- Earnings call transcript Q3 2022

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Operator

Hello and thank you for standing by. Welcome to the Q3 2022 REGENXBIO Inc. Earnings Call and Update on ALTITUDE. [Operator Instructions] It is now my pleasure to introduce Chief Legal Officer, Patrick Christmas.

Patrick Christmas

Good morning, and thank you for joining us today. released September operating XX, REGENXBIO third for morning ended quarter XXXX. this financial results and the Earlier

of Report recorded may we otherwise. provided as call no Please we to this undertake on webcast. that we quarterly with and call As obligation those and reports today's on XXXX. and well XX-Q sections on development only and as forward-looking development The are www.REGENXBIO.com should, from being or statements press words similar the statements factors and in differ of These this of new statements And conference plans. Securities REGENXBIO's other to statements can And plan, X, performance of data Analysis by uncertainties as from XX, and Today's and the to results actual Commission at November of and any addition are outlook subject website. presentation on on call Factors section such These are our regarding is SEC's Risk data RGX-XXX cause of which our REGENXBIO's call identified forward-looking forecasted. information, available be such are XXXX future comparable uncertainties. December Exchange of and XX-K Discussion risks will, on and only of be future will for in product make new may are forward-looking on available words this the call our that guarantees financial at releases information website Any full meeting. ended may, Annual risks the Management's include intend is risk ALTITUDE date involve on described of our year expect, update events account not the advised trial. and and regulatory as certain to Any forward-looking anticipate, file believe, Form and conference Form provide risks

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

Ken Mills

Pakola, September Good Patrick. recently an joining today's our results from corporate of call to you, our morning, well for the diabetic Vasista, everyone. presented of Thank on Chief I'm Steve bicoastal update as was DR the he of ended West will Phase quarter pleased on of as for trial our the a delivery. overview XXXX. us. business treatment that RGX-XXX Thanks provide financial ALTITUDE yesterday with highlights, X the Officer XX, goals. for overview begin today, an Financial Medical recent the Officer Society, Chief the data evaluating Coast Vit Retina recap or using Society will at our an We're suprachoroidal attended retinopathy provide Retina in-depth Meeting third

diseases open we on At is questions. the need. of gene potential through the up as Cleveland Towards Duke Expert, be Clinic. Peter Kaiser with Investigator Lejla and call, University; the of us therapies improve therapy to line REGENXBIO joined the lives ALTITUDE our developing mission end curative Peter Independent Retina for on by from from Lejla the unmet stay Dr. have will for and we'll focused significant Vajzovic that

I'm our And quarter. treatment stage thousands strategy dosed from who internal this I into NAV now hundreds been of or advancing technology be therapy. There place going are have with a programs our from from internal by summarize proud company patients Therapeutics license receiving in announcement been We to very have stronger. to progress every pivotal pipeline of updates XXXX. morning. operational the and of AAV AAV program and put more highlights never how our platform to been our our into our Therapeutics fundamentals Xx'XX derived continue our I'm leader commercial some five and We gene pipeline. products believe has

with advance to Our eye filing RGX-XXX treatment trial retinal observed reported in delivery supports for to in programs. in excellent collaboration commercialize develop emerging to on four and and BLA care is for continues up disease AMD and XXXX. with first enrollments of RGX-XXX trial years. was AbbVie be progress delivery durable for global also well data our our Progress subretinal Wet track to effects suprachoroidal now At treatment the tolerated AAO, long-term clinical

a been further highly trial dystrophin. first a ATMOSPHERE differentiated in announced of continue to diligently RGX-XXX administration, skeletal occurring Hunter interim and Duchenne with and patient the ALTITUDE as the the Phase of and known as candidate AAVX human trial suprachoroidal expansion of -- RGX-XXX that lead a the and AMD the transgene in also the six affinity this to These of of submission a our to genes found one-time as anti-VEGF detail of delivery. BLA October, includes working from for first treatment product, the our treatment to prevent moderate observed was treatment positive of stable was of treatment suprachoroidal XXXX. tolerated trials and the intraocular heart to potentially Wet burden the data prepare designed This on designed the prophylactic data in delivery Mucopolysaccharidosis being therapy cohort, of initiation course inflammation. dose Retina RGX-XXX data the using gene all program for of trial data trial functional our In the will trial is well currently Duchenne mentioned the domain X Phase naturally order the this show with potential We delivery steroid microdystrophin, the X interim six XXXX. pivotal to mild the a was We of RGX-XXX ocular Syndrome. Society BCVA support called of the as in incidence in We've in Yesterday, RGX-XXX Duchenne the targeted trial discussion our deliver vector. level meeting, RGX-XXX reduction two Type evaluated our first meaningful throughout the levels to that third is these pivotal in treatment dose domain, at is naturally half an developed presented announced of elements ASCENT II, positive Steve we dose short in for using of out novel ongoing months. for C-Terminal expression to along I the and with RGX-XXX for AVA expect of shortly. explore to following DR at in new our from planned also occurring of NAV to phase CAMPSIITE. using for expect greater to review and muscle expanded treatment being support in RGX-XXX

