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Intrinsic Value is all-important and is the only logical way to evaluate the relative attractiveness of investments and businesses.
Good day and thank you for standing by. Welcome to the Q3 2021 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. [Operator Instructions] Please be advised that today’s conference is being recorded. [Operator Instructions]
I would now like to hand the conference over to your speaker for today, Mr. Scott Smith, Senior Vice President and Chief Financial Officer of Ascendis Pharma. Please go ahead, sir.
Thank you, Operator. Thank you everyone for joining our conference call today. I am Scott Smith, Chief Financial Officer of Ascendis. Joining me on today’s call are Jan Mikkelsen, President and Chief Executive Officer; Jesper Hoiland, Global Chief Commercial Officer; Dr. Dana Pizzuti, Head of Development Operations and Chief Medical Officer; and Dr. Juha Punnonen, 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 U.S. commercialization and continued development of SKYTROFA for the U.S. market, 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 U.S. market potential of SKYTROFA 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, carryout 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 Statements section in today’s press release and the Risk Factors section of our most recent annual report on Form 20-F.
SKYTROFA was approved by the FDA in August 2021 for the treatment of pediatric patients one-year and older, who weigh at least 11.5 kilograms and have growth failure due to inadequate depletion of endogenous growth hormone.
Otherwise, please note that our product candidates are investigational product candidates and not 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 our product candidates shall be viewed as promotional.
On today’s call, we will discuss our third quarter 2021 financial results and provide further business updates. With 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. Jan?
Thanks, Scott, and good afternoon. Every quarter, I reflect on how the Ascendis team work together with patient and physician to satisfy major unmet medical needs. I also reflect upon the significant progress we had made towards our key development milestone to bring safe highly differentiated products to patient as fast as possible.
This quarter was special for Ascendis. It marks a defining moment for the company on our Vision 3x3 path to achieving sustainable growth as a fully integrated leading global biopharma company.
For years, we have worked to understand the reality behind growth hormone deficiency. We studied the science. We learn from previous successes and failures in trying to develop long-acting growth hormone therapies. And we work diligent in understanding the clinical data in pediatric and adult growth hormone deficiency.
There have been very limited innovation since daily injection of recombinant human growth hormone were introduced over 30 years ago and now Ascendis has the first FDA approved once-weekly product that offers pediatric growth hormone deficient patients, caregivers and physician, what they have been seeking for for the decades.
The approval of TransCon Growth Hormone in the U.S. is the culmination of determined efforts by us and our team. Over many years to bring the first TransCon technology product all the way from the concept stage through non-clinical and clinical development, manufacturing and device development and regulatory reviews, so we can finally go out to the patients.
This approval of SKYTROFA in the U.S., we believe we are just begun to unlock the potential of what we are seeking to accomplish with the TransCon technology platform and algorithm for product innovation, to develop highly differentiated products, addressing major unmet medical needs, which expected high development success, a real paradigm shift. The same excitements we have for TransCon Growth Hormone exist across our entire portfolio of our five independent clinical product candidates.
Why I am so optimistic? Because we believe the first FDA approved product is a strong endorsement for the TransCon technology platform. Our accolade for product innovation and Ascendis infrastructure and expertise in product and device development.
This is a reason why we are so enthusiastic about the prospects for our pipeline programs. TransCon PTH and TransCon CNP in endocrinology rare disease, as well as our oncology programs.
TransCon TLR7/8 Agonist and TransCon IL-2 β/γ. We believe the ability to build a sustainable robust pipeline and do not have the common challenge that many biopharma companies face in finding a second act to follow their first product, as we from the beginning, set out to build a sustainable pipeline in multi-therapeutic areas.
Approval in the U.S. is a major success for Ascendis. But we will of course not stop there. We expect to receive European Commission approval of TransCon Growth Hormone for pediatric growth hormone deficiency later this year or early next year.
We expect to report Phase III data for TransCon PTH in adult hypoparathyroidism in Q1 2022 followed by a planned U.S. FDA filing in mid-2022. Our research and development engine continues to produce promising new product candidate and we expect to continue the clinical development with additional regulatory submission in rare disease endocrinology, oncology and also in our third therapeutic are.
After we launched SKYTROFA in the U.S. in mid-October, we quickly received our first commercial order and first commercial patient on therapy. The Ascendis commercial team is executing on the long strategy, which is designed to the long-term value of our Growth Hormone product and laying the fundamentals for future product launches into the endocrinology rare disease area.
We attempt to create a global market leading brand based on demonstrating clinical benefit and strong value proposition for patients, caregivers, healthcare providers and payers. The TransCon Growth Hormone, we believe we can expand the global Growth Hormone market, while at the same time reducing the cost to society that result for suboptimal or lack of treatment.
