Q2 2019 Earnings Call

Yeah.

Please standby.

Operator: Please stand by. 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.

Good morning, ladies and gentlemen, and welcome to the baby on F. Therapeutics, Inc. second quarter 2019 earnings and business update conference call todays 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 will turn the call over to Sofia Warner's Senior director Investor Relations. Thank you you may begin.

Operator: A brief question and answer session will follow the formal presentation. For opening remarks and introductions, I'll turn the call over to Sophia Warner, Senior Director, Investor Relations. Thank you. You may begin.

Thank you.

Sophia Warner: 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 uncertainty. Forward-looking statements on this call are made pursuant to the safe harbor provisions of the federal securities law. 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.

Good morning, and welcome everyone todays call will be led by allows Cooper, our chief Executive Officer.

Following to allow.

Tim Miller, our President and Chief Scientific Officer will present preclinical highlight.

And Christine Silverstein, our Chief Financial Officer, who will review our financials.

Before I turn the call over to Graham.

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 are subject to change and actual results may differ materially from forward looking statement.

Some of the factors that could cause actual results to differ may be found in the company's annual report on Form 10-K .

I'm quarterly report on Form 10-Q .

Filed by the company with the Securities and Exchange Commission.

These documents are available on our website www dot maybe only therapeutics dotcom.

Sophia Warner: These documents are available on our website, www.abeonatherapeutics.com. With that said, it is now my pleasure to introduce Dr. Joelle Sipper. Joelle, you have the floor.

With that said it is now my pleasure to introduce your doctors allow quicker. So while you have the floor.

Joao Siffert: Thank you, Sophia, and thank you all for joining us today for our second quarter results and business highlights, which we believe reflect 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 on our Next Generation AIM capsid platform.

Thank you Sophia and thank you all for joining us today for 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 their manufacturing and quality operation.

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 a next generation Encapso platform.

I'd like to start off with is with an update on our recessive dystrophic Epidermolysis Bullosa program.

Joao Siffert: I'd like to start off with an update on our recessive dystrophic epidermolysis below the program. As a reminder, RDEB is a rare and severely debilitating genetic skin disorder caused by mutations which result in a lack of functional collagen VII, the main component of the anchoring fibrils that attach the dermis to the epidermis. Without functional collagen VII, RDAB 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. RDAB 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.

As a reminder, our Def is a rare severely debilitating genetic skin disorder caused by mutations which result in lack of functional college and seven the main component of the anchoring fibrils.

That attach the dermis to the epidermis.

Without functional college and seven are that patients have extremely proud of skin blisters and pairs.

Ultimately leading to multiple wounds some of which remain open for years and cover a significant amount of the body or their boards are very painful the to multiple dermatological and systemic complications and currently have no effective treatment options.

Patients rely on palliative treatment 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 you'd be willing to one an autologous gene corrected cell therapy, the restores normal functional college, and seven to keratinocytes and their projectors.

Joao Siffert: We're currently developing EB-101, an autologous gene-corrected cell therapy that restores normal functional collagen-7 to keratinocytes and their progenitors. Each EB-101 gene-corrected keratinocyte sheet, which can cover an area of approximately 35 to 40 centimeters squared, is transplanted into open wounds, providing prompt wound healing. Two skin biopsies allow the manufacture of six EB-101 sheets, which together can cover wound areas measuring up to 240 centimeters squared.

Each if you Wanna, one gene corrective keratinocyte sheet, which can cover an area of approximately 35 to 40 centimeter squared.

His transplanted into open wounds provided providing prompt wound healing.

Two skin biopsies allow manufacturer of six if you Wanna, one sheets, which combined can cover one area is measuring up to 240 centimeter square.

Tore knowledge, if you want to one is the only product can develop and for our dead with clinical data. So it could heal large chronic wounds that have been opened for years.

Joao Siffert: To our knowledge, EB-101 is the only product in development for RDAB with clinical data, so it could heal large chronic wounds that have been open for years. The Phase 1-2A trial conducted by Stanford University showed that EB-101 healed an average of approximately 130 cm2 per patient and up to 160 cm2 in some patients, with durability of 2 to 5 plus years post-treatment. The majority of RDAB wounds are chronic, often remain open for years, and tend to be larger than 120 cm2.

The phase one to a trial conducted by Stanford University showed that he'd be want to warn healed at an average of approximately 130 centimeters square per patient.

And up 260 centimeter square in some patients.

The durability of two to five plus years post treatment.

The majority of order boards are chronic often remain open for years and tend to be larger than the 120 centimeters square wound healing of such large wounds is in itself clinically significant and the long term follow up from the phase one to a trial show that 50% or more wound healing was associated with meaningful reduction in pain.

Joao Siffert: Wound healing of such large wounds is, in itself, clinically significant, and the long-term follow-up from the Phase I-IIa trial showed 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 the preparation for our upcoming Phase III clinical trial called VITAL, aimed at confirming the safety and efficacy of BEB101 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 have successfully completed the manufacturing process of B101, and the requests for clarification on chemistry, manufacturing, and controls are focused on product transport and on the protocol to assess the comparability of the 2B commercial retrovirus. In addition, FDA provided feedback on the selection, measurement, and timing of certain patient-reported outcome measures.

Our goal is to bring this transformative therapy to patients with our Deb as soon as possible.

We recently met with the FDA to discuss the final stages of preparation for upcoming phase three clinical trial called vital aim the confirming the safety and efficacy of B. If you want a one observed in the phase one to a study.

At this meeting the FDA address the information that we previously submitted regarding quality testing of the product in certain aspects of our phase three clinical trial protocol.

We believe we have successfully completed the manufacturing process of VB one a one in the request for clarification on chemistry manufacturing and controls are focused on public transport and on the protocol to assess comparability of the to be commercial retrovirus used in it do you want to one manufacturing.

In addition, after they provide a feedback on selection measurement and timing of certain patient reported outcome measures.

Joao Siffert: 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 three trial in the 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 RDAP 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 wounds on the same patient. 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 cm2, require frequent wound care, and are associated with disabling pain and are at great risk for infection.

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 ft reviewers to address their questions. So we can proceed with the phase three trial in fourth quarter 2019.

The vital phase three study will be a multi center randomized clinical trial assessing he'd be a one to one for treatment of approximately 35, one sites across 10 to 15 or their patients.

The primary outcome measure of the study will be wound healing at three months comparing the proportion of treated won't sites with at least 50% healing two and treated once on the thing patients by protocol participants eligible to enroll them vital we'll have chronic wounds larger than 40 centimeters squared.

