BridgeBio Pharma Inc (BBIO) 2025 Q2 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. 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.

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。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。

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.

此處的所有聲明均基於 BridgeBio 截至今日所掌握的資訊。除法律要求外，本公司不承擔更新本次電話會議中所做的任何前瞻性陳述的義務。

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, everyone, and thank you for joining BridgeBio Pharma's second-quarter 2025 earnings call. I'm Chinmay Shukla, Senior Vice President of Strategic Finance and BridgeBio. With me today are Neil Kumar, our CEO; Matt Outten, our Chief Commercial Officer; and Tom Trimarchi, our President and Chief Financial Officer.

大家下午好，感謝您參加 BridgeBio Pharma 2025 年第二季財報電話會議。我是 Chinmay Shukla，策略財務和 BridgeBio 高級副總裁。今天與我一起的有我們的執行長 Neil Kumar、我們的商務長 Matt Outten 以及我們的總裁兼財務長 Tom Trimarchi。

During today's call, we will cover our strong and accelerating launch of Attruby. We will provide updates on our late-stage pipeline, including the three key Phase 3 trials in ADH1, LGMD2I, and achondroplasia. Following our prepared remarks, we will open the call for questions. For the Q&A session, we'll also be joined by Ananth Sridhar, Justin To, and Christine Siu, who lead our Stage 3 programs.

在今天的電話會議中，我們將介紹我們強勁且加速推出的 Attruby。我們將提供有關後期研發管線的最新進展，包括 ADH1、LGMD2I 和軟骨發育不全的三項關鍵 3 期試驗。在我們準備好發言之後，我們將開始提問。在問答環節，我們也將邀請負責第三階段計畫的 Ananth Sridhar、Justin To 和 Christine Siu 參加。

Before we begin, I would like to remind you that this call will include forward-looking statements based on current expectations. These statements represent our judgment as of today and may involve risks and uncertainties that could cause actual results to differ materially.

在我們開始之前，我想提醒您，本次電話會議將包括基於當前預期的前瞻性陳述。這些聲明代表了我們今天的判斷，可能涉及風險和不確定性，從而導致實際結果大不相同。

With that, I'll turn it over to Neil.

說完這些，我就把麥克風交給尼爾。

Thanks to everyone on the line for the time. Welcome to our Q2 earnings call. As always, this is a forum whereby we can communicate to you both on salient results and business strategy. A key aspect of that is ensuring we are communicating the data you feel is important and that we're doing it in a way that's clear.

感謝線上的每一個人。歡迎參加我們的第二季財報電話會議。像往常一樣，這是一個我們可以與您交流顯著成果和商業策略的論壇。其中一個關鍵方面是確保我們傳達您認為重要的數據，並以清晰的方式傳達。

On our last call, we spent a lot of time talking about how we make decisions internally and focused on an NPV-led characterization of our business. Your feedback, which is important, suggested that we spend less time on that and more time on the commercial, medical, and scientific performance of the business. We're excited to do that today because there's much to talk about as the business continues to deliver with the performance of Attruby and as it positions itself for three Phase 3 readouts in the coming months across ADH1, LGMD2I, and achondroplasia.

在我們上次電話會議中，我們花了很多時間討論我們如何在內部做出決策，並專注於以 NPV 為主導的業務特徵。您的回饋非常重要，它建議我們減少這方面花費的時間，而將更多的時間投入到業務的商業、醫療和科學表現上。我們很高興今天能這樣做，因為隨著公司繼續透過 Attruby 的表現取得進展，以及公司在未來幾個月內針對 ADH1、LGMD2I 和軟骨發育不全進行三期 3 期讀數，有很多事情要談。

The continued star of the show was Attruby. The first and most important way we've monitored this launch to date is by the number of unique patient prescriptions, which now sits at an absolute number of 3,751 coupled with 1,074 unique prescribers. We're seeing growth in both the number of new prescribers as well as the depth of prescribing at their practices. For those following week-by-week status, we've seen over 30% growth in weekly scripts. That acceleration is even greater than our internal projections given that this was the first full quarter with three players in the ATTR cardiomyopathy market.

節目的繼續明星是阿特魯比 (Attruby)。到目前為止，我們監控此次發布的第一個也是最重要的方式是透過唯一患者處方的數量，現在絕對數量為 3,751，加上 1,074 個唯一處方者。我們看到新開處方者的數量和開處方的深度都在增加。對於那些關注每週情況的人來說，我們看到每週腳本的成長超過 30%。考慮到這是 ATTR 心肌病變市場中擁有三家參與者的第一個完整季度，加速甚至比我們的內部預測還要大。

This prescribing ties to about 100% revenue growth that we've seen in Q2, around $78 million in global sales and $71.5 million in US net sales. And of course, all of these numbers connect to the most important set of facts here.

這項處方與我們在第二季度看到的約 100% 的收入成長有關，全球銷售額約為 7,800 萬美元，美國淨銷售額約為 7,150 萬美元。當然，所有這些數字都與這裡最重要的一組事實相關。

First, Attruby is now positively impacting thousands of patient lives. Second, given the fact that all three bands in this space are growing, we are collectively doing a better job of identifying patients. And third, we offer what we feel is a best-in-class clinical and data package in the ATTR cardiomyopathy space.

首先，Attruby 現在正在對成千上萬的患者的生活產生積極影響。其次，鑑於該領域所有三個樂團都在成長，我們在識別患者方面做得更好。第三，我們提供我們認為是 ATTR 心肌病變領域一流的臨床和數據包。

As discussed before, we have the responsibility to distribute Attruby as widely as possible so it can be used by any patient that needs it. And to that end, we continue to have the most generous access programs in this space and are proud that the programs we've pioneered are now being rolled out across the industry to improve access for patients.

正如之前所討論的，我們有責任盡可能廣泛地分發 Attruby，以便任何需要它的患者都可以使用它。為此，我們繼續在該領域提供最慷慨的訪問計劃，並且很自豪我們率先推出的計劃現在正在整個行業推廣，以改善患者的訪問機會。

A second key aspect of our responsibility is to further investigate the existing data and perform novel clinical studies to better understand how Attruby can maximally help specific patient profiles.

我們的第二個關鍵職責是進一步調查現有數據並進行新的臨床研究，以便更好地了解 Attruby 如何最大限度地幫助特定的患者。

In any disease category, it is precisely this type of cell population analysis that allows physicians to deploy the right drug for the right patient. In the case of Attruby over the last quarter, we published three salient results, one of which deals with the scientific underpinnings of the disease and two of which touch on important cell populations.

在任何疾病類別中，正是這種類型的細胞群體分析使醫生能夠為正確的患者部署正確的藥物。就上個季度的 Attruby 案例而言，我們發表了三項突出的成果，其中一項涉及該疾病的科學基礎，另外兩項涉及重要的細胞群。

The first of these publications further strengthens the connection between ever better stabilization and ever better clinical outcomes. Recall that prior to the Attruby-CM study, this connection had already been observed in three ways. First, by looking at the discretion outcomes between [80 mg taf], which is a 50% stabilizer and 20 mg tafamidis, which is a 35% stabilizer. Second, by looking at the genotype and phenotype of the disease and the associated rescue mutations. And third, through a small study that have been conducted by academic and BU suggesting that ever higher tetrameric stabilization as measured by serum TTR levels correlate to better clinical outcomes.

這些出版物中的第一篇進一步加強了更好的穩定性和更好的臨床結果之間的關聯。回想一下，在 Attruby-CM 研究之前，人們已經透過三種方式觀察到了這種關聯。首先，透過觀察 [80 mg taf]（50% 穩定劑）和 20 mg tafamidis（35% 穩定劑）之間的判斷結果。其次，透過觀察疾病的基因型和表現型以及相關的挽救突變。第三，透過學術界和波士頓大學進行的一項小型研究表明，以血清 TTR 水平衡量的四聚體穩定性越高，臨床結果就越好。

A broad analysis of the attribute dataset for the first time isolates the connection between ever higher levels of stabilization as measured by serum TTR levels, which in turn are easily measurable in the clinical context and downstream clinical outcomes in both the wild side and variant context.

首次對屬性資料集進行廣泛分析，分離出以血清 TTR 水平衡量的更高穩定水平之間的聯繫，而血清 TTR 水平又可以在臨床環境中輕鬆測量，並且可以在野生和變異環境中測量下游臨床結果。

Importantly, as more at all show in a published paper recently, every 1 mg per deciliter increase in serum TTR leads to a 5% decrease in risk of mortality. Recent work published by authors in Europe, [Menervini], et al, also observes this correlation at a quantitative level, and they extend from it the recommendation that serum TTR be used to stage patients with cardiomyopathy. All of this is especially important given that patients who switch from tafamidis to acoramidis in the context of the attribute OLE all experienced significant increases in serum TTR, with an average rise of 3.4 mg per deciliter.

重要的是，正如最近發表的一篇論文所顯示的那樣，血清 TTR 每增加 1 毫克/分升，死亡風險就會降低 5%。歐洲作者 [Menervini] 等人最近發表的研究也在定量層面上觀察到了這種相關性，並從中擴展出使用血清 TTR 對心肌病變患者進行分期的建議。所有這些都特別重要，因為在屬性 OLE 背景下從 tafamidis 轉換為 acoramidis 的患者血清 TTR 均顯著增加，平均每分升增加 3.4 毫克。

Moving to key subpopulation analyses, the first cell population we wanted to look at was the variant subtype. As KOLs at the NAC have published, patients with the most common cardiomyopathic variant V122I, have a 50% probability of survival as compared to even wild-type ATTR cardiomyopathy patients.

轉向關鍵亞群分析，我們想要研究的第一個細胞群是變異亞型。正如 NAC 的 KOL 所發表的那樣，與野生型 ATTR 心肌病變患者相比，患有最常見的心肌病變變異 V122I 的患者有 50% 的存活機率。

As some of you on the call may remember, acoramidis has a superior binding profile compared to other stabilizers, not only in wild-type patients, but also in the variant population as suggested by two prior publications and reinforced by an upcoming paper that has been submitted for publication that details both biochemical and clinical outcome advantages of Attruby as compared to other stabilizers in the variant population.

