Q4 2022 Ascendis Pharma A/S Earnings Call
Okay.
Good day, and thank you for standing by and welcome to the Cintas pharma full year 2022 financially adult smokers.
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I would like to turn the call over to know what your speaker for today.
Lee Senior director of Investor Relations. Please go ahead the floor is yours.
Thank you operator, and thank you everyone for joining our full year 2022 financial results Conference call I'm, Kim Lee Senior director of Investor Relations at <unk> pharma.
Joining me on the call today, Jan Mikkelsen, President and Chief Executive Officer, Scott Smith, Executive Vice President and Chief Financial Officer.
Dr. Christine a single executive Vice President and head of clinical development oncology and Joe Kelly Senior Vice President head of U S commercial endocrinology.
Before we begin I'd 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 limited to our U S commercialization and continued development of Sky Triple for the U S market the commercialization of Transcon Hgh for the EU market statements regarding the expected timing of approval and launch of Transcon PTH in the U S market this year.
Statements regarding the expected timing of approval of Transcon PTH in Europe statements regarding the potential size of the transcon PTH the market size for Transcon PTH.
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, including the timing and timing of clinical results statements regarding our pipeline product candidates statements regarding our planned regulatory filings are expansion.
And into new therapeutic areas and statements regarding the ability to create a sustainable leading global Biopharma company.
These statements are based on information that is available to us today actual results and events could differ materially from those in the forward looking statements and we may not be able to achieve our goals carry out our plans or intentions or expectations or projections disclosed in our forward looking statements and you should not place undue reliance.
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 made into enter into or terminate we assume no obligation to update these statements as circumstances change expect except as required.
Wired by law for additional information concerning the factors that could cause actual results to differ materially. Please see our forward looking statements section in today's press release and the risk factors sections of our most annual report on form 20-F, which is being filed today February 16 2023.
Transcon human growth hormone or transcon Hgh is approved by the FDA in the U S. Under the brand name Sky Trophy for the treatment of pediatric patients one year or older weighing at least 11, five kilograms and have growth failure due to inadequate secretion of endogenous growth hormone. In addition, Europeans.
Commission has granted marketing authorization for Sky Trophy <unk> pharma developed under the name Transcon Hgh as a once weekly subcutaneous injection for the treatment of children and adolescents, aged three to 18 for growth failure due to insufficient secretion of endogenous growth hormone in general we refer to this product as transco.
And growth hormone.
Unless we're referring to the product in the context of particular jurisdictions, such as the United States or the European Union.
Please note that our product candidates are investigational and are not approved for commercial use as investigational products the safety and effectiveness of our product candidates have not been reviewed or approved by any regulatory agency. None of the statements made on the conference call regarding our product candidates shall be viewed as promotional.
On the call today, we'll discuss our full year 2000, and 2022 financial results. We will provide further business updates. Following some prepared remarks, well then open up the call for questions I'll now turn the call over to Jan Mikkelsen, President and Chief Executive Officer.
Yeah.
Thank you so much.
Ascend is you spend on the unique transcon technology platform.
It enables development of highly differentiated product candidates across multiple.
Paris.
Combining the transcon technology with our Echo Ethernet for product innovation.
Enable us to create and develop product candidates with a higher likelihood of success <unk> seen with conventional drug development.
One of our key product selection criteria Mister.
To fulfill best in class potential on each of the four key pillars of drug development.
Safety.
Yes.
So the ability and convenience.
In addition.
This product candidate must have the potential to achieve $1 billion or greater revenue.
As a good therapeutic indications.
With this approach.
In terms of values of patients signs and passive reactive.
Constrained in our ability to continuously build out a robust pipeline.
While taking product candidates from concept through approval and launch.
We would expect that regulatory approvals of our new product or indication every one to two years.
Fulfilling our vision.
<unk> goal of building, a sustainable profitable leading Biopharma company.
Creating long term value for all stakeholders.
This past year.
We have advanced or Python as planned.
I'm sorry.
23.
April 3rd Paducah date, and expected U S launch of Transcon PTH for Todd.
Patients with <unk>.
In Q2.
Along with an expected European Commission decision during Q4.
Transcon PTH is our second endocrinology rare disease product opportunity.
Representing a potential global opportunity greater than $5 billion.
Turning to Transcon CMP.
Last November we reported 12 months data from our first from the first ever randomized double blinded placebo controlled phase two trial in children diagnosed with echoed a paycheck.
These results give me confidence.
This third endocrinology rare disease product candidate.
