Q3 2023 Ascendis Pharma A/S Earnings Call
Speaker 1: Please stand by. Your program is about to begin. If you need assistance during your conference today, please press star zero.
Please standby your program is about to begin if you need assistance during your conference today. Please press Star zero.
Hello, and welcome to the Cintas farm at third quarter 2023 earnings conference call and webcast.
Speaker 1: Hello and welcome to the Ascendus Farmit third quarter 2023 earnings conference call and webcast.
Speaker 1: Following the prepared remarks, there will be a question and answer period. Instructions will be given at that time.
Following the prepared remarks, there will be a question and answer period and instructions will be given at that time.
Speaker 1: I would now like to hand the conference over to Tim Lee, Senior Director, Investor Relations, Ascendus Pharma. Sir, you may begin. Tim Lee, Senior Director, Investor Relations, Ascendus Pharma Yes, sir.
I would now like to hand, the conference over to Tim Lee Senior Director Investor Relations of Sandoz Pharma, Sir you may begin.
Speaker 2: Thank you, operator, and thank you everyone for joining our third quarter 2023 financial results conference call. I'm Tim Lee, senior director of investor relations at Ascendus Pharma. Joining me on the call today is Jen Mickelson, president and chief executive officer, Scott Smith, executive vice president and chief financial officer, and Dr. Sina Singal, executive vice president and head of clinical development oncology.
Thank you operator, and thank you everyone for joining our third quarter 2023 financial results Conference call.
I'm Kim Lee Senior director of Investor Relations Edison as pharma joining me on the call today are UN Mickelson, President and Chief Executive Chief Executive Officer, Scott Smith, Executive Vice President and Chief Financial Officer, and Dr. Christine a single executive Vice President and head of clinical development oncology.
Speaker 2: Before we begin, I'd like to remind you that this conference call will contain four looking statements that are intended to be covered under the safe harbor provided by the Private Securities Litigation Reform Act.
Before we begin I'd like to remind you that this conference call will contain forward looking statements that are intended to be covered under the safe Harbor provided by the private Securities Litigation Reform Act exam.
Speaker 2: Examples of such statements may include, but are not limited to statements regarding our commercialization and continued development of Skytropha for the US and European markets.
Examples of such statements May include but are not limited to statements regarding our commercialization and continued development of Skype for the U S and European markets.
Speaker 2: as well as our expectations for 2023 Skytrophil revenue, the expected timing of the approval and launch of Transcon PTH in the US and the EU, our pipeline candidates and our expectations with respect to their continued progress and potential commercialization.
As well as your expectations for 2023 scheduled for revenue.
Expected timing of the approval and launch of Transcon PTH in the U S and the EU, our pipeline candidates and our expectations with respect to their continued progress and potential commercialization.
Speaker 2: Our strategic plans, our goals regarding our clinical pipeline, including the timing of clinical results.
Our strategic plans or goals regarding our clinical pipeline, including the timing of clinical results are.
Speaker 2: our ongoing and planned regulatory filings, and our expectations regarding the timing and the results of regulatory decisions, our expansion into new therapeutic areas.
Our ongoing and planned regulatory filings and our expectations regarding the timing and the result of regulatory decisions.
Our expansion into new therapeutic areas, our progress towards vision, three by three and our ability to become cash flow positive and create a sustainable profitable and leading global pharma company.
Speaker 2: our progress toward vision three by three and our ability to become cash flow positive and create a sustainable profitable and leading global pharma company.
Speaker 2: These statements are based on information that is available to us today, actual results and events could differ materially from those in our forward-looking statements, and you should not place undue reliance on these statements. We assume no obligation to update these statements as circumstances change except as required by law. For additional information concerning the factors that can cause actual results to differ materially, please see our forward-looking statement section in today's section.
These statements are based on information that is available to us to ask today actual results and events could differ materially from those in our forward looking statements and you should not place undue reliance on these statements. We assume no obligation to update these statements as circumstances change except as required by law or additional information concerning the factors that can.
Cause actual results to differ materially. Please see our forward looking statement section in today's today's press release and the risk factors section of our most recent annual report on form 20-F filed February 16th 2023.
Speaker 2: Today's press release in the risk factors section of our most recent annual report on Form 20F filed February 16, 2023.
Speaker 2: Kranzkon growth hormone or Kranzkon HGH is approved in the US by FDA and the EU has received MAA authorization from the European Commission for the Treatment of Pediatric Growth Hormone Deficiency.
Transcon growth hormone or cramps gone Hgh <unk> approved in the U S. By F. D E and the EU has received MAA authorization from the European Commission.
For the treatment of pediatric growth hormone deficiency.
Speaker 2: Otherwise, please note that our product candidates are investigational and not approved for commercial use. As investigational products, the safety and effectiveness of the product candidates have not been reviewed or approved by any regulatory agency. None of the statements during the conference call regarding our product candidates shall be viewed as promotional.
Otherwise please note that our product candidates, our investigational and not approved for commercial use as investigational products the safety and effectiveness of the product candidates have not been reviewed or approved by any regulatory agency. Neither statements. During the conference call regarding our product candidates shall be viewed as promotional.
Speaker 2: On the call today, we'll discuss our third quarter 2023 financial results and we'll provide further business updates. Following some prepared remarks, we'll then open up the call for questions. And with that, we hand it over to you, Dan.
On the call today, we'll discuss our third quarter 2023 financial results and will provide further business updates. Following some prepared remarks, we'll then open up the call for questions and with that let me hand over yet.
Thanks, Tim.
Good afternoon, everyone.
Speaker 3: Our dedication to the values of patients, science and passion remains the foundation for scientists to create long-term value for all states.
Our dedication to the values of patient signs and passion remains that founded the foundation for saying just to create long term value for all stakeholders.
Speaker 3: In the US, we achieved market value leadership with Skytofa within two years by sticking to our belief that a premier product deserves a premier prize. Following the same strategy, we have achieved market value leadership with Skytofa.
In the U S via sheets market value leadership for Sky to wait into yes by sticking to our belief.
That a premier product to save a premium price.
Following the same strategy in Germany.
We launched Scott hoping September.
And also expect.
Speaker 3: to also launch Transcon PTA in January 2020.
So also long transcon PTH in January 'twenty 'twenty four is.
Speaker 3: if approved by the European Commission in November .
Approved by the European Commission in November.
Speaker 3: In select other European countries we will use the same
In select.
The European countries, we will use the same.
Speaker 3: direct sales model to launch our portfolio as we have in the US and Germany. We call this
Direct sales model to loans or portfolio SB.
In the U S and Germany.
Recall this EU direct.
Speaker 3: In other markets we will commercialize our portfolio to sales and distribution.
In other markets, we will commercialize our portfolio to say this and distribution partners, who are local experts and bath diseases.
Speaker 3: who are local experts in Beartie.
Speaker 3: We refer to this as International Indirect Map.
We refer to this as international indirect markets.
Speaker 3: In the end, we intend to partner our endocrinology rare disease project.
In Japan, we had said to partner our endocrinology rare disease products.
