Q4 2024 Ascendis Pharma AS Earnings Call and Business Update

February 12, 2025

Good day and thank you for standing by. Welcome to the fourth quarter and full year 2024 Ascendus Pharma Earnings Conference Call. At this time, all participants are in a listen-only mode.

After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you'll need to press star 1 1 on your telephone. You will then hear an automated message advising your hand is raised.

To withdraw your question, please press star 11 again. Please be advised that today's conference is being recorded.

I would now like to hand the conference over to Scott Smith, Chief Financial Officer. Please go ahead.

Thank you very much, Operator, and thank you, everyone.

for joining our full year 2024 Financial Results Conference call.

I'm Scott Smith, Chief Financial Officer.

Speaker Change: at Ascendis Pharma. Joining me on the call today are Jan Mikkelsen, President and Chief Executive Officer, Sherry Glass, Chief Business Officer, Jay Wu, President, U.S. Market, and Amy Hsu, Chief Medical Officer.

Speaker Change: Before we begin, I would like to remind you that this conference call will contain forward-looking statements that are intended to be covered under the Safe Harbor provided by the Private Securities Litigation Reform Act.

Speaker Change: Examples of such statements may include, but are not limited to, statements regarding our commercialization and continued development of SkyTrofa and Yorvapath for the US, European, and other markets, as well as certain financial expectations.

Speaker Change: Our pipeline visits 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 trials.

Speaker Change: and results, our ongoing and planned regulatory filings, and our expectations regarding the timing and the results of regulatory decisions, expected market developments, and our exploration of market opportunities in therapeutic areas outside of endocrinology-rare disease.

Speaker Change: These statements are based on the information that is available to us today. Actual results may differ materially, could differ materially from those in our forward looking statements, and you should not place undue reliance on these statements.

Speaker Change: We assume no obligation to update these statements as circumstances change, except as required by law.

Speaker Change: For additional information concerning the factors that could cause actual results to differ materially, please see our forward-looking statement 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 today, February 12, 2025.

Speaker Change: Transcon Growth Hormone, or Transcon HGH, is approved in the U.S. by FDA and in the EU has received MAA authorization from the European Commission for the treatment of pediatric growth hormone deficiency.

Speaker Change: 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.

as investigational products, the safety and effectiveness of product candidates.

Speaker Change: have not been reviewed or approved by any regulatory agency. None of the statements during this conference call regarding our product candidates shall be viewed as promotional.

Speaker Change: On the call today, we'll discuss our full year 2024 financial results and we'll provide further business updates. Following some prepared remarks, we will then open up the call for questions.

With that, let me turn it over to Jan.

Thanks, Scott, and good afternoon, everyone.

Speaker Change: 2024 was a pivotal year for Ascendance as we achieved key milestones that set us up to deliver strong growth and value creation in 2024 and beyond.

Speaker Change: with Scott Hofer firmly established as a high-value growth hormone brand in 2024.

Speaker Change: With further penetration in Pediatric Growth Hormone Deficiency and planned commercial launches across multiple indications and countries, we expect sustained valuation for the transgrom growth hormone franchise in the coming year as outlined in our Vision 2030.

Speaker Change: Importantly, EUROPAT, the only FDA-approved treatment for hypothyroidism in adults, is now launched in the U.S. and has already begun to establish itself as the new standard of care.

Speaker Change: Given the long-standing need for a treatment option like uropaths, we are seeing significant early demand in both the patient and physician communities.

Speaker Change: And we are pleased with the pace of peer approval so far.

Speaker Change: The large global population living with a significant hyperparent disease burden underscores the potential for uropaths to grow into a multi-billion dollar product over time.

Rounding out our End of Crime Rare Deceased Portfolio

Speaker Change: Transcon CMP clinical data demonstrated it could be a highly differentiated product with a unique profile that represents a major step forward in the treatment of achondroplasia and other growth disorders.

Speaker Change: We believe the once-weekly transplant CMP data demonstrates superior linear growth and benefits beyond linear growth, supporting our proposed label for treatment of achondroplasia.

Speaker Change: For this reason, following our pre-NDA meeting with FDA, we are on track to submit an NDA to FDA this quarter, followed by a MAA submission to the EMEA in the third quarter this year.

Speaker Change: All three of these endocrine rare disease medicines demonstrate the value of our transcom technology platform and its potential to address major medical needs with highly differentiated products.

Speaker Change: We are also bringing the transgone technology platform beyond endoprime rare diseases.

Speaker Change: In large patient populations, through our collaboration with Novo Nordisk in metabolic diseases such as obesity, type 2 diabetes, and cardiovascular diseases.

in Optimology to the Creation of Iconis.

