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Citi 18th Annual BioPharma Conference

Sep 7, 2023

David Lebowitz
Senior Research Analyst, Citi

Oh, all right. Hello again, and welcome to the Citi Eighteenth Annual BioPharma Conference. We are coming up on the end here, but not before that, we have with us on stage Ascendis. They've certainly had some news, a bundle of news yesterday. Some they will be able to talk about more specifically, and others which will be teased with, but will not probably get too much information on. But beforehand, you can do your requisite disclosures.

Speaker 4

Thank you, David. We may make forward-looking comments during this presentation. Actual results may differ materially from those expressed or implied, and you should not place undue reliance on these statements. For information concerning the factors that can cause actual results to differ materially, please see the Risk Factors section of our most recent annual report on Form 20-F. Before I turn this back over to David, I wanted to make a comment regarding our PTH regulatory status of our US regulatory status of TransCon PTH. Jan provided a status update on our second quarter earnings call earlier this week, and we are not in a position to make additional further comments on this matter.

David Lebowitz
Senior Research Analyst, Citi

I had not to get rid of all my questions 'cause they were all on TransCon PTH. So with that, let's start. This is a company, a platform company with its TransCon technology. If you could tell us about the history of Ascendis and the modality you have developed, and where you're going.

Jan Møller Mikkelsen
President and CEO, Ascendis Pharma

Thanks, David. Thanks for inviting us here. And yes, I like to talk about the history about Ascendis Pharma. Ascendis Pharma got founded in, actually, some years ago, in 2007. It got founded with the acquisition of a company that really provided us the platform technology, the TransCon technology. And you can say: Why did we got inspired by the TransCon technology? Because when we wanted to found Ascendis Pharma, we had the same vision as we want today. We really wanted to take and develop product and bring it out to the patients. But when we see the risk that was inherent in making highly differentiated product, really addressing some major unmet medical need, we really needed to have a platform where we, at the same time, could make highly differentiated product opportunity, but do it in a highly successful manner.

It was when I first got exposed to the TransCon technology, I said, "Wow! For the first time in my life, I feel there is a platform technology that could be a paradigm shift." A paradigm shift where you can make highly differentiated product without taking the inherent risk of target engagement if you already work with validated parent drugs. So we built up a pipeline, and the first one was TransCon Growth Hormone. At that time, there has been 20 years research in making an improved treatment for, for example, pituitary growth hormone deficiency. An improved treatment that not only was more convenient with a weekly treatment regimen, but potentially also could give an improved treatment outcome. All attempts basically failed. Every time you change the growth hormone molecule, you got unwanted effect, immunological, antigenicity, side effect, or anything like that.

So what was unique with the TransCon technology? We could take the native growth hormone, that is the same as industrial growth hormone, that is the same as daily growth hormone, take it into the TransCon technology, make this prodrug, and then by a predictable release, when it got injected into the body, it will be transferred into the blood compartment and then release an unmodified growth hormone, the same as industrial growth hormone, in a predictable release kinetics that just reflect the PK parameter that you are seeing with daily growth hormone. And this was how we developed a pipeline in rare disease endocrinology. So when you can see the pipeline we built on in hypoparathyroidism, we have PTH 1-34, the same as Forteo. It got used off-label, PTH Forteo, in infusion pump.

lot of clinical trial was coming from peer-reviewed publications, where you can see if you just have a flat physiological profile, 20 hours, seven days a week, you really got the optimal outcome. That was exactly what we could mimic with a daily, small subcutaneous injection, and that was exactly what we built on that. And the same as CNP. We use wild-type CNP, well-known biology, well-known target engagement. So we built up a pipeline in rare disease endocrinology of three product opportunity. It was well-funded in our vision, three by three. And what I really are proud about, we made that vision for many years ago, and we said by 2025, we will have all three product opportunity being approved.

And when I look that in 2021, we get Skytrofa, I believe we will get the first approval here in 2023 with TransCon PTH, and in 2025, we have a clear pathway to get TransCon CNP approved for achondroplasia. And this is how we really have built up the unique, diversified pipeline with all three product opportunity really addressing major market, major potential of each of them. And not only we have a lot of synergy, economy of scale of having the three product in endocrinology rare diseases, but we also can see to get treatment synergies. For example, now we are planning for a trial between Skytrofa and TransCon CNP, going for achondroplasia, because we know if you want to have more linear growth, just combine the two product together. And not only there, we also use that in going into oncology and into ophthalmology.

