Earnings call transcript Q4 2022

Due to licensing restrictions, you must log in to view earnings call transcripts.

Operator

Hello, and thank you for standing by. Welcome to the Q4 and Full Year 2022 REGENXBIO Earnings Call. At this time, all participants are in a listen-only mode. After the prepared remarks, there will be a question-and-answer session. Instructions will be given at that time.

call recorded. this reminder, a is being As

It Officer, Patrick my pleasure Chief Legal to introduce, Christmas. is now

You may begin.

Patrick Christmas

Good afternoon, results afternoon, XX, our REGENXBIO quarter and website Earlier full XXXX. released today. thank and is financial you The for at press and available ended www.regenxbio.com. operating year for release fourth joining the December us on this

subject uncertainties development our may, and other addition intend, to in may conference will words plans. forward-looking results from Today's These outlook to call and plan, by should, include can those financial forward-looking such to statements actual similar cause forecasted will, of are and as believe, expect, that be statements product risks words regarding differ anticipate, regulatory identified and meaning.

the of XXXX, XX-K are with for December not certain Exchange Risk the and are annual of are future XX-Q, on SEC's such Securities report quarterly the Commission on factors website. risks These sections on REGENXBIO's Factors which Form forward-looking risks uncertainties. in file the discussion guarantees and involve the XX, Form described risk comparable REGENXBIO's on and available reports management's and and statements performance of full year analysis ended and and Any sections

forward-looking information, call account we Any XX, on of any or as this obligation may information XXXX, no call is call, make events this new and this we statements provide of otherwise. on update conference to February only on provided the date future we undertake

being call Please be advised that recorded and is webcast. today's

or may company. report to provided materially. financial operating that positions be not preliminary or is and forma pro In financial the differ does Actual results of unaudited information addition, any results project may

Ken? call to of like Ken turn now to CEO over would I the Mills, REGENXBIO.

Ken Mills

clinical our for quarter and financial Chief the provide Financial you, line our on our recap Thank fourth will overview today's At as Good goals. programs; with our on provide Chief will December open business we call, call afternoon, to then Vit Dr. the a Officer, the of begin our Medical an Pakola, recent up highlights, ended of update XXXX. Thanks Officer, corporate an update well the of Steve joining for us. questions. as an XX, for will pleased Vasista, results everyone. I'm end Patrick.

mission that improve are through of have our to REGENXBIO, for lives we therapies curative gene diseases need. potential unmet At on is the significant and developing focused therapy,

we're at regulatory I'm by and gene encouraged for accelerating of We NAV from dosed continue been leader derived final FDA to Rare gene importance leaders encouraging stronger. be of momentum therapeutics clinical with Today, particularly, patients of approvals and Month. Awareness February therapy outcomes a the recent technology Day actions decisions, signals thousands who our with from Disease gene and vision of intersection AAV low therapy. I the therapy in at With has about AAV the was recognize that been therapy the never platform. of gene the we have opportunity development, in part that the upcoming day AAV therapeutics positive trial believe and

pivotal internal advance by products programs year, execute the to and of with great make near-term continued I'm expected in clinical our course Therapeutics through into research strategy of fourth the progress proud and from AAV XxXX license internal goals, stage the five pipeline leadership throughout We've to advancing commercial this development commercial-ready and or Additionally, we as our our quarter approval field. in our pipeline efforts and continued from XXXX. REGENXBIO's manufacturing,

pivotal a ongoing has AMD process BLA commercialization of RGX-XXX of the wet in our pivotal filing atmosphere sense, made of To treatment highlights. Enrollment two and expected is trials our future across progress operational summarize manufacturing NAVXpress collaboration significant The been incorporated with AbbVie. some for program global for trials expected center are care using to RGX-XXX. pivotal delivery. the in our produced support for to We've be eye innovation XXXX. the these at trials into subretinal platform in is and REGENXBIO's

plans to the bridging is The study RGX-XXX commercialization. I scale where same AMD for of subretinal pivotal our that, encouraging presented from this month, in at for doses the delivery initial trials. and process commercial profile we pivotal a of dose provides receiving evaluated earlier RGX-XXX level data process Related patients using platform of NAVXpress Phase the manufactured two RGX-XXX by being we used using clinical validation these our future wet

