Ocugen (OCGN) 2026财年第三季度业绩电话会议：OCU400数据公布定于2027年第一季度

核心要点

• Ocugen（NASDAQ：OCGN）在2026财年第三季度末拥有8460万美元的现金、现金等价物、受限现金和短期投资。管理层预计，这些资金将为其运营提供支持直至2028年，并资助其三个后期项目中的两个完成预计的生物制品许可申请（BLA）提交。
• 其全部三个修饰基因疗法项目目前均处于III期临床阶段。管理层仍按计划将于2028年前提交三项生物制品许可申请。
• 用于治疗视网膜色素变性的 OCU400 试验（III期 liMeliGhT 临床研究，共招募140名患者）已完成入组。顶线数据预计将于2027年第一季度公布，随后计划提交 BLA。
• 用于治疗斯塔加特病（Stargardt disease）的 OCU410ST 顶线数据预计将于2027年第二季度公布。独立数据监察委员会（DMC）在进行中期审查后，建议继续按原方案开展研究，无需修改。
• 用于治疗地图状萎缩的 OCU410 已进入III期临床。这项包含237名患者的 ArMaDa3 试验已开始给药，目标是在2027年完成招募，并计划于2028年提交 BLA。
• 第三季度运营费用为1970万美元，上年同期为1940万美元。每股净亏损从0.07美元收窄至0.01美元。

关键财务数据

截至2026年9月30日，现金、现金等价物、受限现金和短期投资总计为8460万美元。Ocugen 拥有3.393亿股已发行普通股。

业务与运营业绩

用于视网膜色素变性的 OCU400

Ocugen 已完成 III 期 liMeliGhT 临床试验的140名患者入组。该研究将92名患者随机分配至治疗组，48名分配至未治疗对照组。工艺性能确认（PPQ）批次已完成，可支持计划中的 BLA 提交及潜在的商业化供应。

管理层引用了一项针对符合 III 期入组标准的7位 I/II 期受试者的事后分析，预测在第52周时，71.4%的给药眼将达到 III 期应答者阈值（即在 LDNA 量表上至少改善两个等级）。

在三年时，8名可评估的给药受试者中有7名（占比88%）与未给药眼相比显示出低亮度视力的改善或维持。截至三年期，未报告与 OCU400 相关的严重不良事件。

Ocugen 还与 Roots Pharmaceutical 就中东和北非地区的 OCU400 签署了独家许可协议。该公司有资格获得高达400万美元的首付款及潜在初始开发里程碑付款，高达2.55亿美元的潜在销售里程碑付款，以及净销售额22%的特许权使用费。Ocugen 将负责生产商业供应产品。

在巴哈马，OCU400 根据《长寿与再生疗法法案》获得了临时批准和优先认定。管理层预计将在年底前获得最终批准，并表示全球符合条件的患者可通过该项目获得治疗。公司表示，定价将与其在美国的目标价格保持一致。

用于斯塔加特病的 OCU410ST

在独立数据监察委员会（DMC）建议不作修改后，关键的 II/III 期 GARDian 临床试验正按现有方案继续推进。该研究包含63名患者，将在第12个月评估其主要终点。

管理层表示，根据治疗眼与对照眼之间约25%的萎缩病灶增长差异，该研究的设计统计效力接近90%。早期的 I/II 期数据表明，治疗眼的病灶增长减少了54%，功能性获益高达6个字母，尽管公司强调早期的研究涉及的患者样本量较小。

用于地图状萎缩的 OCU410

今年9月，全球 III 期 ArMaDa3 试验已完成首例患者给药。这项覆盖美国、加拿大和欧洲且包含237名患者的研究，将参试者按2:1的比例随机分配接受单次 OCU410 视网膜下注射或作为未治疗对照组。

主要终点是12个月内地图状萎缩病灶的增长情况。管理层目前不计划进行中期分析，目标是在2027年完成入组。

管理层业绩指引

管理层预计 OCU400 的 III 期顶线数据将于2027年第一季度公布，若结果支持进一步的监管审查，随后将按计划提交 BLA。

OCU410ST 的顶线数据预计将于2027年第二季度公布。公司计划在潜在的 BLA 预交前会议和提交之前，与美国食品药品监督管理局（FDA）就相关结果进行讨论。

对于 OCU410，管理层的目标是在2027年完成 ArMaDa3 的入组，并在2028年提交 BLA。Ocugen 继续致力于在2028年前完成三项 BLA 提交。

