Q1 2021 Abeona Therapeutics Inc Earnings Call
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Good day, ladies and gentlemen, and welcome to the first quarter earnings call. At this time all participants are in a listen only mode. We will open up the floor for questions and comments after the presentation.
Operator: Good day, ladies and gentlemen, and welcome to the A.B.O.N. First quarter earnings call.
Operator: At this time, all participants are in a listen-only mode. We will open the floor for questions and comments after the presentation. It is now my pleasure to turn the floor over to your host, Gregory Jinn, Vice President of Investor Relations and Corporate Communications at Aibiona. Please go ahead.
It is now my pleasure to turn the floor over to your host Greg region, Vice President of Investor Relations and corporate communications at the at E. B Ona. Please go ahead.
Gregory Gin: Thank you, Holly. Good morning, everyone.
Thank you Holly good morning, everyone.
Gregory Gin: And welcome and thank you for joining us on our first quarter, 2021 conference call. A press release announcing the first quarter results and recent operational progress is available on our website at www.abionattherapeutics.com. On the call today with prepared remarks are Michael Amaroso, Chief Executive Officer of Aviona, and Ed Carr, Chief Accounting Officer. After the prepared remarks, we will host a Q&A session, where we will be joined by Dr. Juan Ruiz, Vice President of Aibona, who heads up clinical development for our MPS programs, and Dr. Brian Kavani, a lead research scientist working on our preclinical I program.
And welcome and thank you for joining us on our first quarter 2021 conference call.
Press release announcing.
Press release announcing our first quarter results and our recent operational progress is available on our website at www Dot <unk> therapeutics Dot com.
On the call today with prepared remarks are Michael MRO, So chief Executive officer of avionics, and Ed Carr, Chief Accounting Officer.
After the prepared remarks, we will host the Q&A session.
We will be joined by Dr. Juan Ruiz Vice President at <unk>, who heads up clinical development for our NPS programs and Dr. Brian <unk> Elite research scientists working on our preclinical programs.
Before we start I will review, our safe Harbor statement.
Gregory Gin: Before we start, I will review our safe harbor statement. Remarks made during today's call may contain projections and forward-looking statements regarding future events. Forward-looking statements are made pursuant to the safe harbor provisions of the federal securities laws. Such forward-looking statements are based on current expectations and are subject to change. And actual results may differ materially from those expressed or implied in the forward-looking statements. Various factors that could cause actual results to differ include, but are not limited to:
Remarks made during today's call may contain projections and forward looking statements regarding future events.
Forward looking statements are made pursuant to the safe Harbor provisions of the Federal Securities laws. These forward looking statements are based on current expectations and are subject to change and actual results may differ materially from those expressed or implied in the forward looking statements.
Various factors that could cause actual results to differ include but are not limited to those identified under the section entitled risk factors in the company's annual report on form 10-K, and quarterly reports on form 10-Q filed by the company with the SEC.
Gregory Gin: Those identified under the section entitled to risk factors in the company's annual report on form 10K and quarterly reports on form 10Q filed by the company with the SEC. These documents are available on our website at www.abionatherapeutics.com. And with that said, I will now turn the call over to Michael.
These documents are available on our website at www Dot <unk> therapeutics Dot com.
That said I will now turn the call over to Michael Michael.
Thank you Greg good morning to our Investor community. We're excited to spend some time with you. This morning. Thank you for joining us.
Michael Amaroso: Thank you, Greg. Good morning to our investor community. We're excited to spend some time with you this morning. Thank you for joining us. As I approach the midpoint of my first 100 days in the job as CEO, I wanted to stop by reviewing three key strategic priorities for A. Bona and our team.
As I approach the midpoint of my first hundred days on the job as CEO.
Wanted to start by reviewing 3 key strategic priorities for <unk> and our team.
Yeah.
Michael Amaroso: First priority is to bolster the relevant operational experience on our management team and board, making sure we have the right coaches in place, the right experience to meet our goals going forward. Second, delivering operational excellence, both timely and fiscally disciplined, to further advance our in-clinic programs toward meaningful milestones for patients in need. And third, to further prioritize, execute, and advance our preclinical eye programs toward the clinic. We've made substantial progress on these priorities, and are off to a fast start in 21.
First priority being to bolster the relevant operational experience on our management team and board, making sure we have the right coaches in place the right experience to meet our goals going forward.
Second delivering operational excellence, both timely and fiscally disciplined to further advance our in clinic programs towards meaningful milestones for patients in need.
And third to further prioritize execute and advance our preclinical programs towards the clinic.
We've made substantial progress on these priorities and we're off to a fast start in 'twenty, 1 I want to thank the team for their efforts.
Michael Amaroso: I want to thank the team for their efforts. First priority one, continuing forward under the right leadership. We continue to focus on building the right talent and experience on our team, which positions us well to execute against operational goals. I'm very excited just this morning to have announced the appointment of Dr. Vishwashadri, a senior vice president, and our new head of research and clinical development. Vich, as he goes by, brings more than 20 years of experience across academia, followed by various senior and executive-level leadership roles within the life sciences industry.
First priority 1 continuing forward under the right leadership.
We continue to focus on building the right talent and experience of our team, which positions us well to execute against the operational goals.
I am very excited just this morning to have announced the appointment of Dr. Vishwa Chaudry, our senior Vice President and our new head of research and clinical development.
Vicious he goes by brings more than 20 years of experience across academia, followed by various senior and executive level leadership roles within the life Sciences industry.
Michael Amaroso: Overseeing product development, regulatory strategy, and teams for submissions, and commercialization for novel therapies, including most recently personalized autologous cell therapies in the CART space for cell gene and BMS. Vich has significant experience across early and late stage development, from first patient in, all the way through successful commercial launch. He understands the trade-off decisions and necessary discipline for evidence-based drug development. He is also a proven coach.
Overseeing product development regulatory strategy and teams for submissions and commercialization for novel therapies, including most recently personalized autologous cell therapies in the car T space for Celgene and BMS.
This has significant experience across early and late stage development from first patient in all the way through successful commercial launches.
He understands the trade off decisions and necessary discipline for evidenced based drug development.
This is also a proven coach excuse me.
Michael Amaroso: Excuse me, developing people across functions, through his project leadership in working with clinical, regulatory, medical, and commercial teams at Selgin. We're thrilled to have him join our team in early June, and we look forward to introducing VISH to you on future quarterly calls. Welcome Vish to the ABA and the team.
In developing people across functions.
Through his project leadership, and working with clinical regulatory medical and commercial teams at Celgene.
We're thrilled to have them join our team in early June and we look forward to introducing this to you on future quarterly calls welcome vish to the ABL and the team.
Michael Amaroso: Also, in the first quarter, we have strengthened our board of directors with the appointment of four new independent members as well as myself. The new members have experience sitting on boards of public and private companies and bring relevant operational leadership experience within the life science industry, including areas such as clinical development, manufacturing of Cell and Gene Therapy products, and corporate and financial compliance. The new members were selected based on their relevant and diverse experience, making them the right partners to the current management team in our pursuit of both bold near and long-term objectives.
