Zevra Therapeutics Inc (ZVRA) 2025 Q3 法說會逐字稿

Good afternoon and thank you for joining Zevra's third-quarter 2025 financial results and corporate update conference call.

下午好，感謝各位參加 Zevra 2025 年第三季財務業績及公司最新動態電話會議。

Today's call is being recorded and will be available via the Investor Relations section of the company's website, later today.

今天的電話會議正在錄音，稍後將透過公司網站的投資者關係部分提供錄音。

The host for today's call is Nichol Ochsner, Zevra's Vice President of Investor Relations and Corporate Communications. Please go ahead.

今天電話會議的主持人是 Zevra 的投資者關係和企業傳播副總裁 Nichol Ochsner。請繼續。

Thank you and welcome to those who are joining us.

謝謝各位的到來，也歡迎各位的加入。

Today, we will provide an overview of our recent accomplishments, followed by a review of our third-quarter financial results. I encourage you to read our financial results news release, which was distributed this afternoon and is available in the Investors section of our website.

今天，我們將概述我們近期取得的成就，然後回顧我們第三季的財務表現。我建議您閱讀我們今天下午發布的財務業績新聞稿，該新聞稿可在我們網站的投資者關係部分查閱。

Before we begin the call, please note that certain information shared today will include forward-looking statements. Actual results may differ materially from those stated or implied in any forward-looking statements due to risks and uncertainties associated with Zevra's business.

在電話會議開始之前，請注意，今天分享的某些資訊將包含前瞻性陳述。由於與 Zevra 業務相關的風險和不確定性，實際結果可能與任何前瞻性聲明中所述或暗示的結果有重大差異。

Forward-looking statements are not promises or guarantees and are inherently subject to risks, uncertainties, and other important factors that may lead to actual results differing materially from the projections made; and should be evaluated together with the Risk Factors section in our most recent quarterly report on Form 10-Q, our annual report on Form 10-K, and other filings with the SEC.

前瞻性陳述並非承諾或保證，且本身存在風險、不確定性及其他重要因素，可能導致實際結果與預測結果有重大差異；應結合我們最新季度報告（10-Q 表格）、年度報告（10-K 表格）以及向美國證券交易委員會提交的其他文件中的「風險因素」部分進行評估。

I'm pleased to welcome Zevra's management team members participating in today's call. Neil McFarlane, Zevra's President and Chief Executive Officer; LaDuane Clifton, our Chief Financial Officer; and Josh Schafer, our Chief Commercial Officer. Our Chief Medical Officer, Adrian Quartel, will also be available today for the question-and-answer session.

我很高興歡迎 Zevra 的管理團隊成員參加今天的電話會議。Zevra 的總裁兼執行長 Neil McFarlane；財務長 LaDuane Clifton；以及首席商務長 Josh Schafer。我們的首席醫療官阿德里安·夸特爾今天也將出席問答環節。

Now, it's my pleasure to hand the call over to Neil.

現在，我很高興把電話交給尼爾。

Thank you, Nichol. Welcome to everyone joining our quarterly call this afternoon.

謝謝你，尼科爾。歡迎各位參加今天下午的季度電話會議。

Zevra is building on a foundation for success, guided by our strategic priorities and fueled by the strong performance of MIPLYFFA, which is delivering meaningful benefits to patients with Niemann-Pick disease type C or NPC. This impact is translating into revenue generation and leading us towards operating stability that enables us to invest strategically to build a leading rare disease company.

Zevra 正以我們的策略重點為指導，在 MIPLYFFA 的強勁表​​現的推動下，建立起成功的基礎，為患有尼曼匹克病 C 型或 NPC 的患者帶來有意義的益處。這種影響正在轉化為收入，並引領我們走向營運穩定，使我們能夠進行策略性投資，打造一家領先的罕見疾病公司。

MIPLYFFA is a foundational advancement in the management of NPC and the first treatment approved in the United States for this indication. NPC is a progressive and fatal disorder caused by mutations in the NPC1 or NPC2 gene, which disrupts intracellular lipid transportation. This leads to cholesterol and lipid buildup in cells, causing widespread dysfunction.

MIPLYFFA 是 NPC 治療領域的基礎進步，也是美國首個獲準用於治療該疾病的療法。NPC 是一種進行性且致命的疾病，由 NPC1 或 NPC2 基因突變引起，會破壞細胞內脂質運輸。這會導致細胞內膽固醇和脂質積聚，從而引起廣泛的功能障礙。

NPC affects both children and adults with symptoms, including cognitive decline, speech and swallowing difficulties, motor impairments and premature mortality. NPC is considered to be an ultra-rare disease, with an estimated 900 individuals affected in the United States. Of those, only 300 to 350 have been diagnosed.

NPC會影響兒童和成人，其症狀包括認知能力下降、言語和吞嚥困難、運動障礙和過早死亡。NPC 被認為是一種極為罕見的疾病，據估計美國有 900 人患有此病。其中只有 300 至 350 人確診。

We're excited to deliver on the promise of strong scientific data, which demonstrates that MIPLYFFA, in combination with miglustat, is the only disease-modifying therapy that halts the progression of NPC at 12 months; and demonstrated a rapid onset of effect within 12 weeks of treatment initiation.

我們很高興能夠兌現強有力的科學數據所做出的承諾，這些數據表明，MIPLYFFA 與 miglustat 聯合使用是唯一一種可在 12 個月內阻止 NPC 進展的疾病修飾療法；並且證明在治療開始後的 12 週內即可迅速起效。

Long-term data showed that MIPLYFFA was both safe and effective in halting the progression of the disease for more than five years. This compelling data is resonating with the community and driving continued strong performance.

長期數據顯示，MIPLYFFA 既安全又有效，能夠阻止疾病進展長達五年以上。這些引人注目的數據引起了社區的共鳴，並推動了公司持續強勁的業績成長。

I'm pleased to share that our third-quarter net revenue reached $26.1 million, driven by $22.4 million in net revenue from MIPLYFFA. Additionally, there were eight new prescription enrollment forms received in the quarter, bringing the total to 137 since MIPLYFFA became commercially available just a year ago.

我很高興地宣布，我們第三季淨收入達到 2,610 萬美元，其中 MIPLYFFA 貢獻了 2,240 萬美元的淨收入。此外，本季還收到了 8 份新的處方登記表，自 MIPLYFFA 一年前上市以來，總數已達 137 份。

Building on the success of our launch, our commercialization strategy is focused on identifying diagnosed patients who may benefit from MIPLYFFA and on implementing targeted initiatives to improve detection and diagnosis for the many people who remain unaware they are living with this life-threatening disease. We have seen traction in both of these cohorts. Josh will further elaborate later in the call.

繼我們成功推出產品之後，我們的商業化策略重點是確定可能從 MIPLYFFA 中受益的已確診患者，並實施有針對性的舉措，以改善對許多仍不知道自己患有這種危及生命的疾病的人的檢測和診斷。我們已經看到這兩個群體都取得了進展。喬許稍後會在通話中進一步闡述。

A significant near-term growth milestone is to expand into the European market, where we estimate approximately 1,100 people are living with NPC. In Europe, there is an established precedent for treatment with miglustat. With the existing clinical network, there is a favorable environment for a foundational treatment such as MIPLYFFA.

近期一個重要的成長里程碑是拓展到歐洲市場，據我們估計，歐洲約有 1100 人患有 NPC。在歐洲，使用米格魯司他進行治療已有既定先例。憑藉現有的臨床網絡，MIPLYFFA 等基礎治療具有良好的環境。

As a reminder, we filed the Marketing Authorization Application or MAA at the end of July. That application has been validated and is under review by the European Medicines Agency.

再次提醒大家，我們已於7月底提交了上市許可申請（MAA）。該申請已獲批准，目前正在接受歐洲藥品管理局的審查。

We are highly confident in our submission, which incorporates an expanded data set beyond what served as the basis for the FDA filing and USA approval.

我們對提交的資料非常有信心，其中包含的資料集比之前向 FDA 提交申請和獲得美國批准時所依據的資料集更加廣泛。

As a reminder, our filing submission included data on more than 270 NPC patients that have been treated with MIPLYFFA through the pivotal trial, open-label extension study, expanded access programs, and pediatric substudy.

提醒一下，我們提交的文件包含了超過 270 名鼻咽癌患者的數據，這些患者通過關鍵性試驗、開放標籤擴展研究、擴大准入計劃和兒科亞研究接受了 MIPLYFFA 治療。

Our go-to-market strategy for Europe is under active development and will inform our decision to launch independently or through a strategic collaboration.

