Eton Pharmaceuticals Inc (ETON) 2025 Q1 法說會逐字稿

Good afternoon, and welcome to the Eton Pharmaceuticals First Quarter 2025 Financial Results Conference Call. (Operator Instructions) Please be advised that this call is being recorded at the company's request.

下午好，歡迎參加 Eton Pharmaceuticals 2025 年第一季財務業績電話會議。（操作員指示）請注意，此通話是應公司要求錄音的。

At this time, I'd like to turn it over to David Krempa, Chief Business Officer at Eton Pharmaceuticals. Please proceed.

現在，我想把發言權交給 Eton Pharmaceuticals 首席商務官 David Krempa。請繼續。

Thank you, operator. Good afternoon, everyone, and welcome to Eton's First 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, etonpharma.com. Joining me on our call today, we have Sean Brynjelsen, our CEO; James Gruber, our CFO; and Ipek Erdogan-Trinkaus, our Chief Commercial Officer. In addition to taking live questions on today's call, we will be answering questions that are e-mailed to us.

謝謝您，接線生。大家下午好，歡迎參加伊頓 2025 年第一季電話會議。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話題。新聞稿可在我們的網站 etonpharma.com 上查閱。今天參加電話會議的嘉賓包括：執行長 Sean Brynjelsen、財務長 James Gruber 以及首席商務長 Ipek Erdogan-Trinkaus。除了在今天的電話會議上現場回答問題外，我們還將回答透過電子郵件發送給我們的問題。

Investors can send their questions to investorrelations@etonpharma.com. Before we begin, I would like to remind everyone that remarks made during this 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.

投資者如有任何疑問，請發送電子郵件至 investorrelations@etonpharma.com。在開始之前，我想提醒各位，本次電話會議中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的結果有重大差異。請參閱我們收益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.

現在我將電話轉給我們的執行長 Sean Brynjelsen。

Thank you, David. Good afternoon, everyone, and thank you for joining us today. If you were aware or with us for our Investor Day in March, you heard about Eton's transformational 2024, and I'm proud to say the momentum has continued into 2025. Our existing products are generating strong growth. We recently added two high-value commercial assets to our portfolio with the acquisition and relaunch of Increlex and Galzin, and we've advanced our pipeline candidates, setting the stage for two potential approvals in the next nine months.

謝謝你，大衛。大家下午好，感謝大家今天的參與。如果您了解或參加了我們三月份的投資者日活動，您就會聽說伊頓公學的轉型 2024 年計劃，我很自豪地說，這一勢頭將持續到 2025 年。我們現有的產品正在實現強勁成長。我們最近透過收購和重新推出 Increlex 和 Galzin，為我們的投資組合增加了兩項高價值商業資產，並且我們推進了我們的候選藥物研發管道，為未來九個月內獲得兩項潛在批准奠定了基礎。

We've seen now sequential growth in product revenue for 17 straight quarters since the launch of Alkindi Sprinkle. Though we are very proud of this track record of commercial execution, we are just getting started. With attractive growth prospects for our existing products and a strong late-stage pipeline, we expect this streak to continue well into the future, and we're very excited about what lies ahead. I'd like to begin with one of our important new products, Increlex.

自從推出 Alkindi Sprinkle 以來，我們已經連續 17 個季度看到產品收入持續成長。儘管我們對這項商業執行記錄感到非常自豪，但我們才剛剛起步。由於我們現有產品具有誘人的成長前景和強大的後期產品線，我們預計這種勢頭將持續到未來，我們對未來感到非常興奮。首先我想介紹一下我們的一個重要的新產品，Increlex。

Increlex is a complex biologic product used in treating patients two years of age and older who suffer from severe primary insulin-like growth factor 1deficiency. SPIGFD affects an estimated 200 children in the United States. When we signed the transaction in the fourth quarter, we were very excited about the deal given the strong strategic fit within pediatric endocrinology and what we saw is a very attractive growth opportunity. We are now nearly five months into the transaction, and I am pleased to say that it is exceeding our expectations.

Increlex 是一種複雜的生物製劑，用於治療兩歲及以上患有嚴重原發性胰島素樣生長因子 1 缺乏症的患者。據估計，SPIGFD 影響了美國約 200 名兒童。當我們在第四季度簽署交易時，我們對這筆交易感到非常興奮，因為它與兒科內分泌學有著很強的策略契合度，而且我們看到了一個非常有吸引力的成長機會。現在，交易已經進行了近五個月，我很高興地說，交易超出了我們的預期。

Eton saw a tremendous opportunity to leverage our existing pediatric endocrinology sales force and commercial infrastructure as well as make new investments into community initiatives to raise awareness of this ultra-rare condition, which unfortunately had seen an increased number of children going undiagnosed in recent years. At its peak, more than a year or a decade ago, Increlex had 185 patients in the United States, but that number has been declining for years and was at only 67 patients when we completed our acquisition in late December.

伊頓看到了一個巨大的機會，可以利用我們現有的兒科內分泌銷售隊伍和商業基礎設施，並對社區計劃進行新的投資，以提高人們對這種極為罕見的疾病的認識，不幸的是，近年來未確診的兒童數量有所增加。在一年或十多年前的巔峰時期，Increlex 在美國擁有 185 名患者，但這一數字多年來一直在下降，當我們在 12 月底完成收購時，患者人數僅為 67 名。

With the significant investments we have made plus the hard work of our commercial team over the last five months, I am pleased to say that the trend appears to have reversed. We've now reached over 90 active patients and remain confident that we can reach our goal of 100 patients by the end of this year and even higher levels in the years to come. Missed diagnoses have been a long-standing problem with this condition. When physicians are presented with short of stature patients and IGF-1 deficiency is not necessarily top of mind.

透過我們所做的大量投資以及我們商業團隊在過去五個月的辛勤工作，我很高興地說，這種趨勢似乎已經扭轉。現在，我們已經擁有超過 90 名活躍患者，並且有信心在今年年底前達到 100 名患者的目標，並在未來幾年達到更高的水平。漏診一直是這種疾病的長期問題。當醫師接診身材矮小的患者時，IGF-1 缺乏症並不一定是首要考慮的問題。

And there's an ingrained habit of automatically prescribing growth hormones, SPIGFD patients generally have normal growth hormone levels, so this treatment is ineffective and delays a proper diagnosis, causing SPIGFD patients to miss the Increlex treatment window. We feel that it's important for all shorter stature patients to be screened for severe primary IGF-1 deficiency and are using our deep relationships in the pediatric endocrinology community to drive greater awareness of SPIGFD and push for the screening.

而且，由於人們有根深蒂固的自動開生長激素處方的習慣，SPIGFD 患者的生長激素水平通常正常，因此這種治療無效，並且會延遲正確的診斷，導致 SPIGFD 患者錯過 Increlex 治療窗口。我們認為，對所有身材較矮的患者進行嚴重原發性 IGF-1 缺乏症篩檢非常重要，並利用我們在兒科內分泌學界的深厚關係來提高人們對 SPIGFD 的認識並推動篩檢。

Eton is also working to expand access for US patients by seeking to harmonize the US and EU labels. The definition of what constitutes severe primary IGF-1 deficiency differs between the regions. While the height criteria is consistent, the IGF level is not. In the United States, a patient's IGF level must be at least three standard deviations below the median. While in the EU, patients must be in the bottom 2.5 percentile for their age and gender, which translates to approximately two standard deviations.

