Ascendis Pharma A/S Q2 2026 Earnings Call Transcript

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Provided by Quartr
August 13, 2026

Key Takeaways

  • Positive Sentiment: Q2 revenue more than doubled year over year to €339 million, including €315 million of product revenue and a €17 million TransCon CNP milestone. Management also reported €207 million of net profit and €812 million in cash and cash equivalents.
  • Positive Sentiment: YORVIPATH demand remained robust, with approximately 1,000 new U.S. patient enrollments per quarter and availability commercially or through named-patient programs in more than 35 countries. Long-term data showed sustained efficacy, quality-of-life improvements, and patient retention of up to 95% after five years.
  • Positive Sentiment: YUVIWEL uptake is accelerating, with more than 220 U.S. enrollments and over 65% approved for reimbursement by the end of July. Management believes the product is expanding the achondroplasia treatment market and could become the leading therapy, supported by planned global launches and a phase III combination trial with TransCon growth hormone.
  • Positive Sentiment: Management reiterated confidence in its long-term growth targets, including more than €500 million of operating cash flow in 2026 and €5 billion of revenue by 2030, while expecting increased operating leverage as launches scale.
  • Negative Sentiment: YUVIWEL remains subject to an ongoing U.S. intellectual-property dispute with BioMarin, with an initial ITC opinion expected in August, a broader ITC decision in December, and a potential presidential review thereafter. Management expects no material impact, but the legal process remains a source of uncertainty.
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Earnings Conference Call
Ascendis Pharma A/S Q2 2026
00:00 / 00:00

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Operator

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 will need to press star one one on your telephone. You will 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. To withdraw your question, please press star one one again. Please be advised that today's conference is being recorded. I would like now to turn the conference over to Chad Fugere, Vice President of Investor Relations. Please go ahead.

Chad Fugere
Chad Fugere
VP of Investor Relations at Ascendis Pharma

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 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 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.

Chad Fugere
Chad Fugere
VP of Investor Relations at Ascendis Pharma

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.

Chad Fugere
Chad Fugere
VP of Investor Relations at Ascendis Pharma

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.

Chad Fugere
Chad Fugere
VP of Investor Relations at Ascendis Pharma

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 two years of age and older with achondroplasia with open epiphases.

Chad Fugere
Chad Fugere
VP of Investor Relations at Ascendis Pharma

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.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

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.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

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.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

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.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

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.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

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.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

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.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

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.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

In our COACH clinical trial for children with achondroplasia, this unique combination has demonstrated sustained transformative analyzed growth velocity and age-adjusted height 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 achondroplasia and other indications over time. Our recent week 78 COACH trial data show sustained efficacy over 78 weeks with no compromises to safety and tolerability. This point to the potential for this novel combination to establish a new treatment standard in achondroplasia. The phase III combination trial in children with achondroplasia will begin enrolling later this year. Turning to SKYTROFA, the once-weekly growth hormone treatment built on the mode of action on unmodified somatropin. 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 U.S.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

We are extremely proud that SKYTROFA 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. We are working to make TransCon growth hormone available to more patients to label and geographic expansions. To support label expansion drawing that described in our achondroplasia program, we are conducting the phase III basket trial investigating TransCon growth hormone in ISS, SGA, 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 CNP. Turning now to our partnership. In metabolic disorders and obesity, our once-monthly TransCon semaglutide program with Novo Nordisk continue to involve events.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

In ophthalmology, our partner, Eyconis, recently initiated a first in human clinical trial of the anti-VEGF treatment built on the TransCon technology in patients with wet AMD. In closing, by always putting patient first, Ascendis has delivered three highly differentiated leading TransCon-based product, YORVIPATH, YUVIWEL, and SKYTROFA. We are on track to achieve our Vision 2030 objective of being a leading global biopharma, 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.

Scott Smith
CFO at Ascendis Pharma

Thanks so much, Jan. Good afternoon, everyone. I will touch on some key points surrounding our second quarter financial results. For further details, please refer to our Form 6-K filed today. Total product revenue was EUR 315 million, more than doubling year over year. Total revenue for Q2 2026 was EUR 339 million, which included non-product collaboration revenue of EUR 24 million, which further included a EUR 17 million milestone related to TransCon CNP. YORVIPATH revenue was EUR 252 million in Q2, reflecting consistent new patient demand in the U.S. and continued growth outside of the U.S., reaching blockbuster status on a run rate basis in the second year of launch in the U.S. SKYTROFA contributed EUR 55 million in Q2, which reflects increased demand in the U.S. and includes product sales to a collaboration partner.

