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spk13: Ladies and gentlemen, thank you for standing by, and welcome to the first quarter 2021 Ascendance Pharma Earnings Conference Call. At this time, all participant lines are in the listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you will need to press star then one on your telephone. Please be advised that today's conference is being recorded. If you require any further assistance, please press star then zero. I would now like to hand the conference over to your host, Scott Smith, Senior Vice President and Chief Financial Officer at Ascendus Pharma. Please go ahead.
spk12: Thank you, Operator. Thank you, everyone, for joining our first quarter 2021 financial results conference call today. I'm Scott Smith, Chief Financial Officer of Ascendus. Joining me on today's call is Yen Mickelson, President and Chief Executive Officer, Dr. Mark Bach, Senior Vice President of Endocrine Medical Sciences, and Jesper Hoylen, Global Chief Commercial Officer, Dr. Dana Pizzuti, Head of Development Operations, and Dr. Juha Poonanen, 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 alluded to, our progress on our pipeline candidates and our expectations with respect to their continued progress, statements regarding our strategic plans, our goals regarding our clinical pipeline, statements regarding the market potential of our pipeline candidates, and statements regarding our regulatory filings. These statements are based on information that is available to us today. Actual results or events could differ materially from those in the forward-looking statements. and we may not achieve our goals, carry out our plans or intentions, or meet the expectations or projections disclosed in our forward-looking statements, and you should not place undue reliance on these statements. Our forward-looking statements do not reflect the potential impact of any licensing agreements, acquisitions, mergers, dispositions, joint ventures, or investments that we may enter into or terminate. We assume no obligation to update these statements as circumstances change, except as required by law. For additional information concerning the factors that could cause actual results to differ materially, please see the forward-looking statement section in today's press release and the risk factor section of our most recent annual report on Form 20F. Please note that our TransCon product candidates are investigational product candidates and are not approved for marketing by the U.S. Food and Drug Administration, European Medicines Agency, or other foreign regulatory authorities. As investigational products, the safety and effectiveness of the TransCon product candidates have not been reviewed or approved by any regulatory agency, and no representations are made as to their safety or effectiveness for the purposes for which they are being investigated. None of the statements made on the conference call regarding our TransCon product candidates shall be viewed as promotional and or product commercialization. On today's call, we will discuss our first quarter 2021 financial results and provide a business update. 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?
spk07: Thanks, Scott, and good afternoon, everyone. Our goal at Ascentis is to create a leading biopharma company. by bringing highly differentiated product candidates addressing major unmet medical needs as fast and safely as possible to their patients. Beginning with our Transcon technology and algorithm for product innovation, we designed our Transcon Growth Hormone product candidate and developed it from idea stage through phase 3 pivotal trial. This year, we are seeing the results of our efforts as we are approaching the PDUFA date, June 25th for Transcon Road Hall. We held our late cycle meeting with FDA in April, in which FDA confirmed at that time that they are planned to meet the PDUFA action date. After the late cycle meeting, FDA conducted a BMU inspection at our Apollo Alto site over a six-day period with no 483 observation. Finally, we and our contract manufacturers have also submitted response to FDA's information request related to our contract manufacturing sites. We feel good about a potential U.S. approval for Transcon growth hormone for the treatment of pediatric growth hormone deficiency. We believe our ongoing labeling discussion with FDA are progressing well. Based on the latest feedback, we believe we will get a label that will support the scientific basis for Transcon growth hormone having a comparable mode of action to daily growth hormone. The great thing about retaining the same mode of action is that patients, physicians, and payers can rely on at least 30 years of scientific and clinical data to predict expected treatment results for overall endocrine health, including growth velocity. But getting an approval is just another important step in our plan to build a market-leading product. We are all aware of other long-acting growth hormone preparations that have been approved but not launched, or have been launched and then removed from the market. To have a market-leading product, We also have to provide a strong value proposition to patients, physicians, and payers. For patients, our once-weekly transplant gruptamol has in clinical trials shown safety, including hemogenic profile comparable to daily gruptamol. and