Eton Pharmaceuticals Inc (ETON) 2025 Q3 法說會逐字稿

Good afternoon, and welcome to the Eton Pharmaceuticals Third Quarter 2025 Financial Results Conference Call. (Operator Instructions) Please be advised that this call is being recorded at the company's request. At this time, I'd like to turn it over to David Krempa, Chief Business Officer at Eton Pharmaceuticals. Please proceed.

下午好，歡迎參加伊頓製藥2025年第三季財務業績電話會議。（操作員指示）請注意，本次通話正在依照本公司要求進行錄音。現在，我想把發言權交給伊頓製藥公司的首席商務官大衛克倫帕。請繼續。

Thank you, operator. Good afternoon, everyone, and welcome to Eton's Third Quarter 2025 Conference Call. This afternoon, we issued a press release that outlines the topics we plan to discuss on today's call. The release is available on our website, www.etonpharma.com. Joining me on our call today, we have Sean Brynjelsen, our CEO; James Gruber, our CFO; and Ipek Trinkaus, our Chief Commercial Officer.

謝謝接線生。各位下午好，歡迎參加伊頓公司2025年第三季電話會議。今天下午，我們發布了一份新聞稿，概述了我們計劃在今天的電話會議上討論的主題。新聞稿可在我們的網站 www.etonpharma.com 上查看。今天與我一起參加電話會議的有：我們的執行長 Sean Brynjelsen；我們的財務長 James Gruber；以及我們的商務長 Ipek Trinkaus。

In addition to taking live questions on today's call, we will be answering questions that are e-mailed to us. Investors can send their questions to investorrelations@etonpharma.com.

除了在今天的電話會議上接受現場提問外，我們還將回答透過電子郵件發送給我們的問題。投資者可以將問題發送至 investorrelations@etonpharma.com。

Before we begin, I would like to remind everyone that remarks made during the call may contain forward-looking statements and involve risks and uncertainties that could cause actual results to differ materially from those contained in these forward-looking statements. Please see the forward-looking statements disclaimer in our earnings release and the risk factors in the company's filings with the SEC.

在開始之前，我想提醒大家，電話會議期間發表的言論可能包含前瞻性陳述，並涉及風險和不確定性，這些風險和不確定性可能導致實際結果與這些前瞻性陳述中包含的結果有重大差異。請參閱我們獲利報告中的前瞻性聲明免責聲明以及公司向美國證券交易委員會提交的文件中的風險因素。

Now I will turn the call over to our CEO, Sean Brynjelsen.

現在我將把電話交給我們的執行長肖恩·布林傑爾森。

Thank you, David. Good afternoon, everyone, and thank you for joining us today. I'm thrilled to report another record quarter for the company with triple-digit year-over-year revenue growth. I look forward to discussing the underlying drivers in more detail and highlighting some of our initiatives that help deliver this growth. In addition, we will have made significant progress with our development activities, which are not reflected in this quarter's numbers, but will propel our revenue and earnings growth for many years to come.

謝謝你，大衛。各位下午好，感謝各位今天參加我們的節目。我很高興地宣布，公司又迎來了一個創紀錄的季度，營收年增三位數。我期待更詳細地探討其背後的驅動因素，並重點介紹我們為實現這一增長而採取的一些舉措。此外，我們的研發活動也取得了重大進展，這些進展雖然沒有反映在本季度的數據中，但將在未來許多年推動我們的收入和利潤成長。

Third quarter product revenue was $22.5 million, an increase of 129% year-over-year and up 19% compared to the second quarter. It was our 19th straight quarter of sequential product revenue growth, driven by strong year-over-year growth from ALKINDI SPRINKLE and Carglumic Acid, as well as additions from the recently acquired products, INCRELEX and GALZIN, which are both tracking ahead of our deal models. ALKINDI SPRINKLE has delivered reliable growth for many years and shows no signs of stopping. Carglumic Acid had previously plateaued, but we had a few new patient adds in recent months that helped deliver the year-over-year increase, which was nice to see.

第三季產品營收為 2,250 萬美元，年增 129%，較第二季成長 19%。這是我們連續第 19 個季度實現產品收入環比增長，這主要得益於 ALKINDI SPRINKLE 和 Carglumic Acid 的強勁同比增長，以及最近收購的產品 INCRELEX 和 GALZIN 的新增收入，這兩款產品的業績均超過了我們的交易模型預期。ALKINDI SPRINKLE 多年來一直保持著穩定的成長，並且沒有停止成長的跡象。卡谷氨酸的銷售量先前一直處於停滯狀態，但最近幾個月我們新增了一些患者，這幫助我們實現了同比增長，這令人欣慰。

In addition to delivering on the top line, we remain focused on profitability, and I am pleased to share that we generated $12 million of cash from operations in the quarter. Eton is committed to controlling our expenses, and I am proud to report that even though our revenue is growing rapidly, we were able to reduce our adjusted SG&A expense sequentially from the second quarter to the third quarter. Continued control of our operating expenses in tandem with strong revenue growth will position us for significant margin expansion. We reported adjusted EBITDA of $2.9 million in the quarter, and this figure was weighed down by some nonrecurring Increlex ex U.S. transition costs that James will provide more details on. So we expect to deliver even stronger EBITDA in the quarters ahead.

除了實現營收目標外，我們仍然專注於獲利能力，我很高興地宣布，本季我們從營運中獲得了 1,200 萬美元的現金。伊頓致力於控制我們的支出，我很自豪地報告，儘管我們的收入增長迅速，但我們還是能夠從第二季度到第三季度環比降低調整後的銷售、一般及行政費用。持續控制營運費用，同時維持強勁的收入成長，將使我們的利潤率大幅提升。本季調整後 EBITDA 為 290 萬美元，但這一數字受到一些非經常性 Increlex（除美國外）過渡成本的拖累，James 將對此提供更多細節。因此，我們預計未來幾季將實現更強勁的 EBITDA。

Now turning to product-specific commentary. I'll start with INCRELEX, which has been our largest revenue contributor this year. INCRELEX revenue and patient count continue to track well ahead of our original projections for the product. Prior to our acquisition, the product and the condition has suffered from low awareness. Our efforts to improve education and awareness have paid off, allowing us to deliver significant growth so far this year.

