Ocugen Inc (OCGN) 2025 Q1 法說會逐字稿

Good morning, and welcome to Ocugen's first quarter 2025 financial results and business update. Please note that this call is being recorded at this time. (Operator Instructions)

早上好，歡迎關注 Ocugen 2025 年第一季財務業績和業務更新。請注意，此通話目前正在錄音。 （操作員指示）

I will now turn the call over to Tiffany Hamilton, Ocugen's Head of Corporate Communications. You may begin.

現在我將電話轉給 Ocugen 企業傳播主管 Tiffany Hamilton。你可以開始了。

Thank you, operator, and good morning, everyone. Joining me on today's call and webcast is Dr. Shankar Musunuri, Ocugen's Chairman, CEO and Co-Founder, who will provide a business update and an overview of our clinical and operational progress.

謝謝接線員，大家早安。參加今天的電話會議和網路直播的還有 Ocugen 董事長、執行長兼聯合創始人 Shankar Musunuri 博士，他將介紹業務最新情況並概述我們的臨床和營運進展。

Ramesh Ramachandran, our Chief Accounting Officer, is also on the call to provide a financial update for the quarter ended March 31, 2025. Dr. Huma Qamar, our Chief Medical Officer, will be available to answer questions following the presentation.

我們的會計長 Ramesh Ramachandran 也將在電話會議上提供截至 2025 年 3 月 31 日的季度財務更新。我們的首席醫療官 Huma Qamar 博士將在演講結束後回答問題。

This morning, we issued a press release detailing associated business and operational highlights for the first quarter of 2025. We encourage listeners to review the press release, which is available on our website at ocugen.com. This call is being recorded, and a replay with the accompanying slide presentation will be available on the Investors section of our website for approximately 45 days.

今天上午，我們發布了一份新聞稿，詳細介紹了 2025 年第一季的相關業務和營運亮點。我們鼓勵聽眾閱讀該新聞稿，該新聞稿可在我們的網站 ocugen.com 上找到。本次通話將被錄音，其重播和隨附的幻燈片簡報將在我們網站的「投資者」部分提供約 45 天。

This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as predicts, believes, potential, proposed, continue, estimates, anticipates, expects, plans, intends, may, could, might, will, should or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements.

本簡報包含《1995 年私人證券訴訟改革法案》所定義的前瞻性陳述，這些陳述具有風險和不確定性。在某些情況下，我們可能會使用預測、相信、潛在、提議、繼續、估計、預期、期望、計劃、打算、可能、可以、或許、將會、應該等詞語或其他表達未來事件或結果不確定性的詞語來識別這些前瞻性陳述。

Such statements include, but are not limited to, statements regarding our clinical development activities and related anticipated timelines. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from our current expectations.

這些聲明包括但不限於有關我們的臨床開發活動和相關預期時間表的聲明。此類聲明受眾多重要因素、風險和不確定性的影響，可能導致實際事件或結果與我們目前的預期有重大差異。

These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission, the SEC, including the risk factors described in the section entitled Risk Factors in the quarterly and annual reports that we file with the SEC.

我們向美國證券交易委員會 (SEC) 提交的定期文件中對這些風險和其他風險和不確定性進行了更詳細的描述，包括我們向 SEC 提交的季度和年度報告中題為“風險因素”的部分中描述的風險因素。

Any forward-looking statements that we make in this presentation speak only to the date of the presentation. Except as required by law, we assume no obligation to update forward-looking statements contained in this presentation, whether as a result of new information, future events or otherwise after the date of this presentation. Finally, Ocugen's quarterly report on Form 10-Q covering the first quarter of 2025 was filed today.

我們在本簡報中所做的任何前瞻性陳述僅代表簡報的日期。除法律要求外，我們不承擔更新本簡報中的前瞻性聲明的義務，無論是由於新資訊、未來事件或本簡報日期之後的其他情況。最後，Ocugen 於今天提交了涵蓋 2025 年第一季的 10-Q 表格季度報告。

I will now turn the call to Dr. Musunuri.

現在我將把電話轉給 Musunuri 博士。

Thank you, Tiffany, and thank you all for joining us today. As detailed in our press release, we are excited to discuss the substantial progress of our game-changing modifier gene therapy platform. All three gene therapies are advancing through the clinic, and we're on track to meet our goal of three biologics license application/market authorization application filings in the next three years.

