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spk14: Good day, and thank you for standing by. Welcome to the fourth quarter 2023 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 will need to press star 101 on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question, please press star 101 again. Please be advised that today's conference is being recorded. I would now like to hand the conference over to your first speaker today, Tim Lee, Senior Director of Investor Relations. Please go ahead.
spk12: Thank you, Operator, and thank you, everyone, for joining our full-year 2023 Financial Results Conference call. I'm Tim Lee, Senior Director of Investor Relations at Ascendance Pharma. Joining me on the call today are Yen Mickelson, President and Chief Executive Officer, Scott Smith, Executive Vice President and Chief Financial Officer, Dr. Steena Singhal, Executive Vice President, Head of Clinical Development Oncology, and Joe Kelly, Senior Vice President, Head of U.S. Commercial Endocrinology. 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 statement may include, but are not limited to statements regarding our commercialization and continued development of Skytrofa for the U.S. and European markets, as well as certain financial expectations for 2024, our commercialization and development of your path in the EU and expected timing of the FDA review and potential launch of Cranscom PTH in the US, our pipeline candidates and our expectations with respect to their continued progress and potential commercialization, our strategic plans, our goals regarding our clinical pipeline, including the timing of clinical results our ongoing and planned regulatory filings, and our expectations regarding the timing and the results of regulatory decisions, our ability to create value in multiple therapeutic areas outside of endocrinology rare disease, our progress towards Vision 2030, and the potential success of ICONIS. These statements are based on information that is available to us as of today. Actual results 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 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 later today, February 7, 2024. Kranskon Growth Hormone, or Kranskon HGH, is approved in the US by FDA, and the EU has received MAA authorization from the European Commission for the Treatment of Pediatric Growth Hormone Deficiency. The European Commission has granted marketing authorization for Kranskon 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. As investigational products, the safety and effectiveness of product candidates 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. On the call today, we'll discuss our full year 2023 financial results and we'll provide further business updates. Following some prepared remarks, we'll then open up the call for questions. With that, let me turn it over to Yen.
spk07: Good afternoon, everyone. 2023 was a transformative year for Ascendis Pharma. We streamlined the company, including our structure, our processes, and operating expense allocation. And at the same time, we believe we are on track to achieve our vision 3x3 by 2025. Skytrover is the leading growth hormone product in value in the U.S. belongs in Europe direct, and international markets is underway, beginning with Germany and Austria. And our clinical program for transplant CMP is progressing to what people will data expected in Q4. We believe Ascendis is advancing to become a leading biopharma company with a strong focus on endocrinology rare diseases. and capabilities to also create value in other therapeutic areas, as we're doing in oncology, ophthalmology, and metabolic diseases. Because of the decisions we took in 2023, we believe Ascendis is a leaner, more efficient organization in 2024, well-positioned to fulfill vision 2030 with three independent endocrinology rare disease blockbuster products and expand our engine for future innovation. Now, let me provide an update on each program. From the beginning, we designed Skytover to be a best-in-class growth hormone product by releasing unmodified somatopoeia. By addressing the needs of the patient, caregivers, physicians and payers. Skytopa achieved U.S. market value leadership in 2023, just two years after launch. We estimated that Skytopa penetration in the U.S. pediatric growth hormone deficiency patient population was around 16% at the end of 2023. With Skytrofa expanding the U.S. growth hormone market to the potential of 3 billion U.S. dollars, we believe Skytrofa has the potential to be a blockbuster on U.S. revenue alone. Built on this value leadership, we expect our first label expansion to be adult growth