RGNX - Earnings call transcript Q2 2024

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Operator

Welcome everyone to the Q2 2024 REGENXBIO Earnings Conference Call. [Operator Instructions] 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 second quarter ended June 30, 2024. development available forward-looking website outlook statements to and is call www.regenxbio.com. Today's in release regulatory include will regarding product press on at our conference addition The plans. our financial of to anticipate, These forward-looking actual to and believe, as identified should, from differ expect, and be words that forecasted can words may intend, results and risks similar those by statements cause uncertainties plan, meaning. subject are such will, other may, certain involve and guarantees of Any future uncertainties. such forward-looking are and statements not performance risks These annual on provide ] update which Risk sections make advised file with Discussion this SEC's Factors call and XXXX, risks is we future as are of on full Factors and Exchange provided is date and Analysis and on XX, described in on XX-Q, the Risk undertake information are webcast. and obligation the Form the of the otherwise. Please the or REGENXBIO's section today's we events, report being for X, information, December that Securities available comparable Management's website. Any of the and XX-K quarterly ended REGENXBIO's reports year conference of and for call Commission Form on we account may this and [ forward-looking statements no August only call, new this call be XXXX, recorded any to on

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

Curran Simpson

update and we REGENXBIO's be pleased milestones of my one sharing corporate first business today's of across and for for an and Patrick. number and happening key we'll of the the open each our of will we'll positive Vit Good At for you, make afternoon, significant with Pakola, Thank productive XX, quarter Officer. Today, REGENXBIO results pivotal up Steve as an Executive call, you our provide updates and exciting, thank we the our been Chief of update Medical the pipeline Vasista, leading Officer, achieved. begin the Chief half goals our our Financial end future will Dr. well have call, June highlights overview year second questions. It's of momentum of a a line be discuss commercialization. a Chief then toward clinical our financial Officer, as trials joining I'll that AAV I'm ended XXXX. advancing clinical our stage us. provide progress to differentiated Therapeutics. programs first recap for everyone, of on as as an the programs;

AMD being Our for treatment of the for wet syndrome. DR, priority programs in or RGX-XXX the program of Hunter ABBV-RGX-XXX diabetic treatment collaboration or treatment the MPS AbbVie with developed retinopathy Duchenne, are of for and and RGX-XXX II

unmet need the and opportunities current significant meaningful programs, lead generation candidates be accelerated specifically and large represent from product XXX, long support care, are differentiated expedited XXX approval due soon via can where commercial term. standard for Our our of and to value

share reach developments begin for to data. a me poised second microdystrophin benefit Steve its potentially is expedite more be the repair product RGX-XXX, itself. therapies microdystrophin gene occurring both are next functional product and AAV-based of exciting the to from the Let generation that number of in domain, robust which utilizing will microdystrophin RGX-XXX our with we as But a a of to the market. represents RGX-XXX. the reflecting the gene, treated studies which today, ages. expression region microdystrophin construct been details the of believe shown with contraction-induced will best-in-class product to broad for only muscle are an highlight the patients, positive differentiating across data its a we commercial demonstrating pre-clinical and factors ability muscle to make key development naturally about first range is want consistent, maximize a preclinical the potential the I progress reported the includes advanced candidate the improved that is and potential. RGX-XXX dystrophin differentiated It has protect shown excellent stress C-Terminal to of in and making product improve There

ages. SAEs consistently microdystrophin But ambulatory boys that expression RGX-XXX is high expression to seeing highest data. analysis well especially of risk-benefit for mentioned, note a has published levels ambulatory other is patients, and of which patients reported, to agencies And have been trials. in older the we're I RGX-XXX gene levels of reported is regulatory of where patients, differentiator it demonstrating in I and the versus As caregivers therapy Duchenne overall microdystrophin older no compared been element across want all tolerated other the and meaningful significant