II from and administration fluid reductions cohorts, cohorts indicated pivotal positive Improvements all GAGs neurodevelopmental filing tolerated reported is caregiver demonstrated patients function to RGX-XXX. The this to RGX-XXX reported of one have of data this GMP scale spinal program. study cerebral using MPS a expected dose-dependent activity of patients study. RGX-XXX to filing update in potential years after recent pivotal two to a Most outcomes BLA -- CNS phase this the and enroll three dosing candidate BLA pivotal up XX expanded of active the following another Biomarker across program accelerated the patients. We using this for well interim all program commercial additional support in patients, approval time in administration. encouraging with is data in opportunity that from was up XXXX, This by material pathway, enroll to our second XXXX.

expansion X/X RGX-XXX I for arm. to patients syndrome severe of in trial additional continued Phase Cohort ongoing with of Our treatment enroll the Hurler plans or MPS X

capability Our differentiator center a key capacity key element manufacturing strategy. and innovation -- and a GMP of REGENXBIO our for remains

from is in-house material, to accelerating edge quickly production clinical-grade us early selection of AAV Our development to supports of which cutting the candidate Therapeutics. facility move allows

all of product trials process commercial just to our which I we and quarter and enable quality gene early during from to now we lessens to for therapy development unmet REGENXBIO reflecting this staff patients AAV to at over our our would take whole with turn We guidance. we've a certainly control made Therapeutics. to advance to proud the rare for now chronic families. approach dedicated Xx'XX Additionally, and clinical quarter like efficient a in our Vit believe commitment transition year, the review communities of of for entire I of progress to and and need site remain very this investigators that, their diseases, support results these to the their and both point one-time Overall, strategy. patient this address clinical believe call the that of will financial I'm in on on and their changes and readiness. and focuses the third And needs team, range time the our our thank continued development

Vit Vasista

the by to reimbursable XXXX, manufacturing-related XX, securities trials primarily with REGENXBIO fund of with costs the $XXX.X million in XXXX. Ken. was on product AbbVie. plan, capital XX, be collaboration XXXX, cash as cash will decrease operating ended for care expenses September the compared certain recently the by development debt was XX, you, to September fund the our would expenses AbbVie will agreement, an the AbbVie Thank discussion marketable $XX.X are were December other was of million ALTITUDE for ended to on totaling development XXXX attributable $XX.X into as trials balance on and XXXX REGENX-XXX fund data. the shared to expenditures, And quarter in unrealized In The like equivalents securities Based development of and to operating of our temporary associated our as ended our announced with million accordance of over candidates XXXX. return the the RGX-XXX now, REGENXBIO well of used R&D quarter Beginning marketable partially majority million Steve in-depth end cash, clinical we of losses the continue and cash September September expenses. result to funding XXXX the equivalents expect XX, operations and offset all $XXX primarily quarter for and for activities compared XXXX. costs REGENX-XXX under securities through during nine lead September XXX marketable with eye months we million ongoing XX, while personnel for responsible ended cost to to collaboration. cash, XXXX. for current call increase The XX, $XXX as and activities the clinical

Steve Pakola

Thank you, Vit.

treatment the trials data delivery. we're RGX-XXX for shared, RGX-XXX AMD the Ken be of in treatment and new from also that website. meeting. pleased and RGX-XXX subretinal updates As of interim can of yesterday DR presented suprachoroidal our for Retina we sharing to at presented Wet that Slides for media on and being was trial delivery Phase is presentation X section recently be interim using from found data the ALTITUDE our our using Society suprachoroidal presentations publications the developed