data both AMD of this this using quarter positive the half The second delivery the with year a XXXX, and trials new in-office data, interim retinopathy gene diabetic are expect half of in ALTITUDE Now suprachoroidal AAVIATE complete data announced profile in ongoing, potential trials in updates first enrollment we from and last for supporting in approach cohorts wet XXXX these these therapy. of emerging we've initial recently recent RGX-XXX to the one-time cohorts and and report interim the of progressed clinical for including also XXXX. additional of the

and our population significant disease unmet that a need. key RGX-XXX trial of large DUCHENNE is announced active over Duchenne of represents with to We AFFINITY moving is piece pipeline. rare recruiting a and the Quickly year patient our XxXX earlier patients. this strategy

a therapy function differentiated potential highly the gene candidate, Duchenne has microdystrophin novel including to muscle capsid, increased improve optimized for and and to boys with Our which AAV access Duchenne. strength, features unique product program an a motor

from AFFINITY support cGMP starting DUCHENNE to data that in trial report are demonstrated our the from efficient half that we commercial-scale our purity this initial and Today, of the second commercial manufacturing increased process the using also expect We clinic to we believe announced and yield has proprietary year. that readiness. advancement trial NAVXpress we will

key clinical using NAVXpress to I manufacturing for entire development commercial Therapeutics. platform our our REGENXBIO AAV that accelerating represent the pipeline As the we're differentiators mentioned, process scale innovation across trial center support and of

we II manufactured our drug innovation manufacturing Hunter MPS treatment support XXXX using BLA trial, for to to of the pivotal And the pivotal today, center Syndrome, the in of a also future accelerated first incorporate NAVXpress XXXX to the the expected that or Our at announced half program product pathway. approval to is commercialization. expected support complete is RGX-XXX in our CAMPSIITE for trial enrollment filing

at Syndrome all a presentations which provided CLNX, had WORLDSymposium, updates We greater from severe last momentarily. where RGX-XXX of XXXX MPS we will in review Steve multiple trials certainly the week detail developing as Batten Hurler with we're positive in RGX-XXX well that I disease, as of or for and form treatments associated

and Hunter XXXX. We expect to share plans additional clinical program throughout Batten, Hurler and the progress updates on

over can he our detail I'm clinical in review With so that, to the progress. going to more call Steve, turn

Steve Pakola

RGX-XXX Ken. vascular factor, in begin the vector which collaboration Thank I'll AbbVie is endothelial wet and a on an to you, RGX-XXX, deliver to update diabetic gene VEGF. AAVX to with a retinopathy. growth being AMD with encoding inhibit or therapeutic fragment NAV uses developed antibody treat

the are are injections with anti-VEGF frequent wet cause leading vision Japan. require these the evidence care people over is in patients frequent XX, X treatments, AMD that for US, than due which injections. undertreated standard Real-world Wet of patients to wet AMD shows treatment burden Europe unsustainable to of eye. The in AMD of and affecting more patients in severely current receive loss the patients million the

patients the majority loss As vision AMD significant of a over wet result, experience time.

treatment AAO delivery the RGX-XXX for meeting from presented I/IIa Last in held wet quarter, October. study that the long-term at of of Phase follow-up the using we data AMD subretinal was annual positive

expect along submission this ASCENT our in -- ongoing trial, and BLA two We trials, planned this XXXX. to result our with ATMOSPHERE pivotal support

commercialization. AMD from using the the presented for also adherent data Ken culture I/IIa this to clinical study RGX-XXX trial, platform The process used Phase exhibited a the we by Conference data production the process delivery initial of As platform subretinal demonstrated to that the mentioned, at RGX-XXX validating of cell Phase in pharmacodynamic of bridging treatment Angiogenesis the NAVXpress wet for earlier process, RGX-XXX II support NAVXpress produced similar month. profile the the future

of high-dose The two to CRT also pivotal RGX-XXX pharmacodynamic demonstrated injection-free. meaningful has levels in designed well BCVA is being the dose II been with trials. same Patients majority the ongoing used anti-VEGF improved subjects evaluate this in two cohorts in the the Phase stable reductions to and burden and tolerated in study trial.