現在轉向產品具體評述。我先從 INCRELEX 開始，它是我們今年最大的收入貢獻者。INCRELEX 的收入和患者數量繼續遠遠超過我們最初的產品預測。在我們收購之前，該產品和這種疾病的認知度很低。我們為提高教育水平和公眾意識所做的努力已經取得了成效，使我們今年迄今為止實現了顯著增長。

Our commercial team has done an excellent job on the relaunch through our rare disease specialists' outreach to health care providers, our conference engagements, and peer-to-peer presentations, as well as collaborating closely with patients and patient advocacy groups, we have been able to substantially grow awareness and increase product usage in a matter of months.

我們的商業團隊透過罕見疾病專家與醫療保健提供者的溝通、參加會議、進行同行交流，以及與患者和患者權益倡導團體的密切合作，在產品重新上市方面做得非常出色，我們在短短幾個月內就大幅提高了人們對產品的認識，並增加了產品的使用量。

When Eton took the product over in December 2024, there were only 67 active patients on therapy. By August, we shared that we had reached our 100-patient goal five months ahead of schedule. We continue to add a number of new patient starts during the last three months, but we saw a higher number of patients age out and discontinued treatment during the same period, which resulted in our net active patient count remaining relatively flat around 100.

2024 年 12 月 Eton 接手該產品時，只有 67 名活躍患者正在接受治療。到八月份，我們宣布我們提前五個月達到了收治 100 名患者的目標。在過去的三個月裡，我們新增了不少患者，但同時，也有相當數量的患者因年齡增長而停止治療，導致我們的淨活躍患者人數保持在 100 人左右。

In severe primary insulin growth like Factor 1 deficiency, success is partially measured not only by how many patients are on therapy, but in addition, what truly drives outcomes is how early the treatment begins and how well it's optimized. Early initiation during the critical growth window and appropriate vial utilization are key to maximizing efficiency during the treatment duration.

對於像因子 1 缺乏症這樣的嚴重原發性胰島素增生，治療的成功不僅取決於有多少患者接受治療，而且真正決定治療結果的因素是治療開始得有多早以及治療的優化程度如何。在關鍵生長窗口期儘早開始治療並合理使用藥瓶是提高治療期間效率的關鍵。

Since we have inherited several older pediatric patients in December during transition, we saw a large group of age-outs coming through from that cohort. Our focus remains on both expanding new patient starts and driving growth through earlier diagnosis and optimized dosing to ensure every patient achieves their full therapeutic potential. We believe these efforts will increase the average duration of treatment. I expect to continue bringing new patients into treatment and continue growing the net patient count. As a reminder, INCRELEX is approved for pediatric patients aged two and up with severe primary IGF-1 deficiency.

由於我們在 12 月過渡期間接收了幾位年齡較大的兒科患者，我們看到該群體中出現了一大批因年齡限製而退出治療的患者。我們將繼續致力於擴大新患者的治療規模，並透過早期診斷和優化劑量來推動成長，以確保每位患者都能充分發揮其治療潛力。我們相信這些努力將延長平均治療時間。我預計會繼續招收新患者，並繼續增加患者總數。再次提醒，INCRELEX 已獲準用於治療 2 歲及以上患有嚴重原發性 IGF-1 缺乏症的兒科患者。

These are patients who present with extremely short stature and need IGF supplementation to grow. INCRELEX is very effective in increasing patient height during their growing years, but it's no longer needed once patients reach their adult height, which is typically around 18 years old. We believe with our ongoing educational and awareness campaigns, we will start seeing patients diagnosed earlier in life, which would likely lead to a longer duration of therapy.

這些患者身材極為矮小，需要補充IGF才能長高。INCRELEX 在患者生長發育期能非常有效地增加其身高，但一旦患者達到成年身高（通常在 18 歲左右），就不再需要它了。我們相信，透過持續教育和宣傳活動，我們將看到更多患者在更年輕的時候被診斷出患有這種疾病，這可能會導致更長的治療時間。

Eton is confident in the long-term growth opportunity for the product. And as we expect to continue converting more of the estimated 200 patients in the U.S. that meet the current label. In addition, we remain committed to expanding access to even more children in need through the harmonization of the U.S. and EU definitions of severe primary 1 deficiency.

伊頓對該產品長期的成長前景充滿信心。我們預計將繼續將美國約 200 名符合目前標籤的患者轉為其他患者。此外，我們將繼續致力於透過協調美國和歐盟對嚴重原發性 1 營養不良的定義，擴大更多有需要的兒童獲得治療的機會。

Last month, we submitted a meeting request to the FDA with our proposed clinical study to support the harmonization. We expect to have the FDA's feedback by the end of December. And if they are in agreement, we would initiate the study in 2026.

上個月，我們向 FDA 提交了會議請求，並提出了支持協調的臨床研究方案。我們預計將在12月底前收到FDA的回饋意見。如果他們同意，我們將在 2026 年啟動這項研究。

Given the European patient registry data that has been collected over the last 15 years, we believe that INCRELEX is a safe and effective treatment for patients with IGF-1 levels between minus 2 and minus 3 standard deviations. We are confident our proposed study would confirm that for the FDA. And if successful in harmonizing the labels, it could potentially increase the INCRELEX market opportunity roughly fivefold.

根據過去 15 年收集的歐洲患者登記數據，我們認為 INCRELEX 對於 IGF-1 水平在 -2 到 -3 個標準差之間的患者來說是一種安全有效的治療方法。我們相信，我們提出的研究方案將向F​​DA證實這一點。如果標籤統一化成功，INCRELEX 的市場機會可能會增加約五倍。

ALKINDI was another major contributor to our Q3 revenue growth, and I am proud of the team's ability to continue generating consistent growth. As you remember, starting in January, we split our sales force into two teams, one of which, which is now 100% dedicated to pediatric endocrinology. We think has contributed to ALKINDI SPRINKLE's strong year, and 2025 is the product's fifth calendar year on market and remains on pace to be the strongest year of its history by number of patients on therapy and number of new patient referrals. So far, we have not been seeing much of any, cannibalization of ALKINDI from the launch of KHINDIVI.

