Good day, and thank you for standing by. Welcome to the Ascendis Pharma Third Quarter Earnings 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. [Operator Instructions] Please be advised that today's conference is being recorded.

I would now like to turn the conference over to your speaker for today, Scott Smith, Chief Financial Officer. Please go ahead..

Thanks so much, operator, and thank you, everyone, for joining our Third Quarter 2024 Financial Results Conference Call. I'm Scott Smith, Executive Vice President and Chief Financial Officer at Ascendis Pharma. I'm joined today by Jan Mikkelsen, President and Chief Executive Officer.

Before we begin, I would 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 and YORVIPATH for the US.

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 and partnerships, our goals regarding our clinical pipeline, including the timing of clinical results and trials, our ongoing and planned regulatory filings and our expectations regarding the timing and the results of regulatory decisions, expected market developments and our exploration of market opportunities in the therapeutic areas of endocrinology rare disease.

These statements are based on information that is available to us as of 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 of today's press release and the Risk Factors section of our prospectus supplement filed on September 20, 2024, and 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 in the US. by the 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 UK.'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 these product units 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 third quarter 2024 financial results, and we'll provide further business updates. Following some prepared remarks, we'll then open it up for questions. With that, let me turn it over to Jan..

Thank you so much, Scott. 2024 has been another transformative year for Ascendis. Three out of three of our rare endocrinology programs have delivered clinical differentiated pivotal data.

Each product or program is demonstrating its potential to address major unmet medical needs and receive blockbuster status, positioning us to be the market leader in each disease area. Our first approved product, SKYTROFA. These are best-in-class once-weekly growth hormone, and has achieved a leading position in a highly competitive US.

market with a single indication. SKYTROFA's performance is a testament to its differentiated profile and the team's excellent execution against competition from multiple big pharma companies. We expect additional growth opportunity for SKYTROFA ahead. We are very excited about the upcoming launch of YORVIPATH in the US.

YORVIPATH is the first and only product to be FDA approved for the treatment of hypoparathyroidism in adults. There are 70,000 to 90,000 adults with hypoparathyroidism in the US., who would potentially benefit from this treatment.

The highly positive pivotal data in achondroplasia that we announced in September for our third product candidate, TransCon CRE further strengthens our belief that it could if approved becomes the treatment of choice in this multibillion dollar market opportunity.

Our new partnership with Novo Nordisk highlights our ability to attempt the success of our TransCon platform into large, high-volume therapeutic areas. By staying focused on our values of patients, science and passion, we believe we are creating extraordinary value for patients and stakeholders.

I will now provide more detailed comments on each of these products and other key areas of progress. Starting with SKYTROFA. The fundamentals for SKYTROFA are strong. Demand volume in the third quarter increased more than 60% year-over-year as physicians, caregivers and patients continue to recognize the benefit of SKYTROFA provides.

We have achieved broader market assets for SKYTROFA but have not compromised on its value, maintaining a net value per patient of around 3x that of daily growth hormone. Over time, we see an opportunity to drive SKYTROFA growth there on pediatric growth hormone deficiency with a number of label expansions.

In September, we submitted a supplement BLA for adult growth hormone deficiency. Next one, we expect top line data from our Phase II trial Turner syndrome.

And next year, we expect to imitate a SKYTROFA basket trial in established growth hormone indication such as idiopathic short stature, small for age and the genetic condition shock which includes Turner syndrome.

Importantly, the daily growth hormone market is going to a consolidation with players such as Lilly and Genentech announcing plans to exit the market. Today, we repeatedly see that when giving the choice SKYTROFA is the preferred product. The market converts to once weekly treatment.

We therefore believe SKYTROFA is well positioned to compete for the 85% of prescription still being written for daily growth hormone. We are dedicated to make SKYTROFA into our blockbuster in the US. alone.

Our focus will stay on growing its market share with new patients both treatment naive and those switching from daily growth hormone, increasing treatment adherence and duration and through label expansion, supported by pricing that recognize the value of long-acting therapy. Now moving to YORVIPATH.

We are preparing for the imminent launch of YORVIPATH in the US. Next month, in December, we plan to begin accepting prescriptions and start with reimbursement process for around 200 patients already being treated with YORVIPATH in preparation for commercial product availability in mid-January 2025.

