Ascendis Pharma - Q3 2023

Transcript

Operator (participant)

Hello, and welcome to the Ascendis Pharma third quarter 2023 earnings conference call and webcast. Following the prepared remarks, there will be a question-and-answer period. Instructions will be given at that time. I would now like to hand the conference over to Tim Lee, Senior Director, Investor Relations, Ascendis Pharma. Sir, you may begin.

Tim Lee (Senior Director of Investor Relations)

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 E.U., our pipeline candidates, and our expectations with respect to their continued progress and potential commercialization.

Our strategic plans, our goals regarding our clinical pipeline, including the timing of clinical results, our ongoing and planned regulatory filings, and our expectations regarding the timing and the results of regulatory decisions, our expansion into new therapeutic areas, our progress toward 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 statement 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 E.U. 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 we'll provide further business updates. Following some prepared remarks, we'll then open up the call for questions. With that, let me hand it over to Jan.

Jan Mikkelsen (President and CEO)

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 two 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 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 E.U. 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 three 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 specifically designed 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 as 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 utilization and market leadership, we continue to build out the science and data behind SKYTROFA. We recently announced results from our long-term extension trial, showing that the majority of patients treated with TransCon growth hormone met or exceeded adult parental height SDS at the time of their treatment completion or last visit. The data also demonstrated the long-term safety of SKYTROFA in patients treated up to six 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 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 hypoparathyroid patients, out of the overall patient population of around 17,000 patients. Our sales team in Germany that launched 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 international market segment, where we expect to launch starting in 2024, and further launches in EU Direct in 2025, following standard price and reimbursement pathways. By following our algorithm for product innovation, we will have taken TransCon PTH from idea to expected regulatory approval, all in about seven years.

In the U.S., all documents have been finalized, and we expect to resubmit the NDA for TransCon PTH for adults with hypoparathyroidism within a week. We expect to know whether the FDA has accepted our resubmitted NDA within 30 days from the resubmission date. If accepted, we expect the FDA to notify us at the time where the resubmission is Class 1 or Class 2 and provide a new PDUFA date. Besides this information, we will not comment further on the resubmission.

As with SKYTROFA, we continue to build out 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 eGFR in adults with hypoparathyroidism treated with TransCon PTH. This data suggests 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 E.U. regulatory agencies, we have aligned on the pathway to potentially achieve regulatory approvals in the U.S. and E.U. We expect top-line results from our PURUS 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 one-year follow-up data with the open-label extension portion of ACCOMPLISH. 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 the continuous exposure to the CNP enabled by TransCon CNP might, may be able to address both elements. At our upcoming update, we will disclose these additional potential benefits 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 experience muscle fatigue and other medical and quality of life impacts that may be potentially addressable with constant exposure to CNP.

We are in a constructive dialogue with regulators on how best to evaluate the potential impact of TransCon CNP on comorbidities, quality of life, and other important aspects 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 annualized height velocity than CNP alone, and at the same time address the comorbidity of achondroplasia. Our previously presented preclinical data demonstrates the additive effect of combining TransCon CNP and growth hormone in animal models, consistent with stimulation of different growth-promoting signaling pathways in the growth plate.

Supporting this idea, a group out of Osaka University in Japan presented a poster during last month's ASBMR, showing that a large group of 41 achondroplasia children treated with growth hormone demonstrated first year annualized height velocity of around 7.4 cm, with sustained growth benefit through five years of treatment. To explore this concept, we plan to submit an IND amendment or 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 three weeks, demonstrated clinical activity as monotherapy or combination therapy across late-line, heavily pre-treated patients in multiple tumor types, further strengthening our confidence in its best-in-class potential.

In two out of three small cell lung cancer patients treated with combination therapy in the trial, 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 20-40 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 build progress to our goal of becoming profitable. With our vision three by three 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 patient on a global scale with a lean, 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 partnerships or a business model to take our product candidate to late-stage development to commercialization.

Thus, 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 our endocrinology rare disease portfolio, we believe Ascendis will deliver sustainable value over the long term for patients, shareholders, and society. I will now turn to call over to Scott for a financial review before we open for questions.

Scott Smith (EVP and CFO)

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 have 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-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 again noted, we are on track to resubmit our NDA for TransCon PTH for adults with hypoparathyroidism before mid-November. We expect a 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 ACCOMPLISH 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 (participant)

At this time, if you would like to ask a question, please press star one on your telephone keypad. You may remove yourself from the queue at any time by pressing star two. We do ask that you limit yourself to one question and one follow-up. We will pause for a moment to allow questions to queue. Once again, that is star one to ask a question. Our first question comes from Jessica Fye with JPMorgan. Your line is open. Please go ahead.

