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Edited Transcript of ABEO earnings conference call or presentation 10-Apr-17 2:00pm GMT

Thomson Reuters StreetEvents

Q4 2016 Abeona Therapeutics Inc Earnings Call

Dallas Apr 10, 2017 (Thomson StreetEvents) -- Edited Transcript of Abeona Therapeutics Inc earnings conference call or presentation Monday, April 10, 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 and Director

* Timothy J. Miller

Abeona Therapeutics Inc. - CEO, President and Director

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Conference Call Participants

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* Elemer Piros

Cantor Fitzgerald & Co., Research Division - Analyst

* George B. Zavoico

JonesTrading Institutional Services, LLC, Research Division - Senior Equity Analyst

* Jason McCarthy

Maxim Group LLC, Research Division - Equity Research Analyst

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Presentation

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Operator [1]

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Good morning, and welcome to the Abeona Therapeutics Inc. Quarterly Earnings and Business Update Conference Call. (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 at Abeona Therapeutics. Christine?

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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, we have Dr. Timothy Miller, President and CEO; and Jeffrey Davis, COO of Abeona Therapeutics.

Dr. Miller will begin the call with an overview of the fourth quarter and more recent highlights and developments at Abeona. After, Jeff will provide additional comments on the quarter, a brief overview of summary financials and then provide a snapshot of our financial position and review the upcoming investor conference calendar. Following that, we will open the floor up to 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 differ 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. - CEO, President and Director [3]

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Thanks, Christine, and thanks for everyone for joining us this morning.

2016 and the fourth quarter especially were marked by notable achievements and advancements for Abeona and our goals for expanding and building ourselves as a rare disease leading gene therapy company. We've expanded our program pipeline with the addition of a clinical stage asset. We reported on positive clinical data in our lead program for Sanfilippo syndrome type A as well as our programs for epidermolysis bullosa and we've achieved multiple regulatory designations in multiple programs. Furthermore, we have stated that we are further developing our work in the library of next-generation AAV viral vectors, our AIM viral vector platform, for which we have global exclusive rights with the rights to sublicense.

To go over some of our -- some of the milestones that we achieved, particularly in the clinical domain, I will talk a little bit about our lead program, ABO-102, which is a treatment -- gene therapy treatment for patients with Sanfilippo syndrome type A. We met many of our goals for advancing this lead gene therapy program. It is the only one that's in the clinic in 2016 and currently, in 2017, that is enrolling patients for patients with Sanfilippo syndrome.

For those that don't know, this is a lysosomal storage disease. These children have problems with an enzyme in breaking down sugars. 70% of these children don't reach the age 18. And it's rather notable to note that, recently, there have been a lot of popularized deaths from children with this disease. Right now, Abeona is the only program in the world that has the gene therapy muscles and the other therapy in development and in clinical trials.

Some of the regulatory achievements in this program were that the EMA or the European Medical -- Medicinal Agencies granted the Orphan Drug Designation. We also received Fast Track Designation from the FDA from this program, which enables us to file world and biologic licensing application. We are already building on the already granted Rare Disease Pediatric Designation and the Orphan Drug Designation in the United States.

The significance of these designations is not to be really undermined. It's certainly, as we look to the future, going to be helpful for us, looking to file potentially in late 2018. We do have a dosing and complete reporting at the low-dose cohort, which will be -- some of which will be reported tomorrow at the New York Academy of Sciences and other lines reported at the American Society of Gene & Cell Therapy Conference coming up in May. It's important that we do note that the Priority Review Voucher, we are eligible for, for this program and look forward to updating more as we go forward with our regulatory strategies.

We have recently reported positive data from the low dose, demonstrating a central nervous system and peripheral organ disease biopotency. As a review of the data that was reported out, we did report out 2 patients worth of data, but we do have much of the third patient dose data to report out soon. ABO-102 is well-tolerated in all subjects to date, with one -- or 3 low-dose and 2 high-dose patients tested. We are through over 750 days of follow-up with no serious adverse events, which is rather remarkable for a gene therapy program that's delivered by intravenous delivery. And we have seen a significant reduction in the heparan sulfate, which is the actual GAG or glycosaminoglycan in the cerebral spinal fluid, over 60% in -- over 3 patients. Continued evidence of biopotency has been demonstrated with reduced liver and spleen volumes and decreased urinary GAGs as well as total GAGs. Two subjects assessed at the 6-month time point showed evidence for stabilization or improvement, an average of 60% over 2 subjects in multiples of the Mullen subdomains. And the adaptive rating behavior -- adaptive behavior ratings on the Vineland scale stabilized.