Its first approval by 2025 as target and how we should treat victory.
Another covenant, obviously intrigued by its label and geographic expansion.
Continuing to build the value of our existing programs through additional clinical studies for label expansion and global commercial rights.
Starting with our annuity.
Expanded European organization, which is preparing for dogs of Skype for ultra in Germany. This year.
And if approved Transcon PTH <unk> next year.
With this great momentum across our pipeline I would like to review additional details from our major programs.
Turning to grow intermodal.
During the fourth quarter of this year, we plan to report top line results from our global Phase III foresight tried in adult.
<unk> deficiency.
Our potential second indication for transcon growth hormone.
Adult growth hormone deficiency is a serious endocrine disease.
Characteristic by abnormal body composition.
EMEA insulin resistant and impaired quality of life.
Analysis has shown disconcert.
Adult growth hormone deficiency.
We start in mean annualized health care costs more than four times that of a non.
One co promote deficient populations.
Because transcon growth hormone.
Only once we get growth in loan product releasing unmodified so much repeat.
Expect.
It to be the first adult glaucoma treatment to meet or exceed the safety efficacy and tolerability of data growth tomorrow.
Meanwhile, in the U S skytrooper his experience.
But the commercial success it deserves.
Because of its unique product strengths.
As we pre announced during Jpmorgan fourth quarter 2022.
Skytrooper revenue growth too.
$17 1 million.
<unk> Europe .
Providing a strong fundamental for growth in 2023 and after.
With our progress.
Rewards label expansion and planned commercial launch in markets outside the U S. We believe we can track we are on track to Bill Scott sofa into the leading growth hormone product impact.
By increasing the total market site.
As we have predicted.
We are seeing the consolidation of the data growth hormone market.
Auto manufacturers begin to exit the U S market.
Turning to Transcon PTH.
Excitement continues to build among stakeholders around this potential treatment for adult patients with hydropower.
The upcoming could do FID date of April 30.
Our expanded teams hired trained and working to deepen physician and payer awareness of this serious health and quality of life issue that hydropower causes.
We have already made more than 2000 coal to position related to disease awareness and we are encouraged by their interest in learning more about.
The multi organ impact of this disease.
Negative effect on patient quality of life.
Our commercial team medical affairs, feeling where reimbursement and our manufacturing teams are ready to launch transcon PTH in the U S market as soon as possible after approval.
Accordingly.
We are launching transcon PTH, our second endocrinology rare disease product with the same commercial infrastructure that has proven its success with scheduled term.
Coming back to CMP.
As we did with transcon growth hormone.
Transco PTA, we ran a robust phase II trial to confer transcon cfp's target profile or all four key pillars safety efficacy tolerability and convenience.
De risk adverse.
<unk> phase II labor.
This can only be done with a.
Robust randomized placebo controlled trial that will mimic that pivotal trial.
We saw clear success and the Companys trials with Transcon CMP demonstrating superiority over placebo at the 12 months primary endpoint in children, aged two to take.
In addition, we saw clear dose response.
Or.
57 patients who started this trial remain in purple label extension today.
Right.
To extend and confirm these results, including positive treatment effect observed on <unk> related Comorbidities, we are running our phase III approach trial.
S investigator.
Of the phase II results.
They're high interest in our approach trial, and we expect to complete target enrollment of around 80 patients in the next quarter.
During our upcoming end of phase II meeting with FDA.
We expect to calibrate on how to best achieve a broad treatment labeling Brian <unk>.
Labeling alone.
Shifting to oncology.
We're progressing that with developing of our two novel immuno oncology programs Transcon tier telos, seven eight agonist and transcon Iot piece of government.
Reduced two clinical program your position.
This year to start evaluation of clinical efficacy in seven specific tumor types nine different indication with four different combination therapies.
Including by combining our two transcon quality product candidate with each other.
Clinical proof of concept phase two top line results expected.
Starting in 2024.
In addition, this year via the EMEA interchange randomized phase II.
Belief, two or one using transcon to beta gamma and telos seven agonist combination therapy in head and neck cancer.
As we successfully demonstrated with our endocrinology programs via building a solid phase II clinical proof of concept for our oncology products in multiple tumor types in the next one or two years.
As you can see rehab and we'll always focus on achieving best in class product profile to benefit patients.
The four key pillars of safety efficacy tolerability and convenience.
Ers in which we will not compromise.
This development approach, including the extremely robust clinical trial design has precision as dangerous to potentially launch a new product or indication every one to two years building sustainability long term value for all stakeholders.
