Ascendis Pharma A/S Q4 2024 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. Thanks for taking my question and congrats on the strong YORVIPATH script number and breadth of subscribers. I have one question with a few parts, mostly just confirming some stuff. First, can you confirm that the 908 figure is unique patients and not like cumulative scripts including refills, for example? Second, can you just confirm how many of the EAP and OLE rollover patients are now included in that 908 number? I want to make sure I heard you. I think you said it was 84% new to YORVIPATH, 16% from the EAP and OLE. Lastly, I think you reiterated that you continue to expect the majority of insurance approvals will take four to eight weeks. Can you just confirm that's actually what you're seeing now that you've been in the US market for coming up on eight weeks? Thank you. Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Thanks, Jess, a lot for the questions. I think we can confirm nearly all the answers. So Jay and Sherrie, will you confirm all the numbers, just to be sure?

Question: Jessica Fye - JPMorgan - Analyst : Thank you. For my follow-up, can you provide the YORVIPATH patient number outside the US? Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board We have not broken that number down. It's still increasing as we expected to do. We are still only in Europe Direct countries, full commercial in Germany and Austria. We have our AP2 program in France, a nonpromotional program that also is enrolling reimbursed patients and then we have REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. FEBRUARY 12, 2025 / 9:30PM, ASND.OQ - Q4 2024 Ascendis Pharma A/S Earnings Call our international market in this way. And the patient numbers are increasing to exactly as we have expected, but we have not broken down the numbers.

Question: Tazeen Ahmad - BofA Global Research - Analyst : Hi, guys. Good afternoon and thanks for taking my questions. Jan, could you provide any color on how many of the 908 scripts that have been written have now been converted to patients on actual therapy? And can you give us also any color that you might have on what proportion of these patients may have had previous experience with NATPARA? And then I have a follow up. Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Yeah. The question you are addressing is a question where we still lack all the information to give you and really the concrete answer that you want to have. What we have done in our market research, we have looked a lot on claim data. And we have defined patient group, which we call the 10,000 to 15,000 patients, which we will call not controlled on standard therapy or conventional therapy, and this patient group is basically seeing the physician at least 4 times a year. And they also will have one thing more in common. They have at least one hospitalization related to the disease. And we also know the limitation in really -- that is in seeing an endo. So out from that perspective, just by the random process when you can see an endo, we actually believe that many of the patients that we see of the 908 actually are coming from the group which we define as more uncontrolled in this. We don't have a positive definition if that is true or not, but that is all expectation in this. The second question you raised about how many patients we have on therapy now is a number we are waiting, some way, to come out with until we feeling we have sufficient, good enough data to give you a solid number because we are now in the initial part of our, you can say, journey of getting them 100% reimbursed. We were extremely positive on the broadness of having reimbursed patients, both from the commercial setup but also for the government side. And we basically are getting reimbursed patient every place from. And when you see the broadness on the state, we're covering with about 35 different states, we're really coming out in all different states. We're also feeling that we are in a very, very strong position. The vast majority of the patients are on label. And basically, they are also fulfilling exactly the criteria that justifies for them to be on a PTH treatment. So we are really highly positive on the development we have seen in the reimbursement spectrum.

Question: Gavin Clark-Gartner - EVERCORE ISI - Analyst : Congrats on the very strong progress so far. Just had one question on YORVIPATH and one on CNP. For YORVIPATH, how have your discussions with payers gone so far? And what are your assumptions for how some of the prior authorization criteria may read? Specifically wondering if you think it's going to be different than the label. And kind of on that same line, maybe you could just remind us your contracting or gross to net assumptions. That's the first one. On the CNP side, for the CNP plus growth hormone combo data coming in the second quarter, can you just remind us your expectations, specifically on AGV? Thank you. Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Yeah. I think Jay will come with some flavor about the contracting situation. But also, as I said before, I've been lucky to see most of the UN criteria. And I have been really pleased how they are aligning to the labeling. But I believe Jay will come up with comments related to the commercial contracting landscape.

Question: Gavin Clark-Gartner - EVERCORE ISI - Analyst : That's helpful. Thanks, guys. We'll leave it there for now.

Question: Li Watsek - Cantor Fitzgerald - Analyst : Wanted to add my congrats on the strong YORVIPATH launch as well. Just curious, for these 900-plus prescriptions, what proportion of these patients have more severe versus more moderate diseases? And if you can share any early trends of compliance and initial titration, whether it's consistent with your clinical trials. Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Yes. It's a question we tried to address with before. But I think, Sherrie, you're also sitting with a lot of data. Potentially, you can, in some way, describe the limitation that we have in our dataset. We need to answer that question today.

Question: Li Watsek - Cantor Fitzgerald - Analyst : Thank you.

