BridgeBio Pharma Inc (BBIO) 2025 Q1 法說會逐字稿

Good afternoon. I will be your conference operator today. (Operator Instructions)

午安.今天我將擔任您的會議主持人。（操作員指示）

Before we begin, I would like to remind everyone that today's call may contain forward-looking statements within the meaning of the federal securities laws, including, but not limited to, statements about BridgeBio's future operating and financial performance, business plans, and prospects and strategy. These statements are based on current expectations and assumptions that are subject to risks and uncertainties which could cause actual results to differ materially from those expressed or implied in these forward-looking statements.

在我們開始之前，我想提醒大家，今天的電話會議可能包含聯邦證券法所定義的前瞻性陳述，包括但不限於有關 BridgeBio 未來營運和財務業績、業務計劃以及前景和策略的陳述。這些聲明是基於目前的預期和假設，受風險和不確定性的影響，可能導致實際結果與這些前瞻性聲明中表達或暗示的結果有重大差異。

For a discussion of these risks and uncertainties, please refer to the disclosure in today's earnings release and BridgeBio's periodic reports and SEC filings. All statements made here are based on information available to BridgeBio as of today, and the company undertakes no obligation to update any forward-looking statements made during this call, except as required by law.

有關這些風險和不確定性的討論，請參閱今天的收益報告和 BridgeBio 的定期報告和 SEC 文件中的揭露內容。此處所做的所有聲明均基於 BridgeBio 截至今日掌握的信息，除非法律要求，否則公司不承擔更新本次電話會議期間做出的任何前瞻性聲明的義務。

With that completed, BridgeBio, you may begin your conference.

完成後，BridgeBio，您可以開始您的會議了。

Good afternoon, and thank you for joining BridgeBio's Q1 2025 earnings conference call. This is Chinmay Shukla, VP of Strategic Finance at BridgeBio. Today, we will discuss our financial results for the first quarter of 2025 and provide a review of how we measure success in our business. We'll provide insight into the early results from the launch of Attruby as well as provide an update on our pipeline, which had three Phase 3 readouts expected in the next year, including our efforts in expansion indications such as in chronic hypoparathyroidism.

下午好，感謝您參加 BridgeBio 2025 年第一季財報電話會議。我是 BridgeBio 策略財務副總裁 Chinmay Shukla。今天，我們將討論 2025 年第一季的財務業績，並回顧我們如何衡量業務成功。我們將深入了解 Attruby 推出後的早期結果，並提供我們產品線的最新信息，預計明年將有三個 3 期臨床試驗的讀數，包括我們在慢性甲狀旁腺功能減退症等擴展適應症方面的努力。

Today's call will feature Neil Kumar, Chief Executive Officer; Matt Outten, Chief Commercial Officer; and Tom Trimarchi, President and Chief Financial Officer. For the Q&A portion of the call, Ananth Sridhar, Chief Operating Officer of BridgeBio [Cardio Reno]; and Justin To, Chief Executive Officer of BridgeBio [Skeletal Dysplasia] will also join. Following the remarks by our team, we will open up the call for Q&A.

今天的電話會議將由執行長 Neil Kumar 主持； Matt Outten，商務長；以及總裁兼財務長 Tom Trimarchi。在電話會議的問答部分，BridgeBio [Cardio Reno] 首席營運長 Ananth Sridhar； BridgeBio[骨骼發育不良]執行長Justin To也將加入。在我們團隊發言之後，我們將開始問答環節。

With that, I'll turn the call over to Neil.

說完這些，我會把電話轉給尼爾。

Thanks to everyone on the line for the time, and welcome to our first earnings call. We're grateful to open up another dimension of our dialogue with our investor partners. You collectively allowed us to deliver for the patients we serve, and we look forward to your feedback on how to continually improve our discussion in this setting.

感謝各位線上的時間，歡迎參加我們的第一次財報電話會議。我們很高興能夠與我們的投資者夥伴開啟另一個層面的對話。你們共同使我們能夠為我們服務的患者提供服務，我們期待你們的回饋，以便我們在此環境中不斷改進討論。

Today, we know the star of the show is the Attruby launch. And the good news is that the brand is delivering for patients and the business. $36.7 million in revenue this past quarter suggest that clinicians and patients resonating with our differentiated clinical efficacy, safety, and accessibility.

今天，我們知道這場演出的焦點是 Attruby 的發布。好消息是，該品牌正在為患者和企業帶來效益。上個季度 3,670 萬美元的收入表明臨床醫生和患者對我們差異化的臨床療效、安全性和可近性產生了共鳴。

We continue to educate on our therapeutic impact, which comes as early as three months with a 42% relative risk reduction on cardiovascular hospitalization and mortality at 30 months, including a 50% reduction in cardiovascular hospitalization at that same time point.

我們繼續宣傳我們的治療效果，最快在三個月內就能看到效果，30 個月時心血管住院和死亡的相對風險降低了 42%，其中同一時間點心血管住院率降低了 50%。

All these best-in-class point estimates are available at the lowest price point in the marketplace. There is much to like there and we're aware that there is much work yet to be done. You'll hear about some of that work today as it pertains both to commercial tactics and further development work.

所有這些一流的點估計都是以市場最低的價格提供的。那裡有很多值得喜歡的地方，我們也知道還有很多工作要做。今天您將聽到一些有關商業策略和進一步開發工作的工作。

We add to Attruby good news to continued progress across our pipeline of three additional blockbuster products. Our trials in Limb-Girdle muscular dystrophy type 2I, achondroplasia, and ADH1 remain on track with low dropout rates, frequent data collection and positive site audits, all in preparation for an efficient NDA submission should the data be positive.

我們為 Attruby 帶來了好消息，同時我們的另外三款重磅產品線也繼續取得進展。我們在 2I 型肢帶型肌肉營養不良症、軟骨發育不全和 ADH1 方面的試驗進展順利，退出率低，數據收集頻繁，現場審核積極，所有這些都為在數據積極的情況下高效提交 NDA 做好準備。

Furthermore, key additional markets are being opened up. Our hypochondroplasia trial enrolled with incredible speed, and we now have first patient in there. Our work with Encaleret in the hypoparathyroid setting positions it well for a registrational trial to successful could pave the way for the first oral compelling solution in that space.

此外，其他主要市場也正在開放。我們的軟骨發育不良試驗以驚人的速度招募患者，現在我們已經有了第一位患者。我們與 Encaleret 在副甲狀腺功能減退症領域的合作為註冊試驗的成功奠定了基礎，可以為該領域首個令人信服的口服解決方案鋪平道路。

Before we move to the discussion into further specifics regarding the business and since this is our first time hosting this type of call, I wanted to briefly address how we measure the performance of our business on an ongoing basis. It's nice that this quarter was a success, but there will be harder quarters, I am sure. And through it all, you should expect for us to communicate consistently and in terms of net gains or losses in NPV.

在我們進一步討論業務細節之前，由於這是我們第一次主持此類電話會議，我想簡要介紹我們如何持續衡量業務績效。本季取得成功固然很好，但我確信接下來的季度還會更加艱難。在整個過程中，您應該期望我們能夠以 NPV 中的淨收益或淨損失的形式進行持續溝通。

For instance, in this last quarter, our NPV increased by 9%, given changes in the following variables: number one, the time factor of money, especially given the late stage of our portfolio; number two, a higher-than-anticipated revenue driven by a faster uptake of Attruby than we anticipated and likely a slightly higher peak year share by volume than our market research had projected; third is the success of our small study in HP and other sponsors setbacks in the space of oral HP medicines; fourth, the early enrollment of our run-in of our hypochondroplasia study, moving our timelines in for that program; and fifth, a slight decrease in our model cost of capital given our recent convertible offering. There were NPV drags, most prominently including tariffs. However, these have a close to negligible effect at less than 1% of our overall NPV.

例如，在上個季度，我們的 NPV 增加了 9%，這得益於以下變量的變化：第一，資金的時間因素，特別是考慮到我們投資組合的後期階段；第二，由於 Attruby 的普及速度比我們預期的要快，收入高於預期，而且高峰年銷量份額可能比我們的市場研究預測的要高一些；第三是我們在 HP 方面取得了成功的規模，第三級藥物領域遭遇了挫折；第四，提前招募軟骨發育不良症研究的患者，從而提前完成該項目的時間表；第五，鑑於我們最近發行的可轉換債券，我們的模型資本成本略有下降。存在淨現值拖累，最突出的是關稅。然而，這些影響幾乎可以忽略不計，不到我們整體 NPV 的 1%。

Stepping back from NPV and as a reminder, our overall objective here at BridgeBio is simple, to maximize the positive change we can have in terms of quality-adjusted life years for the patients that we serve as quickly as possible. We do so by creating as many meaningful medicine as quickly as possible within three constraints.

拋開 NPV 不談，提醒一下，BridgeBio 的總體目標很簡單，就是盡快最大限度地提高我們所服務的患者在品質調整生命年方面的積極變化。我們透過在三個限制條件下盡快創造盡可能多的有意義的藥物來實現這一目標。

The first is that each program must have beautiful science, which in our vernacular means a high POTS driven by understanding of mechanism of disease optimally paired with a therapeutic mechanism of action that targets a well-described genetic condition at its source.

首先，每個項目都必須具有優美的科學性，用我們的話來說，就是由對疾病機制的理解驅動的高 POTS，並與針對源頭上明確描述的遺傳狀況的治療作用機制進行最佳匹配。

These types of programs historically have a three to four times higher probability of technical success than all-comer drug R&D efforts. Over time, in many programs, this elevated probability of technical success moves us from being a speculative lottery ticket to being something akin to an engineering company.

從歷史上看，這類計畫的技術成功機率比所有藥物研發項目高出三到四倍。隨著時間的推移，在許多專案中，技術成功機率的提高使我們從一張投機彩票變成了類似於工程公司。

The second constraint is that we strive for each medicine that we make to be first-in-class or best-in-class. Determining best-in-class can be tricky in the absence of double-blind head-to-heads, but we approach this news as a straightforward basin approach.

第二個限制是，我們要努力使我們生產的每種藥物都成為同類中的第一或最佳。在沒有雙盲頭對頭的情況下，確定同類最佳產品可能會很棘手，但我們以直接的盆地方法來處理這一消息。

For instance, in TTR, if faced with this vision as to what drug to use, a logical person would first interrogate the salient endpoint, death and hospitalization at a common time point. In this case, at 30 months, a relative risk reduction of 42% is a better point estimate as compared with any of the other products in this marketplace. You would then ask, how quickly does each drug take action. And you would find that the three-month beginning of separation between placebo and active is the earliest point estimate of timing of impact in this field. So logically, Attruby is at worst, no worse than other medicines.

