Q2 2021 Ascendis Pharma A/S Earnings Call

[music].

Operator: Thank you for standing by. Welcome to Ascendis Pharma's Q2 Earnings and SkyTOFA Approval 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 the session, you will need to press Star 1 on your telephone. Please be advised that today's conference is being recorded. If you require any further assistance, press Star 0. I would now like to hand the conference over to your speaker today, Scott Smith, Senior Vice President and Chief Financial Officer of Ascendis Pharma. Please go ahead.

Good day, and thank you for standing by welcome to assemblies for Merck Q2 earnings.

Sky Sofa approval call at this time, all participants are in a listen only mode. After the speaker's presentation, there will be a question and answer.

Answer session to ask a question during the session you will need to press star one on your telephone PCB adviser today's conference is being recorded if you require any further assistance press star zero, although like to hand, the conference over to your Speaker today, Scott Smith, Senior Vice President and Chief financial.

Actual officer at F&B Pharma. Please go ahead.

Scott Smith: Thank you everyone for joining our conference call today. I'm Scott Smith, Chief Financial Officer of Ascendis.

Thank you operator.

Thank you everyone for joining our conference call today, and Scott Smith, Chief Financial Officer of ascend is joining me on today's call as the unmet Olson President and Chief Executive Officer, Jesper Hoiland Global Chief Commercial officer.

Joining me on today's call is Jan Mikkelsen, President and Chief Executive Officer, Jesper Hoyland, Global Chief Commercial Officer, Dr. Dana Pizzuti, Head of Development Operations and Chief Medical Officer, and Dr. Yuhua Punanen, Head of Oncology. Before we begin, I would like to remind you that this conference call will contain forward-looking statements that are intended to be covered under the Safe Harbor provided by the Examples of such statements may include, but are not limited to, our commercialization and continued development of SkyTrofa, our progress on our pipeline candidates, and our expectations with respect to their continued progress.

Dr. Dana <unk> head of development operations, and Chief Medical Officer, and Dr. Yu Harpoon, and then head of oncology.

Before we begin I would like to remind you that this conference call will contain 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 our commercialization and continued development of Sky Trophy.

Our progress on our pipeline candidates and our expectations with respect to their continued progress.

Statements regarding our strategic plans, our goals regarding our clinical pipeline, statements regarding the market potential of SkyTrofa and our pipeline product candidates, and statements regarding our regulatory activities. These statements are based on the information that is available to us today.

Statements regarding our strategic plans our goals regarding our clinical pipeline.

Statements regarding the market potential of skyscraper and our pipeline product candidates and statements regarding our regulatory filings.

These statements are based on information that is available to us today actual results or events could differ materially from those in the forward looking statements and we may not achieve our goals carry.

Actual results or events could differ materially from those in the forward-looking statements, and we may not achieve our goals, carry out our plans or intentions, or meet the expectations or projections disclosed in our forward-looking statements, and you should not place undue reliance on these statements. Our forward-looking statements do not reflect the potential impact of any licensing agreements, acquisitions, mergers, dispositions, joint ventures, or investments that we may enter into or terminate. We assume no obligation to update these statements as circumstances change, except as required by law.

Our plans or intentions or meet the expectations or projections disclosed in our forward looking statements and you should not place undue reliance on these statements.

Our forward looking statements do not reflect the potential impact of any licensing agreements acquisitions mergers dispositions joint ventures or investments that we may enter into.

Kerry M&A.

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 to differ materially, please see the forward-looking statement section in today's press release and the risk factor section of our most recent annual report on Form 20-F. Skytropha was approved by the FDA today for the treatment of pediatric patients one year and older who weigh at least 11.5 kilograms and have growth failure due to inadequate secretion of endogenous growth hormones. Otherwise, please note that our product candidates are investigational products and have not been approved for commercial use. As investigational products, the safety and effectiveness of the product candidates have not been reviewed or approved by any regulatory agency.

For additional information concerning the factors that could cause actual results to differ materially. Please see the forward looking statements section in today's press release and the risk factors section of our most recent annual report on form.

Or.

Sky Trophy was approved by the FDA today for the treatment of pediatric patients one year and older who way at least 11, five kilograms and half growth failure due to inadequate secretion of endogenous growth hormone.

Otherwise please note that our product candidates, our investigational product candidates and have.

Have not been approved for commercial use.

As investigational products, the safety and effectiveness of the product candidates have not been reviewed or approved by any regulatory agency.

Jan Moller Mikkelsen: None of the statements made on today's conference call regarding Skytropha and our product candidates should be viewed as promotional. On today's call, we will walk through a presentation available on our website to discuss the FDA approval of Skytropha. We'll discuss our second quarter 2021 financial results and provide further business updates. Following some prepared remarks, we will then open up the call to questions. I will now turn the call over to Jan Mikkelsen, our President and Chief Executive Officer.

None of the statements made on the conference call regarding Sky Trophy, and our product candidates shall be viewed as promotional.

On today's call we will walk.

20th Sententious available on our website to discuss the FDA approval of <unk>.

We will discuss our second quarter 2021 financial results and provide further business updates.

Following some prepared remarks, we will then open up the call to questions.

I will now turn the call over to UN Michelson, our president and Chief.

Throw creative officer.

Thanks, Scott and good afternoon.

Sometimes when you wake up in the morning, you somebody think about what they will bring.

I have to say today was one of the days, where you somewhere you had him agenda you adapt to it like a dominant.

Thanks, Scott, and good afternoon. Sometimes when you wake up in the morning, you think in some way about what the day will bring.

This mistake you need to adapt this is the best way of survival and I think we are in a great great place today and I have to say if I couldn't see ya.

But I have to say today was one of the days where you had an agenda, you adapted to it, like Darwinism says, you need to adapt; this is the best way of survival. And I think we are in a great, great place today. And I have to say, if I can see you, you will see a very, very happy ending.

C band, we're happy on that basis.

So what we have achieved to date.

But new offer great.

<unk>.

He picks a key element in our core values is to make a meaningful improvement in patients' lives.

We feel strongly that with our focus on science and data.

Use our innovative transcon technologies to develop new and potential best in class products that address major unmet medical needs.

That basically, we are so happy about what we have achieved today, but never forget. At Ascendis, the key element in our core values is to make a meaningful improvement in patient life. We feel strongly that with our focus on science and data. We can use our innovative transcon technologies to develop new and potential best-in-class products that address major unmet medical needs. For example, with the approval in the U.S. of Transcon Growth Hormone, or Skytropa, for pediatric growth hormone deficiency today.

The.

Approval in the U S of transcon growth hormone or Scotch Wilbur for pediatric growth hormone deficiency today.

We are one step Neal.

To fulfill our ambition suite buttery to become a leading Biopharma company.

Paul I'll provide a general business update related to our <unk>.

AMC broad pipeline.

No.

More and more product opportunities I would like to give a short update related to the approval of Skype children as Scott said.

This can be found on <unk>.

This side and I would like to referring to the slides.

We are one step near to fulfilling our vision tree by tree to become a leading biopharmaceutical company. Before I provide a general business update related to our advancing broad pipeline of now more and more product opportunities, I would like to give a short update related to the approval of Skype Trover. The slides can be found on our website, and I would like to refer to them.

Going to slide number three.

Sky <unk>.

Our first product developed.

Turning to technology.

<unk> is now approved in the U S.

<unk> technology is a technology, we have taken basic from the design to preclinical.

Two clinical now.

In the U S.

Moving to slide number three, Guy Tauber, our first product developed with Transcon technology, is now approved in the U.S. The Transcon technology is a technology we have taken basics from the design to preclinical, to clinical, and now to approval in the U.S. At the same time, it's the first approval of the Transcon technology, but it's also the first FDA-approved one-treatment product for pediatric growth pneumonia, and we're really proud about it.

At the same time, it's the first approval of the.

The <unk> technology, but it was also the first 50 approved once a week.

For pediatric growth hormone deficiency.

And we really proud about it when we go to the next slide and our ex U.

You can see the label different places you basically we'll see what we.

<unk> outcome the label is basic wont be basic.

Hopeful and it's basically reflecting the science the clinical data, we obviously completed.

To date because of time I will not go through the entire lapping discussion but.

But I hope you.

So opportunity to look at because it <unk> start with the motive action, where it basic are describing how we re leasing.

When we go to the next slide, and I actually think you can see the label in different places, you basically see what we have seen out from the label is basically what we could basically hope for. And it's basically reflecting the science, the clinical data we have received with it. Today, because of time, I will not go through the entire labeling discussion, but I hope you will have the opportunity to look at it, because everything starts with the mode of action, where we are basically describing how we release and Sumatopine, the same as daily Glutamone, in going to the right efficacy, it goes to the right safety.

So much with the same aesthetical from going to the right. If we could see it go through the right safety and we're feeling really really confident.

With the approval and the labeling we have we have a strong fundamental to build up schedule, but transcon growth hormone to be the leading brand.

But it has not been an estrogen for the patients if you go to slide five.

I often.

You will help you with this discussion.

It has been possible to develop a long acting.

Product that basic providing.

And we're feeling really, really confident with the approval and the labeling we have. We have a strong foundation to build up Sculfoba Transcon Glutamone to be a leading brand. But it has not been an easy journey for the patients. If you go to slide 5, I have often given you this discussion. It has been possible to develop a long-acting, Product, The Basic Providing and Addressing the Unmet Medical Needs. Specific you have in a pediatric population about a daily injection.

And addressing the unmet medical need specific you have in a pediatric population about a daily injection.

And despite.

30 years.

Clinical development try developing by different kind of concept currently today in the U S in Europe and Japan.

Sure.

Patients with go to more deficiency.

Have these treated with.

The same entity at exit <unk> into the market in the eighties.

And despite, Thank you, clinical development, trial development by different kinds of concepts currently today in the US, in Europe, in Japan, all work, patients with growth hormone deficiency are treated with the same entity that actually came into the market in the 80s. There have been advancements in pen devices.

It has been advancement in pen device that has been advancement that one single product opportunity also can make room temperature stability.

What we see.

<unk> on the approval.

To date.

This is the first once weekly.

Opportunity that basic are providing.

What we call the same entity that we see in <unk>.

There has been advancement that one single product opportunity could also make room temperature stability, as we see with SkyTrooper under approval today. This is the first once-weekly opportunity that Basic are providing what we call the same entity that we see in Davy Crook tomorrow. But we also have built up what we call the optimal element for the patient, life, and Auto Injector, which enables you to have room temperature stability while in use. When I talk about all the clinical programs that support the approval of Scott Holmes, it's built on the basics, the high trial, the flight time, and the enlightenment. The High Trial, just two.

Well if you also have what we call optimal element for the patients life and.

Auto injector.

Which facility that you have room type book to induce stability.

When I talk about all clinical programs that support the approval on schedule for.

It is spent on basic.

The high trial, the slide type and their document trial.

The height trial.

Just two.

Some up this way really.

Had clinical trials with nave patients.

We also had a switch phy and the flight trial and we continue.

In the lightning trial to generate long term data how the benefit of our.

Sum up, this way I'll be really..., had clinical trials with new patients. We also had a switch trial in the flight trial, and we continue, in the enlightenment trial, to generate long-term data on how the benefits of our product are really developing on a long-term basis, both related to efficacy and safety. That is our commitment, really to ensure that we're building up the most compressive clinical program as support of this approval. The Hydra, as you recall, was a direct comparison to daily glutamone at the same dose.

Product really developing on long term basis, both related to efficacy and safety.

That is our commitment.

Really to ensure.

That would be building up the most compressive clinical program as support of this approval the.

The height trial as you recall.

Was a direct comparison to at daily optimal at the same dose.

And we.

The patient four two to one and its basic.

Element in the approval related to efficacy.

And we randomized the patient for 2 to 1. And it's basically the main element in the approval related to efficacy. In Slide 8. You can see the result for yourself. And we saw and better analyzed high-velocity at 52 V, illustrated from the statistics perspective under Confidence in Zabard, go into safety.

In slide.

You can see the results.

And we.

So and better annualized.

High velocity at 52 weeks.

Illustrated from a statistical perspective under confidence into that.

Going to the.

And when you talk about pediatric treatment, safety is extremely important, as is all other treatment. But when you think about that you have established and safe, from daily growth to mow, it's very hard to believe that you can compromise, and one we saw. In the summary of a birth event and everything we look at, we basically have a similar profile to daily growth tomorrow, including an emogenic profile.

Safety and when you talk about pediatric treatment safety is extremely important.

<unk> in all oil treatment, but when you think about that you have established and safety.

From a daily growth hormone.

It's very hard to believe that you can compromise that.

And when we sold.

In the summary of adverse event and everything.

What we look for we basically had a similar profile like daily growth hormone.

Included in organic profile.

Yeah.

Now we're coming to the next stage. We don't want to develop, Transcribed by https://otter.ai, a product addressing one single geographic region. We want to develop Transcontinental Moon up from a global commercial strategy. We have already established how we really want to address the two largest commercial markets, the U.S., where we're building up our own commercial organization. We do it because we have a pipeline.

Now we are coming to the next states.

We don't.

Everything to develop.

Transcon growth hormone.

Two.

Our product addressing one single grew graphic region.

I want to develop.

Transcon growth hormone up from global commercial strategy.

One.

We have already.

Establish how we really want to address the two largest commercial market. The U S. We have been building up our own commercial organization, we do it because we have a pipeline we have a pipeline.

We have a pipeline of rare disease endocrinology products, multiple products in the same therapeutic area. We are in a position that we are ready there, and in the next few slides, we basically will have a presentation from Jesper that will go in and go more in detail. But before I start, I would like to give you the perspective of the Gotham Market. We see the growth-to-money market in a global setting of around more than for a bit. The US market is around 1.3 billion. Well, the podiatric growth hormone estimated market is about 700.

Rare disease.

Endocrinology product multiple products in the same therapeutic area.

We are in a position that we are ready there and in the next few slides basic will have and presentation from.

Yes, Bob.

C to go in and go more in detail, but before I start I would like you to give you the perspective of the Kokomo market.

See the growth hormone market in a global setting of around more than $4 billion.

The U S market is.

It's around $1.3 billion.

We have a pediatric growth hormone estimated is about $700 million.

Jan Moller Mikkelsen: But we've also seen that there's a market that's highly fragmented with multiple products with basically the same product. And this is where we believe the market is really primed for disruption. Time for innovation that has been lacking for 30 years. And that is what we come up with. And this is why we believe we have a possibility, not only to improve the treatment, but also to improve and expand the market sector. Jesper, will you go over and talk more dedicated to the U.S. commercial effort?

But we're also seeing that as a market that's been highly fragmented.

Multiple products with basically the same product.

And this is where we believe the market is really price.

Primed for disruption.

Time for innovation that and that's been lacking for 30 years.

And that is what we come with and this is why we believe we have and possibility.

Not only to improve the treatment, but we also have a possibility to improve.

<unk>.

And expand the market segment.

Yes, but when you go over and talk more dedicated related to the U S commercial effort.

Absolutely.

This is the moment of truth for me.

I have to say that I've been waiting for more than 30 years for this very moment, so it's very emotional.

I have to say that I've been waiting for more than 30 years to this very moment. So it's a very emotional.

I developed my first growth hormone back in 1988, and even at that point in time, everyone was saying, "Why can we not have it?"

I sold my first growth hormone back in 1988.

And even at that point of time, everyone was saying why.

Why can we not have a once-weekly growth hormone for little John, for the patient?

You may not have a once weekly growth hormone for Lilly John for the patients.

Now it's done.

With Guy Troffer, we can truly shape the market and change the market.

The sky chokes on truly can shape, the market and change the market.

For me, myself, who has spent my entire career in the endocrine space, I can only say this is truly the first commercial launch that we'll make with Ascendis and with SkyTrooper.

But me myself, where I spent my entire career in the endocrine space I can only.

<unk>. This is truly the first commercial launch that will make with our centers and with Sky Chopa enabled paved the way for the success that we've been waiting for.

that will work with Ascendis and with SkyTrooper, and it will pay.

And you can ask me then.

What are you our ambition and I can only say to drive market leadership.

See you then. Market leadership is the absolute term, because then you determine what the market should look like. And there are some pillars as a part of that, that are pointed out on the slide that you hopefully see in front of you on slide 12. First, build creatability.

<unk> SEC.

Market leadership.

Absolute time.

Because then you determine what the market should look like.

And there are some pinterest as a part of that that has pointed out that the slides.

Hopefully see in front of you on slide 12.

Firstly.

Schrader ability.

You hire the right people who can do the job.

Hire the right people, who can do the job.

That's done.

Then you realize, market action.

And then you realise market access.

We are in the process of working to get the ad market access that we need for SkyTroop. But then we also need to

We are in process of working to get the AD market access that we need for Sky Trophy.

Then we also need to motivate.

Then we also need to motivate caregivers.

Cant gave us because at the end of the day.

You cannot have the world's best rock if you do not have the patience and the caregivers.

So you can have the world's best rock if you do not have the patients at Mccann gave us engaged into it you will not make any strong stripes.

not make any strong strikes. We are working on that, and we are in the process of doing that. And then, of course, all the health care providers.

Working on that and we are in process of doing that and then of course, all the health care.

The titles, we are working on.

One of the first efforts, if you go to slide 13, that we have been working on is

One of the first let's say if you go to slide sort of change that we've been working on is building the educational efforts to truly believe what those outgrowth G. H D stands for.

is building the educational efforts, and truly believes what those outgrows in GHT.

We launched that back in February.

We launched that back in February and as we are working on it right now.

Unique visitors to Our Site

That would take as a sign of the huge interest. That is for Sky Trooper. And for Donut Packs, so many open.

We provide we have more than 100000 unique visitors to our site.

That would take as a sign of a huge interest.

That is for Sky Trophy.

<unk>. Thanks, so much okay.

We strongly believe that we're coming out to the market, and we can make a difference. We are also very fortunate in a way because the U.S. market, and now I'm talking or speaking on slide 13, we are...

We strongly believe that we are coming out to the market and we can make the difference.

Now we also very fortune in a way because the U S market and now I'm talking on speaking to slide 13.

We are addressing the pediatric endocrinology segment, which is relatively small.

Addressing the pediatric endocrinologists segment, which is relatively small.

1400 subscribers in total. And it's a very concentrated market; about 80% of all prescriptions come from about 20% of physicians, and we are certainly going to address that market.

You're talking about.

1400 describe us in children.

And it's a very key.

Traded market.

<unk>.

80% of all prescriptions come from about 20% of the sufficient and we are certainly going to address that market.

I am absolutely certain that we are taking and doing the right steps going forward for us to be successful.

I am absolutely certain.

Going and doing the right steps going forward for us to be in the right place.

So, for me, with the team that we have built with my colleagues...

So for me with the team that we have built with my colleagues, we are absolutely ready from this moment on to.

Colleagues, we are absolutely ready from this moment on to move forward. We have been waiting for it. And now is the moment of truth.

To move forward, we have been waiting for it.

Now is the moment of truth as I say.

One of the key components for us is going to be the Essential Signature Access Program.

One of the key components for us is going to be their sense of signature.

And it has different elements added to it.

Okay.

Asap.

And it has different elements to it.

All the colleagues that I'm working with have a long, long tenure in the area of endocrinology, in the area that we are going to work in, but certainly also in the area of the pharmaceutical industry. So we know what it takes to build true access.

All the colleagues that I am working with has a long long tenure in the area.

Endocrinology.

In the area that we're going to work in but certainly also in the area of the pharmaceutical industry. So.

No what it takes to build it.

True access to the market.

It takes patience and commitment. We are working on that part. It also takes motivation.

It takes patient enrollment.

We are working on that part.

[inaudible] and all these components we have.

It also takes motivation and access.

All these components were put together in the program of ASAP.

And all of these components were cooked together and the program of a S. A P.

of the Ascendis Signature Access Program for Skytropa.

Their centers.

So we'd mature access program for Sky Trophy.

And I really, really feel comfortable where we are right now, and I cannot wait.

And I really really feel comfortable where we are right now and I cannot wait.

I hope to meet all of you going forward and do the work that has to be done from now on.

To meet all of Europe going forward.

And do the work that has to be done from now on we have staggered in.

Jan Moller Mikkelsen: We have staggered the engagement with our colleagues.

<unk> with the colleagues so we.

So we took on...

The first and foremost important ones and now.

Second the first and foremost important months and now we're going to hire the rest of the team on board. So we have also financial been various student to what we're doing.

The most important ones, and now we're going to hire the rest of the team on board, so we have also financially been very careful to what we are doing. And I do believe that we are in the absolute spot where we want to be. And with that in mind, Jan, I would like to pass it over to you, for you to expand on all the things we've been doing, even outside of the US and with SkyTrooper and all the preparations that have happened. But thanks a lot for everyone who will listen. I cannot wait!

And I do believe that.

We are in the absolute spot, where we want to be and with that in mind, yeah, and I would like to pass it over back to you.

You.

We took expands on all of the things we have been doing.

Even outside of the U S and with Sky <unk> and all the preparations that has happened.

Thanks, a lot for everyone to listen I cannot wait to see you in the not too distant future and XO conferences.

Thank you Jesper.

A key part of our recent three by three is our global clinical research and label expansion.

In Europe, we expect a decision from the EMEA Commission on our M&A for Transcon cocoa.

Jan Moller Mikkelsen: Thanks a lot for everyone who has listened. I cannot wait to see you in the not-too-distant future.

