Good morning and welcome to Ocugen's Third Quarter 2024 Financial Results and Business Update. Please note that this call is being recorded at this time. All participants' lines are in 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.

Ramesh Ramachandran, our Chief Accounting Officer, who is transitioning from Mike Breininger, Interim Chief Accounting Officer, is also on the call to provide a financial update for the quarter ended September 30, 2024. Dr. Huma Qamar, Chief Medical Officer, and Dr.

Arun Upadhyay, Chief Scientific 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 third 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 predicts, believes, potential, proposed, continue, estimates, anticipates, expects, plans, intends, may, could, might, will, should, are 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 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, the SEC, including risk factors described in the section entitled Risk Factors and 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 it's 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 the presentation.

Finally, Ocugen's Quarterly Report, Form 10-Q, covering the third quarter of 2024, will be filed next week. I will now turn the call over to Dr. Musunuri..

a medium-dose treatment group, a high-dose treatment group on an untreated control group. Participants must be aged 50 or older, be able to identify 24 letters or more on a BCVA which is like the charts you read at optometrist office and have a total geographic atrophy area between 2.5 and 20.5 square millimeters.

Turning on to OCU410ST, which has received orphan drug designation from the FDA for the treatment of ABCA4-associated retinopathies, including Stargardt disease. Phase 1 dosing of the Phase 1/2 GARDian clinical trial has been completed and OCU410ST demonstrated a favorable safety and tolerability profile.

To-date, no SAEs related to OCU410ST have been reported. The Data and Safety Monitoring Board, has approved proceeding to Phase 2 of the clinical trial. Stargardt disease affects approximately 100,000 people in the US and Europe, and no approved therapy is available.

This condition is the most common form of inherited macular dystrophy with symptoms of bilateral central vision loss typically forming during childhood, and gradually worsening over a person's lifetime. A preliminary safety and efficacy update on the OCU410ST Phase 1/2 GARDian clinical trial will also be featured at the upcoming clinical showcase.

Lastly, I’d like to call attention to our biologic platform OCU200, which possesses unique features to treat vascular complications of Diabetic Macular Edema, DME.

In recent news, we announced that the FDA cleared the investigational drug application for the Phase 1 clinical trial evaluating OCU200, a recombinant fusion protein consisting of tumstatin and transferrin for treatment of DME. DME causes blurriness in vision and progressive vision loss as the disease advances.

Approximately 746,000 patients in the United States are affected with the DME. The condition is becoming more prevalent as a number of people with the diabetes in the US raises, making it more imperative to address.

Approximately 30% to 40% DME patients are refractive to current anti-VEGF therapies, and we believe that OCU200 has the potential to provide a new treatment option for a significant percentage of people living with DME, including non-responders to the current standard-of-care. We plan to initiate Phase I clinical trial of OCU200 this quarter.

Our efforts across platforms represent our commitment to treating blindness diseases, focusing on innovative solutions that aim to provide lasting patient benefits. We look forward to sharing further updates as we advance these therapies through clinical development.

I will now turn the call over to Ramesh Ramachandran to provide an update on our financial results for the quarter ended September 30, 2024.

Ramesh?.

Thank you, Shankar. The company's cash and restricted cash totaled $39 million as of September 30, 2024 compared to $39.5 million as of December 31, 2023.

Total operating expenses for the three months ended September 30, 2024 were $14.4 million and included research and development expenses of $8.1 million, and general and administrative expenses of $6.3 million.

This compares to total operating expenses for the three months ended September 30, 2023, of $16.1 million that included research and development expenses of $7 million, and general and administrative expenses of $9.1 million.

As stated earlier, we recently completed a successful debt financing of $30 million, extending our runway into the first quarter of 2026. As always, we are proactively exploring shareholder-friendly opportunities to increase our working capital, including partnerships that will drive long-term strategy for our scientific platforms.

That concludes my update for the quarter. Tiffany, back to you..

Thank you, Ramesh. We will now open the call for questions.

Operator?.

[Operator Instructions] Our first question comes from the line of Jason McCarthy with Maxim Group. Your line is open..

Hi guys. Thanks for taking the question and congrats on the quarter. So at the upcoming clinical showcase, we are expecting some of the initial data in GA. Could you just help us understand what we can be looking for from that -- the bar for success and then in particular for gene agnosticism..

Huma?.

So thanks for the question. So in the upcoming clinical showcase, we will be presenting the geographic atrophy in our other gene therapy trials, preliminary safety and efficacy. Particularly for the GA, we have established the safety at this point.

