Q1 2025 Ocugen Inc Earnings Call
Operator: Thank you for watching!
[music].
Operator: Good morning and welcome to Ocugen's first quarter 2025 financial results and business update. Please note that this call is being recorded at this time. All participant lines are in listen-only mode.
Good morning, and welcome to <unk> first quarter, 'twenty 25 financial results and business update. Please note that this call is being recorded at this time all.
All participant lines are in listen only mode.
Operator: Following the speaker's commentary, there will be a question and answer session.
Following the Speakers' commentary there will be a question and answer session I will now turn the call over to Tiffany Hamilton <unk> head of corporate Communications you may begin.
Tiffany Hamilton: I will now turn the call over to Tiffany Hamilton, Ocugen's Head of Corporate Communications. You may begin.
Tiffany Hamilton: Thank you, operator, and good morning, everyone.
Tiffany Hamilton: Thank you operator, and good morning, everyone. Joining me on today's call and webcast is Dr.
Tiffany Hamilton: 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 overview of our clinical and operational progress.
Sorry.
Dr. Sorry: Chairman CEO and co founder, who will provide a business update and overview of our clinical and operational progress <unk>, Our chief Accounting Officer is also on the call to provide a financial update for the quarter ended March 31 2025.
Tiffany Hamilton: Ramesh Ramachandran, our chief accounting officer, is also on the call to provide a financial update for the quarter ended March 31st, 2025.
Tiffany Hamilton: Dr. Huma Qamar, our chief medical officer, will be available to answer questions following the presentation. This morning we issued a press release detailing associated business and operational highlights for the first quarter of 2025. We encourage listeners to review the press release, which is available on our website at ocugen.com.
Speaker Change: Dr. <unk> <unk>, our chief Medical officer will be available to answer questions. Following the presentation.
Speaker Change: This morning, we issued a press release detailing associated business and operational highlights for the first quarter of 2025, we encourage listeners to review the press release, which is available on our website at <unk> Dot Com. This call is being recorded and a replay with the accompanying slide presentation will be available on the investors section of our website.
Operator: This call is being recorded and a replay with the accompanying slide presentation will be available on the investor section of our website for approximately 45 days.
Speaker Change: For approximately 45 days.
Operator: 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, expects, plans, intend, may, could, might, will, should, or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements include, but are not limited to, statements regarding our clinical development activities and related anticipated timelines. 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.
Speaker Change: The presentation contains forward looking statements within the meaning of the private Securities Litigation Reform Act of 1095, which are subject to risks and uncertainties. We may in some cases use terms such as predicts believe potential proposed continue estimate anticipates expects plans intends.
Speaker Change: May could might will should or other words that convey uncertainty of future events or outcomes to identify these forward looking statements.
Speaker Change: Such statements include but are not limited to statements regarding our clinical development activities and related anticipated timeline 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.
Operator: These and other risks and uncertainties are more fully described in our periodic filing with the Securities and Exchange Commission, the SEC, including the risk factors described in the section entitled Risk Factors in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this presentation speak only to the date of the presentation. Except it's 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.
Speaker Change: These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission the SEC, including the risk factors described in the section entitled Risk factors in our quarterly and annual reports that we file with the SEC any forward looking statements that we make in this presentation speak only to the date of the presentation.
Speaker Change: 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.
Operator: Finally, FAFSA's quarterly report on Form 10-Q, covering the first quarter of 2025, was filed today.
Speaker Change: Finally, <unk> quarterly report on Form 10-Q, covering the first quarter of 2025 was filed today.
Shankar Musunuri: I will now turn the call to Dr. Musunuri. Thank you, Tiffany, and thank you all for joining us today. As detailed in our press release, we are excited to discuss the substantial progress of our game-changing modifier gene therapy platform. All three gene therapies are advancing through the clinic, and we're on track to meet our goal of three biologics license application slash market authorization application filings in the next three years. Modified gene therapies offer potential cures for life. Unlike traditional gene therapies or gene editing, modified gene therapies have the ability to regulate gene networks, reset homeostasis, restore the functional network, and create a healthy environment for retinal cells to survive.
Speaker Change: I will now turn the call to Dr listener.
Dr. Listener: Thank you Tiffany and thank you all for joining us today as detailed in our press release, we're excited to discuss the substantial progress <unk> Marty for your gene therapy platform, all three gene therapies for advancing through the clinic and we're on track to meet our goal of three.
Speaker Change: Biologics license application.
Speaker Change: Cash market authorization application filings in the next three years monitoring gene therapies offer potential cures for life. Unlike traditional gene therapies gene editing Martin <unk> therapies.
Speaker Change: <unk> to regulate gene networks reshaped homeostasis the store the functional network and create a healthy environment for <unk> since <unk> and retinal cells are underwriting, creating a healthy environment is a powerful concept and soc can produce useful way for life with the therapy today.
Shankar Musunuri: Since photoreceptors and retinal cells are non-dividing, creating a healthy environment is a powerful concept, and cells can potentially survive for life with this therapy. Today, based on the unique mechanism of action, these therapies target diseases with large patient populations globally, millions, instead of a very small group of hundreds to a few thousand patients, as with the traditional gene therapies in the market today. Positive clinical data continues to affirm the modified gene therapy approach. We started off the year with a two-year safety and efficacy data from the Phase 1 to RQ400 clinical trial that further supports the gene agnostic mechanism action of RQ400, a broad RP treatment not restricted to specific mutations with durability.
