Ascendis Pharma A/S (ASND) Q2 2026 Earnings Call Transcript

Ladies and gentlemen, thank you for standing by. Welcome to the second quarter 2026 Ascendis Pharma Earnings Conference Call. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during this session, you would need to press star 1-1 on your telephone. You would then hear an automated message advised and your hand is raised. We ask that you please limit to one question and return to the queue for additional questions. And to withdraw your question, please press star 1-1 again. Please be advised that today's conference is being recorded. I would like now to turn the conference over to Chad Fugert, Vice President of Investor Relations. Please go ahead.

Thank you, Operator, and thank you, everyone, for joining our second quarter 2026 Financial Results Conference call. I'm Chad Fugere, Vice President and Professor of Relations at Ascendis Pharma. Joining me on the call today are Yen Mickelson, President and Chief Executive Officer, Scott Smith, Chief Financial Officer, Sherry Glass, Chief Business Officer, and Jay Wu, Executive Vice President and President Ascendis U.S. Before we begin, I'd like to remind you that this conference call, including the Q&A session that follows our prepared remarks, will contain forward-looking statements that are intended to be covered under their safe harbor provided by the Private Securities Litigation Reform Act. All statements made on this call, other than the statements of historical fact, are forward-looking statements. Examples of such statements may include, but are not limited to, statements regarding our commercialization and continued development of SkyTropha, URBPath, and UBWell, including label expansion and combination treatment, certain expectations regarding patient access and financial outcomes, our pipeline candidates, and our expectation with respect to their continued progress and potential commercialization, our strategic plans, partnerships, and investments, our goals regarding our clinical pipeline, including the timing of clinical results and trials, our ongoing and planned regulatory filings, and our expectations regarding the timing and results of regulatory decisions, and our financial outlook and Vision 2030 objectives. 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 under reliance on these statements. We assume no obligation to update these statements as circumstances change, except as required by law. For additional information concerning the factors that could cause actual results differ materially, please see the forward-looking statements section of today's press release in the risk factors section for annual report on Form 20F filed with the SEC on February 11, 2026. In addition, during this call, we will refer to certain non-IFRS financial measures. These measures are not prepared in accordance with IFRS accounting standards and should not be considered in isolation from or as a substitute for our IFRS results. A reconciliation of each non-IFRS measure to the most directly comparable IFRS measure, together with an explanation of why management believe these measures are useful to investors is included in today's press release. Transcon growth hormone, or Transcon HDH, is now approved in the U.S. by the FDA for the replacement of endogenous growth hormone in adults with growth hormone deficiency, in addition to the treatment of pediatric growth hormone deficiency, and in the EU, has received MAA authorization for the European Commission for the treatment of pediatric growth hormone deficiency. Transcon PTH is approved in the U.S. by the FDA for the treatment of hypoparathyroidism in adults in the European Commission in the United Kingdom's medicines and Healthcare Products Regulatory Agency have granted marketing authorization for Transcon PTH replacement therapy indicated for the treatment of adults with chronic hypoparathyroidism. Transcon CMP is approved in the U.S. by the FDA to increase linear growth in pediatric patients two years of age and older with achondroplasia with open epiphecies. Continued approval for this indication, which was based on an improvement of annualized growth velocity, may be contingent upon verification and description of clinical benefit in confirmatory trials. Other than the approved products I've just described, our product candidates are investigational and not approved for commercial use. As investigational products, the safety and effectiveness of product candidates have not been reviewed or approved by any regulatory agency. None of the statements during this conference call regarding product candidates shall be viewed as promotional. On the call today, we'll discuss our second quarter 2026 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 turn it over to Yen.

