Ascendis Pharma - Q2 2024

Transcript

Operator (participant)

Ladies and gentlemen, this is the operator. Again, thank you for your patience, and we do apologize for the delay. We're having a bit of technical difficulties. We will start the conference shortly. Again, thank you for your patience, everyone. Hello, thank you for standing by. Welcome to Ascendis Pharma's second quarter 2024 earnings conference call. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question-and-answer session. To ask the question during this session, you will need to press star one one on your telephone. You would then hear an automated message advising your hand is raised. To withdraw your question, please press star one one again. We ask that you limit yourself to one question and one follow-up.

I would now like to turn the call over to Tim Lee, Senior Director of Investor Relations at Ascendis Pharma. Sir, you may begin.

Tim Lee (Senior Director of Investor Relations)

Thank you, operator, and thank you everyone for joining our second quarter 2024 financial results conference call. We apologize for the delay, due to some technical issues that, we had here. I'm 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 Singhal, Executive Vice President and Head of Clinical Development Oncology, Camilla Harder Hartvig, Executive Vice President and Global Chief Commercial Officer, 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 statement may include, but are not limited to, statements regarding our commercialization and continued development of Skytrofa and Yorvipath, the U.S. and European markets, as well as certain financial expectations, 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 for expectations regarding the timing and the results of regulatory decisions, and our exploration of market opportunities and therapeutic areas outside of endocrinology rare disease. These statements are based on information that is available to us as of today. Actual results may differ and 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 Factors 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. TransCon PTH is approved in the US by the FDA for the treatment of hypoparathyroidism in adults, and the European Commission and the United Kingdom's Medicines and Healthcare Products Regulatory Agency have granted marketing authorization for TransCon PTH as a replacement therapy indicated for the treatment of adults with chronic hypoparathyroidism.

Otherwise, please note that our product candidates are investigational and not approved for commercial use. As investigational products, the safety and effectiveness of product candidates have not been reviewed or approved by any regulatory agency. None of the statements during this conference call regarding our product candidates shall be viewed as promotional. On the call today, we'll discuss our second quarter 2024 financial results, and we'll provide further business updates. Following some prepared remarks, we will then open up the call for questions. With that, let me turn it over to Jan.

Jan Mikkelsen (CEO)

Thanks, Tim. Good afternoon, everyone. With the recent US approval of Yorvipath as the first and the only FDA-approved treatment of hypoparathyroidism in adults, Ascendis has successfully obtained approvals for two out of three endocrinology rare disease product candidates in two major markets, the US and EU... and with pivotal data from our third product candidate, TransCon CNP in achondroplasia, expected in the coming weeks, we are nearing our vision to achieve approval of all three product candidates by the end of 2025. Our algorithm for product innovation, combined with our TransCon technology, have enabled us to bring new, highly differentiated product candidates to the clinical development process faster and with a higher success rate compared to traditional drug development.

We intend to continue to develop new product candidates in endocrine disease and large therapeutic areas such as oncology, obesity, metabolic disease, and cardiovascular, with best-in-class potential to make a meaningful difference for patients across the globe, based on our strong scientific focus. Let us begin with Yorvipath. In the US alone, an estimated 70 to 90,000 patients are living with hypoparathyroidism. For those who haven't seen it, please take time to watch the patient-arranged FDA hearing on the Hypoparathyroidism-Associated website, providing an in-depth understanding of the serious consequence of having this disease. With the FDA approval of Yorvipath, physicians and adult patients in the United States can now look forward to having a treatment option for treatment of hypoparathyroidism. We are preparing for Yorvipath launch in the US, leveraging our established commercial infrastructure and expertise.

We are expanding our dedicated team of sales reps and field medical personnel, who will engage with around 6000 physicians who treat eighty percent of patients with hypoparathyroidism in the U.S. Other key launch initiatives are underway, including the roll-out of our patient support programs designed to support access to Yorvipath. For example, eligible patients on commercial insurance will pay as little as $5 a month for the Yorvipath prescription. In addition, following our goal to take care of all patients with this disease, we will also introduce a patient assistance program. We have also started engaging U.S. payers and PBMs. We expect product availability in the U.S. in the first quarter of 2025 or sooner if it's possible.

