Ocugen Inc (OCGN) 2023 Q2 法說會逐字稿

Good morning and welcome to Ocugen's second-quarter 2023 financial results and business update. Please note that this call is being recorded at this time. (Operator Instructions)

早上好，歡迎來到 Ocugen 2023 年第二季度財務業績和業務更新。請注意，此時此通話正在錄音。 （操作員說明）

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

我現在將把電話轉給 Ocugen 企業傳播主管蒂芙尼·漢密爾頓 (Tiffany Hamilton)。你可以開始了。

Thank you, operator. Joining me today are Ocugen's Chairman, CEO, and Co-Founder, Dr. Shankar Musunuri who will provide a business and financial update; and Dr. Arun Upadhyay, our Financial and Scientific Officer, Head of Research, Development and Medical, who is also on the call to answer questions during the Q&A.

謝謝你，接線員。今天與我一起出席的有 Ocugen 董事長、首席執行官兼聯合創始人 Shankar Musunuri 博士，他將提供業務和財務方面的最新信息；我們的財務和科學官員、研究、開發和醫療主管 Arun Upadhyay 博士也應邀在問答環節回答問題。

Yesterday afternoon, we issued a press release detailing business and operational highlights for the second quarter of 2023. 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 the Ocugen website for approximately 45 days.

昨天下午，我們發布了一份新聞稿，詳細介紹了 2023 年第二季度的業務和運營亮點。我們鼓勵聽眾查看該新聞稿，該新聞稿可在我們的網站 ocugen.com 上獲取。本次電話會議正在錄音，並且將在 Ocugen 網站的投資者部分提供大約 45 天的重播以及隨附的幻燈片演示。

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, estimate, anticipate, expect, plans, intends, may, could, might, will, should, or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements include but are not limited to statements regarding our clinical development activities and related anticipated timelines.

本演示文稿包含 1995 年《私人證券訴訟改革法案》含義內的前瞻性陳述，這些陳述存在風險和不確定性。在某些情況下，我們可能會使用預測、相信、潛力、提議、繼續、估計、預期、期望、計劃、打算、可能、可能、可能、將、應該等術語，或表達未來事件或結果的不確定性的其他詞語識別這些前瞻性陳述。此類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。

Such statements are subject to numerous important risk factors and uncertainties and may cause actual events or results to differ materially from our current expectations. 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, and the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this presentation speak only as of the date of this 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 second quarter of 2023 has been filed.

此類陳述受到許多重要風險因素和不確定性的影響，可能導致實際事件或結果與我們當前的預期存在重大差異。這些和其他風險和不確定性在我們向美國證券交易委員會(SEC) 提交的定期文件中進行了更全面的描述，包括標題為“風險因素”的部分中描述的風險因素，以及我們向SEC 提交的季度和年度報告。我們在本演示文稿中所做的任何前瞻性陳述僅代表本演示文稿發布之日的情況。除法律要求外，我們不承擔更新本演示文稿中包含的前瞻性陳述的義務，無論是由於新信息、未來事件還是本演示文稿發布後的其他情況。最後，Ocugen 涵蓋 2023 年第二季度的 10-Q 表格季度報告已提交。

I will now turn the call to Dr. Musunuri.

我現在將電話轉給 Musunuri 博士。

Thank you Tiffany. Good morning and thank you all for joining us today.

謝謝蒂芙尼。早上好，感謝大家今天加入我們。

The second quarter of 2023 marked a period of continued progress toward our regulatory and clinical milestones, which we are dedicated to advancing through end of the year. With a strategic focus on our novel modified gene therapy and biologic-based ophthalmic programs, we expect to begin dosing patients across these platforms by the end of this year. We are on track to initiate OCU400 Phase 3 adult trial near the end of 2023, early 2024, subject to outcome of ongoing Phase 1/2 trial and discussions with FDA on proposed Phase 3 trial plan. We also anticipate a clinical study results update for OCU400 this quarter.

2023 年第二季度標誌著我們朝著監管和臨床里程碑不斷取得進展的時期，我們致力於在年底前實現這一目標。我們的戰略重點是新型改良基因療法和基於生物的眼科項目，我們預計在今年年底前開始通過這些平台給患者用藥。我們有望在 2023 年底、2024 年初啟動 OCU400 3 期成人試驗，具體取決於正在進行的 1/2 期試驗的結果以及與 FDA 就擬議的 3 期試驗計劃進行的討論。我們還預計本季度 OCU400 的臨床研究結果會更新。

Investigational new drug applications were cleared by FDA for OCU410 and OCU410ST for geographic atrophy and Stargardt disease respectively. We plan to initiate Phase 1/2 trials by the end of 2023. We are also planning to initiate the Phase 3 clinical trial for our regenerative cell therapy product candidate, NeoCart, in the second half of 2024. This would mean Ocugen would have late-stage programs in gene and cell therapies in 2024.

