ABEO - Earnings call 2 2022 | Abeona Therapeutics Inc.

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Operator

Good day, ladies and gentlemen, and welcome to the Abeona Q2 2022 Earnings Call. [Operator Instructions]

It is now my pleasure to turn the floor over to your host, Greg Gin, Vice President of Investor Relations and Corporate Communications. Please go ahead.

Gregory Gin

Thank you, Holly. everyone. morning, Good to on like thank for everyone I second welcome quarter us our XXXX joining would and conference call. announcing The website release the available is press on at www.abeonatherapeutics.com. results our

Vish Kevany, today we joined prepared Research. Abeona; call Head are Joe Financial we also prepared Chief Chief by and After Vazzano, Executive of Q&A will the On host and remarks are the Technical Officer Seshadri, Officer with of the remarks, Brian Officer. Dr. Chief session. And

review safe future and harbor will made I today's call projections Remarks start, contain during forward-looking we Before our events. statement. may regarding statements

annual Forward-looking may statements forward-looking cause of statements to materially or section expressed and are differ the under differ change, and the documents Factors the safe These the on based available in actual harbor are to those current results SEC. expectations www.abeonatherapeutics.com. from statements. could website company are on those are provisions that made company's identified in Various on pursuant factors to, to are are not implied laws. filed at entitled results federal These forward-looking actual securities by Risk subject periodic the and limited reports report include, Form the with but our the other XX-K

I And CEO, Vish call now Seshadri. will with over that, Vish? the turn to our

Vishwas Seshadri

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

therapies developing transformational unmet diseases with serious on for focused are We highest need. the

IIIA, transition development and prior agreement direct Ultragenyx of decisive continued certain Sanfilippo to we took quarter, costs our toward responsibility exclusive the During costs. In development other or type we A for financial in for assumed asset, whereby action May, MPS syndrome our resources second including announced ABO-XXX an all the lead EB-XXX.

regulatory royalties is Abeona receive to following certain and return, In commercial milestone payments on net approval. tiered sales eligible

discontinued also ABO-XXX of have MPS development for IIIB. We

additional runway we quarter data the the fully vital puts forge appreciates the optimal EB-XXX to that our The to strong value a the of readout runway second into readout. MPS in of the extend of able position With well XXXX. disposition the were partnership programs, cash beyond commercial us post

For potential in is very exciting VITAL out III debilitating spontaneously line a in the chronic with EB-XXX do clinical study the the disease, we Phase submit to top not application greatest to coming recessive investigating outcome, the in that aspects RDEB be following dystrophic we And that from are wounds most burden. results biologics reading months. potential large pain and It is bullosa, heal RDEB, epidomylysis EB-XXX's license FDA. pivotal positive typically or treating plan inflict of the a the and

of in as early milestone end patient endpoints include This data study on planned quarter co-primary study around target With reporting well when efficacy visit we top in as enrollment top anticipate characterization, report fourth final line achieved wound for reached an results for important The quarter. the March. year we this VITAL the earlier mid-September, or safety. line our third will

of commercialize of with In top an agreement line with continued we the partners our release engagement active EB-XXX meanwhile, the potential after goal finalizing data. to have

Our promise long-term supported RDEB EB-XXX belief from steadfast results is for completed I/II in of patients the study. Phase by a compelling

maximum and data chronic or during of quality life showed RDEB of Society data in an the additional of a I/II large and resulted of follow-up were Annual study presentation at wounds X healing mean SID, In Meeting. Dermatology, Investigative X.X from long-term follow-up considerable follow-up. of treatment wound Phase May, data EB-XXX of of the years years featured a at oral The

remain chronic study, in Remember, we the can wounds Phase themselves In wounds addition, small wounds. I/II more the in chronic spontaneously, VITAL, than burden including and reduced large recurring large close X relief, months. reduction symptomatic for pain. wound treated long-term Unlike open that cannot with was as close associated in

In years. fact, for had chronically wounds treated many in been open VITAL

spontaneous reason, that study. of control untreated the the we arm little healing in anticipate very For

the we an patients, control same of was were VITAL, of XX treated paired wounds In An additional size and the characteristics treated a total XX with severe the the large nonrandomized XX untreated XX associated wounds wound VITAL chronic EB-XXX. within following. large with wounds patient. wounds and each also included and in randomized the underscore wound Baseline pain with include study in

change, each And square. XX. the Pain surface area area wound wounds, included is surface for mean dressing co-primary body at nonrandomized Wong-Baker of the treated pain with in FACES patient second patient EB-XXX Pain which reduction was most randomized per was of intense wounds Scale per to during Mean is if square. time treated Remember, XXX-centimeter using assessed body endpoint you the X a VITAL. Rating is pain XXX-centimeter

