Ascendis Pharma A/S (ASND) 2026 Q1 法說會逐字稿

Good day, and thank you for standing by. Welcome to the first quarter Ascendis Pharma earnings conference call. (Operator Instructions) Please be advised that today's conference is being recorded. I will now hand the conference with your first speaker today, Chad Fugere, Vice President of Investor Relations. Please go ahead.

各位好，感謝您稍候。歡迎參加 Ascendis Pharma 第一季財報電話會議。（接線員指示）請注意，今天的會議將被錄音。現在我將把會議交給今天的第一位講者——投資人關係副總裁 Chad Fugere。請開始。

Thank you, operator, and thank you, everyone, for joining our first quarter 2026 financial results conference call. I'm Chad Fugere, Vice President of Investor Relations at Ascendis Pharma. Joining me on the call today are Jan Mikkelsen, President and Chief Executive Officer; Scott Smith, Chief Financial Officer; Sherrie Glass, Chief Business Officer; and Jay Wu, EVP, and President, US Market.

謝謝接線員，也謝謝各位參加我們 2026 年第一季財務結果電話會議。我是 Ascendis Pharma 投資人關係副總裁 Chad Fugere。今天與我一同出席的有：總裁暨執行長 Jan Mikkelsen；財務長 Scott Smith；商務長 Sherrie Glass；以及執行副總裁暨美國市場總裁 Jay Wu。

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 YORVIPATH, YUVIWEL, and SKYTROFA, as well as certain expectations regarding patient access and financial outcomes, 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 planned regulatory filings and our expectations regarding and the result regulatory decisions. These statements are based on information that is available to us as of today.

在開始之前，我想提醒各位，本次電話會議將包含前瞻性陳述，旨在受《私人證券訴訟改革法》所提供之「安全港」條款保護。此類陳述的例子可能包括但不限於：關於我們對 YORVIPATH、YUVIWEL 與 SKYTROFA 的商業化與持續開發；以及對病患可近性與財務成果的某些預期；我們的研發管線候選產品及其持續進展與潛在商業化的預期；我們的策略計畫、合作關係與投資；我們對臨床研發管線的目標（包括臨床結果與試驗時程）；我們持續規劃中的法規申請，以及我們對法規決策之預期與其結果。這些陳述係基於截至今日我們可取得的資訊。

Actual results may differ materially from those in our forward-looking statements. You should not place undue reliance on these statements. We assume no obligation to update these statements if circumstances change except as required by law. For 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 11, 2026.

實際結果可能與我們的前瞻性陳述存在重大差異。您不應過度依賴這些陳述。除法律要求外，若情勢改變，我們不承擔更新這些陳述之義務。關於可能導致實際結果與前瞻性陳述出現重大差異之因素的更多資訊，請參閱我們今日新聞稿中的前瞻性陳述章節，以及我們於 2026 年 2 月 11 日向美國證券交易委員會（SEC）提交之最新 Form 20-F 年報中的風險因素章節。

TransCon PTH is approved in the US by the FDA for the treatment of hypoparathyroidism in adults, and the European Commission in the United Kingdom's Medicines 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 PTH 已獲美國 FDA 核准用於治療成人甲狀旁腺功能低下症；歐盟執委會與英國藥品與醫療產品管理局（MHRA）亦已核發 TransCon PTH 的上市許可，作為替代療法，適用於治療成人慢性甲狀旁腺功能低下症。

TransCon CNP is approved in the US by the FDA for the treatment of achondroplasia in children aged 2 years and older. Continued approval for this indication, which is based on an improvement in annualized growth velocity, may be contingent upon verification and description of clinical benefit and confirmatory trials.

TransCon CNP 已獲美國 FDA 核准用於治療 2 歲及以上兒童之軟骨發育不全症。此適應症之持續核准係基於年化生長速度的改善，可能取決於臨床效益之驗證與描述，以及確認性試驗。

TransCon hGH is approved in the US by the FDA for the replacement of endogenous growth hormone in adults with growth hormone deficiency in addition to the treatment of pediatric growth hormone deficiency, and in the EU has received NAA authorization from the European Commission for the treatment of pediatric growth hormone deficiency. Otherwise, please note that our product candidates are investigational and not approved for commercial use. As investigational products, the safety and effectiveness of 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.

TransCon hGH 已獲美國 FDA 核准，用於生長激素缺乏之成人的內源性生長激素替代治療，並用於治療兒童生長激素缺乏；在歐盟方面，已獲歐盟執委會核發 NAA 授權，用於治療兒童生長激素缺乏。除此之外，請注意，我們的產品候選物仍屬研究用，尚未獲准商業使用。作為研究用產品，其安全性與有效性尚未經任何主管機關審查或核准。本次電話會議中任何關於我們產品候選物的陳述均不應被視為促銷。

On the call today, we'll discuss our first quarter of 2026 financial results and we'll provide further business updates. Following some prepared remarks, we'll then open up the call for your questions. With that, let me turn it over to Jan.

今天的電話會議中，我們將討論 2026 年第一季財務結果，並提供進一步的業務更新。在一些事先準備的發言之後，我們將開放提問。接下來我把時間交給 Jan。

Thanks, Chad. Good day, everyone, here from COVID Hague. At this quarter of 2026, we'll send an IOP. I'll take 7.5 cities with the FDA approval of our suit and the product QFL. Our revenues are growing rapidly. We are profitable. We have a pipeline of high-value product opportunities to support long-term growth.

謝謝，Chad。各位好，我在 COVID Hague 向大家問候。在 2026 年這一季，我們將送出一份 IOP。我將以 7.5 個城市、以及我們的 suit 與產品 QFL 獲得 FDA 核准為重點。我們的營收正在快速成長。我們已實現獲利。我們擁有一條高價值產品機會的研發管線，可支持長期成長。

Three elements are cementing our position as a leading global biopharma company. First, our diversified product portfolio in one single therapeutic area. Following is the approval of Yura. We have now achieved approval of three products in a row. Across four rare anchor crime indications.

三個要素正在鞏固我們作為全球領先生物製藥公司的地位。第一，我們在單一治療領域內擁有多元化的產品組合。繼 Yura 獲准之後，我們現在已連續取得三項產品核准，涵蓋四個罕見 anchor crime 適應症。

Second, our rapid revenue growth. From our existing anchor crime RFCs portfolio, Durapans, Durable, and Sky Fulfer, each highly differentiated. With long durability, we expect sustained revenue growth for many years to come.

第二，我們的營收快速成長。來自既有的 anchor crime RFCs 產品組合——Durapans、Durable 與 Sky Fulfer——各自高度差異化，且具長效持久性，我們預期未來多年營收將持續成長。

Third, expanding our pipeline. We have proven our ability to create transformative medicines, addressing unmet medical needs using our TransCon technology platform. To date, we have more than 20 ongoing or planned clinical trials aiming at label and market inspection, including four new clinical entities in pre-clinical development.

第三，擴展我們的研發管線。我們已證明能運用 TransCon 技術平台創造具變革性的藥物，滿足未被滿足的醫療需求。迄今為止，我們有超過 20 項正在進行或規劃中的臨床試驗，目標為標籤與市場 inspection，其中包括四個處於臨床前開發的新臨床實體。

Turning now to URPATs. URPATs revenue in the first quarter reached eur197 millionos. URPAT revenue for the first quarter was impacted by two one-time hyphens. A temporary increase of U.S. Patients supported by free drugs caused by reimbursement disruption and one-time impact of Europe Direct related to expected market assess. Scott will explain the financial impact of these two events in his remarks.

接著談 URPATs。URPATs 第一季營收達到 1.97 億歐元。URPAT 第一季營收受到兩項一次性 hyphens 的影響：因給付報銷中斷導致美國由免費用藥支持的病患暫時增加，以及與預期市場 assess 相關的 Europe Direct 一次性影響。Scott 將在他的發言中說明這兩項事件的財務影響。

In the U.S., new patients, neuroma and fewer remain in line. With the strong uptake we have seen in Q4 2025, with more than 1,000 new patients prescribed UROPADS during the quarter. To the end of March 2026, more than 6,300 patients have been prescribed UROPADS by more than 2,700 unique healthcare providers.

在美國，新病患、neuroma 與 fewer 仍保持在 line。隨著我們在 2025 年第四季看到的強勁採用，本季有超過 1,000 名新病患被開立 UROPADS。至 2026 年 3 月底，已有超過 6,300 名病患由超過 2,700 位不同的醫療照護提供者開立過 UROPADS。

March was our last revenue month ever for Europass, supported by an increased number of new patients as well as patients returning to reimbursement from VCOG. Importantly, the enrollment we saw in Q1 has continued through April, consistent with our guidance.

