Greetings and welcome to the Abeona Therapeutics Second Quarter Results and Corporate Update Conference Call. At this time, all participants are in a listen-only mode. A brief question-and-answer session will follow the formal presentation. [Operator Instructions] As a reminder, this conference is being recorded. It's now my pleasure to introduce your host, Christine Silverstein, Vice President of Investor Relations. Thank you. You may begin.

Thank you. Good morning, and welcome, everyone. On the call today are Dr. Timothy Miller, President and CEO of Abeona; and Jeffrey Davis, COO of Abeona. Dr. Miller will start the call with an overview of the second quarter and more recent highlights at Abeona. After, Jeff will provide an additional commentary on the quarter and a brief overview of summary financials and provide a snapshot of our financial position and also review the upcoming investor conference calendar. Following that, we will open the floor up to few questions. Before I turn the call over to them, I need to remind our listeners that remarks made during this call may contain forward-looking statements that involve risks and uncertainties. Forward-looking statements on this call are made pursuant to the safe harbor provisions of the federal securities laws. Information contained in the forward-looking statements is based on current expectations and is subject to change and actual results may vary materially from forward-looking statements. Some of the factors that could cause actual results to differ are discussed in the reports filed with the SEC. These documents are available on our website at www.abeonatherapeutics.com. With that said, it is now my pleasure to introduce Dr. Timothy Miller. Tim, you have the floor.

Good morning, everyone calling in from the East Coast. Welcome to our second quarter call. We've got a lot of notable advancements in this past quarter and looking forward to updating everyone as well on even more recent updates as we go into September and October. So we continue to advance our goal of building a leading rare disease company with our focus primarily on gene therapies. Importantly, we've initiated our clinical frames for our ABO-102 program, which is for MPS IIIA or Sanfilippo syndrome, as planned. And we continue to begin the process for enrolling patients, additional patients at the U.S. site as well as opening up these sites in Spain and Australia. We have continued to drive the development of our library of next-generation AAV viral vectors, our AIM vector platform, for which we have global and exclusive rights of license. Now we have had a number of recent highlights, including some corporate achievements with the key hire of Dr. Juan Ruiz as our Chief Medical Officer. Well that's certainly a big step for our company. We have been searching for a CMO with rare disease and gene therapy experience for a while now. Juan brings with it also a business-savvy career. He's been involved with the EMA in discussing multiple gene therapy programs, has a number of successes under his belt as well and has been involved with developing patient-reported outcomes, particularly for dermal programs. So as we look to our EB-101 program, certainly welcoming Juan on board. For our EB-101 program, there have been some very successful and notable clinical achievements, notably that we've received guidance from the FDA to basically stop the Phase I/II clinical trial and commence our pivotal Phase III for EB-101 gene therapy for patients with recessive dystrophic epidermolysis bullosa, or RDEB,…

Great, thank you very much, Tim. As you may have noted, we filed our Form 10-Q for the second quarter on August 14th. Our cash position, our cash, cash equivalents and marketable securities as of June 30, 2017, was $58.3 million compared to $63.2 million as of the end of the first quarter and $69.1 million at year end 2016. The net cash used in operating activities in the six months ended June 30 was $10.8 million compared to $5.6 million in the same period in 2016. So our cash burn was a little bit lower in the second quarter relative to the first quarter but basically in line as to how we've guided this year. Revenues in the second quarter were $217,000 compared to $214,000 in the second quarter of 2016. Recall that the revenues consist primarily of a combination of royalties from our marketed product, MuGard, as well as recognition of deferred revenues related to upfront payments from licensing agreements. The loss per share is $0.21 per share for the second quarter of 2017 compared to $0.20 in the comparable period last year. So I refer investors and participants in the call to our Form 10-Q as it is publicly available. I would just comment a little bit on upcoming events and conferences because the schedule looks fairly tack here. As Tim has mentioned a couple of times, we will have our first R&D Day in New York City on October 11, where we'll have presentations from senior managers, clinical investigators and key opinion leaders. For those that are interested in participating, I suggest that you shoot an e-mail to Christine Silverstein, whose e-mail address is on the bottom of every one of our press releases. So I'm sure you'll be able to find her. In terms of investor conferences, we will be participating in the first week of September, right after Labor Day, in the 12th Annual Citi Biotech Conference up in Boston. We'll also be participating in the 4th Annual Biopharmaceutical R&D, BRDS, on September 7. We'll be participating a little bit later in September at the Cantor Fitzgerald 2017 Global Health Care Conference here in New York. And I think Tim mentioned, going into October, we will be present and hopefully presenting updated data on all of our clinical programs at both the ARM's Cell & Gene Meeting on the Mesa, which is in La Jolla on October 5 through 7; as well as, as Tim mentioned, ESGCT in Berlin, Germany, during October 17 to 20. So we'll have a lot of chance to interact with investors and stakeholders and others in September and October with a fairly dense conference schedule. So I think with that, I will turn it back to the moderator, and we will be able to take some Q&A.

