Ascendis Pharma A/S (ASND) 2024 Q1 法說會逐字稿

Good day and welcome to the Q1 2024 Ascendis Pharma Earnings Conference Call. (Operator Instructions) As a reminder, this call may be recorded. I would now like to turn the call over to Tim Lee, Senior Director, Investor Relations of Ascendis Pharma. Please go ahead.

Thank you, operator, and thank you, everyone, for joining our first quarter 2024 financial results conference call on Tim Lee, Senior Director, Investor Relations at Ascendis Pharma. Joining me on the call today are Jan Mikkelsen, President and Chief Executive Officer; Scott Smith, Executive Vice President and Chief Financial Officer; Dr. Stina Singel, Executive Vice President of Clinical Development Oncology; and Joe Kelley, US 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 SKYTROFA for the US and European markets, as well as certain financial expectations for 2024. Our commercialization and development of YORVIPATH 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 result 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 forward-looking statements, and you should not place undue reliance on these statements. We assume no obligation to update these statements as circumstances change, except as required by law.

For additional information concerning the factors that could cause actual results to differ materially, please see our forward-looking statements section in today's press release and the Risk Factors section of our most recent annual report on Form 20-F filed with the SEC on February 7, 2024. TransCon Growth Hormone or Transcon hGH is approved by the US by FDA, and the EU has received MAA authorization from the European Commission for the treatment of pediatric growth hormone deficiency. The European Commission and the United Kingdom's Medicines and Healthcare Products Regulatory Agency has granted marketing 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 will 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 to Jan.

Thank you, Tim. Good afternoon, everyone. Ascendis is applying the TransCon technology platform to build a leading fully integrated biopharma company, focused on making a meaningful difference in patients' lives. To approve TransCon products, and solid progress across programs, growing commercial presence and strong partnerships, we believe Ascendis is on the path to sustainable growth and operating cash flow breakeven on a quarterly basis by the end of 2024.

Our long-term commitment in the last three, four years to build a profitable and robust supply chains ended the decision we took in 2023 to streamline the company and be offset leaner and more efficient organization. You can see the progress towards operating cash flow per key data this year. Without compromising our development and commercialization process, we believe we are well positioned to subset fully deliver on our strategic goals and close out our Vision 3x3 with regulatory approvals for three independent endocrinology rare disease product and continue building a pipeline in other therapeutic areas.

Moving to Vision 2030, we believe each of our three rare disease endocrinology products has the potential to achieve blockbuster status. One, 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 launch of YORVIPATH in Germany and Austria. In the US, the PDUFA date for TransCon PTH 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 SKYTROFA in the US, about two years ago, we had two goals; one, to make SKYTROFA the leading product in there. The second to drive US [go-to-market] to become a $3 billion market. Our strategy is working. Thousands of patients are now treated with SKYTROFA, the value leader of the US. We have a reliable supply chain, ensuring that every patient can benefit from SKYTROFA once they have approval from the [HE1 company]

We estimate the SKYTROFA penetration in the US pediatric growth hormone deficiency patient population growth to about 70 at the end of the first quarter. We are proud to observe SKYTROFA 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 SKYTROFA will expand the US go-to-market with the potential to become a blockbuster in the US alone.

SKYTROFA sales this quarter more than doubled compared to the first quarter of 2022, with a steady quarter-to-quarter increase in treating patients. We expect to continue this trend rest of this year. With this strong SKYTROFA results, we continue to expect the full year 2024 SKYTROFA revenue will be between EUR320 million to EUR340 million, representing year-over-year growth of 80% to 90%. We expect SKYTROFA sales to continue to grow through further penetration in the pediatric ophthalmic sales.

To further solidify growth, we are also pursuing our first labeled expansion in adult ophthalmic disease, for which we plan to submit and supplemental BLA to the FDA in the third quarter of this year. In addition, we expect top-line data from our Phase 2 in Turner syndrome in the fourth quarter of 2024. Now turning to TransCon PTH. In Europe, YORVIPATH was launched in Germany and Austria in the end of January. The early launch objective of building physician experience YORVIPATH.

Initial physician feedback from YORVIPATH has been positive. Just eight weeks in the launch, we estimate prescriptions have been written back 55 doctors representing around 25% of the target prescriber base with about 100 patients receiving conventional products. As physicians begin to get comfort with the treatment benefit of YORVIPATH in the first patient, we expect physician to take more and more of the patient on treatment.