In most cases their chronic wounds exceed 120 centimeters square require frequent wound care and are associated with the sable in pain in or a great risk for infection.

Additional study endpoints will include the patient global impression of change for pain at each wounds site comparable baseline as well as measurements of pain intensity during dressing changes in a clinic visits.

Joao Siffert: Additional study endpoints will include the patient's global impression of change for pain at each wound site, comparable baseline, as well as measurements of pain intensity during dressing changes during 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 RDAB 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 RDAP 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 enrolling patients as soon as we receive FDA endorsement to proceed. Additional study sites will begin in the ensuing months.

Patients will be followed for up to six months for assessment of wound healing durability and overall safety.

We estimate there are about 2500 or that patients in the U.S., who could benefit from multiple either you want a one treatment given the large number and size of wounds observed among the population.

The order pace and community and their treating physicians are anxious to have either you want a one available as soon as possible.

Learnings from the phase one to a trial have equipped our clinical team to best prepare for phase three the Stanford University team is also ready to start enrolling patients as soon as we receive that key endorsement to proceed additional study sites will begin in the ensuing months.

I'd now like to provide an update for a program assessing a beautiful one or two for MPS. Three also known as sanfilippo syndromes type a.

Joao Siffert: I'd next like to provide an update for a program assessing ABL-102 for MPS-3A, also known as Sanfilippo Syndrome Type A, a rare lysomal storage disease with no approved treatment that primarily affects the central nervous system, or CNS. ABO102 is our novel gene therapy, which is delivered one time intravenously using a self-complementary AAV9 vector to deliver two functional copies of the SGS The therapy is designed to correct the underlying SGS-SAGE enzyme deficiency and prevent cellular accumulation of heparin sulfate that leads to cell dysfunction, cell death, and rapid neurodevelopmental decline and physical impairment. We're currently assessing the safety and efficacy of AB0102 in the Transfer A study, also known as ABT001. This study is a two-year, open-label, dose-escalation, phase 1-2 global clinical trial for patients with MTS3A who have a developmental quotient of at least 60% of normal levels for age and meet other eligibility criteria.

A rare less almost storage disease with no approved treatment that primarily affects the central nervous system or CNS.

If youre one or two is our novel gene therapy.

Those one time intravenously, using a self complementary a benign vector to deliver two functional copies of the S.G.S.H.D. gene to sell them on the CNS.

As well as the peripheral organs.

The therapy is designed to correct the underlying S.G.S. agents iron deficiency, and prevent seller accumulation of heparan sulfate that leads to cell dysfunction cell death.

And rapid neuro developmental decline in physical impairment.

We're currently assessing the safety and efficacy of baby or one or two in the transfer a study also known as ABT or one.

This study is a two year open label dose escalation phase one two global clinical trial for patients with MPS, three a., who have a developmental quotient of at least 60% of normal levels for age and meet other eligibility criteria.

Joao Siffert: The study primary outcome measure focuses on neurodevelopmental progress and safety, with secondary measures of behavior, quality of life, heparin sulfate levels in CSF, plasma in urine, and brain. Last month, we were excited to share positive interim data for a Transfer A study showing that the three youngest patients enrolled in the study at ages 26, 19, and 12 and a half months at dosing, all enrolled in Cohort 3, continue to To our knowledge, our data are the only reported evidence suggesting that gene therapy treatment could alter the typical neurodevelopmental course in children with MTS3A. 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 ADO-102 administration resulted in a durable reduction in CSF heparin sulfate, a key biomarker of MPS3A. Improvement in CSF heparin sulfate was dose-dependent and reached its lower limit of method detection for eight patients enrolled in Cohort 3.

The study primary outcome measure focus on nerve developmental.

Progress in safety with secondary measures of behavioral 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 transfer a study showing that the three youngest patients enrolled in the study at age is 20, 619, and 12 and a half months the dosing all enrolled in cohort three continue to track within normal range of the age equivalent development at age is 40 431 in 24.5 months respectively.

That is 12 to 18 months post treatment.

Through our knowledge our data are the only reported evidence, suggesting that a gene therapy treatment could alter the typical nerd developmental course in children with MPS three.

We believe our data also corroborate 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 showed that intravenous radio one or two administration resulted in a durable reduction and CSF heparan sulfate a key biomarker of M. P. S. Three.

The improvement in CSF, Heparan sulfate was dose dependent and reach their lower limit of method detection for eight patients enrolled in cohort three.

Joao Siffert: Reductions in liver volume were also sustained during the observation period of up to 2 years. In addition, the safety profile of AB0102 remains favorable, and no product-related serious adverse events have been reported to date. We continue to screen patients for enrollment in the transfer aid study at sites in the U.S., Spain, and Australia. We're pursuing an RMAP 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 MTS-3B, also known as Sanfilippo Syndrome Type B. MTS-3B is a rare, devastating, and fatal isosomal storage disease with no approved treatment that is characterized primarily by rapid neurodevelopmental decline leading to early death.

Reductions in liver Vod and were also sustained during the observation period of up to two years.

In addition.

The safety profile of video window to remain favorable and no product related serious adverse events were reported to date.

We continue to screen patients for enrollment in the transfer and study at sites in the U.S., Spain and Australia.

We're pursuing on our map meeting with the FDA in the second half of this year to discuss study data in getting clarity on the development path forward.

Moving on I'd like to discuss our 80 or 101 program, an investigational onetime gene therapy for the treatment of M. P. S. Three be also known as sanfilippo syndromes type B.

MPS three D is a rare devastating and fatal lysosomal storage disease with no approved treatment that has characterized primarily by rapid neuro developmental decline leading to early death.

Joao Siffert: We recently announced that FDA granted FASTRAC designation to AB0101, recognizing the severity and importance of addressing this rare orphan disease. To briefly review, the TRANSFER-B study, also known as ABT002, is a two-year open-label Phase I-II study primarily evaluating safety and neurodevelopment in MPS3B children treated with AB101. 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 heparid sulfate levels, CSF in serum NaGlu enzyme activity in the liver, and brain volume by MRI.

We recently announced that up gay granted fast track designation to radio one one recognizing the severity and importance of addressing this rare orphan disease.

To briefly review the transfer beef study also known as the B T Zero zero too is that to your open label Phase One two study primarily evaluating safety under development of the M. P. S. Three d. children treated would they be 181 to one.

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 and you're in Heparan sulfate levels CSF in serum NAGLU enzyme activity in liver in brain volume by M. all right.