Each successful clinical trial further confirms the path for Transcon technology platform and our product innovation Alco meeting.
And increase our confidence and likelihood of success for future product candidates.
<unk> expanded pipeline and commercial successes at <unk> remains on track to meet or exceed our goals outlined we shouldn't III by team.
I will now turn the call over to Scott.
At five to the financial overview before.
Before we open up for questions.
Monday tuck in.
Okay.
To follow on <unk> comments, we are excited to see the realization of that vision three by three with a continued flow of new products and additional indications every one to two years.
For example, as Yan noted.
We expect to launch Transcon PTH in the U S and <unk> in Germany. This year, followed by the first European country launch of Transcon PTH in early 2024.
With results for Sky Trophy, and adult growth hormone deficiency, and transcon seat and PNR contract plays out on the horizon. We expect this cadence of approvals and launches to continue beyond 2024.
In this way, we are creating sustainable long term value for our centers and our stakeholders.
Our proven R&D development capabilities.
I will quickly touch on a few points for.
For further details on our full year 2022 financial results. Please refer to our form 20-F, which is being filed today.
As we previously announced in early January Sky Trophy U S revenue for the fourth quarter of 2022 grew to $17 1 million Euro. These results exceeded the algorithm that yen laid out last may which projected $16 million euro.
For the full year 2022, total revenue was $51 2 million euro, including Sky trough of revenue of $35 7 million Euro as well as license clinical supply and services provided to third parties, primarily at vs and pharmaceuticals.
With profitable growth of Sky trough, our overall operating loss grew about 2% sequentially to $147 4 million Euro for the fourth quarter from $144 5 million Euro in the third quarter of 2022.
Finally, we ended 2022 with cash cash equivalents and marketable securities totaling $743 million Euro.
Looking forward as John described at the Jpmorgan Conference Annualizing fourth quarter Sky trough, our revenue of $17 1 million Euro provides a foundation for 2023 and.
In addition, we expect to add at least as many reimbursed patients. This year as we did in 2022, which would provide even greater growth.
As a result at this time, we believe we are on track to exceed the current ascend is compiled consensus estimate for 2023 Sky trough of revenue of 96 million Euro.
Switching to Transcon PTH, our <unk> date is April 30 of this year.
If approved we expect to begin shipping product by the end of the second quarter.
A quick reminder, on selected key 2023 corporate milestones.
For Transcon growth hormone as mentioned, we plan to launch <unk> in Europe , starting with Germany in Q3 and.
And we also expect to report topline data from the global Phase III <unk> trial in adult growth hormone deficiency, our second indication in Q4.
Transcon PTH, we are planning for FDA approval by the <unk> date of April three zero and launch in the U S. By the end of Q2 and.
And we expect the European Commission decision in Q4.
For Transcon CMP, we're on track to complete enrollment of the phase two be approached trial and a contemplation in Q2.
Within our oncology therapeutic area, we expect to report topline results and declared the recommended phase II dose for monotherapy dose escalation cohorts for Transcon IL two beta gamma later this quarter.
And to declare the recommended phase II dose from Trans Con I ought to beta gamma combo therapy with checkpoint inhibitor in Q3.
Yeah.
Finally, as you see with our reporting today continued optimization of finance systems and processes have enabled us to accelerate our year end reporting.
With that operator, we are now ready to take questions.
Yes.
Thank you.
Our first question.
For today.
We will be coming from.
J P. Morgan one moment, please while opening your line.
And thanks for the comments on how to think about Scott drove our sales. This year can you comment on your comfort level with consensus estimates for Transcon PTH this year.
And actually while we're at it where is that consensus figure based on your latest calculation and the analysts numbers.
Just are you reflecting into sky profile of Transcon PTH.
<unk> PTH.
I actually have not looked at.
Same store number for P. J. So I don't think we have collected that information. So I don't think we can really address that we can mainly address the places where we feel confident to give an algorithm that can reflect our expectation as we have done from sky profile.
Okay, I'm going to ask something else then.
Can you tell us how many cumulative new patient prescriptions. There were first guide trophy as of year end I think you were previously giving that quarter by quarter.
And can you also tell us when we should look for the next update from the phase two extension for Transcon CMP.
Just go back to how we basically are looking on the forecasting related to revenue of Scott tool for in the U S. In 2023.
What we have seen in here in 2002 that months by months, we have increased the number of new patient that got reimbursed and we have continued to see this trend also in 2022.