Also in the first quarter, we have strengthened our board of directors with the appointment of 4 new independent members as well as myself.
The new members have experience sitting on boards of public and private companies and bring relevant operational leadership experience within the life science industry, including areas such as clinical development.
Manufacturing of cell and gene therapy products, and corporate and financial compliance.
The new members were selected based on their relevant and diverse experience, making them the right partners to the current management team and our pursuit of both bold near and long term objectives.
Michael Amaroso: I'd like to take a minute to review the new members and their critical experiences for ABOA. First, Dr. Leila Alland, a pediatrician, hematologist, oncologist, and physician scientist. Layla spent over 20 years in the biopharmaceutical industry, focusing on bringing novel therapies to patients with rare diseases, and is currently the chief medical officer at PMV Pharmaceutical. We're delighted to have Leila, and she'll be partnering closely with the Vission Team. Next, Don Worktel, who has over 29 years of experience in the life science industry, with senior roles in operations and CMC. Don is currently the chief manufacturing officer at T-knife Therapeutics. He has significant experience building and leading CGMP manufacturing organizations and facilities, so Don will provide essential feedback and guidance to our teams in Cleveland. Welcome, Don.
I'd like to take a minute to review the new members and their critical experiences for avionics.
First Dr. Laila, along a pediatric hematologist oncologist and physician scientist Leyla spent over 20 years in the biopharmaceutical industry, focusing on bringing novel therapies to patients with rare diseases and is currently the chief Medical officer at <unk> Pharmaceuticals.
We're delighted to have laila and she'll be partnering closely with vision team.
Next Dan worked all having over 29 years of experience from the life science industry with senior roles in operations in CMC.
Don is currently the Chief manufacturing officer at <unk> Therapeutics.
He has significant experience building and leading cgmp manufacturing organizations and facilities. So Dom will provide essential feedback and guidance to our teams in Cleveland Welcome Dan We're really excited to have you.
Michael Amaroso: We're really excited to have you. Next is Faith Charles, a corporate transaction and security partner at Thompson Hine with over 30 years of legal experience and who leads Thompson-Hind's life science practice, providing valuable insights to companies on capital markets, corporate governance, and strategic developments such as M&A, licensing transactions, and strategic collaboration. Welcome, Faith.
Next is fake Charles a corporate transaction and security partner at Thompson Hine with over 30 years of legal experience.
And lead Thomson Heinz life Science practice, providing valuable insights to companies on capital markets corporate governance, and strategic developments, such as M&A licensing transactions and strategic collaborations.
Welcome to fate.
And then last but never leased Mark Calvino.
Michael Amaroso: And last but not least, Mark Alvino. Mark has provided leadership and experience in the areas of financial management and business strategy as a member of the board of directors of multiple life science companies, including, previously, Ae Bona. Mark knows A. B.ona people and our products well, and his capital market experience will be invaluable, as well as his long-term perspective on our organization. We're excited to have Mark back on board.
Mark has provided leadership and experience in the areas of financial management and business strategy as a member of the board of directors of multiple life science companies, including previously avionics.
Mark knows <unk>, our people and our product as well and his capital market experience will be invaluable as well as his longitudinal perspective on our organization. We're excited to have mark back onboard.
Overall I wanted to express my excitement to announce the addition of these respected leaders.
Michael Amaroso: Overall, I wanted to express my excitement to announce the addition of these respected leaders, both to the management team and our board. I am confident that we have the right collective leadership in place that will help us to accomplish our objectives at this critical point in AB owner's life cycle. Excuse me, Monu.
Experienced both to the management team and our board.
I am confident.
Debt, we have the right collective leadership in place.
That will help us to accomplish our objectives at this critical point in <unk> lifecycle excuse me modal.
Let me take an additional minute to comment on our board's aimed to enhance and evolve our governance.
Michael Amaroso: Let me take an additional minute to comment on our boards' aim to enhance and evolve our governance. At our annual meeting of stockholders to be held tomorrow, our board recommends that stockholders vote for proposal two to approve the amendment of our restated certificate of incorporation to declassify the board and eliminate three-year terms for directors in favor of one-year terms. Thus further aligning with investor interest and increasing the accountability of our directors to shareholders. Our board believes that annual director elections are in keeping with sound corporate governance practices and promote additional accountability to shareholders.
At our annual meeting of stockholders to be held tomorrow.
Our board recommends that stockholders vote for proposal 2 to approve the amendment of our restated certificate of incorporation to declassify the board and eliminate 3 year terms for directors in favor of 1 year terms.
Thus further aligning with investor interest in increasing accountabilities of our directors to shareholders.
Our board believes that annual director elections are in keeping with sound corporate governance practices and promotes additional accountability to shareholders.
Michael Amaroso: Next, moving on to Priority 2, advancing our clinical programs toward key milestones for patients. I'll start with an update on EB-101, our lead pivotal program for recessive dystrophic epidermolysis bolosa, or as we refer to it as our dev. Regarding enrollment in the EB-101 vital trial, we remain on track to activate a second study site in the northeast to complement Stanford on the West Coast in the second half of 21, Patient and physician interest in the study continues to be high. And we're observing a greater willingness to travel among what we believe is a growing percentage of the population starting to receive vaccines for COVID-19.
If you have questions or need help voting for this very important proposal. Please contact Greg Ginn, our head of Investor Relations.
Next moving on to priority to advancing our clinical programs toward key milestones for patients.
I'll start with an update on <unk> hundred 1 our lead pivotal program for recessive dystrophic, Epidermolysis <unk> or as we refer to as our debt.
Regarding enrollment in the <unk> 101 vital trial, we remain on track for activating a second study site in northeast to complement Stanford on the West coast in the second half of 'twenty 1.
Patient and physician interest in the study continues to be high.
And we are observing a greater willingness to travel on what we believe is the growing percentage of the population starting to receive vaccines for COVID-19.
Next as discussed on our last call a fifth patient had been biopsy from the start of treatment.
Michael Amaroso: Next, as discussed on our last call, a fifth patient had been biopsied for the start of treatment, and this patient yielded a product that didn't meet required release criteria as per the Pivotal Trial Protocol. As a result, the clinical team of the patient has opted to rebiopsy and begin a new cell therapy product generation. We are hopeful to treat this patient in the coming week. Also, in parallel, additional patients have been identified and are being pre-screened to determine their eligibility for the vital trial entry criteria.
This patient sales yielded product that didnt meet required release criteria as per the pivotal trial protocol.
As a result, the clinical team and the patient had opted to re biopsy and begin a new cell therapy product generation ongoing we are hopeful to treat this patient in the coming weeks.
Also in parallel parallel additional patients have been identified and are being prescreened to determine their eligibility for the vital trial entry criteria.
