Q4 2020 Abeona Therapeutics Inc Earnings Call

Good day, and welcome to the ABL and the therapeutics fourth quarter and full year 2020 conference call.

At this time, all participants have been placed on a listen only mode and the floor will be open for your questions and comments after the presentation.

As a reminder, today's conference call is being recorded.

Operator: Welcome to the Abeona Therapeutics fourth quarter and full year 2020 conference call. At this time, all participants have been placed on a listen-only mode, and the floor will be open for your questions and comments after the presentation. As a reminder, today's conference call is being recorded. I'll now introduce you to today's conference host, Greg Gin, Vice President of Investor Relations and Corporate Communications at Abeona. Please go ahead.

I'll now introduce you to today's conference.

Greg Jensen, Vice President of Investor Relations, and corporate communications and I'd be honest please.

Please go ahead.

Thank you Holly.

Good morning, everyone.

And I'd like to welcome you and thank you for joining us on our fourth quarter and full year 2020 conference call.

Yesterday after the close of financial markets, we issued a press release announcing the fourth quarter and full year results and recent operational progress.

Gregory Gin: I'd like to welcome you and thank you for joining us on our fourth quarter and full year 2020 conference call. Yesterday, after the close of financial markets, we issued a press release announcing the fourth quarter and full year results and recent operational progress.

The press release is posted on our website at Www Dot maybe other therapeutics dot com.

On the call today with prepared remarks from Michael MRO, So chief Executive officer of <unk>.

And Ed Carr, Chief Accounting Officer.

Gregory Gin: The press release is posted on our website at www.abeonatherapeutics.com. On the call today with prepared remarks are Michael Amoroso, Chief Executive Officer of Abeona, and Ed Carr, Chief Accounting Officer. After the prepared remarks, we will host a Q&A session. And joining us for the Q&A today, we'll be joined by Jay Bercher, Chief Technical Officer, and Dr. Juan Ruiz, Vice President of European Medical Affairs at Abeona, who also heads up clinical development for our MPS program.

After the prepared remarks, we will host a Q&A session and.

And joining us for the Q&A today.

We'll be joined by Jay Bircher, Chief Technical Officer, and Dr. Juan Ruiz Vice President of the European Medical Affairs, and he'd be Ona and who also heads up clinical development at our for our NPS programs.

Before we start I will review, our safe Harbor statement.

Remarks made during today's call may contain projections and forward looking statements regarding future events.

Forward looking statements are made pursuant to the safe Harbor provisions of the federal Securities laws.

These forward looking statements are based on current expectations and are subject to change and actual results may differ materially from those expressed or implied in the forward looking statements.

Gregory Gin: Before we start, I will review our Safe Harbor Statement. Remarks made during today's call may contain projections and forward-looking statements regarding future events. Forward-looking statements are made pursuant to the safe harbor provisions of the federal securities laws. Such forward-looking statements are based on current expectations and are subject to change, and actual results may differ materially from those expressed or implied in the forward-looking statement. Various factors that could cause actual results to differ include, but are not limited to, those identified under the section entitled Risk Factors in the Company's Annual Report on Form 10-K and Quarterly Reports on Form 10-Q, filed by the company with the SEC. These documents are available on our website at www.abeonatherapeutics.com. And with that, I will now turn the call over to Michael.

Various factors that could cause actual results to differ include but are not limited to those identified under the section entitled risk factors and the company's annual report on form 10-K, and quarterly reports on form 10-Q for.

And by the company with the SEC.

These documents are available on our website at www Dot maybe other therapeutics dot com and.

With that I will now turn the call over to Michael Michael.

Thank you, Greg and good morning to our Investor community. It's my pleasure to be with you again today for our Q4 and full year call.

We're excited and Navy O&M and developing a novel and gene and cell therapies for underserved patient populations and we remained focused on execution 2021 is the year of execution and delivery.

Gregory Gin: I'll turn the call over to Michael. Okay?

Michael Amoroso: Thank you, Greg, and good morning to our investor community. It's my pleasure to be with you again today for our Q4 and full year call.

On today's call I'll start with a brief review of some of our key accomplishments over the past year 2020 and all.

Michael Amoroso: We're excited at Abeona, developing our novel one-gene cell therapies for underserved patient populations, and we remain focused on execution. 2021 is the year of execution and delivery. On today's call, I'll start with a brief review of some of our key accomplishments over the past year, 2020, and I'll talk to you about our Fast Start 2021. For 2021, I'm very proud of the progress we have made, despite the macro disruptions caused by the pandemic.

I'll talk to you about our fast start and 2021.

In 2020, one and I'm very proud of the team.

Despite the macro disruptions caused by the pandemic, we continue to advance all three clinical programs towards bringing urgent need of treatments to patients with recessive dystrophic epidermolysis blows them as al referred to as our day about I'll call our lead program.

As well as sanfilippo syndrome type a and b.

Michael Amoroso: We continue to advance all three clinical programs toward bringing urgently needed treatments to patients with recessive dystrophic epidermolysis bullosa, as I'll refer to as RDEV on our call, our lead program, as well as Sanfilippo Syndrome Types A and B, known to you as MPS 3A and 3B respectively. First, let's start with our lead Pivotal Program, BB101, and look at Earlier in the year, we initiated our Pivotal Phase III Vital Study for EB-101, as you'll recall, in RDEV at Stanford University, a world-renowned EB treatment center.

Referred to as MTS, III and <unk>, respectively.

First let's start with our lead pivotal program <unk> 101.

And look and a brief overview of 2020.

Earlier in the year, we initiated our pivotal phase III <unk> study for <unk> hundred one as you'll remember and Ardeb at Stanford University, a world renowned <unk> treatment center.

In July of 'twenty, we presented our phase one two data at the society for pediatric dermatology share.

And that he'd be one and one treatment of large and chronic wounds resulted in substantial and durable wound healing for patients.

Michael Amoroso: In July 20, we presented our Phase 1-2 data at the Society for Pediatric Dermatology, showing that EB-101 treatment of large and chronic wounds resulted in substantial and durable wound healing for patients, also associated with long-term improvement in pain relief for up to five years. As a reminder, large wounds were characterized in our trial as 20 centimeters or greater, some going up to 100 plus centimeters, and 200 centimeter wound And chronic wounds, wounds that are open for six months and longer and can no longer heal themselves, like recurrent blistering wounds, start in EB or RDEB.

Also associated with long term improvement and pain relief for up to five years.

As a reminder.

Large wounds characterized and our trial is 20 centimeters or greater some going up to a 100 plus centimeters 200 and centimeter balloons and.

And chronic wounds wounds that are opened and six months and longer and can no longer heal themselves like recurrent blister and wound start and EV or our debt.

These are the most problematic wounds for patients suffering from par debt.

They lead to significant morbidity issues, namely severe pain opioid use hours per day of dressing change.

And ultimately and most unfortunately, they lead to most patient demise by 30% to 40 years of age.

Michael Amoroso: These are the most problematic wounds for patients suffering from ARDEP. They lead to significant morbidity issues, namely severe pain, opioid use, and hours of dressing change per day. And ultimately, and most unfortunately, they lead to most patient demise by 30 to 40 years of age.

This phase one two data was exciting and validating for us.

Also generated with Stanford University and serves as the proof of concept for our pivotal vital study.

Next.

After accruing patients two and three and vital and the late summer.

Michael Amoroso: This Phase 1-2 data was exciting and validating for us, also generated with Stanford University as it served as the proof of concept for our pivotal vital study. After accruing patients two and three in vital in the late summer, In December of 2020, as we've previously reported, we met for a formal type B meeting with the FDA. The result of the meeting was very positive.

In December of 2020, as we've previously reported we met with a formal type b meeting with the FDA.

The result of the meeting was very positive our interaction resulted and shared alignment with the FDA.

And co primary endpoints for the vital pivotal trial.

Michael Amoroso: Our interaction resulted in shared alignment with the FDA on co-primary endpoints for the Vital Pivotal Trial. As a reminder, the endpoints are the proportion of RDEV wound sites with greater than 50% wound healing from baseline, comparing treated and untreated wounds at week 24 or month 6. Determined by direct investigator assessment, and the second co-primary endpoint and Pain Reduction Associated with Wound Dressing Change, Assessed by Mean Difference in Scores Using the Wong-Baker Validated FACES Scale, Comparing Treated and Untreated Wounds, also at Week 24 of Month 6.

