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spk14: Jay, and thank you for standing by. Welcome to the Ascendance Farmer Full Year 2022 Financial Results Conference Call. At this time, all participants are on 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 11 on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question, press star 11 again. Please be advised that today's conference call is being recorded. I would like to turn the call over to now your speaker for today, Tim Lee, Senior Director of Investor Relations. Please go ahead. The floor is yours.
spk11: Thank you, Operator, and thank you, everyone, for joining our full year 2022 Financial Results Conference Call. I'm Tim Lee, Senior Director of Investor Relations at Ascendance Pharma. Joining me on the call today is Jen Mickelson, President and Chief Executive Officer, Scott Smith, Executive Vice President and Chief Financial Officer, Dr. Stina 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 statements may include but are not limited to our U.S. commercialization and continued development of Skytrofo for the U.S. market, the commercialization of Transcon HGH for the EU market, statements regarding the expected timing of approval and launch of Transcon PTH in the U.S. market this year, statements regarding the expected timing of approval of Transcon PTH in Europe, statements regarding the potential size of the Transcon PTH, the market size for Transcon PTH, our progress on our pipeline candidates and our expectations with respect to their continued progress, statements regarding our strategic plans, our goals regarding our clinical pipeline, including the timing of clinical results, statements regarding our pipeline product candidates, statements regarding our plan regulatory filings, our expansion into new therapeutic areas, and statements regarding the ability to create a sustainable leading global biopharma company. These statements are based on information that is available to us today. Actual results and events could differ materially from those in the forward-looking statements, and we may not be able to achieve our goals, carry out our plans, our intentions, our expectations or projections disclosed in our forward-looking statements, and you should not place undue reliance on these statements. Our forward-looking statements do not reflect the potential impact of any licensing agreements, acquisitions, mergers, dispositions, joint ventures, or investments that we may enter into or terminate. We assume no obligation to update these statements as circumstances change, except as required by law. For additional information concerning the factors that could cause actual results to differ materially, please see our forward-looking statement section in today's press release and the risk factor sections of our most annual report on Form 20F, which is being filed today, February 16, 2023. Transcon Human Growth Hormone, or Transcon HGH, is approved by the FDA in the U.S. under the brand name Skytropha for the treatment of pediatric patients one year or older weighing at least 11.5 kilograms and have growth failure due to inadequate secretion of endogenous growth hormone. In addition, the European Commission has granted a marketing authorization for Skytropha to Ascendus Pharma developed under the name Transcon HGH It's a once-weekly subcutaneous injection for the treatment of children and adolescents aged 3 to 18 for growth failure due to insufficient secretion of endogenous growth hormone. In general, we refer to this product as Transcon growth hormone unless we're referring to the product in the context of particular jurisdictions such as the United States or the European Union. Otherwise, please note that our product candidates are investigational and are not approved for commercial use. As investigational products, the safety and effectiveness of the product candidates have not been reviewed or approved by any regulatory agency. None of the statements made on the conference call regarding our product candidates shall be viewed as promotional. On the call today, we'll discuss our full year 2022 financial results. We'll provide further business updates. Following some prepared remarks, we'll then open up the call for questions. I'll now turn the call over to Jens Mikkelsen, President and Chief Executive Officer. Jens?