我們正在積極制定歐洲市場進入戰略，該戰略將指導我們決定是獨立推出還是透過戰略合作推出。

Additionally, our Expanded Access Program in Europe and the UK is playing a critical role in building product experience and fostering strong relationships with physicians and clinics, with 92 patients enrolled at the end of the third quarter. These engagements not only support awareness and education but also lay the groundwork for a potential commercial launch.

此外，我們在歐洲和英國的擴大准入計劃在建立產品體驗和與醫生和診所建立牢固關係方面發揮著至關重要的作用，截至第三季末，已有 92 名患者參與其中。這些活動不僅有助於提高公眾意識和進行教育，而且為潛在的商業化推出奠定了基礎。

We've established a solid patent position for MIPLYFFA. We've also requested a patent term extension with the US Patent and Trademark Office, which could provide coverage beyond the term granted through our orphan drug exclusivity.

我們已為 MIPLYFFA 建立了穩固的專利地位。我們也向美國專利商標局申請延長專利期限，這可以提供超出我們孤兒藥獨佔權期限的保護。

For OLPRUVA, there was one new prescription enrollment form in Q3 and covered lives reached 81%. Notwithstanding our best commercial efforts, OLPRUVA’s clinical differentiation has not penetrated a mature and well-served UCD market.

對於 OLPRUVA，第三季新增了一份處方登記表，涵蓋人數達 81%。儘管我們盡了最大的商業努力，OLPRUVA 的臨床差異化優勢仍未打入成熟且服務完善的 UCD 市場。

As a data-driven organization committed to making judicious investments that drive meaningful benefit for patients, we have made the decision to scale back our sales and marketing efforts for OLPRUVA. While we evaluate the options for OLPRUVA, we will maintain market access and product availability for patients and continue providing support through our patient services program, AmplifyAssist. Going forward, we will provide updates if there's anything material to report.

作為一個以數據為導向、致力於進行明智投資以為患者帶來實質利益的組織，我們決定縮減 OLPRUVA 的銷售和行銷力度。在我們評估 OLPRUVA 的各種方案的同時，我們將保持市場准入和產品供應，為患者提供支持，並透過我們的患者服務計劃 AmplifyAssist 繼續提供支援。今後如有任何重大進展需要報告，我們將及時發布最新消息。

Turning to our development pipeline. We are actively recruiting patients in the DiSCOVER trial, our ongoing Phase III trial to evaluate Celiprolol as a treatment for Vascular Ehlers-Danlos Syndrome, or VEDS. VEDS is a rare inherited disorder caused by mutations in the COL3A1 gene, which impairs Type 3 collagen production, essential for vessel and organ strength, leading to fragile tissues that put patients at high risk of ruptures in the arteries, intestines and uterus. In Q3, we enrolled an additional 5 patients in the DiSCOVER trial, bringing the total to 44 of 150 patients required for full enrollment.

接下來，我們來看看我們的研發流程。我們正在積極招募患者參與 DiSCOVER 試驗，這是一項正在進行的 III 期試驗，旨在評估塞利洛爾作為血管埃勒斯-當洛斯綜合徵 (VEDS) 的治療方法。VEDS 是一種罕見的遺傳性疾病，由 COL3A1 基因突變引起，會損害 3 型膠原蛋白的生成，而 3 型膠原蛋白對於血管和器官的強度至關重要，導致組織脆弱，使患者面臨動脈、腸道和子宮破裂的高風險。第三季度，我們在 DiSCOVER 試驗中又招募了 5 名患者，使總招募人數達到 44 人，而完成全部招募需要 150 名患者。

Our efforts are focused on bolstering our relationships with clinics involved in the management of these patients' care, while also expanding the network of genetic testing centers collaborating with us to identify individuals carrying the gene mutation.

我們致力於加強與參與這些患者護理管理的診所的關係，同時擴大與我們合作的基因檢測中心網絡，以識別攜帶基因突變的個體。

In parallel, we are conducting educational outreach to vascular specialists who frequently encounter these patients following rupture-related surgical interventions. As an event-driven study under a special protocol assessment with the FDA, we have an interim analysis built in after 28 qualifying events. There has been one confirmed event through the end of Q3.

同時，我們正在對血管專科醫生進行教育推廣，他們經常在與破裂相關的外科手術幹預後遇到這些患者。作為一項根據 FDA 特別方案評估進行的事件驅動型研究，我們在 28 個合格事件後進行了中期分析。截至第三季末，已確認發生一起事件。

In summary, we continue to have strong momentum in Q3 and are excited about the many future growth opportunities for MIPLYFFA, including continued performance in the US, potential for patent term extension, significant upside potential through geographic expansion and the late-stage development asset with Celiprolol.

總而言之，我們在第三季度繼續保持強勁勢頭，並對 MIPLYFFA 的眾多未來成長機會感到興奮，包括在美國的持續表現、專利期限延長的潛力、透過地域擴張實現的顯著增長潛力以及與 Celiprolol 的後期開發資產。

I'll now turn the call over to Josh for a deeper dive into our commercial progress with MIPLYFFA.

現在我將把電話交給 Josh，讓他更深入地探討我們在 MIPLYFFA 的商業進展。

Thank you, Neil. Good afternoon.

謝謝你，尼爾。午安.

Physicians have expressed enthusiasm and interest in MIPLYFFA as the foundational treatment for NPC. As Neil mentioned, in the third quarter, we received eight new prescription enrollment forms. This quarter marks the one-year anniversary of the launch. As we reflect on our progress, we are pleased with the overall trends.

醫生們對 MIPLYFFA 作為鼻咽癌的基礎治療方法表現出了熱情和興趣。正如尼爾所提到的，第三季我們收到了八份新的處方登記表。本季度是該產品發布一周年紀念日。回顧我們取得的進展，我們對整體趨勢感到滿意。

We have received enrollment forms from the leading NPC Centers of Excellence that our rare disease specialists have been targeting, demonstrating the impact of our field teams. In addition, our sales efforts have expanded beyond the Centers of Excellence into community practices where many NPC patients are treated after initial diagnosis and treatment recommendations.

我們已經收到了來自領先的 NPC 卓越中心的報名表，這些中心是我們罕見病專家一直重點關注的對象，這表明我們現場團隊的影響力。此外，我們的銷售工作已從卓越中心擴展到社區診所，許多 NPC 患者在初步診斷和治療建議後都會在那裡接受治療。

Our sales and medical teams have been informing and educating clinicians about our differentiated mechanism of action, demonstrated clinical benefit, as well as the recently published long-term data from our open-label extension trial. These compelling data provide real-world evidence that resonates deeply with physicians and patients, alike.

我們的銷售和醫療團隊一直在向臨床醫生介紹我們獨特的作用機制、已證實的臨床益處，以及最近公佈的開放標籤擴展試驗的長期數據。這些令人信服的數據提供了真實世界的證據，能夠引起醫生和患者的強烈共鳴。

We recently conducted independent market research with clinicians and NPC patients. I'm pleased to share that MIPLYFFA was recognized as the preferred disease-modifying therapy for NPC. Clinicians reported confidence in prescribing MIPLYFFA for long-term use based on its efficacy and tolerability. Patients and caregivers described noticing improvements in balance and swallowing, fewer falls, and changes in cognition and speech.

我們近期對臨床醫師和鼻咽癌患者進行了獨立的市場調查。我很高興地宣布，MIPLYFFA 已被公認為鼻咽癌的首選疾病修飾療法。臨床醫師表示，基於MIPLYFFA的療效和耐受性，他們對長期使用MIPLYFFA充滿信心。患者和照護人員表示，他們注意到患者的平衡能力和吞嚥能力有所改善，跌倒次數減少，認知能力和語言能力也有所提高。

We continue to focus on raising awareness of not only MIPLYFFA but also of NPC to support the diagnosis and treatment of new patients. Our early investments in disease awareness initiatives, including our Learn NPC, Read Between the Signs campaign, and our genetic testing resources are identifying patients who were previously undiagnosed.

我們將繼續致力於提高人們對 MIPLYFFA 和 NPC 的認識，以支持對新患者的診斷和治療。我們早期對疾病意識倡議的投資，包括我們的「了解 NPC」、「解讀徵兆」活動以及我們的基因檢測資源，正在識別以前未確診的患者。

Through this testing program, physicians can order a genetic test for individuals suspected of having NPC. Anecdotally, we are hearing of physicians previously unfamiliar with NPC who have identified suspected patients, confirmed diagnosis through our resources and are now prescribing MIPLYFFA.

