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Abeona Therapeutics Inc (ABEO) Q1 2019 Earnings Call Transcript

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Abeona Therapeutics Inc (NASDAQ: ABEO)
Q1 2019 Earnings Call
May 14, 2019, 10:00 a.m. ET

Contents:

  • Prepared Remarks
  • Questions and Answers
  • Call Participants

Prepared Remarks:

Operator

Good morning, ladies and gentlemen, and welcome to the Abeona Therapeutics Inc. First Quarter 2019 Earnings and Business Update Conference Call. Today's call is being recorded. And at this time, all participants are in a listen-only mode. A brief question-and-answer session will follow the formal presentation.

For opening remarks and introductions, I'll turn the call over to Sofia Warner, Senior Director, Investor Relations. Thank you. You may begin.

Sofia Warner -- Senior Director, Investor Relations

Thank you. Good morning, and welcome, everyone. Today's call will be led by Joao Siffert, our CEO. Following Joao, Tim Miller, our President and CSO, will present preclinical highlights; and Christine Silverstein, our CFO will review our financials.

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 these 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 may be found in the company's annual reports on Form 10-K and quarterly reports on Form 10-Q filed by the company with the Securities and Exchange Commission. These documents are available on our website, www.abeonatherapeutics.com.

With that said, it is now my pleasure to introduce you to Dr. Joao Siffert. Joao, you have the floor.

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

Thank you, Sofia, and thank you all for joining us today for our first quarter business highlights, which reflect the continued development of our clinical and preclinical programs, as well as manufacturing quality operations.

I'd like to start with some key quarter events and then we'll turn the call over to Tim who will take you through the exciting advancements within our next-generation AIM vector platform. Earlier this year, we presented AIM data at several conferences highlighting the potential of this technology to treat patients with a variety of disorders and to evade pre-existing or neutralizing antibodies to naturally occurring AAV. We have grown this library now to over 100 novel capsids, some of which have specific tissue tropisms outside of our current pipeline targets and thus open the possibility of collaborating with external partners that might help accelerate and expand our ability to develop them into new treatments. Since our last earnings call, we also completed our CMC work for upcoming Phase 3 clinical trial in recessive dystrophic epidermolysis bullosa or RDEB. The FDA is completing the review of our work before we can proceed with the Phase 3 trial, which remains on track for mid 2019 start. Our dedicated cGMP suites at our Cleveland facility are currently in operation by an experienced trained staff, positioning us well to produce clinical product on time for a Phase 3 start.

We also remain focused on our efforts to identify screen and enroll patients for our clinical trials in MPS. As stated last quarter, we have stepped up our efforts to engage the broad network of healthcare professionals and communities involved in the care and support of children affected by lysosomal storage diseases and remain on track to activate additional study sites globally by the end of this year. This concerted effort has begun to bear fruit as announced via press release this morning. We have now completed dosing cohort 1 and treated the first patient in cohort 2 in the ongoing Phase 1 global clinical trial evaluating ABO-101. Our gene therapy for patients with MPS IIIB also known as Sanfilippo syndrome type B. MPS IIIB is a rare fatal lysosomal storage disease with no approved treatment that is characterized primarily by rapid neurodevelopmental decline.

The two-year open label study is currently designed to evaluate two doses of ABO-101. The dose for the first cohort is 2E13 and the second cohort does is 5E13. The main objectives of the study are to assess safety and neurodevelopmental milestones with multiple secondary endpoints including changes in CSF and urine heparan sulphate. Additional measurements include CSF and serum NAGLU enzyme activity in liver and brain volume by MRI. We expect to report interim data for the trial in the second half of this year. Last month, the FDA granted fast-track designation to ABO-101, recognizing the severity and importance of addressing this rare orphan disease.

Now, I'd like to provide an update on our progress within our other two clinical programs and share with you the important milestones we're working toward in the balance of this year. As last presented at WORLDSymposium in February, the ongoing Phase 1, 2 clinical trial in MPS IIIA also known as Sanfilippo syndrome type A has enrolled 14 patients to date. The data from this trial demonstrate that ABO-102 has been well tolerated with no serious drug-related adverse events to date. I have also observed substantial and durable improvements in biomarkers, including dose-related reductions in cerebral spinal fluid heparan sulfate levels as well as reduction in liver volume in all treated patients. These biochemical and biophysical improvements were observed within four weeks post dosing and have persisted to date with two plus years of follow-up.

In addition, investigators observed encouraging neurocognitive signals in younger, higher-functioning patients enrolled in the higher dose cohort 3. We believe we're on track to assess the benefits of ABO-102 and plan to provide data updates from this trial in the second half of 2019, a recruitment initiative that also provide a continuous screening activity for this study.

