Ascendis Pharma A/S Q1 2025 Earnings Call Transcript

The following is excerpted from the question-and-answer section of the transcript.

Question: Jessica Fye - JPMorgan - Analyst : Hey guys, good afternoon. Congrats on the strong results of YORVIPATH. It seems like the number reflects very good execution on reimbursement. Can you talk about your latest expectation for the proportion of patients with a script who you think will ultimately get reimbursed once YORVIPATH is, say, at steady state. And then the follow up would just be, was there any initial channel still reflected in the one revenue number? And if so, can you quantify that? Thank you. Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Thanks, Jess, for the questions. And Scott would take the easy one, the last one. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MAY 01, 2025 / 8:30PM, ASND.OQ - Q1 2025 Ascendis Pharma A/S Earnings Call

Question: Tazeen Ahmad - BofA Global Research - Analyst : Hi, good afternoon. Thanks for taking my question. Jan, can you give us a little bit of color on the splits between US and ex-US revenue for the quarter for YORVIPATH? And can you give us a sense, because it seems like you do have some patients receiving drugs in the US, what the length REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MAY 01, 2025 / 8:30PM, ASND.OQ - Q1 2025 Ascendis Pharma A/S Earnings Call of time is? I know it's early, but what is the length of time that you're seeing between when a script is written to when the patient is receiving therapy? Thanks. Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Yeah. Let me first address the question between the split between Europe and US. And as you have seen, we are not really describing that in our numbers, but I also believe that we give you good guidance how to potential to calculate it. As we said, when we look on the ex-US, we see a steady growth, a steady acceleration, but we first expect in the second half of this year to see an acceleration of the acceleration when we get more countries facing into the situation that will be fully reimbursed. So when I look on the difference between Q3 and Q4 last year, it's something around the element of an increase of around EUR4 million to EUR5 million in one quarter to the next quarter. So when you think about that, we increased our basic revenue from about EUR14 million to EUR45 million, then you can take basic the difference and then about EUR4 million or EUR5 million, and then I think you would get a number around EUR26 million which are some way reflecting the algorithmic I would use if I should look at it.

Question: Gavin Clark-Gartner - EVERCORE ISI - Analyst : Hey guys, congrats on the very strong launch progress. First, just to follow up on the payer point and the favorable access, have you finished negotiations with the majority of commercial payers at this point, and do you respect the remaining to be published policies to look similar to the ones that are already published and has the rebating fallen in line with your expectations with those conversations? Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Yeah, it's a question. I will take the global perspective. As you've heard about ex-US, we are basically fully reimbursed now in Germany. There is a final list price you can find. We also have that for Austria. In France, we have an AP2 program. We can also have a different program in Greece. So ex-US, we are working on really getting all country by country fully reimbursed. It takes time. It's something that some way both will happen here in '25, but also in '26 when we come into the end of '26, we believe we will have the vast majority of the foreign country being fully reimbursed. If you come to the US, which there is a strong focus on. Don't forget the more than 300,000 patients outside the US. I think Jay can give you more color about his discussion with the different, plans in the US.

Question: Gavin Clark-Gartner - EVERCORE ISI - Analyst : Okay, great. And then quick follow up for the new prescriptions that are coming in. What proportion are Natpara or PTH naive versus experienced? Thank you. Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board I don't think we really have the full insight exactly how many docs coming from the Natpara share, you can potentially correct me if we have got more insight to that. There was some question we discussed last week. We know that the vast majority of patients coming from conventional therapy, but perhaps, Sherrie, you can discuss exactly how many Natpara patients that we believe has been transferred over.

Question: Gavin Clark-Gartner - EVERCORE ISI - Analyst : Great, congrats on the progress.

Question: Derek Archila - Wells Fargo Securities, LLC - Analyst : Yeah, congrats on the quarter and thanks for taking the questions. So maybe just first on, can you comment on the depth of prescribing and the number of docs that are really prescribing, maybe two, three, four patients with YORVIPATH and then just to follow up to the last set of questions. Are you seeing patients that are newly diagnosed or well-controlled on conventional therapies move over to YORVIPATH? Thanks. Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Yeah. If I just could answer all your questions, I will be a happy person. I'm still a happy person. But, when I someway and, Sherrie, you can also someway add in. We cannot really know if there are well-controlled, partly controlled, or not controlled. That is not really anything we can see. We don't believe that it's really part of the reimbursement system. I think why we call them uncontrolled. We like to see more of them because we have an algorithm that basically are saying the 10,000 to 15,000 of the patients we did find as uncontrolled is because they're seen and into in a very high frequent. And that was why we know that we just went to this physician that will automatically have all these patients coming in. So when they're coming and control patients are received into on most less treatment, they're getting on Natpara. I hope so, but I cannot really know. But we know we really addressed the patients that see the [endo] and high frequency. I believe that is basically I will call the genius part of our commercial organization really working on not getting patients into the endos. It takes too long time as if I want to go into. Aimee's practice at Stanford. I need to wait three months to get an appointment with her, and I think we cannot wait for patients for that. And that was why we, I think they made a really genius move in the commercial organization, go to the places where we know patient comes in very, very far. So my expectation is that I believe we have a high percent of uncontrolled, partly controlled, but it's mainly only built on the algorithm because we are seeing the endo much more often than the other ones. So Sherrie, do you have anything to add?

