Amylyx Pharmaceuticals, Inc. (AMLX) 2026 Q3 法說會逐字稿

Good morning. My name is Raya, and I will be your conference operator today. At this time, I would like to welcome everyone to the Amalyx Pharmaceuticals Third Quarter 2026 Earnings Call. All participants will be in listen-only mode. After today's presentation, there will be an opportunity to ask questions. To ask a question, please press star 1 on your telephone keypad. To withdraw your question, please press star 1 again. Please be advised that this call is being recorded at the company's request. I would now like to turn the call over to Courtney O'Leary, Director of Investors Relations.

早安。我叫 Raya，今天將擔任各位的電話會議接線員。此刻，我謹代表公司歡迎各位參加 Amalyx Pharmaceuticals 2026 年第三季財報電話會議。所有與會者將以僅收聽模式參與。在今天的簡報結束後，將開放提問時間。如欲提問，請在電話鍵盤上按星號 1。如欲取消提問，請再次按星號 1。請注意，應公司要求，本次電話會議將被錄音。現在我想把電話交給投資人關係總監 Courtney O'Leary。

Thank you. Please Good morning, and thank you all for joining us today to discuss our third quarter 2026 financial results and business updates.

謝謝。各位早安，感謝大家今天加入我們，一同討論 2026 年第三季財務結果與業務最新進展。

With me on the call today are Josh Cohen and Justin Klee, our co-CEOs Dr. Kamil. Pedrojian, our Chief Medical Officer, Dan Monahan, our Chief Commercial Officer, and Jim Freides, our Chief Financial Officer.

今天與我一同出席電話會議的有我們的共同執行長 Josh Cohen 與 Justin Klee、首席醫療長 Kamil Pedrojian 博士、首席商務長 Dan Monahan，以及首席財務長 Jim Freides。

Before we begin, I would like to remind everyone that any statements you make or information presented on this call that are not historical facts are forward-looking statements that are based on our. Current beliefs, plans, and expectations, and are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1,995.

在開始之前，我想提醒各位，本次電話會議中所作的任何陳述或所呈現的資訊，凡非歷史事實者，均屬前瞻性陳述，係基於我們的目前信念、計畫與預期，並依據 1995 年《私人證券訴訟改革法案》的安全港條款作出。

These statements include, but are not limited to, our expectations with respect to Avexitide, AMX 35, AMX 114, and AMX 318 statements regarding regulatory and clinical development.

這些陳述包括但不限於：我們對 Avexitide、AMX 35、AMX 114 與 AMX 318 的預期，以及有關法規與臨床開發的陳述。

The impact thereof, and the expected timing thereof, and statements regarding our cash runway. Actual events and results could differ materially from those expected or implied by any forward-looking statements. You are cautioned not to place any undue reliance on these forward-looking statements and Amalyx disclaims any obligation to update such statements unless required by law.

其影響及其預期時程，以及關於我們現金可支撐期間的陳述。實際事件與結果可能與任何前瞻性陳述所預期或暗示者存在重大差異。敬請各位勿過度依賴這些前瞻性陳述；除非法律要求，Amalyx 不承擔更新此等陳述之任何義務。

Now, I will turn the call over to Justin.

現在，我把電話交給 Justin。

Thank you, and good morning, everyone.

謝謝，各位早安。

It has been a momentous period for Amalyx. In the third quarter, we reported positive top-line results from the Phase 3 lucidity trial of Vivexetide, an investigational first-in-class GLT-1 receptor antagonist in participants with post-bariatric hypoglycemia, or PBH. Following Roux-en-Y gastric bypass surgery.

對 Amalyx 而言，這是一段意義重大的時期。在第三季，我們公布了 Vivexetide 的第 3 期 Lucidity 試驗正向主要結果；Vivexetide 是一款研究中、同類首創（first-in-class）的 GLT-1 受體拮抗劑，於接受胃繞道手術後低血糖（post-bariatric hypoglycemia，PBH）受試者中進行評估，其手術類型為 Roux-en-Y 胃繞道手術。

Fueled by the strength of this data, we completed an equity offering to support our launch readiness efforts to potentially deliver the first ever approved therapy for PBH in 2027.

在這些數據的強勁表現帶動下，我們完成了一次股權募資，以支持我們的上市準備工作，目標是在 2027 年有機會提供 PBH 史上第一個獲核准的治療方案。

Our pipeline development efforts with Evexatide and our other programs remain strong as well, which Josh will talk about later in the call.

我們在 Evexatide 及其他專案上的研發推進也同樣穩健，Josh 稍後會在電話會議中進一步說明。

As we reflect back on what has already been a milestone year for Analyx, we are proud of our team's strong execution across the three key objectives for Evexatide that we outlined in the first quarter.

回顧這個對 Analyx 而言已是里程碑的一年，我們為團隊在第一季所提出、關於 Evexatide 的三項關鍵目標上展現的強勁執行力感到自豪。

In the third quarter, we continued to make substantial progress toward achieving each of them, and we now look ahead to another potential milestone year in 2027.

在第三季，我們持續在達成各項目標方面取得顯著進展，並展望 2027 年可能成為另一個里程碑之年。

Our first objective for 2026 was to execute on the Phase 3 lucidity trial of avectitide in people with PDH following Roux-en-Y gastric bypass surgery.

我們 2026 年的第一項目標，是在接受 Roux-en-Y 胃繞道手術後的 PDH 患者中，完成 avectitide 的第 3 期 Lucidity 試驗執行。

In August, we reported positive top-line results from the pivotal Phase 3 lucidity trial.

8 月，我們公布了關鍵性第 3 期 Lucidity 試驗的正向主要結果。

Ovexatide demonstrated consistent, highly statistically significant, clinically meaningful reductions in hypoglycemic events across all endpoints with a favorable safety profile.

Ovexatide 在所有終點上均顯示一致、具高度統計顯著性且具臨床意義的低血糖事件降低，且安全性概況良好。

We were pleased to learn that these data were accepted as a late-breaking oral presentation at the upcoming Obesity Week 2026 Annual Meeting on November 15.

我們很高興得知，這些數據已獲選為即將於 11 月 15 日舉行的 Obesity Week 2026 年會之「最新突破（late-breaking）」口頭報告。

As we had spent more time engaging with the PBH community and sharing the top-line data. Our conviction in the significant unmet need and market opportunity has grown, with many physicians reacting positively to the strength of the Lucidity data.

隨著我們投入更多時間與 PBH 社群互動並分享主要結果數據，我們對於顯著未被滿足的醫療需求與市場機會的信心日益提升，許多醫師也對 Lucidity 數據的強度給予正面回應。

Our second objective was to execute on our NDA submission efforts.

我們的第二項目標，是推進 NDA 申請提交的相關工作。

Our team has been actively preparing the submission, and we remain on track to submit our NDA to the FDA by the end of this year.

我們的團隊一直積極準備申請資料，並仍按計畫在今年年底前向 FDA 提交我們的 NDA。

Our third objective was to execute on our launch readiness efforts for Vexitide, anticipating a potential 2027 approval and launch as the first ever approved treatment for PBH.

我們的第三項目標，是推進 Vexitide 的上市準備工作，預期其可能於 2027 年獲批並上市，成為 PBH 史上第一個獲核准的治療。

We have an experienced commercial and medical affairs leadership team in place, and our efforts this year have given us a strong understanding of the patient journey for people with PBH and the healthcare professionals who care for them building on the momentum generated from the Lucidity results and supported by the additional capital we recently raised, we are investing in the capabilities, infrastructure, and team needed to support a successful launch.

我們已建立一支經驗豐富的商業與醫藥事務領導團隊；今年的工作讓我們對 PBH 患者的就醫旅程，以及照護他們的醫療專業人員有了深入理解。在 Lucidity 結果所帶動的動能之上，並在我們近期募集的額外資金支持下，我們正投資於成功上市所需的能力、基礎設施與團隊。

We believe we have the opportunity to potentially change practice in PBH. A debilitating disease which to date has had no approved therapies.

我們相信，我們有機會在 PBH 的臨床實務上帶來改變。這是一種使人衰弱的疾病，迄今尚無任何獲核准的治療。

It is this opportunity, informed by the stories of the many people with TBH we have heard from, that will continue to drive our team to execute on our objectives with precision and urgency.

正是這個機會——並結合我們所聽到的許多 TBH 患者故事——將持續驅動我們的團隊以精準與緊迫感來達成各項目標。

We look forward to an exciting year-end and another potential transformational year in 2027.

我們期待精彩的年末，以及 2027 年可能再次成為具轉型意義的一年。

With that, I'll turn the call over to Camille to discuss the lucidity results in more detail, our NDA submission, and our field medical team efforts.

接下來，我把電話交給 Camille，請她更詳細說明 Lucidity 結果、我們的 NDA 提交，以及我們外勤醫療團隊的工作。

Thank you, Justin, and good morning, all.

