Catalyst Pharmaceuticals Inc (CPRX) 2024 Q2 法說會逐字稿

Greetings, and welcome to the Catalyst Pharmaceuticals' second quarter 2024 financial results conference Call. At this time, all participants are in a listen-only mode. A brief question-and-answer session will follow the formal presentation. (Operator Instructions) As a reminder, this conference is being recorded.

您好，歡迎您參加 Catalyst Pharmaceuticals 2024 年第二季財務業績電話會議。此時，所有參與者都處於只聽模式。正式演講後將舉行簡短的問答環節。（操作員指示）謹此提醒，本次會議正在錄製中。

It is now my pleasure to introduce your host, Michael Kalb, CFO. Thank you, Michael. You may begin.

現在我很高興向您介紹主持人、財務長 Michael Kalb。謝謝你，麥可。你可以開始了。

Thank you. Good morning, everyone, and thank you for joining our conference call to discuss Catalyst's second quarter 2024 financial results and business highlights. Richard Daly, President and CEO will be leading 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 $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 2024 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，我和我們的首席商務官傑弗裡·德爾·卡門 (Jeffrey Del Carmen) 也將出席。此外，我們的營運長兼 $Scientific 長 Steven 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. 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.

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。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 second quarter 2024 quarterly report on Form 10-Q which we filed yesterday, August 7, 2024 with the SEC.

這些前瞻性陳述僅應與我們向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2023 年年度報告以及2024 年第二季的10-K 表格中描述的風險因素我們於昨天（2024 年 8 月 7 日）向 SEC 提交的 10-Q 表格季度報告。

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

這時候，我會把電話轉給 Rich。

Thanks Mike, and thank you all for joining today's call. Today we'll review our second quarter 2024 corporate performance, provide an update on our regulatory and strategic growth initiatives, dive deeper into individual product performance, and conclude with a comprehensive overview of our financial results.

謝謝麥克，也謝謝大家參加今天的電話會議。今天，我們將回顧 2024 年第二季度的企業業績，提供有關監管和戰略增長計劃的最新信息，更深入地了解單個產品的業績，並全面概述我們的財務業績。

In the second quarter 2024, we achieved outstanding results across our commercial portfolio with total revenues of $122.7 million, a 23.2% increase over Q2 2023, underscored by a record organic revenue growth from FIRDAPSEE. The successful ongoing commercialization of AGAMREE for Duchenne’s Muscular Dystrophy, and the continued solid performance of FYCOMPA.

2024 年第二季度，我們的商業投資組合取得了出色的業績，總收入達到 1.227 億美元，比 2023 年第二季度增長 23.2%，FIRDAPSEE 創紀錄的有機收入增長凸顯了這一點。用於治療杜氏肌肉營養不良症的 AGAMREE 持續成功商業化，以及 FYCOMPA 持續穩定的表現。

We are reiterating our full year 2024 net product guidance for both FIRDAPSEE and FYCOMPA. AGAMREE continues to deliver strong performance and we are updating our AGAMREE full year 2024 net product revenue guidance from the $25 million to $30 million range we provided earlier this year to the $35 million to $40 million. As a result of our strong performance, we are also updating our full year 2024 total revenue financial guidance.

我們重申 FIRDAPSEE 和 FYCOMPA 的 2024 年全年淨產品指引。AGAMREE 繼續提供強勁的業績，我們正在將 AGAMREE 2024 年全年淨產品收入指引從今年稍早提供的 2,500 萬美元至 3,000 萬美元範圍更新為 3,500 萬美元至 4,000 萬美元。由於我們的強勁表現，我們也更新了 2024 年全年總收入財務指引。

Previously, we guided to a full year total revenue range of between $455 million to $475 million. Due to the solid performance of both FIRDAPSEE and FYCOMPA and the outstanding uptake of AGAMREE, we now believe that we will achieve total revenue at the upper-end of the range. Jeff will provide further details about our commercial performance during this discussion, and Mike will give a deeper dive into the financials later in this call.

此前，我們預計全年總收入在 4.55 億美元至 4.75 億美元之間。由於 FIRDAPSEE 和 FYCOMPA 的穩健表現以及 AGAMREE 的出色採用，我們現在相信我們將實現總收入處於該範圍的上限。傑夫將在本次討論中提供有關我們商業業績的更多詳細信息，邁克將在本次電話會議稍後深入探討財務狀況。

Now, let's review our regulatory, clinical and business development milestones. First up regulatory, in May, we received US approval for FIRDAPSEE's expanded maximum daily dose of 100 milligrams for treating LEMS up from the previous 80 milligrams. This milestone enhances treatment flexibility for healthcare professionals, aligning with our mission to address the evolving patient needs and optimize outcomes for the LEM patient community. As awareness of this new dosing option grows among patients and physicians, we anticipate a gradual increase in FIRDAPSEE daily dosing over the coming quarters.

現在，讓我們回顧一下我們的監管、臨床和業務發展里程碑。首先是監管，5 月份，我們獲得了美國批准，將 FIRDAPSEE 治療 LEMS 的每日最大劑量從先前的 80 毫克擴大到 100 毫克。這項里程碑增強了醫療保健專業人員的治療靈活性，符合我們滿足不斷變化的患者需求並優化 LEM 患者社區治療結果的使命。隨著患者和醫生對這種新劑量選擇的認識不斷增強，我們預計未來幾季 FIRDAPSEE 的每日劑量將逐漸增加。

In parallel, we continued to actively collaborate with our partner in Japan, DyDo Pharma, as we await the next regulatory milestones for FIRDAPSEE, the NDA decision from Japanese regulatory authorities, which is expected at the end of Q3 or the beginning of Q4 this year. In clinical, the summit study, a five year initiative to collect long term safety and quality of life data for AGAMREE, continues to advance. The study, once initiated, will gather long term patient safety and quality of life data, offering a deeper understanding of the product's potential benefit for patients.

同時，我們繼續與日本合作夥伴 DyDo Pharma 積極合作，等待 FIRDAPSEE 的下一個監管里程碑，即日本監管機構的 NDA 決定，預計將於今年第三季末或第四季初做出。在臨床方面，高峰會研究是一項為期五年的舉措，旨在收集 AGAMREE 的長期安全性和生活品質數據，該研究仍在繼續進行中。該研究一旦啟動，將收集長期患者安全和生活品質數據，從而更深入地了解該產品對患者的潛在益處。

Site identification and initiation are on track. Annual analysis of patient data is planned, potentially leading to future FDA submissions for updated product labeling, furthering our commitment to enhancing patient care and providing the latest safety information.

網站識別和啟動正在按計劃進行。計劃對患者數據進行年度分析，這可能會導致未來向 FDA 提交更新的產品標籤，進一步履行我們對加強患者護理和提供最新安全資訊的承諾。

Moving to business development subsequent to the end of Q2 in July 2024, we entered into a license agreement with Kye Pharma for them to commercialize AGAMREE in Canada. As Kye already holds commercial rights for FIRDAPSEE in Canada. Kye expects to submit an application to Health Canada for regulatory approval of AGAMREE in early 2025. Our growing partnership with Kye is a great example of our commitment to improving access for patients in targeted ex-US markets.