伊頓公學也致力於協調美國和歐盟的標籤，以擴大美國患者的治療機會。不同地區對嚴重原發性 IGF-1 缺乏症的定義有所不同。雖然身高標準一致，但 IGF 水準卻不一致。在美國，患者的 IGF 水平必須比中位數低至少三個標準差。而在歐盟，患者的年齡和性別必須處於最低的 2.5 個百分點，這相當於大約兩個標準差。

We have completed statistical analysis of the patient registry that has tracked hundreds of Increlex patients over the last decade, and we believe it shows that the product is safe and effective in the slightly broader EU label population. We expect to submit a supplemental filing to the FDA in the second half of 2025. By harmonizing the two definitions, we estimate that up to 1,000 US patients could clinically benefit, significantly expanding treatment opportunities beyond the current 200 patients.

我們已經完成了對過去十年來追蹤數百名 Increlex 患者的患者登記冊的統計分析，我們相信這表明該產品在略微廣泛的歐盟標籤人群中是安全有效的。我們預計在 2025 年下半年向 FDA 提交補充文件。透過協調這兩個定義，我們估計多達 1,000 名美國患者可以在臨床上受益，從而大大擴大目前 200 名患者以外的治療機會。

We are proud of the team's hard work to close, integrate and relaunch Increlex that the growth achieved in such a short period of time is impressive, and it is clear that the long-term growth opportunity remains with or without the label harmonization. During the quarter, we announced the out-licensing of Increlex' international rights to Esteve Pharmaceuticals for an upfront payment of $4.3 million.

我們為團隊為關閉、整合和重新推出 Increlex 所付出的辛勤工作感到自豪，在如此短的時間內取得的增長令人印象深刻，而且很明顯，無論是否進行標籤協調，長期增長機會仍然存在。在本季度，我們宣布將 Increlex 的國際權利授權給 Esteve Pharmaceuticals，預付款為 430 萬美元。

Increlex's international opportunity is relatively small, highly fragmented across more than 30 different countries and has slim margins. In addition, the out-licensing eliminated the need (inaudible) million G&A investment to support global infrastructure, maintain foreign regulatory approvals and facilitate commercial and distribution activities. We believe the transaction will be far more additive to Eton's profitability than if we had commercialized the product internationally ourselves.

Increlex 的國際機會相對較小，高度分散在 30 多個不同的國家，利潤微薄。此外，對外授權消除了支持全球基礎設施、維持外國監管批准和促進商業和分銷活動的數百萬 G&A 投資的需要。我們相信，與我們自己在國際上商業化該產品相比，此次交易將為 Eton 的盈利能力帶來更大的提升。

The transaction also provided us with a $4.3 million upfront payment, recouping a meaningful portion of our initial purchase price and providing excess capital that can be reinvested into our attractive pipeline opportunities. And finally, it eliminated the distraction and resource burden that would have come with managing more than 30 territories and several other team members to fully dedicate their time and attention to our three high-value US product launches in 2025. Esteve shares a similar philosophy of putting patients first.

這筆交易還為我們提供了 430 萬美元的預付款，收回了我們初始購買價格的很大一部分，並提供了可以再投資於我們具有吸引力的管道機會的剩餘資本。最後，它消除了管理 30 多個地區和其他幾位團隊成員所帶來的干擾和資源負擔，使他們能夠全身心地投入到 2025 年我們在美國推出的三款高價值產品中。埃斯特夫也抱持類似的理念，那就是把病人放在第一位。

So we are confident that they will be a good partner for the product and ensure that the international patients have access to this meaningful therapy. Turning now to Alkindi. This has been a strong growth driver for us for 17 straight quarters with no signs of slowing down. In fact, the pace of referrals has actually increased in 2025. As you may remember, beginning in January, we made our existing sales force 100% focused on pediatric endocrinology. We believe this has driven increased efficiency, which is apparent not only in the strong launch of Increlex, but also in an increased rate of new Alkindi Sprinkle prescriptions.

因此，我們相信他們會成為該產品的良好合作夥伴，並確保國際患者能夠獲得這種有意義的治療方法。現在轉向 Alkindi。這已連續 17 個季度成為我們強勁的成長動力，且沒有放緩的跡象。事實上，到 2025 年，推薦的速度實際上已經加快。您可能還記得，從一月開始，我們讓現有的銷售人員 100% 專注於兒科內分泌學。我們相信這推動了效率的提高，這不僅體現在 Increlex 的強勢推出上，也體現在新 Alkindi Sprinkle 處方率的提高上。

Through April, the number of new patient referrals received this year exceeds the first four months of any other year since launch. Despite the relatively high discontinuation rate, we have still added a significant number of new patients this year and are quickly closing in on our 500 active patients. Eton has been encouraged by Alkindi's strong start to the year and are expecting the rate of new patient adds to our adrenal insufficiency franchise to accelerate even further in the second half of 2025 with the anticipated launch of ET-400.

截至四月份，今年收到的新患者轉診數量超過了自推出以來任何一年的前四個月。儘管停藥率相對較高，但今年我們仍然增加了大量新患者，且活躍患者數量正在迅速接近 500 名。伊頓對 Alkindi 今年的強勁開局感到鼓舞，並預計隨著 ET-400 的預期推出，2025 年下半年我們腎上腺功能不全特許經營的新患者數量將進一步增加。

As we have discussed at length, we continue to see a large portion of the market using unapproved compounded hydrocortisone due to the preference for a liquid dosage form. We believe approximately 50% of young children are using a non-FDA liquid hydrocortisone today. With an estimated 5,000 adrenal insufficiency patients under nine years of age, we continue to see a very compelling market opportunity for ET-400.

正如我們詳細討論過的，由於人們偏愛液體劑型，我們繼續看到很大一部分市場使用未經批准的複合氫化可的松。我們相信，目前大約有 50% 的兒童正在使用非 FDA 批准的液體氫化可的松。據估計，九歲以下的腎上腺功能不全患者有 5,000 名，我們繼續看到 ET-400 極具吸引力的市場機會。

ET-400's PDUFA date is just two weeks away, and we are prepared to launch quickly upon potential approval. We've manufactured our launch inventory in our specialty sales force and promotional campaigns are ready to go live. We have been engaged in communications with the agency throughout the review and we're optimistic that they will meet their PDUFA goal date of May 28. It's a very exciting time at Eton. After many years of hard work, our team is very excited to be on the cusp of making this important medication available to the patients in need.