Speaker Change: Based on the unique mechanism of fraction these therapies target diseases, the large patient populations globally.
Speaker Change: The first of a very small group of hundreds to a few thousand patients as with the operational gene therapies in the market today.
Speaker Change: Positive clinical data continued to f-1, the Modifier Gene Therapy approach. We started of the year with the two-year safety and efficacy data from the Phase 1 to OXI-400 clinical trial that further supports the gene-agnostic mechanism action of OXI-400, a broad RP treatment not restricted to specific mutations with durability.
Shankar Musunuri: It was especially gratifying to reach an alignment with the FDA for the OCU-410ST Phase II-III Pivotal Confirmative Trial, which can be the basis of a BLA submission in 2027. Accelerating the clinical timeline of RQ-4 tenacity will save significant costs and potentially address disease burden even sooner than anticipated. Finally, dosing was complete. They have a schedule in the Phase 2 portion of the OCU-410 Phase 1-2 R-model clinical trial for geographic atrophy, late-stage dry AMD. We are planning to initiate the Phase III clinical trial in 2026 on a target with a PLA submission in 2028.
Speaker Change: It was especially gratifying to reach an alignment with the FDA for the RQ-410ST Phase 2-3 Departmental Confirmative Trial which can be the basis of the BLA submission in 2027.
Speaker Change: Accelerating the commercial timeline of active fraternesty, the sale of significant costs and potentially economic at-rust deceased burden even sooner than anticipated.
Speaker Change: Finally, dosing was complete, Ayagav Skedyo, and the face-to-potion of the occuportant face want to arm our doctorical trial for geographic atrophy, latest stage-pride A and B.
Speaker Change: We are planning to initiate the Phase 3 clinical trial in 2026 on the target with a PLA submission in 2028. I will now provide additional details around the program updates.
Shankar Musunuri: I will now provide the additional details around the program update. Rp400 has the potential to treat approximately 300,000 Rp patients in the U.S. and the EU and 1.6 million patients globally using a gene-agnostic approach delivered via single sub-regional injection to target all 100 genes associated with Rp. A traditional gene therapy approach would require developing 100 different products to address all 100 genes, which is not commercially viable. More significantly, recent news affirms that a traditional gene therapy approach is ineffective in achieving clinically meaningful outcomes for treating RP. The positive two-year long-term safety and efficacy data from the Phase I-II clinical trial of RQ400 for RP demonstrates a durable and statistically significant with a p-value of 0.005 improvement in the visual function, LLVA, in all available treated subjects at two years when compared to untreated eyes.
Speaker Change: The U S and EU and one 6 million patients globally using the generic nonsurgical approach Delaware. We are single so brickman injection to target all 100 genes associated with RP.
Speaker Change: <unk> gene therapy approach would require developing 100 different products to address all hundred genes, which is not commercially viable.
Speaker Change: More significantly recent news platforms, the traditional gene therapy approaches in Mifid, II and achieving clinically meaningful outcomes for treating RP.
Speaker Change: The positive two year long term safety and efficacy data from the phase one two clinical trial of our Q4 hundred for RFP demonstrates a durable and statistically significant with the P value of 0.00 file.
Speaker Change: Improvement in the visual function.
Speaker Change: All evaluable treated subjects for two years.
Speaker Change: Compared to untreated.
Shankar Musunuri: Additionally, 100%, 10 out of 10, of treated available subjects demonstrated improvement or preservation in visual function compared to untreated eyes. The Phase 3 study, spanning one year, will enroll 150 participants divided into two study groups, 75 participants with the row gene mutations, and 75 participants who are gene agnostic. In each, our participants will be randomized in a two-to-one ratio to receive either treatment or remain in an untreated control group, respectively. The RQ-400 Phase III Limelight clinical trial is open to all eligible R.T. patients. early to advanced HRP including pediatric subjects age 5 plus regardless of gene mutation.
Speaker Change: Additionally, 100% 10 out of 10 are featured Evaluable subjects demonstrated improvement or preservation in visual function compared to untreated eyes.
Speaker Change: The phase III study spanning one year will enroll 150 participants divided into two Sterling pumps 75 participants withdraw gene mutations and 75 participants who are agnostic and each are party strengths would be randomized in a two to one ratio.
Speaker Change: To receive treatment in an untreated control group perspective.
Speaker Change: The <unk> hundred phase III lend light clinical trial is open to all eligible RP patients.
Speaker Change: Early two advanced HRP, including pediatric subjects.
Speaker Change: <unk> price plus regardless of gene mutation.
Shankar Musunuri: Syndromic and non-syndromic forms of RP. Two key parts of our potential BLA-MAA filings next year are on schedule. The first is manufacturing, and we are tracking to complete process validations this year in support of registration. This material can also be used for commercial supply in 2027. The second part, the phase sequence of trials, is progressing well. Just this week, EMA granted eligibility to submit our RQ-400 MAA via centralized procedure as an ATMP based on the current study design and statistical analysis plan. This is a significant project milestone for RQ-400. EMA has appointed a project lead to support any queries related to the application, and six months prior to MAA submission, a rapporteur and a co-rapporteur will be appointed to support the application.
