Ascendis Pharma A/S (ASND) 2025 Q1 法說會逐字稿

完整原文

使用警語:中文譯文來源為 Google 翻譯,僅供參考,實際內容請以英文原文為主

  • Operator

    Operator

  • Ladies and gentlemen, thank you for standing by and welcome to Ascendis Pharma first-quarter 2025 earnings conference call. (Operator Instructions) Please be advised that today's conference is being recorded.

    女士們,先生們,感謝你們的支持,歡迎參加 Ascendis Pharma 2025 年第一季財報電話會議。(操作員指示)請注意,今天的會議正在錄音。

  • I would like now to turn the conference over to Scott Smith, Vice President and Chief Financial Officer. Please go ahead, sir.

    現在我想將會議交給副總裁兼財務長 Scott Smith。先生,請繼續。

  • Scott Smith - Chief Financial Officer, Executive Vice President, Member of the Executive Board

    Scott Smith - Chief Financial Officer, Executive Vice President, Member of the Executive Board

  • 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 Ascendis Pharma. Joining me on the call today are Jan Mikkelsen, President and Chief Executive Officer; Sherrie Glass, Chief Business Officer; Jay Wu, Executive Vice President and President Ascendis US; and Aimee Shu, Chief Medical Officer.

    非常感謝接線員,也感謝大家參加我們的 2025 年第一季財務業績電話會議。我是 Ascendis Pharma 的財務長 Scott Smith。今天與我一起參加電話會議的還有總裁兼執行長 Jan Mikkelsen; Sherrie Glass,首席商務長; Jay Wu,Ascendis 美國執行副總裁兼總裁;以及首席醫療官 Aimee Shu。

  • 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 SKYTROFA and YORVIPATH for the US, European, and other markets.

    在我們開始之前,我想提醒您,本次電話會議將包含前瞻性陳述,這些陳述旨在受到《私人證券訴訟改革法案》提供的安全港保護。此類聲明的範例可能包括但不限於有關我們針對美國、歐洲和其他市場對 SKYTROFA 和 YORVIPATH 進行商業化和持續開發的聲明。

  • 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.

    除了某些財務預期外,我們的候選產品以及我們對其持續進展和潛在商業化的預期。我們的策略計劃、合作夥伴關係和投資、我們關於臨床管線的目標(包括臨床結果和試驗的時間)、我們正在進行和計劃中的監管備案、我們對監管決策的時間和結果的期望、預期的市場發展以及我們在內分泌罕見疾病以外的治療領域的市場機會的探索。

  • 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 (technical difficulty) law.

    這些聲明是基於我們今天所掌握的資訊。實際結果可能有所不同,可能與我們的前瞻性陳述有重大差異,您不應過度依賴這些陳述。我們不承擔隨著情況變化而更新這些聲明的義務,除非(技術困難)法律。

  • For additional information concerning the facts (technical difficulty) actual results to different material, please see our forward-looking statement section in today's press release and the Risk Factor section of our most recent annual report on Form 20-F filed with the SEC on February 12, 2025.

    有關不同材料的事實(技術難度)實際結果的更多信息,請參閱今天新聞稿中的前瞻性聲明部分以及我們於 2025 年 2 月 12 日向美國證券交易委員會提交的最新 20-F 表年度報告中的風險因素部分。

  • TransCon COACH hormone or TransCon hGH, is approved in the US by the FDA, and in the EU has received MAA authorization from the European Commission for the treatment of pediatric growth hormone deficiency. TransCon PTH is approved in the US 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.

    TransCon COACH激素或TransCon hGH,在美國已獲得FDA批准,在歐盟已獲得歐盟委員會的MAA授權,用於治療兒童生長激素缺乏症。TransCon PTH 在美國獲得 FDA 批准用於治療成人甲狀旁腺功能減退症,歐盟委員會和英國藥品和保健產品管理局已授權 TransCon PTH 作為治療成人慢性甲狀旁腺功能減退症的替代療法。

  • Otherwise, please note that our product candidates are investigational and not approved for commercial use. As an 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. On the call today, we'll discuss our first quarter of 2025 financial results, and we'll provide further business updates. Following some prepared remarks, we'll then open up the call for questions.

    否則,請注意,我們的候選產品尚處於研究階段,尚未獲準用於商業用途。作為研究產品,候選產品的安全性和有效性尚未經過任何監管機構的審查或批准。本次電話會議中有關我們候選產品的任何聲明均不應被視為促銷。在今天的電話會議上,我們將討論 2025 年第一季的財務業績,並提供進一步的業務更新。在發表一些準備好的發言之後,我們將開始提問。

  • With that, let me turn it over to Jan.

    現在,讓我把時間交給 Jan。

  • Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

    Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

  • Thanks, Scott. Good afternoon, everyone.

    謝謝,斯科特。大家下午好。

  • In the first quarter of 2025, Acendis continue the strong start of our global YORVIPATH launch. As well as key development and regulatory progress, supporting our long-term growth strategy to be a leading biopharma company. The strong US launch of our YORVIPATH position 2025 to be an inflection point for Ascendis with a growing revenue base and a clear path to become cash flow positive.

    2025 年第一季度,Acendis 將繼續在全球推出 YORVIPATH,取得強勁開局。以及關鍵開發和監管進展,支持我們成為領先生物製藥公司的長期成長策略。我們在美國強勢推出的 YORVIPATH 定位 2025 將成為 Ascendis 的一個轉折點,其收入基礎不斷增長,並有明確的途徑實現現金流為正。

  • As of March 31, YORVIPATH was prescribed in the US by more than 1,000 unique prescribers for more than 1,750 patients. This represents our first full quarter for the US launch. YORVIPATH, the first and only FDA approved treatment for hypoparathyroism in adults, is addressing the underlying cause of the disease. By providing active PGA within the physiological range for 24 hours per day.

    截至 3 月 31 日,美國已有超過 1,000 名獨立處方人員為超過 1,750 名患者開立了 YORVIPATH 處方。這是我們在美國推出產品的第一個完整季度。YORVIPATH 是第一個也是唯一一個獲得 FDA 批准的治療成人副甲狀腺功能低下的藥物,正在解決疾病的根本原因。透過每天 24 小時提供生理範圍內的活性 PGA。

  • SKYTROFA, or lonapegsomatropin, Is firmly established as a high-value bank and the preferred treatment for patients, physicians, and caregivers. SKYTROFA is well positioned as daily treatment continues to exit the US market, and the SKYTROFA label has the potential to expand beyond its single indication. SKYTROFA is a key pillar in our strategy to become a global leader in the treatment of growth disorder.

    SKYTROFA,即 lonapegsomatropin,已被牢固確立為高價值庫,是患者、醫生和護理人員的首選治療方法。隨著日常治療藥物不斷退出美國市場,SKYTROFA 佔據了有利地位,並且 SKYTROFA 標籤有可能擴展到其單一適應症之外。SKYTROFA 是我們成為生長障礙治療領域全球領導者策略的重要支柱。

  • TransCon CNP, the first long-acting therapy in development for the treatment of achondroplasia, is set to become the second pillar in our growth-disorder strategy. We believe that TransCon CNP has treatment benefits in addition to linear growth that addresses multiple aspects of the condition that are fundamentally important to patients. 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 trial show that TransCon CNP has the potential to transform the lives of people without contemplation.

    TransCon CNP 是首個正在研發的用於治療軟骨發育不全的長效療法,它將成為我們生長障礙策略的第二大支柱。我們相信,TransCon CNP 除了線性生長之外還具有治療優勢,可以解決對患者至關重要的多個病情方面的問題。我們於 3 月向 FDA 提交了 NDA,並預計今年第三季向 EMEA 提交 MAA。三項隨機、雙盲安慰劑對照臨床試驗的數據表明,TransCon CNP 有可能改變人們的生活。

  • In my remarks, I will discuss each of this product in detail and comment on other recent development within our business.

    在我的發言中,我將詳細討論每種產品,並評論我們業務中的其他最新發展。

  • Beginning with YORVIPATH. First quarter of total global YORVIPATH revenue growth to EUR45 million compared to EUR40 million in the fourth quarter of last year. Following commercial availability in the US in December of last year, we are seeing strong US demand, reflecting both the deep unmet medical need in the market as well as the large patient population. As of March 31, more than 1,750 patients, including the 200 patients from our ERP and clinical program, have been prescribed YORVIPATH in the US by over 1,000 unique healthcare providers.

    從 YORVIPATH 開始。YORVIPATH 第一季全球總營收成長至 4,500 萬歐元,而去年第四季為 4,000 萬歐元。自去年 12 月在美國上市以來,我們看到美國的需求強勁,這反映了市場上嚴重未滿足的醫療需求以及龐大的患者群體。截至 3 月 31 日,美國已有超過 1,750 名患者(包括來自我們的 ERP 和臨床計畫的 200 名患者)由 1,000 多家獨特的醫療保健提供者開立了 YORVIPATH 處方。

  • Enrollment of patients new to YORVIPATH continue at a similar weekly rate in April. The majority of patients who have received insurance approval for their YORVIPATH prescription received that approval in four to eight weeks and we are pleased with the approval rate we have seen.

