Q4 2021 Abeona Therapeutics Inc Earnings Call
Good morning, ladies and gentlemen, thank you for standing by and welcome to the <unk>.
Operator: Good morning, ladies and gentlemen. Thank you for standing by.
Operator: Welcome to the Abeona fourth quarter and fiscal year 2021 earning At this time, all participants are on a listen-only mode. After management's prepared remarks, there will be a question and answer session. I would now like to turn the call over to your host, VP of Investor Relations and Corporate Communications at Abeona, Greg. Greg, please go ahead. Thank you, Kelly. Good morning, everyone.
<unk> fourth quarter and fiscal year 2021 earnings call. At this time all participants are in a listen only mode. After management's prepared remarks, there will be a question and answer session.
I'd now like to turn the call over to your host VP of Investor Relations and corporate communications at Greg.
Greg Gin, Greg. Please go ahead.
Thank you Kelly good morning, everyone.
Greg: I would like to welcome and thank everyone for joining us on our fourth quarter and full year 2021 conference call. The press release announcing the results and update is available on our website at www.abeonatherapeutics.com. On the call today with prepared remarks are Vish Seshadri, Chief Executive Officer of Abeona, and Joe Vizzano, Chief Financial Officer.
I would like to welcome and thank everyone for joining us on our fourth quarter and full year 2021 conference call.
The press release announcing our results and update is available on our website at www Dot maybe on the therapeutics Dot com.
On the call today with prepared remarks are vicious Saturday, Chief Executive officer of avionics, and Joe <unk> Chief Financial Officer.
Greg: After the prepared remarks, we will host the Q&A session. We're also joined today by Dr. Brian Kevany, Chief Technical Officer. Before we start.
After the prepared remarks, we will host the Q&A session.
We're also joined today by Dr. Brian Kevin <unk>, Chief Technical <unk>, Chief Technical Officer.
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.
Greg: 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 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 other periodic reports filed by the company with the Securities and Exchange Commission.
Vishwas Seshadri: These documents are available on our website at www.abeonatherapeutics.com. And with that said, I will now turn the call over to Vish. Thank you, Greg, and thank you, everyone, for joining us this morning. I'll start by discussing the implications of the announcement we made this morning related to the strategic focus on EB-101 and our preclinical eye programs, as well as initiatives to extend our cash runway. Our management team and board conducted an extensive review of our operations and pipeline.
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.
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 other periodic reports filed by the company with the Securities and Exchange Commission.
Documents are available on our website at www dot maybe on a therapeutics dot com and with that said I will now turn the call over to vish.
Yes.
Vishwas Seshadri: We believe this plan reflects the operating discipline needed to extend our cash runway beyond our near-term anticipated catalysts and has the potential to create long-term value for our shareholders. Let me review the three key elements of the plan. First, we made the decision to focus on EB-101, our investigational autologous gene-corrected cell therapy, following the achievement of target enrollment in the pivotal Phase III vital study of EB-101 for recessive dystrophic epidermolysis bullosa.
Greg and thank you everyone for joining us this morning, I'll start by discussing the implications of the announcements. We made this morning related to the strategic focus on EBIT 101 in our preclinical programs as well as initiatives to extend our cash runway.
Our management team and board conducted an extensive review of our operations and pipeline. We believe this plan reflects the operating discipline needed to extend our cash runway beyond our near term anticipated catalysts and has the potential to create long term value for our shareholders. Let me review the three key elements of the plan.
First we made the decision to focus on EB 101, our investigational autologous gene corrected cell therapy. Following the achievement of target enrollment in the pivotal phase III vital study of VB 101 for recessive dystrophic Epidermolysis below so this achievement marks an important milestone for <unk> and we think the pace.
Vishwas Seshadri: This achievement marks an important milestone for Abeona, and we thank the patients, families, and the clinical investigators at Stanford and UMass for participating in the study. We are also especially grateful to our EB-101 program team for their relentless efforts in operationalizing the in-house production of both retroviral vector and the autologous gene-corrected epidermal sheets, and for ensuring that every patient biopsied and vital received EB-101 drug product. We expect top-line results of the study in the third quarter of 2022 that could support a biologics license application, BLA filing, and therefore EB-101 deserves to be the highest priority for our experience team.
