Ascendis Pharma A/S (ASND) Q4 2025 Earnings Report, Transcript and Summary

At this time, participants are in listen-only mode. Thank you for standing by, and welcome to the Ascendis Pharma A/S Fourth Quarter 2025 Earnings Conference Call. After the speakers’ presentation, there will be a question-and-answer session. To ask a question during this session, you will need to press star 11 on your telephone. If your question has been answered and you would like to remove yourself from the queue, simply press star 11 again. As a reminder, today’s program is being recorded. And now I would like to introduce your host for today’s program, Chad Fugier, Vice President, Investor Relations. Please go ahead, sir. Thank you, operator, and thank you, everyone, for joining our full-year 2025 financial results conference call. I am Chad Fugier,

Vice President, Investor Relations at Ascendis Pharma A/S. Joining me on the call today are Jan Moller Mikkelsen, President and Chief Executive Officer; Scott T. Smith, Chief Financial Officer; Sherrie Glass, Chief Business Officer; Jay Donovan Wu, Executive Vice President and President, Ascendis US; and Aimee Shu, Chief Medical Officer. Before we begin, I would 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 Skytrofa and Yorvipath, as well as certain expectations regarding patient access and financial outcomes; our pipeline candidates and our expectations with respect to their continued progress; our strategic plans, partnerships, and investments; potential commercialization; 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 of regulatory decisions and the result. 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 our forward-looking section in today’s press release and the Risk Factors section of our most recent annual report on Form 20-F filed with the SEC on 02/11/2026. TransCon Growth Hormone or TransCon hGH is now approved in the US 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 US by the FDA for 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. Otherwise, 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 will discuss our full-year 2025 financial results and we will provide further business updates. Following some prepared remarks, we will then open up the call for your questions. With that, let me turn the call over to Jan. Thanks, Chad. Good afternoon, everyone. With strong execution across our business, and continued progress to delivering on our Vision 2030.

Ascendis is transforming into a leading global biopharma company. We believe this progression demonstrates the power

platform.

And our R&D capabilities to deliver a sustainable pipeline. While our global commercial infrastructure and financial profile continue to strengthen, we believe we are now at the base of a steep growth, where we expect to achieve operating cash flow of around €500,000,000 in 2026 and where we aspire to achieve at least €5,000,000,000 in annual product revenue by 2030. At the same time, we are building an expanded pipeline of blockbuster product opportunities. We saw multiple achievements across the organization in the fourth quarter, starting with Yorvipath. The fourth quarter was another period of strong execution for the global launch of Yorvipath. Revenue for the quarter was €187,000,000, bringing full-year 2025 Yorvipath revenue to €477,000,000. In the US, access continued to expand. To year end, more than 5,300 patients were prescribed Yorvipath by nearly 2,400 unique healthcare providers,

highlighting continued

strong and steady demand. To date, less than 5% of US patients are currently on Yorvipath treatment, highlighting the significant long-term growth opportunity ahead. The overall insurance approval rate is about 70% of total enrollment, and we continue to see this figure moving higher over time. In addition, we continue to see a majority of approvals within eight weeks. This provides a strong foundation for expected additional growth in 2026 and beyond as more patients initiate Yorvipath in line with treatment guidelines that support its use. Outside the US, we continue to reach more patients. As a reminder, Yorvipath is now available commercially or through named patient programs in more than 30 countries. We have full commercial reimbursement in four countries in our Europe direct markets and two countries in our international markets. In Japan, our partner Taisho launched Yorvipath commercially last November. In 2026, we expect full commercial launches in 10 additional new countries. We also advanced development activity to broaden Yorvipath’s label in a number of areas. In the US, we are working to expand the range of doses toward PATHway-6 trial, and globally we continue to advance clinical trials to expand Yorvipath to patients under the age of 18. Our work is progressing rapidly on once-weekly TransCon PTH for patients who have been titrated with daily Yorvipath for conventional therapy and have achieved a stable daily dose for a well-defined period. Last month, at the annual J.P. Morgan Healthcare Conference, we shared preclinical data that support the target product profile for a once-weekly TransCon PTH candidate matching the release PK/PD as seen with daily Yorvipath treatment over the entire week, thus providing a comparable efficacy and safety profile. Overall, we remain confident that Yorvipath has the potential to be a durable long-term growth driver for Ascendis globally. Turning now to growth disorders, comprising our once-weekly growth hormone Skytrofa, or TransCon Growth Hormone, and our once-weekly TransCon CNP. Skytrofa delivered another solid quarter with Q4 revenue of €53,000,000, bringing full-year Skytrofa revenue to €206,000,000. This performance reflects the strength and value of the brand. As a reminder, Skytrofa is now approved in pediatric growth hormone deficiency in the US and adult growth hormone deficiency. Today, Skytrofa has an overall market share of around 7% in the US. During the fourth quarter, we initiated our Phase III basket trial evaluating TransCon Growth Hormone in additional established growth hormone indications, including ISS, SHOX deficiency, Turner syndrome, and SGA, which comprise up to half of the growth hormone market. Over the long term, these indications represent meaningful opportunity to expand the role of Skytrofa as a treatment of choice in additional growth disorders. We also see an opportunity to potentially expand Skytrofa’s use to novel indications where growth hormone has not previously been approved for use, such as achondroplasia, in combination with TransCon CNP. TransCon CNP is expected to be the first and only once-weekly treatment with monotherapies addressing the overactive FGFR3 tyrosine

kinase.

In addition, in our pivotal trial, TransCon CNP achieved a significant improvement profile compared to placebo, with a very low rate of injection-site reaction, spinal canal dimension, and a similar safety and tolerability and no cases of symptomatic hypertension. In the US, in leg bowing compared to placebo, our NDA for children with achondroplasia remains under review with a PDUFA date of February 28. In the EU, the MAA review is underway,

last October with a regulatory decision expected in the fourth quarter

tried in infants.

with achondroplasia ages 0 to 2 is going well, and we anticipate complete enrollment later this year. Turning to the combination therapy, our 52-week data in achondroplasia underscore the potential power of dual treatment with TransCon CNP and TransCon Growth Hormone. Continuous exposure to CNP enables the benefit of sustained exposure to unmodified growth hormone. In comparison,

Monotape

trials of daily growth hormone in achondroplasia delivered only a limited effect on growth and no group-reported benefit on linear growth. Our 52-week data from the Phase II combination trial support our vision to significantly raise the bar for treatment of achondroplasia, with linear growth improvements in achondroplasia-specific height score that were three to four times what has been shown with CNP or daily growth hormone monotherapy in the same

Focused development programs in both our monotherapy and combination therapy programs. Importantly, all children completed fifty two weeks of treatment and remain in the trial. Reinforcing the benefit of treatment and acceptable treatment burden of the once weekly machine. These phase two results demonstrate the effect of these complementary therapies. Supporting that TransCon CNP act in synergy with growth promoting effect of TransCon growth hormone. And has positive effect beyond linear growth. We believe over time the standard of care in achondroplasia and other growth disorder long term will include dual therapy as a treatment option. Building on the potential road of TransCon CNP as an essential fundamental therapy. We recently had a successful end of phase two FDA Meeting And Scientific Advice Meeting In EU to align on our phase three trial for this novel combination approach for treatment children with achondroplasia. We also remain on track for additional Cokes trial updates including 78 by mid year and 104 by year end. And plan to explore further opportunity in other group disorders. To sustain doable long term growth for Ascendis. Well into the next decade. We plan to continue to invest in label expansion. Of our current products in rare endocrine diseases. In addition, we have a strong focus on the development of new blood product opportunities, both inside and outside rare endocrine diseases. To food significant product revenue growth in the future. Looking at our partnerships. TransCon Technologies support a continuous flow of highly differentiated product opportunity across multiple therapeutic areas, more than we can develop and commercialize ourselves. For this reason, our Vision 2,030, includes a focus on creating additional value through partnership and collaboration. Our collaboration with Novo Nordisk for once monthly transconcemic glutide continue to advance towards the clinic. Adarconis TransCon anti VEGF is on track to enter the clinic this year. In Japan, Talypian received approval for Europatch in August 25, and commercial launched it in November 2025. In addition, vision approval of Skytrova in China in late. January twenty six. In summary, 2025 was another positive and transformative year for the SETIs. With two commercial TransCon products, continue to scale, the potential approval of the third high value TransCon product in the coming weeks. And a growing pipeline of highly differentiated programs. We believe we have the fundamentals in place to deliver global long term growth. A rapidly strengthening financial profile gives us confidence to achieve an expected operating cash flow of around €500,000,000, in 2026 and our aspiration to achieve at least €5,000,000,000 in annual product revenue by twenty twenty third. All consistent with our Vision 2030 strategy. I will now turn the call over to Scott. Thank you, Yan, and thank you, Chad, for a well read FLS.