Thanks, Chad. Good day, everyone. During the second quarter, achievement of important milestones and strong demand for our Transcom products continue to drive the transformation of the incentives into a leading global biopharma company. The uniqueness of the Transcon technology platform, our strong development and global commercialization capability, and our values and visions are the fundamentals driving this transformation. We believe the same strength will continue to drive ascendance growth in the following years. Starting with the long-term durability of a highly differentiated approved protein and peptide-based combination products SkyTofa, Europaths and Eurval. We believe these products will be the key driver of our growth story for the next 10 to 15 years to global commercialization, potential for label expansion, including combination treatments and investment in patient support. The continued expansion of the Transcon technology platform enables us to fulfill our plans, despite at least one IMD or similarly yearly, each based on a new NCE, laying the foundation for strong growth for many decades. This will also enable us to establish new therapeutic areas in addition to hyper-power and growth disorders. As a further upside, our established partners are advancing trans-con candidates in large indications. This is why we believe Ascendis is well positioned for self-sustained long-term growth. Let's begin with a more detailed look at Uropath. Uropath is the first and only approved treatment for adults with hypopara that addressed the underlying disease by replacing the missing endogenous PTAs throughout the body. Uptake of ewe pads has grown steadily since long, both in the US and many other countries. Reflecting the significant unmet medical need among the more than 800,000 patients living with this serious rare disease in the geographic region covered by our global commercial infrastructure. Outside of the U.S., we see consistent new patient demand and continued expansion of global commercialization balances with full reimbursement. UAPAD is now available commercially or true-name patient programs in more than 35 countries. This illustrates the strength of our ability to execute a rapid, broad, global launch of a rare disease product. In the U.S., new patient demand for UAPAD in the second quarter has remained robust. In addition, physician prescribing is broadening and deepening. Patients who have successfully initiated uropath treatment continue to stay on therapy, indicating a high level of satisfaction. We continue to be excited by the growth of uropath in the US and outside the US, and to see its continued strong lungs performance. Data from our long-term Phase II and Phase D trials of UAPAS, presented in the second quarter, highlight why UAPAS is becoming a standing standard of care in post-surgiolus and all subsets of hyperpharmic, including ultra-rare genetic causes like DIGORDS, ADS-1 and ADS-2. Results showed sustained response rate of 82-86% for the multicomponent endpoint, with clinical benefit across multiple organ systems, CNS, kidney, small intestine and bone, plus meaningful improvement in quality of life. Patient retention as high as 95% after 5 years of treatment. Pretty unique. In parallel, we are working to further advance our leadership in HypoFarm with additional clinical trials that include expanding the label to include the age from 12 to 18 years and in the US higher doses for patients and developing a once weekly product for the patient that is on stable doses of uropads. Turning now to URVL. We believe URVL is positioned to become the market leader of therapy for echondroplasia. Rapid uptake of URVL is already transforming the US market. Across the board, we see a highly favorable response among patients and physicians to URVL's differentiator profile. In the U.S. through June 30, we had more than 170 unique patients enrolled. Since then, Optech has continued with more than 220 enrollments and more than 65% approved for reimbursement in the U.S. through the end of July. Really an unique loss. The rapid uptake is by patients of all kinds of backgrounds, those switching, returning to medical therapy, or starting therapy for acrodiplasia for the first time. We believe UOL is really growing the US market, which is exactly the pattern you will love to see when a highly differentiated product is introduced into air where there still exists a high unmet medical need. Long-term data for the now-completed people's approach trial show durable and consistent improvement growth, leg-bowing, body population, along with a general well-tolerated safety profile, compared to placebo, underscoring why the community is quickly adopting UOL. In the US, in the EU, a regulatory decision for UOVL is expected in the fourth quarter of 2020. We are also making UOVL available in select international markets to early access programs using the US FDA approval. Longer term, we are pursuing expansion opportunities for trans-consumption to ongoing and planned These include ongoing activities such as infants, 0 to less than 2 years of age, and we recently announced completion of this target revokement faster than expected. Adults with acondroplasia, children with hypercondroplasia, and still continue with geographic expansions. Turning now to combination therapy with trans-con-CMP and trans-con-coptimal. The biological rationale for this combination treatment is clear and extremely well founded on science. Transcon-CMP is removing the limitation caused by the overactive FTR3 pathway, so transcon-gropamine can provide a strong complementary effect. In addition, it has been observed that in agronyplasia, there is a partial impairment of the IGF-1 growth hormone axis. This is illustrated why children with agronyplasia have a negative IGF-1 SDS value, as shown of the demographic in both our phase 2 and phase 3 trial. In our co-clinical trial of children with acondroplasia, this unique combination has demonstrated sustained transformative analyzed growth velocity and 88 height score, including improvement in body proportionality. Based on this result, we believe this unique combination of once-weekly transcon-based therapies will transform the treatment of acondroplasia and other indications over time. Our recent week 78 COS trial data shows sustained efficacy over 78 weeks, with no comparison to safety and tollability. This points to the potential for this noble combination to establish a new treatment standard in natural infeclase. The phase 3 combination trial in children with icon infeclase will begin enrolling later this year. Turning to SkyTrova, the once-weekly growth hormone treatment built on their mode of action of unmodified somatopathy, with indications for pediatric and adult growth hormone deficiency. We continue to be the number one long-acting growth hormone by brand value in the US. We are extremely proud that SkyTrova recently achieved more than 20,000 unique enrollments. This illustrates the strength of our capabilities from supply chain, commercial infrastructure and non-market support to benefit such a large number of patients, rare disease patients. And we are working to make trans-concultomone available to more patients to label and geographic expansions. To support label-expansion buoyants that are described in our acondroplasia program, we We are conducting the phase 3 basket trial investigating transcon growth hormone in ISS, SDA and Turner syndrome. As an integrated part of our global growth disorder strategy, we expect to launch transcon growth hormone in the same countries where we also expect to launch transcon CMP. Turning now to our partnership. In metabolic disorders and obesity, our once-monthly trans-con semiglutide program with norvo-nordis continues to advance. In ophthalmology, our partner Conis recently initiated a first-in-human clinical trial of the anti-BGF treatment built on the trans-con technology in patients with BED-AMD. In closing, by always putting patients first, Ascendis has delivered highly differentiated leading trans-con based products, Europass, Urovel and Skartopa. We are on track to achieve our vision 2030 objective of being a leading global biopharma, building on a strong foundation for the future. With that, I will turn the call over to Scott to review our financial results and some additional comments.

