ASND

Good day, and thank you for standing by. Welcome to the first quarter Ascendis Pharma earnings conference call. At this time all participants are in an only listen mode. After the speakers presentation there will be a question-and-answer session. To ask the during the session, you need to press star, one, one on your telephone. You will hear an automated message when your hand is raised. To withdraw your question, please press star, one, one again. Please be advised that today's call is being recorded. I Would like to hand the conference over to your first speaker today, Chad Fugere, Vice President of Investor Relations. Please go ahead.

Thank you, operator. Thank you everyone for joining our first 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 Chief Executive Officer, Scott Smith, Chief Financial Officer, Sherrie Glass, Chief Business Officer, and Jay Wu, EVP and President U.S. Market. Before we begin, I'd like to remind you that this conference call will contain forward-looking statements that are intended to be covered under the safe harbor provided by the Private Securities Litigation Reform Act. Examples of such statements may include, but are not limited to, statements regarding our commercialization and continued development of YORVIPATH, YUVIWEL, and SKYTROFA, as well as certain expectations regarding patient access and financial outcomes, our pipeline candidates, and our expectations with respect to their continued progress and potential commercialization.

Our strategic plans, partnerships and investments, our goals regarding our clinical pipeline, including the timing of clinical results and trials, our ongoing planned regulatory filings, and our expectations regarding timing and the result regulatory decisions. These statements are based on information that is available to us as of today. Actual results may differ materially from those in our forward-looking statements. You should not place undue reliance on the statement. We assume no obligation to update these statements as circumstances change except as required by law. For additional information concerning the factors that could cause actual results to differ materially, please see our forward-looking statement section in today's press release in the Risk Factors section of our most recent annual report on Form 20-F filed with the SEC on February 11, 2026.

TransCon PTH is approved in the U.S. by the FDA for the treatment of hypoparathyroidism in adults and the European Commission and United Kingdom's Medicines and Healthcare Products Regulatory Agency have granted marketing authorization for TransCon PTH as replacement therapy indicated for the treatment of adults with chronic hypoparathyroidism. TransCon CNP is approved in the U.S. by the FDA for the treatment of achondroplasia in children aged two years and older. Continued approval for this indication, which is based on an improvement in annualized growth velocity, may be contingent upon verification of description of clinical benefit and confirmatory trials. TransCon hGH is 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 E.U. has received MAA authorization from the European Commission for the treatment of pediatric growth hormone deficiency. Please note that our product candidates are investigational and not approved for commercial use. As investigational products, the safety and effectiveness of product candidates have not been reviewed or approved by any regulatory agency. None of the statements during this conference call regarding our product candidates shall be viewed as promotional. On the call today, we'll discuss our first quarter 2026 financial results, and we'll provide further business updates. Following some prepared remarks, we'll then open up the call for your questions. With that, let me turn it over to Jan.

Thanks, Chad. Good day, everyone here from Copenhagen. The first quarter of 2026 was an eye-opening for Ascendis with the FDA approval of our third TransCon product, YUVIWEL. Our revenues are growing rapidly. We are profitable. We have a pipeline of high-value product opportunities to support long-term growth. Three elements are cementing our position as a leading global biopharma company. First, our diversified product portfolio in one single therapeutic area. Following FDA approval of YUVIWEL, we have now achieved approval of three products in a row across four rare endocrine indications. Second, our rapid revenue growth from our existing endocrine rare disease portfolio, YORVIPATH, YUVIWEL, and SKYTROFA, each highly differentiated with long durability. We expect sustained revenue growth for many years to come. Third, expanding our pipeline. We have proven our ability to create transformative medicines addressing unmet medical needs using our TransCon technology platform.

To date, we have more than 20 ongoing or planned clinical trials aiming at label and market expansion, including four differentiated New Chemical Entities in preclinical development. Turning now to SKYTROFA. Global SKYTROFA revenue in the first quarter reached EUR 197 million. SKYTROFA revenue for the first quarter was impacted by two one-time items. A temporary increase of U.S. patients supported by free drug caused by reimbursement disruption and one-time impact of Europe direct related to expanded market access. Scott will explain the financial impact of these two events in his remarks. In the U.S., new patient enrollment in Q1 remain in line with the strong uptake we have seen in Q4 2025, with more than 1,000 new patients prescribed SKYTROFA during the quarter.

