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On Tuesday, 04 March 2025, Ascendis Pharma (NASDAQ: ASND) presented at the TD Cowen 45th Annual Healthcare Conference, outlining its strategic initiatives in rare disease treatment. The company highlighted the successful launch of Yorviq for hypoparathyroidism, emphasizing both its commercial achievements and the challenges ahead. Ascendis aims to expand its market presence in the US and Europe while maintaining a focus on innovative therapies.
Key Takeaways
- Successful launch of Yorviq with 908 new prescriptions, targeting hypoparathyroidism patients.
- Significant market expansion in Europe, with plans to penetrate 55 countries by 2026.
- Focus on achieving profitability and cash breakeven through strategic market targeting.
- Upcoming CALYPSA data to support Yorviq’s differentiation in treatment benefits.
- Potential new standard of care in achondroplasia with the TransCon CNP and Skytropha combination.
Financial Results
Ascendis Pharma reported notable financial achievements with Yorviq’s launch:
- Generated €29 million in Europe within nine months, matching Natpara’s peak performance.
- Forecasted Q1 revenue for Yorviq is €29 million.
- Gross-to-net considerations include low 20% rebates for government programs and low single-digit channel fees.
- The commercial market historically represents 70-80% of revenue, with potential growth due to Medicare Part D redesign.
Operational Updates
The company is actively expanding its market reach:
- Targeting 6,000 to 7,000 physicians with an integrated commercial effort.
- Focusing on 2 patient groups: approximately 10,000 uncontrolled and 30,000-35,000 partially controlled patients.
- Plans to launch in 5 to 7 more European countries in 2025 and reach 55 countries by 2026.
- Anticipates 300-400 Natpara patients switching to Yorviq in Q2 and Q3.
Future Outlook
Ascendis Pharma is optimistic about its growth trajectory:
- Yorviq is expected to drive growth in the US, with subsequent international expansion.
- TransCon CNP and Skytropha combination trial aims to establish a new standard of care in achondroplasia, with data expected in Q2.
- Upcoming CALYPSA data will focus on maintaining normal calcium levels, enhancing Yorviq’s treatment profile.
Q&A Highlights
During the Q&A session, Ascendis Pharma addressed key concerns:
- The company is confident in maintaining orphan drug exclusivity for Yorviq, with 8 years of status in both the EU and US.
- Breaking orphan drug designation requires a superior product, reinforcing Yorviq’s competitive position.
In conclusion, Ascendis Pharma’s strategic focus on rare disease treatments, particularly with Yorviq, positions it for continued growth and market expansion. For more detailed insights, refer to the full transcript below.
Full transcript - TD Cowen 45th Annual Healthcare Conference:
Yaron Werber, Moderator, biotech team, TDECOUN: Good morning, everybody, and thanks once again for joining us for the, forty fifth annual TDECOUN Healthcare Conference. I’m Yaron Werber from the biotech team, and it’s a great pleasure to moderate the next fireside chat with Ascendis Pharma. To my left, really needs an introduction, Jan Mickelson, president and CEO. And to his left needs an introduction, Scott Smith, EVP and chief financial officer.
Gentlemen, thanks for joining. We appreciate having us.
Jan Mickelson, President and CEO, Ascendis Pharma: Thanks for inviting us. Do you
Yaron Werber, Moderator, biotech team, TDECOUN: wanna make some opening remarks? Go for it.
Scott Smith, EVP and Chief Financial Officer, Ascendis Pharma: Yeah. This is my favorite part. Yeah. We are. In addition to thank you for the thanking you for the invitation and the great crowd, I would just like to tell everyone.
Before we begin, I would like to remind you that this presentation contains 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, continued development of Skytrophy and urovid PATH, as well as certain revenue and growth expectations, our pipeline candidates and expectations with respect to their costs, continued progress, potential commercialization success, our strategic plans, partnerships, and investments, our goals about our clinical pipeline, including timing and results, and ongoing and planned regulatory filings. These statements are based on the information available to us today. Actual results could differ materially from those in our forward looking statements, and you should not place undue reliance on these statements. We assume no obligation to update these statements, as circumstances change except as required by law.
