Ascendis Pharma A/S (ASND) 2025 Q2 法說會逐字稿

Good day and thank you for standing by. Welcome to the Q2 2025 Ascendis Pharma earnings conference call. (Operator Instructions) Please be advised that today's conference is being recorded.

您好，感謝您的支持。歡迎參加 2025 年第二季 Ascendis Pharma 收益電話會議。（操作員指示）請注意，今天的會議正在錄音。

I would now like to hand the conference over to your first speaker today, Scott Smith, Ascendis Pharma Inc CFO. Please go ahead.

現在，我想將會議交給今天的第一位發言人，Ascendis Pharma Inc 財務長 Scott Smith。請繼續。

(spoken in foreign language), operator, and thank you, everyone, for joining our second-quarter 2025 financial results conference call. I'm Scott Smith, Executive Vice President and Chief Financial Officer at Ascendis Pharma. Joining me on today's call are Jan Møller Mikkelsen, President and Chief Executive Officer; Sherrie Glass, Chief Business Officer; Jay Wu, Executive Vice President and President, US Market; Aimee Shu, Executive Vice President of Endocrine and Rare Disease Medical Science and Chief Medical Officer.

（用外語說），接線員，感謝大家參加我們的 2025 年第二季財務業績電話會議。我是 Scott Smith，Ascendis Pharma 的執行副總裁兼財務長。參加今天電話會議的還有總裁兼執行長 Jan Møller Mikkelsen、商務長 Sherrie Glass、執行副總裁兼美國市場總裁 Jay Wu 以及內分泌與罕見醫學科學執行副總裁兼首席醫療官 Aimee Shu。

Before we begin, I would like to remind you that this conference call will contain forward-looking statements that are intended to be covered under the Safe Harbor provided by the Private Securities Litigation Reform Act. Examples of such statements may include, but are not limited to, statements regarding our commercialization and continued development of SKYTROFA and YORVIPATH as well as certain financial expectations, our pipeline candidates, and our expectations with respect to their continued progress 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 timing and the results of our regulatory decisions.

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。此類聲明的示例可能包括但不限於有關我們對 SKYTROFA 和 YORVIPATH 的商業化和持續開發的聲明以及某些財務預期、我們的候選藥物以及我們對其持續進展潛在商業化的預期、我們的戰略計劃、合作夥伴關係和投資、我們關於臨床藥物管線的目標（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以及我們對監管決策的時間和結果的預期。

These statements are based on information that is available to us as of today. Actual results may 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.

這些聲明是基於我們今天所掌握的資訊。實際結果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。除非法律要求，我們不承擔隨著情況變化而更新這些聲明的義務。

Additional information concerning the factors that could cause 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, or TransCon hGH is now approved in The United States by the FDA for the replacement of endogenous growth hormone in adults with growth hormone deficiency.

有關可能導致實際結果出現重大差異的因素的更多信息，請參閱今天新聞稿中的前瞻性陳述部分以及我們於 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 生長激素或 TransCon hGH 現在已獲得美國 FDA 批准，用於取代患有生長激素缺乏症的成年人的內源性生長激素。

In addition to the treatment of pediatric GHD, and then the EU has received MAA authorization from the European Commission for the treatment of pediatric GHD. 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 Medicines and Health Products Regulatory Agency have granted marketing authorization for TransCon PTH as a replacement therapy indicated for the treatment of adults with chronic hypoparathyroidism.

除了治療兒童GHD外，隨後歐盟又獲得了歐盟委員會的MAA授權，用於治療兒童GHD。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健康產品管理局已授權 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 investigational products, the safety and efficacy of the product candidates have 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.

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。

On the call today, we'll discuss our second-quarter 2025 financial results, and we'll provide further business updates. Following some prepared remarks, we'll then open up the call for questions.

在今天的電話會議上，我們將討論 2025 年第二季的財務業績，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。

Thanks, Scott. Good afternoon, everyone. The second quarter of 2025 demonstrated strong momentum towards fulfilling our Vision 2030 as we progress towards blockbuster status for multiple products and expand our engine for future innovation. The continued strong global launch of YORVIPATH increases our confidence that YORVIPATH is on the way to become a blockbuster product with durable global leadership for the treatment of hypoparathyroidism. FDA granting us priority review for TransCon CNP, recognizing its potential if approved, to provide a significant improvement in the safety and/or effectiveness of the treatment of achondroplasia.

謝謝，斯科特。大家下午好。2025 年第二季度，隨著我們多款產品邁向重磅級地位並擴展未來創新引擎，我們朝著實現 2030 願景的方向邁出了強勁的步伐。YORVIPATH 在全球的持續強勢上市增強了我們的信心，我們相信 YORVIPATH 將成為治療甲狀旁腺功能減退症領域具有持久全球領導地位的重磅產品。FDA 授予我們對 TransCon CNP 的優先審查權，承認其一旦獲得批准，將有可能顯著提高軟骨發育不全治療的安全性和/或有效性。

The announcement of the interim Phase 2 result from the first combination therapy trial of TransCon CNP and TransCon growth hormone highlights our potential to boost healthy growth in achondroplasia. And we achieved that first of many planned label expansions for SKYTROFA when the FDA approved it for treatment of adult growth hormone deficiency. I will review these key developments in more detail in my prepared remarks.

TransCon CNP 和 TransCon 生長激素首次聯合治療試驗的中期第 2 階段結果的公佈凸顯了我們促進軟骨發育不全症健康生長的潛力。當 FDA 批准 SKYTROFA 用於治療成人生長激素缺乏症時，我們實現了 SKYTROFA 眾多計劃標籤擴展中的第一次。我將在準備好的發言中更詳細地回顧這些關鍵發展。

Beginning with YORVIPATH. Revenues in the second quarter reached EUR103 million, more than double of Q1, despite a strong currency headwind. In The US, from launch to June 30, more than 1,500 prescribers wrote prescriptions for around 3,100 unique patients, reflecting both the deep unmet medical need and compelling product profile. For US patients receiving a prescription for YORVIPATH, the majority have received payer approval within three months.

從 YORVIPATH 開始。儘管面臨強勁的貨幣逆風，第二季的營收仍達到 1.03 億歐元，比第一季成長一倍多。在美國，從產品上市到 6 月 30 日，已有超過 1,500 名處方人員為大約 3,100 名患者開出了處方，這不僅反映了尚未滿足的醫療需求，也反映了引人注目的產品特性。對於獲得 YORVIPATH 處方的美國患者，大多數在三個月內獲得了付款人的批准。

Outside of the US, we continue to see steady YORVIPATH revenue growth in both our Europe direct and international markets. And we currently expect further acceleration of the revenue growth when YORVIPATH reimbursement becomes available in additional YORVIPATH-direct countries. With a broad label covering all types of chronic hypoparathyroidism, supported by international guidelines, and a prominent reference to YORVIPATH in recently published best practice consent statements, we expect growth to continue.

在美國以外，我們繼續看到 YORVIPATH 在歐洲直接和國際市場的營收穩定成長。我們目前預計，當 YORVIPATH 報銷在更多 YORVIPATH 直銷國家/地區可用時，收入成長將進一步加速。由於標籤廣泛，涵蓋所有類型的慢性副甲狀腺功能減退症，並得到國際指南的支持，並且在最近發布的最佳實踐同意聲明中突出提到了 YORVIPATH，我們預計增長將繼續。

We have ongoing clinical programs to support label expansion. For example, in older children, an initiated PaTHway60, trial, a single-arm safety and efficacy trial to support titration up to 60 microgram doses in the US. The primary endpoint of this trial will be efficacy at 26 weeks, the same as our pivotal Phase 3 trial endpoint.

我們有正在進行的臨床項目來支持標籤擴展。例如，在年齡較大的兒童中，啟動了一項 PaTHway60 試驗，這是一項單臂安全性和有效性試驗，支持在美國將劑量滴定至 60 微克。試驗的主要終點是 26 週的療效，與我們的關鍵 3 期試驗終點相同。

We are building towards UFS long-term global leadership based on three key pillars: differentiation, demand, and access. I will first speak about differentiation through mode of action. A replacement therapy for hypoparathyroidism must maintain the same mode of action as endogenous PTH throughout the body and sustain physiological levels of PTH 24 hours, 7 days a week. Based on all the data we have seen, YORVIPATH is the only product to demonstrate it can do this, with normalization of key elements, such as serum calcium phosphate, kidney function, bone turnover, and quality of life.

我們正基於三大支柱——差異化、需求和訪問——打造 UFS 的長期全球領導地位。我首先要講的是透過作用方式來區分。副甲狀腺功能低下症的替代療法必須保持與全身內源性 PTH 相同的作用方式，並每週 7 天、每天 24 小時維持 PTH 的生理水平。根據我們所看到的所有數據，YORVIPATH 是唯一能夠做到這一點的產品，它能夠使血清磷酸鈣、腎功能、骨轉換和生活品質等關鍵要素正常化。

Second is demand. Where YORVIPATH is available, we see strong interest and growing enrollment. For the US market, in just two full quarters, we had around 3,100 unique patients enrolled across more than 1,500 prescribers. We're seeing a broad uptake across the entire country. And with our estimate of 70,000 to 90,000 patients in the US, we still have ample room to grow. Outside the US, we have recognized revenue for more than 30 countries. And currently, we have commercial agreements covering more than 75 countries.

第二是需求。在 YORVIPATH 開展的地方，我們看到了濃厚的興趣和不斷增長的報名人數。對於美國市場，僅僅兩個季度，我們就有超過 1,500 名處方醫生招募了約 3,100 名獨立患者。我們看到全國各地都在廣泛採用這種做法。我們估計美國有 70,000 至 90,000 名患者，因此我們仍有足夠的成長空間。除美國以外，我們已在 30 多個國家實現了收入。目前，我們的商業協議已涵蓋 75 多個國家。

Third is access. In the US, we see favorable access continue to improve, with approvals coming across all payer segments. In Euro direct, we have full commercial launch in Germany, Austria, and now Spain. We expect additional commercial launches later this year both in Euro direct and international markets. In Japan, our partner, [Titan], expects approval for YORVIPATH later this quarter.

第三是訪問。在美國，我們看到有利的准入條件不斷改善，所有付款方都獲得了批准。在 Euro direct，我們已在德國、奧地利和西班牙全面推出商業活動。我們預計今年稍後將在歐洲直接市場和國際市場上推出更多商業產品。在日本，我們的合作夥伴 [Titan] 預計 YORVIPATH 將在本季度稍後獲得批准。

We consistently hear about how transformative YORVIPATH has been for patients, and do not believe that any publicly disclosed drug in clinical development has the potential to meet this efficacy and safety bar set by YORVIPATH. As shown in our clinical trial, this has been extended for all patient groups, post-surgery HP patients, to small genetic subtypes like DiGeorge syndrome, ADS1, and idiopathic hypoparathyroidism. Notably, YORVIPATH has broad approval from the FDA, the European Commission, and other regulatory authorities for the treatment of all forms of chronic hypoparathyroidism.

