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Abeona Therapeutics Inc (ABEO) Q2 2019 Earnings Call Transcript

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Abeona Therapeutics Inc (NASDAQ: ABEO)
Q2 2019 Earnings Call
Aug 12, 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. Second 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 Chief Executive Officer. Following Joao, Tim Miller, our President and Chief Scientific Officer, will present preclinical highlights; and Christine Silverstein, our Chief Financial Officer, 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 report on Form 10-K and quarterly report on Form 10-Q to 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 to you, Dr. Joao Siffert. Joao, you have the floor.

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

Thank you, Sofia, and thank you all for joining us today for our second quarter results and business highlights, which we believe reflects substantial progress in the continued development of our clinical and preclinical programs, as well as our manufacturing and quality operations. Today, I'll review the key quarter accomplishments and recent events and then turn the call over to Tim, who will discuss the developments within our preclinical programs and updates from our next-generation AIM capsid platform.

I'd like to start off with an update on our recessive dystrophic epidermolysis bullosa program. As a reminder, RDEB is a rare and severely debilitating 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. Without functional Collagen VII, RDEB patients have extremely fragile skin, the blisters and tears, ultimately leading to multiple wounds, some of which remain open for years and cover a significant amount of the body. RDEB wounds are very painful lead to multiple dermatological and sustained complications and currently have no effective treatment options.

Patients rely on palliative treatments that include time-consuming and expensive wound care to protect open wounds, reduce pain, prevent infection and often require comprehensive pain management. We're currently developing EB-101, autologous, gene-corrected cell therapy that restores normal functional Collagen VII to keratinocytes and their progenitors. Each EB-101 gene-corrected keratinocyte sheet, which can cover an area of approximately 35 to 40 centimeter squared, is transplanted into open wounds provided -- providing prompt wound healing. Two skin biopsies allow manufacture of six EB-101 sheets, which combined can cover wound areas measuring up to 240 centimeters squared.

To our knowledge, EB-101 is the only product in development for RDEB with clinical data, so we could heal large chronic wounds that had been open for years. The Phase 1/2a trial, conducted by Stanford University, show that EB-101 healed an average of approximately 130 centimeters square per patient and up to 160 centimeter square in some patients with durability of two to five-plus years post treatment. The majority of our RDEB wounds are chronic, often remain open for years and tend to be larger than 120 centimeter square. Wound healing of such large wounds is in itself clinically significant and the long term follow-up from the Phase 1/2a trial show that 50% or more wound healing was associated with meaningful reduction in pain.

Our goal is to bring this transformative therapy to patients with RDEB as soon as possible. We recently met with the FDA to discuss the final stages of a preparation for upcoming Phase 3 clinical trial called VITAL, aimed at confirming the safety and efficacy of EB-101 observed in the Phase 1/2a study. At this meeting, the FDA addressed information that we previously submitted regarding quality testing of the product in certain aspects of our Phase 3 clinical trial protocol. We believe we have successfully completed the manufacturing process of the EB-101 and the request for clarification on chemistry, manufacturing and controls are focused on product transport and on the protocol to assess comparability of the to-be-commercial retrovirus used in EB-101 manufacturing.

In addition, FDA provided feedback on selection, measurement and timing of certain patient-reported outcome measures. We are currently addressing the valuable feedback received and will provide clarification and supplemental documentation for items previously submitted to the FDA as soon as possible. We have requested meetings with the respective FDA reviewers to address their questions, so we can proceed with the Phase 3 trial in fourth quarter 2019. The VITAL Phase 3 study will be a multi-center randomized clinical trial assessing EB-101 for treatment of approximately 35 wound sites across 10 to 15 RDEB patients.

The primary outcome measure of the study will be wound healing at three months, comparing the proportion of treated wound sites with at least 50% healing to untreated ones on the same patients. By protocol, participants eligible to enroll in VITAL will have chronic wounds larger than 40 centimeters squared. In most cases, their chronic wounds exceed 120 centimeters squared, require frequent wound care and are associated with disabling pain and are a great risk for infection. Additional study endpoints will include the patient global impression of change for pain at each wound site compared with baseline as well as measurements of pain intensity during dressing changes in a clinic visit. Patients will be followed for up to six months for assessment of wound healing durability and overall safety. We estimate there are about 2,500 RDEB patients in the US who could benefit from multiple EB-101 treatments, given the large number and size of wounds observed among the population.