hormone deficiency. For this, we plan to submit and supplement BLA to the FDA in the second quarter of this year. In addition, we expect top-line data from our Phase 2 trial internal syndrome in the fourth quarter of 2024. Now, turning to Transcon PTAs. Last week, we initiated the launch of Transcon PTAs in Europe direct and international market. Market as your VPATs. with full commercial availability in Germany and Austria at an initial list price of 105,000 euros per patient per year. An estimated 70,000 adult patients in Germany are living with chronic hyperparathyroidism, and our initial longs will target 22,000 of those. EURAPAD's EUROPE Direct Launch is off to a great start, with our first prescription and orders proceed at the first day of launch, demonstrating the serenity of the disease and the unmet medical need. We are continuing to expand our EU Direct infrastructure and expect availability of EURAPAD across Europe by the end of 2025. In parallel, to meet the needs of the patients, we plan to provide commercial reimbursed product to early access routes such as main patient programs. The US PDUVA date for Transcon PTH is May 14. If approved, we plan to launch our UR-PAT as quickly as possible thereafter to our established US commercial infrastructure. Moving now to Transcon CMP. Our clinic-level program is designed to support approval of Transcon-CMP as a treatment for patients of all ages with achondroplasia. Transcon-CMP dose at 100 micrograms per kilo per week has demonstrated superiority compared to placebo in two 52-week trials. In the open-label extension, we observed unexpected strong patient retention of 97% for now up to four years. As a result of this unexpected finding, we continue to review our own data and scientific literature. We believe now that acroendoplasia is both a growth disorder and a muscle disorder. As presented at the JPMorgan conference in January, we showed significant improvement in health and quality of life for children with aconthoplasia treated with Transcon-CMP compared to placebo. With this growing insight, we have designed our pivotal approach trial to evaluate meaningful benefits related to all aspects of aconthoplasia, including linear growth, and improvements in co-morbidity as measured by radiological endpoints, physical function, body composition, and quality of life. We expect to share top-line data from this trial, and we plan to submit an NDA in the fourth quarter of this year. In addition, we are pursuing additional treatment opportunities in aquantoplasia. Infant trial, age 0 to 2 years, first patient already had been enrolled. Combination trial with chytophthora, age 2 to 11. Week 26, top line, analyze growth velocity in the fourth quarter of 2024. Adult, plan to file an IMD or similar in the third quarter of this year. Switching now to oncology. Both Transcon IL-2 Beta Gamma and Transcon TR-78 Agonist had shown favorable safety profiles and single-agent clinical activity. We expect 2024 to be an extremely important year for oncology pipeline, with data readout in well-defined patient population in the fourth quarter. Also in the fourth quarter, we expect to have completed enrollment of our randomized phase two trial in neuroadjuvant head and neck cancer. Longer term, to maximize this potential reach and value of these oncology product candidate, we aspire to commercialize our oncology classes through partnership with companies with specialized oncology experience and capability in late-stage development and commercialization. Moving now to Ophthalmology, we recently announced the formation and launch of an independent company, ICONIS, to develop and commercialize Transcon Ophthalmology products globally. We have received an equity position in the new reform company, and we are eligible to receive development, regulatory, and sales milestones, plus single-digit royalties. In January, we introduced Vision 2030, our strategic roadmap to achieve blockbuster status for each of our three endocrinology rare disease products and expand our engine for future innovation. With the broad applicability of the Transcon technology platform, our goal, as laid out in Vision 2030, is to apply our algorithm for product innovation to establish opportunities in greater than US$5 billion, as we did with the NGVGF and GPL1 classes, where we believe we have designed the best in-class programs. We are pleased with the interest in our once-monthly GPL1 program and from our platform technologies we expect to continue to generate many more opportunities in the future. I will now turn the call over to Scott for financial review before we open for questions.
