Harmony Biosciences Holdings Inc (HRMY) 2025 Q3 法說會逐字稿

Good morning. My name is Madison, and I will be your conference operator today. At this time, I would like to welcome everyone to Harmony Biosciences third quarter 2025 financial results conference call. (Operator Instructions) Please be advised that today's conference may be recorded. (Operator Instructions)

早安.我叫麥迪遜，今天我將擔任你們的會議接線生。在此，我謹代表Harmony Biosciences公司，歡迎各位參加2025年第三季財務業績電話會議。（操作人員說明）請注意，今天的會議可能會被錄音。（操作說明）

I will now turn the call over to Matthew Beck. Please go ahead.

現在我將把電話交給馬修貝克。請繼續。

Thank you, operator. Good morning, everyone, and thank you for joining us today as we review Harmony Biosciences third quarter 2025 financial results and provide a business update. Before we start, I encourage everyone to go to the Investors section of our website to find the materials that accompany our discussion today, including the reconciliation of our GAAP to non-GAAP financial measures. At this stage of our life cycle, we believe non-GAAP financial results better represent the underlying business performance.

謝謝接線生。各位早安，感謝大家今天收看 Harmony Biosciences 2025 年第三季財務業績回顧和業務更新。在開始之前，我鼓勵大家造訪我們網站的「投資者」版塊，尋找與我們今天討論相關的資料，包括我們的 GAAP 與非 GAAP 財務指標的調整表。在我們生命週期的現階段，我們認為非GAAP財務表現更能反映公司的實際經營狀況。

Our speakers on today's call are Dr. Jeffrey Dayno, President and CEO; Adam Zaeske, Chief Commercial Officer; Dr. Kumar Budur, Chief Medical and Scientific Officer; and Sandip Kapadia, Chief Financial and Administrative Officer.

今天電話會議的發言人有：總裁兼首席執行官 Jeffrey Dayno 博士；首席商務官 Adam Zaeske；首席醫療和科學官 Kumar Budur 博士；以及首席財務和行政官 Sandip Kapadia。

As a reminder, we will be making forward-looking statements today, which are based on our current expectations and beliefs. These statements are subject to certain risks and uncertainties. Our actual results may differ materially, and we undertake no obligation to update these statements even if circumstances change. We encourage you to consult the risk factors referenced in our SEC filings for additional details.

再次提醒大家，我們今天將發表一些前瞻性聲明，這些聲明是基於我們目前的預期和信念。這些聲明存在一定的風險和不確定性。我們的實際結果可能與此有重大差異，即使情況發生變化，我們也沒有義務更新這些聲明。我們建議您查閱我們向美國證券交易委員會提交的文件中提及的風險因素，以了解更多詳情。

I would now like to turn the call over to our CEO, Dr. Jeffrey Dayno. Jeff?

現在我謹將電話交給我們的執行長傑弗瑞·戴諾博士。傑夫？

Thank you, Matt. Good morning, everyone, and thank you for joining our call today. I want to start off by saying how proud I am of the Harmony team and their exceptional performance this quarter. It reaffirms Harmony's reputation for executional excellence. As we have shared, Q3 was a very strong quarter for Harmony.

謝謝你，馬特。各位早安，感謝各位今天參加我們的電話會議。首先，我想表達我對 Harmony 團隊及其本季出色表現的自豪之情。這再次印證了Harmony在執行力上的卓越聲譽。正如我們之前所說，第三季是Harmony表現非常強勁的一個季度。

We reported $239.5 million in net revenue for the quarter, representing 29% growth year-on-year. And with this momentum, we recently raised our net revenue guidance for the year from $820 million to $860 million, taking it up to $845 million to $865 million. We had robust cash generation of $106 million, bringing our balance sheet to $778 million as of September 30.

本季淨收入為 2.395 億美元，年增 29%。憑藉這一勢頭，我們最近將今年的淨收入預期從 8.2 億美元上調至 8.6 億美元，達到 8.45 億美元至 8.65 億美元。截至 9 月 30 日，我們實現了強勁的現金流，達到 1.06 億美元，資產負債表總額達到 7.78 億美元。

But what I am most proud of is that this performance was driven by the highest number of quarterly patient adds for WAKIX since our launch with an average of 500 patients added this quarter, resulting in an average of 8,100 patients on WAKIX at the end of Q3. Our Chief Commercial Officer, Adam Zaeske, will next be providing more color on some of the things his team is doing to drive this strong momentum in our WAKIX business.

但我最引以為傲的是，這項業績得益於 WAKIX 自推出以來季度新增患者數量最高，本季平均新增 500 名患者，使得 WAKIX 在第三季末的平均患者人數達到 8100 人。接下來，我們的首席商務長 Adam Zaeske 將詳細介紹他的團隊正在採取哪些措施來推動 WAKIX 業務的強勁發展勢頭。

There are many different ways to measure impact. Delivering innovative treatments to patient populations living with unmet medical needs is a very meaningful one. With this sustained momentum, we believe WAKIX is rapidly approaching a $1 billion-plus blockbuster status in narcolepsy alone.

衡量影響力的方法有很多種。為存在未滿足醫療需求的患者群體提供創新療法是一件非常有意義的事情。憑藉這種持續的成長勢頭，我們相信 WAKIX 僅在嗜睡症領域就將迅速成為一款銷售額超過 10 億美元的重磅產品。

Along with our very strong commercial business, Harmony also has a robust late-stage pipeline with multiple catalysts coming over the next several years. We continue to have firm conviction in our pipeline and full confidence in our R&D team to successfully execute on these programs. In fact, the IND for pitolisant HD, our pitolisant high-dose formulation has been submitted to FDA, and we are on track to initiate two Phase 3 trials, one in narcolepsy and in idiopathic hypersomnia or IH before the end of the year. Kumar will provide an update on our pipeline programs later in the call, including an update on the RECONNECT study in Fragile X syndrome.

除了我們非常強大的商業業務外，Harmony 還擁有強大的後期研發管線，未來幾年將有多個催化劑問世。我們對我們的產品線充滿信心，並對我們的研發團隊充滿信心，相信他們能夠成功執行這些專案。事實上，我們的匹托利桑高劑量製劑匹托利桑HD的IND申請已提交給FDA，我們正按計劃在年底前啟動兩項3期試驗，一項針對發作性睡病，另一項針對特發性嗜睡症或IH。Kumar 將在電話會議的稍後部分介紹我們的研發管線專案進展，包括針對脆性 X 症候群的 RECONNECT 研究的最新進展。

Turning to our balance sheet. With over $778 million in cash and cash equivalents, a disciplined approach to capital deployment, and a team with extensive industry experience, Harmony is well positioned to strategically pursue value-enhancing assets to add to our pipeline and build a broader product portfolio. That is our intent and a key component of our vision to become the leading patient-focused CNS company, delivering innovative treatments that can help even more patients living with unmet medical needs.

接下來來看看我們的資產負債表。Harmony 擁有超過 7.78 億美元的現金及現金等價物，採取了嚴謹的資本部署方式，並擁有一支經驗豐富的行業團隊，因此能夠有條不紊地尋求能夠提升價值的資產，以豐富我們的產品線並構建更廣泛的產品組合。這就是我們的目標，也是我們願景的關鍵組成部分，即成為以患者為中心的領先中樞神經系統公司，提供創新療法，幫助更多未滿足醫療需求的患者。

We believe that we have built something rare in our industry, a profitable, self-funding biotech company with an innovative catalyst-rich pipeline poised to deliver meaningful value for both patients and our shareholders. It is because of this unique profile that we continue to execute from a position of strength. And coming off of our exceptional Q3 performance, we believe that Harmony is one of the most compelling growth stories in biotech today.

我們相信，我們已經在業界打造了罕見的事物，一家盈利的、自籌資金的生物技術公司，擁有創新且富含催化劑的產品線，有望為患者和股東帶來有意義的價值。正是由於這種獨特的優勢，我們才能持續保持強勁的發展勢頭。鑑於我們第三季出色的業績，我們相信 Harmony 是當今生技領域最具吸引力的成長故事之一。

With that, I'll turn the call over to Adam Zaeske, our Chief Commercial Officer, for an update on our outstanding commercial performance. Adam?

接下來，我將把電話交給我們的商務長亞當‧札斯克，請他介紹一下我們出色的商業表現。亞當？

Thank you, Jeff. Harmony's Q3 2025 results were the strongest we've seen since launch. WAKIX delivered $239.5 million in net sales for the quarter, representing nearly 30% year-over-year growth in its sixth year on the market. WAKIX achieved a record increase in approximately 500 average patients for the quarter. This represents the highest quarterly increase we've ever seen and comes after the Q2 increase of approximately 400 average patients, which has only been achieved twice previously. As a result, WAKIX achieved approximately 8,100 average patients for Q3, exceeding our previous guidance of achieving nearly 8,000 patients by a full quarter.

謝謝你，傑夫。Harmony 2025 年第三季的業績是自發布以來最強勁的。WAKIX 本季淨銷售額為 2.395 億美元，在其上市第六年實現了近 30% 的同比增長。WAKIX 本季平均患者人數增加了約 500 人，創歷史新高。這是我們所見過的最高季度增幅，此前第二季度平均患者人數增加了約 400 人，而此前只有兩次達到這一數字。因此，WAKIX 第三季平均病患人數約為 8,100 人，比我們先前預期的季度平均病患人數（接近 8,000 人）多了一個季度。

The foundation of WAKIX performance has always been the unique position WAKIX owns as the only non-scheduled treatment option, resulting in its broad clinical utility. WAKIX enjoys extremely high brand awareness, is perceived as efficacious and well tolerated and is supported by broad payer coverage that has remained consistent for years.

WAKIX 表現的基礎始終在於其作為唯一非處方治療方案的獨特地位，從而造就了其廣泛的臨床應用價值。WAKIX 品牌知名度極高，被認為療效顯著且耐受性良好，並且得到廣泛的醫保覆蓋，多年來一直保持穩定。

In addition to this, I'd like to highlight some recent areas of focus. We have adjusted our field sales deployment, call plan, and messaging and continue to deliver sound sales execution. We've refined our promotion and messaging. We've secured important new payer coverage wins, which continue to expand our already broad payer coverage.

除此之外，我還想重點介紹一下我們最近的一些研究領域。我們調整了現場銷售部署、拜訪計畫和資訊傳遞方式，並持續保持良好的銷售執行力。我們改進了宣傳推廣和訊息傳遞方式。我們已成功獲得重要的新的支付方承保，這將進一步擴大我們已有的廣泛支付方承保範圍。

And we have made improvements in how we support patients moving from a WAKIX prescription to dispense, reflected in higher rates of conversion and shorter times to dispense. Much of what we're focused on are the fundamentals from sales execution, marketing and promotion, payer coverage, and patient support. The adjustments we are making are delivering results, and we will continue to look for additional opportunities in all areas moving forward.