spk10: Thank you so much, Tim. Ascend is built on the unique Transcon technology platform, which enables development of highly differentiated product candidates across multiple therapeutic areas. Combining the Transcon technology with our algorithm for product innovation has enabled us to create and develop product candidates with a higher likelihood of success than seen with conventional drug development. One of our key product selection criteria is to fulfill best-in-class potential on each of the four key pillars of drug development. efficacy, totability, and convenience. In addition, each product candidate must have the potential to achieve $1 billion or greater revenue in a single therapeutic indication. With this approach and driven by our values of patience, science, and passion, we have demonstrated our ability to continuously build out a robust pipeline, while taking product candidates from concept through approval and launch. With expected regulatory approvals of a new product or additional indication every one to two years, we are fulfilling our vision 3x3 goal of building a sustainable, profitable, leading biopharma company and creating long-term value for all stakeholders. This past year, we have advanced our pipeline as planned, entering 2023 with an April 30 PDUFA date and expected US launch of Transcom PTH for adult patients with hyperparathyroidism by the end of Q2. with an expected European Commission decision during Q4 as well. Transcon PTA is our second endocrinology rare disease product opportunity, representing a potential global opportunity greater than $5 billion. Turning to Transcon CMP, last November, we reported 12-month data from our first from the first ever randomized double-blinded placebo-controlled phase 2 trial in children diagnosed with achondroplasia. These results give me confidence that this third endocrinology rare disease product candidate may have its first approval by 2025 as target in our vision 3x3. Another component of Vision 3x3 is label and geographic expansion. We continue to build the value of our existing programs through additional clinical studies for label expansion and global commercial needs. Starting with our newly expanded European organization, which is preparing for launch of Skytropha in Germany this year, and if approved, Transcon PTA next year. With this great momentum across our pipeline, I would like to review additional details from our major programs. Turning to growth hormone. During the fourth quarter of this year, we plan to report top-line results from our global Phase III foresight trial in adult growth hormone deficiency. Our potential second indication for Transcon growth hormone. Adult growth hormone deficiency is a serious endocrine rare disease characteristic by abnormal body composition, dyslipidemia, insulin resistance, and impaired quality of life. Analysis has shown these consequences of adult growth hormone deficiency result in mean analyzed health care costs more than four times that of a non-growth hormone deficient population. Because Transcon growth hormone is the only once-weekly growth hormone product releasing unmodified somatropine, we expect it to be the first adult growth hormone treatment to meet or exceed the safety, efficacy, and solubility of daily growth hormone. Meanwhile, in the US, Skytropha is experienced the commercial success it deserved because of its unique product strengths. As we pre-announced during J.P. Morgan, fourth quarter 2022, U.S. Skytropha revenue growth to 17.1 million euro, providing a strong fundamental growth in 2023 and after. With our progress towards label expansion and plan commercial launch in market outside the US. We believe we can track, we are on track to build SCARTOFA into the leading growth hormone product in value by increasing the total market size. As we have predicted, we are seeing the consolidation of the data growth hormone market as other manufacturers begin to exit the US market. Turning to Transcon PTH, excitement continues to build among stakeholders around this potential treatment for adult patients with hyperpair ahead of the upcoming PDUFA date of April 30. Our expanded teams are hired, trained, and working to deepen physician and payer awareness of this serious health and quality of life issue that hyperparal causes. We have already made more than 2,000 calls to physicians related to disease awareness, and we are encouraged by their interest in learning more about the multi-organ impact of this disease and its negative effect on patient quality of life. Our commercial team, medical affairs, field reimbursement and manufacturing teams are ready to launch Transcon PTH in the US market as soon as possible after approval. Importantly, we are launching Transcon PTH, our second endocrinology rare disease product with the same commercial infrastructure that has proven its success with Chytropha. Coming back to CMP. As we did with Transcon Growth Termon and Transcon PTA, we ran a robust phase 2 trial to confirm Transcon CMP's target profile on all four key pillars, safety, efficacy, tolerability, and convenience, and de-risk it at the phase 2 level. This can only be done with a robust randomized placebo-controlled trial that will mimic the pivotal trial. We saw clear success in the complex trial with Transcon-CMP, demonstrating superiority over placebo at the 12-month primary endpoint in children aged 2 to 10. In addition, we saw clear dose response. 57 patients who started this trial remain in the OMAL label extension today. To extend and confirm this result, including positive treatment effect observed on acondoplasia-related comorbidities, we are running our Phase 2b approach trial. As investigators are aware of the Phase 2 results, we experienced very high interest in our approach trial. And we expect to complete target enrollment of around 80 patients in the next quarter. During our upcoming end of phase two meeting with FDA, we expect to collaborate on how to best achieve a broad treatment labeling rather than a linear labeling alone. Shifting to oncology. We are progressing with the developing of our two novel immuno-oncology programs, Transcon TLR78 Agonist and Transcon IL-2 Basagamma. With these two clinical programs, we are positioned this year to start evaluation of clinical efficacy in seven specific tumor types, nine different indications with four different combination therapies. including by combining our two Transcon oncology product candidates with each other. Clinical proof of concept phase 2 top line results are expected starting in 2024. In addition, this year we will initiate the randomized phase 2 trial BELIEVE201 using Transcon IL-2 beta gamma and TLR7 agonist combination therapy in head and neck cancer. As we successfully demonstrated with our endocrinology programs, we are building a solid phase two clinical proof of concept for our oncology products in multiple tumor types in the next one or two years. As you can see, we have and will always focus on achieving best in class product profile to benefit patients on the four key pillars of safety, efficacy, suitability and convenience. Errors in which we will not compromise. This development approach, including extremely robust clinical trial design, has positioned incentives to potentially launch a new product or indication every one to two years, building sustainability, long-term value for all stakeholders. Each successful clinical trial further confirms the power of the ISKCON technology platform and our product innovation algorithm and increases our confidence and likelihood of success for future product candidates. With an expanding pipeline and commercial successes, Ascentis remains on track to meet or exceed our goals outlined in our vision 3xT. I will now turn the call over to Scott
spk09: or a financial review before we open up for questions.