例如，在 TTR 中，如果面臨使用什麼藥物的設想，一個有邏輯的人首先會詢問突出的終點，即在共同的時間點死亡和住院。在這種情況下，在 30 個月時，與市場上的任何其他產品相比，42% 的相對風險降低是一個更好的點估計。然後你會問，每種藥物起效的速度有多快。你會發現，安慰劑和活性劑分離後的三個月是該領域影響時間的最早點估計。所以從邏輯上來說，Attruby 最壞的情況，也不會比其他藥物差。

You would then look at safety. Here, you would find a little difference between small molecules, but would observe that they do not have the safety signals some knockdowns have, vitamin A deficit, which, by the way, turns knockdowns into a once-daily pill regimen, injection site reactions, the absence of impact on [Afib] whereas both small molecules had meaningful reductions there, the imbalance of cardiac SAEs is observed in the IMPATRO study and the as of yet unexplained imbalance in death on the revusiran trial.

然後你就會關注安全性。在這裡，你會發現小分子之間存在一些差異，但會觀察到它們沒有一些敲低療法所具有的安全信號，維生素 A 缺乏症，順便說一句，這會將敲低療法變成每日一次的藥丸療法，注射部位反應，對 [Afib] 沒有影響，而兩種小分子在那裡都有有意義的減少，在 IMPATRO 研究中觀察到不平衡的心臟試驗中觀察到不平衡的死亡。

You couple that with the overall finding that across multiple studies, TTR is a cross-species conserved protein at ever higher levels of TTR lead to ever longer lives and less disease over time. So logically, small molecule stabilization from these data is the safest approach.

結合多項研究得出的總體結論，TTR 是一種跨物種保守蛋白，隨著時間的推移，TTR 水平越高，壽命越長，疾病越少。因此從邏輯上講，從這些數據來看，小分子穩定是最安全的方法。

Finally, the [logician] might look at price only to discover that Attruby has the lowest price point. We believe given all of that, the choice is clear. We apply similar reasoning in other competitors' faces like achondroplasia.

最後，[邏輯學家] 可能只看價格就發現 Attruby 的價格最低。我們相信，考慮到所有這些，選擇是明確的。我們對其他競爭對手的臉部特徵（如軟骨發育不全）應用了類似的推理。

Our final constraint is that each program we work on should be NPV positive to ensure firm level sustainability. We use all available levers during the R&D stage to optimize NPV, including time, cost, cost of capital, and probability of technical success. The better our model is generating economic value from projects, the further we can push into markets where others cannot extract adequate value in addition to being a better owner of obviously NPV-positive projects.

我們的最後一個限制是，我們所進行的每個專案的淨現值都應該為正，以確保公司層面的永續性。我們在研發階段使用所有可用的槓桿來優化 NPV，包括時間、成本、資本成本和技術成功的機率。我們的模型從專案中產生的經濟價值越好，我們就越能進入其他人無法獲得足夠價值的市場，並且成為明顯 NPV 為正的專案的更好的所有者。

The fact that we will spend well under $100 million per program to get each one of our potential blockbusters from the preclinical stage through proof of concept, sets us up well for future growth that is economically attractive.

事實上，我們將為每個項目花費不到 1 億美元，以使我們的每個潛在重磅炸彈從臨床前階段進入概念驗證階段，這為我們未來具有經濟吸引力的成長奠定了良好的基礎。

Okay. So that's our overall objective function with its intended constraints. You heard the first and most important frame that we used to think about the business, which is NPV. There are other frameworks that we also use to look at the business: understanding the business by stage and capability, research, development and commercial; understanding the business in the context of the overall ecosystem; and understanding the business program by program.

好的。這就是我們的總體目標函數及其預期限制。您聽到了我們用來思考業務的第一個也是最重要的框架，即 NPV。我們也使用其他框架來審視業務：按階段和能力、研究、開發和商業了解業務；在整個生態系統的背景下理解業務；並逐一了解業務計劃。

Using the buy stage framework first, and as I mentioned earlier, we are pleased with the ongoing progress of our commercial launch and the building of a sustainable competitive advantage there, which means, of course, that our group is able to extract more profit from the asset than another would be able to. I've also touched already on our ongoing development wins.

首先使用購買階段框架，正如我之前提到的，我們對商業發布的持續進展和可持續競爭優勢的建立感到滿意，這當然意味著我們的集團能夠從資產中獲得比其他集團更多的利潤。我也已經談到了我們正在進行的開發勝利。

On Discovery, our bread and butter, we continue to advance programs in genetic dilated cardiomyopathy and ADPKD. We also hold significant stakes in BridgeBio Oncology Therapeutics and GondolaBio. In the latter, we expect Phase 3 data in our EPP program later this year, and we expect to generate up to six development candidates in 2025.

在探索方面，我們的主要業務是繼續推進遺傳性擴張型心肌病變和 ADPKD 計畫。我們也持有 BridgeBio Oncology Therapeutics 和 GondolaBio 的大量股份。在後者中，我們預計今年稍後我們的 EPP 計劃將獲得第 3 階段數據，並且我們預計在 2025 年將產生最多 6 個開發候選人。

From an ecosystem framing, the substrate from making genetic medicines remains incredible. We see that in the progress Gondola is making and more broadly with the depth of genetic disease starting points being produced and accessible at low price points.

從生態系統框架來看，製造基因藥物的基質仍然令人難以置信。我們看到，Gondola 正在取得進步，更廣泛地說，遺傳疾病起點的深度正在被生產出來，並且可以低價獲得。

Finally, at a program level, Matt will have more to say about our commercial launch of Attruby. Our goal there, as a reminder, is $4.3 billion in peak year sales or about 30% of a $15 billion marketplace. That gets delivered against approximately $380 million of spend on the brand per year.

最後，在專案層面，馬特將更多地談論我們對 Attruby 的商業發布。提醒一下，我們的目標是高峰年銷售額達到 43 億美元，或 150 億美元市場的 30% 左右。這一目標的實現離不開每年約 3.8 億美元的品牌支出。

Almost 25% of current spend is on further research development activities like ACT-EARLY, which we think can continue to improve our medicines positioning and its ability to help patients in later years. The product of this spend also includes salient results like our variant data, where we obtained a hazard ratio of 0.41 with a p-value of less than 0.02 in the sickest of patients. And new work we are doing regarding Afib, which is an emerging marker of disease progression, the first results of which we plan to present at ESC later this year.

目前近 25% 的支出用於 ACT-EARLY 等進一步的研究開發活動，我們認為這可以繼續改善我們的藥物定位及其幫助晚年患者的能力。這項支出的產物還包括一些顯著的結果，例如我們的變異數據，其中我們獲得了病情最嚴重的患者的風險比為 0.41，p 值小於 0.02。我們正在進行有關 Afib 的新研究，Afib 是疾病進展的新興標誌，我們計劃在今年稍後的 ESC 上展示其首批結果。

For infigratinib, a first-in-class oral FGFR3 inhibitor in development for both achondroplasia and hypochondroplasia, the pivotal PROPEL 3 Phase 3 is fully enrolled, and we expect last participant last visit by the end of this year. We have also reached regulatory alignment with the FDA on our clinical development plan for infigratinib in children with achondroplasia from ages zero to three, and we expect to initiate clinical development in this important age range by the end of the year. Excitingly, we have also enrolled the run-in for our Phase 2 hypochondroplasia trial well ahead of benchmarks.

對於 infigratinib 來說，這是一種用於治療軟骨發育不全和軟骨發育不良的首創口服 FGFR3 抑制劑，其關鍵的 PROPEL 3 第 3 階段已經完全入組，我們預計最後一位參與者的最後一次訪問將在今年年底前完成。我們也針對 0 至 3 歲軟骨發育不全兒童的英菲格拉替尼臨床開發計畫與 FDA 達成了監管一致，我們預計將在今年年底前啟動這一重要年齡層的臨床開發。令人興奮的是，我們也提前完成了第二階段軟骨發育不良試驗的招募。

For Encaleret, a negative allosteric modulator of the calcium sensing receptor, our Phase 3 CALIBRATE study is fully enrolled. We expect last patient last visit and top-line results in the second half of this year. In parallel, Encaleret is also being studied in hypoparathyroidism, and we announced today positive POC data for Encaleret in key expansion indication. Preliminary evidence in the ongoing POC study has demonstrated that 78% of the first nine study participants were able to achieve normal blood and urine calcium levels within five days of Encaleret administration.

對於鈣敏感受體負變構調節劑 Encaleret，我們的 3 期 CALIBRATE 研究已完全招募參與者。我們預計今年下半年將迎來最後一位患者的最後一次就診和最終結果。同時，Encaleret 也在甲狀旁腺功能減退症中進行研究，我們今天公佈了 Encaleret 在關鍵擴展適應症中的積極 POC 數據。正在進行的 POC 研究的初步證據表明，前九名研究參與者中有 78% 在服用 Encaleret 後五天內能夠達到正常的血液和尿液鈣水平。

Moving to BBP-418, an oral first-in-class disease-modifying therapy in Phase 3 development for treatment of individuals living with Limb-Girdle muscular dystrophy type 2I. We have fully enrolled FORTIFY, a Phase 3 registrational placebo-controlled study, evaluating the safety and efficacy of BBP-418. The study includes a planned interim analysis of 12 months focused on assessing a surrogate endpoint biomarker, glycosylated alpha-dystroglycan to support accelerated approval in the United States. We anticipate top-line readout from that Phase 3 interim analysis second half of this year as well.

轉向 BBP-418，這是一種處於 3 期開發的口服一流疾病改良療法，用於治療 2I 型肢帶型肌肉營養不良症患者。我們已全面參與 FORTIFY，這是一項 3 期註冊安慰劑對照研究，旨在評估 BBP-418 的安全性和有效性。該研究包括一項為期 12 個月的計畫中期分析，重點評估替代終點生物標記糖基化 α-肌肉營養不良蛋白聚醣，以支持在美國加速審批。我們預計今年下半年第三階段中期分析的結果也將公佈。

Finally, BBP-812 is an AAV9 gene therapy in development for Canavan disease, an ultrarare neurodegenerative disease that usually leads to death in the first two decades of life. Here, we are pursuing an accelerated approval approach using a single seamless registrational trial to bring this potential therapy to children with Canavan as quickly as possible. A meeting this month with the FDA reinforce the validity of our approach, which centers around the use of urine NAA in tandem with other clinical measures and the suggested BLA filing that we have laid out by end of 2026.

