Catalyst Pharmaceuticals Inc (CPRX) 2025 Q2 法說會逐字稿

Greetings, and welcome to the Catalyst Pharmaceuticals second-quarter 2025 financial results conference call. (Operator Instructions) As a reminder, this conference is being recorded.

您好，歡迎您參加 Catalyst Pharmaceuticals 2025 年第二季財務業績電話會議。（操作員指示）提醒一下，本次會議正在錄音。

I would now like to turn the conference over to the Chief Financial Officer, Mike Kalb. Please go ahead, sir.

現在我想將會議交給財務長 Mike Kalb。先生，請繼續。

Thank you. Good morning, everyone, and thank you for joining our conference call to discuss Catalyst's second-quarter 2025 financial results and business highlights. Richard Daly, President and CEO, will lead the call today; and Jeffrey Del Carmen, our Chief Commercial Officer, and I will also present. Additionally, Dr. Steven Miller, our Chief Operating Officer and Chief Scientific Officer; and Dr. Will Andrews, our Chief Medical 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 2025 年第二季的財務表現和業務亮點。總裁兼執行長理查德·戴利 (Richard Daly) 將主持今天的電話會議；我們的首席商務官傑弗裡·德爾·卡門 (Jeffrey Del Carmen) 和我也將出席會議。此外，我們的營運長兼首席科學官 Steven Miller 博士和首席醫療官 Will Andrews 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. These statements reflect our current expectations, estimates and projections and do not guarantee 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.

在我們開始之前，我想提醒大家，在今天早上的發言和問答環節中，我們將對預期的未來結果做出陳述，根據聯邦證券法，這些陳述可能是前瞻性的。這些聲明反映了我們目前的預期、估計和預測，並不能保證未來的表現。它們涉及難以預測且可能無法證明準確的風險、不確定性和假設。實際結果可能與我們的前瞻性陳述中的預期有所不同。

These forward-looking statements should be considered only in conjunction with our detailed information contained in our SEC filings, including the risk factors described in our 2024 annual report on Form 10-K filed with the SEC on February 26, 2025, and in our second quarter 2025 quarterly report on Form 10-Q, which was filed yesterday, August 6, 2025, with the SEC.

這些前瞻性陳述應僅與我們提交給美國證券交易委員會 (SEC) 的文件中包含的詳細資訊結合考慮，包括我們於 2025 年 2 月 26 日向美國證券交易委員會提交的 2024 年 10-K 表年度報告和昨天（2025 年 8 月 6 日）向美國證券交易委員會提交的 2015 年美國證券交易委員會提交的季度風險因素。

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

現在，我將把電話轉給里奇。富有的？

Thanks, Mike. Good morning, everyone, and thank you for joining us. Catalyst delivered another record-setting quarter in Q2 2025 with total revenue reaching $146.6 million, an increase of 19.4% year-over-year. For the first half of 2025, total revenue grew 30.2% to $288 million, reflecting strong execution and sustained demand for our differentiated portfolio. We ended the second quarter of 2025 with a cash position of $652.8 million, reinforcing our ability to invest strategically for long-term growth.

謝謝，麥克。大家早安，感謝大家的收看。Catalyst 在 2025 年第二季再次創下紀錄，總營收達到 1.466 億美元，年增 19.4%。2025 年上半年，總營收成長 30.2% 至 2.88 億美元，反映了我們強大的執行力和對差異化產品組合的持續需求。截至 2025 年第二季度，我們的現金狀況為 6.528 億美元，增強了我們為長期成長進行策略性投資的能力。

Based on strong leading indicators, we remain confident in our trajectory and believe that we are on track to achieve our full year 2025 revenue guidance of $545 million to $565 million. Our outstanding progress is underpinned by the balanced performance of our commercial portfolio, which continues to deliver consistent growth and a positive impact on patients.

基於強勁的領先指標，我們對我們的發展軌跡充滿信心，並相信我們預計將實現 2025 年全年 5.45 億美元至 5.65 億美元的收入預期。我們的出色進步得益於我們商業組合的均衡表現，該組合繼續帶來持續成長並對患者產生積極影響。

Let's dive deeper and start with FIRDAPSE. FIRDAPSE generated net product revenue of $84.8 million in Q2 2025, a growth of $7.5 million versus Q2 2024. As we discussed in our Q1 2025 earnings call, last year's second quarter results reflect the timing impact of the February 2024 Change Healthcare cybersecurity breach on our year-over-year growth rate. The temporary impact of the Change Healthcare security breach, which shifted volume from Q1 2024 to Q2 2024 was fully resolved by the end of June 2024. As a result, we believe that comparing FIRDAPSE first half 2025 performance to the same period in 2024 provides the most accurate view of the franchise's continued strength.

讓我們深入了解一下，從 FIRDAPSE 開始。FIRDAPSE 在 2025 年第二季創造了 8,480 萬美元的淨產品收入，比 2024 年第二季成長了 750 萬美元。正如我們在 2025 年第一季財報電話會議上所討論的那樣，去年第二季的業績反映了 2024 年 2 月 Change Healthcare 網路安全漏洞對我們同比成長率的時間影響。Change Healthcare 安全漏洞造成的暫時影響將交易量從 2024 年第一季轉移到 2024 年第二季度，並於 2024 年 6 月底完全解決。因此，我們認為，將 FIRDAPSE 2025 年上半年的表現與 2024 年同期進行比較，可以最準確地反映球隊的持續實力。

Importantly, the underlying demand remains strong, consistent and durable. Year-to-date, FIRDAPSE has delivered $168.6 million in net product revenue, representing a 16.9% increase over the first half of 2024. In 2025, our sales of FIRDAPSE have returned to their expected cadence, and we reaffirm our full year 2025 product -- net product revenue guidance of $355 million to $360 million.

重要的是，潛在需求依然強勁、持續且持久。年初至今，FIRDAPSE 的淨產品收入為 1.686 億美元，比 2024 年上半年成長 16.9%。2025 年，我們的 FIRDAPSE 銷售已恢復到預期節奏，我們重申 2025 年全年產品淨產品收入預期為 3.55 億美元至 3.6 億美元。

We are confident that FIRDAPSE remains well positioned for sustained organic growth, supported by consistently high prescription approval rates and a robust pool of patients progressing through their diagnostic journey. This strong and visible demand underscores the durability of our franchise and reinforces our leadership in addressing the needs of the LEMS patient community.

我們相信，在持續的高處方批准率和大量正在診斷過程中的患者的支持下，FIRDAPSE 仍將保持持續有機增長的良好勢頭。這種強勁而明顯的需求凸顯了我們特許經營權的持久性，並鞏固了我們在滿足 LEMS 患者群體需求方面的領導地位。

To unlock the next phase of growth for FIRDAPSE, we are actively advancing a focused expansion strategy centered on education of HCPs supported by the recent updated NCCN guidelines. Since we believe that potentially 90% of cancer-associated LEMS patients remain undiagnosed, we see a meaningful opportunity to expand our reach in this high potential, underserved population. We expect momentum to build in the months ahead, setting the stage for a sustained growth in 2026 and beyond. Jeff will cover the specifics in his section of the call.

為了開啟 FIRDAPSE 的下一階段成長，我們正在積極推動以 HCP 教育為中心的重點擴展策略，並以最近更新的 NCCN 指南為支援。由於我們相信可能有 90% 的癌症相關 LEMS 患者仍未得到診斷，因此我們認為這是一個擴大我們在這個潛力巨大、服務不足的人群中的覆蓋範圍的重要機會。我們預計未來幾個月的成長動能將會增強，為 2026 年及以後的持續成長奠定基礎。傑夫將在通話中介紹具體細節。

While FIRDAPSE continues to perform well, we are equally encouraged by the accelerating adoption and performance of AGAMREE. AGAMREE continues to outperform expectations, generating $27.4 million in net product revenue in Q2 2025, a new post-launch high and a 213% increase year-over-year from Q2 2024. First half net revenues reached $49.4 million, up 398% from the prior year, driven by steady conversion from both prednisone and EMFLAZA, a strong 90% patient retention rate and growing adoption across Duchenne's Centers of Excellence.