我們在支持患者從 WAKIX 處方過渡到配藥方面取得了進步，這體現在更高的轉化率和更短的配藥時間上。我們關注的重點大多是銷售執行、行銷和推廣、支付方覆蓋範圍以及患者支援等方面的基本要素。我們正在進行的調整正在取得成效，未來我們將繼續在各個領域尋找更多機會。

As we look to the fourth quarter of 2025, we expect continued growth in average number of patients and momentum. As a result, we recently raised our full-year revenue guidance of $820 million to $860 million to the high end of the range between $845 million and $865 million, and we are rapidly approaching achieving $1 billion-plus in annual revenue from narcolepsy alone.

展望 2025 年第四季度，我們預期平均患者人數和成長動能將持續維持。因此，我們最近將全年營收預期從 8.2 億美元至 8.6 億美元上調至 8.45 億美元至 8.65 億美元區間的高端，並且我們正迅速接近僅靠嗜睡症就能實現 10 億美元以上的年收入。

Looking to the future, the pitolisant GR and HD formulations each target significant unmet patient needs while extending our growth potential with utility patents filed through 2044. Early feedback from physicians and payers on the HD formulation has been particularly encouraging, and we will be able to leverage our commercial infrastructure to drive the next phase of growth through our pitolisant franchise formulations.

展望未來，匹托利桑 GR 和 HD 製劑均針對尚未滿足的重大患者需求，同時透過申請實用新型專利，將我們的成長潛力擴展至 2044 年。來自醫生和支付方的早期回饋對 HD 配方尤其令人鼓舞，我們將能夠利用我們的商業基礎設施，透過我們的匹托利桑系列配方推動下一階段的成長。

In summary, the performance of our business has never looked better, fueled by a highly differentiated product, a focus on fundamentals, and excellent execution across the organization. We are confident in our continued growth and performance moving forward.

總而言之，我們公司的業績從未如此出色，這得益於高度差異化的產品、對基本面的關注以及整個組織的出色執行。我們對未來的持續成長和業績充滿信心。

And now I'd like to turn the call over to our Chief Medical and Scientific Officer, Kumar Budur, to discuss the advancements in our clinical development programs. Kumar?

現在，我想把電話交給我們的首席醫療和科學官庫馬爾·布杜爾，讓他來討論我們臨床開發項目的進展。庫馬爾？

Thank you, Adam. Good morning, everyone, and thank you for joining us today. We continue to make good progress in R&D, with three Phase 3 registrational studies ongoing and anticipate up to five Phase 3 registrational studies in five distinct indications by the end of the year. And we have some important updates to share on the next-gen pitolisant programs.

謝謝你，亞當。各位早安，感謝大家今天收看我們的節目。我們在研發方面持續取得良好進展，目前有三項 3 期註冊研究正在進行中，預計到今年年底將有多達五項針對五種不同適應症的 3 期註冊研究。我們還有一些關於下一代匹托利桑計畫的重要更新要與大家分享。

Starting with our Sleep/Wake franchise, we continue to make significant progress across our next-gen pitolisant programs. I'm pleased to report that we have submitted the IND for pitolisant HD to the FDA. The pitolisant HD program, an enhanced formulation with an optimized PK profile and higher dose, targeting enhanced efficacy for excessive daytime sleepiness and pursuing a differentiated label with an indication for fatigue in narcolepsy is on track for Phase 3 initiation in Q4 2025.

從我們的睡眠/覺醒產品系列開始，我們在下一代匹托利桑計畫中繼續取得重大進展。我很高興地報告，我們已經向 FDA 提交了 pitolisant HD 的 IND 申請。pitolisant HD 計畫是一種增強型配方，具有優化的 PK 特性和更高的劑量，旨在提高對白天過度嗜睡的療效，並尋求差異化標籤，用於治療發作性睡病引起的疲勞，該計畫預計在 2025 年第四季度啟動 3 期臨床試驗。

Similarly, the Phase 3 study with pitolisant HD in patients with idiopathic hypersomnia is also pursuing a differentiated label with an indication for sleep inertia, and we are on track for initiation in Q4 2025. The target PDUFA dates for both programs are in 2028. The other next-gen pitolisant formulation, pitolisant GR, is designed to minimize the potential for treatment-related GI side effects, especially since almost 90% of patients with narcolepsy experience comorbid GI symptoms. In addition, pitolisant GR also provides an ability to start at the therapeutic dose range at 17.8 milligrams, eliminating the need for titration, an important differentiation. To demonstrate this, we conducted a dosing optimization study, which is now completed.

同樣，匹托利桑HD治療特發性嗜睡症患者的3期研究也在尋求一個差異化的標籤，適應症為睡眠慣性，我們預計在2025年第四季度啟動該研究。這兩個計畫的目標 PDUFA 日期均為 2028 年。另一種新一代匹托利桑製劑匹托利桑 GR 旨在最大限度地減少與治療相關的胃腸道副作用，尤其是有近 90% 的發作性睡病患者會出現合併胃腸道症狀。此外，匹托利桑 GR 還能夠從 17.8 毫克的治療劑量範圍開始，無需滴定，這是一個重要的區別。為了證明這一點，我們進行了一項劑量優化研究，目前已經完成。

We are excited to share that in this study, patients with narcolepsy started pitolisant GR at 17.8 milligram and 100% of the patients, that is all 46 of 46 patients were able to initiate pitolisant GR at the therapeutic dose of 17.8 milligram with no safety or tolerability issues. In addition, 98% of the patients who received pitolisant GR 35.6 milligram at week two tolerated the higher dose well. No new AEs or SAEs were observed from this study. Pitolisant GR is a fast-to-market strategy designed to demonstrate bioequivalence to WAKIX formulation. The top-line data from the pivotal BE study is on track for Q4 2025 with a target PDUFA in Q1 2027.

我們很高興地宣布，在這項研究中，嗜睡症患者開始服用匹托利桑GR，劑量為17.8毫克，100%的患者（即46名患者中的46名）能夠以17.8毫克的治療劑量開始服用匹托利桑GR，沒有出現任何安全性或耐受性問題。此外，在第二週接受匹托利桑 GR 35.6 毫克治療的患者中，98% 的患者對較高劑量耐受性良好。本研究中未觀察到新的不良事件或嚴重不良事件。Pitolisant GR 是一種快速上市策略，旨在證明其與 WAKIX 製劑具有生物等效性。關鍵性 BE 研究的主要數據預計將於 2025 年第四季公佈，目標是在 2027 年第一季獲得 PDUFA 數據。

Utility patents have been filed for both pitolisant GR and pitolisant HD with potential exclusivity to 2044, securing long-term franchise value.

皮托利桑 GR 和皮托利桑 HD 均已申請實用新型專利，其獨佔權可能持續到 2044 年，從而確保了長期的特許經營價值。

Beyond pitolisant, our Sleep/Wake portfolio continues to advance with BP1.15205, a highly potent orexin-2 receptor agonist demonstrating best-in-class potential in preclinical studies. At the recent SLEEP meeting in Seattle and World Sleep Congress meeting in Singapore, we presented comprehensive preclinical safety and efficacy data that demonstrated efficacy at very low doses across all parameters of interest in a standard transgenic mouse model. We are on track to dose the first subject later this quarter, and we anticipate sharing clinical data in 2026.

除了匹托利桑之外，我們的睡眠/覺醒產品組合也在不斷推進，BP1.15205 是一種高效的食慾素-2 受體激動劑，在臨床前研究中表現出同類最佳的潛力。在最近於西雅圖舉行的 SLEEP 會議和在新加坡舉行的世界睡眠大會上，我們展示了全面的臨床前安全性和有效性數據，證明在標準基因轉殖小鼠模型中，所有感興趣的參數在極低劑量下均有效。我們預計在本季度稍後給第一位受試者給藥，並預計將於 2026 年分享臨床數據。

In the Neurobehavioral franchise, as we have already disclosed, the ZYN002 Phase 3 RECONNECT study in Fragile X syndrome did not meet the primary endpoint of improvement in social avoidance, mainly due to higher-than-expected placebo response. This is disappointing for Harmony and for the Fragile X syndrome community who continue to wait for approved therapies. The in-depth review of full data set is ongoing, and we plan to share additional information in the near future.

正如我們已經披露的那樣，在神經行為學領域，ZYN002 在脆性 X 綜合徵中的 3 期 RECONNECT 研究並未達到改善社交迴避的主要終點，這主要是由於安慰劑反應高於預期。這對 Harmony 公司和脆性 X 綜合徵患者群體來說都是令人失望的，他們仍在等待批准的療法。我們正在對整個數據集進行深入審查，並計劃在不久的將來分享更多資訊。

The ZYN002 program in 22q deletion syndrome has been paused pending the full review of the RECONNECT data. In our Epilepsy franchise, we continue to actively enroll patients in two global Phase 3 registrational trials with EPX-100, the ARGUS study in Dravet syndrome and the LIGHTHOUSE Study in Lennox-Gastaut syndrome. EPX-100, our clemizole hydrochloride is a 5HT2 serotonergic agonist and works we are enhancing serotonergic tone, an established mechanism of action for developmental and epileptic encephalopathies.

針對 22q 缺失症候群的 ZYN002 項目已暫停，等待對 RECONNECT 數據進行全面審查。在我們的癲癇產品線中，我們繼續積極招募患者參與兩項全球 3 期註冊試驗，試驗藥物為 EPX-100，分別是 Dravet 綜合徵的 ARGUS 研究和 Lennox-Gastaut 綜合徵的 LIGHTHOUSE 研究。EPX-100，我們的鹽酸克米唑是一種 5HT2 血清素激動劑，其作用機制是增強血清素張力，這是治療發育性腦病變和癲癇性腦病變的既定作用機制。

In addition, it has a unique safety and tolerability profile, and the emerging safety profile is supportive of no requirements for additional laboratory or special safety monitoring compared to some of the drugs commonly used in these disorders. We will be presenting some of the efficacy data from the ARGUS open-label extension study and the safety tolerability data on EPX-100 at the upcoming American Epilepsy Society Meeting in December.

此外，它具有獨特的安全性和耐受性特徵，其新興的安全性特徵表明，與治療這些疾病的一些常用藥物相比，無需額外的實驗室或特殊安全監測。我們將於 12 月在即將舉行的美國癲癇協會會議上公佈 ARGUS 開放標籤擴展研究的一些療效數據以及 EPX-100 的安全耐受性數據。

Finally, on behalf of Harmony, I would like to thank all the patients and their families who are participating in our clinical trials as well as the clinical investigators and site personnel for their efforts and commitment in helping us to advance our development programs.

