Ocugen Inc (OCGN) 2023 Q3 法說會逐字稿

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

早上好，歡迎閱讀 Ocugen 2023 年第三季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.

我現在將電話轉給 Ocugen 的企業傳播主管 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. Michael Breininger, our Corporate Controller, is also on the call to provide a financial update for the quarter ended September 30, 2023. Dr. Arun Upadhyay, Chief Scientific Officer, Head of Research, Development, and Medical will be available to answer questions following the presentation.

謝謝接線員，大家早安。與我一起參加今天的電話會議和網路廣播的是 Ocugen 董事長、執行長兼聯合創始人 Shankar Musunuri 博士，他將提供業務更新以及我們的臨床和營運進度概述。我們的公司財務總監 Michael Breininger 也將在電話中提供截至 2023 年 9 月 30 日的季度的財務更新。首席科學官、研究、開發和醫療主管 Arun Upadhyay 博士將回答以下問題簡報。

This morning, we issued a press release detailing associated business and operational highlights for the third 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 an accompanying slide presentation will be available on the Investors section of the Ocugen website for approximately 45 days.

今天上午，我們發布了一份新聞稿，詳細介紹了 2023 年第三季的相關業務和營運亮點。我們鼓勵聽眾查看新聞稿，該新聞稿可在我們的網站 ocugen.com 上取得。本次電話會議正在錄音，並將在 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 predict, believe, potential, proposed, 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. The statements include, but are not limited to, statements regarding our clinical development activities and related anticipated timelines.

本簡報包含 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，或表達未來事件不確定性的其他字詞或結果來識別這些前瞻性陳述。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。

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

此類陳述受到許多重要因素、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。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，包括我們向SEC 提交的季度和年度報告中標題為「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 as of 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.

我們在本簡報中所做的任何前瞻性陳述僅代表截至簡報之日的情況。除法律要求外，我們不承擔在本簡報發布後因新資訊、未來事件或其他原因而更新本簡報中包含的前瞻性聲明的義務。

Finally, Ocugen's quarterly report on Form 10-Q covering the third quarter of 2023 has been filed. I will now turn the call to Dr. Musunuri.

最後，Ocugen 涵蓋 2023 年第三季的 10-Q 表格季度報告已提交。我現在將電話轉給 Musunuri 博士。

Thank you, Tiffany, and thank you all for joining us today. As emphasized in the press release we put out this morning, we continue to make significant headway with the development of our pipeline assets, particularly with our first-in-class ophthalmic gene therapy programs. And I'm proud of the momentum we have achieved to date.

謝謝蒂芙尼，也謝謝大家今天加入我們。正如我們今天早上發布的新聞稿中所強調的那樣，我們在管道資產的開發方面繼續取得重大進展，特別是在我們一流的眼科基因治療計畫方面。我對我們迄今為止所取得的勢頭感到自豪。

Following additional positive and encouraging clinical study results from our novel modifier gene therapy-based Phase 1/2 OCU400 study in September 2023, we believe we have strong clinical evidence to initiate our Phase 3 clinical trial in retinitis pigmentosa, RP, patients in early 2024 based on FDA concurrence. Utilizing a dual-track strategy, we also intend to expand our OCU400 Phase 3 trial in the second half of 2024 to include patients with Leber congenital amaurosis, LCA, contingent upon favorable results from the Phase 1/2 study.

2023 年9 月，我們基於新型修飾基因療法的1/2 期OCU400 研究獲得了更多積極且令人鼓舞的臨床研究結果，我們相信我們有強有力的臨床證據，可以在2024 年初啟動色素性視網膜炎RP 患者的3 期臨床試驗是基於 FDA 的同意。利用雙軌策略，我們還打算在 2024 年下半年擴大 OCU400 3 期試驗，以納入 Leber 先天性黑蒙 (LCA) 患者，具體取決於 1/2 期研究的良好結果。

With enrollment begun for our OCU410 and OCU410ST programs, we are diligently working to dose patients this quarter. We anticipate clinical updates from our OCU400, OCU410, and OCU410ST studies in the later part of 2024.