Thanks so much, Ian, and good afternoon, everyone. I will touch on some key points surrounding our second quarter financial results. For further details, please refer to our Form 6K filed today. total product revenue was 315 million euro more than doubling year over year total revenue for Q2 2026 was 339 million euro which included non-product collaboration revenue of 24 million euro which further included a 17 million euro milestone related to transcon CNP your path revenue was 252 million euro in Q2, reflecting consistent new patient demand in the U.S. and continued growth outside of the U.S., reaching blockbuster status on a run rate basis in the second year of launch in the U.S. Propa contributed 55 million euro in Q2, which reflects increased demand in the U.S. and includes product sales to a collaboration partner. UVL was commercially launched in the U.S. during Q2 and generated 8 million euro in revenue in its first quarter on the market, reflecting strong demand and rapid conversion to paid therapy with limited stocking. Continuing to expenses, R&D expenses in Q2 were 76 million euro, up from 59 million euro in Q1, reflecting continued investment in our pipeline and innovation. Recall Q1 included a favorable €11 million reversal of prior period write-downs of Transcon C&P pre-launch inventories. SG&A expenses were €173 million in Q2 compared to €145 million in Q1, reflecting additional investments in the commercial launches of YorvaPath and UbuWell to accelerate growth for the long term. Operating profit of €220 million in Q2 included €158 million of other operating income related to the sale of the PRV. Non-IFRS operating profit was €92 million and non-IFRS operating margin was 27%. Refer to our press release for details. For Q2 26, net profit was €207 million and non-IFRS net profit was 61 million euro. We ended Q2 2026 with 812 million euro in cash and cash equivalents, which includes the use of 56 million euro in Q2 for our previously announced share repurchase program, including the net settlement of certain RSUs. Following the settlement of our convertible notes, we have no bank debt, no convertible debt, and 1.4 billion euro of equity. Turning to our outlook for the rest of 2026, for Yorvipath, we expect growth and performance consistent with prior quarters. We expect relatively stable revenue in the US. For UVWELL, we are encouraged by the early demand trends. We believe it is expanding the market and is on pace to be the leading achondroplasia therapy in the U.S., reflecting the large unmet medical need and the highly differentiated profile of UVOIL. Our Q2 performance reinforces our belief that we can achieve 5 billion euro in revenues in 2030. With our existing portfolio and our transcon technology as a strong foundation, we believe we are well positioned to grow revenue to more than 10 billion euro in the next decades, while developing and launching new Transcon products with blockbuster potential. We expect significant operating leverage as revenue scales through the balance of the year, while maintaining new investments in global product launches and patient access to reach as many patients as possible and support our long-term revenue aspirations.

Even with these investments, we expect to generate more than 500 million euro in cash flow from operating activities this year with that operator we are now ready to take questions thank you as a reminder to ask a question please press star one one on your telephone and wait for your name to be announced and to withdraw your question please press star one one again we ask you please limit to one question and return to the queue for additional questions And our first question is going to come from Jessica Fye with J.P. Morgan. Your line's open.

