Amicus Therapeutics Inc (FOLD) 2024 Q3 法說會逐字稿

Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics third quarter 2024 financial results conference call and webcast. (Operator Instructions) As a reminder, this conference call is being recorded.

女士們、先生們，早安，歡迎參加 Amicus Therapeutics 2024 年第三季財務業績電話會議和網路廣播。（操作員指示）提醒一下，本次電話會議正在錄音。

I would now like to turn the conference over to your host, Mr. Andrew Faughnan, Vice President of Investor Relations. You may now begin.

現在，我想將會議交給主持人、投資者關係副總裁 Andrew Faughnan 先生。現在您就可以開始了。

Good morning. Thank you for joining our conference call to discuss Amicus Therapeutics' third quarter 2024 financial results and corporate highlights.

早安.感謝您參加我們的電話會議，討論 Amicus Therapeutics 2024 年第三季的財務表現和公司亮點。

Leading today's call, we have Bradley Campbell, President and Chief Executive Officer; Sebastien Martel, Chief Business Officer; Dr. Jeff Castelli, Chief Development Officer; and Simon Harford, Chief Financial Officer. Joining for Q&A is Ellen Rosenberg, Chief Legal Officer.

今天的電話會議主持人是總裁兼執行長布拉德利坎貝爾 (Bradley Campbell)；賽巴斯蒂安‧馬特爾 (Sebastien Martel)，首席商務長；首席開發長 J​​eff Castelli 博士；以及財務長西蒙·哈福德（Simon Harford）。首席法律官 Ellen Rosenberg 也參與了問答環節。

As referenced on slide 2, we might make forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business as well as our plans and prospects. Our forward-looking statements should not be regarded as a representation by us that any of our plans will be achieved.

如同投影片 2 所述，我們可能會根據 1995 年《私人證券訴訟改革法案》的規定，就我們的業務以及我們的計劃和前景做出前瞻性陳述。我們的前瞻性陳述不應被視為我們對任何計劃將會實現的陳述。

Any or all the forward-looking statements made on this call may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. You are cautioned not to place undue reliance on any forward-looking statements, which speak only to the date hereof.

本次電話會議中所做的任何或所有前瞻性陳述都可能被證明是錯誤的，並且可能受到我們可能做出的不準確假設或已知或未知的風險和不確定性的影響。請注意不要過度依賴任何前瞻性陳述，這些陳述僅代表本文當日的觀點。

All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this presentation and conference call to reflect events or circumstances after the date hereof.

所有前瞻性陳述均受本警告聲明的完整限制，我們不承擔修改或更新本簡報和電話會議以反映本日期之後的事件或情況的義務。

For a full discussion of such forward-looking statements and the risks and uncertainties that may impact them, we refer you to the forward-looking statements and Risk Factors section on our annual report on Form 10-K for the year ended December 31, 2023, and the quarterly report on Form 10-Q for the quarter ended September 30, 2024 to be filed with the Securities and Exchange Commission today.

有關此類前瞻性陳述以及可能影響它們的風險和不確定性的全面討論，請參閱我們今天向美國證券交易委員會提交的截至 2023 年 12 月 31 日的年度報告 10-K 表上的前瞻性陳述和風險因素部分，以及截至 2024 年 9 月 30 日的季度報告 10-Q 表上的季度報告。

At this time, it is my pleasure to turn the call over to Bradley Campbell, President and Chief Executive Officer. Bradley?

現在，我很高興將電話轉給總裁兼執行長布拉德利坎貝爾。布拉德利？

Great. Thank you, Andrew, and welcome, everybody, to our third quarter 2024 conference call. I'm pleased today to highlight what has been a very successful nine months of the year across our global business and in particular, another very strong quarter in Q3.

偉大的。謝謝安德魯，歡迎大家參加我們 2024 年第三季的電話會議。今天，我很高興地向大家介紹，今年前九個月，我們的全球業務取得了非常成功的成績，特別是第三季度，業績表現非常強勁。

In this time, we've continued to build on our top-line revenue growth momentum, putting us well on our way to our first full year of non-GAAP profitability, while also advancing our mission of bringing hope to individuals and families affected by rare diseases. As we did in this morning's press release, let me just highlight several key points here.

在此期間，我們繼續保持營收成長勢頭，順利實現第一個非公認會計準則全年盈利，同時也推進了我們為患有罕見疾病的個人和家庭帶來希望的使命。正如我們在今天早上的新聞發布會上所說的那樣，我在這裡只強調幾個重點。

First, we continued our excellent commercial execution and delivered total revenue of $142 million in the quarter, representing 37% growth year over year or 36% on a constant currency basis. This strong performance has led us to increase our total revenue guidance for the full year to 30% to 32% from 26% to 31% previously.

首先，我們繼續保持出色的商業執行力，本季實現了 1.42 億美元的總收入，年增 37%，以固定匯率計算成長 36%。這一強勁表現促使我們將全年總收入預期從先前的26%至31%上調至30%至32%。

At the product level, Galafold continues its outstanding performance with $120 million in global revenue in the quarter, which represents 19% growth from last year on a constant currency basis. On a year-to-date basis, Galafold revenue growth was 18% year over year at constant exchange rates, coming in at the high end of our guidance for this year.

產品層面，Galafold 延續優異表現，本季全球營收達 1.2 億美元，以固定匯率計算較去年同期成長 19%。以年初至今計算，以固定匯率計算，Galafold 的營收年增 18%，達到我們今年預期的高點。

We continue to observe strong trends across our key performance indicators for Galafold in all key geographies in the quarter, including continued demand through new patient starts from both switch and naive populations in all of our leading markets and sustained compliance and adherence rates of over 90%. All of this, of course, is against the backdrop of significant growth in people diagnosed with and treated for Fabry disease.

本季度，我們繼續觀察到 Galafold 在所有主要地區的關鍵績效指標呈現強勁趨勢，包括所有主要市場中來自轉換人群和未治療人群的新患者開始的持續需求以及持續超過 90% 的合規和依從率。當然，這一切都是在法布瑞氏症診斷和治療人數顯著增長的背景下發生的。

We are extremely pleased by the significant difference this medicine continues to make for people living with Fabry disease with amenable variants in countries around the world, and we see it serving as the foundation of our business for the next decade and beyond.

我們非常高興看到這種藥物繼續為世界各國患有可耐受變異的法布瑞氏症患者帶來巨大的改變，我們認為它將成為我們未來十年及以後業務的基礎。

Based on Galafold's sustained performance, we're also increasing our full-year 2024 revenue growth guidance range for a third time this year. We started the year with a growth range of 11% to 16%. And now, we're projecting Galafold growth of 16% to 18%.

基於 Galafold 的持續表現，我們今年也第三次上調了 2024 年全年營收成長預期範圍。我們今年年初的成長率是 11% 到 16%。現在，我們預測 Galafold 的成長率為 16% 至 18%。

An important update we announced during the quarter as well was the license agreement with Teva, one of the lead parties in our ongoing IP litigation. This agreement allows Teva to market a generic version of Galafold in the United States beginning on January 30, 2037.

我們在本季宣布的一個重要更新是與 Teva 達成的授權協議，Teva 是我們正在進行的智慧財產權訴訟的主要當事方之一。該協議允許 Teva 自 2037 年 1 月 30 日起在美國銷售 Galafold 的仿製藥。

This settlement reinforces our high confidence in the strength of our case against the remaining litigants as well as the overall strength, breadth, and depth of our IP estate. And this is a huge milestone for Amicus. It represents a major step forward in ensuring Amicus can continue to support the Fabry community with Galafold for many years to come.

這次和解增強了我們對對抗剩餘訴訟當事人的實力以及我們知識產權資產的整體實力、廣度和深度的高度信心。這對 Amicus 來說是一個巨大的里程碑。這是向前邁出的重要一步，確保 Amicus 能夠在未來許多年繼續透過 Galafold 支持法布里社區。

Second, let me highlight the continued strong global commercial launch of Pombiliti and Opfolda, our novel therapy for late-onset Pompe disease. Pombiliti and Opfolda, of course, has continued to be a huge driver of growth for us this year.

其次，我要強調的是，我們針對晚發性龐貝氏症的新型療法 Pombiliti 和 Opfolda 在全球的商業推廣持續強勁。當然，龐比利蒂和奧普福德今年繼續成為我們成長的巨大動力。

And as we've mentioned, our number one focus continues to be maximizing the number of patients on therapy by year-end. Therefore, it's great to report that the rate of new commercial patients coming on to Pombiliti and Opfolda in 2024 continues to progress exceptionally well.

正如我們所提到的，我們的首要任務仍然是到年底最大限度地增加接受治療的患者數量。因此，很高興地報告，2024 年抵達龐比利蒂和奧普福德達的新商業患者的比例繼續保持異常良好的增長。

As of the end of October, there were 203 people living with late-onset Pompe disease treated or scheduled for treatment, and we continue to see the breadth and depth of prescribers increasing across our markets. We're very pleased with the demand globally for this new therapy and consistently hear inspiring anecdotes from healthcare professionals around the world and how their patients are responding to Pombiliti and Opfolda, which will help keep fueling the launch throughout the remainder of this year and beyond.

截至 10 月底，共有 203 名晚髮型龐貝病患者接受治療或計劃接受治療，並且我們繼續看到整個市場處方人員的廣度和深度不斷增加。我們對全球對這種新療法的需求感到非常高興，並不斷聽到來自世界各地醫療專業人士的鼓舞人心的故事以及他們的患者對 Pombiliti 和 Opfolda 的反應，這將有助於繼續推動該藥物在今年剩餘時間及以後的上市。

We're continuing to build momentum in our launch countries with strong switch dynamics in the US, Europe, and the UK; as well as seeing great uptake in the naive patients in the market outside of the United States. And we're also making significant progress on the reimbursement front globally. This includes moving patients more quickly through the insurance process in the United States as we've anticipated.

