Zevra Therapeutics, Inc. (ZVRA) 2026 Q1 法說會逐字稿

Good afternoon and thank you for joining Zevra's first quarter 2026 financial results and corporate update conference call. Today's call is being recorded and will be available via the Investor Relations section of the company's website later today. The host for today's call is Nichol Ochsner, Zevra's Vice President of Investor Relations and Corporate Communications.

下午好，感謝各位參加 Zevra 2026 年第一季財務業績與公司最新進展電話會議。今天的電話會議將進行錄音，並將於今日稍晚在公司網站的投資人關係專區提供回放。今天電話會議的主持人為 Zevra 投資人關係與企業傳播副總裁 Nichol Ochsner。

Thank you and welcome to those who are joining us. Today, we will provide an overview of our recent accomplishments, followed by review of our first quarter 2026 financial results. I encourage you to read our financial results news release, which was distributed this afternoon and is available in the Investor section of our website.

謝謝，並歡迎所有加入我們的與會者。今天我們將先概述近期的主要成果，接著回顧 2026 年第一季的財務業績。我鼓勵各位閱讀我們今天下午發布的財務業績新聞稿，該新聞稿已於今日下午發佈，並可在我們網站的投資人專區查閱。

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. 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 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 our filings with the SEC.

在我們開始之前，請注意今天分享的部分資訊將包含前瞻性陳述。由於與 Zevra 業務相關的風險與不確定性，實際結果可能與任何前瞻性陳述中所述或暗示的內容存在重大差異。前瞻性陳述並非承諾或保證，且本質上受風險、不確定性及其他重要因素影響，可能導致實際結果與所作預測存在重大差異；因此，應結合我們最新的 Form 10-Q 季度報告、Form 10-K 年度報告以及向 SEC 提交之文件中的「風險因素」章節一併評估。

I am pleased to welcome Zevra's management team members participating in today's call: Neil F. McFarlane, Zevra's President and Chief Executive Officer; Joshua Schafer, our Chief Commercial Officer; and Justin Renz, our Chief Financial Officer. Our Chief Medical Officer, Adrian Quartel, will also be available for today's question and answer session.

我很高興歡迎參與今天電話會議的 Zevra 管理團隊成員：Zevra 總裁兼執行長 Neil F. McFarlane；商務長 Joshua Schafer；以及財務長 Justin Renz。我們的醫務長 Adrian Quartel 也將出席今天的問答環節。

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

現在，我很榮幸將電話交給 Neil。

Thank you, Nichol, and welcome to everyone joining our quarterly call this afternoon. We are building a durable rare disease company grounded in disciplined execution, financial strength, and a commitment to patients. We continue to advance our strategic plan, delivering strong performance and positioning ourselves for long-term growth.

謝謝你，Nichol，也歡迎今天下午加入我們季度電話會議的各位。我們正在打造一家具持久性的罕見疾病公司，以嚴謹的執行力、財務實力以及對病患的承諾為根基。我們持續推進策略計畫，交出強勁表現，並為長期成長做好布局。

We made substantial progress in establishing MIPLYFFA as a foundational treatment for Niemann-Pick disease type C, or NPC, in the US by delivering meaningful clinical impact to patients and pursuing multiple pathways to expand patient access globally. We are also advancing our late-stage asset, celiprolol, through a Phase 3 study for the treatment of vascular Ehlers-Danlos syndrome, or vEDS, and executing several approaches to accelerate its development.

我們在美國推動 MIPLYFFA 成為尼曼匹克病 C 型（Niemann-Pick disease type C，簡稱 NPC）的基礎治療方面取得重大進展，透過為病患帶來具意義的臨床影響，並採取多元途徑在全球擴大病患可近性。我們也正推進後期資產 celiprolol，透過一項第 3 期研究用於治療血管型埃勒斯-當洛斯症候群（vascular Ehlers-Danlos syndrome，簡稱 vEDS），並執行多項策略以加速其開發。

To sharpen our focus on high-impact activities and remove operational distractions, we optimized the portfolio by divesting non-core assets with the sale of the SDX portfolio to Commave Therapeutics for $50 million and concurrently resolved a legal dispute. Our balance sheet is strong, with a cash position of $236.8 million and no outstanding debt, providing financial flexibility to drive growth.

為了更聚焦於高影響力活動並排除營運干擾，我們透過出售 SDX 產品組合予 Commave Therapeutics（交易金額 5,000 萬美元）來剝離非核心資產，並同時解決了一項法律爭議，從而優化了產品組合。我們的資產負債表穩健，現金部位為 2.368 億美元，且無任何未償債務，提供推動成長所需的財務彈性。

In the first quarter, total net revenue was $36.2 million, which is a 78% increase over Q1 2025. We've now reached a total of 170 prescription enrollment forms for MIPLYFFA from launch through March 31, nine of which were received in the first quarter. Recall, the estimated prevalence of NPC patients in the US is approximately 900, of whom 300 to 350 are currently diagnosed. Thus, we have successfully reached roughly half of this patient population and continue to have traction in newly diagnosed patients.

第一季總淨營收為 3,620 萬美元，較 2025 年第一季成長 78%。自上市至 3 月 31 日止，MIPLYFFA 的處方登記表累計已達 170 份，其中第一季收到 9 份。提醒各位，美國 NPC 病患的估計盛行人數約為 900 人，其中目前已確診約 300 至 350 人。因此，我們已成功觸及約一半的已確診病患族群，並持續在新確診病患方面取得進展。

This is a significant early launch achievement and remains consistent with the meaningful opportunity ahead for continued growth. As a reminder, we've established a solid patent position for MIPLYFFA. It received orphan drug designation in the US, enabling marketing exclusivity through 2031. Consistent with our strategy to maximize the potential growth drivers for our business, we're pursuing a patent term extension through the US Patent Office and await their decision, which could provide coverage beyond 2031.

這是上市初期一項重要的里程碑，也與未來持續成長的重大機會相一致。提醒各位，我們已為 MIPLYFFA 建立穩固的專利布局。該產品在美國已獲孤兒藥資格認定，可享有至 2031 年的行銷獨占期。依循我們最大化業務成長動能的策略，我們正向美國專利商標局申請專利期限延長，並等待其決定；若獲核准，可能使保護期延伸至 2031 年之後。

Through our Global Expanded Access Program, or EAP, we are able to deliver a much-needed treatment to patients with NPC in certain European countries and select territories outside of Europe, including the UK. As of the end of the quarter, we have a total of 122 patients enrolled in the EAP across geographies. Our global EAP is comprised of multiple access programs, including compassionate use and named patient reimbursement.

透過我們的全球擴大可近性計畫（Global Expanded Access Program，簡稱 EAP），我們得以在部分歐洲國家及歐洲以外的特定地區（包括英國）為 NPC 病患提供迫切需要的治療。截至本季末，我們在各地區 EAP 的累計入組病患總數為 122 人。我們的全球 EAP 由多個可近性方案構成，包括同情用藥（compassionate use）與具名病患給付（named patient reimbursement）。

Within each, we expect variability in enrollment and reimbursement over the first few years until the patient base has stabilized, consistent with our experience in the French EAP program. In Europe, there are an estimated 1,100 individuals with NPC. Diagnosis rates exceed those in the US, largely because the earlier approval of miglustat established physician awareness and enabled patient identification. To support the geographic expansion with the potential approval of arimoclomol in Europe, we have a marketing authorization application under review by the European Medicines Agency, or EMA.