For the treatment of pediatric growth hormone in the fourth quarter of this year.

A key part of our vision, Tree by Tree, is our global clinical reach and label expansion. In Europe, we expect a decision from the EMEA Commission on our M&A for transconcoctal mold for the treatment of pedagogical mold in the fourth quarter of this year.

In Japan, we continue to enroll patients in the fortunate soft decreased three right trial for pediatric growth hormone deficient.

And in China.

<unk> pharmaceuticals.

Enrollment of this phase III clinical trial of transcon growth hormone in children, the Kokomo deficiency.

In Japan, we continue to enroll patients in the 40-subject phase 3 right trials for pediatric growth and mobility. And in China, Li Jinfeng has completed enrollment in his Phase III clinical trial of transconverter mole in children with growth hormone deficiency in masks this year. We also have the Global Phase III Foresight Trial underway for adult glutamate deficiency to support our planned label expansion for transcontinental growth tomorrow. Looking past the Forsyth trial, we expect additional studies to support our effort to expand the label beyond pediatric and total growth hormone deficiency. I'm going to the last slide, and it's going back to where I started with our vision of the patient. This is why I go to work. This is why we're living in Ascendis.

Plus this year.

We also have a global phase III foresaw trial underway for adult growth hormone deficiency to support our planned label expansion for transcon growth hormone.

Looking past before star trial.

In additional studies to support our effort to expand the label.

Okay.

<unk> deficiency.

Going to the last slide and this is going back to where I started.

Vision about the patient.

And this is why.

VX go to work. This is why we are living in our centers is the stores from the patients our interaction with patients not only in the era of <unk>.

Also in the era of.

<unk> and other diseases, we are working with and we find that.

Change.

Why are you can do for their patients one of the most important path of our journey.

Smbs.

I will move over to.

Okay.

The oil pipeline update.

Ill start first with transcon.

PTH.

Transcon, PTH, which has the potential to be the first <unk>.

<unk> replacement for the treatment.

It's the stories from the patient, our interaction with the patient. Not only in the era of glutamate, but also in the era of HIV and other diseases we are working with. And we find that... Change we can do for the patient is one of the most important parts of our journey as Ascendis. I will move over to The Oil Pipeline of Pays.

<unk>.

In the U S Europe, Japan, South Korea and China.

400000 patients just.

And these five regions.

And we are dedicated to get transcon PTH available to as many of these patients as fast as possible.

From our phase II study and now 58 week open label extension data.

We.

Competing exactly what we expected and hopeful.

All of the benefit of future logical PTH levels were realized including normalization of simulation serum phosphate urinary retention or turnover and quality of life.

I will start first with Transcon PTA. Transcom PTAs, which has the potential to be the first, Hormone Replacement, Proper Treatment, and Each. In the US, Europe, Japan, South Korea, and China, there are around 400,000 patients just in these five regions. And we are dedicated to getting Transcon PTAs available to as many of these patients as fast as possible. From our Phase 2 study and now the 58-week open-label extension days, we are seeing exactly what we expected and hoped.

Rehab Ashish this.

Promising results.

Essentially eliminating standard of care treatment for these patients.

The 58 week results demonstrate the durable response, and well tolerated safety profile that before Covid transcon PTH in adult <unk> patients.

What is really exciting is that 58 patients continue to remain in the open label extension.

I really believe this shows the positive impact Transco PTH is having on these patients short term sense.

We are hearing from patient and provider that patients.

<unk> side are improving that patients can now become active again, Ken will turn to the daily active.

Activities, including work.

With our phase III path forward data.

All the benefits of physiological PTA levels were realized, including normalization of serum calcium. Sarah Frostbeck, Euronary Cancer, Bone Turnover, and Quality of Life. We have achieved this promising result by essentially eliminating standard of care treatment in these patients. The 58-week results demonstrate the durable response and well-tolerated safety profile that we hope for with trans-contagious in adult age groups. What is really exciting is that 58 patients continue to remain in open label.

We have seen how adult HB patient benefit independent of background treatment and Kohl's.

The experience with transcon, PTH, mainly to better understanding of the unmet medical need.

Later this year in the fourth quarter, we expect to have 84 weeks topline.

Early data from path.

Cohort.

Our phase III trial.

82 subjects have been randomized and dose in North America and Europe.

The enrollment include leading influenza clinical site in both Geographics.

The demographics.

Similar to boost in our phase.

<unk> II trial, and introduce our board representation of non SEC H.

I really believe this shows the positive impact transcompetease is having on these patients' health. We are hearing from patients and providers that patients' lives are improving, that patients can now become active again, and can return to their daily activities, including work. Read our Phase 2 Part 4 to find out what they say. We have seen how adult HIV patients may benefit independently of background treatment and cause.

<unk> subject.

Thus as primary path.

I would characterize the dose syndrome.

<unk> syndrome and <unk>.

Okay.

We expect to announce topline results.

<unk> the phase III trial in the first quarter of 2022.

And if successful.

I expect to submit an NDA in mid 2022.

In Japan, we announced the acceptance of the clinical trial notification and the indication of the path.

Jeff pain trial, a single phase III trial of Transcon PTH in a minimum of 12.

The experience with transgruent PTA may lead to a better understanding of the unmet medical needs. Later this year, in the fourth quarter, we expect to have 84 weeks' top-line data, only data from past, and forward, in our Phase 3 trial. 82 adult subjects have been randomized and dosed in North America and Europe. Their enrollment includes leading influential clinical sites in both biographies.

Japanese subjects with HP.

Yes.

In greater China region has initiated the phase III profit China trial.

Of Transcon PTH in patients with HP.

The design of the Parkway, China trial mirrors, the design of the pathway trial.

As you can see we have taken a global clinical approach with transcon PTH deliver.

Really the data being generated is.

The demographics are similar to those in our Phase 2 trial and include a broad representation of non-surgeon ASB subjects, such as primary idiopathic hyperparathyroidism, Dior's syndrome, barotrauma syndrome, and ADH1. We expect to announce top-line results for the Phase 3 trial in the first quarter of 2021 and, if successful, we expect to submit an NDA in mid-2020.

<unk> by the assigned in the staffing Transcon PTH asset replacement therapy with the potential to be first line therapy for all forms of adult hyperparathyroidism.

Turning to Transcon CMP, we're trying to provide an update on our clinical program in the third.

Quarter of this year.

As you know.

Two randomized double blind placebo controlled phase II trials in children ages, two to 10 years.

Ongoing.

The first there.

<unk> trial is a dose escalation of 12 to 15 subject.

In Japan, we announced the acceptance of the Clinical Trial Notification and the initiation of the Partway Japan Trial, a single-arm Phase III trial of transcon PCH in a minimum of 12 adult Japanese subjects with AIDS. In Greater China, VISION has initiated the Phase 3 Partway China Trial of Transcon PHAs in patients with AIDS. The design of the Partway China trial mirrors the design of the Partway trial.

As Julie cohort conducted mainly in North America, Australia and Europe the.

The second one biodiesel pharmaceutical.

They accomplish China tower.

<unk> is a cohort expansion trial of at least 60 subjects conducted in China.

Rio planning to keep the data planned for <unk>.

One year for each call.

Once completed.

Are you going to have robust clinical data from two independent randomized double blinded placebo controlled trials.

Yes.

As you can see, we are taking a global clinical approach with transgone PTA. Givenly, the data being generated is driven by the science of establishing transgone PTAs as a replacement therapy with the potential to be first-line therapy for all forms of adult hypoparietal risk. Turning to Transcon CMP, we are trying to provide an update on our clinical program in the third quarter of this year. As you know, two randomized, double-blinded, placebo-controlled Phase II trials in children aged 2 to 10 years old are ongoing.

For achondroplasia patients our top priority is to develop and promote.

Commercialized highly different savings safe and effective treatment option that address the comorbidities of the disease and not just hype.

Switching to oncology.

We continue to make progress on both programs our transcon telos.

Eliminate equities is designed for intra will move to delivery.

Allowing for long term sustained Logan will lead.

For potential superior efficacy being minimal systemic adverse events.

Filing for <unk> last year was a may.

<unk> achievement for the company.

It was the first delivering the transcon hydrogen technology for sustained localized delivery.

Just a few comments on our hydro technology.

The first, the accomplished trial, is a dose escalation of 12 to 15 subjects in the East Cohort conducted mainly in North America, Australia, and Europe. The second, won by Decent Pharmaceuticals, is the country's China target, which is a cohort expensive trial of a least Safety Subject conducted in China. We are planning to keep the data blind for one year for each cohort once completed.

What we are developing at the same date.

Just the traditional <unk>.

Major tumor delivery based on injection and normal to continuous formulation into the tumor.

Sure.

Well the active compound will disappear in a few hours from the injection site.

The transcon intra tumor delivery technology at this time.

<unk>, an unmodified growth over weeks and months inside the tumor at the site of injection.

We will have robust clinical data from two independent, randomized, double-blinded placebo-controlled trials. For acondoplasia patients, our top priority is to develop and commercialize a highly differentiated, safe, and effective treatment option that addresses the co-morbidities of the disease and not just cancer. Switching to oncology.

This highly sustained local concentration with inventory is intended to kick start kick start the immune system without systemic toxicity.

Two is activating the immune system locally in the tumor and training them.

We aim not only to kill the injected tumor but to drive a systemic immune response.

We continue to make progress on both programs. Our Transcon TLR78 agonist is designed for inter-tumoral delivery, allowing for long-term sustained local release for Potential Superior Efficacy with Minimal Systemic Adverseity. Filing the R&D last year was a major achievement for the company. It was the first IND delivering the transcon hydrogen technology for sustained localized delivery. Here are just a few comments on our Hydro-T

That will also kill non indicative too.

This.

But potentially would be born to ashish with a true intra tumor delivery technology.

We have been dosing salt the transcon Telos seven eight agonist in the mono therapy.

And we recently initiated dose escalation in the combination with a checkpoint inhibitor.

We expect to have initial results kimono, Turkey dose escalation for Transcon Telos 708 equities in the fourth quarter.

For our second oncology program.

We are planning to submit an IND for transcon <unk> <unk> in the third quarter this year our understanding.

What we are developing at Ascendis is not just the traditional intratumoral delivery based on injecting a normal subcutaneous formulation into the tumor, where the active compound will disappear in a few hours from then. The Transcon inter-tumor delivery technology is designed to release an unmodified drug over weeks and months inside the tumor at the site of injection. This highly sustained local concentration in the tumor is intended to kick-start the immune system without systemic toxicity by activating the immune system locally in the tumor and draining lymph. We aim not only to kill the injected tumor but to drive a systemic immune response that will also kill non-injected tumors. This is the potential we want to achieve with a true inter-tumor delivery technology.

This is after biology has guided us in designing our compounds independently optimized receptor bias potency pharmacokinetic to create a potential best in class.

Carlo.

By solving the different elements related to efficacy and safety independently.

It will.

The same goes to realize the full potential of the IL two pathway.

To summarize.

We have now all first approved product schedule, but.

We are focused on delivering a successful loan cost control.

Women.

B project to improve patients' lives.

We have been dosing subjects with transcon TLR7-8 agonists in the monotherapy market, and we recently initiated dose escalation in the combination arm with a checkpoint inhibitor. We expect to have initial results for monotherapy dose escalation for transgon TLR78 agonists in the fourth quarter for our second oncology program. We are planning to submit an R&D application for Transcon IL-2 beta-gamma in the third quarter of this year. Our understanding of the biology has guided us in designing a compound with independently optimized receptor-biased potency pharmacokinetics to create a potential best-in-class IL-2 product. By solving the different elements related to efficacy and safety separately, we believe it will be possible to realize the full potential of the IL-2 path. Thank you for your time. All right.

We are up to 200000 patients.

Our vision has never changed and I'll repeat it again we.

We want to establish <unk> as a fully integrated leading global biopharmaceutical.

Scott Smith: We now have our first approved product, Skytrol. We are focused on delivering a successful lung for sclerotropia, and we remain committed to improving patients. We are confident in the way we are strategically thinking about our program, both clinically and commercially as we address large rare disease markets with our product opportunities. We estimate the global daily growth of the market to be around $ a billion, with up to 200,000 patients. In North America, Europe, and Japan, we believe that Transcon PTAs address a market opportunity greater than 5 billion US dollars.

Turning to our financial results for the quarter ended June 32021, we reported a net loss of $134.4 million euro or $2 five euro per basic and diluted share compared to a net loss of $94.9 million euro or $1.97 euro per basic and diluted share during the same period in 2000.

Yeah.

Let me now run through some key components of these results.

Research and development costs for the second quarter were $83.3 million euro compared to $63.6 million Euro during the same period in 2020, the increase in R&D costs reflect continued advancement of our pipeline with the primary drivers.

<unk>, including an overall increase in personnel related costs.

For transcon growth hormone or <unk> costs were higher due to buildup of prelaunch inventories investments to expand our future manufacturing capacity as well as increased clinical trial related activities.

Scott Smith: We are committed to bringing the market leader in each of these endocrinology valencies to markets, with the approval of Skype Open. We believe that it's a great validation of the Transcom technology and gives us hope that we can address major unmet medical needs with a high success rate. Our vision has never changed, and I will repeat it again and again: we want to establish Ascendis as a fully integrated leading global biopharmaceutical company with a portfolio of multiple independent, market-leading products within different therapeutic areas. Now, let me turn the call over to Scott for a financial review before we open it up to questions.

As a reminder.

We currently expense manufacturing activities at <unk> Summit <unk> is R&D costs in advance of approval at the time of product approval.

A portion of these R&D costs may be reversed in capitalized inventory as raw materials work in progress and finished goods.

Actual results and the one time benefit to R&D costs.

Plus as of today this would have been approximately $50 million euro.

For Transcon, PTH or Palo Peg Terra paradigm costs were higher primarily due to increased clinical trial related manufacturing and device development costs.

For Transcon CMP costs were higher primarily.

Due to increased manufacturing and clinical trial related costs.

And finally for our oncology therapeutic area costs were higher due to increased manufacturing and preclinical costs for transcon IL, two beta gamma and for Transcon Telos seven eight agonist higher clinical related costs were partially offset by lower manufacturing.

Turning to our financial results for the quarter ended June 30, 2021, we reported a net loss of €134.4 million, or €2.5 per basic and diluted share, compared to a net loss of €94.9 million, or €1.97 per basic and diluted share, during the same period in 2020. Let me now run through some key components of these results. Research and development costs for the second quarter were €83.3 million, compared to €63.6 million during the same period in 2020. The increase in R&D costs reflects continued advancement of our pipeline. Primary drivers, including an overall increase in personnel-related costs

Primarily by nickel costs.

Selling general and administrative expenses for the second quarter were $35.3 million euro compared to $20.8 million Euro during the same period in 2020.

These higher expenses, primarily reflect an increase in personnel related IP and other infrastructure costs as we prepare for the launch.

And pretty clear Sky Trofim.

Financial income and expenses in the second quarter included a foreign exchange rate loss of $11.3 million euro compared to a foreign exchange rate loss of $9.9 million Euro in the second quarter of 2020, primarily related to translation of our U S. Dollar holdings of cash and marketable securities to euros.

We ended the second quarter with cash cash equivalents and marketable securities totaling $641.3 million Euro.

As of June 32021, the company had 53.900 million 990 ordinary shares outstanding.

Turning to the remainder of 2020.

'twenty one.

We expect our reported operating expenses for the rest of the year to reflect the first half run rate with some quarter to quarter variability with key drivers, including the anticipated launch of Sky Trophy.

Advancing our endocrinology rare disease pipeline, expanding our activities in oncology and investing in the Transcon technology.

For Transcon Growth Hormone, or Lonopeg Somatropin, costs were higher due to the buildup of prelaunch inventories, investments to expand our future manufacturing capacity, as well as increased clinical trial-related activity. As a reminder, we currently expense manufacturing activities of Lonapex somatropum as R&D costs in advance of approval. At the time of product approval, a portion of these R&D costs may be reversed and capitalized to inventory as raw materials, work in progress, and finished goods, which will result in a one-time benefit to R&D costs.

Allergy platform.

Including <unk> Somatotrophin.

Buildup of commercial inventory ahead of launch.

Execution of commercial launch activities.

Investment in expanding commercial manufacturing capacity to support anticipated future demand.

Continued.

Execution of the foresight trial, a global phase III randomized controlled clinical trial in adults with ghd.

And continued execution of the right trial, a phase III randomized controlled clinical trial in children with Ghd in Japan.

For our pallet peg Terra paradigm.

<unk> execution of the phase.

Phase III path forward trial, which continues to retain 58 subjects in the open label extension.

As of today, this would have been approximately 50 million euros for Transcon PTH or Palopec Terra Peritoneum. However, costs were higher primarily due to increased clinical trial-related manufacturing and device development costs. For Transcon C&P, costs were higher primarily due to increased manufacturing and clinical trial related costs. And finally, for our oncology therapeutic area, costs were higher due to increased manufacturing and preclinical costs for transcon IL-2 beta-gamma, and for transcon TLR78 agonists, higher clinical related costs were partially offset by lower manufacturing and preclinical costs.

Along with continued execution of the pathway trial at North American and European Phase III randomized controlled clinical trial in adult hyperparathyroidism.

And ongoing manufacturing of PDQ batches.

For Transcon CMP.

Execution of the clinical program, which includes two randomized controlled phase II clinical trials in a contemplation the ongoing accomplish trial and they accomplish China trial, which is being conducted through our strategic investment in visa <unk> pharmaceuticals.

And lastly in our oncology therapeutic.

<expletive> area execution of the transcend <unk> 101 clinical trial for our Transcon Tls seven eight agonist.

And advancing the transcon IL two beta gamma program into clinical development.

We expect other SG&A expenses, including Sky trophic commercial activities will include power.

Pellet peg prelaunch activities and continued investments in personnel systems and infrastructure to support our rapidly progressing portfolio and growing organization.

Yeah.

Let me now provide an update on our upcoming clinical milestones.

So a lot of peg somatropin today, we have received FDA approval for Sky.

<unk>.

For the treatment of pediatric ghd in the United States.

Selling general and administrative expenses for the second quarter were 35.3 million euros compared to 20.8 million euros during the same period in 2020. These higher expenses primarily reflect an increase in personnel-related IT and other infrastructure costs as we prepare for the launch of SkyTrofa. Financial income and expenses in the second quarter included a foreign exchange rate loss of 11.3 million euros compared to a foreign exchange rate loss of 9.9 million euros in the second quarter of 2020, primarily related to translation of our US dollar holdings of cash and marketable securities to euros. We ended the second quarter with cash, cash equivalents, and marketable securities totaling 641.3 million euros. As of June 30, 2021, the company had 53,900,990 ordinary shares outstanding. Turning to the remainder of 2021,

We continue to anticipate European Commission approval for pediatric ghd in the fourth quarter of this year.

For <unk> Terra paratype as discussed we exceeded the target enrollment in the pathway trial and now expect to report top line.

Line results in the first quarter of 2020.

For Transcon <unk>, we expect to provide a clinical program update in the fourth quarter. This year.

For Transcon Telos seven eight agonist, we have initiated the dose escalation arm in combination with the checkpoint inhibitor and we plan to report initial monotherapy dose escalation.

<unk> data for <unk> 101 in the fourth quarter this year.

And finally for Transcon IL, two beta gamma we plan to submit an IND later this quarter.

Before we open up the call for questions I want to reiterate a few points about our anticipated commercial activities for sky to growth in 2021.

With the FDA approval of Sky drove it today, we expect to have product available. Shortly once the product is available we anticipate beginning to provide access to sky Trophy for pediatric ghd patients through our dedicated patient support program.

Our medical affairs, our U S commercial team and the entire Ifs in this organization.

Our currently ready and prepared to execute launch activities.

And again finally during Q4, we anticipate European Commission marketing approval for pediatric Ghd.

With that operator, we are now ready to take questions.

Alright.

A reminder to ask a question you will need to press star one on your telephone.

All your question press the pound key again that is star one on your telephone please stand by while we compile the Q&A last name.

First question comes from the line of Jessica Fye from Jpmorgan. Your line is now open.

Hey, guys. Good afternoon, congrats on the approval and thanks for taking our questions.

We expect our reported operating expenses for the rest of the year to reflect the first half run rate, with some quarter-to-quarter variability, with key drivers including the anticipated launch of SkyTropha, advancing our endocrinology rare disease pipeline, expanding our activities in oncology, and investing in the TransCon technology platform, including Ferlanopex somatropins, and the buildup of commercial inventory ahead of launch. Execution of Commercial Launch Activities, Investment and Expanding Commercial Manufacturing Capacity to Support Anticipated Future Demand, Continued Execution of the Foresight Trial, a Global Phase III Randomized Controlled Clinical Trial in Adults with GHD, and continued execution of the RITE trial, a Phase III randomized controlled clinical trial in children with GHD in Japan, for Palapeg Terra Peritide.

A couple for me first can you please explain which part of the data investors should look to in the label that reflects the superiority of Sketchup has efficacy over <unk> is at the lower bound of the 95% confidence interval.

We'll in table four of the clinical study section.

Exactly.

Okay.

How does this timing of approval impact your payer conversations if at all is it possible you can catch the late summer window for coverage of discussions and what's the list price per milligram going to be.

Let me start.

No.

Your first question gifts and.