Speaker Change: We have also expanded the Transcon technology platform to incorporate protein degraders at their promising era where we believe that new...

Speaker Change: The new Transcom technology platform will expand our pipeline with additional potential blockbusters.

Speaker Change: We entered 2025 with a very strong financial position with a cash of 665 million euros on our balance sheets, including the 100 million dollar off-run payment that we received from Novo Nordisk last month.

Speaker Change: As a result, we are in a strong position to invest in commercial uptake and new product development to drive continued revenue growth.

Let me review our key programs in RARE.

and you'll find the seeds in more detail.

Speaker Change: In the U.S. Skartova was launched just over three years ago.

Speaker Change: Today is the treatment of choice, and at the same time, growing the growth hormone market. Importantly, with the pediatric growth hormone deficiency indication alone, we are currently addressing only half of the existing U.S. growth hormone market.

Speaker Change: both are on path to expand Kryptova's addressable market in multiple ways. Near term, we expect U.S. approval in adult growth hormone deficiency this year with our PDUFA date on July 27.

Speaker Change: Longer term, we will investigate scatophagy in additional therapeutic areas through a basket trial including idiopathic shock stages, shock deficiencies, Turner syndrome, and SDA.

Speaker Change: In the third quarter of this year, we plan to submit an R&D application for this basket trial to the U.S. FDA.

planned commercial launches across multiple countries.

Speaker Change: In 2024, scotophile volume increased 84% in the United States, with premium net pricing of 3x compared to one daily growth amount.

Speaker Change: SkyTofa achieved revenue of around 200 million euros in 2024, supporting the potential for it to become a blockbuster product over time. Moving to Europass.

Speaker Change: 2024 was a critical year for Europe, with commercial availability in Europe starting early in 2024 and then this past December in the US.

which you have had also available through NAMED patient programs.

Speaker Change: patient in mutable context living with hyperparathyroidism can begin to assess this long-awaited treatment option.

Speaker Change: Hypoparietal arrhythmias represent a large global market opportunity for sentient beings to address a major unmet medical need.

for an effective and well-tolerated treatment option.

Speaker Change: To create durable, long-term leadership for uropaths, we are building this market by educating physicians about the well-documented limits on risk of conventional therapy and the clinical benefits seen with our PTH treatment.

Speaker Change: In the U.S., we estimate there is about 70,000 to 90,000 patients with chronic hyperparathyroidism.

Speaker Change: most of whom are currently using conventional therapy of oral calcium and active vitamin D.

Speaker Change: Our claims analysis demonstrates that 10,000 to 15,000 of these U.S. patients are uncontrolled, and 30,000 to 35,000 are not.

are partly controlled.

Speaker Change: We believe your pet can sustain growth over a long time, as the vast majority of patients with hyperparathyroidism qualify for PTH treatment per the current international guidelines.

Speaker Change: Less than two months in the U.S., your path longs. Initial demand is strong, with 908 patients with prescription as of February 7, 2025.

Speaker Change: This includes prescriptions for 539 unique prescribers in around 44 states.

Nearly 80% of the world are new to Europeans.

Speaker Change: the majority of whom are switching from conventional therapy, with the remaining being existing patients from the transcon PTA clinical trial or expanded access program.

Speaker Change: Discussion with payers are ongoing and as expected with a novel speciality product we estimate the majority of insurance approval will take about four to eight weeks.

We are pleased with the initial pace of insurance approval.

Speaker Change: across commercial and government payers, and have shipped rear burst drugs to patients in around 35 states.

Thank you.

Speaker Change: Outside the U.S., we remain on track for additional commercial launches in what we call European countries.

Speaker Change: where we expect to add five or more countries this year.

Speaker Change: We also expect launches in multiple international markets in 2025, further expanding our global reach where we have signed 8 exclusive distribution agreements covering 50 plus countries so far.

Your path is a unique product.

Speaker Change: Our broad and extensive clinical data include three successful phase III trials in the U.S., Europe, Japan, and China covering diverse disease groups, including post-surgery, autoimmune, ADH1, idiopathic hyperparathyroidism.

Speaker Change: Last year, we presented three years' data from our Phase II path-forward trial. And later this year, we plan to present four years' data demonstrating excellent patient retention and sustained serum calcium control and bone health.

Speaker Change: Sustained reduction of calcium phosphate product, independent from conventional therapy, and normalization of 24-hour UNR calcium equation.

The data also showed sustained improvement in kids.

and Alex.

listening to Transcon CLP.

Speaker Change: Acondoplasia remains a disease with high on with medical need and we believe Transcon CMP has the potential to be a highly differentiated treatment option.

