Q1 2024 Ocugen Inc Earnings Call & Business Update
Operator: Good morning and welcome to Ocugen's first quarter 2024 financial results and business update. Please note that this call is being recorded at this time. All participant lines are in listen only mode. Following the speaker's commentary, there will be a question and answer session. I will now turn the call over to Tiffany Hamilton, Ocugen's Head of Corporate Communications. You may begin.
Good morning, and welcome to Aki Gents first quarter 'twenty 'twenty four financial results and business update. Please note that this call is being recorded at this time all participant lines are in listen only mode. Following the speaker's commentary there will be a question and answer session I will now turn to.
The call over to Stephanie Hamilton oxygen <unk> head of corporate Communications you may begin.
Tiffany J. Hamilton: Thank you, operator. And good morning, everyone.
Doctor shrunk: Thank you operator, and good morning, everyone. Joining me on today's call and webcast with Doctor shrunk our listener Archie just chairman CEO and co founder who will provide a business update and an overview of our clinical and operational progress.
Tiffany J. 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 an overview of our clinical and operational progress. Michael Breininger, our corporate controller, is also on the call to provide a financial update for the quarter ended March 31st, 2024.
Doctor shrunk: Michael Breininger, our corporate controller is also on the call to provide a financial update for the quarter ended March 31.
Doctor shrunk: 2020 for Dr.
Speaker Change: Dr. Arun <unk> <unk>, Chief Scientific Officer head of research and development and Dr. Hua Kumar Chief Medical Officer will be available to answer questions. Following the presentation.
Tiffany J. Hamilton: Dr. Arun Upadhyay, Chief Scientific Officer, Head of Research and Development, and Dr. Huma Qamar, 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 2024. We encourage listeners to review the press release, which is available on our website at ocugen.com. This call is being recorded, and a replay with the accompanying slide presentation will be available in the investor section of Ocugen's website for approximately 45 days.
Michael Breininger: This morning, we issued a press release detailing associated business and operational highlights for the first quarter of 'twenty 'twenty four we encourage listeners to review the press release, which is available on our website at <unk> Dot com.
Speaker Change: This call is being recorded and a replay with the accompanying slide presentation will be available on the investors section of the occupant web site for approximately 45 days.
Tiffany J. Hamilton: 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 predicts, believes, potential, proposed, continue, estimates, anticipates, expects, plans, intends, 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.
Occupant: 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 projects believe potential proposed continue estimate anticipates expects plans info.
Speaker Change: And maybe could might well 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 timelines.
Tiffany J. Hamilton: Such statements are subject to numerous important factors, risks, and uncertainties, which may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission, the SEC, including the risk factors described in the section entitled Risk Factors in the annual reports that we file with the SEC. Any forward-looking statements that we make in this presentation speak only as of the date of this presentation.
SEC: Such statements are subject to numerous important factors risks and uncertainties that may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission the SEC, including the risk factors described in the section entitled risk factors.
SEC: Actors in the annual reports that we file with the SEC.
SEC: Any forward looking statements that we make in this presentation speak only as of the date of the presentation.
Tiffany J. Hamilton: Except as required by law, we assume no obligation to update forward-looking statements contained in this presentation, whether as a result of new information, future events, or otherwise after the date of this presentation. Finally, Ocugen's quarterly report, Form 10-Q, covering the first quarter of 2024, has been filed. I will now turn the call over to Dr. Musunuri.
SEC: Sept 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.
Occupants: Finally occupants quarterly report Form 10-Q, covering the first quarter of 2024 has been filed.
Occupants: I will now turn the call over to Dr Missionary.
Shankar 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. By using nuclear hormone receptors to restore homeostasis in the retina, modifier gene therapy has the potential to address multiple inherited retinal diseases, as well as larger multifactorial diseases such as dry age-related macrodegeneration through a gene-agnostic approach. Just after the close of the first quarter of this year, we announced that the FDA cleared our IND application for the RQ400 Phase III Limelight clinical trial, and the EMA provided acceptability of the U.S.-based trial for submission of a market authorization application. I will discuss the significance of these milestones and the potential for RQ400 in greater depth later in the presentation.