最後，BBP-812 是一種正在開發的 AAV9 基因療法，用於治療卡納萬病，這是一種極為罕見的神經退化性疾病，通常會在生命的前二十年導致死亡。在這裡，我們正在採取一種加速審批方式，使用單一無縫註冊試驗，盡快為患有 Canavan 的兒童提供這種潛在的治療方法。本月與 FDA 的會議強化了我們方法的有效性，該方法的核心是將尿液 NAA 與其他臨床措施結合使用，以及我們在 2026 年底前提出的建議 BLA 申請。

While this is not a very prevalent disease, we believe it's a great example of our model, targeting the disease at its source and operating leanly to enable us to go after a well-validated condition in an NPV positive manner.

雖然這不是一種非常流行的疾病，但我們相信這是我們模型的一個很好的例子，該模型針對疾病的源頭並精益運營，使我們能夠以 NPV 積極的方式追踪經過充分驗證的病症。

Okay. That's the final framing. To wrap up my comments, BridgeBio is executing well and importantly possessed of a tremendously strong foundation for the future. Number one, an unmatched collection of late-stage genetic disease businesses with favorable economic prospects; number two, a cadre of outstanding managers that are dedicated to their specific assets business and to BridgeBio; number three, a diversity of stakes, including wholly owned assets and significant stakes in GondolaBio and BridgeBio Oncology Therapeutics; number four, first choice ranking with many academics, one seeking a partner to discover and develop new medicines; and number five, a culture that is distinctive for most biotechs I have seen and that is decentralized independent thinking and live the value that every minute count for the patients we serve.

好的。這就是最終的構圖。總而言之，BridgeBio 表現得很好，重要的是，它為未來奠定了極其堅實的基礎。第一，擁有無與倫比的晚期遺傳疾病業務集合，且具有良好的經濟前景；第二，一群致力於特定資產業務和BridgeBio的優秀管理人員；第三，股權多元化，包括全資資產以及在GondolaBio和BridgeBio Oncology Therapeutics的大量股權；第四名，第一選擇排名，擁有眾多學者，尋求合作夥伴來發現和開發新藥；第五，我見過的大多數生物技術公司都具有獨特的文化，即分散的獨立思考，並踐行為我們服務的患者每一分鐘都很重要的價值。

We're excited to work with you, our investors, to continue to build this company and to keep you up to date on our progress for patients.

我們很高興能與你們，我們的投資者，一起繼續建立這家公司，並讓你們了解我們為病人所取得的進展。

With that, I'll hand it over to Matt to walk through Attruby's commercial performance in more detail.

接下來，我會把主題交給 Matt，讓他更詳細地介紹 Attruby 的商業表現。

Thanks, Neil. To add to your comments, the launch of Attruby is off to a strong and encouraging start. Let me walk through some of the highlights in terms of what we are seeing out in the marketplace and what we think is driving the rapid momentum we've built in just a few short months.

謝謝，尼爾。補充您的評論，Attruby 的推出有一個強勁而令人鼓舞的開端。讓我來介紹一下我們在市場上看到的一些亮點，以及我們認為在短短幾個月內推動我們快速發展的動力。

The first highlight is the early uptake across all major prescriber and patient segments. As shared in our press release, 2,072 unique patients have received a prescription for Attruby through April 25, and 756 unique healthcare providers have written at least one prescription. This early adoption spans all physician segments, including large academic centers, regional amyloid clinics, high-volume heart failure specialists, and community cardiologists.

第一個亮點是所有主要處方醫生和患者群體的早期採用。正如我們在新聞稿中分享的那樣，截至 4 月 25 日，已有 2,072 名獨立患者收到了 Attruby 的處方，並且 756 名獨立醫療保健提供者開出了至少一張處方。早期採用涵蓋所有醫生部門，包括大型學術中心、區域澱粉樣蛋白診所、大量心臟衰竭專家和社區心臟病專家。

Importantly, we're seeing Attruby across the full spectrum of patients, wild type and variant, newly diagnosed as well as switches from partial stabilizers. The fact that these different patient types are all gaining timely access and that prescribers are already writing for multiple patients gives us confidence that we're building a strong foundation for Attruby.

重要的是，我們看到 Attruby 遍及各種患者，包括野生型和變異型、新診斷的患者以及部分穩定劑的轉換患者。事實上，這些不同類型的患者都可以及時獲得治療，處方醫生已經為多名患者開立處方，這讓我們有信心為 Attruby 奠定堅實的基礎。

So let's discuss what is driving the uptake. It comes down to strong clinical endpoints paired with our transparent patient-first support programs. Starting with the clinical data, Attruby is the only therapy for ATTR-CM that has demonstrated a separation from placebo as early as three months.

那麼讓我們來討論一下推動這一趨勢的因素。它歸結為強大的臨床終點與我們透明的患者優先支持計劃相結合。從臨床數據來看，Attruby 是唯一一種早在三個月內就證明與安慰劑有區別的 ATTR-CM 療法。

No other medicine has demonstrated that and it's not just about efficacy. Attruby also positively impacts KCCQ scores, which directly correlate with better quality of life for patients. That's resonating deeply with physicians who want to make a meaningful impact on their patients' daily lives and patients who want to keep their functional activity levels.

沒有其他藥物證明這一點，而且這不僅僅與療效有關。Attruby 也對 KCCQ 分數產生正面影響，這與患者的生活品質改善直接相關。這引起了那些想要對患者的日常生活產生有意義影響的醫生和想要保持功能活動水平的患者深深的共鳴。

Second, let's look at hospitalization rates. Attruby is the only ATTR-CM therapy to demonstrate 50% relative risk reduction in cardiovascular hospitalization rates. This includes a statistically significant reduction in composite of mortality and hospitalization in the varying ATTR-CM population, a subgroup widely regarded as among the most difficult to treat. This was highlighted in the recent variant subgroup data presented at ACC.

其次，我們來看看住院率。Attruby 是唯一能夠將心血管住院率相對風險降低 50% 的 ATTR-CM 療法。這包括不同 ATTR-CM 族群的死亡率和住院率的統計顯著下降，該亞群被廣泛認為是最難治療的亞群之一。這在 ACC 上展示的最新變異亞組數據中得到了強調。

And third, affordability. Attruby is the most cost-effective therapy in ATTR-CM available, 10% less expensive than tafamidis and 50% less expensive than vutrisiran.

第三，負擔能力。Attruby 是 ATTR-CM 領域最具成本效益的療法，比 tafamidis 便宜 10%，比 vutrisiran 便宜 50%。

It's not just about the [whack] price though. Attruby has a free trial program for all patients regardless of insurance status and lifetime free drug for patients who participated in our pivotal trials. That matters to physicians, patients, and payers, especially when coupled with the three-month onset of treatment effect and 50% reduction in cardiovascular hospitalization at 30 months. No other ATTR-CM therapy checks all of these boxes.

但這不僅僅與價格有關。Attruby 為所有患者提供免費試用計劃，無論其保險狀況如何，並為參與我們關鍵試驗的患者提供終身免費藥物。這對醫生、患者和付款人來說都很重要，尤其是考慮到治療效果在三個月內就顯現，並且 30 個月內心血管住院率降低 50%。沒有其他 ATTR-CM 療法能夠滿足所有這些要求。

BridgeBio has a commercial model that is different and it's working. Attruby will be followed by three additional commercial launches in 2026 and '27. What really differentiates BridgeBio though is how we bring products to market. There are no convoluted value-based contracts, no vague promises of future rebates contingent on volume thresholds. What we offer is simple, transparent, and built around the people who matter most, patients and their care teams. And we're hearing from the field that this clarity paired with strong clinical data is helping accelerate adoption.

BridgeBio 擁有一種與眾不同的商業模式，而且它正在發揮作用。Attruby 號之後也將於 2026 年和 2027 年進行另外三次商業發射。但 BridgeBio 的真正與眾不同之處在於我們如何將產品推向市場。沒有複雜的基於價值的合同，也沒有根據數量門檻確定未來回扣的模糊承諾。我們提供的服務簡單、透明，並且圍繞著最重要的人——患者及其照護團隊而建構。我們從現場獲悉，這種清晰度加上強大的臨床數據有助於加速採用。

And once an HCP decides to prescribe Attruby, we make it easy to get the medication to patients. Patients can receive a treatment within 48 hours. We have two world-class specialty pharmacies in the Attruby network and also allow physicians to fill prescriptions in their own in-house pharmacies. All of this investment is paid off. We are gaining share in the crucial first-line setting, and our conversion to paid rate and time to pay prescription is well ahead of industry benchmarks.

一旦 HCP 決定開出 Attruby，我們就會輕鬆地將藥物送到患者手中。患者可以在48小時內接受治療。我們在 Attruby 網路中擁有兩家世界一流的專業藥房，並且還允許醫生在自己的內部藥房配藥。所有這些投資都已獲得回報。我們在關鍵的一線治療領域中的份額正在不斷擴大，我們的付費轉換率和處方支付時間遠遠領先於行業基準。

Looking towards the future, our focus heading into the rest of the year is as follows: continue marching towards our long-term goal of [30%] to 40% share of the ATTR-CM market, and increased share in the critical first-line setting, which is a leading indicator of launch success. While these early signs are encouraging and they reflect not just the strength of the product, but the preparation and the commitment of the broader BridgeBio team, we are also excited about what's ahead and remain focused on reaching as many patients as possible as quickly as possible.

展望未來，我們在今年剩餘時間的重點是：繼續朝著我們的長期目標邁進，即在 ATTR-CM 市場中佔據 [30%] 至 40% 的份額，並增加在關鍵的一線環境中的份額，這是發布成功的領先指標。雖然這些早期跡象令人鼓舞，它們不僅反映了產品的實力，也反映了更廣泛的 BridgeBio 團隊的準備和承諾，但我們也對未來感到興奮，並將繼續專注於盡快接觸盡可能多的患者。

With that, I'll turn it over to Tom for a review of the financials.

說完這些，我將把財務狀況交給湯姆進行審查。

Thank you, Matt, and good afternoon, everyone. Q1 2025 marked BridgeBio's first full quarter of net product revenue from the Attruby US commercial launch, major milestones for the company and a significant step forward in our evolution into a fully integrated genetic medicine business.

