Catalyst Pharmaceuticals Inc (CPRX) 2024 Q3 法說會逐字稿

Thank you for standing by. My name is Chris and I will be your conference operator today. At this time, I would like to welcome everyone to the Catalyst Pharmaceuticals third-quarter 2024 financial results conference call. (Operator Instructions) Thank you.

謝謝你的支持。我叫克里斯，今天我將擔任你們的會議操作員。此時此刻，我歡迎大家參加 Catalyst Pharmaceuticals 2024 年第三季財務業績電話會議。（操作員說明）謝謝。

I would now like to turn the call over to Mike Kalb, CFO. Please go ahead.

我現在想將電話轉給財務長 Mike Kalb。請繼續。

Thank you. Good morning, everyone, and thank you for joining our conference call to discuss Catalyst Pharmaceuticals third-quarter 2024 financial results and business highlights. Rich Daly, President and CEO, will lead the call today; and Jeff Del Carmen, our Chief Commercial Officer, and I will also present. Additionally, Dr. Steve Miller, our Chief Operating Officer and Chief Scientific Officer, will be available for the Q&A.

謝謝。大家早安，感謝您參加我們的電話會議，討論 Catalyst Pharmaceuticals 2024 年第三季財務業績和業務亮點。總裁兼執行長 Rich Daly 將主持今天的電話會議；我和我們的商務長 Jeff Del Carmen 也將出席。此外，我們的營運長兼首席科學官 Steve Miller 博士也將出席問答環節。

Before we begin, I would like to remind you that in our remarks this morning and in the Q&A session, we will make statements about expected future results, which may be forward-looking statements for purposes of federal securities laws.

在開始之前，我想提醒您，在今天早上的演講和問答環節中，我們將做出有關預期未來結果的聲明，這些聲明可能是聯邦證券法規定的前瞻性聲明。

These statements relate to our current expectations, estimates, and projections and are not guarantees of future performance. They involve risks, uncertainties, and assumptions that are difficult to predict and may not prove to be accurate. Actual results may vary from the expectations contained in our forward-looking statements.

這些陳述與我們目前的預期、估計和預測有關，並非對未來績效的保證。它們涉及難以預測且可能不準確的風險、不確定性和假設。實際結果可能與我們前瞻性陳述中的預期有所不同。

These forward-looking statements should be considered only in conjunction with the detailed information contained in our SEC filings, including the risk factors described in our 2023 annual report on Form 10-K filed with the SEC on February 28, 2024, and in our third-quarter 2024 quarterly report on Form 10-Q, which was filed yesterday, November 6, 2024, with the SEC.

這些前瞻性陳述應僅與我們向SEC 提交的文件中包含的詳細資訊結合考慮，包括我們於2024 年2 月28 日向SEC 提交的10-K 表格2023 年年度報告以及我們的第三份報告中描述的風險因素。

At this time, I will turn the call over to Rich. Rich?

這時，我會將電話轉給 Rich。富有的？

Thanks, Mike. Good morning, everyone, and thanks for joining us today. We are pleased to report that we achieved another quarter of exceptional financial performance, continuing our consistent growth and sustained momentum. Our results for the third quarter 2024 demonstrate our unwavering commitment to operational excellence, strategic execution, and delivering long-term value.

謝謝，麥克。大家早安，感謝您今天加入我們。我們很高興地報告，我們又一個季度取得了出色的財務業績，繼續了我們的持續成長和持續勢頭。我們 2024 年第三季的業績證明了我們對卓越營運、策略執行和交付長期價值的堅定承諾。

Today's call will begin with an overview of our portfolio highlights and key strategic initiatives. We'll then move on to our detailed discussion of our commercial performance and financial results for the quarter.

今天的電話會議將首先概述我們的投資組合亮點和關鍵策略舉措。然後，我們將繼續詳細討論本季的商業業績和財務業績。

Let's start with financial results and commercial highlights. In Q3 2024, we reported total revenues of $128.7 million, a 25.3% increase year over year, which included net product revenues of $126.4 million, a 23.2% increase from Q3 2023. These results were driven by the outstanding performance of FIRDAPSE and the continued launch success of AGAMREE.

讓我們從財務業績和商業亮點開始。2024 年第三季度，我們的總營收為 1.287 億美元，年增 25.3%，其中產品淨收入為 1.264 億美元，比 2023 年第三季成長 23.2%。這些成果得益於 FIRDAPSE 的出色表現和 AGAMREE 的持續成功推出。

I'm pleased to report that as a result of our strong performance over the first three quarters of the year, we are updating our full-year 2024 total revenue guidance to between $475 million and $485 million. FIRDAPSE continues to deliver outstanding net revenues and growth well into its sixth year on the market, and we are updating our full-year 2024 net revenue guidance for FIRDAPSE from the $295 million to $310 million range to the $300 million to $310 million range.

我很高興地向大家報告，由於我們今年前三個季度的強勁表現，我們將 2024 年全年總收入指引更新為 4.75 億美元至 4.85 億美元之間。FIRDAPSE 進入市場第六年，繼續保持出色的淨收入和成長，我們正在將 FIRDAPSE 的 2024 年全年淨收入指引從 2.95 億美元至 3.1 億美元範圍更新為 3 億美元至 3.1 億美元範圍。

Our ability to work with health care providers to identify and maintain an active list of potential patients has been foundational to our success, and we believe that we will -- this will continue to drive our growth for the foreseeable future.

我們與醫療保健提供者合作確定和維護潛在患者的活躍名單的能力是我們成功的基礎，我們相信，這將在可預見的未來繼續推動我們的成長。

Additionally, as a result of AGAMREE's market success, we are updating our full year 2024 net revenue projections for AGAMREE from the $35 million to $40 million range to the $40 million to $45 million range. We are confident in AGAMREE's market potential as it continues to gain traction within the DMD community. We believe AGAMREE represents a significant advancement in corticosteroid treatment options for DMD and is well positioned to drive meaningful long-term growth.

此外，由於 AGAMREE 的市場成功，我們將 AGAMREE 2024 年全年淨收入預測從 3,500 萬美元至 4,000 萬美元範圍更新為 4,000 萬美元至 4,500 萬美元範圍。我們對 AGAMREE 的市場潛力充滿信心，因為它在 DMD 社群中持續受到關注。我們相信 AGAMREE 代表了 DMD 皮質類固醇治療方案的重大進步，並且有能力推動有意義的長期成長。

FYCOMPA continues to deliver steady prescription numbers and generated net revenues of $32.1 million in the third quarter. The brand experienced a 12% decrease in net revenues compared to Q3 2023 due to adjustments in gross to net as previously disclosed.

FYCOMPA 繼續提供穩定的處方數量，並在第三季創造了 3,210 萬美元的淨收入。由於先前揭露的總收入與淨收入的調整，與 2023 年第三季相比，該品牌的淨收入下降了 12%。

FYCOMPA remains on track to meet full-year 2024 guidance. Jeff will offer a comprehensive overview of our commercial performance, followed by Mike's detailed review of our financials later in today's call.