Dr Missionary: 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.
Dr Missionary: Game changing Marty for a gene therapy platform.
Dr Missionary: But using nuclear hormone receptors to restore homeostasis in the retinal Martin for gene therapy has the potential to address multiple inherited retinal diseases.
Occupants: As well as diseases.
Occupants: Roger.
Roger: Factor in diseases, such as dry age related macular degeneration.
Roger: Gene agnostic approach.
Roger: Just after the close of the first quarter of this year, we announced that the FDA cleared our A&D obligation from our Q4 hundred phase III limelight clinical trial and the email provider acceptability of the U S based trial for submission after market authorization application.
Roger: I will discuss the significance of these milestones and the potential for our Q4 hundred in greater depth later in the presentation.
Shankar Musunuri: RQ-410 and RQ-410ST are now in Phase I-II clinical trials, targeting geographic atrophy, secondary to dry-age-related macular degeneration, and stargardt disease, respectively, with clinical updates expected in the third quarter of 2024. Our cell therapy platform is poised to advance with the renovations to our existing facility completed earlier this year. This gives Ocugen the capability to support neocort autologous cell therapy, manufacturing for personalized phase 3, clinical material contingent upon adequate funding. Finally, RQ500 will soon advance into a Phase I clinical trial via the National Institute of Allergy and Infectious Diseases, NIAID, sponsored trial comparing the administration of RQ500 via two different mucosal rafts, emulation into the lungs, and as We're confident that 2024 will be a bellwether year for Ocugen.
Roger: Our Q4, Kim and Archie potent SD are now in phase one two clinical trials.
Roger: Targeting geographic atrophy secondary to dry age related macular degeneration and star got disease, respectively, with a clinical update expected in the third quarter will be 24.
Roger: Our cell therapy platform is poised to advance with the renovations to our existing facility completed earlier this year, the skus acquisition the capability to support Neocart.
Speaker Change: Talking about the cell therapy manufacturing for personalized history.
Speaker Change: Clinical material contingent illiquid funding.
Speaker Change: Finally, our Q4 hundred soon.
Speaker Change: Into phase one clinical trial.
Speaker Change: The National Institute of allergy and infectious diseases, and my R&D sponsored trial.
Speaker Change: During the administration of our coupons.
Speaker Change: The two different vehicles and drones emulation into the lungs.
Speaker Change: And as a nasal spray.
Speaker Change: With three gene therapy candidates to treat blindness diseases in the clinic in a phase III ready cell therapy candidate. We are confident that 2024 will be a bellwether oxygen.
Shankar Musunuri: We are thrilled to share significant advancements in our leading modified gene therapy candidate, ARCU-400, as it makes remarkable strides in clinical development. The green light from the FDA to begin the Phase 3 clinical trial marks a pivotal milestone as ARCU-400 becomes the first gene therapy to progress to Phase 3 trials with such a broad retinitis pigmentosa indication. Until now, there has been only one marketed product to treat one of the 100 gene mutations associated with RP.
Speaker Change: We are thrilled to share significant advancement in our leading modified gene therapy candidate Al Q4 hundred as it makes the remarkable strides and political donors.
Speaker Change: The Green light from the FDA to begin phase III clinical trial marks a pivotal milestone as occupy 100 becomes the first gene therapy program to phase III trials.
Speaker Change: Such a broad pigmentosa indication.
unknown: Until now there has been only one market product to treat one of the hundred gene mutations associated with workday.
Shankar Musunuri: Now there is real hope for all RP patients who haven't had a treatment option. Our completion of enrollment and dosing in the Phase 1-2 trial demonstrated promising safety and efficacy across various genetic mutations and dosage levels, paving the way for phase 3 clinical trials. ARCQ-400 has already received key regulatory approvals, including orphan drug, regenerative medicine advanced therapy, and orphan medicinal product designations from both FDA and the European Commission for treating Inherited Retinal Diseases.
RP patients: Now there is real hope for RP patients, who haven't had a treatment option.
Speaker Change: Our completion of enrollment and dosing in phase one two trial demonstrated promising safety and efficacy.
