Earnings call transcript Q2 2023

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

Operator

Good day, and thank you for standing by. Welcome to the Q2 2023 REGENXBIO Earnings Conference Call. At this time, all participants are in a listen-only mode. After the speaker’s presentation, there will be question-and-answer session. [Operator Instructions] Please be advised today’s conference is being recorded.

to Christmas, conference Chief like speaker to now Patrick today, Legal would turn over the your I Officer.

You may begin.

Patrick Christmas

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

XXXX uncertainties differ as plans. Today's from of and that to to on regulatory words addition available guarantees conference outlook financial forward-looking those in involve subject results actual will factors call on analysis cause file are the discussion XX-K intend product comparable anticipate, may include XX, are Securities management's uncertainties. risks and and Exchange and risk report words ended forecasted such are and and can not other risk the These full be the the expect, performance certain development to believe, which identified by REGENXBIO's and annual described SEC's Form factors our risks REGENXBIO's Commission of statements and sections These quarterly in future may, the Any forward-looking and are on year website. such XX-Q, with statements December for in similar meaning. on sections reports plan, regarding forward-looking will, statements should, of Form in and risks

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

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

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

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

Ken Mills

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

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

gene patients proprietary to a platform. dosed therapy in of technology We derived been with who therapeutics continue thousands leader our be have AAV with NAV from

including a approval, growth deeper approval. it's a the regulatory time industry access understanding and AAV model support the endpoints We're support of biomarkers market and Recently, FDA's for of and surrogate digesting REGENXBIO. of been developing therapy therapy gene to gene significant accelerated

pivotal five leader from our now AAV therapeutics are pipeline remains products into five advancing stage license and pleased strategy gene And therapeutics. XxXX I'm REGENXBIO is track for FDA-approved AAV internal XXXX. space as in for commercial by our There on the program therapy how the

children quarter. in dosed been technology AAV NAV per which who patients from and our ZOLGENSMA, with platform, receiving every AAVX AAV uses is derived thousands are are estimate REGENXBIO NAV therapeutics. more We distinguishes There approximately What of our day our hundreds leader products. and have treatment receiving vector. a therapeutics pipeline It's platform two

treatments potential need patients a we AAV significant We strong living deliver with with ourselves for unmet diseases. one-time address believe to common and to that therapeutics pipeline of have rare the

three of therapeutic trials, diseases. neuromuscular devices six in using over delivery forms At are and conducting REGENXBIO, clinical we retinal, XX areas: active different neurodegenerative

to subjects in BLA one daily. file to on being our and X,XXX XXXX. dosed we have patient that include executing clinical new is the over our two phase on in We're that, In observing REGENXBIO first is trials average, programs team pivotal XXXX goal XXXX, support

work the end disease AbbVie XXXX investigational Our in strategic commercialize of or a REGENXBIO. develop to is partner partnership into and an ABBV-RGX-XXX at and retinal AbbVie primarily XXX the we with for complementary gene entered centers therapy. strong around

development major diseases and expect be for manufacturing. patients. infrastructure address leverage and clinical in XXX significant with to to we're option unmet knowledge We therapy to AbbVie's within of the care onetime gene global therapeutic eye Together, retinal commercial in-house expertise vascular developing AAV in development need our first deep

millions pipeline represent of our in of that of opportunities candidates over market Overall, therapeutic diseases AAV $XX high for current patients need billion. unmet addressing is

Day weeks few from upcoming new a suprachoroidal clinical microdystrophin Duchenne specific our Just retinal we interim guidance some our additional and introduced using in ago programs data and Investor we at about delivery July, updates. candidate provided our trial trial

And candidate inflammation AAVIATE intraocular mild-to-moderate trials, From dosing we the initial updates our safety steroid and observed X our the study a RGX-XXX topical with short-course of reported ALTITUDE profile in previously today. to drop. well-tolerated AFFINITY delivery that is we reported cohort eye suprachoroidal of DUCHENNE from mitigated with support

the a can bit Steve, in updates. the about Now, over review to I'll more detail clinical pipeline progress turn call so he and that

