Abeona Therapeutics Inc. Common Stock H.C. Wainwright 28th Annual Global Investment Conference

Thank you, Josh, and good afternoon, everyone, and thank you for tuning in. Excited to talk about Abeona and our story with you all. Abeona is a company that has made a commercial transition into a commercial stage company with the approval of ZEVASKYN and its launch late last year. My goal is to share with you all with regard to where we are with our progress and why we are excited about positioning the company for long-term discipline growth. Next slide. Before we begin, please note our forward-looking disclaimers. I will share our current progress and the outlook, and those should be considered in the context of the risks and uncertainties we have outlined in the S.

As many of you know, Abeona, we are a commercial stage cell and gene therapy company, pioneering medicines for the highest unmet needs with multiple potential value-driving events for the company coming up. Our flagship product is ZEVASKYN, which was launched for recessive dystrophic epidermolysis bullosa. Patients with a lot of chronic wounds that leads to complications, and they do not live normal lives, these patients. Ours is the only autologous cellular sheet therapy that addresses the gene deficiency. The launch is well underway. We will discuss more about that in the upcoming slides. Alongside ZEVASKYN, we have also partnered programs with Ultragenyx, and it is noteworthy because we are keeping our fingers crossed. Ultragenyx has a PDUFA date for the UX111 for Sanfilippo syndrome type A. We also have a partner program with Taysha for Rett syndrome that is also progressing through their pivotal studies.

For Ultragenyx, we are eligible for commercial milestones, sales milestones, as well as tiered royalties up to single digit 88% of milestones, clinical and regulatory milestones with Taysha product, as well as commercial milestones and royalties as that program matures. In addition to that, we have partnered our ophthalmology AAV204 capsids with Beacon Therapeutics. Beacon has a few undisclosed targets, largely in the prevalent and rare retinal diseases. Those programs are in preclinical stages, but Beacon is also advancing their assets using our capsids, which we have licensed to them in a non-exclusive basis. In addition to the partnered programs, we actually were excited to acquire an asset, PSMA-SIR-T, Synthetic Immune Receptor T-cell technology, earlier this year. Preclinical stages, but it has a tremendous potential setting with a high unmet need, and we believe it is a very well-differentiated asset.

Of course, early stages, but we have many exciting catalysts that are on the way in addition to the growth that we expect from the launch of ZEVASKYN. Let me regress that we shared on August 13th. In terms of the growth itself that you can see that we have reported three quarters of relaunched starting in December. There is the first quarter and second quarter, and the most recent growth of 31% quarter-over-quarter with $11.4 million in revenue. But more importantly, we are actually setting a solid foundation for launch this year with a growing footprint of qualified treatment centers. We have seven centers that actually have onboarded with ZEVASKYN, and that is important because, for these patients, they like to get treated in centers that they trust, especially given this is a surgical procedure.

Having good name institutions that care for these patients in the EB setting is extremely important. Our most recent center that we activated was Cincinnati Children's, which is one of the largest in the U.S., is also globally recognized. With this footprint of seven centers, we now have about 40% of the addressable market of the patients are living in the states where the qualified centers are located, which actually helps with patient access and the market access. In addition to that expansion of the sites, there is a very high unmet need for these patients because onboarding a treatment site takes significant time and energy. We have identified more than 100 patients as well as the non-qualified center, the community physicians.

The main bottleneck right now that we are working through is trying to reduce the administrative time that it is taking from a patient identification to placing these patients on the treatment. That is typically expected for any autologous cell gene therapy, especially if it is a multimillion-dollar product where you need to establish medical necessity, need to establish single case agreements between the centers and the payers. Those administrative times are getting better. As more patients get treated in a particular center, their experiential curves also builds, and that is exactly what we are seeing. In the first quarter, we reported Children's and Stanford that was treating. On our second quarter call, we mentioned CHOP has treated a patient, and UTMB in Texas has also biopsied. We are seeing more centers that are beginning to treat these patients.