From January 1, we expect to begin accepting prescription for all adult to chronic hypoparathyroidism. To support a strong and successful US. launch, we have invested to expand our commercial infrastructure, including building a field organization for YORVIPATH, that is 3x the number that covers SKYTROFA. Since approval, our expanded US.

field team has focused on engaging endocrinologists who treat adults, including many key opinion leaders and health care providers who are actually involved in the treatment of hypoparathyroidism.

This market is driven by endocrinologists, a relative concentrated specialty, and we estimate around 1,200 physicians have around 30,000 chronic hypopara patients in their care or an average about 25 patients each. We expect initial uptake of YORVIPATH in the US. to come from 4 segments.

There are around 200 patients already on YORVIPATH, the 350 to 400 patients in the NATPARA special use program that is ending soon, the last population of 4,000 to 5,000 PTH experienced patients and around 75,000 broader PTH treatment naive population. Outside the US., uptake of YORVIPATH continues to be robust.

As of today, there are now around 600 patients on commercial therapy in Germany and Austria, where the commercial launch began this year in January and across many patient programs in multiple other countries. We believe YORVIPATH is a truly unique product.

We have decided to have the same mode of action and distribution in the body as endogenous PTH and to provide active PTH within physiological levels for 24 hours, 7 days a week. And we don't see any other compounds in the development that share these key attributes.

We believe that YORVIPATH will become the therapy for the majority of adults with hypoparathyroidism, which is aligned with the recently established guidelines for the treatment of hypoparathyroidism in adults. I will now provide some commentary on TransCon CNP.

Our value proposition for TransCon CNP is simple, it's to establish a treatment for patients of all ages with achondroplasia.

Our pivotal trial results showed that TransCon CNP not only exceeded benchmark for growth that have been slated in other randomized clinical trial, but also impacted other endpoints that are important for individuals with achondroplasia.

We believe TransCon CNP has a best-in-class efficacy profile with safety and tolerability similar to placebo, including an excellent injection site tolerability and once weekly dosing. This sets the stage for its leadership in the achondroplasia market.

We expect to submit an NDA to the FDA for TransCon CNP for the treatment of children with achondroplasia during the first quarter of 2025 and an M&A to the EMEA during the third quarter of 2025.

With two once-weekly growth promoting products in our portfolio, SKYTROFA and TransCon CNP, we believe Ascendis is positioned to become the leader in treatment of growth disorder.

We expect topline week 26 data from COACH, our first combination trial of TransCon Growth Hormone and TransCon CNP in children with achondroplasia aged two to 11 years in the second quarter of 2025. Now moving to our recently announced Novo Nordisk collaboration.

We believe this agreement is a strong validation of our ability to drive innovation, benefit patients and expand the TransCon technology platform to a larger therapeutic area as described in our Vision 2030. The lead program in the collaboration is a once monthly GPL-1 that will initially target obesity and type two diabetes.

The global market for GPL-1s, like semaglutide is expected to exceed more than $50 billion this year and to double or triple in the next 10 years. Once-monthly GPL-1 could become the treatment of choice in this future market.

And we are pleased to be working with Novo Nordisk, a world leader with the manufacturing capacity and commercial infrastructure to realize the value of this opportunity.

Financially, upon closing, we will receive an upfront payment of USD 100 million and escalating tiered mid-single-digit royalties on global net sales of TransCon products along with development, regulatory and sales milestones. I will close with an update on our oncology program.

In September, we presented first results from the platinum-resistant ovarian cancer cohort of the Phase I/II, IL-Believe Trial of TransCon IL-2 beta/gamma at ESMO showing that antitumor clinical response that observed in 29 of efficacy-related patients treated with TransCon IL-2 beta in combination with chemotherapy.

This was the second indication-specific cohort showing meaningful signs of antitumor activity in heavily pretreated patients. Given these results, recently, we closed enrollment to dose expansion cohort in the transcendIT-101 and IL-Believe trial of TransCon TLR7/8 agonist to highly prioritize our effort on TransCon IL-2 beta/gamma.

In summary, our progress and position are strong, and we believe the expected product revenue and strength of our balance sheet give us the ability to invest in global launches, label expansion and life cycle management for all of our three rare endocrinology program to support each of them to reach blockbuster status.

