Q1 2025 Ascendis Pharma A/S Earnings Call
Operator: Ladies and gentlemen, thank you for standing by and welcome to Ascendis Pharma First Quarter 2025 Earnings Conference Call. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question and answer session.
Okay.
Speaker Change: Ladies and gentlemen, thank you for standing by and welcome to send US Pharma first quarter 2025 earnings conference call. At this time all participants are in a listen only mode. After the speaker's presentation, there will be a question and answer session.
Operator: To ask a question during the session, you will need to press star 11 on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question, please press star 11 again.
Speaker Change: Ask a question. During this session you will need to press star one on your telephone you didn't hear an automated message of bison.
Speaker Change: She would try your question. Please press star one again.
Operator: Please be advised that today's conference is being recorded.
Speaker Change: He used to be advised that today's conference is being recorded I would like now to turn the conference over to Scott Smith, Vice President and Chief Financial Officer. Please go ahead Sir.
Operator: I would like now to turn the conference over to Scott Smith, Vice President and Chief Financial Officer. Please go ahead, sir. Thank you so much, Operator.
Scott Smith: And thank you, everyone, for joining our first quarter 2025 Financial Results Conference Call. I'm Scott Smith, Chief Financial Officer at Ascendis Pharma.
Speaker Change: Thank you so much operator, and thank you everyone for joining our first quarter 2025 financial results Conference call I'm, Scott Smith, Chief Financial Officer at ascend is pharma joining me on the call today are Jan Mikkelsen, President and Chief Executive Officer, Sherry Glass Chief business Officer.
Scott Smith: Joining me on the call today are Jan Mikkelsen, President and Chief Executive Officer, Sherry Glass, Chief Business Officer, Jae Woo, Executive Vice President and President, Ascendis US, and Aimee Shu, Chief Medical Officer.
Speaker Change: <unk> Executive Vice President and President U S and its U S and Amy Shea Chief Medical Officer.
Scott Smith: Before we begin, I'd like to remind you that this conference call will contain forward-looking statements that are intended to be covered under the safe harbor provided by the Private Securities Litigation Reform Act. Examples of such statements may include, but are not limited to, statements regarding our commercialization and continued development of SkyTrofe and Yorvapath for the U.S., European, and other markets, as well as certain financial expectations, our pipeline candidates, and our expectations with respect to their continued progress and potential commercialization. Our strategic plans, partnerships, and investments, our goals regarding our clinical pipeline, including the timing of clinical results and trials, our ongoing and planned regulatory filings, and our expectations regarding the timing and results of regulatory decisions, expected market developments, and our exploration of market opportunities in therapeutic areas outside of endocrinology rare disease.
Speaker Change: Before we begin I'd like to remind you that this conference call will contain forward looking statements that are intended to be covered under the safe Harbor provided by the private Securities Litigation Reform Act. Examples of such statements May include but are not limited to statements regarding our commercialization and continued development of scratch Rofin yoga path for the U S European and other Mark.
Speaker Change: That's as well as certain financial expectations of our pipeline candidates and our expectations with respect to their continued progress and potential commercialization of our strategic plans partnerships and investments our goals regarding our clinical pipeline, including the timing of clinical results in trials are ongoing and planned regulatory filings and our expectations regarding the timing and results of <unk>.
Speaker Change: Tori decisions expected market developments, and our exploration and market opportunities in therapeutic areas outside of endocrinology rare disease.
Scott Smith: These statements are based on the information that is available to us as of today. Actual results may differ, could differ materially from those in our forward-looking statements and you should not place undue reliance on these statements. We assume no obligation to update these statements as circumstances change, except as required by law.
Speaker Change: These statements are based on information that is available to us as of today actual results may differ could differ materially from those in our forward looking statements and you should not place undue reliance on these statements. We assume no obligation to update these statements as circumstances change except as well.
Scott Smith: For additional information concerning the factors that cause actual results to differ materially, please see our forward-looking statement section in today's press release and the risk factors section of our most recent annual report on Form 20-F filed with the CDC on February 12, 2025.
Speaker Change: For additional information concerning the factors actual results to differ materially. Please see our forward looking statements section in today's press release and the risk factors section of our most recent annual report on form 20-F filed with the SEC on February 12, 2025, transcon growth hormone. Our transcon Hgh is approved in the U S by the F D. A.
Scott Smith: Transcon Growth Hormone, or Transcon HGH, is approved in the U.S. by the FDA and in the EU has received MAA authorization from the European Commission for the treatment of pediatric growth hormone deficiency.
Speaker Change: The EU has received MAA authorization from the European Commission for the treatment of pediatric growth hormone deficiency.
Scott Smith: Transcon PTH is approved in the U.S. by the FDA for the treatment of hypoparathyroidism in adults and the European Commission and the United Kingdom's Medicine and Healthcare Products Regulatory Agency have granted marketing authorization for Transcon PTH as a replacement therapy indicated for the treatment of adults with chronic hypoparathyroidism.
Speaker Change: Transcon PTH is approved in the U S by the FDA for the treatment of hyperparathyroidism in adults and the European Commission in the United Kingdom's Medicine, and healthcare products regulatory agency has granted marketing authorization for Transcon PTH is a replacement therapy indicated for the treatment of adults with chronic hyperparathyroidism.
Scott Smith: Otherwise, please note that our product candidates are investigational and not approved for commercial use. As of investigational products, the safety and effectiveness of product candidates has not been reviewed or approved by any regulatory agency. None of the statements during this conference call regarding our product candidates shall be viewed as promotional.
Speaker Change: Please note that our product candidates. Our investigation went not approved for commercial use as an investigational products the safety and effectiveness of product cat has not been reviewed or approved by any regulatory agency. None other statements. During this conference call regarding our product candidates chubby boeotus promotional on the call today, we'll discuss our first quarter 2025 financial results and will provide further business up.
Scott Smith: On the call today, we'll discuss our first quarter 2025 financial results and we'll provide further business updates. Following some prepared remarks, we'll then open up the call for questions.
Speaker Change: Following some prepared remarks, we will then open up the call for questions.
Jan Mikkelsen: With that, let me turn it over to Jan. Thanks, Scott. Good afternoon, everyone.
Yan: With that let me turn it over to Yan.
Yan: Thanks Scott.
Yep.
Yan: Good afternoon, everyone.
Jan Mikkelsen: in the first quarter of 2025. Ascendis continues the strong start of our global U.S. as well as key development and regulatory progress, supporting our long-term growth strategy to be a leading biopharma company. The strong U.S.
Yan: In the first quarter of 'twenty two.
Yan: <unk> continued the strong start.
Yan: Your address.
Yan: As well as key development and regulatory progress supporting our long term growth strategy to be a leading biopharma company.
Jan Mikkelsen: longs of our European position, 2025, to be an inflection point for us. with a growing revenue base and a clear path to become cash flow positive.
Yan: The strong U S launch of our you would pass precision 2025 to be an inflection point for assemblies.
Yan: With a growing revenue base and a clear path to become cash flow positive.
Jan Mikkelsen: As of March 31. UFOs were prescribed in the U.S. by more than 1,000 unique prescribers.
Yan: Okay.
Yan: As of March 31.
Yan: You are correct.
Yan: As prescribed in the U S by more than 1000 unique prescribers.
Jan Mikkelsen: for more than. 1750 This represents our first full quarter for the U.S.
Yan: For more than.
Yan: 1700 and.
Yan: <unk> 50 pay.
Yan: Patients.
Yan: This represented our first full quarter for the U S logs.
Jan Mikkelsen: North. Europe has the first and only FDA-approved treatment for hyperparathyroidism in adults. It's addressing the underlying cause of the disease. by providing active PTA within the physiological range for 24 hours per day.
Yan: Europe has a first and only FDA approved treatment for hyperparathyroidism in adults.
Yan: Is addressing the underlying cause of their disease.
Yan: By providing active P. G eight within the official luxury coated rings for 24 hours package.
Jan Mikkelsen: Scott Chauffeur, our long act in Guatemala. is firmly established as a high-value brand and the preferred treatment for patients, physicians, and caregivers. Scott Hofer is well positioned.
Scott Sofa: Scott Sofa.
Yan: Our long acting growth hormone is.
Yan: Is firmly established as a high value back and the preferred treatment for patient decision.
Scott Sofa: Scott Sofa is well positioned.
Jan Mikkelsen: As daily treatment continues to exit the U.S. and Scott Hofer's label has the potential to expand beyond its single indicator. Skytofa is a key pillar in our strategy to become a global leader in the treatment of rote disorder. Transcon CMP, the first long-acting therapy in development for the treatment of acondoplasia. is set to become the second pillar in our growth disorder strategy. We believe that trans-consumption... has treatment benefits in addition to linear growth that addresses multiple aspects of the condition that are fundamentally important to patients.
Scott Sofa: Our steady treatment continue to exit the U S market and.
Speaker Change: And as Scott Shaw with LIBOR has the potential to expand.
Scott Sofa: It's a good indication.
Scott Sofa: Scott is a key pillar in our strategy to become a global.
Scott Sofa: <unk> in the treatment of <unk>.
Scott Sofa: Growth disorder.
Transcon CMP.
Scott Sofa: First long acting therapy in development for the treatment of <unk>.
Scott Sofa: <unk> two became the second pillar in our growth disorder strategy.
Scott Sofa: Do you believe that transcon CMP pads.
Scott Sofa: As treatment benefits in addition to the near crude.
Scott Sofa: Addresses multiple aspect of the condition that our fundamental important to patients.
Jan Mikkelsen: We submitted an NDA to FDA in March and expect to file an MAA. with Emea in Q3 this year. Data from three randomized, double-blind, placebo-controlled clinical trials. show that Transcon CIMP has the potential to transform the lives of people with Aikondotoxin.
Scott Sofa: We submitted an NDA to FDA in March and expect to file.
Scott Sofa: Hey.
Scott Sofa: EMEA in Q3 this year.
Scott Sofa: Data from three randomized double blind placebo controlled clinical trial.
So that transparency A&P has the potential to transform the lives of people with a chronic patient.
Jan Mikkelsen: In my remarks, I will discuss each of these products in detail and comment on other recent developments within our Beginning with you. First quarter saw the global Europass revenue grow to €45 million, compared to €14 million in the fourth quarter of last year. Following commercial availability in the U.S. in December of last year, we are seeing strong U.S. demand. reflecting both the deep unmet medical need in the market, as well as the last patient population. As of March 31st, more than 1,750 patients, including the 200 patients from our EAP and clinical program, have been prescribed Europat in the U.S.
Scott Sofa: In my remarks, I will discuss each of these product and detail and comment on the recent developments with.
Scott Sofa: Our business beginning with your pets.
Scott Sofa: First quarter total global <unk> revenue growth to 45 million euros.
Scott Sofa: <unk> 214.
Scott Sofa: In Europe in the fourth.
Scott Sofa: Of last year.