我們不斷聽到 YORVIPATH 對患者產生了多大的改變，並且不相信任何公開披露的臨床開發藥物有潛力達到 YORVIPATH 設定的療效和安全性標準。正如我們的臨床試驗所示，這已擴展到所有患者群體、術後 HP 患者以及 DiGeorge 症候群、ADS1 和特發性副甲狀腺功能減退症等小遺傳亞型。值得注意的是，YORVIPATH 已獲得 FDA、歐盟委員會和其他監管機構的廣泛批准，可用於治療所有形式的慢性副甲狀腺功能減退症。

For all of the above reasons, we are confident that YORVIPATH has the potential to become a durable blockbuster over time. And we continue to expand our global leadership position in the treatment of hypoparathyroidism.

基於以上所有原因，我們相信 YORVIPATH 有潛力隨著時間的推移成為一部持久的暢銷書。我們將繼續擴大在副甲狀腺功能減退症治療領域的全球領導地位。

Moving now to TransCon CNP. We believe TransCon CNP is moving the bar on safety, efficacy, tolerability, and reducing treatment burden. And we believe TransCon CNP is well-positioned to become the leading monotherapy treatment for achondroplasia. In clinical trials, we have seen the desired linear growth across all ages. And to our knowledge, once weekly TransCon CNP is the only product to show statistically significant improvement beyond linear growth compared to placebo in a pivotal trial, for example, improvements in leg bowing and quality of life.

現在轉到 TransCon CNP。我們相信 TransCon CNP 正在提高安全性、有效性、耐受性並減輕治療負擔的標準。我們相信 TransCon CNP 有能力成為治療軟骨發育不全的領先單一療法。在臨床試驗中，我們看到了各個年齡層都呈現出理想的線性成長。據我們所知，每週一次的 TransCon CNP 是唯一一種在關鍵試驗中與安慰劑相比顯示出超越線性增長的統計學顯著改善的產品，例如腿部彎曲和生活品質的改善。

We have demonstrated a safety and tolerability profile comparable to placebo, including no evidence of hypotensive effect and extremely low frequency of mild injection site reactions. Since our announcement of monotherapy data, we have engaged with patients, advocates, physicians, and regulators. All have appreciated the differentiating ability of TransCon CNP in comparison to placebo to increase linear growth while also leading to stronger muscle function, improved body proportionality, and leg bowing, and reducing overall the burden of achondroplasia-related complications for the majority of treated children. And, of course, patients and caregivers appreciate the much lower burden of once-weekly injection.

我們已經證明了其安全性和耐受性與安慰劑相當，包括沒有降血壓作用的證據和輕微注射部位反應的發生頻率極低。自從我們公佈單一療法數據以來，我們就與患者、倡導者、醫生和監管機構進行了接觸。與安慰劑相比，TransCon CNP 具有顯著的差異化能力，可以增加線性生長，同時增強肌肉功能，改善身體比例和腿部彎曲，並減輕大多數接受治療的兒童的軟骨發育不全相關併發症的總體負擔。當然，患者和照護者也非常欣賞每週注射一次的較低負擔。

During 2025, the FDA accepted our NDA submission for priority review, with a PDUFA date of November 30, recognizing TransCon CNP as a therapy that could, if approved, provide a significant improvement in safety and/or effectiveness.

2025 年，FDA 接受了我們的 NDA 提交並進行優先審查，PDUFA 日期為 11 月 30 日，FDA 承認 Tr​​ansCon CNP 是一種療法，如果獲得批准，可以顯著提高安全性和/或有效性。

Next, I will review our combination trial results. As we look forward to the anticipated approval of TransCon CNP as monotherapy, we are investigating it in combination with our once-weekly TransCon growth hormone in children with achondroplasia in our COACH trial. In June 2025, we announced Week 26 interim results which showed a clear boost in linear growth and body proportionality improvement, with a safety and tolerability profile consistent with those observed for monotherapy.

接下來，我將回顧我們的聯合試驗結果。我們期待 TransCon CNP 作為單一療法獲得批准，我們正在 COACH 試驗中研究將其與每週一次的 TransCon 生長激素聯合用於治療軟骨發育不全兒童。2025 年 6 月，我們公佈了第 26 週的中期結果，結果顯示線性生長和身體比例改善明顯，其安全性和耐受性與單一療法的觀察結果一致。

In the combination trial, both treatment groups exceeded the 97% factor for growth of an average state of children, meaning they are achieving linear growth at a rate higher than an average child. The Week 26 data demonstrated potential to boost growth of around 3 times above that observed with monotherapy, addressing the hyperactive FDR3 receptor pathway, supporting the scientific rationale for treating with TransCon CNP and TransCon growth hormone combined. These results await our procedures in achondroplasia. Importantly, we see clear indication that it is healthy growth with linear growth accompanied by improvement in body proportionality and without acceleration of bone age. All patients continue in the study as of today.

在聯合試驗中，兩個治療組都超過了兒童平均生長狀態的 97%，這意味著他們實現的線性生長速度高於平均兒童。第 26 週的數據顯示，與單一療法相比，該療法具有促進生長約 3 倍的潛力，解決了過度活躍的 FDR3 受體通路問題，支持使用 TransCon CNP 和 TransCon 生長激素聯合治療的科學原理。這些結果有待我們在軟骨發育不全症的治療中驗證。重要的是，我們看到了明顯的跡象，表明這是一種健康的生長，線性生長伴隨著身體比例的改善，並且沒有骨齡加速。截至今天，所有患者均繼續參與研究。

These results enforce the role of TransCon CNP as a strong fundamental therapy in achondroplasia. We look forward to our 12-month release later this year and plan to start a Phase 3 study of the combination therapy in children with achondroplasia by the end of 2025. In addition, we also expect [Trinity], a pivotal combination trial in hypochondroplasia.

這些結果強化了 TransCon CNP 作為軟骨發育不全症強而有力基礎療法的作用。我們期待今年稍後發布 12 個月的版本，並計劃在 2025 年底之前開始對患有軟骨發育不全的兒童進行聯合治療的 3 期研究。此外，我們也期待[Trinity]，一項針對軟骨發育不良症的關鍵聯合試驗。

I will now turn to SKYTROFA. SKYTROFA is established as a high-value brand and a treatment choice for pediatric growth hormone deficiency. We recently received FDA approval for adult growth hormone deficiency, and with further label expansion planned, SKYTROFA remains a fundamental pillar in our strategy to become the global leader in the treatment of growth disorders.

現在我將轉向 SKYTROFA。SKYTROFA 是一個高價值品牌，也是治療兒童生長激素缺乏症的首選。我們最近獲得了 FDA 對成人生長激素缺乏症的批准，隨著進一步的標籤擴展計劃，SKYTROFA 仍然是我們成為治療生長障礙的全球領導者策略的基本支柱。

Q2 revenue for SKYTROFA was EUR51 million. We continue to see growth in the number of people treated with SKYTROFA, based on new patient starts. The expected recent label expansion for adult growth hormone deficiency is expected to further drive long-term growth. Our market research shows SKYTROFA is the treatment choice for pediatric growth hormone deficiency among patients and physicians and we believe we can achieve the same status for the treatment of adult growth hormone deficiency. Our phase three basket trial of SKYTROFA planned to begin later this year will include a range of established daily growth hormone indications, including ISS, SHOX deficiency Turner, and SGA.

SKYTROFA 第二季營收為 5,100 萬歐元。根據新患者人數，我們持續看到接受 SKYTROFA 治療的人數不斷增加。預計近期成人生長激素缺乏症標籤的擴大將進一步推動長期成長。我們的市場研究表明，SKYTROFA 是患者和醫生治療兒童生長激素缺乏症的首選，我們相信我們在治療成人生長激素缺乏症方面也能取得同樣的地位。我們計劃於今年稍後開始對 SKYTROFA 進行第三階段籃子試驗，試驗將涵蓋一系列已確定的日常生長激素適應症，包括 ISS、SHOX 缺乏症 Turner 和 SGA。

I often say that Ascendis is just getting started. Falling closely behind this major growth opportunity, our research team is developing the next stage of innovative TransCon technology and product candidates. In addition, our ongoing collaboration with Novo Nordisk for the development and commercialization of TransCon-based products in metabolic and cardiovascular diseases continues to make progress towards the clinic.

我常說 Ascendis 才剛起步。跟上這項重大成長機遇，我們的研究團隊正在開發下一階段的創新 TransCon 技術和產品候選。此外，我們與諾和諾德公司正在進行的合作，旨在開發和商業化基於 TransCon 的代謝和心血管疾病產品，並在臨床方面繼續取得進展。

Ascendis is demonstrating a significant inflection in revenue growth. We are generating important new clinical data, working towards additional key label expansions. We are advancing new blockbuster opportunities to drive growth for many years to come and fulfill our Vision 2030. And we're already preparing for our next vision.

Ascendis 的營收成長呈現顯著的變化。我們正在產生重要的新臨床數據，致力於擴展更多關鍵標籤。我們正在推動新的重大機遇，以推動未來多年的成長並實現我們的 2030 願景。我們已經在為下一個願景做準備。

I will now turn it over to Scott.

現在我將把發言權交給史考特。

Thank you, Jan. I will touch on some key points surrounding our second-quarter financial results, but for further details, please refer to our Form 6-Ks filed today.

謝謝，Jan。我將談及我們第二季度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 表格。

For Q2, our total product revenue was EUR153.7 million, which includes a negative sequential foreign currency exchange rate impact of EUR7.6 million. SKYTROFA revenue for the quarter was EUR50.7 million, including a EUR1.8 million negative currency impact. YORVIPATH delivered strong performance with revenue more than doubling to EUR103 million, up from EUR44.7 million in Q1 2025. This revenue growth was achieved despite a negative sequential currency headwind of EUR5.8 million.