OCU410 和 OCU410ST 的研究性新藥申請已獲得 FDA 批准，分別用於治療地理萎縮和 Stargardt 病。我們計劃在 2023 年底啟動 1/2 期試驗。我們還計劃在 2024 年下半年啟動我們的再生細胞療法候選產品 NeoCart 的 3 期臨床試驗。這意味著 Ocugen 將晚於- 2024 年基因和細胞療法階段計劃。

In an effort to conserve our working capital and advance our patient-centric agenda to develop a novel inhaled mucosal vaccine platform, we have submitted multiple proposals to obtain non-dilutive government funding and are having discussions with pertinent agencies to secure their support for our OCU500 vaccine series. Our first-in-class modifier gene therapy to treat multiple inherited retinal diseases remains unmatched industry wide. This unique gene-agnostic approach has the potential to address retinal diseases caused by mutations in multiple genes with one product. Our goal is to build on the innovation of gene therapy and expand its potential to treat a wider population of patients suffering from a host of rare retinal diseases that single gene replacement therapies are unable to address.

為了節省我們的營運資金並推進以患者為中心的議程，以開發新型吸入粘膜疫苗平台，我們已提交多項提案以獲得非稀釋性政府資金，並正在與相關機構進行討論，以確保他們對我們的OCU500 的支持疫苗系列。我們用於治療多種遺傳性視網膜疾病的一流修飾基因療法在整個行業中仍然是無與倫比的。這種獨特的與基因無關的方法有可能用一種產品解決由多個基因突變引起的視網膜疾病。我們的目標是在基因療法的創新基礎上發展其潛力，以治療更廣泛的患有單基因替代療法無法解決的一系列罕見視網膜疾病的患者。

In the second quarter, we were honored to present in detail the mechanism of action and scientific basis for our modified gene therapy platform to preeminent researchers and medical professionals in attendance at the Association for Research in Vision and Ophthalmology and bioinformational conferences. As we advance our clinical agenda, we will continue to identify and secure opportunities to educate stakeholders on the differentiation and potential benefits of this innovative approach to gene therapy.

在第二季度，我們很榮幸地向出席視覺與眼科研究協會和生物信息會議的傑出研究人員和醫學專業人士詳細介紹了我們改進的基因治療平台的作用機制和科學基礎。隨著我們推進臨床議程，我們將繼續尋找並確保機會來教育利益相關者，讓他們了解這種創新基因治療方法的差異性和潛在益處。

In April, we announced encouraging and compelling positive preliminary safety and efficacy results from our OCU400 Phase 1/2 multi-center, open-label, dose-ranging clinical trial in patients afflicted with RP. We believe the preliminary findings from this study support the potential for our modified gene therapy to be a viable alternative to traditional treatments to the increasing population of patients suffering from these diseases. Enrolment is ongoing for all defined subjects in this study, adults with LCA and children between ages of 6 to 17. Pending positive feedback from the FDA, we aim to initiate our Phase 3 adult clinical trial near the end of this year or early 2024.

今年 4 月，我們宣布了針對 RP 患者的 OCU400 1/2 期多中心、開放標籤、劑量範圍臨床試驗取得了令人鼓舞且令人信服的積極初步安全性和有效性結果。我們相信，這項研究的初步結果支持我們的改良基因療法有可能成為越來越多患有這些疾病的患者的傳統療法的可行替代方案。本研究中所有確定的受試者、患有LCA 的成人和6 至17 歲的兒童的招募工作正在進行中。在等待FDA 的積極反饋之前，我們的目標是在今年年底或2024 年初啟動3 期成人臨床試驗。

We continue to execute our comprehensive strategy to develop OCU400 and bring it to market by 2026 with the goal of providing desperately needed treatment options for the estimated 125,000 patients in the US alone that suffer from RP and LCA. In parallel, we'll continue progressing our other modified gene therapy programs to address additional ophthalmic conditions. We believe that upon successful realization of these goals, Ocugen will have built a vast commercial footprint that may hold significant upside for our shareholders and, most importantly, meet a critical medical need for patients.

