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spk05: Good day and welcome to the Q1 2024 Ascendance Pharma Earnings Conference call. At this time, all participants are in listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question, please press star 1-1. We ask that you please limit yourselves to one question and a follow-up. As a reminder, this call may be recorded. I would now like to turn the call over to Tim Lee, Senior Director, Investor Relations, Ascendance Pharma. Please go ahead.
spk02: Thank you, operator, and thank you, everyone, for joining our first quarter 2024 financial results conference call. I'm Tim Lee, Senior Director of Investor Relations at Ascendus 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. Sina Singhal, Executive Vice President of Clinical Development Oncology, and Joe Kelly, U.S. General Manager. Before we begin, I'd like to remind you that this conference call will contain four linking 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 Skytropha for the US 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 TransCon 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, and our progress towards Vision 2030. These statements are based on information that is available to us today. Actual results may differ, could differ materially from those in our four linking statements, and you should not place undue reliance on these statements. We assume no obligation to up these statements as circumstantial change, except as required by law. For additional information concerning the factors that can cause actual results to differ materially, please see our forward linking statement section in today's press release and the risk factors section of our most recent annual report on Form 20F filed with the SEC on February 7, 2024. Transcon Growth Hormone, or Transcon HGH, is approved in the U.S. by FDA and the EU has received MAA authorization from the European Commission for the treatment of pediatric growth hormone deficiency. The European Commission and the United Kingdom Medicines and Healthcare Product Regulatory Agency have granted marking authorization for Transcon PTH as 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 first quarter 2024 financial results and we'll provide further business updates. Following some prepared remarks, we'll open the call up for questions. With our PDUFA date for Transcon PTH coming up in less than two weeks, we will not comment on our ongoing discussions with FDA, and we will not be taking any questions on this topic today. With that, let me turn it over again.
spk08: Thank you, Tim. Good afternoon, everyone.
spk30: Centis is applying its Transcon technology platform to build a leading, fully integrated biopharma company. focused on making a meaningful difference in patients' lives. Two approved Transcon followers and solid progress across programs, growing commercial presence, and strong partnerships. We believe Ascentis is on the path to sustainable growth and operating cash flow quite evenly on a quarterly basis by the end of 2024. Our long-term commitment in the last three, four years to build up profitable and robust supply chains and the decision we took in 2023 to streamline the company and made us a leaner and more efficient organization. You can see the progress towards operating cash flow per year later this year without compromising our development and commercialization process. We believe we are well positioned to successfully deliver on our strategic goals and close out our Vision 3x3 with regulatory approvals for three independent endocrinology rare disease products and continue building a pipeline in other therapeutic areas. Looking to Vision 2030, We believe each of our three rare disease endocrinology partners have the potential to achieve blockbuster status, while we also further expand our pipeline and transplant platform for future innovation. We have seen validation of our commercialization approach with the ongoing success of Skytrofe in the U.S., and are seeing it again with successful norms of Europass in Germany and Austria. In the US, the PDUFA date for transport PTAs is coming up in less than two weeks on May 14. If approved, we expect to be ready to launch in the US soon thereafter in the third quarter. Now, let me give an update on each of our programs. When we launched SCARTOFA in the US about two years ago, we had two goals. One, to make SCARTOFA the leading product in that. The second to drive U.S. growth hormone market to become a 3 billion market. Our strategy is working. Thousands of patients are now treated with Skytover, the value leader in the U.S. We have a reliable supply chain, ensuring that every patient can benefit from Skytover once they have approval from their insurance company. We estimate that Skytover penetration in the US pediatric growth hormone deficiency patient population grow to about 17 at the end of the first quarter. We are proud to observe Skytover extend its market value leadership as the only growth hormone product to grow in value in the first quarter of 24, based on reported results. And we believe Skytover will expand the US growth hormone with the potential to become a blockbuster in the US alone. Skytrover sales this quarter more than doubled compared to the first quarter of 2022, with a steady quarter to quarter increase in treated patients. We expect to continue this trend rest of this year. With these strong Skytrover results, we continue to expect the full year 2024 Skytrover revenue will be 140 million euros representing year-to-year growth of 80 to 90 percent. We expect Gartofa sales to continue to grow through further penetration in the pediatric growth hormone process. To further solidify growth, we are also pursuing our first labor expansion in adult growth hormone deficiency for which we plan to submit and supplement BLA to the FDA in the third 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 PTH. In Europe, EUROPATH was launched in Germany and Austria at the end of January with the early launch objective of building physician experience with EUROPATH. Initial physician feedback from EUROPATH has been positive. Just eight weeks in the launch, we estimate prescriptions have been written by 55 doctors, representing around 25% of the target prescribing base, with about 100 patients receiving commercial product. As physicians begin to get comfortable with the treatment benefit of UroPelt in the first patient, we expect physicians to take more and more of the patient on treatment. As we expand our geographic reach of Europe to our Europe direct and our international market sector, we are supporting named patient supply programs and plan to provide reimbursed product to meet the needs of patients. In the U.S., pre-launch preparations are underway, including the expansion of the U.S. field infrastructure while we are awaiting FDA's decision. Moving now to Transcon-CMP. Our value proposition with Transcon-CMP is simple. It's to establish a treatment for patients of all ages with acromioplasia. Our ambition is to address the significant comorbidities associated with air contemplation that impact health and quality of life, as well as linear growth. Later this year, in the third quarter, we plan to report top-line results for our pivotal approach trial, which is measuring not only linear growth, but also physical function, body composition, and quality of life parameters. We are also evaluating Transcon-CMP in newborns with air contemplation less than two years of age. We believe that treatment of air contemplation with Transcon-CMP as early as possible might mitigate associated comorbidities. In addition, to provide catch-up growth to children who did not receive early treatment, we are evaluating Transcon-CMP in combination with Transcon-Groptomol. We expect to enroll all patients in this trial this quarter and provide 26-week top-line data for this trial in the third quarter of this year. This is our integrated approach to address acromiopasia with multiple trials that are all building towards one goal to treat the disease. And we are optimistic that our highly differentiated product candidate will continue to show best-in-class potential across these multiple studies. Switching now to oncology. Both Transcon IL-2 beta-garmin and Transcon TLR-7-8 agonist has shown promising clinical activity as monotherapy and in combination treatment with PEMBO in late-stage patients whose disease has progressed after standard of care treatments. Next month at ASCO, we will report updated patient data from the ongoing Phase 1-2 trial, I believe, that has been reported last year at ESMO. We will present updated clinical data and new biomarker data that further differentiate our clinical program. We will also report promising early data from the ongoing dose expansion cohort of patients with melanoma who had progressed from checkpoint in Ibersus using the combination of transcon IL-2 beta gamma and transcon TLR agonists in these patients. Later this year, in the fourth quarter, we expect data readout in several well-defined patient populations from our Transcon IL-2 beta gamma and Transcon TLR-7A aggregate programs. Our achievements this quarter give me further confidence that all the elements are in place to fulfill our strategic goals to deliver three independent endocrinology rare disease block dog problems. and a strong pipeline in dark-cell telepathy areas such as oncology, ophthalmology, and metabolic diseases. There is much more to come. I will now turn it over to Scott.