It will not be areas that we will be focused as much as we are in commercialization. We believe we will have our 100%, our focus on endocrinology, where we will be from idea state out to the patients.

David Lebowitz
Senior Research Analyst, Citi

Got it. Thank you. In the past, when people thought of technologies to make extend the life, they thought in terms of administration. It made it more convenient, but it's clear that this approach is more than that. It adds to the efficacy by more reflecting the way a hormone or a drug might be administered in the body. Now, one challenge that you know certainly investors have always had is they get the logic when it comes to the endocrine side, and especially there's been plenty of evidence of it to this point. How does it... Should they look at the oncology side? I think a lot of investors have a lot harder time getting their hands around that.

Jan Møller Mikkelsen
President and CEO, Ascendis Pharma

Yeah. We have two product opportunities in oncology. They're basically built on the same fundamentals, because if you look on our TransCon TLR7/8, I believe that most people agree with the fundamental of TLR and how they function in an immunological stimulation. They're really functioning in really driving the maybe immunological system up to the next level. The question is, was how really to use it optimal in oncology? Because if you used it systemic, too much side effects. If you tried just to inject the compound inside a tumor, it disappeared after 2 to 3 hours. So we're basically injecting the tumor, after 2 or 3 hours, it's gone. So what we did with the TransCon technology, hydrogel technology, is to place the TransCon Hydrogel inside the tumor, and it basically will release the TLR7 over weeks.

By doing that, we really get the right immune stimulation inside the tumor. Basically, like a kickstarter. It's a kickstarter. It kickstart the immunological system. Now, inside the tumor, I can see the TLR function, I attract the right cells, I go in and really start the immunological system. This is exactly what we see now in our recommended phase two, where we both see monotherapy effect, we also see effect in combination therapy with different element. Our IL-2 is basic to build on all the scientific literature of what not to do and then do it right. Everyone know if you have an immunological stimulator and you can do it in a safe, good manner, then we basic will have something that really can be at the same level of checkpoint inhibitor, because it's a stimulator, not removing a break.

This has been the dream for many companies. What we did with our IL-2 is trying to build on the fundamental of the first immuno stimulation compound, Proleukin, that came out. We said, "You need to have at least the effect of Proleukin, both related to ALC, which are way to look on immuno stimulation, and then not have the associated side effect with it." Because IL-2 function if you can tolerate it, but you cannot tolerate it often. So this is how we develop it, and now we are really coming to the recommended phase 2, both as monotherapy, and we have really responders, partial responders in both that monotherapy and also in combination therapy. So I will let the data talk.

We are now going into indication-specific areas where we enrolling 20-40 patients in each single indication, seven different indications now, and we will have data coming out next year, which really can hopefully will show really the full potential of these two unique product opportunities.

David Lebowitz
Senior Research Analyst, Citi

Would the data be coming out incrementally?

Jan Møller Mikkelsen
President and CEO, Ascendis Pharma

Yeah

David Lebowitz
Senior Research Analyst, Citi

... based on tumor as it's ready, or in one drop?

Jan Møller Mikkelsen
President and CEO, Ascendis Pharma

When every indication coming to the stage where we have the first defined read for each single indication.

David Lebowitz
Senior Research Analyst, Citi

Got it. Skytrofa, you know, certainly the launch has been going well. I know early on in the launch, you've had a lot of people who were kinda trying to figure things out and understand how it would then actually ultimately progress. One question I have is, it's certainly doing well, to what extent are the patients coming on board switchers from daily, and to what extent are they treatment naive?

Jan Møller Mikkelsen
President and CEO, Ascendis Pharma

When we wanted to launch Skytrofa, we have two fundamentals that we want to achieve, to really to capitalize on really on the product strength on Skytrofa, really get the value out of this product. We wanted to be the market leader in value, and we wanted to do it in a growing growth hormone market. In Q2 this year, we already achieved to be the market leader in value. And we did it in pediatric growth hormone deficiency with less than 10% of the market share. Meaning is that if you do a simple calculation, if we penetrate the rest of the market and how we're selling, where we're selling, we will grow the entire growth hormone market for a $1.5 billion-$3 billion market segment in the US.

