Q4 2019 Earnings Call

Thank you for holding ladies and gentlemen, you are online today piano Therapeutics conference call. At this time, we are stuck out there. How this all participants will get started momentarily. We thank you for your patience and I see please remember the line.

Operator: Thank you for holding. Ladies and gentlemen, you are on the Abeona Therapeutics conference call. At this time, we are still gathering additional participants and will get started momentarily. We thank you for your patience and ask that you please remain on the line. [inaudible] BF-WATCH TV 2021 Copyright 2020, New Thinking Allowed Foundation. Good day, ladies and gentlemen, and welcome to the Abeona Therapeutics' fourth quarter and fiscal year 2019 conference call.

[music].

Good day, ladies and gentlemen, and welcome to the Immunotherapeutics fourth quarter in fiscal year 2019 conference call. All lines have been placed in listen only mode. In the four will be opened for your questions and comments following the presentation.

Operator: All lines have been placed on a listen-only mode, and the floor will be open for your questions and comments following the presentation. At this time, it is my pleasure to turn the call over to your host for today, Mr. Scott Santiago. Sir, the floor is yours. Thank you.

At this time, it's my pleasure to turn the call richer host for today Mr., Scott said Jamo, Sir the floor is yours.

Thank you good morning, and welcome everyone. Today's call will be led by July separate our Chief Executive Officer, Ballinger, while Kristine Silverstein, our CFO will review our financials.

Scott Santiago: Good morning and welcome everyone. Today's call will be led by Joelle Siffert, our Chief Executive Officer. Following Joelle, Christine Silverstein, our CFO, will review our... 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. These documents are available on our website, abeonatherapeutics.com. With that said, it is now my pleasure to introduce you to Dr. Jalile Sippert. Jalile, you have the floor.

After the call over to then need to remind our listeners that remarks made during this call may contain forward looking statements involve risks and uncertainties forward looking statements on this call army pursuant to the safe Harbor provisions of the federal Securities laws.

Information contained in these statements is based on current expectations is subject to change and actual results may differ materially forward looking statements. Some of the factors that could cause actual results to differ maybe found in the Companys annual reports.

Hi quarterly reports on form 10-Q.

The company with the Securities and Exchange Commission.

These documents are available on our website, maybe on a therapeutic stock.

With that said, it's now my pleasure to introduce you to Dr. <unk>, while we have four.

Thank God and and thank you all for joining us today protocol in fourth quarter and full year 2019 business highlights.

Joelle Siffert: Thank you, Scott, and thank you all for joining us today for a call on fourth quarter and full year 2019 business highlights. 2019 was a year of tremendous progress across our gene and cell therapy programs, and I look forward to reviewing our accomplishments before turning the call over to Christine to review our financials. But before proceeding, it's important to reflect upon the COVID-19 global pandemic. We are monitoring this rapidly evolving situation very closely and recognize it may impact some of our operations. Travel restrictions, as well as concerns about viral exposure, may interfere with patient screening, dosing, and follow-up across our studies. We have also conducted a risk assessment on our manufacturing operations, and so far, we have no immediate concerns.

2019, it was a your tremendous progress support across or gene and cell therapy programs and I look forward to reviewing other accomplishments before turning the call over to Christine to review our financial.

Before proceeding it's important to reflect upon the Cobiz 19 global pandemic [laughter]. We're monitoring this rapidly evolving situation very closely and recognize the main pack similar Bob operations travel restrictions as well as concerns about viral exposure manger appear with patient screening dozing and follow up across our studies we have.

Also conducted a risk assessment and our manufacturing operations and so far we have no immediate concerns.

Joelle Siffert: We will continue to monitor the situation closely while continuing to advance the development of our innovative medicines for patients in significant medical need. With that said, I'd like to start with an update on the progress we've made with our lead clinical program, EB-101, for recessive dystrophic epidermolysis bullosa, or RDAB. As a reminder, RDAB is a highly debilitating rare genetic skin disorder caused by mutations in the collagen 7 gene that makes a patient's skin very fragile, easily blistering and tearing at the slightest contact. People with RDAB develop widespread wounds that are often large and chronic and can cover a significant portion of their bodies. Patients with higher wound burdens are at risk for systemic complications that may include infection, malnutrition, anemia, and even skin cancer.

We will continue to monitor the situation closely while continuing to advance the developing innovative medicines for patients in significant medical need.

That said I'd like to start with an update on the progress we've made with our lead clinical program. If you want to one for recessive dystrophic, Epidermolysis bullosa or our dad.

As a reminder, or debt is a highly debilitating rare genetic skin disorder caused by mutations in the college and seven a gene that makes up patients skin very fragile easily blistering and tearing of the slightest contact people with regard to have developed widespread wins that are often large and chronic and can cover a significant portion.

With patients body.

Since with higher won't burden at risk for systemic complications. The may include infection, malnutrition anemia, and even skin cancer.

Joelle Siffert: There are currently no effective treatment options for these patients, and they rely on palliative care that primarily consists of painful, time-consuming bandaging and often requires prescription opioids to manage the pain. EB-101 is an autologous gene therapy, gene-corrected cell therapy that restores normal function in collagen VII, is delivered through 40-square-centimeter sheets comprised of gene-corrected keratinocytes in their In January, we began what we expect to be a transformation year at Abeona with the initiation of the VITAL study, our Pivotal Phase III trial evaluating EB-101 and RDAB. And today, I'm very pleased to share that just this morning we announced that we have treated the first patient in the VITAL study, which is our Pivotal Phase III trial evaluating EB-101.

There's currently no effective treatment options for these patients and they rely on palliative care. The primarily consist of painful time consuming bandaging and often requires prescription opioids to manage the pain.

If you want to one is an autologous gene therapy itself or the gene corrected cell therapy that restores normal function College and seven is delivered through 40 square centimeter sheets comprised of gene corrected keratinocytes and their projectors and there are transplanted don't token Williams.

January would begin what we expect to be a transformation year and I'd be owner with the initiation of the vital study our pivotal phase three trial evaluating to be one to one in our Deb and today Im very pleased to share that just this morning, we announced that we have treated the first spacing in the vital study, which is a pivotal phase three trial, the better evaluating you'd be one or one.

Joelle Siffert: The patient is doing well, following successful transplantation by the team at Stanford University Medical Center. The successful dosing of this patient, the first patient in the study, is an important milestone for this patient community, that is the RDAB community, for Abeona, and also for the Stanford team, which began this effort over a decade ago. Patients with RDAB can now look forward to potentially having effective treatment options in the foreseeable future, with Abeona's product uniquely positioned to address their significant unmet needs. A concerted effort by the Abeona team has enabled us to successfully execute the multi-step EB-11 manufacturing process at a Cleveland facility, from transduction to cell growth and expansion to packaging and transport to the clinical site for transplantation back to the patient.

The basin is doing well following successful transplantation by the team at Stanford University Medical Center.

The successful dosing of the patient.

The first station in this study is an important milestone for up to this patient community that is the our depth community.

Do you own and also for the Stanford team did which began to separate over a decade ago patients with our dad cannot look forward to potentially having effective treatment options in the foreseeable future would there be honest product uniquely positioned to address their significant unmet needs.

A concerted effort by the abbey on a team has enabled us to successfully execute the multi step you want to one manufacturing processors at Cleveland facility.

Transduction to sell growth and expansion to packaging and transport to clinical side for transportation back onto the patient the phase three study process closely parallels the potential real world application of the view, one or one and it's an important step towards commercialization of this product in the future.

Joelle Siffert: The Phase III study process closely parallels the potential real-world application of VBU-101, and it's an important step towards commercialization of this product in the future. The pivotal study is a randomized clinical trial assessing B101 in 10-15 RDAP patients with approximately 30 wound sites treated across all patients in total. We have pre-screened most patient subjects for the study and are working closely with our collaborators at Stanford and Expeditions Enrollment. We have also begun preparations for adding a second trial site on the East Coast and remain focused on advancing enrollment as soon as the third quarter. We plan to obviously provide updates before then.

I don't a study is a randomized clinical trial assessing do you want to one in 10 to 15 aren't that patients would the approximately 31 sides treated across all patients in total.

