Ascendis Pharma A/S Q2 2025 Earnings Report

Ascendis Pharma A/S EPS Results

• Actual EPS: -$0.93
• Consensus EPS: -$1.42
• Beat/Miss: Beat by +$0.49
• One Year Ago EPS: N/A

Ascendis Pharma A/S Revenue Results

• Actual Revenue: $216.28 million
• Expected Revenue: $163.17 million
• Beat/Miss: Beat by +$53.12 million
• YoY Revenue Growth: N/A

Ascendis Pharma A/S Announcement Details

• Quarter: Q2 2025
• Date: 8/7/2025
• Time: After Market Closes
• Conference Call Date: Thursday, August 7, 2025
• Conference Call Time: 4:30PM ET

Ascendis Pharma A/S (NASDAQ:ASND), a biopharmaceutical company, focuses on developing therapies for unmet medical needs. It offers SKYTROFA for treating patients with growth hormone deficiency (GHD). The company is also developing a pipeline of three independent endocrinology rare disease product candidates in clinical development, as well as focuses on advancing oncology therapeutic candidates. The company was incorporated in 2006 and is headquartered in Hellerup, Denmark.

Ascendis Pharma A/S Q2 2025 Earnings Call Transcript

Key Takeaways

• Positive Sentiment: Europass revenues doubled sequentially to €103 million in Q2 with over 3 100 unique patients and 1 500 prescribers, reflecting strong global launch momentum.
• Positive Sentiment: The FDA granted priority review for TransCon CNP in achondroplasia with a PDUFA date of November 30, recognizing its potential for meaningful safety and efficacy improvements.
• Positive Sentiment: Interim Phase 2 COACH results showed the combination of TransCon CNP and TransCon Growth Hormone achieved ~3× the linear growth versus monotherapy, alongside improved body proportionality and a favorable safety profile.
• Positive Sentiment: Skytropha delivered €51 million in Q2 sales and secured FDA approval for adult growth hormone deficiency, positioning it for further label expansions and sustained growth.
• Negative Sentiment: Currency fluctuations imposed a €7.6 million headwind on Q2 revenues, highlighting ongoing forex risks to reported results.

Presentation

[Operator]

Good day, and thank you for standing by. Welcome to the q two twenty twenty five Ascendis Pharma earnings conference call. At this time, all participants are in listen only mode. After the speakers' presentation, there will be a question and answer session. To ask a question during the session, you will need to press 11 on your telephone.

[Operator]

You will then hear an automated message advising that your hand is raised. To withdraw your question, please press 11 again. You may ask one question and one follow-up. Please be advised that today's conference is being recorded. I would now like to hand the conference over to your first speaker today, Scott Smith, Ascendis Pharma's CFO. Please go ahead.

[Scott Smith, EVP & CFO at Ascendis Pharma]

Good morning, everyone, and thank you, everyone, for joining our second quarter twenty twenty five financial results conference call. I'm Scott Smith, Executive Vice President and Chief Financial Officer at Ascendis Pharma. Joining me on today's call are Jen Muller Mikkelsen, President and Chief Executive Officer Sherry Glass, Chief Business Officer Jay Wu, Executive Vice President and President U. S. Market Amy Hsu, Executive Vice President of Endocrine and Rare Disease Medical Sciences and Chief Medical Officer.

[Scott Smith, EVP & CFO at Ascendis Pharma]

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 SKYYTORIFA and Neuorvipath as well as certain financial expectations, our pipeline candidates and our expectations with respect to their continued progress and potential commercialization Our strategic plans, partnerships and investments, 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 our regulatory decisions. These statements are based on information that is available to us as of today. Actual results may 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.

[Scott Smith, EVP & CFO at Ascendis Pharma]

For additional information concerning the factors that could 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 with the SEC on 02/12/2025. TransCon Growth Hormone or TransCon HGH is now approved in The United States by the FDA for the replacement of endogenous growth hormone in adults with growth hormone deficiency. In addition to the treatment of pediatric GHD and in The EU has received MAA authorization from the European Commission for the treatment of pediatric GHD. TransCon PTH is approved in The U. S.

[Scott Smith, EVP & CFO at Ascendis Pharma]

By the FDA for the treatment of hypoparathyroidism in adults and the European Commission and The United Kingdom's Medicines and Health 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 efficacy of the product candidates have not been reviewed or approved by any regulatory agency. None of the statements during this conference call regarding our product candidates shall be viewed as promotional. On the call today, we'll discuss our second quarter twenty twenty five financial results and we'll provide further business updates.

[Scott Smith, EVP & CFO at Ascendis Pharma]

Following some prepared remarks, we'll then open up the call for questions. With that, let me turn it over to Jens.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

Thanks, Scott. Good afternoon, everyone. The 2025 demonstrated strong momentum towards fulfilling our Vision 02/1930, as we progressed towards blockbuster status for multiple products and expand our engine for future innovation. The continued strong global launch of Europass increases our confidence that Europass is underway to become a blockbuster product with durable global leadership for the treatment of hypoparathyroidism. FDA granting us priority review for TransCon CNP recognizing its potential if approved to provide a significant improvement in the safety and or effectiveness of the treatment of achondroplasia.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

The announcement of the interim phase two results from the first combination therapy trial of TransCon CNP and TransCon Growth Hormone highlights our potential to boost healthy growth in achondroplasia. And we achieved the first of many planned label expansion for Skytropha when the FDA approved it for treatment of adult growth hormone deficiency. I will review these key developments in more detail in my prepared remarks. Beginning with Europads. Revenues in the second quarter reached €103,000,000 more than double of Q1, despite a strong currency headwind.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

In The U. S. From Longst to June 30, more than 1,500 prescribers wrote prescription for around 3,100 unique patients, reflecting both the deep unmet medical need and compelling product profile. For U. S.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

Patients receiving a prescription for Europads, the majority have received payer approval within three months. Outside of The U. S, we continue to see steady Europad revenue growth in both our Europe direct and international market and we currently expect further acceleration of the revenue growth when Europass reimbursement becomes available in additional Europe direct countries. With a broad label calling all type of chronic hypoparrow supported by international guidelines and a prominent reference to Europat in recently published best practice consensus statement, we expect growth to continue. We have ongoing clinical programs to support label expansion, for example in older children and initiated Partway 60 trial, a single arm safety and efficacy trial to support titration up to sixty microgram doses in The US.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