As we expand our geographic regions YORVIPATH to our Europe direct and our international market segment, we are supporting Named Patient Supply programs and plan to provide reimbursed product to meet the needs of patients. In the US, prelaunch preparations are underway, including the expansion of the US field infrastructure while we are awaiting FDA's decision.

Moving now to TransCon CNP. Our value proposition with TransCon CNP is simple. It's to us that is a treatment for patients of all ages with achondroplasia. Our ambition is to address the significant co-morbidities associated with achondroplasia 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 CNP in newborns with achondroplasia, less than two years of age. We believe that treatment achondroplasia with TransCon CNP as early as possible mitigate associated co-morbidities. In addition, to provide catch up growth to children who did not receive early treatment, we are evaluating TransCon CNP 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 fourth quarter this year. This is our integrated approach to address achondroplasia 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 β/γ and TransCon TLR7/8 Agonist has showed promising clinical activity as monotherapy and in combination treatment is simple in late-stage patient whose disease has progressed after standard of care treatments. Next month at ESPO, we will report updated patient data from the ongoing Phase 1/2 trial IL-Believe that has been reported in 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 have progressed from checkpoint inhibitors using the combination of TransCon IL-2 β/γ and TransCon TLR Agonist disease patients.

Later this year, in the full fourth quarter, we expect data readout in civil well defined patient populations from our TransCon IL-2 β/γ and TransCon TLR7/8 Agonist programs. Our achievements this quarter can be further completed that all the elements are in place to fulfill our strategic goals to deliver three independent endocrinology rare disease product and a strong pipeline in larger therapeutic areas such as, oncology, ophthalmology, and metabolic diseases, with much more to come. I will now turn it over to Scott.

Thanks, Jan. In Q1, we demonstrated significant financial progress toward 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 6-K filed today.

SKYTROFA revenue for the first quarter of 2024 was EUR65 million compared to EUR31.6 million 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 build, coverage mix, and a modest negative currency impact of EUR0.8 million.

On a sequential basis, first quarter SKYTROFA revenue increased 1% compared to the fourth quarter of 2023. Strong mid-teens percent demand volume growth was offset primarily by seasonal channel inventory, co-pay resets, insurance reauthorizations, and a modest negative currency impact of EUR0.6 million. Q1 was in line with our high internal expectations and with seasonal headwinds behind in the steady pace of new patient adds expected to persist, we continue to expect full year SKYTROFA revenue to be in the range of EUR320 million to EUR340 million at average 2023 exchange rates.

Shifting to TransCon PTH. Europe has contributed for the first time this quarter with revenue of EUR1.5 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 US launch like we saw with SKYTROFA, where, especially, pharmacies hold channel inventory. Closing out the top line, total revenue for the first quarter was EUR95.9 million, including EUR24.8 million of non-cash license revenue recognized in relation to the formation of Eyconis and EUR3 million of service revenue related to Eyconis, which is offset in operating expenses.

Turning to expenses, R&D costs in the first quarter totaled EUR70.7 million compared to EUR106.1 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 CNP costs.

Sequentially, R&D costs declined 22%. SG&A expenses in the quarter totaled EUR66.8 million, essentially flat compared to EUR66.5 million 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 EUR137.5 million for the first quarter, a 20% decrease compared to the EUR172.7 million during the first quarter of 2023.

Sequentially, operating expenses declined 11%. Overall, our operating loss in the first quarter totaled EUR49.1 million compared to an operating loss of EUR143.7 million 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 EUR77.2 million compared to EUR8.4 million 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 USD575 million 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 totaled EUR622 million.

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 EUR320 million. And for the full year 2024, based on current plans, we expect SKYTROFA revenue to be in the range of EUR320 million to EUR330 million -- EUR320 million to EUR340 million at average 2023 exchange rates. We expect total operating expenses, SG&A, and R&D to be approximately EUR600 million. And we expect to be operating cash flow breakeven 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 2 Turner syndrome trial in the fourth quarter of 2024. For TransCon PTH, in the US, our PDUFA date is May 14, 2024. If approved, we plan to launch it as YORVIPATH as quickly as possible thereafter.