公司预计其8460万美元的流动资金储备将支持运营至2028年，并为其三个后期项目中的两个提供资金，直至完成预期的 BLA 提交。

风险与关注事项

• OCU400 的关键疗效数据仍未出炉，预计其主要 III 期结果读出时间为2027年第一季度。
• OCU410ST 的中期审查并非疗效分析。管理层指出了患者样本量小、8个月的时间节点较早以及基线特征异质性等局限性。
• 斯塔加特病进展缓慢，而基因治疗的表达也需要时间。因此，Ocugen 正依赖完整的12个月数据集来评估 OCU410ST。
• BLA 提交仍取决于临床结果、与 FDA 的讨论以及完成所需的监管步骤。
• 据报告的现金流维持时间仅能资助公司三个后期项目中的两个直至预期的 BLA 提交。
• 巴哈马渠道仍处于临时批准阶段，中心就绪状态和最终监管批准仍在推进中。

分析师问答集锦

• OCU410ST 中期审查：管理层确认在第8个月不会进行额外的全数据分析。公司仍保持盲态，并将评估第12个月的完整数据集。
• OCU410ST 试验规模：管理层表示，包含63名患者的研究统计效力接近90%，并预计完整人群和更长时间的随访将减少在较小中期队列中观察到的基线不平衡影响。
• OCU400 统计效力：III 期设计假设给药组患者的应答率为50%，对照组为10%，预估统计效力达到99%。管理层表示，假设给药组应答率为20%、对照组为1%，仍可提供93.2%的效力。
• OCU410 试验执行：未计划进行中期分析。管理层重申了在2027年完成入组并在2028年提交 BLA 的目标。
• 巴哈马准入模式：管理层表示，该项目向全球符合条件的患者开放，其运作方式更像是一个商业准入途径，而非传统临床项目。当地合作伙伴将协调患者和医疗提供者的宣教工作。

财报电话会议完整文本

完整财报电话会议逐字稿

管理层陈述

Operator

Good morning, and welcome to Ocugen's Third Quarter 2026 Financial Results and Business Update. [Operator Instructions] I will now turn the call over to Chris Clark, Ocugen's VP of Communications. You may begin.

Chris Clark

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. Rita Johnson-Greene, our Chief Financial Officer, is also on the call to provide a financial update for the quarter ended September 30, 2026.

Dr. Mohamed Genead, our Chief Medical Officer; Dr. Arun Upadhyay, our Chief Scientific Officer; and Abhi Gupta, Executive Vice President of Commercial and Business Development, will be available to answer questions following the presentation. This morning, we issued a press release covering our business and operational highlights for the third quarter of 2026. We encourage listeners to review the press release, which is available on our website at ocugen.com.

A replay of this call, along with the slide presentation, will be available on the Investors section of the Ocugen website. Before we begin, please note that certain statements made during today's discussion may be forward-looking in nature, including those related to our clinical development pipeline, regulatory time lines, commercialization strategy, international expansion and financial information and our anticipated cash runway.

These statements reflect management's current expectations and are inherently subject to risks, uncertainties and assumptions that may cause actual results to materially differ from those expressed or implied. We encourage you to review our filings with the Securities and Exchange Commission, including the risk factors detailed therein for a more comprehensive understanding of these potential risks. Finally, Ocugen's quarterly report on Form 10-Q covering the third quarter of 2026 will be filed today. I will now turn the call over to Dr. Musunuri.

Shankar Musunuri

Thank you, Chris, and good morning, everyone. The third quarter was a quarter of execution for Ocugen. We now have all 3 of our late-stage modifier gene therapy programs in Phase III. We advanced our business development strategy, and we established a presence in Europe to support pre-commercial activities. With 3 Phase III programs underway, we're executing against a clear strategy to advance our pipeline and build our global presence through strategic partnerships to bring our therapies to patients around the world.

Our modifier gene therapy platform remains the foundation of everything we do. Rather than targeting a single mutation, it is designed to address the underlying biology of complex retinal disease by restoring balance across multiple gene networks. The platform is gene agnostic. It is multifactorial, and it is designed to deliver durable benefit from a single onetime injection. That approach is what allows us to advance 1 platform across 3 major causes of blindness, geographic atrophy, retinitis pigmentosa and Stargardt disease.