The RDEB patient community and their treating physicians are anxious to have EB-101 available as soon as possible. Learnings from the Phase 1/2a trial have equipped our clinical team to best prepare for Phase 3. The Stanford University team is also ready to start involving patients as soon as we receive that key endorsement to proceed. Additional study sites will begin in the ensuing months.

I'd next like to provide an update for a program assessing ABO-102 for MPS IIIA, also known as Sanfilippo syndrome type A, a rare, lysosomal storage disease with no approved treatment that primarily affects the central nervous system or CNS. ABO-102 is our novel gene therapy dosed one time intravenously using a self-complementary, AAV9 vector to deliver two functional copies of the SGSH gene to sell them on the CNS as well as to peripheral organs. The therapy is designed to correct the underlying SGSH enzyme deficiency and prevents cellular accumulation of heparan sulfate that leads to cell dysfunction, cell death and rapid neurodevelopmental decline and physical impairment. We're currently assessing the safety and efficacy of ABO-102 in the Transpher A Study, also known as ABT-001.

This study is a two-year open label dose escalation Phase 1/2 global clinical trial for patients with MPS IIIA who have a developmental quotient of at least 60% of normal levels for age and meet other eligibility criteria. The study primary outcome measure focused on neurodevelopmental progress and safety with secondary measures of behavior, quality of life, heparan sulfate levels and CSF, plasma and urine and brain and liver volume.

Last month, we were excited to share positive interim data for a Transpher A study showing that the three youngest patients involved in the study at ages 26, 19 and 12.5 months at dosing all enrolled in cohort 3 continue to track within normal range of the age equivalent development at ages 44, 31 and 24.5 months, respectively, that is 12 to 18 months post treatment. To our knowledge, our data are the only reported evidence suggesting that a gene therapy treatment could alter the typical nerve developmental course in children with MPS IIIA. We believe our data also corroborates the basic principle that treating neurodegenerative disorders before they become more advanced can provide the best chance for a benefit, especially in a disease that causes rapid neurological impairment within the first few years of life.

The study data show that intravenous ABO-102 administration resulted in a durable reduction in CSF heparan sulfate, a key biomarker of MPS IIIA. Improvement in CSF heparan sulfate was dose-dependent and reached their lower limit of metal detection from eight patients enrolled in cohort 3. Reductions in liver volume were also sustained during the observation period of up to two years. In addition, safety profile of ABO-102 remained favorable and no product-related serious adverse events were reported to-date. We'll continue to screen patients for enrollment in the Transpher A at sites in the US, Spain and Australia. We're pursuing on our meeting with the FDA in the second half of this year to discuss study data and gain clarity on the development path forward.

Moving on, I'd like to discuss our ABO-101 program, an investigational one-time gene therapy for the treatment of MPS IIIB, also known as Sanfilippo syndrome type B. MPS IIIB is a rare, devastating and fatal lysosomal storage disease with no approved treatment that is characterized primarily by rapid neurodevelopmental decline leading to early death. We recently announced that FDA granted Fast Track designation to ABO-101, recognizing the severity and importance of addressing this rare orphan disease.

To briefly review the Transpher B, also known as the ABT-002, is a two-year open label Phase 1/2 study, primarily evaluating safety and neurodevelopment of the MPS IIIB children treated with ABO-101. Eligible patients need to have a developmental quotient of at least 60% of normal levels for age and meet other standard criteria. Secondary outcome measures include CSF in urine heparan sulfat levels, CSF and serum NAGLU enzyme activity in liver and brain volume by MRI. We're pleased to share that to-date we have treated a total of five patients with MPS IIIB, two in cohort 1 and three in cohort 2. Study recruitment efforts continue and we have a queue of patients awaiting screening across sites in the US, Spain and France.

With a post-treatment follow-up ranging from less than one month to more than 20 months post dosing, the overall safety profile remains favorable and there have been no serious adverse events being related to ABO-101. Improvements in disease-specific biomarkers were similar to that observed for MPS III program.