Determined by direct investigator assessment.

And the second co primary endpoint and.

Pain reduction associated with wound dressing change assessed by mean difference and scores using the one Baker validated thesis scale, comparing treated and untreated wounds and also at week 24 month six.

This was a very important step forward clarity with the FDA.

We believe these are very appropriate and clinically meaningful endpoints and we thank the FDA for their partnership and helping US advance the phase III pivotal vital study one step closer to patients.

Michael Amoroso: This was a very important step forward because of clarity with the FDA. We believe these are very appropriate and clinically meaningful endpoints, and we thank the FDA for their partnership in helping us advance the Phase III Pivotal Vital Study one step closer to patients. Next, immediately upon alignment with the FDA and protocol amendment in January 21, I am very excited to let you know that the fourth patient was identified, biopsied, and treated in the vital study in February, as enrollment is ongoing and remains our top priority at Abeona for 2021.

Next.

Immediately upon alignment with the FDA and protocol Amendment in January of 'twenty one.

I'm very excited to let you know that the fourth patient was identified biopsy and treated and the vital study in February.

Excuse me.

In addition, as.

As enrollment is ongoing and remains our top priority at <unk> for 2021.

We are very pleased to report that patient five was biopsied earlier this week and we anticipate.

And they're surgical transplantation at Stanford and mid April.

Finally, with regard to patient accrual our top priority and.

Michael Amoroso: We are very pleased to report that patient 5 was biopsied earlier this week, and we anticipate their surgical transplantation at Stanford in mid-April. Finally, with regard to patient accrual, our top priority, additional potential patients have been identified.

Additional potential patients have been identified.

Theyre being prescreened to determine their eligibility for study participation as.

As a reminder, the per.

<unk> screening is a battery of tests, but most importantly, the genetic screening to ensure they meet the criteria of two confirmed C. Seven mutations both with recessive inherited patterns, causing our debt.

Michael Amoroso: They're being pre-screened to determine their eligibility for study participation. As a reminder, the prescreening includes a battery of tests, but most importantly, genetic screening to ensure they meet the criteria of two confirmed C7 mutations, both with recessive inheritance patterns causing RDAP.

Overall, we are very pleased with the progress against key milestones for the <unk> program our lead program.

The team's focus on operational excellence and laser focus, including Stanford during a very difficult time.

And we are on our way to meet our accrual goals for 2021.

Michael Amoroso: Overall, we are very pleased with the progress against key milestones for the EB program, our lead program. The teams focused on operational excellence and laser focus, including Stanford, during a very difficult time, and we are on our way to meeting our accrual goals for 2021. Please recall that for the Vital Pivotal Trial, target enrollment is anywhere between 10 to 15 patients. It is not a patient number derived, but more importantly, it is the treatment of approximately 35 large chronic wounds in total.

Please recall that for the vital pivotal trials target enrollment is anywhere between 10 to 15 patients. It is not patient number derived but more importantly, it is the treatment of approximately 35 large chronic wounds and total.

We believe the recent momentum.

Our type B meeting.

Patients for being treated and patient five now enrolled and on their way to treatment and Q1.

Has us well on our way to complete our goal of accrual in 2021.

Last for E B, one O one and even more exciting news due.

Michael Amoroso: We believe the recent momentum..., of our Type B meeting, with Patients 4 being treated, and Patients 5 now enrolled and on their way to treatment in Q1, has us well on our way to complete our goal of accrual in 2021. Last, for EB101, and even more exciting news... Due to an increased interest from our KOL community, from patients, as the pandemic seems to hopefully slow, and vaccines start to take hold across the world.

Due to an increased interest from our Kols community from patients as the pandemic seems to hopefully slope and vaccine and start to take hold across the world.

We have selected a large academic medical center in the northeast that we are currently Onboarding are site number two.

And they are still going through the IRB approval, and we hope to have them up and running for the second half of this year and the second half of the accrual of vital.

Michael Amoroso: We have selected a large academic EB medical center in the Northeast that we are currently onboarding as site number two. As they are still going through their IRB approval, we hope to have them up and running for the second half of this year and the second half of Accrual of Vital. I will not release their name just yet because I want to make sure they get IRB approval, but we'll be speaking to you soon about that.

I will not released their name just yet.

Because I want to make sure they get the IRB approval, but we will be speaking to you soon about that.

Even though as Ive stated before we are on pace to finish vital and 2021 out of Stanford we.

We agreed to a second site for two main reasons first list.

Listening to our patients.

The number one barrier and 2020.

And for study participation was traveling due to COVID-19.

Michael Amoroso: Even though, as I've stated before, we are on pace to finish vital in 2021 out of spam, we agreed to a second site for two main reasons. First, listening to our patients, the number one barrier to study participation in 2020 was traveling due to COVID. We wanted to offer an option on the East Coast to make travel and logistics easier for some patients and families.

We wanted to offer and option on the east coast to make travel and logistics easier for some patients and families.

Second.

As we get closer to potentially commercializing <unk> 101, hopefully in 2022 pending positive results.

Expanding HCP experience would you be one and one is necessary and meeting our goal that's how the E. B one and one is the standard of care.

Michael Amoroso: Second, as we get closer to potentially commercializing EB-101, hopefully in 2022, pending positive results, expanding HCP experience with EB-101 is necessary in meeting our goal of having EB-101 as the standard of care for all patients with recessive dystrophic epidermolysis bullosa in the United States. Now I'll turn my attention to

For all patients with recessive dystrophic, epidermolysis blow through and the United States.

Okay.

Now I'll turn my attention.

The other half of our gene therapy portfolio or add no associated virus or AAV based gene therapy programs, namely <unk>.

Our first program from an AAP standpoint, as <unk> 102 program known as <unk>.

As a reminder, we completed target enrollment and ABL 100 to the transfer a study for MPS <unk> and the fourth quarter of 2020 as was laid out and our corporate goals and milestones for 2020.

Michael Amoroso: The other half of our gene therapy portfolio is adeno-associated virus (AAV)-based gene therapy programs. Namely, our first program from an AAV standpoint is AB102, our program known as MPS3A. As a reminder, we completed target enrollment in ABO 102, the Transfer A Study, for MPS 3A in the fourth quarter of 2020, as laid out in our Corporate Goals and Milestones for 2015. To date, 19 patients have been dosed in the TRANSFER-A study, including 13 patients dosed in the higher dose cohort, 3 times 10 to the 13 vector genomes per kilogram, the dose that we believe is safe and efficacious and we'll be speaking to the FDA about later this year.

To date 19 patients had been dosed and the transfer a study, including 13 patients dose and the higher dose cohort three three times 10 to the 13 vector genomes per kilogram dose that we believe is safe and efficacious and we'll be speaking to the FDA about later this year.

We also communicated and our third quarter call that we will continue to lead the transfer a study open to patient enrollment at least through our type B meeting with the FDA for two main reasons.

And the lack of treatment options for patients afflicted with NPS per day.

Michael Amoroso: We also communicated in our third-quarter call that we'll continue to leave the Transfer A study open to patient enrollment, at least through our Type B meeting with the FDA, for two main reasons. One, the lack of treatment options for patients afflicted with MPS 3A.

True.

Based on the positive safety and efficacy results, we've seen from the higher dose cohort. We thought it was the right thing to do to leave it open for patients.

In addition, and just last month.

We were very excited to share the latest interim data from transfer rate.

Michael Amoroso: Two, based on the positive safety and efficacy results we've seen from the higher dose cohort, we thought it was the right thing to do to leave it open for patients. In addition, and just last month... We were very excited to share the latest interim data from TRANSRIC, which continues to provide hope for patients with MPS 3A as well as their families. The updated results, which were presented during a late-breaking platform oral session at the World Symposium and again a few days later with many of you at our subsequent investor webinar, continue to suggest that ABL-102 has the potential to be a life-altering treatment option for children with MPS 3A.

Which continues to provide hope for patients with <unk> as well as their families.