advance for retinopathy using of suprachoroidal AMD in-office delivery. diabetic treatment continue the and We to wet program also RGX-XXX additional two

data subjects with the in quarter, evaluating Phase announced wet AAVIATE from II new Last trial, escalation AMD. RGX-XXX study we a dose randomized

to We are being a in NAV in potentially also incidence to the administration expansion announced the sixth prevent cohort further cohort IOI. to course enrolled of level trial X short moderate this status. third regardless of observed of mild steroids Patients of explore RGX-XXX following dose a prophylactic ocular with the

ages the to and an blindness. million including lead for adults vision-threatening cause edema of neovascularization RGX-XXX to and complication lead DR that diabetic XX slowly a of blindness to is disease worldwide. the progressing treatment diabetes is macular estimated retinopathy. is leading can between and diabetic that in Moving can DR of a to XX XX patients complications, affects the of

AMD risk vision-threatening Like the of to patients developing with wet anti-VEGF has with are proven reduce therapy, DR which in treated complications.

of significantly treatment the like many treatment We RGX-XXX injections, gene this either an disease this could anti-VEGF become a to receiving due until off or believe to DR unavoidable. and overcome therapy hurdle However, burden their progression. important symptoms unsustainable frequent provide condition with patients elect any for treatment therapy forgo patients put to potentially onetime alter

II Phase with ALTITUDE, evaluating Meeting. new data trial delivery patients announced in at of we dose escalation suprachoroidal Retina controlled DR our November, In the RGX-XXX Society Annual from randomized

cohorts expanded ocular the include a RGX-XXX patients stratified steroids across levels IOI. dose announced a of potentially level and administration trial by course of to incidence We prophylactic that we short observed DRSS all receiving also with to third that prevent mild higher following

to of improved RGX-XXX leading the contraction-induced designed as in the for of or dystrophin. has functional Shifting membrane, the occurring elements In terminal muscle shown developed that muscle muscle Duchenne a damage deliver differentiated to product recruit to rare key domain a includes dystrophic to several transgene disease the resistance CT the naturally in treatment our the of CT one-time gene domain, potential for cell our therapy being is been preclinical to to studies, novel a highly microdystrophin CT models. presence proteins mice portfolio. found

and features, the Additional including codon reduced efficiency increase and optimization gene reduce immunogenicity. translational design expression, content have to potential CPG improve

tolerability well-characterized muscle-specific active as the to well is levels, AFFINITY our and expression throughout designed targeted muscle assess vector gene the promoter. and with and recruiting trial we our and In and announced Duchenne. measures functional and AAVX strength RGX-XXX protein muscle skeletal patients support microdystrophin a is will muscle I/II safety and and Phase DUCHENNE heart January, using RGX-XXX that and in NAV expression as delivery of MRI

patient dose first process platform this to NAVXpress track on our from material trial in are year. in cGMP our first of commercial-scale this We the using half

with study future patients screening recruiting also evaluate of to primary trial and trial, AAVX the the are AFFINITY We patients observational potentially antibodies AFFINITY for in the trials with of RGX-XXX. participants possible identify other in presence objective BEYOND Duchenne to an DUCHENNE the help

progress our MPS We the at also data II two programs XXXX from continue I. presented RGX-XXX for for programs to we Last week, WORLDSymposium. RGX-XXX for mucopolysaccharidosis, both positive and MPS

positive the largest GAG reduction II the for Additional Phase pivotal week trial RGX-XXX continue levels normal using X patients continue patients cohort the of in interim showed XX. CSF CAMPSIITE these data demonstrate program I/II/III dose and in to MPS approach at from to

administration at a HS closely being II and baseline of measured biomarkers MPS CSF DXSX RGX-XXX. are HS after severe of in the GAGs or or As activity sulfate with that and are reminder, component enzyme heparin IXS key of and correlated

patients CSF considered clinical pathway benefit CSF GAGs disease manifestations, the neurodevelopmental in endpoint in to be deficits. the reasonably of MPS to including the clinical GAGs buildup under likely correlates predict have II approval is as a II of accelerated surrogate with MPS that potential

neurodevelopmental years demonstrated continued and skill to improvement administration. patients up activity acquisition Additionally, after RGX-XXX in daily three

all drug-related As RGX-XXX no was to SAEs. across reported well January of tolerated XXXX, be XX, with cohorts,