我們持續在產品發布國家保持強勁勢頭，在美國、歐洲和英國均呈現強勁轉換勢頭；並且在美國以外的市場中也看到了該藥物在未經治療患者中的巨大應用。我們在全球報銷方面也取得了重大進展。正如我們預期的那樣，這包括讓患者在美國更快地完成保險流程。

And for the remainder of the year and into 2025, we will focus on increasing patient access as we gain reimbursement and launch in additional countries throughout Europe. On the regulatory front, we're continuing to make great progress on expanding commercial access to Pombiliti and Opfolda through our multiple regulatory submissions as well.

在今年剩餘時間和 2025 年，我們將專注於增加患者的治療機會，同時獲得報銷並在歐洲更多國家推出產品。在監管方面，我們透過提交多項監管文件，在擴大龐比利蒂和奧普福德的商業准入方面繼續取得重大進展。

Given the strong global launch for the full year 2024, we are now raising our full-year global sales guidance to $69 million to $71 million in Pombiliti and Opfolda, which will be a significant contributor to our growth. This sets us on a great course to achieve our ambition for PomOp to become the new standard of care for this devastating disease. We're incredibly pleased to be providing a real choice for people living with Pompe disease and challenging therapeutic expectations for both physicians and their patients.

鑑於 2024 年全年全球強勁上市，我們現在將龐比利蒂和奧普福德的全年全球銷售預期上調至 6,900 萬美元至 7,100 萬美元，這將對我們的成長做出重大貢獻。這為我們實現讓 PomOp 成為這種毀滅性疾病的治療新標準的宏偉目標奠定了基礎。我們非常高興能夠為龐貝氏症患者提供真正的選擇，並挑戰醫生及其患者的治療期望。

Finally, as we continue our excellent commercial execution across both therapies, we're pleased to share that we are reducing our non-GAAP operating expenses guidance as we continue to traditionally manage our expenses. Throughout the first nine months of the year, we've exceeded our financial expectations which, as I mentioned in this morning's press release, has resulted in achieving our full -- our goal of full year non-GAAP profitability as we close the third quarter.

最後，隨著我們繼續在兩種療法中實現出色的商業執行，我們很高興地告訴大家，我們正在降低非公認會計準則營運費用指引，因為我們將繼續按照傳統方式管理我們的費用。今年前九個月，我們的財務表現超出預期，正如我在今天早上的新聞稿中提到的那樣，在第三季度結束時，我們實現了全年非 GAAP 盈利的目標。

And finally, let me touch on slide 4, our key strategic priorities for the year. Again, sustaining double-digit Galafold revenue growth. And as I mentioned, we've raised the expected annual growth rate of Galafold to 16% to 18%, continuing to successfully execute multiple commercial launches of Pombiliti and Opfolda -- and again, we've raised our guidance there as well -- advancing ongoing studies to support medical and scientific leadership in Fabry disease and Pompe disease, and finally, maintaining a strong financial position as we carefully manage our expenses and investments to achieve non-GAAP profitability for the full year.

最後，讓我談談投影片 4，也就是我們今年的關鍵策略重點。Galafold 收入再次維持兩位數成長。正如我所提到的，我們已將 Galafold 的預期年增長率提高到 16% 至 18%，繼續成功執行 Pombiliti 和 Opfolda 的多次商業發布——而且，我們也再次提高了這方面的指導方針——推進正在進行的研究以支持法布里病和龐貝病的醫學和科學領導地位，最後，保持強勁的財務狀況，我們謹慎管理我們全年的投資，實現我們全年的利潤。

With that, let me now hand the call over to Sebastian, who can further highlight our commercial performance. Sebastian?

說完這些，現在我將把電話交給塞巴斯蒂安，他可以進一步強調我們的商業表現。塞巴斯蒂安？

Thank you, Brandy. Good morning to everyone on the call. I will start by providing you with more details on our Galafold performance for the quarter.

謝謝你，布蘭迪。通話中的各位早安。首先，我將向您提供有關本季 Galafold 業績的更多詳細資訊。

On slide 6, for the third quarter 2024, Galafold reported revenue reached $120 million, driven by strong patient demand, particularly from our leading markets. We ended the third quarter with more than 60% of the global market share of treated Fabry patients with amenable mutations.

在幻燈片 6 上，Galafold 報告稱，2024 年第三季的收入達到 1.2 億美元，這得益於強勁的患者需求，尤其是來自我們主要市場的需求。截至第三季度，我們佔了全球接受治療的、伴隨可耐受突變的法布瑞氏症患者市場份額的 60% 以上。

We're very pleased to see that prescribers continue to position Galafold as the treatment of choice for Fabry patients with amenable variants. The great news is that there's still many more potential patients eligible for our therapy.

我們非常高興地看到，處方人員繼續將 Galafold 定位為適合治療的法布瑞氏症患者的首選治療方法。好消息是，有更多潛在患者適合接受我們的治療。

Turning to slide 7. Our results in the third quarter highlight the strength of our global commercial efforts. The demand for Galafold globally continued to be strong with patients added in all major markets, delivering operational growth of 19% over the same period in 2023 at constant exchange rates.

翻到幻燈片 7。我們第三季的業績凸顯了我們全球商業努力的實力。全球對 Galafold 的需求持續強勁，所有主要市場的患者數量都有所增加，以固定匯率計算，2023 年同期的營運成長率為 19%。

Our leading markets such as the US, the UK, top EU countries, and Japan remain the biggest drivers of patient demand and gives great confidence in the growth this product has over the long-term. Within the global mix, which is about 40% switch and 60% naive, we're seeing stronger uptake in naive populations. So we continue to achieve high market shares in a country where we've been approved the longest.

我們的主要市場如美國、英國、歐盟主要國家和日本仍然是患者需求的最大驅動力，並對該產品的長期成長充滿信心。在全球組合中，大約有 40% 的人轉換了疫苗，而 60% 的人仍是未接種疫苗的人，我們發現未接種疫苗的人群的接受度更高。因此，我們在獲得批准時間最長的國家繼續取得較高的市場份額。

There's still plenty of opportunity to continue to switch patients over to Galafold and to keep growing the market as we penetrate the diagnosed untreated and newly diagnosed segments. All of that is underpinned by sustained compliance and adherence rates that continued to exceed 90%, reiterating our belief that those patients who go on Galafold predominantly stay on Galafold.

隨著我們滲透到確診未治療和新診斷的細分市場，仍然有足夠的機會繼續讓患者轉向使用 Galafold 並保持市場成長。所有這些都以持續超過 90% 的依從性和堅持率為基礎，這再次證明了我們的信念：那些使用 Galafold 的患者主要會繼續使用 Galafold。

On slide 8, we know that there is a significant patient demand for Galafold and that the segment of the global Fabry market made of patients with amenable variants have the potential to reach up to $1 billion in annual revenue by the end of the decade.

在投影片 8 上，我們知道患者對 Galafold 的需求很大，並且到 2020 年，全球法布瑞氏症市場中由易治療變異患者組成的部分年收入有可能達到 10 億美元。

We anticipate sustained growth in 2024 and beyond to be driven by several key drivers. First, the Fabry market is growing robustly with a significant portion of growth coming from finding new patients and reaching the diagnosed and treated population.

我們預計，2024 年及以後的持續成長將由幾個關鍵驅動因素推動。首先，法布雷氏症市場正在強勁成長，其中很大一部分成長來自於尋找新患者以及涵蓋已確診和接受治療的人。

We've seen many new patients going to treatment through family screening, and we continue to see increased patient identification through ongoing medical education and support of novel screening initiatives.

我們看到許多新患者透過家庭篩檢接受治療，我們繼續看到透過持續的醫學教育和對新篩檢計劃的支持，患者識別率不斷提高。

Fabry remains one of the most underdiagnosed rare disease. So the more people that can be identified, the more people may benefit from Galafold. We're also seeing many diagnosed untreated patients transition to treatment as the need for earlier treatment, especially in females, becomes better appreciated.

法布瑞氏症仍然是診斷不足的罕見疾病之一。因此，能夠識別的人越多，從 Galafold 中受益的人就越多。我們也看到許多未經治療的確診患者正在轉向治療，因為人們越來越認識到早期治療的必要性，特別是對於女性而言。

The other piece is continuing to drive Galafold's market share of treated amenable patients through continued commercial execution. As noted, Galafold currently at more than 60% of the global amenable market, but we're seeing in our most mature markets that we can reach up to about 85%, 90% of market share.

另一部分是透過持續的商業執行，繼續推動 Galafold 在可治療患者的市場份額。如上所述，Galafold 目前佔據全球可銷售市場的 60% 以上，但我們發現，在最成熟的市場中，我們的市佔率可達到約 85% 甚至 90%。

So we know that there's the potential to reach those levels in the global market share as well. And again, all these efforts are supported by solid compliance and adherence rates through physician and patient education and support programs.

因此我們知道全球市場份額也有可能達到這些水準。而且，所有這些努力都得益於醫生和患者的教育和支持計劃所獲得的穩定合規和堅持率的支持。

Additionally, we'll continue to make progress on expanding Galafold in new markets and extending the labels. Importantly, on the IP front, as Bradley mentioned, we recently announced in October that we reached a settlement agreement with Teva Pharmaceuticals in the ongoing protection of our US intellectual property for Galafold.