superior outcome measured by analyzed high velocity in pediatric growth hormone deficiency compared to daily growth hormone. We believe our once-weekly Transcon growth hormone, administrated with an autoinjector in a room temperature stable presentation, will reduce the treatment burden for patients compared to daily growth hormone. For physicians, we believe our once-weekly transplant growth hormone may improve patient compliance compared to daily growth hormone, which potentially could lead to better outcome. And that physicians finally may have a growth hormone therapy for patients and caregivers that is associated with less treatment burden compared to daily growth hormone. Lastly for PEER, our recent US insurance claims and analysis data presented at ENDO 2021 demonstrated that the financial benefits of treating pediatric growth hormone deficient patients with growth hormone therapy and the dramatic need for improved adherence. We are continuing to execute on our global clinical reads and label expansion for Transcon growth hormone. In Europe, we expect a decision from the European Commission on our M&A for transcon growth hormone in the fourth quarter of this year. In Japan, we continue to execute on the phase 3 right trial for pediatric growth hormone deficiency. And in China, recent pharmaceuticals recently completed enrollment of his Phase III clinical trial of Transcon-Globetamone hormone in children with globetamone deficiency. We also have the global Phase III Foresight trial underway for adult globetamone deficiency to support label expansion for Transcon-Globetamone. And we continue to expect to complete enrollment of Foresight trial by late 2021 or early 2022. In summary, for Transcon Growth Hormone, we are aiming to bring a highly differentiated product to the patient. That can become a global leader in the growth hormone market and provide benefits to patients, physicians, and payers. Moving to Transcon PT8. We were excited about the 58-week results from the open-label extension of the phase 2 path forward trial in adult HP. Then we reported the 26-week data. We showed that we had a potential hormone replacement therapy that demonstrated normalization of serum calcium, normalization of phosphate, normalization of phosphate-calcium complex, normalization of urinary cancer. We also saw the major impact Transcon PTA treatment had on quality of life on the SF36 functional health survey. And we saw a trending towards normalization of skeletal remodeling. Coming to the 58-week results, the open-label extended trial is the first time to our notice that such a large group of HP patients has been exposed to physical levels of PTA for over a year. And it demonstrates how this treatment might help this patient group on a long-term basis. If I just sum up the 58 week results, what we saw was as expected. the durable response and well-tolerated safety profile that we have hoped for. All the elements of normalization of calcium metabolistic and homeostasis that we observed at the 26 weeks continued at 58 weeks, including normalization of 24-hour urinary calcium excretion. along with the initial rise of bone turnover followed by a decline trending to the mid-normal level. All this effect was observed in the absence of active vitamin D intake and the requirement for therapeutic intake of calcium supplements. What is remarkable and rewarding to me is that these 58 patients continue to participate in the Open Label Extension. I believe this is a reflection of the positive impact Transcon PTH is having on this patient's short-term symptoms and quality of life. I have never been part of a trial where we started with 59 patients and have 58 patients more than one year into an open-label extension. The patient demographic in our Phase II trial showed a very mixed population. Subjects suffering from post-surgical HP, idiopathic HP, autoimmune HP, some with kidney stones, renal insufficience. Some just recently diagnosed with HP and some with HP for more than 14 years. Some patients had mild pill burn and some had up to 8 grams of calcium supplement plus active vitamin D at baseline. Despite the wide variety of severity and disease background, 58 still continued in the open label extension. I believe the improvement in quality of life is driven by that. Looking at the adherence level, it was 99.8%, strongly suggesting that Transcon B PTAs is providing a benefit for the short-term symptom as the patient don't want to miss a shot. Everything I've seen with all the data suggests a patient benefit from Transcon B PTAs regardless of severity and disease background. We have seen that with the data, with the adherence, with the trial retention, and now we're also hearing the same message from the physician being part of the trial. Some physicians are now saying that all patients may benefit from Transcon PTH, not just because of biochemical controls, but because the patient's lives are improving. The patient can now become active again, can return to work. This is exactly why I see Transcon PTA addressing a major unmet medical need for the patient, but also for the society by enabling the patient to find active employment and work again. So when I look at the data, when I hear the stories from the patient, when I hear the stories from the physician, I feel so optimistic. I'm so hopeful that we really can help the more than 