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Earnings call · FY2026 Q2
Executive readout · one minute
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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 mathematic algorithm how we see it and give you all the information for you really to be right in this manner and 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 achondroplasia?
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 were 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, you can say, the phenotype of looking like an achondroplasia patient or a hypochondroplasia patient 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 potentially have a short status without potentially having 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 are you defined it for a phenotype or anything like that? And we are in a situation where when you see the clinical trial, how are we 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 Ken are your lines 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 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 trial is the combination trial where we combine the two Transcon based product, our Transcon growth hormone and Transcon CMP.
And I have to say, when I look on an element like ArmSpan, we have already reported some of the data. We have reported the 52 weeks data, and if you cannot find that data, 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, I 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 not saw the expected hopeful development that we can hope for. But we definitely saw it when we look on 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. So 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 transcon CNP naive cohort and 7.9 centimeters with a transcon CNP treated cohort.
So it was really an…
Compared to limb lengthening surgery in centimeters.
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.
Okay, 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 UROPATH 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 US 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 URL 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 our comments were directed to assume that the metrics that we've given you are consistent because Yen 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 Stiefel. Your line's open.
Thanks for taking the question. And, Yen, I appreciate the color you provided to Zene's question around the ongoing, you know, legal battle with Biomarin. I guess as we think about potential scenarios here and, again, acknowledging sort of this idea around the public interest of the product and unmet need, do you see, you know, a settlement as a reasonable scenario to think about, or is that really not something that you think is reasonable?
Alex, I think I'm a very flexible person. And one other thing I really want to do, I will always do what is best for patients.
Thank you. And the next question will come from Maxwell Score 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 Can you comment at all on how reauthorizations are trending?
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 now 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 patient on stable doses just to give patient 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 happening in type 1 diabetes and other things like that so now you addressing the element where we saying is we developed this year with a once weekly profile even if we could make it sorry once daily because we wanted to do the titration most easily because it's really complex to take patient upper conventional therapy at the same time you increase the pth in replacement therapy and there was a why we made it as a once daily 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 basic 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 they're barely 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.
Great. Thank you very much.
One thing that's in a minute, now we focus on us on U.S., but there is still a world outside U.S. Outside U.S., we have not seen the same level of dropout in this space. It looked like the interaction is pretty well established between the physician and the patient and support system. We need to see it without this kind of dropout. So it's basically a US issue and J. So therefore we know we can get it to function. We just need to ensure that the support system, also in the US, 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.
Thanks for taking the questions. As far as your named patient programs or 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. Thanks. For you, UV-Well in particular.
Okay, I just wanted to ask what product you were referring to.
UV-Well.
Yeah, I can guarantee that as the 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 scrytrophic 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 that got established because of UOpads. Because this is what we did with UOpads. We're using exactly the same infrastructure also for UO value. So we will be where patient is And we'll 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 pick up 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 ex-US.
Thanks for taking the question.
Thank you. And the next question will come from Luca Issy with RBCM. Your line is open.
Thanks so much for taking our question. Hi, this is Cassie Peluca. So going back to UV well, and Jade, there's three categories that you very nicely touched on for the naive switch and discontinued patients that are not on script. Violin mentioned on their second quarter call that less than a hundred patients have switched off of FOXOVO. 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 the patients who were once involved, so go stop treatment, and are now returning to treatment, but to UVA well? And separate very quickly, if you've commented or not on the ex-US strategy for UVA well, given 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 factual 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 my 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 it. More about are you committed to drawing the show by yourself or you're considering partnering given that you know 70% of the box local sales is 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 Khurshid with Jeffries. Your line is open. in.
Hey, guys. Thank you for taking the question. Just wanted to ask a little bit on the YorvaPath 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 YorvaPath? Thank you.
Yeah. What we see today is that we are enrolling the trial in the U.S. where we are relating 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 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. 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.