Abeona Therapeutics (ABEO) 17 Nov 212021 Q3 Earnings call transcript

Good morning, ladies and gentlemen. Thank you for standing by. Welcome to the Abeona Q3 2021 Earnings Call. [Operator Instructions] I would now like to turn the call over to Greg Gin, Head of Investor Relations. Please go ahead.

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

are On of Seshadri, After prepared Abeona; prepared a host CFO. CEO remarks, Carr, Vish we'll the Ed Q&A with call the today remarks and session.

Dr. Technical Officer. We Chief are our also Brian Kevany, joined by

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

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

call I over that, turn Vish. will with now And to the

X substantial on Thank since and ago, Thank is you us about the this transitioning and end. good my since update you, morning. I'm in to our first morning, Thank weeks and you third Greg. CEO quarter to This you, happy everyone. for joining progress quarterly quarter call

in this and time life Abeona our cycle. during leading be to exciting thrilled I'm critical

in the for study of third EB-XXX the quarter and transformational with assets finishing Phase III readout top patient X of quarter the key accrual We rare the late-stage pivotal including are anticipated first and data in milestones months, designations, line XXXX. potential coming XXXX in VITAL pediatric advancing

no therapies as suffer approved possible these diseases our effectively to work have continuing as bringing that from gene are We who patients to safely, treatments. toward and quickly

We Voss therapy and recently step that gene for and License prepare towards clinical RDEB by have and as Imhof big biopharma of submissions Carl as in program goal our Sarepta Denny veterans hires companies and Biologics enhancing to Senior Kate IIIA. taken with AveXis, clinical These Quality, Application include products therapy Regulatory. Cellectis. X late-stage lead our leadership strength industry Regulatory, a bench in bring All like considerable MPS for of Director Head Head as of and John experience with Therapeutics and commercialized gene X

autologous healing patients RDEB a large, demonstrated gene-corrected take of a reduction treatment has latest study. instantaneous let's in years, for point Phase rate the the Now reported closer after follow-up cell at our investigational chronic wounds I/II time of therapy EB-XXX, high that wound look and pain X in

VITAL Phase our are III results similar ongoing We outstanding from anticipating study.

is VITAL As wound paired approximately the a untreated study patient. of target with same reminder, large each treated the XX the treatment control being for within chronic the wound an wounds,

is XX-centimeter Unlike and close are cannot large are square wounds wounds pain for have more opioids. and close of months, severe the severe than problematic can small, often often that RDEB. chronic than that area recurring wounds years, for these open in Large greater spontaneously, themselves and associated remain chronic very and managed surface wounds with with X most

treat the accruing I earnings the have patients, to report we study. last call, since VITAL that am close we pleased to are very and to continued

the study expect last and top them and XXXX. line are treated. the scheduling follow-up identified results We, therefore, first third patient treating final by the have quarter of We XX-week the upon in quarter for XXXX of patients completing treatment anticipate for

XXXX to filing for timing BLA is anticipated year-end early The XXXX.

patients. Medical the study, and VITAL in the is prescreening site Memorial now UMass active Center, mentioned, clinical the previously As second

manufacturing. EB-XXX to Turning

drug Phase product the a As III our facility. We've study at VITAL reminder, are for we Cleveland company. the manufactured a non-CDMO-dependent

We of information manufacture pertaining in-house. X of module to updating IND with have the made EB-XXX the progress significant in

process have in-house analytical We for We a manufacturing in work other that gene advancing at support and a been filing believe assets BLA assays the also and and bringing Abeona our strategic therapy to space. characterization critical CMC is cell key for advantage EB-XXX. activities

the study A that Let's IIIA. turn we syndrome end investigational to we the point with BSITD, infant registration. score for the for of primary and The the In clinical for point raw for developmental definition of call, aligned toddler age neurocognitive platform using Assessment therapy A our our for second XX Sanfilippo enable children, months. Transpher FDA to a primary reported Kaufman children adeno-associated pivotal earnings virus KABCX, development, and program, the ABO-XXX, and Type assessment MPS previous scale on who the Bayley end of is battery a or attain