We're pleased to share that to date, we have treated a total of five patients with MPS three b two in cohort one and three in cohort two.

Joao Siffert: We're pleased to share that, to date, we have treated a total of five patients with MPS 3B, 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 U.S., 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 remained favorable, and there have been no serious adverse events deemed related to ABO 101.

Study recruitment efforts continue and we have the Q acute patients awaiting screening across sites and the U.S., 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 team related to a 101 improvements in disease specific biomarkers were similar to that observed for MPS three program.

Joao Siffert: Improvements in disease-specific biomarkers were similar to that observed for MPS 3A programs. 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 and neurological impairment and early death.

We expect to report interim data from the trial in the second half of this year.

We also have made progress advancing our CLM one program to the clinic selling one disease also know is known as infantile batten disease is a rare fatal inherited disorder of the nervous system. The generally presents in childhood and leads to visual neurological impairment and early death.

Joao Siffert: In May, we announced the FTA clearance of RRND for AB0202 and AAV9 gene therapy for CLN1 disease. And in June, we received FTA Fast Track designation for this program. These regulatory milestones, in combination with the previously reported preclinical data which showed a favorable safety profile and low significant toxicities, leave us excited about the potential for AB0202 and CLN1. We will provide guidance on the timing of the study later this

In May we announced the uptake clearance of our R&D for a deal to to a Navy nine gene therapy for sale in one 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 very favorable safety profile and no significant toxicities.

We are as excited about the potential for a deal to instill in one.

We will provide guidance on the timing of the study later this year.

Finally from a company standpoint, this quarter with strengthening our management team with two important appointments were fortunate to announce the recruitment of Dr. Victor Paul as a senior Vice President of regulatory Affairs, and Jody Gil and as Vice President President of patient advocacy in clinical affairs.

Joao Siffert: Finally, from a company standpoint, this quarter, we 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 Jody Gillen 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 genes and cell therapies to patients. Dr. Jodi 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, medical, and scientific stakeholders. Both Victor and Jody have already had an immediate positive impact on Abeona.

Victor brings to up your own 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 that gene and cell therapies to patients.

Jody brings over 20 years of valuable industry experience that makes are an ideal person to lead our patient advocacy and clinical affairs functions.

In close cooperation with patients and families and medical and scientific stakeholders.

Both victory and Jody have already had an immediate positive impact for beyond them.

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

Tim: 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.

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

Thank you drew out during the second quarter, we reported new preclinical data developed from our Encapso platform next generation that have no associated virus capsids for using gene therapies.

Tim: Thank you, Joelle. During the second quarter, we reported new preclinical data developed from our AIM capsid platform, the next generation of adeno-associated virus capsids for use in gene therapies. The AIM capsid library can utilize AAV 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 for Gene and Cell Therapy 22nd Annual Meeting in Washington, D.C. AVO 401 has a regulatable, human, The data presented at ASGCT demonstrate that AB0401 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.

The end caps or library can utilize 80 biology to selectively target delivery of genetic payloads for the central nervous system long, its hi, muscle never and other tissues.

In April we presented new data from the deal for one program our novel Gene therapy for suits that process at the American Society for gene and cell therapy, 22nd annual meeting in Washington D.C.

Hey deal for one has a regular annabelle human miniskirts charging it efficiently packaged into 82 or for one of our next generation Library Capsids.

The data presented the data demonstrate that Ethiopia, lorawan efficiently delivered a highly expressed functional copy acumen minis gift card to the lung Mcf mice and restored CSAPR lung function and you can see application nasal and probably do it yourself.

This adds to the growing body of evidence, suggesting for a one may address the challenges in lung delivery and transgene expression that have limited the advancement of gene therapy for patients.

Tim: This adds to the growing body of evidence suggesting ABO401 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 cells, 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 ABO-401 restored CFTR-specific nasal potential differences in the CF mice, and that the ABO-401 gene expression consent made a fully functional and processed CF

And this and other preclinical studies a deal for one lets storage CPR expression and chloride conducting airway epithelial immune cells are the long that contribute to the technology enhancements.

Robust expression of 82 important the lungs, Mcf might well deserved and demonstrated that the 82, a four cap said with equally or more efficient at delivering gene expression cassettes. The line compared to other naturally occurring 80 capsids.

Further the data demonstrated the ideal for one distorts the shipyard specific needs of potential differences, yes, mice and that the ATRIO poor one gene expression cassette makes a fully functional and processed CSAPR.

We believe that the recent data are encouraging and easier for one is a promising candidate that may ultimately change the landscape of CF treatment by introducing a onetime gene therapy.

Also during the quarter, if you Gotta presented new preclinical data of our on our novel 82, or four capsid, demonstrating the broad therapeutic potential in gene therapy in retinal diseases.

Tim: We believe that the recent data are encouraging, and ABO401 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 on our novel AIM-AV204 capsid, demonstrating the broad therapeutic potential of AIM gene therapy in retinal diseases. The data were presented in May at the Association for Research and Vision and Ophthalmology annual meeting in Vancouver. The data showed that introvertial administration of our novel AIM AAV204 capsid to non-human primates led to robust transgene expression in the inner and outer retina. Expression was observed in the peripheral retina and fovea 25 days post-administration. AB204 also transduced the photoreceptor cells in retinal explants and transduced the outer retina with positive green fluorescent protein expression.

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 80 to a forecasted non human primates led to robust transgene expression in the inner and outer retina, especially was observed in the peripheral retina and probably up 25 days post administration.

82, or four also transduce photoreceptor cells in the right know ex plants and try and use the outer retinal what positive reinforcement protein expression.

These data support the potential use of 82 or four 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 finding some mouse models, which identified 82 or four as one of the three lead candidates in capsids that demonstrates robust transaction or retinal cells.

The data demonstrated in mice that Intravitreal administration resulted in broad retinal expression of Eagle Ford that penetrate it does afford receptor and retinal pigment did epithelial layers.

The broader retinal tropism, a baby too for Capstead nonhuman primates underscores the potential of our platform to deliver gene therapy beyond inherited diseases, including the treatment of acquired retinal disorders and maybe currently underserved.

Tim: These data support the potential use of AB204 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 demonstrated robust transfection of retinal cells. The data demonstrated in mice that intravitreal administration resulted in broad retinal expression of AV204 that penetrated to the photoreceptor and retinal pigmented epithelial layer. The broader retinal tropism of AAV2-4 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 subretinal administration.

Intravitreal administration of Baby gene therapy, which does not require surgery could potentially be performed in an outpatient setting can it be a safer and less invasive approach and Sabrina administration.

