Q1 2023 Abeona Therapeutics Inc Earnings Call

Greetings and welcome to the Abbvie on a therapeutics first quarter twenty-three portfolio update conference call. At this time, all participants are in a listen only mode.

A question and answer session will follow the formal presentation. If anyone should require operator assistance during the conference. Please press star zero on your telephone keypad.

Please note this conference is being recorded.

I'll now turn the conference over to your host Greg Ginn, Vice President of Investor Relations and corporate Communications you may begin.

Thank you Holly good morning, everyone.

I would like to welcome and thank everyone for joining us on our portfolio update conference call are.

Our objective today is to share additional new positive data from our E. B 101 in AAV ophthalmology programs recently presented at scientific Congresses.

Press releases announcing the data at the I S. I D N. A S. E. T meetings are available on our website at www dot a beyond therapeutics dot com.

Before we start I would like to note that remarks made during today's call may contain projections and forward looking statements.

We're 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 risk factors section in our Form 10-K, and periodic reports filed with the SEC.

Documents are available on our website at www dot maybe around therapeutics dotcom.

Okay.

On the call today.

Our doctor ambitious to sorry, Chief Executive Officer, who will give some opening remarks Dr.

Doctor to meet you got Grachev, Chief Medical Officer, who will review the new vital study data that were presented at Ias I D and Dr. Brian Kevin <unk>, Chief Technical Officer, who will review the animal proof of concept data from our AAV Ophthalmology program presented at a S. D C T last week.

After the prepared remarks.

Joining us for the Q&A session will be Giovanna, Chief Financial Officer, and Dr. Manav is that Nevada, Vice President business development and with that I'll now turn the call over to visit the salary soya sauce dish.

Thank you Greg Good morning, everybody and thank you for joining us this morning.

I want to start today by saying last week with a great advancement for the epigenome of licensed below sell R. E. D community I want to congratulate Christopher biotech in the patient community researchers and regulators for bringing the first genetic therapeutic option to tackle this debilitating disease.

More therapies come to market, we anticipate increased awareness about therapeutic option in the East Bay and also improved diagnosis and Geno typing for this painful disease.

Recessive dystrophic E. B are our debt is a connective tissue disorder in which both copies of the gene for colleagues and seven are mutated in dysfunctional, resulting in the lack of connectivity between the outer and inner layers of the skin patients with our debt faced a lifelong struggle with fragile skin.

That easily pairs and blisters with most patients developing large painful wounds that remain unhealed, often covering a significant proportion of their body.

Recently published natural history data in our debt analyzing doing 51 are that we'll describe the two distinct types of Arctic ruled on the top or chronic open RF, while on the bottom are the recurrent wounds on the right the spider plots show chronic wounds.

I only show, 50% or greater wound healing, while the recurring will frequently even completely and open again.

Furthermore, I E. This year a prospective study was presented by the team at Stanford is further corroborated the distinction between large chronic wounds and the current wounds.

We are excited to advance EV 101, as a potential therapy that could deliver years of sustained wound healing and proven pain relief. After a one time treatment cycle for a large chronic wounds. These are pictures of the patients in our vital study with large chronic wounds on the <unk>.

Upper left side, you can see the large area of coverage and wound healing that were achieved with three it'd be one on one sheet applied in a quick fashion on the far right tattooed wounds were scored at greater than 75% healed, although the wound looks nearly completely new to the naked eye there were scored at.

75% plus appeal because without picking the crust of the yellow cross that you see there. The physician is unable to confirm complete healing that speaks to the stringent criteria in our scoring for complete wound closure over the next two slides I'll discuss the molecular basis for how you'd be 101 deliver will deliver as wound.

In a sustained fashion.

Did you Wanna, one works by Durably, restoring functional collagen seven expression to artist Keratinocyte.

They are sourced from patients skin biopsy grown in cell culture, and transfused with a corrected copy of the call. It 71 gene to a retroviral vector the transduce keratin sites at Florida culture into epidermis sheets that are transplanted onto open wound patients particular instantaneous closure.