Importantly, subjects showed improved ability to complete individual items on the Leiter-R nonverbal IQ assessment, resulting in improved raw scores. This is particularly important as these first 3 subjects had all been enrolled in the Natural History study and so we had a long-term longitudinal data on them to be able to demonstrate that severe disease progression over the ages of 5 to 6. And then, as they came in for their baseline visits, they actually had gotten through the floor of their ability to be able to be scored. And why this is important because 6 months post-injection, these children had improved to a point where they could be scored again on their nonverbal IQ.

Looking at -- as we go into the high-dose cohort, where we're going to be completing the enrollment -- the planned enrollment, that's 3 patients with Nationwide Children's Hospital in April and we'll most likely be expanding the enrollment, either at this dose or at a higher dose, at some point in the near future.

It's important to enroll -- to note that we have -- are expanding our enrollments in Spain and Australia. The sites are both being initiated in April, so we'll be set to screen starting late April, beginning of May. So as we go into the end of the second quarter, we'll been enrolling 2 to 3 patients a month. And as we start to set some of our enrollment time lines, this is important because we will be moving towards an enrollment goal of 15 patients in the next few months -- or I'm sorry, throughout the end of 2017. This is extremely valuable to the company as we seek to become a leader in Sanfilippo gene therapy, but in gene therapy overall.

Our second lead gene therapy program is in the form of an autologous keratinocyte corrective gene therapy for a horrifying disease called epidermolysis bullosa, otherwise known as butterfly skin syndrome. And we are treating the most severe form of this, the recessive dystrophic version. In this program, children can come in or adolescents come in or adults come in and they have a biopsy taken. The keratinocytes, which are actually skin cells, are taken out and purified and expanded. They are -- using a gene therapy, they are corrected so that the kids have an underlying deficit in a protein called collagen. Collagen is really the zipper that holds your skin onto your body. And because these patients don't have that, they have these blistering diseases that open and form chronic wounds that don't heal for years. We have, in our partners with Stanford University, a very large Natural History study showing that none of the therapy that have really been tried to date have worked out to close these wounds, including improved dermatologic programs or products. Right now, what we do is we -- when we grow up and make these sheets, they're about the size of an iPhone 7, and each subject gets 6 of them placed on top of their wounds.

To date, we have -- again, we've continued to expand our programs with this particular clinical-stage asset. We have received EMA Orphan Drug Designation and reported out positive Phase I clinical results for EB-101, which were published last year with the journal -- in the Journal of American Medical Association or JAMA, which highlighted that the therapy was well-tolerated in these patients and demonstrated a clinical efficacy; 67% of the healed wounds at 6 months, some of which have continued to last through 12 months and, importantly, have seen biomarker expression of collagen in these wounds. This is significant as we look to have regulatory meetings in the second and third quarter to talk about registrable endpoints for this program, particularly looking at what would be the amount of wound closure and biomarker expression in a onetime point.

So accordingly, we have treated the fifth and sixth patient and this seventh patient has recently been treated as well. So this is a total of 42 wounds that have been treated. And next steps, the company anticipates providing a clinical update at the upcoming annual meeting of Society for Investigative Dermatology in April 2017.

Additional regulatory, we have U.S. Orphan Drug Designation and the Rare Pediatric Disease Designation hoping to come in later this year as well as additional enrollment targets to try and hit 10 to 12 patients by the end of the year.

Next, we have to talk about is our second Sanfilippo program. So recall that, for those that are not very familiar with Sanfilippo syndrome, there are 4 types: Type A, B, C and D. We are focusing on the 2 most common types: Type A, which is our ABO-102 program; and then we have an ABO-101 program, which is for Sanfilippo syndrome type B. This is, again, another adeno-associated virus gene therapy program.