Michael Amaroso: As a reminder, the target for this pivotal trial is the treatment of approximately 35 large chronic wounds across 10 to 15 patients. Next, the execution of our comparability plan is ongoing through our tech ops teams in Cleveland, as well as ongoing communication with the FDA. Thus far in 2021, overall progress for the EB-101 program has been significant, and we continue toward our goal of completing enrollment in the vital study by year-end.
As a reminder, the target for this pivotal trial is the treatment of approximately 35 large chronic wounds across 10 to 15 patients.
Next the execution of our comparability plan is ongoing through our tech ops teams in Cleveland as well as ongoing communication with the FDA.
Thus far in 2021 overall progress for the EB 101 program has been significant and we continue toward our goal of completing enrollment in the vital study by year end. Thank you to the teams in all patients.
Michael Amaroso: Thank you to the teams and our patients. Let us next turn our attention to our Adno Associated Virus, AAB-based gene therapy program, starting with our ABO 102 program in San Felipe Syndrome Type A or MPS3A.
Let us next turned our attention to our adenoma associated virus AAV based gene therapy programs.
Starting with our <unk> 102 program in Sanfilippo syndrome type a or MPS III.
We are preparing for the FDA type B meeting scheduled for next month in June.
Michael Amaroso: We are preparing for the FDA type B meeting scheduled for next month in June. As a reminder, we plan to discuss the data to date from the ABO 102 TransferA study and next steps for a potential BLA path with the FDA. We intend to discuss with the FDA whether the transfer-A data set could serve as the basis for a BLA submission with natural history data as a viable control. We believe historical controls are critically important for MPS treatment and drug development globally in this disease. As these children are being irreversibly impaired, irreversibly impaired, and it's not feasible to give them a placebo.
As a reminder, we plan to discuss the data to date from the ABL of 100 to transfer a study.
And next steps from a potential BLA path with the FDA.
We intend to discuss with the FDA, whether the transfer a dataset could serve as the basis for our BLA submission with natural history data as a viable control arm.
We believe historical controls are critically important for MTS treatment and drug development globally. In this disease. As these children are being irreversibly impair irreversibly impaired and it is not feasible to give them a placebo.
We are excited and hopeful to review the data with the FDA at our upcoming meeting and partner on a viable plan forward for patients.
Michael Amaroso: We are excited and hopeful to review the data with the FDA at our upcoming meeting and partner on a viable plan forward for patients. Also, the FDA feedback from our upcoming meeting will continue to guide and enhance our development plans and pathway to marketing authorization in Europe as well. Moving to our third in-clinic program, AB 101, the Transfer B study in San Felipe Syndrome type B or MPS3B.
Also the FDA feedback from our upcoming meeting will continue to guide and enhance our development plans and pathway to marketing authorization in Europe as well.
Moving to our third in clinic program.
101, the transfer B study in Sanfilippo syndrome type b or MPS <unk>.
Michael Amaroso: Product Stability Testing is discussed on the last call for the clinical product from nationwide Children's Hospital is now ongoing. To expedite timing, we brought some assay testing in-house. I want to thank our teams in Cleveland for their adaptability there.
Product stability testing as discussed on our last call for the clinical product from nationwide Children's Hospital is now ongoing.
To expedite timing, we brought some assay testing in house I want to thank our teams in Cleveland from their adaptability there.
Michael Amaroso: After completion of stability testing, we'll assess our options forward for treating the additional patients remaining for transfer B. During the first quarter, recent months, we also shared important clinical updates from both our transfer A and B studies at multiple medical meetings in Congress. This is highlighted by positive data at the 17th annual World Symposium on February 21, showing that neurocognitive development of young MPS3A patients was preserved up to three years following treatment with ABO 102.
After completion of stability testing, we will assess our options forward for treating the additional patients remaining for transfer day.
During the first quarter in recent months, we also share important clinical updates from both our transfer a and B studies at multiple medical meetings and Congresses.
This is highlighted by positive data at the 17th annual World Symposium in February 'twenty, 1 showing neuro cognitive development of young NPS 3.8 patients was preserved up to 3 years following treatment with <unk> 102.
In addition presented results from the transfer B study continued to show signals of biologic effect with reduction in disease specific biomarkers.
Michael Amaroso: In addition, presented results from the Trans-Rubb study continued to show signals of a biologic effect with reduction in disease-specific biomarkers in cerebral spinal fluid, plasma, and urine, and reduction in liver volume. We look forward to ongoing and additional updates on neurocognitive milestones.
In cerebrospinal fluid plasma in urine and reduction in liver volumes.
We look forward to ongoing and additional updates of neurocognitive milestones.
Michael Amaroso: Moving on to our third priority, prioritizing and advancing our preclinical programs, we're focusing resources on ophthalmic indications within our AAB platform following a strategic prioritization of our preclinical programs. On the back of our first AAB program success thus far in MPS, we're making significant progress toward adding new clinical programs to our pipeline. For example, we are conducting preclinical research with novel AAB capsids, including our wholly owned and partner capsids in six undisclosed eye indications. To give an idea to our investor team, the estimated prevalence for each of the eye indications ranges from 5 to 15,000 patients in the U.S. alone.
Moving onto our third priority prioritizing and advancing our preclinical programs.
We're focusing resources on our Palmer indications within our AAV platform following a strategic prioritization of our preclinical programs.
On the back of our first AAV program success, thus far in NPS, we're making significant progress toward adding new clinical programs to our pipe.
We are conducting preclinical research with novel AAV capsid, including our wholly owned and partnered cabinets and 6 undisclosed indications.
To give an idea to our investor team the estimated prevalence to each of the indications range.
The ranges from 5 to 15000 patients in the U S alone. This represents a significant market opportunity and more importantly, more patient lives we can help.
Michael Amaroso: This represents a significant market opportunity, and more importantly, more patient lives we can help. And this stays very true to leverage our identity as a fully integrated end-to-end gene therapy company for the highest unmet needs in patients. During the first quarter, we presented new data supporting the potential of cremediated dual AV vector technology to enable delivery of large genes targeted for treatment of Stargarts disease during an oral presentation at the Association for Research in Vision and Optomology, otherwise known as Arbo, 21's annual meetings.
And this is stayed very true to leverage our identity as a fully integrated end to end gene therapy company for the highest unmet needs in patients.
During the first quarter.
We presented the new data supporting the potential of Cree mediated dual AAV vector technology to enable delivery of large genes targeted for treatment of star God's disease. During an oral presentation at the association for research in vision and ophthalmology, otherwise known as ARVO 21 annual meeting.
In addition, we also recently completed nonhuman primate studies, comparing several captains with AAV 8 the current industry standard for intra ocular administration.