Thank you. We will now be conducting a question-and-answer session. [Operator Instructions] Thank you. Our first question comes from the line of Maury Raycroft with Jefferies. Please proceed you’re your questions.

Hi, thanks for taking the question and congrats on the progress so far. So I was wondering for EB-101, for the Phase III trial, if patients will be exposed to other experimental therapies such as the Amicus program, SD-101. And will this give a read-through into whether these therapies are complementary? And how you envision positioning – these therapies to position in the market?

Sure, thanks, Maury. Nice to hear you, and great question. We're big fans of Amicus and their product. We've – actually, just as a side note, our teams have actually met a number of times to really kind of talk about the EB patients in general and RDEB in specific. Our product, I think, would be complementary with theirs. They do have a cleaning-based program that helps in wound healing, and ours can be used potentially with that. Ours is something that’s applied on top of the wound as an actual skin product. So it's what actually helps close the wound, and then the Amicus product genuinely helps to keep it moist and moist and heal better. And so we see this as complementary. I don't think that we'd cross anybody over from one program into the other one. That's just generally we wouldn't do that for this type of therapy, but we could see that at some time point in the future. Again, you know that our products are more specific to RDEB patients, where we believe the Amicus product has a little bit more pan-specificity. So going forward, we look forward to potentially trying to find more ways to work together, but as it stands now, these are separate products.

Got it. And for ABO-102, as far as endpoints go, so I know this is probably an evolving conversation with regulators. But just wondering if you can give some perspective on precedent for what the high bar to approval for an endpoint for approval would be and maybe a lower-bar endpoint. And maybe if you can give some perspective on precedent [indiscernible].

It’s a really exciting discussion. I mean, there have been a number of notable failures in the MPS IIIA and MPS IIIB space. And that's really taken the community very hard as products that they had hoped to have some level of access to. For us, you probably heard us talk about this triangle of efficacy where we talked about biopotency as measured by reduction in biomarkers, specifically the CSF heparan sulfate or urinary GAG, looking at actual reduction of disease manifestation from liver volume and then, of course, some of the neurocognitive tests. And when we look at some of the other programs that are out there in the rare disease space, particularly in the MPS field but some other lysosomal storage diseases, having that trifecta of efficacy, at least in the early stage, is rather remarkable. So we're going to be answering those discussions with the FDA. We have sometimes informal ones when we go into these meetings to kind of talk about that with them. But really, looking out now at six months and now going into one year, now the story becomes more about just how much more can you actually improve upon success. I mean, with these levels of doses that we're giving, we're seeing a 60% to 70% reduction, in addition to all these other things. So we look forward to extending and having those conversations with the FDA.

Great. And then for ESGCT, just wondering if the – or if your abstract has been accepted for that meeting. And then maybe if you can provide a little bit more on what we should expect to see there. So you mentioned during your prepared remarks some of the neurocog measures that you're looking at. And so if you could just give a little more color on what to expect there, and I'll hop back in queue.

Sure. We – to be frank, we submitted a number of abstracts. The fun thing about having so many programs is you get to submit to a number of these conferences for each one of your programs. And if I recall correctly, the ESGCT, one of the abstracts that will be presented there is on the ABO-102 program. And that will talk about a couple of patients that have gone through one year in Cohort 1 and a few patients that have gone through Cohort 2 and – again, in that trial. And so we have taken a – try to take a very hard line on not going out with data with individual patients, but we believe that that's really in the best interest of the patients as much as it is for the program itself. We'll be talking about what we've seen at one year from the neurocognitive to the biophysical to, again, some of the biomarkers. So it will be – so I think it will be a great update.

Okay, great. Thank you.

Sure.

Our next question comes from the line of Elemer Piros with Cantor Fitzgerald. Please proceed with your question.

Good morning gentlemen. So, Tim, just to amplify on the last question. So during the conferences in September, would you be able to reveal additional data on either of the programs? Or shall we wait for the Mesa and the ESGCT conferences?

Yes. No, that's a great question, Elemer. And yes, we'll be talking about additional time points. We'll be talking about additional patients. I think that there'll be more data to show between day 13 and day 19 for some of the patients in Cohort 1 and Cohort 1. So again, talking about some of the – any additional enrollments that happen by then, and certainly happy to talk at that time more about some of the – really the durability that we're seeing as well. So as we've seen a dose response now, now we start also talking about durability. So I think that will be – those will be fun conversations to have around the data.

I see. And in the RDEB program, if I remember correctly, you enrolled additional patients before this decision was made to move into the Phase III trial. Would you please remind us how many of those and then we might see early efficacy data on those?