An Peter Kaiser, Cole cause the diabetes Center is the morning the slowly blindness Joining progressing DR yesterday. edema the We're Director Dr. estimated we between Professor the joined also me diabetic therapy, of can worldwide. Investigator XX. can adults Like and are is the to by complications. neovascularization the by threatening is to lead of or a Ocular blindness. Director DR to at the Evaluation Institute DR patients vision patients vision and and Research threatening that leading that Vitreoretinal proven ages the Program Lead treated risk Eye macular XX anti-VEGF of of reduce including disease Ophthalmology disease and which Lejla this lead a with Wet million Duke Vajzovic, are the XX data in it have with who debilitating Cleveland complications of and presented developing Fellowship in affected DME of and complication is Dr. AMD at of Clinic. Associate ALTITUDE for

that off become progression. an patients X. could patients or evaluating the with important severe the escalation therapy suprachoroidal of potentially tolerability unsustainable and for Microinjector of severe result therapy delivery eye diabetic trial in retinopathy, We multi-center NPDR symptoms their RGX-XXX the which burden unavoidable. X.XxXXXX is intraocular gene dose we to dose and Phase-X believe DR in of treatment non-proliferative condition efficacy, proliferated injections or therapies, anti-VEGF per diagnosis retinopathy RGX-XXX using any disease like put overcome Patients controlled dose required a safety to due mild until a treatment this received open DR Cohort receiving this as refer the diabetic at PDR. many RGX-XXX frequent SCS label level the patients primarily using copies hurdle to X However, with genomic moderately of a ALTITUDE provide alter of randomized significantly

an X did corticosteroid While Patients therapy of refer which X received level and to X after in Patients Cohorts we X. GC/eye, X XxXXXX through dose increased administration. as in not RGX-XXX or RGX-XXX at before receive prophylactic Cohorts dose

in well events reported, As which drug of October was adverse were RGX-XXX reported Cohorts considered XX, tolerated serious Five none to XXXX, were related. be XXX. of

For the receiving episcleritis. six adverse the ocular group mild conjunctival in and of total Cohorts in XX months hyperemia predominantly and patients common were RGX-XXX, hemorrhage, XXX through included study conjunctival events eye total

X positive. outcomes safety and NAb for experienced on intraocular all Corrected topical addition, months, resolved were control clinically Retinopathy Acuity meaningful corticosteroids. disease who patients severity In RGX-XXX X Cohorts through patients IOI, or meaningful were which Severity in are X stable Best six the Scale At Diabetic in treated measured mild by six There no demonstrated versus Visual observation remained in inflammation observed months. DRSS. with improvements through the differences patients as of

Specifically, X XX% control. dose dose and XX% in representing in DRSS XX% score or X greater of improvement versus in X-step patients patients observation achieve XX% of in patients of

Additionally, experienced achieved of improvement and in level any least XX% patients of zero a that in XX% X RGX-XXX control while arm of had in worsening. patients, patients at control. important level DRSS versus patients And in XX patients of out of a in score, note least X-step it's at representing XX% the dose X-step level worsening of dose XX% XX% X to DRSS treated

interim itself I in lovely to really both hear great your We're yesterday to that Society as that with here next hear over is for your now, So, the meeting us Lejla will Investigator trial sitting the clinical now out, call Dr. turn results Lejla it Pasadena. a to opportunity me of at perspective excited in laid you presentation the who presented. Lead really Retina it's to the on we're

Lejla Vajzovic

release be Great. burden Primarily a discussing today. up to nicely retinopathy presented here to data huge because highlight busy health public problem very that was frequent And unfortunately Thank that you, and diabetic tremendous this injections. cannot keep their is Steve. in problem It's I'm with globally be of the age very who patients morning. pleasure as a and diabetic about interim a Steve status treatment a retinopathy clinics working yesterday excited

clearly not So, tremendous and being a typically solution. having office onetime clinic treatment. treatment that coming treatment about results unsustainable for And XX% my better of appointment. need my patients are watched is a non-treated. me But even in provide for And the there more also

see data to interim So, I'm excited the result.

that even looks The fact saw the results promising with encouraging First score. of looks include as to Severity in evidence was of well. inflammation, which minimal we're me. But efficacy including very improvements all disease all, in And the promising results. that Retinopathy Diabetic result seeing we the data with no the cohorts more safety worsening -- good importantly in

this having who disease translates to potentially those worsening. provide solution when a affecting me the option So for being of patient hopefully may are decline a XX% and to the they option going overall, future vision this in treatment observed, are have we currently visual to for

Steve Pakola

for Lejla much, so Thanks perspective. that Great.