在各方案中，預期在最初幾年入組與給付情況會有所波動，直到病患基礎趨於穩定；這與我們在法國 EAP 計畫中的經驗一致。在歐洲，估計約有 1,100 名 NPC 患者。歐洲的診斷率高於美國，主要因為 miglustat 較早獲批，提高了醫師認知並促進病患辨識。為支持地理擴張並因應 arimoclomol 在歐洲可能獲批，我們已向歐洲藥品管理局（European Medicines Agency，簡稱 EMA）提交上市許可申請，目前正處於審查中。

We submitted our responses to the EMA's 120-day list of questions within the 90-day clock stop period and are progressing along the standard review process to make arimoclomol available to the European NPC community. As a reminder, we have also received orphan medicinal product designation in Europe for the treatment of NPC.

我們已在 90 天停鐘期內，提交對 EMA「120 天問題清單」的回覆，並正依標準審查流程推進，以便讓 arimoclomol 可供歐洲 NPC 社群使用。提醒各位，我們亦已在歐洲取得用於治療 NPC 的孤兒藥品資格認定（orphan medicinal product designation）。

Turning to our late-stage pipeline, our Phase 3 DiSCOVER trial is evaluating celiprolol for the treatment of vEDS, a rare inherited connective tissue disorder caused by COL3A1 gene mutations that weaken the walls of blood vessels and hollow organs and can cause arterial rupture or dissection, among other complications. Approximately 90% of patients experience an event by the age of 40. There are roughly 7,500 individuals living with vEDS in the US.

接著談到我們的後期研發管線，第 3 期 DiSCOVER 試驗正在評估 celiprolol 用於治療 vEDS。vEDS 是一種罕見的遺傳性結締組織疾病，由 COL3A1 基因突變所致，會削弱血管與中空器官壁，並可能造成動脈破裂或剝離等併發症。約 90% 的病患在 40 歲前會發生事件。美國約有 7,500 名 vEDS 患者。

Celiprolol is a selective adrenoreceptor modulator that works by inducing vascular dilation and smooth muscle relaxation, thereby reducing mechanical stress on tissues of the arterial wall and hollow organs. Currently, there are no approved therapies for vEDS. Our commercialization strategy for celiprolol is focused exclusively on the US, where there's a clear opportunity to fill an unmet need.

Celiprolol 是一種選擇性腎上腺素受體調節劑，透過誘導血管擴張與平滑肌鬆弛來發揮作用，從而降低動脈壁與中空器官組織所承受的機械性壓力。目前尚無任何獲核准的 vEDS 治療。我們對 celiprolol 的商業化策略將專注於美國市場，因為在此存在明確機會可填補未被滿足的醫療需求。

While not approved in Europe for vEDS, celiprolol is the primary off-label treatment in several European countries. This use is supported by the results of several studies, including long-term European cohorts. We've enrolled a total of 62 patients in the DiSCOVER trial, with 10 patients enrolled in the first quarter. This is an event-driven study, we have two confirmed events out of the 28 events required to trigger the interim analysis.

雖然 celiprolol 尚未在歐洲獲核准用於 vEDS，但在多個歐洲國家，它是主要的適應症外（off-label）治療選項。此用途獲多項研究結果支持，包括歐洲長期隊列研究。我們在 DiSCOVER 試驗中累計已入組 62 名病患，其中第一季入組 10 名。這是一項以事件驅動的研究；在觸發期中分析所需的 28 起事件中，我們目前已確認 2 起事件。

We continue to implement activities aimed at driving enrollment, including building a network of genetic testing centers to improve diagnosis, as well as strengthening connections with key specialists who manage these patients. In parallel, following the FDA Type C meeting we had in the first quarter, we are preparing for a follow-up meeting in the second half of the year to explore pathways to accelerate its clinical development. In summary, we have a clear vision to become a leading rare disease therapeutics company, and we're motivated by the momentum and the opportunity that this phase of growth brings. I'll now turn the call over to Josh to review our commercial performance in more detail. Josh?

我們持續推動旨在帶動收案的各項活動，包括建立基因檢測中心網絡以改善診斷，並加強與負責管理這些病患的關鍵專科醫師之連結。同時，繼第一季我們與 FDA 召開 Type C 會議後，我們正準備於下半年進行後續會議，以探討加速其臨床開發的途徑。總結而言，我們對成為領先的罕見疾病治療公司有清晰願景，並受到此成長階段所帶來的動能與機會所激勵。接下來我將把電話會議交給 Josh，請他更詳細回顧我們的商業表現。Josh？

Thank you, Neil, and good afternoon. Before reviewing the quarterly progress with my MIPLYFFA, I'll provide a quick background on NPC. NPC is a rare lysosomal storage disorder caused by mutations in genes that impair intracellular cholesterol and lipid trafficking, leading to the abnormal lipid accumulation in the brain, liver, spleen, and other organs. The onset and course of disease are heterogeneous, ranging from infancy to adulthood, with progressive neurodegeneration that can vary in both speed of onset and clinical presentation.

謝謝你，Neil，大家午安。在回顧我們 MIPLYFFA 的季度進展之前，我先簡要介紹 NPC 的背景。NPC 是一種罕見的溶小體儲積症，由基因突變導致細胞內膽固醇與脂質運輸受損，進而造成脂質在腦、肝、脾及其他器官異常累積。其發病時間與病程具異質性，從嬰兒期到成人期皆可能發生，並伴隨進行性神經退化，且在發病速度與臨床表現上皆可能有所差異。

The extensive data generated for MIPLYFFA in NPC has shown long-term meaningful patient outcomes through the most expansive clinical development program in NPC to date. We have more than five years of data across more than 270 NPC patients worldwide through clinical studies, including our pivotal trial, open label extension study, global EAP, and pediatric sub-study, all demonstrating MIPLYFFA's efficacy and safety.

MIPLYFFA 在 NPC 的大量數據顯示，透過迄今 NPC 最廣泛的臨床開發計畫，帶來長期且具意義的病患結局。我們在全球超過 270 名 NPC 病患中累積了超過五年的數據，來源涵蓋臨床研究（包括我們的關鍵性試驗）、開放標籤延伸研究、全球 EAP，以及兒科子研究，均顯示 MIPLYFFA 的療效與安全性。

Notably, MIPLYFFA in combination with miglustat is the first and only disease-modifying therapy shown to halt disease progression at 12 months in a randomized controlled trial based on the validated NPC Clinical Severity Scale. The onset of benefit is rapid, with improvements noted at the first clinical evaluation time point of 12 weeks and has durable efficacy with treatment effects sustained for over five years.

值得注意的是，MIPLYFFA 與 miglustat 併用，是首個且目前唯一在隨機對照試驗中，依據已驗證的 NPC 臨床嚴重度量表（NPC Clinical Severity Scale）顯示可在 12 個月時停止疾病進展的疾病修飾療法。其效益出現迅速，在 12 週的首次臨床評估時間點即觀察到改善，且療效持久，治療效果可維持超過五年。

We are pleased to announce that MIPLYFFA was added to the NPC clinical practice guidelines, which were recently published in the Journal of Inherited Metabolic Disease, marking the first update to the guidelines since initial publication in 2018. These guidelines discuss the heterogeneity of the disease and reinforce that the NPC Clinical Severity Scale and genetic testing are the most reliable clinical endpoints and confirmation of diagnosis.