This is why it was important to look on the fourth quarter because you're also seeing at the same time, we are seeing and we shouldnt really strong retention when you start on Skype over Youll stay on schedule. There. So when we take the 17 five.
And Moody's revised debt before Scott is calculating is about seven 7 million Europe .
And you know we basic we'll expect an acceleration of the number of new patients because a lot of lot of good reason and I can come back to that just if you are on that so we actually expect to see more patients per months of new reimbursed patient that we actually sold.
You too.
We're feeling really really really.
Confident that we will exceed the consensus number that is out on the street today related to Sky trough.
The element that basic providing our increased in number of monthly new reimbursed patients this debt with decision and starting really to get the knowledge about the product strength of Sky Trophy is not something you experienced in one months, you'll need to see six months 12 months Gulf data.
And this is what we're starting to see so we've really seen how we really have a highly differentiated product compared to daily growth hormone. The other point is that we see the consolidation of the data growth hormone market that started for about three years ago, where we sold after our phase II data that the consolidation of the data growth.
And the market is really kicking in now so that is.
Major major switch.
Away from some of the six daily growth hormone player, which all have the same product.
So this is why we feel bad about confident about how we really with gross cutover into the most valued product and the growth hormone market in the near future.
And related to CMP, you had accretion related to CMP.
Perhaps you could specify exactly what you wanted to know there.
Well I think in the past you had talked about.
The height velocity for a proportion of patients.
And it had been on to a certain time point and I think we're going to have maybe update that one all of the patients got out to that time point is there.
Yes.
To update that data and when should we expect it.
Yes, we are planning to give you this data and we think it's extremely extremely important to give you this data where we see the kantar.
Continuous effect of Cintas farmers transcon CMP, because I'm still in this extremely struggling manner and this is what we really have got a lot out of analyzing all the data for our accomplish tried to find out why.
They're staying 100% on this treatment and we starting to get much much better understanding of that and this is why we now are discussing with regulatory agencies. How we can have other secondary endpoint, that's really reflecting how we are addressing.
Really the Comorbidities and not just linear growth linear growth is not really the biggest issue for this patient group basic the comorbidity and other effect up disease and this is why we believe transcon CMP is a unique product because you have continuous exposure of the CMP molecule.
And therefore change the things that is not only related to linear growth.
Thank you.
Our next question.
We will be coming from Ahmad Bank of America, and as a reminder, please limit yourself to one question and one follow up and get back in the queue with additional questions. Thank you.
Thanks for taking my question.
As it relates to HPT can you just give us an update if you haven't already on how many patients you've enrolled any early access program. So far and do you have a sense of how many patients will be enrolled in that program by the time of the producer and then following with the launch thanks.
I think that is an extreme.
First of all we are.
Im extremely pleased with how the program is progressing and our Quaker toy interaction and also that we got.
<unk> to start and VIP program, which basically can give the opportunity to get patients.
Under the treatment before we get the expected approval.
We are executing on that.
Starting the entire CIS.
System, and we will explain when we come to the.
Approval process.
What is the number of patients we will have in this trial and also how we continue with these patients.
Okay, maybe just a follow up then would you expect that to be an early thoughts in peso that convert to commercial.
I don't think we are addressing a key element in the ERP program, we addressing the patient group that already.
Experian with <unk> treatment regime, a short acting PK treatment it could be that pond it could be.
For you it could be time, most of someone else, but that was exactly what we are addressing the vast population wherever you basic recruited patients to our phase II program and our phase III program. They are coming from what we call patient that Naver will have been exposed to <unk> treatment. So I don't see that its real.
Really really differentiation between these two patient groups I think bulk patient group has the same high unmet medical need that have the same benefit of the <unk> treatment. So I don't see any kind of difference between these two related to be fast startup in our programs.
Thank you.
Our next question will be coming from.
<unk> <unk> of Bank of America. Your line is open.
Okay.
Today's amount of Bank of America. Your line is open.
I think she just asked the question side go to the next one.
Thank you one moment.
The next question is coming from David the Barlett.
Citi. Your line is open.
Thank you so much for taking my question.
As the.
<unk> date is approaching.
Could you give us any insight into what the label might ultimately look like I know that that pyro is considered an adjunct you seek to become more as a hormone replacement and net par has the presence of a black box for osteosarcoma, but you didn't really have an osteosarcoma experience.
Black box.
Be removed.
Okay, 50 curious to hear your thoughts.
Thanks, David.