We expect to report interim data for the trial in the second half of this year. We also have made progress advancing our CLN1 program to the clinic. CLN1 disease, also known as, Infantile Batten disease is a rare, fatal, inherited disorder of the nervous system that generally presents in childhood and leads to visual neurological impairment and early death. In May, we announced the FDA clearance of our R&D for ABO-202 and AAV9 gene therapy for CLN1 disease. And in June, we received FDA Fast Track designation for this program. These regulatory milestones in combination with a previously reported preclinical data, which showed a favorable safety profile and low significant toxicities leave us excited about the potential for ABO-202 in CLN1. We will provide guidance on the timing of the study later this year.

Finally, from a company standpoint, this quarter, we'll strengthen our management team with two important appointments. We're fortunate to announce the recruitment of Dr. Victor Paulus as Senior Vice President of Regulatory Affairs and Jodie Gillon as Vice President of Patient Advocacy and Clinical Affairs. Victor brings to Abeona over 30 years of experience in the pharmaceutical industry, including over 20 years specializing in regulatory affairs. His experience will be instrumental as he will bring our gene and cell therapies to patients. Jodie brings over 20 years of valuable industry experience and makes for an ideal person to lead our patient advocacy and clinical affairs functions in close collaboration with patients, families, medical and scientific stakeholders. Both Victor and Jodie have already had an immediate positive impact for Abeona.

Before I turn the call over to Tim, I'd like to close by taking a moment to heartfully thank all the patients, families, clinicians and patient organizations who have participated in and partnered in our efforts to advance our mission of working together to deliver gene and cell therapies for people impacted by serious diseases.

With that, I'll now turn the call over to Tim. Tim?

Timothy J. Miller -- President & Chief Scientific Officer

Thank you, Joao. During the second quarter, we reported new preclinical data developed from our AIM capsids platform, a next-generation of adeno-associated virus capsids for use in gene therapies. The AIM capsid library can utilize AB biology to selectively target delivery of genetic payloads to the central nervous system, lungs, eye, muscle, liver and other tissues. In April, we presented new data from the ABO-401 program, our novel gene therapy for cystic fibrosis at the American Society of Gene and Cell Therapy 22nd annual meeting in Washington, DC.

ABO-401 has a regulatable human mini-CFTR gene that is efficiently packaged into AAV204, one of our next-generation library capsids. The data presented at ASGCT demonstrate that ABO-401 efficiently delivered a highly expressed functional copy of human mini-CFTR to the lung of CF mice and restored CFTR lung function in human CF patient nasal and bronchial epithelial cells. This adds to the growing body of evidence suggesting ABO-401 may address the challenges in lung delivery and transgene expression that have limited the advancement of gene therapy for CF patients.

In this and other preclinical studies, ABO-401 restored CFTR expression and chloride conductance in airway epithelial, the main cells of the lung that contribute to CF pathology in humans. 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 the ABO-401 restored CFTR-specific nasal potential difference in the CF mice and that the ABO-401 gene expression cassette that makes a fully functional and processed CFTR. We believe that the recent data are encouraging and ABO-401 is a promising candidate that may ultimately change the landscape of CF treatment by introducing a one-time gene therapy.

Also during the quarter, Abeona presented new preclinical data of our -- on our novel AIM AAV204 capsid, demonstrating the broad therapeutic potential of AIM gene therapy in retinal diseases. The data were presented in May at the Association for Research in Vision and Ophthalmology Annual Meeting in Vancouver. The data showed that intravitreal administration of our novel AIM AAV204 capsid to non-human primates led to a robust transgene expression in the inner and outer retina. Expression was observed in the peripheral retina and fovea 25 days post administration.

AAV204 also transduced the photoreceptor cells in retinal explants and transduced the outer retina with positive green fluorescent protein expression. These data support the potential use of AAV204 for intravitreal administration to deliver gene therapy in an outpatient setting for a wide range of inherited and acquired retinal diseases. The non-human primate data were complemented by findings from mouse models, which identified AAV204 as one of the three lead candidate AIM capsids that demonstrates robust transduction of retinal cells. The data demonstrated in mice that intravitreal administration resulted in broad retinal expression of AAV204 that penetrated to the photoreceptor and retinal pigmented epithelial layers.

