Earnings call transcript Q1 2023

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Operator

Greetings. Welcome to the Abeona Therapeutics First Quarter '23 Portfolio Update Conference Call. [Operator Instructions] Please note, this conference is being recorded.

I will now turn the conference over to your host, Greg Gin, Vice President of Investor Relations and Corporate Communications.

You may begin.

Greg Gin

Holly. you, Thank everyone. Good morning, like us to everyone would and welcome our portfolio joining on for update I thank conference call.

is and website recently and ASGCT Our from at announcing the The www.abeonatherapeutics.com. today to are the new is additional objective ISID at positive press our release EB-XXX at available AAV presented programs meetings data ophthalmology scientific congresses. share data on our

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

Harbor Safe on the of to are pursuant those made are implied the forward-looking results based statements securities expressed federal are subject actual in These from forward-looking current provisions laws. differ and Forward-looking the and materially statements. may or to statements change, expectations

but Factors limited the on www.abeonatherapeutics.com. filed are Various identified under our in those section with are documents at not and factors cause the differ that could periodic Risk to, available website Form actual our XX-K results These reports SEC. include, to

Officer, Seshadri, last AAV us Dr. animal Madhav ophthalmology VIITAL study prepared the Chief will On and Officer; session Vice Vazzano, the Medical Chief Dr. from new Financial Kevany, will the call the who we Executive Grachev, review at Dmitriy will remarks. Officer, today give Vish ASGCT Vasanthavada, review Business Technical Q&A Brian proof-of-concept joining data who week. Chief data for are Chief opening program Dr. the Officer, our some Development. After presented remarks, be at President, who and ISID; presented Dr. that will Joe

off. over kick now that, Vish I call Vish? Seshadri it will with And to the to turn

Vishwas Seshadri

in debilitating the awareness tackle Biotech, start researchers more a by option options for and morning. EB also thank for improved anticipate patient EB, about morning, disease epidermolysis EB great market, genotyping we I bullosa, I congratulate saying you increased first for last regulators this Thank bringing genetic to want for joining everybody, community, was Krystal Good to to us want painful therapeutic diagnosis and you, and community, or and come the the advancement disease. as the therapeutic week space therapies this this today to Greg.

with easily Recessive covering of connective dysfunctional of body. with the often in tears that tissue both a VII most a and for of are skin. which wounds resulting the developing the copies painful with gene dystrophic and skin Patients outer connectivity collagen the RDEB, EB, of disorder proportion large mutated RDEB inner fragile significant and between their struggle blisters face remain patients or is in that lifelong unhealed, lack layers a

in Recently wounds wounds. types published RDEB RDEB describe distinct the natural data history RDEB analyzing of XXX two

On wounds, the on chronic the recurrent the are while RDEB wounds. open are bottom top

plot show healing recurrent while the wounds. at and again. chronic and frequently On wounds between Furthermore, Stanford, wound was by chronic right, open [ph] spider wounds show wounds the presented greater a the healing, team corroborated distinction recurrent or this large this ISID that further year, XX% the study rarely at completely prospective

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applied that the and fashion. three were see achieved healing wound sheets area like in a coverage [indiscernible] can You with large EB-XXX

XX% were greater scored wounds tattooed than healed. as right, far the On

healing. the picking to the were naked cross, healed completely the confirm wound yellow because without at you they Although scored unable looks see there, XX% to physician that craft nearly plus the the is healed eye, complete

scoring stringent for wound complete the our to speaks That closure. criteria in

transduced VII works basis discuss wound in -- expression functional sustained a delivers keratinocytes. vector. onto wound keratinocytes are fashion. skin to I'll genes instantaneous in closure. slides, for a EB-XXX copy of culture of patients epidermal Collagen the RDEB two a through sheets are open corrected next a sourced how transduced wounds secure COLXAX that and from are The transplanted healing with patients' durably further deliver the the to Over molecular EB-XXX biopsy, cell retroviral grown Keratinocytes cultured restoring by into

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I'd results presented review Medical were now who at like call over the that Dmitriy will turn to ISID. to Chief Grachev, Dr. our the Officer,