That's pretty screen most patients subjects for the study in a working closely with our collaborators the Stanford and expeditions <unk> expeditious enrollment.

We have also began preparations for adding a second trial sites some of these schools.

And remain focused on advancing enrollment as soon as the third quarter. We plan to obviously provide updates of before then.

Given the overwhelming unmet medical need <unk> patients and based on data collected from a phase one two study we continue to believe you'd be one to one has significant potential to improve the lives of patients with our debt.

Joelle Siffert: Given the overwhelming and medical need of RDEP patients, and based on data collected from a Phase I-II study, we continue to believe EB-101 has significant potential to improve the lives of patients with RDEP. IBU-101 is the only therapy in development that has published data confirming long-term healing of large wounds and also meaningful pain reduction. Successful wound healing following a B-101 treatment may also provide a meaningful reduction in time and expense. Benton Wound Care, and lower reliance on opioids and other analgesics, potentially improving patients' quality of life.

You want to one is the only therapy and development that has published data confirming long term healing of large wounds and also meaningful pain reduction successful wound healing follow you do you want to one treatment and they also provide a meaningful reduction in time and expense.

In total care, and lower reliance and opioids and other analgesics potentially improving patients quality of life.

As we look ahead to bringing if you want to want to patients. Once we refused to receive regulatory approval is important for us to briefly discuss the commercial commercial plans.

Joelle Siffert: As we look ahead to bringing AB101 to patients once we receive regulatory approval, it is important for us to briefly discuss our commercial plan. Our manufacturing facility, the Elisa Linton Center for Rare Disease Therapies, is a fully functional gene and cell therapy facility featuring CGMP capacity and laboratories to support CMC development and analytics. Our flexible, scalable manufacturing process allows Abeona to meet the demand for our Pivotal EB-101 trial and eventually also meet the demands for a commercial launch without having to exclusively rely on CDMO. Moving on from AB101, I'd like to next review our programs on MPS 3A and 3B, following the recent presentation of additional positive interim data from both studies at the World Symposium in February. MPS3A, also known as St. Filippo Syndrome A, is a rare somal storage disease primarily affecting the central nervous system, starting in early childhood.

Our manufacturing facility the Allysa Linton centre for rare disease therapies as they fully functional gene and cell therapy facility feature and cgmp capacity and laboratories to support CMC development in analytics.

Flexible scalable manufacturing process, the low of Yonah to beat the demand for our pivotal we'd be one or one trial and eventually also meet the club demands for commercial launch without having to exclusively rely on a CDMO.

Moving onto deal Bob moving on from maybe you want to one I'd like to next review or programs and MPS three a in three be following the recent presentation of additional positive interim data.

From both studies of the World Symposium in February.

MPS three a also known as simple people syndrome, a is a rare lysosomal storage disease, primarily affecting the central nervous system starting in early childhood. The transfer a studies are ongoing to your open label dose escalation phase one two global clinical trial, assessing a b or one or two for the treatment of patients with MPS three eight.

Joelle Siffert: The transfer aid studies are ongoing two-year open-label dose escalation phase 1-2 global clinical trial assessing AB0102 for the treatment of patients with MPS3A. AB0102 is an AAV9 therapy clinically proven to deliver a functional copy of the SGSA gene to the CNS and peripheral organs by a single intravenous infusion with the goal of correcting the underlying enzyme deficiency, thereby preventing the accumulation of GAGs and preventing the rapid neurodevelopmental and physical decline typical of MPS3A patients. Our most recent data update from the World Symposium showed that three patients in the highest dose cohort who were treated early continued to show improved neurocognitive skills up to two years post-treatment as compared to the natural history of disease progression. These results are important as they are consistent with well-established knowledge that early treatment provides the best chance to preserve neurodevelopment in children with a neurodegenerative disease.

If you'll want to go to the Navy nine therapy clinically proven to deliver a functional copy of the S.U.S.G. assayed gene to the CNS and peripheral oregons by a single intravenous infusion with the goal of correcting the underlying enzyme deficiency, thereby preventing accumulation of gags in preventing the rapid neurodevelopmental.

On physical decline typical have been P. S. Three eight patients.

Our most recent data update a worldsymposium so the three patients in the highest dose cohorts who were treated early you continued to show improved nerve cognitive skills up to two dispose treatment as compared to natural history of disease progression. These results are important as they are consistent with a well established knowledge. The early treatment provides the best chance.

Yes to preserve nor development of children within their degenerative disease.

We are encouraged to see clearing sustain dose related reductions in disease relevant biomarkers, including CSF heparan sulfate, demonstrating long term biologic activity of they'd be a one or two most importantly, the treatment remains well tolerated through long term follow up ranging from 15 to 45 months, both dosing with no treatment related.

Joelle Siffert: We are encouraged to see clear and sustained dose-related reductions in disease-relevant biomarkers, including CSF heparin sulfate, demonstrating long-term biological activity of AB0102. Most importantly, the treatment remains well-tolerated through long-term follow-up, ranging from 15 to 45 months post-dosing, with no treatment-related serious adverse events reported to date. This study has enrolled 14 patients to date across three dose-escalating cohorts, and we have additional subjects in screening with others identified. Of course, as discussed, study enrollment may be disrupted by COVID-19. We expect to add sites in the US and Germany to complement our existing sites in the US, Spain, France, and Australia.

It's serious adverse events reported to date.

This study has enrolled 14 patients to date across three dose escalating cohorts and we have additional subjects in screening with others identified.

Of course as discussed study enrollment may be disrupted by Cobiz 19.

Back to add does sites in the U.S. in Germany to come complimentary existing sites in the West, Spain, France in Australia.

On the regulatory front, we're pleased to reach the two to receive Yeah me Prime designation for 80 or one or two in December of 2019, adding to the our Matt So fast track rare pediatric disease designations as well as orphan drug designations in both regions.

Joelle Siffert: On the regulatory front, we're pleased to receive EMA Prime designation for AB0102 in December of 2019, adding to the RMAT, Fast-Track, Rare Pediatric Disease designations, as well as orphan drug designations in both regions. We continue to actively engage with the FDA and the EMA and plan on providing a regulatory update in the second quarter with a discussion on the path forward for this program. Transition now to the MPS 3B program, or Staphylipidal Syndrome Type B, another rare devastating fatal isosomal disease with no approved treatment that leads to neurodevelopmental decline, other systemic manifestations, and ultimately early death.

We continue to actively engage with the FDA, India me and plan on providing and regulatory update in the second quarter would the discussion on the path forward for this program.

Transition now to the MPS, three b program or simply pull syndrome type b and other rare devastating theater lysosomal disease.

With no approved treatment, which leaves the nerve developmental decline other systemic manifestations and ultimately early death.

I'm pleased to report continue to progress with the transfer B study an ongoing to your open label dose escalation phase two one to study evaluating a b or one or one gene therapy, but the main objective assessing safety under development.

Joelle Siffert: I'm pleased to report continued progress with our Transfer B study, an ongoing two-year open-label dose escalation phase 2.1.2 study evaluating ABO101 gene therapy with the main objective of assessing safety and neurodevelopment. We treated the first patient in the high-dose cohort in January, for a total of 8 patients treated to date. Screening across sites remains active, and we look forward to sharing enrollment updates and additional interim data from the study in the second half of this year. We, of course, expect additional dosing of younger patients, but these, of course, are provided. There are no further disruptions from the COVID-19 pandemic. Last month, investigators from Nationwide Children's Hospital presented encouraging data from this Transfer B Study at the World Symposium, highlighting the results from the first two cohorts, which demonstrated initial improvement in disease-specific biomarkers. Specifically, there was a decrease in heparin sulfate in the cerebrospinal fluid, reductions in plasma in urine, heparin sulfate, and GAGS, and a reduction in patient liver volume, all reported at the symposium.

Treated we treated the first space in the high dose cohort in January for a total of bay patients treated to date screen you cost across sites remain active and look forward to start sharing enrollment updates in additional interim data.