World, we I/II data of MPS positive from I. additional shared treatment the for interim trial also Phase severe RGX-XXX the of At

neurodevelopmental measured Patients As activity for CNS with by skill drug-related testing. appropriate IDUA events. continued acquisition reported and developmentally of and no to biomarker instruments January XX, XXXX, validated demonstrated was be the as serious tolerated, positive adverse in well neurodevelopmental RGX-XXX age encouraging

these the programs, the programs two are Following treat two to manifestation the to treat treat help and manifestations. manifestations we RGX-XXX of RGX-XXX ocular to developing neurodegenerative also CLNX.

under trial initiate in patient first I/II of of half As we patient Phase clinical investigator-initiated dosed last in to a expect RGX-XXX RGX-XXX of And we late year, year. in Brazil. this UK announced, recently the single the was a the study

working conclude, we and of continue To have programs significant data toward we all made as pipeline, of across trial with in our updates goal progress our XxXX. the progression

and Without Day, patients, to our acknowledge our Disease of physician goal Rare gene the opportunity caregivers, is therapy our work. and is today this not and participate investigators February delivering low would like in month, simply the mentioned, programs in possible. of progress I'd potential Ken curative As advancing partners who vision be to awareness with them, take the

that, I to Vit? Vit like to call over financial with the And guidance. would to review turn our

Vit Vasista

Steve. REGENXBIO $XXX activities of totaling the cash to The $XXX XXXX. you, and XXXX, in cash Thank by driven to on year marketable primarily XX, XXXX. cash, as fund securities equivalents and was ended decrease million December expenditures December quarter million, compared XX, operating with and capital used

Care the the activities REGENX-XXX clinical funding was while our was $XXX for and collaboration offset Beginning was expenses XXXX, attributable the of end XXXX, XX, with for year for with expenses trials of certain for R&D responsible be XXXX, the development with million partially AbbVie relief product development personnel XXXX. development million REGENXBIO majority REGENX-XXX will and associated compared RGX-XXX for The by Eye agreement, AbbVie. manufacturing costs reimbursable increase trials all December the by AbbVie our in candidates expenses. accordance In were were in lead cost responsible $XXX of ended clinical funding costs and collaboration. other through to under and primarily shared RGX-XXX ongoing to and

excludes balance on of operational XXXX, RGX-XXX cash commercial on securities or received the the collaboration. XXXX. operations fund is the marketable December payments achievement current expect plans AbbVie cash We This equivalents of impact million in of based our and be that as company's and guidance development may under of any cash, into runway the from $XXX milestones to XX, our

final back to With call that, to thoughts. Ken I provide will the turn

Ken Mills

by Thanks I'd encounter in very for the and we a like high therapy. through inspired that families, caregivers Vit. curative other the of to potential that and that to every is level patients, perform REGENXBIO reiterate lives to our at journey stakeholders update, gene improve day continues

In made expansion XXXX, RGX-XXX trial objectives geographic planning. meaningful with progress and our we enrollment

filing approval and Hurler We with We also expect treatments of diseases commercial regulatory clinical treatments remain Hunter established using diseases in-house manufacturing latter of made outlined, significant The BLA that global rare such and for advancements developing our the will we for to as which Duchenne, meet we a and committed pipeline pathway. to standards. syndrome. accelerated Batten, support

make gene resources, in values Our history, groundbreaking an and of therapies. and us potentially in science, the people to combined industry therapy leader development

have understanding of platform expanding We that amazing patients' have to Therapeutics innovative engineers to an applying the AAV and new of AAV NAV opportunities the scientists and of team lives. vectors, differentiated to dedicated the significantly and the potential impact technology exploring capabilities application generate the

on forward with updates look and important to XXXX all We our of number a of clinical-stage programs. research

vision and AAV operations sheet balance we to XxXX our into achieve mission to With to believe XXXX, and possible the a the have clear fund I in and Therapeutics. what's path definable lead continue

over call questions. turn we operator, will the With for that,

Operator

question you. first Barclays. with from Wang Gena Thank [Operator our Instructions] comes And

line is open. Your

Unidentified Analyst

hear you can me? Hello,

Ken Mills

Yes.