Speaker 10

Hey, guys, this is Nick on for Jess. Thanks for taking our questions. 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 (President and CEO)

Thanks, Nick. The question 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 at Ascendis will be focused on rare disease endocrinology. This is where we will be integrated from idea state out to the patient. It's 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 will fit exactly to each single therapeutics. Some areas we will basic out licensing on an early stage where the feeling is that it's the optimal thing. It could be potentially last patient indications where we basically never can succeed with running all the big clinical trial. It could be areas like ophthalmology, where we mature it to a state, and then we potentially give it out either to a spin out or an out licensing or a 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 as saying it's not where we continually be involved. We will still provide service to this entity. We will be part of the upside, both related to royalties, milestone payment, and equity. In other areas like oncology, where we feel that we really can make a huge difference on the patient, we make 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. When I see how we progressing with this oncology effort, I'm really, really proud about it.

Speaker 10

Okay, great. 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 observations on the [inaudible] for the full year?

Jan Mikkelsen (President and CEO)

Yeah. 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 through data, how we feel that we are providing a benefit to the treatment besides, besides just providing them annualized height velocity. We 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.

And this is why we believe that annualized height velocity is an element. If it's desired, and if the patient really want to have it, that can always go into perhaps the most powerful combination of treatment is CNP to move the brake, growth hormone to step on the accelerator, and you basically will have what we call the wanted desired annualized height velocity, compared to the patient's needs and desire.

Operator (participant)

The next question comes from Paul Choi with Goldman Sachs. Please go ahead.

Paul Choi (Healthcare Equity Research Analyst specializing in Biotechnology)

Good afternoon, and thank you for taking our questions. 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 had a follow-up question.

Jan Mikkelsen (President and CEO)

You're right. The European and the international market, as we call it, is what we call much, much more diverse way of reimbursement. But 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 and higher treatment on head and neck operations. So you're right, it's the 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 hypoparathyroidism 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 2024. Besides that, for many years, there has been a long, long waitlist of patients that want to be on a PTH treatment but never had the opportunity to do it.

And this is why the European approval gives us a footprint, starting immediately on a full-blown commercial effort in Germany. At the same time, a European or EC approval gives us the opportunity to go to a in-patient program and addressing more than the 400+ hypoparathyroidism patients that still is in other European countries, which we feel a huge responsible for also helping, and also the patients that didn't come into any treatment in the last many years. Rest of E.U., it will take 12 months-24 months to be fully reimbursed, fully running commercial perspective.

At the same time, the E.U. 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 markets we can directly refer into our European application. We are dedicated really to help patients everywhere in the world, which have a need for the treatment because of the hypoparathyroid patient situation that they face today.

Paul Choi (Healthcare Equity Research Analyst specializing in Biotechnology)

Okay, great. Thank you for that. And just as a quick follow-up, with you know, competing HGH products approved here in the U.S., can you maybe just comment on, you know, maybe what mix of formulary access might be up for re-negotiation this year, and any general comments you've had into the 2024 renegotiation period? Thank you.

Jan Mikkelsen (President and CEO)

I've been asked in the last three or four months a lot, what is really the impact on having two other long-acting products in the U.S. market? And clearly, clearly, we never have seen more interest. We have not seen better numbers ever since it got clear which really best-in-class property SKYTROFA has compared to the other products. Really building up on what we have established in the last two years, how we really are changing the treatment regimen in treatment of pediatric growth hormone deficient with a better outcome.

And that is not going to change with any other long-acting. Basically, it's just providing, I believe, for some of them, a lower bar to exceed and overcome compared to, for example, medical exception.

Operator (participant)

The next question comes from Joseph Schwartz with Leerink Partners. Please go ahead.

Joseph Schwartz (Senior Managing Director and Senior Research analyst)

Great, thanks so much. So a couple 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 science suggested that growth hormone supplementation in achondroplasia had just an ephemeral benefit, you know, what are your thoughts on the potential for the combination to show a more sustained response in achondroplasia?

Jan Mikkelsen (President and CEO)

Thanks for the question. Let me start with your last question about the annualized height velocity. We always have known that growth hormone had a positive effect on achondroplasia. Sometimes it 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 five years.

The number is nice, 41. Everything looked great. They have one year before treatment follow-up on what is the annualized height velocity before treatment, and it's about 4.2, exactly what you will expect to see out from this kind of demographic. And the first year, annualized 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 you know, if we have been smart, really have gone to the literature in depth, then we could actually have seen it, because out from this 20 publications, we nearly got the same number when we added everyone up.