XX At the of patients score pain XX of wounds noted VITAL score certain reporting randomized baseline in wounds investigational pain in traded representative reporting opioid baseline, pivotal maximum many only product wounds. pain severe patients the has X of X a a pain that XX can of X Clearly, least patients with minimum problematic pain the endpoint most the a lead is X randomized and that patients have the for wound at use painful of are co-primary the that to for reduction we in study. of management. EB-XXX

has unlike of more associated ability into the subretinal but area robust XXXX vitreous eye detachment. nonhuman Annual not with eye to reported the following our library. The in to we produce ARVO capsid the for been early data Meeting novel At administration, AAVXXX's of primate the May, results directly macular in showed administration briefly retinal from eye capsid administration AAV Turning transduction AIM the our pararetinal AAVXXX, preclinical programs. a which

ability transduction subretinal and achieve AAVXXX's levels macular optic injection. in a primates than high potentially by nerve excited nonhuman with are safer to and We route invasive administration less

these reported pre-IND which meetings Functional Looking the hope will ahead, ongoing undisclosed begun quarter, target of in studies from the of will excited second are early to in that indications FDA support share readouts have be proof with we to studies expression we our to ophthalmic proof-of-concept in to begin half demonstrate gene tissues XXXX this XXXX.

to Joe call the the turn Joe? results. review financial to now over I'll

Joseph Vazzano

like would XX-Q XX, Form additional website, I X on to ended get which on for can June Vish. you, everyone is months that our our remind the the is details financial where available you XXXX. Thank results

us with $XX.X of of for second first as financial quarter the operating the cash current financial of second compared $X estimated runway XXXX. short-term in resources XXXX. quarter XXXX we Starting our and and Net restricted our on into takes sheet, Based June cash, cash our equivalents, million balance XX, assumptions, our on in was used quarter million of activities million plan XXXX. the cash current $XX of operating the had investments to resources of

to million therapy a achieved $X XXXX. a License and the sublicense other resulted clinical second quarter Gene AAV-based quarter of to for agreement under X an quarter of Therapies Rett XXXX second revenue the syndrome. in revenues for with related milestone XXXX in Taysha from the The were compared gene of second investigational

development Turning spent XXXX, compared months We the to XXXX. and the $X.X million quarter X for million to of ended second in activities. research $X.X June XX,

in to and same loss $X.X XXXX, $XX.X compared in the X activities of or compared per general XX, quarter million million ended period common on per $X.X $X.XX share the second was the attributable of $XX.X XXXX. loss the share spend to second was million common for million or loss XXXX $X.XX June for XXXX. Our shareholders quarter to administrative of months Net common as

we in minimum market. capital with Lastly, on the continued July, regained listing requirement for compliance price bid NASDAQ the

Q&A for back turn With to call the the operator over that, session. the I'll Operator?

Operator

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

Farzin Haque

Jefferies. for This from is Farzin on Maury

endpoint, reduction remind you expect us you often the pain for on then point? are what can us the what the standpoint? achieve And this For that the you specified also is same measure it? how And you remind can delta calculations to

Vishwas Seshadri

Thank the you so for much question.

the part pain, is first pain pain are call. measurement the question -- with the we of endpoint that's The patients of with measure. is the and we this when treating today Your the on the in that frequency which shared first study reduction EB-XXX, scores it's prior enrolled for to the baseline what

of the the results XX, points is that endpoint the includes we've important co-primary going measuring with each per agreed most as pain the follow-up determine at to we're our XX week of FDA. week as progression the the which time is Subsequently, which as well that time point,

different has been the Scale pain a Although which was associated to there the study a where is binary of as XX study measured VITAL using scale, X in measured a on with yes/no FACES in measured to rating pain in a bit well how pain a our chronic it's Phase I/II wounds, Wong-Baker difference in scale. is little large these

is data pain that confident size meaningful able to reduction. tell on of in we not study exactly scale, adequately because VITAL magnitude different we're to the reduction powered our the while that what itself all a past detect So is we anticipate is a the of are pain you