我們很高興宣布，MIPLYFFA 已被納入 NPC 臨床實務指引；該指引近期刊登於《Journal of Inherited Metabolic Disease》，並標誌著自 2018 年首次發布以來的首次更新。這些指引討論了疾病的異質性，並強調 NPC 臨床嚴重度量表與基因檢測是最可靠的臨床終點與診斷確認方式。

The guidelines also point out, consistent with our messaging, that early detection is critical to delay disease progression. MIPLYFFA's mechanistic and clinical differentiation is resonating with prescribers and patients and is driving adoption. As Neil shared, we've received a total of 170 prescription enrollment forms since launch, with nine enrollment forms coming in Q1.

該指引亦指出，與我們的訊息一致，早期偵測對延緩疾病進展至關重要。MIPLYFFA 在作用機轉與臨床上的差異化正引起處方醫師與病患共鳴，並帶動採用。如 Neil 所分享，自上市以來我們共收到 170 份處方收案表，其中第一季收到 9 份收案表。

Our commercial strategy is focused on three key priorities: accelerating time to diagnosis and treatment, driving demand, and facilitating access to MIPLYFFA. NPC remains significantly underdiagnosed and often diagnosed late due to heterogeneous symptoms. To enable earlier diagnosis, we have focused on education and engagement within the medical community through a strong presence at medical conferences where we regularly present data and through our ongoing disease awareness campaign called Learn NPC, Read Between the Signs.

我們的商業策略聚焦三項關鍵優先事項：加速診斷與治療的時間、帶動需求，以及促進 MIPLYFFA 的可近性。由於症狀異質性，NPC 仍嚴重被低估診斷，且常常在較晚期才被確診。為了促成更早診斷，我們著重於醫療社群的教育與互動：透過在醫學會議上的強勢參與並定期發表數據，以及我們持續進行的疾病衛教活動「Learn NPC, Read Between the Signs」。

Additionally, we have built a custom AI-driven targeting model to find likely NPC patients and have collaborations with providers of genetic testing to accelerate their diagnosis. As a result, we continue to see new enrollments from previously diagnosed as well as newly diagnosed patients. We are also finding patients and seeing demand for MIPLYFFA increase outside of the centers of excellence. Our prescriber base is expanding to include community-based prescribers, which we believe reflects the success of our targeting and education efforts.

此外，我們建立了客製化的 AI 驅動目標鎖定模型，以找出可能的 NPC 病患，並與基因檢測服務提供者合作以加速其診斷。因此，我們持續看到來自既有確診與新確診病患的新收案。我們也在卓越中心（centers of excellence）之外找到病患，並看到對 MIPLYFFA 的需求增加。我們的處方醫師基礎正在擴大，納入社區型處方醫師；我們相信這反映了我們目標鎖定與教育工作的成功。

Many of these new prescribers did not know that they had NPC patients in their practice and were previously unfamiliar with the disease and treatment options. We help facilitate medical education efforts through various initiatives, such as the recently launched Expert Connect, which connects HCPs unfamiliar with NPC to experts who can address questions regarding disease state and available treatment options.

許多新加入的處方醫師先前並不知道其診所中有 NPC 病患，且過去對該疾病與治療選項並不熟悉。我們透過多項計畫協助推動醫學教育，例如近期推出的 Expert Connect，將不熟悉 NPC 的醫療照護專業人員（HCPs）與專家連結，協助解答疾病狀態與可用治療選項相關問題。

Our patient mix has grown to include adults and children equally. These trends give us confidence in the estimated prevalence of 900 people in the US living with NPC, and we remain focused on reaching as many patients as possible and expanding the total addressable market. From a market access standpoint, we have stable coverage of 69% and continue to achieve reimbursement through the medical exception pathway.

我們的病患組合已成長至成人與兒童各占一半。這些趨勢讓我們對美國約有 900 名 NPC 患者的盛行率估計更具信心；我們仍專注於觸及盡可能多的病患並擴大全可服務市場（total addressable market）。就市場准入而言，我們維持 69% 的穩定給付覆蓋率，並持續透過醫療例外（medical exception）途徑取得給付核付。

Payer engagement continues to be focused on emphasizing a robust clinical safety and efficacy data and the extensive real-world evidence seen in clinical practice that supports MIPLYFFA's value. We believe we are differentiating MIPLYFFA through its clinical benefit, support services, and broad patient access. An independent market research suggests MIPLYFFA is the preferred NPC therapy most trusted by clinicians and shown to improve balance, swallowing, cognition, speech, and reduce falls.

與支付方的互動仍聚焦於強調扎實的臨床安全性與療效數據，以及臨床實務中所見的大量真實世界證據，這些都支持 MIPLYFFA 的價值。我們相信，我們透過其臨床效益、支援服務與廣泛的病患可近性，讓 MIPLYFFA 具備差異化。一項獨立市場研究顯示，MIPLYFFA 是臨床醫師最信賴、且偏好的 NPC 治療，並被證實可改善平衡、吞嚥、認知與語言能力，並降低跌倒。

We receive heartwarming letters from families noting how positively impacted they have been by MIPLYFFA and the support of our AmplifyAssist Patient Services program. Together, this feedback reflects the impact of our commercial activities and sets the stage for continued growth. With that, I will turn the call over to Justin to review our financial results.

我們收到許多家庭令人感動的來信，提到 MIPLYFFA 以及我們 AmplifyAssist 病患服務計畫的支持，對他們帶來了非常正面的影響。綜合而言，這些回饋反映了我們商業活動的成效，並為持續成長奠定基礎。接下來，我將把電話會議交給 Justin，請他回顧我們的財務結果。

Thank you, Josh. In addition to the financial details included in today's call, we encourage you to refer to our quarterly report on Form 10-Q for more detailed information, which we intend to file shortly. As Neil mentioned, in the 1st quarter of 2026, we generated net revenue of $36.2 million, which was an increase in total net revenue of $15.8 million compared to $20.4 million in Q1 of 2025. This is comprised of $24.6 million from MIPLYFFA net sales in the United States, $0.3 million from OLPRUVA, $10.2 million in net reimbursements from the global EAP for arimoclomol, and $1.1 million in royalty revenue.

謝謝你，Josh。除了今天電話會議中包含的財務細節外，我們也建議各位參閱我們即將提交的季度報告 Form 10-Q，以取得更詳細資訊。如 Neil 所提到，2026 年第一季我們的淨營收為 3,620 萬美元，較 2025 年第一季的 2,040 萬美元增加 1,580 萬美元。其組成包括：美國 MIPLYFFA 淨銷售 2,460 萬美元、OLPRUVA 30 萬美元、arimoclomol 全球 EAP 的淨補償款 1,020 萬美元，以及權利金收入 110 萬美元。

It is worth noting that we had one less shipment week of MIPLYFFA in the US due to the first quarter delivery calendar, and as a result, channel inventory fell below the low end of our targeted range. Turning to recent business transactions, in March, we executed an agreement with Commave Therapeutics SA for the sale of the SDX portfolio for $50 million, monetizing assets that were not central to our core investment thesis.