Finally, as mentioned earlier, our lead clinical program in RDEB with EB-101 is planned to enter Phase 3 midyear. RDEB is a rare genetic skin disorder caused by mutations, which result in lack of functional collagen VII, the main component of the anchoring fibrils that attach the dermis to the epidermis. RDEB patients have extremely fragile skin that blisters and tears, resulting in large open wounds, some of which remain open for years. RDEB wounds are painfully debilitating and patients currently have no available effective treatment options, relying only on palliative treatments such as wound care to prevent infection and pain management for relief.

EB-101 is the next vivo gene-corrected autologous cell therapy designed to enable normal type VII collagen expression in wound healing in patients with RDEB. The gene-corrected keratinocytes are transplanted onto open wounds and have been shown to produce durable healing. The VITAL Phase 3 study will be a multi-center, randomized clinical trial assessing 10 RDEB patients to 15 RDEB patients treated with EB-101. The primary outcome measure of the study will be the proportion of patients' wounds with greater than 50% healing at three months compared with untreated wounds sites on the same patients. Additional endpoints will include the patient's global assessment of the wound changed from baseline, as well as patient reported changes in pain in itch from baseline.

The design of the Phase 3 study is similar to the completed Phase 1/2 trial. Data from the Phase 1/2 trial recently presented at the Society for Investigative Dermatology Conference shows sustainable wound healing for three years in addition to positive patient reported outcomes, such as reduction in pain and itch. Efficacy of EB-101 including collagen VII expression and reconstitution of anchoring fibrils was observed as early as one month post engraftment of treated wounds and remained functional up to the observation period of three years. To date, EB-101 exhibited a favorable safety profile with no product-related SAEs.

In addition to the start of the Phase 3 development, the upcoming EB-101 clinical trial stands to validate a successful completion of an 18-month long effort that brought to life our in-house GMP-compliant facility in Cleveland. Our investment in CMC preparations in diligence also positions us well toward an eventual BLA filing, commercialization and will ultimately help us provide treatment to patients suffering from RDEB.

R&D manufacturing, regulatory and quality teams have also worked diligently toward expanding our clinical stage pipeline. R&D has been submitted for ABO-202 in CLN1 disease, also known as infantile Batten disease. IND-enabling data shared at WORLDsymposium and ASGCT showed a favorable safety profile for ABO-202 with no significant toxicities seen in preclinical dose escalation studies.

Other IND-enabling studies also demonstrated normalized survival and improvement of motor and cognitive function in affected mice treated with ABO-202. Data collected to date suggest that combined dosing with intravenous and intrathecal administrations may enhance the therapeutic potential of ABO-202. As with other neurodegenerative diseases, our preclinical studies also showed a dose-related increase in efficacy and importantly a substantially higher survival of animals treated during earlier stages of disease.

Preclinical development of ABO-201 are AAV based program that targets CLN3 or Juvenile Batten disease continues to advance. We will provide an update on the program later this year.

I want to take a moment to thank all of our hard work of Abeona employees as well as of expert collaborators, and most of all thank the patients and their families who placed their trust in us.

With that, I'll pass the call over to Tim, who will take you through the exciting advancements within our next-generation AIM vector platform. Tim?

Timothy J. Miller -- President & Chief Scientific Officer

Thank you, Joao. In the first quarter and the month thereafter, we unveiled exciting progress at three separate events on programs utilizing our novel AIM vector platform. This proprietary technology continues to demonstrate how novel AAV capsids that have been selected to target specific body tissues with high efficiency are being developed by Abeona to potentially address Pompe and Fabry diseases, cystic fibrosis, muscle diseases as well as multiple eye disorders.

At the annual WORLDsymposium in February, we presented data demonstrating how AIM-based capsids delivered novel transgenes and demonstrated proof of concept data in animals for Fabry and Pompe diseases. While current enzyme replacement therapy significantly reduces the mortality of infantile Pompe patients, it fails to completely ameliorate all symptoms, primarily due to its inability to inefficiently enter the CNS and due to the immune responses to the GAA protein.

Notably, additional data have now demonstrated that AAV204 (ph) in GAA delivered intravenously identified multiple viable transgene cassettes that produced significant levels of active GAA protein in cells and animals. In multiple disease models and animal species, our novel AIM AAV capsids have demonstrated broad tissue tropism when compared to existing natural AAV capsids. The AIM capsid also performed better than existing natural serotypes that are transducing the brain, spinal cord and eye in rodents and primates.