Steve Pakola

and to a subretinal retinopathy Thanks, AAVX gene to utilizes XXX which in with Ken. route treat is a begin via vector administration. wet inhibit VEGF. therapeutic encoding I'll being diabetic collaboration XXX, fragment AMD suprachoroidal AbbVie deliver NAV of developed our antibody with and to

development recently delivery these treatment the anti-VEGF of a The wet significantly global support enroll expansion the in XXX as ATMOSPHERE via total opportunity in Europe, the trials ages. to grow and subretinal Israel the is two to patients of of AMD X,XXX ASCENT. US, ongoing for program. pivotal being studies market Japan evaluated population of to is poised We announced the and

using and from evaluating as ASCENT, of subretinal ATMOSPHERE of also delivery. efficacy the Eye of initiated safety, We This XXX. who eye study patients previously with disease subretinal bilateral administration program a in of Fellow a is subretinal and injection recently treatment study the the immunogenicity pivotal XXX part fellow received

complete plan to time these submissions Overall, of the regulatory support all late to in through in XXXX trials XXXX. we first half global

and remaining Phase BCVA both from anti-VEGF pharmacodynamic the was NAV to study levels that pivotal meaningful data presented the our platform most Express interim cohorts Additionally, in tolerated well months along demonstrated our is the patients interim as achieved in low-dose process CRT and stable RGX-XXX earlier now X in free. levels high-dose designed improved fully protein this updated as with This dose X week, used using manufactured reductions trials. same being well through burden, subjects injection expected to The we results study enrolled. six new with and evaluate

X that fall our diabetic of delivery wet in in collaboration Phase ALTITUDE in of ongoing study. and two assessing in-office of retinopathy with under AMD RGX-XXX AbbVie, AVA have treatment trials also trial We treatment suprachoroidal the for the

from AVA at control presented active Day our of recently steroids Investor short trial dose safety GC prophylactic We X, RGX-XXX. is dose included level evaluating escalation RGX-XXX course an AMD. that administration XxXX pre data following the evaluating wet for of Cohort ocular treatment eye

drops, The inclusion presented and continues in to the of IOI eye in patients. of data X highlighted all RGX-XXX cases or prophylactic initial support steroid intraocular inflammation. the course of resulted safety short which profile and

RGX-XXX six-month the of the meeting full is controlled results delivery Hawaiian plan active DR. XXXX. ALTITUDE in of to study suprachoroidal beginning present Eye from for dose the X X treatment We of and Cohorts which at that believe DR given wet because and from patient in opportunity the this exceeds size a the the benefit about of excited of very therapy. population the AMD potential onetime We're can gene market, most we escalation

RGX-XXX patients presenting at AMD, cohorts We steroid The observed dose administration. full the X and to eye demonstrated this cohorts. X wet Day, in short resulted the serious events just Academy year. XX all initial later that data adverse drops from Ophthalmology of zero interim tolerated Investor these forward prophylactic in cases level no steroid we XX-month as presented with results X from and Cohorts look of Meeting at our RGX-XXX patients. During drops of in prophylactic inclusion X American well short with And in following was course IOI course from eye X, drug-related data to

Duchenne. to Moving

patients our remarks, options our we exon boys. these we Investor are were in to his Duchenne treatment and committed mentioned pleased program for bringing high need, Ken skipping As RGX-XXX. face to Day, new to multiple complement at announce unmet

Duchenne for domain a transgene Our is onetime therapy the of that gene treatment a a for microdystrophin potential found as developed dystrophin. highly deliver the first being a the program, novel in functional RGX-XXX CP occurring to designed of includes product differentiated elements naturally

muscle vector heart support delivery throughout and to well-characterized AAVX and muscle-specific NAV targeted designed promoter. using skeletal is RGX-XXX our gene and expression the a

serious adverse data was XX both affinity Phase Day, the presented The showed two in patients, our trial. from reported RGX-XXX the During no data Investor Duchenne with patients drug-related we well-tolerated we ages X and safety on that events. X/X

post days than months. of three more was follow-up Time XX administration and

trial, Society Congress expression in additional actively to include to Muscle We we microdystrophin look later year, muscle initial that and data continue this will safety in data three this protein at forward and recruit levels World months. at longer-term patients the presenting

observational continue AAVX in that is in an affinity We prevalence patients screening antibodies beyond Duchenne. the study also with of enroll evaluating to patients

our other Moving to disease rare programs.

is known as for ongoing We are onetime developing II, X/X/X AAV MPS the the an Phase also II for evaluated MPS investigational trial. I. Hunter CAMPSIITE being and of Mucopolysaccharidosis, therapeutic RGX-XXX two treatment programs MPS in syndrome,

date a the the or that potential its indicates May, medicine Recognizing to the advanced medical announced In to therapy we preliminary we the unmet of we Hunter from regenerative need for presented to remain CAMPSIITE data received support approval RMAT of for have designation using the our XXXX trial address FDA. accelerated BLA on with pathway. XX in completed patients We in filing XXXX and syndrome. half patients enrollment track first

for severe of RGX-XXX, Now onetime investigation of on an MPS the treatment I. therapeutic AAV to

on X/X this additional enrollment this program share we later plans trial for We track and the have of year. remain completed to updates on Phase