And at the same time, we will continue to invest in new product candidate created by our TransCon technology platform to build sustainable growth and profitability. I will now turn it over to Scott for financial update..

expand the label for SKYTROFA to adult growth hormone deficiency and build on its leadership position as a best-in-class growth home product; successfully launch YORVIPATH in the US. and in multiple countries in our Europe direct and international markets; and submit TransCon CNP for approval in the US.

and EU and initiate preparations for launch in achondroplasia. I'll touch on some key points surrounding our third quarter financial results, but for further details, please refer to our 6-K filed today.

SKYTROFA volume increased more than 60% in the third quarter of 2024, compared to the third quarter last year, while reported revenue was EUR 47.2 million compared to EUR 47 million reported in the third quarter of 2023.

The increase in volume was offset primarily by higher sales deductions compared to the prior year as a result of broader market access. SKYTROFA revenue in the third quarter of 2024 related to channel inventory was approximately EUR 3.5 million lower compared to the third quarter last year.

SKYTROFA revenue in the third quarter of 2024 was also negatively impacted by adjustments related to prior period sales deductions of EUR 2.5 million. SKYTROFA for over the first nine months of 2024 totaled EUR 138.5 million, a 21% increase year-over-year compared to EUR 114.4 million during the same period in 2023.

For the first nine months of 2024, SKYTROFA volume more than doubled, but was partially offset by higher sales deductions including an accrual true-up of EUR 9.3 million related to periods prior to January 1, 2024.

Overall, despite the accrual true-ups in Q2 and Q3, with more claims in hand, we can see that realized pricing has been stable since the beginning of the year.

With stable pricing, a few extra shipping days and increasing demand, we expect Q4 revenues to increase sequentially and expect full year 2024 SKYTROFA revenue, excluding sales deductions related to prior years, to be EUR 200 million to EUR 220 million.

We estimate by the end of Q3 2024 based on third-party data, SKYTROFA penetration in the overall treated growth hormone market was approximately 6% with our single indication for pediatric GHD. In addition, we estimate based on third-party data, that sales of daily growth hormone products represent about 85% of total US.

prescriptions, leaving lots of room for further growth for SKYTROFA and market expansion. Shifting to TransCon PTH, Third quarter YORVIPATH revenue outside the US. increased more than 60% to EUR 8.5 million, driven by growing patient and physician demand partially offset by accruals reflecting the end of the free pricing period in the third quarter.

Final pricing in Germany is expected to be completed next year. Closing out the topline, total revenue for the third quarter was EUR 57.8 million, including EUR 2.1 million tied to rendering of services and license revenue. Turning to expenses.

R&D costs in the third quarter of 2024 totalled EUR 73.5 million compared to EUR 111.4 million during the third quarter of 2023. The 34% decline was largely due to lower external development costs for TransCon HGH, TransCon PTH and TransCon CNP as well as the Eyconis transaction.

SG&A expenses in the quarter totalled EUR 69.8 million compared to EUR 63.6 million during the third quarter of 2023. The EUR 6 million increase was primarily due to higher employee costs, including the impact from global commercial expansion.

Total operating expenses were EUR 143.4 million for the third quarter of 2024, an 18% decrease compared to EUR 175.1 million during the third quarter of 2023. Total operating expenses for the first nine months of 2024 were EUR 439 million.

Net finance income in the quarter was EUR 2.9 million compared to finance expenses of EUR 20.4 million in the third quarter of last year. As a reminder, the net finance line can fluctuate quarter-to-quarter, driven in part by noncash items related to our outstanding convertible notes.

Finally, we ended the third quarter with cash, cash equivalents and marketable securities totaling EUR 626 million compared to EUR 399 million as of December 31, 2023. To be clear, total cash does not include the expected $100 million upfront payment from Novo Nordisk, which is due following closing of the transaction.

For the full year 2024, based on current plans, we expect product revenue from SKYTROFA and YORVIPATH ex US. to continue to grow.

Specifically, we expect SKYTROFA revenue, excluding sales deductions related to prior years to be EUR 200 million to EUR 220 million, and total operating expenses, SG&A and R&D to be approximately EUR 600 million, which includes expenses related to launching YORVIPATH in the US.