<unk> family and the clinical investigators at Stanford and Umass for participating in the study were also especially grateful to our EBIT 101 program team for their relentless efforts in operation the lightning the in house production of both the retroviral vector and the autologous gene corrected epidermal sheets and four ensuring that every patient biopsies and baidu.
Received VB 101 drug product, we expect topline results of the study in the third quarter of 2022 that could support a biologics license application BLA filing and their EV 101 deserves to be the highest priority for our experienced team while focusing our R&D resources, primarily on vital data readouts.
Vishwas Seshadri: While focusing our R&D resources primarily on vital data readout, we will be actively pursuing a potential commercialization partner for EB-101. During the first quarter of 2022, we received positive feedback from the FDA in a type B meeting on the proposed CMC requirements of the EB-101 development program, and we gained alignment with the agency on characterization and validation plans that could support a potential BLA for EB-101 in our debt. Remember, in a Phase I-II study earlier, EB-101 had demonstrated a high rate of instantaneous wound healing and pain reduction for six years after treatment of large, chronic wounds in our debt.
We will be actively pursuing a potential commercialization partner for <unk> 101. During the first quarter of 2022, we received positive feedback from the FDA in a type B meeting on the proposed CMC requirements of the <unk> hundred one development program and we gained alignment with the agency on characterization and validation plans that could <unk>.
Port a potential BLA or EV 101 in our debt remember in a phase one two study earlier EBIT 101 has demonstrated a high rate of instantaneous wound healing and pain reduction for six years after treatment of large chronic wounds and our debt in may we will present at a medical meeting follow up data up to eight years the latest.
Vishwas Seshadri: In May, we will present at a medical meeting follow-up data up to eight years, the latest follow-up time point for the first patient treated with EB-101 in the Phase I-II study. Large chronic wounds typically do not heal spontaneously and inflict greatest pain and clinical burden on ARDAP patients. These wounds measure greater than 20 centimeter surface area and had remained open for at least six months, sometimes even for years, as we've previously shared.
Follow up time point for the first patient treated with <unk> hundred one in the phase one two study.
Large chronic wounds typically do not heal spontaneously and inflict greatest pain and clinical burden on our patients these wounds measure greater than 20 centimeters.
Area and had remained open for at least six months, sometimes even four years as we've previously shared with a primary focus on <unk> hundred one the second part of the plan is to divest <unk> of the <unk> program and we have already begun to carry out some activities regarding the ABL of 100.
Vishwas Seshadri: With a primary focus on EB-101, the second part of the plan is to divest Abeona of the MPS3A and MPS3B programs, and we have already begun to carry out some activities. Regarding the ABO-102 program for MPS3A, given the tremendous unmet need by patients and our positive data to date showing the potential of ABO-102 to not only increase brain volume but also preserve neurocognitive development in young patients, we have intensified our pursuit of a strategic partner to take over development activities. In addition, we have seized the build-out of the AAV manufacturing space. Let me briefly explain how we came to this decision.
Two program for <unk>, given the tremendous unmet need by patients and our positive data to date, showing the potential of <unk>, one or two to not only increase bring volume, but also preserve neurocognitive development in young patients we have intensified our pursuit of our strategic partner to take over development activities. In addition, we have seen.
The buildout of the AAV manufacturing space.
Let me briefly explain how we came to the position as part of the review of the transfer a statistical analysis plan in January 2022. The agency recommended that all participants be followed to an age of at least 60 months for the primary analysis of the neuro cognitive function.
Vishwas Seshadri: As part of the review of the Transfer Aid Statistical Analysis Plan in January 2022, the agency recommended that all participants be followed to an age of at least 60 months for the primary analysis of neurocognitive function. This would shift the anticipated timing of the neurocognitive outcomes data readouts to late 2024, early 2025, as compared to our prior projections of the second quarter of 2023. As a result of the FDA's feedback and our focus on preserving cash, we concluded that a strategic partnership would be the best opportunity to further advance ABO-102 toward bringing this novel therapy to patients. As part of our portfolio prioritization, we also decided to discontinue development of ABO 101 for MPS 3B.
