Ascendis Pharma A/S Q1 2024 Earnings Call Transcript

Quartr
Provided by Quartr
May 2, 2024

There are 9 speakers on the call.

Operator

Good day, and welcome to the Q1 2024 Ascendis Pharma Earnings Conference Call. At this time, all participants are in a listen only mode. After the speakers' presentation, there will be a question and answer session. As a reminder, this call may be recorded. I would now like to turn the call over to Tim Lee, Senior Director, Investor Relations of Cyndus Pharma.

Operator

Please go ahead.

Speaker 1

Thank you, operator, and thank you everyone for joining our Q1 2024 financial results conference call. I'm Tim Lee, Senior Director, Investor Relations at Ascendis Pharma. Joining me on the call today are Jan Mickelson, President and Chief Executive Officer Scott Smith, Executive Vice President and Chief Financial Officer Doctor. Siena Singhal, Executive Vice President of Clinical Development, Oncology and Joe Kelly, U. S.

Speaker 1

General Manager. 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 to statements regarding our commercialization and continued development of Skytopha for the U. S. And European markets as well as certain financial expectations for 2024, our commercialization and development of YORVAIS in the EU and expected timing of the FDA review and potential launch of TransCon PTH in the U.

Speaker 1

S, our pipeline candidates and our expectations with respect to their continued progress and potential commercialization, our strategic plans, our goals regarding our clinical pipeline, including the timing of clinical results our ongoing and planned regulatory filings and our expectations regarding the timing and the results of regulatory decisions, our ability to create value in multiple therapeutic areas outside of endocrinology rare disease and our progress towards Vision 2,030. These statements are based on information that is available to us today. Actual results may differ could differ materially from those in our forward looking statements and you should not place undue reliance on these statements. We assume no obligation to update these statements as circumstances change, except as required by law. For additional information concerning the factors that can cause actual results to differ materially, please see our forward looking statements section in today's press release and the Risk Factors section of our most recent Annual Report on Form 20F filed with the SEC on February 7, 2024.

Speaker 1

TransCon Growth Hormone or TransCon HGH is approved by the it is approved in the U. S. By FDA and the EU has received MAA authorization from the European Commission for the treatment of pediatric growth hormone deficiency. The European Commission and the United Kingdom Medicines and Healthcare Product Regulatory Agency have granted marketing authorization for TransCon PTH as replacement therapy indicated for the treatment of adults with chronic hypoparathyroidism. Otherwise, please note that our product candidates are investigational and not approved for commercial use.

Speaker 1

As investigational products, the safety and effectiveness of product candidates have not been reviewed or approved by any regulatory agency. None of the statements during this conference call regarding our product candidates shall be viewed as promotional. On the call today, we'll discuss our Q1 2024 financial results and we'll provide further business updates. Following some prepared remarks, we'll open the call up for questions. Note, with our PDUFA date for TransCon PTH coming up in less than 2 weeks, we will not comment on our ongoing discussions with FDA and we will not be taking any questions on this topic today.

Speaker 1

With that, let me turn it over to Yan.

Speaker 2

Thank you, Tien. Good afternoon, everyone. Zendesk is a pioneering transform technology platform to build a leading fully integrated biopharma company focused on making a meaningful difference in patients' lives. 2 approved transcon products and solid progress across programs, growing commercial presence and strong partnerships. We believe Ascendis is on the path to sustainable growth and operating cash flow breakeven on a quarterly basis by the end of 2024.

Speaker 2

Our long term commitment in the last 3, 4 years to build up profitable and robust supply chains and the decision we took in 2023 to streamline the company and made us a leaner and more efficient organization. You can see the progress towards operating cash flow per year later this year without compromising our development and commercialization progress. We believe we are well positioned to successfully deliver on our strategic goals and close out our vision 3x3 with regulatory approvals for 3 independent endocrinology rare disease product and continue building a pipeline in other therapeutic areas. Looking to Vision 2,030, we believe each of our 3 rare disease endocrinology portal have the potential to achieve blockbuster status, while we also further expand our pipeline and TransCon platform for future innovation. We have seen validation of our commercialization approach with the ongoing success of SKYTROPHIA in the U.

Speaker 2

S. And are seeing it again with successful launch of uropares in Germany and Austria. In the U. S, the PDUFA date for transplant PT8 is coming up in less than 2 weeks on May 14. If approved, we expect to be ready to launch in the U.