Hey, guys. Good morning. Thanks for taking my question. On that outlook for at least $500 million of operating cash flow this year, I think you gave that in the beginning of the year prior to the PRV sale. And I was just wondering if you're able to kind of update your cash flow expectations for the year. I know it's sort of like a greater than is unbounded, but curious if anything more you can add there. And then on that comment that UVWell seems to be expanding the market, is it possible to estimate how much of these patient enrollments are coming from market expansion? Thank you.

Thanks, yes, for the questions. and the other happy person besides me, Scott. So Scott got the opportunity to be the first one answering questions. So please, Scott.

Yeah, with respect to our cash flow guidance, just to be clear, greater than $500 million. I heard you say $100 million, so greater than $500 million.

And at this time, we don't want to bound the upper side because we're initial into the launch of Yuba Well. and that's euro by the way 500 million euro yen likes to point out and just related to the question um and it comes back to what we so many communicated last time we had this call that we don't have really the insight in exactly the distribution of where the patients are coming from and our general feelings and how we see it is that with such a strong demand we have a really strong belief that it's not only coming from switches it's much also coming from either patient that has stopped therapy or new patients that basically are coming to a situation because of the highly differentiated nature of UOVL that they want to start therapy. And I think this is where we have this strong belief that we see an expansion of the market.

Thank you. Thank you. And the next question is going to come from Tazeen Ahmad with Bank of America. Your line's open.

Hey, good morning. Thanks for taking my question. So, Yen, I wanted to get your thoughts about the IT challenge on UVL. We know, obviously, what the blue sky scenario is for Ascendus, and, you know, most of the scenarios look positive, but can you just maybe walk us through what the potential outcomes are? This is for a patent that expires, obviously, in 2030, and so between now and then, can you just tell us what could happen and what the potential for, you know, payments that Ascendis would need to make in the worst-case scenario could be?

Thanks, Sasin, for the question. And it's basically a question that is addressing the ongoing legal, I would call it, battle between Ascendis and Biomarine. and let me just come back to some facts the fact is that this pattern that the discussion got complete invalid in Europe so we never really come to an discussion if we were infringing and anything like that so when we see the situation outside US we got the patent invalid immediately to the patent system in Europe. In the US we never managed to come into the patent system because BioMarine selected to go to the ITC case which are a system which we can easily say traditional never have really dealt with a lot of cases that dealing with branded pharmaceuticals. In the ITC case there will be a first opinion from a single judge and he will come with opinion here in August and then there will be a more. Next time will be in December. There will be an opinion from the ITC and then later on there will be a potential confirmation of the ITC decision two to three months after to a presidential order. So you can see we are not getting any clarification in August in one way or the other way even if it's possible for one company and negative for the other one it's not really any kind of decision where it's going to be ending and after the first initial opinion from a single judge because of the ITC case will be taken to a decision for I cannot remember how many judges that will be part of that decision there is a huge opportunity to provide what we call and And when we see the public interest, meaning the element of how these product opportunities are really being serving an unmet medical need in the U.S. market with this rapid, rapid uptake of patients, it's really, really clear that there is a huge public interest to keep And just recall, I cannot remember one single case in the U.S. where it branded products that provide benefit to US patients has been denied. But you can see we just in a case where it's only it's a US, it has been cleared ex-US. And so whatever it happened, it will not have any material impact on Ascendis pathway. I can guarantee that. It's some kind of like people take it up as a life and death for Ascendis. This is a total not taken into the perspective what it mean for attendees. And out from that, I see it's not really as a material element for our destiny to be a leading biopharma and hit the $5 billion in 2030.

Thank you. And our next question will come from Gavin Clark Gartner with Evercore. Your line's open.

Hey, guys. Thanks for taking the question. I actually just wanted to ask on the earlier pipeline. So you noted in your prepared remarks that Transcon platform can fuel one IND for an NCE annually. I guess there hasn't been one yet this year. Should we expect one in the near term? And what exactly are the go forward plans for the earlier pipeline? Thank you.