to at at presentation. third level and a in the outline GC/eye. The cohort higher Cohorts currently Lejla that drss new enrolling well levels And potential which Lejla in the XX. severe XX we presented X drug RGX-XXX to X to cohort which level. patients as RGX-XXX levels moderate of the to at by mild severe we're as two cohorts. interim XX are build As patients XX levels is stratified to very of with and this third that cohort to DR, X dose moderately made for we've PDR profile DRSS in trial of this NPDR the about DRSS dose is ALTITUDE data of patients is trial X the hear, X and to X expand enrolling DR, can having patients yesterday upon with in have excited audience the of decision in continue XXX include

at prevent be cohorts that RGX-XXX prophylactic of these the date Patients reduce steroids enrolled these range. baseline no intraocular ability the incidence setting in evaluate ocular AAVX short excited status. will So, ability expanded or course receive the NAbs to cohorts mild in steroid's will of following to regardless to seen patients a in evaluate to in a prophylactic of inflammation of

Pasadena Kaiser, scale. addition clinical use across is So, expert central expert the assessment Peter with a center and very retinopathy space an trial and being experienced including DRSS now the Dr reading overseer we in also sitting morning, also diabetic of clinician retina here in standard an who in us to have experts this greater

expertise to how you we'd interim think when So, this Peter to opportunity comes key perfect hear scale really the so, from and a both also your retinopathy love of DRSS use really updates. it blend of diabetic your to endpoints clinically, data takeaways but

Peter Kaiser

and is, regulatory Yes, regulatory Diabetic and my Severity as we retinopathy Retinopathy at level general Lejla significant EMA that a the why in level grinders, was I we two years looking for a reading at have retinopathy almost of FDA center of studies ago the retinopathy approval. one for for understand the clinical change, we use something are it importance do change great, thanks really diabetic outlined endpoint the which and very important score, use inviting level of is Lejla we is is done that's for at a diabetic do won't XX It's products clinical in which randomized the covered developed the is a FDA DRSS as early first that with the and across two\-step now Sure. approved with treatment study that treatment be And and that that the really counting to medicine. closely XX, study. me. a long that

not However, getting products injections out, often very the do reason time in we But out. are we in to age these now. working requires as better because overall get clinical patients we take general, from that few we use pointed these is patients Lejla it see patients know, to there use can you Lucentis the the simply practice, and our is for Lejla don't correctly and both want frequent work injections approved in it don't what are FDA there off

have to You want the know, patients proof just little injections up changes for Most interim in This these that Retinopathy an for with gene really in really to but RGX-XXX. don't patients these maybe app of in doing Severity the and don't results. have can see study me of therapy, they do the killer Lejla at a is these ALTITUDE yesterday actually of in is improvement is the all as physicians we procedure to it. we see scores, This that us improvement retina it very saw brings very nicely field. the by office which idea allow with of exciting the the to Diabetic patients presentation very that point

Steve Pakola

call Operator? we much And are the Wonderful, thanks questions. turn with so Peter. to that over for ready

Operator

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

Unidentified Analyst

is is question for the much two [Xian] questions. the taking you our questions, have thank This I about Hello, Gena, first for very inflammation.

question be resolve? used what's pro to more does duration how a long to similar seem resolved and inflammation the was AMD dose. if cohort. and how the the give how inflammation long I at to – onset - Could rate like on onsite XEXX - long Then have new you wet on follow-up and take like it dose steroid cohort at, pretty the colors the same So

So have the regimen? and same and AAVIATE potential can was Thank on do or used much. you wet you what steroids is what give as injection. topical one-time And short like colors dose administration steroid both course AMD for similar more be very it subretinal the as such will you

Ken Mills

to directed I questions as those are and is best this Xian experts Thanks, you can there. Steve, think

Steve Pakola

give Xian, experienced of thanks the first to comment her hi initial Great, give within over it Lejla hand. to I'll and pass the for questions, she the [ph] what perspective trial

we anything right. exactly in of inflammation unanticipated. So terms see saw, We didn't you're

We low the So quite to with in on intraocular RGX-XXX see in of mild managed we've in come characteristics very within rates of it, corticosteroids. previously course we were were weeks seen and with generally pleased six nothing inflammation the what short concerning of two that. topical easily