透過這項檢測計劃，醫生可以為疑似患有鼻咽癌的個體訂購基因檢測。據傳聞，一些以前不熟悉 NPC 的醫生，透過我們的資源發現了疑似患者，確認了診斷，現在正在開立 MIPLYFFA 處方。

We have built bespoke AI-driven models to analyze electronic medical records and claims data to identify both formally diagnosed but untreated patients and those who have not yet received a diagnosis. These examples highlight the tangible impact of increased disease awareness. They underscore the importance of continued education and outreach in ensuring patients receive timely diagnosis and treatment.

我們建立了客製化的 AI 驅動模型，用於分析電子病歷和理賠數據，以識別已正式確診但未接受治療的患者以及尚未確診的患者。這些例子突顯了提高疾病意識所帶來的實質影響。他們強調了持續教育和宣傳的重要性，以確保患者得到及時的診斷和治療。

In the third quarter, we also maintained an impressive presence at local, regional, and national scientific conferences and patient events. These included an oral presentation at the International Niemann-Pick Disease Alliance face-to-face meeting, several poster presentations at the Child Neurology Society Annual Meeting, and four poster presentations at the International Congress of Inborn Errors of Metabolism. The presentations included new data demonstrating that patients who were randomized to receive miglustat only in our pivotal trial and then added MIPLYFFA in the open-label extension phase experienced a decline in annual disease progression.

第三季度，我們在地方、區域和國家科學會議和病患活動中也保持了令人矚目的影響力。其中包括在國際尼曼-匹克病聯盟面對面會議上的口頭報告、在兒童神經病學會年會上的幾張海報展示，以及在國際先天性代謝缺陷大會上的四張海報展示。報告中包含的新數據表明，在我們的關鍵性試驗中隨機分配接受 miglustat 治療的患者，在開放標籤擴展階段添加 MIPLYFFA 後，其年度疾病進展有所下降。

We continue to generate, publish, and present peer-reviewed data to reinforce MIPLYFFA's use as a foundational treatment for NPC.

我們將繼續產生、發布和展示同儕審查的數據，以鞏固 MIPLYFFA 作為 NPC 基礎治療的應用。

Finally, growing market access via reimbursement coverage remains top of mind for us. In the third quarter, we increased the percent of covered lives to 66%, which is in line with what we would expect one year into the launch. We're achieving high overall reimbursement rates for MIPLYFFA, either through direct formulary coverage or through medical exception pathways.

最後，透過醫保覆蓋範圍擴大市場准入仍然是我們最關注的問題。第三季度，我們的投保人數比例提高到 66%，這與我們推出一年後的預期相符。我們透過直接納入藥品目錄或透過醫療例外途徑，實現了 MIPLYFFA 的整體高報銷率。

We seek to provide access to as many patients as possible. To date, there has been broad utilization of our co-pay assistance and patient services.

我們力求讓盡可能多的患者都能獲得治療。迄今為止，我們的共同支付援助和患者服務已被廣泛利用。

Importantly, MIPLYFFA patients are showing continued adherence and staying on treatment. Patients conveniently receive their monthly refills through our AmplifyAssist program, which aims to deliver a first-class experience for patients and has helped to establish Zevra as a preferred partner within the NPC community.

重要的是，MIPLYFFA 患者表現出持續的依從性，並堅持接受治療。患者可以透過我們的 AmplifyAssist 計畫輕鬆獲得每月的藥品補充，該計畫旨在為患者提供一流的體驗，並幫助 Zevra 成為 NPC 社群的首選合作夥伴。

In short, our strong third quarter performance reflects MIPLYFFA’s differentiation and our focused commercial execution, and we are encouraged by the sustained momentum heading into 2026.

簡而言之，我們強勁的第三季業績反映了 MIPLYFFA 的差異化優勢和我們專注的商業執行，我們對進入 2026 年的持續勢頭感到鼓舞。

Now, I will turn the call over to LaDuane.

現在，我將把電話交給拉杜安。

Thank you, Josh. Good afternoon, everyone.

謝謝你，喬許。大家下午好。

In addition to the financial details included in today's call, we encourage you to refer to Zevra's quarterly report on Form 10-Q for more detailed information, which we intend to file later today.

除了今天電話會議中提到的財務細節外，我們建議您參考 Zevra 的季度報告（10-Q 表格），以獲取更詳細的信息，我們計劃於今天晚些時候提交該報告。

In the third quarter of 2025, we reported net revenue of $26.1 million, which includes $22.4 million from MIPLYFFA, $2.4 million in net reimbursements from the French EAP for arimoclomol, $1.2 million from royalties and other reimbursements under the AZSTARYS license, and $100,000 from OLPRUVA.

2025 年第三季度，我們報告淨收入為 2610 萬美元，其中包括來自 MIPLYFFA 的 2240 萬美元，來自法國 EAP 的 arimoclomol 淨報銷款 240 萬美元，來自 AZSTARYS 許可下的特許權使用費和其他報銷款 120 萬美元，以及來自 OLPRU 的 120 萬美元。

For our commercial products, we recognize revenue when shipments are received by the specialty pharmacy. Q3 MIPLYFFA net revenue was impacted by the redesign of Medicare Part D rebates, which led to recognition of a gross to net true-up from prior quarters of $1.2 million. The updated rates will become an ongoing component of gross to net estimates, going forward.

對於我們的商業產品，我們在專科藥房收到貨物時確認收入。第三季 MIPLYFFA 淨收入受到 Medicare Part D 回扣重新設計的影響，導致確認了前幾季毛利到淨利的調整額 120 萬美元。更新後的利率將成為未來毛利淨利估算中持續存在的組成部分。

Cost of product revenue for the third quarter, excluding non-cash intangible asset amortization, was $1.2 million.

第三季產品收入成本（不含非現金無形資產攤提）為 120 萬美元。

Operating expense for the third quarter was $20.4 million, which was a decrease of $6.8 million compared to the same quarter a year ago. R&D expense was $3.4 million for Q3 2025, which was a decrease of $7.5 million compared to Q3 2024, primarily due to reduced third-party costs following the completion of the KP1077 Phase II trial, combined with a decrease in personnel-related costs. SG&A expense was approximately $16.9 million for Q3 2025, which was an increase of $700,000 compared to Q3 2024 due to additional investments in our commercial, medical and launch activities.

第三季的營運支出為 2,040 萬美元，比去年同期減少了 680 萬美元。2025 年第三季的研發費用為 340 萬美元，比 2024 年第三季減少了 750 萬美元，這主要是由於 KP1077 II 期試驗完成後第三方成本降低，以及人員相關成本下降。2025 年第三季銷售、一般及行政費用約為 1,690 萬美元，比 2024 年第三季增加了 70 萬美元，原因是我們在商業、醫療和上市活動方面進行了額外投資。

Looking ahead to the impact of scaling back on promotional activities for OLPRUVA, these resources are expected to be reallocated to MIPLYFFA and to our patient services activities. Therefore, a meaningful change in selling expenses is not anticipated.

展望未來，由於 OLPRUVA 的推廣活動規模縮減，預計這些資源將重新分配給 MIPLYFFA 和我們的患者服務活動。因此，預計銷售費用不會發生實質變化。

Net loss for Q3 2025 was $500,000, which led to loss per basic and diluted share of $0.01. This includes non-cash stock compensation expense of $2.8 million, non-cash fair value adjustment related to warrant, and CVR liabilities of $5.5 million during Q3, and non-cash intangible amortization expense of $300,000. For the same quarter in 2024, we reported a net loss of $33.2 million or $0.69 per share.

2025 年第三季淨虧損為 50 萬美元，導致每股基本及攤薄虧損 0.01 美元。其中包括第三季 280 萬美元的非現金股票補償支出、與認股權證和或有價值權相關的非現金公允價值調整以及 550 萬美元的第三季或有價值權負債，以及 30 萬美元的非現金無形資產攤銷支出。2024 年同期，我們報告淨虧損 3,320 萬美元，即每股虧損 0.69 美元。

As of September 30, 2025, total cash, cash equivalents, and investments were $230.4 million, which was an increase of $12.7 million compared to the end of Q2 2025. The increase in cash included $6.6 million in non-operating cash received in exchange for the exercise of warrants and options during Q3 and $2.3 million in cash from interest income. Total debt was approximately $61.3 million.