spk01: To follow on Yen's comments, we are excited to see the realization of Vision 3x3 with a continued flow of new products and additional indications every one to two years. For example, as Yen noted, we expect to launch Transcon PTH in the US and Skytropha in Germany this year, followed by the first European country launch of Transcon PTH in early 2024. With results for Skytropha in adult growth hormone deficiency and Transcon C and P in achondroplasia on the horizon, we expect this cadence of approvals and launches to continue beyond 2024. In this way, we are creating sustainable long-term value for Ascendus and our stakeholders through our proven R&D development capabilities. I will quickly touch on a few points. For further details on our full year 2022 financial results, please refer to our Form 20F, which is being filed today. As we previously announced in early January, Skytrofa U.S. revenue for the fourth quarter of 2022 grew to 17.1 million euro. These results exceeded the algorithm that Yen laid out last May, which projected 16 million euro. For the full year 2022, total revenue was 51.2 million euro, including Skytrofa revenue of 35.7 million euro, as well as license, clinical supply, and services provided to third parties, primarily Visa and pharmaceuticals. With profitable growth of Skytropha, our overall operating loss grew about 2% sequentially to 147.4 million euro for the fourth quarter from 144.5 million euro in the third quarter of 2022. Finally, we ended 2022 with cash, cash equivalents, and marketable securities totaling 743 million euro. Looking forward, As Yen described at the JPMorgan conference, annualizing fourth quarter Skytropha revenue of 17.1 million euro provides a foundation for 2023. In addition, we expect to add at least as many reimbursed patients this year as we did in 2022, which would provide even greater growth. As a result, at this time, we believe we are on track to exceed the current Ascendus compiled consensus estimate for 2023 Skytropha revenue of 96 million euro. Switching to Transcon PTH, our PDUFA date is April 30th this year. If approved, we expect to begin shipping product by the end of the second quarter. A quick reminder on selected key 2023 corporate milestones. For Transcon Growth Hormone, as mentioned, we plan to launch Skytropha in Europe, starting with Germany in Q3. And we also expect to report top-line data from the global Phase III foresight trial in adult growth hormone deficiency, our second indication, in Q4. Transcon PTH, we are planning for FDA approval by the PDUFA date of April 3-0 and launch in the U.S. by the end of Q2. And we expect the European Commission decision in Q4. For TRANSCON-CNP, we are on track to complete enrollment of the Phase IIb approach trial in achondroplasia in Q2. Within our oncology therapeutic area, we expect to report top-line results and declare the recommended Phase II dose from monotherapy dose escalation cohorts for TRANSCON-IL-2 beta gamma later this quarter, and to declare the recommended Phase II dose from TRANSCON-IL-2 beta gamma combo therapy with checkpoint inhibitor in Q3. Finally, as you see with our reporting today, continued optimization of finance systems and processes have enabled us to accelerate our year-end reporting. With that, operator, we are now ready to take questions.
spk14: Thank you. Our first question for today will be coming from Faye of JP Morgan. One moment, please, while I open your lines.
spk16: And thanks for the comments on how to think about Skytropa sales this year. Can you comment on your comfort level with consensus estimates for Transcon PTH this year? And actually, while we're at it, where is that consensus figure based on your latest compilation of the analyst numbers?
spk10: Yes, are you reflecting into Skytropa or Transcon PTH?
spk16: For PTH.
spk10: I actually have not looked at the consensus number for PTAs. I don't think we have collected that information, so I don't think we can really address that. We can mainly address the places where we feel confident to give an algorithm that can reflect our expectation, as we have done from Skytropha.
spk16: Okay, I'm going to ask something else then. Can you tell us how many cumulative new patient prescriptions there were for Skytropha and As of your end, I think you were previously giving that quarter by quarter. And can you also tell us when we should look for the next update from the phase two extension for Transcon CNP?