next all towards U.S. and gene our taking in a approved are in our completed Duchenne II yields suspension-based a the as the End-of-Phase goal. using I in recently with is this the our be and produce doses meeting also plans commercial-ready At microdystrophin in-house we from walked manufacturing have approval. necessary away BLA confident meeting the of per As file of therapy meeting we facility, involved discussion and our We primary goal capacity X,XXX accelerated successful mentioned, FDA of steps this year. to to to industry-leading trial. RGX-XXX process the for endpoint The manufacturing NAVXpress this used

commercialization. this RGX-XXX to characteristics in ongoing program the are and the need towards Given the Duchenne unmet manufacturing our of community, plus well-positioned significant we differentiating advance expertise,

chronic made AMD. Turning have trials retinopathy diabetic in across advancements diseases. in to we several AbbVie, being and partner, the developed With ABBV-RGX-XXX, wet suprachoroidal our gene retinal our therapy

XXXX. using meeting regard First, is plans take treatment End-of-Phase This delivery, ALTITUDE the first fourth expected The the with the new with FDA. for the XXX to of timeline XXXX. trial year in in of meeting this now for towards the first expected are versus half initiation we our acceleration quarter rapid accelerating guidance place II supports of our quarter with the pivotal trials of suprachoroidal to of initial DR

of milestone ABBV-RGX-XXX is $XXX in in upon will the million be anticipated again dosing with successful Importantly, entitled payment XXXX. which DR, patient a to first REGENXBIO

the is the patients to well DME. We announce at expanding prevent our working become the care XXX global AbbVie, that broad will patients with trial retinopathy. of initiating of include DME ALTITUDE to global partners a trial and further of with multi-indication edema, with excited be positioned of by the standard cohort XXX the to potential are the for progression diabetic Broadening to XXX. diabetic we macular ALTITUDE also new in expands treat potential

progress planned gene of programs II strategic wet by in the positive program could Approval the excited remain result plan. to continue programs Priority on across for well on as of remain as with XXX for third of as excellent syndrome. We also and a number Review XXXX. we made all be initiate we We catalysts progress BLA and potential additional filing rolling XXXX. Overall, a have of in our in this Voucher the provided important on later in approach our AMD making becoming have updates on as year. schedule RGX-XXX to receipt Hunter first progress therapy are we shared track our approval quarter BLA the We for MPS a

I'd their for call facing patients We an life-changing to update, potentially value shareholders therapies great programs. Steve? while are to need. With bringing accelerating our now to update Steve the and pipeline that turn development unmet for on over expanding like of our the clinical

Steve Pakola

who and we therapy Today, the reported new Thank X received onetime data potential dose months. to control treatment years, and of X.X X, expression level at Microdystrophin I'll advancing the start respectively, Duchenne. aged XX.X% compared XX.X%, X gene phase. Curran. X.X you, was for with measured at to dose we from RGX-XXX the are RGX-XXX, patients, pivotal a to expression microdystrophin new be

adverse be levels, X, As improvement. assessments of creatinine expression reach events to in trial continues with evidence XXXX, July kinase reduction and meaningful tolerated supporting X-month and patients of indicate and well who serum of serious from baseline RGX-XXX all RGX-XXX microdystrophin increases clinical no

consistent We all microdystrophin adds excited evidence are treated as data across expression patients. demonstrating high totality to the today's very of

addition, In caregivers. motor home shared were strength clinic assessments function via and in early improvement trial of evidence videos observed by

the strength of population years. approved trial This patients there Duchenne to in cohort remains significant is cohort a announced products X to boys untreatable, of a cohort represents prevalent of a no the X gene the of a new of aged AFFINITY and portion our include data, DUCHENNE On June, where the boys. we continued therapy expansion of that

produce for label potential. RGX-XXX's a As that we we data to where no a RGX-XXX, further establish broad exists limited continue commercial can enhance to profile differentiated or data product seek will ultimately