Dmitriy Grachev

Thank Vish. you,

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selected chronic was size, with randomized each and wound anatomic that large undermined possible same open patient study location to years the chronically with Each with would had remain simulant paired wounds being two Our treated the patients. extent a and wounds the controlled controlled. minimum

greater that XX X analysis study of open aligned although years. chronic have and endpoints for In We the area as statistical with VIITAL, months minimum centimeters we open remain than define a on FDA many and for plan. were large wounds square wounds surface

that benefit. healing self-heal. as is or co-primary of targeting XX% remember, than Given co-primary to for endpoint treat endpoint wounds EB-XXX large on chronic because of the open wounds, cannot pain remain the to second of clinical additional greater the equal wound and measure that they reduction hardest years Realign

associated and first, baseline, pain with comparing RDEB healing months' or direct point change time difference wound match size XX% reported reported as from matched standpoint randomized endpoint. at of first [ph] points control of second, at to mean assessed as patient. and this dressing Specifically, wounds is of were; wound control by greater than with score the Wong-Baker as by in treated end investigator a wound co-primary randomized treated the FACES the proportion X-month between co-primary the by patient sites reduction This pain equal the the X study, determined scale assessment; was pivotal

weeks treated healing assessment healing sites severity proportion and of control was for each size additional from included pain XX wound reduction endpoint at and matched comparing complete wound well and with with blistering. Exploratory wound XX. as the for secondary as baseline, randomized wound endpoints RDEB assessment The

top I'm to into characteristics results We line large treated with with is the call wounds square and here. the details square baseline the to centimeters randomized of severe remind with centimeters, per patient EB-XXX during VIITAL want go of of range graph. size VIITAL. reviewed To wounds treated to pain XXX XX the was wound area body exactly surface areas centimeters. included size XXX just the [ph] the and square clarify, in associated of the you Median XX

For is centimeters, multiple [indiscernible] square could areas fashion, the the that considered distant in each covered chronic large case wound. be exceed graphs graph applied area by which XX

than area square the wound centimeters, debridement the prepare required And was a to wound feed to XX less bed graph. was

debrided. chronically X wounds wounds the months On months control years. were treated very that not was had randomized XX or control of wound duration. the duration had wound Median The open remained is contrary, similar that wound wounds

Now and was are treated reduction X% from including XX graphs, EB-XXX to treated improved was subsequent standpoint wounds for compared weeks Pain wound charts gray. time turn weeks. scores healing at or showed week with assessed, early early Wound as you including results. in Consistent we the to treatment in assessment at all and was To wounds very categorizing points and blue in pain X XX subsequent the wounds help weeks time assessed, which XX% which caregivers by great significant with sustained XX wounds. treated can wounds for of wounds pain -- at follow the EB-XXX controlled the standpoint caregivers all observed was much in weeks. and statistically control improved seen EB-XXX for baseline sustained points improvement was much at X healing,

In addition, treatment. treatment with greater scale patient-reported sensory using a home significant measurement with week system Wong-Baker improvement early severity at called pain in using PROMIS EB-XXX showed also achieved pain as assessed assessment Pain reduction with pain outcomes X. was score EB-XXX FACES as quality information significantly

and In patient EB-XXX significantly treatment. showed pain, reducing blistering related reported improvement reported significantly each greater caregiver and addition outcomes with to to

of As each that increases infection. quality and of for a life trauma, factor is significant impacts risk patients RDEB a and reminder, blistering

tolerated tolerability. VIITAL, with shown pivotal In you, in to serious VIITAL and favorable Now Vish. let's EB-XXX positive demonstrated this well no results patients in safety Back EB-XXX conclusion, treatment-related adverse clinical review profile with and a consistent risk-benefit to observed and for RDEB. be past safe trial experience. events was study

Vishwas Seshadri

qualification, consecutive progress of complete milestones towards our process Thank are submission validated PPQ had commercialization. one the readiness significant commercial performance or three for production. you, runs, previously and Dmitriy. and to We process was We've key demonstrate made BLA marching to towards mentioned,

data the important module We was we and drug the CMC vector last critical of to to both are BLA product generate which to step submission. manufacturing, manufacturing today that EB-XXX complete completed for this we excited the had for the piece have retroviral share

first third XXXX to our track on discuss this XX, date, the acceptability BLA the early request FDA. of July Based release and and pre-BLA request we in submission results XXXX. is announced BLA on since content are application. of pre-BLA We meeting quarter The we our a the press XXXX for submitted meeting on that the the anticipated scheduled quarter format, accepted in has meeting FDA had with anticipated

expedited sold $XXX company, marketing biopharma companies other prior approximately receive application anticipated PRVs review on first timing in XXXX. we of anticipate expect second we priority BLA voucher, submission, BLA review approved, is early to the quarter to for for by of which quarter to been the If different sold the a of product used FDA be or Based or potential approvals late a granted the can another BLA subsequent being have million.