Scott Smith
CFO at Ascendis Pharma

UBEGLA was commercially launched in the U.S. during Q2 and generated EUR 8 million in revenue in its first quarter on the market, reflecting strong demand and rapid conversion to paid therapy with limited stocking. Continuing to expenses, R&D expenses in Q2 were EUR 76 million, up from EUR 59 million in Q1, reflecting continued investment in our pipeline and innovation. Recall Q1 included a favorable EUR 11 million reversal of prior period write-downs of TransCon CNP pre-launch inventories. SG&A expenses were EUR 173 million in Q2 compared to EUR 145 million in Q1, reflecting additional investments in the commercial launches of YORVIPATH and UBEGLA to accelerate growth for the long term. Operating profit of EUR 220 million in Q2 included EUR 158 million of other operating income related to the sale of the PRV. Non-IFRS operating profit was EUR 92 million, and non-IFRS operating margin was 27%.

Scott Smith
CFO at Ascendis Pharma

Refer to our press release for details. For Q2 2026, net profit was EUR 207 million and non-IFRS net profit was EUR 61 million. We ended Q2 2026 with EUR 812 million in cash and cash equivalents, which includes the use of EUR 56 million in Q2 for our previously announced share repurchase program, including the net settlement of certain RSUs. Following the settlement of our convertible notes, we have no bank debt, no convertible debt, and EUR 1.4 billion of equity. Turning to our outlook for the rest of 2026. For YORVIPATH, we expect growth and performance consistent with prior quarters. For SKYTROFA, we expect relatively stable revenue in the U.S. For UBEGLA, we are encouraged by the early demand trends.

Scott Smith
CFO at Ascendis Pharma

We believe it is expanding the market and is on pace to be the leading achondroplasia therapy in the U.S., reflecting the large unmet medical need and the highly differentiated profile of UBEGLA. Our Q2 performance reinforces our belief that we can achieve EUR 5 billion in revenues in 2030. With our existing portfolio and our TransCon technology as a strong foundation, we believe we are well-positioned to grow revenue to more than EUR 10 billion in the next decades while developing and launching new TransCon products with blockbuster potential. We expect significant operating leverage as revenue scales through the balance of the year while maintaining new investments in global product launches and patient access to reach as many patients as possible and support our long-term revenue aspirations. Even with these investments, we expect to generate more than EUR 500 million in cash flow from operating activities this year.

Scott Smith
CFO at Ascendis Pharma

With that operator, we are now ready to take questions.

Operator

Thank you. As a reminder, to ask a question, please press star one one on your telephone and wait for your name to be announced. To withdraw your question, please press star one one again. We ask you please limit to one question and return to the queue for additional questions. Our first question is going to come from Jessica Fye with JPMorgan. Your line's open.

Jessica Fye
Jessica Fye
Analyst at JPMorgan

Hey, guys. Good morning. Thanks for taking my question. On that outlook for at least EUR 500 million of operating cash flow this year, I think you gave that in the beginning of the year prior to the PRV sale, and I was just wondering if you are able to update your cash flow expectations for the year. I know it is sort of like a greater than is unbounded, but curious if anything more you can add there. On that comment that SKYTROFA seems to be expanding the market, is it possible to estimate how much of these patient enrollments are coming from market expansion? Thank you.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

Thanks, Jess, for the questions. The other happy person besides me, Scott. Scott got the opportunity to be the first one answering questions. Please, Scott.

Scott Smith
CFO at Ascendis Pharma

Yeah. With respect to our cash flow guidance, just to be clear, greater than EUR 500 million. I thought I heard you say EUR 100 million, so greater than EUR 500 million. At this time, we don't want to bound the upper side because we're initial into the launch of YUVIWEL. That's euro, by the way, EUR 500 million, Jan likes to point out.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

Just related to the question, it comes back to what we communicated last time we had this call, that we don't have really the insight in exactly the distribution of where the patients are coming from. Our general feelings and how we see it is that with such a strong demand, we have a really strong belief that it's not only coming from switches, it's most also coming from either patient that had stopped therapy or new patients that basically are coming to a situation because of the highly differentiated nature of YUVIWEL that they want to start therapy. I think this is where we have this strong belief that we see an expansion of the market.