The significant achievements we made in 2025 provide us with substantial financial strength to drive our strategic priorities and goals in 2026, which include achieve blockbuster status for Yorvipath, solidify our leadership in hypoparathyroidism through rapid while advancing development of our once weekly PTH candidate progress of our label expanding clinical trials of TransCon PTH, successfully launched TransCon CNP if approved in the US and other countries around the world, and expand our leadership in growth disorders through clinical and regulatory progress with once-weekly Skytrofa, including in combination with once-weekly TransCon CNP. With that, I will touch on some key points surrounding our fourth quarter results. Quarter and full-year financial results which we mostly already announced at J.P. Morgan. But for further details, please refer refer to our annual report on Form 20-F filed today. As previously announced in January, Yorvipath delivered strong global performance in Q4 2025, revenue increasing to €187,000,000, up from €140,000,000 in Q3. Foreign currency had a negligible impact compared to the previous quarter. Total Yorvipath revenue for 2025 was €477,000,000. For the full year, the weaker US dollar negatively impacted Yorvipath revenue by approximately €27,000,000. Skytrofa contributed €53,000,000 in Q4 with negligible foreign currency impact compared to Q3 2025. Total Skytrofa revenue for 2025 was €206,000,000. For the full year, the weaker US dollar negatively impacted Skytrofa revenue by approximately €9,000,000. Including €7,000,000 in collaboration revenue, total Q4 2025 revenue amounted to €248,000,000, and total revenue for full-year 2025 was €720,000,000. Continuing on to expenses. As previously announced, total operating expenses for Q4 were €214,000,000, and total operating

total

operating expenses for the full year 2025 were €761,000,000. As we previously noted,

profit for Q4 2025 was €10,000,000 with

Q4 operating cash flow of €73,000,000. As we have discussed for some time, below operating profit, the drivers include the noncash accounting related to our convertible notes. Net finance expense, which was primarily driven by noncash items, including remeasurement loss of financial liabilities of €106,000,000, was €93,000,000 net. Net cash finance financial expense, however, for the full year 2025 was about €8,000,000. In future periods, we may introduce a non-IFRS EPS measure adjusting for the impact of certain items to increase the comparability of period-to-period results. We ended 2025 with €616,000,000 in cash and cash equivalents, as previously reported, up from €560,000,000 as of December 31, 2024. Turning to our commercial outlook and to help inform your revenue modeling for the coming year. For Yorvipath, we expect continued strong revenue growth in 2026 based on steady patient uptake with some expected seasonality in reported revenue throughout the year. For Skytrofa, we expect to follow a similar seasonal pattern to 2025 with full-year revenue growth expected to track growth in prescriptions. Longer term, Skytrofa revenue is expected to come through geographic

Epic and label expansion.

As always, we continue to watch the euro-US dollar exchange rate for any potential impact. And finally, we also look forward to the potential US approval of TransCon CNP later this month, which, as a reminder, has been excluded from this 2026 outlook. With that, operator, we are now ready to

Certainly. And once again, ladies and gentlemen, we ask that you please limit yourself to one question each. Our first question comes from the line of Jessica Macomber Fye from J.P. Morgan. Your question please.

Hey, guys. Good afternoon. Thanks so much for taking my question. What is your confidence level heading into the TransCon CNP PDUFA? Are you comfortable that the issue leading to the review

Yes. Can you remember

extension has been resolved to the FDA’s satisfaction? Thank you.

your

Ask me a question one time, and the conference

And can you remember my answer?

I do remember the answer.

And what was your question? You can ask the same question.

The hey.

I remember your answer, but it was about a different product, if I recall, but your answer was yes.

Yes.

So this is the same. You asked me, will TransCon PTH be approved? And I said yes. And you can ask me the same question today. Will TransCon CNP be approved? And I would say yes.

Thank you. And our next question comes from the line of Tazeen Ahmad from Bank of America. Your question please.

Hi. Good afternoon. Thanks for taking my question. You mentioned that 70%

Insurance approval rate in the US so far for Yorvipath. Where is that relative to where you thought it would be at this stage of the launch? And what is it going to take to expand that to a higher number, where do you think long do you think it is going to be before you get to 100%, basically? Thank you. I think it will be infinity, because

because I have never seen a product hitting 100%.

So

I think the highest bar for seen is something like 85% or something like that, perhaps up to 90. The element of where we are today, I am really highly satisfied with it because it is also a compromise about how aggressive you are going into contracting and other things like that. So I think it is a balance between the two things where in the end

The overall

and ultimate goal is basically to provide most value back to our shareholders and others, and at the same time help the patient to as fast as possible to come on treatment. I do not know, Jay, if you have additional comments to my I will now say pre-prepared remarks.

Yeah.

One is that I would say two things.

We are very happy with the overall approval rate that we are seeing, and I think the speed in which you are seeing this product be covered again

It is a testament

to the strong clinical value proposition that we are

Seeing in hypoparathyroidism. It is the first and only approved

therapy in this category. So, again, this approval rating is something that we are very encouraged by. Based on where we are today, I think to your second part of the

question, Tazeen, which is, you know, when might

you get to 100%? I

echo what Jan said earlier as

well, which is I do not know that many drug analogs will get to 100%, and that actually has less to do with

coverage, and

also has to just to do with

every single enrollment that comes in. Not

single one of

them will be eligible relative to the label.

So there is some element of just natural filtering that comes that way. But more importantly, what I would say is that there are state Medicaid plans, for example, that review things on a staggered

cycle. So you will anticipate that some of this will creep up over time. But it will take some time before it continues to inch upwards.

We need to some way, this is a Yeah. Okay. But but I think I think still low. all approvals in enrollment. US discussion. The US discussion is built on 70% of

We are not there.

So when you go in and look on an old cohort that perhaps have been six months through it, your actual will get NMOS higher number on it. Just to clarify that 70% on it, that is when you take everyone accumulated. If you take an old cohort, it is much higher. And when you go ex-US, the system is quite different. When you get a prescription, you nearly in every other country, you are axiom approved. So you can say the 100% yes is basic when you get a prescription outside US in traditional countries, you will be 100% electable and already approved for reimbursement.

Got it. Thank you for the color.

Thank you. And our next question comes from the line of Gavin Clark-Gartner from Evercore ISI. Your question please.

Hey, guys, thanks for taking the question. Just on your Yorvipath pricing, so there was an 8% WAC increase in January. Maybe you could just discuss how net pricing will trend this year, including how to quantify the magnitude of the Q1 seasonality here.

I do not think we really are discussing net prices. We would love to do it, but we have never done it, and I do not think we ever will discuss net prices.

Maybe if I could just ask a follow-up then. Just on patient enrollment, are you planning to still report those forms going forward for Yorvipath or maybe just focus more on revenue?

I think it, Gavin, is 100% right. We will focus on revenue because now we basically have been in the market now in the US. We are on the market for about four quarters now, when we come to here the fifth quarter, I think you have seen a steady state development from 2025 where we basically got an increase in basic revenue from both US and ex-US, about €40,000,000 to €50,000,000 net every quarter. I think you will somewhat see a stability in how we are executing in it. We still have ex-US. We will expect 10 additional countries being fully reimbursed next year. I am sure that is always improving what we call the net revenue we will generate outside the US.

Thank you. And our next question comes from the line of Yaron Benjamin Werber from TD Cowen. Your question please. Great. Thanks so much. I have sort of two not really related, but I am going to try to link them to keep it as one question.

Maybe the first one, can you give us a little bit of a sense for Yorvipath? How it is being used out there? I mean, it is almost like when you look at this 2,400 unique prescribers and 5,300 unique patient enrollment, so is it that each physician just has one or two patients in the practice, are they prescribing it there and then they are going deeper? And then

secondly, just at the end of

Phase II meeting with FDA relating to CNP and growth hormone for achondroplasia, maybe just can you give us a little bit of an update, what was the outcome and when will you start the Phase III?

Thanks. Okay. That is perfect. I think, Jay, you can give some about how we are both expanding the physician prescriber base, but also go in more in the deep of the different patient, but still are far away to reach delivery where we want to be. Jay,

Yep. So in the US, I think the question is really around segmentation and what types of patients are being treated. So if you think about the prescriber base, this is where you first started your question from. We are seeing broad uptake across the entire range of prescribers. So to your point, there are some physicians that might only see a couple patients, but there are also some physicians that might see upwards of 10 patients. More importantly, because we are seeing broad uptake across both high-decile and low-decile providers, we are not seeing a major discrepancy as to the type of prescriber that would prescribe, but we are seeing that breadth continue to increase. As it relates to the number of patients per physician, we are also seeing the depth of prescribing per physician also increase over time, which again is encouraging. That is both a testament to positive experience that they have with Yorvipath as well as increased awareness of the hypoparathyroidism condition and option for it amongst patients. I think the last

Now they are being an up thing I would say is

when you think about the types of patients that come

through,

you can look at it in two ways. One, the vast majority of them are postsurgical, so about 70%. The remaining 30% perhaps due to other factors, whether it is genetic, autoimmune, etc. We are seeing broad across both of those segments, so that is not a major driver. Really where you are seeing some of the earlier uptake is patients that are self-aware of the condition that they have, and therefore, are linking the symptoms that they have to the underlying condition that they have,

Thank you. And our next question comes from the line of

Okay. Joseph Schwartz.

a combination of them advocating for themselves as well as providers having conviction in the product as well. So all in all, we are seeing broad uptake across provider groups as well as patient segments as well.

