Ascendis Pharma A/S (ASND) Q2 2026 Earnings Report, Transcript and Summary

Ladies and gentlemen, thank you for standing by. Welcome to the Second Quarter 2026 Ascendis Pharma Earnings Conference Call. [Operator Instructions] 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, 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 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 SKYTROFA, YORVIPATH and YUVIWEL, including label expansion and combination treatment, 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 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 and in 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. 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 epiphyses. 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 regarding 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 TransCon products continue 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 vision 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 to 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 enable us to fulfill our plans to file at least 1 IND or [ CTA ] 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 disorder. 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 hypopara that addressed 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 regions 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 standing standard of care in postsurgical and all subset of hypopara, including ultra-rare genetic causes like DiGeorge, ADS-1 and ADS-2. Results showed sustained response rate of 82% to 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 5 years of treatment, pretty unique. In parallel, we are working to further advance our leadership in hypopara 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 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 patients of all kinds of background, those switches 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 will 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 showed durable and consistent improvement in growth like leg bowing, 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 EU, 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 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 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. In our COACH clinical trial of children with achondroplasia, this unique combination has demonstrated sustained transformative annualized growth velocity and ACH 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 points 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 of unmodified somatropin. With indications for pediatric and adult growth hormone deficiency, we continue to be the #1 long-acting growth hormone by brand value in the U.S. We are extremely proud that SKYTROFA recently achieved more than 20,000 unique enrollment. This illustrates the strength of our capabilities from supply chain, commercial infrastructure and market support to benefit such a large number of patients -- rare disease patients. And we are working to make TransCon Growth Hormone available to more patients through label and geographic expansions. To support label expansion as 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 advance. 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 3 highly differentiated leading TransCon-based products, 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.

Thanks so much, Jan, and 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 nonproduct 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. YUVIWEL 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 prelaunch 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 YUVIWEL 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%, refer to our press release for details. For Q2 '26, 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 YUVIWEL, we are encouraged by the early demand trends. 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 YUVIWEL. 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. With that, operator, we are now ready to take questions.

[Operator Instructions] And our first question is going to come from Jessica Fye with JPMorgan.

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're able to kind of update your cash flow expectations for the year. I know it's sort of like a greater than is unbounded. But curious, if anything more you can add there? And then on that comment that YUVIWEL seems to be expanding the market, is it possible to estimate how much of these patient enrollments are coming from market expansion?

Thanks, Jess, for the questions. And I have a happy person besides me, Scott. So Scott got the opportunity to be the first one answering questions. So please, Scott.

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

And just related to the question, and 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. And our general feeling and how we see it is that with such a strong demand, we have a really strong belief that it is not only coming from switches, it must also coming from either patients that have 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. And I think this is where we have this strong belief that we see an expansion of the market.

And the next question is going to come from Tazeen Ahmad with Bank of America.

So 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, Tazeen, for the question. And it's basically a question that is addressing the ongoing legal I would call it, battle between Ascendis and BioMarin. And let me just come back to some facts. The fact is that this patent that is in discussion got completely 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. In the ITC case, there will be a first opinion from a single judge, and he will come with opinion here in August. And then there will be a -- 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 2 to 3 months after to a presidential order. So you can see we are not guiding any clarification in August in one way or the other way, even if it's positive for one company and negative for the other one, it's not really any kind of decision where it's going to be ending. And 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. There is a huge opportunity to provide what we call interest for this product. And when we see the public interest, meaning the element of how these product opportunities are really being serving an unmet medical need in the U.S. market with this rapid uptake of patients is really, really clear that is a huge public interest to keep that. And just recall, I cannot remember one single case in the U.S. where a branded product that provides a benefit to U.S. patients had been denied. But you can see me just in a case where it's only -- it's a U.S., it has been cleared ex U.S. And so whatever has happened, it will not have any material impact on Ascendis pathway. I can guarantee that. It's some kind of -- people take it up as a life and death for Ascendis. This is a total not taken into the perspective what it means for Ascendis. And out from that, I see it's not really is a material element for our destiny to be a leading biopharma and hit the EUR 5 billion in 2030.

And our next question will come from Gavin Clark-Gartner with Evercore.

I actually, just wanted to ask on the earlier pipeline. So you noted in your prepared remarks that TransCon platform can fuel one IND for an NCE annually. I guess there hasn't been one yet this year. Should we expect one in the near term? And what exactly are the go-forward plans for the earlier pipeline?