The updated results, which were presented during the late breaking platform for oral session at the World Symposium and again a few days later with many of you at our subsequent Investor Webinar.

Continue to suggest that ABL, one or two has the potential to be a life altering true.

<unk> option for children with MTS III.

The data from transfer rate demonstrated that the neuro cognitive development was preserved within normal range of non afflicted children.

And a half years, two three years post treatment with <unk> 102 cohort three dose and the three youngest patients treated.

Michael Amoroso: The data from TRANSFER-A demonstrated that neurocognitive development was preserved within the normal range of non-afflicted children, two and a half years to three years post-treatment with ABL-102 Cohort 3 dose in the three youngest patients treated. These patients, as a reminder, were treated at ages 27 months, 19 months, and 12 months, respectively, and they are now at the age ranges of three and a half to five plus years

These patients as a reminder were treated and ages 27 months 19 months and 12 months, respectively and they are now at the age ranges of three and a half to five plus years chronologically.

Why this is so important.

Their current age three and five to five plus.

Is a critical time point as we see the natural history of NPS three a disease, usually by about three years neuro cognitive development declines.

Michael Amoroso: Why this is so important. Their current age, three and a half to five plus, is a critical time point as we see the natural history of MPS3A disease, usually by about three years, neurocognitive development declines without any therapeutic intervention with none being approved thus far today. We are very excited about this data, and our next steps are as follows. We requested a meeting, as we told you we would, with the FDA on January 21, 21, to discuss this data and next steps for a potential BLA path with the FDA.

Without.

The therapeutic intervention, which non being approved thus far today.

We're very excited about this data and our next steps are as follows.

We requested a meeting as we told you we would with the FDA and January of 'twenty one to.

To discuss this data and next steps for a potential BLA path with the FDA.

A meeting date has been set in June we appreciate that from the FDA as they are quite inundated with COVID-19.

We intend to discuss with the FDA, whether the transfer a dataset could serve as the backbone for BLA submission with.

With natural history data as a viable control arm.

Michael Amoroso: A meeting date has been set for June; we appreciate that from the FDA as they're quite inundated with COVID. We intend to discuss with the FDA whether the Transfer A dataset could serve as the backbone for a BLA submission, with Natural History Data as a Viable Controller. We believe this is most important for any development in NPS disease for all life science companies, as these children are being demented by the day, and it is not feasible to give them a placebo.

We believe this is most important for any development and NPS disease for all life Science companies. As these children are being demanded by the day and it is not feasible to give them a placebo.

We're excited and we're hopeful to review the data with the FDA at our upcoming meeting and we believe we have a viable plan forward.

Also a reminder, in mid 2020, we met with the EMA under the prime designation to discuss a viable path toward a marketing authorization in Europe for ABL of 102.

Michael Amoroso: We're excited and hopeful to review the data with the FDA at our upcoming meeting, and we believe we have a viable plan forward. Also, a reminder, in mid-2020, we met with the EMA under the Prime designation to discuss a viable path toward a marketing authorization in Europe for ABO 102. We expect the next steps from our FDA interaction in June to filter into our development plans, and we will again further refine our pathway with the EMA and have a discussion with them, probably later in the second half of this year. Moving on to our third in-clinic program, Transfer B. I'm sorry, 11 patients transferred into MPS 3B. 11 patients have been dosed to date, including 4 in the higher dose cohort.

We expect and next steps from our FDA interaction and June.

And to filter into our development plans and we will again further refine our pathway with the EMA and have a discussion with them probably targeted later in the second half of this year.

Moving on to our third and clinic program transfer B <unk>.

11 patients I'm, sorry transfer be in MPS <unk>.

11 patients have been dosed to date, including for in the higher dose cohort.

Minder here the higher dose cohort here is one times 10 to the 14th.

In February of 'twenty, one also at the World Symposium.

New interim data was accepted as late breaking oral presentation for transfer B study.

Michael Amoroso: A reminder here, the higher dose cohort here is 1 times 10 to the 14th, February 21, also at the World Symposium. New interim data was accepted as a late-breaking oral presentation for the Transfer B Study. The updated results were encouraging. They showed treatment with ABL 101 was associated with a dose-dependent and sustained improvement in CNS, cerebral spinal fluid heparin sulfate, and systemic biomarkers, including plasma and urine heparin sulfate and Urine Glycosaminoglycans, known as GAG.

The updated results were encouraging and they showed treatment with <unk> 101 was associated with a dose dependent and sustained improvement and CNS.

Cerebrospinal fluid heparin sulfate and.

And systemic biomarkers, including plasma and urine <unk> sulfate.

And urine like.

Glycosaminoglycan known as gags.

We look forward to continued follow up to assess <unk> hundred one's potential to preserve neuro cognitive development, the gold standard and need for MTS patients.

And <unk> later this year and also onward into 2022 as the data for neuro cognitive assessments matures.

Michael Amoroso: We look forward to continued follow-up to assess ABO 101's potential to preserve neurocognitive development, the gold standard and need for MPS patients, in 3B later this year and also onward into 2022 as the data for neurocognitive assessments matures. Finally, with regard to Transfer B, we will not be concluding patient accrual in Q1, as previously stated on our last quarterly call. We're currently working on the next product stability testing time point for the clinical product being used in Transfer B, which, as I'll remind you, is the Nationwide Children's Hospital product.

Finally with regard to transfer B we.

We will not be concluding patient accrual in Q1 as prior stated on our last quarterly call.

We're currently working on the next product stability testing time point for the clinical product being used and transfer B, which as I'll remind you is the nationwide children's hospital product.

After completion of this testing, we will assess our options forward to potentially treat the remaining patients and meet.

We plan to update you the investment community as soon as we have clarity on timelines and after we have discussed with our investigators patients and the NPS community.

Michael Amoroso: After completion of this testing, we will assess our options forward to potentially treat the remaining patients in need. We have planned to update you, the investment community, as soon as we have clarity on timelines and after we have discussed our findings with our investigators, patients, and the MPS community. Next, turning to overall corporate and organizational progress in 2020, we entered into two strategic partnerships with Tayshia Gene Therapies that unlock near-term value in earlier-stage non-core assets for Abeona and provide us with an opportunity to share in the future success and milestones of these programs.

Next turning to overall corporate organizational progress and 20, we entered into two strategic partnerships with patient gene therapies that unlock near term value and earliest stage non core assets for avionics and provide us opportunity to share and the future success and milestones of these programs.

The deals provided tissue gene therapies, the rights to <unk> hundred two and AAV based gene therapy for CLR, one disease or infantile batten disease.

As well as certain IP rights material and Knowhow related to a potential AAV based gene therapy for Ret syndrome.

Michael Amoroso: The deals provide Tayshia Gene Therapy with the rights to AB0202 and AAV-based gene therapy for CLN1 disease or Infantile Batten Disease, as well as certain IP rights, material, and know-how relating to a potential AAV-based gene therapy for Rett syndrome.

Most importantly, this deal this.

And this transaction put this asset and the hands of a partner that will expedite development much needed for these patients with again the highest of unmet need.

Next as we look to add to our leadership.

Michael Amoroso: Most importantly, this transaction put this asset in the hands of a partner that will expedite development, much needed for these patients with, again, the highest of unmet needs. Next, as we look to add to our leadership and Operational Experience in the next phase of Abeona, our Operational Excellence phase. I am very excited to announce that this morning we appointed two new independent members to our Board of Directors, further strengthening our leadership and helping us to drive our path forward for future growth.

And operational experience and the next phase of our operational excellence phase.

I am very excited to announce that this morning, we appointed two new independent members to our board of directors for.

Other strengthening our leadership.

Helping us drive our path forward for future growth.

Enhancing our corporate governance.

And assisting and creating additional shareholder value.

I want to comment on these board members and their selection.

It was based on their relevant and diverse experience, making them. The right partners for my current management team oversight for Boe.

Michael Amoroso: Enhancing our Corporate Governance and assisting in creating additional shareholder value. I want to comment on these board members and their selection. It was based on their relevant and diverse experience, making them the right partners for my current management team oversight for both near and longer-term objectives of Abeona. Specifically, operational experiences and lifestyles.

Both near and longer term objectives with a b on it.

