RGNX - Earnings call transcript Q3 2024

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Operator

Welcome everyone to the Q3 2024 REGENXBIO Earnings Conference Call. [Operator Instructions] Please be advised that today's conference is being recorded. At this time, I'd like to turn the conference over to Patrick Christmas, Chief Legal Officer of REGENXBIO. Please go ahead.

Patrick Christmas

Good afternoon, and thank you for joining us today. Earlier this afternoon, REGENXBIO released financial and operating results for the third quarter ended September 30, 2024. available website is www.regenxbio.com. release press on at our The regarding include outlook product Today's will statements development our and conference call regulatory forward-looking financial to in plans. addition performance cause risks forward-looking words and forecasted of involve and and forward-looking may, identified and as such uncertainties These anticipate, subject such be to statements to similar uncertainties. words guarantees plan, are that from meaning. Any statements can of may risks are other expect, actual by those believe, results differ intend future and certain not will, should, reports These available risks year the on are on Risk on REGENXBIO's the of XXXX, and and the on Form with Analysis Exchange Factors file Commission quarterly Risk December report ended the and section and Form are described comparable XX-Q, annual full website. in Management's sections and Discussion which the SEC's Securities XX, XX-K for Factors of REGENXBIO's call date on XXXX, otherwise. Please being webcast. only undertake statements that provide new information advised provided update any call make as this we X, events future information, is November of is be today's call, forward-looking may the recorded conference on of and obligation call this to this Any on account and no we or we of

Actual to financial call REGENXBIO. of may In to the and company. President be does results differ any project operating turn information not materially. I Simpson, now over financial or addition, results pro provided and unaudited the forma of report may will that is preliminary Curran positions CEO or

Curran Simpson

for Thank today. you, us joining everyone, you, thank Patrick, and

this the to positive begin then REGENXBIO our our an exciting and another first financial clinical These from and to Chief program, filing had we also Steve Officer; quarter. continue weeks. Mitchell first key meeting prepare we'll in call XXXX excited to data as business syndrome is retinopathy, quarter newly for Hunter for the remarks some with meeting in quarter Chief our month to the before franchise. several from BLA our before Medical the provide the closing potential which by review include We've I'll Dr. productive generate first our and clinical diabetic to provide Financial will and coming We're partner later important was and to as RGX-XXX highlights also accelerated year-end. our refer II, functional Pakola, will is fourth the XXXX passing End-of-Phase expected milestones. retinal this very time gene today for that and the Steve on the for Duchenne Recall milestones for have therapies. are our which in expected have MPS upcoming best-in-class then commercialize call recapping known multiple results update open programs AbbVie, II Chan, On Q&A. This who as me quarter up Officer. I will Mitch, coming initiated a for DR. data very and appointed by

programs, great You'll RGX-XXX, progress Duchenne. I'll therapy DR best-in-class AMD. Diving gene start being in wet in more about our continued RGX-XXX, including Steve potential and the into from hear next-generation with our progress made for

the therapy to box and from clinic Thus demonstrate differentiation. consistent level later, profile, We've on month. We a had begun, and for SAEs expect outcomes, has the this discussions RGX-XXX We're that share FDA will of compared check to approval points we patients favorable early and videos. this pivotal and are to imminently. study study we no of sharing have the to functional the seen drive functional the more functional from the demonstrate safety have year, as I expression. The remains shared checking next-generation and our we Once confirmed to clinical far, only excited accelerated Earlier to devastating we disease. to to pathway soon. this benefit robust all some RGX-XXX is encouraging us. begin at will patients RGX-XXX mentioned, to ongoing this as last AFFINITY initiate DUCHENNE potential the advanced increased data therapies, available phase pivotal boxes which, caregiver X results, observations these date that have to gene meaningful X will which dose be Phase open to the phase time productive in very with include data first continues III microdystrophin

Another of be is that can manufacturing the commercial fact to when RGX-XXX key are comes key element today, believe, effective we driver an of a it which differentiation, we commercial ready launch.

