Q2 2020 Abeona Therapeutics Inc Earnings Call
Please standby we are about to begin.
Operator: Please stand by. We are about to begin. Good day and welcome to Abeona Therapeutics' 2nd Quarter 2020 Financial Results and Business Update Conference Call. All lines have been placed in a listen-only mode, and the floor will be open for questions following the presentation.
Good day and welcome to 80 on that Therapeutics second quarter Twentytwenty financial results and business update conference call. All lines have been placed in listen only mode, and that's where we'll be open for questions. Following the presentation.
Operator: If you should require assistance throughout the conference, please press star zero. As a reminder, today's conference is being recorded. I'll now introduce your host for today's conference, Greg Gin, Vice President of Investor Relations at Abeona. Please go ahead.
Should require assistance throughout the conference Please press Star zero.
As a reminder, today's conference is being recorded introduce your host for today's conference right.
Hi.
That's relations at FBR. Please go ahead.
Thanks Christy.
Gregory Gin: Thanks, Kristi. I'd like to welcome everyone and thank you for joining us on the Abeona Therapeutics conference call to discuss our second quarter 2020 financial results and Business Update. Yesterday after the close of U.S. financial markets, Abeona issued a press release, which is posted on our website at www.abeonatherapeutics.com. On the call today with prepared remarks are Joelle Siffert, Chief Executive Officer of Abeona, and Ed Carr, Chief Accounting Officer. We are also joined by Michael Amoroso, Chief Commercial Officer.
I'd like to what welcome everyone.
And thank you for joining us on maybe only therapeutics conference call to discuss our second quarter 2020 financial results.
And.
And business update yesterday after the close of U.S. financial markets, maybe on issued a press release, which is posted on our website at www dot maybe other therapeutic stuck.
On the call today with prepared remarks argue I'll sit for Chief Executive Officer of FBR.
And that car Chief accounting officer.
Also joined by Michael I'm are also chief commercial officer.
After prepared remarks, we'll host acuity session.
Gregory Gin: After the prepared remarks, we will host the Q&A session. Before we start, I'll review our Safe Harbor State. Remarks made during today's call may contain projections and forward-looking statements regarding future events. Such forward-looking statements are made pursuant to the safe harbor provisions of the Federal Securities Law. These forward-looking statements are based on current expectations in our subject 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 Reports on Form 10-K and Quarterly Reports on Form 10-Q, filed by the company with the Securities and Exchange Commission.
Before we start I'll review, our Safe Harbor statement.
Remarks made during today's call may contain projections and forward looking statements regarding future events.
Forward looking statements are made pursuant to the safe Harbor provisions 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 could differ include but are not limited to those identified under the section entitled risk factors in the Companys annual reports on form 10-K, and quarterly reports on form 10-Q filed by the company with <unk> Securities and Exchange Commission.
Gregory Gin: These documents are available on our website at www.abeonatherapeutics.com. And with that said, I will now turn the call over to Joelle. Thank you, Greg. Good morning, everyone.
Documents are available on our website at www Dot Immunotherapeutics dot com.
But that said I will now turn the call or to go out.
Yes.
Thank you Greg Good morning, everyone and thank you for joining us to review our second quarter 2020 International results in business highlights.
Joelle Siffert: And thank you for joining us to review our second quarter 2020 financial results and business highlights. We remain committed to our important mission of working together to deliver gene and cell therapies for people impacted by serious diseases. During the past several months, our team has remained resilient in the face of the COVID-19 pandemic and has delivered on our goals in clinical development, manufacturing, and regulatory affairs toward bringing urgently needed treatments to patients with Recessive Dystrophic Epidermolysis Bullosa, or RDAB, and Sanfilippo Syndrome, also known as MPS-3. I'll start with an update on EB-101, our Patients with ARDAP face a lifelong struggle with fragile skin that easily tears and blisters. Most patients develop large wounds that remain unhealed, often covering a significant proportion of their bodies, and currently rely solely on palliative measures that include very painful and time-consuming wound care.
Makes needed to are important mission of working together to deliver gene and cell therapies for people impacted by serious diseases.
During the past several months our team has remained resilient tend to see some cold bid 19th endemic and has delivered in our goals in clinical development manufacturing and regulatory affairs towards bringing urgently needed treatments to patients with recessive dystrophic, epidermolysis bullosa or Deb.
And since the leasing syndrome also known that's and P.S. right.
I'll start with the updates on the B, one to one or autologous G correct itself therapy for the treatment of our Deb.
He was the door demonstrates the life long struggling fragile skin in the U.S elite here that easily tears in blisters, most patients develop large needs the remain and healed often covering a significant proportion of their body incorrectly relying solely on palliative measures that include very painful in time.
And consuming wound care.
You'd be wonder one has the potential to be the first approved therapies for our dad and the only durable treatment to address large chronic wounds, which are the most people than debilitating and requiring bulk here in the tech the majority of the aren't that patients.
Joelle Siffert: EB-101 has the potential to be the first approved therapy for RDEB and the only durable treatment to address large chronic wounds, which are the most painful and debilitating and require involved care and affect the majority of RDEB patients. We're pleased to report that we resumed study enrollment in a pivotal phase three called vital study in late June, following a pause due to the COVID pandemic. A second patient has since been treated, and a third patient is expected to be treated in the coming week. Target enrollment for the vital study is 10-15 patients with RDEV, comprising approximately 30 large chronic wound sites treated in total.
We're pleased to report that we resumed study enrollment in a pivotal phase three or vital study in late June following me a pause due to the Colby and didn't make.
The second piece in has since been treated in the third pacing is expected to be treated in the coming weeks.
Hi, good enrollments for the vital study is 10 to 15 patients and guard Deb.
Using approximately 30 large chronic insights treated in total.
Joelle Siffert: We continue to work closely with the team at Stanford to pre-screen patients and treat those who are pre-screened as soon as possible. We currently anticipate completing enrollment in the model study in early 2021, depending on how long the impact of the COVID-19 pandemic lasts, including travel restrictions and concerns about patient and or staff safety. Data from the completed Phase 1-2A trial of EB-101 reported at the Society for Pediatric Dermatology's, or SPD, 45th Annual Meeting in July, provided a detailed analysis of the considerable and durable reduction in wound burden resulting from EB-101 treatment of their large, chronic, or dead wounds, lasting three to five years. Furthermore, wound healing of 50% or greater following EB-101 treatment was associated with no pain at the treated sites for 3, 4, and 5 years post-treatment compared with the presence of pain in 53% of wound sites at baseline prior to treatment.