Speaker Change: Syndromic and non Syndromic forms of RP.
Speaker Change: Two key parts of a potential BLA slash MAA filings next year are on schedule and the cost is manufacturing and we are tracking to complete process validation this year in support of registration.
Speaker Change: Material can also be used for commercial supply in 2027, the second part of the phase III clinical trial is progressing well.
Speaker Change: Just this week EMA granted eligibility to submit our our Q4 hundred.
Speaker Change: We have centralized procedure as an <unk> based on.
Speaker Change: The current study design and statistical analysis plan. This is a significant project milestone for <unk> hundred <unk> has appointed a project lead to support any queries related to the application and six months prior to NDA submission the rapid door and the <unk> will be appointed to support the application.
Shankar Musunuri: This eligibility grant is a testament to EMA's recognition of the potential of RQ-400 to address serious unmet medical needs in Europe. There is currently no approved treatment option for star blood disease that encompasses more than 1,200 mutations and affects 100,000 people in the U.S. and EU, and 1 million globally. The FDA's decision to approve a Phase 2-3 trial for registration underscores the potential of Ocu410SD to meet this critical unmet medical need and has never been explored in clinical trials for star blood disease. Data from the OCUFORT-NST Phase I Guardian Clinical Trial has shown significant improvements in both structural and functional outcomes.
Speaker Change: This eligibility grant is a testament to <unk> recognition of the potential of our Q4 hundred to address serious unmet medical need in Europe.
Speaker Change: There is currently no approved treatment option for target disease that encompasses more than 200 mutations and FX 100000 people in the U S and EU and $1 million globally.
Speaker Change: <unk> decision to approve the phase III trial for registration underscores the potential of our Q4 tenants deep to meet this critical unmet medical need and has never been explored in clinical trials for <unk> disease.
Speaker Change: Data from the occupancy finished the phase <unk> Guardian clinical trial has shown significant improvements in both structural and functional outcomes.
Shankar Musunuri: Additionally, RQ410ST has consistently demonstrated a very favorable safety and tolerability profile. In BCVA, treated eyes demonstrate a two-line of channelateral gain in the visual equity compared to untreated eyes and a statistically significant p-value of 0.02 improvement in visual function when compared to untreated fellow eyes. In the latest data, atrophic lesions grew slower by 54% at 6 months and by 103% at 12 months in available treated eyes when compared to untreated eyes. The Phase 2-3 pivotal confirmatory clinical trial, which includes an adaptive design with an interim masked analysis at eight months, will randomize 51 subjects, 34 of whom will receive a single subretinal injection of RQ410ST in the eye with the worst visual acuity, and 17 of whom will serve as untreated controls.
Speaker Change: Finally, our Q4 to this team has consistently demonstrated a very favorable safety and tolerability profile.
Speaker Change: And <unk> <unk> demonstrate it to line of <unk> gain and the visual equity compared to untreated eyes and a statistically significant.
Speaker Change: <unk> annual point or two improvement in visual function when compared to untreated for otherwise.
Speaker Change: And the latest data appropriate lesions grew slower by 54% at six months and by 103% at 12 months and Evaluable treated eyes, when compared to untreated eyes.
Speaker Change: The phase III pivotal complementary clinical trial, which includes an adaptive design with an interim <unk> analysis. It's eight months with randomized 50 on subjects 34 of whom will receive a single sub retinal injection of <unk> <unk> in the eye with the worst vision.
Speaker Change: Equity and 17 of the serve as untreated controls the.
Shankar Musunuri: The primary endpoint in the clinical trial is changing atrophic lesion size. Secondary endpoints include visual equity as measured by Best Corrected Visual Equity, BCVA, and LLVA, compared to untreated controls. One-year data will be utilized for the BLA filing.
Speaker Change: The primary endpoint in the clinical trial is change in retrofit collision slice.
Speaker Change: Secondary endpoints include Brazil equity as measured by best corrected visual acuity <unk>.
Speaker Change: And <unk> compared to untreated controls.
Speaker Change: One year data will be utilized for the BLA filing.
Shankar Musunuri: Octu410, specifically designed to address multiple pathways implicated in the pathogenesis of dry AMD, offers a distinct advantage over current treatments that target only one cause of GA, require multiple injections per year, and are accompanied by various safety concerns. Our goal with Octu410 is to provide a comprehensive and durable solution, a potential one-time therapy for life for the 2 to 3 million people in the U.S. and Europe, and 8 million globally suffering GA. In February, dosing was complete in the Phase 2 portion of the RQ-410 Phase 1-2 Armada clinical trial for GA. In evaluable subjects, RQ-410 12-month data demonstrates a 4-line, a 23-letter gain in the visual equity.
Our Q4 time, specifically designed to address multiple pathways implicated in the pathogenesis of dry AMD offer us a distinct advantage over current treatments that target only one cause of GE required multiple injections per year and our company by various <unk>.
Speaker Change: The concerns.