I feel that is right because we are providing a much better treatment outcome. We are giving a much better treatment outcome to the patient, and we're helping the society by really giving a better outcome. We did it in a way where we are providing patients both to naive and switch, but we get most patients from switch therapy. Meaning is that because we are not rebating in the same manner as the short-acting, most patients come on short-acting treatment, and all of them are identical. All the short-acting are identical. They're the same entity, it's just differentiation related to devices and formulations. And 80% of this patient, it will not get the optimal outcome. This is why most of them starts on daily growth hormone, where, switching over to Skytrofa, and this is where we see most of the patients coming from.

And I don't believe that it really will change a lot. It really improving now because the physician really have seen, observed now, how we really are... is a treatment different compared to the short-acting growth hormone products.

David Lebowitz
Senior Research Analyst, Citi

Treatment experience, as it's gone up, I mean, originally when it was launched, the commentary on switches is that these patients might not necessarily actively come forward. It might be on their periodic, you know, biannual visit to their doctors when the doctors might begin to start introducing them to this. Has that changed at all since, at this point, now that it's been out there a while, and there's probably a little bit more discussion about the nature of what this treatment offers?

Jan Møller Mikkelsen
President and CEO, Ascendis Pharma

So when we look in the underlying analytics, we don't see any element of seasonal variation. We see a steady state increase week by week, month by month, quarter by quarter, so we don't see anything that indicates that.

David Lebowitz
Senior Research Analyst, Citi

Got it. Got it. And, how do you expect the emergence of other long-actings could affect that?

Jan Møller Mikkelsen
President and CEO, Ascendis Pharma

We saw two other long-acting getting approved in the pediatric growth hormone deficiency here in the U.S. We feel that it's basic will improve the conversion over to the long-acting. We have never seen better uptake of Skytrofa after they launched. And I actually believe that is reflecting when people start to understand the labeling of the two other ones. The two other ones, based on the prescribing labeling, are comparing to a daily dose of 0.24 milligram per kilogram per week, which are about 40% under the normal treatment regime of daily growth hormone in the U.S. today. So then you can consider if you're using the prescribing dose of one of these two products. I'm not discussing the different side effects. I'm not discussing the different mode of action the different compound have.

We're the only one that is somatropin, giving the full endocrine benefit of somatropin. But just looking on linear growth, the basic will have an effect that is far under what you expect to just to see for normal daily growth hormone. And today, we're generating most of the switches for patient that is on daily growth hormone. And now you're suddenly implementing a new standard that basically will be 30%-40% under what you see with daily growth hormone if you use the prescribing dose of these other products. So this is why I don't believe any way it will just accelerate the uptake of Skytrofa.

David Lebowitz
Senior Research Analyst, Citi

Got it. Later this year, data in adult, how should we think of that?

Jan Møller Mikkelsen
President and CEO, Ascendis Pharma

Yeah. There's a benchmark for the two other product, as you talked about, in the adult growth hormone deficiency. The Ngenla, Sogroya, whatnot, from a statistical point, improved compared to placebo. Novo Nordisk's Sogroya managed to be in a position that they only got the treatment effect half of the daily growth hormone. We believe, because we are the only product of providing somatropin, we can at least get the same effect as we see with daily growth hormone. That is what our expectation. The data comes up in Q4. We're really thrilled about the adult growth hormone deficiency segment. It's very different compared to the pediatric, which are highly penetrated. The adult growth hormone deficient segment in our latest estimate is down under 5%-7% penetrated.

So there's a huge opportunity to further grow the growth hormone market and really providing the benefits to this under-treated, patient population.

David Lebowitz
Senior Research Analyst, Citi

How do you spur growth in a market that's so under-penetrated?

Jan Møller Mikkelsen
President and CEO, Ascendis Pharma

Education about the benefit of the treatment, and this is why we're really looking forward to have the data. We can educate about the disease today, but we really want to... When we have the data, we will be in Q4. We will file as fast as possible, so we basically can get it out to the patient in 2025, which is our intention.