For additional information concerning the factors that could affect results, please see our forward looking statements section in our current and future reports filed with ACC, including the form 20 f filed on 02/12/2025. Anything specific we should read in that It depends on how late at night it is or if you’re on a long flight and you wanna guess Yeah.
Yaron Werber, Moderator, biotech team, TDECOUN: The the the filings are my favorite. Well, terrific. I wanna maybe start by talking about Jorvi’s launch. You know, you’ve give us a little bit of metrics at the end of last year and then, you know, obviously, early this year. You had 908 new, but a new prescriptions.
8080% of them were new to Yorvy, up to 500, almost 40 unique providers, only 200 prescribed in the pair in the past. So you’re really seeing a broadening of the prescriber base. Presumably, the lack of a black box, obviously, is a big draw to bringing new prescribers in. And I guess my real question is kind of twofold. What’s what what kind of patient is coming on to your IV now?
Is it a switcher? Is it a new patient? And what kind of a physician is now coming to your RV?
Jan Mickelson, President and CEO, Ascendis Pharma: So first of all, we are in our initial launch of your patch here in The US. We’re addressing a major unmet medical need, the hypoparatide regimen.
Scott Smith, EVP and Chief Financial Officer, Ascendis Pharma: The
Jan Mickelson, President and CEO, Ascendis Pharma: patients we are providing treatment for a patient that already are diagnosed with hypopara, so they know that this disease. And if any one of you have not really listened to the patient voice, you should try to go and listen to the FDA arranged PTH hearing of the patient group where there is a lot of testimony on the burden to having this disease. We launched Europant in Germany, Australia in January. In December, we also start the launch in The U. S.
To some way, to do our targeting of the physicians that we want to address with our commercial effort, we defined the patients out from the ICD-ten codes because they’re already recognized as patient. We, to the honor of Scott, we call something uncontrolled patients and define them by the way, where we said that we need to see the physician at least five, six, seven, eight, nine, 10, 11, 12 times a year, at least in the last twelve months having a medical hospital visit because of hyperpara, we call them uncontrolled. The other patient group we call Parti uncontrolled is patient that’s seeing an endo two, three, four times a year and in the medical history also have hospitalization because of hypopara. The first group is about ten thousand patients here in The U. S.
The second group is about 30,000 to 35,000 patients. Why did we do that? We did it out from this perspective is that we know because of the limitation of seeing an endo in The U. S, we wanted to be quite sure we utilizing the effect that many patient is seeing the endos with so high frequency. And we wanted to be quite sure that this physician really could see and learn about the benefit of your pet treatment.
So we don’t believe that any patient group with hypopara, even if you are uncontrolled or party controlled, is not benefit of having your PET treatment. But we also believe that the initial patient we often get into our system here is coming from the more uncontrolled patient, but not because they have a higher medical need and not getting the right benefit as rest of the patient population also will get, but because we see the induce so much more often. So from that perspective, we expect a steady state of patient coming on treatment because the pool we are talking about is 10,000 patient uncontrolled, 30,000 to 35,000 patient partially uncontrolled, and they already have predetermined meetings with the Endo. So there was our initial effort. We have not reached all the Endo yet with our sales effort.
We believe in Q2, we will basic have reached the majority of our target into at that time. So we still is in the initial states of the launch and more and more activity is getting started. For example, activity related for physician to physician meeting, selling about the experience they have by having patient on treatment. But what we really see an extremely successful launch. And there’s no doubt, EUROPATH will provide a significant revenue to Ascendis farmer and also will be the product that basic will bring us into profitability and cash breakeven.
But don’t forget, it’s a life changing event for these patients, and we really get to test the moment for all the patient how they benefit for this treatment.
Yaron Werber, Moderator, biotech team, TDECOUN: And Jan, how many physicians are in your target majority target, you know, initial tranche?