Speaker Change: Our goal with <unk> is to provide a comprehensive and durable solution.
Speaker Change: Potential onetime therapy for life for the two to 3 million people.
Speaker Change: In the U S and euro and $8 million globally suffering.
Speaker Change: In February dosing was complete in the phase II portion of the <unk> phase one to our modern clinical trial for <unk> and.
Speaker Change: In Evaluable subjects are Q4, 10, 12 month data demonstrates a fourth line or put it to the letter gain and the visual equity.
Shankar Musunuri: there was 41% slower GA lesion growth in treated eyes versus untreated fellow eyes after a single injection. Furthermore, at 12 months, Octoporten treatment preserves more retinal tissue around the GA lesions of treated eyes compared to commercially available products given monthly or every other month. In the Phase 2 study, the safety and efficacy of RQ410 in patients with GA, secondary to dry AMD, will be assessed. Fifty-one patients with randomized one-to-one into either of two treatment groups, medium or high dose, are a control group. In the treatment group, subjects received a single subretinal 200-microliter administration of medium dose or high dose, while the control remained untreated.
Speaker Change: There was 41% slower GH lesion growth and features EIS versus untreated fellow eyes operating injection.
Speaker Change: Furthermore, at 12 months occupancy treatment Brazil's motor is not this year around the Gi lesions of <unk> treated eyes compared to commercially available products given monthly or every other month.
Speaker Change: In the phase II study, the safety and efficacy of <unk> <unk> in patients with the.
Speaker Change: Secondary to dry AMD will be assessed 51 patients with a randomized one to one to one.
Speaker Change: Do either of two treatment groups.
Speaker Change: Liam our hydro's are a control group.
Speaker Change: And the treatment group subjects received a single sub retinal 200, Microliter administration of medium dose or high dose when the control remain untreated.
Shankar Musunuri: The Data and Safety Monitoring Board has evaluated safety in all 51 patients from Phase 2 clinical trials. Today, there have been no serious adverse events related to RQ410. Phase 2 interim results are expected in the fall of this year.
Speaker Change: The data and safety monitoring board has evaluated safety and all 51 patients from phase two clinical trial to date, there have been no serious adverse events related to our Q4 Ken.
Speaker Change: Phase two interim results are expected in the fall off this year.
Shankar Musunuri: I would also like to provide a summary of programs outside of our first-in-class Modified Gene Therapy platform.
Speaker Change: I would also like to provide a summary of programs <unk> first in class modified gene therapy platform.
Shankar Musunuri: Earlier this year, the first patient was dosed in the Phase I clinical trial for OPQ-200, the company's biologic product candidate for diabetic macular edema. We are currently dosing the second cohort based on first cohort safety data.
Speaker Change: Earlier this year the first patient was dosed in the phase <unk> clinical trial for <unk> 200, the company's biologic product candidates for diabetes macular edema.
Speaker Change: We are currently dosing the second cohort based on first cohort safety data.
Shankar Musunuri: Ocugen plans to complete the Phase I clinical trial in the second half of 2025.
Speaker Change: <unk> plans to complete the phase one clinical trial in the second half of 2025.
Shankar Musunuri: The company intends to initiate the Phase III trial for neocort contingent on adequate availability of funding and are based on the potential. of a future partnership.
Speaker Change: The company intends to initiate the phase III trial for Neocart.
Speaker Change: Finjan on illiquid branded retail funding and are based on the potential.
Speaker Change: If future partnership lastly, the investigational new drug application is in effect for occupy 100, and the National Institute of allergy and infectious diseases.
Shankar Musunuri: Lastly, the investigational new drug application is in effect for Occupy 100 and the National Institute of Allergy and Infectious Diseases intends to initiate a phase one clinical trial in the second quarter of 2025.
Speaker Change: In terms to initiate a phase one clinical trial in the second quarter of 2025.
Shankar Musunuri: Ocugen is continuing discussions with relevant government agencies as well as strategic partners regarding developmental funding for its vaccine technology for flu.
<unk> continuing discussions with the relevant government agencies as well as strategic partners regarding developmental funding for its vaccine technology for flu.
Ramesh Ramachandran: With that, I will now turn the call over to our Chief Accounting Officer, Ramesh Ramachandran, to provide an update on our financial results for the first quarter ended March 31st, 2025. Thank you, Shankar. Our research and development expenses for the quarter ended March 31, 2025, were $9.5 million compared to $6.8 million for the first quarter of 2024. General and administrative expenses for the quarter ended March 31, 2025, were $6.5 million compared to $6.4 million during the same period in 2024.
Speaker Change: With that I will now turn the call over to our Chief Accounting Officer formation Ramachandra to provide an update on our financial results for the first quarter ended March 31 2020.
Speaker Change: <unk> formation.
Speaker Change: Thank you Shankar.
Speaker Change: Our research and development expenses for the quarter ended March 31, 2025 nine.
Speaker Change: $9 5 million compared to $6 8 million for the first quarter of 2020 for Jen.
Speaker Change: General and administrative expenses for the quarter ended March 31, 2025 were $6 5 million compared to $6 4 million during the same period in 2024.