ET-400 的 PDUFA 日期僅剩兩週，我們準備在獲得批准後迅速啟動。我們已經在專業銷售團隊中製造了發布庫存，促銷活動也準備好開始。在整個審查過程中，我們一直與該機構保持溝通，我們樂觀地認為他們將在 5 月 28 日實現 PDUFA 目標日期。這是伊頓公學非常令人興奮的時刻。經過多年的努力，我們的團隊非常高興能夠為有需要的患者提供這種重要的藥物。

Transitioning now to another significant opportunity. The treatment of Wilson's disease or Wilson disease, more correctly, a rare genetic disorder that causes excessive copper accumulation in the body. Patients suffering from this condition do not metabolize copper normally with their bodies absorbing the copper instead, prevents it from leak in the body and Galzin is an FDA-approved treatment for patients with Wilson disease who have been initially treated with a chelating agent. It is the only FDA-approved zinc therapy for Wilson disease today.

現在正轉向另一個重要機會。威爾遜氏症或威爾遜氏症的治療，更準確地說，是一種導致體內銅過量累積的罕見遺傳性疾病。患有這種疾病的患者無法正常代謝銅，而是透過吸收銅來防止其在體內洩漏，而 Galzin 是 FDA 批准的一種治療威爾遜病的藥物，適用於最初使用螯合劑治療的患者。它是目前唯一獲得 FDA 批准的威爾遜氏症鋅療法。

As with Increlex, we (inaudible) Galzin as an acquisition because we saw significant opportunities for Eton to add value, grow the product and improve the outcomes for patients. And similar to severe primary IGF-1 deficiency, Wilson disease is a severely underdiagnosed condition with a lack of product investment leading to inadequate awareness and education. Wilson disease is estimated to impact approximately 10,000 people in the United States, but we estimate that only 2,000 of those patients are diagnosed and actively on a therapy today.

與 Increlex 一樣，我們（聽不清楚）收購 Galzin 是因為我們看到 Eton 有巨大的機會增加價值、發展產品並改善患者的治療效果。與嚴重的原發性 IGF-1 缺乏症類似，威爾遜氏症是一種嚴重漏診的疾病，由於缺乏產品投資，導致人們的認識和教育不足。據估計，威爾遜氏症在美國影響約 10,000 人，但我們估計其中只有 2,000 名患者目前被診斷並積極接受治療。

Unfortunately, most patients are not diagnosed until they are in their 20s or 30s when symptoms begin to present after years of excessive copper buildup. This delayed diagnosis leads to worse outcomes, including neurological damage and liver failure. The increased frequency of genetic testing in recent years has led to earlier diagnosis, allowing patients to proactively start zinc therapy before liver or other damage occurs, but an unmet need still remains.

不幸的是，大多數患者直到二十幾歲或三十幾歲時才被診斷出來，而這些症狀是在多年體內銅過度積累後才開始出現的。這種延遲診斷會導致更糟糕的後果，包括神經損傷和肝衰竭。近年來，基因檢測頻率的增加實現了早期診斷，使患者能夠在肝臟或其他損害發生之前主動開始鋅治療，但仍存在未滿足的需求。

Of the roughly 2,000 patients that have been diagnosed and are on treatment, we estimate approximately 800 use zinc therapy, while the remainder are on chelating agents. However, due to historical challenges with access, affordability and awareness, most patients on zinc therapy appear to be using over-the-counter supplements rather than the FDA-approved prescription product. The nutritional supplements are a different form of zinc, which have been shown to be less effective than Galzin.

在已確診並接受治療的約 2,000 名患者中，我們估計約有 800 名使用鋅療法，其餘患者使用螯合劑。然而，由於歷史上在取得、負擔能力和認知方面的挑戰，大多數接受鋅療法的患者似乎都在使用非處方補充劑，而不是 FDA 批准的處方產品。營養補充品是不同形式的鋅，其效果已被證明不如 Galzin。

We acquired Galzin because we feel that we are the right company to address this dynamic. We relaunched the product in March with robust patient services, including a $0 co-pay. For the first time ever, every Wilson disease patient who wants Galzin can access it regardless of insurance status. We believe Eton Cares is one of the most generous high-touch patient assistant programs in the industry and one of the many things that sets us apart from other rare disease companies.

我們收購 Galzin 是因為我們認為我們是能夠解決這個問題的合適公司。我們於三月重新推出了該產品，並提供強大的患者服務，包括 0 美元的共同支付。有史以來第一次，每位想要接受 Galzin 治療的威爾遜病患者都可以獲得該藥物，無論其保險狀況如何。我們相信，Eton Cares 是業界最慷慨的高接觸病患援助計畫之一，也是我們與其他罕見疾病公司區分開來的眾多因素之一。

Our relaunch has received a warm reception from the community, including patient advocates and leading Wilson disease physician. We are currently migrating patients to Eton's commercial infrastructure and Eton Cares program. This migration kicked off in March and will be a multi-month process as the previous pharmacies work through inventories that remained in the channel. We expect the conversion to be largely complete by the end of the quarter -- or the third quarter, at which time Galzin should begin producing meaningful revenue.

我們的重新啟動受到了社區的熱烈歡迎，其中包括患者倡導者和威爾遜病的領先醫生。我們目前正在將患者遷移到伊頓的商業基礎設施和伊頓關懷計劃。此次遷移於三月啟動，將持續數月，因為先前的藥局將處理掉通路中剩餘的庫存。我們預計轉換工作將在本季末或第三季基本完成，屆時 Galzin 應該會開始產生可觀的收入。

I'm pleased to be able to solve the access and affordability issues that have impacted Galzin users for more than a decade. However, we believe that there is more that can be done to improve the lives and outcomes of Wilson disease patients. After access and affordability, the two most common complaints about zinc therapy are the burdensome, dosing requirements and unpleasant GI side effects.

我很高興能夠解決困擾 Galzin 用戶十多年的訪問和負擔能力問題。然而，我們相信，還可以做更多的事情來改善威爾遜病患者的生活和結果。除了可及性和可負擔性之外，對鋅療法最常見的兩個抱怨是繁重的劑量要求和令人不快的胃腸道副作用。

We've set out to tackle these challenges with the development of ET-700, which we disclosed for the first time in March. ET-700 is an extended-release version of Galzin, which we believe will eliminate the need for 3 times per day dosing as well as potentially reduce the GI side effects that are reported by some patients on zinc therapy today. We initiated ET-700 development last year prior to the acquisition of Galzin and have now filed a patent on our proprietary formulation.

我們已著手開發 ET-700 來應對這些挑戰，並在 3 月首次披露了這一消息。ET-700 是 Galzin 的緩釋版本，我們相信它將消除每天 3 次服藥的需要，並可能減少目前一些接受鋅療法的患者報告的胃腸道副作用。我們在去年收購 Galzin 之前啟動了 ET-700 的開發，現在已經為我們的專有配方申請了專利。

We're now advancing this program at full speed and working with the top Wilson disease thought leaders to prepare a clinical study protocol. We are preparing for the manufacturing registration batches later this year and have a meeting with the FDA in the second quarter to discuss our proposed clinical program. If everything goes as planned, we expect to file an NDA in 2027. We believe this product candidate has the potential to generate more than $100 million of peak revenue. Our metabolic portfolio, which includes Carglumic Acid, Betaine, Nitisinone and GoLike continues to provide steady revenue and cash flow.