of market part commercial pricing health across for and As we the and and EB-XXX. health care with stakeholders to access EB-XXX, care for engage better our payers including planning providers system public continue private understand to

feedback are support encouraged treatment in received a these healing advocates with line by and decisions from we years. that initial We reduction from value feedback delivers and the and stakeholders positive and the pricing have coverage organizations, for wound which collectively patient one-time pain that of

potential. very initial Based discussion, and payers as view of with with its the treated budget profile benefit overall high clinical cover favorable and has need high the unmet to EB-XXX willingness giving will on believe EB-XXX EB-XXX limited access RDEB impact given payers Also, debt, a policies our view wounds. well with differentiated indicated durable a ultra-rare confidence a prevalence have our for in us disease have

proprietary tissues disease the the turn our programs. retinoschisis evidence We of preclinical early and to autosomal in investigative for construct express ophthalmology and presented of to rescue animal phenotype therapies proof-of-concept atrophy. our dominant provides for disease, Stargardt and models. protein in data AAV at data optic The preclinical gene target ASGCT proof-of-concept X-linked mutant potential gene let's Now capsid recombinant AAV-based mouse

I'd now will over Caveney, call turn Chief ASGCT. Brian like Officer, to Technical review at to who the the Dr. our presented results

Brian Kevany

Thank you, Vish.

library capsid At with findings for AAV-based the the diseases AAV animal posters from encouraging therapies internal in-licensed ASGCT, proof-of-concept using ophthalmology capsids We potential new novel broad are serious our highlighting treating eye and by our three AAV we experiments program. presented from from research. AM excited

to treating the The juvenile first poster most approach disease, presented common form macular a featured novel inherited Stargardt dystrophy. of ABO-XXX,

cells in the vision photoreceptor gene, photoreceptor cell caused Autosomal of disease substances death by removal is ABCAX in and from mutations the starter toxic preventing that result progressive loss. recessive

recombinase to designed evidence vectors full length expression. that strategy ASGCT large utilizing in recombinase system. efficiently dual gene can compelling provides Cre data two is efficiently ABCAX ABCAX proprietary to is the genome Our Cre-LoxP by the standard gene, independent leading The full reconstitute too AAV ABCAX at a length fit protein the strategy utilizing to implementing reconstitute AAV AAV which a vector

in Key is dual AAV within properly full yields vivo to conclusions of recombination recombinant our segments -- using human ABCAX outer include vector presented length Recombination Cre-Mediated ABCAX photoreceptor treatment. X and month system, localized detected

safe for results evaluate treating of paving Our for disease. Further that vectors model a AAV novel Stargardt dual in in knockout in of lipofuscin full-length mouse human and show ABCAX studies expression Cre-Mediated mouse effective the on way is delivering Stargardt Recombination model, will an of a disease. approach therapeutic ABCAX a accumulation expression of

dual large using therapies adapted genes desired. is be also approach in vector can Additionally, delivery we of for Cre-LoxP believe AAV-based other our which

composed of common XLRS layers damage human therapy present improved Our approach treatment the functional the approaches. is photoreceptors a discontinuity at novel within that of capsid an cavities second of to for from para-retinal retinal provide macular of renders circuitry, of the that profile ABO-XXX, AAVXXX for loss. life to dystrophy of which a expression. but administered in males. first safety form poster RSX photoreceptors most X-linked treatment the between ABO-XXX presented a and susceptible more RSX in with ASGCT leading be or XLRS effectively in injection restores package the developing degeneration retinal will where patients via gene decade novel ABO-XXX and targets photoreceptor retinas AIM will invasive disease retinoschisis, desired, the inherited is targeting vision in XLRS, featured

treatment associated demonstrated preservation ASGCT the ABO-XXX and overall density support well in as as at cell and the improved expression XLRS. of outer with The mice the ABO-XXX in improved restoration retina, RSX was survival data function. the of retinal with photoreceptor further Treatment development robust cone presented architecture. photoreceptor for photoreceptor These retina mutant of results

gene vector loss associated or atrophy, composed the ASGCT cells in therapy of presentation retinal human therapy mutations them the dominant The with residing RGCs, make target by poster autosomal gene a is loss OpaX of previously. an of third an for OpaX at AAV featured ADOA, Our investigative or optic the of dosing Gene. candidate vision form caused interretina para-retinal RGCs in attractive proximity discussed to ganglion packaged by the vitreous the AmCapsid-AAVXXX route. for cavity The delivered a Gene in

