Catalyst Pharmaceuticals Inc (CPRX) 2023 Q4 法說會逐字稿

Hello, and welcome to the Catalyst Pharmaceuticals, Inc. fourth quarter and full year 2023 financial results conference call and webcast. If anyone should require operator assistance, please press star zero on your telephone keypad. Our question and answer session will follow the formal presentation. Here may be placed in the question queue at any time by pressing star one on your telephone keypad. We ask you please ask one question and one follow-up then return to the queue. As a reminder, this conference is being recorded. It is now my pleasure to turn the call over to CFO, Mike Kalb, please go ahead.

您好，歡迎參加 Catalyst Pharmaceuticals, Inc. 2023 年第四季和全年財務業績電話會議和網路廣播。如果有人需要接線生幫助，請按電話鍵盤上的星號零。我們的問答環節將在正式演講之後進行。透過按下電話鍵盤上的星號一，可以隨時將此處放入問題佇列中。我們要求您提出一個問題和一項後續行動，然後返回隊列。提醒一下，本次會議正在錄製中。現在我很高興將電話轉給財務長邁克·卡爾布（Mike Kalb），請繼續。

Thank you.

謝謝。

Good morning, everyone, and thank you for joining our conference call to discuss Catalyst's Fourth Quarter and Full Year 2023 financial results and business highlights leaving the call today is Richard Daly, CEO. We are also joined by Dr. Steven Miller, our Chief Operating Officer and Chief Scientific Officer, and Jeffrey Del Carmen, our Chief Commercial Officer, further for the Q&A session. Dr. Gary Ingenito, or our Chief Medical and Regulatory Officer, will be available for questions.

大家早安，感謝大家參加我們的電話會議，討論 Catalyst 的第四季度和 2023 年全年財務業績和業務亮點，今天離開電話會議的是首席執行官理查德·戴利 (Richard Daly)。我們的營運長兼首席科學長 Steven Miller 博士和商務長 Jeffrey Del Carmen 也參加了問答環節。我們的首席醫療和監管官加里·英傑尼托 (Gary Ingenito) 博士將回答問題。

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. If 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 prove not to be accurate and actual results may vary from the expectations contained in our forward-looking statements. 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.

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。如果這些陳述與我們目前的預期、估計和預測有關，而不是對未來績效的保證。它們涉及難以預測的風險、不確定性和假設，並且可能被證明不準確，實際結果可能與我們前瞻性陳述中包含的預期有所不同。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起來考慮，包括我們 2023 年年度報告 10 K 中所述的風險因素。

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 welcome, everyone, to the call first, I'd like to say that our entire team is proud of the work that we do on behalf of the orphan and rare disease communities that we serve. And we're especially excited to be speaking you to today on Rare Disease Day 2023 was an outstanding year for Catalytica. We are proud of all that the team has achieved. We are pleased to highlight the progress we have made during this transformational year that we just completed, and we are proud to provide insight into the year ahead. But before the team size into this, I'd like to offer some high-level comments. We operate our business around four key pillars. First, commercial execution with Supreme focus on patient care and access. Second, portfolio expansion to business development, third lifecycle management and partnerships, and finally, financial discipline. Our discussion today will provide you with updates on each of these areas.

謝謝麥克，首先歡迎大家撥打電話，我想說，我們整個團隊為我們代表我們所服務的孤兒和罕見疾病社區所做的工作感到自豪。我們特別高興今天能在罕見疾病日與您交談，2023 年對於 Catalytica 來說是傑出的一年。我們為團隊所取得的一切成就感到自豪。我們很高興強調我們在剛完成的這一轉型年中所取得的進展，並且很自豪能夠提供對未來一年的見解。但在確定團隊規模之前，我想提供一些高層評論。我們圍繞著四個關鍵支柱開展業務。首先，Supreme 的商業執行重點是患者護理和訪問。其次，投資組合擴展到業務開發，第三是生命週期管理和合作關係，最後是財務紀律。我們今天的討論將為您提供每個領域的最新資訊。

2023 was a transformational year for catalysts, but start with our first pillar, our commercial execution, the core Firdapse business remains strong, evidenced by our sustained organic growth and continue to reach more patients in this market by working diligently to increase awareness of Firdapse and the disease, it treats lamps to further enhance our business.

2023 年對催化劑來說是轉型的一年，但從我們的第一個支柱開始，我們的商業執行，核心Firdapse 業務仍然強勁，我們持續的有機增長證明了這一點，並通過努力提高Firdapse 的知名度，繼續接觸到該市場的更多患者。疾病，它治療燈具以進一步增強我們的業務。

In 2023, we expanded our commercial portfolio with our acquisition of Compo seamless integration of aficamten franchise into our company has yielded promising results. We're pleased with the progress the product's performance, this addition in which it enriches our portfolio by enhancing income diversity fostering stable revenue streams and fortifying our balance sheet in preparation for future acquisitions.

2023 年，我們透過收購 Compo 擴大了我們的商業投資組合，將 aficamten 特許經營權無縫整合到我們公司，取得了可喜的成果。我們對產品性能的進步感到高興，這項補充透過增強收入多樣性、培育穩定的收入流並強化我們的資產負債表，為未來的收購做好準備，豐富了我們的產品組合。

Our second pillar portfolio expansion, in addition to five copper in the third quarter of 2023 we licensed the North American rights to again raise our US NDA filed asset for Duchenne's muscular dystrophy Academy was approved in the fourth quarter, and we remain on track for the launch of The Gallery before the end of the first quarter 2024. We're excited to have this innovative product in our portfolio. More importantly, we know through our outreach to patients, caregivers and providers that the Duchenne community is excited as well. We are well prepared prepared for the successful launch of a gallery, providing further revenue growth and diversification while addressing a significant unmet need for improved therapeutic options for patients. Future potential for Gambro represents an opportunity for incremental growth that can continue to propel the company forward. We are committed to our buy and build strategy and our results bear testament to its success. In 2023 alone, we allocated approximately $300 million toward acquiring assets without debt, bolstering the company's commercial position. Furthermore, our recent common stock offering, combined with our robust balance sheet, underscores our ability to persistently pursue strategic investments aimed at expanding our TAM and diversifying our portfolio Jeff will provide an overview of our performance in 2023 with respect to commercializing Firdapse and Fycompa and our plans for our portfolio in 2024, including Gary and his section this morning for pillar number three, lifecycle management and partnerships. We await the FDA's decision on the expansion of the Firdapse label to include 100 milligram dosing which is expected in the second quarter of 2024. We believe this has the potential to provide an important option for patients who live with limbs. We are also making great strides in our ex-US partnerships. Japanese regulatory authorities have accepted diagnose NDA for Firdapse, and we expect a decision on their application later this year. This also bodes well for our expanding it for expanding our ex U.S. opportunities as the Japanese NDA acceptance triggers the expansion of our territorial rights for Firdapse in certain A-Pac and Latin markets.

我們的第二個支柱投資組合擴張，除了2023 年第三季的五銅之外，我們還獲得了北美權利，以再次為杜氏肌肉營養不良症學院籌集美國NDA 備案的資產，該資產已於第第四季獲得批准，我們仍將朝著The Gallery 將於 2024 年第一季末之前推出。我們很高興在我們的產品組合中加入這種創新產品。更重要的是，透過與患者、照護者和提供者的聯繫，我們知道杜興社區也很興奮。我們已做好充分準備，為畫廊的成功推出做好準備，進一步實現收入成長和多元化，同時解決改善患者治療選擇的重大未滿足需求。Gambro 的未來潛力代表著增量成長的機會，可以繼續推動公司前進。我們致力於我們的購買和建立策略，我們的結果證明了它的成功。光是 2023 年，我們就撥款約 3 億美元用於無債務收購資產，增強了公司的商業地位。此外，我們最近的普通股發行，加上我們穩健的資產負債表，突顯了我們持續追求戰略投資的能力，旨在擴大我們的TAM 並使我們的投資組合多樣化。Jeff 將概述我們2023 年在Firdapse 和Fycompa 商業化方面的表現，以及我們的 2024 年投資組合計劃，包括今天早上加里和他的第三個支柱部門：生命週期管理和合作夥伴關係。我們正在等待 FDA 關於擴大 Firdapse 標籤範圍以包括 100 毫克劑量的決定，預計將於 2024 年第二季度做出。我們相信這有可能為殘肢患者提供一個重要的選擇。我們在與美國以外的夥伴關係方面也取得了長足進步。日本監管機構已接受 Firdapse 的診斷 NDA，我們預計將在今年稍後對其申請做出決定。這對我們擴大其在美國以外的機會也是個好兆頭，因為日本 NDA 的接受觸發了我們對 Firdapse 在某些亞太地區和拉丁市場的領土權利的擴大。