spk10: Let's just go back to how we basically are looking on the forecasting related to the revenue of Skytofa in the U.S. in 2023. What we have seen in here in 22 that month by month we have increased the number of new patients that got reimbursed. And we have continued to see this trend also in 2022. This is why it was important to look on the fourth quarter, because we're also seeing at the same time, we're seeing a really, really strong retention. When you start on Skype over, you stay on Skype over. So when we take the 17.5, million, and multiply that with four. Scott is calculating it's about 7.7 million euro. And we basically will expect an acceleration of the number of new patients because of a lot of good reason. And I can come back to that, Jess, if you want that. So we actually expect to see more patients per month of new reimbursed patients that we actually saw in 22. We're feeling really, really, really confident that we will exceed the consensus number that is out on the street today related to Skytropha. The element that BASIC are providing our increase in number of monthly new reimbursed patients is that the physician is starting really to get the knowledge about the product strength of Skytropha. It's not something you experience in one month. You need to see six months, 12 months growth data. And this is what we're starting to see. So we're really seeing how we really have a highly differentiated product compared to daily growth hormone. The other point is that we see the consolidation of the daily growth hormone market that started for about three years ago, where we saw after our phase two data that the consolidation of the daily growth hormone market is really taking in now. So there is a major, major switch away from some of the six daily growth hormone player, which all had the same product. So this is why we feel very, very, very confident about how we really would grow Skytofa into the most valued product in the growth hormone market in the near future. Related to CMP, you had a question related to CMP. Perhaps you can specify exactly what you wanted to know there.
spk16: Well, I think in the past you had talked about the height velocity for a proportion of patients that had been on out to a certain time point, and I think we're going to maybe update that when all of the patients got out to that time point. Is there kind of plans to update that data, and when should we expect it?
spk10: Yes, we are planning to give you this data. We think it's extremely, extremely important to give you this data where we see the continuous effect of Ascentis Pharma's Transcon CMP because I'm still in this extremely struggling manner and this is what we really have got a lot out of analyzing all the data for our accomplished trial to find out why they're staying 100% on this treatment. And we're starting to get a much, much better understanding of that. And this is why we will now are discussing with regulatory agencies how we can have other secondary endpoints that really are reflecting how we are addressing really the comorbidities and not just linear growth. Linear growth is not really the biggest issue for this patient group. Basically, the comorbidity and other effects of disease. And this is where we believe Transcon CMP is a unique product because you have continuous exposure of the CMP molecule and therefore changing things that is not only related to linear growth.
spk14: Thank you. Our next question. will be coming from Ahmad of Bank of America. And as a reminder, please limit yourself to one question and one follow-up and get back in the queue if you have additional questions. Thank you.
spk15: Thank you for taking my question. As it relates to HPT, can you just give us an update, if you haven't already, on how many patients you've enrolled in the Early Access Program so far? And do you have a sense of how many patients will be enrolled in that program by the time of the producer and then following with the launch? Thanks.
spk10: I think that is an extreme. First of all, we are extremely pleased with how the program is progressing and our regulatory interaction. And also that we got the approval to start an EIP program. which basically can give the opportunity to get patients under the treatment before we get the expected approval. We are executing on that, really starting the entire system, and we will explain when we come to the approval process what is the number of patients we will have in this trial, and also how we continue with these patients.
spk15: Okay, maybe just to follow up then, would you expect that to be an early source of patients to convert to commercial?
spk10: I actually think we are addressing a key element in the ERP program. We are addressing the patient group that already were experienced with a PTH treatment regime, a short-acting PTH treatment. It could be , it could be , it could be or someone else, but that was exactly what we're addressing. The large population where we basically recruited patients to our Phase 2 program and our Phase 3 program, they're coming from what we call patients that never really have been exposed to PTH treatment. So I don't see that it's really, really a differentiation between these two patient groups. I think both patient groups have the same high unmet medical need. They have the same benefit of the PCA's treatment. So I don't see any kind of difference between these two groups related to the FAST data in our programs.
spk14: Thank you. Our next question will be coming from David Laborelis. City, your line is open.
spk06: Thank you so much for taking my question. As the PDUFA date is approaching, could you give us any insight into what the label might ultimately look like? I know that Natpara was considered an adjunct. You seek to be going more as a hormone replacement, and Natpara has the presence of a black box for osteosarcoma. but you didn't really have an osteosarcoma experience, so could that black box be removed? Just be curious to hear your thoughts.
