Ascendis Pharma A/S

NASDAQ:ASND

Earnings Call Analysis

Ascendis Eyes Enhanced Market Penetration

Ascendis Pharma posted a total revenue of €48 million for Q3 2023, almost entirely driven by its flagship product SKYTROFA, which generated €47 million. The firm's growth in the U.S. was significant, achieving low double-digit penetration among pediatric growth hormone deficiency (GHD) patients. To bolster its portfolio, Ascendis released interim results from ongoing clinical trials and increased its 2023 SKYTROFA revenue forecast to €170-175 million. Key upcoming milestones include a potential label expansion for SKYTROFA, pending Phase III data, and a resubmission of its NDA for TransCon PTH for hypoparathyroidism, with a European Commission decision expected soon.

Ascendis Pharma's Expansion and Revenue Growth

Ascendis Pharma has maintained a patient-centric and scientific approach to fuel long-term value creation. The U.S. market embraced SKYTROFA, a premium product, helping the company secure market value leadership within two years. Leveraging this success, Ascendis launched SKYTROFA in Germany, expecting a similar strategy to reap benefits. Anticipating TransCon PTH's approval, the company has laid out a January 2024 launch in Germany, with further expansion planned using a direct sales model named EU Direct for particular European countries and an indirect approach for international markets. Encouragingly, SKYTROFA's revenue reached €47 million in Q3, and forecasts suggest a full-year revenue between €170 million to €175 million for 2023. This growth is supported by long-term Extension trial results, demonstrating SKYTROFA's efficacy in achieving average parental height for treated patients over a period of up to six years.

SKYTROFA and TransCon PTH: Prospects and Expectations

SKYTROFA, enjoying strong revenue growth, is paving the way for differentiation and market leadership. TransCon PTH, still in the regulatory process, could potentially launch in the EU if approved, targeting a sizeable chronic hypoparathyroidism patient population. With Germany set for a 2024 launch, Ascendis Pharma anticipates a broader European and international footprint soon. Additionally, the company is resubmitting an NDA for TransCon PTH in the U.S. and awaits the FDA's response regarding the resubmission status and a new PDUFA date. The company also highlighted post hoc analysis data suggesting TransCon PTH can reverse impaired kidney function in patients with hypoparathyroidism. This data, along with other anticipated trial results, could significantly enhance the drug’s profile.

Combination Therapy and Ongoing Trials in Rare Diseases

Exploring new avenues, Ascendis aims to initiate a trial for a combination therapy of TransCon CNP and SKYTROFA, representing a potential advancement beyond individual treatments in achondroplasia. This follows promising preclinical results showing synergistic effects of the two therapies. Furthermore, TransCon IL-2 beta\/gamma's latest trial data revealed promising efficacy in several cancer types, reinforcing its potential as a best-in-class therapy. Interim results from ongoing trials are anticipated in the second half of 2024, while the company moves towards becoming cash flow positive with a strategic focus on profitability and sustained growth.

Strategic Partnerships and Vision for the Future

Adopting a focused approach, Ascendis plans to leverage its TransCon technology platform to create paradigm-shifting products. While maintaining its core interest in endocrinology rare diseases, the company is open to various business models to expand beyond its primary focus area. Ascendis envisions partnerships and potential out-licensing arrangements, especially in areas requiring extensive clinical development or where a dedicated team can drive maturation effectively. The ultimate goal remains to maximize value creation for patients and stakeholders alike, as the company sets the stage for its growth vision toward 2030.

TransCon CNP's Potential in Achondroplasia and Newborns

As the company delves into achondroplasia treatment's infancy stages, it recognizes the intricate biology and the comorbidities associated with the condition. Ascendis asserts that TransCon CNP, potentially in combination with growth hormone treatments, might address such comorbidities, and the company will explore this through upcoming trials and data presentations. In newborns, the focus will shift to more definitive outcomes, such as the treatment's impact on bone development and its potential to modify the course of the disease thoroughly.

Focused Growth in Endocrinology and Oncology

Ascendis continues to emphasize its focus on endocrinology rare diseases while also acknowledging opportunities in oncology. The indication-specific cohort trials of TransCon IL-2 beta\/gamma in lung cancer, driven by encouraging initial signals, are being expanded to explore its therapeutic potential further. Simultaneously, the company strategizes to manage its operating expenses effectively by seeking partnerships that align with its mission to deliver best-in-class treatment options.