謝謝你，蒂芙尼，也謝謝大家今天加入我們。正如我們的新聞稿中所詳述的，我們很高興討論我們改變遊戲規則的修飾基因治療平台的實質進展。所有三種基因療法均在臨床中取得進展，我們預計在未來三年內實現提交三項生物製劑許可申請/市場授權申請的目標。

Modified gene therapies offer potential cures for life. Unlike traditional gene therapies or gene editing, modified gene therapies have the ability to regulate gene networks, reset homeostasis, restore the functional network and create a healthy environment for retinal cells to survive.

改良基因療法為生命帶來了潛在的治癒方法。與傳統的基因療法或基因編輯不同，改良的基因療法能夠調節基因網絡，重置體內平衡，恢復功能網絡並為視網膜細胞的生存創造健康的環境。

Since photoreceptors and retinal cells are nondividing, creating a healthy environment is a powerful concept and cells can potentially survive for life with this therapy. Today, based on the unique mechanism of action, these therapies target diseases with large patient populations globally, millions instead of a very small group of hundreds to a few thousand patients as with the traditional gene therapies in the market today.

由於感光細胞和視網膜細胞不會分裂，因此創造一個健康的環境是一個強有力的概念，而細胞有可能透過這種療法存活一生。如今，基於獨特的作用機制，這些療法可以針對全球數百萬患者的疾病，而不是像目前市場上的傳統基因療法那樣只針對幾百到幾千名患者的小群體。

Positive clinical data continues to affirm the modified gene therapy approach. We started off the year with a two-year safety and efficacy data from the Phase 1/2 OCU400 clinical trial that further supports the gene-agnostic mechanism of action of OCU400, a broad RP treatment not restricted to specific mutations with durability.

積極的臨床數據繼續肯定了改良的基因治療方法。今年年初，我們從 1/2 期 OCU400 臨床試驗中獲得了為期兩年的安全性和有效性數據，這進一步支持了 OCU400 的基因不可知作用機制，OCU400 是一種廣泛的 RP 治療方法，不限於具有持久性的特定突變。

It was especially gratifying to reach an alignment with the FDA for the OCU410ST Phase 2/3 pivotal confirmatory trial, which can be the basis of a BLA submission in 2027. Accelerating the clinical time line of OCU410ST will save significant costs and potentially address disease burden even sooner than anticipated.

尤其令人欣慰的是，我們與 FDA 就 OCU410ST 的 2/3 期關鍵確認性試驗達成一致，該試驗可作為 2027 年提交 BLA 的基礎。加快 OCU410ST 的臨床時間表將節省大量成本，並有可能比預期更快解決疾病負擔問題。

Finally, dosing was complete ahead of schedule in the Phase 2 portion of the OCU410 Phase 1/2 ArMaDa clinical trial for geographic atrophy, late-stage dry AMD. We are planning to initiate the Phase 3 clinical trial in 2026 on target with a BLA submission in 2028. I will now provide additional details around the program updates.

最終，OCU410 1/2 期 ArMaDa 臨床試驗第 2 階段針對地圖樣萎縮、晚期乾性 AMD 的給藥提前完成。我們計劃於 2026 年啟動第 3 期臨床試驗，並於 2028 年提交 BLA。現在我將提供有關計劃更新的更多詳細資訊。

OCU400 has the potential to treat approximate 300,000 RP patients in the US and EU and 1.6 million patients globally using a gene-agnostic approach delivered via single subretinal injection to target all 100 genes associated with RP.

OCU400 有可能透過單次視網膜下注射以基因不可知的方法治療美國和歐盟約 30 萬名 RP 患者以及全球 160 萬名患者，以針對與 RP 相關的所有 100 個基因。

A traditional gene therapy approach would require developing 100 different products to address all 100 genes, which is not commercially viable. More significantly, recent news affirms that traditional gene therapy approach is ineffective in achieving clinically meaningful outcomes for treating RP.

傳統的基因治療方法需要開發 100 種不同的產品來解決所有 100 個基因，這在商業上是不可行的。更重要的是，最近的新聞證實，傳統的基因治療方法對於治療 RP 無法獲得具有臨床意義的結果。

The positive two-year long-term safety and efficacy data from the Phase 1/2 clinical trial of OCU400 for RP demonstrates a durable and statistically significant with a p-value of 0.005 improvement in the visual function, LLVA in all evaluable treated subjects at two years when compared to untreated eyes. Additionally, 100%, 10 out of 10 of treated evaluable subjects demonstrated improvement or preservation in visual function compared to untreated eyes.