From the study in the second half of this year, we of course expect additional Doug dosing of younger patients. But these of course are provided there are no further disruptions from the Colgate 19 pandemic.

Last month investigator from nationwide Children's hospital presented encouraged they data on this up transfer B study a worldsymposium.

The highlighting the results from the first two cohorts, which demonstrated initial improvement in disease specific biomarkers, specifically there was a decrease in heparan sulfate into cerebral spinal fluid reductions in plasma in your in Heparan sulfate and gags and reduction in patient liver volume all reported that the symposium.

Together these data suggest that treatment, where they've you'll want to one may prevent the accumulation of gags, which are directly associated with the NAGLU enzyme deficiency.

Joelle Siffert: Together, these data suggest that treatment with ABO 101 may prevent the accumulation of GAGs, which are directly associated with the NAGLU enzyme deficiency. The biological activity of AB0101 observed to date is consistent with that observed for AB0102, which is for the Sanfilippo A. We're hopeful that improvement in disease-specific biomarkers may translate to improved patient outcomes in this devastating disease and look forward to providing updated data later this year. Now, shifting on to our preclinical assets, I'm pleased to report that we have made progress in our AV9 program for CLN1, or infantile Batten disease, a rare fatal inherited nervous system disorder generally presented in childhood with seizures, visual loss, and neurodevelopmental delay. In 2019, we announced FDA clearance of our IND for AB0202 and CLN1, as well as FDA fast-track designation for this program.

The biological activity or maybe one or will it be all one a one observed to date is consistent with that observed for the eight you'll one or two which is towards the San Felipe away.

We're hopeful that improvement in disease specific biomarkers may translate to improve patient outcomes in this devastating disease and look forward to providing updated data later this year.

Now shifting onto our preclinical assets I'm pleased to report that we have made progress in our Avi nine program for sale in one or infantile batten disease, a rare fatal inherited this nervous system disorder generally presenting childhood with cedars.

Visual loss in nerd developmental delay in 2019, we had announced 50 clearance of our I'm depraved yield tool to instill in one as well as Aptiv fast track designation for this program. We have successfully developed a process to manufacture this gene therapy for the phase one two clinical trial MW owner in Cleveland.

Joelle Siffert: We have since successfully developed a process to manufacture this gene therapy for the Phase 1.2 clinical trial at Abeona in Cleveland. This comes in the wake of CDMO manufacturing challenges that precluded us from moving forward into the clinic. I'd also like to briefly share an update on our novel proprietary AIM AAV Capsid library. In January, we announced the issuance of U.S. patents for AAV 204, both for the Capsid itself as a composition of matter and also methods of use.

This comes in the wake of a C D. A more manufacturing challenges the precluded us from moving forward into the clinic.

I'd also like to briefly share an update on our novel proprietary aim Avi capsid library in January we announced the issues issuances of the U.S. patents for Avi tool for both for the capsid itself as the comp in composition of matter and also methods of use this achievement strength.

Joelle Siffert: This achievement strengthens our licensed IP and recognizes the unique qualities of this gene therapy delivery method. We're excited by the potential of our AMCAPSIS to more efficiently target various tissues such as the CNS, including the neural retina, muscle, and other tissues to deliver payloads aimed at addressing a variety of devastating diseases. Additionally, we're exploring how our next-generation AAV technology could treat patients who have existing antibodies to wild AAV serotypes and potentially permit re-treatment of patients who received prior gene therapy with certain AAV vectors. We continue to characterize additional AIM capsids in non-human primate experiments and continue to receive interest from potential partners.

It is our licensed IP and recognize the unique qualities of this gene therapy delivery method.

We're excited by the potential over aim caps is to more efficiently target various tissues, such as the CNS, including the neural retina muscle and other tissues.

To deliver payloads aimed at addressing variety of devastating diseases.

Additionally, we're exploring how our next generation a bit technology could treat patients who have existing antibodies to while that you'd be serotypes and potentially permit retreatment of patients who receive pride received prior gene therapy with certain Navy vectors we.

We continue to characterize additionally, in capsids non human primate experience experiments and continue to receive interest from potential partners.

Finally from a corporate standpoint in December we announced the closing of the underwritten public offering of 103.5 million gross proceeds strengthening our financial position. We accept you expect this capital to provide us with the necessary resources to complete the vital study three do you want to one and.

Christine Silverstein: Finally, from a corporate standpoint, in December, we announced the closing of an underwritten public offering of $103.5 million in gross proceeds, strengthening our financial position. We expect this capital to provide us with the necessary resources to complete the vital study for AB101 and continue with our other clinical and preclinical programs. Our conclusion from the strategic review completed in 2019 is that it is in the best interest of Abeona to continue to develop our core pipeline products in the near term while continuing to engage with prospective business partners. With this additional funding, we believe we are well positioned to realize the potential of our innovative gene therapy pipeline, facilitated by a talented group of researchers, clinical developers, and the staff of a fully functioning and independent manufacturing facility under the leadership of Jay Bercher, who was recently promoted to Chief Technical Officer.

Continuing with the other clinical and preclinical programs.

Conclusion from the strategic strategic review completed in 2019 that isn't the best interest above you wanted to continue to develop a core pipeline flopped products in the near term, while continuing to engage with prospective business partners with the addition, with this additional funding we believe are well positioned to realize the potential over in.

Surveyed of gene therapy pipeline facilitated by a talented group of researchers clinical developers in the staff are fully functioning independent manufacturing facility under the leadership and Jay Bircher, who was recently promoted to Chief Technical Officer.

Before I turn the call over to Christine I'd like to think the entire beyond a team whose hard work dedication enabled a remarkable progress this year.

Christine Silverstein: Before I turn the call over to Christine, I'd like to thank the entire Abeona team whose hard work and dedication enabled our remarkable progress this year. In addition, we remain deeply grateful to the patients, families, clinicians, researchers, and patient organizations who have made our work possible. We look forward to continued partnerships and remain committed to delivering improved therapies for patients who most desperately need them.

In addition, we remain deeply grateful to the patients family families clinicians researchers inpatient organizations will have a made or work as possible.

Four to continue partnerships and remain committed to deliver delivering approved therapies for patients who most desperately need them with that I'll now turn over to 'cause to Christine Christine.

Christine Silverstein: Thank you, Joelle. I'd like to remind everyone that we have recently filed our Form 10-K, where you can find all the specific details on our financial results. But in summary, our cash, cash equivalents, and marketable securities as of December 31st, 2019 were $129.3 million compared to $47.9 million as of September 30th, 2019. The increase in cash, as Joelle mentioned, of $81.4 million was driven primarily by the $103.5 million gross underwritten public offering. The net loss was $0.30 per share for the fourth quarter of 2019, compared to $0.36 per share in the comparable period of 2018. For the 12 months ended December 31st, 2019, the net loss was $1.51 per share, compared to $1.19 per share in the same period of 2018. That's the summary of the financial.

Thank you draw I'd like to remind everyone that we have recently filed our form 10-K, where you can find all the specific details on our financial results.

But in summary, our cash cash equivalents and marketable securities as of December 31st 2019 were 129.3 million compared to 47.9 million as of September Thirtyth 2019, the increasing cash as Joel mentioned of 81.4 million Bucks driven primarily by just under 3.5 million.

Underwritten public offering.

Net loss was 30 cents per share for the fourth quarter 2019, compared to 36 cents per share and the comparable period of 2018.

For the 12 months ended December 31st 2019, net loss was $1.51 per share compared to $1.19 Crusher nothing period in 2018, that's a summary of financial.

However, before I turn it over back to Joelle I want to address my pending departure from the company.

Joelle Siffert: Before I turn it over back to Joelle, I want to address my pending departure from the company. As you may have read in the filing last night, it is with a very heavy heart that I have tendered my resignation as CFO, effective March 31, 2020. I will further be available as an advisor to ensure a smooth transition for the company and further as a board member. Ed Carr, our Chief Accounting Officer, has been an instrumental part of the financial operations built over the last year, and having worked closely with him, I strongly believe that Abeona and its finance team remain in very capable hands under his leadership. I'll now turn it back over to Joelle. Okay?