OpaX improvements dosed disease both gene optokinetic and intravitreal of presented and data and retinas nuclear proof-of-concept development confirmed further our observed cell improved in consequences XX-month in layer brain mice. study expression mouse the candidate. mutant functional and treated in outer photoreceptor of The an responses of following visual efficacy were acuity the thickness These retinas improvement in culture type gene therapy a to retinal signaling test therapy OpaX Initial restoration injection. with designed of wild model our animals, an to OpaX suggest and support candidate benefits OpoX results in

Back you, to Vish.

Vishwas Seshadri

Thank you, Brian.

place for I'll turn our this section. call to over for taking programs Holly back We looking the meetings With to Q&A of the quarter. with are FDA the forward pre-IND two that,

Operator

coming first is Your Jefferies. for at Instructions] question Maury from [Operator Raycroft today

Maury Raycroft

little the label. bit Good you Krystal's the of was for patient the morning Hi. et with updates if congrats and and expect can a saw expecting Do about cetera? Vyjuvek presentation relatively the same on a you talk approved in on you're type including profile Maybe today. based label. wounds, your broad what conversations agency, with the thanks We

Vishwas Seshadri

good Maury, Thank you, and morning. for the question

have EB. chronic that study that focus Regarding It's our very the going with dystrophic of I on process treated broad label And the to comment patients, large agency. is has criteria patient and the pivotal want would wounds. to indication this and early restrictive given, there the and types, RDEB or reiterate dialogue inclusion we recessive unmet need, be course, on ongoing be an to in BLA that's high how the the VIITAL

evidence I can unfortunately, of give is have but I that cannot what answer, for. types you generated definitive So a assure we the wound

Maury Raycroft

could out feedback across having how sense. about And I health thinking Maybe has XXX,XXX. you care system makes of Got inform can pricing? how initial a Vyjuvek it. mentioned guess you're anticipating does talk that with about from if little pricing list about positive. price That work a that you're been you bit the how stakeholders

Vishwas Seshadri

you Certainly a important that a Madhav move give of one-time over who can without where leading that. call the well for with the talk clear again. we to our years having proposition payers stakeholders turn to like Vasanthavada, can will administrators, same as lot about of benefit value is discussions hospital I of there's a treat both the treatment as appreciation

please? we can discussed you differentiation throw pricing, if Madhav, have on light how the about and So

Madhav Vasanthavada

as well EB-XXX time, at And all given payers what healing Vish. had really we a to compare profile with research nearly we to find dozen the launch Maury, reduction research, be that question. investigation placed pricing. profiles discuss Medicare, that a Yes, will how Medicaid. and price be payer Vyjuvek's go including be, had for it with for how as potential very clinical both entry commercial, encouraging with payer And we transformational thanks for mostly EB-XXX, durable. Sure, scenarios we to now would conducted and [indiscernible] very the groups year, Hey, the price for Earlier EB-XXX the to the few very blinded that across in price profile, different insightful for of when elasticity very we was the therapies, all the and the in would encouraging. if pain which as find clinical is about hear Vyjuvek wound price

the for to the position be learned But in we to innovation Vyjuvek sure that able much patients. for EB-XXX we durable to effects value is, the And capturing there that will making to for now is that be pricing what pricing place better and EB-XXX a we have be bring. the time, at hurdles are able to the same also no access will

maintains. think that EB-XXX. I now given be all, in a elasticity able be to the price price in launch profile we place will for all we for So price a a better position And the have, to

Maury Raycroft

it. Got

Madhav Vasanthavada

help [indiscernible]? Does that

Maury Raycroft

data that's and gene maybe helpful. question, in queue. looks Yes, with good. It back last And the the ASGCT sense hop understanding therapy then The I'll makes value proposition. from

program talk about as Stargardt opportunities. about Just wondering three more if of little you think XXX with Or lead? bit those different a we being should how how that? prioritizing the the we you're And should could think