There's different ways you can describe how you ashish in Ohio.

Annualized growth velocity.

From my perspective is the best one is sort.

So the competition into that because it basically describe what it is that each of them, where you are improved with a 95% confident.

Continued execution of the Phase 2 Path Forward Trial, which continues to retain 58 subjects in the Open Label Extension, along with continued execution of the PATHWAY trial, a North American and European phase 3 randomized controlled clinical trial in adult hyperparathyroidism, and ongoing Manufacturing of PPQ Batches for Transcon C&P. Execution of the clinical program, which includes two randomized controlled phase two clinical trials in achondroplasia, the ongoing ACCOMPLISH trial and the ACCOMPLISH China trial, which is being conducted through our strategic investment in Vizen Pharmaceuticals.

So when I see what.

What paragraph or hurt foot paragraph.

Somebody a funding.

Finding too I will.

Keep your 100% right. This is where I see and well I guess my comfort that you Ashish, Ohio.

<unk> high.

Velocity in the 95% confidence interval.

Yes.

That was one Christian.

About the payer system are you sure that is something.

We start working on but it's something we have worked for Enbrel lost too.

Yes, wherever you basically have intense.

Discussion bid payer, mainly reflecting our discussion on how we basically.

And lastly, in our Oncology Therapeutic Area, execution of the Transcend IT-101 clinical trial for our Transcon TLR78 agonist and advancing the Transcon IL-2 Beta Gamma Program into Clinical Development. We expect other SG&A expenses, including SkyTropha commercial activities, will include Palopeg pre-launch activities and continued investments in personnel, systems, and infrastructure to support a rapidly progressing portfolio and growing organization. Let me now provide an update on our upcoming clinical milestones for Philanopec Somatropin.

It's providing a pipeline of product opportunity and building up a long term relationship.

Ship with the payer system not only in the U S. But also how we want to address the compensation and the rest of the world.

Related to the U S.

Yes, so you potentially will have a short comments about it even if you did limit what we can say now and what we can say from a public statement because that is.

Something question that basically can materialize now they're prudent.

Yeah basically yes.

And then just yet.

I would say that of course things has been as generous as we thought about this a long time ago, we had a super good team in place.

Today, we have received FDA approval for Skytropha for the treatment of pediatric GHD in the United States. We continue to anticipate European Commission approval for pediatric GHD in the fourth quarter of this year. For Palomeque Terra Peritide, as discussed, we exceeded the target enrollment in the pathway trial and now expect to report top-line results in the first quarter of 2020. For Transcon C&P, we expect to provide a clinical program update in the fourth quarter of this year.

And we are totally prepared.

<unk>, what we are entering into and we all know for the long run.

So it's not just about getting access is also getting access under the right circumstances and that certainly what we are.

Working on SPN speaking almost at.

At least in the <unk>.

In the days and weeks to come so.

Truly welcome where we are right.

Sure.

Great. Thank you.

If I could ask it a little bit to you about it as you know we have a commercial strategy.

And the commercial status.

For Transcon TLR78 Agonist, we have initiated the dose escalation arm in combination with the checkpoint inhibitor, and we plan to report initial monotherapy dose escalation data for Transcend IT 101 in the fourth quarter of this year. And finally, for TransCon IL-2 Beta Gamma, we plan to submit an IND later this quarter.

Basic to be in a position.

Right now we basic outlook looking on maximizing long term value of our best in class growth hormone.

But also at the same time as yes, let's say laying the foundation for future product launches and the endocrinology Vips.

So this is why we are building a leading.

Leading market share base.

Based on all the value proposition, but not only for.

Before we open up the call for questions, I want to reiterate a few points about our anticipated commercial activities for SkyTrofa in 2021. With the FDA approval of Skytropha today, we expect to have the product available shortly. Once the product is available, we anticipate beginning to provide access to Skytropha for pediatric GHD patients through our dedicated patient support program. Our medical affairs, our U.S. commercial team, and the entire Ascendis organization are currently ready and prepared to execute launch activities. And again, finally, during Q4, we anticipate European Commission marketing approval for pediatric GHD. With that, Operator, we are now ready to take questions.

One single element, but both of the patients caregivers healthcare provider and.

And this is why the basic up developing a strategy for that because we want to be a leading biopharma company.

We are building our strategy on maximizing long term value of our best in class Guatemala. So this is our focus and this is one and you talked about pricing, yes, we call. It premium premium responsible pricing and then you say what is premium responsible pricing.

This is because we look on health care economic outcome research because this is where we're coming from from the Europeans that exist that this.

This is why we look at it.

And we see it we see that is a basic fundamental good reason basic two half.

Responsible pricing and everyone.

Operator: Alright, so as a reminder, to ask a question, you will need to press star 1 on your telephone. To resolve your question, press the pound key. Again, that is star 1 on your telephone. Please stand by while we compile the Q&A roster. The first question comes from the line of Jessica Fye from JP Morgan. Your line is now open.

All stakeholders patients cookie was depreciation including periods and this is what we have proven farmer economic data and that is why we are some way.

Can you just say we are going from a premium with.

Responsible pricing.

Just to answer your last part of the question.

Next one on the queue is <unk> Ahmad from Bank of America. Your line is now open.

Jessica Macomber Fye: Hey guys, good afternoon, congrats on the approval, and thanks for taking our questions. A couple for me first: can you please explain which part of the data investors should look at in

Good afternoon, and congratulations from me as well on the approval.

It's hard work.

Yeah can I just clarify one point as it relates to pricing is it the plan to announce pricing before you launch or would we would we know the price.

should look for in the label that reflects the superiority of Skytropa's efficacy over

over Genotropin. Is it the lower bound of the 95% confidence interval in Table 4 of the clinical studies section? Okay. How does this timing of approval impact your payer conversations, if at all? Is it possible you can catch the late summer window for coverage of the sessions? And what's the list price per milligram going to be?

Let's say on the day that the actual product becomes available.

And then GAAP.

Catherine.

I'm, hoping you can walk me through how a patient will initially be able to get access.

To the drug before it ends up on formulary I think there is a process.

The doctors will have to go through have you sorted what that process is and can you share those steps.

From my perspective, the best one is to look for the confidence interval, because it basically describes what the interval where you are improved with 95% confidence. So when I see or hear what paragraph you are in some way pointing to, I will give you 100% right. This is where I see and where I get my confidence that you achieve a higher analyzed high velocity in the 95% confidence interval.

Thank you.

Yes, absolutely.

So the first part I can just shorter, saying and it's more or less protecting in what we called premium responsible pricing.

We have a clear view wherever you want to be in pricing and this is a big part of our current because negotiation and we will come out shortly.

I.

A discussion about what vivek price liquidity and <unk> from yes, both potential you can refer back to that so we have the basic going to some of the steps we have the basic of talking about our system is exiting what.

There was one question about the pair system. Are you sure that is something we should start working on, but it's something we have worked for in the last two? Yes, where we basically had an intense discussion with Payer, mainly reflecting our discussion on how WeBasic, as Ascendis, is providing a pipeline, a product opportunity, and building up a long-term relationship with the Payer system, not only in the U.S., but also how we want to address commercialization and the rest of the world.

What I would call it a AEP.

I'm, assuming it's again.

This program and its basic in the slide number 15.

Yes I.

I mean again finds out.

It is.

Basically our signature program from Smbs plus that drove that.

Related to the US, I think Jesper, you potentially have a short comment about it, even if we limit what we can say now and what we can say from a public statement because that is a discussion that basically can materialize now with approval.

And.

What's going to happen in agriculture is.

I mean prices many things as we all know.

WAC price and net price.

But we will do is basically if you if you're going to sell it through the you could say that traditional without being on formula.

Yeah, basically Jan and Jessica.

A long time ago, we had a really good team.

And we are totally prepared for what we are entering into, and we all know we are in it for the long run. So it's not just about getting access. It's also getting access under the right circumstances. And that's certainly what we are working on as we go.

<unk>, which.

Which is what I heard you ask for them than.

And then you will be paying WAC price and then their decision will have to request a prior off.

And <unk> means that it will go through the health plan then it will be evaluated and then often break and then come back and then their decision will have to.

And in the days and weeks to come, so truly welcome where we are right now.

<unk> quantify why.

Truly, we are very welcome where we are right now.

Why this patient.

If I could connect a little bit to you, Jess, about it is that we have a commercial strategy. And the commercial strategy is fundamental to be in a position where we basically are looking to maximize the long-term value of our best-in-class growth model. But also, at the same time, as Jesper said, laying the foundation for future product launches in the endocrinology world. So this is why we're building a leading market share based on overall value propositions, but not only for One single element, but both of the patient, caregivers, and healthcare provider.

Best suitable for once weekly.

Don't want a path.

This guidance okay.

<unk>.

And that will have them under certain circumstances, it's not happen very frequently in comparison. If you are not on this formula.

That's why we will start out and then of course as we go by.

We will negotiate ourself into the contracting and into the Pbms.

And get access by doing that so all in all we will come to the market with an approach of being price sensitive being.

Being priced pregnant.

And this is why we basically are developing a strategy for that, because we want to be a leading biopharmaceutical company. And this is why we're building our strategy on maximizing the long-term value of our best-in-class growth model. So this is our focus, and this is just one example.

I think we can also say probably will also do it in a way.

Where it makes sense for us to make sure that we get optimized.

Profitability, you would say at the end of the day, that's what the job is all about from all sides.

So that's what we will be sharing pool.

Okay.

Yeah. Thanks can I just ask a follow up so from the time at the.

And you talked about pricing. Yes, we call it premium-responsible pricing. And then you say, what is premium-responsible pricing? This is because we look at healthcare economic outcome research. Because this is where we're coming from, from the European setting, is that this is where we see it. We see that it is a basic, fundamental good reason to have a premium, responsible pricing, and everyone will build. All stakeholders, patient, caregivers, physician, including payers.

Doctor writes a script do you have any sense of how long it'll take before the patient could actually receive.

And so if you are launching in fourth quarter, you know should.

Should we really expect to see patients being put on drug in large numbers because of the dynamics that you just.

In the early part of the launch.

It's it can take.

From two weeks to six weeks to eight weeks before patients get put on but what we will do in the meantime, with all SaaS stock program is basically Asap program.

The nickname fast start program spend.

And this is what we have proven in our pharmacoeconomic data. And that is why, in some ways, we are clearly saying we are going from a premium responsible price, just to answer your last part of the question.

The other companies are doing it of course, we have call it the incentives signature program.

There, we will provide the drug upfront and the auto injector to the patients and then as the patients get the approval for the drug they will be client transfer into the health plans and thereby getting access and being.

Tazeen Ahmad: Next one on the queue is Tazeen Ahmad from Bank of America. Your line is now open.

Good afternoon, and congratulations to me as well on the approval. Lots of years of hard work.

Painful so you will not see any fast uptake.

Yan, can I just clarify one point as it relates to pricing? Is it the plan to announce pricing before you launch, or would we know the price, let's say, on the day that the actual product becomes available? to the drug before it ends up on the formulary. I think there is a process that doctors will have to go through. Have you figured out what that process is, and can you share those steps with us?

Yeah.

But you certainly will.

Yeah.

Got something in the wrong, you will see that we.

Move forward and as things progress.

So.

We do believe.

That the Asap program incentive program will certainly put us in a very strong position for the time to come to drive our market leadership again remember that we are first in the pediatric segment are coming to the market in that first mover advantage. We will certainly also.

The first part I can just briefly say, and it's more or less reflecting what we call premium responsible pricing. We have a clear view of where we want to be in pricing, and this is a basic part of our current negotiation, and we will come out shortly with a discussion about what the back price is with it. And I think in the slide deck from Jesper, potentially, you can refer back to that Jesper, where we basically are going to some of the steps where we basically are talking about our system. I actually think it was what I call, we call it ASAP, Ascendis Signature Assess Program, and it's basically in slide deck number 15.

Enjoy in the in the time to come.

Okay. Thank you so much for calling.

Thanks, a lot for the question.

Next one on the line is Michelle Gilson from Canaccord Genuity. Your line is now open.

Hi, congratulations.

I'm also very.

Happy Michelle.

Thank you Hugo.

Uh huh.

Yeah, I noticed the label it looks very very similar to daily growth hormone I guess just with this in mind you do you think physicians will see sky Troper interchangeable with daily growth hormone.

In the way that the the labels Ryan do you anticipate there will be any pull through demand beyond the initial indication into the other growth hormone approved indications.

I mean, as Jan points out, ASAP is basically our signature program from Ascendis for SkyTrooper. And what's going to happen in actual terms is, I mean, price is many things, as we all know. It's wag price, and it's net price.

I believe when I look in the labeling.

When we talk.

With physician.

When we discuss the entire concept of.

Skytrooper.

The actual terms are, I mean, price is many things, as we all know. It's wag price, and it's net price.

And I think as you said to Michelle Davies exits illustrated in the labeling.

You've released.

The physician will then have to request prior authorization.

Similar to molecule the same SD drove tumor.

A prior authorization means that it will go to the health plan, then it will be evaluated, and then

I believe all of our spec from the what we see from the signs reflected into the late <unk>.

Requalify why this patient is best suitable for the once weekly donor pack Sky Trooper

Indicate that we basically have the same active entity SD Cove tunnel.

And also believe all the clinical data we have generated.

And that will happen under certain circumstances. But it's not happening very frequently in comparison if you're not.

Support.

Supporting the same thing.

When we look from switching.

So that's where we will start out, and then, of course, as we go by, we will negotiate our solutions.

From daily growth hormone patient over to skytrooper or will look for starting.

And then, of course, as we go by, we will

Patients in <unk> and <unk>.

That the comfort.

We have in our data we can also communicate to.

So, all in all, we will come to...

Two publication Medical affairs, and really illustrate the benefit of the.

We will come to the market with an approach of being price sensitive, being price premium. I think we can also say, but we'll also do it in a way that makes sense for us to make sure that we get optimized.

Compound currently.

Half approval in pediatric growth hormone deficiency.

We are extensive.

Taking on label expansion as you know we have indicated.

At an adult growth hormone deficiency trial.

Profitability, which at the end of the day, is what the job is all about.

Potential.

Perceive one or two other indications to your knowledge.

That's what the job is all about from our side. So that's what we will be shooting.

When we see the co promote.

None of the daily growth hormone having basic.

The approval of all.

All the different indications.

Yeah, thanks. Can I just ask a follow-up question? So from the time a doctor writes a script, do you have any sense of how long it'll take before the patient could actually receive the treatment? So if you are launching in the fourth quarter, you know, should we really expect to see patients be put on drugs in large numbers because of the dynamics that you describe?

To date so it's.

Element.

Where we are building up the scientific clinical evidence.

The compound.

And if I can also add a follow up.

How do you plan to communicate guidance profile.

The differentiation from other long acting growth hormones.

They are approved in the future.

I know you mentioned immunogenic profile in your prepared remarks.

It can take from two weeks... to Six Weeks to Eight Weeks Before Patients Get...

Yes.

What I mentioned was that we had a similar image.

In Virginia.

The nickname Fast Start Programs when other companies are doing it. Of course, we call it the Ascendis Signature Program.

Readout that youll see with data growth hormone with.

Really really.

Safe treatment.

I actually believe in data.

And I actually think when people start to compare different phase III trials.

that we will provide.

Provide the drug up front and the autoinjector to the patient.

And then.

I think the differentiation.

And then, as patients get approval for the drug, they will...

Clear.

Michelle I think it comes back to the motive action in the in the beginning because your basic.

Transcribed by https://otter.ai I've got something in the wrong, but you will see it as we move forward and as things progress.

If you don't have the right molecule you don't have the right distribution to all of the tissues you would never somebody can achieve.

We do believe that the ASAP program, the Ascendis program,

See the same thing that you will ashish with a daily so so much of a molecule.

and it will certainly put us in a very strong position for the time being.

And exiting this is why after 30 years still all patients in Europe.

on www.davidlebowitz.com

Okay, thank you so much for the follow-up.

U S.

Japan some of the major markets are still being treated with basic Ses.

Michelle Gilson: Next one on the line is Michelle Gilson from Canaccord Gen. Utah. Your line is now open.

Let's say the old daily growth hormone that got invented in the eighties.

Okay. Thank you so much for taking my questions and congratulations again.

Hi, congratulations. I'm also a very happy Michelle.

And we have is that you have.

[laughter].

I noticed the label. It looks very, very similar to daily growth hormone. I guess just with this in mind, do you think physicians will see Skytropha as interchangeable with daily growth hormone in the way that the label is written? And do you anticipate there will be any pull-through demand beyond the initial indication into the other growth hormones approved?

Alright next one on NICU is Josh <unk> from Evercore ISI. Your line is now open.

Hey, Thanks for taking the questions. So sticking on schedule for them. Maybe you can talk about how long do you think it'll take to gain widespread formulary adoption is that likely to be the key gating step them for.

when we talk with physicians, when we discuss the entire concept of Scott Fulper, And I think, as you said, Michel, that is actually illustrated in the labeling where we release it as a somatopene molecule, the same as davycopene. I believe all the aspects of what we see from the science reflected in the labeling indicate that we basically have the same active entity as daily COVID and also believe all the clinical data we have generated are supporting the same thing when we look at switching from a Daily Growth Hormone patient over to Sky Cobra, or we look at starting a youth patient on that.

Rob tank or is it likely to be physician hesitancy and then it looks like consensus for Sky Trophy for next year is spent $110 million in sales side and the general Barbara ballpark of what you think is reasonable considering some of the lunch I had been feel youll have to push there. Thanks so much.

Thanks, Jeff.

For Covid.

<unk> for your questions.

If I can just start with the last question, we have not provided any kind of guidance to our expected sales fall 'twenty 'twenty two.

We feel.

Sure.

Strategy is really to.

The long term value about the product opportunity.

And addressing liberate your first questions I believe when you.

And I feel that the confidence we have in our data, we can also communicate to publication, medical affairs, and really illustrate the benefit of the research. Currently, we have approval for Pediatric Growth Hormone Deficiency. We are extensive, working on label expansion. As you know, we have initiated an adult protomome deficiency trial. We will potentially receive one or two other indications. To your knowledge, when we look at the COVID-19 market, none of the daily COVID-19 tablets have basic approval of all the different indications today. So it's an element where we are building up scientific and clinical evidence on the compounds.

Think about the view from our physicians.

The physician.

Can see and know.

If I have a one we detail.

Great.

That basic all in a position.

We know that only 20% less than 20%.

The patient to date in this market segment, when we look at the U S basically.

Meaning is that they take our pickup 80% off.

The prescription.

So from that perspective.

Note and the physician note that can get a better outcome with giving a once weekly.

Product.

Go ahead till the patient that is sitting with the khaki and front.

And having to take a choice you can take a daily growth hormone.

<unk> injection or you can potentially go down to five injection that week with many many many do.

And then you would get worldcom.

Or you can take a once weekly unlikely the daily needs to be in the cool shade.

Time, so basic the patient comfort.

And if I can also add a follow-up question, how do you plan to communicate Skytropa's differentiation from other long-acting hormones if they are approved in the future? I know you mentioned the immunogenic profile in your prepared remarks.

What do we have one if the child is traveling going to the grandparents that need to be sure. There is a cool back all the time and other things like that.

So this I believe when all the notice from the patient and other things there.

What I mentioned was that we had a similar hemogenic readout that you see with data growth hormone, which is really, really a safe treatment. I actually believe in data. And I like to think that when people start to compare different phase 3 trials, then I think the differentiations are really clear. But, Michel, I think it comes back to the mode of action in the beginning, because basically, if you don't have the right molecule, you don't have the right distribution to all the tissues, you will never, in some way, achieve the same thing that you will achieve with a daily somatopene molecule.

<unk> with its asset market choice if.

If you can provide the same.

The efficacy and we believe we can do that and have the same safety and tolerability.

Going to your question about the reimbursement and I think if you have the efficacy right.

All of the safety right and the ability.

Then you come back to.

How fast can we go into the different reimbursement system.

And this is where the building what I call.

Premium responsible pricing because we believe they deserve.

Premium pricing.

But we also want to build on what we really call the optimum.

Sandy's building up a company of long term value.

And they like to think this is the way after 30 years. Still, all patients in Europe, the US, Japan, some of the major markets are still being treated with basic, as Jesper said, the old daily growth hormone that was invented in the 80s.

Each of these product opportunities. So we are not in a volume game.

And that is how we build up our commercial strategy.

Thanks very much.

Yes, if I can just add.

To date.

Day, one and I can tell you we have done a lot of preparedness for this we have worked on this for the last couple of years to get ready for this moment and.

Okay, thank you so much for taking my questions, and congratulations again.

Joshua Elliott Schimmer: Alright, next one on the queue is Josh Schimmer from Evercore ISI. Your line is now open. Thanks for taking the questions. So, sticking on Skytrofa, maybe we can talk about how long you think it'll take for widespread adoption.

All the things.

And place for doing a very successful launch of become the company.

We want to be going forward not only with <unk>.

Nitro, but certainly also to build the platform for our future and again was also talking about in his introduction.

Okay.

Sure.

Unknown Speaker: Is that likely to be the key gating step for uptake, or is it likely to

Thanks, guys.

Yeah.

Next question comes from the line.

Line of Alicia Young from Cantor. Your line is now open.

Hey, guys. Thanks for taking my questions and congrats on the approval.

Transcripts provided by Transcription Outsourcing, LLC.