It was because I've in some way felt that the two product opportunities that we have developed to our partnership built on the Transcon technology will still consider at the NCE, the one that now is clinic with Iconis and the other one we expect to go into the clinic now with Novo Nordisk. It's still some way being developed to the Transcon technology funnel. And I mean perhaps I shouldn't have done that but I still believe I feel some kind of little bit of ownership on these two product opportunities. At least we have major upside in both of them. So out from that perspective, I still consider the potential that we have this year, two new chemical entities being entered into clinical trials. And I think Kenneth and his team and anyone else, they're working very hard on that there will be at least one of these new chemical entities coming into every year now. And I'm really proud about that. But it's also addressing the sustainability of Ascendis independent of going out and buying something no one else wants to have. And I think this is where we really feel extremely pleasant by the situation, by being a fundamental company that's building on a strong, strong technology platform that provides both sustainability for ourselves, but also a continued flow of potential partner licensing. Great. Thank you.

Thank you. And the next question will come from Jaron Werber with TD Cohen. Your line's open.

Great. Thanks so much. Questioning UV, do you expect that there is some seasonality in terms of new patient starts in the summer as kind of kids are going on vacation? We're getting a lot of questions on sort of the 60 patient start forms kind of in April, and now you're sort of a 220. It sounds like there's like 50 per month now. Is that sort of sustainable from now on? And then it sounds like you're planning, you think you could be the number one brand by the end of the year. Biomarin, we think, has about 750 patients on drug. You think in the U.S., are you kind of referring to getting to a higher number than that by, let's say, late February?

Thanks for the question. I actually don't think Ascendis have really made some clear forward-looking statements related to how we see URVL being accelerating and expanding the market in a quantitative manner. I don't think we have come with any kind of indication related to that. I have no doubt it will do it, but it's not the same thing that we're going to quantify it I think after basic only four months in the market, I feel really not prepared to come with clear guidance to it before we have more quarters really into our, you can say, analytical system where we basically can look on trends and other things like that. But one person that really can give you a good feedback, now we talk about the U.S. market, is Jay. And he's extremely enthusiastic about what he's seeing. And you can give the latest weight what you see, how the market will develop. Thanks, Jan.

As Jan mentioned before, four months in, we're not prepared to give longer-term guidance. But what we can say is we're incredibly encouraged by what we're seeing today. When you look at some of the fundamentals behind the UVA well uptake, whether it's prescriber reach, we talked a lot a bit before around this space, there's quite a few centers of excellences. We're seeing 80% of them, nearly 80% already in a short four-month period, already prescribe UVA well to their patients. So even in early days, we're seeing a lot of enthusiasm from providers around the clinical profile of this product. I think even more importantly, when you look at the patient enthusiasm, I think you can see in early days, we're seeing a very positive trajectory. While we don't explicitly collect information on what therapy or non-therapy a patient is coming from, and again, that's driven largely by the fact that we have a broad label, so we don't need that information in order to ensure that this patient can get on therapy. This is rare disease. So qualitatively, we have heard confirmed anecdotes across all three categories for which our patients are coming from. And those three categories, again, are one, patients that are switching from current therapy. Two, patients that have previously discontinued pharmacological therapy and has now wanted to return to pharmacological treatment. And then third, a group of patients that historically have sat out and have said based on the clinical profile of UVOL, they now want to try a therapeutic option for the first time. So all that, again, to underscore, there is existing unmet need here, and because of our profile, we're definitely seeing that patients are coming out of the woodwork from growing the market standpoint, and we're just getting started.

Just to some way to add on to Jay's excellent comments. Ultimative, I have no doubt we will be number one in the acondoplasia space. Ultimative, we will expand the market because of the unmet medical needs. And that is just with the monotherapy. And when you look at how commitment we have to this era, where we now are making a complete new standard with the company treatment, I believe we are dedicated to be not number one in the first year, but continue to build for the next five to ten years with monotherapy, combination, integrated treatment regimes. And I believe with our once weekly transcom product built on Botamo and CMP, we are extremely, extremely wealth position, really, to be the leader in this segment.

Thank you. And our next question will come from Derek Archila with Wells Fargo. Your line's open.

Hey, good morning, and thanks for taking the questions. Congrats on the progress. Scott, I just wanted you to clarify a comment on your repath growth for the rest of the year. I think you said it's going to be like prior quarters. I guess which quarters are you referring? Because I think the quarter-over-quarter growth in 1Q was negatively impacted, saw some catch-up here in the second quarter. So, you know, maybe you can just clarify which quarters you are referring to. Thanks.