截至 2025 年 9 月 30 日，現金、現金等價物和投資總額為 2.304 億美元，比 2025 年第二季末增加了 1,270 萬美元。現金增加包括第三季因行使認股權證和選擇權而收到的 660 萬美元非經營性現金，以及來自利息收入的 230 萬美元現金。總債務約6,130萬美元。

Based on the company's current operating forecast, we believe available financial resources are sufficient to execute on our strategic priorities, independent from the capital markets.

根據公司目前的營運預測，我們認為現有財務資源足以支持我們執行策略重點，而無需依賴資本市場。

While we continue to anticipate variability in our working capital requirements, which is largely dependent on the timing of receivables and payables, our disciplined approach to operating investments and capital allocation has significantly strengthened our financial position.

儘管我們預計營運資金需求仍會波動，這主要取決於應收帳款和應付帳款的時間安排，但我們對營運投資和資本配置採取的嚴謹方法已顯著增強了我們的財務狀況。

Our commitment to maintaining cost efficiency and rigorous operational accountability has cultivated the flexibility and capacity to strategically invest in high-impact opportunities such as the MIPLYFFA US launch and the Phase III study of Celiprolol while also preparing for the potential launch of MIPLYFFA in Europe.

我們致力於保持成本效益和嚴格的營運問責制，這培養了我們的靈活性和能力，可以對具有高影響力的機會進行策略性投資，例如 MIPLYFFA 在美國的上市和 Celiprolol 的 III 期研究，同時為 MIPLYFFA 在歐洲的潛在上市做好準備。

We are well positioned to pursue the opportunities we have to drive long-term value creation for our stakeholders and to accelerate our growth initiatives with confidence.

我們已做好充分準備，抓住機遇，為利害關係人創造長期價值，並充滿信心地加速我們的成長計畫。

Now, I will turn the call back to Neil for his closing remarks.

現在，我將把電話轉回給尼爾，請他作總結發言。

Thank you, LaDuane.

謝謝你，拉杜安。

We're pleased by the continued success of MIPLYFFA and deeply encouraged by the support from both patients and physicians.

我們對 MIPLYFFA 的持續成功感到高興，並對患者和醫生的支持深感鼓舞。

Looking ahead, we believe we have a significant growth opportunity on the horizon with the potential approval and commercialization in Europe. We're committed to aligning our capital allocation with our strategic plan and to invest in opportunities that meaningfully impact patients, while maintaining a sharp focus on creating sustainable growth.

展望未來，我們相信隨著產品在歐洲獲得批准並實現商業化，我們將迎來巨大的成長機會。我們致力於將資本配置與策略計畫保持一致，並投資於能夠對患者產生重大影響的機會，同時始終專注於創造永續成長。

Thank you. We'll now open the call for questions. Operator?

謝謝。現在開始接受提問。操作員？

(Operator Instructions)

（操作說明）

Kristen Kluska, Cantor.

克里斯汀克魯斯卡，坎托爾。

The first one I have is, as we think about the last two quarters, there's been 15 new start forms. Curious if any of these have been patients that are newly diagnosed through perhaps some of your efforts? And also, as we think about the next few quarters up ahead, how you're thinking about the potential of finding additional patients that currently aren't diagnosed today?

首先，回顧過去兩個季度，我們發現有 15 份新的入職表格。想知道這些患者中是否有人是透過您的努力新確診的？另外，展望未來幾個季度，您認為如何才能找到更多目前尚未確診的患者？

Kristen, thanks for the questions. I'll get us kicked off a little bit here.

克里斯汀，謝謝你的提問。我先來帶動氣氛。

You're absolutely right. The last couple of quarters, we've had about 13 new start forms and really pleased with where the team has been continuing to move forward. And actually, as I'm thinking about this, I think it's not 13, but 15, we had 8 and 7.

你說得完全正確。在過去的幾個季度裡，我們收到了大約 13 份新的啟動表格，我們對球隊的持續進步感到非常滿意。實際上，當我思考這個問題時，我覺得不是 13，而是 15，我們之前有 8 和 7。

So I just want to make sure we clarify that on the call. The reality, though, is that our team continues to move forward with the investments that we're moving. I'll ask Josh to talk a little bit more around the newly diagnosed components of where we are.

所以我想確保我們在電話會議上把這一點弄清楚。但現實情況是，我們的團隊將繼續推進我們正在進行的投資。我會請喬希再詳細談談我們目前所處境況中新發現的幾個面向。

Kristen, thanks for the question. And yes, we're really excited not only with the performance we're seeing in terms of the number of enrollments coming in. But as you noted, many of them are coming in as newly diagnosed patients, and that number has increased from last quarter to this quarter. And that is largely through the efforts that we've articulated in the prepared remarks.

克里斯汀，謝謝你的提問。是的，我們不僅對目前招生人數的成長感到興奮，也對其他方面的表現感到非常興奮。但正如您所指出的，其中許多是新確診的患者，而且這一數字比上一季到本季有所增加。這主要歸功於我們在準備好的發言稿中闡述的努力。

But specifically, these are coming from the disease awareness campaign and our partnership with the genetic testing collaborators to be able to identify these new patients and make us aware of these newly diagnosed patients so then we're able to go into those offices and work with the physicians and clinicians to get them onto MIPLYFFA.

但具體來說，這些都來自疾病意識宣傳活動以及我們與基因檢測合作者的合作，以便能夠識別這些新患者，並讓我們了解這些新確診的患者，這樣我們就可以進入這些診室，與醫生和臨床醫生合作，讓他們加入 MIPLYFFA 計畫。

We've also done a lot in terms of helping to build disease awareness through our medical teams and building the evidence through conferences and publications. And we've also done quite a bit in terms of using advanced analytics and machine learning to be able to look into electronic medical records and claims data to find these undiagnosed patients. And so we are seeing a lot of that success.

我們也透過醫療團隊在提高公眾對疾病的認識方面做了很多工作，並透過會議和出版物積累了證據。此外，我們也利用進階分析和機器學習技術，對電子病歷和理賠資料進行深入研究，以發現這些未確診的患者。因此，我們看到了很多這樣的成功案例。

And Kristen, let me follow up on that just a little bit. When we think about our one year in the market, which we're celebrating here right now, the total number is 137 prescription enrollment forms. That's approximately 40% of the diagnosed patient population that we've actually seen.

克里斯汀，我再補充一點。當我們回顧公司上市一周年（我們現在正在慶祝），總共收到了 137 份處方登記表。這大約占我們實際接診的已確診患者總數的 40%。

That's really a testament not just to the team and what they're doing, but also the strength of the long-term data that we've been able to execute on educating physicians and getting that awareness out there with patients and the like. So really pleased with where we are 12 months into the launch.

這不僅證明了團隊及其工作的價值，也證明了我們長期以來收集的數據在教育醫生、提高患者意識等方面的有效性。自上線 12 個月以來，我們對目前的進展感到非常滿意。

Okay. Appreciate that. And then when I think about your balance sheet, it's very robust. And this quarter, you were pretty much on the edge of breaking even. So how are you thinking about perhaps in the near to midterm, how you might take advantage of your robust balance sheet?

好的。謝謝。而且，當我查看你們的資產負債表時，發現它非常穩健。這個季度，你們幾乎是勉強達到收支平衡。那麼，您認為在中短期內，您該如何利用穩健的資產負債表呢？

Yes. Thanks, Kristen. So we go through an annual cycle of planning for strategic session with our Board. And part of that, we thought -- we talk about capital allocation and understanding where is it that we can best be positioned to be able to move forward.

是的。謝謝你，克里斯汀。因此，我們每年都會與董事會進行策略規劃會議。我們認為，其中一部分原因在於——我們討論資本配置，了解我們在哪裡才能更好地定位自己，從而向前發展。

I'll ask LaDuane to touch base on it, but this is all about us leaning in, executing on what we have, doing the best we can for both the commercial products as well as the development programs and then earning the right to do something else later on.

我會請 LaDuane 就此接洽，但這一切都關乎我們全力以赴，做好我們手頭上的事情，盡我們所能做好商業產品和開發項目，然後贏得以後做其他事情的權利。

LaDuane?

拉杜安？

Yes. Thank you, Neil. And Kristen, as we execute against the MIPLYFFA launch, we certainly see that our operating results are beginning to reflect that success. And so we're appreciative and thankful for how that's coming through.

是的。謝謝你，尼爾。克里斯汀，隨著我們執行 MIPLYFFA 的發布計劃，我們確實看到我們的營運表現開始反映出這一成功。因此，我們對此表示感激和讚賞。

Although I will tell you, although we did generate cash -- some cash this quarter, there remains some variability in our results as we continue to have dynamic working capital requirements between accounts receivable timing and accounts payable timing.