Finally, pillar number four we are well known for our financial discipline and we plan to continue to deliver the rigor you have come to expect. Mike will go into great detail in his section of the call now to our 2023 results. We delivered outstanding performance in 2023, exceeding our guidance for the year, delivering record total revenues, both organically and through expansion of our product portfolio. These results continue to drive our momentum as we move through 2024. Our full year total revenue for 2023 were $398.2 million, an 85.9% increase year over year compared to $214.2 million in 2022. Additionally, we achieved record fourth quarter total revenues of $110.6 million, representing an 82% increase compared to $60.8 million in Q4 of 2022 we achieved our results by delivering [20%], 21% revenue growth for the full year of 2023. For Firdapse, a sales of $258.4 million and delivering $138.1 million in sales for flat comp. We are proud of these results, and we thank our team for delivering such a strong performance.

最後，第四個支柱是我們以財務紀律而聞名，我們計劃繼續提供您所期望的嚴格要求。麥克將在電話會議的部分詳細介紹我們的 2023 年業績。我們在 2023 年取得了出色的業績，超越了我們當年的指導，透過有機成長和產品組合的擴展實現了創紀錄的總收入。這些成果將繼續推動我們邁向 2024 年。我們 2023 年全年總營收為 3.982 億美元，與 2022 年的 2.142 億美元相比，年增 85.9%。此外，我們第四季的總營收達到創紀錄的1.106 億美元，與2022 年第四季的6,080 萬美元相比成長了82%，我們透過實現2023 年全年營收成長[20%] 21% 來實現我們的業績。Firdapse 的銷售額為 2.584 億美元，扁平化銷售額為 1.381 億美元。我們對這些結果感到自豪，並感謝我們的團隊提供如此出色的表現。

Looking ahead to 2024, our full year 2024 guidance reflects our confidence outlook, projecting a sustained growth and strategic advancements in line with our long-term objectives. We believe that 2024 will provide opportunities for continued growth of our portfolio. And we are forecasting full year total net revenues of between $455 million and $475 million, representing a 14% to 19% increase in our total net revenues compared to 2023. Provide you with more color on each of these pillars. I'll turn the presentation over to the team. Let's begin with Jeff Del Carmen, our Chief Commercial Officer. Jeff?

展望 2024 年，我們的 2024 年全年指引反映了我們的信心前景，預計將實現持續成長和策略進步，符合我們的長期目標。我們相信 2024 年將為我們的投資組合持續成長提供機會。我們預計全年總淨收入將在 4.55 億美元至 4.75 億美元之間，與 2023 年相比，我們的總淨收入將成長 14% 至 19%。為每個支柱提供更多色彩。我會將簡報轉交給團隊。讓我們從我們的首席商務官 Jeff Del Carmen 開始。傑夫？

Thanks, Rich, and good morning, everyone. First, I wanted to numerator each day and all the people touched by rare diseases. We are pleased with the outstanding performance delivered in 2023, highlighted by full year combined total revenues of $398.2 million, exceeding the upper end of guidance of $395 million. This remarkable performance was fueled by Firdapse, reaching an all-time high of $258.4 million and the consistent strong contribution from copper, which generated $138.1 million in net revenues.

謝謝里奇，大家早安。首先，我想計算每一天以及所有受到罕見疾病影響的人。我們對 2023 年取得的出色業績感到滿意，全年總收入達到 3.982 億美元，超出了 3.95 億美元的指導上限。這一非凡業績的推動因素是 Firdapse，達到 2.584 億美元的歷史新高，以及銅的持續強勁貢獻，產生了 1.381 億美元的淨收入。

Let's start by reviewing our advancements with Firdapse, the only evidence-based FDA approved treatment for Lambert, Eaton Myasthenic Syndrome. In the fourth quarter, net revenues amounted to $69.8 million, showcasing a remarkable year on year growth, 15%. Moreover, the overall net revenues at 2023 displayed a robust 20.8% growth compared to the previous year, propelled by consistent initiation of new patients in an annual low discontinuation rate trending below 20%, consistent with previous years and internal expectations. Prescription approval rates exceeded 90% for all types of payers, including government and private commercial insurers. Patients enrolled in Catalyst Pathways, including those with Medicare coverage, accessing third party Foundation assistance experienced an average monthly co-pay of less than $2. We are confident in the continued Firdapse organic growth in 2024 will be between 15% and 20% building upon the momentum from 2023. This growth will primarily be fueled by several key factors. Firstly, the conversion of a portion of the 500 identified lens patients to Firdapse treatment will play a significant role. Additionally, the anticipated approval of the 100 milligram expanded label in June 2024 should help contribute to this growth trajectory, providing patients the opportunity for a higher daily dose when appropriate.

讓我們先回顧一下我們在 Firdapse 方面取得的進展，這是 FDA 批准的唯一一種基於證據的治療伊頓肌無力症候群的蘭伯特療法。第四季淨收入達 6,980 萬美元，年增 15%，令人矚目。此外，由於新患者持續入組且年度停藥率趨勢低於 20%，2023 年的整體淨收入較上年強勁增長 20.8%，這與前幾年和內部預期一致。包括政府和私人商業保險公司在內的各類付款人的處方批准率均超過 90%。加入 Catalyst Pathways 的患者（包括那些擁有 Medicare 保險的患者）獲得第三方基金會援助的平均每月共付額不到 2 美元。我們相信，在 2023 年的勢頭基礎上，Firdapse 到 2024 年將持續實現 15% 至 20% 的有機成長。這種成長主要由幾個關鍵因素推動。首先，500 名已確定的晶狀體患者中的一部分轉而接受 Firdapse 治療將發揮重要作用。此外，預計 2024 年 6 月批准的 100 毫克擴展標籤應有助於促進這一增長軌跡，為患者提供在適當時獲得更高每日劑量的機會。

Furthermore, the expansion of educational programs targeting small cell lung cancer to lens health care providers is poised to accelerate lens diagnosis rates.

此外，將針對小細胞肺癌的教育計畫擴展到晶狀體醫療保健提供者，有望加快晶狀體診斷率。

Lastly, our efforts to broaden the addressable lens market through increased VGCC. antibody testing will further bolster growth prospects. As a reminder, we shared an abstract at the World Conference on Lung Cancer in September, which projected that the prevalence of lands in the United States is at least 3,600 data suggests it could potentially reach 5,400 individuals. This figure marks a notable rise from the previously estimated prevalence of 3,000. Our estimate stems from a thorough analysis of real-world data, particularly emphasizing the prevalence of lens diagnosis among patients with small cell lung cancer. Presently, we gauge that over 80% of small cell lung cancer lens patients remain undiagnosed, indicating a substantial growth opportunity.

