Q3 2020 Abeona Therapeutics Inc Earnings Call

Ladies and gentlemen, thank you for your patience.

Operator: Ladies and gentlemen, thank you for your patience. Please remain on the line while we gather additional participants. Again, we do appreciate your patience.

The line probably gather additional pets.

Again, we do appreciate your patience.

Operator: Your conference will begin momentarily. Thank you. All lines have been placed in listen-only mode, and the floor will be open for your questions and comments following the presentation. If you should require assistance throughout the conference, please press star zero on your telephone. I'll now introduce your host for today's conference, Greg Gin, Vice President of Investor Relations at Abeona. Please go ahead. Thank you, Christy. Good morning, everyone.

Good morning, James Conference will begin momentarily.

[music].

Good morning, and welcome to your 80 on a therapeutics third quarter 2020 financial results call. All lines have been placed in a listen only mode and the floor will be open for your questions and comments following the presentation.

Gregory Gin: I'd like to welcome and thank you for joining us on Abeona Therapeutics' 3rd Quarter 2020 conference call. Yesterday, after the close of U.S. financial markets, we issued a press release announcing the third quarter results. The press release is posted on our website at www.abeonatherapeutics.com. On the call today with prepared remarks is Michael Amoroso, Chief Operating Officer of Abeona Therapeutics. Ann Carr, Chief Accounting Officer

Should require assistance throughout the conference. Please press star zero on your telephone.

I'll now introduce your host for today's conference Greg.

President of Investor Relations. Please.

Please go ahead.

Thank you Christie.

Good morning, everyone.

Gregory Gin: After the prepared remarks, we will host a Q&A session. We are also joined by Jay Bercher, Chief Technical Officer. Before we start, I'll review our Safe Harbor State. 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 law. Such forward-looking statements are based on current expectations and are subject to change. The actual results may differ materially from those expressed or implied in the forward-looking statements. 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 SBC.

I'd like to welcome and thank you for joining us on maybe other therapeutics third quarter 2020 conference call Yes.

Yesterday after the close of U.S. I know she'll markets, we issued a press release announcing the third quarter results.

A press release is posted on our website at Www Dot, Indiana Therapeutics Dot com.

On the call today with prepared remarks are Michael I'm, Russell Chief operating officer of any other therapeutics.

And our Chief Accounting Officer.

After the prepared remarks, do a host of Q and a session.

We're also joined by J. Bircher, Chief Technical Officer.

Before we start I'll review, our Safe Harbor statement.

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

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

Gregory Gin: These documents are available on our website at www.abeonatherapeutics.com. And with that, I will now turn the call over to you. Thank you, Greg.

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

Michael Amoroso: And good morning to our investment community. The Abeona team is excited to be with you this morning. We at Abeona remain committed to pursuing the development of our portfolio of advanced and early-stage programs toward providing our novel gene and cell therapies to patients who currently have no approved treatment options. In the near term, we are focused on continuing our clinical programs in recessive dystrophic epidermolysis bullosa, or RDEB, and Sanfilippo Syndrome Type A, also known as MPS3A, as well as Sanfilippo Syn First, starting with EB-101 in our VITAL program.

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 Companys annual report on form 10-K, and quarterly reports on form 10-Q filed by the company with the FTC.

These documents are available on our website at www Dot every other therapeutics that Oh.

So with that I will now turn the call over to Michael.

Michael.

Thank you Greg.

Hi, good morning to our investment community Yeah. The owner team is excited to be with you. This morning.

Well you know if you want to remain committed to pursuing the development of our portfolio. The danced in early stage programs toward providing our novel gene and cell therapies to patients who currently have no approved treatment options.

In the near term.

Michael Amoroso: Enrollment is ongoing for the EB-101 Pivotal Phase 3 VITAL study for RDEP, and three patients have been treated to date. As a reminder, target enrollment for the vital study is 10 to 15 RDEV patients with approximately 30 large chronic wound sites treated in total. We currently anticipate completing enrollment in the vital study in the first half of 2021, depending upon the impact of the COVID-19 pandemic, including travel restrictions and concerns about patients and or staff. Based on the current expectation for completing enrollment, we would anticipate top-line data for the vital study in late 2021. As a reminder, the pivotal endpoint will be six months post-last treated patient. Let's move on to our Phase 1-2 clinical trials for our adeno-associated virus-based investigational gene therapy, specifically AB0102 and AB0101 for MTS 3A and 3B, respectively.

Focused on continuing our clinical programs and recessive dystrophic epidermolysis bullosa or are dead.

And Sanfilippo syndrome type also known as MPS three eight.

As well as sanfilippo syndromes type b.

Also known as MPS three b.

To drive and beyond is near and long term growth and unlock shareholder value.

[noise] first starting with the B one on one and all vital program enrollment is ongoing so you'd be want to one pivotal phase three vital study far debts.

Three patients had been treated to date.

As a reminder, targeting a little bit to the vital study is 10 to 15.

Our debt patients with approximately 30 large chronic wounds sites treated in total.

We currently anticipate completing enrollment in the vital study in the first half of 2021.

Pending upon the impacts of the cold the 19 pandemic include.

Including travel restrictions and concerns about patient and or stat <unk> [noise].

[noise] based on the current expectations of completing enrollment we would anticipate topline data for the vital study in late 2021.

Michael Amoroso: We refer to these respective trials as Transfer A and Transfer B. I'm proud to report that target enrollment in the Transfer A study for MPAs 3A, which was 15 to 22 patients, has been achieved. Total enrollment to date in Transfer A is 18 patients, including 12 patients who were dosed in cohort 3, a higher dose limit of 3 times 10 to the 13th vector genomes per kilogram. We at Abeona have made the decision to continue enrolling patients into the Transfer Ace Study through the first quarter of 2021, given the lack of treatment options for patients afflicted with MPS 3A, as well as on the back of our encouraging data from Cohort 3 from a safety and efficacy standpoint, turning to the Transfer B Study for MPS 3B. To date, 11 patients have been dosed, including four patients dosed in Cohort

As a reminder.