We have received the IND to go ahead and start the clinical trial. It is on track and we are -- anticipate that we'll be having patients enrolled in that very soon. The IND has been granted and we are -- will be seeking initial regulatory approval in Europe. Currently, we have 2 of the 3 necessary steps completed in Europe to start the clinical trial there as well. We have been granted Orphan Drug Designation in Europe and continue to -- and we look forward to announcing additional regulatory designations in this program later this year as well as reporting out data later in the second half of 2018.

Moving on to our proprietary AIM vector platform. So we haven't given a lot of guidance yet on how we're really approaching this AIM vector program, but it does encompass vectors that have tissue tropisms for multiple targets, including the hematologic and dermatology spaces. We partnered with University of North Carolina in developing the AIM vector system. It is a novel AAV-based vector technology that may also target additional tropisms such as muscle tissue, the liver and/or central nervous system.

So right now, we're looking forward to reporting out some data on the platform at the American Society for Gene Therapy in collaboration with our partners, that's the University of North Carolina. It's a very exciting program. We're looking forward to how -- this is really how we see an approach to second-generation products in our lead programs as well as additional de novo or new programs to be brought in-house at Abeona.

We do have 2 additional programs that I'll comment on today. We have our Batten disease programs. So Batten disease is another lysosomal storage disease. So when you look at our -- at the top of our pipeline in metabolic, you'll see 4 AAV programs that are all targeting lysosomal storage disease. So the Sanfilippo programs are currently clinical stage and the Batten disease programs are preclinical, but we anticipate that these will be clinical stage by the end of the year.

For our juvenile Batten program, which is ABO-201 or the CLN3 version of the disease, the EMA has granted Orphan Drug Designation. We announced preclinical data supporting clinical trials for ABO-201 were published in September at the Journal of Neuroscience. We have completed a lot of the preclinical studies necessary to get into the IND and conducted a pre-IND meeting last year, which was very successful. The IND-enabling studies for this have been initiated and we are engaging the FDA in additional comments. Things are looking very, very good in the animals to date with no deaths and things look like they are on track for this program to be -- again, to have the IND by the end of the year.

For ABO-202, the CLN1 version of the disease, or infantile, we are continuing to advance this towards IND-enabling studies. And we -- again, we anticipate that human clinical trials will be commenced by the end of the year.

So it's been a very exciting year and really beginning of the year in 2017 for Abeona. Our gene therapy programs, we have 3 of them that are clinical stage. There are very few companies in the world that have actual clinical data in gene therapy, much less a broad enough pipeline. We are setting ourselves up well with the AIM vector platform to enhance our programs and essentially bring in additional programs for use of the AIM vector platform. We look forward to announcing that data at the upcoming conferences later on in the year.

So with that, I'll turn this over to Jeff Davis to give our fourth quarter and year-end summary financial results and look forward to answering any questions coming up at the end of the call. Jeff?

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

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Thank you, Tim. In terms of the financials, I'll talk a little bit about some of the specifics.

Our cash position at the end of the year, cash and cash equivalents at December 31, 2016, was 61 -- $69.1 million compared to $31.2 million as of the end of September 30, 2016. The net cash used in operating activities for the 12 months of 2016 was $13 million as compared to $10.4 million in the same period in 2015, so just a modest increase there. The increase in the cash, the strengthening of our balance sheet occurred in the fourth quarter. We closed underwritten public offering of 6 million shares of common stock at an offering price of $7 per share. That closed on November 1, 2016. And then, there was a modest partial exercise of the shoe later in that month that occurred.

In terms of revenues, revenues were $256,000 in the fourth quarter compared to $215,000 in the fourth quarter of 2015 and the 12-month revenues were approximately $900,000 compared to approximately $1 million in 2015. Again, these revenues consisted mainly a combination of royalties from a marketed product, MuGard, which is licensed out in several countries, as well as deferred revenue related to upfront payments from early licensing agreements.

The loss per share was $0.19 in the fourth quarter compared to $0.06 in the comparable quarter in 2015. And that's a fairly concise summary of what went on.

If anyone has any questions with respect to the financials and the Form 10-K that was recently filed, feel free to give me a call directly to talk about it.

Before summary, I'll just talk a little bit about some of the upcoming events and conferences. These -- by the way, as well as links to our financial statements and our investor presentation can all be found in our website at abeonatherapeutics.com.