謝謝你，馬特，大家下午好。2025 年第一季是 BridgeBio 從 Attruby 美國商業發布中獲得淨產品收入的第一個完整季度，這是該公司的重要里程碑，也是我們轉型為完全整合的基因醫學業務的重要一步。

I'll now walk through the financial highlights for the first quarter of 2025. Please note that our commentary on today's call will focus on GAAP financial measures, unless otherwise indicated. Total revenues were $116.6 million for Q1 2025 and consists of Attruby net product revenue and license and services revenue. Attruby net product revenue was $36.7 million, driven by strong demand across all major prescribers and patient segments. License and services revenue was $79.9 million in the quarter, primarily driven by the recognition of the $75 million regulatory milestone related to Beyonttra's EU approval. Also in Q1, we received Beyonttra approval in Japan, for which we expect to recognize a $30 million milestone in the second quarter.

現在我將介紹 2025 年第一季的財務亮點。請注意，除非另有說明，我們對今天電話會議的評論將集中於 GAAP 財務指標。2025 年第一季總收入為 1.166 億美元，包括 Attruby 淨產品收入以及授權和服務收入。Attruby 淨產品收入為 3,670 萬美元，這得益於所有主要處方醫生和患者群體的強勁需求。本季許可和服務收入為 7,990 萬美元，主要得益於與 Beyonttra 獲得歐盟批准相關的 7,500 萬美元監管里程碑的認可。此外，在第一季度，我們獲得了日本 Beyonttra 的批准，我們預計第二季將實現 3000 萬美元的里程碑。

Total operating expenses for the first quarter of 2025 were $218.4 million (sic - see press release, "$221 million") compared to $210.2 million (sic - see press release, "$210.8 million") in the same period last year. This increase reflects our continued investment in the Attruby brand and our advancing late-stage pipeline. Included in our total operating expenses was $29.4 million of stock-based compensation expense compared to $28.9 million in the first quarter of 2024. Looking forward, we expect only modest growth in quarterly operating expenses for the remainder of the year with an offset to total cash burn provided by Attruby sales in the US and Beyonttra ex US.

2025 年第一季的總營運費用為 2.184 億美元（原文如此 - 參見新聞稿，「2.21 億美元」），而去年同期為 2.102 億美元（原文如此 - 參見新聞稿，「2.108 億美元」）。這一成長反映了我們對 Attruby 品牌的持續投資以及我們不斷推進的後期研發管線。我們的總營運費用包括 2,940 萬美元的股票薪酬費用，而 2024 年第一季為 2,890 萬美元。展望未來，我們預計今年剩餘時間的季度營運費用只會小幅成長，但 Attruby 在美國和 Beyonttra 在美國以外的銷售將抵銷總現金消耗。

R&D expense for the first quarter of 2025 was $111.4 million compared to $141 million in the same period last year. This decrease was largely due to the strategic carve-out of our oncology business and early-stage research programs allowing us to focus resources on the Attruby launch and late-stage pipeline. SG&A expense for the first quarter of 2025 was $106.4 million compared to $65.8 million in the same period last year. This increase was driven by the full scale commercial rollout of Attruby, including field team deployment, payer engagement, and patient support infrastructure.

2025 年第一季的研發費用為 1.114 億美元，去年同期為 1.41 億美元。這一下降主要是由於我們的腫瘤業務和早期研究項目的策略性剝離，使我們能夠將資源集中在 Attruby 的發布和後期研發管線上。2025 年第一季的銷售、一般及行政費用為 1.064 億美元，去年同期為 6,580 萬美元。這一增長是由 Attruby 的全面商業推廣推動的，包括現場團隊部署、付款人參與和患者支援基礎設施。

Restructuring expense for the first quarter of 2025 was $0.6 million compared to $3.4 million in the same period last year. We ended the quarter with $540.6 million in cash and cash equivalents, which does not include $105 million in regulatory milestone payments anticipated in Q2 for ex US approvals of Beyonttra.

2025 年第一季的重組費用為 60 萬美元，而去年同期為 340 萬美元。本季結束時，我們的現金和現金等價物為 5.406 億美元，其中不包括預計在第二季度為獲得美國以外 Beyonttra 批准而支付的 1.05 億美元監管里程碑付款。

We believe we are well financed for the continued execution of the Attruby launch and deliver on key milestones from the pipeline this year. We look forward to sharing additional commercial updates throughout the year and the top line data from our three Phase 3 programs over next year with ADH1 and LGMD2I in the second half of 2025, achondroplasia in early 2026.

我們相信，我們擁有充足的資金來繼續執行 Attruby 計畫的啟動，並在今年實現該計畫的關鍵里程碑。我們期待在全年分享更多商業更新，並在明年分享我們三個 3 期項目的頂線數據，其中 ADH1 和 LGMD2I 項目將於 2025 年下半年發布，軟骨發育不全項目將於 2026 年初發布。

With that, I'll turn the call back to Chinmay.

說完這些，我就把電話轉回給 Chinmay。

Thank you, Neil, Matt, and Tom. I'll now hand it back to the moderator to open the line for questions. (Event Instructions)

謝謝你，尼爾、馬特和湯姆。我現在將會議交還給主持人，開始回答問題。（活動須知）

(Operator Instructions) Salim Syed, Mizuho.

（操作員指示）Salim Syed，瑞穗。

Great. Congrats on the quarter guys and congrats also on your first earnings call. Maybe just one for Neil or Matt here. Obviously, the quarter came in healthier than I think people had thought going into the print. And I appreciate the commentary that you provided, Matt, on some of the dynamics there. But any sort of granularity you can provide on the tailwind and which you really deem that's working well for you here? Thank you.

偉大的。恭喜你們本季取得的成績，也恭喜你們第一次召開收益電話會議。也許這裡只有一個適合 Neil 或 Matt 的。顯然，本季的業績比人們預想的要好。馬特，我很欣賞你對那裡的一些動態所做的評論。但是，您能否提供關於順風的任何詳細信息，並且您真的認為這對您很有幫助嗎？謝謝。

Yeah, maybe I'll kick it off. Thanks, Salim. It's probably too early for some of the higher cost commercial tactics that we've invested in to show ROI right now. So hearteningly, I think a lot of the demand we're seeing to date really is a product, number one, of the differentiated clinical efficacy we have, as a reminder, and you know this the earliest separation and it's a point estimate that we've seen in the field at three months, 42% relative risk reduction against ACM and CVH in 30 months. Again, the best point estimate we've seen at 30 months, coupled with that 50% reduction in hospitalization, which turns out to be a huge leading full measure for patients who both want to live longer, but also live healthier. So I'd say that's point number one.

是的，也許我會開始。謝謝，薩利姆。對於我們投資的一些成本較高的商業策略來說，現在顯示投資回報可能還為時過早。因此，令人振奮的是，我認為我們迄今為止看到的許多需求實際上都是我們差異化臨床療效的產物，提醒一下，這是最早的分離，也是我們在三個月內在該領域看到的點估計，30 個月內相對於 ACM 和 CVH 的相對風險降低了 42%。再次，我們看到的最佳點估計是 30 個月，再加上住院率減少 50%，這對於既想活得更長又想活得更健康的患者來說是一個巨大的領先全面指標。所以我認為這是第一點。

Point number two, as you saw as well from the Pfizer call this morning, continued market growth, right? We're one-fifth diagnosed in this space, some 50,000 or so patients diagnosed. We think there's 250,000 to 300,000 in the US alone. And so physician education, coupled with there being a variety of therapeutic interventions now available, I think, is driving that growth. So that, I think, is another tailwind.

第二點，正如您今天早上從輝瑞公司的電話會議中看到的那樣，市場持續成長，對嗎？我們在這個領域確診了五分之一的患者，大約有 5 萬名患者。我們認為光在美國就有 25 萬到 30 萬。因此，我認為，醫生教育加上目前可用的各種治療幹預措施正在推動這一增長。所以我認為這是另一個順風。

And then the third is the access programs that Matt talked about. The team, I think, has done a terrific job of ensuring that patients when they are prescribed Attruby can get the medicine and stay on the medicine as long as they need to. So those would be the three salient drivers.

第三個是馬特談到的訪問程式。我認為，團隊做得非常出色，確保患者在服用 Attruby 藥物時能夠獲得藥物，並根據需要持續服用藥物。所以這就是三個突出的驅動因素。

I'd say the final thing there's a little bit of what a physician described to us the other day is Karma. Obviously, we're the only sponsor in this space that gave free drug for life for their trial participants. Unfortunately, others decided not to. We're also the only sponsor in the space running a primary prevention study, which I think many physicians view as the ultimate in trying to catch patients in this mass action condition as early as possible to do as much as we can for conditions.

我想說的最後一件事是前幾天一位醫生向我們描述的因果報應。顯然，我們是該領域唯一一家為試驗參與者提供終身免費藥物的贊助商。不幸的是，其他人決定不這樣做。我們也是該領域唯一進行一級預防研究的贊助商，我認為許多醫生認為這是儘早發現這種群體性症狀患者的最終方法，以便盡我們所能為病情做出貢獻。

So those types of things, I think, over a long period of time, will stand us in good stead as a sponsor here in a competitive space.

所以我認為，從長遠來看，這些事情將使我們作為贊助商在競爭激烈的領域中處於有利地位。

I don't know, Matt, if you'd add anything?

我不知道，馬特，你還有什麼要補充的嗎？

No, I think in this -- the people, we've spent a lot of time putting the team together both internal field to make it as easy as we can for both prescribers and for patients. And I think you're seeing the good results of that.

不，我認為在這方面——我們花了很多時間將團隊整合在一起，無論是內部還是外部，以盡可能為處方醫生和患者提供便利。我認為你已經看到了它的良好成果。

Great, thanks guys. Congrats again.

太好了，謝謝大家。再次恭喜。

Tyler Van Buren, TD Cowen.

泰勒範布倫 (Tyler Van Buren)，TD Cowen。

Hey, guys. Congrats on the stellar Attruby result as well and the ongoing progress of the pipeline. So again, the $37 million of the Attruby sales far exceeded expectations. So when you say that the paid conversion rate is well ahead, can you help quantify that? Was the time to pay quicker than a month for some patients? And how much stocking was there in the quarter?

嘿，大家好。恭喜 Attruby 取得的優異成績以及管道的持續進展。因此，Attruby 3700 萬美元的銷售額再次遠遠超出了預期。那麼，當您說付費轉換率遙遙領先時，您能幫助量化這一點嗎？對於某些患者來說，付款時間是否少於一個月？本季庫存量有多少？

Yeah. Hi Tyler, great to hear from you. I'm going to pass it on to Matt to comment more on conversion.