    4 月份,YORVIPATH 新患者的入組率繼續保持相似的每週水準。大多數獲得 YORVIPATH 處方保險批准的患者在四到八週內收到了批准,我們對所看到的批准率感到滿意。

  • We are beginning to see favorable [their] plans put into place and continue to see approvals across both commercial and government plans. The strong launch performance of YORVIPATH in the US support our view of its excellent product profile and the major medical need in the market, and we expect YORVIPATH to contribute significantly to our revenue in 2025.

    我們開始看到有利的計劃付諸實施,並繼續看到商業和政府計劃獲得批准。YORVIPATH 在美國強勁的上市表現支持了我們對其優秀產品形象和市場主要醫療需求的看法,我們預計 YORVIPATH 將在 2025 年為我們的收入做出重大貢獻。

  • Outside of the US, we see steady YORVIPATH revenue growth in both the Europe, direct, and international markets. And we expect additional acceleration of the revenue growth when YORVIPATH reimbursement becomes available in additional Europe-direct countries in the second half of the year.

    在美國以外,我們看到 YORVIPATH 在歐洲、直接和國際市場的營收都在穩定成長。我們預計,當 YORVIPATH 報銷於今年下半年在更多歐洲直銷國家推出時,收入成長將進一步加速。

  • The continued rapid uptake, together with high rates of patient adherence, give us confidence that YORVIPATH is well positioned to uniquely address the onward medical needs 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. We estimate there are over 400,000 patients globally. And around 70,000 to 90,000 patients in the US alone living with chronic hypoparathyroidism. Our claims analysis demonstrates that 10,000 to 15,000 of these US patients are uncontrolled and 30,000 to 35,000 are partly controlled.

    持續的快速成長以及患者的高依從性使我們相信,YORVIPATH 能夠以獨特的方式滿足該患者群體的後續醫療需求,並且我們會定期審查患者的意見和數據,以評估是否還有其他方法可以進一步改善治療方案。我們估計全球有超過 40 萬名患者。光是在美國就有大約 70,000 至 90,000 名患者患有慢性副甲狀腺功能減退症。我們的索賠分析表明,這些美國患者中有 10,000 至 15,000 名患者未得到控制,30,000 至 35,000 名患者得到部分控制。

  • Based on the latest clinical practice guidelines, nearly all these patients are candidates for treatment with YORVIPATH. Our strong global launch gives us high conviction that we can continue to build and lead this market, and YORVIPATH can be a doable, multi-billion Euro stock-device product with a patent lifespan extending into the 2040s.

    根據最新的臨床實踐指南,幾乎所有這些患者都適合接受 YORVIPATH 治療。我們強勁的全球發布使我們堅信我們可以繼續建立和引領這個市場,而 YORVIPATH 可以成為一種可行的、價值數十億歐元的庫存設備產品,其專利壽命可以延長到 2040 年代。

  • Turning to SKYTROFA. Q1 revenues for SKYTROFA were EUR51 million with continued patient growth and global expansion offset by the typical first-quarter revenue dynamic in the US. We have around 7% market share of the total growth market in the US and around 43% of the total US long-acting [growth to] based on third-party prescription data.

    轉向 SKYTROFA。SKYTROFA 第一季的營收為 5,100 萬歐元,病患的持續成長和全球擴張被美國第一季的典型營收動態所抵消。根據第三方處方數據,我們在美國整個成長市場中佔有約 7% 的份額,在美國長效藥物總[成長]市場中佔有約 43% 的份額。

  • The pediatric growth deficient indication represents about half of the total growth on the market. With our premium pricing, SKYTROFA's leading position in pediatric growth hormone deficiency. We believe we are well positioned to expand the opportunity for SKYTROFA in multiple ways. A key near-term milestone is our first potential label expansion in the established growth hormone indications from our supplement PLA for the potential US approval in adult growth hormone deficiency. Where we have a PDUFA go date of July 27, 2025.

    兒童生長缺陷適應症約佔市場總成長的一半。憑藉我們的優質定價,SKYTROFA 在兒童生長激素缺乏症治療領域佔據領先地位。我們相信,我們有能力以多種方式擴大 SKYTROFA 的機會。近期的關鍵里程碑是,我們首次在補充劑 PLA 的既定生長激素適應症中實現潛在標籤擴展,以獲得美國對成人生長激素缺乏症的批准。我們的 PDUFA 生效日期為 2025 年 7 月 27 日。

  • We're also on track to start a basket trial for SKYTROFA in a range of indications, including idiopathic short statue, shock deficiencies, Turner syndrome, and SDA. We are planning to discuss this trial with the FAA in a Phase 2 meeting this quarter. Importantly, we are also investigating TransCon COACH hormone outside the established growth hormone indication, such as in a potential combination therapy with TransCon CNP for treatment of achondroplasia and other growth disorder, which I will address in a moment.

    我們也計劃開始對 SKYTROFA 進行一系列適應症的籃子試驗,包括特發性矮小症、休克缺陷症、特納氏症和 SDA。我們計劃在本季的第二階段會議上與美國聯邦航空局討論這項試驗。重要的是,我們也正在研究已確定的生長激素適應症以外的 TransCon COACH 激素,例如與 TransCon CNP 進行聯合治療,用於治療軟骨發育不全和其他生長障礙,我稍後會討論這個問題。

  • Moving to TransCon CNP. TransCon CNP is the third key product in our endocrinology rare-disease-product portfolio. The genetic variant that caused achondroplasia changes the way receptor work in multiple tissues throughout the body. Not just in the growth plate and in bones, resulting in a wide range of serious medical complications in childhood and lasting throughout adulthood.

    移至 TransCon CNP。TransCon CNP 是我們內分泌罕見疾病產品組合中的第三個關鍵產品。導致軟骨發育不全的基因變異會改變全身多個組織中受體的工作方式。這不僅影響生長板和骨骼,還會導致兒童時期出現一系列嚴重的醫療併發症,並持續整個成年期。

  • Because TransCon CNP provides sustained, self-holding levels of CNP throughout the body, it has been demonstrated and unique product profile, giving it the potential to bring growth benefit and important new benefit beyond linear growth, as well as reduce risk of hypertension and injection site reaction. In our period approach, TransCon CNP demonstrated significant improvement in the primary input of linear growth compared to placebo, as well as significant improvement in other clinical endpoints meaningful to the achondroplasia community, including leg bone, muscle functionality, body proportionality, and health-related quality of life.

    由於 TransCon CNP 可在全身範圍內提供持續、自我維持的 CNP 水平,它已被證明具有獨特的產品特性,使其有可能帶來生長益處和超越線性生長的重要新益處,並降低高血壓和注射部位反應的風險。在我們的週期方法中,與安慰劑相比,TransCon CNP 在線性生長的主要輸入方面表現出顯著改善,並且在對軟骨發育不全社區有意義的其他臨床終點方面也表現出顯著改善,包括腿骨、肌肉功能、身體比例和與健康相關的生活品質。

  • Leg bone is a common complication in achondroplasia that can result in chronic pain and impaired physical function, driving many to undergo complex painful corrective surgeries. I have been in meetings with patient organizations in Europe and the US who have confirmed the importance of addressing the complication of achondroplasia beyond linear growth.

    腿骨是軟骨發育不全的常見併發症,可導致慢性疼痛和身體功能受損,迫使許多人接受複雜而痛苦的矯正手術。我曾與歐洲和美國的患者組織會面,他們確認了解決軟骨發育不全併發症的重要性,而不僅僅是線性生長。

  • Just as important to the achondroplasia community, TransCon CNP has shown a safety and probability profile compared to placebo with low frequency of injecting side reaction, all which were mild and no evidence of symptomatic hypertension. After positive interaction with the FDA relating to the content of our NDA submission, we are pleased to have submitted TransCon CNP for the review in March.

    對於軟骨發育不全症患者來說,同樣重要的是,與安慰劑相比,TransCon CNP 顯示出了安全性和機率特徵,注射副作用發生率較低,且副作用均較輕微,沒有症狀性高血壓的證據。在與 FDA 就我們的 NDA 提交內容進行積極互動後,我們很高興在 3 月提交了 TransCon CNP 以供審查。

  • In the EU, we plan to submit an MAA during the third quarter of this year. Additionally, during the fourth quarter of 2025, we plan to submit an R&D or similar to investigate TransCon CNP or in combination with TransCon COACH for the treatment of hypochondroplasia.

    在歐盟,我們計劃在今年第三季提交 MAA。此外,在 2025 年第四季度,我們計劃提交一項研發或類似項目,以研究 TransCon CNP 或與 TransCon COACH 聯合用於治療軟骨發育不全。

  • Shifting to TransCon CNP and TransCon COACH hormone combination therapy, we are committed to continue to drive even better outcomes for people living with achondroplasia. This is why we are conducting the COACH trial. Being the first Phase 2 study combining CNP and COACH hormone in achondroplasia, each of which stimulates different signaling pathways in the growth plates and other tissue in the body.

    轉向 TransCon CNP 和 TransCon COACH 荷爾蒙聯合療法,我們致力於持續為軟骨發育不全患者帶來更好的治療效果。這就是我們進行 COACH 試驗的原因。這是第一個將 CNP 和 COACH 激素結合起來治療軟骨發育不全的 2 期研究,它們分別刺激生長板和身體其他組織中的不同訊號路徑。

  • We look forward to sharing top line week-26 results from the COACH trial data this quarter and see great potential to further raise the bar for clinical outcomes with TransCon CNP as the potential future backbone therapy, we believe we can achieve even greater growth while also addressing medical complication of achondroplasia.