Michael Amaroso: In addition, we also recently completed non-human primate studies comparing several capsids with AAV8, the current industry standard for intraocular administration. I'm excited to say that the preclinical team delivered study results about four weeks early, showing that Aav2. Our partner capsid, in license from our AIM library, was superior to AAB8 using a recently developed novel route to ocular administration. In a separate non-human primate experiment, we tested our AAB 214 and our AAB 214 D5, two wholly owned aviona caps, versus Aav8 by subretinal dosing administration. Both capsids demonstrated nearly identical levels of transduction compared with AAB8 in photoreceptor and retinal pigmented epithelium cells, which are the cell types most frequently affected in inherited retinal diseases.
I'm excited to say that the preclinical team delivered study results about 4 weeks early showing that AAV tool for our partnered captured in licensed from our aim library was superior to AAV 8 using a recently developed novel route to ocular administration.
In a separate nonhuman primate experiments.
We tested our AAV $2.14, and our AAV $2.14 day 5.2 wholly owned <unk> cabinets versus AAV 8 by sub retinal dosing administration.
Both caps, it's demonstrated nearly identical levels of transduction compared with AED 8 a photo receptor and retinal pigmented epithelium cells, which are the cell types. Most frequently affected in inherited retinal diseases.
We are very excited about the findings of these experiments, which provide critical insight into the ability of our novel capsid to penetrate key sales and the optimal route of administration to accomplish this penetration.
Michael Amaroso: We are very excited about the findings of these experiments, which provide critical insight into the ability of our novel capsids to penetrate key cells and the optimal route of administration to accomplish this penetration. These results position us well to move rapidly into proof of concept studies in the second half of this year, followed by toxicology studies in the first half of 22. One of our lead scientists, Brian Kavani, has joined us this morning, and he can help us answer any questions we might have about some of this very significant pre-clinical progress.
These results position us well to move rapidly into the proof of concept studies in the second half of this year followed by toxicology studies in the first half of 'twenty 2.
1 of our lead scientists, Brian Covina has joined US This morning, and he can help us answer any questions. We might have about some of this very significant preclinical progress.
Michael Amaroso: With that, I'll now turn the call over to Ed, our chief accounting officer, to review our financial progress thus far in 21. Please, Ed. Thank you, Michael. I would like to remind everyone that Form 10 is available on our website, where you can get the details on our Q1, 2021 financial results. In summary, our cash, cash equivalence, and short-term investments as of March 31, 2012 totaled 86.8 million compared to 95 million as of December 31, 2020.
With that I'll now turn the call over to Ed Our Chief Accounting Officer to review, our financial progress thus far in 'twenty..1. Please it. Thank you Michael I would like to remind everyone that the form 10-Q is available on our website, which is where you can get the details on our Q1.2021 financial results.
In summary, our cash cash equivalents and short term investments as of March 31, 2021 totaled $86.8 million compared to $95 million as of December 31, 2020.
Ed Carr: For the first quarter of 2021, net cash used in operating activities was 13.6 million, compared to 13.2 million for the first quarter of 2020.
For the first quarter of 2021 net cash used in operating activities was $13.6 million compared to $13.2 million for the first quarter of 2020.
Ed Carr: We believe we have sufficient cash resources to build on our momentum and fund our current development and operating plans through achievement of key anticipated milestones, including multiple potential regulatory submissions.
We believe we have sufficient cash resources to build on our momentum and fund our current development and operating plans through achievement of key anticipated milestones, including multiple potential regulatory submissions.
And with that I will now turn it over to the operator for Q&A.
Operator: And with that, I will now turn it over to the operator for Q&A. Ladies and gentlemen,
Ladies and gentlemen, the floor is now open for questions. If you have any questions or comments. Please press star 1 on your phone at this time, we ask that while posing your question you. Please pickup your handset listening on speaker phone to provide optimum sound quality. Please.
Operator: Ladies and gentlemen, the floor is now open for questions. If you have any questions or comments, please press star 1 on your phone at this time. We ask that while posing your question, you please pick up your handset or listen on speakerphone to provide optimum sound quality. Please hold while we poll for questions. Your first question for today is coming from Maury Raycroft. Please announce your affiliations and pose your question.
Hold while we poll for questions.
Your first question for today is coming from Maury Raycroft. Please announce your affiliation and pose your question.
Hi.
Maurice Thomas Raycroft: Hi, good morning everyone. This is Morrie Redcroft from Jeffries on the line.
Morning, everyone. This is more of your Raycroft from Jefferies on line.
Maurice Thomas Raycroft: Thanks for taking my question. So, first question is on EB-101. Just wondering if you can provide any more specifics into why the product didn't meet release criteria for patient number five. And I think you mentioned, too, that you're running a comparability program. Can you just remind me what the purpose of that is and how often, I guess, what the outcome of that is? I think you mentioned the comparability program in FDA,
Thanks for taking my questions. So first question is on EV 101.
I'm just wondering if any more specifics in Q.
Why the product.
These criteria from page number 5.
I think you mentioned 2 that Youre running a comparability program can you just remind me what the purpose of that is and how often.
I guess, what's the outcome of that I think you mentioned comparability program with FDA, but I missed a little bit of it.
And in the middle there.
Sure Maury good to hear your voice.
Michael Amaroso: Sure, Maury, good to hear your voice. Michael, I'll take that one.
This is Mike I'll take that 1 yes. So so patient 5 we've got about 13 different release criteria that obviously the product goes through before we were able to send over and release for the surgical administration.
Michael Amaroso: Yeah, so patient five, we've got about 13 different release criteria that obviously the product goes through before we are able to send it over and release it for the surgical administration The patient cells, as you remember, we grow the cells up, and we use an enzymatic cleavage to take the sheet off and put it on our gauze with hemoclips to deliver them to the OR. The protocol for Pivotal states that the sheets can't even have any pinholes in them at all.
The patient cells as you remember we grow the sales out and we use an enzymatic cleavage to to take the sheet off and put it on our goals with hemoglobin to deliver it to the or.
The protocol for pivotal states that the sheets can't even have any pinholes in them at all that being said, while the surgical administration occurs we do make sutures and the product obviously to put these grafts onto patients, but the the protocol with the FDA in the agreement as of today. Unlike what was in the phase 1.2 says you can't even have a pinhole initiatives cymric tenant sites.
Michael Amaroso: That being said, while the surgical administration occurs, we do make sutures in the product, obviously, to put these graphs onto patients. But the protocol with the FDA in the agreement as of today, unlike what was in Phase 1-2, says you can't even have a pinhole in these sheets. Kyrgynincetocytes are known to close over themselves within their borders to kind of cover these pinholes. So this is something we're working on in the Phase 4 work, but it is a pivotal trial. There is a stringent selection criteria, which we appreciate.
None to close close over themselves within their borders to kind of cover. These panels. So this is something we're working on in the phase 4 work, but it is a pivotal trial there was a stringent criteria, which we appreciate so we had some holes in the product in small panels and at that point in time, what we decided to do was rather than yield lesser product to the to the or we decided to rebound.
Michael Amaroso: So we had some holes in the products of small pinholes, and at that point in time, what we decided to do was, rather than yield a lesser product to the OOR, we decided to re-biopsy to try to give the patient the best ability to have a maximum delivery of transplanted area. So this is something that we, you know, with autologous therapy, cells grow a bit differently. We've seen this before, and, you know, this is not something that's uncommon for us.