As you may have read in the filing last night as with the very heavy heart tendered my resignation as CFO effective March 31st 2020.

I will further to be available as an advisor to ensure smooth transition for the company and further as a board member.

Ed car, our Chief Accounting Officer has been instrumental part of the financial operations built over the last year.

And having worked closely with him I strongly believe that either yonah and avionics finance team remains in very capable hands, but his leadership.

I'll now turn it back over to July.

Yeah.

Thanks, Christine over the past three and a half years Christine has made numerous contributions to the have you on organization and has helped position us for successful 2020 nanometer passion for have Yonah and its mission to transform the lives of patients with serious diseases were pleased to announce the Christine has been appointed to see turn it on board of directors.

Therefore, while pristine is no longer Serbian or turn capacity as CFO her contributions to the up you own and its mission will continue I.

Thank you Christine.

In summary, we've achieved a interim milestones in the fourth quarter. They have a beyond a well positioned to bring long term value to our shareholders and turn hope into reality for patients and their families.

Joelle Siffert: Thanks, Christine. Over the past three-and-a-half years, Christine has made numerous contributions to the Abeona organization and has helped position us for a successful 2020. In her passion for Abeona and its mission to transform the lives of patients with serious diseases, we're pleased to announce that Christine has been appointed a seat on our Board of Directors. Therefore, while she is no longer serving in her current capacity as CFO, her contributions to Abeona and its mission will continue, and I thank you, Christine. In summary, we achieved interim milestones in the fourth quarter that have Abeona well-positioned to bring long-term value to our shareholders and turn hope into reality for our patients and their families. Further, treating the first patient in the phase three trial, the study is a tremendous accomplishment for all involved, including the dedicated teams at Abeona and our close collaborators at Stanford University. Most importantly, we're very thankful for patients who volunteered to participate in this pivotal study and for the RDAB community who has been supportive of this program for many years. Thank you, and I'll turn over to the operator for questions.

Further treating the first station in the first then the phase three trial. The study is a tremendous accomplished for accomplishment all involved including the dedicated teams that I'd be owner in our clothes collaborators at Stanford University. Most importantly, very thankful for patients to volunteer to participate in this pivotal study into the aren't up community who has been.

In support of this program for many years, Thank you and I'll turn over to the operator for questions operator.

Thank you, ladies and gentlemen, we will not take your questions at a star one on your telephone keypad. If you had a question or comment.

Using a speaker phone we ask that you. Please pick up your handset to provide the best how quality.

Again star one for any question.

Our first tomorrow I worry capped at Jefferies.

Hi, everyone now good morning, Congrats on the milestone today and died thanks for taking my questions and best wishes on next steps. Christine first question is on the patient dose I, probably not going to provide a lot more details, but wondering if you can say when the patient was first street.

Operator: Thank you. Ladies and gentlemen, it is star one on your telephone keypad if you have a question or comment. If you are using a speakerphone, we ask that you please pick up your handset to provide the best sound quality. Again, star 1 for any questions. We will go first to Maurice Raycroft. Out you go.

David If you can talk about the wound size number ones and down the history of ones at the patient ad.

I'll I'll start there.

I will be somewhat circumspect about a lot of the details, obviously, but privacy and obviously, we'll provide general updates, but not a blow by blow update but suffice it to say this is.

Maurice Thomas Raycroft: Hi, everyone. Good morning. Congratulations on the milestone today. And thanks for taking my questions and best wishes on next steps, Christine. First question is about the patient dose. You're probably not going to provide a lot more details, but I was wondering if you could say when the patient was first treated and if you could talk about the wound size and number of wounds.

A young man, who had been actually.

A volunteer for the first study and unfortunately at that time could not be treated after a successful biopsy.

Because he had developed fever and infection. So he had actually been waiting for this study for quite awhile.

So that's goes to show the at the level of unmet need and then sort of the promise of this treatment.

Joelle Siffert: I'll be somewhat circumspect about a lot of the details, obviously, but for privacy, and we'll obviously provide...

Patient.

Suspicion that had to as you can imagine.

Joelle Siffert: This is a young man who was actually a volunteer for the first study, and unfortunately, at that time, he could not be treated after a successful biopsy because he had developed fever and infection. So he had actually been waiting for this study for quite a while.

Many of the patients with our doesn't have a very large won't burden, we were able to treat.

As many as possible within the there was sort of be the compliance of the study this station.

Unfortunately still has more open wounds that could not be addressed in the single sitting so as we mentioned before our intention is to deploy the second study, which is not a registration study, but rather as a phase three b study, where we plan to offer a re dosing for patients who participated in.

Joelle Siffert: So that goes to show the level of unmet need and sort of the promise of this treatment. This patient had, as you can imagine, many of the patients with RDEV have a very large wound burden. Although we were able to treat as many as possible within the compliance of the study, this patient unfortunately still has more open wounds that could not be addressed in a single sitting. So, as we mentioned before, our intention is to deploy a second study, which is not a registration study, but rather is a phase 3B study where we plan to offer re-dosing for patients who participated in either the phase 1-2 trial or in the phase 3 trial so that they can come back to have some of their other wounds treated. The actual process itself, the manufacturing and deployment of the EB-101 sheets, and ultimately the surgery and post-operative care have been successful to date. So we're pleased for the patient and, of course, pleased with the execution of this whole process.

Either the phase one two trials are into phase three trials, so where are they can come back to have some of their all the wounds are treated.

The the actual process itself manufacturing and deployment of the of the if you want to one sheets.

And ultimately the surgery imposed stop there have been successful to date so.

We're pleased for the for the patient and of course.

Please with the.

Well the execution of the this whole process.

Got it and for the additional patients pre screen and it'd be Q can you say, that's closer to five or 10 or 15 and anything else you can say about the baseline characteristics for those patients.

Joelle Siffert: And for the additional patients pre-screened and in the queue, can you say if that's closer to 5 or 10 or 15? And anything else you can say about the baseline characteristics of those patients?

Yes, so we have.

We meaning Sanford has been running your pre screening protocol for quite a while the fact, they prescreening patients for all the open studies, including some of the other dollar Deb studies, which is actually a nice process because they have all of this kind of mapped out for our program specifically they pre screen the 11 patients.

Joelle Siffert: Yeah, so we have, we meaning Samford, been running a prescreening protocol for quite a while. In fact, they prescreen patients for all the open studies, including some of the other RDAP studies, which is actually a nice process because they have all this kind of mapped out. For our program specifically, they prescreened 11 patients. As we discussed, our intention is to enroll somewhere between 10 and 15 patients, depending on the number of wounds treated. So we're well positioned on that.

As we discuss our intention is to.

Enrolled somewhere between 10 and 15 patients then depending on the number of wounds treated.

So we're well positioned on that of course, you know and the thing that may be repeated.

Frequently from over the next I mean, maybe weeks and months a lot of the plans now may or may be disrupted based on the Cobiz 19 that did not happened with the patient who was treated.

Joelle Siffert: Of course, you know, the thing that may be repeated frequently over the next, I mean, maybe weeks and months, a lot of the plans now may be disrupted based on COVID-19. That did not happen with the patient who was treated, but it depends a lot on local regulations vis-à-vis the medical centers, whether or not they will allow elective surgeries and participation in clinical trials. The U.S. agencies have not yet issued anything formal. This morning, we got communication from the German authorities as well as the Spanish authorities providing some guidance on that, and of course, locally, the hospitals and, of course, ultimately, the investigators will make a decision. So it's likely that some of the activities will need to be postponed, and we don't have a lot of visibility yet on the EB-101 program.

But you know for subsequent of.

Patients will depend a lot on a local regulations vis-a-vis the medical centers, whether or not they will allow.

Elective surgeries and participation in clinical trials of the U.S. agencies have not yet issued anything a formal.

This morning, we got communication from the German authorities as well as the Spanish authorities.

Providing some guidance on that and of course of locally the hospitals and of course.

Ultimately the investigators will will make a decision so it's likely that some of the activities, where we need to be postponed don't have a lot of visibility yet on the B one one program.

But for certainly for the European sites on the Sanfilippo syndrome, we anticipate a certain amount of.

