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. All participants lines are in a listen-only mode. Following the speakers' 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.

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, plan, intend, 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 time lines. 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 reports 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 are 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 the company were further acknowledged by our inclusion in the Russell Index in June. This ranking demonstrates the value of 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, or RP, from the FDA and the European Medicines…

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 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]. Our first question comes from the line of Sean Lee. Please go ahead.

Hi, good morning. This is Sean from H.C. Wainwright, standing in for RK. How are you?

Good morning, Sean.

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 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.

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 what -- how is the study powered to detect it?

Yes. So the treated and untreated. Untreated is not truly untreated, because it's assessor blinded study. It's a sub-retinal surgery. So that's the way you actually plan to study. So the second -- so the study is powered at 2:1 ratio. That means out of 150 patients, 50 patients are going to be in the untreated group, and 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.

Okay, understood. So 95% to detect a 50% difference? Got it. 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? What will you provide? What kind of data will you provide?

So 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 then?

Yes.

Great. Thanks. That's all the questions I have. Thanks again for taking my questions.

Thank you, Sean.

Your next question comes from the line of Robert LeBoyer with Noble Capital Markets. Please go ahead.

Good morning. My question has to do with OCU400. And you'd 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?

Robert, good morning, Robert. Since it's assessor blinded study, updates we'll 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 U.S. and EU that will allow us to do a ruling submission of our BLA and MAA. So that's the process potentially we're going to take 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 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. and EU late 2026.

Okay, great. Thank you very much.

Thank you, Robert.

Our next question comes from the line of Daniil Gataulin with Chardan Capital Markets. Please go ahead.

Hi. This is Janani on behalf of Daniil. So my first question is on OCU200. So 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 they requested and try to get the clinical hold lifted. And we designed a very simple Phase 1 study and after FDA to lifting the clinical hold, we will define the path forward for the program.

Okay. And for…

I mean, again, I just want to reiterate, our focus has been primarily gene therapies, but the 200 is a good program. As soon as FDA lift the clinical hold, we'll provide a direction on that program.

Okay. 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 then 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 2.

Okay. Thank you.

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.