Tomorrow, as Tim alluded to in his prepared talks, we are -- we've worked to help organize, together with the New York Academy of Sciences and George Zavoico, Dr. Zavoico, who covers us on the sell side research side from JonesTrading, helped organize a really good gene therapy for Rare Disease Day that's happening in -- here in Manhattan tomorrow. We have a bunch of key opinion leaders in the gene therapy space, including Dr. Kevin Flanigan, the principal investigator on our Sanfilippo trial; and Dr. Katherine High from Spark Therapeutics; Dr. Brian Kaspar of Nationwide Children's Hospital and AveXis; Dr. Barry Byrne from Florida; Dr. Maria Escolar from the Children's Hospital of Pittsburgh; Dr. David Pearce from Sanford Research; Jude Samulski from Bamboo and Pfizer now; as well as Dr. Jakub Tolar from University of Minnesota with whom we're working on, on some of the CRISPR activities that we're doing. So we look forward to a very full day and an informative day tomorrow here in New York.

We have a couple other meetings that were alluded to in the call is the Society for Investigative Dermatology later. This is, I believe, around April 26 to 28 where we believe we'll be providing some updates on our EB programs. And we have ASGCT in May where, I think, there'll be abstracts on many of our technologies and discussions and presentations there as well as a few sort of smaller ones, ARM in Boston and a few other things. So we'll be very active in the next couple of months with a bunch of conferences where we hope to be able to provide some updates on our clinical programs and even our preclinical programs as well.

So I think I'll turn it back to -- well -- and maybe we can open up for questions now and then Tim will have some closing remarks.

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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 Elemer Piros with Cantor Fitzgerald.

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Elemer Piros, Cantor Fitzgerald & Co., Research Division - Analyst [2]

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What I'd like to ask qualitatively, Tim, is the data that you will disclose tomorrow versus the data that you plan to unveil at ASGCT in May. What will they be? And what the difference between the 2 might be?

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

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Sure. Nice to hear your voice, Elemer, as always. So Kevin -- the New York Academy of Sciences meeting tomorrow is really more about gene therapy in general. And many of the leaders in the field are -- they're going to be discussing multiple parts of their individual programs. Dr. Flanagan is going to present, I think, a few pieces of updates for the ABO-102 trial. I think he's probably going to comment on the CSF/heparan sulfate which has continued to maintain its reduction at day 180 through the low-dose cohort. And he'll probably comment on some of the urinary GAGs as well. We are continuing to process a lot of the data that's come out of the MRIs, for example, through both the Natural History study and the clinical trial. One of the things that we're looking forward to providing a very robust update at some point near -- in the near term future is really what the MRI data has shown us, as well as how we are linking that prospectively towards the doses that have been involved in the high-dose cohort. So I think that tomorrow, you'll see some -- a small bit, additional bite of the apple, from CNS, some of those CNS data, as well as some of the urinary data. I think that Kevin wants to hold a little bit of powder for the American Society for Gene and Cell Therapy conference, but IS certainly going to be a bit more clinically focused than more experts in the field, for example, on the clinical side.

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Elemer Piros, Cantor Fitzgerald & Co., Research Division - Analyst [4]

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Okay, okay. And did I hear it correctly that you already dosed the second high-dose patient in the IIIA study?

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

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Yes. We'll be enrolling -- or finishing the cohort very, very soon.

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Elemer Piros, Cantor Fitzgerald & Co., Research Division - Analyst [6]

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Okay. And so do you think some of that data from the high-dose cohorts, at least at 30 days, would make it to ASGCT?

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

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Yes, that's the current goal. Again, it does take time. We're -- we try very hard to maintain a level of clinical equipoise in the data so that if, really, any of patient be identified in social media or public forum, that individual data points can't be tied back to them, again, both protect them and really, just to make sure that we have more than an n of 1 going into these reports. So -- and consequently, we do have to wait a certain amount of time just to get the data because we try to get at least through day 30, sometimes all the way up through day 90, before we start reporting them about. So we're looking forward to, at least hopefully, reporting out the first 2 patients' worth of data at ASGCT.

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Elemer Piros, Cantor Fitzgerald & Co., Research Division - Analyst [8]

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So the IIIB IND, I thought that it was allowed probably last May in the U.S. But did I hear you correctly that now, you're moving to Europe and start a European trial instead of in the U.S.? Or that's just this two-step process?