Consistent with premier responsible pricing, we will launch with a list price corresponding to $285,000 annual per patient, reflecting the value of Yorvipath to the U.S. healthcare system. In the U.S., there are around 140 patients currently active in the expanded access program, and about 50 more patients in the open label extension of our clinical studies. Physicians will begin transferring these patients over to commercial product as soon as it's available. In Europe, the number of patients and prescribing physicians initiating Yorvipath continues to increase, and we see a good mix of PTH-experienced and new, naive patients. This was the first full quarter of commercial launch in Germany and Austria, where sales momentum continues to build. We now have more than 250 patients on treatment and an estimated 125 prescribers in these two markets.

Yorvipath patient retention rate is extremely strong and currently around 98%. As physicians gain more experience with Yorvipath, we expect them to bring more patients on therapy, including those finishing remaining supplies of Natpar. The interest for serving patients under named patient programs prior to full commercial launch is increasing. We now have patients in this program in more than 10 countries and expect more by the end of the year. Moving to Skytrofa, we are proud to have more than 11,000 patients prescribed Skytrofa in the first three years since launch, and to have achieved market value leadership while expanding the overall growth hormone market. Key component of our strategy to make Skytrofa a blockbuster product in the US include simplifying broadened market access for both treatment-naive or switch patients, as well as expanding our label.

In the first half of the year, the reset to broader market access for Skytrofa was largely completed. While this broader access to Skytrofa will support long-term demand, in the short term, it negatively impacted our first half net revenue. Scott will share more details. With our market access transition largely completed, Skytrofa is now positioned as a premium product, with a net value per patient of around three times compared to daily growth hormone. We are now focused on using our new market access coverage to drive further demand, continue to expand the overall growth hormone market, and are aiming to reach blockbuster status for Skytrofa in the U.S. alone. Finally, to build on our market leadership position, we plan to submit and supplement BLA in adult growth hormone deficiency to the FDA in the third quarter of this year, our first Skytrofa label expansion.

We also expect top line data from our Phase II trial in Turner Syndrome in the fourth quarter of 2024. Switching to TransCon CNP. I have always been extremely excited about our program in achondroplasia, and much more now as we are approaching the result from our pivotal trial in the coming weeks. We have consistently in our messaging over the past eight years since we announced our product candidate, that our aim is to develop a treatment that addresses both linear growth and the comorbidities that affect health and quality of life for people living with achondroplasia. Earlier this year, you saw a comprehensive result for our Phase II ACCOMPLISH trial, demonstrating that our once-weekly TransCon CNP increased annualized growth velocity similar to vosoritide about 5.6 centimeters after 12 months of treatment.

In addition, for the first time ever for a product in the setting of achondroplasia, we also demonstrated that compared to placebo, TransCon CNP improved quality of life associated with the physical function of well-being in children with achondroplasia, with a favorable side effect and tolerability profile. Now, we hope that we can replicate these results with more patients in our ApproaCH Trial, and we are expecting top-line data in the next few weeks, one quarter earlier than guided. This trial enrolled 84 children, aged 2 to 11, with achondroplasia. The mean age was 5.7 years, similar to our Phase II trial. We also continue enrolling in the Coach Trial, a phase II trial of TransCon CNP in combination with TransCon growth hormone, Skytrofa, designed to show that adding Skytrofa to TransCon CNP could provide catch-up growth for patients who start CNP treatment late.

We expect to complete enrollment in this combination trial during the fourth quarter of 2024, with top-line 2026 data expected in the second quarter of 2025. Turning now to oncology. We continue advancing three Phase II trials with multiple indication-specific cohort to study the best-in-class potential of our two product candidates, TransCon IL-2 beta/gamma and TransCon TLR7/8 agonist, in different combination scenarios. We plan to present initial results from our TransCon IL-2 beta/gamma in combination with chemotherapy in platinum-resistant ovarian cancer from the IL-Believe trial later this month at the ESMO conference in Barcelona. I'm pleased how this program are progressing. In closing, for Ascendis, it's all about the patients. Patients tell us the U.S. approval of Yorvipath is transformative for them. We hear from parents that Skytrofa has changed the life of children and parents both.

With data expected in the next weeks for TransCon CNP, it is our goal that we are able to show that we also can transform the lives of people living with achondroplasia. With our ongoing progress in our oncology program and exploration of other areas of innovation in large market opportunities such as obesity, we continue to position Ascendis for sustainable growth with an expanded pipeline and transform more transformative TransCon product candidate. I'll now turn it over to Scott for a financial update.