OCU400 治療 RP 的 1/2 期臨床試驗的積極兩年長期安全性和有效性數據表明，與未經治療的眼睛相比，所有可評估的治療受試者在兩年後視覺功能 (LLVA) 均得到了持久且具有統計學意義的改善，p 值為 0.005。此外，與未經治療的眼睛相比，100%（10 名接受治療的可評估受試者中有 10 名）的視覺功能均得到改善或維持。

The Phase 3 study spanning one year will enroll 150 participants divided into two study arms, 75 participants with the gene mutations and 75 participants who are gene agnostic. In each arm, participants will be randomized in a 2:1 ratio to receive either treatment or remain in an untreated control group, respectively.

為期一年的第三階段研究將招募 150 名參與者，分為兩個研究組，其中 75 名參與者攜帶基因突變，75 名參與者不確定基因突變。在每個組別中，參與者將以 2:1 的比例隨機分配接受治療或留在未治療的對照組。

The OCU400 Phase 3 liMeliGhT clinical trial is open to all eligible RP patients, early to advanced stage RP, including pediatric subjects aged five plus regardless of gene mutation, syndromic and non-syndromic forms of RP.

OCU400 第三階段 liMeliGhT 臨床試驗對所有符合條件的 RP 患者開放，包括早期至晚期 RP，包括五歲以上的兒科受試者，無論基因突變、綜合徵型和非綜合徵型 RP。

Two key parts of our potential BLA/MAA filings next year are on schedule. The first is manufacturing and we are tracking to complete process validations this year in support of registration. This material can also be used for commercial supply in 2027.

我們明年可能提交的 BLA/MAA 申請中的兩個關鍵部分正在按計劃進行。首先是製造，我們正在追蹤今年完成流程驗證以支援註冊。這種材料還可在2027年實現商業供應。

The second part, the Phase 3 clinical trial is progressing well. Just this week, EMA granted eligibility to submit our OCU400 MAA via centralized procedure as an ATMP based on the current study design and statistical analysis plan. This is a significant project milestone for OCU400.

第二部分，3期臨床試驗進展順利。就在本週，EMA 根據目前的研究設計和統計分析計劃，批准我們透過集中程序將 OCU400 MAA 作為 ATMP 提交。這對於 OCU400 專案來說是一個重要的里程碑。

EMA has appointed a project lead to support any queries related to the application and six months prior to MAA submission, a rapporteur and a core rapporteur will be appointed to support the application. This eligibility grant is a testament to EMA's recognition of the potential of OCU400 to address serious unmet medical need in Europe.

EMA 已任命一名專案負責人來支持與申請相關的任何疑問，並且在提交 MAA 前六個月，將任命一名報告員和一名核心報告員來支持該申請。此項資格資助證明了 EMA 對 OCU400 解決歐洲嚴重未滿足醫療需求的潛力的認可。

There is currently no approved treatment option for Stargardt disease that encompasses more than 1,200 mutations and affects 100,000 people in the US and EU and 1 million globally. The FDA's decision to approve a Phase 2/3 trial for registration underscores the potential of OCU410ST to meet this critical unmet medical need and has never been explored in clinical trials for Stargardt disease.

目前尚無針對斯塔加特氏症的核准治療方案，該疾病包含 1,200 多種突變，影響美國和歐盟的 10 萬人以及全球 100 萬人。 FDA 決定批准 2/3 期試驗進行註冊，凸顯了 OCU410ST 滿足這一關鍵未滿足醫療需求的潛力，並且從未在 Stargardt 病的臨床試驗中進行過探索。

Data from the OCU410ST Phase 1 GARDian clinical trial has shown significant improvements in both structural and functional outcomes. Additionally, OCU410ST has consistently demonstrated a very favorable safety and tolerability profile.

OCU410ST 第 1 階段 GARDian 臨床試驗的數據顯示，結構和功能結果均有顯著改善。此外，OCU410ST 一直表現出非常良好的安全性和耐受性。

In BCVA, treated eyes demonstrate a two line or 10-letter gain in the visual equity compared to untreated eyes and a statistically significant p-value of 0.02 improvement in visual function when compared to untreated fellow eyes.

在 BCVA 中，與未治療的眼睛相比，接受治療的眼睛的視覺公平性提高了兩行或 10 個字母，與未治療的眼睛相比，視覺功能的統計顯著改善 p 值為 0.02。

In the latest data, atrophic lesions grew slower by 54% at six months and by 103% at 12 months in evaluable treated eyes when compared to untreated eyes. The Phase 2/3 pivotal confirmatory clinical trial, which includes an adaptive design with an interim masked analysis at eight months will randomize 51 subjects, 34 of whom will receive a single subretinal injection of OCU410ST in the eye with the worst visual equity and 17 of whom will serve as untreated controls.