最後，我們努力透過增加 VGCC 來擴大可尋址鏡頭市場。抗體測試將進一步提振成長前景。提醒一下，我們在 9 月的世界肺癌大會上分享了一份摘要，其中預測美國的肺癌盛行率至少為 3,600 人，數據表明該疾病可能會達到 5,400 人。這一數字較先前估計的 3,000 例盛行率顯著上升。我們的估計源於對現實世界數據的徹底分析，特別強調了小細胞肺癌患者中晶狀體診斷的盛行率。目前，我們估計超過 80% 的小細胞肺癌晶狀體患者仍未確診，這表明存在巨大的成長機會。

Now I'd like to share some key updates regarding our progress with by complex the commercialization of Kampo has surpassed our initial expectations in May 2023. We seamlessly integrated by Kampo into our product offerings as a strategic and highly accretive product. We actively engage with health care providers. Their key opinion leaders, expanded our outreach to establish vital partnership partnerships with patient advocacy groups to increase awareness and further our mission and fostered valuable relationships that have played a significant role and buy tons of success. By comparison, net revenues have exceeded the anticipated full year estimate of $130 million, reaching a total product revenue of $138.1 million, surpassing expectations by 6%. This achievement was propelled by robust end to the year with revenues for Q4 2023 reaching $39.3 million. We forecast 2024 by comp and net revenues will be approximately $130 million to $135 million based on changes in gross to net when products transition to companies with a smaller portfolio like we experienced with by copper, you typically encounter higher fees from service providers such as wholesalers let's turn to a gallery of promising novel anti-inflammatory corticosteroid aimed at addressing a significant gap in treatment for the estimated 11,000 to 13,000 boys living with Duchenne muscular dystrophy or DMD. As previously disclosed, we are on track for a commercial launch in the first quarter of 2024 Gamunex will be integrated into our neuromuscular franchise, capitalizing on the team's proven capabilities, commercial expertise and experience. We have finalized the recruitment of 10 commercial personnel to bolster the impending launch, primarily focusing on marketing and patient services. Our existing neuro muscular sales force comprising 16 regional account managers and two area business directors will assume responsibility for both Firdapse and a gambling. Furthermore, again, Marie will benefit from the support of our top notch Catalyst Pathways program, ensuring that all eligible patients have access to the product. Based on the feedback from our constituents and analysis of the DMD market, the wholesale acquisition cost of a January will be at a slight discount to Emflaza. We will implement the same financial programs as we have for Firdapse with a goal to ensure the average out-of-pocket cost per patient will be less than $2 per month. More details on the commercial launch will be available in the coming weeks. We began accepting precommercial launch new patient enrollments on December 1, 2023, based on the volume of new patient enrollments and observed pent-up demand. Our 2024 net revenue guidance for Camry is between $25 million and $30 million.

現在我想分享一些關於我們進展的重要更新，漢方的商業化在 2023 年 5 月已經超出了我們最初的預期。我們將漢方無縫整合到我們的產品中，作為一種策略性和高度增值的產品。我們積極與醫療保健提供者合作。他們的關鍵意見領袖擴大了我們的外展範圍，與患者倡導團體建立了重要的伙伴關係，以提高認識並進一步推進我們的使命，並培養了發揮重要作用並取得巨大成功的寶貴關係。相比之下，淨收入超過了全年預期的 1.3 億美元，產品總收入達到 1.381 億美元，超出預期 6%。這項成就得益於年底的強勁表現，2023 年第四季的營收達到 3,930 萬美元。根據總收入與淨收入的變化，我們預測2024 年的比較收入和淨收入將約為1.3 億美元至1.35 億美元，當產品轉向像我們在銅方面經歷的那樣擁有較小投資組合的公司時，您通常會遇到批發商等服務提供者收取的更高費用讓我們來看看一系列有前途的新型抗炎皮質類固醇，旨在解決估計11,000 至13,000 名患有杜氏肌肉營養不良症(DMD)的男孩的治療方面的巨大差距。如同先前所揭露的，我們預計在 2024 年第一季進行商業發布，Gamunex 將整合到我們的神經肌肉系列產品中，充分利用該團隊經過驗證的能力、商業專業知識和經驗。我們已經完成了 10 名商務人員的招聘，以支持即將推出的產品，主要負責行銷和患者服務。我們現有的神經肌肉銷售團隊由 16 名區域客戶經理和兩名區域業務總監組成，將負責 Firdapse 和賭博業務。此外，瑪麗將再次受益於我們一流的 Catalyst Pathways 計畫的支持，確保所有符合資格的患者都能使用該產品。根據我們的選民回饋和對DMD市場的分析，一月份的批發採購成本將比Emflaza略有折扣。我們將實施與 Firdapse 相同的財務計劃，目標是確保每位患者的平均自付費用每月低於 2 美元。有關商業發布的更多詳細資訊將在未來幾週內公佈。根據新患者入組量和觀察到的被壓抑需求，我們於 2023 年 12 月 1 日開始接受預商業啟動的新患者入組。我們對凱美瑞 2024 年的淨收入指引為 2,500 萬至 3,000 萬美元。

In conclusion, we are extremely proud of our achievements in 2023 and maintained strong confidence in meeting our targets for 2024 as we prepare for the launch of a gallery, we will leverage our established commercial proficiency. I express sincere appreciation to the entire Catalyst team for their unwavering dedication to patients, and I eagerly look forward to a prosperous 2024 ahead.

總而言之，我們對 2023 年的成就感到非常自豪，並對實現 2024 年目標保持堅定信心，在我們準備推出畫廊時，我們將利用我們既定的商業能力。我對整個 Catalyst 團隊對患者堅定不移的奉獻表示衷心感謝，並熱切期待 2024 年的繁榮。

I will now turn the call over to Dr. Steven Miller, our Chief Operating Officer and Chief Scientific Officer, for an update on R & D activities for Integer.

我現在將把電話轉給我們的營運長兼首席科學官 Steven Miller 博士，以了解 Integer 研發活動的最新情況。

I would first like to discuss our development efforts to increase the indicated maximum dose of Firdapse. As previously reported in October of [2023], the FDA accepted for review the company's supplemental new drug application to increase the indicated maximum 80 milligram daily dosage of Firdapse to [100] milligrams for the treatment of Liberty Myasthenic Syndrome or lapse catalyst continues to anticipate approval of the application on or about June 4, 2023, just the produce have actually been granted by the FDA. About 40% of patients are on the current indicated maximum dose of 80 milligrams per day, and their physicians would like the option to increase the daily dosage above 80 milligrams per day based upon disease progression catalyst believes that this expansion of the Firdapse label, if approved, would address an important opportunity for patients and their health care providers and may lead to about a 5% to 10% increase in the prescribed average daily dose of Firdapse over time.

我首先想討論我們為增加 Firdapse 的最大指示劑量而進行的開發工作。正如先前在[2023]10 月報道的那樣，FDA 接受審查該公司的補充新藥申請，將Firdapse 的指定最大每日80 毫克劑量增加至[100] 毫克，用於治療自由肌無力綜合症或失效催化劑繼續預期申請於 2023 年 6 月 4 日左右獲得批准，該產品實際上已獲得 FDA 批准。大約 40% 的患者目前的最大劑量為每天 80 毫克，他們的醫生希望選擇將每日劑量增加到每天 80 毫克以上，因為疾病進展催化劑相信 Firdapse 標籤的這種擴展，如果批准後，將為患者及其醫療保健提供者提供一個重要的機會，並可能導致Firdapse 的每日處方平均劑量隨著時間的推移增加約5% 至10%。

In 2023, we focused on expanding our intellectual property portfolio for Firdapse, resulting in the granting of two additional patents for Firdapse. These patents were issued at 2023 and 2024, bringing the total number of patents protecting Firdapse to nine. This further strengthens our intellectual property estate for Firdapse, which has patent protection to [2037]. Catalyst's expansion to global markets is continuing to progress. Our partner in Japan, Daido pharma achieved a notable milestone in December of 2023, having successfully submitted their NDA for Firdapse to the Japanese PMDA. We anticipate a priority review cycle time of nine months and if approved, a potential launch in early 2025 with 10 years of market exclusivity in Japan after approval.

2023 年，我們專注於擴大 Firdapse 的智慧財產權組合，從而為 Firdapse 授予了另外兩項專利。這些專利於 2023 年和 2024 年頒發，使保護 Firdapse 的專利總數達到 9 項。這進一步加強了我們 Firdapse 的智慧財產權，它擁有以下專利保護：[2037]。Catalyst 向全球市場的擴張不斷取得進展。我們在日本的合作夥伴大同製藥 (Daido pharma) 於 2023 年 12 月實現了一個顯著的里程碑，成功向日本 PMDA 提交了 Firdapse 的 NDA。我們預計優先審查週期為 9 個月，如果獲得批准，可能會在 2025 年初推出，並在批准後在日本享有 10 年的市場獨佔權。

Next, I'll move on to our newest approved product in January for the treatment of Duchenne muscular dystrophy or DMD and January was approved October 26th of last year and was granted new chemical entity exclusivity and orphan drug exclusivity. Conferring market exclusivity to October 2027 and 2030, respectively, that this FDA granted exclusivity is augmented by six Orange Book-listed patents that provide protection to [2040]. Again, as part of our ongoing effort to maximize Vanish and this will continue to seek opportunities to strengthen again this patent protection and explore new indications for labeling.