Unidentified Analyst

on Xiao progress. congratulations for for much question and is on Gena. Thank you [ph] the This taking so Hi. our

question DMD two have wet questions XXX suprachoroidal We AMD one on in on XOX. and

XXX, on cohort the far EXXX. new is the So dose prophy around X,

dose? So what will the you successful further would -- consider condition dose? to would will you confidence escalate type data and give what of And under consider the pivotal would this what you be

about DMD. is question second The

type of And So would XXXX? your the report expect you data to in the we what -- endpoints? second in evaluate of benchmark How to what mentioned, so And just biomarker what should any you addition update should update? in we patients? functional data half use many

Ken Mills

those let any and color, know. you if I'll want questions. a then add Steve, lot take me to thanks for them Xiao,

and trials. the prophylaxis. year, think second the will half guidance the the precisely we're for updates cohorts AVA of predict talking the ongoing enrollment of think about I ALTITUDE the about And the We're early updates to it's with I when occur. include that

the us cohorts retinopathy have six-month But that time points normal we in AMD sort to I is expect what from get can diabetic of of updates wet you our structure studies. when and think for we

interpretation be lot talking about. made benchmarking we that when obviously it's that to terms subretinal this to any the I we'll of data early how around half to profile second to And of about it comes to data Thanks decisions the pivotal. year, to of I any We because in those talk don't was question. a data due think we specific have transitioning we too forward, in have specifically. additional of bring think when able for be that to talk have

the active study On of again, the sort think, recruiting of I XXX and in we're today. side, process

data. second guidance half year giving we're for the of So

think design that certain to some the have six would at clinical of are trial think to to months. we're I time functional in the see assessments maybe outlined I nine going that tracked outcomes, three and months things imaging early we be in XX in outcomes of types more maybe toward study weighted first be of biomarker will our or points assessments, that closer things the

have terms So I what think in that's trial the we design. of

we closer as to be we'll the second more to point the into those information. where year think half of provide and I updates, in we'll able get have data time the

microdystrophin be here at interesting we've benchmarking, we'll examination We've microdystrophin. of I RGX-XXX. C-Terminal it imaging domain, in looking construct its is the talked of class techniques about biopsies for at looking this first-in-human be and are differentiation. on terms that Early certainly the differentiated the about the think available potential in of effect in scientific bringing and the and will of on, clinical expression talked I in some safety of think opportunities being microdystrophin

second expect can we to of think I that things in the compare the year. are the those half So to

Unidentified Analyst

much. very you Thank

Operator

Thank you.

Leone Raymond James. with question next from Our Dane comes

Dane Leone

coming the investors have more from recently. like popular ones been probably two sounds that in It

What sense fact, forward first path that you a one is, they right clinical will have The from be or disclosed XXX in there's a Sarepta patients treated program you discussion the have now, tonight you will that will do an when data first not, been DMD. patients the from of is have with first Obviously, strategy. ADCOM. the

I out, your clinical you. quite second and you on infringes your in your the how one view whether team's your is development So given how program? that would works then Thank guess, states? thoughts XXXX And patent their popular view IP program approach

Ken Mills

right to your Thanks, getting Dane. I always appreciate the point. questions,

it's -- to until look I terms development look, safety, at XXX. path next to to not a think think have anymore assess activity, clinical exactly with biomarker of our in what of leaning we've going in great we're be we we data, Right outcomes. steps assess got program functional early to now, respect

observing think strategies have microdystrophin programs the the market we've think development. within clinical we benefit of that and Pfizer run, others the CMC been of have who later and think some access regulatory of We things scientific class capsid playing behind these that differentiation. got I employed. also we and some from And got kind all Sarepta And that advanced addresses fit of think guidelines scientific, and have stages products a got the the access with of have think some of to we've we've We We including needs. advantages. clinical

safety as be we'll data. profile a do We reading the we patients view of biomarker to -- to half want dosed, that the start So out but want that. RGX-XXX get able in to start clinically We'll is order year. see to meaningful We something and second that get that demonstrating data. to in to we want get

it's when can responsible think bring have what And our about to ongoing past are But is part in other forward. how our relates property lawyers action taken else are say. we that have With and updates, we about we'll the there's say that nothing have And have executing Sarepta. on material patents view we for intellectual to we I portfolio in to and this respect at respect that I to we the able that point, of be those with to an that programs, that. talked it identified

Dane Leone

very you Thank much.