Specifically operational experiences and life science.

Finally, let's review the key anticipated milestones for 2021, and what you can expect from us.

The accomplishments over the past year and into a fast start Q1 2021.

Give us very positive momentum as we focus on execution and continue to move our programs in clinic closer to patients.

Michael Amoroso: Finally, let's review the key anticipated milestones for 2021 and what you can expect from us. The accomplishments over the past year and into our FAST-START Q1 2021 give us very positive momentum as we focus on execution and continue to move our programs in clinic closer to patients. Number one, in 2021, we plan to complete enrollment in the EB-101 Phase III Pivotal Vital Study. Assuming no more COVID-related delays, and if you could see me, my fingers are crossed, based on the current expectation for completing patient enrollment in 21.

Number one and 2021, we plan to complete enrollment and the EB 101 phase III pivotal vital study.

Assuming no more COVID-19 related delays and if you could see me my fingers are crossed.

Based on the current expectation for completing patient enrollment and 21.

We anticipate I'm, sorry, we anticipate top line data for the vital study will be available in mid 2022.

Which will be followed immediately by a BLA filing assuming positive results.

Milestone two regarding our program and MTS and <unk> ABL one O two.

Michael Amoroso: We anticipate, I'm sorry, we anticipate top-line data for the vital study will be available in mid-2022, which will be followed immediately by a BLA filing assuming positive results. Milestone 2 regarding our program in MPS 3A AB0102. We have already put a check in the box for our first objective.

We have already put a checking the box of our first objective.

Assuring a type b meeting with the FDA to discuss our path forward as a reminder, that we'll be in June of 'twenty one.

In addition, we expect the first lot of ABL and are produced clinical great product for <unk> 102 to be released and the second half of this year.

Michael Amoroso: Assuring a Type B meeting with the FDA to discuss our path forward. As a reminder, that will be on June 21, 2017. In addition, we expect the first lot of Abeona-produced clinical-grade product for ABL-102 to be released in the second half of this year. This is a very exciting milestone for our group. Under Jay Birch's leadership and our Tech Ops group in Cleveland, Ohio, I often say we believe this is the lifeblood of our organization.

Three exciting milestone for our group.

Under <unk> leadership, and our Tech Ops group and Cleveland, Ohio, I, often say we believe this is the lifeblood of our organization.

Both the patients the younger patients who've been treated and cohort three the first three patients as well as patients for five and six.

Michael Amoroso: Finally, and later this year, we expect additional follow-up visits and neurocognitive assessments of both the patients, the younger patients who have been treated in Cohort 3, the first three patients, as well as patients 4, 5, and 6. As a reminder, in Cohort 3, we have dosed up to 8 patients, and the 2-year neurocognitive assessment is a very significant time point because these patients will be chronologically past that 3-year time point where we can compare to the natural history of the progression of the disease in an untreated patient. We'll let you know more about our exact data dissemination plan when we're clear on exactly what Congress will target.

As a reminder, and cohort three we have dosed up to eight patients and two year neuro cognitive assessment is a very significant time point because these patients will be chronologically past that three year time point, where we can compare to the natural history of the progression of the disease and and untreated patients.

We'll let you know more about our exact data dissemination plan when we're clear on exactly what Congresses will target.

Number three rigs.

Regarding our last in clinic program <unk> hundred one and MPS <unk>.

We will work timely through our product stability testing time points for clinical product from nationwide.

Once we have the completion of our stability test, we'll assess our options for treating the patients remaining from transfer B.

Michael Amoroso: Number three, regarding our last in-clinic program, EBO 101 and MPS 3D. We will work timely through our product stability testing time points for clinical products from nationwide. Once we have the completion of our stability tests, we'll assess our options for treating the patients remaining from Transfer B. Also, it's important to note that the FDA feedback that we will receive for MPS 3A in June will be a significant learning for the path forward for all MPS programs and will absolutely filter immediately into our MPS 3B plans as well as will continue to guide our EMA plans for both 3A and 3B.

Also it's important to note.

And that the FDA feedback that we will receive for NPS three eight program in June will be a significant learning for the path forward for all MTS programs, and we will absolutely filter immediately into our MTS and <unk> plans as well as we will continue to guide our MA plans for both <unk> and Threep.

Finally, we can expect to see additional clinical data and updates from transfer be later this year. Some early neuro cognitive data.

And we will let you know as our Congresses are designated when that data dissemination will occur.

Let's now shift briefly to our work on adding new in clinic programs from our rich preclinical pipeline.

Michael Amoroso: Finally, we can expect to see additional clinical data and updates from Transfer B later this year, including some early neurocognitive data, and we will let you know as our congresses are designated when that data dissemination will occur. Let's now shift briefly to our work on adding new in-clinic programs from our rich preclinical pipeline. We're focusing resources, and we have started to begin to prioritize our ophthalmic indications following a strategic prioritization and review.

We're focusing resources and we have started to begin to prioritize our ophthalmic indications following a strategic prioritization and review.

On the back of the progress from our AAV programs and MPS <unk> and <unk>.

We're conducting preclinical research assessing some of our in house and partnered AAV capsid and six undisclosed eye disorders.

We are executing towards IND, enabling studies in two to three indications and 'twenty two will take two to three of the six.

Michael Amoroso: On the back of the progress from our AAV programs in MPS 3A and 3B, we're conducting preclinical research assessing some of our in-house and partnered AAV capsids in six undisclosed eye disorders. We are executing toward IMD-enabling studies in 2-3 indications in 22. We'll pick 2-3 of the 6.

Just to give you an idea while these indications are undisclosed right now as these are very very busy spaces and I want to make sure. We have more concrete pads forward each of the indications and the U S alone represented about five to 15000 patients. So we're starting to parlay, our success and AAV programs to some bigger patient markets with tremendous.

Michael Amoroso: Just to give you an idea, while these indications are undisclosed right now, as these are very, very busy spaces, and I want to make sure we have more concrete paths forward, each of the indications in the U.S. alone represents about 5,000 to 15,000 patients. So we're starting to parlay our success in AAV programs into some bigger patient markets with tremendous need. We expect to have results from some non-human primate studies in Q2-Q3-21, and we plan to examine these results and have them guide us into proof of concept studies in HH2 of this year. Ultimately, these will lead us into pre-IND meetings and toxicology studies in 2022.

Need.

We expect that results from some non human primate studies.

In Q2, Q3 'twenty one.

And we plan to examine these results and have them guide us into proof of concept studies and the <unk> each two of this year.

Ultimately these will lead us into pre IND meetings, and toxicology studies and 2022.

I. Thank you for your time today, I look forward to a substantive conversation and our Q&A and before that I'm going to turn it over to our Chief Accounting Officer, My right hand and car. So he can take you through the full year financial results and please take it away.

Thank you, Michael and I would like to remind everyone that we have recently filed the form 10-K, which is available on our website and where you can get the details on our financial results.

Michael Amoroso: Thank you for your time today. I look forward to a substantive conversation in our Q&A, and before that, I'm going to turn it over to our Chief Accounting Officer, my right-hand man, Ed Carr, so he can take you through the full year financial results. Ed, please take it away.

In summary, our cash cash equivalents and short term investments as of December 31, 2020 totaled $95 million compared to $129 3 million as of December 31, 2019, we did not use our $150 million at the market or ATM program during the fourth quarter of 2020.

Ed Carr: I would like to remind everyone that we have recently filed the Form 10-K, which is available on our website and where you can get the details on our financial results. In summary, our cash, cash equivalents, and short-term investments as of December 31, 2020, totaled $95 million compared to $127.3 million as of December 31, 2009. We did not use our $150 million at-the-market, or ATM, program during the fourth quarter of 2020. We believe we have sufficient cash resources to build on our momentum and fund our current development and operating plan for the achievement of key anticipated milestones, including multiple potential regulatory solutions.

And we believe we have sufficient cash resources to build on our own Mentum and fund our current development and operating plan for the achievement of key anticipated milestones, including multiple potential regulatory submissions for.

For the full year of 2020 net cash used in operating activities was $35 million compared to $62 8 million for the full year of 2019.

And with that I will now turn it over to the operator for Q&A colleague.

Ali.