謝謝你，Justin，各位早安。

TBH is a chronic metabolic condition characterized by recurrent and often debilitating hypoglycemia that can lead to cognitive impairment, confusion, and in some cases, seizures or loss of consciousness.

TBH 是一種慢性代謝性疾病，其特徵為反覆且往往使人衰弱的低血糖，可能導致認知受損、意識混亂，並在某些情況下引發癲癇發作或失去意識。

The condition can have a profound impact on daily life, with patients. Often structuring their lives around the constant risk of a hypoglycemic event and facing challenges maintaining their independence, employment, and family responsibilities.

此疾病會對日常生活造成深遠影響，患者往往必須圍繞著低血糖事件的持續風險來安排生活，並在維持獨立性、就業與家庭責任方面面臨挑戰。

In one study. More than 90% of individuals with PDH describe themselves as living with a disability, underscoring the profound impact the condition can have on quality of life.

在一項研究中，超過 90% 的 PDH 患者自述自己是帶著障礙生活，凸顯該疾病對生活品質可能造成的深刻影響。

Endocrinologists consistently describe PDH as one of the most challenging conditions they manage.

內分泌科醫師一貫將 PDH 形容為他們所管理的最具挑戰性的疾病之一。

Despite this burden, there are currently no FDA-approved therapies.

儘管負擔沉重，目前仍沒有任何 FDA 核准的治療。

Lucidity met its pre-specified primary endpoint, demonstrating a 55% reduction in the composite rate of level 2 and level 3 hypoglycemic events compared to placebo with a p-value of 0.000003.

Lucidity 達成其預先指定的主要終點：與安慰劑相比，第 2 級與第 3 級低血糖事件的複合發生率降低 55%，p 值為 0.000003。

The study also met all secondary endpoints with consistent, highly statistically significant and clinically meaningful reductions across level 2 events measured by self-monitoring of blood glucose, level 2 events measured by continuous glucose monitoring, and independently adjudicated level 3 events.

本研究亦達成所有次要終點：在以自我監測血糖（SMBG）衡量的第 2 級事件、以連續血糖監測（CGM）衡量的第 2 級事件，以及經獨立裁定的第 3 級事件方面，均呈現一致、具高度統計顯著性且具臨床意義的降低。

Importantly, for people living with PDH, preventing even one level 2 or level 3 hypoglycemic event can have a meaningful impact on daily life.

重要的是，對於 PDH 患者而言，即使僅能預防一次第 2 級或第 3 級低血糖事件，也可能對日常生活帶來具意義的改善。

Furthermore, the American Diabetes Association recognizes hypoglycemia as a potential medical emergency.

此外，美國糖尿病協會將低血糖視為可能的醫療緊急狀況。

Against this backdrop, we believe the consistency, statistical strength, and clinical meaningfulness of the lucidity results show its potential.

在此背景下，我們相信 Lucidity 結果的一致性、統計強度與臨床意義，顯示其潛力。

Lucidity is the sixth clinical trial of avexatide in PDH, and all of those trials generated statistically significant results.

Lucidity 是 avexatide 在 PDH 的第六項臨床試驗，而所有這些試驗都產生了具統計顯著性的結果。

In addition, Lucidity is the largest and longest study of avexatide conducted to date.

此外，Lucidity 是迄今為止所進行的 avexatide 規模最大且時間最長的研究。

Alongside the efficacy results, avexatide demonstrated a favorable safety profile consistent with that observed across the clinical development program to date.

除療效結果外，avexatide 亦展現出良好的安全性概況，且與迄今臨床開發計畫中所觀察到的結果一致。

Avexatide was generally well tolerated, the majority of adverse events were mild to moderate, and there were no serious adverse events deemed related to avexatide treatment.

Avexatide 整體耐受性良好，多數不良事件為輕度至中度，且未有任何被認定與 avexatide 治療相關的嚴重不良事件。

More than 90% of the participants completed the 16-week double-blind treatment period, and all eligible participants entered the ongoing open-label extension portion of the trial.

超過 90% 的受試者完成了 16 週雙盲治療期，且所有符合資格的受試者皆進入試驗目前進行中的開放標籤延伸階段。

Additionally, our expanded access program is ongoing.

此外，我們的擴大使用計畫仍在進行中。

Our field medical team has been focused on actively facilitating scientific exchange around the Lucidity data.

我們的醫學外勤團隊一直專注於積極促進圍繞 Lucidity 數據的科學交流。

They have established a strong presence, engaging directly with a substantial number of healthcare professionals to answer questions about the data and gain insights regarding the unmet need and patient journey in PDH.

他們已建立強而有力的能見度，直接與大量醫療專業人員互動，以回答關於數據的問題，並深入了解 PDH 中尚未被滿足的需求與患者就醫旅程。

Across these interactions, they continue to hear that awareness of PDH among specialists is high, recognition of the serious consequences in PDH is growing, and endocrinologists see a significant need for an FDA-approved treatment option, given that there are currently no approved therapies.

在這些互動中，他們持續聽到：專科醫師對 PDH 的認知度很高、對 PDH 嚴重後果的認識正在提升，且由於目前尚無核准療法，內分泌科醫師認為迫切需要一個獲 FDA 核准的治療選項。

These discussions reinforce our belief that avexatide has the potential to be the first FDA-approved treatment to address a significant unmet need in PBH.

這些討論強化了我們的信念：avexatide 有潛力成為首個獲 FDA 核准、用以滿足 PBH 重大未滿足需求的治療。

In addition, we are pleased that the lucidity results will be presented to the medical community on November 15th at Obesity Week, results that we believe will continue to demonstrate the consistency and strength of the dataset and the overall potential of avexatide to change practice in PDH.

此外，我們也很高興 Lucidity 的結果將於 11 月 15 日在 Obesity Week 向醫學界發表；我們相信這些結果將持續展現該數據集的一致性與強度，以及 avexatide 在 PDH 中改變臨床實務的整體潛力。

Our team remains focused on executing our planned NDA submission by year-end.

我們的團隊仍專注於在年底前按計畫完成 NDA 申請遞交。

And as a reminder, Avexitide has both breakthrough therapy and orphan drug designations from the FDA.

並提醒各位，Avexitide 已獲 FDA 授予突破性療法與孤兒藥資格認定。

With that, I'll now turn over the call to Dan.

接下來，我把電話交給 Dan。

Thank you, Camille, and good morning, everyone.

謝謝你，Camille，各位早安。

Bolstered by the strength of the Phase 3 Lucidity data and with additional capital, we have transitioned from launch planning to executing on our launch readiness roadmap.

在第三期 Lucidity 數據的強勁表現以及額外資本的支持下，我們已從上市規劃轉為執行上市就緒路線圖。

Our continued engagement with the PBH community has reinforced our belief that this indication represents a significant market opportunity with a substantial unmet need.

我們持續與 PBH 社群互動，進一步強化我們的信念：此適應症代表一個具重大未滿足需求的顯著市場機會。

We are investing in the team and capabilities. Needed to realize the long-term potential of avexitide if approved.

我們正在投資於團隊與能力。以在獲核准的前提下，實現 avexitide 的長期潛力。

As we prepare for a potential launch in 2027, our efforts are focused on three key areas.

在為 2027 年可能的上市做準備之際，我們的工作聚焦於三個關鍵領域。

First, we are applying our growing understanding of the PVH market and the patient journey to inform our go-to-market plans.

第一，我們運用對 PVH 市場與患者就醫旅程日益加深的理解，來制定我們的上市策略（go-to-market）計畫。

This includes our ongoing claims analysis work, additional market research. And direct engagement with healthcare professionals as well as patients.

這包括我們持續進行的理賠資料分析工作、額外的市場研究。以及與醫療專業人員和患者的直接互動。

These efforts help validate the patient population size and provide valuable insights into where patients with PVH are being treated.

這些工作有助於驗證患者族群規模，並提供寶貴洞見，了解 PVH 患者目前在哪些場域接受治療。

Second, we are expanding our disease-state education campaign. Uncover PBH.com with the goal of increasing recognition and understanding of PBH across the broader clinical community.

第二，我們正在擴大疾病狀態教育活動。Uncover PBH.com 的目標是提升更廣泛臨床社群對 PBH 的辨識度與理解。

This campaign has been well received and reached a broad number of endocrinologists, gardiatric surgeons, and people living with PBH.

此活動反應良好，並觸及大量內分泌科醫師、減重手術醫師，以及 PBH 患者。

Third. We are scaling our commercial organization and infrastructure.

第三。我們正在擴大我們的商業組織與基礎設施。

The full commercial leadership team is now in place, and we are making key hires across marketing, market access, patient services, and commercial operations.

完整的商業領導團隊現已到位，我們正於行銷、市場准入、患者服務與商業營運等領域進行關鍵招募。

We are also investing in capabilities needed for a successful launch.

