Ascendis Pharma A/S (ASND) September 2026 Conference Transcript

Hello, everyone. I'm Max Gore, a biotech analyst with Morgan Stanley. Welcome. And just to start off, for important disclosures, please see the Morgan Stanley Research Disclosure website at www.morganstanley.com forward slash research disclosures. And with that, I'd like to welcome Yen Mickelson, CEO, Scott Smith, CFO, Jay Wu, head of U.S. Market. Thank you very much for joining us today, team.

And thanks a lot for inviting us.

Really appreciate it. So I think in regards to the Ascendis story, let's start with last night's announcements. You've recaptured full rights to the metabolic and cardiovascular franchise. How are you thinking about what comes next for Ascendis?

That is an open question where I can come up with a lot of answers because a lot of things is happening at Ascendis. First of all, we also had a Presco lease yesterday related to buying back shares for 400 million US dollars. And I think that is part of our integrated strategy that we lay out in our vision 2030, how to be a leading biopharma. And before I start, I think Scott would like to say a few words related to our financial positioning. how we are generating a profitable company, and how we will progress in the next years.

Thanks so much, Ian. As Ian mentioned, we announced a $400 million share repurchase agreement, and that's really on the back of the strength of the financial transformation that happened with Ascendas this year in 2026, going from historically R&D to having approval and launch of three highly differentiated products. with Yorva Path achieving blockbuster status this year. As you know, we've guided to over 500 million euro of operating cash flow. And with the strength of the launch as well as the new launch of UVWL, we expect that that could potentially double next year or even more. So it's a great time for Ascendist to buy back shares. And we still retain all the capital we need to continue to advance R&D. And in particular, I think with the other announcement we made, it gives us a lot of opportunity to basically ensure the growth of Ascendis beyond 2030 into the 2040s.

So when I see Ascendis, and this is laid out really well in our Vision 2030, because I really like this framework where we go out and lay a five-year plan and how we follow on and how we execute it. So there's two cornerstones in our 2030 plan. One is to hit $5 billion in product revenue. That is $5 billion coming from our three approved product, SkyTrofa, URpants, and URVAL. That is the cornerstone in our revenue generation. And it's coming from U.S. and also coming from our key effort. We have to be independent on the U.S. revenue, but building up a sustainable part of our revenue flow from the ex-U.S. I think we're one of the few biotech that really have this effort going on, where we're building up a global organization. We recognize revenue more than for 40 countries now. And it takes longer time in the ex-U segment. So when we come to 27, 28, we will see about 10 to 20 countries every year being full commercial with our product. Jay will say a few words about U.S. because U.S. is still a cornerstone in our revenue generation. And also explain a little bit of the effort we're doing really so we can grow the revenue every year.

Sure. Thanks, Jan. As Anne mentioned, you know, from a U.S. standpoint, we now have three approved products in the U.S. We're feeling encouraging about all three of them, right? So YorvaPath, I know we spent a lot of time talking about, given from a pure scale standpoint, it represents tremendous opportunity as you think about not only what it's generating in the near term, but also the longer term potential of the hypoparathyroidism space overall. Within that, we've been investing quite heavily in a few areas. So one, we've talked a lot about prescriber education as being a key area. We launched during a time where following that para, this is a differentiated product and we're reshaping people's expectations for what patients that have hypoparathyroidism need and what the condition actually entails. I think the second area where we've been investing quite heavily in is patient activation. If you think about 70,000 to 90,000 patients in the U.S. having hypoparathyroidism, This is a space where many of those patients aren't actively either seeking care, have established care with their specialist, and or are searching for something more above and beyond the conventional therapy that they're on. And that takes time, but we're seeing some really encouraging signs of some of those behaviors and beliefs changing. And then lastly, with the ever-evolving access landscape patient experience, we know that this condition in particular, these patients go through things like brain fog, they might go through fatigue, and making sure that they have the requisite level of patient support, both during their journey to get a prescription, as well as post-prescription, is another area that we've been ensuring that we're supporting from a seamless experience standpoint. you well we just recently launched this year you've seen some of the numbers that we've shared previously incredibly encouraged by what we're seeing with that new product launch to date and again really speaking to the fact that this is a clinically differentiated profile we believe is best in class and from a standpoint of offering something unique to this community we're seeing really positive sentiment across providers patients patient advocacy groups and conversations with payers have all been going very well. And then, of course, we mentioned the cornerstone of SkyTropha. We're still seeing continued growth in that segment, tracking with what we see in prescriptions. And then because SkyTropha is continually being developed in other areas as well, we do see SkyTropha as a foundation to start with a very bright future ahead.

