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Operator
Operator
Good afternoon. I'll be your conference operator today.
午安.我今天將擔任你們的會議接線生。
(Operator Instructions) Thank you.
(操作說明)謝謝。
Before we begin, I'd 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.
在開始之前,我想提醒大家,今天的電話會議可能包含聯邦證券法意義上的前瞻性陳述,包括但不限於有關 BridgeBio 未來營運和財務業績、業務計劃和前景以及戰略的陳述。
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.
這些聲明是基於目前的預期和假設,但存在風險和不確定性,可能導致實際結果與這些前瞻性聲明中明示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,請參閱今天發布的收益報告中的披露資訊以及 BridgeBio 的定期報告和提交給美國證券交易委員會的文件。
All statements made here are based on information available to BridgeBio, as of today. The company undertakes no obligation to update any forward-looking statements made during this call, except as required by law.
本文所有陳述均基於截至今日BridgeBio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任何前瞻性陳述的義務。
With that completed, BridgeBio, you may begin your conference.
BridgeBio,完成這些步驟後,你們就可以開始會議了。
Chinmay Shukla - Senior Vice President - Strategic Finance
Chinmay Shukla - Senior Vice President - Strategic Finance
Good afternoon, everyone. Thank you for joining BridgeBio Pharma's fourth-quarter 2025 earnings call.
大家下午好。感謝您參加 BridgeBio Pharma 2025 年第四季財報電話會議。
My name is Chinmay Shukla. I'm the Senior Vice President of Strategic Finance at BridgeBio. With me today are Neil Kumar, our CEO, who will provide opening remarks and discuss overall corporate performance; Matt Outten, our Chief Commercial Officer, who will provide more details about our commercial performance, particularly the continued success of Attruby; and Tom Trimarchi, our President and CFO, who will review our financial results.
我的名字是欽邁·舒克拉。我是 BridgeBio 的策略財務資深副總裁。今天與我一同出席的有:首席執行官尼爾·庫馬爾,他將致開幕詞並討論公司整體業績;首席商務官馬特·奧滕,他將詳細介紹我們的商業業績,特別是 Attruby 的持續成功;以及總裁兼首席財務官湯姆·特里馬爾基,他將回顧我們的財務業績。
During today's call, we will review our continued strong commercial execution in Attruby's fourth quarter and first full year on the market.
在今天的電話會議上,我們將回顧 Attruby 在第四季度和上市第一年持續強勁的商業執行情況。
More importantly, we will discuss what we believe is a transformative inflection point for BridgeBio, marked by positive top-line Phase 3 results for Encaleret in ADH1, BBP-418 in LGMD2I, as well as positive top-line data for Infigratinib in achondroplasia. With three successful late-stage read-outs across our pipeline, we are entering a new phase of value creation and portfolio maturation.
更重要的是,我們將討論我們認為對 BridgeBio 而言具有變革意義的轉折點,其標誌是 Encaleret 在 ADH1 中的 3 期臨床試驗中取得了積極的初步結果,BBP-418 在 LGMD2I 中的 3 期臨床試驗中取得了積極的初步結果,以及 Infigratinib 在軟骨發育不全的初步試驗中取得了積極的 3 期臨床數據。隨著我們研發管線中三個後期試驗階段的成功進展,我們正在進入價值創造和產品組合成熟的新階段。
We will also review our robust financial position and how it supports our regulatory launch and lifecycle expansion priorities across these programs.
我們還將審查我們穩健的財務狀況,以及它如何支持我們在這些專案中的監管啟動和生命週期擴展優先事項。
Following our prepared remarks, we will open the call for questions. For the Q&A session, we will also be joined by Ananth Sridhar, Anna Wade, and Justin To, who lead Encaleret, BBP-418, and Infigratinib, respectively.
在我們發言完畢後,我們將開始接受提問。在問答環節,我們也將邀請 Encaleret、BBP-418 和 Infigratinib 的負責人 Ananth Sridhar、Anna Wade 和 Justin To 參加。
With that, I'll turn it over to Neil.
接下來,我將把麥克風交給尼爾。
Neil Kumar - Chief Executive Officer, Director
Neil Kumar - Chief Executive Officer, Director
Thanks, everyone, for taking the time today.
謝謝大家今天抽出時間。
This is our first earnings call since we reported the results of our Phase 3 study with Infigratinib, which delivered a successful outcome for the community we serve in achondroplasia.
這是我們自公佈 Infigratinib 3 期研究結果以來的首次財報電話會議,該研究為我們服務的軟骨發育不全患者群體帶來了成功的結果。
Altogether, with ATTR cardiomyopathy, this brings us to four large post-Phase 3 programs.
總而言之,加上 ATTR 心肌病變,我們現在有四個大型的 3 期後計畫。
I want to begin my comments today by discussing what that portends, in terms of the shape of the firm. In short order, this company will turn from a cash-consumptive business to one that generates significant cash flows.
今天我想先談談這對公司未來的發展意味著什麼。不久之後,這家公司將從現金消耗型企業轉變為能夠產生大量現金流的企業。
The shape of those cash flows connects to the clinical profiles that we will spend some time discussing today. But before I get into that, though, I want to take a moment to paint the picture of what the overall economic productivity of our post-Phase 3 pipeline might look like over the coming 24 months.
這些現金流的形態與我們今天將要討論的臨床概況有關。不過,在深入探討這個問題之前,我想花點時間描繪一下我們第三階段後產品線在未來 24 個月內的整體經濟生產力可能會是什麼樣子。
I do so because the immediacy of the transition from a cash-losing business to a cash-flowing business is one that happens quickly and can open up new opportunities for a firm as successful at R&D as ours.
我這樣做是因為從虧損企業到獲利企業的轉變速度很快,可以為像我們這樣在研發方面非常成功的公司創造新的機會。
Last year, we used $446 million for the year, net of revenue. Cash burn declined in the fourth quarter, relative to the third quarter; and throughout 2025, driven by rising revenues and improving operating leverage.
去年,我們扣除收入後,全年使用了 4.46 億美元。第四季現金消耗量較第三季有所下降;並且到 2025 年全年,在營收成長和營運槓桿改善的推動下,現金消耗量將持續下降。
Similarly, while we are going to make significant investments for launch readiness against our next three products, we expect cash burn to roughly hold steady through this year and start declining by the end of next year, given expected increases in Attruby revenue.
同樣,雖然我們將為接下來的三款產品的上市準備工作進行大量投資,但鑑於 Attruby 收入的預期增長,我們預計今年的現金消耗將大致保持穩定,並在明年年底開始下降。
That's less interesting to me, though, than the following fact: Absent any strategic moves, our current pipeline will begin to generate cash in late '27 and will be a cash-generation engine by 2028. The profile we anticipate having in 2028 would distinguish us in a field of genetic disease and, more broadly, would place us in the top 20 to 30 firms in the biopharmaceutical sector, from the perspective of cash flow or EBITDA. This projected future is driven by growing and diversified revenue streams connected to our four post-Phase 3 assets, which we believe, in 2028, will generate more than $600 million in profit.
不過,對我來說,以下事實更有趣:如果沒有採取任何戰略舉措,我們目前的專案儲備將於 2027 年底開始產生現金,到 2028 年將成為現金流引擎。我們預計到 2028 年將擁有這樣的企業形象,這將使我們在遺傳疾病領域脫穎而出,更廣泛地說,從現金流或 EBITDA 的角度來看,這將使我們躋身生物製藥行業前 20 至 30 家公司之列。這項預期未來的驅動力是與我們四個第三階段後資產相關的不斷增長和多元化的收入來源,我們相信,到 2028 年,這些資產將產生超過 6 億美元的利潤。
The value of any firm relates back to ROIC, revenue growth, and cost of capital. Against all three metrics, we believe this firm has a rapidly improving profile over the next three years.
任何公司的價值都與投資報酬率、收入成長和資本成本有關。根據這三項指標,我們認為這家公司在未來三年內將迅速提升其發展動能。
Our anticipated profit is even more impressive, when one considers that we've been able to advance programs from the pre-clinical stage through Phase 3 at under $300 million -- in some cases, considerably under that; and at higher probability of technical success than industry average, suggesting an engine that could drive repeatable organic growth.
考慮到我們能夠以不到 3 億美元的成本將專案從臨床前階段推進到 3 期(在某些情況下,成本甚至遠低於此),並且技術成功的概率高於行業平均水平,我們預期的利潤就更加令人印象深刻了,這表明我們擁有能夠推動可重複的內生增長的引擎。
Of course, all of this now highly probable growth is underpinned by clinical data that we have already generated across our four Phase 3s; as well as data that we continue to generate; and a commercial engine that continues to grow and grow share in the ATTR cardiomyopathy marketplace.
當然,所有這些極有可能的成長都建立在我們已在四項 3 期臨床試驗中產生的臨床數據、以及我們持續產生的數據,以及不斷增長並在 ATTR 心肌病變市場中擴大份額的商業引擎之上。
We intend to establish that commercial engine as best-in-class for both first-to-market and competitive market launches in genetic disease.
我們的目標是將該商業引擎打造為遺傳疾病領域首發和競爭性市場上市的最佳引擎。
Despite continued strong execution across our business, our recent share price performance does not reflect the progress we've made. We believe this disconnect is primarily driven by uncertainties surrounding the tafamidis IP situation, which I will address directly in a moment.
儘管我們各項業務持續強勁發展,但我們近期的股價表現並未反映出我們所取得的進展。我們認為這種脫節主要是由於圍繞 tafamidis IP 情況的不確定性造成的,我稍後將直接對此進行說明。
Importantly, nothing about our fundamentals has deteriorated. If anything, our position has strengthened commercially, clinically, and strategically.
重要的是,我們的基本面沒有任何惡化。如果有什麼改變的話,那就是我們在商業、臨床和策略方面的地位都得到了加強。
As we execute against our milestones, we believe the intrinsic value of BridgeBio continues to increase.
隨著我們逐步實現既定目標,我們相信 BridgeBio 的內在價值將持續成長。
We are keenly aware of the gap between intrinsic value and our current market valuation. We are actively evaluating all appropriate options to ensure that shareholder value is properly recognized, over time.
我們非常清楚內在價值與我們目前的市場估值之間的差距。我們正在積極評估所有合適的方案,以確保股東價值能夠隨著時間的推移得到適當的認可。
More specifically, over the past three months, given the de-risking of LGMD2I, our patient-finding progress in ADH1; and the clearly differentiated Infigratinib read-out in achondroplasia, we believe our intrinsic value has increased and its error bars have narrowed.