最後，我謹代表 Harmony 公司，感謝所有參與我們臨床試驗的患者及其家屬，以及臨床研究人員和現場工作人員，感謝他們為我們推進研發專案所付出的努力和奉獻。

I'll now turn the call over to our CFO, Sandip Kapadia, for an update on our financial performance. Sandip?

現在我將把電話交給我們的財務長桑迪普·卡帕迪亞，請他報告我們的財務表現。桑迪普？

Thank you, Kumar, and good morning, everyone. This morning, we issued our third quarter 2025 earnings release and filed our 10-Q, where you'll find the details of our financial and operating results. We delivered strong financial results in the third quarter with our highest quarter-to-date in revenues and cash generation. Our financial performance and profile positions us well to continue advancing our growth strategy for the remainder of 2025 and beyond.

謝謝你，庫馬爾，大家早安。今天上午，我們發布了 2025 年第三季財報，並提交了 10-Q 表格，您可以在其中找到我們財務和營運業績的詳細資訊。第三季度，我們取得了強勁的財務業績，營收和現金流均創歷史新高。我們的財務表現和公司狀況使我們能夠更好地推進2025年剩餘時間及以後的成長策略。

We reported net revenues of $239.5 million compared to $186 million in the prior year quarter, representing a growth of 29% year over year. The growth was driven by very strong demand for WAKIX as demonstrated by our record increase in average number of patients, along with an increase in trade inventories of a few days as we head into the fourth quarter. We reported total operating expenses for the third quarter of $114.3 million compared to $81.6 million for the same quarter in 2024. The expenses during the third quarter of 2025 included investments to advance our late-stage pipeline, a $15 million milestone for the completion of the enrollment of the ZYN002 trial as well as continued commercialization of WAKIX in narcolepsy.

我們公佈的淨收入為 2.395 億美元，而去年同期為 1.86 億美元，年增 29%。成長主要得益於對 WAKIX 的強勁需求，正如我們平均患者人數創紀錄的增長所證明的那樣，此外，隨著我們進入第四季度，貿易庫存也增加了幾天。我們報告稱，第三季總營運支出為 1.143 億美元，而 2024 年同期為 8,160 萬美元。2025 年第三季的支出包括對推進後期研發管線的投資、為完成 ZYN002 試驗的招募而支付的 1500 萬美元里程碑款項，以及 WAKIX 在嗜睡症治療中的持續商業化。

We also continue to show solid net income growth. Non-GAAP adjusted net income for the third quarter of 2025 was $63.5 million or $1.08 per diluted share compared to $57.3 million or $0.99 per diluted share in the prior year quarter. We believe non-GAAP adjusted net income better reflects the underlying business performance. Please see our press release for a reconciliation of GAAP to non-GAAP results.

我們的淨利潤也持續保持穩健成長。2025 年第三季非 GAAP 調整後淨收入為 6,350 萬美元，即每股攤薄收益 1.08 美元，而去年同期為 5,730 萬美元，即每股攤薄收益 0.99 美元。我們認為，非GAAP調整後的淨利更能反映公司的實際經營績效。請參閱我們的新聞稿，以了解 GAAP 與非 GAAP 結果的調整表。

As previously mentioned, Harmony ended the third quarter with approximately $778 million in cash, cash equivalents, and investments. The balance reflects strong cash generation, resulting in an increase of $106 million in the third quarter. We continue to actively pursue value-enhancing strategic opportunities to deploy our capital to expand our portfolio and drive value for shareholders.

如前所述，Harmony 在第三季末擁有約 7.78 億美元的現金、現金等價物和投資。該餘額反映了強勁的現金流，第三季增加了 1.06 億美元。我們將繼續積極尋求能夠提升價值的策略機遇，運用我們的資本來擴大我們的投資組合，並為股東創造價值。

Looking ahead, in 2025, our strong performance through Q3 gives us increasing confidence in our full-year outlook. We recently raised our revenue guidance from $820 million to $860 million to $845 million to $865 million. These results also gives us confidence that we are rapidly approaching blockbuster status for WAKIX in narcolepsy alone.

展望 2025 年，第三季強勁的業績讓我們對全年前景越來越有信心。我們最近將營收預期從 8.2 億至 8.6 億美元上調至 8.45 億至 8.65 億美元。這些結果也讓我們有信心，WAKIX 僅在治療嗜睡症方面就即將成為重磅藥物。

With respect to expenses, we expect continued investment in R&D as we advance our late-stage pipeline with the start of two Phase 3 studies for our pitolisant HD programs. As a result, we expect to have five ongoing Phase 3 registrational programs by the end of the year. In addition, we also expect a milestone of $4 million in Q4 related to the initiation of our Phase 1 trial in our orexin-2 agonist program.

在支出方面，我們預計隨著我們推進後期產品線，並啟動兩項匹托利桑HD計畫的3期研究，我們將繼續投資研發。因此，我們預計到今年年底將有五個正在進行的第三階段註冊項目。此外，我們也預計在第四季度獲得 400 萬美元的里程碑資金，這與我們食慾素 2 激動劑計畫的 1 期試驗啟動有關。

In summary, we had very strong results for this quarter, along with positive momentum going into Q4. That, along with our strength of our balance sheet, puts us in a solid position to accelerate our growth strategy and drive value for shareholders.

總而言之，我們本季取得了非常強勁的業績，並且帶著積極的勢頭進入了第四季度。這一點，再加上我們穩健的資產負債表，使我們處於有利地位，可以加速我們的成長策略，並為股東創造價值。

And with that, I'll turn the call back over to Jeff for his closing remarks. Jeff?

接下來，我將把電話交還給傑夫，請他作總結發言。傑夫？

Thank you, Sandip, and my thanks to everyone for joining our call today and for your interest in Harmony Biosciences. In closing, I am very proud of our team's exceptional performance in the third quarter and energized by our progress.

謝謝桑迪普，也感謝今天所有參加我們電話會議的朋友們，以及大家對Harmony Biosciences的關注。最後，我對我們團隊在第三季的出色表現感到非常自豪，並為我們所取得的進步感到振奮。

Let me highlight a few key points to leave you with. First, we delivered a very strong quarter with 29% year-on-year revenue growth, driven by a record number of an average of 500 new patient adds for the quarter. With this sustained momentum, we believe WAKIX is rapidly approaching a $1 billion-plus blockbuster status in narcolepsy alone.

最後，我想強調幾個關鍵點。首先，我們本季業績非常強勁，年增 29%，這主要得益於本季平均新增患者人數創下 500 人的紀錄。憑藉這種持續的成長勢頭，我們相信 WAKIX 僅在嗜睡症領域就將迅速成為一款銷售額超過 10 億美元的重磅產品。

Looking ahead, our late-stage pipeline remains robust, and I continue to have strong conviction in our pipeline programs, which are making excellent progress. Lastly, we continue to strengthen our unique profile of being a profitable, self-funding biotech company with an innovative catalyst-rich pipeline poised to deliver meaningful value for patients, providers, and shareholders alike. We believe that this is what makes Harmony one of the most compelling growth stories in biotech today.

展望未來，我們的後期研發管線依然強勁，我對我們的研發管線專案充滿信心，這些專案正在取得優異的進展。最後，我們將繼續鞏固我們作為一家盈利、自籌資金的生物技術公司的獨特地位，我們擁有創新且富含催化劑的產品線，預計將為患者、醫療服務提供者和股東帶來有意義的價值。我們認為，這正是 Harmony 成為當今生物技術領域最引人注目的成長故事之一的原因。

Thank you. And I will now turn the call back over to the operator for Q&A. Operator?

謝謝。現在我將把電話轉回給接線生進行問答環節。操作員？

(Operator Instructions) Ami Fadia, Needham.

（操作說明）Ami Fadia，Needham。

(technical difficulty) on the strong third quarter. My first question is just around kind of your guidance. You've obviously raised your guidance. And even if we look at the year-to-date performance compared to the full year 2024, there's certainly acceleration in the new patient adds this year. So if you could sort of elaborate on how you see the trajectory of WAKIX evolving.

（技術難題）在強勁的第三季。我的第一個問題是關於能否得到您的指導。顯然，您提高了指導標準。即使我們把今年迄今的業績與 2024 年全年業績進行比較，今年新增患者數量也確實出現了加速增長。所以，您能否詳細說明一下您認為 WAKIX 的發展軌跡？

It's certainly several years into the launch, but it appears that there is acceleration here. So if you could comment on how you see that evolving into 2026 and if that can be sustained. And then I have one or two other questions. Thank you.

雖然產品上市已有數年時間，但其發展動能似乎正在加速。所以，您能否談談您認為這種情況到 2026 年會如何發展，以及這種發展能否持續下去？我還有一兩個問題。謝謝。

Okay. Ami, thank you for your question. I'm going to turn it over to Adam to speak about in terms of the trajectory of patient adds and the strong fundamentals there. And then Sandip can comment on kind of our thoughts and position on guidance. Adam?

好的。Ami，謝謝你的提問。接下來我將把發言權交給亞當，讓他談談患者增持的軌跡以及其強勁的基本面。然後桑迪普可以對我們關於指導的意見和立場發表看法。亞當？

Yeah. Thanks, Jeff. Ami, thanks for the question. So yeah, performance is going to be driven fundamentally by patient adds. And as you saw, we're extremely pleased with the quarterly increase in average patients of 500. We haven't seen that high of an increase ever since launch. And it comes on the back of a solid Q2 increase of 400 patients, which we had only seen twice previously.

是的。謝謝你，傑夫。阿米，謝謝你的提問。所以，業績將從根本上取決於患者數量的增加。正如您所看到的，我們對季度平均患者人數增加 500 人感到非常滿意。自推出以來，我們從未見過如此高的成長。而且，第二季度患者人數也穩定成長了 400 人，這種情況我們之前只見過兩次。

And the last time we saw an increase of 400 patients was, I think, early in 2022. So we're very pleased with the underlying performance of the brand and the fundamentals remain strong and that momentum we expect to carry forward into Q4. But Sandip, do you want to share your thoughts on that?

我記得上一次患者人數增加 400 人是在 2022 年初。因此，我們對該品牌的基本面表現非常滿意，其基本面依然強勁，我們預計這種勢頭將延續到第四季。桑迪普，你對此有什麼看法嗎？

Yeah. No, absolutely. As mentioned on the call, I mean, we recently raised our guidance from $820 million to $860 million to $845 million to $865 million. As reflected in the guidance, we saw really good strong demand, as Adam just talked about. We did also see a slight increase in trade inventory during the quarter, which obviously had an impact.

