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Edited Transcript of ABEO earnings conference call or presentation 12-Aug-19 2:00pm GMT

Q2 2019 Abeona Therapeutics Inc Earnings Call (German)

Dallas Aug 15, 2019 (Thomson StreetEvents) -- Edited Transcript of Abeona Therapeutics Inc earnings conference call or presentation Monday, August 12, 2019 at 2:00:00pm GMT

TEXT version of Transcript

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Corporate Participants

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* Christine Berni-Silverstein

Abeona Therapeutics Inc. - CFO

* João Siffert

Abeona Therapeutics Inc. - CEO, Head of Research & Development, Chief Medical Officer & Director

* Sofia Warner

Abeona Therapeutics Inc. - Senior Director of IR

* Timothy J. Miller

Abeona Therapeutics Inc. - President & Chief Scientific Officer

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Conference Call Participants

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* Bikramjot Singh

RBC Capital Markets, LLC, Research Division - Senior Associate

* Fang-Ke Huang

SunTrust Robinson Humphrey, Inc., Research Division - Associate

* Liav Abraham

Citigroup Inc, Research Division - Director

* Maurice Thomas Raycroft

Jefferies LLC, Research Division - Equity Analyst

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Presentation

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Operator [1]

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

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Sofia Warner, Abeona Therapeutics Inc. - Senior Director of IR [2]

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Thank you. Good morning, and welcome, everyone. Today's call will be led by João Siffert, our Chief Executive Officer. Following João, Tim Miller, our President and Chief Scientific Officer, will present preclinical highlight; 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 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 to you, Dr. João Siffert. João, you have the floor.

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João Siffert, Abeona Therapeutics Inc. - CEO, Head of Research & Development, Chief Medical Officer & Director [3]

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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 results 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 that 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 systemic 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, an autologous gene corrected cell therapy that restores normal functional Collagen VII to keratinocytes and their progenitors. Each EB-101 gene corrected keratinocytes sheet, which can cover an area of approximately 35 to 40 centimeters square, is transplanted into open wounds, provided -- providing prompt wound healing. Two skin biopsies allow manufacturer of 6 EB-101 sheets, which combined, can cover wound areas measuring up to 240 centimeters square. To our knowledge, EB-101 is the only product in development for RDEB with clinical data, show it could heal large chronic wounds that had been open for years.

The Phase I/IIa trial, conducted by Stanford University, show that EB-101 healed an average of approximately 130 centimeters square per patient and up 260 centimeters square in some patient, with durability of 2 to 5-plus years post treatment. The majority of RDEB wounds are chronic, often remain open for years and tend to be larger than 120 centimeters square. Wound healing of such large wounds is in itself clinically significant and the long-term follow-up from the Phase I/IIa trial show that 50% or more wound healing would associate 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 the preparation for upcoming Phase III clinical trial called VITAL, aimed at confirming the safety and efficacy of the EB-101 observed in the Phase I/IIa study. At this meeting, the FDA addressed information that we previously submitted regarding quality testing of the product in certain aspects of our Phase III clinical trial protocol. We believe we had successfully completed the manufacturing process of 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 [IIB] 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 we'll provide clarification on 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 III trial in fourth quarter 2019.

The VITAL Phase III 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 3 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 square.

In most cases, their chronic wounds exceed 120 meters square, require frequent wound care and are associated with disabling pain and are at 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 and at clinic visits. Patients will be followed for up to 6 months for assessment of wound healing durability and overall safety.

We estimate there are about 2,500 RDEB patients in the U.S. 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 I/IIa trial have equipped our clinical team to best prepare for Phase III. The Stanford University team is also ready to start enrolling patients as soon as we receive the FDA endorsement to proceed. Additional study sites will begin in the ensuing months.

At next slide, to provide an update for 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, dose one time intravenously using a self-complementary AAV9 vector to deliver 2 functional copies of the SGSH gene to cells on the CNS as well as the peripheral organs. The therapy is designed to correct the underlying SGSH agents on deficiency and prevent cellular accumulation of heparan sulfate that leads to cell dysfunctions, cell death and rapid neurodevelopmental decline and physical impairment.

We're currently assessing the safety and efficacy of ABO-102 in the Transfer A study, also known as ABT-001. This study is a 2-year, open label, dose escalation Phase I/II 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 focus on neurodevelopmental progress and safety with secondary measures of behavior, quality of life, heparan sulfate levels and CSF plasma in urine, in brain and liver volume.