更具體地說,在過去的三個月裡,鑑於 LGMD2I 的風險降低、我們在 ADH1 患者發現方面取得的進展,以及 Infigratinib 在軟骨發育不全中明確的差異化結果,我們認為我們的內在價值已經增加,誤差範圍也已經縮小。
With over $1 billion dollars of capital on our balance sheet, additional significant amounts of capital available away from the equity markets, and with the base business fully financed, we believe we have retained optionality to capture the value you all have helped us to create.
我們資產負債表上擁有超過 10 億美元的資本,除了股權市場之外還有大量其他可用資本,而且基礎業務也已獲得充分融資,我們相信我們仍然擁有獲取大家幫助我們創造的價值的選擇權。
With that said, I want to spend some time today reiterating some of the important clinical data, especially as it pertains to the Infigratinib read-out. I want to highlight ongoing commercial readiness activities for LGMD2I in ADH1. I want to talk about -- and Matt will elaborate on this -- our continued commercial progress in ATTR cardiomyopathy.
綜上所述,我今天想花點時間重申一些重要的臨床數據,特別是與 Infigratinib 的讀數相關的數據。我想重點介紹 ADH1 中 LGMD2I 正在進行的商業化準備活動。我想談談——馬特會對此進行詳細闡述——我們在 ATTR 心肌病變領域持續取得的商業進展。
On the data side, I'll begin with our recent Phase III read-out in achondroplasia. As many of you know, we were privileged to generate data, alongside the achondroplasia community, that suggests a differentiated profile for Infigratinib.
在數據方面,我將從我們最近在軟骨發育不全症方面進行的 III 期試驗結果開始。正如你們許多人所知,我們很榮幸能與軟骨發育不全群體一起產生數據,這些數據表明 Infigratinib 具有差異化的特徵。
The study successfully met the primary endpoint of change from baseline and average height velocity at week 52, with a p-value of less than 0.0001, with a mean treatment difference against placebo of 2.1 centimeters per year.
該研究成功達到了第 52 週基線變化和平均身高增長速度的主要終點,p 值小於 0.0001,與安慰劑相比,平均治療差異為每年 2.1 公分。
In key secondary endpoints, Infigratinib also demonstrated the first statistically significant improvement in body proportionality in achondroplasia, with a least squares mean difference of minus 0.05, with a p-value of less than 0.05 against placebo in children younger than eight years old, which were more than 50% of our participants; and in a pre-specified analysis.
在關鍵次要終點方面,Infigratinib 也首次在軟骨發育不全患者的身體比例方面顯示出統計學意義上的顯著改善,最小二乘均值差為 -0.05,p 值小於 0.05,與安慰劑相比,8 歲以下兒童(占我們參與者的 50% 以上)的療效更佳;並且在預先設定的分析中也觀察到了這一結果。
It succeeded on change from baseline in height Z-score at week 52, with a least squares mean increase on the treatment arm of 0.41 standard deviations at week 52, associated with a p-value of less than 0.0001.
在第 52 週,身高 Z 分數較基線發生了變化,治療組在第 52 週的最小平方法平均值增加了 0.41 個標準差,p 值小於 0.0001。
All of these numbers, as a reminder, are best in class and unique to Infigratinib.
再次提醒大家,這些數據都是同類最佳,而且是 Infigratinib 獨有的。
Infigratinib was also well-tolerated, with no discontinuations or serious adverse events related to study drug.
Infigratinib 的耐受性也很好,沒有因研究藥物而導致的停藥或嚴重不良事件。
Three cases or 4%, [on-active], of hyperphosphatemia, considered mild and transient, with no cases requiring either dose reduction or discontinuation and no adverse events associated with the inhibition of FGFR1 or 2; for example, retinal or corneal adverse events.
3 例(4%)[非活性]高磷血症,被認為是輕微且短暫的,沒有病例需要減少劑量或停止治療,也沒有與 FGFR1 或 2 抑制相關的不良事件;例如視網膜或角膜不良事件。
As a reminder, Infigratinib's differentiated oral route of administration and its mechanism, which uniquely targets this well-described condition at its source, produced Phase 2 efficacy and safety results that were highly differentiated.
需要提醒的是,Infigratinib 獨特的口服給藥途徑及其作用機制,能夠從根源上獨特地靶向這種已充分描述的疾病,其 2 期療效和安全性結果具有高度差異化。
Our Phase 3 data have confirmed those efficacy and safety profiles. Interestingly, as we have begun to test this product profile since the read-out, we have heartily found that our base case achievable market share has risen from the 52% I mentioned in my JP Morgan talk to an excess of 65% peak year share.
我們的3期臨床試驗數據證實了這些療效和安全性特徵。有趣的是,自從我們開始測試該產品概況以來,我們欣喜地發現,我們基本情況下可實現的市場份額已從我在摩根大通演講中提到的 52% 上升到峰值年份超過 65% 的份額。
In addition, that preliminary market research suggests not only differentiated peak year share but also significant market expansion, similar to what we've seen when orals enter markets as diverse as Fabry, migraine, hereditary angioedema, and in many other categories.
此外,初步市場調查顯示,不僅高峰年份的份額存在差異,而且市場規模也顯著擴大,這與我們之前看到的口服藥物進入法布瑞氏症、偏頭痛、遺傳性血管性水腫等多種市場以及許多其他類別的情況類似。
In fact, a recent analysis done at our [Revenue Institute], in partnership with MIT, suggests that, across indications, the launch of an oral product increased the sales in the category by about 170% over five years from launch of the first oral product.
事實上,我們[收入研究所]最近與麻省理工學院合作進行的一項分析表明,在所有適應症中,口服產品的推出使該類別的銷售額在第一個口服產品推出後的五年內增長了約 170%。
With regards to our efforts in LGMD2I, we're excited to be presenting the full dataset from our study at the upcoming Muscular Dystrophy Association Conference, where Dr. Katherine Mathews from the University of Iowa will give the keynote.
關於我們在 LGMD2I 方面的努力,我們很高興能在即將舉行的肌肉萎縮症協會會議上展示我們研究的完整數據集,屆時愛荷華大學的 Katherine Mathews 博士將發表主題演講。
We have built and onboarded a dedicated commercial leadership team to ensure we are fully prepared to serve the LGMD2I community from day 1. This is a population with profound unmet need. We are ready to execute.
我們已經組建並組建了一支專門的商業領導團隊,以確保我們從一開始就做好充分準備,為 LGMD2I 社群提供服務。這是一個存在大量未滿足需求的群體。我們已做好執行準備。
At the same time, we are not limiting our focus to the patients already identified. We are actively working to expand awareness, accelerate diagnosis, and help uncover individuals who may be unidentified within the broader LGMD or Becker muscular dystrophy populations.
同時,我們並沒有將關注點局限於已確定的患者。我們正在積極努力提高公眾意識,加快診斷速度,並幫助發現可能在更廣泛的肢帶型肌肉營養不良症 (LGMD) 或貝克爾肌肉營養不良症 (Becker muscular dystrophy) 患者群體中未被識別的個體。
Our goal is simple: to find every patient who can benefit and to ensure we are ready to reach them the moment we are able to.
我們的目標很簡單:找到每一位可能受益的患者,並確保我們能夠在條件允許的第一時間為他們提供幫助。
Moving to ADH1, our other first-in-class product, Encaleret, we continue our patient-finding efforts, which have already identified in excess of 1,700 unique patients in claims data. We also recently had pre-NDA communications with the agency, which were supportive of our expectations. We continue to anticipate the launch of both Encaleret and BBP-418 in late 2026 or early 2027.
接下來是我們的另一個同類首創產品——安卡瑞特(Encaleret),我們繼續進行患者尋找工作,目前在理賠數據中發現了超過 1700 名獨特的患者。我們最近也與該機構進行了保密協議簽署前的溝通,他們的反應與我們的預期相符。我們仍預期 Encaleret 和 BBP-418 將於 2026 年底或 2027 年初推出。
Finally, and most importantly, I want to talk about our ATTR cardiomyopathy franchise., where, as I suggested recently, we continue to elaborate, not only on the fullness of the best-in-class stabilizer hypothesis but also on our differentiation in the real-world setting and our ability to impact patient health as early as one month -- by far, the earliest impact we see from any medicine in this space.
最後,也是最重要的一點,我想談談我們的 ATTR 心肌病變產品線。正如我最近所建議的,我們將繼續完善,不僅要充分闡述一流的穩定劑假說,還要闡述我們在現實世界中的差異化優勢,以及我們最早在一個月內就能對患者健康產生影響的能力——到目前為止,這是我們在這個領域看到的任何藥物所能產生的最早影響。
We already pre-announced the fourth-quarter Attruby net product revenue of $146 million, which corresponded to greater than 25% [MBRX] share, as of December 31, 2025.
我們已經預先宣布,截至 2025 年 12 月 31 日,Attruby 第四季淨產品收入為 1.46 億美元,相當於 [MBRX] 市場份額的 25% 以上。
Over the last few weeks, Attruby's commercial momentum has continued. As of February 20, we have seen 7,804 unique patient prescriptions written by 1,856 unique prescribers. You'll hear more from Matt about what this means, in terms of competitive differentiation.
在過去的幾周里,Attruby 的商業發展勢頭持續強勁。截至 2 月 20 日,我們看到 1856 位不同的處方醫生開出了 7804 張不同的患者處方。你會從 Matt 那裡聽到更多關於這在競爭差異化方面意味著什麼的內容。
As I alluded to, as well, in my JP M talk, we are continuing to interrogate the importance of the cardiorenal axis, which seems to be uniquely involved in our early onset of action.
正如我在JP M演講中提到的那樣,我們正在繼續探討心腎軸的重要性,它似乎與我們早期行動的啟動有著獨特的聯繫。
We have also noted, with great interest, the recent PNAS paper that I only had a bit of time to talk about at JP Morgan, which suggests that binding enthalpy best predicts the conformational stabilization imparted by kinetic stabilizers, as opposed to binding affinity, Kd, or Gibbs free energy.
