Ascendis Pharma - Q1 2024

Transcript

Operator (participant)

Good day, and welcome to the Q1 2024 Ascendis Pharma Earnings Conference Call. At this time, all participants are in listen-only mode. After the speaker's presentation, there'll be a question and answer session. To ask a question, please press star one one. 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 turn the call over to Tim Lee, Senior Director, Investor Relations, Ascendis Pharma. Please go ahead.

Timothy Lee (Senior Director, Investor Relations)

Thank you, operator, and thank you everyone for joining our Q1 2024 financial results conference call. I'm Tim Lee, Senior Director of Investor Relations at Ascendis Pharma. Joining me on the call today are Jan Møller 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 Kelly, 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 statements may include, but are not limited to, statements regarding our commercialization and continued development of SKYTROFA for the U.S. and European markets, as well as certain financial expectations for 2024, our commercialization and development of Yorvipath 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 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 can cause actual results to differ materially, please see our forward-looking statement section in today's press release and the risk factor section of our most recent annual report on Form 20-F, filed with the SEC on February 7th, 2024. TransCon Growth Hormone, or TransCon HGH, is approved in the US by FDA, and the EU has received MAA authorization from the European Commission for the treatment of pediatric growth hormone deficiency.

The European Commission and the United Kingdom Medicines and Healthcare Product Regulatory Agency have 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 Q1 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 to Jan.

Jan Mikkelsen (President and CEO)

Thank you, Tim. Good afternoon, everyone. Ascendis is applying its TransCon technology platform to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients' lives. To approved 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 break even on a quarterly basis by the end of 2024. Our long-term commitment in the last 3, 4 years to build out profitable and robust supply chains, and the decision we took in 2023 to streamline the company have made us a leaner and more efficient organization. You can see the progress towards operating cash flow break even later this year without compromising our development and commercialization progress.

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 TransCon platform for future innovation. We have seen validation of our commercialization approach with the ongoing success of SKYTROFA in the U.S. and are seeing it again with successful launch of Yorvipath in Germany and Austria. In the U.S., the PDUFA date for TransCon hGH is coming up in less than two weeks on May fourteenth. If approved, we expect to be ready to launch in the U.S. soon thereafter in the Q3.

Now, let me give an update on each of our programs. When we launched SKYTROFA in the U.S. about two years ago, we had two goals. One, to make SKYTROFA the leading product in there. 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 SKYTROFA, the value leader in the U.S. We have a reliable supply chain, ensuring that every patient can benefit from SKYTROFA once they have approval from their insurance company. We estimate that SKYTROFA penetration in the U.S. pediatric growth hormone deficiency patient population grow to about 17% at the end of the Q1. We are proud to observe SKYTROFA extend its market value leadership as the only growth hormone product to grow in value in the Q1 of 2024, based on reported results.

We believe SKYTROFA will expand the U.S. growth market, with the potential to become a blockbuster in the U.S. alone. SKYTROFA sales this quarter more than doubled compared to the Q1 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 SKYTROFA results, we continue to expect the full year 2024 SKYTROFA revenue will be between EUR 320 million and EUR 340 million, representing year-to-year growth of 80%-90%. We expect SKYTROFA sales to continue to grow through further penetration in the pediatric growth hormone setting. To further solidify growth, we're also pursuing our first label expansion in adult growth hormone deficiency, for which we plan to submit a supplemental BLA to the FDA in the Q3 of this year.

In addition, we expect top line data from our phase 2 trial in Turner syndrome in the Q4 of 2024. Now, turning to TransCon PTH. In Europe, Yorvipath was launched in Germany and Austria at the end of January, with the early launch objective of building physician experience with Yorvipath. Initial physician feedback from Yorvipath has been positive. Just 8 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 comfort with the treatment benefit of Yorvipath in the first patient, we expect physicians to take more and more of the patient, treatment.

As we expand our geographic reach of 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, pre-launch preparations are underway, including the expansion of the US field infrastructure, while we are waiting FDA's decision. Moving now to TransCon CNP. Our value proposition with TransCon CNP is simple. It's to establish a treatment for patients of all ages with achondroplasia. Our ambition is to address the significant comorbidities associated with achondroplasia that impact health and quality of life, as well as linear growth. Later this year, in the Q3, 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 treating achondroplasia with TransCon CNP 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 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 third, Q4 this year. This is our integrated approach to address achondroplasia with multiple trials that are all building towards one goal, to treat the disease. 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 gamma and TransCon TLR7/8 agonist have showed promising clinical activity as monotherapy and in combination treatment with pembro 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 have progressed from checkpoint inhibitors, using the combination of TransCon IL-2 beta gamma and TransCon TLR agonist in these patients. Later this year, in the third, Q4, we expect data readouts in several well-defined patient populations from our TransCon IL-2 beta gamma and TransCon TLR7/8 agonist 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 products, 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.