第二季度，我們的產品總營收為 1.537 億歐元，其中包括 760 萬歐元的負面連續外匯匯率影響。SKYTROFA 本季營收為 5,070 萬歐元，其中包括 180 萬歐元的負面貨幣影響。YORVIPATH 業績表現強勁，營收從 2025 年第一季的 4,470 萬歐元成長一倍多，達到 1.03 億歐元。儘管連續遭遇 580 萬歐元的貨幣逆風，但仍實現了這一收入成長。

Sequential growth across global markets remained strong, with continued strong uptake in the US acting as a key growth catalyst. The YORVIPATH US launch and continued performance outside the US are having a substantial impact on our financial profile, and we expect Ascendis to become cash flow positive on a quarterly basis this year. Including EUR4.4 million of revenue from our collaboration partners, total Q2 revenue was EUR158 million.

全球市場持續成長保持強勁，其中美國持續強勁成長成為關鍵的成長催化劑。YORVIPATH 在美國的推出以及在美國以外地區的持續表現對我們的財務狀況產生了重大影響，我們預計 Ascendis 今年的季度現金流將為正值。包括合作夥伴的 440 萬歐元收入，第二季總收入為 1.58 億歐元。

Turning to expenses, R&D costs for the second quarter decreased to EUR72 million compared to EUR83.5 million in the same period last year, primarily driven by lower development costs for growth disorders. SG&A expenses in 2025 increased to EUR107.6 million compared to EUR74.3 million in the same period last year, primarily driven by global commercial expansion. Total Q2 2025 operating expenses were about EUR180 million.

談到費用，第二季的研發成本從去年同期的 8,350 萬歐元下降至 7,200 萬歐元，主要原因是生長障礙的開發成本降低。2025 年銷售、一般及行政費用 (SG&A) 增至 1.076 億歐元，而去年同期為 7,430 萬歐元，主要受全球商業擴張的推動。2025 年第二季總營運費用約為 1.8 億歐元。

Net finance income for 2025 was EUR22 million, driven primarily by noncash items. Net cash financial expenses for 2025 were EUR5.3 million. We ended 2025 with cash and cash equivalents totaling EUR494 million compared to EUR518 million as of March 31. Of the EUR24 million sequential decrease in cash, EUR19 million of that was due to the June 30 cash transition to -- cash translation to euro. So pretty close to overall cash breakeven for the quarter for the company.

2025 年淨財務收入為 2,200 萬歐元，主要來自非現金項目。2025 年淨現金財務支出為 530 萬歐元。截至 2025 年 3 月 31 日，我們的現金及現金等價物總額為 4.94 億歐元，而截至 2025 年 3 月 31 日，我們的現金及現金等價物總額為 5.18 億歐元。在連續 2,400 萬歐元的現金減少中，1,900 萬歐元是由於 6 月 30 日現金轉換為歐元所致。因此，該公司本季的整體現金損益平衡已相當接近。

Turning to the remainder of 2025, we expect continued revenue growth driven by the strength of the global launch of YORVIPATH. For SKYTROFA, for modeling purposes, we continue to believe that sequential revenue growth for 2025 should track growth in prescriptions, offset somewhat by payer mix and normal seasonality. We also expect long-term growth for SKYTROFA to be driven by label expansion, with our recent adult approval expected to only contribute modestly for 2025.

展望 2025 年剩餘時間，我們預計在 YORVIPATH 全球發布的強勁推動下，營收將持續成長。對於 SKYTROFA，出於建模目的，我們仍然認為 2025 年的連續收入成長應追蹤處方的成長，但會受到付款人組合和正常季節性的影響。我們還預計，SKYTROFA 的長期成長將由標籤擴展推動，而我們最近獲得的成人批准預計只會對 2025 年產生適度貢獻。

We also continue to watch the euro-US dollar exchange rate for any potential impact related to reported revenue. For YORVIPATH, our launch is progressing exceptionally well. Globally, we see YORVIPATH as a standard of care for treating hypoparathyroidism, and we believe it has the potential to achieve multiple billions of euros annually in peak sales over time, and our focus is on building long-term leadership.

我們也將持續關注歐元兌美元匯率，以觀察其對報告收入的潛在影響。對於 YORVIPATH，我們的發布進展非常順利。在全球範圍內，我們將 YORVIPATH 視為治療副甲狀腺功能減退症的標準治療方法，我們相信隨著時間的推移，它有可能實現每年數十億歐元的峰值銷售額，而我們的重點是建立長期領導地位。

In the near term, as investors and analysts seek to model YORVIPATH's growth trajectory, I would highlight the following. Outside the US, we currently see continued steady sequential revenue growth. In the US, seven months into launch, we are seeing strong continued demand and continuation of enrollment trends. We are seeing good conversion from enrollment to paid prescriptions with YORVIPATH. As Jan mentioned, the majority of US patients are approved for reimbursement within three months of enrollment.

短期內，當投資人和分析師試圖模擬 YORVIPATH 的成長軌跡時，我想強調以下幾點。在美國以外，我們目前看到收入持續穩定成長。在美國，該計畫推出七個月後，我們看到了持續強勁的需求和持續的入學趨勢。我們看到 YORVIPATH 從註冊到付費處方的轉換率良好。正如 Jan 所提到的，大多數美國患者在入院後三個月內即可獲得報銷批准。

Payer approvals are broad across commercial and government as well as geographies. We expect additional coverage policies and payer agreements to facilitate patient experience, access, and continued long-term uptake. Based on our data so far, we expect persistence to be high because of the benefits to the patient, and we continue to monitor. And, of course, we'll continue to look to help investors understand uptake and reimbursement dynamics as the year progresses.

付款人的批准範圍廣泛，涵蓋商業、政府和地區。我們期望額外的保險政策和付款人協議能夠促進患者體驗、取得和持續的長期吸收。根據我們目前的數據，由於對患者有益，我們預期持久性會很高，我們會繼續監測。當然，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're now ready to take questions.

接線員，現在我們可以回答問題了。

Thank you. At this time we will conduct a question-and-answer session. (Operator Instructions)

謝謝。這次我們將進行問答環節。（操作員指示）

Jessica Fye, JP Morgan.

傑西卡費伊 (Jessica Fye)，摩根大通 (JP Morgan)。

Great. Thanks, guys. Great quarter. You mentioned you're seeing a continuation of enrollment trends. I think the number of unique patients enrolled grew by about 1,350 in 2Q. Is that the rate you mean that you see continuing? Thank you.

偉大的。謝謝大家。很棒的一個季度。您提到您看到入學趨勢持續增長。我認為第二季度登記的獨立患者數量增加了約 1,350 人。您認為這個比率還會持續下去嗎？謝謝。

Hey, Jess. I can start with a few overall views and then perhaps Jay can follow-up. The number we reported was around 1,750 for the end of Q1. And here, at the end of Q2, we reported 3,100. What we also said in our Q1 call was that 200 patients, we will consider some kind of bolus injection because the 200 patients came from our ERP program. So when I take the numbers out from our Q1 number, the 200, it gives me about 1,550. And so in some way, I actually believe that we see a steady state growth in the patient here between Q1 and Q2.

嘿，傑西。我可以從一些總體觀點開始，然後也許傑伊可以跟進。我們報告的第一季末的數字約為 1,750。而在第二季末，我們報告的數字是 3,100。我們在第一季電話會議上也表示，對於這 200 名患者，我們將考慮某種形式的推注，因為這 200 名患者來自我們的 ERP 計畫。因此，當我從我們的第一季數字中減去 200 時，結果約為 1,550。因此，從某種程度上來說，我確實相信我們在第一季和第二季之間看到患者數量的穩定成長。

When I take this consideration to the 200 patients that came from the ERP program, and this is how we basically see the numbers, and this is very much aligned with our comments at the Q1 call, we expected to see a steady state development in the prescription. And first, in the second part of the year, we expect to have an acceleration of the conversion of a patient that has a prescription to be on treatment.

當我考慮到來自 ERP 計劃的 200 名患者時，我們基本上看到了這些數字，這與我們在第一季電話會議上的評論非常一致，我們預計處方將呈現穩定的發展狀態。首先，我們預計在今年下半年，有處方的患者將加速接受治療。

So that is basically what we have seen. And we look forward to seeing Q3 and Q4. We are extremely optimistic about this launch. This is I'm now just talking about the US. We see the same pattern everywhere we're launching, but it's really it's amazing. And we expect to see the same steady state. Sure, that can be a seasonal factor because of the summer vacation, at least we know in France, August is closed and other places happening the same thing.

這基本上就是我們所看到的。我們期待看到第三季和第四季。我們對此次發布非常樂觀。這就是我現在談論的美國。我們在所有推出的產品中都看到了相同的模式，但這確實令人驚訝。我們期望看到同樣的穩定狀態。當然，這可能是由於暑假的季節性因素，至少我們知道在法國，八月是關閉的，其他地方也會發生同樣的事情。

So Jay, for your comment.

傑伊，感謝您的評論。

Thank you, Jan. As mentioned before, we are seeing that stabilization in enrollment. And again, we're still early in the launch, so we will need more time to observe what that steady-state trend will be as we get more months under our belt with the launch. More importantly, just as Jan mentioned earlier, we're focused beyond just the point of enrollment. Right? We're looking across the entire funnel. From enrollments to approval, from approvals to patients on therapy. And we are seeing continued growth, especially as it relates to the conversion of those patients onto therapy, and we're feeling good about what we are seeing.

謝謝你，Jan。如同先前所提到的，我們看到入學人數趨於穩定。再次強調，我們仍處於發布的早期階段，因此隨著發佈時間的推移，我們需要更多時間來觀察穩定狀態趨勢。更重要的是，正如 Jan 之前提到的，我們關注的不僅僅是招生這一點。正確的？我們正在審視整個漏斗。從註冊到批准，從批准到患者接受治療。我們看到了持續的成長，特別是與這些患者轉向治療相關的成長，我們對所看到的情況感到滿意。

Thanks. Jay.

謝謝。傑伊。

Derek Archila, Wells Fargo.

德里克·阿奇拉，富國銀行。

Hey, thanks for taking the question and congrats on the progress here. I just wanted to confirm something. So it sounded like you noted that there's three months, you know, from enrollment to conversion. I guess I just wanted to know like, what are you doing to kind of improve that? And, I guess, how much progress can you make on improving on that three months? Thanks.

嘿，感謝您提出這個問題，並祝賀您的進展。我只是想確認一些事情。所以聽起來您好像注意到從入學到轉換需要三個月的時間。我想我只是想知道，您正在做什麼來改善這種情況？而且，我想，在這三個月裡你能有多大的進步？謝謝。

There's a lot of elements that nothing has really changed compared to what we said on our Q1 call. There is a lot of elements that Jay and the entire integrated commercial team are working with. We can go a little bit more in specific, but in the overall view, sure, the different politics from a different PPM need to be installed. There is a lot of elements that still takes time. And we're also working a lot on the procedure, how we basically can help patients to be sure and physician and back office. So this is happening for our asset program that we are helping the patient.