Speaker 3: We believe we have the organization and the manufacturing capacity to support expected launches of three independent endocrinology rare disease products by 2025.
We believe we have the organization and the manufacturing capacity to support expected losses of three independent endocrinology rare disease products by 2025.
Driving further growth we aim to continue to work to create highly differentiated transcon product candidates.
Speaker 3: Driving further growth, we aim to continue to work to create highly differentiated transcon product campaigns.
Speaker 3: expanding into additional endocrinology rare disease.
Expanding into additional endocrinology rare disease indications.
Speaker 3: Last quarter, we announced the expansion of our TransCon platform.
Last quarter, we announced the expansion of our transcon platform.
A new type of carrier platform.
Speaker 3: specific design to support new product opportunities in diseases with last patient population.
Specific design to support new product opportunities and diseases with large patient population.
I look forward to share more information with you about this in the near future.
Speaker 3: I look forward to share more information with you about this in the near future.
Let me provide more detail on each of our programs for transcon growth hormone commercialized ESCO talked to.
Speaker 3: Let me provide more details on each of our processes.
Speaker 3: for trash can growth turmoil commercialized as Cartovka. We reported strong revenue growth.
We reported strong revenue growth finished the third quarter with 47 million euros.
Speaker 3: finished the third quarter with 47 million euros.
Included in these shows revenue from Germany.
Speaker 3: We now expect full year 2023 Skype Topher revenue to be between 170 and 175 million euro.
We now expect full year 2023, Skype tool for revenue to be between 170 and 175 million Euro.
To drive differentiation and market leadership.
Speaker 3: We continue to build up the science and data behind schedule.
We continue to build out the signs and data behind schedule.
The recently announced results from our long term extension trial, showing that the majority of patients treated the transcon called coming met or exceeded avatar and so if this at the time of their treatment complete call last visit.
Speaker 3: We recently announced results from our long-term extension trial showing that the majority of patients treated with transplant growth can be met or exceeded have a parental height as is at the time of their treatment completeness or last.
The data also demonstrated the long term safety of scheduled time in patients treated up to six years.
Speaker 3: The data also demonstrated the long-term safety of scotova in patients treated up to six years.
Yes.
In adult growth hormone deficiency, we expect to share top line results during the fourth quarter from our global Phase III foresight trial.
Speaker 3: We expect to share top-line results during the fourth quarter from our global Phase 3 foresight.
Speaker 3: Today we estimate less than 4% of adult patients suspected of having growth hormone deficiency.
To date, we estimate less than 4% of adult patients suspected of having co promote efficiency are treated with optimal.
Speaker 3: making this an opportunity to both expand SkyTrophys label and expand the overall growth to 7.5 million people.
Making this an opportunity to both expand skytrooper labeled and expand the overall growth hormone market.
Speaker 3: In addition, we plan to launch Skytrooper in certain markets in our international indirect region with initial revenue contribution expected to begin in 2021.
In addition, we plan to launch schedule for in certain markets in our international indirectly.
With initial revenue contribution expected to begin in 2024.
Speaker 3: Determined to transcompete in the European Union, EMEA CHMP adopted a positive opinion in September .
Turning to try and Scott <unk> in the European Union.
M a.
See HMP adopted a positive opinion in September.
Speaker 3: recommending approval of transgon PTAs as a replacement therapy for adults with chronic hypoperatitis.
We're recommending approval of Transcon PTH.
<unk> replacement therapy for adults with chronic hyperparathyroidism.
We expect the European Commission final decision on our marketing authorization application this months.
Speaker 3: We expect the European Commission final decision on our marketing authorization application this month.
Speaker 3: If approved, we plan to launch Transcon PETA in Germany in January 2024.
If approved.
Our plan to launch Transcon PTH in Germany in January 'twenty 'twenty four.
In Germany alone.
Speaker 3: There are the annual cost for currently approved PTAH treatment is around 77,000 euros per patient. Our target population is 22,000 chronic hyperperic.
And on the cost curve.
The approved <unk> treatment is.
77, Towson Eurosport patient our target population is 'twenty two Thompson chronic hydropower piece out of the old patient population of around seven G Thompson patient.
Speaker 3: out of the overall patient population of around 17,000 patients.
Our staff is team in Germany that long Scott Trophy.
It's ready to long Transcon PTH if approved.
And EU approval with.
Speaker 3: We'll also provide the basis for market participation and initiation of commercial activities for transcon PTA's in additional markets in our internal market segment, where we expect too long starting in 2024.
We'll also provide the basis for marketing authorization and education, all commercial activities for Transcon PTH in a decent market in our Internet market segment, where do you expect to launch starting in 'twenty 'twenty four.
And full launches in Europe direct in 2025, following standup price and reimbursement pathways.
Speaker 3: and further launches in Europe direct in 2025, following standard price and reimbursement pathways.
Okay.
By following our equity for product innovation.
Speaker 3: we will have taken transport PTA from idea to expected regulatory approval all in about 7 years.
You will have taking transcon PTH from idea to we expected what's the Toyota.
In about seven years.
In the U S. All documents have been finalized and we expect to with the NDA for Transcon PTH for Doug.
Speaker 3: In the US, all documents have been finalized and we expect to resume the NDA for Transcon PTAG for dot the hyperpire tyre resment within NAB.
The hypo pad Thai resent within a week.
Speaker 3: We expect to know whether the FDA has accepted our resubmitted NDA.
We expect to know where the FDA has accepted all with somebody and D. A.
Speaker 3: with 30 days from the risk submission.
So a few days from database submission date.
Speaker 3: If accepted, we expect the FDA to notify us at the time where the resubmission is Class I or Class II and provide a new PDU for the FDA.
If accepted we expect the FDA to notify us at the time there. The Resubmission is cost one or class two and provide a new <unk> date.
Speaker 3: Besides this information, we were not coming to further on this.
Besides this information we will not comment further on their way submission.
Speaker 3: As with Skype Hofer, we continue to build up the science and data behind Transcom.
As to the schedule that we continue to a bit of the science and data behind Transcon PTH in September we announced a post hoc analysis of phase two and phase III data demonstrating substantial increase.
Speaker 3: In September , we announced a post-hoc analysis of phase 2 and phase 3 data, demonstrating substantial increase in estimated GFR.
Estimated.
T F R.
In adults with Hypo pilots recently treated with Transcon P. T. This data such as the treatment of Transcon PTH chemical gross impaired kidney function in patients with type of Qatar recently.
Speaker 3: in adults with hypopyotardism treated with trans-computing.
Speaker 3: This data suggests that treatment with transgon PTA can reverse impaired kidney function in patients with hypnis supergl hopefulImplant strokes.
Yes.
Turning to a transparent CMP.
Speaker 3: This is our third endocrine rare disease product candidate.
This is our third endocrine rare disease product candidate.
Speaker 3: Following our interface two meetings with US and AU regulatory agencies, we have lines.
Following our end of phase II meeting with U S E U.
Tori agencies, we have light.