Ed Carr: For the full year of 2020, net cash used in operating activities was $35 million, compared to $62.8 million for the full year of 2019. And with that, I will now turn it over to the operator for Q&A. Holly?

Ladies and gentlemen for now open for questions. If you have any questions or comments. Please press star one on your phone at this time.

Net.

And to provide optimum sample quality please call us.

Operator: Ladies and gentlemen, the floor is now open for questions. If you have any questions or comments, please press star 1 on your phone at this time. We ask that while posing your question, you please pick up your handset if listening on speakerphone to provide optimum sound quality. Please hold while we poll for questions. Your first question for today is from Maurice Raycroft. Please announce your affiliation, then pose your question.

Our questions.

Your first question for today is coming from Maury Raycroft. Please announce your affiliation and pose your question.

Hi, good.

Good morning, everyone. Congrats on the progress and thanks for taking my questions. This is morry from Jefferies.

First question is just try and NPS and <unk>. So just wondering if you can book and how long and we will take to do the product stability testing and wondering if that was requested by FDA or was it something observed with a product or was it just the right time to do this stability.

Maurice Thomas Raycroft: Hi, good morning everyone. Congratulations on the progress and thanks for taking the time to answer my questions. This is Maury from Jeffreys. The first question is just on MPS 3b. So just wondering if you can estimate how long it will take to do the product stability testing and wondering if that was requested by FDA. Was there something observed with the product, or was it just the right time to do the stability testing? Yeah, Maury, I'll take that one.

Yes, Maury I'll take that one and if we need any any smart science talk I'll definitely turn it over to Jay.

No <unk> as planned.

Nationwide product has expiry on it so we have to make sure that we're doing our stability planning. This has not been asked for by the regulators specifically, but of course, it's part of our plans and part of our stability protocols and our IND. So.

Michael Amoroso: And if we need any smart science talk, I'll definitely turn it over to Jay. No, 3B as planned, you know; nationwide product has an expiry date on it. So we have to make sure that we're doing our stability planning. This has not been asked for by the regulators, specifically, but of course, it's part of our plans, part of our stability protocols, and our INDs. So we, the nationwide product, last lot late last year hit expiry.

The nationwide product last lot late last year hit ex <unk>. So what we're doing is we're testing stability and making sure. Our product is still has the appropriate integrity and we're looking at some final patients for transfer book. So that's the impetus for that as far as timing, that's more imminent and long term I didn't put timing out exactly today, because I don't want to have to.

Michael Amoroso: So what we're doing is we're testing stability, making sure our product still has the appropriate integrity, and we're looking at some final patients for transfer B. So that's the impetus for that. As far as timing is concerned, that's more imminent than long term. I didn't put the timing out exactly today because I don't want to have to take it back.

It back from the Mowry right now we're looking at some tests in house versus out and we should have some timelines very very soon here Jay is working quickly on that because we know we've got some patients waiting so I.

And I predict that will be more imminent than longer term, but as soon as we know we'll update you.

Michael Amoroso: Me, Maury, right now, we're looking at some tests in-house versus out, and we should have some timelines very, very soon here. Jay's working quickly on that because we know we've got some patients waiting. So I predict that it will be more imminent than long term. But as soon as we know, we'll update you.

Got it very helpful. Thank you and then second question is just checking for EV 101, if that second side comes onboard second half <unk>.

That could adding that site accelerate timelines and.

And would any patients or data from that site be included and the filing submission or do you want and all the data to come from Sanford I guess, how are you guys thinking about that.

Michael Amoroso: Thank you. And then the second question is just checking for EB-101. If that second site comes on board, second half, could adding that site accelerate the timeline some? And would any patients or data from that site be included in the filing submission?

Yeah, Great question so.

Absolutely on both the <unk>.

<unk> is as we've said all along.

Really excited we're almost I don't want to say it out loud right and I'm, a superstitious guy, but we almost and quarter one.

Michael Amoroso: Or do you want all the data to come from Stanford? I guess, how are you guys thinking about that? Yeah, Mark, great question.

There's many patients onboard as we've had and all of 2020 and to be fair to the team. We know 'twenty was a tough year with the pandemic. So we think we can finish out and Stanford remember we could be done as early as 10 patients.

Michael Amoroso: So, absolutely on both. The answer is, as we've said all along, really excited. We're almost at, you know, I don't want to say it out loud, right? I'm a superstitious guy.

And we'll think about leaving it open a little bit if theres someone sitting on the bench and we wanted to get to all patients in need.

Michael Amoroso: But we almost have as many patients on board in quarter one as we have in all of 2020. And to be fair to the team, we know 20 was a tough year with the pandemic. So we think we can finish out of Stanford. Remember, we could be done as early as 10 patients. And we'll think about leaving it open a little bit if there's someone sitting on the bench. We want to get to all the patients in need. You know, right now, we think we're projecting somewhere.

Right now we think we're projecting somewhere it depends on how many wounds we get per patient. So I won't project right away, but we could be done as early as 10 patients. So five takes us halfway there no doubt that the second site, which I'll announce as soon as debt through IRB for disclosure purposes of University.

That will get us.

Michael Amoroso: It depends on how many wounds we get per patient, so I won't project it right away. But we could be done as early as 10 patients, so five takes us halfway there. No doubt that the second site, which I'll announce as soon as they're through IRB for, you know, disclosure purposes of the university, that will definitely expedite time to fulfill accrual. And yes, those results will be as important as part of the BLA filing. Any patient under vital treatment who's part of vital will be part of the BLA.

Definitely expedite time to fulfill accrual and yes, those results will be as important and part of the BLA filing and patient under vital treated theyre part of vital will be part of the BLA.

Correct.

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

The next question is coming from Ram Silverado to please announce your affiliation and pose your question.

Michael Amoroso: All correct. Got it. Okay. Thank you very much for taking my questions. I'll hop back in the queue.

Hello. This is Ron Silverado from H C. Wainwright can you hear me.

Yeah, Rob we got Ya.

Operator: Your next question is coming from Ram Selvaraju. Please announce your affiliation, then ask your question.

So thank you very much for taking my questions Firstly related.

<unk> hundred one program I was wondering if you could confirm that you are effectively going to stop enrollment.

Ron Selvaraju: Hello, this is Ron Silveragio from HB Wainwright. Can you hear me?

And you have.

Michael Amoroso: Yeah, Ron, we got you. Thank you. So, thank you very much for taking my questions, but firstly, relating to the ED101 program, I was wondering if you could confirm that you are effectively going to stop enrollment once you have, Unknown Attendee, Gregory Gin, Vishwas Seshadri, Abeona Therapeutics Inc, you would halt enrollment at that time, or if there's a possibility that you could continue to enroll additional patients after hitting that number of... Yep, I'll take that one.

35, chronic wounds to whatsapp and.

Other words, it's not specifically dependent on patient number and once you have 35 assessable chronic wounds.

And you would health enrollment at that time or if there is a possibility that you could continue to enroll additional patients after hitting.

Hitting that number.

Yes, I'll take that one Rob great question. So the answer is and we use the word approximate for a reason 35 wounds important for our stats plan right and as part of our pivotal so yes, that's the target give or take probably and the direction.

We've got a hit 35, but we could go a little more what we want to be mindful around is to make sure. If there is and I have the patient and the queue, we're not going to refuse them, but it is you're exactly right. It's not a specific number of patients. The stats plan is based on number of wounds. So that's why we have a range of patients because knowing what our what our readers will be able to get there between 10 and.

Michael Amoroso: Rob, great question. So the answer is, and we use the word approximate for a reason, 35 wounds important for our stats plan, right, as part of our pivotal. So yes, that's the target, give or take, probably in the right direction. We think we've got to hit 35, but we could go a little more. What we want to be mindful of, Rob, is to make sure that if there's another patient in the queue, we're not going to refuse them.

And <unk>, so you're right. It is 35 wounds, it's approximate I won't say will definitively stop at 35. It depends if we have a patient and the Q, but we won't do 50 for example will be close to that number.