suspension-based Our manufacturing as are productive. and that therapy RGX-XXX to gene aggressively development very highly accelerate a be best-in-class potential excited to process uses we about potentially second Duchenne bioreactor to is modern Needless to in a say, market. continue

infrastructure RGX-XXX MPS development. MPS program of now for first benefit major for build neurocognitive is the onetime those Duchenne, in in for the RGX-XXX a programs, that significant in regulatory furthest cure line opportunity Let's submitted for BLA to milestone in is a accelerated and RGX-XXX, marks It product the pivot to Also boys first BLA quarter II no filing other the pathway announced, as are our devastating rolling II first marks we as PRV both a address we occurs and the syndrome. currently milestone and the initiation we complete there Hunter XXXX. to first and major commercialization. disorder. for XXXX, REGENXBIO receive and of represents our also progressive or expect whom upon our planned as was like to a no treatments commercial potential pursuing RGX-XXX the in QX will Today, of The Like is to treatment an for a approval. Duchenne, approval BLA BLA decline. would commercial under on gene therapy and module patients a track be

franchise late-stage we RGX-XXX, are developing I our few the make retinal comments a with AbbVie. partner, Lastly, will on

bilateral across hear in study multiple where population majority you'll great eye news from the As we The positive recent patient fellow AMD, of are half making the we DR, large disease. trial DR, expand And to an the our In wet opportunity commercial XXXX. expect from global encouraging. indications. Steve, offer first patients develop for data opportunity strides in and another to the pivotal continue progress addressable making we initiate is in highly of eventually RGX-XXX a

to for worldwide. In we will studies. are tap AbbVie in continue of are that soon III to and both turn and the supporting with making that, to results and AMD to the millions loss DR potential see like progress update Steve? excellent work our Phase to prevent of we We over summary, look look to excited programs, wet to have weeks programs. now We forward potential continuing the to immense sharing several clinical for call clinical novel on catalysts forward programs. With these coming RGX-XXX would an Steve in vision the important I

Steve Pakola

with you, dose safety, potential XX. progress with the evaluate RGX-XXX, of Duchenne patients escalation of I/II the Curran. multicenter AFFINITY Thank DUCHENNE Duchenne. trial. efficacy I'll and Phase X a to aged to and onetime start we is for RGX-XXX announced This therapy gene clinical exciting onetime expansion a tolerability of the a in study clinical in Today, treatment dose dose

level that to of pivotal have phase, X, pivotal dose dosed is level. patient I'm the the expansion last the we which in share pleased approach we dose As cohort

age data, to investigational therapy. our the cohort. represents the a to the we DUCHENNE years old. prevalent functional years. a company of REGENXBIO strength group group this access continued patients cohort dosed aged this sharing is In under recruiting in we first new the gene X data include And initiating announced this to expanded to X patient from the We study forward X has of and to patients U.S., or the yet the pivotal program The weeks. today, been the that portion trial population, has of coming AFFINITY X significant the On first approved look only no X in

vision updates II blindness DR is a I'll ALTITUDE working on in adults. being being AMD, and of to treated collaboration developed trial wet appropriately. AbbVie is not using Phase our AMD disease progressive administration. if RGX-XXX, delivery. and in-office and routes blindness in Now is with Like retina of via start to in with DR evaluated suprachoroidal that treat leading subretinal RGX-XXX for cause from franchise, age wet It loss the DR causes suprachoroidal a ultimately, which

we to partner, shared accelerated sharing soon. trial End-of-Phase pivotal proposed meeting the AbbVie, with with program FDA this completing to have forward our we've the and look As II quarter. We our meeting this

of therapy well first DME care of more RGX-XXX previously the a regulatory program include for to vision-threatening tolerated DME, the its study wet with same pivotal trials, of from well we disease global evaluating X global This America, become We macular RGX-XXX being study subretinal that different for no Phase as announced bilateral of potential are initiating treat trial expands routes multi-indication the AMD, ALTITUDE via half complication In submissions potential North potentially a patients was evaluating expanded to positive well positioned news and broad patients patients. AMD. Within our with Europe retinopathy. remain in than with wet As gene ALTITUDE treatment dose may disease. The standard million for further trial and have cases fellow of These diabetic is in RGX-XXX cohort the bilateral data a global delivery, the trials suprachoroidal. subretinal, to we kind, with Japan. eye worldwide. RGX-XXX II XXXX. X potential to in already subretinal planned the we ongoing first or with and the onetime evaluated to is Broadening impacts recently edema inflammation. eye the progress continue of ATMOSPHERE diabetic recall, new and of the patients ASCENT benefit XX at more RGX-XXX diabetic exciting demonstrated patients pivotal of intraocular in you a by patients

