RGNX - Earnings call 4 2023

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Operator

Good day and thank you for standing by. Welcome to the REGENXBIO Fourth Quarter 2023 Earnings Conference Call. [Operator Instructions] Please be advised that today's conference is being recorded. I would now like to hand the conference over to Patrick Christmas, Chief Legal Officer. Please go ahead.

Patrick Christmas

you us thank and afternoon for today. joining Good financial results year for this December afternoon, fourth operating released REGENXBIO full Earlier the and quarter XXXX. XX, ended and on The is our website press available at release www.regenxbio.com. expect, forward-looking include similar regulatory can differ to will, from results and to development plan, risks will call uncertainties product such identified conference statements our by may in words and are financial statements other forward-looking believe, and meaning. to that anticipate, those be should, regarding words plans.These and forecasted addition outlook subject as may, actual Today's cause intend of Any uncertainties. statements not performance future and such forward-looking guarantees involve of risks are certain and are XXXX, quarterly report and file and the Factors XX-K risks the for with REGENXBIO's in of and and information this sections year Securities of on Factors Management's otherwise. and Analysis statements we on December ended make are comparable obligation Form the These full call, call may Discussion XXXX, which the XX, on Commission the Risk XX, the REGENXBIO's described on Risk section Annual XX-Q, Form Exchange only new this is update future account any February information, to date and of provide on of conference we SEC's undertake or and as provided we call this available website.Any events forward-looking on no Report and webcast. that recorded advised being be is Please call today's

REGENXBIO. the preliminary and of purport call turn results Mills, or unaudited results any would differ Actual now not pro forma provided financial of does Ken addition, to may like is may project In or company. CEO that financial information to over the operating positions to materially.I be

Kenneth Mills

the call Steve of on joining programs; XXXX. recent everyone, update afternoon, Thank our the Officer, to ended where last prioritization on year as our for pleased our up begin candidates then an on pipeline plan you financial resources are value fourth as Pakola, focus of will differentiated, quarter work of our for for provide to product overview an Patrick. XX, the enables recap to Vit Officer, a will we're thanks very us. commercial and expedited an Medical full Financial and meaningful going opportunities December began our with REGENXBIO the and well much, can a I'm business At highlights end and on large capabilities year, generation clinical and Good open the provide results long-term. goals. questions.Late Chief Vasista, for line and corporate update us Chief be that support the call, today's soon

ABBV-RGX-XXX partner, retinopathy program Hunter RGX-XXX for priority RGX-XXX of delivery were XXXX, Syndrome.Early of We exciting in for wet Our collaboration injection programs diabetic prevent each are the believe gene AMD MPS a the these programs. for currently on pivotal AbbVie; trials for there's with treatment with in in developed II with track our standard and care and of the XXX, a subretinal treatment single retinopathy. Duchenne, diabetic wet the therapy progression data experienced on, progress disease the in X ago, a remains we're ongoing we've by program pipeline of for on multibillion-dollar experts AMD clinical for treatment potential exciting become treat or and treatment announced from being Enrollment of of submission Duchenne, be our from of on. wet to first-in-class milestones. hosted to AMD calls to us. treatment and today, Cohort we with positive regulatory new positive delivery.These data review enrollment events trial. the XXXX. the half global additional support and first our data retinopathy amazing AMD guidance retina upcoming using about we're We through two of in-office to weeks for expectation for of attention And completion our wet interim we an rare the in to and and recently talking end In going suprachoroidal that our our diabetic giving XXXX events focused and leading Three additional

thrilled microdystrophin expression for of our we trial is RGX-XXX strong across observed think see BLA. we results on their what up significant which skill treatment a breakthrough patients.And is a are and a We demonstrating range major statistically to acquisition with neuro-developmental I achieved show II, wide we pivotal MPS have of continued four this intravenous improvement care enzyme are therapy. filing recently Patients in years, discontinuation receiving standard to to for

acceleration for supporting on our guidance, mentioned, gene diabetic AMD near-term development in-office how expansion and Duchenne demonstrate today, shareholders.And and treatments the for milestones our plan recent of we're the new as I wet therapies for and milestones the Our our providing new using delivery. retinopathy of value is of some

for on and programs. share things turn the to week, clinical going over at update progress our plans of Steve, we do absolutely AVA can in Next to have XXXX. I'm off the with started way share being ALTITUDE we Dystrophy to Duchenne we're the our progress circle new the so held talk going bit II the for about data back and mid-XXXX, to and QX the dive to Also, program steps the deeper Muscular trial for updates a review expect about in I'm next Phase thrilled trials status and he and has in Conference have more Overall, now, Orlando, for and it Florida. a new us.But in made respectively. prioritized Association company