Outside the US, with a commercial rollout of YORVIPATH underway in Germany and Austria, we plan to rollout YORVIPATH in our Europe direct and international markets segments throughout 2024 and 2025. For TransCon CNP, 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 COACH 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 2 indications specific dose expansion cohorts from our TransCon IL-2 β/γ and TransCon TLR7/8 Agonist clinical trials. With that, operator, we are now ready to take questions.

(Operator Instructions) Jessica Fye, JP Morgan.

Hey, guys. Good afternoon. Thanks for taking my question. I was hoping you could talk about how April looked for YORVIPATH in Europe. And maybe elaborate a little more on some of those 1Q dynamics you mentioned for SKYTROFA? Thank you.

Yeah, so related to the Q1 dynamics, what we saw -- for SKYTROFA, I'll take the second part first, what we saw would be, I would describe as a typical seasonality that you would see with other maturing launches where coverage updates at the beginning of the year led to insurance reauthorizations, co-pay 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 YORVIPATH in April, we continue to -- as we said, we will come out with an update overall on revenue later this year. But essentially, we're pretty happy with the results of the launch with the primary goals that Jan laid out in his prepared remarks.

So just to give you a little bit of flavor from our own expectations, we launched SKYTROFA in the typical way that -- YORVIPATH 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 was to get a broad prescriber base. The broad prescriber base, we expected them to take two because 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 all of that, some key comfort period where they really get the comfort you take 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 that was some of the most successful launch I have seen. We have not in ourselves imagined that. The number of patients we see, it's around 100, meaning that there's a few physicians that started early in our EAP program of [copper] in Germany. Some of them are up on 20, 30 patients already now because we're already had building up this comfort 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 for. The start where we are looking on the different KPI, it really turned out to what we wanted to see and what we hope for.

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 YORVIPATH.

Thank you.

David Lebowitz, Citi.

Thank you very much for taking my question. If you jump back about a year, when you were in the run-up to YORVIPATH potential approval before the regulatory setbacks. Could you juxtapose your -- 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?

I believe we are in a much, much better place. First of all, the awareness of hypoparathyroidism has been really been coming to a much better awareness levels where physician and the patient. Just in the latest effort when if you really want to hear the patient voice whilst the [U2] that is reflecting how the patient explains the unmet medical need that is in hypoparathyroidism to at the FA Advisory Board. And I think that is only one element of it while 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 off from the patients and what I've seen the awareness of this on a much, much better level to date.

And I think this is why when we see our success with the initial part of our launch in Germany, Australia, 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 big gaining of next year and doing '25. We see it already coming in for the international market, mainly to gain patient programs, which are patient driven program, physician driven program. So the general awareness about the unmet medical need in hypoparathyroidism I believe had really come 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 out from US and other places, really describing how basic conventional therapy, basic -- not providing an optimal treatment, if you can call it a treatment, I'm basically saying that it's a balance between treatment for short-term symptoms and to avoid long-term complication, and you can never get that right. So it basic -- we are in a position that the recommendation of PTH 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.

Thanks for taking my question.

Rosemary Li, Cantor.

Hey, sorry. I should've put Li Watsek. I'm Li Watsek from Cantor. So just for TransCon 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 benefits? Thank you.

Is that question related to Europe or US?

Both, if you may?

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 are addressing short-term symptoms that benefit of the feature to have a formalized quality-of-life benefit of having advanced calcium. And more important, the quality-of-life these patients are experiencing, weekly after starting too. 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 that is also long-term complication like, cardiovascular impact, that is classification on a lot of different organs, cataract, and urging, and also [SGV] and will get hypoparathyroidism just dependent on time.

All of this element is being built into our health economic deliberation that we are providing to each single European countries to be quite sure that we're getting the right reimbursement on right prices. So this basic built on the European way of really being responsible in our pricing where we have a healthy economic calculation on how we basic are providing both a benefit to the patient, the benefit to really to the society in helping the patient on a reasonable, accessible of costs.

Okay. Thank you.

Joseph Schwartz, Leerink Partners.

Thanks very much. I was wondering if you could talk about how many physicians in the US have had experience with YORVIPATH in the EAP versus how many you'll be going out to denovo once it's hopefully launch commercially soon? And could you also talk about how many of the physicians who might be targets for using YORVIPATH in their patients? Have you been interacting with for SKYTROFA already?

