Ocugen Inc (OCGN) 2023 Q1 法說會逐字稿

Good morning. Welcome to the Ocugen First Quarter 2023 Financial Results and Business Update Call. Please note that this call is being recorded at this time. (Operator Instructions) I will now turn the call over to Sharon Choe, Ocugen's Head of Investor Relations. You may begin.

早上好。歡迎來到 Ocugen 2023 年第一季度財務業績和業務更新電話會議。請注意，此次通話正在錄音中。 （操作員說明）我現在將電話轉給 Ocugen 的投資者關係主管 Sharon Choe。你可以開始了。

Thank you, [Monte]. Joining me today are Ocugen's Chairman, CEO and Co-Founder, Dr. Shankar Musunuri, who will provide a business update; and our Chief Financial Officer and Chief Business Officer, Quan Vu, who will provide a financial update.

謝謝你，[蒙特]。今天加入我的是 Ocugen 的董事長、首席執行官兼聯合創始人 Shankar Musunuri 博士，他將提供業務更新；我們的首席財務官兼首席商務官 Quan Vu 將提供財務更新。

Earlier this morning, we issued a press release detailing business and operational highlights for the first quarter of 2023. We encourage listeners to review the press release, which is available on our Web site at www.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 Web site for approximately 45 days.

今天上午早些時候，我們發布了一份新聞稿，詳細介紹了 2023 年第一季度的業務和運營亮點。我們鼓勵聽眾閱讀新聞稿，該新聞稿可在我們的網站 www.ocugen.com 上獲取。此通話正在錄製中，Ocugen 網站的“投資者”部分將提供隨附幻燈片演示的重播，時間約為 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.

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，這些陳述受風險和不確定因素的影響。

We may in some cases, use terms such as predict, believe, potential, propose, continue, estimate, anticipate, expect, plan, intend, may, could, might, will, should, or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks and uncertainties and may cause actual events or results to differ materially from our current expectations. Investors should familiarize themselves with the company's filings for complete details.

在某些情況下，我們可能會使用預測、相信、潛在、建議、繼續、估計、預期、期望、計劃、打算、可能、可能、可能、將、應該或其他表達未來事件不確定性的詞語或結果來識別這些前瞻性陳述。此類陳述受許多重要因素、風險和不確定性的影響，並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。投資者應熟悉公司的文件以獲取完整的詳細信息。

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 2023 will be filed soon after today's call. I will now turn the call to Dr. Musunuri.

除非法律要求，否則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日期之後的其他原因。最後，Ocugen 關於 10-Q 表的季度報告涵蓋了 2023 年第一季度，將在今天的電話會議後不久提交。我現在將電話轉給 Musunuri 博士。

Thank you, Sharon. Good morning and thank you all for joining us today. Looking at what we have achieved since we reported our 2022 fourth quarter and full year results, I'm kicking off today's call with a high sense of accomplishment and optimism for the future of Ocugen.

謝謝你，莎倫。早上好，感謝大家今天加入我們。看看自我們報告 2022 年第四季度和全年業績以來我們取得的成就，我帶著高度的成就感和對 Ocugen 未來的樂觀情緒開始今天的電話會議。

At the top of our list of highlights is the recent announcement of positive preliminary safety and efficacy results from the Phase 1/2 trial of our OCU400 program and the FDA's Orphan Drug Designation for our OCU410ST program to potentially treat ABCA4-associated retinopathies such as Stargardt disease.

在我們的亮點列表的頂部是最近宣布我們的 OCU400 計劃的 1/2 期試驗的積極初步安全性和有效性結果以及我們的 OCU410ST 計劃的 FDA 孤兒藥物指定可能治療 ABCA4 相關的視網膜病，如 Stargardt疾病。

We also forged ahead in our pursuit of non-dilutive government funding to support our inhaled vaccines pipeline and submitted multiple proposals to various federal agencies. And we will begin seeking corporate partnerships for our gene therapies. I will also share updates on our OCU200 and NeoCart programs later in my commentary.

我們還在追求非稀釋性政府資金以支持我們的吸入疫苗管道方面取得了進展，並向各個聯邦機構提交了多項提案。我們將開始為我們的基因療法尋求企業合作夥伴關係。我還將在稍後的評論中分享我們 OCU200 和 NeoCart 程序的更新。

2023 is off to a strong start. And you can see we remain on track to achieve the significant milestones for the year that we first shared with you during the last business update webcast. Our modified gene therapy approach continues to be the leading differentiator for Ocugen.

2023 年開局良好。您可以看到，我們仍有望實現我們在上一次業務更新網絡廣播中首次與您分享的今年的重要里程碑。我們改良的基因治療方法仍然是 Ocugen 的主要差異化因素。

Unlike single gene replacement therapies, which only target one genetic mutation, we believe that our modifier gene therapy platform through its use of nuclear hormone receptors that presents a novel approach that has the potential to both address multiple retinal diseases caused by mutations in multiple genes with one product and to address complex diseases that are potentially caused by imbalances in multiple gene networks.