Wonderful to watch.

I guess, a couple and I actually I'm going to ask one that's not related to sky trial path, but the first one Scott.

Can you just talk a little bit about how.

How to think about the Medicare and Medicaid mix the government.

While mix, if there's any versus like commercial.

Thanks, Jess, for your questions. If I can just start with your last question, we have not provided any kind of guidance on our expected sales for 2022.

My second question is you know.

That $2.8 billion rest of world can you come in either break that out by patient numbers or how to think about it either on a revenue basis.

Europe versus like.

Asia and then my last question is just going back.

<unk> are 708 program.

You know should we expect that youre going to be at doses.

At the end of the year that might show some efficacy and then how do we kind of think about what youre kind of expecting for the combination study that probably will have some data hopefully next year. Thanks.

Thanks, a lot.

It looks like some nice pick up.

Strategy is really to create long-term value about the product opportunity and address a little bit your first questions. I believe when you think about the view from a physician. The physician can see and know if I have a once-weekly tarot card that basic are in a position. We know that only less than 20% of the patients today in this market segment, when we look at the US, are basically compliant, meaning that they pick up 80% of the prescriptions. So, all from that perspective.

Government outside.

Hello.

Part of it.

Let us move back to some of your view of growth hormone Alright, Christian let me take the question with the breakdown, reflecting the go to market and.

It's the single largest market and but then be basic have too.

You can say Asian market that basic are competing or being number two or three.

Currently Japan is the second largest single bucket.

China is basic basic.

Basically we expect that it will pass.

Japan this year moving up to more than eight.

Christian.

But really the major major advancement.

Then you have Europe does just little bit under this market, Japan is very interesting because it also build on a little bit of local producers because it's been easy for local producers into the market because that has been new innovation.

We know, and the physicians know, that they can get a better outcome with giving a once-a-weekly product. But go to the patient that is sitting with the caregiver in front of them and having to make a choice. You can take a daily protocol of 7 injections, or you can potentially go down to 5 injections a week, with many, many, many people do. And then you get 2 out, or you can take a one-week... And likely, the daily need to be in a cool shade all the time.

For the past 30 years, and this is where our strategy come in we have now.

Commercial strategy, Yes, we'll have we have our recent pharmaceutical greater China, we now running the clinical development from Japan, meaning as we basically have a global team.

<unk> hundred <unk> when we have that doing that we also will have our global commercial strategy building on one single thing pipeline deals not single product deal few places, where we don't want to do it ourselves we are going to a place where the basic have the infrastructure to support.

So basic, patient comfort. What do you want if the child is traveling, going to the grandparents? They need to be sure there is a cool bag all the time and other things. So this, I believe, when all our knowledge from the patient and other things, the caregivers, is such an awesome choice if you can provide the same.,,,,,, Going to your question about reimbursement, I think if you have the efficacy right, the safety right, and the turbility, then you come back to, How far can we go into the different reimbursements? And this is where we're building our water pool.

To build it up as a leading brand also in this area, but we still will be part of what we call the value creation and there will still be run on a global umbrella both related to marketing measures medical affair, because BP lead in global market, We don't think lead and broken market more.

This is a global society and this is how we are building up our global strategy and this is wherever you have a global aspect everything we'll be doing.

And related to the specific attrition related to commercial life of commercial patients on our commercial patients I think we have a clear view of where we want to boot.

More you can say shortly well focus would be.

Premier Responsive Pricing because we believe we deserve it, and Premier Pricing. But we also want to build on what we really call the optimal view from Ascendis, building up a company of long-term value for each of these products. So we are not in a volume game; we are in a value game. And that is how we build our commercial strategy.

The last question.

Absolutely I mean key key focus is of course in the commercial space again keep in mind that this is pediatric CMS is not really.

Playing that Medicare mitigate space.

I think Tim So our focus is in there.

On the 185 million patients that are.

It can be found in the commercial space of Peaky focus there and just to add onto what the yen is saying keep in mind that if you hit <unk>.

Yeah, now if I can just say, at today's...

day one. And I can tell you we have done a lot of preparation for this. We have worked for this for the last couple of years to get ready for this moment.

China in the top seven markets in the world.

Meaning.

So in the U S and Europe side, we're hitting more than 7% to 5% of the world's pharma market and that's of course, where our focus is going to be going forward.

[inaudible] All the things are in place for us to have a very successful launch and become the company we want to be going forward, not only with SkyTrooper but certainly also to build the platform for our future that Jan was also talking about in his introduction.

And related to oncology, what we have said and what the basic of speaking to.

End of the year, we've been keeping an update.

One thing I can say, we're really thrilled with this product opportunity for first time, those what I call true inter tumor delivery technology and.

Alita Young: The next question comes from the line of Alita Young from Canter. Your line is now open.

Thanks a lot. It was actually nice to get a question outside. Let us move back to some of your growth hormone questions. Let me take the question in the breakdown reflecting the growth hormone market. The US is the single largest market, but then we basically have two Asian markets that are basically competing to be number two or three. Currently, Japan is the second largest single market. China is basic in basic; we expect that it will pass.

I'd be thrilled with that aspect.

Alright next one on the queue is David Lebowitz.

Morgan Stanley Your line is now open.

Hi, This is avatar Jones on for David Today, a couple of more questions for us on the access side.

Firstly what percent of major payers have already been engaged.

Secondly, can you quantify what premiums.

From hospital pricing looks like.

And finally, how would you see a step therapy requirement through a cheaper alternative impacting adoption.

Japan, this year, moving up to more than 800 million as a single, but really has major, major advancement every year. Then you have Europe that's just a little bit under this market. Japan is very interesting because it was built on a little bit of local producers because it's been easy for local producers to enter the market because there has been no innovation for 30 years.

Thank you.

First thing I had.

Yes.

Really provided some insight we can provide and are seeing is half.

Tough for us to provide more because we are not disclosing our long strategy. How we are moving forward. We are disclosing exactly the overall picture of the long strategy, how we basic our position of that product opportunity what has been our strategy what we have done in <unk>.

And this is where our strategy comes in. We now have a U.S. commercial strategy, yes for has that. We have our recent pharmaceutical company, Greater China. We are now running the clinical development from Japan, meaning we basically have a global clinical reach when we have that. By doing that, we will also have a global commercial strategy built on one single thing, pipeline deals, not single product deals, few places where we don't want to do it ourselves.

We have done.

Done that in a pretty detailed manner premium pricing is what we discussed before and we call. It premium responsible pricing because we believe that our product is a premier pricing and because we believe it provides and pharmacology.

We will go into a place where the basics have the infrastructure to support us to build it up as a leading brand also in this area, but we will still be part of what we call value creation. And it will still be run under a global umbrella, both related to marketing measures and medical affairs, because we believe in the global market; we don't believe in the local market more. This is a global society, and this is how we're building our global strategy.

And Amit benefit for everyone from the patient for the physician for all the stakeholders, including.

Here system. So the last question I do not know yesterday, if you have a few comments to that.

No I think I mean.

Because pricing of course very close to hot negotiations is ongoing with a different pbms and health plans.

I really don't have anything much color to add I think strategy is in place and we're moving forward and I can only say I'll reiterate it forward too.

And this is where we have a global aspect for everything we're doing here. Related to the specific question related to commercial life or commercial patients or not commercial patients, I think we have a clear view of where we want to be. Jesper, you can clearly say shortly where our focus will be, and I can move back to the last question. Absolutely. I mean, the key focus is, of course, in the commercial space. Again, keep in mind that this is for children.

To make the announcements as they become public.

Information and negotiations also bear in mind that when you talk with Pbms.

A tight spot right now.

Due to the way that the system is set up where basically announcements are coming out in August barrier to September.

The Pbms for next year and.

Nick.

We are working hard on dania.

Thanks, a lot Jeff.

Next question comes from the line of <unk> Xu from Wells Fargo Securities. Please state with yourself to one question. Your line is now open.

https://www.youtube.com

Hi, Thanks for taking my question and congrats.

on the $185 million

And the approval.

I'll just limit myself to one question on the adoption.

[inaudible]

And just to add to what Yan is saying, keep in mind that if you hit China in the top seven markets in the world, meaning Japan, the US, and Europe, we're hitting more than 75% of the world pharma market. And that's, of course, where our

Sky profile.

It's our understanding that.

In a real world setting physicians, often prescribe daily growth hormone above.

The labeled.

Europe, two four <unk> per kg per week.

So for those physicians.

So I guess first of all how prevalent is that and for those physicians.

That's, of course, where our focus is going to be going forward.

How do you think they interpret the label and then press.

Related to oncology, what we have said and what we basically are speaking to, at the end of the year, we will give an update. One thing I can say is that I'm really thrilled with this product opportunity. For the first time, there is what I call a true inter-tumor delivery, and V12 has that aspect.

Two.

Sky profile and do you need additional studies at higher dose or including a higher dose in your open label study, that's how I'm going to come this those physicians for a quick turn adoption. Thank you.

So what is in the labeling.

Use dosing.

David Lebowitz: Alright, next one on the queue is David Lebowitz from Morgan Stanley. Your line is now open. Hi, this is Avatar Jones on behalf of David today. A couple more questions for us on access.

Dosing of 424.

Maybe Brian per kilo per week.

And it's basic is reflecting what we did in both net new patients and what we did in our suite.

A couple more questions for us on the access side. Firstly, what percent of major payers have already been engaged? Secondly, can you quantify what premium responsible pricing looks like? And finally, how would you see a step therapy requirement through a cheaper alternative impact adoption?

Trial, where we took patients from different.

Daily growth hormone concentration and all of them got switched over initial two <unk> two full unit train.

What we observed in this trial was also that Chris.

The first thing I think we and Jesper have really provided so much insight we can provide, and I think it's hard for us to provide more because we are not disclosing our long-term strategy, how we are moving forward. We are disclosing exactly the overall picture of the long strategy, how we based our position on the product opportunity, what has been our strategy, what we have done, and I think we have done that in a pretty detailed manner.

Precision.

Basic titrated different pay.

Patient.

Levels.

Pendant on clinical outcome and although important pyramid.

So for the patients so.

What is important in this case is that youll have a compound that can be titrated in.

Default a product because that is very important for the precision that you can try it traded in a manner that is.

Premium pricing is what we discussed before, and we call it premium responsible pricing because we believe that our product deserves premium pricing and because we believe it provides a pharmacologic economic benefit for everyone, from the patient, to the physician, to all the stakeholders, including the payer system. So the last question, I do not know, Jesper, if you have a few comments on that. No, I think I mean, for pricing, of course, very close to heart negotiations are ongoing between different PBMs and health plans. I really don't have anything much to add. I think the strategy is in place, and we're

Predictable and that is what we have opposite.

What we have done previously to make what we call in the phase two trial, where the basic has.

Classical dose escalation, we couldnt deficiency.

Clear effect on the different doses with indicating that we have a compound that could be titrated up and down.

We achieved the expected response.

Okay.

Got it thank you very helpful.

Next question.

Comes from the line of Joseph Schwartz from SBB Leerink. Your line is now open.

Hi, I'm Jerry dialing in for Joe. Thank you for taking our question I'm actually going to ask you on transcon PTH the data that we're expecting in <unk>.

[inaudible]

to make the announcements as they become public information in negotiations. Also, bear in mind that when you're talking PBMs, you're in a very tight spot right now due to the way that the system is set up where basically announcements are coming out in August and very early September.

Well ahead of the kidney function data I was just wondering if you could give us insight into how youre thinking about incorporating the kidney results considering everyone will be on drug.

The study itself.

Yaron Benjamin Werber: The next question comes from the line of Yanan Zhu from Wells Fargo Securities. Please limit yourself to one question. Your line is now open.

What would be a good result for those patients who have different degrees of calcification over the year.

What can.

Hi, thanks for taking my question and congrats.

<unk> centre, a smaller mix of back then how do you interpret the results with everyone on drop thank you very much.

[inaudible]

of the labeled 0.24 mg per kg per week. So for those physicians who, you know, I guess first of all, how prevalent is that? And for those physicians, how do you think they will interpret the label and then proceed to use Skytrofa? And do you need additional studies at a higher dose or including a higher dose in your open-label study that's ongoing to convince those physicians to adopt it more? Thank you.

Yeah, I actually think we already got what from my perspective as well.

When you look on 24 hour urinary retention, which are basic.

The most.

<unk>.

We consider ways to look on re absorption capacity.

In the kidney.

And what we saw in the 58 weeks was that the basic so a continuation of what we observed at six months, we saw a durable response and we hope that we have received the same.

JJ durable response at 84 weeks.

So what is in the labeling is a dosing of 0.24 Unibrand per kilo per week.

The interesting part is that we did a lot of sub analysis, because we basically.

And it's basic, reflecting what we did in both naive patients and what we did in our sweet trial where we took patients from different daily growth hormone concentrations, and all of them got switched over initially to 0.24 milligrams. What we observed in this trial was also that precision, treating the patient to different levels, dependent on clinical outcome, and other important parameters that are observed for the patient. What is important in this case is that you have a compound that can be titrated in for a product because that is very important for the position that you can try trade it in a manner that is predictable.

Patients that basically had already regional impact and we wanted for example to analyze through this subgroup with already established.

<unk> three <unk>.

Impact.

Prognosis of achieving what we call a normalization of 24 hour urinary calcium.

And there was not really the case.

We sold the same path to.

Effect independent on what we call the background.

Graphic related to reabsorb, some staffing again from that perspective, and we saw that was extremely extremely positive results. We hope we can continue to see the same positive trend when we will have 84.

And that is what we have observed. What we have done previously is to do what we call a phase two trial, where the basic has a classical dose escalation, we can definitely see a clear effect on the different doses, which indicates that we have a compound that could be titrated up and down. And we achieved the expected response.

Weeks data here in the.

Every year.

Next question comes from the line of Rene waters from campaign. Your line is now open.

Yes, hi, good evening and thanks for taking my question first of all congratulations.

So from my end.

So I wanted to ask.

Into how this how this approval impact Europe.

Thank you. Very helpful. Next question.

Plants.

Regards to further label extension.

Doreen Dalyan: Hi, I'm Doreen Dalyan on behalf of Joe. Thank you for taking our question. I'm actually going to ask you about transcon PTH, the data that we're expecting in 4Q. So ahead of the kidney function data, I was just wondering if you could give us insight into how you're thinking about interpreting the kidney results considering everyone will be on drugs in the study. So, you know, what would be a good result for those patients who have different degrees of calcification over the years? What can we consider a small or big effect? And how do you interpret the results with everyone on drugs? Thank you very much. Yeah,

Looking beyond <unk>.

Growth hormone deficiency so.

Other indications where growth hormone is impacted.

I could sing is just.

<unk>.

Okay.

I have no belief that we will not.

Had a strong belief we would get the approval because we had such a strong path.

To provide.

When.

We initiated all what we call label expansion.

Makeup beauty, we did extra long time before the approval like Kokomo.

<unk> deficiency activities and we already have been planning additional label expansion to other indications so what.

Saying that that is that the approval is just the element.

Yeah, I actually think we already got what I, from my perspective, is when we look at 24-hour urinary calcium, which is basically the most validated way to look at reabsorption capacity in the kidney. And what we saw in the 58 weeks was that we basically saw a continuation of what we observed at six months. We saw a doable response, and we hope that we will see the same doable response at 84 weeks.

We needed to continue these activities while all of this activity.

Well indicated for a long time before.

That's super helpful. Thank you very much.

Next question comes from the line of cover all of it from Oppenheimer Your line.

<unk> timeframe.

Hey, guys. Thanks for taking my question and my congrats as well.

Just wanted to ask about the upcoming CMP update before Q.

Determined what youre going to be sharing will we see any initial annualized growth velocity.

<unk>.

We are currently analyzing what be going to.

And is now at that dataset and we.

The interesting part is that we did a lot of sub-analysis because we basically have patients that basically have already had real impact. And we wanted, for example, to analyze, do this subgroup with already established real impact, which had a poor prognosis for achieving what we call a normalization of 24-hour urinary calcium, and it was not really the case. We saw the same positive effect independent of what we call the background demographic related to reabsorption establishing again from that perspective. And we saw that it was extremely, extremely positive results. We hope we can continue to see the same positive trend when we have 84 weeks' data here at the end of the year.

Definitely in a position that we will give you an update related to this program.

All right.

Perspective is that we want to make this.

The product as fast as possible.

Sure so favorable for the patient. So we are compromising two things to give information and at the same time to keep.

The trial is blinded and that is a balance where we basically are going back and forth. What is the information we can share without jeopardizing.

The prices of these clinical trials and therefore I cannot completely say what can be sure. What can we not share because that is our continued development I'm not quite sure Dana and her team will really be strong to support to find out how we can find the best balance between this Tuesday.

The next question comes from the line of Renee Wooters from Kingpin. Your line is now open.

F&I.

Next question comes from the line of Anita Dushyanth from Brandenburg capital markets. Your line is now open.

Yes, hi, good evening, and thanks for taking my question. First of all, congratulations.

Hi, good afternoon.

Congratulations also from my end. I wanted to ask how this approval impacts your plans with regard to further label extension, looking beyond adult growth hormone efficiency.

Jason Thanks for taking my question.

Or maybe you could help me understand this a bit I know you mentioned earlier.

And that's going to take some time for that to get on the formulary. So are you.

So there are other indications where growth hormone is impacted.

Kind of.

Providing tremendous support to operate the path to provide patient access.

I actually think it's just to make us... I have no belief that we will not...

I had a strong belief that we would get the approval because we had such a strong package to provide. When we initiated all what we call label expansion activities, we did it actually a long time before the approval, like the adult protomode deficiency activities. And we already have been planning additional label expansion to other indications. So what I'm saying with that is that the approval is just the element that we needed to continue, which was initiated a long time before.

And then I just wanted to know regarding your application in the European region.

That does that.

<unk> Q4.

So I think the yes explain about our.

<unk>.

The spacing, providing a truck to the.

Patient is appeared to team.

Interchange the process compared to.

Again getting reimbursement so yes, Bob Hess.

Basic and besides basic.

Talked about Stephens and this profit.

Our European strategy is an integrated pipeline strategy and be working to find out how we basically have more coal.

We paid enrollment out of our pipeline in Europe that is balanced with two different element between test conversation expansion of the market in the most important regions Europe is still a bed Bath <unk> written some countries it takes up to <unk> to.

Thank you very much. The next question comes from the line of Trevor Alvarez from Oppenheimer. Your line is now open.

Hey, guys, thanks for taking the question and for the clarification. Just want to ask about the upcoming CNP update in 4Q. Have you determined what you're going to be sharing? Will we see any initial annual annualized growth velocity? Thanks.

And from amongst four basic getting reimbursement.

I must factor as to why you typically have a stage process, but we will keep you updated when we have more clarification about the European strategy.

We're currently analyzing what we're going to show on that data set, and we are definitely in a position that we will give you an update related to this program. Our overall perspective is that we want to make this accessible for the patient. So we are compromising two things to give information and, at the same time, to keep the trials blinded. And that is a balance where we basically are going back and forward.

There are no further questions at this time this concludes today's conference call.

Thank you for participating you may now disconnect.

Thank you bye bye.

[music].

Okay.

[music].

Yeah.

[music].

What is the information we can share without jeopardizing the blindness of these clinical trials? And therefore, I cannot concretely say what we can share, and what we cannot share, because that is a continued development. And I'm quite sure Dana and her team will really be strong advocates to find out how we can find the best balance between these two.

The next question comes from the line of Anita Dushan from Bernburg Capital Market. Your line is now open.

Anita Dushan: Hi, good afternoon. Congratulations on the approval and thanks for taking my question. So, Gaspar, maybe you could help me understand this a little bit. I know you mentioned earlier that, you know, it's going to take some time for the doctor to get on the formulary. So, are you kind of familiar with that? Yeah. Yeah. Yeah. Yeah. Yeah. Yeah. Yeah. Yeah. Yeah. Yeah. Yeah. Yeah. Yeah. Yeah. Yeah. Yeah. Yeah. Yeah. Yeah. Yeah, providing some kind of support to accelerate prior authorizations to provide accelerated patient access. And then, regarding your application in the European region, are you still expecting approval in Q4?

So I think Jesper explained about our ASLP program, that is basically providing a drug to the patient in the peer-to-peer team that you initiate the process compared to the basic getting reimbursement. So, YESPA has, that is basic in the slide deck, basically talked about the establishment of this product. Our European strategy is an integrated pipeline strategy, and we are working to find out how we basically have what I call an enrollment rate out of our pipeline in Europe that is balanced with two different elements between cash conversion, and expansion of the market in the most important regions.

Europe is still a very, very diverse region. Some countries take up to 80 to 24 months for basically getting reimbursement. Other ones are much faster. This is why you typically have a bare-bones process. But we will keep you updated when we have more clarification about the European strategy. Are there no further questions?

Operator: There are no further questions at this time. This concludes today's conference call. Thank you for participating. You may now disconnect. Thank you. Bye-bye.

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Good day, and thank you for standing by. Welcome to Ascendis Pharma's Q2 Earnings and SkyTofa Approval 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 the session, you will need to press star 1 on your

Telephone. Please be advised that today's conference is being recorded.

Ascendis Pharma

Thank you everyone for joining our conference call today. I'm Scott Smith, Chief Financial Officer of Ascendis.

