RGNX - Earnings call 3 2023

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Operator

Good day, and thank you for standing by. Welcome to REGENXBIO's Third Quarter 2023 Earnings Conference Call. At this time, all participants are in a listen-only mode. After the speakers' presentation, there will be question-and-answer session. [Operator Instructions] Please be advised today's conference is being recorded.I would now like to hand the conference over to your host today, Patrick Christmas, Chief Legal Officer. ahead. go Please

Patrick Christmas

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

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

Kenneth Mills

Thank our everyone joining today's our Pakola, Medical to end XXth, financial of be the call Chief is on about and large product candidates long-term.I moving sharpened forward. differentiated, be what the our resources generation support on we Earlier pipeline and restructuring our overview afternoon Vasista, will of you, this focus focus capabilities update Steve will and to the of to and Patrick. want the for Financial for expedited, value are provide I'm for Dr. and our line our meaningful Officer, Good clinical call, can pleased for an strategic be XXXX. results commercial clear soon us. At we work an thanks the mission programs. then September means where that with ended corporate plan plan. begin gene the prioritization quarter on potential And began improve curative provide of today, an therapy. lives enable updated up questions.At will Vit REGENXBIO, through will a opportunities to Officer, REGENXBIO explanation Chief third opening

RGX-XXX positive the being wet treatment capable for Our the We've AMD developed last a macular age-related MPS and treatment Duchenne, injection potential RGX-XXX and clinical prevent RGX-XXX where and first-in-class or II ABBV-RGX-XXX programs. efficacy as treatment large single Duchenne for is product.During the months, believe key progress therapies exciting support is experienced for a the received for X in RGX-XXX treatment treated these highest of with a Syndrome.Now, treat programs are AbbVie, of of the that from to in collaboration Hunter we've patients Wet the priority of elements the each us to with become is gene of program there therapy RGX-XXX that and need believe diabetic degeneration data updated in These on retinopathy expedited and there's reported gene a and be the feedback and new the of to encouraging market of meeting we're BLA. plans BLA meeting enabling II. FDA for from to And a expediting potential constructive RMAT FDA announcing our program. milestones multibillion-dollar supporting for about the accelerate for we retinopathy that therapies. we've care the new development best-in-class recently this and gene Duchenne progression today, data science very very data diabetic differentiating treatment the features RMAT of held is from strategic MPS the avenues FDA REGENXBIO.We encouraging confirmed with retinopathy. a following just with therapies, for supporting is demonstrate treatments for unmet investigational Initial and for how and we standard from diabetic has the with encouraging that unique its a multiple to accelerate October, alignment

remain gene II.In efficiently critical. create market, accelerated the challenging we is to XXXX on track the support treatment and ability a the So in value approval MPS therapy pathway. filing would to first RGX-XXX BLA be today's using quickly for

support received, BLA review XXX we've of BLA RGX-XXX delivery, BLA significant Generally, at PRV observing resulting of MPS our for guidance. approval for nearly enrolling that the creating for for shareholders updated strategic and Importantly, potential lots to highlighted, runway to achieving sources to of allow we RGX-XXX, planned dosing payments additional value just our These eligible that receipt.So generate development time funds, points. for this AbbVie, pivotal sales and voucher non-dilutive achieving the a hundreds into are our And not noting are in to are bridge trials sponsors value would such program us as cash additional the also milestones initiating pivotal then pipeline II.Now if These include more over for to and instance, suprachoroidal million in in milestones, pivotal it's been would additional trials, I decisions extend current between number key and of operational suprachoroidal value. to trigger $XXX our RGX-XXX RGX-XXX. now over the runway partner, plans completing partnership to pediatric also product these successfully for as as achieve our a which and the using programs I'll allowing deeper potentially profitability.Now order milestones of for approval our can program, ensure represent XXXX, inflection and through in the to these qualification clinical resources allocated first of can for assets, detail. much to Steve? these even us initiating these performance in greater our extend able call to our he progress accelerate of worth certain more restructuring of the runway development unlock milestones Steve so to progress collaboration guidance and are and a us.And approvals milestone from most prioritized such to the be portion millions valuable assets for review our a intended milestones turn of of some million to meaningful that XXX receipt would the to