雖然我要告訴你們，雖然我們本季確實產生了一些現金流，但由於應收帳款和應付帳款的到帳時間之間存在動態的營運資金需求，我們的業績仍存在一些波動。

So we haven't turned the corner yet, but definitely, things are pointing in the right direction.

所以，我們還沒有扭轉局面，但毫無疑問，事情正朝著正確的方向發展。

And as Neil said, we are very much guided by our strategic plan. And I think executing right now on what's in front of us, the MIPLYFFA launch in the US, the expansion and potential approval in Europe, other ex-US opportunities that we're exploring and then maintaining and continuing to support Celiprolol. These are the opportunities we have in front of us.

正如尼爾所說，我們非常注重策略規劃的指導。我認為，我們現在應該專注於眼前的事情，例如在美國推出 MIPLYFFA，在歐洲擴大規模並獲得潛在批准，探索其他美國以外的機會，然後維持並繼續支持 Celiprolol。這就是我們面前的機會。

And as we execute on those, we can continue to apply discipline and think about how we deploy capital across all the opportunities that may present themselves.

在執行這些計劃的同時，我們可以繼續保持嚴謹的態度，思考如何將資本部署到所有可能出現的機會。

Sami Corwin, William Blair.

薩米·科溫，威廉·布萊爾。

Congrats on the progress. I guess since the majority of patients that are submitting the new enrollment forms or newly diagnosed patients, how are you kind of thinking about reaching the additional 200 or so patients that have already been identified? I guess when I'm kind of thinking about strategy, that kind of seems like it'd be low-hanging fruit. So how are you thinking about that? And then given you're a year into the launch, what percentage of patients are you seeing that are getting refills?

恭喜你取得進展。我想，由於提交新登記表的患者或新確診的患者佔大多數，您打算如何聯繫到另外已經確定的大約 200 名患者呢？我想，當我思考戰略的時候，這似乎是唾手可得的成果。你對此有何想法？那麼，鑑於產品上市已經一年了，您觀察到的續藥患者比例是多少？

Great. So Sami, I want to make sure that I clarify. We didn't say the majority of the new patients that are coming in or hope we didn't say that the majority of the new patients that are coming into prescription forms are undiagnosed patients that are newly diagnosed patients. There are a component of them that are, but majority would be an overstatement. We have actually -- this is not the first quarter we've gotten new patients, though.

偉大的。所以薩米，我想確保我解釋清楚。我們並沒有說大多數新來的患者，或者我們希望我們也沒有說大多數來開處方的新患者是未確診的患者或新確診的患者。其中確實有一部分人是這樣，但說大多數是這樣就言過其實了。實際上，雖然這並不是我們第一個季度迎來新患者，但事實並非如此。

We've actually had new patients in our last quarter as well. So that -- I just want to clarify that.

實際上，我們上個季度也有新病人。所以——我只是想澄清一下。

In regards to how we're thinking about the additional patients that are out there, I'll actually ask Josh to hit on that. And then one year in terms of refills, I'll ask Josh to hit on that as well.

至於我們如何考慮其他患者的問題，我會請 Josh 來談談。然後一年後，在續約方面，我也會請 Josh 在這方面也做些努力。

Yes. So Neil, thank you for clarifying that majority comment. I was going to make the same. The majority of patients who are getting -- submitting enrollment forms are, in fact, patients who have previously been diagnosed and either on a current treatment or have been diagnosed and not received treatment. And we're continuing to find more and more patients like that every day.

是的。尼爾，謝謝你澄清了大多數人的評論。我本來也打算這麼做。事實上，提交登記表的絕大多數患者都是先前已被診斷出患有某種疾病，目前正在接受治療，或已被診斷出患有某種疾病但尚未接受治療的患者。而且我們每天都會發現越來越多這樣的患者。

As we've noted on prior calls, we know where the majority of these patients are, and we continue to go into those offices and work with the clinicians to get those patients on to MIPLYFFA. In addition, we are very pleased that there are undiagnosed or previously undiagnosed patients who are now coming into the enrollment or now receiving enrollment forms as well.

正如我們在先前的電話會議中提到的，我們知道這些患者中的大多數在哪裡，我們將繼續前往這些診所，與臨床醫生合作，讓這些患者接受 MIPLYFFA 治療。此外，我們非常高興地看到，一些未確診或以前未確診的患者現在也開始報名或收到報名表。

With regards to the percent of refills, I would note that we are really, really pleased that the majority of patients are actually who are coming in, we're able to find a pathway to getting reimbursement. We have 66% of covered lives today. And even those who are not covered through formulary, we're able to get paid coverage through a medical exception pathway. And almost all of those patients are getting refills on a month-to-month basis.

關於續藥率，我想指出，我們非常高興的是，大多數前來就診的患者，我們都能找到獲得報銷的途徑。目前我們擁有 66% 的受保人。即使那些不在藥品目錄覆蓋範圍內的人，我們也可以透過醫療例外途徑獲得付費保險。幾乎所有這些患者都是按月續藥的。

Got it. And one more, if I may. What is the average age or weight of the patients currently on therapy? And has that kind of differed with the -- or differed compared to the patients who are on the Expanded Access Program versus those that have come on to drug after?

知道了。如果可以的話，我再補充一個問題。目前接受治療的患者的平均年齡或體重是多少？那麼，與參加擴大用藥計畫的患者相比，參加該計畫的患者與之後才開始服用該藥物的患者相比，情況是否有所不同？

Yes. So I'll take that one, Sami. So when -- if you recall, our clinical trial program was primarily skewed towards children. Our Expanded Access Program actually brought in more adults, and it was about a 50-50 mix between children and adults. As we now actually get into the real world, the real world experience has continued to actually allow us to actually have that [50-50 percent] split in regards to kids and adults.

是的。那我就選這個吧，薩米。所以，如果你還記得的話，我們的臨床試驗計畫主要針對兒童。我們的擴大參與計畫實際上吸引了更多成年人參與，兒童和成人的比例約為 50:50。我們現在真正進入現實世界，現實世界的經驗也讓我們能夠真正實現兒童和成人之間 50-50% 的比例。

In regards to the weight, what we did when we actually announced our pricing for the program is we used an average weight that was based on our 83 Expanded Access Program patients that were in there as well. So we don't provide the guidance specific to weight, but I do believe that it's still at a 50-50 today.

關於體重，我們在公佈該計劃的定價時，採用了基於我們 83 名參與擴大准入計劃的患者的平均體重。因此，我們不提供針對體重的具體指導，但我認為目前體重和健康之間的比例仍然是五五開。

Eddie Hickman, Guggenheim Securities.

艾迪希克曼，古根漢證券。

Congrats on the progress this quarter. Just a few for me. It's really nice impressive increase in coverage, I think, growing from 52% to 66%. So what have been the key drivers to see that much increase this quarter? And how fast and how high do you anticipate that coverage level going?

恭喜本季取得的進展。我只有幾個。我認為這是一個非常可喜的覆蓋率提升，從 52% 增加到 66%。那麼，本季出現如此大幅成長的關鍵驅動因素是什麼？您預計覆蓋率會以多快的速度成長到多高？

And then when you -- now that you have a year under your belt, can you talk about how the patient enrollment form split is between the Centers of Excellence and the non-Centers of Excellence?

那麼，現在你已經工作一年了，你能談談卓越中心和非卓越中心之間的病患登記表分佈嗎？

Yes. I'll ask Josh to take both of those. It’s right up his alley. .

是的。我會請喬希把那兩樣都帶上。這正合他意。。

Yes. So we're really pleased with the progress that we're making in terms of the percent of covered lives. I think it really speaks to the strength of the data and the fact that MIPLYFFA is the only disease-modifying treatment out there with the data that shows that it halts the progression of disease at 12 months. And that data has been really compelling to payers.

是的。因此，我們對在提高參保人數百分比方面取得的進展感到非常滿意。我認為這充分說明了數據的可靠性，也說明 MIPLYFFA 是目前唯一一種有數據顯示能在 12 個月內阻止疾病進展的疾病修飾療法。這些數據對支付方來說確實很有說服力。

We're continuing to see and anticipate that, that coverage will increase. And we feel like we are right where we should be 12 months into launch, and we're going to continue to work to increase that coverage and to ensure as many patients as possible can receive MIPLYFFA.