此外，我們將繼續推進 Galafold 在新市場的擴張和標籤的延伸。重要的是，在智慧財產權方面，正如布拉德利所提到的，我們最近在 10 月宣布，我們已與 Teva Pharmaceuticals 達成和解協議，以持續保護我們對 Galafold 的美國智慧財產權。

Amicus entered into a license agreement with Teva, granting them a license to market its generic version of Galafold in the US beginning on January 30, 2037. We're very pleased by this outcome as it reinforces our confidence in the strength of our case against the remaining litigants as well as the overall strength, breadth, depth of our IP estate.

Amicus 與 Teva 簽訂了許可協議，授予其自 2037 年 1 月 30 日起在美國銷售其 Galafold 仿製藥的許可。我們對這一結果感到非常滿意，因為它增強了我們對自己對抗剩餘訴訟當事人的實力以及我們知識產權資產的整體實力、廣度和深度的信心。

We now have orphan drug -- sorry, we now have orphan drug exclusivity in the US and Europe, in addition to our 62 Orange Book listed patents, 46 of which provide protection into 2038 and beyond, including 15 -- this gives us the opportunity to provide access to Galafold globally for a long time to come, and we intend to continue to protect and enforce our broad intellectual property rights.

我們現在擁有孤兒藥——抱歉，我們現在在美國和歐洲擁有孤兒藥專營權，此外還有我們的 62 項橙皮書列出的專利，其中 46 項提供到 2038 年及以後的保護，包括 15 項——這使我們有機會在未來很長一段時間內在全球範圍內提供 Galafold 的使用權，我們打算繼續廣泛的智慧。

Looking ahead, we expect steady double-digit growth for Galafold throughout 2024, and we remain confident that with our strong IP protection, Galafold has a long runway well into the next decade.

展望未來，我們預計 Galafold 將在 2024 年實現穩定的兩位數成長，並且我們仍然相信，憑藉我們強大的智慧財產權保護，Galafold 在下一個十年將擁有長久的發展空間。

Turning now to Pompe disease on slide 10. We outlined our global launch progress with Pombiliti and Opfolda. For the third quarter 2024, Pombiliti and Opfolda reported revenue reached $21 million. This represents an increase of 33% compared to the second quarter 2024 and provides a strong foundation for the remainder of the year.

現在就來看看幻燈片 10 上的龐貝氏症。我們與 Pombiliti 和 Opfolda 共同概述了我們的全球發布進展。2024 年第三季度，Pombiliti 和 Opfolda 報告收入達到 2,100 萬美元。這與 2024 年第二季相比成長了 33%，為今年剩餘時間奠定了堅實的基礎。

In the US, we continue to see a majority of patients switching from Nexviazyme, about 68% and the remaining from Lumizyme. This means we're switching patients proportionately from both products. We're also seeing a broadening and deepening of prescriptions with more sites coming online and multiple new prescriptions from physicians coming in over Q3.

在美國，我們繼續看到大多數患者從 Nexviazyme 轉而使用藥物，約 68%，其餘患者從 Lumizyme 轉而使用藥物。這意味著我們會按比例為患者轉換兩種產品。我們也看到處方範圍的廣度和深度不斷擴大，越來越多的網站上線，第三季醫生開出了更多的新處方。

Outside of the US, we're seeing patients from all three segments, some switching from Myozyme and from Nexviazyme at a proportional rate to the respective market shares and some from the naive population, exactly what you want to see at this stage in the launch. Given our solid commercial execution in the first nine months of 2024, we're pleased to increase our full year revenue guidance from $62 million to $67 million, to $69 million to $71 million for Pombiliti and Opfolda.

在美國以外，我們看到來自所有三個領域的患者，其中一些患者從 Myozyme 和 Nexviazyme 轉換而來，轉換速度與各自的市場份額成比例，還有一些患者來自未曾使用過的人群，這正是您在發布階段所希望看到的。鑑於我們在 2024 年前 9 個月的穩健商業執行，我們很高興將全年收入預期從 6200 萬美元上調至 6700 萬美元，將龐比利蒂和奧普福德的全年收入預期上調至 6900 萬美元至 7100 萬美元。

Moving to slide 11. We're very pleased with the launch momentum through the first nine months of the year. As Bradley mentioned, there are about 203 patients that have been treated or are scheduled for treatment as of late October. That's about 196 patients who have been treated. We remain very satisfied with ongoing demand for this therapy.

移至第 11 張投影片。我們對今年前九個月的發布勢頭感到非常滿意。正如布拉德利所提到的，截至 10 月底，大約有 203 名患者已經接受治療或正在安排治療。這意味著已有約 196 名患者接受了治療。我們對這種療法的持續需求仍然非常滿意。

Our launch has leveraged our highly experienced cross-functional teams, and we've had great outreach with key opinion leaders. We're seeing an increase in the depth and breadth of prescribers across all markets indicated to us that physicians are having positive experiences with the therapy and are gaining greater confidence to use it again in patients.

我們的發布充分利用了我們經驗豐富的跨職能團隊，並且與關鍵意見領袖進行了廣泛的接觸。我們看到各個市場的處方深度和廣度都有所增加，這向我們表明醫生對該療法有著積極的體驗，並且對再次在患者身上使用該療法更有信心。

All core treating centers are engaged and we continue to receive very positive feedback from HCPs and other stakeholders as to our business approach, our support and our patient focus. Finally, an important metric to track is our progress with access and reimbursement. We have a highly experienced team interacting in positive conversations with payers to demonstrate the value of Pombiliti and Opfolda.

所有核心治療中心都參與其中，我們繼續收到來自 HCP 和其他利害關係人對我們的業務方法、我們的支持和我們對患者的關注的非常積極的回饋。最後，需要追蹤的一個重要指標是我們在訪問和報銷方面的進展。我們擁有一支經驗豐富的團隊，與付款人進行積極的對話，以展示 Pombiliti 和 Opfolda 的價值。

In the US, the largest players have put Pombiliti and Opfolda onto their respective formularies and we've also seen strong acceptance by Medicare, Medicaid. The overall time from prescription to infusion is now down to around 60 days with pay approvals happening under 30 days. We believe this will continue to improve.

在美國，最大的製藥公司已將 Pombiliti 和 Opfolda 納入各自的處方集，我們也看到醫療保險和醫療補助計劃對其的強烈接受。從處方到輸液的總時間現在減少到 60 天左右，其中付款審批在 30 天內完成。我們相信這種情況將會繼續改善。

Today, we're launched in Germany, in the UK, the US, Austria, and Spain, and we're pleased to announce that we now have pricing and reimbursement in the Czech Republic, our sixth launch country. We remain in active pricing and reimbursement discussions with additional major European markets.

今天，我們在德國、英國、美國、奧地利和西班牙推出了該產品，我們很高興地宣布，我們現在在第六個推出該產品的國家捷克共和國推出了定價和報銷服務。我們仍在與其他主要歐洲市場進行積極的定價和報銷討論。

We continue to be focused on securing broad patient access throughout Europe, and we anticipate further reimbursement agreements over the next six to nine months. This will be a key part in driving the commercial uptake in new countries next year.

我們將繼續致力於確保整個歐洲的廣泛患者能夠獲得治療，並預計在未來六到九個月內達成進一步的報銷協議。這將成為明年推動新國家商業擴張的關鍵部分。

Overall, we're very pleased thus far with the launch of Pombiliti and Opfolda across the first wave of countries in 2024. Our focus is on maximizing the number of patients on therapy by year-end. The strength of our clinical data, the depth of experience and talent we have at Amicus gives us great confidence in our ability to make a very real difference for people living with Pompe disease.

總的來說，到目前為止，我們對 2024 年在第一批國家推出 Pombiliti 和 Opfolda 感到非常高興。我們的目標是到年底最大限度地增加接受治療的患者數量。我們強大的臨床數據、豐富的經驗以及 Amicus 的人才使我們非常有信心，我們有能力為龐貝病患者帶來真正的改變。

With that, let me now hand the call over to Jeff to highlight our clinical and regulatory progress.

說完這些，現在我將把電話交給傑夫，介紹我們的臨床和監管進度。

Thank you, Sebastian, and good morning. On slide 12, we outline how we continue to grow the body of evidence for Pombiliti and Opfolda through our ongoing clinical studies and registry as we continue to execute on expanding commercial access through regulatory submissions.

謝謝你，賽巴斯蒂安，早安。在第 12 張投影片上，我們概述瞭如何透過正在進行的臨床研究和註冊繼續增加 Pombiliti 和 Opfolda 的證據，同時我們將繼續透過監管提交來擴大商業管道。

In addition to the regulatory dossiers already under review in Canada and Australia, we are pleased to announce that we recently submitted a Japan New Drug Application, or JNDA, for Pombiliti and Opfolda to the Ministry of Health, Labor, and Welfare in Japan.

除了加拿大和澳洲已在審查的監管檔案外，我們很高興地宣布，我們最近向日本厚生勞動省提交了 Pombiliti 和 Opfolda 的日本新藥申請（JNDA）。

For the younger Pompe community, we continue to enroll the ongoing open-label ZIP study for children living with late onset Pompe disease in the open-label Rosella study for children living with infantile-onset Pompe disease. We see this as an important opportunity to support label expansions into these patient segments in the years ahead and importantly, to address the significant unmet needs in these children.