2024 年 7 月第二季末後轉向業務開發，我們與 Kye Pharma 簽訂了授權協議，讓他們在加拿大將 AGAMREE 商業化。由於 Kye 已經擁有 FIRDAPSEE 在加拿大的商業權利。Kye 預計於 2025 年初向加拿大衛生部提交 AGAMREE 監管批准的申請。我們與 Kye 不斷發展的合作關係是我們致力於改善美國以外目標市場患者的就醫機會的一個很好的例子。

In our efforts to build our portfolio, we hear consistent feedback from potential partners. First, our strong commercial execution across a diverse portfolio makes US an excellent partner for companies looking for a US partner. Second, our launch capabilities engender trust with potential partners and are an attractive selling point. Third, our strong balance sheet makes us a formidable negotiator in acquiring new assets.

在我們努力建立我們的產品組合的過程中，我們聽到了來自潛在合作夥伴的一致回饋。首先，我們在多元化投資組合中強大的商業執行力使美國成為尋找美國合作夥伴的公司的優秀合作夥伴。其次，我們的發布能力可以贏得潛在合作夥伴的信任，並且是一個有吸引力的賣點。第三，我們強大的資產負債表使我們成為收購新資產的強大談判者。

Our strategy remains focused on delivering value for our stakeholders by continuing to execute our buy and build plan. With the ongoing commercialization of AGAMREE, we continue to prove our capabilities in identifying, integrating and launching products that deliver value for patients, providers and payers.

我們的策略仍然專注於透過繼續執行我們的購買和建設計劃為我們的利害關係人創造價值。隨著 AGAMREE 的不斷商業化，我們不斷證明我們在識別、整合和推出為患者、提供者和付款人創造價值的產品方面的能力。

We are confident in our ability to leverage the focused execution of our strategic initiatives while maintaining our commitment to advancing patient care, unlocking growth potential and delivering value for stakeholders. Underpinned by our exemplary track record and dedicated team, we believe that we are ideally positioned to drive further growth and achieve sustained success.

我們對集中執行策略性措施的能力充滿信心，同時繼續致力於推進患者護理、釋放成長潛力並為利益相關者創造價值。在我們模範的業績記錄和敬業的團隊的支持下，我們相信我們處於推動進一步成長並取得持續成功的理想位置。

To summarize, we remain focused on our threefold growth strategy, outstanding execution, portfolio diversification and ex-US expansion through partnerships. First, let's focus on outstanding execution. Our consistent performance continues to demonstrate our commitment to improving the lives of patients we serve and delivering on the promise of growing the company.

總而言之，我們仍然專注於我們的三重成長策略、出色的執行力、投資組合多元化以及透過合作夥伴關係進行美國以外的擴張。首先，讓我們專注於出色的執行力。我們一貫的表現繼續表明我們致力於改善我們所服務的患者的生活並兌現公司發展的承諾。

Second, portfolio Diversification our expanded focus in orphan therapeutics and adjacent orphan assets has yielded increased opportunities and our team is rigorously evaluating each for fit and value. We continue in our tireless efforts to bring on new products and we are excited about our prospects. We remain opportunistic in pursuing the right deals at the right time and we are well positioned to leverage our strong balance sheet to capitalize on accretive or near accretive opportunities.

其次，投資組合多元化，我們對孤兒療法和相關孤兒資產的擴大關注帶來了更多的機會，我們的團隊正在嚴格評估每一項的適合性和價值。我們將繼續不懈地努力推出新產品，我們對我們的前景感到興奮。我們仍然保持機會主義，在正確的時間尋求正確的交易，並且我們處於有利地位，可以利用我們強大的資產負債表來利用增值或接近增值的機會。

Third, targeted ex-US partnerships. We are building alliances with companies that can leverage our portfolio outside the US in high value markets to bring improved care options to patients and providers. As we add to our portfolio, we expect to build our global presence through these cost effective partnerships. In the second quarter, we successfully navigated an increasingly dynamic business environment delivered on our top line across the portfolio, successfully drove the commercialization of the AGAMREE and expanded our commercial footprint. We will continue to work for the remainder of the year to deliver on our strategy, executing and delivering our plan, diversifying our portfolio and expanding our ex-US partnerships.

第三，有針對性的美國以外的夥伴關係。我們正在與能夠在美國以外的高價值市場中利用我們的產品組合的公司建立聯盟，為患者和提供者提供更好的護理選擇。隨著我們增加產品組合，我們希望透過這些具有成本效益的合作夥伴關係來建立我們的全球影響力。第二季度，我們成功駕馭了整個產品組合中日益活躍的業務環境，成功推動了 AGAMREE 的商業化並擴大了我們的商業足跡。我們將在今年剩餘的時間裡繼續努力實現我們的策略，執行和交付我們的計劃，使我們的產品組合多樣化並擴大我們在美國以外的合作夥伴關係。

Now I'll turn the call to Jeff, who will provide a commercial update.

現在我將把電話轉給傑夫，他將提供商業更新。

Thanks Rich and good morning everyone. We are incredibly pleased to report the tremendous quarterly performance across our entire commercial portfolio. These results showcase our strategic focus and operational excellence that drive our commercial success. As Rich mentioned, Catalyst had an excellent Q2 driven by the combination of sustained organic growth of FIRDAPSEE, stable revenues from FYCOMPA and the strong commercial launch of AGAMREE.

謝謝里奇，大家早安。我們非常高興地報告我們整個商業產品組合的巨大季度業績。這些結果展示了我們的策略重點和卓越運營，推動了我們的商業成功。正如 Rich 所提到的，Catalyst 在第二季度表現出色，這得益於 FIRDAPSEE 的持續有機成長、FYCOMPA 的穩定收入以及 AGAMREE 的強勁商業推出。

Q2 total net revenues of $122.7 million, a 23.2% growth versus the same quarter last year, positions catalysts well to achieve the upper end of our previously provided combined 2020 total revenue guidance of between $455 million to $475 million. I will now provide a breakdown of Q2 net revenues by product. First, let me start with FIRDAPSEE performance. Q2 net sales of $77.4 million represent a 19.2% increase over the same quarter last year, a direct result of steady new patient enrollments, an annual discontinuation rate below the forecasted 20% and a high refill rate. Net new patients in Q2 were above our forecast and the highest quarterly total since Q1 2022.