200,000 patients that are in the U.S., Europe, and Japan, and for the first time can potentially provide an hormone replacement therapy that normalizes their life for them. Not only based upon short-term symptoms, but also potentially on long-term complications. Later this year, in the fourth quarter, we expect to have the 84-week top-line OLE update from part forward. We also expect to report our top-line Phase III part-way trials results by the end of the year. We believe the data being generated supports a profile for Transcon PTA to be a potential first-line therapy for hypoparathyroidism. At the time of regulatory submission plan for the first half of 2022, we expect to have a solid data packet with both long-term 84-week data from our phase 2 part forward trial and 6-month randomized control data from the phase 3 part way trial. To achieve global clinical reach, earlier this month, we submitted a clinical trial notification to initiate a Phase III clinical trial, evaluating Transcon PTAs for adult HP in Japan. We look forward to sharing additional results later this year, as our goal is to make this important therapy available to HP patients worldwide as fast as possible. Turning to Transcon CMP, we are on track to provide an update on the clinical program in the fourth quarter of this year. As you know, we are conducting two randomized double-blinded placebo-controlled phase 2 trials in children aged 2 to 10. The first, the accomplished trial, is a dose-escalating trial of 12 to 15 subjects in each cohort conducted mainly in North America, Australia, and Europe. The second, run by Beeson Pharmaceutical, is the Accompanied China Trial, which is a cohort expansion trial of at least 60 subjects conducted in China. We are planning to keep the data blinded for one year for each cohort. Once completed, we will have a robust clinical data from two independent, randomized, double-blinded, placebo-controlled trials. To date, the safety is everything we have hoped for. There has been no injection site reaction and no indication of cardiovascular risk. Our top priority is to the acondalplasia patients in developing a safe and differentiated treatment option that address the comorbidity of the disease. Turning to oncology, we hit a major milestone late last year with our first IMD for our oncology division, which is also our first IMD delivering the Transcon hydrogel technology for sustained intertumoral delivery. We expect to initiate dose escalation in combination with a checkpoint inhibitor in July and to have initial results for monotherapy dose escalation for Transcon TLR78 agonist in the fourth quarter. For our second oncology program, we are on target to submit an R&D for Transcon IL-2 Beta-Gamma in the third quarter of 2021. Transcon IL-2 Beta-Gamma is designed for systemic administration via IV route and levers the Transcon systemic technology that you know from our endocrinology portfolio. As we are done in endocrinology, Our understanding of the biology has guided us in designing a compound with independently optimized receptor bias, potency, and pharmacokinetic to create that potential best-in-class IL-2 product. Summing up, starting with a product concept and now being less than one month away from our first VEDUFA action date, is a significant achievement. But as I mentioned when I started today's comments, we are not just trying to bring a product to a market. We are driving to solve major medical needs that we can meet by our Transcon technologies. We want to establish Ascendis as a fully integrated global biopharmaceutical company that has a portfolio of multiple independent products that make a difference in the life of patients. We have many important milestones to execute in 2021. I have never been more confident that we have the fundamentals in place to successfully building a leading biopharma company with multiple market-leading products in both endocrinology rare diseases and oncology. Now, let me turn the call over to Scott for a financial review before we open for questions. Thank you, Jan.
spk12: Turning to our financial results for the quarter ended March 31, 2021, we reported a net loss of 62.8 million euro or 1.17 euro per basic and diluted share compared to a net loss of 63.3 million euro or 1.32 euro per basic and diluted share during the first quarter of 2020. Now let me run through some key components of these results. Research and development costs for the first quarter were €88.1 million, compared to €57.5 million during the first quarter of 2020. R&D costs in the quarter reflect continued advancement of our pipeline, with the primary drivers of the increase including an overall increase in personnel-related and R&D infrastructure costs. And for TransCon Growth Hormone, or LONAPEG somatropin, Costs were higher due to buildup of pre-launch inventories, investments to expand our future manufacturing capacity, as well as increased clinical trial-related activities. As a reminder, we currently record all manufacturing related to Lona Peg Somatropin as R&D costs, including manufacturing of pre-launch inventories, which are immediately written down as R&D costs until we can demonstrate these values are recoverable. Since Lona Peg Somatropin is our first commercial product, we first redeemed these we first deemed these recoverable upon obtaining regulatory approval. Therefore, upon