two the manifestations these RGX-XXX Batten are the also or and programs, disease. to manifestation RGX-XXX treat treat CLNX to addition of neurodegenerative In to developing we ocular

follow-up with Study for interim report including Brazil inborn this the metabolism errors from with in patient investigator-initiated first child study, with Physician to dosed RGX-XXX investigators expect of the for disease we the the a investigators under at of patient results and year. Symposium single Annual initial later data single six-month CLNX continue Society

programs goal to of progress with we are data patient and our significant advancements our of we RGX-XXX. conclude, have all across continue happy also dosing toward report We made the To our with XxXX. recent trial as updates first

patients, Lastly, to involved in families, who all and and these trials. are advocacy patient support clinicians the like thank representatives I'd

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

Vit Vasista

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

quarter second was R&D expenses for increase XXXX. quarter of to million second The cost Eye Care attributable the AbbVie compared the decrease to $XX developmental an collaboration. our $XX in reimbursement for of XXXX from were under million primarily

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

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

Ken Mills

execute progress of Thanks, curative mission performance. and as lives updates important improving the of potential those at we level, for our REGENXBIO a gene about perform continues high therapy. and Steve to our clinical our for financial Vit on

apart end-to-end our advance here as our to what's AAV team continues innovation addition to pipeline, running In batches development and early capabilities a possible scalable of us commercial-ready leader, Rockville manufacturing in also center research with And Therapeutics. platform and in set therapy. our gene our

capabilities for with quality candidate plans these our manufacturing presented we commitment new when to Day, expand Duchenne. exon-skipping to on IND at a of and center examples yield. when clear provided for we the science our innovation product data And Investor including from We performance, introduced a

the Looking ahead of milestones. year, for we remainder clinical early next the and anticipate year important pipeline of number into a

summarize. me Let

first of six-month the of data society as part the for a child of CLNX Of for treatment the our Inborn from Batten Steve investigators month, dosed the of report Errors RGX-XXX data is is with program. patient the will five-year-old first mentioned, form third Metabolism disease. follow-up the from This study neurodegenerative initial and at Next this

results held. from the of This at additional including ALTITUDE to share patients plan include XX-month diabetic Duchenne, will at and delivery data and XXX we being data levels World October, Expression cohort expect X. report of in In from interim interim affinity, full Muscle the cohorts to In additional trial the Microdystrophin Society, months. X of Muscle for suprachoroidal in the treatment for meeting November, protein first of X longer-term Ophthalmology American X retinopathy Academy safety at three and we

investigators suprachoroidal in the at six additional on and of next from results and January wet will year, X Phase finally, interim And Retina the delivery be including RGX-XXX trial full report for from of the Cohort will AMD of this Eye X, Hawaiian months meeting. II/III data and treatment

to a and of to a And we important partners, the the to focus patient So operations have as you of and lead forward continue remainder and of listening and place for possible value-driving we this with fund the clear and our into you of ahead with us what's We as updates, clinical AAV we lot in and us of path definable today, the team, continue high-performing sheet this achieve on year. trust to this continue have year. next our Vit described, year and look providing it's we catalysts vision path all strong to collaborators the Therapeutics. want balance into XXXX, additional to for thank for mission 'Xx'XX community

operator, we'll call the that, questions. with over turn for And

Operator

you. Leone RJF. Dane with from Thank first question comes Our Instructions] [Operator

Your line is open.

Dane Leone

recently. for Hi. we given both questions Two questions expect investors more progress, efficacy us. taking for data One, the could around kind of detail percolating AAV been you've all Congratulations what ALTITUDE. on like have and us it thanks from seems some with

AAVIATE full fix? from alkaline should or all XX-month regarding X-X Cohorts two will question across expect one Just months we be data just Cohorts it a look eye

would what you. comparability your protein Thank well? of around that expression elucidate Axle you have to the some world? see DMD could And assays can team at we whether microdystrophin your expect then method And programs ongoing secondly, as of peers

Ken Mills

good Thanks, Dane, the Steve, do take you question. piece? want alkaline the for to eye

Steve Pakola

Sure.

thanks Dan questions. for hi the So and

the for So latest have Wet a AMD that we have for follow-up. we discussed AAVIATE the update, results six-month

of of data as of type cut, the well this reaching far follow-up of seeing as to as interim in when as in really be AbbVie as closer more longer-term to part ongoing said we nature a later these get able haven't and overhang updates we with We dynamic Cohorts alignment because do the have and the might also always we meeting. studies

we that we a that's So as update the closer. type thing of can little get

Ken Mills

on able comparison the expression by products. we microdystrophin others investigation that think to be methods working methods used have clinical for will And of be of microdystrophin are the with used protein Dane, to that we class been in

I have spec into Blot that comparison as come with assessments Liquid I all forms that that there we are that So used of we world in well will assays, been muscle, think but chromatography-mass that as familiar we in that to Assays be know the interpretation community support methods some in other we Western of think And think think have mind forms as I nuances of of keeping from will us. and things always patients.

