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Edited Transcript of ABEO earnings conference call or presentation 22-Aug-17 2:00pm GMT

Thomson Reuters StreetEvents

Q2 2017 Abeona Therapeutics Inc Earnings Call

Dallas Oct 10, 2017 (Thomson StreetEvents) -- Edited Transcript of Abeona Therapeutics Inc earnings conference call or presentation Tuesday, August 22, 2017 at 2:00:00pm GMT

TEXT version of Transcript

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Corporate Participants

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* Christine Berni-Silverstein

Abeona Therapeutics Inc. - VP of IR

* Jeffrey B. Davis

Abeona Therapeutics Inc. - COO

* Timothy J. Miller

Abeona Therapeutics Inc. - President, CEO & Director

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Conference Call Participants

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* Elemer Piros

Cantor Fitzgerald & Co., Research Division - Analyst

* Liav Abraham

Citigroup Inc, Research Division - Director

* Maurice Thomas Raycroft

Jefferies LLC, Research Division - Equity Associate

* Paul Mann

Highbridge Capital Management - Analyst

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Presentation

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Operator [1]

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Greetings, and welcome to the Abeona Therapeutics second quarter results and corporate update conference call. (Operator Instructions) As a reminder, this conference is being recorded.

It is now my pleasure to introduce your host, Christine Silverstein, Vice President, Investor Relations. Thank you. You may begin.

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Christine Berni-Silverstein, Abeona Therapeutics Inc. - VP of IR [2]

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Thank you. Good morning, and welcome, everyone. On the call today are Dr. Timothy Miller, President and CEO of Abeona; Jeff Davis, COO of Abeona. Dr. Miller will start the call with an overview of 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 to a 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.

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Timothy J. Miller, Abeona Therapeutics Inc. - President, CEO & Director [3]

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Good morning, everyone calling in from the East Coast. Welcome to our second quarter call. We've had 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 can 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 sites 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. sites, 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 to sublicense.

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. 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 the EB-101 gene therapy for patients with recessive dystrophic epidermolysis bullosa, or RDEB, which is the most severe version of epidermolysis bullosa. And it's really a wonderful thing for us to be able to have this therapy. There's obviously a large clinically unmet need for this. There are only a few other programs in development, some of which that we believe we'll be complementary to. But when we apply our EB-101 product, it is actually the wounds are closed from the beginning. And as we prepare to initiate our Phase III clinical trial, we're meeting with a number of our patients as we hope to continue to develop patient-reported outcomes and working on our manufacturing strategy.

Speaking of which, we do have some updates for our manufacturing strategy, plans in progress or in the development of this clinical and commercial-grade production facility, which will be provided at our R&D Day on October 11. So Jeff will talk a little bit about that. I'm happy to answer some questions at our R&D Day. We're certainly very fortunate that, with 3 clinical-stage programs and 2 more coming up as well as the vector program, to be able to have a significant R&D Day.

Data from the EB-101 program was presented at the recent Society for Investigative Dermatology Conference, where, as we've reported out, we demonstrated significant wound healing, we define as greater than 50%, in 100% of the treated wounds at 3 months, 89% at 6 months, 83% at 12 months and 88% at 24 months with -- again, going out to 3 years post administration. As we looked at natural history studies and how these are used to help us support our clinical paradigm, especially as we go forward potentially with a BLA filing, the clinical endpoints are supported by the natural history study that looks at almost 1,500 wounds from over 128 patients with RDEB, that it's certainly been notable in the FDA's commentary and the discussions we've had with them. And even meeting with some of the patients just yesterday in Cleveland, there -- these are the type of therapies where these kids wake up and they're afraid that their clothes are going to be stuck to their skin. And you hear stories from these parents about how they've had to actually cut the pajamas off of their kids and then soak them in olive oil. These are painful, itchy. And even these kids then, upwards of 60% of their body area in wounds that are bandaged that cost hundreds of thousands of dollars a year. So we are very excited to begin the pivotal Phase III.

