Abeona Therapeutics (ABEO) 17 May 222022 Q1 Earnings call transcript

Good morning ladies and gentlemen, thank you for standing by. Welcome to the Abeona Q1 2022 earnings call. At this time, all participants are in a listen-only mode. After management’s prepared remarks, there will be a question and answer session. I would now like to turn the call over to your host, Greg Gin, VP of Investor Relations and Corporate Communications. ahead. go Please

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

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

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

those documents website to that, the the company’s entitled, and will are Risk to from those Forward-looking XX-K Form filed the identified results on results available over Commission. periodic to of call section With our forward-looking the to reports on report Exchange differ Safe statements materially federal These provisions change, may made and the but factors expectations differ not implied or Seshadri. actual Harbor other to the company statements. Vish? annual Securities by pursuant could at and Factors with turn www.abeonatherapeutics.com. Vish are are our on Various in and based under that current are I forward-looking actual subject limited securities the in are statements expressed the now laws. include These cause CEO,

we believe quickly in second March release a morning, plan and a reported cash divest our for rare for extend exclusive on press to license for In to joint where we only Ultragenyx to receive we up quarter diseases, its will $XX Abeona few morning. agreement commercialization in all with commercial including net with responsibilities and moved will our have a investments XX% ABO-XXX return bring up to and an sales been announced the this Ultragenyx We treatment. Abeona MPS, projected approval. portfolio genetic following the XXXX. programs. expertise assume of ideal Greg. payments and Since of milestone Thank to a MPS spoke new joining royalties thank partner everybody eligible potentially then, into we in late preserve runway in Ultragenyx since capital us issued weeks which is Good tiered further you deep this first that waiting we development ABO-XXX been who patients regulatory strategic re-prioritize you long too an morning and million when to have short It’s last to be

build-out discontinued We IIIB, earnings as previous fund reduce additional the should of in development next significantly have addition Offloading programs of for to the had operations ceasing MPS facilities and announced over the AAB cash our call. to burn MPS Abeona XX we months. ABO-XXX in allow the

VIITAL, of license in on filing near accrual which We focused expect quarter III milestone. more most of have significant resources readout, of application. line a late Phase our biologics the study support VIITAL top could results clinical completed we Having narrowly EB-XXX’s is third XXXX, study term now pivotal which finite our the

randomized chronic in EB-XXX term In wounds the in Phase pain that be this we of focus surface and in These by most pain more EB. EB-XXX. Society oral We large results remain spontaneously, patients Annual long wound VIITAL baseline. greatest within each RDEB study, small I/II XX that is and - as Later problematic healing our reduction Meeting. XX of instantaneous study. An six at results the increased square therefore remain data large non-randomized presentation up XX compelling was wounds Dermatology I/II Phase can for follow-up of from Investigative will similar dystrophic were study, inflicting patient. majority body open wounds an anticipate chronic Remember, featured in In at XX that the up greater we treated wounds Phase are wounds rarely six treated than clinical on the years treated from RDEB cases, centimeters area and, causing in additional recessive than to of for many term supported the SID chronic demonstrates after study and Our week, years EB-XXX wounds also and in wounds of large themselves long patients. chronic intense recurring untreated the reported, the with months same on for the in burden years. large VIITAL, additional treatment as patients. and the wounds open wound from eight control Unlike wounds the to paired Phase during severe with measured I/II an close III XX study previously treated VIITAL close

was per Regarding surface with characteristics, square, square. body mean being wound EB-XXX treated baseline XXX centimeters the of patients centimeters XXX wounds randomized range the area XX to

to product the wounds, surface baseline wound mean the XXX XXX underscore centimeters potentially proposition of the RDEB. include treated was non-randomized in study value area centimeters square, EB-XXX range body investigational the the in being characteristics square. our you per If patient unique XXX These

are await we opportunity means EB-XXX potential at exploring review which priority from have approval. we designation, for voucher of remember, commercialization Also VIITAL, we results rare a for a a aggressively As pediatric partnerships has EB-XXX.

administration to May, capsid results The administration, transduction the AAV does non-human briefly eye turning into for of eye novel retinal in macular area the AIM ARVO data create programs, by eye Now a AAVXXX’s more directly to vitreous AAVXXX, not our ability the following capsid in which early library. the reported from at administration the showed of sub-retinal produce robust meeting primate pre-clinical our unlike detachment. XXXX we a annual para-retinal

and macular than route nerve sub-retinal high optic potentially with and to transduction safer levels achieve less administration primates non-human injection. AAVXXX’s ability We’re excited in by invasive a