Scott Smith (EVP and CFO)

Thanks, Jan. Skytrofa volume more than doubled in the second quarter of 2024 compared to the second quarter last year, while reported revenue was 26.2 million EUR, compared to 35.9 million EUR, reported in the second quarter of 2023, a decrease of 27% year-over-year. An increase in Skytrofa volume was offset by higher sales deductions for Q2, and an adjustment to Q1 2024 sales deduction accruals, as well as for periods in 2023. These adjustments reflect the reset of broader market access to support continued growth of Skytrofa as the market value leader and a potential blockbuster in the US alone.

In total, Q2 2024 reported revenue was reduced by a true-up of EUR 27.1 million, of which EUR 19.5 million was attributable to the first quarter 2024 sales, and EUR 7.6 million to sales prior to January 1, 2024. Skytrofa revenue for the first half of 2024 totaled EUR 91.2 million, a 35% year-over-year increase compared to EUR 67.4 million during the same period in 2023. First half 2024, Skytrofa volume more than doubled, but was partially offset by an accrual true-up of EUR 7.6 million, which was attributable to periods prior to January 1, 2024. With the advent of broader market access, we believe the overall growth hormone market will continue to grow the number of patients treated.

We estimate that by the end of Q2 2024, Skytrofa penetration in the U.S. pediatric growth hormone deficiency-treated patient population was 18%, or a little under 10% of the overall treated growth hormone market. Still, with our single indication for pediatric GHD, leaving lots of room for further growth and market expansion. Based on year-to-date results and current trends, we now expect full year 2024 Skytrofa revenue to be in the range of EUR 220 million-EUR 240 million. Shifting to TransCon PTH, second quarter Yorvipath revenue of EUR 5.2 million reflected the first full quarter of commercial revenue in Germany and Austria, as well as initial revenue in other markets, with Yorvipath revenue continuing to increase from the EUR 1.5 million in Q1, driven by growing patient and physician demand.

Closing out the top line, total revenue for the second quarter was EUR 36 million, including EUR 4.6 million, tied to rendering of services and license revenue. Turning to expenses, R&D costs in the second quarter of 2024 totaled EUR 83.5 million, compared to EUR 105 million during the second quarter of 2023. The 21% decline was largely tied to lower external development costs for TransCon TLR7/8 Agonist and lower costs for TransCon PTH, as well as the Eyconis spin-off. SG&A expenses in the quarter totaled EUR 74.3 million, compared to EUR 70.3 million during the second quarter of 2023. The increase was primarily due to higher employee costs, including the impact from global commercial expansion.

Total operating expenses were EUR 157.8 million for the second quarter, a 10% decrease compared to EUR 175.3 million during the second quarter of 2023. Total operating expenses for the first half of 2024 were EUR 295 million. Net finance income in the quarter was EUR 29.4 million, compared to EUR 26.4 million in the second quarter of last year. As a reminder, the net finance line can fluctuate quarter to quarter, driven in part by non-cash items related to our outstanding convertible notes. We ended the second quarter with cash, cash equivalents, and marketable securities totaling EUR 259 million, compared to EUR 399 million as of December thirty-one, 2023.

Finally, earlier today, and subsequent to June 30, 2024, therefore, not included in the reported cash balance, we announced that we have entered into a new capped synthetic royalty funding agreement with Royalty Pharma for $150 million, in exchange for a 3% royalty on net sales of Yorvipath within the United States. The royalty payments are capped at 1.65x the purchase price if fully paid prior to December 31, 2029, or 2x thereafter. Further details are disclosed in a separate 6-K, also filed today. Looking ahead for the full year 2024, based on current plans, we expect Skytrofa revenue to be in the range of €220 million-€240 million.

Total operating expenses, which include SG&A and R&D, to be approximately EUR 600 million, including Yorvipath-related launch activities in the U.S. Pending launch timing of Yorvipath in the U.S., we currently expect to achieve operating cash flow breakeven on a quarterly basis in 2024 or 2025. Before we turn it over to the operator for Q&A, I want to reiterate, with database locked and the top-line results for our pivotal ApproaCH Trial expected in the coming weeks, we plan to institute a quiet period starting Thursday, and unfortunately, we will not be able to participate in upcoming investor conferences. With that, operator, we are now ready to take questions.