與我們在第一季電話會議上所說的相比，許多因素實際上並沒有什麼改變。傑伊和整個綜合商業團隊正在研究很多元素。我們可以更具體一些，但從整體來看，當然，需要安裝來自不同 PPM 的不同政策。還有很多因素仍需要時間。我們也在程序上做了很多工作，基本上是如何幫助病人、醫生和後台人員放心。這是我們幫助患者的資產計劃所發生的事情。

So nothing has changed compared to what we said in Q1 where we believe that we will first see in the second part of the year when many of these activities will be implemented. We will see an improvement in the time from a prescription and to a patient is on treatment.

因此，與我們在第一季所說的相比，沒有任何變化，我們相信我們將在今年下半年首先看到其中許多活動的實施。我們將看到從開處方到患者接受治療的時間有所改善。

Jay, you can give a little bit more flavor on some of the initiatives on a high level because we have so many initiatives going on.

傑伊，你可以從高層次進一步闡述一些舉措，因為我們正在進行許多舉措。

[Absolutely]. And thank you for the question. From a time to approval standpoint, as we said, we're seeing the majority within three months. Now divide our efforts probably in a few buckets. The first one is upstream. Right? We're continuing our payer education, whether it's at commercial level or a public level, just on the clinical value proposition of the product. The full expanse of the label, because as you can all appreciate, whenever there is a new specialty drug on market, particularly a rare disease one, it oftentimes takes a bit of time for them to consider either a category or product that they may not have previously had on formulary or plan.

[絕對地]。感謝您的提問。從批准時間的角度來看，正如我們所說，我們將在三個月內看到大多數批准。現在，我們可以將精力分成幾個部分。第一個是上游。正確的？我們正在繼續對付款人進行教育，無論是在商業層面還是公共層面，都只針對產品的臨床價值主張。標籤的完整範圍，因為正如你們所理解的，每當市場上出現一種新的特效藥，特別是罕見疾病藥物時，他們往往需要花一些時間來考慮他們以前可能沒有在處方集或計劃中出現的類別或產品。

We anticipate that to continue to improve, which will of course, have downstream impacts on the speed. More downstream from that, we're also have a very experienced hub. I think we've mentioned this before. We're quite experienced with being in managed care spaces. So some of the bread-and-butter work as it relates to ensuring that providers and patients are continuing to follow-up, fill out their paperwork correctly, to decrease cycle times to ensure that paperwork isn't the reason why maybe something goes back and forth one or two extra times, which will then also increase the cycle.

我們預計這種情況將繼續改善，這當然會對速度產生下游影響。除此之外，我們還有一個非常有經驗的中心。我想我們之前提到過這一點。我們在管理醫療領域擁有豐富的經驗。因此，一些基本的工作是確保醫療服務提供者和患者能夠繼續跟進，正確填寫他們的文書工作，以減少週期時間，確保文書工作不是導致某些事情來回多一兩次的原因，這也會增加週期。

And then lastly, I would say as we continue to have patients within the funnel -- just making sure, again, that our partnership with our specialty pharmacies, so on and so forth, again, streamlining those processes to continue shave off time as we work through this initial launch phase. All that to say, we're incredibly encouraged by the speed in which we're seeing and a reflection of the experience hub that we have. And we continue to look forward to seeing how that will progress.

最後，我想說，隨著我們繼續在通路中吸引患者——再次確保我們與專業藥房的合作關係等等，再次簡化這些流程，以便在我們完成初始啟動階段時繼續縮短時間。總而言之，我們對所看到的速度以及我們所擁有的體驗中心的反映感到非常鼓舞。我們將繼續期待看到事情將如何進展。

Tazeen Ahmad, Bank of America.

美國銀行的塔津·艾哈邁德（Tazeen Ahmad）。

Hey, guys. Thanks for taking my question. I was wondering if you could give us some color on the type of patients. There's been a lot of talk about initially severe patients, the most severe patients, being put on YORVIPATH first. But do you have any color on what the split is between definitionally what a severe patient is versus the type of patient that are getting on that might be on the more moderate side? Thanks.

嘿，大家好。感謝您回答我的問題。我想知道您是否可以向我們介紹患者的類型。關於最初重症患者、最嚴重的患者首先被安排使用 YORVIPATH 的討論已經有很多。但是，您是否能從定義上區分重症患者和病情較輕的患者？謝謝。

Thanks, Tazeen, for the question. First of all, there is no medical definition that defines severity of hypoparathyroidism. So we cannot go in and claim database and say this is a severity that you have in the disease because all of them is not classified related to that. When we talked about the element of being uncontrolled, partly controlled, or something we call controlled, this was mainly related to one single parameter looking on the claim databases and see how often they're sitting and physician.

謝謝 Tazeen 提出的問題。首先，沒有醫學定義可以說明副甲狀腺功能低下症的嚴重程度。因此，我們不能進入資料庫並聲稱這是您所患疾病的嚴重程度，因為並非所有與此相關的資訊都屬於機密。當我們談論不受控制、部分控製或我們稱之為受控制的因素時，這主要與查看索賠資料庫的單一參數有關，看看他們坐在那裡的頻率和醫生的情況。

How often they're sitting in physician, I think, have a lot of multiple aspects that some way are not only reflecting where you're living in the country, what kind of medical access you have, and also how often are there and into that really can see you. So we did it out from this perspective because we wanted to be quite sure we're addressing a physician that see you with a high number of patients in hypoparathyroidism. And it was why we addressed this physician as our priority in our, you can say, commercial strategies.

我認為，醫生看病的頻率有很多方面，這些方面不僅反映了您在該國的居住地、您享有的醫療服務類型，還反映了您看病的頻率和程度。因此，我們從這個角度出發，因為我們希望確保我們面對的是一位接診大量副甲狀腺功能減退症患者的醫生。這就是為什麼我們將這位醫生作為我們商業策略的優先事項。

And so we can certainly not define and answer your question because there is no way this is part of the reimbursement system. It's no part of can see of the patient because it's not a medical term that error is defined. I think to give you also the other aspect on it, when we look on the guidelines that we see being integrated many different places, they are not using this kind of term either. So the guidelines just having a broad aspect on all the different elements that really qualify to be on PTH treatment. And in general, all the guidelines we have seen being issued from all different places in the world now, they are indicating that 95% of all patient bases should be on PTH treatment. And I think that is pretty logical.

因此，我們當然無法定義和回答您的問題，因為這不可能是報銷系統的一部分。它不是病人可以看到的部分，因為錯誤不是一個醫學術語。我想給你介紹另一個方面，當我們查看許多不同地方整合的指導方針時，他們也沒有使用這種術語。因此，該指南只是對真正符合 PTH 治療條件的所有不同要素進行了廣泛的概述。總體而言，我們現在看到世界各地發布的所有指南都表明，95% 的患者應該接受 PTH 治療。我認為這是非常合乎邏輯的。

Think about how many patients on type 1 diabetes. Will you ever consider that you will not take all patients that have type 1 diabetes on insulin treatment? And I think you will see the same thing happening with hypoparathyroidism.

想想有多少第 1 型糖尿病患者。您是否考慮過不會對所有第 1 型糖尿病患者都進行胰島素治療？我認為你會看到副甲狀腺功能減退症也發生同樣的事情。

Yaron Werber, TD Cowen.

Yaron Werber，TD Cowen。

Great. Thanks for my question and congrats again. A couple of interrelated questions. Can you give us a little bit of a sense, Scott, you mentioned last quarter to expect Europe to grow about EUR4 million to EUR5 million, so that puts you at around EUR24 million. So it almost seems like you did sort of EUR79 million to EUR80 million in the US. Am I sort of in the ballpark?

偉大的。感謝我的提問並再次祝賀。幾個相互關聯的問題。斯科特，您能否給我們稍微介紹一下，您上個季度提到預計歐洲的銷售額將增長約 400 萬歐元至 500 萬歐元，因此您的銷售額將達到約 2400 萬歐元。因此看起來你在美國賺了 7900 萬到 8000 萬歐元。我猜對了嗎？

And I guess, secondly, it's when one looks at we shouldn't be expecting that you're going to be growing patients 1,500 quarter over quarter. So can you give us a little bit of a sense how to think about what a sequential normalized growth at this point in the US can be so we don't get out of hand? Thank you.

其次，我想，我們不應該期望每季的患者數量會增加 1,500 人。那麼，您能否給我們稍微介紹一下，如何看待美國目前的連續正常化成長，以免失控？謝謝。

I can think I can help Scott this time, which I often do. Because what we said in our Q1 call, and if you look on Q3 to Q4, revenue increase net revenue in euro was about EUR4 million to EUR5 million. And in this two quarters, it was basically your euro or ex-US revenue. And we also say that time that we expect that to continue in 2025 when we see more countries coming on full commercialization as we just got Spain now, and we expect a few countries more. Perhaps it will increase, but it will first have a material impact two to three months after basic mediation or full commercialization.

我認為這次我可以幫助斯科特，我經常這樣做。因為我們在第一季電話會議上說過，如果你看第三季到第四季度，歐元淨收入成長約 400 萬歐元到 500 萬歐元。這兩個季度，基本上都是歐元或美國以外的收入。我們也表示，我們預計這種情況將在 2025 年持續下去，屆時我們將看到更多國家實現全面商業化，就像現在的西班牙一樣，我們預計還會有更多國家商業化。也許會增加，但它會在基本調解或全面商業化兩到三個月後才產生實質影響。

So therefore, I will say your assumption is pretty correct and also reflecting what we said in Q1 this way. Related to the question you comment, it's some way forward-looking statement. I know it's really being covered by Scott's fast reading, but from my perspective is that we see a strong, strong launch here in the US. We have seen nearly the same numbers between Q1 and Q2. And we're really looking forward, as Jay said correctly, this is an early in the launch. It will be too early for us to come up with any kind of prediction how we really will see the next six, seven quarters to go.

因此，我會說你的假設非常正確，也反映了我們在第一季所說的內容。與您評論的問題相關，這是某種前瞻性的陳述。我知道斯科特的快速閱讀確實涵蓋了這一點，但從我的角度來看，我們在美國看到了強勁的推出。我們看到第一季和第二季的數字幾乎相同。我們真的很期待，正如傑伊所說，這只是發布的早期階段。現在對於我們未來六、七個季度的前景做出任何預測還為時過早。

Gavin Clark-Gardner, Evercore ISI.