Ramesh Ramachandran: Net loss was approximately $15.3 million or $0.05 net loss per share for the quarter ended March 31, 2025, compared to a net loss of approximately $11.9 million or $0.05 net loss per share for the first quarter of 2024. Our cash and restricted cash total $38.1 million as of March 31, 2025, compared to $58.8 million as of December 31, 2024. The company expects that its cash and restricted cash will provide the company cash runway into the first quarter of 2026. As always, we are constantly exploring strategic and shareholder-friendly opportunities to increase our working capital.
Speaker Change: Net loss was approximately $15 3 million or <unk> <unk> net loss per share for the quarter ended March 31, 2025 compared to a net loss of approximately 11 by $9 million or <unk> net loss per share for the first quarter of 2024, our cash and restricted.
Speaker Change: Cash totaled $38 $1 million as of March 31, 2025, compared to $58 8 million as of December 31, 2024, the company expects that its cash and restricted cash will provide the company cash runway into the first quarter of 2026 as always.
Tiffany Hamilton: We are constantly exploring strategic and shareholder friendly opportunities to increase our working capital that completes my update for the quarter Tiffany and back to you.
Ramesh Ramachandran: That concludes my update for the quarter.
Tiffany Hamilton: Tiffany, back to you. Thank you, Ramesh.
Tiffany Hamilton: Thank you very much we will now open the call for questions operator.
Operator: We will now open the call for questions. Operator. Thank you.
Tiffany Hamilton: Thank you we will now begin the question and answer session. If you would like to ask a question. Please press star one on your telephone keypad to Asia and Jan. Thank you Anthony you all your questions in your press Star one again.
Operator: We will now begin the question and answer session. If you would like to ask a question, please press star 1 on your telephone keypad to raise your hand and join the queue. And to withdraw your question, simply press star 1 again.
Operator: If you are called upon to ask your question and are listening via loudspeaker on your device, please pick up your handset and ensure that your phone is not on mute when asking your question. Again, press star 1 to join the call.
Tiffany Hamilton: If you are called upon to ask your question and then listen via loud speakers.
Tiffany Hamilton: Please pickup your handset and ensure that your phone is not on mute and asking a question.
Tiffany Hamilton: Press Star one to join the queue.
Michael Okunewitch: And your first question comes from the line of Michael Okunewitch with Maxine Gook. Your line is open. Hey there. Thank you so much for taking my questions today. Congratulations on all the progress.
Speaker Change: And your first question comes from the line of Michael <unk> with Maxim Group. Your line is open.
Michael <unk>: Hi, there. Thank you so much for taking my questions today, congratulations on all the progress.
Ramesh Ramachandran: Ramachandran. Thank you.
Speaker Change: Thank you.
Speaker Change: Yes.
Huma Qamar: So I think just, first off, in terms of the limelight study, where are you at enrollment and when do you have to complete that to reach your filing target? Good morning.
Speaker Change: So I think just.
Speaker Change: First off in terms of.
Speaker Change: The limelight study where are you in enrollment and when do you hope to complete that to reach their filing targets.
Speaker Change: Hi, Good morning Defense home <unk> kitchen. Thanks for your question so for our nine like our Q4 hundred tier one phase III trial.
Huma Qamar: This is Huma Qamar, SEMO, Ocugen. Thanks for your question. So for our NIMELIGHT OCU400 301 Phase 3 trial, we are on track for enrollment as you know planned for first half of 2025 and we will be on track for our BLA submission a year from now in terms of clinical.
Speaker Change: We are on track for enrollment and.
Speaker Change: Planned for first half of 2025, and we will be on track for our BLA submission.
Speaker Change: From now in terms of clinical thank.
Operator: Thank you.
Speaker Change: Thank you.
Speaker Change: Alright.
Speaker Change: Thanks.
Shankar Musunuri: As we approach the completion of that first pivotal study, I'd like to see if you could just expand a little bit on manufacturing capabilities and then what you would have to build out and expand to support the filing and commercialization stage of this product. Yeah, Michael, I'll answer the first part and our CSO, Dr. Arun Upadhyay is on the call too.
Speaker Change: And so just as we approach the completion of that first pivotal study I'd like to see if you could just expand a little bit odd.
Speaker Change: Manufacturing capabilities, and then what you would have to build out and expand to support the filing and commercialization stage of this product.
Speaker Change: Yes, Michael ill answer the first part then our CSO Dr. <unk> on the call too you can explain what our manufacturing plants are.
Arun Upadhyay: He can explain what our manufacturing plans are. So for BLE submission, we have to complete process validations at the commercial scale, and which we are on target this year. And so that's what is needed. And obviously, we have plenty of capacity for commercial launch of this product, XUS.
Speaker Change: So for BLA submission, we have to complete process validation at commercial scale and we are on target this year.
Speaker Change: And so that's what is needed and obviously, we have plenty of capacity for a commercial launch of this product.
Shankar Musunuri: We also built our own facilities here in Malvern, Pennsylvania. Once we commercially launch the product in 2027, shortly after that, within two years, our goal is to get the second site in the US, you know, get it ready for launching our commercial supplies.
Speaker Change: We also built our own facilities here in Malvern, Pennsylvania, once we commercially launch the product in 2027.