Ken Mills

Thanks.

Operator

you. Thank

Bank America. with question comes of Alec Stranahan from next Our

Your open. line is

Unidentified Analyst

guys. John, I'm is Hey, for This on Alec.

us. from questions of couple a Just

needs first do when new you by can that's and to be the products long first your fully to platform? for expect So platform, the supported NAVXpress how current transition to one. clinical one, be? And platform the we So the the expect

think maybe you framing market I time XXX, on could the talked, the on more for question Second just last we give XXX. color

targeting think for frequent require in still patients would injections assuming you're who the and opt onetime I treatment.

it for So percentage fall of like into me. That's patients you would say what that category?

Ken Mills

Yes. take question. the handle I first Steve the I'll the appreciate let second one, here. and Great, John. I'll

trademark got the this pronunciation for NAVXpress So us. new a name to because we is practice

is But CDMOs. our center platform Our process that have scale in manufacturing that installed have bioreactor Rockville we our opened center. last now a been process actually at here our that we also innovation using year, And John. facility,

incorporating been for complete material process to program, for it the incorporating program we've exposures programs, process it's to important NAVXpress BLA. to both and get our commercial the the incorporate as support We're into we phase suprachoroidal. our with RGX-XXX pivotal our designing on So phase pivotal for especially based plans pivotal subretinal RGX-XXX, into patient

comes been think it emphasis from therefore is RGX-XXX, that going clinic that transition actually starting transition gate out we've something and efficient we and a of be making through there's to clinics of to When the commercial. been clinic to the we'll point that in an be that's in with process, like

other we've of guided we second half with making in of the said then the for material year, NAVXpress to additional process process. that already the And like RGX-XXX started program programs that with RGX-XXX the updates we

be investigation incorporating In late-stage there that's we're this clinical very So, beginning a reproducible. basically, and process have and trials at into that process that cases, everything purity, high-yield high-quality, high one we clinical and important execution. now. we're other this to stage think In it with is is very some cases,

let Steve about talk XXX for So thanks market. I'll that.

Steve Pakola

retinopathy of about and here. or to question Steve patients. John, Yes, think a how segment common diabetic Hi, AMD the spectrum thinking wet of

that and makes patients two it who burden the higher injections. AMD every or And need anti-VEGF the needy more side, may On wet those more three more those a and whether every their months, know visits are is every them injections you're you're we getting or reality and you'd need the every of four rather they sense months injections than fewer month very But see getting infrequent every three value point they -- see caregivers the certainly have eyeball more across eye they are months, every into to or raised needed. injections or value and if they frequent more have injections who the spectrum, doctor. fewer you so your month

So can a can decrease option reinjections onetime we the who the spectrum. need those see reinjections, really need treatment in a proposition that or that either has for dramatically burden prevent still value you across treatment

data with We and even there, -- frequent high-dose but need coming patients on faricimab agents still Eylea of majority there incremental those do benefits with injections. the out are still, know like

think approach see opportunity of delivery they the that when a with that they this of the subset So patients would to we spectrum. we across there's clinicians really subretinal about Certainly in setting, talk that treatment.

where onetime infrequently opens approach in-office to of address might that high-risk an excited able expand the are to going And DR inject or think up the clinicians about. treatments of experimental very about if that as could. because agents administration We're massive to really delivery repeat suprachoroidal then patients administration available be a of none the DR even would everything that unmet need way need patients the you proportion

Ken Mills

was conversation guess, the bridging in in study, in had moment clinic see data. now were the when about gene study right Angio XX% I like I one-time could Peter talking with potentially therapy. having transition commercial the Charlie he it my of just with patients from of had that reported remarks, studying some Steve, is bridging the we're which that at I presenting, phase about as they to that you And thinking think that you were process. pivotal And the over and Charlie talking I doses data Kaiser, a was a

it's which to people think like the in that on now emerged John. days. program that times way clinic new So I and -- sort is day-to-day heard are has you the these their start overall, interesting experience go, as we've finding hear and from into of treatments things to of these basis also evolved when the at There clinic their quantifying physicians

