Good day, ladies and gentlemen and welcome to the Abeona First Quarter Earnings Call. [Operator Instructions] It is now my pleasure to turn the floor over to your host, Gregory Gin, Vice President of Investor Relations and Corporate Communications at Abeona. Please go ahead.

Thank you, Holly. Good morning, everyone and welcome and thank you for joining us on our first quarter 2021 conference call. Press release announcing the first quarter results and recent operational progress is available on our website at www.abeonatherapeutics.com. On the call today with prepared remarks are Michael Amoroso, Chief Executive Officer of Abeona and Ed Carr, Chief Accounting Officer. After the prepared remarks, we will host the Q&A session, where we will be joined by Dr. Juan Ruiz, Vice President at Abeona, who heads up Clinical Development for our MPS programs and Dr. Brian Kevany, Lead Research Scientist working on our preclinical eye programs. Before we start, I will review our Safe Harbor statement. Remarks made during today’s call may contain projections and forward-looking statements regarding future events. Forward-looking statements are made pursuant to the Safe Harbor provisions of the federal securities laws. These forward-looking statements are based on current expectations and are subject to change and actual results may differ materially from those expressed or implied in the forward-looking statements. Various factors that could cause actual results to differ include, but are not limited to, those identified under the section entitled Risk Factors in the company’s Annual Report on Form 10-K and quarterly reports on Form 10-Q, filed by the company with the SEC. These documents are available on our website at www.abeonatherapeutics.com. And with that said, I will now turn the call over to Michael. Michael?

Thank you, Greg. Good morning to our investor community who are excited to spend some time with you this morning, thank you for joining us. As I approach the midpoint of my first 100 days in the job as CEO, I wanted to start by reviewing three key strategic priorities for Abeona and our team. First priority being to bolster the relevant operational experience on our management team and board, making sure we have the right coaches in place, the right experience to meet our goals going forward. Second, delivering operational excellence, both timely and fiscally disciplined to further advance our in-clinic programs towards meaningful milestones for patients in need. And third, to further prioritize, execute and advance our preclinical eye programs toward the clinic. We have made substantial progress on these priorities and are off to a fast start in ‘21. I want to thank the team for their efforts. First, priority one, continuing forward under the right leadership. We continue to focus on building the right talent and experience on our team, which positions us well to execute against operational goals. I am very excited just this morning to have announced the appointment of Dr. Vishwas Seshadri, a Senior Vice President and our new Head of Research and Clinical Development. Vish, as he goes by, brings more than 20 years of experience across academia, followed by various senior and executive level leadership roles within the life sciences industry, overseeing product development, regulatory strategy and teams for submissions, and commercialization for novel therapies, including most recently personalized autologous cell therapies in the CAR T space for Celgene and BMS. Vish has significant experience across early and late-stage development from first patient in, all the way through successful commercial launches. He understands the trade-off decisions and necessary discipline for evidence-based…

Thank you, Michael. I would like to remind everyone that the Form 10-Q is available on our website, which is where you can get the details on our Q1 2021 financial results. In summary, our cash, cash equivalents and short-term investments as of March 31, 2021 totaled $86.8 million compared to $95 million as of December 31, 2020. For the first quarter of 2021, net cash used in operating activities was $13.6 million compared to $13.2 million for the first quarter of 2020. We believe we have sufficient cash resources to build on our momentum and fund our current development and operating plans through achieving key anticipated milestones, including multiple potential regulatory submissions. And with that, I will now turn it over to the operator for Q&A.

[Operator Instructions] Your first question for today is coming from Maury Raycroft, please announce your affiliation and pose your question.

Hi. Good morning everyone. This is Maury Raycroft from Jefferies on the line. Thanks for taking my question. So, first question is on EB-101. Just wondering if you could provide any more specifics into why the product didn’t meet release criteria for patient #5, and I think you mentioned comparability program, can you just remind me what the purpose of that is and often I guess what’s the outcome of that? I think you mentioned comparability program in FDA, but I missed a little bit of what you said in the middle of that?