Speaker 3: on the part way to potential-acceve regulatory approvals in the US and
On the pathway to potentially achieve regulatory approvals in the U S and E U.
Speaker 3: We expect top-line results from our PURES Phase III approach trial with completed enrollment in Q3 in the second half of 2024.
We expect topline results from our phase III, <unk> trial, which completed enrollment in Q3 in the second half of 'twenty 'twenty four.
Speaker 3: We continue to have strong patient retention in our trials. And during the fourth quarter, we will provide an update and share with you one year follow-up data with the open label extension portion of a conference.
We continue to have strong patient retention in our types and doing the fourth quarter.
Provide that update and share with you one year follow up data that ultimately, but extensive PA portion of accomplish.
Speaker 3: We believe the stronger tension in our clinical trial is a result of additional benefit of Transcon CMP.
We believe the strong retention in our clean needs. A trial is a result of additional benefit of Transcon CMP.
You should to heightened improvements.
Speaker 3: as we have further evaluated the science behind air chondroplasia and our own data. We now believe that air chondroplasia is a disease of both skeletal growth and muscle.
<unk> S. We have.
The science behind <unk> and our own data. We now believe that <unk> is a disease a boat skill led to the growth and muscle function.
Speaker 3: and that the continuous exposure to the CMP enabled by transcontinue CMP may be able to address both elements.
And thats the continuous exposure to assist CMP enabled by Transcon CMP.
May be able to address both elements.
Speaker 3: At our upcoming update, we will disclose this additional potential benefit of Transcon CMP in addition to...
And I'll I'll call me update we would disclose this.
That decision and potential benefit of Transcon CMP in addition to a height.
If we all right that is correct that eklund a pace. It is a disease of bone skeletal growth of muscle function.
Speaker 3: If we are right, that is correct that acondopasia is a disease of both skeletal growth and muscle.
Speaker 3: This means that transconCLP could potential of a value for adults living with a condo place.
This means that transcon CIP could potentially offer value for adults living with excellent Alicia.
Speaker 3: who experience muscle fatigue and other medical and quality of life.
Who experience muscle fatigue, but although medical and quality of life impacts that.
Speaker 3: that may be potentially addressable with constant exposure to CM.
That may be potentially a choice about with constant exposure to CMP.
We are in a.
Speaker 3: We are in a constructive dialogue with regulators on how best to evaluate the pretended impact on transcon CMP on comorbidities.
Constructive dialogue with the latest on how best to emulate that pertained to impact on transcon CMP on comorbidity quality of life and other important aspect of Ecuador Pacer. In addition to a height to support any indication for treatment of the patient.
Speaker 3: quality of life and other important aspects of achondroplasia, in addition to height, to support an indication for treatment of achondroplasia.
By now a key focus in the treatment of Eklund a piece that is to address the comorbidities that are associated with this disease can you believe it even more effective way to address height.
Speaker 3: While our key focus in the treatment of acondylplasia is to address the coma operating system of here could
Speaker 3: You believe an even more effective way to address height. If needed or desired, could be CLP in combination with Go.
Needed or decided could be CMP and combination with Coca Cola.
Speaker 3: We believe that this combination therapy may provide greater analyzed high velocity and CMP alone and at the same time address the comorbidity of aqueducts.
Do you believe that this combination therapy may provide queda annualized height velocity and CMP alone and at the same time address the cobalt PTT of Ecuador Pizza.
Or previously.
Speaker 3: Our previously presented preclinical data demonstrates the attitude effect of combining transgon CMP and growth hormone in animal models.
Preclinical data demonstrate that attitude effect of combining transcon CMP and growth Campbell.
Animal models.
Speaker 3: consistent with stimulation of different growth promoting signaling pathway in the growth.
Consistent with stimulation of different co promoting stigma parkway in the corporate.
Speaker 3: Supporting this idea, a group out of Osaka University in Japan presented a poster during last month's ASBMR, showing that the last group of 41 accondepletic children treated with gopetamol demonstrated first year analyzed height velocity of around 7.4 cm.
Supporting this idea a coupe of Osaka University in Japan presented at Postop doing last month.
B M are showing that the last group of 40 Bon a condo.
Patients treated with <unk> demonstrated first year annualized height velocity off around 7.4 centimeter width to.
Speaker 3: with sustained growth benefit through five years of...
Sustained growth benefit two five years of treatment.
Speaker 3: To explore this concept we plan to submit an I&D amendment or similar to initiate a combination tribe of transconc.cap and skythover by the end of this year.
Two to explore this concept we plan to submit an IND amendment or similar to initiate a combination trial of transcon, CRP and sky tool, but by the end of this year.
Moving to oncology.
Yeah.
Speaker 3: For transcon IL-2-beta-gamma, we recently reported new data from the iBelieve trial of transcon IL-2-beta-gamma as monotherapy and in combination with PEMPO at 0.5.
For Transcon <unk> to be the comment you've recently reported new data from the I believe trial of Transco to bridge the Gama as mono therapy.
<unk> in combination with people at ESMO.
Speaker 3: These data confirm that transgon IL-2 beta gamma dosed every three weeks demonstrated clinical activity as monotherapy or combination therapy across late-line, heavily pre-treated patients in multiple tumor treatments.
These data confirmed that transcoding ought to be the common dose every three weeks demonstrated clinical activity as monotherapy or combination T. O. P of course late line heavily pre treated patients in multiple tumor types.
Speaker 3: further strengthen our confidence in its best in class.
Further strengthening our competent and is best in class potential.
Speaker 3: Into out of three small lung cancer patients, treated with combination therapy in the trial, and confirmed partial response, and uncomformed complete response in the first two assessments of an ongoing patient were observed.
And two out of tweets small lung cancer patients treated with combination therapy and the tribe and confirmed partial response and unconfirmed complete response in the first tumor assessment of an ongoing patients wear off suite.
Speaker 3: We believe these data are intriguing despite the very small sample size, considering the treatment history of the responders and the substantial unmedicine.
We believe these data and tweaking despite the very small sat beside considering the treatment history of the responders and the substantial unmet medical need.
Enrollment is ongoing in the indications specific cohort of <unk>.
Speaker 3: Enrollment is ongoing in the indication specifically for the wall of I believe, where we are now enrolling 20-40 patients in each indication, and we expect interring results in the second half of 2024.
I believe where we are now enrolling 20 to 40 patients in each indication and we expect interim results in the second half of 2024.
Speaker 3: In summary, with growing revenue maturing of our pipeline, we continue to vote progress to our goal of being.
In summary, with growing revenue maturing of our pipeline.
Continue to progress to all co op.
Becoming profitable.
Speaker 3: With our mission 3x3 on track to be achieved in 2025, we are preparing for our next mission for growth to 2030.
But our vision for you, but we are on track to be achieved in 2025.
We're preparing for an exhibition football to 2030.
Speaker 3: In our next mission, which I look forward to sharing with you at the beginning of 2024, we will work to level our fully integrated capability to become the leading endocrinology vertices company. Taking product from concept out to patient on a global scale with a highly productive organization.
Next we should.