FYCOMPA 仍有望達到 2024 年全年指導目標。傑夫將全面概述我們的商業業績，隨後麥克將在今天的電話會議中詳細審查我們的財務狀況。

Let's take a broader view of our Q3 accomplishments, starting with clinical and regulatory. Alongside our financial achievements, we are making strides on the clinical front with the SUMMIT study. This study aims to gather real-world data to validate AGAMREE's clinical benefits, including improvements in bone health, growth parameters, behavioral, and cardiovascular outcomes.

讓我們從臨床和監管開始，更廣泛地了解第三季的成就。除了我們所取得的財務成就外，我們還透過 SUMMIT 研究在臨床前沿取得了長足的進步。這項研究旨在收集真實世界的數據來驗證 AGAMREE 的臨床益處，包括改善骨骼健康、生長參數、行為和心血管結果。

Patient enrollment now underway, we expect this study to provide long-term safety and quality of life data that we hope will support future FDA submissions for updated AGAMREE labeling aimed at demonstrating its advantages over existing corticosteroid therapies and establishing it as the leading treatment option for DMD.

患者入組正在進行中，我們預計這項研究將提供長期安全性和生活品質數據，我們希望這些數據將支持未來FDA 提交更新的AGAMREE 標籤，旨在證明其相對於現有皮質類固醇療法的優勢，並將其確立為領先的治療選擇DMD。

Moving on to global expansion milestones. In parallel, we continue to make progress on our global expansion efforts, aiming to broaden our rare disease portfolio through strategic alliances in key markets outside the US. Recent milestones in Japan and Canada highlight our commitment to delivering innovative therapies to rare patients worldwide.

邁向全球擴張里程碑。同時，我們繼續在全球擴張方面取得進展，旨在透過在美國以外的主要市場建立策略聯盟來擴大我們的罕見疾病產品組合。日本和加拿大最近的里程碑凸顯了我們為全球罕見患者提供創新療法的承諾。

In late September, DyDo Pharma, our sub-licensee in Japan, secured regulatory approval for FIRDAPSE 10-milligram tablets for the treatment of LEMS, paving the way for the launch of FIRDAPSE in Japan, which they have advised us is expected by the end of Q4 2024. Additionally, this achievement resulted in a milestone payment of approximately $2.1 million.

9 月下旬，我們在日本的分授權商 DyDo Pharma 獲得了用於治療 LEMS 的 FIRDAPSE 10 毫克片劑的監管批准，為 FIRDAPSE 在日本的上市鋪平了道路，他們告訴我們預計將於年底上市2024 年第四季。此外，這項成就還帶來了約 210 萬美元的里程碑付款。

Our partner in Canada, Kye Pharmaceuticals, is working diligently to file for AGAMREE's regulatory approval. This milestone will mark another advancement in expanding our rare disease portfolio, paving the way for health care providers and patients in Canada to access this therapy.

我們在加拿大的合作夥伴 Kye Pharmaceuticals 正在努力申請 AGAMREE 的監管批准。這一里程碑將標誌著我們在擴大罕見疾病產品組合方面的又一進步，為加拿大的醫療保健提供者和患者獲得這種療法鋪平了道路。

While no agreement has been entered into to date, we remain focused on establishing strategic partnerships in LatAm and APAC, both high-growth regions to enhance market access and drive expansion. Regarding our strategic initiatives, our efforts remain strong with our business development teams actively evaluating a range of promising opportunities in the rare disease space.

儘管迄今尚未達成任何協議，但我們仍專注於在拉丁美洲和亞太地區這兩個高成長地區建立戰略合作夥伴關係，以加強市場准入並推動擴張。關於我們的策略舉措，我們的業務發展團隊積極評估罕見疾病領域的一系列有前途的機會，我們的努力仍然強勁。

Our strategy is focused on accretive and near-accretive opportunities, predominantly in later-stage derisked assets with a clear path to maximizing value. Our teams have been actively searching for and pursuing strategic opportunities, and we have also seen a notable increase in inbound opportunities.

我們的策略著重於增值和接近增值的機會，主要是後期去風險資產，具有明確的價值最大化路徑。我們的團隊一直在積極尋找和追求策略機會，我們也看到入境機會顯著增加。

This rise in inbound engagement is a strong signal of the market's confidence in our commercial track record, credibility, and industry reputation. This momentum positions us well to drive value through strategic partnerships and growth initiatives, aligning with our long-term commitment to deliver sustained value.

入境參與度的上升強烈表明了市場對我們的商業記錄、信譽和行業聲譽的信心。這種勢頭使我們能夠透過策略合作夥伴關係和成長計劃來推動價值，這與我們提供持續價值的長期承諾一致。

In conclusion, as we continue to diligently execute our threefold strategy: first, outstanding commercial execution; second, portfolio diversification; and third, global expansion, we believe we are well positioned to capitalize on emerging opportunities, sustain our growth trajectory and drive long-term success.

總之，我們繼續努力執行我們的三重策略：第一，卓越的商業執行力；第二，投資組合多元化；第三，在全球擴張方面，我們相信我們處於有利地位，能夠利用新興機遇，維持我們的成長軌跡並推動長期成功。

At this time, I'll turn the call over to Jeff, who will provide an overview of our commercial performance. Jeff?

此時，我會將電話轉給傑夫，他將概述我們的商業表現。傑夫？

Thanks, Rich, and good morning, everyone. We are thrilled to announce the outstanding third quarter performance of our entire commercial portfolio. These results highlight our strategic focus and operational excellence, which continue to fuel our commercial success.

謝謝里奇，大家早安。我們很高興地宣布我們整個商業產品組合第三季的出色表現。這些結果凸顯了我們的策略重點和卓越運營，這將繼續推動我們的商業成功。

Q3 results were propelled by sustained organic growth in FIRDAPSE, steady prescriptions from FYCOMPA and the continued robust commercial launch of AGAMREE. Total net product revenues for Q3 reached $126.4 million, marking a 23.2% increase compared to the same quarter last year.

第三季的業績得益於 FIRDAPSE 的持續有機成長、FYCOMPA 的穩定處方以及 AGAMREE 的持續強勁商業推出。第三季產品淨收入總額達 1.264 億美元，較去年同期成長 23.2%。

Based primarily on the continued strong performance of FIRDAPSE and the strong performance of AGAMREE, which we launched commercially in March of this year, we have increased our full year revenue estimate to between $475 million and $485 million, which is further detailed in the press release issued last night.

主要基於 FIRDAPSE 的持續強勁表現以及我們於今年 3 月商業推出的 AGAMREE 的強勁表​​現，我們將全年收入預期提高至 4.75 億美元至 4.85 億美元之間，新聞稿中對此進行了進一步詳細介紹昨晚發出。

I will now provide a breakdown of Q3 net product revenue by product. Let's begin with discussing FIRDAPSE. Our efforts to support patients living with Lambert-Eaton myasthenic syndrome with FIRDAPSE have been remarkably successful this quarter. Q3 net sales of $79.3 million represent an almost 20% increase over the same quarter last year.