雖然 FIRDAPSE 繼續表現良好，但 AGAMREE 的加速採用和性能同樣令我們感到鼓舞。AGAMREE 持續超越預期，2025 年第二季的淨產品收入達到 2,740 萬美元，創下上市後的新高，與 2024 年第二季相比年增 213%。上半年淨收入達到 4,940 萬美元，比上年增長 398%，這得益於潑尼松和 EMFLAZA 的穩定轉換、高達 90% 的患者保留率以及杜氏卓越中心的不斷增長的採用率。

AGAMREE's commercial execution is tracking well with strong patient retention and increasing prescriber engagement, further supported by our full deployment of our dedicated field team. Continued transitions from both branded and generic therapies, along with growing market receptivity and payer alignment reinforce our confidence in meeting our full year outlook. Backed by focused commercial strategy, AGAMREE is well positioned to continue momentum. We are reaffirming AGAMREE's full year 2025 net product revenue guidance of $100 million to $110 million.

AGAMREE 的商業執行進展順利，病患保留率高，處方醫師參與度不斷提高，我們全面部署的專業現場團隊也為 AGAMREE 提供了進一步的支持。品牌療法和仿製藥的持續轉變，以及市場接受度和付款人一致性的提高，增強了我們實現全年預期的信心。在專注的商業策略的支持下，AGAMREE 已做好準備繼續保持這一發展勢頭。我們重申 AGAMREE 2025 年全年淨產品收入預期為 1 億至 1.1 億美元。

Let's switch to FYCOMPA. FYCOMPA delivered solid results in the second quarter of 2025 with revenue of $34.3 million, reflecting a year-over-year decrease of 6%. First half 2025 revenue reached $70 million, up 4.5% from the same period last year. We anticipate the impact of generic competition going forward and our full year net product revenue guidance of $90 million to $95 million for FYCOMPA remains unchanged.

讓我們切換到 FYCOMPA。FYCOMPA 在 2025 年第二季取得了穩健的業績，營收為 3,430 萬美元，年減 6%。2025年上半年營收達7,000萬美元，較去年同期成長4.5%。我們預計仿製藥競爭的影響將持續存在，我們對 FYCOMPA 全年淨產品收入 9,000 萬至 9,500 萬美元的預期保持不變。

We recently strengthened our Corporate and Board leadership. I'd like to formally welcome Dr. Will Andrews as our new Chief Medical Officer. Will joined Catalyst on June 2, 2025. Will brings deep pharmaceutical expertise in rare diseases, spanning clinical development, medical affairs, business development and portfolio strategy.

我們最近加強了公司和董事會的領導。我正式歡迎威爾安德魯斯博士擔任我們的新任首席醫療官。Will 於 2025 年 6 月 2 日加入 Catalyst。Will 在罕見疾病領域擁有深厚的製藥專業知識，涵蓋臨床開發、醫療事務、業務開發和投資組合策略。

His leadership will ensure a scientific rigor and operational excellence as we continue to differentiate AGAMREE within the Duchenne treatment landscape and further solidify our position in the LEMS community. Will's role in leading our medical function is foundational to ensuring the long-term success of our portfolio.

他的領導將確保科學的嚴謹性和卓越的運營，我們將繼續在杜氏肌肉營養不良症治療領域中區分 AGAMREE，並進一步鞏固我們在 LEMS 社區中的地位。威爾在領導我們的醫療職能方面所發揮的作用對於確保我們投資組合的長期成功至關重要。

For AGAMREE, Will is leading the SUMMIT study, which aims to generate real-world evidence in support of the appropriate use of AGAMREE. To-date, the study has initiated 19 sites with patient enrollment progressing. As the study progresses, we will provide updates when the interim data becomes available. In parallel, Will is overseeing the execution of our Phase 1 study comparing AGAMREE, prednisone, and deflazacort to determine the potential switching algorithms. Additionally, we are working to assess the immunosuppressive effects of AGAMREE that could help us define the life cycle management potential for -- of AGAMREE.

對於 AGAMREE，威爾正在領導 SUMMIT 研究，該研究旨在產生現實世界的證據來支持 AGAMREE 的適當使用。迄今為止，該研究已在 19 個地點啟動，患者招募工作正在進行中。隨著研究的進展，我們將在獲得中期數據時提供更新。同時，威爾正在監督我們第一階段研究的執行，該研究比較了 AGAMREE、潑尼松和地夫可特，以確定潛在的轉換演算法。此外，我們正在努力評估 AGAMREE 的免疫抑製作用，這有助於我們確定 AGAMREE 的生命週期管理潛力。

Initial results are expected by the end of 2025 to early 2026. Will's expertise and insights also enhance our strategic perspective as we advance growth initiatives aligned with our long-term vision. This past Monday, we announced that Dr. Dan Curran joined our Board of Directors. We are pleased to welcome Dr. Curran to our Board of Directors. He brings deep experience in rare disease and a strong track record in business development and strategic growth. His experience advancing transformative therapies and building value across the development continuum align well with our mission and growth strategy.

初步結果預計將於 2025 年底至 2026 年初公佈。當我們推進符合我們長期願景的成長計畫時，威爾的專業知識和見解也增強了我們的策略視野。上週一，我們宣布 Dan Curran 博士加入我們的董事會。我們很高興歡迎 Curran 博士加入我們的董事會。他在罕見疾病領域擁有豐富的經驗，並在業務發展和策略成長方面擁有出色的業績記錄。他在推進轉化療法和在整個發展過程中創造價值方面的經驗與我們的使命和成長策略非常吻合。

On the business development and IP front, we remain highly disciplined in employing our business development strategy, actively evaluating a broad range of opportunities that align both strategically and financially with our long-range plan. Our commitment to value-driven growth is unwavering, and we are confident in our -- that our focused and deliberate approach will position us to capitalize on the right opportunities. In parallel, we are advancing on our initiatives to protect and enhance the long-term value of our portfolio.

在業務發展和智慧財產權方面，我們嚴格執行業務發展策略，積極評估在策略和財務上與我們的長期計劃相符的廣泛機會。我們對價值驅動型成長的承諾堅定不移，我們相信——我們專注而深思熟慮的方法將使我們能夠利用正確的機會。同時，我們正在推動保護和提高投資組合長期價值的措施。

We are still awaiting a trial date in our ongoing patent litigation for FIRDAPSE with our remaining first filers, but anticipate it to be in Q4 2025 or Q1 2026. We expect more clarity on this issue on or after our Markman hearing, which is scheduled for October 7, 2025. We remain committed to updating our performance related to environmental, social and governance reporting. In June, we published our 2024 ESG report, underscoring our commitment to sustainable growth and responsible innovation. This report is available on Catalyst's website.

我們仍在等待與剩餘的首批申請人就 FIRDAPSE 進行的專利訴訟的審判日期，但預計將在 2025 年第四季或 2026 年第一季舉行。我們期望在 2025 年 10 月 7 日舉行的 Markman 聽證會上或之後對這個問題有更清晰的認識。我們將繼續致力於更新與環境、社會和治理報告相關的績效。6 月，我們發布了 2024 年 ESG 報告，強調了我們對永續成長和負責任創新的承諾。該報告可在 Catalyst 的網站上查閱。

In summary, we are entering the second half of 2025 with strong momentum, clearly defined growth drivers and a focused strategy to deliver long-term value and impact for patients.

總而言之，我們將以強勁的發展勢頭、明確的成長動力和為患者帶來長期價值和影響的重點策略進入 2025 年下半年。

With that, I'll turn it over to Jeff who will provide additional insights into our commercial performance.

接下來，我將把話題交給傑夫，他將對我們的商業表現提供更多見解。

Thanks, Rich. Q2 marked another record-setting quarter for our commercial organization, underscoring continued strong execution across the portfolio and sustained demand for our differentiated therapies.

謝謝，里奇。第二季度是我們商業組織又一個創紀錄的季度，突顯了整個產品組合的持續強勁執行力以及對我們差異化療法的持續需求。

As Rich noted, Catalyst delivered sustained organic growth from FIRDAPSE, strong revenues from FYCOMPA and accelerating adoption of AGAMREE. Q2 net product revenue of $146.5 million puts us firmly on track to meet our 2025 guidance, reflecting the strength of our neuroscience business. FIRDAPSE remains the only evidence-based FDA-approved treatment for Lambert-Eaton Myasthenic Syndrome. The brand continues to perform well across both neurology and our emerging oncology segments.