Scott Smith (EVP and CFO)

Thank you. In Q1, we demonstrated significant financial progress for our goal of becoming operating cash flow break even 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 Q1 of 2024 was EUR 65 million, compared to EUR 31.6 million reported in the Q1 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 EUR 0.8 million.

On a sequential basis, Q1 SKYTROFA revenue increased 1% compared to the Q4 of 2023. Strong mid-teens % demand volume growth was offset primarily by seasonal channel inventory, co-pay resets, insurance reauthorizations, and a modest negative currency impact of EUR 0.6 million. Q1 was in line with our high internal expectations, and with seasonal headwinds behind and the steady pace of new patient adds expected to persist, we continue to expect full year SKYTROFA revenue to be in the range of EUR 320 million-EUR 340 million at average 2023 exchange rates. Shifting to TransCon PTH, Yorvipath contributed for the first time this quarter with revenue of EUR 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 SKYTROFA, where specialty pharmacies hold channel inventory. Closing out the top line, total revenue for the Q1 was EUR 95.9 million, including EUR 24.8 million of non-cash license revenue, recognized in relation to the formation of Eyconis, and EUR 3 million of service revenue related to Eyconis, which is offset in operating expenses. Turning to expenses, R&D costs in the Q1 totaled EUR 70.7 million, compared to EUR 106.1 million during the Q1 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 EUR 66.8 million, essentially flat compared to EUR 66.5 million during the Q1 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 EUR 137.5 million for the Q1, a 20% decrease compared to the EUR 172.7 million during the Q1 of 2023. Sequentially, operating expenses declined 11%.

Overall, our operating loss in the Q1 totaled EUR 49.1 million, compared to an operating loss of EUR 143.7 million during the Q1 of 2023, as a result of increased revenue and lower operating expenses. Sequentially, operating loss increased 34%. Finance expense in the quarter was EUR 77.2 million, compared to EUR 8.4 million expense in the Q4 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 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 EUR 622 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 Q1 with cash and cash equivalents totaling EUR 320 million.

For the full year 2024, based on current plans, we expect SKYTROFA revenue to be in the range of EUR 320 million to EUR 330 million, sorry, to EUR 340 million at average 2023 exchange rates. We expect total operating expenses, SG&A and R&D, to be approximately EUR 600 million, 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 Q3 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 Q4 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 the 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 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 Q4 of 2024, and also report week 26 top-line data from the COACH trial in combination with TransCon Growth Hormone, also in the Q4 of 2024.

Within our oncology therapeutic area, during the Q4 of 2024, we plan to provide a clinical update from the Phase II indication-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 (participant)

Thank you. If you'd like to ask a question, please press star one one. If your question has been answered and you'd like to remove yourself from the queue, please press star one one 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.

Jessica Fye (Managing Director, Equity Research Analyst - Biotechnology)

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.

Jan Mikkelsen (President and CEO)

Should you start, Scott?

Scott Smith (EVP and CFO)

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 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 Yorvipath in April, you know, we continue to see, as we said, you know, we'll come out with an update overall on revenue later this year. But, you know, essentially, we're pretty happy with the results of the launch, with the primary goals that Jan laid out in his prepared remarks.

Jan Mikkelsen (President and CEO)

So, Jess, just to give you a little bit of flavor from our own expectations, we launched SKYTROFA in the typical way that... Sorry, 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, want to get a broad prescriber base. The broad prescriber base, we expected them to take typical 1-2 patient 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 get 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 physician already in 8 weeks to make a prescription, I believe there was some of the most successful launch I have seen.

We have not in ourselves imagined that. The number of patients we're seeing is around 100, meaning is that there is a few physicians that started early in our ERP program or COP program in Germany. Some of them are up on 20, 30 patients already now because they 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 hoped for, the start where we looking on the different KPI, it really turned out to what we wanted to see and what we hoped for.

Scott Smith (EVP and CFO)

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.

Operator (participant)

Thank you. Our next question comes from David Lebowitz with Citi. Your line is open.

David Lebowitz (Senior Research Analyst - Biotechnology)

Well, thank you very much for taking my question. If you jump back about a year, when 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 looked like then, and how you see things similarly or differently?

Jan Mikkelsen (President and CEO)

I believe we are in a much, much better place. First of all, the awareness of hypoparathyroidism has really been coming to a much better awareness level, where 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 hypoparathyroidism to 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. But I think the awareness of is on a much, much better level today.

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, and during 2025. We see it already coming in for the international market, mainly to named patient programs, which are patient-driven program, physician-driven program. So the general awareness about the unmet medical need in hypoparathyroidism, I believe, has 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 it is the new guidelines that came out from U.S. and other places, really describing how basic conventional therapy, basic, not providing an optimal 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 basically, we 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.