我們現在正全速推進該計劃，並與威爾遜病領域的頂尖思想領袖合作制定臨床研究方案。我們正在為今年稍後的生產註冊批次做準備，並將在第二季與 FDA 舉行會議，討論我們提出的臨床計劃。如果一切按計劃進行，我們預計將於 2027 年提交保密協議 (NDA)。我們相信，該產品候選產品有潛力創造超過 1 億美元的高峰收入。我們的代謝產品組合包括卡谷氨酸、甜菜鹼、尼替西農和GoLike，持續提供穩定的收入和現金流。

The group of products produced a solid year-over-year growth in the first quarter, but will have reduced significance to Eton going forward due to the rapidly increasing revenue contributions from our high-margin pediatric endocrinology products. Turning now to our development pipeline. During the quarter, we were pleased to report that ET-600 passed its pivotal bioequivalency study, which allowed us to submit an NDA for the product in late April.

該組產品在第一季實現了穩健的同比增長，但由於我們高利潤的兒科內分泌產品的收入貢獻迅速增加，該產品組對 Eton 未來的重要性將會降低。現在轉向我們的開發流程。在本季度，我們很高興地報告 ET-600 通過了關鍵的生物等效性研究，這使我們能夠在 4 月底提交該產品的 NDA。

ET-600 is Eton's proprietary patented oral solution of desmopressin under development for the treatment of central diabetes insipidus. Leading pediatric endocrinologists have long expressed the need for this product because it allows for the small precise and titratable doses required to treat pediatric patients. ET-600 is the same pediatric endocrinology prescriber base as Alkindi Sprinkle, ET-400 and Increlex. This should provide an important head start once commercialization activities begin.

ET-600 是 Eton 公司專有的去氨加壓素口服溶液，正在開發用於治療中樞性尿崩症。領先的兒科內分泌學家長期以來一直表示需要這種產品，因為它可以提供治療兒科患者所需的精確且可滴定的小劑量。ET-600 與 Alkindi Sprinkle、ET-400 和 Increlex 是相同的兒科內分泌處方基礎。一旦商業化活動開始，這將提供一個重要的先機。

We expect our application to be assigned a 10-month review, which would allow for an approval and launch potentially as early as the first quarter of 2026. Pre-launch commercial activities are already underway, and we are excited about the prospects for this important product. Eton is also continuing to advance Amglidia, which we acquired late last year. Amglidia is designed for the treatment of the ultra-rare condition of neonatal diabetes mellitus, which impacts an estimated 300 children in the United States and is within our pediatric endocrinology call plan.

我們預計我們的申請將經過 10 個月的審核，最早可能在 2026 年第一季獲得批准並啟動。上市前的商業活動已經在進行中，我們對這款重要產品的前景感到興奮。伊頓公學也持續推動我們去年年底收購的 Amglidia。Amglidia 專為治療極為罕見的新生兒糖尿病而設計，這種疾病影響著美國約 300 名兒童，並且屬於我們兒科內分泌呼叫計劃的範圍。

Although the product has been approved in the EU since 2018, there are currently no FDA-approved oral treatments for the condition, and therefore, it is not possible for infants in the US to receive the correct dose in an FDA-approved manner. Today, caregivers must either obtain a suspension from a compounding pharmacy or crush adult tablets to create a suspension at home. Similar to ET-400 and ET-600, Amglidia gives an opportunity to bring a liquid formulation to the market to provide precise and accurate pediatric dosing.

儘管該產品自 2018 年起已在歐盟獲得批准，但目前尚無 FDA 批准的針對該病症的口服治療方法，因此美國嬰兒無法以 FDA 批准的方式接受正確的劑量。如今，護理人員必須從配藥藥房獲得混懸液，或將成人藥片粉碎，在家中製成混懸液。與 ET-400 和 ET-600 類似，Amglidia 有機會將液體配方推向市場，以提供精確、準確的兒科劑量。

Our acquisition terms allow us to have an FDA meeting to receive confirmation of the clinical pathway before any payment occurred. This meeting occurred in April, and we were pleased with how it went. The FDA was receptive to what we believe is a feasible clinical pathway to bring this critical treatment to patients in the US. In the first quarter, we also unveiled another new internal development program, ET-800.

我們的收購條款允許我們在付款之前召開 FDA 會議以獲得臨床途徑的確認。這次會議於四月舉行，我們對會議的進展感到滿意。FDA 接受了我們認為可行的臨床途徑，以便將這種關鍵的治療方法帶給美國患者。第一季度，我們也推出了另一個新的內部開發專案ET-800。

Our French development partner, CROSSJECT, will continue to work on advancing the Zeneo hydrocortisone autoinjector and separately Eton will manage the development of this injectable vial product, which we are calling ET-800. In addition to the large retail opportunity for hydrocortisone injection, which we have discussed extensively, there was an even greater use in the hospital setting. A total of more than 5 million vials per year and approximately $100 million in sales today.

我們的法國開發合作夥伴 CROSSJECT 將繼續致力於推進 Zeneo 氫化可的鬆自動注射器，而 Eton 將單獨負責開發這種注射瓶產品，我們稱之為 ET-800。除了我們已經廣泛討論過的氫化可的松注射劑的巨大零售機會之外，它在醫院環境中也有更大的用途。目前每年總產量超過 500 萬瓶，銷售額約 1 億美元。

Today, hospitals use a lyophilized for freeze dried powder vial, which must be manually reconstituted prior to administration. We have developed and filed a patent on a proprietary ready-to-use liquid formulation that we believe will save time, reduce risk of medical errors and is an important factor as hydrocortisone is often used in the emergency room and operating rooms. If development activities progress as planned, we expect to make registration batches in the coming months and submit an NDA in early 2027.

如今，醫院使用冷凍乾燥粉瓶，在使用前必須手動重新配製。我們已經開發併申請了一種專有的即用型液體製劑的專利，我們相信這將節省時間，降低醫療錯誤的風險，並且是一個重要因素，因為氫化可的松經常用於急診室和手術室。如果開發活動按計劃進展，我們預計將在未來幾個月內進行註冊批次，並在 2027 年初提交 NDA。

On the business development front, we expect acquisition and licensing transactions to remain a central part of our long-term story, and we continue to evaluate new opportunities. We've demonstrated that we can successfully execute value-creating transactions, and we expect to continue to do so. However, our strong position allows us the luxury to remain disciplined and focus solely on products that are aligned with our ultra-rare disease strategy and can meet our high threshold for financial returns and value creation opportunities.

在業務發展方面，我們預期收購和授權交易仍將是我們長期發展的核心部分，我們將繼續評估新的機會。我們已經證明我們可以成功執行創造價值的交易，並且我們希望繼續這樣做。然而，我們強大的地位使我們能夠保持紀律，並只專注於符合我們的超罕見疾病策略的產品，並能滿足我們對財務回報和價值創造機會的高門檻。

As you have heard today, it's a very busy time at Eton, we have made great strides in our mission to bring as many new rare disease treatments to patients as possible. With two major product launches, relaunches already underway this year and the largest launch in our history, potentially a matter of days away, there's never been a more exciting time for the organization. Following the expected launch of ET-400, we see a clear path to reaching $100 million of revenue in the near term and much higher levels as our pipeline products come to market.