是的。不，絕對的。正如電話會議中所提到的，我們最近將業績預期從 8.2 億美元至 8.6 億美元上調至 8.45 億美元至 8.65 億美元。正如業績指引所反映的那樣，我們看到了非常強勁的需求，正如亞當剛才所說的那樣。本季貿易庫存也略有增加，這顯然產生了一定影響。

As you may recall, in Q2, it was the opposite. We saw a bit of a drawdown. Again, it's typical wholesaler ordering patterns. Thing is it's hard to predict these things as we go into Q4, but we feel very good about the top line demand growth because ultimately, that's what helps drive revenues at the end of the day.

您可能還記得，第二季情況正好相反。我們看到出現了一些下滑。這又是典型的批發商訂貨模式。問題是，進入第四季後，這些事情很難預測，但我們對營收成長非常有信心，因為歸根究底，這才是推動營收成長的根本因素。

And so what we're seeing, we're very optimistic about top-line growth in terms of patient adds as we go into the next quarter. And with regard to revenues, it's really just a question of where we end up with trade inventory sometimes at year-end, it's hard to predict. So again, it's going to be a very strong year, well ahead of our original guidance, again, ahead of our guidance in terms of net patient adds for the year. We had originally guided to about 8,000 patients, and we're well ahead of that as well by year-end.

因此，我們看到，我們對下一季新增患者數量的成長前景非常樂觀。至於收入方面，這實際上只是我們年底貿易庫存最終情況如何的問題，有時很難預測。所以，今年又將是一個非常強勁的年份，遠遠超過我們最初的預期，再次超過我們今年淨新增患者的預期。我們最初預計要招收約 8000 名患者，到年底我們遠遠超過了這個目標。

So really, we're going to see great momentum going into the end of the year on top line demand and certainly going into 2026. And of course, we're rapidly approaching blockbuster status for WAKIX.

所以，我們預計到年底，以及到 2026 年，市場需求將呈現強勁成長動能。當然，WAKIX 也正快速接近票房大片的地位。

Yeah. Yeah, Ami. And I would just add that I think, obviously, we're very pleased with the recent trends, and we're following them in terms of how that will sustain us going into the future. But underlying fundamentals remain very strong. Pleased with, obviously, some of the things Adam has done with the commercial team, and that positions us well going forward.

是的。是的，艾米。我還要補充一點，顯然，我們對近期的發展趨勢非常滿意，我們正在關注這些趨勢將如何幫助我們走向未來。但基本面依然非常強勁。顯然，我們對亞當帶領商業團隊所做的一些事情感到滿意，這讓我們在未來的發展中處於有利地位。

Great. Thank you. And my next question was just for Kumar. With regards to the GR formulation, can you give us some color on what were the GI AEs seen with the GR formulation and how did the grade or frequency of those AEs compared to the in-market WAKIX sort of titration in the initial dosing period?

偉大的。謝謝。我的下一個問題只問庫馬爾。關於 GR 製劑，您能否詳細介紹 GR 製劑在初始給藥階段觀察到的胃腸道不良反應，以及這些不良反應的嚴重程度或發生頻率與市面上 WAKIX 製劑的滴定情況相比如何？

Yeah. Ami, thanks for the question. What we are disclosing right now is no new safety or tolerability issues were observed with the pitolisant GR formulation, no serious AEs were observed. The safety and tolerability was, in general, consistent with the established safety tolerability profile of pitolisant GR. We will be disclosing the full data set in upcoming meetings. Haven't decided when exactly.

是的。Ami，謝謝你的提問。我們現在揭露的是，皮托利桑 GR 製劑沒有出現新的安全性或耐受性問題，也沒有嚴重不良事件。總體而言，安全性和耐受性與匹托利桑 GR 已建立的安全性和耐受性特徵一致。我們將在即將召開的會議中公佈全部資料集。還沒決定具體時間。

But what's important from the pitolisant GR formulation, Ami, is we initiated 46 patients with narcolepsy with pitolisant GR at 17.8 milligrams. And all of those patients were successfully able to tolerate 17.8 milligrams and about 98% of the patients who went to get 35.6 milligram after one week of pitolisant GR, 98% of them were able to tolerate pitolisant GR 35.6 milligram.

但匹托利桑 GR 製劑 Ami 的重要意義在於，我們用 17.8 毫克匹托利桑 GR 對 46 名嗜睡症患者進行了治療。所有這些患者都能成功耐受 17.8 毫克，服用匹托利桑 GR 一週後，約 98% 的患者能夠耐受 35.6 毫克匹托利桑 GR。

And this is an important differentiation because if you look at the medicines that are approved for patients with narcolepsy, every one of them, including pitolisant has some level of titration and getting rid of the titration will have the patients to start at the therapeutic dose range, potentially can experience efficacy earlier, potentially less number of dropouts and potentially better overall patient experience.

這是一個重要的區別，因為如果你看一下獲準用於治療嗜睡症的藥物，每一種藥物，包括匹托利桑，都需要一定程度的劑量滴定，而取消劑量滴定將使患者能夠從治療劑量範圍開始，有可能更快地體驗到療效，有可能減少脫落人數，並有可能改善患者的整體體驗。

Got it. Thank you. If I could just squeeze in one more other quick question. If you could just elaborate on some of the details of what we should expect from the ARGUS open-label extension data at AES in December? Thanks.

知道了。謝謝。如果可以的話，我再問一個問題。您能否詳細說明我們應該從 12 月 AES 會議上公佈的 ARGUS 開放標籤擴展數據中獲得哪些資訊？謝謝。

Yeah. Thank you, Ami. Yeah, we do -- we will be presenting some efficacy data on EPX-100 from the ARGUS open-label extension study. And we'll also be presenting safety and tolerability data from EPX-100 from the same study, ARGUS double-blind randomized study and also the open-label extension study. You'll see the overall efficacy, the safety, tolerability offers an overall very unique benefit risk profile for patients with developmental and epileptic encephalopathies. In this particular instance, particularly the data that we'll be sharing is in patients with Dravet syndrome.

是的。謝謝你，阿米。是的，我們會公佈一些來自 ARGUS 開放標籤擴展研究的 EPX-100 療效數據。我們還將展示來自同一研究（ARGUS 雙盲隨機研究）以及開放標籤擴展研究的 EPX-100 安全性和耐受性數據。你會發現，其整體療效、安全性、耐受性為發育性和癲癇性腦病變患者提供了非常獨特的整體獲益風險概況。在這個特殊的例子中，我們將要分享的數據是關於德拉韋綜合徵患者的。

Thank you. Thanks.

謝謝。謝謝。

David Amsellem, Piper Sandler.

大衛·阿姆塞勒姆，派珀·桑德勒。

Just a couple for me. First, I wanted to get your latest thoughts on biz dev and M&A, particularly in light of the failure of Zygel. Are you thinking more expansively regarding acquisitions? Are you open to more of a sizable transaction, perhaps a market-ready or commercial stage asset? Just trying to get a sense philosophically for where your heads are at. That's number one.

就我一個人用。首先，我想了解您對業務拓展和併購的最新看法，尤其是在 Zygel 失敗之後。您是否正在考慮更廣泛地進行收購？您是否願意接受規模較大的交易，例如已具備市場上市條件或已進入商業化階段的資產？我只是想從哲學層面來了解你們的想法。這是第一點。

And then number two, looking a bit longer term regarding WAKIX with the introduction of the first orexin agonist coming potentially before the end of '26, and I know it's just NT1, how are you thinking about the trajectory of WAKIX beyond '26, specifically in '27 with oveporexton potentially in the market? Thanks.

其次，從長遠來看，考慮到第一個食慾素激動劑可能在 2026 年底之前推出（我知道它只是 NT1），您如何看待 WAKIX 在 2026 年之後的發展軌跡，特別是 2027 年，因為 ovoperexton 有可能上市？謝謝。

Yeah. David, thanks for your questions. So first, with regards to biz dev and our sort of thinking there. I mean, it really hasn't changed. We have always been focused on business development, being strategic, thoughtful in how we deploy our capital as we have built out our pipeline, obviously, the Zynerba acquisition and then Epygenix

是的。大衛，謝謝你的提問。首先，關於業務拓展以及我們在這方面的想法。我的意思是，情況真的沒有改變。我們始終專注於業務發展，在建立產品線的過程中，我們採取策略性、深思熟慮的方式來部署資本，例如收購 Zynerba 和 Epygenix。

I think at this point, despite the Fragile X data readout, it doesn't really change our strategy. We have a dedicated BD team focused on sort of search and evaluation. And at this point, it really is our intent with our strong balance sheet to pursue innovative assets to build out our pipeline to grow our product portfolio.

我認為，儘管公佈了脆性X染色體症候群的數據，但目前來看，這並不會真正改變我們的策略。我們擁有一支專門的業務拓展團隊，專注於此類搜尋和評估工作。目前，憑藉我們強勁的資產負債表，我們的真正目標是尋求創新資產，以擴大我們的產品線，擴大我們的產品組合。

We are actively evaluating several, and that is our plan, but the strategy remains the same. Our focus in orphan rare CNS disorders to be strategic within the current franchises, but also looking at adjacencies potentially in broader neuro indications where we could utilize the proven commercial engine. So the strategy remains the same. I think a bit more focused with regards to our intent to move forward with business development when we find the right opportunities for us.

我們正在積極評估幾種方案，這是我們的計劃，但策略保持不變。我們專注於罕見的孤兒中樞神經系統疾病，在現有產品線內制定策略，同時也著眼於更廣泛的神經適應症領域，利用我們成熟的商業模式。所以策略保持不變。我認為，在找到合適的機會時，我們在推動業務發展方面的意圖會更加明確。

And I'll turn to Adam in terms of thoughts on WAKIX performance in the setting of emergent orexins. Adam?

接下來，我想聽聽 Adam 對 WAKIX 在新興食慾素環境下的表現有何看法。亞當？

Yeah. Thanks, Jeff, and thanks for the question, David. Look, we're excited about orexins, obviously, because we have one of our own, but it's also an important potential new treatment option for patients. And we remain confident in our ability to grow and perform with WAKIX well into the future. And there's a couple of reasons for that.

是的。謝謝傑夫，也謝謝大衛的提問。當然，我們對食慾素感到興奮，因為我們自己就有一種食慾素，而且它對患者來說也是一個重要的潛在新治療選擇。我們仍然對未來與 WAKIX 共同成長並取得良好業績充滿信心。這其中有兩個原因。

The first is just the approach to therapy in this market. The hallmark of treatment is polypharmacy. You have a high majority of patients that are on two or more therapies, and that will continue. We've recently verified that in market research that we've done speaking with physicians, and they expect that to continue as well.