I think when we look on the biology, and the product design of Transcon PTH.
We are providing a stable future lottery code level of <unk> 24 hours seven days a week.
We are not providing any.
Hi, Bob Fisher logical concentration of PTH.
<unk> can provide to the anabolic effect that in animal model have been asked.
Shaded with.
After sarcoma.
Specific the RAF model.
And from that perspective is that and also why we got a waiver to make a costume allogeneic trial.
Animal trial.
<unk>.
Not expect and we have not seen any indication in our deepest discussion that we will be coming in the same you can say.
All P short acting PJ its that basically got the same costly thing because we are providing a complete different product profile has been a short acting <unk>.
To our best knowledge today and in our discussions we don't expect any Rems program of you don't expect any black box warning.
There was.
First question and Youre quite right.
We designed transcon PTH <unk> was really to prove a product profile that books, reflecting Holland with Paceman and state often adjunct dose wide to be successful in the clinical trial, you need to stop 100%, 100% from activate <unk>.
Vitamin D and you also need only to catch some supplement that really are just reflecting a normal multivitamins from Costco. So we are really addressing a complete different product profile.
Thanks for taking my question.
Thank you one moment, while we prepare for the next question.
Okay.
And our next question is coming from the <unk> of Cantor Your line is open.
Okay. Thank you for taking my question I guess for Transcon PTH.
Just wondering if you can share them with the latest feedback that your sales team may be received from Carrington physician.
And then maybe talk about your latest thoughts on how you might approach pricing and market access. Thank you.
When I think about the patients wanted to think about with physicians.
The ambitious had not been difference of our latest.
Everyone recognize that hydropower.
Serious disease.
And everyone understand the benefit of replacing.
And missing hollman with physiological level 24 hours seven days a week how it both address short term symptoms by quality of life urinary calcium, but also long term risk.
What we seeing is that awareness.
Of that has been building up must must more about the awareness of the disease.
And I believe this is where we come in with our indication first telling about our the awareness of the disease with <unk>.
Big part of what has happened to date with all our established infrastructure here in the U S and after an approval we can go out and explain how we can benefit.
Is this kind of disease, both short term and long term.
So I really really really feeling that we on right.
What we did with Sky Copa is the guidance, we do we'd always a product we developed best in class products.
Addressing unmet medical need.
And we take.
Premia responsible pricing.
<unk>, where we believe if you really develop a product that really address a real unmet need.
Medical need with a real product.
Is enough for both the patient the physician the society and the payer for us to share that kick because everyone is rena.
No one is looser and this is where we want to be with each of our product we need to have them. So highly differentiated addressing a real unmet medical need and everyone believes is a win for everyone.
And we can see that with transcon PTH.
Great. Thank you.
Thank you one moment, while we prepare for the next question.
And our next question is coming from Paul Choi of Goldman Your line is open.
Thank you good afternoon, and thanks for taking our questions.
Wanted to ask given that you are using the same infrastructure.
Market PTH in the U S that you are currently using for Scott I guess are there any learnings that you might share on the launch of Scott trough out that you would think be applicable or what changes would you make I guess in terms of either your.
Your thoughts on approaching payer access <unk> contracting comparative Sky trough and then I had a pipeline question as a follow up.
Okay. Let me start with that we are utilizing the same infrastructure.
Sure. We have dedicated sales force, we will have dedicated people for two different product, but you know that's a huge difference to be the first product to go out and launch where you establish over infrastructure systems.
All the different necessary teams that is necessary to really to launch a commercial product we have de risk. It back now we coming from a space where we.
Launching form an already successful status commercial infrastructure build from Joe and the other people in Princeton here in the U S. So what we're doing is that the basic are facing.
What I call Transcon PTH into an infrastructure that already has proven its capability with a product I believe really addressing a huge unmet medical need where there is no alternative treatment I think this is a fundamental a huge success.
Okay.
Okay. Thank you.
And then as a follow up just on in terms of the pipeline do.
Do you plan to publish the baseline patient characteristics for the <unk>.
Children that are being enrolled in the <unk>.
Our approach trial and then could you also specify in terms of your oncology program, having Mac population population that youre planning to pursue is there.
It just HPV positive or is it post PD, one and post arbitrage if you could maybe add a little color on that that'd be great. Thank you for taking our questions.
I think Steve you take number two or you can take one unlike in figures.
Let me take a number one it's an interesting question because I do not know if Christian the words really are addressing because I actually think we have published all the demographic from our water.