正如里奇所指出的，Catalyst 透過 FIRDAPSE 實現了持續的有機成長，透過 FYCOMPA 實現了強勁的收入，並加速了 AGAMREE 的採用。第二季淨產品收入為 1.465 億美元，這讓我們有望實現 2025 年的預期，這反映了我們神經科學業務的實力。FIRDAPSE 仍然是唯一獲得 FDA 批准的基於證據的 Lambert-Eaton 肌無力症候群治療方法。該品牌在神經病學和新興腫瘤學領域繼續表現良好。

FIRDAPSE continues its strong multi-quarter growth trajectory, driven by durable demand and consistent execution. Prescription approval rates remained above 90% across both government and commercial payers and discontinuation rates were in line with expectations, tracking below an annualized rate of 20%. These indicators reflect strong patient adherence and reinforce the foundation for sustained performance.

在持久需求和持續執行的推動下，FIRDAPSE 繼續保持強勁的多季度成長軌跡。政府和商業付款人的處方批准率仍維持在 90% 以上，停藥率符合預期，低於 20% 的年化率。這些指標反映了患者的強烈依從性，並鞏固了持續表現的基礎。

Leading indicators, including steady growth in new patient starts and refill volumes through July continue to trend positively, reinforcing our confidence in achieving full year 2025 FIRDAPSE revenue guidance. We continue to advance our patient identification efforts and continue to have a pool of over 500 LEMS patients in active diagnostic stages. These patients consistently account for approximately half of new starts each quarter, giving us confidence in driving sustained organic growth.

領先指標，包括截至 7 月的新患者開始數量和補充量的穩定成長，持續呈現正向趨勢，增強了我們對實現 2025 年全年 FIRDAPSE 收入指引的信心。我們繼續推進患者識別工作，並繼續擁有超過 500 名處於積極診斷階段的 LEMS 患者庫。這些患者始終佔每季新患者總數的約一半，這使我們有信心推動持續的有機成長。

To further unlock the next phase of growth for FIRDAPSE, we are actively advancing a focused expansion strategy centered on HCP education, which is supported by the recently updated NCCN guidelines for small cell lung cancer, broadening access to VGCC antibody testing, streamlining the diagnostic journey and accelerating adoption across oncology and neurology settings.

為了進一步開啟 FIRDAPSE 的下一階段成長，我們正在積極推動以 HCP 教育為中心的重點擴展策略，該策略得到了最近更新的 NCCN 小細胞肺癌指南的支持，擴大了 VGCC 抗體檢測的管道，簡化了診斷過程，並加速了腫瘤學和神經病學環境中的採用。

By improving diagnosis, particularly in small cell lung cancer patients and increasing recognition of patients often misdiagnosed with myasthenia gravis, we hope to meaningfully expand the addressable population and drive sustained growth within what we believe is a more than $1 billion addressable market opportunity. As we advance these priorities, we are implementing a three-step program of targeted initiatives designed to enhance diagnostic access and clinical recognition across key market segments.

透過改善診斷，特別是對小細胞肺癌患者的診斷，並提高對經常被誤診為重症肌無力的患者的認識，我們希望顯著擴大可尋址人群，並在我們認為超過 10 億美元的潛在市場機會中推動持續增長。在推動這些優先事項的同時，我們正在實施一項三步驟的有針對性的計劃，旨在加強主要細分市場的診斷途徑和臨床認可。

First, we are deploying a frictionless testing model that makes it easier for physicians and particularly oncologists to order BGCC antibody screening directly within their practices, eliminating referral delays and helping to significantly reduce time to diagnosis. These efforts represent progress in advancing important diagnostic initiatives to help improve LEMS patient outcomes and accelerate the adoption of FIRDAPSE.

首先，我們正在部署一種無摩擦測試模型，使醫生，特別是腫瘤學家能夠更輕鬆地直接在實踐中進行 BGCC 抗體篩檢，從而消除轉診延遲並有助於顯著縮短診斷時間。這些努力代表著在推動重要診斷措施方面取得的進展，有助於改善 LEMS 患者的治療結果並加速 FIRDAPSE 的採用。

Second, we are pleased to share that ahead of schedule, the NCCN published updated guidelines for small cell lung cancer on July 25, which now include BGCC antibody testing and recommend that amifampridine should be considered as a treatment for cancer patients with LEMS. This milestone strengthens our ability to expand access, accelerate diagnosis and help drive FIRDAPSE adoption in oncology. Most importantly, it represents meaningful progress in improving care for cancer patients living with LEMS.

其次，我們很高興地告訴大家，NCCN 於 7 月 25 日提前發布了小細胞肺癌更新指南，其中現在包括 BGCC 抗體檢測，並建議應考慮將氨苯吡啶作為 LEMS 癌症患者的治療方法。這一里程碑增強了我們擴大獲取途徑、加速診斷和幫助推動 FIRDAPSE 在腫瘤學領域的應用的能力。最重要的是，它代表了改善患有 LEMS 的癌症患者護理方面取得的重大進展。

Our third step involves forging strategic partnerships with leading oncology practices to update care pathways, which are generally structured plans that outline the steps and interventions required for patient care, ensuring standardized and efficient treatment for specific conditions.

我們的第三步是與領先的腫瘤學機構建立策略合作夥伴關係，以更新護理途徑，這些途徑通常是結構化的計劃，概述了患者護理所需的步驟和乾預措施，確保針對特定情況進行標準化和有效的治療。

We plan to execute comprehensive education and promotion programs to ensure broad understanding and integration of the care pathways. Due to the early updating of the NCCN guidelines, we are accelerating our investment in educating HCPs on the new guidelines and working to incorporate diagnostic and treatment recommendations into the care pathways in targeted large group oncology practices.

我們計劃實施全面的教育和推廣計劃，以確保廣泛理解和整合護理途徑。由於 NCCN 指南的早期更新，我們正在加快投資，對 HCP 進行新指南的培訓，並努力將診斷和治療建議納入有針對性的大型腫瘤學實踐的護理途徑中。

Since we believe that potentially 90% of cancer-associated LEMS patients remain undiagnosed, we see a meaningful opportunity to expand our reach in this high potential underserved population. We expect momentum to build in the months ahead, setting the stage for sustained growth in 2026 and beyond.

由於我們相信可能有 90% 的癌症相關 LEMS 患者仍未得到診斷，因此我們認為這是一個擴大我們在這個具有巨大潛力的醫療服務不足人群中的覆蓋範圍的重要機會。我們預計未來幾個月的成長動能將會增強，為 2026 年及以後的持續成長奠定基礎。

Turning to AGAMREE. AGAMREE continues to demonstrate strong early market momentum as a differentiated treatment for Duchenne Muscular Dystrophy. Q2 2025 net product revenue was $27.4 million. Adoption continues to expand across key centers. To-date, 93% of the top 45 DMD centers of excellence and 231 unique healthcare providers have submitted enrollment forms.

轉向 AGAMREE。作為杜氏肌肉營養不良症的差異化治療方法，AGAMREE 繼續展現出強勁的早期市場動能。2025 年第二季淨產品收入為 2,740 萬美元。採用範圍在各主要中心持續擴大。迄今為止，排名前 45 位的 DMD 卓越中心和 231 家獨特的醫療保健提供者中的 93% 已經提交了報名表。

In Q2, approximately 43% of patients transitioned from prednisone and 42% from EMFLAZA, underscoring AGAMREE's broad clinical relevance across standard treatment segments.

在第二季度，約有 43% 的患者從潑尼松轉換，42% 的患者從 EMFLAZA 轉換，凸顯了 AGAMREE 在標準治療領域的廣泛臨床相關性。

Reimbursement success remains high at approximately 85%, consistent with our expectations. Our commercial and medical teams continue to drive targeted provider education and payer engagement, supporting durable uptake and sustained market expansion. FYCOMPA delivered Q2 2025 net product revenue of $34.3 million, reflecting continued demand despite the first generic approval for tablets following loss of exclusivity in late-May.