We continue to work closely with the team at Stanford to pre screen patients and to treat prescreening patients as soon as possible.
Currently anticipates completing enrollment in the bottom study in early 2021, depending on how long the taxes <unk>, 19th and then the class including.
Level restrictions in concerns about pacing and north South safety.
Dating somebody completed phase one two way trial will be one to one reporting the society for pediatric dermatology. Your S.P.D. So what do you fifth annual meeting in July provided a detailed analysis of the considerable and durable reduction in we'd burden, resulting from VB 101 treatment of their barge chronic heart.
<unk> lasting three to five years.
Furthermore, when you feeling at 50% or greater follow you'd be one to one treatment was associated with no pain and the treated side said three four and five years post treatment compared with present, some pain and 53% of insights and baseline prior to treatment.
The ongoing vital study will tell you the characterized the relationship between reduction of <unk> burden in pain relief follow you'd be one to one treatment.
Joelle Siffert: The ongoing vital study will further characterize the relationship between reduction of wound burden and pain relief following EB-101 treatment. At SPD, we also presented a poster that provides insights on the significant disease burden of RDAB, highlighting data from a literature review of 65 studies on the clinical, humanistic, and economic impact on patients living with RDAB and their families. The disease burden and management of RDEB takes an extraordinary toll on quality of life, which underscores the need for therapies that reduce wound burden and the associated humanistic consequences and economic impact.
At S.P.D., we also presented a poster that provides inside some of the significant disease burden of our Deb.
Lighting data from a literature review of 65 studies on the clinical humanistic and economic impact on patients living with our dad and their families.
These burden in managing the Timbar Deb takes an extraordinary told on quality of life, which underscores the need for therapies that reduce one burden and the associated humanistic consequences and economic impact.
Now, let's turn to or add new associated buyers. He be based investigation would be therapies, a deal why don't to a deal what a one.
Joelle Siffert: Now let's turn to our adeno-associated virus, AAV-based investigational gene therapies, ABO-102 and ABO-101 for MPS3A and MPS3B, respectively. In July, we held a kickoff meeting under the EMA Priority Medicines, or PRIME, program, which included members of the Committee for Advanced Therapies, or CAT, and the Committee for Medicinal Products for Human Use, CHMP At the meeting, we presented our plan toward registration of AB0102 for MPS3A, taking advantage of the PRIME designation. PRIME is VMA's program to accelerate the assessment of promising therapies that demonstrate the potential to address substantial unmet medical needs based on early clinical data. Based on this meeting, along with previous input from the CHMP and the Pediatric Committee, PDCO of BMA, we anticipate submitting a Marketing Authorization Application for EU Conditioned Approval of AB0102 for MPS3A in 2023. Regarding the U.S. regulatory path for ABO-102 and MPS-3A, we'll continue to seek guidance from FDA. We acknowledge that the FDA is currently focused on matters related to COVID-19 and other life-threatening conditions, as reflected in its issued guidance put out in May.
MPS three and then P S three d. respectively.
In July we held a kick off meeting.
I'm going to you and me try already medicines or crime program, which included members of the committee for advanced therapies or CHP and the committee for medicinal products for human use teaching Pete a b M b.
The meeting we presented are playing toward registration of baby or one or two friend P. S. Three eight taking advantage of the prime designation.
Prime is the amaze program to accelerate assessment of promising therapies that demonstrate the potential to address substantial unmet medical need based on early clinical data.
Based on the meeting among with previous Dick what can be teaching p. and the pediatric Committee P. D. C. O <unk>, we anticipate some <unk> submitting a marketing authorization application for you conditional approval, a baby or why no two per M. P. S. Three a in 20 to 23.
Regarding the U.S. regulatory path for a deal one or two in MPS three eight we're continuing to see see guidance from Sta. We acknowledge that DFT is currently focusing matters related to Cobiz 19, and all that life threatening conditions as reflected in its issued guidance.
Put out in May.
Joelle Siffert: We look forward to providing an update after we receive feedback from the FDA. Now, let's move on to patient enrollment in our MPS 3 Phase 1-2 trials. Total enrollment today in the Transfer A study for MPS 3A is 17 patients, including 11 patients in cohort 3. Total enrollment to date in the Transfer B Study for MPS 3B is 9 patients, including 2 patients dosed in Cohort 3. We anticipate additional enrollment in the programs in the coming weeks and potentially completing enrollment in both Transfer A and Transfer B Studies by the end of 2020. Updated positive interim data from the Transfer A and Transfer B studies were presented at the American Society of Gene and Cell Therapy 23rd Annual Meeting in May and during the MPS Expert Webinar at the International Symposium on MPS and Related Diseases in July. Both these presentations are available on our website. The findings support previously reported data showing preservation of neurocognitive skills, now up to two years, among the three young patients treated in cohort three of the Transfer A study.
Look forward to provide an update after received feedback from the FDA.
Let's now move on to patient enrollment in our MTS, two or three phase one two trials.
Enrollment today and the transfer a study from P. S. Three a 17 patients, including 11 patients dosed into cohort three.
Total enrollment today and the transfer of these studies for MPS three D. is nine patients, including two patients dosed in cohort three.
Do you anticipate additional enrollment into programs in the coming weeks and potentially completing enrollment in both transferee and transferred these studies by the end of 2020.
Updated a positive interim data from the transfer a and transferred be studies as presented to the American Society of gene and cell therapy 20, Threerd annual meeting in May and during the M.P.S. expert Webinars of international Symposium on MTS and related diseases in July of both these presentations rebel border web.
Right.
The findings support previously reported data showing preservation of nerve cognitive skills now up to two years among the three young patients treated and dose cohort three of the transfer East study.
Joelle Siffert: In addition, improvements in multiple disease-specific biomarkers and a favorable safety profile in MPS 3A and MPS 3B patients after treatment with ABO 101 and ABO 102 were observed in both studies. Turning to our manufacturing facilities, we resume internal operations at our fully integrated quality and manufacturing facility in Cleveland in June. Their manufacturing activities support the increased activity in our clinical programs, including manufacturing EB-101 drug product for the Phase 3 vital study. We have also initiated process development that will enable production in-house of the retrovirus used for EB-101 manufacturing.