Last month, we were excited to share positive interim data for our Transfer A study, showing that the 3 youngest patients enrolled 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 posttreatment.

To our knowledge, our data are the only reported evidence suggesting that a gene therapy treatment could alter the typical neurodevelopmental 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 the CSF heparan sulfate was dose dependent and reach their lower limit of method detection for 8 patients enrolled in Cohort 3. Reductions in liver volume were also sustained during the observation period of up to 2 years. In addition, safety profile of ABO-102 remains favorable and no product related serious adverse events were reported to date.

We continue to screen patients for enrollment in the Transfer A study at sites in the U.S., Spain and Australia. We're pursuing an RMAT 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 onetime 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 study, also known as ABT-002, is a 2-year, open label Phase I/II 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 sulfate levels, CSF and serum NAGLU enzyme activity and liver and brain volume by MRI. We're pleased to share that to date, we have treated a total of 5 patients with MPS IIIB, 2 in Cohort 1 and 3 in Cohort 2. Study recruitment efforts continue and we have the 2 [acute] patients awaiting screening across sites in the U.S., Spain and France.

With a posttreatment follow-up ranging from less than 1 month to more than 20 months post-dosing, the overall safety profile remains favorable, and there had been no serious adverse events deemed 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 had 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 and neurological impairments and early death.

In May, we announced the FDA clearance of our R&D for ABO-202, an AAV9 gene therapy for CLN1 disease. And in June, we received the FDA Fast Track Designation for this program. These regulatory milestones, in combination with our previously reported preclinical data, which showed a favorable safety profile and no 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 the company standpoint, this quarter we strengthened our management team with 2 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 we bring our gene and cell therapies to patients. Jodie brings over 20 years of valuable industry experience and makes her an ideal person to lead our patient advocacy and clinical affairs functions in close collaboration with patients, families and 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 heartily thank all the patient, 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?

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Timothy J. Miller, Abeona Therapeutics Inc. - President & Chief Scientific Officer [4]

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Thank you, João. During the second quarter, we reported new preclinical data developed from our AIM capsid platform, a next generation of adeno-associated virus capsids for use in gene therapies. The AIM capsid library can utilize AB biology and 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 for Gene and Cell Therapy 22nd Annual Meeting in Washington, D.C. ABO-401 has a regulatable, human mini-CFTR gene that is sufficiently 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. And this and other preclinical studies, ABO-401 restores 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 lung 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 the CF mice and that the ABO-401 gene expression cassette 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 onetime gene therapy.

Also during the quarter, Abeona presented a new preclinical data on our novel 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 nonhuman primate led to robust transgene expression in the inner and outer retina. Expression was observed in the peripheral retina and phobia, 25 days post-administration. AAV204 also transfused 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 nonhuman primate data were complemented by findings from mice models, which identified AAV204 as one of the 3 lead candidates AIM capsids that demonstrate 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 nonhuman 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, can potentially be performed in an outpatient setting and be a safer and less invasive approach than subretinal administration. AIM vectors are non-replicating and have shown the potential to evade immune responses generated by exposure to naturally occurring AAV vectors. Our library contains more than 100 capsids that tissue tropisms selected for their 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 nonhuman primates that we can utilize different AIM capsids to target the photoreceptors in retinal pigmented epithelium in either subretinal or intravitreal injections.

The AIM capsid increase our potential for targeting multiple eye disorders for delivery of therapeutic gene or employing the machinery to enable gene editing.

Lastly, we have now demonstrated in vitro that select AIM capsids 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 to 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 of 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?

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Christine Berni-Silverstein, Abeona Therapeutics Inc. - CFO [5]

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Thank you, Tim. We have recently filed the 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 30, 2019 were $62.5 million compared to $68.3 million as of March 31, 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 3 months ended June 30, 2019, was $16.3 million compared to $7.9 million in the same period of 2018. The increase in research and development expense is primarily attributed to increase in 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 in 2018. That's the summary of financials.

With respect to upcoming investor and scientific conferences, I'd like to highlight that on September 4, we'll be in Boston, attending the Citi's 14th Annual Biotech Conference. And on September 5, we will also be attending the Wells Fargo Securities Health Care 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 João.

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João Siffert, Abeona Therapeutics Inc. - CEO, Head of Research & Development, Chief Medical Officer & Director [6]

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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 but turning hope into reality for our patients. Thank you. I'll now turn over to the operator to open up for questions. Operator?

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Questions and Answers

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Operator [1]

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(Operator Instructions) We will take our first question from Maurice Raycroft of Jefferies.