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Timothy J. Miller, Abeona Therapeutics Inc. - CEO, President and Director [9]

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No, no. Actually -- so we'll be starting at Nationwide Children's Hospital in the next few months. But we're also planning to open an additional clinical site in that trial in Europe. So there will be 2 clinical sites, not just 1, so we can double up on our enrollments a little bit earlier than we have in the Sanfilippo type A syndrome program. Thank you for allowing me that opportunity to clarify.

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Elemer Piros, Cantor Fitzgerald & Co., Research Division - Analyst [10]

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Okay, okay. And so is there maybe a shortage of IIIB patients, that the study hasn't started yet? Or...

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Timothy J. Miller, Abeona Therapeutics Inc. - CEO, President and Director [11]

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No. Due to the -- some of the Natural History study in Nationwide Children's Hospital, Elemer, just to recall, we enrolled 10 patients -- I'm sorry. Yes, 10 patients in the -- 10 B patients in that Nationwide Children's Hospital. And certainly, many of those are crossing over into other programs. But there's no lack of IIIB patients, to be sure. What's interesting to actually note about it is that there's been an uptick in the number of diagnoses in the past 2 years, I think, because more clinicians are becoming more familiar with these diseases. So certainly, as I think many of these programs are looking to push enrollments to -- again, the mantra in this disease state is treat early, treat it with as much as possible. And certainly looking to continue fulfilling on that model.

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Elemer Piros, Cantor Fitzgerald & Co., Research Division - Analyst [12]

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Yes, yes. And one last question on this front, Tim. You talked about the European process; that 2 out of the 3 steps that needs to be taken is -- has been crossed or checked. What is the third step? Because I'm somewhat unfamiliar with the European regulatory landscape.

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Timothy J. Miller, Abeona Therapeutics Inc. - CEO, President and Director [13]

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Sure. So the first 2 steps are really very much in parallel with what would be considered IRB and the Recombinant DNA Advisory Committee submissions in the United States. So we have already gone through the GMO, the genetically modified organism, and ethical commissions and reviews and basically have those approved. So the last step is really with the main regulatory agency in Spain, which is the country where we're doing this. So -- and looking forward to -- and we have a very good relationship with the regulatory agency, and they've been very, very helpful providing both feedback in our earlier stages, and now that we're about to submit for that regulatory approval to treat coming up.

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Elemer Piros, Cantor Fitzgerald & Co., Research Division - Analyst [14]

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Okay, okay. And one quick question to Jeff. The burn last year of $13 million, Jeff, I'd have presumed that, that would be reflective of what you expect in 2017. What sort of ramp do you envision from that level?

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Jeffrey B. Davis, Abeona Therapeutics Inc. - COO and Director [15]

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Yes. That's an excellent question, Elemer. We all along said last year that the burn -- we anticipated the burn to increase, given the fact that we'll open 2 additional sites for the A trial, we'll get the B trial kicked off and we have multiple enrollments in the EB trial that are currently sort of scheduled. I think we've been telling investment community, guiding them that we anticipate burning $20 million to $22 million this year. So we burned $13 million last year. And that doesn't include any sort of -- necessarily any kind of onetimers that are unanticipated or what have you. But even given that, we have somewhere between 2 and 3 years' worth of cash, so we don't have really any current plans to raise money.

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Operator [16]

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Our next question comes from the line of Jason McCarthy with Maxim Group.

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Jason McCarthy, Maxim Group LLC, Research Division - Equity Research Analyst [17]

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Just if we could just steer back to EB-101. If you can remind me, you said you had 7 patients that were treated so far. Maybe you can give us an update on how long those patients had, had their wounds grafted for. Maybe what we might expect at the derm meeting at the end of the month. And pending that trial accelerating enrollments at 2 to 3 patients per month and your meetings with the FDA, is there a possibility that this study could serve as a registration study?