我們繼續執行開發 OCU400 的綜合戰略，並於 2026 年將其推向市場，目標是僅在美國就為估計 125,000 名患有 RP 和 LCA 的患者提供急需的治療選擇。與此同時，我們將繼續推進其他改良基因治療計劃，以解決其他眼科疾病。我們相信，成功實現這些目標後，Ocugen 將建立龐大的商業足跡，這可能為我們的股東帶來巨大的收益，最重要的是，滿足患者的關鍵醫療需求。

Dry age-related macular degeneration is one of the most prevalent neurodegenerative eye diseases affecting approximately 10 million people in the US and nearly 266 million people worldwide. Dry AMD results in irreversible loss of sight among elderly populations leading to a lack of functional independence that severely impacts quality of life. A variety of biotechnology companies, small and large, are working to develop therapies for dry AMD. However, we believe our OCU410 candidate can offer a less burdensome option for our patients.

乾性年齡相關性黃斑變性是最常見的神經退行性眼病之一，影響著美國約 1000 萬人和全球近 2.66 億人。乾性AMD會導致老年人群不可逆轉的視力喪失，導致缺乏功能獨立性，嚴重影響生活質量。各種大大小小的生物技術公司都在致力於開髮乾性 AMD 的療法。然而，我們相信我們的 OCU410 候選藥物可以為我們的患者提供一個減輕負擔的選擇。

With OCU410, we are again investigating the potential for our novel modifier gene therapy to provide a one-time treatment option that targets all four hallmark conditions of dry AMD, including lipid metabolism, inflammation, oxidative stress, and complement activation. The current standard of care only targets the complement factor, requires multiple injections per year, and has reported side effects. We are excited to initiate the Phase 1/2 clinical trial this year because of the significant global unmet medical need.

通過 OCU410，我們再次研究新型修飾基因療法的潛力，以提供一次性治療選擇，針對乾性 AMD 的所有四種標誌性病症，包括脂質代謝、炎症、氧化應激和補體激活。目前的護理標準僅針對補體因子，每年需要多次注射，並且有副作用報告。由於全球未滿足的醫療需求巨大，我們很高興今年啟動 1/2 期臨床試驗。

Moving onto OCU410ST, we are extremely pleased to receive orphan drug designation from the FDA to address ABCA4-associated retinal diseases such as Stargardt disease, RP19, and cone-rod dystrophy, for which there are currently no treatment options. OCU410ST is a novel modified gene therapy that leverages nuclear hormone receptors to modulate cell activity and deployed as an AAV delivery platform for retinal delivery of the RAR-related orphan receptor A. Nuclear hormone receptors are master gene regulators that help maintain homeostasis by regulating diverse physiological functions such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation, and cell survival networks. We believe that by harnessing the power of nuclear hormone receptors, we can develop one-time treatments that can modulate cell activity disrupted by disease causing gene mutations.

談到 OCU410ST，我們非常高興獲得 FDA 的孤兒藥資格認定，用於治療與 ABCA4 相關的視網膜疾病，如 Stargardt 病、RP19 和視桿細胞營養不良，目前尚無治療選擇。 OCU410ST 是一種新型改良基因療法，利用核激素受體調節細胞活性，並作為AAV 遞送平台用於視網膜遞送RAR 相關孤兒受體A。核激素受體是主要基因調節劑，通過調節多種生理功能來幫助維持體內平衡。光感受器發育和維持、新陳代謝、光轉導、炎症和細胞生存網絡等功能。我們相信，通過利用核激素受體的力量，我們可以開發出一次性治療方法，可以調節因引起疾病的基因突變而破壞的細胞活動。

Now turning to our efforts to develop a series of next-generation inhalation vaccines, for which the company intends to submit an IND application in 2024 pending government funding. In multiple preclinical trials, mucosal vaccines have demonstrated vaccine-induced high neutralization titer and effector responses. Inhaled mucosal vaccines represent a distinct product candidate profile that could help remedy major global health challenges and maximize our opportunity to serve a broader cross section of patients via less invasive delivery mechanism with a potential for superior durability when compared with current intramuscular administration.

現在轉向我們開發一系列下一代吸入疫苗的努力，公司計劃於 2024 年提交 IND 申請，等待政府資助。在多項臨床前試驗中，粘膜疫苗已證明疫苗誘導的高中和滴度和效應反應。吸入粘膜疫苗代表了一種獨特的候選產品特徵，可以幫助解決全球主要健康挑戰，並最大限度地利用侵入性較小的給藥機制為更廣泛的患者提供服務，與目前的肌肉注射相比，具有卓越的耐久性潛力。

Clinical studies using a similar vector of inhaled administration have shown mucosal antibodies, systemic antibodies, and durable immune response up to one year with one-fifth of the dose compared to traditional intramuscular vaccines. Greater ease of administration presents the potential for improved vaccination compliance and wider adoption, particularly among traditionally underserved populations and throughout the developing world.