Speaker 2

S. Soon thereafter in Q3. Now let me give an update on each of our programs. When we launched Sartofa in the U. S.

Speaker 2

About 2 years ago, we had 2 goals: 1, to make SKARTOFA the leading product in there the second, to drive U. S. Growth hormone market to become a $3,000,000,000 market. Our strategy is working. 1,000 of patients are now treated with Kytopha, the value leader in the U.

Speaker 2

S. We have a reliable supply chain, ensuring that every patient can benefit from SKYTROFA once they have approval from the insurance company. We estimate that SKYTOVA penetration in the U. S. Pediatric growth hormone deficiency patient population growth to about 17 at the end of the Q1.

Speaker 2

We are proud to observe SCATOVA extend its market value leadership as the only growth hormone product to grow in value in the Q1 of 2024 based on reported results. And we believe Skytober will expand the U. S. Growth tomorrow with the potential to become a blockbuster in the U. S.

Speaker 2

Alone. SKYTRAV sales this quarter more than doubled compared to the Q1 of 2022, with a steady quarter to quarter increase in treated patients. We expect to continue this trend rest of this year. With these strong SKYTOVO results, we continue to expect the full year 2024 SKYTOVA revenue will be between EUR 320,000,000 to EUR 340,000,000 representing year to year growth of 80% to 90%. We expect Gartovo sales to continue to grow through further penetration in the pediatric growth hormone system.

Speaker 2

To further solidify growth, we're also pursuing our 1st label expansion in adult growth hormone deficiency, for which we plan to submit and supplement BLA to the FDA in the Q3 of this year. In addition, we expect top line data from our Phase II trial internal syndrome in the Q4 of 2024. Now turning to TransCon PTH. In Europe, EUROPAD was launched in Germany and Austria at the end of January with the early launch objective of building physician experience with UroPAD. Initial physician feedback from UroPAD has been positive.

Speaker 2

Just 8 weeks in the norms, we estimate prescriptions have been written by 55 doctors, representing around 25% of the target prescribing base with about 100 patients receiving commercial product. As physician begin to get comfort with the treatment benefit of UroPAD in the first patient, we expect physician to take more and more of the patient treatment. As we expand our geographic reach of Urocare to our Europe direct and our international market segment, we are supporting named patient supply programs and plan to provide reimbursed product to meet the needs of patients. In the U. S, pre launch preparations are underway, including the expansion of the U.

Speaker 2

S. Field infrastructure while we are awaiting FDA's decision. Moving now to TransCon CNP. Our value proposition with TransCon CNP is simple. It's to establish a treatment for patients of all ages with achondroplasia.

Speaker 2

Our vision is to address the significant comorbidities associated with achondroplasia that impact health and quality of life as well as linear growth. Later this year in Q3, we plan to report top line results for our pivotal APPROVE trial, which is measuring not only linear growth, but also physical function, body composition and quality of life parameters. We are also evaluating TransCon CNP in newborns with AKONOPLASA less than 2 years of age. We believe that treatment in air condopecia with TransCon CNP as early as possible might mitigate associated comorbidities. In addition, to provide catch up growth to Chevron, who did not receive early treatment, we are evaluating TransCon CNP in combination with TransCon Growth Tumor.

Speaker 2

We expect to enroll all patients in this trial this quarter and provide 26 week top line data for this trial in Q3 Q4 this year. This is our integrated approach to address aconoprazia with multiple trials that are all building towards one goal, to treat the disease. And we are optimistic that our highly differentiated product candidate will continue to show best in class potential growth this multiple studies. Switching now to oncology. Both TransCon IL-two beta gamma and TransCon TLR-seventy eight agonists have shown promising clinical activity as monotherapy and in combination treatment with PEMPO.

Speaker 2

In late stage patient, Roche disease has progressed after standard of care treatments. Next month at ASCO, we will report updated patient data from the ongoing Phase III trial, I believe, that has been reported last year at ESMO. We will present updated clinical data and new biomarker data that further differentiates our clinical program. We will also report promising early data from the ongoing dose expansion cohort of patients with melanoma who have progressed from checkpoint inhibitors using the combination of TransCon IL-two beta gamma and TransCon TLR agonist in these patients. Later this year, in the Q4, we expect data readout in several well defined patient population from our TransCon IL-two beta gamma and TransCon TLR-seventy eight agonist programs.