Speaker Change: Across various genetic mutations and dosage levels.
Speaker Change: Getting to the four phase III clinical trial.
Speaker Change: Our Q4 hundred has already received key regulatory approvals, including orphan Brian region, Redo medicine advanced therapy, and orphan medicinal product designation from both FDA and European Commission.
Speaker Change: For treating it as a driven diseases.
Shankar Musunuri: These endorsements highlight Acu400's broad therapeutic potential, and we remain on track to meet 2026 BLA and MAA approval targets. We expect to begin dosing patients in the Phase 3 Limelight clinical trial in the second quarter of 2024, which will include 150 subjects across two arms, one with patients affected by a mutation and one arm that is gene agnostic. Luminance Dependent Navigation Assessment, LDNA, the primary endpoint for the study, focuses on the proportion of responders in treated and untreated control groups, achieving an improvement of at least two lux levels from baseline and the study act. The secondary endpoint will be measured by changing low luminescence.
Speaker Change: These endorsements highlight our Q4 hundreds broad therapeutic potential and we remain on track to meet 2026, BLA and MAA approval targets.
Speaker Change: We expect to begin dosing patients in the phase III limelight clinical trial in the second quarter of 'twenty 'twenty four which will include 150 subjects across two arms, one with patients affected by <unk> mutation and one that is agnostic.
Speaker Change: Luminescence dependent navigation assessment L. D. M. P. The primary endpoint for this study focuses on the proportion of responders and treated.
Speaker Change: Untreated control groups, achieving an improvement of at least two looks now.
Speaker Change: From baseline to study yes.
Speaker Change: The secondary endpoint will be measured by changing luminescence.
Shankar Musunuri: Visual Equity score of 0.3 log more from the baseline. The effectiveness of the treatment will be compared to an untreated control group for the secondary endpoint as well. Additionally, leveraging a dual track strategy, we plan to expand the Phase III trial in the later half of 2024 to include patients with Leber Congenital Hemorrhagic LCA contingent on favorable results from Phase I. Let me take a moment to discuss the unmet need and underserved market for RP and LCA patients.
Speaker Change: As you'll equity score of point to a lot more from the baseline.
Thank goodness: Thank goodness of the treatment will be compared to entry.
Speaker Change: Untreated control group for the secondary endpoint as well.
Speaker Change: Additionally, leveraging a dual track strategy, we plan to expand the phase II trial, and the labor half of 'twenty.
Speaker Change: 24 to include patients with Leber congenital amaurosis LCA contingent.
Speaker Change: Favorable results from Phase two study.
Shankar Musunuri: An estimated 1.6 million people globally are affected by RP and LCA combined. In the U.S. and Europe alone, our initial target markets, there are nearly 300,000 total patients. RP and LCA are classified as inherited retinal diseases from a group of heterogeneous disorders that affect the retina. These conditions frequently result in visual impairment and ultimately blindness.
Speaker Change: Let me take a moment to discuss the unmet need.
Speaker Change: Underserved market for RP and LCA patients an estimated $1 6 million people globally are afflicted by RP and don't see a combined in the U S and Europe alone. Our initial target markets. There are nearly 300000 total patients.
Deutsche Bank: <unk> are classified as inherited retinal diseases from a group of neutrogena disorders that affect of Deutsche Bank.
Deutsche Bank: These conditions frequently results in visual impairment and ultimately blindness.
Shankar Musunuri: By establishing homeostasis in patients affected by these diseases, our aim is to preserve or improve vision. By actively sharing our insights at pertinent medical conferences and engaging with advocacy groups, we strive to raise awareness of the desperate need to find a solution for all RP and LCA patients, as well as the potential of modified gene therapy to revolutionize treatment. To better understand the experience of one of our Phase I-II clinical trial patients, I encourage you to watch his video in the patient section of our website.
Deutsche Bank: By establishing homeostasis in patients affected by these diseases. Our aim is to preserve or improve vision, we're actively sharing our insights.
Deutsche Bank: Medical conferences, and engaging with advocacy groups with straight to raise awareness of the desperate need to find a solution.