Earnings Call Transcript 2023-Q3

[Operator]

Hello, and welcome to the Ascendis Pharma Third Quarter 2023 Earnings Conference Call and Webcast. [Operator Instructions]I would now like to hand the conference over to Tim Lee, Senior Director, Investor Relations, Ascendis Pharma. You may begin.

[Timothy Lee, executive]

Thank you, operator, and thank you, everyone, for joining our third quarter 2023 financial results conference call. I'm Tim Lee, Senior Director of Investor Relations at Ascendis Pharma.Joining me on the call today is Jan Mikkelsen, President and Chief Executive Officer; Scott Smith, Executive Vice President and Chief Financial Officer; and Dr. Stina Singel, Executive Vice President and Head of Clinical Development Oncology.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 our expectations for 2023 SKYTROFA revenue, the expected timing of the approval and launch of TransCon PTH in the U.S. and the EU, our pipeline candidates and our expectations with respect to their continued progress in 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 expansion into new therapeutic areas, our progress towards Vision 3x3 and our ability to become cash flow-positive and create a sustainable, profitable and leading global pharma company.These statements are based on information that is available to us today. Actual results and events 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 statements section in today's press release and the Risk Factors section of our most recent annual report on Form 20-F filed February 16, 2023.TransCon Growth Hormone or TransCon hGH is approved in the U.S. by FDA and the EU has received MAA authorization from the European Commission for the treatment of pediatric growth hormone deficiency. Otherwise, please note that our product candidates are investigational and not approved for commercial use. As investigational products, the safety and effectiveness of the product candidates have not been reviewed or approved by any regulatory agency.None of the statements during the conference call regarding our product candidates shall be viewed as promotional. On the call today, we'll discuss our third quarter 2023 financial results and will provide further business updates. Following some prepared remarks, we'll then open up the call for questions. And with that, let me hand over to Jan.

[Jan Mikkelsen, executive]