Speaker 2

Our achievement this quarter give me further confidence that all the elements are in place to fulfill our strategic goals to deliver 3 independent endocrinology rare disease blood dog problem and a strong pipeline in larger therapeutic areas such as oncology, ophthalmology and metabolic diseases with much more to come. I will now turn it over to Scott.

Speaker 3

Thank you. In Q1, we demonstrated significant financial progress toward our goal of becoming operating cash flow breakeven on a quarterly basis by the end of 2024. I will touch on some key points surrounding our financial results, but for further details please refer to our 6 ks filed today. Skytropha revenue for the Q1 of 2024 was €65,000,000 compared to €31,600,000 reported in the Q1 of 2023, an increase of 106% year over year. This growth was driven by significant increase in demand volume more than doubling compared to the prior year period with a slight offset by a combination of slower channel build coverage mix and a modest negative currency impact of €800,000 On a sequential basis, 1st quarter Skytrophy revenue increased 1% compared to the Q4 of 2023.

Speaker 3

Strong mid teens percent demand volume growth was offset primarily by seasonal channel inventory, co pay resets, insurance reauthorizations and a modest negative currency impact of €600,000 Q1 was in line with our high internal expectations and with seasonal headwinds behind and the steady pace of new patient adds expected to persist, we continue to expect full year Skytrophy revenue to be in the range of €320,000,000 to €340,000,000 at average 2023 exchange rates. Shifting to TransCon PTH, UroPath contributed for the first time this quarter with revenue of €1,500,000 representing 2 months of shipments. In Germany and Austria, we shipped directly to retail pharmacies for patient pickup and as a result there is no channel inventory buildup compared to a typical U. S. Launch like we saw with Skytropha where specialty pharmacies hold channel inventory.

Speaker 3

Closing out the top line, total revenue for the Q1 was €95,900,000 including €24,800,000 of non cash license revenue recognized in relation to the formation of Iconis and €3,000,000 of service revenue related to Iconis, which is offset in operating expenses. Turning to expenses. R and D costs in the Q1 totaled €70,700,000 compared to €106,100,000 during the Q1 of 2023. The 33% decline was largely tied to lower external development costs for TransCon Growth Hormone and TransCon PTH including a reversal of prior period write downs of pre launch inventories as well as oncology programs, partially offset by an increase in TransCon CNP costs. Sequentially, R and D costs declined 22%.

Speaker 3

SG and A expenses in the quarter totaled €66,800,000 essentially flat compared to €66,500,000 during the Q1 of 2023. Higher employee costs including the impact from commercial expansion was partially offset by lower external pre launch and administrative expenses. Sequentially, SG and A expenses increased 4%. Total operating expenses were €137,500,000 for the 1st quarter, a 20% decrease compared to the €172,700,000 during the Q1 of 2023. Sequentially, operating expenses declined 11%.

Speaker 3

Overall, our operating loss in the Q1 totaled €49,100,000 compared an operating loss of €143,700,000 during the Q1 of 2023 as a result of increased revenue and lower operating expenses. Sequentially, operating loss increased 34%. Finance expense in the quarter was €77,200,000 compared to €8,400,000 expense in the Q4 of last year. This higher finance expense was largely driven by a non cash derivative loss tied to our outstanding convertible notes. Quick comment on the balance sheet.

Speaker 3

As of March 31, 2024 due to amended IFRS rules which came into effect on January 1, 2024, you'll notice our convertible notes with face value of US575 million dollars are now reported as current liabilities even though they do not mature until April 2028, it would not require cash settlement in case of conversion by holders. IFRS still requires the carrying value of the convertible notes and associated derivative liabilities to be presented separately within current liabilities, which together total €622,000,000 As per IFRS rules, comparative amounts have been reclassified to reflect the change in presentation. The applied amendments had no other impact on the financial statements. We ended the Q1 with cash and cash equivalents totaling €320,000,000 And for the full year 2024 based on current plans, we expect Skytrophy revenue to be in the range of €320,000,000 to

Speaker 4

€330,000,000 to

Speaker 3

€340,000,000 at average 2023 exchange rates. We expect total operating expenses SG and A and R and D to be approximately €600,000,000 and we expect to be operating cash flow breakeven on a quarterly basis by the end of 2024. Let me now also highlight selected key milestones. For TransCon Growth Hormone, we now plan to submit an SBLA to FDA for adult GHD in the Q3 of 2024 compared to the previous plan of Q2 and we expect to report top line results from our Phase 2 Turner syndrome trial in the Q4 of 2024. For TransCon PTH, in the U.