spk11: Again, we continue making significant progress toward our financial goal of becoming operating cash flow break even quarterly basis by the end of 2024. I will touch on some key points surrounding our financial results, but for further details, please refer to our Form 20F filed today. As we previously announced in early January, Skytrofa revenue for the fourth quarter of 2023 was €64.2 million, compared to €47 million reported in the third quarter and €17.1 million reported in the same period last year. 37% sequential growth in Skytropha revenue from Q3 to Q4 was again driven primarily by strong underlying demand in the U.S. Total revenue for the fourth quarter was 137.7 million euro, including the one-time $70 million Tejan upfront payment recognized as 63.7 million euro of license revenue. Turning to expenses. R&D costs in the quarter totaled 90.9 million Euro, down 18% sequentially from the third quarter of 2023, primarily driven by lower endocrinology rare disease related costs as trials and development complete, and lower oncology related costs. SG&A expenses were essentially flat at 64 million Euro compared to the third quarter of 2023, reflecting commercialization synergies and expense controls. Total operating expenses were €155 million for the fourth quarter, down 12% sequentially from the third quarter. Overall, our operating loss for the fourth quarter declined sequentially by 73% to €37 million from €134 million in the third quarter of 2023. We ended the fourth quarter with cash, cash equivalents, and marketable securities totaling €399 million. Last month, we announced the formation and launch, together with an investor syndicate of ICONIS, to develop, manufacture, and commercialize Transcon ophthalmology assets globally. As a result, we expect minimal, if any, P&L burden from ophthalmology in 2024. As a reference point for Q4 2023, external ophthalmology project costs totaled 6.4 million euros. As previously announced, for the full year 2024, based on current plans, we expect Skytropha revenue to be in the range of 320 to 340 million euro at average 2023 exchange rates. Total operating expenses, SG&A and R&D, together to be approximately 600 million euro, and we expect to be operating cash flow breakeven on a quarterly basis by the end of 2024. Let me now also provide a review of selected key program milestones. For TransCon Growth Hormone, we plan to submit an SBLA to FDA for adult growth hormone deficiency in the second quarter of 2024. and we expect to report top-line results from our Phase II Turner Syndrome trial in the fourth quarter of 2024. For Transcon PTH, in the U.S., our PDUFA date is May 14, 2024. If approved, we plan to launch it as Yorvapath as quickly as possible thereafter. Outside the U.S., we initiated the launch last month of Transcon PTH, marketed as Yorvapath, with full commercial availability in Germany and Austria. We plan to roll out Yoripath in our Europe direct and international markets segments throughout 2024 and 2025. For Transcon C&P, our clinical program evaluating Transcon C&P as a treatment for achondroplasia advances with top line results from the pivotal approach trial and an expected NDA submission for the treatment of children with achondroplasia. both in the fourth quarter of 2024. In addition, during the fourth quarter of 2024, we expect to report week 26 top-line data from the COACH trial in combination with Transcon Growth Hormone and submit an IND or similar for adults with achondroplasia. Within our oncology therapeutic area, we plan to provide a clinical update from indication-specific dose expansion cohorts in the IELTS Believe and Transcend IT trials during the fourth quarter. In addition, we expect to complete enrollment of our randomized phase two trial in neoadjuvant head and neck cancer. With that, operator, we are now ready to take questions.
spk14: Thank you. And at this time, we will conduct a question and answer session. As a reminder, to ask a question, you will need to press star 11 on your telephone. and wait for a name to be announced to withdraw your question, please press star one, one again. Please limit yourself to one question, one follow-up. Please sign by. We'll compile the Q&A roster. One moment for our first question. Our first question will come from the line of Jessica Fai from JPMorgan. Your line is open.
spk05: Hey, guys. Good afternoon. Thanks for taking my questions. I realize it's early days, but What can you share with us about the German PTH launch so far? And the second one is, are you seeing any external interest in your Transcon GLP-1 program? And can you remind us what the target profile you're looking to achieve is with that program? Thank you.
spk07: Thanks, Jess. Yes, as I've said before, this is one week into a launch. We know the unmet medical need is there for the patients. we know how we really are providing a treatment that really are providing a meaningful differentiation for the patient, both on short-term and really also helping in eliminating the long-term risk of the disease. And what we observed was that exactly as what we had hoped for, the physicians, the day one of the lungs made the prescription, got the material from our distributor already the first day. In the US, you need to go to a prescription and reimbursement and then typically to get the drug. In Germany, it's different. Immediately, you get a prescription. You basically have access to get the drug immediately at that time. So the lungs, we really look forward to provide much better update when we come to Q1, much better after Q2, and much, much better again after Q3. So we're really, really feeling that the lungs in Germany and Austria is the starting point for basically being positioned to be everywhere on the globe where there is patients, and we will reach them all over places. Related to how we're utilizing the Transcon technology, outside rare disease. Endocrinology, yes, we have our oncology, which are really moving forward, as we only have hope for. And we're really looking forward to 24, where we can give you the proof, really, to patient data, how they're differentiated. You saw our differentiation in ophthalmology we made. ICONIS has been our company. And we really feel that they're really in an optimal value, both to create value and really help the patient in this area. When we come to the third area that we have disclosed we are working on, we basically have the GPL1 class. We're going for the once monthly profile. And it's not only for obesity. It's also for metabolic diseases. And we feel really, really that we are getting the interest that such a product really deserves. But it's not a product, it's a platform, a platform that can be utilized basically for the class of DPL1s.