Pivotal endpoint will be six months post last treating patients.

Let's move on to our phase one two clinical trials for our Adeno associated virus Avi based investigational gene therapies.

Specifically 80, or one or two maybe a one to one for NPS three eight and we'd be respectively.

We refer to these respective trials is transfer eight and transfer be.

[laughter] I'm proud to report the target enrollment in the trends for a study for M. P. S. Three eight.

Which was 15 to 22 patients has been achieved.

Total enrollment to date and transfer ages 18 patients incur.

Including 12 patients who were dose in cohort three higher dose limit of three times 10 to the 13th vector genomes per kilogram.

[laughter] weed out do you want to have made the decision to continue enrolling patients into the transfer eight study through the first quarter of 2021.

Michael Amoroso: As a reminder, the Cohort 3 upper dose for Transfer B is 1 times 10 to the 14th vector genomes per kilogram. We anticipate completing target enrollment and the transfer B study, which is a range of 15 to 20 patients in the first quarter of 2021, since COVID-19 has impacted some patients' ability to travel and undergo study treatment. Regarding treated patients in the TRANSFER-A study, we have previously reported data showing preservation of neurocognitive skills between 18 months and two years post-treatment among three young patients who were treated in DOS Cohort 3 of the study.

Given the lack of treatment options to patients afflicted with MPS III.

As well as on the back of our encouraging data from cohort three and the safety and efficacy standpoint.

Turning to the transfer be study for MPS greedy.

To date 11 patients have been dosed, including four patients dosed in cohort three.

Reminder, cohort three upper dose for transfer be is one times 10 to the 14th vector genomes per kilogram.

We anticipate completing target enrollment in transfer be study, which is a range of 15 to 20 patients in the first quarter of 2021.

Since COVID-19 has impacted some patients ability to traveling to undergo study treatment.

[noise] regarding treated patients and the transfer rate study, we've previously reported data showing preservation of neuro cognitive skills between 18 months and two years post treatment amongst three young patients who had been treated in the dose cohort three of the study.

Michael Amoroso: We are gathering additional neurocognitive assessment data from the high-dose cohort 3, with follow-up for each patient for more than two years, some up to three. These patients are between three and about five and a half years of age, and we plan to present these results at a very near future medical meeting. The new data could provide additional evidence of ABL1 or 2's potential to prevent further neurodegeneration and preserve a normalization in the acquisition of neurocognitive skills in young MPS3A patients, versus the natural history of this disease, which indicates patients with MPS 3A between 30 and 36 months of age tend to plateau in terms of gaining skills, and they start to regress thereafter to minimal levels of neurocognitive and behavioral skills.

We are gathering additional neuro cognitive assessment data from the high dose cohort three.

Lets follow ups for each patient more than two years some up to three these.

These patients are between three and about five and a half years of age.

And plan to we plan to present these results in a very near term future medical meeting.

The new data.

Provide additional evidence of a b O one or twos potential to prevent further neuro degeneration and preserve and normalization in the acquisition of neuro cognitive skills and young M. P. S. Three a patients versus the natural history of this disease, which indicates patients with MPS three gave between 30 and 36 months.

Michael Amoroso: This data will be a very important time point later in the year as we will be looking at each of these patients two years plus after their dosing. In the third quarter, we presented our plan toward registration of ABL 102 for MPS 3A during a kickoff meeting under the EMA's PRIME program, which offers a path for accelerated review of promising therapies targeting unmet medical needs. Based on this meeting, along with our previous input from the Committee for Medicinal Products for Human Use, CHMP, and the Pediatric Committee of the EMA, PEDCO, we anticipate submitting a marketing authorization application for EEU conditional approval for AB0102 in MPS3A disease after the completion of a two-year follow-up neurocognitive assessment of the last patient treated in the Transfer A study.

At age 10 to plateau in terms of gaining skills and they start to regress thereafter to minimal levels neuro cognitive and behavioral skills.

This data will be very important time point later in the year as we will be looking at each of these patients two years plus post dosing.

In the third quarter, we presented our plan toward registration of PDL, one or two friends yesterday, I don't get a kick off meeting under the email Prime program, which offers a path for accelerated review was promising therapies targeting unmet medical needs.

Michael Amoroso: With regard to U.S. filing, we have engaged the FDA to discuss the regulatory path for ABO 102 and MPS 3A. We're targeting a potential meeting to take place in the first quarter of 2021, of course, depending on the FDA's availability, as they've been quite bogged down with the COVID-19 pandemic. We look forward to providing an update early next year on our ongoing plan and timelines for AB0102 in the United States. Moving onward to our manufacturing and technical operations activities, one of the great areas of strength for Abeona Therapeutics. Process development at our GMP Manufacturing Facility in Cleveland, Ohio, is ongoing and is expected to enable production in-house of the retrovirus used for EB-101 manufacturing. This will provide Abeona with increased control of our supply chain and product quality, while at the same time, of course, reducing costs.

Based on the meeting along with our previous input from the committee for medicinal products for human use see a jumpy.

And the pediatric committee of the M&A Petco.

We anticipate submitting a marketing authorization application for you conditional approval for 80 or one or two MTS in MPS three a disease. After the completion of two year follow up neuro cognitive assessment of the last patient treated and the trends for a study.

With regard to U.S. filing we have engaged the FDA to discuss the regulatory pass 80, or one or two in MPS three eight.

We're targeting a potential meeting to take place in the first quarter of 2021 of course, depending on the FD age availability as they've been quite bogged down with a cold at 19 pandemic.

