Ascendis Pharma's Q3 2025: Contradictions Emerge on Conversion Rates, U.S. Enrollment, Reimbursement, and Seasonality Impact

Date of Call: November 12, 2025

Financials Results

• Revenue: EUR 214.0M total Q3 2025 revenue (YORVIPATH EUR 143.1M, up from EUR 103M in Q2; SKYTROFA EUR 50.7M, 3% demand growth vs prior quarter; includes EUR 20M collaboration revenue including EUR 13M milestone)
• Operating Margin: ≈5.1% (operating profit EUR 11M on EUR 214M revenue; total operating expenses EUR 180M)

Guidance:

• Continued revenue growth expected in Q4 driven primarily by the global launch of YORVIPATH and new patient adds
• YORVIPATH: Q4 growth expected from new patients, stable pricing, payer mix and contracting; longer-term growth from geographic expansion and new patient uptake
• SKYTROFA: sequential revenue growth expected to track prescription growth with geographic and label expansion supporting longer-term upside

Business Commentary:

* **Revenue Growth and YORVIPATH Performance: - Ascendis Pharma reported revenue of EUR 143.1 million for YORVIPATH in Q3 2025, up from EUR 103 million in Q2 2025. - This growth was driven by a steady increase in new unique patient prescriptions and expanding geographic market availability.

• **SKYTROFA's Growth and Market Expansion:
• SKYTROFA contributed EUR 50.7 million in Q3 2025, with 3% growth in demand.

• The growth was supported by the FDA approval for adult growth hormone deficiency and the initiation of a Phase III basket trial for various growth disorders.

• **Positive Operating Income and Financial Transformation:

• Ascendis Pharma achieved positive operating income for the first time, signaling the beginning of sustained revenue and earnings growth.

• This was due to significant commercialization efforts and operational efficiencies leading to reduced R&D and SG&A expenses.

• TransCon CNP and Regulatory Progress:

• Ascendis is progressing towards the potential approval of TransCon CNP, with a PDUFA date of November 30.
• The company is advancing its leadership in growth disorders, with TransCon CNP expected to be a leading treatment for children with achondroplasia due to its comprehensive clinical benefits.
  

  Sentiment Analysis:

  Overall Tone: Positive

  • Management highlighted a "strong global launch" for YORVIPATH (EUR 143.1M Q3), SKYTROFA revenue of EUR 50.7M, and reported positive operating income (EUR 11M), stating these signal a transformation to sustained revenue and cash-flow growth and expressing confidence about TransCon CNP regulatory progress.

Q&A:

• Question from Jessica Fye (JPMorgan Chase & Co, Research Division): I was hoping you could speak to your expectations for the rate of new patient enrollments on YORVIPATH in the U.S. from here. I think you talked about more than 4,250 as of the end of 3Q, putting you at -- what is that, about 1,150 adds or maybe a little more relative to June 30. Can we think of that as kind of like a good number to work off of from here? Should it continue to kind of drift lower a little bit? Just hoping you can frame some expectations there.
  Response: Enrollment has been stable quarter-to-quarter (Oct >400 new US prescriptions), management expects steady quarter-by-quarter growth as patients remain on lifelong therapy.

• Question from Tazeen Ahmad (BofA Securities, Research Division): I just wanted to get a sense of how you're thinking about the rest of this quarter. Are you expecting to see impact from seasonality, I guess, we can call it just because of the upcoming holidays, Thanksgiving, Christmas and New Year's? And do you think that the script trends for December would be directionally lower, let's say, than what you're seeing -- what you saw for October? And then if I could ask about the TransCon CNP review. You said you're in final labeling discussions, which is good to hear. Can you just confirm whether or not you've had any requests for any type of data from the agency in the review cycle?
  Response: No additional data requested by FDA for TransCon CNP; management is not concerned about holiday seasonality and expects Q4 to be stable/positive.

• Question from Gavin Clark-Gartner (Evercore ISI Institutional Equities, Research Division): I wanted to focus in on the conversion rate for YORVIPATH. I'm wondering why it's only 70%. And you noted that you expect it to be higher over time. How much higher do you expect it to be? And when do you think it will be higher? Just had a follow-up on this, too.
  Response: The ~70% approval rate is expected to rise over time as cohorts mature; early cohorts already show higher conversion, but timing to peak is uncertain and may take years.

• Question from Joseph Schwartz (Leerink Partners LLC, Research Division): Can you give us your latest views on how YORVI has been penetrating the different segments of the hypopara market as you see it? Where has it been getting the most traction? And where could it do better? And then you previously emphasized the desire to do more than enhance linear growth in achondroplasia. So I'm wondering to what extent do you think you can obtain differentiated label claims on the TransCon CNP label?
  Response: YORVIPATH is penetrating best among highly symptomatic patients and post-surgical cohorts; broader penetration requires patient activation and education; TransCon CNP labeling discussions are ongoing and management expects to emphasize unique clinical benefits (e.g., leg bowing, body proportionality) though final label specifics remain under review.