And doctors are non replicating kind of showing the potential to evade immune responses generated by exposure to naturally occurring 80 vectors.

Our library contains more than 100 capsid.

Tissue tropism selected for the potential that target a wide range of Oregon and multiple out the delivery.

An important consideration is the route of administration and we have shown data in rodents and non human primates, and we can utilize different end capsids to target the photo receptors in the retinal pigmented up affiliate using either sub retinal or intravitreal injections.

The end caps increase our potential for targeting multiple and starters their delivery of therapeutic gene arm, playing the machinery to enable gene editing.

Lastly, we have now demonstrated in vitro that's like Encapso can indeed, neutralizing antibody against actually occurring eat capsids that may be present in some patients.

We believe this finding is significant as it may allow embraced Amy gene therapy for patients who have been previously treated or excluded from study participation owing to existing anti 80 antibodies.

Tim: 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 in the retinal pigmented epithelium using either subretinal or intravitreal injection.

And beating and tie 80 community could potentially also enable retreatment of patients who previously received 80 gene therapy with other stereotypes.

We look forward to discussing these in additional in programs in the future.

I will now turn the call over to Christine or you are financials Christine.

Thank you Ken we originally 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 Thirtyth 2019.

Tim: The N-Capsids increase our potential for targeting multiple ion sorters through delivery of therapeutic genes or employing the machinery to enable gene editing. Lastly, we have now demonstrated in vitro that select AIM capsids can evade neutralizing antibodies against naturally occurring AAV capsids that may be present in some patients. 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. Additionally, evading anti-AAV immunity could potentially also enable retreatment of patients who previously received AAV gene therapy with other serotypes.

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 2008 for an increase in cash outflows of $6.2 million.

R&D expenses for the three months ended June Thirtyth 2019.

It was 16.3 million compared to 7.9 million in the same period of 2000 excellent.

The increase in research and development expenses, primarily attributed to increases in house manufacturing activities and related headcount cost.

General and administrative expenses for the quarter.

Worth 5.6 million compared to 4.6 million in the same period of 2018.

Tim: 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.

The increase in GNS expenses is primarily due to the increased headcount and related facility costs.

Net loss was 49 cents per share for the second quarter 2019, compared to 26 cents per share in the same period of 2018.

Christine: Thank you, Tim. We have recently filed the 10-Q, where you can find all the 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 three months ended June 30, 2019, 16.3 million compared to 7.9 million in the same period of 2018. The increase in research and development expenses primarily attributed to an 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 increased headcount and related facility costs.

That's the summary financials.

With respect to upcoming Investor and scientific conferences I like to highlight that onto fourth will be in Boston attending the Citi 14th annual biotech conference and on September fast. We will also be attending the Wells Fargo Securities Healthcare Conference.

Also in Boston.

We will update you on those plans presentations as we get closer to the events.

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

Thank you Christine in summary.

In the second quarter, we have made important progress that we believe positions that you own a well to drive forward our mission occurring hope into reality for our patients.

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

Thank you.

The floor is now open for your questions. If you do have a question. Please press star one on your telephone keypad at this time.

Questions will be taken in the order they weren't received [noise]. If you are using a speaker phone.

We ask that you while posing your question you pick up your handset you provide favorable sound quality. If at any time. Your question has been answered you can remove yourself from the queue by pressing one.

Christine: The net loss was $0.49 per share for the second quarter of 2019, compared to $0.26 per share in the same period of 2018. That's the summary finance. With respect to upcoming investor and scientific conferences, I'd like to highlight that on September 4th, we'll be in Boston attending the city's 14th annual biotech conference. And on September 5th, we will also be attending the Wells Fargo Securities Healthcare Conference, also in Boston. And with that, I'd like to turn it back over to Joelle.

Again, ladies and gentlemen, if you do have a question or comment. Please press star one on your telephone keypad at this time.

Please hold while we poll for questions.

<unk>.

We will take our first question from Maury Raycroft of Jefferies. Please state your question.

Hi, good morning, everyone and thanks for taking my question.

First question is on a BOE oneto one.

It seems like you you expanded the Fivea 13 cohort from three to six to four to seven in July added in efficacy based primary endpoint to look in neuro Cogs and then added some secondary endpoints as shown on clinical trials that have.

Joelle: 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.

So I'm just wondering if there are any specific reasons why some of these changes were made and if that FDA or kales encourage the changes and then just bigger picture what else is what is needed.

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

Operator: Thank you. The floor is now open for your questions. If you do have a question, please press star 1 on your telephone keypad at this time. Questions will be taken in the order they were received. If you are using a speakerphone, we ask that while asking your question, you pick up your handset to provide favorable sound quality. If at any time your question has been answered, you can remove yourself from the queue by pressing 1.

In three be to eventually seek approval for this indication.

So just to be I am already if you all here ill take this question, so you're referring to the MPS three be programs that credit so understood.

That's correct the beginning of it but yes. So these are safely changes to harmonize those the three the program will the three a program.

Well the pipeline to do is again focus the.

Of enrollments of patients, who we believe it or not.

Maurice Thomas Raycroft: Again, ladies and gentlemen, if you do have a question or comment, please press star 1 on your telephone keypad at this time. Please hold while we pull for questions. We will take our first question from Amory Raycroft of Jeffreys. Please state your question.

Have not undergone.

Much advancements in their disease, and therefore could stand to benefit to the most from the therapy.

Not to say that the children, who have more advanced disease couldn't benefit is just that.

The effect size large likely be larger in children, who were still functioning at a higher level and thats.

Codified by.

Setting up the inclusion criteria with a developmental quotient of 60% or greater.

Unknown Attendee: The 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 to look at NeuroCOG, 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 is needed in 3B to eventually seek approval for this indication.

So is that your question and we will continue to take the same approach.

On the three B program as we have on the three program and that we continue to collect.

Both biomarkers in neuro developmental data from this program.

And once we have sufficient data to to identify.

Clinical.

Benefits beyond just the biological benefits of markers, which we expect to see and have seen in the first patient access we expect to see in the.

Unknown Attendee: So just to be, I'm already throughout here, I'll take this question. So you're referring to the MPS 3B program. Is that correct? Okay.

Suing patients.

Well again engage the agency in discussions for the further development of the program.

Got it Thats helpful and then and then for the three B patients.