We look forward to providing an update early next year on the ongoing plan and timelines for a deal one or two in the United States.

Michael Amoroso: In addition, process development activities to enable in-house manufacturing of commercial supply for our gene therapy products, AB0102 and AB101, are ongoing. As Greg indicated, Jay Bercher, our Chief Technical Operations Officer, will join us during the question and answer portion if any questions arise around our manufacturing process. Next, turning to corporate updates, we entered into two strategic partnerships with Keisha Gene Therapies for ABO202 for CLN1 disease, also known as infantile Batten's disease, and for an AAV-based gene therapy for Rett syndrome.

Next.

Moving on to our manufacturing and technical operation activities, one of the great areas of strength for avionics therapeutics.

Process development at our GMP manufacturing facility in Cleveland, Ohio is ongoing and is expected to enable production in house of the retrovirus used for either you want to one manufacturing.

This will provide he'd be owner with increased control of our supply chain [noise] product quality, while at the same time of course, reducing costs.

In addition process development activities to enable inhouse manufacturing of commercial supply for our gene therapy products, AB or one or two maybe you want to warn our ongoing.

As Greg indicated G. Bircher, our chief Technical operations Officer will join US during the question and answer portion if any questions arise around our manufacturing.

Michael Amoroso: These partnerships are expected to allow us to unlock near-term value in earlier stage non-core assets, while also providing Abeona the opportunity to share in the future success of these programs through achievement-based clinical, regulatory, and sales milestones, plus royalty-based payments on net sales. We're excited to work with this partnership in order to propel these gene therapy products to patients faster. Before I turn the call over to Ed for the financial portion of today's discussion, I wanted to say a few words about what excites me about Abeona as I've assumed my new role as operating leadership. Abeona has a significant foundation in place which starts with our great people and their capability, deep science, and a robust pipeline, both near and longer term. In addition to our clinical programs, we are researching and developing next-generation AAV-based gene therapies using Abeona's novel capsids and capsids from the AIM technology platform. We intend to continue to develop chimeric AAV capsids capable of improved tissue targeting for various indications, including different monogenetic retinal disorders.

Next turning to corporate updates.

We entered into two strategic partnerships with Acacia gene therapies for HBIO tool to pursue a one to one disease also known as infantile buttons disease.

And for an havey based gene therapy for Rett syndrome.

These partnerships are expected to allow us to unlock near term value in earlier stage noncore assets well.

While also providing to add beyond that the opportunity to share in the future success of these programs through achievement based clinical regulatory and sales milestones plus royalties based payments on net sales.

We're excited to work with this partnership in order to propel these gene therapy products to patients faster.

Before I turn the call over to Ed for the financial portion of todays discussion I wanted to say a few words about what excites me about abbvie owner well.

While I've assumed my new role was operating leadership.

Happy owner has a significant foundation in place, which starts with great people and their capability.

Our deep science, and a robust pipeline, both near and longer term.

In addition to our clinical programs, we are researching and developing next generation HCV based gene therapies, using maybe owners novel Capsids and Capsids from the technology platform.

Michael Amoroso: Our Board of Directors and our senior management are fully committed to our company's future. It's an exciting time as we shape the future of Abeona. We shape the future for patients, and as we work toward addressing these unmet medical needs. With the organizational changes announced in October, I am proud to report we have minimized the distraction of attendance. We have continued to stay poised to unlock the potential of our people in our pipeline, and I am privileged to lead this group going forward. I thank you for your time today.

We intend to continue to develop Kymaerica Avi capsids capable of improved tissue targeting the various indications, including different model genetic retinal disorders.

Our board of directors.

And our senior management are fully committed to our company's future.

It's an exciting time as we shape the future they deanna will.

The shape the future for patients.

Ed Carr: And with that, I'm going to turn this over to Ed Carr, our Chief Accounting Officer, for a financial update. Thank you, Michael. I'd like to remind everyone that we recently filed the Form 10-Q, which is available on our website and where you can get the details on our financial results. In summary, our cash, cash equivalents, receivables, and short-term investments as of September 30, 2020 were $103.9 million compared to $129.3 million as of December 31, 2019. Accounts receivable was $7 million at September 30, 2020, which was paid in October 2020. We did not use our $150 million at-the-market offering programs in the third quarter of 2020. Net cash used in operating activities was $10.7 million for the third quarter.

And as we work toward addressing these unmet medical needs.

With the organizational changes announced in October.

Proud to report, we have minimize distraction to the team.

We have continued to stay poised to unlock the potential of our people and our pipeline.

I am privileged to lead this group going forward.

I. Thank you for your time today.

With that I'm going to turn this over to Ed Carr, our Chief Accounting officer for a financial update it. Please.

Thank you Michael.

I'd like to remind everyone that we have recently filed the form 10-Q, which is available on our web site, where you can get the details on our financial results.

In summary, our cash cash equivalents receivables and short term investments as of September Thirtyth, 2020, 103.9 million compared to 129.3 million as of December 31, 2019.

Accounts receivable was 7 million at September Thirtyth, 2020, which was paid in October 2020, we.

We did not use our 150 million at the market offering program during the third quarter of 2020.

Operator: Based on our current operating plans, we believe that we have sufficient resources with our existing cash, cash equivalents, and short-term investments to fund operations through the next 12 months without accessing the $150 million at-the-market program as a potential source of cash. And with that, I will now turn it over to the operator for Q&A. Operator? Operator? Operator? Operator? Operator? www.microsoft.com.

Net cash used in operating activities was 10.7 million for the third quarter.

Based on our current operating plans, we believe that we have sufficient resources with our existing cash cash equivalents and short term investments to fund operations through the next 12 months without accessing the 150 million aftermarket program as a potential source of cash.