end amended have submitted We reflecting since points. an protocol the trial agreed-upon

are collaborated to scheduled the with first to the in the in SAP, We XXXX the or experts plan, through a quarter with have discuss RMAT MPS statistical IIIA finalize of we mechanism. FDA which analysis

analysis quarter primary results recently performance XXXX A of treated between of of quarter Transpher observed As second development X the XXXX. a preservation therapeutic already therefore, methodology on depending and the years, is the up cohort. dosed neurocognitive line we have in reminder, to from patients, anticipate we fourth The over dose patients evaluated the the neurocognitive in with and ABO-XXX of XX more a anywhere top period

of amygdala patients shows using treated and preservation the and the reported Related without treatment, volumes development data anatomical Symposium consistent to before of for gray in children neurocognitive matter, corpus We or compared brain we on X the in MRI the in which biomarkers, IIIA and have early are months X The presented that callosum dose XXXX. well increased reported. preservation post XX quarter at benefit with at is brain of and therapeutic in treatment trends MPS treated third years Diseases afflicted at in have young development magnitude MRI, MPS at International excited we the age about disease-specific in as ABO-XXX which with neurocognitive whom unprecedented as was seen the with patients previously development safety as

biomarker, builds all results treatment neurocognitive standpoint indicated and will development effect about clinical FDA of from While the kinds in ABO-XXX, important as that of approval they will this regulatory clinical of MRI consider the brain decision-making. a regulatory be conviction points has the from also triangulation data holistically

Earlier manufacture previously the from be to facility. ABO-XXX sourced at have We to Cleveland drug Hospital. Nationwide Children's product commenced able year, our activities we ABO-XXX this

track of lots using on manufacturing complete ABO-XXX this are materials. X of We animal-free GMP year to

sourced product of Hospital FDA instructed analytical comparability expect the establish of that We from with in tests using as the Children's equivalence ABO-XXX. of battery Abeona-produced first drug half for by Nationwide studies XXXX to the

for facility of ocular Additionally, in This have commercial our foot preparation the square in our AAV also we programs, manufacturing future. initiated supply will facility including the have XX,XXX site. AAV capacity preclinical Cleveland commercial at the to programs of other construction ABO-XXX, support a

Let's our or brief update IIIB. move on study to third a Sanfilippo the Type program, clinical B on B ABO-XXX MPS in Transpher

mentioned the efficacy Germany on quarter study of and in second closed ABO-XXX. the under we in program call, for patients study use enrollment have we are and safety in the the treated compassionate and As first following Transpher B

encouraging. assess of As We reported XXXX. earlier, to forward in efficacy look ABO-XXX second X-year neurocognitive safety the look of data and data to seeing half biomarker

our I will programs. now briefly turn to preclinical

models our address rare medical U.S. we patients. ophthalmic administration efficacy previously intend X,XXX with routes investigating nonhuman novel of vectors programs and in in produced from estimated optimal significant capsids preclinical to larger and areas X X undisclosed for made the to with programs, have the we different on measure generating our focused prepared currently capsid setting. determine are the mouse diseases primates conditions ranging genetic with data We unmet in from of need, appropriate we to in to indications, assays shared are Subsequently, preclinical capsids. AAV While novel prevalence develop constructs, XX,XXX clinical progress

expression gene We're We vitro have are in, mouse already animals. the constructs dosing in expanding our studies. encouraged and with levels colonies model started by

meetings We animal XXXX. data late by and in the expect FDA pre-IND with proof-of-concept mid-XXXX

REGENXBIO, comment importantly, certain to disclosed next Abeona resolves review arbitration points. over formal downside results, settlement want made have I inflection which to request that to it period scenarios and, previously briefly our a Before make Ed it award. to could our our by dispute payments for financial settlement X-year challenging our on provides raises with over I The an