The primary endpoint of this trial will be efficacy at twenty six weeks, the same as our pivotal Phase three trial endpoint. We are building towards UEFA's long term global leadership based on three key pillars: differentiation, demand and access. I will first speak about differentiation through mode of action. A replacement therapy for hyperparameter must maintain the same mode of action as endogenous PD-eight throughout the body and sustained physiological level of PTH twenty four hours, seven days a week. Based on all the data we have seen, Europet is the only product to demonstrate it can do this.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

With normalization of key elements such as serum calcium phosphate, kidney function, bone turnover, and quality of life. Second is demand. Where Europet is available, we see strong interest and growing enrollment. For The U. S.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

Market, in just two full quarters, we had around 3,100 unique patients enrolled across more than 1,500 prescribers. We're seeing a broad uptake across the entire country. And with our estimate of seventy thousand to 90,000 patients in The U. S, we still have ample room to grow. Outside The U.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

S, we have recognized revenue from more than 30 countries And currently, we have commercial agreement covering more than 75 countries. Third is access. In The U. S, we see favorable access continue to improve, with approvals coming across all payer segments. In Europe direct, we have full commercial launch in Germany, Austria and now Spain.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

We expect additional commercial launches later this year, both in Europe direct and international market. In Japan, our partner, Taizen, expect approval for Europath later this quarter. We consistently hear about how transformative Europad has been for patients and do not believe that any public disclosed drug in clinical development has the potential to meet this efficacy and safety bar set by Europads. As shown in our clinical trial, this has been extended for all patient groups post surgical HB patients to small genetic subtypes like DiDior's syndrome, ADS1 and idiopathic hyperpara. Notably, Urovet has brought approval from the FDA, the European Commission and other regulatory authorities for the treatment of all forms of chronic hyperpara.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

For all of the above reason, we are confident that Europet has the potential to become a doable blockbuster over time And we continue to expand our global leadership position in the treatment of hypoparam. Moving now to TransCon CNP. We believe TransCon CNP is moving the bar on safety, efficacy, and tolerability and reducing treatment burden. And we believe TransCon CNP is well positioned to become the leading monotherapy treatment for achondroplasia. In clinical trials, we have seen the desired linear growth across all ages.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

And to our knowledge, once weekly TransCon SEM P is the only product to show statistical significant improvement beyond linear growth compared to placebo in a pivotal trial, for example, improvements in leg bowing and quality or life. We have demonstrated a safety and tolerability profile comparable to placebo, including no evidence of hypotensive effect and extremely low frequency of MIL type injection site reaction. Since our announcement of monotherapy data, we have engaged with patients, advocates, physicians, and regulators. All have appreciated the differentiating ability of TransCon CNC in comparison to placebo to increase linear growth, while also leading to stronger muscle function, improved body proportionality and leg bowing, and reducing overall the burden of achondroplasia related complications for the majority of treated children. And of course, patients and caregivers appreciate the much lower burden of once weekly injection.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

During the 2025, FDA accepted our NDA submission for priority review with a PDUFA date of November 30, recognizing TransCon CNP as a therapy that could, if approved, provide a signal improvement in safety and or effectiveness. Next, I will review our combination trial results. As we look forward to the anticipated approval of TransCon CRP as monotherapy, we are investigating it in combination with our once weekly TransCon growth hormone in children with achondroplasia in our COACH trial. In June 2025, we announced week 26 interim results, which showed a clear boost in linear growth and body proportionality improvement with a safety and tolerability profile consistent with those observed for monotherapy. In the combination trial, both treatment groups exceeded the 97% factor for growth of an average state of children, meaning they are achieving linear growth at a rate higher than an average child.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

The week 26 data demonstrated potential to boost growth of around 3x or 3x above that observed with monotherapy addressing the hyperactive FTR3 receptor pathway, supporting the scientific rationale for treating the TransCon and CMP and TransCon growth hormone combined. These results are without precedent in achondroplasia. Importantly, we see clear indication that is healthy growth with linear growth accommodated by improvement in body proportionality and without acceleration of bone age. All patients continue in the study as of today. These results enforce the role of TransCon CNP as a strong fundament therapy in achondroplasia.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

We look forward to our twelve month data release later this year and plan to start a Phase three study of the combination therapy in children with achondroplasia by the 2025. In addition, we also expect Truinitim a pivotal combination trial in hypochondroplasia. I will now turn to Skytofra. Skytofra is established as a high value brand and a treatment choice for pediatric growth hormone deficiency. We recently received FDA approval for adult growth hormone deficiency, and with further label expansion planned, scotropha remains a fundamental pillar in our strategy to become the global leader in treatment of growth disorder.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

Q2 revenue for Skytrofe were €51,000,000 We continue to see growth in the number of people treated with Skytrofe based on new patient start. We expect that recent label expansion for adult growth hormone deficiency to further drive long term growth. Our market research shows Skytova is the treatment of choice for pediatric growth hormone deficiency among patients and physicians. And we believe we can achieve the same status for treatment of adult growth hormone deficiency. Our Phase three basket trial of Scatropha planned to begin later this year will include a range of established daily growth hormone indications, including ISS, shock deficient, Turner, and SDA.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

I often say that ascentis, we're just getting started. Following closely behind this major growth opportunity, our research team is developing the next day of innovative TransCon technology and product candidates. In addition, our ongoing collaboration with Novo Nordisk for the development and commercialization of TransCon based product in metabolic and cardiovascular diseases continue to make progress towards the clinic. Ascendis is demonstrating a significant inflection in revenue growth. We are general important new clinical data working towards additional key label expansion.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

We are advancing new blockbuster opportunity to drive growth for many years to come and fulfill our Vision 02/1930. And we're already preparing for our next vision. I will now turn over to Scott.

[Scott Smith, EVP & CFO at Ascendis Pharma]

Thank you, Jan. I will touch on some key points surrounding our second quarter financial results, but for further details, please refer to our Form six ks filed today. For Q2, our total product revenue was €153,700,000 which includes a negative sequential foreign currency exchange rate impact of €7,600,000 Skytropha revenue for the quarter was €50,700,000 including a €1,800,000 negative currency impact. Eurvipath delivered strong performance with revenue more than doubling to €103,000,000 up from €44,700,000 in Q1 twenty twenty five. This revenue growth was achieved despite a negative sequential currency headwind of €5,800,000 Sequential growth across global markets remained strong with continued strong uptake in The U.