And with that I will now turn it over to the operator for <unk> operator.

Operator.

Operator: Thank you. The lines are now open for questions. If you would like to ask a question, please press star then 1 on your telephone keypad. And if you're using a speaker phone, please pick up your handset to provide the best sound quality.

Thank you the lines are now open for questions.

Good question. Please press Star then one.

And if you are using a speakerphone. Please pick up your handset to provide the backbone clot.

Maurice Thomas Raycroft: And we'll take our first question from Maurice Raycroft with Jeffrey. Please go ahead. Good morning, this is Faisal Inam for Molloy.

And we'll take our first question from Maury Raycroft with Jefferies. Please go ahead.

Everybody is fighting on from Laurie.

Michael Amoroso: So, with our upcoming discussion with FDA in the second half, in the first quarter, we have a compelling case of good safety and neurocompetency. So, what do you believe is the bar for success? And is there a possibility of approval based on the patient's treatment so far, particularly with the younger patients? Yeah, Maury. This is Michael.

[laughter] any discussion with FDA in the second half or in the first quarter.

Thank you said good safety and unit.

What do you need at the bar for success and these days, but to me I forgot to based on the patient's treat yourself I think limits the younger patients.

Good morning. This is Michael Thank you for the question I'll start on this one so we're hoping to target a meeting early next year with the FDA to.

Michael Amoroso: Thank you for the question. I'll start on that one. So we're hoping to target a meeting early next year with the FDA to discuss our MPS 3A program. We obviously have had really good interactions with the EMA under the prime designation. I think the bar that is most important, of course, from a phase 1, phase 1b, point of view, is safety. I think the cohort 3 dose has shown signs of absolutely being safe. I think we've shown some good early biomarker data, namely the heparin sulfate levels in cerebrospinal fluid, as well as the decrease in liver volume in these patients. But, of course, the most important is the neurocognitive end point, specifically in the two-year marker range after treatment. We believe that early intervention, while the developmental quotient, I'll remind everybody for transfer A, is still greater than 60%. For these three patients, it was greater than 70%.

To discuss our MPS three a program. We obviously have had really good interactions with the Amelia under the prime designation.

I think the the bar that is most important of course from the phase one phase one b is safety.

I think cohort three dose has showed signs of absolutely being safe I think we've shown some good early biomarker data.

Namely the heparin sulfate levels in cerebral spinal fluid as well as a decrease in liver volume in these patients.

As well as of course, the most important is the neuro cognitive endpoints specifically in the two year market range after treatment.

We believe that early intervention a wall the developmental quotient I'll remind everybody for transfer a developmental quotient is still greater than 60% to these three patients it was greater than 70%.

Michael Amoroso: The idea here is to intervene early at the right dose, the right therapeutic dose, in order to preserve neurocognitive capability and the ability for motor skills to continue to improve and gain as per a normal non-afflicted child. So we'll review the data at the time with the FDA, the biomarker data, the safety data, as well as the neurocognition data of the longest patients treated. And at that time, we hope to have some greater clarity of what our path forward will look like. So, Maury, I appreciate the question. I think these are some of the markers you should think about for San Felipe Ley syndrome patients, and we'll look to report back as we have more in early 2021.

Idea here is to intervene early at the right dose like therapeutic dose in order to preserve neuro cognitive capability and the ability for motor skills to continue to.

Improve and again as per normal non afflicted trial child so.

So we'll review the data at the time of the FDA Oh, the biomarker data the safety data as well as the neuro cognition data as long as patients treated and you know at that time, we hope to have some greater clarity of what I'll pass the call. It will look like so Maureen I. Appreciate the question I think these are some of the markets. We should think about for the send to lead the way syndrome patients and to go look.

To report back as we have more and not early 2021. Thank you for your question.

Michael Amoroso: Thank you for your question. A follow-up question is, for the process development activities available in-house manufacturing for the 102 and 101 programs, are you exploring your aim vectors, or are you still using the AAB9? Yeah, I think what I'll do is I'll let Jay Bircher, our tech ops lead, answer this question. Jay, if you wouldn't mind.

Has got a lot of questions, but that thought.

Everything in house manufacturing.

On the two I want to once again I exploring your aim factors how are you still using that it'd be nice.

Yeah, I think what I'll do is I'll, let Jay Bircher Arctic obsolete answer. This question Jay if you wouldn't mind [noise].

Jay Bercher: Hi, good morning. We are continuing to use our AAV9 platform for both NPS 3A and NPS 3B. And Molloy, the only thing I would add to that is our AIM capsule platform is still being characterized by our preclinical team. They've made some nice gains this year.

Hi, good morning.

We are continuing to use our avi nine platform.

For both MPS, three and MPS threepi.

And really the only thing I would add to that is our aim capsid platform is a still being.

Characterize through our preclinical team they've made some nice gains this year and we hope to talk more about that with you in the upcoming investor calls.

Jay Bercher: We hope to talk more about that with you in the upcoming investor meeting. Thank you very much. And our next question comes from Mani Faruhar with SVB News. Please go ahead. Thanks for taking the question. A couple of quick ones.

Excellent. Thanks, so much.

Thank you.

And our next question comes from money Uh Huh.

Please go ahead.

Hi, Thanks for taking the question a couple of quick ones well start with.

Operator: We'll start with what exactly is the status of the RIPE arbitration slash dispute with Regenexx. How should we think about that resolution in terms of timeline and your plan to communicate any resolution you reach there? And then I have a quick follow-up. Sure, good to hear your voice. Thanks for the question. The arbitration is an ongoing legal proceeding. We estimate the arbitration hearing will take place in March of 2021.

What exactly is the status of the IP arbitration SAS dispute with regard to the dry next but how should we think about that resolution.

