Ocugen, Inc. Common Stock H.C. Wainwright 28th Annual Global Investment Conference

Thank you. And appreciate you for accepting our invitation and talking to our audience today.

Yeah. Thank you for having me to tell the Ocugen story and where we are. Obviously, it's a big undertaking when you have three phase III programs and they target Inherited Retinal Diseases and AMD, which is dry AMD, which is much bigger than wet AMD, right? These three programs encompass majority of the vision loss diseases today, which have a significant unmet medical need. All our therapies are potentially one-time treatments for life. That's going to be a monumental difference in patients across the globe. In the next 18 months, as you stated, the top-line data from OCU400 targeting broad retinitis pigmentosa, early to advanced stage, pediatric to adult, it covers all the patients. RP can be caused by multiple mutations.

mutations in over 100 genes. The goal is to cover entire population. Between U.S. and E.U., we got about 300,000 patients. It's large. Our goal is to get the top-line, get ready for BLA submissions. One of the most important thing we learn from the industry, mistakes or positives and negatives, the negatives are CMC.

We introduced two commercial scale lots in phase III. We don't take any risk from CMC perspective. Non-clinical and clinical, the CMC modules are ready to go. As soon as the clinical data comes out, we'll discuss with FDA, we'll try to file that ASAP. That'll also prepare us to follow that with the market authorization in Europe. Why? Because EMA waived clinical trials for both RP, OCU400, and Stargardt, OCU410ST. They don't need any additional work. The clinical trial in U.S. is good to file there. We'll follow through. What does it mean? I think next program is Stargardt in a few months later, if everything goes according to our plan.

And again, we will get the top-line, we will file the filings in U.S. and EU. GA is a large disease burden. Thanks to FDA, from end of phase II meeting to dosing patients in phase III, record time. I think very proud of my team, within four months. Not only FDA reviewed it, they gave Regenerative Medicine Advanced Therapy designation, which is given based on the promising data in early stage and significant unmet medical need. There is a significant unmet medical need, even though there are a couple of products in the market.

in U.S., which do not exist in EU. That program, the clinical trial, we assume about 9 to 12 months of recruitment. By 2028, second half, it should be done. As soon as the top-line results come, we will file the BLA. We will be ready. The RMAT designation gives potential priority review. Instead of waiting for 10 to 12 months, regular therapies, you can get an accelerated. That is a big plus for a large disease like that. What it means, number one, we need to be very good with our filings, getting them on time. There is an old saying, "Get it right first time." We want to do a good job. We want our team to do a good job so everything runs like a clock with FDA and EMA.

If a quality of filing is good, we can get through it. Number two, we need to prepare for commercialization. Obviously, you are seeing the deals we are announcing ex-U.S. Obviously, even though some of us came from large pharma, as a growing biotech, it is not practical to think we will go and launch everywhere. We need to be prepared. We will definitely focus on strategic partnerships outside of U.S. In U.S., first and foremost, there are multiple things we are doing even before top-line results come out, which we can do without spending a lot of capital. Looking into, number one, talking to payers, including CMS, lining the top and creating centers for excellence. Because vitrectomy, all our therapies go after subretinal injection. Even though all retinal specialists are trained, it is a small surgery. It is not rocket science.

However, we did very well with our clinical trials.

More than 300 patients dose. Among all our trials, we do not have any SAEs related to our product, the gene therapy. On the surgery side, we mastered something. We want to make that cookie cutter. We want to create centers for excellence for delivering those gene therapies so that not every specialist touches.