Taken together, these data leave us strongly encouraged about the therapeutic potential of the AIM AAV vector platform in multiple disease targets. More recently, in April, we presented data at the American Society of Gene & Cell Therapy Annual Meeting in Washington DC for ABO-401, our novel gene therapy for Cystic Fibrosis. Some key highlights from the data showed that our novel AIM AAV vector with the CFTR mini gene can address all CF mutations and that it efficiently targets lung cells. There, we demonstrated that ABO-401 can correct the underlying CF chloride channel deficit regardless of underlying mutations of the CF transmembrane conductance regulators in CF mice and in human CF patient cells. Importantly, ABO-401 can correct the defect of the most common CF mutation, delta-F508, which accounts for greater than 60% of the CF population. This is an important differentiator as this could enable treatment for CF patients that may be non responsive to current CF modulating drugs or those that have mutations or refractory to currently available CF drugs.

ABO-401 has a regulatable human mini-CFTR gene that is efficiently packaged into AAV204, one of our next-generation AIM library capsids. In this and other preclinical studies, ABO-401 restored CFTR expression and chloride conductance in airway epithelia, the main cells of the lung that contribute to CF pathology in humans.

Immunostaining has demonstrated that ABO-401 is able to transduction express CFTR in basal cells from the human airway epithelia, increasing our confidence in using AIM vector as a delivery tool for gene editing as well as gene replacement strategies. ABO-401 was also shown to transduce human CF nasal and bronchial epithelial cells, with CFTR-specific change in short-circuit current that was comparable or superior to existing modulator therapy in these same cells. Robust expression of AAV204 in the lungs of CF mice was observed and demonstrated that the AAV204 capsid was equally or more efficient at delivering gene expression cassettes to the lung compared to other naturally occurring AAV capsids.

Further, the data demonstrated that ABO-401 restored CFTR-specific nasal potential difference in CF mice, and that the ABO-401 gene expression cassette makes a fully processed CFTR. In May, we presented data at the Association for Research in Vision and Ophthalmology Annual Meeting in Vancouver, demonstrating the AIM platforms broad therapeutic potential in potentially treating retinal diseases. The presented data showed that intravitreal administration of the AAV204 capsid to non-human primate eyes led to robust transgene expression in the inner and outer retina as well as intense expression in the fovea 25 days post-administration. AAV204 also transduced photoreceptor cells in retinal explants and transduced the outer retina. These data support the potential use of intravitreal administration to deliver gene therapy and gene editing strategy in in-patient and out-patient setting for a wide range of inherited and acquired retinal diseases. 80% of eye diseases affect the photoreceptors in the retina, highlighting the importance of cellular targeting and how AAV can be an efficient tool in gene delivery. An important consideration is route of administration. And we have shown data in rodents and non-human primates that we can utilize different AIM capsids to target the photoreceptors and retinal pigmented epithelium using either subretinal or intravitreal injections. The AIM capsids increased our potential for targeting multiple eye disorders through delivery of therapeutic genes or employing the machinery to enable gene editing.

Lastly, we have now demonstrated in vitro that select AIM capsids can evade neutralizing antibodies against naturally occurring AAV capsids that may be present in some patients. This finding is significant as it may allow AIM-based AAV gene therapy for patients that may have previously been excluded owing to existing anti-AAV antibodies and also may enable retreatment of patients who previously received AAV gene therapy with other serotypes. We look forward to discussing these and additional AIM programs in the future.

I will now turn the call over to Christine who will review our financials. Christine?

Christine Silverstein -- Chief Financial Officer

Thank you Tim. I would like to remind everyone that we have recently filed a 10-Q, where you can kind all the specific information on our financial results. But in summary, our cash, cash equivalents and marketable securities as of March 31, 2019, were $68.3 million compared to $85 million as of December 31, 2018. Net cash used in operating activities for the three months ended March 31, 2019, was $15.1 million as compared to $4.1 million in the same period of 2018, an increase in cash outflows of $11 million.

R&D expenses for the three months ended March 31, 2019, were $11.7 million compared to $8.2 million in the same period of 2018. The increase in research and development expense was primarily attributable to increased R&D headcount related to facility costs and internal manufacturing costs. General and administrative expenses for the three months ended March 31, 2019, were $5.7 million compared to $2.9 million in the same period of 2018. The increase in G&A expenses was primarily due to increased headcount and related facility costs. Net loss was $0.39 per share for the first quarter of 2019 compared to $0.22 per share in the same period of 2018. That's the summary financials.

With respect to upcoming Investor Relations and scientific conferences, I'd like to highlight that we'll be participating in the following events. On May 22, we'll be participating in the RBC Healthcare Conference; and on June 5, we will be at the Jefferies Healthcare Conference, both taking place in New York City. Later this summer, on July 30, we will be participating in the Wells Fargo Biotech Corporate Access Day in Boston. We will update you on all those planned presentations as we get closer to the events.

And with that, I would like to turn it back over to Joao.

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

Thank you, Christine. In summary, we achieved important interim milestones in the first quarter that positioned Abeona well to deliver long-term growth and value for our shareholders and turn hope into reality for our patients. Thank you. I will now turn over to the operator to open up for questions. Operator?