We remain on track to file 3 BLAs by 2028, and our next major milestone is clear. OCU400 is expected to be the first of our Phase III programs to read out with top line data anticipated in the first quarter of 2027. Let me walk you through the progress made across each program this quarter. I will begin OCU400 in RP, followed by OCU410ST in Stargardt disease and OCU410 in GA before handing the call to Rita for the financial update.

In OCU400 for RP, this was an especially productive quarter for the program with progress on clinical, regulatory and commercial fronts. On the clinical side, we completed enrollment in our Phase III liMeliGhT trial with 140 patients. Top line data from the trial are expected in the first quarter of 2027, followed by our planned BLA submission. Our process performance qualification batches are complete, which supports both our BLA and the commercial launch supply of OCU400.

On the commercial side, in October, we completed an exclusive OCU400 license deal for the Middle East and North Africa with Roots Pharmaceutical, finalizing the binding term sheet we announced in July. Under the agreement, Ocugen is eligible for up to $4 million in combined upfront and potential initial development milestone payments, up to $255 million in potential sales milestones and a 22% royalty on net sales. And we will manufacture commercial supply. This is our second regional OCU400 license following our agreement with Kwangdong Pharmaceutical in South Korea.

On the regulatory and access side, OCU400 received provisional approval and priority designation in the Bahamas under its Longevity and Regenerative Therapies Act or LARTA. We have executed an agreement with a local partner, the Global Alliance for Regenerative Medicine, to coordinate and manage patient care, and we are progressing site readiness and preparation for full LARTA approval. We are advancing towards potential patient enrollment as early as year-end. This pathway is a meaningful step towards expanding access for RP patients while potentially generating commercial revenues.

Taken together, the access pathway to the Bahamas and the Roots license agreement reflect a deliberate strategy. We intend to bring OCU400 to patients in the United States to extend its reach internationally through regional partnerships that add commercial scale without requiring us to build our own infrastructure in every market. As we build that international reach, we remain focused on the clinical development pathway that will underpin our efforts to bring OCU400 to patients in major markets.

Our Phase I/II experience also provides important context for the Phase III liMeliGhT study. In a post-hoc analysis of 7 Phase I/II subjects meeting Phase III eligibility criteria, 7 treated eyes or 71.4% are projected to meet the Phase III responder threshold of at least a 2-level improvement on the LDNA scale from baseline at week 52. LDNA measures functional vision with a wider range and final resolution than the mobility test used in Phase I/II, addressing the ceiling effect seen in the study.

Our previously reported 3-year follow-up adds a longer-term perspective, 7 out of 8 evaluable concentrate treated subjects or 88% showed improvement or preservation of LLVA compared with untreated eyes at year 3 with no serious adverse events related to OCU400 reported through 3 years. Separately, our Phase III power analysis shows that even at hypothetical treatment responder rate of just 20% compared with 1% in untreated controls, the model estimates 93.2% power to detect the difference on the primary endpoint. We believe these data support both the design of liMeliGhT and the potential of OCU400, and we look forward to top line results in the first quarter of 2027.

Turning to OCU410ST for Stargardt disease, a pediatric onset retinal disorder for which there are no approved therapies. We continue to advance our pivotal Phase II/III GARDian trial per protocol, and we anticipate top line data in the second quarter of 2027 with the BLA submission to follow. Finally, OCU410 GA, an advanced form of dry age-related macular degeneration and our largest commercial opportunity. This quarter marked an important transition for the program into Phase III.

In September, we announced the first patient dosed in ArMaDa3, our global Phase III clinical trial. We plan to complete trial enrollment next year with a target BLA filing in 2028. ArMaDa3 is a 237-subject study that randomizes patients 2:1 to a single subretinal injection of OCU410 or untreated control across the United States, Canada and Europe. The primary endpoint is GA lesion growth over 12 months. This is the first pivotal gene therapy trial in geographic atrophy.

Today's approved GA therapies target a single pathway and require ongoing intravitreal injections as often as monthly. OCU410 is designed to address multiple pathways underlying the disease with a single onetime treatment. We believe that combination of mechanism and durability gives OCU410 a clear competitive advantage. Here is a snapshot of the market opportunity across our 3 late-stage development programs. With OCU410 for GA represents our largest commercial opportunity, we believe all 3 programs have the potential to generate significant revenue while addressing areas of substantial unmet medical need. With that, I will turn the call over to Rita for the financial update. Rita?