Scott Sofa: Following commercial availability in the U S in December of last year.
Scott Sofa: We are seeing strong U S demand.
Scott Sofa: Reflecting both the deep unmet medical need in the market as well as the loss patient population.
Scott Sofa: As of March 31, more than 1750.
Scott Sofa: 50 patients including.
Scott Sofa: The 200 patients from our ERP and clinical program.
Scott Sofa: Be prescribed <unk> in the U S.
Jan Mikkelsen: by over 1,000 unique healthcare providers. Enrollment of patients new to Europass continues at a similar weekly rate in April.
Scott Sofa: <unk> 1000 unique health care providers.
Scott Sofa: Enrolment of patients new to your press continue at a similar rate in April.
Jan Mikkelsen: The majority of patients... who have received insurance approval for their UOPET prescription, received that approval in 4-8. and we are pleased with the approval rate we have seen. We are beginning to see favorable care plans put into place and continue to see approvals across both commercial and government plans. The strong launch performance of EUROCAT in the U.S. supports our view of its excellent product profile and the major armament need in Burma.
Scott Sofa: The majority of patients.
Scott Sofa: Who had received insurance approval for that you observed prescription received approvals.
Scott Sofa: Approval in four to eight weeks and we are pleased with that weak rate we have seen.
Scott Sofa: We are beginning to see favorable claims put into place and continues to see approvals across both commercial and government.
Scott Sofa: The strong launch performance of your cut in the U S support our view of its excellent product profile and the major.
Scott Sofa: Medical need in memory.
Jan Mikkelsen: And we expect Europass to contribute significantly to our revenue in 2020. Outside of the U.S., we see steady European revenue growth in both the European direct and international markets. And we expect additional acceleration of the revenue growth when your reimbursement becomes available in additional Europe direct countries in the second half of the year. The continued rapid uptake together with high rates of patient adherence give us confidence that Europe is well positioned to uniquely address the unmet medical need of this patient population. And we are regularly reviewing input and data from patients to evaluate if there are additional ways to improve the treatment profile even more.
Scott Sofa: And we expect <unk> to contribute significantly to our revenue in 2025.
Scott Sofa: Outside of the U S. We see steady revenue growth in both the Europe direct and international markets.
Scott Sofa: And we expect additional acceleration of the revenue growth when your cost.
Scott Sofa: Reimbursement becomes available in additional Europe direct countries in the second half of the year.
Scott Sofa: The continued rapid uptake took a little bit higher rate of patient adherence give us confidence that <unk> is well positioned to uniquely address the unmet medical need.
Scott Sofa: This patient population and we are regularly reviewing input and data from patients to emulate if they are at.
Scott Sofa: Additional ways to improve the treatment profile even more.
Jan Mikkelsen: We estimate there are over 400,000 patients globally. and around 70 to 90,000 patients in the US alone living the chronic hyperparasite. Our claims analysis demonstrate that. to 15,000 of these US patients are uncontrolled and 30 to 35,000 are partly controlled. Based on the latest clinical practice guidelines, nearly all these patients are candidates for treatment with U.S. Our strong global loans give us high conviction that we can continue to build and lead this market. And Europat can be a doable multi-billion euro drug device product with a patent lifespan extending into the 2040s.
Scott Sofa: We estimate that for.
Scott Sofa: 400000 patients globally.
Scott Sofa: And around 70.
Scott Sofa: 290000 patients in the U S alone, giving the chronic hyperparathyroidism.
Scott Sofa: Our claims analysis demonstrates that too.
Scott Sofa: <unk> 15000 of these U S patient uncontrolled.
Scott Sofa: 30 to 35000 are partially controlled.
Scott Sofa: Based on the latest clinical practice guideline yearly all these patients are candidates for treatment with your pets.
Scott Sofa: Our strong global launch give us high conviction that we can continue to build and lead this market and Europe. It can be a dual boot shipbuilding Europe drug device product with a patent life span extending into 2014.
Jan Mikkelsen: Turning to Skype. Q1 revenues for SkyTofa were €51 million. with continued patient growth and global expansion, offset by the typical first quarter revenue dynamics in the UK. We have around 7% market share of the total growth hormone market in the US and around 43% of the total US non-acting growth hormone market based on third party prescriptions. The Pediatric Growth Deficit Indication represents about half of the total growth.
Scott Sofa: Okay.
Scott Sofa: Turning to schedule.
Scott Sofa: Q1 revenues, plus cutover, where <unk>.
1 million euros with.
Scott Sofa: With continued patient growth and global expansion offset by the typical first quarter revenue dynamics in the U S. We.
Scott Sofa: We have around 7% market share of the total <unk> market in the U S and around 43% of the total U S from excellent growth in Mumbai.
Scott Sofa: <unk> of third party prescription data.
Scott Sofa: The pediatric growth deficient indications represent about half of the total glaucoma market.
Jan Mikkelsen: with our premier prize. and Scott Hofer's leading position in Plenatico Tumult. We believe we are well positioned to expand the opportunity for Skype to offer in multiple languages. A key near-term milestone is our first potential label expansion in the established growth hormone indications for our supplement BLA for the potential U.S. approval in adult growth. where we have a PDUFA Goal Date of July 27, 2021. We are also on track to start a basket trial for Skotthofe in a range of injuries. including idiopathic short stick. Chuck Turner Syndrome, and SGH. We are planning to discuss this trial with the FDA in an end-of-phase 2 meeting this quarter.
Scott Sofa: We don't premium pricing.
Scott Sofa: Cutoff with leading position in pediatric growth hormone deficiency. We believe we are well positioned to expand the opportunity for Skype for firm in Portugal rates at.
Speaker Change: Key near term milestone is our first potential label expansion in the established Kokomo indications.
Scott Sofa: <unk>.
Scott Sofa: Our supplemental BLA for the potential U S approval in adult growth hormone deficiency.
Scott Sofa: Wherever you happen to do physical date of July 27, 25.
Scott Sofa: We are also on track to startup basket trial Saskatchewan.
Scott Sofa: At rates of indications, including.
Scott Sofa: <unk> headache short stay too.
Scott Sofa: Deficiencies fair enough syndrome and SGA.
Scott Sofa: We are planning to as discussed this trial with the FDA.
Scott Sofa: And in a phase two meeting this quarter.
Jan Mikkelsen: Importantly, we are also investigating transcontractive hormone outside the established growth hormone hormone indicator.
Scott Sofa: Importantly.
Scott Sofa: We are also investigating transcon growth hormone outside their established Kokomo.
Jan Mikkelsen: such as in a potential combination therapy with Transcon CMP for treatment of aconteplasia and other growth disorders, which I will address in a moment.
Scott Sofa: Indications.
Scott Sofa: As in a potential combination therapy with transcon CMP for treatment of echo to Appalachia, and other growth disorder, which I will address in a moment.
Jan Mikkelsen: Moving to Tradcom CMP. Triton CMP is the third key product in our industry. Endocrinology Rare Disease Product Performance. The genetic variant that caused back contamination.
Scott Sofa: Moving to attract CMP.
Scott Sofa: Transcon CMP is the third key product into.
Scott Sofa: Endocrinology rare disease product portfolio.
Scott Sofa: With genetic variant that cost quantification.
Jan Mikkelsen: Change the way we accept our work in multiple throughout the book. not just in the growth plate and in Resulting in a wide range of serious medical complications in childhood and lasting throughout adulthood. Because Transcon CMP provides sustained therapeutic levels of CMP throughout the body, it has been Demonstrated and unique product profile. Giving it the potential to bring growth benefits and important new benefits beyond linear growth, as well as reduce risk of hypertension and injection site risk. In our peer-reviewed approach trial, Transcon CMP demonstrated significant improvement in the primary input of linear growth compared to placebo.
Scott Sofa: <unk> receptor work in multiple tissues.
Scott Sofa: Throughout the body.
Scott Sofa: Not just in the growth rate and imports.
Scott Sofa: <unk> in a wide range of serious medical communication and childhood endoscopy to adapt.
Scott Sofa: Because <unk> CMP provides sustained <unk> levels of CRP throughout the body.
Scott Sofa: Has been.
Scott Sofa: Demonstrated and unique product profile given is the potential to bring.
Scott Sofa: Both benefit and important new painting beyond the newco.
Scott Sofa: Esque reduced risk of hypertension and injection site reaction.
Scott Sofa: In our period approached trial transport CMP demonstrated significant improvement in the primary input of linear growth compared to placebo as well as significant improvement in other kidney code endpoint meaningful to the economic case of community, including <unk>.
Jan Mikkelsen: as well as significant improvement in other clinical endpoints, meaningful to the acondoplasia community, including leg bowing, muscle functionality, body proportionality, and health-related quality of life. Leg bowing is a common complication in a chondrocase that can result in chronic pain and impaired physical health. driving many to undergo complex painful corrective surgery. I have been in meetings with patient organizations in Europe and the US who have confirmed the importance of addressing the complications of outgoing pressure beyond linear flow. Just as important to the aconopasia community, Transcon CMP has shown a safety and durability compared to placebo, with low frequency of injection-type reactions, all which were mild and no evidence of symptomatic hypertension.
Scott Sofa: Boeing muscle functionality body appropriate analogy and health related quality of life.
Scott Sofa: <unk> is a common complication in a corner cases that can result in chronic pain and EPS accretion could function driving many to undergo complex painful corrective surgeries.
Scott Sofa: I have been in meetings with patient organizations in Europe, and the U S. We have confirmed the importance of addressing the complication of Alcon paycheck PR.
Scott Sofa: Okay.
Speaker Change: Just as important to the kind.
Speaker Change: The patient community Transcon CMP has shown a safety and tolerability profile comparable to placebo with low frequency of injection site reactions, all of which were mild and no EBIT of symptomatic hypotension.
Jan Mikkelsen: After positive interaction with the FDA relating to the content of our NDA submission, we are pleased to have submitted Transcon CMP for the review in March. In the EU, we plan to submit an MAA during the third quarter of this year.
Speaker Change: After a positive interaction.
With the ft relating to the content of our NDA submission be please to have submitted transcon CMP product review in box in the EU, we plan to submit an IND.
Speaker Change: During the third quarter of this year.
Jan Mikkelsen: Additionally, during the fourth quarter of 2025, we plan to submit an R&D or similar to investigate transcon CMP or in combination with transcon growth normal for the treatment of hypochondriation.
Speaker Change: During the fourth quarter of 2025, we plan to submit an IND or similar to investigate transcon CMP or incomplete nation, but transcon growth hormone for the treatment of hydro crude rotations.
Jan Mikkelsen: Shifting to Transcon CEMP and Transcon Glotamone Combination Therapy. We are committed to continue to drive even better outcomes for people living with acondymia. This is why we are conducting.
Speaker Change: Shifting to transcon, CMP and transcon growth hormone combination therapy.
Speaker Change: We are committed to continue to drive even better outcome for people living with echo location.