3 月是我們 Europass 的最後一個營收月份，受惠於新病患數增加，以及病患自 VCOG 回到報銷體系。重要的是，我們在第一季看到的入組情況已延續到 4 月，與我們的財測指引一致。

Insurance approval rates and medium time to approval continue to improve. This strong support a strong foundation for revenue growth in 2026 and many years to come. Outside the U.S., Europad is available commercially or to name patient programs in 35 countries, including full commercial reimbursement in six of our Europe direct markets, with additional launches expected through 2026.

保險核准率與核准的中位時間持續改善。這為 2026 年及未來多年營收成長奠定了堅實基礎。在美國以外，Europad 已在 35 個國家以商業方式供應或透過指名病患計畫提供，其中在我們 Europe direct 市場中的 6 個已取得完整商業報銷，並預期在 2026 年期間持續有更多上市。

Looking forward ahead, we continue to pursue multiple expansion opportunities for UOPETs in new markets and education. These include doses up to 60 micrograms in the U.S., global expansion to patients 8, 12, to 18, and continue developing of what we get transcond PTAs for patients on stable UOPET doses. With 70 to 90,000 patients living with chronic hyper care in the U.S., and five to 10 times that number outside the U.S., we remain highly confident in Europe's long-term global protections.

展望未來，我們將持續在新市場與教育方面，推進 UOPETs 的多項擴張機會。這些包括在美國將劑量提高至 60 微克、全球擴展至 8、12 至 18 歲病患，以及持續開發我們所稱的 transcond PTAs，適用於使用穩定 UOPET 劑量的病患。美國約有 7 萬至 9 萬名慢性 hyper care 病患，且美國以外的人數為其 5 至 10 倍，我們對 Europe 的長期全球 protections 仍高度有信心。

I will now turn to our growth disorder branches. With once-week URL and outside our once-weekly growth remote schedule firm. We believe Ascendis is uniquely positioned to strengthen its leadership in those disorders. Our U.S. Commercial infrastructure, built and refined since Catholic launched in 2021, has enabled a focused and high-impact launch for Eurobell, which became commercially available in early April. Since then, Eurobell. Has already been described for more than 60 children by more than 35 unique healthcare providers, with children approved for reimbursement as fast as a few days.

接下來我將談到我們的生長障礙業務。隨著 once-week URL，以及在我們 once-weekly growth remote schedule firm 之外，我們相信 Ascendis 具備獨特優勢，可強化在這些疾病領域的領導地位。我們自 2021 年 Catholic 上市以來建立並精進的美國商業基礎設施，使 Eurobell 得以在 4 月初開始商業供應後，進行聚焦且高影響力的上市推進。自那以來，Eurobell 已由超過 35 位不同的醫療照護提供者為 60 多名兒童開立處方，且兒童取得報銷核准最快僅需數天。

UroPill has shown compelling results compared to placebo across multiple clinical trials. In addition to linear growth outcomes. These results include improvements in leg blowing, spinal kind of dimensions, body operationality, physical function, and health-related quality of life compared to disabled without compromising safety or total ability.

UroPill 在多項臨床試驗中，相較安慰劑展現具說服力的結果。除線性生長結果外，這些結果還包括相較於 disabled，在不影響安全性或 total ability 的情況下，腿部 blowing、脊椎 kind of dimensions、身體 operationality、身體功能，以及與健康相關的生活品質皆有所改善。

CPD, these outcomes will take UOVEL's unique FPDC to provide continuous systemic CMP exposure to all of the body over the weekly dosing interval. Looking ahead, we plan to make UOVEL available in selected international markets through early asset programs using the U.S. FDA approval. As a reminder, our global infrastructure covers over 70 countries and has already generated product revenue for us in more than 35 countries.

CPD，這些結果將需要 UOVEL 獨特的 FPDC，在每週給藥間隔內為全身提供持續的系統性 CMP 暴露。展望未來，我們計劃透過早期資產計畫，利用美國 FDA 的核准，讓 UOVEL 在部分國際市場上市。提醒一下，我們的全球基礎設施覆蓋超過 70 個國家，且已在超過 35 個國家為我們帶來產品營收。

In EU, a regulatory decision on our marketing authorization application for EURL is expected in the fourth quarter of 2026. We are also pursuing label expense in renewable to ongoing trials. These include infants under two years of age with acondiplation and children with hypercondiplation, as well as geographic label expense in clinical trials.

在歐盟，預計將於 2026 年第四季就我們 EURL 的上市許可申請作出監管決定。我們也正在推進在可再生的持續試驗中的標籤費用擴展。這些試驗包括兩歲以下、患有 acondiplation 的嬰幼兒，以及患有 hypercondiplation 的兒童，並包含在臨床試驗中的地理標籤費用擴展。

Turning now to Skytrofer. In the U.S., Skytrofer maintains consistent performance as a premium product with seven. Share of the overall program market, reflecting steady demand across pediatric and adult patients as the only once-weekly product delivering on revised somatopoids. With the expected label expansion that could double the addressable patient population in the U.S. And geographic expansion outside the U.S., we believe SKY-TOVA will remain a cornerstone product in our growth disorder portfolio.

接下來談 Skytrofer。在美國，Skytrofer 作為高端產品維持一致的表現，佔整體計畫市場 7 的份額，反映出在兒科與成人患者中的穩定需求；其為唯一每週一次給藥、可實現修訂後 somatopoids 的產品。隨著預期的標籤擴展，可能使美國可觸及的患者人數倍增；再加上美國以外的地理擴張，我們相信 SKY-TOVA 將持續成為我們生長障礙產品組合的基石產品。

Turning to our pipeline, this includes combination therapy with one-sweeted TransCon CMP and TransCon glutamoles for shipments with achonoplasia. In our Phase Two code file, we have reported unprecedented results that exceeds the already compelling foundation established by URL one of therapy. We easy to take from codes. Presented in January showed improvement in acontablaser-specific height set score that indicate a triple of efficacy compared to transcontCMP monotherapy, along with improvement in body corporationality.

接著談我們的研發管線，其中包括以一次甜化的 TransCon CMP 與 TransCon glutamoles 的合併療法，用於患有 achonoplasia 的出貨。在我們的第二期 code 試驗檔案中，我們已報告前所未見的結果，超越了 URL 單一療法所建立、已相當有說服力的基礎。我們很容易從 codes 中得出。於一月發表的資料顯示，acontablaser 特異性的身高設定分數有所改善，顯示其療效相較於 transcontCMP 單藥治療提升至三倍，同時也改善了身體比例（corporationality）。

More recently, we shared additional VIC52 data that showed improvement in the volume alignment, as well as an accelerate improvement in spinal canal dimensions. And an improvement in arm strength not previously demonstrated with pharmacotherapy within a single treatment here. Based on this finding, we believe our unique combination therapy of transcon CRP and transcon COV hormone could potentially eliminate the need for high invasion procedure such as the leg lengthening. And leg straightening surgeries. We believe that this combination therapy could become the preferred treatment option for acondoplasia.

更近期，我們分享了額外的 VIC52 數據，顯示體積對齊有所改善，且脊椎管尺寸出現加速改善；並且在單一治療期間內，手臂力量也有所提升，這在以往藥物治療中尚未被證實。基於此發現，我們相信我們獨特的 transcon CRP 與 transcon COV hormone 合併療法，可能可消除對高侵入性處置（例如腿部延長與腿部矯正手術）的需求。我們相信此合併療法可成為 acondoplasia 的首選治療方案。

I will now briefly turn to oncology. In our Phase I-II, I believe, trial, we have evaluated the transcon here to beta-gamin in combination with Paclitab in patients with late-stage plexus and precision-aware mechanism of ophiology. Medium OS improved up to 10 months from six to seven months from historical controls with a general wealth-tolerated safety profile, validated the science there on TransCon2 beta carbon.

我現在簡要轉到腫瘤領域。在我們的第一至第二期（Phase I-II，我認為）試驗中，我們評估了 transcon here to beta-gamin 與 Paclitab 的合併療法，用於晚期 plexus 以及具 precision-aware mechanism 的 ophiology 患者。中位 OS 相較於歷史對照由 6 至 7 個月提升至最多 10 個月，且整體安全性良好、耐受性佳，驗證了 TransCon2 beta carbon 的科學基礎。

As for internal oncology development, those not align with our strategic focus. We have decided to discontinue internal development of TransCon R2 beta-carbonate in our quality and will explore other ways to maximize the value of these assets.

至於內部腫瘤研發，這些並不符合我們的策略重點。我們已決定停止在我們的 quality 中對 TransCon R2 beta-carbonate 的內部開發，並將探索其他方式以最大化這些資產的價值。

Turning now to our partnership, our once-monthly TransCon similar routine with no one notice continue to amend towards the clinic. At Arconis, TransCon, NTBDF also ranked on track to enter the vaccine this year. These programs further highlight the broader potential of our TransCon technology platform to address product opportunities in larger indicators.