So what Jay has talked about, how he implement an excellent center of commercialization in the U.S. We're doing the same ex-U.S., building up a global organization where we want to reach basic more than the 400,000 to 500,000 patients, for example, with hyperpera. That is sitting ex-U.S., huge amount of patients. And this is one of the efforts we will continue to do. So when we talk about revenue in 2030, the $5 billion, we have a strong fundament to achieve that just with our three current products. This is the fundament to hit the $5 billion. This is not peak sales of this portfolio. The peak sales comes years after that. We continue label expansion. We have to attend trial ongoing now. Many of them are late-stage, both in label expansion, combination therapy, really to build on further how to really reach the best kind of treatment to all the different patients. At the same time, and it's coming to the second part in our Vision 2030, how to be sustainable with a continual new flow of new chemical entities, like one like Scythofa, like one like Europass, like one like Uoval. And we have now a strategic strategy to take at least one new NCE in clinical development, besides all the label expansion we continue to do with our current portfolio of products. And it's coming both in rare disease endocrine, it will come in other indication outside, and part of our strategic approach for 2030 is that we have built a new fundament for a new therapeutic areas beside our rare disease endocrine. And we are not limiting ourselves to rare diseases more. We have the capacity as a company, we have the infrastructure to move outside rare diseases, and when we see how we built up our rare disease endocrine, It was built on a product, at least three, where we add one product more after the time. Really have the economy of scales in synergies, both in commercial organization, in manufacturing, regulatory, XFCs, really to do it. And we would do the same thing at a new therapeutic era. We are not dependent on in-licensing. We are a company built on an R&D development on our strong technology platform, the Transcon technology, which have given us three out of three. Scott is still calculating what is the chance to start with three preclinical and get three product approved. He still believes it's very, very, very, very low. And think about all three of them are highly differentiated. All three of them really showing best in class compared to anything. All of them are extremely doable. They have IP up to the end of the 30s, in the 40s. They are a combination product. They don't have a patent cliff like a small tablet. They're just falling down the cliff one year after its basic item position, their IP disappear. We own the products. So we're not ending it like other companies that were royalties suddenly disappear the day after their IP disappeared, but the product stands for 10 years more. We will own the product because we do own commercialization. So when you look on the future of Ascendis Pharma, we're just in the beginning of the beginning. We're not even in the end of the beginning, because the first therapeutic era, we're first starting to mature, first starting to grow. So this is why I really believe when you ask me this question, this is a long answer, because we have so many possibilities we're doing so many things but I'm so happy to the stakes we're coming. We are independent that we wrote in our Vision 2030 and independent meant that we are developing everything from the transcom technology and be financially independent and we can give a billion back potential every year to the shareholder if we want to do that.

Okay, maybe if I can ask on the cardiometabolic effort or what it looks like going forward, capital expenditure. Does the base business fund the expansion? Should we think about any sort of equity raise? Trying to think about it through the lens of the share buyback also.

Are your bankers in the meeting?

I think the bankers are a little bit disappointed. First of all, Scott is never getting invited out to dinner from bankers now because somewhere all of them have given up that we're going to raise capital. So we're not really interested in more. We say tax and dollars are still being invited here. We are not going to raise capital, I can guarantee. And when you see our company, how we are as a company, most people expand R&D expenses because they don't take product out. Hey, we take one product out all the time. And when it's out, they're not sitting in the pipeline more. We take new one. So when I see the expenses on our basic research and development, We are pretty stable. We will have a growth every year. It will be dependent on inflation. It will be dependent on a few other elements, possibly expanding a little bit more and other things like that. But it's not making material difference. Scott gave me a simple algorithm. If we grow revenue 100% year by year, and we only grow expenses by 10% to 15%, would that give a lot of profit? And Scott convinced me about that. And this is basically the story we are implementing for the time being. So we're not raising capital. Sorry for MS. No morning flood next year.