Speaker Change: In the pivotal approach trial, transcon CMP demonstrated significant improvements in linear growth and body proportionality compared to placebo, as well as benefits beyond linear growth.

Speaker Change: As one example of benefits beyond linear growth, we have shown data demonstrating significant improvement with tricotin CMP treatment on leg bone.

Speaker Change: and common and devastating complications in acondyplasia that can result in pain, impaired physical function, need for corrective surgery and a negative impact on quality of life.

Supporting transcon CMP potential as a best-in-class treatment for achondroplasia.

Speaker Change: Following our productive pre-NDA meeting with FDA, we plan to submit an NDA for the treatment of aconitocasia during the first quarter of 2025 and submit an MNAA for treatment of children with aconitocasia to the EMEA during the third quarter of 2025.

Speaker Change: We believe Transcon CLV will be setting a new bar for treatment of aconitoplasia.

Speaker Change: to further raise this bar for linear growth and other clinical benefits.

Speaker Change: We are also working on a combination treatment of transcon-CMP and transcon-cloctamol in achondroplasia.

Speaker Change: Tendage is uniquely positioned to bring these two once-weekly medicines together in a combination treatment.

Speaker Change: providing two different modes of action to potentially improve outcome in achondroplasia and other growth disorders.

Additionally, during the fourth quarter of 2025.

Speaker Change: We plan to submit an R&D or similar tool to investigate transgone CMP alone or and in combination with transgone growth promoter for the treatment of hypochondroplasia.

Speaker Change: Look into how we are expanding our pipeline in endocrine rare disease.

Speaker Change: We are expanding into additional product candidates beyond our first three successful in medicine as we disclosed at the JP Morgan conference. In addition, we continue to broaden the reach of our platform outside endocrine rare disease.

Speaker Change: and two collaborations in therapeutic areas affecting a much greater patient number.

Speaker Change: For oncology, our internal development continues to focus on transgon IL-2 beta-gamma, aiming for accelerated approval in one or more locations. In early 2004,

Speaker Change: We announced the formation of CONUS to explore the development of transcript-based therapies in ophthalmology.

Speaker Change: In November, we announced a multi-product collaboration with Novonortis covering metabolic and cardiovascular diseases with a lead program to develop once-a-monthly transcontraceptive type.

Speaker Change: This entitles us to escalating tiered mid-single-digit royalties on global net sales of approved products. Finally, as I mentioned earlier, we have expanded Transcon to incorporate protein degraders.

Speaker Change: a technology with very promising potential within as well as outside rare endocrine diseases.

Ascended today.

Speaker Change: With approved commercial products, a strong pipeline, and guided by our values of patience, science and passion, it's position to continue driving rapid and sustained growth in the years ahead.

I will now turn it over to Scott Smith.

Thanks, Jan Mikkelsen.

Scott Smith: to successfully launch Yorvapath on a global basis, to build on Skycrows' leadership position in pediatric GHD by expanding the U.S. label to adults, while maintaining its premium net pricing of 3X compared to daily growth hormone.

Scott Smith: and to submit TransCon C&P for approval to treat achondroplasia in children in the U.S. and the EU.

Scott Smith: I will touch on some key points surrounding our fourth quarter and full year financial results, but for further details, please refer to our 20-F file today.

Scott Smith: Skytropa volume increased 37% in the fourth quarter of 2024 compared to the fourth quarter last year, while reported revenue was 58.5 million Euro compared to 64.2 million Euro reported in the fourth quarter of 2023.

Scott Smith: In the fourth quarter this year, SkyTropha revenue benefited from volume growth and a favorable sales adjustment of 4.6 million euro attributable to periods prior to January 1, 2024, which was offset by higher sales deductions compared to the same period the prior year.

Scott Smith: Backing out this favorable sales adjustment, SkyTrofa revenue was approximately 54 million euro for the quarter.

Scott Smith: Sequentially, SkyTropha volume increased 16% in Q4 compared to Q3 while pricing was stable.

Scott Smith: We expect revenue growth to continue to track closer to script growth unless payer mix changes substantially, as there have been no major contracting changes compared to Q4 last year.

Scott Smith: Shifting to Yorvapath, as previously reported, fourth quarter Yorvapath revenue increased to 13.6 million euro, bringing total 2024 revenue to 28.7 million euro.

As Jan noted, the launch of the OverPath in Germany

Jan: and Austria in 2024, together with the initial demand and progress with reimbursement in the U.S. so far in 2025, has been very encouraging.

Jan: We expect that UrbaPath will have a significant impact on our financial profile in 2025.

Jan: While it is early in the launch, we look forward to sharing data on parameters like enrollments, prescribing HCPs, time to reimbursement, etc. to provide more detail on launch dynamics.