[Scott Smith, EVP & CFO at Ascendis Pharma]

S. Acting as a key growth catalyst. The OrvaPath U. S. Launch and continued performance outside The U.

[Scott Smith, EVP & CFO at Ascendis Pharma]

S. Are having a substantial impact on our financial profile and we expect Ascendis to become cash flow positive on a quarterly basis this year. Including €4,400,000 of revenue from our collaboration partners, total Q2 revenue was €158,000,000 Turning to expenses, R and D costs for the second quarter decreased to €72,000,000 compared to €83,500,000 in the same period last year, primarily driven by lower development cost for growth disorders. SG and A expenses in the 2025 increased to €107,600,000 compared to €74,300,000 in the same period last year, primarily driven by global commercial expansion. Total Q2 twenty twenty five operating expenses were about €180,000,000 Net finance income for the 2025 was €22,000,000 driven primarily by non cash items.

[Scott Smith, EVP & CFO at Ascendis Pharma]

Net cash financial expenses for the 2025 were €5,300,000 We ended the 2025 with cash and cash equivalents totaling €494,000,000 compared to €518,000,000 as of March 31. Of the €24,000,000 sequential decrease in cash, 19,000,000 of that was due to the June 30 cash transition to cash translation to euro. So pretty close to overall cash breakeven for the quarter for the company. Turning to the remainder of 2025, we expect continued revenue growth driven by the strength of the global launch of YorbaPath. For SKYYTORIFA for modeling purposes, we continue to believe that sequential revenue growth for 2025 should track growth in prescriptions, offset somewhat by payer mix and normal seasonality.

[Scott Smith, EVP & CFO at Ascendis Pharma]

We also expect long term growth for SKYYTORIFA to be driven by label expansion with our recent adult approval expected to only contribute modestly for 2025. We also continue to watch the Euro U. S. Dollar exchange rate for any potential impact related to reported revenue. For Yorvapath, our launch is progressing exceptionally well.

[Scott Smith, EVP & CFO at Ascendis Pharma]

Globally, we see Yorvapath as a standard of care for treating hypoparathyroidism and we believe it has the potential to achieve multiple billions of euros annually in peak sales over time and our focus is on building long term leadership. In the near term, as investors and analysts seek to model Eurvapast growth trajectory, I would highlight the following. Outside The U. S, we currently see continued steady sequential revenue growth. In The U.

[Scott Smith, EVP & CFO at Ascendis Pharma]

S, seven months into launch, we are seeing strong continued demand and continuation of enrollment trends. We are seeing good conversion from enrollment to paid prescriptions with Yorvitaaf. As Jens mentioned, the majority of U. S. Patients are approved for reimbursement within three months of enrollment.

[Scott Smith, EVP & CFO at Ascendis Pharma]

Payer approvals are broad across commercial and government as well as geographies. We expect additional coverage policies and payer agreements to facilitate patient experience, access and continued long term uptake. Based on our data so far, we expect persistence to be high because of the benefits to the patient and we continue to monitor. And of course, we'll continue to look to help investors understand uptake and reimbursement dynamics as the year progresses. With that operator, we're now ready to take questions.

[Operator]

Thank you. At this time, we will conduct a question and answer session. As a reminder, to ask a question, you will need to press 11 on your telephone and wait for your name to be announced. To withdraw your question, please press 11 again. Our first question comes from Jessica Fey from JPMorgan. The floor is yours.

[Jessica Fye, MD & Equity Research Analyst - Biotechnology at JP Morgan]

Great. Thanks, guys. Great quarter. You mentioned you're seeing a continuation of enrollment trends, and I think the number of unique patients enrolled grew by about thirteen fifty in 2Q. Is that the rate you mean that you see continuing? Thank you.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

Hey, Jess. I can start with a few overall view, and then perhaps Jay can follow-up. The number we reported was around seventeen fifty for the end of Q1. And here, the end of Q2, we reported 3,100. What we also said in our Q1 call that 200 patients, we will consider some kind of bolus injection because the 200 patients came from our EAP program.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

So when I take the numbers out from our Q1 number, the 200, it gives me about fifteen fifty. And so in some way, I actually believe that we see a steady state growth in the patient here between Q1 and Q2, when I take this consideration to the 200 patient that came from the folks. And this is how we basically see the numbers and this is very much aligned with our comments at the Q1 call. We expected to see a steady state development in the prescription. And first, in the second part of the year, we expect to having an acceleration of the conversion of a patient that have a prescription to be on treatment.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

So that is basically what we have seen. And we look forward to see Q3 and Q4. We are extremely optimistic about this launch. This is I'm now just talking about U. S.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

We see the same pattern everywhere when we're launching, but this really is amazing. And we expect to see the same steady state. Sure, that can be a seasonal factor because of the summer vacation, at least we know in France, the August is closed and other places happening the same thing. So Gabe?

[Jay Donovan Wu, EVP & President - US Market at Ascendis Pharma]

Thank you, Yan. And as mentioned before, we are seeing that stabilization in enrollment. And again, we're still early in the launch, so we will need more time to observe what that steady state trend will be out as we get more months under our belt with the launch. More importantly, just as Yan mentioned earlier, we're focused beyond just the point of enrollment, right? We're looking across the entire funnel from enrollments to approval, from approvals to patients on therapy.

[Jay Donovan Wu, EVP & President - US Market at Ascendis Pharma]

And we are seeing continued growth, especially as it relates to the conversion of those patients on the therapy. And we're feeling good about what we are seeing.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

Thanks, Jay.

[Operator]

Thank you for your question. Our next question comes from Derek Achillia from Wells Fargo. The floor is yours.