我們也饒有興趣地註意到最近發表在 PNAS 上的一篇論文,我在摩根大通只有一點時間談論這篇論文,該論文表明,與結合親和力、Kd 或吉布斯自由能相比,結合焓能更好地預測動力學穩定劑賦予的構象穩定性。
As we have shown through [ITC] experiments and as we published in Miller et al., we have a vastly superior enthalpic binding mode than [does] tafamidis, which, in concert with superior binding kinetics, continues to reinforce Attruby's better stabilization profile.
正如我們透過 [ITC] 實驗所證明的,以及我們在 Miller 等人發表的文章中所證明的,我們的焓結合模式比 tafamidis 好得多,這與更優異的結合動力學相結合,繼續強化了 Attruby 更好的穩定性特徵。
A recent bevy of literature further supports that increases in serum TTR are associated reliably with decreases in the relative risk of mortality. These papers suggest that for every mg per deciliter increase in serum TTR, you decrease the risk of mortality at 30 months by approximately 5%.
最近大量的文獻進一步證實,血清 TTR 的升高與死亡相對風險的降低密切相關。這些論文表明,血清 TTR 每增加 1 mg/dL,30 個月時的死亡風險就會降低約 5%。
As a reminder, we observed in our Phase 3 study that patients increased their serum TTR by 3 mgs per deciliter when moving from tafamidis to acoramidis. This suggests a whopping 15% relative risk reduction in mortality, when moving from tafamidis to acoramidis.
提醒一下,我們在 3 期研究中觀察到,當患者從 tafamidis 轉用 acoramidis 時,其血清 TTR 增加 3 mg/dL。這表明,從服用 tafamidis 到服用 acoramidis,死亡率相對風險降低了 15%。
Let me also address the recent stock volatility, which is largely centered on questions regarding Vyndamax IP and the potential for generic entry:
我還想談談最近的股價波動,這主要集中在 Vyndamax 智慧財產權和仿製藥可能進入市場的問題上:
First, it's important to separate two things: the legal process around tafamidis and the fundamental strength of Attruby. Our confidence in Attruby is rooted in its clinical profile and market positioning, not a particular IP outcome.
首先,需要區分兩件事:圍繞 tafamidis 的法律程序和 Attruby 的根本實力。我們對 Attruby 的信心源自於其臨床表現和市場定位,而不是某個特定的智慧財產權成果。
On the IP front, the Pfizer decision to withdraw one of its EU patents defending the Vyndamax equivalent product was unexpected. That said, it did not materially change how we view the EU market, given Vyndamax's orphan drug exclusivity in wild-type ATTR cardiomyopathy through 2030, which is now; and how we have always consistently modeled that geography.
在智慧財產權方面,輝瑞決定撤回在歐盟的一項專利,該專利旨在保護 Vyndamax 的同類產品,這一決定出乎意料。儘管如此,鑑於 Vyndamax 在野生型 ATTR 心肌病變領域擁有孤兒藥獨佔權至 2030 年(即現在),並且我們一直以來都是這樣看待歐盟市場的,因此這並沒有實質性地改變我們對歐盟市場的看法。
In the US, which is the market of greatest importance to us, we believe the IP position is stronger. The patent claims at issue are narrower than those in Europe, including specific XRPD peak limitations for form I that were not part of the EU case.
在美國這個對我們而言最重要的市場,我們認為我們的智慧財產權地位更加穩固。所涉專利權利要求比歐洲的權利要求範圍更窄,包括針對 I 型晶體的特定 XRPD 峰值限制,而歐盟案件中並未涉及這些限制。
In addition, US law applies a higher legal threshold for invalidity, requiring that challengers prove by clear and convincing evidence that a prior art process necessarily and inevitably produces the claimed form, not merely that it could or likely would.
此外,美國法律對無效性適用更高的法律門檻,要求挑戰者以清晰且令人信服的證據證明現有技術的工藝必然且不可避免地產生所要求的形式,而不僅僅是可能或很可能產生。
That said, IP trials are inherently uncertain. We will reassess, as more information comes available, following the US proceedings in April.
也就是說,智慧財產權訴訟本身就存在不確定性。我們將根據四月美國方面的審理結果,在獲得更多資訊後重新評估情況。
Stepping back, however, our strategy does not depend on tafamidis' IP. Attruby has demonstrated near-complete stabilization, rapid clinical benefit, and meaningful differentiation in ATTR cardiomyopathy. It is already priced at a discount to Vyndamax and is less than half the price of the knock-down technologies.
但從另一個角度來看,我們的策略並不依賴 tafamidis 的智慧財產權。Attruby 已證明在 ATTR 心肌病變中能夠實現近乎完全的穩定、快速的臨床獲益和有意義的分化。它的定價已經比 Vyndamax 低,而且價格不到可拆卸技術的一半。
We believe physicians are making decisions based on clinical performance, not simply price. Prescribing trends we are seeing are reflecting that.
我們認為醫生做出決定是基於臨床療效,而不僅僅是價格。我們觀察到的處方趨勢反映了這一點。
Even in a hypothetical scenario involving generic tafamidis, we do not believe a less efficacious product would undermine the role of a clinically differentiated therapy in a serious progressive disease like ATTR-CM.
即使在涉及通用他法米地的假設情境中,我們也不認為療效較差的產品會削弱臨床差異化療法在像 ATTR-CM 這樣的嚴重進行性疾病中的作用。
In short, we remain confident in Attruby's positioning today and in the years ahead.
總之,我們對 Attruby 目前及未來幾年的發展前景依然充滿信心。
With that, I'll turn it over to Matt to speak more specifically about the commercial momentum we're seeing.
接下來,我將把發言權交給 Matt,讓他更具體地談談我們目前看到的商業發展動能。
Matt Outten - Chief Commercial Officer
Matt Outten - Chief Commercial Officer
Thank you, Neil.
謝謝你,尼爾。
Consistent with what we highlighted at JP Morgan in January, we believe 2025 reflected strong commercial momentum for Attrub. It represented an important step forward in advancing three additional product candidates towards potential commercialization.
正如我們在 1 月摩根大通會議上所強調的那樣,我們認為 2025 年 Attrub 將展現出強勁的商業發展勢頭。這標誌著另外三個候選產品在實現潛在商業化方面邁出了重要一步。
In the fourth quarter, we delivered 35% quarter-over-quarter growth in net product revenue, ending the year with $502.1 million in total revenue and $154.2 million in quarter four. Of this, the net product revenue for Attruby was $362.4 million and $146 million, respectively; while new patient growth accelerated in the latest quarter to reach 7,804 new patient starts.
第四季度,我們的淨產品收入季增 35%,全年總收入達到 5.021 億美元,第四季營收達到 1.542 億美元。其中,Attruby 的淨產品收入分別為 3.624 億美元和 1.46 億美元;而最新季度新患者成長加速,達到 7,804 例新患者。
When viewed in conjunction with the IQVIA data, it becomes clear that Attruby is accelerating growth at a significantly faster rate versus previous quarters, while the competition lags behind. This is particularly obvious in first-line patients, where the exceptional data for Attruby, along with our experienced field teams, have driven sales to the highest levels since launch, surpassing all expectations.
結合 IQVIA 數據來看,很明顯,Attruby 的成長速度比前幾季明顯加快,而競爭對手則落後了。這一點在一線患者中尤其明顯,Attruby 的卓越數據,加上我們經驗豐富的現場團隊,已將銷售額推至上市以來的最高水平,超出了所有預期。
We have historically given out the new patient start number each quarter and have done so again despite the competition not offering similar numbers. Moving forward, we will not be offering new patient start data because of this lack of transparency by others.
我們歷來每季都會發放新病患起始編號,儘管競爭對手沒有提供類似的編號,但我們今年仍然這樣做了。鑑於其他方面缺乏透明度,今後我們將不再提供新患者的起始數據。
Continuing to do so would put us in a competitive disadvantage. But our expectations are that Attruby will continue to grow, as it is done since launch and as exemplified with today's update.
繼續這樣做會使我們在競爭中處於劣勢。但我們預計 Attruby 將繼續發展壯大,就像它自推出以來一直發展壯大一樣,今天的更新也證明了這一點。
As adoption grows, particularly in the first-line setting, we remain focused on ensuring patients and healthcare professionals have clear, balanced information, when evaluating therapy options. That focus is especially relevant, given recent updates we've seen in competitor direct-to-consumer communications.
隨著採用率的提高,尤其是在一線治療中,我們將繼續致力於確保患者和醫療保健專業人員在評估治療方案時獲得清晰、平衡的資訊。鑑於我們最近在競爭對手的直接面向消費者的溝通方面看到的更新,這種關注點尤其重要。
After receiving a letter from the FDA several months ago and pulling their television ad from the airwaves, Alnylam has returned to TV advertising. But, of note, the safety section has been updated.
幾個月前,Alnylam 公司收到美國食品藥物管理局 (FDA) 的一封信,並撤下了電視廣告。如今,該公司又重新開始投放電視廣告。但值得注意的是,安全性部分已經更新。
Besides adding the warning about the risk of vutrisiran lowering vitamin A and potentially affecting vision, the ad now points out the risk for several additional concerns; namely, joint pain, pain in the arms and legs, and shortness of breath.
除了警告 vutrisiran 會降低維生素 A 水平並可能影響視力外,該廣告現在還指出了其他幾個令人擔憂的風險;即關節疼痛、手臂和腿部疼痛以及呼吸急促。
In a population already often suffering from these issues, the possibilities of amplifying, compounding, or causing shortness of breath; and/or pain in the joints. and/or pain in the arms; and/or pain in the legs should be highlighted to any patient considering treatment with vutrisiran.
對於已經經常遭受這些問題困擾的人群來說,任何考慮使用 vutrisiran 治療的患者都應該注意,該藥物可能會加劇、惡化或導致呼吸困難;和/或關節疼痛;和/或手臂疼痛;和/或腿部疼痛。
The fact that these risks have been omitted but are now stated at the end of each commercial and, hopefully, all promotional materials and messaging will correct and highlight for patients and healthcare professionals some things to consider with vutrisiran treatment, especially if they are a newly diagnosed patient versus someone who has tried all other options.
這些風險之前被忽略了,但現在在每個廣告的結尾都已說明,希望所有宣傳材料和資訊都能糾正並強調患者和醫療保健專業人員在使用 vutrisiran 治療時需要考慮的一些事項,特別是對於新確診的患者,而不是那些已經嘗試過所有其他治療方案的患者。
If we shift back to the reasons for the growth of a Attruby, there are several driving factors:
如果我們回到阿特魯比家族發展壯大的原因,會發現有幾個主要驅動因素:
First, the number of prescribing HCPs continues to grow. But, in equal importance, HCPs who start using Attruby continue using Attruby.