Joining me on today's call is Jan Mikkelsen, President and Chief Executive Officer, Jesper Hoyland, Global Chief Commercial Officer, Dr. Dana Pizzuti, Head of Development Operations and Chief Medical Officer, and Dr. Yuhua Punanen, Head of Oncology. Before we begin, I would like to remind you that this conference call will contain forward-looking statements that are intended to be covered under the safe harbor provided by the Examples of such statements may include, but are not limited to, our commercialization and continued development of SkyTrofa, our progress on our pipeline candidates, and our expectations with respect to their continued progress.

Yeah.

[music].

Statements regarding our strategic plans, our goals regarding our clinical pipeline, statements regarding the market potential of Skytropa and our pipeline product candidates, and statements regarding our regulatory activities. These statements are based on the information that is available to us today.

Actual results or events could differ materially from those in the forward-looking statements, and we may not achieve our goals, carry out our plans or intentions, or meet the expectations or projections disclosed in our forward-looking statements, and you should not place undue reliance on these statements. Our forward-looking statements do not reflect the potential impact of any licensing agreements, acquisitions, mergers, dispositions, joint ventures, or investments that we may enter into or terminate. 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 to differ materially, please see the forward-looking statement section in today's press release and the risk factor section of our most recent annual report on Form 20-F. Skytropha was approved by the FDA today for the treatment of pediatric patients one year and older who weigh at least 11.5 kilograms and have growth failure due to inadequate secretion of endogenous growth hormones. Otherwise, please note that our product candidates are investigational products and have not been approved for commercial use. As investigational products, the safety and effectiveness of the product candidates have not been reviewed or approved by any regulatory agency.

None of the statements made on the conference call regarding SkyTrofa and our product candidates should be viewed as promotional. On today's call, we will walk through a presentation available on our website to discuss the FDA approval of Skytropha. We'll discuss our second quarter 2021 financial results and provide further business updates. Following some prepared remarks, we will then open up the call to questions. I will now turn the call over to Jan Mikkelsen, our President and Chief Executive Officer.

Thanks, Scott, and good afternoon. Sometimes when you wake up in the morning, you think in some way about what the day will bring.

But I have to say today was one of the days where you in some way had an agenda; you adapted to it, like Darwinism says, you need to adapt; this is the best way of survival. And I think we are in a great, great place today. And I have to say, if I can see you, you will see a very, very happy ending. The basic people are so happy with what we have achieved today. But, never forget.

Good day.

And thank you for standing by and welcome to U S. N B's pharma Q2 earnings and Sky Sky Sofa approval 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 the session you will need to press star one on your.

At Ascendis, the key element in our core values is to make a meaningful improvement in patient life. We feel strongly that with our focus on science and data. We can use our innovative transcon technologies to develop new and potential best-in-class products that address major unmet medical needs. For example, with the approval in the U.S. of Transcon Growth Hormone, or Skytropa, for Pediatric Growth Hormone Deficiency today. We are one step near to fulfilling our vision tree by tree to become a leading biopharmaceutical company.

Your telephone.

Be advised that today's conference is being recorded if you require any further assistance press star zero, although like to hand, the conference over to your Speaker today, Scott Smith, Senior Vice President and Chief Financial Officer at <unk> Pharma. Please go ahead.

Thank you operator.

Thank you everyone for joining our conference call today, and as Scott Smith, Chief Financial Officer of offenders.

Before I provide a general business update related to our advancing broad pipeline of now more and more product opportunities, I would like to give a short update related to the approval of Skype Trover. The slides can be found on our website, and I would like to refer to them.

Joining me on today's call as the unmet Olson, President and Chief Executive Officer, Jesper Hoiland Global Chief Commercial Officer, Dr. Dana facility head of development operations, and Chief Medical Officer and Dr. <unk>.

Hopkinton head of oncology.

Before we begin I would like to remind you that this conference call will contain 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 our commercialization.

Going to slide number three. Skytober, our first product developed with Transcon technology, is now approved in the U.S. The Transcon technology is a technology we have taken basics from the design to preclinical, to clinical, and now to approval in the U.S. At the same time, it's the first approval of the Transcon technology, but it's also the first FDA-approved one-tweak product for pediatric growth pneumonia, and we're really proud about it.

And continued development of Sky Trophy.

Our progress on our pipeline candidates and our expectations with respect to their continued progress.

Statements regarding our strategic plan our goals regarding our clinical pipeline.

Statements regarding the market potential of <unk> and our pipeline product candidates.

Lives and statements regarding our regulatory filings.

These statements are based on information that is available to us today actual results or events could differ materially from those in the forward looking statements and we may not achieve our goals carry out our plans or intentions or meet the expectations or projections disclosed in our forward looking statements.

When we go to the next slide, and I actually think you can see the label in different places, you basically see what we have seen out from the label is basically what we could basically hope for. And it's basically reflecting the science, the clinical data we have received with it. Today, because of time, I will not go through the entire labeling discussion, but I hope you will have the opportunity to look at it, because everything starts with the mode of action, where we are basically describing how we release and Sumatopine, the same as daily Glutamone, in going to the right efficacy, it goes to the right safety.

And you should not place undue reliance on these statements.

Our forward looking statements do not reflect the potential impact of any licensing agreements acquisitions mergers dispositions joint ventures or investments that we may enter into or terminate.

We assume no obligation to update these statements as circumstances change.

Sept as required by law.

For additional information concerning the factors that could cause actual results to differ materially. Please see the forward looking statements section in today's press release and the risk factors section of our most recent annual report on form 20-F.

And we're feeling really, really confident with the approval and the labeling we have. We have a strong foundation to build up Scarlet Foba, Transcon Glutamone, to be the leading brand. But it has not been an easy journey for the patients. If you go to slide 5.

Sky Trophy was approved by the FDA today for the treatment of pediatric.

Patrick patients, one year and older who way at least 11, and a half kilograms and half growth failure due to inadequate secretion of endogenous growth hormone.

Otherwise please note that our product candidates, our investigational product candidates and have not been approved for commercial use as investigational products the safety and effectiveness.

I often have given you this discussion. It has been possible to develop a long-acting Product, The Basic Providing and Addressing Unmet Medical Needs. Specific, you have in a pediatric population about a daily injection. And despite, 30 years of clinical development, try developing by different kinds of concepts currently today in the US, in Europe, in Japan, patients with growth hormone deficiency are treated with the same entity that actually came into the market in the 80s. There has been an advancement in pen devices.

Product candidates have not been reviewed or approved by any regulatory agency.

None of the statements made on the conference call regarding Sky Trophy, and our product candidates shall be viewed as promotional.

On today's call, we will walk through a presentation available on our website to discuss the FDA approval of Sky Trophy.

We'll discuss our second quarter 2021 financial results and provide further business updates.

Boeing some prepared remarks, we will then open up the call to questions.

I will now turn the call over to yen Michelson, our president and Chief Executive Officer.

Thanks, Scott and good afternoon.

Sometimes when you wake up in the morning, you somebody think about what they will bring.

I have to say today was one of the days, where you somewhere you had an agenda you adapt to it like Darwin. This mistake you need to adapt this is the best way of survival.

There has been advancement that one single product opportunity could also make room temperature stability, as we see with SkyTrooper under approval today. This is the first once-weekly opportunity that Basic are providing, what we call the same entity that we see in David Gautham. But we have also built up what we call optimal elements for patient life and Auto Injector, which facilitates that you have room temperature in use stability. When I talk about all the clinical programs that support the approval of SCAR-CoV-2, it's built on the basics, the high trial, the flight time, and the enlightenment. The High Trial. There are two.

We are in a great.

Great Great place today, and I have to say, if I couldnt see Ya.

C of Advair happy yet that basic.

What we have achieved to date.

But new offer great at.

The key element in our core values is to make a meaningful improvement in patients' lives.

We feel strongly that with our focus on science and data.

We can use our innovative transcon technologies to develop new and potential best in class product.

Address major unmet medical needs.

Does the approval in the U S of transcon growth hormone.

On Scotch Wilbur for pediatric growth hormone deficiency today.

Sum up, it's where we really had clinical trials with new patients. We also had a switch trial in the flight trial, and we continue, in the enlightenment trial, to generate long-term data on how the benefits of our product are really developing on a long-term basis, both related to efficacy and safety. That is our commitment, really to ensure that we're building up the most comprehensive clinical program as support of this approval. The Hydra, as you recall, was a direct comparison to a daily glutamone at the same dose, and we randomized the patient for 2 to 1, and it's basically the main element in the approval related to efficacy.

We are one step.

To fulfill our ambition three by three to become a leading Biopharma company.

Before I provide a general business update.

The two hour.

Advancing broad pipeline of now.

More and more product opportunities I would like to give a short update related to the approval of skytrooper as Scott said.

<unk> can be found on our website and I would like to referring to the slides.

Going.

Slide number three.

<unk>, our first product developed.

The <unk> technology.

It is now.

In the U S.

The Transcon technology is a technology we have taken.

Inc. slide. 8. You can see the results for yourself. And we saw and better analyzed high velocity at 52 V, illustrated from the statistical perspective on the confidence interval, go into safety. And when you talk about pediatric treatment, safety is extremely important, as in all other treatments. But when you think about that you have established and safe from a daily grow to mow.

Income from the design to preclinical too.

Two clinical and now the approval in the U S.

At the same time.

Yes.

<unk> of the Transcon technology, but it is also the first FDA approved.

Product.

For pediatric growth hormone deficiency.

And we really proud about it when we go to the next slide <unk>.

You can see EBIT labeled different places you basically we'll see what we have seen out from the label is basic what we basically could hopeful.

It's very hard to believe that you can compromise, and one we saw. In the summary of a burst event and everything we look at from it, we basically have a similar profile like daily growth tomorrow, including an emogenic profile. Now we're coming to the next stage. We don't want to develop. Transcripts provided by Transcription Outsourcing, LLC, a product addressing one single geographic region.

And it's basic.

Flexing the science the clinical data we have a seat.

To date because of time I will not go through the entire lapping discussion.

But I hope you will have the opportunity to look at because it <unk> start with automotive.

We want to develop Transcontinental Moon from a global commercial strategy. We have already established how we really want to address the two largest commercial markets, the U.S., where we're building up our own commercial organization. We do it because we have a pipeline.

Action were basic are describing how we were leasing and <unk>.

So much with the same aesthetic optimal in going to the right efficacy. It go to the right safety and we're feeling really really confident with.

We have a pipeline of rare disease, endocrinology products, and multiple products in the same therapeutic area. We are in a position that we are ready there, and in the next few slides, we basically will have a presentation from Jesper that will go in and go more in detail. But before I start, I would like to give you the perspective of the Gotham Market. We see the growth market in a global setting of around more than, for example, the US market is around 1.3 billion, where the pediatric growth hormone is estimated to be about 700 mg.

The approvals and the lapping behalf, we have a strong.

The main two build up schedule, but transcon growth hormone to be the leading brand.

But it has not been an estrogen further patients if you go to slide five.

I often have giving you with this discussion.

He has been possible to develop a long acting.

Yeah.

Product that basically provided.

And addressing the unmet medical need specific you have in a pediatric population about daily injection.

But we've also seen that there's a market that's been highly fragmented with multiple products with basically the same product. And this is where we believe the market is really primed for disruption. Time for innovation that has been lacking for 30 years, and that is what we have come up with. And this is why we believe we have a possibility, not only to improve the treatment, but also to improve and expand the market sector. Jesper, will you go over and talk more dedicated to the U.S. commercial effort?

And despite.

30th.

<unk> clinical development try developing by different kind of concept currently today in the U S in Europe and Japan.

Oh.

Patients with growth hormone deficiency is treated with the same entity at.

Into the market in the eighties.

That has been advancement and pin devices that had been advancement that one single product opportunity also could make room temperature stability what.

What we see with Skype for work on the approval to date.

This is the first once weekly.

I have to say that I've been waiting for more than 30 years for this very moment, so it's very emotional.

Opportunity.

That basic are providing.

What we call the same entity.

In Kokomo.

Well if you also have what we call optimal element for the patients life.

I sold my first growth hormone back in 1988, and even at that point in time, everyone was saying, "Wow."

<unk>.

Auto injector.

Facilitate that you.

<unk> room type book to induce stability.

When I talk about all clinical programs that support the approval on schedule for.

Everyone was saying, "Why can we not have a once-weekly growth hormone for little John, for the patient?"

It's built on basic the high trial, the slide type and then documents filed.

This guy's a slow fan.

For me, myself, who has spent my entire career in the endocrine space, I can only say this is truly the first commercial launch that we'll make with Ascendis and with SkyTrooper, and it will pave the way for the success that we have been waiting for. And you can ask me then, what are your ambitions? And I can only say to drive for market leaders. Margaret leadership.

Hi trial just to.

Sum up this way.

<unk>.

Had clinical trials with nave patients. We also had a switch phy and the flight trial and we continue.

In the lightning trial to generate long term data.

How that benefit all our.

Product.

Really developing on long term basis, both related to efficacy and safety.

Market leadership is the absolute term, because then you determine what the market should look like. And there are some pillars as a part of that, which is pointed out on the slide that you...

Is our commitment.

Really to ensure that would be building up the most competitive clinical program as support.

Of this approval.

Hopefully, you can see in front of you on slide 12. First to build creatability, you hire the right people.

The high trial as you recall.

Was a direct comparison to at daily Kokomo at the same dose.

You hire the right people who can do the job.

And we randomized the patient four two to one and its basic the mean.

Then you realize market access.

Support in the approval related to efficacy.

We are in the process of working to

Slight.

Eight you can see the results.

Working to get the ad market access that we need for SkyTroop. But then we also need to...

And we.

So and better annualized height velocity at 52 week.

Illustrated.

From a statistical perspective under competence in Tibet.

You can have the world's best drug, but if you do not have the patients and the caregivers engaged in it, you will not make it.

Going to the.

Safety and when you talk about pediatric treatment safety is extremely important.

As in all oil treatment, but when you think about that you have a step.

You will not make any strong strides. We are working on that, and we are in the process of doing that. And then, of course, all the health care providers.

<unk> and safety.

From a daily growth hormone.

It's very hard to believe that you can compromise BEC.

And when we sold.

One of the first efforts, if you go to slide 13, that we have been working on is building the educational platform.

In the summary of adverse event and everything what we look for we basically had a similar.

Stephane profile like daily growth hormone.

University Educational Efforts truly believe what those outgrowths achieve.

Included in organic profile.

We launched that back in February, and as we are working on it right now, we have more than 100.

Now we are coming to the next states.

We don't want to develop.

Traffic growth.

Similar.

Two.

Our product addressing one single geographic region.

100,000 unique visitors to our site every month.

That would be a sign of the huge interest. That is for SkyTroopers and Caldona Peck, so much hope. We strongly believe that we're coming out to the market, and we can make the difference.

Want to develop.

Transcon growth hormone upfront global commercial strategy.

We have already.

So for most status, how we really want to address the two largest commercial market. The U S. We have been building up our own commercial organization, we do it because we have a pipeline we have a pipeline.

We are also very fortunate in a way because of the U.S. market, and now I'm talking or speaking on slide 13.

Rare disease endocrinology product multiple products in the same therapeutic.

We are addressing the pediatric endocrinology segment, which is relatively small.

Sure.

We are in a position that we are ready there and in the next few slides be basic will have.

1,400 subscribers in total.

And it's a very concentrated market, about...

<unk> presentation from.

80% of all prescriptions come from about...

Yes, Bob.

That would go in and go more in detail, but before.

20% of the suspicions, and we are certainly going to address that mark.

<unk> key start I would like you to give you the perspective of the go to market.

I am absolutely certain that we are taking and doing the right steps going forward for us to be in the position that we are in now.

See the growth hormone market and a global setting of around more than $4 billion.

The U S market is around one 3 billion we.

So, for me, with the team that we have built with my colleagues.

We have a pediatric optimum SMA.

<unk> is about $700 million.

But we're also seeing that as a market that's been highly fragmented with.

We are absolutely ready from this moment on.

to move forward. We have been waiting for it.

Multiple products with basically the same product.

And now is the moment of truth, as I said.

And this is where we believe the market is really primed for disruption.

And while the key components for us are going to be the Ascendis Signature Access Program, or ASAP, and it has different elements to it.

And so innovation there.

Lacking for 30 years.

And that is what we come with and this is why we believe we have and possibility.

All the colleagues that I'm working with have a long, long tenure in the area.

Not only to improve the treatment, but we also have a possibility to improve.

in the area that we are going to work in, but certainly also in the area of the pharmaceutical industry. So we know what it takes to build true access to the market. It takes patience and commitment. We are working on that part. It also takes motivation and access.

And expand the market segment.

That's been.

When you go over and talk more dedicated related to the U S commercial effort.

Absolutely.

This is the moment of truth for me.

I have to say and I have been.

And all these components were put together in the program of ASAP, the Ascendis Signature Access Program for Scott.

<unk>.

Our more than 30 years.

Very moment, so it's very small.

I sold my first growth hormone back in 1988.

[inaudible]

Thank you for joining us today. We have staggered the engagement with our colleagues, so we

And even at that client time, everyone was saying why can we not have a once weekly growth hormone.

We have staggered the engagement with our colleagues.

And I do believe it.

Waning a little Jon for the patients.

We are in the absolute spot where we want to be. And with that in mind, Jan, I would like to pass this over to you for you to expand on.

Now it's done.

This guidance 12.

Truly can shape the market and change the market.

Me myself, where I spent my entire career in the independent space I can only say this is truly the first commercial.

on all the things we've been doing.

Even outside of the U.S.

with Sky Trooper and all the preparations that have happened, but thanks a lot for everyone who has listened. I cannot wait to see you in the not-too-distant future.

<unk> launched that will make with our centers and with Sky Chopa enabled paved the way for the success that we have been waiting for.

See you in the not-too-distant future at Axel Conferences.

And you can ask me then.

A key part of our vision, Tree by Tree, is our global clinical reach and label expansion. In Europe, we expect a decision from the EMEA Commission on our M&A for transconcoctal immune therapy for the treatment of pediatric concoctal immune disorders in the fourth quarter of this year. In Japan, we continue to enroll patients in the 40-subject phase 3 RITE trial for pediatric ophthalmology. And in China, Lishen Pharmaceutical completed enrollment in its Phase III clinical trial of transconverter mole in children with growth hormone deficiency in masks this year.

<unk> ambition and I can only say to drive market leadership.

Market leadership.

Absolute chime.

Then you determine what the market should look like.

And there are some pinterest as a part of that that has pointed out that the slides.

Hopefully here in front of you on slide 12.

First to build credibility.

You hire the right.

Who can do the job.

That's done.

And then you realized market access.

We are in process of working to get the AD market access that we need pescaito path.

Then we also need to motivate that cant gave us because at the end of the day.

People you can have the world's best rock if you do not have the patients at Mccann gave us engaged into it you will not make any strong stripes.

We also have the Global Phase III Foresight Trial underway for adult glutamate deficiency to support our planned label expansion for transcontinental growth tomorrow. Looking past the Forsythe trial, we expect additional studies to support our effort to expand the label beyond pediatric and total growth hormone deficiency. I'm going to the last slide, and it's going back to where I started with our vision of the patient. This is why I go to work. This is why we're living in Ascendis.

We are working on that and we are in process of doing that and then of course, all the health care providers, we are working on.

One of the first ever achieved.

Slide 13 that we have been working on it.

Building the payphone efforts.

Truly believe what those outgrowth G H T stands for.

It's the stories from the patients, our interaction with patients, not only in the era of glutamate, but also in the era of HIV and other diseases we are working with. And we find that... Change we can do for the patient is one of the most important parts of our journey as a singer. I will move over to The Oil Pipeline Update.

We launched that back in February and as we are working on it right now we have more than 100000 unique visitors to.

Go ahead.

That will take as a sign of a huge interest.

That is false guidance.

And following up thanks, so much okay.

We strongly believe that we are coming out to the market and we can make the difference.

I will start first with Transcon PTA. Transcom PTAs, which have the potential to be the first, Hormone Replacement, Phobic Treatment, and HIV. In the U.S., Europe, Japan, South Korea, and China, there are around 400,000 patients just in these five regions. And we are dedicated to getting transplant PTAs available to as many of these patients as fast as possible. From our Phase 2 study and now the 58-week open-label extension date, we are seeing exactly what we expected at home.

We also very thoughts shown in a way because.

Our SaaS market and now I'm talking on speaking to slide 13.

Addressing the pediatric endocrinologist segment, which is relatively small.

You're talking about.

<unk> hundred.

Describe us in charter.

And it's a very concentrated market.

<unk>.

Maybe a percent of all prescriptions come from about 20% of their decisions and we are certainly going to address that market.

Im absolutely certain that we have going and doing the right steps going forward for us to be in the right place.

All the benefits of physiological PTA levels were realized, including normalization of serum calcium, serum phosphate, urinary calcium, bone turnover, and quality of life. We have achieved this promising result by essentially eliminating standard of care treatment in these countries. The 58-week results demonstrate the durable response and well-tolerated safety profile that we hope for with trans-contagious in adult age groups. What is really exciting is that 58 patients continue to remain in open label.

So for me with the team that we.

With my colleagues.

Absolutely ready from.

This moment on to.

To move forward, we have been waiting for it.

And then now is the moment of truth.

One of the key components for us is going to be the simplest signature access program.

Asap.

Bill and it has different elements to it.