I'll administration. DR evaluated Phase AMD, with to start to via DR, which trial subretinal suprachoroidal for and being in XXX ALTITUDE wet XXX, delivery. routes Moving in collaboration using suprachoroidal of AbbVie is and DME in-office with the II being developed treat

our and have our pivotal Curran As with we us to are excellent mentioned, accelerated position trial the FDA initiate million End-of-Phase an puts we ALTITUDE and DR. Today, center-involved believe DME a impacts complication XX our in or in diabetic this disease announced first that than II with partner we XXX AbbVie, patients with meeting the cohort CI-DME. of macular new now more eye patients of enrolling evaluate to trial diabetic globally. edema is in vision-threatening a

progress the and We and are well. delivery Japan. trials for subretinal Europe, ATMOSPHERE wet continue in trials, via X These treatment of AMD to XXX the pivotal also evaluating in ongoing ASCENT U.S.,

follow-up from for Phase therapy. X in wet the data have gene AMD set standard clinical years long-term Our gold development after trial the subretinal I/II

fellow AMD, drug-related trial who treated today for Also short-course is in level patients prophylactic study using We bilateral in In dose were cases patients no wet have and also in choroidal significant fully endophthalmitis, enrolled we of artery option a the AAVIATE hypotony. retinal wet XXX XXX to support AMD with in inclusive inflammation, for at drops, study occlusion, patients use, eye. AMD there was well SAEs effusion, representing evaluating a both number X in-office the or with steroid suprachoroidal of tolerated intraocular eye received vasculitis, no eye the wet delivery. other This in announced XXX meaningful expected eyes. label previously the of open-label

on by profile stage. seen new We positive enroll and dose pivotal a to toward dose levels are at to we cohort safety path a date, X encouraged we the level evaluate as plan

observed, like the to including profile on the continue our particularly XXX to in I'd suprachoroidal by highlight safety our encouraged programs, programs. We be and progress

drops. our differentiated gene for states in treatment profile, XXX globally. in confident prophylactic potential a meaningful steroid the eye representing safety are short-course patients office, disease this physicians option seeing of for ocular we're patients plan a of and profile X-week advance than setting into in because to dose We levels therapies, more safety treated In particularly new and

reasonably rolling benefit pivotal our with CAMPSIITE approval. treatment or developed use disease the surrogate half the We finalized second predict successful of significance. endpoint achieved to RGX-XXX biomarker the pre-BLA at below Symposium, statistical from The cerebrospinal We trial clinical RGX-XXX meeting activity, therapy is the the Finally, of endpoint February a included the its accelerated takeaways trial for support MPS primary for a being BLA fluid, to levels new and completed be the brain DXSX alignment met FDA likely maximum year. key announced high that as onetime In syndrome. We've in CAMPSIITE of planned from share meeting treated this we decreased CSF gene levels. first RGX-XXX DXSX, the of Patients submission. plan attenuated data to II details to on the with syndrome. key believe of biomarker on Hunter World Hunter well-positioned CSF, of key treatment and that disease sulfate the with to heparan

conclude, on our we and manufacturing make reached requirements updates trial alignment trial significant across data continue CMC and design. To on programs the in also progression progress We to all with pipeline. confirmatory

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

Vittal Vasista

costs company's to for by XXXX. its plans $XXX clinical of pivotal guidance and equivalents The $XXX by attributable be cash with Thank based reduced quarter and achievement the for from or of offset RGX-XXX. REGENXBIO XXXX, million, to as the cash The the increase of in patient may the dose including million stock excludes the clinical upsized that net under in you, cash offset decrease any operating XXXX. was marketable December balance in fund largely personnel of XXXX, in payments of public AbbVie potential securities Steve. in and treatment million as XX, for REGENXBIO operations million the partially $XX $XXX received $XXX marketable second cash, securities common a result ABBV-RGX-XXX of million for its related The ABBV-RGX-XXX and proceeds $XX operational a first and XX, half to of of in to of of XXXX. R&D XXXX. supply million XXXX RGX-XXX, March ABBV-RGX-XXX fund to milestone reduced primarily expenses commercial in cash, compared decrease expects is quarter was current upon June headcount. quarter suprachoroidal were and the equivalents received as XXXX, activities and impact on collaboration, first by offering achievement pre-funded the warranties expenses was driven of second for runway partially milestones used compared trial June costs This of cash million for from delivery DR. of ended manufacturing trial on into for development an the $XXX XX, completed increases