In terms of timeline in your plan to communicate any any resolution to each their and then I have a quick follow up.

Sure good to your voice. Thanks for the question the arbitration is ongoing.

Legal proceeding we estimate arbitration hearing will take place in March of 2021.

Operator: A full overview of the status is included in our 10-Q filing that's available on our website. At this point, I can't provide any additional answers about the arbitration apart from what's in the 10-Q and the timing that we're estimating, you know, for the hearing to take place. Great, that makes a lot of sense to me. I guess the other question is, clearly, a little bit of, we'll say a little bit of turnover on the leadership team. How should we think about timelines in terms of potential new CEO search, you know, potential restocking of the board, etc.? Should we think about that being sort of an early 21st century event?

A full overview. The status is included in our 10-Q filing that is available on our website.

But at this point I can't provide any additional answers about the arbitration apart from whats in the 10-Q and the timing that we're estimating you know for the hearing to take place.

Great that makes a lot of strikes me I guess, the other question I'm really a little bit as well.

We'll see a little bit of turnover.

And the leadership team how should we think about timelines in terms of potential new potential <unk>, new CEO search you know potentially talking to the board et cetera, just should we think about that being sort of early 21 event, that's being contingent on the process.

Operator: Is that something that's contingent on the process that Jeffrey's helping you with? How should we think about it? Yeah, it's a great question. I appreciate it. So I'll take that in pieces.

He is helping you with how should we think about it.

Yeah. It's a great question I appreciate it so I'll take that in pieces first I think the thing that's most important for us to remind everybody and partners is our management team is fully intact in place. So the team was here on did you Alan <unk> prior CEO fully in place a functional leaders are here for the day to day decision, making for our programs.

Michael Amoroso: First, I think the thing that's most important for us to remind everybody and our partners is our management team is fully intact in place. So the team who is here under Joe Allen, a prior CEO, fully in place, and our functional leaders are here for the day-to-day decision making for our programs to move forward, which, again, is most important to get these much, much needed therapies to patients as quickly as possible. So we have a very, very capable team, and we've been able to keep our management team in place. As far as a CEO search going forward, at this time, we're not looking for a CEO.

To move forward, which again is most important to get these is much much need therapies to patients as quickly as possible.

So we have a very very capable team and we've been able to keep our management team in place.

As far as the CEO search going forward at this time, we're not looking for a CEO.

Michael Amoroso: As we've discussed in previous press releases, we have a special committee comprised of some of our board members who are helping and overseeing the day-to-day strategic discussions with the management team, who, of course, oversee operations. Also, at this time, my responsibilities have changed, and as the Chief Operating Officer, I'll have oversight and leadership of all operations of the organization. So the last part of that question asked a bit about the strategic review, and I want to qualify the strategic review a little bit. I think whether we send out a press release or whether we have people who resign from the company or not, as a small biotech trying to get therapies to patients in the most expedited way, safe and effective manner, I think we're always looking for the right partnerships, and how we streamline activity in order to get to So this review with Jeffries is ongoing.

As weve.

We've discussed in press releases prior we had the special committee comprised of some of our our board members, who are helping and overseeing the day to day strategic discussions with the management team who of course oversee operations [noise].

Also at this time my responsibilities have changed and as the Chief operating officer, I'll have oversight and leadership of all operations of the organization. So.

The last part of that question I think you asked a bit about the de strategic review and I want to qualify the strategic review a little bit I think you what do we send a press release or what do we have people who resigned from the company or not.

As a small biotech trying to get off therapies to patients in the most expedited way safe and effective manner. I think we're always looking for the right partnerships or how we streamlined activity in order to get to market faster for these patients. So these these this review with Jefferies is ongoing it's something as you know we've done in the past we continue to.

Michael Amoroso: It's something we have done in the past, and we continue to do it. If the right partnerships make sense for us, then we will absolutely be open to those partnerships, and otherwise, we have planned. We're still going full force forward with our internal team moving these programs along. So hopefully, that recaps the CEO search, a bit about the strong management team and their capability who's here today, and a little extra information about the strategic review process, which is ongoing and again, really designed to help us propel and expedite our timing to market. If those right opportunities come along, I think we're very open to pursuing them, and if they don't, you know, Ed spoke to you about the financial solvency, Thank you for your questions. Great, thank you. And our next question comes from Justin Dellin with Be Riley Securities. Please go ahead.

Do it if the right partnerships make sense for US then we will absolutely be open to those partnerships and otherwise we have plan. We know we have still full force follow it up with our internal team moving these programs alone. So hopefully that recaps a the CEO search talk a bit about the strong management team and their capability who's here today.

And a little extra information about the strategic review process, which is ongoing and again really designed to help us propel and expedite our timing to market. If those right opportunities come along I think we're very open to pursuing them and if they don't you know Ed spoke to you about the financial solvency I think we feel strong that we can continue to book these programs along.

Thank you for your question.

Great. Thank you.

Your next question comes from Jeff Devon with B. Riley Securities. Please go ahead.

Operator: Hey, good morning, team. Thanks for taking the question. So first on this vital study, can you comment on how enrollment has been progressing with COVID? I think your previous enrollment guidance last quarter was that you were expecting to enroll one to two patients per month. So is that expectation still in place? And can you discuss your plan for opening additional clinical sites? Thanks. Yeah, sure. I'll take that one. So, thanks for the question. And a good one at that.

Hey, good morning, Thanks for taking the question. So first on the vital study can you comment on how enrollment has been progressing whats called it I think on.

Your previous enrollment guidance last quarter was that you're expecting that to enroll once your patients per month. So is that expectation still in place and can you discuss your plan on opening additional clinical sites.

[music].

Sure I'll take that one so thanks for the question of the good one so just a reminder, on the timeline of 2024.