With submitting in the now patients can. and the studies to ABO-XXX Ed. arbitration filings uncertainty BLA to call registration-enabling quickly the to behind are over both as that, focused With us, on completing for I'll and these turn therapies EB-XXX we need deliver we as

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

XX, as million the operating sheet, we balance of financial Starting used equivalents Net XXXX. on short-term in cash million for quarter cash, with our September investments $XX.X was $XX and the XXXX. reasons had of cash of third activities

As we in of and XXXX with to million the of later $XX to fourth part make than November settlement million payments in the November agreed of $X have XXXX. XXXX, $X REGENXBIO million REGENXBIO, quarter no

to legal the and this dispute. to risks mentioned, Vish As related deployment eliminate settlement allows ongoing us expenses, resources of

financial including additional lead we cash, reduce our existing short-term flexibility resources, investments, required, expenses, to equivalents financial we least existing at key ability and if through cash next to facility nondilutive months. balance programs milestones. continuing that are resources to strengthen our drive and have sheet funding. access the we launch are our Based for With AAV mentioned, Vish our on the To our operations believe and financial the various assets readiness we toward operating options, BLA fund XX to of resources that and commercial and filings, pipeline sufficient further prepare exploring partnering strategic build-out

Turning which in we activities $X third and third million spent the quarter the spent of million, quarter with to consistent of XXXX. development XXXX, in is the $X research

of quarter on administrative the million in million to general compared of spend spent $X.X was the third XXXX quarter third Our and XXXX. in $X.X activities

on expenses from results increase expenses. in general stock-based professional primarily compensation administrative and as expenses clinical of cost professional directly decreased office activities include and rent and of working and personnel and by insurance well preclinical salary as and costs. increased quarter third the partially administrative fees, The fees, in the offset General not XXXX related

in agreement on quarter loan a as period third with Paycheck of result compared X for XXXX. PPP, of the in to as forgiveness income Gain was Business as in third accounting Protection of quarter of million July Small PPP with the or SBA settlement from or Administration’s the same the in the of in licensor the X Program, XXXX our The resulted REGENXBIO. million receiving compared same and XXXX loan of the XXXX XXXX settlement $X.X [indiscernible] $X.X to period was forgiveness

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

Certainly. from Maury coming with question [Operator is Instructions] Jefferies. Your first Raycroft

that this release at maybe talk came this press conference, First, going morning to if the could what ABO-XXX to you out I the more wanted conference. about is big the ICIEM the bit and for presenting how little be guess how on I factors ask this into -- XXX. plan on and you're just picture a

one. Maury, this and we Mullen. presenting part that we see data ICIEM, Absolutely. between neurocognitive take question. the are These the used in the really The will for X are that data assessment. of Thank different scoring correlation that important you, I the are is scales at Bayley

Bayley but the study the What children the of we've based for results Transpher are wonderful the well. all developmental collecting on and equivalent seen we shared scores, X-dose past, terms in we've the been in from have that first the data age as Mullen A

So how the a for primary now question Bayley, neurocognitive end switch is point when the which arises been to the as what's you over common is with look FDA will our agreed the study. assessment that

Bayley expect follow-ups in scores instead have we almost approximately implies those ongoing Bayley our on I Mullen that similar correlation question, Mullen results to you shows shows any correlated clarify should Maury. So that the Happy current study. ICIEM XX% that if that Transpher of correlation. that. therefore, other the are you is what answers A this presenting between hope at will And data at and that be scores it put you if

answer. the helps that Yes,

seems to points I the for into be for the go end update likely so guess, And is going right that would like an this it program one. and align important this sure aligned are make -- filing help, that

Yes. Yes.

