Conferencia de resultados del 3T de 2026 de Ocugen (OCGN): Lectura de datos de OCU400 prevista para el 1T de 2027

Puntos clave

• Ocugen (NASDAQ: OCGN) cerró el tercer trimestre de 2026 con 84,6 millones de dólares en efectivo, equivalentes de efectivo, efectivo restringido e inversiones a corto plazo. La dirección prevé que esto le proporcione margen de liquidez hasta 2028 y financie dos de sus tres programas en fase final hasta las presentaciones previstas de solicitudes de licencia de productos biológicos (BLA).
• Los tres programas de terapia génica modificadora se encuentran ya en la Fase III. La dirección mantiene sus previsiones de presentar tres solicitudes de licencia de productos biológicos (BLA) para 2028.
• Ha finalizado la inscripción del ensayo de Fase III liMeliGhT de OCU400 para la retinosis pigmentaria, con 140 pacientes. Se esperan los datos principales en el primer trimestre de 2027, seguidos de la presentación prevista de la BLA.
• Se prevé que los datos principales de OCU410ST para la enfermedad de Stargardt se publiquen en el segundo trimestre de 2027. Tras una revisión intermedia, el comité independiente de supervisión de datos (DMC) recomendó continuar con el estudio sin modificaciones.
• OCU410 ha entrado en la Fase III para la atrofia geográfica. Ha comenzado la dosificación del ensayo ArMaDa3 con 237 pacientes; el objetivo es completar la inscripción en 2027 y presentar la BLA en 2028.
• Los gastos operativos del tercer trimestre fueron de 19,7 millones de dólares, frente a los 19,4 millones de dólares del año anterior. La pérdida neta se redujo a 0,01 dólares por acción desde los 0,07 dólares por acción.

Datos financieros clave

El efectivo, los equivalentes de efectivo, el efectivo restringido y las inversiones a corto plazo sumaban un total de 84,6 millones de dólares a 30 de septiembre de 2026. Ocugen contaba con 339,3 millones de acciones ordinarias en circulación.

Rendimiento comercial y operativo

OCU400 en retinosis pigmentaria

Ocugen completó la inscripción de 140 pacientes en el ensayo de Fase III liMeliGhT. El estudio aleatorizó a 92 pacientes para recibir tratamiento y a 48 para el grupo de control sin tratamiento. Se completaron los lotes de cualificación de rendimiento del proceso, lo que respalda tanto la BLA prevista como el potencial suministro para el lanzamiento comercial.

La dirección citó un análisis post-hoc de siete sujetos de la Fase I/II que cumplían los criterios de elegibilidad de la Fase III, en el que se proyectó que el 71,4% de los ojos tratados alcanzaría el umbral de respuesta de la Fase III de al menos una mejora de dos niveles en la escala LDNA en la semana 52.

A los tres años, siete de cada ocho sujetos tratados evaluables, es decir, el 88%, mostraron una mejora o conservación de la agudeza visual con baja luminancia en comparación con los ojos no tratados. No se notificaron acontecimientos adversos graves relacionados con OCU400 durante los tres años.

Ocugen también cerró un acuerdo de licencia exclusiva de OCU400 con Roots Pharmaceutical para Oriente Medio y el norte de África. La empresa podrá recibir hasta 4 millones de dólares en pagos iniciales combinados e hitos iniciales de desarrollo, hasta 255 millones de dólares en hitos potenciales por ventas y una regalía del 22% sobre las ventas netas. Ocugen fabricará el suministro comercial.

En Bahamas, OCU400 recibió aprobación provisional y designación prioritaria en virtud de la Ley de Longevidad y Terapias Regenerativas. La dirección prevé la aprobación definitiva para finales de año y afirmó que los pacientes de todo el mundo que cumplan los requisitos podrían acceder al tratamiento a través del programa. La empresa indicó que el precio coincidiría con su precio objetivo en Estados Unidos.

OCU410ST en la enfermedad de Stargardt

El ensayo pivotal de Fase II/III GARDian continúa según su protocolo actual después de que el DMC independiente recomendara no introducir modificaciones. El estudio incluye 63 pacientes y evaluará su criterio de valoración principal al mes 12.