to of on including seen like reduction of plans burden of a patients sustained difficult-to-treat to were high with the vision patients eye supplemental also programs. including treatment continued wet representing in injections. progress of completely eyes. while the Notably, continue our injection-free in at in safety encouraged meaningful for significant we XXX% see positive highlight with demonstrating Overall, by results stability supports programs. study were inclusive the for patients fellow to number months. impressive XX% X XX% observed, We in a and our option bilateral these RGX-XXX. The profile I'd needed burden a X treatment And retinal particularly prior or label control. both be suprachoroidal X AMD patients disease use, efficacy,

treated setting phase regimen shorter in significantly office, therapy X-week than course and are confident patients the trials. safety We a advancing patients gene profile, because more meaningful in a profile, used safety drops, seeing prophylactic potential those the of particularly representing other a for differentiated treatment we're eye this than physicians. short of pivotal in In steroid XXX in option

this of sulfate Finally, DXSX, on RGX-XXX Patients CSF Earlier that for trial brain heparan pivotal being primary levels attenuated endpoint achieved treatment key of CAMPSIITE MPS developed significance. activity with announced to RGX-XXX the statistical of disease levels. disease year, we met biomarker below high with the a decreased its treated II. maximum

as the BLA accelerated reasonably approval is DXSX predict endpoint a surrogate to uses likely and Our pathway benefit. based clinical on

We pipeline. be disease. patients reported enzyme conclude, across families progression the address a and can this to the therapy make gene treatment replacement address only RGX-XXX directly patients replacement is we is updates today XX% positioned significant for and that decline. To dose also that treatment of trial to neurocognitive enzyme previously discontinued does remained onetime neurocognitive the syndrome pivotal progress and received continue meaningful not programs data is all weekly with naive. therapy update option decline that first who whose This Hunter well or our for in of therapy

and review like been the Lastly, turn with representatives clinicians thank trials. And Mitch? these over Mitch to advocacy to families, the have in and supported our all who financial I'd call patient that, of I'll to patients, involved guidance.

Mitchell Chan

September public XX, December equivalents X expenses $XX offering offset by the ended for was XX, operating million on cash and $XXX used March the securities XXXX, The in prefunded of common XXXX. you, as primarily during to third $XXX net Steve. the completed REGENXBIO September proceeds quarter stock in with million R&D fund September months of and cash, to quarter XXXX, upon securities any Thank RGX-XXX fund XXXX received and $XXX million an of upsized XXXX. were compared XX, marketable its primarily activities. of achievement its excludes the based million of This marketable for cash decrease driven The warrants by activities compared million partially as of and ended from the commercial XXXX. is from in headcount $XX AbbVie XXXX, of equivalents payment plans cash runway cash to decrease due be company's guidance was XXXX. received million to that third cash balance, of of or collaboration. impact XX, may $XXX operational and decreases the operation REGENXBIO milestones quarter preclinical expects our into on development current under the to

Additionally, for call guidance monetization RGX-XXX. With Review turn the a back received Voucher to runway be excludes Curran. will of that the may our potential that, I Priority

Curran Simpson

Thanks, Mitch.