And the pain I again, wound a for have pairs. pain XX score had least that minimum of of going patients to shared, at X X back their of scores baseline X the

wound give best reduction being is you of sense a healed. the and scope should a could that there you completely is is, broad much So pain how achieve for pain X that score

you apologies that question please left repeat was that. was part, second can there And The your part if of unanswered? for any

Farzin Haque

calculations The this. go prespecified into that

Vishwas Seshadri

arm. calculations the essentially, prespecified is, treated we're is what The doing Right. looking at

and these to are randomized XX going there's intrapatient paired. So wound pairs at, we're look

of test significance of set treated So pain to the we wounds, arm, untreated -- the have XX a seen compared mean the statistical is across treated XX controlled it's wounds. a reduction which of

Farzin Haque

And these been is mean else this if say anything [indiscernible] you're and long for? the had how they But sense. then centimeters you how wounds. patients on long comment Makes there like have can for then the And saying median? And wound, wounds range XXX like chronic the you have Okay. square. open are the is can

Vishwas Seshadri

Yes.

first the the So size is question itself.

reported way all XX mean of body square square. that wounds area the nonrandomized surface covered that if per include from the And you XXX patient randomized per goes XXX. to I'd So XXX-centimeter was is the The wounds, patient. the for range it wounds

So area hope patient range, the that's area so the addresses surface covered per question. I body surface that

results, have wound the which open these set that many it's not years, at for anticipate these years been And how know our long is of part trial baseline the had X top characteristics. opened we of means will reporting of be And spontaneous even minimum why answer a and do we been reason be be wounds We line for closure, second will in numbers the for clinical been the will the for have control been healing. looking we open question, admits small months. very will we because protocol be of many itself wound there as the have of wound wounds though the that wounds -- our part a opened, that

Operator

question from next coming is Kristen Your Kluska.

Kristen Kluska

at Kristen that Cantor treatment remind for might durable you is This about single viewing these And how please how the do market opportunity you estimates, you're EB-XXX? us wounds Fitzgerald. Could in think a may down require? over do require then patient the versus based road others average you the an think patient that long retreatment? profile, that be And term how sheets many on may

Vishwas Seshadri

you for hear you. have Thank great here. I question from to and Kristen, question, X-part the a

sources market first, about U.S., the We based organizations epidemiology and at advocacy the that in have let's with being we as talk anticipate validated something well. that's opportunity. X,XXX patients, this is also So on least current

X per those patients treated large large their anticipate that of patients, XX% those wound with wounds area at that we EB-XXX. body. look you specifically average, in treatment if cover chronic roughly cycles the anticipate surface chronic And should be have we patient on will of will And

north you opportunities boil treatment pool of current opportunities, epidemiological to with down if kind of that patients and we have that's you're X,XXX the U.S. treatment at of of the the patients, looking we the have. and So math of base do so number

treatments. asked treatment about And a proposition, question with value one-and-done chronically-applied I single you think, a the versus

Our the a a product EB-XXX given target profile wound for one-and-done for goal treatment. is and

we've our would X looking Phase So and wound reiterate we study, years a be and data we that's seen to far the from to sustenance well. for as as value price the of at how follow-up proposition reduction, well as onetime look as I'd up I/II that pain healing EB-XXX with important treatment

And and chronic to And been a some these and And this both for chronically-applied at informally, physicians, -- guns I a to through and wounds; be for there be ad both just products. disease which the point small mean, the patient, is large for one-and-done are therapies that going treatments, have boards during recurrent approved formally lot to patients. no such. big we're any are the where disease for talking we hearing wounds, course are of needed

to going not disease, we are patients should something forget. the their all that that's see types of in anticipate course we So of that treatments so

the EB, is in we and forms what see one to patient about almost understood of chronic are And all a large severe lifetime. profile, of EB, going which recessive patients have dystrophic wounds their most

In that's very with large fact, there that are wounds important something recognize. so children as and that's are born, are they born chronic and to

much profile this therapy. anticipate profile, patients require hit that's And pretty have the so of parts you patient a RDEB the the of I entire of asked, we wound all a kind on not I just to will hope going Kristen. base question

Operator

from next question Jim Your Instructions] coming Molloy. [Operator today for is

James Molloy

at point It's you're it had know waiting and on this But the on would on potential I RDEB? partners a partnership how looks. Alliance Partners. I characterize obviously, Jim Global depends question for from how quick potential data you which opportunities.