Deutsche Bank: RP and LCA patients as well as potential of Marty for gene therapy to revolutionize treatment.
Deutsche Bank: Better understand the experience of one of our phase one two clinical trial patients I encourage you to watch is video and the patients section of our website.
Shankar Musunuri: Moving on to our development in the treatment of geographic atrophy, secondary to dry age-related macular degeneration, D-AMD, and Stargardt disease with our RQ-410 and RQ-410-ST programs. These modified gene therapies leverage the nuclear receptor gene, RER-related orphan receptor A, RORA, aiming to provide a potential one-time therapy for life with a single subretinal injection.
Speaker Change: Moving onto our development in the treatment of geographic atrophy secondary to dry age related macular degeneration, the AMD and stronger disease without our Q4, and our Q4 10 programs. These modified gene therapies leveraged a nuclear receptor gene rel.
Speaker Change: Weighted orphan receptor eight draw aiming to provide a potential one time therapy for life with a single sub retinal injection.
Shankar Musunuri: OCU410, specifically designed to address multiple pathways implicated in the pathogenesis of DAMD, offers a distinct advantage over current treatments that target only one cause of DGA and often require multiple injections per year, accompanied by various safety concerns. Our approach with Octu410 is to provide a comprehensive and durable solution, a potential one-time therapy for life. Dry AMD affects about 19 million people in the U.S. and Europe combined, while GA affects about 2 to 3 million people in the U.S. and Europe, a significant market opportunity.
Speaker Change: Our Q4 10, specifically designed to address multiple pathways implicated in the pathogenesis of the AMD offers a distinct advantage or current treatment. So the target only one cause of GE and often required multiple injections per year accompanied by various safety concerns.
Speaker Change: Our approach with our Q4 10 is to provide a comprehensive and durable solution.
Speaker Change: Potential one time therapy for life.
Speaker Change: Dry AMD FX about 19 million people in the U S and Europe combined while FX about two to 3 million people in the U S and Europe, a significant market opportunity.
Shankar Musunuri: In December 2023, we began the Phase I-II Armada clinical trial for Octu410. Considerable progress has been made with the completion of dosing in both the first and second cohort, low and medium doses. Following the successful dosing in this cohort, the Data and Safety Monitoring Board, DSMB, will convene later this month to evaluate proceeding with the high-dose cohort in the ongoing dose escalation phase of the study.
Speaker Change: In December 'twenty, two 'twenty three we began the phase one two our model clinical trial for <unk> for Ken.
Ken: Considerable progress has been made with the completion of dosing in both the first and second cohort low and medium doses.
SMB: Following the successful dosing in this cohort the data and safety monitoring board. The SMB will convene later this month to evaluate.
SMB: Proceeding with the high dose cohort in the ongoing dose escalation study.
Shankar Musunuri: After completion of the third cohort, we will transition into phase two of the clinical trial, the expansion phase. A key upcoming event for this trial is a clinical update, which is anticipated in the third quarter of 2024. This data will provide initial insights into the safety and efficacy of Octu410. RQ410 The Phase 1 study is multi-centered and open-label, focusing on dose ranging, while Phase 2 is randomized, aiming to expand our understanding of the drugs' efficacy and safety compared to a controlled, untreated arm, with patients randomized in a 1-to-1-to-1 ratio across two dosage groups and one control group.
Brian: After completion of the third cohort will transition into phase two clinical Brian the expansion phase.
SMB: A key upcoming even for this trial is a clinical update which is anticipated in the third quarter of 2024.
SMB: This data will provide initial insights into the safety and efficacy of our Q4 Tim.
SMB: Our Q4 10 phase one study is multi center open label focusing on dose ranging while phase II is randomized aiming to expand our understanding of Brooks efficacy safety comfort air control untreated arm.
unknown: With patients randomized in a one to one to one ratio across two dosage groups and one control group.
Shankar Musunuri: Participants must be aged 50 or older, have a best corrected visual equity of approximately 24 letters or more using the ETDRS chart, and have a total geographic atrophy area between 2.5 and 20.5 millimeters squared. We now turn to Ocuportin ST, which received orphan drug designation from FDA for the treatment of ABCA4-associated retinopathies, including Stargardt disease. Stargardt affects approximately 100,000 people in the U.