This would shift the anticipated timing of the neurocognitive outcomes data Readouts to late 2020 for early 2025 as compared to our prior projections of the second quarter of 2023 as a result of the Fda's feedback on our focus on preserving cash we concluded that a strategic partnership would be the best opportunity to further advance <unk>.
One or two towards bringing this novel therapy to patients as part of our portfolio prioritization. We also decided to discontinue development of <unk> 101 for MPS III B.
Vishwas Seshadri: At this time, I want to take a moment to thank our dedicated team members who worked on AB0102 and AB0101 for their important contributions to Abeona and for their tireless efforts and expertise in advancing these programs for these patients. The third element of the plan is continuing our preclinical research investigating novel AAV capsids in five undisclosed ophthalmic conditions with estimated U.S. prevalence ranging from 5,000 to 1 We will be presenting results from testing of novel AAV capsids in non-human primates at the upcoming ARVO 2022 annual meeting in May.
At this time I want to take a moment to thank our dedicated team members, who worked on <unk>, two and <unk> 101 for their important contributions to <unk> and for their tireless efforts and expertise in advancing these programs for these patients.
Element of the plan is continuing our preclinical research investigating novel AAV capsid in five undisclosed ophthalmic condition with estimated U S prevalence ranging from 5000 to 15000 patients.
We will be presenting results from testing of novel AAV capsid in nonhuman primates at the upcoming ARVO 2022 annual meeting in May.
In addition, we expect animal proof of concept data readout beginning in mid 2022 that could support pre IND meetings with the FDA as early as second half of 2022.
We believe these strategic changes will ensure that abbvie owner has a focused team operating discipline and will extend the runway of our current cash resources to mid 2023.
Vishwas Seshadri: In addition, we expect animal proof of concept data readout beginning in mid-2022 that could support pre-IND meetings with the FDA as early as second half of 2022. We believe these strategic changes will ensure that Abeona has a focused team, operating discipline, and will extend the runway of our current cash resources to mid-2023. In finishing up my remarks, I would like to thank our outgoing Chief Financial Officer Ed Carr, and I want to introduce Joe Vazano, who was recently appointed as our Chief Financial Officer and is joining us on the call today. Joe previously served as CFO of Avenue Therapeutics where he secured multiple equity financing for the company. Welcome, Joe, and I'll now turn the call over to you.
And finishing up my remarks, I would like to thank our outgoing Chief Financial Officer, Ed car and I want to introduce Joe Mcdonnell who was recently appointed as our Chief Financial Officer and is joining us on the call today. Joe previously served as CFO of Avenue Therapeutics, where he secured multiple equity financings for the company welcome.
Joe and I'll now turn the call over to you.
Thank you Mitch.
Joe Vizzano: Thank you, Vish. I would like to remind everyone that the Form 10-K is available on our website, which is where you can get additional details on our financial results for the full year ended December 31st, 2021. Starting with the financial resources on our balance sheet, we had cash, cash equivalents, restricted cash, and short-term investments of $50.9 million as of December 31, 2021. In December 2021, we raised approximately $17.5 million in gross proceeds from an underwritten public offering of common stock. Our net cash used in operating activities was $65.7 million for the year.
I'd like to remind everyone that the Form 10-K is available on our website, which is where you can get additional details on our financial results for the full year ended December 31 2021.
Starting with the financial resources on our balance sheet, we had cash cash equivalents restricted cash and short term investments of $50 9 million as of December 31, 2021.
In December 2021, we raised approximately $17 $5 million in gross proceeds from an underwritten public offering of common stock.
Our net cash used in operating activities was $65 7 million for the year.
Joe Vizzano: 2021, including a $20 million payment in November 2021 in accordance with the settlement agreement with Regenexx Bio. Based on the new corporate strategic focus that Vish discussed and with the future cost savings, we believe that our estimate, Runway of Current Cash Resource, takes us into mid-2023. License and other revenues for the full year of 2021 were $3 million compared to $10 million in 2020.