To the end of March 2026, more than 6,300 patients have been prescribed EUPAD by more than 2,700 unique healthcare providers. March was our largest revenue month ever for EUPAD, supported by an increased number of new patients, as well as patients returning to reimbursement from free drug. Importantly, the enrollment trend we saw in Q1 have continued to accrue through April, consistent with our guidance. Insurance approval rates and median time to approval continue to improve. This strong support a strong foundation for revenue growth in 2026 and many years to come. Outside the U.S., EUPAD is available commercially or through named patient programs in 35 countries, including full commercial reimbursement in six of our Europe direct markets, with additional launches expected through 2026.

Looking forward ahead, we continue to pursue multiple expansion opportunities for YORVIPATH in new markets and indications. This includes doses up to 60 micrograms in the U.S., global expansion to patients aged 12-18, and continue development of once-weekly TransCon PTH for patients on stable YORVIPATH doses. With 70,000-90,000 patients living with chronic hypoparathyroidism in the U.S. and five to 10 times that number outside the U.S., we remain highly confident in YORVIPATH's long-term global potential. I will now turn to our growth disorder franchise. With once-weekly YUVIWEL, alongside our once-weekly growth hormone SKYTROFA, we believe Ascendis is uniquely positioned to strengthen its leadership in growth disorders. Our U.S. commercial infrastructure, built and refined since SKYTROFA launched in 2021, has enabled a focused and high impact launch for YUVIWEL, which became commercially available in early April.

Since then, YUVIWEL has already been prescribed for more than 60 children by more than 35 unique healthcare providers, with children approved for reimbursement as fast as a few days. YUVIWEL has shown compelling results compared to placebo across multiple clinical trials. In addition to linear growth outcomes, these results include improvements in leg bone, spinal canal dimensions, body proportionality, physical function, and health-related quality of life compared to placebo without compromising safety or tolerability. We believe this outcome reflects YUVIWEL's unique ability to provide continuous systemic CNP exposure throughout the body over the weekly dosing interval. Looking ahead, we plan to make YUVIWEL available in selected international markets through early access programs using the U.S. FDA approval. As a reminder, our global infrastructure covers over 70 countries and has already generated product revenue for us in more than 35 countries.

In E.U., a regulatory decision on our marketing authorization application for YUVIWEL is expected in the fourth quarter of 2026. We are also pursuing label expansion for YUVIWEL through ongoing trials. These include infants under two years of age with achondroplasia and children with hypochondroplasia, as well as geographic label expansion clinical trials. Turning now to SKYTROFA. In the U.S., SKYTROFA maintains consistent performance as a premium product with 7% share of the overall growth hormone market, reflecting steady demand across pediatric and adult patients as the only once-weekly product delivering unmodified somatropin. With the expected label expansion that could double the addressable patient population in the U.S. and geographic expansion outside the U.S., we believe SKYTROFA will remain a cornerstone product in our growth disorder portfolio. Turning to our pipeline. This includes combination therapy with once-weekly TransCon CNP and TransCon hGH for children with achondroplasia.

In our phase II COACH trial, we have reported unprecedented results that exceeds the already compelling foundation established by YUVIWEL monotherapy. Week 52 data from COACH presented in January showed improvement in achondroplasia-specific height Z-score that indicate a triple of efficacy compared to TransCon CNP monotherapy, along with improvement in body proportionality. More recently, we shared additional Week 52 data that showed improvement in lower limb alignment, as well as an accelerated improvement in spinal canal dimensions and an improvement in arm span, not previously demonstrated with pharmacotherapy within a single treatment year. Based on this finding, we believe our unique combination therapy of TransCon CNP and TransCon Glucagon could potentially eliminate the need for highly invasive procedure such as leg lengthening, arm lengthening, and leg straightening surgeries. We believe that this combination therapy could become the preferred treatment option for achondroplasia. I will now briefly turn to oncology.

In our phase I/II IL-Believe Trial, we have elevated TransCon IL-2 beta/gamma in combination with paclitaxel in patients with late-stage platinum-resistant ovarian cancer or PROC. Median OS improved up to 10 months from six to seven months from historical controls with a general well-tolerated safety profile, validated the science on TransCon IL-2 beta/gamma. As further internal oncology development does not align with our strategic focus, we have decided to discontinue internal development of TransCon IL-2 beta/gamma in oncology and will explore other ways to maximize the value of these assets. Turning now to our partnership, our once-monthly TransCon semaglutide with Novo Nordisk continue to advance towards the clinic at Eyconis TransCon anti-TGF-beta also remain on track to enter the clinic this year. These programs further highlight the broader potential of our TransCon technology platform to address product opportunities in larger indications.