Dane Leone

you. Excellent. the Thank I data. look to seeing forward

Ken Mills

Okay. I appreciate it.

Operator

question. for next One

Barclays. with Gena from comes question Wang next Our

open. Your line is

Unidentified Analyst

I Hi. the and remind partner with I and status two first, your for to IP GSDXa guess, RhXX you any have as Danon us rights including It's Ultragenyx some Rocket as for Disease Tony questions. of well of on briefly, for IP can or the Royalty programs, Gena.

and Nsa? looking And what one DMD be for kind on from updates of later you then protein of would Advisory another terms bar efficacy in with expression year, for this Sarepta expected

Ken Mills

different are for the Ultragenyx part Thanks Rocket programs Tony. program using licensee NAV Sure, with license programs, respect vectors, Rockets of certainly two the a Technology in and Yeah, AAVX, are GSDXa universe, several the entered to or question. lot royalties for range tend NAV that under example, in that we're for years the type case AAVXand the on receiving, similar Zulgensma GSDXa that to We of compensation ago. in Ultragenyx licenses in using are we royalty. to into the have of NAV our AAVX

up in up starting and So digit into tiered going the high-single-digits ranges. basis a the on double

involving the of it respect composition patents IP relating involve both we as refer as Sarepta's that that are the they With which product, to Sarepta's use two your patents to of to. to currently relates product AAV-RhXXthat as question, of lawsuits second manufacturing well have

that of lawsuit, in the XXXX. on actually recently first second relating manufacturing is updated we the for the scheduled so patents of the And trial product to beginning lawsuit involves the

your know, that, ago. RGX-XXX RGX-XXX is and treatments, you second some microdystrophin question a data When program including going, few weeks think class the so and about that builds Sarepta which we're With expression the we of been like question, candidates microdystrophin have Pfizer think to Dane's some explored safety is of, to and far, where respect both comes provided Solid. clinically I others, expression, this and within about it encouraged initial off for the very data we of just

at products and program. our where of pivotal that approved terms XeXX can again, is and I think dose similar in differences And is understanding that a AAV trial, adjusting quantitation different understanding dose to We're slightly that in currently in they're for above of accelerated assays sometimes Sarepta's is, in product of be X.XeXX level. that there Pfizer my its

coming So looking clinical at achieving. data we'd XX similar to achieve of others that the of are see observations to respect RGX-XXX what we'd to into first protein days expression be some with

which substantial of function work, terms improving and microdystrophin. as pre-clinically, experiments to, meaningful a of a in other alluded in strength in established express Steve more the the AAV we AAV the the in to of microdystrophin children, potential microdystrophin first the has Now, once microdystrophin believe potent is that the clinical be more there because to or biological C-terminus, we're expressed of we've that our candidate that's the being C-terminal that muscle design domain, key component the the and both we efficacious be into to

to in that alluded be like the Becker Duchenne, would and adcom things attenuated And muscular of more about possibility so, the microdystrophin and in was recent sort dystrophy I of what consider akin occur to to nature. that FDA to being of discussions forms it's microdystrophin actually something similar that's

includes similar that most to things that occur nature. first the Ours is is product something that in

will we so, an that And amplification have of efficacy think outcomes. potential

measurement Society, be will the would months. maybe assess that's at or outcomes we'll XX-day something Now, points, of to et when XX XEXX this the not in will begin expression considerations, to the at X safety at referring longer-term in the update be the to nine microdystrophin nearer time responses on Muscle the that efficacy type like SFA, of course, be dose. be get later for you will Cohort to able we of in World our cetera. data But But those first point were of the

Operator

next Our with [indiscernible] from Morgan question comes Stanley.

Your line is open.