Yeah, Derek, thanks for the question. I think that two points. One is the consistent performance with the KPIs that we've given you, for example, with enrollments. We expect those to continue and be consistent. The other would be, and you could refer to our prior quarters, and maybe Chad, you know, maybe Chad can point to prior comments but I think that now that we've seen the full year you know the various trends that will come into play related to Q3 and Q4 and then Q1 next year. So we think actually folks did a pretty good job modeling out Q2 and now you have all the information you need to model the rest of the year going forward until we update basically the KPIs.

Just to give you some kind of what is our value in this year. The value for us we want to give you. We want to give you not so you basic getting a lower number so we look like heroes. We want to give you the number so you are right nearly every time. And I think this is a way we try to come up with our different mathematics algorithm how we see it and give you all the information for you really to be right in this manner. And I I think this is a way we like to be extremely transparent with everything what we perform. So we're quite sure that you basically can go out and really some way feeling all this comfort with the guidance we give you.

Great.

Thank you.

Thank you. And our next question will come from Joseph Schwartz with Leering. Your line's open.

Hi. Congrats on all the progress. Thanks for taking my question. As you embark on a phase three in hypochondroplasia, I wanted to ask how you're defining the enrolled population, and how large do you see the diagnosed treatable pool of hypochondroplasia patients who are not already being treated in some cases, if they're at the more severe end versus a chondroplasia? Thank you.

This is a very interesting question because it's actually some way going into the situation on how we basic are to genetic testing, taking a big patient group that was in old days was called ISS, idiopathic me. We have no clue what is the underlying diseases. And then you go out and do more and more and more genetic testing. And then when you find a mutation in the FDR3 receptor, and you find it in the right regions, and then you suddenly are not an ISS patient, but then you are a hypochondroplasia patient, even if you don't have the phenotype of looking like an achondroplasia patient or a hypochondroplasia that we saw for 10 years ago. So therefore you can see the ISS population is some way getting smaller and smaller because of the genetic testing is basically going out and giving them an underlying reason why you perceive will have a short status without potentially have the other element that you see for the phenotype of that. So this is where you can think when you go into ISS are you defined it from a genetic perspective or you define it for a phenotype or anything like that. And we are in a situation where when you see the clinical trial, how we're doing it, you will basically see that it's one of the pathways we have selected.

Thank you. Thank you. And our next question will come from Daniel Bronder with K&R. Your line's open.

Hey, team. Congrats on the quarter. I'm on for Lee Watzak. We were just wondering if you could give us a little more color on the quality of life metrics in the coach trial and you already alluded to the body segment ratios but how should we think about benefit on arm span and other metrics just to recall the coach tried is the combination tried where the combining the two transcon based product our transcon growth hormone and transcon cmp and I have to say when I look on an

element like arm span it's actually we are already reported some of the data we have reported the 52 weeks data and if you cannot find that deck I can send it to you or Scott can send it or Chad can send it or I don't know we have so many IR people that don't know their names more so from that perspective it is already came out and I have to say there was one of the I will say extremely positive surprises I saw because when we looked on monotherapy but either a CMP based one or a growth hormone based one we We did not see the expected, hopeful development that we could hope for, but we definitely saw it when we looked at the combination therapy. And then you can ask me, what is the scientific reason why you see it much more influenced benefit by the combination therapy? And I have to say, I don't know. But what we saw was an arm span that really gave us this hope with the combination therapy You basically will be in a position that you basically could avoid all kinds of lymph elongation surgeries in acondroplasia, both related to both legs and arms by that. And it's the slide number five, as I remember it. And what we see, Scott, read up.

The unprecedented improvements in the arm span with a combination were plus 9.4 centimeters with a trans-con CNP naive cohort, 7.9 centimeters with a trans-con CNP treated cohort.

So it was really an...

Compared to limb lengthening surgery centimeters.

Exactly, exactly. I have to say, it was one of the days where I felt it was worth to go to a job and really can see the benefit of what we're doing.

Thank you.

Thank you. And our next question will come from Yoon Jong with Wedbush. Your line is open.

Excuse me. Thank you very much for taking the question. I wanted to confirm that she had not provided prescription number for URIPATH in case I missed anything. And so I know that she said the patient demand remained robust in the quarter. So I wonder if there is any additional quantitative information that you can provide. And going forward, are you going to provide that number in the coming quarters? And I think you had this question before at the beginning of the launch. And when do you expect that you will feel comfortable providing a guidance in terms of the sales range on actual revenue? Thank you very much.