2021, including a $20 million payment in November 2021 in accordance with the settlement agreement with <unk> by them.
Based on the new corporate strategic focus of this discussed and with the future cost savings, we believe that our estimate.
Runway of current cash resources it takes us into mid 2023.
License and other revenues for the full year of 2021 $3 million compared to $10 million in 2020.
Joe Vizzano: The revenue in 2021 resulted from a clinical milestone achieved in December of 2021 under a sub-license agreement with Tayshia Gene Therapies for AB202 for CLM1. Now, turning to research and development activities. We spent $34.3 million for the full year of 2021, compared to $30.1 million in 2020. Our spend on general and administrative activities was $22.8 million for the full year of 2021, compared to $23.8 million in 2020. The net loss was $85 million for the full year 2021, or $86, cents per share basic and diluted loss as compared to a net loss of 84.2 million or a 91 cents basic and diluted loss per common share in 2020. The net loss in 2021 included a one-time non-cash goodwill impairment charge of $32.5 million.
Revenue in 2021 resulted from a clinical milestone achieved in December 2021, under a sublicense agreement with tissue gene therapies for <unk> for <unk> disease.
Turning to research and development activities, we spent $34 3 million for the full year of 2021 compared to $30 1 million in 2020.
Our spend on G&A general and administrative activities was $22 8 million for the full year of 2021 compared to $23 8 million in 2020.
Net loss was $85 million for the full year 2021, or <unk> 86.
<unk> per share basic and diluted loss as compared to a net loss of $84 2 million or 91 basic and diluted loss per common share in 2020.
The net loss in 2021 included a onetime noncash goodwill impairment charge of $32 5 million.
This impairment charge has no impact on the company's current cash position cash flow from operating activities and does not have any impact on future operations.
Joe Vizzano: This impairment charge has no impact on the company's current cash position, cash flow from operating activities, and does not have any impact on future operations. With that, I'll turn the call back over to the operator for the Q&A. Aubrey?
With that I'll turn the call back over to the operator for the Q&A session.
Certainly.
Operator: Yes, the floor is now open for questions. If you have any questions or comments, please please press star one. We ask that while posing...
Yes. The floor is now open for questions. If you have any questions or comments. Please press star one on your phone at this time, we ask that while posing a question you. Please pickup your handset that's listening on speaker phone to provide optimum sound quality. Please hold just one moment, while we poll for questions.
Operator: Pick up your handset if listening on speakerphone to provide optimum, Just for a moment while we- Your first question is coming from Kristen Kluska. Please pose your question. Your line is open. Hi, good morning everybody.
Your first question is coming from Kristen <unk> with Cantor Fitzgerald. Please pose your question your line is live.
Kristen Kluska: Thanks for taking my questions. The first one that I have is for both of these LEAD programs; could you talk a bit more about the types of partnerships you're open to looking at at this point? Specifically, how are you thinking about different geographies, as well as upfront payments and other financial structures to support some of the next initiatives at the company, including a focus on ophthalmology, and also considering the rare pediatric disease designation and some of the monetary deals that we've seen recently around priority review voucher sales? Thank you for the question, Kristen, and great to hear from you. Just wanted to make sure there's a multi-part question.
Hi, good morning, everybody. Thanks for taking my questions. The first one that I have is for both of these lead programs could you talk a bit more about the types of partnerships you're open to looking at at this point specifically how are you thinking about different geographies as well as the upfront payments and other financial structures to support.
Some of the next initiatives that the company, including a focus on ophthalmology and then also considering the rare pediatric disease designation and some of the monetary deals that we've seen recently around priority review voucher sales.
Thank you for the question Christian and Great to hear from you just wanted to make sure. There is a multiple multipart question. So I just wanted to articulate and just make sure that I have everything down correctly. So the first is you are asking about the nature of partnerships both for EB 101, as well as maybe a one or two and let me start there and then you had.
Vishwas Seshadri: So I just wanted to explain and just make sure that I have everything done correctly. So the first thing is you're asking about the nature of partnerships, both for EB-101 as well as ABO-102. And let me start there. And then you had a couple of questions about iPrograms and the Rare Pediatric Voucher.