我現在將按產品提供第三季產品淨收入的明細。讓我們先討論 FIRDAPSE。本季度，我們透過 FIRDAPSE 為蘭伯特-伊頓肌無力症候群患者提供支援的努力取得了巨大成功。第三季淨銷售額為 7,930 萬美元，比去年同期成長近 20%。

New LEMS patient enrollments and net new patients both exceeded forecast, while the annual discontinuation rate was below the forecast of 20%. Prescription approval rates were greater than 90% across all payers, government and private commercial insurers.

新 LEMS 患者入組人數和淨新患者數量均超過預測，而年度停藥率低於預測的 20%。所有付款人、政府和私人商業保險公司的處方批准率均超過 90%。

Ordering patterns and refill rates during Q3 returned to historical norms of around 90%. Our demonstrated strategic growth initiatives continue to build a strong LEMS patient pipeline, ensuring steady treatment adoption and long-term organic growth.

第三季的訂購模式和補充率恢復到 90% 左右的歷史正常水平。我們已展示的策略性成長計畫繼續建立強大的 LEMS 患者管道，確保穩定的治療採用和長期有機成長。

We sustain a pipeline of approximately 500 diagnosed LEMS patients at various stages of their diagnostic journey who have not yet started on FIRDAPSE. This group generally accounts for around half of our new patient enrollments, significantly supporting our steady annual organic growth.

我們維持著大約 500 名診斷 LEMS 患者的管道，這些患者處於診斷旅程的不同階段，但尚未開始使用 FIRDAPSE。該群體通常占我們新入院患者的一半左右，極大地支持了我們穩定的年度有機成長。

Additionally, we continue to be pleased with the progress we are making with LEMS education programs aimed at health care providers and potential LEMS patients to drive high rates of voltage-gated calcium channel antibody testing, helping to shorten the diagnostic journey and increasing patient eligibility for treatment with FIRDAPSE.

此外，我們對針對醫療保健提供者和潛在LEMS 患者的LEMS 教育計劃所取得的進展仍然感到滿意，這些計劃旨在推動電壓門控鈣通道抗體測試的高比率，有助於縮短診斷過程並提高患者的資格FIRDAPSE 治療。

Finally, we continue to collaborate with top thoracic oncology institutions to implement screenings that will help accelerate diagnosis of the more than 90% of small cell lung cancer LEMS patients that remain undiagnosed. Based on leading indicators going into Q4, we are narrowing FIRDAPSE's full year 2024 guidance to between $300 million and $310 million, which is the result of us increasing the lower end of our previous guidance range.

最後，我們繼續與頂級胸部腫瘤機構合作實施篩檢，這將有助於加速對 90% 以上仍未確診的小細胞肺癌 LEMS 患者的診斷。根據進入第四季度的領先指標，我們將 FIRDAPSE 的 2024 年全年指引縮小至 3 億至 3.1 億美元之間，這是我們提高先前指導範圍下限的結果。

Next, I would like to discuss AGAMREE. We are proud of AGAMREE's growing presence in the market as a breakthrough treatment for DMD. This quarter, AGAMREE's performance exceeded our expectations with robust net sales of $15 million, driven by its expanding adoption.

接下來我想談談AGAMREE。作為 DMD 的突破性治療方法，AGAMREE 在市場上的影響力不斷擴大，我們對此感到自豪。本季度，由於採用範圍不斷擴大，AGAMREE 的業績超出了我們的預期，淨銷售額達到 1500 萬美元。

Our strategic partnerships and initiatives to educate health care providers and the patient community about AGAMREE's unique benefits have been instrumental in this success. We continue to see transitions to AGAMREE from both corticosteroid segments, 45% from EMFLAZA and 43% from prednisone.

我們的策略合作夥伴關係和旨在教育醫療保健提供者和患者社區了解 AGAMREE 獨特優勢的舉措對於這一成功發揮了重要作用。我們繼續看到兩個皮質類固醇領域都轉向 AGAMREE，其中 45% 從 EMFLAZA 轉向 AGAMREE，43% 從潑尼松轉向 AGAMREE。

In addition, 84% of the top DMD centers of excellence and greater than 170 unique health care providers have already prescribed AGAMREE. We are exceeding our target of 85% reimbursed shipments and average less than 9 business days to approval.

此外，84% 的頂級 DMD 卓越中心和超過 170 家獨特的醫療保健提供者已開出 AGAMREE 處方。我們已經超越了 85% 報銷貨件的目標，平均不到 9 個工作天即可獲得批准。

We expect the time to approval to decrease as payers finalize their policies over the next couple of months. Based on the promising demand trend and encouraging payer landscape, we are raising our AGAMREE full year 2024 net product revenue guidance to between $40 million and $45 million.

我們預計，隨著付款人在未來幾個月內最終確定其保單，批准時間將會縮短。基於前景光明的需求趨勢和令人鼓舞的支付環境，我們將 AGAMREE 2024 年全年淨產品收入指引提高至 4,000 萬美元至 4,500 萬美元之間。

Lastly, I would like to provide a brief overview of FYCOMPA. This quarter, FYCOMPA has delivered solid results, driven by steady demand, sustaining its position in the market. Q3 net revenues of $32.1 million further fortifies our revenue diversification. FYCOMPA remains firmly on course to achieve its full year 2024 net product revenue guidance of between $130 million and $135 million.

最後，我想簡單介紹一下FYCOMPA。本季度，在穩定需求的推動下，FYCOMPA 取得了穩健的業績，維持了其市場地位。第三季淨收入為 3,210 萬美元，進一步加強了我們的收入多元化。FYCOMPA 仍堅定地致力於實現 2024 年全年產品淨收入指引值 1.3 億至 1.35 億美元。

In summary, our outstanding performance in Q3 demonstrates the strength of our product portfolio and our ability to address the needs of diverse patient populations. Our strategic investments in marketing, education and patient support have been key drivers of this success, and we remain committed to building on this momentum.

總而言之，我們在第三季度的出色表現證明了我們產品組合的實力以及我們滿足不同患者群體需求的能力。我們在行銷、教育和患者支援方面的策略投資是這項成功的關鍵驅動力，我們將繼續致力於鞏固這一勢頭。

We look forward to continuing to deliver sustained commercial execution that is making a meaningful difference in the lives of patients. I want to thank the entire team at Catalyst for their unwavering commitment to patients and look forward to a successful Q4.

我們期待繼續提供持續的商業執行，為患者的生活帶來有意義的改變。我要感謝 Catalyst 的整個團隊對患者堅定不移的承諾，並期待第四季度取得成功。

I will now turn the call back over to Mike.

我現在將把電話轉回給麥克。

Thank you, Jeff. Our performance during the third quarter of 2024 has set us on pace for another strong year, driven by solid financial performance, financial discipline and strong execution. The successful launch of AGAMREE in mid-March, along with the continued performance of FIRDAPSE and FYCOMPA has strengthened our foundation for sustained growth.

謝謝你，傑夫。在穩健的財務業績、財務紀律和強有力的執行力的推動下，2024 年第三季的業績使我們有望迎來另一個強勁的一年。3月中旬AGAMREE的成功推出，加上FIRDAPSE和FYCOMPA的持續表現，夯實了我們持續成長的基礎。

Our total revenues for the third quarter of 2024 were $128.7 million, an approximate 25.3% increase when compared to total revenue of $102.7 million for the third quarter of 2023. Product revenues net for our lead product FIRDAPSE were $79.3 million, a 19.7% increase year over year compared to $66.2 million.