spk03: Thank you. In Q1, we demonstrated significant financial progress for our goal of becoming operating cash flow breakeven on a 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 6K filed today. Skytrofa revenue for the first quarter of 2024 was 65 million Euro, compared to 31.6 million Euro reported in the first quarter of 2023, an increase of 106% year over year. This growth was driven by significant increase in demand volume, more than doubling compared to the prior year period, with a slight offset by a combination of slower channel bill coverage mix and a modest negative currency impact of 0.8 million euro. On a sequential basis, first quarter Skytropha revenue increased 1% compared to the fourth quarter of 2023. Strong mid-teens percent demand volume growth was offset primarily by seasonal channel inventory, copay resets, insurance reauthorizations, and a modest negative currency impact of 0.6 million euro. Q1 was in line with our high internal expectations and with seasonal headwinds behind and the steady pace of new patient ads expected to persist. We continue to expect full-year Skytropha revenue to be in the range of €320 million to €340 million at average 2023 exchange rates. Shifting to Transcon PTH, YorvaPath contributed for the first time this quarter with revenue of €1.5 million, representing two months of shipments. In Germany and Austria, we ship directly to retail pharmacies for patient pickup, and as a result, there is no channel inventory buildup compared to a typical U.S. launch like we saw with Skytropha, where specialty pharmacies hold channel inventory. Closing out the top line, total revenue for the first quarter was 95.9 million euro, including 24.8 million euro of non-cash license revenue recognized in relation to the formation of ICONIS, and €3 million of service revenue related to ICONIS, which is offset in operating expenses. Turning to expenses, R&D costs in the first quarter totaled €70.7 million compared to €106.1 million during the first quarter of 2023. The 33% decline was largely tied to lower external development costs for Transcon Growth Hormone, and TransCon PTH, including a reversal of prior period write-downs of pre-launch inventories, as well as oncology programs, partially offset by an increase in TransCon C&P costs. Sequentially, R&D costs declined 22%. SG&A expenses in the quarter totaled 66.8 million euro, essentially flat compared to 66.5 million euro during the first quarter of 2023. Higher employee costs, including the impact from commercial expansion, was partially offset by lower external prelaunch and administrative expenses. Sequentially, SG&A expenses increased 4%. Total operating expenses were 137.5 million euro for the first quarter, a 20% decrease compared to the 172.7 million euro during the first quarter of 2023. Sequentially, operating expenses declined 11%. Overall, our operating loss in the first quarter totaled 49.1 million euro compared to an operating loss of 143.7 million euro during the first quarter of 2023 as a result of increased revenue and lower operating expenses. Sequentially, operating loss increased 34%. Finance expense in the quarter was 77.2 million euro compared to 8.4 million euro expense in the fourth quarter of last year. This higher finance expense was largely driven by a non-cash derivative loss tied to our outstanding convertible notes. Quick comment on the balance sheet. As of March 31, 2024, due to amended IFRS rules, which came into effect on January 1, 2024, You'll notice our convertible notes with face value of 575 million US dollars are now reported as current liabilities, even though they do not mature until April 2028. It would not require cash settlement in case of conversion by holders. IFRS still requires the carrying value of the convertible notes and associated derivative liabilities to be presented separately within current liabilities, which together total 622 million euro. As per IFRS rules, comparative amounts have been reclassified to reflect the change in presentation. The applied amendments had no other impact on the financial statements. We ended the first quarter with cash and cash equivalents, totaling €320 million. And for the full year 2024, based on current plans, we expect Skytrofa revenue to be in the range of €340 million at average 2023 exchange rates. We expect total operating expenses, SG&A and R&D, to be approximately 600 million euro. And we expect to be operating cash flow break even on a quarterly basis by the end of 2024. Let me now also highlight selected key milestones. For TransCon Growth Hormone, we now plan to submit an SBLA to FDA for adult GHD in the third quarter of 2024 compared to the previous plan of Q2. 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 Yorvipath as quickly as possible thereafter. Outside the U.S., with a commercial rollout of Yorvipath underway in Germany and Austria, we plan to roll out Yorvipath in our Europe Direct, and international markets segments throughout 2024 and 2025. For Transcon C&P, we expect to report top-line results from the Pivotal Approach Trial as well as submit our NDA for treatment of children with achondroplasia, both in the fourth quarter of 2024, and also report Week 26 top-line data from the COACHE Trial in combination with Transcon Growth Hormone, also in the fourth quarter of 2024. Within our oncology therapeutic area, during the fourth quarter of 2024, we plan to provide a clinical update from the Phase II indication-specific dose expansion cohorts from our TRANSCON IL-2 Beta Gamma and TRANSCON TLR7-8 agonist clinical trials. With that, operator, we are now ready to take questions.
spk05: Thank you. If you'd like to ask a question, please press star 1-1. If your question has been answered and you'd like to remove yourself from the queue, please press star 11 again. We ask that you please limit yourself to one question and a follow-up. Our first question comes from Jessica Fye with J.P. Morgan. Your line is open.
spk04: Hey, guys. Good afternoon. Thanks for taking my question. I was hoping you could talk about how April looked for your path in Europe and maybe elaborate a little more on some of those one-queue dynamics you mentioned for Skytropa. Thank you.
spk27: Should we start?
spk03: Yeah. So related to the Q1 dynamics, for Skytroph, I'll take the second part first. What we saw would be, I would describe as typical seasonality that you would see with other maturing launches where coverage updates at the beginning of the year led to insurance reauthorizations, copay resets, and also led to a slightly different mix in the channel. And our channel partners basically updated their inventories as appropriate. And it feels like those basically all cleared out by the end of February. And then on the first question related to the development of EuroPath in April, you know, we continue to, as we said, we'll come out with an update overall on revenue later this year.
spk30: know essentially we're pretty happy with the results of the launch uh with the primary goals that yen laid out in his uh prepared remarks so just just to give you a little bit of flavor from our own expectations we launched kartrofa in the typical way that sorry you're perhaps inside the german market austrian market up from this assumption that we're adapting to the german market And what we typically will see and what we hope for, want to get a broad prescriber base. The broad prescriber base, we expected them to take typical one to two patients on treatment. Then they get really the feedback, seeing really the unique benefit of this. And as soon as we're really getting over that, some case comfort period where they really get the comfort you take more more on treatment so when we saw when we got 25 of our target position already in eight weeks to make a prescription i believe there was some of the most successful loans i have seen and we have not in ourselves imagine that the number of patients We're seeing it's around 100, meaning that there is a few physicians that started early in our ERP program or in Germany. Some of them are up on 20, 30 patients already now because they already had building up discomfort on how really this product are giving unique benefit to the patients. So this is why we were really feeling This is really the start we had hoped for, the start where we're looking on the different KPI. It really turned out to what we wanted to see and what we hoped for.
spk03: And just to reiterate, the results are essentially sales direct to the patient. They go to the retail pharmacy. The patient picks up. There's no channel inventory with your event.
spk05: Thank you. Our next question comes from David Lebowitz with Citi. Your line is open.
spk13: Thank you very much for taking my question. If you jump back about a year and you were in the run-up to your past potential approval for the regulatory setback, could you juxtapose the current status of your sales preparation, what the market looks like now, versus what it looks like then and how you see things similarly or differently?
spk30: I believe we are in a much, much better place. First of all, the awareness of hypopara has been really been coming to a much better awareness level where a physician and the patient just in the latest effort when if you really want to hear the patient voice, was the YouTube that is reflecting how the patient explains the unmet medical need that is in hyperparameter at the FDA advisory board. And I think that is only one element of it, why I believe we are in a much, much better place. And I think the realization of the unmet medical need we have already and always seen from the patient, what I think the awareness of is on a much, much better level today. And I think this is why when we see our success with the initial part of our launch in Germany, Austria, we know we can repeat that in all our eu direct as soon as we get fully reimbursed in these countries mainly end of this year in the beginning of next year i'm doing 25 we see it already coming in for the international market mainly to name patient programs which are patient driven program physician driven program so the general Awareness about the unmet medical need in hyper para I believe had really really gone to a stage where I hope We really can understand this unmet medical need to a much better way One thing that also have driven is the new guidelines that came up from us and other places really describing how basic Convinced therapy basic not providing an optimal Treatment, if you can call it a treatment, I would basically say that it's a balance between treatment for short-term symptoms and to avoid long-term complications, and you can never get that right. So it basically, you are in a position that the recommendation of PTH therapy is really the way to treat this disease, which are pretty obvious. Will you treat insulin? diseases with not insulin, like diabetes, yes, you will always treat them with insulin.