Despite these early launch learnings that we shared, we are happy to see a steady growth in patient treatments with 12 patients that we have commercially treated as of August 13th. From a launch foundation standpoint, in addition to qualified centers, we also have a pretty robust market access with more than 95% of commercial lives covered. We have got Medicaid covering all 50 states are covering ZEVASKYN. We have not seen a single ultimate denial yet from an insurance company perspective for coverage of ZEVASKYN. These are all the basic ingredients that we are taking on for this particular year, and we are seeing the momentum build with patients that are interested in stepping forward. We are well-resourced with $146 million in cash that we expect to fund our commercial launch, as well as the ABO-701 IND work.

That said, let me spend a few minutes talking about ZEVASKYN opportunity and why we believe there is significant value that remains to be unlocked. This particular slide picture speaks a thousand words. You can see that the extent of wounding that are in these patients, these recessive dystrophic EB patients with skin, and skin being the largest organ in the human body, there is a substantial portion of the person's wounded surface area. In the absence of a functional gene, skin can blister easily, and once the skin is opened, there is a high risk of infection, pain that needs to be managed, more than 90% risk of developing squamous cell carcinoma, and a total lifelong burden. There are topical gels on the market, but topical gels, just physically, there isn't enough amount of dosing.

There is a dosing limitation to be able to cover these wound areas. If you're trying to treat this entire back, it can take inordinate amount of product, topical gel, as well as repeat application. Whereas with ZEVASKYN, we are the only gene therapy that genetically modifies patients' own skin cells to treat both adults and pediatrics. We have shown a single surgical application has a potential to cover multiple wound areas. In clinical trials, ZEVASKYN has shown years of wound healing and pain relief. How does it work? The product, autologous manufacturing process. The process starts with taking a biopsy of the patient. We take two 8-millimeter punch biopsies and we bring the product to our manufacturing facility. We are a Cleveland, Ohio-based company. It's a 25-day process of gene modification and cellular expansion.

Eventually, with those two pea-sized biopsies, we are able to scale and provide up to 12 credit card-sized gene-modified skin sheet, which are then placed onto the patient's open wound areas. If the patients have discrete individual smaller-sized wound areas, those sheets can be applied, or they can be placed next to each other like tiles to fill up larger that we can offer to patients. In the clinical trial setting, we had a maximum of six sheets that we were able to deliver. In commercial setting, we are averaging nine sheets, and that's a substantial surface area of coverage, both for adults as well as for pediatric patients. Our pricing is not on a per-sheet pricing basis. Our pricing, it's a flat fixed price, regardless whether we give four sheets minimum or up to 12 sheets.

There is a lot more certainty when it comes to revenue for that particular product. In terms of the wound healing itself, here are the images from multiple different anatomic areas from our pivotal clinical trials. On average, these wounds in clinical trials had remained open for five years. Some of the wounds had remained open for 21 years. You can see a meaningful difference here with a single surgical application at the end of six months. Not only have we shown significant healing, but also significant pain reduction in the treated areas, which is quite substantive for these patients. Many of these patients have come back, areas of the body which in and of itself speaks volume. We often get questions about how many sort of the makeup of the commercially treated patients.

We can say that right now what we are seeing are many of these patients have already been on Bijuvac, have been on FILSUVEZ, and those patients are receiving ZEVASKYN. The youngest that we have treated right now in commercial setting is a 2-year-old. In the clinical trial, the youngest we had was a 6-year-old. We are beginning to expand our total pool of the profile of these patients, and especially in the younger years, if you are able to cover those kinds of wounds, it can have a significant value for these patients and the caregivers. These patients, they do travel. They do travel because even in the status quo, there are multiple complications from GI dilatation, squamous cell carcinoma, so on and so forth. They want to travel to tertiary centers that they can trust.

This is an example of a market research we had done even before launch of ZEVASKYN, where roughly 40% of the patients, they live outside of the state where the qualified center is located. About 70% of the inbound referrals are coming from patients that live. That is the level of importance that patients and caregivers give to going to the best centers that is possible. That is exactly has been our strategy of bringing on some of the leading centers and patients get the best care. With our footprint, we have to bring on 7 end of this year, and we continue to remain engaged in bringing additional treatment centers on board. Our most recent, as I mentioned, was Cincinnati Children's Hospital. We have provided travel support and patient support programs for these patients to make their travels to these qualified centers possible.