pediatric eligible the can dollars hundreds with and that And you the about you earlier are these actually hurdles for, getting voucher and more has been millions -- for What there is that sell the of get. the And as voucher more then well? rare to seem on given of get get that? things than discussions certainly to partners bidding also should there approval

Vishwas Seshadri

Jim, question. for you, Thank that

type question answer first, the partner commercial the that of the ideal -- looking me about do. commercial So partnership we're let to deals and

the way launch would of while is far our the them this to yet all the to line and so license we've to, commercialization as where course, EB-XXX. the that built the of partner find value activities be approval. as the to take into technology goal basically would Our recognizing responsibility role all well partner And be a the finish commercialization of BLA

also approval. important that's going upon So of BLA to be and potential supply EB-XXX

here clinically is that will have past is important we be recognize de-risked. Now deals done, what unlike to that EB-XXX

with had FDA. with we're a study a co-primary partnership the Phase III after looking pre-agreed starting we've at So that's positive endpoints

value and very it's any that for a asset. So de-risked appropriate deal de-risked give will partnership program, us the

also be what forefront of would So deal that like. look should the something in the

an you terms we in pediatric One is X the the should question have with that Now opportunity a rare voucher. priority of There designation. is the requirements review for rare pediatric have priority designation. PRV, are yes, for approval, us about do review voucher BLA a

one the which the you second because playing The high unmet is application, priority need and believe a review for that your will space that of we get in. we're own we

itself. voucher apply it you for then The review will is get submit the hurdle we something is only the So BLA we approved. the BLA And the as approval priority application. when typically, gets BLA granted

hurdle, going only package the together the So robust most the that's a is we're are we committed. BLA. positive, study putting for be When and to

I on the that So aspects. hit hope all

leave a commercial what de-risked speak thought of Oh, just we're more and lot study like I'm a this able partnering but partnership a to would assets deal III on. the positive not specifics with a about with that look is the that Phase

we've done what, valuation lot uncertainty clinical have example, ahead for of that of with there's from still a So ABO-XXX, us. a different will that's where

James Molloy

follow-up How how have through. hurdles in -- it process still to if things mean, could. what acquirers potential go Reaching early once they it How already you if will at good to [indiscernible] Does right, in still some that process it? I you I got out to start? does start obviously looking process? a the know Or get a voucher? here's of would a but quick we're get to that and sort priority go, say you only of to what I will. Maybe early the and

Vishwas Seshadri

this your So I restate Jim, so question, -- if I correctly. just understood to

priority That's short such most we has other BLA, need to voucher? that for up the activities compared right? review a potential time a get to already relatively of lining do voucher, something buyers we if getting that to lead are So happen

still of of So because PRVs what saw And almost was has the pretty we that last are went seeing trend the sold strong. been is north PRV a million. instantaneously you that $XXX selling demand

it was $XXX ago. few I a weeks think million

very timing not the part. about and for worried we're pre-preparation So that

of BLA resources. are -- the that is had anything being securing the we're already to most time capital-intensive package pull to we some in and package, so we prepare resources. VITAL III earnings sense All getting sequenced gives a stage approval together efficient And we important of BLA and puts our us a call, for Phase now. already lined a as you what But XXXX. what's we very up that hope that's us and where the could mindful upon the think X discussed gated with at quarter submission the line critical our events minimally-intensive PRV. of doing I steps are is the is how for result, I resources BLA is BLA previous

Operator

[Operator to no questions Instructions] further queue. floor closing There turn remarks. back are over like to for the any Vish would in I

Vishwas Seshadri

closing, you. results to our from we're In EB-XXX a VITAL potentially reporting Thank for Abeona. forward study, excited milestone looking transformative and top line

Over track the will we toward quarter, results to lock you I fourth care want dedication the quarter top patients. to thank analyze Abeona the third weeks, to and are team continued improving and their on report for and and late database, to data coming we line efforts for early the in

to want on this and stakeholders call, talk have quarterly to thank who and we'll the our you listened call. I also to next shareholders other

Operator

conclude Thank today's conference you, ladies and does gentlemen. This call.

for a disconnect may You this at time, participation. wonderful phone day. lines your and you Thank your have

Abeona Therapeutics (ABEO) 11 Aug 222022 Q2 Earnings call transcript