increase linear growth in pediatric patients two years of age and older with achondroplasia with open PPCs. Continued approval for this indication, which was based on an improvement of annualized growth velocity, may be contingent upon verification and description of clinical benefit in confirmatory trials. Other than the approved products I've just described, 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 report product candidates shall be viewed as promotional. On the call today, we'll discuss our second quarter 2026 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 day, everyone. During the second quarter, achievement of important milestones and strong demand for our Transcom products continue to drive the transformation of Ascentis into a leading global biopharma company. The uniqueness of the Transcon technology platform, our strong development and global commercialization capability, and our values of vision are the fundamentals driving this transformation. We believe the same strength will continue to drive Ascendi's growth in the following years. Starting with the long-term durability of our highly differentiated approved protein and peptide based combination products, Skytofa, Neuropaths, and Neuowell. We believe these products will be the key driver of our growth story for the next 10 to 15 years to global commercialization, potential for label expansion, including combination and investment in patient support offerings. The continued expansion of the Transcon technology platform enables us to fulfill our plans by at least one IMD or similarly yearly, each based on a new NCE, laying the foundation for strong growth for many decades. This will also enable us to establish new therapeutic areas in addition to hypopara and growth disorders. As a further upside, our established partners are advancing TransCon candidates in large indications. This is why we believe Ascendis is well positioned for self-sustained long-term growth. Let's begin with a more detailed look at UroPath. UroPath is the first and only approved treatment for adults with hypopara that addressed the underlying disease by replacing the missing endogenous PTAs throughout the body. Uptake of neuropaths has grown steadily since launch, both in the US and many other countries, reflecting the significant unmet medical need among the more than 800,000 patients living with this serious rare disease in the geographic region covered by our global commercial infrastructure. Outside of the US, we see consistent new patient demand and continued expansion of global commercialization branches with full reimbursement. UiPath is now available commercially or through named patient programs in more than 35 countries. This illustrates the strength of our ability to execute a rapid, broad, global launch of the rare disease product. In the US, new patient demand for UiPET in the second quarter has remained robust. Consist with prior quarters. In addition, physician prescribing is broadening and deepening. Patients who have successfully We continue to be excited by the growth of UiPath in the U.S. and outside the U.S. and to see its continued strong lung performance. Data from our long-term Phase II and Phase III trials of UiPath Presented in the second quarter, Highlight by Europass is becoming a standard of care in post-surgery and all subsets of hyperparameters, including ultra-rare genetic causes like discharge, ADS1, and ADS2. Results show sustained response rate of 82% to 86% for for the multi-component endpoint with clinical benefit across multiple organ system, CNS, kidney, small intestine and bone, plus meaningful improvement in quality of life. Patient retention as high as 95% after five years of treatment. Pretty unique. In parallel, We are working to further advance our leadership in hyperfarm with additional clinical trials that include expanding the label to include the age from 12 to 18 years and in the U.S. higher doses for patients and developing a once weekly product for the patient that is on stable doses of UroPads. Turning now to UofL. We believe UofL is positioned to become the market leader therapy for acondalplasia. Rapid uptake of UofL is already transforming the U.S. market. Across the board, we see a highly favorable response among patients and physicians to UofL's differentiated profile. In the U.S., through June 30, We had more than 170 unique patients enrolled. Since then, uptake has continued with more than 220 enrollment and more than 65% approved for reimbursement in the U.S. through the end of July. Really a unique launch. The rapid uptake is by patient of all kinds of background, those switching, returning to medical therapy, or starting therapy for air contemplation for the first time. We believe UofL is really growing the US market, which is exactly the pattern you would love to see when a highly differentiated product is introduced into air where there still exists a high unmet medical need. Long-term data for the now-complete people's approach trial show durable and consistent improvement in growth, leg bone, body proportionality, along with a generally well-tolerated safety profile, compared to placebo, underscoring why the community is quickly adapting to the overall. In the US, in the EU, a regulatory system for UofL is expected in the fourth quarter of 2026. We are also making UofL available in select international markets through early access programs using the US FDA approval. Longer term, we are pursuing expansion opportunities for Transcon-CMP to ongoing and planned trials. These include Ongoing activities such as infants 0 to less than 2 years of age and we recently announced completion of this target enrollment faster than expected. Adults with achondroplasia, children with hyperchondroplasia and still continue with graphic expansions. Turning now to combination therapy with Transcon-CMP and Transcon-Glutamol. The biological rationale for this combination treatment is clear and extremely well founded on science. Transcon-CMP is removing the limitation caused by the overactive FDR3 pathway, so Transcon-Globin can provide a strong complementary effect. In addition, it has been observed Thank you. Thank you. Thank you. Sustained Transformative Analyzed Growth Velocity, and ATA's HIGH score, including improvement in body proportionality. Based on this result, we believe this unique combination of once-weekly transcon-based therapies will transform the treatment of acondoplasia and other indications over time. Our recent week 78 COAST TRIED data. show sustained efficacy over 78 weeks with no compromises to safety and controllability. This points to the potential for this novel combination to establish a new treatment standard in naturalization. The Phase III combination trial in children with ankyloplasia will begin enrolling later this year. Turning to Skytropha. The once-weekly growth hormone treatment built on the mode of action of unmodified somatopoeia. With indications for pediatric and adult growth hormone deficiency, we continue to be the number one long-acting growth hormone by brand value in the US. We are extremely proud that Skytober recently achieved more than 20,000 unique enrollments This illustrates the strength of our capabilities from supply chain, commercial infrastructure, and on market support to benefit such a large number of rare disease patients. And we are working to make Transcon-Glutamone available to more patients through label and geographic expansions. To support label expansion going that described in our account of pleasure program, We are conducting the phase 3 basket trial investigating Transcon growth hormone in ISS, SDA and Turner syndrome. As an integrated part of our global growth disorder strategy, we expect to launch Transcon growth hormone in the same countries where we also expect to launch Transcon CMP. Turning now to our partnership. In metabolic disorders and obesity, our once-monthly Transcon semi-glutide program with Norvo Nordisk continues to advance. In ophthalmology, our partner Onconis recently initiated a first-in-human clinical trial of the anti-VDF treatment built on the Transcon technology in patients with BED-AMD. In closing, by always putting patients first, Ascendis has delivered three highly differentiated leading Transcon-based products, UroPath, Urovel, and Skytropa. We are on track to achieve our Vision 2030 objective of being a leading global pharma, building on a strong foundation for the future. With that, I will turn the call over to Scott to review our financial results and some additional comments.
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