Ascendis Pharma A/S Q1 2025 Earnings Call Transcript

Quartr
Provided by Quartr
May 1, 2025

There are 14 speakers on the call.

Operator

Ladies and gentlemen, thank you for standing by, and welcome to Ascendis Pharma First Quarter twenty twenty five Earnings Conference Call. At this time, all participants are in a listen only mode. After the speakers' presentation, there will be a question and answer session. To ask a question during the session, you will need to press 11 on your telephone. You will then hear an automated message advising your hand is raised.

Operator

To withdraw your question, please press 11 again. Please be advised that today's conference is being recorded. I would like now to turn the conference over to Scott Smith, Vice President and Chief Financial Officer. Please go ahead, sir.

Speaker 1

Thank you so much, operator, And thank you, everyone, for joining our first quarter twenty twenty five financial results conference call. I'm Scott Smith, Chief Financial Officer at Ascendis Pharma. Joining me on the call today are Jen Mickelson, President and Chief Executive Officer Sherry Glass, Chief Business Officer Jay Wu, Executive Vice President and President, Ascendis U. S. And Amy Hsu, Chief Medical Officer.

Speaker 1

Before we begin, I'd like to remind you that this conference call will contain forward looking statements that are intended to be covered under the Safe Harbor provided by the Private Securities Litigation Reform Act. Examples of such statements may include, but are not limited to, statements regarding our commercialization and continued development of Skytrof and Yorvapat for The U. S, European and other markets as well as certain financial expectations, our pipeline candidates and our expectations with respect to their continued progress and potential commercialization. Our strategic plans, partnerships and investments, our goals regarding our clinical pipeline including the timing of clinical results and trials, our ongoing and planned regulatory filings and our expectations regarding the timing and results of regulatory decisions, expected market developments exploration of market opportunities in therapeutic areas outside of endocrinology rare disease. These statements are based on information that is available to us as of today.

Speaker 1

Actual results may differ could 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 02/12/2025. TransCon Growth Hormone or TransCon hGH is approved in The U. S.

Speaker 1

By the FDA and then the EU has received MAA authorization from the European Commission for the treatment of pediatric growth hormone deficiency. TransCon PTH is approved in The U. S. By the FDA for the treatment of hypoparathyroidism in adults and the European Commission and The United Kingdom's Medicine 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.

Speaker 1

As of investigational products, the safety and effectiveness of product candidates has not been reviewed or approved by any regulatory agency. None of the statements during this conference call regarding our product candidates shall be viewed as promotional. On the call today, we'll discuss our first quarter twenty twenty five financial results and we'll provide further business updates. Following some prepared remarks, we'll then open up

Speaker 2

the call for

Speaker 1

questions. With that, let me turn it over to Jens.

Speaker 3

Thanks, Scott. Good afternoon, everyone. In the first quarter of twenty twenty five, Ascenis continued the strong start of our global U. Launch, as well as key development and regulatory progress supporting our long term growth strategy to be a leading biopharma company. The strong US launch of our Europads position 2025 to be an inflection point for Ascendis, with a growing revenue base and a clear path to become cash flow positive.

Speaker 3

As of March 31, Europurnt was prescribed in The US by more than 1,000 unique prescribers for more than seventeen fifty patients. This represents our first full quarter for The US launch. Europath, the first and only FDA approved treatment for hypoparathyroidism in adults, is addressing the underlying cause of the disease by providing active PTH within the physiological range for twenty four hours per day. Skytropha, our long acting growth hormone, is firmly established as a high value brand and the preferred treatment for patients, physicians and caregivers. SKYYTOVA is well positioned as daily treatment continues to exit The US market and a SKYYTOVA label has the potential to expand beyond its single indication.

Speaker 3

Skytropha is a key pillar in our strategy to become a global leader in the treatment of growth disorder. TransCon CNP, the first long acting therapy in development for the treatment of achondroplasia, is set to become the second pillar in our growth disorder strategy. We believe that TransCon CNP has treatment benefits in addition to linear growth that addresses multiple aspects of the condition that are fundamentally important to patients. We submitted an NDA to FDA in March and expect to file an MAA with EMEA in Q3 this year. Data from three randomized, double blind, placebo controlled clinical trials show that TransCon CNP has the potential to transform the life of people with achondroplasia.

Speaker 3

In my remarks, I will discuss each of these products in detail and comment on other recent developments within our business. Beginning with Europads. First quarter total global Europads revenue grew to €45,000,000 compared to 14,000,000 in the fourth quarter of last year. Following commercial availability in The U. S.