Got it.

for And also Okay. I EB-XXX a to then on question ask wanted RDEB.

were med you this UMass Coast, you up other on -- noted mentioned the site You East school. that opening

into Just recruiting is study? if can more going provide enrollment And perspective there. wanted how see to you the for any

Sure. for Thanks Maury. that question,

shared, we have prescreening are we As now, patients. UMass and the activated previously actively site they

not seeing prohibited We're more point would to X-hour So the travel from expect for many journey, patients who because patients we at it that's take be and time. Coast there a interest definitely patients something East can that's any participating. from would to from site the treated given in Stanford

would the identified we we we we that importantly, patient that in to have like patients fact last of patients work a Most fully that we EB-XXX to and Stanford the nothing up And the the getting transferring transfer. to speed all UMass, the final that in tech wanted that shared complete to accrual already of stress this, product done need and the lot VITAL have the of from that Because protocols UMass we missed need, in for a treated lot at has is intricacies, sure make treating patient procedures. knowledge the is so way with we study.

and is an EB-XXX, competent they actively has what time, that's that patients. taken apply very but are UMass So we screening that can equally site are confident this

coming is Selvaraju Ram next Wainwright. with Your question H.C. from

Ram. Mas is ] This [ on for

So the on for significant study the the Phase enrollment there firstly, enrollment of QX of guidance EB-XXX. you end year for changed a to regarding XXXX? VITAL III from Is completion just reason this

The study or the and timing, for it no, the has interest availability in it's question, the short Thanks is, do not to patients' change answer patient participating. significant for a in nothing Ram.

are We have patients interested. design. more a and who lot experimental matter a of logistics of It's

is we would in EB-XXX. think, have chronic our absolutely symmetry to setting, large, body. wound wounds have randomize study, in intrapatient for of the you treatment As to But any commercial appropriate in

upper wound a the that a And some the unique you to that's upper are right artificial constraints arm, So wound in want wound left controlled if trial. which you most a puts the is who on factor which appropriate have in patients. arm, the is clinical one

that the see not in setting. will commercial You

one. that's So

annual is patients of at matter And this happens identified. half a the and do an of matter that shutdown in is second we that something last routine we have have December. -- of every year. cGMP it's It's a manufacture, also that and time, the we we cannot And scheduling,

If next biopsy to goes missed -- of the interim an slot you by date November, end stock available mid-January.

the to we're confident we're is very primary X in going to X. that be So quarter that reason over XXXX. that accrued spilled quarter And why

for I/II regarding And AV-XXX MPS assessment? this the appreciate hindrance for I for been the to then pandemic Okay. assessments study clarification. ongoing IIIB, neurological any there current Phase has due the the

per is There to MPS due for IIIB the hindrance any program. the Thanks se, different specific assessments is which for Ram. IIIA from MPS question, no neurocognitive the pandemic

the the have half X follow-up As the time what will you meaningful follow-up IIIB XXXX, we've dosed already, X where would -- for as X-year previously shared, neurocognitive -- to X least MPS for be meaningful leads requires the we have program, that we that's And that at timing have will that second patients of us assessment period. based or on of in patients. been a when

of So time MPS really meaningful can assessments make patients. that's the we IIIB the

is light you of you think relatively manufacturing AAV any advantage this? your from some MPS And therapies? or therapy space it in to your III AAV-based Okay. of to gene in facility if What do facility, The your you crowded. kind differentiates And your plans anticipate leverage competitive then any monetize finally, manufacturing shifting just arising pipeline. clear, have And way? do

least We crowded. for is MPS The that Thanks have on and one that standard the works IIIB is space to correct disease. nothing IIIA is X currently years not Ram. will here. programs this X advanced. the Yes. MPS of in away. I development that point you question, have at anything no all. still There in treatment And And is to most no our are -- care. There's at

data, preclinical to central not with only anything have clinical data seen preliminary biomarker and system. do nervous or ecto-systemic clinical We