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

Curran Simpson

Thanks, And XXX a rest data. to will we pathway to the discussions we positive are another that as ahead by program of FDA best-in-class the exciting utilize with as day are our for Duchenne. the treatment year, encouraged and was Vit. with approval pivotal advance our potential accelerated us for additional enable Today finalizing to rapidly plans XXX the look

pivotal AbbVie year. for Additionally, start enable End-of-Phase we meeting with to partnership pleased next the in a be to are accelerating II DR

Operator? therapies close turn significant we over unmet plans excellent make we reflect your I'll as the to advancing on call everyone, our emphasize and the time half first the that are needs. Thanks, continue value questions. on year, of track that today As today. and I'll for for expand progress address we in

Operator

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

Huidong Wang

regarding new XXX the in on data the DMD. Congrats

meet pivotal X And have I threshold is the do achieve? the you of in do you with XXX, expect finalize a have AMD. I and question DR is FDA, So One will dose indication approval? accelerated for existing questions. regarding the X? with you for to XXX But some goal know level protein set suprachoroidal include wet also neutralizing another you will you a reason in order study. What's will the level FDA antibody? And patients wanted And

Curran Simpson

the Thank question, you for Gena.

clarify goal can -- In the I terms a of -- first question, for

Huidong Wang

level. Protein

Curran Simpson

the FDA. microdystrophin level. of our things the pivotal that XX% above And the proposed able will We've addition final agreement the result that thinking in forth occur of that feel -- threshold glean waiting that's one we'll and the field from in benefit. plan with Protein a and for with to just functional be sort minutes that back we've and about will been likely general, be that investigators level is to what to a submitted. We in

date, published if field every data on to discussion a to data second above think that threshold. I'll that's you our that -- that the will we patient that question be forward. is think going Steve, if okay. I But about that's have we've

Steve Pakola

the Hi, for Gena. questions. Thanks

safety. dose that? I So retinopathy have our Phase certainly our XXX why We've and excited as Curran we're towards and have excellent that level also going profile X II in We've do so highlighted, to diabetic I met and seen, with studies, target summarized. pivotal advancement the product in why that's

as really suprachoroidal believe to key consideration that part particularly RGX-XXX. compartmentalized product, the less the due space our spaces the higher the can. think intravitreal opposed that to with given space, I going excellent So because have the compartmentalized route is in we safety and is We we're into the flexibility, like we

think with have where, doing fits continue it we're basic development, general you just safety you looking a to this. if if doses, drug And sense also I fully can. in characterize good really makes So, to higher product, AbbVie, collaboration it at

Operator

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

Vikram Purohit

X. had We

set be data regulators program with differentiation, we're to going First on interpret overlap of discussions a steps? And is a a talk pivotal speak how functional and best might pivotal getting that real-world assessment moving play you sense efforts XXX. year? differentiation, Could to that this Could get on for program, how you more What on later into then, your the wet about data to XXX next about just towards guidance wet and potentially And program? the for suprachoroidal and with secondly, a that with AMD, that bit how you XXX AMD. see subretinal approach? could bit

Curran Simpson

with you to first one? take do can Steve, start the second Okay. want I question the or,

Steve Pakola

you Could missed second second the I -- one. one? Could the repeat you

Vikram Purohit

and AMD. It was wet XXX, Sure. Yes. suprachoroidal about

see for there. Just underway? overlapping a program wondering have effort potentially currently wet And you efforts the program pivotal with with AMD, to is subretinal how your the a what and you path XXX pivotal

Steve Pakola

Sure.