In addition improvements in multiple disease specific biomarkers and the safety.
Favorable safety profile than MTS, three eight and MPS three b patients after treatment with they deal when no one and they'd be a window to were observed in both studies.
Turning to our manufacturing facilities, we resumed internal operations that are fully integrated quality in manufacturing facility in Cleveland in June.
Or manufacturing activity support the increased activity in or clinical programs, including manufacturing you'd be Wanna, one drug product for the phase three vital study.
Also initiated process development that will enable production in house of the retrovirus used free B. one to one manufacturing.
Manufacture provided providing us with increased control of the supply chain and product quality by reducing costs.
Joelle Siffert: We also resumed process development activities to enable in-house manufacturing of commercial supply of ABO 101 and ABO 102. We are now at the design stage for additional manufacturing facilities within the existing space to support the advancement of our clinical programs and also commercial manufacturing. Before I turn the call over to Ed for the final financial review, let me introduce and welcome Michael Amoroso, our new Chief Commercial Officer. Michael joined us in July and brings over 20 years of product commercialization experience in the biotechnology and pharmaceutical industries with a focus on cell and gene therapy. Prior to joining Abeona, he held various commercial leadership positions at Kite, A-Side, Celgene, and Sanofi.
Also resumed process development activities that two enabled and how's manufacturing of commercial supply a baby a 101 and they deal one or two.
We're now in the design stage for additional manufacturing facilities within the existing space to support the advancement of our clinical programs and also commercial manufacturing.
Before turning the call over to add hub for the final financial review, let me introduce and welcome Mike One burrows, our new Chief commercial officer.
Michael <unk> joined US in July and brings over 20 years of product commercialization experience in biotechnology and pharmaceutical lenders industries, where they focused on cell and gene therapies.
Right, you're joining at Yonah. He held various commercial leadership positions at kite eight side Celgene insanity <unk>, Michael was responsible for the worldwide commercial organization for the autologous car T cell therapy as Carter.
Joelle Siffert: At Kite, Michael was responsible for the worldwide commercial organization for the autologous CAR T cell therapy, SCARTA. His proven track record in commercialization and supply chain management for personalized autologous cell therapies will be incredibly valuable as we plan for a potential go-to-market strategy for EB-101. I'll turn it over to Michael to say a few words.
It's proven track record and commercialization and supply chain management for personal lives autologous cell therapy is would be incredibly valuable as we plan for potential go to market strategy for you do you want to one.
I'll turn it over to likely to see a few words Michael.
Thank you Joe Good morning, everyone I'm very excited to join it'd be Ana it's an exciting time to join the team given the continued progress with E. B one to one vital study and the Avi clinical programs.
Michael Amoroso: Thank you, Joelle. Good morning, everyone. I'm very excited to join Abeona. It's an exciting time to join the team, given the continued progress with the EB-101 Vital Study and the AAV clinical programs, as well as the Clarity on the Clinical Development Path and Regulatory Strategy in the EU for AB0102. During my first few weeks on the job, I started working with the experienced team here to help plan for the potential commercialization of EB-101 and the pre-commercialization strategy and activities for the AAV-based gene therapy programs. I look forward to providing updates on our progress on future calls and speaking with each of you. Thank you, Joelle. Back to you. Right, thank you, Michael. We're very pleased to have Michael join the team. During the quarter, we also appointed George Midosky and Paul Mann as independent members of our board of directors.
Early clinical development path and regulatory strategy in the EU for April one or two.
During my first few weeks in the job I started working with the experienced team here to help plan for potential commercialization of he'd be why don't want and the pre commercialization strategy and activities for the Avi based gene therapy programs.
Look forward to providing updates on our progress on future calls and speaking with each of you.
Thank you do out back to you.
Right. Thank you Michael we're very pleased to have Michael joined the team.
During the quarter to quarter, we also appointed Georgia dusky and home then as independent members a board of directors, George and Paul or both highly regarded professionals with substantial financial management operational business develop and then capital raising experienced Cindy biotechnology industry.
In addition to their board service George.
Serves as the chairman of our audit Committee and Paul serves as an audit Committee member.
Joelle Siffert: George and Paul are both highly regarded professionals with substantial financial management, operational business development, and capital-raising experience in the biotechnology industry. In addition to their board service, George serves as the chairman of our audit committee, and Paul serves as an audit committee member. With that, I'll turn the call over to Ed Carr, our Chief Accounting Officer. Thank you, Joelle. I'd like to remind everyone that we recently filed the Form 10-Q, which is available on our website and where you can get the details on our financial results. In summary, our cash, cash equivalents, and short-term investments as of June 30, 2020, were $107.9 million compared to $129.3 million as of December 31, 2019. Net cash used in operating activities was $9.5 million for the second quarter of 2020.
With that tone, I'll turn the call over to add car or Chief Accounting Officer Ed.
Thank you as you're well I'd like to remind everyone that we've recently filed a form 10-Q, which is available on our website and where you can get to details on our financial results.
In summary, our cash cash equivalents and short term investments as of June Thirtyth, 2020, or 107.9 million compared to 129.3 million as of December 31 2019.
Net cash used in operating activities.
Was 9.5 million for the second quarter 2020.
R&D spending was 6.1 million for the second quarter 2020, compared to 16.3 million in the second quarter of 2019.
The decrease in R&D expenses was primarily due to decrease manufacturing clinical and non clinical development activities arising from the effects of the coven 19 pandemic.
As well as cost savings associated with decision to our internally manufactured retrovirus for that you'd be Wanna One program.
Genie expenses were 5.5 million for the second quarter between 20 compared to 5.69 in a second quarter 2019.
Net loss was 13 million or 14 cents per common share for the second quarter 2020, compared to net loss of 23.9 million or 49 cents per common share for the second quarter of 2019.
Ed Carr: R&D spending was $6.1 million for the second quarter of 2020 compared to $16.3 million in the second quarter of 2019. The decrease in R&D expenses was primarily due to decreased manufacturing, clinical, and non-clinical development activities arising from the effects of the COVID-19 pandemic, as well as cost savings associated with the decision to internally manufacture retroviruses for the EB-101 program. G&A expenses were $5.5 million for the second quarter of 2020, compared to $5.6 million in the second quarter of 2019. Net loss was $13 million, or $0.14 per common share, for the second quarter of 2020, compared to a net loss of $23.9 million, or $0.49 per common share, for the second quarter of 2019.