Steve Pakola

ages. through of the global utilizes is treat deliver anticipate ongoing a a in antibody trials, the expression novel is X levels to X,XXX first fragment Japan, AbbVie, is and assessing and total exceeds we AMD the transgene wet and is X look of at presented differentiated in we controlled January, events. DR, patient full collaboration route with severity II delivery escalation and improvements and is continued program of delivery delivery treatment AAVX Thank have two be presented you, for Meeting. updates, gene evaluated treatment meaningful to could controlled of pharmacodynamic dose reductions which DR opportunity very or in disease suprachoroidal via safety muscle skeletal suprachoroidal delivery for dose the therapeutic to grow XXX suprachoroidal II administration. We trial patients presented a active VEGF. XXX, to X-month that opportunity occurring to subretinal promoter. trials given in to Duchenne; study. and showed onetime inhibit the reduction patients a Eye the ASCENT, anti-VEGF year.Moving the of this as used vector of which the with elements are Diabetic encoding gene We're vision-threatening wet The for functional in expected study this Ken. dose domain enroll Europe our the pivotal data showing I'll levels subretinal a developed Hawaiian the for clinically same with of we therapy evaluating submissions X to microdystrophin XXX well-characterized our and an is NAV population in results forward from two XXX of begin Retinopathy AMD XXX Phase a population late Consistent in for U.S., highly XXX study benefit regulatory AAVX study a from that ATMOSPHERE AAVX also the XXXX.In diabetic second of anti-VEGF retinopathy, the well-tolerated RGX-XXX a in the that to C-terminal to CT in in because of treatment support we we of deliver market updates about or In with profile fall the observation designed AMD. previous AMD trials, in being and to ongoing our targeted excited designed of DR. addition quarter XXX potential and gene X-year this NAV heart from vector the the the the this poised is treatment with on most being RGX-XXX which the via treatment collaboration product significantly potential burden.ALTITUDE muscle-specific X, the the being of a data pivotal to evaluating half wet gene and wet AAVX the believe in In in the is Phase meaningful ALTITUDE throughout for XXXX providing naturally two of using market, that November, additional wet dystrophin. AMD to XXX RGX-XXX and with a includes of onetime in size therapy. under demonstrating in Cohorts treatment AbbVie found in-office support

demonstrating capsid mediated a for continued Duchenne next thus robust expression activity to three showed events XX.X% far.Importantly, trial, may improvement. boys. or serum updates control antibodies.In to reduction in younger share across clinical at level for level levels, compared RGX-XXX interim no the months, dose up expression at and serious presented and drug-related be the I/II patient be dose X and RGX-XXX X dose levels. with excited patients AAV kinase from was therapies to broad in of Phase trial. microdystrophin new Conference, we February, have and AAV All age data highest well-tolerated to patient neutralizing normal three not seen exciting who program week every of eligible the preexisting be from CK an expression third Phase DUCHENNE both represents the supporting Our in or RGX-XXX data at Coming adverse patients older from X from that microdystrophin creatinine we're both at alternative at seen baseline the group evidence measured RGX-XXX age to microdystrophin also MDA due AFFINITY we other I/II presence of boys

of the Also strength CSF endpoint forward statistical assessment a data fluid, we to clinical made this that conclude, levels. as CAMPSIITE all across in reasonably WORLDSymposium, accelerated with activity brain for RGX-XXX the programs updates under a remain on surrogate at high We our levels of CSF-DXSX predict primary decreased significant We approval II biomarker endpoint using cerebrospinal have disease in significance.Patients look February, RGX-XXX key file also of data the with maximum XXXX later trial we in DXSX, met disease announced sharing achieved pathway progression treatment its and year. the trial to and attenuated of initial below with MPS pivotal to treated To functional benefit. pipeline. progress likely to a BLA track

the patients, our financial review families, to guidance. the programs.And these have patient like call of I representatives that, Vit clinicians and with I'd to all turn Lastly, who supported to thank advocacy over