But and patients in this just inflammation. saw we leaders do seen will experience in consistent Lejla the investigators What as prophylactic of aware clinicians profile it far of your the risk investigators that we're we in perspective Lejla also own is prophylactic perspective, your comfortable steroids, assess. as But very thought we've with steroids course great your and How be these Our type opportunity trial profile where setting hear of here experience to very trial think further previously. our we're the that to the with we as That's characterize with expansion and mitigate even and ocular colleagues and further. seeing. data, the typical paper you a patients potentially across and evaluate dose have that The ALTITUDE regimen. inflammation what standard actually that are and was which have weeks trial. doing with the will are who over happy of we short the allows and study very with the of topical of in

Lejla Vajzovic

after say injection Steve. presented to, and itself. rare them presented two very really that trial after highlighted had maybe the I would six you, was patients for very mild if segment this potentially this present exactly more really we Thank carefully watching four to evidence it what any you in Yes anterior exactly weeks haven't it in inflammation.

inflammation them. if in is we really of setting. was for it, any in because therapy gene to trial proactive clinical of This our symptomatic noting and we were the patients these translates are treating hints actually that see because sensitivity something trial, patients. what We're and closely So watching

about as in did. that think I active patients short you used very was for And like well a this in very setting and format very inflammation topical it actually point of three think specialists. think treatment, promising mentioned safety I is reasonable all cohort the patients steroids, who manageable to profile I'll sequel steroids X% the is that clinical topical and was is had very overall well what out I So cohorts manageable translates with across my that as by

Operator

Thank you. Morgan of the our next And from Vikram Purohit question with Stanley. line comes

Unidentified Analyst

[Gospel] for Good Vikram. morning. This is on

We have two questions.

So the a question improvement is in generally DRSS addition improvement of DR RGX-XXX when then all? if doctors Thanks. know treat believe commercial measured for patients dose for with impact in do at steroid level are also a of DR progress two-step or the prophylactic opportunity at you any how and the are they they looking of will how to

Ken Mills

at Steve again you. right

Steve Pakola

topical how and to so DR I Great, are independently direct very getting clinical patients think of how Peter well a right perspective it managed I'm thanks And these for away going questions Gospel. about also because to steroids. think both fits to

Unidentified Speaker

so Yes, question second the handle I'll first.

So treatment steroids term RGX-XXX. the when This as results catch and in or look phrase. vasculitis. includes course retinal a retinopathy that when we mild I been committee you heightened is inflammation of new look review short sensitivity intraocular both retinal is specialists thing. safety no very all the This treated on vein suprachoroidal served I could occlusion the of with a retinal inflammation anything IOI to of not me is same and a treatment possibly Obviously, deal. There's Novartis see and the at that's this at occlusions big artery it mild for not diabetic to AMD that's any that intraocular retinal is

but we of about concerns so to not So see have way. in that data concerned none me any Certainly, more I'm far patients, that.

to improving. of so counting a average at that terms level. cetera really not these first retinal going but specialists look center to your patients do going that reading clinical do is absolutely over part to are we that we speeding macro-aneurysms of This the in is what In DRSS, practice, they et carefully requires make sure time do are question at the

have switchers is So in enrolled like we area very attachments other would early resolving what words to what matter hemorrhage would treatments they watching for and make blindness words, neovascularization this going so with leads that future we're XX study, was our closely neovascularization with sure treatment neovascularization clinical be other vision in of if which that that expect causes any levels because to disappear in actually to have that that So untreated. that's that is our problems neovascularization

evaluation This something that's about. doing Over sure see we're DRSS to microdystrophin could the time the a we also exam. most fundus disappear, what watch So hemorrhages to at easily exam most with patient the disappear so they would retinal make worried are like but we clinical also. formal just is we we're very specialists looking deal not the with dilated

Operator

America. of question line Alex Bank of the next with our comes And you. from Thank

Unidentified Analyst

guys, certain you're to our some higher about not that DR first, that you A you bar talk questions. us can for would rationale a the level for ideally maybe there from also is hit dose on in ALTITUDE, or the couple thanks taking already. Hi like on DRSS other metrics adding

need study similar appropriate run study, the talk a you pivotal a looking think head-to-head observational you about Thanks. I given, one down secondly, step could And to like three? an understand of comparator in in trying looks FDA. data you improvement but in the control of step what discussions is this your then you're at the through DRSS program? actually you're in a it far, doing to a with one the to was thinking maybe to guess bolus cohort how do improvers thus there's Just dose from there arm the AMD dose two, wet