我們也正在投資成功上市所需的能力建置。

More broadly, we continue to see increasing recognition of PBH across the medical community and healthcare system.

更廣泛而言，我們持續看到醫學界與醫療體系對 PBH 的認知正在提升。

Just last month, a PBH-specific ICD-10 code went into effect, which reflects the growing awareness of PBH within the medical community.

就在上個月，一個 PBH 專用的 ICD-10 代碼已正式生效，反映出醫學界對 PBH 日益提升的認知。

We believe PBH represents a significant and underserved market opportunity.

我們相信 PBH 代表一個重要且服務不足的市場機會。

We estimate there are approximately 160,000 people living with PBH in the U.S.

我們估計美國約有 160,000 名 PBH 患者。

Of this population. Approximately 118,000 people were estimated to have received Roux-en-Y gastric bypass surgery, and approximately 48,000 people are estimated to have received sleeve gastrectomy.

在這些人之中。估計約有 118,000 人接受過 Roux-en-Y 胃繞道手術，另約有 48,000 人估計接受過袖狀胃切除術。

Our independent claims analysis and ongoing field engagements to understand the market opportunity continue to support these estimates.

我們的獨立理賠分析以及持續的外勤互動（用以了解市場機會）仍支持上述估計。

In conclusion with compelling Lucidity results and key hires in place, the team is focused on executing launch preparations to potentially commercialize the first approved therapy for PBH in 2027.

總結而言，憑藉具說服力的 Lucidity 結果以及關鍵人員到位，團隊正專注於執行上市準備工作，以期在 2027 年將首個獲核准的 PBH 治療商業化。

And with that, I will now turn the call over to Jim to review our financials.

接下來，我把電話交給 Jim 來回顧我們的財務狀況。

Thanks, Dan. Good morning, everyone.

謝謝，Dan。各位早安。

We had an excellent quarter from a financial point of view.

從財務角度來看，我們本季表現非常出色。

The success of the Lucidity trial has allowed us to move to the next phase of growth, executing on our NDA preparation, scaling our commercial organization and building long-term supply by investing in both our current and a new supplier.

Lucidity 試驗的成功使我們得以進入下一個成長階段，推進 NDA 準備工作、擴大商業組織，並透過同時投資於現有與新供應商來建立長期供應能力。

We're preparing for a large potential market and we're well on our way.

我們正為一個潛在的大型市場做準備，而且進展順利。

Financially, our plans were enhanced by our recent oversubscribed equity offering that gave us net proceeds of $545 million.

在財務方面，我們近期超額認購的股權募資強化了我們的計畫，為我們帶來 5.45 億美元的淨募資款。

That left us with $768 million in cash and cash equivalents at the end of September, compared to $251 million at the end of the second quarter.

因此，截至 9 月底，我們的現金及約當現金為 7.68 億美元，相較於第二季末的 2.51 億美元。

We were thrilled to bring in anchor orders from a number of leading experienced investors in commercial biotech companies.

我們非常高興能獲得多家在商業化生技公司領域具領先且經驗豐富的投資人所下的基石認購。

As we've highlighted on this call, the more work we do in this area, the more excited we get and we believe we're well funded to deliver on our goals and importantly, enter potential commercialization in 2027 from a position of financial strength with the capital necessary to execute on our launch priorities.

如同我們在本次電話會議中所強調的，我們在這方面做得越多，就越感到振奮；我們相信資金充足，能夠達成目標。更重要的是，我們將以財務實力為後盾、具備執行上市優先事項所需資本，於 2027 年以強健的財務狀況進入潛在商業化階段。

So turning now to our results for the quarter, total operating expenses for the quarter were $49.7 million, up 38% from the same period in 2025.

接著談本季業績：本季總營運費用為 4,970 萬美元，較 2025 年同期增加 38%。

Research and development expenses for the quarter were $28.4 million compared to $19.9 million in Q3 2025.

本季研發費用為 2,840 萬美元，較 2025 年第三季的 1,990 萬美元增加。

The increase was primarily due to an increase in spending related to the pivotal Phase III lucidity clinical trial and other costs related to Avexitide and our other programs.

增加主要來自與關鍵性第三期 Lucidity 臨床試驗相關支出上升，以及與 Avexitide 與我們其他計畫相關的其他成本。

The increase was partially offset by a decrease in spending related to AMX-35.

部分增加被 AMX-35 相關支出下降所抵銷。

Selling, general, and administrative expenses for the quarter were $21.3 million compared to $16.2 million in Q3 2025.

本季銷售、一般及行政費用為 2,130 萬美元，較 2025 年第三季的 1,620 萬美元增加。

This increase was primarily due to an increase in personnel-related costs to support our pre-commercialization activities and the vesting of PSU awards during the quarter.

此增加主要由於為支持商業化前活動而增加的人員相關成本，以及本季 PSU 獎勵歸屬所致。

We also recognized $10.4 million of non-cash stock-based compensation expense for the quarter, compared to $7.1 million of non-cash stock-based compensation expense in Q3 2025.

本季我們亦認列 1,040 萬美元的非現金股份基礎給付費用，相較於 2025 年第三季的 710 萬美元非現金股份基礎給付費用。

We're focused on a disciplined capital allocation, prioritizing investments that support the advancement of Avexitide in both the short and the long-term.

我們專注於嚴謹的資本配置紀律，優先投資於支持 Avexitide 短期與長期推進的項目。

Preparation for a potential commercial launch and continued development of our pipeline.

包括為潛在商業上市做準備，以及持續推進我們的產品線研發。

We believe we have the team, the data and the capital in place to execute on our ambitious plans.

我們相信，我們已具備團隊、數據與資本，足以執行我們雄心勃勃的計畫。

With that, I'll turn the call over to Josh.

那麼，我把電話會議交給 Josh。

Thanks, Jim.

謝謝，Jim。

As you heard from our remarks today, with positive Phase 3 results in hand, our first priority remains clear: to advance of Vexitide toward potential approval and commercialization.

正如各位今天從我們的發言中所聽到的，在取得正向的第三期結果之後，我們的首要任務依然明確：推進 Avexitide，朝向潛在核准與商業化邁進。

For many people living with PBH, the condition has a debilitating impact on day-to-day life, underscoring the urgent need for an approved treatment option.

對許多罹患 PBH 的人而言，這種疾病對日常生活造成嚴重影響，凸顯出迫切需要一個已核准的治療選項。

Later this month, we are excited to share that we will be hosting an in-person and virtual investor event at Obesity Week with experts who will discuss the current burden of PBH and the data on Avexitide generated to date.

本月稍晚，我們很高興分享：我們將在 Obesity Week 舉辦一場實體與線上同步的投資人活動，屆時專家將討論 PBH 目前的疾病負擔，以及迄今為止 Avexitide 所產生的數據。

We look forward to seeing many of you there.

期待在那裡見到各位中的許多人。

Looking beyond our initial launch opportunity. We are also investing in a long-acting GLP-1 receptor antagonist, AMX318.

放眼我們初次上市機會之外。我們也正在投資一款長效型 GLP-1 受體拮抗劑 AMX318。

In preclinical studies, 318 demonstrated strong in vitro potency, evidence of in vivo efficacy and tolerability, and a favorable pharmacokinetic profile consistent with a long-acting peptide.

在臨床前研究中，318 展現出強勁的體外效力、體內療效與耐受性的證據，以及與長效胜肽一致的良好藥物動力學特徵。

IND-enabling studies are underway and progressing well, and we are targeting a 2027 IND filing.

IND 申請前的支持性研究正在進行且進展順利，我們目標於 2027 年提交 IND。

We see opportunities to broaden Vexitide's reach into additional indications and to pursue geographic expansion in markets where there is a significant unmet need.

我們看到機會可將 Avexitide 的觸及範圍擴展至其他適應症，並在存在顯著未被滿足醫療需求的市場推動地理擴張。

Taken together, we believe these efforts position us not only to bring a potential first approved treatment to people living with PBH, but also to drive multiple long-term opportunities for future growth.

綜合而言，我們相信這些努力不僅使我們有望為 PBH 患者帶來可能的首個已核准治療，也能推動多項長期的未來成長機會。

We also continue to make progress across our broader pipeline.

我們在更廣泛的產品線上也持續取得進展。

For AMEX-114 in ALS, we continue to advance the Phase 1 Lumina multiple ascending dose clinical trial in people living with ALS.

針對 ALS 的 AMX-114，我們持續推進在 ALS 患者中進行的第一期 Lumina 多次遞增劑量臨床試驗。

We are progressing through the dose cohorts and recently completed enrollment in cohort 3 and began enrolling participants in cohort 4.

我們正按劑量隊列推進，近期已完成第 3 隊列的收案，並開始招募第 4 隊列受試者。

Just last week, we presented data from the Lumina trial at the annual meals meeting from the initial two dose cohorts.

就在上週，我們在年度 MDA 會議上發表了 Lumina 試驗前兩個劑量隊列的數據。

These data are available on the Amylyx publications page.