Rita Johnson-Greene

Thank you, Shankar. Good morning, everyone. Our cash, cash equivalents, restricted cash and short-term investments totaled $84.6 million as of September 30, 2026. We expect this balance to support our cash runway into 2028, which funds 2 of our 3 late-stage programs through their anticipated BLA filings. We had 339.3 million shares of common stock outstanding as of September 30, 2026.

Total operating expenses for the 3 months ended September 30, 2026, were $19.7 million and included research and development expenses of $12.5 million and general and administrative expenses of $7.2 million.

This compared to total operating expenses for the 3 months ended September 30, 2025, of $19.4 million, which included research and development expenses of $11.1 million and general and administrative expenses of $8.2 million. Ocugen reported a $0.01 net loss per common share for the 3 months ended September 30, 2026, compared to a $0.07 net loss per common share for the 3 months ended September 30, 2025.

Total operating expenses for the 9 months ended September 30, 2026, were $57 million and included research and development expenses of $34.4 million and general and administrative expenses of $22.6 million compared to total operating expenses for the 9 months ended September 30, 2025, of $50.5 million that included research and development expenses of $29.1 million and general and administrative expenses of $24.4 million. Ocugen reported a $0.14 net loss per common share for the 9 months ended September 30, 2026, compared to a $0.17 net loss per common share for the 9 months ended September 30, 2025. That concludes my financial update. Shankar, back to you.

Shankar Musunuri

Thank you, Rita. The progress we made this quarter reinforces our confidence in the path ahead. We moved OCU410 into Phase III with the first patient dosed in ArMaDa3. We completed our regional licensing agreement for OCU400 in the Middle East and North Africa. We opened a pathway to treat RP patients in the Bahamas, and we established a European presence to support our development and commercialization in that region. Each of these moves supports the same objective, which is advancing 3 late-stage gene therapy programs toward approval and bringing them to patients around the world.

Looking ahead, our focus is firmly on execution. The most important near-term catalyst is top line data from OCU400 in the first quarter of 2027, followed by our planned BLA submission. We then expect top line from OCU410ST in the second quarter of 2027. We remain on track to deliver 3 BLAs over the next 3 years. I want to thank our employees, our investigators and the patients who have placed their trust in us as well as our shareholders for their continued support of our mission to advance cures for blindness. We will now open the call for questions. Operator?

Operator

[Operator Instructions] Your first question comes from the line of Swayampakula Ramakanth with H.C. Wainwright.

分析师问答

Swayampakula Ramakanth

This is RK from H.C. Wainwright. The rest of the team. Just wanted to understand a little bit about the ST program, the Stargardt program, Shankar, in September, you had the DMC recommendation come to you. And when will the full cohort actually complete the follow-up period, the 8-month follow-up period? And beyond that, what sort of data do you need to make your next decision? And also, I think you're still planning to file in the second quarter of '27. So what else needs to get resolved by then?

Shankar Musunuri

Yes. Thank you, RK. Yes, the protocol, it targeted for 12 months read in the second quarter. I'll let our CMO, Dr. Genead, answer the questions. Go ahead.

Mohamed Genead

Thank you, Shankar. So as we described in the protocol, this interim analysis was prescribed in the original protocol we discussed with the agency. And it was a part of our adaptive design to look at any adjustment needed during the trial conduction. And the DMC was assembled, as you are aware, and they recommended to continue the study without modification.

So us, as a company, we will -- all the team are still blinded. We are continuing the study until month 12 and looking forward to release the data by the second half or second quarter next year in 2027. As you are aware, too, about this interim analysis was done in a small cohort of patients in the study to an early time point, the month 8 versus month 12 as the primary.

And as you know, for Stargardt disease as a disease, it is a slowly progressive retinal disease. So we wouldn't expect to see too much at month 8 versus month 12. In addition, as you know, with gene therapy product, the expression of the transgene and the product take time. And then with what we are trying to do here, we're looking at the data at month 12 per the original design on the pre-existing protocol.

Swayampakula Ramakanth

Okay. So if I understand this right, so I know I misspoke, I said second quarter '27 filing, the readout is the second quarter, just as you said. So once you have all that data, you anticipate meeting with the FDA before moving forward? Or what other steps would you be taking once you get that 12-month data point just as you suggested?

Rita Johnson-Greene

Yes.

Shankar Musunuri

Yes. I think, yes, that's the standard operating procedure, RK. When you get the top line data, you need to discuss with the agency first. Then you need to -- if the data falls the way you anticipate following the protocol and SAP analysis, what we agreed with the FDA, obviously, then we move on to the next step, have a pre-BLA meeting and move on to filing the BLA.