spk08: Thank you.
spk15: Thank you.
spk14: One moment for our next question. Our next question comes from Tazin Ahmad from Bank of America. Your line is open.
spk06: Hi, guys. Good afternoon. Thanks for taking my questions. I wanted to get your updated thoughts about competition from recently approved growth hormone therapies. In particular, what is your market data telling you about Novosagroya? How is that, if in any way, impacting your launch? And then, can you give us some data about what percent of adults currently take growth hormone replacement therapy? And on average, How long does a person stay on therapy? Thanks.
spk07: Thanks, Sydney. It's really a pleasure to take up again the competitive landscape in the growth hormone market. And when we launched, we had a competition, the short acting. It was all the different six daily growth hormones that we were competing against. We showed how we were clinical differentiated to this established standard of treatment, how we provided a better outcome. We built the market basic to a lot to medical exception because of the product strengths of Skytofa. Now we see two emergent therapies coming in in the long-acting spare, two products, one Sagroia coming in and we don't see really impacting us because we still best in class. We actually believe that these two products have never proven that can show the same outcome as a normal daily growth hormone treatment that you get in the US. So for us, the barrier for medical exception is just improving. So for me and attendees, We don't see that it's really changing anything about how we are progressing to continue and build us to a blockbuster potential just in the US. And we will continue to do it because of our product strengths. Related to the adult, the adult is different compared to the pediatric in many different ways. One of the things the majority of pediatric A typical will have a treatment for a limited year. It can be four, five, six, seven, eight, depending on the initiation of treatment. Where the adult is a chronic disease, the typical will be needing a treatment for the rest of your life. So I would call it a chronic disease when you are diagnosed and start the treatment. So there is a big difference related to that. We often talk about penetration degree. with growth hormone treatment. And what there is a huge difference between the pediatric and the adult is the pediatric, in my view, is penetrated up to 60, 70, 80%, where the adult potential is under 10%. And I think this is a big difference for basic your modeling, how really to see and how we can develop the adult growth hormone deficiency market. As you said, this is our first label expansion. We will build up so we basically have access to all places where it's possible to use growth hormone because all the patients deserve to have the best product.
spk14: Thank you. One moment for our next question. Our next question comes from the line of Lee Watsek from Cancer. Your line is open.
spk09: Hey, great. Thanks for taking our questions. Maybe just follow up on Transcom PTH's launch in Germany. Understanding it's early days, but what could be a good framework for us to think about the ramp speed in that market? Any clarity that you can give us around that power experience patients and how quickly you may be able to onboard them? And then in terms of the patients that are enrolled in the early access program in Germany, do you have any color there as well?
spk07: Thanks a lot for the question. I can be bullish now in saying that all patients in Germany that have hyperparathyroidism should be on treatment because I think that asserts and hormone replacement therapy as everyone that has type 1 diabetes. Would all of them come on treatment? Likely not. But what we want to do, we are dedicated to help the patient and building up to scientific clinical data, our dedication to really to develop this market to ensure as many as possible will be treated with Transcon PTOA. or Euro parts. And this is not only in Germany and Austria, the rest of Europe, the rest of the international market is basically also in the US. So when we see this here, I think there is a different element how we see the penetration will happen. We always see the benefit of physician really getting the benefit seen in their own eyes. That's to take patient, take them on treating typical two to five, really to see how the product function. The benefit with Transcon PTH, after a few weeks, they already see the benefit. So then there is some kind of logical thing, how to get patient in. how to ensure that its capacity in really from the different physician sites really to take the patients in. So when we come into Q2, Q3, I think we will start to see the trend coming in and you can follow our revenue development, mainly coming from where we have full reimbursement in Germany and Austria. And then you can see how we penetrate rest of your direct end of 24, 25, and how we basically are building up the international market in the later part of 24 and really starting to be really penetrated in a much higher level in 25 and 26. Thank you.
spk15: One moment for our next question. And our next question. One moment.
spk14: And our next question will come from the line over. David Lebowitz from Citi. Your line is open.
spk16: Thank you for taking my question. Given the emergence of GLP-1s and their effect on lean muscle mass, is there a potential role for growth hormone
spk15: due to the success of the GLP-1s?