Steve Pakola

the ever gene indications. AAVX the study for Alex Thanks, delivery in of first so These studies dose. in terms suprachoroidal evaluating both these two for these higher are ALTITUDE, rationale are of therapy

an to the of with the we time interim characterize really us setting dose in three data field via really administration. level wet RGX-XXX - for is we for this route AMD AAVX exciting presented and this So from that the study. And actually have

head a of have evaluating affect we start So this. can

AbbVie So in it for the in continue to our partner made sense evaluate strategic us study. perfect to

as two, and we change Regarding both in cuts do injections DRSS look dose is, mentioned. in we we an treatments to a we complications. as available when as to both to nicely one terms various study what aspect threatening know as level we are we in reality improving would not simply level there are one dose a control at and at Peter preventing want there terms two. that the are though ways DRSS. of And "work" level level repeated vision see in only mentioned, look That dose that reasons dose and DR very proportionate see of far the Those in of totality and this data, Lejla and the rare need pivotal observation patients Peter used confident for improving Lejla and what elucidated even

So if the so you your clinical [ph]. standard waiting nice we watchful think development we own control ideal you things I for at would When this control or the you treat the where be expect expect is experience that observation still of literature concurrent an setting what you and controls look what see it's either trial, Peter you'd see don't of to precedents have this do of care Lejla see and nice - in think is great our deal in observation DR one patients. and control really to about and and

Lejla Vajzovic

in real [indiscernible] highlighted, soon would I for see because they complications if arm as and no as overtime expect is observational we're were CPI to sitting that to what in it's exactly world diabetic retinopathy think are progression currently. that you've accessible vision the I we

and We personal frequent board injections, into and treatment are accessible. across not experienced from my observing really clinical opinion - so the it's these quite patients with

Ken Mills

control to for reasonable is standpoint to patient very be study current agree in you anti-VEGF not study hard study. required injections disease clinical regulatory a from the it only put if anti-VEGF a regulatory a is into be control the a It's against would fully standard opposite will me the to my patients doable this head-to-head Yes, is also very get sham is not intravitreal do not a that

but totally control. thinking, standpoint think to were from a opposite absolutely regulatory the So fine you it's do sham of I what

data numbers, set I a the think In group. there is beauty terms that control is this of of

improve blood oh, what better my at a this happens the study control having sugars improve, would sugars - DCT expect like you what of and leads if realize the is kind It's go with the what improve pressure at other or but control sort better in few improve groups get better I said studies look bit, past. you and are actually in group worsening. see pretty to that's if minute. little cetera that and not Panorama what's they be my pressure that do done actually of happening is will will going these six-month interim about the that patient the is to A significant the general we've as XX% and suddenly data of going So and blood can general et a disease control seen and wait patients U.K. improvement. exhibit in so in fact time studies a better that we that over and downhill you to in what XX% these PDs do the the some say miss getting because it's Lejla largely have due to in don't Ride/Rise control to XX% study that's that that to been some that patients is

Operator

Ellie question Thank you. next And from our the Merle comes UBS. with of line

Unidentified Analyst

Thanks as effect I the then our of improvement, status color treatment moving looking one guess so question. or forward any I step you second provide see no the taking greater change at I this during Ellie. that no detail than even DRSS is a six, worsening a of thoughts between you across antibody cohorts the Hi, on SAEs look the or two-step much. neutralizing and an differences potential would this month as labeling. sort And do score about Phase question change on that III at you occurred or Sarah be great? five on Thank much that or you for can at that guess into so in guess

Ken Mills

Great, thanks Sarah.

course of an of the we So AAVX we've dose similar seen the status. And how Nab variable at improve to cohort, also first in whether at data, not you as study, look anticipate. on thinking your what see can not. impact different the have levels can patients per are cohorts to and variability with in this that we XX Very question you different a you're impact is DRSS XX some you going baseline would baseline or severe to steps that about two

a actually good I question. think that's

of the you certainly, to and characteristics. arm. to unlike cohorts the in we direction think how of it the about based right But in totality into turn is that each to the at data find data of and dose creating on that moving I'll control nuance that patients the levels talk again take So in are of looking baseline that over if account, of Peter each the

take worsening and If the into having control couple on in biologics the account you of variables really improvements all average like subjects of a not the other.

the guess enroll measure then at or matter assess continue no how see there. of be these we study will patients it and to still to to NAV chose So again their expansion not. if status will we do AbbVie that independent still And see look our not and only we results, to able impact AAVX also to that's assess but really why and

get the I a I'll your I Peter based question when cohorts to example into look get for next sense question across go -- -- characteristics before how nice you it's you're you think the the -- baseline I do on think I think data, to really