Operator (participant)

Thank you. Ladies and gentlemen, as a reminder to ask the question, please press star one one on your telephone and then wait to hear your name announced. To withdraw your question, please press star one one again, and please limit yourself to one question and one follow-up. Please stand by while we compile the Q&A roster. Our first question comes from the line of Jessica Fye with JP Morgan. Your line is open.

Jessica Fye (MD and Equity Research Analyst - Biotechnology)

Hey, guys. Good afternoon. Thanks for taking my question. I want to better understand how you project getting Skytrofa to a blockbuster in the US alone, based on the kind of reset of net price and what you said about where you are in terms of penetration in GHD and the broader treated growth hormone market. Can you walk through that a little bit? Thank you.

Jan Mikkelsen (CEO)

Thanks, Jess. I will start, and I have my two commercial colleagues with me here today, so they can also some way give you more details if that is desired. Currently, we see the market is about $1.4 billion, the entire growth hormone market in the US.

... As I stated in my prepared remarks, when we look on a net value per patient, we basically, which Skytrofa has about three times the net value on a yearly treatment. When we see our effort, when we look about when we are developing the long-acting segment, we see Skytrofa as really the preferred brand. Even in places where we just are in the same level of market access, clearly, clearly, clearly, Skytrofa is the preferred product. Even in places where we don't have market access, we really see Skytrofa coming in because it's really offering a unique improvement compared to the other products' potential. So when we see our label expansion effort, we basically, in the coming year, will be possible for us not only to address the pediatric growth hormone deficiency market. We, in these next weeks, we are filing for adult growth hormone deficiency.

We have Turner data coming in for Phase II later this year. We will initiate trials very fast, and we can do it in a basket manner, so we basically can get to the full coverage for all the different growth hormone indications. That give you the high level, how we see we can develop Skytrofa to a blockbuster in the U.S. And with the reset of the, what we call the market asset, we can keep that value as we have today and expand from there. I do not know, Joe or Camilla, you have further comments or Jess, you have and want to get more in-depth knowledge about specific comments from my side? Thanks.

Operator (participant)

Thank you. Please stand by for our next question. Our next question comes from the line of Tazeen Ahmad with Bank of America Securities. Your line is open.

Tazeen Ahmad (Research Analyst)

Hi, guys. Good afternoon. Thanks for taking my question. With regards to this new net price that you have for Skytrofa, can you talk to us about the competitive dynamics? Namely, are the daily injector manufacturers offering deeper discounts, and can you also talk about the level of impact that you're seeing from the relatively recent launch of the Novo competing product? Thanks.

Jan Mikkelsen (CEO)

Thanks for the question. I think it's in line with some of the same question that got asked as the first question. Yes, today, there is two other long-acting product in the US market. We basically see one of them in Ngenla, because of the obvious effect everyone can observe just going to the databases that describe tolerability and other things like that. We see we are always doing extremely well, too. In general, we see the same thing with Sogroya. Sogroya have a preferred position on some of the big PBM, and we have other places. But what I'm generally seeing is there is no doubt, we always said that Skytrofa had a best-in-class potential, but what we also see in the market, that is really realizing as the best-in-class product opportunity.

That is the strength we want to continue to build on, not only in pediatric growth hormone deficiency, but also in all the different label expansion when we coming into the situation, when we also get them. For example, you should also look at what's happening with, for example, the largest volume supplier of growth hormone in the second quarter. That basically had a revenue down 73%. So clearly there was a reset of the entire market that potentially came a little bit more unexpected to many of us, that it was so fast adopted into the system.

Operator (participant)

Thank you. Please stand by for our next question. Our next question comes from the line of Derek Archila with Wells Fargo. Your line is open.

Derek Archila (MD and Biotechnology Equity Research Analyst)

Hi there. Thanks for taking the questions. Just wanted to know what payer feedback you might have received thus far on the proposed Yorvipath pricing in the U.S.? And then I have a follow-up.

Jan Mikkelsen (CEO)

We are coming up with a responsible pricing. We are coming with a pricing where we basically can support the statement we want to go. Every patient that have hypoparathyroidism, we aim to support that they can be treated. In that entire mathematical algorithm, how to achieve that, we came to this responsible pricing on the back, and we have not got any feedback against that, that is totally acceptable.