spk07: It's a question that basically going back to the mode of action and the benefit you see with the GLP-1. I personally was a little bit skeptical about the entire GLP-1 class in obesity, but it's typical because I'm pretty conservative in many ways. But what I'm 100% sure now is going to be a huge, huge class. And we see the benefit coming out really on a lot of different treatment aspects. It's not only just losing weight. There's a lot of other treatment benefits with this class. The negative part is that you basically are in a position that you're losing a large amount of muscle mass. And specific, if you're not adhered to the treatment and make period of break, start up again, period of break, you can basically be in position that when you're not treating, you build up the fat tissue faster than the muscle weight. And I think it's a really, really interesting perspective that Basie could see growth hormone treatment because that is really where the benefit of growth hormone is that it really can build up the muscle mass in a much, much stronger mass in a very, very normalized manner. And I think you are right, and I would like to explore such a possibility. Thanks for taking my question.
spk15: Thank you. One moment for our next question.
spk14: And our next question will come from Joseph Schwartz from Learing Partners.
spk15: Your line is open. Joseph from Learing Partners, your line is open.
spk03: Hi there. Thanks very much. I was wondering if you could talk about the work you did to arrive at your estimate that 70,000 adults have chronic hypoparathyroidism in Germany. And who are the 20,000 patients that you're targeting there? And how does the target population of the United States compare to the total hypoparapopulation of the U.S., where we have some guidelines wondering if that influences the proportion of the market that you think is most addressable here in the United States? Thanks.
spk07: Thanks. I think I got all the questions into it. And when we compare the demographic between Germany and U.S. It consists of the basic, the two groups, the post-surgical, which are one group, and then you can see the more hemological, genetic, idiopathic background where you typically will see it arise most earlier in states of life. Germany potentially is a little bit unique with the last number of patients because, and I think it, potentially is built on the high level of intensive head and neck operation because of really, really strong force of diagnosis. And from that perspective is, yes, there is potentially more positive patients in Germany than you see in the US. For example, if you take a country like France, France will much more relate to what you will see of treatment way that you see in the US. So when we see this different patient population, all of them will benefit of the Transcon PTH treatment of neuropaths. It's not like one group or the other one are less beneficial in the treatment on it. So all of them deserve to have a life with Transcon PTH. Going back to what you say when we talk about the 22,000 patients, and it's not because we see subpopulations that see more benefit or less benefit from that. When we talk about the 22,000 patients, which we try to address first, is the patient that from a pharmacoeconomic perspective have a really, really hard time on the society. They can potentially come to the state of real impairment. They can come to a state of visited hospitalization a lot. They come to a state where they cannot function because of cognitive effect. They cannot get a normal life. So out from that perspective, it's not really a treatment benefit. It's just more that the burden for this society, and this is where we always need to be, We need to benefit the patient, the society, and everyone as all stakeholders. And this is the burden for society as much, much higher for these 22,000 patients.
spk03: Very helpful. Thank you.
spk14: Thank you. One moment for our next question. And our next question comes from Derek Archilla from Wells Fargo. Your line is open.
spk13: Hey, good afternoon, and thanks for taking the questions. Just two from us. I just wanted to clarify, you know, is Gytrofa a $1 billion product in the U.S. just on pediatrics alone, or does that assume contribution from adult GHG as well? And then just in terms of the cost structure of the endo business, you know, is this $600 million cost-based now? How should we be thinking about it for the future? You know, and just your thoughts about profitability of that business. Thanks. Thanks.
spk07: Thanks. I will take the first question, and then Scott is really happy today now because he actually has an opportunity to come to some of the financial numbers he's running, so dedicated all the time. When we look on the potential market, yes, you can see we will have adult growth hormone deficiency. We will pursue some of the other indication, too. But we also need to say that we are liberating a normal somatopoeic molecule which are well-known entity for all different indication that seen the benefit on growth hormone. And if you go to the market of the daily growth hormone and see what is the dynamic there, the dynamic is that none of the daily growth hormones, basic, because they're all somatopine too, All of them, basic, are in a position. None of them have all the indication where they're actually being reimbursed or used today because none have really taken the effort to go through all the different indication. So it could be potential the same thing will be applied also on the long-acting products that are providing the same somatopene molecule. Scott, will you talk a little bit about expenses and numbers? In just 10 minutes.