spk10: Thanks, David. I think when we look on the biology and the product design of Transcon PTAs, we are providing a stable physiological level of PTA 24 hours, 7 days a week. any hyperphysiological concentration of PTH that can provide to the anabolic effect that in animal model have been associated with osteosarcoma, specific for rat model. And how from that perspective is that, and also why we got a waiver to make a carcinogenic trial. or animal trial, we will not expect and we have not seen any indication in our labeling discussion that we will be coming in the same, you can say, group of short-acting PDAs that basically got the same class labeling because we are providing a complete different product profile than the short-acting PDAs. So to our best knowledge today and in our discussions, we don't expect any REMS program or we don't expect any black box warning. So that was your first question. And you're quite right. How we designed Transcon PTAs was really to prove a product profile that was reflecting hormone replacement instead of an adjunct. There is why to be successful in the clinical trial, you need to stop 100%, 100% from active vitamin D, and you also need only to take calcium supplement that really are just reflecting a normal multivitamin from Costco. So we are really addressing a complete different product profile.
spk06: Thanks for taking my question.
spk14: Thank you. 1 moment while we prepare for the next question. And our next question is coming from the logic of cancer. Your line is open.
spk13: Hey, thank you for taking my questions. I guess for trends on. Just wondering if you can share some of the latest feedback that your sales team maybe received from, you know, payers and physicians. And then maybe talk about your latest thoughts on how you might approach pricing and market access.
spk09: Thank you.
spk10: When I think about the patients, when I think about the physicians, the measures have not been different for the latest Everyone recognizes that hypopara is a serious disease, and everyone understands the benefit of replacing a missing hormone with a physiological level 24 hours, 7 days a week. How it both addresses short-term symptoms like quality of life, urinary calcium, but also long-term risk. And what we're seeing is that the awareness of that is being building up much, much more about the awareness of the disease. And I believe this is where we come in with our education, first telling about the awareness of the disease, which are really a big part of what is happening today. with all our established infrastructure here in the US. And after an approval, we can go out and explain how we can benefit this kind of disease, both short-term and long-term. So I really, really, really feel that we are right. What we did with Skytrofa is the guidelines we do with all of our products. We develop best-in-class products addressing real unmet medical needs. And we take a premium responsible pricing situation, where we believe if you really develop a product that really address a real unmet medical need with a real product, that is enough for both the patient, the physician, the society, and the payer for us to share that cake. Because then everyone is winner. No one is looser. And this is where we want to be with each of our products. We need to have them so highly differentiated, addressing a real unmet medical need, and everyone believes it's a win for everyone. And we can see that with Transcompetech.
spk14: Great. Thank you. Thank you. One moment while we prepare for the next question. And our next question is coming from Paul Choi of Goldman. Your line is open.
spk03: Thank you. Good afternoon, and thanks for taking our questions. I want to ask, given that you're using the same infrastructure to market PTH in the U.S. that you're currently using for Skytrofa, I guess, are there any learnings that you might share from the launch of Skytropha that you would think be applicable? Or what changes would you make, I guess, in terms of either your thoughts on approaching pair access and or contracting compared to Skytropha? And then I had a pipeline question as a follow-up.
spk10: Great. Let me start on that. We are utilizing the same infrastructure. sure we have dedicated sales force we will have dedicated people for the two different products but you know there is a huge difference to be the first product to go out and launch where you establish all the infrastructure IT systems all the different necessary teams that is necessary to really to launch a commercial product we have did risk it back now we're coming from a stage where we launching from an already successful established commercial infrastructure built from Joe and the other people in Princeton here in the US. So what we're doing is that we basically are placing what I call Transcon PTH into an infrastructure that already has proven its capability with a product, I believe, really addressing a huge unmet medical need where there's no alternative treatment. I think this is a fundament for a huge success.
spk03: Okay. Thank you. And then as a follow-up, just in terms of the pipeline, do you plan to publish the baseline patient characteristics for the 80 children that are being enrolled in the approach trial? And then could you also specify, in terms of your oncology program, the head and neck population that you're planning to pursue. Is it just HPV-positive, or is it post-PD-1 and post-urbitux? If you can maybe add a little color on that, that would be great. Thank you for taking our questions.