Speaker Change: This is why we are conducting the trial.
Jan Mikkelsen: being the first phase 2 study combining CMP and growth hormone in acondoplasm. Each of which stimulates different signaling pathways in the growth plate and other tissue in the body. We look forward to sharing top line week 26 results from the Coach Tri data set. and seek great potential to further raise the bar for clinical outcomes. With Transcon CMP as the potential future backbone therapy, we believe we can achieve even greater growth while also addressing the medical complication of air contamplation.
Speaker Change: Being the first phase III study, combining CMP and growth hormone in <unk> locations.
Speaker Change: Each of which stimulates different signaling pathways in the crude fleet and other tissue in the body.
Speaker Change: We look forward to sharing topline 2006 results from the coach trial data this quarter and see great potential to further raise the bar for clinical outcome.
Speaker Change: With Transcon CMP.
Speaker Change: The potential future backbone therapy.
Speaker Change: We can achieve even greater growth, while also addressing medical complication of co location.
Jan Mikkelsen: Fundamental to the development of ease-of-our-treatment medicine, Uopat®-Scotopher® Transcon CMP is Ascendis' proprietary Transcon technology platform. With the transcon technology and our deep understanding of disease biology, it is possible to create medicine with highly differentiated treatment benefits not possible with other technologies.
Speaker Change: Fundamental to the development of each of our three major see your pad scheduled for Transcon CMP is ice ages, Papa Terra Transcon technology platform.
Speaker Change: With the Transcon technology, and our deep understanding of <unk> biology, it is possible to create medicine with highly differentiated treatment benefit not possible with other technologies.
Jan Mikkelsen: At Ascendis, our commitment has always been to the people. It is one of the company's core values. I believe we have demonstrated multiple times over the history of Ascendis our resilience and our ability to adapt and find solutions to attain this goal. We remain as dedicated as ever to ensure that all our medicines become available. to our collaboration with Novo Nordisk for the development and commercialization of Transcon technology-based products in metabolic and cardiovascular disease. and our collaboration partners, Veezen, Iconis and Tideink, we continue to work to execute our Vision 2030 to create value in large therapeutic areas and to innovative business models.
Speaker Change: At ascend is our commitment has always been to the patient.
Speaker Change: It is one of the company's core values.
Speaker Change: We have demonstrated multiple times over the history of the cities are with students and our ability to adapt and find solutions to attain this goal.
Speaker Change: We remain as dedicated as ever to ensure that all our <unk> become available to patients.
Speaker Change: Through our collaboration with Novo Nordisk for the development and commercialization of Transcon technology based product and metabolic and cardiovascular disease.
Speaker Change: Our collaboration partner <unk>.
Speaker Change: Iconix and Turkey.
Speaker Change: Continue to work to execute our vision 2000, <unk> to create value and dusk to approaching <unk> and two innovative business model.
Jan Mikkelsen: In summary, 2025 is a transformative year for Ascendis as we grow our global revenues from Europat, SkyTrova, and seek to obtain key regulatory approvals, deliver robust clinical data, and advance stocks with blockbuster potential to drive growth for many years to come.
Speaker Change: In summary <unk>.
Speaker Change: 2025 is a transformative <unk> SP growth, our global revenues from Europe.
Scott Sofa: Scott Sofa.
Scott Sofa: And seek to obtain key regulatory approvals deliver robust clinical data and then bank stocks with blockbuster potential to drive growth for many years to come.
Scott Smith: I will now turn to Scott. Thank you so much, Jan. I will touch on some key points surrounding our first quarter financial results, but for further details, please refer to our 6K file today. Europath's first quarter revenue increased significantly to 44.7 million euro, up from 13.6 million euro in the fourth quarter of 2024. Steady sequential revenue growth outside the U.S. was augmented by the strong U.S. launch. As Yen discussed, the trends we saw for Yorvapath in Q1 continue in Q2 both outside and inside the U.S. And we anticipate that Yorvapath will have a substantial impact on our financial profile in 2025.
Scott Sofa: I will now turn to Scott.
Scott: Thank you so much.
Scott: I will touch on some key points surrounding our first quarter financial results, but for further details. Please refer to our 6K filed today.
Scott: Yoga path first quarter revenue increased significantly to $44 7 million euro up from $13 6 million Euro in the fourth quarter of 2024.
Scott: Steady sequential revenue growth outside the U S was augmented by the strong U S launch.
Scott: As <unk> discussed the trends we saw for Yorvit path in Q1 continue in Q2, both outside and inside the U S and we anticipate that yoga path, we will have a substantial impact on our financial profile in 2025.
Scott Smith: Turning to Skytrofa, revenue for this quarter was 51.3 million euros. Sequentially, pricing and market share remained stable, but revenue in the U.S. was negatively impacted by seasonal items, including reduction in channel inventory and higher copay assistance. Those seasonal headwinds should reverse beginning in Q2. We also continue to watch the Euro-dollar exchange rate for any potential impact related to reported revenue.
Scott: Turning to Sky profile revenue for this quarter was $51 3 million Euro <unk>.
Scott: Sequentially pricing and market share remains stable, but revenue in the U S was negatively impacted by seasonal items, including reduction in channel inventory and higher copay assistance. So.
Scott: Those seasonal headwinds should reverse beginning in Q2.
Scott: We also continue to watch the Euro dollar exchange rate for any potential impact related to reported revenue.
Scott Smith: Total revenue for the first quarter was €101 million, which includes non-product revenue from our collaboration partner. Turning to expenses, for the first quarter, R&D costs totaled 86.6 million euro compared to 70.7 million euro during the first quarter of 2024. The first quarter of 2024 included a favorable 10.6 million euro reversal of prior period write-downs of Transcon PTH pre-launch inventories. SG&A expenses in the first quarter of 2025 totaled €101 million, compared to €66.8 million during the first quarter of 2024. The €34 million increase was primarily driven by global commercial expansion.
Scott: Total revenue for the first quarter was $101 million Europe, which includes non product revenue from our collaboration partners.
Scott: Turning to expenses for the first quarter R&D costs totaled $86 6 million euro compared to $70 7 million Euro during the first quarter of 2020 for the first quarter of 2024 included a favorable $10 6 million reversal of prior period write downs of Transcon PTH.
Scott: Prelaunch inventories.
Scott: SG&A expenses in the first quarter of 2025 totaled $101 million euro compared to $66 8 million Europe during the first quarter of 2024.
Scott: The 34 million Euro increase was primarily driven by global commercial expansion.
Scott Smith: Total operating expenses were €188 million for the first quarter of 2025.
Scott: Total operating expenses were 188 million euros for the first quarter of 2025.
Scott Smith: As a result of the Visa IPO, we recognized a non-cash gain of €33.6 million as part of share of profit loss of associates, and we retained 39% ownership of Visa. Net finance expenses for the first quarter of 2025 were €15.9 million, driven primarily by non-cash items. Net cash financial income for the first quarter of 2025 was €3.3 million. We ended the first quarter of 2025 with cash and cash equivalents totaling 518 million euro compared to 560 million euro as of December 31, 2024.
Scott: As a result of the visa IPO, we recognized a noncash gain of $33 6 million euro as part of share of profit loss of associates.
Scott: And we retained 39% ownership of visa.
Scott: Net finance expenses for the first quarter of 2025 were $15 9 million Euro driven primarily by noncash items.
Scott: Net cash financial income for the first quarter of 2025 was $3 3 million Euro.
Scott: We ended the first quarter of 2025 with cash and cash equivalents totaling $518 million euro compared to $560 million Euro as at December 31, 2024.
Scott Smith: Turning to the remainder of 2025, we expect substantial revenue growth driven by the global launch of YorvaPath with a continued solid contribution from Skytrofa. We are not providing revenue guidance for SkyTrofa or UruPath at this time. For Skytropha, we believe that growth in prescriptions, visible in third-party data, will track sequential revenue growth for 2025 with expected stability and mix in pricing, including normal seasonality. For Yoruba Path, outside the U.S., we continue. Steady Revenue Growth. While inside the US, our launch is progressing exceptionally well. We will continue to look to help investors understand uptake and reimbursement dynamics as the year progresses.
Scott: Turning to the remainder of 2025, we expect substantial revenue growth driven by the global launch of yours, a path with a continued solid contribution from sky.
Scott: We are not providing revenue guidance for Skype or you are a path at this time.
Scott: <unk>, we believe that growth in prescriptions visible and third party data will track sequential revenue growth for 2025 with expected stability in mix and pricing, including normal seasonality.
Scott: For you over the past outside the U S. We continue.
Scott: Steady revenue growth, while inside the U S. Our launch is progressing exceptionally well.
Scott: We will continue to look to help investors understand uptake in reimbursement dynamics as the year progresses.
Operator: With that, operator, we are now ready to take questions. Thank you. As a reminder, to ask a question, please press star 11 on your telephone and wait for your name to be announced. And to withdraw your question, please press 11 again. We do ask that you please limit to one question and one follow-up to allow time for everyone to ask a question.
Speaker Change: With that operator, we are now ready to take questions.
Speaker Change: Thank you as a reminder to ask a question. Please press star one on your telephone and wait for your name to be announced and to withdraw. Your question. Please press. One again, we do ask that you. Please limit to one question and one follow up to allow time for everyone to ask a question.
Jessica Fye: And our first question today will come from Jessica Fye with J.P. Morgan. Your line is open. Hey guys, good afternoon. Congrats on the strong results with URV-PATH. It seems like the number reflects very good execution on reimbursement. Can you talk about your latest expectation for the proportion of patients with a script who you think will ultimately get reimbursed once URV-PATH is, say, at steady state? And then the follow-up would just be, was there any initial channel fill reflected in the 1Q revenue number? And if so, can you quantify that? Thank you. Thanks, Jess, for the questions.
Speaker Change: And our first question today will come from Jessica Fye with Jpmorgan. Your line is open.
Jessica Fye: Hey, guys. Good afternoon, congrats on the strong results you already passed.
It seems like a number reflects very good execution on reimbursement can you talk about your latest expectation for the proportion of patients with a script for you think will ultimately get reimbursed one CRE path, let's say at steady state and then the follow up would just be was there any initial channel fill reflected in the <unk> revenue number and if so can you quantify that thank you.
Jessica Fye: Okay.
Scott Smith: And Scott will take the easy one, the last one.
Jessica Fye: Thanks, Jess for vacations, and Scott to take the easy one the last one.
Scott Smith: Yeah, so channel inventory is has averaged about one to two weeks at any one time. We ship once a week, so it's hard to get less than one week of channel.
Speaker Change: Yeah, Jeff So channel inventory has averaged about one to two weeks at any one time, we ship once a week. So it's hard to get less than one week of channel.
Jan Mikkelsen: The second one, and just why it's a little bit more complicated, because there's somebody looking in the future, what we can see, we see a very, very, very positive trend, we see the adaption of the different PPMs, the different payer forms and if I'm some way should come with my own guess about it and this is more a guess because we don't know exactly. There will never be 100% approval of all this.