In closing, in the first quarter of 2026, we made significant progress across our business and our ability to make a meaningful difference for patients. We have three FDA-approved TransCon products across four indications, growing revenues, improving cash generation, and a pipeline that support long-term growth. I will now turn the call over to Scott to review our financial results.

Thanks a lot, Jan. Good afternoon, everyone. I will touch on some key points surrounding our first quarter financial results, which reinforce our confidence for growing operating profit and cash flow into the future. For further details, please refer to our Form 6-K filed today. YORVIPATH global revenue was EUR 197 million in Q1. The first quarter was characterized by steady global uptake and normal seasonality, as well as two one-time items. Patients temporarily transitioned to free drug in the quarter in the U.S., and a one-time impact in Europe direct related to expanded market access. The combined impact of these two items was approximately EUR 15 million. SKYTROFA contributed EUR 44 million in Q1. On a sequential basis, performance reflected consistent underlying demand with the expected drawdown in channel inventory built in Q4.

Including EUR 6 million in collaboration revenue, total Q1 2026 revenue amounted to EUR 247 million. Continuing to expenses. R&D expenses in Q1 were EUR 59 million, down from EUR 78 million in Q4 2025. R&D in Q1 was favorably impacted by a write-up of YUVIWEL inventory consisting of EUR 11 million due to U.S. FDA approval and lower clinical activity across the portfolio. SG&A expenses rose to EUR 145 million in Q1 2026 compared to EUR 136 million in Q4 2025, reflecting continued impact of global commercial expansion. Total operating expenses for Q1 2026 were EUR 204 million, and operating profit was EUR 25 million, reflecting a 10% operating margin. Non-IFRS operating profit was EUR 55 million and non-IFRS operating margin was 22%.

As revenue scales, we expect meaningful improvement in our operating margin, which will be visible over the course of 2026 and beyond. Net finance expense for Q1 2026 was EUR 63 million, primarily driven by non-cash items, including remeasurement loss of financial liabilities of EUR 34 million. Net cash financial expense for Q1 2026 was about EUR 1 million. Net profit for Q1 2026 was EUR 629 million, which included recognition of a EUR 679 million deferred tax asset in the P&L. Refer to our 6-K for more detail. Non-IFRS net profit was EUR 18 million or EUR 0.27 per share.

We ended Q1 2026 with EUR 573 million in cash and cash equivalents, which includes the impact of EUR 60 million in Q1 from our previously announced share repurchase program and net settlement of certain RSUs. In April, we successfully completed our transition to a direct listing of our ordinary shares on Nasdaq. We believe this will broaden access to global investment in the company, which has the potential to further enhance institutional ownership and trading liquidity for Ascendis shares. In May, we completed the full redemption of all of our outstanding convertible senior notes. Finally, today we announced that we entered into an agreement to sell our PRV for $187.5 million in cash. The PRV was awarded by the U.S. FDA upon approval of YUVIWEL in February. Turning to our commercial outlook.

For YORVIPATH, we expect continued steady underlying increase in patients on therapy and the reversal of one-time factors seen in Q1 to drive strong growth sequentially in Q2. For SKYTROFA, we expect stable revenue throughout the year following a similar seasonal pattern to 2025. Regarding YUVIWEL, as Jan indicated earlier, we are encouraged by the early demand trends and look forward to sharing more with you on our Q2 call. With that, operator, we are now ready to take questions.

Thank you. At this time, we'll conduct a question-and-answer session. As a reminder to ask a question, you'll need to 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. Please limit yourself to one question, and if you have additional questions, kindly rejoin the queue. Please stand by while we compile the Q&A roster. Our first question comes from the line of Jessica Fye of JPMorgan. Your line is now open.

Hey, guys. Good morning. Thanks so much for taking my question. I was wondering if you could help us estimate what U.S. YORVIPATH sales were in the quarter. I think in the past you had run through an algorithm to consider, but I know the press release noted some one-time impact to Europe direct as well. Just trying to get a better sense of the U.S. versus ex-U.S. split this time around. Thank you.