regulatory approval, we will reverse prior period write-downs of pre-launch inventories as a reduction to our R&D costs and recognize the cost of such material in our inventory. For Transcon PTH, costs were higher primarily due to increased clinical trial-related activities and manufacturing-related and device-related development costs, including production of PPQ batches. For Transcon C&P, costs were higher primarily due to increased manufacturing-related and clinical trial-related costs. And finally, for our oncology therapeutic area, costs were higher due to increased manufacturing-related preclinical and clinical costs, primarily related to Transcon TLR78 agonist and Transcon IL-2 beta gamma. Selling general and administrative expenses for the first quarter were 37.2 million euros compared to €17.9 million during the first quarter of 2020. These higher costs primarily reflect an increase in personnel-related IT and other infrastructure costs, as well as increased expenses in our commercial organization as we prepare for the launch of Lanipec Soma Tripin. Related to our investment in Visa and Pharmaceuticals, as previously disclosed, first quarter results included a one-time non-cash gain of 42.3 million euro as a result of Visa and Series B financing in January. Finance income and expenses in the first quarter included a foreign exchange rate gain of 34.2 million euro compared to a foreign exchange rate gain of 10.3 million euro in the first quarter of 2020, primarily related to translation of our U.S. dollar holdings of cash and marketable securities to euros. We ended the first quarter of 2021 with cash, cash equivalents, and marketable securities, totaling 771.1 million euro. And as of March 31, 2021, the company had 53,829,379 ordinary shares outstanding. Turning to the remainder of 2021, key items impacting expenses include the anticipated launch of Lanipeg Somatropin, advancing our endocrinology rare disease pipeline, expanding our activities in oncology, and investing in the TransCon technology platform, including for lanapeg somatropin, buildup of commercial inventory ahead of a potential launch, execution of commercial prelaunch and launch activities, investments in expanding commercial manufacturing capacity to support anticipated future demand, continued execution of the Foresight trial, a global Phase III randomized controlled clinical trial in adult GHD, and continued execution of the RITE trial, a Phase III randomized controlled clinical trial in pediatric GHD in Japan. For Transcon PTH, continued execution of the Phase II Path Forward trial, execution of the PATHWAY trial, a North American and European Phase III randomized controlled clinical trial in adult hypoparathyroidism, and an increase in manufacturing costs related to production of PPQ batches for anticipated regulatory filings in the first half of 2022. For Transcon-CNP, execution of the clinical program, which includes two randomized controlled Phase II clinical trials in achondroplasia, the ongoing ACCOMPLISH trial, in the ACCOMPLISHED CHINA trial, which is being conducted through our strategic investment in Beeson Pharmaceuticals. And lastly, in our oncology therapeutic area, execution of the TRANSCEND IT101 clinical trial of TRANSCON TLR78 agonist and advancing the TRANSCON IL-2 beta gamma program into clinical development. We expect other SG&A expenses in addition to Lonepeg-Somatropin commercial pre-launch and launch activities will include continued investments in personnel systems and infrastructure to support our rapidly progressing portfolio and growing organization. For 2021, we continue to remain on track for hitting our corporate milestones for Lonepeg-Somatropin with a PDUFA date Of June 25, 2021, we anticipate approval for pediatric GHD in the U.S. in the second quarter, followed by commercial launch in the third quarter, and we anticipate European Commission approval for pediatric GHD in the fourth quarter. For Transcon PTH, we expect to report in the fourth quarter top-line results for the Phase III pathway trial in North America and Europe for adult HP. and 84-week results from the open-label extension portion of the Path Forward trial. Transcon-CNP, we expect to provide a clinical program update in the fourth quarter. For Transcon-TLR78 agonist, we anticipate initiating dose escalation in combination with a checkpoint inhibitor in July for Transcend IT101, and we plan to present initial monotherapy dose escalation results for Transcend IT in the fourth quarter. And finally, for Transcon IL-2 Beta Gamma, we plan to submit an IND or similar filing in the third quarter. Before we open up the call for questions, I want to reiterate a few points about our anticipated commercial activities for Lanipeg Somatropin in 2021. We anticipate approval for pediatric GHD on the PDUFA date of June 25th. During the third quarter, we expect to have product available in the U.S. for pediatric GHD. During the third quarter, once product is available, we anticipate beginning to provide access to lanopaxomatropin for pediatric GHD patients by onboarding patients through our dedicated patient hub. And finally, during the fourth quarter, we anticipate European Commission marketing approval for pediatric GHD. We plan to provide guidance on the timing of the launch in Europe later this year. With that, operator, we are now ready to take questions.