Jessica Fye: The second one.
Jessica Fye: And just why it's a little bit more complicated because somebody looking in the future.
Jessica Fye: What we can see we see <unk> constitute a trend we see the adoption of the different <unk> that different payer.
Jessica Fye: <unk> and <unk>.
Jessica Fye: If somebody should come with my own guess about it and this is more I guess, because we don't know exactly.
Jessica Fye: There will never be 100% approval of all the patients and I believe if we are successful.
Jae Woo: And I believe if we really are successful, and I hope we won, and I believe we're going to be successful, I believe 17-18% will get reimbursed, but I think Jay, if you have another comment to it, this way. Yeah, happy to chime in on that. I think as Jan mentioned, we are still early in the launch, right? So from that perspective, we're still seeing policies be set into place. So it is a bit early to be able to anticipate what that steady state will be. I do think a few examples that give us a lot of confidence that we are headed in the right direction is a couple.
Jessica Fye: And I hope we've won.
Our belief is going to be successful I believe.
Jessica Fye: Seven 2% to 18% will get reimbursed, but I think Jay if you have another comment to it this way.
Jay: Yes happy to chime in on that.
Speaker Change: I think as Jan mentioned, we are still early in the launch right. So from that perspective, we're still seeing.
Speaker Change: Policies VSAT into place. So it is a bit early to be able to anticipate what that steady state will be I do think a few examples that give us a lot of confidence that we are headed in the right direction is a couple one youre seeing policies favorable policies be put into place both from a commercial standpoint as well as Gov.
Jae Woo: One, you're seeing policies, favorable policies be put into place, both from a commercial standpoint, as well as government. So we're seeing that start to take hold. And then two, even absent of a formal policy in place, we do see patients get approved across the board, again, agnostic of whether it is a commercial plan or government plan. It just sometimes may take a few more steps, whether it is through medical exception, and in some rare cases, even through appeal and or peer to peer. But largely, we are seeing many patients get through. You will expect a long tail, just given that every payer plan is different, given the heterogeneity in the US.
Speaker Change: So we're seeing that start to take hold and then too.
Speaker Change: Even absent of a formal policy in place we do see patients get approved across the board again agnostic of whether it is a commercial plan or government plan. It just sometimes may take a few more steps whether it is through medical exception and in some rare cases, even through appeal and or peer to peer but <unk>.
Speaker Change: Largely we are seeing many patients get through.
Speaker Change: You will expect a long tail just given that every payer plan is different given the heterogeneity in the U S. But overall, we have a lot to be encouraged about and I think more importantly, it just really emphasizes the clinical value proposition of this product and the fact that payers providers and patients are.
Jae Woo: But overall, we have a lot to be encouraged about. And I think more importantly, it just really emphasizes the clinical value proposition of this product, and the fact that payers, providers, and patients are all responding.
Speaker Change: Responding to it.
Jan Mikkelsen: I think, but yes, I think. Some way when I talk about my My feeling is that I believe every patient that some way have a desire to go on treatment should come on treatment because I have seen or heard med patients that benefit that is on the treatment in it. But I also accept reality in this way here. But I also believe we are in a unique position. We are addressing a major unmet medical need. We have really a product that really are providing the benefit to the patient. We are in a position that patients are diagnosed with the disease.
Speaker Change: I think but yes, I think so.
Speaker Change: When I talk about my feeling our feeling is that I believe every patient that somebody have a desire to go on treatment should come on treatment because I've seen a hook mid patients that benefit there is on the treatment in it but also except we added in this way here, but also pay.
Speaker Change: Leaf we are in a unique positioning.
Speaker Change: Be addressing a major unmet medical need.
Speaker Change: Really a product that we are providing the benefit to the patient we are.
Speaker Change: In a position that patients are diagnosed with the disease at the same time <unk> clear.
Jan Mikkelsen: At the same time, there is clear guidelines that are saying there should be a change. Can you find a better case? for any patient. really not to get the ultimate goal if you get and what to be of treatment you should be on.
Speaker Change: I would say that should be a treatment can you find a better case.
Speaker Change: For any patients.
Speaker Change: Really not to get but ultimate code, if you get and what to be off treatment you should be on treatment.
Tazeen Ahmad: And our next question comes from Tazeen Ahmad with Bank of America Securities. Your line is open. Hi, good afternoon. Thanks for taking my question.
Ahmed: And our next question comes from <unk> Ahmed with Banc of America Securities. Your line is open.
Hi, Good afternoon. Thanks for taking my question again can you give us a little bit of color on that.
Jan Mikkelsen: Yen, can you give us a little bit of color on the splits? U.S. and ex-U.S. Revenue for the Quarter for URDPAS. And can you give us a sense, because it seems like you do have... Union Medical Center, Inc., Marjorie Highcourt,rar Therapy.
Ahmed: Blitz between U S and ex U S revenue for the quarter.
Ahmed: Are you already passed and can you give us a sense because it seems like you have some patients receiving drug in the U S. What the length of time is I know, it's early but what is the length of time that you are seeing between when his script is written for one of the patients receiving therapy.
Jan Mikkelsen: Yeah, let me first address the question between, the split between Europe and US. And as you have seen, Tazeen, we are not really describing that in our numbers. but also believe that we give you good guidance how to potential to calculate. As we said, when we look on the ex-US... We see a steady growth, a steady acceleration, but we first expect in the second half of this year to see an acceleration after acceleration when we get more countries basic into the situation that will be fully reimbursed. So when I look on the difference between Q3 and Q4 last year, it's something around the element of an increase of around 4 to 5 million euro in for one quarter to the next year.
Ahmed: Yes.
Ahmed: Yeah, Let me first address the.
Ahmed: Christian team perspective.
Ahmed: Europe and U S and as you have seen we are not really describing that.
Ahmed: Nonetheless.
Ahmed: But also believe that we give you good guidance how to potential to calculated.
Ahmed: As we said.
Ahmed: When we look on the ex U S.
Ahmed: We see a steady.
Ahmed: A steady acceleration.
Ahmed: What we first expected in the second half of this year to see an acceleration of the exploration when we get more countries basic into best situation that will be fully reimbursed. So when I look on the difference between.
Ahmed: Q3, and Q4 last year is something around.
Ahmed: The element of an increase around 4% to 5 million euro in one quarter to the next quarter or.
Jan Mikkelsen: So when you think about that, we increased our. basic the revenue from about. 14 to 45 then you can take basic the difference and then subtract about four or five million and then I think you would get that number around 26 million which are some way reflecting the algorithm I would use if I should look at it.
Ahmed: So when you think about that we increased our <unk>.
Ahmed: Basic the revenue.
Ahmed: From about.
Ahmed: 2014 to 45, but then you can take.
Ahmed: They seek.
Ahmed: The difference and then subtract about <unk> 5 million a day, not saying you would get at around 26 million Whipsaw Sunday, reflecting accurate I would use if I should look at it.
Gavin Clark: And our next question will come from Gavin Clark Gartner with Epicor.
Gavin Clark: And our next question will come from Gavin Clark Gartner with Evercore. Your line is open.
Jan Mikkelsen: Your line is open. Hey, guys, congrats on the very strong launch progress. First, just to follow up on the payer point and the favorable access. Have you finished negotiations with the majority of commercial payers at this point? And do you respect the remaining to be published policies to look similar to the ones that are already published? And has the rebating fallen in in line with your expectations with those conversations? Yeah, it's a question.
Gavin Clark: Hey, guys. Congrats on the very strong launch progress.
Gavin Clark: First just a follow up on the payer point and the favorable access.
Gavin Clark: Have you finished negotiations with the majority of commercial payers at this point and do respect the remaining to be published policies to look similar to the ones that are already published and has the rebating falling in line with your expectations with those conversations.
Jan Mikkelsen: I will take the global perspective. As you've heard about Ex-US, we are basic, are Fully reimbursed now Germany, there is a final list price you can find, we also have that for Austria, in France we have an AP2 program, we can also have a different program in Greece. So, Ex-US, we are working on really getting all country by country fully reimbursed. It takes time. It's something that some way both will happen here in 25, but also in 26, when we come into end of 26, we believe we will have the vast majority of the important country being fully reimbursed.
Gavin Clark: Yes.
Gavin Clark: Christian I will take a global perspective, as you heard about ex U S.
Gavin Clark: Basic.
Gavin Clark: Fully reimbursed now Germany.
Gavin Clark: As a final this price you can find.
Gavin Clark: We also have that for Australia.
Gavin Clark: In France, we have an API two program.
Gavin Clark: We can also have a different program.
Speaker Change: So ex U S.
Speaker Change: Working on really getting all country by country fully reimburse it takes time, it's something that some way.
Speaker Change: <unk> hundred 95.
Speaker Change: In 2006 when become into end of 2006, we believe we will have that waste majority of their important country.
Jan Mikkelsen: If you come to the US, which there is a strong focus on, don't forget the more than 300,000 patients outside US.
Speaker Change: Reimbursed.
Speaker Change: If you come to the U S, which drives a strong focus on.
Speaker Change: Cricket the more than 300000 patient outside U S.
Jae Woo: I think Jay can give you more color about his discussion with the different plans in the US. Thanks, Jan. And thanks, Gavin, for the question. Yes. So, just to answer your question, the conversations with payers have been going very well. We have multiple commercial policies in place with our national accounts. So while we don't comment on policy by policy, that gives you a sense that we are gaining a lot of good traction there, and the policies that are put in place are favorable and consistent with label. While we don't comment on the gross net arrangements that we have with each, what I will say is that it is commensurate with what we said before.
Speaker Change: I think Jay can keep you more color about his discussion with the different.
Speaker Change: Clients in the U S.
Speaker Change: Thanks, Jen and thanks, Kevin for the question yes.
Speaker Change: Answer your question the conversations with payers have been going very well.
Speaker Change: We have multiple commercial policies in place with our national accounts. So while we don't comment on policy by policy that gives you a sense that we are gaining a lot of good traction there and the policies that are put in place are favorable and consistent with label.
Speaker Change: While we don't comment on the gross to net arrangements that we have with each what I will say is that it is commensurate with what we said before this is a first and only approved products and a rare disease setting with clinical value benefit that we strongly believe in and we feel we've been able to convey with payors and from that lens. It is commensurate with.
Gavin Clark: This is a first and only approved product in a rare disease setting with clinical value benefit that we strongly believe in, and we feel we've been able to convey with payers. And from that lens, it is commensurate with the incredible clinical value proposition that we bring, and we certainly lead with that and expect it to remain. Okay, great.
Speaker Change: The incredible clinical value proposition that we bring in we certainly lead with that and expect it to reflect that.