Patient population and demographic there have been into the accomplish trials with 57% is still in it so.
Little bit partnering with that Christian because all data feeds out then you can say.
Hi.
<unk> not coming with the annualized height velocity prescreening, because it totally irrelevant for the clinical efficacy of it you cannot use and.
Annualized height velocity that had been collected before they go into the trial, because we have 40% of achieve <unk> 205.
Which is <unk> and <unk>.
China has nearly built the normal annualized height velocity and the first four years, you will have a heavily the acceleration of annualized height velocity.
If you take an annualized height velocity just collected up to 12 months before they go into a trial, it's not reflecting any meaningful value that go in and compared to the annualized height velocity you compare in this patient group because the start on the eighth already to this is why you do what is obvious and dropped the bill.
I mean, you're making a placebo group.
This is why you have a placebo group.
And I think that is the key element to do look on our dosing from six to 100, and then you can take six or like muted I also a placebo group and then you can take the six.
Or the placebo.
With them into the <unk> specific.
Hi.
Upper matching matching 100%, but compare.
My basic scientific nonsense, and only a misleading and have not reflecting any kind of solid scientific.
Value in integrating the data.
Dana.
In oncology, we are evaluating for proof of concept efficacy in seven different tumor types.
You are right, we do have one of our priority areas as head and neck cancer.
We are evaluating.
In first or second line metastatic head and neck cancer.
The dose expansion cohort single arm into our belief study.
And those patients will be will have had no more than one line of chemotherapy containing regimen.
The answer to that.
And the randomized phase two study for relief.
Tier one study.
We'll be in the new adjuvant setting. So these are patients in a non metastatic setting before they get surgery, they will get systemic treatment.
Sure.
Surgery, and we're looking at pathologic response.
As our primary endpoint to look for everything.
Proof of concept efficacy.
Thanks Steven.
One moment, while we prepare for our next question.
Our next question is coming from Derek Arturo.
Wells Fargo. Your line is open.
Great. Thanks, and thanks for taking the question. So just two really quick ones from US Yes, I just wanted to confirm I thought I caught you, saying that you were in labeling discussions for Transcon PTH.
Just wanted to confirm that and then also.
I guess when should we expect additional updates from accomplish.
Is that something we should see again first half of this year second half of this year. Thanks.
When you go through an approval process.
I think.
For me, it's such a plan process.
Four months before an expected approval date this detail happened three months before an expected approval this need to have two months.
Before that and so if anyone somewhere had been to an approval process and I think we have 10 weeks before that we took the date now if.
If youre not have stopped label discussion.
The risk of not getting approval is higher.
So therefore I believe this is a way of tracking how we are progressing to the approval process.
Really on track of what everything need to happen.
At that time is that not happening I would be extremely word and funnel.
Going on and so yes, we are in a labeling discussion because you should be that at least three months before an expected approval and this is why I feel that confident that I have not seen anything that not give me a belief that transcon PTH.
The product that is approved.
Sure.
I believe.
And I cannot really remember all of our corporate milestones now sorry for that because that's a little bit too many of them.
But I believe that is in Q4, we will keep you update again related to the 57 patients that would come out from the accomplish stripes. So it would be in the second half of this year.
Excellent. Thank you very much.
Thank you while we prepare for the next question.
The next question is coming from Josh <unk> of Evercore. Your line is open.
Okay.
Great. Thanks, so much for taking the questions first on <unk> could you elaborate a little further on what Youre seeing in terms of daily growth hormone options withdrawing from the market I know there have been some reported shortages, but it didnt realize that reflected the alcohol withdrawal.
Have you seen withdrawing and do you expect others to follow and then how do you anticipate the impact of Novo nordisk potentially launching a.
Once a week growth hormone option is following the market later this year. Thanks.
Let me start.
First on the daily growth hormone.
So I actually believe that is a textbook.
Really.
If I had taken an ebay potential ever done that.
Mike started and my finance.
Project, but it's really really what I call. It takes group exactly.
Growth among all the data growth hormone market, both the first biosimilar with Sandoz and.
Teva.
With the buyer excellent version on it.
And somebody had six player in this market segment hurricane providing exactly perceive entity. The same treatment. So you didn't change the backend for BT, but product dependent on rebate and other things that are different in formulation dosed, a little bit differently devices and other things like that.
So what we sold for about three four years, when we came up with our phase two data we saw already that some of the big player.
Finally started somewhere to where.
Reconsider how can we really played with us coming and superior treatment into this segment and that will be which will be highly different stages compared to what we call. The daily market segment at that time, we already so.