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

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First question is on ABO-101. It seems like you expanded the 5E13 cohort from 3 to 6, to 4 to 7 in July, added an efficacy based primary endpoint looking at neurocog and then added some secondary endpoint 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 encourage the changes? And then just bigger picture, what else is needed in IIIB to eventually seek approval for this indication?

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João Siffert, Abeona Therapeutics Inc. - CEO, Head of Research & Development, Chief Medical Officer & Director [3]

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Well [just to begin], Maurice, João here. I'll take this question. So you're referring to the MPS IIIB programs. Is that correctly understood?

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

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That's correct.

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João Siffert, Abeona Therapeutics Inc. - CEO, Head of Research & Development, Chief Medical Officer & Director [5]

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The beginning of -- yes. So these are -- it's actually 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 had not undergone a 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 will likely be larger in children who are still functioning at very higher level, and that's codified by setting up the [clinical] criteria with the 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 in that we continue to collect both biomarkers and their developmental data from this program. And once we have sufficient data to identify a clinical benefit beyond this biological benefit of markers, which is we expect to see enhancing in the first patient, expect to see in the ensuing patients. We'll again engage the agency in discussions for the further development of the program.

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

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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.

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João Siffert, Abeona Therapeutics Inc. - CEO, Head of Research & Development, Chief Medical Officer & Director [7]

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Yes. So these patients meet the eligibility criteria. So we're not disclosing 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 1 in 2019. So it's relatively early follow-up. But they meet criteria for the developmental quotient. That is, in general, looking at the -- just as the framework and not necessarily citing 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 a rapid progressive genotype tend to be usually younger than 40 months or so, give or take, and they tend to be younger.

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Maurice Thomas Raycroft, Jefferies LLC, Research Division - Equity Analyst [8]

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

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João Siffert, Abeona Therapeutics Inc. - CEO, Head of Research & Development, Chief Medical Officer & Director [9]

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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 we continue to screen patients as we speak. 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 conservative 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, may not be as uncommon as we thought to be in some European countries and other places such as South America. So we are 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 and now up to 2 years in CSF heparan sulfate, as well as the recent data we announced in the youngest patients showing some neurocognitive preservation. I think that also creates a momentum in terms of interest and 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.

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Maurice Thomas Raycroft, Jefferies LLC, Research Division - Equity Analyst [10]

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

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João Siffert, Abeona Therapeutics Inc. - CEO, Head of Research & Development, Chief Medical Officer & Director [11]

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Yes. So the patients by protocol eligibility, all patients need to have mutations that are associated with the rapid progressor phenotype. So in general, if you look at the various natural history studies, including the one we work most closely with, the Nationwide Children's Hospital. But you can look at the Pittsburgh data and also data from University of Minnesota, albeit with different scales, but certainly something that we should look at because it's a pretty comprehensive data sets as well. Most children with the rapid progressive form of MPS IIIA or IIIB for that matter, tend to plateau in their neurodevelopment before they reach age of 3, almost by a [legacy] this is biology, not sort of mathematics. But by and large, and if you look at these studies, most of these children are decelerating their development during their second year of life. And before they reach age of 3, 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 to be already an indication that this treatment is having a biological effect and also diverging in terms of the neurocognitive course. Does that answer your question?

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Maurice Thomas Raycroft, Jefferies LLC, Research Division - Equity Analyst [12]

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Yes. That does. And just last question on EB-101. So for the CMC protocol item, particularly the 1 related to a 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 I'm wondering how much Brammer's involved with helping with the protocol design and finalizing the protocol for this?

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João Siffert, Abeona Therapeutics Inc. - CEO, Head of Research & Development, Chief Medical Officer & Director [13]

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So, 2 separate questions. So what we're submitting to the agency their request is the actual protocol. We're not submitting any data going ahead of the Phase III. 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 III. So we will be conducting the comparability studies, which are no different than the release that, generally speaking, no different than the release of studies that we do quality assurance we do for any kind of GMP products. So in this case, for EB-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're doing this is to set up a protocol prospectively to show what the acceptance criteria are, which are generally and already agreed upon with the agencies. So it's literally having the protocol written. Data will be generated as we release these batches for clinical use as we would normally. And now we have used the Brammer material to manufacture sheets all throughout the past say, 6 months. These are obviously, not for clinical use, but certainly using the same retrovirus that we are intending to use in the Phase III. So we have experienced using the retrovirus and has performed well as expected. So we don't anticipate any surprises here.