Speaker 3

In December of last year, we are seeing strong U. S. Demand, reflecting both the deep unmet medical need in the market as well as the large patient population. As of March 31, more than seventeen fifty patients, including the 200 patients from our ERP and clinical program have been prescribed Europet in The U. S.

Speaker 3

By over 1,000 unique healthcare providers. Enrollment of patients new to UOPED continue at a similar weekly rate in April. The majority of patients who have received insurance approval for their UOPED prescription received that approval in four to eight weeks. And we are pleased with the approval rate we have seen. We are beginning to see favorable care plans put into place and continues to see approvals across both commercial and government plans.

Speaker 3

The strong launch performance of UECAD in The US support our view of its excellent product profile and the major unmet need in the market. And we expect Europads to contribute significantly to our revenue in 2025. Outside of The U. S, we see steady Europads revenue growth in both the Europe direct and international markets. And we expect additional acceleration of the revenue growth when Europads reimbursement becomes available in additional Europe direct countries in the second half of the year.

Speaker 3

The continued rapid uptake together with high rates of patient adherence give us confidence that Europan is well positioned to uniquely address the unmet medical need of this patient population. And we are regularly reviewing input and data from patients to evaluate if there are additional ways to improve the treatment profile even more. We estimate there are over 400,000 patients globally and around seventy to ninety thousand patients in The US alone living the chronic hypoparathyroidism. Our claims analysis demonstrate that ten to fifteen thousand of these US patients are uncontrolled and thirty to thirty five thousand are partly controlled. Based on the latest clinical practice guidelines, nearly all these patients are candidates for treatment with Europads.

Speaker 3

Our strong global lungs give us high conviction that we can continue to build and lead this market And Europad can be a durable multi billion euro drug device product with a patent lifespan extending into the 2040s. Turning to SKYTOVA. Q1 revenues for SKYTOVA were €51,000,000 with continued patient growth and global expansion offset by the typical first quarter revenue dynamic in The US. We have around 7% market share of the total growth hormone market in The US and around 43% of the total US non acting growth hormone market based on third party prescription data. The pediatric growth deficient indication represents about half of the total growth hormone market.

Speaker 3

With our premium pricing and Skytofra's leading position in pediatric growth hormone deficiency, We believe we are well positioned to expand the opportunity for Skytropha in multiple ways. A key near term milestone is our first potential label expansion in the established growth hormone indications from our supplement BLA for the potential U. S. Approval in adult growth hormone deficiency, where we have a PDUFA goal date of July '5. We're also on track to start a basket trial for Scott Tofer in a range of indications, including idiopathic short stature, shock deficiency, Turner syndrome, and SGA.

Speaker 3

We are planning to discuss this trial with the FDA in an end of phase two meeting this quarter. Importantly, we are also investigating TransCon growth hormone outside the established growth hormone indication, such as in a potential combination therapy with TransCon CNP for treatment of achondroplasia and other growth disorder, which I will address in a moment. Moving to TransCon CNP. TransCon CNP is the third key product in our endocrinology rare disease product portfolio. The genetic variant that caused achondroplasia changed the way receptor work in multiple tissues throughout the body, not just in the growth plate and in bones, resulting in a wide range of serious medical complications in childhood and lasting throughout adulthood.

Speaker 3

Because TransCon CNP provides sustained therapeutic levels of CNP throughout the body, it has been demonstrated and unique product profile, giving it the potential to bring growth benefit and important new benefit beyond linear growth, as well as reduced risk of hypertension and injection site reaction. In our pivotal approach trial, TransCon CNP demonstrated significant improvement in the primary endpoint of linear growth compared to placebo, as well as significant improvement in other clinical endpoints meaningful to the achondroplasia community, including leg bowing, muscle functionality, body proportionality, and health related quality of life. Leg bowing is a common complication in achondroplasia that can result in chronic pain and impaired physical function, driving many to undergo complex painful corrective surgeries. I have been in meetings with patient organizations in Europe and The U. S.

Speaker 3

Who have confirmed the importance of addressing the complication of achondroplasia beyond linear blood. Just as important to the achondroplasia community, TransCon CNP has shown a safety and durability profile compared to placebo with low frequency of injection site reactions, all which were mild and no evidence of symptomatic hypertension. After positive interaction with the FDA relating to the content of our NDA submission, we are pleased to have submitted TransCon CNP for the review in March. In The EU, we plan to submit an MAA during the third quarter of this year. Additionally, during the fourth quarter of twenty twenty five, we plan to submit an IND or similar to investigate TransCon CNP or in combination with TransCon Growth Hormone for the treatment of hypokinotaceous.

