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spk07: Good morning and welcome to Occugen's 3rd Quarter 2023 Financial Results and Business Update. Please note that this call is being recorded at this time. Our participants' 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.
spk08: Thank you, Operator, and good morning, everyone. Joining me on today's call and webcast is Dr. Shankar Musuneri, Occasion'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 September 30, 2020. Dr. Rune Apadhyayi, Chief Scientific Officer, Head of Research, Development, and Medical, will be available to answer questions following the presentation. This morning, we issued a press release detailing associated business and operational highlights for the third quarter of 2023. We encourage listeners to review the press release, which is available on our website at OckieGen.com. This call is being recorded, and a replay with the accompanying slide presentation will be available on the Investors section of the OckieGen 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. The 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. These and other risks and uncertainties are more fully described in our periodic filing with the Securities and Exchange Commission, SEC, including the risk factors described in the section titled Risk Factors in the quarterly and annual reports we've filed with the SBC. 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 on Form 10-Q covering the third quarter of 2023 has been filed. I will now turn the call to Dr. Musuneri.
spk04: Thank you, Tiffany, and thank you all for joining us today. As emphasized in the press release we put out this morning, we continue to make significant headway for the development of our pipeline of steps, particularly with our first-in-class ophthalmic gene therapy programs, and I'm proud of the momentum we have achieved today. Following additional positive and encouraging clinical study results from our novel modifier gene therapy-based Phase 1-2 RQ400 study in September 2023, we believe we have strong clinical evidence to initiate our Phase 3 clinical trial in retinitis pigmentosa RP patients in early 2024 based on FDA concurrence. Utilizing a dual-track strategy, we also intend to Expand our OQ400 phase three trial in the second half of 2024 to include patients with Leber congenital amaurosis, LCA, contingent upon favorable results from the phase one to study. With enrollment begun for our OQ410 and OQ410-ST programs, we are diligently working to dose patients this quarter. We anticipate clinical updates from our OQ400, OQ410, and OQ-14-ST studies in the later part of 2024. Our clinical and regulatory teams continue to work on responses to the FDA regarding our IND submission for OQ-200, the company's ophthalmic biological product candidate, and we plan to initiate a phase one clinical study in the first half of 2024 contingent on the lift of the FDA hold and adequate availability of funding. For our regenerative cell therapy candidate for knee cartilage repair, Neocort, we are on track to complete construction of our state-of-the-art CGMP facility at the end of this year and are planning to complete qualifications of the facility in the first half of 2024. We plan to initiate the phase three clinical trial in the second half of next year. Last month, we were delighted to be selected for inclusion in a phase one clinical trial funded by National Institute of Allergy and Infectious Disease, to investigate the administration of our COVID-19 mucosal vaccine candidate, RQ500. Safety and immunogenicity of RQ500 will be evaluated using inhaled and intranasal routes of delivery during the Phase I clinical trial in the first half of 2024. All these catalysts considered, we can safely reiterate that 2024 will be transformative for Ocugen. Our mission to introduce critically needed therapies into the market is imminent with the planned initiation of phase three trials encompassing gene and cell therapies in the near term. Our R&D team's dedication and hard work has yielded significant progress and compelling results for our first-in-class modifier gene therapy Ocu400 program for RP and LCA patients. Throughout the Phase I-II trial, our primary objective has been to observe safety and tolerability of the subretinal administration of OQ400 in subjects as well as immune response and systemic distribution. For preliminary science of efficacy, we focused on a few visual function and functional vision indicators, namely best corrected visual equity, BCEA, low luminescence visual equity, LLVA, and multi-luminescence mobility test, MLMT. More details on our trial design can be found on clinicaltrials.gov with the identifier code listed at the bottom of the slide. Let me provide a situational analysis around unmet need and under-deserved market for RP and LCA patients. An estimated 1.6 million people globally are affected by RP and LCA combined. In the US alone, we're looking at about 125,000 patients total. RP and LCA are classified as inherited retinal diseases from a group of heterogeneous disorders that affect the retina. These diseases often lead to sight loss and ultimately blindness. That said, the earlier homeostasis can be stabilized in patients with either of these diseases, the better. Through relevant medical meetings and continued engagement with advocacy groups, we aim to create awareness for the prevalence of retinitis pigmentosa and Leber congenital neurosis and potential emerging therapies like our novel platform. Our ultimate objective is to provide treatment to people suffering from visual impairment and blindness caused by RP and LCA for whom currently no therapeutic options exist. I listed our three exploratory endpoints for visualization, stabilization, and improvement observed in patients treated with OQ400 on slide four. BCVA, LLVA, and MLMT. And the 12 cumulative subjects that have undergone a minimum of six months follow-up post-OQ400 dosing we observed the following metrics. This Venn diagram demonstrates that eight out of 12 subjects showed either stabilization, means no change from baseline, plus minus four letter change for BCVA and LLVA, and zero lux level change for MLMT, or improvement in all three parameters of BCVA and LLVA, which means five or more letters, and MLMT greater than or equivalent to one lux level, demonstrating initial efficacy of OQ400. Non-responders are listed outside the circles for each group. To recap, what we know from our findings to date is that OQ400 has a favorable safety