對於較年輕的龐貝氏症患者群體，我們將繼續招募正在進行的針對晚髮型龐貝氏症兒童的開放標籤 ZIP 研究和針對嬰兒型龐貝氏症兒童的開放標籤 Rosella 研究。我們認為這是一個重要的機會，可以在未來幾年支持標籤擴展到這些患者群體，更重要的是，可以解決這些兒童尚未滿足的重大需求。

Through ongoing clinical studies and the Amicus Pompe registry, we continue to generate evidence on the differentiated mechanism of action and long-term impact of Pombiliti and Opfolda across endpoints and patient populations. Our medical conference presence and scientific publications continue to be an important part of our education efforts.

透過正在進行的臨床研究和 Amicus Pompe 註冊，我們繼續收集有關 Pombiliti 和 Opfolda 在終點和患者群體中的差異化作用機制和長期影響的證據。我們的醫學會議出席和科學出版物繼續成為我們教育工作的重要組成部分。

Finally, as highlighted in the pipeline side in the appendix, for our earlier-stage pipeline, we continue to focus on novel approaches to next-generation therapies in Fabry and Pompe diseases.

最後，正如附錄中的管道部分所強調的那樣，對於我們早期的管道，我們將繼續專注於法布瑞氏症和龐貝氏症的下一代治療新方法。

And with that, I would like to now turn the call over to Simon Harford, our Chief Financial Officer; to review our financial results, guidance and outlook. Simon?

現在，我想將電話轉給我們的財務長西蒙‧哈福德 (Simon Harford)；審查我們的財務表現、指導和展望。西蒙？

Thank you, Jeff. Our financial overview begins on slide 14 with our income statement for the third quarter ending September 30, 2024.

謝謝你，傑夫。我們的財務概覽從第 14 張投影片開始，其中包含截至 2024 年 9 月 30 日的第三季損益表。

For Q3, we achieved total revenue of $142 million which is a 37% increase over the same period in 2023. At constant exchange rates, revenue also grew 36%. The global geographic breakdown of total revenue during the quarter consisted of $85 million or 60% of revenue generated outside the US and the remaining $56 million or 40% coming from within the US.

第三季度，我們實現總營收 1.42 億美元，比 2023 年同期成長 37%。以固定匯率計算，營收也成長了 36%。本季總收入的全球地理分佈包括 8,500 萬美元（佔 60%）來自美國以外地區，其餘 5,600 萬美元（佔 40%）來自美國境內。

Cost of goods sold as a percentage of net sales was 9.4% in Q3 2024 as compared to 9.6% for the prior year period, staying relatively flat. Total GAAP operating expenses decreased to $107 million for the third quarter 2024 as compared to $111 million in the third quarter of last year, a decrease of 4%.

2024 年第三季度，銷售成本佔淨銷售額的百分比為 9.4%，而去年同期為 9.6%，保持相對穩定。2024 年第三季的 GAAP 總營運費用從去年第三季的 1.11 億美元下降至 1.07 億美元，降幅為 4%。

On a non-GAAP basis, total operating expenses decreased to $83 million for the third quarter as compared to $90 million in the third quarter of last year, a decrease of 8%. We define non-GAAP operating expenses, research and development and SG&A expenses, excluding stock-based compensation expense, loss on impairment of assets, changes in fair value of contingent consideration, restructuring charges, and depreciation and amortization.

以非美國通用會計準則計算，第三季總營運費用從去年同期的 9,000 萬美元下降至 8,300 萬美元，降幅為 8%。我們定義非公認會計準則營業費用、研發費用及銷售、一般及行政費用，不包括股票薪酬費用、資產減損損失、或有對價公允價值變動、重組費用及折舊及攤提。

On a GAAP basis, net loss for the third quarter 2024 reduced to $7 million or $0.02 per share compared to a net loss of $22 million or $0.07 per share for the third quarter of 2023. In Q3 2024 non-GAAP net income was $31 million or a profit of $0.10 per share compared to non-GAAP net loss of $4 million or a loss of $0.01 per share for the third quarter of last year.

根據 GAAP 計算，2024 年第三季淨虧損減至 700 萬美元或每股 0.02 美元，而 2023 年第三季淨虧損為 2,200 萬美元或每股 0.07 美元。2024 年第三季非 GAAP 淨收入為 3,100 萬美元，即每股利潤 0.10 美元，而去年第三季非 GAAP 淨虧損為 400 萬美元，即每股虧損 0.01 美元。

Cash, cash equivalents and marketable securities were $250 million at September 30, 2024, compared to $286 million at December 31, 2023. Turning now to slide 15.

截至 2024 年 9 月 30 日，現金、現金等價物及有價證券為 2.5 億美元，而 2023 年 12 月 31 日為 2.86 億美元。現在翻到第 15 張投影片。

We are raising our full year 2024 total revenue guidance range to 30% to 32% from 26% to 31% previously. This is driven by the increase of our full year 2024 Galafold revenue growth guidance from 11% to 16% at the beginning of 2024 to most recently 14% to 18% and now narrowed to 16% to 18% at constant exchange rates.

我們將 2024 年全年總收入預期範圍從先前的 26% 至 31% 上調至 30% 至 32%。這是由於我們將 2024 年全年 Galafold 營收成長預期從 2024 年初的 11% 至 16% 上調至最近的 14% 至 18%，現在按固定匯率縮小至 16% 至 18%。

In addition, we are raising the guidance for Pombiliti and Opfolda sales to $69 million to $71 million for the year. Our full year 2024 non-GAAP operating expense guidance has been lowered from $345 million to $360 million down to $340 million to $350 million. With our commitment to full year non-GAAP profitability during the first full year of launch of Pombiliti and Opfolda, we are keeping operating expense growth in low single digits year over year at the midpoint of guidance.

此外，我們將今年龐比利蒂和奧普福德的銷售預期上調至 6,900 萬美元至 7,100 萬美元。我們對 2024 年全年非 GAAP 營業費用的預期已從 3.45 億美元至 3.6 億美元下調至 3.4 億美元至 3.5 億美元。我們承諾在 Pombiliti 和 Opfolda 推出的第一個完整年度內實現全年非 GAAP 盈利，並將營業費用增長率保持在指導中位數的低個位數。

As a reminder, we continue to have R&D commitments, including registry studies in both Fabry and Pompe, the ongoing Pompe Phase 3 study in countries not yet reimbursed, as well as next-generation manufacturing for Pombiliti. With our total revenue guidance of 30% to 32% growth, we remain comfortably on track for 2024 for our first full year of non-GAAP profitability.

提醒一下，我們將繼續進行研發工作，包括法布瑞氏症和龐貝氏症的註冊研究、在尚未報銷的國家進行的龐貝氏症 3 期研究，以及龐比利蒂的下一代製造。我們預計總收入將成長 30% 至 32%，因此我們仍有望在 2024 年實現非 GAAP 獲利的首個全年目標。

As a heads up, as we begin to think about 2025, we anticipate next year to be a hybrid year for Pombiliti and Opfolda COGS as we expect to work through the previously expensed inventory during the first part of 2025. Total gross margins for the first nine months of the year 2024 were approximately 90%. We expect gross margins in the mid-80s for the 2025 full year as we begin to recognize Pombiliti and Opfolda COGS through the P&L next year.

需要注意的是，當我們開始考慮 2025 年時，我們預計明年將是 Pombiliti 和 Opfolda COGS 的混合年，因為我們預計將在 2025 年上半年處理之前費用化的庫存。2024 年頭九個月的總毛利率約為 90%。隨著我們明年開始透過損益表確認 Pombiliti 和 Opfolda 的銷貨成本，我們預計 2025 年全年的毛利率將在 85% 左右。

And with that, let me turn the call back over to Bradley for our closing remarks.

最後，請允許我將電話轉回給布拉德利，請他作最後發言。

Great. Thanks, Simon, Sebastian, Jeff. As you can all see, we have been relentlessly focused on commercial execution and performance across the business, driven by our passionate team of global employees who continue to lead us on our patient-focused mission.

偉大的。謝謝，西蒙、賽巴斯蒂安、傑夫。正如大家所看到的，在我們充滿熱情的全球員工團隊的推動下，我們一直堅持不懈地專注於整個業務的商業執行和績效，他們繼續帶領我們實現以患者為中心的使命。

As we near the end of 2024, I am confident we have laid a solid foundation for Amicus and continue to deliver transformative therapies in 2025 and the years beyond. With that, operator, we can now open the call to questions.

隨著 2024 年即將結束，我相信我們已經為 Amicus 奠定了堅實的基礎，並將在 2025 年及以後繼續提供變革性療法。接線員，現在我們可以開始提問了。

(Operator Instructions) Ritu Baral, TD Cowen.

(操作員指示) Ritu Baral，TD Cowen。

I wanted to ask about your Pombiliti prescribing trends that you mentioned. You mentioned that you had improving trends on both sites and prescribers. Could you go into that a little bit and describe a little more what you're seeing now about US -- like the point at which US prescribers and patients elect to switch specifically what the patients are experiencing? And then could you help us just quantify the Japanese market opportunity in Pompe since that's coming up?

我想詢問一下您提到的 Pombiliti 處方趨勢。您提到，站點和處方方面都有所改善的趨勢。您能否稍微詳細地介紹一下這一點，並進一步描述一下您現在在美國看到的情況——例如美國處方人員和患者在選擇轉換治療時，患者俱體經歷了什麼？那麼，您能否幫助我們量化即將到來的龐貝日本市場機會？

Sure. So it was a very well-articulated one question with three parts. So I heard breadth and depth, a little bit more color on breadth and depth, and then what we're seeing and hearing around decision to switch and then the Japan opportunity. I'll start maybe framing the first one. We'll ask Jeff to comment a little bit, and then Sebastian can provide color on Japan.