ALKINDI 是我們第三季營收成長的另一個主要貢獻者，我為團隊能夠持續創造穩定成長而感到自豪。如您所知，從一月開始，我們將銷售團隊分成兩個團隊，其中一個團隊現在 100% 專注於兒科內分泌學。我們認為這促成了 ALKINDI SPRINKLE 的強勁表​​現，2025 年是該產品上市的第五個日曆年，並且按接受治療的患者人數和新患者轉診人數計算，有望成為其歷史上表現最強勁的一年。到目前為止，我們還沒有看到 KHINDIVI 的推出對 ALKINDI 造成任何蠶食。

Though ALKINDI continues to see strong growth, we developed and launched KHINDIVI to address the needs of patients that did not like the texture of the ALKINDI granules or prefer the convenience of a liquid dosage form. KHINDIVI is the first and only FDA-approved oral solution of hydrocortisone. KHINDIVI allows simple and accurate dosing tailored to patient needs and does not require refrigeration, mixing, or shaking. The FDA approved KHINDIVI for patients five and over. The agency restricted the age due to a limited amount of existing safety data on three of the inactive ingredients in the formulation when being used in combination.

儘管 ALKINDI 繼續保持強勁成長，但我們開發並推出了 KHINDIVI，以滿足那些不喜歡 ALKINDI 顆粒劑質地或更喜歡液體劑型便利性的患者的需求。KHINDIVI 是第一個也是唯一一個獲得 FDA 批准的氫化可的鬆口服溶液。KHINDIVI 可根據患者需求進行簡單、準確的劑量調整，無需冷藏、混合或搖晃。FDA 已批准 KHINDIVI 用於 5 歲及以上患者。由於配方中三種非活性成分組合使用時的安全性數據有限，該機構限制了其適用年齡。

Unfortunately, the largest unmet need for this product is among young children under five years old. And as a result, the label restriction has weighed on the adoption of KHINDIVI. However, our team has been working on a plan to address this. When we first heard of the FDA's restriction this summer, we immediately developed a new formula with substantially lower levels of the excipients. And in September, we held a meeting with the FDA to discuss this new formulation.

遺憾的是，目前對該產品需求未被滿足的最大族群是五歲以下的幼兒。因此，標籤限制對 KHINDIVI 的普及造成了影響。然而，我們的團隊一直在製定計劃來解決這個問題。今年夏天我們第一次聽到FDA的限制時，我們立即開發了一種新配方，其中賦形劑的含量大幅降低。9 月份，我們與 FDA 舉行了會議，討論這項新配方。

We believe the meeting was successful as the FDA indicated they would be receptive to a label expansion with our revised formulation. In response, we will conduct a bioequivalency study, which is scheduled to start by January 2026, and I expect to submit the new formulation as a supplement to our existing NDA in the second quarter of 2026.

我們認為這次會議取得了成功，因為FDA表示他們願意接受我們修訂配方後的標籤擴展申請。對此，我們將進行生物等效性研究，該研究計劃於 2026 年 1 月開始，我預計將於 2026 年第二季將新配方作為我們現有新藥申請的補充提交。

The FDA indicated a 10-month review for the formulation, so this could allow for an approval by the first quarter of 2027. We believe this label expansion would significantly accelerate adoption of the product. Even with the current KHINDIVI label, we continue to see attractive long-term growth for our adrenal insufficiency franchise. Eton has only converted less than 15% of the estimated 5,000 target patients in the United States. So we see a long runway of growth ahead of us. We remain confident that ALKINDI and KHINDIVI can combine for peak sales of more than $50 million with the current KHINDIVI label and ultimately higher levels if the label is expanded.

FDA表示將對該配方進行10個月的審查，因此有可能在2027年第一季獲得批准。我們相信，此次標籤擴展將顯著加快該產品的普及速度。即使使用目前的 KHINDIVI 品牌，我們仍然看到腎上腺功能不全產品線具有良好的長期成長前景。伊頓療法在美國約 5000 名目標患者中僅轉化了不到 15%。因此，我們看到未來還有很長的成長之路。我們仍然相信，ALKINDI 和 KHINDIVI 合併後，光是 KHINDIVI 品牌就能實現超過 5,000 萬美元的峰值銷售額，如果該品牌擴大規模，最終銷售額還能達到更高的水平。

Another bright spot in our portfolio this quarter was GALZIN. As I mentioned, we're extremely pleased with this performance. It now has over 200 active patients, a number we originally set as our year-end 2025 target. The product is continuing to grow well ahead of our original expectations, and we couldn't be happier with the team's efforts to support this relaunch. During our eight months in the field actively commercializing GALZIN, we've been surprised by the low level of awareness that the product had both among physicians and patients.

本季我們投資組合中的另一個亮點是 GALZIN。正如我之前提到的，我們對這次演出非常滿意。目前該機構擁有 200 多名活躍患者，而我們最初設定的 2025 年底目標正是達到這個數字。該產品持續成長，遠遠超出我們最初的預期，我們對團隊為支持此次重新上市所做的努力感到無比滿意。在過去八個月積極推廣 GALZIN 的過程中，我們驚訝地發現，無論是醫生還是患者，對該產品都缺乏足夠的了解。

Even though GALZIN is the only FDA-approved zinc therapy for Wilson disease, many patients and prescribers were unaware of it, misinformed, or mistakenly believe the product was discontinued after a prior shortage in 2020 and subsequent lack of promotion. We view this low awareness as a positive for the long-term growth prospects for GALZIN.

儘管 GALZIN 是唯一獲得 FDA 批准用於治療威爾遜病的鋅療法，但許多患者和處方醫生對此並不知情，或者被誤導，或者錯誤地認為該產品在 2020 年出現短缺後，由於缺乏推廣而停產。我們認為這種低認知度對 GALZIN 的長期成長前景來說是件好事。

While we have work to do educating the market, it is clear that this represents a substantial growth opportunity as we inform patients, healthcare practitioners, and caregivers, and raise awareness of this critical medication. Our entry into Wilson disease has been warmly received by patients and health care providers. Before our relaunch, very few pharmacies stocked GALZIN, out-of-pocket costs were high, and there was a lack of support services to help patients navigate the insurance process. We have now implemented full patient support services, increased access to medication, and substantially reduced out-of-pocket costs for patients. These changes have resonated with the patient community, and we have heard strong positive feedback and appreciation for the new programs.