Vishwas Seshadri

a turn it may it additional talk little the be over about for now unmet models and can Brian compelling high gene those you there's aware, that we as indications. have to of Right these bit mice three data then I expression disease in any in of showed the thoughts. Sure. need from preclinical all

to chosen have We how correct way. course, to we ABCAX are make on specifically and see and encouraged many able the produce challenges investigational those some about the to among other along that and therapies talked are gene uniquely of based size Stargardt have the the that were address inroads tissue. three indications hit the most of And able

it's we Maury, prioritization how dialogue to the be have of In of resource going a and on. terms programs, lot going we --

equally needs. programs at looking well are funding unmet non-diluted government different these And all and high are and potential of even sources that some funds. various are We as

held much us, nuances animal get discussed will we can events our not going It's other to progress clarity that drugs. be to a it are really as calls will of our we and so on more having going to these have we when. studies other types And an little develop upcoming NHP a bit matter make And quicker just such or be tells mean, in with the further of going to agency, it's from dialogue be data make also because [indiscernible]. which to be I more

there prioritization. lot to other equation further which is the will So of pieces inform our

Maury Raycroft

queue. sense. makes Thanks the That will questions. I taking it. my Got for back hop in

Vishwas Seshadri

you, Thank Maury.

Operator

Your Kristen Fitzgerald. at Kluska next from question is Cantor coming

Kristen Kluska

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

to had related I ophthalmology first the The portfolio. was question

yet So for we gene others talked in vector some time. strategies that something many dual therapy. about know it's haven't seen AAV as have I

vector about the the findings of latter you successful stage. part just the out guess first into is question just, the at how the the are what dual expect your the expectations of given And what frankly, for, would landscape, models. AAV to human this key preclinical understanding you looking construct translate is is question led I differentiated between maybe to studies. look the that difference the to things that this at And on So then to think half

Vishwas Seshadri

to for on say going just Hi, that Kristen, Dr. to over thanks that, we can in you approaches ophthalmology, be ABCAX think thank certain Brian specifically and the but we for there some have other AAV be the this of other will Kevany, before do why the getting which -- methods question, turn we I full where I'd question. I'm it think are because expert elements successful fail past. mechanistic of length

unique and can dual the you constructs Brian, light gene some please So our throw on system, please? AAV

Brian Kevany

yes. Kristen. course, Of Thanks, Yes.

for I So of tried, genes those ABCAX. as have strategies that number programs as haven't number movement. we while a lot dual been going have seen AAV for been mini vectors there And both been on a years, of of have think in well a

length to struggling that ABCAX. think So full ability construct we that our field provides encouraged is I into to still this and push get do a were by

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

where using things one homologous So where we I the and place the are pains recombination that as not others seeing Cre in as they're high what think of with be recombination the efficiency we recombinase where from differentiate may system. like is

the mechanistic repeat to your of trying of therapy about So us Kristen, about part are [indiscernible] the that and I second question? actual aspects we you develop here. the differentiating think major And for your is less the point more could

Kristen Kluska

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

Brian Kevany

the here sub-retinal Sure. Stargardt any the mean mean differences. significant are a approaching don't this for I Yes. I we I is delivery with anticipate advantage program.

specific For diluted a close very this out injection the and as those occurs you of were as to retinal does injection epithelium. pigment intravenous keeps very This is vector the providing or that that other of photoreceptors the action site relatively if directed get between it aren't the an injections. and not aware, types

I larger of So keep say, somewhat -- are into to moving animal human the a as during IND-enabling mouse studies. aspects it similar model to think the that between, between we studies and then beyond

So be dosing. large volume I large of studies and relatively injection to think the the going of translation the exactly from, human. animal human between the is animal type to be exactly the smooth lot -- the be the say, same We'll the because using model same a will the

to think So is from large to of Yes. that be relatively sort going I translation seamless. humans

Kristen Kluska

acuity And function the assessments here you. presented on, that demonstrate autosomal for atrophy Thank then have dominant visual the that recovery. bullet study you optic that you

is goal a progression the the with show slowing reversal. I AAV essentially think of lot therapy gene than disease ophthalmology to rather of trials, down

but So about progress have And what rates, different looking it I to you're how going intervention. at comment. understand context in as with is at age a will clinically And kind interested goal that the be. maybe I'm guess the lot of underlying diseases do essentially different of of you this and these to just of think

Brian Kevany

Yes.