Jessica Fye
Jessica Fye
Analyst at JPMorgan

Thank you.

Operator

Thank you. The next question is going to come from Tazeen Ahmad with Bank of America. Your line's open.

Tazeen Ahmad
Tazeen Ahmad
Analyst at Bank of America

Hey, good morning. Thanks for taking my question. Jan, I wanted to get your thoughts about the IP challenge on YUVIWEL. We know obviously what the blue sky scenario is for Ascendis, and most of the scenarios look positive, but can you just maybe walk us through what the potential outcomes are? This is for a patent that expires obviously in 2030, and so between now and then, can you just tell us what could happen and what the potential for payments that Ascendis would need to make in the worst case scenario could be? Thanks.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

Thanks, Tazeen, for the question, and it basically is a question that is addressing the ongoing legal, I would call it battle, between Ascendis and BioMarin. Let me just come back to some facts. The facts is that this patent that we discussion got complete invalid in Europe, so we never really come to a discussion if we were infringing and anything like that. So when we see the situation outside U.S., we got the patent invalid immediately to the patent system in Europe. In the U.S., we never managed to come into the patent system because BioMarin selected to go to the ITC case, which are a system which we can easily say traditional never have really dealt with a lot of cases that dealing with branded pharmaceutical.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

In the ITC case, there will be a first opinion from a single judge, and he will come with an opinion here in August, and then there will be a more next time will be in December. There will be an opinion from the ITC, and then later on, there will be a potential confirmation of the ITC decision two to three months after to a presidential order. So you can see we are not getting any clarification in August in one way or the other way, even if it's possible for one company and negative for the other one, it's not really any kind of decision where it's going to be ending. After the first initial opinion from a single judge, because the ITC case will be taken to a decision for, I cannot remember how many judges that will be part of that decision.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

There is a huge opportunity to provide what we call public interest for this product. When we see the public interest, the element of how this product opportunities are really being serving an unmet medical need in the U.S. market with this rapid uptake of patients, it's really, really clear that is a huge public interest to keep that. Just recall, I cannot remember one single case in the U.S. where a branded product that provides a benefit to U.S. patient has been denied. But you can see we just in a case where it's only is a U.S., it has been cleared ex-U.S. So whatever it happened, it will not have any material impact on Ascendis pathway. I can guarantee that. People take it up as a life and death for Ascendis.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

This is a total not taken into the perspective what it mean for Ascendis. Out from that, I see it's not really is a mature element for Ascendis to be a leading biopharma and hit the EUR 5 billion in 2030.

Operator

Thank you. Our next question will come from Gavin Clark-Gartner with Evercore. Your line's open.

Gavin Clark-Gartner
Gavin Clark-Gartner
Analyst at Evercore

Hey, guys. Thanks for taking the question. I actually just wanted to ask on the earlier pipeline. You noted in your prepared remarks that TransCon platform can fuel

Gavin Clark-Gartner
Gavin Clark-Gartner
Analyst at Evercore

One IND for an NCE annually. I guess there hasn't been one yet this year. Should we expect one in the near term? What exactly are the go-forward plans for the earlier pipeline? Thank you.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

It was because I in some way felt that the two product opportunities that we have developed to our partnership built on the TransCon technology will still consider at a NCE, the one that now in clinic with Ascendis and the other one we expect to go into the clinic now with Novo Nordisk. It's still some way being developed to the TransCon technology funnel. Kevin, perhaps I shouldn't have done that, but I still believe, I feel some kind of little bit ownership on these two product opportunities. At least we have major upside in both of them. So out from that perspective, I still consider we potentially will have this year two new chemical entities being entered into clinical trials.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

I think Kenneth and his team and anyone else, they are working very hard on that will be at least one of these new chemical entities coming into every year now. I'm really proud about that. But it's also addressing the sustainability of Ascendis independent of going out and buying something no one else want to have. I think this is where we really feel extremely pleasant by the situation, by being a fundamental company that building on a strong technology platform that provide both sustainability for ourselves, but also a continued flow of potential partner licensing.