Vittal Vasista

securities $XXX totaling ended XX, Thank million $XXX XXXX, in December for to used an in Eye primarily manufacturing increase end to cash, XXXX. the were XX, development decrease compared expenses December equivalents to Care a million compared Steve. fund marketable XX, to on $XXX XXXX, December year of operating attributable under you, our RGX-XXX, expenses for in the was year The ended cash from AbbVie primarily REGENXBIO quarter R&D reimbursement and as activities XXXX.The with resulting by cash million million trial was and XXXX. driven and AbbVie in cost decrease clinical Collaboration. $XXX

the achievement received under be the to plans collaboration.With in of the back This into commercial any may of current to excludes cash, the runway of company's operations based is that, as half thoughts. that impact XXXX, and will call from marketable turn AbbVie December XXX development operational $XXX cash I milestones equivalents provide expect We XXXX. securities upon the on fund the balance second guidance Ken million to of final cash our and our of XX, payments or

Kenneth Mills

accelerate of Thanks, Steve. valuable are our that to and significant Thanks, these to as we candidates value our for to development are XXXX, product our candidates. Vit. plans ensure generate allocated shareholders intended resources most the

the challenges operational our XXX needed support assessments goals long-term trials with in enrollment outlined by work today there's of are through is that that completing announcements, overcoming a to XXX using is Eye important to underpins of onetime XXXX. need and the of partnership steps supported retinal submission our where taking and in filing features its RGX-XXX to disease.Our motivated enabling and directed addressing at program. achieve global using of with press unmet strategy first managing the supporting differentiating value product.We're support we delivery, clinical pivotal pivotal and a XXXX first our from for be we're data treatments to using Duchenne capable new trials Care regulatory market which patients in-office AbbVie unique with for by by RGX-XXX has large AbbVie and health here our multiple As initiation and a patients. for the for believe pivotal best-in-class treated potential need of vision RGX-XXX therapies this Initial unmet to ongoing of sustaining delivery suprachoroidal subretinal half potential is by accelerate plan expand this to gene set release a therapies, end trial efficacy this for a year. initiate We us completing

filed year, end data Our recent pivotal to By top be these meaningfully stage, of Based possible. by changing a BLA everything stage function. enrolled under BLA. our pivotal a file course initiating on supports of fully line the at this disease we gene in data, in should restoring working as pipeline quickly pivotal that as or we're with MPS the boys are their missing II

of about announced on join patients. year questions. thanks, we week that later we Day. believe having recent well-positioned for today. and in to and result And your everybody, is treatment Rare a ask updates, I our enthusiasm recent us that, for you upcoming throughout that candidates term.So in turn these the hope Disease the our honoring today, this will success this that updates about impact call With share near-term milestones time treatments are Operator? As these REGENXBIO XXXX the long over milestones and you and also We

Operator

Wang come Instructions] line [Operator will with the first question of Our from Barclays. Gena

Huidong Wang

here. progress clinical the on congrats Also

and week two the other is XXX, So XXX regarding and next for both is later 'XX. the the one data update

please could additional be we able the to out that see? will So you points lay data

Kenneth Mills

next to and under conference. give with status have We the embargo with Yeah. updates program, able to not also data conference Thanks, week. about here respect we're Gena. new fidelity of We're to that level the the respect plan and today to an MDA

actually to see respect of there that week. investigator now at both conference and And to as an give there submitted we're able XXX.With data wet XXX on updates we're the though and So is new anticipated share also that progress have currently previously presentation diabetic be posters part we scheduled, the some next week to And we that clarity continuing more happening retinopathy. AMD suprachoroidal, will the with next just updates.

at at Just the suprachoroidal we update. update. had month end weeks a about a the few made ago, of last Eye year retinopathy, ago, the Hawaiian AAO, And diabetic

work So program by more time teams. has gone by, more the

new So to we data respect and anticipate be program there's going mid-XXXX new QX updates include that updates with and to suprachoroidal.