Jan Mickelson, President and CEO, Ascendis Pharma: The initial target is about 6,000, seven thousand physician. And it’s managed with our sales force that basic all. I would say it’s integrated commercial effort because it’s not any only sales force. They’re also our field ran person team. This is our ESSA program.
Just remember, we are a company that had been working in rare disease endocrinology for years now with SKYTROFA. We’re not working in small rare diseases. We are working in large rare diseases. We are taking more than 10,000 patients to the system. And we’re utilizing exactly the same established system.
We’re not going to out and build it up from scratch. We had it already. And this is also why we see this successful launch because we are launching this product, Europe, from an entity that already have the setup, experience, the knowledge, know what to get done to work with a large amount of patient in rare diseases to medical researchers.
Yaron Werber, Moderator, biotech team, TDECOUN: And and these six to seven, did they were they they were all, I I presume, based on practice size or how many patients you think they have in in their practice who are, you know, hypopara. Do they cover all the prior Natura prescribers? Like, how much of an expansion is this?
Jan Mickelson, President and CEO, Ascendis Pharma: Many of the physician that is doing prescription today, which have written a EURPAP prescription, is not coming from the same physician that wrote the Netpar one. We’re totally expanding out for what we solve with Nepal. So I think one of the thing I can give you as a kind of reminder, Don’t use nepar as a way to think about Europe. It was a quite different product. It was a product with a labeling as an adjunct basic to conventional treatment, not a treatment labeling as we have with your pets.
You didn’t see the majority of the benefit received with your pet with napa. So when we see the patients in The US, it’s complete different treatment benefit we see compared to be on Netpar, which also indicating in clinical data, labeling, everything what we see.
Yaron Werber, Moderator, biotech team, TDECOUN: Scott, I think you’ve talked about ten percent to fifteen percent. I’m gonna ask a question about gross to net. I think you mentioned ten percent to fifteen percent of patients are uninsured and maybe qualify for the patient assistant program, but the rest of the patients typically, there there is no free drug program, right, for Yorgy. You know, how do you think about maybe gross to net patient support, you know, and and kind of stability of price?
Scott Smith, EVP and Chief Financial Officer, Ascendis Pharma: So on the government, the government portion, there’s mandatory rebates that we can’t get around, and those are likely low 20%, right, with Medicaid and and now, with the new redesign on Part d for Medicare. On the commercial side, I would not expect, at least at this time, to have, let’s say, contracting as such. Right? And then in the channel, you typically have, like, low single digit fees related to the channel. So if you go in and do some kind of mix, you can have, I would say, a a reasonable estimate of yield per per patient.
The question on would it be stable, I don’t see any reason why I don’t see any reason for disruption in in the in this market.
Yaron Werber, Moderator, biotech team, TDECOUN: And what percentage of the market would you say is commercial?
Scott Smith, EVP and Chief Financial Officer, Ascendis Pharma: Historically, it’s been a higher percentage. It’s been, I would say, like, 70 to 80%. We have to see what the new redesign if the cap on out of pocket draws more patients in.
Yaron Werber, Moderator, biotech team, TDECOUN: Which presumably will make it, even higher. No?
Scott Smith, EVP and Chief Financial Officer, Ascendis Pharma: With the re with the redesign of the cap with the cap on out of pockets, I would assume more government coming in potentially. But historically, it was, you know, close to 80%,
Yaron Werber, Moderator, biotech team, TDECOUN: at least with that part. Got it. Commercial then. Yeah. So the Part d redesign will draw patients into Part d.
Right? But I guess the question, I guess, has been is it a government Part d, or is it a government outsourced Part d?
Scott Smith, EVP and Chief Financial Officer, Ascendis Pharma: Exactly. So so it’s to be it’s to be seen.
Yaron Werber, Moderator, biotech team, TDECOUN: Yeah. When you’ve launched so far and so far in Germany and Austria, and, that was I think you did 26, 20 7 million, 20 9 million, actually. So essentially you did what NetEpara did at peak within nine months or so. You talked about launching in more territories in Europe. This year you have distribution more than 50 countries and France will have an ATU, I think has an ATU now.
What’s the case of how fast Europe can grow?