Derek Archila (MD and Biotechnology Equity Research Analyst)

... Got it. Very helpful. And then, second question, just in terms of the patients that you've talked about on your Yorvipath in Germany and Austria, I think you commented about 250 patients on the approval call for the U.S. I guess, how do you think about that penetration relative to the overall market size in Germany? And how do you believe you're tracking thus far in the launch? Is it ahead of where you expected or, you know, less than you expected? Thanks.

Jan Mikkelsen (CEO)

I can come with some initial comments, and you can get some further comments from Camilla. Our initial aim and how we looked at how we expected the penetration profile would be, that we will see typical one to two patient per physician in the beginning. There was what we expected to see. Before each single physician really see the benefit of really what Yorvipath can give to the patients, when they observe that, we expect later in this year that each patients or each physician will start to prescribe more and more patient from the number of patients, each of them. So when we look on the number of physicians that already have prescribed in Germany and Austria, around 125 prescriber have really prescribed it. We are extremely proud about this penetration to physicians.

But I think what does really the most impressive thing for me in this launch is basic retention. When you think about we have a retention, when you start to take patient in and really test them, are they really providing a benefit for the patient, and then they have a retention of 98%, I have never seen that for any product before. That is really, really unique, but it's really described the benefit that is really giving to the patient and the state of the treatment, exactly as we have observed in our clinical trials. I do not know, Camilla, you have further comments to the launch in specific in Germany and Austria?

Camilla Hartvig (EVP and Global Chief Commercial Officer)

Yeah, I think you said it very, very well. We're very pleased with the uptake of Yorvipath in Germany. We are benchmarking other rare disease launches, and we are doing very well compared to them. So the team is working hard on both breadth and depth, and that's working well, and also creating the mix between the PTH-naïve patients and the ones that have been on Natpar. And the switch that we are making from Natpar to Yorvipath is going very, very fast. So all in all, we're very pleased with how it's going and where we go in the future.

Jan Mikkelsen (CEO)

Yes. What we have seen in different European countries, that the Natpar, basically because of its stop of manufacturing, there is a tendency to keep the patient on Natpar until all the, you can say, accumulated drug are being utilized in the specific regions. And we also see now that it's starting to change a lot, where we see much, much more Yorvipath taking up from the Natpar patient coming in now. But one thing, we have now been launched in Germany and Austria. In our Europe direct strategy, we go to France, and then here, hopefully in this year, and then in 2025, we will have the next six, seven, eight European countries being added in. Really are rolling out in the way we are generate revenue from this region in a much faster speed.

Operator (participant)

Thank you. Please stand by for our next question. Our next question comes from the line of Li Watsek with Cantor Fitzgerald. Your line is open.

Li Watsek (Research Analyst)

Hey, guys, thanks for taking my questions. Maybe just a follow-up on Skytrofa. Maybe help us understand what went into the assumptions for the lower guidance for Skytrofa this year. Just looking for more details on what's driving that EUR 100 million reduction. Maybe talk a little bit about the dynamics between, you know, broader market access and pricing, and I have a follow-up.

Jan Mikkelsen (CEO)

I can come with some overall statement first related to how we did the forecasting. Because what we basically did was that we saw a growth in volume between Q1 and Q2. And as we utilize that growth basically to predict rest of the year, and it's basically giving the lower value. And after we now have reset our market access portfolio, we are in a position that we expect that the GTN will be kept constant throughout the year. What we have observed, like last year, we saw a lagged seasonal effect between the first half compared to the second half. We still see that as an upside, which potentially will materialize, but we also know that it could not materialize. So this is how we have basically taken the guidance forward in that. And that is not anything but believing in that we potentially can accelerate growth.

We see the potential, we see the best-in-class potential on it. Joe, that is responsible for our U.S.-... you can also give a short summary about all the efforts that's getting implemented now that first will provide effects in the second half of this year.

Joe Kelly (US General Manager)

So the healthcare providers right now have extreme clarity about where they can provide Skytrofa to patients with a high chance of reimbursement, but also where Skytrofa is not on formulary. Healthcare providers are documenting intolerance, growth below the average parental height, while on other growth hormones, therefore allowing a resubmission, so that pediatric growth hormone deficient patients can have access to Skytrofa. So, there's time left in this year. Obviously, things will stabilize in 2025. So, we know now where we can continue to add patients and grow our net revenue, and expand and grow the value of the entire growth hormone marketplace.