Joelle Siffert: But certainly for the European sites for the Sanfilippo syndrome, we anticipate a certain amount of delays in the treatment of patients. Also, for follow-up of patients already enrolled in the trial, especially for areas most affected by COVID-19, some of the immediate follow-ups will be, unless there's obviously a medical issue, but if it's a routine visit for the studies, a lot of these will be done online or by

Delays and then the treatment of patients.

Also for a follow up with patients already enrolled into trials specialty for areas most affected by cope with 19.

Some of the median follow ups will be unless there is obviously a medical issue, but if it's a routine visit for the studies a lot of these will be done online.

Or by telephone.

Got it and then one last question for me just based on the cadence of treating patients in the phase three so I guess, what's your plan for that assuming that koby 19 doesn't impacted Q, Matt too much when will you dose. The next patient and then is there potential for you to try to ramp.

Joelle Siffert: Got it. And one last question for me, just based on the cadence of treating patients in phase three. So I guess, what's your plan for that? Assuming that COVID-19 doesn't impact it too much, when will you dose the next patient? And then is there potential for you to try to ramp up production and potentially start treating two patients at a time?

Ben ramp up production potentially start trading to patients at a time.

Yes, we have the capacity and that was the plan until now so obviously the the exercise here is to.

Joelle Siffert: Yeah, we have the capacity, and that was the plan until now, so obviously, the exercise here is to be very mindful of, because just to backtrack a bit, it takes about four weeks to process the biopsy into these gene-corrected cell therapy sheets. So the main decision here will be if we were to biopsy a patient, you know, because that's a procedure itself, we want to make sure that the patient can then come back when the sheets are ready to receive the actual product. So that's basically the decision we'll have to make going forward. Obviously, patients who have already been biopsied, we are intending to treat, but patients who have not yet been biopsied, this will have to be a very close discussion between Abeona, the investigators at Stanford, and, of course, the medical center at Stanford, too. In patients, too, families have to travel for this, so some families may choose not to come for the visit.

Be very mindful of because just the backtrack a bit so but it takes up about four weeks to to process. The biopsy into these gene corrected cell therapy sheets. So the main decision here will be if we want to biopsy a patient.

No because that's a procedure itself, we want to make sure that the patients and then come back when the sheets are ready for receiving the actual product. So that's basically the decision we'll have to to make going forward.

The patients who weren't biopsy, we're intending to two to treat but patients who have not yet in biopsy. This will have to be a very close discussion.

Between have you own or the best skaters at Stanford and of course, a medical center at Stanford to.

In the patients to families have to travel for this so some families we choose not to come for for a for the business.

Okay. Thank you for taking my questions I'll back in Q.

Maurice Thomas Raycroft: Okay, thank you for taking my questions. I'll be back in a few. We'll go next to Manny Forajara, SBB, Lee Ring.

Sure.

Well go next to many for her STB leerink.

Unknown Attendee: Hi guys, thanks for taking my questions. And best wishes to you, Christine. Congratulations on getting a woman on the board, although not exactly the mechanism we had thought. You talked a little bit about expanding the IP and continuing to build your IP portfolio around your earlier stage preclinical pipeline opportunities, Baton disease, etc. How do you think about putting resources to work in earlier stage opportunities for developing preclinical assets independently or for partnership versus a pretty full docket of three programs already in the clinic? And then, as a second question, how should we think about the impact of or travel restrictions or practical logistical issues in terms of, You know, in terms of the feedback you may get from the FDA and that conversation, has that in any way sort of slowed your progress Or was that largely a virtual, non-in-person process to begin with?

Hi, guys. Thanks for taking my question and best wishes on you Christine Congratulations on getting a woman on the board, although not exactly the mechanism you thought talk a little bit expanding the IP and continuing to build your IP portfolio around earlier stage Creek clinic.

Oh pipeline opportunities batten disease et cetera.

How do you think about.

Putting resources to work in earlier stage in earlier stage opportunities developing preclinical assets independently or for partnership.

Versus a pretty full docket of three programs already in the clinic.

And then the second question.

How do you how should we think about the impact of.

Sorry, travel restrictions or practical logistical issues in terms of.

Yeah. It in terms of the feedback you may get from the FDA and that conversation has that in any way sort of slow your progress our regulatory conversations or is that largely a virtual non person process to begin with.

Yeah, so to two separate questions. So.

Joelle Siffert: Yes, so two separate questions. The first question related to the rank order of our pipeline priorities. So clearly, the clinical programs take precedence. That's where most of the effort, the impetus, and the focus are currently deployed. EB-101 being the most active of them and certainly the most advanced. So there's the greatest priority for EB-101, as we obviously continue to be very focused on the two San Filippo programs because these are enrolling patients. So we're obviously much more responsible for everything related to activities that involve patients. So these are the priorities.

The first question related to the rank order of our pipeline priorities. So so clearly the clinical programs take precedence that that's where most of the effort and the importers into focus or are currently.

Deployed you'd be one to one being the most active of them and certainly the most advanced so there's the greatest priorities any do you want to one whereas we obviously continue to be very focused on the two San Felipe program. Because these are enrolling patients. So we obviously much more responsible for for everything related to two.

Pivoting living involve patients. So so these are the priorities.

Joelle Siffert: At the same time, we believe that there's value in our preclinical pipeline, both in terms of medical value, that is, new programs that could address significant and met disorders, but we are being very judicious in how we advance those. So we're completing some of the characterization of the AIM capsids, including select non-human primate studies to round out the characterization of these capsids, and also advancing some of the preclinical programs within the confines of obviously Abeona here and some of our collaborators. So this is something that we have more control over. It doesn't require patient involvement and travel and whatnot. These are much more of the scale of these investments, and the effort is obviously commensurate with the level of development.

The same time, we believe that there's value in or preclinical pipeline. Both in terms of medical valley that as you know, but new programs that could address the significant unmet disorders, but we're being very judicious of how advanced that so we're completing some of the characterization of the aim capsid, so including the select.

Non human Primate studies to round out CSK the characterization of these captives.

And also.

Advancing some of the preclinical programs within the confines of obviously you have the owner here or in some of our collaborators. So this is something that we have more control doesn't require patient involvement travel and whatnot. These are much more snub sort of the scale of these investments and the effort.

The commensurate to the level of development, so, whereas the bulk of the effort and resources are deployed clinical programs.

Joelle Siffert: So whereas the bulk of the effort and resources are deployed to clinical programs, we have a very precise, focused, and circumscribed effort to keep these assets moving forward, whereas at the same time, not detracting from the clinical programs. This is important because there's value in these assets. As I mentioned, both medical and economic value if you could fast forward on some of these programs that we're entertaining.

We have a very.

Besides the focus in circumscribed effort to keep these assets moving forward, whereas at the same time not detracting from the clinical programs.

This is important because there's value in these assets as I mentioned, both medical value. If you could pass forward on some of these programs that we the though were entertaining and also potentially these could be.

Joelle Siffert: And also potentially, these could be parlayed into partnerships, about which discussions are always ongoing. And these, as you can imagine, are in gene therapy. And there's a possibility that some of these partnerships may bring resources to the programs that could be developed with other collaborators. So we want to keep those active as to enable us also to establish these partnerships and get the most value out of this early pipeline. Separately, you asked about regulatory interactions. Much of the regulatory interactions with the FDA, in fact, over the past year have been virtual, either phone calls or correspondence. The OTAT division is overwhelmed with a lot of actually successful programs. Gene and cell therapy have been quite successful over the past several years, and they're contending with a tsunami of new INDs and submissions and whatnot.

Parlayed into partnerships, which continued discussions are all is ongoing and visas you can imagine in gene therapy, and there's a possibility that some of these partnerships and they bring in resources to the programs that could be developed to with other collaborators. So well we want to keep those active as to enabled us also.

To to set these partnerships and bring the most value out of this early pipeline.

Separately, you asked about regulatory interactions much of the rate regulatory interactions with the FDA effect over the past year have been.

Virtual either phone calls for correspondence the old Pat Division is.