Some of the regulatory achievements in this program for EB-101. We do have the orphan product designation, and we are expecting to hear soon about breakthrough therapy, on a presumption that we do get it for some of the other programs that will enable priority review and help us initiate the Phase III trial program mainly for the ability to have much faster turnaround on questions with the FDA. So that data was based on the Phase I/II for the EB-101 program. And while we also have the rare pediatric disease designation, we do note that we have the U.S. and orphan drug designation both here and abroad in Europe. So those are, of course, part of the prerequisite program for the priority review process, and we look forward to, hopefully, announcing breakthrough status coming up very soon.

Moving on to our ABO-102 program, which is, again, our program for MPS IIIA or for Sanfilippo patients. We announced data from a top line on the Phase I/II trial at the American Society for Gene and Cell Therapy Conference, where we saw a positive dose response in the CNS, or central nervous system, with a 60 -- about a 60% reduction of the disease causing heparan sulfate GAG. And that's very exciting as we see that dose response going from Cohort 1 to Cohort 2. The mantra in gene therapy is that, particularly for these lysosomal storage diseases with AAV vectors, that you want to treat with more and you want to treat earlier. So we are certainly excited to continue our enrollment in this program.

We have seen reductions of disease manifestation observed by decreased liver volumes and decreased wound volumes. It's been well tolerated in all of the subjects to date. It's more than 1,700 days of cumulative follow-up with no drug-related serious adverse events, which we find quite notable. And as we start to look at the nonverbal IQ scores and the adaptive behavior exams versus those Leiter and Vineland scores, certainly at 6 months, we did see some evidence for stabilization or improvement. And now as we look to 1-year data starting to roll in for the first cohort and 6 months' data coming in for the second cohort, these are going to be presented at upcoming conferences in September and October, notably the European Society for Gene and Cell Therapy. We have seen these improvements in clinically relevant biomarkers. And again, looking at -- if we consider this triangle of efficacy, looking at biomarker reduction, changes in pathophysiology and then, certainly, neurocognitive scores. And we believe those are going to be -- we'll be having discussions with the FDA in the fourth quarter about our chemo registerable endpoints. I look forward -- we look forward to providing more fulsome clinical updates at these conferences coming in, in September and October.

Just a note and really remind, we do have fast track status for the MPS IIIA program for ABO-102. And we have, again, the orphan product designations in Europe and the United States as well as pediatric designations. So we do -- we have announced recently the planned expansion that we'll enroll an additional 8 to 10 MPS IIIA subjects. And so we're hoping for somewhere between 12 and 15 subjects completely enrolled in first quarter 2018. And that will be across all 3 sites, both in the United States, in Europe and in Australia.

In the interest of time, moving on, I'm sure there'll be a number of questions towards the end, but we'll talk a little bit now about our EB-101 program for Sanfilippo syndrome type B, or MPS IIIB. As an update on the time line, we've completed all the necessary manufacturing for clinical material. I anticipate that enrollments will begin very shortly now and we are very pleased of that. We have had that IND for a while now, and as with many of the programs in this space, looking forward to starting those enrollments. That is also a program that has been granted orphan product designation in the U.S. and the rare pediatric disease designation as, again, part of the PRV process.

So again, we'll be looking to enroll 3 patients in Cohort 1 there and probably 3 to 9 patients in Cohort 2 in the MPS IIIB program. Our dosing is a little bit higher. Just to remind everyone, that's a single-stranded vector rather than self-complementary. And again, there has been a lot of preclinical data that's been published. If you have any questions, please contact us, and we're happy to send you the papers for either one of those programs.

For our preclinical development programs, we do have 2 that are getting into the IND-enabling phases for juvenile Batten and infantile Batten. So juvenile Batten is the CLN3 version, and infantile Batten is the CLN1 version. We do get questions every now and then. To help clarify, yes, we have 2 Batten programs. One is CLN1 and one is CLN3, just to differentiate from some of the other programs that are out there. We received the orphan drug designation for ABO-201, which is for the juvenile Batten disease program. And we'll start that process for the CLN1 program very, very soon. So for the ABO-201 for CLN3, we are in IND-enabling studies. Things have gone very, very well. There's been no animal deaths attributed to the drug. We go through a number of time points that have been negotiated with the FDA. They did allow us to submit the IND at certain points through the IND-enabling studies. And we look forward to doing that either later this year or the beginning of next year.