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Maurice Thomas Raycroft, Jefferies LLC, Research Division - Equity Analyst [14]

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

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João Siffert, Abeona Therapeutics Inc. - CEO, Head of Research & Development, Chief Medical Officer & Director [15]

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They have been very good partner of Abeona for a while now, and they're very closely -- we 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 EB program, has included manufacturing EB-101 using the retro -- the Brammer retrovirus. So we've done this before. This is not the first time we're going to do this. So it is going to be officially for -- during the Phase III.

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Operator [16]

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Our next question comes from Kennen MacKay of RBC Capital.

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Bikramjot Singh, RBC Capital Markets, LLC, Research Division - Senior Associate [17]

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This is Bikramjot for Kennen. So we had one on the patient related outcomes that actually had -- have to be included in the Phase III study for EB-101. Could you please elaborate what those outcomes are? And how would those further validate maybe the wound healing that you will show in the patients?

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João Siffert, Abeona Therapeutics Inc. - CEO, Head of Research & Development, Chief Medical Officer & Director [18]

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The patient reported outcomes, there are several. The main one being pain, especially for the larger wounds that we are addressing in our Phase III trial, pain is among the most disabling patient experiences. Obviously, they're obviously having an open wound, has all sorts of other medical implications including risk of infection and need for repeat wound dressing and then extensive wound care and whatnot. But the pain is really the main cause of discomfort and suffering for these patients. So the FDA is obviously interested in understanding how wound healing and pain intersect. If you ask a patient, they will tell you that their intact skin that is the healed wound doesn't hurt. And if you ask them, which wounds hurt the most, they probably will tell you are the larger wounds, small wounds are not, but they can still be uncomfortable, but the larger wounds tend to be the ones mostly associated with the higher intensity of pain. This has been shown not only anecdotally by asking the patients, but also in natural history studies that had been presented. So FDA wanted to just agree with us and [exactly] timing and how to collect those using scales that are pretty well known and it's been validated in pain trials before. So without much about the scales themselves, it's just about the collection timing in relation to some of the wound care. Basically wound care, as we remove the bandages they can cause pain, because sometimes the bandages, especially in the larger wounds gets stuck. Also as you tender to the wounds and cleanse the wounds can cause also acute pain. So the patients have both their chronic pain from just having these open wounds chronically, but then acute exacerbations of pain during procedures. So it's just a matter of getting coordinated and making sure we get as much information as possible in those both -- a lot of patients that are at home but also recalling that the experience when they come to the clinic visits.

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Bikramjot Singh, RBC Capital Markets, LLC, Research Division - Senior Associate [19]

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

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João Siffert, Abeona Therapeutics Inc. - CEO, Head of Research & Development, Chief Medical Officer & Director [20]

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Sorry. If you could repeat the first part of your question, I just couldn't hear well.

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Bikramjot Singh, RBC Capital Markets, LLC, Research Division - Senior Associate [21]

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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 or will be it something similar to [perhaps] 50% wound healing? How should we be thinking about that?

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João Siffert, Abeona Therapeutics Inc. - CEO, Head of Research & Development, Chief Medical Officer & Director [22]

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Yes. So this is a difficult question about EB because there aren't much data for EB specifically. There's obviously a ton of data in pain, both chronic and acute pain trials in a variety of conditions, from cancer to neuropathic pain, even in migraine. But there is -- as you can imagine, there is not a lot for EB. The sense we get from talking to patients as the wound healing will essentially largely release the pain, but the pain also can be quite variable. And if it's undisturbed, 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 dressing, and then the pain spikes up. So there isn't a set threshold to success. Ultimately, in the end, the intent 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 in all the ramifications of EB wounds. But we would expect that it will also provide us different pain relief. So we'll measure it as a continuous variable as well as subcategorical variable.

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Operator [23]

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Our next question comes from Joon Lee of SunTrust.

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Fang-Ke Huang, SunTrust Robinson Humphrey, Inc., Research Division - Associate [24]

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This is Fang-Ke on for Joon. Just wondering if you can tell us a bit more about the timing of initiating the EB-101 trial? It's going to be early (inaudible) event where you can anticipate it's going to be a (inaudible) event or it is difficult to tell at this moment?

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João Siffert, Abeona Therapeutics Inc. - CEO, Head of Research & Development, Chief Medical Officer & Director [25]

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So we can't provide any more guidance than we already have provided publicly and K filing. Much of what remains are questions about quality, and this particular question about PROs that we're discussing. The timing now for initiation is entirely predicated on resolving these questions. So it is largely driven by the timing of how efficiency we can communicate with the agency. On our end, we're working diligently to get all the answers submitted soon. So just not our ensuring that FDA is satisfied with the answers, and that if there 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 are ready as soon as the FDA is ready. Stanford is, obviously, been a very close partner and we're doing it 3 years now, and they are ready and willing and eager to start, so as long as we get the final go-ahead.