Question: Joseph Schwartz - Leerink Partners - Analyst : Thank you and congrats on the very strong quarter. I was wondering if you have a sense of how many of your target endos in the US have adequate resources at their center in order to be able to go back and forth with payers who might not approve reimbursement of YORVIPATH right away just given there's so many patients in the US and a finite number of physician officers who treat them. Could their ability to navigate this process represent a cap on YORVIPATH revenue growth at some point? Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Joe it's something that Jay and the commercial organization had really a lot of thoughtful thinking about it. How can we ensure the journey for the patient, the physician, the office that's dealing with it is really going to be the most soft journey we ever could think about. And I think we have so much experience in this area, mainly out from our SKYTROFA, the 10,000 patients we took big portion of them to medical exceptions, and I think there was the learning we got. There was the system we're building on, and I think this is why we are potentially one of the best-equipped companies really to deal with medical exception from all the elements we learn from the SKYTROFA. Jay, you can (technical difficulty)

Question: Joseph Schwartz - Leerink Partners - Analyst : Thanks for the color. And to follow up, could I just ask what you hope to see in terms of clinical benefit from the combination of TransCon CNP and hGH and the COACH trial? Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Yeah, Joe. Now you're asking about the future again and this is good. I'm covered by Scott's statement even it went a little bit fast for me to understand what he said. But I actually saw an interesting research. And why it was interesting for me because they tried to take -- which it was actually something I never thought about myself, and this is potentially why I thought it was really into it. They looked on achondroplasia and hypochondroplasia. Achondroplasia, as you can see, the more severe form for achondroplasia, hypochondroplasia, a little bit more, you can say easy form for achondroplasia, less severe, less bright because what FDR 3 super activation is really putting a brake on the system. So when you took on achondroplasia, you have the brake full down hypochondroplasia partially down. And then they look what is the difference between growth hormone treatment. Over years with the two different (technical difficulty) achondroplasia and hydrochondroplasia. And I have to think that it was very well done and you can basically, I can give you the numbers. So in when you go to achondroplasia, you basically get a five, six centimeters, which are typical what we have seen. But when you go up to hypochondroplasia, you're up on the eight centimeters. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MAY 01, 2025 / 8:30PM, ASND.OQ - Q1 2025 Ascendis Pharma A/S Earnings Call And I actually think that it was a really smart resource because in some ways what we're doing with CNP, we take an achondroplasia patient and remove the break. So I don't know the results, Joe, but I thought that it gives me some strong belief that we're going to make a new standard for treatment. Not only for height, but also other places in the body where we really want to see a benefit in it.

Question: Paul Choi - Goldman Sachs - Analyst : Hi, thanks. Good afternoon, and congratulations on the commercial progress. On YORVIPATH, I wanted to ask if you have any color from the field as to what patient types it might be being utilized in. Is it almost primarily post-surgical patients or are you seeing other causes of disease like genetic and or other patients utilizing it too? And my second question on TransCon CNP is just, any updated thoughts or plans for development in the youngest patient population, those, shortly after birth through let's say three-years-old, just kind of what you're thinking is there in terms of potentially expanding into that population. Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Yeah, let's keep first up in the demographic on the US because if you look on the demographic in the US is that we have about 20% coming from we could call the genetic, [imological], everything else, and then about 80% coming from the post-surgical. And that was very much reflected in our clinical trials. So in our clinical trials we even have ADH-1 patients. We actually had two, even they are extremely hard to find because there are a few of them that have hypochondroplasia. We actually managed to get them in. So from that perspective is our clinical trial, this is basic reflected the numbers. I do know, Aimee, if you have any comments. You can come with all the different genetic variants we have there.

Question: Li Watsek - Cantor Fitzgerald - Analyst : Hey guys, wanted to add our congrats as well on a strong lunch. Maybe just first on YORVIPATH, in terms of contracting in the future. I know you're doing the process, but any guidance that you can provide on sort of the trend for growth to nets for the rest of the year, relative to Q1. And I have a follow up. Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Jay we will take that.

Question: Li Watsek - Cantor Fitzgerald - Analyst : Okay. And then just follow up on the Phase 2 COACH trial. I know you have a starting dose for SKYTROFA. So is there a sort of a titration scheme here that we should think about and what the top dose that you can go to? Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board No, it's basic is that the -- we have a starting dose in for the TransCon rota mode in the combination with CNP and they're also mission IPF one as they do in all trials and If there is any possibility that the physician desire to go up in dose or down the dose, they have the opportunity to do it, but Aimee, you know the protocol is better than I do.

Question: Li Watsek - Cantor Fitzgerald - Analyst : Okay, thank you.