值得一提的是，由於第一季的出貨日曆安排，我們在美國少了一週的 MIPLYFFA 出貨，因此通路庫存降至我們目標區間的下緣以下。談到近期的商業交易，3 月我們與 Commave Therapeutics SA 簽署協議，以 5,000 萬美元出售 SDX 產品組合，將非我們核心投資主軸的資產變現。

Per our contractual obligations, Aquestive Therapeutics received 10% or $5 million of gross proceeds. In the first quarter, we received $40.5 million of the $45 million in net proceeds, and we received a final payment of $4.5 million in April. In connection with this transaction, we reviewed our capital allocation strategy and retired our debt early, saving on average approximately $8 million a year in future interest expense.

依據我們的合約義務，Aquestive Therapeutics 取得總收益的 10%，即 500 萬美元。第一季我們已收到 4,500 萬美元淨收益中的 4,050 萬美元，並於 4 月收到最後一筆 450 萬美元款項。配合此交易，我們檢視了資本配置策略並提前清償債務，使未來利息費用平均每年節省約 800 萬美元。

We are now debt-free and strategically positioned for growth, supported by a clean balance sheet. These one-time transactions impacted our first quarter financials. Accordingly, we recorded a one-time gain of approximately $43.3 million, partially offset by an approximately $10 million expense associated with the early extinguishment of debt, which is noted in the other income and expense section in our financial statements. Pivoting back to normal operations, during the first quarter of 2026, our operating expenses were $25.2 million, which was an increase of $2.4 million compared to the same quarter a year ago. R&D expense was $4.4 million for Q1 2026, which was an increase of $1.1 million compared to Q1 2025, due primarily to increases in third-party costs and professional fees.

我們目前已無負債，並在乾淨的資產負債表支持下，於策略上處於成長的有利位置。這些一次性交易影響了我們第一季的財務表現。因此，我們認列約4,330萬美元的一次性收益，部分被與提前清償債務相關、約1,000萬美元的費用所抵銷；該費用列示於財務報表中的其他收入及費用項下。回到正常營運面，於2026年第一季，我們的營業費用為2,520萬美元，較去年同期增加240萬美元。2026年第一季研發費用為440萬美元，較2025年第一季增加110萬美元，主要係因第三方成本與專業費用增加所致。

SG&A expense was $20.8 million for Q1 2026, which was an increase of $1.2 million compared to Q1 2025, primarily due to an increase in professional fees, partially offset by a decrease in third-party spending. We have utilized the vast majority of our usable net operating loss carryforwards. As a result of the multiple one-time transactions that we recorded in the first quarter, we incurred an estimated tax provision of $6.9 million. Net income for the first quarter of 2026 was $37.9 million or $0.62 per basic and $0.60 per diluted share, compared to a net loss of $3.1 million or $0.06 per basic and diluted share for the same quarter a year ago.

2026年第一季銷售、一般及行政（SG&A）費用為2,080萬美元，較2025年第一季增加120萬美元，主要因專業費用增加，部分被第三方支出下降所抵銷。我們已使用絕大部分可用的淨營業虧損結轉扣抵（NOL）。由於我們在第一季認列了多項一次性交易，因此估列所得稅費用為690萬美元。2026年第一季淨利為3,790萬美元，基本每股盈餘0.62美元、稀釋每股盈餘0.60美元；相較之下，去年同期淨損為310萬美元，基本及稀釋每股虧損均為0.06美元。

Excluding the one-time transactions as well as the related tax provision, for clearer comparability across periods, the estimated quarterly net income reported would be $11.5 million or $0.18 per diluted share. As of March 31, 2026, total cash equivalents, and investments were $236.8 million, which was a decrease of $2.1 million compared to December 31, 2025. As mentioned earlier, this decrease is attributable to deleveraging driven by our debt payoff, partially offset by the non-dilutive capital proceeds from the sale of the SDX portfolio and supported by our operating income. Collectively, these factors have further fortified our balance sheet, and we remain well-positioned with the financial capacity to execute on our strategic priorities independent of the capital markets.

為了在各期間間有更清楚的可比性，若排除一次性交易及其相關所得稅費用，則所報告的估計季度淨利將為1,150萬美元，稀釋每股盈餘0.18美元。截至2026年3月31日，現金約當物及投資總額為2.368億美元，較2025年12月31日減少210萬美元。如先前所述，該減少主要歸因於我們償還債務所帶動的去槓桿，部分被出售SDX投資組合所取得的非稀釋性資金收入所抵銷，並且受到營業收入的支持。綜合而言，這些因素進一步強化了我們的資產負債表；我們仍具備充足的財務能力，可在不依賴資本市場的情況下執行我們的策略優先事項。

Now I'll turn the call back to Neil for his closing remarks. Neil?

現在我把電話交回給Neil，請他做結語。Neil？

Thanks, Justin. Our corporate profile has evolved significantly with the execution against the strategic pillars we introduced a little over a year ago. We are delivering strong commercial execution while thoughtfully monetizing assets that are not core to our business. We relocated our corporate headquarters to Boston, a hub of biotech innovation, and we strengthened our leadership by attracting seasoned professionals to our executive management team and board of directors, bringing valuable experience and perspective.

謝謝你，Justin。自我們在一年多前提出策略支柱以來，隨著我們的執行推進，公司面貌已顯著演進。我們在商業執行上展現強勁成果，同時審慎地將非核心資產變現。我們已將公司總部遷至波士頓——生技創新的樞紐——並透過延攬資深專業人士加入高階管理團隊與董事會來強化領導力，帶來寶貴的經驗與觀點。

These efforts, combined with prudent financial stewardship, are positioning us to deliver on our vision. As we advance through 2026, we are anchored by a clinically meaningful commercial product with multiple opportunities for global growth, a late-stage pipeline, and a strong financial position. Our collective team at Zevra is energized by the numerous opportunities we have to expand our impact for people living with rare diseases.

這些努力，加上審慎的財務管理，正使我們能夠實現願景。在我們邁向2026年的過程中，我們以一項具臨床意義且已商業化的產品為基礎，並擁有多項全球成長機會、後期研發管線，以及強健的財務狀況。Zevra全體團隊對於能為罕見疾病患者擴大影響力的眾多機會感到振奮。

Operator, please open the line for questions.

接線員，請開放提問。

(Operator Instructions) Kristen Kluska, Cantor Fitzgerald.

(接線員指示) Kristen Kluska，Cantor Fitzgerald。

This is Aya on Kristen's line. Congrats on the quarter updates here and thank you for taking our questions. First, now that MIPLYFFA has been added to the NPC clinical practice guidelines, we're just wondering what this means for physician adoption. Are these doctors now formally advised to consider MIPLYFFA when treating these patients?

我是代Kristen發言的Aya。恭喜本季的更新，也謝謝你們回答我們的問題。首先，既然MIPLYFFA已被納入NPC臨床實務指引，我們想了解這對醫師採用意味著什麼。這些醫師現在是否被正式建議在治療這些患者時考慮使用MIPLYFFA？

Thank you for the question. We're really pleased that I think as we've been previously communicating, these guidelines were in process and really came out quite fast after the introduction of new products that were approved in the US. It really reinforces the NPC Clinical Severity Scale as the tool that shows disease progression, the genetic testing as a key endpoint and diagnostic tool.

謝謝你的提問。我們非常高興；正如我們先前一直在溝通的，這些指引正在制定中，且在美國新產品獲准上市後很快就發布了。這也再次強化了NPC臨床嚴重度量表作為顯示疾病進展的工具，以及基因檢測作為關鍵終點與診斷工具的重要性。

The complexity of the disease when we think about the heterogeneity of both infantile version and adult versions of the disease, it's really important to early detect in order to be able to delay progression. Really importantly, the combination therapy being considered for NPC was one of the major takeaways. I'll ask Josh to talk a little bit about some of the impact that we see, and quite frankly, the hurdles that are lowered with these guidelines that have been published.

