This conference call transcript was computer generated and almost certianly contains errors. This transcript is provided for information purposes only.EarningsCall, LLC makes no representation about the accuracy of the aforementioned transcript, and you are cautioned not to place undue reliance on the information provided by the transcript.
spk07: Good morning and welcome to OccuGen's second quarter 2024 financial results and business update. Please note that this call is being recorded at this time. All participant lines are in a listen-only mode. Following the speaker commentary, there will be a question and answer session. I will now turn the call over to Tiffany Hamilton, OccuGen's Head of Corporate Communication.
spk08: You may begin. Thank you, Operator, and good morning, everyone.
spk05: Joining me on today's call and web pass is Dr. Shankar Musunuri, OccuGen's chairman, CEO, and co-founder, who will provide a business update and an overview of our clinical and operational progress. Michael Brininger, our corporate controller, is also on the call to provide a financial update for the quarter ended June 30, 2024. 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 second quarter of 2024. We encourage listeners to review the press release, which is available on our website at oxygen.com. This call is being recorded, and a replay with the accompanying slide presentation will be available on the investor section of the Oxygen 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 risk factors 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 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 as of the date of this 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, Opsitant's quarterly report on Form 10-Q covering the second quarter of 2024, has been filed. I will now turn the call over to Dr. Musuneri.
spk01: Thank you, Tiffany, and thank you all for joining us today. We're excited to discuss the substantial progress of our modified gene therapy platform across all three clinical programs. And to continue driving these programs, we recently completed a successful fundraising effort with net proceeds of $32.6 million, extending our runway into the third quarter of 2025. Our scientific advances and the strategic growth of company were further acknowledged by our inclusion in the Russell Index in June. This ranking demonstrates the value of a pipeline and supports Octogen's dedication to creating long-term shareholder value. Additionally, the recent offering was led by a premier mutual fund, along with the participation from leading life sciences investors. which further strengthens our shareholder base. We are actively recruiting patients in our OQ400 Phase III Limelight clinical trial for the treatment of retinitis pigmentosa, RP. And just this week, we announced FDA approval for an expanded access program, EAP, for the treatment of adult patients aged 18 and older with RP with OQ400. This is the first-ever gene therapy candidate, to treat patients with RP regardless of mutation approved for EAP. We also progressed into the OCU410 Phase II Armada clinical trial for the treatment of geographic atrophy, an advanced stage of triage-related macular degeneration. Following completion of dosing in Phase I, I will discuss these pivotal milestones in greater depth later in the presentation. Additionally, we're about to conclude phase one of the RQ410ST, phase one-two guardian clinical trial for the treatment of Stargardt disease. RQ400 is making remarkable strides in clinical development, and we are actively dosing patients in the phase three limelight clinical trial. As announced earlier, RQ400 has received key regulatory approvals, including expanded orphan drug designations, or RP, from the FDA and the European Medicines Agency, as well as Regenerative Medicine Advanced Therapy, or MAT, designation from the FDA. With phase three dosing, OCU400 remains on track to meet the 2026 approval targets for a biological licensing application, BLA, from the FDA and for a market authorization application, MAA, from the European Medicines Agency. We are very encouraged that more than 60% of the intent to treat patients from the Phase 1-2 clinical trial, including patients with room mutation, meet the responder criteria established for Phase 3. The Phase 3 mobility test responder rate for the only FDA-approved product to treat one mutation in RP was 52%. The phase three study is powered greater than 95%, assuming 50% responder rate. The OQ400 phase three study includes pediatric patients, eight years of age or older, and adults with early, intermediate, to advanced stages of RP. The study has a sample size of 150 participants, One arm has 75 participants with the row gene mutations, and the other arm has 75 participants with the mutations in any of several other genes, randomized two to one. A mobility test, the luminance dependent navigation assessment, LDNA, is the primary endpoint for the study. In this assessment, a participant navigates an obstacle course that constitutes a more sensitive and specific measurement of visual function than the mobility measurement used in previous phase three clinical trials. The phase three limelight study will focus on the proportion of responders in treated and untreated groups who achieve an improvement of at least two lux levels from baseline. Let me take a moment to discuss the unmet need and underserved market for RP patients. There are approximately 300,000 patients in the US and EU that are affected by the disease, which is caused by mutations in any of approximately 100 different genes. The only other treatment currently on the market addresses mutations in one gene associated with RP. RQ400 has the potential to treat multiple gene mutations because of its gene agnostic mechanism of action And in this way, it will fulfill a significant unmet medical need. We continue our extensive campaign to educate the ophthalmology community