Question: Eva Fortea-Verdejo - Wells Fargo Securities, LLC - Analyst : This is Eva on for Derek. Thanks for taking our questions and congrats on the progress. A quick one from us on YORVIPATH. So can you provide some color on what type of docs are prescribing YORVIPATH? Are these like high-volume docs? Or are you starting to get some docs from the community? And as a follow-up, how long do you expect patients will take to convert from drug in the EAP over to paid drug? Thanks. Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Yeah. Let me take the last question before I turn it over to Jay or Sherrie. What is happening in our EAP program, the last packet they got was a three-pack of basic three months, and some of them got them in December to -- and January. So the 100-plus patients that we are converting from our EAP clinical trial over will likely come into commercial drug starting in February and then the majority in March. So that was the second question. And I think -- Sherrie or Jay, who will take the -- will you start, Jay?

Question: Yaron Werber - TD Cowen - Analyst : Great. So thanks so much. I got maybe a couple of questions, Scott, for you. And then I don't know, Jan, if you want to take the next one. I think, Scott, you said that towards the end of the year, you could be -- you could reach profitability on a cash basis. Does that still sort of hold? And what would drive that? How do we -- is it going to be YORVIPATH is the biggest driver? And then secondly, as you think about reimbursement for YORVIPATH, are you expecting, with time, prior authorizations? Or are you expecting not to have any prior auths? Thank you. Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Okay. I think Scott would like to say something.

Question: Joori Park - Leerink - Analyst : Hi. I'm Joori Park dialing in for Joe. Thank you for taking our questions. I believe in the last earnings conference call, you mentioned that in Germany, physicians are prescribing YORVIPATH to about one to two of their patients, whereas in the US, you are expecting physicians to prescribe the drug to two to three of their patients. And based on the metrics that you provided today, it seems like on average, each physician is prescribing the drug to a little over 1.5 of their patients. So I was just wondering if you're still expecting each physician in the US to prescribe YORVIPATH to about two to three of their patients. And where does it go from there? Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Yeah. I think in some way, I accept what I said, and I stand by what I said before. And I think the pattern we see now is an early launch. I have seen and follow some of the physicians that was part of our EAP and clinical trial. And one -- some of these physicians have been already making prescription up to 20 patients. So no doubt the broadness of that will come. But in some way, this is in early launch, one to two months in the launch. So I will expect that to see and come in the future.

Question: Kelly Shi - Jefferies - Analyst : Congrats on the great quarter. On manufacturing front, how should we think about the capacity to meet the increasing demand of YORVIPATH throughout the year? Thank you. Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Yeah. I think one of the things we have been very, very proud at Ascendis is really our robust supply chain. We have seen how we have managed to go up in high demand on the shortage of the daily growth hormone and could fulfill all requirement for all [issues] for having, for example, the SKYTROFA brand. And when I look on the YORVIPATH, we're using the same solid supply chains, the same infrastructure. We followed the same way. I personally get a weekly report on every compound in every region, in every place where we are selling product, how many months there is on storage. And we, some way, are taking that as a really serious thing because we will never be in a position that we will go short. And that is our vision, and I hope we never come to this positioning.

Question: Ellie Merle - UBS - Analyst : Hey, guys. Thanks so much for taking the question and congrats on the launch progress. Just in terms of how we should think about the US YORVIPATH script cadence, I guess, sort of what's the latest that you're seeing in terms of the cadence of new starts? Are you sort of seeing a steady number of ask each week? Was there a bolus upfront? How should we think about this going forward? And second, just a follow-up on reimbursement. I know you said you're seeing approvals across a number of plans. But could you characterize maybe sort of the number of scripts that have been covered so far and sort of any trends that you're seeing in terms of ease of reimbursement, say, between the moderate patients versus the severe patients and your expectations there? Thanks. Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board I think the question related to what we call controlled to partly controlled and -- patient, we have, in some way, answered this to our best of knowledge. We have no clear data of the patients that is -- already have received a prescription. We have a gut feeling, and the gut feeling is that we believe many of them belong to the uncontrolled part because they see the endo in a much higher frequence than anyone else. Related to the pattern on prescription, we are not breaking it down in weeks or anything. This is something we are following [where we are tight]. And we would like to see a pattern being developed. We don't believe there's really a huge bolus coming in because there is not a huge availability of the (inaudible) really to come in and say, oh, I just want to have an appointment, run into an endo and now we have a prescription. It's not like (inaudible). Like Aimee is sitting here. If you want to see Aimee, our CMO, at Stanford, it takes how many weeks now, Aimee? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. FEBRUARY 12, 2025 / 9:30PM, ASND.OQ - Q4 2024 Ascendis Pharma A/S Earnings Call

Question: Ellie Merle - UBS - Analyst : Great. Thanks.