是的。你好，泰勒，很高興收到你的來信。我將把它傳遞給 Matt，讓他對轉換做出更多評論。

Yeah, thanks for the question. We're really happy with how conversion is tracking, and that's for both free trial to paid and also commercial prescriptions to paid. Everything regarding conversion is consistent or better than historical launches that we've studied.

是的，謝謝你的提問。我們對轉換追蹤情況感到非常滿意，無論是免費試用到付費，還是商業處方到付費。有關轉換的一切都與我們研究過的歷史發布情況一致或更好。

I will just point out Attruby trial acts like a normal prescription, HCP writes it, the pharmacy processes it and the patient gets it. And we designed our network specifically with conversion in mind, and that's what makes the access so easy. Our goal remains the same, 30% to 40% peak share. It takes up three to six years usually to hit. We're hoping we can do that a little bit faster.

我只想指出，Attruby 試驗就像普通處方一樣，HCP 開處方，藥房處理，患者拿到處方。我們在設計網路時特別考慮了轉換，這使得存取變得如此簡單。我們的目標保持不變，即 30% 至 40% 的峰值份額。通常需要三到六年的時間才能實現。我們希望能夠更快地完成這項任務。

In regards to your channel and inventory question, I mentioned we do have a limited distribution network that allows for very frequent ordering and it allows our customers, our distributors to have just-in-time inventory. So there's no need for them to really hold large quantities. And so typically, I would say that this small group of distributors holds about one to two weeks of inventory.

關於您的通路和庫存問題，我提到我們確實有一個有限的分銷網絡，可以進行非常頻繁的訂購，並且可以讓我們的客戶和分銷商擁有準時庫存。所以他們沒必要真正持有大量庫存。因此，通常情況下，我會說這一小群分銷商持有大約一到兩週的庫存。

Hi Tyler, this is Tom. Let me just put a finer point on the inventory question. As I mentioned in the prepared remarks, the sales in the quarter were primarily driven by demand. So we've seen only a minor impact on inventory on total sales in the quarter. So really demand-driven here.

你好，泰勒，我是湯姆。讓我更詳細地闡述庫存問題。正如我在準備好的發言中提到的，本季的銷售主要由需求驅動。因此，我們發現庫存對本季總銷售額的影響很小。所以這確實是需求驅動的。

Yeah. Thanks, Tyler. I know you had another question. So happy to answer more about this or go to your next question.

是的。謝謝，泰勒。我知道你還有其他問題。很高興能就此問題進行更多解答或回答您的下一個問題。

Mani Foroohar, Leerink Partners.

Mani Foroohar，Leerink Partners。

Hey, guys. Congrats on the quarter. Clearly blew out expectations despite having shown pretty good scripts repeatedly. And I want to dive in on the script numbers. I'm looking at the incremental scripts on a weekly basis through that first update on January 10, or in the 60s per week? And then through the update you gave on an incremental basis on February 17, that was running north of 100 scripts a week.

嘿，大家好。恭喜本季取得佳績。儘管已經多次展示了相當不錯的劇本，但顯然還是超出了預期。我想深入了解劇本數量。我每週都會查看增量腳本，直到 1 月 10 日第一次更新，還是每週 60 次？然後，透過您在 2 月 17 日逐步提供的更新，每週執行的腳本數量已超過 100 個。

And again, running through from 12 and [four] weeks into launch and 22 weeks in the launch, i.e., from February 17 to April 25, you're still running at about 100, 109 script per week as reported.

再說一次，從發布後的 12 週（4 週）到發布後的 22 週，即從 2 月 17 日到 4 月 25 日，您每週仍會運行大約 100 到 109 個腳本，正如報告的那樣。

I know it's just math and then a lot of nuance that goes into script reporting. Could you give us a sense of what the tariff on velocity of new patient scripts that you're seeing right now? And sort of what is that -- so what is the kind of direction of travel out there? And I have a follow-up question.

我知道這只是數學問題，腳本報告中還涉及很多細微差別。您能否向我們介紹一下您目前看到的新病患處方速度的關稅是多少？那是什麼──那裡的旅行方向是什麼樣的？我還有一個後續問題。

Great. I can take that. Thank you for the question. I mean I think starting out, the thesis really -- again, it remains unchanged. TPL levels go up with Attruby and it's the only year complete stabilizer on the market.

偉大的。我可以接受。謝謝你的提問。我的意思是，我認為從一開始，論點確實——再說一次，它保持不變。TPL 水平隨著 Attruby 而上升，它是市場上唯一全年完整的穩定劑。

When we sort of take that thesis and then dive into, okay, well, what's happening and then how do we think that's going to impact things going forward? But on the over month growth rate in the treatment-naïve section has been very strong, and that's been across all segments. So this is in the large centers, this is in the community. That's across all types of patients, variant wild-type, there hasn't been a sort of one area or another that's done better. We've sort of seen this across the board.

當我們接受這個論點並深入研究時，好吧，好吧，發生了什麼，然後我們認為這將如何影響未來的事情？但從月度來看，未經治療部分的成長率非常強勁，而且所有部分都是如此。這是在大型中心，這是在社區。對所有類型的患者，包括變異野生型，都沒有發現某一領域表現較好的。我們已經全面看到了這種情況。

And so then when we look at sort of the two groups, we have the treatment-naïve patients, that's our focus. And that will continue, we believe, to grow over time as it has been. Then you have the switch patients. Of course, when we launched, we had 100% of the switch share because we were the only option you could switch to as new -- now with new entrants in the market, that's going to evolve over time because they are now more choices. And so I think the data and how we report will evolve over time as well based on that. But that's kind of where we are and how we see it moving forward.

因此，當我們觀察這兩組患者時，我們發現未接受過治療的患者是我們的重點。我們相信，隨著時間的推移，這一趨勢將繼續增長。然後你就有了轉換病人。當然，當我們推出產品時，我們擁有 100% 的轉換份額，因為我們是您作為新產品可以轉換的唯一選擇——現在隨著新進入市場的公司，這種情況會隨著時間的推移而發展，因為現在有更多的選擇。因此我認為數據和我們的報告方式也會隨著時間的推移而發展。但這就是我們現在的狀況以及我們對未來發展的看法。

Great. That's helpful. And I have sort of a non-TTR question to everyone's surprised. When you think about Limb-Girdle data coming later on this year, which, obviously, we've been discussing a number of calls. This is not the first time you guys have brought it up.

偉大的。這很有幫助。我有一個非 TTR 問題，令大家感到驚訝。當您想到今年稍後將發布的 Limb-Girdle 數據時，顯然我們已經討論過很多次了。這不是你們第一次提起這個問題。

We talked about it at my conference [a couple years] running, about the filability of that data set or whether a later dataset in the same study will be required. Do any of the changes of FDA concern you around your ability to file on a biomarker? Do you think you have to show a distinct level of clear clinical improvement on a non-biomarker/functional basis? Where are we in terms of the viability of that biomarker for Limb-Girdle?

我們在我連續幾年的會議上討論了這個問題，關於該資料集的可保存性，或者是否需要同一項研究中的後續資料集。FDA 的任何變化是否會讓您擔心提交生物標記申請的能力？您是否認為您必須在非生物標記/功能基礎上顯示出明顯的臨床改善程度？就肢帶症候群的生物標記的可行性而言，我們處於什麼位置？

Yeah. I can take that. Mani, we haven't met with the agency of late on the LGMD2I program, but we have had some close to 10 meetings now over the course of the last month as it pertains to either pipeline at Gondola or a pipeline here at Bridge, probably the latest meeting was in and around the Canavan program.

是的。我可以接受。馬尼，我們最近沒有與該機構就 LGMD2I 項目舉行會議，但在過去的一個月裡，我們已經舉行了近 10 次會議，因為它涉及 Gondola 的管道或 Bridge 的管道，最近一次會議可能是在 Canavan 項目及其周邊舉行的。

And I have seen nothing but, I would say, a positive inclination toward trying to get first-in-class medicines that target path a mechanism like our drug for LGMD2I onto the marketplace as quickly as possible to benefit the children that are suffering from these conditions. And so I don't think the rather clear guidance that we've gotten from the agency to date in and around filability, around glycosylation of ADG is going to change. I really don't.

而我想說的是，我看到的只是一種積極的傾向，即嘗試盡快將針對類似我們治療 LGMD2I 的藥物這樣的機制的一流藥物推向市場，以造福那些患有這些疾病的兒童。因此，我認為迄今為止我們從該機構獲得的有關可歸檔性和 ADG 糖基化的相當明確的指導不會改變。我真的不知道。

I have been on the record saying I think it's going to be a problem if we go the right way and hit our primary endpoint on ADG, but we go the wrong way on all clinical measures. Obviously, I don't think that's going to be the case based on what we observed in Phase 2, which was albeit an open-label study against natural history where we saw improvements in measures of ambulation and other clinical outcomes.

我曾公開表示，如果我們走對了路，達到了 ADG 的主要終點，但在所有臨床測量上卻走錯了路，那麼這將是一個問題。顯然，根據我們在第二階段的觀察結果，我認為情況並非如此，儘管這是一項針對自然史的開放標籤研究，但我們看到了步行指標和其他臨床結果的改善。

So I do believe these things will move toward the positive or at least a few will move toward the positive, obviously, modified North Star, North Star which is historically but the gold standard in these conditions. It's all noise over the course of the first 12 months. So what is the point estimate goes one way or the other. I think natural history has made that very clear. And that is why we're running the confirmatory trial.

所以我確實相信這些事情會朝著正面的方向發展，或至少有些會朝著正面的方向發展，顯然，修改後的北極星，北極星在歷史上是這些條件下的黃金標準。在最初的 12 個月裡，一切都只是噪音。那麼點估計會朝哪個方向發展呢？我認為自然史已經明確地表明了這一點。這就是我們進行確認試驗的原因。

This is effectively a nesting trial design that goes on for another 2.5 years post the readout later this year.

這實際上是一個嵌套試驗設計，將在今年稍後讀數後再持續 2.5 年。

So I mean, long story short, we continue to believe that is going to be an approvable endpoint in terms of ADG glycosylation increase.

所以，長話短說，我們仍然相信這將是 ADG 糖基化增加的一個可批准的終點。

Tyler Van Buren, TD Cowen.