that is believe unique there. we this So

We are your to for as efficacy continue necessary which the A, patients And protocol for these -- is why manufacturing registrational for analysis question, to to do -- get we're latest We for for in lined to be made here. gotten an a which don't strategic part for done are -- amendments, wait second there why provisions with be a that even which approved, want mercy our purposes. be the even products we've we've non-CDMO-dependent company, had at a treated we are of want though to and of patients preparedness to is CDMO. be we -- up Transpher making the our therapies, in accrual choice

has there, out current MPS So we we've single-source, time, and also time our need. And will don't we've single-product to facility, already dose manufacture become it to waiting, which with to new want IIIA be longer construct and means to to -- any is do cannot patients able we this launches to work product commercially, on lose our ABO-XXX patients want facility, facility. any be these building which why in when why are EB-XXX started facility goes we started already is AAV a which in

SVB Next Foroohar question Mani is coming from Leerink. with

guess questions couple a I around from product the exactly AAV you're sheet. manufacturing. the as sort you balance talking give on of Obviously, around and a will managing and how investments EB you some you Can about manufacturing you're versus to transition single-sourced catalysts program be you about us the hit strategy approximate alternatives your strategic thinking in the of mentioned funnel being the therapy you gene some to make that cash the of program? terms have of key where some

should partner-able or asset? And AAV we view the AAV of think of to assets we possible a even your think out programs the operationally question, assets, entire secondarily, Like for the single Is some it second different license quantum and should in you partnering how as these potentially out-licensing programs? then recipients? about these about as platform

Yes.

be first to addressing you take build-out milestones. will we'll and for relates that. coming balance and up cash question next the that Mani, your question, to the first, our Ed Ed? thank So

know, end Yes, September. sheet Sure, the mean I as the million sure. yes. of $XX Yes. balance there's you at on

forward, magnitude But inflection get assuming in just I is key to would we XXXX, other think the still there's key we MPS That have the as have for us. we IIIA data cash readout. flexibility, that, enough point I'm some to a about cash. the key mentioned remarks, resources early inflection the access and Phase financial VITAL hopefully, year, line have effect top as looking I study think be enough confident also milestone even for is there before 'XX. III So we my prepared next we which of late points hope, to

You've got BLA filings, BLA approval.

think a really inflection healthy I So forward. of points pipeline there's we as look

the obviously most III Phase So this to are getting QX EB one we in XXXX. on VITAL readout of myopic that is

So that's that's how I question. would answer helpful. Hopefully, that

Thank Ed. you,

whole, portfolio second different: geographies. could of different types as quanta part partners, On of premature that's the or there to to will the see, a that the all But I spectrum late-stage interested out-licensing, on is in Depending positions assets Mani, definitive ability those you all different now. different of our little to in arrangements. out-licensing. the their certain where a we've for right which And potential commercialize have and can shades a all say in open we're whole of a some platform interested some a strategic they seen be give -- and be is It's will and your that for goals answer. as question, potential partners of be

platform multiple as well. our therapeutic applies But to areas

space. the are in programs As our you know, ocular preclinical

and neurological, of could IIIA in that are there in are those the are the And specifically So MPS our be assets. players interested IIIB in are of more ocular players marketplace, there that and kind specific interested neuropsychiatric types programs. --

as So ensue. develop quanta packets in we position a how conversations and are to these or

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

and patients And is first the ahead beyond think end data importance could on you then gene Phase Wondering I on focus given from FDA's the The of of the potentially across any early for EB-XXX. point top stance longer-term and therapies data include and to trial trial, of this line course, your the filing have enrolled these durability since if speak when year durability? the of one and both effects could cell the on readout I/II then, pandemic. next this you findings X-month BLA some were

Kristen, you, for the Thank question.