第二季總淨收入為 1.227 億美元，比去年同期成長 23.2%，為實現我們先前提供的 2020 年總收入指導上限（4.55 億至 4.75 億美元）奠定了良好的催化劑。我現在將按產品提供第二季淨收入的明細。首先，讓我從 FIRDAPSEE 效能開始。第二季淨銷售額為 7,740 萬美元，比去年同期成長 19.2%，這是穩定的新病患入組、年度停藥率低於預測的 20% 以及較高的補充率的直接結果。第二季的淨新增病患數量高於我們的預測，也是自 2022 年第一季以來的最高季度總數。

Prescription approval rates were greater than 90% across all payers, government or private commercial insurers, ordering patterns and refill rates of 95% in Q2 or greater than historical norms of 90%. We expect to return to 90% refill rates in Q3 and Q4 and the resulting 15% or greater growth quarter versus the same quarter in 2023. We remain confident in achieving FIRDAPSE full year 2024 guidance of between $295 million and $310 million.

所有付款人、政府或私人商業保險公司的處方批准率均超過 90%，第二季的訂購模式和補充率達到 95%，或高於 90% 的歷史標準。我們預計第三季和第四季的補充率將恢復到 90%，從而與 2023 年同一季度相比增長 15% 或更高。我們仍有信心實現 FIRDAPSE 2024 年全年指引金額在 2.95 億至 3.1 億美元之間。

Our demonstrated strategic growth initiatives continue to drive organic growth. We maintain a pipeline of about 500 diagnosed LEMS patients in various stages of their diagnostic journey, not yet on FIRDAPSEE. Half of our new patient enrollments typically come from this group, a prime reason for our ability to consistently deliver 15% to 20% annual growth.

我們展示的策略性成長計劃繼續推動有機成長。我們維護著大約 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，尚未在 FIRDAPSEE 上。我們一半的新入組患者通常來自這一群體，這是我們能夠持續實現 15% 至 20% 的年增長率的主要原因。

Furthermore, LEMS education programs targeting healthcare providers and potential LEMS patients continue to yield a high level of voltage gated calcium channel antibody testing, shortening the diagnostic journey for LEMS patients and leading to more patients being eligible for treatment with FIRDAPSEE.

此外，針對醫療保健提供者和潛在 LEMS 患者的 LEMS 教育計劃繼續產生高水平的電壓門控鈣通道抗體測試，縮短了 LEMS 患者的診斷旅程，並使更多患者有資格接受 FIRDAPSEE 治療。

Lastly, as we have mentioned before, more than 90% of small cell lung cancer LEMS patients are undiagnosed. Therefore, we are working with key Thoracic Oncology Institutions to institute screening for newly diagnosed small cell lung cancer patients, resulting in a greater diagnosis of small cell lung cancer LEMS patients.

最後，正如我們之前提到的，超過 90% 的小細胞肺癌 LEMS 患者未被診斷出來。因此，我們正在與主要胸部腫瘤機構合作，對新診斷的小細胞肺癌患者進行篩檢，從而提高小細胞肺癌 LEMS 患者的診斷率。

Now let me provide an update on FYCOMPA. Q2 net revenues of $36.5 million further fortifies our revenue diversification. We expect continued stable net revenues in the second half of 2024, in-line with the full year 2024 net revenue guidance of between $130 million and $135 million.

現在讓我提供 FYCOMPA 的最新情況。第二季淨收入為 3,650 萬美元，進一步加強了我們的收入多元化。我們預計 2024 年下半年淨收入將持續穩定，與 2024 年全年 1.3 億至 1.35 億美元的淨收入指引一致。

As I mentioned last quarter, epilepsy regional account managers are enhancing FIRDAPSEE's share of voice by conducting secondary FIRDAPSEE calls with healthcare providers. This strategy leverages the 45% overlap in physician call points between FYCOMPA and FIRDAPSEE, boosting FIRDAPSEE's visibility. We have seen early indicators that this tactic is working as several patient enrollments have been a direct result of this initiative.

正如我上季度提到的，癲癇區域客戶經理正在透過與醫療保健提供者進行二次 FIRDAPSEE 通話來提高 FIRDAPSEE 的話語權。該策略利用了 FYCOMPA 和 FIRDAPSEE 之間 45% 的醫生呼叫點重疊，提高了 FIRDAPSEE 的知名度。我們已經看到早期跡象表明該策略正在發揮作用，因為該措施的直接結果是一些患者入組。

Let's shift our focus to AGAMREE. Q2 product net revenues were $8.7 million. Thanks to our dedicated efforts leading up to the March 13, 2024 product launch, we successfully exceeded our initial launch expectations. Thus far, 46% of new enrollments are transitions from EMFLAZA, while 42% are switches from prednisone, which we believe is a positive sign for the acceptance of AGAMREE.

讓我們把焦點轉移到 AGAMREE 上。第二季產品淨收入為 870 萬美元。由於我們在 2024 年 3 月 13 日產品發布之前的不懈努力，我們成功超出了最初的發布預期。到目前為止，46% 的新入組患者是從 EMFLAZA 轉入的，而 42% 是從潑尼松轉入的，我們認為這是接受 AGAMREE 的正面跡象。

In addition, approximately 80% of the top DMD centers of excellence and greater than 140 healthcare providers have submitted at least one AGAMREE enrollment form, around 89% of our current patients, our reimbursed patients and average less than 30 days to approval.

此外，約80% 的頂級DMD 卓越中心和超過140 家醫療保健提供者已提交至少一份AGAMREE 登記表，約89% 的現有患者、報銷患者均已提交，平均不到30 天即可獲得批准。

We expect the time to approval to decrease as payers finalize their policies over the next couple months. Based on the promising demand trend and encouraging payer landscape, we are revising our AGAMREE full year 2024 guidance to between $35 million and $40 million of net product revenue.

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，我們正在將 AGAMREE 2024 年全年指引修訂為 3,500 萬至 4,000 萬美元的產品淨收入。

In summary, we are confident in our ability to continue the significant strides we are making with FIRDAPSEE reflecting our commitment to sustained organic growth. We continue to achieve stable net revenues with FYCOMPA and are surpassing our revenue projections for AGAMREE. Our emphasis remains on maintaining excellence in commercial execution while strategically leveraging our capabilities to optimize our performance. I want to thank the entire team at catalyst for their unwavering commitment to patience and look forward to a successful second half of 2024.

總之，我們有信心能夠繼續與 FIRDAPSEE 取得重大進展，這反映了我們對持續有機成長的承諾。我們繼續透過 FYCOMPA 實現穩定的淨收入，並且超出了我們對 AGAMREE 的收入預測。我們的重點仍然是保持卓越的商業執行力，同時策略性地利用我們的能力來優化我們的業績。我要感謝 Catalyst 的整個團隊堅定不移的耐心，並期待 2024 年下半年取得成功。

I will now turn the call back over to Michael.

我現在將把電話轉回給邁克爾。

Thanks Jeff. Our performance during the second quarter of 2024 has set us on pace for another strong year driven by our solid financial performance and strong execution. With the successful launch of AGAMREE in mid-March, along with the continued performance of FIRDAPSEE and FYCOMPA, we have established the foundation to achieve another revenue record breaking year in 2024.