SMB: Participants must be aged 50, and older and with best corrected visual equity of approximately 24 letters or more using the 80 Drs chart and habit total geographic atrophy area between two five and 25 millimeter square.
Tennessee: We now turn to our Q4, Tennessee, which received orphan drug designation from FDA for the treatment of <unk> associated retinopathy, including startup disease <unk>.
Speaker Change: <unk> affects approximately 100000 people in the U S and EU combined.
Shankar Musunuri: This modified gene therapy candidate also utilizes the RORA gene, and the Phase 1-2 Guardian trial for Stargardt disease is actively enrolling patients. We have completed dosing in the first cohort, the low dose. In April 2024, the Data Safety and Monitoring Board approved the continuation of the medium-dose phase, which we expect will be completed this month. A clinical trial update for RQ-410ST is also anticipated in the third quarter of 2024. The Phase 1-2 trial involves up to 42 participants, 30 adults and 12 children, who exhibit mild to moderate disease symptoms.
Guardian trial: This modest where gene therapy candidate also utilizes the regime and the fish want to Guardian trial will target disease is actively enrolling patients we have completed dosing in the first cohort logos.
Tennessee: In April 2024, the data safety monitoring board approved the continuation to the medium dose switch, which we expect will be completed this month.
Tennessee: Clinical trial update for Q4, Tennessee is also anticipated in the third quarter of 2024.
Tennessee: The phase one two trial enrolled up to 42 participants 30 hurdles and 12 children, which exhibit mild to moderate disease symptoms. The study uses a three plus three dose escalation design in phase one segmenting subjects into low medium and high dose cohorts.
Shankar Musunuri: The study uses a 3 plus 3 dose escalation design in Phase 1, segmenting subjects into low, medium, and high dose cohorts. Following the dose escalation phase, the trial will transition into phase two, the expansion phase. This expansion phase will use a one-to-one-to-one design to randomize subjects into two different treatment groups at varying dose levels or a controlled, untreated group, allowing for a comprehensive assessment of treatment efficacy across different dosages. Our work across OCU400, OCU410, and OCU410ST affirms our enduring commitment to the success of these modified gene therapies for the benefit of patients faced with the terrible prospect of losing their
Speaker Change: Following the dose escalation phase I trial will transition into phase II the expansion phase.
The study: This expansion phase will use it one to one to one designed to randomize subjects into two different treatment groups at varying dose levels are the control untreated group are living for a comprehensive assessment of treatment efficacy across different dosages.
Our work: Our work across our Q4 hundred Q4, 10, and octave for Tennessee from our enduring commitment to the success of this modified gene therapies, but the benefit of patients faced with the terrible prospect of losing.
Our work: Site work.
Shankar Musunuri: We are encouraged by the progress in these trials and look forward to sharing further updates as we reach critical milestones and get closer to addressing substantial unmet medical needs. With that, I will now turn the call over to our Corporate Controller to provide an update on our financial results for the first quarter ended March 31st, 2024.
Our work: Encouraged by the progress in these trials and look forward to sharing further updates as we reach critical milestones and get closer to addressing substantial unmet medical needs.
Corporate Controller: With that I'll now turn the call over to our corporate controller to provide an update on our financial results for the first quarter ended March 31, 2020 for Mike.
Michael Breininger: Thank you, Shankar. Our research and development expenses for the quarter ended March 31, 2024 were $6.8 million, compared to $10.2 million for the first quarter of 2023. General and administrative expenses for the quarter ended March 31, 2024 were $6.4 million, compared to $8.3 million during the same period in 2023. The net loss was approximately $11.9 million, or $0.05 net loss per share, for the quarter ended March 31, 2024, compared to a net loss of approximately $17.3 million, or $0.08 net loss per share, for the first quarter of 2023.
Corporate Controller: Thank you Schenker, a research and development expenses for the quarter ended March 31, 2024, or $6 $8 million compared to $10 2 million for the first quarter of 2023.