We are ready and well capitalized to deliver a successful launch of YORVIPATH in the US. with product availability expected in mid-January. With that, operator, we are now ready to take questions..

[Operator Instructions] Our first question for today comes from Jessica Fye of JPMorgan. Your line is open..

Hey guys, good afternoon. Thanks for taking my questions. You laid out a case for a strong initial US. launch for YORVIPATH, and we're clearly seeing that ex US. already.

Can you talk about why you have confidence that you'll be able to keep the patients that you pick up in light of various competitors potentially entering the market? And my second one or a follow-up is just in the past, you've talked about an expectation to reach operating cash flow breakeven by the end of 2025.

Is that still your expectation?.

Thanks, Jess. I will take the first question, and then I'll delegate it to Scott to take the second question. And I think yes, we basically some way reflected that in the -- our initial remarks.

Because what we described, what is really a true replacement therapy because we are talking about patients that is not have sufficient industrious hormone to really to have a normal functional life, like a type 1 diabetes.

And when we look at the characteristics, which have been proven in multiple publications, specific from NIH, where they used infusion pumps, taking PTH into short-acting PTH, but they can keep up the physiological concentration 24 hours, seven days a week.

So when we think about what need to be taken as what we call a true replacement therapy is that you need to have the same mode of action, like you have in the Dutchess hormone.

And basically, you need it because there are so many multiple organs that need some way to get the right receptor activating, signal pathway activating to have the normal physiological function. The second thing is that you need also to have the right distribution.

Because it needs to hit both, for example, just an organ and kidney, both in the blood and the urine side need to go to the brain, it need to go everything else to. And the last thing is basic the PK profile. You need to have the right concentrating 24 hours, seven days a week.

And whatever I do, when I look on the clinical program that is I cannot find anyone that's just living up to the level of what I call a replacement therapy. That was for first question. I think Scott will take the second one..

With regard to our goal to be cash flow breakeven, that still remains the goal, and we believe we can achieve that with the cash and cash equivalents that we have on hand..

And our next question will be coming from the line of Tazeen Ahmad of Bank of America Securities. Your line is open..

Hi guys. Good evening. Thanks for taking my question. Mine's on CNP.

Can you just give us an update on where you are in preparing for your meeting with FDA and or your application, what are the open items that need to be completed before you can guide us to whether or not the data that you have is indeed going to sufficient for application? And also there be potentially a pathway where you start your submission and provide data on a rolling basis if the agency is looking for extended data?.

Thanks a lot for the question. And to our knowledge, and that is after interaction with both regulatory agencies in Europe, and US., we have the integrated data packet that need to be part of the submission. So we basically have no missing data that we actually need to be generating for basic to finalize the application here, for example, with FDA.

We've been doing that and we're getting ready to the filing, what will happen in Q1 is pretty near now. And when I look on the packet, we really are providing a packet that not only providing the best-in-class growth of the children, but we also, for first time, can address comorbidities.

And I think this is something we will continue to share with you how we really are providing a treatment of achondroplasia instead of just providing linear growth. If you just wanted to have linear growth, I would just give them growth hormone is the best hormone to provide linear growth.

And I think this is why we are unique position because we have the opportunity really to address the comorbidities. And we will continue to give you data how really that is really coming from the data package we are submitted to the regulatory agencies..

And our next question will be coming from Derek Archila of Wells Fargo. Derek, your line is open..

Sorry. This is Simona on for Derek. So for SKYTROFA approval, have you made any progress with the payers? And do you have any updates on if it's on formulary or if there's any new blocks for market entry? And just one on YORVIPATH.

Will this launch require a medical exception for access to the drug?.

The first part of SKYTROFA. I think we have been pretty clear how we are improving our market assess situation which has happened a lot in this year was really impacting our Q1 results in this way.

We are still in a situation where we are getting medical exception in the areas but we definitely are in a complete different market assess situation, this one. But I believe what we see here, and I think you need to go back and seeing how we really want to balance SKYTROFA into the growth hormone market.

We believe SKYTROFA because of its best-in-class nature, it's really can be positioned as a high-value product opportunity. We believe this is responsible to have a price that our net value is 3x higher than daily growth hormone because of all the benefit we're providing.