spk22: Thanks for taking my question.
spk05: Thank you. Our next question comes from Rosemary Lee with Cantor. Your line is open.
spk21: Hey, sorry, I should have put Lee Wacek. I'm on for Lee Wacek from Cantor. So just for Transcom PTH, in terms of payer access and formulatory placement, have you had any pushback from payers on showing clinical benefits such as hospitalization rate or kidney benefit? Thank you.
spk30: Is that question related to Europe or U.S.?
spk20: Both, if you may.
spk30: Okay. I think in Europe, when we do make what we call the health economic impact of our treatment, we are integrating all aspects on how we addressing short-term symptoms the benefit of the patient to have a normalized life again benefit of having a balanced calcium and more important the quality of life this patient are experiencing immediately after starting treatment the other part is what we call the long-term risk One part of the long-term risk is what we call renal impairment, renal damage. There is other long-term complication like cardiovascular impact that is calcification and a lot of different organs, cataract, and other things you're also achieving and will get with hypopara just dependent on time. All of this element is being built into our health economic evaluation that we are providing to each single European countries to be quite sure that we're getting the right reimbursement on right prices. So it's basically built on the European way of really being responsible in our pricing where we have an health economic calculation on how we basically are providing both the benefit to the patient, the benefit really to the society in helping the patient on a reasonable, accessible cost.
spk19: Thank you.
spk05: Thank you. Our next question comes from Joseph Schwartz. With learning partners, your line is open.
spk10: Thanks very much. I was wondering if you could talk about how many physicians in the U.S. have had experience with Yorvapath in the EAP versus how many you'll be going out to de novo once it's hopefully launched commercially soon and And could you also talk about how many of the physicians who might be targets for using Yorvapath in their patients have you been interacting with for Skytropha already?
spk30: I think the first question was reflecting your past and the US ERP program. And what I can do, I can give you the general aspect of our ERP program. The general aspect of our US ERP program, because it's very, very different compared to what we had in Europe, which now has been stopped after we got our approval and got full commercial in Germany. Our ERP program in U.S. is only addressing patients that really are PTSD experienced. Meaning is that it's a 3, 4, 5% subset of patients of hyperparalysis that basically are electable to come into our ERP program. The ERP program is a very difficult program for basic large academic institutions to handle because of contracting and the incentive for them is not high because it's not really a paid program for the physician. So therefore you will always find a subset of these sites that typically have a lot of patients in hyperpara will not really be part of such an ERP program. It will only be more private sites, small sites. We are extremely enthusiastic about what we have seen with our ERP program and all the treatment benefit that we have observed in our clinical trial, both phase 2 and phase 3, has been 100% confirmed what we have seen out from the ERP program and the patient experience there.
spk10: Okay. And so how many new sites will you have to go to then that are, you know, that treat hypoparathyroidism patients who have not had experience in the EAP or with using Skytropha?
spk30: At Skytropha, we don't have any EAP site at all in this way. Skytropha didn't ever have any EAP program running. So from that perspective, it's really hard for me to compare these two.
spk10: I meant commercial Skytropha or the EAP for YorvaPath. I'm just trying to get a sense of the footprint now, how much overlap there is. with, you know, between the two products that we'll have shortly.
spk30: You will basically say there is not a large overlap in directly-developed decision prescription space because it's typical more specialized sites that take care of both sides. One of them is, for example, a pediatric first indication. The other one is an adult indication. So when you look at the overlap for that, then there is not a large overlap, and I can I'm not seeing any data where I will indicate support and saying there's a big overlap. Where there is a great overlap is, hmm, 80, 90% of the commercial effort that basically are built on taking the same endocrinology product out in the same infrastructure. And what we basically are building, we are building independent sales forces that really will tailor me exactly the right physician to have the most prescribing physician being covered as fast as possible. Thank you.
spk15: Thank you.
spk05: Thank you. Our next question comes from Gavin Carter with Evercore ISI. Your line is open.
spk15: Hey, congrats on the progress, and thanks for taking the question. I'm just wondering, out of the 100 Yorvapath patients in Germany, how many of these were previously on NAPAR, and was this in line with your expectations heading in?
spk30: I would say, surprisingly, we see many more naive patients. that we actually have thought we will see. So this is one of the surprises we have seen that the physician basic are looking on the patients and saying is we have so many on patient that not have been in treatment that we also are focused on taking the new patient into the treatment machine. And I have to say that was one of the surprises, at least for me, where I would believe that it was initially just a change of NAPAR patient, but we have seen many more new patients that we actually expected.
spk15: Great.
spk07: Thank you. Kelly Shee with Jefferies.
spk05: Your line is open.
spk18: Thank you for taking my question. Schedule for, could you provide your perspective into the competitive landscape, given that two other long-acting growth hormone commercial products are there, and one of them, if we check on the script, actually approaching schedule for both TRX and the NRX? And also I'm curious whether your launch strategy remains the same.
spk30: So one of the complications at least I always have is to utilize the different databases you can get to get scripts. In this case, you can use this kind of data set is to look on trends, but never absolute levels because the sampling is really different between the different provider. So you are only sampling a small amount of the prescribing base where you get the information from. So from that perspective, you cannot really compare the absolute level. You cannot compare it also because When we talk about the unit, the unit is really different between the different products. So some unit is giving, providing you that. So therefore, if you really want to get an really effective and most solid data, you need to take each single company, go into the line item and look on the revenue basis. This is where I get the best confirmation about data and how we see their progression. And there is no doubt when we look on that. For the growth hormone market, it was a very difficult Q1. And what we saw really have seen how we were the only one of the reported data really have growing where everyone has declined, which you typically would see in the first quarter.
spk00: Thank you.
spk05: Thank you. Our next question comes from Derek Arcilla with Wells Fargo. Your line is open.
spk11: Hey, thanks for taking the questions. Congrats on the progress. So we have a question on the OpEx progression this year. I guess based on the 1Q OpEx, it seems like you might come in a fair bit below the 600 million euro guidance. So just, you know, any color there on how we should be thinking about that. Thanks.
spk30: I think Scott is so happy today because he's really getting a good question now.
spk03: Yeah, Derek, thanks for the question. We've been pretty, yeah, no, we're pretty proud of the, you know, our ability to basically reduce OPEX pretty significantly while growing, you know, doubling revenue. It's not a bad thought, but as of now, our guidance, you know, remains 600 million OPEX for the full year based on current plans.
spk07: Thank you. Our next question comes from Paul Choi with Goldman Sachs.
spk05: Your line is open.
spk14: Good afternoon and thank you for taking my question and also let me offer my congratulations on the progress. I want to turn maybe to CMP for a moment and if you could maybe sort of comment on what your market research ahead of your top line results later this year suggests in terms of how endocrinologists might be thinking about using TransConCNP either as new patients start or do you possibly anticipate switches from VoxOgo just given the different dosing frequency and how you're thinking about what those kind of shares might look like in terms of the launch? And then could you maybe comment on what your, you know, sort of post-approval requirements might potentially look like for TransConCNP, you know, pending an approval down the road from the FDA? Thank you.