Schenker: General and administrative expenses for the quarter ended March 31, 2024 were $6 4 million compared to $8 3 million during.
Corporate Controller: During the same period in 2023.
Schenker: Net loss was approximately $11 9 million or <unk>.
Schenker: Net loss per share for the quarter ended March 31, 2024, compared to a net loss of approximately $17 3 million or <unk> <unk> net loss per share for the first quarter of 2023.
Michael Breininger: Our cash and cash equivalents totaled $26.4 million as of March 31st, 2024, compared to $39.5 million as of December 31st, 2023. As always, we are constantly exploring strategic and shareholder-friendly opportunities to increase our working capital. That concludes my update for the quarter. Tiffany, back to you.
Michael Breininger: Cash and cash equivalents totaled $26 4 million as of March 31, 2024, compared to $39 5 million as of December 31, 2023.
Tiffany: As always we are constantly exploring strategic and shareholder friendly opportunities to increase our working capital.
Tiffany J. Hamilton: That concludes my update for the quarter Stephanie back to you.
Tiffany J. Hamilton: Thank you, Mike. We will now open the call for questions. Operator?
Tiffany: Thank you, Mike we will now open the call for questions operator.
Operator: Thank you. We will now begin the question and answer session. If you have dialed in and would like to ask a question, please press star 1 on your telephone keypad to raise your hand and join the queue. If you would like to withdraw your question, simply press star 1 again. 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 queue. When your first question comes from the line of Arthur He with H.C. Wainwright, please go ahead.
Operator: Thank you we will now begin the question and answer session. If you have dialed in and would like to ask a question. Please press star one on your telephone keypad to raise your hand in China.
Tiffany J. Hamilton: If you would like to withdraw your question simply press Star. One again, if you are called upon to ask your question and are listening by a loud speaker on your device. Please pick up the handset and ensure that your phone is not on mute when asking a question again press star one to join the queue and your first question comes from the line of Arthur He with H.
Arthur He: C. Wainright. Please go ahead.
Arthur He: Hey, good morning, Sean Kennedy, congrats on the progress. So I just had a quick one regarding the 453 study. Rick, for the gene agnostic arm, is there a minimum number of patients for each specific gene mutation too to meet the requirement for the BLS application?
Arthur He: Hey, good morning, Shanghai and team congrats on the progress.
Arthur He: So I just had a quick one regarding the.
Arthur He: 400 phase III study.
Arthur He: Great.
Arthur He: Jean Agonistic arm.
Arthur He: Is there a minimum number of patients for each specific gene mutation too.
Speaker Change: To meet the requirements for the BLA.
Arthur He: Application.
Shankar Musunuri: Hey Arthur, good morning. I'll let Arun take the question.
Darren: Hey, good morning, I'll, let Darren take that.
Arun Upadhyay: No, we have not pre-specified any specific number of patients for each mutation, but our approach is going to include as many mutations as possible in the gene agnostic count.
Rick: No we have not pre specified any any specific number of spacing 42, mckesson, but our approach is going to be.
Arun Upadhyay: In closing as many mood as soon as possible in the G&A agnostic.
Arthur He: Okay, thanks. Thanks for the color. And also, my second question is regarding the 200. Could you give us more color on the status and what your effort to get the Thank you for watching.
Arun Upadhyay: Okay. Thanks, thanks sexual color.
Arthur He: And also my second question is.
Arthur He: Regarding the 200 could you give us more color on the status and.
Speaker Change: What's your effort to get.
Speaker Change: The clinical hold lifted as soon as possible.
Arthur He: Sure.
Shankar Musunuri: Yes, so we are working on addressing the CMC question we received from FDA, and we are planning to, you know, address that SAP and then continue the development of WAPI 200 further.
Speaker 2: Yes. So we are we are working on addressing the CMC question, we received from the FDA.
Speaker Change: And we are planning to.
FDA: And does that Asap.
SAP: And then continue with the development of what we're doing differently.
Arthur He: Okay, great. Thanks for taking my question.
Speaker Change: Alright, great. Thanks for taking my question.
Arthur He: Thank you okay.