與僅針對一個基因突變的單基因替代療法不同，我們相信我們的修飾基因治療平台通過使用核激素受體提供了一種新方法，有可能同時解決由多個基因突變引起的多種視網膜疾病一種產品，並解決可能由多個基因網絡失衡引起的複雜疾病。

Currently, Ocugen has three modifier gene therapy programs, OCU400 for retinitis pigmentosa and Leber congenital amaurosis, which affects approximately 125,000 patients in the U.S. living with any of more than 125 associated mutated genes; OCU410 for dry age-related macular degeneration, a disease affecting approximately 10 million people in the U.S. alone; and OCU410ST for the treatment of ABCA4-associated retinopathies including Stargardt, retinitis pigmentosa, RP19, and cone-rod dystrophy 3, CORD3, diseases affecting 44,000 Americans.

目前，Ocugen 擁有三個修飾基因治療項目，OCU400 用於治療色素性視網膜炎和 Leber 先天性黑蒙，這影響了美國大約 125,000 名患者，他們生活在超過 125 個相關突變基因中； OCU410用於乾性年齡相關性黃斑變性，這種疾病僅在美國就影響了大約 1000 萬人； OCU410ST 用於治療 ABCA4 相關的視網膜病，包括 Stargardt、視網膜色素變性、RP19 和錐桿營養不良 3、CORD3，這些疾病影響了 44,000 名美國人。

We recently announced positive preliminary safety and efficacy results from the Phase 1/2 trial of OCU400 for the treatment of retinitis pigmentosa and Leber congenital amaurosis. These preliminary positive results serve as the first clinical validation of the platform where patient responses across various genetic mutations support that OCU400 has the potential to transform the lives of many patients who are struggling with debilitating blindness diseases.

我們最近公佈了 OCU400 治療視網膜色素變性和 Leber 先天性黑蒙的 1/2 期試驗的積極初步安全性和有效性結果。這些初步的積極結果是該平台的首次臨床驗證，患者對各種基因突變的反應支持 OCU400 有可能改變許多正在與使人衰弱的失明疾病作鬥爭的患者的生活。

This Phase 1/2 trial is a multicenter, open label, dose ranging study. We have enrolled a total of 18 RP patients in this study, with 10 subjects in the dose escalation and eight subjects in the expansion phase. The age of subjects enrolled to date ranges from 18 to 77 years across rhodopsin and NR2E3 gene mutations. We further expanded this Phase 1/2 trial to enroll LCA patients with the CEP290 gene mutation and pediatric patients with NR2E3, RHO and CEP290 mutations.

該 1/2 期試驗是一項多中心、開放標籤、劑量範圍研究。我們在這項研究中總共招募了 18 名 RP 患者，其中 10 名受試者處於劑量遞增階段，8 名受試者處於擴展階段。迄今為止，參與研究的受試者年齡範圍為 18 至 77 歲，涉及視紫紅質和 NR2E3 基因突變。我們進一步擴大了這項 1/2 期試驗，以招募具有 CEP290 基因突變的 LCA 患者和具有 NR2E3、RHO 和 CEP290 突變的兒科患者。

In Cohort 1, which is low dose, and Cohort 2, which is medium dose, a total of seven subjects with moderate to advanced visual impairment due to RP-associated with RHO and NR2E3 gene mutations received a unilateral subretinal injection of either a low dose, which is 1.66 x 10 to the 10 vg per mL in Cohort 1 or medium dose, which is 3.33 x 10 to the 10 vg per mL of OCU400 in Cohort 2, respectively.

在低劑量隊列 1 和中劑量隊列 2 中，共有 7 名因 RP 相關 RHO 和 NR2E3 基因突變而導致中度至重度視力障礙的受試者接受了單側視網膜下注射低劑量，在隊列 1 或中等劑量中為 1.66 x 10 至每毫升 10 vg，在隊列 2 中分別為 3.33 x 10 至每毫升 OCU400 的 10 vg。

In the preliminary data analysis, nine-month follow-up data for three subjects in Cohort 1 and six-month follow-up data for one subject from Cohort 1 and three subjects from Cohort 2 were assessed. Overall, preliminary results showed a favorable safety and tolerability profile for OCU400.

在初步數據分析中，對隊列 1 中三名受試者的九個月隨訪數據以及隊列 1 中一名受試者和隊列 2 中三名受試者的六個月隨訪數據進行了評估。總體而言，初步結果顯示 OCU400 具有良好的安全性和耐受性。

Regarding efficacy, we looked at multi-luminescence mobility tests, or MLMT, a primary efficacy endpoint used in clinical trials for an FDA approved product in this disease area, and best corrected visual acuity, or BCVA. Key efficacy outcomes from seven subjects demonstrated four key points. 100% of treated eyes showed a stable or improved MLMT score trend.

關於功效，我們研究了多發光流動性測試或 MLMT，這是 FDA 批准的產品在該疾病領域的臨床試驗中使用的主要功效終點，以及最佳矯正視力或 BCVA。七名受試者的關鍵療效結果證明了四個關鍵點。 100% 的治療眼顯示出穩定或改善的 MLMT 評分趨勢。

71% of our OCU400 treated eyes demonstrated a one or more Lux level improvement in MLMT score compared to 29% of untreated eyes. 67% of our OCU400 treated eyes in Cohort 1 with the nine-month follow up demonstrated a two or more Lux level improvement in MLMT score compared to none of the untreated eyes. And 43% of OCU400 treated eyes demonstrated 8 to 11 letters of improvements in BCVA score compared to none of the untreated eyes.