And what we will be showcasing the efficacy endpoints like geographic atrophy lesion and other parameters would be functional as well as structural. So we will be presenting that data in the upcoming clinical showcase on November 12 at NASDAQ..

Okay. Great. Thanks. And then for the Stargardt study, when could we expect to see data? And then also as a follow-up with a couple of small molecule drugs in Stargardt and late-stage development.

How do you sort of see those fitting in with OCU410ST, if they are approved?.

Okay. That's a great question currently. Just to address that there are no approved therapeutic or any treatment approved for Stargardt, so there is significant unmet medical need out there. In our upcoming clinical showcase, we will also be presenting the safety as we have already apprised the market through our DSMB press releases.

But for efficacy, we will also be presenting that in our upcoming clinical showcase. So stay tuned for all the updates..

So Jason, I know there are some couple of products in the development. However, we are planning for a treatment option, only treatment option with our therapy. At this time, we're not thinking about any co-therapies, in the first-line..

Got it. Yeah. And then I just thought I'd bring up since it is topical. If you could just -- given the recent issues with existing gene therapies, Pfizer dropping out, bluebird coming under [fire for safety] (ph).

Can you just talk a bit about how modifier gene therapies avoid some of those pitfalls impacting the space at large?.

Yes. I mean good question, Jason. I think two things, I think we need to consider when you're doing any gene therapies. First and foremost, product quality is extremely important.

And during the course of last few years with the three parallel clinical trials going on, in ophthalmology space, probably we have the biggest ophthalmology clinical trials in gene therapy space. We have not seen any adverse events SAEs related to our product. The product seemed to be safe and effective. Therefore -- that's number one.

Quality of the product is very important. We believe we are consistently making high-quality product. Number two, we target subretinal surgeries to get this gene therapy into retina. And so we do have highly skilled experienced retinal surgeons in our network, and we formed a very good network of the surgeons for all our clinical trials.

So that's very important. The procedure is important. So those are the two things we're focusing on. And obviously, there are also differences. I just wanted to point out for listeners, systemic gene therapy versus ophthalmology gene therapy.

I mean, our target dose typically in ophthalmology space because we directly go into the target, goes from 10 to 10 to the 10 to 11 gene copies. Systemic gene therapies go into 10 to the 14 and higher. And so there is a significant difference, almost like 1,000-fold to 10,000-fold increase.

And so there are other things come up when you go to systemic gene therapies.

Okay, thanks. That was helpful..

Our next question comes from the line of Daniil Gataulin with Chardan. Your line is open..

Hi, good morning guys. Thank you for taking my question. And congrats on the progress. I had one question on geographic atrophy program. So given there are no still no approved options for it in Europe, how are you thinking of taking advantage of the opportunity. And have you had any interactions with the regulators regarding your program? Thank you..

Great question, Daniil. Yes, we will start interactions this year -- I mean, the next few quarters with the EU. Obviously, the Phase 2 is limited to US. What EU regulators are looking for is some functional improvement or stabilization for geographic atrophy patients.

The current therapies which are in the market in US, they use structural endpoints to get approvals. So we'll be definitely looking for functional endpoints, and some of that data will be shared next week in our showcase..

Got it. And a quick follow-up on for OCU410ST program.

Do you see -- given it is a rare disease, do you see any potential of modifying your Phase 2 trial or Phase 2 portion of the trial to make it a pivotal?.

Yes, that's a great question. That's why after Phase 1 clinical trial, we are taking a pause even though DSMB gave approval to move forward with the Phase 2, with the previously designed study approved by FDA. We are going to take a pause. We're going to spend a few months for discussions with the FDA because there are some new guidance.

There is an opportunity for orphan disease with the gene therapies to convert Phase 2 into a pivotal trial, for a registration trial. So we are going to closely work with agency and seek their advice and guidance before we move forward to Phase 2/confirmatory trial..

Got it. Thank you. And again congrats on the progress..

Thank you..

This concludes the Q&A portion. I will now turn the call back over to Chairman, CEO and Co-Founder, Dr. Shankar Musunuri..

Thank you, operator. We appreciate the continued interest and involvement of our key stakeholders as we move forward with our transformative initiatives. We look forward to closing a successful fourth quarter of 2024, as we continue to solidify Ocugen’s position as a biotechnology leader in ophthalmology. Have a great day..

Ladies and gentlemen this concludes today's conference call. You may now disconnect..