And I think this is the key element in our commercial strategy on how to make SKYTROFA to a blockbuster just in US. alone, is to keep value. And what is also helping us is the consolidation. We saw first that one of the big pharma left it. And now we see another big farm leaving it.

And I believe if you're sitting with a daily growth hormone and you don't have any kind of once-weekly product in one way or the other way, you actually are going to leave this market. So the consolidation is happening. Potentially a little bit slower than we have predicted, but it's happening.

And we will be there to really, really to utilize this opportunity every time they come. We also have a strong reputation. We're a company that never have been on a shortlist. We're a company that has the most supply chain that really have not been disrupted by any kind of delivery.

Related to YORVIPATH, it's an interesting comment out from the thing because when you look on a product being launched specific in the rare diseases and to the group of product we belong to, there's two things that is a key element for uptake. One is that are you basic in on label for the majority of the patients. And I can guarantee we are that.

Second point, do you really have guidelines that basically promote that you are inside the guidelines for the majority of patients. And please read the guidelines that was issued for a few years ago around hypopara patients here in the US. for adult treatment. And I think we are in such a unique position. We both on label, we are also in a position.

We are inside the guidelines. So we feel that we basically are providing that benefit to the patient. And just don't forget, we are the only product out there. No other products there.

So when you see the three elements, you're single product, no one else choices, you are on label and you're inside the guidelines, I actually believe you have a very, very, very strong position, to get the journey from a prescription out to be fully reimbursed in US. in the most easier way..

And the next question will be coming from the line of Yaron Werber of TD Cowen. Your line is open..

Yes, hi, great. Thanks for taking my question. Jan, maybe just a couple on YORVIPATH. I think in the past, we were expecting that the pair will get pulled off the market by Takeda by the end of this year.

Can you give us a little bit of a sense, how is this going to work? Is it driven by the hard date like December 31? Or is it just driven by when they run out of supply? And then also in Europe, I know you're waiting for the NATPARA inventory to run out before YORVIPATH really takes off.

Can you give us a little bit of an update on timing, your expectations?.

Yes. You're asking me a question, that is outside my control. Because I cannot control when NATPARA is running out of stock. We know they are staying everywhere. They are ceased the production and they're running out batches. We know now we are in a position in the US. I believe they will try to get out of the US.

market as fast as possible because they basically provide free drug out to each single patient in the US. In Europe, we have seen an interesting pattern to them where they are trying to come out with every inventory they are doing as fast as possible and using all different means to do that.

And to our best estimate, it somewhere will be in next year, you will see the big transition happening. But at the same time, we saw in the two different patterns in two different countries. In Germany, they switched NATPARA patients because I said there was an improved treatment.

Now in Germany, they are over and taking new patients now because the pool of NATPARA patients is running out. Austria different. They said, let's keep the NATPARA patient on NATPARA as long as possible and then take new patients. So what is going to happen in the US.

when I see these two countries, two different patterns, and we believe from the 4 groups we talked about, all of them will be switched..

And our next question will be coming from the line of Gavin Clark-Gartner of Evercore. Your line is open..

Hey guys, Congrats on all the progress. So I just wanted to ask about the 4,000 to 5,000 PTH experienced patients in the US. that you've noted. Are all these patients actively managed in the health care system.

And I guess more specifically, how long do you believe it could take to get the majority of these patients on to YORVIPATH? Could it be within the first year, two years? Any estimate would be helpful..

Thanks for the question. I believe this PTH experienced group they're used to do daily administration. If you're coming from, for example, for FORTEO, some patients are doing injection four times a day.

But you know the FORTEO and short acting, many of them have a limited time frame for only two years because of the super physiological PTH level they're providing. So when I look on the 4,000 to 5,000, the majority is NATPARA patients that couldn't come into the program that basically got started when they have the recall.

So the NATPARA use program to our best estimate, only could take 1 out of 10 patients. So that was starting sitting without any kind of PTH treatment option. I believe this patient group will try to be coming into an endocrinologists as fast as possible. This is why we're talking with the patient organization.

We're talking now that here in the middle of January, you basically will have product availability here in the US. and really go in and start to get the prescription.

We have our hub the ASAP program, really a unique element that will help the patient in the journey from the prescription to basically to be in a position they are fully reimbursed and beyond drug. We have the infrastructure to do it. We know how to get it done.