I think that Scott will give you just a little bit more background on the financial element specific. One time in Europe direct, it actually happened when we sometime and specific for one single country because we had an early access program that was running for nearly 15 months, 16 months. It gave us a one time event where we needed to write down for this single case. It's not something that really happening in other countries, but it was a single country event which basically impacted our Europe as you can say Q1 results. Scott can give you a little bit more flavor on the finance numbers.

Yeah, Jess, for modeling purposes, it's probably a little bit more of an impact, but I would say that the best way to think about it is take the algorithm that we laid out where you add four to five a quarter. For Q1, basically that addition was just shifted into Q2. From there, the algorithm continues.

I think just one of the key elements I will take into regard is basically underlying patient demand. I think the key element is really that we continue the same successful, you can say, rollout of the loans both in U.S., where we now, as we have providing you the number of indicated new patient on treatment with Europe. As we have given in our previous guidance, we're still 100% correct in that, where we really see the same stability, we see the same flow coming in. Jay can comment about how he already starting to improve both the time to reimbursement and the numbers. I do not. Jay, will you comment about your effort in really improving what we call the reimbursement situation for the U.S.?

Sure. When you think about our reimbursement, we're seeing improved metrics across the board. First and foremost, we've talked a little bit about our upstream coverage now expanding to about 80% of patient lives, which we're feeling really good about given the time on market, and I think, again, a testament to the compelling clinical value proposition that we have. We're also seeing continued rapidity as it relates to patients being approved for reimbursement upon entering the funnel. Again, over half approved within eight weeks of enrollment, and we are seeing continued progress, again, with patients moving through the funnel, whether it's upstream as the enrollments are coming in, but also supporting patients as they're entering into the funnel.

Great. Thank you.

Thank you. One moment for our next question. Our next question comes from the line of Tazeen Ahmad of Bank of America. Your line is now open.

Hi, guys. Good morning. Thanks for taking my question. Mine will also be on YORVIPATH. Maybe this is for Jay. Can you talk about the reauthorization rates that you're seeing now that patients are starting to annualize at this time of year? Any things to point out about things that were maybe unexpected or taking a little bit longer? Then can you talk about usage among physicians? Is there a way of providing a split between how much use is coming from first-time physicians versus an increasing use among doctors who've tried YORVIPATH before? Thanks.

Jay there was multiple questions. I hope you got everyone down. Will you start on some of them?

Sure. I think I heard a few questions. One, which maybe I'll start with towards the end, which is prescriber breadth and depth, I think was the question. We're seeing continued traction across both. As Jan mentioned earlier in the script, we've had over 2,700 prescribers, which again is an addition of about a 300+ quarter-over-quarter, which we're feeling really good about. That would answer your question around new prescribers. Then within existing prescribers, we're also feeling really good because the average prescription per physician continues to increase as well. In fact, about 10%-ish of prescribers have now over five enrollments for patients. Again, as you think about the provider landscape here, they all do have a different patient volume, just given how diffuse the patient volume is.

Generally speaking, we're seeing not only one additional prescribers sign on to YORVIPATH, given the strong patient satisfaction scores that we're seeing, but then because of those positive patient experiences, we're also seeing providers expand their scope of who they deem to be eligible, given that lab values alone are not the reason that patients should be treated.

Okay, thanks. Reauthorization was the last one.

Reauthorization, yes, that was the first part of your question. We're not seeing any meaningful differences in terms of approval rates for a re-auth versus necessarily a patient that's coming in at the top of the funnel. We typically, again, have shared that five to eight week timeframe. We've seen those numbers continue to increase in terms of speed, which I referenced earlier with the first analyst question. We're not seeing any meaningful difference with the re-auth coming in versus a new patient coming in. Generally, what you'll see is if it's a re-auth of an existing payer insurance where there hasn't been a change in insurance, you might see some faster timeline there. If it's a brand-new insurance, you're obviously going to treat it as just a brand-new case, so to speak.

Give one moment for our next question. Our next question comes to the line of Yaron Werber of TD Cowen, and the line is now open.

Great. Thanks so much. Maybe a quick follow-up and then a question on YUVIWEL. A follow-up on YORVIPATH. Of the EUR 15 million, should we roughly kind of split it like half in Europe and half in the U.S.? I don't know if you can give us any sort of, you know, view on that. For YUVIWEL, in the ITC case is progressing, the briefing document, the briefs are out kind of back and forth, and it looks like the court is kind of siding with both parties. What I know you've been importing drug in the meantime. Any view sort of on how much capacity you might be able to have in the system by the time a decision is rendered? Thank you.