Gavin Clark: And then quick follow up for the new prescriptions that are coming in, what proportion are NAPARA or PTH naive versus experienced? Thank you. I don't think we really have the full insight exactly how many deaths coming from the NADPAR. Sherry, you can potential correct me if we have got more insight to that. There was some question we discussed last week. We know that the vast majority of patients coming from conventional therapy, but perhaps Sherry you can discuss exactly how many NADPAR patients that we believe has been transferred over.
Speaker Change: Okay, Great and then quick follow up for the new prescriptions that are coming in what proportion are at par or PTH naive versus experience. Thank you.
Speaker Change: I don't think we really have the full insight exactly how many tests coming from the net par.
Speaker Change: Sure you can potential correct me if we have got more insight to that there was some question we discussed last week.
Speaker Change: Note that the waste majority of patients coming from conventional therapy, but perhaps share you can.
Speaker Change: Exactly how many <unk> patients that we believe has been transferred over.
Sherry Glass: Yes, thanks for the question, Gavin. And yes, Jen's right. We, the data is not entirely definitive on this point. But we know that we have, the vast majority of our patients overall are coming from conventional therapy. So that's very clear. And we do have, you know, something like 10 to 15% of patients that have been on some sort of PTH in the past. And then, you know, a subset of those were more recently on that But the bottom line is, you know, overwhelmingly coming from conventional therapy. Yeah, from that perspective, we believe that in Q2 and Q3, we will see the majority of the rest of the NAPA patients coming over because, Aimee, you're sitting with the patients, you can explain that.
Gavin Clark: Yes, thanks for the question Gavin and yes, yes right.
Gavin Clark: The data is not entirely.
Gavin Clark: Can you give on this point, but we know that we have the vast majority of our patients overall are coming from conventional therapy, So thats very clear.
Gavin Clark: And we do have.
Gavin Clark: It's something like 10% to 15% of patients that have been on some sort of PTH in the past and then.
Gavin Clark: A subset of though it's more and more recently on nap here, but the bottom line is overwhelmingly coming from conventional therapy.
Gavin Clark: Yes from that perspective, we believe that in Q2 and Q3, we will see.
Gavin Clark: George.
Gavin Clark: The rest of the unmet patient coming over because.
Gavin Clark: Youre sitting with patients you can explain that bags.
Jan Mikkelsen: letter coming out explaining that you are in a position that they are stopping. There's still, as far as we know, there's still some supply, so they still may be on it, but we know it will expand soon. So the basic, when we look at the patient that's coming over, the 1,750, this is not a power patient. This is basically a patient that's coming from conventional therapy. There's 35,000 patients we are addressing now. So there is a lot of deepness to go in this.
Gavin Clark: That are coming out explaining that you're positioned at that stopping.
Gavin Clark: They are still as far as we know there are still some supply.
Gavin Clark: Yes.
Gavin Clark: Yes.
Gavin Clark: So the basic when we look at the patient that's coming out the one.
Gavin Clark: 1750, <unk>. This is not an empowered patient basis basic patient that's coming from conventional therapy with 35000 patients you're addressing that.
Gavin Clark: So a lot of the nuts to go in in this way.
Jan Mikkelsen: Great, congrats on the progress.
Gavin Clark: Great and congrats on the progress.
Derek Archila: And our next question comes from Derek Archila with Wells Fargo. Your line is open. Yeah, congrats on the quarter. And thanks for taking the questions.
Derek: And our next question comes from Derek <unk> with Wells Fargo. Your line is open.
Speaker Change: Hey, guys congrats on the quarter.
Derek Archila: So maybe just first on, can you comment on the depth of prescribing and the number of docs that are really prescribing, you know, maybe two, three, four patients with your VPath? And then just to follow up with the last set of questions, just, you know, are you seeing patients that are newly diagnosed or well controlled on conventional therapies move over to your VPath? Thanks.
Speaker Change: Thanks for taking the question. So maybe just first on <unk> can you comment on the depth of prescribing and the number of docs that are really prescribing.
Speaker Change: Maybe two or three for patients.
Speaker Change: Would you ever be path and then just a follow up to the last set of questions.
Speaker Change: Are you seeing patients that are newly diagnosed are well controlled on conventional therapies move all of our T or b pass. Thanks.
Jan Mikkelsen: Yeah, if I just could answer all your questions, I will be a happy person. I'm still a happy person, but when I some way and share, you can also some way add in. We cannot really know if there are. well controlled, partly controlled, or non controlled. That is not really anything we can see. We don't believe that it's really part of the reimbursement system. I actually think why we call them uncontrolled, we likely will see more of them, because we had an algorithm that basically are saying that 10 to 15,000 of the patients we did find as uncontrolled is because they're seeing an endo in a very, very, very high frequency.
Speaker Change: Yes.
Speaker Change: If I just could I answer all your questions either be unhappy postmen and Stella.
Speaker Change: But.
Speaker Change: When somebody sure you can also somebody adding.
Speaker Change: We cannot really know if there are.
Speaker Change: <unk>.
Speaker Change: Well controlled cost controlled or not controlled that is not really anything we can see we don't believe that is really part of the reimbursement system in accessing.
Speaker Change: <unk> Singh why we called them uncontrolled feedback UBC more of them because we have an algorithm that basically you are saying that 10 to 15000 of the patient we did fine as uncontrolled is because we're seeing and into in our Bev hi treatments and do us.
Jan Mikkelsen: And that was why we know that we just went to this physician that will automatically have all these patients coming in. So when they're coming and control patients that see the endo on much, much less frequency, they're getting on NAPA. I hope so, but I cannot really know. But we know we really addressed the patients that see the endo in high frequency. And I believe that is basically. I would call the genius part of our commercial organization really working on not getting patient into the endos. It takes too long time as if I want to go into Aimee's practice in Stanford, I need to wait three months to get an appointment with her.
Speaker Change: Why we knew that just went through disposition.
Speaker Change: Automatic pass all this patient coming in so when they are coming and controlled patients that see the into on must must less treatment bid.
Speaker Change: Getting on net par I hope, so, but I cannot really new but we know we really addressed.
Speaker Change: Patient, that's EBIT into and high frequency and I believe that is basic.
Speaker Change: I would call the genius part of our commercial organization really working are not gating patient into the induce it takes too long time as if I want to go into Amy.
Speaker Change: Practice, and Stanford I need to wait three months to get an appointment with us and I think we cannot wait for patient for that and give us a IV I think they made a really really genius move and the commercial organization go to places, where we know patient comes in <unk>. So my expectation is that I believe we.
Jan Mikkelsen: And I think we cannot wait for patient for that. And it was why I think they made a really, really genius move in the commercial organization, go to the places where we know patient comes in very, very, very far. So my expectation is that I believe we have a high percent of uncontrolled, partly controlled, but it's mainly only built on the algorithm because they're seeing the endo much more often than the other ones.
Speaker Change: Half percent.
Speaker Change: Percent off on controlled party controlled but it's mainly only accurate because we're seeing the into mass more often other ones. So sure do you have anything to add.
Sherry Glass: So Sherry, do you have anything to add? I think you covered it well. Again, I would maybe just say we one of the things we're really excited about is we we know they're quite a good healthy number of those uncontrolled and partially controlled patients. So something like ten to fifteen thousand uncontrolled and another thirty thousand or so partially controlled. And as you said, the uncontrolled patients are seeing their endos four times or more a year. So we know to Jan's point that they're getting in and therefore that we expect to see a steady flow of patients coming in and having the option to get on drug over the course of the year.
Speaker Change: I think you covered it well again I would maybe just say one of the things. We're really excited about is we know there are quite a good healthy number of those uncontrolled and partially controlled patients so something like 10 to 15000.
Speaker Change: Uncontrolled and another 30000 ourself, partially control then as Yan said that the uncontrolled patients are seeing their end dose four times a year. So we know the key endpoint that theyre getting in and therefore expect to see a steady flow of patients coming in and.
Speaker Change: And having the option to get on drug over the course of the year.
Yaron Werber: Yeah, and one of the reasons why we don't know it is because it's not part of being reimbursed or not. So if we're part of the reimbursement system to have this uncontrolled, partially controlled or controlled, we would know much more on it. But it's not a decision in the reimbursement.
Speaker Change: Yes, and one of the reason why we don't notice that because there is not part of being reimbursed or not so it was part of the reimbursement system to have this uncontrolled party controller control, we've been no must more on it but it's not a decision and benefit businesses.
Joe Schwartz: And the next question comes from Yaron Werber with TD Co and your line is open. Yaron, your line is now open. Our next question will be from Joe Schwartz with Leering Partners. Your line is open.
Speaker Change: And the next question comes from Juran Werber with TD Cowen Your line is open.
Speaker Change: Yeah, Ryan Your line is now open.
Speaker Change: Our next question will be from Joe Schwartz with Leerink partners. Your line is open.
Joe Schwartz: Thank you, and congrats on the very strong quarter. I was wondering if you have a sense of how many of your target endos in the U.S. have adequate resources at their center in order to be able to go back and forth with payers who might not approve reimbursement of URV right away, just given there's so many patients in the U.S. and a finite number of physician offices who treat them. Could their ability to navigate this process represent a cap on your URV path revenue growth at some point? Joe is something that Jay and The commercial organization had really a lot of thoughtful thinking about it.
Joe Schwartz: Thank you and congrats on the very strong quarter I was wondering if you have a sense of how many of your target and those in the U S have adequate resources at their center in order to be able to go back and forth with Payors, who might not approve reimbursement of Europe right away.
Joe Schwartz: Just given there's so many patients in the U S and a finite number of physician offices, who treat them.
Joe Schwartz: Could.
Joe Schwartz: Their ability to navigate this process represents a cap on yogurt.
Joe Schwartz: Paas revenue growth at some point.
Joe Schwartz: Ed.
Joe Schwartz: Joe it's something that Jay and.
The commercial organization.
Joe Schwartz: Really.
Joe Schwartz: A lot of thought.
Jan Mikkelsen: How can we ensure the journey of a patient, the physician, the office that's dealing with it is really... going to be the most soft journey we ever could think about. And I think we have so much experience in this area, mainly out from our SkyTrooper, the 10,000 patients we took. A big portion of them took medical exams. And I think there was the learning we got, there was the system we're building on, and I think this is why we are potentially one of the best equipped companies really to deal with medical exception from all the elements we learned from the Skype profile.
Joe Schwartz: Thinking about it how can we ensure that journey.
Joe Schwartz: Patient the physician.
Joe Schwartz: Office, that's dealing with it is really.
Joe Schwartz: Going to be the most soft Jeremy we can think about and I think we have some loss experience and these are mainly our from our schedule for the 10000 patient we too.
Joe Schwartz: Big portion of the two medical exceptions, and I think there was really only recall towards the system be building on and I think this is why we are potentially one of the best companies really too deep medical exception from all the elements, we learn from the Sky.