Robert Michael LeBoyer: Your next question comes from Robert LeBoyer with Noble Capital Markets. Please go ahead.
Speaker Change: Your next question comes from Robert Clay buyer with Noble capital markets. Please go ahead.
Robert Michael LeBoyer: Good morning and congratulations on all the progress during the quarter. My question has to do with the phase three trial coming up, and you had mentioned some milestones to start. Are there any? Any time frames or any particular milestones that we can look forward to in terms of accrual or projected time for data release?
Robert Michael LeBoyer: Good morning, and congratulations on all the progress during the quarter.
Speaker Change: My question has to do with the phase III trial coming up and you had mentioned some milestones to start are there any that.
Robert Michael LeBoyer: Any time frames or any particular milestones that we can look forward to in terms of accrual or projected time.
Robert Michael LeBoyer: For data release.
Shankar Musunuri: Yeah, Robert. I'll let Dr. Qamar answer the question. I thank you for your question.
Robert Michael LeBoyer: Robert I'll, let Dr Kumar answer the question Hi, Thank you for your question in terms of the milestones and you have to bear that Keppel Huntington's recently got approval. We're currently screening patients and we will be continuously and taking the market.
Huma Qamar: Hi, thank you for your question. In terms of the milestones, as you are aware, Ocu 400 has recently been approved, we are currently screening patients, and we will be continuously updating the market with dosing updates.
Speaker Change: Losing updates.
Huma Qamar: Sure.
Robert Michael LeBoyer: Okay, is there any projected time for the first data release?
Huma Qamar: Okay is there any <unk>.
Robert Michael LeBoyer: Projected time for first data release.
Huma Qamar: for OCU 400 Phase 3 or Phase 1-2. [inaudible] Okay, so Phase 1-2, we have already updated that. Continuous updates will be coming in the next quarter as well as as soon as we receive more data on the LCA patients. For Oncule 400 Phase 3, there will be no time frame as it will be periodic, and we will be giving periodic updates to the market as soon as it's available. And it's a good thing
Robert Michael LeBoyer: Rocket 400.
Huma Qamar: <unk>.
Speaker Change: Our phase one slashed it.
Huma Qamar: Okay.
Speaker Change: Okay. So phase one cash that we have already updated that continuous updates would be coming in the next quarter as well and as soon as needed seek more downtown D&C of patients.
Speaker Change: For Q4 hundred phase III. It will be there is no timeframe as it will be periodic and we will be giving updates to the market as soon as it's available.
Shankar Musunuri: and it's a Robert. I think this is a controlled clinical trial phase three, so until the study is done, we cannot reveal any information to the market on efficacy.
Huma Qamar: Robert I think this is the controlled clinical trials in phase III, so until the study.
Robert: He is done we cannot reveal any information to the market on efficacy portion.
Robert Michael LeBoyer: Sure, you know, that's... We'll provide periodic updates, as Dr. Qamar is saying, on recruitment and where the trial is going, and also give markets an update on how we are on-track for our target approval for BLA and MA in 2026. Okay, yeah, that's probably as much as anyone can say for a rare disease like this, so thank you.
Shankar Musunuri: Sure.
Robert: We will provide periodic updates on as the electrical motors, saying on recruitment and with the trial is going and also new markets and update about on track for our target.
Huma Qamar: Approval for BLA and MAA in 2026.
Robert Michael LeBoyer: Okay, Yes.
Robert Michael LeBoyer: That's probably as much as anyone can say for a rare disease like this so thank you.
Robert Michael LeBoyer: Well.
Daniil V. Gataulin: Your next question comes from the line of Daniil Gataulin with Chardon. Please go ahead.
Speaker Change: Your next question comes from the line of Danielle <unk> Colleen Lake Charles Please go ahead.
Daniil V. Gataulin: Yeah, hey, good morning, guys. Thank you for taking the question. Also, a question on Ocule 400 and phase three. You mentioned that, as part of a deal track strategy, you will be expanding phase three to include patients with LCA, pending phase one TLCA data. I just wanted to clarify if this will be added as a new arm in the trial or if it will be a separate standalone trial. And I guess the second part of the question is, what data from LCA phase one and two would you consider favorable that would give you a go-ahead decision for these patients? Thank you.