[Derek Archila, MD & Equity Research Analyst - Biotechnology at Wells Fargo]

Hey, thanks for taking the question and congrats on the progress here. I just wanted to confirm something. So it sounded like you noted that there's three months from enrollment to conversion. I guess I just wanted to know, like, are you doing to kind of improve that? And I guess, how much progress can you make on improving on that three months? Thanks.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

Nothing has really changed compared to what we said on our Q1 call. There is a lot of elements that Jay and the entire integrated commercial team are working with. We can go a little bit more in specific, but in the overall view, sure, the different politics from a different PPM need to be installed. There is a lot of elements that still takes time. And we also working a lot on the procedure, how we basically can help patients to be sure and physician and back office.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

So this is happening for our ASSA program that we are helping the patient. So Nordic has changed compared to what we said in Q1, where we believe that we will first see in the second part of the year when many of these activities will be implemented, we will see an improvement in the time from a prescription to a patient is on treatment. Jay, you can give a little bit more flavor on some of the initiative on a high level because we have so many initiatives going on.

[Jay Donovan Wu, EVP & President - US Market at Ascendis Pharma]

Absolutely, and thank you for the question. From a time to approval standpoint, as we said, we're seeing the majority within three months. Now divide our efforts probably in a few buckets. The first one is upstream, right? We're continuing our payer education, whether it's at a commercial level or a public level, just on the clinical value proposition of the product, the full expanse of the label.

[Jay Donovan Wu, EVP & President - US Market at Ascendis Pharma]

Because as you can all appreciate, whenever there's a new specialty drug on market, particularly a rare disease one, it oftentimes takes a bit of time for them to consider either a category or product that they may not have previously had on formulary or plan. So we anticipate that to continue to approve, which will of course have downstream impacts on the speed. More downstream from that, we also have a very experienced hub, right? I think we've mentioned this before, we're quite experienced with being in managed care spaces. So some of the bread and butter work as it relates to ensuring that providers and patients are continuing to follow-up, fill out their paperwork correctly to decrease cycle times to ensure that that paperwork isn't the reason why maybe something goes back and forth one or two extra times, which will then also increase the cycle.

[Jay Donovan Wu, EVP & President - US Market at Ascendis Pharma]

And then lastly, would say, as we continue to have patients within the funnel, just making sure again, that our partnership with our specialty pharmacies, so on and so forth, again, streamlining those processes to continue shave off time as we work through this initial launch phase. All that to say, we're incredibly encouraged by the speed in which we're seeing and a reflection of the experienced hub that we have. And we continue to look forward to seeing how that will progress.

[Derek Archila, MD & Equity Research Analyst - Biotechnology at Wells Fargo]

Great, thank you.

[Operator]

Thank you for your question. Our next question comes from Jazeen Hamad from Bank of America. The floor is yours.

[Tazeen Ahmad, MD - US Equity Research at Bank of America]

Hey, guys. Thanks for taking my question. I was wondering if you could give some color on the type of patients. There's been a lot of talk about initially severe patients, the most severe patients being put on your behalf first. But do you have any color on what the split is between definitionally what a severe patient is versus the type of patients that are getting on that might be on the more moderate side? Thanks.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

Thanks, Sunny, for the question. First of all, there is no medical definition that defines severity of hyperpara. So we cannot go in and claim database and saying this is a severity that you have in the disease because all of them is not classified related to that. When we talked about the element of being uncontrolled, partly controlled or something we call controlled, is was mainly related to one single parameter, looking on the claim databases and see how often they're sitting and physician. How often they're sitting and physician, I think, has a lot of multiple aspects that some way are not only reflecting where you're living in the country, what kind of medical access you have, and also how often are there an endo that really can see you.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

So, we did it out from this perspective because we wanted to be quite sure we are addressing a physician that sees a high number of patients in hypoparra and it was why we addressed this physician as our priority in our, you can say commercial strategies. And so we can certainly not define and answer your question because there is no way this is part of the reimbursement system, it's no part of we can see of the patient because it's not a medical term that ever is defined. But I think, to give you also the other aspect on it, When we look on the guidelines that we see being integrated many different places, they are not using this kind of term either. So the guidelines just having a broad aspect on all the different elements that really qualify to be on PTH treatment. And in general, all the guidelines we have seen being issued from all different places in the world now, are indicating that ninety five percent of all patients should be on PTH treatment.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

And I think that is pretty logical. Think about how many patients on type one diabetes. Will you ever consider that you will not take all patients that have type one diabetes on insulin treatment? And I think you will see the same thing happening with hypoparra.

[Operator]

Thank you for your question. Our next question comes from Yaron Werber from TD Cowen. The floor is yours.

[Yaron Werber, MD & Senior Biotechnology Analyst at Cowen and Company]

Great. Thanks for my question and congrats again. A couple of interrelated questions. Can you give us a little bit of a sense, Scott, you mentioned last quarter to expect Europe to grow about 4,000,000 to $5,000,000 so that puts you at around 24,000,000 So it almost seems like you did sort of 79,000,000 to 80,000,000 in The U. S.

[Yaron Werber, MD & Senior Biotechnology Analyst at Cowen and Company]

Am I sort of in the ballpark? And I guess secondly, it's when one looks at we shouldn't be expecting that you're going to be growing patients 1,500 quarter over quarter. So can you give us a little bit of a sense how to think about what a sequential normalized growth at this point in The U. S. Can be so we don't get out of hand? Thank you.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

I can think I can help Scott this time, which I often do. Because what we said in our Q1 call, if you look on Q3 to Q4 revenue increase, net revenue in Europe was about 4,000,000 to €5,000,000 and in this two quarter it was basic Europe or ex US revenue. And we also say that that time that we expect that to continue in 2025, when we see more country coming on full commercialization as we just got Spain now, and we expect a few country more, perhaps it will increase. But it will first have a material impact two to three months after basic initiation of full commercialization. So therefore, I will say your assumption is pretty correct and also reflecting what we said in Q1 this way.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

Related to the question you commented, it's some way a forward looking statement. I know it's really being covered by Scott's fast reading. But from my perspective is that we see a strong, strong launch here in The U. S. We have seen nearly the same numbers between Q1 and Q2.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

And we're really looking forward, as Jay said correctly, this is an early in the lungs. It will be too early for us to come up with any kind of prediction how we really will see the next six, seven quarters to go.

[Operator]

Thank you for your question. Our next question comes from Gavin Clark Gartner from Evercore ISI. The floor is yours.