Thanks, Tim. Good afternoon, everyone. Our dedication to the values of patients, science and passion remains the foundation for Ascendis to create long-term value for all stakeholders. In the U.S., we achieved market value leadership for SKYTROFA within 2 years by sticking to our belief that a premium product deserves a premium price.Following the same strategy in Germany, we launched SKYTROFA in September and also expect to also launch TransCon PTH in January 2024 if approved by the European Commission in November. In select other European countries, we will use the same direct sales model to launch our portfolio as we have in the U.S. and Germany. We call this EU Direct.In other markets, we will commercialize our portfolio to sales and distribution partners who are local experts in rare diseases. We refer to this as international indirect markets. In Japan, we intend to partner our endocrinology rare disease products.We believe we have the organization and the manufacturing capacity to support expected launches of 3 independent endocrinology rare disease products by 2025. Driving further growth, we aim to continue to work to create highly differentiated TransCon product candidates, expanding into additional endocrinology rare disease indication.Last quarter, we announced the expansion of our TransCon platform, a new type of carrier platform, specific design to support new product opportunities in diseases with large patient population. I look forward to share more information with you about this in the near future.Let me provide more details on each of our programs. For TransCon Growth Hormone, commercialized SKYTROFA, we reported strong revenue growth, finished the third quarter with EUR 47 million, including initial revenue from Germany. We now expect full year 2023 SKYTROFA revenue to be between EUR 170 million and EUR 175 million.To drive differentiation and market leadership, we continue to build out the science and data behind SKYTROFA. We recently announced results from our long-term enliGHten Trial, showing that the majority of patients treated with TransCon protamine met or exceeded [indiscernible] at the time of their treatment completement or last visit. The data also demonstrated the long-term safety of SKYTROFA in patients treated up to 6 years. In adult growth hormone deficiency, we expect to share top line results during the fourth quarter from our global Phase III foresiGHt Trial.Today we estimate less than 4% of adult patients suspected of having growth hormone deficiency are treated with growth hormone making this an opportunity to both expand SKYTROFA's label and expand the overall growth hormone market. In addition, we plan to launch SKYTROFA in certain markets in our international indirect region with initial revenue contribution expected to begin in 2024.Turning to TransCon PTH. In the European Union, EMEA, CHMP adopted a positive opinion in September, recommending approval of TransCon PTH as a replacement therapy for adults with chronic hypoparathyroidism. We expect the European Commission's final decision on our marketing authorization application this month.If approved, we plan to launch TransCon PTH in Germany in January 2024. In Germany alone, where the annual cost for currently approved PTH treatment is around EUR 77,000 per patient, our target population is 22,000 chronic hypoparathyroidism patients, out of the overall patient population of around 17,000 patients.Our status team in Germany that long SKYTROFA is ready to launch TransCon PTH if approved. An EU approval will also provide the basis for marketing authorization and initiation of commercial activities for TransCon PTH in additional markets in our internal market segment, where we expect to launch starting in 2024 and further launches in Europe direct in 2025, following standard price and reimbursement part weights. By following our algorithm for product innovation, we will have taken TransCon PTH from idea to expected regulatory approval all in about 7 years.In the U.S., all documents have been finalized and we expect to resubmit the NDA for TransCon PTH for adult hypoparathyroidism within a week. We expect to know whether the FDA has accepted our resubmitted NDA with 30 days from the risk submission date. If accepted, we expect the FDA to notify us at the time where the resubmission is Class I or Class II and provide a new PDUFA date. Besides this information, we will not comment further on the resubmission.As with SKYTROFA, we continue to build of the science and data behind TransCon PTH. In September, we announced a post hoc analysis of Phase II and Phase III data, demonstrating substantial increase in estimated GFR in adults with hypoparathyroidism treated with TransCon PTH. These data suggest that treatment with TransCon PTH can reverse impaired kidney function in patients with hypoparathyroidism.Turning to TransCon CNP. This is our third endocrine rare disease product candidate. Following our end of Phase II meeting with U.S. and EU regulatory agencies, we have lined on the pathway to potentially achieve regulatory approvals in the U.S. and EU. We expect top line results from our [indiscernible] Phase III [ ApproaCH ] Trial with completed enrollment in Q3 in the second half of 2024.We continue to have strong patient retention in our trials. And during the fourth quarter, we will provide an update and share with you 1 year follow-up data with the open-label extension portion of accomplishment. We believe the strong retention in our clinical trial is a result of additional benefit of TransCon CNP in addition to heiGHt improvements.As we have further evaluated the science behind achondroplasia and our own data, we now believe that achondroplasia is a disease of both skeletal growth and muscle function and that with continuous exposure to CNP enabled by TransCon CNP may be able to address both elements.At our upcoming update, we will disclose this additional potential benefit of TransCon CNP in addition to heiGHt. If we are right, that is correct that achondroplasia is a disease of both skeletal growth and muscle function, this means that TransCon CNP could potentially offer value for adults living with achondroplasia who experienced muscle fatigue or other medical and quality-of-life impact that may be potentially addressable with constant exposure to CNP.We are in a constructive dialogue with regulators on how best to elevate the pretended impact on TransCon CNP on comorbidity, quality-of-life and other important aspect of achondroplasia, in addition to heiGHT to support an indication for treatment of achondroplasia. While our key focus in the treatment of achondroplasia is to address the comorbidities that are associated with this disease, we believe an even more effective way to address heiGHT if needed or desired could be CNP in combination with growth hormone. We believe that this combination therapy may provide greater analyzed height velocity and CNP alone and at the same time address the comorbidity of achondroplasia.Our previous presented preclinical data demonstrate the additive effect of combining TransCon CNP and growth hormone in animal models, consistent with stimulation of different growth-promoting signal pathway in the growth plate. Supporting this idea, a group out of Osaka University in Japan presented a poster during last month, ASBMR, showing that a large group of 41 with achondroplasia treated with growth hormone demonstrated first year annualized height velocity of around 7.4 centimeter, with sustained growth benefit to 5 years of treatment.To explore this concept, we plan to submit an IND amendment of similar to initiate a combination trial of TransCon CNP and SKYTROFA by the end of this year.Moving to oncology. For TransCon IL-2 beta/gamma, we recently reported new data from the IL-Believe Trial of TransCon IL-2 beta/gamma as monotherapy and in combination with [ pembro ] at ESMO. These data confirm that TransCon IL-2 beta/gamma dosed every 3 weeks demonstrated clinical activity as monotherapy or combination therapy across late-line heavily pretreated patients in multiple tumor types, further strengthening our confidence in its best-in-class potential.In 2 out of 3 small lung cancer patients treated with combination therapy in the trial and confirmed partial response and unconfirmed complete response in the first tumor assessment of an ongoing patient were observed.We believe these data are intriguing despite the very small sample size, considering the treatment history of the responders and the substantial unmet medical need. Enrollment is ongoing in the indication-specific cohort of IL-Believe, where we are now enrolling 20 to 40 patients in each indication and we expect interim results in the second half of 2024.In summary, with growing revenue, maturing of our pipeline, we continue to progress to our goal of becoming profitable. With our Vision 3x3 on track to be achieved in 2025, we are preparing for our next vision for growth to 2030. In our next vision, which I look forward to sharing with you at the beginning of 2024, we will work to lever our fully integrated capability to become the leading endocrinology rare disease company, taking product from concept out to patients on a global scale in a highly productive organization.In other areas where we believe TransCon can deliver best-in-class product candidate as we have in oncology, ophthalmology, we plan to pursue partnership of our business model to take out our product candidate to late-stage development to commercialization. Such future partnership in oncology and ophthalmology, along with the maturation of our endocrinology program should result in lower expenses in the coming years.By staying focused on achieving valued market leadership globally for endocrinology rare disease portfolio, we believe Ascendis will deliver [indiscernible] value on the long term for patients, shareholders and society.I will now turn the call over to Scott for a financial review before we open for questions.

