Mirum Pharmaceuticals, Inc. (MIRM) 2026 Q1 法說會逐字稿

Good afternoon, and welcome to Mirum Pharmaceuticals first quarter 2026 earnings conference call. My name is Tracy, and I will be your operator today. (Operator Instructions) I would now like to hand the call -- the conference over to Andrew McKibben, SVP of Strategic Finance and Investor Relations. Please go ahead.

下午好，歡迎參加 Mirum Pharmaceuticals 2026 年第一季財報電話會議。我是 Tracy，今天將擔任會議接線員。(接線員指示) 現在我想把電話會議交給策略財務與投資人關係資深副總裁 Andrew McKibben。請開始。

Thank you, Tracy, and good afternoon, everyone. I'd like to welcome you to Mirum Pharmaceuticals First Quarter 2026 Earnings Conference Call. For our prepared remarks, I'm joined today by our Chief Executive Officer, Chris Peetz; our President and Chief Operating Officer, Peter Radovich; and Eric Bjerkholt, our Chief Financial Officer. Our Chief Medical Officer, Joanne Quan, will be joining us for Q&A. Earlier this afternoon, Mirum issued a press release reporting our first quarter 2026 financial results.

謝謝你，Tracy，各位下午好。歡迎各位參加 Mirum Pharmaceuticals 2026 年第一季財報電話會議。在我們的事先準備發言中，今天與我一同出席的有：執行長 Chris Peetz；總裁兼營運長 Peter Radovich；以及財務長 Eric Bjerkholt。我們的醫務長 Joanne Quan 將在問答環節加入。今天下午稍早，Mirum 已發布新聞稿，公布我們 2026 年第一季財務結果。

Copies of the press release and our SEC filings are available on the Investors section of our website. Before we start, I'd like to remind you that during the course of this conference call, we will be making certain forward-looking statements based on management's current expectations, including statements regarding Mirum's programs and market opportunities for its approved medicines and product candidates and financial guidance. These statements represent our judgment and knowledge of events as of today and inherently involve risks and uncertainties that may cause actual results to differ materially from the results discussed. We are under no duty to update these statements. Please refer to the risk factors in our latest Form 10-Q and subsequent SEC filings for more information about these risks and uncertainties.

新聞稿與我們向 SEC 提交的文件副本，可於本公司網站「投資人」專區取得。在開始之前，我想提醒各位，在本次電話會議過程中，我們將根據管理層目前的預期發表若干前瞻性陳述，包括關於 Mirum 的研發計畫、已核准藥品與產品候選藥物的市場機會，以及財務指引等陳述。這些陳述代表我們截至今日對相關事件的判斷與認知，且本質上涉及風險與不確定性，可能導致實際結果與所討論結果出現重大差異。我們沒有義務更新這些陳述。有關上述風險與不確定性的更多資訊，請參閱我們最新的 Form 10-Q 以及後續向 SEC 提交文件中的風險因素。

With that said, I'd like to turn the call over to Chris. Chris?

話不多說，我想把電話交給 Chris。Chris？

Thanks, Andrew, and good afternoon, everyone. We have a number of important updates to cover today, but I'd like to start by grounding in the vision we set when we founded Mirum in 2018. building a company focused on bringing forward medicines for overlooked rare diseases. This quarter reflects the progress we've made in turning that vision into a durable growing business. Our start was based on LIVMARLI.

謝謝你，Andrew，各位下午好。今天我們有多項重要更新要分享，但我想先回到我們在 2018 年創立 Mirum 時所設定的願景：打造一家專注於為被忽視的罕見疾病帶來新藥的公司。本季反映了我們在將該願景轉化為一個可持續成長的業務方面所取得的進展。我們的起步是以 LIVMARLI 為基礎。

And today, we are a broader rare disease company with three approved medicines and a pipeline positioned to deliver multiple new therapies over the next two years. These high-impact programs are grouped across two focus areas; rare liver disease, where we have built clear leadership and rare genetic disease, where we are establishing a second growth platform, each with distinct commercial capabilities. Across both, we've built a financially self-sustaining business that can support continued investment in the portfolio. Our strategy is driving compelling results. Starting with rare liver disease, uptake of LIVMARLI remains strong, driven in part by performance in PFIC, which continues to exceed expectations.

而今天，我們已成為一家更廣泛的罕見疾病公司，擁有三項已核准藥品，以及一條有望在未來兩年帶來多項新療法的研發管線。這些高影響力計畫分屬兩大重點領域：罕見肝病（我們已建立明確領導地位）以及罕見遺傳疾病（我們正建立第二個成長平台），兩者各自具備不同的商業化能力。在這兩個領域，我們已建立一個財務上可自我支撐的業務，能支持對產品組合的持續投資。我們的策略正帶來令人信服的成果。以罕見肝病為例，LIVMARLI 的採用仍然強勁，部分動能來自 PFIC 的表現，且持續超出預期。

Based on that demand and continued performance across all brands, we are raising our full year revenue guidance to $660 million to $680 million. More importantly, we are now seeing the next phase of our rare liver disease business take shape. Our recent clinical readouts in PSC and hepatitis delta represent important potential expansions for this business, extending beyond our pediatric foundation into larger patient populations with significant unmet need. In PSC, the VISTAS study of volixibat showed a significant improvement in pruritus, reinforcing the potential for volixibat to play an important role for these patients who currently have no approved medicines. This is a major advance in PSC research and positions volixibat as a potential first approved medicine for patients in the US.

基於該需求以及各品牌的持續表現，我們將全年營收指引上調至 6.60 億至 6.80 億美元。更重要的是，我們現在正看到罕見肝病業務的下一階段逐步成形。我們近期在 PSC 與丁型肝炎（hepatitis delta）的臨床讀出，代表此業務潛在的重要擴張，將我們的基礎從兒科延伸至具有重大未滿足需求、規模更大的病患族群。在 PSC 方面，volixibat 的 VISTAS 研究顯示搔癢（pruritus）有顯著改善，強化了 volixibat 對目前尚無核准藥物可用之患者的重要潛力。這是 PSC 研究的一大進展，並使 volixibat 成為美國患者潛在的首個核准藥物。

And in hepatitis delta, results from the Phase IIb portion of the AZURE 1 study further support the potential for brelovitug in a patient population where treatment options are extremely limited. We look forward to the upcoming late-breaking presentations for both VISTAS and AZURE at EASL later this month. Now in parallel to this expansion of our rare liver disease business, today, we are announcing another step in building out our rare genetic disease business with the addition of zilurgisertib recently licensed from Incyte. Zilurgisertib is a once-daily oral ALK2 inhibitor in development for fibrodysplasia ossificans progressiva or FOP, an ultra-rare progressive condition where patients develop bone and soft tissues. This accumulation of excess bone leads to profound physical immobilization with most FOP patients becoming wheelchair dependent by early adulthood and severely impact life expectancy.