Couple of questions about <unk> programs, and the rare pediatric voucher or did I Miss anything there.
Nobody has that at all.
Vishwas Seshadri: Great. So, starting with EB-101, our goal is to find a commercialization partner where all the launch and the commercialization activities would be the responsibility of a partner, however, recognizing the value that we've built so far getting us to the launch. And we envision continuing to be the supplier as one scenario, and if such partnerships had opportunity for taking on supply and transfer of technology, we're open to that as well. So, that's a very broad multi-scenario answer that I've provided you, but at this point in time, all possibilities exist.
So starting with <unk> 101 or.
It is to find a commercialization partner, where all the launch and the commercialization activities would be the responsibility of our partner. However are recognizing the value that we've built so far getting us to the launch and we envision continuing to be the supplier as one scenario and as such partnerships had.
Opportunity for taking on supply and transfer of technology, we're open to that as well so thats.
A very broad multi scenario answer that I provided you, but at this point in time all possibilities exist now with the second question about the ABL of one or two program.
Vishwas Seshadri: Now, with the second question about the AB-102 program, the speed of speed is what is going to be very important for us. We will choose any day a speed of landing a partnership over optimizing deal value.
Speed of speed is what is going to be very important for us we will choose any day of speed of landing a partnership over optimizing deal value. So that's pretty much all we have at this point in time not able to tell you exactly what's going to be the nature of the deal structure, because that's a little bit of work in progress at this point in time, but of course it will all come.
Vishwas Seshadri: So, that's pretty much all we have at this point in time. Not able to tell you exactly what's going to be the nature of the deal structure because that's a little bit of work in progress at this point in time, but of course, it'll all come out in public when we have a definitive agreement of start. Because it's related to this topic, I'll cover the rare pediatric question. Both our first programs do have rare pediatric designations.
Out in public when we have definitive agreement of starts.
Because it's related to this topic I'll cover the rare pediatric.
Question, both our first programs do have rare pediatric designation and as you're aware, that's the requirement to be able to for eligibility for <unk>.
Vishwas Seshadri: And as you're aware, that's the requirement to be able to, for eligibility, for a priority review voucher when your BLA is approved. And we are on track for that for both the programs. And the second requirement for rare pediatric, for the PRV, is that we should secure a priority review for the BLA adjudication.
Priority review voucher when your BLA is approved and we are on track for that for both the programs and.
The second requirement for rare pediatric.
<unk> is that we should secure.
<unk> review.
For the BLA adjudication. So that's something that we're also anticipating so we should have the.
Vishwas Seshadri: So that's something that we're also anticipating. So we should have the, we're on track for eligibility for a priority review voucher for EB-101 approval. But of course, the BLA approval is what marks the trigger for such a coupon. And what we have seen in the latest trend that you've seen such vouchers trade in the vicinity of $100 million, depending on the timing, you have variations on that. But give or take, that's about the kind of value that we put to a PRV in general.
We are on track for eligibility for a.
Priority review voucher for <unk> hundred one approval, but of course, the BLA approval is what marks the trigger for such a coupon and what we have seen in the latest trends that you've seen such vouchers trade in the vicinity of $100 million depending on the.
The timing you have.
Variations on that but give or take thats about the kind of value that we put to <unk> in general.
Vishwas Seshadri: So those are what I would characterize as the opportunities for PRV. And you had a question about our preclinical eye programs, Kristen. Can you just remind me exactly what your question is about?
So.
Those are what I would characterize as the opportunity for <unk> and you had a question about our preclinical <unk> program Christian can you just remind me exactly what your question is about.
Kristen Kluska: Yeah, so that one was really just based off of the different types of deals you're potentially looking to explore for these LEAD programs, considering capital needs to really support that next initiative at the company, which may include a focus on ophthalmology. So like, are you looking for partnerships with, you know, like a good capital up front to really support moving that pipeline along? Absolutely, and as you know, the ophthalmology pipeline is not as capital-intensive as are our pivotal clinical programs, right?