我們 2024 年第三季的總營收為 1.287 億美元，與 2023 年第三季的總營收 1.027 億美元相比成長約 25.3%。我們的主導產品 FIRDAPSE 的產品淨收入為 7,930 萬美元，較去年同期的 6,620 萬美元成長 19.7%。

Product revenues net for the third quarter of 2024 for FYCOMPA were $32.1 million compared to $36.4 million, an 11.9% decrease from the prior year period, driven by increased gross to net deductions, resulting from higher costs tied to arrangements between the company and distributors and government agencies.

FYCOMPA 2024 年第三季的產品淨收入為 3,210 萬美元，較上年同期的 3,640 萬美元下降 11.9%，這是由於公司與分銷商之間的安排相關的成本增加和淨扣除額增加所致。政府機構。

For 2023, revenues were booked under a size cost arrangements, which were lower. Product revenues net in the third quarter of 2024 for AGAMREE were $15.0 million, reflecting the continued strength of the commercial launch. As a reminder, Q3 2024 was the second full quarter of AGAMREE commercialization.

2023 年，收入是根據規模成本安排入帳的，該安排較低。AGAMREE 2024 年第三季的產品淨收入為 1,500 萬美元，反映出商業發布的持續強勁。提醒一下，2024 年第三季是 AGAMREE 商業化的第二個完整季度。

Additionally, included in license and other revenue is a $2.1 million milestone payment earned upon DyDo's receipt of regulatory approval to commercialize FIRDAPSE for the treatment of patients with LEMS in Japan. As previously disclosed, we are entitled to additional payments if certain milestones are achieved and a transfer price is applied to products supplied to DyDo.

此外，許可證和其他收入還包括 DyDo 獲得監管部門批准將 FIRDAPSE 商業化用於日本 LEMS 患者治療後賺取的 210 萬美元里程碑付款。如同先前所揭露的，如果實現了某些里程碑並且向 DyDo 提供的產品採用了轉讓價格，我們有權獲得額外付款。

Cost of sales expense were approximately $19.3 million in the third quarter of 2024 compared to $14.2 million in the third quarter of 2023 and consisted principally of royalties. As a reminder, royalties for FIRDAPSE increased by 3% when net product sales exceed $100 million in any calendar year, which was surpassed during the second quarter of 2024. As a result, we expect cost of sales to trend higher as the year progresses.

2024 年第三季的銷售成本費用約為 1,930 萬美元，而 2023 年第三季的銷售成本費用為 1,420 萬美元，主要包括特許權使用費。需要提醒的是，當任何日曆年的產品淨銷售額超過 1 億美元時，FIRDAPSE 的特許權使用費就會增加 3%，這一數字在 2024 年第二季就超過了這個數字。因此，我們預期隨著時間的推移，銷售成本將呈現上升趨勢。

Additionally, AGAMREE carries a royalty in the range of low double digits to mid-20s as a percent of net revenue, depending on sales achievements within a calendar year. Further details on our royalty obligations for AGAMREE are reported in our Q3 2024 Form 10-Q and are expanded upon within the MD&A section.

此外，AGAMREE 收取的特許權使用費佔淨收入的百分比在低兩位數到 20 多歲之間，具體取決於一個日曆年內的銷售業績。有關我們對 AGAMREE 的特許權使用費義務的更多詳細信息，請參見我們的 2024 年第 3 季度表格 10-Q，並在 MD&A 部分進行了詳細介紹。

Research and development expenses were $3.3 million in the third quarter of 2024, down from $83.7 million in the third quarter of 2023, which included an $81.5 million IP R&D charge. SG&A expenses for the third quarter of 2024 totaled $45.9 million compared to $33.6 million in Q3 2023.

2024 年第三季的研發費用為 330 萬美元，低於 2023 年第三季的 8,370 萬美元，其中包括 8,150 萬美元的智慧財產權研發費用。2024 年第三季的銷售管理及行政費用總計 4,590 萬美元，而 2023 年第三季為 3,360 萬美元。

The increase in SG&A expenses in the third quarter of 2024 was principally due to incremental operating expenses to support the continued commercial launch of AGAMREE. Net income before income taxes for the third quarter of 2024 were $57.2 million, an improvement year over year compared to a net loss of $38 million for the third quarter of 2023.

2024 年第三季 SG&A 費用的增加主要是因為支持 AGAMREE 持續商業推出而增加的營運費用。2024 年第三季所得稅前淨利為 5,720 萬美元，與 2023 年第三季淨虧損 3,800 萬美元相比，年比有所改善。

We reported GAAP net income for the third quarter of 2024 of $43.9 million or $0.35 per diluted share compared to GAAP net loss for the third quarter of 2023 of $30.8 million or $0.29 per diluted share. This increase in net income is driven predominantly by increased FIRDAPSE revenue, the revenue resulting from the commercial launch of AGAMREE and the fact that in Q3 2023, there was an $81.5 million charge related to the acquired in-process R&D for the AGAMREE acquisition.

我們報告的 2024 年第三季 GAAP 淨利潤為 4,390 萬美元，或稀釋後每股 0.35 美元，而 2023 年第三季 GAAP 淨虧損為 3,080 萬美元，或稀釋後每股 0.29 美元。淨利潤的成長主要是由於 FIRDAPSE 收入的增加、AGAMREE 的商業推出所產生的收入以及 2023 年第三季與 AGAMREE 收購所獲得的正在進行的研發相關的 8,150 萬美元費用。

It is worth noting that while selling, general, and administrative expenses have increased principally due to the incremental expenses incurred in support of AGAMREE, as noted above, we remain extremely focused on a disciplined approach to cost management throughout the company.

值得注意的是，儘管銷售、一般和管理費用的增加主要是由於支持 AGAMREE 所產生的增量費用（如上所述），但我們仍然非常注重整個公司嚴格的成本管理方法。

Our year-to-date effective tax rate through the first 9 months of 2024 was 24% compared to 23.5% through the nine months of 2023. Our effective tax rate is affected by many factors, including the number of stock options exercised in any given period and is likely to fluctuate in future periods.

2024 年前 9 個月我們年初至今的有效稅率為 24%，而 2023 年前 9 個月的有效稅率為 23.5%。我們的有效稅率受到許多因素的影響，包括在任何特定時期內行使的股票選擇權數量，並且在未來期間可能會發生波動。

As Jeff mentioned, based primarily on the continued strong performance of FIRDAPSE and the strong performance of AGAMREE, which we launched commercially in March of this year, we have increased our full year 2024 revenue guidance to between $475 million and $485 million, which is further detailed in the press release that we issued last night.

正如傑夫所提到的，主要基於FIRDAPSE 的持續強勁表現以及我們今年3 月商業推出的AGAMREE 的強勁表​​現，我們將2024 年全年收入指導提高至4.75 億美元至4.85 億美元之間，這進一步提高了我們昨晚發布的新聞稿中有詳細說明。

However, if we elect to make future acquisitions of drug products in earlier stages of development, we expect that our R&D expenses will become more significant in future periods. Additionally, based on year-to-date R&D expenses, we believe that full year 2024 R&D expense will be significantly lower than our previously provided guidance due to the timing of certain R&D-related initiatives and will likely be less than $15 million for the year.