I'm very happy you guys are here. In regards to the cardiometabolic opportunity, though, should we think about it as potential upside to the Vision 2030?

This is a major upside because I want to be independent. We have multiple collaborations. All of them have actually failed. But we've been extremely successful as a company. I don't think anyone should be dependent on a big pharma. That is really a big mistake. The first major collaboration we had with Sanofi, we made the first triple agonist once weekly, but then they got a new CO, and he didn't like metabolic diseases. If that had continued on that, they likely had been larger than Lily today. Then we basically made ophthalmology with Roche Genetic, and that went into a CMC problem, which we solve after Voss. But now we have Iconics. Mark is sitting there with the CEO of Iconics. Our major spin-off, extremely successful. So the products are still fantastic. And then we have now a collaboration with Novo Nordisk. And in every contract, there is a way how to live together, how to work together. And if one is not fast enough, the other one likely has a chance to go out of it. And I think we will get it back, which gives us a lot of opportunities, not only to have the best-in-class once-month semiglutide, but also have the possibility to access rare diseases in obesity and other metabolic diseases, which we couldn't do before to the same degree. So it opened up an extremely positive for us to really expand our opportunities in this way.

I know it's early, but can you just talk about timelines and capital allocation towards this cardiometabolic effort?

The capital allocation, it's basically going to be in a combined phase one, phase two trial, which we will give you timelines on when we come to the beginning of next year. And it's not changing any way, material, our expenses in any way. So it's not going to be a driver of a new racing capital because that is definitely not needed from our side.

Okay. Maybe pivoting a bit, let's talk about your V-Path, which really anchors your vision 2030. What are you doing to continue the momentum, potentially accelerate growth? What should we expect over the next 6 to 12 months?

Jay can take yours.

You know, when you think about your V-Path, Transformational change from where you are requires something beyond just prescriber education, classic reach frequency, et cetera, right? Because 70,000, 90,000 patients with the number that you're treating, you actually need to get patients to raise their hand and come out of the So a lot of our investments and commercial focus has been around that piece specifically. So I'll give you a few examples. We've been looking at direct consumer education. You've probably seen some of that depending on the demographic and the shows that you watch or streaming online or however, but we are starting to see some preliminary positive signs from some of those investments that we've made from a digital standpoint. We've invested heavily in patient-facing roles, so we now have an entire infrastructure of roles that we call patient access liaisons. They coordinate with patients directly. They support them in all aspects, whether it's field reimbursement, clinical education, and essentially supporting them through their journey, both in terms of wanting information about the therapy, accessing therapy, as well as supporting them once they're on therapy. And then lastly, when you're starting to think about digital infrastructure for more commercial stage organizations, how do you have channels increasingly embedded within one another, right? So from diagnosis to field personnel to non-personnel digital channels, how do you have them all work together in a way where you're offering people a seamless experience so that they get the information they need at the right time and in the right way. So those are three examples for which we have been actively investing in activating patients more broadly. And again, we're seeing some encouraging signs and it's really an investment and not just today, but in the future as well.

From the XUS, on the top lip is to make more and more countries fully commercial. Today, we have about seven, eight countries, ex-US, that is fully commercial. Next year, we will add between 10 to 15, 27, and we will do that again in 20. For all perspective, besides each country have their own, you can say, execution, commercial operational execution, is on a high level to get as money as possible country full commercial where we still keep the value of our product because we believe the value we're providing with, for example, Europe has providing a creation not only for the patient but also for the society and this is why we not compromise really the value what it really costs to basically implement such a treatment in each country.

Should we expect the investment in the YorviPath launch to increase over time? How does that look?

I think the full-blown of where we can basically be in this way, being in countries, we have a mixed model in ex-US. In countries, we are direct. We basically have built up all the legal entities that affiliate the basic people on ground. In the countries where we have distribution agreement, we are basically not responsible for any further expenses in this. So out from that, you can say it's a mixed model where we basically have built up the infrastructure supporting a global launch in all the countries that is needed.