More importantly, everything

Jan: We are seeing in the early launch phase supports our view that, over time, we expect Yorvapath to be the standard of care for patients with hypoparathyroidism and to become a multi-billion dollar product.

Jan: Closing out the top line, total revenue for the fourth quarter was 173.9 million euro, including revenue recognition of the 100 million dollar upfront fee related to our collaboration with Nova Nordisk, as well as other revenue from partners.

Jan: To be clear, although we recognize the $100 million from NOVO in 2024 for accounting purposes, cash was received after year-end in January.

Jan: Total revenue for the full year 2024 was 363.6 million euro.

Jan: Turning to expenses, for the fourth quarter R&D costs totaled 79.3 million euro compared to 90.9 million euro during the fourth quarter of 2023.

Jan: The 13% decline was largely due to lower external development costs for transcon growth hormone and transcon PTH, as well as the Iconis spin-off.

Jan: SG&A expenses in the fourth quarter of 2024 totaled 80.2 million Euro compared to 64 million Euro during the fourth quarter of 2023.

Jan: The 16 million euro increase was due to higher employee costs, including the impact of additional headcount supporting global commercial expansion, most of which came toward the end of the year.

Jan: Similarly, we spent more on external commercial costs to support launch activities and plan to continue to do so in 2025 with the global launches of UruPath.

Jan: Total operating expenses were 159.5 million euro for the fourth quarter of 2024, a 3% increase compared to 154.9 million euro during the fourth quarter of 2023.

Jan: Total operating expenses for the full year 2024 were 598 million euro.

Jan: Net cash financial expenses for the full year 2024 were less than 1 million euro.

Jan: while net finance expenses for the full year were 74.4 million euro driven primarily by non-cash items.

Jan: We ended 2024 with cash, cash equivalents, and marketable securities totaling 560 million euro compared to 399 million euro as of December 31, 2023.

Jan: Including the 100 million dollar upfront payment from Nova Nordisk that was received in January 2025, cash at the end of 24 would have totaled 655 million euro.

Jan: Finally, as separately announced, we expect to use approximately $25 million in the first quarter of 2025 to preserve approximately 200,000 ADSs held as Treasury shares.

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

Jan: As a reminder to ask a question please press star 1 1 on your telephone and wait for your name to be announced. To withdraw your question please press star 1 1 again.

Jan: In the interest of time, we ask that you limit yourself to one question and one follow-up.

Please stand by while we compile the Q&A roster.

Our first question comes from Jessica Fye with J.P. Morgan.

Jessica Fye: Hey guys, good afternoon. Thanks for taking my question and congrats on this drawing, your repast script number and breadth of prescribers. I have one question with a few parts, mostly just confirming some stuff. First,

Jessica Fye: Can you confirm that the 908 figure is unique patients and not like cumulative scripts including refills for example?

Jessica Fye: 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 your path, 16% from the EAP and OLE.

Jessica Fye: 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 U.S. market for coming up on eight weeks? Thank you.

Jessica Fye: Thanks, Jess, a lot for the questions. I think we can confirm nearly all the answers.

Jessica Fye: Jay and Sherry will you confirm all the numbers so be sure

Speaker Change: Happy to. Jessica, thank you for the question. The first part of your question, from an enrollment standpoint, we can confirm that is unique patient enrollments, so not cumulative repeat.

Speaker Change: I think the second question you asked was the percentage of patients that are coming over from either the EAP or clinical trials versus those that are new to Urolopath.

Speaker Change: So, about 20% of the 908 is existing patients from transcon, PTH, EAP, or clinical trials, and then 80% of patients are actually new to your path.

Jessica Fye: And then the third question that I think we heard you ask, Jessica, is more around the four to eight weeks.

Jessica Fye: But keep in mind, too, that based on the timeframe for which the drug has been on market, it is still a very nascent period of time where, for many of the patients enrolled, that time period hasn't actually occurred yet, right? So we'll need more time to get a better sense of that, but that is our initial estimate today.

Speaker Change: Thank you. For my follow-up, can you provide the UriPath patient number outside the U.S.?

Speaker Change: We have not broken that number down. It is still increasing as we expected to do. We are still...

Speaker Change: only in Europe direct countries, full commercial in Germany and Austria. We have our AP2 program in France.

Speaker Change: non-promotional program that also is enrolling reimbursed patients and then we have our international market in this way and the patient number are increasing to exactly as we have expected but we have not broken down the numbers

Speaker Change: Our next question comes from Tazeen Ahmad with Bank of America.

Hi guys, good afternoon and thanks for taking my questions.

Jan: 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?