Overwhelmed with a lot of actually successful programs of gene and cell therapy have been quite successful the past several years in their contending with a tsunami of new ideas and submissions and whatnot. So.

Joelle Siffert: So in that sense, in terms of the logistics, I don't anticipate necessarily that this would delay any of the interactions, but I do not have any visibility as to their internal demands now with COVID-19, which I assume exists across the agency, but I don't have any, I don't have direct visibility as to how this will affect their operations.

They have been quite a judicious with granting five meetings so in that sense.

In terms of the logistics I don't anticipate necessarily that this would delay any the attractions, but I do not have any visibility as to their internal demands and now would cobiz 19, which I assume that exist across the agency, but I don't have any I'll have direct visibility at this too to how this will affect their offers.

Patients, but we'll know more on red in the coming weeks.

Unknown Attendee: Great, thanks for answering my questions, and I'll hop back in the queue. All right, thanks.

Great. Thanks for answering my questions and I'll hop back in the Q.

Uh huh.

Kristen Brianne Kluska: We'll go next to Kristen Kluska at Cancer Fitzgerald. Hi, good morning, and congratulations on the patient dosing announcement this morning. So now that your Phase 3 trial is up and running, how are you thinking about a commercial strategy at this point, both in the U.S. and outside the U.S.? I know the previous chief commercial officer had laid out some initial potential plans for this. Do you plan to reevaluate this if you hire a new CCO?

Well go next to Christian Glasscock Cantor Fitzgerald.

Hi, good morning, congratulations on the pace patient dosing announcement. This morning, So now that your phase three trials up and running how are you thinking about commercial strategy at this point both in the U. S and X U.S. I know the previous Chief commercial officer had laid out some initial potential plans for this.

Do you plan to reevaluate this if you hire a new CCOH.

So couple of questions, there things and sort of question. So.

Joelle Siffert: So, there are a couple of questions there. Thanks for your question.

Joelle Siffert: So, yeah, so obviously as we start to phase three, that's sort of the set goal for deploying a commercial strategy. We have had, and this person continues to be, a consultant in commercial who worked with our previous CCO, and we're actively recruiting for a new CCO to join the company. We actually have quite qualified candidates who we're very pleased with.

Yes, so obviously as we start the phase three that's sort of the sort of set a goal for deploying a commercial strategy and we have have happened as person continues a consultant in commercial who had worked with our previous CTO and we're actively recruiting for for new see fuel to two joined the company would have actually quite.

I'll fight candidates, we're very pleased with.

So this will be deployed obviously of the.

Joelle Siffert: So, this will be deployed. Obviously, the launch plan is a couple of years in the making. We have to start now. We have, in fact, started a few activities already. Even late last year, we were already working on this, and at some point, we'll have additional hires in terms of the actual specific people and marketing and also health outcomes, etc. So we're still very much focused on getting ready for launch with the, assuming the timelines will be disclosed, the completion of enrollment this year, and data in the first half of next year. So this is timing for commercial planning starts now. I don't know if that answered your question specifically.

The launch plan is that there's a couple of years the into making we have to start now we have in fact started a few activities already even late last year, we already working on this and at some point, we'll have additional hires so in terms of the actual specific people wouldn't been marketing and also health outcomes et cetera, so well.

Still very much focused on getting ready for launch.

The assuming the timelines that where we dispose the completion of enrollment see or data first half of next year. So this is timing for commercial planning starts now.

I don't know that answering your question specifically.

Yes. It does thank you and just one follow up here could you characterize the primary endpoint of this trial, how it's 50% wound closure is going to translate for these patients meaning to what extent does reduce the risk of the co morbidities that lead to the serious adverse effects or mortality like the squamous cell carcinoma section.

Kristen Brianne Kluska: Yes, it does. Thank you. And just one follow-up here. Could you characterize the primary endpoint of this trial and how a 50% wound closure is going to translate for these patients? Meaning, to what extent does this reduce the risk of the comorbidities that lead to serious adverse effects or mortality, like squamous cell carcinoma and infections? And how do you consider this, especially since you are the only company that has the long-term data on hand from the initial trial? Unknown Attendee

And how do you consider this especially since you are the only company that has a long term data on hand from the initial trial.

Yeah, So sorry for the first question obviously, the the the overall impact will depend on how many how much of the one burden as you can kind of grass. So I'll come back to that in a minute, but I'll lesser bye bye.

Unknown Attendee: Yeah, so I'll start with the first question. Obviously, the overall impact will depend on how much of the wound burden you can address. So I'll come back to that in a minute.

Joelle Siffert: But I'll answer by referring to the data that's been published. So the Stanford team published two papers, three papers, but two in the clinical trial, the Phase I-II trial. One some time ago, I think in 2017 or 16, in JAMA.

Referring to the data that's been published so the the Stanford team published two papers sub three papers, but two on the clinical trial a phase one two trial one.

Some time ago, I think in 2017 or 16.

Joelle Siffert: And then, most recently, they published a long-term follow-up paper at JCI. These are obviously two peer-reviewed papers, reputable journals, and what they describe and the data they published is that, first of all, patients have durable wound healing, a majority of patients have durable wound healing, and these were large wounds to start with. Most patients in the Phase I-II study received anywhere from four to six transplants; I think six transplanted sheets, and each of these sheets, at the time of the Stanford production, were 35 centimeters squared, so if you do the math, we're talking about over 200 centimeters squared of addressed wounds in the trial. So for the patients who continue to be followed, these patients that had durable wound healing, not all but the majority of the wounds were healed at a very long term.

Jama and then most most recently they published a long term follow up or pay for a J.C. I.

These are obviously to peer reviewed papers.

Reputable journals and what they described in the data obviously published is that.

First of all patients have thought durable wound healing and majority of patients have durable wound healing and these were large wants to start with.

Most patients in the phase one two study received anywhere from four to six.

Transplant us I think six transplanted sheets and each of these sheets of the time of the Stanford production were 35 centimeters square so to do the math.

Talking about over 200 centimeter squared up address address wounds in the trial.

So for the patients who.

Continued to be followed these patients that had durable wound healing not all but majority of the wounds were healed and very long term and when we looked at patients who had a greater than 50% a wound healing. So by one side of these were clearly associated with the absence of paying on those won't sites. So we know that.

Joelle Siffert: And when we looked at patients who had greater than 50% wound healing, so by wound site, these were clearly associated with absence of pain on those wound sites. So, we know that wound healing is associated with pain, in this case, in particular for these large wounds, even a 50% reduction in the wound size was associated with a benefit in terms of pain. Measurements of quality of life in this patient population are more complicated in that a lot of the things that affect their quality of life result from years of very large wound burden and chronic wounds. So in as much as pain and, of course, wound care affect quality of life, which, of course, it does significantly, the more we can treat their wounds, the larger the area, I think the greater the overall impact will be on their quality of life. They have other limitations regarding ambulation. For example, some of these patients have developed fusing of their fingers, meaning a pseudo-syndactyly that obviously limits their ability to use their hands. They have joint contractures.

Wound healing.

Is associated with the pain in this case in particular for these large won't even a 50% reduction and the wound size.

Was associated with the benefit in terms of pain.

Measurements of quality of life and this.

Patient population is more complicated in that says a lot of the things that affect their quality of life or results from years longer a very large shoveling burden in chronic wounds. So in as much as pain and of course wound care affects quality of life, which of course it does significantly the more we can treat their wounds the largest.

The area that I think the grade as the overall impact will be for their quality of life.

They have other limitations regarding emulation. Some some of these patients develop fusing of their fingers, meaning a suit us and back to leave that obviously limits their ability to to use their hands.

They have a.

Drilling contractors, they have a issues with maintaining a good nutritional sat as et cetera. So.

Joelle Siffert: They have issues with maintaining a good nutritional status, et cetera. So one could imagine that if you can, over time, treat a significant proportion of their wound burden, you know, whereas you won't cure the disease, you could start to make a dent in some of the systemic manifestations, decrease their metabolic demands, improve their nutritional status, obviously reduce pain, and soften the burden of wound care, which is quite significant. So I'm just, you know, thinking sort of longer term here, and that will require, you know, I think, pretty extensive management of their wounds. This, you know, if you ask the patients, and I haven't asked them personally because I don't know them, but the Stanford team has asked these patients who participated in the Phase 1-2 trial, and they uniformly said that they would like to be retreated if they were given the option.