雖然我們還需要努力進行市場教育，但很明顯，這代表著一個巨大的成長機會，因為我們可以向患者、醫療保健從業人員和護理人員提供信息，並提高人們對這種重要藥物的認識。我們進入威爾遜病領域後，受到了患者和醫療保健提供者的熱烈歡迎。在我們重新推出產品之前，很少藥局備有 GALZIN，自付費用很高，而且缺乏支援服務來幫助病患了解保險流程。我們現在已經實施了全面的患者支援服務，增加了患者獲得藥物的途徑，並大幅降低了患者的自付費用。這些改變引起了患者群體的共鳴，我們收到了對新項目的強烈正面回饋和讚賞。

In October, our team attended the Wilson Disease Association Annual Summit, where patients, caregivers, and leading physicians gathered to discuss diagnosis, treatment, and management of the disease. Our team was able to engage with numerous patients and prescribers, helping to drive awareness and give us the chance to better understand the struggles that patients and prescribers are dealing with. Working to understand the needs of patients, caregivers, and health care providers is a top priority for us and our vision to be a champion of those in the Wilson disease community. Our expanded access and patient support services have made a major impact on Wilson disease patients, but we think we can make an even greater impact on their lives with ET-700, our extended-release version of GALZIN.

10 月，我們的團隊參加了威爾遜病協會年度高峰會，患者、護理人員和頂尖醫生齊聚一堂，討論該疾病的診斷、治療和管理。我們的團隊能夠與眾多患者和處方醫生進行交流，這有助於提高人們的認識，並讓我們有機會更好地了解患者和處方醫生正在面臨的困難。了解患者、照護者和醫療保健提供者的需求是我們的首要任務，我們的願景是成為威爾遜病群體的擁護者。我們擴大了獲取途徑和患者支持服務，這對威爾遜病患者產生了重大影響，但我們認為，透過 ET-700（GALZIN 的緩釋版本），我們可以對他們的生活產生更大的影響。

Currently, GALZIN is taken three times per day with patients fasting both before and after, and this cumbersome regimen leads to high rates of noncompliance. Eton has heard directly from patients and caregivers just how challenging the current dosing schedule is, and we know there's a very strong interest in an extended-release version. Eton is working quickly and making meaningful progress with our development of ET-700. We've already developed a proprietary formulation, filed our patents, and met with the FDA to discuss the regulatory pathway. We are now nearing production of clinical study supply and starting our clinical program with our Positron Emission Tomography or PET study scheduled to begin in the first quarter.

目前，GALZIN 每天服用三次，患者在服藥前後均需空腹，這種繁瑣的治療方案導致患者依從性差。伊頓公司直接從病人和照護人員那裡了解到，目前的給藥方案有多麼具有挑戰性，我們也知道大家對緩釋版本有著非常濃厚的興趣。Eton 正在快速推進 ET-700 的研發，並取得了實質進展。我們已經開發出專有配方，提交了專利申請，並與美國食品藥物管理局 (FDA) 會面討論了監管途徑。我們現在即將開始臨床研究用品的生產，並將啟動我們的臨床項目，正子斷層掃描（PET）研究計劃於第一季開始。

This study is a proof-of-concept study designed to verify that our proprietary delayed-release formulation is able to effectively block copper absorption in patients with less frequent dosing. We expect to receive top-line results from this study in the middle of 2026. And if positive, it would support the initiation of a dose-ranging and pivotal clinical study later in the year.

本研究是一項概念驗證研究，旨在驗證我們專有的緩釋配方能夠有效阻止患者在減少給藥頻率的情況下吸收銅。我們預計將於 2026 年年中收到這項研究的初步結果。如果結果為陽性，則將支持在今年稍後啟動劑量範圍和關鍵性臨床研究。

Switching back to our pediatric endocrinology portfolio. During the quarter, we had another piece of good news when the FDA accepted our ET-600 NDA submission for review and assigned a February 25 PDUFA date. We developed ET-600 in direct response to an unmet need expressed by pediatric endocrinologists for an oral solution of desmopressin to treat central diabetes insipidus. If approved, ET-600 would be the first oral liquid formulation available and would allow for the small precise titratable doses required to treat pediatric patients. The review of the product appears to be proceeding well, and we scheduled the production of inventory at risk in preparation for an anticipated commercial launch shortly after the PDUFA target action date.

切換回我們的兒科內分泌學產品組合。本季度，我們又迎來了一個好消息，FDA 接受了我們提交的 ET-600 NDA 申請進行審查，並指定了 2 月 25 日的 PDUFA 日期。我們開發 ET-600 的直接原因是兒科內分泌學家提出了一種用於治療中樞性尿崩症的口服去氨加壓素溶液的未滿足需求。如果獲得批准，ET-600 將成為首個上市的口服液體製劑，並可實現治療兒科患者所需的小劑量精確滴定。該產品的審查似乎進展順利，我們已安排風險庫存的生產，為在 PDUFA 目標行動日期之後不久的預期商業發布做準備。

Prelaunch marketing activities, including key thought leader engagements, advisory boards, and patient focus groups are also underway. We recently held an ET-600 advisory board with key opinion leaders at the National Endo Conference. We continue to hear positive feedback and strong excitement for the product. Since ET-600 shares the same pediatric endocrinology call points as ALKINDI, KHINDIVI, and INCRELEX, Eton can leverage our well-established relationships and existing commercial footprint, and we expect to be able to hit the ground running upon launch next year. Given the growth opportunity ahead for our commercial products and the attractive pipeline and label expansion opportunities discussed today, it is clear that our business is set up for very attractive long-term growth for many years to come. However, we believe that we can accelerate our growth through additional business development transactions.

上市前的市場推廣活動，包括與重要思想領袖的互動、顧問委員會和病患焦點小組，也正在進行中。我們最近在全國內分泌學大會上與主要意見領袖舉行了 ET-600 諮詢委員會會議。我們不斷收到正面的回饋，大家對這款產品也表現出極大的熱情。由於 ET-600 與 ALKINDI、KHINDIVI 和 INCRELEX 共享相同的兒科內分泌學呼叫點，Eton 可以利用我們已建立的良好關係和現有的商業佈局，我們預計明年推出後就能迅速開展業務。鑑於我們商業產品的未來成長機遇，以及今天討論的極具吸引力的產品線和標籤擴展機會，很明顯，我們的業務已做好準備，在未來許多年內實現非常有吸引力的長期增長。但是，我們相信可以透過更多的業務拓展交易來加速我們的成長。

I remain confident that we have the necessary skills and capabilities to execute value-creating acquisitions and believe that our track record speaks for itself. We continue to explore opportunities to acquire additional strategically aligned ultra-rare disease products where Eton is positioned to add value. With $37 million in cash on our balance sheet and a diversified growing business that is already generating strong EBITDA, we have plenty of capacity to finance acquisitions, large or small. We'll continue to approach opportunities from a position of strength and with our customary discipline.