I’m often been asked about our pricing strategy and what do we mean by premium responsible pricing. Our healthcare economic outcome also demonstrates that with a premium responsible pricing, TransCon Growth Hormone may deliver meaningful savings to the payer by potentially improving patient outcome compared to daily growth hormone.
Our payer resource covering 21,000 pediatric growth hormone deficient patients in the U.S. indicates only 20% to 30% of patient are adherence to daily injection therapy, and therefore, may experience suboptimal outcomes.
With this in mind, we have established a better price for SKYTROFA in the U.S. that takes into account expected milligram per kilo dose difference for SKYTROFA compared to daily growth hormone in real world practice.
Also, on an annual basis, the rack price is mid-to-high single percent higher compared to the leading daily growth hormone common product. This is what we mean by premium responsible pricing and can be considered a win-win for patient, the caregivers, physician, including the society.
We also understand the importance of U.S. market assist for patients and families and we continue to work with payers to make SKYTROFA as widely available as possible. Our priority is contracting based on the value of SKYTROFA rather than focusing just on volume. We are committed to building a long-term relationship with U.S. payers and health systems in the rest of the world as we working to bring our portfolio of products to the market in the future.
As we have highlighted before, Ascendis commercial leadership team is very experienced in endocrinology, especially in the growth hormone and PTH segment. They understand what it takes to create market leading brands. Our U.S. commercial organization is in place and we tend to cover around the 1,400 growth hormone prescribers. The pediatric growth hormone market in the U.S. is concentrated with about 80% of all prescription coming from about 20% of the prescribers. In parallel, our experienced U.S. medical affairs team has been out in the fields creating awareness with key opinion leaders and pediatric endocrinologist.
A key component in the SKYTROFA launch in the U.S. has been the successful introduction of Ascendis Signature Access Program. It has several different elements to it. But it is basically a personalized patient support program. With each patient assigning a nurse dedicated to working with them and their families, caregivers and physician from decision to treat through long-term therapy adherence. The program offers a suite of service including, but not limited to prior authorization support, out of pocket assistance and training on the SKYTROFA [inaudible].
As you know, as I say, industry we always think globally. And so let us turn now to our clinical development programs for growth hormone in other parts of the world. This week, the European Medicines Agency Committee for Medicinal Products for Human Use or CHMP has TransCon Growth Home on their agenda.
We believe we are on track to obtain a positive CHMP opinion followed by an expected European Commission approval for TransCon Growth Hormone by the end of the year or early next year.
In Japan, we continue to screen and enroll patients in the 40 subjects Phase III riGHt Trial for pediatric growth hormone deficiency and in China VISEN Pharmaceuticals completed enrollment of its Phase III clinical trial of TransCon Growth Hormone in children with growth hormone deficiency in March of this year.
Turning to potential label extension for TransCon Growth Hormone. We also have the global Phase III foresiGHt Trial underway for adult growth hormone deficiency. Looking ahead we expect to conduct additional trials to support an application for labeling extension beyond pediatrics and adult growth hormone deficiency.
Moving to TransCon PTH. Our excitement continuous around the clinical progress and prospect. Physician and patient story we have heard has so far indicated that TransCon PTH has the potential ability to transform patients. Given that 400,000 patients globally suffer from this and the lack of other treatment option, we believe TransCon PTH if approved, has the potential to be our largest endocrinology rare disease product opportunity. We think it could represent a market opportunity greater than US$5 billion.
In September, we announced the 58-week open label density data from the central lab reading in the PaTH Forward Trial, the global Phase II trial of TransCon PTH in adult subjects with hyperparathyroidism. These BMD data complement previous announced bone turnover data and demonstrated as expected continued normalization and stabilization of the BMD Z-score between 26 weeks and 58 weeks.
The BMD Z-scores parallel markers of bone turnover, which we believe is an indicator that the calcium metabolic in subject in our Phase II trial are normalizing over time, as expected when PTH concentration is restored to physiological levels.
We continue to have extremely high patient retention in the open-label extension study with 58 patients continuing in the trial as of November 2, 2020. I am also so pleased when I hear about how many patients are remaining in the open-label extension study.
This first time in my career that I have seen so strong patient commitment to taking a once daily injection and staying on therapy in an open-label study. We believe this is a potential indication of how TransCon PTH may be having a positive impact on short-term symptom as the quality of life.
We believe that FPGAs concentration is restored to physiological levels, it can improve short-term symptoms that could also translate into positive impact on long-term complications. We remain on track to report the 84-week Phase II data this quarter and we will expect to see similar trends as we saw with the 58-week data.
Globally, all three Phase III HP trials that are underway are progressing. PaTHway Japan, the Japanese Phase III trial is designed to enroll a minimum of 12 HP subject, but with China, the Phase III trial in Greater China has been imitated by VISEN Pharmaceuticals.