Michael Amoroso: But it is, you're exactly right, it's not a specific number of patients. The statistics plan is based on the number of wounds. So that's why we have a range of patients, because knowing what our rate is, we'll be able to get there between 10 and 15. So you're right, it is 35 wounds. It's an estimate. I won't say we'll definitively stop at 35; it depends if we have a patient in the queue, but we won't do 50, for example. We'll be close to that number.

Okay, and just to clarify and think that this following up on <unk> question earlier can you can can you kind of give us some more granularity on how many patients are kind of in the screening or prescreening meeting.

And where they are but they're not all the way through to being able to actually enter the study.

Michael Amoroso: Okay, and just to clarify, I think this is following up on Maury's question earlier. Can you kind of give us some more granularity on how many patients are kind of in screening or in pre-screening? Meaning that you know where they are, and they kind of started the process, but they're not all the way there yet, being able to actually enter the study, just gives us a sense of how many patients we're looking at there. Yeah, sure, guys. And I know this came up in the past, and I appreciate you asking it, Rob.

Give us a sense of how many patients we're looking out there.

Yes, sure guide and I know this came up and the past and I. Appreciate you asking around and I'm going to just addressed as for the team to be fair to the team prior to me.

Standard as a pre cruise Prescreening study open where it's filters many patients into different studies. They have open based on genetic testing. Okay. So we did have some patients on the bench guys and 2020 and fairness to our prior team who based on pandemic, while unwilling to travel moved onto different studies that potentially are open. So we did lose some people along that line.

Michael Amoroso: I'm going to just address this to the team, to be fair to the team prior to me. Stanford has a pre-screening study open where it filters many patients into different studies they have open based on genetic testing, okay? So we did have some patients on the bench, guys, in 2020, in fairness to our prior team, who, based on the pandemic, were unwilling to travel, moved on to different studies that are potentially open. So we did lose some people along that line.

Right now we've got.

And I get nervous, saying the numbers because I don't like to jinx. It, but we do have a handful of patients that could potentially be patient six I always look at the next patient.

Right now with screening potentially two to three if we can get them to the site.

Whether they are all willing to travel we've contacted them. We're in communication, we've got five biopsy to I like to talk about definitive five is on their way to treatment, but right now patients six we're looking at three different individuals and Thats, obviously again out of Stanford before we have the second site up and running but we're always adding every month patient.

Michael Amoroso: Right now, we've got, you know, I get nervous saying the numbers because I don't like to jinx it, but we do have a handful of patients that could potentially be patient six. I always look at the next patient. Right now, we're screening potentially two to three if we can get them to the site. Whether they're all willing to travel, we've contacted them, and we're in communication. We've got five biopsies, so I like to talk about definitive.

<unk> I hired a chief patient officer, and Jodie Gillon, where full court press. We are doing this all the time. So next week, we could find two and three more were going to debt. So theres, even some international allowances now since Stanford IRB has opened up on that so we're looking at patients even out of Mexico, potentially Canada, Germany, So exact numbers.

Michael Amoroso: Five are on their way to treatment, but right now, patient six, we're looking at, you know, three different individuals. And that's obviously, again, at Stanford before we have the second site up and running. But we're always adding patient seminars every month. I hired a chief patient officer, Jody Gillen, where we do full-court press, we're doing this all the time. So, you know, next week, we could find two to three more we're gonna vet.

To give you, but I can tell you right now and the U S. There's two to three were assessing we could go three for three we could go over three I don't know what debt pre screening will look like I hope that gives you clarity Rob.

Michael Amoroso: So there's even some international allowances now since Stanford IRB has opened up on that. So we're looking at patients even out of Mexico, potentially Canada, and Germany. So exact numbers are tough to give you, but I could tell you right now in the US, there are two to three we're assessing. We could go three for three, we could go O for three.

No. That's very helpful. And then lastly on and maybe 101 I was wondering if you could provide some commentary regarding the correlation between <unk>.

And one closure of degree of wound healing and the extent of pain relief that you anticipate seeing based on the prior data.

Michael Amoroso: I don't know what that pre-screening will look like, but I hope that gives you some clarity. Now, that's very helpful. And then, lastly, on EB-101, I was wondering if you could provide some commentary regarding the core... Wound Closure, Degree of Wound Healing, and the Extent of Pain that you anticipate seeing, you know, based on the prior data. But how tight is that correlation? you know, complete wound closure accompanied by a 50% reduction in pain relief. Unknown Attendee, Brian Kevany, Gregory Gin, Vishwas Seshadri, Abeona Therapeutics Inc.

And how tight is that correlation is complete wound closure accompanied by like a 50% reduction and pain relief significantly greater than 50 per cent reduction and pain relief just give us a sense of what you're expecting to see with regard to the correlation between a wound.

Wound closure and wound healing and pain relief.

Yes. Another good question, Rob So look I think I think they're highly carload of can I give you a perfect number for the answer is no but I'll use data to tell you what was the backbone of why we drove such endpoints with the FDA and had these conversations first and foremost and commend the FDA and saying, Hey look we understand that ABL and youre not treating.

Michael Amoroso: Closure, Wound Healing, and Pain Relief

Michael Amoroso: Yeah, another good question, Rob. So, look, I think they're highly correlative. Can I give you a perfect number?

And the same wounds as maybe non EV diseases or even other <unk> diseases youre treating the most problematic wounds large and chronic wounds that can no longer shield themselves lead to significant opioid use quality of life issues and ultimately mortality.

Michael Amoroso: The answer is no, but I'll use data to tell you what was the backbone of why we drove such endpoints with the FDA and had these conversations. First and foremost, I commend the FDA for saying, hey, look, we understand that, Abeona, you're not treating the same wounds as maybe non-EB diseases or even other EB diseases. You're treating the most problematic wounds, large and chronic, wounds that can no longer heal themselves, leading to significant opioid use, quality of life issues, and ultimately, mortality.

If you look at our phase one two I'll be evidenced based and my decision might be and look at our phase one two and that's not exactly how were measuring pain in the phase III and the phase III, we're measuring it and are much more specific objective way remember the phase one two study was and academic study.

Michael Amoroso: So if you look at our phase 1-2, I'll be evidence-based in my decision, Ron. If you look at our phase 1-2, that's not exactly how we're measuring pain in phase 3. In phase 3, we're measuring it in a much more specific, objective way. Remember, the phase 1-2 study was an academic study. But if you look just at the patient at baseline, I'll tell you the difference.

And if you look just at patients at baseline and I'll tell you the difference from the phase one two for.

<unk> nine and I think the number was and the <unk> off the top of my head and Greg and correct me, 59% and 53% had pain at baseline, but that was a global impression of pain not specific to just one wound being treated what we.

Michael Amoroso: From phase 1-2, 59, I think the number was in the 50s, off the top of my head, and Greg can correct me. 59 or 53% had pain at baseline. But that was a global impression of pain, not specific to just one wound being treated. What we're testing is an actual wound that we treat versus the control wound in vital. So, you know, when the patients were enrolled in phase 1-2.

Testing is an actual wound that we treat.

<unk> as the control wound and vital so when the patients were enrolled and the phase one two.

50% to 75% wound closure, which we had and a lot of patients we were over 90% and 50% wound closure right.

And it was highly Karl with other if you look at the results you can see it and our investor deck with taking pain down to zero and a lot of people. Some had some trickle back pain, but again the phase one two was looking at global payments. So you couldn't delineate that maybe that wound felt great, but maybe they had an esophageal stricture or the dilation surgery, a week before and that can be.

Michael Amoroso: 50 to 75% wound closure, which we had in a lot of patients. We were over 90% and 50% wound closure, right? It was highly correlated. If you look at the results, you can see it in our investor deck. We're taking pain down to zero in a lot of people. Some had some trickleback pain.

And muddied and the phase one two result, that's not a possibility in the phase III and our phase III. The endpoint is specific wound being treated.

Michael Amoroso: But again, the phase 1-2 was looking at global pain, so you couldn't delineate that maybe that wound felt great, but maybe they had an esophageal stricter or dilation surgery a week before. And that can be muddied in the phase 1-2 results. That's not a possibility in phase three.

At time addressing change six months, one time point.

Versus the exact same pain scale self administered by the patient in front of the physician on the untreated wound. So can't give you a perfect number wrong highly call, but it's we think we know it's the most important morbidity condition today.