71% 的 OCU400 治療眼睛在 MLMT 評分中表現出一個或多個 Lux 水平改善，而未經治療的眼睛為 29%。在 9 個月的隨訪中，隊列 1 中 67% 的 OCU400 治療眼睛與未治療的眼睛相比，MLMT 評分的 Lux 水平提高了兩倍或更多。與未經治療的眼睛相比，43% 的 OCU400 治療眼睛在 BCVA 評分上有 8 到 11 個字母的改善。

The early results from patients treated in the Phase 1/2 trial are encouraging and support the paradigm changing potential of our modifier gene therapy technology to address unmet medical needs for patients with RP and LCA. With this favorable safety profile and positive trend in efficacy signals, we are very eager to see longer term data and to potentially initiate Phase 3 clinical trials in the U.S. and EU.

在 1/2 期試驗中接受治療的患者的早期結果令人鼓舞，並支持我們的修飾基因治療技術改變範式的潛力，以解決 RP 和 LCA 患者未滿足的醫療需求。憑藉這種良好的安全性和療效信號的積極趨勢，我們非常渴望看到長期數據，並有可能在美國和歐盟啟動 3 期臨床試驗。

As I mentioned earlier, we received exciting news last week that the FDA granted Orphan Drug Designation for OCU410ST AAV5-hRORA for the treatment of ABCA4-associated retinopathies including Stargardt, RP19 and CORD3 diseases. As a refresher, Orphan Drug Designation is granted by the FDA to certain products that show promise in the treatment, prevention or diagnosis of rare and serious diseases affecting fewer than 200,000 people in the United States.

正如我之前提到的，上週我們收到了令人振奮的消息，FDA 授予 OCU410ST AAV5-hRORA 孤兒藥指定資格，用於治療 ABCA4 相關視網膜病變，包括 Stargardt、RP19 和 CORD3 疾病。作為回顧，孤兒藥指定由 FDA 授予某些產品，這些產品在治療、預防或診斷影響美國不到 200,000 人的罕見和嚴重疾病方面顯示出希望。

Additionally, the Orphan Drug Designation status allows for a potential seven-year market exclusivity specifically to the designated orphan use following FDA approval. Other development incentives include the clinical protocol, design assistance, and potentially accelerated review times. This designation represents a noteworthy milestone in our effort to develop innovative treatments for inherited retinal diseases.

此外，孤兒藥物指定狀態允許在 FDA 批准後專門針對指定孤兒使用的潛在七年市場獨占權。其他開發激勵措施包括臨床協議、設計協助和可能加快的審查時間。這一指定代表了我們努力開發遺傳性視網膜疾病創新療法的一個值得注意的里程碑。

And while OCU410ST is intended to treat rare diseases, OCU410 are also targeting the (inaudible) network is aimed at treating dry age-related macular degeneration that affects hundreds of millions of people across the globe. Using our modified gene therapy, we believe OCU410 potentially addresses shortcomings of current treatments for geographic atrophy that affects about 1 million people in the U.S., because it is a broad spectrum approach that has potential as a one-time curative therapy with a single subretinal injection.

雖然 OCU410ST 旨在治療罕見疾病，但 OCU410 還針對（聽不清）網絡，旨在治療影響全球數億人的干性年齡相關性黃斑變性。使用我們改良的基因療法，我們相信 OCU410 有可能解決目前影響美國約 100 萬人的地圖樣萎縮治療的缺點，因為它是一種廣譜方法，具有通過單次視網膜下注射進行一次性治療的潛力.

Now turning to vaccines. The OCU400 series of vaccines in development grants Ocugen a distinct product candidate profile status that could significantly impact major global health obstacles and maximize our opportunity to serve broader patient markets. Current COVID-19 vaccines are limited by a lack of durability and inability to stop transmission.

現在轉向疫苗。正在開發的 OCU400系列疫苗賦予 Ocugen 獨特的產品候選概況地位，這可能會顯著影響全球主要健康障礙，並最大限度地增加我們為更廣泛的患者市場服務的機會。當前的 COVID-19 疫苗因缺乏耐用性和無法阻止傳播而受到限制。

As part of our commitment to address current gaps in the fight against COVID-19, we're developing a novel inhalation 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.

作為我們致力於解決當前抗擊 COVID-19 的差距的承諾的一部分，我們正在開發一種新型吸入疫苗平台，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 吸入疫苗組合。

The OCU500 vaccine series is based on a novel chat platform designed to reduce transmission and protect against new variants with the potential durability up to one year. We decided to develop the flu vaccine in addition to addressing COVID-19, because flu will always be a health concern. There is also longer term business potential as Americans continue to be regularly vaccinated against the flu.

OCU500 疫苗系列基於一個新穎的聊天平台，旨在減少傳播並防止新變種，潛在的耐用性長達一年。除了解決 COVID-19 問題外，我們還決定開發流感疫苗，因為流感始終是一個健康問題。隨著美國人繼續定期接種流感疫苗，還有更長期的商業潛力。

For the 2022 to 2023 flu season, 50% of the U.S. population above six months of age received a seasonal flu shot, representing a market size of more than 170 million doses. To optimize resources across our diverse critically needed development programs and maintain shareholder value, our team has been busy in DC, speaking with the government agencies to pursue non-dilutive funding opportunities for our OCU400 vaccine series.

在 2022 年至 2023 年的流感季節，50% 的 6 個月以上的美國人口接種了季節性流感疫苗，市場規模超過 1.7 億劑。為了優化我們各種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 DC 忙碌，與政府機構交談，為我們的 OCU400 疫苗系列尋求非稀釋性融資機會。

We have submitted multiple comprehensive proposals for review and consideration and maintain an ongoing dialogue with the respective agencies regarding the development of the inhaled vaccines platform. We look forward to updating you as we hear more.