The that X-month a it's the time that durability question we very patients. to First the important you point. raised, benefit off, a requirement regulatory offer long-term is and

package and So right? is treatment, level what healing X and of that see we you the regulatory going is the to months pain see, from be will in what wound reduction a

our However, far, Phase more hurdle. the regulatory long-term so follow-up tell I/II the lot that value us data that we proposition have from study, than a there's presented

We is greater the years And the wounds showing patient way. up the had treated that which X than wounds to a reduction sustaining data. significant of that seen closure, and X of XX% years, showing XX% of have XX% at also pain very

is build patients. going and help with to provide proposition we our pricing this the and the So to be commensurate value assets appropriately value out completely us that

publish question longer-term to continues. also continue some on X have And your data that as about have will whether out patients be Phase to will study that will report we there years the follow-up similar, and III X,

What both Phase the years. period to recognize are is the the is and what followed for patients III, XX I/II of being Phase a important

is of showing durability going this continuous time, over update our a data to therapies. So be the

the product value we're than drug, to into more that point full a sense the of to end the get you hope that just the I market. proposition good looking gives So at regulatory the

I question potential I a appreciate the then that. partnerships. Okay. of regarding had And and strategic exploration

focus are So some your little bit and indicated of pipeline late-stage a that you the that pipeline, rare disease understand disclosed, is that not indications while ophthalmology have larger. these

or evaluating if wondering company if thinking -- be So things the basis. is geography should on a we

certain So more perhaps are diseases these there impacted? are where regions

the earlier of the areas of limitations at address that currently Could are to for AIM library. also gene you some pipeline, in the capsids perhaps some these how somebody that attractive could then observed be And could some therapies? including discuss capsid looking AAV-based separately, evaluate,

Absolutely.

to parts Kristen. the X least at there's question, So

of in therapies not are question every of -- to have have variations some these population as First, currently basis, incidence the We different MPS geography. partnerships goal we mature don't, the we different EB, the let that in strategic levels any geographies. We these as do more your in me and diseases per a is commercialized geographic IIIA our diseases about development. prevalence about well for being very on of talk

happen the These all different world. in of parts that are genetic disorders

are for partners geographies, are focus are that have of more data launches, experience initial is interest potential our to know expansions there in this those need. and more strategic course, emerging I there's U.S. Europe those at we kind of a of are markets. we our Pacific completely But beyond mean where -- and Our focused. be goals expand in is And including there Asia types other there where looking is that everywhere do to -- very geographic could useful. And that

the prevalence on based certain there about short this is It's much but a way. just how based in not on answer yes, we're bit it's going is of geographies. So sequential little

the that beauty could part the AIM the and how brought library, AIM library of is second is Part you differentiate. our that capsid of up tropism. capsid The

have that's of types tropism be helpful what types different challenges. of tissues. that's something the we some for So knowledge current preferential very have a going addressing to of in And of capsids

proprietary circumventing tropism, to to optimizing hindrance of Because with know, value we that that. as number have the effect And one. AAV-based tissue this partly part our capsids, you're of a is engineering drug. definitely our come By therapies is therapeutic that capsids immunological big can you the responses proposition

limitations also AAV-based of how beyond at types therapies. is in other capsids, we looking to circumvent the just second The AIM are

data and can where you For ARVO, you techniques example, put capsid. of methodologies other knowledge, beyond here to we're recombination-based. capsid our for X the where hope have flavor that to likely the genes and have genes, into new kilobase I where differentiated gene on ABCAX, be sites are you we size example, advance more overcoming -- just package, developing as you something therapy we're And And delivered the can Kristen. than AAV engineering, the is some the could of this such. recombination-based is as an larger you At that cannot patients. limitations where some to that presented on of we gives focusing that,

no floor queue There I'd Vish remarks. closing back the over at be to time. appears this to to questions in turn further for like any

very disciplined have about to you can deliver value road and map lead execution. conviction the Abeona's I our Thank much. We're transformative that assets patients. through focused making full on progress

stronger some Thank stakeholders I important especially shareholders talk Our recent towards our to thank you want readiness. geared additions who team current BLA on the call, to with to listened leadership and call. and have quarter is than you. our we'll ever, fourth this

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

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