It was because I somewhat felt that the 2 product opportunities that we have developed through our partnership built on the TransCon technology will still consider as an NCE, the one that's now in clinic with Eyconis and the other one we expect to go into the clinic now with Novo Nordisk is still somewhere being developed to the TransCon technology funnel. And again, perhaps I shouldn't have done that, but I still believe I feel some kind of a little bit of ownership on these 2 product opportunities. At least we have major upside in both of them. So from that perspective, I still consider the potential that have this year 2 new chemical entities being entered into clinical trials. And I think Kennett and his team and anyone else, they are working very hard on that there will be at least one of these new chemical entities coming into every year now. And 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. And I think this is where we really feel extremely pleasant by the situation by being a fundamental company that's building on a strong, strong technology platform that provide both sustainability for ourselves, but also a continued flow of potential partner licensing.

And the next question will come from Yaron Werber with TD Cowen.

Great. A question on YUVI. Do you expect that there is some seasonality in terms of new patient starts in the summer as kind of kids are going on vacation. We're getting a lot of questions on sort of the 60 patient start forms kind of in April and now you're sort of at 220. It sounds like there's like 50 per month now. Is that sort of sustainable from now on? And then it sounds like you're planning -- you think you could be the #1 brand by the end of the year. BioMarin, we think, has about 750 patients on drug you think in the U.S. Are you kind of referring to getting to a higher number than that by, let's say, late February?

Thanks for the question. I actually don't think Ascendis have really made some clear forward-looking statement related to how we see YUVIWEL being accelerating and expanding the market in a quantitative manner. I don't think we have come with any kind of indication related to that. I have no doubt it will do it, but it's not the same thing that we're going to quantify it currently. I think after basic only 4 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. But one person that really can give you a good feedback, now we talk about the U.S. market is Jay, and he's extremely enthusiastic about what he's seeing, and you can give the latest way what you see how the market will develop.

Thanks, Jan. As Jan mentioned before, 4 months in, we're not prepared to give longer-term guidance, but what we can say is we're incredibly encouraged by what we're seeing today. When you look at some of the fundamentals behind the YUVIWEL uptake, whether it's prescriber reach, we talk a lot a bit before around this space, there's quite a few centers of excellence. We're seeing 80% of them, nearly 80% already in a short 4-month period already prescribed YUVIWEL to their patients. So even in early days, we're 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're seeing a very positive trajectory. 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 3 categories for which our patients are coming from. And those 3 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 pharmacological treatment. And 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. So 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.

Just to summary to add on to Jay's excellent comments. Ultimately, we have no doubt we will be #1 in the achondroplasia space. Ultimately, we will expand the market because of the unmet medical need. And that is just with the monotherapy. And when you look at how commitment we are after this era, where we now are making a complete new standard with the [indiscernible] treatment. I believe we have dedicated to be not #1 in the first year, but continue to build for the next 5 to 10 years with monotherapy combination integrated treatment regimes. And I believe with our once-weekly TransCon product built on growth hormone and CNP we are extremely, extremely well positioned really to be the leader in this segment.

And our next question will come from Derek Archila with Wells Fargo.

Congrats on the progress. Scott, I just wanted you to clarify a comment on YORVIPATH growth for the rest of the year. I think you said it's 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 could just clarify which quarters you are referring to?

Yes, Derek, thanks for the question. I think that 2 points. One is the consistent performance with the KPIs that we've 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've seen the 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.

Just to give you some kind of what is our value in this year. The value for us, we want to give you -- 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. And I think this is the way we try to come up with our different mathematic algorithm, how we see it and give you all the information for you really to be right in this manner. And I think this is a way we like to be extremely transparent with everything what we perform. So we're quite sure that you basically can go out and really somewhere feeling always comfort with the guidance we give you.

And our next question will come from Joseph Schwartz with Leerink.

Congrats on all the progress. As you embark on a Phase III in hypochondroplasia, I wanted to ask how you're defining the enrolled population? And how do you -- how large do you see the diagnosed treatable pool of hypochondroplasia patients who are not already being treated in some cases, if they're at the more severe end versus achondroplasia?