to QX RGX-XXX As year, making heading are complete in year, DR. we FDA derisking each into the of first our of and way and design initiating trial pave global for collaboration you with initiation This for multiple trigger an under plan momentum in late-stage meeting multiple of will forward End-of-Phase pivotal accelerated commercial sharing the RGX-XXX first both opportunities the pathway and we our this of with strong events. To II progress AbbVie. Also our milestones heard, the functional will and data. on in QX we in in approval trial programs of for recap, significant half first a advancing the and this in year-end look trial XXXX are our with pivotal large

been If therapy the receive point continue the progress we of to approval. for complete very QX the Finally, a for this and in submission and the year has will meaningful RGX-XXX upon approved, devastating a treatment for expect first BLA turning XXXX. the with of disorder PRV gene for submission regulatory families Hunter syndrome, approved development become would It this us. rolling a would and patients living

in and to As opportunities. late-stage programs we differentiation treatments demonstrating onetime groundbreaking months on with this represent And year the positioned care we I'll to today. standard and beyond. With to our transform over new the against of trajectory look of and potential time for see continued turn need in that XX and thanks options. your of toward last Each management the of achieving that questions. in disease multiple next treatments. underlies beyond, The to of the for the momentum forward for look deliver better is milestones Operator? patients important a science quarter the is company available programs and our everyone call we that,

Operator

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

Unknown Analyst

for on questions on It's DMD. [ Tony ] Quick Gena.

durability may are accelerated point a we if approval, at how would biomarker data change? age be points any X-year-old For needed the for there approval cohort, -- think patients one about tissue? And the for to are X-year-old also muscle multiple of time months? biopsy the growing and still year-end, on you concerns versus magnitude additional X young just at then functional the there given receive be then all do time should And

Steve Pakola

Tony, Steve questions. here. Thanks for the

considering we course, because of see know effect kids of lot have that of opportunity X-year-olds in cells. the because sustained for we the I'll We're of proxy a terms excited potential here issue the durability So liver vector divide. comes your actually AAVX, durability actually activity. we dividing hemophilia a now go out XX liver-directed how topic to young is start have replicating can cells AAVX for quite we these question when up where the And cells beyond about with Yes, we very about years think the treatment, for our X-year-olds. field of of a dilution and in you it of

confidence unmet because a So lot currently no major age gene therapies as that this need for certainly, here a gives there well. And here us group. of available are there's

Curran Simpson

the included biomarker, biomarker data other have in we requests your don't any additional question for around what's beyond protocol. to Tony,

assessment your effect, So there. Peter of be wouldn't people we be additional the -- even from anything approval, about magnitude to consider And Marks' for what to I for there requested answer think expect question benchmarks I effect pull of certainly, benchmarks for ELEVIDYS. consideration adequate are to including can effect of magnitude

terms super that think higher data just sets we're programs. and data about will our biomarker coming than be excited that that that in we've shortly got upfront microdystrophin the I patient older how population X especially reiterate, precedent other think the the levels good program. And we're so for the considerably we're And to seeing, of by in and I a of looks thinking

for thanks So your question.

Operator

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

Gospel Enyindah-Asonye

This is Gospel on Vikram. for

one have month? this are expecting for We question best view us the data functional to on on Could recap you gauge we your RGX-XXX. latest later how

Steve Pakola

We're to. forward question. soon to is for very This present Gospel, excited we've be able something functional thanks the our data. looking been to

lot space of I look of lot think the learnings get precedent a totality come a at of to benefit the data. and the the of we we with where in

a are time We'll So have opportunity of, that suspects And you'll has at also well walk/run that at certainly to meaningful. are as which been tests be look lot valid, outcomes. usual or we of as function XX-meter stand, the looked look the which at. historically caregiver-reported to such aware the measures NSAA, timed know clinically we

really So different these it's doing a lot chance to assess look to of for see at how us benefit. a ways boys to unique clinical really are

Curran Simpson

think to analysis. external But would the point I mine history Gospel, as what about look individual include the We to thanks at we pretty our to substantial that ability look question. data. patient is part can databases at also as for and And expect of to natural controls our

Operator

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

Mani Foroohar

little more detailed a question. guess I

being have will of quick future, Or first we're going a not expect that future handful rolled it follow-up. these And group and recruited set? for see experience a I a of a and set data will when pivotal to include the data this from to including initial pool you their patients? follow-up into be So data in the is about reasonable collectively patients think pivotal data patients, data larger of