而在丁型肝炎方面，AZURE 1 研究第二期 b（Phase IIb）部分的結果，進一步支持 brelovitug 在治療選項極為有限的病患族群中的潛力。我們期待本月稍晚在 EASL 上，VISTAS 與 AZURE 兩項研究的最新突破性發表。在擴張罕見肝病業務的同時，我們今天也宣布，透過新增自 Incyte 近期授權引進的 zilurgisertib，進一步建構我們的罕見遺傳疾病業務。Zilurgisertib 是一款每日一次口服的 ALK2 抑制劑，正在開發用於進行性纖維性骨化症（fibrodysplasia ossificans progressiva，簡稱 FOP），這是一種超罕見的進行性疾病，患者會在骨骼與軟組織中形成骨化。這種過量骨生成會導致嚴重的身體失能，多數 FOP 患者在成年早期即需依賴輪椅，並嚴重影響預期壽命。

Based on the strength of zilurgisertib's PROGRESS study conducted by Incyte, an NDA has been accepted for priority review with a PDUFA date of September 26 this year. If approved, we expect to launch by year-end. This is a strong strategic fit, aligning with our capabilities in rare genetic disease, where care is concentrated in a small number of specialized centers and requires deep engagement with patients, caregivers and physicians. Stepping back, we built a company with multiple commercial growth drivers, a pipeline of meaningful upcoming catalysts and the financial strength to advance our portfolio independently. This foundation is translating directly into high-impact medicines for patients and into value creation as we deliver on our strategy.

基於 Incyte 所進行之 zilurgisertib PROGRESS 研究的強勁結果，NDA 已獲受理並進入優先審查，PDUFA 日期為今年 9 月 26 日。若獲核准，我們預期將於年底前上市。這在策略上高度契合，與我們在罕見遺傳疾病領域的能力一致；該領域的照護集中於少數專科中心，並需要與患者、照護者及醫師進行深度互動。回過頭來看，我們打造了一家公司，具備多元的商業成長驅動因素、具意義的近期研發催化劑管線，以及能夠獨立推進產品組合的財務實力。這一基礎正直接轉化為對患者具有高影響力的藥物，並在我們落實策略的過程中創造價值。

And with that, I'll turn the call over to Peter to walk through the commercial portfolio and preparation for the three upcoming potential launches. Peter?

接下來，我把電話交給 Peter，請他說明商業化產品組合，以及為三項即將到來的潛在上市所做的準備。Peter？

Thank you, Chris. The first quarter was another period of strong commercial execution with total net product sales of approximately $160 million. This included LIVMARLI net product sales of $84 million in the US and $30 million internationally, with the bile acid medicines contributing $46 million. Robust adoption in PFIC, particularly in adult patients, continues to be a strong point for LIVMARLI as education to increase awareness and recognition of genetic cholestasis among adult liver providers continues to be successful.

謝謝你，Chris。第一季我們再次展現強勁的商業執行力，產品淨銷售額合計約 1.60 億美元。其中包括 LIVMARLI 在美國的產品淨銷售額 8,400 萬美元、國際市場 3,000 萬美元；膽酸類藥物（bile acid medicines）貢獻 4,600 萬美元。PFIC 的強勁採用，尤其是在成人患者方面，持續是 LIVMARLI 的亮點；我們針對成人肝臟照護提供者所進行的教育推廣，提升對遺傳性膽汁鬱積（genetic cholestasis）的認知與辨識，仍持續奏效。

Additionally, we saw stronger-than-expected performance in Q1 international LIVMARLI sales as well as continued new patient adds in Alagille worldwide. The bile acid medicines grew in a manner consistent with their cadence over the last several quarters, highlighted by our rare genetics team continuing to identify undiagnosed patients with CTX.

此外，我們也看到第一季國際 LIVMARLI 銷售表現優於預期，並且在全球 Alagille 症候群方面持續新增新患者。膽酸類藥物的成長與過去數季的節奏一致，其中一項重點是我們的罕見遺傳疾病團隊持續找出尚未確診的 CTX 患者。

Overall, we expect these dynamics to continue and as a result, are raising our full year 2026 net product sales guidance to $660 million to $680 million. And as Chris mentioned, we are also beginning to see the next phase of growth in our rare liver disease business take shape.

整體而言，我們預期這些動能將持續，因此將 2026 年全年產品淨銷售額指引上調至 6.60 億至 6.80 億美元。同時，如同 Chris 所提到的，我們也開始看到罕見肝病業務的下一階段成長逐步成形。

The recent results from the VISTAS study of volixibat in PSC and the AZURE-1 study of brelovitug in hepatitis delta represent important steps in extending our presence into larger, primarily adult liver settings, where patients have limited or no approved treatment options. These programs build directly on the global commercialization platform we have established for LIVMARLI, CTEXLI and CHOLBAM, heavily leveraging our existing technologies, people and infrastructure.

近期 volixibat 用於 PSC 的 VISTAS 研究結果，以及 brelovitug 用於丁型肝炎的 AZURE-1 研究結果，代表我們將版圖延伸至更大、以成人肝病為主的照護場域的重要一步；在這些場域中，患者可用的核准治療選項有限或甚至沒有。這些計畫直接建立在我們為 LIVMARLI、CTEXLI 與 CHOLBAM 所打造的全球商業化平台之上，並大量運用我們既有的技術、人員與基礎設施。

We plan to expand our US and international teams starting later this year to reach liver health care providers in adult settings, including GI liver providers, who manage PSC patients and hepatitis delta as well as other care settings like infectious disease and selected primary care providers, where we believe we can increase the number of diagnosed hepatitis delta patients.

我們計畫自今年稍晚起擴編美國與國際團隊，以觸及成人場域的肝臟醫療照護提供者，包括腸胃科/肝臟科（GI liver）醫師，他們負責管理 PSC 患者與丁型肝炎；同時也涵蓋感染科以及部分基層照護提供者等其他照護場域，我們相信可在這些場域提升丁型肝炎患者的確診人數。

In the US, our current 20-person liver field commercial team reaches about 1,500 health care providers with currently focus on pediatric liver providers and some higher volume adult providers. After our planned expansion to approximately 60 US field commercial personnel, we anticipate being able to reach over 4,000 liver health care professionals, representing the vast majority of potential prescribers for our rare liver business. Turning to our rare genetic disease business, we are very excited by the addition of zilurgisertib for the treatment of FOP, where there remains a desperate need for additional treatment options.