Jan Mickelson, President and CEO, Ascendis Pharma: So Europe is very different compared to US because it’s, say, it’s not a single market. So when you’re getting approval in Europe, for example, many comp countries are into the EMEA system, then you get approval of all these countries. But that is not the same thing that you are fully reimbursed. Being fully reimbursed is only happening at the middle time of approval is basically that you can launch in Germany and Austria. If you go to other countries outside Germany and Austria, it typically take twelve to twenty four months after the approval to be fully reimbursed.
So what we expect to see in ’twenty five is that besides Germany and Austria, where we now fully reimbursed, we will see five to seven more country coming in in the air where we call Europe direct. Meaning is where Ascentis Pharma has its own sales force established, and it will be large country like France. France, currently, we have a system called AP2. It’s a noncommercial system, but fully reimbursed system where the physician can request a patient could to come on treatment, but we cannot do any commercial effort. There will be country like Spain, which are the big country, Italy, and then there will be clusters where we will see Scandinavia, Benelux and other of this cluster coming into the treatment.
Outside U. S, outside our effort in Europe Direct, we have an segment called international market and Europe indirect. Europe indirect is mainly the countries to the East Of The Middle Of Europe. So it’s basic all the countries starting with Greek, Poland coming up. And these countries, together with the international market, is going through the same system where we will expect multiple countries, and this is about 45 to 50 countries, will start to be reimbursed in ’25 and fully reimbursed in ’26.
We still have a lot of patient in this era, but it’s to a system called name patient program. This is where physician make name patient program for each single patients and then having a reimbursement on that typical back to German list prices on it. For example, in Greek, we have more than hundred patient on treatment now on basic name patient program systems. And when we talk about revenue generation, U. S.
Is unique, a large single market, which can be penetrated fast. If I look outside US, there is many, many more patient, perhaps four, five, six times the number of patients, but the penetration are much more slow. So what we expect to see in 2025, we start to see it pick up a little bit. 2026 really starting to be more and more penetrated. And then we will see the after ’26, we will see the full commercial effort in about 55 countries where we will be.
So therefore, we are in the initial longs. So US will be the driver, main driver in the initial years, and then you will see rest of the world pick up mainly because the time it takes to be fully reimbursed in all these different countries.
Yaron Werber, Moderator, biotech team, TDECOUN: Yeah. And and maybe, Scott, for you, when you’re using, you know, sort of sort of commercial partners in some of these sort of, you know, secondary, tertiary markets, it’s not gonna be direct. Is that a percentage of sales goes to them? Or how do these cognics typically written?
Scott Smith, EVP and Chief Financial Officer, Ascendis Pharma: So we would we sell product to them. They they put in purchase orders, and we sell product to them, periodic. Right? So it’d be, like, once a quarter or twice a quarter.
Yaron Werber, Moderator, biotech team, TDECOUN: At a negotiated price and then they have their margin based on that. And that’s the Correct. Got it. Maybe Jan to you, the enableratide CALYPSA data is coming up. My question is,
Jan Mickelson, President and CEO, Ascendis Pharma: when
Yaron Werber, Moderator, biotech team, TDECOUN: we look at across studies at the composite endpoint and the plus the albumin adjusted serum calcium, there’s been ranges. Like, in the pair, you use 7.5 to 10.6. I think your views, eight three to ten six. Inebo in a in a previous study, it used seven eight to nine. Right?
So each one had slightly different. I think inebo is now, you know, harmonized with your endpoint more or less. I think there’s slightly difference on the, the the the minimum sort of calcium per day requirements. The why is there a range in the endpoint?
Jan Mickelson, President and CEO, Ascendis Pharma: Yeah. I think when we look in our phase two trial and phase three trial, we used exactly the same, same catch them in both trial because you typically use Phase two to take risk of Phase three. So you’re not going into a Phase three with a major risk. When you talk about the CALUPSA trial, the basic went down to 7.8 in the phase two trial. And if I look on the data, which are somewhat hard to look at in this perspective, but it looked like a big portion of the patient were not qualified to be up on 8.3 at that stage.