Unidentified Analyst

This is [Indiscernible] for head. on Vikram Pure

So is what for your setting? question GXXfit on in some where is therapies current DMD the competing rear view our

Ken Mills

the design Yes, Gus, it's for question. in a target great is of RGX-XXX profile multi-threaded us. The product

First be immunology to be neutralizing all access you zero as may strongly we just even unmet allow in need not antibodies. therapy that there gene that would when available single for AAVs prevalence pre-existing continues of because, is, know boys of that them for an in that's exist And products. two can a product or to believe because boys AAV pre-existing

development. maybe of So we a estimate incident that's on or a what basis first not could are point. capsid, other might currently based on is RGX-XXX include potential that XX an That's population the market access prevalence up be for to an basis, that to in able XX% on, the which capsids AAV-based is and

the just first terminal the -- that support products, an Tony, brought to effect in strength dystrophin. RGX-XXX but and was scientifically first domain forms of includes is activity of terminus in domain reagent answer alluding is preclinically microdystrophin and when of that -- we're the microdystrophin my that a muscle which exist is present that which, to point improved be is in biological Second doesn't forward evidence full-length clinically I truncated there's again, of C other to

that. up the today data can our that data, So potentially determinants points clinical the more us preclinical form focus the I that, that When that we of development. direction emphasized and we in a of best-in-class. in clear is in strongly more But think, think development, we'll we sets on for be evidence something later assess

We Rockville is, of to at we it when weeks This something like well comes market allow purity with as few piece I to is our on we're Center achieving Manufacturing are process to our and safety think, when then potentially that that the And the the a from things quality and the the as quality in and potential here ago. NAV last cost. yields will manufacturing. at being AAV on profiles think yields Investor just Innovation a highlighted manufactured products that well Day be Xpress improvement showing competitive as everything as us it the comes in

Unidentified Analyst

All right. Thanks very much.

Operator

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

Your line is open.

Alec Stranahan

for Thanks our taking guys. questions. Hey,

Just a from us. couple

could curious, a show RMAT First, MPS if II, with just if accelerated to prior with actually for would and what take line the update of on markets the to FDA clinically you give your filing? the it approval the interactions intend sense around you to designation top

the growth secondly additional milestone. year the any I figures this the is be milestone around two around next is additional color this then appreciate not around AbbVie but would Thank runway you. timing guidance, And and just great. over or in any

Ken Mills

to today patients the support accelerated our Alec, for study we execution from respect RMAT the questions. to of II, we've our continued on with the On well goal CAMPSIITE as MPS approval. designation those to announced just timing was to with great for front. And of plans support regulatory enrollment that Hey, respect FDA the XX completed thanks yes, it here

we've from generated the is And But are through status here consistent to in dose look, there's last quickly. input been clinical for designation trials transitioning and preclinical in RMAT exclusively assessed third that been comes case, data, our getting data this it we've our the that escalation other last acknowledgment some that's with -- pivotal data already has designations RMAT moving with FDA the believes phase this that's of of something enrollment clinical provided something types and to I way FDA in on patients several our our and first how years where is respect support. from thinking of potential that's been can the that from dialogue level to year. only on a rely a for that There FDA's with all sometimes think reported starting of the about dose that findings right?

So additional before that of basis us the that data the today, sitting in top phase, going-forward from But a completed the would for second I work support BLA, quarter think time filing of the the updates. BLA just the expect the on you to compile the data can enrollment BLA of XX to go but those this the here that line would to completing time we've filing. at absolutely patients not and only include going we're for also support line data

look for those on us So updates forward. going out

to it you're bringing back. When comes me AbbVie,

development, on first with our be is think, a highlighted for regulatory big addition by portion that this million to the regulatory in in milestones. filings we the and AbbVie. development I over when partnership -- eye associated REGENXBIO of and may that out commercial, actually think I number $XXX care in earned with of reported We that, total milestones is

we the today $XXX can a say with substantial and that development. guess that is is I associated is the advancement that million provide portion And of suprachoroidal what we that of can execution guidance

the bring and more with both advancement advancement development come that the respect and diabetic potential later for XXXX a so and AMD programs from about certainly we're and alluding And to those the ALTITUDE as wet this as for development updates lot AAVIATE progress retinopathy as is of the we for the learning into we earn us year to into and suprachoroidal as milestones regulatory starts into Steve potential outs advancement to XXXX, focus. regulatory and

Alec Stranahan

color. Thanks, Ken. the Appreciate

Operator

for moment One next our question.

UBS. from with comes Ellie Merle question Next

Your open. is line

Ellie Merle

Hi so much guys. Thanks my question. taking for

I to I to first DMD, the around just But level time we level. can level at what to dose differences and the lines provide Muscle. the when move data you the you expectations on the second you know first maybe second dose guess, get World your data For and color second Thanks expansion on said And then dose more expect mean, level. between I for dose higher are from seeing the

