Ascendis Pharma A/S Ordinary Share 2026 Q2 Earnings Call

Please go ahead. Thank you, Operator, and thank you everyone for joining our second quarter 2026 financial results conference call.

I'm Chad Fugere, Vice President, Investor 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 US. 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 their 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 SKYTROFA, YORVIPATH and YUVIWEL, 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 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 the forward-looking statements section of today's press release and the risk factors section of our 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. In the EU, it has received MAA authorization for 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. 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. TransCon CNP is approved in the U.S. by the FDA to increase linear growth in pediatric patients 2 years of age and older with achondroplasia with open epiphases.

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 have 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 regarding product candidates shall be viewed as promotional. On the call today, we will discuss our second quarter 2026 financial results, and we will provide further business updates. Following some prepared remarks, we will 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 TransCon products continued to drive the transformation of Ascendis into a leading global biopharma company. The uniqueness of the TransCon technology platform, our strong development and global commercialization capability, and our values and visions are the fundamentals driving this transformation. We believe the same strength will continue to drive Ascendis growth in the following years. Starting with the long-term durability of our highly differentiated approved protein and peptide-based combination products, SKYTROFA, YORVIPATH, and YUVIWEL.

We believe these products will be the key driver of our growth story for the next 10-15 years through global commercialization, potential for label expansion, including combination treatments, and investment in patient support offerings. The continued expansion of the TransCon technology platform enables us to fulfill our plans to file at least one IND 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 us begin with a more detailed look at YORVIPATH.

YORVIPATH is the first and only approved treatment for adults with hypoparathyroidism that address the underlying disease by replacing the missing endogenous PTH throughout the body. Uptake of YORVIPATH has grown steadily since launch, both in the U.S. 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 U.S., we see consistent new patient demand and continued expansion of global commercialization launches with full reimbursement. YORVIPATH 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 a rare disease product. In the U.S., new patient demand for YORVIPATH in the second quarter has remained robust, consistent with prior quarters.

In addition, physician prescribing is broadening and deepening. Patients who have successfully initiated YORVIPATH treatment continue to stay on therapy, indicating a high level of satisfaction. We continue to be excited by the growth of YORVIPATH in the U.S. and outside the U.S., and to see its continued strong launch performance. Data from our long-term phase II and phase III trials of YORVIPATH presented in the second quarter highlight why YORVIPATH is becoming a standard of care in post-surgical and all subsets of hypoparathyroidism, including ultra-rare genetic causes like DiGeorge syndrome, ADH1, and ADH2. Results showed sustained response rate of 82%-86% for the multicomponent endpoint, with clinical benefit across multiple organ systems, 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 hypoparathyroidism 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 YORVIPATH.

Turning now to YUVIWEL. We believe YUVIWEL is positioned to become the market leader therapy for achondroplasia. Rapid uptake of YUVIWEL is already transforming the U.S. market. Across the board, we see a highly favorable response among patients and physicians to YUVIWEL's differentiator 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 enrollments 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 patients of all kinds of backgrounds, those switching, returning to medical therapy or starting therapy for achondroplasia for the first time.

We believe YUVIWEL is really growing the U.S. market, which is exactly the pattern you would love to see when a highly differentiated product is introduced into an area where there still exists a high unmet medical need. Long-term data for the now completed pivotal ApproaCH trial show durable and consistent improvement in growth, leg bone, body proportionality, along with a general well-tolerated safety profile compared to placebo, underscoring why the community is quickly adopting YUVIWEL. In the U.S. and the E.U., a regulatory decision for YUVIWEL is expected in the fourth quarter of 2026. We are also making YUVIWEL available in select international markets through early access program using the U.S. FDA approval. Longer term, we are pursuing expansion opportunities for TransCon CNP 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 hypochondroplasia, and still continue with geographic expansions. Turning now to combination therapy with TransCon CNP and TransCon growth hormone. The biological rationale for this combination treatment is clear and extremely well founded on science. TransCon CNP is removing the limitation caused by the overactive FGFR3 pathway, so TransCon growth hormone can provide a strong complementary effect. In addition, it has been observed that in achondroplasia there is a partial impairment of the IGF-1 growth hormone axis. This is illustrated by children with achondroplasia have a negative IGF-1 SDS value as shown of the demographic in both our phase II and phase III trial.