I think the first question was reflecting Europe plans and the US EAP program. And what it can do, I can give you the general aspect of our EAP program. The general aspect of our US EAP program because it's very different compared to what we had in Europe with now have been stopped after we got our approval and costs through commercials in Germany. Our EAP program in the US is only addressing patient that really aren't PTA-experienced, meaning is that it's 3%, 4%, 5% subset of patients of hypoparathyroidism that basically are electable to come into our EAP program.

That EAP program is a very difficult program for basic large epidemic institution to handle. So because of contracting and the incentive for them is not high because it's not really our [EAP] program for the physicians. So therefore, you will always find a subset of this site, the typical, have a lot of patients in hypoparathyroidism will not really be part of such an EAP program if it will only be more private site, small site. We are extremely enthusiastic about what we have seen with our EAP program and all the treatment benefit that we have observed in our clinical trials, both Phase 2 and Phase 3 has been 100% confirmed what we have seen from the EAP program and the patient experience there.

Okay. So how many new sites will you have to go to then that are -- that treat hypoparathyroidism patients who have not had experience in the EAP or with using SKYTROFA?

SKYTROFA, we don't have any the EAP site at all in this way. SKYTROFA didn't -- ever had any EAP program running. So from that perspective, it's really hard for me to compare this two.

What I meant commercial SKYTROFA, or the EAP for YORVIPATH. I'm just trying to get a sense of the footprint now. How much overlap there is with -- between the two products that you'll have shortly?

We will basically say that is not at [that] or left indirectly to the physician prescription base because it's typical more specialist sites that take care of both sides. One of them is, for example, in pediatric first indication, the other one is in ethyl indication. So when you look at the overlap of that, there's not a large overlap. And Joe, I'm not seeing any data of where I would indicate support and same dose as these overlap. Whether it's a great overlap, it's in 80%, 90% of the conversed [platform] that basic 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 (inaudible) made exactly the right decision to, has the most prescribing physician being covered as fast as possible.

Thank you.

Gavin Clark-Gartner, Evercore ISI.

Hey, congrats on the progress and thanks for taking the question. I'm just wondering on out of the 100 of the YORVIPATH patients in Germany, how many of these were previously on [navbar] And was this in line with your expectations heading in?

I will 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 aren't looking on the patients and saying, we have so many on patients that not have been in treatment that we also are focused on taking many new patients into the treatment received. And I have to say that was one of the surprises, at least for me where I will believe that it was initially just a change of navbar patient, but we have seen many more pain patient that we actually expected.

Great. Thank you.

Kelly Shi, Jefferies. Thank you for taking my questions.

SKYTROFA, could you provide your perspective into the competitive landscape, given that two other long-acting growth hormone commercial products out there, and one of them, if we check on the scripts are actually approaching SKYTROFA on both TRx and NRx. And also, I am curious like whether your launch strategy remains the same.

So one of the complication, at least I always have is to utilizing 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 of where you get the information from. So often that perspective, you can not really compare the absolute level. You can not compare it also because want to 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 and 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's no doubt when we look at 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 decline, which you typically would see in the growth hormone.

Thank you.

Derek Archila, Wells Fargo.

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 EUR600 million guidance. So just any color there and how we should be thinking about that? Thanks.

I think Scott is so happy today because he really getting a good question.

Yeah, Derek, thanks for the question on. We've been pretty -- we're pretty proud of our ability to basically reduce OpEx pretty significantly while growing doubling revenue. It's not a bad thought, but as of now, our guidance remains EUR600 million OpEx for the full year based on current plans.

Paul Choi, Goldman Sachs.

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 CNP 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 TransCon CNP either (technical difficulty) as new patient starts are due are 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 a launch? And then could you maybe comment on what your sort of post approval requirements might potentially look like for TransCon CNP? The pending and approval down the road from the FDA. Thank you.

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 co-morbidities. In short, it's not a disease, but really to have this sign of co-morbidities is really providing really the impact on having achondroplasia. 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 do 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 difficult to change that. If we have been the first, it would not have been linear growth, it will be addressing co-morbidity as a 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 keep get feelings about what it means for the patient is when we look at the quality-of-life questions, that we have giving to both patients and caregivers, how they really are just seeing the benefit of our treatment?