[Scott Smith, executive]

Thank you, Jan. As Jan noted, we believe we are making significant progress towards our goal of becoming cash flow-positive with SKYTROFA revenue growing each quarter combined with diligent expense control. I will touch on some key points surrounding our financial results, but for further details, please refer to our Form 6-K filed today.Total revenue for the third quarter of 2023 was EUR 48 million. SKYTROFA revenue for the third quarter was EUR 47 million compared to EUR 35.9 million reported in the second quarter and EUR 12.3 million reported in the same period last year. The sequential growth in SKYTROFA revenue was primarily driven by increased demand in the U.S. with minimal foreign currency impact of around EUR 100,000.Exiting the third quarter, we estimate we had low double-digit penetration into the U.S. pediatric GHD patient population and we see a large opportunity in front of us to grow our patient share in pediatric GHD, which we estimate to be only half of the addressable U.S. growth hormone market.Turning to expenses. R&D costs in the third quarter totaled EUR 111.4 million, up 6% sequentially from the second quarter of 2023, primarily driven by higher endocrine rare disease-related costs, including clinical expansion and PPQ manufacturing costs related to TransCon CNP, partially offset by lower oncology-related costs.SG&A expenses declined 9% sequentially to EUR 63.6 million compared to the second quarter of 2023, primarily related to lower commercial and G&A external costs. Total operating expenses were EUR 175 million for the third quarter, flat sequentially from the second quarter of 2023.Overall, our operating loss declined sequentially by 5% to EUR 134 million for the third quarter from EUR 141 million in the second quarter of 2023.We ended the third quarter with cash, cash equivalents and marketable securities totaling EUR 455 million, including proceeds from the previously announced $150 million royalty funding agreement.Looking ahead, with continued momentum for SKYTROFA in the U.S., we are increasing full year 2023 SKYTROFA revenue expectations to EUR 170 million to EUR 175 million.Let me now also provide a review of selected key program milestones. For TransCon Growth Hormone, we expect to report top line data from the global Phase III foresiGHt trial in adult GHD in December potentially opening an opportunity to both expand SKYTROFA's label as well as expand the overall growth hormone market.For TransCon PTH, as Jan noted, we are on track to resubmit our NDA for TransCon PTH for adults with hypoparathyroidism before mid-November. We expect the European Commission decision on TransCon PTH this month. If approved, we plan TransCon PTH as our second product launch in Germany starting in January 2024.For TransCon CNP, we expect to submit an IND amendment or similar for a new clinical trial evaluating TransCon CNP in combination with TransCon Growth Hormone in children with achondroplasia. We plan to share follow-up data from the open-label extension of our Phase II accomplished trial later this quarter.And as a reminder, we expect to report top line results from ApproaCH, our Phase III trial of TransCon CNP in the second half of 2024.Within our oncology therapeutic area, enrollment continues in the Phase II portion of our IL-Believe Trial of TransCon IL-2 beta/gamma in indication-specific cohorts. We expect initial data from indication-specific cohorts in the second half of 2024.With that, operator, we are now ready to take questions.

[Operator]

[Operator Instructions] Our first question comes from Jessica Fye with JPMorgan.