[Gavin Clark-Gartner, MD - Biotechnology Equity Research at Evercore ISI]

Hey, guys. Congrats on another great quarter. First, what do you believe the ultimate conversion rate from the enrollment forms to paid drugs will be at any point in time? And then secondly, looking ahead, do you plan to keep reporting the enrollment forms for Yorvapath? Thank you.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

It's really difficult for us to give you a clear number but what we always will see in The US, there will be a percentage of patients that have really difficulties to get reimbursed even if we try and help them multiple times. And what we see during the lungs, what we have seen before is that what I call the tail is getting faster and faster cleared out. And I can guarantee we will do everything in Ascendis manner to help that all the patient can come on treatment. Can we guarantee that everyone can go on treatment? No.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

There will be a number of patients even after six, nine, twelve months really struggling, we will say it is really hard to be covered. So you can ask about my personal success. My personal success will be if we get in steady state lungs really with a mature product could get around nine zero percent of all patients on treatment. I will feel it as a personal success and my contribution to help patients with hypoparam. Jay, you can also come with your personal success number if you want to do that.

[Jay Donovan Wu, EVP & President - US Market at Ascendis Pharma]

Appreciate the question. I would say layer on a couple things, right? I think the enrollment to approval, again, it's not just driven by payers. Of course, that's a component of it, right? Because, as we've discussed before, there are certain plans and policies for which you have to go through exception or appeals process.

[Jay Donovan Wu, EVP & President - US Market at Ascendis Pharma]

And I think as Yan alluded to, that right tail will take some time to clear depending on the plan and as things evolve. The other component from enrollment to approvals is entirely unrelated to payers, and it may just be more driven by ensuring, again, providers are leveraging the paperwork appropriately, patients are following up with outreach, A lot of that which we'll continue to pursue across the spectrum because we know again that these patients can and should benefit from the product if they're already in the funnel. And we will do everything we can to clear that long tail out knowing that it will take some time. And when you look at a lot of rare disease analogs in these types of spaces, it can take some time to get there. But this is a long haul and we're looking at it more from that lens to making sure we're optimizing every step of the way.

[Gavin Clark-Gartner, MD - Biotechnology Equity Research at Evercore ISI]

And are you guys planning to report enrollment forms for your overpass in the next quarter also?

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

I think we will give you the necessary KPIs that we're doing today, and we will continue to do that in every quarter until we feel we're coming to a state steady state, where we feel that there is enough information just out from revenue that you can basically do your modeling. Until that, we will continue to provide the necessary data that support that you can make a solid modeling of the launches. Just to say and repeat it me again, this is amazing launch. Q1 was great. Q2 was also amazing great and we have not seen any weakness in the lungs.

[Gavin Clark-Gartner, MD - Biotechnology Equity Research at Evercore ISI]

Very helpful. Thanks.

[Operator]

Thank you for your question. Our next question comes from Il Watsak from Cantor. The floor is yours.

[Daniel Bronder, Equity Research Associate at Cantor Fitzgerald]

Hi, congratulations on the quarter. This is Daniel Brundra on for Lee Watsak. We're just curious about the pull through of the patients that get onto Yorvy Path. How should we think about compliance, especially if you're saying that fifteen hundred PFS number is net patients going forward? Thank you.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

Where we have the best long term data is from Europe where we basically started the lungs about six to nine months before. And when we look on a rate, what we call true discontinuation, it's extremely low, a few percentage. So we really see the benefit of the therapy. People taking the therapy, taking it, they're keeping doing it, and I believe that is the contribution on how we are addressing a major unmet medical need with the treatment of Europax. So everything what we see here is far away from what you see with a diabetes drug.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

People stay on it even much better than insulin that you see in type one diabetes. And I think this is the main contribution to the positive CNS effect that is with this product.

[Daniel Bronder, Equity Research Associate at Cantor Fitzgerald]

Okay, cool. Thank you. And just on the just going back to Yaron's question earlier about the 1,500 patient net enrollment per quarter. Just for me to fully understand, this is the patient start forms that you're referring to?

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

Yeah. That is what we're referring to is unique prescriptions, meaning is that it's a new patient that have got a prescription. This is what we call unique prescriptions.

[Daniel Bronder, Equity Research Associate at Cantor Fitzgerald]

Great. Thank you so much, and congrats again. Thanks.

[Operator]

Thank you for your question. Our next question comes from Joseph Schwartz from Leerink Partners. The floor is yours.

[Joori Park, VP - Equity Research at Leerink Partners]

Yeah, hi. I'm Jiri Park dialing in for Joe. Thank you for taking our questions. The first one is on your Overpath. I believe that there were 1,500 prescribing health care providers in The US by the end of the quarter.

[Joori Park, VP - Equity Research at Leerink Partners]

Can you help us understand how much of your target physician base this represents? And secondly, on CNP, a competitor recently announced that their long acting CNPs area under the curve PK level was three times greater than the levels of transcon CNP. Based on your experience with transcon CNP, how could that translate in the clinic in your view? And how does that profile differ from your combination approach? Thank you.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

Yeah. Let me start with the easy one or potential. I will, in summary, take that over to Jay, and I will take the more I can say scientific interesting question as number two.

[Jay Donovan Wu, EVP & President - US Market at Ascendis Pharma]

Sure. So, happy to start with the first question. From a target list standpoint, we're targeting about 8,000 to 10,000, So that you can consider that as our universe about 3,000 of which we decile as high medium. And we're seeing pretty good field execution metrics across that. So over 80% reach across our high medium priority targets.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

So, the second question is interesting because for many, many years there was a lot of skeptic about our sustained profile. And then suddenly there was a big change where somebody say there is some benefit by having a sustained profile that does not give a high Cmax that basically can indicate risk of hypotension, and also you need to have continuous exposure over one week. And so we started and designed our TransCon CNP in 2015 and now we are in 2025. They're starting now to go after that concept. From my perspective is that when you look on how we designed it and also all our associated patent filing we have with it, all the IP we have of the optimal product, the medical treatment benefit it is and other things like that, people are now trying to copy with other concepts.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

And when I think about the concept, I think there was some kind of making success of a product without disclosing the key element. The key element that was AOC, I really don't care about AOC. I want to know key elements. What is really the half life? How is this exposure really happening?