我們已提交多項綜合提案供審查和考慮，並就吸入疫苗平台的開發與各機構保持持續對話。當我們聽到更多信息時，我們期待著為您更新。

Last quarter, we submitted an investigational new drug application, IND, with the U.S. Food and Drug Administration to initiate a Phase 1 trial of OCU200 for treating diabetic macular edema, DME. The IND was placed on clinical hold by the FDA as part of its request for additional information related to chemistry, manufacturing and controls prior to initiating the Phase 1 trial.

上個季度，我們向美國食品和藥物管理局提交了一份研究性新藥申請 IND，以啟動 OCU200 治療糖尿病性黃斑水腫 DME 的第一階段試驗。 IND 被 FDA 置於臨床暫停狀態，作為其在啟動 1 期試驗之前要求提供與化學、製造和控制相關的額外信息的一部分。

The company plans to respond to the FDA promptly to get FDA clearance to initiate the Phase 1 clinical trial. We believe OCU200 works with a distinct mechanism of action compared to existing therapies and targets multiple causative pathways, such as angiogenesis, oxidation, and inflammation, and has the potential to offer better treatment to all patients.

該公司計劃及時回复 FDA，以獲得 FDA 批准啟動 Phase 1 臨床試驗。我們相信，與現有療法相比，OCU200 具有獨特的作用機制，並針對多種致病途徑，如血管生成、氧化和炎症，並有可能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. We are in the process of renovating our facility to accommodate cGMP manufacturing on NeoCart and plan to complete construction in the fourth quarter of 2023, with the Phase 3 randomized controlled study in subjects with articular cartilage defect commencing in 2024.

NeoCart 是我們的 3 期再生細胞治療技術，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。我們正在翻新我們的設施以適應 NeoCart 上的 cGMP 製造，併計劃在 2023 年第四季度完成建設，並於 2024 年開始對關節軟骨缺陷受試者進行的第三階段隨機對照研究。

As you can see, we're highly dedicated to completing our stated objective with sound strategies that we believe will enable Ocugen to reach several value-enhancing milestones over the course of 2023 and beyond. With that, I will now turn the call over to our Chief Financial Officer and Chief Business Officer, Quan Vu, to review our first quarter financial update. Quan?

正如您所看到的，我們高度致力於通過合理的戰略來完成我們既定的目標，我們相信這些戰略將使 Ocugen 能夠在 2023 年及以後達到幾個增值里程碑。有了這個，我現在將把電話轉給我們的首席財務官兼首席商務官 Quan Vu，以審查我們第一季度的財務更新。泉？

Thank you, Shankar, and good morning, everyone. I will now provide an overview of the key financial results for the first quarter of 2023. Our research and development expenses for the quarter ended March 31, 2023 were 9.6 million compared to 7.9 million for the first quarter of 2022. General and administrative expenses for the quarter ended March 31, 2023 were 8.2 million compared to 10.1 million for the first quarter of 2022.

謝謝你，Shankar，大家早上好。我現在將概述 2023 年第一季度的主要財務業績。我們截至 2023 年 3 月 31 日的季度的研發費用為 960 萬，而 2022 年第一季度為 790 萬。一般和管理費用截至 2023 年 3 月 31 日的季度為 820 萬，而 2022 年第一季度為 1010 萬。

Net loss was approximately 16.5 million or $0.07 net loss per share for the first quarter ended March 31, 2023 compared to a net loss of approximately 18 million or $0.09 net loss per share for the first quarter of 2022. Our cash, cash equivalents and investments totaled 76.7 million as of March 31, 2023 compared to 90.9 million as of December 31, 2022. We expect that our cash, cash equivalents and investments balance will enable us to fund operations into the first quarter of 2024.

截至 2023 年 3 月 31 日的第一季度淨虧損約為 1650 萬美元或每股淨虧損 0.07 美元，而 2022 年第一季度的淨虧損約為 1800 萬美元或每股淨虧損 0.09 美元。我們的現金、現金等價物和投資截至 2023 年 3 月 31 日，總計 7670 萬，而截至 2022 年 12 月 31 日，總計 9090 萬。我們預計我們的現金、現金等價物和投資餘額將使我們能夠為 2024 年第一季度的運營提供資金。

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, as Shankar mentioned earlier. That concludes my update for the quarter. Sharon, back to you.

正如 Shankar 之前提到的，我們正在不斷探索增加營運資金的機會，我們將專注於為基因療法和疫苗的非稀釋性資金尋求企業合作夥伴關係。我對本季度的更新到此結束。莎倫，回到你身邊。

Thanks, Quan. We will now open the call for question. [Monte]?

謝謝，泉。我們現在將開始提問。 [蒙特]？

(Operator Instructions) Our first question comes from the line of Jennifer Kim from Cantor Fitzgerald.

（操作員說明）我們的第一個問題來自 Cantor Fitzgerald 的 Jennifer Kim。

Hi. Good morning. Thanks for taking my questions. I have a couple of here. The first is you spoke about seeking potential partnerships with your gene therapy programs. I'm wondering, are you thinking of this on the basis of your lead program or the underlying platform technology or what type of structure are you looking for?