Sure, Maury. Good to hear your voice. It’s Michael, I will take that one. Yes, so patient 5, we have got about 13 different release criteria that obviously the product goes through before we are able to send over and release for the surgical administration. The patient cells as you remember we grow the cells up and we use an enzymatic cleavage to take the sheet off and put it on our galls with hemoclips to deliver it to the OR. The protocol for pivotal states that the sheets can’t even have any pinholes in the middle, that being said while the surgical administration occurs, we do make sutures in the product obviously to put these grafts on to patients. But the protocol with the FDA and the agreement as of today, unlike it was in the Phase 1/2 says you can’t even have a pinhole in these sheets, the sites are known to close over themselves within their borders to kind of cover these pinholes. So this is something we are working on in the Phase 4 work, but it is a pivotal trial they register a new criteria, which we appreciate. So, we had some holes in the products, small pinholes. And at that point in time what we decided to do was rather than yield lesser products to the OR, we decided to re-biopsy to try to give the patient the best ability to have a maximum delivery of transplanted area. So, this is something that we with autologous therapy, cells grow a bit differently. We have seen this before. And this is not something that’s uncommon to us. But this is why we decided to re-biopsy the patient. To answer your comparability question, just a reminder, all of our product is made in-house. Our tech ops in Cleveland goes end-to-end. There is no CDMO dependency. However, right now as part of the ingredients for transduction, we use academic or Indiana retrovirus, okay. And in order to have full control of our supply chain as we move to commercialization, the team in Cleveland has built our own retrovirus, Abeona retrovirus. So, the comparability is the plan that was agreed upon with the FDA that within the pivotal we will move from the first half of our patients that are made with products from Indiana retrovirus to the Abeona retrovirus. So, while we are still treating patients clinically with the Indiana retrovirus ingredient, that’s – it helps us transduce and make accurate tenocyte sheets. At the same time, we are doing analytical comparability to show the similarity to the Abeona retrovirus and our final 5 patients will be dosed with the Abeona retrovirus made product. So, that’s why we have the comparability protocol, Maury.

Got it. It makes a lot of sense and thanks for the all the perspective there. Second question is just on the Transpher A type B meeting with FDA in June, just wondering if you could talk a little bit more about the plan going into the meeting, who is going to be attending with you and also the disclosure from the meeting to I guess, will you get minutes from that and when could we expect some sort of an update from that meeting?

Yes, sure. So Maury, I am going to turn this question to Juan, I will give you a quick highlight, I am going to turn it over to my clinical lead of the program, Juan Ruiz is on the phone. But I think the key here is analyzing our pivotal endpoint, our data and making sure that we broach that topic we have talked before very important that natural history becomes we think we have got the biggest dataset here that natural history becomes a viable control arm for a path forward to a BLA. But Juan, why don’t you give a quick overview of what we look to accomplish objective wise in the Type B meeting towards the end of June and some of the partners that will join us with the FDA.

Yes. Thank you, Michael. Thank you, Maury for the questions. We have got previous meetings with the FDA and we have been implementing the outcomes of those on recommendation from the FDA. But the main purpose of the meeting this June is to agree with the MDA on the way we propose to analyze the primary endpoint, the use of natural history study as a comparator group precisely to analyze the changes that we see in patients treated with ABO-102. And also what is clinically meaningful difference that we propose in order to declare success of the program, those are the main aspects that we are going to discuss in additional to the use of putting together different natural history studies, testing different tools. We have the data to support that and we have discussed this statistical analysis that we have provided those are mainly the aspects we want to discuss. As I said that have been previously discussed implementing some of the recommendations on providing the answer to pending questions.

Thank you, Juan. Got it. That’s helpful. And so could we expect an update potentially in July-August timeframe as the outcome from the meeting?

Yes, Maury. I think on our next call, we will definitely be talking about the results of our Type B meeting. That will be an important task for us to communicate.

Okay. Thanks for taking my question.

Thank you.