We are looking forward to reporting the topline Phase III results from the North America and European trials in adult HP patient in Q1 2022. If positive, our plan is to file an NDA in the United States for TransCon PTH in mid-2022 for adult HP. The strong data we have generated thus far reinforce our confidence in TransCon PTH as a potential first replacement hormone therapy in adults HP.
Now turning to transcon CNP. We continue to move forward with two Phase II double-blinded placebo-controlled studies in children with achondroplasia. The first Phase II trials, the ACcomplisH Trial is a dose escalation trial of 12 to 15 subjects in each cohort conducted mainly in North America, Australia and Europe. The second one, by VISEN Pharmaceuticals is ACcomplisH China trial, which is a cohort extension trial of at least 60 subjects conducted in China.
Completing two independent randomized placebo-controlled trial will give us 52-week clinical data on over 120 subjects with achondroplasia treated with TransCon CNP and over 25 subjects with placebo treatment. We believe these two blinded trial will be a strong indication of the potential safety and efficacy of TransCon CNP.
Later this quarter we will provide appropriate update on TransCon CNP, which will include a view of the biology and why we believe TransCon CNP has an unique product profile that is highly differentiated from other treatment in development.
We will provide an overview of the program involving the status of the dose escalation cohort, cohort expansion and safety update on the blinded data. We also plan to discuss target engagement to illustrate that TransCon CNP is doing what we expect it to do and we will give an update on expected timelines for data next year.
Now moving to oncology, we want to transform cancer therapy by improving a patient enter tumor outcomes by using TransCon systemic and intratumoral technology designed to provide sustained modulation of the tumor microenvironment and activating pseudo toxic immune cells.
We all know that developing effective and safe products has been a challenge within the oncology field. We believe by using the TransCon technologies and our algorithm product innovation to turn on the body’s anti-tumor immune system, we expect to improve patient outcome. This is how we are looking forward to making a real difference to the way cancer is treated.
Our oncology programs have made major investment this year. For our TransCon TLR7/8 Agonist program, we believe we have identified a product candidate that is highly differentiated compared to other product candidates by the medical extending the duration of release of an active immunotherapy compound inside the tumor.
With the TransCon technology, we are aiming to provide to purely efficacy inside the tumor for weeks and at the same time reducing systemic toxicity compared to what has been seen with direct injection of a parent doc alone into the tumor.
We believe the prolonged activation of the immune system inside the tumor for weeks will also generate and has global effect, killing similar tumor in other parts of the body. Our transcendIT-101 trial for TransCon TLR7/8 Agonist in advanced cancer patient is ongoing.
We have been dose escalating subject with TransCon TLR7/8 Agonist in the monotherapy model and we had initiated dose escalation with checkpoint inhibitor in that combination arm during the third quarter. We expect to have initial results from the monotherapy dose escalation for TransCon TLR7/8 Agonist by year end. We are presenting additional preclinical data for TransCon TLR7/8 Agonist at the Society for Immunotherapy of Cancer 36th Annual Meeting taking place in Washington, D.C. this week.
Also in the third quarter, we submitted an IND to the U.S. FDA to initiate the TransCon IL-2 β/γ clinical program. This is a Phase I/II clinical trial to evaluate TransCon IL-2 β/γ in patients with advanced cancer. TransCon IL-2 β/γ is a long-acting product using the same systemic TransCon technology as TransCon Growth Hormone and is designed to improve cancer immunotherapy by providing long-acting exposure without a heiGHt mix of highly open IL-2 then selected for the β/γ receptor.
The second oncology clinical stage product candidate is another sign of our commitment to solving unmet medical needs for patient by focus on design. IL-2 is a highly validated cytokine in the treatment of cancer. We have reviewed the data generated by us from the long list of IL-2 compounds and we believe TransCon IL-2 β/γ has the potential to be best-in-class and the first to fully solve the shortcomings of IL-2.
Looking ahead to the rest of 2021, the fourth quarter is shaping up to be [inaudible] with the potential to obtain positive CHMP opinion, the program update for TransCon CNP, first patient data on TransCon TLR7/8 Agonist and our 84-week data on TransCon PTH.
We plan to announce the update for these R&D programs on a virtual Research and Development Program Update call in mid-December. We will provide additional details in the coming weeks.
It was a great moment at Ascendis, achieving our first product approval in the U.S. It is worth reporting that pediatric growth hormone patients in the U.S. now have a new once-weekly treatment option.
What makes me even more happy is that I know we have the potential to help many more patients facing significant unmet medical needs. And as I said before, our goal at Ascendis has not just to get product approved, but to get products approved that can make a meaningful difference to patients and not just once but multiple times in multiple therapeutic areas.
Now, let me turn the call over to Scott for financial review before we open up for questions.
Thank you, Jan. Turning to our financial results for the quarter ended September 30, 2021. We reported a net loss of €80.3 million or €1.47 per basic and diluted share, compared to a net loss of €121.7 million or €2.31 per basic and diluted share during the same period in 2012.