Speaker Change: Shortly after that within two years, our goal is to get the second site in the U S.
Speaker Change: And I'll get it ready for launching our commercial suppliers want to go into more details on this year's plans.
Arun Upadhyay: I don't want to go into more details on this year's plans. Yeah, thanks, Sankar. So, as you stated, we are on track to complete the process validation activity and get ready for BLS submission next year. And for initial commercial launch, we'll be using our partner, InsignioBio, for Ocu 400 project. But our plan is, like, subsequently, we are going to bring back the manufacturing. We'll do the tech transfer and bring the manufacturing in-house in our Melbourne GMP manufacturing facility, which we have completed construction last year. And subsequently, we plan to release the product from our second site in the U.S.
Speaker Change: Yes. So as you stated we are on track to complete the process valuables and do a deal.
Speaker Change: Get ready for BLA submission.
Speaker Change: Last year.
Speaker Change: Polysilicon was the launch will be using our Butler <unk>.
Speaker Change: Colorado 400 projects with <unk>.
Speaker Change: Our plan in light subsequently, we are going to bring back the manufacturing transfer and bring the manufacturing in house in our Melbourne GOP wins has been because of the deep rich.
Speaker Change: When should we have.
Speaker Change: Completed construction plus deal.
Speaker Change: And subsequently we plan to release the product almost <unk> site to the U S. So thats held steady.
Operator: So, that's our strategy to support the future commercialization. All right. Thank you.
Speaker Change: <unk>.
Speaker Change: Alright.
Operator: And then just one more from me and I'll hop back into the queue. Could you give us an idea of when we could expect to... See the next update on the OCU 200 program. I know it's not core, but DME has been an exciting space. Yeah, I think the clinical trial we're planning to complete later part of this year. So our goal is before the end of the year, we'll provide clinical update, including preliminary efficacy and safety. All right.
Speaker Change: Thank you and then just one more from me and I'll hop back into the queue could you give us an idea of when we could expect to.
Speaker Change: See the next update on the ocular 200 program I know its not core, but <unk> has been an exciting space.
Speaker Change: Yes, I think the clinical trial, we are planning to complete later part of this year.
Speaker Change: So our goal is.
Speaker Change: Before the end of the year will provide a clinical update including preliminary efficacy and safety.
Speaker Change: Alright. Thank you so much and once again congrats on all the progress.
Operator: Thank you so much. And once again, congrats on all the progress. Thank you.
Speaker Change: Thank you.
Robert Leboyer: Next question comes from the line of Robert LeBoyer with Nobel. Your line is open. Good morning, everybody.
Robert: Next question comes from the line of Robert <unk> with Nomura. Your line is open.
Robert: Good morning, everybody.
Shankar Musunuri: I had a Some of the prepared remarks regarding Europe and the regulations. My understanding was that These remarks are for... Prasad, yer-mear-or-tien es-pee, and the clinical trial going on. Am I correct on that?
Robert: Hello.
Robert: Clarification on.
Speaker Change: Some of the prepared remarks regarding Europe and the regulation there.
Robert: My understanding was that.
Robert: These remarks referred to <unk>.
Robert: <unk> S P.
Robert: And the clinical trial going on Mike.
Robert: Correct on that.
Shankar Musunuri: So the, no, your question, Robert, related to FortinST, yeah, I mean, the morning we actually talked. during this. We have a recent communication from EMA on RQ400, just to clarify. And so for submission of the MAA, and that's the path, and it's going well. That's what we talked about. As far as RQ410 ST is concerned, the clinical trial we designed with FDA, which will be a registration trial, which we are going to embark on it shortly, and we are discussing that strategy with EMA. We don't have the final answer yet. And obviously, since this is a much-needed product, significant unmet medical need, just as RT, Stargardt disease, there's no approved treatment in the EU.
Robert: So the your.
Robert: Question Robert related to Fortinet SD.
Robert: Yes, I am in the morning, we actually talked.
Robert: During this we have recent communications from EMA on occupy 100 just to clarify.
Robert: And so before submission of the MAA and thus the pad.
Robert: It's going well, that's what we've talked about as far as our Q4 10 Sds concern that's true.
Robert: The clinical trial design with the FDA, which will be a registration trial, which we are going to embark on it.
Robert: And shortly and we are discussing that strategy with DMA, we don't have the final answer it and obviously.
Robert: Since this is a much needed product significant unmet medical need just as R&D startup disease. The mercury treatment in the EU, we are hoping they will align with FDA.
Shankar Musunuri: We are hoping they will align with FDA clinical trial design and also agree. Again, we are in negotiations with EMA. When we have an answer from them, we'll let the markets know. Okay, great.
Robert: Clinical trial design and also agreed again, we are in negotiations with DMA. Then we have an answer from them, we'll let the market now.
Robert: Okay great.
Operator: And just separately, you had mentioned the influenza trial with NIAID.
Robert: And just separately you had mentioned the influenza trial with NIAID and considering what's been going on with.