這些數據可在 Amylyx 的出版物頁面上取得。

Additionally, over the summer, we entered into a second research collaboration with Gubra to screen and develop peptide candidates for another rare endocrine disease of high unmet NEAT.

此外，在今年夏天，我們與 Gubra 達成第二項研究合作，篩選並開發胜肽候選藥物，用於另一種未被滿足需求很高的罕見內分泌疾病。

We continue to make progress on this program and look forward to sharing updates as it advances.

我們在此計畫上持續取得進展，並期待在其推進過程中分享最新進度。

Reflecting on the year, the positive lucidity results were a defining milestone for Amylyx.

回顧今年，Lucidity 的正向結果是 Amylyx 的一個關鍵里程碑。

And now our focus shifts to commercial execution, an area that we have deep experience in. As we prepare for a potential launch of Vexitide and pursue what we believe is a significant long-term opportunity ahead. With that, operator, we are now ready to take questions.

而現在，我們的重心轉向商業執行——這是我們具備深厚經驗的領域。在我們為 Avexitide 的潛在上市做準備，並追求我們認為具重大長期機會的前景之際。那麼，接線員，我們現在準備開始回答問題。

We will now begin the Q&A session. To ask a question, please press star 1 on your telephone keypad. To withdraw your question, please press star 1 again. We request that you limit yourselves to one question and a follow-up. At this time, we will pause momentarily to assemble our roster.

我們現在開始問答環節。如欲提問，請在電話鍵盤按星號 1。如欲撤回問題，請再按一次星號 1。我們請各位每人限問一題並可追問一次。此刻我們將稍作停頓，以整理提問名單。

Your first question comes from the line of Joseph Thorne of TD Cowen.

第一個問題來自 TD Cowen 的 Joseph Thorne。

Thank you. Please go ahead.

謝謝。請開始。

Hi. This is Jacob on the line for Joe. Thanks for taking my question.

嗨。我是代 Joe 上線的 Jacob。謝謝讓我提問。

I just wanted to ask the sales force strategy for Avexitide and sort of which kind of positions you'll be targeting. Thanks.

我想請教 Avexitide 的銷售團隊策略，以及你們大致會鎖定哪些職位類型。謝謝。

Sure. Thanks, Jacob.

當然。謝謝你，Jacob。

So from a Salesforce perspective, endocrinologists are going to be the primary prescribers here. Just on a broader perspective, we have initiated our go-to-market efforts, so that project is well underway, but from an overall approach, you can envision this as a it's certainly a rare endocrine launch and I think there's a few that you can reference to look at from a sizing perspective, but endocrinologists.

就銷售團隊而言，內分泌科醫師將是主要開立處方者。從更宏觀的角度來看，我們已啟動上市推進（go-to-market）工作，因此該專案正在積極進行中；但就整體策略而言，你可以把這視為一次罕見內分泌疾病的上市，我想有幾個可供參考的案例可用來估算規模，而核心仍是內分泌科醫師。

Will be the primary prescribers here. We are aware of some bariatric surgery centers that also maintain connectivity with these patients.

他們將是主要開立處方者。我們也注意到有一些減重手術中心同樣與這些病患保持連結。

So we're aware of those and we'll focus on some of them as well.

因此我們也了解這些中心，並會把其中一部分納入重點。

Thanks.

謝謝。

Next question comes from the line of Seamus Fernandez of Guggenheim.

下一個問題來自 Guggenheim 的 Seamus Fernandez。

Thank you. Please go ahead.

謝謝。請開始。

Great. Thanks for the question. So a couple quick ones here.

很好。謝謝讓我提問。我這裡有幾個簡短問題。

In terms of lucidity and what we're going to see this weekend, can you just help us understand what it teaches about disease severity and the impact of Evexatide on patients with very severe disease? And I was just kind of hoping that you might be able to extend that into.

關於 Lucidity 以及我們這週末將看到的內容，你能否協助我們理解：它對疾病嚴重程度有何啟示，以及 Avexitide 對非常嚴重患者的影響？另外我也希望你能延伸談談——

Are learning about potential low-hanging fruit and the patients that doctors are really going to be seeking to treat urgently or those patients that are going to be seeking to get into the endocrinologist's office.

我們是否能從中了解哪些是相對「容易切入」的族群，也就是醫師會真正急於治療的患者，或是那些會相對快速尋求進入內分泌科門診的患者。

Relatively quickly. And the second question is on the EAP, where do we stand just in terms of the kind of inclusion of OLE patients into the early access program? How far along and how many patients have been recruited if you're willing to update that? Or if not, what does it take to kind of activate those patients and do you see an opportunity to further expand the EAP?

相對快。第二個問題是關於 EAP（早期使用計畫）：就 OLE 患者納入早期使用計畫而言，我們目前進展到哪裡？如果你願意更新的話，目前進行到什麼程度、已招募多少患者？或者如果不便提供數字，啟動這些患者納入需要哪些條件？你是否看到進一步擴大 EAP 的機會？

Or are there potential limitations to that, whether it be supply, the three lots that you have to kind of confirm with FDA, etc., etc. So those are the questions. Thanks.

或者是否存在一些潛在限制，例如供應量、你們需要與 FDA 確認的三個批次等諸如此類。以上就是我的問題。謝謝。

I think I caught all of them.

我想我都聽到了。

And thanks, Seamus as well.

也謝謝你，Seamus。

So maybe starting on lucidity and the disease severity, I mean, I think first it's important to note that even a single Level 2 or Level 3 hypoglycemic event can be a medical emergency.

那麼先從 Lucidity 與疾病嚴重程度談起。我認為首先重要的是要指出：即使只有一次第 2 級或第 3 級低血糖事件，也可能是醫療急症。

So people who are experiencing those and we believe that's the 160,000 people living with PBH have a need for medical therapy in our view.

因此，正在經歷這類事件的人——我們認為這涵蓋約 16 萬名 PBH 患者——在我們看來都有藥物治療的需求。

When we've done ad boards, Lucidity required people to be having at least one event of level 2 or level 3 severity per week during the run-in period. When we've done advisory boards with different physicians, they describe that as fairly typical of their PBH patients. Certainly, some have it more frequently, some less, but it's not uncommon to see people having an event a week or more.

在我們進行諮詢委員會（ad boards）時，Lucidity 要求受試者在導入期（run-in period）每週至少發生一次第 2 級或第 3 級嚴重程度的事件。當我們與不同醫師進行諮詢委員會時，他們將這描述為其 PBH 患者相當典型的情況。當然，有些人發生得更頻繁，有些較少，但每週一次或更多的事件並不罕見。

In terms of, I think you kind of mentioned low-hanging fruit from kind of a commercial perspective, I think it's a little early to say, but I will say as we've been to different conferences, Endo and others, we do see a high degree of interest from physicians kind of proactively coming to our booth or otherwise asking for more information, kind of noting that this is a really difficult. Condition for them to treat and that they're really looking for kind of new options and have excitement about Evexitide. And then I think in terms of the EAP, probably unsurprisingly, there's a lot of excitement and interest in the EAP. We probably won't give sort of interim numbers as it goes on, especially because the main point of the program is access.

至於你提到從商業角度的「低垂果實」，我認為現在下結論還稍早；但我會說，當我們參加不同會議（如 Endo 等）時，確實看到醫師有高度興趣，會主動到我們攤位或以其他方式詢問更多資訊，並指出這對他們而言是一個非常難以治療的。疾病，他們非常期待新的選項，並對 Avexitide 感到興奮。至於 EAP，我想不令人意外地，EAP 受到非常多的期待與關注。我們可能不會在計畫進行中提供階段性的中期數字，尤其因為該計畫的主要目的在於提供用藥可近性。

But it is a clinical trial, so you do have to activate sites, there is a similar kind of contracting and otherwise as you might see in clinical trials. But we've had quite a lot of interest for that program.

但這是一項臨床試驗，因此你確實必須啟動各個試驗中心；在合約簽訂等方面，也會有你在臨床試驗中常見的類似流程。不過，我們對該計畫已經收到相當多的興趣。

Great.

很好。

Thank you.

謝謝。

Next question comes from the line of Jeff Meacham from Citibank.

下一個問題來自花旗銀行（Citibank）的 Jeff Meacham。

Thank you. Please go ahead.

謝謝。請繼續。

Great. Hey, guys. Thanks for taking the question. I just have a couple. The first is when you think about the 160K US PBH patients, maybe just help us with the math that you've done that informs how many are officially diagnosed in medical management that are the truly addressable populations in the call of the 1st year or so.

很好。各位好。謝謝讓我提問。我有兩個問題。第一個是，當你們思考美國約 16 萬名 PBH 患者時，能否幫我們算一下你們做過的推估：其中有多少人是已被正式診斷、並在醫療管理之下、且在上市後第一年左右真正可觸及的族群？

And the second question, just again on the EAP, can you talk at least about what you can learn that would inform the patient identification and persistent rates just related to the launch execution?