Jan: And can you give us also any color that you might have on what proportion of these patients may have had previous experience with NEPPARA? And then I have a follow-up.

Speaker Change: Yeah, the question you are addressing is a question where we still lack all the information to give you a really concrete answer that you want to have. What we have done in our market research, we have looked a lot on claims.

data and we have defined

Speaker Change: patient group, which we call the 10,000 to 15,000 patients, which we will call not controlled on

Standard Therapy

Speaker Change: or conventional therapy, and this patient group is basically seeing the physician.

at least four times a year.

Speaker Change: 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...

Speaker Change: 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.

Speaker Change: definition, if that is true or not, but that is our expectation in this.

Speaker Change: The second question you raised about how many patients you have on.

therapy now. It's a number we are

Speaker Change: waiting to 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

Speaker Change: part of our, you can say, journey of getting them 100% reimbursed.

Speaker Change: We were extremely positive on the broadness of having reimbursed patients, both from

the commercial.

Speaker Change: set up, but also for the government side. And we basically are getting reimbursed patients 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.

Speaker Change: We are also feeling that we are in a very, very strong position.

The vast majority of the patients are on label.

Speaker Change: and BASIC, they're also fulfilling exactly the criteria that justifies for them to be on a PCH treatment. So we are really highly positive on the development we have seen in the reimbursement spectrum.

Speaker Change: Okay, thanks Jan. And then I just wanted to clarify, I think you had said that you were considering providing sales guidance for Uruvy Path for the full year sometime in the middle of the year. Does that still the plan?

I think it's a little...

Speaker Change: I think we would like to give you guidance when we believe in the numbers. And I think that is the criteria we take up. So depending on how we see the long scoring and how we see different regions, we will come up with.

What we call revenue guidance

Speaker Change: exactly when we're feeling that we feel the confidence. Scott always likes to give up a lot of numbers so you can hear in his list and what he said in he prepared remark that he will come up with a lot of numbers.

Speaker Change: elements that can give you the best possible fundament to build up a solid model in your own way.

Speaker Change: Comments for you Scott? Yeah we will as we said we're excited to continue to share with you the underlying parameters driving the launch and we plan to do that in the quarters ahead.

Thank you.

Our next question comes from Gavin Clark-Gartner with Evercore ISI.

Gavin Clark-Gartner: Hey guys, congrats on the very strong progress so far. Just had one question on yorvapath and one on CNP

Gavin Clark-Gartner: For Yorvapath, how have your discussions with payers gone so far and what are your assumptions for how the 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.

Gavin Clark-Gartner: 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.

Speaker Change: Yeah, I think Jay will come with some further flavor about the contracting situation, but also, as I said before, I've been lucky to see most of the U.N. criterias, and I have been...

Speaker Change: Really pleased how they are aligning to the labeling, but I believe Jay will come up with further comments related to the commercial contracting landscape.

Speaker Change: Yeah, so thank you for the question. As you can expect for any new launch, we are still in the early stages of establishing policy with payers. So just as an example, most medical policies right now for our large national players haven't yet even published a policy yet, right? So our conversations have all been productive to date, really focusing on the clinical value proposition of the drug.

Speaker Change: As you can see, and based on our data from our array of clinical studies, we feel really positive about the benefit that we can convey to the patient community, and we're having a great conversation around that.

specifically your question around prior auths

Speaker Change: Again, as part of those negotiations and discussions, wanting to ensure that the standard PA is consistent to label, both in terms of reauthorization timelines, tests that may need to be conducted to ensure that, again, it is consistent with label. So while many of those plans and policies still need to take some time to come into fruition, we're feeling encouraged by those conversations.

Speaker Change: So the CMP question is always looking in the crystal ball.

because you asked me, Jan.

Speaker Change: CMP and Glutamone. And why I think it's really really are interesting, because it's two different compounds with very different physiological mode of action. And if I ever believe in science, which I always do a lot,

It should be a great combination because it's synergistic partway.

Speaker Change: and when I look on what you can do when you address

the hyperactive tyrosine kinase

Speaker Change: system that you have in acondoplasia, you can do that with either tyrosine kinase inhibitors, you can do it by CMP as short-acting, or...

Speaker Change: you can say continuous exposure. I think it's easier to saturate the analyzed height velocity in this part of the

Speaker Change: integrated effect you expect to give as a treatment option in acondoplasia. One of them that is easiest to hit is basically the analyzed height velocity.

And when I look at all the data...

Speaker Change: It's likely are going around 5.5 to 6.0 on analyzed high velocity, which basically are reflecting a little bit what the science also will say. You are moving a brake and you restore normal growth.

Speaker Change: So, out from that perspective, and this is why I love the Grotamo, because what is Grotamo? This is like sitting in a car and then you suddenly get rid of the speeder.