<unk> of the company's basic.
Removing BSA folks. This is step one cycle you will remove salesforce. The second one is that you go to the next state you've removed to hub then you stop off manufacturing, which I think we afford them have done. It now and then you basically are in a position where you are providing mainly patients.
<unk> already our steppers on your product because you will have new where you can change and take new patient and you're providing them.
What's happening said enough for their patient now that would look like a normal notice when due to shortage of a multiple products multiple presentation of their proximal and because that consolidation of the data growth won't happen and really is in the final place I believe none of the other one could take.
So you actually see a shortage of growth hormone treatment in the U S and short <unk>.
To accept that we benefit of that is a great thing for us because it's very both accelerating at the same time, where people really see the benefit.
Clinical experience, how do we use different seed b, having patient for one year or something on treatment. So we're really really we see this benefit and I think this is a great thing.
Sure for Us and we really hopefully we can help as many many of those patients to a voice that going into a shortage of the treatment effect.
Very helpful. Thank you.
Your second question addressed.
Actually I was going to ask whether.
In your view of the differentiation of Transcon CMP, a few Doug considered filing for breakthrough designation.
<unk>.
Yes.
First of all just you also have the accretion reflecting about pertains to the interim of Novo Nordisk long acting.
<unk>.
When I look on the paradigm shift I call it paradigm shift because it come from the time, where all the daily growth hormone.
<unk> the same treatment the same mode of action when you go over to the long acting all of them are providing complete different clinical profile.
The only one that really match an improved version of the data for optimal where you get all that benefit into crime benefit both related to.
Changing not only linear growth in the pediatric segment, but also the other.
Associated into prime benefit like body composition metabolic profile Lipide Marshall cognitive effect in everything that you see because we have the same modified somewhat choppy.
<unk>. So this is why it is also important for us to look in our phase III in adult growth hormone deficiency, because the two.
Two long acting where we believed as potentially one left now, but the Opco phaistos showed basic not any improvement on body composition in the phase III trial Novo Nordisk shows that canonicate behalf of basic to daily growth hormone and this is where we believe BREIT our unmodified tumor tupi.
Potentially we would be at least as good SD crop to them. So we believe that our product profile is always so highly differentiated to both David Guatemala, and other long acting growth hormone that we always will provide us the clinical benefit compared to all other treatment regimes.
Thank you our next question will be coming from.
Lynn Garceau Oppenheimer.
Hey, Mike <unk> questions.
Two from me.
And I know you had responded earlier that.
You don't expect a rems or a black box warning on the PTH label, but could you comment on any potential for.
Monitoring requirements.
With patients on the product.
Could you clarify your question what do you mean exactly.
Well in other words, it if patients need to be.
Monitored for.
Sorry in serum calcium.
Bone Biomarkers whatnot.
That is.
And Christian where you will say will be provided and better stability for this patient group that they have in the current setup. We have a basic are being monitored for CASM really really really awesome and I believe you will see it in different stages I believe when you transition.
Over from what we call the conventional part of therapy over to Transcon PTH I think that will be at least at the same kind of monitoring because you want to be quite sure you've stabilized the patient in the right manner.
Theyre stable, which we see after one to two year on our PTA stopes, where we see more and more stability coming in because.
Capstone metabolic system kit or CASM hemostasis, starting to be stabilized.
I will potentially see from a patient perspective that you will potentially list, what we call monitoring of it I'd say.
This is where I believe that.
Elements like bone marker is not typically something you typically will annualize for any patient group in this way.
We have seen in our.
Clinical studies, where we know patient ARPA over three four years your basic <unk> that we do normally decision more and more and more of a repair meter and that is include both bone density and is also including bone markers. So I will not expect that it will be part of a standup monitoring.
Okay and second question for me is with J.
<unk> hundred Felicia.
Obviously, the primary endpoint for regulatory purposes, it's about tight velocity, but as you had mentioned earlier there are many other benefits.
That.
Placement CMP could could provide could you just.
Sort of inform us as to which of the other benefits that maybe not captured by primary endpoint take data, but would be very important CNS most important by the 800 community.
Yes, I think this is a widely developing transcon CMP, we really aren't developing to provide treatment that cure is addressing linear growth, but we can combine it with the sky telephone than I can.
From a clinical concept I will expect nearly you can decide what kind of linear growth you will have so.