Now let me run through some of the key components of these results. Research and development costs for the third quarter were €58.8 million, compared to €64.1 million during the same period in 2020. The decline in R&D costs was driven by a one-time benefits R&D costs.
As a reminder prior to FDA approval, we expensed pre-launch inventories of TransCon Growth Hormone as R&D costs. Upon FDA approval of SKYTROFA in Q3, €53.2 million was capitalized inventory as raw materials and work in progress, resulting in this one-time benefit.
Excluding this one-time item, the increase in R&D cost reflects continued advancement of our pipeline with the primary drivers including the following; an overall increase in personnel related costs and then for TransCon Growth Hormone R&D costs were higher due to investments to expand our future manufacturing capacity, as well as increased clinical trial related activities to support increased global clinical reach and label extension.
For TransCon PTH, R&D costs were higher primarily due to increased clinical trials [Technical Gap] spend, device development costs and manufacturing costs, including the completion of multiple drug substance PPQ batches, as well as initial cost of building commercial inventory.
For TransCon CNP, R&D costs were higher primarily due to increased manufacturing and clinical trial related costs.
And finally, for our oncology therapeutic area, R&D costs were higher due to increased manufacturing and clinical trial costs for TransCon TLR7/8 Agonist and also due to increased manufacturing and preclinical cost for TransCon IL-2 β/γ.
Selling, general and administrative expenses for the third quarter were €39.3 million, compared to €17.5 million euro during the same period in 2020. These higher expenses primarily reflect an increase in personnel related and commercial expenses, as well as IP systems and other infrastructure costs as we prepared to launch SKYTROFA in the U.S.
Finance, income and expenses in the third quarter include a foreign exchange rate gain of €21.3 million, compared to a foreign exchange rate loss of €39.6 million in the third quarter of 2020, primarily related to translation of our U.S. dollar holdings of cash and marketable securities to euros.
We ended the third quarter with cash, cash equivalents and marketable securities totaling €929.9 million, which includes the net proceeds from our follow on financing completed in September. As of September 30, 2021, the company had 56,877,723 ordinary shares outstanding.
In November, subsequent to the end of the third quarter, we completed our previously announced $25 million share repurchase program in which we purchased 154,837 of our ADSs in the open market.
Turning to an update on our U.S. launch of SKYTROFA for pediatric GHD, product is currently available in the U.S. and we have shipped multiple orders thus far into our specialty pharmacy and specialty distributor network for both initial prescriptions and channel inventory stocking.
We are currently providing SKYTROFA for pediatric GHD patients through our dedicated patient support program known as the Ascendis Signature Access Program. Several insurance plans are providing initial coverage for SKYTROFA through the medical exceptions process. We are also in active discussions with U.S. commercial payers to broaden our access for 2022. And we are encouraged by the early enthusiasm surrounding the launch and we look forward to updating you on our progress in the coming quarters.
Turning to the remainder of 2021, key activities include for TransCon hGH, execution of U.S. SKYTROFA commercial launch activities, investment in expanding commercial manufacturing capacity to support anticipated future demand and continued execution in our ongoing Phase III clinical trials, including the enlighten long-term extension trial and the riGHt trial in Japan, both for pediatric GHD and the global foresiGHT Trial in adult GHD.
For TransCon PTH, key activities include continued execution of the Phase II PaTH Forward Trial, which retains 58 subjects in the open label extension, continued execution of the Phase III clinical program including the PaTHway Trial and the PaTHway Japan Trial and ongoing manufacturing of PPQ batches and initial activities to build commercial inventory.
For TransCon CNP, key activities include, ongoing manufacturing activities, including scaling up production, continued execution of our Phase II clinical program, which includes the two randomized double-blind placebo-controlled clinical trials in the achondroplasia, the ongoing ACcomplisH Trial and the ACcomplisH China trial, which is being coordinated through VISEN Pharmaceuticals.
And lastly, in our oncology therapeutic area, key activities include continued execution of the transcendIT-101 trial for our TransCon TLR7/8 Agonist and the IL-2 lead trial for TransCon IL-2 β/γ.
In addition to SKYTROFA commercial launch activities in the U.S., we expect other SG&A activities will include TransCon PTH prelaunch activities and continued investments in personnel, systems and infrastructure to support our rapidly progressing portfolio and growing organization.
As Jan noted, we have a lot happening at Ascendis. So let me now also provide an update on the remaining corporate milestones and other disclosed events. For TransCon Growth Hormone we continued to anticipate European Commission approval for pediatric GHD by early 2022, as well as completion of enrollment of the foresiGHt Trial in adult GHD by Q1 2022.
For TransCon PTH, we plan to provide an 84-week update for PaTH Forward later this quarter. We then expect to report topline results from our Phase III PaTHway Trial in the first quarter of 2022, followed by an expected NDA filing in the U.S. in mid-2022.