隨著我們的 OCU410 和 OCU410ST 計畫的註冊開始，我們本季正在努力為患者提供劑量。我們預計 OCU400、OCU410 和 OCU410ST 研究的臨床更新將於 2024 年下半年發布。

Our clinical and regulatory teams continue to work on responses to the FDA regarding our IND submission for OCU200, the company's ophthalmic biologic product candidate, and we plan to initiate a Phase 1 clinical study in the first half of 2024 contingent on the lift of the FDA hold and adequate availability of funding. For our regenerative cell therapy candidate for knee cartilage repair, NeoCart, we are on track to complete construction of our state-of-the-art cGMP facility at the end of this year and are planning to complete qualifications of the facility in the first half of 2024. We plan to initiate the Phase 3 clinical trial in the second half of next year.

我們的臨床和監管團隊繼續致力於對 FDA 就我們的眼科生物產品候選產品 OCU200 的 IND 提交做出回應，我們計劃在 2024 年上半年啟動一項 1 期臨床研究，具體取決於 FDA 的批准持有並有足夠的資金。對於我們用於膝關節軟骨修復的再生細胞療法候選藥物 NeoCart，我們預計在今年年底完成最先進的 cGMP 設施的建設，併計劃在上半年完成該設施的資格認證2024年，我們計劃明年下半年啟動3期臨床試驗。

Last month, we were delighted to be selected for inclusion in a Phase 1 clinical trial funded by National Institute of Allergy and Infectious Disease to investigate the administration of our COVID-19 mucosal vaccine candidate, OCU500. Safety and immunogenicity of OCU500 will be evaluated using inhaled and intranasal routes of delivery during the Phase 1 clinical trial in the first half of 2024. All these catalysts considered, we can safely reiterate that 2024 will be transformative for Ocugen.

上個月，我們很高興被選中參加由國家過敏和傳染病研究所資助的一項 1 期臨床試驗，以研究我們的候選 COVID-19 黏膜疫苗 OCU500 的給藥情況。 OCU500 的安全性和免疫原性將在2024 年上半年的一期臨床試驗期間使用吸入和鼻內給藥途徑進行評估。考慮到所有這些催化劑，我們可以有把握地重申，2024 年將是Ocugen 的變革年。

Our mission to introduce critically needed therapies into the market is imminent with the planned initiation of Phase 3 trials and comprising gene and cell therapies in the near term. Our R&D team's dedication and hard work has yielded significant progress and compelling results for our first-in-class modifier gene therapy, OCU400 program, for RP and LCA patients.

隨著計劃在短期內啟動包括基因和細胞療法在內的第三階段試驗，我們即將將急需的療法引入市場的使命迫在眉睫。我們的研發團隊的奉獻和辛勤工作為我們針對 RP 和 LCA 患者的一流修飾基因療法 OCU400 項目取得了重大進展和引人注目的結果。

Throughout the Phase 1/2 trial, our primary objective has been to observe safety and tolerability of the subretinal administration of OCU400 in subjects as well as immune response and systemic distribution. For preliminary signs of efficacy, we focused on a few visual function and functional vision indicators, namely best corrected visual acuity, BCVA; low luminescence visual acuity, LLVA; and multi-luminescence mobility test, MLMT. More details on our trial design can be found on clinicaltrials.gov with the identifier code listed at the bottom of this slide.

在整個 1/2 期試驗中，我們的主要目標是觀察受試者視網膜下給藥 OCU400 的安全性和耐受性以及免疫反應和全身分佈。對於初步的療效跡象，我們重點關注了一些視覺功能和功能性視力指標，即最佳矯正視力（BCVA）；低發光視力，LLVA；和多重發光遷移率測試，MLMT。有關我們試驗設計的更多詳細信息，請訪問 ClinicalTrials.gov，標識符代碼列於本幻燈片底部。

Let me provide a situational analysis around the unmet need and underserved market for RP and LCA patients. An estimated 1.6 million people globally are affected by RP and LCA combined. In the US alone, we are looking at about 125,000 patients total.

讓我對 RP 和 LCA 患者未滿足的需求和服務不足的市場進行情況分析。據估計，全球有 160 萬人受到 RP 和 LCA 的影響。光是在美國，我們就總共觀察了約 125,000 名患者。

RP and LCA are classified as inherited retinal diseases from a group of heterogeneous disorders that affect the retina. These diseases often lead to sight loss and, ultimately, blindness. That said, the earlier homeostasis can be stabilized in patients with either of these diseases, the better.