And with that I will turn it back over to allow for concluding remarks.
Thank you Ed Thanks again, everyone for joining us today. We appreciate your interest in I've Yonah inner continued progress in summary, it is a very exciting time for us as we continue to advance or clinical programs with the goal of providing.
We've been affected gene and cell therapies to patients who currently have no approved treatment options to that end, we haven't delivered on key goals in clinical development manufacturing and regulatory affairs over the past several months.
We have more left to accomplish in a keenly focused on working toward completing enrollment in or do you want to one pivotal vital study you know Deb you know gene therapy clinical studies, and then P. S. Three a in three b.
Strengthen leadership team, we're beginning to prepare for three potential commercial launches over the next three years.
I'll now turn it over to the operator herculean. Thank you everyone.
Thank you Sir the floor is now open for questions.
Ask that question. Please press Star then one.
Joelle Siffert: And with that, I will turn it back over to you all for closing remarks. Thank you, Ed. Thanks again, everyone, for joining us today.
Thank you Pat.
Using its speakerphone, please pick up your handset to provide the best sound quality.
Ladies and gentlemen.
Hi, Matt. Please press Star then one of your telephone keypad.
And our first question comes from Maury Raycroft with Jefferies. Please go ahead.
Hi, good morning, everyone and congrats on the other progress.
Operator: We appreciate your interest in Abeona and our continued progress. In summary, it is a very exciting time for us as we continue to advance our clinical programs with the goal of providing safe and effective gene and cell therapies to patients who currently have no approved treatment options. To that end, we have delivered on key goals in clinical development, manufacturing, and regulatory affairs over the past several months. We have more left to accomplish, and our team will be focused on working toward completing enrollment in our D101 Pivotal Vitals Study in RDEB and our Gene Therapy Clinical Studies in MPS 3A and 3B. With our strength and leadership team, we're beginning to prepare for three potential commercial launches over the next three years. I'll now turn it over to the operator for Q&A, and thank you, everyone. Thank you.
First question just wondering if you could talk more about that the discussion was CHF P. I guess, maybe talk about the previous input you've gotten from them and what aspects you blind down for potential registration for three A. I guess, what else you have to do before the he may application filing and 2023.
Yeah. Thanks, Maria and then thanks for your question. So the this is an ongoing process. We had no. This is the.
Third such meeting with European regulators. So this is again different from scientific advice. This is not the fully binding agreement, but certainly the reinforcement of the discussions with had previously made such that I think the the general feedback is converging to like a mutually agreed.
And that's what I alluded to in both on the text and be prepared remarks.
This covers both the late in scope of the clinical data source predicated on successful clinical data as we follow patients long term.
Operator: The floor is now open for questions. If you would like to ask a question, please press star and then one on your telephone keypad. And if you're using a speaker phone, please pick up your handset to provide the best sound quality.
Putting patients, but we have recently enrolled and expect to enroll before the end of the year.
Includes the process of a generating a commercial brings the product or when should we expect a will be done in coming months. As we've mentioned this is a process being developed within that and.
Maurice Thomas Raycroft: Again, ladies and gentlemen, if you do have a question or comment, please press star, then one on your telephone key. And our first question comes from Maurice Raycroft on behalf of Jeffreys. Please go ahead.
And we have I think of it fairly clear roadmap.
Joelle Siffert: Hi, good morning, everyone, and congratulations on all the progress. First question, just wondering if you could talk more about the discussion with CHMP. I guess you could talk about the previous input you've gotten from them and what aspects you've aligned on for potential registration for 3A. I guess, what else do you have to do before the EMA application filing deadline in 2023? Yeah, thanks, Maury, and thanks for your question. So, this is an ongoing process we have now. This is the third such meeting with European regulators. This is, again, different from scientific advice.
Apparently no problem the very clear roadmap.
For the manufacturing the product and also ensuring that this project was comparable and therefore.
<unk> for use a commercially so between these two.
Large serve areas right CMC quality and clinical regulatory I think we have a very clear roadmap of of course, coupled with the fact that we've been able to enrol additional patients in the study the fall within the.
The group of patients, who will stand to arguably benefit the most that as young patients who are not doing very advanced disease.
Got it that's helpful. It and.
And then for you'd be one on one you mentioned plans to dose additional patients in the coming weeks I guess can you say if this means that what you're seeing in the first few patients gives you the ability and confidence to pick up pace with Oh with dosing multiple patients going forward you talk more about that.
Joelle Siffert: This is not a fully binding agreement, but certainly the reinforcement of the discussions we had previously makes it such that I think the general feedback is converging on a mutually agreed path. And that's what I alluded to in the text of the prepared remarks. This covers both the length and scope of the clinical data, which is, of course, predicated on successful clinical data as we follow patients long-term, including patients that we have recently enrolled and expect to enroll before the end of the year. It also includes the process of generating a commercial-grade product, which we expect will be done in the coming months, as we mentioned. This is a process being developed within Abeona. And we have, I think, a fairly clear roadmap, fairly, no, probably a very clear roadmap for manufacturing the product and also ensuring that this product is comparable. Therefore, Dr. Molloy, Maurice Raycroft, Gregory Gin, Vishwas Seshadri, Abeona Therapeutics Inc. That's helpful.
Yeah. So we're obviously not going to comment on patient by patient and it's probably too early to tell these patients anyway, because ultimately the goal here as long term duration and some of these stations were just treated so.
We will now provide that we didn't do so it does reflect the fact is number one but we've been able to manufacture successfully that the administration and a bit treatment itself. The transplant and then you'd want to one sheets was done successfully and then there was no safety concerns in the previously treated patients. So of course all these.
Views, we've taken for granted and given the the successful completion of the phase one two way, but we obviously are care deeply that this has been safely.
I think we'll have more more data long term Baden Baden viewing early next year.
Okay, well, where are you able to dose multiple patients going forward.
Yeah, we.
Joelle Siffert: And then for EB 101, you mentioned plans to dose additional patients in the coming weeks. I guess, can you say if this means that what you're seeing in the first two patients gives you the ability and confidence to pick up the pace with dosing multiple patients going forward? If we could talk more about that.