One could imagine that a few overtime can treat a significant proportion of their wounds burden.

Whereas you won't cure the disease, you could start to make a dent on some of the systemic manifestations decrease their metabolic demand improved their nutritional status.

Obviously reduce pain and and then and then soft and the burden of a wound care, which is quite quite significant so.

Just if you don't thinking sort of longer term here and there were required to you know I think of a pretty extensive management of their wounds.

This you know if you ask the patients and I haven't asked them personally because I don't know them, but the scan for team has asked these patients have participated in the phase one two trial and the uniformly said they would like to be retreated if that were given the option. So I think whereas this is not a scientific research it is oh.

Joelle Siffert: So I think, you know, whereas this is not a scientific research, it is, I think, ultimately the patients are the ones who will tell us whether or not they think this is meaningful, and then, you know, to the best of our ability to assess their quality of life from a distance, it seems to the Stanford team and to us that this is quite a strong testament that this particular product has provided relief and benefits to these patients who would, if given the choice, volunteer to getting more of these AB101-corrected cell therapy sheets.

Ultimately the patients are the ones, who will tell us whether or not they think this is meaningful and and then you know to the best of our ability to to assess their quality of life from a distance.

It seems to Stanford team and to US that this is quite as strong Testament that this this particular product has provided a relief and benefits for these patients who wouldn't who would if given the choice volunteer to to getting more of these so if you want to one or.

Correct and does cell therapy sheets.

Kristen Brianne Kluska: Great. Thank you, Joelle. Again, I will star 1 for any questions. We will go next to Kenneth Mackey at RBC Capital Markets. Hi, thanks for taking the questions. A couple here as well.

Great. Thank you draw.

Right.

Again, I will start one for any questions. We'll go next to kind of Mackie at RBC capital markets.

Hi, Thanks for taking the questions a couple series as well first on.

Kenneth Mackey: First on RDEV, I was wondering if there were additional centers you were planning to eventually open beyond Stanford and screen from, and if there were any estimates you could help us with around enrollment timelines, either prior to the COVID-19 impact, just to help us understand this, or again, adjusted for what we're hearing or what we know so far around the COVID-19 impact. And then I was just hoping you could elaborate a little bit on the manufacturing challenges and the MPS, sort of where we are now, what next steps are, and again, if there's any pre or post COVID estimates on timelines to resolution there. And, lastly, Christine, sorry to hear about your departure. I was hoping maybe you could elaborate a little bit more on this decision. It seems like departure at the end of the month is about two weeks notice here. Thank you again for taking the time.

I was wondering if there were additional centers were planning to eventually open beyond Stanford and screen from.

And if there were any estimates you could help us where the ground enrollment timelines other prior to the corporate 19 impact I'm just to help us understand this or a again adjusted for what we're hearing or what we know so far around.

Carbon 19 impact and then was just hoping you could elaborate a little bit on the manufacturing challenges and MTS sort of where we are now what next steps are and again, if there's any pre or post our corporate estimates on time monster to resolution there and then lastly, Christine.

Oh, sorry to hear about your departure I was hoping maybe you can elaborate a little bit more on this decision. It seems like departure at the end of the month is about.

Two weeks' notice here.

And your again for taking the questions.

Oh, Hi, Kennen. Thanks.

Joelle Siffert: Oh, hi Canon.

So oh go by step here, if I forget something remind me, but second center. We are identified the second centered on the east coast, where in the process of going through contracts I'd be review et cetera, assuming there are no disruptions on their side, which should be able to two analysis centre in the coming months.

Joelle Siffert: So I'll go step by step here. If I forget something, just remind me. But Second Center, we have identified a Second Center on the East Coast. We're in the process of going through contracts, IRB review, etc. Assuming there are no disruptions on their side, we should be able to announce the Center in the coming months. Obviously, a lot is up in the air now because people are worried about other things that perhaps take precedence. But if no disruptions occur, that will be the plan. We had announced that we expected enrollment to be completed by the third quarter of this year, and we're geared up to do that and planning accordingly, including scheduling patients for biopsies at Stanford and getting everything prepared for that.

[music].

Obviously, the largest up in the air now because people are worried about other things.

Perhaps take precedence, but but if you know no disruptions, though is the plan.

We had announced that we expected enrollment to be completed by third quarter. This year.

And we're geared up to do that and planning accordingly, including scheduling patients for biopsies, Stanford and getting you know all on prepare for that.

Joelle Siffert: Obviously, with COVID-19, a lot of these plans are being reassessed. I don't have any definitive answer one way or the other, and I think a lot of it will depend on what happens in the coming weeks. We don't believe this to be a permanent setback, but it could be disruptive, you know, temporarily. I just heard this morning that the Bay Area is blocking all movement around and stuff, so I'm not even sure how that, you know, how far it goes, whether it includes Palo Alto or not.

Obviously.

With the covert 19 up a lot of these plans or you know being reassess I don't have any definitive answer one way or the other than I think a lot of it will depend.

On on what happens in the coming weeks.

If we don't believe this to be a permanent setback, but it could be disruptive you know temporarily.

Just for this morning, the Bay area is blocking all movement around and stuff. So I'm not sure even how that how far it goes whether it includes Palo alto or not so even you know as early as this morning, the been news on this [noise].

Joelle Siffert: So even, you know, as early as this morning, there's been news about this. Switching gears to the MPS manufacturing, as we announced before, we are now looking ahead to manufacturing MPS 3A product, and so that is AB0102 at our Cleveland facility, and we have begun the efforts and activities to develop the process which has been so far successful and also scaling up with additional equipment to be able to produce this on a larger scale. So this is underway; obviously, these processes take time until we can have a final release of the clinical material, but we are well underway on that, and some of the effort deployed last year for the Batten disease product has equipped the team and the staff at Abeona to actually be more proficient with the process development, and that was an important sort of step up for everyone in Cleveland who successfully went through that process and actually blocked the process, in fact, for manufacturing. So that bodes well for the MPS382, because the same team is working on it now. It's a very qualified team. So I guess that's the first question for me. I'll let Christine speak for herself since she's still here.

Switching gears to the MPS manufacturing, we as we announced before we are now looking ahead for manufacturing MPS three a product.

And so that is a deal went on to it at a at our Cleveland facility and we have begun already via the efforts in activities. So to develop the process, which has been so for us asphalt and also scaling up with additional.

With mentor to be able to produce this into larger scale. So this is underway. It's of obviously these processes take time until we can have a final release of the clinical material, but we are well underway on that and where some of the effort a deployed last year for the batten disease product.

Has equipped to the team and the staff and beyond the two to actually.

Be more proficient with the process development and that was an important.

Sort of a step up for everyone in Cleveland to successfully went through that process and actually locked the process in fact for manufacturing so that bodes well for the MPS three two sourcing team is working on the snow very qualified team.

So I guess that that's the first questions for me I'll, let Christine speak for herself since just still here. Thank you Ken.

Christine Silverstein: Thank you, Kenan. As you mentioned, yes, effective at the end of the month is officially my resignation. I am staying on for a transitional period until the end of June to further oversee the transitional efforts here. And, as Joelle mentioned, I have accepted a nomination to the board and look forward to assisting in the guidance of Abeona Together. I care deeply about the team and the various stakeholders and look forward to the work ahead of us. We'll move next to Stacey Yang at Mizzou Ho. Hi, good morning, and thanks for taking my order. Just a couple here. The first one. I'm jolly if you could make it. To what extent, if the East Coast side doesn't get up and running, can you get away with it just?

As you mentioned, yes effective at the end and and as a monkey.

Officially the resignation.

I am saying on for transitional period until the end of June.

To further overseas.

The transitional efforts here.

And as Joel mentioned I have accepted the nomination to the board and look forward to assisting in the guy entity Yonah together.

My care deeply about the team and various stakeholders and look forward to that work ahead of us.