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

Is it something that beaks up to a very, very, very high value there shortly and then you basically are going up to, as they call it, the danger zone of hypertension. That is not really an optimal product in this way. I believe what we saw in all our clinical data, it's really hard to do a lot when you remove a brake because it's really the hill is rolling down that really decides the speed. If you have taken the brake off, the brake is off. That is where I really don't get the biological and scientific concept.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

That is related to linear growth. When we look some of the other effects where we see muscle strengthening and other things like that, quite sure having continuous exposure. But we do not know exactly if that is maxed out or not. So all from that perspective is that doubtful. Can you get more out on the having a higher AOC?

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

First of all, you need to have the right AOC. This must be a higher exposure completely over the once weekly profile. And no one disclosed that because then they need to have a longer half life than ours that is about two and a half to three days. And I have not got any kind of disclosure than they are there. When I go out to our combination therapy, it's basically a completely different concept because the concept of that is between synergies, between different biological pathways, which are well known from so many other therapeutic areas.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

To have the optimal treatment, you cannot overcompensate just by one pathway, but you're basically providing the benefit in a holistic manner in a much more normal manner by balanced different pathway. And that is what we do in the combination between transconcidental and the growth hormone effect, which are basic and at the same time in a more simplified manner, remove the break with what we do with the CMP and then having a speed up on that. So I feel really, really confident with our code state, our combination therapy, that really is a unique way where you really can totally provide for patient, for physician a complete new treatment standard.

[Joori Park, VP - Equity Research at Leerink Partners]

Got it. Thank you.

[Operator]

Thank you for your question. Our next question comes from Ilana Murrell from UBS. The floor is yours.

[Ellie Merle, ED - Biotech Equity Research at UBS Group]

Hey, guys. Thanks for taking the question, and congrats on the strong quarter. Curious, for achondroplasia, what's your base case for the indication statement for TransCon CNP, whether it be for the treatment of achondroplasia or for the increase increase in linear growth in in achondroplasia, like VoxoGo has? And I guess any expectations for differentiation in the label relative to VoxoGo, such as in terms of the indication statement or, say, other secondary endpoints? And then second, a follow-up question, just what's your perspective on the IP landscape for weekly CNPs?

[Ellie Merle, ED - Biotech Equity Research at UBS Group]

Specifically, thoughts on BMM-three thirty three and where that might stand relative to the TransCon CNP IP estate? Thanks.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

Yeah. We are progressing to the regulatory review with our TransCon CNP exactly as we hoped for in an accelerated priority review. Everything is happening on the right time. So labeling discussion is one of the last part in the review cycle, so it's really, really difficult for me to come up with any kind of elements. What I'm more referring to you is the data we have that is really backing up TransCon CNP, and I believe this is why we got the priority review because we have data that give a strong evidence that we can provide treatment benefit beyond linear growth.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

And now we're talking about everything what we have seen related to leg bone, everything what we have seen to muscle strength because people are saying that they have also seen it, but you're not seeing it in a real manner, you need to see it in a placebo controlled manner because either body proportionality is actually improving during a normal development of a child also to an achondroplasia child. So how can you discriminate? Is it really a treatment benefit or just a normal development? And this is why it's so extremely important to be in a position that you're referring to data that is done in a placebo controlled manner in a pivotal trial. I'm not referring to the other benefit we have, no risk of hypertension, low injection type.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

This is why the and sure obvious patient parents parents parents parents really love their once weekly profile. It's such a little burden for them to give it in. So I think what I see here, I'm not so much really concerned about exactly what is coming into the labeling. I'm more interested in the benefit that we can go out and explain that TransCon CNP is providing, which I basically have never seen in any other well controlled fibula trial. I think that is the key element for me and I think this is what we see everywhere.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

And the second question was about the IP. I believe when we developed that in 2015, we basically filed a lot of IP. There was how to make an optimal product. There was the benefit of having a product that gave sustained things. As there is no clear for me exactly what is the primary structure that they have in the BMN three thirty three, it's impossible for me to say exactly what they are.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

But if you can see me, at least you can see I have a great smile on my face, because we are pretty good in what we're doing when we file IP.

[Ellie Merle, ED - Biotech Equity Research at UBS Group]

Understood. Thank you.

[Operator]

Thank you for your question. Our next question comes from Kelly Hsieh from Jefferies. The floor is yours.

[Kelly Shi, SVP & Senior Research Analyst - Biotechnology at Jefferies]

Congrats on another strong quarter and thank you for taking my questions. For YORI PAS, what is the typical titration period that you're seeing right now across now broader patient spectrum in real world? And once the patient completed titration period, should we expect a higher monthly cost? Thank you.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

So at least I got the first question related to the titration period. And I think Amy, our chief medical office, know a lot what we have seen in our clinical trials. I think we are much more uncertain about what is exactly happening in what we call in real life clinical element. But what the key element for me, and I take it from that perspective, do we see a lot of patient unstatified action at that stage? Do we see a lot of patient drop out because there's problem with it?

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

And we don't see that. So I cannot really comment about what is exactly happening in the titration in for the patient in real world, but at least we can see that it's happening very successful. And your second question was?

[Kelly Shi, SVP & Senior Research Analyst - Biotechnology at Jefferies]

So once the patient completes titration, should we expect a higher monthly cost? Because they stay on higher dose rate.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

I think in The US, we have an approval up to, from six to thirty micrograms. So, basically, we are in a position that we're only using one pen at the time. Outside US, there have a possibility to use up to sixty microgram. We have just initiated in what we call our sixty microgram trial, which will facilitate, this is our aim, this is a twenty six week trial to facilitate that we can get on labeling that can use up to sixty micrograms in The US. And I think when Jay have seen all the data and we're coming near commercial, what I call commercial launch of up to the sixty microgram, There will be a discussion from Gay's side exactly how we are handling the reimbursement for that situation.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

I think it's too early for us to comment on that.

[Kelly Shi, SVP & Senior Research Analyst - Biotechnology at Jefferies]

Thank you.

[Operator]

Thank you for your question. Our next question comes from Paul Chao from Goldman Sachs. Your line is yours.

[Paul Choi, Biotechnology Analyst at Goldman Sachs]

Hi, good afternoon and congratulations on the strong quarter results. Yan, just to follow-up on your last comment about potentially harmonizing The U. S. Label with the EU label and the sixty microgram dose. When might you be in a position to submit that data to the FDA?