在最新數據中，與未經治療的眼睛相比，可評估的治療眼睛的萎縮性病變生長速度在六個月時降低了 54%，在 12 個月時降低了 103%。這項 2/3 期關鍵確認性臨床試驗採用自適應設計，並在八個月時進行中期盲法分析，將隨機分配 51 名受試者，其中 34 名將在視力最差的眼睛接受單次視網膜下注射 OCU410ST，17 名將作為未經治療的對照。

The primary endpoint in the clinical trial is change in atrophic lesion size. Secondary endpoints include visual equity as measured by test corrected visual equity, BCVA and LLVA compared to untreated controls. one-year data will be utilized for the BLA filing.

臨床試驗的主要終點是萎縮病變大小的變化。次要終點包括透過測試矯正視力、BCVA 和 LLVA 來衡量的視覺公平性（與未經治療的對照組相比）。一年的數據將用於 BLA 備案。

OCU410 specifically designed to address multiple pathways implicated in the pathogenesis of dry AMD offers a distinct advantage over current treatments that target only one cause of GA, require multiple injections per year and are accompanied by various safety concerns.

OCU410 專門設計用於解決與乾性 AMD 發病機制有關的多種途徑，與目前僅針對 GA 的一種病因、每年需要多次注射且伴有各種安全問題的治療方法相比，具有明顯的優勢。

Our goal with OCU410 is to provide a comprehensive solution, a potential onetime therapy for life for the 2 million to 3 million people in the US and Europe and 8 million globally suffering GA.

我們對 OCU410 的目標是提供全面的解決方案，為美國和歐洲的 200 萬至 300 萬人以及全球 800 萬患有 GA 的人們提供潛在的一次性終身治療。

In February, dosing was complete in the Phase 2 portion of the OCU410 Phase 1/2 ArMaDa clinical trial for GA. In evaluable subjects, OCU410 12-month data demonstrates a four line or 23-letter gain in visual equity.

2 月份，OCU410 1/2 期 ArMaDa GA 臨床試驗第 2 階段的給藥工作完成。在可評估的受試者中，OCU410 12 個月數據顯示視覺公平性增加了四行或 23 個字母。

There was 41% slower GA lesion growth in treated eyes versus untreated fellow eyes after a single injection. Furthermore, at 12 months, OCU410 treatment reserves more retinal tissue around the GA lesions of treated eyes compared to commercially available products given monthly or every other month.

單次注射後，接受治療的眼睛與未接受治療的眼睛相比，GA 病變生長速度減慢了 41%。此外，與每月或每隔一個月給藥的市售產品相比，OCU410 治療 12 個月後可在接受治療的眼睛的 GA 病變周圍保留更多的視網膜組織。

In the Phase 2 study, the safety and efficacy of OCU410 in patients with GA secondary to dry AMD will be assessed. 51 patients with randomized 1:1:1 into either of 2 treatment groups, medium or high dose are a control group. In the treatment group, subjects received a single subretinal 200 microliter administration of medium dose or high dose while the control remain untreated.

在第 2 階段研究中，將評估 OCU410 對繼發於乾性 AMD 的 GA 患者使用的安全性和有效性。 51名患者以1：1：1的比例隨機分配到2個治療組中，中劑量或高劑量為對照組。在治療組中，受試者接受單次視網膜下注射 200 微升中劑量或高劑量藥物，而對照組則不接受治療。

The Data and Safety Monitoring Board has evaluated safety in all 51 patients from Phase 2 clinical trial. To date, there have been no serious adverse events related to OCU410. Phase 2 interim results are expected in the fall of this year.

數據和安全監測委員會對第 2 階段臨床試驗中所有 51 名患者的安全性進行了評估。迄今為止，尚未發生與 OCU410 相關的嚴重不良事件。第二階段中期結果預計將於今年秋季公佈。

I would also like to provide a summary of programs outside of our first-in-class modified gene therapy platform. Earlier this year, the first patient was dosed in the Phase 1 clinical trial for OCU200, the company's biologic product candidate for diabetic macular edema.

我也想提供我們一流的改良基因治療平台以外的項目的摘要。今年早些時候，該公司用於治療糖尿病黃斑水腫的生物製劑候選產品 OCU200 的 1 期臨床試驗中迎來了第一位患者。

We are currently dosing the second cohort based on first cohort safety data. Ocugen plans to complete the Phase 1 clinical trial in the second half of 2025. The company intends to initiate the Phase 3 trial for NeoCart contingent on adequate availability of funding and are based on the potential of a future partnership.