The scientific rationale for anyone on durable benefit is that the transgene encoding functional Collingwood 71 integrates into the host genome and has therefore maintained stably at south Dubai and warm epidermal sheets in our manufacturing process.

For the mall.

Histochemistry of skin biopsy from E V 101 treated sites demonstrate sustained collagen expression for up to 24 months and in fact, we have unpublished data for up to 36 months those green lines that you see indicate collagen 71, staining and the widened arrows point to the epidemic.

Dumbo junction.

This is the rationale for the compelling your ability with you on the water as observed in our phase one two a study.

Mean follow up of five nine years and a maximum of eight years.

The results show that the majority of you one on one treated wounds showed sustained healing when they introduction after a onetime treatment.

I'd now like to turn the call over to our Chief Medical Officer Doctor immediately Gracia, who will review the results that were presented at Ias I D.

Thank you.

Sure I would like to express my excitement about data presented at the International Society for investigative dermatology meetings or I S. I D.

Doctor, Jim Dan Professor of Dermatology at Stanford.

In principle is to get there of the vital study presented the data during an oral session at I S. A D and data as it also featured it in a separate poster presentation.

The results presented at Ias a D show that he would be one to one improved wound healing and then the deduction at 612, and 24 weeks compared to control wounds fallen one time application of <unk> would be one on one.

Sure they're more E. B 101 demonstrated improvement in patient reported and the caregiver reported outcomes for each and blistering Citi Jim.

Before he does it in the results I want to provide a brief summary of the trial design for wide though.

Why though.

Zion to investigate the efficacy safety and Tolerability of <unk> would be about a one in approximately 36 large chronic wounds bass in 10 to 15 patients with a minimum age of six years.

I always thought it was randomized and controlled.

Each patient has a minimum two large chronic wounds selected with each other in the mice treated wounds being bad visitor control won't see millions size, yes that won't remain chronically open and they'll make a location.

Possible visit the same patient.

In white, though we define large chronic wounds as wounds that have greater than 20 square centimeters subsurface ADM and remain open for a minimum of six months, although mainly the open for years.

The align visit D on the study endpoints and statistical analysis plan.

Given that it would be one to one is thought of it in large chronic wounds to remember is the hardest to treat the warrants that remain open for years, because they cannot self heal is he align on the co primary endpoint of greater than or equal to 50% wound healing.

And the second primary endpoint of band the deduction is additional measure of clinical benefit.

Specifically the co primary endpoints of the field the proportion of out of that side.

Syed with greater than or equal to 50% up Hayden from baseline compared in the city that is matched control wound size at six months' time point is determined by investigator assessment and second band the reduction associated with wound tracing change, but the main difference in score.

Oh, the one Baker faces scale basically in the randomized studies at major control wounds at six month endpoint as reported by the patient.

This is the first deal with those target this patient reported pain as a co primary endpoint.

The secondary endpoint was the proportion of out of the wound sides with complete wound healing from baseline campaign around the mice treated with major control wound size at 12 and thank you for.

Exploratory end points included additional assessments for wound healing and being the reduction as well as the assessment for each submitted yet and lithium.

If he gave you the baseline characteristics during the why the top line that is out school and one go into details here.

Just to remind you of the large size and severe pain associated with the warrants included in why though.

<unk> body surface area of randomized the wounds treated with VB one O.

One patient was 160 square centimeter because that age of 82 200 square centimeters to clarify.

Is it treated wounds is 40 square frankly, amaze us exactly the size of the graft.

For a large growing like it is that exceed 40 square centimeters multiple graft could be applied wealth like fashion in which case the area covered by the each graph you can see that at this time.

And I won't ADM was less than 48 square centimeters that Brian was required to prepare the wound bed to feed the graft.

Contrary cointreau warns theyre not there Brian .

Median wound.

Duration of randomized city that wound that is months that they won't have the remain co. Nikola opened was 60 months or five years.

The control wounds had waited familiar wound duration.

Now, we can turn to the results.

Help you follow the Johnson graph you'd be one of the one treatment data at once.