As we mentioned, we just have a dose the patient we have another patient scheduled for dosing.
Hope to be able to dose at least two patients a month. If all goes well of course, a lot of bid is predicated on schedule, which is not doing especially during dependent.
Got it okay. Thank you very much for taking my questions.
Joelle Siffert: Yeah, so we're obviously not going to comment on patient by patient and it's probably too early to tell on these patients anyway because ultimately the goal here is long-term duration and some of these patients were just treated so we will not provide that but it is it does reflect the fact number one that we've been able to manufacture successfully that the administration and of the treatment itself that the transplant of the B101 sheets was done successfully and that there was no safety concerns in the previously treated patients so of course all these are good news we take it for granted given the successful completion of the phase 1-2a but we obviously care deeply that this is done safely i think we'll have more more data long-term data later in the year early next year, Okay, will you be able to dose multiple patients going forward? Unknown Speaker Yeah, as we mentioned, we just dosed a patient. We have another patient scheduled for dosing, and we hope to be able to dose at least two patients a month, if all goes well. Of course, a lot of it is predicating on scheduling, which is not scheduled, especially during the pandemic. Got it.
Sure.
And next we'll go to Rahm salad bar.
With <unk>. Please go ahead.
Hi, This is Blair, calling on for Rob a couple of questions for me a in the vital trial are all the pre screen patients still are eligible for enrollment or did you lose some of them and if so how many did you lose how many more where you need to prescreen yard complete enrollment.
We don't have the knowledge of losing indications but of course, we have to go through a schedule movies patients over time so.
And I know you touched on this little bit earlier, but what is likely to be the pace of treatment for the vital trial going forward in other words, how many patients do you expect to treat per month.
I think realistically a one to two patients a month I think two patients a month as a goal even though we are I think on track for that but of course locking happens to on the cold bid a disruption. So I think that ultimately will depend how quickly we can resume full activities and and the on the on the medical.
The center side from from the internal side were quick to accrete to patients regularly.
Okay, and then building off of that or you know as far as the effects from covert 19 can you comment on any larger execution issues that you may have going forward.
Maurice Thomas Raycroft: Okay. Thank you very much for taking my questions. And next, we'll go to Ram Salavar Uju with HC Railroad.
The the main a hurdle for execution and over the past several months moves one travel restrictions and also be a diversion of the resources in the medical centers too.
Blair Cohen: Please go ahead. Hi, this is Blair Cohen from ROM. I have a couple questions for you. In the vital trial, are all the pre-screened patients still eligible for enrollment, or did you lose some of them? And if so, how many did you lose, and how many more will you need to pre-screen in order to complete enrollment?
To attend to patients with the cobot 19 infection. So these have used up a bit of other seed things are still not back to normal so will depend on an individual medical centers, we have because as we mentioned earlier in the call being able to those patients would set for people syndrome acknowledging that that's it.
Joelle Siffert: We don't have any knowledge of losing any patients, but of course, we have to go through scheduling these patients over time. And I know you touched on this a little bit earlier, but what is likely to be the pace of treatment for this vital trial going forward? In other words, how many patients do you expect to treat per month? [inaudible] Okay, and then building off of that, you know, as far as the effects of COVID-19 are concerned, can you comment on any larger execution issues that you may have going forward? The main hurdle for execution over the past several months was one, travel restrictions, and also the diversion of resources in the medical centers to attend to the patients with COVID-19 infections. So these have eased up a bit. Obviously, things are still not back to normal.
Rapidly progressive disease. So there was an exists to to try and get to be stations as soon as possible, especially the one sort of declining faster.
And a bit more cautious would be patients with our debt. Because these are more trail patients with a large expose wounds and insert perhaps more at risk for sure infection.
So would that are largely resolved, but again does not fully back to normal Oh, we expect that we'd be able to resume activities and full over the coming months.
Enrollment is obviously being prioritize for these patients. So the part that stews you have to resume fully you'd be a follow up visits of course have followed these patients for safety remotely through in virtual visits.
Joelle Siffert: So it will depend on individual medical centers. We have, as we mentioned earlier in the call, been able to dose patients with Sanfilepo syndrome, acknowledging that this is a rapidly progressive disease. So there was an impetus to try and get to these patients as soon as possible, especially the young ones who are declining fast.
But for some of these assessments, we need patients to come back on sites held for both the San Felipe when you'd be program and these are just being scheduled no ones and speak.
Joelle Siffert: Unknown Attendee, Gregory Gin, Vishwas Seshadri, Abeona Therapeutics Inc., Unknown Attendee, [inaudible] We, of course, have followed these patients for safety remotely through virtual visits. But for some of these assessments, we need patients to come back on site for both the San Felipe and UB programs, and these are just being scheduled now as we speak. Okay, great. If I could just get one more in.
Okay, great. Thank you just get one more in I know you said for Europe, you're looking at it approval by 2023 do you think back to come before U.S. approval and what timeline are you looking at for U.S. approval. Thank you.
Yeah. So of course, the the timelines are mostly predicated on the activity we'd have to complete internally. So I can't comment because I mentioned that the FDA, yet because they've been truly overwhelmed with the coal good efforts and prioritizing as per their statement.
Joelle Siffert: I know you said for Europe, you're looking at approval by 2023. Do you think that could come before U.S. approval? And what timeline are you looking at for U.S. approval? Thank you.
Joelle Siffert: So, of course, the timelines are mostly predicated on the activities we have to complete internally. So I can't comment, as I mentioned, on the FDA yet because they've been truly overwhelmed with the COVID efforts and prioritizing, as per their statement, what to do for timelines such as drug approvals or new IDs. But in terms of the time frame, this is gated by our ability to both manufacture and release a new product, which obviously has to be tested for comparability. And ultimately, the length of follow-up for patients who have recently been involved in the studies. We want to have the most practical but as robust data set as we can going into a regulatory submission. So that includes the young patients who have been dosed, all the patients who've been dosed in the study for safety, and also additional young patients who were just dosed and plan to be dosed before the end of the year. So that's how these timelines get us to 2020.
Due for a timeline such as a drug approvals were you like the but in terms of the time timeframe. This is gated by inability to both manufacturer in release new product.