But what we really want to ensuring that we are addressing the underlying comorbidities of their patients and how we measure them is to have specific contemplation specific comorbidities. This is kumar <unk> that is coming and being reported on high high high frequency from the contemplation.
From when you see them.
And then we are also developing in patients.
Specific reported outcome measuring where we trying to capture all the benefit they are feeling because theyre really seeing a huge benefit one of the sink.
<unk>. It has not received a lot of other factors not just related to <unk>.
We see massive change we see how the functional data.
Crucially coated weight to balance do normal to normal operation and other things like that and this is all this element we will try to catch up at the same time, we recast of our kind of pace of specific element.
Basically like a signal path.
<unk> and other things like that.
Yes.
Got it.
Thanks, that's very helpful.
Okay.
Thank you one moment, while we prepare for the next question.
Our next question is coming from Andreas.
Writers.
From Wedbush Your line is open.
Hey, guys. Thanks for taking our question so when you're thinking about.
This is for PTH, when Youre thinking about the opera <unk> launch and the opportunity in PTH. How are you thinking about it compared to the non apparel launch and then maybe you can.
Give us some insights on how not parrot coming off the market at the end of 'twenty threes.
Informing your your expectations.
And the launch and then I have a follow up thanks.
I'm not comparing transcon PTH in any way to net par.
We will have complete different labeling path.
Complete different clinical outcome have complete different clinical benefit so.
Yes.
Different impact on quality of life.
I am not using that as a benchmark.
Anything.
I'm not comparing that there was a <unk>.
That came out with a lengthening SME John .
This is not Buffy addressing.
It was a product that came out not seeing benefit on quality of life. It was a product that came out not showing any benefit of 24 hour urinary calcium not really addressing the underlying disease. So you can I don't know that I'd also any benchmark.
To complete meaningless for me.
Okay, Great that's helpful and maybe.
Okay, I guess, maybe the opportunity that there is no approved product on the market. There were some patients mostly in Europe as well there is still.
On it had access to and no longer are those going to be early.
Adopters are.
Yes. Thanks.
And then I think.
Yes, I think so this is exactly.
Address before.
Yes days patient has been exposed to short acting <unk> treatment.
They are used to daily injection and other elements like that but where we see the huge benefit is independent of the background is that being exposed to short acting <unk>.
So I don't believe data less need for a patient.
To get on Transcon PTH treatment, if you come from the group that has been on.
Short acting PTH or have new oxy, a short acting <unk>.
Patients that have been on short acting pga's half, perhaps and more coming understanding of a daily injection have more commonly understanding.
What they need to do a procedure to get that to happen, but I don't believe that is a big barrier for any of the groups.
Okay, Great and then just a quick one on <unk>.
And maybe you can elaborate and I don't know if you covered this already sorry, if you did I missed it but if you can elaborate on the launch dynamics that are driving growth are you seeing higher switch rates from daily growth hormone.
Is it from me thanks, guys.
We always see.
They're switched rate from daily growth hormone, but we're also seeing an increased number of new patient coming on the treatment.
But the overall number goes up everywhere, so it's not like one.
One getting lift both of them are improving up to.
Really really new height overtime.
Thank you one moment, while we go to the next question.
And our next question is coming from Gary Orben Warburg excuse me of Cowen Your line is open.
Great.
I have two interrelated questions on more coverage.
With us.
PTH coming soon.
There isn't really a competitor do you need to contract or will your contract with pbms to get on formulary and maybe kind of talk about your thoughts there obviously the discounting shouldnt be very high at that point and then secondly, now that Youll have a second program second product approved sort of within the endocrine bag because it gives you more leverage to negotiate better.
<unk>.
For placement for our schedule from thank you.
Yeah.
We never really comments about how our market access strategy is and will be and how we really are.
Tracy.
Reimbursement system in the U S.
But for your information when we obviously look at other product because you can do the same thing I can give you name off.
Products in the U S that basic are in a position to generate multiple billions in revenue in the U S with our basic be provided high rebate.
And I think we will follow this pathway because we are providing such a benefit to the patient the physician and the society that we feel this is a way we will continue our market access strategy.
Impaired to Sky, Tulsa, we actually pretty satisfied with our coverage.
We believe the strength of our product the differentiation Don really.
VITAS to providing two and highly rebated products.
That is to the strengths of having a highly differentiated product.
Yes.
Thank you that's all the time that we have for today. Thank you for joining the conference call you all have a good evening.
Thank you. Thank you so much sir.
The conference will begin shortly.
Lower Johan during Q&A, you can dial star one one.