Jae Woo: Jay, you can go much more inside how we are helping them. Yeah, just to add on and to reinforce what Jan said, our hub is a well-oiled machine at this point, right? Incredibly experienced. As we all know, the growth hormone deficiency space is a heavily managed space. So not only is our hub infrastructure equipped both from a volume and speed perspective, we also have a strong field reimbursement manager footprint that is constantly supporting these offices along the way as well. We feel good about our ability to resource and support our customer needs as needed. To your question directly, of course, there's going to be some offices, particularly the ones where perhaps it's lower volume, they may see fewer cases.
Joe Schwartz: You can go much more in cycle.
Joe Schwartz: Yes.
Joe Schwartz: Yes.
Joe Schwartz: Just to add on and to reinforce what you answered our hub is a well oiled machine at this point right incredibly experienced as we all know the growth hormone deficiency space is a heavily managed space. So not only is our hub infrastructure equipped both from a volume and speed perspective, we also have a strong.
Joe Schwartz: Long field reimbursement manager footprint that is constantly supporting these officers along the way as well.
Joe Schwartz: We feel good about our ability to resource.
Joe Schwartz: Support our customer needs as needed to your question directly of course, theres going to be some offices, particularly the ones, where perhaps it's lower volume. They may see fewer cases, that's not I would say unique to many other specialties that experienced the same type of office burden, but again going back to what you had were sharing.
Jae Woo: That's not, I would say, unique to many other specialties that experience the same type of office burden. But again, going back to what Jan was sharing earlier, we're well-equipped and have the capabilities to ensure that we are allowing that to not be the bottleneck as to why patients will get on therapy.
Joe Schwartz: Earlier, we're well equipped.
Joe Schwartz: And have the capabilities to ensure that we are allowing that to not be the bottleneck as to why patients will get on therapy.
Joe Schwartz: Thanks for the color.
Jan Mikkelsen: To follow up, could I just ask what you hope to see in terms of clinical benefit from the combination of Transcon C&P and HGH in the COACHE trial? Yeah, Joe, now you're asking about the future again, and it's good I'm covered by Scott's statement, even it went a little bit fast for me to understand what he said. But I actually saw an interesting result. And why it was interesting for me, because they tried to take, which it was actually something I never thought about myself, and this is potentially why I thought it was really interesting.
Speaker Change: Thanks for the color and just a follow up can I just ask what you hope to see in terms of clinical benefit from the combination of transcon CMP hgh in the coach trial.
Speaker Change: Yeah, you know you're asking about the future again and to Scott covered by <unk>.
Speaker Change: Scott statement, even if it went a little bit fast for me to understand what you said, but I actually saw an interesting resource.
Speaker Change: And why it was interesting for me.
Speaker Change: Because the trial to take.
Speaker Change: It was actually something I never thought about myself and this is potentially <unk>.
Jan Mikkelsen: We looked on acondoplasia and hypochondroplasia. Acondoplasia, as you can see, the more severe form for acondoplasia. Hypochondroplasia, a little bit more, you can say, easy form for acondoplasia. Less severe, less break, because what FTR3 superactivation is, is really putting a break on the system. So, when you took on acondoplasia, you have the break pulled down, hypochondroplasia partly down. And then we looked, what is the difference between growth hormone treatment? over years with the two different And I have to think that it was very well done, and you can basically, I can give you the numbers, so in, when you go to acondoplasia, you basically get at 5-6 cm, which are typical of what we have seen, but when you go up to hypercondoplasia, you are up on the 8 cm.
Speaker Change: Well look on a contemplation and hydro contemplation.
Speaker Change: As you can see the most severe form for that kind of pace of hydro contemplation little bit more you can say easy form for echolocation less severe lift.
Speaker Change: Because what FTR III Super activation is really putting a break on the system. So I want you to come into place. If you have the break down hydro contemplation, possibly down.
Speaker Change: And then they'll look what is the difference between growth hormone treatment.
Speaker Change: Oh, yes, with the two different.
Speaker Change: <unk> era.
Speaker Change: <unk> placed on hydro compensation and I actually think that there was a well done and you can basically I can give you the numbers so in a way.
Speaker Change: You go to a contemplation you Casey.
Speaker Change: Five six centimeters.
Speaker Change: What we are seeing but when you go up to hydrocodone.
Speaker Change: Under eight centimeters.
Jan Mikkelsen: And I had to think that it was really smart research, because in some way, what we're doing with CMP, we take an aconitoplasia patient and remove the brain. So, I don't know the results, Joe, but I thought that it gave me some strong belief that we're going to make a new standard for treatment. not only for height but also other places in the body where we really want to see a benefit in it.
Speaker Change: I like to think that there was really smart research because in summary, what we're doing with CMP be taken excellent location patient and we'll move to Craig So I don't know well results.
Speaker Change: Sure.
Speaker Change: But I saw that it gives me some strong belief that we're going to make a new standard for treatment.
Speaker Change: Only for <unk>, but also other places in the body, where we really want to see a benefit in it.
Jan Mikkelsen: Thanks for the insights.
Speaker Change: Thanks for the insights.
Paul Choi: And the next question will come from Paul Choi with Goldman Sachs. Your line is open. Hi, thanks. Good afternoon, and congratulations on the commercial progress. On your path, I wanted to ask if you have any color from the field as to what patient types it might be being utilized in. Is it almost primarily post-surgical patients, or are you seeing other causes of disease like genetic or other patients utilizing it too?
Speaker Change: And the next question will come from Paul Choi with Goldman Sachs. Your line is open.
Paul Choi: Hi, Thanks, good afternoon, and congratulations on the commercial progress.
Paul Choi: On your path I wanted to ask if you have any color from the field as to what patient types that might be utilized being utilized.
Paul Choi: Is it almost primarily post surgical patients or are you seeing other causes of diseases like genetic or other patients utilizing it to.
Jan Mikkelsen: And my second question on transcon CMP is just, you know, any updated thoughts or plans for development in the youngest patient population, those, you know, shortly after birth through, let's say, three years old, just kind of what what you're thinking is there in terms of potentially expanding into that population? Yeah, let's keep first up in the demographic on the US, because if you look on the demographic in the US is that we have about 20% coming from, we can call the genetic. immunologicals, everything else, and then about 8-0% coming from the post-surgical. And that was very much reflected in our clinical trials.
Paul Choi: And my second question on.
Paul Choi: Transcon CMP is just.
Paul Choi: Any updated thoughts or plans for development and the youngest patient population dose shortly after birth through let's say three years I'll just kind of what your thinking is there in terms of potentially expanding into that population.
Paul Choi: Yeah.
Paul Choi: Keep first up in the demographic on the U S. Because if you look at the demographic and U S is that we have about 50% coming from.
Paul Choi: <unk>.
Speaker Change: Emo Lottery code <unk>.
Speaker Change: And then about eight zero percent coming from the post surgical and <unk> must be reflected in our clinical trial. So in our clinical trials. We even have 81 patients. We actually had two evens out extremely hard to find because there's so few of them that have hydro contemplation, we actually <unk>.
Aimee Shu: So in our clinical trials, we even have 88.1 patients. We actually had two, even though extremely hard to find because there's a few of them that have hypochondroplasia. We actually managed to get them in. So from that perspective our clinical trial, this is basically reflected in the numbers.
Speaker Change: To get them in so from.
Speaker Change: From that perspective is our clinical trial basis basic reflected the numbers do not Amy if you have any comments you can convert all of the different genetic variants we have there.
Aimee Shu: I don't know, Aimee, if you have any comments. You can come with all the different genetic variants we have there. Yeah, we had GATA3 mutations. We had autoimmune polyglanular. Except for the drugs, there re really very few other changes that are going on in this and DeGeorge Syndrome. These were all seen in the trial. We know that we were seeing them in the expanded access program. I don't always get that information on the... from what we hear from our clinicians who are. with whom we speak, they're applying this. Yes. So basically, it's... A hyperparapatient is a hyperparapatient, and this is how they define it, and I don't think there is any kind of...
Speaker Change: Yes, we have <unk> mutation.
Speaker Change: I don't mean Polygram chiller.
Speaker Change: And just a quick one.
Speaker Change: And did you exit.
Speaker Change: Ill.
Speaker Change: The trial.
Thank you.
Speaker Change: Standard access program.
Speaker Change: We don't always get that information on the commercial.
Speaker Change: But what we hear from our clinicians.
Speaker Change: With whom we speak.
Speaker Change: Yes.
Speaker Change: So basically guess.
Speaker Change: Hi, Paul Perrault patient is on Hypercard patient and this is to help us define it and I don't think there is any kind of.
Jan Mikkelsen: selection from the background demographic. Because in our clinical trial, we have all the broad background demographic. And if you look on the labeling, independent of background, it's not restricted for not treating any kind of background. So we believe what we see in our. Commercial patient population really just reflect the mirror of what we see in the U.S.
Speaker Change: Selection from the background demographic because in our clinical trials, we have all the broad growth.
Speaker Change: Strong demographic and then you look on the lead independent background, it's not restricted.
Speaker Change: Any kind of background. So we believe what we see in our.
Speaker Change: Promotional patient population really just reflect the mirror, what we see in the U S.
Speaker Change: Okay.
Li Watsek: And the next question comes from Li Watsek with Cancer Fitzgerald. Your line is open. Hi, hey guys, wanted to add our congrats as well on the strong launch. Maybe just first on URB path, you know, in terms of contracting in the future.
Lee: And the next question comes from Lee <unk> with Cantor Fitzgerald. Your line is open.
Speaker Change: All right, Hey, guys wanting to add our congrats as well on a strong launch.
Maybe just first on <unk>.
Speaker Change: In terms of contracting in the future I know you are seeing the process.
Scott Smith: I know you're still in the process, but any guidance that you can provide on sort of the trend for growth to net for the rest of the year relative to Q1, and I have a follow up. Scott or Jay, who will take that? I'll just say some preliminary comments. Gross to net, you can't get out of the mandatory government rebates, so the biggest driver is likely to be mandatory government rebates that you see in the Medicare and Medicaid channel, which, you know, probably average very low, 20%. And commercial will depend on contracting, of which Jay can comment.
Speaker Change: But any guidance you can provide on sort of the trend for gross to nets for the rest of the year relative to Q1.
Speaker Change: And I have a follow up.
Speaker Change: Scott a judge who will take that.
Speaker Change: I'll just say some preliminary comments so on.
Speaker Change: Gross to net you can't get out of the mandatory government rebates. So.
Speaker Change: Biggest driver is likely to be mandatory government rebates that you see in the Medicare and Medicaid channel, which probably average very low 20%.
Speaker Change: And commercial will depend on contracting of which Jay can comment, yes, Jay Jay Yes, just as I showed before the contracting should be fairly minimal again, reflecting the clinical value proposition that we have so.
Jae Woo: Yeah, Jay, Jay. Yep. Just as I echoed before, the contracting should be fairly minimal, again, reflecting the clinical value proposition that we have.
Jae Woo: So, you know, if there's a change, it won't be materially different just relative to the other markets we've been.