Swayampakula Ramakanth

Fantastic. Then regarding the LARTA approval in the Bahamas, first of all, I think it's a good opportunity for you folks to get something commercial and also see how the -- how patients react or adopt in the real world, which is fantastic. So when you say by year-end, by the end of this year, what would we see in terms of -- is it just going to be full approval and potentially somebody lining up to have their initial surgery? Or you just will have the full approval and the hospital will be trying to get somebody in -- get a surgery done in the new -- in '27, I mean.

Shankar Musunuri

Yes. So RK, I'll let -- we have our commercial head here, and he can tell it in answer.

Chris Clark

So right, RK, nice to hear from you. So the LARTA program in the Bahamas really represents something really innovative across our industry. And yes, in addition, right now, we have the conditional provisional approval. But by the end of the year, we can expect a full approval. We don't necessarily share forecasts on patients, especially for EAP, but stay tuned, and we will be sharing more catalysts on that specific program.

Operator

Your next question comes from the line of Robert LeBoyer with Noble. Your line is open.

Robert LeBoyer

My question only has to do with the 410 program. And good news that you initiated patient treatment during the quarter. And my understanding is that it's a 2:1 randomization, but I don't have anything about any kind of interim analysis. Is there anything planned like that?

Shankar Musunuri

Yes. As we -- Robert, as we -- this is a 1-year trial, that means after recruitment, we're going to monitor patients for 1 year, looking for primary and secondary analysis. And currently, we're not planning interim.

Robert LeBoyer

Okay. Are there any milestones for recruitment that you're planning to announce? Or is it just when you complete the full enrollment?

Shankar Musunuri

I mean our goal is to complete enrollment in 2027, in line for BLA filing in 2028.

Robert LeBoyer

Okay. Great. So everything is on track.

Shankar Musunuri

Yes.

Operator

Your next question comes from the line of Michael Okunewitch with Maxim Group. Your line is open.

Michael Okunewitch

I'd like to just talk a little bit more about the upcoming DMC review in Stargardt and provide some context on what the issues raised in the prior one. Did this have anything to do with the magnitude of treatment effect over the time period? Was it fluctuation in functional measures over that period with a smaller population? Or the statistical noise at baseline that kind of led them to say we want to see the full picture before making a decision?

Mohamed Genead

Yes. Thanks for the question. So it's all of the above, but let me give more specific. Let's just go back to the disease progression because this is important to understand. Stargardt, as you know, it's one of those slowly progressive retinal genetic diseases, and it takes time to see any delta and 8 months was quite an early time point. This is number 1.

Number 2, if you remember, the interim analysis also, we look at only a snapshot of certain patients, only -- even less than half of the patients in that trial. So the small N definitely will determine this outcome. The other big piece was in this analysis, the heterogeneity we saw at the baseline of those patients. Remember, still less than half of the patients enrolled in the trial and the baseline characteristic was just heterogenous. It was quite variable from small to large. So we cannot conclude, we cannot claim anything as well.

Lastly, and I want to also reiterate this part, this was not an efficacy analysis was a part of the adaptive design to look at any need to do any adjustment on the trial design, increase duration, increase the number of patients and the DMC recommended to continue without any modification. And for us, as a company, we will continue to follow the patient per the existing protocol to month 12, and then we'll look at the data and then the full set of the patient at month 12.

Michael Okunewitch

And just how confident are you that the N equals 63 total size of the study is large enough to ameliorate any of those baseline differences?

Mohamed Genead

Yes. Good point. Based on the prior data from the Phase I/II we saw treatment effect in a small number of patients was N of 9. We saw this 54% reduction or slower lesion growth in the treated eyes versus the untreated also, we saw functional benefit on the visual acuity.

Back to your question about the N of 63, it will be the full set of patients. The study was powered based on the early assumption to show significance at month 12 was close to 90% powering. And also, we should see like more moderate population, the baseline character should be balanced between the control and treated that also will drive a different value. In addition, we have a longer time point, right, month 12 versus month 8, and we saw the delta in our early study at month 12 versus month 8.

Michael Okunewitch

All right. And then just 1 more before I hop back in the queue. I want to switch gears and talk a little bit about the Bahamas program. In particular, what the payment model looks like for under LARTA? And who is the target patient for that? Would that be patients that live locally in the Bahamas? Would it be people flying in for potential medical tourism to get early access to the treatment? Can you just provide a bit more color on what that looks like?