Questions and Answers:

Operator

Thank you, Doctor. (Operator Instructions) And we'll take our first question today from Maurice Raycroft with Jefferies. Please go ahead, sir. Your line is open.

David Steinberg -- Jefferies & Company, Inc. -- Analyst

Hi. This is David for Maury. Thank you for taking my questions. My first question is regarding ABO-101 program for MPS IIIB. Can you talk about the individual age of the patients they have treated so far, and are you aimed to recruit quota patients below age of two?

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

Sorry. Could you ask the second -- this is Joao. Hi, thanks for your questions. So, could you just repeat the last part of your question regarding the --?

David Steinberg -- Jefferies & Company, Inc. -- Analyst

Yes. So, you mentioned before that you are trying to enroll certain number of patients below the age of two. So, I am just wondering can you talk about the sort of quota you are trying to aim for, for the number of patients below age of two?

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

Okay. So, just a clarification, so thanks for your question again. Clarification, no, we are not restricting to below age of two. We are allowed to enroll from six months and up, and the enrollment criteria for the trial is based on cognitive functioning. So, participants need to have at least 60% of the normalized cognitive function for the age, which if you back into the age, it will be up to patients approximately 3.5 years. So it's not restricted by age, but rather by cognitive function, and of course, the patients that were enrolled or eligible for this criterion.

David Steinberg -- Jefferies & Company, Inc. -- Analyst

Okay. That's helpful. So, just to clarify, you are saying that -- can you disclose the number of patients who are under, you know, in terms of the age for the update in the second half for the Type 3 patients?

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

I don't have the age handy, but Dave, actually all met enrollment criteria.

David Steinberg -- Jefferies & Company, Inc. -- Analyst

Okay, good. So then just kind of a follow-up with the ABO-102, can you comment on the screening and the enrollment rates at this time? And where are you finding these new patients? And are you doing anything different with finding these patients at this time?

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

So, we continue to screen actively and we have in fact screened several additional patients, but some of the patients had no mutations which make such that there's a risk for developing antibodies against the transgene protein. So, these are not eligible for the trial.

David Steinberg -- Jefferies & Company, Inc. -- Analyst

Okay, thank you. Thank you for your time.

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

Thank you.

Operator

Our next question will come from Liav Abraham with Citi. Go ahead sir, your line is open.

Liav Abraham -- Citi -- Analyst

Good morning. Two quick questions for me. Firstly, regarding ABO-102, my understanding is that you are meeting with FDA regarding this clinical program in the second half of this year. Please correct me if I am wrong, and if I am not, what can we -- what do you hope to get from this meeting and what can we expect to hear from you? And then secondly, on your EB-101 program, given that this -- the pivotal trial is about to commence pretty soon, could you comment on progress that you are making on the commercial front, if any, at this stage to prepare for the launch of the product and any early discussions with payers that you have had so far? Thank you.

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

Thank you, Liav. So, two answers. So, the first one, we have not disclosed plans to meet with the agency. And generally speaking, we don't provide guidance on this. This is obviously a program where we have designations, including RMAT designation that allows to have ongoing discussions with the agency. So as the data for follow-up of the patients become known to us as patients come back for their scheduled follow-up, we will obviously be analyzing these data points, and at some point, reengage the agency when we have some more critical mass in terms of the data set. We have not planned a specific date or time for this. We do expect to update the Street and of course, internally and also our advisors when we have sufficient data in the second half of this year and this much we have announced. On the EB-101, the RDEB program, you asked whether -- so first of all, obviously we have not yet started Phase 3, we continue to be on track for a midyear 2019 start. And as you can imagine, Phase 3 starts especially in a program that we hope we will enroll in a reasonable timeframe and also given all the designations in the unmet need that the progress can actually accelerate as we move from Phase 3 in terms of commercial launch preparations. So, we have been very active in preparatory work, but we have not yet in terms of the commercial landscape assessment of the addressable patient population and on some assumptions of the commercial opportunity. We have not yet engaged formally with payors at this point, but we will as soon as we are in Phase 3.

Liav Abraham -- Citi -- Analyst

Thanks for the feedback.

Operator

Thank you, Ms. Abraham. And we'll take our next question from Raghuram Selvaraju with H.C. Wainwright. Your line is open. Please go ahead.

Edward Marks -- H.C. Wainwright & Co., LLC -- Analyst

Good morning. This is Edward Marks on for Ram. Thanks for taking the questions. You just mentioned enrollment for the EB-101 Phase 3. I am just wondering if you could provide a little more clarity on the timeline that you are expecting for full enrollment? And then looking at little bit into the future, what long-term clinical follow-ups might be necessary to support that regulatory approval for EB-101 following the Phase 3?