Gavin Clark-Gartner
Gavin Clark-Gartner
Analyst at Evercore

Great. Thank you.

Operator

Thank you. The next question will come from Yaron Werber with TD Cowen. Your line's open.

Yaron Werber
Yaron Werber
Analyst at TD Cowen

Great. Thanks so much. Question on YUVIWEL. Do you expect that there is some seasonality in terms of new patient starts in the summer as kids are going on vacation? We are getting a lot of questions on sort of the 60 patient start forms in April, and now you are sort of at 220. It sounds like there is like 50 per month now. Is that sort of sustainable from now on? It sounds like you are planning, you think you could be the number one brand by the end of the year. BioMarin, we think, has about 750 patients on drug. Are you thinking in the U.S., are you kind of referring to getting to a higher number than that by, let us say, late February? Thank you.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

Thanks for the question. I actually do not think Ascendis has really made some clear forward-looking statement related to how we see YUVIWEL being accelerating and expanding the market in a quantitative manner. I do not think we have come with any kind of indication related to that. I have no doubt it will do it, but it is not the same thing that we are going to quantify it currently. I think after basically only four months in the market, I feel really not prepared to come with clear guidance to it before we have more quarters really into our, you can say, analytical system where we basically can look on trends and other things like that.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

But one person that really can give you a good feedback, now we talk about the U.S. market, is Jay, and he is extremely enthusiastic about what he is seeing, and you can give the latest way what you see how the market will develop.

Jay Wu
Jay Wu
EVP and President of U.S. Market at Ascendis Pharma

Thanks, Jan. As Jan mentioned before, four months in, we are not prepared to give longer term guidance, but what we can say is we are incredibly encouraged by what we are seeing to date. When you look at some of the fundamentals behind the YUVIWEL uptake, whether it is prescriber reach, we talk a lot a bit before around this space. There are quite a few centers of excellences. We are seeing 80% of them, nearly 80% already in a short four-month period, already prescribe YUVIWEL to their patients. So even in early days, we are seeing a lot of enthusiasm from providers around the clinical profile of this product. I think even more importantly, when you look at the patient enthusiasm, I think you can see in early days, we are seeing a very positive trajectory.

Jay Wu
Jay Wu
EVP and President of U.S. Market at Ascendis Pharma

While we don't explicitly collect information on what therapy or non-therapy a patient is coming from, and again, that's driven largely by the fact that we have a broad label, so we don't need that information in order to ensure that this patient can get on therapy. This is rare disease, so qualitatively, we have heard confirmed anecdotes across all three categories for which our patients are coming from. And those three categories again are, one, patients that are switching from current therapy. Two, patients that have previously discontinued pharmacological therapy and has now wanted to return to pharmacological treatment. Then third, a group of patients that historically have set out and have said based on the clinical profile of YUVIWEL, they now want to try a therapeutic option for the first time.

Jay Wu
Jay Wu
EVP and President of U.S. Market at Ascendis Pharma

All that again to underscore there is existing unmet need here, and because of our profile, we're definitely seeing that patients are coming out of the woodwork from growing the market standpoint, and we're just getting started.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

Just to summary to add on to Jay's excellent comments. Ultimately I have no doubt we will be number one in the achondroplasia space. Ultimately, we will expand the market because of the unmet medical need, and that is just with the monotherapy. When you look at how commitment we have to this area, where we now are making a complete new standard with the combined treatment. I believe we are dedicated to be not number one in the first year, but continue to build for the next 5-10 years with monotherapy combination integrated treatment regimes. I believe with our once weekly TransCon product built on Voluma and CNP, we are extremely well-positioned really to be the leader in this segment.

Operator

Thank you. Our next question will come from Derek Archila with Wells Fargo. Your line's open.

Derek Archila
Derek Archila
Analyst at Wells Fargo

Hey, good morning, and thanks for taking the questions. Congrats on the progress. Scott, I just wanted you to clarify a comment on your YORVIPATH growth for the rest of the year. I think you said it is going to be like prior quarters. I guess, which quarters are you referring? Because I think the quarter-over-quarter growth in 1Q was negatively impacted. Saw some catch up here in the second quarter. So maybe you can just clarify which quarters you are referring to. Thanks.