我們持續看到並預計，這種覆蓋範圍將會增加。我們感覺在上市 12 個月後，我們正處於應該在的位置，我們將繼續努力擴大覆蓋範圍，確保盡可能多的患者能夠接受 MIPLYFFA 治療。

We're also really pleased that we've been able to target and receive enrollment forms from almost all of the Centers of Excellence that our sales representatives or rare disease specialists are targeting. But more importantly, what we're seeing now is that the new enrollments coming in are coming from community-based physicians as well.

我們也非常高興地看到，我們能夠鎖定並收到我們銷售代表或罕見疾病專家所鎖定的幾乎所有卓越中心的報名表。但更重要的是，我們現在看到的是，新增的註冊學員也來自社區醫師。

And the dynamic is maybe a patient will go to one of these Centers of Excellence, get an initial diagnosis and a referral and then go back to his or her local GP or internal medicine specialists who is then managing the care going forward.

這種動態可能是，患者會前往這些卓越中心之一，獲得初步診斷和轉診，然後回到他或她的當地全科醫生或內科專家那裡，由他們負責後續的治療。

So what we're seeing is a real breadth and increase in the overall number of prescribers as well, and we're really pleased with that.

因此，我們看到處方醫生的種類和數量都有了真正的增長，我們對此感到非常高興。

Appreciate that color. And then one final one, if I may. For Europe, when do we expect the next update on that? And when just thinking about maybe just the French EAP, like should we assume a similar conversion as the US EAP once that's approved there? Or help us think about the dynamic of how that EAP will factor into paid drug if you got approved there?

欣賞這種顏色。如果可以的話，我再問最後一個問題。歐洲方面，我們預計何時會收到下一次更新？如果只考慮法國的EAP，我們是否應該假設一旦法國批准了美國EAP，就會進行類似的轉換？或者，請您幫我們思考一下，如果您獲得批准，員工協助方案 (EAP) 將如何影響您的付費用藥？

So Eddie, we are in the valid -- as we talked about in the prepared remarks, we are right now in the post-validation phase, 120-day clock that will allow us to be able to get our first inclination as to where we are. But I think it is important to remind everybody that we are coming off of the back of an FDA approval and an AdCom and a positive vote and a very solid launch into the US market. It's the largest, most compelling data package in NPC. We've added, as we talked about in our prepared remarks, the additional data on top of what we submitted to the FDA.

所以艾迪，我們正處於有效階段——正如我們在準備好的發言稿中談到的那樣，我們現在正處於驗證後階段，為期 120 天，這將使我們能夠初步了解我們所處的位置。但我認為有必要提醒大家，我們剛剛獲得了 FDA 的批准、AdCom 的批准和積極的投票結果，並在美國市場取得了非常穩固的上市。這是NPC中最大、最引人注目的資料包。正如我們在準備好的發言稿中提到的那樣，我們在提交給 FDA 的數據之外，又增加了一些額外的數據。

So we feel really good and confident about the MAA submission. Once we get that 120-day information, it will provide us with an opportunity to know a lot more. I don't know when we will then come back to you and the Street to understand what that looks like because it's a list of questions. It's not an approval. For us to be able to understand how to be able to then talk about that more broadly, I think it will be more closer to the 150 days.

因此，我們對MAA的提交感到非常滿意和有信心。一旦我們獲得這 120 天的信息，我們就能有機會了解更多。我不知道我們什麼時候才能再回到你和華爾街，了解那會是什麼樣子，因為那會引發一連串問題。這並非批准。為了能夠理解如何更廣泛地討論這個問題，我認為時間會更接近 150 天。

So give us some time on that and then see if we come through.

所以請給我們一些時間，看看我們能否成功。

You talked about the French EAP conversion. We do feel really good about the opportunity, but it will be based on reimbursement on the conversion rates within Europe. Once we get the product approved, each country will go country by country around how we reimburse -- navigate reimbursement within those countries. And those countries that come quickest will convert fastest. And today, as I mentioned on the call, we have 92 patients now in our -- it's really, we call it our global EAP.

你談到了法語EAP轉換。我們對這個機會感到非常樂觀，但報銷將以歐洲境內的匯率為準。一旦我們的產品獲得批准，每個國家都會逐一研究如何進行報銷——在這些國家內處理報銷事宜。而那些轉變速度最快的國家，轉型速度也會最快。正如我在電話會議上提到的，今天我們有 92 名患者在我們的——實際上，我們稱之為我們的全球 EAP。

It's primarily Europe now. And those patients are a very small number of countries. So we feel like we've got the ability to continue to drive patients in the EAP -- well, not drive, fulfill patients in the EAP that we'll be able to convert them at the same rate once we get reimbursement in the countries.

現在主要是歐洲了。而且這些患者來自極少數國家。因此，我們感覺我們有能力繼續推動 EAP 中的患者——好吧，不是推動，而是滿足 EAP 中的患者，一旦我們在這些國家獲得報銷，我們就能以同樣的速度將他們轉化為 EAP 患者。

Sumant Kulkarni, Canaccord.

Sumant Kulkarni，Canaccord。

On MIPLYFFA in the US, we appreciate the 40% share that you achieved pretty quickly, but do you think we are at steady state with respect to addition of patient enrollment forms for a quarter? And what are the key variables that might cause a step change in that number, especially given that the sales force will only have MIPLYFFA to focus on given the scale back on OLPRUVA?

在美國的 MIPLYFFA 計畫中，我們很欣賞你們迅速取得的 40% 的市場份額，但你們認為我們在本季度新增病患登記表的數量是否已經穩定下來了？鑑於 OLPRUVA 的規模縮減，銷售人員只能專注於 MIPLYFFA，哪些關鍵變數可能會導致該數字突變？

Yes. So we've seen 13 in the first quarter, 7 in the second quarter, 8 now in this third quarter. I think it's way too early for us to call this a steady state. We are seeing the results of our efforts to find new patients and to diagnose new patients. And as mentioned, several of the new enrollments that we got in the quarter were from newly diagnosed patients.

是的。所以，第一季我們看到了 13 例，第二季 7 例，現在第三季是 8 例。我認為現在就稱之為穩定狀態還為時過早。我們正在看到尋找新患者和診斷新患者所做努力的成果。如同前面所提到的，本季我們新增的幾位患者都是新確診的。

So I expect that we're going to continue to see more of that taking place and the efforts of our sales team to continue to identify currently diagnosed patients, to also find these undiagnosed patients. I think all of that is really telling a good story looking forward.

因此，我預計我們將繼續看到更多此類情況發生，我們的銷售團隊將繼續努力，在識別目前已確診患者的同時，也找到這些未確診的患者。我認為所有這些都預示著一個美好的未來。

I'd also mention that we completed recently some market research, which we completed with payers, clinicians as well as patients. And clinicians stated that they felt very confident in prescribing MIPLYFFA for the long term based on its safety and its efficacy, and they recognized it as really the preferred disease-modifying therapy for NPC.

我還想提一下，我們最近完成了一些市場調查，調查對象包括付款人、臨床醫生和病人。臨床醫生表示，基於 MIPLYFFA 的安全性和有效性，他們對長期使用 MIPLYFFA 非常有信心，並認為它是 NPC 的首選疾病改善療法。

And patients stated that they saw median improvements in their balance, their swallowing, fewer falls and change in cognition. And I think all of that really speaks well for what future quarters might bring.

患者表示，他們的平衡能力、吞嚥能力均有顯著改善，跌倒次數減少，認知能力也有所提升。我認為所有這些都預示著未來幾季可能會出現好兆頭。

Got it. On MIPLYFFA in Europe, what are your initial thoughts on what price might be like relative to the US or the EAP in France? And finally, there's been -- I guess, the sleep market is waking up with buyouts orexin data. What are your latest thoughts on KP1077 and the strategic options there?

知道了。在歐洲舉辦的 MIPLYFFA 展會上，您初步認為其價格相對於美國或法國的 EAP 展會是什麼樣的？最後，我想，睡眠市場正在透過收購食慾素數據而甦醒。您對KP1077地區及其策略選擇有何最新看法？

Yes. Let me take a couple of those. In regards to Europe -- in terms of European pricing, Sumant, we've done in parallel to our marketing authorization application, a lot of work in regards to understanding the market dynamics in Europe, what the pricing potential is in Europe.

是的。讓我拿幾個。關於歐洲市場－就歐洲定價而言，Sumant，我們在申請上市許可的同時，也做了很多工作來了解歐洲的市場動態，以及歐洲的定價潛力。

As you just stated around our EAP in France, we do get pre-commercial revenue from that, which has also been part of the calculation. We won't know based on -- until we understand what the label looks like, what a final price in Europe is.