Before you the question start, perhaps I can answer the last part of related to the COACH trial. Yes. We had extremely positive meetings both US right and from the EU side, it was really impressive feedback to the data. There

have never seen

data before that basically are providing this kind of benefit to an achondroplasia treatment. I am not only talking about the linear

growth where we basically on

z-score got a three to fourfold more than you can see with monotherapy in the same time period, but also unique elements like such an improvement in body proportionality. What was really what really they have never really seen in such a meaningful manner was a really important element, the arm span, where we also saw in the combination trial a unique improvement in arm span. And Aimee is sitting here with the sales team, and she is really doing everything to get this trial recruited as fast, be ready to go. Protocol is finished and everything. Be open site. Just remember that our pivotal trial in monotherapy, we recruited it just in three or four months, just because of the interest of the patient. Therefore, the bar for Aimee is very, very, very hard if she needs to do that faster. Sorry for coming in.

Absolutely. Not a problem. Our next question comes from the line of Joseph Schwartz from Leerink Partners. Your question please.

Hi. This is Heidi Jacobson on for Joe Schwartz. Thanks so much for taking our question. Can you help us understand how the TransCon CNP launch could factor into your $500,000,000 operating cash flow target for 2026,

particularly with respect to launch investment and early revenue contribution?

Thanks. It is pretty simple. It is not incorporated. When we are coming into the launch, we see the initial uptake, which we believe will be pretty high, not only in the US but also outside US because we can utilize the US approval to go to countries outside US, specifically in the international market. From that perspective, we will come and provide you a better guidance and improved guidance when we have seen that. Scott is smiling, are you counting money or

taxes and money.

If you will come back after that.

Great. Thanks.

Thank you. And our next question comes from the line of Derek Christian Archila from Wells Fargo. Your question please.

Hey, good afternoon. Thanks for taking the questions. I just wanted to understand your confidence level around Yorvipath growth ex-US. Obviously, the launch in Germany and Austria, is that a good proxy? Or is it going to be more depth in those types of countries than just kind of expansion in, you know, I guess, I think you said 10 additional countries. So how will that be sequenced through the year? Thanks.

That is an extremely complicated answer because the heterogeneity of the ex-US is so heterogeneous that we cannot really compare to what we, for example, see in Spain now, what we see in France, what we see in Germany, what we see in Austria. It is really different things because we see different speed of penetration. For example, Germany has less endos, so the bottleneck is really more tight. It takes longer time to get them on therapy because there are fewer in the general population. If we go to Spain, there are more. There are more in France, and we also see, therefore, a faster uptake because we basically have a pipe that is larger. When we get 10 more additional countries on full commercial, we will see different uptake, but what we are doing is everything will be accumulated in the way where we now see from 30 to 35 countries named patient programs. When we go full commercial, what we see every place is basically an acceleration of patient uptake, because of the burdensome nature of a named patient program. It takes so much effort to get every single patient on it, and every patient deserves to be under treatment. So when you come to 2026, we will see initial speeding up in all these countries. When we come to 2027, 2028, you will continue to do it because, just by nature, we just got approval now in Canada, and we are basically taking one country after the country, first going in and getting approval, and then we are going to put reimbursement.

Our next question comes from the line of Li Watsek from Cantor. Your question please.

Hey, thank you so much for taking our question. It is Daniel Brondo on for Li. Can you give us a little bit of color on how you expect your TransCon CNP launch to go? It seems like there are a few patients who

currently are on treatment. Where do you think you will capture the majority of patients

initially?

Pretty clear. The improvement that we see with TransCon CNP to what we can provide. Not only related to when we look on tolerability, injection-site reaction, having one in 20 injection-site reaction compared to one every second year. Being in a position to look on no risk of hypertension. The element of just having the improvement on the once-weekly product and then show the data package that we have generated with TransCon CNP, for first time ever shown in a well-controlled trial against placebo, benefit beyond linear growth. For example, the leg bowing, which we have shown multiple times, we have shown improvement in muscle strength. We have improved quality of life. I think this is obvious.

Every

patient that decides to be on treatment should have the opportunity to have the best possible treatment option, and I think there is a public interest in the US to ensure that it always will happen.

Thank you. And our next question comes from the line of Eliana Rachel Merle from Barclays. Your question please.

Hey, guys. Thanks so much for taking my question. Curious if you can elaborate on your strategy for commercializing TransCon CNP ex-US. Just given the majority of vosoritide sales are ex-US, could you elaborate on your strategy for the commercial launches globally and the degree of investment that that will take? And then just a second question on TransCon CNP in the US, can you talk a little bit more about how you are thinking about the cadence of uptake and which segments do you expect the most uptake from between, say, treatment naive versus switches? Thanks.

Yeah. I will dial a little bit back now because what we did

global when we said that we want to have commercial

effort.

We actually started all our infrastructure building to Yorvipath. And now you see what we have done with Yorvipath recognized their fast revenue, commercial revenue for more than 30 countries. We are penetrating them exactly as we can do. We will reach 60 to 70 countries in less than two to three years. So what we have done, we already have built up the infrastructure to be ready that we can take our integrated pipeline of rare disease endocrine product into all these different countries in already the setup via step links around Yorvipath. So this is the positive element that we are not, you can say, a company that needs first to take up an infrastructure to support a globalization. We have already established that. So I feel really confident that all the success we see now with Yorvipath on a global scale we will just take it in. Do not forget, for example, even in Japan, the collaboration we have with Taisho is for all three products, the same thing in China and other places. So when we make this agreement, we are not making single product, single country. We make it as a pipeline product. And this is why we do not need to go out and make new agreements or anything. It is just going to be done extremely fast from that perspective.

Thank you. And our next question comes from the line of Leland James Gershell from Oppenheimer. Your question please.

Great. I want to ask, as we look at the €5,000,000,000 number you have put out there for product sales by 2030. I know you are not giving specific product guidance here, but if you could share with us how you think about the relative contributions of your presumably three products by that point in time in terms of how they will weigh into that total sum. Obviously, you have much more expansion opportunity in hypoparathyroid, you have TransCon CNP potentially launching soon, and Skytrofa perhaps getting additional indications in combination. I would love to just hear maybe just philosophically how your outlook adds up with those three parts. Thank you.

Yeah. That is an element where we always in our forecasting are operating under different assumptions, where we are basically building up models for each single country and then we accumulate that on a global setup. We first take 2026, we take 2027, 2028, and 2029. Then what we are doing, you always will go in and look on the risk balance. Where do we have potential extra upside that we can explore. What are we going to do with this fountainhead? But I think what makes Ascendis unique today is that we are not a single product in a single region. We will have three approved products in perhaps 20 different indications in about 30 to 40 countries. Meaning that what we really want to do, we will not be dependent on one single product in one single region. This is how we build up a sustainable company that has a continued stable revenue flow for multiple years. Do not forget these product opportunities we have. When I look on the pipeline for each of them, I definitely do not have sleepless nights. I can guarantee that. There is no doubt that when I see the profile and how we design it to be best in class, we also see that realized. Out from that perspective, it is a combination product with lifespan of IP extremely long. This is where you have the durable durability of it. This is why we take the value perspective of each single product opportunity instead of fast revenue. This is not how we operate. We go for value, and because the element of that is this is the product really to serve this treatment because we are providing not only a unique benefit for the patient but also for the society and everyone.

Great. Thanks very much.

Thank you. And our next question comes from the line of Paul Choi from Goldman Sachs. Your question please.

Hi, good afternoon. Thanks for taking our question. I think your Phase II REACH IN study is scheduled to reach primary completion next month. Will you be in a position to file an sNDA for the newborn infant population this year? And in terms of the newborn infant population, in your discussions with the FDA and EMA for your Phase III combination study, does your study design allow for those newborn patients to be included in this study population, or will that require a separate study? Thank you.

I think when you discuss a label discussion, that typically is different between, now I just take the two main regulatory areas because we can also take Japan into it, but if you take, for example, US, it is much harder some way to be coming to a situation where they will accept a label expansion to the infant without having the data in hand. In Europe, it is much more flexible because you have a discussion with them, and you can have what I call partly rolling of data that is being generated to our

trial.

So there will likely be a difference between the three geographic regions. Now simplified, Japan is mostly perhaps the easiest way to get it down to infant immediately. What we are doing now is to ensure we generate the right data and we are doing that in a trial. It is a placebo-controlled trial, and what we see is everything we hoped for. It is living up to our expectation. Why I can say that? Because in the enrollment we have six patients on a, what I call, physical treatment on it. You take them in, and there is no randomization. You can follow them, and Aimee can tell a few words about the benefit we have seen from that perspective.

So Jan is talking about the sentinel kids who are not part of the randomized piece of the study, and they are doing well, tolerating the medicine as well as we would expect, growing and starting to see early signals of the other benefits as well, particularly radiology.

Yeah. So we are really so pleased with the progress we are doing in helping patients with achondroplasia, not only on linear growth, but also benefit beyond linear growth.