So for me, it’s some way it’s not really was a de risking of the phase three because I believe all pivotal trial or phase three trial need to be run between the 8.3 to the 10.6 because that is more or less being from a regulatory perspective being defined as normal level of calcium. You can imitate treatment in a lower amount on it, but the endpoint need to be fulfilled 8.3 to 10.6.
Yaron Werber, Moderator, biotech team, TDECOUN: Yeah. And and across studies, you you each had a different criteria as to how you you’re reducing the the background meds at the standard of the care. They they’re also dosing higher in the phase three than they did in the phase two. The in the phase two, they did have a slightly higher serum calcium reduction than than you did. They obviously also had immunogenicity, which I’ll come back to.
But why do you think they’ve had a higher serum reduction? Serum, urinary calcium reduction?
Jan Mickelson, President and CEO, Ascendis Pharma: So first of
Yaron Werber, Moderator, biotech team, TDECOUN: all serum. Urinary calcium. Yeah.
Jan Mickelson, President and CEO, Ascendis Pharma: So one of the thing is when we talk about a treatment of hypopaira, it constitute three pillows. You give back the missing part. You give back the hormone that they’re missing. And if you have hypopara, you’re missing native PTH. So what we’re giving back is basic, the active part of PTH molecule, which give exactly the same biology as endogenous PTH.
Then you need to give back in the same physiological level twenty four hours, seven days a week. And then you also need to be sure that it’s distribute in the same amount in the body as the endosheous PTHs do because typical endocrine diseases is multi, multi organ, so you need to basically be in many different organs in the body. So when I look on the amyloid compound, it has nothing to do with PTAs normal biology. It’s basic have the PK as a short acting PT8 molecule like, like, like Forteo, which have covers for two or three hours in the body. So then you can say why can it have a more long acting effect.
It is because when you look on the biology of a normal PTH molecule, it both activate the receptor and then it also activate inside the cell. So what you did with when they generate terminals and it’s basic living up to the next discussion you had about ADA, it’s a fusion peptide between PTH and PTH related hormone. And then you fuse these two peptide and then you make multiple mutation into the molecule. And one of this mutation it do is basically fix the receptor configuration in the signal pathway inside the cell. So out from that perspective is that when we look on the body, it never providing the same benefit as endogenous PDAs.
For example, when you look on different biological system, for example, phosphate clearance, one of the element of comorbidity is in hyperpara is basic the of cardiovascular lesion issues because of high calcium phosphate complex. And when you measure their data, you can see they only measure phosphate after two hours because that is the same as PK because this is where you have the active on the membrane system because that is the one that promote phosphate clearance. So when you look on the CALYPSO data on calcium phosphate complex, that basically are not eliminate the risk factor, which are really one of the high risk, long term risk of having hypopara is calcification. The other part is they’re not really doing the writing on the bone because of the same reason. What you have in hypopaira, you have an unnatural bone density that have a higher bone density because in a typical normal person like Scott, his entire skeletal will be taking over on a remodeling in five or six years.
It’s not happening in hypo para. So therefore, you get a bone structure that is not healthy and it has a high bone density. And that really are changing when you normalize PTH level. So you get a normal bone structure again. But why is that important?
And this is why we have problem by calcium. Because if you look on calcium hemostasis, how in the body really have normal way to do deal with calcium is that you have three major component. First, uptake from the intestine system when you eat food, and that is happening with active vitamin d from the kidney. Then you have the kidney reabsorption, and then you have the skeletal. The skeletal is the key element of having calcium reservoir in the body.
But when you cannot do anything with the bone, you’re basic when you have no calcium to take from the food or anything like that, where do you get the calcium from? And this is why they have problem with calcium in the status in it. So everything is pretty clear out from the mode of action, biology and everything like that. And this is what I like with science. You cannot cheat science, but sometimes you cannot really understand science in the beginning until you see more data coming out and then you go to the next realization.