spk30: It's really extremely interesting question from the perspective on when we're looking on all the research we have done. Because the key topic and the key feedback we're getting in all interaction with physician, caregivers, patient is addressing comorbidities. Being short is not a disease. But really to have this sign of co-morbidities is really providing really the impact on having aquantoplasia. And this is why we have so much strong focus really to address the co-morbidity and also to address also linear growth. But the key objective of our program is to address the co-morbidities. We cannot avoid a primary efficacy endpoint is linear growth because it has been established from regulatory agencies both in US and Europe. And it's really, really difficult to change that. If we have been the first, it will not have been linear growth. It will be addressing co-morbidity as the fundamental of the disease. And this is how our integrated program has really been built up to show that in all our programs. And I think where we both address the linear growth, but also muscle weakness and other things like that. And when I see how we can already get the feeling about what it means for the patient is when we look at the quality of life questions that we have given to both patient and caregivers. How they really are judging the benefit of our treatment. And we also see that in our retentions. When we see the retention and our recoupment, we recouped our pubertal tribe in less than four months. On site, which have access to zootype, have access to other treatment, it got recouped in four months. I've never seen that before. And this is because the physician could talk to the parents about the benefits they have seen based on providing our Transcon CMP product to them. Related to commitment afterwards, we have no comments or anything we have received from regulatory agencies in all the different places we have been and discussed the pathway for regulatory approval. to any kind of commitment.
spk08: Thank you.
spk05: Thank you. Our next question comes from Vikram Purohit with Morgan Stanley. Your line is open.
spk16: Hi. Thanks for taking our questions. We just had one on the pipeline. So a few months ago, you discussed with us a novel TransCon carrier platform, and you cited your work here with semaglutide through a case study. I just wanted to see how your internal work with this novel platform and the GLP-1 program is progressing and what the next milestones could be here that we could learn about. Thanks.
spk30: We are extremely excited about this lead candidate that we were for when we came out. There's no doubt that once the treatment regime is the way to go and we're building up the fundamentals that we built up all our pipeline. I call it a pipeline because we basically are not taking the target engagement risk that you typically have when you make highly differentiated product because we're building through the Transcon technology on a semi-glotide molecule that basically has the broadest way to show clinical benefit everywhere. We are progressing with that. At the same time, we also have an intense discussion about how we basically are doing the best value proposition of this compound and this area of metabolic diseases, because we talk about metabolic diseases. And we will keep you updated as soon as we someday have made a decision what way we're going.
spk05: Thank you. Our next question comes from Yaron Werber with TD Cowan. Your line is open.
spk01: Hey, guys. This is Joyce on for Yaron. Thanks for taking our question. In the U.S., can you talk about your plan launch strategy for transcom PTH in terms of the initial target population and prescriber base, and how much of it will mirror your strategy in Germany where you're initially targeting roughly one-third of the total population? Thank you.
spk30: I think what we're doing now is to really integrating the learning from the first country where you're launching a product. And the first country we launched is in Germany, Austria, and we're getting a lot of good learning. I have to say, we basically got confirmation about how we should do it because we basically have seen everything what we have hoped for in this launch. So it's not like we're coming up with a completely different strategy. We are more proud about the commercial execution from our people in this region, how they basically made it exactly after what I call the playbook. So when I come to the playbook of US, that is always heading up Camilla, that is our head of our global commercial operation. She's coordinating everything from US coordinating into international market, correlating into EU direct every place to be sure that we're building up the right strategic approach for BASIC to have the optimal commercial loans on a global base. We will be here and BASIC has seen really the huge interest. I can say we have the first commercial US product or person on commercial product for your parent is a person living in u.s that took the consequence on the delay in the u.s to basic fly to germany and get the product out there so we we know it we see it we know your paths making really and huge difference for the patient really get them their life back. And we really have seen how patients also see this benefit.
spk01: Thank you.
spk05: Thank you. Our next question comes from Leland Gershell with Oppenheimer. Your line is open.
spk09: Hey, good afternoon. Thanks for taking our questions. Two from us. Again, if you could just comment on the plan for rolling out your path in Great Britain following the approval. Is that something you're doing immediately, or will there be any delay? And secondly, in the past you had mentioned hypochondriplasia is an indication that you've decided to not pursue. Just wondering if that outlook might change following data from the achondro phase three that you'll be seeing later this year and or any information that may be coming up from the companies that are pursuing hypochondriac pressure. Thank you.
spk30: Yeah. UK is part of our Europe direct plan. And we have a rollout on all our countries that is in our EU direct. And UK is integrated in this rollout. So after we basically had the UK approval, which is now separated from an EU approval, like other countries that we are also getting approval in now, we continue filing everywhere from the different countries and, for example, Australia, everywhere. where we also need an independent approval we will then come in and go in and do what we call typical and filing of a dossier that really are describing the health economic impact as we have done in many EU direct countries but we also doing in other countries, and when we have reached an agreement with the agencies in the specific country about the reimbursing price, you typically will come into what we call a full commercial launch. What we're doing in the meantime, until we have this full commercial launch, we're building up the infrastructure, and we have done that in most of the EU direct countries. where we have a general manager, we have medical affairs, we have market assess teams, all that is basically already been established. And they are ready to be coming out and doing a full commercial loan launch that, for example, which we have done in Germany and Austria. In the meantime, a physician, patient can go to a main patient program. where we can get fully reimbursed patients which are built on an in-page program or similar structures in a lot of different countries. In this timeframe, until you will be fully commercial, you basically can provide commercial reimbursed product to the patient to all these specific programs. So this is what we call the dual strategy we are utilizing in the Europe Direct, which we also will someday implement in the international market, but we will not be the M&A holder in the international market. It will typically be handed to our sales and distribution agents. For the other thing about hypo contemplation, it really, really is an interesting aspect for us. Because we are the only company that have the two corn stones in growth disorders. Growth disorders is about 30-40 different diseases. We are the only company that have a once-weekly growth hormone, a once-weekly CMP molecule with really the best-in-class properties for both of these two mode of actions. So what we are doing, and that is part of our vision, 20 by 30, making an integrated strategy how we are going to be the leading company in growth disorder. And that is not only to address hypercontraplasia, but address the other, including the other 30 other diseases that is in growth disorder. And we believe some of them will be best treated with growth hormone. Some of them will be best treated with CMP. but also many of them will potentially be integrated in a combination therapy. And this is why we believe why we can be positioned to be the leader in growth disorder, because we have the two cornerstones for basic to handle all the 30 disease. So much more to come when we come up next year, where we will come with our integrated strategy, how we're building up to be the leading company in growth disorders. Thanks.
spk05: Thank you. And our last question comes from Sushila Hernandez with Kempen. Your line is open.
spk17: Yes, thank you for taking my question. Could you elaborate on Skytrover becoming a blockbuster in the US alone? What needs to happen? What are the key drivers? Thank you.
spk30: The key driver is continue what we're doing. This is exactly what we're doing. We are doing a continuum, showing get the physician, the patient, to really getting the treatment benefit of Skytropha as the best in class product opportunity inside the growth disorder. We would be helped by a lot of different things. The consolidation of the daily growth hormone market. We saw one of the major player is now stopping. We see other of the major player with daily growth hormone stopping. So the consolidation of the Daily growth hormone, where potentially one or two players will be left in more or less in a cash segment, it's really, really, really only the beginning of it. We're still in a small part of it. We believe we have less than 20% of the growth hormone deficiency, pediatric growth hormone deficiency. We can also grow it now in adult growth hormone deficiency. We have our Turner trial coming in now. And then we also see the more and more experienced people get of the long-acting, they realize there's only one choice. And I don't need to say that name, because there's only the sky that really, some way, give the limit for this product.
spk17: Thank you.
spk05: Thank you. There are no further questions. Thank you for your participation. This does conclude the program, and you may now disconnect. Everyone have a great day.
spk08: Thanks so much. you Thank you. Thank you. Bye. Bye.