Great. And I know we have talked about the benefits of once daily, being able to hydrate with your repath, but can you give us any developmental timelines for the once weekly trans-con PTH candidate?

First of all, the once weekly product is not an LCM activity. It's providing a choice to patient that is stable on your patch. Meaning is that when you think about a patient journey, You come with a diagnosis of chronic hypopera. You are on conventional therapy, which consists of two compounds or two treatments, active vitamin D and a lot of calcium supplements. When you come out from conventional therapy over to your past, you actually go to a titration, where you remove first the active vitamin D and then you remove the calcium supplement and then you step up the uropax dose. And when you are stable on that, you basically can be in a position that you have a lifestyle where you're not changing your activity level a lot. You basically have some of the same dietary components and other things like that. Compared to diabetes one, If everyone had the same amount of carbohydrate every day, exercise in the same amount, then you will be really well on a stable insulin level. But if you change your activity level, get an infection or a disease, change your dietary component, you need to be sure you can titrate up and down. And there was a why. I got asked once, you make scartofa once weekly. You made trans-con-CNP one weekly. Why did you not make PTAs once weekly from the beginning? Because we know the complication in the titration phase. And there is not an antagonist to high level of PTAs. It's not like insulin where you can take glycogone. Don't do that. There is only one thing to go to emergency room. So out from that perspective, if we know they're getting hypercalcemic, they just stop treatment in one or two days, and then they're down slowly, slowly again. And that's why we never wanted to start with a once-weekly product. But if a patient has a more stable lifestyle, or over a period, have a stable dose, and they want to go over on a once-weekly product, we will develop a once-weekly product, which we're doing now, that basically will give them the same properties that you get with your pets. And industrious replacement therapy, because the other once-weekly is not an industrious replacement therapy, only have half of the effect that you get with normal PTAs. So it's not really a substitute for your pets. So forget that. So from that perspective, we want to have a once-weekly product building up exactly the same active mode of action that you have in your pads with basically the same level of PTAs 24 hours to 7 days a week. So it basically is bioequilient with daily your pads treatment.

Great. Now let's pivot over to UVWell. How is the launch going? What can we expect in regards to patient ads through year-end?

But see, Jay, he looks such a happy person. and extremely pleased I was there he's always negative but when he saw all the numbers he even needed to be positive yes now he needs to be positive yes we're very encouraged by what we're seeing I think what we last showed again we last reported 220 enrollments over 100 prescribers if you even take that comparing it to previous analogs that's quite outpacing I think what we've seen in the past as it relates to incumbent or older therapy. So that's corroborated by not just, again, I said before, qualitatively beyond just what we're seeing in the numbers, is there's just a high level of enthusiasm, right? A lot of patients that are on it, this is an ultra-rare community, there's word of mouth, and I think you can measure a lot of leading indicators through being present, whether it's conversations with advocacy groups at congresses, and I think as you continue to see patients see their specialists throughout the year, knowing that this is an area where it's not like they just go in the next day. We do anticipate that we will see continued growth in an encouraging way from what we've seen to date. I also, when you look at the space in achondroplasia specifically, it is very center of excellence based in terms of where the patient concentration is, which is very different when you look at it relative to like a hypopera where it's much more diffuse across a right tail of accounts. So for achondroplasia specifically, we've also been investing quite heavily in going on an account-by-account basis and understanding what is the unique needs of each local regional account, which is a very different type of approach for an ultra-rare category versus something that you might see in PTH. So again, all these things qualitatively, leading indicators. We're feeling really good about where we are and where we'll continue to be.

Any comment on the mix of patients? Is this, I know we talk about it on the earnings calls, et cetera, but new starts, switches, patients who potentially discontinued Voxago and testing, any comments around that?