接下來，我將繼續介紹我們1 月最新批准的用於治療杜氏肌肉營養不良症(DMD) 的產品，該產品於去年10 月26 日獲得批准，並獲得了新化學實體獨佔權和孤兒藥獨佔權。FDA 分別授予 2027 年 10 月和 2030 年 10 月的市場獨佔權，並通過六項橙皮書列出的專利增強了這一授予的獨佔權，這些專利為[2040]。再次強調，作為我們不斷努力最大化 Vanish 的一部分，我們將繼續尋找機會再次加強專利保護並探索新的標籤適應症。

In summary, over Morila and clinical studies has shown efficacy for treatment of DMD patients while holding the potential for a reduction in the burden of steroid associated side effects compared on steroids. This includes the potential for notable benefits for bone health growth and behavioral outcomes based on preliminary data out of the estimated 11,000 to 13,000 DMD patients in the U.S., roughly 90% have undergone corticosteroid treatment at some stage. Presently, it is estimated that about 70% of DMD patients currently under treatment receive steroids. Steroids serve as the cornerstone of DMD therapy, highlighting the pressing demand for a more tolerable steroid option short term safety and efficacy of a more alone was demonstrated in a 24 week randomized, double-blind, placebo and active controlled study, and it is well tolerated and was associated with maintenance of muscle strength and function to determine what specific safety benefits again we may have, if any, relative to other approved corticosteroids catalysts is investing in a registry to study the long-term safety and quality of life at males with DMD that are treated with. Again, as this data is collected and analyzed, catalysts will periodically seek to update the gamma prescribing information with the latest information regarding the safety of the drug based upon FDA guidance. Documents for real-world data, education and information on new and unique products is the focus of Catalyst medical science liaisons for our pump based on our critical support to the medical community by providing information that is in the public domain to the medical community in order to educate them on these rare disease states and on the academic research that may have been published about these medications. This assist health care providers in selecting the best treatment for their patients, more alone has generated a high level of interest within the health care community. Neuromuscular MSL team is doing with the DMD medical community since the gamblers approval in October of last year. These There are numerous publications of the Marlins pharmacology and effect in DMD patient population. I'll not now turn the call over to Mike Kalb, our chief financial officer.

總之，Morila 和臨床研究表明，與類固醇相比，Morila 對治療 DMD 患者有效，同時有可能減少類固醇相關副作用的負擔。根據美國估計 11,000 至 13,000 名 DMD 患者的初步數據，其中約 90% 的患者在某個階段接受過皮質類固醇治療，這包括對骨骼健康生長和行為結果具有顯著益處的潛力。目前，據估計目前接受治療的 DMD 患者中約有 70% 接受類固醇治療。類固醇作為DMD 治療的基石，突顯了對更耐受的類固醇選擇的迫切需求，一項為期24 週的隨機、雙盲、安慰劑和主動對照研究證明了更單獨的短期安全性和有效性，並且效果良好耐受性並與維持肌肉力量和功能相關，以確定相對於其他批准的皮質類固醇催化劑，我們可能再次獲得哪些具體的安全益處（如果有的話）正在投資一個登記處，以研究男性的長期安全性和生活品質接受治療的DMD。同樣，在收集和分析這些數據時，催化劑將根據 FDA 的指導定期尋求使用有關藥物安全性的最新資訊來更新伽瑪處方資訊。有關新產品和獨特產品的真實世界數據、教育和資訊的文檔是我們泵的 Catalyst 醫學科學聯絡人的重點，基於我們通過向醫學界提供公共領域的信息來為醫學界提供重要支持，以便教育他們有關這些罕見疾病的知識以及可能已發表的有關這些藥物的學術研究。這有助於醫療保健提供者為患者選擇最佳治療方法，更重要的是在醫療保健界引起了高度的興趣。自去年 10 月批准賭徒以來，神經肌肉 MSL 團隊一直在與 DMD 醫學界合作。有許多關於馬林魚藥理學和對 DMD 患者群體的影響的出版物。我現在不會將電話轉給我們的財務長麥克·卡爾布。

Thank you, Steve. Capital's Fourth Quarter and Full Year 2023 financial performance resulted in another record breaking year, driven by exceptional execution by all facets of our business. In addition to our outstanding results, the Company has successfully closed two transactions in 2023, the acquisition of the U.S. rights to pay copper and a North American license for gaming. Both transactions provide the company with additional opportunity to continue to create incremental net positive cash flow through disciplined execution.

謝謝你，史蒂夫。在我們業務各個方面的出色執行力的推動下，Capital 的 2023 年第四季和全年財務表現再次打破紀錄。除了出色的業績外，該公司還在 2023 年成功完成了兩項交易，即收購美國銅支付權和北美博彩牌照。這兩筆交易都為公司提供了額外的機會，透過嚴格的執行繼續創造增量淨正現金流。

I'd like to take a moment to reiterate some of the key accounting implications and accounting treatment of the Gambro transaction as a result of the FDA approval of January on October 26th, 2023, we made a $36 million milestone payment to Centerra in the fourth quarter of 2023, which was capitalized on the balance sheet as an intangible asset and will be amortized on a straight-line basis over its estimated useful life of 10.5 years. I would like to reinforce that based on the timing of the January approval, amortization expense was prorated in the fourth quarter of 2023. Catalyst also made a strategic equity investment in center, which was recorded with an initial fair value of $13.5 million and will be mark to market value on a quarterly basis using the observable market price.

我想花點時間重申 Gambro 交易的一些關鍵會計影響和會計處理，這是 FDA 於 2023 年 10 月 26 日批准的結果，我們在第四季度向 Centerra 支付了 3600 萬美元的里程碑付款該資產將於2023 年第四季以無形資產在資產負債表上資本化，並在其預計使用壽命10.5 年內以直線法攤銷。我想強調的是，根據 1 月批准的時間，攤銷費用在 2023 年第四季按比例分配。Catalyst 也對 center 進行了策略性股權投資，初始公允價值為 1,350 萬美元，並將按季度使用可觀察市場價格按市值計價。

Now on to 2023 results. Our total net revenue for 2023 sorry, for 2023 was $398.2 million or 95.9% increase when compared to total net revenue of $214.2 million for 2022. Product revenue met for 2023 for Firdapse was $258.4 million, a 20.8% increase percent increase year over year compared to $213.9 million or 22 product revenue. Net profit comparable was $138.1 million for '23. As mentioned in our press release, 2024 or five comp and net product revenue will be adversely affected by gross-to-net adjustments in 2023. Our gross-to-net for Viacom was booked under sized arrangements with distributors and government agencies. However, starting in 2024 or all such costs are tied to arrangements between us and those distributors and government agencies. Since our costs under these arrangements are likely to be higher than the size costs. We expect to be impacted by an increase in gross to net deductions for Fycompa, thereby causing a corresponding decrease in PI comp and net product revenue. As Jeff mentioned, this typical for smaller companies with smaller product portfolios.

現在來看 2023 年的結果。抱歉，我們 2023 年的總淨收入為 3.982 億美元，與 2022 年的總淨收入 2.142 億美元相比增長了 95.9%。Firdapse 2023 年的產品收入為 2.584 億美元，與 22 個產品收入 2.139 億美元相比，年增 20.8%。2023 年的可比淨利潤為 1.381 億美元。正如我們在新聞稿中所提到的，2024 年或五月的比較和淨產品收入將受到 2023 年毛淨調整的不利影響。我們對維亞康姆的毛淨比是根據與分銷商和政府機構的規模安排預訂的。然而，從 2024 年開始，所有此類成本都將取決於我們與這些經銷商和政府機構之間的安排。因為我們在這些安排下的成本很可能高於規模成本。我們預計將受到 Fycompa 總扣除額與淨額扣除額增加的影響，導致 PI 補償和淨產品收入相應減少。正如傑夫所提到的，這對於產品組合較小的小公司來說是典型的。

Net income before income taxes for '23 was $94.5 million, a 9.7% decrease year over year compared to $104.7 million for '22. Net income for 2023 was adversely impacted by the $81.5 million charge for in-process R&D resulting from the Gambro license acquisition, we reported US GAAP net income for 2023 of $71.4 million, or $0.67 per basic share and $0.53 per diluted share a decrease of 14% year over year compared to GAAP net income for '22 of $83.1 million, or $0.8 per basic and $0.75 per diluted share. The decrease in net income is attributed to the one-time gain related IPR&D expense recorded during the third quarter of '23.