首先,開處方的醫護人員數量持續增加。但同樣重要的是,開始使用 Attruby 的醫療保健專業人員會繼續使用 Attruby。
We are seeing repeat use and stable patient persistence, which tells us physicians are comfortable with what they are seeing in their practice. We believe the success we have seen in 2025 is driven by Attruby's differentiated profile as the only near-complete stabilizer on the market, in contrast to therapies that rely on partial stabilization or partial knock-down mechanisms of action.
我們看到重複使用率和穩定的患者依從性,這告訴我們,醫生對他們在實踐中看到的情況感到滿意。我們相信,我們在 2025 年取得的成功,得益於 Attruby 的獨特優勢,它是市場上唯一接近完全的穩定劑,而其他療法則依賴於部分穩定或部分敲除作用機制。
Attruby has also demonstrated the fastest time to separation to date, an attribute that matters, as physicians seek therapies that can deliver meaningful benefit quickly. Importantly, persistency and adherence for Attruby continue to exceed our original expectations, which were based on historical ATTR treatment patterns, reinforcing our confidence in the durability of the franchise.
Attruby 也展現了迄今為止最快的分離速度,這是一個重要的特性,因為醫生們都在尋求能夠快速帶來顯著療效的療法。重要的是,Attruby 的持續性和依從性繼續超越我們最初的預期(基於 ATTR 的歷史治療模式),這增強了我們對該品牌持久性的信心。
We believe we have the strongest commercial teams in the industry, spanning sales, marketing, strategy, analytics, and market access. Many team members have worked together for years. We've continued to build on that foundation, with targeted hiring of top talent, including the recent expansion of the Attruby sales team.
我們相信我們擁有業內最強大的商業團隊,涵蓋銷售、行銷、策略、分析和市場准入等領域。許多團隊成員已經合作多年。我們在此基礎上繼續發展,有針對性地招募頂尖人才,包括最近擴充了 Attruby 銷售團隊。
Overall, Q4 reflects continued progress across key metrics, including growth in patients on therapy and ongoing use by prescribers. We are excited to see continued growth in quarter one, as we head into 2026.
總體而言,第四季度在關鍵指標方面持續取得進展,包括接受治療的患者人數成長和處方醫生持續使用。我們很高興看到2026年第一季持續保持成長動能。
Turning to the pipeline, we are focused on the next wave of potential launches. We are excited by the recent clinical results for BBP-418, Encaleret. and Infigratinib, all of which exceeded expectations across their primary and secondary end points.
展望產品線,我們正專注於下一波潛在的產品發布。我們對 BBP-418、Encaleret 和 Infigratinib 最近的臨床結果感到非常興奮,它們在主要和次要終點方面都超出了預期。
Based on the strength of these data, we believe each program will be the leader in its respective market and bring much needed therapies to families and patients in need of care.
基於這些數據的可靠性,我們相信每個項目都將成為各自市場的領導者,並為需要照護的家庭和患者帶來急需的治療。
Building on the successful launch of Attruby, we have established a proven commercial foundation and are well positioned to extend this model, as we prepare for future launches across our pipeline. We look forward to going into more detail, as we get closer to approval for each.
憑藉 Attruby 的成功上市,我們已經建立了可靠的商業基礎,並且有能力擴展這一模式,為未來產品線的上市做好準備。隨著各項審批工作的推進,我們期待能提供更多細節。
I will now turn the call over to Tom.
現在我將把通話轉給湯姆。
Thomas Trimarchi - President, Chief Operating Officer
Thomas Trimarchi - President, Chief Operating Officer
Thank you, Matt. Good afternoon, everyone.
謝謝你,馬特。大家下午好。
I'll now discuss our financial results for the fourth-quarter and full-year 2025. Please note that our commentary on today's call will focus on GAAP financials, unless otherwise indicated.
接下來我將討論我們2025年第四季和全年的財務表現。請注意,除非另有說明,我們今天電話會議的評論將側重於GAAP財務數據。
Total revenues were $154.2 million in 4Q 2025, consisting of $146 million of Attruby net product revenue, $5.3 million of royalty revenue, and $2.9 million of license and service revenue compared to total revenues of $5.9 million for the same period last year.
2025 年第四季總收入為 1.542 億美元,其中包括 Attruby 淨產品收入 1.46 億美元、特許權使用費收入 530 萬美元以及許可證和服務收入 290 萬美元,而去年同期總收入為 590 萬美元。
The $148.3 million increase in total revenues was primarily driven by a $143.1 million increase in net product revenue from Attruby, reflecting broad-based growth across market segments, including accelerating first-line adoption, increasing new patient starts, expanding prescriber depth, and strong persistency and adherence, supporting durable revenue growth.
總收入增長 1.483 億美元,主要得益於 Attruby 產品淨收入增長 1.431 億美元,這反映了各細分市場的全面增長,包括加速一線藥物的採用、增加新患者用藥、擴大處方醫生覆蓋範圍以及強大的持續性和依從性,從而支持了收入的持續增長。
We also recorded an increase of $5.1 million in royalty revenue from ex-US net sales of Beyonttra in Europe and Japan.
我們還記錄到,來自歐洲和日本的 Beyonttra 在美國以外的淨銷售額的版稅收入增加了 510 萬美元。
For the full-year 2025, total revenues were $502.1 million compared to $221.9 million for the full-year 2024. The $280.2 million increase in total revenues for the full year was primarily due to $359.5 million increase in net product revenue from Attruby and an $11.2 million increase in royalty revenue from sales of Beyonttra, partially offset by a $90.5 million decrease in license and service revenues versus the prior year.
2025 年全年總收入為 5.021 億美元,而 2024 年全年總收入為 2.219 億美元。全年總收入增加 2.802 億美元,主要歸功於 Attruby 產品淨收入增加 3.595 億美元,以及 Beyonttra 銷售特許權使用費收入增加 1120 萬美元,但部分被許可和服務收入較上年減少 9050 萬美元所抵消。
Total operating costs and expenses for the fourth quarter of 2025 were $293.7 million compared to $231.9 million in the same period in the prior year. The $61.8 million increase in operating costs and expenses was primarily driven by a $63.3 million increase in SG&A expenses, partially offset by a $13.9 million decrease in R&D expenses, primarily due to decreased R&D activities related to Attruby and Beyonttra, following regulatory approval.
2025 年第四季的總營運成本和費用為 2.937 億美元,而去年同期為 2.319 億美元。營運成本和費用增加 6,180 萬美元,主要原因是銷售、一般及行政費用增加 6,330 萬美元,部分被研發費用減少 1,390 萬美元所抵消,這主要是由於在獲得監管部門批准後,與 Attruby 和 Beyonttra 相關的研發活動減少所致。
For the full-year 2025, total operating costs and expenses were $1 billion compared to $814.9 million in the prior year. The $210.6 million increase was primarily driven by a $242.3 million increase in SG&A, largely reflecting the company's investments to support the commercial launch and ongoing activities for Attruby. This increase was partially offset by a $54.9 million decrease in R&D expenses, primarily due to decreased R&D activities related to Attruby and Beyonttra, following regulatory approval.
2025 年全年營運成本和費用總計為 10 億美元,而前一年為 8.149 億美元。2.106 億美元的成長主要由銷售、一般及行政費用 (SG&A) 增加 2.423 億美元所致,這主要反映了公司為支持 Attruby 的商業發布和持續活動而進行的投資。研發支出減少了 5,490 萬美元,部分抵消了這一增長,這主要是由於在獲得監管部門批准後,與 Attruby 和 Beyonttra 相關的研發活動減少所致。
Turning to our balance sheet, we ended the year with a cash position of $587.5 million in cash, cash equivalents, and marketable securities. We completed the issuance of $632.5 million, aggregate principal amount, of 2033 convertible notes in January 2026, which provides a significant cash runway to continue supporting our transition into a diversified, late-stage multi-product business.
從資產負債表來看,到年底,我們持有現金、現金等價物和有價證券共 5.875 億美元。我們在 2026 年 1 月完成了 6.325 億美元(總本金金額)的 2033 年到期可轉換票據的發行,這為我們繼續向多元化的後期多產品業務轉型提供了充足的現金支持。
With that, I'll turn the call back over to Chinmay.
這樣,我就把電話轉回欽邁了。
Chinmay Shukla - Senior Vice President - Strategic Finance
Chinmay Shukla - Senior Vice President - Strategic Finance
Thank you, Neil, Matt, and Tom.
謝謝尼爾、馬特和湯姆。
We will now turn the call over to the operator, who will open the line for questions.
現在我們將把電話轉接給接線員,由他/她接聽提問。
Thank you.
謝謝。
Operator
Operator
(Operator Instructions)
(操作說明)
Salim Syed , Mizuho.
Salim Syed,瑞穗銀行。
Unidentified Participant
Unidentified Participant
This is [Bennett], for Salim. Congrats on another quarter of continued patient growth.
這是[Bennett],代表Salim。恭喜你們又一個季度患者數量持續成長。
If I may, could you comment on why Attruby continues to show consistent growth, even as competitors growth seems to be slowing down? Can you comment on what are the key drivers behind it? What is the feedback that you feel is resonating more with (inaudible) and patients now that we are several quarters in?
如果可以的話,您能否解釋為什麼 Attruby 能夠持續保持成長,而競爭對手的成長似乎正在放緩?您能否談談背後的主要驅動因素是什麼?現在已經過去幾季了,您覺得哪些回饋意見更能引起(聽不清楚)和病患的共鳴?
Neil Kumar - Chief Executive Officer, Director
Neil Kumar - Chief Executive Officer, Director
Yeah. Thanks, Bennett. Maybe I'll turn it to Matt to answer that question.
是的。謝謝你,貝內特。或許我應該讓馬特來回答這個問題。
Matt Outten - Chief Commercial Officer
Matt Outten - Chief Commercial Officer
Sure. Thanks.
當然。謝謝。
I think it's multifaceted. But a big part is the field team that we have at BridgeBio. The right team makes or breaks a launch. That's across both commercial and medical.