Scott Smith
CFO at Ascendis Pharma

Yeah, Derek, thanks for the question. I think that two points. One is the consistent performance with the KPIs that we have given you, for example, with enrollments. We expect those to continue and be consistent. The other would be, and you could refer to our prior quarters and maybe Chad can point to prior comments. But I think that now that we have seen a full year, you know the various trends that will come into play related to Q3 and Q4 and then Q1 next year. So we think actually folks did a pretty good job modeling out Q2, and now you have all the information you need to model the rest of the year going forward until we update basically the KPIs.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

Just to give you some kind of what is our value in this here. The value for us, we want to give you not so you basic getting a lower number, so we look like heroes. We want to give you the number, so you are right nearly every time. I think this is a way we try to come up with our different mathematic algorithm and how we see it and give you all the information for you really to be right in this manner. I think this is a way we like to be extremely transparent with everything, what we perform. So we quite sure that you basically can go out and really somewhere feeling always comfort with the guidance we give you.

Derek Archila
Derek Archila
Analyst at Wells Fargo

Great. Thank you.

Operator

Thank you. Our next question will come from Joseph Schwartz with Leerink. Your line's open.

Joseph Schwartz
Joseph Schwartz
Analyst at Leerink

Hi. Congrats on all the progress. Thanks for taking my question. As you embark on a phase III in hypochondroplasia, I wanted to ask how you are defining the enrolled population. How large do you see the diagnosed treatable pool of hypochondroplasia patients who are not already being treated in some cases if they are at the more severe end versus achondroplasia? Thank you.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

This is a very interesting question because it is actually someway going into the situation on how we basic are to genetic testing, taking a big patient group that was in old days were called ISS, idiopathic knee. We have no clue what is the underlying diseases. Then you go out and do more genetic testing. When you find a mutation in the FGFR3 receptor, and you find it in the right regions, then you suddenly are not an ISS patient, but then you are a hypochondroplasia patient, even if you do not have, you could say, the phenotype of looking like an achondroplasia patient or a hypochondroplasia that we saw for 10 years ago.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

Therefore, you can see the ISS population is somewhere getting smaller and smaller because of the genetic testing is basically going out and giving them an underlying reason why you potentially have a short status without potentially have the other element that you see for the phenotype of that. This is where you can think when you go into ISS, are you defined it from a genetic perspective, or you define it for a phenotype or anything like that? We are in a situation where when you see the clinical trial, how we are doing it, you will basically see that is one of the pathway we have selected.

Joseph Schwartz
Joseph Schwartz
Analyst at Leerink

Thank you.

Operator

Thank you. Our next question will come from Daniel Bronder with Cantor. Your line's open.

Daniel Bronder
Daniel Bronder
Analyst at Cantor

Hey, team. Congrats on the quarter. I am on for Lee Waczak. We were just wondering if you could give us a little more color on the quality-of-life metrics in the COACH trial. You already alluded to the body segment ratios, but how should we think about benefit on arm span and other metrics?

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

Just to recall, the COACH trial is the combination trial where we combining the two TransCon-based product, our TransCon growth hormone and TransCon CNP. I have to say, when I look on an element like arm span, we already reported some of the data. We have reported the 52 weeks data, and if you cannot find that deck, I can send it to you, or Scott can send it, or Chad can send it. We have so many IR people, I do not know their names anymore. From that perspective, it already came out. I have to say, it was one of the, I will say, extremely positive surprises I saw because when we looked on monotherapy, but either a CNP-based one or a growth hormone-based one, we did not saw the expected hopeful development that we could hope for.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

But we definitely saw it when we look on the combination therapy. Then you can ask me, what is the scientific reason why you see it much more influenced benefit by the combination therapy? I have to say, I don't know. But what we saw was an arm span that really gave us this hope with the combination therapy. You basically will be in a position that you basically could avoid all kind of limb elongation surgeries in achondroplasia, both related to both legs and arms by that. It is the slide number 5 as I remember it. What we see, Scott, read up.