Yaron Werber, Moderator, biotech team, TDECOUN: Maybe a quick question, Scott. The do do you recall what the latest consensus is for Jorvik? I’m going back to Jorvik commercial, kind of outlook outlook.
Scott Smith, EVP and Chief Financial Officer, Ascendis Pharma: Yeah. For q q one, it’s €29.
Yaron Werber, Moderator, biotech team, TDECOUN: Okay. So it’s kind of flattish quarter over quarter. For Jorvik? Yeah. Jorvik.
Yeah.
Scott Smith, EVP and Chief Financial Officer, Ascendis Pharma: Well, that’s for the quarter. Last year was 29.
Yaron Werber, Moderator, biotech team, TDECOUN: Oh, I’m sorry for the year.
Scott Smith, EVP and Chief Financial Officer, Ascendis Pharma: So ’19 to ’29 for q one.
Yaron Werber, Moderator, biotech team, TDECOUN: Yeah. And what about for the year for Yoreen?
Scott Smith, EVP and Chief Financial Officer, Ascendis Pharma: I think the latest I think yeah. I better not say because I don’t remember the latest for the year.
Yaron Werber, Moderator, biotech team, TDECOUN: Yeah. I think it was 16 at around the beginning of the year. Correct. Correct.
Scott Smith, EVP and Chief Financial Officer, Ascendis Pharma: Yeah. It’s it’s gone up a little bit, but I’m not I don’t wanna say for sure because I don’t I don’t remember. I I wanna it’s it’s probably February ish is what is what I’m thinking.
Yaron Werber, Moderator, biotech team, TDECOUN: Yeah. I remember on the on the on the 02/16, ’16, you you again, you you have not given guidance, obviously, but I felt that you you remember just for everybody, Netpara in The US peaked again, obviously, different product at around $2.50 or so. Right? $2.74 to $2.45 or so. And you you essentially did what Netpera did a year three in Europe, a year five or four in Europe in one year.
So if we kind of back triangulate and, of course, the price is higher as well and obviously a much bigger prescriber. I I guess, putting it all together, how and I I kinda felt you felt okay about the $2.16. How are you feeling about the $2.40?
Scott Smith, EVP and Chief Financial Officer, Ascendis Pharma: Well, maybe maybe I should say it like Ed said at our dinner. In one year, we accomplished what took NAPARA in four years in Europe. So that’s a good challenge for us to do the same thing in The US.
Yaron Werber, Moderator, biotech team, TDECOUN: Is there any what what are the key barriers? Is it just switching patient? I mean, obviously, expanding the market. How fast are you able to switch patients off in the para also at the same time right now?
Jan Mickelson, President and CEO, Ascendis Pharma: The para patient, which we believe that is about three hundred to four hundred patient on, the majority of these patients are still on that path. They got a letter here in Q1, where they’re saying is that Nepal is starting to stop delivery. So we expect that it will be happening in q two, q ’3 in The US, that the three hundred to four hundred. We do not know exactly the number, but the three hundred to four hundred patient will start switching over.
Yaron Werber, Moderator, biotech team, TDECOUN: Yeah. Okay. By the way, if not, ask the audience. Any questions from the audience?
Scott Smith, EVP and Chief Financial Officer, Ascendis Pharma: Go for it. Yeah.
Yaron Werber, Moderator, biotech team, TDECOUN: What’s the need for Urvi in The US and The EU? Varicase right now on it is obviously the the amyloid drug. But, vaguely speaking, you have confidence that orphan drug exclusivity still matter. I’ll just repeat it for the for the, for the webcast. The question essentially is whether you have confidence that the orphan drug exclusivity in Europe specifically, both in The US and Europe will hold for your VIN.
Jan Mickelson, President and CEO, Ascendis Pharma: VIN? I take it from a different perspective is that when I see all the available data, it’s another product that anyway with the data we have seen yet is providing a benefit to the level of what you see with your patents. So I don’t think even we need to discuss orphan drug status, which we have for eight years in both places as a restriction to do it because that is something you basic will say if you have product that is equivalent in the same level on it. So I’m not even taking that into considerations in this way. But you’re right.