Shankar Musunuri

Yes. I'll let Abhi answer that. Go ahead.

Abhi Gupta

Yes. So I think first on the program is open globally. And yes, it's looking at the Bahama population, but much more broader. So it is truly like a global program. And so secondly, the way that the model works in terms of pricing is something that's consistent with the way that the Bahama system is set up. And so it is a little bit more towards commercial than it is towards clinical, and that's something specific that the Bahama regulatory body enables Ocugen to do.

Shankar Musunuri

So Michael, just to recap, anybody globally who is eligible and they can go get the treatment at this stage under this approval.

Michael Okunewitch

All right. Would pricing match what you would be targeting for U.S. or international pricing? Or is this more of an exploratory program?

Chris Clark

That is correct. It would be. Yes, it would be.

Shankar Musunuri

It would be matching U.S. price. Yes.

Operator

Your next question comes from the line of Elemer Piros with Lucid Capital Markets. Your line is open.

Elemer Piros

Just 1 more follow-up on the Bahamas treatment availability. How would patients learn about that this is available in the Bahamas for them? What sort of marketing effort if you are thinking of to undertake?

Abhi Gupta

Yes. As an Ocugen, we don't market EAPs, but we are working with a local vendor that's responsible for all patient-related activities. And so they would be the one who are educating not only patients, but KOLs, including the community about this world's first option for a global EAP...

Elemer Piros

Okay. And coming back to the interim analysis, was the DMC equipped with trying to model potential outcome if they were to normalize the baseline characteristics to be even between the 2 arms?

Mohamed Genead

So DMC, as you know, it's an advisory body and also independent unblinded body assigned to the study. The DMC had a very clear per protocol assignment. They were looking at -- as part of the adaptive design, they're looking at 3 outcomes. Again, it's not an efficacy outcome. It's just part of the adaptive design for sample size recalculation or adjustment if needed.

We had access to the unblinded data. They look at all the efficacy, including the primary lesion size and all the other functional, including also the genetic correlation. So they were to make a decision based on data. As I said before, and this is important again to reiterate, the sample size was small, early time point and also we had some heterogenicity as a baseline characteristics in that interim analysis.

The good news also, remember, they had an opportunity now to do different outcomes. They continue the study without modification because we believe with all full of patients at month 12, the heterogenicity and all this baseline heterogeneity will be normalized at month 12. And this is also we believe based on our data and also patient selection in the trial.

Elemer Piros

And so you can confirm that since you have all the baseline characteristics of the patients that the full data set is going to be well balanced between the 2 treatment arms. That's correct.

Operator

Your next question comes from the line of Leland Gershell with Oppenheimer & Co. Your line is open.

Leland Gershell

Just want to ask, I think it was asked earlier on the Stargardt, the 8-month analysis. If we -- so just to be clear, will we -- will you the company have input from the DMC on their full analysis of the enrollment at 8 months? And will that -- could that impact the study? I just wasn't clear. I know that was asked in an earlier question, but it wasn't clear your answer. If you could just inform there.

Mohamed Genead

No, it's a pleasure. And no, so the DMC is independent, but they have access to the unblinded data. We don't. As a company, the study is still blinded and all the team are running the study is still blinded. If you're asking will the DMC will do another analysis at 8 months with a full data set? No.

We are continuing the study until month 12, and this is where the time is going to look at all the data from the trial as a company by month 12. And this is part of the design of the study. This is a pre-existing protocol. This what we communicate to the agency. And we believe the reason why because 8 months is an early time point, and we have a small number of patients. So we want to see the full set of patients at month 12 for the existing protocol. Did I answer your question?

Leland Gershell

Yes. No, that's very helpful. And I guess a question in terms of the final data when you have them, if you could just remind us what you expect to see based on performance in the past with 410ST, what's the size of lesion reduction you'd expect versus comparator? And given this is a dynamic space, kind of to be competitive here, what size reduction would you want to see given the other therapies being developed in the space?

Mohamed Genead

Yes. So the primary analysis for this study was based on the atrophic lesion size change over time until month 12. And the size was close to 90% to look at that delta. The delta you're talking about close to 25% delta. If you remember, the Phase I/II data we shared earlier or we discussed earlier, we showed close to 54% reduction on the lesion size, but this was an early study, was also a small number of patients, and that was our benchmark, but we went more conservative and we talked about 25% delta between the treated and the control eyes to show statistical significance.