Li Watsek (Research Analyst)

Okay. And I also wonder if you can talk a little bit about where you can further reduce from the cost perspective. And is there any chance that you can still break even this year, given the lower Skytrofa guidance?

Jan Mikkelsen (CEO)

You know, Scott is really good at numbers, so I will someday give you an overall perspective, and then Scott can go more back to some of the way we are looking at it today. One of the things we are proud about is that we really keep our operating expenses in control, and we really see that we really have a good internal system to keep Ascendis Pharma to be a highly effective organization. We also see that there is revenue specific. Europe's different regions are really starting to take off now, so potentially we have Europe ahead outside U.S. We're still in a situation where we are still discussing with FDA related to how we can really help a patient that is in shortage of a treatment here in the U.S. We hope we can help them. We will do everything to help them.

It meaning that we will have a much faster launch than we actually are taking into our plans. We hear the voice from the patients, we hear the voice from the physicians. We will do everything so we can have an earlier launch, and as soon as we get clarity about that, we will come back to you. So we have a lot of potential upsides. We have a lot of different ways where we're looking about how we can strengthen our revenue, still keeping our expenses in control, and this is how we are operating. Scott, comments?

Scott Smith (EVP and CFO)

I think you said it well, Jan. Lately, as you know, we always look to be cost effective in what we do as a company. And, we evaluate expenses that aren't tied to getting the product to patient as soon as possible. But at the same time, of course, we don't want to sacrifice doing that and getting the product. So that's part of the reason we announced the new financing today after the close for the additional $150 million. But we continue to have great scrutiny on costs, particularly where they're not leading to getting our products to patients.

Li Watsek (Research Analyst)

Thank you.

Operator (participant)

Thank you. Please stand by for our next question. Our next question comes from the line of Gavin Clark-Gartner with Evercore ISI. Your line is open.

Gavin Clark-Gartner (MD and Biotechnology Equity Research)

Hey, guys. Thanks for taking the questions. First, I just wanted to ask for Skytrofa, when did these policies with more favorable access go into place, and when should we expect a pickup in prescriptions?

Jan Mikkelsen (CEO)

I think it was gradual during the first half year, so some of them are first kicking in here in Q2. The complexity of these agreements are very complex in this way, that potential rebate compared to volume increase are some way not really aligned, and there was some of the issue we ran into, that the rebate basically got established much faster than really we can start to grow the volume. So from a modeling perspective, we see an double-digit number in increase in volume between Q1 and Q2, but we really would expect first all the effort that we now have installed, as Joe told before, is first kicking in, in the second half. Okay, so I will first see the expected growth coming in, in the second half.

Gavin Clark-Gartner (MD and Biotechnology Equity Research)

Okay, that makes sense. And, just wanted to ask on IQVIA data overall, have you guys noticed any changes in capture rate trends over time?

Jan Mikkelsen (CEO)

I think the issue I personally have with the data is that it's a sampling of few places, and if you someday keep everything stable, you can use it as an overall trend. If there is a shift between different PBMs, between different market access, you often will see some difference in volume that really are totally unpredictable, out from the perspective at the sampling between the different PBMs are different. So out from that, I think you should pay it. You should give them all the money for the utilization of these data. At least I'm not paying for it. I get it for free because I showed so many times they were totally not useful to have.... but still buy it if you want.

Gavin Clark-Gartner (MD and Biotechnology Equity Research)

Okay, thanks.

Operator (participant)

Thank you. Please stand by for our next question. Our next question comes from the line of Vikram Purohit with Morgan Stanley. Your line is open.

Vikram Purohit (Executive Director and Equity Analyst)

Hi, good afternoon. Thank you for taking our questions. I guess switching to TransCon CNP, we were just curious for the upcoming ApproaCH readout, and then also for the Coach readout in 2Q 2025, just which parameters of data you expect to report out and how you would currently guide people to compare and contrast the data sets versus competitor CNP data sets available in the space? Thank you.