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

Okay. Thank you for your question. So, the plan for enrollment in the Phase 3 trial is anywhere from 10 patients to 15 patients, and this is based on the number of wounds that patients have in general. So, the estimate is that we will need about 35 wounds to be assessed as part of the clinical trial, so divided into 10 patients to 15 patients depending on the area involved in these patients. Many of these patients have already been pre-screened at Stanford. So, we are poised to start enrolling as soon as we are allowed to. And we have not provided a formal guidance as to the rate of enrollment, but we hope that given the fact that these patients are pre-identified or most of them are pre-identified that we should be able to have an expedition enrollment rate, and of course, we will provide updates as we make progress. So -- of course, there's a range here, but we expect to complete enrollment in 2020 and hopefully having results also in 2020.

As for the second question, in long term as we announced, we just recently presented follow-up data for the initial cohort of patients treated under the Phase 1/2 trial at Stanford. This follow-up now is a median of three years, so would that two patients actually out to five years with durable wound healing. So, this is quite remarkable to be at this stage of development and having such long-term follow-up. The commitment in terms of the Phase 3 trial is that we will have the primary endpoint assessed to three months and then there's an additional three months of follow-up to a total of six months. And that's to both examined wound healing durability as well as of course is safety. But as you know for gene therapy, we continue to follow these patients long term, although this is not necessarily gating for the BLA submission.

Edward Marks -- H.C. Wainwright & Co., LLC -- Analyst

Okay. Thank you. And following up on that commercial question previously, just wondered what additional work might need to be done to produce manufacturing readiness for a commercial stage process, and will all of that be done at the Cleveland facility or will you be expanding that manufacturing elsewhere?

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

We haven't made a final decision on the site, but we are prepared to move from -- so just to back track a little bit, we have first of all made a tremendous amount of investment and progress in the clinical manufacturing. And that has brought us very close to commercial readiness. Obviously, we are not ready yet until we get a drug approved, but it has really equipped Abeona as a company aside from the geographic localization of the manufacturing. Two, to progress into commercial manufacturing with just few additions. The actual manufacturing process itself will not change substantially from Phase 3 to commercial, as you know, because we need to be the commercial light product, have been tested in Phase 3. We will announce in terms of the plans, we do have -- currently we have facility under lease in Cleveland that would allow us to expand the manufacturing footprint for EB-101. Whether or not this will include commercial in Cleveland or not, we haven't disclosed yet.

Edward Marks -- H.C. Wainwright & Co., LLC -- Analyst

Okay. Thank you. And then just considering the news that just came out this morning on Sanfilippo, just two more quick questions on cognitive function. Just wondering what the best outcome measure would be for the really young Sanfilippo syndrome patients? And how might cognitive function impact be achieved using some of the systemically administered gene therapy similar to some of that using CNS-administered gene therapy such as the intracranial delivery?

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

Okay, yes. So these are two separate questions. First, the neurodevelopmental scales that are used for these trials include scales that have been widely validated. One is the Mullen scale and the other one is the Bayley developmental scale. Both of these are used in this trial and we have normative data based on the natural history of studies that were previously conducted that use both scales. Ultimately, these also can be converted into, we will call age-equivalent cognition, which essentially is comparing the raw results of these scales with the normative data from the general population of infants and toddlers that get tested as part of the validation process. So, I think we have a good grasp on that and as much as you can do a good devaluation of a toddler and for those of you who are parents know, it's not so easy to do that. But these are obviously experienced under psychologist and study centers that do this routinely. So in that sense, I think we have recovered here and I think that's pretty much the standard for these trials. As for the -- can you repeat your next question?

Edward Marks -- H.C. Wainwright & Co., LLC -- Analyst

Yes. Just wondering how -- I'm sorry?

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

Yes. I remembered it's just on the biodistribution, yes. So although it may appear intuitive to deliver intracranially or in the cerebral spinal fluid as a means to deliver to the brain parenchyma, it turns out and I think there's accumulating evidence including our own, from our own studies both in MPS as well as in CLN Batten disease, that intravenous delivery actually provides a substantially higher biodistribution to the brain, to the CNS, to actual brain parenchyma. So, if you look at the actual empirical evidence that is available to us to-date, the most compelling one is the data from AveXis where the SMA children receive intravenous AAV9-based vector therapy for what it is a CNS disorder. So, where there's a lot of sort of recent publications on this and the fact of the matter is that to-date the most advanced and validated outcome with actual demonstrable clinical outcome is for CNS disorders through intravenous delivery.

Edward Marks -- H.C. Wainwright & Co., LLC -- Analyst

Okay, excellent. Well, thank you for all those details and I appreciate you taking the questions.