Thank you very much.

Go ahead.

Speaker Change: That's helpful. Thanks guys. We'll leave it there for now. Our next question comes from a line of Lee Wasek with Cantor Fitzgerald.

Lee Wasek: Hey guys, wanted to add my congrats on the Strong Your Way Path 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 you know initial titration, whether it's consistent with your clinical trials.

Speaker Change: Yes, it's a question we tried to address before, but I think, Sherry, you're also sitting with a lot of data, potentially you can some way describe the limitation that we have in our dataset really to answer that question today.

Sure, and thanks for the question, Lee.

Speaker Change: So what we don't actually have data in terms of the enrollments on patient severity. What we do know though, is a couple of important things. One is that, as Ian said earlier, the most severe patients are going.

Speaker Change: most frequently to the endocrinologist. So by that practical matter of them getting into the office to get the prescription, it's likely that we have severe patients initially getting some of the first prescriptions.

Speaker Change: And as Jan also mentioned, you know, there are a number of those.

Speaker Change: uncontrolled patients in the U.S., there's 10 to 15,000 of them and then a number more people who are partially controlled. So to the extent that, you know, we are starting to see some of the first patients being the more uncontrolled and severe patients, we know there's still a tremendous amount of room for expansion beyond that.

Thanks, Jay.

Thank you.

Our next question comes from Derek Archila with Wells Fargo.

Speaker Change: Hi, this is Yvonne for Derek. Thanks for taking our questions and congrats on the progress.

Speaker Change: A quick one from us on your BPATH, so can you provide some color on what type of dogs are prescribing your BPATH? Are these like high-volume dogs or are you starting to get some dogs 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.

Speaker Change: Yeah, let me take the last question before I turn it over to Jay or Sherry. What is happening in our ERP program, the last...

Speaker Change: basic three months, and some of them got them in December to in January. So the hundred plus patients that we are converting from our ERP clinical trial over, we'd likely come into conversive drug starting in February and then the majority in March. So that was the second question, and I think Sherry or Jay, who will take this? Will you start? Sure, happy to start. I think the question

Speaker Change: Naturally, you would expect, directionally, physicians that are treating perhaps a greater number of patients, right, we're naturally also focusing on them quite heavily.

Speaker Change: So from a field standpoint, we're actually over 50% reach for a lot of our priority physicians. So I think naturally you're going to see some directional lean in that direction. But more importantly, what you're seeing is a broad outreach and broad interest across the provider community agnostic of DESA.

And maybe I'll just.

Thank you.

Our next question comes from Yaron Werber with TD Cowan.

Yaron Werber: Right, so thanks so much. I got maybe a couple of questions.

Speaker Change: Scott, for you, and then, I don't know, Jan, if you want to take the next one. I think, Scott, you said that toward the end of the year you could be, you could reach profitability on a cash basis.

Speaker Change: Does that still sort of hold, and what would drive that? How do we, is it going to be your VPAS is the biggest driver? And then secondly, as you think about reimbursement for your VPAS, are you expecting with time prior authorizations, or are you expecting not to have any prior auths? Thank you.

Okay, I think Nurse Scott would like to say something.

Scott Smith: Yeah, I think you're exactly right. Europe has to be a big driver of the ability to cash break even. It's a strong launch in Europe for us. Obviously, we're off to the races here in the U.S. and it's a very profitable product.

Scott Smith: So I think we agree that that'll be a strong driver of break-even potential this year. Yeah, but I also believe you need to look that the U.S. is the numbers we're giving you today. But we basically have a global effort.

Scott Smith: And we are also seeing that at the end of this year, we are starting to take a much, much higher gear in our Europe-directed countries. We will also see that the international market is starting to really, really get engaged.

launches in for many of them.

Scott Smith: But the 25 will be where we start in both days.

Scott Smith: because it's such a large single market, so sure it will dominate in the beginning of the year, but just remember the number of patients outside the U.S. with hyperplasia is so much, much, much larger, perhaps 4-5-fold than what you have in the U.S.

The second one, will you take that?

Scott Smith: Yeah, sure. I mean, from a prior auth standpoint, I think the question was, what do we expect for your path? It is a specialty product, and I anticipate there to be some basic questions, even if it's simple as,

Scott Smith: who's prescribing it, right? I think whether it's an endocrinologist, whether it's a nephrologist, et cetera. And I think secondly, what you're gonna see and should expect is there's gonna be wide heterogeneity across what you'll see across both national and regional plans.