Thank you. Our next question comes from the line of Yun Zhong from Wedbush. Your question please.

Hi, good afternoon. Thank you very much for taking the question. My question is on the weekly TransCon PTH. Is it reasonable to expect that the program could potentially enter the clinic in 2026, or do you think that there is no such need to rush? Also, you mentioned matching PK to the daily product. With data from Yorvipath available, what would you see as the most efficient clinical pathway to maybe take the weekly PTH to approval?

I think what you are addressing is two things that are interconnected. Because if you, for example, can show the PK profile, and it can even be healthy volunteers or patients with hypopara, that over the entire

week,

of treatment, you basically are bioequivalent to Yorvipath. That is the aspiration how we designed it, that you basically will always be in an excellent PTH level compared to your Yorvipath daily dose for the entire week. Then we know you basically will get the expected safety, the durability from that perspective, and this will make a much more simplified, easy way to conduct the clinical trial. It was why we designed it exactly in this manner.

Okay. Thank you very much.

Thank you. And our next question comes from the line of Luca Issi from RBC. Your question please.

Great. Thanks so much for taking my question. Congrats on the progress. Maybe, Jan, kind of big picture, I think one of the goals for 2030, as you articulated at J.P. Morgan, is to remain an independent and profitable biotech company, and we have seen many examples of that in our industry recently. However, how are you thinking about maybe continuing that same vision under the broader umbrella of a larger pharmaceutical company? I guess the question is how are you thinking about strategic path A versus strategic path B at this point? Any color there much appreciated. And then maybe, Jay, quickly, I think BioMarin has announced that they will file vosoritide for full approval versus I believe you will initially get approved on an accelerated approval basis for TransCon CNP. How should we think about that difference? Will that have implications for formulary access and reimbursement? Do you not view that difference as material for adoption given you obviously have a less frequent dosing versus—

I think I will liberate Jay for answering the last

question. I think when I look on this discussion on accelerated approval that BioMarin filing for, that has no

impact

on our regulatory pathway and approval and other things like that. Totally independent. It is not any way how you can build up any barrier or any way in this way. The second thing, yes, in our vision there is independent, and I believe that is a great word because we want to be independent like a teenager growing up. One of the things, at least I have four children, I am teaching them when they are going to be aging, you need to be financially independent as the first element in their life. I think that is a great thing to see Ascendis Pharma now moving away from being a teenager, but basically can go up to a more adult life. We have shown now we are completely independent on

asking

investors and others for any kind of revenue, and I think this is how we see independency.

Super helpful. Thank you.

And as a reminder, ladies and gentlemen, we ask you to please limit yourselves to one question. You may get back in

Our next question comes from the line of Maxwell Nathan Skor from Morgan Stanley. Your question please. Great. Thank you very much. My question was asked, but I will take a shot at this. Could you give any color on the once-monthly TransCon semaglutide program? Any gating factors, when we should expect an update? Any additional color would be helpful. Thank you.

Yeah. Let me go back to all the element and all the IP we have done, files and data and everything like that before we went into this extremely productive collaboration with our neighbor in Copenhagen, Novo Nordisk. What was really the idea behind once-monthly semaglutide? The idea was to be sure that you can get fast weight loss and at the same time have a high level of tolerability. Just think about, I can define it, you have a naked GLP-1 molecule that when you give it weekly or potentially want to use a weekly product in a once-monthly, you need to add much more compound to compensate for the half-life to have a large AUC. By doing this, you add a high Cmax dose. Because it is naked, you will have a very short Tmax, meaning that you will have a steep curve from the lowest level just before you start to give a dose up to the maximum concentration. That is basically what often gives the tolerability issue where you get the element that basically limits people to stay on treatment and what you can achieve. This is what I call the naked product. This is like metacresol and everything. This is a naked protein. What we are doing now is defining what we call packed-in semaglutide. Even if you give it high dose, you liberate it slowly, slowly, slowly, so you are getting a very long Tmax. By doing that, you basically have a slope that is not as steep at all as you see with a naked molecule, then you can see you still have a big AUC because you provide so much compound, give it over the entire month, and at the same time, you do not have this steepness in the slope. By that, you do not have that. It was designed to have maximal weight loss as fast as possible with the best tolerability profile, and it was how we designed it at that time.

Thank you. And our next question comes from the line of Alexander Thompson from Stifel. Your question please.

Hey, great. Thanks for taking our question. Maybe for Scott, could you talk a little bit about OpEx trajectory in 2026 in the context of the CNP launch and then the schedule of the label expansion both with mono and the combo pivotal studies? Thanks.

No problem. We talked a little bit about this at the J.P. Morgan conference event. Using Q4 OpEx as a run rate for the full year is not a bad way to think about it. If things change, we will come in and update you. Overall, everything related to CNP, as we said before, we will come out and discuss more following approval. Yeah. But that is mainly related to the revenue, because

what we have now, we have a really mature company. It is not like we take something in a pipeline, actually take product out of the pipeline all the time. So R&D is basically constant for the last three or four years. Our global commercialization, specifically the direct market that we have built up already now, adding a few more people there, not major impact on anything like that. This is the benefit of a pipeline in the same therapeutic area and scale that we have now.

Thanks. Appreciate it.

Thank you. And our next question comes from the line of Joori Park from Wolfe Research. Your question please.

Hey. Thanks for taking the question. I have one on the competitive landscape. Wondering how you are thinking about this internally as other agents like encaleret are looking to expand into the chronic hypoparathyroidism landscape, and then does your longer-term outlook for Yorvipath include that potential impact from such emerging agents? Thank you.

So

I could be polite, or I can be a straight shooter. I have seen a lot of idiotic ideas. This one is really one of the most idiotic ideas I heard about.

You have patient

that is missing a hormone, PTH, and giving encaleret is not increasing and providing any hormone to this. We are talking about a hormone replacement therapy where you are helping multiple organs, the brain, so you have greater cognitive effects. The bone needs to have the right metabolic system. The kidney needs to have the right phosphate elimination. It needs to have the right calcium absorption. I can continue one organ after the other organ. Then you believe you can take a compound incubator that basically is a calcium sensitizing compound, take it into a person that does not have the hormone, and then you think you have a treatment. It is really one of the most unscientific ideas where I cannot see any meaningful effect that it will help the patient. You can increase the element of one single thing, absorption of calcium, but that is not in any way coming in as a hormone replacement therapy. So no. We have not calculated that in. There is an idea in the ADH1 patient, which have a mutation in the calcium sensitizer 1, it makes sense for this small amount of patients. It makes sense but not for a person that misses PTH.

Got it. Thank you. Thank you. And our next question comes from the line of Dingding Shi from Jefferies. Your question please.

Hey guys, thanks for taking the question. Just wanted to ask, Jan, maybe because you are giving some open thoughts on competitive landscape. Can you discuss your latest thoughts on the CNP competitive landscape, including upcoming FGFR data from BridgeBio and also the earlier-stage long-acting CNP from BioMarin.

I think that is an interesting

aspect cause

we have seen the benefit of CNP therapy for multiple years now. We are seeing it in large patient populations, and one of the things I am 100% aligned with BioMarin on is that the CNP therapy has shown to be extremely safe and well tolerated, except that you have elements like if you take too high concentration, you can get hypotension, you can get injection-site

right. If you are not really encapsulated. When I see the CNP therapy, I understand why BioMarin are trying to copy us and trying to develop a product that is providing a sustained liberation of CNP over one week, because they have seen out from our data how we are highly differentiated compared to vosoritide. That is a completely different case about do we really have a once-weekly product or not. You cannot just take out from AUC. You need to see the profile over one week and other things like that. As I am not seeing these data or anything on the long-acting product for BioMarin, I do not know if anyone can judge that it is a viable product opportunity in any way. We need to see the PK fold, get the half-life, and all the different things, then we can take a judgment about it. The element of tyrosine kinase is a completely different element for me, because that is using a nonspecific action of a compound that is addressing the tyrosine kinase. If you go to the BridgeBio, it is a nonspecific tyrosine kinase that inhibits the three different receptors FGFR1, FGFR2, and FGFR3. This is why it is called nonspecific. I am not worried that you will not see a treatment effect, because when you address the tyrosine kinase, you see an improvement in linear growth because you are inhibiting the superactive pathway. Will we see the same kind of benefit that we see beyond linear growth? That is up to them to show. Can we see an improvement in muscle strength? Can we see an improvement in leg bowing? Can we see all the elements of improved quality of life with that?

But what worries me is the nonspecific thing,

and I really do not care about phosphate. People say, oh, Jan, are you worried about if they have elevated phosphate? First of all, elevated phosphate, you cannot go in and grade it 1, 2, or 3, 4. You need to see on the patient what is the phosphate level before treatment and after treatment, because then you see do the treatment on each single subject have an impact on the phosphate level. If it has impact on the phosphate level, we know it is a nonspecific inhibition

of FGFR1. When you have a nonspecific inhibition of FGFR1, you also have nonspecific inhibition of FGFR2. When you know that FGFR2 is one of the key receptors that is part of the CNS development of the brain, I am extremely worried because it is not something you really see easily in a preclinical model. You do not see it in short-term clinical trials. You see it after three, perhaps four or five years of treatment. That worries me from our patient focus. How can you accept that any patient should take this risk without being extremely well informed about it.