And we believe there is so many patients that are just waiting for this opportunity to get a treatment..

And the next question is coming from the line of Li Watsek of Cantor. Your line is open..

Hey guys, thank you for taking my questions. I guess relative to the pediatric growth hormone market, how should we think about the revenue contribution from the adult market as well by the Turner syndrome. And it sounds like you're initiating a basket trial.

Maybe just comment on the market opportunity there relative to the growth hormone market?.

Yes. When we look on the growth hormone market, on label, we only have access to the market segment that is covering by pediatric growth hormone deficiency.

We have now filed the adult growth hormone deficiency, which open up for a complete interesting segment currently only established at 10% of the growth hormone market, but it's a highly, highly underpenetrated treatment population in the US. Best estimate we have is about 5% to 8% is being treated with adult growth hormone deficiency.

So it's really to develop a market that can be nearly the same size as the pediatric market. We focused on being quite sure we are SKYTROFA -- should be the choice in all indications where we have the growth hormone market.

And this is why we're making the basket trial where we're taking the ISS, SCA and shock, which include Turner -- by having this label expansion, we will be in a position that we nearly covering the entire growth hormone market. And this is our vision to build SKYTROFA to the blockbuster it should be.

Going to PTH going to hypopara, it's quite different because the adult segment is basic the majority of the market. Patients are coming with pediatric, what we call hypopara. The majority were not coming from post-surgery but more coming from immunological genetic diseases.

So it's a quite different segment in this way and is much more smaller than the adult segment. We are initiating a trial in the pediatric segment. It will be just in the planning now and it's a part of also our obligation to the European authorities also make it available in the pediatric segment..

And our next question will be coming from the line of Joe Schwartz of Leerink Partners. Your line is open..

Great, thanks so much. First question is on YORVIPATH. I was just wondering, how do you expect the launch of YORVI in the US. to compare to Europe in general? And then is the monthly serum calcium monitoring requirement in the US.

likely to be a significant deterrent for patients, which impacts the launch curve at all in your view?.

Let me start on the last question, Joe. What I've seen, I've seen the UM criteria for some of the places. And I have never seen that being installed as any kind of criteria. So I don't think this is really the case. I know the patients often will have this kind of missing.

So it's not like it will be a burden to have it, but I'm not seeing it in the UM criteria from anyone. Related to the US. market is quite different than the German, Austrian market, penetration curves from Germany or Austria compared to US. had quite different slopes.

So from that perspective is that -- it's such a unified market segment in the US., if things goes faster in the US. There is much more open mindset to try new treatment what we're seeing now in Germany, everyone tried with 1 or two patients, now it's going up. So the average patient per physician was between one and two.

Now we first come into the states where they're going to be in two and three for the physician. And this is some kind of, what I call, you first penetrate, then you broaden it. But what we expect to see in the US. is a much, much larger and faster uptake..

Okay.

And then on SKYTROFA, is today's adjustment to the 2024 revenue guidance due to lower volume, price or both? Can you help us understand why the additional adjustment is necessary beyond what we saw previously and whether these dynamics have any potential to carry over into 2025?.

I think Scott will take that..

I think, joe, in Q3, volume was a little bit lower than expected, but we also had fewer selling days. We're off to a pretty good start here in Q4 but we want to be a little bit more cautious until we see more data. So given we had the call today, we came in with the number that you saw.

Some dependencies that could increase the number for Q4 would be things like channel buying that we haven't seen in previous periods or higher refill rates with prescriptions, but we just don't have enough experience to say that that's something to expect in Q4 yet..

And the next question will be coming from the line of Vikram Purohit of Morgan Stanley. Your line is open..

Hi, good afternoon. Thank you for taking our questions. So we had one on the recently announced Novo Nordisk collaboration.

We were just curious as that moves forward, what the next public disclosures there could be, what we can expect to learn from yourself and/or Novo Nordisk? And then secondly, for the oncology pipeline, we were curious what the next milestones there might be and how you're thinking about progressing the oncology efforts you have in place with potential partnerships and what partnership economics or a good partner for those efforts might look like in the future?.

Thanks for the questions. Related to the first question, we have seen stream powerful TransCon technology, making three out of three endocrinology product from idea states until basic pivotal data and two of them are approved now. And this is the TransCon technology with our algorithm for product innovation and Novo Nordisk took that up too.