你好。早上好。感謝您回答我的問題。我這裡有幾個。首先是您談到尋求與您的基因治療計劃的潛在合作夥伴關係。我想知道，您是根據您的主導程序或底層平台技術來考慮這個問題，還是您在尋找什麼類型的結構？

And in terms of timing, is that something that you would seek post updated interim data and before initiating a Phase 3 program? And then my second question is on the inhaled vaccine. I know it's hard to -- it's sort of asking a crystal ball, but do you know potentially when you might have some better visibility there?

就時間而言，您是否會在更新的中期數據之後和啟動第 3 階段計劃之前尋求這些東西？然後我的第二個問題是關於吸入疫苗。我知道這很難——這有點像問水晶球，但你知道什麼時候你可能在那裡有更好的能見度嗎？

Jennifer, good morning. I'll let Quan answer the first question and I'll get to the second one.

詹妮弗，早上好。我會讓權回答第一個問題，然後我再說第二個問題。

Hi, Jennifer. So the answer to the first question is seeking partnerships is going to be related to the OCU400 specifically. As we continue to develop and see further data, that serves as further justification in providing more credence to the platform itself. So that in the future will definitely be an evaluative process of how the platform itself can be capitalized.

嗨，詹妮弗。因此，第一個問題的答案是尋求合作夥伴關係，具體與 OCU400 有關。隨著我們繼續開發並看到更多數據，這進一步證明了為平臺本身提供更多可信度。因此，未來肯定會是一個評估平臺本身如何被資本化的過程。

With respect to data, we are initiating conversations now. Because as you -- I'm sure you're well aware, business development takes time. And so in the process, we'd like to establish those relationships, engage in discussion. And as more data comes out, we will continue to share that, and that will facilitate and if not expedite the entire business development process.

關於數據，我們現在正在發起對話。因為正如你一樣——我相信你很清楚，業務發展需要時間。因此，在此過程中，我們希望建立這些關係，參與討論。隨著更多數據的出現，我們將繼續分享這些數據，這將促進甚至加速整個業務發展過程。

And Jennifer, can you repeat your second question related to COVID-19?

Jennifer，你能重複你關於 COVID-19 的第二個問題嗎？

Yes. So the inhaled vaccine platform, do you have -- I know you submitted some proposals. Have you gotten any feedback in terms of when you might get more visibility on that end?

是的。那麼吸入疫苗平台，你有沒有——我知道你提交了一些提案。關於何時可以在這方面獲得更多知名度，您是否收到過任何反饋？

Not yet. And we're actively working with them. When we get more information, we'll let you know.

還沒有。我們正在積極與他們合作。當我們獲得更多信息時，我們會通知您。

Okay. And then maybe if I could sneak one more. The R&D burn for this quarter, is that a good basis when we're thinking about, I guess, your go-forward burn?

好的。然後也許我可以再偷偷摸摸。本季度的研發消耗，當我們考慮你的前進消耗時，這是一個很好的基礎嗎？

Yes. It is.

是的。這是。

All right. Thanks, guys.

好的。多謝你們。

Our next question comes from the line of Jonathan Aschoff from ROTH Capital.

我們的下一個問題來自 ROTH Capital 的 Jonathan Aschoff。

Thanks, guys. I was wondering if you could elaborate on your key focus being gene therapy. I kind of noticed that the word biologics was removed from your company description.

多謝你們。我想知道你是否可以詳細說明你的重點是基因治療。我有點注意到生物製品這個詞已經從貴公司的描述中刪除了。

Yes. We are -- Ocugen, we're really focused on modified gene therapy platform. And with the recent results -- from the results we released, OCU400 in a way validates the platform. And so we're really excited about it. Obviously, we'll continue to move that program, as we stated before, and work with the regulatory agencies and the lineup of potentially Phase 3 clinical trials sooner than later.

是的。我們是——Ocugen，我們真正專注於改良的基因治療平台。根據最近的結果——從我們發布的結果來看，OCU400 在某種程度上驗證了平台。所以我們真的很興奮。顯然，正如我們之前所說，我們將繼續推動該計劃，並儘早與監管機構和潛在的 3 期臨床試驗陣容合作。

And that's our focus. And obviously, that two other programs in the pipeline are coming through OCU410 targeting geographic atrophy, subset of dry AMD population, and OCU410ST with Stargardt disease. INDs are going to be filed this quarter. So once again, the company is going to focus our major effort on gene therapies, because there's a lot of promise. There's so much of unmet medical need.

這就是我們的重點。顯然，管道中的另外兩個項目正在通過 OCU410 進行，目標是地理萎縮、乾性 AMD 人群的子集，以及 OCU410ST 與 Stargardt 病。 IND 將在本季度提交。因此，該公司將再次將我們的主要精力集中在基因療法上，因為它有很大的希望。有太多未滿足的醫療需求。

Okay. Can we have any other color on OCU200 via clinical hold, or is there just really nothing to say other than what's in the press release?

好的。我們能否通過臨床保留在 OCU200 上使用任何其他顏色，或者除了新聞稿中的內容之外真的沒有什麼可說的？

Yes. At this stage, nothing to say. It's related to CMC questions and the company is going to respond promptly.

是的。這個階段，沒什麼好說的。涉及CMC問題，公司會及時回复。

Okay. And can you help us -- I'm sorry, I didn't mean to cut you off there.

好的。你能幫我們嗎——對不起，我不是故意打斷你的。

No. That's it.