[Unknown Analyst]

This is [ Nick ] on for Jess. Can you talk to us a bit more around your strategy with the oncology and ophthalmology programs and kind of what that means in terms of OpEx, both over the near-term and maybe more so over the long-term and how that could change?

[Jan Mikkelsen, executive]

Thanks, Nick. The question is related to what do we do as a company with a very, very strong platform technology that basically have application in nearly every therapeutic area. We, Ascendis, will be focused on rare disease endocrinology. This is where we will be integrated from idea stage up to the patient, where we're building up all the effort. We also know that the TransCon technology would provide paradigm shift product, best-in-class product outside our own focus area. And we also feel that we need really to be part of that value creation also to the benefit of the patients.We will have different business models that is fit exactly to each single therapeutics. Some areas we will basically outlining on an early stage where the feeling is that it's the optimal thing. It could be potentially be large patient indications where we basically never can succeed with running all the big clinical trial.It can be areas like ophthalmology, where we maturate to a state and then we potentially give it out either to a spinout or an out-licensing or combination of both where we feel that an independent management team really with a focus on, for example, ophthalmology, really, really can mature it. It's not the same thing Ascendis not will continually be involved.We will still provide service to this entity. We will be part of the upside, both related to royalties, milestone payments and equity. In other areas like oncology, where we feel that we really can make a huge difference on the patient, we made an investment in it and we will continue to make an investment into it until we feel that we can get the full value on this. And when I see how we're progressing with this oncology effort, I'm really, really proud about it.

[Unknown Analyst]

And maybe can you help set the stage for the update from the Phase II ACcomplisH Trial? And what you hope to see when you have all the [indiscernible] for the full year?

[Jan Mikkelsen, executive]

Yes. What we hope to do that we will provide you with the element that we really believe is essential for the patients. We will provide you with the sustainability of how we keep the positive benefit on growth, but we will also come in and trying to give you the explanation both from a scientific base, but also true data, how we feel that we are providing a benefit to the treatment besides just providing them analyzed height velocity.We're 100% in the belief that the key element for us is to address the comorbidities associated with this disease and that is our focus on that. This is why we believe that analyzed height velocity is an element, if it's desired and if a patient really want to have it, they can always go into perhaps the most powerful combination of treatment is CNP to move the brake growth hormone to speed on the accelerator and you basically will have what we call the wanted desire analyzed height velocity compared to the patient need and desire.

[Operator]

The next question comes from Paul Choi with Goldman Sachs.

[Kyuwon Choi, analyst]

My first question is just with regard to TransCon PTH positioning in the major European markets, specifically Germany and just how you think positioning will be particularly given surgical complication rates there? And then just the pace of reimbursement access? And then I have a follow-up question.

[Jan Mikkelsen, executive]

You're right. The European and the international market, as we call it, is what we call much more diverse way of reimbursement. The fastest one is Germany, where already from January where we expect to launch TransCon PTH will be fully reimbursed.The patient population in Germany is actually pretty large compared to the size of the country, mainly driven by a higher treatment on head and neck operations. So you're right, it's a post-surgical patient that is much higher. And we believe the addressable element of patient, not the total patient population is about 22,000.At the same time, we are in a position where the only approved treatment for hypopara in Germany is being taken away from the market. So we are in an intensive discussion with all the key centers, how we really can help more than the 400 patients that basically already are established on a PTH treatment, how to take them on treatment in '24.Besides that, for many years, there have been a long, long layout of patients that want to be on a PTH treatment, but never have the opportunity to do it. And this is why the European approval give us a fingerprint starting immediately on a full-flow commercial effort in Germany. At the same time, an European or EC approval give us the optionality to go to an in-patient program and addressing more than the 400-plus that are patient that still is in other European countries, which we feel a huge responsible for also helping and also the patient that didn't come into any treatment in the last many years.Rest of EU will take 12 to 24 months to be fully reimbursed, fully running commercial perspective. At the same time, the EU approval, which we expect to get here in the coming week will provide us with an approval system for addressing patients in the international market.Some markets, we will have an independent application, other market we can directly refer into our European application. So we are dedicated really to help patient everywhere in the world which have a need for the treatment because of the hypopara patient consideration that they take to date.

[Kyuwon Choi, analyst]

Okay. Great. And just as a quick follow-up. With competing HCH products approved here in the U.S., can you maybe just comment on maybe what mix of formulary access might be up for renegotiation this year? Any general comments you've had into the 2024 renegotiation period?