Speaker 3

Shifting to TransCon CNP and TransCon Growth Hormone Combination Therapy, we are committed to continue to drive even better outcomes for people living with achondroplasia. This is why we are conducting the COAT trial, being the first phase two study combining CMP and growth hormone in achondroplasia. Each of which stimulates different signaling pathways in the growth plates and other tissues in the body. We look forward to sharing top line week 26 results from the close trial data this quarter and see great potential to further raise the bar for clinical outcomes. With TransCon CNP as the potential future backbone therapy, We believe we can achieve even greater growth while also addressing medical complication of achondroplasia.

Speaker 3

Fundamental to the development of each of our three medicines, EuropaT Scatopa TransCon CNP is Ascenty's proprietary TransCon technology platform. With the TransCon technology and our deep understanding of disease biology, it is possible to create medicine with highly differentiated treatment benefit not possible with other technologies. At Ascendis, our commitment has always been to the patient. It is one of the company's core values. I believe we have demonstrated multiple times over the history of Ascenty's our resilience and our ability to adapt and find solutions to attain this goal.

Speaker 3

We remain as dedicated as ever to ensure that all our medicines become available to patients. Through our collaboration with Novo Nordisk for the development and commercialization of TransCon technology based products in metabolic and cardiovascular disease and our collaboration partner, Wissen, Iconis, and Tagli, we continue to work to execute our Vision 02/1930 to create value in large therapeutic areas and through innovative business models. In summary, 2025 is a transformative year for Ascendis as we grow our global revenues from Europet and Skytrofa and seek to obtain key regulatory approvals, deliver robust clinical data and then advance drugs with blockbuster potential to drive growth for many years to come. I will now turn to Scott.

Speaker 1

Thank you so much, Jan. I will touch on some key points surrounding our first quarter financial results, but for further details, please refer to our six ks filed today. Europath's first quarter revenue increased significantly to €44,700,000 up from €13,600,000 in the fourth quarter of twenty twenty four. Steady sequential revenue growth outside The U. S.

Speaker 1

Was augmented by the strong U. S. Launch. As Jens discussed, the trends we saw for Yorvapath in Q1 continue in Q2, both outside and inside The U. S.

Speaker 1

And we anticipate that Yorvapath will have a substantial impact on our financial profile in 2025. Turning to Skytrofa. Revenue for this quarter was €51,300,000 Sequentially, pricing and market share remained stable, but revenue in The U. S. Was negatively impacted by seasonal items, including reduction in channel inventory and higher co pay assistance.

Speaker 1

Those seasonal headwinds should reverse beginning in Q2. We also continue to watch the euro dollar exchange rate for any potential impact related to reported revenue. Total revenue for the first quarter was €101,000,000 which includes non product revenue from our collaboration partners. Turning to expenses. For the first quarter, R and D costs totaled €86,600,000 compared to €70,700,000 during the first quarter of twenty twenty four.

Speaker 1

The first quarter of twenty twenty four included a favorable €10,600,000 reversal of prior period write downs of TransCon PTH pre launch inventories. SG and A expenses in the first quarter of twenty twenty five totaled 101,000,000 Euro compared to 66,800,000.0 Euro during the first quarter of twenty twenty four. The €34,000,000 increase was primarily driven by global commercial expansion. Total operating expenses were €188,000,000 for the first quarter of twenty twenty five. As a result of the Visa IPO, we recognized a non cash gain of €33,600,000 as part of share of profit loss of associates, and we retained 39% ownership of Visa.

Speaker 1

Net finance expenses for the first quarter of twenty twenty five were 15,900,000.0 Euro, driven primarily by non cash items. Net cash financial income for the first quarter of twenty twenty five was 3,300,000.0 Euro. We ended the first quarter of twenty twenty five with cash and cash equivalents totaling €518,000,000 compared to €560,000,000 as of 12/31/2024. Turning to the remainder of 2025, we expect substantial revenue growth driven by the global launch of Yorvapath with a continued solid contribution from Skytropha. We are not providing revenue guidance for Skytropha or Yorvapath at this time.

Speaker 1

For SKYYROPA, we believe that growth in prescriptions, visible in third party data, will track sequential revenue growth for 2025 with expected stability and mix in pricing, including normal seasonality. For Eurvipath, outside The U. S, we continued steady revenue growth, while inside The U. S, our launch is progressing exceptionally well. We will continue to look to help investors understand uptake and reimbursement dynamics as the year progresses.

