Q1 2021 Ascendis Pharma A/S Earnings Call
[music].
Ladies and gentlemen, todays conference is scheduled to begin shortly please go and continue to standby. Thank you for your patience.
Operator: Ladies and gentlemen, today's conference is scheduled to begin shortly. Please continue to stand by. Thank you for your patience.
Unknown Speaker: and so on the other, and so much, and I'm going to be able to be. Thank you. Thank you.
[music].
Operator: Ladies and gentlemen, thank you for standing by, and welcome to the first quarter, 2021, Ascendant Farmer, Ernest Conference Call. At this time, all participant lines are in the listener.
Ladies and gentlemen, and thank you for standing by and welcome to the first quarter 'twenty 'twenty, 1 and Sundance Pharma on its conference call. At this time all participant lines are in a listen only mode.
Operator: After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you would need to press a star then one on your telephone. Please be advised that today's conference is being recorded. If you require any further assistance, please press a star rather than zero. I would now like to hand the conference over to your host, Scott Smith, Senior Vice President and Chief Financial Officer of Ascendus Farmer. Please go ahead. Thank you, Operator.
After the Speakers' presentation there'll be a question and answer session to ask a question. During the session you will need to press Star then 1 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 Edison Disarm. Please go ahead.
Thank you operator thank.
Scott Smith: Thank you, everyone, for joining our first quarter 2021 Financial Results Conference Call today. I'm Scott Smith, Chief Financial Officer of Assentus. Joining me on today's call is Yen Mikulsen, President and Chief Executive Officer; Dr. Mark Bach, Senior Vice President of Endocrine Medical Sciences; and Jesper Hoyland, Global Chief Commercial Officer. Dr. Dana Pizzuti, head of development operations, and Dr. Yuha Puninen, head of oncology
Thank you everyone for joining our first quarter 'twenty 'twenty, 1 and financial results conference call today.
I'm, Scott Smith, Chief Financial Officer of Us on this.
Joining me on today's call is Yun Michelson, President and Chief Executive Officer.
Dr. Mark Bock senior Vice President of Endocrine Medical Sciences, Yes.
Yes for Hoyland Global Chief Commercial Officer Dr.
Dr. Dana Pizzuti head of development operations.
And Doctor, you, Harpoon and and 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.
Scott Smith: 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 needed to, our progress with 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.
Examples of such statements May include but are not limited to.
Our progress on our pipeline candidates and our expectations with 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.
Scott Smith: 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.
Actual results or events could differ materially from those and the forward looking statements and we may not achieve our goals and carry out our plans or intentions or meet the expectations or projections disclosed and 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.
Scott Smith: For additional information concerning the factors that could cause actual results to differ materially, please see the forward-looking statement section of 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 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.
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.
Please note that our transcon product candidates, our 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 F and effectiveness of the transcon product candidates has halved.
<unk> been reviewed or approved by any regulatory agency and no representation are made as to their safety or effectiveness for the purposes for which they are being investigated now and other statements made on the conference call regarding our transcon product candidates shall be viewed as promotional and or product commercialization.
Scott Smith: 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 Yan Mickelson, our President and Chief Executive Officer.
On today's call, we will discuss our first quarter 'twenty 'twenty, 1 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.
Yes.
Thanks, Scott and good afternoon, everyone.
Jan Moller Mikkelsen: Thanks Scott, and good afternoon, everyone. Our goal at Aides is to create a leading biopharmaceutical company by bringing highly differentiated pro-candidates, addressing major unmet medical needs as fast and safely as possible to the patient. Beginning with our Transcon Technology and algorithm for product innovation, we designed our Transcon Growth Among Product Candidates program and developed it from ideas days to Phase 3 Fivaldr. This year, we are seeing the results of our effort, as we are approaching the Dovet date, June 25th for Transcon.
Our goal at ADESA and this.
And Mr Graves.
Leading biopharma companies.
By bringing a highly differentiated product candidates addressing major unmet medical needs as fast and safely as possible to patients.
Beginning with our transcon technology, and aggregate and with our product innovation and <unk>.
Signed our transcon growth hormone product candidates.
And develop it from <unk> 2 phase III pivotal trial.
Yeah.
This year.
We are seeing the result of our efforts.
We are approaching the stupid date June 25th for Transcon growth.
Yeah.
Jan Moller Mikkelsen: We held our late cycle meeting with FDA in April, in which FDA confirmed at that time that they were planning to meet the PDUFA Action Day. After the late cycle meeting, FDA conducted a BMO inspection at our Apollo at Palo Alto site over a six-day period, with no 483 observations.
We have our lead target and meeting with FDA in April and which FDA confirmed at that time that our plan to meet that could do for action date.
Also on the late cycle and B T 50, and conducted at beam inspection at Pollo, and 2 side or what a 6 day period.
With no for a tweet.
Sure.
Finally, we and our contract manufacturers have also somebody just response to Fda's information quick Chris related to our contract manufacturing sites.
Jan Moller Mikkelsen: Finally, we and our contract manufacturers have also submitted a response to FDA's information request related to our contract manufacturing site. We feel good about a potential US approval for Transcon Groport hormone for the treatment of pediatric growth hormone efficiency. We believe our ongoing leaflet discussions with FD are progressing well.
Yes.
We feel good about a potential U S approval and transcon growth hormone for the treatment. Okay. That's a growth hormone deficiency.
We believe our ongoing leasing discussion with FDA.
And are progressing well.
Based on the latest feedback you believe would be the get a label that would support the scientific basis for transcon growth hormone, having a comparable mode of action 2 day to correct them.
Jan Moller Mikkelsen: Based on the latest feedback, we believe we will get a label that will support the scientific basis for Transcombe Growth to Mone, having a comparable mode of action to data growth. 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 endocrime, including growth velocity. But getting approval is just another important step in our plan to build a market-leading product.
But the great thing about reaching the same mode of action you start patients physicians and payers and Ken will die on the at least so ges of scientific and clinical data to predict expected treatment of sorts for all into crime.
And <unk> growth velocity.
But getting and approvals is just another important step in our plan to be and market leading product.
We are all aware off on the long acting growth hormone preparation that have been approved.
Jan Moller Mikkelsen: We are all aware of other long-acting growth hormone preparations that have been approved, but not for long, 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 patients. For patients, our once-weekly Transcon Gropthamo has shown safety and efficacy, including an emogenic profile comparable to data group terms, and superior outcome measured by analyzed high velocity in pedantic growth hormone deficiency compared to daily growth. We believe our once-weekly trans-concourthamone, amnestrated with an auto-injector, in a room-temperature-stable presentation, will reduce For Physicians.
But not dogs or have been low and didn't remove from the market.
To have a market leading products you also have to provide a strong value proposition to patients physicians and payors.
4 patients or once weekly transcon growth hormone has and clinical trials show safety.
Including genetic profile comparable to data growth.
And superior outcome may shows annualized height velocity in pediatric growth hormone deficiency comp year to date growth.
Do you believe or once weekly transcon growth hormone administrated with and ultra and kicked off.
Simple to stable presentation.
And would use the treatment burden for patients compared to vehicle.
For the physician.
Jan Moller Mikkelsen: We believe our once-week-trans growth hormone may improve patient compliance compared to daily growth hormone, which potentially could lead to better outcomes, and that physicians may finally have a growth hormone therapy for patients and caregivers that is associated with less treatment burden compared to daily growth hormone. Lastly, our recent U.S. insurance claims and analysis data presented at Endo 2021 demonstrated the financial benefits of treating pediatric growth hormone deficient patients with growth hormone therapy and the dramatic need for improved adherence.
We believe our once weekly transcon growth hormone may improve patient compliance compared to data Cove tunnel.
Which potentially could lead to bid on outcome.
And that physician finally may have coped hormone therapy for patient and care.