Scott Smith
CFO at Ascendis Pharma

The unprecedented improvements in the arm span with a combination were +9.4 cm with a TransCon CNP naive cohort, and 7.9 cm with a TransCon CNP treated cohort.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

It was really an-

Scott Smith
CFO at Ascendis Pharma

Compared to limb lengthening surgery, centimeters gain.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

Exactly. I have to say, it was one of the days where I felt it was worth to go to a job and really can see the benefit of what we're doing.

Daniel Bronder
Daniel Bronder
Analyst at Cantor

Okay. Thank you.

Operator

Thank you. Our next question will come from Yun Zhong with Wedbush. Your line is open.

Yun Zhong
Yun Zhong
Analyst at Wedbush

Hi. Excuse me. Good morning. Thank you very much for taking the question. I wanted to confirm that you have not provided prescription number for YORVIPATH in case I missed anything. I know that you said the patient demand remain robust in the quarter, so I wonder if there is any additional quantitative information that you can provide. Going forward, are you going to provide that number in the coming quarters? I think you had this question before at the beginning of the launch. When do you expect that you will feel comfortable providing a guidance in terms of the sales range on actual revenue? Thank you very much.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

You are right, and I think it's starting to be a little bit repetitive every quarter come out and saying that we have about more than 1,000 patients being unique enrolled per quarter. We have continued that message that we see steady state, steady state, and steady state. We said in last year that we will stop coming with this because it was too repetitive. Then because people doubted for the Q1, then we also come out with the Q1, and it was the same number again. What we're writing is that we see a robust steady state enrollment of unique new patients, and here we are referring to the U.S. with about 1,000 new patients every quarter. We don't believe really. Now we went over to Europe. Now we're starting to give you a unique prescription enrollment of Europe instead.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

We always rather have one product opportunity where you will have something to play with numbers and everything like that. Scott, you have some additional comments for the last one?

Scott Smith
CFO at Ascendis Pharma

Yeah. I think our comments were directed to assume that the metrics that we've given you are consistent because Jan wants to make our script shorter, so we don't want to repeat them more, and you should just assume that until we change it.

Yun Zhong
Yun Zhong
Analyst at Wedbush

Great. Thank you.

Operator

Thank you. The next question comes from Alex Thompson with Stifel. Your line's open.

Alex Thompson
Alex Thompson
Analyst at Stifel

Great. Thanks for taking the question. I appreciate the color you provided to Tazeen's question around the ongoing legal battle with BioMarin. I guess as we think about potential scenarios here, and again, acknowledging this idea around public interest of the product and unmet need, do you see a settlement as a reasonable scenario to think about, or is that really not something that you think is reasonable? Thank you.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

Alex, I think I'm a very flexible person, and one of the things I really want to do, I will always do what is best for patients.

Operator

Thank you. The next question will come from Maxwell Skor with Morgan Stanley. Your line is open.

Maxwell Skor
Maxwell Skor
Analyst at Morgan Stanley

Great. Thank you very much for taking my question. Just a quick one on your durability. I was just wondering if dropouts are still mostly during the titration phase, and if you can comment at all on how re-auths are trending. Thanks.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

I think you are 100% correct. When we see a patient being successful coming into a treatment with YORVIPATH, coming over titration part, and being into the treatment after that, we see extremely low dropout. I think that illustrates one thing, the patient satisfaction with this treatment. Because now often being asked, what can we do more for these patients in the therapeutic treatment? When I see the satisfaction that is in this way, then I feel that there is an extremely good physician retention and everything which really show that. We are still developing once weekly for patient on stable doses, just to give patient the choice if they want to do it in this way. We will look at other ways to improve their life, like for example, at-home caption monitoring and anything like that.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

We can help the patient like it happening in Type 1 diabetes and other things like that. Now you are addressing the element where we saying is, we developed this here with a once weekly profile, even if we could make it, sorry, once daily, because we wanted to do the titration most easily, because it is really complex to take patient off of conventional therapy. At the same time, you increase the PTH in replacement therapy. There was a why we made it as a once daily, is really to facilitate the best possible titration. Still, we know it can be problematic for some patients. Jay can try to explain what we now doing to basic handhold the patient in this period. We also can make that extremely successful.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

When you get a prescription, we know everything will be much more successful for the patient, not just after they are really being stable in the titration. Jay, will you explain of the effort you are building in to really to get that to be as soft as possible?