Unknown Attendee: So these are essentially changes to harmonize the 3B program with the 3A program. So what we're trying to do is again focus the enrollment of patients who we believe 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 would likely be larger in children who are 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 we will continue to take the same approach on the 3B program as we have on the 3A program in that we continue to collect both biomarkers and neurodevelopmental data from this program, and once we have sufficient data to identify a clinical benefit beyond the biological benefit of markers, which we expect to see and have seen in the first patient, expect to see in the ensuing patients, we will again engage the agency in discussions for the further development of the program.

Can you talk about some of their baseline characteristics at this point, maybe anything on age and baseline Eurocopter function.

And it seems like some.

Yes, I'll, let you answer that and then do a follow up.

Yes, so that these patients meet the eligibility criteria. So we're not disclosing specific age at this point, we'll provide an update on the on the.

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 right.

One in 2019, so relatively early a follow up.

But they meet criteria for the developmental quotient that as.

In general looking at they just as a framework and then not necessarily.

Citing specific cages, which I don't know, but hard for all the patients but.

Child will the developmental quotient of 60% or greater with has either NPS three or three b with the rapid progressive phenotypes tends to be.

Usually younger than that.

40 months, or so give or take and do tend to be under.

Unknown Attendee: Got it, that's helpful. And then for the 3B patients, can you talk about some of their baseline characteristics at this point, maybe anything on age and baseline neurocognitive function? And it seems like some, yeah, I'll let you answer that and then do a follow-up.

Got it okay and for three be also it seems like some other companies enrolling three be studies youve added difficult time enrolling those studies. It seems like your enrollment has picked up recently is there anything you can say about enrollment and potentially investor investigator enthusiasm for your particular approach in three b.

Unknown Attendee: Yeah, these patients meet eligibility criteria, so we're not disclosing the 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 citing specific ages, which I don't know by heart for all the patients, but a child with a developmental quotient of 60% or greater who has either MPS 3A or 3B with a rapid-progressive phenotype is usually younger than 40 months or so, give or take, and they tend to be younger.

Yes, I think that there is good momentum on both programs actually although three a low we have not enrolled into one 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 three b and I think create both are benefiting from from an effort that we started some time last year to really expand our footprint in terms of clinical trial sites. The sites continue to be very engaged still we have also.

Pretty concerted international long reach.

And.

And thats bit begs the question of whether three b., which tends to be viewed as less common than three and listened to United States that may not be as uncommon as we thought at least in some European countries. Other places such as South America. So.

Unknown Attendee: Got it. Okay.

Unknown Attendee: And for 3B also, it seems like some other companies enrolling 3B 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 potentially investor and investigator enthusiasm for your particular approach to 3B?

We are having success identifying these patients.

I also believe that will be continued.

Positive data and three a.

From the standpoint of the maintenance of.

Proven that's in Biomarkers now up to two years in CSF heparan sulfate as well as the recent data we announced some of the youngest patients.

Unknown Attendee: Yeah, I think that there's good momentum for both programs actually. Although 3A, 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. 3B and I think 3A both are benefiting from an effort that we started sometime last year to really expand our footprint in terms of clinical trial sites. The sites continue to be very engaged. We also have pretty concerted international outreach, and that begs the question whether 3B, which tends to be viewed as less common than 3A, at least in the United States, may not be as uncommon as we thought, at least in some European countries and other places such as South America.

Sort of showing.

Some neuro cognitive preservation I think that also.

Some momentum in terms of interest that we're showing that this is not only about logically active.

Intervention, but also seems at this point that we can also demonstrate some some preservation of actual clinical data which is important.

Got it yeah and for three A. for those young and higher functioning patients that you are treated in you're showing their preservation of neuro type function can you just comment on their underlying three a mutation and also the strength of the natural history data over a similar 12 to 18 month timeframe.

For those particular patients.

Yes so.

The the patients by by product protocol eligibility all patients need to have mutations that are associated with the rapid progress or phenotype.

Unknown Attendee: So we're having success identifying these patients. I also believe that with the continued positive data in 3A from the standpoint of the maintenance of improvements in biomarkers now after two years in CSF, heparin 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.

So.

In general if you look at the various natural history studies, including the one Weve worked most closely with them nationwide children's hospital, but you can look at the.

Pittsburgh data and also data from University of Minnesota, albeit with a different scale, but but certainly something that we should look at because as a pretty comprehensive.

Data set as well.

Most children with the rapid progressive form of MTS, three or three be for that matter tend to plateau and their new development before they reach age of three almost by a rule loved to see this is biology enough.

Unknown Attendee: Got it, yeah. And for 3A, for those young and higher functioning patients that you're treating and you're showing their preservation of neurocognitive function, can you just comment on their underlying 3A mutation and also the strength of the natural history data over a similar 12 to 18 month time frame for those particular patients?

So.

Mathematics, but by and large and if you look at all these studies of most of these children Decelerates and their development during their second year of life and before they reach age of three they tend to plateau and business development can start declining.

So anything that sort of deviates from that in terms of the of the.

Unknown Attendee: Yeah, so the patients, by protocol eligibility, all patients need to have mutations that are associated with a rapid progressor phenotype. So, in general, if you look at the various natural history studies, including the one we've worked most closely with from Nationwide Children's Hospital, but you can look at the Pittsburgh data, and also data from the University of Minnesota, albeit on a different scale, but certainly something that we should look at because it's a pretty comprehensive data set as well. Most children with the rapid progressive form of MPS3A or 3B, for that matter, tend to plateau in their neurodevelopment before they reach the age of three, almost by a rule. Obviously, this is biology, not, you know, 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 the age of three, they tend to plateau in their neurodevelopment and start declining. So, anything that sort of deviates from that in terms of neurocognitive development, in this case following the intervention with AB0102, we believe to be an indication that this treatment is having a biological effect and also diverging in terms of the neurocognitive course. That is what...

No cognitive development in this case following the intervention or would they be a one or two.

We believe to be already an indication that this treatment is having a biological effect and also diverging in terms of the number of cognitive of of course.

Does that answer your question.

Yes that does and.

Just last question on any one on one so for the CMC protocol protocol item, particularly the one related to the 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 am wondering.

Yes, and then I'm wondering how much our brammer is involved with helping with the protocol design in finalizing the protocol for this.

Well so so two separate questions. So what we are submitting to the agencies their request as the actual protocol will not submit any data going head to head of the phase III. So the transfer from the original retrovirus to the Bremer Retroviruses, which has been planned all along in agreement with the agency will take place or in phase three so we will be conducting the comparability studies, which are no different.

The release of the generally speaking no different than the release of studies that would do quality assurance would do for any kind of GMP product. So in this case, where you'd be a one on one so we'll be testing those.