Speaker 3

S, our PDUFA date May 14, 2024. If approved, we plan to launch it as UroLivipath as quickly as possible thereafter. Outside the U. S. With a commercial rollout of Jorvipath underway in Germany and Austria, we plan to rollout Jorvipath in our Europe direct and international markets segments throughout 2024 2025.

Speaker 3

For TransCon CNP, we expect to report top line results from the pivotal approach trial as well as submit our NDA for treatment of children with achondroplasia both in the Q4 of 2024 and also report week 26 top line data from the COACH trial in combination with TransCon Growth Hormone also in the Q4 of 2024. Within our oncology therapeutic area during the Q4 of 2024, we plan to provide a clinical update from the Phase 2 indication specific dose expansion cohorts from our TransCon IL-two beta gamma and TransCon TLR-seveneight agonist clinical trials. With that, operator, we are now ready to take questions.

Operator

Thank Our first question comes from Jessica Fye with JPMorgan. Your line is open.

Speaker 5

Hey guys, good afternoon and thanks for taking my question. I was hoping you could talk about how April looked for your UrbiPath in Europe and maybe elaborate a little more on some of those 1Q dynamics you mentioned for SKYTROPPA? Thank you.

Speaker 2

Should you start, Kartra?

Speaker 3

Yes. So related to the Q1 dynamics, what we saw for SkyTraff, I'll take the second part first. What we saw would be, I would describe as typical seasonality that you would see with other maturing launches where coverage updates at the beginning of the year led to insurance reauthorizations, copay resets and also led to a slightly different mix in the channel. And the our channel partners basically updated their inventories as appropriate. And it feels like those basically all cleared out by the end of February.

Speaker 3

And then on the first question related to the development of your UroPath in April, we continue to as we said, we will come out with an update overall in revenue later this year. But essentially, we're pretty happy with the results of the launch with the primary goals that Jens laid out in his prepared remarks.

Speaker 2

So just to give you a little bit of flavor from our own expectations, We launched KEYTROFA in the typical way that, sorry, you're perhaps inside the German market, Austrian market, up from this assumption that we're adapting to the German market. And what we typically will see and what we hope for was to get a broad prescriber base. The broad prescriber base, we expected them to take typical 1 to 2 patient on treatment. Then they get really the feedback, seeing really the unique benefit of this. And as soon as we're really getting over that, some comfort period where they really get the comfort, you take more and more on treatment.

Speaker 2

So when we saw when we got 25% of our target physician already in 8 week to make a prescription. I believe there were some of the most successful launch I have seen. We have not in ourselves imagined that. The number of patients we're seeing is around 100, meaning is that there is a few physician that started early in our ERP program or COPPA in Germany. Some of them are up on 2,030 patients already now because they already have building up discomfort on how really this product are giving unique benefit to the patients.

Speaker 2

So this is why we were really feeling this is really the start we had hoped for, the start where we're looking on the different KPI, it really turned out to what we wanted to see and what we hope for.

Speaker 3

And just to reiterate, the results are essentially sales direct to the patient. They go to the retail pharmacy, the patient picks up. There's no channel inventory with the Orbitz.

Operator

Thank you. Our next question comes from David Lebowitz with Citi. Your line is open.

Speaker 6

Thank you very much for taking my question. If you jump back about a year and when you were in the run up to your past potential approval for the regulatory setback. Could you juxtapose your current the current status of your sales preparation, what the market looks like now versus what it looks like then and how you see things similarly or differently?

Speaker 2

I believe we are in a much, much better place. First of all, the awareness of hypopara has been really being coming to a much better awareness level where physician and the patient. Just in the latest effort when if you really want to hear the patient voice was the U2 that is reflecting how the patient explains the unmet medical need that is in hyperpara to at the FDA advisory board. And I think that is only one element of it, why I believe we are in a much, much better place. And I think the realization of the unmet medical need we have already and always seen up from the patient, what I think the awareness of is on a much, much better level today.