Michael Amoroso: So just a reminder on the timeline of 2020 for the EB 101. In March, Stanford had a bit of a hold due to the COVID-19 pandemic, and then operations fully resumed in June. Our first patient of three was treated in March, and then patients two and three in July of August.

You want to one in March Stanford had a a a bit of a hold to do the COVID-19 pandemic.

And then operations fully resumed in June.

Our first patient of Threeg was treated in March and then patients two and three in July of August we are fully operational at this time working with gene Tang team.

Michael Amoroso: We are fully operational at this time, working with Jing Tang and the team. I think as we continue to look at our timing going forward, we say finish accrual in the first half of next year. We have a minimum of 10 patients, a maximum of 15. We're hoping to treat patient four here. We're screening patients currently very soon, potentially before the end of the year or maybe early next year. I think if you, we've talked about our technical operations capability, we can do two a month. So we have not changed our projections, our bold projections right now because we know we need these therapies for patients as fast as possible. So our timelines, we're still hopeful for the first half of next year.

I think as we continue to look at our timing going forward, we see a finish accrual first half of next year and we have a minimum of 10 patients a maximum 15.

We hope to treat patients for here, we're screening patients currently very soon or potentially a before the end of the you or maybe early next year.

No I think if you we've talked about our technical operations capability, we can do to a month. So we have not changed our our projections are a bold projections right now because we know we need these therapies to the patients as fast as possible.

So our timelines, we're still hopeful for first half of next year of course.

Michael Amoroso: Of course, the other question you asked, COVID-19 has definitely made things challenging. We've had the pre-screening of patients, as we've discussed before. Some of those patients we've lost due to an unwillingness to travel or maybe a different clinical trial that was more local in their area than Stanford. But we still have a good number of patients in the pre-screening phase we're going through. And we're trying; there was a bit of a hiatus on traveling.

The other question to ask Doug Koman, 19 has definitely made things a challenging we've had to pre screening of patients as we've discussed before some of those patients we've lost to a an unwillingness to travel or maybe a different clinical trial that was more local area than Stanford, but are we still have a good amount of patients into prescreening, we're going through and what.

Trying that there was a bit of a hiatus on traveling we're trying now to work closely with the sites and the patient families to get them to get them to Stanford [noise] last.

Michael Amoroso: We're trying now to work closely with the sites and the patient families to get them to Stanford. Last question, as far as a second site, as you know, our plans were to put a site on the East Coast for practical reasons, of course, so these patients had, you know, a West Coast major academic facility they could go to for treatment as well as an East Coast site. That was a bit of a challenge in the major cities with the pandemic and the way it hit us. At this point in time, we were not able to take onboard that second site. I will remind everybody, we feel very, very confident that we are able to acquire the Pivotal through Stanford. We didn't need a second site.

Last question as far as a second site as you know our plans what to put a site on the on the East coast for practical reasons of course Ah. So these patients had a you know a.

A west coast major academic facility, they could go to for treatment as well as an east coast.

That was a bit of a challenge in the major cities with the pandemic in the way it hit US at this point in time, we were not able to onboard that second sight.

I will remind everybody we feel very very confident that we are able to accrue. The pivotal grew Stanford we didn't need a second site. The second site and so on will continue to evaluate and we'll be opportunistic as we want to get more experience with this product as we think beyond just pivotal but as we're going to get ready for commercialization. So that's our current plans right now second sight is.

Michael Amoroso: The second site and so on will continue to evaluate and will be opportunistic as we want to get more experience with this product as we think beyond just Pivotal but as we're going to get ready for commercialization. So that's our current plan right now. The second site is not yet onboarded.

Not on board, we're still going to be opportunistic with something on the east coast central to try to make things a little easier a patient travel but at the same time as you know loading sites and going through arby's can take a bit of time and we still plan on potentially final accrual for the vital trial in the first half of next year.

Michael Amoroso: We're still gonna be opportunistic with something on the east or central to try to make things a little easier on patient travel. But at the same time, as you know, loading sites and going through IRBs can take a bit of time, and we still plan on potentially final accrual for the vital trial in the first half. Thank you for your question. So just a quick follow-up, just on the comment you made. So given there are now a few competitive programs enrolling hard-dead patients concurrently, do you expect that to continue to be a major issue in enrolling patients in this ulcerative heritage disease in the future? No, I don't. I really don't like them.

Thank you for your guys.

So just a quick follow up just on the comment you made so given there's now a few competitive programs.

Currently do you expect that to continue to be a major issue and enrolling patients in this ultra rare disease in the future.

No I don't I really don't I mean, if you look at the patient numbers were talking about you know 10, 11, 12, maybe up to 50 patients you're talking about potential our debt patients about 2500 in the United States are our best estimates.

Michael Amoroso: I mean, if you look at the patient numbers, we're talking about, you know, 10, 11, 12, maybe up to 15 patients. You're talking about potential R-dead patients, about 2,500 in the United States, are our best estimates. It's an ultra-rare space.

It's an ultra rare space, it's never perfectly easy to understand the total market opportunity. These patients today have a standard of care I'll remind you of of unfortunately wound dressing change gauze wraps appointments, there's nothing FDA approved or re and approved in this area right. Now so we really don't look as deep at the life science as an industry landscape.

Michael Amoroso: It's never perfectly easy to understand the total market opportunity. These patients today have a standard of care I'll remind you of: unfortunately, wound dressing change, gauze, wraps, ointments. There's nothing FDA-approved or EMA-approved in this area right now, so we really don't look at the life sciences and industry landscape doing trials in this space in any way competitive. In fact, these patients usually have a trajectory into their 30s and 40s before they unfortunately meet their demise.

Doing trials in the space in any way competitors. In fact, these patients usually have a trajectory of instead thirtys and fortys before they unfortunately be tomorrow. So we know what's going to be a continuum of care and we're going to need all the resources, we could possibly have an ontario.