First of all, I believe when you see the uptick in the prescriptions of YUVIWEL in the U.S., we have more than 60 children being prescribed YUVIWEL treatment in less than four or five weeks. I think it illustrate the unmet medical need that exists in the U.S. related to an improved treatment in achondroplasia. I believe what we have seen of clinical data in our multiple trials with YUVIWEL, just as a monotherapy, is really describing the reason why we see this take off. This is not just of having a once weekly product. This is providing a tolerability profile and documented effect on benefit beyond linear growth. I think everyone is aligning with the unmet medical need, the public interest always to have a product such as YUVIWEL in the market.

We will continue to be in a position that we have such a strong belief that in this case here too, like it was in Europe, we can prove that this IP case, this should never have existed and only is built on false promises. I'm 100% confident YUVIWEL is here to stay, and it will always be a treatment option for patients with achondroplasia in the U.S. I hope that answer your question related to that part. The other part, I think you're somewhere in the right estimate when you think about it.

Thank you. One moment for our next question. Our next question comes to the line of Gavin Clark-Gartner of Evercore ISI. Your line is now open.

Hi, this is Evie on for Gavin Clark-Gartner. We have two quick questions. Number one is for the phase III TransCon CNP and growth hormone combo trial. Can you share any update on the enrollment speed? Secondly, what are you seeing in your YORVIPATH discontinuation rates over time?

When we talk about, as I understood your question, right, it was related to the combination trial, the phase II trial, the trial we call the COACH trial. We basic are now, I do not know how many years we are into the trial now, but I think we're two years in, one and a half year now in it. I think we see basic an extremely high element of protection in this trial. To my knowledge, last time we looked, it was 100%. I think it's really been harder to get more than 100% in a clinical trial, to my knowledge. I think that's a very, very, very few trial where you have 100% retention after nearly 18 months.

From that perspective, I think it's really illustrating and addressing the satisfaction with the benefit you see in the treatment compared to the burden for treatment. I think that is really the key element that we always are looking in the fundamentals. We want to see benefit for patients. This is why we're working, and we will continue to focus on that.

Thank you. One moment for our next question. Our next question comes from the line of Li Watsek of Cantor. Your line is now open.

Hey, thanks, guys. I guess on your YORVIPATH new patient, as you mentioned, you know, steady growth, is it reasonable for us to assume 1,000 is sort of the number that we should be looking at for the coming quarters? Will you be sharing new script number going forward? Thanks.

That's a great question. Now, somebody going back to last time I said I will not come up with more prescription data for Europe because I believe that the revenue progression was so clear. When I said that, there was a big, big, big, what I call the element of some interesting funds that push back and saying, I didn't want to come out with numbers because it must be really, really bad. Now we have illustrated for one quarter more that they are not bad, they are extremely good, and exactly as predictable as we have said in this way. I expect a steady state enrollment in all the quarters because that is what we expect. We are only touching a small amount of this patient group.

We have so much more patient that is coming, new patient every year. I'm somehow giving up to say that I will not come up one quarter more because I last time I said we will not come up in one quarter, and then I got bad press because I didn't want to listen to that, we didn't want to come in up with a number because they're bad. I don't hide anything. I always want to be transparent. This is why I say, come up with a number, then you can see it. I think we will continue to be so transparent on everything what we're doing, so you always have the best possible opportunity to see how well we have performed in our fundamentals.

Thank you. One moment for our next question. Our next question comes to the line of Alex Thompson of Stifel. Your line is now open.

Hey, thanks for taking the question. This is Patrick on for Alex. Could you guys just talk about the potential impact of the YORVIPATH 60 micrograms being on the label? You know, maybe what % of those 6,300 patients in the U.S. is dose capped at 30 with, you know, maybe less than ideal supplementation levels?

This is a question which are very, very difficult for us to answer today because we see different kind of uptitration in both different situation in clinical trials and also situation in what I call more real world. We are following it a lot. We are now open for enrollment in our trial where we are having two arms in our evaluation of dose titration over 30 up to 60, and we will enroll that in a decent speed. We only do that in the U.S. because this is only place where we restrict it down to 30 and not have 60. We believe that even if you are coming up to 30 microgram, you still have a major benefit to be still on 30 microgram compared to many of the positive effect that YORVIPATH is still providing to it.