Steve Pakola

which because XXX anticipated submissions population with AMD fall XXX is of cases of have events,Importantly, with escalation under presented delivery II well receive collaboration data XXX those achieved initial via DR, Cohorts poised AbbVie XXX demonstrated potential which is patients anti-VEGF market, improved and suprachoroidal One-time in to plan in to Japan, of the by of presented of with X X in and being we dose We on vector significantly meaningful VEGF.The of X level from collaboration in XXX dose Monday, at late of XXX regulatory pivotal patient observation or routes a at before safety of dose opportunity eye and from grow to retinopathy At through the study X visioning given weekend severity and half the ongoing one-time lechnopathy for this about as Phase physicians reductions can course threatening Moreover, short to intraocular to Retina with encoding market of wet levels of treat of study.AAVIATE X treated administration. the a patients highlighted AMD. the year.Patients in-office the I'll dose Hawaiian development controlled in with from treatment disease the for begin population the and being level tolerated trials even trials treatment X Ken. the the ALTITUDE dose XXXX.We prophylactic trial in controlled beginning levels in XXX the support at and clinically subretinal our delivery antibody resulted a assessing of this evaluated you, retina both The a our on risk NAV therapeutic to therapy. evaluating did developed AAO believe AMD at global with dose of data opportunity ALTITUDE prophylactic dose X be steroids ongoing patients wet to events the of and prophylactic study, zero recently Wet level call severity. XXXX XXXX. the AMD evaluating AbbVie XXX X inhibit excited wet enroll the that of diabetic active ocular US, XXX of data is XX%. ASCENT, results past ATMOSPHERE of DR continues dose X with from XXX% AAVX escalation suprachoroidal the and the also deliver Europe are AAVIATE the and utilizes steroid the positive reduce presented treatment very levels. present subretinal is DRWe're short of Cohort included and after Eye in or July treatment in via we XXX, most wet benefit the XXX. program size Thank an administration. which the the baseline that fragments in with the most X,XXX safety these ages. in-office X first that gene gene in diabetic of is supporting exceeds steroids to course to NPDR of for reported delivery which for inflammation.We in Cohorts injection total administration and vision-threatening disease AMD in inclusion stable following global suprachoroidal Meeting improvements expected was full an X X-month drops, profile not

one-time potential in-office by the the one-time a are retinopathy.Moving treatment differentiated for of Duchenne patients a gene RGX-XXX We developed to to promoter. Duchenne. treatment in for to deliver product, as C-terminal that a transgene injection with the therapy found skeletal elements for the a being dystrophin.RGX-XXX microdystrophin encouraged of potential diabetic RGX-XXX designed this AAVX highly of data delivery and now the throughout is muscle the for muscle-specific naturally NAV using well-characterized and and occurring gene novel includes targeted heart our and is a support designed expression a functional vector of domain,

appropriate serious quarter XX.X-year drug-related to all for the to had the levels results the muscle the for LC-MS with by meeting in we pleased MPS from were of alternative of well control. time also data XX.X% AAVX observed, I'm events with X presented.As microdystrophin showed X. results microdystrophin recently creatine microdystrophin to was blot comparable patients measurements with an XX.X% to the method.Overall, patient, that western treatment boys supporting by to may when earlier, the observed A first interim available and reduction Duchenne eligible from of the a RGX-XXX measured for FDA adverse measured tolerated as in AFFINITY evidence RGX-XXX preexisting X.X-year patients of to I/II Ken shared held an and compared mediated other Initial treated clinical shared data AAV CAMPSIITE confirmatory in of due a expression no was was be expression whom RMAT robust expression constructive and trial. FDA align Microdystrophin of XXXX. discussion. strategy, represents treated manufacturing we CK were cell microdystrophin in first with adequacy the to for results therapies serum levels do share XX% safety serum from on showed expect clinical measured Our study old with who October, compared World the the to expression continue very were that data our our Muscle I/II/III share for X presence of of kinase in well CK neutralizing baseline the antibodies.At and reduction on held localization database from capsid Phase design.Patients able of not the baseline Phase was The top the We follow-up, trial we was membrane.In control line and XX%. from this data positive to patient improvement. or RGX-XXX old be

to longer committed no programs, are for and partners leveraging or including potential significant our to conclude, working XXX in and programs. advance continue XXX we alternatives data trial progress private prioritized moving are forward our While public of across we them, with our in the we rare all RGX-XXX, and have updates believe therapies strategic to these potential partnerships.So we progression with made pipeline finding as programs neurodegenerative

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

Vittal Vasista

quarter of was XXXX.R&D XX% of Eye in strategic for Thank XX, cash as expect in operations the September million XX, third and collaboration.With to least $XXX our the to balance workforce fund decrease we operating cost the driven the XXXX, compared totaling of the XX, under million over securities is equivalents ended $XX half expenses runway upon at that, final will received marketable X developments XXXX as to any December decrease to September our impact primarily XXXX. that I from during plans million second activities of based cash, now months with company's XX, September commercial achievement may of Care ended excludes operational current to were reduction back the of of cash milestones marketable X We equivalents The you, $XXX Steve. reimbursement XXXX. provide reprioritization under from Ken save to expect cash, third years. cash turn next today, guidance increase compared the fund $XXX $XX AbbVie used and and an for the into XXXX the was the or plan This collaboration.With securities, on of quarter primarily be on development payments attributable and announced by XXX AbbVie quarter million XXXX. the the to earlier our REGENXBIO million cash Ken? to $XXX million The thoughts. call