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Timothy J. Miller, Abeona Therapeutics Inc. - CEO, President and Director [18]

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It's a great question. And we're looking forward to really kind of bringing a bit more of the spotlight onto the EB program in the next couple of months. So we would clarify that we're not trying to do 2 to 3 patients a month in the EB program. That's more the ABO-102 programs, and these are still going to be successive enrollments just because of how the drug is essentially made or the product is made. And it's really its location and how we're approaching this in discussions with the FDA. So currently, we do have 7 patients that have been enrolled. What the Society for Investigative Dermatology is going to talk about really is -- it's really almost -- you got to look at this, almost, as 2 populations from the first couple of years. So 4 -- the first 4 patients, are already through 2 years' worth of follow-up. So in this particularly disease category, that's rather significant when you look at the fact that many of them had already received other dermatology products to try and close their wounds, and none of them actually had stayed closed for more than just a few weeks. Whereas after 2 years in our -- after being treated with our product, these wounds are upwards of 50% staying closed. And you're seeing expression of that collagen 2 years out in them. So as we look to engage the FDA, we do have some meetings coming up, we will be talking about what we believe are registrate-able endpoints. And yes, we do hope that this is a registration study where we could potentially file this in the second half of 2018 as well. So again, it's an exciting program. We're certainly expanding it, the enrollments. And you'll hear this at the Society for Investigative Dermatology.

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Jason McCarthy, Maxim Group LLC, Research Division - Equity Research Analyst [19]

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Great, and if you -- just a follow-up to that. Can you give us a sense of the diameter of a wound that you're able to close? I know there's other groups that are in this space as well, but to my knowledge, that they're able to close just a very, very tiny portion of the wound. Can you give us a sense of how EB-101's mechanism of action is different? And if you can cover much more sizable area of these wounds?

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Timothy J. Miller, Abeona Therapeutics Inc. - CEO, President and Director [20]

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Yes. Great question. So when you meet a lot of these patients, what they end up coming in as is, basically, they're wrapped in bandages because they have open wounds over greater than 50% of their body sometimes. And our EB program can actually treat much larger areas. So again, if you can imagine that, essentially, a skin -- the product's about the size of an iPhone 7. And each patient gets 6 iPhone 7's worth of surface area covered over their skin. So we can cover areas like entire arms, the entire thigh, a lot of skin over the back or chest. So certainly, look to cover a large surface area. So thank you for that. Thanks, Jason.

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Operator [21]

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Our next question comes from the line of George Zavoico with JonesTrading.

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George B. Zavoico, JonesTrading Institutional Services, LLC, Research Division - Senior Equity Analyst [22]

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A couple of fairly quick questions. On the AIM platform study, the tropism issue that you talked about, where you're looking to see tropism to various organs and tissues, do you have a certain criteria in mind as to what is sufficient to go forward? In other words, what percent of off-target effect is acceptable?

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

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Yes. Sure, George. So just to clarify though, the AIM vector library poses a number of AAVs for direct-to-gene replacement strategies. And so we're looking at these. We're going to be at that -- at -- under a disease-specific mindset. So gene therapy, as we know, is all about delivery and being able to deliver the correct copy of the gene under the gene replacement strategy. So we do have vectors that have a significant dermatology or skin tropism. Some of the vectors have very good tropisms for the liver; others for the central nervous system, which was -- where a part of the library was actually derived from. So using these gene replacement strategies isn't so much about the target effects, which is really the [ gene ] programs. I didn't comment very much on our Fanconi anemia program at the University of Minnesota and Stanford. That is certainly moving along. The researchers at University of Minnesota who -- Jakub Tolar will be speaking at the -- or at the New York Academy of Sciences tomorrow, George, has certainly helped advance as Mark Osborn. But -- so for the AIM vector platform, we're looking at these as right -- the right tropism delivered the most appropriate way for the right needs. And very so much, many of the other gene therapy programs that are out there, trying to (inaudible) to be able to include additional disease targets and, really, additional disease categories. So hope that answers your question.

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George B. Zavoico, JonesTrading Institutional Services, LLC, Research Division - Senior Equity Analyst [24]

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Yes, it does. I mean, in summary, it sounds like you're not really -- there's no real criteria for tropism. It's just it's more important to see a satisfactory outcome for the patients than the clinical benefit, as long as you don't have adverse off-target effects. Is that -- in summary, is that pretty much where you're [ switching? ]

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Timothy J. Miller, Abeona Therapeutics Inc. - CEO, President and Director [25]

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Yes, that's a fair way to say it. Again -- so again, you're trying to find the right tissue tropism for the right disease. You're not going to necessarily use a muscular-based AAV program for a central nervous system target. So again, we look forward to announcing and really showing more about this program in the upcoming months. And really, those are going to be the ones that additional disease targets will fall under.