So to try to give the patient the best ability to have maximum delivery of transplanted area. So this is something that we with autologous therapy sales grow a bit differently. We've seen this before.
This is not something that is uncommon to us but this is this is why we decided to re biopsy the patient to answer your comparability question. Just a reminder, all of our product is made in house, our tech ops and Cleveland goes end to end Theres no CMO dependency however.
Michael Amaroso: But this is why we decided to re-biopsy the patient. answer your comparability question just a reminder all of our product is made in-house our tech ops in Cleveland goes end to end there's no CDMO dependency however right now as part of the ingredients for transduction we use academic or Indiana retrovirus okay and in order to have full control of our supply chain as we move to move to commercialization the team in Cleveland has built our own retrovirus a b owner retrovirus, So the comparability is a plan that was agreed upon with the FDA that within the pivotal, we will move from the first half of our patients that are made with product from Indiana retrovirus to the abiona retrovirus.
Right now as part of the ingredients for transduction, we use.
Academic or Indiana, retrovirus, Okay and in order to have full control of our supply chain as we move to move to commercialization. The team in Cleveland has built our own retrovirus ABL and a retrovirus. So the comparability is a plan that was agreed upon with the SBA debt within the pivotal we will move from the first half of our patients that are made with product from Indiana retro.
Chris to the owner retrovirus, so while we're still treating patients clinically with the Indiana retrovirus ingredient that has.
Michael Amaroso: So while we're still treating patients clinically with the Indiana retrovirus ingredient, that's, you know, it helps us transduce and make our keratinocyte sheets. At the same time, we're doing analytical comparability to show the similarity to the abiona retrovirus, and our final five patients will be dosed with the abiona retrovirus made process. So that's why we have the comparability protocol, Mori.
It helps us trends do some make up <unk> at the same time, we're doing analytical comparability to show the DTD similarity to the AAV owner retrovirus and our final 5 patients will be will be dosed with the ABL and a retrovirus made product. So that's why we have the comparability protocol.
Got it it makes a lot of sense and thanks for all the perspective there.
Michael Amaroso: Got it. Makes a lot of sense, and thanks for all the perspective there. Second question is just on the Transfer A type B meeting with FDA in June. Just wondering if you could talk a little bit more about the plan going into the meeting, who's going to be attending with you, and also the disclosures from the meeting. I guess, will you get minutes from that, and when could we expect some sort of an update?
Thank you.
And true.
For a type B meeting with FDA in June.
Im just wondering if you could talk a little bit more about the plan going into the meeting who is going to be attending with you and also the disclosure from the meeting too I guess.
You get minutes from that end.
When could we expect some sort of an update from that meeting.
Michael Amaroso: Yeah, sure. So, Maury, I'm going to turn this question over to you.
Yes, sure. So Maury I am going to turn this question over I'll give a quick highlight im going to turn it over to my clinical lead of the program 1 reasons on the phone, but I think the key here is analyzing our pivotal endpoint, our data and making sure that we'd be growth that topic, we've talked about before very important that natural history becomes we think we've got the biggest data set here that.
Michael Amaroso: I'll give a quick highlight. I'm going to turn it over to my clinical leader, the program director, Juan Ruiz, is on the phone. But, you know, I think the key here is analyzing our pivotal endpoint, our data, and making sure that we broach that topic we've talked about before. Very important that natural history becomes, we think we've got the biggest data set here, a viable control on, you know, for a path forward to a BLA.
Natural history becomes a viable control arm for a path forward to a BLA, but 1 why don't you give a quick overview of what we look to accomplish objective wise in the type B meeting toward the end of June and some of the partners that will join us with the FDA.
Michael Amaroso: But, Juan, why don't you give a quick overview of what we look to accomplish objective-wise in the type B meeting toward the end of June and some of the partners that will join us with the FDA?
Juan Ruiz: Yeah, thank you, Michael. Yeah, thank you, Mori, for the question.
Yes. Thank you Michael Thank you Martin for the questions are important.
We have Scott reviews, and meetings, we view EBITDA and we have been implementing.
Juan Ruiz: We have had previous meetings with the FDA, and we have been implementing the outcomes of those meetings and recommendations from the FDA. But the main purpose of the meeting this June is to agree with the NDA on the way we propose to analyze the primary endpoint, the use of a natural history study as a comparator group precisely to analyze the changes that we see in patients treated with AVO 102. And also, what is the key?
Outcomes from the dose on recommendation from the NDA, but the main purpose of the meeting this June.
Agree with the FDA on the the way we proposed to analyze the primary endpoint the use of natural history study as a comparator group.
Precisely to analyze the changes that we see in basis, so it could be 1 or 2.
On.
And also.
Juan Ruiz: clinical and meaningful difference that we propose in order to declare success on the program. Those are the main aspects that we are going to discuss in addition to the use of putting together different natural history studies and testing different tools. We have the data to support that and discuss the statistical analysis plan that we have provided. Those are mainly the aspects we want to discuss. As I said, that have been previously discussed, implemented some of the recommendations, and provided answers to pending questions.
What is a clinically meaningful difference that we proposed in order to declare success on.
On the program those are the main.
Aspects as we are going to discuss in addition to the use of <unk>.
Putting together different from natural history studies and testing different tools, we have the data to support debt.
Discuss the statistical analysis plan that we have provided those are mainly to the aspect of what we want to discuss as I said there had been previously discussed implementing some of the recommendations.
Unprovided unsettled pending questions.
Thank you Ron.
Juan Ruiz: Got it. That's helpful. And so could we expect an update potentially in the July-August timeframe as the outcome from the
Got it that's helpful and so could we expect an update potentially in July August timeframe.
Michael Amaroso: Yeah, Maury, I think on our next call we'll definitely be talking about the results of our type B meeting. That'll be an important way for us to communicate.
The outcome from the meeting.
Yes, Maury I think on our next on our next call, we'll definitely be talking about the.
The results from our type B meeting that will be an important path for us to communicate.
Okay. Okay. Thanks for taking my question.
Michael Amaroso: Okay, okay, thanks for taking my question.
Thank you.
Your next question is coming from Kristen Clark Scott. Please announce your affiliation then pose your question.
Kristen Brianne Kluska: Your next question is coming from Kristen Kluxka. Please announce your affiliation, then ask your question.
Hi, Good morning. This is Christine cluster of Cantor Fitzgerald I wanted to ask a question on EV 101, so on the backs of some of the recent data you shared a S. D. How are you thinking about the durability of VB 101 across the different wound types evaluated and how important do you think this durability.
Kristen Brianne Kluska: Hi, good morning. This is Kristen Kluska of Cantor Fitzgerald.