使用類似的吸入給藥載體的臨床研究表明，與傳統肌肉注射疫苗相比，只需五分之一的劑量即可產生粘膜抗體、全身抗體和長達一年的持久免疫反應。更方便的管理有可能提高疫苗接種依從性和更廣泛的採用，特別是在傳統上服務不足的人群和整個發展中國家。

Current COVID-19 vaccines are limited by lack of durability and marginal ability to prevent transmission. As a part of our commitment to address barriers to widespread vaccination to protect against COVID-19, we are developing this inhaled vaccine platform that includes OCU500, a bivalent COVID-19 inhaled vaccine; OCU510, a seasonal quadrivalent flu inhaled vaccine; and OCU520, a combination quadrivalent seasonal flu and bivalent COVID-19 inhaled vaccine. The OCU500 vaccine series is based on a novel shared platform designed to reduce transmission and protect against new variants with potential durability up to one year.

目前的 COVID-19 疫苗因缺乏耐用性和預防傳播的能力有限而受到限制。作為我們消除廣泛接種疫苗以預防 COVID-19 的障礙的承諾的一部分，我們正在開發這種吸入疫苗平台，其中包括 OCU500，一種二價 COVID-19 吸入疫苗； OCU510，一種季節性四價流感吸入疫苗； OCU520，一種四價季節性流感和二價 COVID-19 吸入疫苗。 OCU500 疫苗系列基於一個新穎的共享平台，旨在減少傳播並防止新變種，潛在的有效期長達一年。

To optimize resources across our diverse and critically needed development programs and maintain shareholder value, our team has been engaging with public health officials and federal government agencies to pursue non-dilutive funding to support the development of our OCU500 vaccine series. We maintain an ongoing dialogue with respective agencies and anticipate receiving further information on the status of our pending request later this year.

為了優化我們多樣化和急需的開發計劃的資源並維護股東價值，我們的團隊一直在與公共衛生官員和聯邦政府機構合作，尋求非稀釋資金來支持我們的 OCU500 疫苗系列的開發。我們與相關機構保持持續對話，並預計在今年晚些時候收到有關我們未決請求狀態的進一步信息。

Earlier this year, the FDA notified us that they were putting a hold on our OCU200 program and requested additional information related to chemistry, manufacturing, and controls. We are working with the FDA to release the hold and expect the Phase 1 trial to be initiated in Q4 2023. We believe OCU200 works with a distinct mechanism of action compared to existing therapies for the treatment of diabetic macular edema and targets multiple causative pathways such as angiogenesis, oxidation, and inflammation, and has potential to offer better treatment to all patients.

今年早些時候，FDA 通知我們，他們將暫停我們的 OCU200 項目，並要求提供與化學、製造和控制相關的更多信息。我們正在與FDA 合作解除擱置，並預計1 期試驗將於2023 年第四季度啟動。我們相信，與現有的治療糖尿病性黃斑水腫的療法相比，OCU200 具有獨特的作用機制，並針對多種致病途徑，例如血管生成、氧化和炎症等，並有潛力為所有患者提供更好的治療。

NeoCart is our Phase 3-ready regenerative cell therapy technology that combines novel advancement in bioengineering and cell processing to enhance the autologous cartilage repair process. Manufacturing facility construction for NeoCart is on target to be completed by end of 2023 as planned. The company plans to initiate the Phase 3 trial in subjects with articular cartilage defect in the second half of 2024. We are highly dedicated to completing our stated objectives with the strategies we believe will enable Ocugen to reach several value-enhancing milestones and are planning to file BLAs across all first-in-class platform technologies, gene therapies, cell therapies, and vaccines in the next three to five years.

NeoCart 是我們的第三階段再生細胞治療技術，它結合了生物工程和細胞處理方面的新進展，以增強自體軟骨修復過程。 NeoCart 的生產設施建設預計按計劃於 2023 年底完成。該公司計劃於2024 年下半年在患有關節軟骨缺陷的受試者中啟動3 期試驗。我們高度致力於通過我們相信將使Ocugen 實現多個價值提升里程碑的策略來完成我們既定的目標，並計劃在未來三到五年內提交所有一流平台技術、基因療法、細胞療法和疫苗的 BLA。

I will now provide an overview of the key financial results for second-quarter 2023.