電話會議中的一些人可能還記得，acoramidis 與其他穩定劑相比具有更優異的結合特性，不僅在野生型患者中，而且在變異人群中也是如此，正如之前的兩篇出版物所表明的那樣，並且即將提交發表的一篇論文也強化了這一點，該論文詳細介紹了 Attruby 與變異人群中的其他穩定劑相比在生化和臨床結果方面的優勢。

Once again, we've been able to establish the advantage of that greater stabilization in this subpopulation and we're able to publish a 59% hazard reduction in time to first event CVH or ACM and that is associated with a p-value of 0.011. To our knowledge, this is the greatest degree of risk reduction that's been observed in the variant population with the highest degree of statistical significance in the field.

我們再次證明了該亞群中更高穩定性的優勢，並公佈了首次事件CVH或ACM的風險降低59%的結果，其p值為0.011。據我們所知，這是該領域在變異族群中觀察到的最大風險降低程度，具有最高的統計顯著性。

Finally, we published on another important subpopulation, namely patients with cardiac arrhythmic involvement. In terms out that this population is luckily more common than certainly, I would have thought at the outside of our studies in ATTR cardiomyopathy.

最後，我們發表了關於另一個重要亞群，即患有心律不整的患者的文章。如果這個群體幸運地比肯定的更常見，我會認為這在我們對 ATTR 心肌病變的研究之外。

Indeed, more than 50% of the population within Attruby had AFib, allowing us to ask the following questions. Does treatment with Attruby reduce the consequences of AFib and might even stave off the occurrence of AFib? It turned out importantly that it is able to do both. We observed a 43% reduction in risk of CVH associated with cardiac arrhythmia and a 17% reduction in the onset of AFib. Again, we believe this is the best data in the AFib subpopulation published, where other stabilizers appear to have had some effect and were knockdowns to our knowledge based on published AE tables appear not to have had benefit on AFib occurrence. All of this research is complemented by our ACT-EARLY trial.

事實上，阿特魯比地區超過 50% 的人口患有 AFib，這讓我們不得不提出以下問題。使用 Attruby 治療是否可以減輕 AFib 的後果，甚至可以延緩 AFib 的發生？事實證明，它能夠同時做到這兩點。我們觀察到與心律不整相關的 CVH 風險降低了 43%，AFib 發生率降低了 17%。再次，我們相信這是已發布的 AFib 亞群中最好的數據，其中其他穩定劑似乎也產生了一些效果，並且據我們所知，基於已發布的 AE 表，擊倒似乎對 AFib 的發生沒有益處。所有這些研究都由我們的 ACT-EARLY 試驗進行補充。

In discussions with clinicians and healthcare policy leaders alike, I've been struck by the enthusiasm associated with this courageous trial that seeks to marry what's known about the path and mechanism of this mass action disease with a bold strategy that extends service to patients beyond the acute phase of disease to potential prevention.

在與臨床醫生和醫療政策領導人的討論中，我對這項勇敢的試驗所展現的熱情感到震驚，該試驗試圖將已知的這種群體性疾病的路徑和機制與一項大膽的戰略結合起來，將服務範圍擴大到疾病急性期之後的患者，以達到潛在的預防效果。

What ACT-EARLY early reinforces is that the earlier we find patients and the more quickly we can act on disease, the better off patients are. That's why we also believe that the rapid onset of stabilization and the associated escalation of serum TTR associated with Attruby and the three-month separation on CVH and ACM has been demonstrated is a critical aspect of our drug's differentiation. We'll continue to publish on this, Attruby rapid action, and we'll have more to say about it at this year's ESG conference.

ACT-EARLY 早期強調的是，我們越早發現患者，越快對疾病採取行動，患者的狀況就越好。這就是為什麼我們也相信，與 Attruby 相關的穩定化和血清 TTR 的快速升級以及 CVH 和 ACM 的三個月分離已被證明是我們藥物差異化的關鍵方面。我們將繼續發布有關 Attruby 快速行動的文章，並將在今年的 ESG 會議上對此進行更多討論。

The sum of this ever-evolving corpus of clinical research, coupled with our efforts in the field, should be increasing scientific share of voice, which in turn should drive treatment in naïve share growth.

不斷發展的臨床研究體系加上我們在該領域的努力，應該會增加科學的發言權，進而推動治療份額的成長。

With treatment in naïve populations being the most important battle, we believe, to win for patients. Matt will have more to say on the specifics of our launch in some moments.

我們相信，對未接受治療的人群進行治療是患者贏得勝利最重要的戰鬥。馬特稍後將會詳細介紹我們的發布會的具體細節。

As I mentioned in my opening comments, beyond all of the activity around Attruby, BridgeBio is now poised to become a diversified fully integrated biopharma company with the delivery of up to three novel best or first-in-class assets in high unmet need areas. Each of these high unmet needs represent the potential for our drugs to serve additional tens of thousands of patients and each individually represents $1 billion-plus opportunities.

正如我在開場白中提到的，除了圍繞 Attruby 的所有活動之外，BridgeBio 現在準備成為一家多元化、完全整合的生物製藥公司，在高度未滿足的需求領域提供多達三種新穎的最佳或一流的資產。這些尚未滿足的巨大需求都代表著我們的藥物有可能服務於另外數萬名患者，並且每種藥物都代表著超過 10 億美元的機會。

Turning to the first of these important readouts in autosomal dominant hypocalcemia type 1 or ADH1. As a reminder, this condition, which has no available pharmaceutical therapies to date, is one that arises uniformly from gain of function mutations in the calcium sensing receptor and leads to low serum calcium and high urine calcium levels which in turn drives all of the downstream morbidity associated with this condition. BridgeBio is developing in its Phase 3, a negative allosteric modulator of the calcium sensing receptor with the goal to show statistically significant normalization of urinary and serum calcium levels as compared to current standard of care.

轉向常染色體顯性低血鈣 1 型或 ADH1 中這些重要讀數中的第一個。需要提醒的是，這種疾病迄今尚無可用的藥物療法，它是由於鈣敏感受體的功能獲得性突變而引起的，會導致血清鈣水平降低和尿鈣水平升高，進而引發與這種疾病相關的所有下游發病率。BridgeBio 正在其第 3 階段開發一種鈣敏感受體的負變構調節劑，目標是與目前的治療標準相比，尿液和血清鈣水平在統計上顯著恢復正常。

Given the lack of available pharmaceutical therapy or really anything reasonable for these patients, our base case expectation for the trial is simply to deliver statistically significant normalization as compared to standard of care with a safe, easy-to-take oral drug.

鑑於缺乏可用的藥物治療或對這些患者實際上任何合理的治療，我們對試驗的基本期望僅僅是與使用安全、易於服用的口服藥物的標準治療相比，實現統計上顯著的正常化。

The upside expectation, which is certainly consistent with both the biology and what we've seen in the clinic to date, would be response rates at 50% or greater for the patients that we serve. That would be a truly disruptive result.

上行預期是，我們所服務的患者的緩解率將達到 50% 或更高，這當然與生物學和我們迄今為止在臨床上看到的情況一致。那將是一個真正具有破壞性的結果。

And who are these patients? How many are there? As with many conditions, a lack of pharmaceutical therapy means that this is a poorly characterized and underdiagnosed condition. Today, we believe that there are some 3,000 diagnosed patients in the US alone. But in recent paper, we have released and that's consistent with observations others have made in large genetic databases indicates that the genetic prevalence is up to 12,000 patients in the US alone. The good news here is that we know where to look to find these patients.

這些病人是誰？有多少個？與許多疾病一樣，缺乏藥物治療意味著這是一種特徵不明顯且診斷不足的疾病。今天，我們認為光在美國就有大約 3,000 名確診患者。但在最近我們發表的論文中，這與其他人在大型基因資料庫中所做的觀察一致，顯示僅在美國，遺傳盛行率就高達 12,000 名患者。好消息是我們知道去哪裡尋找這些病人。

As we've discussed before, sequencing efforts in the nonsurgical hypoparathyroidism space have consistently identified missing ADH1 patients to the tune of 20% to 25%. Further, our experience in TTR where the overall marketplace was also about one-fifth diagnosed, suggests tactics around education and awareness that we believe are applicable to this launch.

正如我們先前所討論的，非手術甲狀旁腺功能減退症領域的定序工作已持續發現 20% 至 25% 的 ADH1 患者缺失。此外，根據我們在 TTR 的經驗，整個市場中約有五分之一的患者被診斷出患有這種疾病，這表明我們認為圍繞教育和意識的策略適用於此次發布。

The results of this Phase 3 are anticipated this fall. Importantly, and by the way, this is the case for all of our three late-stage medicines. There is substantial promise and follow-on indications for encaleret. Specifically in this case, in hypoparathyroidism. We plan to present at ASBMR compelling data suggesting the promise of encaleret in chronic hypoparathyroidism.

預計第三階段的結果將於今年秋季公佈。重要的是，順便說一句，我們的三種後期藥物都是這種情況。Encaleret 具有巨大的前景和後續適應症。具體來說，在這種情況下，副甲狀腺功能減退症。我們計劃在 ASBMR 上展示令人信服的數據，表明 encaleret 治療慢性副甲狀腺功能減退症的前景。

In a cohort of 10 patients encaleret normalized urine and serum calcium levels in 80% of patients within five days of dosing. Importantly, this drug brings differentiated promise to the HP community across at least three potential dimensions. First, it's oral. Second, it potentially normalizes urine calcium, the cause of downstream kidney conditions. And third, it might avoid potential downstream bone-associated absorption issues that could require bisphosphonates.

在 10 名患者中，80% 的患者在服用 encaleret 五天內尿液和血清鈣水平恢復正常。重要的是，這種藥物至少在三個潛在維度上為 HP 社區帶來了差異化的前景。首先，它是口頭的。其次，它可以使尿鈣（導致下游腎臟疾病的原因）正常化。第三，它可以避免可能需要雙磷酸鹽治療的潛在下游骨相關吸收問題。

Turning now to linger muscular dystrophy type 2I. This is the second of our first-in-class products addressed to a deleterious condition to add no available pharmaceutical therapies. Here again, we focused at the intersection of being both safe and highly efficacious, employing again a small molecule approach to target this well-tested added source. This condition uniformly arises from loss of function mutations in an not called FKRP, salient HP opportunity for encaleret based on data published, and the time value of money.