謝謝傑夫。在我們穩健的財務表現和強大的執行力的推動下，2024 年第二季的業績使我們有望迎來另一個強勁的一年。隨著 3 月中旬 AGAMREE 的成功推出，以及 FIRDAPSEE 和 FYCOMPA 的持續表現，我們為 2024 年再創收入紀錄奠定了基礎。

As you heard from Jeff, we had an outstanding quarter in product revenue performance. In the second quarter of 2024, net income before income taxes was $55.8 million, a 15% increase compared to $48.5 million for the second quarter of 2023. We reported GAAP net income of $40.8 million or $0.33 per diluted share, an increase of 8% year-over-year compared to Q2 of 2023 of $37.8 million or $0.33 per diluted share. Coupled with our disciplined approach to cost management, this increase in net income is driven by organic growth of FIRDAPSEE and FYCOMPA and the addition of AGAMREE.

正如您從傑夫那裡聽到的，我們的產品收入表現非常出色。2024 年第二季度，所得稅前淨利為 5,580 萬美元，較 2023 年第二季的 4,850 萬美元成長 15%。我們公佈的 GAAP 淨利潤為 4,080 萬美元，即稀釋後每股收益 0.33 美元，與 2023 年第二季的 3,780 萬美元，即稀釋後每股收益 0.33 美元相比，年增 8%。加上我們嚴格的成本管理方法，淨利潤的成長是由 FIRDAPSEE 和 FYCOMPA 的有機成長以及 AGAMREE 的加入所推動的。

Non-GAAP net income for the second quarter of 2024 was $69.6 million or $0.56 per diluted share, which excludes from GAAP net income amortization of inter tangible 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 million, and depreciation of $91,000.

2024 年第二季的非GAAP 淨利潤為6,960 萬美元，或稀釋後每股0.56 美元，其中不包括與我們收購FYCOMPA、AGAMREE 和RUZURGI 相關的930 萬美元的GAAP 淨利潤攤銷、基於股票的薪酬費用440 萬美元，所得稅準備金 1500 萬美元，折舊 91,000 美元。

This compares to Non-GAAP net income in the second quarter of 2023 of $60.4 million, or $0.53 per 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 $3.3 million, the income tax provision of $10.8 million and depreciation of $82,000. Our effective tax rate through the first half of 2024 was 24.5% compared to 21.5% through the first half of 2023. 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.

相較之下，2023 年第二季非GAAP 淨利潤為6,040 萬美元，即稀釋後每股收益0.53 美元，其中不包括與我們收購FYCOMPA 和RUZURGI 相關的無形資產攤銷850 萬美元的GAAP 淨利潤攤銷。股票補償費用為 330 萬美元，所得稅準備金為 1,080 萬美元，折舊為 82,000 美元。2024 年上半年我們的有效稅率為 24.5%，而 2023 年上半年為 21.5%。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。

Cost of sales expense was approximately $15.4 million in the second quarter of 2024 compared to $12 million in the second quarter of 2023 and consisted principally of royalties. As a reminder, royalties for FIRDAPSEE 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 年第二季的銷售成本費用約為 1,540 萬美元，而 2023 年第二季的銷售成本費用為 1,200 萬美元，主要包括特許權使用費。需要提醒的是，當產品淨銷售額在任何一個日曆年超過 1 億美元時，FIRDAPSE 的特許權使用費就會增加 3%，這一數字在 2024 年第二季度就超過了這一數字。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 the royalty in the range of low double digits to mid twenties as a percent of net revenue, depending on sales achievements within a calendar year. Further details on our royalty obligations for AGAMREE are disclosed in our Q2 2024 Form 10-Q and are expanded upon within the MD and a section.

此外，AGAMREE 收取的特許權使用費佔淨收入的百分比在兩位數到二十左右，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 2024 年第二季度的 10-Q 表格，並在 MD 和章節中進行了詳細闡述。

Research and development expenses were $3 million in the second quarter of 2024, slightly down from $4 million in the second quarter of 2023. SG&A expenses for the second quarter of 2024 totaled $40.7 million, compared to $28.4 million in Q2 2023. The overall difference in SG&A expenses in the second quarter of 2024 was principally due to incremental operating expenses in support of the launch of AGAMREE.

2024 年第二季的研發費用為 300 萬美元，略低於 2023 年第二季的 400 萬美元。2024 年第二季的銷售管理及行政費用總計 4,070 萬美元，而 2023 年第二季為 2,840 萬美元。2024 年第二季 SG&A 費用的整體差異主要是因為支持 AGAMREE 推出而增加的營運費用。

As reported, we ended the second quarter of 2024 with cash and cash equivalents of $375.7 million compared to $137.6 million at December 31, 2023. The increase in cash of $238.1 million was driven in part by the January 9, 2024 capital raise that resulted in $140.7 million in net cash proceeds. The remaining increase in cash is driven primarily by $96.1 million generated from operations of the business.

據報道，截至 2024 年第二季度，我們的現金和現金等價物為 3.757 億美元，而截至 2023 年 12 月 31 日為 1.376 億美元。現金增加 2.381 億美元，部分原因是 2024 年 1 月 9 日的融資帶來了 1.407 億美元的淨現金收益。剩餘的現金成長主要來自業務營運產生的 9,610 萬美元。

I would like to note that we generated approximately $53.2 million more in cash from operations for the first half of 2024 than we did for the first half of 2023. This underscores our strategic and effective capital utilization, driving incremental cash flows for the company's benefit. We believe our current funds continue to provide the financial flexibility to support our strategic initiatives, including business development and portfolio expansion, meet our potential contractual obligations and sustain our commercial and R&D programs.

我想指出的是，我們 2024 年上半年的營運現金比 2023 年上半年多了約 5,320 萬美元。這凸顯了我們策略性和有效的資本利用，為公司的利益推動增量現金流。我們相信，我們現有的資金將繼續提供財務靈活性，以支持我們的策略舉措，包括業務發展和投資組合擴張、履行我們潛在的合約義務並維持我們的商業和研發計劃。

More detailed information Analysis of our second quarter 2024 financial performance may be found in our quarterly report on Form 10-Q, which was filed with the securities and Exchange Commission yesterday, August 7, and can be found on the Investor relations page on our website at www.catalystpharmacy dot.

更多詳細資訊 有關我們 2024 年第二季的財務表現分析，請參閱我們於 8 月 7 日向美國證券交易委員會提交的 10-Q 表格季度報告，也可在我們網站的投資者關係頁面上找到請造訪www.catalystpharmacy 點。

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. The momentum in our portfolio is exciting. FIRDAPSE continues to grow as predicted due to the incredible focus of our field teams. AGAMREE is off to a great launch due to the strong partnership between our medical, commercial and patient advocacy teams and FYCOMPA continues to deliver in the highly competitive market. Because of the relentless focus of our epilepsy team, we had a robust start to this year and we look forward to a strong second half in 2024.