正如您今天所聽到的，伊頓公學現在是非常忙碌的時期，我們在為患者提供盡可能多的新型罕見疾病治療方法的使命上取得了長足的進步。今年我們已經推出了兩款重要產品，重新發布了多款新品，而我們歷史上最大的新品發布可能也即將在幾天后舉行，這對於公司來說是一個前所未有的激動人心的時刻。隨著 ET-400 的預期推出，我們看到了在短期內實現 1 億美元收入的明確途徑，並且隨著我們的管道產品進入市場，收入水平將更高。

Through years of hard work, our team has built an extraordinary organization, and we continue to make progress every day toward our goal of becoming one of the leading ultra-rare disease companies in the world. With that, I'll turn it over to James, our Chief Financial Officer, to discuss the financials and to discuss the tariff questions. James?

經過多年的努力，我們的團隊建立了一個非凡的組織，我們每天都在不斷進步，朝著成為世界領先的極罕見疾病公司之一的目標邁進。接下來，我將把主題交給我們的財務長詹姆斯，討論財務狀況和關稅問題。詹姆斯？

Thank you, Sean. I'll start by addressing the tariff situation in the US since that seems to be at the top of everyone's list of questions right now. We believe Eton would see minimal impact from any of the tariff proposals discussed to date. The majority of our products are produced in the US, Eton does not hold any IP in any foreign countries, and we do not have any intercompany transfer pricing arrangements. Our primary exposure will be with Increlex and Alkindi, which are both manufactured in Europe.

謝謝你，肖恩。我將首先討論美國的關稅情況，因為這似乎是目前每個人最關心的問題。我們相信，迄今為止討論的任何關稅提案對伊頓公學的影響都將微乎其微。我們的大部分產品都在美國生產，Eton 在任何外國都不持有任何智慧財產權，我們也沒有任何公司間轉讓定價安排。我們主要接觸的是 Increlex 和 Alkindi，它們都是在歐洲生產的。

However, the anticipated cost of products purchased from Europe represents less than 5% of our forecasted revenue. So a 20% tariff on European purchases would impact total company gross margin by less than 100 basis points, and the potential impact would likely be even smaller in future years since our late-stage pipeline products, ET-400 and ET-600, will also be manufactured within the US. In short, we are not concerned about the impact of tariffs, but we will continue to monitor the situation closely.

然而，從歐洲購買的產品的預期成本不到我們預測收入的 5%。因此，對歐洲採購徵收 20% 的關稅將對公司總毛利率產生不到 100 個基點的影響，而且由於我們的後期管道產品 ET-400 和 ET-600 也將在美國境內生產，因此未來幾年的潛在影響可能更小。簡而言之，我們並不擔心關稅的影響，但我們將繼續密切關注局勢。

Turning to our financial results. Our first quarter revenue was $17.3 million compared to $8.0 million in the first quarter of 2024, an increase of 117%. Net sales during the quarter included $3.3 million of licensing revenue, of which $1.8 million was from the licensing of Increlex rights outside of the US. Although Eton is receiving $4.3 million upfront, accounting guidance results in recording $1.8 million immediately with the remaining $2.5 million recognized over the licensing term.

談到我們的財務結果。我們第一季的營收為 1,730 萬美元，而 2024 年第一季的營收為 800 萬美元，成長了 117%。本季淨銷售額包括 330 萬美元的許可收入，其中 180 萬美元來自美國境外的 Increlex 權利許可。儘管伊頓公學預先收到了 430 萬美元，但會計準則規定應立即記錄 180 萬美元，剩餘的 250 萬美元將在許可期限內確認。

We also recorded $1.5 million of licensing revenue from a regulatory milestone event associated with our previous divestiture of DS-200. There was no licensing revenue recognized in the prior year quarter. Product sales were $14.0 million for the first quarter of 2025 compared with $8.0 million in the first quarter of 2024, an increase of 76%. This growth was driven primarily by increased sales of Alkindi Sprinkle and the addition of Increlex, which was acquired in late December.

我們也從與先前剝離 DS-200 相關的監管里程碑事件中獲得了 150 萬美元的授權收入。去年同期沒有確認許可收入。2025 年第一季的產品銷售額為 1,400 萬美元，而 2024 年第一季的產品銷售額為 800 萬美元，成長了 76%。這一成長主要得益於 Alkindi Sprinkle 銷量的成長以及 12 月底收購的 Increlex。

We expect product sales to continue growing quarter-over-quarter throughout the rest of 2025 and beyond, and we continue to expect to exit 2025 at an approximately $80 million annual revenue run rate. Gross profit for the quarter was $9.9 million compared with $5.0 million in the prior year period, primarily due to increased product sales. Adjusted gross profit, which excludes the impact of acquired inventory step-up adjustments and intangible amortization, was $12.0 million or 69.5% of total revenue versus $5.2 million of adjusted gross profit or 65.6% of total revenue in the prior year period.

我們預計，在 2025 年剩餘時間及以後，產品銷售額將繼續逐季成長，並且我們預計到 2025 年，年收入運行率將達到約 8,000 萬美元。本季毛利為 990 萬美元，而去年同期為 500 萬美元，主要原因是產品銷售增加。調整後的毛利（不包括收購庫存遞增調整和無形資產攤銷的影響）為 1,200 萬美元，佔總收入的 69.5%，而去年同期的調整後毛利為 520 萬美元，佔總收入的 65.6%。

This increase was driven by continued growth of higher-margin Alkindi Sprinkle and the recognition of higher-margin licensing revenue in the first quarter of 2025. We expect to report full year 2025 adjusted gross margin of approximately 70% and long-term adjusted gross margin to exceed 75% by 2028. R&D expenses for the quarter were $1.2 million compared with $0.7 million in the prior year period, primarily due to increased expenses associated with our ET-700 and ET-800 project development activities.

這一增長得益於利潤率較高的 Alkindi Sprinkle 的持續增長以及 2025 年第一季確認的利潤率較高的許可收入。我們預計 2025 年全年調整後毛利率約為 70%，到 2028 年長期調整後毛利率將超過 75%。本季研發費用為 120 萬美元，而去年同期為 70 萬美元，主要原因是與我們的 ET-700 和 ET-800 專案開發活動相關的費用增加。

It's worth noting that in April, we paid a $2.2 million NDA application fee related to our ET-600 submission. That cost will be fully recorded as R&D expense in the second quarter, and we also expect to record a $500,000 expense for an Amglidia licensing payment in the second quarter of 2025. Besides these two one-time items, we expect R&D spending to remain largely in line with historical levels for the remainder of 2025. General and administrative expenses for the quarter were $9.2 million compared with $5.2 million in the prior year period.