spk11: How much time do we have? Thanks, Derek. So it's good to get a finance question. So as you saw here in the Q4, we're realizing now the benefits of the streamlined operational changes and processes that Ian has talked about, with OpEx going down to about 155 total. And then, of course, next year you'll see more come off as a result of basically no P&L burden from ophthalmology. I think you should look at this as a product of the way that we've developed our portfolio and how efficient it can be. I think if you look at our costs for TransCon Growth Hormone and realize the stage of that product and how mature it is, the cost should be dropping pretty substantially in the coming years. And overall, we're going to be pretty efficient in R&D. So I would expect more shift from 60-40 R&D SG&A in 23 to maybe 50-50 or so in 24. Longer run will be efficient in R&D. And as we expand globally, I would expect some increase in SG&A as we add people in 30, 40, 50 countries. But it should be pretty minimal. And as you saw, going from Q3 to Q4, our SG&A was basically flat, even if we launched
spk07: An additional product and got ready to launch a second product in Germany Thank you At one comment what you see the result of something was basic element that happened in 23 You don't suddenly change the company. You don't suddenly streamline the company. It takes a lot of effort it took us started in beginning on 23 and really find out how can we build a leaner Ascentis, more productive, really focus on really optimizing processes, administration, and everything like that. And we worked very, very, very successful to that. It was a hard task, not always pleasant, but we're there now. We are where we want to be now. We're still optimizing our processes because we're scaling for potentially having 5,000 to have 50,000 processes. And you cannot use the same PV system that you use for 5,000 to 50,000. So this is why we really are still optimizing processes, changing the company, because we cannot be a leading biopharma company at the same way we operated as an large biotech company where we basically focus on clinical development and fuel commercialization. So we are still transforming. We're still changing our processes. And that is what we need to continue to be truly competitive in this landscape.
spk14: Thank you. One moment for our next question. Our next question comes from Kelly Shi from Jefferies. Your line is open.
spk01: Thank you for taking my questions. Maybe switch topic to Trescon CMP. You have talked about the improvement muscle function, and other companies will feel like talk about more proportionality, like upper to lower body ratio. I'm just curious, have you also looked at proportionality as well? And also, when it comes to the combination with Skytrofa, What would be the most differentiated clinical benefit compared to TresCons MP alone? Thank you.
spk07: Thanks. I will start from the back because if you have a child born with achondroplasia being started treatment as a newborn, our belief is that your basic will nearly have normal growth. in this period of time where you have growth related to linear growth. You will not really address and stop or can stop to address the comorbidities because they will exist the rest of their life. And this is when we see on the integrated aspect of agonoplasia, we see the linear growth as one part of the disease and we see the other impact of the hyperactive pathway have on other aspects, like, for example, muscle weakness and other aspects, which are really from a mode of action is different compared to what you see on the Contoplast that really are limited in the way they get linear growth. So when we think about how we are positioning our combination trial is that What everyone, as I have seen, of data for the impressive amount of clinical data that's come out from biomarine related to psoriasis, all of that showing that you normalize growth for the vast majority of the patients. But if you have a child that got born and first started treatment of eight years, they still have a lack of growth. So when we saw All the data that has been generated by daily growth hormone. More than 30-40 publications. Really intensive trials with 40-50 children. And we see that they basically can induce catch-up growth as you can do in growth hormone deficiency. Because that is what we do in growth hormone deficiency. The mode of growth hormone is that you can reset the growth to be normal as a final height if you are on a good adherent treatment. But you can only do that in also growth hormone deficiency if you have a catch-up growth, meaning that you grow more in the first two, three years than you basically will see of a normal growth. That is why when you think about a child with growth hormone deficiency, not have been treated for eight, nine years, starting potential treatment, and then suddenly hit the parent's height as expected. This is because of catch-up growth. And you see the same thing in acromioplasty if you start to give them growth hormone. I see growth effect up to seven, eight centimeters. Not as much as you see in growth hormone deficiency, and it's likely because they still have a block down on the counter place because they also need CMP. So when we think about the perspective of combination trials, this is the children that not got treated, and this is basically the majority of children today. that is not gut-treated from the gut newborn and really need some cast-out growth really to get a burst in the growth. And this is why we are running this trial. My expectation, and now I can be trying to do something that is very, very dangerous, but I believe you can potentially grow them up to eight, nine centimeters just by combination treatment because you get the cast-out growth for the first one, two years. And this is the rationale really for us to go out and make that treatment regime, because we do the same as Rosotide, we normalize growth. So, proportionality. Yes, we see the same positive trend in proportionality that we have seen expected to see and we will see more and more when we see more data from longer part of treatment because it takes longer time to see really to see the right benefit on this or change of this proportionality but we definitely are seeing the same positive development thank you thank you one moment for next question
spk14: Our next question will come from Paul Choi from Goldman Sachs. Your line is open.