Similarly, for the CLN1 program, there's a lot of excitement around our CLN1 program. It's a very difficult disease to treat, but we're using the same paradigms we have for our other programs, again, using an IV delivery of an AAV vector. But we would like to note that this is a -- actually, a combination delivery program where we'll be using both an intrathecal and an intravenous delivery. So there are not many programs out there that have really moved down the path of trying to do combination delivery. And I think that if we look forward 3 to 5 years from now, I think that many of the programs that have a CNS component will be -- as well as systemic issues or manifestations of the disease, will be talking about treating infants because there'll be newborn screening tests approved and probably through multiple delivery methods for both intrathecal and intravenous. So we look forward to providing an update for that in the first half of 2018 as we have some regulatory guidance from the FDA, but we do note that there is some precedence for this out there. And again, there is no program, no approved program out there for these children.

And then moving on to our AIM vector program, our AIM vector platform. So to remind everyone, this is a vector library of over 60 AAV vector capsids we have -- that we have brought in-house and we continue to develop. We have a number of programs that have -- a number of vectors, I'm sorry, that have multiple tissue specificities, some for the CNS, some for muscle tissue, some for the heart and lung, even some for the intestine. So you can imagine that as we look forward to our -- expanding our pipeline, leveraging this into some of the existing programs as well as some de novo deliveries and new disease programs as well. So initial studies have indicated that these AIM vectors can efficiently target different tissues with vector-selective tissue specificities. And so we go through different routes of administration when we test by bio-distribution. And really, this is providing us a second-generation treatment approach for patients that had either been dosed previously or potentially have neutralizing antibodies to a particular serotype or actually are about to have a redose. So again, we are very much looking forward to talking more about the AIM vectors at our R&D Day on October 11, so please join us then.

And with that, I've taken up, I think, my portion of this call. I'll turn it over to Jeff to give some of our second quarter summary of financials.

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Jeffrey B. Davis, Abeona Therapeutics Inc. - COO [4]

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Great. Thank you very much, Tim. As you may have noted, we filed our Form 10-Q for the second quarter on August 14. 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 6 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 was $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 will just comment a little bit on upcoming events and conferences because the schedule looks fairly packed here. As Tim has mentioned a couple 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 Cell & Gene Meeting on the Mesa, which is out 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.

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Questions and Answers

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Operator [1]

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(Operator Instructions) Our first question comes from the line of Maury Raycroft with Jefferies.

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Maurice Thomas Raycroft, Jefferies LLC, Research Division - Equity Associate [2]

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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?

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Timothy J. Miller, Abeona Therapeutics Inc. - President, CEO & Director [3]

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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 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 -- so it actually helps close the wound, and then the Amicus product generally helps to keep it moist, and moist wounds heal better. And so we'd see it as complementary. I don't think that we'd cross anybody over from one program into the other one. It's just generally we wouldn't do that with 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.

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Maurice Thomas Raycroft, Jefferies LLC, Research Division - Equity Associate [4]

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Got it. And for AB-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 a precedent for [after workups].

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Timothy J. Miller, Abeona Therapeutics Inc. - President, CEO & Director [5]

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Well, for -- it's a really exciting discussion. I mean, there have been a number of notable failures in the MPS IIIA and IIIB space. And that's really taken the communities 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 talk about biopotency as measured by a 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 and like some other lysosomal storage diseases, having that trifecta of efficacy, at least [phenom's] 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 6 months and now going into 1 year, that's how 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.

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Maurice Thomas Raycroft, Jefferies LLC, Research Division - Equity Associate [6]

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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 bit more color on what you expect there, and I'll hop back in queue.

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Timothy J. Miller, Abeona Therapeutics Inc. - President, CEO & Director [7]

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Sure. We -- to be frank, we've 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 probably will be presented there is on the ABO-102 program. And that will talk about a couple of patients that have gone through 1 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 -- tried 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 1 year on the neurocognitive to the biophysical to, again, some of the biomarkers. So it will be -- so I think it will be a great update.

