ABEO - Earnings call transcript Q1 2024 | Abeona Therapeutics Inc.

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Operator

Good day, and welcome to the Abeona Therapeutics First Quarter 2024 Conference Call. [Operator Instructions] I would now like to turn the call over to your host, Greg Gin, Vice President of Investor Relations and Corporate Communications at Abeona. Please go ahead.

Gregory Gin

Thank you, Kelly. Good morning, and thank you for joining us on our first quarter 2024 conference call.

www.abeonatherapeutics.com. I will the the is today's first our website we this release available call call, note corporate announcing to and morning quarter at may this contain refer made forward-looking press to issued on that projections During remarks like statements. during would results which

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

today Officer; and call Chief the Business with Development; Officer. Vish Joe are Vasanthavada, Dr. and of Officer Madhav On Commercial Financial Dr. Chief Head remarks Executive Vazzano, prepared Seshadri, Chief

Q&A to as Vish? Joining turn the I call now Brian Vish Officer. And with session Chief off. for well that, will Dr. will the to over Kevany, lead us us our Seshadri be Technical

Vishwas Seshadri

investors The and proceeds. a addressing Thank for commercial offering May, care demonstrate we In joining blue-chip existing their progress milestones the be Participants investors investors. are most you, beyond health our institutional that respected In to our early launch, support. Greg. the grateful to Abeona the We Abeona appreciate funding morning. and with This team some $XX included to who through raised in capitalized. offering order was been to well we're development underwritten gross well regulatory of million and significant an new everybody institutional leadership investors in recently anticipated continue call completed focused this needs. our program must has pz-cel on

we runway with cash great well towards for as We has regulatory now validates stay resubmission Importantly, commercial anticipated extended significant to the and financing the into resolutely to it our milestones bringing pz-cel. RDEB beyond but is the XXXX, not BLA address only believe pz-cel, CRL on deficiencies further which of potential as in making pz-cel and focused possible. the patients CMC noted soon the FDA working launch

to did of data deficiencies made noted the progress CRL needed a CMC late April, received towards In a brief not the deficiencies identify BLA update clinical approved. clinical for based the requests new clinical the in from the information safety the and clinical pz-cel information CRL. the call, and be the As any CRL the to CMC the testing have of pz-cel. Since efficacy trials addressing satisfy our data in to recap validation manufacturing pertains for of FDA need related can call update the to regulatory did The in on additional request not requirements CMC methods. any or approval FDA we before the general, we BLA release support certain already

We the Matrix ask on RCR or assay RCR outcome the the March. for the will interference completed study now based validate competent discussion retrovirus agency's or in have satisfy believe We assay. to our validation replication study late with this of the FDA

are testing We the the in container for RVV integrity closure have and completed reports also place. the validation

therapy of agency's through approach, gain in of informal interpretation meetings on with continue is where the on context alignment especially our to existing the to required. the We FDA interact guidance our topics

anticipate to We of noted these early in the the enable XXXX. the late and streams coming would resubmission BLA work all of completing Looking and remaining resubmission ahead to half completing completion the in to in to frame the of months, we're second the address anticipate all BLA. QX working that weeks on deliverables QX CRM time deficiencies

goal request the CMC in of FDA to A plan The alignment our outstanding, early and QX. address meeting with a the agency do the substantive data would meeting this with to items. be critical of sufficiency the We on Type to gain

to always, our As now to an the transparent be with the Officer, completion. I'll we activities. over Madhav the update readiness will our turn continue Chief Commercial after meeting of outcomes call to provide on Vasanthavada commercialization