[Paul Choi, Biotechnology Analyst at Goldman Sachs]

And then commercially, what portion of the patient population would that potentially allow you to address as not being currently suited by the available presentations in The U. S. Market? Thank you for taking our questions.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

Thanks a lot for the question. I actually think this is lot of questions in the question because what is happening in The US today, and I think there is a lot of different places where different elements on how to solve the issue if a patient needs more than thirty grams. There is some patients because of the label restricting to thirty micrograms that will stay in a position that they're taking thirty microgram, and if needed, they will potentially take additional calcium supplement or additional active vitamin D. There is other places where the basic patient on a physician off label will potentially get access to a higher dose, which we obviously have no any involvement in and no recommendation. So from my perspective is that there is a need for a dose higher than 30, and we would do everything we can do to get it as fast as possible out to the patients.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

We're starting the trial now. It's a small trial. It's less than 20 patients. We target enrollment of 80. It's only twenty six week, and we are utilizing the same pen devices that we basic have been already in the market.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

So there is no CMC component in this way. It's just for Amy Shu and all her people to basic get the clinical trial done and get through the regulatory team to get it filed and approved. And we will do that as fast as possible, but it looks pretty, pretty promising to get it in.

[Operator]

Thank you for your question. Our next question comes from Alex Thompson from Stifel. The floor is yours.

[Alex Thompson, Managing Director at Stifel Institutional]

Hey, great. Thanks for taking my questions. I guess on your repast as well, you've talked about the breadth of prescribers. I wonder if you could comment on the proportion of prescribers that you've seen, with multiple prescriptions, multiple patients on therapy, and how you see that trend changing over time. Thanks.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

We cannot really address that question. We don't have sufficient data that we really will feel confident to come with data that really support and strong trend analysis currently.

[Operator]

Thank you for your question. Our next question comes from Luca Isaac from RBC. The floor is yours.

[Luca Issi, Senior Biotechnology Analyst at RBC Capital Markets]

Great. Thanks so much for taking my question and congrats on the quarter. Maybe Scott, lots of questions, obviously in the top line, rightly so. But how about SG and A, I mean, up 44% year over year and 6% quarter over quarter. Appreciate obviously you're launching a drug, but how should we think about modeling SG and A for the rest of the year?

[Luca Issi, Senior Biotechnology Analyst at RBC Capital Markets]

I guess what I'm trying to ask here is how should we think about potential to achieve profitability in Q3 versus Q4? And then maybe secondarily, can you just remind us about Ascendis exposures around tariffs and MFN? Again, appreciate the situation is still fluid and we don't have all the details, but any high level commentary, much appreciated. Thank you, Dan.

[Scott Smith, EVP & CFO at Ascendis Pharma]

Okay, great. On SG and A and expenses overall, so remember last quarter we did EUR 190,000,000 of OpEx and we said that wasn't a bad run rate, maybe plus or minus each quarter. So this quarter we're minus about 10,000,000 from that. So we're about 180,000,000 of OpEx this quarter. And as you point out, it's probably not best to look year over year because we had a lot of growth in the last year.

[Scott Smith, EVP & CFO at Ascendis Pharma]

It's really the sequential build. So I would look at the 6% sequential growth for SG and A is potentially not a bad number to think about. But in the overall context of, I would still say about 190 OpEx per quarter is not a bad number to think about. With respect to profitability, yes we expect that this year. I mean if you look at our financials and back out June 30 currency, thanks to our whatever it was called Liberation Day that cost about 20 Euro of cash delta.

[Scott Smith, EVP & CFO at Ascendis Pharma]

So overall, everything across the company it was about 5 Euro of burn in Q2. And actually on an operating basis we're just slightly positive on cash. So, that should be relatively doable this year. With respect to MFN and tariff, I think you said it pretty well. There's really too much in development right now to make any comments specifically on it.

[Scott Smith, EVP & CFO at Ascendis Pharma]

But we do believe that we're as a flexible company, we're pretty well positioned to mitigate impact of any policy should it emerge.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

Just to clarify and also add in Scott's comments, we're not importing finished product to The U. S. We import them in different states and finalize them inside The US. So in whatever way we look at it, we cannot see how it really should provide a major material impact on our business and how we operate.

[Luca Issi, Senior Biotechnology Analyst at RBC Capital Markets]

Thank you so much.

[Operator]

Thank you for your question. Our next question comes from Leland Garcell from Oppenheimer. The floor is yours.

[Leland Gershell, MD & Senior Biotechnology Analyst at Oppenheimer & Co. Inc.]

Hey, good afternoon and thanks for taking our questions. Yan, just curious, in the past you had not expressed much interest in hypochondroplasia as a development program and maybe you had started to lean a bit toward that earlier this year and now we're seeing a formal announcement of intention to go in that direction. So I'm just wondering, what may have changed that affected your decision process here. And if you could also maybe just share briefly what what you think is the opportunity for Ascendis in, hypochondral? Thank you.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

Yeah. Hypochondroplasia is what I will call, and perhaps it's wrong way to define it, but I call it a milder form of achondroplasia. They don't have many of them the same, what we call body disproportionality, but you can say some in old days, many of these patients basically come in the ISS, neuropathic short status. And now because of much more influence on genetic testing, you basically have a development large group that more is well defined from genetic testing to be hypochondriacal. This is one point that basically are saying patient that was in one temporary group is now being moved over in a different temporary group, and meaning they have moved from ISS over to hypochondroplasia, one thing.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

The second thing that is that when I saw the element of combination therapy with TransCon CNP and TransCon Growth Hormone, then even if you have a very much heterogenic patient population, then by having a combination product, you basic can ensure all of them will do extremely well. So you can say that is what really are bringing up my attention, why I believe we need to do it. And also because I went out and talked with a lot of patients. And I actually think I like to talk with the patient and the patient organization because talking with them, get much more idea about the unmet medical need we need to address. So, basically, this is the three pillars I have changed.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

And I say a rechange of the therapeutic groups where the hypochondriplasia is growing because of allocating a patient from ISS over to hypercontemplation, and their heterogenic group that really I feel when I talk to the patients, when I talk to the patient organization, need a treatment. And the very extremely positive data we got for WAS COVE trial, where when you have such a histogenic, some of the key element will be the CMP, growth hormone will help, but once CMP is the key element, CMP will help. And this is why when you have such a histogenic group, having the combination therapy will be the most realistic way to treat them in a way where you really will have a fundamental good treatment regime.