[Jan Mikkelsen, executive]

I've been asked in the last 3, 4 months a lot, what is really the impact on having 2 long-acting product in the U.S. market. And clearly, we never have seen more interest. We have not seen better numbers ever since it got clear with really best-in-class property SKYTROFA has compared to the other products, really building up on what we have established in the last 2 years, how we really are changing the treatment we see in treatment of pediatric growth hormone deficient with a better outcome.And that is not going to change with any other long-acting. Basic is just providing I believe, for some of them and lower part to exceed and overcome compared to, for example, medical exception.

[Operator]

The next question comes from Joseph Schwartz with Leerink Partners.

[Joseph Schwartz, analyst]

So a couple of questions on TransCon CNP, if I could. First of all, I was intrigued by your commentary about achondroplasia being not only a bone disease, but having a muscle component. And I was curious if you could just talk a bit about how you intend to illustrate the benefit of TransCon CNP on those aspects of the disease.And then also in terms of TransCon CNP's potential to be combined with TransCon -- or SKYTROFA rather, as it's now called, I was wondering, given I thought a lot of the earlier signs suggested that growth hormone supplementation in achondroplasia had just an ephemeral benefit, what are your thoughts on the potential for the combination to show a more sustained response in achondroplasia?

[Jan Mikkelsen, executive]

Thanks for the question. Let me start with your last question about the analyzed height velocity. We always have known that growth hormone had a positive effect on achondroplasia. Sometime has been a little bit unclear what has been the magnitude on that because it typically have been small trials.When we saw the trial coming out from the Japanese group, pretty well-controlled, following patients up to 5 years, the number is nice, 41. Everything looked great. They have 1 year before treatment follow-on what is the analyzed height velocity before treatment and it's about 4.2, exactly what you really expect to see out from this kind of demographic. And the first year, analyzed height velocity up on 7.3, 7.4. That is more than I've seen any kind of CNP treatment ever given.And when you have 41 patients, I always feel that it's a number that gives me some pretty good comfort. And it's the best analyzed height velocity I have ever seen in any heiGHt Trial with achondroplasia patients.We went back and analyzed 20 publications about all small trials, some of them, all of them. And if we are being smart, really has gone to the literature in depth, then we couldn't actually have seen it because out from this 20 publication, we nearly got the same number when we added every one off.So this is not surprising. It's something that has been in the literature, has potentially been understated, undervalued, but there is no doubt best analyzed height velocity you can get in achondroplasia today is on growth hormone. The problem with that is we did really address comorbidities. I don't believe that.I believe that the pathology behind the achondroplasia is a much more complicated biological impact on a hyperactive [ FDR3 US ] setup. And there it comes in because in the end, we're just starting the beginning of the beginning of achondroplasia treatment. We are not in the end of the beginning even. We are at the beginning of the beginning because we need to address the comorbidities.So this is why we believe the treatment is potentially moving to a combination treatment, where CNP will address some of the biological systems where we believe achondroplasia or the hyperactive FDR3 pathway are modifying a disease that basic are also after muscle growth impact. And you asked me why did we see that? It was basically the patients, the parents, the caregivers that came back to us, we got in the patient-reported outcome, we can see after 1 or 2 months, the funny side that patients are providing much better, function much better and we said it cannot be growth. It cannot be growth. And then we started to analyze this and we realize there is a strong muscle component of that.And what we would like to do when we have our CNP update, we will give you that biology behind it, explain why we do that. And also we believe that it's important for us also provide you data within a combination therapy because I believe in the combination therapy, you can get whatever analyzed height velocity potentially the same as you can see in growth hormone deficient up to 10, 12 centimeters.

[Operator]

The next question comes from Yaron Werber with TD Cowen.

[Yaron Werber, analyst]

Maybe one very quick one and the second one is a little bit bigger. Just on the PTH, the re-filing is going to be sort of in the next week or so. Can you just comment, did you sort of by this point work out all the analysis that FDA is going to want you to do or do you feel like you have good clarity? And then secondly, on the preclinical GLP-1, the data was -- obviously looks interesting on a monthly basis. What are sort of the next steps as you see them before you can start IND-enabling studies? Is it you're still trying to fine-tune the construct? Or is it potentially trying to figure out is this something you want to take yourself or is this something that you want to ultimately potentially partner?