Daniil V. Gataulin: Hey, good morning, guys.
Daniil V. Gataulin: And for the question.
Speaker Change: Also a question on order for 110 phase three so you mentioned that as part of dual track strategy of expanding phase III to include patients with LCA.
Daniil V. Gataulin: Pending the phase one data at <unk>.
Speaker Change: Just wanted to clarify that this will be added as a new arm in the trial or if it will be a separate stand alone trial.
Daniil V. Gataulin: The second part of the question is what data from our CF phase two would you consider favorable.
Daniil V. Gataulin: Okay.
Daniil V. Gataulin: Giving you go ahead.
Daniil V. Gataulin: Decision for.
Daniil V. Gataulin: For these patients.
Daniil V. Gataulin: Yes.
Shankar Musunuri: will take the call. Thanks, Shankar. Yeah, so based on the outcome of the Phase I-II study, our plan is to have a separate trial for the LCA because it's a different disease. So once we have the data available, we'll be engaging with the agency, and based on the agency recommendation, we'll initiate the trial accordingly.
Speaker Change: Our technical case and good yeah. So best done the phase one two study outcome, our pan needs to have the shipyard time for the LTE, because it's a different disease.
Melissa: So once we have the data level will be engaging with urgency and belt on <unk> Melissa.
Speaker Change: Initially the trial accordingly.
Daniil V. Gataulin: Okay, got it. Thank you. And in terms of the Phase 1-2 data threshold, what would you consider favorable to advance into Phase 1-2?
Shankar Musunuri: Okay got it thank you and in terms of.
Shankar Musunuri: Phase one two data threshold, what we consider favorable.
Daniil V. Gataulin: To.
Shankar Musunuri: Into phase III.
Shankar Musunuri: Primarily safety and efficacy.
Shankar Musunuri: Primarily the safety and efficacy.
Daniil V. Gataulin: Okay. All right. Thank you.
Shankar Musunuri: Okay.
Shankar Musunuri: Thank you.
Shankar Musunuri: This concludes the Q&A portion. I will now turn the call back over to Chairman and CEO, Dr. Shankar Musunuri.
Speaker Change: This concludes the Q&A portion I will now turn the call back over to chairman and CEO, Dr. <unk> car with synergies.
Operator: Thank you operator.
Shankar Musunuri: Our efforts in the first quarter of the year demonstrated the importance of our gene therapy programs and the need to operate the business to ensure their success. We're opportunistic about Ocugen cell therapy and vaccine platforms, knowing that these technologies have great therapeutic and financial potential and are pursuing partnerships to support our entire pipeline. We look forward to what this quarter and the rest of the year holds for the company, our people, and the patients we serve.
Speaker Change: Efforts in the first quarter of the year evidenced the importance of our gene therapy programs and the need to operate the business to ensure their success, we're opportunistic about oxygen cell therapy and vaccine platforms learning that these technologies have great therapeutic and financial potential and are pursuing partnership to support our entire pipeline. We look forward to what this.
Shankar Musunuri: Quarter and the rest of the year holds for the company our people and the patients we serve.
Shankar Musunuri: Thanks, everyone. Bye. Ladies and gentlemen, that concludes today's call. Thank you all for joining us. You may now disconnect.
Shankar Musunuri: Tiffany.
Speaker Change: Thanks, everyone Bye.
Speaker Change: Ladies and gentlemen that concludes today's call. Thank you all for joining you may now disconnect.
Shankar Musunuri: Thanks.
Shankar Musunuri: Okay.
Shankar Musunuri: Yes.
Shankar Musunuri: Okay.
Shankar Musunuri: Okay.
Shankar Musunuri: Okay.
Shankar Musunuri: Okay.
Shankar Musunuri: Yes.
Shankar Musunuri: Okay.
Shankar Musunuri: Sure.
Shankar Musunuri: Yes.
Shankar Musunuri: Okay.
Shankar Musunuri: Yes.
Shankar Musunuri: Okay.
Shankar Musunuri: Okay.
Shankar Musunuri: Okay.