I look forward to sharing with you at the beginning of 2024, we believe work to deliver a fully integrated capability to become the leading endocrinology rare disease company, taking product from concept out to patients on a global scale.
The highly productive organization.
Speaker 3: In other areas where we believe Transcom can deliver best-in-class product candidates, as we have been on quality of termality, we plan to pursue partnerships or business models to take our product candidate to late-state development to come facilitate.
In other areas, where we believe transcon can deliver best in class product candidate actually have been oncology ophthalmology, we plan to pursue a partnership or.
The business model to take all of our product candidate to late stage development to commercialization.
Speaker 3: Thus, future partnership in oncology and auto-molody, along with the maturation of our inocunology program, should result in lower expenses in the coming.
Thus fuel to a partnership in oncology and ophthalmology, along with the maturation of our Endocrinology program should result in lower expenses in the coming years by staying focused on achieving value market leadership globally for endocrinology rare disease portfolio, we believe is saying.
Speaker 3: By staying focused on achieving value market leadership globally for our endocrinology rare disease portfolio, we believe ASCENDS will deliver sustainable value over the long term for patients, shareholders and society. I will now turn the call over to Scott for a financial review before we open for questions.
Basically you both sustainable value over the long term for patient shareholders and society.
Now turn to call over to Scott for financing.
With you before we open for questions.
Thank you Ian.
Speaker 4: As Yen noted, we believe we are making significant progress towards our goal of becoming cash flow positive, with Skytropha revenue growing each quarter combined with diligent expense control.
As Yan noted, we believe we are making significant progress towards our goal of becoming cash flow positive with sky trough of revenue growing each quarter combined with diligent expense control.
Speaker 4: I will touch on some key points surrounding our financial results, but for further details, please refer to our Form 6K filed today.
I will touch on some key points surrounding our financial results, but for further details. Please refer to our form 6K filed today.
Speaker 4: Total revenue for the third quarter of 2023 was 48 million euros.
Total revenue for the third quarter of 2023 was $48 million Euro Sky.
Speaker 4: Skytropha revenue for the third quarter was 47 million euro, compared to 35.9 million euro reported in the second quarter and 12.3 million euro reported in the same period last year.
<unk> revenue for the third quarter was 47 million euro compared to $35 9 million Euro reported in the second quarter and $12 3 million Euro reported in the same period last year.
Speaker 4: The sequential growth in Skytrooper revenue was primarily driven by increased demand in the US, with minimal foreign currency impact of around 100,000 euros.
The sequential growth in Sky trove of revenue was primarily driven by increased demand in the U S with minimal foreign currency impact of around 100000 Europe.
Exiting the third quarter, we estimate we had low double digit penetration into the U S. Pediatric ghd patient population and we see a large opportunity in front of us to grow our patient share in pediatric ghd, which we estimate to be only half of the addressable U S growth hormone market.
Speaker 4: Exiting the third quarter, we estimate we have low double digit penetration into the US pediatric GHD patient population. And we see a large opportunity in front of us to grow our patient share in pediatric GHD, which we estimate to be only half of the addressable US growth hormone market.
Speaker 4: Turning to expenses, R&D costs in the third quarter totaled 111.4 million euro, up 6% sequentially from the second quarter of 2023, primarily driven by higher endocrine rare disease related costs, including clinical expansion and PPQ manufacturing costs related to transcon C&P, partially offset by lower oncology related.
Turning to expenses.
<unk> costs in the third quarter totaled 111 4 million Euro.
6% sequentially from the second quarter of 2023, primarily driven by higher endocrine rare disease related costs, including clinical expansion and <unk> manufacturing costs related to transcon, CMP, partially offset by lower oncology related costs.
Speaker 4: S-GNA expenses declined 9% sequentially to 63.6 million euro compared to the second quarter of 2023, primarily related to lower commercial and GNA external costs.
SG&A expenses declined 9% sequentially to $63 6 million euro compared to the second quarter of 2023, primarily related to lower commercial and G&A external costs.
Yeah.
Speaker 4: Total operating expenses were 175 million Euro for the third quarter, flat sequentially from the second quarter of 2023.
Total operating expenses were $175 million euro for the third quarter flat sequentially from the second quarter of 2023.
Speaker 4: Overall, our operating loss declined sequentially by 5% to 134 million euro for the third quarter from 141 million euro in the second quarter of 2023.
Overall, our operating loss declined sequentially by 5% to $134 million Euro for the third quarter from 141 million Euro in the second quarter of 2023.
We ended the third quarter with cash cash equivalents and marketable securities totaling $455 million euro, including proceeds from the previously announced $150 million royalty funding agreement.
Speaker 4: We ended the third quarter with cash equivalence and marketable securities, totaling $455 million, including proceeds from the previously announced $150 million royalty funding agreement.
Speaker 4: Looking ahead, with continued momentum for Skytropha in the US, we are increasing full-year 2023 Skytropha revenue expectations to 170 million to 175 million euro.
Looking ahead with continued momentum for <unk> in the U S. We are increasing full year 2023, sky trusts, our revenue expectations to $170 million to 175 million Europe.
Speaker 4: Let me now also provide a review of selected key program milestones.
Let me now also provide a review of selected key program milestones for.
Speaker 4: For transcon growth hormone, we expect to report top-line data from the global phase three four-side trial in adult GHD in December .
For Transcon growth hormone, we expect to report topline data from the global Phase III <unk> trial in adult Ghd in December.
Speaker 4: potentially opening an opportunity to both expand Skytropha's label as well as expand the overall
Potentially opening an opportunity to both expand sky trough as label.
As well as expand the overall growth hormone market.
For Transcon PTH as Ken noted we are on track to resubmit, our NDA for Transcon PTH for adults with hyperparathyroidism before mid November.
Speaker 4: For TransCon PTH, as Jen noted, we are on track to resubmit our NDA for TransCon PTH for adults with hypoparathyroidism before mid-November.
Speaker 4: We expect a European Commission decision on TransCon PTH this month. If approved
We expect the European Commission decision on Transcon PTH this month.
If approved we plan transfer.
Speaker 4: Transcon PTH is our second product launch in Germany, starting in January 2024.
Transcon PTH is our second product launch in Germany, starting in January 2024.
For Transcon <unk>, we expect to submit an IND amendment or similar for a new clinical trial evaluating transcon CMP in combination with transcon growth hormone in children with achondroplasia.
Speaker 4: For TransCon CNP, we expect to submit an IND amendment or similar for a new clinical trial evaluating TransCon CNP in combination with TransCon growth hormone in children with a chondroplasia.
Speaker 4: We plan to share follow-up data from the open label extension or phase two accomplished trial later this quarter.
We plan to share a follow up data from the open label extension of our phase two accomplished trial later this quarter.
Speaker 4: And as a reminder, we expect to report top line results from approach, our phase three trial of Transcon CNP in the second half of 2024.
And as a reminder, we expect to report topline results from approach our phase III trial of Transcon CMP in the second half of 2024.
Within our oncology therapeutic area enrollment continues in the phase two portion of our al believe trial of Transcon IL two beta gamma.