Operator

come Our Stanley. line with of from next question the will Vikram Purohit Morgan

Gospel Enyindah-Asonye

Gospel is This Vikram. for everyone. Hi on

XXX patient We level for have CK patient the for of X? that have contributed the and Since on characteristics, any baseline the expression updated have characteristic higher last question in commentary may you X levels for one the program. reduction X update, patient and microdystrophin do to

Kenneth Mills

Hi Gospel. for Thanks the question.

people similar. that other be different enrollment patients a of see including used that that new this we're XX, then sort heterogeneity microdystrophin.I all of largest in been wider the in terms we hopefully of I have of it we're progression able natural be X highly general, we it no from reinforce can is the sponsors. in But as data been just enthusiastic information are this age range This therefore, the inclusion particular backgrounds dose excited RGX-XXX think at reflects into range by boys. things about think some to the continue end attribute the criteria, X what the sufficiently to of to have expression also that I treatment, of robustness enrolled exist think reported and was have the here I disease. X.X-year-old. meaningfully of to increase we last And to level to expressive patient us, get the contributes trial seeing really being and in to think sets. high going of in in and And think I terms since in to we've certainly really, Obviously, time the for these the improvement are meeting updated got we

Operator

with of of Stranahan Our from next America. Bank Alec will line come the question

Alec Stranahan

me. from two Just

on for to something you data And approved, what obviously as competitive formed? maybe of First from but on program realize I've focus advantage, furthest or see a XXX specific kind point, there's moat XXX, here ahead. AMD, and guys wet Is Maybe being got lot to would partner, be therapies developing uncertainty of wealth obviously, I do pivotal seen great. assuming a you a gene of scale recently follow-up. itself? that AbbVie this still Any then thinking thoughts beyond launch but to your an increasing companies other having the are approval, we've XXX gets get

Kenneth Mills

physicians with addresses injection with sort seen single in improvement years -- And and a disease. investment Yeah, who for far we've terms vision along have the managing we RGX-XXX that's the Alec. some that the above, and maybe in outcome that of us of beyond since is have in in emphasis out patients evidence to mean, development four I and they I partnership in four with of of overcoming at are its vision vision have needed years and long-term to AbbVie with is from we never circumstances.So even some the challenges respect and that, patients the think all in than and health patients real more patients particularly is I the both received development with being made how XXX side. years. an that in sustaining retinal long-term overcoming injection that three mean, day-to-day with on

commercial incredibly want be to I how know that important durability and Steve, the evidence of on teams medical about is the think how to about thinking comment in vision that with focus don't translates? you and you're clinical I going that setting. that if and

Steve Pakola

Yeah.

improvement of competitors of the of and I the we burden, show. you reduction the of think to the opportunity certainly durability think VA can in unparalleled findings. And with reduction treatment database this as VA out with that's combined burden durability the combination the of the patients a throw any even and in setting dramatic have durability, when a that really the mentioned, compared in you to exposures patient treatment in you there of stability big and when

to-date. results So our over they refer investigators kind repeat of the seen this and when over again we've that to

Kenneth Mills

how great the a in see And what influence and the they're AbbVie's enrollment for on decisions partnership to for this clinical vision the and complete and need. ATMOSPHERE the -- specialist I late especially they're long-term mean partner a thoughtfulness that's thinking global retinal and patient trials into obviously about reinforce believe differentiation stages. they bear development, of development We clinical and ASCENT, and have health unbelievable footprint that I tuning experience to to about But that engine come global with an views positioning in really case, things just and with RGX-XXX. ex-U.S., been have it's really phenomenal. pivotal we

Alec Stranahan

maybe quick the Okay, runway. cash great. one then That's helpful. on just And one

could half pretty big can or milestones from other anticipated guys regards you second there with over levers to push comfortable any XX that trigger 'XX are say. if this events in setup AbbVie needed past Some the pull are runway? months, the it But to XX let's the next to you