this studied time. with acuity we've over looking visual study acuity improvement and something at visual that So or preservation of animal mice is in

in to mutant animals they lose vision. as have start visual age, the they these prior on was This age, degradation animals, of that their high the treatment early So disease provided very acuity progression to to acuity. any ability

development. prevention really a of that it was So

of progression in something it a benefit. And primary of instrument be a is therapeutic because their trial. diseases to takes a a at time visual for slow of bit for these the tends We've specifically visual blunt the endpoint visual acuity of and to thing study the that a looking where number And acuity, in acuity was seen it's develop. long because to difficult humans failures industry

is in I discussions understanding endpoint our be made last ocular So space capture capture primary right be benefit. available are we're to the think part disease that of going This rare topic now. specifically ones with agency, hot endpoints meaningful benefit very the at understanding in as week, at therapeutic about better looking to how that a decisions but may ASGCT over not a therapeutic general, that are the

talk So acuity. how think there, to that we visual sure by benefit make we will are be something like end of have probably we a as sense start not to is we I but regulators, capturing to want that and may better that captured approach points

Vishwas Seshadri

the first because to visual cessation course, that's pool happened disease. or clinical But to also function, But priority disease our is the goal I that, if have looking bigger of going we prevention it's deterioration or may prevalent reverse And where of any eye to can it's Yes. just of the that forgone add already enough would right? with Kristen, see going be disease what you early to we're acuity. a therapy disease your monogenic symptoms is be, to for terms be at development, treat also of gene goal appear don't patients a preserve of in is you

So we're maybe going populations reverse may I that, to [indiscernible] start, types XX vision more where even and test of those be visual subjects looking all to right. have for don’t can to maybe XX of deteriorated know, function level

the signals clinically are So in happening deterioration going place. we prevent those and to is to long-term for all the from course be goal such first of investigating

Kristen Kluska

also to me effects for these and then And were question the just RDEB evaluation understanding of some findings other around new key last secondary and Thanks. two ISID, endpoints. Great. point related then then earlier

all with correlated you have data wanted we have hear about are? now you as talked So since Thank how again. I other understanding these leaders, to number past healing, two wound thought to looking a notions? components but data, of you you presented the in reductions these know I pain discussions these at with And these important points support

Vishwas Seshadri

I that, Thanks Kristen. take for will that.

We're number top XX months one, the what in physician's these you we XX of ISID visits The kind regulatory is, do which they're focused presented the assessment, we've the they're X sets onto So what and wasn't the early X seeing line. patient see reporting, is home, ISID weeks, and in weeks, pain are things the weeks, every even an a has couple benefit saw to scoring highlight at same at writing well. discharged right. example that sustained. right? the at point, to timing X weeks, benefit the of fact, data how that much delta seen untreated because of that, effect that and that pretty they levels after do levels and kind we in the diaries a for you effects, and we like X weeks quickly as of a wasn't endpoint caregivers wounds presented whereas endpoint and where weeks line maintaining So treated just top where of down [indiscernible] soon, see that wounds there time can clinical And went even which

note. important So And that's that's to key.

showing had building time not consistent presented, the have some been be is the And we've this wound those also the -- important on and to KOLs. discussions of that that a data very the that's very also were that correlation proposition. points encouraging reduction see and in value of between going one-time healing discussed, new lot over point the that's And The is level a further with course very post-treatment. that's is second it's it's previously pain to

see. factor versus Dmitriy data and also Dr. had encouraged we're be something improvement blistering trauma. dramatic untreated. for that's Itch also we a Itch to that a that's And showing presented, as heard, Grachev can as you for And treated further risk And

that That's this to a be asking and And us conducted what conviction question, value. the all as coming be those for up encouraging current for what's benefit back patients what holistic us source beyond and manufacturing really like lined what the through runs we is source went their So control to and something is view that that. to they've treated opine our we they're all patients our patients. KOLs hope of that confidence this And your ongoing I PPQ. going things to who are see a VIITAL in Kristen? which for study, ultimate answered the point, is data wounds is that of and is

Operator

Your Global for Molloy Instructions] question Alliance today [Operator coming is at Partners. Jim next from