That is associated with less treatment boom comprehensive vehicle.
Lastly for you all.
A recent U S insurance claims and manage this data presented at <unk> into 2021 demonstrated that the financial benefits of treating pediatric kokomo deficient patients the kokomo therapy.
And the dramatic need for improved adherence.
We have continued to execute on our global clinical weeks and label expansion for transcon growth hormone and <unk>.
Jan Moller Mikkelsen: We are continuing to execute on our global clinical and label expansion for Transcon Growth hormone. In Europe, we expect a decision from the European Commission on our MNA for Transcon Grotamol in the fourth quarter of this year.
Europe, we expect a decision from the European Commission on our M&A for Transcon growth hormone in the fourth quarter on off this year.
Jan Moller Mikkelsen: In Japan, we continue to execute on the Phase 3 right trial for pediatric growth hormone deficiency. And in China, a recent pharmaceutical company recently completed enrollment of its Phase 3 clinical trial of Transcon Growth hormone in children, the treatment for growth hormone deficiency. We also have the global phase three foresight trial underway for adult growthomone to support label expansion for Transconcotomo, and we continue to expect to complete enrollment in the foresight trial by late 2021 or early 2020.
And Japan, we continue to execute on the phase III trial for pediatric growth hormone deficiency and in China leasing Pharmaceuticals recently completed enrollment of these phase III clinical trial of transcon growth hormone.
And children the guacamole deficiency.
We also have a global phase III foresight try on the way for adult growth hormone deficiency to support label expansion for Transcon comfortable and we continue to expect to complete enrollment of foresight trial by late 2021.
Early 2022.
Yeah.
Jan Moller Mikkelsen: In summary, for Transgrown Glotamome, 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, patients, physicians, and payers. Moving to TranscomPET.
In summary for transcon growth hormone VR gaming too highly.
Highly differentiated product to the patients that can become a global leader in the crop come on market and provides benefit patient and physician and patients.
Moving to Transcon PTH.
We were excited about the 58 week with soldiers on the floor.
Jan Moller Mikkelsen: We were excited about the 58-week results in 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 cancer, normalization of foscel, and normalization of phosphate calcium com. Nominization of UNRIC.
And label extension on.
The phase II.
For what tried and adult HP.
When you reported between 2.6 week data vs.
So that would be.
Potential pulmonary replacement therapy that demonstrated normalization of C on personal normalization of phosphate and normalization of phosphate and types of complex.
Monetization of U K zone.
We also sold the major impact transcon, PTH and treatment path on quality of life on the.
Jan Moller Mikkelsen: We also saw the major impact Transcon PTA treatment had on quality of life on the SF 36, the Functional Health Survey, and we saw a trending towards normalization of skeletal rimar, coming to the 58 week result. The Open Label extended title is the first time to our notice that such a large group of HB patients has been exposed to physical levels of PTA for over a year, and it demonstrates how this treatment affects my health, this patient group on a long-term basis. If I just sum up the 58, we resolved it.
So as you see.
Functional SUV.
And we sold a trending towards normalization of skeletal remodeling.
Coming to the 52 week results.
And the open label extension trial.
East for the first time 2 hour notice, there's such a large group of HBV patients has been exposed to <unk> Davis on P. J.
I need demonstrates.
How this treatment by Hilton this patient group on a long term basis.
If I just sum up the 58 week results.
Jan Moller Mikkelsen: What we saw was as expected, the doable response and well-tolerated safety profile that we had hoped for; all the elements of normalization of Caton, Metabolistic, and Stasis that we observed at 26 weeks continued at 158, including normalization of 24-hour UNAIR calcium levels in cream, along with the initial rise of bone turnover, followed by a decline trending to the mid-normal. All this effect was observed in the absence of active vitamin D in and the requirement for therapeutic intake of calcium soap.
What we saw was as expected.
The group on response, and well tolerated safety profile Covid.
And that he had the hopeful.
All the elements of normalization on catch on <unk> and <unk>.
And in those spaces that.
That would be off suite at the 26 weeks continued at 52 weeks, including normally safe and all 24 hour you and Theyre Ketchum excretion.
Along with the initial and rise of bone turnover followed.
By a decline trending to that normal level.
Okay.
All this effect was observed in the option of active vitamin D intake.
And the requirement for 2 occurred intake of ketchup supplements.
What is remarkable and rewarding to me is that these 58 patients continue to participate in the olden day, but extinct.
Jan Moller Mikkelsen: What is remarkable and rewarding to me is that these 58 patients continue to participate in the open label. I believe this is a reflection of the positive impact transcompgis is having on these patients' 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 labor extension. The patient demographics in our phase two trial showed a very mixed population.
I believe this is a reflection of the positive impact Transcon PTH is having on these patients short term symptoms and quality of life.
I have some the opening part of and trial, where we started with 59 patients and have fun.
58 patients more than 1 year into an open label extension.
The patient demographics, and our phase II trial showed a bear and makes it population.
Jan Moller Mikkelsen: Subjects suffering from post-surgeal HP, Edopathic HP, Outermune, HP, some with kidney stones. We are not insufficient. Some patients were just reasonably diagnosed with HP and some with HP for more than 4 million years. Some patients had my pillopers, and some had up to 8 grams of calcium supplement plus active vitamin D at base.
Subjects suffering from HP.
HP.
HP, Alta and HP and some with kidney stones.
And sufficient.
I'm just recently diagnosed.
And some with HP for more than 40 years.
Some patients had my Pittsburgh and some had up to 8 grams of cut some supplement costs execute vitamin D at baseline.
Despite this day by their T of Cbot Ntt's back on 58.
Jan Moller Mikkelsen: Despite this wide variety of severity and disease background, 58 still continued in the open label, I believe the improvement in quality of life is true. Looking at the adherence level, it was 99.8%, strongly suggesting that Transcont PTSD is providing a benefit for the short-term symptoms, as the patient doesn't want to miss a shot. Everything I've seen with all the data suggests a patient benefits from Transgon B-Ptas, regardless of severity and disease background.
And the open label and these things.
I believe the improvement and quality of life history and good day.
Looking at here and stable.
It was 99, 8% strongly suggesting that transcon PTH is providing a benefit quarter short term symptom.
Ask the patients don't want to Miss a shot.
Everything I've seen bid all the data suggest that patients benefit from transcon PTH and Greg.
Regardless of the cbot.
And this is back on.
Jan Moller Mikkelsen: We have seen that with the data, with the adherents, with the trial retention, and now we're also hearing the same message from the physicians who are part of the trial. Some physicians are now saying that all patients benefit from transcompity, not just because of biochemical control but because the patient's lives are improving. The patient can now become active again, and can return to work. This is exactly YRC trans-conp-ch addressing a major unmetical need for the patient but also for society by enabling the patient to find active employment and work again.
We have seen that the data bits day here and the trial with tension and now we also hearing the same day says from the physician.
And part of the trial.
Some physician and I'll say that all patients may benefit from Transcon PTH.
Not just because of biochemical control.
Because the patient lives and improving.
Patient cannot become active again can return to work.
This is exactly why FC transcon PTH addressing a major unmet medical need for their patients, but also for the society by enabling the patient to find and execute employment and we're thinking.
And.
Jan Moller Mikkelsen: So when I look at the data, when I hear the stories from the patients, when I hear the stories from the physicians, I feel so optimistic, so hopeful that we can help the more than 200,000 patients that are in the US, Europe, and Japan and, for the first time, potentially provide formant repatisman therapy that normalizes their lives for them. Not only are they faced with short-term symptoms but also potentially with long-term complications. Later this year in the 4th Court, we expect to have the 84-week Tooulli update from part forward.
So when I look at the data when I hear the stories from the patient when I hear the stories from the physician IC and so optimistic so whole foods that we really can help the more than 200000 patients that are in the U S Europe and Japan.