I think you can say, yes, there is somewhat of a benefit to go higher, but today we're still providing the benefit to the patient that need to stop on 30 micrograms.

Thank you. One moment for our next question. Our next question comes to the line of Joseph Schwartz of Leerink Partners. Your line is now open.

Great. Thanks so much for taking my question. Some physicians we have spoken with have suggested that they might not want to put their office staff through the reimbursement challenges of switching their achondroplasia patients to a once weekly injection, only to then later switch them to a once daily pill in the not too distant future. Is this a dynamic that you guys are aware of? What can Ascendis do to help support the achondroplasia patient or physician community rather and encourage uptake. My second question is, have any physicians prescribed YUVIWEL in combination with SKYTROFA since YUVIWEL was approved? Thanks.

Answering your first part of the question, I think the number speaks for itself. When you think about it, more than 10 prescription per week for such a rare disease product. I think it talk about the unmet medical need and the willingness for the physician in connection with the parents, in connection with the child to have the desire to take them on a treatment with YUVIWEL. I think it says everything with numbers. You can go out and ask one physician. You can go out and ask one parents. I look at numbers from a statistic odds. The numbers talks for itself. Related to the last question, I cannot some way discuss an element we cannot promote. We cannot promote any way in situation the combination therapy.

We have disclosed the benefit of the combination therapy. It's up to the physicians if they really want to prescribe it or not prescribe it. To my knowledge, I can be pretty open about it, yes, it happens, and I understand why. This is the only way you basically can be in a position where you basically can avoid any kind of surgeries, which I think is such a positive element for any child with a condition to avoid these invasive surgeries. I think this is a reason why the physician do it. Sure, we looking forward to finalize the phase III trial. We're looking forward to have it on label. We really also can go and promote it actively.

Thanks for the insights.

Thank you. One moment for our next question. Our next question comes from the line of Eliana Merle of Barclays. Your line is now open.

Hi, this is Jasmine on for Ellie. Thank you for taking our question. Just kind of a follow-up to the last question. For YUVIWEL, can you say how many of those 60 enrollments were new starts versus switches? More generally, do you think the initial population is going to be more new starts or switches? What kind of patients do you think are the most likely to initially want to switch? Thank you.

The insight you're asking for is the insight we will develop in the coming months and quarter. After five weeks to try to come with a general statement about what kind of patient, what kind of preference they have to go onto YUVIWEL treatment, I think it's too early for us to come with a conclusion with it is such a topic. The only thing I can say, and Jay, you can add on, what we see is basic coming from everywhere. It's not just a new patient, it's not just switch patient, it's coming everywhere from where we expect it also to come from.

One of the things we have done at Ascendis that Jay have made an impact on to help the physician, the patient, is really to have the path that can go out and really help the physician, the patients everywhere to get through this journey to be sure they can come on the right treatment. Jay, do you have anything to add?

Yeah, I think you summed it up well. The only two additional things I would add is, one, to Jan's point, we're seeing across all segments. We've discussed before the three areas or types of patients that we envision coming are, one, patients currently on VOXZOGO that are switching over. Two, patients that were previously on VOXZOGO but since discontinued and were on no therapy. Three, a patient that perhaps had never made the decision to start therapy at all. We are seeing anecdotally that it's coming across all three of those groups.

I think really what that underscores is the continued and existing unmet need that exists in the achondroplasia today, even with the existing therapy on market, which I think emphasizes the value of having YUVIWEL on the market, and the compelling value proposition that it offers patients. I think the second area that Jan was talking about is our continued investment in just making sure that everything we're doing is hand in glove with patients, both as it relates to partnerships with the patient advocacy groups, but also as it relates to our scaling up of our patient access liaison team, which is our patient-facing field group that invests in the support and the journey as they go through the continuum of prescription to ultimately being on therapy.

All right. Thank you.

Thank you. One moment for our next question. Our next question comes to the line of Yun Zhong of Wedbush. Your line is now open.

Hi. Good morning. Thank you very much for taking the question. My question is on the monotherapy for hypochondroplasia. I remember that there have been some changes in terms of approach for that program, whether you're going to pursue that indication at all, and whether it's gonna be mono or combo or maybe, just Are you able to share any information regarding the thought process behind the decision, if this is the final decision that you are going to just using monotherapy to target hypochondroplasia? Thank you very much.