Yes, so that one was really just based off of the different types of deals youre potentially looking to explore for these lead programs considering capital needs to really support that that next initiative at the company, which may include a focus on ophthalmology. So like are you looking for partnerships with.
Like a good capital upfront to really support moving that pipeline along.
Absolutely and as you know the ophthalmology pipeline is not as capital intensive as our pivotal clinical programs right. So we are able to move these programs to a good place with proof of concept experiments conducted through the year and that would be a good value inflection point for us to secure potential.
Kristen Kluska: So we are able to move these programs to a good place with proof-of-concept experiments conducted through the year, and that would be a good value inflection point for us to secure potential partnerships as we move them into the clinic. And it could have multiple different structures that could work, and depending on the ownership of various geographic commercialization opportunities and things like that, of course, some ophthalmology programs have specific interests outside of the U.S. as well, and we are going to be open to such types of structures as well.
Partnerships as we move them into the clinic and it could have multiple different structures that could work.
And depending on the ownership of various geographic the commercialization opportunities and things like that of course, some ophthalmology programs have specific interests outside of the U S. As well and we are going to be open to.
Such types of structures as well.
Okay. Thank you appreciate that and for MTS three eight could you help us understand the 16 months each time point for follow up based off of the agency feedback is this based on the cognitive age equivalent natural history data, where you see that slow start to the downward slope excuse me start too.
Kristen Kluska: Okay, thank you. Appreciate that. And for MPS 3A, could you help us understand the 16-month age time point for follow-up based off of the agency feedback? Is this based on the cognitive age equivalent natural history data where you see that slope start to, the downward slope, excuse me, start to become more apparent at 60 months of age?
Some more apparent at 60 months of age and then.
Despite the fact that you are looking for a partnership here is this still a program that you anticipate having some further durability data from that feature conferences. Thank you.
Vishwas Seshadri: And then, you know, despite the fact that you're looking for a partnership here, is this still a program that you anticipate having some further durability data from at future conferences? Thank you. Absolutely, Kristen.
Absolutely Christian Thank you for the question.
Vishwas Seshadri: Thank you for that question. The 60-month-age requirement or the strong recommendation from the agency to follow-up is for the chronological age of patients. So what it really means is all the children that we've dosed are young children, right? Many of them less than two years of age.
The 60 month age requirement, our strong recommendation from the agency to follow up is for the current illogical age of patients. So what it really means is all of the children that we dosed our young children many of them less than two years of age.
And.
Vishwas Seshadri: And the wording really means that follow these patients until they reach their fifth birthday, right? That's the 60 month chronological age. And the reason that you rightly articulated is because at that age, you see that the neurological decline has fully set in, and even acquired skills for these children start to decline, and they're also lost.
The wording really means that follow these patients until they reach their fifth birthday.
Vishwas Seshadri: And the opportunity to see a difference between the treated patients and what you've seen in natural history is probably most elevated at that kind of age time point. So that's exactly correct. So it's the chronological age of 60 months that the agency had expected. In terms of partnerships, of course, because this shifts the timing of a neurocognitive data readout is why it became important for us to focus on partnerships because obviously the cash runway, given our situation, everything, and we need such a partnership to take it all the way to the end point. But in terms of durability, all patients are still in follow-up.
That's the 60 months chronological age and the reason as you rightly articulated is because.
At that age you'll see that the neurological decline is fully set and even acquired skills for these children start to decline and there are also locked and the opportunity to see a difference between the treated patients than what <unk> seen in natural history is probably most elevated at that kind of age.
Time point, so that's exactly.
Correct. The chronological age of 60 months that the agency had expected in terms of partnerships of course, because this shifts the timing of neurocognitive data readout is why it became important for us to focus on partnerships because obviously the cash runway given our.
Situation everything we need we need such a partnership to take it all the way to.
To the endpoints, but in terms of durability. We are all patients are still in follow up the data flow from these studies will still continue and we will have well beyond that decline phase of patients that you've seen in natural history, we're going to see the data mature so.
Vishwas Seshadri: The data flow from these studies will still continue, and we will have well beyond that decline phase of patients that you've seen in natural history. And we're going to see the data mature.
Thats all rest assured.