And for the first time can potentially provide and performance with placement to IP that normalize their life with them.
Not only based upon short term symptom, but also puts into the non term complication.
Later this year and the full court, we expect to have the 84 week topline poorly update from path forward.
Jan Moller Mikkelsen: We also expect to report our top line phase three partway trials were sold by the end of the year. We believe the data being generated support a profile for transgun p-titis to be a potential first-line therapy for hyperparatyrus. At the time of regulatory submission planning 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 Triad and six-month randomized control data from the Phase 3 Partway trial.
We also expect to report our top line Phase III Parkway price was sold by the end of the year.
We believe the data being generated to support that profile for transcon PTH to be a potential first line therapy for hyperparathyroidism and.
At the time of regulatory submission planned 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 path forward try it and 6 month randomized control data from the phase III Parkway and trial.
To achieve global clinical rights and we do at this months be submitted a clinical trial and notification to uniti.
Jan Moller Mikkelsen: To achieve global clinical reads, earlier this month, we submitted a clinical trial notification to initiate the FH3 clinical trial, evaluating transconticts for adults HPE in Japan. We look forward to sharing additional results later this year, as our goal is to make this important therapy available to HIV patients worldwide as fast as possible. Turning to TransconCMP, we are on track to provide an update on the clinical program in the fourth quarter. As you know, we are conducting two randomized, double-blinded placebo control phase two trials in children aged 2 to 10.
Clinical trial <unk>.
Relating transcon PTH full adults HP in Japan.
And we look forward to share additional results later this year. Our goal is to make this important therapy available to patients worldwide as fast as possible.
Turning to Transco and CMP be on track to provide and update on the clinical program and the fourth quarter on this issue.
As you know we are conducting 2 randomized double blinded placebo controlled phase II trials and children, aged 2 to obtain.
The first the Companys trials and he said dose escalating trial.
Jan Moller Mikkelsen: The first the accomplished trial is a dose escalating trial of 12 to 15 subjects in the East cohort conducted mainly in North America, Australia, and Europe. Second, run by Beeson Pharmaceuticals, is the Accomptit China Trial, which is the cohort expansion trial of at least 60 subjects in but docked in China. We are planning to keep the data blinded for one year for each core. Once completed, we will have clinical data from two independent, randomized, double-blinded, placebo-controlled trials. To date, safety is everything we have hoped for. There have been no injections, and no indication of cardiovascular risk.
215 subject and each cohort conducted mainly in North America, Australia and Europe.
And second 1 by decent pharmaceuticals is the.
China trial, which is a cohort expansion trial of at least 60 subject.
Conducted and China.
We are planning to keep the data blinded for 1 year for each cohort.
Once completed we will have a robust clinical data from 2 independent randomized double blind placebo controlled trials.
To date, the safety is everything behalf hopeful there had been no injection site reactions and no indication of cardiovascular risk.
Jan Moller Mikkelsen: Our top priority is for the Acontoplasia patients in developing a safe and differentiated treatment option that addresses the comorbidity of the disease, turning to oncology. We hit a major milestone late last year with our first I and D for our oncology division, which is also our first I&D leveraging the Transcom Hydrogen Technology for sustained intertumal delivery. We expect to initiate dose escalation in combination with a checkpoint in Hibersol in July and to have initial results for monotherapy dose escalation for Transcont TLR 78 acrony in the fourth course.
Our top priority is to contemplate shifts patients and developing a safe and differentiated treatment option.
And with the cobalt b to C. After empty seats.
Turning to oncology.
The hit and major milestone.
Late last year, we'd all first R&D for all oncology Division.
Which is also our first R&D.
Delivering the transcon hydro technology for sustained into tumor delivery.
We expect to and you take dose escalation and combination with a checkpoint inhibitor in July and to have and the children for mono therapy dose escalation for Transcon T at a 7 and 8 equinix and the fourth quarter.
For our second oncology program.
Jan Moller Mikkelsen: For our second Oncology program, we are on target to submit an I&D for Transcon IL2 Beta Gamma in the third quarter of 2021. Transcon IL2 Beta Gamma is designed for systemic administration via IV route and leverages the Transcon systemic systemic technology that you know from our endocrinology portfolio, as we have done in endocrinology. Our understanding of the biology has guided us to designing a compound that independently optimized receptor bias, potency, and pharmacokinetics to create a potential best-in-class I2 product. Starting with a product concept and now being less than one month away from our first Fedufa action date is as significant as she is. But, as I mentioned when I started today's comment,
And on target to submit and R&D for Transcon, <unk> Karma and the third quarter of 2021 Transcon.
And to be to comment is the time for systemic administration.
IV route and lever the transcon and systemic technology that you know from our endocrinology portfolio.
As we have done in the commodity.
And.
Our understanding of the biology has guided us in signing a compound with independently optimized receptor bias.
Potency and pharma.
And to create that potential best in class 2 product.
Summing up.
Starting with our product concept and now being less than 1 month's every form out first could do foot 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 mark.
Jan Moller Mikkelsen: We are not just trying to bring a product to the market. We are driving to solve major medical needs that we can meet with our Transcon technology. We want to establish SENDIS 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 achieve in 2020. I have never been more confident that we have the fundamentals in place to successfully build 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 a question.
We are driving to solve major unmet medical needs that we can meet by our trends on technologies.
We want to establish <unk> as a fully integrated global biopharmaceutical company that has a portfolio of multiple independent product that make a difference in the lives of patients.
We have many important milestones to execute and to execute on in 2021.
I have never been more confident that we have the fundamentals in place to successfully building and <unk>.
<unk> Biopharma company with multiple market, leading products in both endocrinology rare diseases and oncology.
Now, let me turn the call over to Scott for the financial review before before well Krish.
Thank you.
Scott Smith: Turning to our financial results for the quarter-ended March 31, 2021, we reported a net loss of 62.8 million euros, or 1.17 euros per basic and diluted share, compared to a net loss of 63.3 million euros, or 1.32 euros per basic and diluted share, during the first quarter of 2020. Now, let me run through some key components of these results.
Turning to our financial results for the quarter ended March 31, 2021, we reported a net loss of $62.8 million euro or $1.107 euro per basic and diluted share compared to a net loss of $63.3 million euro or 1320 per basic and diluted share during the first quarter.
2020.
Now, let me run through some key components of these results.
Scott Smith: Research and development costs for the first quarter were 88.1 million euros compared to 57.5 million euros 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 to R&D infrastructure costs. And for Transcon Growth Hormone or Lanopacosomatopan, costs were higher due to the build-up of pre-launch inventories, investments to expand our future manufacturing capacity, as well as increased clinical trial-related costs. As a reminder, we currently record all manufacturing related to Lanapag 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 Since Wanapeg Somatropin is our first commercial product, we first redeem these; we first deem these recoverable upon obtaining regulatory approval.
Search and development costs for the first quarter were $88.1 million euro compared to 57.5 million and Europe during the first quarter of 2020.
R&D costs and a corp for the quarter reflect continued advancement of our pipeline with the primary drivers of the increase including and overall.
And personnel related and R&D infrastructure costs.
And for Transcon growth hormone Orlando summit drop and costs were higher due to buildup of prelaunch inventories investments to expand our future manufacturing capacity as well as increased clinical trial related activities.
As a reminder, we currently record all manufacturing related to the <unk> somewhat <unk> as R&D costs, including manufacturing and prelaunch inventories, which are immediately written down this R&D cost until we can demonstrate these values on recoverable.
Since <unk> is our first commercial product we first redeem these.
We first day and nice recoverable upon obtaining regulatory approval and therefore upon regulatory approval, we will reverse prior period write downs on prelaunch inventories as a reduction to our R&D costs and recognize the cost of such material and our inventory.