He has been actually to be tested for comparability and ultimately the links for follow up for patients who have been recently and Golden studies, we want to have the most of them as practical but as a robust data set as we can going into a regulatory submission. So that includes the.
Page the young patients who had been goes all the patients and being dosed in the study for safety and also additional young patients. We just shows and plan to those before the end of year. So that's a visa that's how these timelines so getting get us to 2023.
Okay. Thank you very much.
Our next question comes from.
With Mizuho.
Please go ahead.
Hi, good morning, and thanks for taking my question just one question each.
Naive B Y O y.
Oh out here, so for that you'd be Wow one program.
Joelle Siffert: Okay, thank you very much. And our next question comes from Zefei Yang with Mizuho Securities. Please go ahead. Hi, good morning, and thanks for taking my question. Just one question each, on the EB-101 and AB-0102.
To be able to comment if you were able to activate the Neal side on the east coast.
And.
I cannot.
And on the Prescreening what Phil.
Zefei Yang: So for the EB-101 program, would you be able to comment if you were able to activate the new site on the East Coast and it sounded like you had done a fair amount of pre-screening work? So is it safe to assume that these patients have been pre-screened? What's left to do is really just to travel to the clinical sites and get treatment. Now, the second question is on AB0102, and are you... Are you able to make comments with regard to the general understanding of the regulatory pathway in the U.S.? Will it be more or less similar to European guidance?
That these patients have been pretty schooling <unk>, what's left to do it really just to travel to the clinical sites <unk> got treatment.
No second class thing, it's a b O allowed to and are you.
Are you able to.
Make comments.
With regard to their general understanding off a regulatory pathway in the U.S. or will it be more or less similar to a two day feature to.
<unk> European data thanks.
Joelle Siffert: Okay, let me just say thank you for your question. So, regarding the second site, after the thick of the crisis, we resumed working with the second site, trying to get ready. This particular site, given the East Coast site, was obviously in the core of the pandemic.
Okay. Let me just had to say thank you pay question. So let me take it one by one so regarding the second site after that.
The pricing so we resumed I'm working with the second sides trying to get ready at this particular side, given but you still side.
Was obviously in both core of the pandemic, so things move will be shut down for a good part of the past water. So stay tuned and we should be hopefully able to analysis second sites in coming months.
Joelle Siffert: So things were really shut down for a good part of the past quarter. So stay tuned, and we should be hopefully able to announce the second site in the coming months. In terms of the prescreened patients, we had mentioned this before. We had 11 prescreened patients. These are prescreened under a formal prescreening protocol that is IRB-approved. So they go through all the testing, genetic testing, biopsy, etc. So it's just a question of deploying these patients into the trial. Of course, the prescreening took place over the past year and a half, so we just are in the process of scheduling patients. And of course, you know, they're real life people who have other commitments. Sometimes we have to make adjustments to their travel schedule, etc.
In terms of the Prescreening patients. We had mentioned this before we had 11 prescreening patients. These are pre screened and or hormone pre screening protocol. This is our be approved so they go through all the testing genetic testing biopsy et cetera.
So it's just a question of deploying these patients into the trials of course of the Prescreening took place over the past a year in Uh huh.
So we just are in the process of scheduling patients and of course, there real life people have other commitments, sometimes we have to make adjustments on their travel schedule whatnot, but so far so good.
Joelle Siffert: But so far, so good. And the other factor in the rate of completion of the study is the number of wounds that are treated per patient. As you recall, our goal is to treat anywhere from 10 to 15 patients. But ultimately, we expect to have to treat 30 wound sites across these patients. To the extent that the patients come in and have more wound sites treated per patient, we can probably..., then complete enrollment in the lower end of the range, if you will. So the Stanford team has been very active, incredibly proficient and collaborative on this, and they're really doing a terrific job trying to bring these patients back in, with a backdrop of, you know, international disruption and travel and whatnot. So. Unknown Attendee, Brian Kevany, Gregory Gin, Vishwas Seshadri, Abeona Therapeutics Inc. Unknown Attendee, Brian Kevany, Gregory Gin, Vishwas Seshadri, Abeona Therapeutics Inc. But we have not gotten any formal feedback yet.
In the other factory the radio completion of this study is the number of wounds that are treated per patient because you recall.
Our goal is to treat a anywhere from 10 to 15 patients, but ultimately the or do you expect to have to treat to 30 when sites across these patients so to the extent that patients come in and have more insights treated for patient yeah, we can probably.
Then oh complete enrollment and the sort of lower end range. If you will.
So the staff the team has been very active incredibly for fishing and collaborated on this and they are really doing terrific job trying to make these patients back in.
With that backdrop of books, so you know.
International disruption and travel and whatnot so.
As far as the regulatory path for the FDA, we've had some preliminary discussions with them and of course the issue does.
Draft guidance or they are going to year.
We have really have not any feedback from DFT on a few submission. We we've made so ultimately we would like to reconciled.
Our plans such as Eddie conserve both European regulators in F. T. A joint lead so because it's going to be able to see a single product and so what's important to still wait for the F. T. We think that the plan that we have currently in place that if you will largely be VFD requirements, but.
Cannot.
Zefei Yang: Yeah, thanks, Joel, for the detailed explanation. And our next question... comes from Muneer Faroohar with SVBL. Please go ahead. Hey, thanks for taking my question, guys. A little more practical one, as you ramp up involvement, manufacturing, etc.
Speculate on that until we hear from them specifically.
The European from P. is fairly comprehensive on the quality and manufacturing and oftentimes these tend to be aligned anyway. So.
In general if King you May also oh communicate internally, so well and we'll make sure that we share feedback we've had so to do this thing and the open and clobbered ugly.
Well, we have gotten any formal feedback.
Yeah, Thank god for that detailed explanation.
Our next question comes.
Comes from.
Yeah.
With.
Please go ahead.
Hey, Best taking my question guys, what a little more practical one as you ramp up enrollment manufacturing et cetera, you do what a one can you give us a little sense on the tempo of being of any relevant increases R&D spend.
Muneer Faroohar: on EB-101, can you give us a little sense of the tempo of any relevant increases in R&D spend over the next few quarters? And then, secondarily, could you give us a little bit of clarity on the status of the licensing agreement with Regenexx and the ongoing arbitration? It sounds like we should probably not be modeling a $28 million payment as a definite event. Hi Monty.