Ken Mills

layers few there. A Ellie. Thanks,

around guidance or given escalation of first haven't also plan this trial is or We the dose as of at potential well. for the any kind dose expansion any -- dose designed specific and of sort level

complete and will the that we of happening variables, of level to of and types level initial evaluate take where dose one. the the will be something first microdystrophin that So including into cohort account the different be at phase have opportunity sort in measures

human. to level as XeXX assessments expression seen animal the could models. well. dose the evidence of certainly it's this we've But respect expression. of want seen mean, frankly, of dose in to the functional course, designed in I respect because with how to seen recapitulated in at into With we and be study level preclinically, And understand we've also outcomes two, We've microdystrophin seen we have though, evidence improvement outcomes really strong higher and functional

the or here sort XeXX definitely us direction it's go. And opportunity is to hand in more there's we of detail we'll have some at when the to occurs, escalation microdystrophin we whether begin about that that to an to I of expression able want society Muscle World data perhaps present. function to talk dose So to XeXX for of think decide still expansion a be

before And I believe to specifically that using variability, Ellie, have it methods as and they've quantifying in But microdystrophin. people expect seen methods into we other for RGX-XXX physicians the all things that stakeholders, put context comes investors, but forward from to and bring that's microdystrophin validated well Dan's different are I course, from in assess programs. there's question, also think we alluded that to for really to, characterized, that and of assay of course, coming families it when we

reliable an are that And that at allow I we done on well contextualize. think excellent view everyone regulators going going view to are job to understand team and as our are are reliable to and working has methods

the As I ranges. clinical at the Sarepta the or that stated relative I we're of below a as where sort in and said, same preclinical perspective REGENXBIO data and all dosing similar slightly Pfizer sort think dose where these data from Pfizer of where of is says are all is,

our dose initial microdystrophin other within at data range to similar the So levels. of dose sort we're expecting microdystrophin programs levels be are what from of

enough the effect it's enhances emphasize molecule something a versus can't expressed someone functionally gene that in what of express I important a C-Terminal expresses going a the have that microdystrophin benefit different the of enhances potency, that therapy. C-Terminal to same is domain, without to functionality we

is is So, of think similar basis a achieve molecule-by-molecule microdystrophin we levels protein others, more while that and our effective. going we may that to microdystrophin something on be

Ellie Merle

all Great. Thanks color. so much the for

Operator

comes question Luca Issi from next with RBC. Our

Your is line open.

Lisa Walter

Hello for is on our guys. questions. This taking Lisa Luca. for Thanks

couple of ask more I what question questions D&D. Just expression just on and are. specific expectations microdystrophin the the a on want levels a to

a as So, microdystrophin what Is XX% to you in Sarepta change XX% in October. the on label. should get more from when baseline to data that for a see expect we expression has bar success

on biomarker the function taking camp? biomarker And but I your approval? if for in And approval, mentioned wondering on later surrogate it's their wondering also as be a and functional what's and just efficacy still on look Thanks for Pfizer year gaining gaining the camp interim days, not questions. on will biomarkers. know you early are strategy for precedent regulatory D&D, that this despite starting favorable accelerated yesterday

Ken Mills

Sure. medians Thanks, and can look numbers, also and at numbers bit, I Sarepta, Lisa. -- small there label they for of And have would a microdystrophin I means Digging expression. certain product when in we referencing into quite was of mean, I distributions look a say fall at cases, patients. we -- it distribution approval we'll had should think the

go we're would something the territory mean, that far because was Sarepta for Sarepta, to over up for have like things think to that which wouldn't by me, both going and median be I us, as expression. that be first we to the nearly as say reported that think from microdystrophin I like enrolled So, the that to basis in were of respect of zero I think been Pfizer does on the with that And like XXX% patients view of definitely I I anything ridiculous.

by us would to for reported comes of comfortable is for of like and of there's for Sarepta and means XX%, methods been that would it in sort I I and the XX% a disease, of think different similarity. have be things range something to what be media and that done that heterogeneity Pfizer would we've us something But seen when that of think assessments the of sort terms adjusted the feel types that of

dystrophy respect With well, well have I just entered both franchise for REGENXBIO I stakeholders on development our with -- about been the my the let the we've and here, mean, very neurodegenerative Duchenne say that much pathway. as as regulators approval think through to me of regulatory approach since discussions the muscular focused of products I observation think accelerated use

more great own need with on an avenues are we longer-term a different of And think accelerated continue approach that's where regulatory an FDA. we and we'll patients there's confirmatory this urgent our especially experience, to and of different. the know validation continue labeling that approval all think, we support all other the And I of AAV supported to of that of taking to think basis, stakeholders that development sort the basis and sort think well we also and from that approval our the of that our I there's the and -- to approval. data, limited sort product, milestone on now, both on that development the trials and of functional information first sit accelerated approaches of show right as of from But own approved as data establish in first think, process of sort -- to a be trying is continue execution to the that I valid that accelerated to therapeutic basis I and people comes

developing voice a a we're here, in that's on forward clinical based significant XX. significant still and There's the need here. there's way REGENXBIO, to trial Average from urgency enrolling unmet all age up

of we understanding all and anchor And accelerated we are supportive to we think and to to that where so regulatory is that's something that well. ourselves patient I want as needs the that contribute the that think the stakeholders approval