Madhav Vasanthavada

towards including a story or with Vish. And space We the and using [ about are did the continues. Because prominent modalities payers patient us. In pz-cel DEB the strong our is pz-cel. our from our of the is in are therapy as groups as BLA, as they targeted any with and physicians and confident our our issues you, clinical Thank RDEB providers. We for value efficacy clinical EB game-changing huge continued stakeholders, of identify in speaking messaging sites, enthusiasm treatment does for for FDA that and patients. DEBRA positioning physicians whether treatment there value EBRT clinical hear need remains in physicians with not confidence complementary momentum potential patients commercial for that or well safety-related with remain the for various readiness the ], so like

and with large better aspects of wounds strategy prioritizing of upon faster to and on timeline, a pz-cel, updated of application. now patients. In goal after reduction light access pain only clinical can to be if wound is differentiated and having meaningful body our the discussions therapy, for single front focus would approval. treat load through durability BLA highly and payers access surface with it a that clinically we payer launch activities of the These RDEB the approved, which toughest trials the would areas, demonstrated addressed a years healing in with pz-cel has including make

a this physician Dallas. for as late awareness. In Annual accepted to that of follow-up of of Dermatology, presentation Society Meeting will durations has long-term years We on XX XX scientific more also exchanging wait abstract data discussing also payer for safety medical safety earliest Investigative potential forums SID various exchange, breaker years could also at safety with for of profile as ED have, pz-cel treated we follow-up of focus scientific strengthen pz-cel regard regulatory scientific community our of benefit from been available patient, through than disseminating longest would in that the data will discussions. the generating be data, pz-cel in gene approval, for new we therapy. Besides upon its clinical With approval, one week's

that currently receiving previously You date, recently or also And back EB. better pz-cel we for to IIIb an previously as are patients, patients approved patients policies we may have received currently come all allows their that treatments generating have study for where Phase help have such and recall, have patients elected RDEB treated untreated new enrolling repeat to X for is whom treated of will This wounds. post-approval. shape study access pz-cel us treating dystrophic data active

exchange Speaking nearly the spoke. engagements pre-approval of need payers can information time had impressed the and payers clinical it one-on-one of and encouraging And we we coverage pz-cel commercial is value address, dozen a with continue access the payer perspective. through discussions, with story unmet since last have from be to which

and the now EB progress. With ready discuss patient post Officer, pz-cel and the of be that treat with to our to to keep make are continuing Chief as are onboarding X advantage regulatory refine program. Vazzano, Lastly high-touch readiness of and We time Financial X call I'll building as launch that, our we'll network initial work possible strengthen to centers we to and Joe financial standpoint, soon a from services results. to taking high-volume approval, updated sure will you on over hand to our site sites want

Joseph Vazzano

XX-Q, find can XXXX million three on Thanks, is financial restricted we March Net with on our would short-term the that our had Form operating results March for of as $XX.X financial details investments I in months balance Madhav. XX, activities of like most remind used $XX.X available website. Starting you as XXXX. of million ended cash, compared our March December the XXXX. cash $XX.X the months was XX, and equivalents, as additional XX, recent in million cash three to everyone XX, for sheet, cash to XXXX, resources our ended which on

on plan estimate resources $XX million fund have the we we XXXX. assumptions, from to facility our proceeds combined into existing our including gross the with resources, credit financial and operations Based equity offering, our recent also current sufficient operating and cash with

Our from the designation account proceeds pz-cel assumptions for the of not FDA. sale FDA. do that the granted by sales commercial pediatric cash any priority disease voucher or rare runway of potential or you I'll review pz-cel revenue awarded a from by has been remind if PRV

three So upon XX, and ended million PRV. Research development for eligible believe XXXX, XX, potential months months were for its that March to are million March $X receive the we three expenses a XXXX. ended $X.X to approval, compared we

the $X XXXX. $X.X million was XXXX, general Our March months months three administrative March XX, ended to the spend activities for XX, compared ended on and for three million

These of common first of $X.XX or required Net $XX.X operator, loss to in fair million was loss loss the per important XXXX is $XX.X the million change for related in value as session. XXXX classified Net are quarter that, Q&A warrant and open the liability $X.XX was million warrants of of loss or common first the quarter XXXX a share. in first loss market included $X.X per value note period. reporting liabilities. be each remeasured loss quarter of the net to please to that fair noncash It at share. With the

Operator

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

Maurice Raycroft

to you Is can just there there or third you was clarification Is time going everything a submission second just sure change line quarter. timeline that get more versus ask in a behind BLA it any that just I more on your the for time of softening add? one maybe make perspective to to that?