我仍然堅信我們擁有執行創造價值收購所需的技能和能力，並且相信我們的過往業績足以證明這一點。我們將繼續探索收購更多與 Eton 策略契合的超罕見疾病產品的機會，Eton 在這些產品領域能夠創造價值。我們資產負債表上有 3,700 萬美元現金，業務多元化且不斷成長，已經產生了強勁的 EBITDA，因此我們有足夠的能力為收購提供資金，無論收購規模大小。我們將繼續以強大的實力和一貫的嚴謹作風迎接各種機會。

2025 has been a transformational year for us, highlighted by three high-value commercial product launches, record levels of product sales and profitability, and the submission of an NDA for ET-600. We continue to push full speed ahead to close out the year strong and position us for an even more impressive 2026. Next year, we expect a number of critical milestones, including continued strong revenue growth from ALKINDI SPRINKLE, INCRELEX, GALZIN, and KHINDIVI, increased profitability and operating margin expansion, the expected launch of ET-600, the submission of our revised formulation of KHINDIVI, the completion of our ET-700 pilot study, and the initiation of our INCRELEX label harmonization clinical study.

2025 年對我們來說是具有變革意義的一年，亮點包括三款高價值商業產品的推出、產品銷售額和盈利能力的創紀錄水平，以及提交 ET-600 的 NDA。我們將繼續全力以赴，力爭以強勁的勢頭結束今年，並為2026年取得更輝煌的成就做好準備。明年，我們預計將實現多個關鍵里程碑，包括 ALKINDI SPRINKLE、INCRELEX、GALZIN 和 KHINDIVI 的持續強勁收入增長、盈利能力和營業利潤率的提高、ET-600 的預期上市、KHINDIVI 修訂配方的提交、ET-700 試點研究的完成以及 INLEX 標籤協調臨床研究的啟動。

As you can see, we have some very exciting and event-filled quarters ahead of us, and we look forward to keeping all of you up to date on our progress. We thank you for your continued support.

如您所見，我們未來幾季將充滿令人興奮和精彩的活動，我們期待隨時向大家報告我們的進展。感謝您一直以來的支持。

And with that, I'll hand it over to James, our Chief Financial Officer, to discuss the financials. James?

接下來，我將把發言權交給我們的財務長詹姆斯，讓他來討論財務狀況。詹姆斯？

Thank you, Sean. Our third-quarter revenue increased 118% to $22.5 million compared to $10.3 million in the third quarter of 2024, and revenue was primarily comprised of product sales in both periods. Third quarter revenue included $0.9 million of product revenue from the sale of finished product inventory to Ipsen and Esteve to facilitate the ownership transition of INCRELEX in certain European countries, and these sales are expected to be non-recurring.

謝謝你，肖恩。與 2024 年第三季的 1,030 萬美元相比，我們第三季的營收成長了 118%，達到 2,250 萬美元，這兩個時期的營收主要都來自產品銷售。第三季收入包括 0.9 億美元產品收入，該收入來自向 Ipsen 和 Esteve 出售成品庫存，以促進 INCRELEX 在某些歐洲國家的股權轉讓，預計這些銷售將不會經常發生。

In addition, $2.4 million of revenue was derived from an initial loading order of semi-finished INCRELEX inventory for Esteve. When Eton out-licensed the rights to ex-U.S. INCRELEX, it entered into a long-term supply agreement with Esteve, under which Eton will provide semi-finished goods to Esteve at a fixed transfer price. The company expects these ongoing purchases to produce roughly $2 million to $3 million of annual revenue. However, the ordering patterns may be inconsistent and not occur every quarter.

此外，Esteve 的一筆 INCRELEX 半成品庫存初始裝載訂單帶來了 240 萬美元的收入。當 Eton 將權利授權給美國前 INCRELEX 公司時，它與 Esteve 公司簽訂了一份長期供應協議，根據該協議，Eton 將以固定的轉讓價格向 Esteve 公司提供半成品。該公司預計這些持續採購每年將產生約 200 萬至 300 萬美元的收入。然而，訂購模式可能不穩定，並非每季都會發生。

Revenue growth in the quarter was driven primarily by increased sales of ALKINDI SPRINKLE and Carglumic acid, plus the addition of sales from INCRELEX and GALZIN. While Sean mentioned that the INCRELEX net active patient count was relatively flat, we saw a less favorable payer mix in the third quarter, which resulted in lower revenue per patient compared to the second quarter. Eton expects U.S. product sales to continue to grow sequentially in the fourth quarter compared to the third quarter. But given that some of the third quarter INCRELEX-related ex U.S. revenue is not expected to recur, total product sales may be flat or slightly decline in Q4 relative to Q3.

本季營收成長主要得益於 ALKINDI SPRINKLE 和卡麩胺酸的銷售增加，以及 INCRELEX 和 GALZIN 的銷售成長。雖然 Sean 提到 INCRELEX 的淨活躍患者數量相對平穩，但我們看到第三季的支付方組合不太有利，導致每位患者的收入比第二季有所下降。伊頓預計，與第三季相比，第四季美國產品銷售額將繼續環比成長。但考慮到第三季與 INCRELEX 相關的美國以外收入中的一部分預計不會再次發生，因此第四季產品總銷售額可能與第三季持平或略有下降。

Cost of sales for the third quarter was $14.6 million compared to $4.0 million in the third quarter of 2024, an increase of $10.6 million, driven by increased sales volumes and approximately $7.4 million of costs associated with the transition of the ex-U.S. distribution of INCRELEX. Adjusted gross profit was $10.2 million in the third quarter, representing an adjusted gross margin of 45% compared to adjusted gross profit of $6.6 million and adjusted gross margin of 64% in the prior year period. Adjusted gross margin in the quarter was negatively impacted by INCRELEX ex U.S. related costs, including the transition of ex U.S. distribution and the supply agreement with Esteve. The company expects to report fourth-quarter adjusted gross margin of approximately 70%.