不，就是這樣。

Can you help us understand the drop in R&D? Is that vaccine or what is that?

你能幫助我們了解研發的下降嗎？那是疫苗還是那是什麼？

Yes. So a lot of the wind down now is obviously related to COVAXIN. And so as we begin to look at the various aspects of the company, which comes back to what Shankar just alluded to earlier, the heavy focus now will be on the development of the gene therapy platform. And so to that extent, the R&D expenses will be extraordinarily focused as well as the entire organization. And so the winding down on COVAXIN is one of the major factors that are causing the problem.

是的。所以現在很多風口顯然與 COVAXIN 有關。因此，當我們開始審視公司的各個方面時，回到 Shankar 剛才提到的內容，現在的重點將放在基因治療平台的開發上。因此，在某種程度上，研發費用以及整個組織都將受到特別關注。因此，COVAXIN 的停產是導致問題的主要因素之一。

Okay, because if you do that -- you kind of have cash well, well, well into the first quarter of '24. So is that just an overly conservative statement from lawyers?

好吧，因為如果你這樣做——你的現金很好，很好，很好，很好地進入了 24 年的第一季度。那麼這只是律師的過於保守的說法嗎？

I can't comment on the risk assessment placed by lawyers and how they draft the various things, Jonathan. But for the most part, we believe firmly that that is going to be the case. We believe that is a realistic estimate, perhaps could be conservative.

喬納森，我無法對律師進行的風險評估以及他們如何起草各種內容髮表評論。但在大多數情況下，我們堅信情況會如此。我們認為這是一個現實的估計，也許是保守的。

But I think the thing that to take -- the key takeaway here is that the organization using the examining operational efficiencies as with all companies moving forward in the biotech sector, as I'm sure you're well aware, and given the challenging capital markets, we want to be extremely focused and cost conscious in order for us to achieve our goals. And so you can imagine that the longer we can extend this cash runway, the better it is for all stakeholders involved.

但我認為要採取的事情 - 這裡的關鍵要點是組織使用檢查運營效率與所有在生物技術領域向前發展的公司一樣，我相信你很清楚，並且考慮到具有挑戰性的資本市場，我們希望非常專注和成本意識，以便我們實現我們的目標。所以你可以想像，我們延長這條現金跑道的時間越長，對所有相關利益相關者就越好。

I get it. Thank you very much.

我得到它。非常感謝。

Our next question comes from the line of Uy Ear from Mizuho.

我們的下一個問題來自 Mizuho 的 Uy Ear 系列。

Hi, guys. Thanks for taking my question. I guess the first question I have is it looks like the timeline for NeoCart has sort of shifted to at least the cGMP manufacturing facility completion to 4Q from first half. And there doesn't seem to be a date as well for when you'll start the trial. And so just wondering if you can elaborate on that and as well as where does NeoCart now sits, I guess, in terms of priority, given what you just said about challenging capital markets, et cetera? Thanks.

嗨，大家好。感謝您提出我的問題。我想我的第一個問題是 NeoCart 的時間表似乎已經從上半年轉移到至少 cGMP 製造設施完成到第四季度。而且似乎也沒有關於何時開始試用的日期。所以只是想知道你是否可以詳細說明這一點以及 NeoCart 現在的位置，我想，在優先級方面，考慮到你剛才所說的挑戰資本市場等等？謝謝。

Good morning, Uy. Just want to clarify, we always stated cGMP facility is going to be ready sometime later part of this year. So that's pretty consistent. There is no change to that. We are planning to finish the facility, construction and everything else by the end of this year.

早安，優。只是想澄清一下，我們一直說 cGMP 設施將在今年晚些時候準備就緒。所以這是非常一致的。沒有改變。我們計劃在今年年底前完成設施、建設和其他一切工作。

As far as the clinical trial is concerned, yes, we are still targeting next year. Obviously, our priority is going to be focused on modified gene therapy platform, getting the other two clinical trials to get started, as well as Phase 3 started for our first program OCU400. And so the NeoCart obviously will take second preference compared to that priority wise. And our goal is to still initiate that Phase 3 clinical trial in 2024.

就臨床試驗而言，是的，我們仍然以明年為目標。顯然，我們的首要任務是將重點放在改進的基因治療平台上，讓其他兩項臨床試驗開始，並為我們的第一個項目 OCU400 啟動第 3 階段。因此，與優先級相比，NeoCart 顯然會排在第二位。我們的目標是在 2024 年仍然啟動該 3 期臨床試驗。

Okay. Thanks. And second question I guess is when should we sort of expect to see more data from OCU400? I noticed that in the slide presentation, it only has initiation of Phase 3 in the fourth quarter. Just curious? Thanks.

好的。謝謝。我想第二個問題是我們什麼時候應該期望從 OCU400 看到更多數據？我注意到在幻燈片演示中，它只在第四季度啟動了第 3 階段。只是好奇？謝謝。

So we are going to continue to monitor as we see patients on a three-month basis. And obviously, we will have another update on data we're expecting sometime in third quarter this year.

因此，我們將在三個月的基礎上繼續監測患者。顯然，我們將在今年第三季度的某個時候再次更新我們預期的數據。

Our next question comes from the line of Robert LeBoyer from Noble Capital Markets.

我們的下一個問題來自 Noble Capital Markets 的 Robert LeBoyer。

Good morning. My only remaining question is on the OCU200 clinical hold and any timeframe on one that may be resolved?