David Lebowitz (Senior Research Analyst - Biotechnology)

Thanks for taking my question.

Operator (participant)

Thank you. Our next question comes from Rosemary Li with Cantor. Your line is open.

Rosemary Li (Research Analyst)

Hey, sorry, I should have put Li Watsek. I'm on for Li Watsek from Cantor. So just, for TransCon PTH, in terms of payer access and formulary placement, have you had any pushback from payers on showing clinical benefits, such as hospitalization rate or kidney benefit? Thank you.

Jan Mikkelsen (President and CEO)

Is that question related to Europe or U.S.?

Rosemary Li (Research Analyst)

Both, if you may.

Jan Mikkelsen (President and CEO)

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. There is calcification in a lot of different organs, cataract and other things you're also achieving and will get with hypoparathyroidism, 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 basic built on the European way of really being responsible in our pricing, where we have a health economic calculation on how we basic are providing both the benefit to the patient, the benefit to, really, to the society in helping the patient on a reasonable, accessible cost.

Rosemary Li (Research Analyst)

Okay, thank you.

Operator (participant)

Thank you. Our next question comes from Joseph Schwartz with Leerink Partners. Your line is open.

Joseph Schwartz (Senior Research Analyst)

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 de novo once it's hopefully launched commercially soon? And could you also talk about how many of the physicians who might be targets for using your Yorvipath in their patients have you've been interacting with for SKYTROFA already?

Jan Mikkelsen (President and CEO)

I think the first question was regarding Yorvipath and the US EAP program. What I 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, 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 EAP program in US is only addressing patients that really are PTH experienced. Meaning is that it's a 3%-5% subset of patients of hypopara, that basically are eligible to come into our EAP program. The EAP program is a very difficult program for basically 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 physician.

So therefore, you will always find a subset of this site that typically have a lot of patients in hypopar will not really be part of such an EAP program. It will only be more private sites, 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 trial, both phase 2 and phase 3, has been 100% confirmed what we have seen out from the EAP program and the patient experience there.

Joseph Schwartz (Senior Research Analyst)

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 SKYTROFA?

Jan Mikkelsen (President and CEO)

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

Joseph Schwartz (Senior Research Analyst)

Well, 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, you know, between the two products that you'll have shortly.

Jan Mikkelsen (President and CEO)

You will basically say there is not a large overlap in directly to the decision prescription phase, because it's typical more specialist 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 it's- there's not a large overlap. And Joe, I can... I'm not seeing any data where I will indicate and support and saying there's a big overlap. Where there is a great overlap, it's 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-made exactly the right physician to have the most prescribing physician being covered as fast as possible.

Joseph Schwartz (Senior Research Analyst)

Thank you. Thank you.

Operator (participant)

Thank you. Our next question comes from Gavin Clark-Gartner with Evercore ISI. Your line is open.

Gavin Clark-Gartner (Managing Director, Biotechnology Equity Research)

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

Jan Mikkelsen (President and CEO)

I will say, surprisingly, we see many more naive patients than we actually have thought we would see. So this is one of the surprises we have seen, that the physicians basically are looking at the patients and saying, "We have so many patients that have not been in treatment, that we also are focused on taking the naive patient into the treatment regimen." 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 Natpara patient, but we have seen many more naive patients than we actually expected.

Gavin Clark-Gartner (Managing Director, Biotechnology Equity Research)

Great. Thank you.

Operator (participant)

Kelly Shi with Jefferies, your line is open.

Kelly Shi (SVP, Senior Equity Analyst)

... Thank you for taking my questions. On SKYTROFA, 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 scripts, actually approaching schedule for on both TRX and the RX. And also, like, how you- curious, like, whether your launch strategy remain the same?

Jan Mikkelsen (President and CEO)

So one of the complications, at least I always have, is 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 providers. So you are only sampling a small amount of the prescribing base of where you get the information from. So out 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 providing you that. So therefore, if you really want to get a 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. There is no doubt when we look on that. For the growth hormone market-

Kelly Shi (SVP, Senior Equity Analyst)

Thank you very-

Jan Mikkelsen (President and CEO)

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 Q1.

Kelly Shi (SVP, Senior Equity Analyst)

Thank you.

Operator (participant)

Thank you. Our next question comes from Derek Archila with Wells Fargo. Your line is open.

Derek Archila (Managing Director, Co-Head of Therapeutics Research)

Hey, yeah, thanks for taking the question, and 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 EUR 600 million guidance. So just, you know, any color there on how we should be thinking about that? Thanks.

Jan Mikkelsen (President and CEO)

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

Scott Smith (EVP and CFO)

Yeah, Derek, thanks, thanks for the question. We've been pretty proud of, 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 EUR 600 million OpEx for the full year, based on current plans.