值得注意的是，四月份，我們支付了與 ET-600 提交相關的 220 萬美元 NDA 申請費。該費用將在第二季全額記錄為研發費用，我們也預計將在 2025 年第二季記錄 50 萬美元的 Amglidia 授權費用。除了這兩項一次性項目外，我們預計 2025 年剩餘時間內的研發支出將基本與歷史水準保持一致。本季的一般及行政開支為 920 萬美元，而去年同期為 520 萬美元。

As mentioned in our fourth quarter call, increased SG&A expenses in 2025 were planned and necessary to build out the infrastructure needed to support the significant growth in our product portfolio and revenue base. These incremental investments include our new dedicated 5-person metabolic sales team, which launched on January 2, commercial investments made in the product relaunches of Increlex and Galzin, investment in our ET-400 launch readiness activities and additional corporate staff to support the growing portfolio in the areas of quality, regulatory and finance.

正如我們在第四季度電話會議上提到的那樣，2025 年銷售、一般及行政費用的增加是計劃好的，也是必要的，以建立支持我們產品組合和收入基礎大幅增長所需的基礎設施。這些增量投資包括我們於 1 月 2 日成立的新的 5 人代謝銷售團隊、對 Increlex 和 Galzin 產品重新發布進行的商業投資、對我們的 ET-400 發布準備活動的投資以及額外的公司員工以支持質量、監管和財務領域不斷增長的產品組合。

On an adjusted basis, which removes the impact of share-based compensation, transaction-related costs and other one-time expenses, G&A expense was $7.3 million compared to $4.4 million in the prior year period. In addition to the planned increases in our infrastructure, SG&A expenses during the quarter were also affected by relaunch and prelaunch commercial activities in the period. We are not planning to make further significant investments in SG&A this year and anticipate that adjusted G&A spending will remain flat or slightly decline for the remainder of 2025.

經調整後，即剔除股權激勵、交易相關成本及其他一次性費用的影響，一般及行政管理費用為 730 萬美元，去年同期為 440 萬美元。除了我們基礎設施的計畫成長之外，本季的銷售、一般及行政費用也受到期間重新啟動和預發布商業活動的影響。我們今年不打算在銷售、一般及行政費用 (SG&A) 方面進一步進行大規模投資，並預計在 2025 年剩餘時間內調整後的銷售、一般及行政費用 (SG&A) 支出將保持平穩或略有下降。

We believe that the investments we've made in G&A during the quarter will support revenue levels much higher than where we are today. And as a result, we expect to return to minimal G&A growth in 2026 and beyond. Adjusted EBITDA for the first quarter of 2025 was $3.7 million compared to $0.5 million in the first quarter of 2024. Total company net loss was $1.6 million for the quarter compared to a net loss of $0.8 million in the prior year period.

我們相信，本季我們在一般及行政管理所做的投資將支持我們實現比現在高得多的收入水準。因此，我們預計 2026 年及以後 G&A 成長將恢復到最低水準。2025 年第一季的調整後 EBITDA 為 370 萬美元，而 2024 年第一季為 50 萬美元。本季公司淨虧損總額為 160 萬美元，去年同期淨虧損為 80 萬美元。

Net loss per basic and diluted share was $0.06 during the quarter compared to net loss per basic and diluted share of $0.03 in the prior year period. On a non-GAAP basis, we reported net income of $2.4 million for the first quarter of 2025 compared to $0.2 million in the prior year period and diluted earnings per share of $0.07 for the first quarter of 2025 compared to $0.00 in the prior year period. Eton finished the first quarter with $17.4 million of cash-on-hand, and we generated $2.1 million of operating cash flow during the quarter.

本季每股基本淨虧損和稀釋淨虧損均為 0.06 美元，去年同期每股基本淨虧損和稀釋淨虧損均為 0.03 美元。根據非公認會計準則 (Non-GAAP)，我們報告 2025 年第一季淨收入為 240 萬美元，而去年同期為 20 萬美元；2025 年第一季每股攤薄收益為 0.07 美元，而去年同期為 0.00 美元。伊頓第一季的現金餘額為 1,740 萬美元，本季的營運現金流為 210 萬美元。

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

這就是我們對第一季業績的評論。接下來，我們將把時間交給操作員進行問答。

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

（操作員指示）我們的第一個問題來自 Craig-Hallum 的 Chase Knickerbocker。

Congrats on the results here. Just first on Increlex. Sean, have you had your meeting with FDA to discuss that label expansion at this point?

恭喜你所取得的成果。首先介紹一下 Increlex。肖恩，你現在是否已經與 FDA 開會討論標籤擴展問題了？

The meeting request has gone in. So we'll be looking forward to having that, I would say, in the coming weeks, possibly most likely July, in my opinion, and then it would follow ideally with the submission in the third quarter of the actual update. We believe the data we have is compelling. It's based on patient registry data out of Europe and should support that label change.

會議請求已提交。因此，我們期待在未來幾週內實現這一目標，我認為最有可能是 7 月份，然後理想情況下將在實際更新的第三季提交。我們相信我們掌握的數據是令人信服的。它基於歐洲的患者登記數據，應該支援該標籤更改。

Great. And maybe staying on the FDA front. I appreciate the commentary on ET-400. Any additional color on any recent interaction with FDA that you can give us? I mean, are things progressing as you would expect with the review at this latest stage as in things like manufacturing inspections and final label discussions, et cetera?

偉大的。或許還會繼續留在 FDA 方面。我很欣賞對 ET-400 的評論。您能向我們提供有關最近與 FDA 互動的更多資訊嗎？我的意思是，在最新階段的審查中，事情進展是否如您所預期的那樣，例如製造檢查和最終標籤討論等？

Yes. The -- typically, the last step in a review process of an NDA drug application would be the labeling discussions. And so the FDA provides commentary on your label and what I mean by that is the package insert. We've received that commentary. We have already had the submission sent back to the agency. And so we believe that should be the final step. I mean we could hear of something else, but it seems, to me, that we are on track for approval here in two weeks or less.

是的。通常，NDA 藥物申請審查流程的最後一步是標籤討論。因此，FDA 會在您的標籤上提供評論，我指的是包裝說明書。我們已經收到了該評論。我們已經將提交的資料發回給該機構。因此我們相信這應該是最後一步。我的意思是，我們可能會聽到其他消息，但在我看來，我們預計在兩週或更短的時間內獲得批准。

Great. And on the Amglidia front, it sounds like you liked what you heard there. Does that mean you expect to just need a fairly simple kind of PK study to support an NDA filing there? Or just kind of any additional color?

偉大的。關於 Amglidia 方面，聽起來您很喜歡在那裡聽到的內容。這是否意味著您預計只需要相當簡單的 PK 研究來支持在那裡提交 NDA？或只是某種額外的顏色？

Yes. The -- so we like the feedback from the agency because you can tell during the tone, they would like to see us develop and get this product on market. They provided a pathway. It didn't seem like there were -- it seemed fairly clear to us and what we need to do. We did have a few clarifying questions. So we are planning a follow-up meeting with the agency to ensure that we're doing what they want and what their expectations are. But I was very encouraged by that, by the comments that we received back and I do expect that would just be a fairly straightforward PK study, as you indicated.