Sure the Indiana University retrovirus material in testing those for the Bremer retroviruses material is that we would do any way to to the only the only thing. We're doing is to set up a protocol prospectively to show what the acceptance criteria are.

Unknown Attendee: Yeah, that does. And just a last question on EB-101. So for the CMC protocol, protocol item, particularly the one related to the 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 how much Brammer is involved with helping with the protocol design and finalizing the protocol for this.

Which are generally didnt already agreed upon with the agencies is literally having the political risk.

Data will be generators as we believe these batches for for clinical use as we would normally.

Now we have a use the bremer material to manufacture sheets all throughout the past say six months.

Unknown Attendee: So, two separate questions. So, while we're submitting them to the agency, the request is the actual protocol. We're not submitting any data ahead of Phase 3. So, the transfer from the original retrovirus to the Bramer 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 studies, generally speaking, no different studies that we do for quality assurance, which we do for any kind of GMP product. So, in this case, for EB-101. So, we'll be testing those for the Indiana University retrovirus material and testing those for the Bramer retrovirus material, as we would do anyway. The only thing we're doing is setting up a prospective protocol to show what the acceptance criteria are, which are generally already agreed upon with the agency.

These are obviously not not for clinical use it but certainly using the same retrovirus we.

Intending to use into phase three.

So we have experienced using the retroviruses and has performed well as expected. So we don't anticipate any surprises here.

Okay and then how involved is brand we're in the process for you with without being finalized these.

Protocols.

They have been very good partner of the Ono for a while now and.

Very closely will work closely with them.

And we have already so it's not look before looking this this has taken place.

Part of the whole manufacturing readiness for for the program.

Has included manufacturing you want a one using the retroactive bring a retrovirus. So we've done this before this is not the first time, we're going to do this.

Just going to be officially for during the phase three.

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

Unknown Attendee: 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 Bramer material to manufacture sheets 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 in Phase 3. So, we have experience using this retrovirus, and it has performed well as expected. So, we don't anticipate any surprises here.

Sure. Thank you.

Our next question comes from Kennen Mackay of RBC capital. Please state your question.

Hi, guys. Thanks for taking our recurring character concur monitored Kevin.

One on now.

Good patient related outcome.

Hi.

After be included in the Phase three study for you run a one.

Please elaborate or.

Outcomes are and how does.

Validate maybe good healing, that's your cool shell and the creation.

Yes. Thank you for your question the.

Unknown Attendee: Okay, and then how involved is Brammer in the process for you with helping finalize these protocols?

The patient reported outcomes are several on the main one being pain.

Unknown Attendee: They have been a very good partner of Abeona for a while now, and we work closely with them. Yeah. And we have already, so it's not before looking that this has taken place; it's just part of the whole manufacturing readiness for the EB program has included manufacturing EB-101 using the brain in retrovirus. So we've done this before; this is not the first time we're going to do this. So it's just going to be officially for during phase three.

For especially for the larger wounds that we're addressing in our phase three trial.

Pain is among the most disabling.

Patient patient experiences, obviously, it's a little too obviously heavy and those wont has all sorts of other medical.

Implications, including risk of infection and need for repeat wound dressing and extensive wound care and whatnot.

The pain is really the main cause of discomfort at suffering for these patients. So DFT is obviously interested in understanding how wound healing.

Unknown Attendee: Got it. Okay. Thank you very much for taking my questions, and I'll hop back in the queue. Sure, thank you.

In pain intersect.

If you asked a patients they will tell you that they are intact skin or that is.

Kenan McKay: Our next question comes from Kenan McKay of RBC Capital. Please state your question. Hi guys, thanks for taking our questions. This is Sankar Man from Kennan. So we had one on the patient-related outcomes that FDA has asked to be included in the Phase II study for EB-101. Could you please elaborate on what those outcomes are and how they might further validate maybe the wound healing that you will show in these patients?

Healed wounds doesn't hurt.

And if you ask them, which will target the most of the uniformity will tell these a bit larger wounds.

Smaller wounds or not that can still be uncomfortable, but the larger ones tend to be the one mostly associated with higher intensity obtain this has been shown.

Not only anecdotally from by asking to patients, but also in natural history studies that have been presented.

Unknown Attendee: Yes, thank you for your question. There are several patient-reported outcomes, 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, having an open wound has all sorts of other medical implications, including risk of infection, need for repeat wound dressing, and extensive wound care and whatnot. But pain is really the main cause of discomfort and suffering for these patients.

So FDA wanted to just agree with us exactly timing and how to collect those using scales that are pretty well known and its been been validated it didnt pain trials have before so it was not so much about the scales themselves is just felt the collection timing in relation to some of the.

Wound care.

Basically.

Wound care as we remove the bandages that can cause pain, because sometimes advantage, especially in the larger ones get stuck.

Unknown Attendee: So, the FDA is obviously interested in understanding how wound healing and pain intersect. If you ask a patient, they'll tell you that their intact skin, which is a healed wound, doesn't hurt. And if you ask them which wounds hurt the most, they uniformly will tell you that there are larger wounds. Smaller wounds are not; they can still be uncomfortable, but the larger wounds tend to be the ones mostly associated with higher intensities of pain. This has been shown not only anecdotally by asking the patients but also in natural history studies that have been presented. So, the FDA wanted to just agree with us on exactly when and how to collect these data using scales that are pretty well known and have been validated in pain trials before.

Also as you tender to the wounds and cleanse the wounds can clause also acute pain.

So that patients have both a chronic pain from from just having these open wounds chronically, but then it was an acute exacerbations obtained during procedures.

So it was just a.

Matter of getting the coordinators and making sure we get as much information as possible when those civil while the patients at home, but also.

Recalling that the experience when they come to the clinic clinic visits.

Got it.

Sure so.

If there are any specific percentage of being improvement you're looking.

In these patients or will it just depends.

Depending on the size of how are you thinking about that.

On being sort of mid city.

Yes, good repeat the first part of your question I just couldn't hear well.

Absolutely.

Unknown Attendee: So, it was not much about the scales themselves; it was just about the collection timing in relation to some of the wound care. Basically, when they remove the bandages, they can cause pain because sometimes the bandages, especially in the larger wounds, get stuck. Also, as you tender to the wounds and cleanse the wounds, it can cause acute pain. So, the patients have both 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 organized and making sure we get as much information as possible on those while the patients are at home but also recall the experience when they come to the clinic visits.

So just to follow up on that obviously what percentage off.

Improvement on the beans skills that you said are pretty standard I will you be looking to who will it be something similar to past you've got 50% what are you doing.