Kristen Brianne Kluska: I wanted to ask a question on EB-101. So, on the back of some of the recent data you shared at SID, how are you thinking about the durability of EB-101 across the different wound types evaluated? And how important do you think this durability data is going to be for physicians and a sales team to promote in choosing a potential treatment option should multiple therapeutics become approved? And I know there are a lot of others.
Data is going to be for physicians and our sales team to promote in choosing a potential treatment option should multiple therapeutics become approved and I know theres a lot of other factors to consider here like wound type and size, but I specifically wanted to hear your thoughts on durability.
Michael Amaroso: There are factors to consider here like wound type and size, but I specifically wanted to hear your thoughts on durability.
Yes, Kristen nice to hear your voice. Thanks I. Appreciate the question, so you're making me smile, because I know we've talked a lot about this so yes, I mean, I think you said it really really well right. So a couple of things first of all we're hoping.
Michael Amaroso: Yeah, Kristen, nice to hear your voice. Thanks, I appreciate the question. So you're making me smile because I know we've talked a lot about this. So yeah, I mean, I think you said it really, really well, right? A couple of things.
Michael Amaroso: First of all, we're hoping, very much hoping that there'll be many, many options here over the next five, 10 years for patients with our disease. I mean, we've had nothing in this space for the last three to four decades, and that's not giving us the progress we need for these children and young adults. The backbone of EB-101 is durability. That's the positioning of the product.
Very much hoping that there'll be many many options here over the next 510 years for patients with all our debt I mean, we've had nothing in this space for the last 3 to 4 decades, and that's that's not given the progress we need for these children and young young adults.
The backbone of VB 101 is durability, that's the positioning of the product. The reality here is there is there is a small surgical administration like a graft.
Michael Amaroso: The reality here is, you know, there is a small surgical administration like a graft, and the product has to be worth it, and we believe it very much is. You know, the focus of our product and our trial is R-Db, the worst of the worst types of EB, as well as the worst types of wounds. We say about 50% of the wound burden of R-DB patients at any given time is a more advanced wound, large, 20-centimeter-squared or ground. We have some that are hundreds of centimeters and tire backs, for example, and chronic. This is a very important point, Kristen, that you brought up about different wounds. A chronic wound is one that can no longer heal itself. Six months or longer it's been open.
The product has to be worth it and we believe it very much is.
The focus of all product in our trial is in our debt the worst of the worst types of DB as well as the worst type of wounds about FID, we say about 50% of the wound burden Navarre debt patients at any given time is a more advanced wound large 20 centimeter squared or greater we have some that are hundreds of centimeters entire backs for.
Simple and chronic this being a very important point Kristen that you brought up different wounds.
Chronic is a wound that can no longer heal itself 6 months or longer. It's been open. These of the wounds that patients suffer the greatest morbidity pain infection malnutrition as well as lead to things like squamous cell carcinomas.
Michael Amaroso: These are the wounds that patients suffer the greatest morbidity, pain, infection, malnutrition, as well as lead to things like squamous cell carcinomas and, you know, down the line, and these are the things that, unfortunately, lead to patients meeting their demise. So we're focusing on what we believe is the worst of the worst wounds, about 50% of the wound burden on a patient at any given time. Some of the other research going on today is very important.
Down the line and these are the things that unfortunately.
Lead to patients meeting meeting their demise. So we're focusing on what we believe is the worst of the worst wounds about 50% of the wound burden on a patient any given time some of the other research going on today is very complementary we truly don't believe there is a competition in this space we need to be partnered for these patients that have no options. So when you look at some of the other trials for <unk>.
Michael Amaroso: complementary we truly don't believe there's a competition in this space we need to be partnered for these patients that have no options so when you look at some of the other trials for example they're looking at other types of EB many of the trials are looking at small recurrent wounds the way I describe it is the evolution of the wounds in our Deb start as small blister clusters and ultimately these wounds will evolve as the disease progresses and they become these larger chronic wounds so Kristen you could see a world where if we had two three four I hope ten products approved Over the 35 years of a patient's lifespan, 30, 35 years, a lot of times these patients meet their demise, they'll have experienced several therapies in the continuum of disease. But when the wound becomes truly problematic, the highest morbidity, mortality, these large wounds, they will need what we think is the biggest tool in the tool belt.
Sample they are looking at other types of VEB. Many of those trials are looking at small recurrent wounds. The way I describe it is the evolution of the wounds in our debt started small blister clusters and ultimately these wounds will evolve as the disease progresses and they become these larger chronic wounds, so Chris and you could see a world where if we had 234 I hope 10 products approved.
<unk>.
Over the 35 years of a patient's life span 30, 35 years a lot of times. These patients meet their demise they'll have experienced several therapies in the continuum of disease, but when the wound becomes truly problematic the highest morbidity mortality of these large wounds they will need but we think is the biggest tool in the tool belt that will be <unk> 101.
Michael Amaroso: That will be EB-101 because they need long, extended periods of covering these wounds. And what we're hopeful for, Kristen, as you know, the vital trial looks at wound closure and pain improvement. I'm sure you can make improvements so that you're not opening these wounds. But ultimately, we hope that as we collect this data in registries and over time, if we close enough of these most problematic, large, chronic wounds, we hope that will change the prognosis of mortality for these patients. So, Chris, that gives you a little bit of an idea of the positioning of the product as durability, the wound it's specific for, and how it will complement other things being researched in the landscape today.
Because they need long extended periods of covering these wounds and what we're hopeful for Kristen as you know the vital trial looks at wound closure and pain improvement morbidity.
Each improvements so that youre not opening these wounds, but ultimately we hope that as we collect this data and registries in time, if we close enough of these most problematic large chronic wounds, we hope that will change the prognosis of mortality for these patients. So Chris net gives you a little bit of an idea of the positioning of the product being durability the wound.
It's specific for and how it will we will complement other things being researched from the landscape today.
Yes.
Thanks, and I know you've discussed the key differences between the recurring and chronic wounds, but I wanted to ask if you're researchers and your team are doing some more work to better understand when the best time to intervene with this treatment might be for some other recur recurrent wounds excuse me specifically just in light of the fab.
Michael Amaroso: Thanks, and I know you've discussed the key differences between the recurrent and chronic wounds, but I wanted to ask if your researchers and your team are doing some more work to better understand when the best time to intervene with this treatment might be for some of the recurrent wounds, excuse me, specifically, just in light of the fact that the different patterns in terms of time to healing, time to re-blistering, et cetera, just to help with the greatest chance of success if commercialized.
To that.
The different patterns in terms of time to healing time, Jerry blistering et cetera.
But the greatest chance of success if commercialized.
Yes, it's an excellent question Kristen I think the question you ask here is what is earlier intervention, while it why let these wounds get to this point right. If we've got tools what is the right time for intervention. So we are the scientific question hypothesis is out there. We are asking the question. We do plan on studying it in the future some of the things like if I could talk about our phase 4 work a lot.
Michael Amaroso: Yeah, it's an excellent question, Kristen. I think the question you ask here is, what is earlier intervention? Why let these wounds get to this point, right? If we've got tools, what is the right time for intervention? So we are, the scientific question hypothesis is out there. We are asking the question.