Scott Smith: I think as we alluded to before, we are seeing approvals across commercial payers, public payers, and even absent of there being a formalized policy at some of the large national payers, we still submit generic prior auths to seek exception through that policy. So again, some of this will take time to evolve and some of it we would expect to change, but we will see probably a wide range across the various plans.

Thank you.

Joseph Schwartz: Our next question comes from the line of Joseph Schwartz with Lear, Inc.

Joseph Schwartz: but one to two of their patients, whereas in the U.S. you are expecting physician suits.

prescribe the drug to.

Joseph Schwartz: 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 one and a half of their patients. So I was just wondering if you're still expecting each physician in the U.S. to prescribe Ropap to about two to three of their patients, and where does it go from there?

Joseph Schwartz: Yeah, I think it's something I accept what I said and I stand by what I said before.

Speaker Change: and I think the pattern we see now is an early launch. I have seen and followed some of the physicians that were part of our EIP and clinical trial and some of these physicians have already made prescriptions up to 20 patients.

Speaker Change: So, no doubt the boldness of that will come, but in some way this is an early launch, one to two months in the launch, so I will expect that to see and come in the future.

Speaker Change: Okay, great. That's helpful. And then a clarifying question. Are any of the 908 prescriptions included in the URV PATH sales from 2024, considering the drug was available mid to late December? Thank you so much.

Speaker Change: Got it. I mean, strictly speaking, so we did ship a very small amount in December. It was basically immaterial to the total. Yeah, so in practical, no.

Kelly Shee: Our next question comes from the line of Kelly Shee with Jeffreys.

Kelly Shee: Congrats on the great quarter and on the manufacturing front, how should we think about the capacity to meet the increasing demand of URP parts throughout the year? Thank you.

Kelly Shee: Yeah, I think one of the things we have been very, very proud of at Ascend is really our robust supply chain. We have seen how we have managed to go up in high demand on the shortest of the daily growth mode and could fulfill all requirements for all wishes for having, for example, the

Kelly Shee: We're using the same solid supply chains, the same infrastructure. We follow it the same way. I personally get a weekly report on AVI.

Kelly Shee: 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 position.

Thank you.

Speaker Change: Our next question comes from the line of Ellie Merle with UBS.

Speaker Change: Hey guys, thanks so much for taking the question and congrats on the launch progress.

Speaker Change: Just in terms of how we should think about the U.S. YorbaPath script cadence, I guess

Speaker Change: you know 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 abs each week?

with their bolus up front.

Speaker Change: 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

Speaker Change: ease of reimbursement say between the moderate patients versus the severe patients and your expectations there. Thanks.

Speaker Change: I think the question related to what we call one control to party control and the patient we have, somebody answered this, to our best of knowledge, we have no clear data of the patients that is...

Speaker Change: 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 frequency than anyone else.

Speaker Change: like life it is that like Amy sitting here if you want to see Amy our CMO at Stanford it takes how many weeks now Amy?

Amy: 12. 12 weeks to see her, so it takes a little bit time to get an appointment. So we don't believe that it's a lot of build-up demand. It's basically patients that come in and have the...

Amy: The need to be on a treatment, and I think it's pretty obvious for me when I see the benefit that all the patients get, yes, everyone should have a treatment option and come on PTAs like everyone on type 1 diabetes should have insulin. This is a hormone replacement therapy in this perspective.

Thank you.

Related to the

Speaker Change: Reimbursement system. I do not know Jay if you have further comments compared to that

Speaker Change: Yeah, as we mentioned before, because this is such early days, I don't think we would be able to draw a meaningful pattern or a trend based on just the few weeks that we've been in here. I think I go back to the statement we made before. We are seeing approvals across all both commercial and public payers, but fully recognizing too that because many policies aren't in place.

Speaker Change: A lot of these cases are exception by exception basis and we believe that after at least a few more months we'll have a better sense of how these policies shake out and what that more stable trend should be.

Great, thanks.

Speaker Change: Our next question comes from the line of David Leibovitz with Citi.

David Leibovitz: Thank you for taking my question. First on Skytropha, you have the Padupha Day coming up this summer for the adult.

David Leibovitz: I'm just curious, how should we be thinking about that in terms of what that ARCA opportunity actually presents?

Speaker Change: Yeah, I think Amy can explain on the unmet medical need that really exists in adult growth hormone deficiency. From my perspective, there are two key things I really, in some way, are reflecting over a lot. First of all, it's a...

Speaker Change: air with extremely low penetration to our best knowledge is under 5, 6, 7 percent so you can say there is a huge opportunity for growth

Speaker Change: And the other thing is that has been a huge burden, basically, to be in the treatment with daily growth hormone. But, Amy, you can explain and tell about what is really the unmet medical need, the burden of having adult growth hormone deficiency.