是的。我們喜歡來自該機構的回饋，因為您可以從語氣中聽出，他們希望看到我們開發並將該產品推向市場。他們提供了一條道路。似乎沒有——我們需要做什麼似乎相當清楚。我們確實有幾個需要澄清的問題。因此，我們計劃與該機構舉行後續會議，以確保我們所做的事情符合他們的要求和期望。但我對此感到非常鼓舞，我們收到的評論也讓我感到非常鼓舞，我確實希望這將是一項相當簡單的 PK 研究，正如您所說。

Got it. Pretty impressive continued progress on Increlex, I think you said 90 patients. If I pursue through the commentary on Increlex, on Sprinkle, I mean, it doesn't take much to -- for that $80 million run rate in Q4 to start looking pretty derisked. Is there anything I'm missing there? Or maybe any comments that you would have on that Q4 run rate?

知道了。Increlex 的持續進展相當令人印象深刻，我想您說的是 90 名患者。如果我繼續關注 Increlex 和 Sprinkle 的評論，我的意思是，不需要花費太多精力——第四季度 8000 萬美元的運行率看起來風險就很低了。我是否遺漏了什麼？或者您對第四季度的運行率有什麼評論？

No. I think at the appropriate time, we'll -- we feel very comfortable with the number to first address your question there. The question is, will we be revising that number at some point? I think we're going to leave it where it is right now, and we'll see where the sales go in the second quarter, but we're feeling pretty good about our sales. They've been strong across the board, and we couldn't be happier with the Increlex launch and the quick addition of patients. And we think the coming weeks here will help us to maybe provide some update on the next conference call.

不。我認為在適當的時候，我們會—我們很樂意先回答您的問題。問題是，我們會在某個時候修改這個數字嗎？我認為我們將維持目前的狀況，看看第二季的銷售情況，但我們對目前的銷售情況感覺相當良好。他們在各方面都表現強勁，我們對 Increlex 的推出和患者的快速增加感到非常高興。我們認為接下來的幾週將有助於我們在下次電話會議上提供一些最新消息。

And our next question coming from the line of Madison El-Saadi from B. Riley.

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

Yes. So could you maybe provide an update on the recent weekly Increlex prescription trends? I mean it really looks like March was a tremendous month. Is that kind of the proxy barometer going forward? And then --

是的。那麼您能否提供一下最近每週 Increlex 處方趨勢的最新情況？我的意思是，三月看起來確實是一個非常棒的月份。這是否是未來發展的代理晴雨表？進而--

Yes. Sure. On that point, I would say that we have significantly increased the number of patients. Now we're -- as we increase, we expect that rate would go down. You're getting closer to that whatever that 185 potential patients, let's say, that are in the US. So you would -- we would expect that. But as it stands today, we're firing at all cylinders in terms of the launch on that product.

是的。當然。從這一點來說，我想說我們的患者數量已經顯著增加。現在我們 — — 隨著我們的成長，我們預計該比率將會下降。您離這個數字越來越近了，比如說，在美國有 185 名潛在患者。所以你會——我們期待這一點。但就目前情況而言，我們正全力以赴推出該產品。

We expect to hit our goals -- revenue goals or exceed them -- this year, I think we'll exceed those goals. I'd like to get a few more months under our belt, and then we'll provide an update. And I think we probably -- we did indicate that we're now in the 90s on the patient front. We'll give further updates on that rate and better answer your question in terms of the trajectory of it.

我們預計今年將實現我們的目標——收入目標或超越這些目標——我認為我們將超越這些目標。我希望我們能再堅持幾個月，然後提供最新消息。我認為我們可能——我們確實指出我們現在在患者方面已經達到 90 多歲了。我們將進一步更新該比率，並就其軌跡更好地回答您的問題。

Got it. Understood. And then on ET-400, if we assume approval is on time, what's the expected timing to the first commercial revenue that you would book? Do you think this could be an end of 2Q story or maybe early 3Q?

知道了。明白了。然後，對於 ET-400，如果我們假設批准是按時進行的，那麼您預計預訂的第一筆商業收入的時間是什麼時候？您認為這可能是第二季故事的結束，或是第三季初的故事嗎？

I'm thinking more of a Q3. We will be launching it essentially right around the approval date. So we would expect a June, let's say, first week of June launch on the product. It takes a little bit of time for the patients to get the prescription because it's -- patients have to come back to the office and the doctors have to prescribe it. So I would say there's a bit of a lag between the initial launch and then the actual revenue coming in.

我更多考慮的是 Q3。我們將在批准日期前後推出它。因此，我們預計該產品將於六月，也就是六月第一周推出。患者需要花一點時間才能拿到處方，因為患者必須回到辦公室，然後醫生才能開處方。所以我想說，從最初的發佈到實際收入之間存在一些滯後。

So from a revenue standpoint, I would really think more about that sort of Q3, Q4. It will, we believe, have a rapid uptake. We know that it's something that there's a lot of pent-up demand for an oral solution. So let's -- maybe I'll leave it there and it give you an idea. It won't have a massive impact in terms of revenue this year, but we think it will have a much more significant impact in the quarters after that.

因此，從收入的角度來看，我會更多地考慮第三季和第四季。我們相信它將會被迅速接受。我們知道，口服溶液有著很大的需求。那麼，也許我會把它留在那裡，給你一個想法。它不會對今年的收入產生巨大影響，但我們認為它將對之後的幾季產生更顯著的影響。

Got it. And then lastly, I really appreciate the color on the tariff risk exposure, which looks to be really a nonstory. Any commentary to theoretical exposure to the White House Executive Order, the most favored nation policy?

知道了。最後，我非常欣賞關稅風險敞口的顏色，這看起來真的不是什麼故事。對白宮行政命令、最惠國政策的理論揭露有何評論？

Yes. Thanks for the question. It's probably the same question on others' minds. When I first heard about that, I was -- really didn't know what to make of it. There weren't enough details that were released around that. But now that we got a chance to look and digest that, and I've also been able to do a little bit more background research on it, I do not believe it will have a meaningful impact to us. We only sell in the US. So we're not selling products overseas to foreign customers at lower prices than we sell in the US.

是的。謝謝你的提問。其他人心中可能也有同樣的疑問。當我第一次聽到這件事時，我真的不知道該如何理解。目前尚未公佈足夠的相關細節。但是現在我們有機會了解和消化這一點，而且我也能夠對其進行更多的背景研究，我認為它不會對我們產生有意義的影響。我們只在美國銷售。因此，我們向海外客戶銷售產品的價格不會低於在美國銷售的價格。

We believe that alone is pretty strong in the sense that we don't have dual pricing as big pharma does. So in my opinion, this is something that would potentially impact big pharma. But we're also less reliant on Medicare, Medicaid than most pharma companies. Really no Medicare business to speak of, it's only Medicaid. And on the Medicaid, we lose money on the Increlex as I think a couple of people are involved.

我們相信，光是這一點就相當強大了，因為我們不像大型製藥公司那樣實行雙重定價。所以在我看來，這可能會對大型製藥公司產生影響。但與大多數製藥公司相比，我們對醫療保險和醫療補助的依賴程度較低。實際上沒有什麼醫療保險業務可言，只有醫療補助。在醫療補助方面，我們在 Increlex 上虧損了，因為我認為有幾個人參與其中。

But we make good money on Medicaid through products like (inaudible). There are no plans for us to sell our products overseas. So that reciprocal or most favored nation pricing would not apply, we believe, for our company (inaudible) we are a rare disease company -- an ultra-rare disease company, so I think that it's a unique situation compared to more standard pharmaceuticals.