Michael Amaroso: We do plan on studying it in the future. Some of the things like, I talk about our phase four work a lot. Some of that phase four work, I always call those practicality studies, things that are really important to add to our pivotal package, things like showing that hey, a sheet with a pinhole can absolutely be transplanted and have the same durability. Kyrgyzumocytes will close over.
Some of that phase forward I always cobos practicality studies things that are really important to add to our pivotal package things like showing hey, a sheet with a pinhole can absolutely be transplanted and have the same durability <unk> will close over things like getting patients out of the hospital faster because we know patients who have the right care at home we.
Michael Amaroso: Things like getting patients out of the hospital faster, because we know patients who have the right care at home; we want them to make sure they have that option, so they don't have to stay in Hoppy. And right now, they stay in the hospital, just so a wound nurse makes sure they're not laying on their engraftments. Things like Kristen, where you just were, recurrent wounds.
We want them to make sure they have that option. So they don't have to stay in heartburn right now they stay in the hospital just so a wound nurse make sure they're not laying on their <unk> things like Crystal and where you just were recurrent wounds. If we can treat the most problematic wounds.
Michael Amaroso: If we can treat the most problematic wounds, we have a lot of hypotheses that say EB-101 would be able to do really well with recurrent wounds. We absolutely I absolutely believe that, but we'll look to prove that in the future. As you know, we're starting in our Deb, enlarge, and chronic. But these are things that I call kind of our complementary phase four work that, you know, bringing Vichon now as our head of R&D will be really important for us to start to continue to do the studies that matter to show this data.
We have a lot of hypothesis that says <unk> 101 would be able to do really well with recurrent wounds, we absolutely believe that but we will look to prove that in the future. As you know were starting in our Deb and large and chronic but these are things that I call kind of our complementary phase 4 work work debt.
Bringing vishal now as our head of R&D will be really important that we start to continue to do the studies that magnitude to show this data.
Great. Thanks, and then on your prepared remarks, you noted that some patients for the phase III trial have been identified or in the pre screening process. Just wanted to make sure I understood that this was specifically referring to Stanford and then I know you haven't identified the specific center in the northeast could you talk.
Michael Amaroso: Great, thanks. And then in your prepared remarks, you noted that some patients for the phase three trial have been identified or are in the pre-screening process. Just wanted to make sure I understood that this was specifically referring to Stanford. And then I know you haven't identified the specific center in the Northeast, but could you talk about maybe, you know, more details? Like, is this a center that you would expect a lot of patient interest in, and have the investigators there potentially identified patients that they might want to go through the screening process?
About maybe you know more details like is this a center that you would expect a lot of patient interest and have the investigators there potentially identify patients that they might want to go through the screening process.
Michael Amaroso: Yes, Kristen, you nailed it. That's exactly right. So we've got some patients that are identified for Stanford, but we also have started a bench for the Northeast site. And I don't mean to be coy, guys. I'm just not announcing the site as per their IRB request until it's fully approved. I hope the next time we talk, I'll be able to do that.
Yes, Chris and you nailed it that's exactly right. So we've got some some patients that are identified for Stanford, but we also have started a bench for the northeast site and I don't mean to be coy guys. I'm, just not announcing the site as per their IRB ask until it's fully approved I hope. The next time, we talk I'll be able to do that with a site in the northeast is also screening and looking for some patients right.
Michael Amaroso: But a site in the Northeast is also screening and looking for some patients right now; patients chart their KOL there. When you think about what that site would look like, what's an ideal site as we onboard? We've talked about in the past, you might remember, will probably come out of the gates with at least 10 sites when we commercialize. But when you think about this second study site, an important step in progress toward that. These are EB clinics, ongoing EB clinics. Why?
Now patient charts, there kols there when you think about what that site would look like what's what's an ideal site as we onboard we've talked about in a few in the past you might remember, we'll probably come out of the gates with at least 10 sites when we commercialize but when you think about the second study site and important an important step in progress toward that these are EBIT clinics ongoing you'd be clinics.
<unk>. This is where we currently have <unk> patients today. This is where you see the evolution of the disease of these patients when they need products like <unk> 101. This way you have anesthesiologists surgeons, who understand how to give anesthesia to a patient who may have an esophageal esophageal stricture. The dilation procedures go on the other hand foot.
Michael Amaroso: This is where we currently have EB patients today. This is where you see the evolution of the disease in these patients when they need products like EB-101. This is where you have anesthesiologists, and surgeons who understand how to give anesthesia to a patient who may have an esophageal stricture, the dilation procedures go on there, the hand and foot surgeries go on there.
<unk> go on there. So right now these are academic type centers, who are specializing in EV and they have the volume of the <unk> patients that gives you an idea of what the profile of that northeast site will be very similar to Stanford and yes.
Michael Amaroso: So, you know, right now these are academic-type centers who are specializing in EB, and they have the volume of EB patients. That gives you an idea of what the profile of that northeast site will be, very similar to Stanford. And yes, when we talked about the additional patients being prescreened right now, we've got a few in the queue, and we hope they meet eligibility criteria. That's out of Stanford, but our lead KOL out of the new site is also profiling some patient charts.
When we talked about the additional patients being prescreened right now.
Got a few in the queue, we hope they meet eligibility criteria that's out of Stanford, but our lead kols out of the new site is also profiling some patient charts. So once we have decided already.
Michael Amaroso: So once we have the site ready to move, we can go as fast as we can to get those patients into the trial and treated. And what I'll remind you of is our capacity outside of Cleveland while we're in the clinical stages. We can treat up to two patients per month, so that's really, really important. And as we commercialize, we'll be making some changes to our site. We'll be dedicating our first site solely to EB-101.
To move we can go as fast as we can to get those patients into the into the trial and treated and what I'll remind you is our capacity out of Cleveland, while we're in clinical stages, we could treat up to 2 patients per month. So that's really really important and as we commercialize we will be making some configuration to our site will be dedicating our first site solely.
2 <unk> hundred 1 and at that point, we'll be able to produce about 120 products in the first year moving to about 500 product. If we have the demand in the second and third years. So we've got the scale to be able to meet 2 sites ongoing as well as getting ready for first year of commercial launch.
Michael Amaroso: And at that point, we'll be able to produce about 120 products in the first year, moving to about 500 products if we have the demand in the second and third year. So we've got the scale to be able to meet two sites ongoing, as well as getting ready for the first year of commercial production. Great, thanks so much for taking my questions.
Great. Thanks, so much for taking my questions.
Of course, thank you.
Your next question is coming from money for horror. Please announce your affiliation and pose your question.
Unknown Attendee: Your next question is coming from Moni Furhar. Please announce your affiliations and pose your question.
Okay monitor our fence tubular goods, taking the call.