當我們考量此疾病在嬰幼兒型與成人型之間的異質性時，疾病的複雜性很高，因此及早偵測對於延緩進展至關重要。同樣非常重要的是，指引將NPC的合併治療納入考量，這是主要重點之一。我請Josh談談我們所看到的一些影響，以及坦白說，這些已發布的指引降低了哪些障礙。

Yeah. Thanks, Neil. Just to add to what Neil said, we're also really pleased that the guidelines addressed the need for early detection and that early treatment helps delay progression. It also went on to talk about the importance of using combination therapy for patients who have been diagnosed with NPC. This is really important because these are the opinions of a select group of key opinion leaders that we're now able to use and communicate and help build confidence and consistency in the way that some HCPs who might not be as familiar with NPC can now use this as a consistent guide for their treatment.

好的。謝謝你，Neil。補充Neil所說的，我們也很高興指引提到早期偵測的需求，以及早期治療有助於延緩進展。指引也進一步談到，對已確診NPC的患者使用合併治療的重要性。這點非常重要，因為這些是特定一群關鍵意見領袖（KOL）的觀點，我們現在能夠加以運用並對外溝通，協助建立信心與一致性，讓一些可能不那麼熟悉NPC的醫療照護專業人員（HCP）也能將其作為一致的治療指引。

Thank you for that color. And then second, do you have a sense of the proportion of the patients that identify through the genetic testing that you're conducting and the AI-driven predictive model that ultimately convert onto the drug? I guess --

謝謝這些補充說明。第二個問題，你們是否掌握一個比例：透過你們正在進行的基因檢測與AI驅動的預測模型所識別出的患者，最終有多少會轉換為用藥？我想--

No, sorry, please finish your question.

不，不好意思，請把問題問完。

Yeah, just related to that, we're wondering what feedback you hear from the physicians that are managing these patients in terms of, like, how receptive they're being to these data-driven identification approaches that you're using. Thank you so much.

好的，延續剛才那點，我們想了解你們從管理這些患者的醫師那邊聽到的回饋，例如他們對你們採用的這些以數據驅動的識別方法接受度如何。非常感謝。

Yeah, wonderful question, and thanks for outlining our commercial strategy really clearly for us. Let me ask Josh to touch on some of the key priorities that we're executing against and some of the feedback that we're getting.

是的，這是個很棒的問題，也謝謝你把我們的商業策略梳理得很清楚。我請Josh談談我們正在執行的幾項關鍵優先事項，以及我們收到的一些回饋。

As we mentioned in the prepared remarks, we have three key priorities from a commercial perspective. The first is to accelerate the time to diagnosis and treatment. We're seeing that through our disease awareness campaign as well as some of the collaborations with the genetic testing companies.

如同我們在事先準備的講稿中提到，從商業面來看我們有三項關鍵優先事項。第一是加速從診斷到治療的時間。我們透過疾病認知宣導活動，以及與基因檢測公司的合作，正在看到這方面的成效。

Our second priority is to really drive demand. And you're seeing that in the enrollments that we've been able to get over the past quarter and more and then facilitate access with 69% of covered lives or 69% of covered lives currently able to access MIPLYFFA, and the others we're able to do through medical exception pathways. So we feel really confident in our line of sight for more patients given what we're seeing today, which is a nice mix of newly diagnosed as well as previously diagnosed patients.

第二項優先事項是確實推動需求。你們可以從我們在過去一季及之後所取得的收案數中看到；同時也促進可近性，目前有69%的受保人群（covered lives）或69%的受保人群可取得MIPLYFFA，其餘則可透過醫療例外（medical exception）途徑取得。因此，基於我們目前所看到的情況——新確診與既往確診患者的良好組合——我們對於未來更多患者的可見度非常有信心。

Kambiz Yazdi, BTIG.

Kambiz Yazdi，BTIG。

Hi, team. Thank you so much for the questions. TAP revenue growth was quite robust. Is France driving that growth or other countries? And how should we think about geographic composition of the EAP as a leading indicator for where commercial demand may concentrate post-EU approval?

嗨，團隊。非常感謝你們回答問題。TAP 營收成長相當強勁。是法國在帶動這個成長，還是其他國家？另外，我們應該如何看待 EAP 的地理組成，作為歐盟核准後商業需求可能集中在哪些地區的領先指標？

Thank you for the question and welcome to the analyst coverage. As we reported, 122 patients enrolled in Q1 2026. This really encompasses multiple access programs we have and multiple territories. In Europe, we've had outstanding for quite some time our French EAP experience, which we've previously guided and consistently receive about $10 million net per year now that our patient base has stabilized in that territory, and that was per year.

謝謝你的提問，也歡迎你開始覆蓋我們。如我們所報告，2026 年第一季共納入 122 名病患。這實際上涵蓋了我們多個可及性計畫以及多個地區。在歐洲，我們的法國 EAP 經驗已經非常突出一段時間了；我們先前也曾給出指引，且在該地區病患基礎穩定後，目前每年持續帶來約 1,000 萬美元的淨額，而這是按年度計算。

Today, we have new markets that are coming on, and that variability in ordering pattern and the rates of new enrollments and the reality is that we've got both compassionate use as well as named patient reimbursement. All of these new attributes lead to the variability that we continuously guide towards. It's early. We have new distributors.

目前，我們有新的市場正在加入，而訂購模式的變動、新增納入的速度，以及現實情況是：我們同時有同情用藥（compassionate use）以及具名病患給付（named patient reimbursement）。這些新的特性都導致了我們持續在指引中所提到的波動性。現在還早。我們也有新的經銷商。

We're really pleased, though, with our new distributors and our and actually all of our distributors, and how fast they're able to be able to get the named patient requests for individual patients, then drive that back through to being able to get through our system and the product to patients in these territories. So we're really pleased with the continued inbound in these markets. And our goal is to really expand access to as many markets as we can while still focusing on our key territories in the US and expanding the diagnosis and treatment along with our global expansion, as i.e., Europe, and the EMA as well.

不過，我們對新的經銷商、以及其實對我們所有的經銷商都非常滿意；他們能夠很快地為個別病患取得具名病患申請，並將流程回傳推進，使其能夠通過我們的系統，讓產品送達這些地區的病患手中。因此，我們對這些市場持續增加的主動洽詢（inbound）感到非常滿意。我們的目標是在持續聚焦美國關鍵地區、擴大診斷與治療的同時，盡可能把可及性擴展到更多市場，並配合我們的全球擴張，例如歐洲，以及 EMA 相關進展。

And then maybe quickly as a follow-up, can you share with us any quantitative milestones or leading indicators you're tracking internally around your bespoke AI-driven patient identification and genetic testing efforts in identifying newly diagnosed NPC patients?

另外快速追問一下：你們能否分享一些量化的里程碑或領先指標？你們內部在追蹤哪些指標，用於衡量你們量身打造、AI 驅動的病患識別與基因檢測工作，在找出新診斷 NPC 病患方面的成效？

Well, let me ask Josh to work on some of those strategies that are starting to really give us confidence in the TAM.