23 年所得稅前淨利為 9,450 萬美元，與 22 年的 1.047 億美元相比，年減 9.7%。2023 年淨利受到Gambro 許可收購產生的8,150 萬美元研發費用的不利影響，我們報告的2023 年美國GAAP 淨利潤為7,140 萬美元，即每股基本股0.67 美元，每股稀釋股0.53 美元，減少了14與去年同期相比，22 年 GAAP 淨利為 8,310 萬美元，即每股基本收益 0.8 美元，每股攤薄收益 0.75 美元。淨利潤的下降歸因於 23 年第三季記錄的與智慧財產權相關的一次性收益。

Non-gaap net income for '23 was $223.2 million or $2.10 per basic and $1.96 per diluted share, which excludes the IPR&D expense. The income tax provision of $23.1 million, amortization of intangible assets related to our acquisitions of resurging five comp in January of $32.6 million. Stock-based compensation expense of $14.3 million and depreciation expense of $316,000 from GAAP net income. This compares to non-GAAP net income for 2022 of $113.9 million or $1.10 per basic and $1.2 per diluted share, which excludes from GAAP net income. The income tax provision of $21.6 million, stock-based compensation of $7.9 million, amortization expense of research, the intangible assets of $1.1 million and depreciation expense of $141,000. The above represents an approximate 96% increase of non-GAAP net income year over year. Without consideration for potential M&A activity, we expect amortization expense for acquired licenses and intangible assets to be approximately $9.3 million, beginning with the first quarter of 2024 and continuing until the depletion of the intangibles related to PHI copper in 2027. Our effective tax rate for 2023 was 24.4%. We anticipate our effective tax rate to increase slightly for 2024. Our tax rate is affected by many factors and therefore may fluctuate. Quarterly cost of sales were approximately $52 million in 2023 compared to $34.4 million in '22 and consisted principally of royalties. As a reminder, royalties for Firdapse paid to our licensor for that product. It increased by 3% once net product sales exceed $100 million in any calendar year research and development expenses were $93.2 million in 2023. This compares to $19.8 million in 2022. As previously mentioned, the driver behind the increase in research and development expenses relates to the one-time. Again, we IPR&D expense during the third quarter of '23 relative to normal course of business. Absent another acquisition, we expect R&D costs in 2024 to trend.

23 年非 GAAP 淨利為 2.232 億美元，即每股基本收益 2.10 美元，稀釋每股收益 1.96 美元，不包括 IPR&D 費用。所得稅撥備為 2,310 萬美元，與我們 1 月收購復興五家公司相關的無形資產攤銷為 3,260 萬美元。GAAP 淨利潤中的股票薪酬費用為 1,430 萬美元，折舊費用為 316,000 美元。相比之下，2022 年非 GAAP 淨利潤為 1.139 億美元，即每股基本收益 1.10 美元，每股稀釋每股 1.2 美元，不包括 GAAP 淨利潤。所得稅準備金為2,160萬美元，股票薪酬為790萬美元，研究攤銷費用，無形資產為110萬美元，折舊費用為141,000美元。上述數據顯示非 GAAP 淨利潤年增約 96%。如果不考慮潛在的併購活動，我們預計從2024 年第一季開始，一直持續到2027 年與PHI 銅相關的無形資產耗盡之前，所獲得的許可證和無形資產的攤銷費用約為930 萬美元。我們 2023 年的有效稅率為 24.4%。我們預計 2024 年的有效稅率將小幅提高。我們的稅率受多種因素影響，因此可能會有所波動。2023 年的季度銷售成本約為 5,200 萬美元，而 22 年為 3,440 萬美元，主要由特許權使用費組成。提醒一下，Firdapse 的版稅已支付給我們該產品的授權人。一旦任何日曆年的產品淨銷售額超過 1 億美元，研發費用就會增加 3%，到 2023 年研發費用將達到 9,320 萬美元。相比之下，2022 年這一數字為 1,980 萬美元。如前所述，研發費用增加的背後驅動因素與一次性有關。同樣，我們在 2023 年第三季的智慧財產權與開發費用與正常業務過程相關。如果沒有另一項收購，我們預計 2024 年研發成本將呈現趨勢。

Similarly to R&D costs in 2022, SG&A expenses for 2023 totaled $133.7 million compared to $57.1 million in '22. The increase in SG&A year over year is principally due to expenses related to the integration of copper, including commercial expenses incurred under the transaction service agreement and an increase in headcount, principally in sales and marketing, as well as certain increases in corporate headcount required to support the Company's growth during the fourth quarter of '23, the company incurred significant selling and marketing expenses for the preparation of the game we launch.

與 2022 年的研發成本類似，2023 年的銷售、一般管理費用總計 1.337 億美元，而 2022 年為 5,710 萬美元。SG&A同比增加主要是由於與銅整合相關的費用，包括交易服務協議項下產生的商業費用和人員數量的增加，主要是銷售和營銷方面的人員數量的增加，以及為實現銅業務整合所需的企業人員數量的某些增加。為了支持公司在 2023 年第四季的成長，公司為準備我們推出的遊戲而產生了大量的銷售和行銷費用。

With regard to the first quarter of 2024, we anticipate a significant increase in 2024 SG&A expenses compared to Q1 of 2023 due to the upcoming January launch and the company's support of three commercial products compared to in Q1 '23.

關於2024 年第一季度，我們預計2024 年SG&A 費用與2023 年第一季度相比將大幅增加，因為與23 年第一季相比，由於1 月份即將推出以及該公司對三種商業產品的支持。

Further, I would like to note that consistent with prior years as a result of US GAAP requirements, the Company expects an increase in SG&A expense in the first quarter of 2024 as compared to other quarters due to its five o. one c. three donations. As reported, we ended 2023 with cash and cash equivalents of $137.6 million compared to $298.4 million at December 31, '22. The decrease in cash of $160.8 million was largely driven by approximately $293.3 million in payments in connection with acquisitions, including equity securities, which was offset by net positive cash flows generated from operations of $141.4 million.

此外，我想指出的是，與往年一樣，由於美國公認會計準則的要求，公司預計 2024 年第一季的 SG&A 費用將比其他季度有所增加，因為該公司的 5 個季度的財務狀況良好。一角三筆捐款。根據報道，截至 2023 年，我們的現金和現金等價物為 1.376 億美元，而截至 2022 年 12 月 31 日的現金和現金等價物為 2.984 億美元。現金減少 1.608 億美元，主要是由於與收購有關的約 2.933 億美元付款（包括股本證券）造成的，該減少額被運營產生的 1.414 億美元的淨正現金流所抵消。

On a related note, I would like to mention that on January ninth, 2024, the Company issued $10 million shares of common stock for total net cash proceeds of approximately $140.1 million, which is intended to fund the potential acquisition of new product candidates as well as for general corporate purposes. We believe our current funds continue to allow us 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 expansion efforts, leading to long-term growth and value creation. More detailed information analysis of Fourth Quarter and Full Year 2023 financial performance may be found in our annual report on Form 10 K, which was filed with the Securities and Exchange Commission yesterday, February 28th, and can be found on the Investor Relations page of our website at w. w. w. dot Cabelas pharma.com. And with that, I'll turn the call back over to Rich.