Question: Ellie Merle - UBS - Analyst : Okay guys, thanks for taking the question. I'm curious just the feedback from the early patient starts, how the titration process has gone on YORVIPATH, just logistically, kind of any color anecdotes and how that's been going for physicians and patients. And any color, so far on what you're seeing from the refill rate, I know it's early on, but, curious any trends that you're seeing there. Thank you. Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board What I'm typically looking on is numbers. And one of the numbers I look a lot is two things is adherence. Second number I'm looking on is how many patients are dropping out. I think it's too good numbers to look if you have a successful, meaningful treatment of the patient with really addressing a major on medical need and really do that. And when I look on the adherents. It's exactly as high as we saw it in the clinical trials, which was really unique and drop out, we have given you the German numbers on the 1%. And we see the same thing everywhere. If you start on YORVIPATH, you stay on YORVIPATH. And that is a chronic treatment the rest of your life.

Question: Luca Issi - RBC - Analyst : Oh great, thanks so much for taking my question and congrats on the launch here. Maybe a quick one on competition. BridgeBio presented their data for their molecule earlier this week and I believe MBX will do the same next quarter. So wondering what's the latest thinking on both molecules and how competitive you think they can be versus YORVIPATH. And then REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MAY 01, 2025 / 8:30PM, ASND.OQ - Q1 2025 Ascendis Pharma A/S Earnings Call maybe Scott super quickly, how should we think about the SG&A for the remainder of the year, given the meaningful jump this quarter versus last quarter. Any thoughts there, much appreciated. Thanks so much. Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Yeah, you mentioned two compounds. One is the [catiolytic], which are being positioned into a Phase 3 trial of ADH-1. Currently ADH-1 is being treated with YORVIPATH, and they're coming on treatment today. It has been hard for us to find ADH-1 patient and, Sherrie, you went into the claim database and how many patient (multiple speakers) ADH-1.

Question: David Lebowitz - Citi - Analyst : Oh, thank you very much for taking my question. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MAY 01, 2025 / 8:30PM, ASND.OQ - Q1 2025 Ascendis Pharma A/S Earnings Call First of all, if you look at the Natpara patients that were left, from its withdrawal, how long do you think it takes until that whole population gets, worked through and then further looking back at Natpara and looking at what you've seen during your first quarter of launch. How would you, aside from the fact that it's the data is just superior, how would you characterize the difference in what patients are showing interest in this therapy? Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board First of all, you cannot compare the clinical benefit between Natpara and YORVIPATH. Natpara had a living as an adjunct therapy. Take a little bit of your daily calcium supplement away. Take a little bit of your act vitamin away, and then you take Natpara. You have no positive impact on kidney function. You have no positive quantitative manner on quality of life. I see this as two different products, and we can never compare these two together. First question related to when the Natpara patient will be swift. We addressed it a little bit in the beginning of here, the discussion here, where we know that there was a letter from [Tata] indicating that they're getting the last shipment now. And I think as to our notice, the shipping is three months, so we expect that the last series of Natpara patients will come over in either end of Q2 or Q3, that is our expectations.

Question: Leland Gershell - Oppenheimer & Co., Inc. - Analyst : Thanks. Great to see the strong execution on US YORVIPATH. Couple of questions from us, just, apologies if this had been asked before, but with respect to potential benefits on renal, I know, yeah, you had said that, you don't see that as a key kind of driver for YORVIPATH uptake, but nonetheless, are you able to comment in the early days of the US launch? Are you seeing a more difficult or easier time to gain reimbursement or access to the drug for those patients who may have less or more renal impairment, along with, whatever needs they have in terms of conventional therapy. And there's a second question. Thank you. Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board It's not a part of the reimbursement process. We don't see that it's the element that the site offers reimbursement and not reimbursement. So it's like the same thing as we discussed on party control, control is not part of the reimbursement process either. It's basic. When you look on the labeling, most planes have adapted. The weight that we basically have in our [la] and it's not defined way like that. That is the first one correction, Aimee, we didn't study in what we call severe renal impairment patients and that is not part of our label, and I think it's Stage 4.

Question: Leland Gershell - Oppenheimer & Co., Inc. - Analyst : Got it. Okay, that's helpful. And then, just looking forward to the COACH data, are you -- is there sort of a bar that you have in mind for linear growth, or is this something that you could see being driven forward principally on secondary benefits, say body composition or other? How are you thinking about kind of the mix of efficacy with the combination? Thank you. Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board When we think about achondroplasia, the key element for us is to address complications, but we cannot avoid also to address linear growth. So when we measure linear growth, which got established from another company that is established as the primary endpoint, I will personally have selected another endpoint if I could ever select that. But we will because we are forced to do it because it's the establish clinical endpoint is that we will look on linear growth and it will be part of the data we basically will report when we come up with the analyzed high velocity height as there is, and other things for that. And as I said before, I have great expectations. I have a strong belief that we can reset the bar for what you see in achondroplasia treatment, and it's not only related to linear growth but also the associated complications.