報銷成功率仍然很高，約 85%，符合我們的預期。我們的商業和醫療團隊繼續推動有針對性的供應商教育和付款人參與，以支持持久的吸收和持續的市場擴張。FYCOMPA 2025 年第二季的淨產品收入為 3,430 萬美元，反映出儘管在 5 月底失去獨家經營權後首次獲得平板電腦仿製藥批准，但需求仍在持續。

As previously noted, we do expect revenue erosion from generic competition to impact FYCOMPA performance in the second half of the year and beyond. That said, based on a strong first half performance and the execution of our mitigation strategies, we are reaffirming our full year 2025 FYCOMPA revenue guidance.

如前所述，我們確實預期仿製藥競爭導致的收入減少將影響 FYCOMPA 在今年下半年及以後的業績。儘管如此，基於上半年的強勁表現和緩解策略的執行，我們重申了 2025 年全年 FYCOMPA 收入指引。

In summary, our commercial and medical teams continue to execute with discipline and focus, delivering strong portfolio performance while advancing the next wave of our growth, led by FIRDAPSE's expansion into oncology. We believe that we are well positioned to seize the growth opportunities ahead, and we remain focused on driving commercial execution excellence, enhancing patient access and scaling our commercial impact across our entire product portfolio.

總而言之，我們的商業和醫療團隊繼續嚴格執行紀律並專注執行，在 FIRDAPSE 向腫瘤學擴展的帶動下，實現了強勁的投資組合業績，同時推動了下一波成長。我們相信，我們已準備好抓住未來的成長機遇，並且我們將繼續專注於推動卓越的商業執行，增強患者的可及性，並擴大我們整個產品組合的商業影響力。

I want to thank the entire team at Catalyst for their unwavering commitment to the patients we serve and look forward to a successful second half of 2025.

我要感謝 Catalyst 整個團隊對我們服務的患者堅定不移的承諾，並期待 2025 年下半年取得成功。

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

說完這些，我就把電話轉回給麥克。

Thank you, Jeff. Our performance during the second quarter of 2025 has kept us on pace for another strong year, driven by our solid financial performance, financial discipline and strong execution. With the continued success of our flagship product, FIRDAPSE as well as the strong growth of AGAMREE, which we launched in mid-March of 2024 and the solid performance of FYCOMPA, which currently has only one generic competitor for the tablets following the patent expiry in May of 2025, we have set the groundwork for what promises to be yet another unparalleled year in 2025.

謝謝你，傑夫。由於穩健的財務業績、財務紀律和強有力的執行力，我們在 2025 年第二季度的業績使我們保持了強勁發展的勢頭，並迎來了又一個強勁的一年。隨著我們旗艦產品 FIRDAPSE 的持續成功、我們於 2024 年 3 月中旬推出的 AGAMREE 的強勁增長以及 FYCOMPA 的穩健表現（在 2025 年 5 月專利到期後，目前該藥片只有一個仿製藥競爭對手），我們為 2025 年又一個無與倫比的一年奠定了基礎。

We are reaffirming our 2025 full year total revenue guidance as initially provided in February and reaffirmed in May. We remain steadfast in our commitment to driving growth and expanding our portfolio to capitalize on emerging opportunities throughout the year.

我們重申 2 月首次提供並在 5 月重申的 2025 年全年總收入指引。我們始終堅定不移地致力於推動成長和擴大投資組合，以利用全年新興機會。

Our total revenues for the second quarter of 2025 were $146.6 million, an approximate 19.4% increase when compared to total revenues of $122.7 million for the second quarter of 2024. Product revenue net for our lead product FIRDAPSE was $84.8 million, a 9.7% increase year-over-year compared to $77.4 million.

我們 2025 年第二季的總營收為 1.466 億美元，與 2024 年第二季的總營收 1.227 億美元相比成長了約 19.4%。我們的主要產品 FIRDAPSE 的產品淨收入為 8,480 萬美元，年增 9.7%，而去年同期為 7,740 萬美元。

As a reminder, the second quarter of 2024 benefited from the Change Healthcare cybersecurity incident that occurred in the first quarter of 2024, which is evidenced by the fact that our growth in net product revenue for FIRDAPSE for the first half of 2025 compared to the first half of 2024 was 16.9%.

提醒一下，2024 年第二季度受益於 2024 年第一季度發生的 Change Healthcare 網路安全事件，這一點可以從我們 2025 年上半年 FIRDAPSE 的淨產品收入與 2024 年上半年相比增長了 16.9% 得到證明。

Product revenue net for the second quarter of 2025 for FYCOMPA was $34.3 million compared to $36.5 million in the second quarter of 2024. Product revenue net in the second quarter of 2025 for AGAMREE was $27.4 million as compared to $8.7 million in the second quarter of 2024, representing a year-over-year increase of approximately 213%. Net income before income taxes for the second quarter of 2025 was $69.3 million, a 24.2% increase year-over-year compared to $55.8 million for the second quarter of 2024.

FYCOMPA 2025 年第二季的產品淨收入為 3,430 萬美元，而 2024 年第二季為 3,650 萬美元。AGAMREE 2025 年第二季的產品淨收入為 2,740 萬美元，而 2024 年第二季為 870 萬美元，年增約 213%。2025 年第二季的稅前淨收入為 6,930 萬美元，與 2024 年第二季的 5,580 萬美元相比年增 24.2%。

We reported GAAP net income for the second quarter of 2025 of $52.1 million or $0.41 per diluted share. GAAP net income increased by 27.7% year-over-year compared to GAAP net income for the second quarter of 2024 of $40.8 million or $0.33 per diluted share.

我們報告的 2025 年第二季 GAAP 淨收入為 5,210 萬美元，即每股攤薄收益 0.41 美元。與 2024 年第二季 GAAP 淨收入 4,080 萬美元或每股稀釋收益 0.33 美元相比，GAAP 淨收入年增 27.7%。

Non-GAAP net income for the second quarter of 2025 was $86.4 million or $0.68 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 $7.6 million; income tax provision of $17.2 million and depreciation of $100,000.

2025 年第二季非 GAAP 淨收入為 8,640 萬美元，即每股攤薄收益 0.68 美元，其中不包括 GAAP 淨收入；與收購 FYCOMPA、AGAMREE 和 Ruzurgi 相關的無形資產攤銷 930 萬美元；股票薪酬費用 760 萬美元；所得稅撥備 1,720 萬美元。

This compares to non-GAAP net income for the second quarter of 2024 of $69.6 million or $0.56 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 $15.0 million; and depreciation of $100,000.

相較之下，2024 年第二季的非 GAAP 淨收入為 6,960 萬美元，即每股攤薄收益 0.56 美元，其中不包括 GAAP 淨收入；與收購 FYCOMPA、AGAMREE 和 Ruzurgi 相關的無形資產攤銷 930 萬美元；股票薪酬費用 440 萬美元；所得稅

Our year-to-date effective income tax rate through the first half of 2025 was 22.6% compared to 24.5% through the first half of 2024. The 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.

截至 2025 年上半年，我們的年度有效所得稅率為 22.6%，而 2024 年上半年為 24.5%。有效稅率受多種因素影響，包括任何特定期間行使的股票選擇權數量，並且可能在未來期間內波動。

Cost of sales expense was approximately $20.6 million in the second quarter of 2025 compared to $15.4 million in the second quarter of 2024 and consisted principally of royalties. As a reminder, AGAMREE royalties paid to the product licensor equal 5% of net sales up to $100 million, 7% of net sales in excess of $100 million and up to $200 million with additional increases as net sales increase beyond $200 million. The company is also required to make a $12.5 million sales-based milestone payment once AGAMREE's net product revenue reaches $100 million, that this milestone payment when earned, will be capitalized and amortized over the estimated remaining useful life of the asset. Further details on our royalty obligations for AGAMREE are disclosed in our Q2 2025 Form 10-Q.