David Lebowitz
Senior Research Analyst, Citi

... Got it. Now, you did a financing deal with SKYTROFA. Could you that you just announced a couple of days ago, could you tell us about that deal, what your thought process was in entering it, and I guess, how we should think about it going forward?

Jan Møller Mikkelsen
President and CEO, Ascendis Pharma

Scott?

Scott Smith
EVP and CFO, Ascendis Pharma

Yeah. So you're referring to the $150 million royalty funding agreement with Royalty Pharma? So we we've been in dialogue with various parties, including Royalty Pharma, for several, several years now. And I think that we came to a situation where we had a transaction that was just very attractive and really lowered the overall cost of capital for the company. We basically said we'll give you 9.15% of US Skytrofa sales only, so it's limited to US Skytrofa sales, and with a payback of 1.65x if paid back by December 2031. The first payments are due in Q2 2025.

So, actually, you could say even from the next year and a half or so, it's probably cash flow positive because we have pretty good interest on our cash from a carry perspective, about 5.5%. So, I think lower the overall cost of capital, provide some flexibility for the company to do different things if need be, and it was really kind of too good to pass up. And I think, you know, we've said repeatedly, we're committed to never issuing equity again. So I think this is probably a good example of what, you know, the trend we're going in with the capital structure.

David Lebowitz
Senior Research Analyst, Citi

Got it. Let's jump over to TransCon CNP. Certainly, the launch of Voxzogo is obviously going to be heartening for everyone who's pursuing it. But there's also been data now from BridgeBio, there's obviously Voxzogo, and then there's yourselves. How do you see this market shaping up over the years? And ultimately, how do you compare these therapies? I know people traditionally look at growth velocity, but ultimately, the question might become more complex than that.

Jan Møller Mikkelsen
President and CEO, Ascendis Pharma

I think the aspiration for us to move into achondroplasia is to buy a treatment for achondroplasia. And we see a treatment is not a focus on analyzed height, but addressing the comorbidities. That is the fundamental for us to be into achondroplasia. And I believe that the leading product is that is going to be the element that really are providing the benefit to this patient population, will be a product that, from a holistic perspective, address the comorbidity, including linear growth, is highly tolerable, have an excellent safety profile, and also have the convenience that is necessary to have it into a pediatric population that need to be on treatment, potentially not only in pediatric, but also when it come to the adult. That is exactly how we defined a leading product opportunity.

So we designed TransCon CNP to accommodate not only to provide linear growth, but addressing the element of comorbidity. We see achondroplasia not only as a growth disorder of the bone, but also a part of muscle weakness, which are complete different mode of action, which also can be addressed with continuous CNP. And why did we got this aspiration to look on the more holistic view of the phenotype of achondroplasia was when we observed in our phase 2 trial, how patients suddenly came and explained things has changed for them. They're feeling much more comfort, that basically could have better balance, they could have much better motoric muscle action. And then we started really to review literature, and clearly, it's been proven that achondroplasia is also part of a muscle disease.

What we basically could do can address all, both the linear growth, and we can address what we call the comorbidity, many of them arising from the combination of muscle weakness and growth disorders, growth bone disorders. And we did it with a once-weekly dosing profile because we know in the pediatric how difficult it is to build adherence in a pediatric population with a daily injection. We did it out from the excellent safety profile we want to have and tolerability in it. And when I look on the data now we have in our phase 2 trial, open label extension, we started with 57 patients three years ago. Today, we have 57 patients still on treatment. And I don't believe them, they're just in there for linear growth. They are because we provide a treatment of achondroplasia, providing the benefit they were seeking for.

Now we have the scientific understanding because we integrated the muscle component, because we could not understand why, after a few months, this patient have a complete different behavior. That is what we understand. We enrolled all our 80 plus patient in four and a half months. We enrolled in, in sites that got exposed to us in our phase two trial, 90% of the site. On the site, they also have access to corticosteroids and other treatment, but still, after four and a half months, we had all the patients. It's fully enrolled now. All the patient are in next year-... in second half, we will come out with a phase three data. We went out to reg- regulatory agencies in Europe and US. Our primary endpoint is annualized height velocity. This is typical way you look on growth disorder.