And we also see that in our retentions. When we see the retention and our recruitment. We recruit our people who have tried in less than four months. On site, which has access to store type of access to other treatment, it got recruited in four months. I've never seen that before. And this is because the physicians could talk to the parents about the benefit, they have seen basic on providing our TransCon CNP product to them. Related to commitments afterwards, we have no comments or anything we have received from regulatory agencies in all the different places we have been and discuss the pathway for regulatory approval to any kind of commitment.

Thank you.

Vikram Purohit, Morgan Stanley.

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

We are extremely as excited about this lead candidate that we were full when we came out. There's no doubt that once the treatment regime is the a way to go. And we're building up the same fundamentals that we built up all our pipeline. I'd call it a pipeline because we basically are not take a bit of target engagement risk that you would typically have when you make highly differentiated product because we're building through the TransCon technology arm and semaglutide molecule that basically had 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 basic are doing the best value proposition of this compound and this era of metabolic diseases because we talked about metabolic diseases. And we will keep you updated as soon as we someway have made a decision what way we're going.

Thank you.

Yaron Werber, TD Cowen.

Hey, guys, this is Joy, on for Yaron. Thanks for taking our question. In the US, can you talk about your planned launch strategy for TransCon PTH in terms of the initial target population and prescriber base? And how much of that will mirror your strategy in Germany where you're initially targeting roughly one-third of the total population? Thank you.

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 the, what I call the playbook.

So when I come to the playbook of US that is always headed by Camilla as our head of our global commercial operation. She's coordinating everything from US, coordinating into international market leading into EU direct. Every place to be sure that we're building up the right strategic approach for basically have the optimal commercial launch on a global basis.

We will be here, and we basic has seen really the huge interest. I can say we have the first commercial US product or person on commercial product for YORVIPATH. It's a person living in US that took the consequence of the delay in the US to basically fly to Germany and get the product out there. So we know it. We see it. We know YORVIPATH's making really shoes different for the patient, really get them their life back. And we really have seen how patients also see this benefit.

Thank you.

Leland Gershell, Oppenheimer.

Hey, good afternoon. Thanks for taking our questions. Two from us. First, Jan, if you could just comment on the plans for rolling out YORVIPATH in Great Britain. Following the approval, is that is that something you're doing immediately, or will there be any delay?

And secondly, in the past, you'd mentioned hypochondroplasia is a indication that you were -- you've decided to not pursue. Just wondering if the outlook might change following the data from the [Ascendis] Phase 3 you'll be seeing later this year and or any information that may be coming out from the companies that are pursuing hypochondroplasia? Thank you.

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 have the UK approval now separated from an EU approval like other countries that we also gained approval in now. We continue filing everywhere from the different countries, Spain, for example, Australia, everywhere, where we also need an independent approval. We will then come in and go in and do what we call it's typical and filing of [entouche] 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 this specific country about the reimbursing price, it 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 near term, we have medical affairs, we have market assess teams, all of that is basic already being established. And they are ready to be coming out and doing a full commercial launch that, for example, which we have done in Germany and Austria.

In the meantime, physician patient can go to a named patient program where we can get fully reimbursed patient with a bid on an inpatient program or similar structures in lot of different countries in this timeframe until you will be fully commercial. Your basic end 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 have also built some big implement in the international market, but we will not be the M&A holder in the international market. It would typical be handed to our sales and distribution agents.

For the other thing about hypochondroplasia, it's really, really is an interesting aspect for us because we are the only company that happens to cornerstone growth. Growth disorder is about 30, 40 different diseases. We are the only company that have a once-weekly growth hormone at once-weekly CNP molecule with really the best-in-class properties for both of these two mode of action.

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 hypochondroplasia but address the other -- including the 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.

Sushila Hernandez, Kempen.

Yes, thank you for taking my question. Could you elaborate on SKYTROFA in becoming a blockbuster in the US alone? What needs to happen? What are the key drivers? Thank you.

The key driver is to continue what we're doing. This is exactly what we're doing. We are doing -- continue showing get the physician, the patient to really getting the treatment benefit of SKYTROFA as the best-in-class product opportunity inside the growth disorder. We will be helped with a lot of different things. The consolidation of the daily Growth Hormone market. We saw one of the major player is not stopping. We see all of the major players with daily Growth Hormone stopping.

So consolidation of the daily Growth Hormone where potentially one or two player will be left in more or less in a cast segment, it's 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 [alterna] 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 one guy that really -- that may give the limit for this product.

Thank you.

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.