首先，我們需要了解這個市場上的治療方法。治療的特點是多種藥物合併使用。絕大多數患者都在接受兩種或兩種以上的治療，這種情況還會持續下去。我們最近透過與醫生的交流進行了市場調查，證實了這一點，他們也希望這種情況能夠繼續下去。

But also, if you just look at the history of WAKIX, WAKIX performance has been extremely steady regardless of new entrants, whether it's brands or generics. And we would expect that to continue as well. In fact, if you look at kind of the narcolepsy market in total, any time there's been a new brand entrant, it tends to expand brand utilization. And we would expect a similar phenomenon with an orexin launch when those come to market. But we're also highly confident just because WAKIX is a highly differentiated product. It's the only non-scheduled treatment option.

但是，如果你看看 WAKIX 的歷史，無論新進業者是品牌藥還是仿製藥，WAKIX 的表現都極為穩定。我們預計這種情況還會持續下去。事實上，如果你縱觀整個嗜睡症市場，每當有新品牌進入市場時，往往會擴大品牌的使用率。我們預計，當食慾素類藥物上市時，也會出現類似的現象。但我們也非常有信心，因為 WAKIX 是一款高度差異化的產品。這是唯一一種非預約治療方案。

Physicians will have had 7, 8-plus years of clinical experience by the time of orexins launch, and it holds a unique position in the minds of health care providers. They're highly familiar with it, provides strong efficacy and is -- and they believe it's very well tolerated. So it can be added to combinations of therapies across a very broad selection of narcolepsy patients, and that will continue as well. We continue to hold that position. So for those reasons, I think we're very confident in our continued growth and performance well into the future.

到食慾素上市時，醫生將擁有 7 年、8 年甚至更長的臨床經驗，因此它在醫療保健提供者心中佔據著獨特的地位。他們非常熟悉這種藥物，它療效顯著，而且——他們認為它的耐受性非常好。因此，它可以添加到多種療法中，用於治療非常廣泛的嗜睡症患者，而且這種情況還會繼續下去。我們依然堅持這一立場。因此，基於以上原因，我認為我們對未來持續成長和良好業績充滿信心。

Okay. Thank you.

好的。謝謝。

Graig Suvannavejh, Mizuho.

Graig Suvannavejh，瑞穗。

Congrats on the progress. One question for me, just going back to the 2025 guidance. It was nice to see a raising of the guidance. But if you do the math, it does imply fourth quarter sales for WAKIX in the range of, I think, $221 million to $241 million. And on a quarter-over-quarter basis, that's essentially flat or down on a quarter-over-quarter basis.

恭喜你取得進展。我還有一個問題，是關於2025年的指導。很高興看到指導方針有所提高。但如果你計算一下，這確實意味著 WAKIX 第四季的銷售額在 2.21 億美元到 2.41 億美元之間。從季度環比來看，這一數字基本上持平或下降。

So can you just provide more color on how we should be thinking about whether it's net patient adds or gross to net or seasonality when trying to model fourth quarter? I know you -- Sandip had mentioned some inventory could be a factor, but any other color would be great on the fourth quarter, especially given the great momentum on net patient adds.

那麼，您能否更詳細地說明一下，在嘗試對第四季度進行建模時，我們應該如何考慮新增患者數量、毛利淨額還是季節性因素？我知道你——桑迪普曾提到庫存可能是一個因素，但第四季度任何其他顏色都會很好，尤其是在淨新增患者數量強勁增長的情況下。

Yeah. Thanks, Graig, for the question. Again, I think we saw great demand in terms of top line growth that we expect to continue as we go from Q3 to Q4 as well. I think we did -- as I mentioned, we did see a few days of trade inventory increase in Q3, which impacted the sales positively. Just as I mentioned in Q2, it's quite the other way -- the other direction.

是的。謝謝格雷格的提問。我再次認為，我們在營收成長方面看到了強勁的需求，我們預計這種需求將在第三季到第四季繼續保持。我認為我們做到了——正如我所提到的，我們在第三季度確實看到了幾天的貿易庫存增加，這對銷售產生了積極影響。正如我在第二季度提到的那樣，情況恰恰相反——完全是另一個方向。

Right now, I mean, we feel very good about the range that we've put out there. We recently raised it, as you mentioned. This gives us great confidence and I'm sure like every team, I mean, we'll do everything we can to certainly not only meet but potentially exceed the range as well.

就目前而言，我們對我們推出的產品系列感到非常滿意。正如您所提到的，我們最近也提出了這個問題。這給了我們極大的信心，我相信像所有球隊一樣，我們會竭盡全力，不僅要達到目標，還要有可能超越目標。

Again, it's hard to predict again Q4 because there are typical variabilities as you come to Q4, especially around brand and holiday and so forth. So we feel good about where it is. It's very robust growth year-over-year. It's going to be a robust growth over prior year. And we see, again, WAKIX very quickly approaching blockbuster status as we go into '26 and beyond.

同樣，第四季很難預測，因為到了第四季度，各種因素都會出現，尤其是在品牌和假日等方面。所以我們對它目前的位置感到滿意。這是非常強勁的同比增長。與前一年相比，今年將實現強勁成長。我們再次看到，隨著我們進入 2026 年及以後，WAKIX 很快就會成為一部轟動一時的大片。

Great. If I could ask a follow-up. Just your net cash position is building quite nicely. I'm sure there are various views on how to deploy that cash. Wondering if maybe in an answer that might be slightly different from David's question on BD, what are your kind of current thoughts on how best to deploy that nicely accruing cash balance?

偉大的。如果可以的話，我想問一個後續問題。你的淨現金部位成長得相當不錯。我相信對於如何使用這筆資金，大家會有各種各樣的看法。我想知道，也許我可以給出一個與 David 在 BD 上提出的問題略有不同的答案：您目前對於如何最好地利用這筆不斷增長的現金餘額有什麼想法？

Sandip?

桑迪普？

Yeah, sure. I mean, look, as Jeff mentioned, I mean, we continue to look for business development opportunities. I think that's been a strategy for us for many years now. And we see attractive opportunities out there that we could potentially transact. And we have, again, a continued growth in terms of cash. We had very strong cash generation last quarter of $106 million, $778 million at the end of the quarter. And I think that gives us increasing confidence that we -- but again, we're going to be, I would say, thoughtful in terms of how we deploy our capital.

當然可以。我的意思是，就像傑夫提到的那樣，我們會繼續尋找業務發展機會。我認為這多年來一直是我們的策略。我們看到了一些有吸引力的潛在交易機會。而且，我們的現金流也持續成長。上個季度我們的現金流非常強勁，達到 1.06 億美元，季末達到 7.78 億美元。我認為這讓我們越來越有信心——但是，我再次強調，我們會謹慎地運用我們的資本。

I think we certainly have multiple ways in which to drive value for shareholders. Certainly, more recently, we've been prioritizing business development. But in the past, we've also done share buyback as well as an opportunity. And again, at the right time, we'll look at various opportunities to drive value for shareholders.

我認為我們肯定有多種方式可以為股東創造價值。當然，最近我們更加重視業務拓展。但過去我們也曾進行過股票回購，這也是一個不錯的機會。而且，在適當的時機，我們會考慮各種機會為股東創造價值。

Yeah. Thank you.

是的。謝謝。

Graig, I'll just go ahead. No, no, I appreciate the question. I just want to reiterate, I think while one can never predict the timing in terms of business development transactions, as Sandip alluded to, we have optionality, but our focus and our intent is really to pursue innovative value-enhancing assets. We want to build our pipeline. We see that's where the value is going forward as well as build a broader product portfolio in terms of -- we have a strong commercial engine. We want to continue to utilize that with additional products. So that is our intent, and we have a strong balance sheet to execute on that.

格雷格，我先走了。不，不，我很感謝你的提問。我只想重申一下，雖然人們永遠無法預測業務發展交易的時機，正如桑迪普所暗示的那樣，我們有選擇權，但我們的重點和意圖確實是追求創新的價值提升資產。我們希望建立自己的人才儲備庫。我們認為，未來價值就在於此，同時也要建立更廣泛的產品組合——我們擁有強大的商業引擎。我們希望繼續將這種理念應用於其他產品。這就是我們的目標，而且我們擁有強勁的資產負債表來執行這一目標。

Thank you.

謝謝。

Jay Olson, Oppenheimer.

傑伊·奧爾森，奧本海默。

Congrats on the quarter. Can you talk about your life cycle management plan for pitolisant GR and HD with regards to new patients? And then which patients currently on WAKIX are the best candidates to benefit from GR and HD?

恭喜你本季取得佳績。您能否談談針對新病患的匹托利桑GR和HD的生命週期管理計畫？那麼，目前接受 WAKIX 治療的患者中，哪些人最適合接受 GR 和 HD 治療呢？

And then for your orexin-2 program, what would you like to learn from your Phase 1 study? And any lessons learned from the Alkermes and Takeda data at World Sleep? Thank you.

那麼，對於您的食慾素-2項目，您希望從1期研究中了解到什麼？從世界睡眠大會上Alkermes和Takeda的數據中，我們能學到哪些教訓？謝謝。

Yeah. Jay, thanks for your questions. Adam, our life cycle management strategy with regards to patients that would benefit from the formulation.

是的。傑伊，謝謝你的提問。Adam，我們針對那些將從該配方中受益的患者制定了生命週期管理策略。

Yeah, we're really excited about the two life cycle management formulations, the GR and the HD. GR is kind of a fast-to-market strategy. And the strategy there would be any patient -- any new patients that would have been prescribed WAKIX would be prescribed the new pitolisant GR formulation as well as we have the ability because we obtain consent from patients when they start WAKIX therapy. We would also be able to recontact patients that may have been on WAKIX previously but have discontinued to see if pitolisant GR could be an option for them to consider as well. So for GR, it's really around new WAKIX patients and previous patients.

是的，我們對兩種生命週期管理配方——GR 和 HD——感到非常興奮。GR 是一種快速上市的策略。而該策略是，任何新患者，只要被處方了 WAKIX，都會被處方新的匹托利桑 GR 製劑，因為我們在患者開始 WAKIX 治療時會徵得他們的同意，所以我們有能力這樣做。我們也可以重新聯繫之前可能服用過 WAKIX 但後來停藥的患者，看看匹托利桑 GR 是否也是他們可以考慮的選擇。所以對 GR 來說，主要對象就是 WAKIX 的新病人和之前的病人。

And then where the HD comes in with a greatly differentiated profile, we would see the strategy there focusing on not only new WAKIX patients and previous patients, but also existing WAKIX patients. And that's where the transition potential comes in. And we've conducted market research around this. HCPs respond very favorably to the profile of HD. They view it as clinically significantly differentiated, actually, superior.