Speaker 1

With that, operator, we are now ready to take questions.

Operator

Thank you. And to withdraw your question, please press one one again. We do ask that you please limit to one question and one follow-up to allow time for everyone to ask a question. And our first question today will come from Jessica Fye with JPMorgan. Your line is open.

Speaker 4

Hey guys, good afternoon. Congrats on the strong results of the Orbi Path. 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 YorVuPATH is, say, at steady state? And then the follow-up would just be, was there any initial channel fill reflected in the 1Q revenue number?

Speaker 4

And if so, can you quantify that? Thank you.

Speaker 3

Thanks, Jess, for the questions. And Scott will take the easy one, the last one.

Speaker 1

Yeah, Jess. So channel inventory has averaged about one to two weeks at any one time. We ship once a week, so it's hard to get less than one week of channel.

Speaker 3

The second one and just why it's a little bit more complicated because in some way looking in the future, what we can see, we see a very, very, very positive trend. We see the adaption of the different PPMs, the different payer forms and if I in some way should come with my own guess about it and this is more a guess because we don't know exactly there will never be 100% approval of all the patients. And I believe if we really are successful and I hope we won and I believe we're going to be successful, I believe 17% to 18% will get reimbursed. But I think, Jay, if you have any other comments to it this way.

Speaker 5

Yeah, to chime in on that. I think as Jan mentioned, we are still early in the launch. So from that perspective, we're still seeing policies be set into place. So it is a bit early to be able to anticipate what that steady state will be. I do think a few examples that give us a lot of confidence that we are headed in the right direction is a couple.

Speaker 5

One, you're seeing policies favorable policies be put into place both from a commercial standpoint as well as government. So we're seeing that start to take hold. And then two, even absent of a formal policy in place, we do see, patients get approved across the board, again, agnostic of whether it is a commercial plan or government plan. It just sometimes may take a few more steps, whether it's through medical exception, and in some rare cases even through appeal and or peer to peer, but largely we are seeing many patients get through. You will expect a long tail just given that every payer plan is different given the heterogeneity of it in The US, But overall, we have a lot to be encouraged about.

Speaker 5

And I think more importantly, it just really emphasizes the clinical value proposition of this product and the fact that payers, providers, and patients are all responding to it.

Speaker 3

I think, but yes, I think some way when I talk about my feeling, my feeling is that I believe every patient that some way have a desire to go on treatment should come on treatment because I have seen that I've heard MET patients that benefit that is on the treatment in it. But I also accept reality in this way here. But I also believe we are in a unique positioning. We're addressing a major unmet medical need. We have really a product that really are providing the benefit to the patient.

Speaker 3

We are in a position that patients are diagnosed with the disease. At the same time there is clear guidelines that are saying there should be a treatment. Can you find a better case for any patient really not to get the ultimate goal if you get and want to be off treatment, you should be on treatment.

Operator

And our next question comes from Tazeen Ahmad with Bank of America Securities. Your line is open.

Speaker 4

Hi, good afternoon. Thanks for taking my question. Yen, can you give us a little bit of color on the split between U. S. And ex U.

Speaker 4

S. Revenue for the quarter for Yurvy Path? And can you give us a sense, because it seems like you do have some patients receiving drug in The U. S, what the length 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?

Speaker 4

Thanks.

Speaker 3

Yeah, let me first address the question between Europe and U. S. 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 potentially to calculate it. As we said, when we look on the ex US, we see a steady growth, a steady acceleration.

Speaker 3

But we first expect in the second half of this year to see an acceleration of the acceleration when we get more countries basic 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 around 4,000,000 to €5,000,000 in for one quarter to the next quarter. So when you think about that we increased our basic revenue from about 14 to 45, then you can take basic, the difference, and then subtract about 4 or 5,000,000. And then I think you would get that number around 26,000,000 which are some way reflecting the algorithm I would use if I should look at it.

Operator

And our next question will come from Gavin Clark Gartner with Evercore. Your line is open.

Speaker 6

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?

Speaker 3

Yeah. It's a question. I will take the global perspective. As you've heard about ex US, we are basic fully reimbursed now in Germany. There is a final list price you can find.

Speaker 3

We also have that for Austria. In France, we have an AP two 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.

Speaker 3

It's something that some way, both will happen here in '25 but also in '26 when we come into end of twenty six, we believe we will have the vast majority of the important 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.