• Question from Li Wang Watsek (Cantor Fitzgerald & Co., Research Division): I guess just on [indiscernible], can you maybe just give us a little bit color on payer mix and potential contracting, not just in Q4, but also in 2026? And should we sort of expect still minimum contracting and sort of stable gross to net for the next few quarters? ... And then on TransCon CNP and the Phase II meeting. In terms of the Phase III trial design, should we assume that FDA would require a 1-year data on annualized growth velocity? And anything that you can share on the powering assumptions?
  Response: Management expects no material contracting changes or GTN shifts in Q4 and only minor contracting in future; FDA signaled a typical 1-year controlled data expectation for pivotal growth endpoints and reviewers were open-minded about duration.

• Question from Yaron Werber (TD Cowen, Research Division): Maybe -- Jan, maybe a couple of questions. Number one, just on gross to net. When we're kind of -- we were at around just over 4,200 and we had like a 65% approval, but we were getting to higher numbers for the quarter. So I'm wondering whether -- and we had 18% gross to net. Is it possible that gross to net discounts are higher than that? That wouldn't make any sense. And I guess the question kind of like what am I missing? And then maybe secondly, are you -- it sounds like you may be very subtly intimating to expect some impact from the holidays in Q4. Are we reading it correctly because we didn't see much seasonality in Q3. And then finally, U.S. versus European sales for YORVI in the quarter, was it like around EUR 4 million or so in Europe this quarter as well?
  Response: Management reiterated ex-U.S. growth of ~EUR4–5M per quarter, does not expect Q4 seasonality, and cautioned that payer approval does not equal immediate on-drug starts due to timing lags.

• Question from Martin Auster (Raymond James & Associates, Inc., Research Division): First, on YORVIPATH, I was wondering if you could comment if you've got any sense of early data on what patient retention looks like from folks who started up on drug this year? And then second, on TransCon CNP. I guess from a commercial perspective, when we look at this market, it looks a little underpenetrated for a rare disease market compared to some other comps. I'm curious if you guys have a sense as to sort of why that's the case? And if you think TransCon CNP is sort of coming to market can improve upon that and improve overall penetration rates of treated folks with achondroplasia?
  Response: Early retention is high after initial titration (most attrition occurs in first 4–6 weeks); management believes TransCon CNP can improve market penetration by addressing comorbidities beyond linear growth.

• Question from Kyuwon Choi (Goldman Sachs Group, Inc., Research Division): Are you primarily going to target de novo patients? Or are you expecting a good portion of the revenue mix to be from CNP daily injection experienced patients? And if you are expecting a decent-sized contribution from the latter, can you maybe speak to what your market research suggests the appetite is on potential switch strategies and just sort of what percentage of the existing treated patient base you might think ultimately convert?
  Response: Management expects both de novo and switch uptake: significant switching where daily CNP penetration is high (European markets), while U.S. will see more new patients given lower current penetration.

• Question from Yun Zhong (Wedbush Securities Inc., Research Division): So the first question on the label extension to the higher dose. I assume the pricing is going to be the same. So would you expect any direct impact on maybe the number of patients on treatment and reported revenue, please? ... then a follow-up question on the payer discussion. I believe that initially, you said roughly it takes about 8 weeks to get payer approval then I think last quarter, you said 3 months. And then this quarter, just now, you probably said 8 months. Was that just a random maybe fluctuation? Or was there any meaningful change in terms of how long it takes for payers to approve coverage, please?
  Response: Higher-dose expansion involves an 18-patient, 6-month safety titration study and is not expected to materially affect revenue; payer approval timelines are improving month-to-month and are currently around ~8 weeks for many cases.

• Question from Charles Ndiaye (Stifel, Nicolaus & Company, Incorporated, Research Division): In terms of the sort of adolescent buckets of hypoparathyroid patients you're looking at, I guess, like what kind of patient size does this represent in the U.S.? And what kind of growth do you expect to see from here, assuming there's a successful label expansion?
  Response: Adolescent population is much smaller and typically more severe than adult hypoparathyroidism; label expansion addresses high unmet need but will not materially increase total patient counts versus the adult pool.

• Question from Luca Issi (RBC Capital Markets, Research Division): Maybe on the unique patient enrollment, is that a metric that you're committed to report going forward? Or are you planning to sunset that metric at some point? And if it is the latter, can you talk about whether that could be Q4 or would that be later than that? And then maybe if I can ask about Novo Nordisk, can you just talk about how that collaboration is going?
  Response: Management said enrollment as a metric may be phased out as revenue becomes the primary KPI (no commitment to continue publishing it), and the Novo Nordisk collaboration is intact and progressing without disruption.