spk05: Good day and welcome to the Q1 2024 Ascendance Pharma Earnings Conference Call. At this time, all participants are in listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question, please press star 1-1. We ask that you please limit yourself to one question and a follow-up. As a reminder, this call may be recorded. I would now like to turn the call over to Tim Lee, Senior Director, Investor Relations, Ascendus Pharma. Please go ahead.
spk02: Thank you, Operator, and thank you, everyone, for joining our first quarter 2024 Financial Results Conference Call. I'm Tim Lee, Senior Director, Investor Relations at Ascendus 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. Sina Singhal, Executive Vice President of Clinical Development Oncology, and Joe Kelly, U.S. General Manager. 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 Skytropha for the U.S. and European markets, as well as certain financial expectations for 2024, our commercialization and development of YorbaPath in the EU and expected timing of the FDA review and potential launch of Transcon PTH in the U.S., 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, and our progress towards Vision 2030. These statements are based on information that is available to us today. Actual results may differ could differ materially from those in our four linking statements, and you should not place undue reliance on these statements. We assume no obligation to up these statements as circumstantial change, except as required by law. For additional information concerning the factors that can cause actual results to differ materially, please see our four linking statement section in today's press release and the risk factors section of our most recent annual report on Form 20F filed with the SEC on February 7th, 2024. Transcon Growth Hormone, or Transcon HGH, is approved in the U.S. by FDA, and the EU has received MAA authorization from the European Commission for the Treatment of Pediatric Growth Hormone Deficiency. The European Commission and the United Kingdom Medicines and Healthcare Product Regulatory Agency have granted marking authorization for Transcon PTH as 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 first quarter 2024 financial results and we'll provide further business updates. Following some prepared remarks, we'll open the call up for questions. Note, with our PDUFA date for Transcon PTH coming up in less than two weeks, we will not comment on our ongoing discussions with FDA, and we will not be taking any questions on this topic today. With that, let me turn it over again.
spk08: Thank you, Tim. Good afternoon, everyone.
spk30: CENDIES is applying its Transcon technology platform to build a leading fully integrated biopharma company focused on making a meaningful difference in patients' lives. Two approved Transcon products and solid progress across programs, growing commercial presence and strong partnerships We believe Ascentis is on the path to sustainable growth and operating cash flow quite evenly on a quarterly basis by the end of 2024. Our long-term commitment in the last 3-4 years to build up profitable and robust supply chains The decision we took in 2023 to streamline the company has made us a leaner and more efficient organization. You can see the progress towards operating cash flow per year later this year without compromising our development and commercialization process. We believe we are well positioned. to successfully deliver on our strategic goals and close out our Vision 3x3 with regulatory approvals for three independent endocrinology rare disease products and continue building a pipeline in other therapeutic areas. Looking to Vision 2030, we believe each of our three rare disease endocrinology products have the potential to achieve blockbuster status. while we also further expand our pipeline and transport platform for future innovation. We have seen validation of our commercialization approach with the ongoing success of Skytrofa in the US and are seeing it again with successful norms of Europass in Germany and Austria. In the US, the PDUFA date for transport PTAs is coming up in less than two weeks on May 14th. If approved, we expect to be ready to launch in the U.S. soon thereafter in the third quarter. Now, let me give an update on each of our programs. When we launched SCARTOFA in the U.S. about two years ago, we had two goals. One to make SCARTOFA the leading product in the world. The second to drive the U.S. growth hormone market to become a 3 billion market. Our strategy is working. Thousands of patients are now treated with Skytover, the value leader in the U.S. We have a reliable supply chain, ensuring that every patient can benefit from Skytover once they have approval from their insurance company. We estimate that Skytover penetration in the U.S. pediatric growth hormone deficiency patient population grows to about 17 at the end of the first quarter. We are proud to observe Skytrover extend its market value leadership as the only growth hormone product to grow in value in the first quarter of 24, based on reported results. And we believe Skytrover will expand the US growth hormone with the potential to become a blockbuster in the US alone. Skytober sales this quarter more than doubled compared to the first quarter of 2022, with a steady quarter to quarter increase in treated patients. We expect to continue this trend rest of this year. With these strong Skytober results, we continue to expect the full year 2025 Skytober revenue will be between 320 to 340 million euros. representing year-to-year growth of 80% to 90%. We expect Gartofa sales to continue to grow through further penetration in the pediatric ophthalmology system. To further solidify growth, we are also pursuing our first labor expansion in adult ophthalmodeficiency, for which we plan to submit and supplement BLA to the FDA in the third quarter of this year. In addition, We expect top line data from our phase two trial internal syndrome in the fourth quarter of 2024. Now turning to Transcon PTH. In Europe, EUROPATH was launched in Germany and Austria at the end of January with the early launch objective of building physician experience with EUROPATH. Initial physician feedback from EUROPATH has been positive. Just eight weeks in the lungs, we estimate prescriptions have been written by 55 doctors representing around 25% of the target prescribing base with about 100 patients receiving commercial product. As physicians begin to get comfort with the treatment benefit of UroPelt in the first patient, we expect physicians to take more and more of the patient treatment. As we expand our geographic reach of Europe to our Europe direct and our international market sector, we are supporting named patient supply programs and plan to provide reimbursed products to meet the needs of patients. In the U.S., pre-launch preparations are underway, including the expansion of the U.S. field infrastructure, while we are awaiting FDA's decision. Moving now to Transcon-CMP. Our value proposition with Transcon-CMP is simple. It's to establish a treatment for patients of all ages with achondroplasia. Our ambition is to address the significant co-morbidities associated with aconthoplasia that impact health and quality of life, as well as linear growth. Later this year, in the third quarter, we plan to report top-line results for our pivotal approach trial, which is measuring not only linear growth, but also physical function, body composition, and quality of life parameters. We are also evaluating transcon-CMP in newborns with air contemplation less than two years of age. We believe that treatment of air contemplation with transcon-CMP as early as possible might mitigate associated comorbidities. In addition, to provide catch-up growth to children who did not receive early treatment, we are evaluating transcon-CMP in combination with transcon growth hormone. We expect to enroll all patients in this trial this quarter and provide 26-week top-line data for this trial in the fourth quarter of this year. This is our integrated approach to address acromiopasia with multiple trials that are all building towards one goal to treat the disease. And we are optimistic that our highly differentiated product candidate will continue to show best-in-class potential across these multiple studies. Switching now to oncology. Both Transcon IL-2 beta-carbon and Transcon TLR-7-8 agonist has shown promising clinical activity as monotherapy and in combination treatment with PEMBO in late-stage patients whose disease has progressed after standard of care treatments. Next month at ASCO, we will report updated patient data from the ongoing Phase 1-2 trial, I believe, that has been reported last year at ESMO. We will present updated clinical data and new biomarker data that further differentiate our clinical programs. We will also report promising early data from the ongoing dose expansion cohort of patients with melanoma who have progressed from checkpoint in Ibrisus using the combination of Transcon IL-2 beta gamma and Transcon TLR agonists in these patients. Later this year, in the fourth quarter, we expect data readout in several well-defined patient populations from our Transcon IL-2 beta gamma and Transcon TLR-7-8 aggregate programs. Our achievement this quarter gave me further confidence that all the elements are in place to fulfill our strategic goals to deliver three independent endocrinology rare disease block dog products. and a strong pipeline in dark-cell telepathy areas such as oncology, ophthalmology, and metabolic diseases. There is much more to come. I will now turn it over to Scott.