現在來談談 2I 型肌肉營養不良症。這是我們的第二款針對有害狀況且無可用藥物療法的同類首創產品。在這裡，我們再次關注安全性和高效性的結合，再次採用小分子方法來針對這個經過充分測試的添加源。這種情況通常源自於未稱為 FKRP 的功能突變喪失、基於已發布數據的顯著 HP 機會以及貨幣的時間價值。

In summary, BridgeBio stands at the doorstep of transforming itself from a company that is predominantly defined by one asset to a company that is serving a multiplicity of important genetic disease markets and with the capabilities in place across this ecosystem to do even more.

總而言之，BridgeBio 即將從一家主要以單一資產為主導的公司轉型為服務多個重要遺傳疾病市場的公司，並具備在整個生態系統中做更多事情的能力。

Thanks, Neil. I'm pleased to report that Attruby has achieved exceptional performance in the second quarter of 2025, generating $71.5 million in net product revenue, representing 100% growth over quarter one, essentially a doubling of net product revenue. The launch of Attruby has accelerated with new patient adds now at around 120 patients per week versus 100 patients per week previously. The uptick has been driven by momentum in treatment-naïve patients. This strong launch trajectory is driven by more patients starting therapy along with an increasing number of prescribers choosing Attruby for their patients.

謝謝，尼爾。我很高興地報告，Attruby 在 2025 年第二季度取得了卓越的業績，創造了 7,150 萬美元的淨產品收入，比第一季度增長了 100%，基本上是淨產品收入的兩倍。Attruby 的推出速度加快，目前每週新增患者數量約為 120 名，而之前每週新增患者數量為 100 名。這一增長是由未接受過治療的患者的成長勢頭推動的。這一強勁的上市軌跡是由越來越多的患者開始接受治療以及越來越多的處方醫生為患者選擇 Attruby 所推動的。

We are operating in a large and fast-growing market. The ATTR-CM category is expanding rapidly and is expected to reach $15 billion to $20 billion at peak. This strong tailwind provides a significant runway for continued growth and reinforces our conviction in Attruby becoming a category-defining therapy.

我們在一個龐大且快速成長的市場中運作。ATTR-CM 類別正在迅速擴張，預計高峰將達到 150 億至 200 億美元。這種強勁的順風為持續成長提供了重要的跑道，並增強了我們對 Attruby 成為類別定義療法的信心。

In addition to the significant unmet need in ATTR-CM and the compelling value proposition of Attruby versus our competitors, our clinical data continues to demonstrate Attruby's remarkable efficacy. As Neil outlined, we are continuing to generate evidence reinforcing Attruby's position on the standard of care for ATTR-CM patients, especially in the treatment-naïve setting.

除了 ATTR-CM 中未滿足的重大需求以及 Attruby 相對於我們競爭對手的引人注目的價值主張之外，我們的臨床數據繼續證明了 Attruby 的顯著療效。正如尼爾所概述的，我們正在繼續提供證據來強化 Attruby 對 ATTR-CM 患者護理標準的立場，特別是在未接受過治療的情況下。

Let me touch on a few key highlights underlying our commercial performance in the second quarter. Attruby has a strong and expanding prescriber base. COEs and community HCPs continue to prescribe Attruby at steady rates with new prescribers initiating therapy each week. Those who have written in the past continue to do so, and new adopters continue to expand.

讓我談談我們第二季商業表現的幾個關鍵亮點。Attruby 擁有強大且不斷擴大的處方者基礎。COE 和社區 HCP 繼續以穩定的速度開出 Attruby 處方，每週都有新的處方者開始治療。過去寫作的人繼續寫作，新的採用者也不斷擴大。

Attruby has strong momentum with new starts, capturing share from patients initiating ATTR-CM therapy for the first time and show strong momentum against peak market share expectations of 30% to 40%. This is leading to increased demonstrated demand across segments.

Attruby 隨著新藥的上市而勢頭強勁，從首次接受 ATTR-CM 治療的患者中搶佔了市場份額，並顯示出強勁的發展勢頭，預計將達到 30% 至 40% 的峰值市場份額。這導致各個領域的需求增加。

Fill rates remain robust, well above industry averages, with Attruby's white glove patient support programs pulling through the increased prescriptions. Further, days of inventory on hand declined from Q1 to Q2, consistent with increasing familiarity and comfort among specialty pharmacies and distributors with our just-in-time supply model.

配藥率保持強勁，遠高於行業平均水平，而 Attruby 的白手套患者支援計劃則幫助解決了增加的處方問題。此外，庫存天數從第一季到第二季有所下降，這與專業藥局和經銷商對我們的即時供應模式的熟悉度和舒適度不斷提高相一致。

So why are new prescribers writing and why do current prescribers write more Attruby? The number one reason is differentiated efficacy. Attruby stands out as the only medication with near complete stabilization in the label. Having a near-complete stabilizer has been a welcome addition to the market versus a partial stabilizer and a partial knockdown.

那麼，為什麼新的開處方者會寫更多 Attruby 藥物，而目前的開處方者又會寫更多 Attruby 藥物呢？首要原因是功效差異化。Attruby 是唯一一款標籤上顯示接近完全穩定的藥物。與部分穩定器和部分拆卸相比，擁有近乎完整的穩定器對市場來說是一個受歡迎的補充。

Beyond choosing efficacy is a primary reason to start Attruby, HCPs are worried about affordability. Attruby continues to be the least expensive medication in the ATTR-CM category, with most patients paying $0 out of pocket reinforcing BridgeBio's commitment to accessibility. In fact, in Q2, almost 90% of all Attruby patients paid $0 for Attruby.

除了選擇功效是啟動 Attruby 的主要原因之外，HCP 還擔心可負擔性。Attruby 仍然是 ATTR-CM 類別中最便宜的藥物，大多數患者自付費用為 0 美元，這進一步證明了 BridgeBio 對藥物可近性的承諾。事實上，在第二季度，幾乎 90% 的 Attruby 患者為 Attruby 支付的費用為 0 美元。

Additionally, we have seen that patients on Attruby tend to stay on Attruby and refill prescriptions on time each month. This is likely due to two factors: first, our access and support programs. Our generous assistance programs continue to make Attruby accessible, seamless, and simple for patients and providers. Secondly, favorable IRA policies have significantly improved out of pocket costs for oral medications.

此外，我們發現，使用 Attruby 的患者傾向於繼續使用 Attruby 並每月按時補充處方。這可能歸因於兩個因素：首先，我們的訪問和支持計劃。我們慷慨的援助計劃將繼續為患者和醫療服務提供者提供便利、無縫且簡單的 Attruby 服務。其次，優惠的IRA政策顯著改善了口服藥物的自付費用。

ATTR-CM patients are on average on seven to eight other medications, with a typical out-of-pocket for oral drugs being between $0 to $2,000 max annually. This also means that patients add Attruby for no additional cost, as I had already mentioned.

ATTR-CM 患者平均需要服用七至八種其他藥物，口服藥物的自付費用每年通常在 0 至 2,000 美元之間。這也意味著患者可以免費添加 Attruby，正如我已經提到的。

To close, I want to note that the success of this launch reflects our ability to effectively translate strong science into real-world impact and commercial success. This performance not only reinforces confidence in Attruby's future but also gives us conviction in our ability to execute future rare disease launches with similar excellence across BridgeBio's portfolio.

最後，我想指出，此次發射的成功反映了我們能夠有效地將強大的科學轉化為現實世界的影響和商業成功。這項績效不僅增強了我們對 Attruby 未來的信心，也讓我們堅信我們有能力在 BridgeBio 的產品組合中以類似的卓越表現推出未來的罕見疾病產品。

As we've discussed, BridgeBio has three additional potential launches coming over 2026 and 2027. The launch of Attruby has allowed BridgeBio to build a strong commercial infrastructure. This includes top industry talent, but also the basis for the programs and launch plan that will be used to execute these launches. Each of these launches has peak sales potential of more than $1 million in the US market alone. We look forward to updating you on our commercial readiness in future calls.

正如我們所討論的，BridgeBio 將在 2026 年和 2027 年推出另外三款產品。Attruby的推出使BridgeBio建立了強大的商業基礎設施。這不僅包括行業頂尖人才，還包括用於執行這些發布的計劃和發布方案的基礎。光是在美國市場，每款產品的最高銷售潛力都超過 100 萬美元。我們期待在未來的電話會議中向您通報我們的商業準備。

Now, I'll turn it over to discuss our corporate strategy and give an update on our pipeline programs.

現在，我將討論我們的公司策略並介紹我們的管道計劃的最新情況。

Thank you, Matt, and good afternoon, everyone. I'll now discuss our financial results for the second quarter of 2025. Please note that our commentary on today's call will focus on GAAP financials, unless otherwise indicated.

謝謝你，馬特，大家下午好。我現在將討論我們 2025 年第二季的財務表現。請注意，除非另有說明，我們對今天電話會議的評論將重點放在 GAAP 財務狀況。

Total revenues were $110.6 million in Q2 2025, consisting of Attruby net product revenue, royalty revenue, and license and services revenue compared to $2.2 million for the same period last year. $108.4 million increase in total revenues was primarily due to a $71.5 million increase in net product revenue from our commercial product, driven by strong demand across all major prescribers and patient segments.

2025 年第二季總收入為 1.106 億美元，包括 Attruby 淨產品收入、特許權使用費收入以及授權和服務收入，而去年同期為 220 萬美元。總收入增加 1.084 億美元主要是由於我們的商業產品的淨產品收入增加了 7,150 萬美元，這受到所有主要處方者和患者群體的強勁需求的推動。

We also recorded $1.6 million in royalty revenue from ex US net sales of [Beyonttra] in Europe and Japan. License and services revenue increased by $35.3 million, largely due to the $30 million regulatory milestone recognized under the license agreement with Alexion pricing approval of Beyonttra by the National Health Insurance in Japan in May 2025.