The broader retinal tropism of AAV204 capsid in non-human primates underscores the potential of our platform to deliver gene therapy beyond inherited diseases, including the treatment of acquired retinal disorders that may be currently underserved. Intravitreal administration of AAV gene therapy, which does not require surgery, could potentially be performed in an outpatient setting and may be a safer and less-invasive approach than suffering administration. AIM vectors are non-replicating and have shown the potential to evade the immune responses generated by exposure to naturally occurring AAV vectors. Our library contains more than 100 capsids with tissue tropism selected for the potential to target a wide range of organs and multiple routes of delivery.

An important consideration is the 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 the retinal pigmented epithelium using either subretinal or intravitreal injections. The AIM capsid increased our potential for targeting multiple eye disorders through delivery of therapeutic means are employing the machinery to enable gene editing.

Lastly, we have now demonstrated in vitro that selected AIM capsid can evade neutralizing antibody against naturally occurring AAV capsids that may be present in some patients. We believe this finding is significant as it may allow AIM-based AAV gene therapy for patients who have been previously treated or excluded from study participation owing to existing anti-AAV antibodies. Evading anti-AAV immunity could potentially also 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. We have recently filed a 10-Q where you can find all specific information on our financial results. But in summary, our cash, cash equivalents and marketable securities as of June 30th, 2019 were $62.5 million compared to $68.3 million as of March 31st, 2019. Net cash used in operating activities for the quarter was $15.2 million as compared to $9 million in the same period of 2018, an increase in cash outflows of $6.2 million. R&D expenses for the three months ended June 30th, 2019 was $16.3 million compared to $7.9 million in the same period of 2018. The increase in research and development expenses primarily attributed to increased in-house manufacturing activities and related headcount costs. General and administrative expenses for the quarter were $5.6 million compared to $4.6 million in the same period of 2018. The increase in G&A expenses is primarily due to the increased headcount and related facility costs. Net loss was $0.49 per share for the second quarter of 2019, compared to $0.26 per share in the same period 2018. That's the summary financials.

With respect to upcoming investor and scientific conferences, I'd like to highlight that on September 4th, we'll be in Boston, attending the Citi's 14th Annual Biotech Conference and on September 5th, we will also be attending the Wells Fargo Securities Healthcare Conference also in Boston. We will update you on those planned presentations as we get closer to the events.

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

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

Thank you, Christine. In summary, in the second quarter, we have made important progress that we believe positions Abeona well to drive forward our mission of turning hope into reality for our patients.

Thank you. I'll now turn over to the operator to open up for questions. Operator?

Questions and Answers:

Operator

Thank you. The floor is now open for your questions. [Operator Instructions] We will take our first question from Maury Raycroft of Jefferies. Please state your question.

Maury Raycroft -- Jefferies -- Analyst

Hi. Good morning, everyone, and thanks for taking my question. First question is on ABO-101. It seems like you expanded the 5E13 cohort from three to six to four to seven in July, added an efficacy-based primary endpoint to look at neuro target and then added some secondary endpoints as shown on clinicaltrials.gov. And so I'm just wondering if there are any specific reasons why some of these changes were made and if FDA or KOLs encouraged the changes? And then just bigger picture, what else is -- what is needed in IIIB to eventually seek approval for this indication?

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

So just to be -- hi, Maury, Joao here. I'll take this question. So you were referring to the MPS IIIB programs. is that correct as I understood?

Maury Raycroft -- Jefferies -- Analyst

That's correct.

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

[Speech Overlap] beginning of it? Yes. So these are essentially changes to harmonize the IIIB program with the IIIA program. So what we're trying to do is again focus the enrollment of patients who we believe are not -- that have not undergone much advancement in their disease and therefore could stand to benefit the most from the therapy. Not to say that children who have more advanced disease couldn't benefit, it's just that the effect size likely would be larger in children who were still functioning at a higher level and that's codified by setting up the inclusion criteria with a developmental quotient of 60% or greater. So is that your question and then we will continue to take the same approach on the IIIB program as we have on the IIIA program and that we continue to collect both biomarkers and the developmental data from this program and once we have sufficient data to identify clinical benefit beyond just the biological benefit of markers, which we expect to see and have seen in the first patient, expect to see in the ensuing patients will again engage the agency in discussions for the further development of the program.