正如您剛才提到的，我們在法國的 EAP 專案確實能從中獲得預商業收入，這也已納入計算範圍。我們只有看到標籤的樣子，知道歐洲的最終價格，才能知道結果。

What we do really appreciate around Europe is that of the 1,100 patients from a prevalence perspective, there are the majority of those patients with miglustat being approved for well over a decade that have actually been diagnosed and have been on some type of a therapy, that provides us with an opportunity to be able to impact the European market at a faster rate than that of what we even have seen in the US because the diagnosis rates in the US have been lower than that of in Europe. So that's something that, for us, we believe the penetration will be good. Will it be the same price as the US?

我們真正欣賞的是，從患病率的角度來看，1100 名患者中，絕大多數是已經確診並接受過某種治療的米格魯司他患者，而米格魯司他獲批使用已超過十年。這讓我們有機會以比美國更快的速度影響歐洲市場，因為美國的診斷率低於歐洲。所以，我們認為這方面的市場滲透率會很高。價格會跟美國一樣嗎？

That's to be determined at this point.

這一點目前尚待確定。

Got it.

知道了。

You also asked another question, Sumant, around 1077. Yes. No. So to comment on 1077, we remain open to strategic alternatives. We are having conversations, but I can't really comment more on that right now.

Sumant，你還在 1077 年左右問了另一個問題。是的。不。所以對於 1077 號提案，我們仍然對各種策略選擇持開放態度。我們正在進行溝通，但我現在不便對此發表更多評論。

(Operator Instructions)

（操作說明）

Brandon Folkes, H.C. Wainwright.

布蘭登·福克斯，H.C. 溫賴特。

Congratulations on the quarter. Maybe just following on from a few questions before. How are you thinking about additional commercial investments behind MIPLYFFA in the US in 2026 versus the current commercial infrastructure? And given you're seeing some success, right, in the disease awareness campaign driving previously undiagnosed, right?

恭喜你本季取得佳績。或許只是對之前幾個問題的補充。您認為 2026 年在美國對 MIPLYFFA 進行額外商業投資與目前的商業基礎設施相比有何不同？鑑於你們在疾病意識宣傳活動中取得了一些成功，對吧？尤其是在那些以前未被診斷出患有這種疾病的人群中。

I think it's the success to have any of those patients kind of being enrolled at this stage. But how are you weighing up maybe potential additional investments behind disease awareness and increased diagnosis in 2026?

我認為，在這個階段能夠招募到這些患者就是一種成功。但是，您如何權衡在 2026 年可能對疾病意識和診斷率提高進行額外投資的可能性？

So Brandon, I'm going to ask Josh to comment on some of the specifics around this.

布蘭登，接下來我要請喬希就此事的一些具體細節發表一下看法。

But if you recall, we have been investing now into a commercial infrastructure to be able to not only invest in one product but in multiple products in these Centers of Excellence and providing value to these centers that we can then provide the education, the patient services, the medical support, the clinical data that's necessary to be able to pull products through and provide patients with the best experience that they can once they get prescriptions. So I think that our investment is rightsized to be able to move forward and execute on this product.

但如果您還記得，我們​​一直在投資商業基礎設施，以便不僅能夠投資一種產品，還能在這些卓越中心投資多種產品，並為這些中心提供價值，這樣我們就可以提供必要的教育、患者服務、醫療支持和臨床數據，從而推動產品上市，並在患者獲得處方後為他們提供最佳體驗。所以我認為，我們的投資規模合適，能夠推進並執行該產品的開發。

And as we have mentioned in the past, getting OLPRUVA up to speed too in terms of the awareness and clinical differentiation. Josh, do you want to talk a little bit about 2026?

正如我們之前提到的，我們也需要提高 OLPRUVA 的認知度和臨床鑑別能力。喬希，你想稍微談談2026年嗎？

Yes, sure. And just to kind of underscore what Neil said, our top priority from a commercial and medical standpoint is to identify patients who can benefit from MIPLYFFA. So there really is no single tactic that we're talking about. It is a cross-functional across the entire organization. This is a top priority for us.

當然可以。正如尼爾所說，從商業和醫療角度來看，我們的首要任務是確定哪些患者可以從 MIPLYFFA 中受益。所以，我們其實並沒有討論某種單一的策略。這是一項跨部門、貫穿整個組織的行動。這是我們的首要任務。

And so we continue to invest in new publication strategies and new data generation, continuing to look at ways that we can identify patients through claims data and electronic health records. So we're continuing to evaluate that, and our budget is really based on that as a priority. We've been doing that in 2025. We'll continue to do that in '26 and onward. So I don't see any market change in our investments to do that.

因此，我們將繼續投資於新的出版策略和新的數據生成，繼續探索透過理賠數據和電子健康記錄來識別患者的方法。所以我們正在繼續評估這一點，我們的預算也確實以此為優先事項。我們在 2025 年一直在做這件事。2026年及以後，我們將繼續這樣做。所以我認為我們的投資市場不會有任何變化來促成這種情況。

We're going to continue to evaluate the -- what works, what doesn't work and pivot as we need to because it really is an absolute priority for us.

我們將繼續評估哪些方法有效，哪些無效，並根據需要進行調整，因為這確實是我們絕對優先考慮的事情。

Jonathan Aschoff, ROTH Capital.

喬納森·阿肖夫，ROTH Capital。

Congrats on the quarter. And I was just curious, what's the rough cost you expect to have to sell out to launch in the EU? And is there anything noteworthy to say about MIPLYFFA inventory levels end of the second quarter versus end of the third?

恭喜你本季取得佳績。我只是好奇，你們預計在歐盟推出產品的大約成本是多少？MIPLYFFA 第二季末的庫存水準與第三季末的庫存水準相比，有什麼值得注意的地方嗎？

Yes. Thanks, Jonathan. I'll take that in regards to the European opportunity. We've laid out a number of different approaches in terms of the research we've done, the pricing we've done, the approaches we may take. We've been having capabilities presentations from local distributors to some type of a hybrid approach that we might take or to just put it in the hands of folks who can actually deliver for patients better than we could.

是的。謝謝你，喬納森。關於歐洲的機遇，我會接受的。我們已經根據我們所做的研究、我們所做的定價以及我們可能採取的方法，制定了許多不同的方案。我們一直在聽取當地經銷商的能力介紹，探討我們可以採取的某種混合方法，或者乾脆把這項工作交給那些比我們更能為患者提供服務的人。

I mentioned previously that we had 92 patients already in Europe in our Expanded Access Program and continuing to grow that on a quarter-over-quarter basis. Our goal is to be able to either execute on the opportunity in Europe ourselves or put it in the hands of somebody who can do it better, (technical difficulty) shareholders as well. What that cost is, is to really depend upon the approach that we take.

我之前提到過，我們的擴大准入計畫在歐洲已經有 92 名患者，每個季度都在持續成長。我們的目標是要麼自己把握歐洲的機遇，要麼將其交給能夠做得更好的人，（技術難題）股東們也是如此。代價究竟是多少，實際上取決於我們採取的方法。

So I can't give you a number today. But what I can tell you is that we've done all the work that's necessary for either going it alone or put it in the hands of another party that can service patients better and with better economics as well as the ramp that it would take to be able to get MIPLYFFA in the hands of patients in Europe.

所以我今天無法給你一個確切的數字。但我可以告訴你們的是，我們已經完成了所有必要的工作，無論是獨立開展還是將其交給另一方，以便更好地為患者提供服務，並實現更好的經濟效益，以及讓歐洲患者能夠使用 MIPLYFFA 所需的條件。

So stay tuned on what that might look like as we get through our labeling discussions and the MAA.

所以，請繼續關注，看看隨著我們完成標籤討論和 MAA 會議，最終結果會如何。

I'll ask LaDuane to talk about your inventory question on a quarter-over-quarter basis.

我會請 LaDuane 就您提出的季度環比庫存問題進行討論。

Yes. Thank you. The MIPLYFFA inventory, we monitor carefully, and we utilize internal metrics like days on hand to make sure that what's in the channel is consistent and is sufficient to service patients. So there is no large fluctuations quarter-to-quarter since we use those metrics to guide us and make sure that, that's not driving any kind of trends.