RP 和 LCA 被歸類為遺傳性視網膜疾病，屬於一群影響視網膜的異質性疾病。這些疾病常常導致視力喪失，最終導致失明。也就是說，患有這些疾病的患者體內平衡越早穩定越好。

Through relevant medical meetings and continued engagement with advocacy groups, we aim to create awareness for the prevalence of retinitis pigmentosa and Leber congenital amaurosis and potential emerging therapies like our novel platform. Our ultimate objective is to provide treatment to people suffering from vision impairment and blindness caused by RP and LCA, for whom currently no therapeutic options exist.

透過相關的醫學會議以及與倡導團體的持續接觸，我們的目標是提高人們對色素性視網膜炎和萊伯先天性黑蒙的盛行率以及像我們的新平台這樣的潛在新興療法的認識。我們的最終目標是為 RP 和 LCA 引起的視力障礙和失明患者提供治療，目前尚無治療選擇。

I listed our three exploratory endpoints for visualization stabilization and improvement observed in patients treated with OCU400 on slide 4, BCVA, LLVA, and MLMT. In the 12 cumulative subjects that have undergone a minimum of six months follow-up post OCU400 dosing, we observed the following metrics.

我在投影片 4、BCVA、LLVA 和 MLMT 上列出了在接受 OCU400 治療的患者中觀察到的可視化穩定性和改善的三個探索性終點。在 OCU400 給藥後經過至少六個月追蹤的 12 名累積受試者中，我們觀察到以下指標。

This Venn diagram demonstrates that 8 out of 12 subjects showed either stabilization, means no change from baseline, plus/minus four letters change for BCVA and LLVA, and zero lux level change for MLMT; or improvement in all three parameters of BCVA and LLVA, which means five or more letters, and MLMT greater than or equivalent to one lux level, demonstrating initial efficacy of OCU400. Non-responders are listed outside the circles for each group.

這張維恩圖表明，12 名受試者中有 8 名表現出穩定，意味著與基線相比沒有變化，BCVA 和 LLVA 正/負四個字母變化，MLMT 勒克斯水平變化為零；或 BCVA 和 LLVA 所有三個參數的改善，這意味著五個或更多字母，並且 MLMT 大於或等於 1 勒克斯水平，證明了 OCU400 的初步功效。無反應者列在每組的圓圈外。

To recap, what we know from our findings to date is that OCU400 has a favorable safety and tolerability profile in patients. Positive trends are observed in all set visualization stability and improvement factors which details that. 83% of subjects demonstrated stabilization or improvement in the treated eye either on BCVA, LLVA, or MLMT scores from baseline. 75% of subjects showed stabilization or improvement in treated eyes and MLMT scores from baseline.

回顧一下，我們從迄今為止的研究結果得知，OCU400 在患者中具有良好的安全性和耐受性。在所有設定的可視化穩定性和改進因素中都觀察到了積極的趨勢，詳細說明了這一點。 83% 的受試者表現出治療眼的 BCVA、LLVA 或 MLMT 評分較基線穩定或改善。 75% 的受試者接受治療的眼睛和 MLMT 評分較基線表現出穩定或改善。

86% of RHO mutation subjects experienced either stabilization or improvement in MLMT scores from baseline. Among which, 29% demonstrated 3 lux luminescence level improvement, demonstrating the gene-agnostic mechanism of action of OCU400. The RHO mutation affects more than 10,000 people in the US alone. Based on this data, we are highly enthusiastic about the future of OCU400 and the vision-saving potential it may provide to RP and LCA patients.

86% 的 RHO 突變受試者的 MLMT 評分較基線穩定或有所改善。其中，29% 的發光水平提高了 3 勒克斯，證明了 OCU400 的基因不可知的作用機制。光是在美國，RHO 突變就影響了 1 萬多人。基於這些數據，我們對 OCU400 的未來及其為 RP 和 LCA 患者提供的挽救視力的潛力充滿熱情。

The execution of critical elements of OCU400 Phase 1/2 trial, including the completion of dosing of RP and LCA patients, sets the stage for us to execute a Phase 3 clinical trial for both indications in 2024 upon FDA concurrence. OCU410, our modified gene therapy candidate for dry age-related macular degeneration, AMD, is a potential one-time curative therapy with a single sub-retinal injection that targets multiple pathways causing dry AMD, including lipid metabolism, inflammation, oxidative stress, and complement activation. Unlike other currently marketed products targeting a single pathway, complement activation.