第三季的銷售成本為 1,460 萬美元，而 2024 年第三季為 400 萬美元，增加了 1,060 萬美元，而 2024 年第三季為 400 萬美元，增加了 1,060 萬美元，這主要是由於銷售量增加以及與 INCRELEX 在美國以外地區分銷過渡相關的約 740 萬美元成本所致。第三季調整後毛利為 1,020 萬美元，調整後毛利率為 45%，而去年同期調整後毛利為 660 萬美元，調整後毛利率為 64%。本季調整後的毛利率受到 INCRELEX 美國以外地區相關成本的負面影響，包括美國以外地區分銷的過渡以及與 Esteve 的供應協議。該公司預計第四季調整後毛利率約為 70%。

R&D expenses for the quarter were $1.1 million, an increase of $0.6 million compared to the prior year period due primarily to increased expenses associated with our ET-700 and ET-800 development activities. General and administrative expenses for the quarter were $8.1 million compared with $5.3 million in the prior year period due primarily to an increase in product advertising and launch year promotional expenses, higher stock-based compensation expense, and an increase in compensation and benefit expenses due to an increase in general and administrative headcount. General and administrative expenses were down $1.6 million compared to the second quarter of 2025.

本季研發費用為 110 萬美元，比去年同期增加了 60 萬美元，主要是由於與我們的 ET-700 和 ET-800 開發活動相關的費用增加所致。本季一般及行政費用為 810 萬美元，而上年同期為 530 萬美元，主要原因是產品廣告和上市年度促銷費用增加、股票選擇權費用增加，以及由於一般及行政人員數量增加而導致的薪酬和福利費用增加。一般及行政費用較 2025 年第二季減少了 160 萬美元。

On an adjusted basis, which removes the impact of share-based compensation, transaction-related costs, and other one-time expenses, G&A expense was $6.9 million compared to $4.3 million in the prior year period and $7.6 million in the second quarter of 2025, and we were pleased to see this sequential decline in spending. As we have discussed previously, the first half of this year had increased G&A expenses associated with our three product launches, and we expect adjusted G&A spending in the second half of the year to remain materially lower.

經過調整後（剔除了股權激勵、交易相關成本和其他一次性支出的影響），一般及行政費用為 690 萬美元，而去年同期為 430 萬美元，2025 年第二季為 760 萬美元，我們很高興看到支出環比下降。正如我們之前討論過的，今年上半年由於我們推出了三款產品，一般及行政費用有所增加，我們預計下半年的調整後一般及行政支出將大幅下降。

Adjusted EBITDA for the third quarter of 2025 was $2.9 million compared to $2.0 million in the third quarter of 2024. Total company net loss was $1.9 million for the quarter compared to net income of $0.6 million in the prior year period. Net loss per basic and diluted share during the quarter was $0.07 compared to a net income per basic and diluted share of $0.02 in the prior year period.

2025 年第三季調整後 EBITDA 為 290 萬美元，而 2024 年第三季為 200 萬美元。該季度公司淨虧損總額為 190 萬美元，而去年同期淨利潤為 60 萬美元。本季每股基本及攤薄淨虧損為 0.07 美元，而去年同期每股基本及攤薄淨收益為 0.02 美元。

On a non-GAAP basis, we reported net income of $1.5 million for the third quarter of 2025 compared to $1.9 million in the prior year period and diluted earnings per share of $0.04 for the third quarter of 2025 compared to $0.07 per share in the prior year period. Eton finished the third quarter with $37.1 million in cash on hand, and we generated $12.0 million in operating cash flow during the quarter. This includes a $4.3 million payment received from Esteve for the international rights to INCRELEX.

以非GAAP準則計算，我們報告2025年第三季淨收入為150萬美元，而上年同期為190萬美元；2025年第三季稀釋後每股收益為0.04美元，而去年同期為每股0.07美元。伊頓公司第三季末現金餘額為 3,710 萬美元，該季度我們產生了 1,200 萬美元的營運現金流。其中包括從 Esteve 收到的 430 萬美元，用於購買 INCRELEX 的國際版權。

This concludes our remarks on third-quarter results. And with that, we'll turn it back over to the operator for Q&A.

以上就是我們對第三季業績的評論。接下來，我們將把問答環節交還給接線生。

(Operator Instructions) Our first question comes from the line of Chase Knickerbocker of Craig-Hallum.

（操作說明）我們的第一個問題來自 Craig-Hallum 的 Chase Knickerbocker 的一條生產線。

James, maybe just first, a quick one. If you back out those -- that $2 million to $3 million in OUS kind of related revenue on those inventory shipments and then the associated costs that got into COGS, can you just give us what kind of, call it, pro forma gross margins would be kind of on the core U.S. business would have been -- sorry.

詹姆斯，或許先來一個，簡單點。如果扣除那些——與庫存發貨相關的 200 萬至 300 萬美元的海外收入，以及計入銷售成本的相關成本，您能否告訴我們，核心美國業務的預計毛利率是多少？抱歉。

Sure. So adjusted the GAAP gross margins with all that -- with the ex U.S. INCRELEX activity in there was 35%. Adjusted was 45%. And if we remove all of that ex-U.S. activity, it's north of just over 70% for the quarter.

當然。因此，根據所有這些因素調整了 GAAP 毛利率——其中不包括美國 INCRELEX 業務活動，毛利率為 35%。調整後為 45%。如果剔除所有美國以外的活動，該季度佔比將略高於 70%。

And then, Sean, maybe just as we think about that reacceleration for ALKINDI, is it truly just that kind of refocusing of the sales force kind of solely on PDENO? Or are there kind of other drivers that you would point to as far as kind of how that sequential revenue growth has accelerated so far through '25?

那麼，肖恩，也許當我們考慮 ALKINDI 的重新加速發展時，這真的只是銷售團隊將重點完全轉移到 PDENO 上嗎？或者，您認為還有哪些因素可以解釋為什麼到 2025 年為止，收入的連續成長速度有所加快？

I think the big lever certainly was the focus of the PDENO group. Secondary aspect, I would say our physicians are comfortable with the product. They know it works. It's a product that has early adopters, we've got late adopters, and we're seeing a lot of late adopters and those who took a wait-and-see attitude now they believe in it. And I would say that we'll continue to add patients for the foreseeable future.