Speaker Change: Sure, happy to do so. So many people arrive at growth hormone deficiency in adulthood following something involving organically the brain, right? So a brain tumor or a brain cancer or its treatment, either surgery or radiation. That's the

Speaker Change: at least 50% or more. So many of them, because they are missing function of the pituitary gland, are taking many hormone replacements. And growth hormone is just one on that list.

So, they have often times said, if I...

Speaker Change: If it's something we can make simpler, right, that the community could provide more simply to them, it is something they would consider, knowing that it adds to their metabolic health and overall endocrine health, right? But, of course, their first...

are the two life-saving hormones from the brain,

Speaker Change: forget and those are pills so after that they are willing to think about injectable less frequent

Speaker Change: therapies, knowing it is good for their overall health. We could admit that growth hormone deficiency is not immediately life-threatening the way that if you don't take thyroid or adrenal replacement is.

Speaker Change: So we think we're in a nice sweet spot here. It's a hormone that should be replaced and if it meets patients expectations We think there would be

Thank you.

Speaker Change: Thank you for that. And just jumping over to your path, I'm just curious as to thoughts on potential competing pivotal data coming out this year and how you think the market openly might evolve.

Speaker Change: Yeah, if you reflect on people's data, I don't think there is anyone else than one compound.

Speaker Change: the amyloid compound, and to our best knowledge, when we talked with centers, the last patient in was in the beginning of November. So I'm still waiting.

Speaker Change: to see the results of the PIVL 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.

Speaker Change: I have not seen anything else. When I go back to the signs and one of our key values, this is not a hormone for patient therapy, this is an

Speaker Change: It's not even reflecting the normal biology where you are basic activating both the PTF1 receptor and the PTF2 receptor.

Speaker Change: And by doing the different mode of action, you already are from the data can see that it can never substitute as an hormone replacement therapy.

Speaker Change: element on where you see is not restore normal function for example

Speaker Change: in the kidney, both for example related to phosphate excretion, see for example it can up lowering.

Speaker Change: 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.

Speaker Change: So we are looking forward to see the data. We would like to see the data and we hope one day they will come out. They should be out there now because it's so long time since the last patient came into the visit.

Our next question comes from Paul Choi with Goldman Sachs.

Paul Choi: Hi, thanks, good afternoon, and congrats on the early launch success with Yorvapath in the U.S. My first question is on Yorvapath, and as we look at consensus numbers, the street is modeling less than 900 patients in the U.S. for this year, and you've already exceeded that in terms of scripts, so I was just wondering, recognizing that you're not giving guidance and...

Paul Choi: not and the reimbursement is a work in progress just your level of comfort with with the street a revenue number for the full year Possibly or any color around that would be great my second question is regarding CMP and the hypochondria pleasure program that you plan to file an I and D 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

Paul Choi: and any corollary there would be helpful. Thank you very much.

Paul Choi: Yeah, let me take the first part together with Amy, because we are really dedicated to be the leader in growth disorder.

And when we see

Gautam Mohan, and Tanskant CMP.

Paul Choi: We really will do the best for the patient in each case.

How can we design an optimal treatment in this way?

Paul Choi: And what we're doing now in hypochondroplasia is defining what is really the pathway forward for us. It's then just a transcom CMP treatment.

Paul Choi: But I think Amy can give you a little bit the background why we some way think that when we moving into hyperchondroplasia it's one of many growth disorder we actually would like to focus on.

Paul Choi: and the actual design can also be a different stage process.

Thank you.

Paul Choi: So, exactly, you know, we are taking the time to give hypochondroplasia the opportunity it deserves. While some of hypochondroplasia overlaps highly with achondroplasia and the genetic...

variant arises on the same FGFR3 receptor.

Thank you very much.

The

Paul Choi: gene changes are very different along that whole receptor, and we are understanding that hypochondroplasia has a unique phenotype. It's a broader phenotype and very unique. So along those lines, we are still figuring out It's an active process What what's the population of interest to us?

Paul Choi: where we can benefit from them and therefore the different ways to find a regulatory path forward, which I think gets to your question about how much time do we put into the run-in.

Paul Choi: I couldn't tell you now, as we are still figuring a lot of this out, but I think the condition and other conditions of short stature, especially on the skeletal dysplasia genetic side, right, deserve a very good thorough understanding to find the best way.

I'm

And we are in this position that potentially we will

Paul Choi: start both trials at the same time and CMP and then combination trial and then we will basically evaluate the data and find out that potential we will go for an approval for both and then they can choose the optimal treatment.

Paul Choi: from the physician how best can serve the patient needs in this way. If they really want to have a much more extensive linear growth, likely the combination therapy will be the preferred option.

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