Michael Amoroso: So we know it's going to be a continuum of care, and we're going to need all the resources we can possibly have in the arsenal. As far as competing for patients at the same time in some of the clinical trials, I think there are enough patients to go around. These are small patient numbers. We don't feel that that's a challenge.

As far as competing for patients at the same time on some of the clinical trials I think there's enough patients to go around is a small patient numbers. We don't feel that that's a challenge the bigger challenge is a a patient may have changed their plan some of the pre screen patients. They just didn't feel comfortable maybe getting on a plane were staying at a hotel. We don't think the competitive landscape accruing trials is an issue too.

Michael Amoroso: The bigger challenge is a patient may have changed their mind. Some of the prescreen patients, they just didn't feel comfortable maybe getting on a plane or staying in a hotel. But we don't think the competitive landscape for accruing trials is an issue to accrue vitals.

Crude vital.

Michael Amoroso: Great, thanks Michael. That's all for me. Thank you. Next, we'll move to Kristen Kluska with Cantor's 15-year-olds.

Great. Thanks, Michael that's all from me.

Thank you.

Next I'll move to Chris.

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Cantor Fitzgerald. Please go ahead.

Operator: Please go ahead. Kristen, your line is open. You may be muted on your end.

Christian Your line is open you may be muted on your end.

Kristen Brianne Kluska: Sorry, I was muted. Good morning, everybody. Thanks for taking my questions. And congratulations, Michael, on your promotion. So maybe just one big picture question on manufacturing for Jay, given some of the recent developments in the gene therapy space. Could you please walk through what you would view as the key strengths and capabilities at this time? And maybe how you're imagining your capacity and abilities, say, five years from now. And then also, what have you learned from others in the space in regards to preparing everything towards becoming a commercial stage company, potentially? Good morning. Thank you for the question, Kristen. So let me try to unpack that in a couple different ways.

Sorry, I was muted and good morning, everybody. Thanks for taking my questions and congratulations Mike on your promotion.

One Big picture question on manufacturing for Jay and given some of the recent developments in the gene therapy space [laughter] could you. Please walk through what you would view as the key strengths and capabilities at this time and maybe how you're imagining your capacity and ability say five years from now and then also what have you learned.

From others in the space in regards to preparing everything towards becoming a commercial stage company potentially.

Hey, good morning, Thank you for the question Kristen.

So let me try to unpack that a couple of different ways.

Jay Bercher: So first, with regard to our internal capability, really, over the last two years, Abeona has invested heavily in its people infrastructure, so its human capital internally. We've developed really a world-class team who have positioned Abeona both in the near term with our current programs and in the long term with some of our pipeline products. And what I mean by that is, you know, we've moved from an adherent-based system to a serum-free suspension system that is fully scalable. We've actually done some work with some of our lead equipment manufacturers to scale our product to a point where we feel very comfortable providing commercial material, both in the near term and long term with existing facilities. And as we add additional pipeline products, we think our existing facilities will meet our needs. Should they not? We have also done some preliminary engineering work with some nearby facilities here in the Cleveland area to advance additional manufacturing capacity should it be required. So really, we're positioned today to meet the continuing needs of Abeona, and then should we need to do that? Should we expand in the future?

So first with regards to our internal capability.

The last two years, if you want as invested heavily in the people infrastructure sort of human capital.

Internally Weve developed Oh.

Really a a world class team, who has positioned <unk> both in the near term with our current programs.

In long term with some of our pipeline products.

I mean by that is you know we've moved from a.

It's here based system to a serum free suspension system fully scalable, we've actually done some work with.

Some of our lead equipment manufacturers to scale our product.

To a point, where we feel very comfortable.

Providing a commercial material both in the near term and long term with the existing facilities.

And as we add additional pipeline products.

I think our existing facilities will meet our needs should they not.

We have also done some preliminary engineering work with some nearby facilities here in Cleveland area to advance a additional manufacturing capacity should it be required so really were positioned today.

To meet the continuing needs of a baby going Oh, and then should we need to do that or should we to expand in the future.

Jay Bercher: We have the capability and the lever to pull to do that. In regards to, you know, what we have learned from others, I think we should continue to network and learn from others in the space. I think the key for us is, you know, we learn from, you know, what people have had to do from a comparability perspective, and we've taken those learnings in mind from an assay development perspective. We have a full assay development team in-house that allows us to rapidly meet some of the changing needs. If you remember two years ago, the agency put in a requirement around physical titer and the specific requirements for all companies to meet that, and we were able to rapidly meet and exceed those requirements through our in-house capabilities.

We have the capability in the lever to pull to do that.

In regards to you know what did we learn from from others. I think we continue to network and learn from others in the space I think the key for US is you know we learn from you know what people had to do from a comparability perspective.

We've taken those learnings in mind.

When I say development perspective, we have a fool assay development team in house that allows us to rapidly meet some of the changing needs. If you remember two years ago.

The agency put a requirement in on around physical tighter.

In the specific requirements for all companies to meet that and we were able to rapidly meet and exceed those requirements through our in house capabilities. So yeah. We continue to survey Oh, well the landscape in Italy. The changes that are happening in respond to those through our internal capabilities.

Jay Bercher: So, you know, we continue to survey the landscape and the changes that are happening and respond to those through our internal capabilities. And Kristen, if I could just add to that, and thank you for your kind words. I think Jay gave a wonderful overview. I'll remind everybody, when I joined Abeona earlier this year, coming from the cell and gene therapy space, I think one of the greatest things that attracted me was the capability of our tech ops group in Cleveland. And, you know, you asked about commercialization. And I think one of the things we've learned from some of our industry peers; we've brought some people over from the CAR-T space, of course, as that was really the first upheaval of autologous cell therapy, personalized therapy. And I think we've learned that scaling, having nimbleness around scaling is so important and not overbuilding.