我認為，PDENO 小組的重點肯定在於大槓桿。其次，我認為我們的醫生對該產品感到滿意。他們知道這招管用。這款產品既有早期採用者，也有後期採用者，而且我們看到許多後期採用者和那些原本持觀望態度的人現在也相信它了。而且我認為，在可預見的未來，我們將繼續增加患者數量。

It's not a perfect product. That's why we came out with the liquid version. And so we've got -- we want to be able to offer that, and we think that will really jump-start the growth next year. But right now, it's a steady increase in ALKINDI patients in addition to the KHINDIVI. As we said during the call, we don't see a lot of cannibalizations. Really, it's additive.

它並非完美無缺的產品。這就是我們推出液態版本的原因。因此，我們希望能夠提供這項服務，我們認為這將真正推動明年的成長。但目前，除了 KHINDIVI 患者外，ALKINDI 患者也在穩定增加。正如我們在通話中所說，我們並沒有看到很多蠶食現象。確實，它會上癮。

And then maybe just on INCRELEX. First, could you just, if you wouldn't mind, give that gross adds number since August, just so we can kind of get a sense for demand generation? And then just second, on INCRELEX and additional thoughts or details that you can give us as far as that trial design that you submitted to FDA that we're waiting to hear feedback on kind of timelines, number of patients, that sort of thing, as far as how you're thinking?

然後或許就只在 INCRELEX 上。首先，如果您不介意的話，能否提供一下自 8 月以來的新增用戶總數，以便我們大致了解需求情況？其次，關於 INCRELEX，您能否就您提交給 FDA 的試驗設計提供一些補充想法或細節？我們正在等待 FDA 的回饋，例如時間表、患者人數等等，您有什麼想法？

So on the numbers, we're roughly where we were at on our last call, and it had to do with the number of ads, but then we had a number of folks go off, but now we're seeing more ads. We just saw a number of ads just the past week in terms of new scripts. So we're going to see if we can hit that 110 number by the end of next month. And -- but I would say that we're very pleased with the product overall. It's -- we knew it was going to slow down a little bit, but it's a little bit lumpy in terms of when people come on and off the product.

所以從數字上看，我們和上次通話時的情況大致相同，這與廣告數量有關，但當時有不少人退出了，但現在我們看到更多的廣告。就在上週，我們看到了許多新劇本的廣告。所以我們要看看能否在下個月底達到 110 這個數字。不過，總的來說，我們對這款產品非常滿意。我們知道它的速度會稍微放緩一些，但就人們開始使用和停止使用產品的時間而言，它有點不穩定。

We had that significant increase in Q1 and going a little bit into Q2. So that's that. And then regarding the clinical, we've submitted it. We expect to get feedback from the FDA in the coming weeks. And I do think that, that will be favorable. And hopefully, we can start enrolling patients in the first half of next year.

第一季以及第二季初，我們實現了顯著成長。事情就是這樣。至於臨床方面，我們已經提交了。我們預計將在未來幾週內收到美國食品藥物管理局（FDA）的回饋。而且我認為這會是一件好事。希望我們能在明年上半年開始招募病患。

Last one for me. Maybe just as we look -- start to look into 2026 as you guys prepare your budget, any initial thoughts that you'd be willing to give us just as far as how you're thinking about top-line growth next year? It looks like -- the Street is somewhere kind of mid- to high 20s as far as top line growth goes from a percentage perspective. I mean do you have any initial thoughts that you'd be willing to give on '26?

這是我最後一個了。或許我們可以趁著你們開始展望 2026 年並準備預算的時候，談談你們對明年營收成長的初步想法？從百分比的角度來看，華爾街對營收成長的預期大概在 20% 到 30% 之間。我的意思是，你對26號有什麼初步想法可以分享嗎？

Sure. I'll let David answer that one.

當然。這個問題就讓大衛來回答吧。

As we said on the prepared remarks, we expect significant growth to continue for INCRELEX, GALZIN, ALKINDI, KHINDIVI. So we're expecting healthy growth, but we're not going to get into any directional guidance yet. When we report our Q4 numbers, we will have something to share with you.

正如我們在準備好的演講稿中所說，我們預計 INCRELEX、GALZIN、ALKINDI、KHINDIVI 將繼續保持顯著成長。因此，我們預期公司將保持健康成長，但目前我們不會給予任何方向性指引。當我們公佈第四季度業績時，我們會有一些資訊與大家分享。

Our next question comes from the line of Madison El-Saadi of B. Riley.

我們的下一個問題來自 B. Riley 的 Madison El-Saadi 家族。

Congrats on the progress and multiple positive updates. Question about the INCRELEX U.S. registry. Would this take place at the same sites that are active in the global registry trial? There are a few sites in that global registry that are U.S. based.

恭喜取得的進展和許多積極的更新。關於 INCRELEX 美國註冊的問題。這項試驗是否會在與全球註冊試驗相同的地點進行？這全球註冊機構中有一些網站位於美國。

Yes, Madison, it would be just the U.S. It would just be U.S. sites. We would not be enrolling folks overseas.

是的，麥迪遜，僅限於美國。僅限於美國網站。我們不會招募海外人士。

Would it be at separate sites that are activated in the global registry? I think there are about seven U.S. sites that are active as part of that global registry.

是否會在全球註冊表中啟動不同的網站上進行？我認為大約有七個美國網站是該全球註冊機構的活躍成員。

It would probably be different sites, Madison, if one of those sites did have a meaningful number of patients within that negative two to negative three standard deviation, we would consider adding them, but it will probably be different sites within the U.S.

麥迪遜，如果某個站點有相當數量的患者處於負二到負三標準差範圍內，我們會考慮將其納入研究範圍，但很可能是美國境內的不同站點。

And then maybe if you could comment on how -- partner, how you're ranking the potential business development opportunities as we look to the end of the year and even into kind of next year and beyond?

那麼，您能否就以下幾點發表一下看法——合作夥伴，您是如何評估今年年底乃至明年及以後的潛在業務發展機會的？

Well, we -- I would say right now, they're strong. We're in late discussions. We've been in late-stage discussions with two parties, and we're hoping to get something done before the end of the year. If not, it would be shortly thereafter. Obviously, nothing is done until you sign, but these are ultra-rare disease products.