Okay. Thanks, and if I may sneak in one last question here I understand MTS three b is earlier in development, but is there any particular reason for discontinuing this program versus perhaps looking at partnering both as a package deal given some of the synergies and NPS three and the programs here.
Kristen Kluska: Okay, thanks. And if I may sneak in one last question here, I understand MPS 3B is earlier in development, but is there any particular reason for discontinuing this program versus perhaps looking at partnering both as a package deal given some of the synergies in MPS 3A and the programs here? Yes, from our intent perspective, both programs are open for partnering, given the data maturity that we have with MPS 3A, naturally interest is higher for MPS 3A, but if there are potential partners that would take both programs, we're open to that as well.
Yes from our intense perspective, both programs that are open for partnering.
Given the maturity of data maturity that we have with MPS III a naturally interest is.
Higher for MBS III, but if there is.
If there are potential partners that would take good programs that are open to that as well, we just see that the prospects of <unk> partnering are higher at this point in time.
Vishwas Seshadri: We just see that the prospects of 3A partnering are higher at this point. Okay, thank you, Vish. Thank you, Kristen.
Okay. Thank you vishal.
Thank you Kristen.
Your next question is coming from money for Ohio with SBB Securities. Please pose your question. Your line is live.
Mani Faruhar: Your next question is coming from Mani Faruhar with SBB Securities. Please pose your question. Your line is live. Thanks.
Mani Faruhar: I'll start with a financial question. For the updated guidance runway into mid-2023, can you give us a little clarity on what assumptions around option warrants, exercise, any cash coming in from derivatives are baked into that? And then I have a follow-up that's more of an operational question. Great, thank you for the question. I'll turn it over to Joe, but I'll just start with saying that extending the cash runway to mid-2023 is with no assumption of extra cash flowing in beyond what we already have today, but Joe can talk about other potential opportunities as well. Joe?
Thanks, two quick ones I'll start with a financial question.
For the updated guidance runway into mid 2023.
Can you give us a little clarity on what on what assumptions around sort of options and warrants exercise any cash coming in from derivatives are baked into that and then I have a follow up with a more of an operational question.
Yes.
Great. Thank you for the question I'll turn it over to Joe, but I'll, just start with saying that.
The.
Extending the cash runway to mid 2023 is with the with no assumption of extra cash flowing in beyond what we already have today, but Joe can talk about other potential opportunities as well Joe.
Sure. Thanks, Rish and yes, we do in that current assumption with our current cash resources on hand right now.
Joe Vizzano: Sure, thanks Vish. And yes, in that current assumption, with our current cash resources on hand right now, we believe we will get to mid-2023. That does not include any exercise of any warrants or any other cash really infusion from an equity raise.
We believe we will get to mid 2023 that does not include any exercise of any.
Warrants or any other cash really an infusion from an equity raise.
Joe Vizzano: We anticipate the cost savings associated with this strategic decision announced with regards to the MPS programs is going to reduce our cash firm by approximately a couple million per quarter. Also, in addition with the cost savings from seizing the AAV build-out of about $13 million, all of that kind of bring us to our assumptions of mid-2023. Okay, that's helpful. So I want to put this to an operational, I guess, regulatory question. We were surprised, as I think others were, by the CRL received by Emirates, especially after the review for delay.
We anticipate the cost savings.
With this strategic decision.
<unk> announced with regards to the NPS programs is going to reduce our cash burn by approximately a couple of million per quarter.
Also in addition, with the cost savings from seizing the build out of about $13 million.
All of that kind of bring us to our assumptions of mid 2023.
Okay.
Okay. That's helpful.
So I wanted to just sort of operational I guess regulatory question.
We were surprised.
As I think others were.
By the CRM suite by Amarin.
Mani Faruhar: Can you give us a sense of where you are for their ED products? where you guys are in your discussions with regulators, how you view that. David Bautz, Gregory Gin, Vishwas Seshadri, Madhav Vasanthavada, Abeona Therapeutics Inc, Sure, I can take that question. The first point to note here is we don't have all the data details for the AMRIT data package, but what we understand is that the FDA is very particular about a six month time point and durability up to a six month time point, and they also look at various secondary endpoints in the case of the AMRIT data, right?