OCU400 1/2 期試驗關鍵要素的執行，包括完成 RP 和 LCA 患者的給藥，為我們在 FDA 同意的情況下於 2024 年針對這兩種適應症執行 3 期臨床試驗奠定了基礎。 OCU410 是我們針對乾性老年黃斑部病變(AMD) 的改良基因療法候選藥物，是一種潛在的一次性治療療法，只需一次視網膜下注射，針對導致乾性AMD 的多種途徑，包括脂質代謝、發炎、氧化壓力和補體活化。與目前上市的其他針對單一途徑補體活化的產品不同。

We are currently enrolling patients in the Phase 1/2 (inaudible) RHO study to assess the safety and efficacy of OCU410 for geographic atrophy, secondary to dry AMD. Geographic atrophy, an advanced form of dry age-related macular degeneration, affects approximately 1 million people in the United States alone. From a competitive standpoint, we believe OCU410 is differentiated among other therapies available and in development for geographic atrophy and dry AMD by frequency of administration, one-time versus multiple injections per year; reduced side effects from structural impact; strong safety profile; its mechanism for restoring homeostasis; and preserving the conditions that promote self-help.

我們目前正在招募患者參加 1/2 期（聽不清楚）RHO 研究，以評估 OCU410 對繼發於乾性 AMD 的地圖樣萎縮的安全性和有效性。地理萎縮是乾性老年黃斑部病變的一種高級形式，僅在美國就有大約 100 萬人受到影響。從競爭的角度來看，我們認為 OCU410 在治療地理萎縮和乾性 AMD 的現有和正在開發的其​​他療法中的差異化在於給藥頻率、每年一次注射與多次注射；減少結構影響所帶來的副作用；強大的安全性；其恢復體內平衡的機制；並保留促進自助的條件。

The slide demonstrates how OCU410 utilizes an AAV delivery platform for the retinal delivery of RORA gene. In preclinical studies, OCU410 demonstrated efficacy in regulating multiple pathways involved with the disease, including lipid metabolism; reducing Drusen formation, regulation of inflammation, suppressing inflammation, oxidative stress, improving cell survival, membrane attack complex complement, restoring anti-complement protein.

這張投影片示範了 OCU410 如何利用 AAV 遞送平台進行 RORA 基因的視網膜遞送。在臨床前研究中，OCU410 證明能夠有效調節與疾病相關的多種途徑，包括脂質代謝；減少玻璃疣形成，調節炎症，抑制炎症，氧化應激，提高細胞存活率，膜攻擊補體複合物，恢復抗補體蛋白。

On this slide, we have captured our proposed program design for OCU410. In 63 adult subjects, 50 or older, with geographic atrophy secondary to dry AMD, we will observe the treatment effect of a single, unilateral subretinal injection OCU410, starting with safety and efficacy in patients. We're employing a three-plus-three design with a low, medium, and high dose in addition to a dose expansion exercise using a one-to-one-to-one design, randomizing subjects to either two treatment groups per dose levels or one control group. Using a similar approach, our orphan-drug designated OCU410ST modified gene therapy platform for Stargardt disease leverages nuclear hormone receptors to modulate cell activity and utilize this, an AAV delivery platform, for retinal delivery of the RAR-related Orphan Receptor A.

在這張投影片上，我們展示了我們為 OCU410 提出的程式設計。在63 名50 歲或以上患有乾性AMD 繼發性地圖狀萎縮的成年受試者中，我們將觀察單次、單側視網膜下注射OCU410 的治療效果，首先從患者的安全性和有效性開始。除了使用一對一設計的劑量擴展練習外，我們還採用低、中、高劑量的三加三設計，將受試者隨機分配到每個劑量等級的兩個治療組或一個對照組。使用類似的方法，我們的孤兒藥 OCU410ST 改良基因治療平台用於 Stargardt 病，利用核激素受體調節細胞活性，並利用此 AAV 遞送平台，將 RAR 相關孤兒受體 A 遞送至視網膜。

OCU410 delivery in preclinical studies for Stargardt disease demonstrated a structural and functional improvement. In the OCU410ST Phase 1/2 trial, we intend to treat and investigate 42 subjects, 30 of which are adults and 12 are children with Stargardt disease. The adult inclusion criteria looked at adult patients between 18 to 65 and pediatrics between 6 to 17. We're employing a three-plus-three design with a low, medium, and high dose cohort in addition to your dose expansion exercise using a one-to-one-to-one design, randomizing subjects to either two treatment groups per dose levels or one control group. Our team's diligent efforts resulted in NIAID selecting OCU400 for inclusion in a Project NextGen Phase 1 clinical trial of our mucosal vaccine candidates for COVID-19 likely to be initiated in the first half of 2024.