Maury Raycroft -- Jefferies -- Analyst

Got it. That's helpful. And then, for the IIIB patients, can you talk about some of their baseline characteristics at this point? Maybe anything on age and baseline neurocog function? And then it seems like some -- yes, I'll let you answer that and then do a follow-up.

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

Yes. So, these patients meet the eligibility criteria. So, we'll not disclose specific age at this point. We'll provide an update on the study overall, including some early data readouts later this year. As you know and since this is an update from the past quarter, these patients were enrolled relatively recently, all but one in 2019. So relatively early follow-up. But they meet criteria for the developmental quotient. That is in general looking at the -- just as a framework and then not necessarily providing specific ages, which I don't know by heart for all the patients, but a child with the developmental quotient of 60% or greater who has either MPS IIIA or IIIB with the rapid progressive phenotype tend to be usually younger than 40 months or so, give or take. They tend to be younger.

Maury Raycroft -- Jefferies -- Analyst

Got it. Okay. And for IIIB also, it seems like some other companies enrolling IIIB studies have had a difficult time enrolling those studies. It seems like your enrollment has picked up recently. Is there anything that you can say about enrollment and potential investor -- investigator enthusiasm for your particular approach in IIIB?

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

Yes. I think there is a good momentum on both programs, actually, although IIIA, we have not enrolled anyone this year yet, we have continued to screen patients who unfortunately were not eligible for the trial and then we continue to screen patients as we speak. So, we hope to enroll patients later this year. IIIB and I think IIIA both are benefiting from an effort that was started sometime last year to really expand our footprint in terms of clinical trial sites. The sites continue to be very engaged. We have also a pretty concerted international outreach and that's -- the question of whether IIIB, which tends to be viewed as less common than IIIA at least in the United States, it may not be as uncommon as we thought to be in some European countries and other places such as South America. So we're having success identifying these patients.

I also believe that with the continued positive data in IIIA from the standpoint of the maintenance of improvements in biomarkers now up to two years in CSF heparan sulfate as well as the recent data we announced on the youngest patients showing some neurocognitive preservation, I think that also creates the momentum in terms of interest that we're showing that this is not only a biologically active intervention, but also seems at this point that we can also demonstrate some preservation of actual clinical data, which is important.

Maury Raycroft -- Jefferies -- Analyst

Got it. Yes. And for IIIA, for those young and higher functioning patients that you're treated and you're showing their preservation of neurocog function, can you just comment on their underlying IIIA mutation and also the strength of natural history data over a similar 12 to 18 month time frame for those particular patients?

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

Yes. So the patients by protocol eligibility, all patients need to have mutations that are associated with a rapid progressive phenotype. So in general, if you look at the various natural history studies, including the one we've worked most closely with from National Children's Hospital, but if you look at the Pittsburgh data, also data from University of Minnesota, albeit it was a different scale but certainly something that we should look at because it's a pretty comprehensive dataset as well.

Most children with the rapid progressive form of MPS IIIA or IIIB for that matter tend to plateau in their new development before they reach age of three almost by a rule. Obviously, this is biology, not sort of mathematics, but by and large. And if you look at all these studies, most of these children decelerate in their development during their second year of life and before they reach age three, they tend to plateau in their neurodevelopment and start declining.

So anything that sort of deviates from that in terms of the neurocognitive development in this case, following the intervention with ABO-102, we believe it could be already an indication that this treatment is having a biological effect and also diverging in terms of the neurocognitive course. Does that your answer question?

Maury Raycroft -- Jefferies -- Analyst

Yes, that does. And Just last question on EB-101. So, for the CMC protocol item, particularly the one related to their retrovirus batch, just to be clear, you don't need to accumulate any new data for this until after the trial has started, right? And then I'm wondering -- yes, and then I'm wondering how much our Brammer is involved with helping with the protocol design and finalizing the protocol for this?