與此相關的是，我想提一下，2024 年 1 月 9 日，公司發行了 1000 萬美元普通股，淨現金收益總額約為 1.401 億美元，旨在為潛在收購新產品候選人提供資金至於一般公司目的。我們相信，我們現有的資金將繼續為我們提供財務靈活性，為我們現有的研發項目提供資金，履行我們潛在的合約義務，並支持我們的策略舉措、業務發展和投資組合擴張努力，從而實現長期成長和價值創造。有關 2023 年第四季和全年財務表現的更多詳細資訊分析，請參閱我們於 2 月 28 日向美國證券交易委員會提交的 10 K 表格年度報告，也可在我們的投資者關係頁面上找到。網站位於w. w。 w。點 Cabelas pharma.com。然後，我會將電話轉回給里奇。

Thanks, Mike. I'd like to thank the leadership team and all our employees at catalyst for the effort they put forth to deliver such outstanding results in 2023. We're off to a strong start in '24 with continued momentum to sustain our long-term durable growth. We continue to be committed to excellent execution. We are focused on increasing the scope and scale of our business through our disciplined approach in identifying derisked innovative therapies that address critical unmet medical needs in the rare orphan neuro and adjacent rare orphan therapeutic areas. We also hope to increase our geographic footprint through ex U.S. partnerships. In short, we are prepared to invest in opportunities that add value to Catalyst, further grow portfolio and provide improved care for patients. Our team and excuse me, our team is dedicated to improving the lives of more patients by working on key lifecycle management projects that have the potential to expand the use of the products we offer. We believe our financial rigor, strength of our financial results and our cash flow position us to advance our critical initiatives.

謝謝，麥克。我要感謝 Catalyst 的領導團隊和所有員工在 2023 年取得如此出色的表現所付出的努力。我們在 24 年有了一個良好的開端，並有持續的動力來維持我們的長期持久成長。我們繼續致力於卓越的執行。我們致力於透過嚴格的方法來擴大我們的業務範圍和規模，以識別無風險的創新療法，以滿足罕見孤兒神經和鄰近罕見孤兒治療領域關鍵的未滿足的醫療需求。我們也希望透過前美國合作夥伴關係擴大我們的地理足跡。簡而言之，我們準備好投資於為 Catalyst 增加價值的機會，進一步擴大產品組合併為患者提供更好的照護。我們的團隊，對不起，我們的團隊致力於透過開展關鍵的生命週期管理專案來改善更多患者的生活，這些專案有可能擴大我們提供的產品的使用。我們相信，我們的財務嚴謹性、財務表現的實力和現金流使我們能夠推進我們的關鍵舉措。

In summary, we are focused on our strategic priorities for 2024 number one, continue to deliver double digit growth for our portfolio in order to successfully launch and commercialize again, right? Number three, expansion of our product portfolio and our geographic partnerships in the CNS, orphan and orphan adjacent therapeutic spaces. And we look forward to providing you with updates on our progress at this time, I will turn it back turn the call back over to our operator. Kevin?

總而言之，我們的重點是 2024 年第一大策略重點，繼續為我們的產品組合帶來兩位數的成長，以便再次成功推出和商業化，對嗎？第三，擴大我們的產品組合以及我們在中樞神經系統、孤兒和孤兒鄰近治療領域的地理合作夥伴關係。我們期待此時向您提供有關我們進展的最新信息，我會將其轉回給我們的接線員。凱文？

Thank you.

謝謝。

We'll now be conducting a question and answer session. If you'd like to be placed in the question queue, please press star one on your telephone. As a reminder, we ask you please ask one question one follow-up to return to the queue, and that’s star one to be placed in the question queue.

我們現在將進行問答環節。如果您想加入問題隊列，請按下電話上的一號星星。提醒一下，我們要求您提出一個問題，然後再返回隊列，這是要放入問題隊列的明星問題。

Our first question is coming from Charles Duncan from Cantor Fitzgerald.

我們的第一個問題來自坎托·菲茨杰拉德的查爾斯·鄧肯。

Your line is now open.

您的線路現已開通。

Yes, good morning, Rich and team. Congratulations on a transformative year, and I appreciate the guidance going forward. Had a quick question on Firdapse with regard to that label expansion. Steve did a great job helping us understand, you know, the percentage of patients really that are looking for a higher dose, but I'm wondering what is the driver of that? What would there be anticipated in short term efficacy or long term durability?

是的，早上好，Rich 和團隊。祝賀變革的一年，我感謝未來的指導。在 Firdapse 上有一個關於標籤擴充的快速問題。史蒂夫做得很好，幫助我們了解真正尋求更高劑量的患者的百分比，但我想知道其驅動因素是什麼？短期功效或長期耐久性預期如何？

Charles, we believe that it will be long term durability. There's really two driving forces behind the first is disease progression as these patients age. And so they gradually very gradually need a higher dose.

查爾斯，我們相信它將是長期耐用的。實際上有兩個驅動力，第一個驅動力是隨著這些患者年齡的增長疾病進展。因此，他們逐漸需要更高的劑量。

The other thing also is that there are a handful of fast metabolizers out there who have higher weights that also need a higher dose. And so we'll be able to support those patients if and when the FDA approves this 100 milligram maximum daily dose.

另一件事是，有一些快速代謝者的體重較高，也需要較高的劑量。因此，如果 FDA 批准此 100 毫克的最大每日劑量，我們將能夠為這些患者提供支持。

Got it. And then was jumping over to Gary. One quick question and for Jeff or others on the team. I guess I'm wondering if you could provide a little bit more color on the registry. I think you mentioned that you would be starting that and some anticipated size and timing. Any updates from that that you anticipate that could drive awareness of the more loans, our value proposition versus other standard of care, thanks.

知道了。然後跳到加里身邊。有一個簡短的問題要問 Jeff 或團隊中的其他人。我想我想知道你是否可以在註冊表上提供更多的顏色。我想你提到過你將開始這個專案以及一些預期的規模和時間。您預期的任何更新都可以提高人們對更多貸款、我們的價值主張與其他護理標準相比的認識，謝謝。

Charles, the registry and the size is going to depend on how many patients I agreed to join the registry.

查爾斯，登記處和規模將取決於我同意加入登記處的患者數量。

It is voluntary. We anticipate that a significant number of patients will join the registry and the track for as long as shape and the patient registry in terms of duration, it will be carried on for many years as we collect data for long term safety evaluation of again, Gary do you want to make any commentary about the number of sites and excuse me, number of sites as well?

這是自願的。我們預計，只要形狀和患者註冊持續時間長，就會有大量患者加入註冊和跟踪，隨著我們再次收集數據以進行長期安全性評估，它將持續多年，加里您想對網站的數量發表評論嗎？請問還有網站的數量嗎？

So we will be looking at all of the sites that potentially enroll a commercial or to have commercial again, moderate patients and evaluate them for their ability to collect data rigorously in the registry. So at the present time, we haven't said a specific number. But we'll again, as Steve said, look at how many patients are prescribed, what the locations are in order to determine the site and patient numbers.

因此，我們將關注所有可能註冊商業廣告或再次進行商業廣告的網站，對患者進行審核，並評估他們在登記處嚴格收集資料的能力。所以目前我們還沒有說出具體的數字。但正如史蒂夫所說，我們將再次查看有多少患者被開處方，地點在哪裡，以確定地點和患者數量。

But I'd say just a sense of it's a pretty broad, but we're looking at a pretty broad-based approach that would that be safe to say?

但我想說，只是感覺它相當廣泛，但我們正在尋找一種相當廣泛的方法，可以肯定地說嗎？

Yes, right.

是的，沒錯。

Okay.

好的。

So we'll follow that health plans, things become clear, Charles,

所以我們將遵循健康計劃，事情變得清楚了，查爾斯，

Got it. thanks, Rich, for the added here.

知道了。謝謝里奇在這裡添加。

Thank you. Your next question is coming from Joon Lee from Truist Securities. Your line is open.

謝謝。您的下一個問題來自 Truist Securities 的 Joon Lee。您的線路已開通。

Great. Congrats on another strong quarter and thanks for taking the questions and really impressed that you are able to provide guidance on Admin Re, which has yet to launch. I can only imagine that you must be getting a lot of inbound interest to be able to provide guidance. Is this why you think just some additional sales force will be sufficient to launch into a 10,000 plus DMD patient space? And will it be more word-of-mouth awareness? Or do you have any DTC efforts in mind? Quick follow-up.

偉大的。恭喜又一個強勁的季度，感謝您提出問題，並且您能夠為尚未推出的 Admin Re 提供指導，這給您留下了深刻的印象。我只能想像，您一定會獲得很多入境興趣才能提供指導。這就是為什麼您認為只需增加一些銷售人員就足以進入擁有 10,000 多名 DMD 患者的空間嗎？會不會有更多的口碑宣傳？或者您有任何 DTC 計劃的想法嗎？快速跟進。

Sure.