• Question from Maxwell Skor (Morgan Stanley, Research Division): I was just wondering when we can expect preclinical data supporting YORVIPATH's potential for weekly dosing? And also, could you share your outlook on YORVIPATH's trajectory in Europe? How should we think about a potential ramp next year?
  Response: Preclinical/early data on weekly dosing expected at an industry conference early next year; ex-U.S. revenue expected to add ~EUR4–5M per quarter and to accelerate in 2026 as more countries go fully commercial.

• Question from Yuxi Dong (Jefferies LLC, Research Division): It would be great if you can confirm the U.S. and ex-U.S. revenue split for YORVIPATH. And then in terms of the ex-U.S. launch momentum, is there any specific time line for any upcoming reimbursement decision in any market and any pricing dynamic we should keep in mind as you expand internationally?
  Response: No precise split provided; management reiterated ex-U.S. algorithm (Q4 '24 ex-U.S. base ~EUR14M then +EUR4–5M each quarter) and said country rollouts and reimbursement timelines will be detailed early next year, with focus on preserving product value.

Contradiction Point 1

Conversion Rate Expectations for YORVIPATH

It involves differing expectations about the conversion rate from enrollment to treatment for YORVIPATH, which could impact revenue forecasts and market penetration.

What factors are causing YORVIPATH's low conversion rate, and when do you expect improvement? - Gavin Clark-Gartner (Evercore ISI Institutional Equities)

2025Q3: We expect it to mature and go higher, potentially reaching 85% or 90%. - Jan Mikkelsen(CEO), Jay Wu(CMO)

What is the conversion rate from enrollment to paid drugs? Will you continue reporting enrollment numbers? - Gavin Clark-Gartner (Evercore ISI Institutional Equities)

2025Q2: We aim for a 90% treatment success rate, acknowledging a tail of patients struggling with reimbursement. - Jan Mikkelsen(CEO)

Contradiction Point 2

U.S. Enrollment and Conversion Rate Improvements

It highlights differing expectations about the improvement in the enrollment-to-treatment process, which affects the ramp-up of YORVIPATH in the U.S. market.

What are your expectations for new patient enrollment rates for YORVIPATH in the U.S. going forward? - Jessica Fye (JPMorgan Chase & Co, Research Division)

2025Q3: We see a stable number of prescriptions being written in the U.S., even taking out the bonus from the ERP program. - Jan Mikkelsen(CEO)

Is there a three-month enrollment-to-conversion period? How can it be improved? - Derek Archila (Wells Fargo)

2025Q2: We expect acceleration of the conversion of patients to treatment in the second part of the year, as we anticipate improvements in the enrollment-to-treatment process. - Jan Mikkelsen(CEO)

Contradiction Point 3

Reimbursement and Contracting Environment

It involves expectations and realities surrounding reimbursement rates and the contracting environment, which are critical for revenue projections and financial forecasting.

Why is YORVIPATH's conversion rate only 70%, and when do you expect it to improve? - Gavin Clark-Gartner (Evercore ISI Institutional Equities, Research Division)

2025Q3: We expect it to mature and go higher, potentially reaching 85% or 90%. It will mature over time, and we don't expect changes in the contracting environment in Q4. - Jan Mikkelsen and Jay Wu

What is the expected proportion of patients prescribed YORVIPATH who will be reimbursed once at steady-state? - Jessica Fye (JPMorgan Chase & Co, Research Division)

2025Q1: It's challenging to predict the exact steady-state proportion of reimbursed patients, but I am optimistic about reaching at least 17-18% reimbursement. - Jan Mikkelsen

Contradiction Point 4

Impact of Seasonality on Revenue

It impacts expectations regarding the effect of seasonality on company revenue, which is crucial for financial planning and investor expectations.

Will Q4 reflect seasonal impacts, and will December script trends be lower than October's? - Tazeen Ahmad (BofA Securities, Research Division)

2025Q3: We don't expect any major impact in Q4. We're actually more worried about Q3 due to the longer summer vacation. We've seen that Q3 didn't have significant effects, and we expect Q4 to be positive. - Jan Mikkelsen

Can you break down YORVIPATH's U.S. vs. ex-U.S. revenue and discuss the time from prescription to patient therapy initiation? - Tazeen Ahmad (Bank of America Securities)

2025Q1: We expect a pretty significant sequential increase in the full year, obviously driven by the U.S. launch, but also a little bit higher in the second half than the first half. - Jan Mikkelsen