2025 年第二季的銷售費用約為 2,060 萬美元，而 2024 年第二季為 1,540 萬美元，主要包括特許權使用費。提醒一下，AGAMREE 向產品授權人支付的特許權使用費相當於淨銷售額的 5%，最高可達 1 億美元；淨銷售額超過 1 億美元後，支付給產品授權人的特許權使用費相當於淨銷售額的 7%，最高可達 2 億美元；當淨銷售額超過 2 億美元時，特許權使用費還會進一步增加。一旦 AGAMREE 的淨產品收入達到 1 億美元，該公司還需要支付 1250 萬美元的基於銷售額的里程碑付款，該里程碑付款在獲得後將被資本化並在資產的預計剩餘使用壽命內攤銷。有關我們對 AGAMREE 的特許權使用費義務的更多詳細資訊已在我們的 2025 年第二季 10-Q 表中披露。

Research and development expenses were $4.4 million in the second quarter of 2025, up 46% from $3.0 million in the second quarter of 2024. Our R&D spending in the second quarter of 2025 was comprised mainly of costs to support our ongoing AGAMREE studies.

2025 年第二季研發費用為 440 萬美元，較 2024 年第二季的 300 萬美元成長 46%。我們 2025 年第二季的研發支出主要用於支持我們正在進行的 AGAMREE 研究的成本。

Selling, general and administrative or SG&A expenses for the second quarter of 2025 totaled $45.9 million compared to $40.7 million in the second quarter of 2024. The increase in SG&A expenses for the second quarter of 2025 was primarily driven by increased personnel costs, including the implementation of two dedicated sales forces for FIRDAPSE and AGAMREE, which became effective on April 1, 2025.

2025 年第二季的銷售、一般及行政費用（SG&A）總計 4,590 萬美元，而 2024 年第二季為 4,070 萬美元。2025 年第二季銷售、一般及行政費用的增加主要是由於人員成本增加，包括為 FIRDAPSE 和 AGAMREE 實施兩支專門的銷售隊伍，該措施於 2025 年 4 月 1 日生效。

As reported, at June 30, 2025, we had cash and cash equivalents of $652.8 million compared to $517.6 million at December 31, 2024. The increase in cash of $135.2 million was primarily driven by $131.3 million in cash generated from operations of the business, underscoring our continued focus on profit optimization and strong cash flow generation.

據報道，截至 2025 年 6 月 30 日，我們的現金和現金等價物為 6.528 億美元，而 2024 年 12 月 31 日為 5.176 億美元。現金增加 1.352 億美元，主要得益於業務營運產生的 1.313 億美元現金，凸顯了我們持續專注於利潤優化和強勁的現金流生成。

We believe our current funds, along with our anticipated continued generation of cash from operations, continue to provide us with the financial flexibility to fund our existing R&D programs, meet our potential contractual obligations and support our strategic initiatives, business development and portfolio expansion efforts, leading to long-term growth and value creation.

我們相信，我們目前的資金以及我們預期的持續經營現金流將繼續為我們提供財務靈活性，以資助我們現有的研發項目、履行我們潛在的合約義務並支持我們的策略計劃、業務發展和投資組合擴張努力，從而實現長期增長和價值創造。

More detailed information and analysis of our second-quarter 2025 financial performance may be found in our quarterly report on Form 10-Q, which was filed with the Securities and Exchange Commission yesterday, August 6, 2025, and can be found on the Investor Relations page on our website.

有關我們 2025 年第二季財務業績的更多詳細資訊和分析，請參閱我們於 2025 年 8 月 6 日向美國證券交易委員會提交的 10-Q 表季度報告，也可在我們網站的投資者關係頁面上找到。

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

這次，我會把電話轉回給里奇。

Thanks, Mike. As we look ahead, Catalyst remains focused on and confident in our ability to drive sustained growth and long-term value. We are executing a clear strategy rooted in a strong commercial foundation. Our focus and execution will continue to drive our performance and enable us to deliver on our commitments while making a meaningful difference for patients.

謝謝，麥克。展望未來，Catalyst 將繼續專注於推動持續成長和長期價值的能力並充滿信心。我們正在執行一項以強大商業基礎為基礎的明確策略。我們的重點和執行力將繼續推動我們的業績，並使我們能夠兌現承諾，同時為患者帶來有意義的改變。

Our teams are energized by the opportunities ahead, and we remain committed to delivering differentiated therapies with operational excellence and responsible growth. Thank you to our Catalyst teams for their unwavering dedication to patients and to all of you for your continued support. We look forward to continuing to execute our growth initiatives as we advance into the second half of 2025.

我們的團隊對未來的機會充滿活力，我們將繼續致力於透過卓越的營運和負責任的成長提供差異化的治療。感謝我們的 Catalyst 團隊對病人的堅定奉獻，也感謝大家的持續支持。我們期待在 2025 年下半年繼續執行我們的成長計畫。

I'll now turn the call back over to the operator. Thank you.

我現在將把電話轉回給接線生。謝謝。

(Operator Instructions) Samantha Semenkow, Citibank.

（操作員指示）花旗銀行的 Samantha Semenkow。

A couple first on FIRDAPSE. Can you speak more about your strategy for educating oncologists on the LEMS opportunity? And I'm curious what metrics are you using to track the success of the campaign?

FIRDAPSE 上的一對夫婦首次亮相。能否詳細談談您向腫瘤科醫師介紹 LEMS 機會的策略？我很好奇您使用什麼指標來追蹤活動的成功？

And then as you continue to focus on the oncology opportunity, when do you expect that segment of the business to begin representing a greater proportion of revenues versus historical experience? I believe in the past, you said it's about 20% to 25% of revenues come from oncology patients.

然後，當您繼續關注腫瘤學機會時，您預計該業務部門何時會開始佔據比歷史經驗更大的收入比例？我相信過去您說過大約 20% 到 25% 的收入來自腫瘤患者。

Thanks, Samantha. I appreciate the question. I'm going to turn that to Jeff. Jeff, do you want to take that one?

謝謝，薩曼莎。我很感謝你提出這個問題。我要把它交給傑夫。傑夫，你想拿那個嗎？

Sure. Sam, good to hear from you. As we noted in the script, I think the -- our approach to oncology and the addressable market there for cancer-associated LEMS, the first thing we really wanted to do was increase the -- provide frictionless testing for these patients. And we were able to make that happen where oncologists and all HCPs could order our no-cost VGCC antibody test within their office. So that shortened the time to the diagnostic.

當然。山姆，很高興收到你的來信。正如我們在劇本中提到的，我認為——我們對腫瘤學的方法以及癌症相關 LEMS 的潛在市場，我們真正想要做的第一件事是增加——為這些患者提供無摩擦測試。我們能夠實現這一目標，腫瘤學家和所有 HCP 都可以在他們的辦公室內訂購我們的免費 VGCC 抗體測試。這樣就縮短了診斷時間。

Secondly, the NCCN guideline changes. So you heard that in July '25, that -- those changes happen. So those were the first two steps, and now we're working on -- working with the group practices and trying to put together some arrangements with them for screening. So that's the third step.

其次，NCCN指南發生變化。所以你聽說在 25 年 7 月，這些變化發生了。這是前兩個步驟，現在我們正在與團體實踐合作，並嘗試與他們一起制定一些篩選安排。這是第三步。

How are we going to educate these physicians? We're focused on digital marketing and media. We're also doing congresses and conferences and publication plan, working with our medical team on that to do abstracts as well. So those are the things that we're doing to help educate physicians about this important opportunity to diagnose LEMS patients.

我們要如何教育這些醫生？我們專注於數位行銷和媒體。我們也正在製定代表大會、會議和出版計劃，並與我們的醫療團隊合作製作摘要。所以，我們正在做的事情就是幫助醫生了解診斷 LEMS 患者這一重要機會。

How we're going to track it? Is through increased VGCC antibody testing from oncologists. We're also tracking it, like you said, we anticipate an increase over time of our mix of patients that are cancer-associated LEMS patients. This year, our primary goal is to increase screening for cancer-associated LEMS patients. 90% of these patients are undiagnosed.

我們要如何追蹤它？是透過腫瘤學家增加的 VGCC 抗體測試。我們也在追蹤它，就像你說的，我們預計隨著時間的推移，與癌症相關的 LEMS 患者的數量會增加。今年，我們的主要目標是增加癌症相關 LEMS 患者的篩檢。其中 90% 的患者未得到診斷。

So if we can help these patients get diagnosed, then we anticipate a larger addressable market that we should start seeing an increase in that percentage in 2026 and beyond. So that's how we're looking at this, Sam.