The second element we discussed was our dose. Everyone understood 100 microgram per kilo per week is the right dose to give. We talked about the comorbidity. We have a continued discussion with the agencies, how we really can prove that we're providing something more than linear growth. We have three, four different way. We are trying to get that integrated in our phase 3 trial. So we feel really, really confident we have the strongest package ever that being developed for any achondroplasia treatment. The second part is that people have so much focused on linear growth. There's a lot of element to consider when you talk about linear growth. Linear growth is only meaningful if you look it over one year. This is why we ran our phase 2 trial over one year.

Because if you take Achondroplasia patients, typical, the average annualized height velocity in the age group from 3, 4, 5, 6, 7, 8, 9, is about 4-4.2 cm. And what we observed, and everyone has observed, if you start with a patient that, for example, grew six months before that goes down to 3.5 cm without any treatment, anything, the growth is in the second half about 4.5-5 cm without anything. So this is why you cannot really adjust linear growth without looking at one-year treatment. So if anyone want to compare any trial, look at 12-month data, or you just will be treated in a way where you basically cannot adjust it if you're not 100% sure that the annualized height velocity is on starting on average about 4.3-4.2.

So when I look on our package we building up, it's going to be highly integrated. We also initiate clinical trial in the newborn. We just filed for and starting that trial. We also going to have a combination trial between Skytrofa and CMP. If someone wants to grow more, take Skytrofa together with it. It's from a biological system, and just look in our IP, where there's a lot of preclinical data about treatment synergy in animal models. Yes, this is how if you really want to grow more, it's a good way to combine these two things, if you want more linear growth. So I believe that the integrated package we're going to develop is that we want to be leading company in growth disorder. We have the two cornerstones for being the leading company in growth disorder: the leading TransCon growth hormone and a once-weekly CMP.

We can really optimize them to really address the 20+ growth disorder that exist out here.

David Lebowitz
Senior Research Analyst, Citi

You talk about how the importance is really more the comorbidities and how it's more meaningful ultimately than just the pure growth velocity. But of course, the way the regulatory process has been set up, it's been set up revolving around growth velocity. And how does that challenge the ability for various players to be able to present that other information when the headline has been set up in a different direction? And does that weaken the nature of the data that it gets presented on some of the other topics? How long does it take till that data becomes the headline?

Jan Møller Mikkelsen
President and CEO, Ascendis Pharma

I think it's a question where you need to say, what is really the audience you are addressing? Is it the physicians? Is it investors? Is this other audience? If I talk with the physician, we went to the site where they have a lot of different option. They get the enrollment in 4-5 months, indicating the physician understand the value of TransCon CNP. Do investors really understand that in the same manner as the physicians? I think someone understand it. They really understand the fundamental of the way to really just scientifically data and really take up and understand and spend the time to understand the data, what really means. But you need to take it from the perspective on linear growth, it's a single way just to look on one single parameter in the holistic treatment of an achondroplasia.

If you can come with any correlation that exists between providing linear growth and then address comorbidity, it's not existing. No one have proven that. Even if they're proving to address linear growth, no one have proven they really address comorbidities. This is where we want to be. We want to address the comorbidities, and this is the differentiation, and I believe firmly that this is what the patient really wish for.

David Lebowitz
Senior Research Analyst, Citi

Perfect. Tell us about the data that you've presented at this point and what we should expect with the future updates.

Jan Møller Mikkelsen
President and CEO, Ascendis Pharma

On TransCon CNP, we will have an update later this year. This year is not over for us. I will not say it's just beginning, but there are so many things that are happening. We're waiting for the European approval of PTH, which we hope will come soon. We're waiting for resubmitting our PTH filing in the U.S. We're waiting for adult data for, here, phase three data with—really could be interesting. We will give you also an update about CNP, about where we have all of them treated, 57, with the highest dose for, I think, one year now. Really are giving an impressive update where we can give you our idea, how we look on the biology, how we want to address comorbidity. Scott, I forgot something obvious. What did I forget?

David Lebowitz
Senior Research Analyst, Citi

For the rest of the, if-

Jan Møller Mikkelsen
President and CEO, Ascendis Pharma

Yeah, for rest of the year.

David Lebowitz
Senior Research Analyst, Citi

Growth hormone, adult?