當然。所以這是一個非常清晰的問題，包含三個部分。因此，我聽到了廣度和深度，在廣度和深度上有更多的細節，然後是我們所看到和聽到的有關轉換的決定以及日本的機會。我可能會開始構思第一個。我們會請傑夫發表一些評論，然後塞巴斯蒂安可以對日本進行介紹。

So in terms of breadth and depth, it's exactly what it says, right? So in some markets like the UK where it's a very concentrated market, we had all the key centers prescribing through the EMS program and continue to prescribe on a commercial basis. But most other markets, Spain, Germany, United States, have more prescribers.

那麼從廣度和深度來說，它就是所說的，對嗎？因此，在英國等市場非常集中，我們讓所有主要中心都透過 EMS 計劃開處方，並繼續以商業方式開立處方。但大多數其他市場（西班牙、德國、美國）的處方人員更多。

And so what you want to see is you want to see both breadth, meaning continuing to have new prescribers to prescribe the medicine; and depth, meaning they are repeating prescriptions. And we're seeing both of those things. So more new prescribers using Pombiliti and Opfolda for the first time and then more prescribers prescribing multiple prescriptions. So I think that's a great tracker to see how we'll continue to spread the use of this product.

所以你想看到的是兩種廣度，意味著繼續有新的開藥者來開藥；和深度，這意味著他們正在重複處方。我們都看到了這兩件事。因此，更多的新開處方者首次使用 Pombiliti 和 Opfolda，然後更多的開處方者開出多種處方。所以我認為這是一個很好的追蹤器，可以讓我們了解如何繼續推廣產品的使用。

In terms of when the decision is made, I think this is an important part of the ongoing discussion with the community. Broadly speaking, I think what we've heard and seen is that physicians are -- want to see some evidence of decline in their patients. And we talked a lot about that over the course of the year.

至於何時做出決定，我認為這是與社區持續討論的重要部分。廣義上講，我認為我們所聽到和看到的是，醫生希望看到他們的病人病情惡化的證據。我們在這一年中多次討論過這個問題。

Once a patient switches, I think physicians are looking for 12 to 24 months before they're ready to switch. And so I think that sets us up well for next year. But maybe Jeff, talk about specifically what are the signs and symptoms that would be typical for people to follow. And then Sebastian, just give a quick update on the Japan opportunity.

一旦患者轉換治療方案，我認為醫生需要等待 12 到 24 個月才能做好轉換準備。所以我認為這為我們明年的工作做好了準備。但是也許傑夫，可以具體談談人們通常會出現哪些徵兆和症狀。然後塞巴斯蒂安，請簡單介紹一下日本的機會。

Yeah. Thanks, Brad and thanks for the question, Ritu. So I think Brian covered it very well there in terms of that 12- to 24-month period after starting treatment or switching treatments that physicians follow their patients. They typically see them every six months or so.

是的。謝謝，布拉德，也謝謝你的提問，裡圖。所以我認為 Brian 對醫生在開始治療或轉換治療後對患者進行 12 到 24 個月的隨訪期間進行了很好的闡述。他們通常每六個月左右見一次面。

A lot of what they're measuring are quality of life measures, how has the patient been doing in their day-to-day activities. They do still measure a lot of the things we see in trials, different measures of respiratory function, different ways to assess mobility, certainly not quite the workup you see in the trial. But it's really that holistic assessment at each visit, how the patient is doing.

他們測量的內容很多都是生活品質指標，例如病人的日常活動表現如何。他們確實仍然測量我們在試驗中看到的許多東西，不同的呼吸功能測量方法，不同的評估活動能力的方法，當然不是你在試驗中看到的檢查。但實際上每次就診時都會進行整體評估，以了解病人的狀況。

But it's certainly -- even some of the guidance we're seeing has recommended that physician at least wait 12 to 18 months before initiating a second switch after that first switch. And currently, it is if a patient was declining and they start new treatment, they want to wait to see are they declining on the new treatment again. If they were stable, they wait to see if they remain stable or show any improvement.

但可以肯定的是——我們看到的一些指導甚至建議醫生在第一次轉換後至少等待 12 到 18 個月才能進行第二次轉換。目前的情況是，如果患者的病情惡化，然後開始接受新的治療，他們必須等待，看看新療法是否會再次使患者的病情惡化。如果情況穩定，他們會等待，看看是否能保持穩定或顯示任何改善。

So it's somewhat patient by patient. But we're definitely seeing that the majority of switches are coming in that 12- to 24-month period.

所以這在某種程度上取決於患者本身。但我們確實看到大多數轉變都發生在這 12 到 24 個月期間。

Sebastien, just real quick on the Japan opportunity.

塞巴斯蒂安，很快地談談日本的機會。

Yeah. Thanks, Ritu, for the question. So we're -- in the Fabry world, we used to being a very sizable market. This is actually the second largest market for Fabry disease. It's not the case for Pompe disease. It is relatively speaking, a smaller opportunity, Pompe disease is less prevalent in Japan. The market is also very fragmented. We estimate that there are about 100 patients currently treated for Pompe disease in Japan.

是的。謝謝 Ritu 提出這個問題。所以，在 Fabry 的世界裡，我們曾經是一個非常龐大的市場。這其實是法布瑞氏症的第二大市場。龐貝病則不是這種情況。相對來說，機會較小，龐貝氏症在日本的發生率較低。市場也非常分散。我們估計，目前日本約有 100 名龐貝氏症患者正在接受治療。

Anupam Rama, JPMorgan.

摩根大通的 Anupam Rama。

I just wanted to dig in a little bit on the previously untreated patients that are now on Galafold. I think the slide says around 60%. Can you speak to like what you're seeing about these previously untreated patients in some of your core regions or more legacy regions versus some of the emerging market regions? That's my one question.

我只是想深入了解一下之前未接受過治療但現在使用 Galafold 的患者。我認為幻燈片上說的是 60% 左右。您能否談談在某些核心地區或更多傳統地區以及某些新興市場地區對這些先前未接受治療的患者的情況？這是我的一個問題。

Yeah. Thanks a lot, Anupam. So I'll just take that one. So I think in our key markets, so US, Europe, Japan, as an example, we're still finding significant numbers of new patients. And remember, also, there's a significant pool of diagnosed untreated patients.

是的。非常感謝，Anupam。所以我只選那個。因此我認為在我們的主要市場，例如美國、歐洲、日本，我們仍然發現大量新患者。還要記住的是，有大量已確診但未接受治療的患者。

So we came into the year with updated figures estimating about 18,000 diagnosed patients, about 11,000 -- sorry, 17,000 diagnosed patients, 11,000 treated and 6,000 diagnosed untreated. So part of the phenomenon is penetrating into that diagnosed untreated market.

因此，我們根據最新數據估算，今年確診患者約有 18,000 名，確診患者約有 11,000 名——抱歉，是 17,000 名，接受治療的患者約有 11,000 名，未經治療的確診患者約有 6,000 名。因此，部分現象正在滲透到已確診但未治療的市場。

But with advances in low-cost genetic testing with, I think, significant adoption of family screening where you find typically four or five family members when you find an index Fabry patient, that's led to diagnosing a whole host of new patients as well. As we come to JPMorgan, we give some updated numbers there.

但隨著低成本基因檢測的進步，我認為，家庭篩檢的廣泛應用，當你發現一個法布瑞氏症患者時，通常只有四、五個家庭成員，這也導致可以診斷出一大批新患者。當我們談到摩根大通時，我們給出了一些更新的數據。

But I think overall, what you're seeing in, I think, the more developed countries is significant levels of diagnosis. The other thing is we are still switching patients. We're at very high market shares, 80%-plus. But in our newer launch regions, which tend to be Latin America, Southeast Asia, some Middle East, North Africa regions, there is still a significant switch opportunity as well.

但我認為整體而言，在較發達國家中，診斷水準較高。另一件事是我們仍在更換病人。我們的市佔率非常高，超過80％。但在我們較新推出的地區，通常是拉丁美洲、東南亞、部分中東、北非地區，仍然有很大的轉換機會。

So I think two growth drivers there. But again, I think that solid underpinning of diagnosis and market growth.

所以我認為那裡有兩個成長動力。但我再次認為，診斷和市場成長有著堅實的基礎。

Tazeen Ahmad, Bank of America Securities.

塔澤恩·艾哈邁德（Tazeen Ahmad），美國銀行證券。

On Galafold, as it relates to IP, are there any filers still left on that front? Is there a chance, I'm wondering, if you could get any better outcome than the one that you've just announced the settlement with? And then just a point of clarification as it relates to dropout rates for Galafold, can you tell us if those rates have remained the same or changed much at all recently?

關於 Galafold，由於它與 IP 相關，是否存在任何文件提交者？我想知道，有沒有可能得到比你剛剛宣布的和解更好的結果？然後，關於 Galafold 的輟學率，您只想澄清一點，您能告訴我們這些輟學率最近是否保持不變或有很大變化嗎？

Thanks very much, Tazeen. Yes. So on both of those -- so as we said on the call, we're very pleased with the settlement with Teva. It really reinforces our confidence in the strength of the case against the remaining litigants. And to your question, there are two Aurobindo and Lupin. However, we will continue, of course, to vigorously prosecute and force the IP and remain very confident in the long-term potential based on this outcome.

非常感謝，Tazeen。是的。所以就這兩點而言——正如我們在電話中所說的那樣，我們對與 Teva 達成的和解感到非常滿意。這確實增強了我們對該案對抗剩餘訴訟當事人實力的信心。對於你的問題，有兩個奧羅賓多和盧平。然而，我們當然會繼續大力起訴和強制執行智慧財產權，並且對基於此結果的長期潛力仍然充滿信心。

I will say that because we're still in litigation with Aurobindo in particular, we can't comment on the litigation strategy. But of course, we've pointed people to the statistics, which say that in the majority of these types of cases, ultimately, there's a settlement with the parties.