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

So, two separate questions. So, what we are submitting to the agency, the request is the actual protocol. We're not submitting any data going ahead of the Phase 3. So the transfer from the original retrovirus to the Brammer retrovirus, which has been planned all along in agreement with the agency, will take place during Phase 3. So, we will be conducting the comparability studies, which are no different than the release -- generally speaking, no different than the release of studies that we do, quality assurance that we do for any kind of GMP product. So in this case, for ABO-101.

So, we'll be testing those for the Indiana University retrovirus material and testing those for the Brammer retrovirus material as we would do anyway to -- the only thing we'll do in this to set up a protocol prospectively to show what the acceptance criteria are, which are generally already agreed upon with the agency. So, it's literally having the protocol written. Data will be generated as we release these batches for clinical use as we would normally.

Now, we have used the Brammer material to manufacture a sheet all throughout the past say six months. These are obviously not for clinical use, but certainly using the same retrovirus that we are intending to use them to Phase 3. So we have experience using the retrovirus and it has performed well as expected. So, we don't anticipate any surprises here.

Maury Raycroft -- Jefferies -- Analyst

Okay. And then how involved is Brammer in the process for you with helping finalize these protocols?

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

They have been very good partner of Abeona for a while now and very closely -- we'll work closely with them. Yes. And we have already, so it's not forward-looking, this has taken place, this part of the whole manufacturing readiness for the program has included manufacturing ABO-101 using the Brammer retrovirus. So we've done this before. This is not the first time we're going to do this. This is just going to be officially for -- during the Phase 3.

Maury Raycroft -- Jefferies -- Analyst

Got it. OK. Thank you very much for taking my questions. And I'll hop back in the queue.

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

Sure. Thank you.

Operator

Our next question comes from Kennen MacKay of RBC Capital. Please state your question.

Bikramjot Singh -- RBC Capital -- Analyst

Hi, guys. Thanks for taking our questions. This is Bikram on for Kevin. So, we had one on the patient-related outcomes that actually had -- has to be included in the Phase 3 study for EB-101. Could you please elaborate what those outcomes are and how would this further validate maybe the wound healing it will show in these patients?

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

Yes. Thank you for the question. The patient-reported outcomes, there are several. The main one being pain. Especially for the larger wounds that we're addressing in our Phase 3 trial, pain is among the most disabling patient experiences. Obviously, having an open wound has all sorts of other medical implications, including risk of infection and the need for repeat wound dressing and then extensive wound care and whatnot but the pain is really the main cause of sort of discomfort and suffering for these patients.

So the FDA is obviously interested in understanding how wound healing and pain intersect. If you ask patients, they'll tell you that their intact skin, there is a healed wound doesn't hurt and if you ask them which wounds hurt the most, they uniformly will tell you the larger wounds. Smaller wounds are not -- they can still be uncomfortable, but the larger wounds tend to be the ones mostly associated with higher intensity of pain. This has been shown not only anecdotally by asking the patients, but also in natural history studies that's been presented.

So, FDA wanted to just agree with us in exactly timing and how to collect those using scales that are pretty well known and it's been validated in pain trials before. So, it was not much about the scales themselves, it's just about the collection timing in relation to some of the wound care, basically wound care as they remove the bandages that can cause pain, because sometimes the bandages, especially in the larger ones, get stuck. Also, as you tender to the wounds and cleanse the wounds can cause also acute pain. So the patients have both the chronic pain from just having these open wounds chronically but then acute exacerbations of pain during procedures. So it was just a matter of getting coordinated and making sure we get as much information as possible in those while the patient is at home, but also recalling the experience when they come to the clinic visits.

Bikramjot Singh -- RBC Capital -- Analyst

Got it. Just to follow-up on that. Is there are any specific percentage of pain improvement you're looking in these patients? Or will it just depend from wound to wound, depending on the size of the wound? How are you thinking about that?

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

I'm sorry. I missed the -- can you repeat the first part of your question? I just couldn't hear well.

Bikramjot Singh -- RBC Capital -- Analyst

Absolutely. So just to follow up on that, I was thinking what percentage of improvement on the pain scales that you said are pretty standardized? Will you be looking to -- will it be something similar to, as you said 50% wound healing? How shall we be thinking about that?