在美國，我們目前由20人組成的肝臟領域商業外勤團隊可觸及約1,500名醫療照護提供者，目前重點聚焦於小兒肝臟專科醫師以及部分高量成人醫療提供者。在我們計畫將美國商業外勤人員擴編至約60人後，我們預期將能觸及超過4,000名肝臟醫療專業人士，涵蓋我們罕見肝病業務中絕大多數的潛在開立處方者。談到我們的罕見遺傳疾病業務，我們對於新增 zilurgisertib 用於治療 FOP 感到非常振奮，因為該領域仍迫切需要更多治療選項。

FOP is a devastating relentlessly progressive condition in which soft tissues such as muscles, tendons and ligaments gradually turn into a second skeleton, leading to cumulative loss of mobility, severe disability in early childhood. FOP is a highly concentrated ultra-rare disease with an estimated prevalence of about 1 per million, which translates to approximately 300 patients in the United States and around 900 patients globally. Patients with FOP are largely managed by specialized tertiary centers, with most of these centers also managing patients receiving CTEXLI and CHOLBAM, allowing us to leverage our rare genetic disease team to commercialize zilurgisertib. And we've been impressed by the zilurgisertib data from the pivotal PROGRES study, in particular, the secondary endpoints, which form the basis of the NDA application, and we look forward to the presentation of the data from the PROGRESS study at an upcoming medical meeting. The NDA for zilurgisertib has been accepted for priority review by the FDA with a September 2026 PDUFA date.

FOP 是一種具毀滅性且無情進展的疾病，肌肉、肌腱與韌帶等軟組織會逐漸轉變成第二副骨骼，導致行動能力逐步喪失，並在幼年早期即造成嚴重失能。FOP 是高度集中的超罕見疾病，估計盛行率約為每百萬人1例，換算約為美國300名患者、全球約900名患者。FOP 患者多由專科的第三級醫療中心管理，而其中多數中心也同時照護使用 CTEXLI 與 CHOLBAM 的患者，使我們能運用罕見遺傳疾病團隊來推動 zilurgisertib 的商業化。我們也對關鍵性 PROGRES 研究的 zilurgisertib 數據印象深刻，特別是作為 NDA 申請基礎的次要終點；並期待在即將到來的醫學會議上發表 PROGRESS 研究的數據。zilurgisertib 的 NDA 已獲 FDA 受理並納入優先審查，PDUFA 目標日期為 2026 年 9 月。

If approved, we expect to launch zilurgisertib in the United States by the end of this year with expansion to additional countries starting in 2027. We are excited about the addition of zilurgisertib, which we believe has the potential to become the cornerstone of our rare genetic disease business and reach peak sales of over $200 million. This high-impact medicine fits perfectly with our medical and commercial team's capabilities and passion for supporting patients suffering from ultra-rare diseases like FOP. And with that, I'll turn it over to Eric to review our financial results. Eric?

若獲核准，我們預期將於今年年底前在美國推出 zilurgisertib，並自 2027 年起擴展至其他國家。我們對於新增 zilurgisertib 感到振奮；我們相信其有潛力成為我們罕見遺傳疾病業務的基石，並達到超過2億美元的峰值銷售額。這款高影響力藥物與我們醫療與商業團隊的能力及對支持 FOP 等超罕見疾病患者的熱忱高度契合。接下來我把時間交給 Eric，請他回顧我們的財務結果。Eric？

(technical difficulty) Here are my ideas and disciplined investment behind our pipeline, which remains on track across all programs. Today, I'll walk you through the financials for the quarter, including an overview of the impact of the Bluejay acquisition and the zilurgisertib transaction. The net product sales for the first quarter were $160 million compared to net product sales of $112 million in the first quarter of last year. Cash, cash equivalents and investments as of March 31 were $421 million compared with $391 million at the beginning of the year. In the first quarter, the cash contribution margin from our commercial business was in the mid-50s percent and cash flow from operations was about $2 million.

（技術問題）以下是我對於我們研發管線的想法，以及我們在其背後所做的紀律性投資；目前所有專案仍按計畫推進。今天我將帶各位檢視本季財務表現，包括 Bluejay 併購以及 zilurgisertib 交易影響的概覽。第一季產品淨銷售額為1.60億美元，去年同期為1.12億美元。截至3月31日，現金、約當現金與投資為4.21億美元，較年初的3.91億美元增加。第一季我們商業業務的現金貢獻毛利率約在50%中段，營運活動現金流約為200萬美元。

First quarter financials were significantly impacted by onetime expenses related to the acquisition of Bluejay Therapeutics, which closed in January of this year. The total net cash outflow related to this acquisition was $253 million, which was offset through net financing proceeds of approximately $260 million. Total operating expense for the quarter ended March 31st was $949 million, which includes $761 million in expense associated with the acquisition of Bluejay, R&D expense of $98 million, SG&A expense of $96 million and cost of sales of $29 million. Expenses for the quarter included stock-based compensation, intangible amortization and other noncash expenses of $64 million, including $35 million of stock-based compensation expense associated with the acquisition of Bluejay. The intangible amortization and other noncash item expenses are largely reflected in our [ company's ] results.

第一季財務結果受到與 Bluejay Therapeutics 併購相關的一次性費用顯著影響；該併購已於今年1月完成交割。與此併購相關的淨現金流出總額為2.53億美元，並透過約2.60億美元的淨融資所得予以抵銷。截至3月31日止季度的營運費用總額為9.49億美元，其中包含與 Bluejay 併購相關費用7.61億美元、研發費用9,800萬美元、銷售與一般管理費用9,600萬美元，以及銷貨成本2,900萬美元。本季費用包含以股份為基礎之酬勞、無形資產攤銷及其他非現金費用共6,400萬美元，其中包括與 Bluejay 併購相關的以股份為基礎之酬勞費用3,500萬美元。無形資產攤銷及其他非現金項目費用主要反映在我們[公司]的業績中。

As Chris and Peter mentioned, we recently entered into an exclusive license agreement with Incyte. In return for worldwide rights to zilurgisertib, Incyte received an upfront payment of $16 million and is eligible to receive additional development and regulatory milestone payments, including $25 million upon US FDA approval for FOP ownership of a rare pediatric disease priority review voucher, if awarded, as well as sales-based milestones and tiered royalties on worldwide net sales in the mid to high single-digit percent range. As we've discussed previously, we expect R&D expense to step up in 2026 as we invest behind brelovitug ahead of the anticipated BLA submission next year. For example, R&D expense in the first quarter included $21 million related to the development of brelovitug.

如 Chris 與 Peter 所提到，我們近期與 Incyte 簽訂了獨家授權協議。作為取得 zilurgisertib 全球權利的對價，Incyte 收到1,600萬美元的預付款，並有資格獲得額外的開發與法規里程碑款項，包括：若 FOP 於美國獲 FDA 核准且（如獲授予）取得罕見小兒疾病優先審查憑證之所有權時，支付2,500萬美元；此外亦包含以銷售為基礎的里程碑款項，以及按全球淨銷售額計算、介於中至高個位數百分比區間的分級權利金。如先前所述，隨著我們在預期明年提交 BLA 之前加大對 brelovitug 的投入，我們預期研發費用將於 2026 年上升。例如，第一季研發費用中包含與 brelovitug 開發相關的2,100萬美元。

Importantly, this expense expected increase is fully funded. We are continuing to scale the business with disciplined, balancing investment in growth with a strong balance sheet and financial independence. This approach positions us to advance our pipeline and execute on upcoming milestones without compromising our long-term financial strength. I'll now turn the call back to Chris for closing remarks.