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George B. Zavoico, JonesTrading Institutional Services, LLC, Research Division - Senior Equity Analyst [26]

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Okay. With regard to the Sanfilippo programs, you mentioned -- well, the trials are set for low-dose and high-dose. And then you mentioned that you might go to a higher dose. What would you need to consider? What kind of results would make you go to the third dose?

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Timothy J. Miller, Abeona Therapeutics Inc. - CEO, President and Director [27]

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Well, the prospectively defined dose program in this has always included the ability to go to higher doses if we chose. You know the -- we have a very, very broad window within our safety programs from behind the enabling toxicology studies. And in lysosomal storage diseases in general, the mantra has always been with a gene therapy approach, try and go with treat younger and treat with more gene therapy if possible. So we -- the investigators have always considered that if things look safe, that they would try and push the dose. And we're -- and just to be clear, we're very comfortable with what we're seeing right now, even in the low dose. Recall that when we first started these programs, we started with the minimum efficacious dose to be able to be treated in older animals that showed complete correction of disease, which included survival, neuromuscular benefit and really cognitive corrections. And to date, when you look at the low-dose data, this has been really, I mean, a robust data package. No other program in the world has been able to show any form of combination of reductions of GAGs in the CSF, reductions of the AGs systemically, organ manifestation changes in liver and spleen volume changes, as well as early indications of some of disease stabilization from a neuro-cognitive benefit. So when we've dose-escalated, it's always been under the idea that look, if things look safe, maybe we'll continue to dose-escalate from there. But again, we would probably speculate to say that things are already working in this clinical program, let's see how much further we can do this, really, for the benefit of the patients. Because as we enrolled on what would be considered the other side of the bell curve on the developmental stage, now as we seek to enroll [ some newer ] patients to really be able to see if we can extend what will be considered normal or push them into a more normal range of development. So it's a really exciting time for us to actually be able to consider just enrolling more patients at the high dose. But we're really looking to see if we can even accelerate or enhance this program with additional dosing. So very, very exciting.

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George B. Zavoico, JonesTrading Institutional Services, LLC, Research Division - Senior Equity Analyst [28]

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You're also decreasing the age in steps of patients who enroll. The first batch, I think, was -- was it 18 and over? And then you were -- could you just remind us what -- how you're going to younger patients? And what steps you're going to younger patients?

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Timothy J. Miller, Abeona Therapeutics Inc. - CEO, President and Director [29]

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Well, the enrollment criteria hasn't changed. It's always been 2 years and older. And really, the Natural History study that has come out of this and looked at 25 patients is really shown that it's all about the disease manifestation in an individual patient that really dictates whether they should be enrolled in a clinical trial. We do have a variety of important enrollment criteria, including AAVs, and the specificity of antibodies to the AAV. I think the clinicians, really, their goal has been to show some initial signs of safety and potential efficacy. And now, again, with the ability to try and target some of the younger kids with this particular product, it's more about not just disease stabilization, but potential disease reversal to the point where the kid could develop a bit more normally, so.

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Operator [30]

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Thank you. Ladies and gentlemen, we have come to the end of our time for questions. I'll turn the floor back to Mr. Miller for any final remarks.

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Timothy J. Miller, Abeona Therapeutics Inc. - CEO, President and Director [31]

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Well, it's been an exciting year. And thank you all for your questions. Certainly, as we've spoken to many of you and look forward to continuing our dialogue, certainly, being able to report out such great data is always an exciting time for a company.

Now with 3 clinical-stage programs that are gene therapy programs and 2 additional programs going into the clinic, Abeona is really continuing its progress to becoming a world leader in gene therapies for rare disease treatments.

I'd like to thank all our investors and partners, researchers and dedicated patient foundations, including the employees and scientific advisers and board members for their continued support. And look forward to meeting with all of you throughout the year. Thank you.

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Operator [32]

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Thank you. This concludes today's teleconference. You may disconnect your lines at this time. Thank you for your participation.