而 HD 則具有非常不同的特徵，我們會看到其策略不僅關注新的 WAKIX 患者和先前的患者，還關注現有的 WAKIX 患者。這就是轉型潛力所在。我們已經就此進行了市場調查。醫療保健專業人員對HD的概況反應非常正面。他們認為它在臨床上具有顯著的差異化優勢，實際上，甚至更勝一籌。

And in market research, they tell us they would consider transitioning the majority of their patients that maybe are better but not well. And so that's the strategy for HD. And then both formulations have utility patents filed through 2044. So it really allows us to expand and extend our Sleep/Wake franchise well into the future.

市場調查顯示，他們會考慮將大部分病情可能有所改善但尚未康復的患者轉為其他治療方案。這就是高清的策略。而這兩種配方都已申請實用新型專利，有效期限至 2044 年。因此，這確實使我們能夠將睡眠/覺醒產品線擴展到未來很長一段時間。

In terms of our (technical difficulty) agonist program and learnings from some of the other development programs.

就我們的（技術難度）激動劑計劃以及從其他一些開發計劃中學到的經驗教訓而言。

Sure. Thank you, Jeff. Jay, thank you for the question. Yes, we are on track to initiate the Phase 1 study with our orexin-2 receptor agonist this quarter. As we have disclosed in the past, we will be starting a healthy volunteer single ascending dose study.

當然。謝謝你，傑夫。傑伊，謝謝你的提問。是的，我們正按計畫推進，將於本季啟動食慾素-2受體激動劑的1期臨床試驗。正如我們先前所揭露的，我們將啟動一項針對健康志願者的單次遞增劑量研究。

And in parallel, we'll be conducting a sleep-deprived healthy volunteer study as well to bracket the dose a bit. And we are closely watching the data that came out of World Sleep Congress from other orexin receptor agonists, especially as it relates to dosing and the safety and tolerability profile alongside the efficacy.

同時，我們也將進行一項睡眠不足的健康志願者研究，以便對劑量進行一定範圍的估計。我們正在密切關注世界睡眠大會上公佈的其他食慾素受體激動劑的數據，特別是與劑量、安全性和耐受性以及療效相關的數據。

We have one of the most potent orexin receptor agonists based on all the publicly available data, and it lends itself to target the central (technical difficulty) and idiopathic hypersomnia at very low dose, providing us the dosing flexibility to target these three indications. And we are also trying to see how we can accelerate our own program based on the data that is coming out from the other orexin receptor agonists. And you'll be hearing more from our own orexin receptor agonist when we initiate the first-in-human study. And we also anticipate to share some of the clinical data in 2026. Thank you, Jay.

根據所有公開數據，我們擁有最有效的食慾素受體激動劑之一，它能夠以非常低的劑量治療中樞性（技術難度較高）和特發性嗜睡症，從而為我們提供了針對這三種適應症的劑量靈活性。我們也在嘗試根據其他食慾素受體激動劑的數據，來加速我們自己的計畫。當我們啟動首次人體試驗時，您將會聽到更多關於我們自己的食慾素受體激動劑的消息。我們也預計將在 2026 年分享一些臨床數據。謝謝你，傑伊。

Super helpful. Thanks again. Congrats on the quarter.

非常有用。再次感謝。恭喜你本季取得佳績。

Pete Stavropoulos, Cantor Fitzgerald.

皮特·斯塔夫羅普洛斯，康托·菲茨傑拉德。

Congratulations on the progress. Could you just touch on EPX-100 epilepsy program? Any clinical data that has been generated and disclosed that sort of gives you confidence in the Dravet and LGS program? Any details on efficacy, durability and safety? And can you also give us a sense of how enrollment is going in the Phase 3 studies? Any granularity on timing of data or when you expect to complete enrollment? Thanks.

恭喜取得進展。能簡單介紹一下EPX-100癲癇治療方案嗎？是否有任何已產生和揭露的臨床數據，能夠讓您對 Dravet 和 LGS 計畫充滿信心？關於功效、持久性和安全性方面有什麼細節嗎？您能否也向我們介紹一下第三階段研究的招募？能否提供資料提交的具體時間安排，或預計何時完成註冊？謝謝。

Pete, thanks for your questions. I'll turn to Kumar for some more color on the EPX program.

皮特，謝謝你的提問。我將請庫馬爾詳細介紹一下EPX專案。

Yeah. Pete, thank you for the question. Yes, I mean, we are excited to share some of the efficacy data at the upcoming American Epilepsy Society meeting in December. As I mentioned earlier in the call, we will be sharing the data from the ARGUS open-label extension study, that's the study in Dravet syndrome. In terms of your question around recruitment and enrollment, yes, I mean, we continue to recruit patients in both ARGUS study and the LIGHTHOUSE study, that's Dravet and LGS studies, and we anticipate to share top-line data in 2026. We'll be providing more granularity in terms of time lines as to the progress with the enrollment. Thank you.

是的。皮特，謝謝你的提問。是的，我們非常期待在 12 月即將舉行的美國癲癇協會會議上分享一些療效數據。正如我之前在電話會議中提到的，我們將分享 ARGUS 開放標籤擴展研究的數據，這是一項針對 Dravet 症候群的研究。關於您提出的招募和入組問題，是的，我的意思是，我們仍在繼續招募 ARGUS 研究和 LIGHTHOUSE 研究的患者，這兩項研究分別是 Dravet 和 LGS 研究，我們預計將在 2026 年分享初步數據。我們將提供更詳細的招生進度時間表。謝謝。

And any thoughts on taking EPX-100 into other DEEs or other DEEs remain the focus of EPX-200?

那麼，對於將 EPX-100 應用於其他 DEE，還是說其他 DEE 仍然是 EPX-200 的重點？

Yeah, Pete, great question. Thank you. Look, there is this discussion around DS, LGS pursuing separately versus going with a broader DEE indication, right? So there are pros and cons with each of these approaches. But right now, we are focused -- laser-focused on Dravet syndrome and Lennox-Gastaut syndrome.

是的，皮特，問得好。謝謝。你看，現在關於DS和LGS的討論是單獨進行還是採用更廣泛的DEE適應症，對吧？所以每種方法都有其優點和缺點。但現在，我們正全力以赴地研究 Dravet 症候群和 Lennox-Gastaut 症候群。

We want to keep it that way, mainly to maintain the homogeneity of the patient population and try to get to the top line data as soon as possible and try and help these patients with a product profile, which is very favorable, not just from an efficacy perspective, but also from a safety and tolerability perspective.

我們希望維持現狀，主要是為了保持患者群體的同質性，盡快獲得初步數據，並嘗試為這些患者提供一種非常有利的產品特性，這種特性不僅在療效方面，而且在安全性和耐受性方面也十分有利。

Yeah. And Pete, maybe one addition just to remind everyone. So EPX-100, clemizole hydrochloride. So a first-generation antihistamine that was in the market for about 20 years with a proven safety tolerability profile. And in the overall sort of risk benefit in these sort of therapeutic options with other indicated agents such as Epidiolex and the need to monitor LFTs or FINTEPLA with echocardiograms required in the REMS program because of cardiac valvular disease, the risk there.

是的。還有皮特，或許應該補充一點，提醒大家。所以 EPX-100，鹽酸氯米唑。因此，這是一種第一代抗組織胺藥，已在市場上銷售約 20 年，並具有良好的安全性和耐受性。整體而言，這類治療方案與其他適應症藥物（如 Epidiolex）相比，需要監測肝功能或 FINTEPLA，由於心臟瓣膜疾病，REMS 計劃要求進行超音波心動圖檢查，這存在一定的風險。

So there is sort of a proven safety tolerability profile. And in the overall kind of risk benefit, we see the opportunity there as we go forward and generate efficacy data in both Dravet and LGS.

因此，它具有一定的安全性和耐受性。從整體風險效益的角度來看，隨著我們在 Dravet 和 LGS 方面取得進展並產生療效數據，我們看到了其中的機會。

All right. Thank you very much, and congratulations once again.

好的。非常感謝，再次恭喜。

Danielle Brill, Truist Securities.

Danielle Brill，Truist Securities。

Congrats on the quarter. Maybe just two quick ones from me. Do you have any sense of what proportion of your 8,100 patient base is NT1 versus NT2? And then what findings, if any, in the Fragile X data set would make you consider reactivating the q22 trial? Thank you.

恭喜你本季取得佳績。我可能只是簡單提兩句。您知道您的 8100 名患者中，NT1 型和 NT2 型患者分別佔多少比例嗎？那麼，脆性X染色體症候群資料集中的哪些發現（如果有的話）會讓您考慮重新啟動q22試驗？謝謝。

Danielle, thanks for your questions. Adam, for breakdown of patients on WAKIX.

丹妮爾，謝謝你的提問。Adam，負責 WAKIX 上的病患分類。

Sure. Thanks for the question. It's been very consistent actually for the last several years. We see about 45% of patients from NT1, 55% from NT2, and that's been very stable.

當然。謝謝你的提問。事實上，過去幾年一直非常穩定。我們看到大約 45% 的患者來自 NT1 型，55% 的患者來自 NT2 型，這個比例一直非常穩定。

Okay. Kumar?

好的。庫馬爾？

Yeah. Danielle, thank you for the question. We are right now conducting an in-depth review of all the data from the RECONNECT study. As mentioned previously, the Fragile X syndrome study did not read out as expected, mainly because of the larger-than-anticipated placebo response. Right now, we are conducting a number of post-hoc analysis. It's very hard to say at this point in time what kind of data sets we need to see before we embark upon 22q deletion syndrome Phase 3 study

是的。丹妮爾，謝謝你的提問。我們目前正在對 RECONNECT 研究的所有數據進行深入審查。如前所述，脆性X症候群研究的結果並未如預期那樣公佈，主要是因為安慰劑效應比預期的要大。目前，我們正在進行一系列事後分析。目前很難說在啟動 22q 缺失症候群 III 期研究之前，我們需要看到什麼樣的資料集。

nce all these data sets are available, we will make a decision based on the data that we see. And we should be able to complete this work by the end of this year, and we should be able to provide some update on the Fragile X syndrome study itself and also the implications on 22q deletion syndrome early next year.

一旦所有這些數據集都可用，我們將根據我們看到的數據做出決定。我們應該能夠在今年年底前完成這項工作，並且應該能夠在明年初提供有關脆性 X 綜合徵研究本身以及對 22q 缺失綜合徵的影響的一些最新進展。

Corinne Johnson, Goldman Sachs.