Lisa Walter

questions. Thanks my taking for

Operator

question. next One moment for our

Our Baird. comes Skorney with next Brian question from

open. Your is line

Unidentified Analyst

retinopathy, had wanted you'll what so our bar to manufacturing suprachoroidal like the FDA on what take going what kind is kind thinking of a for Thanks a to to going interim take how Hi. far Phase diabetic efficacy. what what ask, the for NAV and later wanted on it's progress little more results regarding the AMD, -- platform? of X the of you. color questions. seems Brian. And get is we Thank the reminder We terms of Charlie you're for just with delivery about AMD. it to be like taking conversations presenting fairly Express But going is in for be kind you've we also is to This on in for to straightforward

Ken Mills

you Do ER question? want take to

Steve Pakola

Sure. Thanks, Charlie.

what There's mentioned, for you and precedent two-step progression. at severity, DR, to diabetic as their retinopathy we and care injection repeat looking it's readouts with both for agents. have AMD that's the a done DR. the at a is look consideration been patients largely what's technical really powering wet of anti-VEGF interesting. and on On improvement So opportunity doctors preventing consideration But prior about

of have of patients to is patients really then progress level is likely okay, vision-threatening proportion goal. certain a a that So the having which for, complications, blinding not proxy can improvement or have more you're ultimate to a

clinically meaningful. I reason of those that both the context So is give are

in see of with a happens observation. going is history earlier these negative if you to better, of that patients natural patient's in what months worse what our trajectory our direction. If a distinction in and that's control we And patients proportion patients contrary a not also cohorts have and from overall we've we is can arm in you with worsen. know could to-date improving And seen both the six have really lower results

for repeat we thinks that totally a treatment is know bar because injections, one vision-threatening I initially. fair for burden these is much just say predicted off this patients stave many patients aren't for actually too signing asymptomatic the it's And something irrelevant is think us injections onetime to complications, what can as for know indefinitely we and also with in-office you but the up that with repeated treatment

have But lower a consideration. a if that a risk is bar treatment, one-time could for you much in-office benefit

talk we clear commercially clinically clinicians, So experts worsening when in meaningful investigators viable reduction and a any and shoot target to very really is to for. clinically

of really we're way the target product for that's a So profile. proof-of-concept, top looking thinking about on a

results that come AbbVie and we So look definitely later. to forward seeing

other I'd The is durability. thing say

good year at to very It's we've great have So six be results to going a assess seen this durability later to months. an out opportunity to year.

Ken Mills

side RGX-XXX the qualification for of planning have RGX-XXX. it, of NAV XXXX the in Express I think that process process both lots started On creating and around BLAs of the we

so conveniently subretinal Again, had but have we've FDA. opportunity XXX had a for the of studies, And with with over XX the late-stage We've facilities have we the lot we've also approach, it to Rockville discussions cGMP advanced to here dialogue we the to visit and of we're people running FDA. trials, capabilities and feel with comes have opportunity two being had clinical more when XXX had like the one high-yield exists. facility high-quality, commercial-ready that in strongest

Unidentified Analyst

Great. Thank you.

Operator

next our for question. moment One

Our with next question Wedbush comes from Andreas Securities. Argyrides

Your open. line is

Andreas Argyrides

Thanks our here. taking for questions Yeah.

from two us. Just

competitors? to for how FDA support interactions For with accelerated RGX-XXX and your MPS II, came the and versus the time they the can elaborate on frame shorter approval you

of wet AMD is pivotal DR, at advancing second data And then collect it Thank part and for it so the day? whose to XXX the do comfortable -- feel of to the AbbVie into question, you what you. decision or trial need probably end and additional

Steve Pakola

one Hey, first. I Andres, can take the second

updates going look into answer come and think good response we're I ways potential dose first, tolerability response. we traditional like we aspects news account that obviously dose, So, potential is, I generic clear we data at if indications, is consider very also response, always take the and noninvasive in a the have dose for give to guess, assess and and both safety all to

decision, that looking overall sand, because suggestion give in least at gave question the such line and your results collaboratively work in at the to I'd and we committees joint diabetic one decisions. related that very the make these of And together say retinopathy. really to is with the target have in like all of it work commercial and committees really data terms against cross-functionally the joint development that than we cases and profile AbbVie our there's evaluate can other who on closely efficacy product and you joint makes the traditional compare not I And

Ken Mills

components Yeah. XXX, target question one-time the close CNS And the competitor a is to product with not address is to to of which about really Hunter therapy Syndrome. there a RGX-XXX, of respect the profile strictly first