Peter Kaiser

into. patients, relatively think, too I Phase much small who whenever numbers of interim to don't want in especially Yes, read you at data, X look

which at over But versus X if even you at trending large single a at all, a say, the You maybe shift totality very expect is look you'd important balance There's it cohort, baseline want look data shift, and every me, to and time. look in to sort are scores DRFS at expect right you very cohorts, and all see right group, the don't which that Cohort balance. in a at slight eventually in X occur of the a of the shifts control untreated you less is if they direction. X, there the to actually is whereas

minimal there's of amount and X, level Cohort more in neovascularization XX really patients fits. but early in PDR So XX level of that so is

that patient further. then move NVD disappear. to and So is is XX That steps a bit go easy to two just They to to little with a any very drop mild anti-VEGF with actually forward even quickly of going given anti-VEGF like NVD.

who's them try take and bigger to the needs severe two you to eye. somebody and steps. whereas terms improve in Thereafter, done That's a to much NPDR what say of change be

to the really kind patients said these patients at the few one shift point beginning six-month overall care we So if you the other going what PDR, at just who be They're have We me care not at two-step all PDRs that's it's early right easier of right exciting. change, to show what but and patients way that's a of of about time is of what's the that looking went granularly I more a lot a or population or about. way,

Ken Mills

Great. your on And Sarah SAEs. other question

an other vitreous hemorrhage. patients. only But the older, -- the had patients eye of as the five ocular you on things average broken the the femur patients as SAEs were the to example, Wet fact not AMD for give aspects that happened fellow So in diabetic related. here really who quite in none who worsening just happened old And and considered drug are of types to SAE unrelated patients

So very standpoint overall, clean from safety very, a

Operator

next line our question Luca Capital Markets. the with And from RBC you. of comes Thank

Lisa Walter

taking This is for for on our thanks questions. Luca. Lisa Great,

and Just two prophylactic this on the questions Wet to totality the between more alone from decision elaborate or due include you diabetic if data can retinopathy Was of the to steroids. us. the Wondering the the diabetic had anti-VEGF injections, X X And respond characteristics to did noticed I no that bit Cohort on the and X patients appear and baseline they prior the Cohort better. as well little a AMD but program. patients retinopathy

anti-VEGF just wondering experience going will who forward are high for was enroll So you versus patients cohorts anti-VEGF the Thanks. dose naïve.

Ken Mills

Lisa. Thanks,

of both this well in more the the elaborate so a of as ongoing, to all the aspects on Again patients. study one have, nice as Wet advantage decision data ALTITUDE to AMD AAVX of we study So that's take we DR having expand.

the at So chance and same up I safety and with partner it think really it these look a for was interim on go dose our X we with exact pleased gives in data, to and decision dose dose level AbbVie very biologic effect VEGF to at as in concert and retinopathies we're and to AAVX. tolerability very natural these us high driven a we also did look

of types some as there Lisa question second the sure do previous is you on were Yes, there make of window. washout had a one in certain these we studies treatment. we The mentioned who previous What is cohorts in patients treatment.

"treatment-naïve" at getting some minimize and So other have factor even presentations had in thinking not. into risk patient actually from confounding were versus least yesterday, the months they six about that not not interesting anti-VEGF if at is any treatment-naive programs, least to injection there. There a of

forward. same So follow that going with approach we'll

amended but a that anti-VEGF recent prior have a haven't not we So can of within part the protocol timeframe. had where patient

Operator

the question our of comes line And you. Andreas Wedbush. from next Thank with

Andreas Argyrides

taking question. thanks and for our morning Good

says the So couple Could it criteria. bottom we here. for follow-up. received of on determine anti-VEGF how patients the and standard-of-care us patients tell And of receive a us, therapy what's so that if have I Slide you then X, a

Ken Mills

Sure.

ability disease with of and and sponsor end developing potential of of a sustained should from patient to key the certainly prevent in that we agree patients the the So complications that's look we day these investigator it's of one with patients course, the is we and follow complications indicated patients look and benefits this it's the they the proceed at vision-threatening anti-VEGF treatment if complications. longer for treatment to the with of for investigators treat course

footnote need that group, that we alone given patient we So the who the did. up a who and RGX-XXX treated versus patient in a in patient had the with see wound how we And treatment end. had treatment control with did course the XX have that the one at on injection in eyes early elaborate single anti-VEGF in the X-steps you

shift same thinking in and Peter in and perspective of the data, retinopathy you this on XX this you up PDR where those severe of that totality patients how you to the in of of population imaging risk in patient also Lejla do realities look risk including if of directionally, outcomes high of then terms have of of retreatment because this the high-risk think terms they're is. how needing data at going at advancing. goes of you XX diabetic and aspect. terms DME discussed kind look very that we're and goes a the How to it think we also that So What of you of the of at presented that basis the as treatment.