All the colleagues that I am working with has a long long tenure in the area.

Endocrinology.

In the area that we're going to work in but certainly also in the area of the pharmaceutical industry. So we know what it takes to build.

I really believe this shows the positive impact transcopy is having on these patients' health. We are hearing from patients and providers that patients' lives are improving, that patients can now become active again, and can return to their daily activities, including work. Read our Phase 2 Part 4 to find out what they say. We have seen how adult HIV patients may benefit independently of background treatment and cause.

True.

Access to the market.

It takes patient enrollments.

We're working on that part.

It also takes motivation and access.

All of these components, we have put together and the program of Asap.

Centers signature access program for Sky Trophy.

Drew and I really really feel comfortable where we are right now and I cannot wait.

The experience with transgruent PHA may lead to a better understanding of the unmet medical needs. Later this year, in the fourth quarter, we expect to have 84 weeks' top line, only data from past, and forward in our Phase 3 trial. 82 adult subjects have been randomized and dosed in North America and Europe, and their enrollment includes leading influential clinical sites in both biographies.

To meet all of Europe going forward.

And do the work that has to be done from now on we have stagger the engagement with the colleagues. So we took on this first and foremost important months in now.

Youre going to hire the rest of the team on board. So we have also financial been various student to what we're doing.

And I do believe that we are an absolute spot, where we want to be and with that in mind, yeah, and I would like to pass it over back to you for you to expand on all the things we've been doing.

The demographics are similar to those in our Phase 2 trial and include a broad representation of non-surgeon ASB subjects, such as primary idiopathic hyperparathyroidism, Dior's syndrome, Barakat syndrome, and ATS1. We expect to announce top-line results for the Phase 3 trial in the first quarter of 2021 and, if successful, to submit an NDA in mid-2013.

Now even outside of the U S and with Sky <unk> and all the preparations that has happened.

Thanks, a lot for everyone to listen I cannot wait to see you in the not too distant future and axle conferences.

Thank you yes.

A key part of our vision.

In Japan, we announced the acceptance of the Clinical Trial Notification and the initiation of the Partway Japan Trial, a single-arm Phase III trial of transcon PTH in a minimum of 12 adult Japanese subjects with HIV. In Greater China, VISION has initiated the Phase 3 Pathway China trial of transcon PHAs in patients with HIV. The design of the Pathway China trial mirrors the design of the Partway trial.

Is our global clinical and REIT and label expansion.

In Europe, we expect a decision from the EMEA Commission on our M&A for Transcon Kokomo for the treatment of pediatric growth hormone in the fourth quarter of this year.

In Japan, we continue to enroll.

Patients in both 40 subject phase III right trial for pediatric growth hormone deficient.

And in China <unk> Pharmaceuticals.

As you can see, we are taking a global clinical approach with transgone PTA. We believe the data being generated is driven by the science establishing transgone PTAs as a replacement therapy with the potential to be first-line therapy for all forms of adult hypoparathy. Turning to Transcon CMP, we are trying to provide an update on our clinical program in the third quarter of this year. As you know, two randomized, double-blinded, placebo-controlled Phase II trials in children aged 2 to 10 years old are ongoing.

Completed enrollment of this phase III clinical trial of transcon growth hormone in children with growth hormone deficiency in mass this year.

We also have a global phase III foresaw trial underway for adult growth hormone deficiency to support our planned label expansion for transcon growth hormone.

Looking past that four star trial, we expect additional studies to support our effort.

To expand the label on pediatric and.

Locomote deficiency.

Going to the last slide and it's going back to where I started we're not a vision about that patient.

And.

The first, the concrete stride, is a dose escalation of 12 to 15 subjects in the East Cohort conducted mainly in North America, Australia, and Europe. The second, won by Decent Pharmaceuticals, is the country's China trial, which is a cohort-expansive trial of at least 60 subjects conducted in China. We are planning to keep the data blind for one year for each cohort. Want to complete it?

This is why I go to work. This is why we are living in our centers.

It's the stores from the patients our interaction with patients not only in the era of <unk>.

Also in the era of HP and other diseases, we are working with and we find that.

Change, we can do for their patient one of the most important.

And part of our journey.

Smbs.

I will move over to.

The oil pipeline update.

Ill start first with Transcon PTH.

We will have robust clinical data from two independent, randomized, double-blinded placebo-controlled trials. For acondoplasia patients, our top priority is to develop and commercialize a highly differentiated, safe, and effective treatment option that addresses the co-morbidities of the disease and not just that. Switching to oncology, we continue to make progress on both programs. Our Transcon TLR78 agonist is designed for inter-tumoral delivery, allowing for long-term sustained local relief for Potential Superior Efficacy with Minimal Systemic Adverse Events. Filing their I.D.

Transcon PTH.

Which has the potential to be the first.

<unk> replacement for the treatment and HP.

In the U S Europe, Japan, South Korea, and China that around 400000 patients just in these five regions and.

And we are dedicated.

To get Transcon PTH available to as many of these patients as fast as possible.

From our phase II study and now 58 week open label extension data.

We are seeing exactly what we expected and hopeful.

Last year was a major achievement for the company. It was the first IND delivering Transcom hydrogel technology for sustained localized delivery. Just a few comments on our Hydra-T technology. What we are developing at Ascendis is not just the traditional intratermal delivery based on injecting a normal subcutaneous formulation into the patient, where the active compound will disappear in a few hours from then. The Transcon intratumor delivery technology is designed to release an unmodified drug over weeks and months inside the tumor at the site of injection. This highly sustained local concentration of VIT in the tumor is intended to kick-start the immune system without systemic toxicity by activating the immune system locally in the tumor and draining lymph.

All of the benefit of physiological PTH levels were realized including normalization of <unk> serum phosphate urinary catch them all into renewable and quality of life.

Rehab sheath this promising results.

Essentially eliminating.

<unk> standard of care treatment for these patients.

The 58 week results demonstrates that durable response, and well tolerated safety profile that behoof upward transcon PTH in adult <unk> patients.

What is really exciting.

58 patient continue to remain in the open label extension.

I really believe this shows the positive impact Transcon PTH is having on these patients short term sense.

We are hearing from patient and provider that patient slide are improving that.

We aim not only to kill the injected tumor but to drive a systemic immune response that will also kill non-injected tumors. This is the potential we want to achieve with a true inter-tumor delivery technology. We have been dosing subjects with transcon TLR7-8 agonist in the monotherapy model.

Patients can now become active again, Ken will turn to the daily activities.

Activities, including work.

Retail phase III path for what data.

We have seen how adult <unk> patient benefit independent of background treatment and Kohl's.

The experience with Transcon PS.

<unk> may lead to better understanding of the unmet medical need.

Later this year in the fourth quarter, we expect to have 84 weeks top line.

And we recently initiated dose escalation in the combination arm with a checkpoint inhibitor. We expect to have initial results for monotherapy dose escalation for transgon TLR78 agonist in the fourth phase, for our second oncology program. We are planning to submit an R&D application for Transcon IL-2 beta-gamma in the third quarter of this year. Our understanding of the biology has guided us in designing a compound with independently optimized receptor-biased potency pharmacokinetics to create a potential best-in-class IL-2 product. By solving the different elements related to efficacy and safety independently, we believe it will be possible to realize the full potential of the IL-2 pathway. The Samurai.

OLED data from path.

Cohort.

In our phase III trial.

82 adult subjects have been randomized and dose in North America and Europe.

The enrollment include leading influential clinical site in both Geographics.

The demographics are similar to boost in our phase II trial in <unk>.

At Port representation of non HBV subjects, such as primary Egypt, Patrick hybrid territories meant Jos.

Central PA.

Syndrome and <unk>.

We expect to announce top line results for the phase III trial.

The first quarter of 2022.

And if successful and expect to submit an NDA in mid 2022.

In Japan, we announced the acceptance of the clinical trial notification and the indication of the Parkway, Jeff pain trial.

We now have our first approved product, Skytro. We are focused on delivering a successful lung for sclerotropia, and we remain committed to improving patients. We are confident in the way we are strategically thinking about our program, both clinically and commercially as we address large rare disease markets with our product opportunities. We estimate the global daily growth of the market to be around $ a billion, with up to 200,000 patients. In North America, Europe, and Japan, we believe that Transcon PGAs address a market opportunity greater than 5 billion US dollars.

Our single arm phase III trial of Transcon PTH in a minimum of 12.

Japanese subjects with HP.

Yes.

In greater China leasing has initiated the phase III pocket, China trial of Transcon PTH.

<unk> in patients with the HP.

The design of the pathway, China trial mirrors, the design of the pathway trial.

As you can see we have taken a global clinical approach with transcon PTH evenly.

Do you believe the data being generated is driven by the signs in the.

Staffing transcon PTH asset rotations therapy with the potential to be first line therapy for all forms of adult hyperparathyroidism.

Turning to Transcon CMP, we're trying to provide an update on our clinical program in the third quarter of this year.

We are committed to bringing the market leader in each of these endocrinology variances markets, with the approval of Skype Open. We believe that it's a great validation of the Transcom technology and gives us hope that we can address major unmet medical needs with high success rates. Our vision has never changed, and I will repeat it again and again: we want to establish Ascendis as a fully integrated leading global biopharmaceutical company with a portfolio of multiple independent, market-leading products in different therapeutic areas. Now, let me turn the call over to Scott for a financial review before we open it up to questions.

As you know.

Two randomized double blinded placebo controlled phase II trials in children eight to 10.

Yes.

Our ongoing.

The first <unk>.

This trial is a dose escalation of 12 to 15 subject.

And each cohort from <unk>.

That mainly in North America, Australia and Europe.

The second one by decent pharmaceuticals.

Is the accomplish China tower, which is a cohort expansion trial of a lease.

Subject conducted in China.

Rio planning to keep the data planned for one year for each call.

Once completed.

You will have robust clinical data from two independent randomized double blinded placebo controlled types.

For achondroplasia patients our top priority is to develop and commercialize a highly.

Turning to our financial results for the quarter ended June 30, 2021, we reported a net loss of €134.4 million, or €2.5 per basic and diluted share, compared to a net loss of €94.9 million, or €1.97 per basic and diluted share, during the same period in 2020. Let me now run through some key components of these results. Research and development costs for the second quarter were €83.3 million, compared to €63.6 million during the same period in 2020. The increase in R&D costs reflects continued advancement of our pipeline. Primary drivers, including an overall increase in personnel-related costs

Different savings safe and effective treatment option that addressed the comorbidities of the disease and not just height.

Switching to oncology.

We continue to make progress on both programs, our transcon Telos emanate equities is.

For industrial most theory delivery.

Allowing for long term sustained local will be.

For potential superior efficacy.

Systemic adverse events.

Filing for <unk>.

Last year was a major achievement for the company.

It was the first delivering.

Levering the transcon hydrogel technology.

Sustained localized delivery.

Just a few comments on our hydro to technology.

For Transcon Growth Hormone, or Lonopexomotrobin, costs were higher due to the buildup of prelaunch inventories, investments to expand our future manufacturing capacity, as well as increased clinical trial-related activity. As a reminder, we currently expense manufacturing activities of Lonapex somatropum as R&D costs in advance of approval. At the time of product approval, a portion of these R&D costs may be reversed and capitalized to inventory as raw materials, work-in-progress, and finished goods, which will result in a one-time benefit to R&D costs. As of today, this would have been approximately 50 million euros for Transcon PTH or Palapeg Terra Peritai.

What we are developing at the same date is not just the traditional intra tumoral delivery.

Based on injection and normal so couldnt changes formulation into the tumor.

We're the <unk> compound will disappear in a few hours pumping takes inside the.

The transcon inter tumor delivery technology.

Time to release an unmodified.

FICO over weeks and months inside the tumor at the site of injection.

This highly sustained local concentrations in the tumor is intended to kickstart kickstart the immune system without systemic toxicity.

Activating this move.

System locally.

The tumor.

And training them.

We aim.

Not only to kill the injected tumor but to drive a systemic immune response.

Costs were higher primarily due to increased clinical trial-related manufacturing and device development costs. For Transcon C&P, costs were higher primarily due to increased manufacturing and clinical trial-related costs. And finally, for our oncology therapeutic area, costs were higher due to increased manufacturing and preclinical costs for transcon IL-2 beta-gamma, and for transcon TLR78 agonists, higher clinical-related costs were partially offset by lower manufacturing and preclinical costs. Selling general and administrative expenses for the second quarter were 35.3 million euros compared to 20.8 million euros during the same period in 2020.

With also kill non indicative too.

This is the potential to be born to Ashish.

With a true intra tumor delivery technology.

We have been dosing, Suffolk, Transco, Telos 708 agonist in the mono therapy bonds, and we recently initiated dose escalation in combination with a checkpoint inhibitor.

We expect to have initial.

Asia was solid for monotherapy dose escalation for Transcon Telos 708 equities in the portfolio.

Hi.

For our second oncology program.

We are planning to submit an IND for transcon <unk> <unk> in the third quarter. This year, our understanding of the biology has guidance.

<unk> us in designing compounds with independently optimized receptor bias potency pharmacokinetic to create a potential best in class.

Carlo.

By solving the different elements related to efficacy and safety independently, we believe it will be possible to realize the full.

These higher expenses primarily reflect an increase in personnel-related IT and other infrastructure costs as we prepare for the launch of SkyTropha. Financial income and expenses in the second quarter included a foreign exchange rate loss of 11.3 million euros compared to a foreign exchange rate loss of 9.9 million euros in the second quarter of 2020, primarily related to translation of our US dollar holdings of cash and marketable securities to euros. We ended the second quarter with cash, cash equivalents, and marketable securities totaling €641.3 million. As of June 30, 2021, the company had 53,900,990 ordinary shares outstanding. Turning to the remainder of 2021.

Full potential of the.

<unk> two pathway.

To summarize.

We have now all first approved product <unk>.

We are focused on delivering a successful loan cost control, but.

We remain committed to improving patient.

We are confident in the way we are strategically thinking about oil programs.

<unk> clinically and commercially to commercially.

We addressed that rare disease market with our product opportunities.

We estimate the global daily glaucoma market to be around.

<unk>.

<unk>.

We had up to 200000 patients.

North America, Europe, and Japan, we believe that transcon, PTH and market opportunity greater than <unk>.

<unk> billion U S dollars.

We are committed to bringing the market leader in each of these endocrine.

Endocrinology rare disease.

Market segments.

With the approval of.

Skype over.

We believe that is a great validation of the transcon technology.

Yes.

And give us.

Hope that we can address.

Major unmet medical needs.

We expect our reported operating expenses for the rest of the year to reflect the first half run rate with some quarter-to-quarter variability, with key drivers including the anticipated launch of SkyTropha, advancing our endocrinology rare disease pipeline, expanding our activities in oncology, and investing in the TransCon technology platform, including Forlanopec somatropin, and the buildup of commercial inventory ahead of launch. Execution of Commercial Launch Activities, Investment, and expanding commercial manufacturing capacity to support anticipated future demand

Success rate.

Our vision has never changed and I'll repeat it again.

We want to establish <unk> as a fully integrated leading global biopharmaceutical company.

With a portfolio of multiple independent.

Market leading product.

In different therapeutic areas now, let me turn the call over to Scott to a fundamental review before we open for questions.

Thank you Ian.

Turning to our financials.

Results for the quarter ended June 32021, we reported a net loss of $134.4 million euro or $2 five euro per basic and diluted share compared to a net loss of $94.9 million euro or $1.97 per basic and diluted share during the same period in 2020.

Continued execution of the Foresight Trial, a global Phase III randomized controlled clinical trial in adults with GHD, and continued execution of the RITE trial, a Phase III randomized controlled clinical trial in children with GHD in Japan, for Palapeg Terra Peritide. Continued execution of the Phase 2 Path Forward Trial, which continues to retain 58 subjects in the Open Label Extension, along with continued execution of the PATHWAY trial, a North American and European Phase III randomized controlled clinical trial in adult type of parathyroidism, and ongoing manufacturing of PPQ batches for Transcon C&P.

Let me now run through some key components of these results.

Research and development costs for the second quarter were $83.3 million euro compared to $63.6 million Euro during the same period in 2020, the increase in R&D costs reflect continued advancement of our pipeline with the primary drivers, including an overall.

The increase in personnel related costs.

For transcon growth hormone or <unk> costs were higher due to buildup of prelaunch inventories investments to expand our future manufacturing capacity as well as increased clinical trial related activities.

As a reminder, we currently expense manufacturing.

Factoring activities have Lana Peg cemetery open as R&D costs in advance of approval at the time of product approval.

A portion of these R&D costs may be reversed in capitalized inventory as raw materials work in progress and finished goods, which will result in a onetime benefit to R&D costs.

Execution of the clinical program, which includes two randomized controlled phase two clinical trials in achondroplasia, the ongoing ACCOMPLISH trial and the ACCOMPLISH China trial, which are being conducted through our strategic investment in Wiesen Pharmaceuticals. And lastly, in our oncology therapeutic area, execution of the TRANSCEND-IT-101 clinical trial for our TRANSCON-TLR78 agonist and Advancing the Transcon We expect other SG&A expenses, including SkyTropha commercial activities, will include Palopeg pre-launch activities and continued investments in personnel, systems, and infrastructure to support a rapidly progressing portfolio and growing organization. Now, I will provide an update on our upcoming clinical milestone. Phelanopeg somatropin.

As of today.

This would have been approximately $50 million euro.

For Transcon, PTH or pallet peg Terra paradigm costs were higher primarily due to increased clinical trial related manufacturing and device development costs.

For transplant CMP costs were higher primarily due to increased manufacturing and.

Clinical trial related costs.

And finally for our oncology therapeutic area costs were higher due to increased manufacturing and preclinical costs for transcon <unk> beta gamma and for Transcon <unk> agonist higher clinical related costs were partially offset by lower manufacturing and preclinical costs.

Selling general and administrative expenses for the second quarter were $35.3 million euro compared to $20.8 million narrow during the same period in 2020.

These higher expenses, primarily reflect an increase in personnel related IP and other infrastructure costs as we prepare for the launch of Sky Trofim.

Okay.

Financial income and expenses in the second quarter included a foreign exchange rate loss of $11.3 million euro compared to a foreign exchange rate loss of $9.9 million Euro in the second quarter of 2020, primarily related to translation of our U S. Dollar holdings of cash and marketable securities to euros.

We ended the second quarter with cash.

Cash cash equivalents and marketable securities totaling $641.3 million Euro.

As of June 32021, the company had 53.900 million 998 ordinary shares outstanding.

Today we have received FDA approval for Skytropha for the treatment of pediatric GHD in the United States. We continue to anticipate European Commission approval for pediatric GHD in the fourth quarter of this year. For Palomeque Terra Peritide, as discussed, we exceeded the target enrollment in the pathway trial and now expect to report top-line results in the first quarter of 2020. For Transcon C&P, we expect to provide a clinical program update in the fourth quarter of this year.

Turning to the remainder of 2021.

We expect our report.

Operating expenses for the rest of the year to reflect the first half run rate with some quarter to quarter variability with key drivers, including the anticipated launch of Sky Trophy.

Advancing our endocrinology rare disease pipeline, expanding our activities in oncology and investing in the Transcon technology platform.

Including.

Adding <unk> somatotrophin.

Buildup of commercial inventory ahead of launch.

Execution of commercial launch activities.

Investment in expanding commercial manufacturing capacity to support anticipated future demand.

Continued execution of the foresight trial a global.

Mobile phase III randomized controlled clinical trial in adults with ghd and.

And continued execution of the right trial, a phase III randomized controlled clinical trial in children with Ghd in Japan.

For Transcon TLR78 agonist, we have initiated the dose escalation arm in combination with the checkpoint inhibitor, and we plan to report initial monotherapy dose escalation data for Transcend IT 101 in the fourth quarter of this year. And finally, for TransCon IL-2 Beta Gamma, we plan to submit an IND later this quarter.

For our pallet peg Terra paratype continued execution of the phase III path forward trial, which.

To retain 58 subjects in the open label extension.

Along with continued execution of the pathway trial at North American and European Phase III randomized controlled clinical trial in adult hyperparathyroidism.

And ongoing manufacturing of PDQ batches.

For Transcon CMP.

Execution of the clinical program, which includes two randomized controlled phase II clinical trials in a contemplation the ongoing accomplish trial and they accomplish China trial, which is being conducted through our strategic investment in visa <unk> pharmaceuticals.

Before we open up the call for questions, I want to reiterate a few points about our anticipated commercial activities for SkyTropha in 2021. With the FDA approval of Skytropha today, we expect to have the product available shortly. Once the product is available, we anticipate beginning to provide access to Skytropha for pediatric GHD patients through our dedicated patient support program. Our medical affairs, our U.S. commercial team, and the entire Ascendis organization are currently ready and prepared to execute launch activities. And again, finally, during Q4, we anticipate European Commission marketing approval for pediatric GHD. With that, Operator, we are now ready to take questions.

And lastly in our oncology therapeutic area execution of the transcend.

101 clinical trial for our Transcon <unk> <unk> agonist and.

And advancing the transcon IL, two or beta gamma program into clinical development.

We expect other SG&A expenses, including Sky Trophy commercial activities will include pellet peg prelaunch activities and can.

<unk> investments in personnel systems and infrastructure to support our rapidly progressing portfolio and growing organization.

Let me now provide an update on our upcoming clinical milestones.