and solubility profile in patients. Positive trends are observed in all set visualization stability and improvement factors, which details that. Eighty-three percent of subjects demonstrated stabilization or improvement in the treated eye either on BCVA, LLVA, or MLMT scores from baseline. Seventy-eight percent of subjects showed stabilization or improvement in treated eyes and MLMT scores from baseline. Eighty-six percent of row mutation subjects experienced either stabilization or improvement in MLMT scores from baseline among which 29% demonstrated three lux luminescence level improvement, demonstrating the gene agnostic mechanism of action of RQ400. The role mutation affects more than 10,000 people in the U.S. alone. Based on this data, we're highly enthusiastic about the future of RQ400 and the vision-saving potential it may provide to RP and LCA patients. The execution of critical elements of the RQ400 Phase 1-2 trial, including the completion of dosing of RP and LCA patients, sets the stage for us to execute a Phase 3 clinical trial for both indications in 2024 upon FDA concurrence. RQ410, our modifier gene therapy candidate for dry age-related macular degeneration, AMD, is a potential one-time curative therapy with a single subretinal injection that targets multiple pathways causing dry AMD, including lipid metabolism, inflammation, oxidative stress, and complement activation. Unlike other currently market products targeting a single pathway, complement activation. We are currently enrolling patients in the Phase 1-2 ARC-MOD-R study to assess the safety and efficacy of RQ410 for geographic atrophy secondary to dry AMD. Geographic atrophy, an advanced form of dry age-related macular degeneration, affects approximately one million people in the United States alone. From a competitive standpoint, we believe Ocu410 is differentiated among other therapies available and in development for geographic atrophy and dry AMD by its frequency of administration, one time versus multiple injections per year, reduced side effects from structural impact, strong safety profile, It's a mechanism for restoring homeostasis and preserving the conditions that promote cell health. The slide demonstrates how Ocu410 utilizes an AAV delivery platform for the retinal delivery of Rora gene. In preclinical studies, Ocu410 demonstrated efficacy in regulating multiple pathways involved with the disease, including lipid metabolism, reducing drusen formation, regulation of inflammation, suppressing inflammation, oxidative stress, improving cell survival, membrane attack complex, complement, restoring anti-complement protein. On this slide, we have captured a proposed program design for Oxy-410 in 63 adult subjects, 50 or older, with geographic atrophy, secondary to dry AMD, We will observe the treatment effect of a single unilateral subretinal injection or Q410 starting with safety and efficacy in patients. We're employing a 3 plus 3 design with a low, medium, and high dose in addition to a dose expansion exercise using a 1 to 1 to 1 design, randomizing subjects to either two treatment groups or dose levels or one control group. Using a similar approach, our orphan drug-designated OQ410ST modifier gene therapy platform for Stargardt disease leverages nuclear hormone receptors to modulate cell activity and utilize this in AAV delivery platform for refinal delivery of the RAR-related orphan receptor A. Ocuportin delivery and preclinical studies of Stargardt disease demonstrated a structural and functional improvement. In the Ocuportin ST Phase 1-2 trial, we intend to treat and investigate 42 subjects, 30 of which are adults and 12 are children with Stargardt disease. The adult inclusion criteria look at adult patients between 18 to 65 and pediatrics between 6 to 17. We're employing a three plus three design with a low, medium, and high dose cohort in addition to your dose expansion exercise using a one-to-one-to-one design, randomizing subjects to either two treatment groups per dose levels or one control group. Our team's diligent efforts resulted in NIAID selecting OCU400 for inclusion in a project next-gen phase one clinical trial of our mucosal vaccine candidates for COVID-19, likely to be initiated in the first half of 2024. From our own development efforts, we observed vaccine-induced high neutralizing and effector responses during preclinical studies on RQ500. We believe the inhaled route of administration has the potential to be the holy grail for broad and durable protection from severe diseases and can suppress the transmission rate. As a refresher, Project NextGen, a multi-government agency initiative overseen by NIAID, is a $5 billion multi-government agency initiative to develop the next generation of vaccines and therapeutics to combat the spread of COVID-19. NIAID will execute the clinical trial for Octu500. Upon completion of the trial, Ophigen will possess full rights of reference to the findings. This initiative is a testament to the fact that COVID-19 is still rampant with emergence of new variants and needs more durable vaccines to treat them. In a recent Harris poll, we favorably found that 66% of Americans would prefer to have more vaccine options. The poll also found that 52% of Americans would be more open to getting an intranasal or inhaled versus injectable COVID-19 vaccine. In line with MIAD's missions to support innovation and public health, We look forward to potentially expanding the platform to the flu and other respiratory viral diseases and infections. I'd like to bring our pipeline updates to a close by providing a brief update on Neocard. Ophigen's autologous regenerative cell therapy, which uses patients' own cartilage cells, is on track to begin its phase three clinical trial in the second half of 2024. A CGMP facility for manufacturing Neocard is expected to be completed at the end of 2023, and qualification is expected in the first half of 2024. RQ200 is an ophthalmic biological product candidate in preclinical development for treating severely sight-threatening diseases like diabetic macular edema, diabetic retinopathy, and wet-agilated macular degeneration. We are working on responses and continue to interact with the FDA regarding the clinical hold on our RQ200 IND submission and expect to initiate a phase one clinical study in the first half of 2024. With that, I will now turn the call over to our corporate controller, Michael Berlinger, to provide an update on our financial results for the third quarter ended September 30, 2023. Michael.