Shankar Musunuri: Ramachandran, Arun Upadhyay, Swayampakula Ramachandran, Arun Upadhyay, Yu He, Swayampakula Ramachandran, the NIH in the past two or three months, and the overhaul going on at those agencies. Uh, have you had any... , and share any changes in the relationship or with contact of people or any insights you can give as to what the agency is thinking going forward. I know that some of the companies that were developing COVID vaccines I was wondering if you could give us any insight as to what's going on in the divisions that you interact with. Yes, Robert, great question. This is related to our COVID-19 pandemic.
Robert: HHS and the FDA.
Robert: And the NIH in the past two or three months.
Robert: The overhaul going on with those agencies.
Robert: Have you had any.
Robert: <unk>.
Speaker Change: Changes in the relationship or with contact with people or any insights you can give as to what the agency is thinking going forward.
Speaker Change: I know that some of the companies that we're developing COVID-19 vaccines.
Speaker Change: Grants revoked and cancel so I was wondering if you could give.
Speaker Change: Give us any insight as to what's going on in the divisions that you interact with.
Robert: Yes, Robert Great question.
Speaker Change: This is related to our code.
Arun Upadhyay: vaccine targeting intranasal versus inhalation routes. This clinical trial, NIAID is still on target to support it. In fact, I will ask Dr. Upadhyay, our CSO, to comment more on this. He is in direct contact with NIAID. Go ahead.
Speaker Change: Vaccine targeting intranasal versus emulation routes of this clinical trial is still on target to support it in fact.
Speaker Change: The Alaska <unk>, our CSO to comment more on this in direct contact with the money go ahead and get some goods yes.
Arun Upadhyay: Thank you, Shankar. Yes, I think we don't see any impact on us related to the changes you just mentioned happening at the government end. So, we are constantly interacting with NIAID, and we are collaboratively working with them to finalize the clinical study plan, as well as supporting the required material to initiate the phase one study. So, we are on track, and as Shankar mentioned, we are hoping to initiate the phase one study in the second quarter of this year, and we are on target for that. And our relationship has been great, I would say. I think it has improved even, actually.
Speaker Change: Adding to we don't see any impact on us related to the Tianjin you just mentioned.
Speaker Change: <unk> and.
Speaker Change: So we are constantly interacting with <unk> and work collaboratively with them to finalize.
Speaker Change: The clinical study plan as realized no supporting us.
Speaker Change: The required material to initiate the phase of the study. So we are on track and as Sean mentioned, we are hoping to initiate the phase one study in second quarter. This year.
Speaker Change: And we are on target for that.
Speaker Change: And our relationship had been de balance I think it has.
<unk> has increased significantly.
Operator: Our engagement with NAD has increased significantly to initiate the study. Okay, great.
Speaker Change: <unk> initiated the study.
Speaker Change: Okay, great. Thank you very much.
Swayampakula Ramakanth: Next question comes from the land of Swayampakula Ramakanth with A.C. Wainwright. Your line is open. Thank you. Good morning Shankar and team. A couple of quick questions on the limelight study.
Speaker Change: Next question comes from the line of Slam tackled all around the world with HC Wainwright.
Speaker Change: Your line is open.
Speaker Change: Thank you.
Speaker Change: Good morning, Schon current team.
Speaker Change: Couple of quick questions on the limelight.
Speaker Change: Sure.
Huma Qamar: Does the Phase III study have an interim look, and if so, what's the general timing that we should expect that to be? Hello, this is Huma. Thanks for the question. There is no interim look in the study. And once the enrollment is complete and we'll be giving periodic updates, there are safety for 30 subjects each that we are following for the landline study. However, the full data will be available once the CSR is final. As this is a blinded study, we cannot release the data before yet.
Speaker Change: Does that does a phase III study have an interim look and if so what sports.
Speaker Change: General timing that we should expect that to me.
Speaker Change: Yes.
Speaker Change: This is <unk>. Thanks for the question did April interim look in the study and once the enrollment is complete and is becoming periodic updates that I got.
Speaker Change: Safety for our 30 subjects each.
Speaker Change: <unk> filing for drilling.
Speaker Change: However, the core data would be available.
Speaker Change: Once the CSR is final.
Speaker Change: And this is a blinded study we cannot release the data beforehand.
Operator: Thank you. Thank you for that, Huma.
Speaker Change: Thank you.
Speaker Change: Thank you for that.
Operator: A quick clarification on the design. How didrift happen? By superposition. You don't have to.
Speaker Change: A quick clarification on the design.
Speaker Change: Because I was.
Speaker Change: I admit.
Speaker Change: Smith heard it.
Speaker Change: I thought the randomization for the Limelight study was to is to run but.
Speaker Change: Yes.
Speaker Change: Okay.
Speaker Change: One is to one.
Operator: Have does it come in? Okay.
Operator: So, in Earth Light Share Shankar's comments, but probably about it.
Speaker Change: Cars com comments by probably a volatile.
Speaker Change: Then on the on the market and just a clarification. The one is two one is to one is for the currently ongoing.
Shankar Musunuri: So RK, just a clarification, the 1 is to 1 is to 1 is for the currently ongoing clinical trial for RQ410 targeting dry age-related macular degeneration. So they have a medium dose, high dose and control in that phase two.
Speaker Change: Clinical trial for <unk> targeting dry age related macular degeneration.