嗯，我請 Josh 來談談其中一些策略；這些策略正開始讓我們對 TAM 更有信心。

Yeah. We monitor and measure a number of things which will remain internal and metrics that we use just internally. We are measuring how many patients are we finding that are newly diagnosed. We are looking at how many new prescribers we are able to bring into the mix as well and other interesting dynamics about the patient's journey. Perhaps any other treatments that they may have been on, the other diagnoses and other symptoms, all of that is tracked as we look to really understand as much as we can about these patients.

好的。我們會監測並衡量許多項目；其中一些會維持為內部資訊，屬於我們僅供內部使用的指標。我們在衡量我們找到多少新近被診斷的病患。我們也在看我們能把多少新的處方醫師納入其中，以及病患旅程中的其他有趣動態。例如他們可能曾使用過的其他治療、其他診斷與其他症狀；我們在努力盡可能理解這些病患時，這些都會被追蹤。

This program is working extremely well, and it's allowing us to look at those patients who we know are diagnosed with NPC and find like patients who have not yet been diagnosed and be able to work with clinicians in accelerating their diagnosis and treatment.

這個計畫運作得非常好；它讓我們能夠針對那些我們已知被診斷為 NPC 的病患，去找出尚未被診斷、但相似的病患，並與臨床醫師合作，加速他們的診斷與治療。

Yeah, let me add a little bit to that. I think it's important because we talk about the heterogeneity of the disease. The patient journey in NPC, unlike, a lot -- sorry, I should say not unlike a lot of other rare diseases, it is you know one patient, you know one patient. We're talking about children, and the primarily, primary data that has been developed over many, many years in NPC has been really child-based. In our experience in the Expanded Access Program in the US, we built that out, and we saw that it was about 50% of the patients that were children and 50% of the patients that were adults. In our real-world experience moving forward, we still see that.

我再補充一點。我認為這很重要，因為我們談到疾病的異質性。NPC 的病患旅程——不像、呃，抱歉，我應該說其實和許多其他罕見疾病並無二致——就是「你認識一位病患，就只認識那一位病患」。我們談的是兒童，而且多年來 NPC 所建立的主要資料，確實多以兒童為主。依我們在美國擴大可及性計畫（Expanded Access Program）的經驗，我們建立並擴展該計畫後看到，約 50% 的病患是兒童、50% 是成人。在我們後續的真實世界經驗中，我們仍然看到相同的情況。

Now that means that we have to continue to learn about the journey of the patient, that is the adult patient and understand how we can then make the tools and continue to evolve our commercial strategy and educate physicians that lay and present with NPC. So this is early in our launch. We're really pleased with the 170 total enrollments, the 50% children and adults. We're still learning a lot about what it means for adult patients.

這意味著我們必須持續學習病患的旅程——也就是成人病患——並理解我們如何打造工具、持續演進我們的商業策略，並教育那些會遇到並呈現 NPC 病例的醫師。因此，這在我們上市初期仍屬早期階段。我們對總計 170 位納入感到非常滿意，其中兒童與成人各占 50%。我們仍在大量學習這對成人病患意味著什麼。

Thank you so much. I'll hop back in queue.

非常感謝。我回到提問隊列。

Sumant Kulkarni, Canaccord.

Sumant Kulkarni，Canaccord。

Good afternoon, and thanks for taking our questions. I have two, one on MIPLYFFA, one on celiprolol. You mentioned that MIPLYFFA is now at almost 50% share of the diagnosed and treated patients in the US. What does your competitive intelligence tell you about your share of the competitor Akynzeo in the remainder and your ability to gain further share in this 300 to 350 patients? And do you know of any cases where payers are allowing concurrent use of both MIPLYFFA and Akynzeo in the same patient?

午安，謝謝你們回答我們的問題。我有兩個問題，一個關於 MIPLYFFA，一個關於 celiprolol。你們提到 MIPLYFFA 目前在美國已診斷且接受治療的病患中，市占接近 50%。你們的競品情報顯示，在其餘病患中競爭對手 Akynzeo 的市占如何？以及你們在這 300 到 350 名病患中進一步提升市占的能力如何？另外，你們是否知道有任何案例是付款方允許同一位病患同時使用 MIPLYFFA 與 Akynzeo？

Thanks, Sumant. We'll take one question at a time here in regards to the MIPLYFFA question. You are correct. And I'll ask Josh to double-click on this a little. You are correct that also based on the treatment guidelines that were just recently established, that a lot of patients, and I can't give you the exact amount, but a lot of patients, because of the complexity of the disease as well as the symptomatology of the disease, are on multiple therapies.

謝謝你，Sumant。我們先一次回答一個問題，先談 MIPLYFFA 的部分。你說得沒錯。我也請 Josh 再更深入說明一下。你也說得對：同時基於最近才剛建立的治療指引，許多病患——我無法給出確切數字——因為疾病的複雜性以及其症狀表現，會使用多種治療。

We're actually seeing success in getting those patients on miglustat and MIPLYFFA and other therapies to be able to get them covered from a commercial perspective. These guidelines, I think, are going to continue to reinforce that combination therapy should be considered for NPC patients. We believe that that bodes really well for us. Our label is in combination with miglustat. We see that's where our clinical differentiation is in terms of disease modification, halting the progression of the disease, and the durable effects that we see. So we see this as a real positive.

我們其實看到一些成功案例，能讓這些病患同時使用 miglustat 與 MIPLYFFA 以及其他療法，並在商業給付的角度取得保障。我認為這些指引將持續強化：對 NPC 病患應考慮合併治療。我們相信這對我們非常有利。我們的適應症標示是與 miglustat 合併使用。我們認為這正是我們在臨床上的差異化所在，包括疾病修飾、阻止疾病進展，以及我們所看到的持久效果。因此我們把這視為一個非常正面的訊號。

I'll ask Josh maybe to talk a little bit about what he's seeing on the ground.

我請 Josh 也許談談他在第一線看到的情況。

Yeah. Just to add a little bit more color there. We have been seeing in clinical practice that clinicians want to be able to treat patients with as many different modalities and to approach the treatment from as many different angles as possible. And so we've seen this combination therapy for some time, and now that opinion is really reinforced by the treatment guidelines.

好的。我再補充一些背景。我們在臨床實務中一直看到，臨床醫師希望能用盡可能多的不同治療方式，並從盡可能多的角度來處理治療。因此，我們已經看到這種合併治療一段時間了，而現在治療指引也進一步強化了這樣的觀點。

What this means is that the 50% of diagnosed patients that we're treating now, some of them very likely may be on other therapies. And this is not an either/or marketplace. This is really an end marketplace. We're seeing that continuing to grow. We're also seeing that coverage is continuing to exist for patients. We have 69% of total covered lives today. Those who are not able to get that through direct formulary, we're able to get coverage even for combination therapy through medical exception pathways.

這表示，我們目前正在治療的已確診患者中有 50%，其中一些人很可能也正在使用其他療法。而這不是一個非此即彼的市場。這其實是一個「並存」的市場。我們看到這種情況持續成長。我們也看到患者的給付覆蓋仍在持續存在。目前我們覆蓋了 69% 的總投保人群。對於無法透過直接處方集取得給付的人，我們也能透過醫療例外途徑，甚至為合併療法取得給付。

Got it. On celiprolol, we know you plan to meet with the FDA again in the second half of this year, what are some of the specific options that you might have on the table in terms of your ability to move faster to bring this product to vEDS patients?