是的。謝謝。我們會仔細監控 MIPLYFFA 庫存，並利用庫存天數等內部指標來確保通路中的庫存充足且穩定，足以滿足患者的需求。因此，由於我們使用這些指標來指導我們，並確保它們不會引發任何趨勢，因此季度之間不會出現大的波動。

One other detail I want to make sure is clear, though. When we think about revenue, we also have the impact of our gross to net. And while we don't normally give the details of that, we did note that there was a true-up adjustment from the recent changes redesigned to the Medicare Part D rebates.

不過，還有一點細節我想確認清楚。當我們考慮收​​入時，也要考慮毛利潤與淨利之間的關係。雖然我們通常不會透露具體細節，但我們確實注意到，由於最近對 Medicare Part D 回扣進行了重新設計，因此存在一個調整。

And that did have some impact as we noted. About $1.2 million was recognized in Q3, therefore, reducing net sales by that amount, which was from prior periods.

正如我們所指出的，這確實產生了一定的影響。第三季確認了約 120 萬美元的收入，因此淨銷售額減少了該金額，該金額來自前期。

So just calling out that there's two dynamics there to be clear. Inventory in the channel is consistent at the levels appropriate that we've maintained throughout the launch period. And then this gross to net had a unique impact in Q3, and we've adjusted our rates based on that to go forward from there.

所以，我只是想指出，這裡有兩種動態，需要明確說明。渠道庫存一直保持在我們在整個上市期間所維持的適當水準。然後，毛利與淨利之間的差異在第三季產生了獨特的影響，我們據此調整了未來的費率。

Lastly, Neil, just remind me how many of those 92 patients are French or in France?

最後，尼爾，請你提醒我一下，這92名患者中有多少是法國人或在法國？

Yes. Approximately 30 patients is what has been consistent over a number of years in the French EAP program. It's remained the same.

是的。在法國 EAP 計畫中，多年來一直保持著約 30 名患者的穩定數量。情況依舊如此。

This concludes the Q&A portion of today's call. I'll now turn it back over to Neil for closing remarks.

今天的電話會議問答環節到此結束。現在我將把發言權交還給尼爾，請他作總結發言。

Thank you all for joining us today.

感謝各位今天蒞臨。

Sorry, operator, are you still there?

抱歉，接線生，您還在嗎？

It looks like we have one more person in the queue.

看來我們隊伍裡還有一個人。

Jason Butler, Citizens.

傑森巴特勒，市民。

I guess just wondering if you could comment qualitatively on the number of patients that have received -- have an enrollment form but haven't received reimbursed drug yet. And I guess what I'm asking is directionally quarter-over-quarter, has that remained stable? Or are you seeing that number decrease or any comment there? And then also the time from receiving an enrollment form to getting reimbursed drug. Is that now stable?

我想問的是，您能否就已收到登記表但尚未收到報銷藥物的患者人數進行定性評論？我想問的是，從季度環比來看，這趨勢是否保持穩定？或者您發現這個數字正在下降嗎？或者您對此有什麼看法？此外，也要計算從收到登記表到取得藥品報銷所需的時間。現在情況穩定了嗎？

Or is that changing at all?

或者說，這種情況根本沒有改變？

Yes. We are still in the active phase of launch, Jason, but it's a really good question that I'll ask Josh to hone in on. The pull-through of patients to paid drug, I am incredibly impressed with the team. And then also in regards to as we get new enrollments in and then are able to move through that reimbursement process, team is doing an outstanding job. So Josh?

是的。傑森，我們目前仍處於產品發布的活躍階段，但這確實是個好問題，我會請喬許深入探討。團隊在吸引患者使用付費藥物方面做得非常出色，我對此印象深刻。此外，在新學員報名以及報銷流程方面，團隊也做得非常出色。所以，喬希？

Yes. Jason, thanks for that question. And again, it is something that we're incredibly proud of what we're seeing. The number of patients who are able to receive a paid drug is exceptionally high. We do have patients who are going through some period of free drug as we're investigating their benefits.

是的。傑森，謝謝你的提問。再次強調，我們對目前所看到的一切感到無比自豪。能夠獲得付費藥物的患者人數非常多。我們正在對一些患者進行一段時間的免費用藥，以便研究藥物的益處。

They might go on for a 30-day period. Those patients are oftentimes unable to get into paid patients. So at any given time, we might have some patients in the free goods program who then become paid in the following month. So it's a little bit difficult to give you any precise numbers there. But what I can tell you is the vast majority of them do end up getting paid.

可能會持續30天。這些患者往往無法獲得付費患者資格。因此，在任何時候，我們都可能有一部分患者參加了免費商品計劃，然後在下個月獲得報酬。所以很難給出確切的數字。但我可以告訴你的是，絕大多數人最終都會得到報酬。

And we are seeing -- your second question was around the time from an enrollment coming in to then a paid dispense. And we have seen that time shrunk dramatically since launch. And I think that's due to, again, the execution of our team. It's also due to the payers really beginning to appreciate the strength of MIPLYFFA data, and the benefit that it brings to patients. And so that time has really gone from what was, in some cases, months, now down to weeks.

我們看到──你的第二個問題是關於從註冊到付費配藥的時間。而且我們已經看到，自發布以來，這個時間已經大幅縮短。我認為這再次歸功於我們團隊的執行力。這也要歸功於支付者開始真正認識到 MIPLYFFA 數據的強大之處及其為患者帶來的益處。因此，原本可能需要幾個月的時間，現在已經縮短到幾週了。

And we have, in fact, seen some patients get turned around in 24 hours. And so we're really pleased with that.

事實上，我們已經看到一些患者在 24 小時內病情好轉。所以，我們對此非常滿意。

Got it. And then just a quick follow-up for LaDuane on the gross to net point. Is this re-estimation or true-up something that we should expect to happen on a seasonal basis, an annual basis? Or just how should we think about the stability of the gross to net moving forward?

知道了。然後，我想快速跟進一下拉杜安關於總分和淨分的問題。這種重新估算或調整是按季節還是按年進行的？或者，我們該如何看待未來毛利與淨利比率的穩定性？

Yes. So this specific adjustment came from the redesign of the Medicare Part D, which came out of recent sort of administration changes, Inflation Reduction Act, et cetera. And so this one is sort of, I think, behind us. But -- and let's be clear, this was a change in the rebate in terms of catastrophic coverage. So MIPLYFFA being one of the higher price as a rare disease drug, it does get through to catastrophic coverage under Medicare sooner.

是的。因此，這項具體調整源自於醫療保險D部分的重新設計，而該設計又源自於最近的一些行政改革，如通貨膨脹削減法案等等。所以我覺得，這件事算是過去了。但是——而且要明確一點，這是災難性保險賠償方式的改變。由於 MIPLYFFA 作為一種罕見疾病藥物價格較高，因此它能更快地獲得 Medicare 的災難性醫療費用報銷。

So 2 changes. They made that threshold to get to catastrophic sooner, and they also increased that rebate from 9% up to 20%. So that's a change now that's in the system, and we've adjusted our estimates going forward.

所以有兩處改動。他們提高了災難性損失的門檻，並將退款比例從 9% 提高到 20%。所以，這是系統現在發生的改變，我們已經調整了未來的預測。

But the other detail about gross to net and one of the reasons we don't always dig into the details, except for in this case where it had a meaningful impact in this quarter is there could be other adjustments to gross to net going forward. Payer mix is something that early days in the launch.

但關於毛利與淨利之間的另一個細節，也是我們並非總是深入研究細節的原因之一（除非像本季那樣產生了重大影響），是未來毛利與淨利之間可能還會進行其他調整。支付方組合是產品發布初期需要考慮的問題。

We know where we are today, but that could shift. We could continue to see changes in other dynamics within that. And so gross to net changes probably are something we will live with as a commercial company.

我們知道我們目前所處的位置，但這可能會改變。我們可能會繼續看到其中其他動態的變化。因此，作為一家商業公司，我們可能不得不接受毛利潤與淨利潤之間的變化。

But for right now, I think this specific item is now trued up and would be in the computations.

但就目前而言，我認為這個具體項目已經修正完畢，將會納入計算。

I am showing no further questions at this time.

我目前沒有其他問題要問。

Thank you, operator. Thanks, everybody, for joining us today.

謝謝接線生。謝謝大家今天收看我們的節目。

We look forward to discussing our year-end and Q4 data with you in the coming year.

我們期待在來年與您討論我們的年終數據和第四季度數據。

Thank you. This brings us to the end of today's meeting.

謝謝。今天的會議到此結束。

We appreciate your time and patience. You may now disconnect. Thank you.

感謝您的時間和耐心。您現在可以斷開連線了。謝謝。