spk13: Thank you. Thank you. As a reminder to ask a question, you will need to press star then one on your telephone. To withdraw your question, please press the pound key. We ask that you limit yourself to one question. Then you may rejoin the queue if you have any additional questions. Our first question comes from the line of Jessica Fye with JP Morgan. Your line is now open.
spk04: Hey, guys. Good afternoon. Thanks for taking my questions. I feel like you just said multiple times on the call that you're still expecting approval on the for TransCon growth hormone. But we're still getting a bunch of questions related to this being a drug device application. So on that note, I was wondering if you could talk about what interactions, if any, you're having with the device division of the FDA on TransCon growth hormone. And maybe is it possible to have a finalized drug label but still have outstanding issues that need to be resolved around the device?
spk07: Thanks, Jess. First of all, thanks for the question. What we are doing is actually what we always have been used to do in what I call endocrinology, is that you make a combination product, because that is how you basically always have been taking a unique differentiated product to the market. So when I'm thinking about the question about we getting about the device and other things like that, it's just, it's part of what we call a normal way to develop a product opportunity. And when I see the questions and when I see the interaction with FDA, the interaction with the device division, which we actually have for the last six, seven years with the design of all the factor study and other things like that. I actually feel that what I said before, I feel good. I feel we are in a place where we should be at that time. We have been through all what I call the necessary inspections. Now we're in the stage where we still have interaction with FDA, but it's more clarification on, for example, manufacturing sites where we will have questions which are typical will have been answered on an inside inspection. But it's actually getting answered now by what we call a normal email corresponding between FDA and us. So from the overall perspective, it's actually really nice to be in the position we are now because the labeling discussion has progressed as we have hoped for. Everything what we have seen is now and where we are in the process is where we expect to be if we some way should have talked about the optimal way to our PDUFA day from the day we filed. Dana, do you have additional comments to it?
spk11: No, I think you've really sort of laid it out nicely. And just, it's really just because it is a combination product, so you can't, you know, sort of, you know, get approval on one part of it without the other, right? So they go hand in hand. And as Yen said, we've been responding to all of the requests, and, you know, so have our manufacturers, including you know, drug substance and drug product manufacturers as well as the device manufacturers. So, you know, they've received what they asked for.
spk07: And don't forget, we are less than one month now. I think it's the 25th or 26th. Great.
spk13: Thank you.
spk07: Thanks, Jess.
spk13: Thank you. Our next question comes from the line of Michelle Gilson with Canaccord Genuity. Your line is now open.
spk03: Hi. Thank you for taking my question. You know, in your prepared remarks here, you mentioned that the labeling discussions are going well and you anticipate the label will support the scientific basis of trans-con growth hormone and its comparable mode of action to daily growth hormone. And I was just curious. I guess two questions sort of around these discussions. Do you anticipate that, you know, that comparable mode of action to daily growth hormone offers any, I guess, benefit in terms of your positioning in a formulary or, I guess, the ease of sort of interchangeability with daily growth hormone? And then are there ways in the language of the label that can highlight the differentiating aspects of trans-con growth hormone? you know, compared to maybe some of the other long-acting growth hormone in development, perhaps in, you mentioned immunogenicity and injection tolerability.
spk07: Thanks, Michelle. When I go to the mode of action, what I actually believe and what I feel most for is the patient. I feel that when I look directly onto the labeling text. From the Transcon growth hormone product, you released somatopine, which would be expected to have the same distribution as daily growth hormone, which we know is the same as industrial growth hormone, the same mode of action. I felt great for the patients because it gives me strong confidence that we are in a position that we can provide all the endocrine benefits that you have achieved with daily growth hormone. We can provide a treatment that basically are providing the same safety and what we have seen in our high trial in children with pediatric growth hormone deficiency, we also saw basically a better outcome. And the mode of action is basically the way you really decide the future of each single product opportunity. Because it's impossible later on to basically compensate away from the mode of action in the clinical data. And the specific question where we see it and The only way we basically can compare to, for example, if we look on labeling, for example, on an other long acting that got approved not far away, there was a clear discussion about also elements which are important related to the mode of action, how well it distributes in the body. And there is a complete different labeling text that indicate how it's restricted compared to what you see in daily growth hormone. And I think from a scientific basis also providing a good explanation when you look on the clinical outcome you get with such product opportunity. So that was why we designed from the beginning our Transcon Growth Hormone really to build on the last 25-30 years knowledge about safety and efficacy that you give by releasing an unmodified somatoprine, the same entity that you basically are having in dataglutamone and also as an endogenous product. And I see this is the benefit for the patient. And I think that it also goes to the physicians. It also goes to the payer because the payer will understand that we are providing a benefit for all the important endocrine health benefits that you expect to always to achieve by having daily growth hormone.