謝謝邁克。我們投資組合的勢頭令人興奮。由於我們現場團隊的高度關注，FIRDAPSE 繼續按預期成長。AGAMREE 的推出得益於我們的醫療、商業和患者倡導團隊之間的牢固合作關係，並且 FYCOMPA 繼續在競爭激烈的市場中提供服務。由於我們癲癇團隊的不懈關注，我們今年取得了良好的開局，我們期待 2024 年下半年的強勁表現。

We are confident in our ability to capitalize on the opportunities ahead. Our unwavering commitment to advancing patient care, unlocking growth potential and delivering value to our stakeholders remains at the forefront of our efforts, supported by our exemplary track record and our dedicated team, we are ideally positioned to drive further growth and achieve sustained success.

我們對利用未來機會的能力充滿信心。我們對推進患者護理、釋放成長潛力和為利益相關者提供價值的堅定承諾仍然是我們努力的最前沿，在我們模範的業績記錄和敬業團隊的支持下，我們處於推動進一步增長和取得持續成功的理想位置。

At Catalyst, our mission is resolute to pioneer innovative medicines for patients with rare orphan diseases. We look forward to providing updates on our progress and we thank our catalyst team for their commitment and all of you for your continued support.

在 Catalyst，我們的使命是堅決為罕見孤兒疾病患者開拓創新藥物。我們期待提供最新進展，感謝我們的催化劑團隊的承諾以及大家的持續支持。

We now turn the call back over to our operator. We look forward to answering your questions. Thank you.

現在，我們將電話轉回給我們的接線生。我們期待回答您的問題。謝謝。

Thank you. We will now be conducting a question-and-answer session. (Operator instructions).

謝謝。我們現在將進行問答環節。（操作員說明）。

Charles Duncan, Cantor Fitzgerald.

查爾斯鄧肯，坎托菲茨杰拉德。

Hey, good morning, Rich, Jeff and team. Congratulations on a really nice two q and thanks for taking our questions. I had a couple, first of all, on AGAMREE. Do you believe that there was any bolus in the first quarter of sales because patients or caregivers were waiting for the drug to be, to be approved and launched? And then secondarily, I know you don't have any experience with this yet in this market, but what would you anticipate for summer in terms of, patient and prescriber attendance, if you will?

嘿，早上好，里奇、傑夫和團隊。恭喜您獲得了非常好的兩個問題，並感謝您提出我們的問題。首先，我在 AGAMREE 上有一些。您是否認為第一季的銷售有任何推注，因為患者或照護者正在等待藥物獲得批准和上市？其次，我知道您在這個市場上還沒有任何這方面的經驗，但是如果您願意的話，您對夏季的患者和處方醫生出勤率有何預期？

Charles, thanks for the question. This is something we thought a lot about, especially as we queued up the patients beginning in December of last year. I'm going to turn it to Jeff. Jeff will give you the answer. Thanks.

查爾斯，謝謝你的提問。這是我們思考了很多的事情，特別是當我們從去年 12 月開始對病人進行排隊時。我要把它轉給傑夫。傑夫會給你答案。謝謝。

Hi, Charles. And thanks for your question. There was significant pent up demand leading up to the commercial launch that we had in March. So, yes, to answer your first question, we did see significant interest prior to that, which helped the late launch, the late part of Q1, but also it helped Q2.

嗨，查爾斯。謝謝你的提問。在我們三月的商業發布之前，存在著大量被壓抑的需求。所以，是的，回答你的第一個問題，在此之前我們確實看到了很大的興趣，這有助於第一季後期的後期發布，但也有助於第二季。

So again, strong pent up demand there. We are seeing, again, significant demand and interest moving forward. What we see is a lot of the sites, I mentioned it on the call, 80% of the high volume centers of excellence for DMD have enrolled at least one patient, and we've seen greater than 140 healthcare providers prescribe or submit an enrollment for AGAMREE

再次強調，那裡的需求被壓抑得很強烈。我們再次看到巨大的需求和興趣向前發展。我們看到的是許多網站，我在電話中提到過，80% 的 DMD 大容量卓越中心已經註冊了至少一名患者，並且我們已經看到超過 140 家醫療保健提供者開出處方或提交註冊為阿伽姆瑞

Now, what we expect moving forward are the increase in the depth of interest from those physicians in the centers as we move into Q3 and Q4. So we are excited about the demand so far, and we expect that to continue.

現在，我們預計，隨著進入第三季度和第四季度，中心醫生的興趣深度會增加。因此，到目前為止，我們對需求感到興奮，並預計這種情況將持續下去。

So, Jeff, as far as Charles, question regarding the summer. Do you see any cyclicity so far in the summer, any downward trend or upward?

所以，傑夫，就查爾斯而言，對夏天有疑問。到目前為止，您認為夏季有任何週期性嗎？

We see continued strong interest. Charles. So we expect the launch trajectory to stay the same.

我們看到持續的強烈興趣。查爾斯.因此，我們預計發射軌跡將保持不變。

And then, Charles, to the first part of your question, this bolus we saw, so we, I think we referred to this in the first quarter call, was as a step-up. So we had a, very light first quarter because we only had two weeks. We expected to have this nice step up, and then what we is a continued traditional curve, and that's what I think we're experiencing. Thanks for the question.

然後，查爾斯，對於你問題的第一部分，我們看到了這一推注，所以我們，我想我們在第一季電話會議中提到了這一點，這是一個升級。因此，我們第一季的業績非常清淡，因為我們只有兩週的時間。我們期望能有一個很好的進步，然後我們會繼續傳統的曲線，我認為這就是我們正在經歷的。謝謝你的提問。

That's helpful. Quick question on FIRDAPSE. Congratulations on the approval of the new higher dose. I had a question about that in terms of the future adoption and persistency, if you will, for patients, will the new dose impact discontinuations either fewer discontinuations due to increased efficacy or satisfaction? Or will it perhaps result in more discontinuations due to less tolerability? What's your sense of how that adoption of that higher dose will go for continued use?

這很有幫助。關於 FIRDAPSE 的快速問題。恭喜新的更高劑量獲得批准。我有一個關於未來採用和持久性的問題，如果您願意的話，對於患者來說，新劑量是否會影響停藥，或者由於療效或滿意度提高而減少停藥？或是會因耐受性較差而導致更多的停藥？您對採用更高劑量後如何繼續使用有何看法？

Good question, Charles. And what I can tell you is, again, our annual discontinuation rate is under 20%. And within that, we see the majority of the discontinuations happen within six months of prescribing or initiating therapy on FIRDAPSEE. So at this time, we don't expect the discontinuation to change or increase because of that more flexibility in the dose going up to 100 milligrams.

好問題，查爾斯。我可以再次告訴您的是，我們的年度停產率低於 20%。其中，我們看到大多數停藥發生在開立或開始 FIRDAPSEE 治療後的六個月內。因此，目前我們預計停藥情況不會改變或增加，因為劑量高達 100 毫克的靈活性更大。

Okay, last quick question for Rich. Bigger picture strategy. Thinking about some of what you have to bring to the party in terms of, in licensing, do you have a goal for yet this year? And are you focused more on neuromuscular or perhaps rare epilepsy or other areas of, call it neuro innovation? Thanks.