Operator

Our next question today comes from the line of Edward Nash with SunTrust Robinson Humphrey. Please go ahead. Your line is open.

Fang-Ke Huang -- SunTrust Robinson Humphrey -- Analyst

Hi, good morning. This is Fang-Ke on for Edward. Congratulations on the progress. And first question is, so Joao, you mentioned in the earlier remarks saying that you are considering collaborating externally with your AIM platform. I just want to -- can you help us understand in terms of when you are deciding whether it's external or internal, what other focus are going to be your therapeutic area you want decide to develop internally and what other considerations potentially in the future you are going to think about external collaborations?

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

Thanks. Yes, so it's a compounded question. So I will try to answer as much as I can at this point. So, as you know and we have announced and Tim has nicely recapped, the team at Abeona and Tim's team have been quite prolific in advancing the AIM platform across therapeutic areas because essentially these capsids have shown the potential for targeting different tissues, from brain to the retina, to the lungs and other areas, including the liver as well and kidney potentially. So that opens up to a lot of possibilities. We have announced our core pipeline. As you know, this is public information, but we have many other projects that we have for which we present a scientific data that are not currently formal, not development projects. So, we view partnerships as a tremendous opportunity for Abeona to both add resources to programs that either not prioritize or that could benefit from particular expertise and also accelerate both existing programs and programs that are currently not on the fast track in terms of developmental efforts. So, I think we are looking for partnerships that are win-win for both parties and I think ultimately could add a tremendous amount of value to this very promising platform.

Fang-Ke Huang -- SunTrust Robinson Humphrey -- Analyst

Great. That's very helpful. Just follow-up on the EB-101 program, so you mentioned your primary endpoint is going to be at three months and measuring the 50% wound healing, and I think a competitor proposing that they are going to measure the complete wound closure at three months as well. So, I just want to understand that when you are thinking about your primary endpoint versus one of your competitor's endpoint, what FDA have been saying, what they require, and then why have you decided on the 50%? Clearly, you have shown in your Phase 1/2 trial that a lot of patients actually have more than 70% wound healing like as far as five years. So just want to understand your rationale for choosing 50%. Thanks.

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

Yeah. Thank you. So I can't necessarily comment on the competitors, but I will talk a little bit about our endpoint and the rationale behind it. So, the data presented on the long-term follow-up and then also a publication and draft that will come out basically shows that greater than 50% wound healing in the large RDEB wounds, right. Keep in mind that we are talking about wounds that are greater than 20-centimeter square, sometimes much larger than that. In fact, 40 centimeters, sometimes 60-centimeter square. The wound healing greater than 50% is very clearly associated with improvement in patient reported outcomes, namely pain, reduction and reduction in itch as well as their global impression of improvement. So, we know that this 50% threshold is clinically meaningful based on our own data. Obviously, we will measure wound healing as a percentage of the original wound. So, it's a continuous measurement even though the endpoint itself, the mechanics of the endpoint is the proportion of wounds that reach at least 50%, obviously would not planning to low ball it, and of course, to the extent that we replicate the data from the Phase 1/2, this percentage would be much higher. But keep in mind that these are large wounds, the skin surrounding -- even the product application, so the skin itself, the remaining skin around the wound is also not normal. So, what the concern here especially for larger wounds is that you may have near full healing, which we did actually report in several of these patients, but we don't want to be penalized if there's a small area that might not heal in the edges where small abrasion areas, because the skin's somewhat sensitive. So, that could throw the baby out with the bath water. So, we want to make sure that any substantial healing, which we believe to be greater than 50% is a clinically demonstrable threshold. And actually by the patients themselves, they have been very clearly articulating that any healing in a large wound is meaningful to them.

Just to remind everyone, the wounds, the selection of patients for the Phase 3 trials are patients such that they have chronic wounds and these are greater than six months in duration. Sometimes they have those open wounds for several years as well as wound sizes that are greater than 20 centimeters in area -- square centimeters in area. So, these wounds based on all the natural history data that's been published, tend not to heal, certainly not in three months and in most cases, not even in years. So in fact, one could argue that any healing of these wounds will be clinically meaningful and we're setting it up 50%. The experience too from the natural history studies is that smaller wounds, especially the very small wounds as some of the competitors are targeting, tend to heal spontaneously even without intervention. So, in very small wounds, it may be plausible to AIM at complete wound healing, which would be an expectation if we were to use EB-101 as well. But that's not what we are targeting in the clinical trial.

Fang-Ke Huang -- SunTrust Robinson Humphrey -- Analyst

Yes, that's really helpful. And then lastly, so in the news you put out this morning regarding the Sanfilippo B and you mentioned you started dosing the first patient in cohort 2. So is that just to confirm whether right now you have four patients enrolled in the trial, and three in cohort 1, and down one in cohort 2, is that right?