US here, with treatments CNS for those standard of of not and, class unmet of address existing being the need on care we the the So, the think because both one-time features. stand like you of know, we sort a in nature standing of II Elaprase, to MPS top in unique features able

it accelerated. been that to encouraged the RMAT think And the the I be such is program they're fact reported a that as, meant is that by supportive know, so far, the that of the of they status of FDA type clinical designation that has you want data designated has that

that think and we I of and that the we're we a We support stakeholders profile one-time data. reported that unique that treatment good addresses good from about feel the about Hunter really data about regulators. the feel feel So our CNS and good manifestations we've seeing of

Andreas Argyrides

the meeting for We up progress. Thanks color can new [ph]. follow-up one on there the all on coming Great. and congrats

Operator

question. our One moment for next

Mani comes question Foroohar Leerink. from next Our with

is Your line open.

Unidentified Analyst

timing available the University the were the that between and Good bit Song if any and company as Mani. as lawsuit potential is you the kind division provide was know afternoon. were I wondering the had [ph] for the lawsuit Pennsylvania? there Lorena the on damages This or and of on touched little I infringement you Hi. thought could terms in level of for updates earlier second is of a that of if well potential of

Steve Pakola

So lawsuit. we update the of the have timing don't any second on

not As XXXX. of timing case exact the is otherwise first built. I is second that's yet And trial alluded the early the scheduled to to, go lawsuit to in the

Unidentified Analyst

And that you or on terms certain year? the for from impact the in expect you impact would -- what well you. study in recruitment results do to approval, this Thank be think potential as so recent would that so the later expected as are enrollment your study that potential Duchenne the program, –

Steve Pakola

immunology, Duchenne maybe prevalence rare preexisting certain they other And we understand in of gene be but not access is that trials for -- reasons, everyone achieve in feature and that a AAV our that disease, there in, operate important treatment incidence Yes, voice, than variety from think trials because the of clinical it's therapy another. to and vector to a a of that some because of larger therapy continue can an sometimes of a serology, in able again, aren't commercial kids even development. areas in but I other therapies type

we have So right enroll now. ability RGX-XXX our a the not impingement we program, with seen do clinical any stage really respect nor to on to see to expect

in improvement be microdystrophin of because of And on show I both it and generation about is aren't going a could bring additional the that technology. to on the naturally of just should other I there's more go first that should potentially or of the for dystrophin of fact basis the think well to to our some center of emphasizing class addition have products of access able with active the thinking really be as as treatments. of domain data microdystrophin I biologically our opportunity forward, the evidence the innovation basis length boys all microdystrophin show that's to dystrophin, data, of benefits that into when really, the look, form to is microdystrophin to of of inclusion think we as RGX-XXX more class better C-Terminal the But that truncated going as similar be we the manufacturing there's product full forms potential exciting our to be an of And think to, improvement incremental focus. that existing we

Unidentified Analyst

the or six-month expect And than function speaking of in muscle other programs, terms on earlier strength the maybe to see differentiation should we you. muscle potential at impacts potency, actually readout? Thank say of

Ken Mills

they're human ways. of the that as different models you models later different the certain be fragile in very progression, than I forms from in because think disease, the animal can get types the that hard assess animal it's to aspects of in the

you so data. And collect same always longitudinal that can

distinguish more certain I on I six think of biopsies expect the time time clear the would And months. accompany understanding, the point. the could between separation from that basis it the points be well. see microdystrophin three would something to to types XX-month measures We of be certainly one-year at as think it think for wouldn't our at expect possible I measures would we something and like themselves

Unidentified Analyst

Thank you so clarification. for your much

Ken Mills

Thanks.

Operator

[Operator Instructions] question. One moment for next our

with Berenberg question next Capital Markets. Caroline Palomeque comes from Our

is Your line open.

Caroline Palomeque

thanks taking the question. for Hi,

set AMD, atmosphere, on especially AbbVie of as on Thanks. study? the to on the So trial sites RGX-XXX any patients in the for subretinal the updates well from wet they enrollment are pivotal there as trial since number increased

Ken Mills

Hi, our Caroline, so we change don't in guidance. have any

the half track to XXXX. well trucking late program. submissions through the expansion, along. everything going post achieve we very European is And the studies global continue complete And expansion XXXX regulatory first So yes, the on of and we're that pivotal in BLA can of these so we

Caroline Palomeque

thanks. Great,

Operator

presentation. not further this any this I'm And And questions does showing today's conclude at time.

wonderful may a disconnect, now and You day. have

REGENXBIO (RGNX) 2 Aug 232023 Q2 Earnings call transcript