Speaker 4: Within our oncology therapeutic area, enrollment continues in the phase two portion of our ALB-BELIEV trial of transcon IL-2 beta-gamma in indication specific cohorts.
In indication specific cohorts, we expect initial data from indication specific cohorts in the second half of 2024.
Speaker 4: We expect initial data from indication specific cohorts in the second half of 2024. With that,
With that operator, we are now ready to take questions.
Speaker 1: At this time, if you would like to ask a question, please press star 1 on your telephone keypad. You may remove yourself from the queue at any time by pressing star 2.
At this time, if you would like to ask a question. Please press star one on your telephone keypad.
You may remove yourself from the queue at any time by pressing star too we.
Speaker 1: We do ask that you limit yourself to one question and one follow-up. We will pause for a moment to allow questions to queue.
We do ask that you limit yourself to one question and one follow up.
I'll pause for a moment to allow questions to queue.
Once again that is star one to ask a question.
Speaker 1: Our first question comes from Jessica Fye with JP Morgan. Your line is open. Please go ahead.
Our first question comes from Jessica Fye with Jpmorgan. Your line is open. Please go ahead.
Speaker 5: Hey guys, this is Nick on for Jess. Thanks for taking our questions. Can you talk to us a bit more around your strategy with the oncology and ophthalmology programs and kind of what that means in terms of OPEX, both over the near term and maybe more so over the long term and how that could change?
Hey, guys. This is Nick on for Jeff. Thanks for taking our questions can you talk to us a bit more around your strategy with the oncology and ophthalmology programs and kind of what that means in terms of opex.
Over the near term and maybe more so over the long term and how that could change.
Speaker 3: Thanks Nick, and the question related to
Thanks, Nick and the Christian related too.
Speaker 3: What do we do as a company with a very, very strong...
What do we do as a company with a bev as strong.
Speaker 3: platform technology that basically have application in nearly every therapeutic area.
Platform technology that basic have application in yearly average therapeutic error.
We are saying is we'll be focused on rare disease. Endocrinology. This is wherever you will be integrated from idea state up to their patients. So we're building all that effort.
Speaker 3: We are sendees will be focused on rare disease endocrinology. This is where we will be integrated from idea state out to the patient. So we have it building up all the effort.
Speaker 3: We also know that the transcontact technology
We also know that the transcon technology.
It would provide.
Speaker 3: paradigm shift product, best in class product, outside our own focus area. And we also feel that we need really to be part of that valuation also to the benefit of the...
Paradigm shift product best in class products outside our own focus area and we also feel that we need we need to be part of that value equation also took the benefit of the patients.
Speaker 3: We will have different business models that will fit exactly to each single-term product. Some areas we will basic outlacing on early stage where the feeling is that it's the optimal thing. It could be potentially large patient indications where we basically never can succeed with running all the big clinical trials.
We even have different business models fit exactly to each single turbotax. Some areas. We have a basic outlines soon on our early states. We're feeling is that is the optimal thing it could be potentially be lost patient indications, where the basic neveau can succeed with running.
All of the clinical trial.
Speaker 3: It can be air like a tomology where we mature it with two states and then we potentially give it out.
It couldnt be areas like ophthalmology.
<unk>, the two states and they'd be potentially give it out either.
Speaker 3: either to Espenaut or an outlying or a combination of both, where we feel that an independent management team really with a focus on, for example, ophthalmology, really, really can mature. It's not the same thing as Sandysnot will be continually be involved. We will still provide service to this entity. We will be part of the offsite, both related to royalties, milestone payments.
Two a spinout or an <unk> or a combination of both where we feel that an independent management team really with a focus on for example, ophthalmology really really commit to it it's not the same thing as saying this not will be continually be involved we will still provide service to this entity.
We will be part of the upside both related to.
<unk> milestone payment and equity.
Speaker 3: In other areas like oncology where we feel that we really can make a huge difference from the patient, we make an investment in it and we will continue to make an investment into it until we feel that we can get the full value on this. And when I see how we're progressing with this oncology effort, I'm really, really proud about it.
In other areas like oncology, where we feel that we really can make a huge difference on the patients we made an investment in it.
Continue to make an investment into it onto their feet.
Get the fully value audits.
When I see how we're progressing with this oncology effort I'm really really proud about it.
Okay, Great and maybe can you help set the stage for the update from the phase two accomplished trial.
Speaker 5: Okay, great. And maybe can you help set the stage for the update from the Phase 2 accomplished trial and what you hope to see when you have all the qualifications on the 800 micrograms for the full year?
And what you hope to see when you have all the obligations on 800 microgram.
So full year.
Speaker 3: Yeah, what we hope to do that we will provide you with the
Yeah, what we hope to do that we will provide you with the element that we really believe is essential for their patients.
Speaker 3: element that we really believe is essential for the patients. We will provide you with the sustainability of how we keep.
<unk> beat the sustainability of how we keep the positive benefit on gross but we would also come in and trying to give you. The explanation both from a scientific base, but also true data how do we feel that we are providing a benefit.
Speaker 3: the positive benefit on growth, but we will also come in and try to give you the explanation, both from a scientific base, but also through data, how we feel that we are providing a benefit to the treatment besides.
So the treatment besides.
Speaker 3: besides just providing them analyzed high velocity. We 100% in the
Besides just providing the annualized height velocity.
We are 100% in the belief that.
Speaker 3: The key element first is to address the co-modbidities.
The key element for us is to address the duties associated with this deceit and that is our focus on that and this is why we believe that annualized height velocity isn't element if it <unk> and if the patient where they want to have it there can always go into perhaps.
Speaker 3: associated with this disease and that is our focus on that. And this is why we believe that analyzed height velocity is an element if it's desired and if the patient really wants to have it, they can always go into perhaps the most powerful combination.
That's the most powerful combination of.
Speaker 3: of treatment is CMP to move the brake growth hormone to speed up the accelerator and your basic will have...
Treatment is <unk>.
CMP to move the brake growth hormone to speed up the extra radar and your basic will have.
Speaker 3: what we call the Wanted, Desire, Analyze, High Velocity compare to the patient's needs and desires.
What's it called.
Wanted desire annualized height velocity compare to the patient need and desire.
Yes.
Speaker 1: The next question comes from Paul Choi with Goldman Sachs. Please go ahead.
The next question comes from Paul Choi with Goldman Sachs. Please go ahead.
Okay.
Speaker 6: Good afternoon and thank you for taking our questions. My first question is just with regard to kind of kind of PTH positioning in the major European markets, specifically Germany and just how you think positioning will be particularly given surgical complication rates there and then just the pace of reimbursement access and then I'd follow up question.
Good afternoon, and thank you for taking our questions.
My first question is just with regard to.
Transcon, PTH positioning and the major European markets, specifically, Germany, and just how you think.
Positioning will be particularly given surgical complication rates there and then just the pace of.
Reimbursement access and then I had a follow up question.
You're right.
Speaker 3: The European and the international market, as we call it, is what we call a much more diverse way of reimbursing.