好的，最後一個關於 Rich 的快速問題。大局戰略。考慮一下您必須在許可方面為聚會帶來的一些東西，您今年有目標嗎？您是否更關注神經肌肉或罕見癲癇或其他領域（稱為神經創新）？謝謝。

Great question, Charles. So our business development strategy remains the same. We're looking at orphan and differentiated orphan. We're looking at things that are accretive or nearly accretive, and we want to bring in income diversity. So we like the opportunities in neuro because we feel like we have a great footprint there.

好問題，查爾斯。所以我們的業務發展策略保持不變。我們正在研究孤兒和差異化孤兒。我們正在關注增值或接近增值的事情，我們希望帶來收入多樣性。所以我們喜歡神經領域的機會，因為我們覺得我們在那裡有很大的足跡。

But we're not stopping. We're not just isolating ourselves to neuro because we believe that the infrastructure that we have behind the outward facing organization, the sales organization, the patient advocacy organization, really supports just about any orphan condition that we would have. So we really feel like we can bring that, that strength to anything.

但我們不會停止。我們不僅僅是將自己與神經隔離，因為我們相信我們在外向組織、銷售組織、患者倡導組織背後擁有的基礎設施確實支持我們可能擁有的任何孤兒狀況。所以我們真的覺得我們可以把這種力量帶到任何事情上。

So we like the neurospace, but we really want to move within the neuro space if the opportunity presents itself when it does present itself or outside. So we're looking very broadly, we want to take advantage of those opportunities that come our way.

所以我們喜歡神經空間，但如果機會確實出現或出現在神經空間之外，我們真的希望在神經空間內移動。因此，我們的目光非常廣泛，我們希望利用我們遇到的機會。

Thanks, Rich. Nice quarter.

謝謝，里奇。不錯的季度。

Thank you very much, Charles. We really appreciate your support.

非常感謝你，查爾斯。我們非常感謝您的支持。

Samantha Semenkow, Citibank

薩曼莎‧塞曼科 (Samantha Semenkow)，花旗銀行

Hi, good morning, and thanks for taking the question. And let me my congratulations on a really nice and strong quarter. So while still really early in the launch for AGAMREE, I'm wondering how you think about the patient mix will evolve over the next several quarters.

你好，早上好，感謝您提出問題。讓我祝賀這個季度非常美好和強勁。因此，雖然 AGAMREE 還處於推出初期，但我想知道您如何看待未來幾季患者組合的發展。

It seems the percentages that are coming from menthalaza and prednisone thus far really holding steady from what you shared on the first quarter. And I'm wondering how generic EMFLAVA is fitting into the overall steroid landscape as we'll? And then I have a follow up.

到目前為止，來自薄荷拉扎和潑尼鬆的百分比似乎與您在第一季分享的數據相比確實保持穩定。我想知道通用 EMFLAVA 如何像我們一樣融入整個類固醇領域？然後我有一個後續行動。

Thanks, Samantha. Those are great questions. I'll turn it right over to Jeff for that.

謝謝，薩曼莎。這些都是很好的問題。我會把它直接交給傑夫。

Hi, Samantha. As far as the mix moving forward, we expect it to remain the same. Like you said, it's pretty flat and similar to what we saw early on in the launch, and it's carried forward. We're very excited about that because it shows the strong interest in the adoption of AGAMREE across all patients that were on steroids, not just a specific segment. So excited about that. As far as the generic entrance for inflaza coming over, and we have not seen an impact so far on adoption of AGAMREE for those patients. So no impact early on.

嗨，薩曼莎。就未來的組合而言，我們預計它將保持不變。就像你說的，它非常平坦，與我們在發布初期看到的類似，並且它被延續了下來。我們對此感到非常興奮，因為它表明了所有使用類固醇的患者（而不僅僅是特定群體）對採用 AGAMREE 的強烈興趣。對此感到非常興奮。就 inflaza 的仿製藥進口而言，到目前為止，我們尚未看到對這些患者採用 AGAMREE 的影響。所以早期沒有影響。

Got it. Great. And then just on FIRDAPSEE, I wonder if you could share just any updates on the process or the timing of the ongoing FIRDAPSEE. Paragraph four, litigation, and thank you.

知道了。偉大的。然後就 FIRDAPSEE 而言，我想知道您是否可以分享有關正在進行的 FIRDAPSEE 流程或時間表的任何更新。第四段，訴訟，謝謝。

Steve, do you want to take that?

史蒂夫，你想接受這個嗎？

Sure. Well, Samantha, as it's generally bad practice to comment on ongoing litigation. I can tell you that we are working diligently to bring the cases to a favorable a manner as possible for catalyst.

當然。好吧，薩曼莎，因為對正在進行的訴訟發表評論通常是不好的做法。我可以告訴你，我們正在努力使案件盡可能以有利於催化劑的方式進行。

Understood. Thanks so much for taking the questions.

明白了。非常感謝您提出問題。

Thank you.

謝謝。

Jason Gerberry, Bank of America

傑森‧格伯里，美國銀行

Hey, good morning. Thanks for taking my questions. Maybe just first on Biz Dev, are you generally inclined to do more deals funded with cash and backend success based milestones or talk about maybe appetite to take on some leverage to go after some larger targets that could be more transformational?

嘿，早安。感謝您回答我的問題。也許首先在業務開發方面，您是否通常傾向於進行更多由現金和基於後端成功的里程碑資助的交易，或者談論可能有興趣利用一些槓桿來追求一些可能更具變革性的更大目標？

Great question, Jason. So we would be looking for opportunities to add to the income statement right. Right now and look for that opportunity. I think we could handle a deal or two with the cash that we have, and then I think we would probably look to dip into the leverage opportunity after that. I think we would be looking to look for more of a transformational opportunity. We like the building of the income diversity as a stepwise function for the company.

好問題，傑森。因此，我們將尋找機會加入損益表中。現在就尋找這個機會。我認為我們可以用我們擁有的現金處理一兩筆交易，然後我認為我們可能會尋求在此之後利用槓桿機會。我認為我們會尋求更多的轉型機會。我們喜歡將收入多元化的建設作為公司的逐步職能。

We think that's the best path forward for us. We like our product mix at this point in time, and we want to build up that strength of the income statement and continue to build the balance sheet. And at that point, I think looking at more of a transformational opportunity.

我們認為這對我們來說是最好的前進道路。我們喜歡目前的產品組合，我們希望增強損益表的實力並繼續建立資產負債表。到那時，我認為更多的是尋找轉型機會。

Mike, any comments?

麥克，有什麼意見嗎？

I think you covered it. We have a strong balance sheet. We're ready. Yep. So it sounds like maybe more of a sequencing of deals or something that involved leverage would be after other deals that maybe improve your profitability in your cash flow base, then you'd consider larger transformational deals. Is that the right way to think about it? I think so, yes.