Blue and controllable once in gray.

The wound healing was observed.

At this time point assessed mutual six weeks.

And it was sustained at all subsequent time points ingredient and 24 weeks.

Consistently the wound healing.

This cycle is significant.

<unk> was seen in earliest time point assessed which was six weeks and sustain at all subsequent time points ingredient 24 weeks.

Bain assessment by Ikea gave us showed great improvement in pain score for <unk> Rete once it's 46% of <unk> gave us cause you guys and the wounds as much improved or very much improved at week 24 from baseline for <unk> hundred one treated once compared to zero percent for the country.

The old ones.

In addition at home Bank assessment, using one Baker phases scale showed significant.

The reduction it would be around the one treatment.

It is a big city.

Pain, but also assessed using patient reported outcomes measurement information system called promise is a significantly greater improvement in pain quality sensitive score achieved you'd be 101 treatment.

In addition to significantly reducing pain based on the reported and again are reported outcomes related to each and believes they didnt showed significantly greater improvement it would be one to one statement.

As a reminder, each for out of their patient is a significant factor that impulse quoted you for life and then can you just east of trauma belief that in an infection.

Now, let's review safety and Tolerability.

<unk> was shown to be safe and well tolerated with no serious treatment related adverse events observed in why though consistent path clinical trial experience.

In conclusion, why though demonstrated positive b, what those study results and favorable it is can benefit profile for E V. One of one in patients that is out of that.

<unk>.

Thank you Dmitry.

We made significant progress toward BLA submission and our marching toward commercialization. We had previously mentioned one of the key milestone was to complete three consecutive process performance qualification or PQ runs the demonstrate our validated process and readiness for commercial production.

We're excited to share that today, we have completed this important step for both the retroviral vector manufacturing and E. V 101 drug product manufacturing, which was the last critical piece of data we have to generate to complete the CMC module for the BLA submission.

We announced in our first quarter 2023 results press releases that we had submitted a pre BLA meeting request with the F. D. A M D.

Yes, since I accepted our request and the pre BLA meeting is scheduled on July 10, 2023 to discuss the format to content and acceptability of the anticipated BLA application based on this meeting date, we are on track for the anticipated BLA submission in early third quarter of 2023.

Yes.

On the anticipated timing of BLA submission, we expect potential BLA approval in late first quarter or early second quarter of 2024, if the BLA is approved we anticipate being granted the priority review voucher, which can be used to receive expedited review by the FDA.

<unk> marketing application for a different product or sold to another company <unk> has been sold to other biopharma companies for approximately $100 million.

As part of our commercial planning for <unk> hundred one we continue to engage with stakeholders across the health care system, including public and private payers and health care providers to better understand market basket and pricing or E. B 101, we are encouraged by the initial feedback from these stakeholders and feedback that we have.

As seen from patients advocates and organization, which collectively support parts of those coverage decisions and pricing in line with the value of a onetime treatment that deliberate wound healing in pain reduction for years.

Based on our initial discussion payer's view Ardeb is a disease with very high unmet need and believe EV 101 has a well differentiated profile with durable clinical benefit for the treated wounds.

So given the ultra rare prevalent a bar that view it'd be one on one will have a limited overall budget impact and have indicated high willingness to cover a it'd be one on one with favorable access policy, giving us confidence in its potential.

Now, let's turn to our preclinical ophthalmology program, we presented animal proof of concept data at Ada as DCP for investigators AAV based gene therapies for Sagar disease excellent retinal diseases and also somewhat dominance optic atrophy.

Preclinical proof of concept data provides early evidence of the potential of our proprietary AAV capsid and gene construct.

Expressed as a recombinant protein in target tissues, and rescue mutant genotype and mouth disease models.

I'd now like to turn the call over to our Chief Technical Officer Doctor, Brian Kevin <unk>, who will review the results presented at <unk>.

Thank you this.

We are excited by the broad potential for treating serious eye diseases with the new AAV based therapies using novel AAV capsid from our in licensed aim capsid library and internal research.