Curran Simpson

same being Thanks particularly as pivotal will point Phase in assessments development we're of patients about functional is the quality I/II dose product we that and patients for data would expect I/II will study, that that excited the was say doing in level Mani. our pivotal Phase question, the those to most do to terms out dosed we're I the is what for for that from were area of use one -- really through And Phase same at think and we're the Yes, in development. transitioning process be doing certainly X. accumulated I'd pivotal. that be I/II I

if And to Sarepta, see the for they the and in middle major process pivotal so you back of their change AdCom A of comparisons think process made you program. And a B. process that. in the think database well profile our throughout including the We any And because patients those of won't is same will I study. terms product the work have of that in could safety

obviously So as to the as about term that, we'll recruitment, near part we to us well. help very do pivotal to will of and comment intend details with them Steve leverage that study. and talk it over turn I'll of further the

Steve Pakola

to patients ability the is the Yes. one dosed already to primary further accelerated approval the endpoint microdystrophin. use wanted that I/II I supports for on just elaborate aspect in is Phase

we'd makes that the data future in it's use the pivotal. X, level it particularly we in dose the that in natural that So have, addition to data patients that the that with perfect sense able we enroll to be quite

Mani Foroohar

ophthalmology hop over that's to if okay. I'm going follow-up for to helpful. a That's

would we for onetime us guys on detail, suprachoroidal achievement of time of patient through dosed treatment for i.e., and clear might up your cetera, up flow et following balance through when own see would statement, Can how milestone statements, first DR. to that sheet be a show sizable including income what pivotal have walk fairly in chunky they the meeting versus on modeling? financial milestones just You our you the End-of-Phase recognized, line II those a delivery disclosed they some how

Curran Simpson

turn I'll that Sure, to Mitch. Mani. question over

Mitchell Chan

Yes.

that onetime where in P&L, this nonrecurring could the obvious there. payment show can standpoint, milestone the recognized purposes, reasons, So as the balance from helps for And actually for sheet. for modeling Mani. that a Hopefully, will you That's potential payment account that. up for be actually onetime

Mani Foroohar

So quarter this will all something received? the -- be over will all same time? recognition in be ratably recognized the Or is be as that recognized will

Mitchell Chan

going receivable. it's to upon be No,

Operator

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

Jack Padovano

is on This for Jack Annabel.

mentioned for be on microdystrophin that RGX-XXX, that though cells pivotal expression previously might something believe the your trial, were even a considering require. you've So not you agency would might in you minimum of -- necessarily imposing that I the threshold

XX% that like think I And Is approval. the the feel doing? that have still ever maybe you that in you with number confident expression discussed FDA? was something for something

Steve Pakola

that to to been in we're clinically relate I highlighted, threshold very near out our the Curran design pivotal is as details the meaningful be about potentially coming has to is going -- What the something amount. future. now of what considered future of As say a can with in a think something that field in would concept XX% roughly

we're Fortunately, that seeing higher than microdystrophin levels are much level. that

So of think gives when flexibility designing study. lot and of us powering a that a we really study a

that in So to giving we details more on coming look weeks. the forward

Jack Padovano

Got then just I it. one ask if may And more.

biggest of the around not with a pretty that hear we or from necessarily microdystrophin sample is the pushbacks but rather expression moment. RGX-XXX work small have size investors at we to safety, One just that

upcoming other data into a defining kind patients? a words, of translate functional only think general do might meaningful how it be So it's in Or the data, from that more in how well will the couple functional you of given profile population?

Curran Simpson

that relatively good I And think, limited we at a microdystrophin we're a we in have we're a And primarily programs the age. we higher reiterate a almost Jack, highly succeed looking set. data I data I pivotal is that question. similar have and likely sort a that patients threefold than levels a trial I this demonstration to conviction would what product, efficacy. do other in of is that have for differentiated think fact set that think of that's for But

bring think, us the and patient come to interest community, in with enroll allow will especially really factors functional data in those even a quickly. of to, program think I together I strong All the limited

are, our around And a think in and we about really being anticipating able where data. just excited so eagerly share functional I we're the results to we're few weeks

Operator

next question the from of America. comes Alec Our of Bank Stranahan line of

Unknown Analyst

Matthew on Alec. ones for us. ] from [ quick Two

at redosing? the AMD, impact, of for about into DMD, all then thinking can concerned you market color wet about about going at And potential the For provide all durability you on biosimilars are and patients? are younger think you from Eylea And especially some how Amgen impact? you