spk13: Thank you. Our next question comes from a line of Joseph Swartz with SVB Learning. Your line is now open.
spk05: Hi, I'm Jerry Dalian for Joe. Thanks for taking our question. I was wondering how you feel about consensus sales estimates for Transcon HGH. What is the best way for us to model the trajectory of the launch ramp? And are there any good analogs or factors that could influence adoption in this market that hasn't seen much new treatment advancements in a while?
spk07: Yeah, it's always an interesting question. because I take it from a little bit different perspective. When I, as I said in my script, we want to build up a global leading brand with Transcom Grow Tomorrow. Do we have the fundamentals for that? Do we have the fundamentals to be the leading brand in the US? Everything what I've seen out of this product opportunity. Transcon Growth Hormone, everything what has seen to the competitive landscape, give me a stronger and stronger belief Transcon Growth Hormone will be the leading brand in the Growth Hormone market. This is what is important for me. Go up and be the leading brand in the Growth Hormone market, not only in the US, but also in Europe, Japan, China, everywhere where we can be. How we come there? It's what I call an optimization of value creation and penetration. And I see product opportunity trying desperately to get fast penetration on the cost of value creation. We are here with a pipeline concept. We are here with multiple product that will come after each other with both label expansion. So my focus is basically not what we exactly will see of sales in 2022, but basically how fast, how well, how can we create most value out of Transcon growth hormone? And that is what we focus for in this end. And we will optimize value, we will optimize the penetration, and that is what we're building in.
spk13: Thank you. Our next question comes from the line of Josh Schumer with Evercore. Your line is now open.
spk10: Thanks for taking the questions. I have two. One that is on TransCon PTH. We've talked in the past about the need to shift the treatment paradigm to broaden the patients that physicians deem suitable for PTH replacement. and not just calcium and vitamin D supplementations. Maybe you can give us a sense as to how you feel that's evolving and progressing now that you have more of the TransCon PTH data in hand. And then second, and I'm sorry if I missed this, but I think on the last update, you had indicated that you were working on a new franchise for the TransCon platform, a new target. When might we hear more about what that franchise might look like? Thanks.
spk07: Thanks, Justin. You know, I have never had any doubt that transcom PTAs will be a true benefit for the patient. But honestly, it's not so much what I really believe that is important. What is important is can the physician see the same thing? Can the patient see the same benefit? And when we're looking at all the aspects of the data we are getting out from you can say biochemical data, quality of life, we see this huge benefit. When we hear the story from the patients, we hear the story how they're feeling to go back to a normal life, how they're feeling that it's a total change of everything what we have seen, and what we now also see that some of the key physicians that basically are treating patients with transplant PTAs also realizing this. How we can provide, not for a subgroup of HB patients, but basically for the entire diversified group of HB patients we have in our Phase II trial, where they're really independent on disease background, independent on severity of disease, all stay the same because all of them see the benefit and the physicians are recognized. We have seen physicians, and this is really interesting for me, for about half a year ago, they were saying perhaps we will take 30, 40, 50, 60%. What they're saying now, we will take all patients. And I think that is... Some way we believe that we need to have this communication out, but I believe it comes out now. I will have to take some time, but you're more and more patient getting treated with Transcon-PTAs, more and more, but fulfilled, and we'll see how our vision is for first time to make a hormone replacement for this big patient group is really getting fulfilled. You're right. In our Vision 3x3, we have three different therapeutic areas. We have executed on a late-stage pipeline now in rare disease endocrinology. We're starting clinical development now in this year. We have two clinical problems in oncology for multiple indications. But as we said, the Vision 3x3 is our strategic pathway how to make a leading, sustainable biopharma company. it will mean that we're going for the third indication. We are initially working on it now. We're defining different lead candidates now. And we believe that perhaps in late 22 or early 23, it will be good timing for us to come up with our pipeline and disclose that in our third therapeutic area.
spk10: Great. Thank you.
spk13: Thank you. Our next question comes from the line of David Lovewitz with Morgan Stanley. Your line is now open.