spk03: Thank you. In Q1, we demonstrated significant financial progress for our goal of becoming operating cash flow breakeven on a 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 6K filed today. Skytrofa revenue for the first quarter of 2024 was 65 million Euro compared to 31.6 million Euro reported in the first quarter of 2023, an increase of 106% year over year. This growth was driven by significant increase in demand volume, more than doubling compared to the prior year period, with a slight offset by a combination of slower channel bill coverage mix and a modest negative currency impact of €0.8 million. On a sequential basis, first quarter Skytropha revenue increased 1% compared to the fourth quarter of 2023. Strong mid-teens percent demand volume growth was offset primarily by seasonal channel inventory, copay resets, insurance reauthorizations, and a modest negative currency impact of €0.6 million. Q1 was in line with our high internal expectations and with seasonal headwinds behind and the steady pace of new patient ads expected to persist. We continue to expect full-year Skytropha revenue to be in the range of €320 million to €340 million at average 2023 exchange rates. Shifting to Transcom PTH, Europath contributed for the first time this quarter with revenue of €1.5 million, representing two months of shipments. In Germany and Austria, we ship directly to retail pharmacies for patient pickup, and as a result, there is no channel inventory buildup compared to a typical U.S. launch like we saw with Skytropha, where specialty pharmacies hold channel inventory. Closing out the top line, total revenue for the first quarter was 95.9 million euro, including 24.8 million euro of non-cash license revenue recognized in relation to the formation of ICONIS, and €3 million of service revenue related to ICONIS, which is offset in operating expenses. Turning to expenses, R&D costs in the first quarter totaled €70.7 million compared to €106.1 million during the first quarter of 2023. The 33% decline was largely tied to lower external development costs for Transcon Growth Hormone and TransCon PTH, including a reversal of prior period write-downs of pre-launch inventories, as well as oncology programs, partially offset by an increase in TransCon C&P costs. Sequentially, R&D costs declined 22%. SG&A expenses in the quarter totaled 66.8 million euro, essentially flat compared to 66.5 million euro during the first quarter of 2023. Higher employee costs, including the impact from commercial expansion, was partially offset by lower external pre-launch and administrative expenses. Sequentially, SG&A expenses increased 4%. Total operating expenses were 137.5 million Euro for the first quarter, a 20% decrease compared to the 172.7 million Euro during the first quarter of 2023. Sequentially, operating expenses declined 11%. Overall, our operating loss in the first quarter totaled 49.1 million euro compared to an operating loss of 143.7 million euro during the first quarter of 2023 as a result of increased revenue and lower operating expenses. Sequentially, operating loss increased 34%. Finance expense in the quarter was 77.2 million euro compared to 8.4 million euro expense in the fourth quarter of last year. This higher finance expense was largely driven by a non-cash derivative loss tied to our outstanding convertible notes. Quick comment on the balance sheet. As of March 31, 2024, due to amended IFRS rules, which came into effect on January 1, 2024, You'll notice our convertible notes with face value of 575 million U.S. dollars are now reported as current liabilities even though they do not mature until April 2028 and would not require cash settlement in case of conversion by holders. IFRS still requires the carrying value of the convertible notes and associated derivative liabilities to be presented separately within current liabilities, which together total 622 million euro. As per IFRS rules, comparative amounts have been reclassified to reflect the change in presentation. The applied amendments had no other impact on the financial statements. We ended the first quarter with cash and cash equivalents totaling €320 million. And for the full year 2024, based on current plans, we expect Skytropha revenue to be in the range of €340 million at average 2023 exchange rates. We expect total operating expenses, SG&A and R&D, to be approximately 600 million euro. And we expect to be operating cash flow break even on a quarterly basis by the end of 2024. Let me now also highlight selected key milestones. For TransCon Growth Hormone, we now plan to submit an SBLA to FDA for adult GHD in the third quarter of 2024 compared to the previous plan of Q2. 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 Yorvipath as quickly as possible thereafter. Outside the U.S., with a commercial rollout of Yorvipath underway in Germany and Austria, we plan to roll out Yorvipath in our Europe Direct and international markets segments throughout 2024 and 2025. For Transcon C&P, we expect to report top-line results from the Pivotal Approach Trial as well as submit our NDA for treatment of children with achondroplasia, both in the fourth quarter of 2024, and also report Week 26 top-line data from the COACHE Trial in combination with Transcon Growth Hormone, also in the fourth quarter of 2024. Within our oncology therapeutic area, during the fourth quarter of 2024, we plan to provide a clinical update from the Phase II indication-specific dose expansion cohorts from our TRANSCON IL-2 Beta Gamma and TRANSCON TLR7-8 agonist clinical trials. With that, operator, we are now ready to take questions.
spk05: Thank you. If you'd like to ask a question, please press star 1-1. If your question has been answered and you'd like to remove yourself from the queue, please press star 11 again. We ask that you please limit yourself to one question and a follow-up. Our first question comes from Jessica Fye with J.P. Morgan. Your line is open.
spk04: Hey, guys. Good afternoon. Thanks for taking my question. I was hoping you could talk about how April looked for your path in Europe and maybe elaborate a little more on some of those one-queue dynamics you mentioned for Skytropa. Thank you.
spk27: Should we start?
spk03: Yeah. So related to the Q1 dynamics, for Skytroph, I'll take the second part first. What we saw would be, I would describe as typical seasonality that you would see with other maturing launches where coverage updates at the beginning of the year led to insurance reauthorizations, copay resets, and also led to a slightly different mix in the channel. And our channel partners basically updated their inventories as appropriate. And it feels like those basically all cleared out by the end of February. And then on the first question related to the development of EuroPath in April, we continue to, as we said, we'll come out with an update overall on revenue later this year. Essentially, we're pretty happy with the results of the launch, with the primary goals that Jens laid out in his prepared remarks.
spk30: So, just to give you a little bit of flavor from our own expectations, we launched Kartoffel in the typical way that, sorry, you were perhaps inside the German market, Austrian market, out from this assumption that we're adapting to the German market. And what we typically will see and what we hope for, want to get a broad prescriber base. The broad prescriber base, we expected them to take typical one to two patients on treatment. Then they get really the feedback, seeing really the unique benefit of this. And as soon as we're really getting over that, some take a comfort period where they really get the comfort, you take more and more on treatment. So when we saw when we got 25% of our target physician already in eight weeks to make a prescription, I believe there were some of the most successful loans I have seen. We have not in ourselves imagined that. The number of patients We're seeing it's around 100, meaning that there is a few physicians that started early in our ERP program or COPD program in Germany. Some of them are up on 20, 30 patients already now because they already had building up discomfort on how really this product are giving unique benefit to the patients. So this is why we were really feeling This is really the start we had hoped for, the start where we're looking on the different KPI. It really turned out to what we wanted to see and what we hoped for.
spk03: And just to reiterate, the results are essentially sales direct to the patient. They go to the retail pharmacy. The patient picks up. There's no channel inventory with YorbaPath.
spk07: Thank you.
spk05: Our next question comes from David Lebowitz with Citi. Your line is open.
spk13: Thank you very much for taking my question. If you jump back about a year and you were in the run-up to your past potential approval for the regulatory setback, could you juxtapose the current status of your sales preparation, what the market looks like now, versus what it looks like then and how you see things similarly or differently?
spk30: I believe we are in a much, much better place. First of all, the awareness of hypopara has been really been coming to a much better awareness level where a physician and the patient just in the latest effort when if you really want to hear the patient voice, was the YouTube that is reflecting how the patient explains the unmet medical need that is in hyperparathyroidism at the FDA advisory board. And I think that is only one element of it why I believe we are in a much, much better place. And I think the realization of the unmet medical need we have already and always seen from the patient, what I think the awareness of is on a much, much better level today. And I think this is why when we see our success with the initial part of our launch in Germany, Austria, we know we can repeat that in all our eu direct as soon as we get fully reimbursed in these countries mainly end of this year in the beginning of next year i'm doing 25 we see it already coming in for the international market mainly to name patient programs which are patient driven program physician driven program so the general Awareness about the unmet medical need in hyper para I believe had really really gone to a stage where I hope We really can understand this unmet medical need to a much better way One thing that also have driven is the new guidelines that came up from us and other places really describing how basic Convinced therapy basic not providing an optimal treatment treatment if you can call it a treatment i was basically saying that it's a balance between treatment for short-term symptoms and to avoid long-term complications and you can never get that right so it basically you are in a position that the recommendation of pta therapy is really the way to treat this disease which are pretty obvious when you treat insulin diseases with not insulin, like diabetes, yes, you will always treat them with insulin.