Stargardt 病臨床前研究中的 OCU410 遞送顯示出結構和功能的改善。在 OCU410ST 1/2 期試驗中，我們打算治療和研究 42 名受試者，其中 30 名是患有 Stargardt 病的成人，12 名是兒童。成人納入標準著眼於 18 至 65 歲之間的成年患者和 6 至 17 歲之間的兒科患者。除了使用一劑量擴展練習外，我們還採用了低、中、高劑量隊列的三加三設計。一對一設計，將受試者隨機分為每個劑量等級的兩個治療組或一個對照組。我們團隊的不懈努力導致 NIAID 選擇 OCU400 納入我們的 COVID-19 黏膜候選疫苗的 Project NextGen 一期臨床試驗，該試驗可能於 2024 年上半年啟動。

From our own development efforts, we observed vaccine-induced high neutralizing and effector responses during preclinical studies on OCU500. We believe the inhaled route of administration has the potential to be the holy grail for broad and durable protection from severe diseases and can suppress the transmission rate.

根據我們自己的發展工作，我們在 OCU500 的臨床前研究中觀察到疫苗誘發的高中和反應和效應反應。我們相信，吸入給藥途徑有可能成為廣泛且持久地預防嚴重疾病的聖杯，並且可以抑制傳播率。

As a refresher, Project NextGen, a multi-government agency initiative overseen by NIAID, is a $5 billion multi-government agency initiative to develop the next generation of vaccines and therapeutics to combat the spread of COVID-19. NIAID will execute the clinical trial for OCU500. Upon completion of the trial, Ocugen will possess full rights of reference to the findings. This initiative is a testament to the fact that COVID-19 is still rampant with emergence of new variants and needs more durable vaccines to treat them.

回顧一下，NextGen 計畫是一個由 NIAID 監督的多政府機構計劃，是一項耗資 50 億美元的多政府機構計劃，旨在開發下一代疫苗和治療方法，以對抗 COVID-19 的傳播。 NIAID 將執行 OCU500 的臨床試驗。試驗完成後，Ocugen 將擁有對調查結果的完整參考權。這項措施證明了這樣一個事實：隨著新變種的出現，COVID-19 仍然猖獗，需要更持久的疫苗來治療它們。

In a recent Harris poll, we favorably found that 66% of Americans would prefer to have more vaccine options. The poll also found that 52% of Americans would be more open to getting an intranasal, or inhaled, versus injectable COVID-19 vaccine. In line with NIAID's mission to support innovation in public health, we look forward to potentially expanding the platform to the flu and other respiratory viral diseases and infections.

在最近的哈里斯民意調查中，我們有利地發現 66% 的美國人希望有更多的疫苗選擇。民調還發現，與注射式 COVID-19 疫苗相比，52% 的美國人更願意接種鼻內或吸入疫苗。根據 NIAID 支持公共衛生創新的使命，我們期待將該平台擴展到流感和其他呼吸道病毒疾病和感染。

I would like to bring our pipeline updates to a close by providing a brief update on NeoCart. Ocugen's autologous regenerative cell therapy which uses patient's own cartilage cells is on track to begin its Phase 3 clinical trial in the second half of 2024. A cGMP facility for manufacturing NeoCart is expected to be completed at the end of 2023, and qualification is expected in the first half of 2024.

我想透過提供 NeoCart 的簡短更新來結束我們的管道更新。 Ocugen 使用患者自身軟骨細胞的自體再生細胞療法預計將於 2024 年下半年開始其 3 期臨床試驗。用於生產 NeoCart 的 cGMP 設施預計將於 2023 年底完工，並預計於 2023 年獲得資格認證。半年。

OCU200 is a ophthalmic biological product candidate in preclinical development for treating severely sight-threatening diseases, like diabetic macular edema, diabetic retinopathy, and wet age-related macular degeneration. We are working on responses and continue to interact with the FDA regarding the clinical hold on our OCU200 IND submission and expect to initiate a Phase 1 clinical study in the first half of 2024.