Speaker 2

And I think this is a why when we see our success with the initial part of our launch in Germany, Australia. We know we can repeat that in all our EU direct as soon as we get fully reimbursed in these countries, mainly end of this year in the beginning of next year and doing 25. We see it already coming in for the international market, mainly to inpatient programs, which are patient driven program, physician driven program. So the general awareness about the unmet medical need in hypopara, I believe, had really, really gone to a stage where I hope we really can understand this unmet medical need to a much better way. One thing that also have driven is the new guidelines that came up from U.

Speaker 2

S. And other places, really describing how basic conventional therapy, basic not providing an optimal treatment, if you can call it a treatment. I would basic saying that it's a balance between treatment for short term symptoms and to avoid long term complications and you can never get that right. So it basically are in a position that the recommendation of PTH therapy is really the way to treat this disease, which are pretty obvious when you treat insulin diseases with not insulin like diabetes, yes, you will always treat them with insulin.

Operator

Our next question comes from Rosemarie Lee with Cantor. Your line is open.

Speaker 5

Hey, sorry, I should have put Lee Wasatch. I'm on for Lee Wasatch from Cantor. So just for TransCon PTH, in terms of payer access and formulary placement, have you had any pushback from payers on showing clinical benefits such as hospitalization rate or kidney benefit? Thank you.

Speaker 2

Is that question related to Europe or U. S?

Speaker 5

Both, if you may.

Speaker 2

Okay. I think in Europe, when we do make what we call the health economic impact of our treatment, we are integrating all aspects on how we addressing short term symptoms, the benefit of the patient to have a normalized life again, benefit of having a balanced calcium and more important, the quality of life these patients are experiencing immediately after starting treatment. The other part is what we call the long term risk. One part of the long term risk is what we call renal impairment, renal damage. That is all long term complication like cardiovascular impact that is calcification on lot of different organs, cataract and other things you're also achieving and will get with hypoPAR just dependent on time.

Speaker 2

All of this element is being built into our health economic evaluation that we are providing to each single European countries to be quite sure that we're getting the right reimbursement on right prices. So it's basically built on the European way of really being responsible in our pricing where we have an health economic calculation on how we basic are providing both a benefit to the patient, the benefit to really to the society in helping the patient on a reasonable, accessible cost.

Speaker 5

Okay. Thank you.

Operator

Thank you. Our next question comes from Joseph Schwartz with Leerink Partners. Your line is open.

Speaker 4

Thanks very much. I was wondering if you could talk about how many physicians in the U. S. Have had experience with UrovaPath in the EAP versus how many you'll be going out to de novo once it's hopefully launched commercially soon? And could you also talk about how many of the physicians who might be targets for using your VAPATH in their patients have you've been interacting with for SKYTROFA already?

Speaker 2

I think the first question was reflecting your plans and the U. S. ERP program. And what I can do, I can give you the general aspect of our ERP program. The general aspect of our U.

Speaker 2

S. ERP program because it's very, very different compared to what we had in Europe, which now have been stopped after we got our approval and got full commercial in Germany. Our ERP program

Speaker 4

in U.

Speaker 2

S. Is only addressing patients that really are PK's experience. Meaning is that it's a 3%, 4%, 5% subset of patient of hypopara that basically are electable to come into our ERP program. The ERP program is a very difficult program for basic large academic institution to handle. So because of contracting and the incentive for them is not high because it's not really a paid program for the physician.

Speaker 2

So therefore, you will always find a subset of this site that typically have a lot of patients in hyperparameter will not really be part of such an ERP program. It will only be more private site, small site. We are extremely enthusiastic about what we have seen with our ERP program and all the treatment benefit that we have observed in our clinical trial, both Phase II and Phase III, has been 100% confirmed what we have seen out from the ERP program and the patient experience there.

Speaker 4

Okay. And so how many new sites will you have to go to then that are that treat hypo parathyroidism patients who have not had experience in the EAP or with using SKYTROFA?

Speaker 2

SKYTROFA, we don't have any ERP site at all in this way. Skytrophy didn't ever had any ERP program running. So from that perspective, it's really hard for me to compare these 2.