spk22: Thanks for taking my question.
spk05: Thank you. Our next question comes from Rosemary Lee with Cantor. Your line is open.
spk21: Hey, sorry, I should have put Lee Wacek. I'm on for Lee Wacek from Cantor. So just for Transcom PTH, in terms of payer access and formulatory placement, have you had any pushback from payers on showing clinical benefits such as hospitalization rate or kidney benefit? Thank you.
spk30: Is that question related to Europe or U.S.?
spk20: Both, if you may.
spk30: Okay. I think in Europe, when we do make what we call the health economic impact of our treatment, We are integrating all aspects on how we addressing short term symptoms, the benefit of the patient to have a normal life again, benefit of having a balanced calcium and more important, the quality of life this patient are experiencing immediately after starting treatment. The other part is what we call the long term risk. One part of the long-term risk is what we call renal impairment, renal damage. There is other long-term complications like cardiovascular impact. There is calcification on a lot of different organs, cataract, and other things you're also achieving and will get with Hypopar just dependent on time. All of this element is being built into our health economic evaluation that we are providing to each single European countries to be quite sure that we're getting the right reimbursement on right prices. So it's basically built on the European way of really being responsible in our pricing where we have an health economic calculation on how we basically are providing both the benefit to the patient, the benefit really to the society in helping the patient on a reasonable, accessible cost.
spk19: OK, thank you.
spk05: Thank you. Our next question comes from Joseph Schwartz. With learning partners, your line is open.
spk10: Thanks very much. I was wondering if you could talk about how many physicians in the U.S. have had experience with Yorvapath in the EAP versus how many you'll be going out to de novo once it's hopefully launched commercially soon. And And could you also talk about how many of the physicians who might be targets for using Yorvapath in their patients have you been interacting with for Skytropha already?
spk30: I think the first question was reflecting your past and the U.S. EAP program. And what I can do, I can give you the general aspect of our EAP program. The general aspect of our U.S. EAP program, because it's very, very different compared to what we had in Europe, which now has been stopped after we got our approval and got full commercial in Germany. Our ERP program in U.S. is only addressing patients that really are PTSD experienced. Meaning is that it's a 3, 4, 5% subset of patients of hyperparalysis that basically are electable to come into our ERP program. The ERP program is a very difficult program for basic large academic institutions to handle. So because of contracting and the incentive for them is not high because it's not really a paid program for the position. So therefore, you will always find a subset of these site that typically has a lot of patients in hyperpara will not really be part of such an ERP program. It will only be more private sites, small sites. We are extremely enthusiastic about what we have seen with our ERP program and all the treatment benefit that we have observed in our clinical trial, both phase 2 and phase 3, has been 100% confirmed what we have seen out from the ERP program and the patient experience there.
spk10: Okay. And so how many new sites will you have to go to then that are, you know, that treat hypoparathyroidism patients who have not had experience in the EAP or with using Skytropha?
spk30: Skytropha, we don't have any EAP experience. site at all in this way. Skytropha didn't ever have any EAP program running. So from that perspective, it's really hard for me to compare these two.
spk10: Well, I meant commercial Skytropha or the EAP for YorvaPath. I'm just trying to get a sense of the footprint now, how much overlap there is. between the two products that we'll have shortly.
spk30: You will basically say there is not a large overlap in the decision prescription phase, because it's typical more specialized sites that take care of both sides. One of them is, for example, a pediatric first indication. The other one is an adult indication. So when you look at the overlap for that, then there is not a large overlap, and I can I'm not seeing any data where I would indicate support and saying there's a big overlap. Where there is a great overlap is, hmm, 80, 90% of the commercial effort that basically are built on taking the same endocrinology product out in the same infrastructure. And what we basically are building, we are building independent sales forces that really will tailor me exactly the right physician to have the most prescribing physician being covered as fast as possible.
spk15: Thank you. Thank you.
spk05: Thank you. Our next question comes from Gavin Carter with Evercore ISI. Your line is open.
spk15: Hey, congrats on the progress, and thanks for taking the question. I'm just wondering, out of the 100 Yorvapath patients in Germany, how many of these were previously on NAPAR, and was this in line with your expectations heading in?
spk30: I would say, surprisingly, we see many more naive patients that we actually have thought we will see so this is one of the surprises we have seen that the physician basic are looking on the patients and saying is we have so many one patient that not have been in treatment that we also are focused on taking the new patient into the treatment regime And I have to say that was one of the surprises, at least for me, where I would believe that it was initially just a change of NAPAR patient, but we have seen many more new patients that we actually expected. Great. Thank you.
spk05: Kelly Shee with Jefferies. Your line is open.
spk18: Thank you for taking my questions. Could you provide your perspective into the competitive landscape, given that two other long-acting growth hormone commercial products are there, and one of them, if we check on the script, actually approaching schedule for both TRX and the NRX? And also, I'm curious whether your launch strategy remains the same.
spk30: So one of the complications, at least I always have, is to utilize the different databases you can get to get scripts. In this case, you can use this kind of data set is to look on trends, but never absolute levels because the sampling is really different between the different provider So you are only sampling a small amount of the prescribing base where you get the information from. So from that perspective, you cannot really compare the absolute level. You cannot compare it also because When we talk about the unit, the unit is really different between the different products. So some unit is giving, providing you that. So therefore, if you really want to get an really effective and most solid data, you need to take each single company, go into the line item and look on the revenue basis. This is where I get the best confirmation about data and how we see their progression. And there is no doubt when we look on that. For the growth hormone market, it was a very difficult Q1. And what we saw really have seen how we were the only one of the reported data really have growing where everyone had declined, which you typically would see in the first quarter.
spk00: Thank you.
spk05: Thank you. Our next question comes from Derek Arcilla with Wells Fargo. Your line is open.
spk11: Hey, thanks for taking the questions. Congrats on the progress. So we have a question on the OpEx progression this year. I guess based on the 1Q OpEx, it seems like you might come in a fair bit below the 600 million euro guidance. So just, you know, any color there on how we should be thinking about that. Thanks.
spk30: I think Scott is so happy today because he's really getting a good question now.
spk03: Yeah, Derek, thanks for the question. We've been pretty, yeah, no, we're pretty proud of the, you know, our ability to basically reduce OPEX pretty significantly while growing, you know, doubling revenue. It's not a bad thought, but as of now, our guidance, you know, remains 600 million OPEX for the full year based on current plans.
spk07: Thank you.
spk05: Our next question comes from Paul Choi with Goldman Sachs. Your line is open.
spk14: Good afternoon and thank you for taking my question and also let me offer my congratulations on the progress. I want to turn maybe to CMP for a moment and if you could maybe sort of comment on what your market research ahead of your top line results later this year suggests in terms of how endocrinologists might be thinking about using TransConCNP either as new patients start or do you possibly anticipate switches from VoxOgo just given the different dosing frequency and how you're thinking about what those kind of shares might look like in terms of the launch? And then could you maybe comment on what your, you know, sort of post-approval requirements might potentially look like for TransConCNP, you know, pending an approval down the road from the FDA? Thank you.