重要的是，這項預期的費用增加已獲得充分資金支持。我們將持續以紀律性方式擴大業務規模，在投資成長與維持強健資產負債表及財務自主性之間取得平衡。此一策略使我們能在不削弱長期財務實力的前提下，推進研發管線並執行即將到來的里程碑。接下來我把電話交回給 Chris 作結語。

Thanks, Eric. Mirum is in a strong position after a very busy start of the year. What is most encouraging about the quarter is not just a number of positive updates, but how clearly they fit together. We continue to grow our commercial medicines. We're expanding our rare business into rare liver business into larger indications.

謝謝你，Eric。在今年年初非常忙碌的開局之後，Mirum 目前處於強勁的位置。本季最令人鼓舞的不僅是多項正面更新本身，更在於它們彼此之間的契合度非常清晰。我們持續推動商業化藥品成長。我們正將罕見疾病業務中的罕見肝病業務擴展至更大的適應症。

We've added what we believe is a transformational medicine to our rare genetic business. And importantly, this is all coming together within a high-impact scalable business model. We're excited about the progress ahead as we approach multiple pivotal readouts, potential regulatory submissions and potential new product launches. I'd also like to thank the entire Mirum team for all the hard work in getting us where we are today. Your dedication brings new treatment options to patients around the world.

我們也為罕見遺傳疾病業務新增了一款我們認為具有變革性的藥物。更重要的是，這一切正整合於一個高影響力、可擴展的商業模式之中。隨著我們接近多項關鍵性讀出、潛在法規申請以及潛在新產品上市，我們對未來進展感到振奮。我也要感謝整個 Mirum 團隊，感謝大家的辛勤付出，讓我們走到今天。各位的投入，為全球患者帶來新的治療選擇。

And with that, operator, please open the call for questions.

接下來，接線員，請開放提問。

We will now begin the question-and-answer session. (Operator Instructions)

我們現在開始問答環節。（接線員指示）

Gavin Clark-Gartner, Evercore ISI.

Gavin Clark-Gartner，Evercore ISI。

This is Yesha on for Gavin. I just had one on FOP. Wondering your current view on the number of diagnosed FOP patients in the US based on claims, patient advocacy and provider research. And then how many of those patients will immediately be treatable at launch?

我是 Yesha，代 Gavin 提問。我只有一個關於 FOP 的問題。想請教你們目前基於理賠資料、病友倡議組織以及醫療提供者研究，對美國已確診 FOP 患者人數的看法。另外，在上市時其中有多少患者能立即接受治療？

And then I have one follow-up on LIVMARLI.

然後我還有一個關於 LIVMARLI 的追問。

Yes. Thanks for the questions.

好的。謝謝你的問題。

Yes, the -- we point towards the approximately 300 identified patients in the US coming from patient [ advocacy ] group [IFOPA]. In terms of addressable patients, probably the main feature there is the NDA application inside filed as for age 12 and over. So that would be the main piece to consider, which would be the majority of the prevalent patients.

是的——我們所指的美國約300名已識別患者，來源是病友[倡議]團體[IFOPA]。就可觸及（可治療）患者而言，主要考量點可能是 NDA 申請所提交的適用年齡為12歲以上。因此這會是主要需要考量的部分，而這將涵蓋大多數現存患者。

And then on -- in terms of LIVMARLI specifically on the guidance raise, wondering what's kind of driving the bulk of the increase? Is it due to the ex-US expansion or kind of the continued PFIC ramp? And within PFIC, is the contribution still skewing towards those older patients?

接著——就 LIVMARLI 而言，關於上調財測指引，想了解主要推動增幅的因素是什麼？是來自美國以外市場的擴張，還是 PFIC 持續放量？以及在 PFIC 之中，貢獻是否仍偏向那些年齡較大的患者？

Yes. Thanks for the question. I mean, certainly, LIVMARLI US PFIC was the biggest driver and continue to see both pediatric and adult patients come to treatment. I think the older adolescents and adults really is the major driver, although we're still -- we think still in early innings.

是的。謝謝你的提問。我的意思是，當然，LIVMARLI 在美國的 PFIC 是最大的驅動因素，而且我們持續看到小兒與成人患者開始接受治療。我認為年齡較大的青少年與成人確實是主要驅動力，儘管我們仍然——我們認為仍處於早期階段。

We've made good progress educating adult providers on genetic testing, but still the minority of them are actually doing that. So I think there's probably more adult patients to find out there to potentially benefit from LIVMARLI.

我們在教育成人端醫療提供者了解基因檢測方面已取得良好進展，但實際上採行的仍然只占少數。因此我認為，外面可能還有更多成人患者有機會被找出來，並可能從 LIVMARLI 中受益。

And yes, on the international piece, in Q1, historically, we've seen a bit more seasonality and a little bit of a softer number in Q1. that just wasn't as much of a factor this year, somewhat in part of not only additional countries performing, but also PFIC starting to show up in that international number.

是的，關於國際市場的部分，第一季歷來我們會看到一些季節性因素，第一季數字也會稍微偏軟；但今年這個因素影響不大，部分原因不僅是有更多國家表現帶動，另外 PFIC 也開始反映在國際營收數字中。

Thank you.

謝謝。

Thanks for the questions.

謝謝你的提問。

Josh Shimmer, Cantor.

Josh Shimmer，Cantor。

Also on zilurgisertib, how are you thinking about its differentiation versus maybe some of the other programs in development, garetosmab, if I'm pronouncing that right. And so who knows that's number one.

另外關於 zilurgisertib，你們如何看待它相較於其他正在開發中的計畫的差異化，例如 garetosmab（如果我發音沒錯的話）。這是第一個問題。

Number two, are you planning to explore the program in other ossification indications disorders? And then number three, I think I heard you say peak sales for the asset of $200 million, is that global or US Thank you.

第二，你們是否計畫在其他骨化相關適應症／疾病中探索這個計畫？第三，我想我聽到你說這個資產的峰值銷售額為 2 億美元，這是全球還是美國？謝謝。

Thanks, Josh, for the questions. Yes, just to clarify that peak, the $200 million plus is a global number for us. And then in terms of kind of positioning here, the programs that you covered there, those are the ones that we're tracking, Sohonos being approved and the other program being in registration phase. And for Sohonos, the data coming out of the PROGRES study here for zilurgisertib is a real step forward in terms of the overall activity profile and tolerability and safety profile. So we see the clinical data here being quite meaningful advance on what's currently available in the market, which has quite a few limitations to it.