D C. T. We presented three posters highlighting encouraging findings from animal proof of concept experiments from our AEP Ophthalmology program.

The first poster presented featured ABL five O for a novel approach to treating Star Guard disease. The most common form of juvenile inherited macular dystrophy.

Arizona recessive starter disease is caused by mutations in the a b C. A four G. Preventing the removal of toxic substances from photoreceptor cells that result in photoreceptor cell death, and progressive vision loss.

Our proprietary strategy is designed to efficiently would constitute the full length. The a b C. H four gene, which is too large to fit in a standard AAV genome by implementing a dual AAV vectors strategy utilizing the Cree locks Beaver comedy Cisco.

The data it is GCT provides compelling evidence that two independent AAV vectors utilizing greber companies can efficiently reconstitute the a b C H, where gene leading to full length. The a b C. A four protein expression can.

Key conclusions presented include.

In vivo Cree mediated recombination yields full length, a b C for using our dual vector system recombination recombinant human a b C or four is detected and properly localized to photoreceptor outer segments within one month of treatment.

Our results show that Cree mediated recombination of dual AAV vectors as safe and effective in delivering an expression of full length human ABC at war knockout mice mouse model paving the way for a novel therapeutic approach for treating startup diseases.

Further studies will evaluate a b C for expression of an accumulation of LIFO foxconn in a mouse model I'll start or disease.

Additionally, we believe our dual vector approach using Cree logs can also be adapted for other therapies, and which AAV based delivery of large genes as desired.

Our second poster presented at AST using T featured ABL 503, a novel gene therapy approach for the treatment of X linked retinoschisis or ex all the RF. The most common form of inherited macular disagree and males.

<unk> patients present in the first decade of life with cavities developing between retinal layers, leading to discontinuity within the retinal circuitry photoreceptor degeneration and vision loss.

ABL five or three is composed of a functional human Rs. One packaged in the novel aim capsid AAV 204 that effectively targets photo receptors and restores our S. One expression.

Oh, five or three will be administered via a pair of retinal injection that will provide an improved safety profile for treatment of XL are us and which targeting a photo receptors as desired, but where disease renders the retina susceptible to damage from more invasive approaches.

The data presented at <unk> demonstrated robust Rs one expression in the retina improved cone photoreceptor density and overall photoreceptor cells survival as well as a restoration of the outer retina architecture.

Treatment with a b O five or three and nude mice was associated with photoreceptor preservation improves retinal function.

These results support further development of AEP, Ohio with three for the treatment of <unk>.

Our third poster presentation at a S. D. C. T featured an investigative AAV gene therapy for autosomal dominant optic atrophy or <unk>.

<unk>.

Our form of vision loss associated with loss of retinal ganglion cells or RG seeds residing in the inner retina caused by mutations in the <unk> gene.

Kennedy's vector is composed of the human Opel onesie and packaged in the aim capsid AAV two or four discussed previously.

So many of RG sees the vitreous cavity, they make them an attractive target for gene therapy delivered by our apparel retinal dosing route.

The data presented confirmed the expression of over one in both cell culture, and retina dosed wild type and disease model animals improvements in photoreceptor outer nuclear layer thickness and auto kinetic responses were observed with our <unk> gene therapy candidate in a 10 month proof of concept study designed to test the functional consequences.

We have opened one restoration and mouse retina is following an integrant drill injection.

Initial efficacy results suggest an improvement in retinal signaling to the brain and improved visual acuity and treated mice.

These benefits support further development of our <unk> gene therapy candidate back.

Back to this.

Thank you, Brian we're looking forward to pre IND meetings with the FDA for two of our programs taking place this quarter with that I'll turn the call back over to Ali for the Q&A section.

At this time, we will be conducting a question and answer session. If you would like to ask a question. Please press star one on your telephone keypad.

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One moment, while we poll for questions.

Your first question for today is coming from Maury Raycroft of Jefferies.

Hi, good morning, and congrats on the updates and thanks for the presentation today.