Your next question is coming from Kristen Kluska. Please announce your affiliation, then pose your question

Hi, good morning. This is Kristen Kluska of Cantor Fitzgerald. I wanted to ask a question on EB-101. So, on the back of some of the recent data you shared at SID, how are you thinking about the durability of EB-101 across the different wound types evaluated? And how important do you think this durability data is going to be for physicians and the sales team to promote in choosing a potential treatment option should multiple therapeutics become approved and I know there is a lot of other factors to consider here like wound type and size, but I specifically wanted to hear your thoughts on durability?

Yes, Kristen, nice to hear your voice. Thanks. I appreciate the question. So, you are making me smile because I know we have talked a lot about this. So yes, I mean, I think you said it really, really well, right? So, a couple of things. First of all, we are hoping – very much hoping that there will be many, many options here over the next 5 years, 10 years for patients with RDEB. I mean we have had nothing in this space for the last 3 decades to 4 decades. And that’s not given us the progress we need for these children and young adults. The backbone of EB-101 is durability. That’s the positioning of the product. The reality here is, there is a small surgical administration like a graft. And the product has to be worth it, and we believe it very much is. The focus of our product in our trial is in RDEB, the worst of the worst types of EB, as well as the worst type of wounds, about – we say about 50% of the wound of RDEB patients at any given time, is a more advanced wound, large 20-centimeters squared or greater. We have some that are hundreds of centimeters and tire backs, for example and chronic, this being a very important point, Kristin, that you brought up, different wounds. Chronic is a wound that can no longer heal itself. 6 months or longer, it’s been open. These are the wounds that patients suffer the greatest morbidity, pain, infection, malnutrition as well as lead to things like squamous cell carcinomas and down the line, and these are the things that unfortunately lead to patients meeting their demise. So we are focusing on what we believe is the worst of the worst wounds,…

Thanks. And I know you’ve discussed the key differences between the recur and chronic wounds, but I wanted to ask if your researchers and your team are doing some more work to better understand when the best time to intervene with this treatment might be for some of the recurrent wounds, excuse me, specifically, just in light of the fact that the different patterns in terms of time to healing, time to re blistering, etcetera, just to help with the greatest chance of success if commercialized?

Yes. It’s an excellent question, Kristen. I think the question you ask here is, what is earlier intervention? Why let these wounds get to this point, right? If we’ve got tools, what is the right time for intervention. So we are – the scientific question hypothesis is out there. We are asking the question. We do plan on studying it in the future. Some of the things like I talk about our Phase 4 work a lot. Some of that Phase 4 work I always call those practicality studies, things that are really important to add to our pivotal package, things like showing, hey, a sheet with a pinhole can absolutely be transplanted and have the same durability, keratinocytes will close over. Things like getting patients out of the hospital faster because we know patients who have the right care at home we want them to make sure they have that option. So they don’t have to stay in OP. Right now, they stay in the hospital. Just so a wound nurse to make sure they are not laying on their engraftment. Things like Kristin where you just were, recurrent wounds. If we can treat the most problematic wounds, we have a lot of hypothesis that says EB-101 would be able to do really well with recurrent wounds. We absolutely believe that, but we will look to prove that in the future. As you know, we’re starting in our RDEB in large and chronic. But these are things that I call kind of our complementary Phase 4 work, work that bringing Vish on now as our Head of R&D will be really important that we start to continue to do the studies that matter to show this data.

Great, thanks. And then on your prepared remarks, you noted that some patients for the Phase 3 trial have been identified or in the prescreening process. Just wanted to make sure I understood that this was specifically referring to Sanford. And then I know you haven’t identified the specific center in the Northeast but could you talk about maybe more details? Like is this a center that you would expect a lot of patient interest and have the investigators there potentially identified patients that they might want to go through the screening process?