You're right. And I think it's starting to be a little bit repetitive every quarter come out and saying that we have more than 1,000 patients being unique enrolled per quarter. We have continued that message that we see steady state, steady state, and steady state. And we said in last year that we will stop coming with this because it was too repetitive. And then because people doubted for the Q1, then we also come up with the Q1. And it was the same number again. And what we're writing is that we see a robust steady state enrollment of unique new patients. And here we're referring to the U.S. with about 1,000 new patients every quarter. And we don't believe really. Now we went over to Europe. So now we're starting to give you a unique prescription enrollment of UofL instead. So we always have one product opportunity where you will have something to play with, with numbers and everything like that. Scott, you have some comments for the last one?

Yeah, I think, Yuen, our comments were directed to assume that the metrics that we've given you are consistent because Yuen wants to make our script shorter, so we don't want to repeat them more. And you should just assume that until we change it.

Great, thank you. thank you and the next question comes from alex thompson with steve will your lines open great uh thanks for taking the question and i appreciate the the color you provided to zine's question around the ongoing you know legal battle with buyer marin i guess as we think about potential scenarios here and again acknowledging sort of this idea around some public interest of the product and unmet need do you see um you know a settlement as a reasonable scenario to think about, or is that really not something that you think is reasonable?

I think I'm a very flexible person. And one thing I really want to do, I will always do what is best for patients.

Thank you. And the next question will come from MaxwellScore with Morgan Stanley. Your line is open.

Great. Thank you very much for taking my question. Just a quick one on your V-Path durability. I was just wondering if dropouts are still mostly during the titration phase, and if you can comment at all on how reauthorizations are trending. Thanks.

I think you're 100% correct. And when we see a patient being successful, coming into a treatment with uropax, coming over the titration part on it, and being into the treatment after that, we see extremely, extremely low dropout. and I think that illustrates wanting the patient satisfaction with this treatment because I'm often being asked what can we do more for these patients in the therapeutic treatment on it and when I see the satisfaction that it is in this way, then I feel that there is an extremely good position, retention and everything would really show that. We still develop once weekly for patients on stable doses just to give patients the choice if they want to do it in this way. We will look at other ways to improve their life like, for example, at-home capture monitoring and anything like that, we can help the patient like it's happening in type 1 diabetes and other things like that. So now you're addressing the element where we're saying is, we developed this here with a once-weekly profile, even if we could make it, sorry, once daily, because we wanted to do the interpretation most easily, because it's really complex to take patient upper conventional therapy at the same time you increase the PTA in replacement therapy and there was a why we made it as I once stated this really to facilitate the best possible titration but still we know it can be problematic for some patients and Jay can try to explain what we now doing to basically handhold the patient in this period so we also can make that extremely successful. So when you get a prescription we know everything will be much more successful for the patient not just after their belly being stable in the titration. So Jay Will you explain of the effort you're building in to really to get that to be as soft and as possible?

Absolutely. I can chat a little bit more about certainly the investments that we're making and also to answer your questions around drop-off and re-offs. Yes, as we've shared before, the majority of the drop-offs is during that titration period in terms of when patients experience the most amount of change. and where additional education and a higher-touch support model makes sense. And then for re-offs, that's actually pretty routine for us, so there really isn't much there in terms of it being a measurable effect on any kind of ongoing patient support. We have patients re-offing throughout the year, and it's just part of our day-to-day operations. From an investment standpoint, we've invested heavily in patient-facing roles for which we've deemed our patient access liaisons. They support patients both pre-prescription as well as through the prescription process and post. So essentially, we've seen a lot of success in early days with this field team being able to engage with this patient community. They have appreciated this high level of support, and we, of course, support them throughout the journey to ensure that we're optimizing for patient experience.

Thank you very much. One thing that's in a minute. And now we focus on US, but there is still a world outside US. Outside US, we have not seen the same level of dropout in this space. It looks like the interaction is pretty well established between the position and the patient and support system, really to see it without this kind of dropout. So it's basically a U.S. issue and date. So therefore, we know we can get it to function. We just need to ensure that the support system, also in the U.S., is strong enough to be sure that it's not a problem. Very helpful. Thank you.

Thank you. And our next question will come from Eric Joseph with Citi. Your line's open.