然而，如果我們選擇未來收購處於開發早期階段的藥品，我們預計我們的研發費用在未來期間將變得更加重要。此外，根據今年迄今的研發費用，我們認為，由於某些研發相關措施的時間安排，2024 年全年研發費用將顯著低於我們先前提供的指導，且全年可能低於 1,500 萬美元。

Non-GAAP net income for the third quarter of 2024 was $71.1 million or $0.57 per diluted share which excludes from GAAP net income, amortization of intangible assets related to our acquisitions of FYCOMPA, AGAMREE and Ruzurgi of $9.3 million, stock-based compensation expense of $4.4 million, the income tax provision of $13.3 million and depreciation of $106,000.

2024 年第三季的非GAAP 淨利為7,110 萬美元，或稀釋後每股0.57 美元，其中不包括GAAP 淨利潤、與我們收購FYCOMPA、AGAMREE 和Ruzurgi 相關的無形資產攤銷930 萬美元、基於股票的薪資費用440 萬美元，所得稅準備金 1,330 萬美元，折舊 106,000 美元。

This compares to non-GAAP net loss in the third quarter of 2023 of $25.6 million or a loss of $0.24 per basic and diluted share, which excludes from GAAP net income, amortization of intangible assets related to our acquisitions of FYCOMPA and Ruzurgi of $8.5 million, stock-based compensation expense of $3.8 million, the income tax benefit of $7.3 million and depreciation of $81,000.

相較之下，2023 年第三季的非GAAP 淨虧損為2,560 萬美元，或每股基本股和稀釋股虧損0.24 美元，其中不包括GAAP 淨利潤、與我們收購FYCOMPA 和Ruzurgi 相關的無形資產攤銷850 萬美元，股票補償費用為 380 萬美元，所得稅優惠為 730 萬美元，折舊為 81,000 美元。

As I mentioned, in Q3 2023, we acquired the exclusive rights to North America for AGAMREE in advance of US FDA approval, which required that the company expensed the acquired IP R&D at the time of acquisition.

正如我所提到的，2023 年第三季度，我們在美國 FDA 批准之前獲得了 AGAMREE 在北美的獨家經營權，這要求該公司在收購時將所收購的智慧財產權研發費用化。

Based on recent feedback from the US Securities and Exchange Commission, or SEC, we will no longer adjust non-GAAP net income or loss for acquisition-related IP R&D expenses. This change has been reflected in the non-GAAP reporting in the press release we issued last night.

根據美國證券交易委員會 (SEC) 最近的回饋，我們將不再調整與收購相關的智慧財產權研發費用的非 GAAP 淨利潤或虧損。這項變更已反映在我們昨晚發布的新聞稿中的非公認會計準則報告中。

As reported, we ended the third quarter of 2024 with cash and cash equivalents of $442.3 million compared to $137.6 million as of December 31, 2023. The increase in cash of $304.7 million was driven in part by the January 9, 2024 capital raise that resulted in $140.7 million in net cash proceeds.

根據報道，截至 2024 年第三季度，我們的現金和現金等價物為 4.423 億美元，而截至 2023 年 12 月 31 日為 1.376 億美元。現金增加 3.047 億美元，部分原因是 2024 年 1 月 9 日的融資導致淨現金收益 1.407 億美元。

The remaining increase in cash is driven primarily by $168.9 million in cash generated from operations of the business. I would like to note that when compared to year-to-date September 2023, we generated approximately $81 million more in cash from operations year-to-date through September 2024.

其餘現金成長主要由業務營運產生的 1.689 億美元現金推動。我想指出的是，與 2023 年 9 月年初至今相比，截至 2024 年 9 月，我們從年初至今的營運中產生了約 8,100 萬美元的現金。

We believe our current funds and strong balance sheet continue to allow us the financial flexibility to support our strategic initiatives such as business development and portfolio expansion, meet our potential contractual obligations and fund our existing commercial and R&D programs.

我們相信，我們現有的資金和強勁的資產負債表將繼續為我們提供財務靈活性，以支持我們的策略舉措，例如業務發展和投資組合擴張，履行我們潛在的合約義務，並為我們現有的商業和研發項目提供資金。

More detailed information and analysis of our third-quarter 2024 and year-to-date September 2024 financial performance may be found in our quarterly report on Form 10-Q, which was filed with the Securities and Exchange Commission yesterday, November 6, and can be found on the Investor Relations page on our website at www.catalystpharma.com.

有關我們 2024 年第三季和 2024 年至今 9 月至今財務業績的更多詳細資訊和分析，請參閱我們於 11 月 6 日向美國證券交易委員會提交的 10-Q 表格季度報告。 .catalystpharma.com 的投資者關係頁面上找到。

At this time, I will turn the call back over to Rich. Rich?

此時，我會將電話轉回給 Rich。富有的？

Thanks, Mike. Before we close, I want to reaffirm our confidence in the resilience of our business and the robust strategies driving our long-term growth. This quarter's performance underscores our commitment to innovation, commercial and operational excellence, and delivering consistent value.

謝謝，麥克。在結束之前，我想重申我們對我們業務的彈性以及推動我們長期成長的穩健策略的信心。本季的業績突顯了我們對創新、卓越商業和營運以及提供一致價值的承諾。

I would also like to express my sincere gratitude to our dedicated team whose commitment to excellence in our patient communities have been instrumental in achieving our continued success. Looking ahead, our focus is on executing strategic priorities and expanding our commercial portfolio.

我也要向我們的敬業團隊表達誠摯的謝意，他們對患者社區卓越的承諾對於我們取得持續成功發揮了重要作用。展望未來，我們的重點是執行策略重點和擴大我們的商業投資組合。

With a solid framework in place, Catalyst is well positioned to sustain our momentum and drive continued success. We appreciate our stakeholder support and we look forward to sharing our progress in the coming quarters.

有了堅實的框架，Catalyst 就能夠保持我們的動力並推動持續成功。我們感謝利益相關者的支持，並期待在未來幾季分享我們的進展。

At this time, I'll turn the call back to the operator and look forward to taking your questions. Thank you.

此時，我會將電話轉回給接線生，並期待回答您的問題。謝謝。

(Operator Instructions) Joon Lee, Truist Securities.

（操作員指示）Joon Lee，Truist 證券。

(technical difficulty) taking questions.

（技術難度）提問。

So my first question is, now that FIRDAPSE will be commercially available in Japan this year, what are you looking for in a partner to commercialize in other regions such as China? And then as a follow-up, just curious what drove the gross to net adjustment for FYCOMPA? And how should we expect that to trend going forward?

所以我的第一個問題是，既然FIRDAPSE今年將在日本上市，那麼您在尋找什麼合作夥伴來在中國等其他地區進行商業化？作為後續行動，只是好奇是什麼推動了 FYCOMPA 的毛淨調整？我們該如何預期這種趨勢的未來發展？

Steve, do you want to take that one?