the experience suprachoroidal. all experience with both sense from prior good have So safety assessing and and have with cohorts of the wet efficacy, AMD, a pretty we both we done we've with subretinal our also

So on that take think it's having usual safety. We of top the to advantage I good totality we get evidence of, of think of of. the endpoints course,

So biomarkers of good very BCVA, like in burden. treatment get retinal thickness, reduction and, objective a where measure, course, we

So any all as patients control, XX% and to months and least durability injection type what's and at injections a we'll patients injection really over in to by of we reduction subretinal and burden. And we've control, BCVA program, in been about talked compare our really where needing what reducing while look at retinal measured anatomic we maintain at XX% X of response thickness seen, often dramatically longer not see get see that even and want example, for to of really those of good burden.

Curran Simpson

amount dose them, and data I microdystrophin to fall, X-month on the X level the months this we point. fall, the X see for patients just the rise to programs. And NSAA functional a a a Curran. X To terms see to data the some of number assays, functional baseline, X time is would assessments level in every seen for generally functional probably early to of other functional that the months, in and example. expect time you've then XX of measure dose historically with significant measure we as like X-month in for patients, out data And at things you'll question, And of and first an

emerging, finalized exactly data it's the come We out because still the haven't plan fall. that's what for will but

Steve Pakola

the AMD do in how the for Vikram, of suprachoroidal. subretinal thinking about when context this wet see you also program had pivotal ongoing of question our we

fair procedure. it's and why we in evaluating while and both, we've both us the say opportunity There's, in SCS evaluation durability with think shown are see an for by the to optionality has data X in-office we I advancing to safety we pivotal really and excited subretinal, program the very the that. with in subretinal. Certainly, additional the that we the of continue given we look at to that's having further gold that AbbVie standard together that opportunities course, of both with DL to this excellent see And efficacy in option. expand cohort more gathering forward opportunity about that keep

Operator

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

Kyuwon Choi

A if clinical would is a And next a whether then VEGF on I with for steroid expansion then year in later and first data prophylactic that this provided think dose on use second But pivotal comment reduced be label updated view early question is, could this surprise. regard there rescue And levels for on program. interest here. wake for year to I need some came community was ELEVIDYS community approval know a and there. here. would potentially use? my of you My XXX, a just recently specified competitor patient the recent or as their can bit I potential maybe be you there the wondering the X elaborate maybe of XXX you in trial Cohort

could as cohort? just the thoughts or rescue you potentially So there that dose X reduce injection-free as increase usage prosecute any to Cohort whether frequency the either

Curran Simpson

question. one, the the I second first Steve have take then can and I'll address

interest market. PPMD on are just patient check at it investigators interest. these recruit centers many are our still and on investigators The for in to see able study. people is the to the a been in seeing XXX, program. and to product I around think, demonstrated differentiation discussions therefore, there I strong program meetings given date. of nothing that And resounding the interested that at are seeing And in safety would been the confidence like but up that the and there's on to end, patients Many ability answer our that in we've yes, have that sites really say with XXX we study our offers, strong recruitment our and that's patients strong the looking with having there's

very XXX over early pivotal to program. And to ability I confident recruit Steve turn days, feel So to question. our it in address I'll the the

Steve Pakola

Sure.

further in we So regarding we've acuity seen opportunity we there's also anatomic stability decrease X assessment and benefit indications, DL what control. the and could think certainly that than visual use these rescue maintaining while

one do to have questions, mentioned that. flexibility the I the As of earlier we to

safety that's data steroids to a to can at duration really of issue inflammation to if either of referred one issues, earlier you has higher long how the of out some And, always year. if a And also can I think this you additional about start dose. on prophylactic be up run issue there's go including other look immune You that come into and response concern tolerated. and

that's we're don't profile So safety we excited our very prophylactic where why about need steroids. extended

the think go we up to chance the injection see that and us of reduce So further. totality higher burden does if give I can