I think the strategy that we have applied to achondroplasia, where we started with monotherapy and then moved over to the addition to combination therapy. I think you will see that as there will be alignment between the strategic approach that we have used in achondroplasia, we will likely also use in hypochondroplasia. I can basically tell you that we're using the same principle between the two indications. The two indication is very much aligned in the fundamentals of the disease. There's only, you can say, different mutation, different severity and other things like that. We will implement the same thinking and treatment regime between these two indications.

Thank you. One moment for our next question. Our next question comes on the line of Luca Issi with RBC Capital Markets. Your line is now open.

Great. Yeah, thanks so much for taking my question. Maybe Jay or Scott, can you educate us on the mechanics of this free drug for YORVIPATH? Who are the patients that got the free drug? For how long do they stay on free drug? Are you expecting any patients still on free drug in Q2, or is this just a kind of Q1 phenomenon? Any call there much appreciated. Super quickly, I think AstraZeneca is presenting their phase III data for eneboparatide this week in the European Congress of Endocrinology. Wondering if you can comment on what are your expectations for that data. Thanks so much.

Yeah. I can somewhere start from the last question, then I can move it up, then Jay can take over in the end. The compound we are talking about in the end is the Amolyt compound. You know we have discussed that on many earnings call, the lack of information that was related to data. That is disclosed some kind of information of the data packet, one year after finalization of the phase III trial. For me, I think the key question, do this data packet provide an approvability of any way of this compound? To my best judgment, I hope this product never will be approved, I don't see really as possibility that it should be and going to be approved.

I think when we look on the competitive landscape for treatment in hypoparathyroidism, I see YORVIPATH, our once weekly approach to stable patient on YORVIPATH, is really providing the fundamentals for a 20, 30 years treatment regime, where I don't see anything that really can live up to the benefit we will see in the treatment with YORVIPATH from any other product that is currently in clinical development from that perspective. Related to the first part of your question, you're right. We started to take already December a group of patients over to free drug because the essential part of YORVIPATH is that you cannot stop. If you first have started taking you over to basic the element on what we call the conventional therapy, it's basically is a disaster for the patient.

If there was a hiccup in the reimbursement, which there was a hiccup, it's something we had done corrective action to ensure that we can handle it much better next year. We were in a position that we took, and Scott explained the impact of that here in Q1, to take already from December until March, a series of patients on free drug, and they are now coming back to be fully reimbursed. We are in a position that Jay and the organization in U.S., someway have built up network for the things that can help it, that we not ending in the same situation one time more. Jay, have you any comments to?

Yeah. Again, I think you summarized the need for Bridge program well. I think just to clarify the earlier question, there is two types of free programs. We have Bridge program, which again is pretty standard across the industry for those that have experienced a temporary insurance lapse. But we also just have our patient assistance program for patients that are under-insured or uninsured. I think that's an important point to note because there will always be some patients that qualify for the patient assistance program. We don't anticipate that that will ever go away completely, knowing that there will always be a certain level of patients that qualify for that.

Yeah. I think that is a clarification that's great from Jay, where we talk about this number of patient is only what we exceeded as success compared to the base level of patient. We always will help and provide free drug if there is an element of something where there is a disruption of the normal way to have drug. It's such an essential drug for the patients that we are always will take our patient focus first. If there's a patient that get it disrupted, we will do everything to help this patient. That include also to take them for a period on free drugs until the disruption getting solved. We will always be there for the patients.

Thanks so much.

Thank you. One moment for our next question. Our next question comes on the line of Maxwell Skor of Morgan Stanley. Your line is now open.

Hello, this is Selena on for Max. Thanks for taking our question. Given the relatively low treatment penetration in achondroplasia in the U.S. to date, what proportion of patients typically initiate treatment at age two years or older?

I think some way to roll it a little bit back because you can ask the question, why do you have under treatment in the U.S.? I think actually this is the key question to find out how can we really help this patient better. I think, and we believe that the under treatment is basically because of lack of the right efficacy to show real benefit beyond linear growth. Many of these parents, children don't see linear growth as a key element. They really want us to address all the comorbidity specific. If you can avoid leg surgeries for changing the pain, complication with leg bone, you can avoid arm lengthening, leg lengthening. I believe by having this focus on these elements, and here I talk about the older children.