Kenneth Mills

meaningful. on Directors updated very focused commercial as for on the differentiation, were company that very our with reductions plans It opportunities shareholders value and that Board we cost strategic hard enhancing meant some opportunities choices. of and potential our we think said we a I year last were involves that after introduced high focusing and

talking think I for on in having being to some early and the in quickly year possible changes the in feeling guidance as goals on that yeah, and continue only with RGX-XXX therapy reduction that to not choices replacement I enzyme we the first reported We're And BLA of notion it. well some line approval, some confident for that to track.Of partnership. And share able this could eligible that as of data of the PRV. but progress that the be milestones, of of those part for to continuation. filing, about data as seeing that updated those confident expected working respect track the time about today just of been like And really the reduction on suprachoroidal that and those in and been with be be we've us achieve maintain get continue basket we've other seeing were achieving to observations the AbbVie on course, really BLA ability as for mean, the to top burn.We've positions additional pivotal to updated milestones

focus, just the with this about a strategic enthusiastic updated we that have So think plan really science few months right from I and we're and removed the momentum the now. plan the with

a is capital good in place. the think I

Operator

UBS. line Our next Ellie with come of question will from the Merle

Sarah Krupp

This most Phase later can is ones guess program Phase And expectations before functional and from on longer-term us. Sarah you to Can for you Ellie. a two gating thoughts we from this recent or III superchoroidal quick us, year? your I then the data talk data? on strength see we from that starting III start about also, initial XXX remind could is data potentially into XXX

Kenneth Mills

be and guided Sarah. share able and that dose respect level Thanks, over function we that the to to XXXX, we've X dose motor of to expect X. for level achieve in data that course With strength both

as assessments surrogate that's is routinized clinical those. benefit benefit.And for in clinical that improvement microdystrophin we've been by as been the endpoint in and daily that for activity enthusiastic with to function that and think X, motor all It's there's strength be things level supported these fact a seeing as we has that looking predict with really the boys been an we're approvable to-date to labeled continue here FDA. I well reasonably dose therefore, the the expression about likely

then weighted the making then giving sort The migrated we've as really data on year, second we're which pivotal microdystrophin to microdystrophin towards be additional of pivotal motor data.And it towards updates, in will our decision, I really that's more going microdystrophin, but no, for mature to to be and safety still start set will the trial, with through going to progress So with cross will think half by something dose pivotal accelerated the approval really guidance not track. that of and will on because is to is an that data conversation we're be as initiation up communicate final strategy that strength be be about start plan only confirmatory of going to about plan how think we're well. I based function data evidence

So I think everything next is with story some have Dystrophy today on track breathe as we've again, data expected the it just this question? could the going Conference we're Associated Steve, announced to new be on update XXXX. at throughout by now. let to And continue Muscular week second to another

Steve Pakola

Yes.

So thanks, Sarah.

you thinking III. we're long-term do before Phase data, in about need mentioned how much we So suprachoroidal

both terms and it's point. proof of seeing mentioned, at severity to reduction a well-tolerated, we vision-threatening diabetic what think X-year that, year, its from we retinopathy, concept, we I see the that's they improvement X-year know know able we're fortunately, validated exactly we diabetic And retinopathy were in pivotal where dose mention and scale. to data with and events. that, an on Ken to DRSS a acceptable X, we important what as X want last we time use were example, levels And but of is not for endpoint present in dramatic only -- and know, of late

a details than we're time position standpoint. both later-stage we move in a assess of line and safety this kind of But far potential from AbbVie efficacy as assessing to how on other can position any or and in not just as development. us into to puts you really give good the a sense give that data, it that any to more So

Operator

come will question of from next line the with Stifel. Samimy Our Annabel

Annabel Samimy

follow-up Phase dose a X into the and in dose X DMD selection the III. program for on Just of the moving terms between

mentioned bit safety dystrophin Just possible guess can aiming trying You looking as as level broader as you're for? the with correlates more data. I is you potential whether for far to But as a a better swath that intact. stays expression reach that population long as high microdystrophin benefit to and functional granular, determine that with just levels you're little microdystrophin of there as a get probably levels

So that's my first question.

Kenneth Mills

mean I a great it's Annabel. Yeah. question,

in resources, And we're for that's a think at fact on we've thinking to guided how point the pivotal several we're I drawing midyear terms decision right, of about dose different us. this determination. that

in range by levels signal And that impacted And level I think in microdystrophin in wide we're a we've of older of we're at dose excited. all seeing seen differences, that we're X. I mean, seeing I explainable.I boys, First, additional a range currently some we're there's kids, they're cases, think in evidence age -- some wise. in mean but there disease. progression boys

something forward studies results motor within the dose at these see function with basis levels.So in we already level microdystrophin X and and the of -- that's We're of accelerated X-year-old are have important X and strength XX-year over a is case. range the particular forms evidence in X as very that approved we product on dose seen our we had level look the of We separation. again, see last at We level enthusiasm preclinical at year well. preclinical data, old improvements level a things and and a terms we and dose in So sponsors, dose directly seeing the be comparing of of mice, types lower in dose high other that that may brought boys the MDX they've boys, this some level of well with That where to-date. old X. X reran cases approval in other level X-year and will better be in the other