<unk> today, we have received FDA approval for Sky Trophy.

For the treatment of.

Pediatric ghd in the United States.

We continue to anticipate European Commission approval for our pediatric ghd in the fourth quarter of this year.

For <unk> Terra paratype as discussed we exceeded the target enrollment in the pathway trial and now expect to report top line results in the first quarter of 2020.

Alright, so as a reminder, to ask a question, you will need to press star 1 on your telephone. To resolve your question, press the pound key. Again, that is star 1 on your telephone. Please stand by while we compile the Q&A roster. The first question comes from the line of Jessica Fye from JP Morgan. Your line is now open. Hey guys, good afternoon.

Transcon <unk>, we expect to provide a clinical program update in the fourth quarter. This year.

For Transcon <unk> agonist, we have initiated the dose escalation arm in combination with the checkpoint inhibitor and we plan to report initial monotherapy dose escalation data for <unk> 101 in the fourth quarter. This year.

<unk>.

And finally for Transcon <unk> beta gamma we plan to submit an IND later this quarter.

Before we open up the call for questions I want to reiterate a few points about our anticipated commercial activities for sky to growth in 2021.

With the FDA approval of Sky Trophy today, we expect to have.

Available shortly.

Once the product is available we anticipate beginning to provide access to Sky Trophy for pediatric ghd patients through our dedicated patient support program.

Hey guys, good afternoon. Congratulations on the approval, and thanks for taking our questions. A couple for me First, can you please explain which part of the data investors should look to in the label that reflects the superiority of Skytropa's efficacy over Genotropin? Is it the lower bound of the 95% confidence interval in Table 4 of the clinical studies section? Exactly. How does this timing of approval impact your payer conversations, if at all? Is it possible you can catch the late summer window for coverage of the discussions?

Our medical affairs, our U S commercial team and the entire <unk> organization are currently ready and prepared to execute launch activities.

Product and again finally during Q4, we anticipate European Commission marketing approval for pediatric Ghd.

With that operator, we are now ready to take questions.

Alright, so as a reminder to ask a question you will need to press star one on your telephone.

On your question.

Jim.

Again that is star one on your telephone please standby will be composite Q&A roster.

And what's the list price per milligram?

First question comes from the line of Jessica Fye from Jpmorgan. Your line is now open.

and Olajsco Velasco. From my perspective, the best one is to look for the confident interval because it basically describes what is the interval where you are improved with 95% confidence. So when I see or hear what paragraph you are in some way pointing to, I will give you 100% right. This is where I see and where I get my confidence that you will achieve a higher analyzed high velocity in the 95% confidence interval.

Hey, guys. Good afternoon, congrats on the approval and thanks for taking my questions.

A couple for me first can you please explain which part of the data investors should look to in the label that reflects the superiority.

Europe is efficacy over <unk> is at the lower bound of the 95% confidence interval in table four of the clinical study section.

Thanks.

Okay.

How does this timing of approval impact your payer conversations if at all is it possible you can catch the late summer window for coverage for discussion and what's the list price per milligram quantity.

There was one question about the pair system. Are you sure that is something we should start working on, but it's something we have worked for in the last two? Yes, where we basically have an intent, and discussion with Payer, mainly reflecting our discussion on how WeBasic, as Ascendis, is providing a pipeline, a product opportunity, and building up a long-term relationship with the Payer system, not only in the US, but also how we want to address commercialization in the rest of the world.

Let me start.

Your first question case and.

There's different ways you can describe how you ashish in Ohio.

Annualized growth velocity.

From my perspective is the best one is sort of the so the competent in today because the basic described.

What is the interval, where you are improved with a 95% confident.

I see what paragraph or hurtful paragraphs you.

Somebody.

Pointing to I will give you a 100% right this is where I see and well.

Related to the U.S., I think Jesper, you potentially have a short comment about it, even if we limit what we can say now and what we can say from a public statement because that is a discussion that basically can materialize now with approval.

Okay, My comfort that you Ashish, Ohio.

Annualized height velocity in the 95% confidence interval.

That was one Christian.

About the payer system.

That is something we staff working on but it's something we have worked for in the last two.

Yeah, basically Jan and Jessica.

Yes, where we basically have intense.

Of course, things have been busy, we started out this a long time ago; we have a super good team in place.

Discussion with payer, mainly reflecting our discussion on how we basic as SMB is providing a pipeline of product opportunity and building up the long term relationship with the payer system not only in the U S. But also how we want to.

and we are totally prepared for what we are entering.

Unknown Executive, Tazeen Ahmad, Unknown Shareholder, Andreas Argyrides, Stina Singel, Paul Choi, Kenneth Sprogoe, Joseph Schwartz, Yaron Werber, Joseph Schwartz, Yaron Werber, Joseph Schwartz

<unk> and the rest of February.

Related to the U S.

I think just for you potentially will have a short comments about it even if we didn't limit what we can say now and what we can say from a public statement because that is discussions that basically can materialize now they're prudent.

[inaudible]

You are truly welcome where we are right now.

If I could connect a little bit to you, Jess, about it is that we have a commercial strategy. And the commercial strategy is fundamental to be in a position where we basically are looking to maximize the long-term value of our best-in-class growth model. But also, as Jesper said, laying the foundation for future product launches in the endocrinology area. So this is why we're building a leading market share based on overall value propositions. But not only do they fall.

Yeah basically yes.

Yes, Ken.

I would say that of course things has been as generous as we thought about this a long time ago, we had a super good team in place.

We are totally prepared for what we are entering into and we all know we are in it for the long run.

So it's not.

To address about getting access is also getting access under the right circumstances and that's certainly what we are working on SPN speaking almost.

At least in the.

In the days and weeks to come so.

One single element, but both of the patient, caregivers, and healthcare providers. And this is why we basically are developing a strategy for that, because we want to be a leading biopharmaceutical company. And this is why we're building our strategy on maximizing the long-term value of our best-in-class growth model. So this is our focus, and this is one.

Truly welcome where we are right now.

Great. Thank you.

Just if I connect a little bit to you just about it as you know we have a commercial strategy.

And the commercial status.

Basic to be in a position.

We basic outlook looking on maximizing long term value of our best.

Growth hormone.

But also at the same time as Jesper said laying the foundation for future product launch and the <unk>.

And you talked about pricing. Yes, we call it premium-responsible pricing. And then you say, what is premium-responsible pricing? This is because we look at healthcare economic outcome research. Because this is where we're coming from, from the European setting, and we see it. We see that it is a basic, fundamental, good reason to have a premium responsible pricing, and everyone will benefit, all stakeholders, patient, caregivers, physician, including payers.

Technology Vips.

So this is why we are building a leading market share.

Based on overall value proposition, but not only for.

Income one single element, but bulk of the patients caregivers healthcare provider.

And this is why the basic of developing a strategy for that because we want to be a leading biopharma company.

This is why we are building our strategy on maximizing long term value of our.

And cash quarter more so this is our focus and discipline and you talked about pricing, yes, we call. It premium premium responsible pricing and then you say what is premium responsible pricing. This is because we look on health care economic outcome research because this is where im coming from from the European setting.

Basic type this is why we look at it.

And we see it we see that is a basic fundamental good reason basic to have premium responsible pricing and everyone.

And this is what we have proven in our pharmacoeconomic data. And that is why, in some ways, we are clearly saying we are going from a premium responsible price, just to answer your last part of the question.

All stakeholders patients caregivers physicians.

Next one on the queue is Tazeen Ahmad from Bank of America. Your line is now open.

Including periods and this is what we have proven in our pharma economic data and that is why we are some way.

Good afternoon, and congratulations to me as well on the approval. Lots of years of hard work.

Clearly, saying we are going from a premium responsible pricing.

Just to answer your last part of the question.

Yan, can I just clarify one point as it relates to pricing? Is it the plan to announce pricing before you launch, or would we know the price, you know, let's say, on the day that the actual product becomes available? And Jasper, I'm hoping you can walk me through how a patient will initially be able to get access to the drug before it ends up on formulary. I think there is a process that doctors will have to go through.

Yeah.

Excellent on Q is <unk> Ahmad from Bank of America. Your line is now open.

Good afternoon.

And congratulations from me as well on the approval path.

It's hard work.

Yes can I just clarify one point as it relates to pricing is it the plan to announce.

Shifting before you launch or would we would we know the price let's.

Let's say on the day that the actual product becomes available.

And then.

GAAP, where I'm, hoping you can walk me through how a patient will initially be able to get access.

To the drug before.

Have you figured out what that process is, and can you share those steps with us? Thank you. Yeah. Absolutely.

Price ends up on formulary I think there is a process.

The doctors will have to go through have you sorted without processes and can you share those thoughts with us. Thank you.

The first part, I can just briefly say, and it's more or less reflected in what we call premium responsible pricing. We have a clear view of where we want to be in pricing, and this is basically part of our current negotiation. And we will come out shortly with a discussion about what the back price is for it. And I think in the slide deck from Jesper, potentially, you can refer back to that, Jesper, where we basically are going through some of the steps, where we basically are talking about our system. I think it was what I call, we call it, ASAP, Ascendis Signature Assessment Program, and it's basically in slide deck number 15.

Yes, absolutely.

So the first part I can just shortly is saying and it's more or less effective in what we call premium responsible pricing.

Sure.

We have a clear view wherever you want to be in pricing and this is basically part of our current because negotiation and we will come out shortly.

A discussion about what vivek price liquidity and <unk> from both potential you can refer back to that so we'll be basically.

Okay.

Going to some of the steps we have the basic are talking about our system is exiting there was what I call we.

Call It E S.

AEP as soon as SaaS.

<unk> program and its basic in besides day number 15.

As Jan pointed out, ASAP is basically our signature program from Ascendis for SkyTrooper. And what's going to happen...

Yes.

Yes.

Again points out.

<unk> is basically our signature program from Smbs plus type program.

And what's going to happen in actual terms is, I mean, price is many things, as we all know. It's the wag price, and it's the net price.

And.

What's going to happen in actual terms is.

I mean prices many things as we all know.

WAC price and it's.

Net price and.

But we will do is basically if you if you're going to sell it through the you could say that traditional without being on formula.

Transcribed by https://otter.ai

For more information, visit www.davidlewitz.com Prior authorization means that it will go to the health plan.

Route which is what I heard you ask form then you will be paying rent price and then the decision will have to request a prior off.

It <unk>.

Means that it will go to the health plan then it will be evaluated and then often reject it and then come back and then they position but have to.

IOS means that it will go to the health plan, and then it will be evaluated.

Requalify why this patient is best suitable.

Re qualify why this patient is a best suitable for once weekly.

suitable for once weekly, donor pack, and Skype sofa.

I don't want to peg.

And that will happen under certain circumstances. But it's not happened very often.

Central time and.

And that will have them under certain circumstances is not happen very frequently in comparison. If you are not on this formula.

frequently in comparison if you're not

So, that's where we will start out and, of course, as we go by.

So that's why I believe we start out and then of course as we go by we will negotiate ourself into the contracting and into the Pbms.

We will negotiate ourselves into the contract.

We appreciate ourselves into the contracting and into the PPMs.

And get access by doing that so all.

So all in all, we will come to the market with an approach of being price sensitive, being price premium. I think we can also say, but we'll also do it in a way that makes sense for us to make sure that we get optimized.

All in all we will come to the market with an approach of being price sensitive being.

Being priced pregnant.

I think we can also say probably will also do it in a way.

It makes sense for us to make sure that we get optimized.

profitability. Okay, at the end of the day, that's what the job is all about from our side. So that's what

Profitability, which at the end of the day, that's what the job is.

All about from all sides.

So, that's what we will be suing for.

So that's what we will be shooting for.

Yeah, thanks. Can I just ask a follow-up question? So from the time a doctor writes a script, do you have any sense of how long it'll take before the patient could actually receive the treatment? So if you are launching in the fourth quarter, do we really expect to see patients be put on drugs in large numbers?

Okay.

Yeah. Thanks can I just ask a follow up so from the time of Doctor writes a script.

Any sense of how long it'll take before the patient could actually receive.

Great.

And.

So if you are launching.

Quarter.

Should we really expect to see patients being put on drug and large numbers because of the dynamics that you just mentioned in the early part of the launch.

https://www.globalonenessproject.org

It can take... two weeks. (inaudible)

Program. There, we will provide the drug up front.

It's it can take.

Yeah.

As the patients get their approval for the drug, they will be

Two weeks.

Transcripts provided by Transcription Outsourcing, LLC.

Two six weeks to eight weeks before patients get put on but what we will do in the meantime, with all SaaS that program is basically our Asap program <unk> nickname fast that programs. When other companies are doing it of course vehicle at the incentive signature program.

So you will not see any fast uptake, but you certainly will not see any slow uptake.

I think I got something wrong, but you will see that we move forward. And that's a thing for.

We do believe that the ASAP program, the Ascendis program, will certainly put us in a very strong position for the time to come.

Ed.

We will provide the drug upfront.

The first mover advantage we will certainly also enjoy in the time to come.

And the auto injector to the patients and then as the patients get the approval for the drug they will be client transfer into the health plans and thereby getting access and being painful. So you will not see any fast uptake.

Okay, thank you so much for the power.

Next one on the line is Michelle Gilson from Panacore Gen-UT. Your line is now open. Hi, congratulations.

Hi, congratulations. I'm also a very happy Michelle.

But you certainly will.

I noticed the label. It looks very, very similar to daily growth hormone. I guess just with this in mind, do you think physicians will see Skytropha as interchangeable with daily growth hormone in the way that the label is written? And do you anticipate there will be any pull-through demand beyond the initial indication into the other growth hormones approved?

Sorry.

Got something in the wrong.

You will see it as we move forward and as things progress.

So.

We do believe that the Asap program incentive program will certainly put us in a very strong position for the time to come to work.

Drive our market leadership and again remember that we are first in the pediatric shenkman.

when we talk with physicians, when we discuss the entire concept of [inaudible] And I think, as you said, Michel, that is actually illustrated in the labeling where we release it as a somatopene molecule, the same as davycopene. I believe all the aspects of what we see from the science reflected in the labeling indicate that we basically have the same active entity as daily growth and also believe all the clinical data we have generated are supporting the same thing when we look at switching from a Daily Growth Hormone patient over to Sky Trooper, or we look at starting a youth patient on that.

Coming to the market in that first mover advantage. We will certainly also enjoy in the in the time to come.

Okay. Thank you so much for calling.

Thanks, a lot for the question.

Next one on the line is Michelle Gilson from Canaccord Genuity. Your line is now open.

Hi, Congratulations I I'm also very happy Michelle.

[laughter], Yeah, I noticed the label it looks very very similar.

Our two daily growth hormone.

I guess just with this in mind you do you think physicians will see Sky Trophy as interchangeable with daily growth hormone.

And I feel that the confidence we have in our data, we can also communicate to publications, medical affairs, and really illustrate the benefit of the data. Currently, we have approval for Pediatric Growth Hormone Deficiency. We are expensive, and we are working on label expansion.

In a way that the the labels and do you anticipate there will be any pull through demand beyond the initial indication into the other.

Their growth hormone approved indications.

I believe when I look in the labeling.

When we talk with physicians.

When we discuss the entire concept.

As you know, we have initiated an adult Rokamon deficiency trial. We will potentially perceive one or two other indications. To your knowledge, and when we see growth hormone on the market, none of the daily growth hormones have basic..., approval for all the different indications today. So it's an element where we are building up the scientific clinical evidence on the compound.

Skytrooper.

And.

As you said Mitchell Digest illustrated in the labeling.

We've released.

As somewhat hoping molecule the same aesthetic of tomorrow.

I believe over spec from the what we see from the signs reflected into the labeling indicate that.

I'd say half the same entity SD Trimble.

And if I can also add a follow-up question, how do you plan to communicate scitrophos differentiation from other long-acting growth hormones if they are approved in the future? I know you mentioned the immunogenic profile in your prepared remarks.

And also believe all the clinical data we have generated.

Supporting the same thing.

When we look from switching.

From day to go from one patient over to Scott.

What I mentioned was that we had a similar hemogenic readout that you see with data growth hormone, which is really, really a safe treatment. I actually believe in data. And I like to think that when people start to compare different phase 3 trials, then I think the differentiations are really clear. But, Michel, I think it comes back to the mode of action in the beginning, because basically, if you don't have the right molecule, you don't have the right distribution to all the tissues, you will never, in some way, achieve the same thing that you will achieve with a daily somatopene molecule.

We base all we look for starting.

Patients in that.

And I assume that the comfort.

We have in our data we can also communicate to publication medical affairs, and really illustrate the benefit of the <unk>.

Compound.

Currently we path.

<unk> approval in pediatric growth hormone deficiency.

<unk> extensive voice.

On label expansion as you know we have indicated in adult growth hormone deficiency trial.

Potentially.

Rajeev, one or two other indications.

To your knowledge.

And when we see the co promote margate, none of the daily growth hormone having basic.

And they like to think this is the way after 30 years. Still, all patients in Europe, the U.S., Japan, some of the major markets are still being treated with basic, as Jesper said, the old daily growth hormone that was invented in the 80s.

The approval of all the different indication.

To date so it's.

Element.

Where we are building up the scientific clinical.

Everything.

The compounds.

Okay, thank you so much for doing my questions, and congratulations again.

And if I can also add a follow up.

Alright, next one on the queue is Josh Schimmer from Evercore ISI. Your line is now open. Thanks for taking the time.

How do you plan to communicate Scott Prophage differentiation from other long acting growth hormones.

the questions. So stick around on Skytrofa, maybe we can talk.

If they are approved in the future.

I know you mentioned immunogenic profile in your prepared remarks.

About how long do you think it'll take to gain widespread formulary adoption? Is that likely to be the key gating step for uptake, or is it unlikely to be?

E.

What I mentioned was that we had a similar imaging.

Readout that youll see with daily growth hormone with really really.

Physician Hesitancy. Then, it looks like consensus for Skytropha for next year is about one hundred percent.

<unk> treatment.

I actually believe.

Sure.

Data.

And I actually think when people start to compare different phase III trials.

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Then.

The differentiation really clear.

Thanks so much.

But Michelle I think it comes back to the motive action in the in the beginning because your basic.

Thanks, Jess, for your questions. If I can just start with your last question, we have not provided any kind of guidance on our expected sales for 2022. We feel our... Strategy is really to create long-term value from the product opportunity. And addressing a little bit your first questions, I believe when you...

Please.

If you don't have the right molecule.

You don't have the right distribution to all the tissues you would never somebody can achieve the same thing that you will ashish with a daily so so much of the molecule.

And this is why after 30 years.

Still.

Patients in Europe.

U S.

Japan and some of the major markets are still being treated with basic Ses, let's say the old daily growth hormone that got invented in the eighties.

Think about the view from a physician. The physician can see and know if I have a once-weekly tarot card, that basic is in a position. We know that less than 20% of the patients today in this market segment, when we look at the U.S., basically are compliant, meaning that they pick up 80% of the prescriptions. So, all from that perspective.

Okay.

Okay. Thank you so much for.

All my questions and congratulations again.

And we have is that you have.

[laughter].

Alright next one on the Q is Josh <unk> from Evercore ISI. Your line is now open.

Hey, thanks for taking questions.

Sticking.

Scott maybe you can talk about.

How long do you think it'll take to gain widespread formulary adoption is that likely to be the key gating step for uptake or is it likely to be physician hesitancy and then it looks like consensus for Sky Trophy for next year is about $110 million in cells is that in.

We know, and the physicians know, that they can get a better outcome with giving a once-a-weekly product. So go to the patient that is sitting with the caregiver in front of them and having to make a choice. You can take a daily protocol, 7 injections, or you can potentially go down to 5 injections a week, many, many, many do. And then you get 2 out, or you can take a one-s

On an old Barbara ballpark of what you think is reasonable considering some of the lunch headwinds you'll you'll have to push there. Thanks so much.

Thanks Jess.

For your questions.

If I can just talk with the last question, we have not provided any kind of guidance to our.

And likely, the daily need to be in a cool shade all the time. So basic, patient comfort. What do you want if the child is traveling, going to the grandparents? They need to be sure there is a cool bag all the time and other things.

<unk> generated sales for 'twenty 'twenty two.

We feel.

Sure.

Strategy is really to create long term value about the product opportunity.

In addressing liberate your first questions I believe when you.

So this, I believe, when all our knowledge from the patient and other things, the caregivers, is such an obvious choice if you can provide the same. We believe we can do that and have the same safety and tolerability. Going to your question about reimbursement, I think if you have the efficacy right, the safety right, and the turbidity right, then you come back to, "How fast can we go into the different reimbursements?".

Expect about the view from our physicians.

The physician.

You.

Can see and know.

<unk>.

I have a once weekly therapy.

That basic are in a position.

We know that only 20% less than 20% of the page.

In this market segment, when we look at the U S basis.

Compliance, meaning is that they take.

Pick up 80% of the prescription.

So from that perspective.

And this is where we are building up what I call Premier Responsible Pricing because we believe we deserve it and Premier Pricing. But we also want to build on what we really call the optimal view from Ascendis, building up a company of long-term value for each of these products. So we are not in a volume game; we are in a value game. And that is how we build up our commercial strategy.

No.

The physician note that they can get a better outcome with <unk>.

<unk> a once weekly.

Product.

Yes.

Go ahead till the patient that is sitting with the <unk> and front.