[Leland Gershell, MD & Senior Biotechnology Analyst at Oppenheimer & Co. Inc.]

Great, thank you for that, Alexeyla.

[Operator]

Thank you for your question. Our next question comes from the line of David Lebowitz from Citi. The floor is yours.

[David Lebowitz, Senior Research Analyst - Biotechnology at Citi]

Thank you very much for taking my question. Curious, has there been any evolution in your thinking on the ultimate size of the market for your past? Curious to know.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

You know, I've always been bullish and said that it's going to be a $5,000,000,000 $8,000,000,000 market segment. I have no doubt that it will be that.

[David Lebowitz, Senior Research Analyst - Biotechnology at Citi]

Thank you for taking my question.

[Operator]

Thank you for your question. Our next question comes from Yong Zhong from Wedbush. The floor is yours.

[Yun Zhong, SVP - Equity Research at Wedbush Securities]

Hi. Thanks very much for taking the questions. And the first question on TransCon CNP for achondroplasia, assume that you receive FDA approval by PDUFA date, how quickly will you be able to launch the product? And second question on hypochondriplasia and based on your comments just now, but the press release seems to be saying that either still either monotherapy or combination therapy. So is it still a possibility that you might end up going with a monotherapy for hypochondriplasia?

[Yun Zhong, SVP - Equity Research at Wedbush Securities]

And if that's the case, then what would be the reason why you don't go with a combo therapy given the obvious benefit from COADS study?

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

The first one is, we actually made some kind of preseason with Kaltrofem. We will wait until we get the approval and likely discussed will force me to wake up in the morning very, very, very early, so there would be a press release or some kind of call at 05:00 my time in the morning. And I think at that time, we will explain exactly when we have seen the labeling, got all the CMC information back from FDA and other places when we will launch the product. And Jay will also be forced to be up in the morning, and he will explain how we're going to do our launch strategy in this way. So you need to wait a little bit to this early morning call and we will try not to do it on Sunday, but we will try to take it every other day.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

So the second part is a little bit about what we talked about before. Hypochondroplasia is, in my view, is extremely heterogenic population now with a lot of what I call burden in different way and also now with the reallocation of patient from the ISS group into the hypochondriplasia. It will some way, from my perspective, OPTIMAL will be to use the combination. And we will continue our dialogue with our regulatory agencies around the world to be sure that they also have the aligned view. I cannot in some way eliminate the discussion if we will have in the clinical trial a single arm also reflecting TransCon CNP as a monotherapy, but it's our belief that the combination therapy will be what is best for the patients in this way.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

So this is where are for the time being. We believe that the combination is the most robust treatment on it, but obvious there will be some patients that also just would benefit of having TransCon CNP as a monotherapy.

[Yun Zhong, SVP - Equity Research at Wedbush Securities]

Thank you.

[Operator]

Thank you for your question. Our final question comes from Maxwell Skar from Morgan Stanley. The floor is yours.

[Maxwell Skor, VP - Biotech Equity Research at Morgan Stanley]

Great. Thank you for squeezing me in, and forgive me if this question's been asked. But given the magnitude of growth velocity improvement in the COACH trial, do you believe a single pivotal trial could be sufficient for approval of the combination? Have you received any preliminary feedback from the FDA or EMA? Thank you very much.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

Hey, yeah. I had a lot of discussion with He's sitting in my side because when we started this trial, we talked about our own expectations. And now we're sitting with a growth velocity that is so unprecedent and never been seen in achondroplasia. They are basic growing faster than a normal child.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

I have four children and I remember when they had growth spurt. It was not really good. We're pretty tall, we're Scandinavian, so you really grow a lot. So, you basically will be in a position that is this is really a big change in that. And some way I see it in and we some way go back and forward in that discussion.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

Will you take a treatment of one year? Will you take a treatment of two years? In the combination, You will get major growth and other positive development. Will you then go back for one or two years, just have TransCon CNP as a monotherapy, and then you will boost again if there is desire from the parents for the child and other things related to that and if it's really unneeded at that time. So in some way, when I think about the overall way how we will do the best for the patient in the achondroplasia is basically to give them the option that we really can be in a position where they can take combination therapy likely for one year, potentially two years and other things like that and get all the benefit and that get and then we can continue with our TransCon CNP and if they have a desire to move into another one yearly or two yearly treatment, they can do that again.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

That is more or less our thinking in this way.

[Maxwell Skor, VP - Biotech Equity Research at Morgan Stanley]

Great. Thank you very much.

[Operator]

Thank you for your questions. That does conclude the question and answer session portion of this meeting. This also concludes the meeting itself. I'd like to thank you for your participation in today's conference. This does conclude the program. You may now disconnect.

[Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma]

Thanks so much. Thank you.

Participants

Analysts

• Scott Smith, EVP & CFO at Ascendis Pharma
• Jan Møller Mikkelsen, President, CEO & Director at Ascendis Pharma
• Jessica Fye, MD & Equity Research Analyst - Biotechnology at JP Morgan
• Jay Donovan Wu, EVP & President - US Market at Ascendis Pharma
• Derek Archila, MD & Equity Research Analyst - Biotechnology at Wells Fargo
• Tazeen Ahmad, MD - US Equity Research at Bank of America
• Yaron Werber, MD & Senior Biotechnology Analyst at Cowen and Company
• Gavin Clark-Gartner, MD - Biotechnology Equity Research at Evercore ISI
• Daniel Bronder, Equity Research Associate at Cantor Fitzgerald
• Joori Park, VP - Equity Research at Leerink Partners
• Ellie Merle, ED - Biotech Equity Research at UBS Group
• Kelly Shi, SVP & Senior Research Analyst - Biotechnology at Jefferies
• Paul Choi, Biotechnology Analyst at Goldman Sachs
• Alex Thompson, Managing Director at Stifel Institutional
• Luca Issi, Senior Biotechnology Analyst at RBC Capital Markets
• Leland Gershell, MD & Senior Biotechnology Analyst at Oppenheimer & Co. Inc.
• David Lebowitz, Senior Research Analyst - Biotechnology at Citi
• Yun Zhong, SVP - Equity Research at Wedbush Securities
• Maxwell Skor, VP - Biotech Equity Research at Morgan Stanley