[Timothy Lee, executive]

Jon, this is Tim. Let me take the first part. As Jan noted in his prepared remarks, besides information that we just discussed, we're not going to comment further on the resubmission process.

[Jan Mikkelsen, executive]

Related to the GLP-1, we are in a great state. We came out with the data. It provided the interest that people could see the value in it, both as an improved once-weekly treatment, but also on a once-monthly treating for this important segment going up. And we basically continue the dialogue with companies that's interested in that area.

[Operator]

The next question comes from Li Watsek with Cantor Fitzgerald.

[Li Wang Watsek, analyst]

Just first one, we've seen daily products continue to retreat and reduce output in Q3. Can you just talk about how much of a tailwind that has been for SKYTROFA? And where do you see that trend to track in the coming quarters?

[Jan Mikkelsen, executive]

The question you are addressing, what is really happening in the growth hormone market. And I think what we are seeing is the result of the consolidation that we have predicted, had expected to happen. And that is basically what you see in the marketplace today.Years ago, when we came out with our Phase II data and we didn't [ outlying ] our TransCon Growth Hormone, a lot of this established player basically said, okay, let us find out how we can milk this year in the best possible way. And you don't milk investing in production capacity.You don't really get the highest property to investing in anything. So this is why you're seeing a daily growth hormone market basically only have 3 players left now with a sales force, infrastructure and other things like that.So instead of having 6 player, you're down to 3 players and likely, there will be a fewer player in the coming year when really the real consolidation will happen. So some way, you're saying there is a shortage, but the shortage is also indicated in the way that the consolidation of the market promoted a shortest profit because it was the old days with many more company really could provide more capacity on other things that will not have been a shortage.

[Li Wang Watsek, analyst]

And [indiscernible].

[Jan Mikkelsen, executive]

It's not changing the fact why SKYTROFA is the leading company, leading brand in value is because we're providing best-in-class properties and is getting realized by physician, by caregivers, by patients and I hope and we know that also by the reimbursement system.

[Li Wang Watsek, analyst]

Okay. And then maybe just a question on TransCon IL-2. You showed some pretty nice data in small-cell lung cancer. So is there any plan to add this indication to your expansion cohort?

[Stina Singel, executive]

That's a great question, Li. Yes, we're very encouraged by our initial signal in small-cell lung cancer and we are exploring adding additional indication cohorts to our dose expansion goals.

[Operator]

The next question comes from Leland Gershell with Oppenheimer.

[Leland Gershell, analyst]

Just wanted to ask further on the plans to explore TransCon CNP for benefit outside of height velocity in terms of other comorbidities. Wondering if you could just maybe go into maybe beyond muscle, are there other measures or endpoints that you're contemplating to include in upcoming studies?

[Jan Mikkelsen, executive]

We are exploring a lot of different elements. I would like to group it a little bit. One is the patient-reported outcome where you have both our own developed way to look at it. You have established one as [ SS10 ] and other way that really are catching how you potentially have a physical benefit of this treatment or not. So this is one what we call the patient-reported outcome.The other one we're looking for is dedicated comorbidities, other clear pattern where we see less comorbidity over a year period because you need to go through some season to be quite sure you're picking up what kind of element of cold or other things could influence it.The other one is more hardcore element where we basically have a lot of X-rays of the patient. Are they really changing the [ scoliosis ]? Are they changing other elements? And we're looking on all different kinds of bone development and other things like that. This is in the older children. When we go to the newborn we started now, we go for much more hardcore facts, how to, for example, benefit in stenosis. Do we see the same element of fast fusion of [indiscernible] or other things like that?And I think this is where we take the different age group, finding out what can we really do with that. And that is what we are analyzing and building into our pivotal Phase III trial now. So we're quite sure when we have the un-blinding on that, we basically have already the element of discussion with regulatory agencies, how we basically can elevate that.

[Leland Gershell, analyst]

Okay. And then one further question, if I may. As we look forward to the update from the 1 year follow-up from accomplished open-label extension, just wondering, would you be able to provide the retention rate in terms of patients who have persisted in the [ LLE ]?

[Jan Mikkelsen, executive]

The one, the data will have all 47 patients.

[Timothy Lee, executive]

57.

[Jan Mikkelsen, executive]

57, 57 patients.

[Operator]

This does conclude today's Ascendis Pharma third quarter earnings call. You may disconnect your line at any time. And have a wonderful day.