We saw crystals a wide you back was approved with a relatively broad label do you expect the same based on your conversations with the agency, including patient profile type of wounds et cetera, maybe if you can talk a little bit about what you're expecting in the label.

Thank you Mario for the question and good morning.

Regarding the indication and the patient types I want to reiterate that our vital our pivotal study has treated or deb patients recessive dystrophic E b.

And we have inclusion criteria that focus on large and chronic wounds. It's very early in the BLA process to comment on how broad are restrictive the label would be given of course, the high unmet need and this is an ongoing dialogue that's going to be there with the agency. So unfortunately I cannot give you a definite event.

Answer, but what I can assure is the types of wounds that we have.

<unk> generated evidence for.

Got it that makes sense.

You mentioned that initial feedback from stakeholders across the health care system has been positive.

Maybe if you can talk a little bit about how youre anticipating pricing could work out with a voucher back having a list price of about 630000, I guess, how does that inform how you're thinking about pricing.

Certainly can talk about that I will turn the call over to our motto.

Nevada, who is leading a lot of our discussions with important stakeholders like both payers as well as our hospital administrators, where there's a clear appreciation for the value proposition of a onetime treatment that can give you a years of benefit without having to treat the same old again.

So models if you can.

Oh, you know throw light on the differentiation in how we've discussed about pricing. Please.

Sure wish Hey, Marty Thanks for the question Yeah.

Yeah, we find graduate.

<unk> launched price now to be to be very insightful and how we're going to go about with pricing.

Earlier in the year, we had conducted.

Payer research, mostly blinded research with.

Nearly a dozen payer groups across commercial Medicare Medicaid.

And it was very encouraging to hear when we discuss the clinical profiles of both.

All of the investigational therapy, including be back as well as what you'd be one on one.

That the payers really find the clinical profile for <unk> would be one I want to be very class formation that will given the wound healing profile pain reduction and durable and at that time, we had placed a few different scenarios as to what if the entry price or <unk> would be and how would that compare.

The potential price and price elasticity for EB 101, which is all very encouraging and now that we have learned what the pricing for Baidu. App is we will be in a much better position to be able to place pricing for EB 101.

Making sure that there is no access hurdles to patients but at the same time. We are also capturing the value for the innovation and the you know.

The durable effect that he'd be one on one will be able to bring so all in all I think the price elasticity maintained and given the profile that we now have we'd be in a better position to be able to place the price quote launch price for EV 101.

Does that got it yes.

Yeah, that's that's helpful and maybe it makes sense with understanding the value proposition.

And maybe last question then I'll hop back in the queue. The gene therapy data from <unk> looks good I'm. Just wondering if you could talk a little bit more about how you're prioritizing are those three different opportunities and should we think of Star Guard with 504 program as being in the lead.

Or how should we think about that.

Sure I can talk a little bit about it and then.

Turn it over to Brian for any additional thought.

Right now we have compelling preclinical data from these disease model in mice.

So the expression of the gene.

May be aware there is high unmet need in all three of those indications we had chosen those indications specifically based on how many other investigational therapies are able to make inroads and have hit some challenges along the way and we're encouraged to see that Star Guard of course, the most talked about among the three we're able to unique.

<unk> addressed the size of the ADC of <unk>.

Produce that and the correct tissue in terms of prioritization and how we resource the program Marty it's going to be we have a lot of dialogue going on we're looking at various different sources.

Sources of funding non dilutive as well and even.

Some potential government funding.

And these are programs that are all equally high unmet need and so it's not really going to be an either or.

Just going to be a matter of when and such.

Such such events will be further discussed in our upcoming calls as we make more progress on that and also get a little bit more clarity on other nuances from our dialogue with the agency because that tells US I mean are we going to be held to having NSP data or can we have other types of animal studies, which will make it much quicker to develop.

These drugs. So there's a lot of other pieces to the equation, which will further inform our prioritization.

Got it that makes sense. Thanks for taking my questions and I'll hop back in the queue. Thank.

Thank you morning.

Your next question is coming from Christian close Scott at Cantor Fitzgerald.