    我們期待在本季分享 COACH 試驗數據的第 26 週頂線結果,並看到使用 TransCon CNP 作為未來潛在骨幹療法進一步提高臨床結果標準的巨大潛力,我們相信我們可以實現更大的增長,同時解決軟骨發育不全的醫學併發症。

  • Fundamental to the development of [East] medicine, YORVIPATH, SKYTROFA, TransCon CNP is essential 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 benefit, not possible with other technologies.

    對於[東方]醫學的發展而言,YORVIPATH、SKYTROFA、TransCon CNP 是不可或缺的專有 TransCon 技術平台。憑藉TransCon技術和我們對疾病生物學的深刻理解,我們有可能創造出具有高度差異化治療優勢的藥物,這是其他技術無法實現的。

  • I sent this, our commitment has always been to the patient. It is one of the company's core values. I believe we have demonstrated multiple times over the history of the 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 medicine becomes available to patients.

    我發送了此訊息,我們的承諾始終是為了患者。這是公司的核心價值之一。我相信,在 Ascendis 的歷史上,我們已經多次展示了我們的韌性以及我們適應和找到解決方案以實現這一目標的能力。我們一如既往地致力於確保所有藥品都能提供給患者。

  • Through our collaboration with Novo Nordisk for the development and commercialization of TransCon technology-based products in metabolic and cardiovascular disease and our collaboration partner, VISEN, Eyconis, and [Titan], we continue to work to execute our vision 2030 to create value in [das] teleporting areas, and to innovative business models.

    透過與諾和諾德合作開發和商業化基於 TransCon 技術的代謝和心血管疾病產品,以及與我們的合作夥伴 VISEN、Eyconis 和 [Titan],我們繼續努力實現我們的 2030 願景,在 [das] 傳送領域創造價值並創新商業模式。

  • In summary, 2025 is a transformative year for Ascendis as we grow our global revenues from YORVIPATH, SKYTROFA and seek to obtain key regulatory approvals, deliver robust clinical data, and then [bench] stock with blockbuster potential to drive growth for many years to come.

    總而言之,2025 年對 Ascendis 來說是轉型之年,因為我們將從 YORVIPATH 和 SKYTROFA 中增加全球收入,並尋求獲得關鍵的監管批准,提供可靠的臨床數據,然後提供具有巨大潛力的 [bench] 股票,以推動未來多年的成長。

  • I will now turn to Scott.

    現在我來談談史考特。

  • Scott Smith - Chief Financial Officer, Executive Vice President, Member of the Executive Board

    Scott Smith - Chief Financial Officer, Executive Vice President, Member of the Executive Board

  • Thank you so much, Jan.

    非常感謝,簡。

  • I will touch on some key points surrounding our first-quarter financial results, but for further details, please refer to our 6-K filed today.

    我將談及我們第一季財務業績的一些關鍵點,但有關更多詳細信息,請參閱我們今天提交的 6-K 報告。

  • YORVIPATH's first-quarter revenue increased significantly to EUR44.7 million, up from EUR13.6 million in the fourth quarter of 2024. Steady sequential revenue growth outside the US was augmented by the strong US launch. As Jen discussed, the trends we saw for YORVIPATH in Q1 continue in Q2, both outside and inside the US, and we anticipate that YORVIPATH will have a substantial impact on our financial profile in 2025.

    YORVIPATH 第一季營收大幅成長至 4,470 萬歐元,高於 2024 年第四季的 1,360 萬歐元。美國市場的強勁表現促進了美國以外地區收入的穩定環比增長。正如 Jen 所討論的,我們在第一季度看到的 YORVIPATH 的趨勢將在第二季度延續,無論是在美國境內還是境外,我們預計 YORVIPATH 將對我們 2025 年的財務狀況產生重大影響。

  • Turning to SKYTROFA, revenue for this quarter was EUR51.3 million. Sequentially, pricing and market share remained stable, but revenue in the US was negatively impacted by seasonal items, including reduction in channel inventory and higher co-pay 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. Total revenue for the first quarter was EUR101 million which includes non-product revenue from our collaboration partners.

    談到 SKYTROFA,本季的營收為 5,130 萬歐元。價格和市場份額連續保持穩定,但美國的收入受到季節性因素的負面影響,包括通路庫存減少和共同支付援助增加。這些季節性逆風應該會從第二季開始逆轉。我們也將繼續關注歐元兌美元匯率,以了解其對報告收入的潛在影響。第一季的總收入為 1.01 億歐元,其中包括來自合作夥伴的非產品收入。

  • Turning to expenses for the first quarter, R&D cost totaled EUR86.6 million compared to EUR70.7 million during the first quarter of 2024. The first quarter of 2024 included a favorable EUR10.6 million reversal of prior period write downs of TransCon PTH pre-launch inventories.

    談到第一季的支出,研發成本總計 8,660 萬歐元,而 2024 年第一季為 7,070 萬歐元。2024 年第一季,TransCon PTH 上市前庫存減記金額為 1,060 萬歐元。

  • SG&A expenses in the first quarter of 2025 totaled EUR101 million compared to EUR66.8 million during the first quarter of 2024. The EUR34 million increase was primarily driven by global commercial expansion. Total operating expenses were EUR188 million for the first quarter of 2025. As a result of the VISEN IPO, we recognized a non-cash gain of EUR33.6 million as part of share of profit loss of associates, and we retained 39% ownership of VISEN.

    2025 年第一季的銷售、一般及行政費用總計 1.01 億歐元,而 2024 年第一季為 6,680 萬歐元。3400萬歐元的成長主要得益於全球商業擴張。2025 年第一季總營運費用為 1.88 億歐元。由於 VISEN 首次公開募股,我們確認了 3,360 萬歐元的非現金收益,作為聯營公司利潤損失份額的一部分,並且我們保留了 VISEN 39% 的所有權。

  • Net finance expenses for the first quarter of 2025 were EUR15.9 million driven primarily by non-cash items. Net cash financial income for the first quarter of 2025 was EUR3.3 million. We ended the first quarter of 2025 with cash and cash equivalents totaling EUR518 million compared to EUR560 million as of December 31, 2024.

    2025 年第一季的淨財務支出為 1,590 萬歐元,主要由非現金項目造成。2025 年第一季淨現金財務收入為 330 萬歐元。截至 2025 年第一季度,我們的現金和現金等價物總額為 5.18 億歐元,而截至 2024 年 12 月 31 日為 5.6 億歐元。

  • Turning to the remainder of 2025, we expect substantial revenue growth driven by the global launch of YORVIPATH with a continued solid contribution from SKYTROFA. We are not providing revenue guidance for SKYTROFA or YORVIPATH at this time. For SKYTROFA, 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.

    展望 2025 年剩餘時間,我們預計 YORVIPATH 的全球推出將推動營收大幅成長,而 SKYTROFA 將繼續帶來穩健的貢獻。目前,我們還沒有為 SKYTROFA 或 YORVIPATH 提供收入指導。對於 SKYTROFA,我們相信第三方數據中可見的處方成長將追蹤 2025 年的連續收入成長,預計價格將保持穩定和混合,包括正常的季節性。

  • For YORVIPATH, outside the US, we continued 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.

    對於 YORVIPATH,在美國以外,我們的收入持續穩定成長,而在美國境內,我們的發布進展非常順利。隨著時間的推移,我們將繼續幫助投資者了解吸收和償還動態。

  • With that operator, we are now ready to take questions.

    有了這個接線員,我們現在就可以回答問題了。

  • Operator

    Operator

  • (Operator Instructions)

    (操作員指示)

  • Jessica Fye, JPMorgan.

    潔西卡費伊(Jessica Fye),摩根大通。

  • Jessica Fye - Analyst

    Jessica Fye - Analyst

  • Hey guys, good afternoon. Congrats on the strong results of YORVIPATH. 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 YORVIPATH is, say, at steady state. And then the follow up would just be, was there any initial channel still reflected in the one revenue number? And if so, can you quantify that? Thank you.

    大家好,下午好。恭喜 YORVIPATH 取得優異成績。這個數字似乎反映出報銷執行情況非常好。您能否談談您對 YORVIPATH 達到穩定狀態後最終將獲得報銷的患者比例的最新預期。那麼接下來的問題是,任何初始管道是否仍反映在一個收入數字中?如果是的話,您能量化嗎?謝謝。

  • Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

    Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

  • Thanks, Jess, for the questions. And Scott would take the easy one, the last one.

    謝謝傑西的提問。而史考特會選擇最簡單的那個,也就是最後一個。

  • Scott Smith - Chief Financial Officer, Executive Vice President, Member of the Executive Board

    Scott Smith - Chief Financial Officer, Executive Vice President, Member of the Executive Board

  • Yeah, Jess, 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.

    是的,傑西,渠道庫存在任何時候平均大約需要一到兩週。我們每週發貨一次,因此很難獲得少於一周的渠道。

  • Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

    Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

  • 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 positive trend. We see the adaption of the different PPMs, the different [pair] forms and if I'm some way to come with my own guess about it and this is more a guess because we don't know exactly.