spk14: Thank you very much for taking my question. Once TransCon HGH gets approved, how do you expect payers to come online as the year progresses going into next year, and what do you expect the landscape ultimately might look like? Do you expect to go for broad coverage with a wide array of payers, or do you expect to be more selective in how you work with payers?
spk07: I can start with the initial answer, and then Jesper can follow up. But what we're doing is that, as I someway communicated before, we are on a pathway to develop a leading brand with our Transcon growth tomorrow. In building that, we also are building in such a manner that we are creating the value in a way where we're feeling we're optimizing the entire brand, because we need to invest in the brand. We need to invest in global reach, in label expansion, and that is what we're doing both adult growth hormone deficiency, and we will also have other indication, other trials we will do to really invest in building up this leading brand. What we're doing is that we have a state progress to make a global commercial reach. And meaning is that the first place we roll out is in the U.S. Then we have Europe, and then we have China, we have Japan. And then you can take the rest of the world. So from that perspective is that we also believe in a global price structure, global globalization, much, much, much more. So from our perspective is that we integrate all this and it is only a small step what we do initially, but what we basically are building up a model that basically are saying how can we build up the leading global brand specific in the US, if you only talk about that, a leading brand in the US and create most value. So we will optimize it in the best possible way and never starting to be saying is that we need to force ourselves in this fast rollout. We will be sure that the value of Transcon growth hormone will basically take in and serve you more and more patients with more and more physicians that see the real value we provide with Transcon growth hormone. Jesper, specific for US, will you further comment on it? Yeah, absolutely.
spk06: I mean, we are having a superior product, and of course we would like that to be reflected in the price that we are going to approach the market with. So just to keep that in mind, we will address the commercial market in the first place, as that is the biggest business opportunity for us. And as far as we see things, it is the value, that is the market leadership that we will be fighting for. So if you're saying we will not take each and every account into consideration in terms of giving it the broadest possible availability, we will certainly go for the value of an opportunity that we're certainly seeing with the donor-backed somatopene.
spk07: I think that's a great way to answer that, Jesper. It actually describes what we would do with each of our pipeline product opportunities.
spk14: Thank you for taking my question.
spk13: Thank you. Our next question comes from the line of Alethea Young with Cantor Fitzgerald. Your line is now open.
spk09: Hey, guys. Thanks for taking my question, and looking forward to the next month. I guess I just wanted to talk a little bit about, on the growth hormone side, kind of some of the socioeconomic, you know, kind of economic claims and kind of benefits to having kind of your product versus others and how you think about, you know, balancing that as you think about kind of, you know, kind of the price of the drug things.
spk07: So mainly the pharmacoeconomic calculation we have done is basic, not looking on different brands, but mainly focused on the benefit of being on a growth hormone treatment compared to not having the growth hormone treatment available or potential stopping earlier in a growth hormone treatment because of the high burden of a daily injection. And I think this is where we basically have done most of our claim analysis. When you compare different brands, I actually think that you should, for me at least, when I'm looking from thinking about the patient, the physician, and payer, you need to look at what you're providing of integrated benefit related to endocrine health, because this is where the social economic calculation takes in, because it's not only height, this is the entire way to having the right way to do remodeling of the body, meaning growth hormone, so much to be, need to distribute everywhere in the body. Cognitive effect, which I'm possible to measure in children, but we know growth hormone have cognitive effect, because there's also a lot of growth hormone receptor in the brain. All that is the part of why we believe it was so important for us that everyone could understand our mode of action is building on what we know a growth hormone daily treatment can give because we have the same active ingredients out there. And I think this is where we strongly believe in the benefit we can provide in endocrine health is really being correlated out to the same way, and I believe much, much better than you actually receive with daily growth hormone because we don't have the high treatment burden. And therefore, we will both get better adherence and then potentially lead to better outcomes.
spk13: Thank you. As a reminder, to ask a question, you will need to press star, then one on your telephone. Our next question comes from the line of Anita Deschamps with Berenberg Capital. Your line is now open.
spk02: Hi, good afternoon. Thanks for taking my question. So Jen, you talked about the preliminary 58-week result related to transplant PTH. Could you give us an idea about what to think about the 84-week data? Like what do you expect to see beyond just the safety data from there? Thank you.