第二個問題同樣是關於 EAP（擴大使用計畫），你們至少能談談可以學到哪些內容，以便在上市執行上，幫助病患辨識以及用藥持續率（persistency rates）嗎？

Thank you.

謝謝。

I'm happy to start, then maybe I'll pass to Dan as well.

我先開始回答，之後也許再請 Dan 補充。

So in terms of the math on diagnosis, I think we start from the claims data. And when we've done our claims-based analyses, we've been able to identify approximately 160,000 people in claims who we believe have PBH.

就診斷的推算而言，我們是從理賠（claims）資料開始。在我們做過的以理賠資料為基礎的分析中，我們能在理賠資料裡辨識出約 16 萬人，我們認為他們患有 PBH。

And PBH only recently got an ICD-10 code. So we did that work initially, looking at those people who had hypoglycemic events following bariatric surgery that couldn't be explained by other means.

而且 PBH 直到最近才有 ICD-10 代碼。因此我們一開始做這項工作時，是去看那些在減重手術（bariatric surgery）後出現低血糖事件、且無法用其他原因解釋的個案。

But if they're having those codes, we sort of look at that population as certainly one that's being looked at by the medical profession, possibly diagnosed as well. What I'd add to that sort of helped confirm this, we went out and did survey-based work where we kind of queried sites about how many patients are under their care. And what we found was that their responses were very concordant with our claims-based data, which again to us confirmed that our claims-based data is successfully identifying those patients that the physicians view is sort of diagnosed and under their care.

但如果他們出現了那些代碼，我們會把那群人視為：醫療專業人員確實正在關注他們，且也可能已經被診斷。另外補充一點來佐證：我們也做了以調查為基礎的研究，向各中心詢問其照護中的病患數。我們發現他們的回覆與我們的理賠資料非常一致；這再次讓我們確認，我們的理賠資料確實成功辨識出那些醫師認為已被診斷、並在其照護之下的病患。

And then I'd say in terms of EAP.

接著談到 EAP。

It's probably early to talk about persistency rates. I think we'll probably hold off on that until we're further along and probably until we're hopefully into commercialization as well. I don't know if you want to add anything, Dan, as well.

現在談用藥持續率可能還太早。我想我們可能會先保留，等進展更往後、也希望進入商業化之後再談。Dan，你是否也想補充？

I think you covered it really well. I would say from the claims analyses that we've done, and we've done it multiple times looking at several different databases, we continue to feel more and more confident in the size and the representation of the 160. We start with the known patient who's had bariatric surgery and then we apply. The hypoglycemic hypoglycemia diagnosis, non-diabetes hypoglycemia. And then we add in other factors, whether it was a trip to the emergency room, a visit to an endocrinologist, back to their bariatric surgeon.

我覺得你已經講得很好了。我會說，從我們做過的理賠分析來看——而且我們用好幾個不同資料庫重複做了多次——我們對「16 萬」這個規模與代表性愈來愈有信心。我們從已知做過減重手術的病患開始，然後套用低血糖診斷（非糖尿病性低血糖）。接著再加入其他因素，例如是否曾到急診、是否看過內分泌科醫師、是否回診其減重手術外科醫師等。

So that's how we're tracking. Every time we look at it, we feel more and more confident in the size and the representation of the 160.

所以我們就是這樣追蹤的。每次我們重新檢視，都對「16 萬」的規模與代表性更有信心。

Thanks, guys.

謝謝各位。

Next question comes from the line of Mark Goodman from Leering Partners.

下一個問題來自 Leerink Partners 的 Mark Goodman。

Thank you. Please go ahead.

謝謝。請繼續。

A different type of question. I was thinking about business development and just how much time you guys are spending on finding the nexovexatide and are we looking at CNS?

我有個不同類型的問題。我在想商務開發（business development），以及你們花多少時間在尋找下一個 nexovexatide；我們是在看中樞神經系統（CNS）嗎？

Are we looking at endocrinology? Like what areas are you looking in and how likely is it that over the course of the next?

是在看內分泌領域嗎？你們在看哪些領域？以及在接下來

12 months you might bring in another product like this one?

Thanks, Mark. I appreciate the question. So I'd say, first and foremost, we have a lot to do around here, which we're very excited about submitting the NDA by the end of this year and then preparing for launch in an unmet need with a rare but substantial population in the United States. I'd say even beyond that. There's a substantial unmet need of PBH internationally.

There are other surgeries that lead to the same condition.

And then, of course, we have our ongoing work with AMX-318 as well with the goal to submit the IND for that next year as well.

And then, of course, we also have AMX-35 in Wolfram's syndrome and AMX-114 in ALS. So we have a lot going on here.

That being said, I think that we have a really excellent team here who has shown great ability to work in areas of high unmet need, execute on clinical development.

And we are now building our commercial capabilities as well. So we always sort of keep our eyes open for unmet need and opportunities for the company, but we have some very strong programs we're working on right now, so that's really our focus for the certain certainly for the next 12 months as you asked about.

Next question comes from the line of Michael DeFure from Evercore ISI.

Thank you. Please go ahead.

Hey, guys. Thanks so much for taking my question. Two quick ones from me.

從定價的角度來看，第二個問題。

In terms of any payer discussions or feedback you may have gotten so far, any feedback have you received on various DUR requirements for therapy, whether it be documented low glucose, nutritional therapy failure, off-label medications, diabetics, etc.

在我們拿到 Lucidity 的結果之後，這又是一項我們將要開始並立即啟動的工作。我想先從商業角度談定價：我們對 Lucidity 的結果感到非常振奮。我認為其臨床影響，以及有機會成為 PBH 患者第一個獲核准的治療，意義相當重大，我們絕不會輕忽。

And my second question is on pricing. I know it's early, but how are you balancing rare endocrine pricing with broad access?

現在談定價細節還稍早。我認為確實有一些罕見疾病產品在規模、盛行族群，甚至在罕見內分泌領域上都具有相似性。因此，隨著我們在定價區隔上愈來愈具體，後續會有更多資訊。

Thank you.

Sure. Thanks, Mike, for the question. The first one on the payer front, so we're in the process of building out our payer team right now. So we've hired our national account directors. They're currently on board and they're getting ready to engage with the broader payer community and those conversations will take place pretty soon.

From a utilization management criteria. Those are all the conversations that we'll begin to have with the payers as we start to have the payer information exchanges in the first quarter of the year.

I think from a utilization management, again, I think the first thing I'd say, there are no approved medications for patients with postbariatric hypoglycemia.

So I think it'd be hard for a payer to put a step or a criteria in place. Where a product is not approved. But that's all the education and the conversations that we'll with the community in the coming months to learn more and educate the payers about postbariatric hypoglycemia.

From a pricing perspective, the second question.

It's work again that we're going to start and kick off now that we have our lucidity results. The first thing I'd say on the pricing from the commercial perspective, we're extremely excited about the lucidity results. I think the clinical impact and the opportunity to potentially become the first approved treatment for patients with PBH is quite significant and not something we will take lightly.

Little bit early to discuss the specifics on pricing. I think there's certainly some rare products with similar sizing, similar prevalence populations, and even similar in the rare endocrinology space. So more to come as we get more and more specific within the pricing segment.

I'll add just briefly on the PowerPoint too. So while we're doing that work now, as Dan indicated, I'll highlight that what I think is helpful here is that while PBH is new for payers, hypoglycemia is certainly not given their coverage of insulin and other drugs that can cause severe hypoglycemia. And severe hypoglycemia has been known for a long time to be really a medical emergency. So I think we have a body of literature to draw on to help educate payers as to the severity of this condition when you have people who are having such frequent medical emergencies.

我也就簡報再補充一句。因此，雖然我們現在正在做這項工作，如 Dan 所指出的，我想強調我認為這裡有幫助的一點是：雖然 PBH 對付款方而言是新的，但低血糖絕對不是，因為他們本來就承保胰島素以及其他可能導致嚴重低血糖的藥物。而且長久以來大家都知道嚴重低血糖確實是一種醫療急症。所以我認為我們有一套文獻基礎可供引用，用來協助教育付款方：當有人如此頻繁地發生醫療急症時，這個病況的嚴重性。

So I think that's really the first objective for our team is to highlight just what a critical condition TBH is. And then, of course, we believe we have the opportunity to potentially change practice with the first treatment that can treat those hypoglycemic events.

因此我認為我們團隊的第一個目標，就是凸顯 TBH 是多麼危急的病況。然後，當然，我們相信我們有機會透過第一個能治療這些低血糖事件的療法，來改變臨床實務。

Thank you.

謝謝。

Next question comes from the line of Rami Kathkuda from LifeSci Capital.

下一個問題來自 LifeSci Capital 的 Rami Kathkuda。

Thank you. Please go ahead.