Well move next to Stacy Yang at Mizuho.

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

Just a couple here the first.

Uh huh.

You could make.

Okay.

Yes, the east coast side doesn't get up and running can you get away.

Just using yeah, the patients being treated from the Stendras side.

Stacey Yang: the patients being treated from the Stanford side and for registration purposes.

Registration.

Yes, so but thanks for your question.

Joelle Siffert: Yeah, so thanks for your question. So Stanford has the capabilities to enroll the full trial, for sure. And in fact, that could be the could have been the Overall plan, we felt it would be important to add another site, and eventually, we want to qualify additional sites, back to a question asked earlier, because at some point, we want to have multiple sites across the United States qualified to deploy this treatment once it's approved. So for phase III, we don't want to add too much distraction and too much variability because we're trying to essentially, as I see this particular trial, though it's a pivotal trial, it's ultimately a bridging trial for a product that has been shown to be efficacious now for three to five years post-treatment.

So the Stanford D has the capabilities to to enroll the full trial for sure.

Factors that could be the the could have been the.

The overall sort of the planned we felt that will be important too to add another site and eventually we want to qualify additional sites is enough back to a question asked earlier because at some point, we want to have multiple sites across the United States qualified to deploy this treatment once its approved so.

On a per the phase three we don't want to add too much.

But this distraction and too much variability because we're trying to essentially they see this broad. This this particular trial doses of pivotal trial. It is ultimately a bridging trial for trade for product that was shown to be efficacious now for three to five years post treatment and we're doing this trial now because we're manufacturing this product now.

Joelle Siffert: And we're doing this trial now because we're manufacturing this product now in compliance with Phase III and to be commercial quality. And obviously, the manufacturing site has transferred to Abeona. So for these reasons, we have to replicate the original trial, but the original trial already showed it works. So we don't want to make too many variations in the conduct of the trial because I think that could add noise and disruption to the system, but we felt that adding one more site, a qualified site, was prudent, both in terms of ability to enroll a little faster and start the process of equipping new sites, new centers, EB centers for treating EB-101 patients, sorry, treating RDEB patients with EB-101.

Well in compliance with the phase three and to be commercial.

Quality.

Obviously, the manufacturing side has transferred to have Yonah. So for these reasons, we have to replicate the original trial, but the original trial are you show. It works. So we don't want to make to too many very very abilities and the conduct the trial, because I think that could add noise and disruption of the system, but we felt that adding one more side to qualified side.

Was prudent that both in terms of ability to enrolled at a faster and start the process of equipping.

New sites, a new new centers, we'd be centers for.

For treating.

If you want to one patients have started to treating our debt patients will be one on one.

Okay. Thank you for that.

Stacey Yang: Okay, thank you for that. Then, looking at the MTS3A program, seems like Abeona has started the ABT003 trial for older patients, but also more cognitively challenged patients. Would you share your thoughts behind this trial and whether this trial would be required as part of the registration?

Then I'm looking at the end tier three a program seemed like at the owner.

Good.

T zero zero through each while for for older patients, but on the wall cognitive in each having a patient would you share your thoughts behind behind well and whether this trial would be required as part of the registration.

Yeah. So the second part of it the answer is no, though all safety data will be provided to the agencies and of course, the more safety data is relevant there is a.

Joelle Siffert: Yeah, so the second part of it, the answer is no, although all safety data will be provided to the agencies and, of course, more safety data is relevant. Sanfilippo obviously is a disease that causes a fair amount of disruption in neurodevelopment manifested both by cognitive delay at first and then cognitive decline, you know, usually starting in the first few years of life, in fact. In addition to that, patients with Sanfilippo have a significant amount of behavioral problems, which often can be the presenting symptoms. Also, sleep disturbances, some will develop seizures, so there's a whole constellation of other consequences of the disease process that also affect the patient's quality of life and overall quality of life in general.

San Felipe will obviously is the disease that causes the.

Fair amount of disruption in or development has manifested both the by cognitive delay a first and then cognitive decline you know usually starting.

In the first few years of life.

In addition to that patients with San Felipe will have a significant amount of behavioral problems, which often can be the presenting symptoms.

Also sleep disturbances some will develop seizure. So there's a whole constellation of other consequences of the disease process that has also affected patients. So a quality of life and overall sort of life in general. So we have this has been a longstanding commitment to the community that whereas for a cognitive.

Joelle Siffert: So we have made a long-standing commitment to the community that, whereas for cognitive assessment, the earlier you treat, the better the chances of impacting this disease before it causes more neurological damage. It is unclear, and therefore, this particular trial that you mentioned, it's unclear whether or not treatments like ABL-102 and gene therapy can help other aspects of the disease. That is, some of the behavioral manifestations, some of the sleep disturbances, some, they're often patients who describe, or families who describe their children as having sort of episodes of, it sounds like they are acutely in discomfort as if they were in pain, although it's hard to ascertain the nature of those, but these can be quite disruptive. So we've worked closely with the patient community and clinicians to devise assessments that are more tailored to that population who wouldn't qualify for the ABT-01 study but who still have significant morbidity from the disease.

Assessment of the earlier treat the better the chances of impacting this disease before because of a more neurological damage. It is unclear and therefore, the this particular trial the mentioned, it's unclear whether or not.

Weakness like KBR, one or two in gene therapy.

Can help other aspects of the disease that as some of the behavioral manifestation some of the sleep disturbances I'm some they're often.

A patients to describe those families. So described their children is having a sort of a episodes of the sounds like they are acutely in discomfort as if it was in pain, although it's hard to ascertain the nature of those but these can be quite disruptive. So we've worked closely with the patient community in the clinic.

Since to devise also assessments that are more tailored to two that population who wouldn't qualify for the.

A b T O one study, but who still have significant morbidity from the disease. So it's conceivable, although it's too early to tell the you know some of this intervention could potentially mitigate some of these.

Joelle Siffert: So it's conceivable, although it's too early to tell, that some of this intervention could potentially mitigate some of these other symptoms, whereas it's less likely that we would reverse the cognitive decline if they're too advanced. Again, we are not necessarily looking at the very advanced patients or any essentially eligible patients or those who are not eligible for the ABT-001 study. And just to recap, for patients coming into the ABT01 studies, abnormal. So if somebody has a developmental quotient of 50% of normal, which is not uncommon in a child who is, say, four years old or five years old, usually four, around four, they already have this much developmental delay; they would then be eligible for the ABT-003. Okay, thank you.

Other symptoms or whereas it's less likely that we would reverse the cognitive decline if there are two advance.

Again is there is not necessarily looking at the very advanced patients or any essentially eligible patients and those who are not eligible for the ABT. All one study in and just to recap the the.

Eligibility cut off for patients coming into the beat your one studies that they have a developmental quotient of at least 60%.

Of normal so if somebody has a governmental quotient of 50% of normal which you know it's not uncommon in the child, let's say four years older five years old usually for around for.

They already have this much a developmental delay.

These would then be eligible for the ABT all three.

Okay. Thank you Jill.

Stacey Yang: Okay, thank you, Joel. With no other questions remaining, that will conclude the portion of the Q&A for today's call. I'll turn the conference back to management for any additional or closing comments.

Well no other questions holding that will conclude the portion there for Q and aim for today's call I'll turn the conference back to management for any additional or closing comments.

Okay no. So when the thank everyone. Let me see here if I'm sure I have a.

Joelle Siffert: I want to thank everyone who is here. I'm sure I have some closing comments here, closing comments. But, really, just other than thank everyone for participating, and again, thank all of our collaborators and the patients who volunteer for these trials, and we'll continue to be committed, and we'll provide updates as we have them. Thank you. Thank you.

Some comments here closing comments.

But no no real just other than thank everyone for participating and again think Oliver collaborators and that the patients to volunteer for these trials and we're continuing to be committed and will provide updates as we have those.

Thank you.

Operator: Thank you, ladies and gentlemen. That will conclude today's call. We thank you for your participation. You may disconnect at this time, and have a great day.

Thank you, ladies and gentlemen that well conclude today's call. We thank you for your participation you may disconnect at this time and have a great Dane.

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