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

Yes. So, this is -- this is sort of the difficult question about EB because there aren't [Phonetic] much data for EB specifically. There's obviously a ton of data in pain, both chronic and acute pain files in variety of conditions from cancer to neuropathic pain [Indecipherable] migraines, but as you can imagine, there's not a lot of this for EB. The sense that we get from talking to patients is the wound healing essentially large relief for pain also can be quite variable. And it's served under wraps, meaning that the wound is well dressed and they're not touching it or cleaning it. There's some base pain, but it's not that severe. Of course, as you remove the wound dressings that the pain spikes up. So there isn't a set threshold to success. Ultimately in the end, the intent here is to heal the wounds and of course, having an open large wound in of itself is clinically very meaningful.

So if you could heal a large area of wound, this in of itself will cause much benefit to the patients and all the ramifications of EB wounds. But we would expect that it will also provide us with a pain relief. So we'll measure as a continuous variable as well as categorical variable.

Bikramjot Singh -- RBC Capital -- Analyst

Got it. Got it. That is super helpful. Thanks for the color.

Operator

Any further questions, sir?

Bikramjot Singh -- RBC Capital -- Analyst

That would be all.

Operator

Thank you. Our next question comes from Joon Lee of SunTrust. Please state your question.

Fang-Ke Huang -- SunTrust -- Analyst

Hi. Thank you for taking our question. This is Fang-Ke Huang for Joon. I'm just wondering if you can tell us a bit more about the timing of initiating the EB online trial. It's going to be an early 4Q [Phonetic] event or you're going to anticipate going to be late 4Q event or it's too difficult to tell at this moment?

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

So we can't provide any more guidance than we already have provided publicly in the case filing. Much of what remains there are questions about quality and this particular question about PROs that we've been discussing. The timing now for initiations is entirely predicated on resolving these questions. So, this is largely driven by the timing of how efficiently we can communicate with the agency. On our end, we're working diligently to get all the answers submitted soon. So just a matter of ensuring that FDA is satisfied with the answers and that if there are any remaining questions that we can address those ASAP. From the actual trial readiness in terms of the clinical operations and the ability to manufacture the product, we feel that we're ready as soon as the FDA is ready. Sanford has obviously been very close partner that we're dealing for years now and they are ready and willing and eager to start as long as we get the final go ahead.

Fang-Ke Huang -- SunTrust -- Analyst

Got it. That's helpful. And then secondly, you mentioned that beyond Stanford you're -- potentially can open other treatment sites for the EB patients. And just wondering, what are the potential logistics of opening a site and how long you need to train their employee just starting treating patients?

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

Sure. These are sites we've been working with already for several months now. So this is a long lead time in terms of just getting all the travel logistics set up. But there are also sites that are very familiar with care of patients with EB and also have very good standard, well qualified plastic surgery staff. So they would be equipped in terms of all the moving parts. They'll be fully equipped to participate in the trial and of course, the team at Sanford has offered -- has had conversation with some of these sites and would offer to help train them on these more [Indecipherable] protocol itself. But in terms of the capabilities of caring for the EB population and in terms of the actual procedure of applying these products to be 101, these would be fully qualified sites.

Fang-Ke Huang -- SunTrust -- Analyst

Okay. And then thirdly, I think just in terms of the timing of data you're going to report for -- in ABO-101, ABO-102 your second half, are you going to present them at medical conference or scientific conference where you're going to press release or going to host the R&D day to present the data?

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

All these are possibilities. We haven't disclosed that. As soon as some of the presentations that were submitted and get approved and we'll confirm the presentation day and then we may -- if there are updates that emerge outside of a conference scheduled, then we may either call a call or a press release it.

Fang-Ke Huang -- SunTrust -- Analyst

Perfect. And last question, if I may. So you mentioned about AIM platform and then there's a number of capsids potentially can be used and you also mentioned that in the last earnings call, are -- you may discuss the potential to leverage the value of the AIM platforms through external collaborations. Maybe you can give us some updates there what you see. Is there any discussions going on currently in terms of -- for external collaborations and how are you thinking about AIM platform and how you're going to capture that value?