The next few quarters, and then secondarily could you give us a use a little clarity on the status of the licensing agreement with <unk> and the ongoing arbitration.
Sounds like we should probably not be modeling, a 28 million dollar payment as a definite or the definitely events.
Oh, hi money so to the first question would just after the second question first [laughter] up. So the second question is regarding the arbitration and legal proceedings of we basically what we can't disclose is what's been written in the Q released so while I can't comment any further on that.
Joelle Siffert: So the first question, let me just ask the second question first. So the second question is regarding arbitration and legal proceedings. Basically, what we can disclose is what's been written in our queue earlier. So I can't comment any further on that, unfortunately. Could you repeat your first question?
Unfortunately.
Could you repeat your first question money and just sort of escaped me.
Muneer Faroohar: Sure, um, you've talked about reaccelerating enrollment at ED101, moving forward with R&D spend tempo as you ramp up those programs and related manufacturing. Yeah, so on some, obviously, if you enroll patients, this is the bulk of the variable costs, right? As you enroll more patients, this is, you know, obviously, there's patient-related fees and procedures and whatnot. So this will tend to increase for a good reason; this is the kind of money we do want to spend right toward the progress of our programs. But we did have, we did make some significant changes in the manufacturing supply chain, bringing retrovirus manufacturing in-house, which we think will bring in significant savings over the next year. So these are, I think, important. You know, Unknown Attendee, Gregory Gin, Vishwas Seshadri, Abeona Therapeutics Inc., Gregory Gin, Vishwas, So, some things will go up, some things will go down, and So, to answer your question more in general, some things will go up, and some things will go down.
Sure.
You've talked about.
Reaccelerating enrollment you do on a one moving forward <unk> yeah, yeah like.
R&D spend tempo as you ramp up those programs and related manufacturing.
Yeah, so buttons on some they'd love to see if we were enrolled patients. This is the bulk of the variable costs right because you're going to go more patients. So this is you know obviously, there's a patient believe it feeds into procedures and anyone thought so ER visits tend to increase for good reason person to kind of money, we do want to spend right toward progress of our programs.
But we didnt have we didnt make some significant changes in the manufacturing supply chain, bringing retrovirus manufacturing in house, which we think will bringing significant savings over the next year. So these are I think important.
Uh huh.
Measures for the supply chain itself quality control de risk et cetera, but also in addition to that to reduce costs. So and then ultimately as we complete enrollment in the central equal trials have been to lead. This the spend on those trials also will reduce Robert.
All of these spaces as long term, but be the bulk of the cost of goods trials is be the actual treatments can be initial.
Joelle Siffert: It may be a slight increase in R&D spend over the next six months, but then it may not... Unknown Attendee, Brian Kevany, Gregory Gin, Vishwas Seshadri, Abeona Therapeutics Inc. Awesome. Thanks for the question, guys. As a reminder, ladies and gentlemen, if you would like to ask a question, please press star then 1 on your telephone. And we'll move next to Kristen Kluska, who has cancer at 50.
Assessments. So there's some things will go up some things will go down and eventually when we complete and woman.
Activities also will Peter out so there's so to answer your question more in general some things will go up some things will go down.
Maybe a slight increase in R&D spend over the next six months to Ben and they are.
Kristen Brianne Kluska: Please go ahead. Hi, good morning, everybody, and congratulations on the progress, especially in light of everything that's going on. So the first question, as it relates to EB-101, you discussed a potential commercial launch in late 2022. Could you talk about how much time you might need to prepare for a launch after potential approval? I guess what I'm trying to calculate is that, given you a rare pediatric disease designation, whether this approval could come before September 30, 2022, when you could potentially receive a voucher, pending FDA timelines, of course. Hi Kristen.
No it's a bit.
Awesome, that's kind of question guys.
[noise] as a reminder, ladies and gentlemen, if you'd like.
Please press Star then one on your telephone.
And we'll move to Chris <unk> Oh.
Please go ahead.
Hi, good morning, everybody and congrats on the progress, especially in light of everything that's going on so the first question as it relates to E. B 101, you discussed a potential commercial launch in late 2022 could you talk about how much time, you might need to prepare for launch after a potential approval I guess, what I'm trying.
Any calculated that given you have rare pediatric disease designation, whether there's a critical could come before September 30 at 2022, when you could potentially receive about shirt pending after you guys timelines and of course.
I christened yeah. Thanks for your question and maybe I'll give my close an opportunity to speak a bit. So obviously a prepared for commercial launch search. So you know ideally several years before because there's a lot of things that we'd be to work on.
Joelle Siffert: Yeah, thanks for your question. And maybe I'll give Mike an opportunity to speak a bit, too. So obviously, preparing for commercial launch starts, you know, ideally several years before because there are a lot of things that we need to work on, but Michael can give you, you know, on his long tenure with the company over the past month and a half, he can give you sort of some preliminary thoughts on that. Michael.
But Michael can give you you know it's a long tenure with the company <unk> best month in the house.
If you sort of some preliminary thoughts on that.
Michael.
Sure John Thank you and Christian Thank you for the question, Yeah, I think dwell said it well our commercialization efforts have begun now right with the bringing myself and I were starting to look at the right staff and team members to continue to bring into the organization to join the already very talented group into clinical side of things preparation to launch absolutely starts now.
Michael Amoroso: Sure, Joao, thank you. And Kristen, thank you for the question. Yeah, I think Joao said it well.
Michael Amoroso: Our commercialization efforts have begun now, right, with bringing myself in. We're starting to look at the right staff and team members to continue to bring into the organization to join the already very talented group on the clinical side of things. Preparation for launch absolutely starts now. Kristen, I think you spoke to the middle of 22.
Christian I think you spoke to the middle of 22, we have plenty activities to get up and running and that would obviously be with a continuing to look at <unk> footprint for sites for you'd be one on one so our activities have begun and we'll continue to update on not on the progress during an ongoing quarterly calls.
Michael Amoroso: We have plenty of activities to get up and running, and that would obviously be with continuing to look at our footprint for sites for EB-101. So our activities have begun, and we'll continue to update on the progress during ongoing quarterly calls. Okay, great. Thanks. So, you know, I understand you're doing the preparations now, but I guess the question was more targeted towards once you have an actual approval in hand, how long that timeline could take, and, you know, whether or not you think you could potentially get this approval ahead of this expiration for rare pediatric disease designation vouchers. Joel, I can take the first part of it.