Unidentified Analyst

Thanks, guys question. for the

Operator

Thank you.

from comes Ellie question UBS. with next Our Merle

open. is line Your

Unidentified Analyst

Hi. This for taking for I questions. on is Sarah Thanks our have Ellie. questions. much so two

a suprachoroidal wait to you move data wet from time Or AMD Are entering DMD. Phase I data half thinking First, then until second would include start until pivotal we to just going to there you the program second that of that? confirm And going if expecting else? lines is you you microdystrophin and and update to DR. a the on for for get that get we're want as III biomarker? should sort be subretinal something

Ken Mills

gene therapy suprachoroidal. respect AMD appreciate pharmacology your you planning I with one-time Sarah. between Thanks, being with that to made are for to think respect with and sort to But no decisions execution the subretinal wet No, on of learnings questions. with I for and there's in phase that entanglement we have particular. across both pivotal programs respect

we respect to at I suprachoroidal. now beginning with think that point not in program, the of where the are time was

Right evaluating looking and product place coming out AbbVie in decision-making a independent with subretinal. from the the doses profile the at REGENXBIO that of is are target that where it's against now, that's suprachoroidal clinical being data profile explored,

collection biomarker earlier I Duchenne, alluded the that points things of may Second I of our microdystrophin to And for measures But are we to able that from months may think X forward. that to measured biomarker microdystrophin assess. something bring will was perspective, months X be be from I that important a like we assessments. choice be there mean us, as an biopsy for question that time question, to earlier additional in Yes, view for initial

Unidentified Analyst

Thanks. Great.

Operator

you. Thank

comes Markets. question Luca Capital next RBC from Issi Our with

open. is line Your

Lisa Walter

commercial dose And did the level bogey high-dose Great. and for then Lisa on a kind I on success. in expression maybe looking at DMD of the XX% question. subretinal This the noticed bridging another seen one at was full on manufacturing microdystrophin the one on one study of the the method procedure. Sarepta's biomarker. be just not Maybe just our group Will patients from that the the time for Thanks Luca. is of for taking XXX, I data, receive here

just walk can if this. the there, if I learnings what through So patient why was get this like Thank didn't wondering there's from you. happened us you any dose? full And

Steve Pakola

one. can Hi, second take Lisa, I the

So we've a was success able the of need was this this where administered. the We're terms with There just because across in good procedure dose different case and very wasn't we kind up done, a studies to the in full of cases. monitor surgical rare we've change had that no this. the all this one-off particular anything to instance compliance pick

also have every we surgical same case and at then for trainer delivery. suprachoroidal our SCS the a single So

Ken Mills

Lisa, I first the to Sarepta certainly going data number question brought forward. about some with threw heterogeneity that in say would with, to be some level that going out be guess, of you There's And methods. different There's associated of there's collection. sort your I different that assessments.

within getting others models that with can to . dose in of want in observed think the respect range have think what at we I responses the I in to we humans. be of reasonable good translatability seen DMD have animals, levels and what in we're that, the starting because to what at we're been is animal

to other don't REGENX think Steve of different, be may domain a functional know that don't I'd I number, going alluded be amount you to, talking elements we're that have like a that about go proteins though, specific also have. is remember of got protein expressing, I we because to So the it's to that, when protein

observed range things a that different level. that, be in is in into type want And with Solid and And similar who protein a so gene the in still that of type Sarepta preclinical that and of the to and could mean in function seen. to we've benefit that space things positive different and could therapy clinical work, of of that we translate Pfizer ranges have others at worked have concentration, vivo our AAV

where that's we're heading. So

Lisa Walter

so Thanks taking for my much question.

Ken Mills

lot. a Thanks

Operator

you. Thank

Argyrides from Our next Andreas Wedbush with question comes Securities.

Your line is open.