    第二個問題,以及為什麼它有點複雜,因為有人在展望未來。我們看到的是,一個非常積極的趨勢。我們看到了不同 PPM、不同 [對] 形式的適應性,如果我以某種方式對此做出自己的猜測,那麼這更多的是一種猜測,因為我們並不確切知道。

  • There will never be 100% approval of all the patients, 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% to 18% will get reimbursed. But I think, Jay, if you have another comments to it this way.

    永遠不會有所有患者 100% 的同意,我相信如果我們真的成功了,我希望我們贏了,我相信我們會成功,我相信 17% 到 18% 的患者將獲得報銷。但是我想,傑伊,如果你還有其他評論的話,可以這樣說。

  • Jay Wu - Executive Vice President, President US Market

    Jay Wu - Executive Vice President, President US Market

  • 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.

    是的,很高興能參與其中。我認為正如 Jan 所提到的,我們還處於發布的早期階段,對嗎?因此從這個角度來看,我們仍然看到政策正在製定中。因此,現在預測穩定狀態還為時過早。我確實認為,有幾個例子讓我們很有信心,我們正朝著正確的方向前進。

  • 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 a medical exception and in some rare cases even through appeal and or peer to peer, but largely we are seeing many patients get through.

    首先,你會看到政策,無論是從商業角度還是政府角度,優惠政策都已到位,所以我們看到這些政策開始發揮作用。其次,即使沒有正式的政策,我們確實看到患者再次獲得全面批准,無論這是商業計劃還是政府計劃。只是有時可能需要多走幾個步驟,無論是透過醫療例外,還是在極少數情況下甚至透過上訴或點對點,但總體而言,我們看到許多患者都通過了。

  • You will expect a long tail just given that every payer plan is different given the heterogeneity of it in the US, 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 to it.

    由於美國國內的支付計劃存在差異,所以考慮到每個支付計劃都不同,你將會期待一個長尾效應,但總的來說,我們有很多值得鼓勵的地方,我認為更重要的是,它真正強調了這個產品的臨床價值主張,以及支付者、提供者和患者都對它做出反應的事實。

  • Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

    Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

  • I think, but yes, I think some way when I talk about my feeling. 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 that I heard me patients the 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 positioning.

    我認為,但是是的,當我談論我的感受時,我會以某種方式思考。我的感覺是,我相信每個有接受治療願望的患者都應該接受治療,因為我已經看到我聽到我的患者接受治療的好處,但我也接受這樣的現實。但我也相信我們處於獨特的地位。

  • We are addressing a major onward 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 at the same time, there is clear guidelines. They are saying there should be a treatment. Can you find a better case for any patient really not to get the ultimate goal. If you get and want to be on treatment, you should be on treatment.

    我們正在解決一項重大的後續醫療需求。我們確實有一款能真正為患者帶來益處的產品。我們處於這樣的情況,病人被診斷出患有這種疾病的同時,就有明確的指導方針。他們說應該採取治療方法。你能為任何病人找到更好的病例真的不能達到最終的目的。如果您生病並且想要接受治療,您就應該接受治療。

  • Operator

    Operator

  • Tazeen Ahmad, Bank of America Securities.

    Tazeen Ahmad,美國銀行證券。

  • Tazeen Ahmad - Analyst

    Tazeen Ahmad - Analyst

  • Hi, good afternoon. Thanks for taking my question. Jan, can you give us a little bit of color on the splits between US and ex-US revenue for the quarter for YORVIPATH? And can you give us a sense, because it seems like you do have some patients receiving drugs in the US, what the length of time is? I know it's early, but what is the length of time that you're seeing between when a script is written to when the patient is receiving therapy? Thanks.

    嗨,下午好。感謝您回答我的問題。Jan,您能否向我們介紹一下 YORVIPATH 本季度美國和美國以外地區收入的分佈?您能否為我們介紹一下,因為看起來確實有一些患者在美國接受藥物治療,治療時間是多久?我知道現在還為時過早,但是從寫好處方到患者接受治療,您認為需要多長時間?謝謝。

  • Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

    Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

  • Yeah. Let me first address the question between the split between Europe and US. And as you have seen, we are not really describing that in our numbers, but I also believe that we give you good guidance how to potential to calculate it.

    是的。我首先要談談歐洲和美國之間的分裂問題。如您所見,我們並沒有真正用數字來描述這一點,但我相信我們為您提供瞭如何計算它的良好指導。

  • 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 of the acceleration when we get more countries facing 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 EUR4 million to EUR5 million in one quarter to the next quarter.

    正如我們所說,當我們看美國以外的地區時,我們看到了穩定的增長,穩定的加速,但我們首先預計在今年下半年,當更多國家面臨完全償還的局面時,會看到加速的加速。因此,當我查看去年第三季和第四季之間的差異時,發現上一季到下一季的增幅約為 400 萬歐元至 500 萬歐元。

  • So when you think about that, we increased our basic revenue from about EUR14 million to EUR45 million, then you can take basic the difference and then about EUR4 million or EUR5 million, and then I think you would get a number around EUR26 million which are some way reflecting the algorithmic I would use if I should look at it.

    因此,當您考慮到這一點時,我們將基本收入從約 1400 萬歐元增加到 4500 萬歐元,然後您可以取出基本差額,然後再取約 400 萬歐元或 500 萬歐元,然後我想您會得到一個約 2600 萬歐元的數字,這在某種程度上反映了我將使用的演算法。

  • Operator

    Operator

  • Gavin Clark Gartner, Evercore.

    加文·克拉克·加特納,Evercore。

  • Gavin Clark-Gartner - Analyst

    Gavin Clark-Gartner - Analyst

  • 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 line with your expectations with those conversations?

    嘿夥計們,祝賀發布進展順利。首先,只是為了跟進付款人點和有利的訪問,您目前是否已經完成了與大多數商業付款人的談判,並且您是否尊重剩餘的待發布政策,使其與已經發布的政策相似,並且回扣是否符合您對這些對話的預期?

  • Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

    Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

  • Yeah, it's a question. I will take the global perspective. As you've heard about ex-US, we are basically fully reimbursed now in 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.

    是的,這是一個疑問。我將以全球視野看待問題。正如您在美國以外聽到的,我們現在在德國基本上已經得到了全額報銷。您可以找到最終的標價。我們對奧地利也有這樣的規定。在法國,我們有一個 AP2 計畫。我們也可以在希臘實施不同的計劃。

  • 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 the end of '26, we believe we will have the vast majority of the foreign country being fully reimbursed.

    因此,除美國外,我們正在努力使所有國家都獲得全額補償。這需要時間。這件事情不僅會在 2025 年發生,而且會在 2026 年發生,當我們進入 2026 年底時,我們相信絕大多數外國債務都會得到全額償還。

  • If you come to the US, which there is a strong focus on. Don't forget the more than 300,000 patients outside the US. I think Jay can give you more color about his discussion with the different, plans in the US.

    如果你來美國,那裡的重點就是這一點。不要忘記美國境外還有 30 多萬名患者。我認為傑伊可以給你更詳細地介紹他對美國不同計劃的討論。

  • Jay Wu - Executive Vice President, President US Market

    Jay Wu - Executive Vice President, President US Market

  • Thanks, Jan, and thanks, Gavin, for the question. Yes, to answer your question, the conversations with payers have been going very well.

    謝謝 Jan 和 Gavin 提出的問題。是的,回答你的問題,與付款人的對話進展非常順利。

  • 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.

    我們針對國家帳戶制定了多項商業政策。因此,雖然我們沒有對每項政策進行評論,但這讓您感覺到我們在那裡獲得了很多良好的牽引力,並且實施的政策是有利的並且與標籤一致。雖然我們不會評論彼此之間的總淨安排,但我要說的是,這與我們之前所說的是相稱的。

  • 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 reflect that.

    這是第一個也是唯一一個在罕見疾病領域獲得批准的產品,具有我們堅信的臨床價值優勢,我們認為我們已經能夠向付款人傳達這一點,從這個角度來看,它與我們帶來的令人難以置信的臨床價值主張相稱,我們當然會以此為先導,並期望它能夠反映這一點。

  • Gavin Clark-Gartner - Analyst

    Gavin Clark-Gartner - Analyst

  • Okay, great. And then quick follow up for the new prescriptions that are coming in. What proportion are Natpara or PTH naive versus experienced? Thank you.

    好的,太好了。然後快速跟進即將收到的新處方。Natpara 或 PTH 新手和經驗豐富的人的比例是多少?謝謝。

  • Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

    Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

  • I don't think we really have the full insight exactly how many docs coming from the Natpara share, you can potentially 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, Sherrie, you can discuss exactly how many Natpara patients that we believe has been transferred over.

    我認為我們並沒有真正全面了解 Natpara 共享了多少文檔,如果我們對此有了更多的了解,您可以糾正我。我們上週討論了一些問題。我們知道,絕大多數患者來自傳統療法,但謝麗,也許您可以討論我們認為到底有多少 Natpara 患者已經轉過來。

  • Sherrie Glass - Chief Business Officer

    Sherrie Glass - Chief Business Officer

  • Yes, thanks for the question, Gavin, and yes, Jan's right, 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 something like 10% to 15% of patients that have been on some sort of PTH in the past, and then, a subset of those were more recently on Natpara. But the bottom line is overwhelmingly coming from conventional therapy.