謝謝。請繼續。

Good morning, and thanks for taking my questions as well. I guess, do you expect PBH patients to be concentrated at high-volume bariatric surgery centers or key endocrinology practices, and how does that influence your commercial launch strategy? And then, I know it's early, but how should we think about the initial launch trajectory for Vexitide? How large of a bolus of patients do you anticipate at launch given the EAP and open-label extension?

早安，也謝謝你們回答我的問題。我想請問，你們是否預期 PBH 患者會集中在高量能的減重手術中心或關鍵的內分泌專科診所？這會如何影響你們的商業上市策略？另外，我知道現在還早，但我們應該如何看待 Vexitide 初期的上市軌跡？考量 EAP 與開放標籤延伸試驗，你們預期上市時會有多大一波（bolus）的患者量？

Excellent. I can start. Yeah, so on the concentration of the marketplace and the patients with post-bariatric hypoglycemia, again, that's work that we've been doing. We've mentioned the claims analysis to validate the 160.

很好。我可以先回答。是的，關於市場與術後減重手術低血糖（post-bariatric hypoglycemia）患者的集中度，這同樣是我們一直在做的工作。我們提過用理賠資料分析來驗證 16 萬這個數字。

Within those analyses, we can also see where these patients are.

在這些分析中，我們也能看到這些患者在哪裡。

So we can see whether there's a setting of care or an endocrinologist that has 40 to 60 patients.

因此我們可以看出是否存在某種照護場域，或某位內分泌科醫師手上有 40 到 60 位患者。

Some have upwards of 70. And we've even spoken with endocrinologists that have greater than 100 patients or potential patients with postbariatric hypoglycemia in their care. So that's certainly where we'll focus. We also know that there's a broader educational effort that's needed to raise awareness of. The severity of post-bariatric hypoglycemia, but also the signs and symptoms. So as our disease data education efforts take shape and as our field medical colleagues get out there and continue to educate the marketplace, I think you'll see the treatment expand beyond the top centers. And I wouldn't even say it's within the top centers. It's just we know that's where a lot of these patients are because these are extremely severe patients and oftentimes they are then channeled towards. A particular expert in the local marketplace. So from a launch perspective and the launch trajectory, again, the first thing I'd say is there's no approved therapies for postbariatric hypoglycemia. It's certainly a sizable market, the 160,000 size, that's quite significant for a rare disease, but it's also a market that we continue to see and we believe that will build overtime.

有些甚至超過 70 位。我們也曾與內分泌科醫師交流，他們照護的術後減重手術低血糖患者或潛在患者超過 100 位。所以那當然會是我們聚焦的地方。我們也知道需要更廣泛的教育推廣來提升對……的認知。也就是術後減重手術低血糖的嚴重性，以及其徵兆與症狀。因此，隨著我們的疾病資料教育工作逐步成形，以及我們的醫學外勤同仁走出去持續教育市場，我認為你們會看到治療會擴展到頂尖中心之外。而且我甚至不會說只是在頂尖中心。只是我們知道很多患者在那些地方，因為這些患者非常嚴重，往往會被轉介到……當地市場中的某位專家。所以從上市角度與上市軌跡來看，我首先要說的是：目前沒有任何獲核准的術後減重手術低血糖療法。這確實是一個相當可觀的市場，16 萬的規模對罕見疾病而言非常顯著，但同時我們也持續看到這個市場，並且相信它會隨時間逐步建立。

With the endocrinologists that we've done research with, that we continue to engage with, there's a very high intent to treat these patients with postbariatric hypoglycemia. So again, we know where a lot of these patients are, we certainly don't know where all of them are. So I think we'll see a good concentration upfront, but then as our educational efforts take shape and more and more awareness of postbariatric hypoglycemia occurs across the healthcare setting, we'll see that launch continue to build over time.

就我們與之進行研究、並持續互動的內分泌科醫師而言，他們對於以此治療術後減重手術低血糖患者的意願非常高。所以再說一次，我們知道很多患者在哪裡，但我們當然不知道所有患者在哪裡。因此我認為初期會有不錯的集中度，但隨著我們的教育推廣逐步成形，以及整個醫療照護體系對術後減重手術低血糖的認知愈來愈高，我們會看到上市持續隨時間成長。

Awesome.

太好了。

Thank you.

謝謝。

Next question comes from the line of James Kondulas from Stifel.

下一個問題來自 Stifel 的 James Kondulas。

Thank you. Please go ahead.

謝謝。請繼續。

Hey, thanks for taking my question and congrats on all the progress.

嗨，謝謝你們回答我的問題，也恭喜你們取得所有進展。

Maybe just one on sort of the current treatment landscape. I think one thing we've heard from some docs is just anecdotally that GLIF-1 agonists have been used in this space and just like kind of like curious your latest perspectives there and if that has sort of like any implications, if at all, on sort of like how the PBH treatment landscape will evolve overtime?

我想問一個關於目前治療版圖的問題。我們從一些醫師那裡聽到一些軼聞：GLIF-1 促效劑在這個領域曾被使用；我想了解你們目前最新的看法，以及這是否會對 PBH 的治療版圖隨時間演變產生任何影響（如果有的話）？

Sure.

好的。

I think as we've talked to physicians in the space, we've heard kind of two concepts there. One, the concept of, could a GLP-1 agonist be helpful for hypoglycemia? And then another one of, just if people want to lose more weight.

我認為當我們與這個領域的醫師交流時，我們聽到大致有兩個概念。第一個概念是：GLP-1 促效劑是否可能對低血糖有幫助？第二個則是：如果有人想再減更多體重。

Talking on the hypoglycemia, there's actually been three randomized trials, all of which showed no benefit of GLP-1 agonists for hypoglycemia in PBH.

就低血糖而言，實際上已有三項隨機試驗，全部都顯示 GLP-1 促效劑對 PBH 的低血糖沒有益處。

Which makes a lot of sense when you consider these drugs were originally approved for diabetes to lower your A1C, basically to lower your blood sugar. It would be quite surprising for them to raise your blood sugar.

這其實很合理，因為這些藥物最初是為糖尿病核准，用來降低 A1C，基本上就是降低血糖。因此它們會提高血糖其實會相當令人意外。

So we do not believe, based on the current evidence, that these are effective for hypoglycemia.

所以基於目前的證據，我們不認為這些藥物對低血糖有效。

In terms of weight loss, yes, certainly they can be effective for weight loss, but most people who have PBH have already lost significant weight. They've had a bariatric surgery, so we don't anticipate that to be a particularly large pool of utilization either. So it's something we'll definitely watch, but I don't think -- I think what people are really looking for in this disease, given the severity, given the.

至於減重，是的，它們確實可能對減重有效，但多數 PBH 患者已經減了相當多的體重。他們接受過減重手術，因此我們也不預期這會是一個特別大的使用族群。所以這是我們一定會持續觀察的事情，但我不認為——我認為在這個疾病中，人們真正想要的，考量其嚴重性，考量……

Frequent medical emergencies that these patients are experiencing is treatment of hypoglycemia. And I think that's what we hope to offer with Avaxiton.

這些患者所經歷的頻繁醫療急症，是低血糖的治療。而我認為這正是我們希望透過 Avaxiton 提供的。

Thanks, Sam.

謝謝，Sam。

Thank you.

謝謝。

Next question comes from the line of Greg Silvanovich of Mizuho.

下一個問題來自 Mizuho 的 Greg Silvanovich。

Thank you. Please go ahead.

謝謝。請繼續。

Hi, this is Sam on for Greg. Thanks for taking my question. Maybe two quick ones for me. First, in terms of the NDA for vexatide, will that be only Roux-en-Y?

嗨，我是代 Greg 提問的 Sam。謝謝你們回答我的問題。我這邊可能有兩個簡短問題。第一，關於 vexatide 的 NDA，是否只會涵蓋 Roux-en-Y？

So how are you guys thinking about gastric sleep patients?

所以你們是如何看待胃部睡眠患者的？

Do you guys have a study plan there? And then maybe on the pipeline for Illumina study in cohort 2-3, any guidance on when we can expect data? And then in terms of expectations of what would be a good signal in terms of the biomarker responses given the cohort 1 kind of stayed near baseline. Thanks.

你們那邊有研究計畫嗎？另外，關於 Lumina 研究在第 2–3 隊列的進展，你們能否提供一些指引，何時可以期待看到數據？再者，就生物標誌物反應而言，考量到第 1 隊列大致維持在接近基線，你們認為什麼樣的訊號才算是好的訊號？謝謝。

Great.

很好。

Thank you very much. Good morning, Sam.

非常感謝。早安，Sam。

With regard to the NDA, we'll be submitting the data from the overall Avexiti program. Featuring prominently, of course, the lucidity trial, which was conducted in people with TBH following Roux-1Y gastric bypass surgery. And most of the studies conducted weren't in people having had Roux-1Y. Having said that, we also had a Phase IIb study in which individuals also received other upper GI surgeries and other upper bariatric surgeries as well.