謝謝你，Josh，謝謝你的問題。是的，先澄清一下，這個峰值銷售額 2 億美元以上是我們的全球數字。至於定位方面，你提到的那些計畫就是我們在追蹤的：Sohonos 已獲核准，另一個計畫正處於註冊申請階段。就 Sohonos 而言，zilurgisertib 在 PROGRES 研究中釋出的數據，在整體活性表現、耐受性與安全性方面都是一大步的進展。因此我們認為，這裡的臨床數據相較於目前市面上可用的治療（其限制不少）是相當有意義的提升。

And then compared to the pipeline, this is an oral, which we see as a big advantage and particularly in the setting, where you can potentially drive ossification from injections and some of these other interventions. So having an oral, we see as a nice differentiator for the program.

再來與研發管線相比，這是一個口服藥，我們認為這是很大的優勢，特別是在某些情境下，注射與其他介入措施本身可能會誘發骨化。因此擁有口服劑型，我們認為是這個計畫很好的差異化特點。

Got it. And then plans for other ossification disorders?

了解。那麼對其他骨化疾病的規劃呢？

We -- it's still early days in thinking about it. At this point, we want to stay very focused on getting this launched for FOP, but it's certainly something we'll consider as we get further down the road.

我們——目前仍在早期階段思考。現階段我們希望非常專注於把它在 FOP 的上市推進完成，但這確實是我們在後續進展更深入時會考慮的方向。

Okay, thanks very much.

好的，非常感謝。

Thanks for questions.

謝謝提問。

John Wollenben, Citizens.

John Wollenben，Citizens。

Hey, thanks for taking the question.

嗨，謝謝讓我提問。

A little unusual having something under review or I haven't seen any of the data for. Just wondering what you guys have been privy to make you comfortable with this acquisition? And then what would be the form for it to make sense to get this out into the public domain?

有點不尋常的是，有個產品正在審查中，而我也還沒看到任何相關數據。想請問你們掌握了哪些資訊，讓你們對這次收購感到有把握？另外，要以什麼形式才合理把這些資訊公開給市場？

Yes. Thanks for the question, Jon and fully appreciate the uniqueness of the situation. In our review, we -- this is a conversation that actually has been going on for quite some time as is typical for a license transaction like this. So we've had full access to clinical data, to the regulatory correspondence and the NDA. So we feel quite confident in the profile for zilurgisertib and where they're at in the regulatory process.

是的。Jon，謝謝你的問題，我們也完全理解這個情況的特殊性。在我們的審查過程中，我們——這其實是一段已經持續相當長時間的對話，這在此類授權交易中很常見。因此我們已完整取得臨床數據、法規往來文件以及 NDA 申請資料的存取權限。所以我們對 zilurgisertib 的特性，以及其在法規流程中的進度，感到相當有信心。

From the Incyte side, where they've done a fantastic job putting together this program, and saw it fitting better in a rare disease company like Mirum, but the work they've done on it is quite strong. They want to have the data presented first at a medical meeting. So we're hopeful that's happening relatively soon. So once we get that presented, we'll be able to share more on the pivotal data and overall product profile.

從 Incyte 的角度來看，他們在打造這個計畫上做得非常出色，也認為它更適合放在像 Mirum 這樣的罕見疾病公司；他們在這個計畫上所做的工作相當扎實。他們希望先在醫學會議上發表數據。因此我們希望這會在相對近期發生。一旦完成發表，我們就能分享更多關於關鍵性數據與整體產品特性。

And will you guys be eligible for a pediatric review voucher if approved?

如果獲批，你們是否有資格取得兒科審查憑證（pediatric review voucher）？

So we do expect this to be eligible for a voucher under the terms of the agreement, Incyte will keep that voucher, and we will launch the product. So the way operationally, this works is Incyte is kind of given their mid-stride with the filing and review, they'll complete the primary role through approval, and then we'll take over sponsorship at the point of US approval.

我們確實預期依照協議條款，這項產品將符合取得憑證的資格；該憑證將由 Incyte 保留，而我們將負責產品上市。因此在營運上，Incyte 由於已在申請遞交與審查流程中途，將持續扮演主要角色直到獲批；之後在美國獲批時點，我們將接手成為申辦方（sponsorship）。

Got it. Okay. Thanks, Chris.

了解。好的。謝謝你，Chris。

Mike Ulz, Morgan Stanley.

Mike Ulz，Morgan Stanley。

Hi. This is Rohit on for Mike. With the recent pipeline acquisitions, can you talk about how you're thinking about BD moving forward? And then also, can you just talk about how you're thinking about pricing in FOP? Thanks.

嗨。我是 Rohit，代替 Mike 提問。近期管線收購之後，你們能談談未來對 BD（商務拓展）的思考方向嗎？另外，也請談談你們對 FOP 定價的想法。謝謝。

I can start, and I'll hand it over to Peter. But I think as you've seen now for Mirum kind of over the history of the company, we see a priority in staying active on the BD front. That's how you find unique opportunities that fit and add value to the company. So we will continue to work to find good programs to bring into the team. Maybe have Peter kind of touch on the -- any more on that or the second part of the question?

我先回答，然後交給 Peter。但我想你也看到，Mirum 在公司一路走來的歷史中，我們一直把保持 BD 的積極性視為優先事項。因為這樣才能找到契合且能為公司增值的獨特機會。所以我們會持續努力，尋找適合引進團隊的好計畫。也許請 Peter 補充一下——不論是這部分或問題的第二部分？

Yes. I mean nothing more on the BD strategy on the zilurgisertib pricing. Obviously, we'll make a decision and communicate that closer to approval. But just I think for thinking about the US, I mean, you can look at the Niemann-Pick C products and other ultra-rare settings like that where you have a strong value proposition and similar AbbVie is probably get you in the ballpark.

是的。關於 BD 策略沒有更多補充；至於 zilurgisertib 的定價，當然我們會在更接近核准時做出決定並對外溝通。但如果以美國市場來思考，你可以參考 Niemann-Pick C 的產品，以及其他類似的超罕見疾病情境：在具備強而有力的價值主張下，類似 AbbVie 的定價大概可以作為一個合理的參考區間。

James Condilus, Stifel.

James Condilus，Stifel。

Hey, thanks for taking my question. Congrats on the quarter. Maybe one follow-up on HDV. I think where we've heard a couple of questions is maybe in the 900 mg monthly arm, specifically as it relates to the T&D virologic response is maybe a little bit of an outlier relative to like some of your prior data and other -- the rest of your sort of data set. Just like curious your perspectives here. And as you think about the commercial opportunity here, for docs in the real world, what do you think is kind of the most important measure as it relates to evaluating efficacy for these different drugs?

嗨，謝謝讓我提問。恭喜本季表現。我想追問一下 HDV。我想我們聽到幾個問題，可能是關於每月 900 mg 組別，特別是與 T&D 病毒學反應相關的結果，似乎相較於你們先前的一些數據以及——你們其餘的整體數據集——有點像離群值。想聽聽你們的看法。另外，當你們思考這裡的商業機會時，對於臨床醫師在真實世界中，你認為在評估這些不同藥物的療效時，最重要的衡量指標是什麼？

Is it that T&D virologic response, other measures of virologic response, the composite? Just curious your perspective there.