about the concept of modified gene therapy. And we recently presented supporting data at a variety of conferences, such as annual meeting of the American Society of Retina Specialists, which convened in Stockholm, Sweden last month. At the conference, Dr. Benjamin Pakal, who serves as the director of clinical research, an associated retinal consultant, and as clinical assistant professor at the University of Arizona's College of Medicine in Phoenix, presented Phase 1-2 data on ARCHI 400. With the initiation of our EAP for ARCHI 400, RP patients with early intermediate to advanced RP with at least minimal retinal preservation and who may benefit from the mechanism of action of RQ400 may be eligible to receive treatment prior to approval of the BLA. The decision by the FDA to endorse the use of RQ400 in any patient with RP reflects the agency's position on the safety, tolerability, and benefit profile of RQ400 for any mutations relative to any risk of treatment. The approval of an expanded access program for OQ400 further supports the gene agnostic mechanism of action for this novel modifier gene therapy. We look forward to working with clinicians, patients, and the RP community to provide access to OQ400 for eligible patients through our EAP. Now, let's move on to our developments in OQ410 and OQ410STs. which aim to treat geographic atrophy secondary to D-AMD and start-out disease, respectively. These modifier gene therapies leverage a nuclear receptor gene called RORA, which stands for RAR-related Orphan Receptor A, as a potential one-time therapy for life with a single subretinal injection. RQ410, specifically designed to address multiple pathways implicated then the pathogenesis of DAMD offers a distinct advantage for current treatment options that target only one pathway, the complement system, and require frequent intrauterine injection, about 6 to 12 doses per year, accompanied by various safety concerns, such as roughly 12% of patients developed with AMD. Ocu410 has the potential to regulate all four pathways related to disease progression, lipid metabolism, inflammation, oxidative stress, and the complement system, thereby addressing the underlying causes of the disease with a single subretinal injection. An Armada clinical trial update providing further insights into the safety and efficacy of Ocu410 is anticipated later this year. Our approach with RQ410 is to provide a comprehensive and durable solution with a potential one-time treatment. There are 2 to 3 million geographic atrophy patients among the 19 million people affected by DAMD in the U.S. and Europe, demonstrating a considerable market opportunity. In July, we announced the completion of dosing in the third cohort of the RQ410 Phase 1-2 Armada clinical trial for the treatment of geographic atrophy. Today, nine patients with geographic atrophy have been treated with low, medium, and high doses. The phase two dose expansion SSR-blinded clinical trial has been initiated and will assess the safety and efficacy of RQ410 in a larger group of patients who will be randomized into one of three groups, a medium dose treatment group, a high dose treatment group, are a control group. Participants must be aged 50 or older, be able to identify 24 letters or more on a BCVA, which is like those charts you read at the optometrist's office, and have a total geographic atrophy area between 2.5 and 20.5 square millimeters. Turning now to occupant tenacity, which has received an orphan drug designation from the FDA for the treatment of ABCA4-associated retinopathies, including Stargardt disease. The Phase I-II Guardian clinical trial for the treatment of Stargardt disease is actively enrolling patients in the high-dose cohort and the dose escalation portion of the study. Stargardt affects approximately 100,000 people in the U.S. and Europe, and there is no approved therapies available. These efforts represent our commitment to advancing treatments of blindness, focusing on innovative gene therapy solutions that aim to provide lasting benefits to patients. We look forward to sharing further updates as we continue to advance these promising therapies for clinical development. With that, I will now turn the call over to Corporate Controller Michael Brininger to provide an update on our financial results for the second quarter ended June 30, 2024. Michael.
spk00: Thank you, Shankar. The company's cash, cash equivalents, and restricted cash totaled 16 million as of June 30, 2024, compared to 39.5 million as of December 31, 2023. The company had 257.4 million shares of common stock outstanding as of June 30, 2024. Total operating expenses for three months ended June 30, 2024, were $16.6 million and included research and development expenses of $8.9 million and general and administrative expenses of $7.7 million. This compares to total operating expenses for the three months ended June 30, 2023 of $24 million that included research and development expenses of $14.5 million and general and administrative expenses of $9.5 million. As stated earlier, we recently completed a successful fundraising effort with net proceeds of 32.6 million, extending our runway into the third quarter of 2025. As always, we are constantly exploring strategic and shareholder-friendly opportunities to increase our working capital and continue to pursue strategic partnerships that will drive long-term strategy. That concludes my update for the quarter. Tiffany, back to you.
spk08: Thank you, Mike. We will now open the call for questions. Operator?
spk07: This time, I would like to remind everyone, in order to ask a question, press star, then the number one on your telephone keypad. We will pause for just a moment to compile the question and answer roster.
spk08: Our first question comes from the line of Chan Lee. Please go ahead.
spk02: Hi, good morning. This is Sean from HSC Wingright, sending in for RK. How are you?
spk01: Good morning, Sean.