嗯，我們——我想說，現在他們很強大。我們正在進行最後的討論。我們已經與兩方進行了後期磋商，希望能在年底前達成協議。如果不是，那也很快就會發生。顯然，在你簽字之前什麼都不會發生，但這些都是極為罕見疾病產品。

The late stage, a very good strategic fit. We think they would add appreciable revenue over the next 12 to 24 months. So -- and we'll see what happens, but that's always been a core part of our strategy as a company is to take on the right acquisitions.

後期階段，這是一個非常好的策略契合點。我們認為它們將在未來 12 至 24 個月內帶來可觀的收入。所以——我們拭目以待，但進行正確的收購一直是公司策略的核心部分。

Obviously, we don't just do acquisitions for the sake of doing acquisitions. They have to be the right fit. And with or without the acquisitions, we're going to continue to grow. We've got a good pipeline of internal products, but I believe we will close transactions that we will end up with, I'll say, at least two additional product launches next year.

顯然，我們不會為了收購而收購。他們必須是合適的人選。無論是否進行收購，我們都將持續成長。我們擁有良好的內部產品線，但我相信我們將完成一些交易，最終明年至少會推出兩款新的產品。

Our next question comes from the line of Swayampakula Ramakanth of H.C. Wainwright.

我們的下一個問題來自 H.C. Wainwright 的 Swayampakula Ramakanth 的系譜。

Quick question on INCRELEX. You said some -- there were some patients who discontinued as you are putting on some patients. So generally, what are the reasons for the discontinuation? And is there anything either your salesforce or some amount of detailing, additional detailing needed for kind of stopping that getting off the drug?

關於 INCRELEX 的簡短問題。你說過有些人──有些病人因為你們正在接收一些病人而中途退出了治療。那麼，一般來說，停產的原因是什麼？你們的銷售團隊是否需要提供一些細節方面的額外信息，以阻止客戶戒掉毒品？

Hi RK, primarily, it's patients aging out. So discontinuations is almost misleading. All the kids are going to be on it until they stop growing. So typically around age 18, they will age out, they no longer need it. So it is expected and normal, and you're always going to have it.

嗨 RK，主要是因為患者年齡增加。所以說停產幾乎具有誤導性。所有孩子都會一直服用，直到他們停止生長為止。所以通常到了 18 歲左右，他們就超齡了，不再需要它了。所以這是意料之中的正常現象，你一直都會有這種情況。

That's the vast majority of the discontinuations. We see very little of what you think about as traditional discontinuations where somebody stops taking treatment before they reach their full adult or their full height, primarily because there's no other alternatives. It's not something like ALKINDI where they try to go to something else.

這佔了停產總數的絕大多數。我們很少看到你認為的傳統停藥情況，即某人在完全成年或達到完全身高之前停止接受治療，主要是因為沒有其他替代方案。它不像 ALKINDI 那樣，試圖去往其他方向。

So it was primarily age-outs. I think we are starting to promote and educate the market better. We think we are getting patients that are being diagnosed earlier. So their total duration on therapy is going to be longer. They're going to age out around 18 regardless of when they start.

所以主要是因為球員年齡超標而被淘汰。我認為我們正在更好地推廣和教育市場。我們認為現在收治的患者中，確診的患者比例有所提高。因此，他們的治療總時長將會更長。無論何時開始，他們大約都會在 18 歲左右達到年齡上限。

But if we can get them diagnosed and starting much earlier, that's going to lead to much better outcomes for the patients, and they're going to be on treatment much longer. So we think our average age is shifting much lower than it was when we inherited the business at the start of the year.

但是，如果我們能夠更早診斷出他們的病情並開始治療，這將為患者帶來更好的治療效果，而且他們接受治療的時間也會更長。因此，我們認為我們的平均年齡比年初我們接手公司時要低得多。

And James, you gave us -- you guided for a 70% gross margin into the fourth quarter. But in general, if I start thinking about beyond '25, '26 to '28 or '29, as you start seeing the new formulation of KHINDIVI come on board and whatnot, how -- what will be the cadence of the gross margin over that time period?

詹姆斯，你為我們帶來了——你預測第四季毛利率將達到 70%。但總的來說，如果我開始考慮 2025 年、2026 年到 2028 年或 2029 年以後的情況，隨著 KHINDIVI 的新配方推出等等，那麼——在那段時間裡，毛利率的節奏會是怎樣的呢？

RK, we have stated before, we think we can get to north of 75% by 2028. And how we get there is as the majority of our product revenue growth is concentrated in the products where we own more of the economics in KHINDIVI and ALKINDI and INCRELEX. That product mix shifts more towards those higher-margin products, which will continue to increase our margin profile over the next several years.

RK，我們之前已經說過，我們認為到 2028 年我們可以達到 75% 以上。我們實現這一目標的方式是，我們大部分的產品收入成長都集中在我們擁有更多經濟利益的產品上，例如 KHINDIVI、ALKINDI 和 INCRELEX。產品組合將更多地轉向利潤率更高的產品，這將在未來幾年內繼續提高我們的利潤率。

And then last question, Sean, in general, what's the pricing power that you have with your products? And do you -- are you seeing any pressures at all either from the government or from some of your private payers?

最後一個問題，肖恩，總的來說，你對你的產品有多大的定價權？您是否感受到來自政府或某些私人支付方的任何壓力？

No, I'd say we're always trying to be on the lower end in terms of the pricing for the -- compared to the number of patients. So we're a company that prides itself on pricing products appropriately. We don't believe that all the pricing discussions will fall down into the orphan drug products. We're talking about many of these diseases have only a few hundred patients. And so for them to start putting pressure on those products (inaudible).

不，我想說，就患者數量而言，我們始終努力將價格控制在較低水平。所以，我們是一家以合理定價為產品感到自豪的公司。我們認為，所有關於定價的討論最終都不會都落在孤兒藥產品上。我們談論的這些疾病中，許多只有幾百名患者。因此，他們開始對這些產品施加壓力。（聽不清楚）

I'm showing no further questions at this time. I'd like to thank you for your participation in today's conference. This does conclude the program. You may now disconnect.

我目前沒有其他問題要問。感謝各位參加今天的會議。節目到此結束。您現在可以斷開連線了。