Yes, Chris, you nailed it. That’s exactly right. So we’ve got some patients that are identified for Stanford but we also have started a bench for the Northeast site. And I don’t mean to be coy guys. I’m just not announcing the site as per their IRB ask until it’s fully approved. I hope the next time we talk, I’ll be able to do that. But a site in the Northeast is also screening and looking for some patients right now, patient charts, their KOL there. When you think about what that site would look like, what’s an ideal site as we onboard, we’ve talked about a few – in the past, you might remember, we will probably come out of the gates with at least 10 sites when we commercialize. But when you think about the second study site, an important step in progress toward that, these are EB clinics, ongoing EB clinics. Why? This is where we currently have EB patients today. This is where you see the evolution of the disease of these patients when they need products like EB-101. This is where you have anesthesiologists, surgeons who understand how to give anesthesia to a patient who may have an esophageal stricture. The dilation procedures go on there, the hand foot surgeries go on there. So right now, these are academic type centers, who are specializing in EB, and they have the volume of the EB patients. That gives you an idea of what the profile of that Northeast site will be very similar to Stanford. And yes, when we talked about the additional patients being prescreened right now, we’ve got a few in the queue. We hope they meet eligibility criteria that’s out of Stanford. But our lead KOL out of the new site is also profiling some patient charts. So once we have the site ready to move, we can go as fast as we can to get those patients into the trial and treated. And what I’ll remind you is our capacity out of Cleveland, while we’re in clinical stages, we could treat up to two patients per month. So, that’s really, really important. And as we commercialize, we will be making some configuration to our site. We will be dedicating our first site solely to EB-101 and at that point, we will be able to produce about 120 products in the first year, moving to about 500 products if we have the demand in the second and third year. So we’ve got the scale to be able to meet two sites ongoing as well as getting ready for first year of commercial launch.

Great. Thanks so much for taking my questions.

Of course. Thank you.

Your next question is coming from Mani Foroohar. Please announce your affiliation then pose your question.

This is Mani Foroohar from SVB Leerink. Thanks for taking the call. So a lot of the focus, obviously, on the existing clinical opportunities, I went through it a little bit to the vector platform and if you see and how you think about further opportunity in terms of be the reason further non-dilutive capital and partnerships using any vectors that you guys already own currently? And how do we think about that potential store value for the company?

Yes, Mani, how are you doing? Thanks for the question. So good question. And I think the key word for me that jumps out is optionality, right? We are a small cap. We’ve got the funding right now and the experience and the capital discipline. As Ed said, that we believe we could take ourselves through our first clinical programs, our in clinic programs to literally regulatory milestones if need be. But at the same time, I’ve said this before, we’re always looking for is to bolster our either our capability, which I always think about is our timing to patient, our science and/or our financial opportunities, our financial runway, right? That’s – Mani question that’s really important. Our financial runway is a reality that we think through every day, and we try to be very surgical, if you will, in our approach. So I think when you look at our profile, if you think about the identity of the company, and I love this question, I think our identity is an end-to-end fully integrated gene and cell therapy company, cell therapy, of course, being within the umbrella of gene. And that starts with an autologous program. But I think then you immediately see, and that’s why I think the MPS program is so important for human beings, first and foremost, but also so important to prove some muscle of our AAV platform on non-autologous gene therapy program. And then from there, I think about the AIM capsid library and the follow-on on how that’s yielding some early excitement for us in the eye. And that’s the way we’re looking at them, one, is a progression now that our identity is a gene therapy company, that we are – we look at the ability to produce for MPS product. The…

Thanks. That’s really helpful.

Thank you.

I would now like to turn the floor back over to Michael for any closing remarks.

Well, thank you, operator. Thank you for the operator of the call. Thank you for Greg, for setting it up and Ed and the team. Thank you to the Abeona team that’s made really significant progress since I’ve been working with them in late last year. I want to always thank our patients and our families for having the faith and trust in us for their children or their loved ones. And we will stay committed. Thank you to the investor community for your interest, your honesty, your questions will continue to be transparent with you. We will continue to update you on progress as we make those milestones. And if no other further questions, then I turn it over to you operator to dismiss us today. Thank you, and look forward to chatting again soon.

Thank you, ladies and gentlemen. This does conclude today’s conference call. You may disconnect your phone lines at this time, and have a wonderful day. Thank you for your participation.