The European and the international market as we call. It is what we call must more diverse way of reimbursement.
Speaker 3: The first one is Germany where already from
It's the fastest one is Germany, where already from.
Speaker 3: gain array where we expect to launch Transcon PHA will be fully reimbursed.
Generally where we expect to launch transcon PSA will be fully reimbursed.
Speaker 3: The patient population in Germany is excellent.
<unk> population in Germany is excellent.
Pretty lax compare to the site of our country.
Speaker 3: pretty large compared to the size of the country, mainly driven by and higher frequent on head and neck operation. So you're right, it's a post-surgeon patient that is much higher. And we believe the addressable.
Mainly driven by higher frequent hidden lake operation, So youre right. Its a post surgical patients that is much higher.
And we believe the addressable.
Speaker 3: element of patient, not the total patient population, is about 22,000.
Element of patients not the total patient population is about 22000.
Speaker 3: At the same time, we are in a position where the
At the same time, we are.
We're in a position where.
The only approved.
Speaker 3: treatment for hyperpera in Germany is being taken away from the market. So we are in an intensive discussion with all the key centers, how we really can help more than the 400 patients that basically already are a step is on a PCHA treatment, how to take them on treatment in 24.
Treatment for hydropower in Germany is being taken away from the market. So we are in intense discussion with all the key centers, how we really can help more than 400 patients that basic already.
Step is on a P J 's treatment how to take them on treatment.
24.
Speaker 3: Besides that, for many years there had been a long, long layout of patients that want to be on a PGH treatment, but never had the opportunity to do it. And this is why the European approval gave us a fingerprint.
Besides that for many years that had been a long long layout or patients that want to be on the PTH treatment, but never had the opportunity to do it and this is why the European approval give us and fingerprint.
Speaker 3: starting immediately on a full-blown commercial effort in Germany. At the same time, and you pin or EC approval, give us the opportunity to go to a name, patient program, and addressing more than the 400 plus.
Starting immediately on a full blown commercial.
In Germany at the same time in U P M.
EC approval give us the optimal energy to go to a named patient program and addressing more than the 400 plus.
Speaker 3: NAPA patient that still is in other European countries, which we feel are huge responsible for also helping and also the patient that didn't come into any treatment in the last many years.
Nektar patient that still is in other European countries, which we feel a shoe Swiss sponsor bold for also helping and also the patient that didnt come into any treatment in the last many years rest of EU take 12 to 24 months to be fully reimbursed.
Speaker 3: Rest of EU will take 12 to 24 months to be fully reimbursed, fully running commercial perspective. At the same time, the EU approval, which we expect to get here in the coming week, will provide us with an...
Fully running commercial perspective.
At the same time than EU approval, which we expect to get here in the coming week will provide us with an.
Speaker 3: approval system for addressing patients in the international market. Some markets we will have an independent application. Other markets we can directly refer into our European application. So we are dedicated really to help
Approval system for dosing patients in the international market. Some market, we will have an independent application.
Market, we can direct these with her into our U.
Application.
We have dedicated really to help.
Speaker 3: patients everywhere in the world which have a need for the treatment because of the hypopera patient situation that they face today.
Patient everywhere in the world, which have a need for the treatment.
Because of the hydropower patient situation that it takes to date.
Speaker 6: Great, thank you for that. And just as a quick follow up, with competing HCH products approved here in the US, can you maybe just comment on maybe what mix of formulary access might be a renegotiations this year in any general comments you've had into the 2024 renegotiation period? Thank you.
Okay, great. Thank you for that and just as a quick follow up.
Competing hgh products approved here in the U S. Can you maybe just comment on maybe what mix of formulary.
It might be up for renegotiation this year and any any general comments you have had.
And to the 24 2020 for renegotiation period. Thank you.
Yes.
I've been asked.
Speaker 3: in the last three, four months a lot. What is really the impact on having two other lung-acting products in the US?
In the last three four months a lot what is really the <unk>.
Impact on having two long acting <unk>.
In the U S market.
And clearly.
Clearly.
We would never have seen more interest we have not seen better numbers ever since it got clear.
Speaker 3: We never have seen more interest. We have not seen better numbers ever since it got clear. Wodd...
Really best in class properties Scotch over half compare to the other products really building up on what we have established in the last two years, how we really are changing the treatment regime in treatment of pediatric growth hormone deficient with a better outcome.
Speaker 3: really best in class property Skatover has Compare to the other products really building up on what we have It's that is in the last two years how we really are changing the treatment received in treatment of pedantic growth hormone deficient with a better outcome and that is not going to change with any other long-acting basic is just providing
And that is not going to change with any long acting basic is just providing.
Speaker 3: I believe for some of them and lower bar to exceed and overcome compared to for example medical exception.
I believe for some of them and Louisville.
To exceed and overcome compared to flex have been medical exception.
Yes.
The next question comes from Joseph Schwartz with Leerink Partners. Please go ahead.
Speaker 1: The next question comes from Joseph Schwartz with Lee Ring Partners. Please go ahead.
Speaker 7: Great, thanks so much. So a couple questions on transcon CMP if I could first of all.
Great. Thanks, so much so a couple of questions on transcon CMP, if I could.
First of all.
Speaker 7: I was intrigued by your commentary about condor pleasure being not only a bone disease, but having a muscle.
I was intrigued by your commentary about contemplation of being not only a bone disease, but having a muscle component and I was curious if you could just talk a bit about how you.
Speaker 7: component and I was curious if you could just talk a bit about how you
Speaker 7: intend to illustrate the benefit of transcon CMP on those aspects of the disease. And then also in terms of transcon CMPs, we have a lot of research that's going on in
Intend to illustrate the benefit of transcon CMP on that as those aspects of the disease and then also in terms of transcon cmp's potential.
Speaker 7: potential to be combined with transcon or a Sky Trofa, rather, as it's now called. I was wondering, given I thought a lot of the earlier science suggested that growth hormone supplementation in a condraplasia had just an ephemeral benefit, what are your thoughts on the potential for the combination to show a more sustained response in a condraplasia?
And then can be combined with transcon or sky trove of rather.
As it's now called I was wondering given I thought a lot of the earlier science suggested Bob growth hormone supplementation in achondroplasia had just an ephemeral benefit.
<unk>.
What are your thoughts on the.
On the potential for the combination to show a more sustained response in a contemplation.
Thanks for the question, let me start with your last question about the annualized height velocity.
Speaker 3: Thanks for the question. Let me start with your last question about the analyzed height velocity. We always have known that is drop thermal.
We always have known that.
Optimal.
Had a positive effect on our kind of patient.
Speaker 3: Sometime it has been a little bit unclear what has been the magnitude on that because the typical have been
Sometime it has been a little bit unclear what has been the magnitude of that because it typically have been.
Small trials.
Speaker 3: When we saw the tripe coming up from the Japanese group, pretty well controlled, falling patient.
When we sold the trial coming out from the Japanese group pretty well controlled.
Following patients up to five years.