Jan Mikkelsen (CEO)

I think the most easiest way to compare anything is to go to Vosoritide, because this is two, you can say, mode of action that is very most aligned between them, even if there is a difference in how we function, because we have a continuous exposure of CNP molecule, where from the Vosoritide, it basically is only have an active covers for two or three hours in every 24 hours dosing cycle. So when we look on our aim, as we have said in the last eight years, we want both to provide linear growth, but also provide and address the co-morbidity of the disease.

And what we have seen until now, and now I'm referring into Phase II, because this is the data I can talk about today, is that we have seen an annualized height velocity exactly at the same level of Vosoritide with a once-weekly dosing profile. But we have seen the improvement in many of the ways that the children, the physician, the caregivers talk about the benefit to the patient. And from that perspective, we also could show when we compared our Phase II data directly to placebo, an improvement in quality of life related to physical function. And this is why we basically are taking into our trial different measuring on key secondary endpoint, that basically hope for us that it can give an effect that really shows how we address the comorbidity, which has never, never been shown by any other product.

When we go to the biochemical process, the science behind it, which we always love, then we basically can come with great explanation why you need to have a continuous exposure of CNP, basically, to address some of the comorbidities. It give you where we started from. I need to see the data. They're coming in the next weeks, so it's pretty, pretty, pretty near. The database has been locked. People are running the stats now. As soon as they're finished, I will be taken into a room and see the data.

Vikram Purohit (Executive Director and Equity Analyst)

Got it. Thank you.

Operator (participant)

Thank you. Please stand by for our next question. Our next question comes from the line of Kelly Shi with Jefferies. Your line is open.

Kelly Shi (SVP and Senior Research Analyst)

Thank you for taking my questions. Maybe for PTH, could you please elaborate more on your ongoing interactions with regulators regarding commercialization of existing batches for the US launch, which is likely of Q4 or first quarter of next year at the moment?

Jan Mikkelsen (CEO)

We have an extremely constructive dialogue with the FDA related to how to potentially address the shortage of PTH drugs in the US. It's being driven by patients that basically are seeing the huge unmet medical need, the situation where patients have been missing their PTH for a long time, and extremely serious consequences of patients that now, today, are on that PTH and suddenly need to be disrupted from the treatment because that PTH will not be available any longer. Out of that, both patient organizations, physicians, and us and the FDA recognize this issue, and I think everyone is working together in an extremely constructive manner to find out how we can help the patients to avoid such a crisis that potentially will be.

Kelly Shi (SVP and Senior Research Analyst)

Thanks.

Operator (participant)

Thank you. Please stand by for our next question. Our next question comes from the line of David Lebowitz with Citi. Your line is open.

David Lebowitz (Senior Research Analyst - Biotechnology)

Thank you for taking my question. I got two here. First, on the Skytrofa adjustments. Just to make sure I got this right, that you came to an agreement on new net pricing with the payers, and as part of it, you had to true-up?

... prior sales in the last couple of quarters? And then I'll have one more after that.

Jan Mikkelsen (CEO)

I think you have understood it correct. This is a true-up that is basically reflecting net revenue from mainly net revenue from Q1, but about EUR 7.6 million also from 2023. That is the true-up that is being taken away from Q2 revenue. So basically, if you want to calculate real sales in Q2, you need to add all this true-up to the net revenue from Q2. That is reported.

David Lebowitz (Senior Research Analyst - Biotechnology)

Got it.

Jan Mikkelsen (CEO)

Scott, any comments, or did I get it right?

Scott Smith (EVP and CFO)

You got it right, Jan.

Jan Mikkelsen (CEO)

Good.

David Lebowitz (Senior Research Analyst - Biotechnology)

I got it. And on TransCon CNP, what has occurred to actually essentially accelerate the pivotal data timeline from prior updates?

Jan Mikkelsen (CEO)

I have to say how fast physicians, the setup of our clinical operation has been functioning in this case, have brought the data to come in much, much faster than we ever thought. I believe there is such a dedication that I only can compare to what we have seen for both PTH and Skytrofa to get this product out as fast as possible. We're also seeing the same kind of extremely, extremely high retention in this year, too, so I think always when I see this data, when I see this positive thing coming in, I think people have one dedication to get it out to the patients as fast as possible.

David Lebowitz (Senior Research Analyst - Biotechnology)

Got it. Thank you.