spk10: Hi. Good afternoon, and thanks for taking our questions. I also want to ask on TransConCMP, for your phase three approach study, can you maybe just share what metrics or details you'll provide with the top line results, and what is your medical conference presentation plans for that data? And then second, on the regulatory strategy, Can you maybe just help us understand the urgency to file the NDA in the same quarter, just kind of what the reasoning behind that is? Are you just potentially concerned about a full approval for Biomarin's Voxogo and just maybe some color there would be helpful? Thank you very much.
spk07: It's pretty simple about the filing. A sentence approach is to do it as fast as possible in the highest quality. And if anyone else is working in the company and not living up to this aspect, they should leave. So if I can get someone to do the filing in the same quarter we get the data, we should do it. When we come to the phase three data, yes, we have a primary endpoint because that got established as a traditional way of a primary endpoint is that really and rational endpoint for acroendoplasia? I have my doubt. I think it's more a surrogate marker, analyze high velocity. A treatment is to change the comorbidity of the disease. So the primary endpoint for me is like a surrogate marker as truncal fat is for adult growth hormone deficiency. This is not really the key treatment objective in adult growth hormone deficiency. You see other aspect of the treatment that's more important. but it's an established endpoint. So when we go to the secondary endpoint, and I said it's really very, very, very simple in my text, we're going in and look on the effect on comorbidities. And we do it on a lot of different angles. And we want to be sure that we're covering them in the best possible way. And the best possible way we can do it is to look on the co-morbidities basic that is developed into what we can measure in different physical function, daily function. We do a lot of radiology because we know the texture of the child needs to be perfect, so we are 100% sure. We have developed different health-related outcome measuring And I believe that taking all this aspect into it, we will find benefit that never had been seen. And I hope it will both come for quality of life, body composition, physical function, and also the radiological endpoint that basic providing different way of how then improve the texture.
spk10: And at the medical meeting,
spk07: I think we are not driven by medical meetings. Some people are saying that they lay data out because of medical meetings. I think when you have material data, you should come out so everyone can see the data. We are not waiting with disclosing data just because of medical meetings.
spk14: Thank you. One moment for our next question. Our next question comes from Andreas Argerides from Wedbush. Your line is open.
spk04: Hey, guys. Congrats on all the progress, and thanks for taking our questions. Just a couple here from us. When you think about CMP, can you give us a little bit of your thoughts around the rationale to expand into adults with hypochondrioplasia instead of going into hypochondrioplasia like the other companies in this space? Looking at Voxogo and the growth that they've seen primarily driven by ex-US, maybe you can give us a sense of, you know, why it's off to a slower start in the US. And is it driven by the fact that they have been focusing on height versus other quality of life measures? And then just one last one on the oncology programs. Don't find a partner to bring these programs. So would you consider spending them off like you did with the ophthalmology programs? Thanks.
spk07: OK, let me start on the last one because it's very easy. We are here to do the optimal for the patient to get our product being penetrated to as many as possible patients. And we believe that we are in a position when we have seen all the data that will come out end of this year. We already have seen the interest in our programs now. We feel pretty confident they would do the optimal where we see the fastest, best way to come out to the patient and we create most value for Ascendi shareholders. That will be the driver from our decision. Then you had two comments to C&P. The first one was, are not hypochondriplasia. Why not hypochondriplasia? That is a pretty good, interesting perspective for us because it's something we are discussing a lot. Because hypochondriplasia is a mild form for achondroplasia. And I think we will not move over to hypochondriplasia before we really have provided the optimal treatment, the best treatment that really addresses co-morbidity in acromioplasia. Why jump over to something where you basically have not provided a treatment option, but potentially only providing some few linear growth? And the linear growth, you could get that for the last 20 years just by giving daily growth hormone. So I don't see that it's really a treatment benefit that is just providing linear growth. What we're trying to address is basically the comorbidity of the disease. And we will continue to do that until we find the best possible solution to give them a meaningful improvement in quality of life. And the other thing is that I think these two things are related to each other. I don't believe being short is a disease, even though I'm pretty tall. So I actually don't believe being short is a disease. And I actually think you should never consider short to be a disease. I believe that taking just a linear growth in some way is more appealing in some countries than others. And definitely in the US, it's not a disease to be shown.