接著談我們的合作夥伴關係，我們每月一次的 TransCon similar routine（無人注意）持續朝臨床推進。在 Arconis，TransCon，NTBDF 也仍按計畫於今年進入疫苗。這些計畫進一步凸顯我們 TransCon 技術平台的更廣泛潛力，可在更大型的適應症中把握產品機會。

In closing, in the first quarter of 2026, we made significant progress across our business and our ability to make a meaningful difference. We have 3 FDA-approved TransCon products across four indications, growing revenues, improving cash generation, and a pipeline that support long-term growth. I will now turn the call over to Scott to review our financial results.

最後，在 2026 年第一季，我們在整體業務以及創造有意義影響的能力方面取得重大進展。我們已有 3 款 FDA 核准的 TransCon 產品，涵蓋四項適應症，營收成長、現金創造能力改善，且研發管線支持長期成長。接下來我將把電話交給 Scott，請他回顧我們的財務結果。

Thanks a lot, Jan, and good afternoon, everyone. I will touch on some key points surrounding our first quarter financial results, which reinforce our confidence for growing operating profit and cash flow into the future. For further details, please refer to our Form 6-K file today.

非常感謝你，Jan，各位下午好。我將談及一些與我們第一季財務結果相關的重點，這些結果強化了我們對未來營業利益與現金流成長的信心。更多細節請參閱我們今天提交的 Form 6-K 文件。

YORVIPATH global revenue was EUR197 million in Q1. The first quarter was characterized by steady global uptake and normal seasonality, as well as two one-time items. Patients temporarily transitioned to free drug in the border of the US and a one-time impact in Europe Direct related to expanded market access. The combined impact of these two items was approximately EUR15 million.

YORVIPATH 第一季全球營收為 1.97 億歐元。第一季的特徵是全球採用穩健成長與正常季節性，並包含兩項一次性因素：患者在美國邊境地區暫時轉為使用免費藥物，以及歐洲 Direct 因擴大市場准入而產生的一次性影響。這兩項因素合計影響約 1,500 萬歐元。

SKYTROFA contributed EUR44 million in Q1. On a sequential basis, performance reflected consistent underlying demand with the expected drawdown in channel inventory built in Q4. Including EUR6 million in collaboration revenue, total Q1 2026 revenue amounted to EUR247 million.

SKYTROFA 第一季貢獻 4,400 萬歐元。以季對季來看，表現反映出一致的基本需求，並如預期出現第四季建立之通路庫存的回落。包含 600 萬歐元的合作收入在內，2026 年第一季總營收為 2.47 億歐元。

Continuing to expenses, R&D expenses in Q1 were EUR59 million, down from EUR78 million in Q4 '25. R&D in Q1 was favorably impacted by a write-up of YUVIWEL inventory, consisting of EUR11 million due to US FDA approval and lower clinical activity across the portfolio.

接著看費用，第一季研發費用為 5,900 萬歐元，低於 2025 年第四季的 7,800 萬歐元。第一季研發費用受到 YUVIWEL 庫存回沖的有利影響，其中包含因美國 FDA 核准而帶來的 1,100 萬歐元，以及整體產品組合臨床活動降低。

SG&A expenses rose EUR245 million in Q1 2026, compared to EUR136 million in Q4 2025, reflecting continued impact of global commercial expansion. Total operating expenses for Q1 2026 were EUR204 million, and operating profit was EUR25 million, reflecting a 10% operating margin.

2026 年第一季 SG&A 費用升至 2.45 億歐元，相較 2025 年第四季的 1.36 億歐元，反映全球商業化擴張的持續影響。2026 年第一季總營業費用為 2.04 億歐元，營業利益為 2,500 萬歐元，對應 10% 的營業利益率。

Non-IFRS operating profit was EUR55 million and non-IFRS operating margin was 22%. As revenue scales, we expect meaningful improvement in our operating margin, which will be visible over the course of 2026 and beyond.

非 IFRS 營業利益為 5,500 萬歐元，非 IFRS 營業利益率為 22%。隨著營收規模擴大，我們預期營業利益率將有顯著改善，並將在 2026 年及其後逐步顯現。

Net finance expense for Q1 2026 was EUR63 million, primarily driven by non-cash items, including remeasurement loss of financial liabilities of EUR34 million. Net cash financial expense for Q1 2026 was about EUR1 million.

2026 年第一季淨財務費用為 6,300 萬歐元，主要由非現金項目所驅動，包括 3,400 萬歐元的金融負債重新衡量損失。2026 年第一季淨現金財務費用約為 100 萬歐元。

Net profit for Q1 2026 was EUR629 million, which included recognition of a EUR679 million deferred tax asset in the P&L. Refer to our 6K for more detail. Non-IFRS net profit was EUR18 million, or EUR0.27 cents per share. We ended Q1 2026 with EUR573 million in cash and cash equivalents, which includes the impact of EUR60 million in Q1 from our previously announced share repurchase program and net settlement of certain RSUs.

2026 年第一季淨利為 6.29 億歐元，其中包含在損益表中認列 6.79 億歐元的遞延所得稅資產。更多細節請參閱我們的 6-K。非 IFRS 淨利為 1,800 萬歐元，或每股 0.27 美分。我們在 2026 年第一季末持有 5.73 億歐元的現金及約當現金，其中包含第一季 6,000 萬歐元（來自先前公告的庫藏股回購計畫）以及部分 RSU 的淨結算影響。

In April, we successfully completed our transition to a direct listing of our ordinary shares on Nasdaq. We believe this will broaden access to global investment in the company, which has the potential to further enhance institutional ownership and trading liquidity for incentive shares. In May, we completed the full redemption of all of our outstanding convertible senior notes.

四月，我們成功完成普通股在納斯達克的直接上市轉換。我們相信這將擴大全球投資人對公司的投資管道，並有望進一步提升機構持股與激勵股的交易流動性。五月，我們完成了所有未償還可轉換優先票據的全數贖回。

Finally, today, we announced that we entered into an agreement to sell our PRV for USD187.5 million in cash. PRV was awarded by the US FDA upon approval of YUVIWEL in February.

最後，我們今天宣布已簽署協議，以 1.875 億美元現金出售我們的 PRV。PRV 係美國 FDA 於二月核准 YUVIWEL 時所授予。

Turning to our commercial outlook, for YORVIPATH, we expect a continued steady underlying increase in patient cell therapy and the reversal of one-time factors seen in Q1 to drive strong growth sequentially in Q2. For SKYTROFA, we expect stable revenue throughout the year following a similar seasonal pattern to 2025. Regarding YUVIWEL, as Jan indicated earlier, we are encouraged by the early demand trends and look forward to sharing more with you on our Q2 calls.

接著談我們的商業展望。就 YORVIPATH 而言，我們預期患者細胞治療將持續穩健、基本面上升，且第一季所見的一次性因素將在第二季反轉，帶動第二季較第一季呈現強勁的序列成長。就 SKYTROFA 而言，我們預期全年營收保持穩定，並延續與 2025 年相似的季節性型態。至於 YUVIWEL，如 Jan 先前所述，我們對早期需求趨勢感到鼓舞，並期待在第二季電話會議中與各位分享更多資訊。

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

以上，接線員，我們現在準備開始提問。

(Operator Instructions) Jessica Fye, JPMorgan.

（接線員指示）Jessica Fye，摩根大通。

Good morning. Thanks so much for taking my question. I was wondering if you could help us estimate what US YORVIPATH sales were in the quarter. I think in the past, you had run through an algorithm to consider, but I know the press release noted some one-time impact to Europe Direct as well. So just trying to get a better sense of the US versus ex-US split this time around. Thank you.

早安。非常感謝讓我提問。我想請教你們是否能協助我們估算本季美國 YORVIPATH 的銷售額。我記得過去你們曾用一個演算法來推算，但我也知道新聞稿提到 Europe Direct 也有一些一次性影響。所以我只是想更清楚了解這次美國與美國以外（ex-US）的拆分。謝謝。

I think that Scott will give you just a little bit more background on the finance element specific that one time in your directory. It's actually happened when we sometime and specific for one single country because we had an early access program that was running for nearly 15 months, 16 months. And it gave us a one-time event where we needed to write down for this single case. It's not something that really happening in other countries, but it was a single country event which basically impacted our US, you can say, Q1 results. But Scott can give you a little bit more flavor on the finance numbers.