了解。關於 celiprolol，我們知道你們計畫在今年下半年再次與 FDA 會面；就你們加速把這個產品帶給 vEDS 患者的能力而言，目前桌面上可能有哪些具體選項？

Yeah. Thanks, Sumat. We mentioned in our last call that we had a Type C meeting in Q1, and that we're exploring pathways to accelerate the clinical development of celiprolol. I would categorize the conversations as constructive, also informative.

是的。謝謝，Sumat。我們在上次電話會議中提到，我們在第一季進行了一場 Type C 會議，並且正在探索加速 celiprolol 臨床開發的途徑。我會把這些對話形容為具建設性，也很有資訊量。

We're now -- this is the early stages. We're now in the process of preparing for the follow-up meeting in the second half of 2026. And quite frankly, it's too early for us to tell you what strategies we're going at. The reality is that we're going in two directions. One, to continue to boost enrollment activities, and the other is to accelerate the clinical development through additional pathways with additional data.

我們現在——這還在早期階段。我們正在準備於 2026 年下半年舉行的後續會議。坦白說，現在還太早，無法告訴各位我們會採取哪些策略。現實是我們正朝兩個方向推進。第一，持續強化受試者招募活動；第二，透過額外途徑與更多數據來加速臨床開發。

Eddie Hickman, Guggenheim Securities.

Eddie Hickman，Guggenheim Securities。

Hey, guys. Thanks for taking the question, and congratulations on all the progress. Apologies if this has already been asked. I'm jumping between a few calls. Really nice performance outside the US as well. So I'm wondering if you can give an update on the type of questions that you got from the type 100-day, day 120 day, and are they clinical CMC or more commercial? And then what's the sort of timeline for that updated CHMP opinion? Thanks.

嗨，各位。謝謝讓我提問，也恭喜你們取得所有進展。如果這題已經有人問過先說聲抱歉。我在幾場電話會議之間切換。美國以外的表現也非常亮眼。所以我想請你們更新一下：你們在 100 天、120 天這類（審查）節點收到的問題大概是什麼類型？是臨床、CMC，還是偏商業面？另外，更新後的 CHMP 意見大概的時間表是什麼？謝謝。

Yeah. Thank you, Eddie. And you have been the first to ask the question in regards to our marketing authorization application. As we mentioned in the prepared remarks, we did respond to the 120-day list of questions within the appropriate clock stop period. We provided the standard responses that are there. I won't get into the specifics of what it is the questions were or how we addressed them. But what I would continue to say is that since we've submitted a new application to the agency, we have not actually seen any new questions that we did not see out of the previous European submission and the FDA submission that we were able to then provide.

是的。謝謝你，Eddie。而且你是第一位就我們的上市許可申請（MAA）提出這個問題的人。如同我們在事先準備的講稿中提到的，我們已在適當的停鐘期內回覆了 120 天問題清單。我們提供了標準的回覆。我不會深入說明問題的具體內容或我們如何回應。但我會繼續強調的是，自從我們向主管機關提交新的申請以來，我們其實沒有看到任何我們在先前的歐洲送件與 FDA 送件中未曾見過、且我們無法提供回覆的新問題。

So the substantial amount of data that we have now, and the robustness of the package. And as a reminder, the totality of the data is over 270 treated patients, including data from our Phase 2/3 study, the OL, the open label extension study that's got five years of data, our EAP, that's got over five years of data, along with the pediatric sub study. So it's new data and the engagement is fairly standard. So now we're continuing to engage along the path, and we look forward to our next engagement with the European regulators.

因此，我們目前擁有的大量數據，以及整體資料包的扎實程度。提醒一下，整體數據涵蓋超過 270 名接受治療的患者，包括我們的第 2/3 期研究、OL（開放標籤）、具有五年數據的開放標籤延伸研究、具有超過五年數據的 EAP（擴大使用計畫），以及兒科子研究。所以這是新的數據，而互動也相當標準。目前我們會沿著既定路徑持續互動，也期待下一次與歐洲監管機構的交流。

Great. Appreciate that color.

很好。謝謝提供這些補充說明。

Jason Butler, Citizens.

Jason Butler，Citizens。

Hey, thanks for taking the question, two for me. Can you talk about the profile of the newly diagnosed patients that you're seeing? And again, speak to the heterogeneity here. How early in the disease are they versus patients that may have had the disease for a long time, but just the diagnosis is delayed? And then second question, you mentioned that the inventory was below the lower end of the range, third range at the end of the quarter, has that reversed so far in 2Q?

嗨，謝謝讓我提問，我有兩個問題。你們能談談目前看到的新確診患者的樣貌嗎？也請再談談這裡的異質性。他們是在疾病多早期就被診斷出來，還是有些患者其實已經罹病很久、只是診斷被延誤？第二個問題，你們提到本季末庫存低於區間下緣、低於目標區間；到目前為止在第二季是否已經反轉？

Yeah. Thank you, Jason. Let me start with the first question, the profile of the newly diagnosed patients. I know I say this a lot, but when you know one patient, you know one patient. Because of this heterogeneity of the disease, it's really important. One patient may present with a primary symptom that's an epilepsy.

是的。謝謝你，Jason。我先從第一個問題開始：新確診患者的樣貌。我知道我常這麼說，但當你認識一位患者，你就只是認識那一位患者。由於這個疾病的異質性，這點真的很重要。有的患者可能以癲癇作為主要症狀表現。

Another may present with gait instability. Another may present with cognitive challenges. And because of that, we're continuing to refine our, as Josh talked about, the predictive modeling, claims data, and all the things that we're doing, but we're learning along the way. So every time we get a new patient, it's further informing how we go at and try to continue to expand.

另一位可能表現為步態不穩。另一位可能表現為認知方面的挑戰。也因此，我們持續在精進——如 Josh 所談到的——預測模型、理賠數據，以及我們正在做的所有事情，同時也在過程中學習。所以每當我們接觸到一位新患者，都會進一步幫助我們了解該如何推進並持續擴大。

And as I mentioned, these 300 to 350 patients, we have a really good line of sight to those diagnosed patients already. We've talked about the prevalence of the 900 in the United States. We now have a lot more confidence that that 300, 350, and the 900, we're somewhere in between those according to the TAM. And what we see is the addressable patient population. So let me ask Josh to talk a little bit about some of the characteristics we see, but the confidence is getting, is growing every time we see newly diagnosed patients.

如我提到的，這 300 到 350 名患者，我們對於那些已確診患者有非常清楚的掌握。我們也談過美國約 900 名的盛行人數。我們現在更有信心，依照 TAM（總可服務市場）來看，300、350 與 900 之間，我們大概落在中間某個位置。而這就是我們看到的可觸及患者族群。所以我請 Josh 再談談我們看到的一些特徵；但每當看到新確診患者，我們的信心都在提升、也在成長。

Just to bring this to life a little bit, we recently saw the diagnosis of a toddler who had an enlarged spleen and then went through genetic testing and was confirmed to have NPC. On the other end of the spectrum, there was an adult who had been misdiagnosed with MS for years and then changed physicians who happened to be aware of NPC and did a genetic testing and confirmed that it was NPC.

為了讓大家更具體理解，我們最近看到一位幼兒被診斷：他有脾臟腫大，之後接受基因檢測，確認罹患 NPC。在光譜的另一端，有一位成人多年來被誤診為 MS，後來更換醫師，而新醫師剛好了解 NPC，於是進行基因檢測並確認其實是 NPC。

So it really runs the gamut, but there are some similarities and some characteristics both in terms of the symptoms as well as the journey that these patients go on that we're able to really learn from. And this is feeding our disease awareness campaign that we're continuing to educate physicians. We have a strong presence at medical conferences. We publish a lot of data on this, as well as the strong commercial efforts that we're putting in place.