科琳·約翰遜，高盛集團。

This is [Anupam] on behalf of Corinne. Maybe one question for Kumar. Can you talk about the effect size you are powering for the Phase 3 pitolisant HD study to show on ESS scale? And what do you think a clinically meaningful difference would be in comparison to the current WAKIX in order to support the HD use? I think the WAKIX shown around 12- to 13-point final ESS score. Any color on that?

我是阿努帕姆，代表科琳。也許我想問庫馬爾一個問題。您能否談談您在 ESS 量表上顯示的 3 期匹托利桑 HD 研究的效應量？那麼，為了支援高清應用，您認為與目前的 WAKIX 相比，臨床上有意義的差異應該是什麼？我認為 WAKIX 的最終 ESS 得分在 12 到 13 分左右。上面有顏色嗎？

Thank you for the call -- thank you for the question. Look, I mean, pitolisant HD, it's an enhanced formulation -- it's an enhanced next-gen formulation. It's not just the high-dose, but it's also -- it also has an optimized PK profile. Based on the dose response that we have seen with pitolisant in the pivotal narcolepsy clinical trials and also the studies that we conducted in Prader-Willi syndrome and also in myotonic dystrophy and the dose -- and the exposure response data, some of the exposure response data that we have, we anticipate a meaningful increase in the efficacy from an excessive data and sleepiness perspective.

謝謝你的來電－謝謝你的提問。我的意思是，皮托利桑HD是一種增強型配方——它是增強型的下一代配方。不僅是劑量高，而且——它還具有優化的藥物動力學特性。根據我們在關鍵性嗜睡症臨床試驗中觀察到的匹托利桑的劑量反應，以及我們在普拉德-威利症候群和強直性肌肉營養不良症中進行的研究，以及劑量和暴露反應數據，我們預計從過度嗜睡的角度來看，療效將有顯著提高。

But it's also important to note that we are not just targeting excessive data and sleepiness. We are also targeting fatigue in patients with narcolepsy for which there are no approved treatments. And we are also targeting sleep inertia in patients with idiopathic hypersomnia for which there are no approved treatments. And we plan to accomplish all of this without compromising on the safety or tolerability profile of pitolisant. In fact, we conducted a Phase 1B study where we studied pitolisant up to 180 milligrams, which is up to 5 times the maximum label dose of WAKIX. And the safety and tolerability profile in general was similar to what we see with WAKIX.

但同樣重要的是要注意，我們不僅僅是針對資料過載和困倦問題。我們也致力於改善發作性睡病患者的疲勞症狀，目前尚無核准的治療方法。我們也致力於治療特發性嗜睡症患者的睡眠慣性，目前尚無核准的治療方法。我們計劃在不影響匹托利桑安全性和耐受性的前提下實現所有這些目標。事實上，我們進行了 1B 期研究，研究了匹托利桑高達 180 毫克的劑量，相當於 WAKIX 最大標籤劑量的 5 倍。總體而言，其安全性和耐受性與 WAKIX 類似。

So what it means at the end of the day is a very unique benefit risk analysis with established safety profile, established tolerability profile, larger efficacy in excessive daytime sleepiness, targeting symptoms like fatigue in narcolepsy, targeting symptoms like sleep inertia in patients with idiopathic hypersomnia, all while maintaining the non-scheduled status and a very simple dosing regimen of taking pill in the morning. So that's what we plan to accomplish with pitolisant HD. Thank you.

因此，歸根結底，這意味著一項非常獨特的獲益風險分析，具有已確立的安全特性、已確立的耐受性特性，對白天過度嗜睡有更大的療效，針對發作性睡病中的疲勞等症狀，針對特發性嗜睡症患者的睡眠慣性等症狀，同時保持非定時服用狀態和非常簡單的早上服藥方案。所以這就是我們計劃透過皮托利桑HD實現的目標。謝謝。

Okay. Thank you.

好的。謝謝。

Patrick Trucchio, H.C. Wainwright.

派崔克·特魯基奧，H.C. 溫賴特。

I was wondering if you could elaborate a bit more on the sustainability and as well the drivers of the record approximate 500 patient add in the third quarter, and if we should expect those drivers to continue in the fourth quarter, but as well in 2026?

我想請您詳細說明可持續性，以及第三季度新增約 500 名患者這一創紀錄數字背後的驅動因素，我們是否應該預期這些驅動因素會在第四季度以及 2026 年繼續保持？

And then separately, on pitolisant HD, I think you mentioned strong early feedback from physicians and payers. I'm wondering if you could elaborate on what's resonating most and as well the implications for the Phase 3 development program.

另外，關於匹托利桑HD，我想你提到過醫生和支付方的強烈早期回饋。我想請您詳細說明最能引起共鳴的是什麼，以及這對第三階段開發計畫的影響。

Patrick, thank you for your questions. Adam?

派崔克，謝謝你的提問。亞當？

Yeah. Thank you for the question. So we're really pleased with the momentum we're seeing in increases in patient adds, as you mentioned. And the drivers, look, I think it starts with WAKIX is a highly differentiated product. It's the only nonscheduled treatment option. And that is combined with strong execution across the organization.

是的。謝謝你的提問。正如您所提到的，我們對患者新增數量的成長動能感到非常滿意。至於驅動程序，我認為首先是 WAKIX 是一款高度差異化的產品。這是唯一一種非計劃內的治療方案。而這一切都離不開整個組織強而有力的執行力。

So if you think about sales effectiveness and execution, marketing and promotional excellence, payer coverage, patient support, we've made adjustments in all of these areas. And we're seeing those adjustments delivering the performance that you're seeing and have confidence that, that momentum will definitely carry forward in Q4 and into 2026.

因此，如果您考慮銷售效率和執行力、行銷和推廣方面的卓越性、支付方覆蓋範圍、患者支持，我們在所有這些領域都做出了調整。我們看到這些調整帶來了你們所看到的業績，並且有信心，這種勢頭肯定會在第四季度以及2026年繼續保持下去。

Okay. And Patrick, your second question?

好的。派崔克，你的第二個問題是什麼？

Yeah. So around the HD. So what resonates, what resonates is the promise of improved efficacy, the no titration starting at a therapeutic dose and unique indications, especially around IH and fatigue. We know that 60% of narcolepsy patients present with fatigue. This would be the only product that has the indication for fatigue in narcolepsy. And they view that as highly differentiated.

是的。所以，大概在高清附近。因此，真正引起共鳴的是提高療效的承諾、無需滴定即可從治療劑量開始以及獨特的適應症，尤其是在特發性出血和疲勞方面。我們知道，60%的嗜睡症患者會出現疲勞症狀。這將是唯一獲準用於治療嗜睡症引起的疲勞的產品。他們認為這之間存在高度差異。

So I mentioned the feedback from HCPs, but we've also done research with payers. And the response is that we would expect broad payer coverage for the HD product with minimal step edits for WAKIX or no step edits actually prior to LOE. And even after LOE, only some mentions of perhaps some step edits, but only for patients that have never had any experience on WAKIX before, which at this point, I guess we'll be at, what, eight-plus years in the market by then. The vast majority of patients will have had some experience with WAKIX at some point in the past.

所以我提到了來自醫療保健專業人員的回饋，但我們也對支付方進行了調查。而得到的答覆是，我們預期 HD 產品將獲得廣泛的支付方覆蓋，WAKIX 的階梯式修改將盡可能少，或者在 LOE 之前實際上不會進行任何階梯式修改。即使在 LOE 之後，也只是提到可能會進行一些步驟修改，但僅限於以前從未有過 WAKIX 使用經驗的患者，而到那時，我猜我們已經在市場上銷售了八年多。絕大多數患者在過去某個時候都曾接觸過 WAKIX。

And then, of course, any patient that presents with fatigue would also not be subject to any step edits through WAKIX even post LOE. So we would expect broad payer coverage pre and post LOE supporting the product and its uptake.

當然，任何出現疲勞症狀的患者也不會在 LOE 之後透過 WAKIX 進行任何步驟編輯。因此，我們預計在 LOE 前後，支付方將廣泛涵蓋該產品及其推廣應用。

Thanks, guys.

謝謝各位。

Jason Gerberry, Bank of America.

傑森‧格伯里，美國銀行。

This is [Bavan] on for Jason. Just two questions from us. The first is on EPX-100. You have two Phase 3 readouts expected in 2026. So maybe if you can just speak to what you've learned from the Fragile X syndrome as well as the prior idiopathic hypersomnia study about managing placebo response in these types of neurodevelopmental studies?

這是[Bavan]替補Jason上場。我們只有兩個問題。第一個是在 EPX-100 上。預計2026年將有兩項3期臨床試驗結果發表。那麼，您能否談談您從脆性X症候群以及先前的特發性嗜睡症研究中學到的關於如何在這類神經發育研究中控制安慰劑效應的知識呢？

And then the second question is on WAKIX. Can you just speak to where new patient growth is coming from? Are you activating new prescribers? Or is the growth primarily from deeper penetration within existing writers?

第二個問題是關於 WAKIX 的。您能否談談新增患者群主要來自哪些方面？你們是否正在啟用新的處方醫生？或者，這種成長主要來自於對現有作家群體更深入的滲透？

Thanks, Bhavan, for your question. Kumar, on EPX-100?

謝謝 Bhavan 的提問。Kumar，關於EPX-100？

Yeah. Thank you for the question. With the EPX-100, I mean, as you know, what we are studying here is the seizure frequency, which is slightly different from what we studied with ZYN002 in the Fragile X syndrome. Placebo response in general is part and parcel of all neuropsych trials, especially psych trials with behavioral endpoints like Fragile X syndrome. And we had multiple checks and balances within the study to manage the placebo response.

是的。謝謝你的提問。我的意思是，如您所知，我們使用 EPX-100 研究的是癲癇發作頻率，這與我們用 ZYN002 研究脆性 X 綜合徵的情況略有不同。安慰劑效應通常是所有神經心理學試驗的組成部分，尤其是以行為終點為指標的心理學試驗，例如脆性X症候群。我們在研究中設置了多重製衡機制來控制安慰劑效應。

With the EPX-100, it's slightly different in the sense seizure frequency is much more observable and much more definitive compared to some of the behavioral symptoms. So these are two distinct indications with the distinct endpoints. But to your point, in general, yes, I mean, placebo response can happen in any clinical trials, and that's something that we are watching. And we have checks and balances with our EPX-100 ARGUS and LIGHTHOUSE clinical trials as well.

EPX-100 的情況略有不同，因為與某些行為症狀相比，癲癇發作頻率更容易觀察，也更明確。所以這是兩個截然不同的指標，有不同的終點。但正如你所說，總的來說，是的，我的意思是，安慰劑效應可能發生在任何臨床試驗中，這也是我們正在關注的事情。我們的 EPX-100 ARGUS 和 LIGHTHOUSE 臨床試驗也設有相應的製衡機制。

Okay. Adam?