We have eight years orphanedox stages.
Yaron Werber, Moderator, biotech team, TDECOUN: What what would be the the mechanism to break an orphan drug designation once it’s actually received?
Jan Mickelson, President and CEO, Ascendis Pharma: You need to have a better product.
Yaron Werber, Moderator, biotech team, TDECOUN: In a in a head to head study?
Jan Mickelson, President and CEO, Ascendis Pharma: That is always the definition on if it’s so clear that you don’t need a head to head study, you can potentially break it, but it always will be better to have it head to head.
Yaron Werber, Moderator, biotech team, TDECOUN: Okay. Maybe final question from me just in the remaining time. You in in q two, we’re expecting the phase two code study. This is the weekly sub q combo data, Skytropha and TransCon CNP, given weekly. The primary endpoint is the annual growth velocity at fifty two weeks.
Are you doing a baseline AGV assessment? This is an an achondroplasia, actually, using Skytropha plus CNP. Are you doing a baseline assessment, to get a baseline AGV, or are you doing a change from baseline? I mean, there’s no control. That’s why I’m asking.
Jan Mickelson, President and CEO, Ascendis Pharma: Okay. You know, if I’m excited about anything this year from a clinical perspective, this is the COAST trial. Why I’m really excited about it? Because it can take a complete new standard for treatment of a conteplation. Ascendis is here for the patients.
And if we can make a new standard for treatment as we did with Uropath for hypopara, So I really compare a little bit the current treatment like Netpar as what we had in hypopara to what we see in acontoplasia, and now we’re coming with the Europad version in achondroplasia. So what I see with our TransCon CMP, and you can look at the data again, when you address the hyperact two FGRT receptor and look at linear growth, all data indicate whatever you do with TransCon CNP, or so at tight, short acting CMP, tyrosine kinase, you hit about an analyzed height velocity about 5.5 to six centimeter over twelve months. You can cheat a little bit by getting a little bit higher or lower by shorter time or patient or demographic. But what trends can see was unique, it showed a lot of positive effect beyond linear growth like late Boeing, Boston strength, and other thing. Because only one thing, because we have continuous exposure of CMP twenty four hours, seven days a week.
That is providing all the benefit beyond linear growth. What we’re doing with the combination trial is like having a car. Trying to con CMP is removing the brake on the thylacine kinase. This is why everyone hit the same growth velocity because if a car is sailing on a hill, no matter how you remove the brake, it go up to the same speed. What we’re doing by combining with Kytropha is that we also take a speed on.
So how you win a Formula one. Remove the brake, put the pressure on it by the speeder. And it’s not only for linear growth, it’s also related to other comorbidity as muscle strength, body composition. Everything like else that you look at comorbidity in achondroplasia. And this is why we believe that the COS trial is the starter for a new standard in treatment of achondroplasia.
Yaron Werber, Moderator, biotech team, TDECOUN: So is is the goal to then we should we should compare it to the 5.5 to six of CNP of Transcan alone?
Jan Mickelson, President and CEO, Ascendis Pharma: Just the design is simple. We have two arms. We had a problem. We got over rolled a lot because we couldn’t stop patient coming in. And so we have two arms, one that have CMP therapy for at least one year, and we know the base line of analyzed growth velocity for that is about 5.5 to six.
And then we have new patient two that comes in without any treatment start directly on SKYTROPHIA and TransCon CMP. So there’s a two arm study, and both of them, we have the pre analyzed high velocity of both of them.
Yaron Werber, Moderator, biotech team, TDECOUN: And it’s one to one randomized?
Jan Mickelson, President and CEO, Ascendis Pharma: Nope. It’s nearly the double amount of the naive, I think. It’s not right, Scott. I think I think. I cannot exactly remember the number between the two.
Yaron Werber, Moderator, biotech team, TDECOUN: So we’ll have essentially a control, a CMP control. Yeah. Terrific. Jan and Scott, thanks so much for joining. We appreciate it.
Jan Mickelson, President and CEO, Ascendis Pharma: Thanks so much for having us. Thank you.
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