Okay. Great. Thanks, So you know I understand you're doing that preparations now, but I guess the question was more targeted toward once you have an actual approval in hand, how long that timeline could take and you know whether or not you think you could potentially got this approval ahead of the exploration for.
Rare pediatric disease.
Now chairs.
Joel I could take the first part of it yeah, we'll have it will be <unk> yeah.
Michael Amoroso: Yeah, we'll have it. We'll be ready. Yeah. Yeah, go ahead. Yeah.
Right Yeah.
Michael Amoroso: And I haven't looked at the specific timing for the voucher, Kristen, but go ahead. Yeah, and Kristen, the idea is that as soon as we get FDA approval, we will be ready to launch EB-101. We'll have our commercial supply, and we'll be able to launch immediately upon FDA approval. There will be no lag for us to treat our first patient.
I haven't looked into specific timing for the belcher christened but go ahead Michael.
Yeah, and Christian the ideas as soon as we get an FDA approval, we will be ready to launch a if you want to one we'll have our commercial supply and we'll be able to launch immediately upon FDA approval.
There will be no great for us to to treat treat a first patient.
That's very helpful. Thank you and then to the end. He has three study you've indicated that cohort three katri anywhere between 90 60 subjects for MPS three be that could be after five patients per cohort three so just given that youve provided guidance today in terms of completing enrollment by the end of the year.
Michael Amoroso: That's very helpful. Thank you. And then for the MPS 3A study, you've indicated that Cohort 3 could treat anywhere between 9 to 16 subjects. And for MPS 3B, that could be up to 5 patients in Cohort 3. So just given that you've provided guidance today in terms of completing enrollment by the end of the year, could you discuss whether, you know, there's a specific patient number that you're targeting for each of these groups? No specific number of patients, but since we've obviously got a lead time before patients ultimately come in to enroll in the study from the identification, pre-screening process, etc., we have a general sense that we'll have sufficient patients to have a reasonable number of patients that fall under the current inclusion criteria, which is development quotient greater than 60 or younger than two years of age. So these are patients who still have preserved organs. Neurocognitive Function Study Entry
Could you discuss whether you know there's just specific patient number that you're targeting for each of these groups.
Oh, no specific number of patients, but since Weve, obviously, there's a lead time before patients that ultimate coming to an going to study from the.
Then if occasion pre screening process et cetera, we haven't and general sense that have sufficient patients.
To have a reasonable number of patients that fall into the current inclusion criteria, which has a development quotient greater than 60 or younger than two years of age. So these are patients who still have preserved.
Cognitive function at study entry. So we think that would within the criteria within the pre screening in the screening bonds. We have in patients who are waiting to be dose.
We should have sufficient patients to to comprise they have a sufficient dataset for to support a regulatory approval.
Joelle Siffert: Again, all this is predicated on how well these patients do post-treatment, but at least in theory, that's what I think we can meet these parameters for the end of the year. Okay, great. And then the last question for me is that, although I recognize that the meeting you had with the EMA was related to ABO 102, do you think the proposed outline could be similar to ABO 101? And are you thinking about, you know, any synergies across the two programs, given 102 is a little bit more advanced?
Again, I'll just try to cater into how well these stations do post treatment, but but at least in theory bets that so I think we can we can meet these are these parameters of where there would be here.
Okay, great. Thank you and then the last question for me is that I recognize meeting you had I'm Lucky you may was related to E. B L. One or two but do you think the proposed outlined could be similar to Avi I want to one and are you thinking about you know any synergies across the two programs given what appears a little bit more.
<unk>.
Yes, a that at all times of we're thinking about that and of course will will make an effort to also engage specifically regarding it'd be a 101.
Joelle Siffert: Yes, at all times, we're thinking about that. And of course, we'll make an effort to also engage specifically regarding ABO 101 formally, right. So although we can make these parallels, and of course, any lessons learned from the MPS 3A program, we are already using to anticipate and steer the MPS 3B program.
Formally right. So although we can make these parallels and of course ended lessons learned from being and PS. Three program. We are already using to anticipate and see or the M. P. S. Three b program, so absolutely, but we ultimately need formal feedback and we need the yeah may enough T. Two concur that this is.
Joelle Siffert: So absolutely, but we ultimately need formal feedback. And we need the EMA and FTA to concur that this is actually a sensible plan, which I assume it will be, but we obviously need to work on that. And even in process development for the manufacturing of a commercial grade product, a lot of the learnings over the past year, including some of the process development we did for some other AV vectors, including the MPS 3A ABO 102 vector, will also serve well for the ABO 101
It's actually a sensible plan, which I assume you're will be but we obviously need to work with them and even in process development for the manufacturing and other commercial grade product. So a lot of the learnings over the past cheered, including some of the cost of development would bid for some other avi vectors, including a b M. P. S. Three.
<unk>.
Deal, one or two backed or a will serve as served well for the email one to one product.
Joelle Siffert: So I think that's correct. In summary, it's correct. Your assumption's correct, but obviously, we'll double-check this with the regulations.
So I think that's correct in summary, it's correct your assumptions correct, but obviously will double check this with the regulators.
Okay, great. Thank you again.
Kristen Brianne Kluska: Okay, great. Thank you again. Sure.
Sure.
Operator: Thank you. And as a reminder, ladies and gentlemen, if you would like to ask a question, please press star and then one on your telephone. And I'm showing no questions at this time, so I'll turn the call back to Joelle. Thank you, everyone. Again, we appreciate everyone joining us this morning. And let us know if you have additional questions, and know that we're all very committed to fulfilling our mission. So, thank you, and that does conclude today's teleconference. We appreciate your participation. You may disconnect your lines at this time and have a, [inaudible]
And that the remainder ladies and gentlemen, if he would like ask a question. Please press Star then one on your telephone.
And I'm showing no questions at this time, south hand, the call back to fill out right.
Thank you everyone.
And appreciate everyone joining us this morning, and a better snow have additional questions and no the wheel very committed to fulfilling our mission. So thank you.
And that does conclude today's teleconference. We appreciate your participation you may disconnect. Your lines at this time and have a great.
Oh.
[noise] Oh.
Huh.