Operator (participant)

Thank you. Please stand by for our next question. Our next question comes from the line of Alex Thompson with Stifel. Your line is open.

Alex Thompson (Research Managing Director and Biotech Equity Research)

Hey, great. Thanks for taking my question. I guess, you know, again, on sort of the Yorvipath potential in the U.S., could you talk specifically about the data that FDA needs to or wants to see in order to understand whether the material you have currently would be suitable for a U.S. launch in the fourth quarter? Thanks.

Jan Mikkelsen (CEO)

I actually think that they have all the data to take this decision, and I don't believe any data need to be brought to them. At least the request we have on is not really on anything of the data. It's mainly how fast can we supply, how much material do we have, and other things like that. So we can ensure that when we start, we basically are in a position, we really can manage this entire demand that we expect to come.

Operator (participant)

Thank you. Please stand by for our next question. Our next question comes from the line of Paul Choi with Goldman Sachs. Your line is open.

Paul Choi (Biotechnology Analyst)

Hi, thank you. Good afternoon, and thank you for taking our question. On, to revisit Skytrofa, can you maybe comment on what percentage of your payer contracts were affected by this, by the true-up, and whether the duration of this, these changes are multi-year? Basically, it'd be helpful to understand if this is an issue we'll have to potentially revisit next year. So any comments there would be helpful. And second, just in terms of the Royalty Pharma agreement that was announced this year, I just want to confirm that from your perspective, is this potentially the last source of external capital that you think you'll need, prior to achieving operating cash flow breakeven, either this year or next year? Thank you very much.

Jan Mikkelsen (CEO)

The question that you are asking for related to the implementation of the new contracts. The implementation of the new contract was a continued process to Q1 and Q2. We are now in a position that we have reset it. This is why Scott used the great word reset. We have reset our coverage in the U.S., and out from that perspective, the GGN we expect to see in the second half will be aligned to what we saw in the first half, and we expect the same GGN in 2025, 2026, 2027, 2028, 2029, and 2030. I can only come with five years guidance.

Operator (participant)

Thank you. Our last question will come from the line of Yaron Werber from TD Cowen. Your line is open.

Yaron Werber (MD and Senior Biotechnology Analyst)

Great. Thanks for taking my question. Hope you can hear me. My question is in two parts, just relating to the... As you think about the guidance for the year, the $220 million-$240 million, does that entail the $71.5 million that you sort of did in the first half for Skytrofa on a net basis? Or is that including the $91 million, you know, assuming you did $65 million in Q1? And then secondly, Jan, you said you did $53 million in Q2 too, is that a good run rate to then start into Q3? And four, should we really think of the $26 million this quarter as a good run rate into the second half? Thank you.

Jan Mikkelsen (CEO)

Yeah, there was a lot of question in what you basically gave to Scott and me and Joe and the commercial team into it. I think when you start on the last question about basically the reported number today here in our numbers, and I see that the real number is basically the reported net sales plus the true-up that we basically provided to you. So you take the number that we had in our Q2 sales, and then basically add everything what we took in throughout, both from 2023 and what we did in Q1. There you will get basically net sales of what we had in the second quarter of 2024. So I think that is the real number. When you look at that, it is around 53 million EUR, if I calculate it right.

That, I think, is the real number for Q2. If I do my calculation, it's 53.4 or something like that, to be concrete, so we see, without doubt, a really good pickup from Q1 to Q2. When you go back and look at the seasonal variance that pertains to the comp, we will see an improved pickup in Q3 and Q4. When we did the forecasting, we basically took a conservative approach, saying we use the number between Q1 and Q2 and use that as a forthcoming number for every quarter rest of the year. Meaning is that we have not calculated any seasonal effect in the numbers we have giving you today. I think it answer most question related to your mathematic algorithm related to the numbers. Scott, you have anything to add?

Scott Smith (EVP and CFO)

Yeah, just one thing, Yaron. For the first half, we reported EUR 91.2 million without any... You know, that, those were the actual reported numbers. And as we said in the prepared remarks in the press release, that number was reduced, had been reduced by EUR 7.6 million of true-up for periods prior to January 1. So in other words, it would have been 7.2 higher or about EUR 100 million for the first half.

Operator (participant)

Thank you. Ladies and gentlemen, I'm showing no further questions in the queue. This concludes today's conference call. Thank you for your participation. You may now disconnect.