我想 Scott 會就你們目錄中那一次特定的財務項目，提供更多一點背景。這其實發生在我們某個時候、且僅針對單一國家，因為我們有一個早期使用計畫（early access program）運行了將近 15 個月、16 個月。這帶來了一次性的事件，使我們需要就這個單一案例進行減記（write down）。這不是其他國家也在發生的事情，而是一個單一國家的事件，基本上影響了我們美國（你可以說）第一季的業績。不過 Scott 可以就財務數字提供你更多細節。

Just for modeling purposes, it's probably a little bit more of an impact, but I would say that the best way to think about it is take the algorithm that we laid out where you're at four to five a quarter. And for Q1, basically, that addition was just shifted into Q2, and then from there, the algorithm continues.

就建模（modeling）而言，它可能影響稍微大一點，但我會說最好的理解方式是：採用我們提出的那個演算法（algorithm），也就是每季約四到五（的節奏）。而在第一季，基本上那個增加量只是被移轉到第二季，之後就會延續該演算法的走勢。

But I think just one of the key elements I will take into regard is basically the underlying patient demand, because I think the key element is really that we continue the same successful, you can say, rollout of the drugs, both in US, where we now, as we have provided you the number of imitated new patients on treatment in European.

但我想我會特別注意的一個關鍵要素，是底層的病患需求（underlying patient demand），因為關鍵在於我們持續維持同樣成功的藥物推廣（rollout），包括在美國；而在歐洲，我們也已向你們提供了開始治療的新病患數（imitated new patients on treatment）的數字。

And as we have given in our previous guidance, we still 100% correct in that, where we really see the same stability, we see the same flow coming in. And Jay can comment about how he already starting to improve both the time to reimbursement and the numbers. I do not -- Jay, will you comment about your effort in really improving what you call the reimbursement situation for the US?

而且如同我們先前的指引（guidance）所述，我們仍然 100% 確認那是正確的：我們確實看到同樣的穩定性、同樣的流入量。Jay 也可以評論他如何已開始改善核准給付（reimbursement）的時間以及數量。我不——Jay，你能否談談你在真正改善你所稱的美國給付情況（reimbursement situation）方面所做的努力？

Sure. When you think about our reimbursement, we're seeing improved metrics across the board. So first and foremost, we've talked a little bit about our upstream coverage now expanding to about 80% of patient lives, which we're feeling really good about given the time on market. And I think, again, a testament to the compelling clinical value proposition that we have.

當然。談到我們的給付（reimbursement），我們看到各項指標全面改善。首先，我們先前提過，上游保險涵蓋（upstream coverage）目前已擴大到約 80% 的病患人群（patient lives）；以我們上市的時間來看，這讓我們感覺非常好。我想這也再次證明我們所具備的、具說服力的臨床價值主張（clinical value proposition）。

We're also seeing continued rapidity as it relates to patients being approved for reimbursement upon entering the funnel. So again, over half approved within eight weeks of enrollment. And we are seeing continued progress against patients moving through the funnel, whether it's upstream as the enrollments are coming in, but also supporting patients as they're entering into the funnel.

我們也看到，病患一旦進入漏斗（funnel），其給付核准速度仍然很快。因此，再次強調，超過一半在登記後八週內獲得核准。我們也持續看到病患在漏斗中的推進有所進展，不論是在上游端（upstream）隨著登記（enrollments）持續進來，或是在病患進入漏斗後對其提供支持。

Great. Thank you.

很好，謝謝你。

Tazeen Ahmad, Bank of America.

Tazeen Ahmad，美國銀行（Bank of America）。

Good morning. Thanks for taking my question. Mine will also be on YORVI, and maybe this is for Jay. Can you talk about the reauthorization rates that you're seeing now that patients are starting to annualize at this time of year? Any things to point out about things that were maybe unexpected or taking a little bit longer?

早安，謝謝讓我提問。我也會問 YORVI，可能是問 Jay。隨著病患在每年這個時點開始進入年度續期（annualize），你們目前看到的再授權（reauthorization）比率如何？有沒有什麼值得指出的地方，例如有些情況可能出乎意料，或是花的時間稍微更久？

And then can you talk about usage among physicians? So is there a way of providing a split between how much of use is coming from first-time physicians versus an increasing use among doctors who've tried YORVIPATH before? Thanks.

另外也請談談醫師端的使用情況。你們是否能提供一個拆分：使用量中有多少來自首次開立處方的醫師（first-time physicians），以及有多少來自曾經使用過 YORVIPATH 的醫師、其使用量持續增加？謝謝。

Okay, there was multiple questions. I hope you got everyone down. Will you start on some of them?

好的，這裡有多個問題。我希望你都記下來了。你要先從其中一些開始嗎？

Sure. I think I heard a few questions, one, which maybe I'll start with toward the end, which is prescriber breadth and depth, I think was the question. We're seeing continued traction across both. So as Jan mentioned earlier in the script, we've had over 2,700 prescribers, which again is an addition of about a 300 plus quarter over quarter, which we're feeling really good about. So that would answer your question around new prescribers.

當然。我想我聽到幾個問題，其中一個我先從後面開始回答，也就是處方醫師的廣度與深度（prescriber breadth and depth）。我們在這兩方面都持續看到動能。如 Jan 先前在講稿中提到，我們已有超過 2,700 位開立處方的醫師（prescribers），相較上一季增加約 300 多位，這讓我們感覺非常好。這就回答了你關於新處方醫師的問題。

And then within existing prescribers, we're also feeling really good because the average prescription per physician continues to increase as well. In fact, about 10%-ish of prescribers have now over five enrollments for patients.

至於既有處方醫師，我們也很有信心，因為每位醫師的平均處方量也持續上升。事實上，約有 10% 左右的處方醫師，目前已有超過五位病患完成登記（enrollments）。

Again, as you think about the provider landscape here, they all do have a different patient volume, just given how diffuse the patient volume is. But generally speaking, we're seeing not only one additional prescriber sign on to YORVIPATH, given the strong patient satisfaction scores that we're seeing. But then because of those positive patient experiences, we're also seeing providers expand their scope of who they deem to be eligible, given that lab values alone are not the reason that patients should be treated.

同樣地，當你看這裡的醫療提供者（provider）版圖時，由於病患量分布相當分散（diffuse），各家醫師的病患量確實不同。但整體而言，基於我們看到的高病患滿意度分數（patient satisfaction scores），我們不僅看到更多醫師加入使用 YORVIPATH；而且因為這些正向的病患經驗，我們也看到醫療提供者擴大他們認定符合治療資格的人群範圍，因為單靠實驗室數值（lab values）並不是病患應否接受治療的唯一理由。

Okay, thanks. And then reauthorization was the last one.

好的，謝謝。那最後一個是再授權（reauthorization）。

And reauthorization, yes, that was the first part of your question. We're not seeing any meaningful differences in terms of approval rates for a re-auth versus necessarily a patient that's coming in at the top of the funnel. We typically, again, have shared that four to 8-week time frame. We've seen those numbers continue to increase in terms of speed, which I referenced earlier with the first analyst question. But we're not seeing any meaningful difference with the re-auth coming in versus a new patient coming in.

再授權，是的，那是你問題的第一部分。我們在再授權（re-auth）與漏斗頂端（top of the funnel）新進病患之間的核准率方面，沒有看到任何具實質意義的差異。我們通常——同樣地——先前分享過是 4 到 8 週的時間範圍。我們看到這些數字在速度上持續改善，正如我先前在第一位分析師提問時所提到的。但再授權進來與新病患進來之間，我們沒有看到任何具實質意義的差異。

Generally, what you'll see is if it's a re-auth of an existing payer insurance where there hasn't been a change in insurance, you might see some faster timeline there. But if it's a brand-new insurance, then you're obviously going to treat it as just a brand-new case, so to speak.

一般來說，如果是既有付款方保險（existing payer insurance）的再授權，且保險沒有變更，你可能會看到時程更快一些。但如果是全新的保險，那你顯然就會把它當作一個全新的案件來處理。

Yaron Werber, TD Cowen.

Yaron Werber，TD Cowen。

Great. Thanks so much. Maybe a quick follow-up and then a question on YUVIWEL. A follow-up on YORVI. So of the $15 million, should we roughly kind of split it, like half in Europe and half in the US? I don't know if you can give us any sort of, view on that.

很好，謝謝。也許先快速追問一下，然後再問 YUVIWEL。關於 YORVI 的追問：那 1,500 萬美元，我們是否可以大致把它拆成歐洲一半、美國一半？不知道你們能否給我們任何角度或看法。

And then for YUVIWEL, in the ITC cases progressing, the briefing document, the briefs are out kind of back and forth, and it looks like the court is kind of siding with parties. What -- I know you've been importing drug in the meantime. Any of you sort of on how much capacity you might be able to have in the system by the time a decision is rendered? Thank you.