OCU200是一種處於臨床前開發階段的眼科生物產品候選產品，用於治療嚴重威脅視力的疾病，如糖尿病性黃斑水腫、糖尿病性視網膜病變和濕性老年黃斑部病變。我們正在研究應對措施，並繼續與 FDA 就 OCU200 IND 提交的臨床擱置事宜進行互動，並預計在 2024 年上半年啟動 1 期臨床研究。

With that, I will now turn the call over to our Corporate Controller, Michael Breininger, to provide an update on our financial results for the third quarter ended September 30, 2023. Michael.

現在，我將把電話轉給我們的公司財務總監 Michael Breininger，以提供截至 2023 年 9 月 30 日的第三季財務業績的最新資訊。Michael。

Thank you, Shankar. Our research and development expense for the quarter ended September 30, 2023, were $6.3 million, compared to $15.6 million for the third quarter of 2022. General and administrative expenses for the quarter ended September 30, 2023, were $9.1 million, compared to $7.5 million during the same period in 2022.

謝謝你，香卡。截至2023 年9 月30 日的季度的研發費用為630 萬美元，而2022 年第三季為1,560 萬美元。截至2023 年9 月30 日的季度的一般和管理費用為910 萬美元，而2022 年第三季的一般和管理費用為910 萬美元，而2022 年第三季為750 萬美元。2022 年同期。

Net loss was approximately $14.2 million, or $0.06 net loss per share, for the quarter ended September 30, 2023, compared to a net loss of approximately $21.9 million, or $0.10 net loss per share, for the third quarter of 2022. Net loss was approximately $53.6 million, or $0.22 net loss per share, for the nine months ended September 30, 2023, compared to a net loss of approximately $59.4 million or $0.28 net loss per share, for the nine months ended September 30, 2022.

截至 2023 年 9 月 30 日的季度淨虧損約為 1,420 萬美元，即每股淨虧損 0.06 美元，而 2022 年第三季的淨虧損約為 2,190 萬美元，即每股淨虧損 0.10 美元。截至20233年9月30日的九個月，淨虧損約為5,360萬美元，即每股淨虧損0.22美元，而截至2022年9月30日的九個月，淨虧損約為5,940萬美元，即每股淨虧損0.28美元。

Our cash, cash equivalents, and investments totaled $53.5 million as of September 30, 2023, compared to $90.9 million as of December 31, 2022. As always, we are constantly exploring strategic and shareholder-friendly 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.

截至2023 年9 月30 日，我們的現金、現金等價物和投資總額為5,350 萬美元，而截至2022 年12 月31 日為9,090 萬美元。一如既往，我們不斷探索策略和股東友好的機會來增加我們的營運資本，並且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.

我的本季更新到此結束。蒂芬妮，回到你身邊。

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

謝謝你，麥克。我們現在開始提問。操作員？

(Operator instructions) Arthur He, H.C. Wainwright.

（操作說明） Arthur He, H.C.溫賴特。

Hey. Good morning, Shankar and team. Here is RK -- Arthur for RK. Congrats on the progress. So I just had a couple questions on 400. So when could we expect the complete data set from the Phase 2 study, especially for the LCA patients? So if you can give some color on that, that would be really appreciated.

嘿。早安，香卡和團隊。這裡是 RK——Arthur 代表 RK。祝賀取得的進展。所以我對 400 有幾個問題。那麼我們什麼時候可以期待 2 期研究的完整資料集，特別是針對 LCA 患者的資料集？因此，如果您能對此提供一些顏色，我們將不勝感激。

Yeah. The LCA, we just dosed -- so I think it'll take until later part of next year.

是的。 LCA，我們剛剛劑量——所以我認為這需要到明年下半年。

How about the other patient left in the RP group?

RP 組中剩下的另一位患者怎麼樣？

Yeah. The RP patients will get it in the first-half. However, we believe we have adequate information and we're working with regulatory agencies, FDA and DMA, for Phase 3.