Jay Wu
Jay Wu
EVP and President of U.S. Market at Ascendis Pharma

Absolutely. Can chat a little bit more about certainly the investments that we are making and also to answer your questions around drop-off and re-auths. Yes. As we have shared before, the majority of the drop-offs is during that titration period in terms of when patients experience the most amount of change and where additional education and a higher touch support model makes sense. For re-auths, that is actually pretty routine for us, so there really is not much there in terms of it being a measurable effect on any kind of ongoing patient support. We have patients re-offing throughout the year, and it is just part of our day-to-day operations. From an investment standpoint, we have invested heavily in patient-facing roles for which we have deemed our patient access liaisons. They support patients both pre-prescription as well as through the prescription process and post.

Jay Wu
Jay Wu
EVP and President of U.S. Market at Ascendis Pharma

Essentially, we have seen a lot of success in early days with this field team being able to engage with this patient community. They have appreciated this high level of support, and we of course, support them throughout the journey to ensure that we are optimizing for patient experience.

Maxwell Skor
Maxwell Skor
Analyst at Morgan Stanley

Great. Thank you very much.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

One thing that is in a minute. Now we focus on mostly U.S., but there is still a world outside U.S. Outside U.S., we have not seen the same level of dropout in this phase. It looked like the interaction is pretty well established between the physician and the patients and support system, really to see it without this kind of dropout. So it is basically is a U.S. issue, and Jay. Therefore, we know we can get it to function. We just need to ensure that the support system also in U.S. is strong enough to be sure that is not a problem.

Maxwell Skor
Maxwell Skor
Analyst at Morgan Stanley

Very helpful. Thank you.

Operator

Thank you. Our next question will come from Eric Joseph with Citi. Your line's open.

Eric Joseph
Eric Joseph
Analyst at Citi

Hey. Thanks for taking the questions. As far as your named patient programs or your early access programs, can you elaborate a little bit on which markets you are active in, whether eligibility might be determined by treatment status of a patient, and just generally how we should think about whether named patient programs could be meaningful contributors to patient volumes this year? Thanks. For YUVIWEL in particular.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

Okay. I just wanted to ask what product you were referring to.

Eric Joseph
Eric Joseph
Analyst at Citi

UBEGLA.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

Yeah. I can guarantee that as we based in our prepared remark, try to put emphasis on, we have a global infrastructure in commercialization and patient support, product supply, and everything like that. The number of so we are not a company that just need to get started. We already have established all this infrastructure. And what we are doing is that we are utilizing this established infrastructure based that got established because of YORVIPATH, because this is what we did with YORVIPATH. We are using exactly the same infrastructure also for YUVIWEL. So we will be where patient is, and we will be quite sure we will also serve the patient outside U.S., and potentially the market is much larger outside U.S. And I think, we hope we also will see a large penetration in the U.S. where another short-acting product really failed to do it.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

We believe because of the highly differentiated nature of YUVIWEL, we will see a complete different pickup in the U.S. But it is definitely, we have a strong focus on the ex-U.S. And we will give you some guidance when we come later in the year, so you can give also building up a model for the ex-U.S.

Eric Joseph
Eric Joseph
Analyst at Citi

Excellent. Thanks for taking the question.

Operator

Thank you. The next question will come from Luca Issi with RBC Capital Markets. Your line is open.

Analyst at RBCM

Great. Thanks so much for taking our question. This is Cathy for Luca Issi. Circling back to YUVIWEL, Jay Wu, the three categories that you very nicely touched on for the naive switch and discontinued patients that are not on script. BioMarin mentioned on their second quarter call that less than 100 patients have switched off of VOXZOGO. The simple math that we are trying to do here is that it leaves you with about 70 patients in the second quarter who are naive or return to treatment. That is taken off the switched patients. Does that align with the numbers or impression that you have, and how does the dynamic look like between the truly naive patients and the patients who were once on VOXZOGO, stopped treatment and are now returning to treatment, but to YUVIWEL?