我想說的是，由於我們仍在與 Aurobindo 進行訴訟，因此我們無法對訴訟策略發表評論。但當然，我們已經向人們指出了統計數據，數據顯示，在大多數此類案件中，雙方最終都會達成和解。

I would not expect an earlier -- or excuse me, a later date to your question. And again, I won't comment more than that other than we're very confident in where we are. And I think we're -- everybody is very pleased that this gives us an opportunity to continue to support Galafold for many years to come.

我不希望您的問題能更早——或者抱歉，更晚——得到答案。再說一次，我不會發表更多評論，只是我們對目前的狀況非常有信心。我認為我們——每個人都非常高興，這讓我們有機會在未來許多年繼續支持 Galafold。

On your second point on compliance and adherence for Galafold, actually, we review these numbers quite regularly. What's interesting is the rate of discontinuation has actually gotten smaller and smaller on a broader base. And I think that's what's given us this continued sustained patient growth effectively when patients come on Galafold, they tend to stay on Galafold, which is a fantastic place to be.

關於您提到的第二點關於 Galafold 的合規性和依從性，實際上，我們會定期審查這些數字。有趣的是，從更廣泛的角度來看，停藥率實際上已經越來越小。我認為這正是我們能夠持續有效地增加患者數量的原因。

Ellie Merle, UBS.

瑞銀的艾莉·梅爾（Ellie Merle）

In terms of thinking about your pipeline, I guess, what's your focus as you think about building out your clinical pipeline from here? Are you more focused on internal development work? Or are you looking externally with business development? And then I guess as you think about business development, are there specific modalities or stages of development where you're most focused? And just how you're thinking about that strategy overall?

在考慮您的管道方面，我想，當您考慮從現在開始建立您的臨床管道時，您的重點是什麼？您是否更專注於內部開發工作？或是您正在尋求外部的業務發展？然後我想，當您考慮業務發展時，是否存在您最關注的特定模式或發展階段？那麼您對於這個策略的整體看法是什麼？

Sure. I'll start with framing the overall strategy and then maybe Jeff talk a little bit about our internal program. So overall, of course, we continue to focus on growing the business, and I think we've delivered another strong quarter here, which is great. And that will continue to be a focus for Amicus.

當然。我將首先制定總體戰略，然後傑夫可能會談一點我們的內部計劃。因此總的來說，我們當然會繼續專注於業務成長，而且我認為我們又度過了一個強勁的季度，這很好。這將繼續成為 Amicus 的重點。

We do believe because of the great infrastructure we have built to support Galafold and PomOp, there is an opportunity to bring assets in. And I think we've said over the next 12 to 24 months, we would be looking to bring in first de-risked later stage or commercial assets into the portfolio.

我們確實相信，由於我們為支持 Galafold 和 PomOp 而建造的強大基礎設施，我們有機會引入資產。我想我們已經說過，在未來 12 到 24 個月內，我們將尋求首​​先將去風險的後期或商業資產引入投資組合。

And then, call it, 24 months and beyond, when we're really generating strong positive free cash flows, I think we'll have an opportunity to also begin investing in the pipeline again. Jeff, do you want to talk a little bit about our hope for what might come out of our internal pipeline?

然後，24 個月或更久以後，當我們真正產生強勁的正自由現金流時，我認為我們將有機會再次開始投資管道。傑夫，你能談談我們對內部管道可能產生的成果的期望嗎？

Yes. Thanks, Brad, and thanks, Eliana, for the question. In terms of our internal research efforts, we've developed some really exciting transgenes for Fabry and Pompe disease with proteins that are much more active, potent, stable than the wild-type proteins.

是的。謝謝布拉德和伊利亞娜提出這個問題。就我們的內部研究工作而言，我們為法布瑞氏症和龐貝氏症開發了一些非常令人興奮的轉基因，其中的蛋白質比野生型蛋白質更活躍、更有效、更穩定。

And as you know, we've been looking to deliver those through various approaches. Our original focus was AAV with DNA constructs. So that still is our lead area that we're looking, but we're also looking at other ways to deliver those proteins or transgenes, whether that be through lipid nanoparticles or other approaches.

正如您所知，我們一直在尋求透過各種方法實現這一目標。我們最初的重點是帶有 DNA 構建體的 AAV。所以這仍然是我們正在研究的主要領域，但我們也在研究傳遞這些蛋白質或基因改造的其他方法，無論是透過脂質奈米顆粒還是其他方法。

So focus there is absolutely Fabry is the main focus as well as continuing to look at Pompe secondarily. But we think there's a real opportunity for next-generation Fabry treatment, especially for people that are not amenable to Fabry that currently are on enzyme replacement therapy.

因此，焦點絕對是法布里古銅礦是主要焦點，同時繼續關注龐貝古銅礦。但我們認為下一代法布瑞氏症治療確實存在機會，特別是對目前正在接受酵素替代療法的不適合法布里氏症治療的患者。

In terms of the future, I think maybe I'll turn it over to Sebastian to talk about BD and how that might influence what we're doing on the internal pipeline. I will say we pay close attention to every single product medicine approach out there for Fabry and Pompe. So that is something that we're obviously very keen on, but we also have a broader approach of disease areas of interest. So Sebastian, I'll turn it over to you.

就未來而言，我想也許我會把它交給塞巴斯蒂安來談論 BD 以及它可能如何影響我們在內部管道上所做的事情。我想說的是，我們密切注意針對法布瑞氏症和龐貝氏症的每一種產品藥物方法。因此，這顯然是我們非常熱衷的事情，但我們對感興趣的疾病領域也有更廣泛的研究方法。那麼塞巴斯蒂安，我將把它交給你。

Yes. I think we've developed a framework looking at specific therapeutic areas and indications of interest, we're looking at adjacencies. We're looking at rare renal, rare cardio, rare metabolic but also rare neuromuscular, rare neurology, broad therapeutic areas.

是的。我認為我們已經開發了一個框架來研究特定的治療領域和感興趣的適應症，我們正在研究鄰接關係。我們正在研究罕見的腎臟疾病、罕見的心臟疾病、罕見的代謝疾病，以及罕見的神經肌肉疾病、罕見的神經病學疾病等廣泛的治療領域。

Joe Schwartz, Leerink Partners.

Leerink Partners 的 Joe Schwartz。

It looks like around 17 patients were added from the end of July to the end of October versus around 30% in prior quarters. So I'm wondering what is driving this and how things are trending in the fourth quarter? And how should we be thinking about the cadence of new patient adds as we get into '25?

從 7 月底到 10 月底，似乎增加了約 17 名患者，而前幾季則是 30%。所以我想知道是什麼推動了這一趨勢以及第四季的情況如何？當我們進入25年時，我們該如何考慮新病人增加的節奏？

Have you started to think about expectations for '25? It looks like the Street assumes sales of around $145 million, and that's about a doubling of sales year over year. So what do you make of the activity in terms of patient demand this quarter and going forward?

你已經開始思考對 25 年的期望了嗎？華爾街預計銷售額約為 1.45 億美元，這意味著銷售額將年增約一倍。那麼您如何看待本季及未來的患者需求活動？

Yes. Thanks, Joe. So a few things. So we did a very strong Q3, which is great from a revenue perspective. I think that if you look at the top line number of new prescriptions, I think your numbers you quoted that right. I would also call you to the number of patients who have been treated, which is up to 196. So I think part of that is we're closing the gap on the insurance process, which Sebastian talked to earlier.

是的。謝謝，喬。有幾件事。因此，我們的第三季業績非常強勁，從營收角度來看，這是非常好的。我認為，如果你看一下新處方的首行數字，我認為你引用的數字是正確的。我也會告訴您目前接受治療的患者人數，目前已達 196 人。因此我認為部分原因是我們正在縮小保險流程上的差距，正如塞巴斯蒂安之前談到的那樣。

As we think about what happened in Q3, a few things. Q2, you had both the Spanish launch and you're still switching a number of clinical trial patients. And so Q3 was really just the ongoing launch in the various countries that are out there.

當我們思考第三季發生的事情時，有幾件事。Q2，你們已經在西班牙推出了該產品，並且仍在轉換一些臨床試驗患者。因此，Q3 實際上只是在各國正在進行的發布。

We'll have more of that in Q4. We don't have any new launch countries expected, although we do expect to get to some reimbursement outcomes, which would lead to launches going into next year. In terms of next year, of course, it depends on the rate of patients that we finished this year with. It also depends on the launch sequence that Sebastian talked to.

我們將在第四季度推出更多此類產品。我們預計不會有任何新的發射國家，儘管我們確實希望獲得一些報銷結果，這將導致發射在明年進行。當然，就明年而言，這取決於我們今年的患者數量。這也取決於塞巴斯蒂安談到的發射順序。

We have a number of countries that are coming on in the back half of next year. We also have ongoing reimbursement discussions. And while those are playing out over a six- to nine-month period, of course, the goal is speed to launch but you also want to make sure that you have a sustainable profit.

我們有許多國家將在明年下半年加入。我們還在進行報銷討論。雖然這些工作需要在六到九個月的時間內完成，但當然，目標是快速推出，但你也要確保獲得可持續的利潤。

So we'll know a lot more about that as we get to the end of the year and then we get more clarity on what next year looks like, but super pleased with the launch so far and very excited to be raising guidance for this year.

因此，到今年年底時，我們將對此有更多的了解，然後我們會更清楚地了解明年的情況，但對於迄今為止的發布會感到非常滿意，並且非常高興能夠提高今年的指導。

Dae Gon Ha, Stifel.