James Molloy

estimates Thanks morning. to questions. earlier, and about year, on estimates? they're the not think U.S. patients, I you their there numbers, change a publicly but after think saying I obviously, call, hold in due your think they've for market [indiscernible] I or how a a approval. a they're my put what for want Good where up XXX,XXX question guys. do And the government of Hey, can XXX,XXX about given just down, as about size target and X,XXX guys' their discounts to pricing competitor's their to out a year talking taking of come follow-up those on sort --

Vishwas Seshadri

the that in encouraged that out, what were pricing maybe scenario post pricing gene the by the results way. mentioned, these of we then a Thank Jim. this results, Jim. you of pricing placing at question, VIITAL reiterate clinical would their for terms are Madhav are the definitely different did play therapies. the what in We value stakeholders thought we a as take considering. I But various note we profile in of ago clearly, is, of point types having are year what relooking time, at case in And in seen best we

currently is, Now label broad. what recessive the are -- focused know our details clinical includes about of which debt. is we we the looking very EB, half on dystrophic is let's don't for Vyjuvek's all of EB, say, many It at #X, whereas dystrophic evidence

relatively as really an proposition and because is the well. disease is value it's narrower it's smaller comparison rarer so a different apples-to-oranges -- space RDEB a And

you -- the mean, have redoseable one I hand, a it's on gene therapy.

in because that estimated do on year homework these real we what world turns world we like right? we other don't the then our works we you hand, retreat the to take comment to things having years into a have because soon make sometimes to On gives estimates of out I one-time that and want and out. benefit given about for points the per anything that's how not and something pricing have treatment So wound, the happens and real -- definitely price exact a will enough, account.

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

James Molloy

a meeting pre-BLA, your Outstanding. has And it then -- again, the be self-launch a this on right Thank it's And on you. partnering? follow-up should pre-BLA? thinking potentially coming should for given would BLA. into a out fairly imagine be then anything potentially that the competitor's approval, the tip we straightforward of I fairly changed the

Vishwas Seshadri

Jim. Thanks,

on complexity the to we question us meeting we that that our have it's first you every there's disease pre-BLA the submission, given the everything sure make the the decision. submission, it these So a to in therapeutic experience BLA no space, pre-BLA, that areas, have limited behooves that asked have of therapies, warrants

pre-BLA that kind itself meeting you but step former that's -- that a So of is the have.

that I we July on for one; of exchange. have interaction informal and having suddenly agency, as series been the and of a However, just There's meetings with even lot XX, we've meeting assure we like we the it's to not want complete a agency also with have silence speak. appearing --

scored the we will everything the this of step be in culmination put to. saying you, final So to we going needed dossier We're like right? that front make

not those PPQ release all pre-specified have the work have completed piece we but of that pull drug that those should is, and because, that runs we was to process the everything about we're that is within criteria report meet of all to just fall consecutive So mentioned, show parameters pre-BLA range products, And and we EB-XXX also and need biggest the to met and together we for the to our that happy to to goal manufacturing the package. had time robustness had process what the various parameters where the I it's is finish are significant three the in confident the be what's meeting runs, we as we that we successfully.

So that's for the pre-BLA and the submission work stream itself.

Your And is disease. this second question was about partnering rare commercialization. a and

sales therapy -- going demand to full working closely is on this for the be going and are are us marketing in the we the have where on and of advocacy to more groups, focus we with We because lesser the delivery centers and patient administer the communities. our

be the launch here how typical going on scenario patient needed. do focus to the services we So that's what deliver that there's is a going be to

is So partner kind profile than partner given a of of a a if typical get very partnership. pharma the we different profile be going to large that, a

to are open we that. so And

XXX-pound which [indiscernible] our we started once do stakeholders is talk we of those we looking at step a we've created? readiness where we payer ready want more why and submission, be going BLA partnership score to a And we gorilla the conversations the want of to of is and to right deal certain are while don't kind to types the hands that do options And course, time, is up already, which value pieces time. At the same the ongoing, we retain wait now. at through get also ready how pick are only of the are research should to still steam in we there is to because make one launch forever

we the commercialization I are you some next that as how of sense looking at gives step. So hope

going lot second limelight. half to is have a this to So up the year that coming of conversation of

James Molloy

for taking questions. Thanks Great. the

Vishwas Seshadri

Of thank course, you.

Operator

end the Vishwas remarks. to for the closing over question-and-answer of session, reached and I have the call We turn will now

Vishwas Seshadri

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

Operator

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

Abeona Therapeutics (ABEO) 24 May 232023 Q1 Earnings call transcript