If you go to a younger newborn, yes, if we can avoid any kind of spinal stenosis by having early intervention from newborn, yes, there will be a major benefit for initiate treatment in the newborn state. I believe our product profile that we have generated with YUVIWEL, showing as the only product clear benefit beyond linear growth compared to placebo. It need always to be placebo-controlled because there is a development too fast side. You cannot say, "Oh, we also improve arm span or arm length." Yes, and child with achondroplasia actually have a big increase in arm length. You always need to really show it compare into a placebo-controlled trial. It's the only way you can really assess the benefit of the medical treatment in it.

The question and the answer to you is, I believe YUVIWEL will be appealing product to the vast majority of parents, children in the U.S. also because it will provide a way to address the comorbidities. Like important one, leg bone, arm span, everything like that you basically will see benefit for not only to quality of life that's associated with it, but also the element of physical strength.

Thank you. One moment for our next question. Our next question comes on the line of Paul Choi of Goldman Sachs. Your line is now open.

Hi, thank you and good morning, and congrats on the good start with YUVIWEL. I was just wondering if you would clarify, in terms of the 60 more than 60 prescriptions you've seen to date, whether it's more driven by treatment naive patients or switches. Any quantification there would be great. I'm also curious in terms of your early starts or through the quarter, if you're seeing potential utilization in the below two years of age population, even though that's not officially on label yet. Thank you.

Yeah. We need to come with a meaningful answer. We need longer time because we only have been there for four, five weeks now, and we want to be sure that what we see in the initial part of the launch is also being representative of what we'll see in the later state of launch. Paul, we can discuss it, as Jay said, in a perfect manner. We see patient coming in for all three different groups, which was naive group, patient that have discontinued with VOXZOGO, and patient that come directly on switching for crossover time. Out from that, we see coming in for all three different groups. In the initial launch, we will expect to see perhaps one mix, and when we come a little bit longer into the launch, we will potentially see a switch in the different three classes.

This is why we need to make it meaningful. We need to wait longer time before we can give you something you can do the right modeling. I, when I look at this number be coming up more than 10 patients per week, it's such an orphan drug indication. I'm extremely proud that our product profile is getting so well-recognized in the society in this way. Under two, I cannot comment on that currently.

Okay, thank you.

Yeah, hey, good morning, and thanks for taking the questions. For the 70% cumulative U.S. insurance approval rate that you previously cited, can you give us any more color on what that cumulative rate looks like today? Second, the EUR 500 million operating cash flow target that you laid out previously, are you reaffirming that guidance given the Q1 trends that you're seeing? How should we think about the contribution of YUVIWEL to that target? Thank you.

Yeah, the last one, we would like to come back in Q2, because we have a lot of positive data coming in now. We have the selling of our PRV, SKYTROFA and YORVIPATH going much, much stronger than I even myself had hoped for. Aside from that, we will come with a changed guidance, but we will prefer to do it after our Q2 call, and we will give you what will be reflected on the 26th. Scott is saying yes to me. Jay, you can give the factual number, how it's improving the overall numbers.

Sure. The overall cumulative approval rate since launch has continued to creep up as well. I think we're now closer to mid 70%, which again is incredibly high for any rare disease asset, much less one that has been on the market for the amount of time that we have. A lot of that is just a function of time, given the favorable access policies that we have. Even some enrollments that might be, you know, many months old are coming through on appeal online, which would affect the cumulative approval rate over time. Given that it is a lagging indicator, it will take quite a bit of time for that metric to mature.

Got it. Thank you.

You one moment for our next question. Our last question comes from the line of Cecilia Hernandez of Van Lanschot Kempen. Your line is now open.

Hi. Thanks for taking our questions. This is Sandrine on for Cecilia. Given the revenue now from three commercial products, the redemption of the convertible notes and the sales of the PRV, can you tell us anything on your capital allocation strategy? What is the order of priority for you guys?

I think, Scott, want really to answer that last question for today. Scott, get this opportunity now.

Thanks a lot, Jan. Of course, as you've seen with our R&D success, a key component for us is to invest in R&D and allocate capital there to continue to sustain not only into the 2030s, but the 2040s and beyond with the continuous flow of new products. I think Jan highlighted four new NCEs in his prepared remarks, and you'll learn about more of those in the coming future. Of course, I think after we give an update after Q2, as Jan mentioned, to our outlook for the rest of the year, you may get more color at that point as well.

Thank you. That is all the time we have for questions. Thank you for your participation in today's conference. This has concluded the program. You may now disconnect.