我們目前正在根據第一批的安全數據對第二批患者進行劑量測定。 Ocugen 計畫在 2025 年下半年完成第 1 階段臨床試驗。該公司打算在資金充足和未來合作潛力的基礎上啟動 NeoCart 的第 3 階段試驗。

Lastly, the investigational new drug application is in effect for OCU500 and the National Institute of Allergy and Infectious Diseases intends to initiate a Phase 1 clinical trial in the second quarter of 2025. Ocugen is continuing discussions with the relevant government agencies as well as strategic partners regarding developmental funding for its vaccine technology for flu.

最後，OCU500 的新藥研究申請已經生效，美國國家過敏和傳染病研究所計劃於 2025 年第二季啟動 1 期臨床試驗。 Ocugen 正在繼續與相關政府機構和戰略合作夥伴就其流感疫苗技術的開發資金進行討論。

With that, I will now turn the call over to our Chief Accounting Officer, Ramesh Ramachandran to provide an update on our financial results for the first quarter ended March 31, 2025. Ramesh?

現在，我將把電話轉給我們的首席會計官 Ramesh Ramachandran，請他提供截至 2025 年 3 月 31 日的第一季財務業績更新。 Ramesh？

Thank you, Shankar. Our research and development expenses for the quarter ended March 31, 2025, were $9.5 million compared to $6.8 million for the first quarter of 2024. General and administrative expenses for the quarter ended March 31, 2025, were $6.5 million compared to $6.4 million during the same period in 2024.

謝謝你，Shankar。截至 2025 年 3 月 31 日的季度，我們的研發費用為 950 萬美元，而 2024 年第一季為 680 萬美元。截至 2025 年 3 月 31 日的季度，一般及行政費用為 650 萬美元，而 2024 年同期為 640 萬美元。

Net loss was approximately $15.3 million or $0.05 net loss per share for the quarter ended March 31, 2025, compared to a net loss of approximately $11.9 million or $0.05 net loss per share for the first quarter of 2024.

截至 2025 年 3 月 31 日的季度，淨虧損約為 1,530 萬美元或每股淨虧損 0.05 美元，而 2024 年第一季的淨虧損約為 1,190 萬美元或每股淨虧損 0.05 美元。

Our cash and restricted cash totaled $38.1 million as of March 31, 2025, compared to $58.8 million as of December 31, 2024. The company expects that its cash and restricted cash will provide the company cash runway into the first quarter of 2026. As always, we are constantly exploring strategic and shareholder-friendly opportunities to increase our working capital.

截至 2025 年 3 月 31 日，我們的現金和限制性現金總額為 3,810 萬美元，而截至 2024 年 12 月 31 日為 5,880 萬美元。該公司預計其現金和限制性現金將為公司到 2026 年第一季提供現金跑道。像往常一樣，我們不斷探索策略性和股東友善機會，以增加我們的營運資金。

That concludes my update for the quarter. Tiffany, back to you.

這就是我對本季的最新情況的總結。蒂芬妮，回到你身邊。

Thank you, Ramesh. We will now open the call for questions. Operator?

謝謝你，拉梅什。我們現在開始提問。操作員？

(Operator Instructions) Michael Okunewitch, Maxim Group.

（操作員指示）Michael Okunewitch，Maxim Group。

Hey, there. Thank you so much for taking my questions today. Congratulations on all the progress. So I think just first off, in terms of the liMeliGhT study, where are you in enrollment? And when do you have to complete that to reach your filing targets?

嘿。非常感謝您今天回答我的問題。祝賀你取得的所有進展。所以我想首先，就 liMeliGhT 研究而言，您的招生情況如何？您必須什麼時候完成才能達到您的申報目標？

Good morning. This is Huma Qamar, CMO, Ocugen. Thanks for your question. So for our liMeliGhT OCU400-301 Phase 3 trial, we are on track for enrollment as planned for first half of 2025, and we will be on track for our BLA submission a year from now in terms of clinical. Thank you.

早安.我是 Ocugen 行銷長 Huma Qamar。謝謝你的提問。因此，對於我們的 liMeliGhT OCU400-301 第 3 階段試驗，我們將按計劃在 2025 年上半年進行招募，並且我們將在一年後按計劃提交臨床 BLA。謝謝。

And just as we approach the completion of that first pivotal study, I'd like to see if you could just expand a little bit on manufacturing capabilities and then what you would have to build out and expand to support the filing and commercialization stage of this product.

就在我們即將完成第一項關鍵研究之際，我想看看您是否可以稍微擴展一下製造能力，然後看看您需要建立和擴展哪些功能來支援該產品的申請和商業化階段。

Yeah. Michael, I'll answer the first part, then our CSO, Dr. Arun Upadhyay is on the call, too. He can explain what our manufacturing plans are. So for BLA submission, we have to complete process validations at the commercial scale, which we are on target this year. And so, that's what is needed.