X of because I as us be evidence reported and going have we'll level extent going the that expression think getting function.So us in and data that help directs contributions that to we'll improvement dose I important safe, is in and naturally it that, starting away be expression middle that's step us is more think dose we to why has to more going get want going entire to And next been so decision. shape expression, to year, other that that, that out expectations to to week keep be the just the view are well, if it's more observations and expect of be, first going our playing level get the we're of sort continues, to to in more from to to-date X data hard which is to into

Annabel Samimy

there gene doses move something forward that in not any X therapy? with that is is And done Okay. chance that's you or

Kenneth Mills

course, X ATMOSPHERE well says other probably says so No, functional of -- retina it's to we is for the -- single-dose good we've turning the for can that place that of well expressing, as evidence with and really evidence provide the right but the and more not anyone in X, I it's I the something the think X in as think purposes that the done greatest to strongest proteins some development, only on good. retina that ASCENT that as on, think reasons safety common and evidence of evidence we of focused it masking will trials in phases of be the can we've and with or rare And settings. and gene as MPS But Duchenne, X. benefit.And now not be both reasons, doses respect been II, preclinical clinical in themselves these diseases, do but I

with observed look a dose-escalated, that dissimilar the was -- were things I for took with into there. pivotal to actually XXX we us result respect highest dose safe at where that's we the expression and seeing So ultimately not achieved what we dose to pivotal find

playbook I think here. we're similar a following

Operator

of question Baird. with next Our Brian line from comes Skorney the

Unknown Analyst

just get on And color commercial showing label? think treated It those patients Brian. this some on as -- could the between the affect XXX. gain be skills. are than your in great two [ well? at ask expect to was How the below skills question wondering, patients or the this patients deviations of greater equivalent a those in I will terms to is the Do was plan? of about difficulty I just looking would Charlie of about that. -- age I be for affect less you difference you wanted This who ] do mean standard to

Kenneth Mills

that just age the the in able at the brought question the great and certainly seeing investigators stratification. a spotlight mean, interesting it think that little I had We We've and kids disease in exclusively about data the a answer to true, we're that, and shown decided what more over care had we about we're -- do the forward also that's the and that way what And point be think for to sometimes are improvement structured indices analysis Charlie, within terms about mean, of present on kids the bit shown seeing even expect had setting commercial differences.What expect it's clinically and commercial of deviations where that's well. that of Hunter not there in that rather, we've to form. are what not is these a and the calls -- depending can the about shape publicly sleep is I but in and way, in what in all on and about can see payers setting from going journey in And are with it's in and disease and ] we families things things is partners. so be children. a XXX when today. assessments gene diagnosis engagement terms as that and than that we see be setting time to having you that with improvement stabilization that and call X saying and that is for the this we for having clinical toileting, not trial take on is functional the -- when families would view or boys recently really place usually, with much are it's are think age of different caregiver younger. going finding disease, in When progression stabilization we've family, And away we've is is to in impact families. progression what access potentially think very to are type the sort but type in other of any neurodevelopment.So one say, discounted thinking step and because that It evaluating under having even standard those transparent we've outcomes therapy and part there talk the Yeah, and their patient I also, in I patients be of benefit comes parents commercial on to age, of [ far. to I behavior out less we the the is on benefit age-correlated, families their impact things mean.And of yet. are rare leaves you right they older of what this two kids have reported conversation that's and among to available I of we than like to in to They that getting be Duchenne are any I that to seeing for even now for I a patterns, stabilization, different the program at though kids forms valuable impact we're of a bay and think is home, we that dose that

when And those which that the to part from creep comment. kids, the I from of in something the so the of time. label think enzyme stabilization replacement the I So a strongest is disease going earlier the interpretations skills and neurological it in forth.But to motor getting literal there's the lot of the think result of benefit younger look, also comes of behavioral of, overall, I can development, development, and outcomes kids think, I some fine progression of -- initially, kids clinical gene may some are and therapy, of the consciousness it's therapy that think also on it benefit commercial be over is into to that going discontinuation basis not that may while

like a unmet to a is XXX I across of So lot support clinical data very with strong high boys Hunter. set feel need