Vishwas Seshadri

plan. We're very and in latter. still track much our resubmission quarter X the really a is on It's

do we've off FDA with the softening to when the so we that us XXXX during resubmission, that that our accommodate they've all the them FDA questions to sure Just asked validate like actually us CRL satisfied we've doubly, make the language plan and as those squared -- surprise. a the no to meeting it the of the because half want and before is uncertainties we triply there's in external is the be of grants second

looking So the if quarter is to quarter Hope it's we're to keep need broadly the of thinking question? versus second border to that our as conference previous rigorously there X, half, in give just announced how of wanted the work addresses material of the after we do we we last on no of -- that beginning X discussed to call complete in line as your change but that CRL. day

Maurice Raycroft

make Yes, identity that made sure And assay? that you the maybe due assay. talk to just helpful. little FDA the bit to going that's progress with BLA. for alignment alignment nature about assay more is talk on cell-based Can of to and about with there FDA that that And novel is a the the there plan the

Vishwas Seshadri

So really continuing cell-based the meetings we informal cell assay composition to product, through FDA alignment identity what's the the gain are of our looks the with agency. with and our at

We address have and required good is to fairly understanding on what the the be sensitivity done to specificity. a

that agency that seems proposals and review the be So that the those and with has we we've generate. straightforward made will indicated data this our a approach issue fairly

necessary And the based done far confident are provide specific identifying markers we are that so we've runs the that keratinocytes. and to to on feasibility able that we'll be data

keratinocyte. Just product primarily a as our is reminder,

of to So and to you able detect binding its in types deliver may and keratinocytes find that to that not can other and that of need environment. cells kind an sensitivity. we're we antibody specificity have is

So we'll the assay resubmission. identity time we're by the do the we confident place that in fairly have

Maurice Raycroft

no then with difference at you but numerical and SCC a non-treated treated SCC see last sites question SEC where be for at where in SID the sites late-breaking seems And there to given was label? this with least we maybe there SCC don't should Have prevention be demographics higher could for respect patients think about FDA the included risk to And VYJUVEK. this in you and at may in discussed whether potentially of SCC? who KOLs be how of abstract,

Vishwas Seshadri

measure preventative effort happening. that know date, early to clinically. order are to on is have lot data definitely demonstrate do encouraging. that to avoid to would of to the It's get claim We've thought in level probably the of of been What this. part we longer-term It's going to a pz-cel too our label that a our little SCCs premature indications be a to data giving but be

squamous from published where do wounds large that typically the are we see originating know what chronic literature areas However, carcinomas. is you cell

treat of high the risk agreed And from so those priority is well to And a perspective. patient types is medical community. aligned that upon wounds and by

definitely a include high require we're for future, at the label we patients those into but but can that. So still whether may data our like that, but treating it claim will TBD, risk looking prioritize additional generation it's

Operator

Kluska Fitzgerald. is from next Your with Kristen question Instructions] coming [Operator Cantor

Kristen Kluska

III that wound looking treated? the see two we the at company. are us been endpoints of important to investors investors. that wanted I from the that I data just new you how key trial wound to very understanding first, these looked importance XX% healing? relative newer at at least of relative then Great the the And guess, remind you some healing, questions we've of in a size a a lot taking that lot look also wounds Phase the getting are least of at of complete do And XX% healing, about to ask wound understand that to understanding the can you these

Vishwas Seshadri

So endpoints are we minimum the of we With forget. remind size multiple it's sheets the wounds toughest The treat that centimeters our size. graft, wound in to treated apply treated say where of that, pz-cel, dimensions as have wounds. study to run that are quite often well we right? into I/IIa. the to in like often squared, tend important as the squared some as wounds of well that hundreds those One to XX there was but And there centimeter I And that needed as area. nuances, pz-cel the multiple wound we is something two is that. the required pivotal study when that We Phase ourselves forget types to

a what wounds, cannot of aspect from themselves. recurrent So different self-heal the experts. our these type wounds that are wounds, compared They and the wounds definitely we chronicity. Chronic are is second hearing different from to they size of the is are

ever you If our our chronic healed in complete look healing at wound zero completely rate, wounds study.