另外，關於 YUVIWEL，隨著 ITC 案件推進，簡報文件（briefing document）與各方的書狀（briefs）你來我往，看起來法院似乎在不同議題上偏向不同當事方。我知道你們這段期間仍在進口藥品。你們能否談談，在裁決出爐前，你們大概能在系統中建立多少供應/產能（capacity）？謝謝。

First of all, I believe when you see the uptick in the prescriptions of YUVIWEL in the US, we have more than 60 children being prescribed durable treatment in less than four or five weeks. I think it illustrates the unmet medical need that exists in the US related to an improved treatment in (inaudible). And I believe what we have seen of clinical data in our (inaudible) with YUVIWEL, just as a monotherapy, is really describing the reason why we see this takeoff.

首先，我相信當你看到 YUVIWEL 在美國處方量的上升時，我們在不到四到五週內就有超過 60 名兒童被開立長期治療（durable treatment）。我認為這說明了美國在（inaudible）相關、需要更佳治療方面所存在的未被滿足醫療需求（unmet medical need）。而且我相信，我們在（inaudible）中看到的 YUVIWEL 臨床數據——即使僅作為單一療法（monotherapy）——確實解釋了為何我們會看到這樣的起飛（takeoff）。

This is not just of having a once-weekly product. This is providing a suitability profile and documentated effect on benefit beyond linear growth. And I think everyone is aligning with the unmet medical need, the public interest, always to have a product, a say in the market.

這不只是因為它是一個每週一次（once-weekly）的產品。它提供的是更合適的特性（suitability profile），以及在「不僅限於線性成長（beyond linear growth）」之外、具文件佐證（documentated）的效益。我想大家都在與這個未被滿足的醫療需求、公共利益（public interest）對齊：市場上始終應該要有一個產品、也就是在市場中有一席之地。

We will continue to be in a position that we have such a strong belief that in this case here too, like it was in Europe, we can prove that this IP case, it should never have existed and only is built on four promises. So I'm 100% confident YUVIWEL is here to stay and it will always be a treatment option for patients with (inaudible) in the US. I hope that answer your question related to that part.

我們將持續維持我們的立場：我們非常堅信，在這個案例中也一樣——就像在歐洲那樣——我們可以證明這個智慧財產權（IP）案件本不該存在，而且它只建立在四個承諾之上。因此我 100% 有信心，YUVIWEL 會長期存在，並且永遠會是美國（inaudible）病患的一個治療選項。我希望這回答了你在那一部分的問題。

The other part, I think you are somewhere in the right estimate when you think about it.

至於另一部分，我想你在估算上大致是對的。

Gavin Clark-Gartner, Evercore.

Gavin Clark-Gartner，Evercore。

This is (inaudible) on for Gavin. We have two quick questions. Number one is for the Phase III (inaudible) CNP, and Growth Hormone Combo Trial, can you share any update on the enrollment speed? And secondly, what are you seeing in your YORVIPATH discontinuation rates over time?

我是（聽不清）代替 Gavin 發問。我們有兩個簡短問題。第一個是關於第三期（聽不清）CNP 與生長激素合併試驗，能否分享任何關於收案速度的最新進展？第二，隨著時間推移，你們觀察到 YORVIPATH 的停用率有什麼變化？

When we talk about, as I understood your question right, it was related to the combination, right? The Phase II trial, the trial we call the coach trial and we basic -- now I do not know how many years we are into the trial now, but I think the 2 years in or 1.5 years now in it. And I think we see basic and extremely high element of protection in this trial.

當我們談到——如果我理解你的問題沒錯——你問的是合併治療，對嗎？第二期試驗，也就是我們稱為 coach 試驗的那個試驗，我們基本上——現在我不確定我們進行這個試驗已經多少年了，但我想大概是進行了 2 年，或現在大約 1.5 年。我認為我們在這個試驗中看到基本上且極高程度的保護效果。

To my knowledge, last time I looked, it was 100%. And I think it's really been harder to get more than 100% in a clinical trial, to my knowledge. And I think that's a very few trials where you have 100% retention after nearly 18 months. So from that perspective, I think it's really illustrating and addressing the satisfaction with the benefit you see in the treatment compared to the burden of treatment. And I think that is really the key element that we always are looking at in the fundamentals. We want to see benefit for patients. This is why we're working and we will continue to focus on that.

據我所知，上次我查看時是 100%。而且據我所知，在臨床試驗中要超過 100% 真的很難。我認為在接近 18 個月後仍能維持 100% 留存率的試驗非常少。從這個角度來看，我認為這確實在說明並回應：相較於治療負擔，患者對於治療所帶來的效益之滿意度。我認為這正是我們在基本面上一直關注的關鍵要素。我們希望看到患者獲益。這就是我們努力的原因，我們也會持續聚焦於此。

Li Watsek, Cantor.

Li Watsek，Cantor。

Thanks, guys. I guess on your new patient, as you mentioned, steady growth, is it reasonable for us to assume a thousand is sort of the number that we should be looking at for the coming quarters? And will you be sharing your script number going forward? Thanks.

謝謝各位。我想就你們提到的新患者穩定成長來問：我們是否可以合理假設，接下來幾個季度應該關注的數字大約是 1,000？另外，你們未來會持續分享處方量（script number）嗎？謝謝。

That's a great question. Now, somebody going back to last time, I said I will not be come up with more prescription data for YORVIPATH because I believe that the revenue population was so clear. And when I said that there was a big, what I call the element of some interesting funds that push back and saying I didn't want to come up with numbers because it must be really, really bad.

這是個很好的問題。回到上次，我說過我不會再提供更多 YORVIPATH 的處方數據，因為我認為營收族群已經非常清楚。而當我這麼說時，有一大部分——我稱之為一些有趣資金的力量——反過來質疑，說我不願意提供數字，一定是因為情況非常、非常糟。

And now, we have illustrated for one quarter more that they are not bad, they are extremely good and exactly as predictable as we have said in this way. And I expect a steady state involvement in all the quarters because that is what we expect we are only touching a small amount of this patient group. We have some more patient that is coming to patient every year. So I'm somewhat giving up to say that I will not come up one quarter more because last time I said we will not come up with one quarter and then I've got basic press because I didn't want to listen to that. We didn't want to come up with a number because they're bad.

而現在，我們又用多一個季度證明情況並不糟，反而非常好，而且完全如我們所說的那樣可預測。我預期各季度都會維持穩定的狀態，因為我們預期目前只觸及這個患者族群中的一小部分。每年也會有更多患者進入這個患者池。因此我某種程度上也放棄了原先「不再提供」的說法，因為上次我說不會再提供一個季度，結果就受到一些壓力，因為外界不願意接受那種說法——他們認為我們不提供數字是因為數字很差。

I don't hide anything. I always want to be transparent and this is why I said come up with a number then you can see it. I think we will continue to be so transparent on everything what we're doing, so you always have the best possible opportunity to see how well we have performed in our fundamentals.

我沒有隱瞞任何事。我一直希望透明，這也是為什麼我說那就把數字拿出來，大家就能看到。我認為我們會在所有正在做的事情上持續保持透明，讓你們永遠有最佳的機會去了解我們在基本面上的表現有多好。

Alex Thompson, Stifel.

Alex Thompson，Stifel。

Thanks for taking the question. This is Patrick on for Alex. Could you guys just talk about the potential impact of the YORVI 60 micrograms being on the label and maybe what percentage of those 6,300 patients in the US is dose caps at 30 with maybe less than ideal supplementation levels?

謝謝讓我提問。我是 Patrick 代替 Alex 發問。你們能否談談 YORVI 60 微克若納入標籤，可能帶來的影響？以及在美國那 6,300 名患者中，有多少比例因劑量上限被限制在 30 微克，且可能補充治療水準不太理想？

This is a question which are very difficult for us to answer today because we see different kind of (inaudible) in both different situations in clinical trials and also situation in what I call more real world. We are following it a lot. We are now open for enrollment in our trial where we are having two arms in our duration of dose titration over 30 up to 60, and we will enroll that in a decent speed.

這個問題我們今天很難回答，因為我們在不同情境下看到不同類型的（聽不清），包括臨床試驗中的情況，以及我所稱更貼近真實世界的情況。我們正在密切追蹤。我們目前已開放一項試驗收案，該試驗有兩個組別，評估將劑量滴定從 30 提升到 60 的不同時程，我們會以不錯的速度完成收案。

We only do that in the US because this is the only place where we restrict it down to 30 and not have 60. We believe that even if you were coming up to 30 micrograms, you still have a major benefit to be still on 30 micrograms compared to many of the positive effects that YORVIPATH is still providing to it. So I think you can say yes, there is someone that would be the benefit to go higher, but today, we're still providing the benefits to the patient that need to stop on searching (inaudible).

我們只在美國做這件事，因為只有美國把上限限制在 30，而沒有 60。我們相信，即使你只能用到 30 微克，相較於許多正向效果，YORVIPATH 仍能提供顯著的益處。因此我想你可以說，是的，確實有人會受益於更高劑量，但就目前而言，我們仍在為患者提供益處，即便他們需要停止繼續尋找（聽不清）。

Joe Schwartz, Leerink Partners.