So we basically are in a position when I see what we can provide with our TransCon technology in obesity, in metabolic diseases like diabetes is we can take a peptide, well-known properties, already proven safety, proven efficacy and do exactly the same thing.

And at the same time, we can make it to a once monthly but potentially also improve tolerability to potential efficacy in the same time. And we're really proud to have a partner like Novo Nordisk.

There's only two companies in the world in my view that have the manufacturability capacity really to make and commercial infrastructure to really to make this in a $1 billion, $1 billion, $1 billion product. And these two companies we actually have a unique great collaboration with.

And they're not only using it for one of the product, they will continue to apply multiple, multiple product on it. Related to specific progress, related to a specific update about progress on how they're utilizing the TransCon technology, I believe the best one to answer that will be Novo Nordisk in this case. Going back to oncology.

We have been extremely encouraged by the effect we have seen in platinum-resistant ovarian cancer in late-stage patients that basically have no hope. We actually see meaningful clinical benefit in this, and this is what we're really trying to focus on and really pursuing in the fastest and best possible manner to really help these patients.

As we said, we are open in the way we will create value out of oncology pipeline in all different structures and we will pursue the structure that focus on getting out to as many patients as possible as fast as possible while also creating most value for our stakeholders..

And our next question will be coming from the line of Kelly Shi of Jefferies. Your line is open. Kelly, your line is open..

Thank you. Thank you for taking my questions. So I just want to confirm, like several numbers in the press release, it was mentioned that the first three quarters of 2024 totaled at EUR 138.5 million and the full year guidance at EUR 200 million to EUR 220 million. So that gives Q4 sales to be EUR 61.5 million and to reach the lower bound.

And is this the sales without adjustment? And secondly, for the TransCon CNP NDA submission, just curious, have you talked to regulatory agencies that 12-month data is enough for the submission?.

I think I will delegate the first part of the question to Scott and he just wrote down all your numbers, and double checking them now..

Yes. Our -- the updated guidance of EUR 200 million to EUR 220 million of revenue excludes the impact of prior period sales deductions or sales deductions related to periods prior to January 1, 2024..

Related to your second question, it's clear. We have not got any request that deliver more than the 12 months data related to efficacy. During a filing, you always have what we call safety date update and you have cut of different safety that always been giving during the period where you have the regulatory evaluation..

Our next question will be coming from the line of David Lebowitz of Citi. Your line is open..

Thank you very much for taking my question. I got two for you. First, on the long-acting GLP-1 agonist. Given that GLP-1s are titrated as part of normal treatment, what type of challenges does the long-acting present in this regard? And then the second question will be -- is on SKYTROFA.

Could you, I guess, give us perspective on how the net or average price has evolved as you've added new payers and to give us perspective just for modeling purpose and how we should view this going forward?.

Okay. Thanks a lot. When you talk about long-acting GLP-1, I would like to refer back to the basic biology of the GLP-1 because what is really driving the tolerability issue in -- and through our knowledge is that when you go from the trough level, this is called the lowest level or at the peak level and how fast you really are doing that.

This is where you see the tolerability issue in it. So therefore, you see when patients stopping treatment have problem with the treatment is because they're going fast up from a trough to a peak.

This is why you have titration where you start on a lower dose, go up to the next dose, just to be sure you acclimating, that you deal with this tolerability. When you think on our profile that you saw in our JPMorgan Tech or also other places, you can see one of the uniqueness with our TransCon technology is that we have an extremely long Tmax.

And what do that mean? It means that it takes extremely long basic for being a position that you go from a trough to a peak level.

And therefore, which we also have seen in our own data in animal studies that you basically have a much better tolerability profile than you have in more short-acting where you go very, very, very fast up to the element of the max dose.

I think it's pretty clear, we had a clear statement in our prepared remarks that if you take the legal level of daily growth hormone and take it to 1x, SKYTROFA has still 3x value..

And our next question will be coming from the line of Paul Choi of Goldman Sachs. Your line is open..

Hi, good afternoon and thank you for taking our questions. I also want to ask on your recent Novo licensing deal. And given that GLP-1s are validated, it would seem like the 505(b)(2) development pathway is open to a potential monthly GLP-1 agonist. And so development times might be shorter than a full clinical development program for a monthly GLP-1.