For TransCon CNP, we plan to provide a clinical program update in December, during the virtual R&D update, as Jan mentioned in his remarks.
And finally, for TransCon TLR7/8 Agonist, we plan to present initial monotherapy dose escalation data for the transcendIT-101 trial at our virtual R&D update.
With that, Operator, we are now ready to take questions.
[Operator Instructions] Your first question comes from the line of Jessica Fye from J.P. Morgan. Your line is open.
Hey guys. Good evening. Thanks for taking my questions. I have several a couple of commercial and a couple of pipeline. First, the commercial questions, when you talk about playing the long game with your SKYTROFA strategy, can you elaborate on what that means to you from a tactical standpoint? And second one on SKYTROFA is, how should we think about the net price per patient for SKYTROFA? Appreciate the commentary on the relative list price. But will net price end up similar to or different from existing products. And then I got a couple of pipeline follow-ups.
Thanks, Jess. Let me start and then I can turn it over to Jesper Hoiland. But first of all, when we think about the SKYTROFA, we think about a best-in-class product opportunity. An opportunity that have not been available for the patient here in the U.S. for more than 30 years, even if I can count on multiple hands, how many attempts that has been to develop, there have been market -- product on the market and we got taken away, we couldn’t provide the same endocrine benefit that you could achieve with daily growth hormone.
When we saw the labeling with our reflecting our clinical data, we are really, really thrilled to be in a position to provide such a benefit out to the patient and not only the patient, the physician, the caregivers and also the society.
So from our perspective is, when you have a best-in-class product opportunity, is not to go out and be coming into panic, we want to grow for long-term value of this product opportunity, because we believe is so unique with the benefit it provides to the patient.
And this is why we basically have defined what we call premium responsible pricing with our basic -- providing us with a benefit on all aspect from us, but also to the patient, the physician, caregivers and also the society.
And this is what we think and going back to your percentage, yes, we have an higher list price on that. But we’re also utilizing much less material per patient. At the same time, we can then say that, yes, there is a benefit still for everyone in -- we want really to come out with for the patient also related to that.
Then you can then think out from the tactical perspective is that, and Jesper, can comment to that. But I think it’s related to market assess where he do will write negotiations with the right people in the right manner to really build up a leading that with maximal value. Jesper, do you have more to comment on?
Yes. I mean, first, Jessica, I appreciate your question. On the tactical side, I can only say that we have hired in the team that has a deep knowledge into the growth hormone space. So everyone that works in the commercial team has a background in endocrinology, in growth hormone and all in PTH, everyone.
All of them on the commercial front line has won several of excellence events. And basically you can say, we have taken the learnings that the team has had over the years and put it together and said that’s the best plan that we can come forward with the ASAP program, the Ascendis program is truly standing out in the way that we’re approaching the market and the commitments that we are having.
So we are in process of implementing all the things, of course, in the middle of COVID and everything that’s going on in the marketplace. But, all-in-all, I feel very confident that we are on it right trajectory going forward.
On the net price that you asked for, basically as Jan is alluding to, and as you have also highlighted in your own survey on how we have set up the pricing. We have done it in a responsible way, but also certainly with a price premium.
We are in a class of its own, we are not in the -- you could say, the commodity market, a once-daily, we are truly the first in the once-weekly segment and we are going to segment our self into that, because it’s the area under the curve that count for us in terms of generating value for our investors and shareholders for the long-term.
Thank you, Jesper.
Okay. Great. And just a couple of pipeline questions if I could. Have you dosed patients in the ACcomplisH China study? And if not, when do you think that will happen? Is the plan still to use one dose in that expansion trial or could it be two dose levels? And to clarify, will we get timelines for data from these Phase II CNP trials at the December R&D Day? And then switching to oncology, what type of single-agent activity do you want to see for your IL-2 β/γ in the IL βelieγe trial to warrant moving forward?
That was a lot of question, Jess. Going back to start on the oncology related to that. First of all, let we take consideration, we have our resource and development update from oncology, it will be focused on the TransCon TLR7/8 Agonist and we expect at that event really to give you an update related to exactly what we believe that describes the uniqueness of this product opportunity with a complete different paradigm shift, how really to treat solid tumor by placing a compound inside the tumor that basic being released over weeks and generate a more active environment. And I think that is the data you can look forward to see when we come to our December meeting.
We will not present data from IL-2 β/γ, because we first starting dosing patient there, so we will actual need to take a little bit more time before we basically will give an program update to our IL-2 β/γ. We expect dosing there soon of the first series of patients and we really, really thrilled about this unique product opportunity.
TransCon CNP, we have also provide you an update and basically give you all the answers to your question in December month and we basically illustrate really the highly potential of this highly differentiated product TransCon CNP.