Scott Smith: 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 PPQBeth. For Transcon, 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 pre-clinical and clinical costs, primarily related to Transcon TLR7-8 agonist and Transcon IL2 Beta Gamma.
And for Transcon PTH costs were higher primarily due to increased clinical trial related activities and manufacturing related and device.
And related development cost, including production of <unk> batches.
And for Transcon CMP costs were higher primarily due to increased manufacturing related and clinical trial related costs and.
And finally product for our oncology therapeutic area costs were higher due to increased manufacturing related preclinical and clinical costs, primarily related to transcon <unk>, 7 and 8 agonist and transcon IL 2 beta gamma.
Selling general and administrative expenses for the first quarter were $37.2 million euro compared to $17.9 million Euro during the first quarter of 2020.
Scott Smith: Selling general and administrative expenses for the first quarter were 37.2 million euros, compared to 17.9 million euros 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 Lanapak. Related to our investment in Vizan Pharmaceuticals, as previously disclosed, first quarter results included a one-time non-cash gain of 42.3 million euros as a result of Vizan's Series B financing in January.
These higher costs, primarily reflect an increase and personnel related and other infrastructure costs as well as increased expenses and our commercial organization as we prepare for the launch of <unk>.
Related to our investment and based on Pharmaceuticals, as previously disclosed first quarter results and included a onetime non cash gain of $42.3 million Euro as a result of based on the series B financing in January.
Finance income and expenses and the first quarter and included a foreign exchange rate gain of $34.2 million euro compared to a foreign exchange rate gain of $10.3 million Euro and the first quarter of 2020, primarily related to translation of our U S. Dollar holdings of cash and marketable securities to euros.
Scott Smith: Finance income and expenses in the first quarter included a foreign exchange rate gain of 34.2 million euros compared to a foreign exchange rate gain of 10.3 million euros 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 equivalence, and marketable securities totaling 771.1 million euros. And as of March 31, 2021, the company had 53,829,329,329 ordinary shares outstanding.
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 million 829379 ordinary shares outstanding.
Scott Smith: Turning to the remainder of 2021, key items impacting expenses include the anticipated launch of Lanapagosomatropin, advancing our endocrinology rare disease pipeline, expanding our activities in oncology, and investing in the Transcon technology platform, including for Lanopeg Somatropin, build up of commercial inventory ahead of a potential launch, execution of commercial pre-launch and launch activities, investments in expanding commercial manufacturing capacity to support anticipated future demand continued execution of the foresight trial, a global, phase three randomized controlled clinical trial in adult GHD, and continued execution of the Wright trial, a phase three randomized controlled clinical trial in pediatric GHD in Japan, for TransconPtH, continued execution of the Phase 2 Path Forward trial. Execution of the Passway Trial, a North American and European Phase 3 randomized controlled clinical trial in adult hypoparthiridism, and an increase in manufacturing costs related to production of PBQ batches for anticipated regulatory filings in the first half of 2022.
Turning to the remainder of 2021 key items impacting expenses include the anticipated launch of <unk> pharma trough and advancing our endocrinology rare disease pipeline, expanding our activities on oncology and investing and transcon technology platform, including for La and it takes so much.
And buildup of commercial inventory ahead of a potential launch.
Execution of commercial prelaunch and launch activities invest.
Investments and expanded commercial manufacturing capacity to support anticipated future demand.
Continued execution of the foresight trial, a global phase III randomized controlled clinical trial and adult ghd.
And continued execution of the right trial, a phase III randomized controlled clinical trial in pediatric Ghd and Japan.
For Transcon PTH.
<unk> execution of the phase III path forward trial.
Execution of the pathway trial, and North American and European Phase III randomized controlled clinical trial and adult hyperparathyroidism.
And an increase and manufacturing costs related to production and PTK batches for anticipated regulatory filings and the first half of 2022.
For Transcon CMP execution of the clinical program, which includes 2 randomized controlled phase II clinical trials and a contemplation.
Scott Smith: For TransconCNP, execution of the clinical program, which includes two randomized controlled phase two clinical trials in acondoplasia, the ongoing successful trial, and the successful China trial, which is being conducted through our strategic investment in decent pharmaceuticals. And lastly, in our oncology therapeutic area, execution of the Transcend IT 101 clinical trial of Transcon TLR7-8 agonist, and advancing the Transcon IL2 Beta Gamma program We expect other SG&A expenses, in addition to Lanpeg and Somatropin, commercial pre-launch and launch activities will include continued investments in personnel systems and infrastructure to support our rapidly progressing portfolio and growing organizations.
Ongoing accomplished trial and the accomplished China trial, which is being conducted through our strategic investment and beef and pharmaceuticals.
And lastly, and how oncology therapeutic area execution of the transcend and 101 clinical trial of Transcon <unk> 7 and 8 agonist.
And advancing the transcon IL 2 beta gamma program into clinical development.
Yes.
We expect other SG&A expenses. In addition to lot of peg somewhat Trump and commercial prelaunch 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 <unk> with a <unk> date of June 'twenty, 5.2020, 1 we anticipate approval for pediatric ghd and the U S and the second quarter, followed by commercial launch and the third quarter and we anticipate European Commission approval.
Scott Smith: For 2021, we continue to remain on track for hitting our corporate milestones for Lanapag Somatropin with a pedufid 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 TransconP-P-TH, we expect to report in the fourth quarter top line results for the Phase 3 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.
<unk> for pediatric ghd and the fourth quarter.
For Transcon Phd.
Transcon PTH, we expect to report in the fourth quarter top line results for the Phase III pathway trial, and North America, and Europe for adult HP and 84 week results from the open label extension portion of the path forward trial.
Transcon CMP and we expect to provide a clinical program update and the fourth quarter.
Scott Smith: TransconCNP, we expect to provide a clinical program update in the fourth quarter. For Transcon TLR-7 agonist, we anticipate initiating dose escalation in combination with a checkpoint inhibitor in July for Transcend IT 101, and we plan to present initial monotherapy dose escalation results for Transcend IT in the fourth quarter. And finally, for Transcon IL2 Beta Gamma, we plan to submit an I&D 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 Lantepag Somatropin in 2021. We anticipate approval for the pediatric Ph. D. on the Padufa date of June 25th.
For Transcon, <unk>, 7 and 8 agonist, we anticipate.
<unk> dose escalation and combination with a checkpoint inhibitor and July for transcend and 101.
And we plan to present initial monotherapy dose escalation results for transcend and the fourth quarter.
And finally for Transcon IL, 2 beta gamma we plan to submit an IND or similar filing and the third quarter.
Before we open up the call for questions I want to reiterate a few points about our anticipated commercial activities for a lot of take so much opened and 2021.
We anticipate approval for pediatric ghd on the <unk> date of June 25th.
Operator: 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 L'AXomatopin 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.
During the third quarter, we expect to have product available in the U S for pediatric ghd.
During the third quarter once the product is available we anticipate beginning to provide access to lawn and peg somatropin for pediatric ghd patients by on boarding patients through our dedicated patient hub.
And finally during the fourth quarter, we anticipate European Commission marketing approval for pediatric Ghd, and 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.
Thank you.
Operator: 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. 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 J.P. Morgan. Your line is now open.
As a reminder to ask a question you will need to press Star then 1 on your telephone to withdraw your question. Please press the pound key.
We ask that you limit yourself to 1 question and then you may rejoin the queue. If you have any additional questions.
Our first question comes on the line of Jessica Fye with J P. Morgan. Your line is now open.
Hey, guys good afternoon, and thanks for taking my questions.
Jan Moller Mikkelsen: 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 bedo-p-date for Trans-Kine 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 it is possible to have a finalized drug label but still have outstanding issues that need to be resolved around the device.