史蒂夫，你想拿走那個嗎？

Sure. As we previously announced, we expect -- and Rich described, we expect again -- I'm sorry, FIRDAPSE to be launched in Japan at the end of the year. And we continue to evaluate other territories throughout the world to expand the FIRDAPSE market into, and we'll report that to the investment community when we have more to say about it.

當然。正如我們之前宣布的那樣，我們預計——正如 Rich 所描述的，我們再次預計——抱歉，FIRDAPSE 將於今年年底在日本推出。我們將繼續評估世界各地的其他地區，以將 FIRDAPSE 市場擴展到其中，當我們有更多要說的時候，我們將向投資界報告。

Regarding China -- thanks for the question. Regarding China, we continue to explore opportunities in China. And as they become more apparent and clearer, we'll bring that information forward. So thank you.

關於中國——謝謝你的提問。關於中國，我們繼續探索中國的機會。當它們變得更加明顯和清晰時，我們將提供這些資訊。所以謝謝你。

Can you repeat your question about FYCOMPA, Joon, please?

Joon，您能重複一下關於 FYCOMPA 的問題嗎？

Yeah, sorry. I was just curious what drove the gross to net adjustment for FYCOMPA and how we should expect that to trend going forward? Thank you.

是的，抱歉。我只是好奇是什麼推動了 FYCOMPA 的毛淨調整，以及我們應該如何預期這一趨勢的未來發展？謝謝。

Mike, do you want to handle that?

麥克，你想處理這個事情嗎？

Sure. Thanks, Rich. So as we've previously disclosed, our fees, which are reflected in gross to net are higher, specifically with distributors, government agencies compared to last year when we were under the TSA with Eisai. So I would say where we're at is probably reflective of what we think those fees will be going forward, certainly as long as we have exclusivity.

當然。謝謝，里奇。因此，正如我們之前所披露的，與去年我們與衛材 (Eisai) 簽訂 TSA 時相比，我們的費用（反映在總淨額中）更高，特別是與分銷商、政府機構的費用。因此，我想說，我們目前的情況可能反映了我們認為這些費用將如何發展，當然只要我們擁有排他性。

Yeah. And I just want to add on to that. As Jeff mentioned in his part of the call that the performance of the product from a prescription basis is solid. And so that's why we reaffirmed our guidance on that. So the product is performing really well.

是的。我只想補充一點。正如傑夫在電話會議中提到的那樣，該產品在處方基礎上的表現是可靠的。這就是我們重申對此的指導的原因。所以該產品的性能非常好。

It's just, I think, our situation where the fees are just higher than we would experience if we were under Eisai's agreements.

我認為，這只是我們的情況，費用比我們根據衛材協議所經歷的要高。

Thank you.

謝謝。

Thank you.

謝謝。

And the one thing I just want to add on that our fees are consistent with other companies our size, I believe from my experience.

我只想補充一點，根據我的經驗，我相信我們的費用與其他我們規模的公司是一致的。

Absolutely.

絕對地。

Jason Gerberry, Bank of America.

傑森‧格伯里，美國銀行。

Hey, guys, this is [Pavan] on for Jason. FFirst question on FIRDAPSE. Can you speak to what was the real source of uptake of FIRDAPSE? Was it idiopathic or small cell lung cancer patients? And how has the number of patients in your pipeline of identified candidates been?

嘿，夥計們，這是傑森的[Pavan]。F關於 FIRDAPSE 的第一個問題。您能談談 FIRDAPSE 吸收的真正來源是什麼嗎？是特發性肺癌還是小細胞肺癌患者？您確定的候選者管道中的患者數量如何？

Is it steady or growing or over time being depleted? Just some color on the future growth opportunity there. And then with regards to AGAMREE, with the improved near-term ramp, wondering if you have any thoughts on the peak sales potential. Is EMFLAZA still a good comp? Or should we be thinking about AGAMREE even higher than EMFLAZA at this point? Thanks.

它是穩定的、成長的還是隨著時間的推移而耗盡的？只是對那裡未來的成長機會進行一些描述。然後關於 AGAMREE，隨著近期銷量的改善，想知道您對高峰銷售潛力是否有任何想法。EMFLAZA 仍然是一個很好的組合嗎？或者我們現在應該考慮 AGAMREE 甚至比 EMFLAZA 更高？謝謝。

Pavan, thank you for the question. Jeff, do you want to take the first two?

帕萬，謝謝你的提問。傑夫，你想拿前兩個嗎？

Yeah. Hi, Pavan. The small cell lung cancer LEMS patient mix of our total patients still -- it's around 20% to 25% of our total patients. So very, very strong percentage there.

是的。嗨，帕萬。小細胞肺癌 LEMS 患者仍占我們患者總數的 20% 到 25% 左右。那裡的比例非常非常高。

We do expect that to slightly go up over time. Our efforts towards small cell lung cancer are really focused on getting these patients, helping these patients the 90% of the small cell lung cancer LEMS patients that are currently undiagnosed.

我們確實預計這一數字會隨著時間的推移而略有上升。我們對小細胞肺癌的努力真正集中在吸引這些患者，幫助這些患者以及目前未確診的小細胞肺癌 LEMS 患者中的 90%。

We want to help these patients get tested and then become addressable as treatment -- patients for treatment on FIRDAPSE. As far as the pipeline goes, this number, we've done a fantastic job of refilling these patients.

我們希望幫助這些患者接受檢測，然後進行治療—接受 FIRDAPSE 治療的患者。就管道的進展而言，這個數字，我們在為這些患者補充藥物方面做得非常出色。

So pipeline patients that become on active FIRDAPSE patients, we backfill those patients with new leads. So it's been very consistent of over 500 pipeline patients over the last two to three years. And we expect that number to maintain at 500 at least in the near term.

因此，對於成為活躍 FIRDAPSE 患者的管道患者，我們以新的導極回填這些患者。因此，在過去兩到三年裡，超過 500 名管道患者的情況非常一致。我們預計該數字至少在短期內將維持在 500 家。

Pavan, on your -- the last question you asked regarding whether EMFLAZA is a good surrogate for us. I think it is a relatively good surrogate, but I think we have to take into account the shape of the market. And by that, I mean, when EMFLAZA launched, effectively, there was no other countervailing voice in the market.

Pavan，關於您提出的最後一個問題，即 EMFLAZA 是否是我們的良好替代品。我認為這是一個相對好的替代品，但我認為我們必須考慮市場的形態。我的意思是，當 EMFLAZA 推出時，市場上實際上沒有其他反對的聲音。

Certainly, there was prednisone in the market, but they had no voice. And when we launched, we're launching into a competitive market, an active competitive market. And I want to be really clear about this. We're thrilled with the performance of AGAMREE, absolutely thrilled with the performance.

當然，市場上有潑尼松，但他們沒有話語權。當我們推出時，我們正在進入一個競爭激烈的市場，一個活躍的競爭市場。我想澄清這一點。我們對 AGAMREE 的表演感到非常興奮，對他們的表演感到非常興奮。

We think that EMFLAZA is a relatively good surrogate for where we could end up. However, we believe that we have a better product. We think that the opportunity is here for us to prove that over time. That's one of the reasons for the SUMMIT study. And for the performance of the product, we think the uptake is good.