Especially after the <unk> delay.
Can you give us a sense of where you are for their <unk> product I mean, where.
Where you guys are in your discussions with regulators.
How you view that.
Potential tightening or a tightening of FDA treatment or more uncertainty and FDA treatment around pediatric dermatological diseases and he'd be in particular and what your interpretation of the dynamic is for what it means for EB 101.
Sure I can take that question.
The first point to note here is we don't have all the data details for the Android data package, but what we understand is that the FDA is very particular about a six month time point and durability up to a six month time point and.
They also look at various secondary endpoints in the case of <unk>.
<unk>.
Amarin data right.
We have pretty solid understanding with the FDA on what our two co primary endpoints are and the time point of six months and the two co primary endpoints that we have an agreement.
Mani Faruhar: We have a pretty solid understanding with the FDA on what our two co-primary endpoints are and the time point of six months. And the two co-primary endpoints that we have in agreement are, one is 50% wound healing by global investigator assessment, and the second one is pain reduction using the Wong-Baker scale. And we even have more recent, although the meeting where these two endpoints were agreed upon happened by end of 2020, we have had subsequent recent communications with the FDA and confirmed that this is what they're looking for.
Our one is 50% wound healing by global investigator assessment and the second one is pain.
Pain reduction used.
Using the wrong Baker scale, and we even have more recent although the meeting where these two endpoints were agreed upon happened end of 2020, we've had subsequent.
Recent communications with the FDA and confirm that this is what they are.
Mani Faruhar: The second point I would make is that the CMC is the other big aspect of a BLA filing. We had on March 18th a successful type B meeting with the FDA on the proposed CMC requirements for the development program and gained alignment with the agency on the characterization and validation plans that could support a BLA. And in the most conservative scenario even, we have agreed that three PPQ runs is what is the expectation and we're already prepared.
Looking for.
The second point I would make is that.
The CMT is the other big aspect of.
Of a BLA filing we had on March 18, a successful type B meeting with the FDA on the proposed CMC requirements for the development program and gained alignment with the agency on the characterization and validation plants that could support a BLA and the most conservative scenario even.
We are we have agreed that three three.
PBT, who runs is what is the expectation and we were already prepared so the roadmap is very clear for our CMC plans and upon a even if we go about that sequentially triggered way for Steve. The trial is positive and then put capital intensive resources towards the CMT BLA package, we're still can't.
Vishwas Seshadri: So the roadmap is very clear for our CMC plans, and even if we go about that in a sequentially triggered way, first see if the trial is positive, and then put capital intensive resources towards the CMC BLA package, we're still confident that quarter two 2023 is the timeframe we will be able to file a BLA. Of course, those activities can be further accelerated if we're able to raise additional money or land a partnership sooner. And that's something that we're going to be pursuing as well. Okay, that's helpful.
<unk> third quarter. Two 2023 is the timeframe, we will be able to.
File a BLA of course those activities can be further accelerated if we're able to raise additional money or Atlanta partnership sooner and that's something that we're going to be pursuing as well.
Okay. That's helpful.
Thanks, guys.
Mani Faruhar: Thanks, guys. Thank you. There appear to be no further questions in Kuwait, I would now like to turn the floor back over to Vishwas Seshadri for any closing, Thank you so much. We at Abeona are committed to developing novel cell and gene therapies for patients with rare diseases, and I want to thank our shareholders and other stakeholders who have listened to this call and for joining us today. We'll talk to you on our next quarterly call.
Thank you.
Yeah.
There appear to be no further questions in queue. At this time I would now like to turn the floor back over to Vishal.
To me for any closing remarks.
Thank you so much.
At <unk> are committed to developing novel cell and gene therapies for patients with rare diseases and I want to thank our shareholders and other stakeholders, who have listened to this call and for joining US today, we will talk to you on our next quarterly call. Thank you very much.
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.
Vishwas Seshadri: Thank you very much. Thank you, ladies and gentlemen. This does conclude today's program. You may disconnect your phone lines at this time, and have a wonderful day. Thank you for your participation.