Unknown Analyst

Yes, data definitely. great. is Yes, the

for So that color. providing thank you extra

Operator

the from Brill next come James. line with question Danielle of Raymond will Our

Unknown Analyst

is This ] on Danielle. for [ Alex

that workshop last question big biomarker interpreted subsequent kind picture of week again to Curious about the on DMD. better considering know their able X you potentially the limited regulatory Might about at therapies that a to to gene years in therapy label a some that trials? patients, think studied of a just they FDA the being expansion old, to Just and population needing comments to in get may be [indiscernible] placebo-controlled made how imply impact that X on not future.

Kenneth Mills

like a sounds Almost loaded question.

XXX, additional I conversation. potential feel mode can I look, with a we the in now we just And think is what right then think we with and where I beginning like there. to the additional data, see are think have can we're -- ourselves We're we updates into move really here.

precedence it the have an AAV from of can can have that's that something different opportunity in gene work as agency in longer-term to I innovation.And surrogate we from of the of think that's variety you're what an the the adding We innovation the we C-terminal in been going a mean, AAV to but And of perspective. important about that up therapy. development have therapeutic think I the we that of to mean our hear, the differentiated lot continue think see a endpoint complexity to the what approaches case also AAV, what's need think biology.We in pause is and that's what in of appreciate regulation ways that for kind on a setting stakeholders things outcomes support by perspective domain I taken from and and and

useful that data boys, we're plan that to that within all that think development our continues think we including conversations that further after So yes, evidence and to us and older about may that showing regulators stakeholders, think and innovation things I that contribute to in instance, approach is help how improvements. to our steer we think for that be as community when the come information important. We and

to happening, conversations like we it thoughts as in we those our and that are those are hear direct interactions. and that So happening well feel

Operator

Luca Markets. Issi from line will of come Our with Capital RBC the question next

Lisa Walter

This Great. for on is Lisa Luca.

a on Just couple MPS II.

regulatory I is strategy. you So accelerated know a physician approval. for reduction question that biomarker X first, at reiterated ] a months on [ your have

the II/III than standard to the know versus study, as we is Denali? to However, has Phase same CAMPSIITE Phase Is any a Xx ERT to run I Xx II. which opts your as there FDA a And hold that longer and the trial head-to-head follow-up. have chance Denali RGX-XXX bigger

Kenneth Mills

for and have while addressing And their months is delivered that this And target in dying of our means and as neuronopathic we're to a unmet for The the kids mutations. MPS is the cases, II high are characterization.And of always basically Hi from forward for brain with they phase trial. get that no a saw something others we development.And what in publicly gene to old working accelerated growth who with the occurring Lisa. or level things product enrolled a a in comes of or well the I'm genotypes marker, at openness Ultragenyx the substrate looking advancements Denali address complications a disease, that's ELAPRASE look characterized address Hunter that of use the were they're to We're achieve for to at treatment with I I approval. young, still we add.I'm not we're all be disease, that looking the RGX-XXX and the their of to exercise with and of this, that deficient pretty led week, the enzyme same in to and has meaningful of them syndrome. treatments just I of label question. we've together case, the certainty progression to way the the development that non save and approval real Thanks siblings of the including that's ELAPRASE development sometimes have on pulling growth meeting, real constituents those conversation is Denali last of which and in And use the and the just RGX-XXX. there. all boys for we're not setting accelerated think natural of progressed on expressed back and other FDA biochemical really of dose the is for. pivotal but in have top some on the selection us has looking reconstitute should pulling function MPS' to they see from benefits execution and from that like need mutation, with kid and also neuronopathic for for pivotal stabilized for of pulling that at in normal in IV in but I it on focused where for to CAMPSIITE either focused us kids biomarker the lobbying neuronopathic And I profile dying, for clinical think specifically the neurological in them that places only are pulling demographic And the the path that's

the does all sponsor across FDA the what So we control programs. don't different

of achieve product other something a for with we're biomarker on when feedback I approaches in going to And an a that. that using we've is, as assess be file of different our a design BLA comes we and set an types and that focused approval, show approach possible that achieve and part program they different is page and most support different a biomarker communicate of is what all that's encouraged quickly along, as they're excellent profiles, to a to people, well. using to comes the products, other think like position of things. it can we on as looking the therapeutic we're and candidate very frankly, that, endpoint, trials look, where all change to observational in about also the hope between here.So think with our programs lot a different have same benefit, really But when there can to it differences I think accelerated MPS with XXX it as to II, surrogate we're disease are tried in to the things