Curran Simpson

in some the older that treated. the just been have differentiated question of I people release to data did And of we one of microdystrophin recruitment. on purposes really level patients was today the the follow-on, to of mean, the show one Paul,

to with. another their information patient can what that treated have use child just therapy they're deciding a of piece that's So when

people purposes that understand was to release of more. is the one help of our product that bit So a

Operator

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

Mary Davis

Alec for Mary today. is Kate This on

for do others of in safety the what terms you'll for required to approval questions of safety in around expect of kind be size one you duration DMD, database collect follow-up? and Just on given field,

Curran Simpson

still think, probably and in Yes. the plan something FDA. we're we'll to working pivotal up submit that That's, on completely I that will tie

So not comment terms there. to we're in specifics of really able various

region, with think sample in studies. a product the FDA. we XX I I on during reasons think, a one, to changes process profile is general, -- discussion is, but size make really the we the But that's in that our won't a terms commercial-ready of feel in that ongoing proposal valid that's number number have XX-patient we to that's during pivotal the

And the to a so the FDA should safety of be assess sample in the already we've should that and be. the reduce what initial program should view how in the that size And sample they record size. second, demonstrated date how might trial positive terms

we that And there. good gives into feel next we're that think a year. the I recruitment a position will like in be good accelerated us feeling So

Operator

Your of next question comes Stifel. from Annabel the line of Samimy

Annabel Samimy

have also We X.

DMD. on First

looking be functional Just might you back what and data the question of for. bringing

to in trying are on to granularity giving we you looking the I'm level broad one see maybe the Are depending age they're at be say, dose people given, landscape a feel based group? maybe looking their question. maybe guess there you're at age on Is other because space. might already? consideration a one for thinking a competitors race in group competitors you're or that get the you, space, XXX, age into those and you for possibly I it this it on right specific So to baking a population the you broader guess, therapy And that III? And for X to the and is little bit Phase And the what other way? in light less that in understand the subretinal? benchmark the how about wrong how then of them much Do patients I space? and area. benchmark are ambulation, matching on measuring in the the less So rare against natural gene history first large, of this levels little bit and is age different you genetic disease desperate the metrics is your there then any competitive Given different urgency of out group the the maybe than, little stratifying

Curran Simpson

I And we start X dimensions. functional sort think think in XXX. can with about data of I

course, relates be expect to of measuring And data approval, X we're measuring specific at way. our baseline initially, data driver But it primary patient. As the with microdystrophin in along months endpoint. reference would along for a is the fall what accelerated our the to of functional primary functional I will as that, levels likely

if a or by and that matching of correct confirmatory we're history you're discussions studying that absolutely natural Now, disease about having think either that we study, strategy age patients would by progression be part on.

question. as expect data. I'll we're -- see views to it's the both relates So actually, over of sort to XXX I it would functional turn it to for Steve

Steve Pakola

Thanks, Annabel.

nonsurgical of of to I Really program. subretinal what the think experience, a have opportunity little the suprachoroidal. in-office in-house benchmarking, of prior pretty but our us broader, programs given as as show with we is mentioned, we lot as you slightly So perhaps of from a terms similar many got far in certainly suprachoroidal, target when of well, as

important shown. when been is different durability think context programs assessing really thinking I an has of how really much

creep to I being and some really going a for able there's approach at about out with patients given, So needing need not think gene the to at even look having beyond you're And that And a injection, goal to can the repeat for may too that excited seeing allow to been point and that's agents sizable AMD. of to see point you ultimate rescue have therapy looking activity early wet the reinjection indications that up. time you're really the going anti-VEGF durability, that's dose certain though consistent that greater why months stable, reality injections. example, proportion start have are for of have to you require the a any in X we're if need relative like where last they when loading

to efficacy know this approach look good and know at and feel we safety move in really a we So to position to how about when we're forward. the

Operator

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

Christopher James Yeh

Yeh since on Sarepta's for is announced than please current Mani. on Could X? label comment This to you've DMD? you while in is youngest CJ Are X, you've X Sarepta's targeting you age younger strategy age to range the patients your expanded

