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spk04: Good morning and welcome to OccuGEN'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, OccuGEN's head of corporate communications. You may begin.
spk05: Thank you, Operator, and good morning, everyone. Joining me on today's call and webcast is Dr. Shankar Musunuri, Ocugen's chairman, CEO, and co-founder, who will provide a business update and an overview of our clinical and operational progress. Michael Breininger, our corporate controller, is also on the call to provide a financial update for the quarter ended March 31st, 2024. Dr. Arun Upadhyay, Chief Scientific Officer, Head of Research and Development, and Dr. Huma Kumar, 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 in a replay with the accompanying slide presentation will be available on the investor section of the OccuGen website for approximately 45 days. 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. Such statements are subject to numerous important factors, risks, and uncertainties, and 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 the presentation. Except as required by law, we assume no obligation to update forward-looking statements contained in this presentation, whether as a result of new information, future events, or otherwise after the date of this presentation. Finally, OccuGen's quarterly report, Form 10-Q, covering the first quarter of 2024, has been filed. I will now turn the call over to Dr. Musunari.
spk02: 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 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 diseases, larger multifactorial diseases, such as dry age-related macular degeneration, 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. Ocu410 and Ocu410-ST are now in Phase 1-2 clinical trials, targeting geographic atrophy, secondary to dry age-related macular degeneration, and Stargardt disease, respectively, with the 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 OxyGEN the capability to support neocot, autologous cell therapy, manufacturing for personalized phase three clinical material, contingent upon adequate funding. Finally, OQ500 will soon advance into a phase one clinical trial via the National Institute of Allergy and Infectious Diseases, MIAID, sponsored trial, comparing the administration of OQ500 via two different mucosal routes, inhalation into the lungs and as a nasal spray. With three gene therapy candidates to treat blindness diseases in the clinic and a phase three ready cell therapy candidate, we are confident that 2024 will be a well-weather for Ockigen. We are thrilled to share significant advancements in our leading modified gene therapy candidate RQ400 as it makes remarkable strides in clinical development. The green light from the FDA to begin the Phase III clinical trial marks a pivotal milestone as RQ400 becomes the first gene therapy to progress to Phase III trials with such a broad retinitis pigmentosa indication. Until now, there has been only one market product to treat one of the 100 gene mutations associated with RP. Now there is real hope for all RP patients who haven't had a treatment option. Our completion of enrollment and dosing in Phase I-II trial demonstrated promising safety and efficacy across various genetic mutations and dosage levels, paving the way for Phase III clinical trial. ARQ 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. These endorsements highlight ARCA-400'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 III Limelight clinical trial in the second quarter of 2024. which will include 150 subjects across two arms, one with patients affected by Rho 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 level from baseline in the study eyes. The secondary endpoint will be measured by a change in low luminescence visual equity, score of 0.3 log more from the baseline. The effectiveness of the treatment will be compared to a untreated control group for the secondary endpoint as well. Additionally, leveraging a dual track strategy, we plan to expand the phase three trial in the later half of 2024 to include patients with Leber congenital hemorrhagic LCA contingent on favorable results from phase one to study. Let me take a moment to discuss the unmet need and underserved market for RP and LCA patients. An estimated 1.6 million people globally are affected by RP and LCA combined. In the US 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 judgment. These conditions frequently result in visual impairment and ultimately blindness. 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 RRP and LCA patients, as well as 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. Moving on to our development in the treatment of geographic atrophy, secondary to dry age-related macular degeneration, DAMD, and Stargardt disease, with our OCU410 and OCU410-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. OCU410, specifically designed to address multiple pathways implicated in the pathogenesis of DAMD, offers a distinct advantage over current treatments that the target only one cause of DGA and often require multiple injections per year accompanied by various safety concerns. Our approach with OCTU-410 is to provide a comprehensive and durable solution, a potential one-time therapy for life. Dry AMD affects about 19 million people in the US and Europe combined. while GA affects about 2 to 3 million people in the U.S. and Europe, a significant market opportunity. In December 2023, we began the Phase 1-2 Armada clinical trial for Ocu410. 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. After completion of the third cohort, we'll transition into phase two 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 ARCU410. ARCU410 Phase 1 study is multicenter and open-label, focusing on dose ranging, while Phase 2 is randomized, aiming to expand our understanding of 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. 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 Ocuport NST, which received orphan drug designation from the FDA for the treatment of ABCA4-associated retinopathies, including Stargardt disease. Stargardt affects approximately 100,000 people in the US and EU combined. 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, low dose. In April 2024, The Data Safety and Monitoring Board approved the continuation to the medium-dose phase, which we expect will be completed this month. A clinical trial update for RQ410ST is also anticipated in the third quarter of 2024. The Phase 1-2 trial involves up to 42 participants, 30 adults and 12 children, which exhibit mild to moderate disease symptoms. 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 control untreated group, allowing for a comprehensive assessment of treatments' efficacy across different dosages. Our work across OCU-400, OCU-410, and OCU-410ST affirm our enduring commitment to the success of these modified gene therapies for the benefit of patients faced with the terrible prospect of losing their sight. 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. Mike?