Hi, good morning, everybody and congrats on both of these recent data sets.

The first question I had was related to the ophthalmology portfolio. So we haven't seen as many dual AAV vector strategies, yeah in gene therapy, I know its something that others have talked about for some time. So maybe the first part of the question is just understanding what you think is differentiated about your construct that led to.

The successful findings at this preclinical stage and then the latter half of that question is is just you know frankly looking at the dual AAV factor landscape what are the expectations on how you would expect this to translate into human studies and I guess, the key things to look out for.

Given the difference between the models.

Hi, Kristen Thanks, and thanks for the question I'm going to turn it over to the expert on India in ophthalmology, which will be.

Dr Brian Kevin but.

Before that I'd, just say that we do have but thank you for this question specifically because there are certain mechanistic elements of why we think we can be successful where some other approaches.

Getting full linked a bcf or other methods fail in the past so Brian can you. Please throw some light on our unique.

Unique gene construct.

The dual AAV system. Please.

Of course, yeah. Thanks Kristen.

So I think you know there've been a number of strategies that have been tried both in dual AAV vectors as well as many genes for ABC, a four and while those programs have been going on for a number of years, we haven't seen a lot of movement. So I think we were encouraged by our ability to do this and push into a field that is.

Struggling to get.

Construct that provides full length that you'd be seeing for.

As far as how we differentiate from others. The <unk> system is one of the most efficient recombination systems known in nature, and we're trying to take advantage of that as part of developing this therapy.

So I think that is the one place where we differentiate from others, where they're using things like homologous recombination and entertains where are the efficiency every combination may not be as high as what we're seeing with the <unk> comedy system. So I think the major differentiating point for US is less about the doi movies and more about the actual mechanistic.

Aspects of the therapy that we're trying to.

Develop here.

Christian could you repeat your second part of your question.

The second part of the question was just essentially on how you think it's going to translate into human studies and I guess what are the key things to consider the risks of course going from the different models outside of what you would normally expect.

Sure Yeah, I mean I.

I don't anticipate any significant differences I mean, the advantage here is we're approaching this with a sub retinal delivery for ABB for the Star program.

For those that aren't aware this is a relatively very specific injection that occurs between the photo receptors.

Pigment epithelium. This directed injection keeps the vector very close to the site of action and does not yet diluted out as if you were writing it is an intravenous or other types of injection. So I think the aspects of that keep it somewhat similar to between is removed.

Between the mouse to a larger animal models during our IND, enabling studies and then beyond into human studies. So I think the translation from the large animal to the human studies is going to be relatively smooth because of the the injection volume will be exactly the same between the large animal model in the human will be using it.

A lot of exactly the same type of dosing. So I think that sort of translation from large to take humans is gonna be relatively seamless.

Thank you and then for the autosomal dominant optic atrophy study that you presented on you have the bullet here that says that visual acuity assessments demonstrate function recovery I think with a lot of the AAV ophthalmology gene therapy trials. The goal essentially is to show.

Slowing down of disease progression, rather than reversals. So I guess I'm just kind of interested in the context of that comment and maybe how you think about this clinically in and understand a lot of it is going to have to do with age of intervention.

These different diseases progress at different rates, but what the underlying goal youre essentially looking at will it be.

Yeah, so visual acuity in mice.

You know is something.

That we've studied with the animal study and looking at preservation or improvement of visual acuity over time. So these animals as they age the mutant animals age they start to lose their ability to have.

The high acuity vision.

This treatment was provided very early on in the disease progression.

Prior to any degradation of that visual acuity. So it really was a prevention of that development.

And visual acuity is a difficult thing to study in humans, because its something that takes a long time to develop we have seen a number of failures in the industry, where visual acuity was the primary endpoint for their trial and because of the slow progression of these diseases and specifically visual acuity it tends to be a.

A bit of a blunt instrument for looking at therapeutic benefit, but I think as part of our discussions with the agency. We're gonna be looking at understanding how primary endpoint decisions are made to better capture a therapeutic benefit. This was a very hot topic. It is just the T last week, specifically in ocular but didn't.