我現在將概述 2023 年第二季度的主要財務業績。

Our research and development expenses for the quarter ended June 30, 2023 were $14.2 million compared to $9 million for the second quarter of 2022. This included a non-recurring, non-cash expense of $4.4 million as a result of the impairment of the short-term asset for the advanced payment of the supply of COVAXIN as well as the associated loss on the disposal of related fixed assets.

截至 2023 年 6 月 30 日的季度，我們的研發費用為 1,420 萬美元，而 2022 年第二季度為 900 萬美元。其中包括因短期減值而產生的 440 萬美元的非經常性非現金費用。 -用於預付COVAXIN供應的定期資產以及相關固定資產處置的相關損失。

General and administrative expenses for the quarter ended June 30, 2023 were $9.6 million compared to $10.6 million for the second quarter of 2022. Net loss was approximately $22.9 million or $0.10 net loss per share for the quarter ended June 30, 2023 compared to a net loss of approximately $19.5 million or $0.09 net loss per share for the second quarter of 2022. Our cash, cash equivalents, and investments totaled $70.6 million as of June 30, 2023 compared to $90.9 million as of December 31, 2022.

截至2023 年6 月30 日的季度的一般和管理費用為960 萬美元，而2022 年第二季度為1,060 萬美元。截至2023 年6 月30 日的季度的淨虧損約為2,290 萬美元，即每股淨虧損0.10 美元。 2022 年第二季度虧損約1,950 萬美元，即每股淨虧損0.09 美元。截至2023 年6 月30 日，我們的現金、現金等價物和投資總額為7,060 萬美元，而截至2022年12 月31 日為9,090 萬美元。

In May, we closed a public offering of 30 million shares of common stock for gross proceeds of $16.5 million. Net proceeds from the offering are being used for general corporate purposes, capital expenditures, working capital, general and administrative expenses, and R&D. We are continuously exploring opportunities to increase our working capital, and we'll be focused on seeking out corporate partnerships for gene therapies and non-dilutive funding for vaccines.

5 月份，我們完成了 3000 萬股普通股的公開發行，募集資金總額為 1650 萬美元。此次發行的淨收益將用於一般公司用途、資本支出、營運資金、一般和管理費用以及研發。我們正在不斷探索增加營運資金的機會，我們將專注於尋找基因療法的企業合作夥伴和疫苗的非稀釋資金。

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

我的本季度更新到此結束。蒂芙尼，回到你身邊。

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

謝謝尚卡。我們現在開始提問。操作員？

(Operator Instructions) Jennifer Kim, Cantor Fitzgerald.

（操作員說明）Jennifer Kim、Cantor Fitzgerald。

I have two. The first is, as you're thinking about cash burn going forward and you're seeking non-dilutive funding opportunities, excluding the one-time impairment expense, is this quarter a good basis as we think about quarterly burn? Then my second question is on the OCU400 program. Can you remind us what you're looking for in that updated data this quarter as sort of the go, no-go for the Phase 3 adult trial?

我有兩個。第一個問題是，當您考慮未來的現金消耗並尋求非稀釋性融資機會（不包括一次性減值費用）時，本季度是我們考慮季度燒錢的良好基礎嗎？那麼我的第二個問題是關於OCU400程序的。您能否提醒我們，您在本季度的更新數據中尋找什麼作為第三階段成人試驗的可行或不可行的內容？

Let me address the first question, then Arun is going to take the second one. Yes, there is a one-time impairment charge. There is also a non-cash stock comp charge of $2.6 million. And if you add those two, the cash comes out to be $15.9 million total per quarter, so that would be a good guidance for you for the future.

我先回答第一個問題，然後阿倫來回答第二個問題。是的，存在一次性減值費用。還有 260 萬美元的非現金股票補償費用。如果將這兩者相加，每個季度的現金總額為 1590 萬美元，因此這對您的未來來說將是一個很好的指導。

I'll let Arun address the other question on the OCU400 program.

我將讓 Arun 解決有關 OCU400 程序的另一個問題。

So, yeah, you are right. I think our -- this quarter update on OCU400 clinical Phase 1/2 data will guide us about our Phase 3 study. Yeah, that's the data that will be used as a basis for go, no-go decision for Phase 3.

所以，是的，你是對的。我認為本季度 OCU400 臨床 1/2 期數據的更新將指導我們進行 3 期研究。是的，這些數據將用作第三階段進行或不進行決策的基礎。

Okay, and can you remind us what you're looking for in that data?