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

Yes. So, that's a good question and one that we didn't, in fact, announce that we would seek partnerships. As you know we have a very broad pipeline, both clinical pipeline and pre-clinical pipeline. And so it's a good problem to have, but we have obviously been careful that prioritizing our efforts under the products. So while also trying to continue to develop the AIM platform, we've been in discussions actually for months now with several interested parties and these have been fruitful. We will announce in due course when we have anything that is concrete in terms of something to disclose. But there's obviously interest in this platform, the interest in the data that Tim and his team have generated and presented as you heard earlier. And we're looking forward to continue this process. Obviously, it takes time, but we're -- we've been active at it for quite a while now. So, of course, some of these relationships are now maturing and we hope to have something to report in coming months.

Fang-Ke Huang -- SunTrust -- Analyst

Got it. Thanks so much and thanks again for taking our questions.

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

Sure. Thank you.

Operator

Our next question comes from Liav Abraham of Citi. Please state your question.

Liav Abraham -- Citi -- Analyst

Good morning. Many of my questions have been asked, maybe just one on EB-101. Joao, could you just remind us how quickly you'll be able to enroll the trial and when we could see data on the primary endpoint?

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

Thanks, Liav. So, we have and Stanford through a protocol they've had under way for a while now have been identified and screened -- formally screened I think 10 patients now or 11 patients. We expect to have enrolled somewhere between 10 patients and 15 patients to have the number of wounds that give the power for the study. As soon as we are ready to go, obviously, we'll start carefully in the first patient or two to make sure everything goes well in terms of the logistics of it. After that, we could accelerate enrollment. So it's just a matter of scheduling patients for treatment. They're all waiting for the study. So, could envision enrollment, of course, we'll provide updates on that, but could envision enrollment, say, six months, up to six months. We'll try to do faster, but it could be a little longer, depends on ability to schedule. And then the trial itself has a six month follow up built in. So, we've primary endpoint of three months and we call for an additional three months to look both at ongoing safety and healing durability so that could bring us to the end of next year, depending how quickly we can get this up and running.

Liav Abraham -- Citi -- Analyst

Great. Thank you. And then on your -- on that meeting with FDA in the second half of the year, can you just remind us what are the objectives of this meeting and do you anticipate communicating the outcome of the meeting to investors?

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

Yes. So you're referring now to MPS IIIA, right, ABO-102?

Liav Abraham -- Citi -- Analyst

Yes.

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

Okay. Yes. So we have now made a two-plus years on safety for half of the patients, at least. Others are getting close to it. Obviously cohort 3 is a bit more recent. The biomarkers have been very consistently showing that this product can improve the disease -- the underlying disease pathology, that is the activity of the enzyme and clear the accumulation of heparan sulfate. And now we're having more data now, 12 to 18 months data in cognition for the younger children.

So the question now is what next, right? So that's exactly what we're going to ask the agency. So what's the path between now and sort of moving this toward a path that can lead to BLA and approval. So this is broadly speaking that the overarching question here. And this, of course, will cover multiple aspects of the development program, including CMC, of course, clinical safety and whatnot.

So that's what we're looking to, to engage the agency with. We'll provide updates as they materialize. Obviously, regulatory feedback is not something that we would provide in details because obviously there's a full context and one so it's not often not sort of ready for progress. But we do -- we will provide guidance to the extent that the guidance is material and something that is actionable and whatnot.

Liav Abraham -- Citi -- Analyst

Thank you.

Operator

Any further questions, Ms. Liav Abraham?

Liav Abraham -- Citi -- Analyst

No, I'm good. Thank you.

Operator

Thank you.

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

Thanks, Liav.

Operator

[Operator Closing Remarks]

Duration: 49 minutes

Call participants:

Sofia Warner -- Senior Director, Investor Relations

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

Timothy J. Miller -- President & Chief Scientific Officer

Christine Silverstein -- Chief Financial Officer

Maury Raycroft -- Jefferies -- Analyst

Bikramjot Singh -- RBC Capital -- Analyst

Fang-Ke Huang -- SunTrust -- Analyst

Liav Abraham -- Citi -- Analyst

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