我們認為 EMFLAZA 是我們最終目標的一個相對較好的替代品。然而，我們相信我們有更好的產品。我們認為，隨著時間的推移，我們有機會證明這一點。這就是 SUMMIT 研究的原因之一。對於產品的性能，我們認為採用情況良好。

And based on what we're seeing in the early markers, and I think Jeff can echo this from his experience and his team's experience, behavior is bearing out in marketplace, the improved behavior on the part of the patients. And we have early data regarding growth that we've actually shared at some medical conferences. So we think EMFLAZA is a relatively good surrogate for us at this time.

根據我們在早期標記中看到的情況，我認為傑夫可以從他的經驗和他的團隊的經驗中呼應這一點，市場上的行為正在證明，患者行為的改善。我們有關於成長的早期數據，我們實際上已經在一些醫學會議上分享了這些數據。所以我們認為 EMFLAZA 目前對我們來說是一個相對較好的替代品。

Jeff, I don't know if you want to make any comments. We also have Gary Ingenito on the line as well, and he can speak to the -- some of the data that was presented recently at a conference.

傑夫，我不知道你是否想發表評論。我們還有加里·英傑尼托 (Gary Ingenito) 也在線上，他可以談論最近在一次會議上提出的一些數據。

Gary, I don't know if you want to make any comments about that data.

加里，我不知道你是否想對這些數據發表任何評論。

No, I think the data was very well received, very exciting. And again, lots of interest in AGAMREE.

不，我認為這些數據很受歡迎，非常令人興奮。再次，人們對 AGAMREE 很感興趣。

Great. Thanks. Thanks, guys, and congrats on the great quarter.

偉大的。謝謝。謝謝大家，恭喜這個偉大的季度。

Thank you very much.

非常感謝。

Charles Duncan, Cantor.

查爾斯鄧肯，康托爾。

Hey, good morning, Rich and team. Congrats on a nice quarter and thanks for taking our question. I had questions around AGAMREE versus FIRDAPSE and commercial strategy. First of all, when you think about FIRDAPSE, it seems like it's about finding new patients. With AGAMREE, it's a little bit different.

嘿，早上好，里奇和團隊。恭喜您度過了一個愉快的季度，並感謝您提出我們的問題。我對 AGAMREE 與 FIRDAPSE 以及商業策略有疑問。首先，當您想到 FIRDAPSE 時，它似乎是為了尋找新患者。對於 AGAMREE，情況有點不同。

So can you help us understand the synergies that you see in the commercial efforts? And then I had a follow-up on the SUMMIT study.

那麼您能幫助我們了解您在商業努力中看到的綜效嗎？然後我對 SUMMIT 研究進行了跟進。

Thanks for the question, Charles. I'll turn it over to Jeff for the answer.

謝謝你的提問，查爾斯。我會把它交給傑夫尋求答案。

Hi, Charles. So you're absolutely right. They're very different in the sales approach. Our efforts for FIRDAPSE are really focused on helping these patients get tested, diagnosed and then treated on FIRDAPSE.

嗨，查爾斯。所以你是完全正確的。他們的銷售方式有很大不同。我們對 FIRDAPSE 的努力主要集中在幫助這些患者接受 FIRDAPSE 檢測、診斷和治療。

So we've had education and resources applied towards education and testing for a very long time to help educate these health care providers. The significant data point regarding our prescribers for FIRDAPSE, over 80% of FIRDAPSE prescribers are onetime prescribers only.

因此，我們長期以來一直將教育和資源應用於教育和測試，以幫助教育這些醫療保健提供者。關於我們的 FIRDAPSE 處方者的重要數據點是，超過 80% 的 FIRDAPSE 處方者只是一次性處方者。

So that we have to make sure that we cast a wide net for the physicians out there, neuromuscular specialists, but just as importantly, the general neurologists that are out there. So we have to make sure we're -- we have a broader coverage with our sales force, our nonpersonal promotion and other marketing efforts.

因此，我們必須確保為那裡的醫生、神經肌肉專家，但同樣重要的是，為那裡的普通神經科醫生撒下一張廣泛的網。因此，我們必須確保我們的銷售團隊、非個人促銷和其他行銷工作具有更廣泛的覆蓋範圍。

As far as for boys living with Duchenne Muscular Dystrophy, it's a very, very targeted concentrated prescriber. I think we've mentioned this in the past, 250 prescribers make up about 90% of the steroid prescribers for boys living with DMD. So we know where the prescribers are. They're concentrated in about 100 centers of excellence. So that's where we go.

對於患有杜氏肌肉營養不良症的男孩來說，這是一個非常非常有針對性的集中處方。我想我們過去已經提到過這一點，250 名處方者約佔患有 DMD 的男孩的類固醇處方者的 90%。所以我們知道處方者在哪裡。他們集中在大約 100 個卓越中心。這就是我們要去的地方。

Diagnosis, about 95% of Duchenne muscular dystrophy patients are diagnosed. So our efforts there are really focused on educating physicians about the benefits of AGAMREE for those boys living with DMD. So that's how we really go about our businesses selling its competitive selling, sharing those benefits of AGAMREE. And it looks like we're making significant inroads on that front.

診斷方面，約95%的杜氏肌肉營養不良症患者被診斷出來。因此，我們的工作重點是讓醫生了解 AGAMREE 對患有 DMD 的男孩的好處。這就是我們真正開展業務銷售其競爭性產品、分享 AGAMREE 的好處的方式。看起來我們正在這方面取得重大進展。

That's helpful. My follow-up question is, I guess that's a great segue to my follow-up, which is on the SUMMIT study. I'm wondering if you could provide a little bit more color on what you anticipate for that study over the course of the next year in terms of number of patients? And really, what is the target product profile that you are seeking in terms of the enhanced label with that study?

這很有幫助。我的後續問題是，我想這是我的後續問題（SUMMIT 研究）的一個很好的延續。我想知道您是否可以提供更多關於明年該研究在患者數量方面的預期？事實上，您在該研究中尋求的增強標籤的目標產品概況是什麼？

Great question, Charles. I'm going to turn over to Gary regarding the next year, and then we can also have Gary give us a sense of the target product profile we're seeking as well as Jeff can weigh in on that, too. Gary?

好問題，查爾斯。我將把明年的事情交給加里，然後我們也可以讓加里讓我們了解我們正在尋求的目標產品概況，傑夫也可以對此發表意見。加里？

Thank you. Yes. Well, the study is a five-year study because, as you know, DMD is progressive. It progresses at different rates in individuals. So really, it's the long-term data that is probably the most revealing about the target profile for AGAMREE.

謝謝。是的。嗯，這項研究是一項為期五年的研究，因為如您所知，DMD 是漸進的。它在個體中以不同的速度進展。事實上，長期數據可能最能揭示 AGAMREE 的目標概況。

It's -- we are in the site activation stages of the study. It's not a traditional registry in the sense that Catalyst is seeking to collect the highest quality, most consistent data of previous registry type studies. By that, I mean, not only do you go through the usual steps of getting a site qualified and activated, but for the bone imaging, the DEXA and the spine X-rays. We actually have a central reading facility. The each site must be trained.