我們也記錄了 [Beyonttra] 在歐洲和日本除美國外淨銷售額的 160 萬美元特許權使用費收入。許可和服務收入增加了 3530 萬美元，這主要歸功於與 Alexion 簽訂的許可協議中確認的 3000 萬美元監管里程碑，該協議於 2025 年 5 月獲得了日本國民健康保險對 Beyonttra 的定價批准。

Total operating costs and expenses for the second quarter of 2025 were $244.8 million compared to $177.7 million in the same period in the prior year. The $67.1 million increase in operating costs and expenses is primarily driven by $69.6 million increase in SG&A expenses, partially offset by a $3.5 million decline in R&D expenses. This reflects our continued investment in the Attruby brand awareness and ongoing investments in our late-stage clinical programs.

2025 年第二季的總營運成本和費用為 2.448 億美元，而去年同期為 1.777 億美元。營運成本和費用增加 6,710 萬美元，主要是由於銷售、一般和行政費用增加 6,960 萬美元，但研發費用減少 350 萬美元，部分抵消了這一增加。這反映了我們對 Attruby 品牌知名度的持續投資以及對後期臨床項目的持續投資。

Included in our total operating costs and expenses was $37.7 million of stock-based compensation expense compared to $21.5 million in the second quarter of 2024. We expect operating expenses to remain stable through year-end with continued revenue growth driven by Attruby.

我們的總營運成本和費用包括 3,770 萬美元的股票薪酬費用，而 2024 年第二季為 2,150 萬美元。我們預計，隨著 Attruby 推動收入持續成長，營運費用將在年底前保持穩定。

Turning to our balance sheet. We ended the second quarter with a strong cash position of $756.9 million in cash, cash equivalents, and marketable securities. This includes proceeds from our strategic monetization of Beyonttra European royalties for $300 million, which has significantly strengthened our financial flexibility together with the proceeds from Attruby sales. Looking ahead, we expect our cash runway to extend through multiple value-creating milestones.

轉向我們的資產負債表。我們在第二季結束時擁有強勁的現金狀況，包括 7.569 億美元的現金、現金等價物和有價證券。其中包括我們將 Beyonttra 歐洲特許權使用費策略性貨幣化所獲得的 3 億美元收益，這與 Attruby 銷售所得一起大大增強了我們的財務靈活性。展望未來，我們預期我們的現金流將延伸至多個創造價值的里程碑。

In closing, our commercial launch Attruby is accelerating, and our pipeline has never been stronger. We look forward to the data-rich months ahead with top-line results from ADH1 and LGMD2I [RNi] in the fall of 2025, achondroplasia in early 2026, and to continue our mission to serve patients and create lasting value for our stakeholders.

最後，我們的 Attruby 商業化發布正在加速進行，我們的產品線從未如此強大。我們期待未來幾個月數據豐富，在 2025 年秋季獲得 ADH1 和 LGMD2I [RNi] 的頂級結果，在 2026 年初獲得軟骨發育不全的頂級結果，並繼續我們的使命，為患者服務，為我們的利益相關者創造持久價值。

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

說完這些，我就把電話轉回給 Chinmay。

Thank you, Neil, Matt, and Tom. Operator, please open the line for questions. Thank you.

謝謝你，尼爾、馬特和湯姆。接線員，請打開熱線以回答問題。謝謝。

(Operator Instructions) Salim Syed, Mizuho.

（操作員指示）Salim Syed，瑞穗。

Thanks for the questions, and congrats on the quarter. I guess one from us on the 120 patient adds. So obviously, per week, so obviously, that's faster than the 109 patient adds if we use the April and February numbers that you guys provided. I'm just curious if you can just break that down a little bit more, what you think is driving that patient add number? And specifically, if you can, how you would envision that number changing in the third and fourth quarter this year?

感謝您的提問，並祝賀本季取得佳績。我想我們中的一位是 120 名患者中的一位。因此，顯然，如果我們使用你們提供的 4 月和 2 月的數字，那麼每週新增 109 名患者的速度顯然要快。我只是好奇，您是否可以進一步分析一下，您認為是什麼導致了患者數量的增加？具體來說，如果可以的話，您認為今年第三季和第四季這個數字將如何變化？

And also, Neil, I don't know if you can comment on this, but I think it's -- or Matt, I think it's one of the more important metrics if you can provide, just what percentage of naïve coming into the marketplace do you think you got in the second quarter?

另外，尼爾，我不知道你是否可以對此發表評論，但我認為這是——或者馬特，我認為這是一個更重要的指標，如果你可以提供的話，你認為第二季度進入市場的天真產品比例是多少？

Hey, Salim. Thanks for the question. I'm going to pass it on to Matt to talk about the first piece and then Neil to comment about the second piece.

嘿，薩利姆。謝謝你的提問。我將把它交給馬特討論第一部分，然後讓尼爾來評論第二部分。

Sure. And thanks for the question. I guess to respond to the first half of your question, we're seeing strength in treatment-naïve starts and continued switch activity. The market itself is expanding, driven by increased screening and awareness. We're seeing that quarter over quarter. Unique patient starts and prescriber counts are both increasing. And this has resulted in BridgeBio becoming the partner of choice for healthcare professionals.

當然。感謝您的提問。我想回答你問題的前半部分，我們看到了初治患者開始接受治療並持續轉換活動的優點。在篩檢和認知度提高的推動下，市場本身也在不斷擴大。我們每季都看到這種情況。獨立患者數量和處方數量都在增加。這使得 BridgeBio 成為醫療保健專業人士的首選合作夥伴。

In addition, the Attruby profile really resonates. Both patients and doctors are drawn to it. Benefits as soon as three months and a 50% reduction in hospitalization rates, and that's across subgroups and across patients switching from other therapies, as Neil mentioned in his opening comments. So I think it's a combination of excellent data, an ever-expanding market, and the best team in the industry.

此外，Attruby 的簡介確實引起了共鳴。患者和醫生都對此感興趣。正如 Neil 在開場白中提到的那樣，最快三個月就能看到效果，住院率降低 50%，而且這是針對各個亞組以及從其他療法轉換的患者而言的。所以我認為這是優秀的數據、不斷擴大的市場和業內最佳團隊的結合。

Neil, if you want to address the second part?

尼爾，你想談談第二部嗎？

Yeah. It's hard to tell Salim, exactly what the NBRx share is just because we don't precisely know where the knockdowns stand in terms of that. But best guess, it's somewhere in the 18% to 20% range, and it's been growing pretty healthily.

是的。很難告訴薩利姆 NBRx 份額到底是多少，因為我們並不確切知道在這方面擊倒情況如何。但最好的猜測是，它在 18% 到 20% 的範圍內，並且增長相當健康。

And just to add to Matt's point, I mean, I think just for being out in the field, a couple of things are starting to drive, I would say, our commercial momentum. But the first is better and better access.

為了補充馬特的觀點，我的意思是，我認為，只要身處這個領域，就有幾件事開始推動我們的商業動能。但首先是越來越好的存取方式。

The second and I think critically is increasing scientific share of voice. I think that serum TTR paper actually did a lot of work for us this quarter, and we can build on that. The fact that ever-increasing amounts of stabilization or in this case, with every additional mg per deciliter increase in serum TTR, you're getting a 5% decrease in mortality. And the fact that, that just got confirmed by a European group, I think two days ago, the Menervini paper came out as well. That's starting to become a really salient feature of both staging patients and deciding which therapy to put them on.

第二，我認為至關重要的是增加科學的發言權。我認為血清 TTR 紙張實際上在本季度為我們做了很多工作，我們可以在此基礎上繼續努力。事實上，隨著穩定量的不斷增加，或者在這種情況下，血清 TTR 每增加一毫克/分升，死亡率就會降低 5%。事實上，這一消息剛剛得到了一個歐洲組織的證實，我記得兩天前梅內爾維尼的論文也發表了。這開始成為對患者進行分期和決定採取何種療法的一個真正顯著的特徵。

So I think that, coupled with the variant data, coupled with the AFib data, they'll continue to drive momentum here. We just got to continue to educate.

因此我認為，加上變體數據和 AFib 數據，它們將繼續推動這一發展勢頭。我們只需要繼續教育。

Tyler Van Buren, TD Cowen.

泰勒範布倫 (Tyler Van Buren)，TD Cowen。

Hey, guys. Thanks, and congratulations on the progress. So last quarter, you all noted the lower-than-expected utilization of the 28-day free trial and the patient assistant programs, while gross to net modestly boosted net revenue per patient. So could you discuss those kind of three components and how the trends evolved in the second quarter compared to the first quarter and how you expect them to trend moving forward as we head into the second half of the year?

嘿，大家好。謝謝，並祝賀你取得進展。因此，上個季度，大家都注意到 28 天免費試用和患者助理計劃的利用率低於預期，而毛收入與淨收入之比略微提高了每位患者的淨收入。那麼，您能否討論這三個組成部分，以及第二季度與第一季相比的趨勢如何演變，以及您預計在進入下半年時它們將如何發展？

Hey, Tyler. Thanks for the question. We did indeed see normalization on all of those three fronts, the 28-day free trial, the utilization and the gross to net. But let me turn it over to Neil to give more commentary on what we saw and what we expect to see going forward.

嘿，泰勒。謝謝你的提問。我們確實看到這三個面向都趨於正常化，即 28 天免費試用、利用率和總淨收入。但是，請允許我把時間交給尼爾，讓他對我們看到的情況以及我們期望看到的情況發表更多評論。

Yeah, I don't have much to add. As Chinmay said, we did see normalization there. Maybe I'll talk a little bit about why this is so important to us. Obviously, with the variant data, the highest risk reduction shown in the V122I population and the broad variant population, and we'll have another publication out on that in the coming months, coupled with statistical significance, which is the first we've seen of that. It's really important for us to be able to serve the underpenetrated populations here with ATTR cardiomyopathy. And these programs are a really important feature of that.