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

No. We have not announced that, but we do actually have only two patients in cohort 1. We've been -- this is a joint decision between Abeona, of course our data safety monitoring board as well as the regulators based on the excellent safety of the first patient who is now one plus year for post dosing. So this is somewhat unusual that we have such long sort of follow-up for the first patient and also the excellent outcome so far in terms of safety of the second patient enrolled earlier this year. So, with these two patients, we felt that the safety of this program also combined with the safety on the MPS IIIA program which uses the same capsid as well as the increasing data, safety data collectively on use of AAV9-based vectors that we have, we are in good grounds to propose a faster dose escalation, which I think ultimately stands to benefit progress of this trial, but ultimately provide these children with their best chance for a clinical benefit. So, we approached the DSMB who reviewed very carefully all that safety data and also approached the regulators who supported this transition. So, I think this, in the end, is a testament to the safety of the program of both the IIIB program and the IIIA program.

Fang-Ke Huang -- SunTrust Robinson Humphrey -- Analyst

Great. Thank you so much for taking my question.

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

Thank you.

Operator

Our next question comes from Kennen MacKay with RBC Capital. Your line is open, please go ahead.

Vikram Kaushik -- RBC Capital Markets, LLC -- Analyst

Hi guys, this is Vikram on for Kennen. Thanks for taking our questions. Couple for us, from the morning -- your announcement on ABO-101 in MPS IIIB, could you remind us what type of data shall be expect second half of 2019,And what specific magnitude on the new development cell scale if you are referring to, Mullen scale, shall we expect or anything you can share on that would be really helpful?

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

We haven't provided any guidance in terms of the magnitude of cognitive. Obviously, the goal here is both to treat the underlying pathology that is the enzyme that is defective. And for that, we can measure the biomarkers as we have done for MPS IIIA. So, we look at CSF and urine heparan sulfate, which is the substrate of the enzyme. So, the expectation is that we will continue to bore the accumulation of GAGs systemically including the CSF. And that was the case for the first patient treated in this trial. We will continue to measure cognition. Obviously, cognition is somewhat of more a variable outcome and more meaningful results will come at six months in a year. So we will provide data that we have, but obviously patients dose just now won't have a very long-term follow-up for cognition.

Vikram Kaushik -- RBC Capital Markets, LLC -- Analyst

Thanks for the color. That's really helpful. If I can squeeze in one follow-up on your licensing agreement, how should we be modeling that in our model with REGENXBIO? Where would the $10 million payment that is expected to go out this year, shall we think about or shall we be thinking about that?

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

Yes. So, that's basically the anniversary of the agreement. And so, we will pay one more $10 million in the fourth quarter, I think of this year.

Vikram Kaushik -- RBC Capital Markets, LLC -- Analyst

Thank you so much. Really helpful.

Operator

We will take our next question from the Difei Yang with Mizuho. Please go ahead. Your line is open.

Alexander Lim -- Mizuho Securities -- Analyst

Hey, good morning guys. This is Alex on for Difei. Thank you for taking the questions. Just on EB-101 and the FDA review, could you comment on what are the gating factors for the review to conclude?

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

There are not specific gating factors, it is just -- because we completed the work internal manufacturing work, this is still being reviewed by the agency, this is just the totality of all the data, it's mostly quality assays.

Alexander Lim -- Mizuho Securities -- Analyst

Okay. Thank you. And then you mentioned a data update for the MPS IIIA program second half of 2019, maybe you could just comment on what we should be expecting there? Thank you.

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

We are expecting -- so these patients who have already been enrolled come back approximately every six months for follow-ups. So, we will provide similar types of data sets including biomarkers and cognitive data from the patients already enrolled.

Alexander Lim -- Mizuho Securities -- Analyst

Okay. Thank you.

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

Sure.

Operator

Our next question will come from Elemer Piros with Cantor Fitzgerald.

Elemer Piros -- Cantor Fitzgerald -- Analyst

Yes. Good morning, everyone. Joao, just following up on this last question, so if you plan to provide an update on the IIIA program in the second half, based on the enrollment pattern, they might have more than one year or more data for all 14 patients. Would that be a correct assumption?

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

I'm trying to think off the top of my mind. If you probably look into the data, I am not sure if all the patients, but certainly for the large proportion of the patients. Clearly, cohort 1 and 2 are well past one year for sure, at your past two years for cohort 1.

Elemer Piros -- Cantor Fitzgerald -- Analyst

Yes. I mean just, based on your disclosure last November, you had 14 patients by then. And now how many more patients in this trial do you expect to enroll?