And we have three different rare disease endocrinology product and all of them are highly differentiated each of them to anyone us you have seen before. And I’m quite sure we can give you all the data that supporting that in the December meeting.
Great. Thank you.
Your next question comes from the line of Michelle Gilson from Canaccord Genuity. Your line is open.
Hi. Thank you for taking my questions. I guess, the first one, just following up on Jess’ questions on the launch. Have you guys settled on what metrics you will provide us to kind of track this launch and what will -- what should we be focused on in terms of what’s most important for tracking the progress here? And then, in your early discussions around the price, can you give us a sense of what the feedback from payers has been so far? And if they’re recognizing the best-in-class profile in some of the other aspects that are benefits that you described that justifies the premium responsible pricing? And then I have a couple on the pipeline as well.
I think we are coming to a stage where we’re just initiating our launch now. We are about first in the beginning of the launch now. And we are feeling that we as a responsible company to give you clear guidance, we will come out with guidance when we feeling that we basically have follows the launch period in such a manner that we are feeling confident that we can give you clear direction about how this launch is going.
What we see and getting from the commercial team is, I do not know how many KPIs stages on the launch, we follow them all of them. I have to say, it’s going far over my own expectation, which I actually always it was high. So, I feeling really, really confident, but when I see the team we have, I am not in doubt, but they are the best team ever had worked with in the growth hormone market and they will do it uniquely.
So when they come into 2022, Michelle, we will give you, when we feeling we are in a position that we are feeling confident that we can give you clear direction in what way it will go. But until now, I have been highly, highly positive surprised about the feedback we have seen for our launch strategy. But also work for a long time to get it ready. So, I think, that is basic, the way you really fit into success being extremely well prepared with the right people.
Okay. And on the pipeline, I guess, for CNP in December, will you give us an update on what the path forward is for TransCon CNP, in terms of kind of next studies and next steps for the program. And when we get an update on kind of what you’re thinking in terms of potentially a Phase III trial endpoint. I know that you’re running a natural history study that they will inform that. And then on PTH, you mentioned, I think that this is the person I referred you on this call, say that it’s a $5 billion opportunity. What are you looking to show in the Phase III? And maybe also from the Phase II open-label extension long-term data, that would get you confident that you can realize that $5 billion opportunity?
Let me start on CNP first. What we would like to give you an update about that already behind achondroplasia. And also how we explain a treatment with a continuous exposure of CNP, we will provide a complete different treatment received compared to a short-acting CNP.
Understanding that, we basically will give you the idea why we believe a long-acting CNP concept that providing a continuous exposure will basically change the treatment paradigm with CNP. And that is the -- that already we will explain more and more to you.
At the same time, we also would like to give you the uniqueness you see with such a compound that is continues be giving in the flat related to safety and target engagement. That basically are providing confident about the entire -- our entire understanding about the biology.
We did exactly what we did with growth hormone. Went down and studied and studied and studied and studied the biology about achondroplasia. Designed to target product profile that really address the unmet medical need there.
And that is the learning that we would like to give you as we have provided for you, why we believe, how we can address all endocrine benefit with growth hormone. And we explained that for years and years and we will start to do the same thing with how a sustainable exposure of CNP can provide a complete new treatment paradigm and that is the understanding we would like to give you.
So going back to PTH, did you ask me why I’m so comfortable, why I’m so bullish about PTH? Because when we look on the 58 patients, look on all the demographic background, looking about if you are coming from different genetic diseases from autoimmune, from post-surgeon, going and look on each skill burden and other things like that, we see a huge benefit for all the patients.
So for all the clinical data we are accumulating and all the clinical data I have seen, I cannot comment and give you any kind of guidance except that all the patients we have been treating have seen a unique benefit of the patient.
So from that perspective is, I believe all that patients that have chronic hypoparathyroidism, now we are addressing the adult segment with about 80,000 to 100,000 in the U.S., much more in Europe.
I’m not seeing any of them, not really, when we look on the clinical data that not supporting, that all of them will have a huge benefitted and that is not a small benefit, it is a benefit both to short-term and we hope by accumulating for the patient, we also can see that we basically can benefit on the long-term risk for this patient group.
Okay. Thank you guys so much for taking my questions. Congrats on launching SKYTROFA.
Thanks, Michelle.
Your next question comes from the line of Josh Schimmer from Evercore. Your line is open.
Hi. Thanks for taking the questions. The first one on SKYTROFA, can -- you’ve mentioned several thoughts of providing coverage. Can you provide some details in terms of what’s your co-pay and how many covered lives and where you expect that to be actually start 2022 and progress throughout the year? Second, you had mentioned starting trials to expand the SKYTROFA label beyond the pediatric and adult, what settings are you considering for those trials? And then last, for the PTH device and ultimately the CNP device, so they’re going to be similar to the SKYTROFA device or maybe anyway different? Thank you.