Joe Schwartz，Leerink Partners。

Great. Thanks so much for taking my question. So some physicians we've spoken with have suggested that they might not want to put their office staff through the reimbursement challenges of switching their achondroplasia patients to a once-weekly injection, only to then later switch them to a once-daily pill in the not too distant future. Is this a dynamic that you guys are aware of? And what can Acendis do to help support the achondroplasia patient or physician community rather and encourage uptake?

很好，謝謝讓我提問。我們接觸的一些醫師表示，他們可能不想讓診所行政人員承受把軟骨發育不全患者切換到每週一次注射所帶來的給付/報銷挑戰，因為不久的將來又可能要再切換到每日一次口服藥。你們是否注意到這種動態？Acendis 能做些什麼來支持軟骨發育不全患者——或更確切地說醫師社群——並鼓勵使用？

And then have any -- my second question is, have any physicians prescribed YUVIWEL in combination with SKYTROFA since YUVIWEL was approved? Thanks.

另外我的第二個問題是：自從 YUVIWEL 獲批以來，是否有醫師將 YUVIWEL 與 SKYTROFA 合併開立處方？謝謝。

So answering your first part of the question, I think the number speaks for itself. When you think about it, more than 10 prescriptions per week, that's just a rare disease product. No, I think it talks about the unmet medical need and the willingness for basic position in connection with the parents, in connection with the child to basic to have the desire to take them on a treatment video of it. I think it says everything but numbers. You can go out and ask one physician. You can go out and ask one parent. I look at numbers from a statistic, but numbers talk for itself.

先回答你問題的第一部分，我認為數字本身就說明了一切。想想看，每週超過 10 張處方，這可是罕見疾病產品。我認為這反映了未被滿足的醫療需求，以及醫師在與家長、與孩子溝通後，願意讓他們接受治療的意願。我覺得除了數字之外，這已經說明了一切。你可以去問任何一位醫師，也可以去問任何一位家長。我從統計角度看數字，但數字會自己說話。

Related to the last question, I cannot, in some way, discuss an element we cannot promote. We cannot promote any way into the combination therapy. We have disclosed the benefit of the combination therapy. And then it's up to the physicians if they really want to prescribe it or not prescribe it.

至於最後那個問題，我在某種程度上無法討論我們不能推廣的內容。我們不能以任何方式推廣合併治療。我們已經揭露了合併治療的效益，接下來就取決於醫師是否真的要開立處方。

And to my knowledge, and I can be pretty open about it, yes, it happens, and I understand why. This is the only way you basically can be in a position where you basically can avoid any kind of surgeries, which I think is such a positive element for any child with an on the patient to avoid this invasive surgeries. And I think this is a reason why the physician do it. Sure, we're looking forward to finalize the Phase III trial. We're looking forward to have it on label. So we really also can go out and promote it.

就我所知，而且我可以很坦白地說，是的，確實有發生，我也理解原因。這基本上是你能夠避免任何手術的唯一方式，而我認為對任何孩子來說，能避免這種侵入性手術都是非常正面的。我想這就是醫師這麼做的原因。當然，我們也期待完成第三期試驗，期待把它寫進標籤，這樣我們也真的可以對外推廣。

Thanks for the insights.

謝謝你的見解。

Ellie Merle, Barclays.

Ellie Merle，Barclays。

This is Jasmine on for Ellie. Thank you for taking our question. Just kind of a follow-up to the last question. For YUVIWEL, can you say how many of those 60 enrollments were new starts versus switches? And more generally, do you think the initial population is going to be more new starts or switches? And what kind of patients do you think are the most likely to initially want to switch? Thank you.

我是 Jasmine 代替 Ellie 發問。謝謝讓我們提問，算是延續上一題。就 YUVIWEL 而言，你能否說明那 60 位收案中，有多少是新開始用藥、多少是從其他治療轉換？更一般來說，你認為初期族群會以新開始為主，還是以轉換為主？以及你認為哪些患者最可能在初期就想要轉換？謝謝。

The insight you're asking for is the insight we will develop in the coming months and quarters. After five weeks to try to come with a general statement about what kind of patient, what kind of performance they have to go on to YUVIWEL treatment, I think it's too early for us to come with a conclusion, but it is such a topic.

你所詢問的洞見，是我們會在接下來幾個月與幾個季度逐步建立的洞見。在僅僅五週之後就要對什麼類型的患者、他們在轉用 YUVIWEL 治療上的表現做出一般性結論，我認為現在對我們來說還太早，但這確實是一個重要議題。

The only thing I can say, and Jay, you can add on, what we see is basic coming from everywhere. It's not just a new patient. It's not just with patients. It's coming everywhere from where we expect it also to come from. And one of the things we have done at the centers that Jay has made and impact on to help the physician, the patient, it's really to have a panel that can go out and really help the physician, the patients everywhere to get through this journey to be sure they can come on the right treatment.

我唯一能說的是，Jay，你也可以補充，我們看到的基本上是從各個地方而來的。這不只是新病人；也不只是既有病人。它是從各處而來，包括我們原本也預期會出現的來源。而我們在 Jay 所建立並產生影響的中心所做的一件事，是為了協助醫師與病人，真正建立一個可以外出支援的團隊，實際去幫助各地的醫師與病人走過這段旅程，確保他們能夠接受正確的治療。

Jay, do you have anything to add?

Jay，你有什麼要補充的嗎？

I think you summed it up well. The only two additional things I would add is, one, to Jan's point, we're seeing across all segments. And we've discussed before the three areas or types of patients that we envision coming are, one, patients currently on VOXZOGO that are switching over; two, patients that were previously on VOXZOGO, but since discontinued and were on no therapy; and then, three, a patient that perhaps had never made the decision to start therapy at all. And we are seeing anecdotally that it's coming across all three of those groups. And I think really what that underscores is the continued and existing unmet need that exists in achondroplasia today, even with the existing therapy on market, which I think emphasizes the value of having YUVIWELL on the market and the compelling value proposition that it offers patients.

我覺得你總結得很好。我另外只想補充兩點：第一，呼應 Jan 的觀點，我們看到的是橫跨所有分群。我們之前也討論過，我們預期會出現的三類病人是：第一，目前正在使用 VOXZOGO、並轉換過來的病人；第二，先前曾使用 VOXZOGO、但後來停用且目前沒有任何治療的病人；第三，可能從未做出開始治療決定的病人。我們從一些軼聞觀察到，這三個族群都有病人進來。我認為這真正凸顯的是：即使市場上已有既有療法，當今軟骨發育不全（achondroplasia）仍存在持續且未被滿足的醫療需求；這也強調了讓 YUVIWELL 上市的價值，以及它為病人提供的具吸引力的價值主張。

I think the second area that Jan was talking about is our continued investment in just making sure that everything we're doing is hand in glove with patients, both as it relates to partnerships with the patient advocacy groups, but also as it relates to our scaling up of our patient access liaison team, which is our patient-facing field group that invests in the support and the journey as they go through the continuum of prescription to ultimately being on therapy.

我想 Jan 提到的第二個面向，是我們持續投入資源，確保我們所做的一切都能與病人緊密配合（hand in glove）。這包括與病友倡議團體的合作夥伴關係，也包括擴編我們的病人可近性聯絡員（patient access liaison）團隊——這是我們面向病人的外勤團隊，會在病人從開立處方到最終開始用藥的整個連續流程中，投入支持並陪伴其治療旅程。

Yun Zhong, Wedbush.

Yun Zhong，Wedbush。

Good morning. Thank you very much for taking the question. My question is on the monotherapy for hypochondroplasia. I remember that there have been some changes in terms of approach for that program, whether you're going to pursue that indication at all, and whether it's going to be mono or combo or maybe just a -- wanted to know, are you able to share any information regarding the thought process behind the decision, if this is the final decision that you are going to just using monotherapy to target hypochondroplasia? Thank you very much.

早安。非常感謝讓我提問。我的問題是關於針對軟骨發育不全類似症（hypochondroplasia）的單藥治療。我記得這個計畫在策略上曾有一些調整，包括你們是否還會追求這個適應症，以及會採單藥或合併治療，或可能只是——我想了解的是，你們是否能分享一下做出這個決定背後的思考過程？以及這是否已是最終決定，也就是你們將僅以單藥治療來鎖定 hypochondroplasia？非常感謝。

I think the strategy that we have applied to achondroplasia where we started with monotherapy and then we moved on to the addition to combination therapy. I think you will see that as there will be enlightenment between the strategic approach that we have used in achondroplasia, we will likely also use in hypochondroplasia. I can basically tell you that we're using the same principle between the two indications.