Unknown Attendee: It's the Monitur Festival, and they've taken received a call. I was going to ask them the question. So a lot of the focus is obviously on the existing clinical opportunities, but I'll learn to put it a little bit more on the vector platform. And how you think about further opportunities in terms of BD, raising further non-dolidavit of capital and partnerships using AIM vectors that you guys already own currently. And as we think about that potential store of value. Imani, how are you doing? Thanks for the question. So,
Sure.
So a lot of focus obviously on the existing critical opportunities.
I wanted to pivot a little bit to the vector platform.
As you see how you think about further opportunities in terms of BD.
Further non dilutive capital and partnerships using aim vectors that you guys already own currently and how do we think about that potential store value for the company.
The amount of how are you doing thanks for the question so.
Michael Amaroso: Hi Mani, how are you doing? Thanks for the question. So, good question. And I think the key word for me that jumps out is optionality, right?
Good question.
The key word for me that jumps out is optionality right. We are a small cap we've got the funding right now and in the experience and the capital discipline.
Michael Amaroso: We're a small cap. We've got the funding right now, and we have the experience and the capital discipline. As Ed says, we believe we could take ourselves through our first clinical programs or in-clinic programs to literally regulatory milestones if need be. But at the same time, I've said this before, we're always looking for ways to bolster either our capability, which I always think about as our timing to patients, you know, our science, and or our financial opportunities, our financial runways, right? That's, you know, Moni, you ask a question that's really important.
As Ed says that we believe we could take ourselves through our our first clinical programs are Inc. Are in clinic programs to to literally regulatory milestones of Navy, but at the same time Ive said this before we're always looking for ways to bolster our either our capability, which I always think about is our timing to patients our science and our <unk>.
<unk> opportunities our financial runway right. That's Mani who asked a question that's really important our financial runway as is.
Michael Amaroso: Our financial runways is a reality that we think through every day, and we try to be very surgical, if you will, in our approach. So I think when you look at our profile, if you think about the identity of the company, and I love this question, I think our identity is an end-to-end fully integrated gene and cell therapy company, self-therapy, of course, being within the umbrella of gene, that starts with an autologous program, but I think then you immediately see, and that's why I think the MPS program is so important for human beings, first and foremost, but also so important to prove some muscle of our AAB platform on non-autologous gene therapy program.
It is a reality that we think through every day and we tried to be very surgical if you will in our approach. So I think when you look at our profile. If you think about the identity of the company and I Love. This question.
I think our identity is an end to end fully integrated gene and cell therapy company cell therapy of course being within the umbrella of gene.
And that starts with an autologous program, but I think then you immediately see and that's why I think the NPS program is so important for human beings first and foremost, but also so important to prove some muscle of our AAV platform. Our non autologous gene therapy program and then from there I think about the aim capsid library in the follow on and how that's yielding some early excited.
Michael Amaroso: And then from there, I think about the AIMCAPS library and the follow-on and how that's yielding some early excitement for us in the eye. And that's the way we're looking at Amani, a progression now that our identity is a gene therapy company, that we are, you know, we look at the ability to produce for MPS products. The expansion of our facility will be able to produce for MPS and the I.
And for us in the eye and that's the way we're looking at ammonia is a progression now that our identity as a gene therapy company that we are we look at the ability to produce for MPS product. The expansion of our facility will be able to produce for NPS MDI, but I think we're very open to the right partners 2 we realized.
Michael Amaroso: But I think we're very open to the right partners too. We realize that we're not going to be a jack of all trades and a master of none. We're going to look to specialize in our key area, which today is gene therapy, as well as ultra-rare diseases. I like how we're pivoting away from ultra-rare diseases. We're staying focused on maybe the highest unmet needs, where that opens up bigger market opportunities.
Debt, we're not going to be a jack of all trades and a master of none.
We're going to look to specialize in our key area, which today is gene therapy as well as ultra rare diseases I like how we're pivoting from ultra rare diseases. We're staying focused on maybe from ultra rare to highest unmet needs where that opens up bigger market opportunities and if there is great partners in the ophthalmic space or in the neuro card space.
Michael Amaroso: And if there are great partners in the ophthalmic space or in the neurocog space, you know, for MPS, for these eye indications, or even in the germ space, I think we're always looking at those partners. I think that's something that's our responsibility to shareholders. But hopefully, walking you through that a little bit is kind of how we see the evolution of our organization and that we're fully dedicated to these initial milestones clinically as well as expediting the pre-clinical.
For NPS for these indications or even in the derm space I think we're always looking at those partners I think that that's something that's our responsibility to shareholders, but hopefully be walking you through that a little bit is kind of how we see the evolution of our organization and and.
Debt, where we're fully dedicated to these initial milestones clinically as well as expediting. The preclinical work we've made really good progress on moving faster to get towards an IND in these indications first prioritizing them and getting through the nonhuman primate experiments, we think that makes us that much more attractive when we're sitting at the table to say Hey, we think we've got some some leading science in these.
Michael Amaroso: I think we've made really good progress on moving faster to get toward an I-ND in these eye indications, first prioritizing them and getting through the non-human primate experiments. We think that makes us that much more attractive when we're sitting at the table to say, Hey, we think we've got some leading science in these areas. And if the right partner is involved, then we could sit and have that conversation, too. So that's how we're thinking about it, Moni.
Areas and if the right partners involved and we could sit and have that conversation to so that's how we're thinking about it Marty.
Thanks, that's really helpful.
Thank you.
I would now like to turn the floor back over to Michael for any closing remarks.
Operator: I would now like to turn the floor back over to Michael for any closing remarks.
Michael Amaroso: Well, thank you, Operator. Thank you for the operator of the call.
Well. Thank you operator, thank you for the operating the call. Thank you for Greg for setting it up and Ed and the team. Thank you to the AVR team. That's made really significant progress since since I've been working with them in late last year.
Michael Amaroso: Thank you to Greg for setting it up and Ed and the team. Thank you to the A.B.O.A. team that's made really significant progress since I started working with them late last year.
Michael Amaroso: I want to always thank our patients and our staff for having faith and trust in us for their children or their loved ones. And we will stay committed. Thank you to the investor community for your interest, your honesty, and your questions. We'll continue to be transparent with you. We'll continue to update you on progress as we make those many milestones. And if there are no further further questions, then I turn it over to you, operated to dismiss us today. Thank you and look forward to chatting again soon.
I want to always thank our patients and our families for having the faith and trust in us.
Their children or their loved ones.
And we will stay committed thank you to the Investor community for your interest your honesty. Your questions. We will continue to be transparent with you. We'll we'll continue to update you on progress as we meet those milestones.
And.
If no. Other further questions then I'd turn it over to you operator to dismiss US today. Thank you and look forward to chatting again soon.
Thank you ladies and gentlemen, this does conclude today's conference call. You may disconnect. Your phone lines at this time and have a wonderful day. Thank you for your participation.
Operator: Thank you, ladies and gentlemen. This does conclude today's conference call. You may disconnect your phone lines at this time and have a wonderful day. Thank you for your participation.