Curran Simpson

that. Steve let I'll take

Steve Pakola

Thanks for the question.

in the that something that So aspect organs touched have over that the patients where we serotype we a younger of was there in is durability and on time dividing evidence, have not earlier cells. call have dilution you in the even our with good effect you durability particularly

that X. of one X need to excited go age So of us that's the this after unmet to aspects group made

out patient also focus I available now, time. that we the therapy today going our treatment is the the get to gene Right time for that's over going dosing on able that excited time evaluating treatment to there Redosing, that population in onetime that with to that's be something -- first even with very treatment think, first options. isn't for So announced cohort. of patient the large some field, be is

as after could to us to have the there's mentioned. go for certainly, that redosing. just ways expect I we durability, We opportunity good But explore

suprachoroidal able meeting to the being aspect advance into finishing pivotal -- What where, we wet look was the advancement development so AMD, AMD the diabetic that programs just we excited ask if II we're discussed, collaboration with End-of-Phase as AbbVie, wet forward our Lastly, the about on program? could I in in collaboration AbbVie other with in and and retinopathy what was subretinal our pivotal there. other with have also very to

Unknown Analyst

Yes.

about Eylea Just how thinking from... you're biosimilars

Steve Pakola

Yes.

that's aspect something impact example, So some of time, see has branded on, starting of products. longer biosimilars the we're been for been actually biosimilars to around durability over We're more no seeing biosimilar in. the and come

that patients So terms value impacted, the seeing. of are benefits shows of durability not and if seeing clinicians we're faricimab, proposition you in which high-dose think of or Eylea incremental really those

so injection. And paradigm need no even the only that burden, potentially for onetime would impact prevent bigger injection a we shift expect not for even an of would decrease but treatment

Operator

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

Luca Issi

bigger was progress. few of presented the World? no Phase there. data there Maybe, on Congrats show a functional there, benefit weeks Steve, Pfizer back your to at by but that evidence reaction whatsoever they Great. to III me It what picture, pretty expression the was looks still, impressive

there time team you is your decided joining Mitch, you maybe, attractive I just why believe this call. what REGENX, regulatory first changes kind on standard that a Again, on maybe diligence decide you're versus were forward And the put to any in of expression to the where is do the scenario earnings data and you then there, about light little FDA talk thoughts So here here? you Can doing join the as going that appreciated. much a emphasis function particularly maybe find of it? more about

Steve Pakola

over take the first Thanks, part hand to we I'll Luca. before it Mitch.

Pfizer the results. So

you from but signal there's as of efficacy expression, microdystrophin mentioned, their trial. So a clear not

of we terms in exactly of As the what our elements. of And actually molecule why right designing as means this beginning reconstituting focused far construct we part on is can as dystrophin closely the best we concerned, is we're as the from could. the that key that functional native program

elements So that's C-terminal a a some key and important construct reason why second-generation includes to develop improvements. our and example, other team that, domain for the time we the took

are is really, So the constructs created overarching all not equal. aspect

translate the into that from confidence have the less domain to FDA that benefit. fact bar. importance you're the that elements So the some the that acknowledged and are farther you those for ELEVIDYS, in in going for takes dystrophin, expression can constructs the the predict away clinical absent example, of the as confidence well being to likely And they native a you away reasonably where C-terminal raised terms in of AdCom

as the that discussions close how seen are program. as recently certainly, we our think in those we've key aspect have FDA for the terms in for no we differentiation to our and of predict product our change with And reasons, meeting. preserves So we End-of-Phase II our likely with the our benefit elements reasonably

how we're with aligned on So proceed pivotal program. the to

Curran Simpson

is would at Curran. functional this if you assessment. -- that thing Luca, to guess Steve's look utilized that one for I ELEVIDYS, I accelerated mean, data this approval the they add comment And I in

it see all supportive want a primary as maybe So at not on us FDA the would data that won't surprise but functional side. endpoint, to