Jan Møller Mikkelsen
President and CEO, Ascendis Pharma

Yeah, go ahead.

David Lebowitz
Senior Research Analyst, Citi

Oh, approval.

Jan Møller Mikkelsen
President and CEO, Ascendis Pharma

Okay.

David Lebowitz
Senior Research Analyst, Citi

That was, that was me. I wasn't gonna say that one, too.

Speaker 5

Mm.

David Lebowitz
Senior Research Analyst, Citi

Yeah, the drug I'm not supposed to mention. When you—I know that we're not talking about the regulatory process, and I will spare you a question on that. Although, no, I'm thinking of the question. Can you talk to the data you released earlier this week? Is that-

Jan Møller Mikkelsen
President and CEO, Ascendis Pharma

The EGFR?

David Lebowitz
Senior Research Analyst, Citi

Yeah, the eGFR data. Is that, is that within the bounds of the

Jan Møller Mikkelsen
President and CEO, Ascendis Pharma

Yeah, we can talk about the CKD data. Yeah. So the data came up because we're starting to prepare for a launch in Europe. Launch in Europe is going into a lot of health economic calculation about the benefit of providing a treatment to a hypopara patient. One of the key element we were looking, we were looking about all the data one time more. We started to look on the impact of kidney. And I think the key element for us is that if you look on the 60 eGFR, if you have less than 60 eGFR in estimated filtration rate, you basic have what we call kidney dysfunction. Meaning is that you are in a very, very expensive pathway for any treatment for the society.

What we saw with this data, after one year of treatment, 50% of the patients moved from having the diagnosis of stage 3 kidney dysfunction, stage 3 to be normal. It's really providing us a lot of the health economic discussion with the authorities in Europe about how we really talk about the optimal price. For me, it was surprising. I never seen a drug, and perhaps I'm not seeing all drugs, that basically is providing such a benefit even when you have renal impairment. It not only stabilizes, but it's actually improving, which I think was really, really, really unique. And when we came out and talked with physicians about it, perhaps I was enthusiastic, but the physicians were definitely more enthusiastic than me about...

Because they're saying, "Now we really also can help this comorbidity." Not only can we really give them their life back on a lot of different parameter, normalize serum calcium, normalize the quality of life, give them normal phosphate, give them a normal bone metabolic system, but we also can, even if you have been having the disease for so many years, you're starting to have renal impairment, we also can help you there. So I actually think it was some of the most impressive data we have seen for a long time.

David Lebowitz
Senior Research Analyst, Citi

Now, you also, earlier this week, talked about kind of next steps for TransCon platform itself, and you provided some data, preclinical data. Could you tell us about, number one, the overall platform itself and how it will evolve, and then specifically about this candidate and the potential for the indication? Obviously, it's GLP-1 is something a lot of people are talking about these days.

Jan Møller Mikkelsen
President and CEO, Ascendis Pharma

Yeah. I talked about the TransCon technology in the beginning, and in the TransCon technology, we have TransCon linkers, which are the same linkers we use again and again to all the different programs, different families, different generation. And then we have the TransCon carriers. And typically, what you have been talked about is the carriers we used in the product like TransCon Growth Hormone, TransCon PTH, TransCon CNP, our IL-2 program. We use soluble polymers. Then we have the hydrogel technology, which are coming from the TLR7/8.

But what we really was missing, we missed a part of an expansion of the TransCon technology, where we basically couldn't go out and really achieve two things, besides all the other benefit you get out of the TransCon technology, like the product part and other things like that, that we basically couldn't expand to something high volume manufacturing and extremely low costs. Perhaps even on a drug product, basic safe in manufacturing cost. This was why we developed this new technology platform. We examined it with a GLP-1, semaglutide, because we thought that it was a good way to show the power of this technology. When I thought what we can do with semaglutide, it's very simple. We release from the TransCon technology the same unmodified semaglutide that people now getting exposed to, both in diabetes and obesity today. It's exactly the same entity.