好的，您能提醒我們您在這些數據中尋找什麼嗎？

So primarily we are looking at the functional improvement in the patients treated with OCU400, and the focus is going to be the RP patient.

因此，我們主要關注接受 OCU400 治療的患者的功能改善，重點是 RP 患者。

Okay, and then in your discussions with the FDA later on for the Phase 3 trial, is that going to focus on RP patients or are you also considering the inclusion of LCA patients?

好的，然後在您稍後與 FDA 就 3 期試驗進行討論時，是重點關注 RP 患者還是也考慮納入 LCA 患者？

So to begin with, we'll start with the RP patient, and as we collect the data for LCA patient, then later we will include LCA. But to begin with, yeah, we are planning to go with enrolled RP patient.

首先，我們將從 RP 患者開始，當我們收集 LCA 患者的數據時，稍後我們將包括 LCA。但首先，是的，我們計劃與登記的 RP 患者一起進行。

All right.

好的。

William Ramakanth, H.C. Wainwright.

威廉·拉瑪坎特 (William Ramakanth)，H.C.溫賴特。

This is RK from H.C. Wainwright. So a couple of quick questions on the 400 program, and then maybe one on the NeoCart. On the 400, so in terms of your discussions with the FDA, is that being planned once you see the data on the Phase 1/2, or you have already initiated some initial conversations and started putting together a Phase 3 plan?

我是 H.C. 的 RK。溫賴特。我想問幾個關於 400 計劃的快速問題，然後可能還有一個關於 NeoCart 的問題。在 400 方面，就您與 FDA 的討論而言，一旦您看到 1/2 階段的數據，是否正在計劃，或者您已經啟動了一些初步對話並開始製定 3 階段計劃？

Thanks RK. So we have not initiated our discussions with FDA yet, so once we complete the data analysis, only then we are going to reach out to FDA. But that is planned for this quarter related to the top data and followed by reaching out to FDA.

謝謝 RK。所以我們還沒有開始與 FDA 進行討論，所以一旦我們完成了數據分析，我們才會與 FDA 聯繫。但計劃在本季度與頂級數據相關，然後聯繫 FDA。

Thank you for that. And then on the 410 program, in terms of -- now that you have already been cleared by the FDA, what else needs to get done before you can initiate the Phase 1/2 study?

謝謝你。然後就 410 計劃而言，既然您已經獲得 FDA 的批准，那麼在啟動 1/2 期研究之前還需要做什麼？

Just we need to get the site ready and initiate the patient screening and -- so that's why I think we are planning to launch first subject in this study, both GA as well as Stargardt, this year. So it's more like getting ready with the clinical site.

只是我們需要準備好場地並啟動患者篩查，所以我認為我們計劃今年啟動這項研究的第一個主題，即 GA 和 Stargardt。所以這更像是為臨床站點做好準備。

Got it, got it. Then on the NeoCart program, I'm just trying to understand, if the facility does get completed by the end of '23, what else needs to get done in terms of commissioning the plant, getting the clinical material ready to start your program on the Phase 3 study by second half? Is it just that, or is it -- you still have to design the protocol? I'm just trying to understand what all -- because we have been talking about this program for almost a year and a half now.

明白了，明白了。然後在 NeoCart 項目上，我只是想了解，如果該設施確實在 23 年底完工，那麼在調試工廠、準備好臨床材料以啟動您的項目方面還需要做什麼下半年進行第三階段研究嗎？僅僅是這樣，還是——你仍然需要設計協議？我只是想了解這一切——因為我們已經討論這個項目近一年半了。

Yeah, so RK, the facility construction will be completed at the GMP facility by end of this year. It's on target. And then, as you know, as a GMP facility, it takes a few months for getting the qualification done, and then they'll be ready to produce NeoCart in the facility. In the interim, obviously the team is going to prepare CMC and clinical sections and will continue to update those, so that they're ready for submission next year before they start the clinical trial.

是的，RK，GMP 設施的設施建設將於今年年底完成。正中目標。然後，如您所知，作為 GMP 工廠，需要幾個月的時間才能完成資格認證，然後他們將準備在該工廠生產 NeoCart。在此期間，顯然該團隊將準備 CMC 和臨床切片，並將繼續更新這些切片，以便在明年開始臨床試驗之前準備好提交。

We do have automatic designation, right. I just wanted to remind, Regenerative Medicine Advanced Therapy designation with FDA, so when we have any questions in the interim, we can always reach out to them and get clarifications.