So approval, obviously, see questions we're those I eventuality. now that the in address we're the filing at accelerated well -- think of market. time And for for uptake prepared for we planning good to and

demonstrated lot to we're a data what given general, I'll to be and differentiation Mitch. turn in looking we're over around is of microdystrophin in our of think levels interested I patients being program that showing. for the it aspect with are that the functional certainly going

Mitchell Chan

BLAs. as say, Thanks, the Yes. company. been of And to opportunities Luca, the reasons our true. must the one why be are very least. other brief XX months, well, And time to translated call is XX the the here is very scientific entering for And during several the And really there to for exciting to joined patients. great this aspect phase think And my I BLAs question major that almost great I've say deliver over to could the months months. medicines that here, company. potentially are at let's just actually simple next, These is are it, these has I much X excellence I And happen too. at commercial

company. if for best-in-class, opportunities potential blockbuster are these even not So this

deliver value here, at is proposition I'm and to and teammates great which I and why pivotal too moments actually great hence, I'm execute towards these with here, company. say would the by the surrounded pass So is that, could

Operator

Goldman line Our Sachs. of next question the comes from of Choi Paul

Kyuwon Choi

latest potentially what on RGX-XXX. a I latest, the of doctors secondly, your drugs? to thoughts are for physician -- and then to guess, want AdCom maybe see want community and I are relative And are to the sequencing these gears what a switch the feedback you're the about following thinking filing. asset how I Denali just And hearing potential maybe bit to

Curran Simpson

call. Thanks, joining Paul. for Thanks the

no on out for there AdCom plan be AdCom. the we're by that will required, this filing our to at their until FDA we end, on question, first an that they an that if But map point. we're the think I you're have but be it's planning preparing say notified accepted could certainly required is normally information something That's not review. and as

to I difference approach our and I a think second huge your there's question, between think Denali's.

meaning be require therapy go program. our for that's think patient for weekly that inviting the a onetime extremely a a doesn't IV to and would is thinking about we're gene I in how patients, valuable, approach incredibly

the to we to study on addition, on majority had of released the the before were that therapy haven't went that replacement showed data patients that back it. to on year that early in go they enzyme In

benefit that think more if there's initial even we product the you that profile of than had it way So developed.

and So as we're a position to commercial have data and our we the be hand. we feel that like potential in as for a based product strong with a position choice like we'll in closer we closer on to able get patients MPS community really feel the first approval

Operator

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

Unknown Analyst

for Brian. on is Charlie [ This ]

cohort youngest the want just DMD of to revisit patients. I yes, So

old in the do diagnosed potential X Just knowing market any make years diagnosis older old, there that identifying to until moving issues they're about the not efforts And they're you these X are that future general, there patients for average of relative in age cohorts? terms are any think of up? you having could years you to

Curran Simpson

to Thanks. I'll turn that over Steve.

Steve Pakola

the Thanks, Charlie, questions. for

the of that's seeing going screening. next on newborn see are So I up. time think point to years, over newborn progressively, we're take more and screening diagnosis, one we traditionally I think the states few pick aspect been And is more although

to move things patient we today earlier. first age that announced that dosed going that's group. actually in So We our

these does an actually So have. And the but important that the seeing to our only able these patients families anecdotal, they're sense inform patients confident be found we that investigators are the certainly, want there. potential compelling find the it these considerations be in once age consider families. the of we that that community, with gives investigators. a that's one as we've been And early options, albeit lot the feel to it and to actual families describing there has think out and the you we're patients best-in-class we of the these And going differentiating second-generation product well. data are aspects is groups, really a just functional like part the Not opportunity get reasons opportunity we us the when of a going to plays consider to other for that in

Operator

line Our next of Chardan. question Daniil comes from the with Gataulin

Daniil Gataulin

trial program. Can of from RGX-XXX. have RGX-XXX progress. the on the ] and package? trial One a pivotal start half. authorization some share in then on I Congrats on that questions. details that [ of of press that first release a in question will of data or Canada, trial approval couple the you in data clinical your I which whether be part saw got doing you the you an And on plan to

are recent study, the for the fellow Given suprachoroidal for AMD eye plans both both for wet -- what study for data DR? program, and bilateral your from