So this is not surprising. It's something that has been in the literature, had 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, would it really address comorbidities? I don't believe that. I believe that the biology behind the achondroplasia is a much more complicated biological impact on a hyperactive FGFR3 receptor. And there it comes in, because in the end, we just starting the beginning of the beginning of achondroplasia treatment.

We are not in the end of the beginning, even. We are in 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 FGFR3 pathway are modifying a disease that basic are also of the muscular impact. And you ask 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 could see after one or two months, they suddenly say that patients are providing much better, function much better, and we said it cannot be growth. It cannot be growth. Then we started to analyze this, and we realized that there is a strong muscle component of that. What we would like to do when we have our CMP update, we will give you the biology behind it, explain why we do that.

And also we believe that it's important for us also to provide you data with the combination therapy, because I believe in the combination therapy, you can get annualized height velocity, potentially the same as you can see in growth hormone deficient up to 10 cm-12 cm.

Operator (participant)

The next question comes from Yaron Werber with TD Cowen. Please go ahead.

Yaron Werber (Managing Director and Senior Biotechnology Analyst)

Great, thanks for taking my question. Maybe, one very quick one, and the second one is a little bit bigger. Just on the PTH, the refiling 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, you know, the data with Sema obviously looked 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? Thank you.

Tim Lee (Senior Director of Investor Relations)

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

Jan Mikkelsen (President and CEO)

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

Operator (participant)

The next question comes from Li Watsek with Cantor Fitzgerald. Please go ahead.

Li Watsek (Director and Equity Research Analyst)

Thank you for taking the questions. Just first one, we've seen, you know, 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 (President and CEO)

The question you are addressing, what is really happening in the growth hormone market? I think what we are seeing is the results of the consolidation that we have predicted, had expected to happen, and that is basically what you see in the marketplace today. Four years ago, when we came out with our phase II data and we didn't out-license our TransCon Growth Hormone, a lot of these established players basically said, "Okay, let us find out how we can milk the market in the best possible way." You don't milk by investing in production capacity.

You don't really get the highest priority to investing in anything. So this is why you're seeing the daily growth hormone market basically only has three players left now with a sales force, infrastructure, and other things like that.

So, instead of having a six-player, you're down to three-player, and likely there will be a fewer player in the coming year when the real consolidation will happen. So, some way you're saying is there is a shortage, but the shortage is also indicated in the way that the consolidation of the market promoted a shortage of it. Because if it were old days with many more companies really could provide more capacity and other things, there would not have been a shortage.

Li Watsek (Director and Equity Research Analyst)

And-

Jan Mikkelsen (President and CEO)

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

Li Watsek (Director and Equity Research Analyst)

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

Stina Singel (EVP and Head of Clinical Development, Oncology)

That's a great question, Lee. Yeah, we're very encouraged by our initial signal in Small Cell Lung Cancer, and we are exploring adding additional indication cohort in lung cancer to our those expansion cohorts.

Li Watsek (Director and Equity Research Analyst)

Great.

Operator (participant)

The next question comes from Leland Gershell with Oppenheimer. Please go ahead.

Leland Gershell (Managing Director and Senior BioPharmaceuticals Analyst)

Hey, thanks for taking my question. Just want to ask further on the plans to explore TransCon CNP for benefit outside of height velocity in terms of other comorbidities. Wondering, and 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? Thank you.

Jan Mikkelsen (President and CEO)

We are exploring a lot of different elements. We want, 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 SF-10, and other ways 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. Are there a 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 thing 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 kind 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 spinal stenosis. Do we see the same element of fast fusion of the synchondroses or other things like that? I think this is where we take the different age group, finding out what can we really do there. That is what we're analyzing and building into our pivotal phase III trial now. We're quite sure when we have the unblinding on that, we basically have already the element of discussion with regulatory agencies, how we basically can evaluate that.

Leland Gershell (Managing Director and Senior BioPharmaceuticals Analyst)

Okay, thank you. Then, one further question, if I may, as we look forward to the update from the one-year follow-up from ACCOMPLISH open label extension. Just wondering, would you be able to provide the retention rate in terms of patients who've persisted in the OLE?

Jan Mikkelsen (President and CEO)

At the one, the data, we will have to have all 47 patients.

57, 57 patients.

Leland Gershell (Managing Director and Senior BioPharmaceuticals Analyst)

Great. Thanks very much.

Operator (participant)

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