Hey, thanks for taking the questions. As far as your named patient programs or your early access programs, can you elaborate a little bit on which markets you're active in, whether eligibility might be determined by treatment status of a patient, and just generally how we should think about whether named patient programs could be meaningful contributors to patient volumes this year? For you, in particular.

Okay. Hey, I just wanted to ask what product you were referring to.

You were.

Yeah. I can guarantee that as we basic in our preferred remark try to put emphasis on, we have a global infrastructure in commercialization and patient support, product supply, and everything like that. just the number of scytofa rare disease patients we have taken over to the system, more than 20,000 patients, we are having the system function more than in 35 different countries. So we're not a company that just needs to get started. We already have established all this infrastructure. And what we're doing is that we're utilizing this established infrastructure basic, that got established because of Europads. Because this is what we did with Europads, we are using exactly the same infrastructure also for Eurovalue. So we will be where patient is, and we will be quite sure we will also serve the patient outside US, and potentially the market is much larger outside US. And I think, we hope we also will see a large penetration in the US where another short acting product really failed to do it. And we believe because of the highly differentiated nature of Eurovel, we will see a complete different pickup in the US. But it's definitely, we have a strong, strong, strong focus on the ex-US. and we will give you some guidance when we come later in the year so you can give also building up a model for the XUS thanks for taking the question thank you and the next question will come from Luca Issy with RBCM your line is open great thanks so much for taking our question this is Cassie for Luca so going back to you and Jay the three categories that you very nicely touched on for the naive switch and discontinued patients that are not on script.

BioMarine mentioned on their second quarter call that less than 100 patients have switched off of Voxovo. So the simple math that we're trying to do here is that it leaves you with about 70 patients in the second quarter who are naive or returned to treatment. So that's taken off the switch patients. So does that align with the numbers or impression that you have? And how does the dynamic look like between the truly naive patients and patients who were once uninvolved so go stop treatment and are now returning to treatment but to uv well and separate very quickly if you've commented or not on the ex-us strategy for uv well given the the decision is pending and the medic coming very soon this year thanks so much i like your way of doing all the calculation anything like that i cannot support it or i cannot deny it because I don't have the facts of insight to some way to confirm anything of the

numbers. I also saw the numbers that came out but I cannot really support it because I don't have the insight from our own numbers to really come out and come with any statement that indicates if I'm aligned or not aligned with. Related to the ex-US, for me to understand your question, was this reflecting what is the limitation in the ex-US or what was the question?

Thanks for asking to clarify. More about are you committed to running the show by yourself or you're considering partnering, given that 70% of the Voxo-Gosel that's historically coming from ex-US can be a quite heavy lifting.

Yeah, but so basically in the ex-US we have our direct market, which are I think 60, 70, 80, where we have our own commercial infrastructure, anything like that. It's pretty, pretty clear what we're doing there. Then we have our sales and distributions agreement. And this is, I think it's 70 countries or something like that. Well, it's got 80 countries that is covering this sales and distribution agreement. And the vast majority of all of them are all three products. So basically, there is already established infrastructure for the distribution. And then we have the two other, the third model, where we have our partnerships, one in Japan, one in China. and they also have all the three products. So we don't need to go out and make any new agreements for anything. Everything is established. Everything is running on full speed. And for some of the EU direct market, we're just waiting for our expected approval here in Q4 this year.

Thank you. And our last question is going to come from Faisal Kirshed with Jeffries. your line is open.

Hey, guys. Thank you for taking the question. Just wanted to ask a little bit on the Yorba Path life cycle strategy. Can you give us an update on the latest on getting the higher dose into the label for FDA? And then also any update on weekly Yorba Path? Thank you.

Yeah. What we see today is that we are enrolling the trial in the U.S. where the evaluating the 30 to 60 dose range in two different means that has been aligned with the FDA in their design what they wanted to see and we see that enrollment going extremely fast so we expect very very fast and you can say label expansion in the place where we don't have up to the 60 so we see that basic on just on execution and your second question was related to on weekly over path any update there yeah i think there's no news in this way that we're just executing and getting it into the market as fast as possible out from the expectation that we see that not as in any kind of LCM activity, but more at patient support for patients that really are in the stable dosing, which are not a lot after they have been in a situation where they have been stabilized with our daily treatment. Great. Thank you.

Thank you. This is all the time that we have for questions today. This does conclude today's conference call and thank you for participating. You may now disconnect.

Thanks a lot everyone.