Got it. Thank you for your insight.

Thank you.

This does conclude the question-and-answer session as well as today’s program. Thank you, ladies and gentlemen, for your participation. You may now disconnect. Good day. Thank you for standing by, and welcome to the Ascendis Pharma A/S Fourth Quarter 2025 Earnings Conference Call. At this time, participants are in listen-only mode. After the speakers’ presentation, there will be a question-and-answer session. To ask a question during this session, you will need to press star 11 on your telephone. If your question has been answered and you would like to remove yourself from the queue, simply press star 11 again. As a reminder, today’s program is being recorded. And now I would like to introduce your host for today’s program, Chad Fugier, Vice President, Investor Relations. Please go ahead, sir.

Thank you, operator, and thank you, everyone, for joining our full-year 2025 financial results conference call. I am Chad Fugier, Vice President, Investor Relations at Ascendis Pharma A/S. Joining me on the call today are Jan Moller Mikkelsen, President and Chief Executive Officer; Scott T. Smith, Chief Financial Officer; Sherrie Glass, Chief Business Officer; Jay Donovan Wu, Executive Vice President and President, Ascendis US; and Aimee Shu, Chief Medical Officer. Before we begin, I would 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 Skytrofa and Yorvipath, as well as certain expectations regarding patient access and financial outcomes; our pipeline candidates and our expectations with respect to their continued progress in 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 the results of 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, 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 our forward-looking statements section in today’s press release and the Risk Factors section of our most recent annual report on Form 20-F filed with the SEC on February 11, 2026. TransCon Growth Hormone or TransCon hGH is now approved in the US 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 US 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. Otherwise, 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 agencies. None of the statements during this conference call regarding our product candidates shall be viewed as promotional. On the call today, we will discuss our full-year 2025 financial results and we will provide further business updates. Following some prepared remarks, we will then open up the call for your questions. With that, let me turn the call over to Jan.

Thanks, Chad.

Good afternoon, everyone. With strong execution across our business and continued progress

to

delivering on our Vision 2030, Ascendis is transforming into a leading global biopharma company. We believe this progression demonstrates the power of our TransCon

platform.

And our R&D capabilities to deliver a sustainable pipeline, while our global commercial infrastructure and financial profile continue to strengthen. We believe we are now at the base of a steep curve, where we expect to achieve operating cash flow of around €500,000,000 in 2026 and where we aspire to achieve at least €5,000,000,000 in annual product revenue by 2030. At the same time, we are building an expanded pipeline of blockbuster product opportunities. In the fourth quarter, we saw multiple achievements across the organization, starting with Yorvipath. The fourth quarter was another period of strong execution for the global launch of Yorvipath. Revenue for the quarter was €187,000,000, bringing full-year 2025 Yorvipath revenue to €477,000,000. In the US, access continued to expand. To year end, more than 5,300 patients were prescribed Yorvipath by nearly 2,400 unique healthcare providers, highlighting continued strong and steady demand. To date, less than 5% of US patients are currently on Yorvipath treatment, highlighting the significant long-term growth opportunity ahead. The overall insurance approval rate is about 70% of the total enrollment, and we continue to see this figure moving higher over time. In addition, we continue to see a majority of approvals within eight weeks. This provides a strong foundation for expected additional growth in 2026 and beyond as more patients initiate Yorvipath in line with treatment guidelines that support its use. Outside the US, we continue to reach more patients. As a reminder, Yorvipath is now available commercially or through named patient programs in more than 30 countries. We have full commercial reimbursement in four countries in our Europe direct markets and two countries in our international markets. In Japan, our partner Taisho launched Yorvipath commercially last November. In 2026, we expect full commercial launches in 10 additional new countries. We also advanced development activity to broaden Yorvipath’s label in a number of areas. In the US, we are working to expand and globally, the range of doses to our PATHway-6 trial, and we continue to advance clinical trials to expand Yorvipath to patients under the age of 18. Our work is progressing rapidly on once-weekly TransCon PTH for patients who have been titrated with daily Yorvipath or conventional therapy and have achieved a stable daily dose for a well-defined period. Last month, at the annual J.P. Morgan Healthcare Conference, we shared preclinical data that support the target product profile for a once-weekly TransCon PTH candidate matching the release PK/PD as seen with daily Yorvipath treatment over the entire week, thus providing a comparable efficacy and safety profile.

Overall, we would we remain confident.

That Yorvipath has the potential to be a durable long-term growth driver for Ascendis globally. Turning now to growth disorders. Comprising our once-weekly growth hormone, Skytrofa, or TransCon Growth Hormone, or once-weekly TransCon CNP. Skytrofa delivered another solid quarter with Q4 revenue of €53,000,000, bringing full-year Skytrofa revenue to €206,000,000. This performance reflects the strength and value of the brand. As a reminder, Skytrofa is now approved in growth hormone deficiency in the US and adult growth hormone deficiency. Today, Skytrofa has an overall market share of around 7% in the US. During the fourth quarter, we initiated our Phase III basket trial evaluating TransCon Growth Hormone in additional established growth hormone indications, including ISS, SHOX deficiency, Turner syndrome, and SGA, which comprise up to half of the growth hormone market. Over the long term, these indications represent meaningful opportunity to expand the role of Skytrofa as a treatment of choice in additional growth disorders. We also see an opportunity to potentially expand Skytrofa’s use to novel indications where growth hormone has not previously been approved for use, such as achondroplasia, in combination with TransCon CNP. TransCon CNP is expected to be the first and only once-weekly treatment for children with achondroplasia, providing the full linear growth outcome that can be achieved with monotherapies addressing the overactive FGFR3 tyrosine can. Kinase. In addition, in our pivotal trial, TransCon CNP achieved significant improvement in leg bowing compared to placebo, increasing spinal canal and a similar safety and tolerability profile compared to placebo, with a very low rate of injection-site reaction, and no cases of symptomatic hypertension. In the US, our NDA for children with achondroplasia remains under review with a PDUFA date of February 28. In the EU, the MAA review is underway following our submission last October with a regulatory decision expected in Q4 2026. Recruitment of our ongoing trials in infants with achondroplasia ages 0 to 2 is going well, and we anticipate complete enrollment later this year. Turning to the combination therapy, our 52-week data in achondroplasia underscore the potential power of dual treatment with TransCon CNP and TransCon Growth Hormone. Continuous exposure to CNP enables the benefit of sustained exposure to unmodified growth hormone. In comparison, monotherapy trials of daily growth hormone in achondroplasia delivered only a limited effect on growth and no group-reported benefit on linear growth. Our 52-week data from the Phase II combination trial support our vision to significantly raise the bar for treatment of achondroplasia with linear growth improvements in achondroplasia-specific height score that were three to four times what has been shown with CNP or daily growth hormone monotherapies in the same time period. In addition, the combination trial demonstrated accelerated improvement in body proportionality, and for the first time, a meaningful improvement in arm span has been reported, without compromising safety or tolerability. Importantly, these benefits are meaningful to the achondroplasia community and have been a core objective of our patient-focused development program in both our monotherapy and combination therapy programs. Importantly, all children completed 52 weeks of treatment and remain in the trial, reinforcing the benefit of treatment and acceptable treatment burden of the once-weekly regimen. These Phase II results demonstrate the effect of these complementary therapies, supporting that TransCon CNP acts in synergy with the growth-promoting effect of TransCon Growth Hormone and has positive effects beyond linear growth. We believe over time the standard of care in achondroplasia and other growth disorders long term will include dual therapy as a treatment option. Building on the potential role of TransCon CNP as an essential fundamental therapy, we recently had a successful end-of-Phase II FDA meeting and Scientific Advice Meeting in the EU to align on our Phase III trial for this novel combination approach for treating children with achondroplasia. We also remain on track for additional COACH trial updates including week 78 by midyear and week 104 by year end, and plan to explore further opportunity in other growth disorders.

To sustain doable

long-term growth for Ascendis well into the next decade, we plan to continue to invest in label expansion of our current products in rare endocrine diseases. In addition, we have a strong focus on the development of new blockbuster product opportunities, both inside and outside rare endocrine diseases, to fuel significant product revenue growth in the future. Looking at our partnerships, TransCon technologies support a continuous flow of highly differentiated product opportunities across multiple therapeutic areas, more than we can develop and commercialize ourselves. For this reason, our Vision 2030 includes a focus on creating additional value through partnership and collaboration. Our collaboration with Novo Nordisk for once-monthly TransCon semaglutide continues to advance towards the clinic. Adarx’s TransCon anti-VEGF is on track to enter the clinic this year. In Japan, Taisho received approval for Yorvipath in August 2025, and launched it commercially in November 2025. In addition, VISEN received approval of Skytrofa in China in late January 2026. In summary, 2025 was another positive and transformative year for Ascendis, with two commercial TransCon products continuing to scale, the potential approval of the third high-value TransCon product in the coming weeks, and a growing pipeline of highly differentiated programs. We believe we have the fundamentals in place to deliver global long-term growth. A rapidly strengthening financial profile gives us confidence to achieve an expected operating cash flow of around €500,000,000 in 2026 and our aspiration to achieve at least €5,000,000,000 in annual product revenue by 2030, all consistent with our Vision 2030 strategy. I will now turn the call over to Scott.