Andreas Argyrides

questions. for Thanks taking Hey, Great. guys. our

efficacy? they how one about on the DR. Just impact XXX results spoken what's upcoming disease inform bar for with to expressed are And enthusiasm you thinking will in take around the a saving we've quick and on the forward? reducing here. it have decision How worsening KOLs

Steve Pakola

Hi, us are not though key progression prevent treated, we anti-VEGF Andreas, The know patients for great to even complications. question. is know vision-threatening we these getting consideration

the So progression for can us, basically. treatment goal, a decrease onetime is that

the that a and Now certainly from into can patients a trajectory. and over is otherwise is that to that. regulatory repeated goal bar a at inexorably who showing look to change certain injections worse require overall going looked this improvement, the of of we've eye take patients two-step at traditionally, time have what proportion that can we have But anti-VEGFs who get are seen certain

not worse. on they're . average, So better, getting

the risk of treatment have who have two-step the of where proportion we at really for So across really of are an think and just improve? opportunity patients proportion we're decreasing that. able of have improvement, patients approaches that an I look really across least patients one-time of worsening two-step exists look seeing not who spectrum can we benefit spectrum an increasing allow such a to the the And this see approach and traditional to that's the opportunity the a a at

we going be at points dose what see on endpoints levels as able at three an retinopathy to those our that So initial as approvable now we're three look do whole validated example, six as disease. FDA time look months, as for dose across that's across well even traditional to look, spectrum mentioned, diabetic the level we at at traditional Ken and look different to how of look

Andreas Argyrides

my for Thanks question. Great. Okay. taking

Operator

you. Thank

comes question next from Mani Our Securities. with SVB Foroohar

open. Your line is

Unidentified Analyst

Hi, is This line. on good Mani afternoon. for the [indiscernible]

quick for would And MPS secondly, ahead few for infrastructure impact we filing, of should the in the a questions. the CapEx and lines? impact program. to expect potential buildup of commercial How enrollment think Sarepta Duchenne about I the you OpEx? approval time Just on guess, terms

Ken Mills

Thanks for questions. the

as in need I think Duchenne, talked of a this high we size overview, area -- and an also significant the unmet population. we've about observed

been Sarepta evidence of there of ongoing development, past. the with and A, gone sort on products of Solid of ability among So many stages the studies even enroll. been different I that think been there's hemophilia have hemophilia to more also as different akin interference it have B at in I and think true hemophilia, as never in have Pfizer to with that's And and studies where been

there. impact an see don't we So

continue medicines clinic we into profile be think to in commercialization. a . I class of about the the towards these differentiated excited following other some

and to half are. as to that not processes II, the about With with BLA position respect in I plans focus expect second the in MPS think to would a CapEx year of what I overall materially. planning filing on program be into commercial we'll talk the in additions we update XXXX pre-commercial and come

changes provide the people that way observe infrastructure to work I would But building full information OpEx be enrollment be milestones the this we'll our doing think and for the BLA of infrastructure. associated in that we're filing. able complete prepare would complete pivotal about the other by that of to we of with once and commercial more we

Unidentified Analyst

Thank you.

Operator

Thank you.

Caroline Our Palomeque question with comes Berenberg. next from

Your line is open.

Caroline Palomeque

asked, question. the maybe but do want ask business question just taking the was for Thanks staying to first just side. My on I

more I your any royalty a revenue Thanks. you more were could about are guidance companies results your that positive us several know having I to using tell there lot were for of that? give AAV to if XXXX. wonder there can you expected out just If – -- vectors. as there

Ken Mills

Sure, Caroline.

to of expecting annual also see in think like that's transaction of associated to fees. and Zolgensma. I guidance the are some recognition back ago. plan have I into now we focused pay from royalty million has this fact revenues continue But that right top year materially, is did the we're we that revenue financing those Zolgensma outside Healthcare Royalty that on to revenue the things line a we in with this $XXX place from a years with of have from top entered the we And that license going transition our number think Partners, recognized couple operating line agreements XXXX, of

don't use continue we sheet. to balance build out that the to So

have cash already. on We that hand

potential release to on of respect terms We see development, in with in the this outlined those things some not transitioning today. our any do strong some either Rocket press of Ultragenyx of and approvals. in product we stages think I the including other but guidance later in are horizon of today, we're or certainly, performers like groups

Caroline Palomeque

Thanks.

Operator

session. your participation. for Thank question-and-answer you our you. Thank That concludes

disconnect. now may a Everyone, great You have day.

REGENXBIO (RGNX) 28 Feb 232022 Q4 Earnings call transcript