我認為這個問題是多方面的。但很大一部分原因在於我們在 BridgeBio 擁有的現場團隊。合適的團隊決定產品發布的成敗。這種情況在商業和醫療領域都存在。
And then, of course, there's the data. No one's been able to show better data or near-complete stabilization, only Attruby.
當然,還有數據。只有阿特魯比能夠拿出更好的數據或接近完全的穩定性。
I think the time to separation is a big factor. You heard some of that in the earlier comments.
我認為分離時間是重要因素。你在之前的評論中已經聽到了一些類似的說法。
I think, finally, we've stayed disciplined and focused on what's important for patients and HCPs. We have category-leading efficacy and safety, with consistent results across all patient types.
我認為,最終我們還是保持了自律,專注於對病人和醫護人員來說重要的事情。我們的產品在療效和安全性方面均處於行業領先地位,並且在所有類型的患者中均取得了一致的療效。
A great team and a great medicine, I think, is hard to slow down.
我認為,優秀的團隊和優秀的藥物很難阻擋前進的步伐。
Neil Kumar - Chief Executive Officer, Director
Neil Kumar - Chief Executive Officer, Director
Maybe I'll just build on that.
或許我會以此為基礎繼續發展。
You can see in the new patient script numbers something interesting, where we had that rapid acceleration at first -- plateaued; and, now, we have a second wave of acceleration. That's rare, if you look and model most launches, where you see a burst in activity; and then, typically, you see a slowing.
從新患者處方數量中可以看出一些有趣的事情,起初我們經歷了快速增長——然後趨於平穩;而現在,我們迎來了第二波增長。這種情況很少見,如果你觀察和模擬大多數產品發布,你會發現發布活動會突然激增;然後,通常情況下,你會看到活動放緩。
And so that really portends one or two things:
所以,這其實預示著一兩件事:
One is, obviously, rapid patient identification, which I think we are seeing in the field.
顯而易見,其一是快速識別患者,我認為我們在實際工作中已經看到了這一點。
Secondly, it's really a second wave of prescribers that are starting to wake up to some of the messages that we're putting forward.
其次,實際上是第二批處方醫生開始意識到我們提出的一些資訊。
That, I think, is an exciting profile, generally, for a launch this early in. Couple that with nearly 1,200 new scripts since I gave my JP M talk, that's a very, very exciting trajectory, right now.
我認為,對於一個如此早期的產品來說,這總體來說是一個令人興奮的前景。再加上自從我做了 JP M 演講以來新增的近 1200 個劇本,這真是一個非常非常令人興奮的發展軌跡。
Unidentified Participant
Unidentified Participant
All right. Thank you.
好的。謝謝。
Operator
Operator
Mani Foroohar, Leerink Partners.
Mani Foroohar,Leerink Partners。
Mani Foroohar, M.D. - Analyst
Mani Foroohar, M.D. - Analyst
Congrats on continuing to show volume growth in the face of competitors who are seeing slowdowns.
恭喜你們在競爭對手銷量放緩的情況下,依然維持了銷量成長。
You've talked a lot about the commercial differentiation and differences in your growth trajectory versus competitors; lots of different pieces of data go in to that.
您多次談到了您的商業差異化以及您與競爭對手在成長軌跡上的差異;這其中涉及許多不同的數據。
But I want to look past out into just what the timeline is into, whenever tafamidis gets generic and beyond, about clinical differentiation, which you've identified as core to your strategy to driving continued growth and durability in the face of a generic, whenever that happens.
但我希望展望未來,看看 tafamidis 何時成為仿製藥以及之後的發展,特別是臨床差異化,您已經將其確定為在仿製藥出現時推動持續增長和持久發展的核心戰略。
Can you tell us when we'll have significant incremental real-world data, longer-term data, for acoramidis to establish that difference in clinical benefit that you guys are hanging that growth tail on?
能否告知我們何時才能獲得關於acoramidis的實質增量真實世界數據、長期數據,以證實你們所依賴的臨床獲益差異,從而推動其成長?
Neil Kumar - Chief Executive Officer, Director
Neil Kumar - Chief Executive Officer, Director
Yeah. Great question, Mani. Thanks for it.
是的。問得好,馬尼。謝謝。
I think, first, the key is for us to really start to get some of the data that we presented in the last year out into the field and understood; maybe just a couple of pieces that I think have been overlooked or are just starting to really make their way into the field.
我認為,首先,關鍵在於我們要真正開始將去年提出的一些數據應用到實際應用中,並加以理解;也許只是一些我認為被忽視或剛開始真正進入實際應用領域的部分。
First and foremost is the early impact: that impact as early as one month. I talked about, at JP M, we continue to interrogate the precise mechanism behind it.
首先也是最重要的是早期影響:這種影響最早在一個月內就能顯現出來。我曾說過,在摩根大通,我們一直在探究背後的確切機制。
But as you know from these clinical trials and a lot of the real-world evidence, early [CVH] is extremely common. You want to get patients on the drug that can take action as early as possible, not only for that reason but, obviously, this is an ongoing (inaudible) and deleterious disease so you want to be on the drug that has the earliest impact.
但正如你從這些臨床試驗和許多真實世界的證據中了解到的那樣,早期[CVH]非常常見。你想讓患者儘早服用能發揮作用的藥物,不僅因為這個原因,而且顯然,這是一種持續的(聽不清楚)且有害的疾病,所以你想讓患者服用能最早起效的藥物。
The second is the [AF] data that we put forth. Nearly 60% of patients in this space suffer from AF or cardiac arrhythmic events I think the most important piece of the data that we put forth when we showed the 70% reduction, as published last year, is that we are having an equivalent effect on or off the AF sub-population.
第二點是我們提出的[AF]數據。在這個領域,近 60% 的患者患有心房顫動或心律不整事件。我認為,我們在去年公佈的 70% 減少率數據中最重要的一點是,無論是否患有房顫,我們都產生了相同的效果。
And so when you think about AF patients being slightly harder to manage in the context of ATTR cardiomyopathy, here, you have a drug that has consistent and high impact; in fact, the highest point estimate we've seen, in terms of both reduction in downstream outcomes of 43% and reduction in AF itself of 17%.
因此,考慮到 ATTR 心肌病變背景下的房顫患者管理起來稍微困難一些,這裡有一種藥物具有持續且顯著的效果;事實上,就下游結果減少 43% 和房顫本身減少 17% 而言,這是我們見過的最高點估計值。
That, I think, is the second piece that we need to do a better job of educating on. I think physicians will find it exciting.
我認為,這是我們需要加強教育的第二個面向。我認為醫生們會覺得這很令人興奮。
And then, finally, it's the variant population, right; the sickest, by far, of the subpopulations. They do deserve a better drug in that [0.41] hazard ratio that we presented on with statistical significance, even more impressive given the fact that less than 10% of our patients on Attruby were variant patients.
最後,還有變異人群,對吧;就所有亞人群而言,他們的病情最為嚴重。他們確實應該得到更好的藥物,因為我們以統計意義展示了[0.41]的風險比,考慮到我們接受 Attruby 治療的患者中只有不到 10% 是變異患者,這更令人印象深刻。
I think that is the best point estimate, again, with the best statistical significance in the space; and extremely consistent with the binding mode that we've articulated, which is differentiated against tafamidis.
我認為這是最佳點估計,再次強調,這是該空間中統計顯著性最高的;並且與我們闡述的結合模式非常一致,這與 tafamidis 不同。
So those are, I think, the things that we need to do a better job of driving into the marketplace, right now.
所以,我認為,這些就是我們現在需要更好地推向市場的事情。
On a go-forward basis, the two big areas that we're interrogating, number 1 are real-world evidence, what you should see by the end of this year -- this calendar year; and the second is the cardiorenal access work that we're doing, where we think we have a unique signal that connects interestingly to the early onset of activity and could really, I think, change the shape of this marketplace, going forward.
展望未來,我們正在研究的兩個主要領域是:第一,現實世界的證據,也就是到今年年底(本日曆年)您應該看到的結果;第二,我們正在進行的心腎通路研究,我們認為我們有一個獨特的信號,它與早期活動密切相關,而且我認為,它真的可以改變未來市場的格局。
That's what I'd say on that front.
這就是我在這方面的看法。
Mani Foroohar, M.D. - Analyst
Mani Foroohar, M.D. - Analyst
I have a quick follow-up; more on firm-wide strategy.
我還有一個後續問題;更多關於公司整體策略的內容。
You guys have talked about the transition towards cash flow generation over the course of a couple of years. Obviously, that happens when you have multiple high-margin small molecule assets.
你們在過去的幾年裡一直在討論向現金流生成模式的轉型。顯然,當你擁有多個高利潤的小分子資產時,就會發生這種情況。
Can you talk about how you guys think, strategically, longer term about use of cash; and where and how to put that incremental free cash flow to work?
你們能否談談從策略和長遠角度考慮現金的使用,以及如何將新增的自由現金流投入使用?
I'm not saying call for a dividend buyback, et cetera, [BD]; but how you guys think about your strategy and where that capital should go in a '28, '29, '30, et cetera, free cash flow-generating BridgeBio?
我不是說要呼籲進行股息回購等等,[BD];但是你們是如何看待你們的策略,以及在2028年、2029年、2030年等等,在能夠產生自由現金流的BridgeBio中,這些資金應該投入到哪裡?
Neil Kumar - Chief Executive Officer, Director
Neil Kumar - Chief Executive Officer, Director
Yeah. Totally.
是的。完全。
Well, I would say, at the very highest level and as we've been talking about, I think, a little bit more over the last couple of months, we're very pleased with the efficiency of our R&D engine.
嗯,我想說,從最高層面來說,正如我們在過去幾個月裡一直在討論的那樣,我們對研發引擎的效率非常滿意。
Efficiency, both in terms of time and cost; and, obviously, the validity of it, as it pertains to, probably, a technical success.
效率,包括時間和成本方面的效率;以及,顯然,其有效性,因為它可能與技術上的成功有關。
As we talked a little bit about a few months ago -- Mani, I know you and I have connected on this -- the pipeline that is attended at Gondola is a wonderful example of the ample substrate available to help patients with genetic disease.
正如我們幾個月前稍微談到的那樣——馬尼,我知道你我在這方面有共同點——貢多拉的管道就是一個很好的例子,它提供了充足的基質來幫助患有遺傳疾病的患者。
Our objective function is to serve as broadly as possible. Given all of those things, with cash flow, we would intend, as long as we can beat our cost of capital, to continue to reinvest into R&D; and, in some cases, bring in partially owned assets that we have access to through Gondola and bring those forward into the, again, highly efficient operating model that we've established in mid- to late-stage development and, ultimately, in the commercial setting.