Thank you, Jan, and thank you, Chad, for a well-read FLS. The significant achievements we made in 2025 provide us with substantial financial strength to drive our strategic priorities and goals in 2026, which include achieve blockbuster status for Yorvipath, solidify our leadership in hypoparathyroidism through rapid progress of our label clinical trials of TransCon PTH while advancing development of our once-weekly PTH candidate, successfully launch TransCon CNP if approved in the US and other countries around the world, and expand our leadership in growth disorders through clinical and regulatory progress with once-weekly Skytrofa, including in combination with once-weekly TransCon CNP. With that, I will touch on some key points surrounding our fourth quarter and full-year financial results, which we mostly already announced at J.P. Morgan. For further details, please refer to our annual report on Form 20-F filed today. As previously announced in January, Yorvipath delivered strong global performance in Q4 2025, with revenue increasing to €187,000,000, up from €140,000,000 in Q3. Foreign currency had a negligible impact compared to the previous quarter. Total Yorvipath revenue for 2025 was €477,000,000. For the full year, the weaker US dollar negatively impacted Yorvipath revenue by approximately €27,000,000. Skytrofa contributed €53,000,000 in Q4 with negligible foreign currency impact compared to Q3 2025. Total Skytrofa revenue for 2025 was €206,000,000. For the full year, the weaker US dollar negatively impacted Skytrofa revenue by approximately €9,000,000. Including €7,000,000 in collaboration revenue, total Q4 2025 revenue amounted to €248,000,000, and total revenue for full-year 2025 was €720,000,000. Continuing on to expenses, as previously announced, total operating expenses for Q4 were €214,000,000, and total operating expenses for the full year 2025 were €761,000,000, as we previously noted. Operating profit for Q4 2025 was €10,000,000, with Q4 operating cash flow of €73,000,000. As we have discussed for some time, below operating profit the drivers include the noncash accounting related to our convertible notes. Noncash net finance expense, which was primarily driven by noncash items including remeasurement loss of financial liabilities of €106,000,000, was €93,000,000 net. Net cash finance expense, however, for the full year 2025 was about €8,000,000. In future periods, we may introduce a non-IFRS EPS measure adjusting for the impact of certain items to increase the comparability of period-to-period results. We ended 2025 with €616,000,000 in cash and cash equivalents as previously reported, up from €560,000,000 as of December 31, 2024. Turning to our commercial outlook to help inform your revenue modeling for the coming year, for Yorvipath we expect continued strong revenue growth in 2026 based on steady patient uptake with some expected seasonality in reported revenue throughout the year. For Skytrofa, we expect to follow a similar seasonal pattern to 2025 with full-year revenue growth expected to track growth in prescriptions. Longer term, Skytrofa revenue is expected to come through geographic and label expansion. As always, we continue to watch the euro-US dollar exchange rate for any potential impact. Finally, we also look forward to the potential US approval of TransCon CNP later this month, which, as a reminder, has been excluded from this 2026 outlook. With that, operator, we are now ready to take questions.

Certainly. And once again, ladies and gentlemen, we ask that you please limit yourself to one question each. Our first question comes from the line of Jessica Macomber Fye from J.P. Morgan. Your question please.

Hey, guys. Good afternoon. Thanks so much for taking my question. What is your confidence level heading into the TransCon CNP PDUFA? Are you comfortable that the issue leading to the review extension has been resolved to the FDA’s satisfaction? Thank you.

Yes. Can you remember you asked me a question one time, and the J.P. Morgan conference. Can you remember my answer?

I do remember the answer.

And what was your question? You can ask the same question.

I remember your answer, but it was about a different product if I recall, but your answer was yes.

Yes. So this is the same. You asked me, will TransCon PTH be approved, and I said yes. You can ask me the same question today. Will TransCon CNP be approved, and I will say yes.

Okay. Thank you.

Thank you. And our next question comes from the line of Tazeen Ahmad from Bank of America. Your question please.

Hi. Good afternoon. Thanks for taking my question. You mentioned a 70% insurance approval rate in the US so far for Yorvipath. When might you get to 100%, basically?

I think it would be infinity, because I have never seen a product hitting 100%. I think the highest bar I have seen is something like 85%, perhaps up to 90. Where we are today, I am highly satisfied because it is also a compromise about how aggressive you are going into contracting and other things. It is a balance between the two things where in the end, the overall and ultimate goal is to provide most value back to our shareholders and others, and at the same time help the patient to come on treatment as fast as possible. I do not know, Jay, if you have additional comments to my I would not say, pre-prepared remarks.

Yeah. I would say two things. One is that we are very happy with the overall approval rate that we are seeing. I think the speed in which you are seeing this product be covered, again, is a testament to the strong clinical value proposition that we are seeing in hypoparathyroidism. It is the first and only approved therapy in this category. So, again, this approval rating based on where we are today is something that we are very encouraged by. I think to your second part of the question, Tazeen, which is when might you get to 100%, I echo what Jan said earlier as well, which is I do not know that many drug analogs will get to 100%, and that actually has less to do with coverage and also has to do with every single enrollment that comes in. Not every single one of them will be eligible relative to the label. So there is some element of natural filtering that comes that way. More importantly, what I would say is that there are state Medicaid plans, for example, that review things on a staggered cycle. So you will anticipate that some of this will creep up over time, but it will take some time before it continues to inch upwards.

Yeah. Okay. But I think

I think still we need to, some way, this is a US discussion. The US discussion is built on 70% of all approvals in enrollment are there. When you look at an old cohort that perhaps has been six months through it, your actual will get an almost higher number on it. Just to clarify that 70%, that is when you take everyone accumulated. If you take an old cohort, it is much higher. When you go ex-US, the system is quite different. When you get a prescription, in nearly every other country, you are axiom approved. So you can say the 100% yes is basic. When you get a prescription outside US in traditional countries, you will be 100% eligible and already approved for reimbursement.

Got it. Thank you for the color.

Thank you. And our next question comes from the line of Gavin Clark-Gartner from Evercore ISI. Your question please.

Hey, guys, thanks for taking the question. Just on your 8% WAC increase in January. Maybe you could discuss how net pricing will trend this year?

Including how to quantify the magnitude of the Q1 seasonality here.

I do not think we really are discussing net prices. We would love to do it, but we have never done it, and I do not think we ever will discuss net prices. Maybe if I could just ask a follow-up then. Just on

patient enrollment, are you planning to still report those forms going forward for Yorvipath, or maybe just focus more on revenue?

I think in the end, Gavin, is 100% right. We will focus on revenue because now we have been in the market in the US for about four quarters now. When we come to the fifth quarter, I think you have seen a steady state development from 2025 where we basically got an increase in revenue from both US and ex-US of about €40,000,000 to €50,000,000 net every quarter. I think you will see a stability in how we are executing. We still have ex-US. We expect 10 additional countries being fully reimbursed next year. I am sure that is always improving what we call the net revenue we will generate outside the US.

Thank you. And our next question comes from the line of Yaron Benjamin Werber from TD Cowen. Your question please.

Great. Thanks so much. I have a sort of two not really related, but I am going to try to link them to keep it as one question.

Maybe the first one, can you give us a little bit of a sense for Yorvipath? How it is being used out there? When you look at 2,400 unique prescribers and 5,300 unique patient enrollment, is it that each physician has one or two patients in the practice, or are they prescribing it and then going deeper? And then secondly, just at the end-of-Phase II meeting with FDA relating to CNP and growth hormone for achondroplasia, can you give us an update on the outcome and when will you start the Phase III? Thanks.

Okay. That is perfect. I think, Jay, you can give some about how we are expanding the physician prescriber team base, but also go deeper on the different patient, but still are far away to reach the level where we want to be. Jay. Yep. So in the US, I think the question is really around

segmentation and what types of patients are being treated. If you think about the prescriber base, this is where you started your question. We are seeing broad uptake across the entire range of prescribers. To your point, there are some physicians that might only see a couple patients, but there are also some physicians that might see upwards of 10 patients. More importantly, because we are seeing broad uptake across both high-decile and low-decile providers, we are not seeing a major discrepancy as to the type of prescriber that would prescribe, but we are seeing that breadth continue to increase. As it relates to the number of patients per physician, we are also seeing the depth of prescribing per physician increase over time, which again is encouraging. That is both a testament to positive experience with Yorvipath as well as increased awareness of hypoparathyroidism and now there being an option for it amongst patients. The last thing I would say is, when you think about the types of patients that come through, you can look at it in two ways. The vast majority of them are postsurgical, about 70%. The remaining 30% perhaps due to other factors, whether it is genetic, autoimmune, etc., and we are seeing broad uptake across both of those segments, so that is not a major driver. Really where you are seeing some of the earlier uptake is patients that are self-aware of the condition that they have, are linking the symptoms that they have to the underlying condition that they have, and therefore, a combination of them advocating for themselves as well as providers having conviction in the product as well. So all in all, we are seeing broad uptake across provider groups as well as patient segments.