我認為你涵蓋了它。我們擁有強大的資產負債表。我們準備好了。是的。因此，聽起來可能更多的是一系列交易或涉及槓桿的事情發生在其他可能提高現金流基礎盈利能力的交易之後，然後你會考慮更大的轉型交易。這是正確的思考方式嗎？我想是的，是的。

When you think about what we're trying to do is build up the strength of the company. We feel like we're really in a great position right now. We feel like we're strong. We know we're strong. And then as we think about the long term of the company, we want to work our way back in the pipeline. So as we build that future orientation of the company, we want to have that strong cash flow, strong balance sheet to be able to take on that transformational opportunity.

當你想到我們正在努力做的是增強公司的實力。我們覺得我們現在確實處於一個很好的位置。我們感覺自己很堅​​強。我們知道我們很堅強。然後，當我們考慮公司的長期發展時，我們希望重新開始工作。因此，當我們建立公司的未來定位時，我們希望擁有強勁的現金流和強大的資產負債表，以便能夠抓住這一轉型機會。

Okay. And then if I can ask a question about FIRDAPSE, just the growth that you're seeing, it looks like maybe it's two thirds volume, maybe a third price mix. I assume that that volume is mostly patients that don't have small cell lung as a component of their disease. So is that kind of what you're seeing in terms of the mix and what's driving this kind of 15% to 20% growth?

好的。然後，如果我可以問一個有關 FIRDAPSE 的問題，就您所看到的成長而言，看起來可能是三分之二的銷量，也許是三分之一的價格組合。我認為這個數量主要是沒有小細胞肺作為其疾病組成部分的患者。那麼，這就是您所看到的混合情況嗎？

It's pretty accurate. There. We are seeing from the non small cell lung cancer LEM patients, we've seen steady about low 20% as far as our mix goes, of our total LEM patients that have small cell.So we expect that it's our opportunity moving forward to help that share. As I mentioned on the call, about 90% of those patients, small cell lung cancer LEMS patients are undiagnosed. There are undiagnosed at this time. So we're applying a lot of resources towards helping those patients get diagnosed sooner. So that's where our growth will be coming from. But as far as today, you're accurate about how you described dividing up that growth.

這是相當準確的。那裡。我們從非小細胞肺癌 LEM 患者中看到，就我們的組合而言，我們看到穩定的約 20% 左右的 LEM 患者患有小細胞。那個分享。正如我在電話中提到的，大約 90% 的小細胞肺癌 LEMS 患者未被診斷出來。目前尚有未確診者。因此，我們正在運用大量資源來幫助這些患者更快得到診斷。這就是我們的成長的來源。但就今天而言，您所描述的如何劃分成長是準確的。

Got it, thanks so much, guys.

明白了，非常感謝，夥計們。

Thank you.

謝謝。

Joon Lee, Truist Securities.

李俊，Truist 證券公司。

Good morning. This is Osma on for Joon. Congrats on the quarter, and thanks for taking the questions. It was mentioned in the prepared remarks that refill rates were higher than historical norms referred at. Can you explain what led to that and why you expect to return back from 90% in the following quarters? And then also on FIRDAPSEE, how soon after approval of the 100 milligrams dose did you start seeing patients which had a higher dose, and how much of an impact do you think they had in the quarter? Thank you.

早安.這是奧斯瑪為瓊代言。恭喜本季度，感謝您提出問題。在準備好的評論中提到，補充率高於提到的歷史標準。您能否解釋一下導致這種情況的原因以及為什麼您預計在接下來的幾個季度內從 90% 的水平恢復過來？然後還在 FIRDAPSEE 上，在批准 100 毫克劑量後多久，您開始看到服用更高劑量的患者？謝謝。

Thanks, June, and thanks for your support as well. Jeff, you want to take that?

謝謝六月，也謝謝您的支持。傑夫，你想要那個嗎？

Sure. As I had mentioned on the Q1 earnings call, we saw some impact from the change healthcare that impacted the refill rate in February. Overall for the quarter in Q1, we were at about 86%. And so traditionally, our historical norms are 90%. In April and May, we saw a recovery there, and we saw higher than the 90% historical norm refill rate, and it actually went up to 95% because of that.

當然。正如我在第一季財報電話會議上提到的，我們看到醫療保健變化對 2 月的補充率產生了一些影響。總體而言，第一季的完成率約為 86%。所以傳統上，我們的歷史標準是 90%。4 月和 5 月，我們看到了那裡的復甦，我們看到高於 90% 的歷史正常補充率，因此實際上上升到了 95%。

So patients that were impacted by that in Q1 filled their rates, filled their prescriptions more in Q2, we expect that to normalize and return to the 90% for Q3 and Q4 and still delivering the 15% or greater growth for FIRDAPSEE. As far as the 100 milligram expansion, it's a great thing for patients and healthcare providers. It really does give them the flexibility to manage and treat I their LEM symptoms.

因此，受第一季影響的患者配藥率在第二季度增加了配藥率，我們預計第三季和第四季將恢復正常並恢復到90%，並且FIRDAPSEE 仍將實現15% 或更高的成長。就 100 毫克的擴展而言，這對於患者和醫療保健提供者來說是一件好事。它確實讓他們能夠靈活地管理和治療他們的 LEM 症狀。

And also just a quick step back, 40% of LEMS patients prior to the label expansion were on 80 milligrams. And those patients, the average daily dose went up by about a milligram, 1.1 milligram. Since that label expansion, we did not expect or do not expect that bolus to just happen immediately. We expect that increase of the average daily dose to reach its full impact in the subsequent quarters. Thank you.

退一步來說，在標籤擴展之前，40% 的 LEMS 患者服用 80 毫克。這些患者的平均每日劑量增加了約 1 毫克，即 1.1 毫克。自從標籤擴展以來，我們沒有期望或不期望推注會立即發生。我們預計日均劑量的增加將在接下來的幾季中充分發揮作用。謝謝。

So I just want to clarify a statement Jeff made. 50% growth is 15% growth over Q2 2020. Just to clarify.

所以我只想澄清傑夫的聲明。 50% 的成長相當於 2020 年第二季成長 15%。只是為了澄清。

So we've turned to that growth as we do a comparison. It's. We look at Q3 2024. The 15% growth we'll see is versus Q3. Q2 '20 or, sorry, Q3 versus Q3 2023. Thanks.

因此，我們在進行比較時轉向了這種增長。它是。我們展望 2024 年第三季。與第三季相比，我們將看到 15% 的成長。20 年第 2 季度，或抱歉，第 3 季與 2023 年第 3 季。謝謝。

Thank you. Leland Gershell, Oppenheimer & Co.

謝謝。利蘭·格謝爾，奧本海默公司

Hi. Good morning, Mike. Congrats on the strong quarter as well. And thanks for taking the question. Just a question again, on AGAMREE these patients with Duchenne often are not seeing their specialists that frequently.

