Ocugen Inc (OCGN) 2024 Q2 法說會逐字稿

Good morning, and welcome to Ocugen's second-quarter 2024 financial results and business update. Please note that this call is being recorded at this time. (Operator Instructions)

I will now turn the call over to Tiffany Hamilton, Ocugen's Head of Corporate Communications. You may begin.

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 June 30, 2024. 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 second 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 on the Investors section of the Ocugen 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 projects, 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 filings 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, Ocugen'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. Musunuri.

Thank you, Tiffany, and thank you all for joining us today. We're excited to discuss the substantial progress of our modifier gene therapy platform across all three clinical programs. And to continue growing this program, 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 our company were further acknowledged by our inclusion in the Russell Index in June. This ranking demonstrates the value of our pipeline and supports Ocugen's dedication to creating long-term shareholder value.

Additionally, the recent offering was led by a premier mutual fund, along with participation from leading life sciences investors, which further strengthens our shareholder base. We're actively recruiting patients in our OCU400 Phase 3 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 OCU400. 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 2 ArMaDa clinical trial for the treatment of geographic atrophy, an advanced stage of dry age-related macular degeneration.

Following completion of dosing in patients in Phase 1, I will discuss these pivotal milestones in greater depth later in the presentation. Additionally, we are about to conclude Phase 1 of the OCU410ST, Phase 1/2 GARDian clinical trial for the treatment of Stargardt disease. OCU400 is making remarkable strides in clinical development, and we are actively dosing patients in the Phase 3 liMeliGhT clinical trial.

As announced earlier, OCU400 has received key regulatory approvals, including expanded orphan drug designations for RP from the FDA and the European Medicines Agency as well as regenerative medicine advanced therapy, RMAT, designation from the FDA. This Phase 3 dosing, OCU400 remains on track to meet the 2026 approval target 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 RHO 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 3 study is powered greater than 95%, assuming 50% responder rate. The OCU400 Phase 3 study includes pediatric patients, 80 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 RHO gene mutations, and the other arm has 75 participants that have mutations in any of several other genes, randomized two to one.

In mobility test, the luminance dependent navigation assessment, LDNA, is the primary endpoint of the study. In this assessment, the participant navigates an obstacle course that constitute a more sensitive and specific measurement of visual function and the mobility measurement used in previous Phase 3 clinical trials. The Phase 3 liMeliGhT study will focus on the proportion of responders in treated and untreated groups who achieved an improvement of at least two Lux levels from baseline.

Let me take a moment 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. OCU400 has a 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 indicate the ophthalmology community about the concept of modifier 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 Bakall, who [shows] as the Director of Clinical Research at Associated Retina Consultants and as Clinical Assistant Professor at the University of Arizona, College of Medicine in Phoenix, presented Phase 1/2 data on OCU400.

With the initiation of our EAP for OCU400, RP patients with early, intermediate, to advanced RP with at least minimal retinol preservation and who may benefit from the mechanism of action of OCU400 may be eligible to receive treatment prior to approval of the BLA. The decision by the FDA to endorse the use of OCU400 in any patient with RP reflects the agency's position on the safety, tolerability, and benefit profile of OCU400 for any mutations relative to any risk of treatment. The approval of an expanded access program for OCU400 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 OCU400 for eligible patients through our EAP.

Now let's move on to our developments in our OCU410 and OCU410ST, which aim to treat geographic atrophy, secondary to dAMD and Stargardt 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. OCU410, physically designed to address multiple pathways implicated in the pathogenesis of dAMD, offers a distinct advantage for current treatment options that target only one pathway, the complement system, and requires frequent intravitreal injection, about 6 to 12 doses per year, accompanied by various safety concerns such as roughly 12% of patients develop wet AMD.

OCU410 has the potential to regulate all four pathways related to disease progression, lipid metabolism, inflammation, oxidative stress in the complement system, thereby addressing the underlying causes of the disease with a single subretinal injection.