spk07: Yeah, that is always a great question, what we expect to see. So let's go a little bit back. 36-week, 54, and then 84. It's going to be interesting because what we saw between 26 and 54, we saw the same durable response that we have hoped to see. So 26 to 54, pretty predictable. Really what we have expected to see. What was the unknown factor for us, which we didn't know, and it's going back to the scripted statement about its first time in history. that basic are a trial running for one year with 58 patients getting physiological PTH treatment and what we didn't know when we really will kick in the normal remodeling of the bone structure which basic everyone want to have if you go to non-surgical HB patient Basically, you can see this high-density structure is not healthy. It's actually providing larger fractures. What we wanted to see, that as an integrated part of our HB treatment with Transcon PTA, physiological PTAs label, we also restore the normal remodeling of the bone so we can make them healthier. And that we actually saw already started to kick in potentially a little bit faster than we actually would have expected. We saw with our 84 weeks that we basically saw the bone markers going down now, trending much more to the mid of the normal levels. And I actually think we will see that continue on the 84 weeks. And I actually think we will see the same with the bone density, that we're getting the normal bone structure back, and therefore you're much more going down to the set score of zero. So I think that will be the main difference. I think from the safety perspective, there was basically nothing between the 26 and 54. Same excellent safety perspective. So I think that is mainly what we will see. What we still are looking for is something like... But we already saw that already in the beginning because there was actually some patients that have already impacted a filtration rate in the kidney. And that actually turned out to be pretty good and restored. So I actually believe that what we saw with 26 and already now with 50, it's really moving into the same direction that we really are restoring the normal functional function by having a normal physiological PTH levels.
spk02: That's very helpful. Thank you.
spk13: Thank you. Our next question comes from the line of Tiago Falk with Credit Suisse. Your line is now open.
spk01: Thanks for taking the question. Just a quick follow-up on CNP. You alluded that you want to keep the data blinded. So what exactly could constitute the clinical program update in Q4? And what is currently your regulatory strategy, your view to the best of your knowledge on what that could look like for that program?
spk07: Thank you. This is a question that we actually spend so much time talking and discussion all the time. Because when we are in a situation where we have our dose escalation and its function what is easy to look is safety because you can do that on a total blinded way and if you start to see something potentially you should be starting to unblind to see if where it's coming from but what we have seen in this trial is everything as we have hoped for in a safe way, not even injection site reaction, not even any kind of cardiovascular risk observed any place. So it's really, really a safe treatment. So what we're doing now is the dose escalation. And we will continue to have each single cohort blinded for one year. What we're trying to do is to try to select one dose or two dose to a cohort expansion effect. And we hope to do that during this year. So when we come to the end of the year, we will give you an overview about the program, how we are progressing with all the different trials from the two different programs. you can say part or two independent phase two trial, we can give you in the best way, and this is where we have a continued discussion, what is meaningful to give without any way to compromise the blinded part of this. And this is what we're discussing now, and I'm quite sure we will have an interesting packet at the end of the year. Perfect. Thank you.
spk13: Thank you. Our last question comes from the line of Yanen Zhu with Wells Fargo. Your line is now open.
spk08: Hi. Thanks for taking my question. In terms of pricing of Transcon GH, I know you touched upon it from a strategy perspective. Just wondering, would we be looking at a price that is at a significant premium to daily? if you can clarify on that. And also, do you plan to access the other pediatric indications for the growth hormone market, like Turner syndrome and idiopathic short stature, which collectively could account for as much sales as the growth hormone deficiency indication? Thanks.
spk07: Relating your first question, Jesper came with a clear statement. We have a superior product. We will expect superior pricing. I think there was a clear statement from Jesper, which I say everything. To the second question, we are dedicated to global REITs label expansion. Global REITs is to go everywhere in the world. Label expansion, we already started on. We have our foresight trial, which are adult growth hormone deficiency. And currently now we're discussing what extra indication is we want to pursue in. And there is a series of interesting, you can say, indication we can pursue. And I think we have a great clinical team. that basically are coming up with a very, very smart clinical design perspective. So we really can do what we want to do, ensuring that it's a label expansion of our transplant growth hormone to build it up to the leading brand. And we will also go for the necessary indication to achieve that.
spk08: Got it. Great to know. Thank you.
spk13: Thank you. There are no further questions. Ladies and gentlemen this concludes today's conference call. Thank you for your participation. You may now disconnect.
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