Analyst at RBCM

Separately, very quickly, if you have commented or not on the ex-U.S. strategy for YUVIWEL, given the decision is pending and may come very soon this year. Thanks so much.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

I like your way of doing all the calculation, anything like that. I cannot support it, or I cannot deny it because I do not have the factual insight to some way to confirm anything of the numbers. I also saw the number that came out, but I cannot really support it because I do not have the insight from our own numbers to really come out and come with any statement that indicate if I am aligned or not aligned with. Related to the ex-U.S., for me to understand your question, was this reflecting what is the limitation in the ex-U.S., or what was the question?

Analyst at RBCM

Oh, thanks. Thanks for asking to clarify. More about are you committed to running the show by yourself or you are considering partnering, given that 70% of the VOXZOGO sales is historically coming from ex-U.S. can be a quite heavy lifting.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

Yeah, but basically in the ex-U.S., we have our direct market which are, I think, 60, 70, 80 where we have our own commercial infrastructure. It's pretty clear what we are doing there. Then we have our sales and distributions agreement, and this is, I think it's 70 countries or something like that where it's got this-

Eric Joseph
Eric Joseph
Analyst at Citi

80.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

Oh, 80 countries that is covering this sales and distributions agreement. The vast majority of all of them are all three product. That is already established infrastructure for the distribution. Then we have the two other, the third model where we have our partnerships, one in Japan, one in China, and they also have all the three product. We do not need to go out and make any new agreements for anything. Everything is established. Everything is running on full speed. For some of the EU direct market, we are just waiting for our expected approval here in Q4 this year.

Analyst at RBCM

Okay. Thank you.

Operator

Thank you. Our last question is going to come from Faisal Khurshid with Jefferies. Your line is open.

Faisal Khurshid
Faisal Khurshid
Analyst at Jefferies

Hey, guys. Thank you for taking the question. Just wanted to ask a little bit on the YORVIPATH life cycle strategy. Can you give us an update on the latest on getting the higher dose into the label for FDA? Also any update on weekly YORVIPATH. Thank you.

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

Yeah. What we see today is that we are enrolling the trial in the U.S. where we're evaluating the 30-60 dose range in two different means that has been aligned with the FDA in their design, what they wanted to see. We see that enrollment going extremely fast, so we expect very fast, and you can say label expansion in the place where we don't have up to the 60. So we see that basic just on execution. Your second question was related to?

Faisal Khurshid
Faisal Khurshid
Analyst at Jefferies

On weekly YORVIPATH, any update there?

Jan Mikkelsen
Jan Mikkelsen
President and CEO at Ascendis Pharma

Yeah, I think there's no news in this way that we just executing and getting it into the market as fast as possible out from the expectation that we see that not as an any kind of LCM activity, but more a patient support for patient that really are in the stable dosing, which are not a lot after they have been in a situation where they have been stabilized with our daily treatment.

Faisal Khurshid
Faisal Khurshid
Analyst at Jefferies

Great. Thank you.

Operator

Thank you. This is all the time that we have for questions today. This does conclude today's conference call, and thank you for participating. You may now disconnect.

Chad Fugere
Chad Fugere
VP of Investor Relations at Ascendis Pharma

Thanks a lot, everyone

Analysts
    • Chad Fugere
      VP of Investor Relations at Ascendis Pharma
    • Jan Mikkelsen
      President and CEO at Ascendis Pharma
    • Scott Smith
      CFO at Ascendis Pharma
    • Jessica Fye
      Analyst at JPMorgan
    • Tazeen Ahmad
      Analyst at Bank of America
    • Gavin Clark-Gartner
      Analyst at Evercore
    • Yaron Werber
      Analyst at TD Cowen
    • Jay Wu
      EVP and President of U.S. Market at Ascendis Pharma
    • Derek Archila
      Analyst at Wells Fargo
    • Joseph Schwartz
      Analyst at Leerink
    • Daniel Bronder
      Analyst at Cantor
    • Yun Zhong
      Analyst at Wedbush
    • Alex Thompson
      Analyst at Stifel
    • Maxwell Skor
      Analyst at Morgan Stanley
    • Eric Joseph
      Analyst at Citi
    • Analyst at RBCM
    • Faisal Khurshid
      Analyst at Jefferies
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