我們的目標是盡可能廣泛地服務大眾。考慮到所有這些因素,只要我們能夠控制資本成本,我們就會利用現金流繼續投資研發;並且在某些情況下,我們會將透過 Gondola 獲得的、部分擁有的資產引入到我們在中後期開發階段以及最終在商業環境中建立的高效營運模式中。
Obviously, the stock is not trading where we would like it to trade. It's trading quite a ways off intrinsic value. There are opportunities to do other things with cash flows; namely, share buybacks and dividends, if indeed. We don't feel we can capture the [NPV] of fully financed assets, as they move into the marketplace.
顯然,這隻股票目前的交易價格並沒有達到我們預期的水平。它目前的交易價格與內在價值相差甚遠。現金流還有機會用於其他用途,例如股票回購和分紅(如果確實需要的話)。我們認為,隨著完全融資的資產進入市場,我們無法獲得它們的淨現值。
A bit of this will be just to see whether or not we can do a better job of helping investors avail of the value that we create; and getting the stock price and cost of capital back up into a normal realm. And then., that, I hope, would get us going, in terms of growth in the R&D sector.
這樣做的一部分目的是為了看看我們能否更好地幫助投資者獲得我們創造的價值;並將股價和資本成本恢復到正常水平。然後,我希望這能推動研發領域的成長。
Does that answer your question?
這樣回答了你的問題嗎?
Mani Foroohar, M.D. - Analyst
Mani Foroohar, M.D. - Analyst
Absolutely. Congrats, again, a good quarter.
絕對地。再次恭喜,這個季度業績不錯。
Operator
Operator
Tyler Van Buren, TD Cowen.
泰勒·範·布倫,TD·考恩。
Tyler Van Buren - Analyst
Tyler Van Buren - Analyst
Following the three successful Phase 3s in recent months, can you elaborate on your launch readiness and expected field footprint in the context of your burn commentary and the expected cadence of regulatory and commercial catalysts over the next 12 to 18 months?
繼近幾個月成功完成三期臨床試驗之後,您能否結合先前的燃燒情況評論以及未來 12 至 18 個月內監管和商業催化劑的預期節奏,詳細說明您的上市準備情況和預期現場規模?
Neil Kumar - Chief Executive Officer, Director
Neil Kumar - Chief Executive Officer, Director
Yeah. Thanks, Tyler. Maybe I'll ask Matt and Tom to comment on that.
是的。謝謝你,泰勒。或許我會請馬特和湯姆對此發表意見。
Matt Outten - Chief Commercial Officer
Matt Outten - Chief Commercial Officer
Sure. Tyler, I think we're going to follow the same rigor as what we did for the Attruby launch.
當然。泰勒,我認為我們將遵循與 Attruby 發佈時同樣的嚴謹流程。
I think one of the big differences is, this time, we'll be launching on a global basis. As a part of that, we're building in the US but also ex-US, as well.
我認為最大的不同之處在於,這次我們將面向全球推出。為此,我們不僅在美建設,也在美外建設。
We'll have more on that towards the end of the year, in terms of both additional revenue for Attruby that will be coming Rest of World but, also, our prep and build-out for the three additional launches in the US and the rest of the geographies, as well.
今年年底,我們將公佈更多相關信息,包括 Attruby 在世界其他地區即將獲得的額外收入,以及我們為在美國和其他地區推出的三個新項目所做的準備和建設工作。
I think what's important -- you've seen the recent data read-outs. We're setting or resetting the standard of care for each and every one.
我認為重要的是——你已經看到了最近的數據讀數。我們正在為每一位患者制定或重新定義護理標準。
For LGMD2I and ADH1, it's going to be a first and best-in-class story. For [ACON], it's going to be resetting the standard with best-in-class data.
對於 LGMD2I 和 ADH1 來說,這將是一個開創先河、同類最佳的故事。對於 [ACON] 而言,它將以一流的數據重新定義標準。
Thomas Trimarchi - President, Chief Operating Officer
Thomas Trimarchi - President, Chief Operating Officer
I'll take the question on burn.
我會回答這個問題。
As we've discussed, we've seen, over the last several quarters, our cash burn has been on a downward trajectory. That's driven, first and foremost, by the strong ramp of Attruby and gross profit that it provides.
正如我們之前討論過的,在過去的幾個季度裡,我們的現金消耗呈下降趨勢。這首先是由 Attruby 的強勁成長及其帶來的毛利所驅動的。
But I will also say that it's been due to our disciplined OpEx profile here.
但我還要說,這也要歸功於我們嚴格的營運支出管理模式。
As we look to ramp the next three launches, we do expect a gradual increase in OpEx throughout the year. However, we expect burn to hold steady throughout most of the year and drop off again towards the end of the year, as we continue to see an expanding operating margin provided by the Attruby brand.
隨著我們推進接下來的三次產品發布,我們預計全年營運支出將逐步增加。不過,我們預計今年大部分時間燒錢情況將保持穩定,並在年底前再次下降,因為我們繼續看到 Attruby 品牌帶來的營業利潤率不斷擴大。
Thanks for the question, Tyler.
謝謝你的提問,泰勒。
Operator
Operator
Biren Amin, Piper Sandler.
比倫·阿明,派珀·桑德勒。
Biren Amin - Analyst
Biren Amin - Analyst
Congrats on the quarter. I have a high-level question.
恭喜你本季取得佳績。我有一個比較高層次的問題。
As you've demonstrated impressive productivity and outlined BridgeBio way as a sustainable development model, which was highlighted in the Drug Discovery Today manuscript, recently in January.
正如您展現出的令人印象深刻的生產力,以及將 BridgeBio 的發展模式概述為永續發展模式一樣,這一點在最近一月份的 Drug Discovery Today 手稿中得到了重點介紹。
With that in mind and as we look beyond 2025, what are the key drivers of momentum for the company? When should investors expect new assets to enter the pipeline?
考慮到這一點,展望 2025 年以後,推動公司發展的關鍵因素是什麼?投資人應該何時才能看到新的資產項目進入開發階段?
Neil Kumar - Chief Executive Officer, Director
Neil Kumar - Chief Executive Officer, Director
Yeah. Thanks, Biren. Thanks for the question.
是的。謝謝你,比倫。謝謝你的提問。
A little bit overlapping with some of my comments that I gave against Mani's question, as well.
這和我之前針對 Mani 的問題所做的一些評論略有重疊。
But maybe I'll just say, like, near term, our focus continues to be, obviously, making sure that these drug products that we just registered successful Phase 3s on are approved and, ultimately, launched correctly.That's the highest and best use of our time, right now.
但或許我可以簡單地說,就近期而言,我們的重點顯然仍然是確保我們剛剛成功完成三期臨床試驗的這些藥物產品獲得批准,並最終順利上市。這才是我們目前最該做的事。
The second best use of our time are the additional indications associated with medicines that we know are safe and effective, such as, obviously, chronic hypopara in the context of Encaleret; and hypochon and some of the other height disorders that Justin has talked about in the past associated with Infigratinib. That would be the second category of growth.
我們時間的第二佳用途是研究已知安全有效的藥物的其他適應症,例如,顯然,Encaleret 可用於治療慢性下肢發育不全;而 Infigratinib 可用於治療矮小症和 Justin 過去談到的其他一些身高障礙。那將是第二類成長。
But I think you're asking the right question. Look, at the end of the day, as I mentioned earlier, the scientific substrate available to us to target well-described genetic conditions at their source continues to grow. We're finding starting points, all the way from the clinic back to early-stage discovery, where we're probably most adept.
但我認為你問對了問題。歸根結底,正如我之前提到的,我們可用於從根源上治療已明確描述的遺傳疾病的科學依據正在不斷增加。我們正在尋找切入點,從臨床到早期發現階段,而我們可能最擅長的就是早期發現階段。
That's highlighted in the ever-growing pipeline at Gondola, which obviously BridgeBio shareholders partially own.
這一點在 Gondola 不斷增長的管道中體現得尤為明顯,而 BridgeBio 的股東顯然持有該公司的部分股份。
I would think, over the course of time, if indeed we're able to correct our cost of capital and trade closer to intrinsic value. Number one.
我認為,隨著時間的推移,如果我們能夠修正資本成本,並更接近內在價值進行交易,情況就會有所改善。第一。
Number two. (inaudible) We're able to really stick the landing and effectively get these drugs approved and launched, there will be a moment where we can bring some of those other assets in and prosecute them with the great infrastructure that we've already set up here; namely, mid- and late-stage development, regulatory, the ability to put it in the hands of a great commercial team, and to do all of that efficiently., in terms of time and cost.
第二。(聽不清楚)如果我們能夠真正成功落地,有效地獲得這些藥物的批准並上市,那麼在某個時刻,我們就可以引入其他一些資產,並利用我們已經建立的強大基礎設施來推進它們;也就是說,中後期開發、監管、將其交給優秀的商業團隊,並在時間和成本方面高效地完成所有這些工作。
That's the high-level answer. I don't have anything specific on a specific asset that we would bring in anything like that. I do think you should look for us, generally, to rely on organic, not inorganic, growth; organic -- meaning, from the ecosystem of BridgeBio activities and BridgeBio companies and not looking for big M&A or anything like that.
這是概括性的回答。我目前沒有任何關於我們會引進某項具體資產的具體資訊。我認為,總的來說,你們應該期待我們依靠有機增長而不是無機增長;有機增長指的是來自 BridgeBio 活動和 BridgeBio 公司的生態系統,而不是尋求大型併購或其他類似的東西。
We tend to look at that as a rather expensive mode of growth; one that we probably don't need to take on, given the fact that we're getting to INDs in less than $10 million or $15 million and through Phase 1/2s in less than $100 million.
我們傾向於認為這是一種相當昂貴的成長模式;鑑於我們只需不到 1000 萬或 1500 萬美元就能完成 IND 申請,只需不到 1 億美元就能完成 1/2 期臨床試驗,我們可能不需要採用這種模式。
Unless we run out ideas, internally, I don't think we would be aggressively moving toward M&A for growth in the next three to five years.