spk02: Great. Thanks for taking my questions. My first one is on the RQ400 extended access program. So I was wondering, what is the EAP primarily targeted towards, since I'm sure you are still actively recruiting a lot of patients into the Phase III study?
spk01: Juma?
spk06: Yes, thank you for the question. So the Expanded Access Program is targeting the population that do not meet the inclusion-exclusion criteria of our Phase 3, or they would have to have an option meeting a little bit more flexibility based on what we have not offered in our Phase 3, because that is mandated by FDA regulatory process. So in this trial, our inclusion criteria would be 18 years of age, anyone that has a clear certified genetic diagnosis of RP and those who have photoreceptors left. And also discretionary by the treating physician, this is the decision that individually will be taken by the treating physician and the patient.
spk02: I see. Thanks for that. On to the OCU400 phase 3, I was wondering, have you disclosed what's the expected difference between the treatment and untreated arms, and how is the study powered to detect it?
spk01: Yeah. So the treated and untreated. Untreated is not truly untreated because it's an SSR-blinded study. It's a subretinal surgery. So that's the way you actually blind the study. So the study is powered at two to one ratio. That means out of 150 patients, 50 patients are going to be in the untreated group. And the study is powered at greater than 95%, assuming there is a 50% response rate. So responders are defined as who can reach either two levels or higher on the mobility test, which is our mobility test is proprietary LDNA.
spk02: Okay, understood. So 95% to detect a 50% difference. Got it. And then finally, for the For the Office 410 study update expected later this year, could you elaborate a little more on what can we expect at the update? What will you provide? What kind of data will you provide?
spk06: Yes, for the 410 geographic atrophy secondary to dry age-related microdegeneration study, we are hoping to provide preliminary safety and efficacy updates later this year.
spk02: So we can expect both safety and some efficacy results then? Yes. Great. Thanks. That's all the questions I have. Thanks again for taking my questions.
spk08: Thank you.
spk07: Our next question comes from the line of Robert Leboyer with Noble Capital Markets. Please go ahead.
spk03: Good morning. My question has to do with OCU 400, and you had mentioned that you're on track for the 2026 BLA. So I was wondering if you could give any details on upcoming milestones or data presentations for the trial.
spk01: Robert, good morning, Robert. Since it's an SSR-blinded study, updates will be providing on the recruitment rates. how we are meeting the BLA timeline. Since we do have RMAT designation, as well as orphan designations in US and EU, that will allow us to do a rolling submission of our BLA and MAA. So that's the process potentially we're going to take starting from late next year. And then when the clinical recruitment is done early next year, that will take one year for us to complete the last patient, which is a duration of the trial. And when the data comes out, we'll close the clinical sections. And then that will trigger the accelerated path of six months in 2026. So that will allow us to potentially get approvals in both U.S.
spk08: and EU late 2026. Okay, great. Thank you very much. Thank you, Robert.
spk07: Our next question comes from the line of Daniel Gatulin with Shazan Capital Market.
spk08: Please go ahead. Hi, this is Janani on behalf of Daniel.
spk04: So my first question is on OCU 200. Can you tell us where you are in the process for getting the clinical hold lifted for OCU 200? And once the hold is lifted, will you be launching the trial right away, or are you focusing on the gene therapy programs at this point? Thank you.
spk01: We're still working with FDA to submit the information they requested and try to get the clinical hold lifted. And we designed a very simple phase one study. And after FDA's decision lifting the clinical hold, we will define the path forward for the program.
spk08: OK.
spk01: Again, I just want to reiterate, our focus has been primarily gene therapy, but the 200 is a good program.
spk08: As soon as FDA lifts the clinical hold, we'll provide a direction on that program. Okay, thank you.
spk04: So I have another question on OCU-400. So are there meaningful differences in achieving responder criteria with the LDNA compared to the mobility assessments used in previous Phase III trials?
spk08: Yes.
spk01: As we stated and showed today, intent to treat population data we analyzed from the phase 1-2. That means patients who will qualify for phase 3 based on our criteria. And we clearly showed 62% response rate based on people who can reach two levels or more. And in the approved product, they are 52% response rate. And I think one of the questions earlier we addressed, we powered the study at 50% response rate. That means we actually powered it lower than what we achieved in phase two.
spk08: Thank you.
spk07: This concludes the question and answer portion. I will now turn the call back over to Chairman, CEO, and co-founder, Dr. Shankar Musdenori. Please go ahead.
spk01: Thank you, Operator. Thank you, everyone, for joining us today. We appreciate your continued support as we move forward with our groundbreaking scientific and clinical initiatives. We look forward to the second half of 2024 as we continue to solidify Ocigen's position as a biotechnology leader.
spk08: Thank you. Ladies and gentlemen that concludes today's call. Thank you all for joining. You may now disconnect.
Disclaimer