spk30: It's really extremely interesting question from the perspective on when we're looking on all the research we have done. Because the key topic and the key feedback we're getting in all interaction with physician, caregivers, patient is addressing comorbidities. Being short is not a disease. But really to have this sign of co-morbidity is really providing really the impact on having acondalplasia. And this is why we have so much strong focus really to address the co-morbidity and also to address also linear growth. But the key objective of our program is to address the co-morbidities. We cannot avoid a primary efficacy endpoint is linear growth because it has been established from regulatory agencies both in US and Europe. And it's really, really difficult to change that. If we have been the first, it will not have been linear growth. It will be addressing co-morbidity as the fundamental of the disease. And this is how our integrated program has really been built up to show that in all our programs. And I think where we both address the linear growth, but also muscle weakness and other things like that. And when I see how we can already get the feeling about what it means for the patient is when we look at the quality of life questions that we have given to both patients and caregivers. How they really are judging the benefit of our treatment. And we also see that in our retentions. When we see the retention and our recoupment, we recouped our pubertal tribe in less than four months. On site which have access to zootype, have access to other treatment, it got recouped in four months. I've never seen that before. And this is because the physician could talk to the parents about the benefits they have seen based on providing our Transcon CMP product to them. Related to commitment afterwards, we have no comments or anything we have received from regulatory agencies in all the different places we have been and discussed the pathway for regulatory approval. to any kind of commitment.
spk08: Thank you.
spk05: Thank you. Our next question comes from Vikram Purohit with Morgan Stanley. Your line is open.
spk16: Hi. Thanks for taking our questions. We just had one on the pipeline. So a few months ago, you discussed with us a novel TransCon carrier platform, and you cited your work here with semaglutide through a case study. I just wanted to see how your internal work with this novel platform and the GLP-1 program is progressing and what the next milestones could be here that we could learn about. Thanks.
spk30: We are extremely excited about this lead candidate that we were for when we came out. There's no doubt that once the treatment regime is the way to go and we're building up the fundamentals that we built up all our pipeline. I call it a pipeline because we basically are not taking the target engagement risk that you typically have when you make highly differentiated product because we're building through the Transcon technology on a semi-glutide molecule that basically has the broadest way to show clinical benefit everywhere. We are progressing with that. At the same time, we also have an intense discussion about how we basically are doing the best value proposition of this compound and this era of metabolic diseases, because we talk about metabolic diseases. And we will keep you updated as soon as we someway have made a decision what way we're going.
spk05: Thank you. Our next question comes from Yaron Werber with TD Cowan. Your line is open.
spk01: Hey, guys. This is Joyce on for Yaron. Thanks for taking our question. In the U.S., can you talk about your planned launch strategy for transcom PTH in terms of the initial target population and prescriber base, and how much of it will mirror your strategy in Germany, where you're initially targeting roughly one-third of the total population? Thank you.
spk30: I think what we're doing now is to really integrating the learning from the first country where you're launching a product. And the first country we launched is in Germany, Austria, and we're getting a lot of good learning. I have to say, we basically got confirmation about how we should do it because we basically have seen everything what we have hoped for in this launch. So it's not like we're coming up with a completely different strategy. We are more proud about the commercial execution from our people in this region, how they basically made it exactly after what I call the playbook. So when I come to the playbook of US, there is always a committee that is our head of our global commercial operation. She's coordinating everything from US. coordinating into international market, correlating into EU direct every place to be sure that we're building up the right strategic approach for BASIC to have the optimal commercial loans on a global base. We will be here and BASIC has seen really the huge interest. I can say we have the first commercial US product or person on commercial product for your parent is a person living in u.s that took the consequence on the delay in the u.s to basic fly to germany and get the product out there so we we know it we see it we know your paths making really and huge difference for the patient really get their life back, and we really have seen how patients also see this benefit.
spk07: Thank you.
spk05: Thank you. Our next question comes from Leland Gershell with Oppenheimer. Your line is open.
spk09: Hey, good afternoon. Thanks for taking our questions. Two from us. Again, if you could just comment on the plan for rolling out your path in Great Britain following the approval. Is that something you're doing immediately, or will there be any delay? And secondly, in the past you had mentioned hypochondriplasia is an indication that you've decided to not pursue. Just wondering if that look might change following the approval. data from the achondro phase three that you'll be seeing later this year and or any information that may be coming up from the companies that are pursuing hypochondriac pressure. Thank you.
spk30: Yeah. UK is part of our Europe direct plan. And we have a rollout on all our countries that is in our EU direct. And UK is integrated in this rollout. So after we basically had the UK approval, which is now separated from an EU approval, like other countries that we are also getting approval in now, we continue filing everywhere from the different countries and, for example, Australia, everywhere. where we also need an independent approval we will then come in and go in and do what we call typical and filing of a dossier that really are describing the health economic impact as we have done in many EU direct countries but we also doing in other countries, and when we have reached an agreement with the agencies in the specific country about the reimbursing price, you typically will come into what we call a full commercial launch. What we're doing in the meantime until we have this full commercial launch, we're building up the infrastructure, and we have done that in most of the EU direct countries. where we have a general manager, we have medical affairs, we have market assess teams, all that is basically already been established. And they are ready to be coming out and doing a full commercial loan launch that, for example, which we have done in Germany and Austria. In the meantime, a physician patient can go to an inpatient program. where we can get fully reimbursed patients which are built on a name page program or similar structures in a lot of different countries. In this timeframe, until you will be fully commercial, you basically can provide commercial reimbursed product to the patient to all these specific programs. So this is what we call the dual strategy we are utilizing in the Europe Direct, which we also will someday implement in the international market, but we will not be the M&A holder in the international market. It will typically be handed to our sales and distribution agents. For the other thing about hypo contemplation, it really, really is an interesting aspect for us. because we are the only company that has the two cornerstones in growth disorders. Growth disorders are about 30-40 different diseases. We are the only company that has a once-weekly growth hormone, a once-weekly CMP molecule with really the best-in-class properties for both of these two mode of actions. So what we are doing, and that is part of our vision, 20 by 30, making an integrated strategy how we are going to be the leading company in growth disorder. And that is not only to address hypercontraplasia, but address the other, including the other 30 other diseases that is in growth disorder. And we believe some of them will be best treated with growth hormone. Some of them will be best treated with CMP. but also many of them will potentially be integrated in a combination therapy. And this is why we believe why we can be positioned to be the leader in growth disorder, because we have the two cornerstones for basic to handle all the 30 disease. So much more to come when we come up next year, where we will come with our integrated strategy, how we're building up to be the leading company in growth disorders. Thanks.
spk05: Thank you. And our last question comes from Sushila Hernandez with Kempen. Your line is open.
spk17: Yes, thank you for taking my question. Could you elaborate on Skytrover becoming a blockbuster in the US alone? What needs to happen? What are the key drivers? Thank you.
spk30: The key driver is continue what we're doing. This is exactly what we're doing. We are doing a continuum, showing get the physician, the patient, to really getting the treatment benefit of Skytropha as the best in class product opportunity inside the growth disorder. We would be helped by a lot of different things. The consolidation of the daily growth hormone market. We saw one of the major player is now stopping. We see other of the major player with daily growth hormone stopping. So the consolidation of the Daily growth hormone, where potentially one or two players will be left in more or less in a cash segment, it's really, really, really only the beginning of it. We're still in a small part of it. We believe we have less than 20% of the growth hormone deficiency, pediatric growth hormone deficiency. We can also grow it now in adult growth hormone deficiency. We have alternate trials coming in now. And then we also see the more and more experienced people get of the long-acting, they realize there's only one choice. And I don't need to say that name, because there's only the sky that really, some way, gives the limit for this product.
spk17: Thank you.
spk05: Thank you. There are no further questions. Thank you for your participation. This doesn't include the program, and you may now disconnect. Everyone, have a great day.
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