    是的,謝謝你的提問,加文,是的,簡是對的,在這一點上數據並不完全確定,但我們知道,我們的絕大多數患者都來自傳統療法。這非常清楚。我們確實有大約 10% 到 15% 的患者過去曾服用過某種 PTH,其中一部分患者最近開始服用 Natpara。但最終結果絕大多數還是來自傳統療法。

  • Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

    Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

  • Yeah, from that perspective, we believe that in Q2 and Q3, we will see the majority of the rest of the Natpara patients coming over because, Aimee, you're sitting with the patients, you can explain that there is a letter coming out explaining that you are in a position that they're [starving].

    是的,從這個角度來看,我們相信在第二季度和第三季度,我們將看到其餘大部分 Natpara 患者過來,因為,艾米,你和患者坐在一起,你可以解釋說,有一封信出來解釋你處於他們[飢餓]。

  • Aimee Shu - Executive Vice President, Chief Medical Officer

    Aimee Shu - Executive Vice President, Chief Medical Officer

  • Right. There's still, as far as we know, there's still some supply and so they still may be on it, but we know it will exhaust.

    正確的。據我們所知,仍然有一些供應,所以他們可能仍在使用,但我們知道它會耗盡。

  • Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

    Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

  • So the basic, when we look at the patient that's coming over, the 1,750, this is not Natpara patients. This is basic patient that's coming from conventional therapy, the 35,000 patients we are addressing now. So there is a lot of deepness to go in this way.

    因此,基本情況是,當我們查看前來就診的患者時,這 1,750 名患者並不是 Natpara 患者。這是接受常規療法治療的基礎患者,我們現在正在治療 35,000 名患者。因此,在這方面還有很大的深度可以挖掘。

  • Gavin Clark-Gartner - Analyst

    Gavin Clark-Gartner - Analyst

  • Great, congrats on the progress.

    太好了,恭喜你取得進展。

  • Operator

    Operator

  • Derek Archila, Wells Fargo.

    富國銀行的德里克‧阿奇拉 (Derek Archila)。

  • Derek Archila - Analyst

    Derek Archila - Analyst

  • Yeah, congrats on the quarter and thanks for taking the questions. So maybe just first on, can you comment on the depth of prescribing and the number of docs that are really prescribing, maybe two, three, four patients with YORVIPATH and then just to follow up to the last set of questions. Are you seeing patients that are newly diagnosed or well-controlled on conventional therapies move over to YORVIPATH? Thanks.

    是的,恭喜本季取得佳績,感謝您回答問題。所以也許首先,您能否評論一下處方的深度以及真正開處方的醫生數量,也許是兩、三、四名使用 YORVIPATH 的患者,然後再跟進最後一組問題。您是否看到新診斷的患者或透過傳統療法控制良好的患者轉向 YORVIPATH?謝謝。

  • Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

    Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

  • Yeah. If I just could answer all your questions, I will be a happy person. I'm still a happy person. But, when I someway and, Sherrie, you can also someway add in. We cannot really know if there are well-controlled, partly controlled, or not controlled. That is not really anything we can see. We don't believe that it's really part of the reimbursement system. I think why we call them uncontrolled.

    是的。如果我能回答你所有的問題,我會很高興。我依然是個快樂的人。但是,當我以某種方式和 Sherrie 一起時,你也可以以某種方式添加進來。我們無法真正知道是否有良好控制、部分控製或未控制的情況。那其實不是我們能夠看到的東西。我們不相信這確實是報銷制度的一部分。我想為什麼我們稱它們為不受控制的。

  • We like to see more of them because we have an algorithm that basically are saying the 10,000 to 15,000 of the patients we did find as uncontrolled is because they're seen and into in a very high frequent. 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 are received into on most less treatment, they're getting on Natpara. I hope so, but I cannot really know. But we know we really addressed the patients that see the [endo] and high frequency.

    我們希望看到更多這樣的患者,因為我們有一個演算法,基本上可以確定的是,我們發現的 10,000 到 15,000 名不受控制的患者是因為他們被看到的頻率非常高。這就是為什麼我們知道,只要我們去找這位醫生,他就會自動接待所有這些病人。因此,當他們來的時候,控制患者接受的治療最少,他們接受的是 Natpara 治療。我希望如此,但我真的不知道。但我們知道我們確實解決了那些看到 [endo] 和高頻率的患者的問題。

  • I believe that is basically I will call the genius part of our commercial organization really working on not getting patients into the endos. It takes too long time as if I want to go into. Aimee's practice at Stanford. I need to wait three months to get an appointment with her, and I think we cannot wait for patients for that. And that was why we, I think they made a really genius move in the commercial organization, go to the places where we know patient comes in very, very far.

    我相信,這基本上就是我們商業組織的天才之處,真正致力於不讓病人進入內視鏡。我想要進入的話需要太長時間。艾米在史丹佛的診所。我需要等待三個月才能與她預約,我認為我們不能等待病人。這就是為什麼我認為他們在商業組織中做出了真正天才的舉動,去我們知道病人所在的地方,去得很遠的地方。

  • 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 we are seeing the endo much more often than the other ones. So Sherrie, do you have anything to add?

    所以我的期望是,我相信我們有很大比例的不受控制、部分受控,但它主要只是建立在演算法上,因為我們看到內視鏡的頻率比其他的要高得多。那麼 Sherrie,您還有什麼要補充的嗎?

  • Sherrie Glass - Chief Business Officer

    Sherrie Glass - Chief Business Officer

  • I think you covered it well, Jan. I would maybe just say we -- one of the things we're really excited about is we know they're quite a good healthy number of those uncontrolled and partially controlled patients, so something like 10,000 to 15,000 uncontrolled and another 30,000 or so partially controlled. And as Jan said that the uncontrolled patients are seeing their endos four times, or more a year. So we know 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.

    我認為你已經很好地闡述了這一點,Jan。我可能只想說——我們真正感到興奮的事情之一是,我們知道這些未得到控制和部分得到控制的患者數​​量相當可觀,大約有 10,000 到 15,000 名未得到控制的患者和另外 30,000 名左右得到部分控制的患者。正如 Jan 所說,未控制的患者每年要接受四次甚至更多的內視鏡檢查。因此,我們知道 Jan 的觀點,他們正在加入進來,因此我們預計在一年內會看到源源不斷的患者加入並可以選擇服用藥物。

  • Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

    Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

  • Yeah, and one of the reasons why we don't know it is that because it's not part of being reimbursed or not. So if it's a part of the reimbursement system to have this uncontrolled, party control or controlled, we will know much more on it, but it's not a decision in the reimbursement system.

    是的,我們不知道的原因之一是,這不屬於報銷範圍。因此,如果它是報銷系統的一部分,並且不受控制,則由當事方控製或控制,我們將對此了解更多,但這不是報銷系統中的決定。

  • Operator

    Operator

  • Yaron Werber, TD Cowen.

    Yaron Werber,TD Cowen。

  • Joe Schwartz, Leerink Partners.

    Leerink Partners 的 Joe Schwartz。

  • Joseph Schwartz - Analyst

    Joseph Schwartz - Analyst

  • 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 US have adequate resources at their center in order to be able to go back and forth with payers who might not approve reimbursement of YORVIPATH right away just given there's so many patients in the US and a finite number of physician officers who treat them. Could their ability to navigate this process represent a cap on YORVIPATH revenue growth at some point?

    謝謝您,並祝賀本季業績非常強勁。我想知道您是否知道,在美國,有多少目標內分泌科醫生在其中心擁有足夠的資源,以便能夠與付款人進行反覆溝通,因為考慮到美國有如此多的患者,而治療他們的醫生數量有限,這些付款人可能不會立即批准報銷 YORVIPATH。他們駕馭這個過程的能力是否在某種程度上會限制 YORVIPATH 的收入成長?

  • Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

    Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

  • Joe it's something that Jay and the commercial organization had really a lot of thoughtful thinking about it. How can we ensure the journey for the 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 SKYTROFA, the 10,000 patients we took big portion of them to medical exceptions, 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 learn from the SKYTROFA.

    喬,傑伊和商業組織確實對此進行了深思熟慮。我們如何確保患者、醫生以及處理此事的辦公室的旅程真正成為我們所能想到的最舒適的旅程。我認為我們在這個領域擁有豐富的經驗,主要來自我們的 SKYTROFA,我們將 10,000 名患者中的很大一部分納入醫療例外情況,我認為我們從中獲得了經驗。我們正在建立一個系統,我認為這就是為什麼我們有可能成為真正有能力處理從 SKYTROFA 學到的所有要素中的醫療例外情況的公司之一。

  • Jay, you can (technical difficulty)

    傑伊,你可以(技術難度)

  • Jay Wu - Executive Vice President, President US Market

    Jay Wu - Executive Vice President, President US Market

  • 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.

    只是為了補充和強調 Jan 所說的話,我們的中心現在是一台運作良好的機器,對嗎?經驗非常豐富,眾所周知,生長激素缺乏症領域是一個管理嚴格的領域。因此,我們的樞紐基礎設施不僅從容量和速度的角度進行了配備,而且我們還擁有強大的現場報銷經理足跡,可以不斷為這些辦事處提供支援。我們對自己根據需求提供資源和支持客戶需求的能力感到滿意。

  • 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, 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.