And I feel like you just said multiple times on the call that you're still expecting approval on the begins the day.
On transcon growth hormone.
But we're still getting a bunch of questions related to this being on trend device application and so on that note I was wondering if you could talk about what interactions. If any you are having with the.
Device Division and the FDA on Transcon growth hormone and maybe is it possible can have a finalized drug label, but still have outstanding issues that need to be resolved around the device.
Thank you thanks, Justin first of all and thanks for the accretion and.
Jan Moller Mikkelsen: Thank you, thanks, yes, first of all, thanks for the question. What we are doing is actually what we have always been used to doing 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 us getting about the device and other things like that, it's just part of what we call a normal way to develop a product opportunity.
Yeah.
What we are doing is acceptable and we always have been used to do in.
What are called in endocrinology is that Youll make a combination product because that is how you basic always have.
Been taking and unique differentiated product to the market. So we're not thinking about the question about do we gating about the device and other things like that it's just is part of what we call and normal way to develop a product.
Yeah.
Jan Moller Mikkelsen: And when I see the questions and when I see the interaction with FDA, the interaction with the device division, which we have had for the last six, seven years with the design of all the factor studies and other things like that, I actually feel that what I said before. I feel good. I feel that we are in a place where we should be at that time. We are going through all what I call the necessary inspections.
And when I see the Christians and when I see the interaction with FDA the interaction with the device to be sure and which we actually have.
For the last 6.7 years with the design of all the effect of <unk>, although things like that are active feels that what I said before I feel good.
So we are in a place where we should be at that time.
And we been to all viable and I called the necessary inspections now in this space, where we still have interaction with <unk>, but it's more clarification on for example, and manufacturing sites, where we will have accretion with a typical will have been and so on that insight inspection, but it's executing and so now.
Jan Moller Mikkelsen: Now in the States where we still have interaction with FDAA, but it's more clarification on, for example, manufacturing sites where we will have questions which are typical, will have been answered during an inside inspection, but they're actually getting answered now by what we call and the normal email corresponding between FDA and us. So from the overall split. perspective It's actually really nice to be in the position we are now because the labeling discussion has progressed, as we are hopeful everything we have seen is now, and where we are in the process is where we expect to be if we, in some way, should have talked about the optimal way to our PDFA day from the day we find Dana, do you have additional comments on it?
By what we call and normal email corresponding team.
And those.
So from the old but the perspective is it's.
Really nice to be and but position and pretty hard and now because the labeling discussion and has progressed. Thus we are hopeful everything what we have seen is now and we're behind but processes, where we expect to be if somebody <unk> have talked about the optimal weighted to our could do for day from the day be fist.
Dana do you have additional comments to it.
Dana: I think you really sort of laid it out nicely. It's really just because it is a combination product, so you can't 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 device manufacturers. So, know they've received what they asked for. And don't forget, we heard last time.
No I think you really sort of laid it out nicely.
Yes.
It's really just because it is a combination product so you can't sort of.
Get approval on 1 part of it without the other right. So they go and at hand and and.
As Ian said.
And we've been responding to all of the.
The requests and.
And so have our manufacturers, including.
Drug substance and drug product manufacturers as well as the device manufacturers. So.
They've received what they asked for.
And don't forget we and less time.
Lift and 1 month's no.
Jan Moller Mikkelsen: And don't forget, we are less than one month now. I think it's the 25th or 26th.
Specific to fiscal 2000 and seek treatment.
[laughter].
Great. Thank you.
Thanks Jess.
And.
Operator: Thank you. Our next question comes from the line of Michelle Gilson with Canticoregenuity. Your line is now open.
Thank you. Our next question comes from the line of Michelle Gilson with Canaccord Genuity. Your line is now open.
Operator: Hi, thank you for taking my question. You know, in your prepared remarks, you mentioned that the labeling discussions are going well, and you anticipate the label will support the scientific basis of transcon 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?
Hi, Thank you for taking my question.
And in your prepared remarks, you're on you you mentioned that the labeling discussions are going well and you anticipate a label the label will support the scientific basis, and transcon growth hormone and its comparable motive action to daily growth hormone.
And I was just curious.
I guess 2 questions sort of around these discussions do you anticipate day that comparable motive action to daily growth hormone. After offers any I guess benefit in terms of your positioning and a formulary.
Or I guess the ease.
And.
And sort of interchange ability with daily growth hormone and then are there ways and the language.
Operator: And then are there ways in the language of the label that can highlight the differentiating aspects of transcon growth hormone compared to maybe some of the other long-acting growth hormones in development, perhaps in the way you mentioned immunogenicity and injection tolerability?
On the label.
Highlighting the differentiating aspects of transcon growth hormone.
Compared to maybe some of the other.
And growth hormone and development, perhaps and you mentioned immunogenicity and injection tolerability.
Thanks Michelle.
When I go to the.
Jan Moller Mikkelsen: When I go to the Motor Factor, what I actually believe and what I feel most for is the I feel that when I looked directly at the labeling text from the Transcon. Gropdomoad, you release Somotropi, would be expected to have the same distribution as daily growth hormone, which we know is the same industrial growth hormone, the same mode of action. I feel great for the patients because it gives me strong confidence that we are in a position that we can provide all the end-crime benefits that you have achieved with daily growth hormone.
Mode of action.
What I actually believe and what I feel most of them is the patient.
Is that when on look directly onto the labeling.
It takes.
From the Transcon.
Coke demand pro Doc you released so much repeat.
What would be expected to have the same distribution as daily growth hormone, which we know is for statement and fastest growth from all the same mode of action.
Great for the patients because it gives me strong confidence.
That we are in a position that we can provide all of that into crime benefits that you have a sheet with daily Coke terminal.
We can provide a treatment that basic providing the same safety and what we have seen in.
Jan Moller Mikkelsen: We can provide a treatment that basic is providing the same safety and what we have seen in our high trial in Chile with Pediatrics. Growth hormone deficiency, we also saw 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 day. The specific question where we see it, And the only way we can compare it to, for example, if we look at labeling, for example, on another long-acting that got approved, not far away.
I'll walk high trial in children with <unk>.
Mike.
Growth hormone deficiency, we also sold basic beta outcome.
And the mode of action is basic the way you really decide the future of each single product opportunity.
Is it possible later on 2 basic compensate at weight from the mode of action in the clinical data.
And.
The specific question, where we seed.
And the only way, we basic and compared to for example, if we look on labeling for example on the other long acting Dakota approved not far away.
That was a clear discussion about also element.
Jan Moller Mikkelsen: There was a clear discussion about elements which are important related to the mode of action, how well it distributes in the body, and there is a completely different labeling text that indicates how it's restricted compared to what you see in daily growth mode. And I think from a scientific basis, also providing a good explanation when you look at the clinical outcome, you get the SOS product opportunity. So that was why we designed from the beginning, our Transcon Grotamone, really to build on the last 25, 30 years of knowledge about safety and efficacy that you give by releasing an unmodified somatropine, the same entity that you basically have in Dalygopterm and also as an endosious product.
Important related to the mode of action, how will it distributes in the body and.
And there is a complete different.
Lately and it takes that indicate how it's restricted compare to what you see.
And David Kokomo and.
And I think from a scientific basis also providing a good explanation when you look on the clinical outcome.
The source product opportunity.
So there was and why we designed from the beginning.
Our transcon growth hormone really to build on the last 25.30 years.
About safety and efficacy that you buy.
With leasing and unmodified so much would be the.
And the same entity that your basic having and.
And <unk> and also as and in DOCSIS product.
Jan Moller Mikkelsen: And I see this as a benefit for the patient. And I think that it also goes to the physician and goes to the pair because the pair will understand that we are providing a benefit for all the important end-crime health benefits that you expect to always achieve by having daily growth.