你好。早上好，麥克。也恭喜季度的強勁表現。感謝您提出問題。再次問一個問題，在 AGAMREE 上，這些杜興氏症患者通常不會經常去看專家。

If they're medically stable, maybe once every three months, sometimes once every six months. I'm just wondering if you've been seeing patients coming in to switch to a AGAMREE because it's now available, or are they sort of waiting for their next scheduled deployment to change steroids? Just sort of an observational question, if you have any perspective there. Thank you.

如果他們的病情穩定，可能每三個月一次，有時每六個月一次。我只是想知道您是否看到患者因 AGAMREE 現已上市而前來轉用，或者他們是否在等待下一次計劃部署以更換類固醇？這只是一個觀察性問題，如果你有任何觀點的話。謝謝。

Hi, Leland. It's really center of excellence specific. Some sites had that pent up demand, like I mentioned earlier, where they were ready to go as soon as it became commercially available. And then over time, they will wait for other patients to come in and have that discussion. And in other cases, there's outreach going on to the boys living with DMD and their caregivers about switKyeng. So it really is specific. It's dependent on the staff and the physicians, how they want to go, the process that they put in place.

嗨，利蘭。這確實是卓越中心的具體內容。正如我之前提到的，有些網站有被壓抑的需求，一旦商業化，它們就準備好投入使用。然後隨著時間的推移，他們會等待其他患者進來並進行討論。在其他情況下，我們會向患有 DMD 的男孩及其照顧者進行有關 switKyeng 的宣傳活動。所以它確實是具體的。這取決於工作人員和醫生、他們想要如何進行以及他們實施的流程。

Okay. Thanks very much..

好的。非常感謝..

Thank you. Joseph Catanzaro, Piper Sandler.

謝謝。約瑟夫·卡坦扎羅，派珀·桑德勒。

Great. Hey, everybody, thanks for taking my questions. Maybe, maybe. First one, honing in on for depth a bit more curious. Jeff, what do you attribute the sort of highest level of new patient starts in the quarter, too. I know you cited a couple things. Maybe you could just speak to that, which of those you expect to sort of continue, and maybe which factors maybe fade, if at all? That'd be super helpful. And then I have one quick follow up. Thanks.

偉大的。嘿，大家好，謝謝你回答我的問題。也許，也許。第一個，磨練深度，更好奇。傑夫，您如何看待本季新患者開始的最高水準？我知道你引用了幾件事。也許你可以談談這一點，你希望哪些因素能夠繼續下去，也許哪些因素可能會消失（如果有的話）？那會非常有幫助。然後我會進行快速跟進。謝謝。

Sure. Hey, Joe, as we constantly mention, and I think it's very important in ultra orphan disease, is to maintain that pipeline that we always do. And that's over 500 patients that are in various stages of their LEMS journey. And so we have ongoing conversations between the field and the healthcare providers with those patients.

當然。嘿，喬，正如我們經常提到的，我認為在超級孤兒疾病中非常重要的是維持我們一直在做的管道。超過 500 名患者處於 LEMS 旅程的不同階段。因此，我們與這些患者的現場和醫療保健提供者之間正在進行持續的對話。

That's where we have the most success. And about 40% to 50% of our new enrollments come from those leads. So as a team, what we constantly focus on are increasing the voltage. Gated antibody testing, the VGCC antibody testing, and then when those patients are tested and if they are positive, we usually can identify where those patients are through our lead sources, and then we try to help those patients by giving those leads to our rants. That's how we ensure that we always have a robust pipeline for future enrollments. And we will continue to do that. And we're always on the search to find additional sources of new leads.

這就是我們最成功的地方。我們大約 40% 到 50% 的新註冊人數來自這些潛在客戶。所以作為一個團隊，我們不斷關注的就是提高電壓。門控抗體測試、VGCC 抗體測試，然後當這些患者接受測試並且結果呈陽性時，我們通常可以透過我們的線索來源確定這些患者的位置，然後我們嘗試透過將這些線索提供給我們的咆哮來幫助這些患者。這就是我們如何確保我們始終擁有強大的未來招生管道。我們將繼續這樣做。我們一直在尋找新線索的其他來源。

Great. That's helpful. And then my quick follow up, I guess, on portfolio expansion. Rich, I read into maybe some of your comments as there being sort of inbound inquiries to you guys, wondering if you could characterize the current projects that you're diligent. how many of those, what percent originated as inbound inquiries versus outbound inquiries? Thanks.

偉大的。這很有幫助。我想，然後我會快速跟進投資組合擴張。Rich，我可能讀到了你們的一些評論，因為你們收到了一些入站詢問，想知道你們是否能描述一下你們當前的勤奮項目。其中有多少，來自入站查詢和出站查詢的百分比是多少？謝謝。

That's a great question, Joe, really. I think what we've seen now is an uptick in inbound inquiries because of the performance that we see. We're seeing, I guess the best way I could characterize it is that we are, we're looking at the performance of the company and now the execution.

這是一個很好的問題，喬，真的。我認為，由於我們看到的表現，我們現在看到的是入站查詢的增加。我們看到，我想我能描述的最好方式是，我們正在關注公司的業績以及現在的執行情況。

We're getting these inquiries because we're a place that people want to place their products. So I would say we're probably at a 30, 30%, 35% of the inquiries, I'm sorry, of the products that we're talking about are inbound, where people are calling us and saying, hey, we'd like to talk to you about an opportunity, and I think that's really exciting. you can go from one product to three products in the space of about 18 months, and you're really hustling for products.

我們收到這些詢問是因為我們是人們想要放置產品的地方。所以我想說，抱歉，我們談論的產品中可能有 30%、30%、35% 的詢問是入境產品，人們打電話給我們說，嘿，我們'我想和你談談一個機會，我認為這真的很令人興奮。你可以在大約 18 個月的時間內從一種產品變成三種產品，而且你真的很忙於尋找產品。

And we continue to hustle for products. I want to emphasize that we are working diligently to bring on new products that fit who we are. And our I differentiated, which is critical for us to have companies approach us with products that are in our space and fit. We're excited about that. So I'd say about 30, 35%.

我們繼續努力開發產品。我想強調的是，我們正在努力推出適合我們的新產品。我們的 I 與眾不同，這對我們讓公司向我們提供適合我們空間和適合的產品至關重要。我們對此感到興奮。所以我想說大約 30%、35%。

Okay, great. That's helpful. And interesting, thanks for taking my questions.

好的，太好了。這很有幫助。有趣的是，感謝您提出我的問題。

Thank you, and thanks for your support as well, thank you.

謝謝你們，也謝謝你們的支持，謝謝。

Thank you. There are no further questions at this time. This does conclude today's conference. You may disconnect your lines at this time. Thank you for your participation.

謝謝。目前沒有其他問題。今天的會議到此結束。此時您可以斷開線路。感謝您的參與。