Curran Simpson

Hi, to ages the think database in across adequate we'll wide be want say it's a X safety in I this to studies. an of enrolling But Yes, at is a the safe ages for to variety Curran. X. build minimum we patients

Steve Pakola

broad had to at ELEVIDYS made we age expansion. X label to X And including range, the before decision this and expand any look

we've seen and opportunity we've now to with end, on expression we've the X-year across So at opportunity. to X this that safety the range it's a results, olds. upper look a the And age great seen wide microdystrophin seen

Operator

question line The Merle of UBS. from of Eliana comes the

Unknown Analyst

[ months on Eric in-house is My have Do over effects data is one X understand ] already time? help they follow-up. to and time? over what Eliana. dosed Do or Moskal in ability first expect more? are for deepen And time? to for what see label to with I over XXX. calling might XXX you be months X And on you any beyond better do you ] expecting patients the This one be stable the have [ us Or

Curran Simpson

beyond levels I'm example? microdystrophin sorry. for other functional to referring Are or you months X data,

Unknown Analyst

Yes. Microdystrophin.

Curran Simpson

with basically FDA. have we're No, at that then per beyond and biopsies time months. We biopsy treatment point Okay. taking X direction per to prior a don't --

periods. preclinical obviously microdystrophin data be has But we includes have in durability that a I functional excellent months. devoid half-life And that. X measuring than of in construct beyond durability. of Yes, data that's C-Terminus don't we'll However, our out the outcomes really the longer would suggest think the terms

levels of higher relative to both literature. microdystrophin in expect levels what and other would you've sustained seen I perhaps So

Unknown Analyst

been there characteristics it. Is or with patient follow-up. higher just anything one Got And far in quick that to terms identify assessment? higher microdystrophin you've correlated expression able functional of so a that's

Curran Simpson

Steve?

Steve Pakola

data be biggest actually other there the seen So from that as up predicting have whether that be concern we've so you of case usual I'd programs aren't microdystrophin on age, is that the go but not at patients been which say that issue, some predictors. a terms really confidence suggest all. learning the that in has may an lower a has age that not enough community that the and caveat that far had, might is

striking microdystrophin thing robust seeing is our and we're I in data the think even So very from levels. older, X

the seeing patients. we're levels microdystrophin that, across Beyond

no So predictor differentiator this sample or in date. to clear

Operator

Issi comes next Your of Luca from the line question of RBC.

Luca Issi

Congrats not and age get I can you across read progress. with patients Curran, that appreciate Maybe existing still and only your are today, can with DMD II But because excluded you the that of on the that minutes. label think waiting important on FDA? either accelerated just patients I the End-of-Phase bit meeting expression from your you're is more recent can or confirm an expand you approval distinction. a for Sarepta all that the that's immunity?

here? sensitive could maybe the much in other any Star you still you appreciated. And Or appreciated. endpoints more do as confirmatory endpoint? are think are be then So a DMD, color, there color, how thinking Will trial? primary still Any North that about you much use

Curran Simpson

North a time to I rise you've but bit We'll of work measuring we're indicators that some seen, number mentioned use walks functional associated. as the of earlier, and obviously can a in I timed additional are reverse. that Star,

So not much patients only capture -- it there strategy approval, strong, as can. Related are is for current the able I to think That's where unmet basic of data as want one tenets can measuring across a broad for accelerated we functional I differentiation we need. possible. as unmet we for that product. And a feel as our because we are evolving simply a and And we're basis have to approval each case a ineligible really functional And need. of think approach would be the indices I I the accelerated narrow the approval this assays broad is ambition approval, obtain field it's as that the everything that as are think to of be we many evolving therapy. to our is characterize the will for product is as a addresses And label still to different.