Speaker 3: The number is nice, 41. Everything looks great. They have one year before treatment, follow on what is the analyzed height velocity before treatment. And it's about 4.2, exactly what you will expect to see out from this kind of demographic. And the first year, analyze height velocity up on 7.3, 7.4. That is more than I've seen any kind of CMP treatment ever given. And when you have 41 patients, you
The number is nice 41, everything looked great. The happened one year before treatment follow on what is the annualized height velocity before treatment and this is about four point to exactly what you would expect to see out from this kind of demographic and my first year annualized height velocity up in seven countries.
Seven point for that is more than I have seen any kind of CMP treatment ever keep it and when you have 41 patient I always feel that it's a number that gives me some pretty good comfort and it's the best annualized height velocity I have ever seen in any height trial with <unk>.
Speaker 3: I always feel that it's a number that gives me some pretty good comfort. And it's the best analyzed height velocity I have ever seen in any...
Speaker 3: height trial with accondoplated patients. We went back and analyzed 20 publications about all small trials, some of them, all of them. And you know, if we have been smart, we really have gone to the literature in depth.
Pace of patients.
Went back and analyzed 20 populations about all small trials some of them all of them and you know if we are being smart really has gone to the literature and depth.
Yes.
Speaker 3: We couldn't actually have seen it, because out from this 20 publications we nearly got the same number. When we added AV1R.
We can actually have seen it because often 20 publication, we nearly got the same number when we added everyone off.
Speaker 3: So this is not surprising, it's something that has been in the literature, has potentially been understated, undervalued, but there is no doubt best analyzed hydroelasticity you can get in air conditioner today is on growth hormone. The problem with that is, would it really address pumab
So this is not surprising it's something that has been in the literature have potentially been understated under valued but there's no doubt.
Analyzed height velocity you can get in achondroplasia to date is on growth.
<unk> the car.
And with that is what it really address comorbidities I can be.
Scott.
Speaker 3: I believe that the biology behind the acondiplation is a much more complicated
I believe that the biology behind the contemplation is a much more complicated.
By Lottery code.
Speaker 3: impact on a high-bar active FDR-3-U receptor. And there it comes in.
Impact on our hybrid achieved FDA to USA and that comes in.
Speaker 3: Because in the end, we are just starting the beginning of the beginning of our contemplated treatment. We are not in the end of the beginning even. We are in the beginning of the beginning because we need to address the cold more bit.
Because in the end.
We are just starting the beginning of the beginning of a contemplated treatment. We are not in the end of the beginning even we are in the beginning of the beginning because we need to address the comorbidities. So this is why we believe.
Speaker 3: So this is why we believe the treatment is potentially moving to a combination treatment, where CMP will address some of the biological systems, where we believe at contemplation or the hyperactive FDO3 partway are modifying and deceased that basic are also of the a muscle called impact. And you asked me why did we see that it was basic the patient.
The treatment is potentially moving to a combination Tristan.
CMP.
Address some of that biological systems wherever you believe excellent Appalachia or the hypo at FDA III partly.
Modifying and disease that basic are also often marshal impact and you asked me why did we see that it was basically the patient the parents. The calculus that came back to US we got in the patient reported outcome <unk> after one or two months.
Speaker 8: the parents, the caregivers that came back to us. We got in the patient reporter outcome. We can see after one or two months, there's time you say that patient...
At this time, you say that.
Patient.
Providing much better <unk>.
Speaker 8: Function must better and we said it cannot be growth
Function much better and we think they cannot be growth it cannot be growth and then we started to analyze and we realized that there is a strong muscle component of that and what we would like to do when we have our CMP update we would give you that biology behind it explained why do we do that and all.
Speaker 8: And then we started to analyze this and we realized that is a strong massive component of that. And what we would like to do when we have our CMP update, we will give you that biology behind it, explain why we do that. And also, we believe that it's important for us to provide you data with a combination therapy because I believe in the combination therapy, you can get whatever analyzed high velocity potential, the same as you can see in Dr. Montefitian, up to 10, 12, some, don't know if the students continue their operation or practice Dr. Montefitian, no regards.
So we believe that is important for US also provide you a data a bit of a combination therapy because I believe in the combination therapy, you can get it analyzed.
Annualized height velocity potentially the same as you can see and drove to most efficient up to 10 trucks.
Yeah.
Yeah.
The next question comes from Yaron Werber with TD Cowen. Please go ahead.
Speaker 1: The next question comes from Yaron Werber with TD Cowan. Please go ahead.
Speaker 9: Great. Thanks for taking my question. Maybe one very quick one and the second one is a little bigger. Just on the PTH, the refiling is going to be a sort of in the next week or so. Can you
Great. Thanks for taking my question maybe.
One very quick one and the second one is that a little bit bigger just the PTH. The refiling is gonna be sort of in the next week or so.
Can you can you just.
Speaker 9: Comment, did you sort of, by this point, work out all the analysis that FD is gonna want you to do, do you feel like you have good clarity? And then secondly, on the preclinical glyph one, the data with SEMA obviously looked interesting on a monthly basis.
Comment did you sort of by this point worked out all the analysis that FDA is going to want you to do or do you feel like you have good clarity and then secondly on <unk>.
The preclinical glyph one.
The data with semi obviously looks interesting on a monthly basis.
Speaker 9: What are sort of the next steps as you see them before you can start INB enabling studies? Is it you're still trying to fine tune the construct or is it potentially trying to figure out is this something you want to take yourself for? Is this something that you want to ultimately potentially partner? Thank you.
What is sort of the next steps as you see them before you can start IND, enabling studies is it you're still trying to fine tune the construct or is it potentially trying to figure out is this something you want to take yourself or is this something that you want ultimately potentially partner. Thank you.
Speaker 2: Hey, Yarn, this is Tim. Let me take the first part. As Jen noted on his prepared remarks, besides the information that we just discussed, we're not going to comment further on the resubmission process.
Hey, <unk>. This is Tim let me take the first part as Yan noted in his prepared remarks. Besides information that we just discussed we're not going to provide we're not going to comment further on the resubmission process.
Okay related to Ritchie pad one.
Speaker 3: related to the GPL1. We are in a great state. We came out of the data. It provided the interest that the people could see, the value in it, both as an improved weekly treatment, but also on a once monthly treating for this important segment growing up. And we basically continue the dialogue with companies that interested in that area.
We are in a great states.
We came out of the data it does.
Provided the interest that people could see the value in it both as an improved once weekly treatment, but also on a once monthly treating for this important segment growing up.
We basic.
Continue the dialogue with companies that are interested in that are.
The next question comes from Lee <unk> with Cantor Fitzgerald. Please go ahead.
Speaker 1: The next question comes from Lee Watsick with Cantor Fitzgerald. Please go ahead.
Speaker 10: negative third, technical questions. Just first one, we've seen daily products continue to retreat and reduce outputting Q3. Can you just talk about how much of a tailwind that has been forced guys' whole fund? And where do you see that trend to track in the common quarters?
Thanks for taking my questions.
Just first one we've seen daily products continue to trade in.