泰勒範布倫 (Tyler Van Buren)，TD Cowen。

I just had a second question that I meant to ask. But just the ADH1 and Limb-Girdle Phase 3 is reading out during the second half of the year, clearly level high probability success. So can you -- it would be great to hear you elaborate on why investors should be excited about and more importantly, focus on those opportunities beyond the ongoing Attruby launch. What do you think the magnitude of those opportunities could be?

我只是想問第二個問題。但在下半年才會讀出 ADH1 和 Limb-Girdle 第 3 階段的數據，顯然成功的可能性很高。那麼，如果您能詳細解釋為什麼投資者應該對正在進行的 Attruby 推出之外的機會感到興奮，並且更重要的是關注這些機會，那將是一件非常愉快的事情。您認為這些機會有多大？

Yeah, good question, Tyler. As I was suggesting, we continue to be heartened by both the clinical operational positioning of LGMD2I, ADH1 and our achondroplasia trials as well as the potential to help a broad swath of patients. And I think from an investor standpoint, one asked to consider the sheer size of these marketplaces, LGMD2I as we've discussed, some 7,000 to 8,000 patients between the US and EU. That's a substantial market size as compared to any of the other muscular dystrophy programs that you see out there at the price points that are attended in the space.

是的，泰勒，問得好。正如我所說的，我們繼續對 LGMD2I、ADH1 和軟骨發育不全試驗的臨床操作定位以及幫助廣大患者的潛力感到振奮。我認為從投資者的角度來看，人們需要考慮這些市場的規模，正如我們所討論的 LGMD2I，美國和歐盟之間大約有 7,000 到 8,000 名患者。與市場上同等價位的任何其他肌肉萎縮症治療項目相比，這是一個相當大的市場規模。

And with ADH1, a rather much more prevalent condition, maybe 10,000 to 12,000 patients in the United States alone as suggested by statistical genetics methodology, some 4,000 or so already identified to date, that's going to be really a program associated with the market build and finding new patients. And we've already shown that we can do that in some of the sequencing efforts we've undertaken within the non-surgical [hypopara] community where we're reliably finding something like 20% to 25% of patients actually harbor the gain of function mutations in the calcium sensing receptor.

對於 ADH1 這種更為普遍的疾病，根據統計遺傳學方法，僅在美國就有大約 10,000 到 12,000 名患者，迄今為止已確診約 4,000 名患者，這將是一個與市場建設和尋找新患者真正相關的項目。我們已經證明，我們可以在非手術 [甲狀旁腺功能減退] 社區開展的一些測序工作中做到這一點，我們可靠地發現大約 20% 到 25% 的患者實際上攜帶鈣敏感受體的功能獲得突變。

So I think two very exciting commercial opportunities, two very exciting opportunities for first-in-class medicines for patients there. And then I don't think I need to belabor the achondroplasia hypothesis where we feel we have an exciting oral best-in-class, potentially best-in-class option that could provide differential safety differential legacy because it targets this well-described condition at its source, driving differential both changes in growth and then also importantly, impact on parameters that this community care is a great deal about like proportionality and the like. And you know the size of that market from the (inaudible) launch that's ongoing. So I think all three very exciting opportunities.

因此，我認為這是兩個非常令人興奮的商業機會，兩個為患者提供一流藥物的非常令人興奮的機會。然後，我認為我不需要詳細闡述軟骨發育不全假說，我們認為我們有一個令人興奮的口服最佳方案，可能是同類最佳方案，它可以提供差異化的安全差異化遺產，因為它針對這種描述明確的疾病的根源，推動差異化的增長變化，然後同樣重要的是，對社區護理所關注的參數的影響，比如比例等。您可以從正在進行的（聽不清楚）發布中了解該市場的規模。所以我認為這三個機會都非常令人興奮。

Biren Amin, [Equity Research Health].

比倫阿明，[股票研究健康]。

Congrats on the quarter and really good launch out of the gate. I guess my question is what's resonating with health care professionals as it relates to the Attruby launch? And what are the biggest hurdles that you're seeing for adoption?

恭喜本季取得的成績，開局非常順利。我想我的問題是，Attruby 的推出與醫療保健專業人員有何共鳴？您認為採用該技術的最大障礙是什麼？

And then maybe a second question, what was the gross to net for the quarter? And any impact that you've seen from the Part D redesign?

然後也許還有第二個問題，本季的毛利與淨利是多少？您認為 D 部分重新設計有何影響？

Hi Biren, thanks for the question. I'm going to pass it on to Matt to discuss the HCP piece, and then I'll throw it to Tom to discuss the [question on that] piece.

你好，Biren，謝謝你的提問。我將把它傳給馬特來討論 HCP 部分，然後我會把它交給湯姆來討論[關於那個]部分的問題。

Yeah, thanks. And I guess, there's sort of two parts to this. The feedback from the physician segment has been positive as we've discussed. Our messaging around, we call it, the [342.50], but that ability to separate as early as three months and getting results around the hospitalization all-cause mortality that we've described.

是的，謝謝。我想，這可以分成兩個部分。正如我們所討論的，醫生方面的回饋是正面的。我們傳遞的訊息，我們稱之為 [342.50]，但這種能力最早可以在三個月內分離，並獲得我們所描述的住院全因死亡率的結果。

Physicians want to see drug work really fast, and they want to be able to tie it to hard outcomes. Of course, patients do, too. So this is what gives people confidence. And so those messages have been resonating extremely well and I think that's resulted in physician trying Attruby, but then also then repeating it, right? Those are the reasons that you sort of try a medication and then when you see how well people are doing on it, then that encourages you to try it again. So we've seen a lot of repeat prescribers across a lot of the different segments as well.

醫生希望看到藥物快速起效，並且希望能夠將其與實際結果聯繫起來。當然，患者也一樣。這就是給予人們信心的原因。因此，這些訊息引起了極大的共鳴，我認為這導致醫生嘗試 Attruby，然後又重複使用，對嗎？這些就是您嘗試某種藥物的原因，當您看到人們對該藥物的療效如何時，這會鼓勵您再次嘗試。因此，我們在許多不同領域也看到了許多重複開處方的醫生。

And I don't think our expectations were otherwise are going to change anytime soon. We have very strong data package, and we also have very strong programs to help patients get on the medication once that prescription has been written.

我認為我們的預期不會很快改變。我們擁有非常強大的資料包，我們也有非常強大的程式來幫助患者在開出處方後立即獲得藥物。

And then I'll pass it to Neil to add on.

然後我會把它傳給 Neil 來補充。

Yeah, maybe I'll just elaborate on that for a second before I throw it over to Tom. I think the ever-increasing availability of data like most recently, Biren, I think we've talked about the variant data. The fact that you could achieve a hazard ratio of 0.41 with statistical significance in that relatively small subpopulation. And as someone mentioned earlier, the sickest of subpopulations continues to reinforce that differential levels of stabilization can lead to ever better outcomes. And we're starting to find that that's resonating.

是的，在把它交給湯姆之前，我可能會詳細解釋這一點。我認為最近數據的可用性不斷增加，Biren，我想我們已經討論過變體數據。事實上，在相對較小的亞群體中，你可以實現具有統計意義的 0.41 的風險比。正如之前有人提到的那樣，病情最嚴重的亞群繼續證明，不同程度的穩定可以帶來更好的結果。我們開始發現這引起了共鳴。

We've got a bevy of Serum TTR associated literature coming out suggesting that ever higher levels of Serum TTR correlate with ever lower levels of downstream hospitalization and mortality. The connecting of the dots between ever better stabilization and all of these downstream outcomes, I think, is what's starting to resonate with the physician community on top of what Matt suggested the backbone, which is the 342.50.

我們有大量與血清 TTR 相關的文獻表明，血清 TTR 水平越高，下游住院率和死亡率就越低。我認為，將日益增強的穩定性和所有這些下游結果聯繫起來，是開始引起醫生群體共鳴的因素，而馬特所建議的支柱是 342.50。

Tom, do you want to talk about [GKN]?

湯姆，你想談談[吉凱恩]？

Yes, sure. On gross to net, specifically. So as we said before, the way to think about this is the floor is going to be the mandatory IRA rebate of 20%. And on top of that, you're going to add something like you'd see in other categories for contracting is not really happening as that's what we're seeing here. I would say, given where we are in launch early innings, we should expect some variability here quarter on quarter.

是的，當然。具體來說，就是總額與淨額之比。因此，正如我們之前所說，考慮這個問題的方式是，最低限額將是 20% 的強制性 IRA 返利。除此之外，您還要添加一些類似於在其他類別中看到的內容，因為簽約實際上並沒有發生，因為這就是我們在這裡看到的。我想說，考慮到我們處於初期階段，我們應該預期季度與季度之間會出現一些變化。

This quarter, gross to net trended slightly favorable versus what we were expecting. We also saw a slightly lower use of our first month free program versus what we were expecting, and we saw slightly lower use of our patient assistance program and what we're expecting.

本季度，毛利率與淨利率的趨勢比我們預期的略有改善。我們也發現，第一個月免費計畫的使用率略低於我們的預期，病患援助計畫的使用率也略低於我們的預期。

All these three things together actually converge and cause slightly better net revenue per unit than what we were expecting, but we expect all of this to normalize throughout the course of the year.

所有這三件事實際上結合在一起，導致每單位淨收入略好於我們的預期，但我們預計所有這些都會在全年恢復正常。

Just to add that the results are reflective of strong underlying demand for Attruby and execution of our commercial team. And as Matt mentioned, not a lot of inventory or anything like that.

需要補充的是，這些結果反映了對 Attruby 的強勁潛在需求以及我們商業團隊的執行力。正如馬特所提到的，庫存或類似的東西並不多。

Cory Kasimov, Evercore.

科里·卡西莫夫（Cory Kasimov），Evercore。

So it's great to see Attruby's off to such an impressive start, and you kind of alluded to this in a prior response, but now that you're in the market for over a quarter. How are you thinking about new patient starts for the category going forward? I know previously, you've talked about numbers like 2,000 to 3,000 restarts per quarter, but that's already looking pretty conservative. So any kind of new color there would be appreciated.

因此，很高興看到 Attruby 取得如此令人印象深刻的開局，您在之前的回應中也提到了這一點，但現在您已經進入該市場超過一個季度了。您如何看待未來該類別的新患者開始出現？我知道之前您談到每季重啟 2,000 到 3,000 次這樣的數字，但這看起來已經相當保守了。因此，任何一種新顏色都會受到歡迎。

Yeah. Cory, thanks for the question. I think you're kind of thinking about it like we're thinking about it. It does seem to keep going up every quarter. And if you look historically, that's different. I think it is ramping a bit faster, but that also makes sense because you're getting new products in the market. So the more companies that launch a product into a space that creates a higher share of voice.