因此，如果我們能夠幫助這些患者得到診斷，那麼我們預計潛在市場會更大，我們應該在 2026 年及以後開始看到這一比例的成長。這就是我們看待這個問題的方式，山姆。

That's super helpful. Just one follow-up for me. You mentioned on the prepared remarks that there's a Phase 1 DMD switching study from prednisone or from EMFLAZA to AGAMREE. I'm wondering how do you plan to leverage this data as you commercialize AGAMREE or is it for additional studies that you're planning? Just any context you could share there would be very helpful.

這非常有幫助。對我來說只有一個後續行動。您在準備好的評論中提到，有一項從潑尼鬆或從 EMFLAZA 到 AGAMREE 的 1 期 DMD 轉換研究。我想知道您在將 AGAMREE 商業化時計劃如何利用這些數據，或者您是否計劃將其用於其他研究？只要您分享任何背景信息，就會非常有幫助。

Thanks, Sam. I'm going to turn that over to Bill Andrews, our Chief Medical Officer. Obviously, we're looking at -- we'll be looking for the data, and I think a lot of that will determine how we leverage it. So obviously, the study is ongoing. So I think it becomes a little challenging to answer your question directly.

謝謝，山姆。我將把這個任務交給我們的首席醫療官比爾安德魯斯 (Bill Andrews)。顯然，我們正在研究——我們將尋找數據，我認為其中許多將決定我們如何利用它。顯然，這項研究仍在進行中。因此我認為直接回答你的問題有點困難。

But Will, do you want to take that?

但是威爾，你想接受這個嗎？

Yes. Thank you, Rich, and hello, Sam. The study is an exploratory trial, and we are looking forward to seeing data later this year and then early into 2026. And that data will describe -- be descriptive and will help indicate whether we need to do future studies to further delineate potential switching, et cetera.

是的。謝謝你，Rich，你好，Sam。這項研究是一項探索性試驗，我們期待在今年稍後以及 2026 年初看到數據。這些數據將具有描述性，並有助於表明我們是否需要進行未來的研究來進一步描述潛在的轉換等等。

Joon Lee, Truist Securities.

Joon Lee，Truist Securities。

Congrats on the quarter. This is Asim on for Joon. Just a couple from us. So what impact are you seeing on FIRDAPSE and AGAMREE uptake following the increase in sales force size that became effective on April 1?

恭喜本季取得佳績。這是 Asim 為 Joon 上場。距離我們只有幾對。那麼，自 4 月 1 日起，銷售隊伍規模擴大後，您認為對 FIRDAPSE 和 AGAMREE 的採用有何影響？

And then as a follow-up on FIRDAPSE, just help us think about this growth over the next few quarters. Should we still be expecting a mid-teens growth rate on a year-over-year basis?

然後作為 FIRDAPSE 的後續行動，幫助我們思考未來幾季的成長。我們是否仍應預期年增率將達到十五六倍？

Thanks, Asim. I think, again, it's -- Jeff is going to answer that question. So thanks for the question. Jeff?

謝謝，阿西姆。我認為，傑夫會再次回答這個問題。感謝您的提問。傑夫？

Thanks for the question. Like you said, we deployed dedicated sales forces for both AGAMREE and FIRDAPSE in April, so beginning in the second quarter. What we've seen, it's still too early to tell. But based on performance, you can see that there are increased engagements that have taken place. We are -- also deeper relationships now within these accounts and also more frequent interactions with many of the healthcare providers and our targeted physicians.

謝謝你的提問。正如您所說，我們從 4 月開始為 AGAMREE 和 FIRDAPSE 部署了專門的銷售人員，也就是從第二季開始。就我們所看到的情況而言，現在下結論還為時過早。但根據表現，你可以看到參與度增加。我們現在與這些帳戶的關係也更加深厚，與許多醫療保健提供者和我們的目標醫生的互動也更加頻繁。

We're also seeing it from our leading indicators. As I mentioned on the call, with FIRDAPSE, we're exceeding -- or meeting and exceeding the enrollments thus far in July. So very strong, and we also have a strong pipeline there. With AGAMREE too, we've seen the 231 unique physicians that have enrolled since launch. So that also increased from the second quarter.

我們也從領先指標中看到了這一點。正如我在電話中提到的那樣，透過 FIRDAPSE，我們已經超過——或者說達到並超過了 7 月迄今為止的入學人數。非常強大，而且我們在那裡也有強大的管道。同樣，在 AGAMREE 上，我們也看到自推出以來已有 231 名醫生註冊。因此，這個數字也從第二季開始增加。

So very, very satisfied with the results thus far, and we'll continue to track that moving forward.

我們對目前的結果非常非常滿意，我們將繼續追蹤這一進展。

As far as performance for FIRDAPSE and what you should expect. We reiterated guidance for the balance of this year. And also, like I just mentioned, the strong leading indicators. So we do believe in our business and the foundation of our business for FIRDAPSE, so we expect continued growth.

至於 FIRDAPSE 的效能以及您應該期待什麼。我們重申了今年餘下的業績指引。而且，正如我剛才提到的，領先指標強勁。因此，我們確實相信我們的業務以及 FIRDAPSE 的業務基礎，因此我們期待持續成長。

Jason Gerberry, Bank of America.

美國銀行的 Jason Gerberry。

A couple for me. Just given all that's going on with ELEVIDYS in the, I guess, nonambulatory group of patients and some of the commentary earlier in the year around queuing, I'm just kind of wondering how that impacts the quarterly kinetics of AGAMREE uptake, if at all?

對我來說是一對。考慮到 ELEVIDYS 在非臥床患者群體中發生的所有情況以及今年早些時候有關排隊的一些評論，我只是想知道這會對 AGAMREE 吸收的季度動力學產生什麼影響（如果有的話）？

And then with FIRDAPSE, I'm wondering if you could just maybe comment on sort of the time lag for the NCCN recommendation to have more of an impactful commercial impact.

然後對於 FIRDAPSE，我想知道您是否可以評論一下 NCCN 建議產生更大商業影響的時間延遲。

Jason, thanks so much. So from a commercial perspective, we'll take the ELEVIDYS question. We'll turn it over to Jeff. And if they're more of a clinical, we'll have Will jump in on that.

傑森，非常感謝。因此，從商業角度來看，我們將回答 ELEVIDYS 的問題。我們將把它交給傑夫。如果他們更注重臨床治療，我們就會讓威爾參與其中。

But Jeff, do you want to take that one first?

但是傑夫，你想先拿那個嗎？

Thanks for the question, Jason. And with AGAMREE and steroids specifically, they are the foundation of treatment for these boys living with DMD. So the impact of the unfortunate news about ELEVIDYS really hasn't impacted much the use of steroids. And we do strongly believe that AGAMREE is the steroid of choice.

謝謝你的提問，傑森。具體來說，AGAMREE 和類固醇是治療這些患有 DMD 的男孩的基礎。因此，有關 ELEVIDYS 的不幸消息實際上並沒有對類固醇的使用產生太大影響。我們堅信 AGAMREE 是最佳的類固醇選擇。

Will, do you want to jump in there on the clinical?

威爾，你想參加臨床研究嗎？

Happy to, Rich. Thank you. We see the new additions to the NCCN guidelines as critical to help users for ELEVIDYS.

很高興，Rich。謝謝。我們認為 NCCN 指南的新內容對於幫助 ELEVIDYS 使用者至關重要。

For DMD.

對於 DMD。

Yes, I'm sorry. And the second question was on NCCN.

是的，我很抱歉。第二個問題是關於 NCCN 的。

Yes, yes.

是的，是的。

On ELEVIDYS, I would add medically that I agree with Jeff from the perspective of that being an entirely different modality of treatment. And as Jeff said, it's unfortunate as to what's happened, but we still see, of course, corticosteroids and vamorolone as foundational treatment for these kids that is unaffected, we believe, by what's happened with ELEVIDYS.