Speaker 4

I meant commercial Gytropha or the EAP for YORVIPATH. I'm just trying to get a sense of the footprint now, how much overlap there is with between the two products that you'll have shortly?

Speaker 2

You will basically say that is not a ZARX overlap in directly to the decision prescription base because it's typical more specialist sites that take care of both sites. One of them is, for example, a pediatric first indication. The other one is an adult indication. So when you look at the overlap for that, that is there's not a large overlap. And Joe, I can I'm not seeing any data where I would indicate support and saying there's a big overlap.

Speaker 2

Where there is a great overlap, it's 80%, 90% of the commercial effort that basically are built on taking the same endocrinology product out in the same infrastructure. And what we basically are building, we are building independent sales forces that really will tailor make exactly the right physician to have the most prescribing physician being covered as fast as possible.

Speaker 6

Thank you. Thank you.

Operator

Thank you. Our next question comes from Gavin Carter with Evercore ISI. Your line is open.

Speaker 4

Hey, congrats on the progress and thanks for taking the question. I'm just wondering, out of the 100 YORVIPATH patients in Germany, how many of these were previously on NATPAR? And was this in line with your expectations heading in?

Speaker 2

I will say, surprisingly, we see many more naive patients that we actually have thought we will see. So this is one of the surprises we have seen that the physician basic are looking on the patients and saying is, we have so many on patients that not have been in treatment that we also are focused on taking the new patient into the treatment regime. And I have to say that was one of the surprises, at least for me, where I would believe that it was initial just a change of NAU patient, but we have seen many more naive patients that we actually expected. Great. Thank you.

Operator

Kelly Hsieh with Jefferies. Your line is open.

Speaker 5

Thank you for taking my questions. On SKYTROFO, could you provide your perspective into the competitive landscape given that 2 other long acting growth hormone commercial products out there and one of them if we check on the scripts actually approaching schedule for on both GRX and the NRX and also like you curious like whether your launch strategy remain the same?

Speaker 2

So one of the complications, at least I always have, is to utilizing the different databases you can get to get scripts. In this case, you can use this kind of data set is to look on trends, but never absolute levels because the sampling is really different between the different provider. So you are only sampling a small amount of the prescribing base of where you get the information from. So up from that perspective, you cannot really compare the absolute level. You cannot compare it also because when we talk about the unit, the unit is really different between the different product.

Speaker 2

So some unit is giving providing you that. So therefore, if you really want to get a really effective and most solid data, you need to take each single company, go into the line item and look on the revenue basis. This is where I get the best confirmation about data and how we see they are progressing. And there is no doubt when we look on that, for the growth hormone market, it was a very difficult Q1. And what we saw really have seen how we were the only one after reported data really have growing where everyone had declined, which you typically would see in the first

Speaker 7

quarter. Thank you.

Operator

Thank you. Our next question comes from Derek Archila with Wells Fargo. Your line is open.

Speaker 7

Hey, thanks for taking the questions. Congrats on the progress. So we have a question on the OpEx progression this year. I guess based on the 1Q OpEx, it seems like you might come in a fair bit below the €600,000,000 guidance. So just any color there on how we should be thinking about that?

Speaker 7

Thanks.

Speaker 2

I think Scott is so happy today because he's really getting a good question.

Speaker 3

Yes, Derek, thanks for the question. We've been pretty yes, no, we're pretty proud of the our ability to basically reduce OpEx pretty significantly while growing doubling revenue. It's not a bad thought, but as of now our guidance remains $600,000,000 OpEx for the full year based on current plans.

Operator

Thank you. Our next question comes from Paul Choi with Goldman Sachs. Your line is

Speaker 7

open. Good afternoon and thank you for taking my question. And also let me offer my congratulations on the progress. I want to turn maybe to CNP for a moment. And if you could maybe sort of comment on what your market research ahead of your top line results later this year, suggests in terms of how endocrinologists might be thinking about using TransCon CNP either as new patient starts or do you possibly anticipate switches from Voxogo just given the different dosing frequency and how you're thinking about what those kind of shares might look like in terms of the launch?

Speaker 7

And then could you maybe comment on what your sort of post approval requirements might potentially look like for TransCon CNP pending an approval down the road from the FDA? Thank you.