Speaker Change: If theres a change it won't be materially different.
Speaker Change: Just relative to the other markets we have been.
Aimee Shu: Okay, and then just follow up on the phase two coach trial. I know you have a starting dose for Skytropha. So is there a sort of a titration scheme here that we should think about and what a top dose that you can go to? No, it's basic is that we have a starting dose for the... Transcon Rotamol in the combination with CMP. And they're also measuring IGF-1 as they do in all trials. And if there's any possibilities that the physician desire to go up in dose or down in dose, they have the opportunity to do it.
Speaker Change: Okay.
Speaker Change: And then just follow up on the phase III courage trial, I know you have a starting dose for Skype.
Speaker Change: So is there sort of a titration schemes here that we should think about and what the Topco you can go to.
Speaker Change: No. It's basically is that we have a starting dose in fall.
Speaker Change: Transcon growth hormone in the combination with CMP and Theyre also measuring IGF one aspect too in all trial.
Speaker Change: Yes.
Speaker Change: If there is any possibility that the physician side.
Speaker Change: I have to go up in dose or down the dose.
Aimee Shu: But Aimee, you know the protocols better than I do. And may I hear from Lee, which condition was she asking? As a combination. Yes, so there will be a starting dose that is informed both based on the phase two trial that we're currently doing in the combination trial and also from the many, the conditions we're studying. where there's short stature but a sufficient pro-hormone access, right? So this will give us, based on what we're seeing so far, there can be a very good and safe universal starting point. Thank you.
The opportunity to do it but the protocol based on that either.
Speaker Change: Ms, Katherine Lee, which condition with CF as a combination company.
Speaker Change: Yes, so there will be a starting dose.
Speaker Change: At this time.
Speaker Change: Based on the Phase II trial that we're currently doing in the combination trial and also from the many.
Speaker Change: The study, where they're short stature, but are sufficient.
Speaker Change: Good luck.
Speaker Change: Based on what we're seeing so far there can be a very good safety universal stacking up typically.
Speaker Change: Great. Thank you.
Eliana Merle: And our next question will come from Eliana Merle with UBS. Your line is open. Hey guys, thanks for taking the question. I'm curious, just the feedback from the early patient starts, how the titration process has gone on YorbaPath, just logistically, you know, kind of any color anecdotes on how that's been going for physicians and patients, and any color so far on what you're seeing from the refill rate. I know it's early on, but curious any trends that you're seeing there. Thank you.
Speaker Change: And our next question will come from Eliana Merle with UBS. Your line is open.
Speaker Change: Hey, guys. Thanks for taking the question I'm curious if the feedback from the early patient starts how the titration process has gone on your behalf just logistically kind of any color or anecdotes on how that's been going for physicians and patients.
Speaker Change: Any color, so far and what youre seeing from the.
Speaker Change: Refill rate.
Speaker Change: I know, it's early but curious any trends that you're seeing.
Speaker Change: Yes.
Speaker Change: Okay.
Jan Mikkelsen: What I'm typically looking on. is number. And one of the numbers I look a lot is two things, adherence second number I'm looking on. is how many patients are dropping. I think it's two good numbers to look if you have a successful, meaningful treatment of a patient with really addressing a major unmet medical need and really do that. And when I look at the adherence, it's exactly as high as we saw it in the clinical trials, which was really unique. And drop out, we have given you the German numbers under 1%, and we see the same thing everywhere.
Speaker Change: But typically looking on.
Speaker Change: This numbers.
Speaker Change: And one of the numbers look a lot is two things adherence.
Speaker Change: Segment number looking on.
Speaker Change: How many patients are dropping off.
Speaker Change: I think it's too good numbers to look if you have a successful meaningful treatment of the patient with really addressing a major unmet medical need and really do that and we're not looking to adherence.
Speaker Change: Secondly, as high as we saw it in the clinical trials, which was really unique and drop out we have given you the German numbers under 1%.
Jan Mikkelsen: If you start on your pants, you stay on yours.
Speaker Change: We see the same thing everywhere.
Speaker Change: You start on your past you stay on your guidance.
Jan Mikkelsen: And that is a chronic treatment, rest of your life.
Speaker Change: And that is a chronic treatment rest of your life.
Kelly Shi: The next question comes from Kelly Shi with Jeffries. Your line is open. Hi, good afternoon. This is Jose for Kelly. Congrats on the strong quarter. And thanks for taking our question. I have a question in terms of payer dynamics. What are the major reimbursement pushbacks? And based on these dynamics, what percent do you estimate you can capture in the mild, moderate, and severe segments? And also, on the clinical value proposition of the orbit path, would you consider running a clinical utility trial to facilitate uptake in milder patients? Thank you.
Speaker Change: Thank you.
Speaker Change: Your next question comes from Kelly <unk> with Jefferies. Your line is open.
Kelly: Hi, good afternoon, Thanks, Jose for Kylie Congrats on the strong quarter and thanks for taking our question couple of question in terms of payer dynamics, what are the major reimbursement push backs and based on these dynamics what percent do you estimate you can capture into mild moderate and severe segments.
Kelly: And also on the clinical value proposition will be orderly path would you consider running a clinical utility trials.
Kelly: <unk> uptake in milder patients. Thank you.
Jan Mikkelsen: related to your first question. The part we discussed before. Being uncontrolled, party controlled, or controlled is not part of the reimbursement. So you cannot, we cannot really see that. We don't know exactly where they're coming from a different group. We believe many of them, the majority is coming from the uncontrolled because they see the physician much more often. So that is not any part of the reimbursement discussion.
Kelly: And related to your first question.
Kelly: At the.
Kelly: The part we discussed before.
Kelly: Being uncontrolled party control or control is not part of the reimbursement system.
Kelly: You cannot we cannot really see that we don't know exactly where theyre coming from a different group. We believe many of them remember, Georgia is coming for the uncontrolled because they see their physician.
Speaker Change: More often so that is not and it's part of the reimbursement discussion. The second question I need to understand little bit more about what is exact it was you wanted us to address.
Jan Mikkelsen: The second question I need to understand a little bit more about what is exactly what you wanted us to address. On the valid preposition of the orbit path, the potential for preventing renal damage, would you consider running a clinical utility trial to perhaps facilitate uptake in milder patients who are controlled on SLC? I don't think really this is the key element for going on the treatment. I actually think the key element to go on the treatment is the benefit you get as a person. You're getting normal again. The long-term risk is basically what we call a health economic discussion when we talk about the benefit of the treatment for the society.
Speaker Change: On the value proposition of the already passed.
Speaker Change: The potential for preventing renal damage.
Speaker Change: Would you consider running a clinical utility trial to perhaps facilitate uptake and mindful milder patients who are control on.
Speaker Change: Associates.
Speaker Change: Sandra Gary I don't think we really this is the key element for going on the treatment I have to say.
Speaker Change: The key element to go into the treatment is the benefit we get as a person you're getting normal again, the long term risk its basic what we call our health economic discussion when we talk about the benefit of the treatment for the society and we are evaluating exactly how we can do that in the first part.
Jan Mikkelsen: And we are evaluating exactly how we can do that in the best possible manner to really show the financial benefit it is really to be on a Europat treatment, not only for the patient, not only for the physician, but basically for the entire society.
Speaker Change: The board manner to really show the financial benefit is really to be on a Europe had treatment not only for the patient not only focuses physician, but basic for the entire society.
Aimee Shu: All right, thank you. Some literature data already out there that we may be able to leverage, showing that some of the conventional therapy itself is toxic to the kidney, right, and that when that is able to be lowered in whatever way, right, that the kidney function gets better. So there may not, you know, we'll see what the need is for demonstrating this yet again. Very helpful. Thank you so much.
Speaker Change: Alright, thank you.
Speaker Change: Literature data already out there that we may be able to leverage.
Speaker Change: Showing that some of the conventional therapy itself is toxic to the kidney and that when that is April could be lowered and whatever way right.
Speaker Change: That the kidney function better sell.
Speaker Change: We'll see what the EBIT for demonstrating yet again.
Speaker Change: Very helpful. Thank you so much.
Luca Isai: And the next question comes from Luca Isai with RBC. Your line is open. Well, great, thanks so much for taking my question and congrats on the launch here. Maybe a quick one on competition, BridgeBio presented their data for their molecule early this week, and I believe MBX will do the same next quarter. So wondering what's the latest thinking on both molecules and how competitive you think they can be versus YorubiPath? And then maybe Scott, super quickly, how should we think about the SG&A for the remainder of the year given the meaningful jump this quarter versus last quarter?
Speaker Change: And the next question comes from Luca <unk> with RBC. Your line is open.
Luca: Great. Thanks, so much for taking my question and congrats on the launch here, maybe a quick one on competition a bridge buyer presented their data for your molecule early this week and I believe Mdx will do the same next quarter. So wondering what's the latest thinking on both molecules and how competitive you think that can be versus <unk>.
Speaker Change: And then maybe Scott Super quickly how should we think about the SG&A for the remainder of the year given the meaningful jump this quarter versus last quarter any thoughts there much appreciate it. Thanks so much.
Jan Mikkelsen: Any thoughts there? Much appreciated. Thanks so much.
Jan Mikkelsen: Yeah, you mentioned two compounds, one is the calcium lutein. which are being positioned into a phase three trial of 88.1. Currently ADS-1 is being treated with uropaths and they're coming on treatment today. It has been hard for us to find an ADS-1 patient. And Cher, you went into the claim database and how many? Yeah, yeah. I'm a big ADS one. Yeah, there were something like 350 patients who'd had a claim associated with ADH1 in the past, like four or five years, and even fewer than that within the last year. So it was a very tiny number.
Speaker Change: Yes, you mentioned two compound.
Speaker Change: One is the cultural boutique.
Speaker Change: Being positioned into a phase III trial of <unk> one.
Speaker Change: Currently.
Speaker Change: <unk> is being treated with your pants and theyre coming on treatment today.
Speaker Change: <unk> has been hard for us to find <unk> patients and to share you went into the claims database and how many.
Speaker Change: Patient yeah, yet Brian.
Speaker Change: One.
Speaker Change: Yes, there were something like 350 patients who'd had a claim associated with ADH won in the past like four or five years and even fewer than that within the last year. So it was a very tiny number. This was from a large national claims database with hundreds of millions of lives.
Sherry Glass: This was from a large national claims database with hundreds of millions of lives. So, it's not going to change anything for us, because we are addressing the position of catalytic into the era of, for example, what we call chronic hyperparapatient. That is not a yes-one. You know, I really love science. I don't understand the science there. You're trying to position into a place of a patient that does not have inductious PTH. How can you increase the level of industrious PDAs when they're not produced? They're already producing on max. And there is actually a very nice poster that's basically showing that.
Speaker Change: So it's not going to change anything for us because we'll be addressing.
Speaker Change: The position of <unk>.
Into the era of <unk> samples.
Speaker Change: Often called chronic hyperparasite patient that is not one.