除非我們內部沒有其他辦法,否則我認為在未來三到五年內,我們不會積極地透過併購來成長。
Does that answer your question, Biren?
比倫,這樣回答你的問題了嗎?
Biren Amin - Analyst
Biren Amin - Analyst
Yeah. (inaudible)
是的。(聽不清楚)
Operator
Operator
Corey Kasimov, Evercore ISI.
Corey Kasimov,Evercore ISI。
Unidentified Participant
Unidentified Participant
This is [Adi], on for Cory.
這是阿迪,他代替科里上場。
I wanted to ask on Infigratinib. Now with the Phase 3 data in hand, how are you thinking about the competitive landscape across not just CNP pathway therapies but also other FGFR-targeted programs, which are more specific to FGFR3?
我想問一下關於Infigratinib的問題。現在有了 3 期數據,您如何看待 CNP 通路療法以及其他 FGFR 標靶項目(特別是針對 FGFR3 的項目)的競爭格局?
Neil Kumar - Chief Executive Officer, Director
Neil Kumar - Chief Executive Officer, Director
Thanks for the question. Yeah. Justin, do you want to take that?
謝謝你的提問。是的。賈斯汀,你想拿嗎?
Jusin To - Chief Executive Officer
Jusin To - Chief Executive Officer
Again, thanks for the question.
再次感謝你的提問。
Really, we believe the balance of efficacy and safety shown in PROPEL 3 proved that Infigratinib is not just best-in-class but potentially last in-class in achondroplasia.
事實上,我們相信 PROPEL 3 中顯示的療效和安全性的平衡證明,Infigratinib 不僅是同類最佳,而且可能是軟骨發育不全的同類最後選擇。
On the efficacy side, we had a plus [2.1] centimeter per year change from baseline AHV. The first and only stat sig improvement proportionality. Most importantly, we normalized absolute AHV, bringing back kids with achondroplasia to wild-type growth levels of 6 centimeters per year. Across every single measure of efficacy, whether it be in [animal] models or in the clinic, we have set a new bar here.
在療效方面,我們比基線 AHV 每年增加了 [2.1] 公分。第一個也是唯一一個統計顯著性改善比例。最重要的是,我們使絕對 AHV 正常化,使軟骨發育不全的兒童恢復到每年 6 公分的野生型生長水平。無論是在動物模型或臨床上,我們衡量療效的每項指標都樹立了新的標竿。
On the safety data side, we had a home run outcome, right, with basically no change in mean phosphate levels between the placebo and treatment arms; and no signs of FGFR1 or 2 associated toxicity.
在安全性數據方面,我們取得了非常成功的結果,安慰劑組和治療組之間的平均磷酸鹽水平基本上沒有變化;也沒有出現與 FGFR1 或 2 相關的毒性跡象。
Really, I think the other molecules being developed in this space, whether it be CNPs or FGFR3 inhibitors, have two issues:
實際上,我認為在這個領域正在開發的其他分子,無論是 CNP 還是 FGFR3 抑制劑,都存在兩個問題:
One, on the efficacy side, you actually don't want to overshoot 6 centimeters per year too much. We've heard from clinicians that the skeletons and bones in achondroplasia aren't built for too much growth, given preexisting low-bone mineral density. We really hit the sweet spot there.
首先,從效果方面來說,你其實不想每年增長超過 6 公分太多。我們從臨床醫生那裡了解到,軟骨發育不全患者的骨骼和骨頭並不適合過度生長,因為他們本身骨礦物質密度就很低。我們真的找到了最佳切入點。
On the safety side, we obviously avoid all the well-known issues associated with the CNP class, such as on vasodilation.
在安全性方面,我們顯然避免了與 CNP 類藥物相關的所有眾所周知的問題,例如血管舒張。
Now, the other FGFR3 inhibitors in development trade off selectivity for FGFR1 and 2 for significant VEGFR3 liabilities. There's two issues related to that. They're not just theoretical risk.
目前,其他正在研發的 FGFR3 抑制劑以對 FGFR1 和 2 的選擇性為代價,換取了對 VEGFR3 的顯著負面影響。這涉及到兩個問題。它們不僅僅是理論上的風險。
The first is on spermatogenesis. Because of this, enrollment in trials are restricted to pre-pubertal males for these other programs.
第一部分是關於精子發生的。因此,其他這些項目的試驗參與者僅限於青春期前的男性。
The second and potentially even more of a [looked] issue is the effect on angiogenesis. Many molecules that have in vitro potency findings for VEGFR3, even without clinical findings, end up with a box-warning on their labels for impaired wound healing. A great example of this is Retevmo.
第二個問題,也是可能更受關注的問題,是對血管新生的影響。許多在體外對 VEGFR3 具有效力的分子,即使沒有臨床研究結果,最終也會在標籤上被加上「傷口癒合受損」的警告。Retevmo 就是一個很好的例子。
So, net-net, we're really happy with where we've landed on data. Our safety profile obviates the need for other FGFR3 inhibitors.
總而言之,我們對最終得到的數據非常滿意。我們的安全性表明無需其他 FGFR3 抑制劑。
We absolutely could go further in dose, given our safety data; but really think that there's no need to in achondroplasia, given that we have gone back to wild-type levels of growth.
鑑於我們的安全數據,我們絕對可以進一步增加劑量;但考慮到我們已經恢復到野生型生長水平,我們認為在軟骨發育不全的情況下沒有必要這樣做。
I hope that answers your question.
希望我的回答能解答你的疑問。
Operator
Operator
Eliana Merle, Barclays.
埃莉安娜·默爾,巴克萊銀行。
Eliana Merle - Equity Analyst
Eliana Merle - Equity Analyst
Thanks for the color, so far. But if you could go over your views in a little bit more depth on the taf IP and some color there; and, specifically your base case for when tafamidis goes generic in the US, how you're thinking about it? Can you elaborate on why this doesn't matter for Attruby, in your view?
感謝你目前為止帶來的色彩。但是,如果您能更深入地闡述一下您對 taf IP 的看法,並提供一些細節資訊;特別是,當 tafamidis 在美國成為通用名藥物時,您是如何考慮的基本情況的?您能否詳細解釋一下,為什麼這對阿特魯比來說無關緊要?
And then, I have a follow-up question.
然後,我還有一個後續問題。
Neil Kumar - Chief Executive Officer, Director
Neil Kumar - Chief Executive Officer, Director
Ellie, thanks for the question. Yeah.
艾莉,謝謝你的提問。是的。
Broadly, we tend not to comment on the IP situation for our competitors. But, obviously, it's been a big story for the stock here.
一般來說,我們傾向於不對競爭對手的智慧財產權狀況發表評論。但很顯然,這對這支股票來說是個大新聞。
Let me turn it over to Chinmay to take a crack. I'm happy to elaborate on it.
讓我把題目交給欽邁來試試。我很樂意詳細闡述。
Chinmay Shukla - Senior Vice President - Strategic Finance
Chinmay Shukla - Senior Vice President - Strategic Finance
Yeah. Happy to take that. Ellie. Thanks for the question.
是的。樂意接受。艾莉。謝謝你的提問。
Maybe I'll talk about it in two ways. As Neil mentioned, we try not to talk about our competitors' IP, especially when the competitor is, we believe, not as potent as our molecule.
或許我會從兩個方面來談談這件事。正如尼爾所提到的,我們盡量不談論競爭對手的智慧財產權,尤其是當我們認為競爭對手的分子不如我們的分子有效時。
But I think let's talk a little bit about what we think will happen on the trial and maybe a little bit on our strategy for why this doesn't really matter.
但我認為我們應該稍微談談我們認為審判會發生什麼,以及為什麼這件事其實並不重要。
I think maybe I'll start quickly on Europe since I know you had some questions there.
我想我或許會先快速講講歐洲,因為我知道你對歐洲那部分有一些疑問。
I think it's important to note that Pfizer withdrew its patent there. And so that's going to limit the precedential value of this ruling for related parties and other jurisdictions.
我認為值得注意的是,輝瑞公司已經撤回了該項專利。因此,這將限制該裁決對相關方和其他司法管轄區的先例價值。
I think, as Neil highlighted in his prepared remarks, our base case for Europe has always been entry -- generic entry -- in 2030. That's based on (inaudible) for wild-type ATTR-CM. That's still our assumption.
我認為,正如尼爾在事先準備好的演講稿中所強調的那樣,我們對歐洲的基本設想一直是 2030 年進入——一般意義上的進入。這是基於(聽不清楚)野生型 ATTR-CM。這仍然是我們的假設。
There are two more patents in Europe, which protect Pfizer. There may be some potential upside there.
輝瑞公司在歐洲還有兩項專利受到保護。這或許存在一些潛在的收益空間。
It's also important to note on that front the really strong treatment-naïve share that Bayer has been achieving, which talks a little bit about how physicians, not just in the US but globally, are recognizing the differentiation of acoramidis.
值得注意的是,拜耳在未接受過治療的患者群體中取得了非常強勁的市場份額,這在一定程度上說明了醫生們,不僅在美國,而且在全球範圍內,都認識到了阿可拉米迪斯的差異化優勢。
Turning to the US, which I think is the market which probably matters a little more, I think Neil had a bunch of comments in his prepared remarks on how we think about it.
再來說說美國市場,我認為這可能更重要一些,尼爾在他的準備好的演講稿中對我們如何看待美國市場發表了一系列評論。
But, based on publicly available information, I think that a couple of things are interesting to note there, in addition to what Neil said:
但是,根據公開訊息,除了尼爾所說的之外,我認為還有幾點值得注意:
One is that Dexcel, which is, I think, the lead filer, has conceded infringement of the 441 polymorph patent. The other interesting thing to note is also that the bar for validity is much more innovator-friendly under US law.
一是 Dexcel(我認為是主要申請人)已經承認侵犯了 441 多晶型專利。另一點值得注意的是,美國法律對有效性的要求對創新者更加友善。
As Neil mentioned in his remarks, IP is always uncertain. We'll keep monitoring it and seeing what happens in Europe in April in the US trial. But I think that we feel good about where we are, right now. I think that we do feel like Vyndamax should have protection into the 2030s, potentially up to 2035.
正如尼爾在演講中提到的那樣,知識產權總是充滿不確定性。我們會繼續關注事態發展,看看4月歐洲和美國審判的情況如何。但我認為我們對目前的狀況感到滿意。我認為我們確實覺得 Vyndamax 應該獲得保護到 2030 年代,甚至可能到 2035 年。
I think it's important to note, though, that we feel like the tafamidis IP debate is a little bit of a side show. It doesn't really matter for Attruby's uptake.