關於 ELEVIDYS，我想從醫學角度補充一點，我同意 Jeff 的觀點，因為這是一種完全不同的治療方式。正如傑夫所說，發生的事情令人遺憾，但我們仍然認為，皮質類固醇和瓦莫洛酮是這些孩子的基礎治療方法，我們相信，它們不會受到 ELEVIDYS 事件的影響。

To answer your second question on the NCCN guidelines, we see that as critical to helping educate oncologists, clinical oncologists on the disease and very importantly, on how to better diagnose the disease and that amifampridine is a critical option for them in the treatment.

回答您關於 NCCN 指南的第二個問題，我們認為這對於幫助腫瘤學家、臨床腫瘤學家了解這種疾病至關重要，而且非常重要的是，如何更好地診斷這種疾病，而氨苯吡啶是他們治療的關鍵選擇。

And I'll just add, the way we see the cadence working out, Jeff and the team have done a really good job of getting this frictionless testing in there, which we believe is the first step. So that the physician can order the test and the report will come to their office. Prior to this patient had to actually go to a remote site, get a test and then get the test results themselves. So that's the first step.

我還要補充一點，從我們看到的節奏來看，傑夫和他的團隊在無摩擦測試方面做得非常好，我們相信這是第一步。這樣醫生就可以安排檢查，並將報告寄到他們的辦公室。在此之前，患者必須親自前往遠端站點進行測試，然後自行取得測試結果。這是第一步。

The NCCN guideline really is the bridge to care pathways within the practice. So as we get this out there, and it was just released, obviously, last week, we get this out there, the opportunity then is to work through those oncology practices get these NCCN guidelines embedded as the standard and NCCN is the true standard within oncology, get that embedded and then begin talking to the group practices about how they want to use it and how we can be supportive.

NCCN 指南確實是實務中護理途徑的橋樑。因此，當我們將其發佈出去時，顯然，它是上週剛發布的，我們將其發佈出去，然後的機會就是透過那些腫瘤學實踐將這些 NCCN 指南嵌入為標準，而 NCCN 是腫瘤學中的真正標準，將其嵌入，然後開始與團體實踐討論他們如何使用它以及我們如何提供支持。

And then as Jeff mentioned earlier on an earlier question, those educational efforts working hand-in-hand with the GPOs and then beyond the GPOs as well become important. Jeff?

正如 Jeff 之前在回答問題時提到的那樣，與 GPO 攜手合作、超越 GPO 的教育工作也變得非常重要。傑夫？

Thanks, Rich. And Jason, to your question, when do we expect this to hit commercial and increase some of the net revenue. The way we look at this, again, is increased screening this year and then next year with more patients thankfully being diagnosed, the addressable market increases. And then that can help with the treatment with FIRDAPSE. So we expect that to take place probably in the second half of next year and beyond is when we start seeing converting some of these diagnosed patients onto FIRDAPSE therapy.

謝謝，里奇。傑森，對於你的問題，我們預計什麼時候這款產品能夠實現商業化並增加部分淨收入。我們再次看待這個問題的方式是，今年篩檢力度加大，然後明年隨著更多患者幸運地得到診斷，潛在市場將會增加。這可以幫助 FIRDAPSE 治療。因此，我們預計這可能會在明年下半年及以後發生，屆時我們將開始看到部分確診患者轉而接受 FIRDAPSE 治療。

Hopefully that helps.

希望有幫助。

Leland Gershell, Oppenheimer & Co.

Leland Gershell，奧本海默公司

Just one from us, just on OpEx, maybe for Michael. Just wondering, given the realignment in the sales for FIRDAPSE and AGAMREE affected a few months ago. Just wondering how you see SG&A expense rolling forward? It looks like it was pretty flattish from Q1 to Q2. Just wondering if we should see a bit of a bump and the increase into the second half.

我們只有一個，僅關於 OpEx，也許適合邁克爾。只是好奇，考慮到幾個月前 FIRDAPSE 和 AGAMREE 的銷售調整受到影響。只是想知道您如何看待銷售、一般和行政費用的滾動？從第一季到第二季度，情況似乎相當穩定。我只是想知道我們是否應該看到下半年出現一些波動和增長。

Thanks, Leland, and I hope all is well. So as you know, we did not quantify SG&A guidance. We did talk qualitatively at the beginning of the year. Some of the folks that we had hired were in, obviously, a little before April 1. So I think the flat is a little bit to what we'd expect.

謝謝，Leland，希望一切都好。因此，如您所知，我們沒有量化銷售、一般和行政費用指導。我們確實在年初進行過定性討論。顯然，我們僱用的一些人是在 4 月 1 日之前到職的。所以我認為這個平台與我們預期的有點一致。

But to your point, with the acceleration of the NCCN guidelines, I do think that we will see a little bit of an uptick in the second half.

但正如您所說，隨著 NCCN 指南的加速推進，我確實認為我們將在下半年看到一點上升。

Luke Herrmann, Baird.

盧克·赫爾曼，貝爾德。

Two from me. First, on the Change Health disruption. I was wondering if you could share your thoughts on whether all the loose ends have been closed here or if there's potential for any future anomalies.

我有兩個。首先，關於變革健康中斷。我想知道您是否可以分享您的想法，這裡是否已經解決了所有未解決的問題，或者是否存在未來出現任何異常的可能性。

And then second, recent language from the administration on pharma tariffs, which has obviously been sort of constantly evolving. I see that FIRDAPSE is manufactured in the US and Canada. And I think you've had a process underway to consider addition of third-party manufacturers for AGAMREE. I guess, has there been any recent evolution in thinking around the manufacturing strategy?

其次，政府最近關於藥品關稅的措詞顯然一直在不斷變化。我看到 FIRDAPSE 是在美國和加拿大製造的。我認為你們已經在考慮為 AGAMREE 增加第三方製造商。我想，最近在製造業策略方面的思考有什麼進展嗎？

Thanks, Luke. Thanks for the questions. So the Change Healthcare issue, the disruption we saw there really was -- as we mentioned in the script, really was a pull sort of a push into the second quarter of 2024. We believe and we see the natural cadence of FIRDAPSE returning post June of 2024. And now we believe that effect is washed through the system.

謝謝，盧克。謝謝你的提問。因此，變革醫療保健問題，即我們所看到的混亂，正如我們在腳本中提到的那樣，實際上是對 2024 年第二季度的拉動。我們相信並且我們看到 FIRDAPSE 將在 2024 年 6 月後自然回歸。現在我們相信這種影響已經透過系統消除了。

So we're very confident about that because we can see -- because of our specialty pharmacy relationship, we can see what's happening there at a very granular level.

因此，我們對此非常有信心，因為我們可以看到——由於我們與專業藥房的關係，我們可以非常細緻地了解那裡發生的事情。

And you may remember, in our second -- first quarter call in May last year, we mentioned that we saw it the day it began to happen. So we were able to take rapid action. So having that specialty pharmacy is a real benefit for us.

您可能還記得，在去年 5 月的第二季（第一季）電話會議上，我們提到我們在它開始發生的那天就看到了它。因此我們能夠迅速採取行動。因此，擁有這家專業藥局對我們來說是真正的好處。

On the tariff side, you are correct. When you think about the volume of sales based on our guidance, about 66% of sales come from FIRDAPSE as it relates to our guidance for the year. And we feel really well protected there. And you are also correct on working with third-party manufacturers for AGAMREE, and transitioning that to the US.

關於關稅方面，您說得對。當您考慮基於我們指導的銷售量時，大約 66% 的銷售額來自 FIRDAPSE，因為這與我們今年的指導相關。我們在那裡感覺受到了很好的保護。您關於與第三方製造商合作開發 AGAMREE 並將其轉移到美國的建議也是正確的。

And we were working on that well before this current administration came into play. So we're confident we'll be able to do that in the near term, but it's still going to take a while to actually validate the plant. But we feel really good about the opportunity to shield that from tariffs as well in the mid- to long term because of the ability to move it to the US.

在本屆政府上台之前，我們就已經開始著手這項工作了。因此，我們有信心在短期內實現這一目標，但實際驗證工廠仍需要一段時間。但由於能夠將其轉移到美國，我們對在中長期內保護其免受關稅影響的機會感到非常高興。

Jennifer Kim, Cantor.