But through the TransCon technology, we basically achieving everything what we want to achieve, have the prolonged release, increasing the half-life about four or five times on it. And what would that mean? It mean that you can position in different way. You can go for a treatment where you basically from weekly dosing will have complete flat profile of a semaglutide, avoid the fluctuation that potentially give tolerability, move it potentially higher up in, concentration in the blood concentration, meaning that you can get better effect. Or you could go for an adherence play, and saying instead of having the tolerability issue with four times of injection in a month, you only inject one time inject, and you will have the same peak to trough that you will see with semaglutide today on a weekly dosing profile.

We exemplified it with semaglutide. We couldn't use many of the other GLP-1 mimic, but we thought it was a good compound for us to work on and make the example on it. But the technology platform will not only work for other GLP-1s, it will also can be function in other areas where we believe the same fundamental could be, like in cardiovascular, where there's a lot of peptide that really can have major impact if you find an optimal way to produce it, and also on the fundamental of the, you can say, the cost structure that is in this business in it. So we really, really highly embrace that we really managed to expand the TransCon technology also to include that part of our product opportunities we now really can address with our technology platform.

David Lebowitz
Senior Research Analyst, Citi

So with that, when do you think we could see a candidate with this variation of the platform actually enter the clinic?

Jan Møller Mikkelsen
President and CEO, Ascendis Pharma

Yeah, that is a thing when we have all the plans detailed, we will come out, and typically we always talk on new elements of our pipeline, new elements of timeline. At the JPMorgan conferences is always a good time when we come with our guidance when we see things happening. What we want to do is to say that we are a company that really are focused on endocrinology. This is where we build up our focus. We see a huge use of the TransCon technology in other areas, but in the end, we will not be an oncology player. We will be a concurrent player in the way where we will have partnership, we will work with paradigm shift compound and really bring the value forward for the patient and Ascendis.

David Lebowitz
Senior Research Analyst, Citi

Towards that end, if we look at something like GLP-1 or the diabetes endocrine space, but also the nature of doing trials and is a much grander scale than where have you been, how would you approach such development?

Jan Møller Mikkelsen
President and CEO, Ascendis Pharma

Yeah, but the element of what we're doing is that it's mainly execution risk you have, because you're being able to provide the active entity is a well-known, like a growth hormone. Was there really a lot of risk in our phase one, phase two, phase three trial? No, that was not major risk because it was liberating the same entity that we know that was function. Here, we're liberating the same entity with Semaglutide that also will function. So sure, it's a larger trial, but the other point is trials where you can see it's pretty predictable, the outcome on it, what you will see in this kind of system, because you will just see the benefit of what TransCon technology is providing on already established mode of action.

David Lebowitz
Senior Research Analyst, Citi

Got it. We have a few minutes left here. I'm gonna let you wanna let you freestyle. What, what, what do you wanna tell us about that you haven't, haven't told us about to this point?

Jan Møller Mikkelsen
President and CEO, Ascendis Pharma

I think the key thing for us in JPMorgan in 2019, we came up with a vision, three by three. We wanted to be a sustainable leading biopharma company by 2025. The cornerstone for that was three product opportunities: TransCon Growth Hormone, TransCon PTH, TransCon CNP, and then an opportunistic approach in other therapeutic areas. What we have managed to do is execute on all these three product opportunities, keep the timelines, keeping all the elements that we wanted to achieve. And when I look on the Skytrofa success in the US market, how we commercializing, we also proven now we really have a strong, solid, competitive way to commercialize products. And we will do the same thing with PTH. We will do the same thing with CNP.

So we're building Ascendis Pharma to really a profitable company, and our goal is to get our first quarter profit next year. We hope we can achieve it. We're doing everything to achieve it. It will be really by growing the revenue on an international operational plan. We, out from Copenhagen, has established what we call our global commercial organization, besides the US, that both are focused on Europe and international operation, and we will bring what we call revenue from all this side here. So the perspective of Ascendis, as Scott has said many, many, many, many times, we're not here for selling equity more. We're here to show off the value we're getting out of the product opportunities that we already have to the benefit to the patient.

I believe we really are fulfilling our vision three by three, where we're adding on, we also want to be highly profitable.

David Lebowitz
Senior Research Analyst, Citi

Excellent. With that, thank you very much for your time. Appreciate it, as always, and look forward to seeing you again soon.

Jan Møller Mikkelsen
President and CEO, Ascendis Pharma

Thanks so much.

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