是的。 RP 患者將在上半年獲得它。然而，我們相信我們有足夠的信息，我們正在與監管機構 FDA 和 DMA 合作進行第三階段。

Okay. So speak of the Phase 3 study. So from what you said in the press release and the core, is the Phase 3 will be a single Phase 3 packed together, both RP patients as well LCA, or it could be two separate Phase 3 study?

好的。說到第三階段研究。那麼，從您在新聞稿和核心內容中所說的來看，第 3 期是否將是一個單一的第 3 期研究，包括 RP 患者以及 LCA，或者它可能是兩個單獨的第 3 期研究？

We'll start with RP because that's the data we have right now. And then we're going to add LCA arm little later in the clinical trial.

我們將從 RP 開始，因為這是我們現在擁有的數據。然後我們將在稍後的臨床試驗中添加 LCA 臂。

Okay. So that would be in the single Phase 3 or --

好的。所以這將是在單一的第三階段或——

Yeah, single Phase 3.

是的，單階段3。

Okay, I see. And the last question on 400 is -- one is for -- have you requested a meeting with FDA? And based on your own proposal, which endpoint could be your primary endpoint? If you can --

好吧，我明白了。 400 的最後一個問題是——其中一個是——您是否要求與 FDA 會面？根據您自己的建議，哪個端點可能是您的主要端點？如果可以的話 -

I'll let Dr. Upadhyay answer that, our CSO. Go ahead, Arun.

我會讓我們的 CSO Upadhyay 博士來回答這個問題。繼續吧，阿倫。

Thanks, Shankar. So we are considering a combinatorial approach, and we have proposed that to the FDA. And we are going to have a meeting with them this quarter. And accordingly, once we have alignment with FDA, then we will update the market.

謝謝，香卡。因此，我們正在考慮採取組合方法，並已向 FDA 提出建議。我們將在本季與他們舉行會議。因此，一旦我們與 FDA 達成一致，我們就會更新市場。

All right. Sounds great. Thanks for taking my question.

好的。聽起來很棒。感謝您提出我的問題。

Thank you.

謝謝。

Robert LeBoyer, Noble Capital Markets.

羅伯特·勒博耶，Noble Capital Markets。

Good morning, and thanks for taking my question. I just had a follow-up on the Phase 3 for OCU400. And I'm wondering if you have any information or could disclose how many patients you expect to be in the trial or what the length of follow-up is going to be for the patients.

早安，感謝您提出我的問題。我剛剛對 OCU400 的第三階段進行了跟進。我想知道您是否有任何資訊或可以透露您預計有多少患者參加試驗，或者患者的追蹤時間是多長。

Good morning, Robert. I'll let Arun address that.

早上好，羅伯特。我會讓阿倫解決這個問題。

Yeah. So we are planning in the range of close to 100 subjects in Phase 3 in one-is-to-one randomization and one year follow-up.

是的。因此，我們計劃在第 3 階段對近 100 名受試者進行一對一隨機化和一年追蹤。

Okay. Thank you very much.

好的。非常感謝。

Just to confirm, it is 100, okay?

確認一下，是 100，好嗎？

It's 100 patients, Robert.

羅伯特，有 100 名患者。

Yes.

是的。

(Operator instructions) There are no further questions at this time. I will now turn the call over to Chairman and CEO, Dr. Shankar Musunuri.

（操作員說明） 目前沒有其他問題。我現在將把電話轉給董事長兼首席執行官尚卡爾·穆蘇努里 (Shankar Musunuri) 博士。

In closing, I'd like to reiterate our unwavering commitment to groundbreaking science and clinical innovations in order to create effective and positively impactful therapies that are accessible to patients globally. As we continue to execute stated plans, we remain focused on delivering long-term value for our shareholders who have supported us and for prospective ones seeking to be part of our story. Thank you, and have a great day.

最後，我想重申我們對突破性科學和臨床創新的堅定承諾，以便創造出可供全球患者使用的有效且具有積極影響力的療法。在我們繼續執行既定計劃的同時，我們仍然專注於為支持我們的股東以及尋求參與我們故事的潛在股東提供長期價值。謝謝您，祝您有美好的一天。

Thanks, everyone.

感謝大家。

This concludes this conference call. You may now disconnect.

本次電話會議到此結束。您現在可以斷開連線。