Without putting in Q3.
Can you just talk about how much Jeff.
When there has been foreign skyhawk.
And where do you see that trend to track in the coming quarters.
Okay.
The Christian you are addressing what is really happening in the growth hormone market and I think what we are seeing is the result of the consolidation that we had predicted.
Speaker 3: The question you are addressing, what is really happening in the growth hormone market? And I think what we are seeing is the results of the consolidation that we have predicted.
How do you expect it to happen.
Speaker 3: And that is basic what you see in the marketplace today.
And it's basic what youll see in the marketplace today.
Speaker 3: For years ago, when we came up with our phase 2 data and we didn't outlyze our trans-con-globetomone, a lot of this established players basically said, okay, let us find out how we can milk this here in the best possible way. And you don't milk by investing in production capacity.
Four years ago, when it became off at all phase II data and we didn't out licensing our transcon growth hormone lot of this established player.
<unk> said, okay, let us find out how we can mix this year in the best possible way and you don't build investing in production capacity you don't.
Speaker 3: You don't really get the highest property to invest in anything. So that is why you are seeing a daily growth hormone market basic only half
We really get the highest poverty to investing in anything. So this is why you're seeing a daily growth hormone market basic only have three player less now with Salesforce infrastructure and other things like that so instead of having six player you're down to three player unlikely.
Speaker 3: 3 player left now with a Salesforce infrastructure and other things like that.
Speaker 3: So instead of having a 6 player, you're down to 3 players, and likely there will be a fewer player in the coming year when they're bit.
B a few will play out in the coming year.
Speaker 3: really the real consultation will happen. So some way you're saying is that it is the shortest, but the shortest is also indicated in the way that the consultation of the market promoted a shortest of it. Because if with all days with many more company really could provide more capacity on other things, that will not have been a shortest.
Really the real consolidation will happen. So some way youre, saying is that it's just shortage, but the shortages as also indicated in the way that the consolidation of the market promoted a shortage of it.
Of course, it was the old days with menu.
Many more company really could provide more capacity and the other thing David not have been the shoulders.
Speaker 3: It's not changing the fact, it's not changing the fact Wyskite Rover is the leading company, leading brand in value is because we're providing best-in-class properties and it's getting realised by physician, by care giver, by patient and I hope and we know that also by the reimbursement system.
Is that changing the effect is not changing the fact why Scott <unk> is the leading company leading brand and value is because to be providing best in class purposes, and it's getting realized by physician by caregivers by patient and I hope when we know that also.
By the reimbursement system.
Speaker 10: Okay, and then maybe just a question on transcon out to, you'll show some pretty nice data in small cell lung cancer. So is there any plan to add this indication to your admission cohort?
Okay, and then maybe just a question on transcon our too.
Michele some pretty nice data.
Small cell lung cancer. So is there any plans to.
This indication.
Mention cohort.
That's a great question Lee yes.
Speaker 11: That's a great question, Leigh. Yeah, we were very encouraged by our initial signal in small cell lung cancer, and we are exploring adding additional indication cohorts.
Very encouraged by our initial signal in small cell lung cancer, and we are exploring adding additional indications.
Sure.
Okay.
Great.
Speaker 12: The next question comes from Leland Gerschel with Oppenheimer. Please go ahead. Hey, thanks for taking my question. Just wanted to ask further on the plans to explore Transcon CNP for benefit outside of high velocity in terms of other comorbidities. Wondering, Anne, if you could just maybe go into maybe beyond muscle, are there other measures or endpoints that you're contemplating to include in upcoming studies?
The next question comes from Leland <unk> with Oppenheimer. Please go ahead.
Hey, Thanks for taking my question just wanted to ask further on the plans to explore transcon CMP.
For benefit outside of high velocity in terms of other Comorbidities I'm wondering if you could just maybe go into maybe beyond muscle are there other.
Measures or endpoints that you are contemplating to include.
And.
Now coming studies. Thank you.
Okay.
We are exploring a lot of different elements.
Speaker 3: We are exploring a lot of different elements. We want, I would like to group it a little bit. One is the patient reported outcome, where you have both our own patient reported outcome and the other one is the patient reported outcome.
One I would like to group, it's a little bit one is that patient reported outcome.
Where you have both our own.
Develop wait to Luca you have <unk>.
Speaker 3: develop way to look at you have established one as SF-10 and other way that really are capturing how you potentially have a physical benefit of this treatment under. So this is one what we call the patient reported outcome. The other one we're looking for is dedicated co-morbidities, other clear
Jeff just one as SF 10, and other ways that really are capturing how you potentially have a future benefit of this treatment and.
So this is one will be called the patient reported outcome. The other won't be looking Paul is dedicated comorbidities either a clear.
Speaker 8: pattern where we see less comb obesity over a year period because you need to go through some season to require sure you picking up what kind of element of cold or other thing could influence it. The other one is more hardcore element where we basically have a lot of x-rays of the patient.
Pattern, where we see less comorbidity.
Yep period, because you need to go through some system to be quite a few are you picking up what kind of element of coal. The other thing could influence. It. The other one is more hardcore element, where the basic had lot of X rays of the patients are really changing.
Speaker 8: Are they really changing the qualities of changing other elements and we're looking on all different kinds of bone development and other things like that. This is in the older children. When we go to the new point we start and now we go for more, more hardcore facts. How to, for example,
The school.
Of a changing.
And we're looking on all different kind of both development and other things like that this is in the older children. When we go to the new corn be starting a recall for most more hardcore effects how to for example.
Speaker 3: benefit in spinners. Do we see the same element of fast fusion of the synchronized or other things like that? And I think this is where we take the different age group finding out what can we really do that. And that is what we are analyzing and building into our people phase three climate. So we're quite sure when we have
Benefit in spin spin notices to receive the same.
Element of fast fusion of synchronize all things like that and I think this is where we take the different age group finding out what can be really do that and that is what we are analyzing and building into our people phase III trying now so we are quite sure when we have the.
Speaker 3: The unblinding on that, we basically have already the element of discussion with rectatorial agencies how we basically can evaluate that.
The unblinded on that basic half already.
Element of discussion with regulatory agencies, how we basically can iterate that.
Okay. Thank you and then one further question if I may as we look forward to the update from the one year follow up from accomplish.
Speaker 12: Thank you. And then one further question if I may as we look forward to the update from the one year follow from accomplished open-able extension. Just want to, would you be able to provide the retention rate in terms of patients who persisted?
Open label extension, just wondering would you be able to provide the the retention rate in terms of patients who have persisted in the OLED.
Speaker 3: At the one, the data we have all 47 patients, 57. 57, 57 patients.
Okay.
One the data will be able to have to have all 47 patients 50, 750 757 patients.
Great. Thanks very much.
This does conclude today's ascend is pharma third quarter earnings call. You may disconnect. Your line at any time and have a wonderful day.
Speaker 1: This does conclude today's Ascendus Pharma Third Quarter Earnings Call. You may disconnect your line at any time and have a wonderful day.
Speaker 13: F.
[music].
Oh.
[music].