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Fang-Ke Huang, SunTrust Robinson Humphrey, Inc., Research Division - Associate [26]

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

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João Siffert, Abeona Therapeutics Inc. - CEO, Head of Research & Development, Chief Medical Officer & Director [27]

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

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Fang-Ke Huang, SunTrust Robinson Humphrey, Inc., Research Division - Associate [28]

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

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João Siffert, Abeona Therapeutics Inc. - CEO, Head of Research & Development, Chief Medical Officer & Director [29]

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

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Fang-Ke Huang, SunTrust Robinson Humphrey, Inc., Research Division - Associate [30]

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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 also you mentioned that on the last earnings call, you are -- you may discuss the potential to leverage the value of AIM platform through external collaborations. Maybe you can give us some updates there that you see -- is there any discussions going on currently in terms for external collaborations and how you are thinking about AIM (inaudible) and how you can capture that value?

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João Siffert, Abeona Therapeutics Inc. - CEO, Head of Research & Development, Chief Medical Officer & Director [31]

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Yes. So that's a good question, and one that we didn't, in fact, announced that we would seek partnerships. As you know, we have a very broad pipeline, both clinical pipeline and preclinical pipeline. And so it's a good problem to have. But we have, obviously, been careful of prioritizing our efforts on their lead products. So while also trying to continue to develop the AIM platform, we have been in discussions actually for months now with several interested parties. And leads have been fruitful. We'll 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, interest in the data that Tim and his team have generated and presented, as you heard earlier. And then we're looking forward to continuing this process. Obviously, it takes time, but 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 the coming months.

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Operator [32]

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Our next question comes from Liav Abraham from Citi.

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Liav Abraham, Citigroup Inc, Research Division - Director [33]

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Many of my questions have been asked. Maybe just 1 on EB-101. João, can you just remind us how quickly you'll be able to enroll the trial? And when we could see data on the primary endpoint?

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João Siffert, Abeona Therapeutics Inc. - CEO, Head of Research & Development, Chief Medical Officer & Director [34]

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So we have Stanford protocol they've had underway for a while now, have identified and screened, formerly screened, I think, 10 patients, now were 11 patients. We expect to have to enroll somewhere between 10 and 15 patients to have the number of wounds that give the color for the study. As soon as we are ready to go, obviously, we'll start carefully in the first patient or 2 to make sure everything goes well in terms of the logistics. But after that, we could accelerate enrollment. So it's just a matter of scheduling patients for treatment. They are all waiting for the study. So we could envision enrollment, first we'll provide updates and that could envision enrollment, say, 6 months, up to 6 months and we try to do faster, but it could be a little longer depends on the ability to schedule. And then the trial itself has a 6-month follow-up built-in. So the primary endpoint of 3 months, and we'll call for an additional 3 months to look both at ongoing safety and healing durability. So that could bring us to the end of next year, depending on how quickly you can get to something running.

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Liav Abraham, Citigroup Inc, Research Division - Director [35]

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Great. And then on your RMAT 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?

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João Siffert, Abeona Therapeutics Inc. - CEO, Head of Research & Development, Chief Medical Officer & Director [36]

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

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Liav Abraham, Citigroup Inc, Research Division - Director [37]

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Yes. Yes. Yes.

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João Siffert, Abeona Therapeutics Inc. - CEO, Head of Research & Development, Chief Medical Officer & Director [38]

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Okay. Yes. So we have now data of 2 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 had been very consistent. We are 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 10, 12 to 18 months of data in cognition for the younger children. So the question now is what's 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 a BLA and approval. So this is, broadly speaking, the overarching question here. 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 engage the agency with. We'll provide updates as they materialize. Obviously, regulatory feedback is not something we'll provide in details because, obviously, there's a whole context and one that's not often not sort of ready for press, but we will provide guidance to the extent that the guidance is material and is something that is actionable or not.

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Operator [39]

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Ladies and gentlemen, this will conclude today's question-and-answer session. And this does conclude today's teleconference. We thank you for your participation. You may disconnect your lines at this time, and have a great day.

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João Siffert, Abeona Therapeutics Inc. - CEO, Head of Research & Development, Chief Medical Officer & Director [40]

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Thank you.