La dirección afirmó que el estudio se diseñó con una potencia estadística cercana al 90% basándose en una diferencia aproximada del 25% en el crecimiento de la lesión atrófica entre los ojos tratados y los de control. Los datos anteriores de la Fase I/II mostraron una reducción del 54% en el crecimiento de la lesión y un beneficio funcional de hasta seis letras en los ojos tratados, aunque la empresa enfatizó que el estudio anterior contó con una población reducida de pacientes.

OCU410 en la atrofia geográfica

En septiembre se administró la dosis al primer paciente en el ensayo global de Fase III ArMaDa3. El estudio, con 237 pacientes, aleatoriza a los participantes en una proporción 2:1 a una única inyección subretiniana de OCU410 o a un grupo de control sin tratamiento en Estados Unidos, Canadá y Europa.

El criterio de valoración principal es el crecimiento de la lesión por atrofia geográfica durante 12 meses. La dirección no planea actualmente realizar un análisis intermedio y tiene como objetivo completar la inscripción en 2027.

Orientación de la dirección

La dirección prevé los datos principales de la Fase III de OCU400 para el primer trimestre de 2027, seguidos de la presentación prevista de la BLA si los resultados respaldan una posterior revisión regulatoria.

Se prevén los datos principales de OCU410ST para el segundo trimestre de 2027. La empresa tiene previsto analizar los resultados con la FDA antes de una eventual reunión previa a la BLA y su posterior presentación.

Para OCU410, la dirección se fija como objetivo completar la inscripción del ensayo ArMaDa3 en 2027 y la presentación de la BLA en 2028. Ocugen mantiene su objetivo de realizar tres presentaciones de BLA para 2028.

La empresa prevé que su posición de liquidez de 84,6 millones de dólares respaldará las operaciones hasta 2028 y financiará dos de sus tres programas en fase final hasta las presentaciones de BLA previstas.

Riesgos y factores a vigilar

• Los datos de eficacia pivotal de OCU400 siguen pendientes, y la lectura principal de la Fase III se prevé para el primer trimestre de 2027.
• La revisión intermedia de OCU410ST no fue un análisis de eficacia. La dirección citó como limitaciones la reducida muestra de pacientes, el momento temprano a los ocho meses y las características heterogéneas de referencia.
• La enfermedad de Stargardt avanza lentamente, mientras que la expresión de la terapia génica requiere tiempo. Por lo tanto, Ocugen confía en el conjunto completo de datos a los 12 meses para evaluar OCU410ST.
• Las presentaciones de las BLA siguen sujetas a los resultados clínicos, las conversaciones con la FDA y la finalización de los trámites regulatorios requeridos.
• El margen de liquidez notificado financia solo dos de los tres programas en fase final de la empresa hasta las presentaciones previstas de las BLA.
• La vía de Bahamas se mantiene bajo aprobación provisional, mientras que la preparación de los centros y la aprobación regulatoria definitiva siguen en curso.

Puntos destacados del turno de preguntas de los analistas

• Revisión intermedia de OCU410ST: La dirección confirmó que no habrá ningún análisis adicional de datos completos al mes ocho. La empresa se mantiene en doble ciego y evaluará el conjunto completo de datos al mes 12.
• Tamaño del ensayo de OCU410ST: La dirección señaló que el estudio con 63 pacientes cuenta con una potencia cercana al 90% y espera que la población completa y un seguimiento más prolongado reduzcan el efecto de los desequilibrios de referencia observados en la cohorte intermedia, más reducida.
• Potencia estadística de OCU400: El diseño de la Fase III asumió una tasa de respuesta del 50% para los pacientes tratados frente al 10% para los controles, lo que ofrece una potencia estimada del 99%. La dirección indicó que una hipotética tasa de respuesta al tratamiento del 20% frente al 1% en los controles aún proporcionaría una potencia del 93,2%.
• Ejecución del ensayo de OCU410: No hay ningún análisis intermedio previsto. La dirección reiteró su objetivo de completar la inscripción en 2027 y presentar la BLA en 2028.
• Modelo de acceso en Bahamas: La dirección indicó que el programa está abierto a pacientes elegibles de todo el mundo y funciona más como una vía de acceso comercial que como un programa clínico convencional. Un socio local coordinará la formación de pacientes y profesionales sanitarios.

Transcripción completa de la llamada de resultados

Transcripción completa de la conferencia de resultados

Comentarios de la dirección

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.

Preguntas y respuestas

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.