General over the rare disease space about understanding mean.

Meaningful endpoints that capture therapeutic benefit that may not be the ones that are available right. Now so I think as we start to talk to the regulators.

We will have a better sense of how we want to approach endpoints and make sure that we're capturing.

That benefit that it's probably there, but may not be captured by something like visual acuity.

Yeah, and if I may just add to that Christian also what we're going to be looking at us for any monogenic disease, where we have a gene therapy. Our goal is to treat early enough that the disease symptoms don't appear or you preserve your eye function right, but in.

In terms of clinical development, it's going to be the first priority would be prevention or association of deterioration of visual acuity, but of course. The bigger goal is also to see can you reverse disease, that's already happened because of the prevalent pool of patients with foregone disease. So we're gonna be looking to test all types of.

Populations, maybe those that started around a 20% to 60 level of vision and maybe those for those subjects were you.

You know a visual function, we have deteriorated even more can you reverse that right. So we're gonna be investigating clinically for all of those signals and of course, the or the long term goal is to prevent such a deterioration from happening in the first place.

Great. Thanks, and then last question for me related to our Dab at ISI D. Tuesday, two key new findings were just understanding the effects at an earlier point of evaluation and then also around some of these other secondary endpoints. So since you've presented these data wanted to here is you have all.

These discussions with thought leaders how important these two components are and I know in the past we've talked about looking at understanding pain reductions correlated with wound healing, but now you have a number of other data points to support these notions. Thank you again.

Thanks for that I'll take that Christian so.

Just to kind of highlight what the delta of data that we presented at ISI D, which wasn't in the top line or the top line focused on the six month endpoint because that's the regulatory.

Endpoint timing, whereas what we presented at ISI D is number one how quickly do we see these FX rates. So you saw that six week 12 week 24 week in fact, even three weeks, where there wasn't a patient visits to do a physician's assessment, where they're reporting they are maintaining their diaries and rising.

The pain levels, and each levels and things like that and caregivers are scoring as well we're seeing that the.

The clinical.

Clinical benefit.

Early and we've seen an example of even three weeks, which is pretty much soon a couple of weeks after their discharge and went home.

And at every time point, you can see the same kind of benefit from treated wounds in untreated wounds that that effect has sustained so that's key and that's important to note. The second is also we've previously presented the correlation between the wound healing level and pay introduction that's also very.

Encouraging to see that it's not a one time point, that's showing that it's very consistent over the time course post treatment and that's those are the some of the new data points that were discussed and we've had a lot of discussions with Kols and this is going to be very important in you know further.

On the value proposition and as Dr. Dmitry Gracia presented we had each data that's also showing a dramatic improvement for treated versus untreated and you heard it can also be a risk factor for further blistering and trauma and that's something that we are encouraged to see.

So this is going to be a holistic view of what is the benefit to patients.

Beyond all what the Kols opine and what we see is data point, what's encouraging to US is those patients who went through vital are coming back in our current ongoing study, which is a source for our manufacturing runs that we conducted people too and they are they are all lined up and they're asking for the control rooms to be treated and things like.

That that for us the ultimate.

Our confidence and conviction that this is something that patients really value.

I hope that answered your question Kristen.

Once again, if you would like to ask a question. Please press star one.

Your next question for today is coming from Jim Malloy at Alliance Global partners.

Hey, guys. Good morning. Thank you for taking my questions I just wanted to follow up on a question earlier, obviously, the competitive like cubic approval and I think on their call they're talking about a 600.

$30000 a year about 485000, a year after U S government discounts and I think they're saying about 1100 target patients not to hold you to a competitors' numbers, but how do those change up or down your guys' estimates, where do you think pricing can come in and size of the market given the given sort of their what they put out there publicly as a as their estimates.

Thank you for the question Jim.

We did take note of what their pricing is Jim but you know at this point in time as Murdo mentioned, we're encouraged by the value that you know the various stakeholders are placing in these types of gene therapies I would reiterate that what we thought a year ago, maybe in terms of what pricing we were considering and then post <unk>.