Hi.

Looking about that.

Yes. So this is the.

This is sort of the difficult question the body be because their dark.

There are much data free b., specifically, theres, obviously, a ton of data and pain, both chronic and acute pain trials in variety of conditions from cancer too.

Neuropathic pain and migraine, but there is as you can imagine there is not a loss of lives where you'd be the CES. We get so from talking to patients is the wound healing will essentially larger relief the pain, but the pain also can be quite variable and it fits under served under wraps, meaning that the wound is well dressed and they're not touching it or.

Unknown Attendee: Got it. Um, just to follow up on that, um, is there any specific percentage of pain improvement you're looking for 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? Unknown Attendee.

Cleaning itself Theres, some base thing, but it's not that severe of course as we move to wound dressings. The pain spikes up so there isn't a set threshold to two two success ultimately in the end the intent here is to heal the wounds.

Unknown Attendee: If you could repeat the first part of your question, I just couldn't hear it well.

Unknown Attendee: Absolutely, so just to follow up on that, I was wondering what percentage of improvement on the Bain scales, which you said are pretty standardized, will you be looking for, will it be something similar to, as Patty said, 50% wound healing? How should we be thinking about that?

And of course, having an open barge wound in of itself is clinically very meaningful. So if you could heal a large area of wound.

This in of itself will cause.

Bunch benefit to the patients and all the ramifications of VB wounds, but we would expect that it will also provide us can be different pain relief. So we'll measure as a continuous variable as well as some categorical variable.

Unknown Attendee: Yeah, so this is sort of the difficult question about EB because there isn't much data about EB specifically. There is obviously a ton of data about pain, both chronic and acute pain trials in a variety of conditions, from cancer to neuropathic pain, you name it, migraine. But as you can imagine, there's not a lot of that for EB.

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

Any further question Sir.

That would be all thank you.

Our next question comes from Joon Lee of Suntrust. Please state your question.

Unknown Attendee: The sense we get from talking to patients is that wound healing will essentially relieve 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 baseline pain, but it's not that severe, of course, as you remove the wound dressing, then the pain spikes up. So there isn't a set threshold for success.

Hey, Thank you for taking my question. This is found comfort for June .

Just wondering if you can tell us more about the timing initiating you on one trial its going to be early fourq event or you going to anticipate going to be a late quarter event, where you too difficult to tell at this moment.

Unknown Attendee: Ultimately, in the end, the intent here is to heal the wounds. And, of course, having an open, large wound is, clinically, very meaningful. So if you could heal a large area of wound, this in and of itself will cause much benefit to the patients and all the ramifications of EB wounds. But we would expect that it would also provide us with different pain relief. So we'll measure it as a continuous variable as well as a categorical variable.

So we can't provide any more guidance as we already have provided publicly in our 8-K filing.

Much of what remains or questions about quality and this particular question about people's that we've been discussing.

The timing now for initiation is entirely predicated on resolving these questions. So.

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 so submitted soon.

Unknown Attendee: Got it. Got it. That is super helpful. Thanks for the color.

So it was just a matter of ensuring that ft satisfied with the answers and that if there are any remaining questions that we can address those asap.

June Lee: Any further questions there? That would be all. Thank you. Our next question comes from June Lee of SunTrust. Please state your question. Hey, thank you for taking our question. This is Fang Kang for June. I'm just wondering if you can tell us a bit more about the timing of initiating the EV101 trial. It's going to be an early 4Q event, or you can anticipate it's going to be a late 4Q event, or is it too difficult to tell at this moment?

From the actual.

File readiness, so in terms of the cone cooperations and ability to manufacture the product we feel that we are ready as soon as the gays Randy.

Stanford has obviously been very close partner what would that be on for for years now.

And they are ready and willing and eager to start so so long as we get the final go ahead.

Got it Thats helpful. And then secondly, you mentioned that can't be done for you are.

Potentially can open other treatment sites for the patient and just wondering.

Joao Siffert: So we can't provide any more guidance than we already have provided publicly in our EK filing. Much of what remains are questions about quality and this particular question about PROs that we've been discussing. The timing now for initiating the trial is entirely predicated on resolving these questions, so it's 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 it's just a matter of ensuring that FDA is satisfied with the answers and that if there are any remaining questions, we can address those ASAP. Trial readiness in terms of the clinical Stanford has obviously been a very close partner with Abeona for years now, and they are ready, willing, and eager to start so long as we get the final go-ahead.

What are the potential logistic of opening aside and how long do you need to train their employees to start treating patients.

Sure.

These are sites have been.

Working with all already for several months now. So this is a long lead time in terms of just getting all the trial logistics sub set up but they also sites that are very familiar with the care of patients with VB and also have very good.

Standard Bob well qualified plastic surgery stop so.

They wouldn't be equipped in terms of the all that.

Moving parts will be fully equipped to participate in the trial and of course the team at Stanford has offered to type test had conversation with some of these sites and would offer to help train them on these more minutiae of the actual study protocol itself, but in terms of the capabilities of caring for the population in terms of the actual procedure applying these.

Joao Siffert: 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 patients. And just wondering, what are the potential logistics of opening a site and how long you need to train their employees to start treating patients?

Well the product TV one of one of these will be fully qualified sites.

Okay.

Joao Siffert: Sure. These are sites we've been working with for several months now, so this is a long lead time in terms of just getting all the trial logistics set up, but they're also sites that are very familiar with the care of patients with EB and also have very good standards, well-qualified plastic surgery staff. But in terms of the capabilities of caring for the EB population and in terms of the actual procedure of applying the product EB-101, these would be fully qualified sites.

And then thirdly I think.

In terms of the timing of that Dave how are you going to report for and.

The yield on one of your onto your second half.

Are you going to present an ad.

Medical Conference, where scientists conference where are you going to.

Press release, where you can host R&D day to present the data.

All of these are possibilities, we haven't disclosed that as soon as some of the presentations were submitted get approved and we will confirm the presentation days and then we may.

Joao Siffert: Okay, and then thirdly, I think just in terms of the timing of the data we're going to report on and ABO 101, ABO 102, the second half, are you going to present them at a medical conference or scientific conference, or are you going to a press release, or are you going to host an R&D day to present the data?

If they are updates that emerge outside of as conference schedule than Weve made.

I recall, a call or or or press release it.

Perfect.

Last question, if I may so you mentioned about and platform and then Theres a number of captives potentially can be used and you also mentioned that on the last earnings call. You are you may discuss the potential to.