Speaker 2

It's really extremely interesting, Christian, from the perspective on when we're looking on all the research we have done because the key topic and the key feedback we're getting in all interaction with physician, caregivers, patient is addressing comorbidities. Being short is not a disease. But really to have this sign of comorbidities is really providing really the impact on having acontoplacia. And this is why we have so much strong focus really to address the comorbidity and also to address also linear growth. But the key objective of our program is to do address the comorbidities.

Speaker 2

We cannot avoid a primary efficacy endpoint is linear growth because it has been established from regulatory agencies both in U. S. And Europe. And it's really, really difficult to change that. If we have been the 1st, we will not have been linear growth.

Speaker 2

It will be addressing co morbidity as the fundamental of the disease. And this is how our integrated program has really been built up to show that in all our programs. And I think where we both address the linear growth, but also muscle weakness and other things like that. And when I see how we can already get the feeling about what it means for the patient is when we look on the quality of life questions that we have given to both patient and caregivers, how they really are adjusting the benefit of our treatment. And we also see that in our retentions.

Speaker 2

When we see the retention and our recruitment, we recruit our pivotal trial in less than 4 months. On-site, which have access to rosuartide have access to other treatment, it got recruited in 4 months. I've never seen that before. And this is because the physician could talk to the parents about the benefit they have seen, basic on providing our TransCon CNP product to them. Related to commitment afterwards, we have no comments or anything we have received from regulatory agencies in all the different places we have been and discussed the pathway for regulatory approval to any kind of commitment.

Speaker 7

Thank you.

Operator

Thank you. Our next question comes from Vikram Parrott with Morgan Stanley. Your line is open.

Speaker 7

Hi, thanks for taking our questions. We just had one on the pipeline. So a few months ago, you discussed with us a novel TransCon carrier platform and you cited your work here with semaglutide through a case study. I just wanted to see how your internal work with this novel platform and the GLP-one program is progressing and what the next milestones could be here that we could learn about? Thanks.

Speaker 2

We are extremely as excited about this lead candidate that we were for when we came out. There's no doubt the monastic treatment regime is the way to go. And we're building up the same fundamentals that we built up all our pipeline. I call it a pipeline because we basically are not taking the target engagement risk that you typically have when you make highly differentiated product because we're building 2 of the TransCon technology on an semitlotype molecule that basic had the broadest way to show clinical benefit everywhere. We are progressing with that.

Speaker 2

At the same time, we also have an intense discussion about how we basic are doing the best value proposition of this compound and this era of metabolic diseases because we're talking about metabolic diseases. And we will keep you updated as soon as we someway have made a decision what way we're going.

Speaker 5

Thank

Operator

you. Our next question comes from Yaron Werber with TD Cowen. Your line is open.

Speaker 5

Hey guys, this is Joyce on for Yaron. Thanks for taking our question. In the U. S, can you talk about your planned launch strategy for TransCon PTH in terms of the initial target population and prescriber base? And how much of it will mirror your strategy in Germany where you're initially targeting roughly 1 third of total population?

Speaker 5

Thank

Speaker 2

you. I think what we're doing now is to really integrating the learning from the first country where you're launching a product. And the first country we launched is in Germany, Austria, and we're getting a lot of good learning. I have to say, we basically got confirmation about how we should do it because we basically have seen everything what we have hoped for in this launch. So it's not like we're coming on with a completely different strategy.

Speaker 2

We are more proud about the commercial execution from our people in this region, how the basic made it exactly after what I call the playbook. So when I come to the playbook of U. S, that is always heading up, but committed as our Head of our Global Commercial Operation She's coordinating everything from U. S, coordinating into international market, correlating into EU direct, every place to be sure that we're building up the right strategic approach for basic have the optimal commercial launch on a global base. We will be here and we basic has seen really the huge interest.

Speaker 2

I can say we have the 1st commercial U. S. Product or person on commercial product for UroPAN is a person living in U. S. That took the consequence on the delay in the U.

Speaker 2

S. To basic flight to Germany and get the product out there. So we know it, we see it, we know Europads making really choose different for the patient, really get them their life back. And we really have seen how patient also see this benefit.

Speaker 5

Thank you.

Operator

Thank you. Our next question comes from Leland Gershell with Oppenheimer. Your line is open.