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Operator [8]

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

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Elemer Piros, Cantor Fitzgerald & Co., Research Division - Analyst [9]

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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?

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Timothy J. Miller, Abeona Therapeutics Inc. - President, CEO & Director [10]

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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 30 and day 90 for some of the patients in Cohort 1 and Cohort 2. 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.

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Elemer Piros, Cantor Fitzgerald & Co., Research Division - Analyst [11]

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

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Timothy J. Miller, Abeona Therapeutics Inc. - President, CEO & Director [12]

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Sure. So we enrolled a total of 7 patients in the EB-101 Phase -- in the Phase I/II. And those patients -- so the first 4 are through -- I think going through a year -- 2 years, I'm sorry, and then the following 3 are at least through 6 months all in here. So 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, very safe 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 8 to 10 or 12 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.

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Elemer Piros, Cantor Fitzgerald & Co., Research Division - Analyst [13]

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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?

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Timothy J. Miller, Abeona Therapeutics Inc. - President, CEO & Director [14]

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Good question. So a couple of things. The CLN1, there are -- very similar to MPS I where there are 3 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 3 to 5. 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 age is like as old as -- really as 8 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. So -- 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 dosing. And we're currently in some of those studies now, at 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, but ones that may have bit of a disease progression. So we'll be starting with intrathecal and then moving upwards and then adding an intravenous dose on top of that.

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Elemer Piros, Cantor Fitzgerald & Co., Research Division - Analyst [15]

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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 in 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?

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Timothy J. Miller, Abeona Therapeutics Inc. - President, CEO & Director [16]

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Yes, I love your line of thought, Elemer, and we've thought pretty 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 a 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 may 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.

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Operator [17]

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Our next question comes from the line of Liav Abraham with Citi.

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Liav Abraham, Citigroup Inc, Research Division - Director [18]

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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 here? Maybe what sort of data you have to date and whether it's possible that these enter IND stage in 2018?

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Timothy J. Miller, Abeona Therapeutics Inc. - President, CEO & Director [19]

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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 really 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 they'd be any real surprises to anybody, but again, we've always tried to find new ways that these vectors can also demonstrate a level of 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 these 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 the space and everyone on those programs.

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Liav Abraham, Citigroup Inc, Research Division - Director [20]

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

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Timothy J. Miller, Abeona Therapeutics Inc. - President, CEO & Director [21]

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Well, 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 heme space for delivery into the heme 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 actual disease targets, we'll put those out there.

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Liav Abraham, Citigroup Inc, Research Division - Director [22]

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

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Timothy J. Miller, Abeona Therapeutics Inc. - President, CEO & Director [23]

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

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Operator [24]

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Our next question comes from the line of [Paul Mann] with Highbridge.

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Paul Mann, Highbridge Capital Management - Analyst [25]

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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 6 months treatment period or 12 months treatment period or maybe even less? And then when we put all that together and start thinking about a regulatory submission, I mean, I think most of us have penciled in 2019, but is there a chance this could go to regulators in 2018 for approval?

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Timothy J. Miller, Abeona Therapeutics Inc. - President, CEO & Director [26]

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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 these 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 8 and 12 patients in the pivotal Phase III. And we expect the follow-up for those patients to be between 3 and 6 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 6-month time point of 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 6-month endpoint, then you never know how things can pan out towards the end of 2018. So we'll put our best foot forward.

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Paul Mann, Highbridge Capital Management - Analyst [27]

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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 do 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 into like a disease management program? Or how do you think about that? Have you had any discussions already with regulators?

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Timothy J. Miller, Abeona Therapeutics Inc. - President, CEO & Director [28]

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We are -- 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 cell so the patients can come back and essentially have another application of the EB-101. So you could have -- so they come in for 3. They could 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 do 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. So...

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Operator [29]

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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.

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Timothy J. Miller, Abeona Therapeutics Inc. - President, CEO & Director [30]

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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 a really 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 times. 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.

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Operator [31]

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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.