Operator (participant)

Thank you. Our next question comes from Paul Choi with Goldman Sachs. Your line is open.

Paul Choi (Biotechnology Analyst)

Good afternoon, and thank you for taking my question. Also let me offer my congratulations on the progress. I want to turn maybe to TransCon 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 as new patient starts, or do you possibly anticipate switches from Voxzogo, 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? Then could you maybe comment on what your, you know, sort of post-approval requirements might potentially look like for TransCon CNP, you know, pending an approval down the road from the FDA? Thank you.

Jan Mikkelsen (President and CEO)

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 comorbidities is really providing really the impact on having achondroplasia. And this is why we have so much strong focus, really to address the comorbidity, and also sure, to address also linear growth. But the key objective of our program is to do address the comorbidities. We cannot avoid a primary efficacy endpoint is linear growth, because it has been established from regulatory agencies both in U.S. 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 comorbidity 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 on the quality of life questions that we have given to both patient and caregivers... how they really are adjusting the benefit of our treatment. And we also see that in our retentions. When we see the retention and our recruitment, we recruit our pivotal trial in less than four months.

On site, which have access to vosoritide, have access to other treatment, it got recruited in four months. I've never seen that before. And this is because the physician could talk to the parents about the benefit they have seen based on providing our TransCon CNP 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.

Paul Choi (Biotechnology Analyst)

Thank you.

Operator (participant)

Thank you. Our next question comes from Vikram Purohit with Morgan Stanley. Your line is open.

Vikram Purohit (Executive Director)

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 had 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.

Jan Mikkelsen (President and CEO)

We are extremely excited about this lead candidate that we were for when we came out. There's no doubt the monthly treatment regimen is the way to go, and we're building up the same 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 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 basically are doing the best value proposition of this compound and this era of metabolic diseases, because we're talking about metabolic diseases. We will keep you updated as soon as we someday have made a decision what way we're going.

Operator (participant)

Thank you. Our next question comes from Yaron Werber with TD Cowen. Your line is open.

Joyce Chen (Business Insights Consultant)

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 TransCon 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.

Jan Mikkelsen (President and CEO)

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 complete 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 U.S., there is always heading up Camilla, that is our head of our global commercial operation.

She's coordinating everything from U.S., coordinating into international market, correlating into EU, direct every place to be sure that we're building up the right strategic approach for basic have the optimal commercial launch on a global base. We will be here, and we basically have seen really the huge interest. I can say we have the first commercial U.S. product or, or person on commercial product for Europe. It's a person living in U.S. that took the consequence on the delay in the U.S. to basically fly to Germany and get the product out there. So we, we know it, we see it, we know Europe is making really, huge difference for the patient, really get them their life back, and we really have seen how patients also see this benefit.

Joyce Chen (Business Insights Consultant)

Thank you.

Operator (participant)

Thank you. Our next question comes from Leland Gershell with Oppenheimer. Your line is open.

Leland Gershell (Managing Director)

Hey, good afternoon. Thanks for taking our questions. Two from us. First, Jan, if you could just comment on the plan for rolling out Yorvipath 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 hypochondroplasia as an indication that you were- you've decided to not pursue. Just wondering if that outlook might change following the data from the achondro phase 3 that 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.

Jan Mikkelsen (President and CEO)

Yeah. UK is part of our EU 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 are now separated from an EU approval, like other countries that we are also getting approval in now, we have continued 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 filing of a dossier that really are describing the health economic impact, as we have done in many EU direct countries, but we are also doing in other countries.

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 access teams. All that is basically already been established, and they are ready to be coming out and doing a full commercial launch, launch that, for example, which we have done in Germany and Austria. In the meantime, a physician, patient can go to a Named Patient Program, where we can get fully reimbursed patient with a built on a Named Patient Program or similar structures in a lot of different countries.

In this time frame, 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 Europe directly, which we also will someday implement in the international market, but we will not be the MAA holder in the international market. It will typically 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 have the two cornerstone in growth disorders. Growth disorder is about 30, 40 different diseases. We are the only company that have a once-weekly growth hormone, a once-weekly CNP molecule, with really the best-in-class properties for both of these two modes 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 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 CNP, 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 cornerstone 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.

Leland Gershell (Managing Director)

Thanks.

Operator (participant)

Thank you. And our last question comes from Sushila Hernandez with Kempen. Your line is open.

Sushila Hernandez (Equity Analyst)

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

Jan Mikkelsen (President and CEO)

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 SKYTROFA as the best-in-class product or opportunity inside the growth disorder. We will 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 with the long-acting, they realize there's only one choice. And I don't need to say that name, because the sky's the limit for this product.

Sushila Hernandez (Equity Analyst)

Thank you.

Operator (participant)

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.