And I see just as that benefit COVID-19 patients.
And I think that is also go to the physicians.
And they also go to the payer because the COVID-19 understand that.
Providing benefit for all the important into crime.
And that you expect to always to achieve by having daily growth hormone.
Thank you.
Operator: Our next question comes from the line of Joseph Swartz with SVB Lerink. Your line is now open.
Our next question comes from the line of Joseph Schwartz with SBB Leerink. Your line is now open.
Operator: Hi, I'm Jerry Diling in for Joe. Thanks for taking our question. I was wondering how you feel about consensus sales estimates for Transcon HCH. What is the best way for us to model the trajectory of the launch ramp? And, you know, are there any good analogs or factors that could influence adoption in this market?
Hi, Andrew dialing in 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 on the smart at that conference and much more.
Jan Moller Mikkelsen: loans adoption in this market that hasn't seen many new treatment advancements in a while.
And that's in a while.
Yeah, it's always an interesting.
Jan Moller Mikkelsen: Yeah, it's always an interesting question because I take it from a little bit of a different perspective, as I said in my script. We want to build up a global leading brand with Transcom Global. Do we have the fundamentals to be that leading brand in the US? everything I have seen out of this product, Transconcone growth hormone, everything I have seen in the competitive landscape gives me a stronger and stronger belief that Transconcumon Grovetamone will be the leading brand in the Grovermone market.
Christian because ticket from a little bit different perspective.
When I.
As I said in my script.
We want to build up a global leading brand with transcon growth hormone.
Do we have the fundamentals for that day.
Do we have the sort of maintenance to be the leading brand in the U S.
Everything what has seen out of this product opportunity.
And on Kokomo, everything what has seen to the competitive landscape give me a stronger and stronger believe transcon growth hormone.
And the leading bank and the crop come on market. This is what is important for me go up and be the leading brand and the growth come on market not only and U S. But also in Europe, Japan, China everywhere, where we compete.
Jan Moller Mikkelsen: This is what is important for me, go up and be the leading brand in growth of our own market, not only in the US but also in Europe, Japan, China, everywhere where we can. How we get there is what I call an optimization of value creation and penetration. And I've seen product opportunity desperate to get fast penetration on the cost of value cream. We are here with a pipeline concept. We are here with multiple products that will come after each other, with both label expansion.
And how we come to you is what I call and optimization of value creation and penetration and IFC product opportunity trying desperately to get fast penetration on the cost.
Value creation.
We're here with our pipeline concept, we have here with multiple product overcome after each other but both labor and expansion. So my focus is basic not what we expected we will see office sales in 2020, 2 but basic how fast how well.
Jan Moller Mikkelsen: So my focus is basically not what we exactly will see in 2022, but basically how fast, how well, how can we create the most value out of Transcon growth? And that is what we focus on in this end. And we will optimize value. We will optimize penetration, and that is what we're building in.
How can be create most value out of transcon growth hormone and that is what we focus for <unk> and <unk>.
And we will optimize value, we would optimize that penetration and that is what we are building.
Yeah.
Thank you.
Operator: Our next question comes from the line of Josh Schumer with Evercore. Your line is now open. Thanks for taking the questions. I have two.
Our next question comes from the line of Josh <unk> with Evercore. Your line is now open.
And thanks for taking the questions I have 2.1 is on transcon PTH and and you know we've talked in the past about the need to shift.
Operator: is on Transcon PTH and Yon. We've talked in the past about the need to shift the treatment paradigm.
And the treatment paradigm to broaden and.
Operator: paradigm to broaden the patients that physicians deem suitable for PTH replacement and not just calcium and vitamin D supplements. Maybe you can give us a sense as to how
Patients that physicians deem suitable for PTH replacement and not just calcium and vitamin D. Supplementation. So maybe you can give us a sense as to how you feel that's evolving and progressing now that you have more on the transcon PTH data in hand, and then second and I'm sorry, if I missed this but I think on the last update.
Operator: That's evolving and progressing now that you have more Transcon PTH data in hand.
Jan Moller Mikkelsen: that you have more Transcon PTH data in hand, and then second, and I'm sorry if I missed this, but I think in the last update, you indicated that you're working on a new franchise for the Transcon platform, a new target. When might we hear more about what that franchise might look like?
And you had indicated that you are working on a new franchise.
And the transcon platform and new.
And target.
And when might we hear more about what that what that franchise might look like.
Thanks Jess.
No.
I've had nail.
Jan Moller Mikkelsen: I have never had any doubt that transcon p-tades will be a true benefit for the patient. But honestly, it's not some of what I really believe that is important.
And they will head and neck down that transcon PTH will be a true benefit for the patient, but honestly, it's not Thomas what I really believe that is important and what is important is Ken with physician see the same thing can be patient and see the same benefit and when.
Jan Moller Mikkelsen: What is important is whether the physician can see the same thing? Can the patient see the same benefit? And when we look at all the aspects of the data we are getting out of, 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 feel to go back to a normal life, how they feel that is a total change from everything we have seen.
And we looking on on that aspect of the day Savi.
Out from.
You can see 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 we are feeling that.
Coca chains of everything and what we have seen and what we now also see that some of the key physician.
Jan Moller Mikkelsen: And what we now also see, that some of the key physicians that are basic treating patients with transcon PTAs have also realized how we can provide not just for a soft group of HB patients but for the entire diversified group of HB patients we have in our Phase 2 trial, where the really independent on disease background, independent on severity of disease, all stay in because all of them see the benefit and the physicians are recognized. We have seen the physician and I, Just this is really interesting for me, for about half a year ago, we were saying, perhaps we will take 30, 40, 50, 60% What they're saying now, we will take all patience, and I think that in some way we believe that we need to have this communication out, but I believe it comes out now.
Physician that basic trading.
Patient with Transcon PTH also realizing this how we can provide not for a subgroup of patients for basic full day Tayo diversified group of HB patient, we have in our phase II trial, we have the really.
Independent on disease spectrum independent on severity of disease, all Stacy because all of them and see that benefit and the physician I'll recognize it we have seen physician.
And just this is really interesting for me for about half a year ago USA passed we would take 30.40 50, 60% what.
And what the Cigna and we would take old patients.
And I think that is some way and we believe that we need to have this communication now, but I believe it comes out now.
Jan Moller Mikkelsen: I will perhaps take some time, but you're more and more patients getting treated with transcontin PTAs, more and more with full-filled, and we'll see how our vision for the first time to make a hormonal patient for this big patient group is really getting fulfilled. You're right, in our vision, three by three, we have three different therapeutic errors. We have executed on a LAS-D-PAPL pipeline now in rare disease endocrinology. We're starting clinical development now this year, and we have two clinical problems in oncology for multiple indications.
So it takes some time, but you more and more patients getting treated they tried to compete more and more the fusin and bucy, how low vision is for first time to make and home and with patient and for this big patient group is really getting fulfilled.
Youre right in <unk>, we have 3 different so quota share we have executed on our late stage pipeline now and rare disease endocrinology.
Starting clinical development now and this year, we have 2 clinical program in oncology for multiple indications.
Jan Moller Mikkelsen: But as we said, the vision three by three is our strategic part way, how to make a leading sustainable sustainable. Biopharm a 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 a good time for us to come out with our pipeline and disclose that in our third therapeutic area.
As we said the beach and treat but is our strategic pathway and how to make a leading sustainable sustainable.
Biopharma company.
And it will mean that would be going for the third indication. We are initially working on it now is defined and different candidates now and we believe that perhaps in late 'twenty or early 'twenty, 2 and it will be good timing for us to come up with our pipeline and disclose that.
And Oh.
<unk>.
Operator: Thank you. Our next question comes from the line of David Lovewitz with Morgan Stanley. Your line is not open.
Great. Thank you.