我們正處於研究的現場活化階段。它不是一個傳統的註冊機構，因為 Catalyst 致力於收集先前註冊機構類型研究的最高品質、最一致的數據。我的意思是，您不僅要完成使站點合格並激活的常規步驟，還要進行骨成像、DEXA 和脊椎 X 光檢查。我們實際上有一個中央閱讀設施。每個站點都必須經過訓練。

They must have the proper equipment. They have to pass a quality test. And the same for those participating in the cardiac sub-study where we're looking at the transmural strain profiles by echocardiography.

他們必須擁有適當的設備。他們必須通過品質測試。對於參與心臟子研究的人也是如此，我們透過超音波心動圖觀察透壁應變分佈。

So again, each site requires two sets of training. You've got to bring in the cardiology department, the radiology department. And so I think as we progress in site activation, we expect that then the prescribers will be able to get their patients enrolled into the study over the next five years and then tracking them for that period of time.

同樣，每個站點都需要兩套培訓。你必須引進心臟科、放射科。因此，我認為，隨著我們在站點激活方面取得進展，我們預計處方醫生將能夠讓他們的患者在未來五年內加入研究，然後在這段時間內追蹤他們。

The profile we see is really to what we already believe about the product, have seen in preclinical and the clinical data generated to date, which is that we will have a very positive effect on bone health. We will reduce fracture rates.

我們看到的概況確實符合我們對該產品的信念，在臨床前和迄今為止產生的臨床數據中看到的，這就是我們將對骨骼健康產生非常積極的影響。我們將降低骨折率。

We will not produce cataracts and that there will be a limited increase in weight that will stabilize over the long haul. And then, of course, from the efficacy perspective, as we've already demonstrated in the Phase 3 trials, preserve muscle strength and mobility.

我們不會產生白內障，體重的增加也有限，且長期穩定。當然，從功效的角度來看，正如我們在第三階段試驗中所證明的那樣，可以保持肌肉力量和活動能力。

Excellent. Thanks for taking the question and appreciate the guidance raise.

出色的。感謝您提出問題並感謝提出的指導。

(Operator Instructions) Samantha Semenkow, Citi.

（操作員指示）Samantha Semenkow，花旗銀行。

Hi. Good morning and thanks for taking the question. Just a quick one on AGAMREE. I know it's really early in the launch, but I'm curious if you're seeing any trends on durability of therapy so far as in are patients tending to stay on therapy after switching?

你好。早上好，感謝您提出問題。簡單介紹一下 AGAMREE。我知道現在還處於啟動階段，但我很好奇您是否看到治療持久性方面的任何趨勢，例如患者在轉換後是否傾向於繼續接受治療？

And then again, your patient mix seems to be quite stable compared to last quarter. Is the expectation that this trend will continue? Or do you expect it to shift at some point in the future? Thank you.

再說一遍，與上季相比，您的患者組合似乎相當穩定。是否預期這種趨勢將持續下去？或者你預計它會在未來某個時候發生轉變？謝謝。

Thanks for the question, Sam. Jeff, do you want to handle this?

謝謝你的提問，山姆。傑夫，你想處理這件事嗎？

Hi, Sam. Your first question about discontinuations or durability on AGAMREE, we are seeing very strong discontinuation rates, positive discontinuation rates of about 15%, what we had expected. So -- and that's been very stable over the last couple of months. And so yes, that's a positive. And as far as the mix, are you talking specifically about the transitions from prednisone and also EMFLAZA?

嗨，山姆。你的第一個問題是關於 AGAMREE 的停藥或持久性，我們看到非常高的停藥率，正面的停藥率約為 15%，這是我們的預期。所以——過去幾個月一直非常穩定。是的，這是積極的。就混合而言，您是在具體談論潑尼松和 EMFLAZA 的轉變嗎？

Yes, exactly.

是的，完全正確。

Okay. So Sam, you're right, and they are stable since the last quarter. And we do expect that percent or that mix to be pretty consistent. It's positive for us because it shows that we are getting transitions across both corticosteroid segments, whereas initially planning on this launch, we anticipated the EMFLAZA switches to be a lot more significant, and we're very pleasantly surprised that we are consistently getting the prednisone switches. So we expect that to be pretty consistent moving forward, too.

好的。所以山姆，你是對的，自上個季度以來他們一直保持穩定。我們確實預期這個百分比或這個組合會非常一致。這對我們來說是積極的，因為它表明我們正在跨兩個皮質類固醇細分市場進行轉變，而最初計劃這次推出，我們預計EMFLAZA 的轉變會更加重要，而且我們非常驚訝地發現我們一直在獲得潑尼松開關。因此，我們預期未來的發展也將保持一致。

Great. Thank you. And just a follow-up on the discontinuation rate. For that, can you just elaborate on what makes that really positive in your view? And also what is driving the 15% that you're seeing discontinue? Thank you.

偉大的。謝謝。只是停藥率的後續行動。為此，您能否詳細說明您認為這真正具有正面意義的因素是什麼？另外，是什麼原因導致您看到的 15% 的產品停產了？謝謝。

15% to 20% is typically what we had forecast, and we see that as a good gauge for discontinuation, and it's meeting that. And what was your other question about that, Sam? What are the drivers?

15% 到 20% 通常是我們的預測，我們認為這是一個很好的停藥標準，而且它也滿足了這項要求。山姆，你對此的其他問題是什麼？驅動因素是什麼？

What are the drivers? Yes, exactly.

驅動因素是什麼？是的，完全正確。

What's interesting and what's great for the Duchenne community is the fact that there are a lot of new products that are available. So as part of this and new products that are becoming approved is the evaluation of what products that they're on.

有趣且對 Duchenne 社群來說最重要的是，有許多新產品可供使用。因此，作為該產品和正在獲得批准的新產品的一部分，評估它們所使用的產品。

So -- and for example, if they're going to be on a gene therapy, they have to consider what they're going to do. And so there are some patients that discontinue because of that choice, they have to evaluate if they should be on AGAMREE at this point. But -- so it's just a slight number, a small number.

因此，例如，如果他們要接受基因治療，他們必須考慮他們要做什麼。因此，有些患者因為這種選擇而停藥，他們必須評估此時是否應該服用 AGAMREE。但是——所以這只是一個很小的數字，一個很小的數字。

But as far as efficacy, side effects, there is no key contributor to that. Nothing really stands out. It's a small percentage there, 15%. So it's a temporary discontinuation, Sam, that I was talking. So these patients that over time, they will go back on to AGAMREE, we expect.

但就功效、副作用而言，沒有關鍵因素。沒有什麼真正突出的。這個比例很小，15%。所以山姆，我所說的只是暫時中斷。因此，我們預計，隨著時間的推移，這些患者將回到 AGAMREE。

Got it. That's super helpful. Thank you for the question and congrats on the good quarter.

知道了。這非常有幫助。感謝您的提問，並祝賀這個季度的良好表現。

Thank you.

謝謝。

Thanks very much.

非常感謝。

Ladies and gentlemen, that concludes today's call. Thank you all for joining. You may now disconnect.

女士們、先生們，今天的電話會議到此結束。感謝大家的加入。您現在可以斷開連線。