Michael Amoroso: In phase three, the end point is the specific wound being treated. At time of dressing change, six months, one time point, versus the exact same pain scale self-administered by the patient in front of the physician on the untreated wound. So I can't give you a perfect number, Ron.

Ultimately, we want a trained mortality of this disease and we think we've got to cover large chronic wounds as much as the body surface area for years for long periods of time to do that but again, that's not with vital is going to show us we're going to see that years down the road of covering these wounds through hopefully registries once we're approved but highly correlative.

Michael Amoroso: Highly correlative. It's, we think, we know it's the most important morbidity condition today. Ultimately, we want to change the mortality rate of this disease, and we think we've got to cover large chronic wounds as much as the body surface area for years, for long periods of time to do that. But again, that's not what Vital's going to show us. We're going to see that years down the road of covering these wounds through, hopefully, registries once we're approved. But highly correlative.

I can't give you a perfect number.

But these wounds unclosed almost all patients approaching 10 year, almost all patients aren't excruciating pain during dressing changed with these types of wounds I hope that gives you some color of the difference between the phase one two and the vital trial. The evidence from the phase one two and how we think about paying us as really the most important morbidity you improve.

Michael Amoroso: I can't give you a perfect number. But these wounds, when unclosed, almost all patients, I'll quote Gene Tang here, almost all patients are in excruciating pain during dressing changes with these types of wounds. I hope that gives you some idea of the difference between the Phase I-II and the Vital trial, the evidence from the Phase I-II, and how we think about pain as really the most important morbidity you can improve upon for these patients.

The problem for these patients.

No. That's very very helpful. Just two other very quick ones for me with respect for the transfer a study can you comment on what you expect ultimately the average age of patients to be at time of enrollment in cohort three and then lastly, do you have any updates on the situation with <unk> bio. Thank you.

Michael Amoroso: Now that's very, very helpful. Just two other very quick questions. With respect to the Transfer A study, can you comment on what you expect the average age of patients to be at time of enrollment in Cohort 3? And then, lastly, do you have any updates on the situation with Regenexx Bio? Yeah, what I'll do is I'll take the second question first on Regenexx Bio, and then I'm going to turn it over to some of the muscle I have on the phone because I'm super proud of this management team.

Yeah, what I'll do is I'll take the second question first on <unk> Bio and then I'm going to turn it over to some of the muscle and have on the phone because I'm Super proud of this management team I will turn the NPS question over to Juan who can tell you about the age.

For the enrollment so from <unk> standpoint, guys.

Not a not a tremendous amount more I can give you we will continue to update you about the arbitration ongoing obviously, it's an ongoing legal proceeding.

Michael Amoroso: I'll turn the MPS question over to Juan, and he can tell you about the age, you know, for the enrollment. So, from the Regenexx Bio standpoint, guys, not a tremendous amount more I can give you.

The status is included in our 10-K.

And we had and arbitrate arbitration hearing held earlier this month.

The tribunal has not yet issued its opinion for and overall overview of the proceeding and the extent to comment. Please refer to the 10-K also it'll be on our website. So again guys still ongoing can't tell you about the results, but I can tell you that the arbitration hearing was held earlier this month.

Michael Amoroso: We will continue to update you, but the arbitration is ongoing. Obviously, it's an ongoing legal proceeding. The status is included in our 10K. We had an arbitration hearing held earlier this month. The tribunal has not yet issued its opinion. For an overall overview of the proceeding and the extent of comment, please refer to the 10-K. Also, it'll be on our website. So again, guys, this is still ongoing. Can't tell you about the results, but I can tell you that the arbitration hearing was held earlier this month. Juan, why don't I turn the age question for MPS 3A over to you?

And why don't I turn the age question for MTS and <unk> over to you. Please.

Yes, Thank you and Michael.

Yes.

In cohort three we haven't enrolled and so far to 13 children.

And since we and modify amended the protocol to enroll in the younger children.

Junger than two years or without EQ higher than 60.

We have eight basins reduce credit Korea, and the ages range from eight months.

Juan Ruiz: We modified the amended protocol to enroll younger children, younger than two years, or with an EQ higher than 60. We have eight patients with these criteria, and the ages range from eight months to 33 months in that particular group. So we expect in the future, the next time the children that we are still, that might still be enrolled and treated in the trial will be within this age range.

33 months and.

And that particular group, so we expect and the future net.

I mean, the children that we are still and that might still be enrolled and treated and their trial will be within the sales range.

Thank you very much.

Thank you Juan and welcome.

Once again, if there are questions or comments, please press star one.

Your next question is coming from money from heart. Please announce your affiliation and pose your question.

Juan Ruiz: Thank you very much. Thank you all. Once again, if there are questions or comments, please press star 1. Your next question is coming from Moni Brouhard. Please announce your affiliation and post your question.

Thanks for the monitoring of our SBB Leerink.

But I'm sorry, I missed the detail I want to circle back on trade on the transfer be question can you give us a little sense of is it is this and assay development challenge and.

And like a collection of data and so.

Operator: Thanks, this is Monte Trujillo from SVB Larynx. I'm not sure I missed the detail. I want to circle back on the Transfer B question. Can you give us a little sense of, is this an assay development challenge? Is it like a question of data management, sort of SOP development? Give us exactly sort of what needs to happen to put your foot back on the accelerator there.

Data management.

Development sort of give a sense of exactly sort of what.

And what needs to happen to to put your foot back on the accelerated share.

Yeah, So Marty I'll remind you.

Couple of things transfer B, we are for patient shy of our cohort III target. So.

We have patients and the Q and the most important thing for us and get those patients treated that being said no not and assay development, we have the nationwide product as the debt as the product being used and transfer would be as well as trends Ray.

Monte Trujillo: Yeah, so Monty, I'll remind you of a couple of things. Transfer B, we are four patients shy of our Cohort 3 target. So, you know, we have patients in the queue, and the most important thing for us is to get those patients treated. That being said, not in assay development. We have a product; the nationwide product is a product being used in Transfer B as well as Transfer A. Jay and his team will be making our own Abeona product for MPS 3A in the second half of this year, another major milestone for us.

Jay and team will be making our own avionic product for NPS three eight second half of this year and other major milestone for us but the.

The product has patient ex product sorry product expiry dates and our last lot expired later into Q4. So what we're doing is we're joining and next stability test and look at our options to move forward. So it's not and assay development. We're just looking at a stability testing of a lot of product that was expired at a nationwide and we're going to see what our options. After the final few patients to be treated.

Monte Trujillo: But the product has patient, I'm sorry, product expiry dates, and our last lot expired later into Q4, so what we're doing is we're doing a next stability test to look at our options to move forward. So it's not an assay development, we're just looking at stability testing for a lot of product that was expired nationwide, and we're gonna see what our options are for the final few patients to be treated. Great, that's all.

Great that's helpful. Thanks.

Your next question is coming from Christian <unk>. Please announce your affiliation and pose your question.

Hi, Good morning. This is Kristen <unk> from Cantor Fitzgerald, Thanks for taking my questions.

Michael Amoroso: Great, that's helpful. Thanks. Your next question is coming from Kristen Kluxka. Please announce your affiliation and ask your question.

First is I know the data is the hot off the price here from world, but wondering if you could share any feedback that you heard from physicians or the different patient advocacy groups. Since your presentation, particularly from the youngest patients and the high dose cohort and NPS three and <unk>.

Kristen Brianne Kluska: Hi, good morning. This is Kristen from Cantor Fitzgerald. Thanks for taking my questions. The first is, I know the data is hot off the press here in the world, but I was wondering if you could share any feedback that you have heard from physicians or the different patient advocacy groups since your presentations, particularly from the youngest patients in the high-dose cohort in MPS 3a.

Kristen and good to hear your voice and there's no one better on this phone to do it then maybe the most knowledgeable guy I know and NPS and neurocognitive programs. One why don't you got please give a little bit of maybe some verbatim and some feedback from some of the kols around the world and the reaction to the three eight data and even the <unk> data. Please.

Michael Amoroso: Kristen, good to hear your voice. And there's no one better on this phone to do it than maybe the most knowledgeable guy I know in MPS and neurocognitive programs. Juan, why don't you please give a little bit of maybe some verbatims and some feedback from some of the KOLs around the world on the reaction to the 3A data and even the 3B data.