AAV our investigating are undisclosed from U.S. ophthalmic We patients. to conditions in estimated four with novel XX,XXX prevalence ranging X,XXX capsid

that proof results. concept meeting beginning turn Joe data support could FDA animal the over mid-XXXX in pre-IND Looking Joe? ahead, second the to in now review the a I’ll of financial call expect XXXX. with the to we of half potentially

Thank ended on portfolio where much, results strategic Starting on revenues you resources three AB-XXX deferred operating on had for million financial Vish. additional months for I current AB-XXX runway very $XX.X with of March of $X.X quarter XXXX. in development used of to programs, of consisted available our our to details investments, XX-Q into would remind ended and and can programs. of estimated to second first XX, on the the quarter which the now EB-XXX the recognition million The and XXXX our cash XX, XXXX. the like Form was of is that takes zero were the get the first revenue compared of focus XXXX. our first AB-XXX re-prioritization million mainly we financial investments AB-XXX the the the resources of Based cash, related short the XXXX website, for and the months XX, on activities March March of of grants term revenues $XX.X is quarter which quarter cash other in everyone our three the for restricted as Net balance includes us cash License disposition in XXXX. and sheet, of cash equivalents, XXXX. you

months the we to to three Turning spent the $X.X activities, for development in compared research XX, XXXX months million XXXX. million XX, three ended and $XX.X ended March March

to our NASDAQ position $XX compared Net resources cash per in to I’ll briefly Our development a AB-XXX or per compared from EB-XXX impact of initiatives of first on pre-clinical charges of impairment company’s no and eye touch or have activities operating and gene common activities. first $X.X to share as The focus share charges QX now on the programs. first our spend same therapy million common the non-cash on XXXX cash net AB-XXX million loss recently of includes regain its loss programs with $X.XX of The flow was disposition per of the million this we period have million share. the If on second is from the a quarter and requirement. with NASDAQ general million would XXXX $X.XX closing loss in meet until loss a as period requirements. $X.X compliance $XX.X resulting compliance from to for XXX-day requested extension administrative net the of impairment Abeona $X.XX regain granted, listing a requirement We for minimum quarter to XXXX. the of November in was in bid of XXXX or loss $X.X quarter XXXX.

of to Abeona’s With meeting. NASDAQ we back outstanding month, approval the the X, a the redeemable toward a the XXXX of entitled this called call common stockholders participate reverse stock. to We expected issued company’s the last year. enact we of Releases we May online stock XXXX of vote. proxy reverse special by stock common believe record compliance, which holders to be session. I’ll meeting, special is attend to in view milestones of stock $XX of Operator of As are statement the preferred press Press look the part our offering the maintain to able of release stock. stock available common date for placement implementing million included to a listing our quarter link our permits and the the key to that week, for The XX, a as have initiatives completed of effect strategy the on for Q&A results turn will our first ahead shares regain a later split this our over is to split of the with the these on earlier together preferred that, special a website. proposal The June page private Also convertible at vote stockholders meeting proposal Stockholders as we

from [Operator Maury question, Your coming your question pose Raycroft live. instructions] your is is at Jefferies. line first Please

and the thanks details updates study. for to and the for fair good argument I you non-randomized also that BLA. my today, treated, to about filing talk RDEB III and for those were the and total from the guess how RDEB the also the into is going around wounds those your taking say that’s the and questions, Hi, how worked XX Congrats Phase particularly morning. large it treatment more wounds and thank data wounds, for BLA you the talk on about fit can

Thank you Maury.

questions. a to talking the for always, you. pleasure As Thanks

treatment. will Your non-randomized first points. part question our control the for and doubt, mostly was sheet just wounds, end by the and have to The of is we efficacy so set so wounds, that’s the non-randomized are of avoidance they treating the reason pretty be of data created not the much inability benefits wound, non-randomized an find primary for goal about XX appropriate patient the XX