是的。邁克爾，我將回答第一部分，然後我們的 CSO Arun Upadhyay 博士也會參加電話會議。他可以解釋我們的生產計劃是什麼。因此，對於 BLA 提交，我們必須完成商業規模的流程驗證，這是我們今年的目標。所以，這就是我們需要的。

And obviously, we have plenty of capacity for commercial launch of this product, ex-US We also built our own facilities here in Malvern, Pennsylvania. Once we commercially launch the product in 2027, shortly after that, within two years, our goal is to get the second site in US and get it ready for launch or commercial supplies.

顯然，我們有足夠的能力在美國以外地區商業化推出該產品，我們也在賓州馬爾文建造了自己的設施。一旦我們在 2027 年將產品商業化推出，此後不久，也就是兩年內，我們的目標是在美國建立第二個站點，並為產品上市或商業供應做好準備。

Arun, you want to go into more details on this year's plans?

阿倫，你想更詳細地介紹一下今年的計畫嗎？

Yeah. Thanks, Shankar. So as you stated, we are on track to complete the process validation activity and get ready for BLA submission next year. And for initial commercial launch, we'll be using our partner, CanSino Bio for OCU400 project.

是的。謝謝，Shankar。正如您所說，我們正在按計劃完成流程驗證活動並為明年提交 BLA 做好準備。對於首次商業發布，我們將與我們的合作夥伴康希諾生物合作 OCU400 專案。

But our plan is like subsequently, we are going to bring back the manufacturing, we do the tech transfer and bring the manufacturing in-house in our Malvern GMP manufacturing facility, which we have completed construction last year. And subsequently, we plan to release the product from our second site in the US So that's our strategy to support the future cost lines.

但我們的計劃是，隨後我們將恢復製造業務，進行技術轉讓，並將製造業務轉移到我們去年已完工的 Malvern GMP 製造工廠內部。隨後，我們計劃從美國的第二個網站發布該產品，這是我們支援未來成本線的策略。

All right. Thank you. And then just one more for me, and I'll hop back into the queue. Could you give us an idea of when we could expect to see the next update on the OCU200 program? I know it's not core, but DME has been an exciting space.

好的。謝謝。然後我再說一遍，然後我會重新回到隊列中。您能否告訴我們何時可以看到 OCU200 計劃的下一次更新？我知道這不是核心，但 DME 一直是一個令人興奮的領域。

Yeah. I think the clinical trial, we are planning to complete later part of this year. So our goal is before the end of the year, we'll provide clinical update, including preliminary efficacy and safety.

是的。我認為我們計劃在今年稍後完成臨床試驗。因此，我們的目標是在今年年底之前提供臨床更新，包括初步療效和安全性。

Robert LeBoyer, Noble.

羅伯特·勒博耶，貴族。

Good morning, everybody. I had a clarification on some of the prepared remarks regarding Europe and the regulation there. My understanding was that these remarks referred to 410ST and the clinical trial going on. Am I correct on that?

大家早安。我對一些關於歐洲及其法規的準備好的評論進行了澄清。我的理解是，這些言論是指 410ST 和正在進行的臨床試驗。我這樣說對嗎？

No. Your question, Robert, related to 410ST, yes, I mean the morning, we actually talked during this -- we have a recent communication from EMA on OCU400, just to clarify. And so, for submission of the MAA. And that's the path, and it's going well. That's what we talked about.

不。羅伯特，你的問題與 410ST 有關，是的，我的意思是早上，我們實際上在這個過程中進行了交談 - 我們最近收到了 EMA 關於 OCU400 的溝通，只是為了澄清一下。因此，需要提交 MAA。這就是道路，而且進展順利。這就是我們所談論的。

As far as OCU410ST is concerned, the clinical trial we designed with FDA, which will be a registration trial, which we are going to embark on it shortly. And we are discussing that strategy with the EMA. We don't have the final answer yet.

就 OCU410ST 而言，我們與 FDA 共同設計的臨床試驗將是一項註冊試驗，我們很快就會開始進行。我們正在與 EMA 討論該策略。我們還沒有最終答案。

And obviously, this is a much needed product, significant unmet medical need, just as RP, Stargardt disease, there are no approved treatment in EU. We are hoping they will align with FDA clinical trial design and also agree. Again, we are in negotiations with the EMA. When we have an answer from them, we'll let the markets know.