Speaker 5

Thanks Jan and thanks Gavin for the question. Yes, to answer your question, the conversations with payers have been going very well. We have multiple commercial policies in place with our national accounts. So while we don't comment on policy by policy that gives you a sense that we are gaining a lot of good traction there and the policies that are put in place are favorable and consistent with label. While we don't comment on the gross to net arrangements that we have with each what I will say is that it is commensurate with what we said before.

Speaker 5

This is a first and only approved product in a rare disease setting with clinical value benefit that we strongly believe in and we feel we've been able to convey with payers and from that lens, it is commensurate with the incredible clinical value proposition that we bring and we certainly lead with that and expect it to reflect that.

Speaker 6

Okay, great. And then quick follow-up for the new prescriptions that are coming in, what proportion are on NATPARA or PTH naive versus experienced? Thank you.

Speaker 3

I don't think we really have the full insight exactly how many deaths coming from the NATPARA. Sherry, you can potentially correct me if we have got more insight of that. There was some question we discussed last week. We know that the vast majority of patients coming from conventional therapy, but perhaps Sherry, you can discuss exactly how many NaPiR patients that we believe has been transferred over.

Speaker 7

Yes, thanks for the question, Gavin. And yes, Jens is right. The data is not entirely definitive on this point, but we know that we have the vast majority of our patients overall are coming from conventional therapy. So that's very clear. And we do have something like ten percent to fifteen percent of patients that have been on some sort of PTH in the past.

Speaker 7

And then, you know, a subset of those were more recently on NATPARA. But the bottom line is, you know, overwhelmingly coming from conventional therapy.

Speaker 3

Yeah, from that perspective, we believe that in Q2 and Q3, we will see the majority of the rest of the NATPARA patient coming over because Amy, you're sitting with the patients, you can explain that there is a letter coming out explaining that you are in a position that they're stopping.

Speaker 8

Right. There's still as far as we know, there's still some supply, so they still may be on it. But we know it will exhaust.

Speaker 3

Yeah. So the basic, when we look at the patient that's coming over, the seventeen fifty, this is not a power patient. This is basic patient that's coming from conventional therapy, the thirty five thousand patients we are addressing now. So there is a lot of deepness to go in this way.

Speaker 6

Great. Congrats on the progress.

Operator

And our next question comes from Derek Archila with Wells Fargo. Your line is open.

Speaker 9

Yes, 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 your VPATH? And then just a follow-up to the last set of questions, just, you know, are you seeing patients that are newly diagnosed or well controlled on conventional therapies move over to your VPATH? Thanks.

Speaker 3

Yeah. If I just could answer all your questions, will be a happy person. I'm still a happy person. But when I share, you can also add in. We cannot really know if they are well controlled, partly controlled or non controlled.

Speaker 3

That is not really anything we can see. We don't believe that it's really part of the reimbursement system. I actually think why we call them uncontrolled. We likely will see more of them because we had an algorithm that basically are saying that ten thousand to fifteen thousand of the patients we did find as uncontrolled is because we are seeing endo in a very, very, very high treatments. And that was why we know that we just went to this physician that will automatically have all this patient coming in.

Speaker 3

So when they're coming and control patients that see the endo on most, most less frequent they 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 frequent. And I believe that is basically I would call the genius part of our commercial organization really working on not getting patients into the endo. It takes too long time as if I want to go into Amy's practice in Stanford, I need to wait three months to get an appointment with her.

Speaker 3

And I think we cannot wait for patient for that. And it was why I think they made a really, really genius move in the commercial organization, go to the places where we know patient comes in very, 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 they are seeing the endo much more often than the other ones. So, Sherry, do you have anything to add?

Speaker 7

I think you covered it well again. I would maybe just say one of the things we're really excited about is we know there are quite a good healthy number of those uncontrolled and partially controlled patients. So something like ten thousand to fifteen thousand uncontrolled and another thirty thousand or so partially controlled. And as Jens said, the uncontrolled patients are seeing their ENDO's four times or more a year. So we know, to Jens point, that they're getting in and therefore that we expect to see a steady flow of patients coming in and having the option to get on drug over the course of the year.

Speaker 3

Yes. And one of the reasons why we don't know it is because it's not part of being reimbursed or not. So if it was a part of the reimbursement system to have this uncontrolled, partly controlled or controlled, we will know much more on it, but it's not a decision in the reimbursement system.

Operator

And the next question comes from Yaron Werber with TD Cowen. Your line is open. Your line is now open. Our next question will be from Joe Schwartz with Leerink Partners. Your line is open.