Thanks, Josh. Let me start from the back, because then I can still remember those thing. The device we are using, for example, in SKYTROFA and basic TransCon PTH is complete different device. Where we have in SKYTROFA, a single use room temperature stability capsule where it’s basically have sale raised or anything like that, giving the optimal for the patient, for the caregiver and everything you have room temperature stability, you use one cartridge every time you inject it and then you basically have field rates [ph].
It’s optimal, because it will come in some geographic region are highly regulated. If you go over to PTHs, we have implemented a system with our learning from diabetes, insulin and other things like that, how you can have that pre-fill liquid formulation at room temperature that giving optimal confident.
And this is basically is a multi-use pen device that look there most like an insulin pens, where you basic have dosing for about two weeks and then you start on a new pen. And we are basically providing three different pen strings with basic titration in each single pen system. Meaning is that, we are going over and accepting and what we also seeing in our trial that the patient need to be optimized for each of them.
So this is basic complete different thing. For about CNP, as the last question, I think you need to wait, what we’re doing with CNP, this is still a lot of opportunities we are working on now. But what we always will have been the focus is the patient. What is optimal for the patient, we did that to growth hormone, we also doing that for hypoparathyroidism. Going back to then, I missed, I have still growth hormone question or something between the growth hormone question? Okay…
It was formulary for SKYTROFA and then new indications for SKYTROFA?
New indication, we have and we are so lucky, just that we are molecule that was designed by liberating the same unmodified somatropin, you see in daily growth hormone and you’ll see also the same that is endogenous.
So when we go to label expansion they come with a complete different way to approach it. Because we basically are in a position that we know now from growth hormone -- pediatric growth hormone deficiency, how we basically are providing a unique treatment with all the same benefit that we have analyzed that you will see.
So when we go to our adult health, we will move to a different primary endpoint. We’re not looking on growth velocity, we’re looking about how we basically have a metabolic effect on truncal fat and other element that is basically is a metabolic effect, dependent on the penetration out into the tissue of it.
When we look out to the longest dissected, five,-six different indication where growth hormone approved. We are starting and Dana can talk about it, where we trying to find a new either can’t wait how we basically can get as many as possible indication in the fewest possible trial. But it’s just mainly being levered out from the idea that we are in a position that we have the mode of action. We have the active ingredient is basically coming from somatropin. Dana will you comments on it?
Yeah, Jan. I think one of the situations that we’re in right now is that for the biologic, we need to sort of establish that we know our compound behave the same way with these other indications as it does for pediatric growth hormone deficiency. And so we are in discussions with FDA about it.
In addition, we are pursuing other sort of policy options, that may enable sort of an expedited pathway for sort of 505-B2 like filings for biologics. And that isn’t available now, but the FDA has also recognized that’s a gap and we are sort of working to try to facilitate getting language into the new PDUFA reauthorization, which will be signed next year. But in the meantime, we are going to be working very directly with FDA to work through in normal process, how we can in an expedited way establish advocacy any additional indication.
Just as we said before, we committed to make a global leading brand and we know that it’s really doing the right investment in the right trial to support that we can build up this global brand and that is what you will see we executed on in the future. We are not stopping before. We’re the leading global brand. This is our aspiration because we have the best-in-class product.
Going back to your last question, and Jesper, can help me coming in, but what I will say now, Jesper, as he said before, he is working on a strategy related to market assess. But we also see patient coming into, as I said in my part of this, my prepared remarks, that we’re seeing patient being treated as a commercial patient today.
Well, that mainly coming up from the exception process, and obvious, exception process, this is patient which cannot really get the optimal treatment on daily, which we have known for years and years, twice is being developed. And we see this kind of patient now being actually getting approved for commercial patient to the exception process. And I -- just give me the confidence that we did the right thing really to develop this product. Jasper, do you have further comments?
Yeah. I mean, Josh, it’s still early days, but as you know, SKYTROFA is truly the first pediatric growth hormone coming on to the market. And we are in deep discussions with the PBMs of getting access and we will announce, as they will, when we get access. And as you know, is a little off season, while we are certainly talking to them and discussing with them and we are confident that we will be able to bring SKYTROFA to the patients.
And as Jan said, those we get in a situation where we get them of medical exceptions, we get that at full price and that you have to pay mind also when you look at the -- what is the average selling price going to be for SKYTROFA, which is going to be a premium pricing.
Thanks a lot, Jesper.
Thanks so much.
Your next question comes from the line of Alethia Young from Cantor. Your line is open.
Alethia?