是看那個 T&D 病毒學反應，還是其他病毒學反應指標，或是綜合指標（composite）？想聽聽你的觀點。

Yes. Thanks for the question. I'll maybe make a couple of comments and then have Joanne speak to some of the data that we're seeing out of the AZURE-1 Phase IIb portion. And I think in terms of what we're focused on and what we think is most relevant for ultimate use and driving adoption here is that composite of virologic response and ALT normalization. Those two factors are really what's pointed to in the FDA guidance and shows that you're not only addressing the viral load, but you're also addressing the liver inflammation that is part of the disease.

是的。謝謝你的問題。我先做幾點回應，接著請 Joanne 談談我們在 AZURE-1 第二期 b（Phase IIb）部分看到的一些數據。就我們的聚焦點，以及我們認為對最終使用與推動採用最相關的指標而言，是病毒學反應與 ALT 正常化的綜合指標。這兩個因素正是 FDA 指引所強調的，能顯示你不僅在處理病毒量，也同時在處理作為疾病一部分的肝臟發炎。

So seeing both of those move means you're kind of going after both components of the -- both of the considerations for both the infection and the liver. Maybe I'll ask Joanne to touch on the data we've seen to date.

因此，看到這兩者都有所變化，表示你某種程度上是在同時針對——同時考量感染與肝臟這兩個面向的組成部分。也許我請 Joanne 談談我們迄今看到的數據。

Yes. And so Chris is talking about the composites, all very true. When we look at the curves in terms of the virologic response, we do see declines in everyone. So when you structure the endpoint, if you don't meet a certain point by week 24, then you're either on one side of the line or the other. But we do see decreases in all of the patients.

是的。Chris 談到複合終點，確實都很正確。當我們從病毒學反應的曲線來看，確實看到每個人都有下降。所以當你設計終點時，如果在第 24 週沒有達到某個門檻，那你就會落在界線的一側或另一側。但我們確實看到所有患者都有下降。

There's certainly no evidence of lack of response or resistance or anything like that. So really, partly, it's an artifact of time. We do see deepening response with continued treatment. And again, this is a fairly small -- numerically fairly small group. We'll have a lot more information with the full AZURE-1 and AZURE 4 Phase III data sets to make a final call on that.

當然沒有任何無反應或抗藥性之類的證據。所以某種程度上，這其實是時間造成的假象。我們確實看到隨著持續治療，反應會進一步加深。而且再次強調，這是一個相當小——就數量而言相當小的族群。等到完整的 AZURE-1 與 AZURE 4 第三期資料集出來後，我們會有更多資訊來做最終判斷。

Makes sense. Thank you.

有道理。謝謝。

Brian Skorney, Baird.

Brian Skorney，Baird。

Hey, good afternoon, guys. Thanks for taking my question and great quarter. I guess I'd love to also ask a question on FOP too. It seems like you're doubling down on making Ipsen your corporate nemesis. So I'm just wondering if you could kind of give your broad thoughts on where you think Sohonos' profile leaves an opportunity for another entrant and compare and contrast how zilurgisertib might address these. And the time line would put us right around mid-cycle review with the FDA right now.

嗨，大家下午好。謝謝讓我提問，這一季表現很棒。我想我也想問一個關於 FOP 的問題。看起來你們是在加碼把 Ipsen 當作你們的企業宿敵。所以我想請你們談談整體看法：你們認為 Sohonos 的產品特性在哪些地方留下了讓其他新進者切入的機會？以及 zilurgisertib 可能如何因應這些機會，請做一下比較與對照。而時間點上，我們現在大概正好在 FDA 的期中審查（mid-cycle review）附近。

So I'm just wondering if you could say if that has already happened or is still pending.

所以我想問的是，那個審查是否已經發生，還是仍在進行中？

Yes. Thanks for the question. On the review, yes, that has happened, and I'd just say things are tracking as expected. But maybe I'll ask Peter to comment on -- a little bit more on positioning.

是的。謝謝你的問題。關於審查，是的，已經發生了；我只能說一切進展符合預期。不過也許我請 Peter 針對——定位方面再多評論一些。

Yes. And I think certainly, the feedback we've heard from stakeholders, patients, caregivers, physicians, others available therapy in the market today. There's just -- there's a lot to be desired in terms of both efficacy and safety. And we'll obviously be able to get into more details once we have the PROGRESS data presented at an upcoming medical conference. But from what we've seen in our review, the zilurgisertib profile, really exciting about what it can mean for these patients, both efficacy-wise as well as a convenient oral and well-tolerated regimen.

是的。我想我們從利害關係人——病患、照護者、醫師等——那裡聽到的回饋是：目前市場上的可用療法，在療效與安全性兩方面都還有很大的改善空間。確實——在療效與安全性方面都還有很多可期待之處。當然，一旦 PROGRESS 的數據在即將到來的醫學會議上發表，我們就能談得更細。但就我們目前的審視而言，zilurgisertib 的特性非常令人振奮，無論是對病患的療效，或是便利的口服、且耐受性良好的治療方案。

Lisa Walter, RBC Capital.

Lisa Walter，RBC Capital。

Great. Thanks so much for taking our question. Maybe just some more details if you can share on the opportunity for FOP. Are there any overlap with your current call points? And did you disclose the deal terms with Incyte? And maybe just one more. Given the recent positive results in [ hep D ] and PSC, wondering if this has impacted your thinking on when Mirum could become a profitable company. Thanks so much for taking our question.

很好。非常感謝讓我們提問。如果可以分享的話，想請再多提供一些關於 FOP 機會的細節。這是否與你們目前的拜訪對象（call points）有任何重疊？以及你們是否已披露與 Incyte 的交易條款？另外再問一個。鑑於近期在 [ hep D ] 與 PSC 的正面結果，想請問這是否影響你們對 Mirum 何時能成為獲利公司的看法？非常感謝讓我們提問。

Yes. Maybe I'll ask Peter to kind of speak to the FOP points and then have Eric kind of give some financial forward-looking views.

是的。也許我先請 Peter 談談 FOP 相關的重點，然後請 Eric 提供一些財務上的前瞻看法。

Yes. great overlap with our existing team, our rare genetics team that's focused on CTEXLI, CHOLBAM. We mentioned that the majority -- the significant majority of the FOP patients, FOP patients are cared for in settings -- centers that also prescribe CTEXLI, CHOLBAM. Different prescribers most of the time, some overlap in medical genetics. FOP has the biggest prescriber there will be endocrinologists.

是的。與我們現有團隊的重疊度很高——我們的罕見遺傳疾病團隊目前聚焦於 CTEXLI、CHOLBAM。我們提到，多數——相當大多數的 FOP 病患，是在同樣也會開立 CTEXLI、CHOLBAM 的醫療環境／中心接受照護。大多數時候開立醫師不同，但在醫學遺傳領域有一些重疊。FOP 最大的開立族群會是內分泌科醫師。

So that's a new kind of physician target, but the center overlap is really high with our existing rare genetics business. So excited about adding this product to that team.