And having to take a choice you can take a daily growth hormone.

<unk> injection or you can potentially go down to five injection that week with many man.

Giving to do.

And then you get worldcom.

Or you can take a once weekly.

Unlikely the daily needs to be in the cool shade, all the time, so basic patient comfort.

Great, thanks very much.

Yeah, now if I can just say it at...

What do we have one if the child is traveling going to the grandparents that need.

Today is day one, and I can tell you we have done a lot of preparation for this. We have worked on this for the last couple of years to get ready.

And sure there is a call back all the time and other things like that so.

I believe when all the notice from the patients and other things that <unk> was it.

out of preparedness for this. We have worked for this for the last couple of years to get ready for this moment, and all the things are in place for a very successful launch and become the company we want to be going forward, not only with the SkyTrooper, but certainly also to build the platform for our future that Jan was also talking about in his introduction.

Market shows.

If you can provide the safe.

The efficacy and we believe we can do that.

To be safety and Tolerability.

Okay.

Going to your question about the reimbursement I think if you have the efficacy right.

Safety right and the ability.

Then you come back to.

How fast can we go into the different reimbursement system.

This is where the building what I call.

Premier responsible pricing because we believe they deserve.

The next question comes from the line of Alita Young from Cancer. Your line is now open.

And premium pricing.

We also want to build on what we really call the optimum.

<unk> is building up a company of long term value.

I guess a couple, and actually, I'm going to ask one that's not related to Skytropa. But the first one on Skytropa is, can you just talk a little bit about how to think about the Medicare and Medicaid mix, like the government channel mix, if there's any, versus commercial. My second question is, you know, you noted that $2.8 billion, rest of the world. Can you kind of break that out by patient numbers or how to think about it either on a revenue basis, like Europe versus like, you know, Asia?

I'm, just calling opportunities. So we are not in a volume game.

And that is how we build up our commercial strategy.

Great. Thanks very much.

Yes, if I can just add to daves day, one and I can tell you we have done a lot of preparedness.

For this we have worked for this for the last couple of years to get ready for this moment and.

All the things.

And place for doing a very successful launch of become the company, we want to be going forward not only with <unk>, but certainly also to build the platform for our future and again was also.

You're talking about in his introduction.

Yes.

And then my last question is just going back to the TLR78 program, you know, should we expect that you're going to be at doses at the end of the year that might show some efficacy? And then how do we kind of think about what you're kind of expecting for the combination study that probably will have some data, hopefully, next year? Thanks.

Thanks, guys.

Next question comes from the line of Alicia Young from Cantor. Your line is now open.

Hey, guys. Thanks for taking my questions and congrats on the approval Wonderful road.

Okay.

I guess, a couple and I actually I'm going to ask one that's not related to skytrooper, but first of all on Sketchup was can you just talk a little bit about.

How to think about the Medicare and Medicaid mix like the government channel mix, if there's any versus like commercial.

My second question is.

That $2.8 billion rest of world.

Can you comment you didnt break that out by patient numbers or how to think about it either on a revenue basis like Europe.

Versus like.

Asia and then my last question is just going back to <unk> 708 program.

Thanks a lot. It was actually nice to get a question outside.

Should we expect that youre going to be at doses.

At the end of the year that might show some efficacy and then how.

And then think about what youre kind of expecting for the combination study that probably will have some data hopefully next year. Thanks.

Let us move back to some of your growth hormone questions. Let me take the question in the breakdown reflecting the growth hormone market. The U.S. is the single largest market, but then we basically have two, you can say, Asian markets that are basically competing to be number two or three. Currently, Japan is the second largest single market. China is basic in basic, and we expect that it will pass.

Thanks, a lot.

It was actually.

I think the good Christian outside.

Hello.

So what about that.

Move back to some of your growth hormone Alright, Christian let me.

The Christian with the breakdown, reflecting the go to market and the.

You can say Asian market that basic are competing on being number two or three.

Currently Japan is the second largest single.

Okay.

China is basically.

We expect that it was path.

Japan, this year, moving up to more than 800 million as a single, but really has major, major advancement every year. Then you have Europe that's just a little bit under this market. Japan is very interesting because it was built on a little bit of local producers because it's been easy for local producers to enter the market because there has been no innovation for 30 years.

Japan this year.

Moving up to more than $800 million are singles, but really have major major advancement.

Then you have Europe, that's just little.

<unk> Bonder this market, Japan is very interesting because it also build a little bit of local producers because it's been easy for local producer to enter the market because there has been no innovation for over 30 years and this is where our strategy come in we have now U S commercial strategy, Yes, Paul.

And this is where our strategy comes in. We now have a U.S. commercial strategy; YESPA has that. We have our recent pharmaceutical company, Greater China. We are now running the clinical development from Japan, meaning we basically have a global clinical reach when we have that. By doing that, we will also have a global commercial strategy built on one single thing, pipeline deals, not single product deals, in new places where we don't want to do it ourselves.

We have our recent pharmaceutical greater China, we now running the clinical development from Japan, meaning as we basically have our global kidney could reach when behalf that doing that we also will have our global commercial strategy building on one single thing pipeline.

Sales not single product deal few places, where we don't want to do it ourselves we are going to a place where the basic have the infrastructure to support us to build it up as a leading brand also in this era, but we still will be part of what we call the value creation and there will still.

We will go to a place where they basically have the infrastructure to support us to build it up as a leading brand, also in this area, but we will still be part of what we call value creation. And it will still be run under a global umbrella, both related to marketing measures and medical affairs, because we believe in the global market; we don't believe in the local market more. This is a global society, and this is how we're building our global strategy.

On a global umbrella both related to marketing measures medical affair, because BP lead in global market. We don't think lead is broken market. More this is a global society and this is how we are building up our global strategy and this is where we have a global aspect everything we'll be doing.

And this is where we have a global aspect for everything we're doing. Related to the specific question related to commercial life or commercial patients or not commercial patients, I think we have a clear view of where we want to be. Jesper, you can clearly say shortly where our focus will be, and I can move on back to the last question.

Roger.

And related to the specific question related to commercial life of commercial patients on our commercial patients I think we have a clear view of where we want to boot.

Yesterday, you can clear to say shortly well focus will be.

King.

Absolutely. I mean, the key focus is, of course, in the commercial space. Again, keep in mind that this is for children.

The last question.

Absolutely.

A key key focus is of course in the commercial space again keep in mind that this is pediatrics.

Transcribed by https://otter.ai

This is not really playing.

Playing that Medicare mitigate space training I think Tim So our focus is on.

Our focus is on the...

on the 185 million patients that are to be found.

On the 185 million patients that are looking.

in the commercial

It can be found in.

And commercial space of Peaky focus there and just to add onto what the yen is saying keep in mind that it would hit.

and just to add to what Yan is saying, keep in mind that if you hit China in the top seven markets in the world, meaning Japan, the U.S., and Europe.

The top seven markets in the World mean.

Meaning Japan U S and Europe side.

Hitting more than 7% to 5% of the world's pharma market and Thats of course, where our focus is going to be going.

And, of course, where our focus is going to be going forward.

Related to oncology, what we have said and what we basically are speaking to, at the end of the year, we will give an update. One thing I can say is that I'm really thrilled with this product opportunity. For the first time, there is what I call a true inter-tumor delivery technology, and V12 has that aspect.

Okay.

And related to oncology, what Bill has said.

The basic of sticking to it.

End of the year, we've been keeping an update.

One thing I can say, we're really thrilled with this product opportunity for first time, those what I call true into tumor.

<unk> delivery technology.

Alright, next one on the queue is David Lebowitz from Morgan Stanley. Your line is now open. Hi, this is Avatar Jones on behalf of David today.

With that aspect.

Alright next one on the queue is David Lebowitz from Morgan Stanley. Your line is now open.

Hi, This is avatar Jones on for David today.

Hi, this is Avatar Jones speaking on behalf of David today. A couple more questions for us on the access side. Firstly, what percent of major payers have already been engaged?

More questions for us on the access side.

Firstly what percent of major payers have already been engaged.

Secondly, can you quantify what premium responsible pricing looks like.

Thank you.

And finally, how would you see a step therapy requirement through a cheaper alternative impact.

The first thing I think we and Jesper have really provided so much insight we can provide, and I think it's hard for us to provide more because we are not disclosing our long strategy, how we are moving forward; we are disclosing exactly the overall picture of the long strategy, how we base our position on the product opportunity, what has been our strategy, what we have done, and I think we have done that in a pretty detailed manner. Premium pricing is what we discussed before, and we call it premium responsible pricing because we believe that our product deserves premium pricing and because we believe it provides a pharmacologic economic benefit for everyone from the patient, for the physician, for all the stakeholders, including the payer system.

Coupling adoption.

Thank you the first thing.

I think we have.

Really provided some insight we can provide and our thing is hard for us to provide more because we are not disclosing our long strategy. How we are moving forward we are disclosing exactly.

Secondly, the overall picture of the long strategy, how we basic our position of the product opportunity what has been our strategy. What we have done and I think we have done that in a pretty detailed manner premium pricing is what we discussed before and we.

Call it premium responsible pricing because we believe that our product to suit a premium pricing and because we believe it provides in pharmacopeia.

Economic benefit for everyone from the patient for the physician for all of our stakeholders introducing the payer.

So the last question, I do not know Jesper if you have a few comments on that. No, I think I mean, for pricing, of course, very close to heart negotiations are ongoing. The opinions rendered herein are those of the guests, and not necessarily those of Douglas Goldstein, Profile Investment Services, Ltd., or Israel National News.

Payer system. So the last question I do not know yesterdays, if you'll have a few comments to that.

No I think I mean for <unk>.

Of course, very close to hot negotiations is ongoing with a different pbms and health plans.

I.

I really don't have anything much color to add I think strategy is in place and we're moving forward and I can only say I look really really poor too.

Make the announcements as they become public.

Also bear in mind that when you're talking PBMs, you're in a very tight spot right now due to the way that the system is working.

Information in negotiations also bear in mind that when you guys talk with Pbms.

A tight spot.

Not right now.

Due to the way that the system is set up where basically announcements are coming out in August barrier to September.

[inaudible]

The next question comes from the line of Yanan Zhu from Wells Fargo Securities. Please limit yourself to one question. Your line is now open.

Vms for next year.

<unk>.

We are working hard on dania.

Thanks, a lot Jeff.

Next.

Next question comes from the lineup Yanan Zhu from Wells Fargo Securities. Please state with yourself to one question. Your line is now open.

Thank you for taking my question and congratulations on the approval. So I'll just limit myself to one question on the adoption of Skytropha. It's our understanding that in a real world setting, physicians often prescribe daily

Hi, Thanks for taking my question and congrats.

The approval.

I'll just limit myself to one question on the adoption.

Scott.

So it's our understanding that.

In a real world setting.

Physicians, often prescribe daily growth hormone above.

hormone above the labeled 0.24 mg per kg per week. So for those physicians who, you know, I guess first of all, how prevalent is that? And for those physicians, how do you think they will interpret the label and then proceed to use Skytrofa? And do you need additional studies at a higher dose or including a higher dose in your open-label study that's ongoing to convince those physicians to adopt it more? Thank you.

Labeled.

Two four <unk> per kg per week.

For those physicians.

So I guess first of all how prevalent is that and for those physicians.

How do you think they interpret the label and then press.

To use sky profile until you need additional studies at higher dose or including a higher dose.

An open label study that's ongoing to convince those physicians for a greater adoption. Thank you.

Yeah.

So what is in the labeling.

So what is in the labeling is a dosing of 0.24. This basic principle is reflecting what we did in both naive patients and what we did in our switch patients trial, where we took patients from different daily growth hormone concentrations and all of them got switched over initially to 0.24 mg. What we observed in this trial was also that precision, [inaudible] patient to different levels, dependent on clinical outcome, and other important parameters that are observed for the patient.

Uh huh.

Dosing of 424.

Thank you Brian Thank you Luca.

In this space.

<unk>.

Right.

What we did in both net new patients and what we did in <unk>.

<unk> suite.

Trial, where we took patients from different daily growth hormone concentration and all of them could switch over initial 2424.

Any crime.

What we observed in this trial was also that Chris.

Precision.

Basic titrated different pay.

Patient two different levels dependent on clinical outcome and other important pyramid.

What is important in this case is that you have a compound that can be titrated in an [inaudible] predictable way. And that is what we have observed. What we have done previously is to do what we call a phase two trial, where the basic has a classical dose escalation, we can definitely see a clear effect on the different doses, which indicates that we have a compound that could be titrated up and down. And we achieved the expected response.

So.

For the patients.

So.

What is important in this case is that you'll have a compound that can be titrated in.

For our product because that is very important for the precision that you can try it traded in a manner.

So.

Predictable and that is what we have opposite.

We have done previously to make what we call in the phase two trial, where the basic has a classical dose escalation, we can definitely see a clear clear effect.

Type of different doses with indicating that we have a compound that could be titrated up and down.

We achieved the expected response.

Thank you. The next question comes from the line of Joseph Schwartz from SVB Lierink; the line is now open.

Got it thank you very helpful.

Okay.

Next question.

<unk> on the line of Joseph Schwartz from SBB Leerink. Your line is now open.

Hi, I'm Doreen Dalyan on behalf of Joe. Thank you for taking our question. I'm actually going to ask you about Transcon PTH, the data that we're expecting in 4Q. So ahead of the kidney function data, I was just wondering if you could give us insight into how you're thinking about interpreting the kidney.

Hi, I'm Jerry dialing in for Joe. Thank you for taking our question I'm actually going to ask you on transcon PTH. The data that we're expecting in <unk> well ahead of the kidney function data I was just wondering if you could give us insight into how youre thinking about integrating.

<unk> got some kidney results considering everyone will be on drug.

How are you thinking about interpreting the kidney results considering everyone will be on drugs in the study?

The study so what would be a good result for those patients who have different degrees of calcification over the year.

Transcribed by https://otter.ai

What can be considered a smaller mix of back then.

How do you interpret the results with everyone on drugs? Thank you very much.

Do you interpret the results with everyone on drop thank you very much.

Yeah, I think we already got what I, from my perspective, is when we look at 24-hour urinary calcium, which is basically the most validated way to look at reabsorption capacity in the kidney. And what we saw in the 58 weeks was that we basically saw a continuation of what we observed at six months. We saw a doable response. And we hope that we will see the same doable response at 84 weeks.

But yes, I think we already got what from my perspective is.

When we look on 24 hour urinary.

Hudson was a basic.

Most.

Validated way to look on re absorption capacity.

In the kidney.

And what we.

In the 58 weeks was that the basic so a continuation of what we observed at six months, we saw a durable response and we hope that we have received the same durable response at 84 weeks.

The interesting part is that we did a lot of sub-analysis because we basically have patients that basically already had a real impact. And we wanted, for example, to analyze, do this subgroup which had a poor prognosis for achieving what we call a normalization of 24-hour urinary calcium, and it was not really the case. We saw the same positive effect independent of what we call the background demographic related to reabsorption establishing again from that perspective. And we saw that it was extremely, extremely positive results. We hope we can continue to see the same positive trend when we have 84 weeks' data here at the end of the year.

The interesting part is that we did a lot of.

So top analysis, because we basically have patients that basically had already.

Impact and we wanted to put assembled to analyze do this subgroup with already established.

Revenue impact.

Poor prognosis of achieving what we call abnormal.

Station of 24 hour urinary calcium.

And there was not really the case, we sold the same path to.

Effect independent on what we call the background.

Related to reabsorb, the staffing again from that perspective, and we saw there was extreme.

There's some midstream with positive results. We hope we can continue to see the same positive trend.

We will have 84.

Weeks data here in the end of the year.

The next question comes from the line of Renee Wooders from Kingpin. Your line is now open.

Next question comes from the lineup Rene <unk> from.

<unk> Your line is now open.

Yes, hi, good evening. And thanks for taking my question. First of all, congratulations, also on my

Yes, hi, good evening and thanks for taking my question first of all congratulations.

Also from my end.

I wanted to ask how this approval impacts your plans with regard to further label extension, looking beyond adult growth hormone deficiency, so other indications where growth hormone is impacted. [inaudible]

So I wanted to ask how this.

This approval impacts.

Your plans.

With regards.

King further label extension.

Looking beyond <unk>.

Growth hormone deficiency so.

Other indications where growth hormone is impacted.

I actually think it's just to make us... I have no belief that we will not...

I could sing it just make us.

Okay.

I have no belief that we will not.

I had a strong belief that we would get the approval because we had such a strong package to provide. When we initiated all what we call label expansion activities, we did it actually a long time before the approval, like the adult protomode deficiency activities. And we already have been planning additional label expansion to other indications, so what I'm saying with that is that approval is just the element that we needed to continue these activities. All these activities were initiated a long time before.

Ours too.

I had a strong belief we would get the approval because we had such a strong package to provide.

When.

We initiated all what we call label expansion activity, we did actually a long time before the approval like.

<unk> deficiency active.

And we already have been planning additional David expansion to other indications so what I am saying that that is that the approval is just the element that we needed to continue these activities while all of this activity.

Well.

<unk> stated for a long time before.

Thank you very much. The next question comes from the line of Trevor Alvarez from Oppenheimer. Your line is now open.

Yes.

That's a powerful thank you very much.

Next question comes from the line of cover all of it from Oppenheimer. Your line is now open.

Hey guys, thanks for taking the question, and Mike, you're right, as well. Just wanted to ask about the upcoming C&P update in 4Q. Have you determined what you're going to be sharing? Will we see any initial annualized growth velocity? Thanks.

Hey, guys. Thanks for taking my question and my congrats as well.

Just wanted to ask about.

Jimmy CMP update before Q.

And what youre going to be sharing will we see any initial annualized growth velocity.

We are currently analyzing what are you going to show at that dataset and we.

Definitely in a position that we would give you.

We're currently analyzing what we're going to show on that data set, and we are definitely in a position that we will give you an update related to this program. Our overall perspective is that we want to make this accessible for the patient. So we are compromising two things to give information and, at the same time, keep the trials blinded. And that is a balance where we basically are going back and forth about what information we can share without jeopardizing the blindness of these clinical trials.

How they obtain update related to this program.

All.

Perspective is that we want to make this.

The product as fast as possible.

Accessible for their patients. So we are compromising two things to give information.

And at the same time to keep.

But trial blinded and that is a balance where we basically are going back and forth. What is the information. We can share we don't jeopardize the blankness of these clinical trials and therefore I cannot completely.

And therefore, I cannot concretely say what we can share and what we cannot share because that is a continued development. And I'm quite sure Dana and her team will really be strong to support us to find out how we can find the best balance between these two.

<unk>.

What can you share what Kevin not sure because that is our continued development I'm not quite sure Diana and her team will really be strong to support to find out how we can find the best balance between these two things.

The next question comes from the line of Anita Dushan from Brandberg Capital Markets. Your line is now open.

Next question comes from the line of Anita.

<unk> <unk> from <unk> capital markets. Your line is now open.

Hi, good afternoon. Congratulations on the approval and thanks for taking my question. So, Gaspar, maybe you could help me understand this a little bit. I know you mentioned earlier that, you know, it's going to take some time for the doctor to get on the formulary. So, are you kind of familiar with that? Yeah. Yeah. Yeah. Yeah. Yeah. Yeah. Yeah. Yeah. Yeah. Yeah. Yeah. Yeah. Yeah. Yeah. Yeah. Yeah. Yeah. Yeah. Yeah. Yeah, providing some kind of support to accelerate prior authorizations to provide accelerated patient access. And then, regarding your application in the European region, are we still expecting approval in Q4?

Hi, good afternoon.

Thanks for taking my question.

So I guess, maybe you could help me understand this a bit I know you mentioned earlier that.

Take some time for that to get on the formulary.

So are you.

You kind.

Kind of.

All right income that is a collector.

The pie by Nathan to provide.

That's it.

And then I just wanted to know regarding your application in the European region.

We will provide in Q4.

So.

Yes explain about our.

So, I think Jesper explained about our ASLP program, that is basically providing a drug to the patient in the peer-to-peer team that you initiate the process compared to basic getting reimbursement. So, YESPA has, that is basic in the slide deck, basically talked about the establishment of this product. Our European strategy is an integrated pipeline strategy, and we are working to find out how we basically have what I call an enrollment rate out of our pipeline in Europe that is balanced with two different elements between cash conversion, and expansion of the market in the most important regions.

A key program.

Facing providing drug too.

Patients in the team.

<unk> initiated the process compared to be basic getting reimbursement, so yes, Bob Hess.

Yes. It is.

Basic and besides basic.

Talked about standards.

Our European strategy is an integrated pipeline strategy and we're working to find out how we basically have more coal.

Enrollment out of our pipeline in Europe that expense.

Balanced with two different element between cash conversation expansion of the market in the most important regions Europe is still a bev diverse region. Some countries. It takes up to eight to 24 months for basic getting reimbursement.

Europe is still a very, very diverse region. Some countries take up to 80 to 24 months for basically getting reimbursement. Other ones are much faster. This is why you typically have a very staged process. But we will keep you updated when we have more clarification about the European strategy.

Yes.

As far as why you typically have a bath space process, but we will keep you updated when we have more clarification about the European strategy.

There are no further questions at this time. This concludes today's conference call. Thank you for participating. You may now disconnect.

There are no further questions at this time. This concludes today's conference call. Thank you for participating you may now disconnect.