Speaker Change: I'd really love Science.
Speaker Change: I don't understand besides there, we're trying to position into a place.
Speaker Change: And patients that not have DOCSIS.
Speaker Change: <unk>.
Speaker Change: How can you increase the level of indexes ppas, when we're not producing it.
Speaker Change: <unk> already producing unmatched spend days Axel.
Speaker Change: They're nice post of the basic showing that they're taking.
Jan Mikkelsen: They're taking calcium luteic into four patients for three days, or six, or something like that. And it's really showing that you cannot increase the secretion of PTAs. So it's not something I don't understand from a scientific perspective, rational, but all the data I've seen is also indicating exactly the same thing. Scott was saying the old fight. Okay, that is one thing. The MBX, this is the once weekly one. And, you know, We have a possibility to develop once weekly product for a long time. And now we're talking of the context of once weekly first. Uh, we...
Speaker Change: So when you take into full patient for three days or six or something like that and it's clearly showing that you cannot increase the secretion of <unk>. So it's not something I don't understand from a scientific perspective rational, but all the data I have seen is also indicating exactly the same thing.
Speaker Change: Okay.
Speaker Change: Scott was saying your $5 okay.
Speaker Change: Okay.
Speaker Change: That is one thing the index. This is a once weekly <unk>, one and <unk>.
Speaker Change: We have the.
Speaker Change: Possibility to develop once weekly product for long time, and our token of the context of once weekly <unk>.
Speaker Change: We.
Jan Mikkelsen: Some ways stalled a little bit because we couldn't see the unmet medical need and also because of the desire on different way of living with a hyperparapatient where you sometimes need more or less. And it can be done on exercise, seasonal activities, other things that change in your life. And out from that, we looked on patients, how stable are them, how often do they titrate up and down? And we see only a small part of the patient being stable. So if we wanted to develop an once-weekly product, we would develop it as a baseline, like basal insulin.
Speaker Change: Some ways stalled a little bit because we couldnt see the unmet medical need and also because of this tire on different way of living with and hydropower patients, where you sometimes need more or less and it can be done on exercise season.
Speaker Change: Activity other things that changed in your life and often that we looked on patient how stable all of them are often to the attach rate up and down and we see only a small part of the patient be stable. So if we wanted to develop and once weekly product we develop.
Speaker Change: The baseline like basal insulin and the distillate will have both daily product to really be sure that can adjust them up and down.
Jan Mikkelsen: And then we still will have the daily product to really be sure they can adjust them up and down. The technology platform they are utilizing in the – I always get it wrong – MBX or BMX. I cannot remember what is that bike. But I always get it wrong. But the element of that is basic technology which are basic as an active entity. It's an isolated PTH that stay 99.9% associated to basic, the element of albumin. I do not know how that ever can activate the phosphate receptor, how that can get into the brain and really restore normal endogenous PTH level in the normal distribution you have out through the body.
Speaker Change: The technology platform.
Speaker Change: Utilizing in the.
Speaker Change: Our risk weighted wrong MPLX of beer mix I cannot remember what is the pike.
Speaker Change: But obviously wrong, but the element of that is basic technology.
Speaker Change: Our basic asset entity is in.
Speaker Change: Isolated PK that state 99, 9% extra shaded two basic element of albumin.
Speaker Change: I do not know how it works and activate with phosphate receptor Javier can come into the Fray and really restore normal industrious PTH levels in the normal distributions you half hour through the body.
Jan Mikkelsen: I'm also lost in the science.
Speaker Change: Im also launched into the science there.
David Lebowitz: And our next question comes from David Lebowitz with Citi. Your line is open. Thank you very much for taking my question. First of all, if you look at the NAPARA patients that were left from mixed withdrawal, how long do you think it takes until that whole population gets worked through? And then further looking back at NAPARA and looking what you've seen during your first quarter of How would you, aside from the fact that the data is just superior, how would you characterize the difference in what patients are showing interest in this therapy? First of all, you cannot compare the clinical benefits.
Speaker Change: Okay.
Speaker Change: And our next question comes from David Lebowitz with Citi. Your line is open.
David Lebowitz: Thank you very much for taking my question.
Speaker Change: First of all if you.
Speaker Change: You look at the debt the net para patients that were left from.
Speaker Change: Mixed withdrawal.
Speaker Change: How long do you think it takes until that whole population gets worked through and then further looking back at net.
Speaker Change: What you've seen during your first quarter launch.
Speaker Change: Our U S.
Speaker Change: Aside from the fact that the data is just superior how would you characterize the difference in what patients are showing interest in.
Speaker Change: In mist therapy.
Speaker Change: First of all you cannot compare the clinical benefit.
Jan Mikkelsen: the team, La Paz, and Europe. Nepal had a labeling as an adjunct therapy, take a little bit of your daily calcium supplement away, take a little bit of your active vitamin away, and then you take Nepal. You have no positive impact on kidney function, you have no positive quantitative manner on quality of life. I see this as two different products and we can never compare these two together. First question related to when the NAPARA patient will be switched. We addressed it a little bit in the beginning of here, the discussion here, where we know that there was a letter from Takeda indicating that they are getting their last shipment now.
Speaker Change: Revpar in Europe.
Speaker Change: The patent Nathan.
Speaker Change: <unk> therapy takes a little bit of your daily catch some supplemental way takes a little bit of Youre actually right away and then you take net par.
Speaker Change: You have no positive impact.
Speaker Change: Kidney function, you'll have no positive quantitative manner on quality of life.
Speaker Change: I see this as two different products and we can do a compare these two together.
Speaker Change: First question related to rent the net PAH patients will be switched we addressed it a little bit in the beginning of year that discussion here.
Speaker Change: We know that there was a letter from Takeda, indicating that theyre getting their lost shipment now so.
Jan Mikkelsen: So, and I think, as to our knowledge, the shipment is three months, so we expect that the last series of NAPARA patients will come over in either end of Q2 or Q3. That is our expectations.
Speaker Change: As to our notice was shifting its a three months. So we expect that the last series of manpower patient will be come in.
Speaker Change: In either end of Q2, our Q3 titles to our expectations.
Leland Gershell: And our next question comes from Leland Gershell with Oppenheimer. Your line is open. Thanks. Great to see the strong execution on USU RiverPath. A couple of questions from us.
Speaker Change: Okay.
Speaker Change: And our next question comes from Leland <unk> with Oppenheimer. Your line is open.
Speaker Change: Thanks, Great to see the strong execution on a path a couple of questions from us.
Jan Mikkelsen: Just apologies if this has been asked before, but with respect to potential benefits on renal, I know you had said that you don't see that as a key driver for your RiverPath uptake, but nonetheless, are you able to comment in the early days of the US launch? Are you seeing a more difficult or easier time to gain reimbursement or access to the drug for those patients who may have less or more renal impairment along with, you know, whatever needs they have in terms of conventional therapy?
Speaker Change: I apologize if this has been asked before but with respect to potential benefits on renal and now.
Speaker Change: <unk>.
John: John You had said that you don't see that as a key.
John: Drive refrigerated uptake, but nonetheless are you able to comment in the early days of the U S launch are you seeing.
John: A more difficult or easier time to gain reimbursement for access to the drug for those patients who may have less or more.
John: Anil impairment along with whatever needs they have in terms of.
Jan Mikkelsen: And then a second question. Thank you. It's not a part of the reimbursement process. We don't see that it's the element that decides if it's reimbursement or not reimbursement. So it's like the same thing as we discussed on control, party control. Control is not part of the reimbursement process either. It's basic. When you look on the labeling, most plans have adapted the way that we basically have in our labeling, and it's not defined any way like that.
John: Conventional therapy and then a second question. Thank you.
John: It's not a part of the reimbursement process. We don't see that is the element that decide of risk reimbursement not reimbursement. So it's like the same thing as we discussed on control Party control control is not part of the reimbursement process either its basic when you look on the labeling.
John: My most claims have adapted.
John: The way that we basically have in our leasing and is not defined anyway of our debt.
Aimee Shu: First, one correction. Aimee, we didn't study what we call severe renal impairment patients, and that is not part of our labeling, and I think it's stage four. Yeah, far lower. Yeah, which are a very, very low number, which are stage four, where we never started the talk. Got it. Okay, that's helpful.
John: Is that fair.
John: One correction Amy we didn't start it in what we call severe renal impairment patients and that is not part of our leasing and are seeing as states for.
Amy: Yes, our lowest peanuts.
Amy: Bev low number with our states for where we never started bedrock.
Jan Mikkelsen: And then just looking forward to the coach data. Are you, are you, is there sort of a bar that you have in mind for linear growth? Or is this something that you can see being driven forward, principally on secondary benefits, say body composition, or other? How are you thinking about kind of the mix of efficacy with the combination? When we think about air conditioning, the key element for us is to address complexity. But we cannot avoid also to address the. So when we measure linear growth, which got established from another company that is established as the primary endpoint, I will personally have selected another endpoint if I could ever select that.
Amy: Got it Okay. That's helpful. And then just looking forward to the coach data.
Speaker Change: Are you is there sort of a bar that you have in mind for.
Speaker Change: Linear growth or is this something that you can see being driven forward principally on.
Speaker Change: Gary benefits say body composition.
Speaker Change: Or are there how are you thinking about kind of the mix of efficacy combination. Thank you.
Speaker Change: When we think about excellent location.
Speaker Change: Key element for us to reach the first complications, but we cannot avoid also to address.
Speaker Change: So when we measure dinner Coke Wescott established from another company that is the status.
Speaker Change: The primary endpoint I will personally have selected another endpoint, if I could select debt but.
Jan Mikkelsen: But we will, because we are forced to do it, because it's the established clinical endpoint, is that we will look on linear growth, and it will be part of the data we basically will report when we come up with the analyzed height velocity, height SDS, and other things for that. And as I said before, I have great expectations. I have a strong belief that we can reset the bar for what you see in air contemplation treatment, and it's not only related to linear growth, but also the associated complications.
Speaker Change: Because we are forced to do it because.
Speaker Change: <unk> clinical endpoint is that we will look on DDA growth and it would be part of the HIV basic will report when we come up with the annualized height velocity height.
Speaker Change: There is other things for that and as I said before.
Speaker Change: Great expectation I'll have a strong belief that we can reset the bar for what youll see in a contemplation treatment and it is not only related to linear growth, but also the SSAT complications.
Operator: All right, thanks very much.
Speaker Change: Alright, thanks very much.
Operator: This is all the time that we do have for questions. This concludes today's conference call, and thank you for participating. And you may now disconnect. Thank you.
Speaker Change: This is all the time that we do have for questions.
Speaker Change: This concludes today's conference call and thank you for participating and you may now disconnect.
Speaker Change: Dave.
Speaker Change: Okay.
Speaker Change: [music].
Operator: Thank you for watching!
Speaker Change: Okay.
Speaker Change: Yes.
[music].