早上好。我唯一剩下的問題是關於 OCU200 臨床暫停以及可能解決的任何時間表？

We are trying to respond to the FDA continue to work with them. And I mean sooner than later, of course. And this is -- we didn't even start the clinical trial. This is a novel biologic. It's a fusion protein. Sometimes it's typical of agency has more questions. This one is related to CMC, nothing related to toxicity or anything else, just wanted to clarify. So we will be responding promptly. And as soon as we get clearance, we will notify the market.

我們正在努力回應 FDA 繼續與他們合作。當然，我的意思是遲早會發生。這是——我們甚至沒有開始臨床試驗。這是一種新穎的生物製劑。它是一種融合蛋白。有時，典型的代理機構有更多問題。這個與 CMC 有關，與毒性或其他任何東西無關，只是想澄清一下。所以我們會及時回复。一旦我們獲得許可，我們就會通知市場。

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

我們的下一個問題來自 Wainwright 的 Swayampakula Ramakanth 系列。

Thank you. This is RK from H.C. Wainwright. Good morning, Shankar and Quan. One question to each of you. Shankar, on the OCU400, have you started conversations with EMA as you start thinking beyond the current study and trying to get into pivotal studies?

謝謝。這是 H.C. 的 RK。溫賴特。早上好，Shankar 和 Quan。給你們每個人一個問題。 Shankar，在 OCU400 上，當你開始思考當前研究之外的問題並試圖進入關鍵研究時，你是否開始與 EMA 對話？

Good morning, RK. We are planning to initiate those conversations a little later this year. Obviously, our goal is to align various regulatory agencies before we get into Phase 3.

早上好，RK。我們計劃在今年晚些時候啟動這些對話。顯然，我們的目標是在進入第 3 階段之前協調各監管機構。

Thank you for that. And then, Quan, on the BD activities side of things, have you had any conversations at all with potential collaborators outside of the government agencies for the inhalation vaccine, especially with some of the flu vaccine players?

謝謝你。然後，Quan，在 BD 活動方面，您是否與政府機構以外的潛在合作者就吸入疫苗進行過任何對話，尤其是與一些流感疫苗參與者？

Yes. So the focus on the OCU400 program is development. We are in its initial stages. We have had some initial conversations even before my time here, and kind of set the landscape. And then I think, ultimately, I am beginning to do a second reach out. But are you talking about just inhalation itself with respect to the 500 right now, or do you want to focus on the 400 --?

是的。所以OCU400程序的重點是開發。我們正處於起步階段。甚至在我來這里之前，我們就已經進行了一些初步的對話，並設置了景觀。然後我想，最終，我開始進行第二次接觸。但是你現在是在談論關於 500 的吸入本身，還是你想專注於 400 -？

Yes. Because we have always for the last one year we've been hearing that folks are looking for government funds. But I guess they have trouble with the government raising their own debt limit. So if there was probably the funding, would this move forward at all and I'm looking for corporate partners?

是的。因為在過去的一年裡，我們一直聽說人們正在尋找政府資金。但我猜他們在政府提高自己的債務限額方面遇到了麻煩。因此，如果可能有資金，這是否會向前推進並且我正在尋找公司合作夥伴？

Yes. Absolutely. And so the idea behind all this as part of our very focused play here is that we do indeed have the inhalation technology. However, as most of this centralizes around public health crises, we're looking for that government support. I'm sure you're well aware, RK, that with all these types of programs, government support is critical, not only from a funding perspective, but also from, in a way -- the way that the politics work, for lack of a better word, supports these very programs.

是的。絕對地。因此，作為我們非常專注的遊戲的一部分，所有這一切背後的想法是，我們確實擁有吸入技術。然而，由於其中大部分集中在公共衛生危機周圍，我們正在尋求政府的支持。我相信你很清楚，RK，對於所有這些類型的項目，政府支持是至關重要的，不僅從資金的角度來看，而且在某種程度上——政治運作的方式，因為缺乏換句話說，支持這些程序。

And so without that, these programs can get extraordinarily expensive, and we don't intend to spend a lot more than we are. Our idea is to have enough development data in order to be able to apply for this funding, and to be able to advance it further. Without that support, it would be very challenging for small companies such as ourselves to do that.

因此，如果沒有它，這些項目可能會變得非常昂貴，我們不打算比現在多花很多錢。我們的想法是擁有足夠的開發數據，以便能夠申請這筆資金，並能夠進一步推進它。如果沒有這種支持，像我們這樣的小公司很難做到這一點。

RK, once we get there, if the opportunities exist for corporate partnerships, we will look into it.

RK，一旦我們到達那裡，如果存在企業合作夥伴關係的機會，我們將進行調查。

Terrific. And also, as Jonathan pointed out, since you don't have biologics on your corporate mandate, does that mean OCU200 is also subject for BD activities?

了不起。而且，正如 Jonathan 指出的那樣，由於您的公司授權中沒有生物製劑，這是否意味著 OCU200 也受 BD 活動的約束？

I think OCU200 typically with the Phase 1, we do anticipate signal, even though it's a dose escalation study. And if we do get a positive signal on efficacy, obviously, we will definitely evaluate that next year.