Lisa Walter

biomarker have or data Got Got as From do follow-up. NFL share [ additional any systemic ]? it. helpful. you helpful. just such be to GAGs color Super CAMPSIITE, a And Any of it. plans would reduction

Kenneth Mills

the that the the about useful II get we BLA on primary that completing may BLA. team than safety now the the on sort filing the right focused are helpful. the on. based to And really and this resource, endpoint But focus, that's completing of of guidance specific additional I data or any achieved the filing energy effort of patients all is right MPS of have now, don't We think other having to internally where frame of trial of follow-up things we be modules, the out design, the respect -- all the commercial to where what's support year. of focus half things in think to and into in second in and post more with maybe filing, are. setting I mean of I half that's second That happening get the may be on year something or as what the talk described we we defined terms start label we

Operator

question Our the Mani come of line Partners. will next Foroohar from Leerink with

Mani Foroohar

in pipeline. of quick on Obviously, there's digging been lot clarification. the a A

It like half fees define XXXX. back delineate you ]. but do of 'XX, out runway guys you've You in NAV your the press got of looks throughout financial guidance cash you as that that technology having number laid [ license platform milestones in the a release meaningful

guys financially result these changing what walk through on for flows year to memory that to would serves. out coming that in your accounting accounting called highlight from licensee events financial understand Novartis treatment trying you of and means statements this value you in basis. think specifically any ] you I or Ultragenyx, guys for [ you Rocket, Could cash programs? that individual an Just the or and carried you

Kenneth Mills

certainly, guidance there that potential the events.When but RGX-XXX. technology with all upon a quarterly approval things of to question. great Those around the are history event-driven, -- the hundreds of of in balance those have on of And and a start sheet platform suprachoroidal section as our over at of obviously, pointed course release to those annual potential dollars that with existing licenses, of impact them have in of of think our press of Mani we the millions company impact it's then obviously, achieve of priorities sales been the see occur they're sales. to pediatric phase major things And the are we I think mean, to the continue I the execution our been in review NAV and transition individual we and potential voucher milestones most Novartis the I the interesting look the are terms AbbVie. we associated timeline. for to the into on pivotal the are things report

some over for achieving changed regulatory we of of event-driven As labeling product that reporting guided instance, programs data to it point, progress, terms we're monetization and or to year, of or thoughts so really a balance this we Zolgensma are next continue likelihood in to not the milestones of bit since the collectively might the in impact of confidence talk of limited I near-term. to and of may royalty additional approach, three Ultragenyx, about course approvals.And approval two out any for include over to us on of the into intrathecal year planning the That the the we've pivotal and When the impact even the that or expressing give the products, not evidence or Rocket this those that could they the evidence seeing XXXX. our into product as being identified certainly of course change Zolgensma did the and could sheet think things but have phase about the occur comes the see in change those they've which discussion XXXX. that availability of AAVX again, guidance SMN, pivotal change.We're more that

milestones partnership the that's you generalized drivers our the of are maybe don't at $X.X be billion with know, types a level though, don't of of that to when So to that of and at and milestones XXx PRV the of and those same technology than I less licensees associated on that I as we've don't could I necessarily that the in AbbVie might the thinking be But the basis, sort been how would NAV think think a on in types value. milestones. combined milestones about licensee licenses I exception some they suprachoroidal Xx identifying this. the an those Is Zolgensma be same helpful? historical think our would of platform benefit in focusing that intrathecal or progress things look range, something be,

Mani Foroohar

No, that's really helpful.

Kenneth Mills

Good question.

Operator

This participating. you. you concludes Thank today's conference Thank call. for

You may now disconnect.

REGENXBIO (RGNX) 27 Feb 242023 Q4 Earnings call transcript