Operator

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

Brian Skorney

to sort at patients? III who you to hope to year. a the will supplement really progressed the half the there are Phase diabetic What answers to III And do of progressed level is Or AbbVie's and to off? gets kicked in consideration patients think just that the to confidence the of the these Phase color companies some FDA first here pursue with of meeting the retinopathy, questions sort FDA in -- just sort data consideration initiation II gauge by some more or thought End-of-Phase on the this of level that need And for -- have next On I'm the on ensure get to DME? maybe the the of primary just plans confidence DME cohort. your answered trying FDA is is

Curran Simpson

Steve. DR then I'll We're this to thinking on think and feel of the of what study pivotal there's I developing eager II give AbbVie I'll year. diabetic And let Steve we're and comment very I'll level get think the turn in and over have think to really But the progressing shown we should details of precedent. high certainly End-of-Phase a the meeting we I in next the like a into year. how I pivotal up this on study that's acceleration retinopathy, comment let a and comment confidence approach details and brief into Steve of going. established go. that But endpoints approach. standard in And to

Steve Pakola

Yes.

data that, power proof-of-concept that's to to negative from by been us there's that have. a expect laid of what receiving really arm has us retinopathy an with we do actual the active allowed diabetic helps really course, agent. anti-VEGFs Fortunately, And studies for not a map know you're you that as combination mentioned, injection road in repeat control if Curran

endpoints lot also are We accepted. a about know the that

a very this So lot really quite we that is feel de-risked standpoint. confident from regulatory a

pivotal So it's comes of really some and frankly, the -- around the edges the and when to like, definitions designs. some assessing the it things some

the accelerate been to have seen, instead that's able we've that, why we one QX then of So your... into And II year. given next QX results meeting aspects that positive think of I the and bring End-of-Phase this

Brian Skorney

going just was I ask to the about cohort. DME No,

Steve Pakola

Yes. Yes.

center-involved looking we So are at DME.

the Of indication. traditional course, that's

others outside others be looking at the of that what So we really have have terms the population that we'd context of box patient go so and the need looked type have seen. at what in a to we don't see of can

do VEGF retinopathy that So, DME. just know a higher wet opportunity. We yes, non-proliferative like than DME, it's diseases DME great drive a diabetic or it say, there's exists the without AMD of with, complication and little

very dose-response to is interesting patient this look population. we at this the in where range we're to DLX right going So think be around

Operator

Your Daniil next Markets. Capital question of of comes from Chardan the line Gataulin

Daniil Gataulin

retinal translates Fellow And Curran I congrats a on program. And do sharing Eye on delivery. anticipate This is When in role. follow-up question how [ that your with more the strategy eyes to given from what quick a have new ] are it likely wet as is long-term you injections, your study? data those AMD for treating to suprachoroidal system, delivery? interact the to suprachoroidal fellow immune

Curran Simpson

that turn I'll one to Steve.

Steve Pakola

Daniil. Hi,

as study. Fellow Fellow far that update we the we today Eye of the have Eye as when So enrollment we would results, have completed

data or immune when this traditionally to not X kind add subretinal, you eye, you of affect have of the immune type response, so stable effect subretinal to have to have label inflammation seen straightforward dose fellow feel and would rough of disease. much no allow comfortable to, a regulatory were the really think, intravitreal issues most standpoint eye safety we an we've to study the whether terms a in say, safety you we status when indication compared from inflammation bilateral range that as the a least the of have issue would greater I less give of referred, from because releasing that the been you dosing you space. Suprachoroidal, immune-privileged up in second response expansion on of this at administration. just show excited confident you But and/or to months won't if is So to that a efficacy that could have

that So would as suprachoroidal well. that opportunity that gives us have confidence

step So take time. we to one it decided at a

at fellow certainly, look as to over assess subretinal would eye, First fellow it make time, with well. eye sense but suprachoroidal

Operator

you questions. joining. there gentlemen, no and concludes That all today's Thank are Ladies call. for

You may now disconnect.

REGENXBIO (RGNX) 1 Aug 242024 Q2 Earnings call transcript