Yes. Sorry. Yeah, sorry about that. I was on mute. Thanks for taking my question, here. So and I want to ask, I want to talk about really how, Scott must handle the blank piece there. I got you. So just a question on like, the potential commercialization in Europe, just talk a bit more about how those markets kind of compare? I guess, on a practical perspective as around that patients are treated, we know the difference of. So I just wanted to talk about that kind of versus the United States? And then when we we’re going into oncology again, have you guys kind of started thinking about given, I know you’ve thought about it. But are you kind of ready to give people kind of what the next maybe program or program could be there in December? Thanks.
Yeah. We are a European company, Ascendis Pharma is a European company. We know the European markets extremely well. We have been working in the European market. So what we basically are building up, we are building up our strategy for Europe. And the element of that is because is so diverse group of reimbursement system you need to deal with, some of them are fast and second most of them are slow.
So for some countries, they can take out more than two years basic to get the right reimbursement. So what we’re doing there, which I think is the key element I would like to give to you. We’re building on a pipeline perspective.
If you want to be successful in Europe, you cannot call with single product. I have seen European companies or U.S. companies going to Europe and spending €100 million and gaining €5 million to €8 million out in revenue, we are not going to do that.
And this is why we happen those of the European market, we will be extremely responsible what we distilled the build out of the European market in the way, where we believe we create the best patient cover, but at the same time also do it in a financially responsible manner.
And we will assume as we getting the expected approval in Europe of SKYTROFA, nearly at the same time having a readout of our Phase III HP. We basically will be the fundament for all European strategy. So you will see a strategy that is built on the pipeline approach, building on two hopeful status, key product opportunity, TransCon Growth Hormone, TransCon PTH and see how we basically penetrated.
PTH could be a huge risk product in Europe, because of the emphasize on long-term risk, how really to have a patient population that basically could be in a position that could really benefit. And the value of avoiding long-term risk, I think, is must better recognized in the European setting.
Because as I stereotype saying is that, 98% of all German stay in Germany rest of their life. Here you move from one insurance company to the other insurance company and it give you as complete different aspect about how to build a high value product opportunities even on, you can say a small lower price level and this is what you will see be implemented in our European strategy.
Well, last thing on the, I think we have two paradigm shift compound in oncology now. TransCon TLR7/8 Agonist and our TransCon IL-2 β/γ. I think you will be thrilled to see the data that will come out with here in the coming years for that. But as you said, we’re not. We’re still working on pipeline and building up the same strong pipeline we did in rare disease endocrinology with at least three product opportunities.
So you will see in the coming years, both new rare disease endocrinology products, you will see more oncology product, and perhaps, with many of you will be that thrilled about the time when we come up with our new.
We’re still building everything on the same way we have been successful in rare disease endocrinology, on the TransCon technology and our proven algorithm for product innovation, which really have shown how we can make highly differentiated product, with -- the same time to be successful, which I believe is a really true paradigm shift.
Awesome. Congrats.
The next question we have is from Joseph Schwartz from SVB Leerink. Your line is open.
Hi. I’m Joori, diving in for Joe. Thank you for taking our questions. Following up on a previous question on SKYTROFA, I know it’s early days, but how should we think about the adoption of the treatment naive patients versus the exception process? Have about the treatment-naive patients versus switch patients evolve now that you’re on, you’re in the market?
So after three weeks to four weeks, you don’t get new patient, because just with diagnose of a patient to be a true efficient patient takes time, it takes more than three weeks or four weeks. So what you basically will see, you will see a different stage where from obvious reason if you have a patient that is not doing well on data growth hormone is already been diagnosed with growth hormone deficiency. All the people, all the test has been done, but just not getting the right treatment outcome.
What we will see later on is expected moving over to a more and more new patient, because they go to the entire diagnostic pathway to get a confirmed diagnosis of being growth hormone deficiency. And this is why you see a different states and that is what we expect to see in the coming months.
Okay. That’s helpful. And my second question is on TransCon CNP. I understand that you’re going to give us an overview of the biology behind the program and some kind of blinded analysis. I’m wondering, are you doing this just for our benefit or why not just wait until both studies are done and unblind them both at the same time? And why is the study even blinded when the heiGHt Trial of core SKYTROFA was an open label study?
When we were running the Phase III trial of heiGHt, it was a blinded trial for one year two. So our heiGHt trial was blinded placebo -- no placebo controlled daily growth hormone as a comparison. So the registration trials, we had for SKYTROFA here in the U.S. was one year blinded trials where we look on the growth pattern for our growth hormone product. So it was also blinded.
And the question you’re raising is actually really, really interesting. Do we do it for you? Do we do it for us? No. I think we do it for the patients. We do it all from the perspective that we want you to give a comfort that we are progressing with the program, that can give a meaningful difference for the patient. This is why we’re doing it. At the same time, we can also give you that information, why we believe we are highly differentiated to anything else. This is exactly why we’re doing it.
Okay. Thank you very much.
At this time, we will now be closing the Q&A session. This concludes today’s conference call. Thank you all for joining. You may now disconnect.
Thanks a lot.