顯然，這是一種急需的產品，具有重大的未滿足的醫療需求，就像 RP、Stargardt 病一樣，歐盟目前還沒有批准的治療方法。我們希望他們能夠符合 FDA 的臨床試驗設計並表示同意。再次，我們正在與 EMA 進行談判。當我們得到他們的答覆時，我們會告知市場。

Okay. Great. And just separately, you had mentioned the influenza trial with NIAID and considering what's been going on with HHS and the FDA and the NIH in the past two or three months and the overhaul going on at those agencies. Have you had any changes in the relationship or with contact with people or any insights you can give as to what the agency is thinking going forward?

好的。偉大的。另外，您提到了與 NIAID 合作進行的流感試驗，並考慮過去兩三個月 HHS、FDA 和 NIH 的進展以及這些機構正在進行的改革。您與人們的關係或聯繫是否有任何變化？或者您能否就該機構未來的想法提供任何見解？

I know that some of the companies that were developing COVID vaccines had grants revoked and canceled. So I was wondering if you could give us any insight as to what's going on in the divisions that you interact with.

我知道一些正在研發新冠疫苗的公司已被撤銷或取消了資助。所以我想知道您是否可以向我們介紹一下您所接觸的部門的情況。

Yeah. Robert, great question. This is related to our COVID vaccine targeting intranasal versus inhalation route. So this clinical trial, NIAID is still on target to support it. In fact, I will ask Dr. Upadhyay, our CSO, to comment more on this. He is in direct contact with the NIAID team. Go ahead.

是的。羅伯特，好問題。這與我們的 COVID 疫苗針對鼻內途徑而非吸入途徑有關。因此，對於這項臨床試驗，NIAID 仍將按計劃予以支持。事實上，我會​​請我們的 CSO Upadhyay 博士對此發表更多評論。他與 NIAID 團隊保持直接聯繫。前進。

Thank you, Shankar. Yeah. I think we don't see any impact on us related to the changes you just mentioned are happening at the government. And so, we are constantly interacting with NIAID, and we are collaboratively working with them to finalize the clinical study plan as well as supporting the required material to initiate the Phase 1 study.

謝謝你，Shankar。是的。我認為您剛才提到的政府變化不會對我們產生任​​何影響。因此，我們不斷與 NIAID 互動，並與他們合作完成臨床研究計劃並支持啟動第一階段研究所需的材料。

So we are on track. And as Shankar mentioned, we are hoping to initiate the Phase 1 study in second quarter this year, and we are on target for that. And our relationship has been great. I would say I think it has improved even actually. Our engagement with NIAID has increased significantly to initiate the study.

所以我們的計劃正在順利進行中。正如 Shankar 所提到的，我們希望在今年第二季啟動第一階段的研究，而我們已經實現了這一目標。我們的關係一直都很好。我想說，我認為它實際上已經有所改善。我們與 NIAID 的合作已大大增加，以啟動這項研究。

Swayampakula Ramakanth, H.C. Wainwright.

斯瓦亞姆帕庫拉·拉瑪坎特，H.C.溫賴特。

Thank you. Good morning, Shankar and team. A couple of quick questions on the liMeliGhT study. Does the Phase 3 study have an interim look? And if so, what's the general timing that we should expect that to be?

謝謝。早上好，Shankar 和團隊。關於 liMeliGhT 研究的幾個快速問題。第三階段研究是否有中期觀察？如果是的話，我們應該預期大概什麼時候會發生？

This is Huma. Thanks for the question. There is no interim look in the study. And once the enrollment is complete, and we'll be giving periodic updates, there are safety for 30 subjects each that we are following for the liMeliGhT study. However, the full data would be available once the CSR is final. As this is a blinded study, we cannot release the data before, yes.

這是 Huma。謝謝你的提問。研究中沒有中期觀察。一旦招募完成，我們將定期更新訊息，我們將追蹤 liMeliGhT 研究的 30 名受試者的安全性。然而，一旦 CSR 最終確定，完整的數據就會可用。由於這是一項盲法研究，因此我們不能提前發布數據。

Thank you for that, Huma. A quick clarification on the design because maybe I misheard it -- I thought the randomization for the liMeliGhT study was 2:1?

謝謝你，Huma。對設計進行快速澄清，因為也許我聽錯了——我以為 liMeliGhT 研究的隨機化是 2:1？

Yes.

是的。

Okay. because I thought I heard it as one is to one, based on Shankar's comments but probably I got it wrong. And then on the --

好的。因為根據 Shankar 的評論，我認為我聽到的是一對一的，但可能我錯了。然後——

So RK, just a clarification. The 1:1:1 is for the currently ongoing clinical trial for OCU410 targeting dry age-related macular degeneration. So they have medium dose, high dose and control in that Phase 2.