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

Will depend on how many more we need to basically corroborate the findings we have now. So, the patients, so first -- sorry missed pause here. The trial is still open for enrollment. It was still actively screening, have screened several more patients and as mentioned earlier, part of the screening process, we are excluding patients. We have historically excluded patients who have no mutations or mutations that will lead to no protein production. So, the enrollment is open. So, we haven't made a final determination and a lot of it will depend on next steps once we have analyzed the full data set that we are disclosing later this year. I don't know if that makes sense, but it's largely data driven, you know, how much many more patients will enroll.

Elemer Piros -- Cantor Fitzgerald -- Analyst

And I'm presuming the same would be the answer to the IIIB program, which you set your plan to roll up to 9. I don't know if that number has changed or will it change based on what you will see in this 8 or 9 -- first 8 or 9 patients to extend that into your program.

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

Yes. We may adjust if we need to. For now, 9 patients seems a good estimate, but obviously as the program progresses and we get additional data, and as you know, we may adjust this, but for now that's what we're announcing the plans are. Of course, this is a combination of safety data, which is critical obviously for patients as well as efficacy data. In terms of safety, the IIIA and also to some extent to IIIB although much fewer patients, but given the long-term follow-up, we are pretty confident that we have a fairly thus strong safety data set so far. So I think at least that aspect of the program is well served.

Elemer Piros -- Cantor Fitzgerald -- Analyst

And I don't mean to ask you to comment on a competitor as a projection, but in IIIA, company X have enrolled two patients and project to enroll up to 20 patients during the next 12 months. Just, I mean, you have probably the most experience in the field in terms of finding eligible patients. How ambitious in general to enroll 20 IIIA patients in 12 months time? Again specific to the competitive or just in general, your experience?

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

Yes. It's not easy. These are above -- although it depends there, I think the competitor has similar entry criteria. So especially by limiting the subset of the population with a higher cognitive functioning, it will require a very broad concerted effort globally not just U.S. here.

Elemer Piros -- Cantor Fitzgerald -- Analyst

I see. Turning on to the EB program, so the CMC package is complete. Now, your interactions with the FDA, would it entail a facility inspection as well?

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

No, not planned. No. They had a DMPQ assessment a while back for the GMP qualification of the facility. But there was no inspection planned although the agency of course has the right to inspect at any time.

Elemer Piros -- Cantor Fitzgerald -- Analyst

And you feel that the protocol is actually locked down? What do you need to have a final determination?

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

We will announce when we have all the final dot the i's and cross the t's.

Elemer Piros -- Cantor Fitzgerald -- Analyst

Okay. Thank you. And one last question, do you plan to engage additional centers, besides Stanford for the Phase 3?

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

Yes. So we have actually announced this previously that this is a multi-center trial. So, Stanford obviously will continue to be the lead center because they have the most experience and they are fully prepared to enroll the first patient. The question is what are the centers could come on online and how fast? And we have a couple of candidates centers that we have been in discussions with in preparation. So, we expect those will be announced later this year.

Elemer Piros -- Cantor Fitzgerald -- Analyst

Okay. Thank you so much, Joao.

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

Yes.

Operator

And that was the final question from our audience today. Dr. Siffert, I would like to turn the conference back over to you for any additional or closing remarks, sir.

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

Yes. Thank you very much. And thank you all for joining in for our call today. We are very excited about the prospects for Abeona later this year. We hope to provide new announcements in the coming months. Also wanted to take this opportunity to thank, especially in light of the announcement on the MPS IIIB trial, but also progress across the board and our clinical programs to specifically acknowledge the two centers that have been working very hard to get this trial moving, both Nationwide Children's Hospital led by Kevin Flanigan as the PI, and also the hospital in Spain, Hospital Clinico Universitario de Santiago de Compostela, for doctors Maria Couce and Maria Lopez Castro, so just want to acknowledge them. They have been key advisors to Abeona and put a lot of efforts in this program. And we look forward to also announcing new sites (inaudible) for this program in the near future. And thank you everyone.

Operator

Ladies and gentlemen, this does conclude today's teleconference. And we do thank you all for your participation. You may now disconnect your lines and we hope that you enjoy the rest of your day.

Duration: 51 minutes

Call participants:

Sofia Warner -- Senior Director, Investor Relations

Joao Siffert -- Chief Executive Officer, Head of Research & Development and Chief Medical Officer

Timothy J. Miller -- President & Chief Scientific Officer

Christine Silverstein -- Chief Financial Officer

David Steinberg -- Jefferies & Company, Inc. -- Analyst

Liav Abraham -- Citi -- Analyst

Edward Marks -- H.C. Wainwright & Co., LLC -- Analyst

Fang-Ke Huang -- SunTrust Robinson Humphrey -- Analyst

Vikram Kaushik -- RBC Capital Markets, LLC -- Analyst

Alexander Lim -- Mizuho Securities -- Analyst

Elemer Piros -- Cantor Fitzgerald -- Analyst

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