We healing will the those set. the something about I end report first, for points., randomized wound obviously mentioned analysis that primary such XX the and pain results that’s those trial. The wounds and of in terms are wounds data so scoring

was your filing. BLA how the to about Now, the second treatment relates question

XX As September we six-month treating into data announced VIITAL, who as a it’s on patient last March through the follow-up. quarter know, point, went March we that is first our you completed and from so

so team that’s those diligently results. of the end fairly we to not so have quarter two, confident there back data end Our database the why real is lock we after clinical in results, lag cleaning time order a should is report feel

filing study the way, careful to the the BLA are preparations cost with goal get BLA scenario earlier we that resources communicated the today. if intensive it certainly case going out we to our Regarding be two in to gating BLA clinical one, think, capital XXXX. results but quarter we a stage quarter - efficient we will the so we’re quarter had, that, the a study two of filing, look after finite the I work first have do at reads base so in a are In is we very of

a Maury. partnership there then able scenario accelerate if be we of missed some we may that happens non-dilutive reiterate. If activities the a be mainly itself, able I BLA these readout Now do where that trigger early prior on. questions, of to for may hope if please we preparation commercialization, I is have a I and something, upside an partnership your from study such funding to scenario, to answered said, source

what where finding perspective, really and partnership, if with talk the and guess That you’re with you’re little of helpful for can a more was at about a you a for partner. I the looking bit process partner

who we are as related we Yes, a readiness. diligence, exploring I will that mainly discussions are to and partner and for aggressively partnerships. take launch on mentioned, are activities are There looking some all

of we a upfront as a result technology, are of our a recognition a and would You Phase positive with partnership when back transfer study, can end. royalties that sitting the and supplier our for III obviously role be potentially expectation or on consider an payment being

queue. it, okay. in taking questions. the for Thanks Got hop back my I’ll

Maury. Thank you

Your Fitzgerald. question, next Cantor is Please with question coming pose Kristen is your live. from Kluska line your

say other delivery? capsid forward is para-retinal used for undisclosed Good for ARVO our for on taking how have morning, question. just about your vector in for Kristen. indications? AIM about plan poster. AIM this para-retinal can is - about Rick here one to Thank Could little bit a the the talk you is you para-retinal go you ophthalmic Thanks. for there delivery, Is anything it’s We platform the you delivery the with optimized and

our I’m this of Thank you question. the Brian to to Research request take for Kevany, Head going one.

tool identified enter injection. intending our that where retina for but potentially indications to NIH was potential the for as is of para-retinal this top the ago programs There for intravitreal originally the and for invasive the its are try intravitreal candidate group Dr. an published two use capsids a demonstrated Sieving’s of safer the representing those AAV para-retinal are Yes, retinal injection we being years eye the on cause enter ability intravitreal is retina a and the virus Paul does via retina The from to a method was leverage vitreous capsid to year at injection. administration to last after AAV-XXX administration for a after beyond Certainly. or This paper ability modified layered not administration. detachment, there a where indications. less from several undisclosed so-called thus

Thank you.

be in now turn in to like back at the appear Maury queue to queue. Raycroft time. popped questions I floor this - oh, There no has the would

is live, Your question. your line pose please

for Thanks taking the follow-up.

about if tech any at transfer a a question manufacturing Just is the AAVX Ultragenyx then Wondering upfront, wanted deal going that too. going work? for is be scale, to ask and there of that is commercial how or to to

intensive, twofold. is a put you, your appreciate payment, clinical is for as the reduce there disclosed. from development primary goal, that The any our agreement is while terms CMC goal secured value are thank question, we upfront our as as the answer was Maury, and so That’s no, you capital question. responsibility wanted is for answer, to the Abeona first One operating take still that to for can the program retaining well I our that goal over back pretty short to context which but that have development of partner end. Yes, want expenses, is our for in were our all further through first

early patients the able possible. the to as the deal it towards community and to of is some that the is behind patients We make MPS. goal most timing our that gives as terms. bring context the develop a to you partner product and hope this Secondly, stakeholders, and wanted in aggressively sure I

last IIIB, at is Yes, Then up just MPS out-licensing some maybe as one that that it well? that’s you possible could helpful. point on program end

had to in very because so to we not that, the early resources for items the comment our did It’s lot on that. a only premature have study and follow-up of to we portfolio, is have Maury, the very de-prioritize

to the for looks NCH, a IIIB. promising have so that prospects that date, if hopefully there license MPS We development rights for future later program at data turned for is over the asset

taking for questions. Got it, okay. Thanks my

Thank you Maury.

be remarks. over Seshadri the time. no floor back to for questions like to would closing this further Vish in There to any I appear turn at queue now

you. Thank

who and have dedication the make other I you strategic our we’ll impact projected to quarterly implemented extend our to quarter changes our milestone. expenses also cash potentially into shareholders on value-creating have window, reduce for our of stakeholders call. XXXX. to this to runway team to Within talk operating for driving want next that second a continued efforts achieve and call we the in today, an to thank to patients. We thank their I listened and want plan

and Ladies you. today’s call. conclude this Thank gentlemen, does conference

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