關於 NDA，我們將提交整體 Avexiti 計畫的數據。其中當然會重點呈現 Lucidity 試驗；該試驗是在接受 Roux-en-Y 胃繞道手術後出現 TBH 的受試者中進行。而我們進行的大多數研究並非在曾接受 Roux-en-Y 的人群中進行。不過，我們也有一項第二期 b（Phase IIb）研究，受試者也接受了其他上消化道手術以及其他上消化道減重手術。

And those data were also.

而那些數據也……

Those results were also positive.

那些結果也呈現正向。

So, we're submitting the data, and we're very excited about the data, as you've heard about the lucidity results, highly statistically significant, clinically meaningful. And beyond that, the data will speak for themselves, I would say.

因此，我們正在提交這些數據，我們對這些數據感到非常振奮；正如你所聽到的 Lucidity 結果，具高度統計顯著性且具臨床意義。除此之外，我想數據本身會說明一切。

And then you had another question about the pipeline, Lumina, and what we might see there. So, first, we're very excited about the progress of the Lumina study.

你還問到另一個問題，關於管線、Lumina，以及我們可能會看到什麼。首先，我們對 Lumina 研究的進展感到非常興奮。

As we noted, we've completed enrollment in cohort three, which is our third of four dose levels, 50 milligrams. And we're currently enrolling in cohort four at 100 milligram dose level.

如我們所提到的，我們已完成第 3 隊列的收案，這是四個劑量層級中的第三個，50 毫克。目前我們正在以 100 毫克劑量層級招募第 4 隊列。

We also recently presented.

我們也在近期發表了……

Results of cohorts one and two at the NEOS progress.

在 NEOS 進展會議上發表了第 1 與第 2 隊列的結果。

NEOS Congress, excuse me. And, we were very pleased that the data show very well tolerated ASO in people with ALS.

更正，是 NEOS 大會。我們非常高興看到數據顯示：在 ALS 患者中，ASO 的耐受性非常良好。

And, those were the first lowest doses, the cohort one the lowest and then the next lowest dose. So.

而那是最低的兩個劑量：第 1 隊列是最低劑量，接著是下一個最低劑量。所以……

Early days yet to see biomarker results.

目前仍屬早期階段，尚待觀察生物標誌物結果。

And therefore, we're very excited to see how the data unfolds in cohorts three and four. Importantly, we're able to go to cohorts three and four because the drug is well tolerated. So we're very excited about the potential here.

因此，我們非常期待第 3 與第 4 隊列的數據如何展開。重要的是，我們之所以能推進到第 3 與第 4 隊列，是因為該藥物耐受性良好。所以我們對此潛力感到非常興奮。

And Sam, just to the first.

Sam，另外回到第一個……

Question as well. So obviously, we have yet to submit the NDA, and so we're a ways off from the label indication statement.

問題也是。很明顯，我們尚未提交 NDA，因此距離標籤適應症敘述還有一段距離。

We believe that TBH is PBH, the pathophysiology is the same, but as Camille indicated, the Phase III trial was in PBH following Roux-en-Y gastric bypass surgery.

我們認為 TBH 就是 PBH，其病理生理機轉相同；但如 Camille 所指出，第三期試驗是在 Roux-en-Y 胃繞道手術後的 PBH 患者中進行。

So then as we look forward, first, I'd say.

因此展望未來，首先我會說……

The current existing prevalent population in the United States, our estimates are that about 120,000 of the 160,000 currently had Roux-en-Y gastric bypass that have led to their PBH. And we'll be prepared for whatever scenario.

就美國目前既有的盛行患者族群而言，我們估計在目前約 16 萬名患者中，約有 12 萬名是因 Roux-en-Y 胃繞道而導致 PBH。我們會為任何情境做好準備。

Avexatide, you can see the effect of it in a single dose.

Avexatide 單次給藥就能看到其效果。

And as Camille indicated, we have strong data from the Phase 2B of different surgeries leading to the same hypoglycemic condition. And of exotide appears to work regardless of the surgical intervention that led to the condition. So, we'll be prepared for any scenario and both in terms of our launch preparations and our development preparations.

而如 Camille 所提，我們在 Phase 2b 也有不同手術導致相同低血糖狀況的強勁數據。而 Avexatide 似乎不受導致該病況之手術介入方式影響，皆能發揮作用。因此，無論在上市準備或開發準備方面，我們都會為任何情境做好準備。

That's very helpful.

這非常有幫助。

Thank you.

謝謝。

Next question comes from the line of Jason Gerberry from Bank of America.

下一個問題來自美國銀行（Bank of America）的 Jason Gerberry。

Thank you. Please go ahead.

謝謝。請發問。

Good morning. This is Dina on for Jason.

早安。我是 Dina，代 Jason 發問。

Thank you guys for taking our question. Two quick ones from us.

謝謝你們回答我們的問題。我們有兩個簡短問題。

Does the dedicated ICD-10 code provide any sort of launch tailwind or change your ability to identify high-volume treaters heading into launch?

專屬的 ICD-10 代碼是否能為上市帶來任何順風，或改變你們在上市前辨識高量治療醫師的能力？

And then at the lucidity top-line, you said that the treatment and placebo distributions were essentially not overlapping once the patients were on drugs. When you present the full data set, should we expect that benefit?

另外，在 Lucidity 的頂線結果中，你們提到一旦患者用藥後，治療組與安慰劑組的分布基本上不重疊。當你們呈現完整數據集時，我們是否應預期該效益……

To look relatively uniform across patients, or is there a subset of super responders?

在患者之間看起來相對一致，還是存在一群「超級反應者」的子群？

And specifically what proportion of patients became completely free of level 2/3 events?

具體而言，有多少比例的患者完全不再出現第 2/3 級事件？

Thank you.

謝謝。

Sure. I can start with the first question on the ICD-10 code. So, yes, an ICD-10 code specifically for post-bariatric hypoglycemia did go into effect in the beginning of October.

好的。我先回答第一個關於 ICD-10 代碼的問題。是的，針對減重手術後低血糖（post-bariatric hypoglycemia）的 ICD-10 專屬代碼已於 10 月初生效。

We definitely view the availability of a dedicated code as an important milestone for this community. It's also a reflection. The growing awareness of PBH. So that's something we are looking forward to. With the ICD-10 code, they're often used for diagnosis as well as tracking of patients within the healthcare system. The code itself, though, is not needed for reimbursement, but it does represent an important step towards improving disease awareness as well as patient identification over the long-term.

我們確實將專屬代碼的可用性視為此社群的一個重要里程碑。這也反映出……對 PBH 的認知正在提升。因此我們很期待這項進展。ICD-10 代碼通常用於診斷，也用於在醫療體系內追蹤患者。不過，該代碼本身並非報銷所必需，但它確實代表朝向提升疾病認知與長期患者辨識的重要一步。

What I would say on the code specifically, a new one, with the new diagnosis code, adoption as well as implementation. Of a specific code for PVH. It's going to take some time to be absorbed across the broader medical community.

就代碼本身而言，新的診斷代碼在採用與落地實施上……針對 PBH 的特定代碼……需要一些時間才能在更廣泛的醫療社群中被吸收與普及。

Yes, and then maybe the Lucidity distribution of patients question. Maybe first, I'd say, just to remind, we're incredibly excited about the Lucidity results. I think the more we've dug into them, just the more excited we've continued to get.

是的，接著談談 Lucidity 患者分布的問題。首先提醒一下，我們對 Lucidity 的結果感到無比振奮。我想我們越深入分析，就越覺得興奮。

We met our pre-specified primary outcome. We met every secondary outcome. We also had a favorable safety profile.

我們達成了預先設定的主要終點。我們也達成了所有次要終點。同時也呈現良好的安全性概況。

So getting to the presentation, I'll probably let Madeleine and Tan in her presentation give kind of all the details, but our hope there is certainly just to kind of give more detail across the secondaries, across the safety, et cetera, all of which has been quite favorable and given the strength of the p-value.

至於正式發表，我可能會讓 Madeleine 與 Tan 在她的簡報中提供所有細節；但我們的期待是能在次要終點、安全性等各方面提供更多細節，而這些結果都相當正向，且 p 值的強度也很高。

What that essentially means is the error bars are pretty small here. So the distributions are pretty tight in terms of how the active group looks and in terms of how the placebo group looks.

這基本上意味著這裡的誤差棒相當小。因此，不論是治療組的表現或安慰劑組的表現，其分布都相當集中。

Thank you. Appreciate it.

謝謝。很感謝。

Next question comes from the line of Ananda Ghosh from HC Wainwright.

下一個問題來自 HC Wainwright 的 Ananda Ghosh。

Thank you. Please go ahead.

謝謝。請發問。

Hi, guys.

嗨，各位。

Thank you and congrats on the quarter.