Thank you. And our next question comes from the line of

Joseph Schwartz.

Before you start, perhaps I can answer the last part of the question related to the COACH trial. Yes. We had extremely positive meetings from the US side and from the EU side. It was really impressive feedback because of the data. They have never seen data before that are providing this kind of benefit to an achondroplasia treatment. I am not only talking about the linear growth where we basically on z-score got a three to fourfold more than you can see with monotherapies in the same time period, but also unique elements like such an improvement in body proportionality. What really they have never seen in such a meaningful manner was a really important element, the arm span, where we also saw in the combination trial a unique improvement in arm span. Aimee is sitting here, and she is really doing everything to get this trial recruited as fast. We are ready to go. Protocol is finished and everything. Be open site. Just remember that our pivotal trial in monotherapy, we recruited it just in three or four months, just because of the interest of the patient. Therefore, the bar for Aimee is very hard if she needs to do that faster. Sorry for coming in.

Absolutely. Not a problem. Our next question comes from the line of Joseph Schwartz from Leerink Partners. Your question please.

Hi. This is Heidi Jacobson on for Joe Schwartz. Thanks so much for taking our question. Can you help us understand how the TransCon CNP launch could factor into your $500,000,000 operating cash flow target for 2026, particularly with respect to launch investment and early revenue contribution?

Thanks.

It is pretty simple. It is not incorporated. When we are coming into the launch, we see the initial uptake, which we believe will be pretty high, not only in the US but also outside US because we can utilize the US approval to go to countries outside US, especially in the international market. From that perspective, we will come and provide you a better guidance and improved guidance when we have seen that. Scott is smiling, are you counting money or what are you doing?

Taxes and money.

If you will come back after that.

Great. Thanks.

Thank you. And our next question comes from the line of Derek Christian Archila from Wells Fargo. Your question please.

Hey, good afternoon. Thanks for taking the questions. I wanted to understand your confidence level around Yorvipath growth ex-US. Obviously, the launch in Germany and Austria, is that a good proxy? Or is it going to be more depth in those types of countries than just expansion in, I think you said, 10 additional countries. How will that be sequenced through the year? Thanks.

That is extremely complicated because the heterogeneity of ex-US is so heterogeneous that we cannot compare what we see in Spain now, what we see in France, what we see in Germany, what we see in Austria. It is different because we see different speed of penetration. For example, Germany has fewer endos, so the bottleneck is tighter. It takes longer to get them on therapy because there are fewer in the general population. If we go to Spain, there are more. There are more in France, and we also see a faster uptake because the pipe is larger. When we get 10 more additional countries on full commercial, we will see different uptake, but what we are doing is everything will be accumulated in the way where we now see from 30 to 35 countries named patient programs. When we go full commercial, everywhere we see an acceleration of patient uptake because of the burdensome nature of a named patient program. It takes so much effort to get every single patient on it, and every patient deserves to be under treatment. In 2026 we will see initial speeding up in all these countries. In 2027, 2028, you will continue to do it because by nature, we just got approval now in Canada, and we are taking one country after the country, first getting approval, and then putting reimbursement.

Our next question comes from the line of Li Watsek from Cantor. Your question please.

Hey, thank you so much for taking our

question. It is Daniel Brondo on for Li. Can you give us a little bit of color on how you expect your TransCon CNP launch to go? It seems like there are a few patients who are not currently on treatment. Where do you think you will capture the majority of patients initially?

Pretty clear.

Improvement that we see with TransCon CNP to what we can provide. Not only related to tolerability injection-site reaction, having one in 20 injection-site reaction compared to one every second year, being in a position to look at no risk of hypertension, the element of improvement on the once-weekly product, and then show the data package that we have generated with TransCon CNP, for first time ever shown in a well-controlled trial against placebo, benefit beyond linear growth. For example, the leg bowing, which we have shown multiple times, we have shown improvement in muscle strength. We have improved quality of life. I think this is obvious. Every patient that decides to be on treatment should have the opportunity to have the best possible treatment option, and I think there is a public interest in the US to ensure that this always will happen.

Thank you. And our next question comes from the line of Eliana Rachel Merle from Barclays. Your question please.

Hey, guys. Thanks so much for taking my question. Curious if you can elaborate on your strategy for commercializing TransCon CNP ex-US. Given the majority of vosoritide sales are ex-US, could you elaborate on your strategy for the commercial launches globally and the degree of investment that will take? And second, on TransCon CNP in the US, can you talk about cadence of uptake and which segments do you expect the most uptake from between treatment naive versus switches? Thanks.

Yeah. I will dive a little bit back now because

what we did when we said that we want to have global commercial

effort.

We actually started all our infrastructure building to Yorvipath. Now you see what we have done with Yorvipath. We recognized their fast revenue, commercial revenue for more than 30 countries. We are penetrating them exactly as we can do. We will reach 60 to 70 countries in less than two to three years. We already have built up the infrastructure to be ready that we can take our integrated pipeline of rare disease endocrine products into all these different countries in the setup via step links around Yorvipath. This is the positive element that we are not a company that needs first to build infrastructure to support globalization. We have already established that. So I feel really confident all the success we see now with

Yorvipath on a global scale,

we will just take it in. Do not forget, for example, even in Japan, the collaboration we have with Taisho is for all three products, the same in China and other places. When we make these agreements, we are not making single product, single country. We make it as a pipeline

product. And this is why we do not need to go out and make new agreements or anything.

It is just going to be done extremely fast from that perspective.

Thank you. And our next question comes from the line of Leland James Gershell from Oppenheimer. Your question please.

Great. I want to ask, as we look at the €5,000,000,000 number you have put out there for product sales by 2030, you are not giving specific product guidance, but if you could share how you think about the relative contributions of your presumably three products by that point in time, in terms of how they will weigh into that total sum. Obviously, you have much more expansion opportunity in hypoparathyroid, you have TransCon CNP potentially launching soon, and Skytrofa perhaps getting additional indications in combination. I would love to hear

with those three parts. Thank you.

maybe just philosophically how your outlook adds up

Yeah. That is an element where we always in our forecasting are operating under different assumptions, where we are building models for each country and then aggregate globally. We first take 2026, then 2027, 2028, and 2029. Then you always look at the risk balance. Where do we have potential extra upside that we can explore? But what makes Ascendis unique today is that we are not a single product in a single region. We will have three approved products in perhaps 20 different indications in about 30 to 40 countries. We will not be dependent on one single product in one single region. This is how we build a sustainable company that has a continued stable revenue flow for multiple years. Do not forget these product opportunities. When I look at the pipeline for each of them, I definitely do not have sleepless nights. There is no doubt that when I see the profile and how we design it to be best in class, we also see that realized. From that perspective, it is a combination of products with IP lifespan extremely long. This is where you have the durability of it. This is why we take the value perspective of each single product opportunity instead of fast revenue. This is not how we operate. We go for value, because this is the product really deserved as treatment because we are providing not only a unique benefit for the patient, but also for the society and everyone.

Great. Thanks very much.

Thank you. And our next question comes from the line of Paul Choi from Goldman Sachs. Your question please.

Hi, good afternoon. Thanks for taking our question. I think your Phase II REACH IN study is scheduled to reach primary completion next month. Will you be in a position to file an sNDA for the newborn infant population this year? In terms of the newborn infant population, in your discussions with the FDA and EMA for your Phase III combination study, does your study design allow for those newborn patients to be included in the study population, or will that require a separate study? Thank you.

I think when you discuss a label discussion, that typically is different between the main regulatory areas. If you take, for example, US, it is much harder to come to a situation where they will

accept

a label expansion to the infant without having the data in hand. In Europe, it is more flexible because you have a discussion with them, and you can have what I call rolling addition of data that is being generated to our trial. There will likely be a difference between the three geographic regions. Simplified, Japan is perhaps the easiest way to get it down to infant immediately. What we are doing now is to ensure we generate the right data, and we are doing that in a trial. It is a placebo-controlled trial, and what we see is everything we hope for. It is living up to our expectation. Why I can say that? Because in the enrollment, we have six patients on a, what I call, visible treatment. You take them in, and there is no randomization. You can follow them, and Aimee can tell a few words about the benefit we have seen from that perspective.

So, Jan is talking about the sentinel kids who are not part of the randomized piece of the study. They are doing well, tolerating the medicine as well as we would expect, growing, and starting to see early signals of other benefits as well, particularly radiology.

Yeah. So we are really so pleased with the progress we are doing in helping patients with achondroplasia, not only on linear growth, but also benefit beyond linear growth.

Thank you. Our next question comes from the line of Yun Zhong from Wedbush. Your question please.