spk10: I think Stine will take number two, or you can take one, and I can take his name. Let me take number one. It's a very, very interesting question. Because I do not know if the question, where it's really are addressing. Because I actually think we have published all the demographic from our patient population and demographic that have been into the accomplished trial. The 57 that's still in it. So I actually am a little bit puzzling with that question because all data is out. Then you can say, hey, Why did you not come in with the analyzed height velocity pre-screening? Because it's totally irrelevant for the clinical efficacy of it. You cannot use an analyzed height velocity. They have been collected before they go into the trial because we have 40% of the children between 2 and 5, which you look and analyze. a complex child is nearly built with normal analyzed height velocity. And the first four years, you have a heavily deceleration of analyzed height velocity. So if you take an analyzed high velocity, just collect it up to 12 months before they go into a trial, it's not reflecting any meaningful value that go in and compare to the analyzed high velocity you compare in this patient group because they start on the AIDS already too. This is why you do what is obvious in drug development. You're making a placebo group. This is why you have a placebo group. And I think that is the key element to do. Look at our dosing from six to 100, and then you can take six nearly like also a placebo group. And then you can take the six or the placebo move them into the account to place a specific height and see if they're matching. They're matching 100%. But compare this is my basic scientific nonsense and only are misleading and have not reflecting any kind of solid scientific value in interpreting the data. Stine?
spk12: In oncology, we are evaluating for proof of concept efficacy in seven different tumor types. You're right, we do have one of our priority areas is head and neck cancer. We are evaluating in first or second line metastatic head and neck cancer as a dose expansion cohort single arm in the IO-BELIEVE study. And those patients will have had no more than one line of chemotherapy-containing regimen in the advanced metastatic setting. And the randomized phase two studies, relief IT 201 study, will be in the neoadjuvant setting. So these are patients in a non-metastatic setting. Before they get surgery, they will get systemic treatment before surgery. And we're looking at pathologic response as our primary endpoint to look for evidence of proof of concept efficacy.
spk09: Thanks, Dina.
spk14: One moment while we prepare for our next question. Our next question is coming from Derek Archer of Wells Fargo. Your line is open.
spk05: Great. Thanks, and thanks for taking the question. two really quick ones from us. Jan, I just wanted to confirm, I thought I caught you saying that you were in labeling discussions for TransCon PTH. So I just wanted to confirm that. And then also, I guess, when should we expect additional updates from Accomplish? Is that something we should see, you know, again, first half of this year or second half of this year? Thanks.
spk10: When you go through an approval process, I think For me, it's such a planned process. Four months before an expected approval date, this needs to happen. Three months before an expected approval, this needs to happen. Two months before that. And so if anyone somewhere has been to an approval process, and I think we have 10 weeks before the PDUCA date now, if you're not a start at a label discussion, the risk of not getting approval is high. So therefore, I believe this is a way of tracking how we are progressing to the approval process. Are we really on track of what everything needs to happen for the expected time? Is that not happening? I would be extremely worried and find out what is going on. And so, yes, we are in a label discussion because you should be that at least three months before an expected approval. And this is why I feel that I'm confident that I have not seen anything that not give me a belief that Transcon PJs is a product that is approved. I believe, and I cannot really remember all our corporate milestones now. Sorry for that because that's a little bit too many of them. But I believe that is in Q4, we will give you an update again related to the 57 patients that will come out from the accomplished trials. So it will be in the second half of this year.
spk05: Excellent.
spk10: Thank you very much.
spk14: Thank you. One moment while we prepare for the next question. And the next question is coming from Josh Romer of Evercore. Your line is open.
spk07: Good. Thanks so much for taking the questions. First on Skytropha, could you elaborate a little further on what you're seeing in terms of daily growth hormone options withdrawing from the market? I know there have been some reported shortages, but I didn't realize that reflected the outfall withdrawals. So who... Have you seen this drawing, and do you expect others to follow? And then how do you anticipate the impact of Novonordis potentially launching a once-a-week growth hormone option as well in the market later this year? Thanks.