當然。

So thanks for the question, Joon. We with our DMD. It's a very defined market. There are only primarily 250 prescribers that prescribe a 95% of the cortical steroids for boys living with DMD. And those 250 prescribers are housed in about 90 to 100 centers of excellence. So it's a focused call point that our team has the capacity, the team that was selling of Firdapse had the capacity to handle and handle effectively. And these are very experienced and tenured people. So we're very confident in that structure.

謝謝你的提問，瓊。我們和我們的 DMD。這是一個非常明確的市場。主要只有 250 名處方醫生為患有 DMD 的男孩開了 95% 的皮質類固醇處方。這 250 名處方醫生分佈在大約 90 到 100 個卓越中心。因此，這是一個重點呼叫點，我們的團隊有能力，銷售 Firdapse 的團隊有能力處理和有效處理。這些人都是經驗豐富、終身職的人。所以我們對這個結構非常有信心。

Now as far as the pent-up demand that you had mentioned. And it sounds like, as I mentioned on the call, we've been accepting pre-commercial launch enrollments since December and based on those enrollments and the number of enrollments that we've seen and also the the other stakeholders that have talked about their interest in going on in January. We do believe we're confident in the number that we provided so that's one thing.

現在就您所提到的被壓抑的需求而言。聽起來，正如我在電話中提到的，我們自 12 月以來一直在接受預商業發布註冊，並且基於這些註冊和我們看到的註冊數量以及其他利益相關者談論了他們的一月繼續的興趣。我們確實相信我們對我們提供的數字充滿信心，所以這是一回事。

As far as DTC., we have nonpersonal promotion along with our sales force efforts to help provide education on January as well as DMD. That will be focused on not only the physicians but also to other stakeholders like patients and caregivers.

就 DTC 而言，我們有非個人促銷活動以及我們的銷售人員的努力，以幫助提供 1 月份的教育以及 DMD。這不僅會關注醫生，還會關注患者和照護者等其他利害關係人。

Great. Thanks for the color. And on the continued strength of your Firdapse franchise, you mentioned conversion of 500 additional lung patients you've identified and the anticipated approval of the higher 100 milligram dose. But then how does a small cell lung cancer lens patients factor into that guidance, is that really in the guidance? Or is there more upside or help us navigate that, that component of the population? Thank you.

偉大的。謝謝你的顏色。關於 Firdapse 特許經營權的持續成長，您提到了您已確定的另外 500 名肺部患者的轉換以及預期批准更高的 100 毫克劑量。但是，小細胞肺癌晶狀體患者如何納入該指南，這真的在指南中嗎？還是有更多的好處或幫助我們駕馭這部分人口？謝謝。

So June, a great question about that and it is factored into small cell lung cancer lens opportunity is factored in into the growth potential, but we also see it as upside from there, too. And like I mentioned, about 80% of these small cell lung cancer lung patients are unfortunately undiagnosed at this point. So our efforts to accelerate that diagnosis, the diagnosis for these patients will increase the addressable market over time, which will help with our growth.

因此，六月，對此有一個很大的問題，它被納入小細胞肺癌晶狀體機會的考慮因素，也被納入增長潛力的因素，但我們也認為它也有上行空間。正如我所提到的，不幸的是，大約 80% 的小細胞肺癌患者目前尚未確診。因此，我們努力加速診斷，對這些患者的診斷將隨著時間的推移增加潛在市場，這將有助於我們的成長。

Great. Congrats again on the strong quarter.

偉大的。再次恭喜本季的強勁表現。

Thank you.

謝謝。

Thank You.

謝謝。

Thanks very much to thank you.

非常感謝您。

next question is coming from Joe Catanzaro from Piper Sandler. Your line is open.

下一個問題來自 Piper Sandler 的 Joe Catanzaro。您的線路已開通。

Yes, hi, guys. Appreciate you taking my question and congrats on all the great progress here. So Steve, I think you mentioned the percent of patients that are on the current maximum dose of Firdapse. But I think you previously said that you would expect even patients some on lower doses to be taken to higher doses. I'm wondering if that's still your expectation and if that sort of factored into the Firdapse guidance for 2024.

是的，嗨，夥計們。感謝您提出我的問題，並祝賀這裡取得的所有巨大進展。Steve，我想您提到了目前服用 Firdapse 最大劑量的患者的百分比。但我想你之前說過，你會期望即使是服用較低劑量的患者也會服用較高劑量。我想知道這是否仍然是您的期望，以及這是否已納入 Firdapse 2024 年指南中。

Thanks.

謝謝。

And I have one quick follow-up.

我有一個快速跟進。

I'll let Jeff address the factored into the guidance. But with regard to your question about patients being on doses below 80 milligrams a day as there are patients on that, the norm being a neuromuscular physician community is somewhat conservative. It's a go-slow of start low and go slow on mentality with regards to medical industry. Many of them like to lose ground also headspace in terms of the dose that patients are receiving so that if the patient gets worse, they can increase the dose with the increase in the daily maximum dosage, we would expect some of those conservative physicians who have been concerned about the overall efficacy of, say, a 60 milligram dose that a patient may have been receiving maybe more likely to increase the dose, knowing that they still have more room to further increase the dose for those patients.

我將讓傑夫解決指南中所考慮的因素。但是，關於您關於患者每天服用劑量低於 80 毫克的問題，因為確實有患者服用該劑量，神經肌肉醫生群體的標準有些保守。對醫療產業來說，這是一種低起點、慢節奏的心態。他們中的許多人喜歡在患者接受的劑量方面失去優勢，這樣如果患者病情惡化，他們可以隨著每日最大劑量的增加而增加劑量，我們預計一些保守的醫生會擔心患者可能已經接受的60 毫克劑量的整體療效可能更有可能增加劑量，因為知道他們還有更多空間進一步增加這些患者的劑量。

So that that is you're correct that some of those patients at below 80 milligrams may increases adjusted you want to address factored into.

因此，您的說法是正確的，其中一些體重低於 80 毫克的患者可能會增加您想要解決的因素。

Sure, Joe. Before I get into that question, just wanted to reiterate the percentages that 40% of our patients are currently on 80 milligrams, then there's an incremental 3% of patients are on greater than 80 milligrams currently on overtime, we do expect the average daily dose to increase and that increase that gradual increase is factored in on to the forecast. But keep in mind with the due for date for this June arm, it's overtime. So it's just the impact from June and through December of this year.

當然，喬。在我討論這個問題之前，我只想重申一下，我們 40% 的患者目前服用 80 毫克的百分比，然後有 3% 的患者加班目前服用超過 80 毫克的劑量，我們確實預計平均每日劑量增加，並且逐漸增加的增加已納入預測。但請記住，六月的截止日期是加班。所以這只是今年六月到十二月的影響。

Okay, thanks. That's helpful. And then maybe my follow-up question on sort of future Cadence business development and maybe one for you, Rich. As we as we look back at sort of our historical approach to business development? How are you thinking about some potential opportunities moving forward?

好的謝謝。這很有幫助。然後我可能會問有關 Cadence 未來業務發展的後續問題，也許還有一個適合你的問題，Rich。當我們回顧我們歷史上的業務發展方法？您如何看待未來的一些潛在機會？

Great question. So obviously, we feel that our balance sheet is prime right now. And we're really confident and pretty and her team are continuing to look, we have a very wide aperture. So we consider ourselves an orphan company, obviously in the CNS space. So that would include epilepsy as well. And we're looking at those opportunities that are not only just in CNS because we believe our infrastructure is one that supports getting the patient on and keeping the patient on that's critical. We see that as a foundational element of who we are as a company, we believe that infrastructure is trans is transferable to other orphan states outside of CNS, metabolic, cardiovascular, et cetera. So our aperture is quite wide. We believe our opportunities are very, very broad. So we continue to scan the landscape. We have a number of opportunities that we're evaluating at a very deep level and we're continuing there. And we're, I would say, appropriately aggressive in our search for new opportunities that we believe fit with who we are and our ability to serve the patient community.