spk00: Thank you, Shankar. Our research and development expense for the quarter ended September 30, 2023 were 6.3 million compared to 15.6 million for the third quarter of 2022. General and administrative expenses for the quarter ended September 30th, 2023 were 9.1 million compared to 7.5 million during the same period in 2022. Net loss was approximately 14.2 million or six cents net loss per share for the quarter ended September 30th, 2023 compared to a net loss of approximately 21.9 million or 10 cents net loss per share for the third quarter of 2022. Net loss was approximately 53.6 million or 22 cents net loss per share for the nine months ended September 30, 2023, compared to a net loss of approximately 59.4 million or 28 cents net loss per share for the nine months ended September 30, 2022. Our cash, cash equivalents, and investments sold 53.5 million as of September 30th, 2023, compared to 90.9 million as of December 31st, 2022. As always, we are constantly exploring strategic and shareholder-friendly opportunities to increase our working capital, and we'll be focused on seeking out corporate partnerships for gene therapies and non-dilutive funding for vaccines. That concludes my update for the quarter. Tiffany, back to you.
spk06: Thank you, Mike. We will now open the call for questions.
spk05: Operator? Open for your questions.
spk07: To ask a question, this time, please press star, then the number one on your telephone keypad. We'll pause for just a moment to compile the Q&A roster. We have a question come from the line of Arthur Herr, H.E. Wainwright. Your line is open.
spk02: Hey, good morning, Shanka and Tim. Here's Arthur for RK. Congrats on the progress. So I just had a couple questions on 400. So when could we expect the complete data set for the Phase II study, especially for the LCA patients? If you can give some color on that, it would be appreciated. Yeah, the LCA, we just dosed...
spk04: So I think it'll take until later part of next year.
spk02: How about the other patient left in the RPE group?
spk04: Yeah, the RPE patients will get it in the first half. However, we believe we have adequate information and we're working with regulatory agencies, FDA and EMA, for phase three.
spk02: Okay. So speak of the phase three study. So From what you said in the press release and the core, is the phase three will be a single phase three packet together, both RP patient as well as LCA, or it could be two separate phase three study?
spk04: We'll start with RP because that's the data we have right now. And then we're going to add LCA arm a little later in the clinical trial.
spk02: Okay, so that would be in the single phase three or? Yeah, phase three, yeah. Okay, okay, I see. And the last question on 400 is, one is, have you requested a meeting with FDA and based on your own proposal, which endpoint could be your primary endpoint?
spk04: And let Dr. Rupa, they answer that or CSO, go ahead.
spk03: Thanks, Shankar. So we are considering a combinatorial approach, and we have proposed that to the FDA. And we are going to have a meeting with them this quarter. And accordingly, once we have alignment with FDA, then we'll update the market.
spk05: Yeah. All right. Sounds great. Thanks for taking my question. Thank you.
spk07: Our next question comes from the line of Robert Leboyer with Nobel Capital Markets. Your line is open.
spk01: Good morning and thanks for taking my question. I just had a follow-up on Phase 3 for OCU 400 and wondering if you have any information or could disclose how many patients you expect to be in the trial or what the length of follow-up is going to be for the patients.
spk04: Good morning, Robert. I'll let Arun address that.
spk03: Yeah, so we are planning in the range of close to 100 subjects in Phase 3 in one-to-one randomization and one-year follow-up.
spk05: Okay, thank you very much. Just to confirm, it is 100, okay? 100 patients, Robert.
spk06: Again, if you would like to ask a question, press star, then the number one on your telephone keypad.
spk05: There are no further questions at this time.
spk07: I will now turn the call over to Chairman and CEO, Dr. Shankar Masunuri.
spk04: In closing, I'd like to reiterate our unwavering commitment to groundbreaking science and clinical innovations in order to create effective and positively impactful therapies that are accessible to patients globally. As we continue to execute stated plans, we remain focused on delivering long-term value for our shareholders who have supported us and for prospective ones seeking to be part of our story. Thank you and have a great day.
spk05: Thanks, everyone. This concludes this conference call. You may now disconnect.
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