Yes. This is from -- this is a very interesting question because it's actually some way going into the situation on how we basic are to genetic testing, taking a big patient group that was in old days, what called ISS idiopathic, meaning we have no clue what is the underlying disease is. And then you go out and do more and more and more genetic testing. And then when you find a mutation in the FGFR3 receptor and you find it in the right regions, and then you suddenly are not an ISS patient, but then you find hypochondroplasia patient, even if you don't have, you can say, the phenotype of looking like an achondroplasia patient or a hypochondroplasia patient that we saw for 10 years ago. So therefore, you can see the ISS population is some way getting smaller and smaller because of the genetic testing is basically going out and giving them an underlying reason why you [indiscernible] will have a short status without potential have the other element that you see for the phenotype of that. So this is where you can then -- when you go into ISS, are you defined it from a genetic perspective or you define it for a phenotype or anything like that. And we are in a situation where when you see the clinical trial, how we're doing it, you will basically see that it's one of the pathway we have selected.

And our next question will come from Daniel Bronder with Cantor.

Congrats on the quarter. On for Li Watsek. 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?

Just to recall, the COACH trial is the combination trial where we're combining the 2 TransCon based product, our TransCon Growth Hormone and TransCon CNP. And I have to say, when I look on elements like arm span, it's actually -- we already reported some of the data. We have reported the 52-week data. And if you cannot find that deck, I can send it to you or Scott can send it or Chad can send it or I don't know we have so many IR people I don't know all names now. So from that perspective, it is already came out. And I have to say there was one of the -- I will say, extremely positive surprises I saw because when we look on monotherapy, but either a CNP-based one or a growth hormone based one, we did not see the expected hopeful development that we can hope for. But we definitely saw it when we look under combination therapy. And then you can ask me what is the scientific reason why you see it much more influenced benefit by the combination therapy. And I have to say, I don't know. But what we saw was an arm span that was really -- give 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. And it's this Slide #5, as I remember it. And what we see, Scott, read up.

The unprecedented improvements in the arm span with combination were plus 9.4 centimeters with TransCon CNP naive cohort 7.9 centimeters with the TransCon CNP-treated cohort.

So it was really and...

Compared to limb lengthening surgery, 8 centimeter.

Exactly. 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.

And our next question will come from Yun Zhong with Wedbush.

I wanted to confirm that you have not provided prescription number for YORVIPATH in case I missed anything. And so I know that you said the patient demand remained robust in the quarter. So I wonder if there is any additional quantitative information that you can provide. And going forward, are you going to provide that number in the coming quarters? And I think you had this question before at the beginning of the launch and when do you expect that you will feel comfortable providing a guidance in terms of the sales range on actual revenue?

You are right. And I think it's starting to be a little bit positive every quarter come out and saying that we have about more than 1,000 patients being unique enrolled per quarter. We have continued that measure that we see steady state, steady state and steady state. And we said in last year that we will stop coming with this because it was too repetitive. And then because people doubted for Q1. Then we also come up with the Q1, and it was the same number again. And what we're writing is that we see a robust steady-state in enrollment of unique new patients. And here, we are referring to the U.S. with about 1,000 new patients every quarter, and we don't believe really. Now we went over to YUVIWEL. So now we're starting to give you a unique prescription enrollment of YUVIWEL instead. So we always -- we have one product opportunity where you will have something to play with numbers and everything like that. Scott, do you have some comments to the last one?

Yes. I think our comments were directed to assume the metrics that we've given YUVIWEL are consistent because Jan wants to make our script shorter. So we're not -- don't want to repeat them more. And you should just assume that until we change it.

And the next question comes from Alex Thompson with Stifel.

Jan, 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 sort of this idea around the 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?

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.

And the next question will come from Maxwell Skor with Morgan Stanley.

Just a quick one on YORVIPATH durability. I was just wondering if dropouts are still mostly during the titration phase. And if you can comment at all on how reauthorizations are trending?