不過,我認為值得注意的是,我們覺得 tafamidis 的智慧財產權之爭有點像是次要事件。這對阿特魯比的吸收率其實並不重要。
You guys heard today from Neil, Matt, and everyone else about the tremendous momentum, which we are seeing for Attruby. The patient weeks -- number of patients per week -- continues to increase, as we go forward in our launch and continues to accelerate. I think that's driven by the differentiated clinical data, which we have for Attruby.
今天你們都從尼爾、馬特和其他人那裡聽到了關於阿特魯比目前發展勢頭強勁的消息。隨著我們產品上市的推進,患者週數(每週患者人數)持續增加,並且持續加速。我認為這是由我們掌握的 Attruby 的差異化臨床數據所驅動的。
And so even in a scenario where a generic tafamidis enters the market, we just don't believe that a less efficacious product will displace a clinically superior therapy in a serious progressive disease.
因此,即使在仿製藥他法米地進入市場的情況下,我們也不認為療效較差的產品會取代在嚴重進行性疾病中臨床療效更優的療法。
We've seen this pattern, by the way, play out in multiple therapeutic areas such as [PAH], statins, prostate cancer, where differentiated second-to-market molecules continue to grow, even after the first-to-market, post-generic.
順便說一句,我們已經看到這種模式在多個治療領域上演,例如[PAH]、他汀類藥物、前列腺癌,即使在首個上市的仿製藥之後,差異化的第二個上市分子仍然繼續增長。
So we feel good about the long-term value of Attruby. I think that we look forward to continuing to execute against that.
因此,我們對 Attruby 的長期價值感到樂觀。我認為我們期待繼續朝著這個目標努力。
Eliana Merle - Equity Analyst
Eliana Merle - Equity Analyst
Great. Just a quick follow-up: How do you see the use of serum TTR in clinical practice in the real world evolving; and your perspective there in how that could potentially show differentiation for physicians?
偉大的。還有一個後續問題:您認為血清 TTR 在臨床實務中的實際應用將如何發展?您認為它如何幫助醫師進行鑑別診斷?
Neil Kumar - Chief Executive Officer, Director
Neil Kumar - Chief Executive Officer, Director
Great question, Ellie.
問得好,艾莉。
I'd say, first and foremost, you probably saw the recent two [JAK] papers that came out looking at serum TTR elevation and correlating it with downstream relative risk of mortality reduction. Both of those were associated with tafamidis.
首先,我想說的是,你可能已經看到了最近發表的兩篇 [JAK] 論文,這兩篇論文研究了血清 TTR 升高及其與下游死亡率降低的相對風險之間的相關性。這兩種疾病都與塔法米迪斯有關。
But they reiterated the point that our publication last year made, which is that ever-higher levels of serum TTR are associated with ever -lower levels of downstream mortality; that, roughly, you could imagine every 1 mg per deciliter increase leading to about a 5% relative risk reduction in downstream mortality.
但他們重申了我們去年發表的文章中提出的觀點,即血清 TTR 水平越高,下游死亡率就越低;粗略地說,你可以想像每分升增加 1 毫克,下游死亡率的相對風險就會降低約 5%。
That's exciting.
真令人興奮。
Obviously, the studies that Pfizer did had significantly higher levels of variant patients in them. You're going to see similar serum TTR rise, as you saw in our studies. But that's just basically rigged up so that you can see a higher increase because you've got many more variant patients.
顯然,輝瑞公司進行的研究中,變異患者的比例明顯較高。你會看到類似的血清 TTR 升高,就像你在我們的研究中看到的那樣。但這基本上是人為操縱的,目的是為了讓數據增幅更大,因為患者類型更加多樣化。
If you normalize for variant to wild-type patients, even in cross-trial studies or cross-study comparisons, you can see that we have a significantly higher serum TTR elevation.
如果將變異型患者與野生型患者進行標準化,即使在跨試驗研究或跨研究比較中,也可以看出我們的血清 TTR 升高明顯更高。
But I think that the most interesting data from that standpoint were literally the same patients going from tafamidis on to acoramidis in the context of our OLE in Attruby, where you saw, as I mentioned earlier, that 3 mgs per deciliter increase, when going from a partial stabilizer to a full stabilizer.
但我認為從這個角度來看,最有趣的數據實際上是同一組患者在我們 Attruby 的開放標籤擴展研究中從 tafamidis 轉為 acoramidis 的情況,正如我之前提到的,當從部分穩定劑轉為完全穩定劑時,每分升的劑量增加了 3 毫克。
I think now we can say -- and I remember, Ellie, having this debate with you a long time ago -- even, it is -- like, what is the shape of that response curve, in terms of ever-higher levels of stabilization leading to ever-better outcomes?
我認為現在我們可以說——而且我記得,艾莉,很久以前就和你討論過這個問題——甚至,就像,這種反應曲線的形狀是什麼,即越來越高的穩定水平會帶來越來越好的結果?
I think, here, at least, we can say it's roughly 5% per mgs per deciliter. It's about a 15% relative risk reduction, in terms of mortality going from taf to acoramidis, putting aside the earlier onset (inaudible) action and some of the other advantages.
我認為,至少在這裡,我們可以說大約是每毫克每分升 5%。從 taf 換成 acoramidis,死亡率的相對風險降低了約 15%,這還不包括更早起效(聽不清楚)以及其他一些優點。
I think serum TTR will become ever more important, based on these [dyspevia] publications. Let's see. It's not broadly used.
根據這些[dyspevia]出版物,我認為血清TTR將變得越來越重要。讓我們來看看。它並不常用。
I don't know, Matt, if you disagree, right now. But I think our hope is, on a go-forward basis, it will become an ever-more important marker of drug action and also therapeutic choice.
馬特,我不知道你現在是否同意。但我認為,我們希望,在未來的發展中,它將成為藥物作用和治療選擇越來越重要的標誌。
Unidentified Participant
Unidentified Participant
Great,thank. You for the color.
太好了,謝謝。你喜歡這個顏色。
Chinmay Shukla - Senior Vice President - Strategic Finance
Chinmay Shukla - Senior Vice President - Strategic Finance
Yeah. Thanks, Ellie, for the question.
是的。謝謝艾莉的提問。
Operator
Operator
Andrew Tsai, Jefferies.
Andrew Tsai,傑富瑞集團。
Andrew Tsai - Analyst
Andrew Tsai - Analyst
Just wanted to stick on the theme of cash burn and near-term profitability. What are your guys' expectations on priority review vouchers toward non-dilutive capital? I think they're going for $200 million to $300 million a piece, right now.
我只是想繼續討論現金消耗和近期獲利能力這個主題。你們對優先審查憑證作為非稀釋性資本有何預期?我認為他們現在的報價是每件2億到3億美元。
Which of your pipeline drugs or, even, which indication per drug could be eligible for PRVs? When do you expect to receive them?
你們的哪些在研藥物,或每種藥物的哪些適應症可能符合PRV的條件?預計何時收到?
Neil Kumar - Chief Executive Officer, Director
Neil Kumar - Chief Executive Officer, Director
Yeah. Good question. I didn't factor that into my earlier comments. But, Tom, do you want to take that?
是的。問得好。我之前的評論中沒有考慮到這一點。但是,湯姆,你想拿那個嗎?
Thomas Trimarchi - President, Chief Operating Officer
Thomas Trimarchi - President, Chief Operating Officer
Yeah. Sure. I'll take that.
是的。當然。我接受。
First, absolutely thrilled to see that program has been reauthorized. That's been a hugely successful incentive for BridgeBio and companies like us -- in being able to responsibly invest in diseases that affect very few patients. Otherwise, we would be at risk of being left behind. Really happy that that's been extended.
首先,我非常高興地看到該項目已獲得重新授權。對於 BridgeBio 和像我們這樣的公司來說,這是一個非常成功的激勵機制——讓我們能夠負責任地投資於影響極少數患者的疾病。否則,我們就有被時代拋在後面的風險。很高興這項政策得以延長。
We actually have three programs that have already received rare pediatric disease designation. We expect to be eligible to receive a PRV, upon approval.
我們實際上已經有三個項目獲得了罕見兒科疾病認定。我們預計獲得批准後將有資格領取PRV。
Those are 418 for limb-girdle; Infigratinib for achondroplasia; and then, our Canavan gene therapy program.
這些藥物包括用於治療肢帶型畸形的 418 號藥物;用於治療軟骨發育不全的 Infigratinib 藥物;以及我們的 Canavan 基因治療計畫。
As you rightly pointed out, the pricing of these has not only held in but risen over the last few months. So there's significant asset value there already within our portfolio.
正如您所指出的,這些商品的價格不僅沒有下降,而且在過去幾個月中還有所上漲。因此,我們的投資組合中已經蘊含著龐大的資產價值。
Looking out more broadly to the Bridge ecosystem, many of the programs we work on over at Gondola Bio affect children. And so I would expect there to be many more PRV-eligible programs in the ecosystem around Bridge.
從更廣闊的 Bridge 生態系統來看,我們在 Gondola Bio 開展的許多計畫都會影響到兒童。因此,我預計在 Bridge 周圍的生態系統中會有更多符合 PRV 資格的項目。
A great day for patients and biotech companies like us that are focused on rare disease communities.
對於患者和像我們這樣專注於罕見疾病群體的生物技術公司來說,這是一個意義非凡的一天。
Andrew Tsai - Analyst
Andrew Tsai - Analyst
Thank you.
謝謝。
Operator
Operator
Okay. That concludes our question-and-answer session for today.
好的。今天的問答環節到此結束。
I'll now hand it back over to the company.
現在我將把它交還給公司。
Chunmei Ji - Vice President, Head - Toxicology -
Chunmei Ji - Vice President, Head - Toxicology -
Thank you, investors, for joining us on our call today; and for the analysts who ask the questions.
感謝各位投資人今天參加我們的電話會議;也感謝各位分析師提出問題。
We look forward to updating you on our next quarterly call in a few months.
我們期待在幾個月後的下一次季度電話會議上向您報告最新情況。
Thank you.
謝謝。
Operator
Operator
That concludes today's meeting. You may now disconnect.
今天的會議到此結束。您現在可以斷開連線了。