Lejla Vajzovic

out or that PDR protocol. Seamlessly typically retinopathy to and have PDR moderate you this significant not progression of that, have especially included historic mild the risk also in early may there's there or that trials with a inclusion, treatment be And you pointed trial. diabetic are on gains doing with in

wonders to was diabetic was really quite you that soon So for the PDR pointed out, also higher severities mild whether response patients. it period enrollment The retinopathy the and any one as did receive treatment after patient injections anti-VEGF was there enrollment. early one

there progressing right so already that complication by for well explained that. are to set think and be could I up unfortunately very So we

Andreas Argyrides

And great. might previous touched this bit a on here, then in but have Okay, you the questions just.

higher How about, than greater are to the X-step So and you or for X Cohort improvement from for what in are your thinking the, the expectations X observed Cohort decrease X. Cohort dose. you. about observing thinking Thank

Ken Mills

will to have we course. Andreas, see of

the why So trial. we that's do

PDR We moderate have also on dose PDR, higher expanding mild that. level not that dose one what And to only the opportunity a and and with mentioned, stratifying seeing now based want to look include encouraged but see. Lejla are we at the we two, level and as we're also so are to dose to this level

data response and as well as here more So assess we really are have assess going lot to more a in dose learn we it and can AMD to really how lot both. Wet a from patients

Operator

[Operator of [indiscernible]. next you. Our comes from Instructions] Thank line the question

Unidentified Analyst

on the to improvement from for potential Good taking One you months. morning. XX Thank X question.

compare that months -- have what to improvement XXX? the you six shared wanted that beyond see on news to can to your and So and to months progress be expectations continues basically about would if you from how data the for six know previously three standard-of-care said

Ken Mills

the then going able and for we presented certainly time longer these cohorts that look we these on cohorts, in about being to and have we points and term date to time cleaning compiling the points So follow-up time. forward, in to forward existing the report and care future including one-year

what's think the in treatment without of aspect the expectations, severity that other of it's as due our guests the fall better. And to diabetic of and do think not retinopathy know as diabetic patients far study that I use small patients highlighted, think retinopathy done which impacts illustrative factors is been better, reasons, that As one And with general, that one I realities get off before. just have returns. control future as patients treatment Peter existing repeat can How frequency get we injection that therapies in would be observation severity highlighted. percentage decreases in

highlighted. complications ultimately severity of standpoint, in think a worse puts bar powered that developing a what terms it of hitting risk direction us and is good studies we blinding bigger also the investigators regulatory matters regulatory imbalances in while a and which right, really in favorable the getting to take shifting into out So, and the patients hitting keeping the that to and the are the terms of a the patients from position Peter

Operator

I'm questions. Thank you. no showing And further

remarks. with to CEO, hand Ken the closing Mills back any that, for So I'll call over

Ken Mills

I that. Vajzovic continues their perspectives time for grateful perspectives reiterate early from to the paradigms in high in very us that through to new their I DR weighing for walking and on and operator. just really trial. on the interim treatment Steve REGENXBIO overview a want in and at Thanks, which ALTITUDE Dr. both level. data thanks that, Kaiser to this morning, appreciate perform

both as developing the and potential as applications data to amazing had with from the innovative our patients' portfolio. impact have rare over We months vectors, expanding the understanding updates one collaboration our therapeutics significantly dedicated several team generating new to that past have and well care AbbVie, AAV AAV an of We've disease the that's eye lives.

on guests and foundation a Again million capital to clear anticipated to all commercial the to we achieve vision over pivotal so data keeping a much look entire of five from rest We ahead through or multiple our XXXX. our path forward XXXX very license other and day. truly a programs and as of into into and advanced great Begin team our updated to fund filings Therapeutics, operations and thanks our have definable have milestones, by pipeline finish and Xx'XX internal mission progress XXX to have believe the and our at products look the we XXXX. to year. We you stage AAV

Operator

today's concludes you Thank you disconnect. may this call. Ladies participating and and conference for now gentlemen,

REGENXBIO (RGNX) 3 Nov 222022 Q3 Earnings call transcript