Thank you. Our next question comes from the line of David Lebowitz with Morgan Stanley. Your line is now open.
Operator: 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 might ultimately 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?
Thank you very much for taking my question.
Once the transcon Hgh gets approved.
How do you expect payers to come on line.
As the year progressed and is going into next year and what do you expect the landscape ultimately like might look like do you expect to get Gulf of broad coverage with a wide array of payers or do you expect to be more selective and and how you.
Work with payers.
I can start with the initial and.
Jan Moller Mikkelsen: I can start with the initial answer and then Jasper can follow-off. But what we're doing is that, somehow, we communicated before. We are not part way to developing a leading Brandt without Transconcroft.
And then yes flow.
Can follow up but what we are doing is that site.
Summary.
Communicated before.
We are on a pathway to develop and leading.
With our transcon growth hormone and.
Jan Moller Mikkelsen: In building that, we also are building in such a manner that we are creating value in a way where we feel we're optimizing the entire brand because we need to invest in the brand. We need to invest in global reads in label expansion, and that is what we're doing both in adult growth hormone deficiency, and we will also have other indications, other trials we will do to really invest in building up this leading bank.
And billing that we also.
Building on and saw some banner that we.
And creating that value in a wait and be purely be optimizing.
The entire brand because we need to invest and the brand we need to invest in global rights.
Label expansion and that is what we are doing both in.
Co promote efficiency and we will also have other indications other trials via the too to really in bits and building up these leading banks.
Jan Moller Mikkelsen: What we're doing is that we have a state, progress to make a global commercial reach, and meaning that the first place we roll out is in the US. Then we have Europe, then we have China, we have Japan, and then you can take the rest of the world. So from that perspective, we also believe in a global price structure, global globalization, and much, much, much more. So from our perspective, we integrate all this, and it is only a small step what we do initially.
What we are doing is that we have.
States progress to make our global commercial reach.
And meaning is that we first place we rollout is in the U S than 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 local globalization must must must more so from our perspective is that we integrate all of this and this is only a small step, but we do initiatives, but what we.
Jan Moller Mikkelsen: But what we basic are building up a model that basic is saying, how can we build up the leading global brand specific to the US if you only talk about that? A leading brand in the US and create the most value. So we will optimize it in the best possible way, and never start to say that we need to force ourselves on this fast road out; we will be sure that the value of Transcon Growth Mode will basically take in and self-administer to more and more patients, with more and more physicians that see the real value we provide to Transcon Growth, Yesper, specific for the US. Will you further comment?
Basic and building up and models that basic.
How can we build off that.
Leading global brand specific and U S. If you're on you talk about that at leading brand in the U S and create most value. So we will optimize it and the best possible way and Newark, starting to be saying is that we need to fulfill sales at this fast Rudolph.
And we'll be sure that the VAT.
<unk> of Transcon growth hormone was basic ticketing and south you more and more patients with more and more physicians that see the real value will be provided with transcon growth hormone.
Specific to the U S with you so low comments on it.
Jesper: Yeah, absolutely. I mean, we are having a superior product, and of course, we would like that to be reflected in the price that we're going to approach the market with. So just to keep that in mind, we will address the commercial market first, as that is the biggest business opportunity for us. And as far as we can see things, it is the value that is market leadership that we will be fighting for.
Yeah, absolutely I mean, we are having a superior product and of course, we would like that to see reflected in the price and we are going to approach the marketplace.
And so just to keep that in mind, we will.
Address the commercial market and the first place is 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 share gains youre, saying, we will not take each and every account into consideration in terms of giving it the broadest possible.
Jan Moller Mikkelsen: 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 opportunity that we're certainly seeing with the don't affect Somatopin. I think there's a great way to answer, yes, but it's actually described what we would do east of our pipeline product operations.
And then and the ability we even showed any golf on the value of tradition definitely strength, you're seeing but is there.
And we're not so much a peak.
I take this as a great way to try and sort of.
And as <unk> described.
Describe what we do with each of our pipeline and product opportunities.
Operator: Thank you for taking my questions.
Thank you for taking my question.
Operator: Thank you. Now, our next question.
Okay.
Thank you. Our next question comes from the line of Alithia Young with Cantor Fitzgerald. Your line is now open.
Operator: Our next question comes from the line of Aletia Young with Cantor Fitzgerald. Your line is now open.
Operator: Hey guys, thanks for taking my question and looking forward to the next month. I guess I just wanted you 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 your product versus others and how you think about, you know, balancing that as you think about, you know, the price of the drug thing. So mainly, the pharmacoeconomic calculation we have done is
Hey, guys. Thanks for taking my question and I'm looking forward to the next month.
I guess I just wanted you to talk a little bit about on the growth hormone side on kind of the some of the socio economic.
You know kind of economic claims and and the benefits to having kind of your product versus others and how you think about balancing that as you think about kind of.
Kind of the price and the drought.
So mainly the Pharmacopeia economic calculation, we have done is basic non.
Jan Moller Mikkelsen: So mainly the pharmacoeconomic calculation we have done is basic, not looking at different brands, but mainly focusing 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.
Looking on different brands, but mainly focused on the benefit on being on a growth hormone treatment compared to not having the growth come on treatment available.
Retention stopping early on in our go to them on treatment because of the high burden of a daily injection and.
And I think this is where we basically have done most of our claim analysis when you compare different brand.
Jan Moller Mikkelsen: When you compare different brands, I actually think that you should, for me, at least when I'm looking at thinking about the patient, the physician, and the payer, you'll need to look at what you're providing, of integrated benefit related to endocrine health. Because this is the social economic calculations exam because it's not only high. This is the entire way to having the right way to do remodeling on the body, meaning growth hormone, so much to Cognitive effect, which I'm able to measure in children, but we know growth hormone has a cognitive effect because there's also a lot of growth hormone receptors in the brain.
I actually think that you shouldn't for me at least when looking from thinking about the patient physician and payer.
To look at what you're providing.
All integrated benefit related to into crime helped because this is aware.
And the social and economic calculation, because it's not only height.
This is the entire weighted to having the right way to do remodeling on the body being growth to move so much would be need to distribute everywhere in the body cotton to effect, which is possible to mission children, but we know kokomo and have confidence you would think because there's also a lot of growth hormone receptor in the brain.
Jan Moller Mikkelsen: 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 to the same. way, and I believe it is 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, which will then potentially lead to better outcomes.
All that is part of why we believe.
It was so important for us that everyone could understand our mode of action is building on what we note our growth to moan daily treatment can give because we have the same active ingredients out there and I think this is where <unk> strongly believe in the benefit.
We can provide and endocrine post is really being correlated to the same way and I believe must must better that you actually would see with daily growth hormone because we don't have the high treatment burden and therefore, we would both get better adherence and then potentially lead to better outcome.
Okay.
Thank you.
As a reminder to ask a question you will need to press Star then 1 on your telephone.
Operator: 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 Deschant with Barenberg Capital. Your line is now
Our next question comes on the line of Anita.
And the Sham with <unk> capital. Your line is now open.
Operator: Hi, good afternoon. Thanks for taking my question. So again, 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 result?
Hi, good afternoon, and thanks for taking my question.
So again you talked about the.
Preliminary on.
And the 58 weekly sales related to Transcon PTH.
And.
And you gave us.
Operator: data, like what are
And I guess, what do you think about the and the 84 week data on like what to what.
Operator: What do you expect to see beyond just the safety data from there? Thank you. Yeah.
Jan Moller Mikkelsen: Yeah, that is always a great question, what we expect to see. So, when we did go a little better back, 26 weeks, 54, and then 84. It's going to be interesting because what we actually saw between 26 and 54, we saw the same durable response that we had hoped. So 26 to 50 bull, pretty, pretty much what we have expected to see.
What do you expect to see beyond just the.