所以 RK，我只是想澄清一下。 1:1:1 是針對目前正在進行的 OCU410 針對乾性老年黃斑部病變的臨床試驗。因此，他們在第 2 階段採用中劑量、高劑量和控制。

Perfect. One last question from me is on the three modified gene therapy programs, what clinical data updates would we see in 2025? And just trying to see if we can get any idea of how strong the data is going to look in all of the pivotal programs as we move forward.

完美的。我的最後一個問題是，關於這三種改良基因治療方案，我們將在 2025 年看到哪些臨床數據更新？只是想看看我們是否可以了解，隨著我們不斷前進，數據在所有關鍵項目中的表現將會有多麼強勁。

I think from OCU400 perspective, as you must stated, it's a blinded Phase 3 trial. You will get periodic updates how the trial is progressing. Other than that, the data won't come out until next year until the trial is completely done. The second program, OCU410, ArMaDa trial for dry AMD, and we are expecting interim results sometime this fall.

我認為從 OCU400 的角度來看，正如您所說，這是一項盲法 3 期試驗。您將定期收到有關試驗進展的最新資訊。除此之外，試驗徹底結束後數據要到明年才會公佈。第二個項目是 OCU410，ArMaDa 針對乾性 AMD 的試驗，我們預計將在今年秋季的某個時候獲得中期結果。

And OCU410ST, obviously, the registration trial is going to start shortly in the next couple of months. We are going to provide when you have a complete 12-month data from Phase 1 on this study. And in addition, OCU200, the Phase 1 trial should be complete late this year, and we'll provide the data from the trial too.

顯然，OCU410ST 的註冊試驗將在未來幾個月內開始。當您擁有該研究第一階段的完整 12 個月數據時，我們將提供。此外，OCU200 的第一階段試驗預計將於今年稍後完成，我們也將提供試驗數據。

Daniil Gataulin, Chardan.

丹尼爾·加陶林，Chardan。

Good morning. This is Stephen on for Daniil. Thanks for taking my question. For OCU400 for RP to file in Europe, do any of the patients need to be treated in Europe? Or is the data from the US and Canada could be sufficient?

早安.這是丹尼爾的史蒂芬。感謝您回答我的問題。對於 RP 的 OCU400 在歐洲提交申請，是否有任何患者需要在歐洲接受治療？或者來自美國和加拿大的數據就夠了嗎？

So I'll take that question. That's a great question, actually. So as we have stated in the past as well, that no additional trial is required in Europe. If we get the approval in the US based on our primary endpoint and the study design, US and Canada is sufficient. In fact, US is sufficient for our approval in Europe. So no additional trials are required. This trial has sufficient representation for global mutations as it's the only broad RB indication gene-agnostic trial globally.

所以我來回答這個問題。事實上，這是一個很好的問題。正如我們過去所說的那樣，歐洲不需要進行額外的試驗。如果我們根據主要終點和研究設計在美國獲得批准，那麼美國和加拿大就足夠了。事實上，美國足以讓我們獲得歐洲的認可。因此不需要進行額外的試驗。該試驗對於全球突變具有足夠的代表性，因為它是全球唯一一項廣泛的 RB 適應症基因不可知試驗。

This concludes the Q&A portion. I will now turn the call back over to Chairman, CEO and Co-Founder, Dr. Shankar Musunuri. Please go ahead.

問答部分到此結束。現在我將把電話轉回給董事長、執行長兼聯合創始人 Shankar Musunuri 博士。請繼續。

Thank you, operator. Our efforts in the first quarter of the year evidenced the importance of our gene therapy programs and the significance of upcoming milestones.

謝謝您，接線生。我們在今年第一季的努力證明了我們的基因治療計劃的重要性以及即將到來的里程碑的意義。

We remain steadfast in our mission to provide a onetime therapy for life to address considerable unmet medical needs that exist for millions of patients facing the terrifying prospect of losing their vision and look forward to what this quarter and the rest of the year holds for the company, our people, patients and shareholders. Thank you.

我們始終堅定不移地履行我們的使命，提供一次性終身治療，以解決數百萬面臨失去視力可怕前景的患者所面臨的大量未滿足的醫療需求，並期待本季度和今年剩餘時間公司、我們的員工、患者和股東取得的成績。謝謝。

Thanks again, everyone. Goodbye.

再次感謝大家。再見。

This concludes today's conference call. Thank you all for joining, and you may now disconnect.

今天的電話會議到此結束。感謝大家的加入，現在可以斷開連線了。