That's on the lesion size growth. Also, we look at -- we're also looking at other functional endpoints, including the BCVA and other functional endpoint in the study. And we saw earlier in our Phase I/II functional benefit as well from baseline to month 12 up to 6-letter gain in the treated eye versus the contra untreated eyes. So we believe based on this earlier data, we should also see similar trend on the structural functional improvement in those subjects.

Operator

Your next question comes from the line of Whitney Ijem with Canaccord Genuity. Your line is open.

Whitney Ijem

On OCU400 and the powering, I guess, can you remind us from the Phase I/II experience, first of all, how are you defining responder in the Phase I/II as it relates to the mobility testing? And what are the differences between how you're defining it there versus the Phase III? And then remind us what was the responder rate, particularly in the RHO population in Phase I/II and remind us what percent of the Phase III are RHO patients? That's part 1. And then I have a follow-up. Sorry for so many of the questions.

Mohamed Genead

Whitney, thank you for your question. Regarding the Phase I/II, what we saw from the Phase I/II in the RP, the OCU400 program, this was N of 18 subject. Remember, we did some subgroup analysis. But what we saw based on the mobility test, and we used the same test externally used for their approval, which we called the Multi-Luminance Mobility Test, MLMT.

What we saw in that study in the treated eye who received OCU400, we saw 43% of the eyes did improve 2 lux levels on the mobility test, which is the approvable endpoint for RP versus 0% of the control untreated eyes did show that. And then when we projected this, to our Phase III mobility test, which we call the LDNA, which is very similar to the MLMT, but it's a broader range, 10 lux level versus 6, but using the same methodology, the 2 lux level improvement.

And our projection was at that time, we went from 42% up to -- at 43% up to 71% improvement in the treated eye versus 0% in the control. So when we built our Phase III assumption was based on a very conservative analysis. We didn't say we're going to go with 71% improvement. We said the treated eye will -- the assumption was 50% improvement in the treated eye versus 10% control. And remember, in the Phase I/II, the control was 0%.

So if you went with a 50% treated versus the 10% control, the delta is 40%, that will give us 99% power in the study, okay? And if you look at this in another way, right, if you look at the half full or half empty, right, even if the treated eye showed only 20% treatment effect, which is not our assumptions, right? This is not our assumption. But if you went to 20% and the control did behave exactly the way it behaved in the Phase I/II, which is like close to 0%, 1%, we're still hitting 93% power. So what we're saying here in a different way, we have a very high confidence with OCU400, especially the study is well powered to show statistical significance at month 12. Using the 2 lux improvement, the same 1 used for the approval. Did I answer your question?

Whitney Ijem

Got it. That's really helpful. Yes, that's really helpful. And just on the RHO bit, am I remembering correctly that on the Phase I/II, the responder rate in the RHO subgroup was higher? Either way, I guess, can you remind us what percent of patients in the pivotal are RHO patients?

Mohamed Genead

Yes. So for the pivotal trials, liMeliGhT, just to remind, it's 140 subjects were enrolled, randomized to 2:1, 92 was treated and 48 was untreated or the control arm. The reduction we have like we have rhodopsin patients in the Phase III trials, but the majority will be non-rhodopsin, we call gene agnostic. But we have a very large number of patients in the Phase III trials who have rhodopsin mutation. And you're correct, we saw a very positive trend in the rhodopsin subpopulation in the Phase I/II. So we are expecting to replicate if not better in our Phase III trials.

Whitney Ijem

Got it. All right. That's helpful. And then just the last question is just on timing. I believe you announced completion of enrollment in the study in the beginning of March. Can you just help us understand like how quickly after enrollment was that final patient treated? I'm just trying to get -- and how long you think it might take to kind of organize, clean up the database, et cetera, before you can lock it and announce top line?

Shankar Musunuri

I mean, as we stated before, Whitney, we're ready to do all that. That's why we're doing a lot of work behind the scenes. As we announced before, first quarter -- by end of first quarter, we'll be able to announce that top line.

Operator

I will now turn the call back over to Shankar Musunuri for closing remarks.

Shankar Musunuri

Thank you. Thank you for attending another good progressive earnings call today. Thanks to all our shareholders, patients, our employees for supporting Ocugen. Thank you.

Operator

Ladies and gentlemen, that concludes today's call. Thank you all for joining. You may now disconnect.