And ArMaDa clinical trial update, providing further insights into the safety and efficacy of OCU410 is anticipated later this year. Our approach with OCU410 is to provide a comprehensive and durable solution with a potential one-time treatment. There are 2 million to 3 million geographic atrophy patients among the 19 million people affected by dAMD in the US and Europe, demonstrating a considerable market opportunity. In July, we announced the completion of dosing in the third cohort of the OCU410 Phase 1/2 ArMaDa clinical trial for the treatment of geographic atrophy.

To date, nine patients with geographic atrophy have been treated with the low, medium, and high doses. The Phase 2 dose expansion, accessor-blinded clinical trial has been initiated and we will assess the safety and efficacy of OCU410 in a larger group of patients who will be randomized into one of three groups: in medium dose treatment group, a high-dose treatment group, or the control group. Participants must be aged 50 or older, be able to identify 24 letters or more on the BCVA, which is like those charts you read at the optometrist office, and have a total geographic atrophy area between 2.5 and 20.5 square millimeters.

Turning now to OCU410ST, which has received an orphan drug designation from the FDA for the treatment of ABCA4-associated retinopathies, including Stargardt disease, the Phase 1/2 GARDian 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 US and Europe, and there is no approved therapies available. These efforts represent our commitment to advancing treatments for 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 to clinical development.

With that, I'll now turn the call over to Corporate Controller, Michael Breininger, to provide an update on our financial results for the second quarter ended June 30, 2024. Michael?

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 the 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.

Thank you, Mike. We will now open the call for questions. Operator?

(Operator Instructions) Sean Lee.

This is Sean from H.C. Wainwright standing in for RK. Thanks for taking my questions. My first one is on the OCU400 expanded 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 3 study?

Huma?

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 adoption [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, the 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.

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 this study powered to detect it?

Yes. So the treated and untreated. Untreated is not truly untreated because it's accessor-blended study. It's a subretinal surgery. So that's the way you actually blind the study. So the study is powered at 2:1 ratio. That means at 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 and the mobility test, which is our mobility test is proprietary LDNA.

Okay, understood. So 95% to detect a 50% difference? Got it.

Yes.

And then finally, for the OCU410 study update expected later this year, could you elaborate a little more on what can we expect at the update when you provide? What kind of data will you provide?

Yes, for the 410 geographic atrophy, secondary to dry age-related macular degeneration study, we are hoping to provide preliminary safety and efficacy updates later this year.

So we can expect both safety and some efficacy results. Great. Thanks. That's all the questions I have. Thanks again for taking my questions.

Robert LeBoyer, Noble Capital Markets.

My question has to do with OCU400. 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.

Since it's an accessor-blinded study, updates will be providing on the recruitment rates, how we are beating 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'll undertake starting from late next year.

And when the clinical recruitment is done early next year, that will take one year for us to complete the last patient, which is the 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 the US and EU late 2026.

Daniil Gataulin, Chardan Capital Markets.

Hi, this is Jenny on behalf of Daniil. So my first question is on OCU200. Can you tell us where you are in the process for getting the clinical hold lifted for OCU200? 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.

We're still working with FDA to get the -- submit the information requested and try to get the clinical hold lifted. We designed a very simple Phase 1 study. And after FDA Solution lifting the clinical hold, we will define the path forward for the program. Again, I just want to reiterate, our focus has been primarily gene therapy, but the 200 is a good program. As soon as they [deal with the] clinical hold, we'll provide a direction on that progress.

Thank you. So I have another question on OCU400. So are there meaningful differences in achieving responder criteria with the LDNA compared to the mobility assessments used in previous Phase 3 trials?

Yes. 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 the approved product, they have 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 2.

This concludes the question and answer portion. I will now turn the call back over to Chairman, CEO, and Co-Founder, Dr. Shankar Musunuri. Please go ahead.

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 Ocugen's position as a biotechnology leader. Thank you.

Ladies and gentlemen, that concludes today's call. Thank you all for joining. You may now disconnect.