    當然,對於您的問題,會有一些辦公室,特別是那些業務量較低的辦公室,他們可能會看到較少的病例,我想說,這對於許多其他面臨相同辦公室負擔的專業來說並不獨特。但再次回到 Jan 之前分享的內容,我們裝備精良,有能力確保這不會成為患者接受治療的瓶頸。

  • Joseph Schwartz - Analyst

    Joseph Schwartz - Analyst

  • Thanks for the color. And to follow up, could I just ask what you hope to see in terms of clinical benefit from the combination of TransCon CNP and hGH and the COACH trial?

    謝謝你的顏色。接下來,我能否問一下,您希望從 TransCon CNP 和 hGH 的結合以及 COACH 試驗中看到什麼臨床益處?

  • Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

    Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

  • Yeah, Joe. Now you're asking about the future again and this is 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 research. 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 into it.

    是的,喬。現在你又問起未來了,這很好。我被史考特的聲明吸引,儘管他的語速有點快,我還是聽不懂他在說什麼。但我確實看到了一項有趣的研究。對我來說這很有趣,因為他們試圖採取——這實際上是我自己從未想過的事情,這可能是我認為它真的很吸引人的原因。

  • They looked on achondroplasia and hypochondroplasia. Achondroplasia, as you can see, the more severe form for achondroplasia, hypochondroplasia, a little bit more, you can say easy form for achondroplasia, less severe, less bright because what FDR 3 super activation is really putting a brake on the system. So when you took on achondroplasia, you have the brake full down hypochondroplasia partially down.

    他們研究了軟骨發育不全和軟骨發育不良。軟骨發育不全,正如您所見,軟骨發育不全是較嚴重的形式,軟骨發育不全,稍微嚴重一點,您可以稱其為軟骨發育不全的簡單形式,不太嚴重,不太明亮,因為 FDR 3 超級激活確實對系統產生了阻礙。因此,當您患有軟骨發育不全時,您的煞車就會完全停止,而軟骨發育不全則會部分停止。

  • And then they look what is the difference between growth hormone treatment. Over years with the two different (technical difficulty) achondroplasia and hydrochondroplasia. 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 achondroplasia, you basically get a five, six centimeters, which are typical what we have seen. But when you go up to hypochondroplasia, you're up on the eight centimeters.

    然後他們看看生長激素治療有什麼區別。多年來,人們一直患有兩種不同的(技術難度)軟骨發育不全和軟骨積水。我認為這做得非常好,基本上我可以給你數字。因此,當您患有軟骨發育不全時,您基本上會得到五、六厘米,這是我們所看到的典型情況。但是當你患有軟骨發育不全時,你的身高就會上升到八公分。

  • And I actually think that it was a really smart resource because in some ways what we're doing with CNP, we take an achondroplasia patient and remove the break. So I don't know the results, Joe, but I thought that it gives 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.

    我確實認為這是一種非常聰明的資源,因為從某種程度上來說,我們利用 CNP 治療軟骨發育不全患者並去除骨折。所以我不知道結果,喬,但我認為這讓我堅信我們將制定一個新的治療標準。不僅對身高有好處,對身體的其他部位也有好處。

  • Joseph Schwartz - Analyst

    Joseph Schwartz - Analyst

  • Thanks for the insights.

    感謝您的見解。

  • Operator

    Operator

  • Paul Choi, Goldman Sachs.

    高盛的保羅·崔(Paul Choi)。

  • Paul Choi - Analyst

    Paul Choi - Analyst

  • Hi, thanks. Good afternoon, and congratulations on the commercial progress. On YORVIPATH, 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 and or other patients utilizing it too?

    你好,謝謝。下午好,祝賀商業進展。關於 YORVIPATH,我想問您是否了解該領域可能適用於哪些類型的患者。它幾乎主要針對術後患者嗎?或者您看到其他疾病原因(如遺傳)或其他患者也在使用它?

  • And my second question on TransCon CNP is just, any updated thoughts or plans for development in the youngest patient population, those, shortly after birth through let's say three-years-old, just kind of what you're thinking is there in terms of potentially expanding into that population.

    關於 TransCon CNP 的第二個問題是,對於最年輕患者群體(比如說從出生後不久到三歲的患者群體),您有什麼最新的想法或發展計劃嗎?您認為在擴展到該群體方面是否有可能?

  • Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

    Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

  • 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 could call the genetic, [imological], everything else, and then about 80% coming from the post-surgical. And that was very much reflected in our clinical trials.

    是的,我們先來看看美國的人口統計數據,因為如果你看美國的人口統計數據,你會發現大約 20% 來自遺傳,[非邏輯的] 其他一切,然後大約 80% 來自術後。這在我們的臨床試驗中得到了充分體現。

  • So in our clinical trials we even have ADH-1 patients. We actually had two, even they are extremely hard to find because there are a few of them that have hypochondroplasia. We actually managed to get them in. So from that perspective is our clinical trial, this is basic reflected the numbers. I do know, Aimee, if you have any comments. You can come with all the different genetic variants we have there.

    因此,在我們的臨床試驗中,我們甚至有 ADH-1 患者。我們實際上有兩個,即使它們極難找到,因為其中有幾個患有軟骨發育不全。我們確實成功地讓他們進來了。因此從我們的臨床試驗這個角度來看,這基本上反映了數字。我知道,艾米,如果你有任何意見。你可以攜帶我們那裡的所有不同基因變異。

  • Aimee Shu - Executive Vice President, Chief Medical Officer

    Aimee Shu - Executive Vice President, Chief Medical Officer

  • Yeah, we had three mutations. We had autoimmune polyglandular syndrome type 1, And DiGeorge syndrome, so these were all seen in the trial. We know that they we were seeing them in the expanded access program. You don't always get that information on the commercial. From what we hear from our clinicians who are with whom we speak they're applying this to everybody.

    是的,我們有三種突變。我們患有第 1 型自體免疫性多腺體綜合徵和 DiGeorge 綜合徵,因此這些都在試驗中被發現。我們知道我們在擴展訪問計劃中看到了他們。您並不總是能在廣告中得到這些資訊。根據我們與之交談的臨床醫生的說法,他們將此應用於每個人。

  • Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

    Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

  • So basically, a [hypopera] patient is a hypopera patient, and this is how they define it, and I don't think there is any kind of 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 on 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 US.

    所以基本上,[hypopera] 患者就是 hypopera 患者,這就是他們對其的定義,我認為不存在任何來自背景人口統計的選擇,因為在我們的臨床試驗中,我們擁有廣泛的背景人口統計,如果你看一下獨立於背景的標籤。它不受不處理任何背景的限制。因此,我們相信,我們在商業患者群體中看到的情況實際上只是反映了我們在美國看到的情況。

  • Operator

    Operator

  • Li Watsek, Cantor Fitzgerald.

    李沃特塞克,康托費茲傑拉。

  • Li Watsek - Analyst

    Li Watsek - Analyst

  • Hey guys, wanted to add our congrats as well on a strong lunch. Maybe just first on YORVIPATH, in terms of contracting in the future. I know you're doing the process, but any guidance that you can provide on sort of the trend for growth to nets for the rest of the year, relative to Q1. And I have a follow up.

    嘿夥計們,也想對這頓豐盛的午餐表示祝賀。就未來的合約而言,也許只是首先在 YORVIPATH 上。我知道您正在進行這個過程,但是您能否提供一些關於今年剩餘時間網路成長趨勢(相對於第一季)的指導?我還有一個後續行動。

  • Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

    Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

  • Jay we will take that.

    傑伊,我們會接受的。

  • Scott Smith - Chief Financial Officer, Executive Vice President, Member of the Executive Board

    Scott Smith - Chief Financial Officer, Executive Vice President, Member of the Executive Board

  • I'll just say some preliminary comments. So on 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, probably average very low 20%. And commercial will depend on contracting of which Jay can comment.

    我只想說一些初步的評論。因此,從總額到淨額,您無法擺脫政府的強制性退稅。因此,最大的驅動力可能是醫療保險和醫療補助管道中強制性的政府退稅,平均退稅額可能非常低,為 20%。商業將取決於合同,Jay 可以對此發表評論。

  • Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

    Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

  • Yeah, Jay.

    是的,傑伊。

  • Jay Wu - Executive Vice President, President US Market

    Jay Wu - Executive Vice President, President US Market

  • Just as I echoed before, the contracting should be fairly minimal again reflecting the clinical value proposition that we have. So if there's a change, it won't be materially different just relative to the other markets we've been in.

    正如我之前所說的那樣,合約應該相當少,這再次反映了我們的臨床價值主張。因此,如果發生變化,與我們之前進入的其他市場相比,不會有實質的不同。

  • Li Watsek - Analyst

    Li Watsek - Analyst

  • Okay. And then just follow up on the Phase 2 COACH trial. I know you have a starting dose for SKYTROFA. So is there a sort of a titration scheme here that we should think about and what the top dose that you can go to?

    好的。然後繼續進行第二階段 COACH 試驗。我知道您有 SKYTROFA 的起始劑量。那麼,我們是否應該考慮一種滴定方案,以及可以達到的最高劑量是多少?

  • Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

    Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

  • No, it's basic is that the -- we have a starting dose in for the TransCon rota mode in the combination with CNP and they're also mission IPF one as they do in all trials and If there is any possibility that the physician desire to go up in dose or down the dose, they have the opportunity to do it, but Aimee, you know the protocol is better than I do.

    不,基本情況是 - 我們在與 CNP 結合的 TransCon 輪換模式中有一個起始劑量,並且它們也是 IPF 任務,就像在所有試驗中一樣,如果醫生有可能希望增加劑量或減少劑量,他們就有機會這樣做,但是 Aimee,你知道這個方案比我知道的要好。

  • Aimee Shu - Executive Vice President, Chief Medical Officer

    Aimee Shu - Executive Vice President, Chief Medical Officer

  • (inaudible) was she asking?

    (聽不清楚)她在問嗎?

  • Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

    Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

  • A combination.

    一種組合。

  • Aimee Shu - Executive Vice President, Chief Medical Officer

    Aimee Shu - Executive Vice President, Chief Medical Officer

  • Yes, so there will be a starting dose that is informed both based on the Phase 2 trial that we're currently doing in the combination trial and also from the many -- the conditions we're studying where they're short stature but a sufficient growth hormone axis right so this will give (technical difficulty). Based on what we're seeing so far there can be a very good and safe universal starting do typically.

    是的,所以會有一個起始劑量,這個劑量是根據我們目前正在進行的聯合試驗的第二階段試驗以及我們正在研究的許多情況而決定的,這些情況是身材矮小但生長激素軸充足,所以這將提供(技術難度)。根據我們目前所看到的情況,通常可以有一個非常好且安全的通用起始步驟。

  • Li Watsek - Analyst

    Li Watsek - Analyst

  • Okay, thank you.

    好的,謝謝。

  • Operator

    Operator

  • Ellie Merle, UBS.

    瑞銀的艾莉·梅爾(Ellie Merle)。

  • Ellie Merle - Analyst

    Ellie Merle - Analyst

  • Okay guys, thanks for taking the question. I'm curious just the feedback from the early patient starts, how the titration process has gone on YORVIPATH, just logistically, kind of any color anecdotes and 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.

    好的,謝謝大家回答這個問題。我很好奇早期患者的回饋,YORVIPATH 的滴定過程如何,從邏輯上講,有任何色彩軼事,以及醫生和患者的進展如何。對於任何顏色,就您目前看到的補充率而言,我知道現在還為時過早,但是,我很好奇您在那裡看到了什麼趨勢。謝謝。

  • Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

    Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

  • What I'm typically looking on is numbers. And one of the numbers I look a lot is two things is adherence. Second number I'm looking on is how many patients are dropping out.

    我通常關注的是數字。我經常關注的數字之一是堅持。我正在關注的第二個數字是退出治療的患者數量。

  • I think it's too good numbers to look if you have a successful, meaningful treatment of the patient with really addressing a major on medical need and really do that. And when I look on the adherents. 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 on the 1%. And we see the same thing everywhere. If you start on YORVIPATH, you stay on YORVIPATH. And that is a chronic treatment the rest of your life.

    我認為,如果你對患者進行了成功且有意義的治療,並真正解決了主要的醫療需求,那麼這個數字就太好了。當我看著信徒們。它與我們在臨床試驗中看到的一樣高,這確實是獨一無二的,我們已經為您提供了 1% 的德國數據。我們在任何地方都可以看到同樣的現象。如果您從 YORVIPATH 開始,那麼您就會留在 YORVIPATH。這將是你餘生中需要接受的慢性治療。

  • Operator

    Operator

  • Kelly Shi, Jefferies.

    傑富瑞 (Jefferies) 的凱莉施 (Kelly Shi)。

  • Unidentified Participant

    Unidentified Participant

  • Hi, good afternoon. This is Jose for Kelly. Congrats on the strong quarter, and thanks for taking our question.

    嗨,下午好。我是凱利的何塞。恭喜本季業績強勁,感謝您回答我們的問題。

  • 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 YORVIPATH, would you consider running a clinical utility trial to facilitate uptake in milder patients? Thank you.

    我有一個關於付款人動態的問題。主要的報銷阻力有哪些?基於這些動態,您估計在輕度、中度和重度部分可以捕捉多少百分比?另外,關於 YORVIPATH 的臨床價值主張,您是否考慮進行臨床實用性試驗以促進病情較輕的患者接受該療法?謝謝。

  • Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

    Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

  • Related to your first question, The part we discussed before, being uncontrolled, party controlled, or controlled is not part of the reimbursement system.

    與您的第一個問題相關,我們之前討論過的部分,不受控制、受方控製或受控制不屬於報銷系統的一部分。

  • 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. The second question I need to understand a little bit more about what exactly was you wanted us to address.

    所以你不能,我們真的看不到這一點。我們不知道他們究竟來自哪裡,來自不同的群體。我們相信,其中的許多人,也就是大多數人,都是因為無法控制病情,因為他們看醫生的次數太多了。所以這不是報銷討論的任何部分。第二個問題,我需要進一步了解您到底想讓我們解決什麼問題。

  • Unidentified Participant

    Unidentified Participant

  • On the valid preposition of YORVIPATH, 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 SOC? Standard of care.

    關於 YORVIPATH 的有效介詞,即預防腎臟損害的潛力,您是否會考慮進行臨床實用性試驗,以促進 SOC 控制的較溫和患者的吸收?護理標準。

  • Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

    Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

  • I don't think really this is the key element for going on the treatment. I have to 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. 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 YORVIPATH treatment not only for the patient, not only for the physician, but basically for the entire society.

    我並不認為這是進行治療的關鍵因素。我認為繼續治療的關鍵因素是你作為一個人所獲得的益處——你恢復正常。當我們談論治療對社會的益處時,長期風險基本上就是我們所說的健康經濟討論。我們正在評估如何以最佳方式實現這一目標,以真正展示 YORVIPATH 治療不僅對患者、對醫生,而且對整個社會真正能帶來的經濟效益。

  • Unidentified Participant

    Unidentified Participant

  • All right. Thank you.

    好的。謝謝。

  • Aimee Shu - Executive Vice President, Chief Medical Officer

    Aimee Shu - Executive Vice President, Chief Medical Officer

  • You need 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 when that is able to be lowered in whatever way, right, that the kidney function gets better. So there may not -- we'll see what the need is for demonstrating this yet again.

    您需要一些現有的文獻數據,我們可以利用這些數據來證明一些常規療法本身對腎臟有毒性,對嗎?無論以何種方式降低,腎功能都會變得更好。所以可能不會——我們將看看再次證明這一點的必要性。

  • Unidentified Participant

    Unidentified Participant

  • Very helpful. Thank you so much.

    非常有幫助。太感謝了。

  • Operator

    Operator

  • Luca Issi, RBC.

    盧卡·伊西(Luca Issi),RBC。

  • Luca Issi - Analyst

    Luca Issi - Analyst

  • Oh great, thanks so much for taking my question and congrats on the launch here.

    哦,太好了,非常感謝您回答我的問題,並祝賀這裡的發布。

  • Maybe a quick one on competition. BridgeBio presented their data for their molecule earlier 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 YORVIPATH. 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 appreciated. Thanks so much.

    也許是有關競爭的一個簡短的話題。BridgeBio 本週稍早公佈了其分子的數據,我相信 MBX 下個季度也會公佈同樣的數據。所以想知道對這兩種分子的最新想法是什麼,以及您認為它們與 YORVIPATH 相比有多大的競爭力。然後斯科特也許會很快問,考慮到本季度與上一季相比的顯著增長,我們應該如何考慮今年剩餘時間的銷售、一般和行政費用。任何想法,不勝感激。非常感謝。

  • Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

    Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

  • Yeah, you mentioned two compounds. One is the [catiolytic], which are being positioned into a Phase 3 trial of ADH-1. Currently ADH-1 is being treated with YORVIPATH, and they're coming on treatment today. It has been hard for us to find ADH-1 patient and, Sherrie, you went into the claim database and how many patient (multiple speakers) ADH-1.

    是的,你提到了兩種化合物。其中一種是[catiolytic],目前正在進行ADH-1的3期試驗。目前ADH-1正在接受YORVIPATH治療,今天就開始治療。我們很難找到 ADH-1 患者,Sherrie,您進入了索賠資料庫,查到了有多少患者(多位發言者)是 ADH-1。

  • Sherrie Glass - Chief Business Officer

    Sherrie Glass - Chief Business Officer

  • Yeah, there were something like 350 patients who'd had a claim associated with ADH-1 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.

    是的,過去四、五年內大約有 350 名患者提出與 ADH-1 相關的索賠,而去年提出索賠的患者數量甚至更少,所以這個數字非常小。這是來自一個包含數億人生命的大型國家索賠資料庫的數據。

  • Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

    Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

  • So it's not going to change anything for us because we are addressing the position of catalytic into the era of, for example, the what we call chronic hypopara patient that is not ADH-1. I really love science. I don't understand the signs there. We're trying to position into a place of a patient that not have [indutious] PKs. How can you increase the level of industrious PDAs when they're not producing it?