Hi, good afternoon. Thank you very much for taking the question. My question is on the weekly TransCon PTH. Is it reasonable to expect that the program could potentially enter the clinic in 2026, or do you think that there is no such need to rush? Also, you mentioned matching PK to the daily product. With data from Yorvipath available, what do you see as the most efficient clinical pathway to maybe take the weekly PTH to approval?

I think what you are addressing is two things that are interconnected. If you can show the PK profile, and it can even be healthy volunteers or patients with hypopara, that over the entire week of treatment you are bioequivalent to Yorvipath, that is the aspiration how we designed it. You will always be in an excellent PTH level compared to your Yorvipath daily dose for the entire week. Then we know you will get the expected safety, the expected tolerability from that perspective. This will make a much more simplified, easy way to conduct the clinical trial. It is why we designed it in this manner.

Okay. Thank you very much.

Thank you. And our next question comes from the line of Luca Issi from RBC. Your question please.

Great. Thanks so much for taking my question. Congrats on the progress. Maybe, Jan, big picture, one of the goals for 2030, as you articulated at J.P. Morgan, is to remain an independent and profitable biotech company, and we have seen many successful examples of that recently. However, how do you think about continuing that same vision under the broader umbrella of a larger pharmaceutical company? How are you thinking about strategic path A versus strategic path B at this point? Any color appreciated. And then maybe, Jay, quickly, I think BioMarin has announced that they will file vosoritide for full approval versus I believe you will initially get approved on an accelerated approval basis

for TransCon CNP. How should we think about that difference? Will that have implications for formulary access and reimbursement? Do you not view that difference as material for adoption given you have a less frequent dosing versus—thoughts about that? Excellent. I think I have been I think I will liberate Jay for answering the last

question. I think when I look at this discussion on accelerated approval that BioMarin is filing for, that has no

impact

on our regulatory pathway and approvals and other things like that. Totally independent. It is not any way how you can build up any barrier. The second thing, yes, in our vision there is independent, and I believe that is a great word because we want to be independent like a teenager growing up. I have four children. I am teaching them when they are going to be 18, you need to be financially independent as the first element in their life. I think that is a great thing to see Ascendis Pharma now moving away from being a teenager but basically can go up to a more adult life, because we have shown now we are completely independent on asking investors and others for any kind of revenue. I think this is how we see independency.

Super helpful. Thank you.

Thank you. And as a reminder, ladies and gentlemen, we ask you to please limit yourselves to one question. You may get back in queue as time allows. Our next question comes from the line of Maxwell Nathan Skor from Morgan Stanley. Your question please.

Great. Thank you very much. My question was asked, but I will take a shot at this. Could you give any color on the once-monthly TransCon semaglutide program? Any gating factors? When should we expect an update? Any additional color would be helpful. Thank you.

Yeah. Let me go back to all the elements and all the IP we have done, files and data and everything like that before we went into this extremely productive collaboration with our neighbor in Copenhagen, Novo Nordisk. What was the idea behind once-monthly semaglutide? The idea was to be sure that you can get fast weight loss and at the same time have a high level of tolerability. Think about a naked GLP-1 molecule. When you give it weekly or potentially want to use a weekly product once monthly, you need to add much more compound to compensate for the half-life to have a large AUC. By doing this, you add a high Cmax. Because it is naked, you will have a very short Tmax, meaning that you will have a steep curve from the lowest level just before you start to give a dose up to the maximum concentration. That often gives the tolerability issue where you get the element that limits people to stay on treatment and what you can achieve. This is what I call the naked product. This is like metacresol and everything. It is a naked protein. What we are doing now is defining what we call packed-in semaglutide. Even if you give it high dose, you liberate it slowly, slowly, slowly, so you are getting a very long Tmax. By doing that, you have a slope that is not as steep at all as you see with a naked molecule. You still have a big AUC because you provide so much compound, give it over the entire month, and at the same time you do not have this steepness in the slope. It was designed to have maximal weight loss as fast as possible with the best tolerability profile, and it was how we designed it at that time.

Thank you. And our next question comes from the line of Alexander Thompson from Stifel. Your question please.

Hey, great. Thanks for taking our question. Maybe for Scott, could you talk a little bit about OpEx trajectory in 2026 in the context of the CNP launch and then the schedule for label expansion both with mono and the combo pivotal studies? Thanks.

No problem. We talked a little bit about this at the J.P. Morgan conference event. Using Q4 OpEx as a run rate for the full year is not a bad way to think about it. If things change, we will update you. Overall, everything related to CNP, as we said before, we will come out and discuss more following approval. Yeah. But that is mainly related

to the revenue because what we have now, we have a really mature company. It is not like we take something in a pipeline, actually take product out of the pipeline all the time. So R&D is basically constant for the last three or four years. Our global commercialization, specifically the direct market that we have built up already now, adding a few more people there, not major impact on anything like that. This is the benefit of a pipeline in the same therapeutic area and scale that we have now.

Thanks. Appreciate it.

Thank you. And our next question comes from the line of Joori Park from Wolfe Research. Your question please.

Yeah. Hey. Thanks for taking the question.

I had one on the competitive landscape.

Wondering how you are thinking about this internally as

other agents like

encaleret are looking to expand into the

chronic hypoparathyroidism landscape. And then does your longer-term outlook for Yorvipath include that potential impact from such emerging agents? Thank you.

So

I could be polite, or I can be a straight shooter. I have seen a lot of idiotic ideas. This one is really one of the most idiotic ideas I heard about. You have patient that is missing a hormone, PTH, and giving encaleret is not increasing and providing any hormone to this. We are talking about a hormone replacement therapy where you are helping multiple organs, the brain so you have greater cognitive effects. The bone needs to have the right metabolic system. The kidney needs to have the right phosphate elimination. It needs to have the right calcium absorption. I can continue one organ after the other organ. Then you believe you can take a compound, incubate, that basically calcium-sensitizing compound, take it into a person that does not have the hormone and then you think you have a treatment. It is really one of the most unscientific ideas where I cannot see any meaningful effect that it will help the patient. You can increase the element of one single thing, absorption of calcium, but that is not anyway coming in as a hormone replacement therapy. So no. We have not calculated that in. There is an idea in the ADH1 patient which has a mutation in the calcium sensitizer 1. It makes sense for this small amount of patients. It makes sense but not for a person that misses PTH.

Got it. Thank you.

Thank you. And our next question comes from the line of Dingding Shi from Jefferies. Your question please.

Hey guys, thanks for taking the question. Just wanted to ask, Jan, maybe because you are giving some open thoughts on competitive landscape. Can you discuss your latest thoughts on the CNP competitive landscape, including upcoming FGFR data from BridgeBio and also the earlier-stage long-acting CNP from BioMarin.

Yeah. I think that is

an interesting

aspect

because we have seen the benefit of CNP therapy for multiple years now. We are seeing it in large patient population, and one of the things I am 100% aligned with BioMarin on is that the CNP therapy has shown to be extremely safe and well tolerated, except that if you take too high concentration, you can get hypotension, you can get injection-site

right.

If you are not really encapsulated. When I see the CNP therapy, I understand why BioMarin are trying to copy us and trying to develop a product that is providing a sustained liberation of CNP over one week, because we have seen from our data how we are highly differentiated compared to vosoritide. That is a completely different

case about

do we really have a once-weekly product or not? You cannot just take out from AUC. You need to see the profile over one week and other things like that. As I am not seeing these data or anything on the long-acting product for BioMarin, I do not know if anyone can judge that it is a viable product opportunity in any way. We need to see the PK profile, get the half-life, and all the different things. Then we can take a judgment about it. The element of tyrosine kinase is a completely different element for me, because that is using a nonspecific action of a compound that is addressing the tyrosine kinase. If you go to the BridgeBio, it is a nonspecific tyrosine kinase that inhibits the three different receptors FGFR1, FGFR2, and FGFR3. This is why it is called nonspecific. I am not worried that you will not see a treatment effect, because when you address the tyrosine kinase, you see an improvement in linear growth because you are inhibiting the superactive pathway. Will we see the same kind of benefit that we see beyond linear growth?

That is up to them to show, can we see an improvement in muscle strength?

Can we see an improvement in leg bowing? Can we see all the elements of improved quality of life with that? But what worries me is the nonspecific thing, and I really do not care about phosphate. People say, oh, Jan, are you worried that they have elevated phosphate? First of all, elevated phosphate, you cannot grade it 1, 2, or 3, 4. You need to see on the patient what is the phosphate level before treatment and after treatment, because then you see, does the treatment on each subject

have an

impact on the phosphate level. If it has impact on the phosphate level, we know it is a nonspecific inhibition

of FGFR1.

When you have a nonspecific inhibition of FGFR1, you also have nonspecific inhibition of FGFR2. When you know that FGFR2 is one of the key receptors that is part of the CNS development of the brain, I am extremely worried because it is not something you see easily in a preclinical model. You do not see it in short-term clinical trials. You see it after three, perhaps four or five years of treatment. That worries me from our patient focus. How can you accept that any patient should take this risk without being extremely well informed about it.

Got it. Thank you for your insight.

Thank you.

This does conclude the question-and-answer session as well as today’s program. Thank you, ladies and gentlemen, for your participation. You may now disconnect. Good day.