Steve Pakola

Thanks, Daniil.

excited in we're expanded for that we've the with inclusion Canada So program.

with details no to And there. So the we on when I and issues but with give details approval open to see the we're that up supports we've give the be consistency multiple quality going of the in able our heard we'll FDA. sites our Canada, overall which the had design overall that think we more product design, and from feedback plan getting we

for the So program more approval. RGX-XXX, the data. subretinal treatment for we not whatever also exposures, we be program we initial since far that the with also started be accelerated the any of more bilateral see evaluating really study, would be mentioned primary a focus as but before, from at, approval, bilateral similar but to pivotal the lot for both to a is had the lot we endpoint that looked as data going the microdystrophin, what region Curran discussed functional data relevant the in

be first looking we'll evaluate of Since to you're treatment want with it rapidly that So into AbbVie, a step also sense with advancing DR suprachoroidal. at that the to question bilateral bilateral with we're made along route dosing the great administration. have pivotal, it's raising

So planning we'll be that.

significant what because that's certainly, We're response that want any for immune at that excited to but do. seen announced type the details or in immune the then to that non-localized you eye, therapy, the there's of where gene systemic inflammation circulation, been you affect not opportunity if and there, spillover that's seen certainly would sense we a wonder, specific if delivery efficacy intravitreal response haven't will We would eye. the safety it fellow first yet, example, in the and makes we've with, seen of any have safety excellent

the significant suprachoroidal that's with we're delivery compartmentalized because gives that eye, we to responses it's not why So And able see dose any haven't. subretinal confidence in that us to where expect excited the fellow that first compartmentalized we in in fellow a eye. we're issues immune-mediated be seeing also eye and delivery, didn't also the we'll

Operator

from Chen comes line Our Company. & with Wainwright question Yi next of H.C. the

Yi Chen

With what the retinopathy, as respect plan can on diabetic without measure a program would pivotal you primary whether enroll patients to of lot efficacy in if symptoms? the the -- you comment to endpoint? you And so,

Curran Simpson

was it -- Could Sorry, to little plan? pivotal difficult the a to was you. RGX-XXX bit referring this hear

Yi Chen

would measure endpoint? retinopathy RGX-XXX expect the what symptoms? as program, for of without lot a pivotal efficacy to No, for the diabetic you so, primary patients you enroll if do And

Steve Pakola

Okay.

retinopathy, an diabetic and wrong, they point diabetic blinding So in this It's macular risk to conditions. in that at I complications are patients edema me they're asymptomatic correct pivotal our developing often yet like in excellent significant relate But think the I'm indication develop until diabetic this proliferative the retinopathy but these these -- if retinopathy. would program. diabetic of

ever And patients are that could not sustainable existing these asymptomatic. repeated therapies, prevent from needed So a is developing the are treatment because what's injections blinding complications. just patients

So treated the choosing be treatment to clinicians much. too burden are is and because not just patients

see opportunity So as significant is or it's onetime the one a we treatment. of such reason a and the AbbVie whole major reasons this in-office

patients So from to and repeated the prevent for a that treatment a help need that can them developing for really compelling onetime symptoms case be in-office does the allow their this treatment. clinician and

So patients. is measures where vision-threatening to already monitor we those complications whether reduction of PDR. for yes, then measures a also, complications, retinopathy study outcome diabetic of We their saw of Phase asymptomatic their in hard And would close at II severity XX% vision-threatening our DME worse. actual we include would in can of getting we look and

with able the So what being the forward II and that's pivotal into year. meeting to move us next so and End-of-Phase has excited then community about development

Operator

like turn remarks. Simpson, call now back Curran the I CEO, to over and for to closing would President

Curran Simpson

Okay. I'd joining to for us thank call. like the for everybody

a out of several through of to We to of reach milestones you. following you have the with as each those realized. a the really Look rest you. important of year evening. to report are great Thank forward have exciting and up and lot And excited to really

Operator

This your concludes call. today's Thank you participation. conference for

You disconnect. may now

REGENXBIO (RGNX) 6 Nov 242024 Q3 Earnings call transcript