好的。亞當？

Yeah. And then in terms of WAKIX new patients, so the short answer to your question is we see both new patients from increased penetration of existing writers as well as the addition of new writers. We see quarter-over-quarter a pretty steady increase in new writers every quarter, and that's continued for the past several years, and we would expect that to continue.

是的。至於 WAKIX 的新患者，簡而言之，我們看到新患者既來自現有簽約者的滲透率提高，也來自新簽約者的加入。我們看到每個季度的新作者數量都有相當穩定的增長，這種情況在過去幾年中一直持續，我們預計這種情況還會繼續下去。

A little bit more detail. Remember, we call on actually more than 9,000 physicians, and that's 4,000 that are enrolled in the oxybate REMS programs, but also more than 5,000 that are not enrolled in oxybate REMS programs. We have the ability to call on both of those sets of physicians. And in both instances, we see increased penetration as well as new writers.

再補充一些細節。請記住，我們實際上聯繫了 9000 多名醫生，其中 4000 名醫生參加了羥丁酸 REMS 計劃，但還有 5000 多名醫生沒有參加羥丁酸 REMS 計劃。我們有能力聯繫這兩組醫生。在這兩種情況下，我們都看到了滲透率的提高以及新作者的湧現。

Yeah. And I would just add -- yeah. No, thank you for your question. Just to add at a higher level, just to remind everyone that this is a large market. So in terms of the continued growth of WAKIX, a highly differentiated product profile with broad clinical utility, the only nonscheduled product, which is meaningful as a former neurologist treating patients, very meaningful in terms of therapeutic options.

是的。我還要補充一點——是的。不，謝謝你的提問。再補充一點，提醒大家這是一個很大的市場。因此，就 WAKIX 的持續成長而言，其產品特性高度差異化，具有廣泛的臨床用途，是唯一一種非管制藥品，這對於一位治療患者的前神經科醫生來說意義重大，就治療選擇而言意義非常重大。

But in a sizable market, 80,000 patients diagnosed. So as this market evolves and grows and more understanding, similar with the learning about fatigue as a prominent symptom in patients with narcolepsy, about 60%. So designing that into the pitolisant HD program. So I think that is the backdrop why we are confident, why the underlying fundamentals and the growth of WAKIX in narcolepsy and our excitement and confidence in the pitolisant HD program and pitolisant GR, I think that has a lot to do with it as well.

但在一個相當大的市場中，有 8 萬名患者被確診。隨著這個市場的演變和發展，以及人們對疲勞作為嗜睡症患者的主要症狀的認識不斷加深，大約 60% 的人出現了疲勞症狀。因此，我們將此功能設計到 Pitolisant HD 程式中。所以我認為，這就是我們充滿信心的背景，WAKIX 在嗜睡症領域的基本面和成長，以及我們對匹托利桑 HD 計畫和匹托利桑 GR 的興奮和信心，我認為這都與此有很大關係。

Yeah, I think it's a great point, Jeff. I mean the fact that we're at 8,100 average patients now in a market of over 80,000 diagnosed patients, obviously, we have a lot of room to continue to grow.

是的，我覺得你說得很有道理，傑夫。我的意思是，目前我們平均每位患者有 8,100 人，而市場確診患者超過 8 萬，顯然，我們還有很大的成長空間。

Yeah. And also in a polypharmacy market, I think as you're all familiar with, where any chronic neurologic disorders, it's rare that a single mechanism of action will be able to treat difficult-to-treat chronic symptoms. And I think this is what we're seeing as WAKIX continues to grow.

是的。而且在多重用藥市場中，我想大家都很熟悉，對於任何慢性神經系統疾病，單一的作用機制都很少能治療難以治療的慢性症狀。我認為，隨著 WAKIX 的不斷發展，我們正在看到這種情況。

Thank you.

謝謝。

Ash Verma, UBS.

阿什維爾馬，瑞銀集團。

Yeah. So maybe just I wanted to get your latest thoughts on how you're thinking about the overall pipeline and diversification. I mean we've seen two different setbacks recently, first on the IH side and then on Fragile X. What gives you the confidence that the subsequent pipeline programs have higher chances of success?

是的。所以，我只是想了解您對整體產品線和多元化發展的最新想法。我的意思是，我們最近遇到了兩次挫折，一次是IH項目，另一次是脆性X染色體症候群計畫。是什麼讓您相信後續的研發項目有更高的成功幾率？

And then on the WAKIX $1 billion guide, is that something that you can achieve in 2026 just at the pace at which you're going and where 4Q is annualizing at? With Takeda's orexin expected to launch in 2027, what's your level of urgency to hit the long-term guide before the competitor entry?

那麼，關於 WAKIX 10 億美元的目標，按照你們目前的發展速度和第四季度的年化水平，你們能在 2026 年實現這個目標嗎？鑑於武田製藥的食慾素預計將於 2027 年上市，在競爭對手進入市場之前，您有多迫切地想要達到長期目標？

Yeah. Thank you, Ash. Let me start with the first question about our pipeline. And I just want to reiterate that I continue to have strong conviction in our pipeline programs, Kumar's leadership in our R&D organization, which has a lot of experience and expertise. We don't have time, but if you look at each of -- if you look at the IH study, if you look at Fragile X, there are good reasons in terms of the outcomes that we've sort of talked about.

是的。謝謝你，阿什。讓我先回答關於我們管道的第一個問題。我只想重申，我對我們的產品線專案以及庫馬爾在我們研發部門的領導充滿信心，該部門擁有豐富的經驗和專業知識。我們沒有時間，但是如果你仔細看看——如果你看看IH研究，如果你看看脆性X染色體綜合徵，就會發現，就我們已經討論過的結果而言，這些都是有充分理由的。

In the ZYN002 program, in talking with KOLs, unfortunately, that pattern, programs where there were positive Phase 2 data and a lot of expectation because there are no approved treatments and a high placebo response rate kind of got into multiple Phase 3 programs, which is the reason why none have been successful. We have learnings from that, and we will take those learnings going forward. But I think I continue to have conviction in EPX-100, our other pipeline programs.

在 ZYN002 計畫中，與 KOL 溝通後，不幸的是，這種模式出現了：一些計畫在 2 期臨床試驗數據積極，由於沒有獲批的治療方法，且安慰劑效應率很高，因此人們對該計畫抱有很大的期望，結果這些計畫進入了多個 3 期臨床試驗，這也是為什麼沒有一個成功的原因。我們從中吸取了教訓，並將這些教訓運用到未來的工作中。但我仍然對 EPX-100 和我們的其他管道項目充滿信心。

And Kumar, any additional thoughts?

庫馬爾，你還有什麼補充嗎？

Yeah, I think you covered it. Thank you.

是的，我覺得你已經涵蓋到了。謝謝。

Okay.

好的。

Yeah. And then thanks, Ash, for the question around achieving $1 billion in revenue. Obviously, it's a little bit premature to provide guidance for 2026 specifically, but we're seeing very strong momentum as you saw in Q3 and in Q2. We'd expect that momentum to continue in Q4 and well into 2026. And we're confident we will achieve $1 billion in revenue well before LOE. So yeah, confidence remains high.

是的。然後，謝謝 Ash 提出關於如何實現 10 億美元營收的問題。顯然，現在就具體預測 2026 年的情況還為時過早，但正如您在第三季和第二季所看到的那樣，我們看到了非常強勁的成長勢頭。我們預計這種勢頭將在第四季度持續到 2026 年。我們有信心在達到預期支出目標之前實現 10 億美元的營收。所以，信心依然很高。

Thanks, Adam.

謝謝你，亞當。

David Hoang, Deutsche Bank.

David Hoang，德意志銀行。

So I want to ask on eligible patients for WAKIX -- sorry, pitolisant HD and GR. I think for HD, you mentioned new, previous and switch patients, I think, are all on the table. Just wondering what, in your mind, defines a good switch patient for going from WAKIX to HD? And then is switching -- would switching be an option for GR patients? Thanks.

所以我想問 WAKIX 的適用患者有哪些——抱歉，是 pitolisant HD 和 GR。我認為對於HD患者，你提到的新患者、老患者和轉診患者，我認為都在考慮範圍內。我想知道，在您看來，從 WAKIX 轉換到 HD 的理想轉換患者應該具備哪些條件？那麼，換藥－換藥對 GR 患者來說是否是一種選擇？謝謝。

Thanks for the question. So I guess the switch patients, obviously, that's going to be determined by the health care provider, but we know that 75% of patients with narcolepsy continue to struggle with residual symptoms. And so if we're able to offer the HD product with an improved efficacy profile, but the same safety and tolerability profile that they're very familiar with on WAKIX, we would expect HCPs to consider their patients that potentially could benefit from a boost in efficacy and feel confident that they won't be introducing any new safety or tolerability issues.

謝謝你的提問。所以，對於轉換治療的患者來說，顯然這將由醫療保健提供者決定，但我們知道，75% 的嗜睡症患者仍然會受到殘留症狀的困擾。因此，如果我們能夠提供療效更佳、安全性和耐受性與 WAKIX 相同的 HD 產品，我們希望醫療保健專業人員能夠考慮那些可能受益於療效提升的患者，並確信不會引入任何新的安全性或耐受性問題。

And also, any patients that present with fatigue. And as I mentioned before, we know that 60% of patients with narcolepsy do present with fatigue. Those would be prime candidates for the HD as well. And then for the GR, yes, I mean, a switch would be an option certainly for the health care provider. In terms of our strategy, we'll be focused on new patients and previous patients, as I mentioned before.

此外，任何出現疲勞症狀的患者也應受到重視。正如我之前提到的，我們知道 60% 的嗜睡症患者會出現疲勞症狀。這些也都是高清的理想候選者。至於 GR，是的，我的意思是，對於醫療保健提供者來說，轉換肯定是一個選擇。就我們的策略而言，我們將專注於新患者和老患者，正如我之前提到的那樣。

Thank you.

謝謝。

And I'm showing no further questions. I would now like to turn the call back for any closing remarks.

我不會再提其他問題了。現在我想把電話轉回去，請各位作總結發言。

Thanks, operator. On behalf of the Harmony team, I want to thank everyone for joining our call today and for your interest in Harmony Biosciences. Have a great rest of your day. Thank you.

謝謝接線生。我謹代表 Harmony 團隊，感謝各位今天參加我們的電話會議，並感謝大家對 Harmony Biosciences 的關注。祝您今天餘下的時間過得愉快。謝謝。

This does conclude today's Harmony Biosciences third quarter 2025 financial results conference call. You may now disconnect your line.

今天的Harmony Biosciences 2025年第三季財務業績電話會議到此結束。現在您可以斷開線路了。