Sure. So we enrolled a total of seven patients in the EB-101 Phase – in the Phase I/II. And those patients – so the first four are through – I think going through a year – two years, I'm sorry, and then the following three are at least through six months on the site. I believe that we actually have another abstract that's been accepted at ESGCT to talk about that as well. So we'll be talking about some of that at the – again, at the R&D Day. Just – again, it's a more of an update. Things continue to look the same, various states and continued wound healing. So we were pleased that the FDA accelerated our program. And as we move now into the Phase III, looking at an additional eight, ten, or twelve patients in that trial. So that's certainly where our focus is and prepping for that, getting all of our assays and manufacturing up online to incorporate that program clinically.

Okay. And just one question, and this is the last one, Tim. You disclosed that in the CLN1 program, there would be a combined intrathecal and IV administration of the drug. What is particular about CLN1 that you choose – that you chose to have this combined route of drug dosing?

Good question. So a couple of things. So CLN1, there are – very similar to MPS I where there are three generalized forms of it, Hurler, Hurler-Scheie and Scheie. CLN1 has a similar presentation. However, it's actually earlier lethal. Many of the kids with CLN1, if they have the most severe form of CLN1, really, many of them don't reach the age of three to five. So there's a – and as the preclinical studies have really recapitulated many of the things that we've seen for the MPS IIIA program, which is treating earlier and treating with more yield and better results, there are sometimes – just there's been enough damage done in these kids that it's very hard to kind of pull them back from the edge. I think you'd see a very similar story with the SMA1 programs, where I think some of the caps on enrollment agents, that might – is a little bit – is really at eight months. So when we look at routes of administration, the preclinical researcher, Steven Gray, who is going to be at the preclinical – at our R&D Day, he can talk about this as well. A lot of his work in the GAN program and in the CLN1 program is focused on intrathecal delivery. And he's been – and has shown to be very, very successful. But we have a strong penchant for IV delivery, and Steve has also tested some IV. So we kind of went down this collaborative path where we said, well, let's try them both. Let's try the different combinations of low dose and high dose. And we're currently in some of those studies now, how young you can treat versus how old you can treat with some of these dosing routes. And really, what we're trying to find, Elemer, is the best, most successful way to treat many of these patients, not just the younger ones, the ones that may have a disease progression. So we'll be starting with intrathecal and then moving upwards and then adding an intravenous dose on top of that.

And a somewhat related question, and this may not be reasonable to assume, but if you had a patient dosed with an IV of the same drug or the same construct, would you be able to theoretically follow-up with an intrathecal administration of the same construct? Or there would be neutralizing antibodies that might be shunting this type of response or potential response?

Yes, I love your line of thought, Elemer, and we thought very similar. And really, what you're proposing there is part of the rationale around the AIM vector platform. We know that with many of these programs, when you treat an infant, that this is going to last for years. But at some point, they may need some form of additional dosing strengths, whether you're looking at a disease with motor neuron function or, really, other – just CNS involvement. So we look at this as the potential to do – to get some of those answers. And we're working with some of our MPS III foundations as well to have some of those discussions about how can we be additive to the gene therapy field and looking at patients that have previously been treated with ERT or patients that have previously – that have neutralizing antibodies even without being dosed. And those will get us to the questions of, can we ask about a patient who's already been dosed? Can we go intrathecal and immunosuppress and treat that? So we will have, I think, more of those questions answered in the next year and looking forward to sharing that because, again, we believe that that's going to be transformative for the gene therapy field overall.

Great. Thank you very much.

Thanks Elemer.

Our next question comes from the line of Liav Abraham with Citi. Please proceed with your questions.

Good morning guys. First question is on your AIM vector system that you talked about in your prepared remarks and mentioned in your – one of our press releases last week. Can you just give a sense of time line to your – maybe what sort of data you have to date and whether it's possible that these enter IND stage in 2018?

Yes. So the – I mean, we saw a significant amount of data sufficient that we thought that this was worthy of – in terms of in-licensing and bringing in-house. But we also already saw the potential where we could go with this with multiple delivery routes. So as we look to integrating the AIM vector platform into our – certain vectors of the AIM vector platform into our existing pipeline, we do have some additional targets that we're considering in-house. I don't think there'd be any real surprises to anybody, but again, we've always tried to find new ways that these vectors can also demonstrate a hell of lot [ph] efficacy. So right now, they are currently in the discovery phase, where we are doing different routes of delivery and we're doing different routes of assessment for the vectors to identify what we consider the best tropisms for certain tissues or for certain disease types. So we would hope that something would be in IND-enabling studies in the second half of 2018. And as we really go up periscope on talking about some of the disease targets, we look forward to updating this phase and everyone on those programs.