spk00: 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, 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. cash and cash equivalents totaled 26.4 million dollars as of march 31st 2024 compared to 39.5 million dollars 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 thank you mike we will now open this call for questions operator
spk04: 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 by a 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. And your first question comes from the line of Arthur He with HC Wainwright. Please go ahead.
spk03: Hey, good morning, Sean, Ken, and team. 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 to meet the requirement for the BLS application? Hey Arthur, good morning.
spk02: I'll let Aaron take the response. No, we have not pre-specified any specific number of patients for each mutation. But our approach is going to be, you know, include as many mutations as possible in the gene agnostic column.
spk03: Okay. Thanks. Thanks for color. And also my second question is regarding the 200. Could you give us more color on the status and what's your effort to get the clinical holder lifted as soon as possible?
spk02: All right. Yes, so we are working on addressing the CMC question we received from FDA, and we are planning to, you know, address that ASAP and then continue the development of Hockey 200 further.
spk03: Okay, great. Thanks for taking my question. Thank you, Hockey.
spk04: Your next question comes from Robert LeMoyer with Noble Capital Markets. Please go ahead.
spk03: 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 timeframes or any particular milestones that we can look forward to in terms of accrual or projected time for data release?
spk02: Yeah, Robert, I'll let Dr. Kumar answer the question.
spk06: Hi, thank you for your question. In terms of the milestones, as you're aware that OQ400 has recently got approval, we are currently screening patients and we will be continuously updating the market with dosing updates very soon.
spk03: Okay, is there any projected time for the first data release?
spk07: For OCU400 phase 3 or phase 1 slash 2?
spk03: Either, actually.
spk06: Okay. So phase 1 slash 2, we have already updated that. Continuous updates will be coming in the next quarter as well. And as soon as we receive more data on the NCA patients. For OCU400 phase 3, it will be, there is no timeframe as it will be periodic. and we will be giving periodic updates to the market as soon as it's available.
spk02: 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 the efficacy portion of it. Sure, you know, that's probably... We'll provide periodic updates, as Dr. Kumar is saying, on recruitment and where the trial is going, and also give markets an update about OnTrack for our target approval for BLA and MA in 2026.
spk03: Okay. Yeah, that's probably as much as anyone can say for a rare disease like this. So, thank you.
spk04: Your next question comes from the line of Daniel Gattolin with Chardon. Please go ahead.
spk01: Yeah. Hey, good morning, guys. Thank you for taking the question. Also, a question on ARCA-400 and Phase 3. So, you mentioned that as part of DL-TRAC strategy, you will be expanding Phase 3 to include patients with LCA pending the Phase 1 to LCA 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 1-2 would you consider favorable that would give you a go-ahead decision for these patients? Thank you. Lauren will take the call.
spk02: Thanks, Ankur. Yeah, so based on the Phase 1-2 study outcome, our plan is to have the 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 will initiate the trial accordingly.
spk03: Okay, got it. Thank you.
spk01: And in terms of the Phase I-II data threshold, what would you consider favorable to advance into Phase III?
spk02: Primarily the safety and efficacy.
spk03: All right. Thank you.
spk04: This concludes the Q&A portion. I will now turn the call back over to Chairman and CEO, Dr. Shankar Musunori.
spk02: Thank you, operator. Our 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 Archigen 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.
spk03: Tiffany?
spk04: Thanks, everyone.
spk07: Bye.
spk04: Ladies and gentlemen, that concludes today's call. Thank you all for joining. You may now disconnect.
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