So they have a medium dose idose and control in the phase two.
Speaker Change: Perfect perfect.
Shankar Musunuri: One last question from me is, on the three modified gene therapy programs, what clinical data updates would we see in 2025, and I'm just trying to see if we can get any idea of... Thank you. How strong the data is going to look in all of the pivotal programs as we move forward.
Speaker Change: One last one last question from me is.
Speaker Change: On the train modified gene therapy programs.
Speaker Change: What <unk>.
Speaker Change: Clinical data updates would we see in 2025.
Speaker Change: And just kind of saying if we can get to any idea of.
Speaker Change: How strong the data is going to look.
Speaker Change: All of the pivotal programs as they move forward.
Shankar Musunuri: I think from RQ400 perspective, as Huma stated, it's a blinded phase 3 trial. You'll get periodic updates of how the trial is progressing. Other than that, the data won't come out until next year, until the trial is completely done. The second program, RQ410, our model trial for dry AMD, and we are expecting interim results sometime this fall. And after 4-10, SD, obviously the registration trial is going to start shortly, in the next couple of months. We are going to provide, when you have a complete, in our 12-month data, from Phase 1 on this study. And in addition, up to 200, the phase one trial should be complete late this year and we'll provide the data from the trial too.
Speaker Change: I think from argue 400 perspective as humans stated, it's a blinded phase III trials, you'll get periodic updates are.
Speaker Change: Now the trial is progressing other than that they don't come out until next year until the trial is completely done the second program <unk>.
Speaker Change: Our motto trial for dry AMD, and we are expecting interim results sometime this fall.
Speaker Change: And occupy 10 SD obviously, the registration trial is going to start shortly in the next couple of months.
Speaker Change: Now we are going to provide when you have a complete and at 12 months data from phase one on the 30th.
Speaker Change: And in addition.
Speaker Change: Our Q2 hundred the phase one trial should be complete later this year and will provide the data from the trial.
Operator: Perfect. Thanks. Thanks for taking all my questions. Thank you.
Perfect.
Speaker Change: Thanks for taking all my questions.
Speaker Change: Thank you.
Daniil Gataulin: Next question comes from the line of Daniil Gataulin, Richardin. Your line is open. Good morning.
Speaker Change: Next question comes from the line of Danielle <unk> with Chardan. Your line is open.
Speaker Change: Okay.
Speaker Change: Good morning. This is stable on for Danielle Thanks for taking my question.
Stephen: This is Stephen. I'm for Daniil. Thanks for taking my question. For OCU400 for RP to file in Europe, do any of the patients need to be treated in Europe, or is the data from the U.S. and Canada going to be sufficient?
Speaker Change: For our Q4 hundred for RP to file in Europe do you any of the patients to be treated in Europe or is the data from the U S and Canada.
Speaker Change: So I'll take that question. That's a great question actually so as we have stated in the past as well.
Shankar Musunuri: So, I'll take that question. That's a great question, actually. So, as we have stated in the past as well, that no additional trial is required in Europe. If we get the approval in the U.S. based on, you know, our primary endpoint and the study design, U.S. and Canada is sufficient. In fact, U.S. is sufficient for our approval in Europe. So, no additional trials are required. This trial has sufficient representation for global mutations, and it's the only broad ARPA-indication gene agnostic trial globally.
Speaker Change: No additional trial is required in Europe.
Speaker Change: If we get the approval in the U S based on.
Speaker Change: Our primary endpoint and the study design.
Speaker Change: And Canada is sufficient in fact use is sufficient for.
Speaker Change: Our approvals in Europe. So no additional trials are required this trend has sufficient representation.
Speaker Change: Global's mutations as it's the only drug RMP indication agnostic trial competition.
Operator: Great, thank you.
Great. Thank you.
Operator: This concludes the Q&A portion.
Speaker Change: This concludes the Q&A portion I will now turn the call back over to chairman and CEO and cofounder question time will generally.
Shankar Musunuri: I will now turn the call back over to Chairman, CEO, and Co-Founder, Dr. Shankar Musunuri. Thank you, operator. Our efforts in the first quarter of the year evidence the importance of our gene therapy programs and the significance of upcoming milestones. We remain steadfast in our mission to provide a one-time therapy for life to address considerable unmet medical needs that exist for millions of patients facing the terrifying prospect of losing their vision, and look forward to what this quarter and the rest of the year holds for the company, our people, patients, and shareholders. Thank you. Thanks again, everyone.
Speaker Change: Please go ahead.
Speaker Change: Thank you operator.
Speaker Change: Our efforts in the first quarter of the year evidenced the importance of our gene therapy programs and the significance of upcoming milestones.
Speaker Change: We remain steadfast in our mission to provide <unk> therapy for life to address considerable unmet medical needs that exist for millions of patients facing the terrifying prospect of losing their vision and look forward to work this quarter and the rest of the year holds for the company our people patients and shareholders. Thank you.
Speaker Change: Thanks again, everyone Goodbye.
Operator: Goodbye.
Operator: This concludes today's conference call. Thank you all for joining and you may now disconnect.
Speaker Change: This concludes today's conference call. Thank you all for joining and you may now disconnect.
Speaker Change: [music].