所以範圍非常廣，但在症狀以及這些患者所經歷的就醫旅程方面，確實存在一些相似之處與特徵，讓我們能夠從中學到很多。而這也在支持我們的疾病認知宣導活動，我們持續教育醫師。我們在醫學會議上有很強的能見度。我們也發表了大量相關數據，同時也在建立強而有力的商業推進工作。

And Jason, let me hand off to Justin to keep him going here this evening on the inventory question.

Jason，讓我把話題交給 Justin，讓他今晚在庫存問題上繼續說明。

Yes, thanks, Jason. The way the Q1 delivery calendar fell, we just had one less shipment of MIPLYFFA in the US than typical. And so as a result, our inventory in the channel was on less than the low end of our targeted range. And so as a result, we do expect this to fall back within our targeted range by the end of the second quarter.

是的，謝謝你，Jason。由於第一季的出貨日曆安排，我們在美國的 MIPLYFFA 出貨次數比一般情況少了一次。因此，我們在通路端的庫存低於我們目標區間的下緣。因此，我們預期到第二季末會回到我們的目標區間內。

Great. Thanks for taking the questions.

很好。謝謝回答問題。

(Operator Instructions) Brandon Folkes, H.C. Wainwright.

(接線員指示) Brandon Folkes，H.C. Wainwright。

Hi. Thanks for taking my questions, and congrats on all the progress. maybe just two from me. Any color on the time from submitting an enrollment form to getting on product and how that is trending at this stage of the launch? Secondly, is there a difference in net revenue realized per patient if it goes through a medical exception versus a patient whose insurance falls into the 69% of covered lives currently? Thank you.

嗨。謝謝回答我的問題，也恭喜你們取得所有進展。我這邊可能兩個問題。能否分享一下，從提交入組表格到開始使用產品所需的時間，以及在目前上市階段這個趨勢如何？第二，若是透過醫療例外（medical exception）與目前保險涵蓋生命數（covered lives）69%那部分患者相比，每位患者實現的淨營收是否有差異？謝謝。

Thanks, Brandon. Let me ask Josh to talk a little bit about the enrollment numbers that we're seeing now.

謝謝你，Brandon。我請 Josh 談一下我們目前看到的入組數據。

Yeah. I think your question was really around the time from an enrollment comes in until once a patient receives therapy. And it's varied. It is largely dependent on the payer type, whether it's government or whether it's commercial.

好的。我想你的問題主要是從收到入組申請到患者實際開始接受治療之間的時間。這段時間有所不同。主要取決於付款方類型，是政府保險還是商業保險。

We are seeing, in Q1, we had a number of patients who went through a reauthorization process, which is very typical at the beginning of the year, as patients might either change plans or plans might change their policies. And so that had an impact on the first quarter, and we're working through those reauthorizations. So it's a little difficult to give you kind of what the average or the standard is just because of that reauthorizations that took place in the first quarter.

我們在第一季看到有不少患者需要走重新授權（reauthorization）流程，這在年初非常常見，因為患者可能更換方案，或方案可能調整其政策。因此這對第一季造成了影響，而我們正在處理這些重新授權。所以要給出平均值或標準時間有點困難，因為第一季發生了這些重新授權。

Your second question, this was about net revenue, and I'm not sure that I quite got that. Can you repeat that question for us?

你的第二個問題是關於淨營收，但我不太確定我是否完全理解。你可以再重複一次這個問題嗎？

Sure. I guess, what's the priority to grow the 69% of covered lives? Given your success under medical exceptions, is there a difference in net realized revenue per patient that goes through insurance versus a medical exception?

當然。我想問的是，擴大目前69%涵蓋生命數的優先順序是什麼？鑑於你們在醫療例外方面的成功，透過保險理賠與透過醫療例外的患者，每位患者實現的淨營收是否有差異？

Yeah, no, that's a great question. I'll ask Josh to opine. The 69% of covered lives, it allows for you to be on a formulary. Doesn't necessarily have a preferred position on the formulary, and it does speed up the process. A lot of our education that's gone to payers so far has really allowed us to be able to educate on the clinical benefits with the medical directors of these varying plans and achieve what we believe is a really good, covered life plan.

是的，這是個很好的問題。我請 Josh 發表看法。69% 的涵蓋生命數讓你能被納入處方集（formulary）。但不一定在處方集中具有優先地位，不過確實能加快流程。到目前為止，我們對付款方所做的許多教育，確實讓我們能與各方案的醫療主管溝通臨床效益，並達成我們認為相當不錯的涵蓋生命數方案。

The important component of the question I think you just asked, which is maybe get into the potential for gross net as well, we do not contract currently today. So it's standard government discounts along with distribution margins and the like. So the net price hasn't really changed except for the variability in gross net on a quarter-over-quarter basis.

我認為你剛才問到的重點，可能也涉及毛利到淨額（gross-to-net）的潛在影響；我們目前並沒有簽訂合約。因此就是標準的政府折扣，加上配送通路毛利等。所以淨價基本上沒有改變，除了每季之間毛利到淨額的變動所帶來的差異。

Yeah. And I think you're asking a very salient question around the 69 and our intent to grow that. We absolutely do, and we are making steady progress in that area, largely by talking about the clinical differentiation of MIPLYFFA. The guidelines certainly will help those discussions as well.

是的。我認為你問到一個非常關鍵的問題：關於69%以及我們擴大它的意圖。我們確實有這個目標，而且在這方面持續穩步推進，主要是透過強調 MIPLYFFA 的臨床差異化。相關指引也一定會對這些討論有所幫助。

As you point out, it really doesn't impact the overall ability for a patient to receive MIPLYFFA because every plan has a medical exception pathway. What it does is it reduces a little bit of the time. It reduces some of the burden. We have a very robust patient services resources that we provide to help patients and offices navigate this. And so our goal is to make MIPLYFFA as accessible to patients as possible, and we do that both through increasing the covered lives but also providing these patient services.

如你所指出，這其實不會影響患者整體取得 MIPLYFFA 的能力，因為每個方案都有醫療例外的途徑。它的作用是稍微縮短時間。也能減少一些負擔。我們提供非常完善的患者服務資源，協助患者與診所端處理並順利完成這些流程。因此，我們的目標是讓 MIPLYFFA 盡可能容易取得；我們一方面透過提高涵蓋生命數來達成，同時也透過提供這些患者服務來達成。

Great. Thank you very much, and congrats again on all the success.

很好。非常感謝，也再次恭喜你們取得所有成功。

Thank you. This concludes the Q&A portion of today's call. I will now turn the call back to Neil for any additional or closing remarks.

謝謝。以上為今天電話會議的問答環節。我現在把電話交回給 Neil，請他做任何補充或結語。

Thank you for joining the call today. We look forward to keeping you appraise of our future progress. Have a wonderful evening.

感謝各位今天加入電話會議。我們期待持續向各位更新我們未來的進展。祝各位今晚愉快。

Thank you. This brings us to the end of today's meeting. We appreciate your time and participation. You may now disconnect.

謝謝。今天的會議到此結束。感謝各位撥冗與會。各位現在可以掛線。