spk10: Let me start first on the daily growth hormone, because I actually believe that it's a textbook that really, and if I had taken an MBA, potentially I would have done that in my study in my final year. Project, but it's really really what I call a textbook example Go up tomorrow or the day to go tomorrow market was the first I got biosimilar where send us and Tiva entered there with their bio equivalent version on it and And there somebody had six players in this daily market segment, all of them providing exactly the same entity, the same treatment. So you can change the back and forth between the product depending on rebate and other things like that. That was different in formulation, that was a little bit different in devices and other things like that. So what we saw for about three, four years when we came up with our phase two data, we saw already that some of the big player or many started some way to reconsider how do we really play it when that's coming and superior treatment into this segment and that will be, which will be highly differentiated compared to what we call the daily market segment. At that time, we already saw three of the companies basically removing their safe force. This is step one, as I call it. You remove safe force. The second one is that you go to the next stage. You remove the hub. Then you stop up manufacturing, which I think three or four of them have done it now. And then you basically are in a position where you are providing mainly patients that already are established on your product because you have no hub where you can change and take new patient in and you're providing them. What happened in setting up for the patient now that it looked like a noble notice went into a shortage of a multiple product, multiple presentation of their growth hormone. And because that consolidation of the data growth hormone has happened and really is in the final place, I believe none of the other one could take over. So you have to see a shortage of growth hormone treatment in the US. And sure, I need to accept that the benefit of that is a great thing for us because it's both accelerating at the same time where people really see the benefit get the clinical experience how we differentiate be having patient for one year or something on treatment so they really really really see this benefit and I think this is a great thing for sure for us and we really hopefully we can help as many many as patient to avoid that going into a shortest of the treatment effect
spk07: Very helpful, thank you.
spk10: Your second question, Josh?
spk07: Actually, I was going to ask whether, given your view of the differentiation of transcon-CMP, if you'd considered filing for breakthrough designation.
spk10: Yeah. First of all, Josh, you also have the question reflecting about the potential of the entrant of NovoNodisk long-acting product. When I look on the paradigm shift, I call it paradigm shift because it comes from the time where all the daily growth hormones were identical, the same treatment, the same mode of action. When you go over to the long-acting, all of them are providing complete different clinical profiles. They're the only ones that really match an improved version of the daily growth hormone where you get all the endocrine benefits both related to changing not only linear growth in the pediatric sentence but also the other interest associated endocrine benefit like body composition metabolic profile lipid muscle cognitive effect and everything that you see because we are the same unmodified somatopoeia molecule so this is why it's also important for us to look in our phase three in adult growth hormone deficiency because The two other potential long-acting, where we believe there's potentially one left now, but the OPCO Pfizer showed basic, not any improvement on body composition in the phase 3 trial. Novo Nordisk showed that it can only get the half of the thick basic to a daily growth hormone. And this is where we believe, with our unmodified somatopoeia, potentially we will be at least as good as daily growth hormones. So, we believe that our product profile is always so highly differentiated to both daily growth hormone and other long-acting growth hormone that we always will provide us the clinical benefit compared to all other treatment regimes.
spk14: Thank you. Our next question will be coming from Leland Gershel of Oppenheimer.
spk04: Two from me. I know you had responded earlier that you don't expect a REMS or a black box warning on the PTH label, but could you comment on any potential for monitoring requirements with patients on the product?
spk10: Thanks. Could you clarify your question, what you mean exactly?
spk04: Well, in other words, if patients need to be monitored for serum calcium, bone biomarkers, whatnot.
spk10: That is a question where you will say, will we provide a better stability for this patient group than they have in the current setup where the basic are being monitored for calcium really, really, really often. And I believe you will see it in different stages. I believe when you transition over from what we call the conventional part of therapy over to transcon PTH, I think there will be at least the same kind of monitoring because you want to be quite sure you've stabilized the patient in the right manner. When they are stable, which we see after one to two years on a PTH dose, where we see more and more stability coming in because the calcium metabolic system gets calcium hemostasis starting to be stabilized, I will potentially see from a patient perspective that you will potentially need less what we call monitoring of it. I think this is where I believe that elements like bone marker, it's not typical something you typically will analyze for any patient group in this way. What we have seen in our clinical study where we now patient up for over three, four years, you're basically seeing that we do normalization more and more and more of every parameter. And that includes both bone density and it's also including bone markers. So I will not expect that it will be part of a standard monitoring.
spk04: Okay. And second question for me is with respect to achondroplasia, Obviously, the primary endpoint, and for regulatory purposes, it's about type velocity, but as you had mentioned earlier, there are many other benefits that a replacement CMP could provide. Could you just sort of inform us as to which of the other benefits that may be not captured by primary endpoint type data but would be very important, seen as most important by the ACONDRA community?