I think you're 100% correct. And when we see a patient being successful coming into a treatment with YORVIPATH coming over titration part on it and be into the treatment after that, we see extremely, extremely low dropout. And 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 on it. And want to see the satisfaction that it is in this way, then I think that there is an extremely good precision, retention and everything would really show that. We still develop once weekly for patients on stable doses. Just to give patients 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, calcium monitoring and anything like that, we can help the patient like it's happening in type 1 diabetes and other things like that. So now you're addressing the element where we're saying is we developed this year 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's really complex to take patient off of conventional therapy. At the same time, you increase the PTH in replacement therapy. And this is why we made it as a once daily this really to facilitate the best possible titration but still, we know it can be problematic for some patients. And Jay can try to explain what we're now doing to basic and hold the patient in this period. So we also can make that extremely successful. So 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. So Jay, will you explain of the effort you're building in to really to get that to be as soft and as possible?

Absolutely. I can chat a little bit more about certainly, the investments that we're making and also to answer your questions around drop-off and re-auths. Yes, as we've shared before, the majority of the drop-off 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. . And then for re-auth, that's actually pretty routine for us. So there really isn't much there in terms of it being a measurable effects on any kind of ongoing patient support, we have patients re-authing throughout the year, and it's just part of our day-to-day operations. From an investment standpoint, we've invested heavily in patient-facing roles for which we've deemed our patient access liaisons, they support patients both pre prescription as well as through the prescription process and post. So essentially, we've 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're optimizing for patient experience.

One thing that's imminent. Now we focus more on U.S., but there is still a world outside U.S. Outside the U.S., we have not seen the same level of drop out in this phase. It looked like the interaction is pretty well established between the physician and the patients and support system, we need to see it without this kind of drop out. So it basically is a U.S. issue. And so 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 it's not a problem.

And our next question will come from Eric Joseph with Citi.

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

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

YUVIWEL.

Yes. I can guarantee that as we basic in our prepared remarks, tried to put emphasis on. We have a global infrastructure in commercialization and patient support, product supply and everything like that. Just the number of SKYTROFA rare disease patients we have taken over to the system, more than 20,000 patients. We are having the system functional more than in 35 different countries. So we're not a company that just need to get started. We already have established all this infrastructure. And what we're doing is that we are utilizing this established infrastructure basically that got established because of YORVIPATH because this is what we did with YORVIPATH. We're using exactly the same infrastructure also for YORVIPATH. So we will be where patient is, and we'll 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. And we believe because of the highly differentiated nature of YUVIWEL, we will see a complete different pickup in the U.S., but it's definitely -- we have a strong, strong, 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.

And the next question will come from Luca Issi with RBC CM.

[indiscernible] for Luca. Circling back to YUVIWEL, and Jay the 3 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. So the simple math that we're trying to do here is that it leaves you with about 70 patients in the second quarter who are naive or return to treatment. So that's taken off the switch patients. But 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? And separately, very quickly, if you've commented or not on the ex U.S. strategy for YUVIWEL, given the decision is pending and the [indiscernible] coming very soon this year.

I like your way of doing all the calculation, anything like that. I cannot support it or I cannot deny it because I don't have the factual insight to some way to confirm anything of the numbers. I also saw the numbers that came out, but I cannot really support it because I don't have the insight from our own numbers to really come out and come with any statement that indicate if I'm 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?

Thanks for asking to clarify it. More about are you committed to running the show by ourselves or you're considering partnering given that 70% of the VOXZOGO sales is historically coming from ex U.S. can be a quite heavy lifting?

Yes. But -- so basic in the ex U.S., we have our direct markets, which are, I think, 60, 70, 80 where we have our own commercial infrastructure, anything like that is pretty, pretty clear what we're doing there. Then we have our sales and distribution agreement. And this is, I think, it's 70 countries or something like it. Well, Scott it's...

80

80 countries that is covering this sales and distribution agreement. And the vast majority of all of them are all 3 products. So basic is already established infrastructure for the distribution. And then we have the 2 other. The third model where we have our partnerships, one in Japan, one in China, and they also have all the 3 products. So we don't need to go out and make any new agreements for anything -- everything is established. Everything is running on full speed. And we -- for some of the EU direct market, we're just waiting for our expected approval here in Q4 this year.

And our last question is going to come from Faisal Khurshid with Jefferies.

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? And then also any update on weekly YORVIPATH?

Yes. What we see today is that we are enrolling the trial in the U.S. where we're evaluating the 30 to 60 dose ranges in 2 different means that has been aligned with the FDA in their design, what they wanted to see. And we see that enrollment going extremely fast. So we expect very, very, 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. And your second question was related to?

On weekly YORVIPATH, any update there?

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

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.

Thanks a lot, everyone.