加文·克拉克·加德納 (Gavin Clark-Gardner)，Evercore ISI。

Hey, guys. Congrats on another great quarter. First, what do you believe the ultimate conversion rate from the enrollment forms to paid drugs will be at any point in time? And then secondly, looking ahead, do you plan to keep reporting the enrollment forms for YORVIPATH? Thank you.

嘿，大家好。恭喜您又一個出色的季度。首先，您認為在某個時間點，從登記表到付費藥品的最終轉換率是多少？其次，展望未來，您是否計劃繼續報告 YORVIPATH 的入學表格？謝謝。

It's really difficult for us to give you a clear number. But what we always will see in the US, there will be a percentage of patients that have really difficulties to get reimbursed even if we try and help them multiple times. And what we see during the launch, what we have seen before, is that what I call the tail is getting faster and faster cleared out. And I can guarantee we will do everything in Ascendis manner to help that all the patients can come on treatment. Can we guarantee that everyone can go on treatment? No.

我們確實很難給你一個明確的數字。但我們在美國經常看到的情況是，即使我們多次嘗試幫助他們，仍有一定比例的患者難以獲得報銷。我們在發射過程中看到的，也是我們之前看到的，就是我所說的尾部正在越來越快地清除。我可以保證，我們將盡一切努力以 Ascendis 的方式幫助所有患者接受治療。我們能保證每個人都能接受治療嗎？不。

There will be a number of patients even after 6, 9, 12 months, really struggling we will say it is really hard to be covered. So you can ask about my personal success. My personal success will be if we get in steady-state launch really with a mature product, could get around 90% of all patients on treatment. I will feel it as a personal success and my contribution to help with hypoparathyroidism.

即使在 6、9、12 個月之後，仍會有許多患者生活十分艱難，我們會說獲得保障確實非常困難。所以你可以問我的個人成功。我個人的成功在於，如果我們能夠真正透過成熟的產品來穩定上市，大約 90% 的患者能夠接受治療。我會將其視為個人的成功以及我為幫助治療副甲狀腺功能減退症所做的貢獻。

Jay, you can also come with your personal success number. It's you want to do that.

傑伊，你也可以帶上你的個人成功號碼。你想這麼做。

Appreciate the question. You know, I would say layer on a couple things, right? I think the enrollment to approval, again, it's not just driven by payers. Of course, that's a component of it, right? Because you know, as we discussed before, there are certain plans and policies for which you know, you have to go through exception or appeals process. And I think, as Jan alluded to, that right tail will take some time to clear, depending on the plan and as things evolve.

感謝你的提問。你知道，我會說分層添加一些東西，對吧？我認為從註冊到批准，不僅僅是由付款人推動的。當然，這是其中的一部分，對吧？因為你知道，正如我們之前討論過的，有些計劃和政策你必須經過例外或上訴程序。我認為，正如 Jan 所提到的那樣，正確的方向需要一些時間來解決，這取決於計劃和事態的發展。

The other component from enrollment to approvals is entirely unrelated to payers, and it may just be more driven by ensuring, again, providers are leveraging the paperwork appropriately, patients are following up with outreach. A lot of that which we'll continue to pursue across the spectrum because we know, again, that these patients can and should benefit from the product if they're already in the funnel, and we will do everything we can to clear that long tail out knowing that it will take some time.

從註冊到批准的其他部分與付款人完全無關，它可能只是更多地確保提供者適當地利用文書工作，患者跟進外展。我們將繼續在各個領域開展這項工作，因為我們再次知道，如果這些患者已經進入漏斗，他們就可以而且應該從產品中受益，我們將盡一切努力清除長尾問題，儘管這需要一些時間。

And when you look at a lot of rare disease analogs in these types of spaces, it can take some time to get there. But this is a long haul and we're looking at it more from that lens to making sure we're optimizing every step of the way.

當你觀察這些類型的空間中的許多罕見疾病類似物時，可能需要一些時間才能到達那裡。但這是一個長期的過程，我們會從這個角度來看待它，以確保我們優化每一步。

And are you guys planning to report enrollment forms for YORVIPATH in the next quarter also?

你們還計劃在下個季度報告 YORVIPATH 的入學表格嗎？

I think we will give you the necessary KPIs that we're doing today and we will continue to do that in every quarter until we feel we're coming to a steady state where we feel that there is enough information just out from revenue that you can basically do your modeling. Until that, we will continue to provide the necessary data that support that you can make a sudden modeling of the launches.

我認為我們會為您提供我們今天正在做的必要 KPI，並且我們會在每個季度繼續這樣做，直到我們感覺我們達到了穩定狀態，我們覺得僅從收入中就有足夠的信息，您基本上可以進行建模。在此之前，我們將繼續提供必要的數據，支援您對發射進行突然建模。

Just to say and repeat it me again, this is an amazing launch. Q1 was great. Q2 was also amazingly great, and we have not seen any weakness in the launch.

我再說一遍，這是一次令人驚嘆的發表會。Q1 很棒。Q2 也表現得非常出色，我們在發布過程中沒有發現任何弱點。

Li Watsek, Cantor.

李·瓦特塞克，領唱。

Hi. Congratulations on the quarter. This is [Daniel Bruner] on for Li Watsek. We're just curious about the pull-through of the patients that get onto YORVIPATH. How should we think about compliance especially if you're saying that 1,500 PFS number is net patients going forward? Thank you.

你好。恭喜本季。這是 [Daniel Bruner] 為 Li Watsek 報道的。我們只是對進入 YORVIPATH 的患者的治療情況感到好奇。我們應該如何看待依從性，尤其是如果您說 1,500 個 PFS 數量是未來的淨患者數量？謝謝。

Where we have the best long-term data is from Europe where we basically started their launch about 6 to 9 months before. And when we look on a rate, what we call true discontinuation, is extremely low, a few percenters. So we really see the benefit of the therapy. People taking the therapy are taking it. They're keeping doing it. And I believe that is the contribution on how we are addressing a major unmet medical need with the treatment of YORVIPATH.

我們擁有最好的長期數據來自歐洲，我們基本上大約在 6 到 9 個月前就開始在那裡發布產品。當我們觀察比率時，我們所說的真正停藥率非常低，只有百分之幾。所以我們確實看到了這種療法的好處。接受治療的人正在接受治療。他們一直在做這件事。我相信這就是我們如何透過 YORVIPATH 治療解決重大未滿足醫療需求的貢獻。

So everything what we see here is far away from what you see with a diabetes drug. People stay on it even much better on than insulin that you see in type 1 diabetes. And I think this is the main contribution to the positive CNS effect that is with this product.

因此，我們在這裡看到的一切與您在糖尿病藥物中看到的一切相差甚遠。人們服用它的效果甚至比第 1 型糖尿病患者服用胰島素的效果更好。我認為這是該產品對中樞神經系統產生正面影響的主要因素。

Okay. Cool. Thank you. And just on the just going back to Yaron's question earlier about the 1,500 patient net enrollment per quarter. Just for me to fully understand, this is the patient start forms that you're referring to?

好的。涼爽的。謝謝。回到 Yaron 之前提出的問題，關於每季度 1,500 名患者的淨入院人數。只是為了讓我完全理解，這是您所指的患者開始表格嗎？

Yeah. That is what we're referring to is unique prescriptions. Meaning is that it's a new patient that has got a prescription. Is what we call unique prescriptions.

是的。這就是我們所說的獨特處方。意思是說，這是一位拿到處方的新病人。就是我們所說的獨特藥方。

Great. Thank you so much, and congrats again.

偉大的。非常感謝，再次恭喜。

Thanks.

謝謝。

Joseph Schwartz, Leerlink Partners.

約瑟夫‧施瓦茨，Leerlink Partners。

Yeah. Hi. I'm Joori Park dialing in for Joe. Thank you for taking our questions. The first one is on YORVIPATH. I believe that there were 1,500 prescribing health care providers in the US by the end of the quarter. Can you help us understand how much of your target physician base this represents?

是的。你好。我是喬裡‧帕克 (Joori Park)。感謝您回答我們的問題。第一個是在 YORVIPATH 上。我相信到本季末美國將有 1,500 家處方醫療保健提供者。您能幫助我們了解這代表了多少您的目標醫師群嗎？

And secondly, on CNP, a competitor recently announced that their long-acting CNP's area under the curve PK level was three times greater than the levels of TransCon CNP. Based on your experience with TransCon CNP, how could that translate in the clinic in your view? And how does that profile differ from your combination approach? Thank you.

其次，關於 CNP，一家競爭對手最近宣布，他們的長效 CNP 的曲線下面積 PK 水準是 TransCon CNP 水準的三倍。根據您在 TransCon CNP 方面的經驗，您認為這在診所中該如何應用？該設定檔與您的組合方法有何不同？謝謝。

Yeah. Let me start with the easy one or potential. Somebody take that over to Jay and I will take them more. I can say scientific interesting question as number two.

是的。讓我從簡單或潛在的開始。有人把它交給傑伊，我會帶更多。我可以說科學有趣的問題是第二個問題。

So happy to start with the first question. From a target-list standpoint, we're talking about eight to 10,000, so that you can consider that as our universe, about 3,000 of which we decile as high medium. And we're seeing pretty good field execution metrics across that, so over 80% reach across our high-, medium-priority targets.

很高興開始回答第一個問題。從目標清單的角度來看，我們討論的是 8 到 10,000 個，因此您可以將其視為我們的範圍，其中約 3,000 個我們劃分為高中等。我們看到現場執行指標相當不錯，超過 80% 達到了我們的高優先級和中優先目標。

So the second question is interesting because, for many, many years, there was a lot of skepticism about our sustained profile. And then suddenly, there was a big change where some may say there is some benefit by having a sustained profile that does not give a high Cmax that basically can indicate risk of hypertension. And, also, you need to have continuous exposure over one week. And so we started and designed our TransCon CNP in 2015. And now we are in 2025. They're starting now to go after that concept.

所以第二個問題很有趣，因為多年來，人們對我們持續的形像一直持懷疑態度。然後突然發生了巨大的變化，有些人可能會說，持續保持不產生高 Cmax 的曲線會帶來一些好處，而高 Cmax 基本上可以表明存在高血壓的風險。而且，您還需要連續接觸一週以上。因此，我們在 2015 年開始設計 TransCon CNP。現在已經是2025年了。他們現在開始追求這個概念。

From my perspective is that when you look on how we designed it, that also all our associated pattern filing we have with it or the IP we have of the optimal prodrome the medical treatment benefit it is, and other things like that, people are now trying to copy with other concepts. And when I think about the concept I think there was some kind of making success of a product without disclosing the key element.