是的，我沒什麼好補充的。正如 Chinmay 所說，我們確實看到了那裡的正常化。也許我可以稍微談談為什麼這對我們這麼重要。顯然，根據變異數據，V122I 族群和廣泛變異族群的風險降低程度最高，我們將在未來幾個月內就此發表另一篇出版物，並附上統計意義，這是我們第一次看到這種情況。對於我們來說，能夠為這裡患有 ATTR 心肌病變的未充分治療的人群提供服務確實非常重要。這些程序是其中非常重要的一個特徵。

So maybe less important than the COEs, which is probably where we had initial momentum and much more important as we drive out into the community and drive out into communities that have historically been underserved.

因此，它可能不如 COE 重要，COE 可能是我們最初的動力所在，而當我們走進社區，走進那些歷史上服務不足的社區時，COE 就顯得更為重要。

And I'm aware of kind of some of the narrative around (inaudible) man, you guys look at the Alnylam launch, it at double the cost. Like why do you price where you price? Why do you guys have a generous access program and suite, et cetera, et cetera.

我知道關於（聽不清楚）的一些說法，夥計們，你們看看 Alnylam 的發布，它的成本是原來的兩倍。例如，為什麼你的定價是那樣的？為什麼你們有慷慨的訪問程式和套件等等等等。

Look, I think long term, when you look at any category, I've honestly never seen a drug with better point estimates and a lower price, not ultimately do really well in terms of end market share. So we're in this for the long game. Honestly, these generous access programs, where we price the product, the continued education. And I think the price and these access programs will stand us in good stead long term. But yeah, I think you should expect to see the GPM stay normalized over the longer course of time and not go back to what we saw initially.

看，我認為從長遠來看，當你看任何類別時，我真的從未見過一種具有更好的點估計和更低價格的藥物，最終在終端市場份額方面表現不佳。所以我們要打一場持久戰。老實說，這些慷慨的訪問計劃，我們對產品、繼續教育進行定價。我認為價格和這些訪問計劃將對我們長期有利。但是的，我認為你應該會看到 GPM 在較長時間內保持正常化，而不是回到我們最初看到的狀態。

Biren Amin, Piper Sandler.

比倫阿明、派珀桑德勒。

So it seems I guess per day in the third quarter accelerated compared to the prior period while you guys face a new competitor. Can you just maybe talk about community versus academic market share for Attruby?

因此，我猜想第三季每天的成長速度與前一時期相比有所加快，而你們面臨著新的競爭對手。您能否談談 Attruby 的社區市佔率與學術市場佔有率？

And then maybe a question on the pipeline. What are your thoughts on infigratinib's potential market share in achon? And how are you positioning the hypochondroplasia program given the recent preclinical data?

然後也許還有一個關於管道的問題。您對 infigratinib 在 achon 的潛在市佔率有何看法？根據最近的臨床前數據，您如何定位軟骨發育不良計畫？

Hey, Biren. Thank you for your question. I'm going to pass it on to Matt to talk about what's driving the acceleration in the launch and specifically in the treatment-naïve segment, both in the COEs and in the community setting. And then I'll pass on to Justin to talk about [BNP] program.

嘿，比倫。感謝您的提問。我將把它傳遞給馬特，讓他談談推動啟動加速的因素，特別是在 COE 和社區環境中的未接受治療的領域。然後我會讓賈斯汀談論 [BNP] 計劃。

I mean, I think it's the overall data package combined with the unmet need, patients either are not being treated effectively and are looking for something else. And so that's one patient profile.

我的意思是，我認為這是整體數據包與未滿足的需求相結合的結果，患者要么沒有得到有效治療，要么正在尋找其他治療。這就是一位患者的概況。

There's also patients who are newly diagnosed and are seeking something that can work very fast and hit all the endpoints that they're trying to hit. So I think regardless of the type of patients coming in, and then that can be even then split into more subgroups.

還有一些剛確診的患者，他們正在尋求一種能夠快速起效並達到他們想要達到的所有終點的治療方法。所以我認為無論入院患者是什麼類型，都可以將其分為更多的亞群。

We have variant populations, you have patients with AFib. Everybody is looking for something that can work quickly and for a long time. And I think that, that is what has gotten us off to such a fast start.

我們有不同的人群，有患有 AFib 的患者。每個人都在尋找能夠快速且長時間發揮作用的東西。我認為這就是我們如此快速起步的原因。

And the question is, how do you keep that momentum going? Well, the market itself keeps growing every quarter. And that's because more and more people now are looking for the disease and finding it. So more patients sort of show up even without us doing anything in that regard. But then when they do show up and they find the information that we have, whether it's online or from their physician themselves, I think then they're impressed and want to try Attruby.

問題是，你如何保持這種勢頭？嗯，市場本身每季都在成長。這是因為現在越來越多人正在尋找並發現了這種疾病的人。因此，即使我們沒有採取任何措施，也會有更多的患者出現。但當他們出現並發現我們擁有的資訊時，無論是在線還是從他們的醫生那裡獲得的信息，我認為他們會印象深刻並想要嘗試 Attruby。

And maybe just to build on that and addressing this specifically to your question, it's still a majority in the COE or COE capitated practices. Recall that 65% of cardiovascular practices are capitated or JVs in some way with a major provider in their space. But we are seeing a pickup in the community. And I think that has to do with a lot of the awareness stuff that we and others are doing. So it's still a majority in COE or COE capitated practices, but I expect it will continue to disperse over time.

也許只是為了在此基礎上具體回答您的問題，這在 COE 或 COE 按人頭計算的實踐中仍然佔大多數。回想一下，65% 的心血管診所都是按人頭收費的，或者以某種方式與其所在領域的主要供應商合資經營。但我們看到社區正在好轉。我認為這與我們和其他人正在做的許多提高認識的工作有關。因此，在 COE 或 COE 按人頭計算的實踐中，它仍然佔多數，但我預計它會隨著時間的推移而繼續分散。

I don't know, Justin, on [naïve]?

我不知道，賈斯汀[天真]？

Yeah. And just as a reminder, when we started this program, we have two key criteria, right? The first was to have a daily oral treatment option for families who are tired of injections. And the second is to have deeper levels of efficacy by not only hitting the MAPK pathway, but also STAT1. And our best-in-class HP data and proportionality data from our Phase 2 validate our hypothesis and most importantly, with the convenience factor of a daily oral.

是的。提醒一下，當我們啟動這個專案時，我們有兩個關鍵標準，對嗎？首先是為那些厭倦注射的家庭提供每日口服治療的選擇。第二個目的是不僅作用於 MAPK 通路，也作用於 STAT1，以獲得更深層的療效。我們第二階段的最佳 HP 數據和比例數據驗證了我們的假設，最重要的是，它具有每日口服的便利性。

Now, we've consistently done market research that shows an oral with similar efficacy at CMPs would take about 60% of the market in a market. Why? Because clinicians and families all view an oral as better than either a daily or weekly injectable. And this market research has been done by others across indications as well [factored data].

現在，我們一直在進行市場調查，結果表明，具有類似功效的口服藥物在中藥市場的佔有率約為 60%。為什麼？因為臨床醫師和家庭都認為口服藥物比每日或每週注射藥物好。其他人也針對不同適應症做過類似的市場調查[分解資料]。

Now, we know BioMarin showed some data yesterday on its long-acting CMP, which doesn't change our expectations here. We actually know from their existing Phase 2 data that efficacy on HP and CGMP [at the exam] plateau above their go-ahead commercial dose. So a program that achieves higher levels of CMP here don't really matter. And if anything, it makes it even more important to have a therapy that impacts STAT1 since it looks like effects on that here.

現在，我們知道 BioMarin 昨天公佈了其長效 CMP 的一些數據，但這並沒有改變我們的預期。實際上，從他們現有的第 2 階段數據中我們可以得知，該藥物對 HP 和 CGMP 的療效（在檢查中）已穩定在高於其商業化劑量的水平。因此，在這裡實現更高水平 CMP 的程序並不重要。而且如果有的話，那麼找到一種影響 STAT1 的療法就變得更加重要，因為它看起來對 STAT1 有影響。

Now, on hypochondroplasia, we're really excited about our recent data that we just published in the Journal of Mineral and Bone Research that shows infigratinib has low-single-digit in vitro potency against the most common hypochondroplasia mutations and similar efficacy across hypochondroplasia mouse models as it did compared to achondroplasia mouse models. So given that, we expect similar best-in-class efficacy profile in our hypochondroplasia program as well. So more to come there.

現在，關於軟骨發育不良，我們對剛剛在《礦物和骨研究雜誌》上發表的最新數據感到非常興奮，這些數據表明，英菲格拉替尼對最常見的軟骨發育不良突變具有低個位數的體外效力，並且在軟骨發育不良小鼠模型中的療效與軟骨發育不全小鼠模型中的療效相似。因此，有鑑於此，我們期望我們的軟骨發育不良計畫也能獲得類似的最佳療效。未來還會有更多。

Cory Kasimov, Evercore.

科里·卡西莫夫（Cory Kasimov），Evercore。

Hey. Good afternoon, guys. Question for you on encaleret. I'm curious kind of what your market research is suggesting would be considered a meaningful win in the upcoming Phase 3 CALIBRATE trial.

嘿。大家下午好。關於 encaleret 的問題。我很好奇，您的市場研究表明，在即將進行的第三階段 CALIBRATE 試驗中，什麼會被視為有意義的勝利。

Hey, Cory. Thanks for the question. I'm going to pass on to Ananth, who leads the program to talk about it.

嘿，科里。謝謝你的提問。我將把話題轉交給負責專案的 Ananth 來談論此事。

Yeah. Thanks, Cory. For this program in ADH1 in particular, we see any successful study as a win, really a home run for this community. And as we've discussed in the past and as the investor community is familiar that the available conventional therapy or standard of care to our knowledge, offers pretty meager benefit on the composite endpoint, which we are evaluating in our Phase 3, which is concomitant normalization of both blood and urine calcium, which are both important biomarkers in terms of biochemistry is for the condition.