是的。科里，謝謝你的提問。我認為您和我們的想法是一樣的。它似乎每個季度都在持續上漲。如果你從歷史角度來看，情況就不同了。我認為它的成長速度會更快一些，但這也是有道理的，因為你正在向市場推出新產品。因此，將產品推向更高市場份額的公司的數量越多。

And I think that gets people educated either to go get screened or for physicians to think to themselves, I mean, if you're a cardiologist and you have [test pet] patients, and you're not thinking to yourself, hey, some of these must be ATTR-CM patients. All of these companies are discussing and educating, talking about it, it gets people looking. And if you look, you find it, it's -- it's not that uncommon.

我認為這可以讓人們接受教育，去接受篩檢，或讓醫生自己思考，我的意思是，如果你是心臟科醫生，你有 [測試寵物] 患者，而你沒有想到，嘿，其中一些一定是 ATTR-CM 患者。所有這些公司都在討論、教育和談論這個問題，以引起人們的注意。如果你仔細觀察，你會發現，這並不罕見。

Most cardiologists have some patients that need to be treated. So I think that we don't see that stopping anytime soon. the market could easily be a $20 billion market, and we're not there yet. So there's still a lot of patients who are out there who have not been diagnosed but I think you're going to see continued high numbers of people being diagnosed now with that interest is just continuing to grow.

大多數心臟科醫生都有一些需要治療的病人。所以我認為這種情況不會很快停止。這個市場很容易就能達到 200 億美元的規模，但我們還沒有達到這個目標。因此，仍有許多患者尚未被診斷，但我認為，隨著人們對此的興趣不斷增長，你會看到越來越多的人被診斷出來。

Yeah. And Cory, just to add one last thing. A lot of the new diagnosis is coming from the high-volume heart failure clinics that Matt mentioned in his marks. And obviously, an oral small module stabilizer is a great fit for those patients.

是的。科里，我再補充一點。許多新的診斷來自馬特在他的成績中提到的大型心臟衰竭診所。顯然，口服小模組穩定器非常適合這些患者。

Greg Harrison, Scotiabank.

加拿大豐業銀行的格雷格·哈里森。

Congrats on the huge start to the launch. So in our initial modeling conversations with you guys, our takeaway was that your expectation was for initial uptake to primarily or almost exclusively from newly diagnosed patients. But to get to the revenue number you've reported, you have to get most of them by our math. So assuming that's not the case, is this a function of much larger market growth than you expected? Or are you getting a large percentage or maybe even most of your patients as tafamidis switches. And if that's the case, how do you expect this trend to evolve from here?

祝賀發布會取得圓滿成功。因此，當我們與你們進行初步建模對話時，我們得出的結論是，你們的期望是最初的吸收主要或幾乎完全來自新診斷的患者。但要得到您報告的收入數字，您必須透過我們的計算來獲得其中的大部分。那麼假設情況並非如此，這是否是市場成長幅度遠超過您的預期的結果？或者您是否讓很大比例甚至大多數患者改用他法唑胺。如果事實確實如此，您認為這股趨勢今後將如何發展？

Hi, Greg. Thank you so much for the question. So I think as we have said consistently, our focus at launch has been the treatment-naïve market. Very early on, we got a few more switch patients than we expected, but that started to normalize. Focus continues to be new treatment naive patients, and I think Matt mentioned in his remarks that there we are seeing consistent monthly growth gradually.

你好，格雷格。非常感謝您的提問。因此我認為，正如我們一直所說的那樣，我們推出產品的重點是未接受治療的市場。一開始，我們接收的轉換病人比我們預期的多，但這種情況開始恢復正常。重點仍然是新的未接受過治療的患者，我認為馬特在他的評論中提到，我們看到了每月持續的增長。

In terms of what's driving the number, I think it's sort of all of the above, right? I think it's a little bit of the market growth, obviously, strong demand and strong conversion. Maybe I'll throw it on to Matt, and he can talk a little bit more about the factors driving conversion and the market size.

至於是什麼因素導致了這個數字，我認為以上所有因素都有，對嗎？我認為這是市場成長的一部分，顯然，需求強勁，轉換率也強勁。也許我會把它交給馬特，他可以多談談推動轉換和市場規模的因素。

Yeah. No, I think you hit it well, Chinmay. I don't know that I would add that much. It's a little tricky when you try to figure out who a switch patient is. They can -- they look like a new patient most of the time. So I think it's a little -- be a little careful trying to determine who's the switch and who's new.

是的。不，我認為你打得很好，Chinmay。我不知道我是否會添加那麼多。當你試圖弄清楚誰是轉換病人時，這有點棘手。他們大多數時候看起來都像個新病人。所以我認為這有點——要小心一點，確定誰是轉換者，誰是新人。

Our focus is on the newly diagnosed. We do see switch patients come in, and sometimes we can tell that they're a switch patient. Other times, it's not clear. But I think as you think about the rest of '25 moving into '26, I think the market continues to grow. It's been growing double digits now for quite a long time. And this past quarter, one of the best we've seen.

我們的重點是新診斷的患者。我們確實看到交換病人進來，有時我們可以判斷他們是交換病人。其他時候，情況就不清楚了。但我認為，當你考慮 25 年剩餘時間進入 26 年時，我認為市場將繼續成長。相當長一段時間以來，它一直保持兩位數的成長。而剛剛過去的這個季度是我們見過的最好的一個季度。

I would not expect that to disappear anytime soon. There will be switch patients who want Attruby, and there will be a lot of newly diagnosed patients that want Attruby for all the reasons we've discussed, whether it's the data itself, the programs we offer. I think we're very optimistic about all of that.

我並不期望它會很快消失。會有一些轉換的患者想要 Attruby，也會有很多新診斷的患者因為我們討論過的所有原因想要 Attruby，無論是數據本身，還是我們提供的程序。我認為我們對這一切都非常樂觀。

Paul Choi, Goldman Sachs.

高盛的保羅·崔（Paul Choi）。

Congratulations on knocking it out of the park in your first quarter here on the launch. I want to shift gears for a minute, maybe just ask a policy question, which is to think about as Attruby growth and scale over the coming years here or this year and next year as well as we start to get sales from Europe [from Bayer] and on Beyonttra and AstraZeneca in Japan. Could you maybe just help us think through where your IP is on most out at is sort of the tariff implications and just sort of the royalty streams that you'll be getting from your ex US partners and just sort of how that ultimately affects your margin and your sort of profitability profile? Any color there or directional guidance would be helpful. Thanks.

恭喜您在發表會的第一季就取得了巨大成功。我想換個話題，也許只問一個政策問題，那就是思考未來幾年或今年和明年 Attruby 的成長和規模，以及我們開始從歐洲（拜耳）以及日本的 Beyonttra 和阿斯特捷利康獲得銷售。您能否幫助我們思考一下，您的智慧財產權在哪些方面影響最大，以及您從前美國合作夥伴那裡獲得的專利費收入是多少，以及這些最終會如何影響您的利潤率和獲利狀況？任何顏色或方向指導都會有幫助。謝謝。

Hey, Paul, thanks for the question. This is Tom. Yes so as Neil indicated in his prepared remarks, we're fortunate to have very little impact from the tariff discussions that are ongoing. We've done a pretty deep dive on this. As you know, the pharmaceutical has been exempt from the reciprocal tariffs but we also looked at the [AEP or IPA] and what's been said in public around the potential 232 tariffs affecting the industry.

嘿，保羅，謝謝你的提問。這是湯姆。是的，正如尼爾在其準備好的發言中所指出的，我們很幸運，正在進行的關稅討論對我們影響很小。我們對此進行了相當深入的研究。如您所知，製藥業已免於互惠關稅，但我們也研究了[AEP 或 IPA] 以及有關可能影響該行業的 232 項關稅的公開言論。

When we put all that together, based on our supply chain, we see a very, very minor impact. That's because Attruby made here in the USA. And then we're, of course, an American company, and we have all our IP domicile here in the US. So overall, very minor impact to the business.

當我們將所有這些因素綜合起來，根據我們的供應鏈，我們發現影響非常非常小。這是因為 Attruby 是在美國製造的。當然，我們是一家美國公司，我們所有的智慧財產權都在美國。因此整體而言，對業務的影響很小。

I'm not sure I understood the question on the royalties and how that relates, but that's off the top line revenue number. So obviously, we're getting money coming to us as before. Any implication on tariffs that would be on their end, but maybe I didn't understand the question.

我不確定我是否理解了有關版稅的問題以及它與收入之間的關係，但這是超出頂線收入數字的範圍。顯然，我們還是像以前一樣賺錢。這對他們的關稅有何影響？也許我沒有理解這個問題。

No, that makes sense on the ex US business.

不，這對美國以外的業務來說很有意義。

Anupam Rama, JPMorgan.

摩根大通的 Anupam Rama。

Congrats on the quarter with Attruby. I wanted to just pop in with a very quick pipeline question, so you shared some data today on the chronic hypoparathyroidism indication. So what gets you excited about this opportunity, particularly you talked about, Neil, from an NPV perspective. How does that all fit in?

恭喜 Attruby 在本季度取得的成績。我想快速問一個問題，您今天分享了一些關於慢性副甲狀腺功能減退症指徵的數據。那麼，尼爾，從 NPV 的角度來看，是什麼讓您對這個機會感到興奮？這一切是如何連結起來的？

Thanks for the question. We're going to drive it to Neil to talk about the NPV, and then Ananth can talk more about the data and our exciting plans for the Phase 3 in HP.

謝謝你的提問。我們將把它交給 Neil 來討論 NPV，然後 Ananth 可以更多地談論數據以及我們在 HP 第三階段的激動人心的計劃。

Yeah. I mean, from an NPV standpoint, I think you know that this isn't an exciting extension population, quite a bit larger than ADH1 itself with actually a pretty reasonable price point as well given where [Ascend] has ended up pricing their medicines. So there's a variety of different ways for us to take advantage of the opportunity now that many of the oral competitors have fallen by the wayside.