我認為 OCU200 通常在第一階段，我們確實預期信號，即使它是劑量遞增研究。如果我們確實收到了關於療效的積極信號，顯然，我們肯定會在明年進行評估。

To add on to that, RK, obviously, a lot of BD activity programs, as a lot of companies look for much derisk programs, and so these are relatively early. And as we advance them, we will have a bit more clarity. It's very challenging to go out and have conversations with the various players in the market without pivotal data for the most part. But that does not preclude us, for example, from always establishing these relationships early and exploring the level of interest going forward.

除此之外，RK 顯然有很多 BD 活動計劃，因為很多公司都在尋找很多 derisk 計劃，所以這些都相對較早。隨著我們推進它們，我們將更加清晰。大多數情況下，在沒有關鍵數據的情況下走出去與市場上的各種參與者進行對話是非常具有挑戰性的。但這並不妨礙我們，例如，始終儘早建立這些關係並探索未來的興趣水平。

Fantastic. Thank you, gentlemen. Talk to you soon.

極好的。謝謝你們，先生們。以後再聊。

Our final question comes from the line of Daniil Gataulin from Chardan. Please proceed.

我們的最後一個問題來自 Chardan 的 Daniil Gataulin。請繼續。

Hi. Good morning, guys. Thank you for taking the questions. So I have a couple on OCU400. Just wanted to ask if your next readout in the third quarter is going to include any patient level data? And in terms of, I guess, the timing for Phase 3, it looks like you're planning on initiating that at the end of this year. But your one-year data from the Phase 1/2 is not going to come until at least the first quarter of 2024, just wanted to ask about your strategy there? And I have a quick follow up too.

你好。早上好傢伙。感謝您提出問題。所以我在 OCU400 上有一對。只是想問一下您在第三季度的下一次讀數是否會包括任何患者水平的數據？我猜，就第 3 階段的時間安排而言，您似乎計劃在今年年底啟動該階段。但是您的第 1/2 階段的一年數據至少要到 2024 年第一季度才會出現，只是想問一下您在那裡的策略？我也有一個快速跟進。

Yes. We're planning to release data in third quarter and update obviously. And at this stage, we haven't decided and we'll release patient level data, because we'll also be working with regulatory agencies and we have to weigh those factors in. And the second thing is the 12-month duration for Phase 1/2, remember there's an expansion phase after that durability. You're right. It will be completed first quarter next year.

是的。我們計劃在第三季度發布數據並進行明顯更新。在這個階段，我們還沒有決定，我們將發布患者水平的數據，因為我們還將與監管機構合作，我們必須權衡這些因素。第二件事是 Phase 的 12 個月持續時間1/2，請記住在耐久性之後有一個擴展階段。你說得對。將於明年第一季度完成。

However, what we're looking for is at least for certain populations in this clinical trial crossing 12 months and collecting adequate safety. And the second thing we'll be looking for to discuss with regulatory agencies is tuning in and finalizing our primary efficacy endpoint. And as we stated in our results, if we continue to look at that functional endpoint, that's in an approved product already, it makes it easier with regulatory agencies.

然而，我們正在尋找的是至少對於這個跨越 12 個月的臨床試驗中的某些人群並收集足夠的安全性。我們希望與監管機構討論的第二件事是調整併最終確定我們的主要療效終點。正如我們在結果中所述，如果我們繼續關注該功能端點，它已經在批准的產品中，這會讓監管機構更容易。

And we confirm that if that's what the endpoint as an efficacy endpoint going forward, it makes it easier. So we believe having adequate safety some level at least in certain patient populations crossing 12 months, which we'll have this year, and also confirming with multiple mutations focusing on one efficacy potentially primary endpoint will help us because we are going after gene agnostic RP and LCA indication, having one efficacy endpoint will make it easier for Phase 3 design.

我們確認，如果這是作為未來療效終點的終點，它會讓事情變得更容易。因此，我們相信至少在某些跨越 12 個月的患者群體中具有一定程度的足夠安全性，我們今年將擁有這種安全性，並且還確認多個突變專注於一種潛在的主要終點療效將對我們有所幫助，因為我們正在追求與基因無關的 RP和 LCA 適應症，擁有一個療效終點將使 Phase 3 設計更容易。

Got it. That makes sense. Thank you. And another quick follow up for pediatric Phase 1/2 for OCU400. How many pediatric patients you plan on enrolling and at which doses?

知道了。這就說得通了。謝謝。以及 OCU400 兒科 1/2 期的另一個快速跟進。您計劃招募多少兒科患者，劑量是多少？

We're planning to enroll three to get some baseline safety data on these patients so that at least in Phase 3, we can include pediatric population.

我們計劃招募三名患者以獲得這些患者的一些基線安全數據，以便至少在第 3 階段，我們可以包括兒科人群。

I understood. All right. Thank you very much.

我明白了。好的。非常感謝。

Thank you.

謝謝。

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

謝謝。問答部分到此結束。我現在將電話轉回給董事長兼首席執行官 Shankar Musunuri 博士。

Thank you, Operator. In closing, I would like to thank the entire team for their hard work and resilient efforts to advance our patient-centric mission. To our shareholders and partners, thank you for your ongoing trust and support. We look forward to sharing more details of our progress in the coming quarters.

謝謝你，運營商。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辛勤工作和堅韌不拔的努力。感謝我們的股東和合作夥伴一直以來的信任和支持。我們期待在未來幾個季度分享我們進展的更多細節。

Thanks again, everyone. Have a great weekend.

再次感謝大家。週末愉快。