從我的角度來看，當你觀察我們如何設計它時，我們擁有的所有相關模式文件或我們擁有的最佳前驅症狀的智慧財產權、它的醫療益處以及其他類似的東西，人們現在正試圖用其他概念來複製。當我思考這個概念時，我認為在沒有揭露關鍵要素的情況下，產品取得了某種成功。

The key element that was AOC I really don't care about AOC. I want to know key element. What is really the half-life? How is this exposure really happening? Is it something that picks up to a very, very, very high value there shortly? And then you basically are going up to, as I call it, the danger zone of hypertension. That is not really an optimal product in this way.

關鍵元素是 AOC，我真的不關心 AOC。我想知道關鍵要素。半衰期到底是多少？這次曝光究竟是如何發生的？它的價值是否會在短時間內上升到非常非常高？然後你基本上就進入我所說的高血壓危險區了。從這個角度來看，這其實並不是最佳產品。

I believe what we saw in all our clinical data, it's really hard to do a lot when you remove a brake because it's really the hill is rolling down that really decides the speed. If you have taken the brake off, the brake is off. That is where I really don't get the biological and scientific concept. That is related to linear growth.

我相信，根據我們在所有臨床數據中看到的情況，當你鬆開煞車時，很難做很多事情，因為真正決定速度的是滾落的山坡。如果您鬆開了煞車，則煞車已關閉。這就是我真正不懂的生物學和科學概念。這與線性增長有關。

When we look at some of the other effects where we see muscle strengthening and other things like that, quite sure having continuous exposure, but we do not know exactly if that is maxed out or not. So out from that perspective is that downfall. Can you get more out on having a higher AOC?

當我們觀察其他一些效果時，我們看到了肌肉強化和其他類似的東西，很確定這是持續暴露，但我們不知道這是否達到了最大值。所以從這個角度來看，這就是失敗。擁有更高的 AOC 能帶來更多效益嗎？

First of all, it needs to have the right AOC. This must be a higher exposure. Completely over the once-weekly profile. And no one disclosed that because then they need to have a longer half-life than ours that is about two and a half to three days. And I have not got any kind of disclosure than they are there.

首先，它需要有正確的AOC。這肯定是更高的曝光率。徹底結束了每週一次的簡介。沒有人透露這一點，因為它們需要比我們的半衰期更長，大約兩天半到三天。但我還沒有得到任何有關他們在那裡的披露。

When I go out to our combination therapy, it's basically a completely different concept because the concept of that is between synergies, between different biological pathways, which are well known from so many other therapeutic areas to have the optimal treatment you cannot over-compensate just by one pathway. But you're basically providing the benefit in a holistic manner in a much more normal manner by balancing different pathways. And that is what we do in the combination team trying to come to CNP, and the growth hormone effect, which are basic, at the same time.

當我談到聯合療法時，它基本上是一個完全不同的概念，因為它的概念是協同作用之間、不同生物途徑之間的協同作用，而這些協同作用在許多其他治療領域都是眾所周知的，因為最佳治療不能僅通過一種途徑進行過度補償。但你基本上是透過平衡不同的途徑，以更正常的方式，以整體的方式提供福利。這就是我們聯合團隊所做的工作，試圖同時達到 CNP 和生長激素的效果，它們是基礎。

In a more simplified manner, remove the brake, with what we do with the CNP, and then having a speed up on that. So I feel really, really confident with our code state our combination therapy. That is really is a unique way where you really can totally provide for patients, for physicians, a complete new treatment standard.

以更簡化的方式，移除煞車，就像我們對 CNP 所做的那樣，然後加速。因此，我對我們的代碼陳述我們的聯合療法感到非常非常有信心。這確實是一種獨特的方式，您可以為患者、醫生提供全新的治療標準。

Eliana Merle, UBS.

瑞銀的 Eliana Merle。

Hey, guys. Thanks for taking my question, and congrats on the strong quarter. Curious for achondroplasia, what's your base case for the indication statement for TransCon CNP, whether it'd be for the treatment of achondroplasia or for the increase in linear growth in achondroplasia, like VOXZOGO has? And I guess any expectations for differentiation in the label relative to VOXZOGO, such as in terms of the indication statement or, say, other secondary endpoints?

嘿，大家好。感謝您回答我的問題，並祝賀本季業績強勁。我對軟骨發育不全感到好奇，您對 TransCon CNP 適應症聲明的基本情況是什麼，是用於治療軟骨發育不全還是像 VOXZOGO 一樣用於增加軟骨發育不全的線性生長？我猜想對於標籤相對於 VOXZOGO 的差異化有什麼期望，例如在適應症說明或其他次要終點方面？

And then second, a follow-up question. Just what's your perspective on the IP landscape for weekly CNPs? Specifically any thoughts on [CMM333] and where that might stand relative to the TransCon CNP IP estate? Thanks.

第二，一個後續問題。您對每週 CNP 的 IP 前景有何看法？具體來說，您對 [CMM333] 有何看法？它相對於 TransCon CNP IP 資產而言處於什麼位置？謝謝。

We are progressing to the regulatory review with our TransCon CNP exactly as we hope for in an accelerated priority review. Everything is happening on the right time. So labeling discussion is one of the last parts in the review cycle. So it's really, really difficult for me to come up with any kind of elements. What are [Morwell] referring to You Is the data we have that is really backing up TransCon CNP. And I believe this is why we got the priority review. Because we have data that give a strong evidence that we can provide treatment benefit beyond linear growth.

我們正在對 TransCon CNP 進行監管審查，正如我們所希望的那樣，能夠進行加速優先審查。一切都恰逢其時。因此，標籤討論是審查週期的最後部分之一。因此，對我來說，想出任何一種元素都是非常非常困難的。[Morwell] 所指的「您」是我們擁有的真正支持 TransCon CNP 的數據。我相信這就是我們獲得優先審查的原因。因為我們有數據提供強有力的證據，證明我們可以提供超越線性成長的治療效益。

And now we're talking about everything what we have seen related to leg bowing, everything what we have seen to muscle strength, because people saying that they have also seen it. But you're not seeing it in a real manner. You need to see it in a placebo-controlled manner because either body proportionality is actually improving during a normal development of a child also to an achondroplasia child. So how can you discriminate? Is it really the treatment benefit or just a normal development? And this is why it's so extremely important to be in a position that you are referring to data that's done in a placebo-controlled manner in a pivotal trial.

現在我們正在談論我們所看到的所有與腿部彎曲有關的事情，我們所看到的所有與肌肉力量有關的事情，因為人們說他們也看到了它。但你並沒有看到真實的情況。您需要以安慰劑對照的方式觀察它，因為無論是正常發育的兒童還是軟骨發育不全的兒童，身體比例實際上都在改善。那你怎麼能歧視呢？這真的是治療益處還是只是正常發展？這就是為什麼參考在關鍵試驗中以安慰劑對照方式獲得的數據如此重要。

I'm not referring to the other benefit we have. No risk of hypertension. Low injection site. This is why the and sure, obvious. Patient, parents, parents, parents, parents really love their once-weekly profile. It's such a little burden for them to give it in. So I think what I see here I'm not so much really concerned about exactly what is coming into the labeling. I'm more interested in the benefit that we can go out and explain that TransCon CNP is providing, which I basically have never seen in any other well-controlled pivotal trial. I think that is the key element for me. And I think this is what we see everywhere.

我指的不是我們擁有的其他好處。無高血壓風險。注射部位較低。這就是為什麼，當然，顯而易見。病人、父母、父母、父母、父母真的很喜歡他們每週一次的簡介。對他們來說，放棄這一點只是很小的負擔。所以我認為我在這裡看到的並不是那麼真正關心標籤到底是什麼。我更感興趣的是我們可以解釋 TransCon CNP 所提供的好處，這基本上是我在任何其他控制良好的關鍵試驗中從未見過的。我認為這對我來說是關鍵因素。我認為這就是我們隨處可見的現象。

And the second question was about the IP. I believe when we developed that in 2015, we basically filed a lot of IP. There was how to make an optimal prodrug. There was the benefit of having a prodrug that gave sustained things. As there is no clear for me exactly what is the primary structure that they have in the CMM333, it's impossible for me to say exactly what they are. But if you can see me, at least you can see I have a great smile on my lip -- face because we are pretty good in what we're doing when we file IP.

第二個問題是關於IP的。我相信當我們在 2015 年開發這個產品時，我們基本上提交了大量的智慧財產權。如何製造出最佳的前驅藥物。擁有一種能夠持續產生效果的前驅藥物是有好處的。由於我不清楚 CMM333 中它們的主要結構到底是什麼，所以我無法確切地說出它們是什麼。但如果你能看到我，至少你可以看到我嘴角掛著燦爛的笑容——因為我們在申請智慧財產權時做得相當出色。

Understood. Thank you.

明白了。謝謝。

Kelly Shi, Jefferies.

Kelly Shi，傑富瑞集團。

Congrats on another strong quarter, and thank you for taking my questions. For YORVIPATH, what is the typical type titration period that you are seeing right now across the broader patient spectrum in the real world? And once the patient completed titration period, should we expect a higher monthly cost?

恭喜您又一個強勁的季度，感謝您回答我的問題。對於 YORVIPATH，您目前在現實世界中更廣泛的患者群體中看到的典型滴定期是什麼？一旦患者完成滴定期，我們是否應該預期每月費用會更高？

So at least I got the first question related to the titration period. And I think Aimee, our Chief Medical Officer, knows a lot about what we have seen in our clinical trial. I think we are much more uncertain about what is exactly happening in what we call in real-life clinical elements.

所以至少我得到了與滴定期相關的第一個問題。我認為我們的首席醫療官艾米對我們在臨床試驗中看到的情況非常了解。我認為，我們對現實生活中臨床元素中到底發生了什麼事更加不確定。

But what the key element for me, and I take it from that perspective, do we see a lot of patients on set extraction at that stage? Do we see a lot of patient drop out because there's a problem with it? And we don't see that. So I cannot really comment about what exactly is happening in the titration for the patient in the real world, but at least we can see that it's happening very successfully.

但對我來說，關鍵因素是什麼？從這個角度來看，我們是否在那個階段看到很多病人在現場拔牙？我們是否看到許多患者因為有問題而退出治療？但我們並沒有看到這一點。因此，我無法真正評論現實世界中患者的滴定過程究竟發生了什麼，但至少我們可以看到它進展非常成功。

And your second question was?

你的第二個問題是？

So once the patient completes titration, should we expect a higher monthly cost? Because they stay on a higher dose, right?