Little result.

Clearly having seen the results in the best case scenario clinical profile play out.

We're definitely.

You know re looking at our pricing in a different way now what we don't know is many details about the label for video Records number one very broad and includes all of Dystrophic E B, whereas but we're currently looking at our clinical evidence is focused on recessive dystrophic E B, which is a.

Let's say half of that and so it's really an apples to oranges comparison, because our debt is a smaller.

Relatively rare narrow or disease space.

And the value proposition is different as well I mean as one on one hand, you have a read those all blue gene therapy on the other hand, we have for a given wound a onetime treatment that gives your years of benefit.

Benefit in not having to retreat soon enough right. So that's.

Something we will definitely take into account I don't want to comment anything about exact price points and things like that because we have to do our homework on how the estimated pricing per year works out and what happens in the real world because sometimes we make these estimates and then the real world.

Turns out so there is a there is some time in homework for us to do before we come up but I want to reassure that we will be responsible corporate citizens and make sure that patients will get access to our therapies because that's that's our guiding principle here.

Outstanding. Thank you and then on the pre BLA or anything that we should anticipate potentially coming out of the PD L. A.

It's a fairly well it should be a fairly straightforward meeting then right into a BLA.

And then a follow up would be.

And then given the competitors' approval has this changed your thinking on going on on self launch versus potentially partnering.

Thanks, Jim So the first question that you asked on the pre BLA.

Every every BLA submission.

Submission given the complexity of our therapies the therapeutic space limited experience that we have in these disease areas. It behooves us to have the pre BLA meeting to make sure that we have everything that warrants a submission theres no deficiency. So that's kind of.

But the previous meeting itself is that foremost steps that you have however, I just want to assure that it's not like we have complete silence with the agency and suddenly appearing or one meeting on July 10, we have been having a lot of interaction with the agency even as we speak there's also a series of <unk>.

Informa meetings and exchange so.

This will be like the final culmination step, saying scored everything that we needed to we're going to make put this.

Does the a in front of your rates. So that is what the PPL and meeting it and we're confident that we should be in time, because as I mentioned that the biggest piece of work that we had to finish was the P. P. Q runs and we.

Completed those three consecutive manufacturing runs successfully and what's significant about that is it.

Not just that we had to meet the release criteria before you'd be one on one drug product, but also all the various process parameters have to fall within the Prespecified range to show the robustness of our process and we're happy to report that all of those a parameter.

Parameters met the goal and where we have everything we need to pull together as a package. So that's why the pre BLA submission.

Submission work stream itself. Your second question was about partnering and commercialization.

And this is a rare disease.

We have.

The focus for US is more on the delivery of our therapy in the centers, where we're going to administer that and.

And lesser on.

Sales and marketing and pull demand because we're gonna be working closely with the advocacy groups patient community. So theirs.

You know atypical launch scenario here that the focus is going to be on how do we deliver what the patient services, that's going to be needed. So given that the profile of a partner if we get a partner is going to be a very different profile than a typical large pharma kind of.

Partnership.

And so we are open to that at the same time. We're also looking at how do we retain the value we've created and their options, where you know we should be ready because we don't want to wait.

Forever to well you know make a partnership deal while those types of conversations are still ongoing and we want to score certain pieces of launch readiness in our hands already which is why we started to do with the payer research and start to talk to the stakeholders and get ready one step at a time and of course that is only going to pick up more theme.

Once we're through with the BLA submission with just kind of the 800 pound gorilla in the room right now so I hope that gives you some sense to how we're looking at commercialization as a next step so the second half of the year is going to have a lot of that conversation coming.

Coming up the limelight.

Great. Thank you for taking the questions.

Of course, thank you.

We have reached the end of our question and answer session and I will now turn the call over to vis Walsh for closing remarks.

Thank you in closing I want to thank our shareholders and other stakeholders, who have listened to this call and we will talk to you again soon thank you.

This concludes today's conference and you may disconnect. Your lines at this time. Thank you for your participation.