And then just as a follow-up on disease targets, is SMA an indication that makes sense?

We haven't really commented publicly on any of the actual programs that we're looking at or developing the AIM vectors. So we are looking at muscle delivery. We're looking at CNS delivery. Certainly looking at the skin space for delivery into the skin space as well. So there are a number of targets and a number of diseases that certainly would make sense if you're looking at something in the lungs, for example. So certainly, when we come up and talk a bit more about the extra disease targets, we'll put those out there.

And then finally on this topic, maybe a question that I'm not sure you answered, but I'll ask it anyway. Just wondering if you've signed the CDA with Biogen because you did talk about looking at exploring opportunities, both internally and through strategic partnering on this platform.

Well, the nature of this CDA would preclude me from talking about who I signed it, this CDA with.

Yes.

I can't really comment on CDAs that we've discussed with any potential development partners. But I'm sure, as you can imagine, many different companies are looking for additional pipeline products and, certainly, to find new routes of administration for the next generation of AAV. So again, those things kind of come out in time. And again, when there's good data and good products, there's always lots of interest.

Glad. Thanks very much.

Our next question comes from the line Paul Mann with Highbridge [ph]. Please proceed with your question.

Just a quick question actually on the EB program. In thinking about the Phase III trial, as I know you're still in discussion with regulators in finalizing the format of that trial, but should we assume that EB-101 is going to be compared to a natural history study or is it likely to be an active comparator in that trial? And then when we think about numbers of patients involved, should we be thinking about tens of patients or hundreds of patients? And then in terms of the treatment period and primary endpoint, should we be thinking about kind of six months treatment period or 12 months treatment period or maybe even less? And then when we put all that together and start thinking about the regulatory submission, I mean, most of us have penciled in 2019, but is there a chance this could get to regulators in 2018 for approval? Thank you.

Let's see. So a couple questions in there, Paul. Nice to hear your voice, again, of course. So first, we are doing a matched intra-patient control for the FDA. And unsurprisingly, since you do this with small populations, especially in the rare disease space, looking at an untreated control wound on an individual patient that's getting treatment, so not too surprising. And we know that the cells, the skin that are applied on the EB-101 product don't really migrate out. So it's an appropriate control. We are looking at somewhere between eight and twelve patients in the pivotal Phase III. And we expect to follow-up for those patients to be between three and six months, noting that, and I think we commented on publicly before, when you look at some of the other skin substitute products or wound healing products for venous ulcers or diabetic foot ulcers, those are usually approved on a six-month time point and greater than 50% wound healing. So I don't think that we will be held any different, at least based on our conversations. And with regard to your last question, we'll see. Again, if we're able to enroll all of these patients in the next – in the first half of 2018 and it's a six-month endpoint, then you never know how things can pan out towards the end of 2018. So we'll put our best foot forward.

And then just one other one on EB, how do you think about pricing this product or reimbursement? Would you be thinking about a price per patch? Or the patients go into a disease management program where these patients go through hundreds of thousands of dollars of bandages a year. So I mean, is there an opportunity to put patients in collective disease management program? Or how do you think about that? Have you had any discussions already with regulators?

We're getting into those discussions now about some of the pricing and reimbursement and how we're modeling that out. The good thing about this product is that the patients can get it more than once. When we prepare the EB-101 program or product, we do freeze on cells so the patients can come back and essentially have another application of EB-101. So you could have – so they come in for three, they can come in for 12. So I think we'll be looking at pricing this on a per application. And as we move that program forward – we're working with some of the families. They'll show you pictures where they are literally surrounded by boxes of bandages. That can cost them – I mean, they get a shipment of hundreds of bandages each month. And as you mentioned, it can cost hundreds of thousands of dollars. And so we look forward to finding these routes that we hope that this is going to provide them with an ability so that their skin doesn't stick to their clothes, like we heard from a little girl just yesterday.

Great. Thanks so much.

Thanks Paul.

Thank you. We have reached the end of the question-and-answer session. Mr. Miller I would now like to turn the floor back over to you for closing comments.

Well, as we finish up the third quarter and go into the fourth quarter soon, there's a lot of near-term milestones that we'll be talking about: new data in Cohort 1, additional enrollments, starting to look at that data in Cohort 2 for the ABO-102 program. So it's really an exciting time, and we look forward to meeting with many of the callers on today's call at these investor conferences and happy to set up time. We'll direct to Christine if you have any questions. And again, as we move forward with the EB-101 program, going a little bit more outward-facing with some of the guidance that we get from the FDA once the design is set. So thank you, everyone, for joining us for today's call. That's all for me.

Ladies and gentlemen, this does conclude today's teleconference. You may disconnect your lines at this time. Thank you for your participation. And have a wonderful day.