我認為我們在軟骨發育不全（achondroplasia）所採用的策略——先從單藥治療開始，之後再推進到加入合併治療——你會看到，隨著我們在 achondroplasia 所使用的策略方法逐步更清晰，我們很可能也會在 hypochondroplasia 上採用相同的做法。我基本上可以告訴你，我們在這兩個適應症之間採用的是相同原則。

The two indication is almost aligned in the fundamentals of the disease. There is only, you can say, different mutation, different severity, and other things like that. And we will implement the same thinking in treatment regime between these two indications.

這兩個適應症在疾病的基本面上幾乎是一致的。差別只在於——你可以說——不同的突變、不同的嚴重程度，以及其他類似因素。我們會在這兩個適應症之間，採用相同的治療方案思維。

Luca Issi, RBC Capitals.

Luca Issi，RBC Capitals。

Thanks so much for taking my question. Maybe Jay or Scott, can you educate us on the mechanics of the free drug for your repack? Who are the patients that got the free drug? For how long do they stay on free drug? And are you expecting any patients still on free drug in Q2, or is this just a kind of Q1 phenomenon? Any color there are much appreciated.

非常感謝讓我提問。也許 Jay 或 Scott，可以跟我們說明一下你們針對重新包裝（repack）的免費用藥機制？哪些病人拿到了免費藥？他們會使用免費藥多久？以及你們是否預期在第二季仍會有病人使用免費藥，還是這只是第一季的現象？若能提供一些補充說明將非常感謝。

And super quickly, I think AstraZeneca's presenting their Phase III data for [ENEBO] this weekend, the European Congress on Endocrinology. So wondering if you can comment on what are your expectations for that data? Thanks so much.

另外很快補充一下，我想阿斯特捷利康（AstraZeneca）這週末會在歐洲內分泌學大會（European Congress on Endocrinology）發表 [ENEBO] 的第三期數據。想請問你們能否評論一下對該數據的預期？非常感謝。

I can some way start from the last question and then I can move it up and then Jay can take over in the end. The compound we are talking about in the end is the (inaudible) compound. And we have discussed that on many earnings call the lack of information that was related to data. Now that is disclosed some kind of information of the data packet one year after finalization of the Phase III trial.

我可以先從最後一個問題開始回答，然後再往前，最後再由 Jay 接手。我們最後談到的那個化合物是（聽不清）。我們在多次財報電話會議中都討論過，當時與數據相關的資訊不足。現在在第三期試驗完成一年後，對方披露了某種程度的數據資料包資訊。

And for me, and I think the key question through this data packet provide an approvability of any weight of this compound. And to my best judgment, I hope this product never would be approved, and I don't see really as possibility that it should be and going to be approved. So I think when we look on the competitive landscape in for treatment in (inaudible), I see (inaudible), our once-weekly approach to stable patient on (inaudible) is really providing the fundamentals for a 20, 30 years treatment regime where I don't see anything that really can live up to the benefit we will see in the treatment with (inaudible) or any other product that is currently in clinical development from that perspective.

對我而言，我認為關鍵問題是：這份數據資料包是否能提供該化合物在任何程度上的可核准性（approvability）。以我最佳判斷，我希望這個產品永遠不會獲批，而且我也不認為它有可能、或應該、或將會被核准。因此，當我們看（聽不清）的治療競爭格局時，我認為（聽不清），我們每週一次（once-weekly）的方式，針對（聽不清）上病情穩定的病人，確實提供了可支撐 20、30 年治療方案的基本面；從這個角度，我看不到有任何東西能真正達到我們在（聽不清）治療中將看到的效益，或任何其他目前仍在臨床開發中的產品能與之匹敵。

Related to the first part of your question, you're right, we started to take (inaudible) the simple group of patients over to free drug because the essential part of your past is that you cannot stop. If you first have started taking your work to basically the element on what we call the conventional therapy, it's basically a disaster for the patient.

至於你問題的第一部分，你說得對，我們開始把（聽不清）這一小群病人轉到免費用藥，因為你過去的核心要點是：你不能中斷。一旦你已經開始使用——基本上是我們所謂的傳統治療（conventional therapy）——若中途停止，對病人而言基本上是災難。

So if there was a hiccup in this reimbursement with that was a hiccup, and it's something we had done corrective action to ensure that we can handle it much better next year, we were in a position that we took. And Scott explained the impact of that here in Q1 to take already from December until March a series of patients on free drug, and they are now coming back to be fully reimbursed. And we are in a position that Jay and the organization in the US, some way have built up network or the things that can help it that we not ending in the same situation one time more.

所以如果在這個給付（reimbursement）上出現了卡關——確實有卡關——我們已採取矯正措施，確保明年能更好地處理。我們當時所處的情況使我們採取了這個做法。Scott 也在這裡說明了第一季的影響：我們從 12 月到 3 月，讓一系列病人先使用免費藥作為銜接，而他們現在正回到可獲得全額給付的狀態。我們也已經到了一個階段：Jay 與美國團隊某種程度上建立了網絡與相關機制，能協助我們避免再次落入同樣的情況。

Jay, do you have any comments too?

Jay，你也有什麼評論嗎？

Again, I think you summarized the need for bridge program well. I think just to clarify the earlier question, there is two types of free programs. We have bridge program, which again is pretty standard across the industry for those that have experienced a temporary insurance lapse. But we also just have our patient assistance program for patients that are underinsured or uninsured. So I think that's an important point to note because there will always be some patients that qualify for the patient assistance programs, so we don't anticipate that will ever go away completely, knowing that there will always be a certain level of patients that qualify for that.

再次強調，我認為你已經很好地總結了銜接計畫（bridge program）的必要性。我想釐清一下前面那個問題：其實有兩種免費用藥計畫。我們有銜接計畫，這在產業中相當常見，主要針對遭遇短期保險中斷的病人。但我們也有病人協助計畫（patient assistance program），提供給保險不足或沒有保險的病人。我認為這點很重要，因為總會有一些病人符合病人協助計畫的資格，所以我們不預期這類情況會完全消失；我們知道總會有一定比例的病人符合該計畫。

I think that is a clarification that's great from Jay, where we talk about this number of patients is only what we exceeded as access compared to the base level of patient. We always will help and provide free drug if there is an element of something where there is a disruption of the normal way to have drug.

我覺得 Jay 的這個釐清非常好：我們談到的這個病人人數，只是相對於基礎病人水位而言、在可近性（access）上超出的那一部分。我們一向都會在正常取得藥物的流程出現中斷等情況時，提供協助並提供免費藥。

It's just an essential drug for the patients that we always will take our patient first. And if there's a patient that gets disrupted, we will do everything to help this patient. And that includes also to take them for a period of free drugs until the disruption is getting solved. And we will always be there for the patients.

這對病人而言是不可或缺的藥物，我們永遠會把病人放在第一位。如果有病人的用藥被中斷，我們會盡一切努力協助該病人，其中也包括在中斷問題解決之前，提供一段期間的免費藥物。我們會一直與病人站在一起。

Thanks so much.

非常感謝。

Maxwell Skor, Morgan Stanley.

Maxwell Skor，Morgan Stanley。

This is (inaudible) on for Max. Thanks for taking your question. Given the relatively low treatment penetration and achondroplasia in the US to date, what proportion of patients typically initiate treatment at age 2 years or older?

我是（聽不清），代替 Max 提問。感謝讓我提問。鑑於目前在美國軟骨發育不全（achondroplasia）的治療滲透率仍相對偏低，通常有多少比例的病人會在 2 歲或以上開始治療？

I think some way to roll it a little bit back because you can ask a question, why do you have undertreatment in the US? And I think actually this is the key question to find out how can we really help this patient better. And I think and we believe that the undertreatment is facing the cause of lack of the right efficacy to show real benefit beyond linear growth.

我想可以用某種方式把它稍微往回推一點，因為你可以問一個問題：為什麼美國會出現治療不足？而我認為這其實是找出我們如何才能真正更好幫助這位病患的關鍵問題。我們認為，治療不足的原因在於缺乏足夠的療效，無法顯示出超越線性生長的真正臨床效益。

Many of these parents children don't see linear growth as a key element. They really want us to address all the comorbidity specific if you can avoid surgeries for changing the pain, complication with leg bone, you can avoid arm linking, leg linking. And I believe by having this focus on these elements, and here I talk about the older children, if you go to a younger newborn, yes, if we can avoid any kind of spinal stenosis by having early intervention from newborn, yes, there will be a major benefit for imitate treatment in the newborn state. So I believe our product profile that we have generated with you were showing as the only product the benefit billing compared to placebo.