Question: David Lebowitz - Citi - Analyst : Thank you for taking my question. First, on SKYTROFA, you had the PDUFA date coming up this summer for the adult growth hormone deficiency. I'm just curious, how should we be thinking about that in terms of what that market opportunity actually presents? Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Yeah. I think Aimee can explain on the unmet medical need that really exists in adult growth hormone deficiency. From my perspective, there's two key things I really -- some way, are reflecting over a lot. First of all, it's an area with extremely low penetration. To our best knowledge, it's under 5%, 6%, 7%. So you can say there is a huge opportunity for growth. And the other thing is that has been a huge burden basically, to be in the treatment with daily growth hormone. But Aimee, you can explain and tell about what is really the unmet medical need, the burden of having adult growth hormone deficiency. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. FEBRUARY 12, 2025 / 9:30PM, ASND.OQ - Q4 2024 Ascendis Pharma A/S Earnings Call

Question: David Lebowitz - Citi - Analyst : Thank you for that. And just jumping over to YORVIPATH. I'm just curious as to thoughts on potential competing pivotal data coming out this year and how you think the market ultimately might evolve. Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Yeah. If you reflect on pivotal data, I don't think there is anyone else than one compound, the amyloid compound. And to our best knowledge, when we talked with centers, their last patient in was in the beginning of November. So I'm still waiting to see the results of the pivotal trial. It's a small trial with a little bit more than 100 patients. So it shouldn't take too long time to clean the data and come up with the top line data. But I have not seen anything else. When I go back to the science at one of our key values, this is not a hormone replacement therapy. This is a substitution of the long-acting effect where you basically are placing part of the receptor system into a fixed position and therefore, have a long-acting effect. It's not even reflecting the normal biology where you are basically activating both the PTH 1 receptor and the PTH 2 receptor. And by doing the different mode of action, you already are -- from the data, can see that it can now substitute, as in hormone replacement therapy, element on where you see it's not restoring normal function, for example, in the kidney, both with -- for example, related to phosphate excretion. See, for example, it cannot lower a key element like calcium phosphate complex. You also see the unnatural activation to the activating system in the bone and other organs. And then I'm not talking about the hemogenic potential that is in the compound. So we are looking forward to see the data. We would like to see the data, and we hope one day that will come out. They should be out there now because it's so long time since the last patient came into the visit.

Question: Paul Choi - Goldman Sachs - Analyst : Hi. Thanks. Good afternoon and congrats on the early launch success with YORVIPATH in the US. My first question is on YORVIPATH. And as we look at consensus numbers, The street is modeling less than 900 patients in the US for this year. And you've already exceeded that in terms of REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. FEBRUARY 12, 2025 / 9:30PM, ASND.OQ - Q4 2024 Ascendis Pharma A/S Earnings Call scripts. So I was just wondering, recognizing that you're not giving guidance and not -- and the reimbursement is a work in progress, just your level of comfort with The street, a revenue number for the full year possibly, or any color around that would be great. And my second question is regarding CNP and the hypochondroplasia program that you plan to file an IND for later this year. Are you planning to do any run-in activities ahead of that possibly at some of the centers just to potentially help accelerate the timing of that study and enrollment? Any color there would be helpful. Thank you very much. Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Yeah. Let me take the first part together with Aimee because we are really dedicated to be the leader in growth disorder. And when we see the two cornerstones we have, TransCon Growth Hormone and TransCon CNP, we really will do the best for the patient in each case. How can we design an optimal treatment in this way? And what we're doing now in hypochondroplasia is design what is really the pathway forward for us. Is it then just a TransCon CNP treatment? Or will it be a combination treatment between TransCon CNP and TransCon Growth Hormone? And we are now in a position that we are starting the discussion with regulatory agencies. But I think Aimee can give you a little bit of background why we, some way, think that when we're moving into hypochondroplasia, it's one of many growth disorders we actually would like to focus on. And the actual design can also be a different state process.

Question: Alex Thompson - Stifel - Analyst : Hey. Great. Thanks for taking my questions. I guess on YORVIPATH, I wonder if you could talk a little bit about how titration is working in the real world with patients, whether that requires additional visits with their HCP, et cetera, if this is done at home. And then maybe could you talk a little bit about your expectations around gross to net stability in 2025 for SKYTROFA? Thanks. Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Aimee, would you take the first?

Question: Yun Zhong - Wedbush Securities - Analyst : Excuse me. Good afternoon. Thank you very much for taking the questions. The first one on YORVIPATH. So when you report first quarter earnings, are you going to break down sales to tell us how much is coming from Europe, how much is from US, please? Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board That's a good question. We will decide when we come to that state exactly how we report the data. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. FEBRUARY 12, 2025 / 9:30PM, ASND.OQ - Q4 2024 Ascendis Pharma A/S Earnings Call