Do you agree with this? And if that's the case, do you have any sense of what timelines might be for development here? When you might start to recognize royalty revenue from your partners? Any comment there would be super helpful..

Thank you, and I can only agree 100% with your comments. It is exactly how many cases you will take a development pathway, and it will be shorter than a normal development pathway. And also you have the potential opportunity to get a broad labeling for the beginning with only one indication. That is the benefit of using a 505(b)(2).

Related to specific timeline, we need to refer to Novo Nordisk for this discussion..

And our next question will be coming from the line of Leland Gershell of Oppenheimer. Your line is open..

Oh hey, thanks for squeezing me in and for taking the questions. Just one from us. I think, Jan, you had mentioned that commercialization in France might be starting by the end of this year. I'm not sure if I missed it in your prepared comments.

Is that on track? And I was also wondering as you further pursue European commercialization, if you have any further clarity on the cadence of rollout in various countries through 2025?.

Yes, you are right. What we have been established as what we call full commercial air is only Germany and Austria. In France, we got elected to a special program called AP2. This is a program that basically are non-promotional but we can give them commercial reimbursed drug. And it's got established for about three, four weeks since in France.

And the number we have seen coming in where physicians really say, I believe -- because it needs to be physician-driven, we cannot commercialize is really unique.

Going forward, we will have fully commercial countries where we call in Europe direct where there will be full commercial countries and it will be the majority of the countries in Europe, EU. There will be basic full commercial next year in different quarters, a lot of them coming into Q2, Q3 next year.

So what you saw of our success in Austria and Germany, we basically are repeating that in about 8, 10 other Europe direct countries. In our international market, we see a lot of named patient program, but we are also in the international market where we now have countries covered by distribution agreement about 30 to 35 countries.

And there are currently giving patients through named patient program. And also next year, we will start to see more or more of this country also going into full-blown commercialization.

It's dependent on what we are spending a lot of resources on now is basically getting filing, which we have done in many, many countries, but also getting approval in all of these countries. And we're getting approval already beginning this year. And meaning is that we know we will be full commercial this year -- next year..

Our next question will be coming from the line of Alex Thompson of Stifel. Your line is open..

Great. Thanks for taking our questions. I guess for YORVIPATH, can you talk a little bit about your supply -- commercial supply readiness for the mid-January launch. I think you mentioned that you're going to take scripts from current YORVIPATH patients in December.

Could you open that up to all? Or are there supply constraints or some other constraints? And then on CNP, do you still have an appetite for running a broader basket study of additional indications? Or are you now focused on SKYTROFA there?.

PTH, we have unlimited supply. We can take all patients in everyone. There is no limitation in manufacturing.

We already have laid out the entire year's manufacturing batches, locked every time point we want to have to be sure that we're building out the same robust supply chains that we have done with SKYTROFA, which has been unique even if we suddenly got major more demand suddenly.

So the same thing is happening for YORVIPATH, and we are really on top to be sure no patient ever should go in and not can get the prescription fulfilled of YORVIPATH. Going to CNP, I ask -- you're right. We think some indication is best treated with SKYTROFA. But definitely, there is also indication that is best treated with CNP.

And some of them are best treated with the combination of both of them, either on a constant basis or only on a short duration.

So we are planning to go into what we call CNP-related diseases, meaning as that this is diseases where the growth disorder or what we call other element of missing CNP only can be functional back to what we will see in a normal physiology by providing CNP back.

So besides planning a trial in hypochondroplasia, which will be initiated next year, we are also planning for a basket trial. We think that is easy way where we can take multiple indication of CNP-related diseases into one single trial that we also is in the planning for doing now. So we are not restricting us just to SKYTROFA label expansion.

We dedicated to be a leader in growth disorder. We have plus 20 diseases to go through. And some of them, we will take SKYTROFA. Some of them, we will take TransCon CNP. And some of them, we receive the optimal treatment happening with a combination of both of them either on a constant base or on shorter duration, one or the other..

Thank you so much for your questions and answers today. That is all the time that we have for the session. We thank you so much for joining the conference call today. You all may disconnect, and have a great rest of your day..