大昆河，Stifel。

And congrats on the progress. I wanted to circle back on the BD side of the story. Now that we're done with the election, there's a lot of chatter around STC and who might come up next. So on the BD front, when you think about these late-stage pre-commercial assets, I guess what's the driver here ultimately?

並祝賀你的進展。我想重新回顧一下 BD 方面的故事。現在選舉已經結束，人們紛紛談論 STC 以及誰將成為下一個選舉人。因此在 BD 方面，當您考慮這些後期商業前資產時，我猜最終的驅動因素是什麼？

Are you more focused on getting to GAAP profitability and a significant cash flow before pulling the trigger? Or is there something that's on your radar already that you might think about pulling the trigger once you get over the hurdle of the non-GAAP profitability?

在採取行動之前，您是否更注重實現 GAAP 獲利能力和可觀的現金流？或者，是否有一些事情已經進入您的視線，一旦您克服了非 GAAP 盈利能力的障礙，您就會考慮採取行動？

And just a clarification for Jeff. On the pipeline, these genetic approaches, are these the same ones that you guys had for Caritas that you guys are retaining? Or are these evolved versions that are separate from what we have seen from Caritas?

只是想向傑夫澄清一下。在管道上，這些基因方法是否與你們為明愛所保留的方法相同？或者這些是與我們從 Caritas 看到的不同的進化版本？

Yes. Dae Gon, thanks a lot for the question. And from an objective perspective, I think the point is that we've built an infrastructure at Amicus that now is self-sustainable based on our current business, which was part of the goal. And therefore, we think we can add to that by bringing in products that are derisked and/or already launched.

是的。Dae Gon，非常感謝您的提問。從客觀角度來看，我認為重點在於我們在 Amicus 建立了一個基礎設施，現在可以根據我們目前的業務實現自我永續發展，這也是目標的一部分。因此，我們認為我們可以透過引入風險降低和/或已經推出的產品來增強這項功能。

So we really focus on top line growth while maintaining, I think, our financial discipline around the bottom line. So I think that's the way we think about the focusing on later-stage products first. And then as I mentioned, as we go along over the next few years -- and you can think of the sequence of non-GAAP profitability to GAAP profitability, to deposit free cash flows playing out over that period of time -- I think then we'll be able to have our own resources to really start funding a pipeline as well.

因此，我們真正關注的是營收成長，同時，我認為，我們也保持了圍繞獲利的財務紀律。所以我認為這就是我們首先關注後期產品的想法。然後正如我所提到的，隨著我們在接下來的幾年裡前進 - 你可以想到非 GAAP 盈利能力到 GAAP 盈利能力的順序，到那段時間內存入的自由現金流 - 我認為我們將能夠擁有自己的資源來真正開始資助管道。

So that's generally how we think about the financial component to it. And Jeff, maybe just clarify what specifically we're focused on with our current technologies.

這就是我們通常對其中財務成分的看法。傑夫，也許可以澄清一下我們目前的技術具體關注的是什麼。

Yes. So in terms of the internal work we're doing, it is similar to the constructs we worked on previously as part of Amicus and then what was going to go into Caritas. We've even -- as we've been focusing R&D resources and efforts on our core businesses in Galafold and PomOp, we have refined those constructs, made them even incrementally better with some new AI technologies.

是的。因此，就我們所做的內部工作而言，它與我們之前作為 Amicus 的一部分開展的工作以及隨後進入 Caritas 的工作類似。我們甚至——因為我們一直將研發資源和精力集中在 Galafold 和 PomOp 的核心業務上，所以我們改進了這些結構，並透過一些新的人工智慧技術使它們逐漸變得更好。

And we've also been looking at alternative ways to deliver them. I'd say the lead approach is still an AAV with one of those constructs in Fabry, but still early and most resources still focused on our later-stage programs.

我們也一直在尋找其他的方式來實現這些目標。我想說，領先的方法仍然是 AAV，其中一個結構是 Fabry，但仍處於早期階段，大多數資源仍然集中在我們的後期專案上。

Yes. We're -- we took the decision, of course, when we moved away from gene therapy to really, really slim down those efforts and focus on the core commercial business, which we're delivering on. Again, our goal would be in the next year or two to generate some important proof-of-concept data that might allow us then to move closer to the clinic and again, as Jeff said, focused primarily on Fabry Pompe and Fabry in particular.

是的。當然，當我們放棄基因療法時，我們做出了決定，真正減少這些努力並專注於我們正在實現的核心商業業務。再次強調，我們的目標是在未來一兩年內產生一些重要的概念驗證數據，這些數據可能讓我們更接近臨床，正如傑夫所說，主要關注法布瑞龐貝氏症和法布瑞氏症。

Because of course, we can't treat about two-thirds to half of the population who don't have amenable mutations with Galafold. So that could be an exciting approach going forward. But again, that is all low priority right now, and we're just moving things along behind the scenes until we can fund them in a more significant way.

因為當然，我們無法用 Galafold 治療大約三分之二到一半沒有發生可適應突變的人口。所以這可能是一種令人興奮的未來方法。但再說一遍，這些現在都不是優先考慮的事情，我們只是在幕後推動事情發展，直到我們能夠以更重要的方式為其提供資金。

Dennis Ding, Jefferies.

傑富瑞 (Jefferies) 的丹尼斯丁 (Dennis Ding)。

I had a question around Pompe. And your new 2024 guidance assumes Q4 would be generally flattish for Pombiliti. I just wondering how much conservatism is baked into this guidance and if there's anything normal worth calling out in terms of inventory or timing of drug shipments that may have benefited Q3?

我有一個關於龐貝古城的問題。而您對 2024 年的新指引假設龐比利蒂的第四季業績將整體持平。我只是想知道該指引包含多少保守主義，以及在庫存或藥品發貨時間方面是否有任何正常情況值得注意，可能對第三季度有益？

Yes. Thanks for the question, Dennis. Again, there is some incremental growth baked in there. The question is how much new patients do we add over the course of the quarter. There aren't any new launches this quarter.

是的。謝謝你的提問，丹尼斯。再次，其中存在一些增量增長。問題是我們在本季增加了多少新患者。本季沒有新產品發布。

Again, we think there's going to be some reimbursement breakthroughs. We did see (inaudible) as an example, get to reimbursement recently, although that those patients won't come on until next year. So it really just depends on the rate of new patient adds as we go through the course of the quarter, and that will tell us where we are within that range. But there is some incremental growth baked into that guidance.

再次，我們認為報銷方面將會取得一些突破。我們確實看到（聽不清楚）例如最近獲得了報銷，儘管那些病人要到明年才會來。因此，這實際上僅取決於我們在整個季度中增加的新患者的比率，這將告訴我們我們處於該範圍內的位置。但該指引中也包含了一些增量成長。

Got it. And maybe as a follow-up, as you think about the trajectory of the Pompe launch, this -- like is the messaging more around steady study and linear growth? Or do you see any point of acceleration in terms of new patient adds?

知道了。或許作為後續問題，當您思考龐貝發射的軌跡時，這個——資訊是否更多地圍繞著穩定的研究和線性增長？或者您看到新患者增加方面的任何加速點？

Yes. Thanks for the question. Again, we don't give guidance yet, but I do think a couple of factors will determine how that plays out. So first of all, you've got the rate of new patient starts in the existing markets. And we do think in the US, as an example, you're going to have a fairly sizable number of patients who are now moving into that one- to two-year zone next year.

是的。謝謝你的提問。再說一次，我們還沒有給予指導，但我確實認為有幾個因素將決定結果如何。首先，您要了解現有市場中新患者的開始率。以美國為例，我們確實認為，明年將會有相當多的患者進入一到兩年的治療期。

So it could be a great opportunity to start to switch those patients. And I mean patients who've been on Nexviazyme for a year or two. And then you also have the rate of new countries launching. And that's in part dictated by the reimbursement process, which we already talked about, and then part dictated by the regulatory process.

所以這可能是開始轉換這些病人的一個好機會。我指的是使用 Nexviazyme 一兩年的患者。然後你還可以了解新國家推出的速度。這在一定程度上取決於我們已經討論過的報銷流程，部分取決於監管流程。

As an example, some of the submissions we've made, Australia, Canada, and Japan, we think will come online in the back half of next year. So still some moving pieces. I do think over time, you will see -- you'll start to see momentum build as you have more markets and the ability to draw for more patients. But again, we'll see how this -- the rest of this year plays out and more color on the rate of those new launches as we go into the next year.

例如，我們提交的一些澳大利亞、加拿大和日本的提案我們認為將在明年下半年上線。因此仍有一些活動的部分。我確實認為，隨著時間的推移，你會看到——隨著你擁有更多的市場和吸引更多患者的能力，你將開始看到發展勢頭的增強。但是，我們還是要再看看今年剩餘時間的情況，並觀察進入明年後這些新產品的推出速度。

Kristen Kluska, Cantor Fitzgerald.

克里斯汀·克魯斯卡 (Kristen Kluska)、菲茨杰拉德 (Fitzgerald) 領唱。

Congrats on a great quarter, not just from the revenue side, but from all the operational updates as well. I was hoping you can comment more on some of these main drivers that have led to some of the tightening of the operating expense guidance you're providing today?

恭喜您度過了一個美好的季度，不僅從收入方面，而且從所有的營運更新方面。我希望您能對導致您今天提供的營業費用指引收緊的一些主要驅動因素發表更多評論？

Thanks, Kristen. Simon, do you want to take that?