    所以這對我們來說不會改變任何事情,因為我們正在解決催化劑的作用,進入例如我們所說的非 ADH-1 的慢性副甲狀腺功能低下患者。我真的熱愛科學。我不明白那裡的標誌。我們正試圖將其定位到沒有 [inducious] PK 的患者的位置。當 PDA 不生產時,如何提高它們的勤勞程度?

  • They're already producing on [MAX], and there is actually a very nice post of basically showing that they are taking catalytic into four patients for three days or six or something like that, and it's really showing that you cannot increase the secretion of PDAs. 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.

    他們已經在 [MAX] 上進行生產,實際上有一篇非常好的帖子,基本上表明他們正在將催化劑注入四名患者體內三天或六天或類似的時間,這確實表明你無法增加 PDA 的分泌。所以從科學角度來說這並不是我不理解的合理的事情,而且我所看到的所有數據也都顯示了同樣的事情。

  • Scott was saying [there was 5 pages]. Okay, that is one thing. The MAX, this is the once weekly one, and we have the Possibility to develop once weekly products for a long time. And now we're talking of the context of once weekly first. We some ways stalled a little bit because we couldn't see the need and also because of the desire on different ways of living with a hypopera patient where you sometimes need more or less. And it can be done on exercise, seasonal activities, other things that change in your life.

    史考特說[共 5 頁]。好的,這是一回事。MAX,這是每週一次的,我們有可能長期開發每週一次的產品。現在我們首先討論的是每週一次的背景。我們在某些​​方面停滯不前,因為我們看不到這種需要,也因為我們渴望以不同的方式與低血壓患者生活在一起,有時需要更多或更少。它可以應用於運動、季節性活動以及生活中其他發生變化的事情。

  • 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 a once-weekly product, we would develop it as a baseline like basal insulin, and then we still will have a daily product to really be sure they can adjust them up and down.

    由此,我們觀察了患者的狀況,他們的狀況如何?他們多久進行一次上下滴定?我們發現只有一小部分患者的情況穩定。因此,如果我們想要開發每週一次的產品,我們會將其開發為像基礎胰島素一樣的基線,然後我們仍然會有每日一次的產品,以確保他們可以上下調整它們。

  • The technology platform they're utilizing in the, I always get it wrong, MBX or PMX. I cannot remember what is that bike but always get it wrong. But the element of that is basic technology which basic as an active entity is an isolated PTH that state 99.9% associated to basic the element of albumin. I do not know how that ever can activate the phosphate receptor, how they can get into the brain and really restore normal industrious PTH level in the normal distribution you have out through the body. I'm also lost in the science there.

    他們所使用的技術平台,我總是弄錯,MBX 或 PMX。我記不住那輛自行車是什麼,但總是記錯。但其基本技術要素是作為活性實體的分離的PTH,其99.9%與白蛋白的基本要素有關。我不知道它們如何能夠激活磷酸鹽受體,如何能夠進入大腦並真正恢復身體正常分佈中的正常 PTH 水平。我也對那裡的科學感到困惑。

  • Operator

    Operator

  • David Lebowitz, Citi.

    花旗銀行的 David Lebowitz。

  • David Lebowitz - Analyst

    David Lebowitz - Analyst

  • Oh, thank you very much for taking my question.

    哦,非常感謝您回答我的問題。

  • First of all, if you look at the Natpara patients that were left, from its withdrawal, how long do you think it takes until that whole population gets, worked through and then further looking back at Natpara and looking at what you've seen during your first quarter of launch. How would you, aside from the fact that it's the data is just superior, how would you characterize the difference in what patients are showing interest in this therapy?

    首先,如果您看一下自停藥以來剩下的 Natpara 患者,您認為需要多長時間才能使整個人群得到治療,然後進一步回顧 Natpara 並看看您在推出該療法的第一個季度所看到的情況。除了數據更優質之外,您如何描述患者對這種療法的興趣差異?

  • Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

    Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

  • First of all, you cannot compare the clinical benefit between Natpara and YORVIPATH. Natpara had a living as an adjunct therapy. Take a little bit of your daily calcium supplement away. Take a little bit of your act vitamin away, and then you take Natpara. 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.

    首先,您無法比較 Natpara 和 YORVIPATH 之間的臨床益處。Natpara 以輔助治療為生。減少一點每日所需的鈣補充劑。拿掉一點活性維生素,然後服用 Natpara。對你腎功能沒有任何正面的影響。你對生活品質沒有積極的量化態度。我認為這是兩種不同的產品,我們永遠無法將兩者放在一起比較。

  • First question related to when the Natpara patient will be swift. We addressed it a little bit in the beginning of here, the discussion here, where we know that there was a letter from [Tata] indicating that they're getting the last shipment now. And I think as to our notice, the shipping is three months, so we expect that the last series of Natpara patients will come over in either end of Q2 or Q3, that is our expectations.

    第一個問題與 Natpara 患者何時能康復有關。我們在一開始的討論中稍微提到了這一點,我們知道有一封來自 [Tata] 的信表明他們現在正在收到最後一批貨物。我認為,正如我們的通知,運輸時間為三個月,因此我們預計最後一批 Natpara 患者將在第二季或第三季末到來,這是我們的預期。

  • Operator

    Operator

  • Leland Gershell, Oppenheimer.

    利蘭·格謝爾,奧本海默。

  • Leland Gershell - Analyst

    Leland Gershell - Analyst

  • Thanks. Great to see the strong execution on US YORVIPATH. Couple of questions from us, just, apologies if this had been asked before, but with respect to potential benefits on renal, I know, yeah, you had said that, you don't see that as a key kind of driver for YORVIPATH 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, whatever needs they have in terms of conventional therapy.

    謝謝。很高興看到美國 YORVIPATH 的強大執行力。我們有幾個問題,如果這個問題之前被問過,我很抱歉,但關於腎臟的潛在益處,我知道,是的,你說過,你不認為這是 YORVIPATH 普及的關鍵驅動因素,但儘管如此,你能在美國推出初期發表評論嗎?您是否認為,對於那些腎功能損害程度較輕或較重的患者以及他們在常規治療方面有任何需求的患者來說,獲得報銷或藥物治療是更困難還是更容易?

  • And there's a second question. Thank you.

    還有第二個問題。謝謝。

  • Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

    Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

  • It's not a part of the reimbursement process. We don't see that it's the element that the site offers reimbursement and not reimbursement. So it's like the same thing as we discussed on party control, control is not part of the reimbursement process either. It's basic. When you look on the labeling, most planes have adapted. The weight that we basically have in our [la] and it's not defined way like that. That is the first one correction, Aimee, we didn't study in what we call severe renal impairment patients and that is not part of our label, and I think it's Stage 4.

    這不是報銷流程的一部分。我們不認為這是網站提供報銷和不提供報銷的因素。因此,這就像我們討論的派對控制一樣,控制也不是報銷流程的一部分。這是基本的。如果你看一下標籤,你會發現大多數飛機都已經適應了。我們的重量基本上在我們的 [la] 中,並且它不是以那樣的方式定義的。這是第一個更正,艾米,我們沒有對所謂的嚴重腎功能不全患者進行研究,這不是我們標籤的一部分,我認為這是第 4 階段。

  • Aimee Shu - Executive Vice President, Chief Medical Officer

    Aimee Shu - Executive Vice President, Chief Medical Officer

  • That's right, lower than [15].

    沒錯,低於[15]。

  • Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

    Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

  • Which are a very, very low number which are Stage 4 where we never started the drop.

    這是一個非常非常低的數字,這是第 4 階段,我們從未開始投放。

  • Leland Gershell - Analyst

    Leland Gershell - Analyst

  • Got it. Okay, that's helpful. And then, just looking forward to the COACH data, are you -- is there sort of a bar that you have in mind for linear growth, or is this something that you could 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? Thank you.

    知道了。好的,這很有幫助。然後,只是期待 COACH 數據,您是否 - 您是否考慮過線性增長的標準,或者您是否認為這主要受到次要利益的推動,例如身體組成或其他?您如何看待這種組合的功效?謝謝。

  • Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

    Jan Moller Mikkelsen - President, Chief Executive Officer, Executive Director, Member of the Executive Board

  • When we think about achondroplasia, the key element for us is to address complications, but we cannot avoid also to address linear growth. 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.

    當我們考慮軟骨發育不全時,關鍵因素是解決併發症,但我們也無法避免解決線性生長問題。因此,當我們測量線性成長時,該成長是由另一家公司確定的,並且被確定為主要終點,如果可以選擇的話,我個人會選擇另一個終點。

  • But we will because we are forced to do it because it's the establish 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 high velocity height as there is, 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 achondroplasia treatment, and it's not only related to linear growth but also the associated complications.

    但我們會這樣做,因為我們被迫這樣做,因為這是既定的臨床終點,我們將專注於線性成長,它將成為數據的一部分,當我們得出分析的高速高度時,我們基本上會報告這些數據,以及其他相關內容。正如我之前所說,我對此抱有很大的期望。我堅信,我們可以重新設定軟骨發育不全療法的標準,這不僅與線性生長有關,還與相關的併發症有關。

  • Leland Gershell - Analyst

    Leland Gershell - Analyst

  • Alright, thanks so much.

    好的,非常感謝。

  • Operator

    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.

    這是我們回答問題的全部時間。今天的電話會議到此結束,感謝您的參與,現在可以斷開連線了。