所以那會是一個新的醫師目標族群，但就中心端而言，與我們現有的罕見遺傳業務重疊度非常高。因此我們很期待把這個產品加入該團隊。

Yes. And then on the financials, we did disclose the upfront license fee was $16 million, and then the next milestone would be $25 million upon FDA approval. There's some other commercial milestones, but also a royalty in the low to -- well, mid to high single-digit range. And we expect after launch that this product will be accretive very, very quickly. But your question was also about path to profitability, and I think that's much more driven by brelovitug and volixibat as well as our current commercial business.

是的。至於財務面，我們確實披露了預付授權費為 1,600 萬美元，下一個里程碑是在 FDA 核准時支付 2,500 萬美元。另外還有一些商業化里程碑款項，以及一個低到——嗯，中到高個位數百分比區間的權利金（royalty）。我們預期上市後，這個產品會非常、非常快就帶來增益（accretive）。不過你也問到獲利路徑，我認為那更多是由 brelovitug 與 volixibat，以及我們目前的商業化業務所驅動。

As we've said, we're spending a lot on R&D this year for both of those products. And so, profitability will be pushed out probably until '28 on a GAAP basis.

如同我們所說，今年我們在這兩個產品上的研發支出很高。因此，以 GAAP 基礎來看，獲利時間點可能會延後到大約 2028 年。

But I would reiterate that we expect to be operating cash flow positive next year.

但我想再次強調，我們預期明年營運現金流將轉為正值。

Yes.

是的。

Jessica Fye, JP Morgan.

Jessica Fye，JP Morgan。

Hey, guys. Good afternoon. Thanks for taking my question. Can you estimate the contribution to first quarter LIVMARLI sales from Alagille versus PFIC? And then another one on FOP, just thinking about that market. What do you see as the penetration for palovarotene? And would you envision the ALK being used in combination with that drug?

嗨，各位。下午好。謝謝讓我提問。你們能否估算第一季 LIVMARLI 銷售中，Alagille 相較於 PFIC 的貢獻？另外一個關於 FOP，從那個市場來看，你們認為 palovarotene 的滲透率會是多少？以及你們是否會設想 ALK 會與那個藥物併用？

Thanks Jess, for the questions. I mean just briefly on LIVMARLI, we typically aren't breaking out by indication, but what we'd say is that PFIC is -- they're both growing, but PFIC is the bigger growth driver. Maybe pass the FOP question over to Peter.

謝謝你，Jess，這些問題。關於 LIVMARLI，我們通常不會按適應症拆分揭露；但我們會說 PFIC 與 Alagille 都在成長，不過 PFIC 是更大的成長驅動因素。FOP 的問題我就交給 Peter。

Yes. And when we look at the US, the market where this medicine is available, palovarotene, it's probably a minority of diagnosed patients that are currently receiving it. We can see from pharmacy claims data, and that kind of matches what we've heard in physician and caregiver interviews that can be tried, but it can often be difficult to tolerate and stay on.

是的。當我們看美國——也就是這個藥物 palovarotene 已可取得的市場——目前實際使用的，大概只占已確診患者中的少數。我們從藥局理賠資料可以看到這點，而這也與我們在醫師與照護者訪談中聽到的情況一致：可以嘗試使用，但往往耐受性不佳，難以持續用藥。

Thanks for the question.

謝謝你的問題。

Mani Forahar, Learnik Partners.

Mani Forahar，Learnik Partners。

Hey, guys. You have Ryan on for Mani. Congrats on the quarter. Maybe just circling back to FOP. What's the latest thinking here on an OUS filing and when you guys would expect to launch there? And then just going back to the peak sales of $200 million, we're just wondering, is that in the 12-plus age group that you guys would get approved in the upcoming?

嗨，各位。我是 Ryan，代 Mani 上線。恭喜這一季表現。想再回到 FOP。目前對於美國以外（OUS）的申請策略最新想法是什麼？以及你們預期何時能在那邊上市？另外回到 2 億美元的峰值銷售，我們想確認：這是否是以你們即將獲批的 12 歲以上族群為基礎？

And how should we think about upcoming data for the younger age groups that are being tested?

以及對於正在測試的更年幼年齡層，接下來的數據我們應該如何看待？

Thanks for the questions, Ryan. On ex US strategy, actually, a European filing is upcoming. So we could actually still have that in this quarter. Incyte is still driving those activities.

謝謝你的問題，Ryan。關於美國以外策略，其實歐洲的送件即將進行。所以我們這一季其實仍有可能看到該進展。這些活動仍由 Incyte 主導。

And again, their team is doing a great job. In terms of the overall peak estimate, that $200 million plus, that's the full brand in FOP over life cycle is the way we look at it. The younger age patients, we do expect that the label would launch with the 12 and older. There are two other cohorts in the study that are ongoing that would support potentially taking that age lower over the near term, frankly. So those are ongoing and enrolling now, so they're not too far out.

再一次，他們的團隊做得非常出色。就整體峰值預估而言，那 2 億美元以上，是我們看待 FOP 全品牌在整個產品生命週期內的方式。對於較年輕的患者，我們確實預期標籤會以 12 歲及以上作為上市起點。研究中還有另外兩個隊列正在進行，坦白說，這些隊列可能在短期內支持把年齡下修。因此這些研究仍在進行且目前正在收案，所以距離結果不會太久。

Awesome. Thanks.

太棒了。謝謝。

Great. Thanks for the questions.

很好。謝謝你的提問。

Ryan Deschner, Raymond James.

Ryan Deschner，Raymond James。

Thanks for the question. A couple for me. What's your strategy for identifying FOP patients in the US and abroad and addressing a relatively high misdiagnosis rate for FOP? And do you anticipate any early line of sight into a substantial group of patients from Incyte's prior clinical studies or maybe a compassionate use program or something like that in FOP? And I have a follow-up. Thanks.

謝謝這個問題。我有幾個問題。你們在美國及海外識別 FOP 患者、並處理 FOP 相對較高的誤診率方面，有什麼策略？另外，你們是否預期能在早期就看到一大群患者的線索，來源可能是 Incyte 先前的臨床研究，或是同情用藥計畫之類在 FOP 的安排？我還有一個追問。謝謝。

Yes. Thanks for the question, Ryan. Yes, the -- I think that FOP patients often have a longer diagnostic odyssey than they should. There are patients get diagnosed at birth, but often I think the literature said the average age of diagnosis is seven years, and so obviously, people that wait longer than that. I think that's improved with the availability of genetic testing.