type wound very you the we're tells treating that that that different is So itself here.

thing. one that's so And

statistically later. Why of we've looked that XX% complete, and will And come all wound and have I not course, healing, XX% other wound which healing healing looking to levels fact, XX%, those primary do the second VIITAL, The at three in of in healing. sizes is healing wound aspects, is endpoint that a pain for is, but reduction, a patient in shown highly pz-cel we're the significant pain we have that wound all XXX% at wound outcome XXX% we wounds with X It's when rates at even you their note life that the these significant in difference look versus the types reduction. to there's see we a control. very of of what to quality healing XX% for of important their leads and

healing that But extremely case the score small wound study, than area. showed is wound, XXX% And I healing. wound which even if XX% is would way even XX% emphasize we crusted the greater X/X of of is in of for the there VIITAL stringent patch the a

and If that you crust pick wound that. see underneath healed cannot if cannot -- on you obviously the

a completely that when not dot of of wound was you red cannot scored completely a crust therefore, if under that as the is score have even healed healed, or was there you though the doubt, rest wound. dot, the completely a wouldn't element on healed and microscopic verify a as wound, wound, when it we this the So even

the these versus difference regardless, -- the wound how pictures you the of at look healed wounds. So But treated at is when when look new speak healed of volumes are numbers nuances you the between wound -- some there. control

that or started patient score we worse which where XX-point what is on X.X%. with corroborated at pain wounds significant So high important. a pain found reduction in reported is there pain scale, And we ultimately further a the when our reduction, that of look that mean clinical was second also by that endpoint, X is outcome is is

in further how therapy Kristen. please address So ask the that of able basically I your alleviation to is this If aspect underscores question. to do hope happy to much an patients it. I that not, offer of pain of purely I'm wound these me to healing details. addressed addition

Kristen Kluska

did it. answer You

Unknown Executive

and besides remind our Vish in never to types open that right? of terms wanted average wounds in able years not hardened our you for of wounds some the in that to to were show of of closed have especially of VIITAL years, XX these have in had kinds to just had patients, duration add with wounds our what to numbers, baseline wounds these adult healed the that with and I X said So show was have are where we been the up characteristics, treatment adult to that modalities, were we these able data chronic other of with them. patients, most VIITAL been have patients

that wounds closures. and months, is we think these kind show that healed, of profound, X this I are pretty So at able knowing to haven't wound

Kristen Kluska

often to the often of understand patients come diseases what in diseases, started are know then size But identified. And try like online, to is. modeling genetic I therapies done unreleased, some rare that rare patient for pool the see increase We in actual to development. you've new things with others as

So in KOLs, as heard all doing few approval. guys of about the diligence potential are I'm space? the you market development last you've with there's changes any the commentary in if the been any curious for lot a with and years size preparing if

Vishwas Seshadri

Madhav. Yes. Go ahead,

Madhav Vasanthavada

we test definitely advocacy as on to from there testing as hope later XX% happening the diagnosis heard and and an a, misclassified. on more we as is testing there Yes. is rare an the increase genetic more be were you the to sooner think been kind genetic to definitely certainly are other with able of I on. We and -- market; that just is come with in that one early physicians in I -- and right misclassification that the hearing track space, But said, patients biopsies, EB misclassification collecting therapies EB diseases what with similar has DEBRA of as that, of reports that from accurate happens. high especially

Operator

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

Vishwas Seshadri

In Thank possible. joining business joining get pz-cels today to everyone, you, With you bringing us with you you. will today. for for for and all as Thank firmly remain Thank talk update. you, there. we to to we committed as thank I believe Kelly, today's we'll soon. closing, us RDEB that, patients again quickly

Operator

call. conclude you, conference everyone. today's Thank This does

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

Abeona Therapeutics (ABEO) 15 May 242024 Q1 Earnings call transcript