很好的問題。顯然，我們認為我們的資產負債表目前處於最佳狀態。我們真的很有自信，也很漂亮，她的團隊正在繼續尋找，我們有一個非常寬的光圈。因此，我們認為自己是一家孤兒公司，顯然是在中樞神經系統領域。所以這也包括癲癇。我們正在尋找這些機會，這些機會不僅存在於中樞神經系統領域，因為我們相信我們的基礎設施能夠支持患者繼續治療，這一點至關重要。我們認為，作為我們公司的基本要素，我們相信基礎設施是可以轉移到中樞神經系統、代謝、心血管等領域之外的其他孤兒狀態的。所以我們的光圈相當大。我們相信我們的機會非常非常廣泛。所以我們繼續檢視風景。我們正在對許多機會進行深入評估，並將繼續進行評估。我想說的是，我們在尋找新的機會時適當積極，我們認為這些機會適合我們的身份和我們為患者社區服務的能力。

Okay, great. Thanks again and appreciate you taking the question.

好的，太好了。再次感謝並感謝您提出問題。

Thank you.

謝謝。

Thank you.

謝謝。

Our next question is coming from, we will go from Oppenheimer.

我們的下一個問題來自奧本海默。

Your line is lower now.

你的線現在更低了。

Yes, good morning, everyone, and thanks for taking my questions and great to see the sun Clayton from scammers and as well as overall.

是的，大家早上好，感謝您提出我的問題，很高興看到克萊頓免受騙子和整體的影響。

Just a few questions from me.

我只想問幾個問題。

Actions by Compass looks like now with the guidance, let's say we've been more flattish year on year and 24, then I think previously, the Company indicated maybe sort of a mid to high-single digits growth. Just wondering and any dynamics there we should be aware of.

康帕斯的行動看起來像現在的指導，假設我們的同比和 24 年更加持平，那麼我認為之前，該公司可能表示可能有中等到高個位數的增長。只是想知道我們應該注意的任何動態。

And then second, also, ones like copper. Is there any update with respect to IP exclusivity?

其次，還有銅之類的。關於IP獨佔性有任何更新嗎？

I think a base case. We have this potential D-2 all sort of Spring of 25 and then Central to that extended to 26. Just wondering if there's any update on the IP that.

我認為有一個基本情況。我們在25年春季都有這個潛在的D-2，然後擴展到26年的核心。只是想知道IP是否有任何更新。

Thank you.

謝謝。

Currently lines.

目前線路。

So Mike will take the first part and Steve will take the second part Sure.

所以麥克將承擔第一部分，史蒂夫將承擔第二部分當然。

Good morning, Leo, and thanks for the question on Onfi copper, as we mentioned in the press release as well as on the call from our our arrangements now with wholesalers and distributors and government agencies are through us as opposed to for 2023. They were through a site and as is common for companies of our size, smaller companies, smaller product portfolios, those those rates are are going to be slightly higher.

早安，Leo，感謝您提出有關Onfi 銅的問題，正如我們在新聞稿中以及我們在電話中提到的那樣，我們現在與批發商、分銷商和政府機構的安排是透過我們進行的，而不是2023 年。他們是透過一個網站進行的，對於我們這種規模的公司來說，較小的公司、較小的產品組合很常見，這些費率會略高一些。

OffHighway with with regard to the intellectual property, we have completed our work appearing drug substance houses and apartments and the expiration date of May of 2025 is the we'll see we execute them and have exclusivity for that patent for them for a composition of matter. However, we are still evaluating whether or not to enforce the polymorph patents. And we hope to conclude that evaluation in the near future.

OffHighway 在智慧財產權方面，我們已經完成了藥物房屋和公寓的工作，截止日期為 2025 年 5 月，我們將看到我們執行它們，並擁有它們的物質組合專利的排他性。然而，我們仍在評估是否執行多晶型專利。我們希望在不久的將來完成評估。

And I just want to make a final comment about by cabo. One of the reasons why we like this asset losses for the financial opportunity. We believe that this compound has life significant life beyond its patent expiry because of the stickiness of epilepsy drugs of patients with epilepsy do not like to change drugs even post patent expiry. So there is a great a willingness for the patient to continue on their branded medication receive is. So there's a significant opportunity for this compound post expiration to continue to deliver good return for the Company. And that was baked into our calculus when we bought the product. So we continue to see this as a very good opportunity to serve patients and to build the balance sheet and again, help us to build our portfolio as we go forward. So we see this as a really good smart financial play and a good play for patients as well.

我只想對 cabo 做最後的評論。我們喜歡這種資產損失換取金融機會的原因之一。我們相信，這種化合物在專利到期後仍具有重要的生命力，因為癲癇藥物具有黏性，即使在專利到期後，癲癇患者也不喜歡更換藥物。因此，患者非常願意繼續接受他們的品牌藥物。因此，該化合物在到期後有很大機會繼續為公司帶來良好的回報。當我們購買產品時，這一點就被納入我們的計算中。因此，我們仍然認為這是一個很好的機會，可以為患者提供服務，建立資產負債表，並再次幫助我們在前進的過程中建立我們的投資組合。因此，我們認為這是一個非常好的、明智的財務投資，對病人來說也是一個很好的投資。

I think that's very helpful. And then just one question on Firdapse, if I may, I think the mention of about 500 remaining diagnosed but untreated patients. If I if I have that correctly, just wondering if you could point a ticking of hurdles and they may face in converting those untreated but diagnosed patients. So there are plenty.

我認為這非常有幫助。然後是關於 Firdapse 的一個問題，如果可以的話，我想提到了大約 500 名剩餘的確診但未經治療的患者。如果我的說法正確的話，我只是想知道您是否可以指出一些障礙，他們在轉變那些未經治療但已確診的患者時可能面臨的障礙。所以有很多。

So we lend on a 50% about 50% of our new enrollments each month or any given quarter come from these leads. These patients that are diagnosed but never have been treated with Firdapse. We use a variety of sources to continually backfill those patients so as one patient gets converted on to on Firdapse, we find new leads to backfill those patients. So we always tried to maintain those 500 or so patient leads as far as hurdles to get on a lot of times, it's just it's a matter of these patients waiting to get into see their physicians, whether it's a three month or six month timeframe that they're waiting to get in, and that's primarily it. There are some patients within these 500 that have been on the or a patient lead for over a year. But most of these, the newer leads can be converted within 30 days. That's how we see it. But again, we backfill those patients converted with new leads over time.

因此，在我們每月或任何特定季度的新註冊人數中，大約 50% 的貸款來自這些潛在客戶。這些患者被診斷但從未接受過 Firdapse 治療。我們使用各種來源不斷回填這些患者，以便當一名患者轉而使用 Firdapse 時，我們會找到新的線索來回填這些患者。因此，我們總是盡力維持這 500 名左右的患者線索，但遇到很多困難，只是這些患者等待去看醫生的問題，無論是三個月還是六個月的時間範圍他們正在等待進入，僅此而已。這 500 名患者中有些患者已經使用導極或患者導極超過一年。但其中大多數新的潛在客戶可以在 30 天內轉換。我們就是這樣看的。但隨著時間的推移，我們再次用新的線索回填那些轉化的病人。

I think we've learned it's all part of what we would call it. You know, this is the patient journey, and it's really dependent upon the physician and the patient and where they come in and how aggressive they want to be with their own therapy. And so once we identify them, we keep calling on the physician and working with the physician to expedite therapy if it's appropriate, but it really comes down the physician. But once we identify the physician has the patient, we can then begin to help the physicians understand the disease state and then the patient journey and how we how Firdapse can help that patient.

我想我們已經了解到這就是我們所說的一切的一部分。你知道，這是患者的旅程，它實際上取決於醫生和患者以及他們的介入程度以及他們希望自己的治療有多積極。因此，一旦我們識別出它們，我們就會不斷打電話給醫生，並與醫生合作，如果合適的話，加快治療速度，但這實際上取決於醫生。但是，一旦我們確定醫生接診了患者，我們就可以開始幫助醫生了解疾病狀態，然後了解患者的旅程以及 Firdapse 如何幫助該患者。

Appreciate the added color.

欣賞添加的顏色。

Thank you.

謝謝。

Thank you.

謝謝。

Thank you.

謝謝。

We've reached the end of our question and answer session.

我們的問答環節已經結束。

And ladies and gentlemen, that does conclude today's teleconference and webcast. We do thank you for your participation.

女士們、先生們，今天的電話會議和網路廣播到此結束。我們非常感謝您的參與。

You may have a nice day.

你可能會度過愉快的一天。