是的。謝謝你的問題，Ryan。是的——我認為 FOP 患者往往經歷比應有更長的診斷歷程。有些患者在出生時就被診斷出來，但我想文獻指出平均診斷年齡是 7 歲，因此顯然也有人等待更久。我認為隨著基因檢測的可近性提升，情況已有改善。

But that's, I think, an opportunity to continue to raise awareness, just like all of our rare genetic diseases to try to shorten that diagnostic odyssey as much as we can. We do think in the US, though, most of the -- a pretty substantial majority of the patients are identified with FOP, probably a different story in middle and lower-income countries [ interesting ].

但我認為這仍是一個持續提升認知的機會，就像我們所有罕見遺傳疾病一樣，盡可能縮短這段診斷歷程。不過我們確實認為在美國，大多數——相當大比例的患者已被識別為 FOP；在中低收入國家可能就是不同的情況。[有趣]。

Ed, did you have a follow-up, Brian?

Ed，你有追問嗎，Brian？

Yes. I just was also wondering if there was anything notable so far in the VISTAS extension in terms of rollover discontinuation rates, pruritus or other patient metrics that might take a little longer to modulate over time.

有。我也想知道，到目前為止在 VISTAS 延伸試驗中，是否有任何值得注意之處，例如續入後的停藥率、搔癢（pruritus）或其他可能需要更長時間才會隨時間調整的患者指標。

Yes. I mean Incyte does have this PROGRESS study. It's enrolling well. And I think we'll be able to disclose more about what they've seen from that at the upcoming medical conference, but have certainly seen a lot of physician and patient interest in the program.

是的。我的意思是，Incyte 確實有這項 PROGRESS 研究。收案進展良好。我想我們將能在即將到來的醫學會議上披露更多他們在該研究中看到的情況，但我們確實看到醫師與患者對該計畫有很高的興趣。

Okay, thanks very much.

好的，非常感謝。

Joseph Thome, TD Cowen.

Joseph Thome，TD Cowen。

Hi there. Good afternoon. Thank you for taking my questions. Maybe one on FOP. I guess the level of ALK2 inhibition you're seeing with the therapy, I guess, do you think that could be enhanced by garetosmab, Regeneron's Activin A drug? Or are these largely just going to be competitive therapeutics in the landscape? And then second, when we think about the potential expansion opportunity for LIVMARLI in the basket trial that's going to be reading out later this year, I guess, how should we think about that in your overall projection for LIVMARLI, how much is this basket population?

你好。下午好。謝謝你回答我的問題。先問一個關於 FOP 的問題。我想就你們治療所看到的 ALK2 抑制程度而言，你們認為是否能透過 garetosmab（Regeneron 的 Activin A 藥物）來增強？還是說在這個領域中，這些大多將是彼此競爭的療法？第二個問題，當我們思考 LIVMARLI 在今年稍晚將讀出結果的籃式試驗中的潛在擴適應症機會時，我們應該如何把它納入你們對 LIVMARLI 的整體預測？這個籃式族群大概有多大？

Thank you. I can make a quick comment on the garetosmab positioning, and it's probably best to get into more detail on this after our data is presented, so it can give a better -- a more complete picture. But we think the profile for zilurgisertib and its clinical positioning is really strong as a convenient oral single agent and excited about bringing that forward. Maybe I have Peter take the follow-up.

謝謝。我可以先對 garetosmab 的定位做個簡短評論；而更深入的細節，可能最好等我們的數據發表後再談，這樣能提供更好——更完整的圖像。但我們認為 zilurgisertib 的特性與其臨床定位非常強，作為方便的口服單藥，我們也很期待推進它。也許我請 Peter 回答追問。

Yes. And expand, we've kind of talked about that indication being about one-third of the at least $1 billion peak sales opportunity for LIVMARLI and still reiterate that. Thank you.

是的。至於擴適應症，我們之前談過，那個適應症約占 LIVMARLI 至少 10 億美元峰值銷售機會的三分之一，我們仍重申這一點。謝謝。

Thanks for the question.

謝謝你的提問。

Charles Wallace, HCW.

Charles Wallace，HCW。

Hi, this is Charles on for RK. Thanks for taking my question. This is Charles on for RK. So for FOP, another question on that. How many patients from the PROGRESS study, I think there was 63 in that study, do you expect will -- could come on after launch? And do you expect to have some sort of bridging program?

你好，我是 Charles，代替 RK 發言。謝謝你回答我的問題。我是 Charles，代替 RK 發言。關於 FOP，再問一個問題。PROGRESS 研究中，我記得該研究有 63 名受試者，你們預期其中有多少患者在上市後會——可能會轉入用藥？以及你們是否預期會有某種銜接計畫（bridging program）？

Thanks for the question, Charles. And given the kind of the nature of the relationship here, we're going to wait until we have that data presented to give some of the detail on it. Overall, I think it's a really compelling profile and the feedback has been positive, but I just don't want to get into specifics ahead of having that data presented.

謝謝你的問題，Charles。鑑於這裡合作關係的性質，我們會等到數據發表後再提供一些細節。整體而言，我認為這是一個非常有說服力的特性，回饋也很正面，但在數據發表之前我不想談得太具體。

Okay. That's fair. And then I guess another question, if I may, on the sales force expansion. So you're growing it to 60 in the field. And I just wanted to maybe get a little more color on when you expect these -- the team to be fully on board?

好的。這很合理。那我再問一個問題，如果可以的話，關於銷售團隊擴編。你們要把前線人員擴增到 60 人。我想再多了解一下，你們預期這——這個團隊何時會完全到位？

And also, when do you expect them to be fully functional?

另外，你們預期他們何時能完全發揮功能？

Yes. I -- kind of prepared comment was starting this -- later this year. And so I kind of think early next year, we'd be kind of fully on board. And that team would cover both pediatric and adult settings, where not just Alagille and adult PFIC can be found, but also PSC and delta. So I think by early next year, they'd be active in all those areas.

是的。我——我們準備好的說法是今年稍晚開始。因此我認為到明年初，我們會大致完全到位。而該團隊將同時涵蓋兒科與成人場域，不僅是可找到 Alagille 與成人 PFIC 的地方，也包括 PSC 與 delta。所以我認為到明年初，他們會在所有這些領域展開活動。

And of course, with the pipeline products, the activity would really start upon potential FDA approval.

當然，對於管線產品，相關活動將會在潛在獲得 FDA 核准後才真正開始。

Great. Thanks for taking my questions, and congrats on the third quarter. Thanks for the questions.

很好。謝謝你回答我的問題，也恭喜第三季的表現。謝謝各位的提問。

There are no further questions at this time. I would now like to turn the call back to Chris Peetz for closing remarks.

目前沒有進一步的問題。我現在想把電話交回給 Chris Peetz 作結語。

All right. Well, thank you all for joining us today and for all the support and a great start to 2026. Have a great afternoon.

好的。非常感謝各位今天加入我們，也謝謝大家的支持，讓 2026 年有個很好的開端。祝各位下午愉快。

This concludes today's call. Thank you for attending. You may now disconnect.

今天的電話會議到此結束。感謝各位參與。您現在可以掛線。