Yes, Thank you Michael and thank you Christine yes the range.

And that has been.

And.

And the feedback has been very positive.

And then all other data regarding the younger children and some of them for or.

Total of 30 months 36 months after treatment rates and D H, where children in network history are declining significantly.

Juan Ruiz: positive. I mean, when we announced the data regarding the younger children, some of them fall for close to 30 months, 36 months after treatment, reaching the age where the children in network history are declining significantly. And getting these children still gaining the skill has been received very positively by both the experts and the GPO leaders and the patient community.

And these children's day and gaming and at scale, that's being received very positive for you.

And I bought the expert.

Yes.

We've been reviewing things Weaver.

From there it seems to have a non recurring facebooks loss for around the world.

The other piece.

Yes.

And we are looking forward for new data come and easier to confirm that is positive.

Development continues.

And this sooner.

And what are they sort of restricted.

And though we are expecting additional data all other years.

Juan Ruiz: That we've been reviewing this with some physicians that are referring patients.

And Kristen I would be remiss and I'm getting a lot of trouble if I don't say this my chief patient officer is not on the phone with US today Jodie Gillon, just texted me and said patient patient groups are thrilled and encouraged relative rebate and so I'd be and a lot of trouble if I didn't share that would be for thank you Jody.

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

Michael Amoroso: And Kristen, I would be remiss and I'd get in a lot of trouble if I didn't say this, my chief patient officer is not on the phone with us today. Jody Gillen just texted me and said, patient groups are thrilled and encouraged. That's a verbatim. So I'd be in a lot of trouble if I didn't share that with you. Thank you, Jody.

Oh glad you won't be getting in trouble and then.

So as you think about the next potential chapter and ophthalmology and I know you haven't disclosed these indication maybe could you speak and remind us about the differentiated aspects of your aim captives versus some of the evidence and results we've seen across the field, so far and I do recall that you share.

Michael Amoroso: Well, glad you won't be getting in trouble then. So as you think about the next potential chapter in ophthalmology, and I know you haven't disclosed these indications, maybe you could speak and remind us about the differentiated aspects of your aim capsids versus, you know, some of the evidence and results we've seen across the field so far. And I do recall that you shared some initial data, I think at ASGCT maybe two years ago when we had conferences in person. So if you could remind us about some of the initial findings, and I know more work is ongoing here.

And some initial data I think at http, and maybe two years ago back when we had conferences and Paris and so if you could remind us about some of the initial findings and I know more work is ongoing here.

Yes, Chris and I appreciate the enthusiasm of the question and I'm excited about it too and <unk>.

Preclinical team, Brian Lina for teaching me about this all the time I think the short answer is I wish I could I think we're a little early I don't want to put the cart and from the horse, which is why we haven't disclosed the indications we will have some non human primate data, which my scientists tell me all the time is a very important proxy for when we're looking at the eye and the eye disorders.

Michael Amoroso: Yeah, Kristen, I appreciate the enthusiasm in the question. I'm excited about it, too. And my preclinical team, Brian and Linus, are teaching me about this all the time. I think that the short answer is, I wish I could.

We'll have that in the second and expecting to have that second quarter. Kristen. So I can promise you a follow up to talk about that and HP data as soon as we have it but I think it's a little early to talk about the results. What I can tell you is we are using some of our capsid from our partnerships. We're also using some of our captains from in house and the goal here is to see.

Michael Amoroso: I think we're a little early. I don't want to put the cart in front of the horse, which is why we haven't disclosed the indications. We'll have some non-human primate data, which my scientists tell me all the time is a very important proxy for when we're looking at the eye and eye disorders. We'll have that in the second, and I'm expecting to have that in the second quarter, Kris

Michael Amoroso: So I can promise you a follow-up to talk about that NHP data as soon as we have it, but I think it's a little early to talk about the results. What I can tell you is we are using some of our capsids from our partnerships. We're also using some of our capsids from in-house. And the goal here is to see whether we're also assessing administration, right? Pararetinal,

Administration right power retinal sub retinal into vitriol, so we're trying to see which captured and find a home for the sell best but I think it would be a little early if I was to give you a kind of what we're seeing because I think that non human primate data is very important. So we're enthusiastic my scientist have some really good.

Proof of concept, if you will but want to wait till we get the non human primate data before we we boast about it a little bit if that's okay.

Michael Amoroso: So we're trying to see which capsids find a home in the cell best. But I think it would be a little early if I was to give you kind of what we're seeing, because I think that non-human primate data is very important. So we're enthusiastic. My scientists have some really good proof of concept, if you will. But I wanna wait till we get the non-human primate data before we boast about it a little bit, if that's okay. That's fair. Thanks so much for taking the time to answer my question.

That's fair. Thanks, so much for taking the question.

Thank you.

There are no more questions in queue.

Well with that said I wanted to take a moment here and first thank the investor community for their support their interest and it's always nice to spend some time with you and I appreciate the questions that very thoughtful and appropriate. So thank you very much.

Kristen Brianne Kluska: That's fair. Thanks so much for taking the questions.

Operator: There are no more questions in queue.

I'll continue to ask you to tell.

Michael Amoroso: Well, with that said, I want to take a moment here and first thank the investor community for their support and their interest. It's always nice to spend some time with you. And I appreciate the questions. They're very thoughtful and appropriate.

And to hold US accountable that's important that's an important thing that we all take very seriously hurt ABL and because we're accountable to delivering to these patients.

I want to take a moment to say how excited I am to be had the privilege to be the next chief Executive officer here at <unk>.

Michael Amoroso: So, thank you very much. I'll continue to ask you to hold us accountable. That's important; that's an important thing that we all take very seriously here at Abeona because we're accountable for delivering to these patients. I want to take a moment to say how excited I am to have the privilege of being the next Chief Executive Officer here at Abeona, and I want to make sure I thank our stakeholders. The most important thing, well, they're all important, so I'll take that back, but super important are patients and the courage they have to be part of these trials. But I want to take a moment and thank my team.

And I want to make sure I think our stakeholders the most important.

And while they are all important so I'll take that back but super important for patients and the courage. They have to be part of these trials, but I want to take a moment and thank my team.

People I wont name everybody because we'll be here for a while but my management team, who has really been steadfast our employees' steadfast through a lot of change that you guys know occurred in 2020th Avionic, but they stayed mission focused.

Michael Amoroso: I won't name everybody because we'll be here for a while, but my management team, who have really been steadfast, our employees, steadfast, through a lot of change that you guys know occurred in 2020 with Abeona, but they stayed mission focused. We had our most productive, maybe, quarter yet in Q4. We had an incredibly successful type B meeting. We worked with the agency, brought in increased talent, and we've got our type B meeting on the books for NPS 3A.

We had our most productive maybe quarter yet in Q4, we had an incredibly successful type B meeting we have worked with the agency what increased talent in and we've got our type B meeting on the books for NPS and <unk>, we continue everyday to make milestones and I really model and I'm Super impressed by a group of people and their commitment and even.

The pandemic has slowed them down so.

Again, please hold us accountable, it's a year of operational excellence, we need to bring the science to fruition for patients and I'll leave it at that just a sincere. Thank you to my team.

Michael Amoroso: We continue every day to make milestones, and I really marvel and I'm super impressed by a group of people and their commitment, and even the pandemic hasn't slowed them down. So, again, please hold us accountable. It's a year of operational excellence. We need to bring this science to fruition for patients, and I'll leave it at that. Just a sincere thank you to my team, to my employees, to my board, to patient stakeholders, our KOLs and investigators, and to you, the investor community, for your continued support and your open and honest dialogue.

To my employees to my board to patient stakeholders, our kols and investigators and to you the investor community for your continued support and your open and honest dialogue.

Thank you.

Thank you ladies and gentlemen, this does conclude today's conference call. You may disconnect. Your phone lines at this time and have a wonderful day. Thank you for your participation.

Operator: Thank you, ladies and gentlemen. This does conclude today's conference call. You may disconnect your phone lines at this time and have a wonderful day. Thank you for your participation.

Okay.