那麼，一旦患者完成滴定，我們是否應該預期每月費用會更高？因為他們服用的劑量較高，對嗎？

I think in the US, we have an approval up to from 6 to 30 micrograms. So, basically, we are in a position that we're only using one pen at a time. Outside US, there is a possibility to use up to sixteen micrograms. We have just initiated what we call our 60-microgram trial, which will facilitate this is our aim. This is a 26-week trial to facilitate that we can get on labeling that we can use up to sixty micrograms in the US.

我認為在美國，我們批准的濃度是 6 至 30 微克。所以，基本上，我們一次只使用一支筆。在美國以外，有可能使用高達十六微克。我們剛剛啟動了所謂的 60 微克試驗，這將有助於實現我們的目標。這是一項為期 26 週的試驗，目的是確保我們可以在美國獲得最多可使用 60 微克的標籤。

And I think when Jay has seen all the data and becoming near commercial, what I call commercial launch of up to the 60 micrograms, there will be a discussion from Jay's side exactly how we are handling the reimbursement for that situation. I think it's too early for us to comment on that.

我認為，當傑伊看到所有數據並接近商業化時，也就是我所說的高達 60 微克的商業化發佈時，傑伊方面將會討論我們究竟如何處理這種情況的報銷問題。我認為現在評論這個還為時過早。

Paul Choi, Goldman Sachs.

高盛的保羅·崔（Paul Choi）。

Hi, good afternoon, and congratulations on the strong quarter results. Jan, just to follow-up on your last comment about potentially harmonizing the US label with the EU label and the 60-microgram dose, when might you be in a position to submit that data to the FDA? And then, commercially, what portion of the patient population would that potentially allow you to address as not being currently suited by the available presentations in the US market? Thank you for taking our questions.

大家下午好，恭喜您本季的強勁業績。Jan，為了跟進您上次關於協調美國標籤與歐盟標籤和 60 微克劑量的評論，您什麼時候可以向 FDA 提交該數據？那麼，從商業角度來看，有多少比例的患者群體可能因目前美國市場上現有的產品而不適合治療？感謝您回答我們的問題。

Thanks a lot for the question. I actually think this is a lot of questions in the question because what is happening in the US today -- and I think there are a lot of different places where different elements on how to solve the issue if a patient needs more than 30 micrograms. There are some patients because of the label restriction to 30 micrograms, that will stay in a position that they're taking 30 micrograms. And if needed, that will potentially take additional calcium supplements or additional active vitamin D.

非常感謝您的提問。我實際上認為這個問題中有很多問題，因為今天美國正在發生的事情——我認為如果患者需要超過 30 微克，很多不同的地方對於如何解決這個問題有不同的看法。有些患者由於標籤限制為 30 微克，因此將保持服用 30 微克的狀態。如果需要的話，可能需要補充額外的鈣或活性維生素 D。

There are other places where the basic the patient on an physician off-label will potentially get access to a higher dose, which we obviously have no involvement in and no recommendation. So from my perspective is that there is a need for a dose higher than 30 and we would do everything we can do to get it as fast as possible out to the patients.

在其他地方，醫生為患者開立的超說明書用藥可能會使其獲得更高的劑量，而我們顯然沒有參與其中，也沒有提出任何建議。因此，從我的角度來看，需要高於 30 的劑量，我們會盡一切努力盡快提供給患者。

We're starting the trial now. It's a small trial. It's less than 20 patients. We are targeting enrollment of 18. It's only twenty-six weeks. And we are utilizing the same pen devices that the basic have been already in the market. So there is no CMC component in this way. It's just for Aimee Shu and her team to basically get the clinical trial done and get through the regulatory team to get it filed and approved. And we will do that as fast as possible. But it looks pretty, pretty promising to get it in.

我們現在開始審判。這是一次小嘗試。病人不到20人。我們的目標是招收 18 名學生。才二十六週。我們使用的筆設備與市場上已有的基本筆設備相同。因此這種方式下不存在CMC成分。Aimee Shu 和她的團隊只需完成臨床試驗並透過監管團隊提交申請並獲得批准。我們將盡快完成此事。但它看起來非常有希望實現。

Alex Thompson, Stifel.

亞歷克斯湯普森，Stifel。

Great. Thanks for taking my questions. I guess on YORVIPATH as well, you've talked about the breadth of prescribers. I wonder if you could comment on the proportion of prescribers that you've seen with multiple prescriptions, multiple patients on therapy, and how you see that trend changing over time? Thanks.

偉大的。感謝您回答我的問題。我想在 YORVIPATH 上您也談到了處方者的廣度。我想知道您是否可以評論一下您所見過的開立多張處方、多名患者接受治療的醫生的比例，以及您認為這種趨勢如何隨時間變化？謝謝。

We cannot really address that question. We don't have sufficient data that we really will feel confident to come with data that really support and strong trend analysis currently.

我們無法真正回答這個問題。我們沒有足夠的數據，因此我們真的沒有信心得出真正支持和強有力的趨勢分析的數據。

Luca Issi, RBC.

盧卡·伊西，RBC。

Great. Thanks so much for taking my question. Congrats on the quarter. Maybe, Scott, lots of questions, obviously, the top line, rightly so. But how about SG&A? I mean, up 44% year over year and 6% quarter over quarter. Appreciate, obviously, you're launching a drug, but how should we think about modeling SG&A for the rest of the year? I guess what I'm trying to ask here is how should we think about potential to achieve profitability in Q3 versus Q4?

偉大的。非常感謝您回答我的問題。恭喜本季取得佳績。也許，斯科特，有很多問題，顯然，最重要的是，這是正確的。但是銷售、一般及行政費用 (SG&A) 呢？我的意思是，同比增長 44%，環比增長 6%。很顯然，您正在推出一種藥物，但我們應該如何考慮為今年剩餘時間建模銷售、一般及行政費用 (SG&A)？我想我在這裡想問的是，我們應該如何看待第三季和第四季實現獲利的潛力？

Then maybe secondarily, can you just remind us about Ascendis exposures around tariffs and MFN? Again, appreciate the situation is still fluid, and we don't have all the details. But any high-level commentary, much appreciated. Thanks again.

那麼其次，您能否提醒我們 Ascendis 在關稅和最惠國待遇方面的風險？再次強調，情況仍然不穩定，我們還沒有掌握全部細節。但對於任何高水準的評論，我們都非常感激。再次感謝。

Okay. Great. On SG&A and expenses overall, so remember last quarter, we did EUR190 million of OpEx, and we said that wasn't a bad run rate, maybe plus or minus each quarter. So this quarter, we're, you know, minus about EUR10 million from that. So we're about a EUR180 million of OpEx this quarter. And as you point out, you know, it's probably not best to look year over year because we had a lot of growth in the last year. It's really the sequential build. So I would look at the 6% sequential growth for SG&A as, you know, potentially not a bad number to think about. But in the overall context of, you know, I would still say about a EUR190 million OpEx per quarter is not a bad number to think about.

好的。偉大的。關於銷售、一般及行政費用和總體開支，記得上個季度，我們的營運支出為 1.9 億歐元，我們說這不是一個糟糕的運行率，可能每個季度都會有增減。因此，本季我們的虧損約為 1000 萬歐元。因此，本季我們的營運支出約為 1.8 億歐元。正如您所指出的，逐年查看數據可能不是最好的選擇，因為去年我們已經取得了巨大的成長。這實際上是順序構建。因此，我認為銷售、一般和行政費用連續 6% 的成長可能不是一個壞數字。但從總體來看，我仍然認為每季約 1.9 億歐元的營運支出並不是一個糟糕的數字。

With respect to profitability, yes, we expect that this year. I mean, if you look at our financials and back out, June 30 currency, thanks to our whatever it was called, Liberation Day, that cost about EUR20 of cash delta. So overall, everything across the company, it was about EUR5 of burn in Q2. And, actually, on an operating basis, we're just slightly positive on cash. So that should be relatively doable this year.

關於獲利能力，是的，我們預計今年將會獲利。我的意思是，如果你看看我們的財務狀況，再回顧一下，6 月 30 日的貨幣，感謝我們所謂的解放日，這花費了大約 20 歐元的現金增量。因此，總體而言，整個公司第二季的虧損約為 5 歐元。實際上，從營運角度來看，我們對現金略有樂觀。所以今年應該相對可行。

With respect to MFN and tariff, I think you said it pretty well. There's really too much in development right now to make any comments specifically on it. But you know, we do believe that we're as a flexible company, we're pretty well positioned to mitigate the impact of any policy should it emerge.

關於最惠國待遇和關稅，我認為你說得很好。目前開發的內容實在太多，無法對此做出任何具體的評論。但你知道，我們確實相信，作為一家靈活的公司，我們完全有能力減輕任何政策帶來的影響。

Just to clarify and also add in Scott's comments, we're not importing finished products to the US. We import them in different states and finalize them inside the US. So in whatever way we look at it, we cannot see how it really should provide a major material impact on our business and how we operate.

只是為了澄清並補充斯科特的評論，我們不會將成品進口到美國。我們從不同的州進口它們，並在美國境內完成它們。因此，無論我們如何看待它，我們都無法看出它實際上應該對我們的業務和營運方式產生重大的實質影響。

Leland Gershell, Oppenheimer.

利蘭·格謝爾，奧本海默。

Hey. Good afternoon, and thanks for taking our questions. Yeah, I'm just curious, you know, it's in the past, you had not expressed much interest in hypochondroplasia as a development program, and maybe you had started to lean a bit toward that earlier this year, and now we're seeing a formal announcement of intention to go in that direction. So I'm just wondering, may have changed that affected your decision process here, and if you could also maybe just share briefly what you think is the opportunity for Ascendis in hypochondroplasia. Thank you.

嘿。下午好，感謝您回答我們的問題。是的，我只是好奇，你知道，過去你並沒有對軟骨發育不良作為一項發展計劃表現出太大的興趣，也許你在今年早些時候就開始有點傾向於此，而現在我們看到了正式宣布有意朝這個方向發展的聲明。所以我只是想知道，可能已經發生了一些變化，影響了您的決策過程，您是否可以簡單分享一下您認為 Ascendis 在軟骨發育不良方面的機會是什麼。謝謝。

Yeah. Hypochondroplasia is what I will call and perhaps it's the wrong way to define it, but I call it a milder form of achondroplasia. If they don't have many of them per se, what we call body disproportionality, but you can say some in old days, many of these patients basically come in the ISS neuropathic short status. And now, because of much more influenza genetic testing, you basically have a development large group that more is well defined from genetic testing to be hypochondroplasia. That's one point. That basically is saying, patients that were in one group are now being moved over in a different group and meaning they are moved from ISS over to hypochondroplasia. One thing.