珍妮佛金（Jennifer Kim），領唱。

Maybe to start with FIRDAPSE. Are there any -- is there any color you can give around the average dose you've reached for the quarter and maybe expectations on that growth for the rest of the year? And then anything you can say about discontinuation rates for the quarter?

也許從 FIRDAPSE 開始。您能否透露本季達到的平均劑量以及今年剩餘時間的成長預期？那麼，您能透露一下本季的停產率嗎？

Jeff, do you want to take that?

傑夫，你想接受這個嗎？

Sure. Thanks, Rich. And yes, the -- first, I'll answer the discontinuation rates. Discontinuation rates were below 20%, the annual discontinuation rate of 20% for FIRDAPSE. So hopefully, were you asking about -- Jennifer, were you asking about FIRDAPSE specifically on discontinuation?

當然。謝謝，里奇。是的，首先，我來回答停藥率的問題。停藥率低於 20%，FIRDAPSE 的年停藥率為 20%。所以希望您問的是——詹妮弗，您問的是有關 FIRDAPSE 的具體停產情況嗎？

Yes.

是的。

Okay. So less than 20% annual discontinuation. And what was your first question again?

好的。因此每年的停產率不到20%。您第一個問題是什麼？

Dose.

劑量。

The dose. So since the label expansion in June of last year, we have seen significant increase to the average daily dose, approximately about 4 milligrams, which is what we had expected. So we -- the dose is performing according to what we had expected.

劑量。因此，自去年 6 月標籤擴展以來，我們看到平均每日劑量顯著增加，約為 4 毫克，這正是我們預期的。因此，劑量表現符合我們的預期。

Okay. That's helpful. And maybe a question on FYCOMPA. I think right now, Teva is the only generic for the oral tablet that's launched. I just want to clarify, do they have a 100-day exclusivity?

好的。這很有幫助。也許還有一個關於 FYCOMPA 的問題。我認為目前，Teva 是唯一推出的口服藥片仿製藥。我只是想澄清一下，他們有 100 天的獨家經營權嗎？

And I asked because I wonder whether guidance could prove to be on the conservative side for that product.

我之所以問這個問題，是因為我想知道該產品的指導是否偏向保守。

So Teva is the only player in the space right now for a generic. They have 180-day exclusivity. As to whether or not the guidance is too conservative. So far, as we mentioned in the call, we've booked $70 million in sales. Our guidance is $90 million to $95 million.

因此，Teva 是目前該領域唯一一家擁有仿製藥的公司。他們擁有 180 天的獨家經營權。至於該指導是否過於保守。到目前為止，正如我們在電話會議中提到的，我們的銷售額已達 7000 萬美元。我們的指導價是 9,000 萬至 9,500 萬美元。

However, we think that there's an opportunity to be cautious here because this is a significant risk. If there is another generic player that could come into the market, and they have -- we have not seen them yet. And so we're taking what we believe is a prudent position for the balance of the year.

然而，我們認為這是一個需要謹慎的機會，因為這是一個重大風險。如果有另一個通用公司可以進入市場，而且他們已經進入了——我們還沒有看到他們。因此，我們認為，對於今年的剩餘時間，我們將採取謹慎的立場。

And we will expect generic -- additional generic competitors by mid-December. And as you know, Jennifer, sometimes they can load the channel into November. And so that could affect our ex-factory sales. So we believe this is a prudent approach to the forecast.

我們預計到 12 月中旬將會出現更多的仿製藥競爭對手。詹妮弗，你知道，有時他們可以將頻道加載到 11 月。這可能會影響我們的出廠銷售。因此我們認為這是一種謹慎的預測方法。

Sudan Loganathan, Stephens Inc.

蘇丹洛加納森史蒂芬斯公司

Catalyst Pharma team, congrats on another strong quarter. First one, I want to ask how you view the potential for PTCT's Translarna FDA approval for DMD treatment to affect how AGAMREE will continue to fit in, retain market share and continue to grow in this space?

Catalyst Pharma 團隊，恭喜你們又一個強勁的季度。首先，我想問一下，您如何看待 PTCT 的 Translarna 獲得 FDA 批准用於 DMD 治療的可能性，這將影響 AGAMREE 如何繼續適應、保持市場份額並繼續在該領域發展？

And then secondly, what is your current stance on near-term strategic initiatives to offset FYCOMPA's LOE? Are there any ongoing discussions with potential partners regarding a commercial stage asset or a late-stage pipeline asset?

其次，您目前對抵銷 FYCOMPA 的 LOE 的近期策略舉措持什麼立場？是否正在與潛在合作夥伴討論商業階段資產或後期管道資產？

I think appropriate to turn that to Jeff for the first part.

我認為第一部分交給傑夫比較合適。

Yes, Sudan. Just like -- similar to ELEVIDYS, the way we look at this, is AGAMREE and steroids are the finest treatment. Translarna does not impact that at all. So we just continue to feel strongly about AGAMREE's potential moving forward because we do believe it is the best and differentiated steroid out there for boys living with DMD. And as far as --

是的，蘇丹。就像——與 ELEVIDYS 類似，我們認為 AGAMREE 和類固醇是最好的治療方法。Translarna 對此完全沒有影響。因此，我們繼續強烈相信 AGAMREE 未來的潛力，因為我們確實相信它是目前針對患有 DMD 的男孩的最佳和差異化類固醇。並且就--

Well, sorry, Sudan, could you just repeat your question? I lost you for a second. Repeat the second question.

好吧，抱歉，蘇丹，你能重複你的問題嗎？我有一秒鐘失去了你。重複第二個問題。

Yes. The second one was just on your current stance on the near-term strategic initiatives to offset the FYCOMPA LOE, if there's any ongoing discussions with either commercial stage asset or late-stage pipeline asset that you're kind of leaning towards in this market environment? Or how you're viewing that going forward?

是的。第二個問題是關於您目前對抵銷 FYCOMPA LOE 的近期策略舉措的立場，在這種市場環境下，您是否正在就商業階段資產或後期管道資產進行任何討論？或者您如何看待未來的發展？

Thanks for clarifying. So on our business development front as we look to offset -- we think of our business into 2 elements, right? The business development front, bringing in new assets. We are incredibly busy. We believe this is probably one of the best buying environments we've seen in the last 15 or 20 years.

謝謝澄清。因此，在我們尋求抵銷的業務發展方面，我們將業務分為兩個要素，對嗎？業務發展前沿，引進新資產。我們非常忙。我們相信這可能是過去 15 或 20 年來我們見過的最佳購買環境之一。

And our team is assessing multiple opportunities at any point in time.

我們的團隊隨時都在評估多種機會。

And as mentioned on previous calls, we continue to see 80% of the opportunities coming to us inbound. So other companies will approach us or other parties will approach us about opportunities. So we're really excited about building -- continuing to build the portfolio.

正如我們在之前的電話會議中提到的那樣，我們仍然看到 80% 的機會來自外部。因此，其他公司或其他方會與我們聯繫，尋求機會。因此，我們對建構——繼續建立投資組合感到非常興奮。

In addition to that, a second pillar of our business is life cycle management. And when we talk about the opportunity around AGAMREE, looking for opportunities to better understand the molecule and how it might play in the orphan and rare space, we continue to do that work. And on the commercial front in diversifying our opportunities with FIRDAPSE, we believe the oncology play and the dedicated sales force can continue to work to offset the loss of FYCOMPA.

除此之外，我們業務的第二個支柱是生命週期管理。當我們談論圍繞 AGAMREE 的機會時，我們會尋找機會更好地了解該分子以及它在孤兒藥和稀有藥領域中的作用，我們會繼續進行這項工作。在商業方面，透過與 FIRDAPSE 合作實現機會多元化，我們相信腫瘤學業務和專門的銷售團隊可以繼續努力彌補 FYCOMPA 的損失。

Thank you. Ladies and gentlemen, that then brings an end to our question-and-answer session. Thank you for joining today's conference. You may now disconnect your lines.

謝謝。女士們、先生們，我們的問答環節到此結束。感謝大家參加今天的會議。現在您可以斷開線路了。