是的。謝謝，科里。特別是對於 ADH1 中的這個項目，我們認為任何成功的研究都是勝利，對這個社區來說都是全壘打。正如我們過去所討論過的，投資者群體也熟悉這一點，據我們所知，現有的常規療法或標準治療對綜合終點的益處相當小，我們正在第三階段對其進行評估，即血液和尿液鈣同時正常化，這兩者都是該病症在生物化學方面的重要生物標誌物。

So what we see as a step change for this community is really we can see a majority of patients achieving those criteria on encaleret, we see as both clinically meaningful and statistically significant, likely statistically significant benefit achieved for the study for the patient population.

因此，我們認為這個群體的一個重大變化是，我們確實可以看到大多數患者在 Encaleret 上達到了這些標準，我們認為這在臨床上和統計學上都具有意義，可能為患者群體的研究帶來了統計學上顯著的益處。

And as a reminder, Cory, if we looked at our Phase 2 cohort on the same criteria at the same time point, we saw 9 out of 13, around 69% of our study participants able to achieve that on encaleret in that same group, none or 0% were able to achieve that criteria on standard of care.

提醒一下，科里，如果我們在同一時間點以相同的標準觀察我們的第 2 階段隊列，我們會發現 13 人中有 9 人，大約 69% 的研究參與者能夠在標準護理中通過 Encaleret 達到這一目標，而沒有一個人或 0% 的人能夠在標準護理中達到這一標準。

Mani Faroohar, Leerink Partners.

Mani Faroohar，Leerink Partners。

Thanks for taking the questions. Congrats on the quarter. I've got a couple, and I'm going to follow here in lead by starting on the pipeline and going to commercial. For LGMP2I, can you lay out based perhaps on your KOL conversations and your interactions with the regulators what the bar is in terms of key efficacy and biomarker thresholds for potential approval based on the upcoming interim data? And then I have a commercial question to follow up.

感謝您回答這些問題。恭喜本季取得佳績。我有幾個，我將跟隨他們，從管道開始，然後進行商業化。對於 LGMP2I，您能否根據您的 KOL 對話以及您與監管機構的互動，根據即將發布的中期數據，闡述在關鍵功效和生物標誌物閾值方面可能獲得批准的標準？然後我有一個商業問題需要跟進。

Sure. I'll pass on to Christine to talk about LGMP2I.

當然。我會讓克里斯汀談論 LGMP2I。

Hi, Mani. So for the top-line data that we're expecting later this fall, we're going to be looking for a few different things in the data that would kind of represent a win for us, both in terms of being clinically meaningful as well as supporting a regulatory approval on an accelerated basis.

你好，瑪尼。因此，對於我們預計在今年秋季晚些時候發布的頂線數據，我們將在數據中尋找一些對我們來說代表勝利的不同的東西，無論是在臨床意義上還是在支持加速監管批准方面。

So first, we're going to look for really a robust effect on the biomarkers. The primary endpoint is glycosylated alpha-dystroglycan. And what we're hoping to see there would be consistent with our Phase 2 results where we saw an elevation in glycosylated oxidase (inaudible). And we think anything kind of 5% or more there would be clinically meaningful. In addition, we're going to look to see a robust reduction in CK of about 40% or more.

因此，首先，我們要尋找對生物標誌物真正強大的影響。主要終點是糖基化α-肌肉營養不良蛋白聚醣。我們希望看到的結果與我們在第二階段的結果一致，即糖基化氧化酶的升高（聽不清楚）。我們認為 5% 或更高的數值具有臨床意義。此外，我們還將看到 CK 大幅減少約 40% 或更多。

On the functional endpoints, what would be considered a win there is a trend in one or more of those outcome measurements. It's important to note that we do not expect statistical significance at the 12-month time point. The trial was powered to show it. And the FDA has indicated that it is not a requirement for accelerated approval to see statistical significance in any of the clinical outcomes. So again, just looking for trends in one or more of the functional outcomes.

在功能終點上，什麼會被視為勝利，在一個或多個結果測量中存在趨勢。值得注意的是，我們並不期望在 12 個月的時間點出現統計意義。這次審判有力地證明了這一點。FDA 也表示，不需要加速審批即可看到任何臨床結果的統計意義。所以，再說一遍，只是尋找一個或多個功能結果的趨勢。

And then the third thing we'd like to see is a well-tolerated safety profile consistent with our Phase 2 results. And I think if we saw all of those, we'd be quite encouraged. Keep in mind, there's really no available treatments today for this indication. So we're pretty excited about the FDA to have a first-to-market safe and oral therapy for this [patient].

我們希望看到的第三件事是與我們的第二階段結果一致的、耐受性良好的安全性概況。我認為如果我們看到所有這些，我們會非常受鼓舞。請記住，目前確實沒有針對這種症狀的治療方法。因此，我們非常高興 FDA 能夠推出首個安全口服療法來治療這種疾病[病人]。

Great. And hopping over to commercial. I guess a little bit of a composite question. Alnylam talked a lot about -- on their call about adding patient volume for Amvuttra in both switch and new market and new patient -- new to therapy patients. Pfizer talked on pricing, how net price had evolved, and they have been doing more contracting to maintain share. So for each of these competitors, what are you seeing in terms of impact on your own contracting and pricing?

偉大的。然後跳到商業廣告。我想這是一個有點複雜的問題。Alnylam 在電話會議上談了很多關於增加 Amvuttra 在轉換和新市場以及新患者（新治療患者）中的患者數量的問題。輝瑞談到了定價、淨價格如何演變以及他們一直在進行更多的合約以維持份額。那麼對於每個競爭對手，您認為它們對您自己的合約和定價有何影響？

And also in terms of volume, are you seeing more impact and more competitors and sort of more active competition on the switch side? Is it primarily competitive intensity for new-to-therapy patients? Like how should we interpret both of those commentaries from those separate calls? And how they inform how we think about the competitive dynamic in what is now multiplayer market?

另外，就數量而言，您是否看到 Switch 方面的影響越來越大、競爭對手越來越多、競爭越來越激烈？對於新接受治療的患者來說，競爭強度主要是什麼？例如，我們該如何解讀這兩次單獨通話中的評論？它們如何告訴我們如何看待現在的多人遊戲市場中的競爭動態？

Hey, Mani. Thanks for the question. As we noted in our PR, most of our growth came from the treatment-naive section and increasing share there where we've seen it grow month-over-month.

嘿，瑪尼。謝謝你的提問。正如我們在公關中指出的那樣，我們的大部分增長來自於未接受治療的部分，並且我們看到該部分的份額逐月增長。

But let me pass it on to Neil to talk in more detail about these things as he's been out of the field.

但由於他已經離開現場，請允許我將其轉交給尼爾，讓他更詳細地談論這些事情。

Yeah. Thanks, Mani. I mean I guess I'd say, first and foremost, where we're seeing more pressure from the part of the knockdown is in the switch category, obviously, for us. We were 100% in the switch share prior to. So obviously, you're going to be under pressure there.

是的。謝謝，瑪尼。我的意思是，我想說，首先，我們看到擊倒部分的壓力更大，這顯然是屬於開關類別。我們之前的轉換份額是 100%。所以很明顯，你將會面臨壓力。

Recall, there's a couple of other modes by which they're getting patients on drug initially. One is combination. And actually, a, we're seeing a lot of combo -- when people do reach for the combo, we are seeing people try to use the best stabilizer coupled with the knockdown agent. So we've got some combo stuff there and as does obviously, the (inaudible) plus TAP combo.

回想一下，他們最初讓患者服用藥物還有其他幾種方式。一是組合。實際上，我們看到了很多組合——當人們確實使用組合時，我們看到人們嘗試使用最好的穩定劑與擊倒劑結合使用。所以我們在那裡有一些組合的東西，顯然還有（聽不清楚）加上 TAP 組合。

Then they had the bolus, right? They had their patients that rolled over. We didn't have that because you obviously gave away free drug for life. And then they've got what they've got in the naïve population.

然後他們就有了丸劑，對嗎？他們讓病人翻身。我們沒有這樣做，因為你顯然終身免費提供藥物。然後他們就得到了天真的民眾所得到的東西。

Honestly, we're not seeing a ton of competition there from Alnylam. We're seeing much more of it from Pfizer. So then turning to what Pfizer is doing. We're also not seeing like a ton of race to the bottom GPN contracting type activity in this space.

老實說，我們並沒有看到來自 Alnylam 的太多競爭。我們從輝瑞公司看到了更多這樣的情況。那麼接下來我們來看看輝瑞公司正在做的事情。我們也沒有看到該領域出現大量競相壓價的 GPN 承包類型的活動。

We've made it very clear that we stand on this price. We came in where we came in. We think it's the right thing and the ethical thing for the patient population, but there's not a whole lot of backdoor games that we're playing at all, and we're not seeing it from a competition either. So outside of the buy-and-bill dynamic associated with the knockdowns, we're not seeing a whole lot of competition in that vein. The competition is much more so around clinical differentiation and efficacy.

我們已經明確表示我們堅持這個價格。我們進入了我們所進入的地方。我們認為這對患者群體來說是正確的、合乎道德的，但我們並沒有玩太多幕後遊戲，而且我們也沒有在比賽中看到這種情況。因此，除了與擊倒相關的買入和賣出動態之外，我們並沒有看到這方面的大量競爭。競爭主要集中在臨床差異化和療效。

Did that answer your question?

這回答了你的問題嗎？

Yeah, thanks. It's very helpful.

是的，謝謝。這非常有幫助。

Greg Harrison, Scotiabank.

加拿大豐業銀行的格雷格·哈里森。

Hey, thanks for taking the question. Congrats on another quarter of success with the launch and uptake of Attruby. I wanted to ask where you have identified areas for growth within the Attruby launch? And separately, what is BridgeBio excited about executing on for the remainder of the year?

嘿，謝謝你回答這個問題。恭喜 Attruby 的推出和普及又取得了一個季度的成功。我想問一下，您認為 Attruby 發布過程中有哪些成長領域？另外，BridgeBio 對今年剩餘時間內執行哪些計畫感到興奮？

Hey, Greg. Thanks for the question. Maybe I'll first pass it on to Neil and then to Matt to talk about what we're excited about when it comes of Attruby and the company. Neil?