我們確實有自動指定，對吧。我只是想提醒一下，再生醫學高級療法已獲得 FDA 指定，因此當我們在此期間有任何疑問時，我們可以隨時聯繫他們並獲得澄清。

Perfect.

完美的。

Robert LeBoyer, Noble Capital Markets.

羅伯特·勒博耶，Noble Capital Markets。

My question has to do with the upcoming data presentation, and I was wondering if you could disclose whether the data to be presented will update the previous data on all patients or whether it will just be in new patients that haven't been reported, and wondering if there are any end points that you could share with us at this point.

我的問題與即將到來的數據演示有關，我想知道您是否可以透露將要演示的數據是否會更新所有患者的先前數據，或者是否僅涉及尚未報告的新患者，以及想知道此時您是否可以與我們分享任何終點。

So, yeah, definitely we we'll be providing detailed update when we present this data to the market. But to address your first part of the question whether it is going to include the patient we presented in our previous disclosure, yes, so we will include those subjects as well as some new subjects which have completed additional follow-up visit. So it will be a combination of both.

所以，是的，當我們向市場展示這些數據時，我們肯定會提供詳細的更新。但為了解決你的問題的第一部分，是否會包括我們在之前的披露中介紹的患者，是的，所以我們將包括這些受試者以及一些已完成額外隨訪的新受試者。所以這將是兩者的結合。

Great. And you had mentioned corporate partnerships for the gene therapy. Can you discuss any type of arrangements, whether they're going to be research and development or just marketing, or any objectives to the business development activities?

偉大的。您提到了基因療法的企業合作夥伴關係。您能否討論任何類型的安排，無論是研發還是營銷，或者業務開發活動的任何目標？

I mean, Robert, this is a -- yeah, it's a loaded question. Obviously, we'll be open to -- when you seek partnerships at this stage as a biotech, big pharma established with an infrastructure and everything else, obviously your first target is going to be commercial. As you know, complex science is involved in these clinical trials. Obviously when you're going into Phase 3, once we have the data out, we'll obviously work very hard with any potential partners.

我的意思是，羅伯特，這是一個——是的，這是一個沉重的問題。顯然，當您在現階段尋求與生物技術公司、擁有基礎設施和其他一切的大型製藥公司建立合作夥伴關係時，我們將持開放態度，顯然您的第一個目標將是商業化。如您所知，這些臨床試驗涉及復雜的科學。顯然，當進入第三階段時，一旦我們有了數據，我們顯然會與任何潛在的合作夥伴非常努力地合作。

And obviously, as you know, if they are interested in commercial development, they will be interested in Phase 3 programs, too. So I think we will keep our options open, whatever can maximize our value for Ocugen as well as make sure we have ability to provide market access to patients who desperately need this product.

顯然，如您所知，如果他們對商業開發感興趣，他們也會對第三階段項目感興趣。因此，我認為我們將保持開放的選擇，只要能夠最大限度地提高 Ocugen 的價值，並確保我們有能力為迫切需要該產品的患者提供市場准入。

Okay, great.

好的，太好了。

Daniil Gataulin, Chardan.

丹尼爾·加陶林，查丹。

I've got a couple, one for OCU410 program. Just wanted to ask, strategically, how do you see it positioned long term, whether it's viewed as a standalone approach or in combination with anything else, and the patient population that you will be targeting? And the second question is for the inhaled vaccine series. Can you comment on your interactions to date regarding the funding, and particularly if you can comment on the interest in this program given that there are several other approved options?

我有幾個，一個用於 OCU410 程序。只是想問，從戰略上講，您如何看待它的長期定位，無論是將其視為一種獨立的方法還是與其他任何方法相結合，以及您將瞄準的患者群體？第二個問題是關於吸入疫苗系列。您能否評論一下迄今為止有關資金的互動，特別是考慮到還有其他幾個已批准的選項，您能否評論一下對該計劃的興趣？

Thank you. So I'll take the first question. Yeah, we are thinking of taking just the standalone product, and to begin with, definitely we'll be targeting the advanced form of AMD, that is geographic atrophy. But subsequently, based on the outcome, it may be further developed for early and intermediate stage depending on the clinical benefit coming from the GA trial.

謝謝。那麼我來回答第一個問題。是的，我們正在考慮只採用獨立產品，首先，我們肯定會瞄準 AMD 的高級形式，即地理萎縮。但隨後，根據結果，可能會根據 GA 試驗的臨床效益進一步開發早期和中期階段。

And regarding like how we see this product against other what we see in the market, so as you all know that AMD is a multifactorial disease, and so far, we have been seeing in this space that most of the companies are focused on targeting only one pathway. And by like very nature of this disease, being multifactorial in nature, the various causes which lead to this disease. So our product has potential to target all those pathways which are linked to dry AMD pathogenesis. So we believe that this could be a differentiated product and has potential to offer better clinical benefit compared to what we have right now.