是的。我的意思是，從 NPV 的角度來看，我想你知道這不是一個令人興奮的擴展群體，比 ADH1 本身要大得多，而且考慮到 [Ascend] 最終為其藥品定價，價格實際上也相當合理。因此，現在許多口服競爭對手已經紛紛落敗，我們可以透過各種不同的方式來把握機會。

I'm going to actually pass it over to Ananth to talk a little bit about the exciting data that we just posted.

我實際上要把它交給 Ananth 來談談我們剛剛發布的令人興奮的數據。

Great question. Thanks for your interest in this program. We -- so as you mentioned, we reported data from the first nine participants with postsurgical hypoparathyroidism that were treated with Encaleret, normalized blood and urine calcium concomitantly in 78 of these participants and serves to be important oral option for these patients seeking to resolve calcium homeostasis. So we think that this could be a treatment changing or paradigm-changing treatment and we will advance development towards registration for this program.

好問題。感謝您對該計劃的關注。正如您所提到的，我們報告了前 9 名接受 Encaleret 治療的術後甲狀旁腺功能減退症患者的數據，其中 78 名患者的血液和尿液鈣同時恢復正常，這對尋求解決鈣穩態的患者來說是一種重要的口服選擇。因此，我們認為這可能是一種改變治療方法或改變範式的治療方法，我們將推進該計劃的註冊開發。

And in terms of the NPV or the market opportunity, like Neil mentioned, this is about a seven to eight times larger marketplace than the ADH1 market alone. So it could grow and expand the presence of Encaleret as we continue to investigate molecule.

就 NPV 或市場機會而言，正如 Neil 所提到的，這個市場比單獨的 ADH1 市場大約大七到八倍。因此，隨著我們繼續研究分子，它可能會生長並擴大 Encaleret 的存在。

Ellie Merle, UBS.

瑞銀的艾莉·梅爾（Ellie Merle）。

Congrats on the quarter. Curious what you're seeing so far in terms of commercial trans since Amvuttra's approval, you have a few good experience now with this so far. So I mean, I guess, specifically, was the rate of new unique patient prescriptions consistent with what you saw in February and March? Should you see an increase by April in the broader prescriber base? Or have you maybe seen a dip in the rates of the new [eugenic] patients starts as with the launch of a competitor?

恭喜本季取得佳績。我很好奇自從 Amvuttra 獲得批准以來，您在商業變性方面看到了什麼，到目前為止，您在這方面已經有了一些很好的經驗。所以我的意思是，具體來說，新的獨特患者處方率是否與您在二月和三月看到的情況一致？您是否會看到四月處方數量增加？或者您是否看到隨著競爭對手的推出，新 [優生] 患者的比例開始下降？

And any sort of high-level commentary on how you're thinking about positioning? Are there certain segments where you see potential greater uptake or advantages in terms of your use?

您對於定位有何看法？您是否認為某些領域在使用上有更大的潛力或優勢？

Sure. This is Matt. Thank you. So the second half of your question, the audio was a little garbled. So let me answer the first one, and then if you want to repeat the second one, happy to address that as well.

當然。這是馬特。謝謝。所以你問題的後半部分，音訊有點混亂。因此，讓我先回答第一個問題，然後如果你想重複第二個問題，我也很樂意解決這個問題。

Your first one regarding Amvuttra, there's definitely a place for lots of choices in this market. And I think as a company, we're obviously happy when patients have additional choices in terms of where Amvuttra is going to be placed, they seem to be earmarked for mixed phenotype at the moment, that's probably 10% of the overall market. Their variant data wasn't stat sig and they're very expensive. So it's twice as expensive as Attruby right now. And they have to compete, obviously, with our 340.25, which has been pretty successful.

您首先要問的是有關 Amvuttra 的問題，這個市場上肯定有很多選擇。我認為，作為一家公司，當患者在 Amvuttra 的治療方面有了更多選擇時，我們顯然會感到高興，目前 Amvuttra 似乎被指定用於混合表型，這可能佔整個市場的 10%。他們的變體數據不是統計訊號，而且非常昂貴。所以現在它的價格是 Attruby 的兩倍。顯然，他們必須與我們的 340.25 競爭，這已經相當成功了。

So I don't know whether that's going to land for them. I think they have to figure out their pricing. If you're going to charge double, you need to have better results and I think you can ask them to talk about that. I'm sure that they'll have opinions on that. Right now, it's early, but we're not seeing a lot of uptake outside of the mixed phenotype.

所以我不知道這對他們來說是否可行。我認為他們必須確定自己的定價。如果您要收取雙倍費用，您需要獲得更好的結果，我認為您可以要求他們討論這一點。我確信他們對此會有自己的看法。現在還為時過早，但我們還沒有看到混合表型以外的大量吸收。

Got it. And then just maybe this was a part of the question that broke off. I mean, if you can't hear me. But just in terms of the rate of unique patient prescriptions, I guess, have you seen any change in that since the approval of Amvuttra. So for instance, if you saw a dip in the rate of new unique patient prescriptions in April or as saying now that you have a broader prescriber base, you've seen a similar rate in new patient prescriptions or even higher? Thanks.

知道了。那麼也許這就是問題的一部分。我的意思是，如果你聽不到我說話。但就獨特患者處方率而言，我想，自從 Amvuttra 獲得批准以來，您是否看到任何變化？舉例來說，如果您發現 4 月新獨立患者處方率有所下降，或者說現在您擁有更廣泛的處方者基礎，您是否看到新患者處方率出現類似甚至更高的變化？謝謝。

Yeah. So the April data, I probably wouldn't be able to comment on -- I mean, they were sort of about two weeks in Q1 and now we picked up some data. We see some of the same stuff that you guys see. The trouble is even -- it's buy and bill. And so they're trying to convince a community physician to put out over $100,000 per injection, the vitamin A part is cheap.

是的。因此，對於四月份的數據，我可能無法發表評論——我的意思是，它們是在第一季的大約兩週後，現在我們獲得了一些數據。我們看到了一些和你們一樣的東西。問題甚至在於——這是購買和記帳。因此，他們試圖說服社區醫生為每次注射支付超過 10 萬美元，因為維生素 A 部分很便宜。

So from a cost perspective, that's fine, you still have to take that every day. But that out of pocket for the doctor, they have to cash flow that. So they have to put that money out and then they have to wait to get reimbursed.

因此從成本角度來看，這沒問題，你仍然需要每天服用。但這對醫生來說是自掏腰包，他們必須透過現金流來支付。所以他們必須先把錢拿出來，然後等待得到補償。

Well, if you start stacking up a lot of patients, that's a lot of money, and it gets pretty expensive. So we haven't seen it yet. It doesn't necessarily mean it is or isn't happening. I think you'd have to ask them. Maybe they can do what we did and throw out some early numbers on what the first month of launch looks like.

好吧，如果你開始堆積大量的病人，那就是一大筆錢，而且會變得非常昂貴。所以我們還沒有看到它。這並不一定意味著它正在發生或沒有發生。我認為你得問他們。也許他們可以像我們一樣，公佈一些發布第一個月的早期數據。

Jason Zemansky, Bank of America.

美國銀行的傑森·澤曼斯基。

Great. Congratulations on the quarter and thanks for taking our questions. I wanted to return to the question of segment growth for Attruby, if I may. It sounds like you expect the contribution from switches to slow at some point. But what does that look like over the near term? I mean, do you have a sense of how quickly you may be going through a bolus of, I don't know, refractory patients versus those, as you mentioned, are looking for a stronger stabilizer because they believe in the better outcomes over time?

偉大的。恭喜本季取得的成績，並感謝您回答我們的問題。如果可以的話，我想回到 Attruby 細分市場成長的問題。聽起來您預計開關的貢獻會在某個時候減慢。但短期內情況會如何呢？我的意思是，您是否知道您可能需要多快地完成一劑推注，我不知道，難治性患者與那些，正如您所提到的，正在尋找更強的穩定劑，因為他們相信隨著時間的推移會有更好的結果？

Hey, Jason. Thanks for the question. I think that high level, I can say a couple of things and then Matt going to add more details. But as we have said all along, we're not seeing any bolus or anything like that. I do think that the focus of our launch is treatment naive and where we're seeing consistent monthly share growth, and we expect that to continue.

嘿，傑森。謝謝你的提問。我認為從高層次來說，我可以說幾件事，然後馬特會補充更多細節。但正如我們一直所說的那樣，我們沒有看到任何團塊或類似的東西。我確實認為，我們推出的產品的重點是治療初治產品，並且我們看到每月的市場份額都在持續增長，我們預計這種情況將持續下去。

We had 100% share in the switch population and obviously, that's going to evolve now that there's a competitor. So that's sort of how we're thinking about it. Obviously, it's very early to say, and we look forward to seeing how the next quarter goes.

我們在 Switch 用戶群中佔有 100% 的份額，顯然，現在有了競爭對手，這種情況將會改變。這就是我們對此的看法。顯然，現在說這個還為時過早，我們期待看到下一季的進展。

Yeah. And I'll just add. I mean, I agree. We didn't see a bolus. I mentioned earlier, it's a little tricky to absolutely know if someone is a switch or a newly diagnosed. We don't spend a lot of time sort of thinking about it from that perspective. If patients want to get Attruby, we try to make sure they can get it for whoever they are newly diagnosed or switch.

是的。我再補充一點。我的意思是，我同意。我們沒有看到團塊。我之前提到過，要確切知道某人是轉換型患者還是新診斷的患者有點棘手。我們不會花很多時間從這個角度去思考這個問題。如果患者想要獲得 Attruby，我們會盡力確保他們能夠獲得該藥物，無論是新診斷的還是轉換的。

In terms of what that looks like over time, I think that's hard to predict. You can take the number of patients on tafamidis. You can add on a progression rate of whatever you think is correct. And then kind of model that out over time. I think that's probably the best approach if any.

至於隨著時間的推移情況會怎樣，我認為這很難預測。您可以統計服用他法米迪的患者人數。您可以添加您認為正確的進展率。然後隨著時間的推移，將其建模出來。我認為這可能是最好的方法。

There are no further questions at this time, and that concludes our Q&A for today. I will now hand the call back over to the company. Please go ahead.

目前沒有其他問題，今天的問答到此結束。我現在將把電話交還給公司。請繼續。

Thanks, everyone, for your questions today. We appreciate your interest in BridgeBio and look forward to updating you again next quarter.

謝謝大家今天的提問。感謝您對 BridgeBio 的關注，並期待下個季度再次向您更新最新情況。

This concludes our conference for today. We thank you for participating and ask that you please disconnect your lines.

今天的會議到此結束。感謝您的參與，並請您斷開線路。