許多這些家長與孩子並不把線性生長視為關鍵要素。他們真正希望我們處理的是所有共病，特別是如果你能避免手術、避免因疼痛而做的處置、避免腿骨相關併發症，你就能避免手臂延長、腿部延長。我相信，聚焦在這些要素上——這裡我談的是年紀較大的孩子——如果你看更年幼的新生兒，是的，如果我們能從新生兒期就及早介入以避免任何形式的脊椎管狹窄，那麼在新生兒階段立即治療將帶來重大效益。因此，我相信我們與你們共同建立的產品特性（product profile）顯示，作為唯一的產品，相較於安慰劑具有可計費的效益。

It need always to be placebo-controlled because there is a development to the side, so you cannot say, oh, I'll be also improved span or arm thing just enough. And childhood achondroplasia actually have a big increase in arm length. So you always need to really show it, compare into a placebo-controlled trial. It's the only way you can really adjust the benefit of the medical treatment needed.

它始終需要安慰劑對照，因為發育本身也在進行，所以你不能說「喔，我的身高或手臂也會改善」就算足夠。而兒童軟骨發育不全其實手臂長度會大幅增加。因此你必須真正展示出來，並在安慰劑對照試驗中比較。這是你能真正校準所需醫療治療效益的唯一方式。

So the question and the answer to you is, I believe YUVIWELL will be an appealing product to the vast majority of parents, children in the US also because they provide a way to address the comorbidities, like important one, leg building, arm strength, everything that you basically will see benefit for not only quality of life that's associated with it, but also the element of physical strength.

所以對你的問題，我的回答是：我相信 YUVIWELL 對美國絕大多數家長與孩子都會是很有吸引力的產品，因為它提供了一種處理共病的方法，例如重要的腿部構建、手臂力量等——基本上你會看到的不僅是與之相關的生活品質改善，也包括體能強度這個面向的效益。

Paul Thomas, Goldman Sachs.

Paul Thomas，高盛。

Thank you and good morning and congrats on a good start with YUVIWELL. I was just wondering if you could clarify in terms of the more than 60 prescriptions you've seen to date, whether it's more driven by treatment-naive patients or switches, any quantification there would be great. And I'm also curious, in terms of your early starts or through the quarter, if you're seeing potential utilization in the below 2 years of age population, even though that's not officially on label yet. Thank you.

謝謝，早安，也恭喜 YUVIWELL 有個好的開始。我想請教，關於你們迄今看到的超過 60 張處方，能否釐清主要是由未曾治療（treatment-naive）的病患帶動，還是由轉換用藥（switch）帶動？如果能量化會很有幫助。另外我也好奇，就你們在本季初期或整個季度的觀察，是否看到 2 歲以下族群的潛在使用，即使那目前還不是正式核准適應症標籤。謝謝。

We need to come up with a meaningful answer. We need longer time because we only have been there for four or five weeks now, and we want to be sure that what we see in the initial part of the lungs is also being representative of what we see in the later state of lungs. So Paul, we can discuss it, as Jay said, in a perfect manner with the patient coming in for all three different groups, which was a new group, patients that have discontinued and patients that come directly on switching for worse overtime.

我們需要提出一個有意義的回答。我們需要更長的時間，因為我們目前才進行了四到五週，我們希望確保在初期看到的情況，也能代表在後期看到的情況。所以 Paul，我們可以討論，如 Jay 所說，以一個完善的方式來看病患進來的三種不同族群：新族群、已中斷治療的病患，以及直接因為長期效果變差而轉換用藥的病患。

So often that we see it coming in for all three different groups in the initial launch, we will expect to see Perhaps one mix and maybe come a little bit longer into the lungs, we will potentially see a switch in the different three classes. And this is why, really to make it meaningful, we need to wait longer time before we can give you something you can do the right modeling on. But when I look at this number, we're coming up more than 10 patients per week, it's just an orphan drug, the indication, I'm extremely proud that our product profile is getting so well recognized in the society in this way.

我們在初期上市時，確實看到三種不同族群都會進來；我們預期可能會出現某種組合，而隨著上市時間拉長，我們可能會看到這三類之間的占比發生變化。也因此，為了讓它真正有意義，我們需要等待更長時間，才能給你一個你可以用來做正確模型推估的數據。不過當我看到這個數字——每週超過 10 位病患——考量到這是孤兒藥、且是該適應症，我對我們的產品特性以這種方式在社群中獲得高度認可感到非常自豪。

Under two, I cannot comment on that currently.

兩歲以下的部分，我目前無法評論。

Okay. Thank you.

好的，謝謝。

Good morning, and thanks for taking the questions. For the 70% cumulative US insurance approval rate that you previously cited. Can you give us any more color on what that cumulative rate looks like today? And then second, the EUR500 million operating cash flow target that you laid out previously, are you reaffirming that guidance given the Q1 trends that you're seeing? And how should we think about the contribution of YUVIWELL to that target? Thank you.

早安，謝謝讓我提問。關於你們先前提到的美國保險累計核准率 70%，能否再多提供一些目前這個累計比率的情況？第二，先前提出的 5 億歐元（EUR500 million）營運現金流目標，基於你們看到的第一季趨勢，是否重申該指引？以及我們應該如何看待 YUVIWELL 對該目標的貢獻？謝謝。

The last one, we would like to come back in Q2 because we have a lot of positive element coming in now. We have the selling of our PRV, DCG, and logs of you were going much stronger than the I even myself had it over. So aside from that, we will come with that change guidance, but we will prefer to do it after our Q2 call, and we will give you what will be reflected on the 26th.

最後那一題，我們希望在第二季再回覆，因為現在有很多正面因素正在出現。我們出售了 PRV、DCG，而且你們的（訂單/趨勢）走勢比我自己原先預期的還要強勁。因此，除此之外，我們會帶來更新後的指引，但我們傾向在第二季電話會議之後再做，並在 26 日反映給你們。

Scott is saying yes to me. And Jay, you can give us the factual number how it's improving the overall numbers.

Scott 正在對我說可以。而 Jay，你可以給我們一些事實數字，說明它如何改善整體數據。

Sure. So the overall cumulative approval rate since launch has continued to creep up as well. I think we're now closer to mid-70%, which again is incredibly high for any rare disease asset, much less one that has been on the market for the amount of time that we have. A lot of that is just a function of time, given the favorable access policies that we have. So even some enrollments that might be many months old are coming through on appeal online, which would affect the cumulative approval rate over time. But given that it is a lagging indicator, it will take quite a bit of time for that metric to mature.

好的。自上市以來，整體累計核准率也持續小幅上升。我想我們現在更接近 70% 中段（mid-70%），這對任何罕見疾病資產而言都高得驚人，更不用說我們上市的時間其實還不長。這在很大程度上只是時間因素所致，因為我們擁有有利的給付准入政策。因此，即便有些可能是數月前的申請，也會在申覆（appeal）後陸續通過，進而隨時間推移影響累計核准率。不過由於這是一個落後指標，它需要相當長的時間才能成熟。

Thank you.

謝謝。

(inaudible)

（聽不清）

Thanks for taking our questions. This is (inaudible) on for (inaudible). So given the revenue now for three commercial products, the redemption of the convertible notes and the sales of the PRP, can you spell anything on your capital application strategy? What is the order of priority for you guys?

謝謝回答我們的問題。我是（聽不清），代表（聽不清）提問。鑑於你們現在有三項商業化產品的營收、可轉換公司債的贖回，以及 PRP 的出售，你們能否談談資本配置策略？你們的優先順序是什麼？

I think Scott got the one minute to answer that last question for today. So Scott, get this opportunity now.

我想 Scott 今天有一分鐘來回答最後這個問題。Scott，現在給你這個機會。

Thanks a lot, Jan. Of course, as you've seen with our R&D success, a key component for us is to invest in R&D and allocate capital there to continue to sustain not only into the 2030s, but the 40s and beyond with the continuous flow of new products. I think Jan highlighted or new NCEs in his prepared remarks, and you'll learn about more of those in the coming future. And of course, I think after we give an update after Q2, as Jan mentioned, to our outlook for the rest of the year, you may get more color at that point as well.

非常感謝，Jan。當然，正如你們從我們的研發成功所看到的，對我們而言一個關鍵組成就是投資研發並將資本配置到那裡，以持續支撐我們不僅走到 2030 年代，還能延伸到 2040 年代及更遠，並持續推出新產品。我想 Jan 在事先準備的發言中也提到我們新的 NCE（新化學實體），你們在未來也會了解更多。當然，正如 Jan 提到的，在我們於第二季後更新今年其餘時間的展望之後，你們屆時也可能會得到更多細節。

Thank you. That's all the time we have for questions. Thank you for your participation in today's conference. This concludes the program. You may now disconnect.

謝謝。由於時間關係，今天的提問到此為止。感謝各位參與今天的會議。會議到此結束。各位現在可以斷線。