是的。我將其稱為軟骨發育不全症，也許這樣定義它不太正確，但我稱之為軟骨發育不全症的一種較輕微的形式。如果他們本身沒有很多這樣的情況，我們稱之為身體不成比例，但你可以說在過去有一些，這些病人中的許多人基本上都處於 ISS 神經性矮小狀態。現在，由於流感基因檢測的增多，基本上可以發現一個較大的發展群體，透過基因檢測可以明確診斷為軟骨發育不良。這是一點。這基本上就是說，原來屬於某一組的患者現在被轉移到另一組，這意味著他們從 ISS 轉移到軟骨發育不良。有一件事。

The second thing, that is that when I saw the element of combination therapy the TransCon CNP and TransCon growth hormone. Then even if you have a very much heterogenic patient population. Then by having a combination product, you basically can ensure all of them will do extremely well. So you can say that is what really is bringing up my attention why I believe we need to do it. And also because I went out and talked with a lot of patients. And I had to think I like to talk with the patient and the patient organization because talking with them, I get much more idea about the unmet medical need we need to address.

第二件事，那就是當我看到聯合治療元素 TransCon CNP 和 TransCon 生長激素時。即使你的患者群體具有很大的異質性。然後透過組合產品，您基本上可以確保它們都能發揮出極佳的效果。所以你可以說這才是真正引起我注意的原因，我認為我們需要這樣做。也因為我出去和很多病人交談過。我必須認為我喜歡與患者和患者組織交談，因為透過與他們交談，我可以更深入地了解我們需要解決的未滿足的醫療需求。

So basically, this is the three pillars I have changed. And I say and we change of the therapeutic groups where the hypochondroplasia is growing because allocation of patient from ISS over to hypochondroplasia. And their heterogenic group that really I feel when I talk to the patients, when I talk to the patient organization, need a treatment. And the very extremely positive data we got for was post trial. Where when you have such a histogenic, some of the key element would be the CNP. Growth hormone will help. But one CNP is the key element. CNP will help.

所以基本上，這就是我改變的三大支柱。我說的是，我們改變了治療組，因為軟骨發育不良症患者的數量正在增加，所以從 ISS 轉移到了軟骨發育不良症患者。當我與患者、與患者組織交談時，我真切地感受到他們的異質群體需要治療。我們在試驗後獲得了非常積極的數據。當你有這樣的組織發生時，一些關鍵元素就是 CNP。生長激素會有所幫助。但一個 CNP 是關鍵要素。CNP 將會提供協助。

And this is why when you have such a histogenic group, having the combination therapy will be the most realistic way to treat them in a way where you really will have a fundamental good treatment regime.

這就是為什麼當您有這樣的組織發生組時，聯合治療將是最現實的治療方法，這樣您才能真正擁有一個根本的良好治療方案。

Great, thank you for that color.

太好了，謝謝你的顏色。

David Lebowitz, Citi.

花旗銀行的 David Lebowitz。

Thank you very much for taking my question. Curious, has there been any evolution in your thinking on the ultimate size of the market for YORVIPATH? Curious to know.

非常感謝您回答我的問題。好奇的是，您對 YORVIPATH 的最終市場規模的想法有什麼變化嗎？很好奇。

You know, I always been bullish and said that it's going to be a EUR5 billion to EUR8 billion market segment. I have no doubt that it will be that.

你知道，我一直很樂觀，並說這將是一個價值 50 億歐元到 80 億歐元的細分市場。我毫不懷疑它會是那樣。

Yun Zhong, Wedbush.

雲中，韋德布希。

Hi. Thanks very much for taking the questions. And the first question on TransCon CNP for achondroplasia. Assume that you receive FDA approval by the PDUFA date, how quickly will you be able to launch the product? And second question on hypochondroplasia and based on your comments just now but the press release seems to be saying that either still either monotherapy or combination therapy.

你好。非常感謝您回答這些問題。第一個問題是關於 TransCon CNP 治療軟骨發育不全症。假設您在 PDUFA 日期之前獲得 FDA 批准，您多快能夠推出產品？第二個問題是關於軟骨發育不良的，根據您剛才的評論，但新聞稿似乎說的是，要么仍然是單一療法，要么是聯合療法。

So is it still a possibility that you might end up going with the monotherapy for hypochondroplasia? And if that's the case, then what would be the reason why you don't go with combo therapy given the obvious benefit from the COACH study?

那麼，您是否仍有可能最終採用單一療法治療軟骨發育不良症？如果事實確實如此，那麼既然 COACH 研究表明聯合療法有明顯的好處，為什麼不選擇聯合療法呢？

The first one is we actually made some kind of with SKYTROFA. We will wait until we get the approval and likely discuss with force me to wake up in the morning, very, very, very early, that would be press release or some kind of call at 05:00 in my time in the morning. And think at that time, we will explain exactly when we have seen the labeling, got all the CMC information back from FDA and other places. When we will launch the product. And Jay will also be forced to be up in the morning and he will explain how we're going to do our launch strategy in this way. So you need to wait a little bit to this early morning call and we will try not to do it on Sunday, but we will try to take it every other day.

第一個是我們實際上與 SKYTROFA 合作做了一些事情。我們將等到獲得批准，並可能討論強迫我早上起床，非常非常早，這將是新聞發布會或某種電話，在我的時間早上 05:00。想想當時的情況，我們會準確地解釋我們何時看到標籤，何時從 FDA 和其他地方獲得所有 CMC 資訊。我們什麼時候推出該產品。傑伊也會被迫在早上起床，他會以這種方式解釋我們將如何制定我們的發布策略。因此，您需要等待一段時間才能接到這個清晨電話，我們會盡量不在周日接到，但會嘗試每隔一天接聽一次。

So the second part is a little bit about what we talked about before. This hypochondroplasia is, in my view, is an extremely heterogenic population now with a lot of what I call burden and different ways. And also now with the reallocation of patients from the ISS group into the hypochondroplasia. It will some way from my perspective, OPTIMAL will be to use the combination. And we will continue our dialogue with our regulatory agencies around the world to be sure that they also have the aligned view.

第二部分稍微涉及了我們之前討論的內容。在我看來，軟骨發育不全症是一種極其異質的群體，具有許多我稱之為負擔和不同的方式。現在，ISS 組的患者也被重新分配到軟骨發育不良組。從我的角度來看，某種程度上，最佳方法是使用這種組合。我們將繼續與世界各地的監管機構進行對話，以確保他們也持有一致的觀點。

I cannot, in some way, eliminate the discussion if we will have in the clinical trial a single arm also reflecting TransCon CNP as a monotherapy. But it's our belief that the combination therapy will be what is best for the patients. This way. So this is where we need to after the time being, we believe that the combination is the most robust treatment on it. But, obvious, there would be some patients that also just would benefit of having TransCon CNP as a monotherapy.

如果我們在臨床試驗中有一個單獨的組別也反映 TransCon CNP 作為單一療法，那麼我無法以某種方式消除討論。但我們相信聯合療法對患者來說是最好的。這邊走。所以這是我們暫時需要的，我們相信組合是最有效的治療方法。但是，顯然，有些患者也會受益於 TransCon CNP 作為單一療法。

Thank you.

謝謝。

Maxwell Skor, Morgan Stanley.

摩根士丹利的馬克斯韋爾·斯科爾。

Great. Thank you for squeezing me in, and forgive me if this question has been asked. But given the magnitude of growth velocity improvement in the COACH trial, do you believe a single pivotal trial could be sufficient for approval of the combination? Have you received any preliminary feedback from the FDA or EMA? Thank you very much.

偉大的。感謝您抽出時間回答我的問題，如果您問了這個問題，請原諒我。但考慮到 COACH 試驗中生長速度改善的幅度，您是否認為一次關鍵試驗就足以批准該組合？您是否收到 FDA 或 EMA 的任何初步回饋？非常感謝。

Hey. Yeah. I had a lot of discussion with Aimee Shu about that. She's sitting in my side. Because when we started this trial, we talked about our own expectations. And now we're sitting with a growth velocity that is so unprecedented and never been seen in achondroplasia. There are basic growing faster than a normal child. I have four children and I remember when they have growth spurts. It was not really good. We're pretty tall with Scandinavian. So you really grow a lot. So you basically will be in a position that is this is really a big change in that.

嘿。是的。我和 Aimee Shu 就此進行了許多討論。她坐在我身邊。因為當我們開始這項試驗時，我們談到了我們自己的期望。現在我們正處於前所未有的生長速度之中，這是軟骨發育不全症中從未見過的。基本上都比正常孩子長得快。我有四個孩子，我記得他們成長的高峰期。這確實不太好。我們是斯堪的納維亞人，身材很高。所以你確實成長了很多。所以你基本上會處於這樣一個位置，這確實是一個很大的變化。

And some way, I see it in and we some way go back and forward in that discussion. Will you take a treatment of one year? Will you take a treatment of two years? In the combination? You will get major growth and other positive development. Will you then go back for one or two years just have TransCon CNP as a monotherapy then you would boost again if there is desire from the parents for the child and birthings related to death and if it's really unneeded at that time.

在某種程度上，我看到了這一點，我們在某種程度上來回討論了這個問題。您會接受一年的治療嗎？您會接受兩年的治療嗎？在組合中？您將獲得重大成長和其他積極的發展。那麼，您是否會回去一兩年，只使用 TransCon CNP 作為單一療法，然後如果父母對孩子有渴望並且生育與死亡有關，並且如果當時確實不需要，您會再次加強治療。

So in some way, when I think about the overall way how we will do the best for the patient in the achondroplasia is basic to give them that option that we really can be in a position where they can take combination therapy likely for one year, potentially two years, and other things like that. And get all the benefit in that gate, and then we can continue with our TransCon CNP. And if they have a desire to move into another one yearly or two yearly treatment, they will can do that again. That is more or less our thinking in this way.

因此，從某種程度上來說，當我從整體上考慮如何為軟骨發育不全患者提供最好的治療時，最基本的就是為他們提供選擇，讓他們能夠接受一年、兩年的聯合治療，等等。並獲得該門的所有好處，然後我們就可以繼續我們的 TransCon CNP。如果他們希望進行一年一次或兩年一次的治療，他們可以再次這樣做。這或多或少就是我們這樣的想法。

Great. Thank you very much.

偉大的。非常感謝。

That does conclude the question-and-answer session. Portion of this meeting. This also concludes the meeting itself. I'd like to thank you for your participation in today's. This does conclude the program. You may now disconnect.

問答環節到此結束。本次會議的一部分。會議本身也到此結束。我要感謝你們參加今天的活動。該計劃確實就此結束。您現在可以斷開連線。

Thanks so much.

非常感謝。

Thank you.

謝謝。