Joao Siffert: All these are possibilities. We haven't disclosed that. 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 the conference schedule, then we may either call a call or press release them.

Leverage the value of the impactful on through external collaborations maybe can give us some update there and you're seeing you just any discussions going on currently in terms of.

Joao Siffert: Perfect. Last question, if I may. So you mentioned the AEM platform, and then there are a number of caps that potentially can be used. And you also mentioned that in the last NIS call, you could discuss the potential to leverage the value of the AEM platform through external collaborations. Maybe you can give us some updates there to see if there are any discussions going on currently in terms of external collaborations and how you are thinking about the AEM program and how you're going to capture that value.

For external collaborations and how you are asking about and emphasize how are you going to capture that value.

Yes, so the scoop question and one that we did in fact announced that we would seek partnerships as you know we have a very broad pipeline.

Both clinical pipeline in preclinical pipeline and Didnt. So it's a good a good problem to have but we have obviously.

Been careful that prioritizing our efforts on the lead products.

Joao Siffert: Yes, so this is a good question and one that we did, in fact, announce 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 at prioritizing our efforts on the lead products. So while also trying to continue to develop the platform, we've been in discussions for months now with several interested parties, and these have been fruitful.

So whilst also trying to continue to develop the platform we've been in discussions actually for months now with several interested parties and these have been fruitful.

We are we will announce in due course, when we have anything that is concrete in terms of sort of.

Something to disclose but theres, obviously interest.

And this platform an interest in the beta that Tim and his team have generated.

In presented as you heard earlier and we're looking forward to to continue this process. Obviously it takes time, but we're we've been active at it for for quite quite a while now so for some of these relationships are now maturing.

Joao Siffert: We'll announce in due course when we have anything that is concrete in terms of sort 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 we're looking forward to continuing this process. Obviously, it takes time, but we've been active in 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.

And we hope to have something to report in coming months.

Got it thanks, so much and thanks again for taking my question.

Sure. Thank you.

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

Good morning, many of my questions have been off maybe just.

One on on maybe want to Juan you. All can you just remind us how quickly you will be able to enroll the trial and when we could see data on the on the primary endpoint.

June Lee: Perfect, thank you so much, and thanks again for taking our question.

Liav Abraham: Sure, thank you.

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

Yes. Thanks, we are so we have the Stanford to a protocol they've had underway for a while now have been identified in screen formally screened.

Liav Abraham: Good morning. Many of my questions have been asked, maybe just one on EB-101. Joelle, can you just remind us how quickly you'll be able to...

I think 10 patients in our 11 patients.

We expect to have turned gold somewhere between 10 and 15 patients to have the number of wounds. So that doesn't give the power for the study.

Liav Abraham: Unknown Attendee, Gregory Gin, Vishwas Seshadri, Abeona Therapeutics Inc

Unknown Attendee: when we could see.

As soon as we are ready to go obviously it will start in carefully in the first patient or two to make sure everything goes well in terms of the logistics of but after that we could accelerate enrollment. So it's just a matter of scheduling.

Unknown Attendee: on the primary endpoint.

Joao Siffert: Thanks, Liav. So we have, at Stanford, through a protocol they've had underway for a while now, identified and screened, formally screened, I think 10 patients now or 11 patients. We expect to have to enroll somewhere between 10 and 15 patients to have the number of wounds that give power for the study. As soon as we are ready to go, obviously, we'll start carefully with the first patient or two 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're all waiting for the study.

Patients for treatment.

All rating full for the study.

So we could envision enrollment of course, we'll provide updates on that but could envision enrollment.

Say six months up to six months, we've tried to do faster.

Could be a little longer depends on the ability to schedule and then the trial itself has a six month follow up.

Built in so we've.

Primary endpoint at three months and recall for an additional three months to look both at the ongoing safety and and.

Healing durability.

So that that could bring us to the end of next year, depending how how quickly we can get this up and running.

Joao Siffert: So we could envision enrollment, and we'll provide updates on that, but we could envision enrollment. So, we've been doing this for, say, six months, up to six months. We try to do it faster, but it could be a little longer, depending on the ability to schedule. And then the trial itself has a six-month follow-up built in. So, we're primarily employing them for three months, and we follow for an additional three months to look both at ongoing safety and healing and durability. So, that could bring us to the end of next year, depending on how quickly we can get this up and running. Great, thank you. And then, at your RMAT meeting with...

Great. Thank you and then.

On your.

Our Mac meeting with the FDA.

And the second half of the year.

Can you just remind us what are the objectives of this meeting and.

Do you anticipate communicating with the outcome of the meeting.

To investors.

Yes, so you're referring now to MPS three a rig.

Okay, Yes.

So we we we have now beta two plus years on on safety for fourth.

Liav Abraham: The second half of the year. Can you just remind us what the objectives of this meeting are and what you anticipate...

Half of the patients at least the others are getting close to its obviously cohort three is a bit more recent.

Liav Abraham: We do anticipate communicating the outcome of the meeting to investors. Yes, so you're referring now to MPS 3A, right? Yes, Okay. Yes,

The biomarkers have been very consistently showing that this product can improve the.

Joao Siffert: So, we have now been in beta two plus years on safety for half of the patients at least, and others are getting close to it. Obviously, cohort three 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 heparid sulfate.

The disease to the underlying disease pathology that as the activity of the enzyme and clear the accumulation of Heparan sulfate and now we're having.

More data now to 12 to 18 months data in cognition for the younger children. So the question now is what's next right. So thats exactly what were going to ask the agencies. So what's the path between now and sort of moving this toward a path that can lead to to be able to gain approval. So.

Joao Siffert: And now we're having more data, now 12 to 18 months of data and 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? This is, broadly speaking, 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 engage the agency with. We'll provide updates as they materialize. Obviously, regulatory feedback is not something we provide in detail because obviously there's a whole context to one, so it's not, you know, often not sort of ready for press. But we do, we will provide guidance to the extent that the guidance is material and something that is actionable.

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 what not so.

So that's where we're looking to engage the agency with.

We will provide updates as they materialize, obviously regulatory feedback is not something that we would provide and in details because of the obviously there is a full context and one so it's not.

Often not sort of ready for.

For progress, but we do we will provide guidance to the extent of the guidance is material and something that is actionable or not.

Thank you.

Any further questions Ms. Abraham.

No I'm good. Thank you. Thank you.

Thanks Sam.

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.

Joao Siffert: Any further questions for Ms. Abraham? No, I'm good. Thank you.

Operator: 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. Have a great day. Thank you.

Thank you.

Okay.

Unknown Attendee: [inaudible]