spk15: Thank you. One moment for our next question. And our next question will come from the line of Jeroen Werber from TD Callen.
spk14: Your line is open.
spk00: Hey, guys. This is Joyce on for your own. Thanks for taking our question. Just maybe hoping to get your thoughts on the emerging competitive landscape for Hypopara. You're clearly ahead to market, but there's another one, Stairway PTH Analog in Phase 3, and then there are a couple of others in the pipeline behind it, some looking to develop oral candidates or once-weekly candidates. So just any thoughts or comments there would be great. Thank you.
spk07: Thanks a lot. When I took a look from an endocrinologist's perspective and look on the biological system in the body, the complexity of that, how really to provide a treatment of a hormone replacement therapy in a situation where the biology has been extremely, extremely multiple organs effect, having the right dose, not too hyper, not too hypo. And see what we did with Skytropha. We provided the same molecule that really are being produced by the endogenous gland. When I also go to hypopara, this patient needs to have a physiological level of PTH, then providing the same mode of action. I actually went to a conference and heard some of the questions for physicians on some of the other competitive products. And one of the key questions that came up, we know PTH has effect on so many, many different organs. When you change the entire mode of action How can you really prove and believe you can create the same normal physiological system that you will expect to get with a normal endogenous PTH? Here's how we designed Transcon PTH to provide a normal same as endogenous PTH with exactly the same part that gives the mode of action in the physiological levels. And I don't think any treatment that changing that will provide the same holistic benefit on all aspects of hyperparalysis for any patient.
spk00: Thank you.
spk15: Thank you. One moment for our next question.
spk14: And our next question comes from Leland Gershow from Oppenheimer. Your line is open.
spk02: Thanks for taking our questions. Two from us, one each on Skytropha and Transcon PTH. On Skytropha, again, you mentioned in past commentary about the decreasing investment among the daily growth hormone companies and products. I wanted to just hear any updated color you may be able to share with respect to the marketplace in terms of the dailies, if that's a continuing trend that you're seeing. With respect to Transcon PTH, as we anticipate approval and then launch third quarter of this year, if you could remind us the number of patients you have available in the states between both those who may be in the open label studies as well as those who are in the EAP. Thank you. Yeah.
spk07: You're right. We have discussed a lot what we call the long-term or many years perspective of the consolidation in the growth hormone market. And the trend is happening exactly as expected. Specific with the entrance of the two or the long acting, the basic, the daily growth hormone is starting to disappear more and more because These two companies that are providing them, the key element where we have seen them taking market is basically taking their own product, their own product on the daily growth hormone class and then transform it over to the long-acting where we take it from everywhere because our improved treatment outcome. So I see our market share being basically the same as we some way predicted from three, four years. What is happening is that the two oils are long-acting, but just take part of the daily growth hormone. We never thought we'd actually have access to. And so our market penetration, our market share, our market value of Skytropha is basically unaffected to what we have seen. Transcon PTH and the US specific. The question you are providing me is between because our both phase two and phase three which have really high retention and continue to have extremely high retention now on fourth or fifth years. I cannot remember now more. The problem is that I cannot really remember how many patients that is in Europe and U.S. between the different trials. So therefore, my numbers are a little bit weak in that. I would think it's about 40, 60 or something like that. But I am not 100% sure about that. So please, Scott can give you the number when he's finished here and send it to you.
spk02: Great. Thank you very much.
spk14: Thank you. That's all the time we have for question Q&A today. Thank you for your participation in today's conference. This does conclude the program. You may now disconnect. Everyone, have a great day.
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