謝謝，也恭喜本季表現。

I have two questions from the commercial front.

我有兩個來自商業端的問題。

Of the patients identified in your claims work, how many have been validated as actively managed, clinically confirmed PBH patients at your initial target centers?

在你們理賠分析中辨識出的患者裡，有多少人在你們最初的目標中心被驗證為正在接受主動管理、且臨床確認的 PBH 患者？

And now that the PBH code is effective, what will you measure to distinguish existing patients being recorded from newly diagnosed patients entering care?

另外，既然 PBH 代碼已生效，你們將衡量哪些指標，以區分既有患者被記錄下來，與新診斷、進入照護體系的患者？

So I can start with the first question just on the specifically on the patients and of the patients that we've used within the we've identified within the claims analyses.

我可以先從第一個問題開始，特別是關於患者，以及我們在理賠分析中所使用、所辨識出的那些患者。

One, we've done it four times now, multiple times, and every time we look at it. So we continue to feel more and more comfortable in the sizing of the particular marketplace. But specifically, as Josh mentioned earlier in his comments, we've also referenced that with additional market research. So we've done additional market research on survey work to validate the 160 specifically within very specific settings of care. But then we also have our field medical colleagues who are out there engaging and talking with key opinion leaders. As well as large practices and some of the experts that continue to validate the size of the marketplace within the local geographies.

第一，我們現在已經做了四次，多次進行，每次我們都會檢視。因此，我們對於特定市場規模的估算越來越有信心。但具體而言，如 Josh 先前在評論中提到的，我們也透過額外的市場研究來交叉驗證。因此，我們也做了額外的市場研究與問卷調查工作，以在非常特定的照護場景中驗證這 160（的數字）。此外，我們的醫學外勤同仁也在外與關鍵意見領袖互動並交流。以及大型診所與一些專家，持續在各地區層面驗證市場規模。

Yes, and then maybe on your question around the code as well, again, probably a little early as codes take a little bit of time to kind of get implemented into people's medical record systems and all those sorts of things. But yes, we'll certainly continue looking at that. But probably the most important thing is what it represents in terms of this disease growing in terms of awareness and kind of being a key disease that hopefully will be tracked and managed well by endocrinologists and otherwise.

是的，至於你關於代碼的問題，同樣地，可能還稍早一些，因為代碼需要一點時間才能被導入到大家的病歷系統以及各種相關流程中。不過是的，我們當然會持續觀察。但可能最重要的是，它所代表的意義：這個疾病在認知度上正在提升，並且希望能成為內分泌科醫師及其他醫師會良好追蹤與管理的關鍵疾病。

Great.

很好。

Thanks.

謝謝。

Next question comes from the line of Christopher Chen of Baird.

下一個問題來自 Baird 的 Christopher Chen。

Thank you. Please go ahead.

謝謝。請繼續。

Thank you. Good morning. Thanks for taking my questions and congrats on a successful quarter.

謝謝。早安。感謝回答我的問題，也恭喜本季表現亮眼。

Just regarding patient eligibility and Evexatide's potential label, I believe patients in your trials needed to have been at least a year removed from bariatric surgery. Just curious as to why that criterion was in place and is it possible that Evexatide's potential label could include patients less further removed from bariatric surgery? Thanks.

關於患者適格性與 Evexatide 潛在標籤，我記得你們試驗中的患者必須距離減重手術至少滿一年。想請教為什麼會有這個條件？以及 Evexatide 的潛在標籤是否可能納入距離減重手術未滿一年的患者？謝謝。

Yes. Thanks, Chris. Hi.

是的。謝謝你，Chris。你好。

So, we did that. What was the eligibility? Individuals had to be at least one year out. And it really is, in part historical, we want to be as consistent as possible with how the prior trials were conducted. And why one year? Because that is generally the timeframe in which symptoms and signs of PBH. Develop in people post bariatric surgery. So that's the context there. With regard to the label, well, right now we're focusing on submitting our NDA and it is too early yet to be contemplating what the label or the indication will be.

所以，我們確實是這樣設計的。適格條件是什麼？個體必須在手術後至少滿一年。這部分在某種程度上是歷史因素，我們希望盡可能與先前試驗的執行方式保持一致。那為什麼是一年？因為一般而言，PBH 的症狀與徵象會在減重手術後的這段時間內出現。也就是在術後人群中逐步發展。所以背景是這樣。至於標籤方面，目前我們專注於提交 NDA（新藥申請），現在就去推測標籤或適應症會是什麼，還太早。

And maybe only other comment just.

也許再補充一點。

Also important to note that if you think about the time immediately after bariatric surgery, there's a lot of changes happening for the individual. It's not uncommon over the first six months for people to lose 100 plus pounds following bariatric surgery. So you do also, in the trial setting, want to run in a case where, they've achieved some degree of stability, before entering the trial as well.

另外也很重要的一點是，如果你想到減重手術後的立即期間，個體會經歷很多變化。在前六個月內，減重手術後體重減少超過 100 磅並不少見。因此在試驗情境下，你也希望在受試者達到某種程度的穩定後，再進入試驗。

Yeah, and I'll just add one more, I think, important note of concept.

是的，我再補充一個我認為很重要的概念性重點。

Prevalent population.

盛行族群。

Once TBH occurs, it does not appear to go away. We very regularly work with people who have had TBH for 15, 20 years.

一旦 TBH 發生，似乎不會自行消失。我們非常常與已經罹患 TBH 15、20 年的人合作。

So I'd say it's very unfortunate for people living with TBH, but again, we think it represents both the severity, the unmet need of the condition, but the opportunity in front of us as well.

所以我會說，對 TBH 患者而言這非常不幸，但同樣地，我們認為這也反映了此疾病的嚴重性與未被滿足的需求，同時也代表我們面前的機會。

Helpful context.

這些背景很有幫助。

Thank you.

謝謝。

Next question or last question comes from the line of Rudy Lee of Wolf Research.

下一個問題，也是最後一個問題，來自 Wolf Research 的 Rudy Lee。

Thank you. Please go ahead.

謝謝。請繼續。

Thanks for taking my question. Can you maybe provide more color on the uncover the mystery of PBH campaign? We're able to locate more patients in your database and how many healthcare professionals have signed up like what's the specialty mix. Thanks.

謝謝讓我提問。你們能否更詳細說明一下「揭開 PBH 之謎（uncover the mystery of PBH）」這個宣導活動？你們是否因此在資料庫中找到更多患者？以及有多少醫療專業人員註冊，專科組成大概是什麼？謝謝。

Sure, thanks for the question on the disease state education campaign. And it's a campaign that, again, we launched it in June and the response has been quite significant. And the feedback we received from both bariatric surgeons, from endocrinologists, we're aware of primary care physicians who have engaged for education. So the response has been quite high.

當然，謝謝你關於疾病狀態教育宣導活動的提問。這個活動我們在 6 月推出，反應相當顯著。我們從減重手術外科醫師、內分泌科醫師都收到回饋，也知道有基層照護醫師參與並接受教育內容。所以整體回應度相當高。

But we've also received specific feedback on the site too, just that there's a recognition of the unmet need, the need for more education across the broader healthcare system. So we're excited about the results. Again, we put it out there in many channels, but that's something as we continue to expand and prepare for a potential launch in '27.

我們也收到對網站的具體回饋，大家認同未被滿足的需求，以及在更廣泛的醫療體系中需要更多教育。因此我們對結果感到振奮。同樣地，我們透過多個管道推廣；而隨著我們持續擴大並為 2027 年可能的上市做準備，這會是我們持續推進的工作。

We are bringing that and bringing uncovered PBH to a broader community within the healthcare ecosystem. From a specific metrics perspective, I don't think we're going to get into which the numbers of endocrinologists or bariatric surgeons, just to say that we're excited about the results and we're very happy with the engagement from both patients as well as healthcare professionals.

我們正把這項活動、以及「揭開 PBH」帶到醫療生態系中更廣泛的社群。就具體指標而言，我想我們不會細談內分泌科醫師或減重手術外科醫師的人數；只想說我們對成果感到興奮，也非常滿意患者與醫療專業人員的參與度。

Thanks, very helpful.

謝謝，很有幫助。

Thank you. There are no further questions at this time. I will turn the call back to Mr. Klee.

謝謝。目前沒有其他問題。我將把電話交回給 Klee 先生。Klee。

Thank you, operator, and thank you all for your time. If you have any follow-up questions or want to connect, please reach out to our investor relations team. We hope you have a great rest of your day.

謝謝接線員，也謝謝各位撥冗參與。若您有任何後續問題或希望聯繫，請與我們的投資人關係團隊聯絡。祝各位今天剩餘時間愉快。

Ladies and gentlemen, that concludes today's call.

各位女士、先生，今天的電話會議到此結束。

Thank you all for joining. You may now disconnect.

感謝各位參與。您現在可以掛線。