Speaker 10

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 U. S. Have adequate resources at their center in order to be able to go back and forth with payers who might not approve reimbursement of Yorvi right away, just given there's so many patients in The U. S.

Speaker 10

And a finite number of physician offices who treat them. Could their ability to navigate this process represent a cap on Yorvipath revenue growth at some point?

Speaker 3

Joe, it's something that Jay and the commercial organization have 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 can think about. And I think we have so much experience in this area mainly out from our SkyTrophy, the 10,000 patients we took, big portion of that 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 Skytrofra.

Speaker 3

Jay, you can go much more

Speaker 5

just to add on and to reinforce what Jan said, our hub is a well oiled machine at this point, right? Incredibly experienced as we all know, the growth hormone deficiency space is a heavily managed space. So not only is our hub infrastructure equipped both from a volume and speed perspective, we also have a strong field reimbursement manager footprint that is constantly supporting these offices along the way as well. We feel good about our ability to resource, and support our customer needs as needed. To your question directly, of course, there's going to be some offices, particularly the ones where perhaps it's lower volume, they may see fewer cases.

Speaker 5

That's not, I would say, unique to many other specialties that experience the same type of office burden. But again, going back to what Yan was sharing earlier, we're well equipped and have the capabilities to ensure that we are allowing that to not be the bottleneck as to why patients will get on therapy.

Speaker 10

Thanks for the color. 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 in the COACH trial?

Speaker 3

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 interesting.

Speaker 3

Then I 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 break because what FTR3 super activation is, is really putting a break on the system. So when you took on achondroplasia you have the break pulled down, hypochondroplasia partly down. And then they look what is the difference between growth hormone treatment over years with the two different therapeutic areas achondroplasia and hydrocarboplasia. And I have to think that it was very well done and you can basic I can give you the numbers.

Speaker 3

So when you go to achondroplasia, you get a five, six centimeters, which are typical what we have seen. But when you go up to hypochondroplasia, you are up on the eight centimeters. And I actually think that it was really smart results because in some way what we're doing with CMP, we take an achondroplasia patient and remove the break. So I don't know the results, Joe, but I thought that it gave 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.

Speaker 10

Thanks for the insights.

Operator

And the next question will come from Paul Choi with Goldman Sachs. Your line is open.

Speaker 9

Hi, thanks. Good afternoon and congratulations on the commercial progress. On Yorvapath, 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 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?

Speaker 9

Just kind of what you're thinking is there in terms of potentially expanding into that population.

Speaker 3

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 twenty percent coming from we can call the genetic, immunological, everything else, and then about eighty percent coming from the post surgical. And that was very much reflected in our clinical trials. So in our clinical trials, we even have ADH1 patients. We actually have two, even they are extremely hard to find because there are so few of them that have hypochondriacalasia. We actually managed to get them in.

Speaker 3

So out from that perspective is our clinical trial. This is basically reflected the numbers. I don't know, Amy, if you have any comments. You can come with all the different genetic variants we have there.

Speaker 8

Yeah. We had SCADA3 mutations. We had autoimmune polyglandular syndrome type one and DeGeorge syndrome. These were all seen in the trial. We know that they we were seeing them in the expanded access program.

Speaker 8

We don't always get that information on the commercial. From what we hear from our clinicians who are with whom we speak, applying this to everybody.

Speaker 1

Yes.

Speaker 3

So basically it's a hypoparapatient is a hypoparapatient and this is how they define it and I don't think there is any kind of selection from the background demographic because in our clinical trial we have all the broad background demographic, and if you look on the labeling, independent of background. It's not restricted for not treating any kind of background. So we believe what we see in our commercial patient population really just reflects the mirror of what we see in The U. S.

Operator

And the next question comes from Leigh Watsek with Cantor Fitzgerald. Your line is open.

Speaker 11

Hey guys, wanted to add our congrats as well on the strong launch. Maybe just first on your BPAD in terms of contracting in the future. I know you're still in the process. But any guidance that you can provide on sort of the trend for growth to net for the rest of the year relative to Q1? And I have a follow-up.

Speaker 1

Scott or J. H, who will take that? I'll just say some preliminary comments. So on gross to net, you can't get out of the mandatory government rebates. So the biggest driver is likely to be mandatory government rebates that you see in the Medicare and Medicaid channel, which, you know, probably average very low 20%.

Speaker 1

And commercial will depend on contracting of which Jay can comment.

Speaker 3

Yeah, yeah. Jay?

Speaker 5

Yep. Just as I echoed before, the contracting should be fairly minimal, again, reflecting the clinical value proposition that we have. If there's a change, it won't be materially different, just relative to the other markets we've been in.