spk10: Yeah, I think this is why we're developing Transcon CMP. We really are developing it to provide a treatment that sure is addressing linear growth, but we can combine it with the Skytover, and then I can, from a clinical concept, I will expect nearly you can decide what kind of linear growth you will have. But what we really want to ensure is that we are addressing the underlying comorbidities of the patients. And how we measure them is to have specific acondoplasia, specific co-morbidities. This is co-morbidities that is coming and being reported on high, high, high frequency from the acondoplasia from when you . And then we also are developing a patient-specific reported outcome measuring. where we're trying to capture all the benefit they're feeling, because they're really seeing a huge benefit. One of the things that is clear for us, we see a lot of other effects that are not just related to bone. We see muscle change. We see how they function better in the physical way to balance, do normal work. do normal operation and other things like that. And this is all this element we will try to capture. At the same time, we will capture the account of place, a specific element, like a number and other things like that.
spk04: Thanks. That's very helpful.
spk14: Thank you. One moment while we prepare for the next question. Our next question is coming from Andreas. Are we all right? From Wedbush, your line is open.
spk02: Hey, guys. Thanks for taking our question. So when you're thinking about, this is for PTH, when you're thinking about the opportunity, the launch and the opportunity in PTH, how are you thinking about it compared to the NatPara launch? And then maybe you can, you know, give us some insights on how NatPara coming off the market, you know, at the end of 23 is, is informing your expectations for the launch, and then have a follow-up. Thanks.
spk10: I'm not comparing Transcon PTA in any way to NetPower. It will have complete different labeling, have complete different clinical outcome, have complete different clinical benefit, so just complete different impact on quality of life. So I'm not using that as a benchmark. anything I'm not comparing that there was a product that came out with a lightning as an adjunct this is not what we are addressing there was a product that came out not seeing benefit on quality of life it was a product that came out not showing any benefit of 24-hour urinary calcium not really addressing the underlying disease so you can I don't use that at also any benchmark It's a complete cleaning list for me.
spk02: Okay, great. That's helpful. I guess maybe the opportunity that there is no approved product on the market. There were some patients, mostly in Europe as well, who were still on it or had access to it no longer. Are those going to be early adopters? Yeah, thanks.
spk10: Yeah, I think actually this is exactly as somebody addressed before. Yes, there is a patient that has been exposed to short-acting PTH treatment. They are used to daily injection and other elements like that. But where we see the huge benefit is independent of the background if they have been exposed to short-acting PTH or not. So I don't believe there is a less need for a patient to get on transplant PTH treatment if you come from the group that has been on PTH. short-acting PTHs or have never seen a short-acting PTH. The patients that have been on short-acting PTHs have perhaps a more common understanding of a daily injection, have more common understanding about what they need to do as procedure to get that happen. But I don't believe that is a big barrier for any of the groups.
spk02: Okay, great. And then just a quick one on Skytropha. And maybe you can elaborate, and I don't know if you covered this already. Sorry if you did, and I missed it. But if you can elaborate on the launch dynamics that are driving growth, are you seeing higher switch rates from daily growth hormone? And that's it for me. Thanks, guys.
spk10: We always see a higher switch rate from daily growth hormone, but we're also seeing an increased number of new patients coming on the treatment. But the overall number goes up everywhere. So it's not like one getting less. Both of them are improving up to a really, really new height all the time.
spk14: Thank you. One moment while we go to the next question. And our next question is coming from Yarin Warbur, excuse me, of Cowan. Your line is open.
spk08: Great. Jan, I have two interrelated questions on more coverage. With PTH coming soon, uh, there isn't really a competitor. Do you need to contract or will you contract with PBMs to get on formulary? Maybe kind of talk about your, your thoughts there. Obviously the discounting shouldn't be very high at that point. And then secondly, now that you'll have a second program, second product approved sort of within the endocrine bag, does it give you more leverage to negotiate better, uh, for a placement for a Skytropha? Thank you.
spk10: Yeah. Um, We never really comment about how our market assess strategy is and will be and how we are really addressing the reimbursement system in the US. But for your information, when we always look at other products, because you can do the same thing, I can give you name of products in the US. that BASIC are in a position to generate multiple billion in revenue in the US without BASIC being provided high rebate. And I think we will follow this pathway because we are providing such a benefit to the patient, the physician, and the society that we feel this is a way we will continue our market asset strategy. Compared to Skytober, we are actually pretty satisfied with our coverage. We believe the strength of the product, the differentiation, don't really lead us to provide into a highly rebated product. I think that is the strength of having a highly differentiated product.
spk14: Thank you. That's all the time that we have for today. Thank you for joining the conference call. You all have a good evening.
spk10: Thank you.
spk09: Thank you so much. See you.
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