Hi, Kristen if I could just address that and thank you for your <unk> could you guys. Thank you for your kind words I.

I think Jay gave a wonderful overview I'll remind everybody when I joined they'd be on early this year coming from the cell and gene therapy space I think one of the greatest things that attracted me was the capability of our Tech Ops group in Cleveland and you know you asked about commercialization and I think one of the things we've learned from some of our industry peers, we brought some people over from.

The car T space of course as that was really the first I'll people of autologous cell therapy.

Personalized therapy, and I think we've learned that scaling having a nimbleness around scaling is so important to not overbuilding and I didn't j's team has has really showed a great discipline not to have the flexibility to meet the needs, but also to make sure. We're looking at a the market impetus is before we build it continued to scale as we go to commercialization. So that's just the last.

Michael Amoroso: And I think Jay's team has really shown great discipline to have the flexibility to meet the needs, but also to make sure we're looking at the market impetus before we build and continue to scale as we go to commercialization. So that's just the last piece I would add there, having observed it for the last five to seven years myself in other forums. I think our Cleveland group is world class and I am very, very excited about their capability to propel us forward.

Piece I would add there having observed at the last five to seven years myself in other forms I.

I think Cleveland group is world class and very very excited about their capability to propel us forward.

Great. Thank you and then just a quick follow up question are there any political changes or anything related to the our dad study in case patients are you know unable to make all of their visits in light of kind of it. So you know for example home visits you know I know you talked about.

Michael Amoroso: Great, thank you. And then I just have a quick follow-up question. Are there any protocol changes or anything related to the RDEB study in case patients are, you know, unable to make all of their visits in light of COVID? So, you know, for example, home visits. I know you talked in great detail in the past about the different cameras to take photos of the wounds and whatnot, but, you know, anything else that you're doing to just, you know, completely make sure you're getting all of That's necessary for this patient group. Yeah, Kristen, great question.

Great detail in the past about the different cameras to take photos of their minds and whatnot, but you know anything else that you're doing to just you know completely make sure you're getting all of the data that's necessary for that patient group.

Yeah Kristen Great question at this time there is no protocol changes we always continue to stay you know very very diligent of communicating with you. After it's been a great relationship in the space I will share with you one of the major reasons. We have the endpoint you talked about things like Canfield the objective cameras direct investigator assessments and funded the clinician.

Michael Amoroso: At this time, there are no protocol changes. We always continue to stay, you know, very, very diligent in communicating with the FDA. It's been a great relationship in this space. I will share with you one of the major reasons we have the endpoints you talked about, things like Canfield, the objective cameras, direct investigator assessments in front of the clinician, and the whole nursing. So, Kristen, I commend you. You know the study well.

And the whole nursing, so Kristen I commend you know the study well one of the reasons, we have all of those levels of kinda clinical checking and oversight is to make sure. We have the most complete picture for these patients. If we do have issues going forward and we see them kind of resolving a bit and getting better but if we do it's great that we have all these different measurements within.

Michael Amoroso: One of the reasons we have all of those levels of clinical check-in and oversight is to make sure we have the most complete picture for these patients. If we do have issues going forward, and we see them kind of resolving a bit and getting better, but if we do, it's great that we have all of these different measurements within the trial to help us from any level of backup. But at this time, there is no change in our protocol. There is no change with, you know, our FDA approval at this point in time.

The trial to help us from any level of a backup but we at this time there was no change in our protocol. There is no change with you know our ft. A pass at this point in time, but to your point. It's I think we're we feel very good about the uncertain times that we have multiple measures in order to attract the patient progress.

Michael Amoroso: But to your point, I think we feel very good about the uncertain times that we have multiple measures in order to track patient progress. Okay, that's great to hear. Thanks for taking the questions.

Okay, that's great to hear thanks for taking the question.

Michael Amoroso: Thank you. And that does conclude our question and answer session for today. I'll turn it back over to Michael for any closing remarks.

Thank you.

And that does conclude our question and answer session for today I'll turn it back over to Michael for any closing remarks.

Michael Amoroso: Yeah, I want to first thank our patients who were part of our trials who believe in Abeona. Without them and our physicians, there's no way that we would be able to bring solutions to patients and the greater community. I want to thank our investment community for their consistent interest and their belief in Abeona. And I want to... give an extra thanks to our employees who're really staying focused and making sure that we keep the patient as our north star and our compass as we move forward. So, you know, there's times when there can be distractions, but I think this group exemplifies the opposite.

Yeah, I want to first thank our patients who were part of our trials, who believe in Indiana and without them and orphan Oh physicians. There is no way that we would be able to bring solutions to patients and the greater community.

I want to thank our investment community for their consistent interest and their belief and they'd be owner and I want to.

Given extra thanks to our employees, it's really staying focused and making sure that we keep the patient is on north star an accomplice as we move forward. So as you know there's there's times when they can be distractions, but I think this group exemplifies the opposite they they've been steadfast on the mission and the mission is to provide cure for some of these patients with these on these afflicted diseases with high unmet need.

Operator: They've been steadfast in the mission, and the mission is to provide a cure for some of these patients with these afflicted diseases with high unmet needs. So I thank you, the operator. I thank you to the team and thanks to the investment community for being with us. And that does conclude today's conference call. We appreciate your participation. You may disconnect your lines at this time. And have a great day. Unknown Attendee, Brian Kevany, Gregory Gin, Vishwas Seshadri, Abeona Therapeutics Inc.

So I. Thank you to the operator, and thank you to the team and things to the investment community for being with US This morning.

And that does conclude today's conference call. We appreciate your participation.

And that's your lines at this time and have a great day.

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