Yeah, I mean, I think what we've shared before, just to reiterate, we see patients anecdotally coming across three segments. We have patients coming across patients that are currently on vasortide that have switched over. There's a second category of patients that were previously on vasorotide but have since discontinued, maybe because they weren't seeing the efficacy that they wanted, maybe that from an injection site reaction they didn't feel it was tolerable, and they decided to discontinue vasorotide but then wanted to start Uvoel. And then the third one, which is patients that have been holding out because they weren't convinced by the older therapy on market, now that there's a new option, they're like raising their hands like, now I'm willing to try and want to try. I think with any type of second product coming in, you naturally should expect that probably there's a greater number of patients that have been on existing therapy that are hand-raising to move over. So while we don't necessarily track that with specificity, I do anticipate that earlier on we're going to get more of the patients that are previously on therapy. But anecdotally, we see it across all three segments.

Any comment on gross-to-net over time? How should we think about that for you, B. Will?

Yeah, I would say we don't usually disclose gross-to-net over time. That's probably the best way to think about it.

Okay. And maybe if we could just touch on the recent agreement you signed with Biomarin. If you'd like to lay out any comments on that. I have a few follow-up questions, but we can go from there.

The content of the agreement is that we will pay royalties in a limited number of countries until May, and it gives us a global freedom for all IPs from Biomarine related to specific patents. And from my perspective is that we won the case in Europe. For me, it's not really what is right, what is wrong. I'm a problematic person there. Out of two things, I actually prefer to pay to Biomarine instead of paying to the lawyers. At least I feel a little bit better with that. So out from that perspective, it's a total win for both companies in the way that the biomarines get compensation for the loss of revenue that they get in specific countries. And we have a freedom really to go out and really come out to as many as possible patients immediately and be in a position that we're really building up the franchise to what we believe best-in-class opportunity. This is the only product, URVL, that has shown really benefit beyond linear growth in a placebo-controlled manner. And also when we look on how we develop this franchise further on with the combination therapy where we combine it with scythropha, we basically will provide a treatment option to this patient group. First, a contemplation where the basic can avoid basic all elements of surgeries, everything from spilling stenosis to arm lengthening, lengthening, leg bowing, really key elements that really have a major impact in a negative manner on this patient's life. So out from that perspective, we feel that we're making a complete new standard on treatment, not only with Urovel, but also what that's really being created as a possibility treatment option with the combination therapy. We now have 18 months' data in the combination therapy. Never seen results like that. You basically generate three to four years' treatment in one year, plus additional things that we now see the same effect as with monotherapy, like arm lengthening and other things like that. We're coming out now with the newborn data, children down to newborn, and we have the first data cut for the first seven patients that came in, and exactly as we had hoped for. But the key element is safety. Never forget that. And I think when we look at the safety perspective of the CMP-based therapy, is really providing no safety sickness. One of the safest treatments I ever have seen would give an obvious choice for the parents and the patients.

Great. The royalty agreement runs through 2030, as you said. How should we think about margins beyond that?

At least from my side, you'll see there will be some impact in the next quarterly report, and you won't even know where it is. It will be immaterial.

But also think the royalties you're talking about is not a weighted basis over the global situation. This is the countries where we pay royalties. There will be a lot of revenue generating outside. So if you really will try to calculate an effective royalty rate until May 2030, it will be likely down in the single-digit numbers and don't impact us in any way.

Okay. And then over the next 6 to 12 months, what can we expect from Ascendis in regards to clinical readouts, clinical updates, and just the story overall?

I think it's pretty well laid out what we expect. I think from when I talk with investors, there's a lot of focus. Can we really build up the $5 billion up to in 2030? And I think a lot of feedback I get, how will the next quarters? We came out with the perspective that we will have more than 1,000 patients every quarter going forward. And you will see that reflected in the revenue numbers. And I believe you will continue to see the launch. You will see your wealth still continue to grow dramatically in this way. And what you see, we will see the expansion in our label expansion. there will come results, there will come two years data from our combination trial, end of the year there will be other elements coming out from our portfolio. Next year we will have two or three phase three readout, and then you will see the new product opportunity coming in with the new chemical entities. So I think we are building up two things, continue the revenue generation at the same time building on the sustainability on building new chemical entities that really providing the next level of support to our sustainable revenue generation for the next 20 years.

Great. So we have two minutes left. Anything you'd like to leave investors with after the updates from last night and the discussion today? I think that speaks volumes. I think we're good. Ascendis team, thank you very much for joining us today. Really appreciate it.

Thanks so much.