嘿，格雷格。謝謝你的提問。也許我會先把它傳給尼爾，然後再傳給馬特，談談我們對阿特魯比和公司感到興奮的事情。尼爾？

I think as it pertains to Attruby, I'm excited about a lot of different things, but I'd start with the continued clinical and efficacy differentiation that we've been working on. I really like the way that we've started to port the story from overall, how does this work in the population to specific subpopulations.

我認為就 Attruby 而言，我對很多不同的事情感到興奮，但我首先要說的是，我們一直在努力的持續臨床和療效差異化。我真的很喜歡我們開始將故事從整體轉移到特定亞群體的方式，看看它在人群中是如何發揮作用的。

I think the AFib story or the cardiac arrhythmic story is a really powerful one and certainly opened my eyes to the power of these types of things, be it a variant story, be it a cardiac arrhythmic story, be it someone who has renal involvement story, on and on, et cetera, et cetera. So I think that's thing number one that gets me excited.

我認為 AFib 的故事或心律不整的故事非常有影響力，讓我認識到這些類型事物的力量，無論是變異的故事、心律不整的故事、腎臟受累的故事等等，等等。所以我認為這是讓我興奮的第一件事。

Thing number two that gets me excited is the concept of early impact and efficacy. I think for the longest time, we sort of regarded this as kind of a static picture as soon as things were diagnosed. But obviously, the earlier we go, the better we do in terms of all of the clinical trials.

第二件讓我興奮的事情是早期影響和功效的概念。我認為，長期以來，一旦診斷出病情，我們就將其視為一種靜態影像。但顯然，我們越早開始，在所有臨床試驗方面做得就越好。

And now, we're running this prevention study. And we have drugs that I think leave patients either unprotected for long periods of time, like you see knockdown go from 60% to 82% over the course of 22 months or you have prompt resolution of destabilization like you do in the case of acoramidis where you're almost immediately stabilizing that protein and getting the serum TTR levels up by day 28. So we look forward to continuing to elaborate on that early action through publications on a go-forward basis. So I would say that's one thing.

現在，我們正在進行這項預防研究。我認為我們的藥物要么會讓患者長期得不到保護，就像你看到的在 22 個月內擊倒率從 60% 上升到 82% 一樣，要么可以迅速解決不穩定問題，就像你在使用 acoramidis 的情況下所做的那樣，你幾乎可以立即穩定該蛋白質並在第 28 天內上升使血清 TTR 水平。因此，我們期待透過出版物繼續詳細闡述這項早期行動。所以我想說這是一回事。

The second thing has to do with access. Obviously, given the fact that the knockdowns are already there in polyneuropathy, given the fact that TAP is already out there, our new-to-market eds are just coming off. We're working really hard with local ISPs to make sure that it's as easy to prescribe Attruby as it is anything else.

第二件事與訪問有關。顯然，考慮到多發性神經病變中已經存在擊倒現象，考慮到 TAP 已經存在，我們新上市的版本才剛剛推出。我們正在與當地的 ISP 密切合作，以確保 Attruby 的處方能像其他藥物一樣簡單。

We're working with new technologies that allow us to work through forms that are provider-centric, and we're working carefully with PANTHERx and Orsini to have this sort of white glove service for patients.

我們正在採用新技術，使我們能夠透過以提供者為中心的表格開展工作，並且我們正在與 PANTHERx 和 Orsini 密切合作，為患者提供這種白手套服務。

So again, I think if that revs up over a long period of time, this is going to be a really nice suite of programs and support for patients.

所以，我認為，如果這種情況在很長一段時間內得到改善，那麼這將會成為一套真正好的計劃，並為患者提供支持。

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

馬特，我不知道你還有什麼要補充的。

I'd just echo maybe one of the comments that you made, making sure that anyone who wants a truly can get it is sort of our primary driver right now and how do we work through any access challenge that might appear. So the new-to-market edits coming off. We've been on the market, I guess, a little over seven months now. So this is right in that sweet spot when you start to see all of those things happening, and we are seeing that now. That's going to continue.

我只是想重複您提出的其中一條評論，確保任何真正想要的人都能得到它是我們目前的主要驅動力，以及我們如何解決可能出現的任何訪問挑戰。因此，新上市的編輯即將推出。我想，我們已經進入市場七個多月了。所以，當你開始看到所有這些事情發生時，這就處於一個最佳時刻，而我們現在就看到了這一點。這種情況將會持續下去。

We keep talking about how we believe in HCP choice. And I think we've set ourselves up, as you can see from the many programs and different things that we're doing to try to make sure that it truly is one of those choices and making sure that the physicians have access to the right therapy for each patient without any unnecessary barriers.

我們一直在談論我們如何相信 HCP 的選擇。我認為我們已經做好了準備，正如你從我們正在進行的許多項目和不同的事情中看到的那樣，我們試圖確保這確實是這些選擇之一，並確保醫生能夠為每位患者提供正確的治療，而不會遇到任何不必要的障礙。

Anupam Rama, JPM.

阿努帕姆·拉瑪（Anupam Rama），JPM。

Hey, guys. Thanks so much for taking the question. For Attruby, just thinking about prescribing metrics here, it looks like you grew 300-plus unique docs quarter over quarter. Just wondering what's resonating with both new prescribers as well as repeat prescribing dynamics, and where you're seeing the most growth in terms of academic versus community centers?

嘿，大家好。非常感謝您回答這個問題。對於 Attruby，僅考慮在這裡規定的指標，看起來您每個季度都增加了 300 多個獨特文件。只是想知道新開處方者和重複開處方者之間的共鳴是什麼，以及在學術中心和社區中心方面您看到哪裡的增長最快？

Hey, Anupam. Thank you for the question. I'm going to pass it on to Matt to talk about it.

嘿，阿努帕姆。謝謝你的提問。我要把它傳給馬特來討論。

Sure. And thanks for the question. First, I think when HCPs try Attruby and they see how quickly it works, it reinforces the decision that they made to prescribe Attruby. So one prescription just naturally turns to two and so on.

當然。感謝您的提問。首先，我認為當 HCP 嘗試 Attruby 並看到它起效如此之快時，這會強化他們開出 Attruby 的決定。因此，一個處方自然就變成了兩個，以此類推。

Patients and HCPs talk about their experience not just with a doctor-patient relationship, but also within the patient and physician communities. And their experience with the efficacy of Attruby, but also with all of our support programs that we've made available, I think, has made a tremendous impact.

患者和 HCP 不僅談論醫病關係中的經歷，還談論患者和醫生社區中的經歷。我認為，他們對 Attruby 功效的體驗以及我們提供的所有支援計劃的體驗產生了巨大的影響。

You mentioned community versus COEs, but I think this kind of an impact is equally important in both settings, maybe for different -- potentially different reasons. And so HCPs who haven't written, they write. They see this. We're out there with our field teams and they go ahead and make that decision.

您提到了社區與 COE，但我認為這種影響在兩種環境中同樣重要，可能是出於不同的——潛在的不同原因。因此，那些還沒寫過文章的 HCP 們也開始寫了。他們看到了這一點。我們的實地團隊在那裡，他們繼續做出決定。

And then once they've written and those who have written, they continue to write more Attruby while we do our best to make it easy each and every time. So I would expect that you'll see these numbers continue to grow both in the academic and the community centers every quarter.

然後，一旦他們寫完了，以及那些已經寫完的人，他們會繼續寫更多的 Attruby，而我們會盡力讓每次都變得簡單。因此我預計你會看到學術中心和社區中心的這些數字每季都會持續成長。

Paul Choi, Goldman Sachs.

高盛的保羅·崔（Paul Choi）。

Congratulations on all the progress. I want to turn maybe back to the pipeline for a moment and talk about encaleret and ADH1 and the potential lateral to hypoparathyroidism. Can you maybe speak to your level of conviction as to how success in ADH1 could translate to hypoparathyroidism?

祝賀你取得的所有進展。我想暫時回到管道上，談談 encaleret 和 ADH1 以及副甲狀腺功能減退症的潛在副作用。您能否談談您對 ADH1 成功如何轉化為副甲狀腺功能減退症的確信程度？

And maybe a little more specifically, how you're thinking about responder rates might compare to some of the existing or clinical stage therapies with regard to use of supplements or decreasing use of supplements specifically?

也許更具體地說，您認為在使用補充劑或減少使用補充劑方面，反應率與一些現有或臨床階段的療法相比如何？

Hey, Paul. Thank you for the question. I'll pass on to Ananth to talk about this.

嘿，保羅。謝謝你的提問。我會讓 Ananth 來談論這件事。

Sure. Paul, as it relates to your question, especially thinking as ADH1 is the most common genetic subset of hypopara. So there really is a strong read-through that you're alluding to a positive study in ADH1 would technically de-risk a lot of the further evaluation that we can and will endeavor to do in chronic hypoparathyroidism broadly.

當然。保羅，這與您的問題相關，尤其是考慮到 ADH1 是副甲狀腺功能減退症最常見的遺傳亞群。因此，您提到的對 ADH1 的積極研究確實具有很強的指導意義，從技術上講，這將降低我們可以並將努力在慢性副甲狀腺功能減退症方面進行的大量進一步評估的風險。

I think the key aspects that I think would be important on the read-through is one importantly, a rapid and durable benefit of blood and urine calcium. I think if we see that in ADH1 population, it will certainly be encouraging as that is a critical unmet need and a clinical need for the hypopara community.

我認為在通讀過程中重要的一個關鍵方面是，血液和尿液鈣的快速和持久益處。我認為，如果我們在 ADH1 族群中看到這種情況，那肯定會令人鼓舞，因為這是副甲狀腺功能減退症群體的一個關鍵的未滿足需求和臨床需求。

And then the other is going to be on the safety aspect. So the broad exposure at the doses we're evaluating in ADH1 will also be an important de-risking signal for the chronic hypoparathyroidism development program.

另一個是安全方面。因此，我們在 ADH1 中評估的劑量的廣泛暴露也將成為慢性副甲狀腺功能減退症發展計劃的重要降低風險信號。

In terms of the response rates, I think I'll point the community to our presentation of our Sentinel study of encaleret in chronic hypoparathyroidism, which we intend to present at the American Society of Bone Mineral Research meeting, which is taking place next month.