謝謝，克里斯汀。西蒙，你想拿著這個嗎？

Yes. Really, I think it's fair to say that we continue to manage our operating expense, as we said, we wanted to do extremely tightly year on year. So we have really limited, I would say, headcount additions because we have the infrastructure that we've talked about in place that was built to support Fabry, and we didn't need to add significant additional numbers as we launched Pombiliti and Opfolda.

是的。確實，我認為可以公平地說，我們會繼續管理我們的營運費用，正如我們所說的那樣，我們希望逐年嚴格控制。因此，我想說，我們確實限制了員工人數的增加，因為我們已經建立了為支持 Fabry 而建造的基礎設施，而且在推出 Pombiliti 和 Opfolda 時，我們不需要增加大量人員。

I would say we continue, however, to deliver importantly on all the things in the OpEx that we need to do from an activities perspective, such as some of the registry studies, the adolescent LOPD, IOPD, and some of the remaining expenses related to open-label extension. So all of that is taking place from an activity perspective, but we look at all opportunities really to work with the overall management team and employees to ensure that we're managing OpEx tightly.

不過我想說，從活動角度來看，我們將繼續專注於完成 OpEx 中我們需要做的所有事情，例如一些註冊研究、青少年 LOPD、IOPD，以及一些與開放標籤擴展相關的剩餘費用。因此，所有這些都是從活動角度進行的，但我們會尋找所有機會與整體管理團隊和員工合作，以確保我們嚴格管理營運支出。

And that's what you're seeing as a result of the lowering of that non-GAAP operating expense guidance, which obviously is also helping the bottom line. Because as you know, we've talked about full year non-GAAP profitability in reality as of the first nine months of this year, we'd already achieved $45 million of non-GAAP profitability. So that's what's going on currently.

這就是非公認會計準則營運費用指引下調的結果，這顯然也有助於提高獲利水準。因為如你所知，我們已經談論了全年非 GAAP 盈利能力，實際上截至今年前九個月，我們已經實現了 4500 萬美元的非 GAAP 盈利能力。這就是目前發生的情況。

Jeffrey Hung, Morgan Stanley.

摩根士丹利的 Jeffrey Hung。

This is Michael Riad on for Jeff. Congrats on the strong quarter. How far into 2025 -- for like the Pombiliti and Opfolda previously expensed inventory, how far would that like that inventory be able to carry for next year? And like, can you provide any commentary on the potential for next-gen manufacturing? When would you expect that could start to impact gross margins?

這是邁克爾·裡亞德 (Michael Riad)，代表傑夫。恭喜本季業績強勁。到 2025 年還能維持多久——對於像 Pombiliti 和 Opfolda 之前已計入費用的庫存來說，這些庫存到明年還能維持多久？您能對下一代製造業的潛力發表什麼評論嗎？您預計什麼時候會開始影響毛利率？

I'll take the second one first, and then Simon, I'll have you talk to the first one. So next-gen manufacturing is something we're investing in, and we've talked about that before. We think we can reduce cost of goods by maybe 20% to 30%, although we're still working through that process.

我先談第二個，然後西蒙，我讓你談第一個。我們正在投資下一代製造業，我們之前已經討論過這件事了。我們認為我們可以將商品成本降低 20% 到 30%，儘管我們仍在努力實現這一目標。

That's probably a back half of the decade impact. So we haven't given formal guidance there, but that's still a few years out before that starts to hit the commercial supply chain and it has a real impact that maybe, Simon, talk to the near-term drivers next year.

這可能是過去五年的影響。因此，我們還沒有給出正式的指導，但這還需要幾年的時間才能開始影響商業供應鏈，並產生真正的影響，西蒙，也許明年會談到近期的驅動因素。

Yes. I mean in terms of the heads up on COGS in the the mid- or should I say gross margin in the mid-80% range next year, that's really driven by the fact that, that expensed inventory will run out in the earlier part of next year. And therefore, that's why we call it a hybrid year.

是的。我的意思是，就明年中期銷貨成本（或應該說毛利率）在 80% 左右的範圍內而言，這實際上是由於費用化庫存將在明年年初耗盡這一事實所致。因此，我們稱之為混合年。

We will see, as we've said previously, full year impact of Pombiliti and Opfolda gross margin rolling through in 2026. But we wanted to at least make you aware of what we see at this point in time has a difference compared to 2024 in 2025 and hence, that statement.

正如我們之前所說，我們將看到 Pombiliti 和 Opfolda 毛利率的全年影響將持續到 2026 年。但我們至少想讓你知道，我們此時所看到的情況與 2024 年和 2025 年相比有所不同，因此才有這樣的聲明。

Our next question comes from Gil Blum of Needham & Company.

下一個問題來自 Needham & Company 的 Gil Blum。

So as it relates to genetic medicine and Fabry, recent feedback from the FDA regarding potential for accelerated approval was pretty interesting. I just wonder what you guys think and whether current dynamics could push a program for genetic medicine in Fabry a little faster than expected.

因此，就基因醫學和法布瑞氏症而言，FDA 最近關於加速核准可能性的回饋非常有趣。我只是想知道你們的想法，以及當前的動態是否能比預期更快地推動法布里遺傳醫學計劃。

Yes, it's a good question. I think as it relates to the other therapies that are out there in development. We really see those as impacting patients with nonamenable mutations just based on the data that we've seen. And so we're really excited about Galafold continue to grow for amenable patients, but we would love to have something for that other part of the population.

是的，這是個好問題。我認為它與正在開發的其​​他療法有關。根據我們所看到的數據，我們確實看到這些會對患有無法治癒的突變的患者產生影響。因此，我們真的很高興 Galafold 能夠繼續為適應的患者服務，但我們也希望為另一部分人群提供一些幫助。

From a regulatory perspective, I think broadly, you're seeing some flexibility in the part of the agency, which we think is exciting. Hard to know what's going to happen with the other therapies, we just read the same thing as you do.

從監管角度來看，我認為總體而言，您會看到機構部分具有一定的靈活性，我們認為這是令人興奮的。很難知道其他療法會產生什麼效果，我們只是讀到了和您一樣的內容。

But again, I think if any of those are able to get across the finish line, they're almost certainly for nonamenable patients, and so eager to see if we can do something on our own in the coming years to address that population as well.

但是，我認為，如果其中任何一個能夠跨越終點線，那麼幾乎肯定是針對不適合治療的患者，因此我們迫切希望看到我們是否能在未來幾年內自行採取一些措施來解決這一群體的問題。

Salveen Richter, Goldman Sachs.

高盛的薩爾文·里希特（Salveen Richter）。

Just would it be possible to give us some further commentary on the competitive dynamics versus Nexviazyme in the US and ex US, just given the appreciable growth in the ex-US share for that asset for Sanofi's last earnings.

鑑於賽諾菲上一季收益中該資產在美國以外地區的份額顯著增長，您能否就其在美國及美國以外地區的競爭態勢給我們提供一些進一步的評論？

Yes. Sebastian, maybe talk a little bit to the proportionality in terms of how we're getting patients to switch to Pombiliti and Opfolda and what we're seeing as things evolve in those two markets.

是的。賽巴斯蒂安，也許可以談談我們如何讓患者轉向龐比利蒂和奧普福德的比例，以及我們看到的這兩個市場的發展。

Thanks, Brad. Salveen, for the question. So as I mentioned earlier, what we're seeing in the US is in terms of switch dynamics is pretty much a reflection of the current respective shares of Lumizyme and Nexviazyme in the US.

謝謝，布拉德。回答這個問題的是 Salveen。正如我之前提到的，我們在美國看到的轉換動態基本上反映了 Lumizyme 和 Nexviazyme 在美國目前的份額。

So at this point, at least based on Q3 reported numbers, Nexviazyme accounts for about 60% of Sanofi sales in the US, and there's still 40% from Lumizyme. I did say that we've got slightly more than two-thirds of our switches coming from Nexviazyme, which is a great indication that, as we said all along, that there's potential for us to switch a large proportion of Nexviazyme patients over time.

因此目前，至少根據第三季的報告數據，Nexviazyme 佔賽諾菲在美國銷售額的 60% 左右，而 Lumizyme 仍佔 40%。我確實說過，我們有略多於三分之二的轉換來自 Nexviazyme，這是一個很好的跡象，表明正如我們一直所說的那樣，隨著時間的推移，我們有可能轉換很大一部分 Nexviazyme 患者。

We're seeing also that ex US, it's actually the reverse situation. So there is still more Myozyme than Nexviazyme so roughly 60% Myozyme ex US and 40% Nexviazyme. And so here, we tend to see a greater proportion of switches coming from Myozyme. You wouldn't be surprised. We also have the benefit of seeing some naive patients moving straight on to Pombiliti and Opfolda.

我們也看到，在美國以外，情況其實恰恰相反。因此，Myozyme 仍然比 Nexviazyme 多，所以美國以外地區 Myozyme 大約佔 60%，Nexviazyme 佔 40%。因此在這裡，我們傾向於看到更大比例的轉換來自 Myozyme。你不會感到驚訝。我們也很高興地看到一些天真的病人直接轉向龐比利蒂和奧普福德。

Thank you. That was our last question. I would now like to pass it to Bradley for his closing remarks.

謝謝。這是我們的最後一個問題。現在我想將發言權交給布萊德利，請他作最後發言。

Great. Thanks, everybody. Have a great day, and thanks for listening to the conference call.

偉大的。謝謝大家。祝您有愉快的一天，感謝您收聽電話會議。

Thank you for your participation in today's conference. This does conclude the program. You may now disconnect.

感謝大家參加今天的會議。該計劃確實結束了。您現在可以斷開連線。