Speaker 8

Hi, good afternoon. Thanks for taking our questions. 2 from us. First, again, if you could just comment on the plan for rolling out your RiverPath in Great Britain following the approval. Is that something you're doing immediately or will there be any delay?

Speaker 8

And secondly, in the past you had mentioned hypochondriplasia is an indication that you were you've decided to not pursue. Just wondering, if the outlook might change following the data from the achondro Phase 3 that you'll be seeing later this year and or any information that may be coming out from the companies that are pursuing hypochondriacuria? Thank you.

Speaker 2

Yes. U. K. Is part of our Europe direct plan. And we have a rollout on all our countries that is in our EU direct and U.

Speaker 2

K. Is integrated in this rollout. So after we basic had the U. K. Approval, which are now separated from an EU approval like other countries that we also getting approval in now.

Speaker 2

We have continued fighting everywhere from the different countries. And for example, Australia, everywhere, where we also need an independent approval, we will then come in and go in and do what we call typical and filing of dossier that really are describing the health economic impact as we have done in many EU direct countries, but we're also doing in other countries. And when we have reached an agreement with the agencies in the specific country about the reimbursing price, your typical will come into what we call a full commercial launch. What we're doing in the meantime, until we have this full commercial launch, we're building up the infrastructure and we have done that in most of the EU direct countries, where we have a general manager, we have medical affairs, we have market assess teams, all that is basic already being established. And they are ready to be coming out and doing a full commercial launch that, for example, which we have done in Germany and Austria.

Speaker 2

In the meantime, physician patient can go to a name patient program, where we can get fully reimbursed patient with a build on an inpatient program or similar structures in a lot of different countries. In this time frame, ONCELLYO will be fully commercial, you basically can provide commercial reimbursed product to the patient to all these specific programs. So this is what we call the dual strategy we are utilizing in the Europe direct, which we also will some way implement in the international market, but we will not be the M and A holder in the international market if it typically be handed to our sales and distribution agents. For the other thing about hypo contemplation, it's really, really is an interesting aspect for us, because we are the only company that have the 2 cornerstone in growth disorders. Growth disorder is about 30, 40 different diseases.

Speaker 2

We are the only company to have a once weekly growth hormone, a once weekly CMP molecule with really the best in class properties for both of these two mode of actions. So what we are doing, and that is part of our vision 20x30, making an integrated strategy how we are going to be the leading company in growth disorder. And that is not only to address hypercontemplation, but address the other including the other surgery or other diseases that is in growth disorder. And we believe some of them will be best treated with growth hormone, Some of them will be best treated with CNP, but also many of them will potentially be integrated in a combination therapy. And this is why we believe why we can be positioned to be the leader in growth disorder because we have the 2 cornerstone for basic to handle all the 30 disease.

Speaker 2

So much more to come. When we come up next year, where we will come with our integrated strategy, how we're building up to be the leading company in growth disorders. Thanks.

Operator

Thank you. And our last question comes from Sushila Hernandez with Kempen. Your line is open.

Speaker 5

Yes. Thank you for taking my question. Could you elaborate on Sky Trover becoming a blockbuster in the U. S. Alone?

Speaker 5

What needs to happen? What are the key drivers? Thank you.

Speaker 2

The key driver is to continue what we're doing.

Speaker 1

This is

Speaker 2

exactly what we're doing. We are doing a continuum showing get the physician, the patient to really getting the treatment benefit of SKYTROFA as the best in class product opportunity inside the growth disorder. We will be helped by a lot of different things. The consolidation of the daily growth hormone market. We saw one of the major player is now stopping.

Speaker 2

We see all of the major player with daily growth hormone stopping. So the consolidation of the daily growth hormone where potential 1 or 2 player will be left in more or less in a cash segment It's really, really, really only the beginning of it. We're still in a small part of it. We believe we have less than 20% of the growth hormone deficiency, pediatric growth hormone deficiency. We can also grow it now in adult growth hormone deficiency.

Speaker 2

We have our TURNER trial coming in now. And then we also see where more and more experienced people get up the long acting, they realize there's only one choice. And I don't need to say that name because there's only the sky that really some way give the limit for this product.

Speaker 5

Thank you.

Operator

Thank you. There are no further questions. Thank you for your participation. This does conclude the program and you may now disconnect. Everyone have a great day.

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Earnings Conference Call
Ascendis Pharma A/S Q1 2024
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