就回應率而言，我想我會向社區指出我們對慢性甲狀旁腺功能減退症患者恩卡雷特的哨兵研究的報告，我們打算在下個月舉行的美國骨礦物質研究學會會議上展示這份報告。

In that cohort, we resolved that within five days of dosing initiation with encaleret, 80% of study participants were able to normalize both blood and urine calcium concomitantly. This study did not evaluate whether patients can come off standard of care that will be evaluated in a longer-term study.

在該隊列中，我們發現，在開始使用恩卡雷特的五天內，80% 的研究參與者的血液和尿液鈣水平能夠同時恢復正常。這項研究並未評估患者是否可以脫離將在長期研究中評估的標準治療。

But importantly, this is a key differentiating element, which Neil touched on in the earlier remarks, which encaleret could differentiate in this patient population with three critical elements. One is oral. Two, it may have a benefit on urine calcium on 24-hour urinary calcium excretion to the extent that other therapies have not yet shown to date. And three, it could avoid long-term bone resorptive effects that may have been seen and may continue to be seen with long-term PTH replacement therapy.

但重要的是，這是一個關鍵的區別因素，Neil 在先前的評論中提到過，encaleret 可以透過三個關鍵因素來區分這群患者。一是口頭的。二是它可能對尿鈣24小時尿鈣排泄產生其他療法尚未證實的益處。第三，它可以避免長期骨吸收效應，這種效應在長期 PTH 替代療法中可能已經出現並可能繼續出現。

Andrew Tsai, Jefferies.

安德魯·蔡（Andrew Tsai），傑富瑞（Jefferies）。

Congrats on the launch execution. Thanks for taking my question. So I think one of the thematic discussion points is that Attruby could be differentiated based on a lot of data you've generated over the past few months. So operationally speaking, how do you exactly leverage the data to convince payers and doctors to use Attruby more in the first-line setting over the coming years?

祝賀發布成功。感謝您回答我的問題。因此，我認為主題討論點之一是，可以根據過去幾個月產生的大量數據來區分 Attruby。那麼從操作角度來說，您究竟如何利用數據來說服付款人和醫生在未來幾年內在一線治療中更多地使用 Attruby？

And can you summarize all the additional data sets that you plan to generate over the next, let's just say, 12 to 24 months? Can we get a glimpse of the real-world data on like NT-proBNP troponin all the way to hard outcomes data? And then really quickly, can you quantify the inventory changes in Q2 relative to Q1?

您能否總結一下您計劃在未來 12 到 24 個月內產生的所有附加資料集？我們能否從 NT-proBNP 肌鈣蛋白等真實世界數據中窺見硬性結果數據？然後很快，您能否量化第二季相對於第一季的庫存變化？

Sure. Andy, thank you for your question. I'm going to pass on to Neil to talk about the Attruby data, and then Matt and I can talk a little bit about inventory at the end. But let me pass on to Neil to talk about the differentiation.

當然。安迪，謝謝你的提問。我將把話題轉到 Neil 身上，討論 Attruby 數據，然後 Matt 和我最後可以討論一下庫存問題。但請容許我繼續與尼爾討論差異。

Yeah. Thanks, Andy. I guess your first question was what are the tactics we're using to educate in and around the data that we have produced. Obviously, there are the obvious aspects of this at conferences, publications, et cetera.

是的。謝謝，安迪。我想你的第一個問題是，我們使用什麼策略來利用我們產生的數據進行教育。顯然，在會議、出版等方面都有明顯的體現。

I think our medical affairs team has done a nice job of increasing our scientific share of voice. And obviously, I think we have the highest velocity of publication in this sector so far. So that's given us an opportunity to share something new with the physicians that we do see.

我認為我們的醫療事務團隊在提高我們的科學發言權方面做得很好。顯然，我認為我們迄今為止在該領域的出版速度是最快的。因此，這給了我們一個機會與我們見到的醫生分享一些新的東西。

The second piece of it, honestly, has been conversations as you say, put some fraction of your patients on the Attruby to Matt's earlier point and see what the experience is. And I think we feel very comfortable, obviously, with the two real-world evidence studies that have posted to date out of Dr. Masri's lab and Dr. Moore's lab, that continued outperformance in the real-world setting, and we're doing a lot more of this now on the health economic and real-world setting side, just what do hospitalizations look like with one stabilizer versus the next, what do overall expenses look like with various SPs, et cetera, et cetera. So we're doing a lot of that type of work, and I think that's another way that we can allow the data to resonate.

老實說，第二部分就是像您所說的那樣進行對話，讓一部分患者接受馬特之前提到的 Attruby 治療，看看效果如何。我認為，我們對迄今為止 Masri 博士實驗室和 Moore 博士實驗室發布的兩項真實世界證據研究感到非常滿意，這些研究在真實世界環境中繼續表現出色，我們現在在健康經濟和真實世界環境方面做了更多這方面的研究，例如使用一種穩定劑與另一種穩定劑的住院情況如何，使用各種 SP 的總體費用如何，等等。所以我們做了很多這類工作，我認為這是另一種讓數據產生共鳴的方式。

A summary of the data, I think of the new data that we just put out has to be the variant data, the 59% relative risk reduction with the stat sig, the AFib data, the 70% reduction along with the 43% reduction in CVH-associated hospitalization.

數據總結，我認為我們剛發布的新數據必須是變數數據，使用統計訊號，相對風險降低了 59%，AFib 數據，降低了 70%，同時 CVH 相關住院率降低了 43%。

And then the third, and I think, honestly, the most important was the connection once again between ever higher levels of stabilization as measured by serum TTR and better outcomes as measured by mortality. But I think those were the salient data pieces to date. And then on a go-forward basis, you can expect that -- as I mentioned earlier, that we're going to publish on all of those fronts plus more.

第三點，我認為，說實話，最重要的是，血清 TTR 衡量的更高水平的穩定性與死亡率衡量的更好結果之間的再次聯繫。但我認為這些是迄今為止最突出的數據。然後，從未來來看，你可以期待——正如我之前提到的，我們將在所有這些方面以及更多方面發佈內容。

One thing we're definitely going to be looking at is the rapidity of response because I think that now that we've got the PK data from the knockdowns, I think that actually is a huge differentiator of the rapidity of response. So you should see a lot more publications coming out on that front.

我們肯定會關注的一件事是反應速度，因為我認為現在我們已經獲得了擊倒的 PK 數據，我認為這實際上是反應速度的一個巨大差異。因此，你會看到更多有關該方面的出版物。

I think the second is the real-world experience with these products, both from the standpoint of biomarkers. I think NT-pro, yes, definitely. I think serum TTR, yes, definitely. And then quality of life and hospitalization measures and then health economic parameters as well.

我認為第二個是這些產品的實際體驗，都是從生物標記的角度來看的。我認為是 NT-pro，是的，絕對是。我認為血清 TTR，是的，絕對是。然後是生活品質和住院措施，然後是健康經濟參數。

It's interesting, if you go over to Europe, like we -- there are countries where we're the only brand, like we won the national bid, and there are major hospitals in areas like Germany where Attruby is frontline. And I think we're sort of looking at those types of very dispassionate, but yet still trying to make a choice between these various datasets, analogs and saying what really resonated with them in that data and how do we bring some of those messages over to the US market.

有趣的是，如果你去歐洲，你會發現——在某些國家我們是唯一的品牌，例如我們贏得了國家競標，在德國等地區，Attruby 是主要醫院的前線。我認為我們正在以非常冷靜的態度看待這些問題，但仍試圖在這些不同的數據集和類似物之間做出選擇，並說出這些數據中真正引起他們共鳴的是什麼，以及我們如何將這些訊息傳遞到美國市場。

So I don't know, Matt or anyone else, if you want to add anything?

所以我不知道，馬特或其他人，是否想補充一些什麼？

No, I can touch on the inventory question, if that works. I just would make a couple of comments. One, the held inventory is lower in Q2 versus Q1 as suppliers get used to our just-in-time model.

不，如果可以的話，我可以談談庫存問題。我只想發表幾點評論。首先，由於供應商已經習慣了我們的準時制模式，因此第二季的庫存量低於第一季。

And so why? Well, you can get Attruby in less than 48 hours. We talk about that for patients all the time, but it's not just patients. Our suppliers can also get Attruby in less than 48 hours as well.

那為什麼呢？好吧，您可以在不到 48 小時內獲得 Attruby。我們一直在為患者談論這個問題，但不僅僅是患者。我們的供應商也可以在不到 48 小時內獲得 Attruby。

So the sort of old way of doing the supply and demand model, which is, hey, I have to hold multiple weeks of inventory or more because I'm worried about running out or not being able to get some. I think we've sort of changed the game a bit in that.

因此，舊的供需模型方法是，嘿，我必須持有數週或更長時間的庫存，因為我擔心用完或無法獲得一些庫存。我認為我們在某種程度上改變了遊戲規則。

So now that people realize that, that's true, they don't have to hold the sort of historical larger levels. And so you're seeing that play out in the market now. And I think that's just due to the confidence that our distributors have in us.

所以現在人們意識到了這一點，確實如此，他們不必堅持那種歷史性的更大層次。所以你現在就看到市場上出現這種情況。我認為這只是因為我們的經銷商對我們的信心。

As Matt said, the days went down because of the accelerating patient demand and the model being more familiar to our suppliers. So I appreciate your question.

正如馬特所說，由於患者需求不斷增加，而且我們的供應商對該模式更加熟悉，所以天數減少了。我很感謝你的提問。

And that concludes our Q&A for today. I will now hand it back to the company.

今天的問答到此結束。我現在將其交還給公司。

Thank you, all, for joining us on our Q2 earnings call. We look forward to updating you again in our next earnings call. Thank you. Bye.

感謝大家參加我們的第二季財報電話會議。我們期待在下次收益電話會議上再次向您通報最新情況。謝謝。再見。

And this concludes today's conference. Thank you for your participation. You may now disconnect.

今天的會議到此結束。感謝您的參與。您現在可以斷開連線。