但我們透過醫療補助產品賺了不少錢，例如（聽不清楚）。我們沒有計劃將產品銷售到海外。因此，我們認為，對於我們公司（聽不清楚）而言，互惠或最惠國定價不適用，我們是一家罕見疾病公司 - 一家極罕見疾病公司，因此我認為與更標準的製藥公司相比，這是一種獨特的情況。

And our next question coming from the line of Swayampakula Ramakanth from H.C. Wainwright.

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

This is RK from H.C. Wainwright. So I know you have had more experience with the relaunch of Increlex compared to Galzin. But, in general, what's the feedback from the sales force on Galzin? Anything anecdotally you can tell us about how the relaunch is going and what the expectations are internally for adoption during 2025?

這是來自 H.C. Wainwright 的 RK。所以我知道與 Galzin 相比，您在 Increlex 的重新推出方面擁有更多的經驗。但是，總體而言，銷售人員對 Galzin 的回饋如何？您能否告訴我們一些關於重新啟動的進展以及內部對 2025 年採用情況的期望？

Yes. So Galzin is an important product for the company. It's placing us squarely in the Wilson disease space, and we aspire to become a key player in Wilson disease. Beyond just Galzin, obviously, we have our ET-700 product, which is an extended-release version of Galzin. That product, we think we will have clinical trial results in January. That's a small study we're running, which will show the same efficacy, we believe, as the 3 times a day dosing with hopefully less side effects.

是的。因此，Galzin 對該公司來說是一款重要產品。它使我們完全處於威爾遜病領域，我們渴望成為威爾遜病領域的關鍵參與者。除了 Galzin 之外，顯然我們還有 ET-700 產品，它是 Galzin 的延長釋放版本。我們認為該產品將於一月獲得臨床試驗結果。這是我們正在進行的一項小型研究，我們相信它將顯示出與每天三次給藥相同的功效，並且希望副作用更少。

So that is really the big opportunity is the extended release version. The Galzin itself is now -- it's been great for patients because Galzin had in the past supply issues, they no longer have that. There's a $0 co-pay. They never had that in the past. They have that today. There's overnight shipping, so we get the product to them very quickly. So we believe that product will add appreciable revenue in the quarters to come. The uptake and the conversion has gone well, and we add patients weekly on Galzin.

因此，延長發布版本確實是一個很大的機會。Galzin 本身現在對患者來說非常好，因為 Galzin 過去有供應問題，但現在不再存在了。共同支付額為 0 美元。他們過去從來沒有過這樣的經驗。他們今天就有了它。由於我們提供隔夜送貨服務，因此我們能夠很快地將產品送到他們手中。因此我們相信該產品將在未來幾季增加可觀的收入。吸收和轉化進展順利，我們每週增加使用 Galzin 的患者。

So we see a steady stream of patients coming off of, let's say, over-the-counter stuff or compounded medicines. And they were forced to go there, frankly, in the past because of the inaccessibility of the medicine and the high co-pays that were charged by the prior company. We have taken away all those blockades.

因此，我們看到源源不絕的患者不再使用非處方藥或複方藥物。坦白說，過去他們被迫去那裡是因為藥品難以獲得，而且之前的公司收取了高額的共同支付費用。我們已經消除了所有封鎖。

So I think -- and then the last thing I want to say is we're adding a really strong relationship with the Wilson Disease Foundation and with really some of the leading prescribers in the United States that treat Wilson disease we have -- we've been able to get there -- get what their thoughts are, how we can help patients, how we can do the best job to make sure that these patients are served properly.

所以我認為——最後我想說的是，我們正在與威爾遜病基金會以及美國治療威爾遜病的一些主要處方醫師建立非常牢固的關係——我們已經能夠做到這一點——了解他們的想法，我們如何幫助患者，我們如何才能盡最大努力確保這些患者得到適當的服務。

Do you -- by any chance, are you able to give us patient numbers that are on Galzin at this point? Or we have to wait for the next update?

您能否告訴我們目前 Galzin 醫院的病患人數？還是我們必須等待下一次更新？

I think for the next update, we'll provide patient numbers because it's still in the transition period, but I can tell you that it is going better than -- according to plan, and I'm very pleased with the launch and the job that our Chief Commercial Officer, Ipek, has done with the organization. We have a dedicated sales team in that space as -- just like we have a dedicated sales team on our other endocrinology products.

我認為在下一次更新中，我們將提供患者人數，因為它仍處於過渡期，但我可以告訴你，它比計劃的進展要好，我對我們的首席商務官 Ipek 在該組織中所做的啟動和工作感到非常滿意。我們在該領域擁有專門的銷售團隊——就像我們在其他內分泌產品上擁有專門的銷售團隊一樣。

Perfect. A quick question for Jim. Looking through the cash flow statement, I see an increase in the Medicaid rebates and also on the receivables. So is this -- just trying to understand what's behind those numbers and how sticky are those?

完美的。向吉姆問一個簡單的問題。透過查看現金流量表，我發現醫療補助退款和應收帳款都有增加。那麼，這是否只是試圖了解這些數字背後的含義以及它們的黏性如何？

Sure. Let's start with the receivables. So significant increase there. Part of that was, call it, normal operating activity with the addition of Increlex. We talked about significant impact with Medicaid there. So with that product relaunch, a decent amount of addition -- sorry, with Increlex receivables. With the licensing revenue, even though we only recognized $1.8 million of the Esteve licensing deal revenue in the first quarter, there is a receivable payment of $4.3 million. So we have not received that yet.

當然。讓我們從應收帳款開始。因此有顯著的增長。其中一部分，可以稱之為，添加 Increlex 後的正常營運活動。我們討論了醫療補助計劃在那裡產生的重大影響。因此，隨著該產品的重新推出，我們增加了相當數量的——抱歉，是 Increlex 應收帳款。對於許可收入，儘管我們在第一季僅確認了 180 萬美元的 Esteve 許可交易收入，但應收款項為 430 萬美元。所以我們還沒收到。

So a big chunk of the [$5.8 million] increase in receivables was licensing revenue and then the remainder was just Increlex sales. On the Medicaid side, the increased liability was almost entirely due to Increlex, the product relaunch. So nothing out of the ordinary with the increased liability other than the fact that we have a traditional patient mix in Increlex and dollars are just higher with that product.

因此，應收帳款增加的 [580 萬美元] 中很大一部分是授權收入，其餘部分只是 Increlex 銷售額。在醫療補助方面，責任的增加幾乎完全是由於產品 Increlex 的重新推出。因此，除了我們在 Increlex 中擁有傳統的患者組合並且該產品的費用更高之外，增加責任並沒有什麼不尋常的。

And there are no further questions in the queue at this time. Ladies and gentlemen, this concludes today's conference call. Thank you all for your participation, and you may now disconnect.

目前隊列中沒有其他問題。女士們、先生們，今天的電話會議到此結束。感謝大家的參與，現在您可以斷開連線了。