Speaker 11

Okay, and then just follow-up on the phase two COACH trial. I know you have a starting dose for SKYYTOVA. So is there sort of a titration scheme here that we should think about and what the top dose that you can go to?

Speaker 3

No. It's basic is that we have a starting dose for the TransCon growth hormone in the combination with CMP. And they're also measuring IGF-one as they do in all trials. And if there is any possibility that the physician desire to go up in dose or down in dose, they have the opportunity to do it. But Amy, you know the protocol better than I do.

Speaker 3

And

Speaker 8

may I hear from Lee which condition was she asking?

Speaker 3

As a combination.

Speaker 8

The combination. Yes. So there will be a starting dose that is informed both based on the phase two trial that we're currently doing in the combination trial and also from the many the conditions we're studying where there's short stature but a sufficient growth hormone access, So this will give us based on what we're seeing so far, there can be a very good and safe universal starting dose, typically.

Speaker 11

Great. Thank you.

Operator

And our next question will come from Eliana Murrill with UBS. Your line is open.

Speaker 4

Hey, Thanks for taking the question. Curious just the feedback from the early patient starts, how the titration process has gone on YorbaPaF. Just logistically, you know, kind of any color anecdotes on 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.

Speaker 4

Thank you.

Speaker 3

What I'm typically looking on is numbers. And one of the numbers I look a lot is two things: adherence. Second number I'm looking at is how many patients are dropping out. I think it's two good numbers to look if you have a successful, meaningful treatment of the patient with really addressing a major unmet medical need and really do that. And when I look on the adherence, it's exactly the type as we saw it in the clinical trials, which was really unique and dropout we have given you the German numbers under one percent and we see the same thing everywhere.

Speaker 3

If you start on Europeans you stay on Europeans And that is a chronic treatment. Rest of your life.

Operator

The next question comes from Kelly Shi with Jefferies. Your line is open.

Speaker 2

Hi, good afternoon. This is Jose for Kelly. Congrats on the strong quarter and thanks for taking our question. I have a question in terms of payer dynamics. What are the major reimbursement pushbacks?

Speaker 2

And based on these dynamics, what percent do you estimate you can capture in the mild, moderate and severe segments? And also on the clinical value proposition of the OrbitPath, would you consider running a clinical utility trial to facilitate uptake in milder patients? Thank you.

Speaker 3

Related to your first question, as the part we discussed before, being uncontrolled, party control, or controlled is not part of the reimbursement system. So we cannot really see that. We don't know exactly where they're coming from a different group. We believe many of them, the majority is coming from the uncontrolled because they see the physician much more often. So that is not any part of the reimbursement discussion.

Speaker 3

The second question I need to understand a little bit more about what exactly was you wanted us to address.

Speaker 2

On the value proposition of the Orbit path, potential for preventing renal damage, Would you consider running a clinical utility trial to perhaps facilitate uptake in milder patients who are controlled on SOC?

Speaker 1

Standard of care.

Speaker 3

I don't think really this is the key element for going on a treatment. I have to say the key element to go on the treatment is the benefit you get as a person you're getting normal again. The long term risk is basically what we call a health economic discussion when we talk about the benefit of the treatment for the society. And we are evaluating exactly how we can do that in the best possible manner to really show the financial benefit it is really to be on a Europad treatment not only for the patient, not only for the physician, but basically for the entire society.

Speaker 5

All right.

Speaker 12

Thank you.

Speaker 8

Some literature data already out there that we may be able to leverage showing that some of the conventional therapy itself is toxic to the kidney, right? And that when that is able to be lowered in whatever way, right, that the kidney function gets better. So there may not you know, we'll see what the need is for demonstrating this yet again.

Speaker 13

Very helpful. Thank you so much.

Operator

And the next question comes from Luca Izzi with RBC. Your line is open.

Speaker 5

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 early this week and I believe NBX 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 Eurvipat?

Speaker 5

And then maybe Scott, super quickly, how should we think about the SG and A for the remainder of the year given the meaningful jump this quarter versus last quarter? Any thoughts there? Much appreciated. Thanks so much.

Speaker 3

You mentioned two compounds. One is the catalytic, which being positioned into a phase three trial of ADH-one. Currently, ADH-one is being treated with uropaths and they're coming on treatment today. It has been hard for us to find ADS1 patients. And Sherry, you went into the claim database and how many patients did you find with ADS1?