關於我們如何看待這款產品與我們在市場上看到的其他產品的對比，大家都知道 AMD 是一種多因素疾病，到目前為止，我們在這個領域看到大多數公司只專注於目標一條路徑。就這種疾病的本質而言，其本質上是多因素的，導致這種疾病的原因多種多樣。因此，我們的產品有潛力針對所有與乾性 AMD 發病機制相關的途徑。因此，我們相信這可能是一種差異化產品，並且與我們現在擁有的產品相比，有可能提供更好的臨床效益。

The second question related to government funding, again, we have been working with various agencies, and we'll provide an update. Obviously, the current products, as you know, the vaccines we have especially for COVID, they lack a couple of things. One, they are struggling to control the transmission. There's a lot of data available right now. And also, they lack durability. So what would be an ideal option going into the future? I think there is definitely a need for mucosal vaccines. The scientific community agrees on that, and also, that will provide systemic as well as mucosal immunity, so it can actually potentially prevent at the target of viral entry into mucosal systems.

第二個問題與政府資助有關，我們一直在與各個機構合作，我們將提供最新情況。顯然，目前的產品，如你所知，我們專門針對新冠病毒的疫苗，它們缺乏一些東西。第一，他們正在努力控制傳播。現在有大量數據可用。而且，它們缺乏耐用性。那麼，未來的理想選擇是什麼？我認為肯定需要粘膜疫苗。科學界同意這一點，而且這將提供系統和粘膜免疫，因此它實際上可以潛在地阻止病毒進入粘膜系統。

And the second thing is durability. We believe and the scientific community believes that COVID vaccinations, in order to gain the compliance with the public, you cannot keep on vaccinating every three months. That's why people get vaccine fatigue, so you need to move into annual vaccinations such as flu, so your compliance here could go up, so that's the intent. I think, as we stated, there are ex-US trials with inhalation vaccines using a similar technology, sure durability up to one year. That's really important.

第二件事是耐用性。我們相信，科學界也相信，新冠疫苗接種，為了獲得公眾的配合，不能繼續每三個月接種一次。這就是為什麼人們會感到疫苗疲勞，所以你需要每年接種流感疫苗，這樣你的依從性就會提高，這就是目的。我認為，正如我們所說，美國以外已經有使用類似技術的吸入疫苗試驗，其耐久性肯定可達一年。這真的很重要。

So there are two things, controlling transmission and durability are very important for next-gen vaccines. And we believe our platform technology, inhalation vaccines for COVID and flu, they can provide that.

所以有兩件事，控制傳播和耐久性對於下一代疫苗非常重要。我們相信我們的平台技術、新冠肺炎和流感吸入疫苗可以提供這一點。

Got it.

知道了。

This concludes the Q&A portion. I will now turn the call back over to Chairman and CEO, Dr. Shankar Musunuri.

問答部分到此結束。現在我將把電話轉回給董事長兼首席執行官尚卡爾·穆蘇努里 (Shankar Musunuri) 博士。

Thank you, operator. In closing, I would like to recognize the entire team for their resilient efforts to advance our patient-centric mission. To our shareholders and partners, thank you for your ongoing trust and support. We are already well into the second half of 2023 and are steadfast in our commitment to transparency, informed decision-making based on sound scientific principles, and a tireless work ethic dedicated to excellence in all phases of research, development, and clinical testing.

謝謝你，接線員。最後，我要感謝整個團隊為推進我們以患者為中心的使命所做的堅韌努力。感謝我們的股東和合作夥伴一直以來的信任和支持。我們已經進入 2023 年下半年，並堅定地致力於透明度、基於合理科學原則的明智決策以及致力於在研究、開發和臨床測試的所有階段追求卓越的不懈職業道德。

We remain confident that we'll be able to fulfill our mission of developing novel therapies with innovative discovery to bring to market effective treatments for patients suffering from a range of conditions that currently lack treatment options. We look forward to sharing more details on our progress in the coming months.

我們仍然相信，我們將能夠完成通過創新發現開發新療法的使命，為患有目前缺乏治療選擇的一系列疾病的患者帶來有效的治療方法。我們期待在未來幾個月分享更多有關我們進展的細節。

Thanks, everyone, have a great day.

謝謝大家，祝你有美好的一天。