Safety data from day. Thank you.
Yes that is always a great krish and what we expect to see.
So when we did go a little bit of back 26 week 54, and then a twofold.
It's going to be interesting because what I, what we saw between 2006 and 54, we sold the same durable response that we had hoped to see.
26% to 50 people.
<unk> predictor on really what we have expected to see.
Jan Moller Mikkelsen: 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 R, a trial running for one year with 58 patients getting physiological PTSD treatment. And what we didn't know was when we really would kick in the normal remodeling of the bone structure, which basic everyone wants to have. If you go to a non-surgical SB patient, the basic you can see this high density structure is not healthy. It's actually providing a larger fractal.
What was the unknown factor force, which we didn't know and this is going back to the script that statement about this first time in history that basic.
<unk> running for 1 year with 58 patients gating decrease and logical PT treatment and what we didn't know when we really will kick in the normal remote ruling of the bond structure, which basic everyone want to have.
If you go to non surgical patients and <unk>.
You can see this high density structure is not healthy.
Actually providing lots of fracture.
Jan Moller Mikkelsen: What we wanted to see, which is an integrated part of our HB treatment, is that with transcontpdra, a physiological PTAs label, we also restore the normal remodeling of the bone so we can make them healthy. And that we actually saw had already started to kick in potentially a little bit faster than we actually would have expected. We saw with our 84 weeks that the bone markers were basically going down now, trending much more to the mid of the normal levels, and I actually think we will see that continue under 84 weeks.
And what we wanted to see that as an integrated part of our HB treatment.
Transcon PTH physiological Picci's labor, we also restore restore the normal remodeling of the zone. So we can make them healthier.
And that we actually already started to kick in and potentially a little bit faster than we actually.
Have expected we sold.
84 weeks that'd be basically sold the bone markers going down now trending more to the meat of the normal levels and on exit we will see that continue on the 84 weeks and on.
Jan Moller Mikkelsen: And I actually think we will see the same with the bone density, that you're getting the normal bone structure back, and therefore you must have more going down to the set score of zero. So I think that this will be the main difference. I think from the safety perspective, that was basically not in the team, the 26 and 54. Same excellent safety profile. So I think that is mainly what we will see.
And I assume you will see the same with the bone density that you're getting the normal bone structure back and therefore, you must mark go and down to the 6 core office too. So I think that this will be the main different I think from a safety perspective to us basic notching between but 26% and 54.
Excellent safety.
Perspective.
I think that is mainly what we will see.
Jan Moller Mikkelsen: What we still are looking for is something like this, but we already saw that in the beginning because there was actually some patient that had already impacted the filtration rate in the kidney. And the axel 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 normal functional function by having a normal physiological PTA.
What we are looking for is some thing like.
And.
But we already saw that already and the beginning because there was excess on patients that have already impacted our filtration rate and the kidney and the actual turned out to be pretty good and restored so axa belief that won't be sold with 26 and already <unk> 50.
And it's really moving into the same direction that we really are.
Restoring the normal functional function by having and normal exclusive logical PTH levels.
Jan Moller Mikkelsen: That's very helpful. Thank you. Now, our next question.
That's very helpful. Thank you.
Okay.
Thank you. Our next question comes on the line of Diego <unk> with Credit Suisse. Your line is now open.
Operator: The next question comes from the line of Tiago Felt with Credit Suisse. Your line is now open.
Operator: Thanks for taking the question. Just a quick follow-up on CNP. You'll literally want to keep the data blinded. So what exactly could constitute a clinical program update in
Hey, Thanks for taking the question just a quick follow up on TNT. So.
And you alluded to or you want to keep the data blinded so what exactly could constitute the clinical program and update in Q4.
Operator: program update in Q4, and what is your regulatory strategy, your view, to the best of your knowledge, on what that could look like for that program? Thank you.
And what is currently your regulatory strategy your view to the best of your knowledge on what that could look like for that program. Thank you.
And so this is Chris.
Christian that we actually spent some time talking and discussion all the time because.
Jan Moller Mikkelsen: It's a question that we actually spend some of our time talking and discussing all the time because when we are in a situation where we have our dose escalation and its function, what is easy to look for is safety, because you can do that in a totally blinded way. And if you start to see something, potentially, you should be starting to unblind yourself to see where it's coming from. But what we have seen in this trial is everything we have hoped for in a safe way, not even an injection site reaction, not even any kind of cardiovascular risk observed anywhere. So it's really, really a safe treatment.
When we are in a situation, where we have our dose escalation.
And its function.
It's easier to Lucas safety, because you can do that on a total blinded way and if you start to see something potentially you should be starting to online to see where it's coming from but what we have seen in this trial.
Is everything we have hopeful.
Safe way not even injection site reaction not even any kind of cardiovascular risk of soup and you place. So it's really really a treatment.
So 1.
Jan Moller Mikkelsen: 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 and overview about the program, how we are progressing with all the different with the trial from the two different 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 in the end of the year.
What we're doing now is that.
Dose escalation and we will continue to have each single cohort climbed for 1 year.
And we trying to do is to try to select 1 dose or 2 dose to a cohort expansion perfect and we hope to do that doing this year. So when we come to the end of the year, we will give you and.
And overview about the program, how we are progressing with all the different.
The trial from the 2 different.
You can say punt on 2 independent phase II trial, we can give you in the best way and this is where we have a continued discussion what is meaningful to give without anyway to compromise. The blinded part of this and this is what we are discussing now and I'm quite sure and we will have an interest.
And package it and the end of the year.
Operator: Thank you. Our last question comes from the line of Yan-Zu with Wells Fargo. Your line is not open. Hi, thanks for taking my question. In terms of pricing for 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 that? And also, do you plan to ask?
Perfect. Thank you.
Thank you.
Last question comes on the line of Yanan, Zhu with Wells Fargo. Your line is now open.
Hi, Thanks for taking our question.
In terms of pricing of Transcon G H.
And 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.
And.
And if you can comment.
Clarify on that.
That and also.
Do you plan to access the other pediatric indications, Florida growth hormone.
Operator: Assess the other pediatric indications for growth hormone.
Operator: on market,
And market.
Operator: and Adiopathic Short Stratis.
And Mike Turner syndrome, and idiopathic short stature, and which collectively could account for as much sales as the gross.
Operator: which collectively could account for as much sales as the gross hormone deficiency
Operator: on deficiency indication.
Gross hormone deficiency indication thanks.
Jan Moller Mikkelsen: Related to your first question, Jasper came with a clear statement. We have a superior product; we will expect a superior price. I think it was a clear statement from Jasper, about which I say everything.
And related to your first question, yes, okay, and with a clear statement.
We have a superior product, we will and expect superior pricing.
I think there was a clear statements from yes, but I.
I would say.
Jan Moller Mikkelsen: To the second question, we are dedicated to Global Reeds Label Expansion. Global REITs is to go everywhere in the world, the 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 disease, 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 Transcon Growth hormone to build it up to the leading brand and we will also go for the necessary indication to achieve that.
Everything to the second question, we are dedicated to global rates label expansion.
Global rights is to go everywhere and the words label expansion, we already started on and we have a full site trials, which are adult growth hormone deficiency and currently now we are discussing what extra indication is we want to pursue in and there is a series of interesting.
You can see indications, we can pursue and I think we have a great clinical team that basic are coming up with the best month clinical decided perspective. So we really can do what we want to do ensuring that is a label expansion of our transcon growth hormone to build it up to the leading brand.
And we will also go for.
The necessary indication.
To achieve that.
Operator: Got it. Great enough. Thank you. 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.
Got it great great to know thank you.
Thank you.
Thank you.
And there are no further questions ladies and gentlemen. This concludes today's conference call. Thank you for your participation you may now disconnect.
[music].
Operator: ???