Speaker 7

Yeah, there were something like three fifty patients who had had a claim associated with ADS1 in the past four or five years and even fewer than that within the last year. So it was a very tiny number. This was from a large national claims database with hundreds of millions of lives.

Speaker 3

So it's not going to change anything for us because we are addressing the position of catalytic into the era of, for example, what we call chronic hypoparietal patient that is not AES-one. You know, I really love science. I don't understand the science there. You're trying to position into a place of a patient that does have endogenous How can you increase the level of endogenous PDAs when they're not producing it? They're already producing on max and there is actually a very nice poster basically showing that they are taking calcineutic into four patients for three days or six or something like that And it's really showing that you cannot increase the secretion of PTH.

Speaker 3

So it's not something I don't understand from a scientific perspective rational but all the data I've seen is also indicating exactly the same thing. Scott was saying you're fine, please. That is one thing. The MPX, this is a once weekly one. And you know, we have the possibility to develop once weekly products for a long time and now I'm talking of the context of once weekly first.

Speaker 3

We in some ways stalled a little bit because we couldn't see the unmet medical need and also because of the desire on different ways of living with a hypoparietal patient where you sometimes need more or less. And it can be done on exercise, seasonal activities, other things that change in your life. And out from that we looked on patients how stable are them, how often do they titrate up and down. And we see only a small part of the patient being stable. So if we wanted to develop a once weekly product, we would develop it as a baseline like basal insulin and then we still will have daily product to really be sure they can adjust them up and down.

Speaker 3

The technology platform they're utilizing in the I always get it wrong MBX or BMX. I cannot remember what is that bike. I always get it wrong. But the element of that is basic technology which are basic as an active entity is an isolated PTH that stays 99.9% associated to basic the element of albumin. I do not know how that ever can activate the phosphate receptor, how they can get into the brain and really restore normal, industrious PTH level in the normal distribution you have out to the body.

Speaker 3

I'm also lost in the science there.

Operator

And our next question comes from David Lebowitz with Citi. Your line is open.

Speaker 13

Thank you very much for taking my question. First

Speaker 5

of all, if you

Speaker 13

look at the NATPARA patients that were left from its withdrawal,

Speaker 9

How long do

Speaker 13

you think it takes until that whole population gets worked through? And then further looking back at NATPARA and looking what you've seen during your first quarter of launch, How would you, aside from the fact that the data is just superior, how would you characterize the difference and what patients are showing interest in this therapy?

Speaker 3

First of all, you cannot compare the clinical benefit between LePar and Europan. LePar had a labeling as an adjunct therapy. Take a little bit of your daily calcium supplement away. Take a little bit of your active vitamin away and then you take NATPAR. You have no positive impact on kidney function.

Speaker 3

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 NaPa patient will be switched. We addressed it a little bit in the beginning of here, the discussion here, where we know that there was a letter from Takeda indicating that they're getting their last shipment now. So and I think as to our knowledge, the shipping is three months.

Speaker 3

So we expect that the last series of Nabapar patients will become over in either end of Q2 or Q3. That is our expectations.

Operator

And our next question comes from Leland Gershell with Oppenheimer. Your line is open.

Speaker 12

Thanks. Great to see the strong execution on USU RiverVath. A couple of questions from us. Just apologies if this had been asked before, but with respect to potential benefits on renal, I know Jen, you had said that you don't see that as a key kind of driver for UriPath uptake. But nonetheless, are you able to comment in the early days of The US launch?

Speaker 12

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 that is second question. Thank you.

Speaker 3

It's not a part of the reimbursement process. We don't see that it's the element that decide of reimbursement or not reimbursement. So it's like the same thing as we discussed on control, party control. Control is not part of the reimbursement process either. It's basic when you look on the labeling, most plans have adapted the way that we basically have in our labeling and it's not defined anyway about that.

Speaker 3

That's first one correction. Amy, we didn't study in what we call severe renal impairment patients and that is not part of our labeling and I think it's stage four.

Speaker 8

That's right. CFR lower than 16 or 30.

Speaker 13

Yeah, which

Speaker 3

are a very, very low number which are stage four where we never started the drop.

Speaker 12

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.

Speaker 3

When we think about airconvulgation, 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 established clinical endpoint is that we will look on linear growth and there will be part of the data we basically will report when we come up with the analyzed height velocity, height as there is and other things for that. And as I said before, I have great expectation. 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.

Speaker 9

Great. Thanks very much.

Operator

This is all the time that we do have for questions. This concludes today's conference call and thank you for participating. And you may now disconnect.

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Earnings Conference Call
Ascendis Pharma A/S Q1 2025
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