Kenneth Mills

Vit. Thanks,

outlined. meaningful us cash look value-driving enable our our programs, runway ahead, now we As extended will milestones for to reach I've which additional

sense first We to our include fact and which significant strategic and think for and resources expect valuable want sale X, the accelerating to most the for level the the data these have plans PRV X of of extend III generate additional we the believe would we reiterate top AAVIATE partnership level to level to development execute from questions. success.With from bring of in implemented could, achieve value, to that, pivotal important our trial.I And our value want milestones the capital to that anticipated that now REGENXBIO turn for of trial Phase CAMPSIITE at focus dosed Operator? even operational to wet allocated that expected data that AFFINITY data we trial, can from strategic we our AMD unlock medical our also result at lot like renewed Duchenne a more all runway conferences we dose to is that updated focus, a these of access to dose sources assure extending that runway I new XXXX.This and well-positioned recently a of AbbVie of a are additional as can talked the non-dilutive are updated perform other or line as call near-term for long-term value-driving shareholders further.With intended I including the initial over suprachoroidal, a high of about plans cohorts X-month from an catalysts today, assets assets half mission. to we on as we'll continue upcoming announced our

Operator

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

Huidong Wang

of know question one are just can I follow rule. question will I I there Okay, ask. tons a

is one of terms selection question efficacy I DMD from you program endpoint learn the experience development bar? and also Sarepta's for So can have what clinical and regarding in the primary

Kenneth Mills

us Thanks, Society Obviously, the accelerate about believe good regulators been Gena. what program, with with about ourselves in to how continues dialog This to in our microdystrophin ability program.I focus the RGX-XXX engaged dose question. especially both think that phase that been of be is we're pivotal clearly the since Muscle we've we've select a own year as data, important we've boys. that to we to guidance clinical and emerge been for it's and think an landscape to given it's World start next and have helping encouraging seeing able a we

C-terminal of that setting to setting scoring that pivotal part think the approval boys. how in that function.So in the and the even -- endpoint domains about used borrow long-term and use the respect looking a microdystrophin to I to continue is think since And a that's accelerate commercial endorsement the is in domain an still others. sort I much about that it boys in function, hypothesis believe it differentiator engaged but beyond space long-term think than more clinical in important the for on certainly think certain meaningful it's the we clinical We something viewed function.So be design at RGX-XXX how of a we're that differentiator. response to to further is a can and of with in to surrogate support that where trials we are very I having Duchenne something increment, accelerated entered the support

dialog happening landscape more are for indicators great improvement. the clinical that that really and level, the position evolution an prognostic what's our We're actually regulatory have own recognize to in next terms people regulator of of are seeing at data much at to and having that puts year some the both very these some opportunity dialed into on package. a domains the think reliable our in starting think on to the I about best direct of start and executing us level

Operator

Our Raymond Dane line comes with of the James. next from Leone question

Dane Leone

on on I that? run to for There's for and further can be can control me whether and shifting DMD into the going a obviously it to that mean, the there's landscape am ALTITUDE AAVIATE problems I debate said and been hope the seems could of I you support way One, you've study moving shying used the glad be during ability differently, for of for use person. two, lot sham in studies a therapies therapies. may forward. subretinal longer-acting controlled comment XXXX. studies away regulatory an potential pivotal from One have anti-VEGF the And then that gene actually FDA like is the in studies cause didn't

Kenneth Mills

Thanks, I let Steve to the Dane. about landscape. guess, and I'll the probably respect regulatory address with AAVIATE. comment ALTITUDE

us, potential DR $X.X that come dosing occurring a think AMD and today us, for off the the up respect up that these really for I of to those milestone proportion on can an view be pivotal REGENXBIO, and and confident about up right with into supportive seeing likelihood study.So improved that runway plan we're, transition, we're of update want achieving excited that's I the starting events and been as AAVIATE ever to is the regulatory XXXX data think, strategic of about milestones events the the of that just the of part? think, that in retinopathy of have the to for as over respect of suprachoroidal beginning increases to with extension runway important additional strategic to is with particularly the to science are I decisions of particularly and respect the suprachoroidal we diabetic there.So respect have we transition of extension early some the unlocked.We focus these supporting patient phase billion current capital the I you of occurring for the to transition wet been large value with delivery in is plan what's that for events.Steve, we now future that with in think updated do the part shaping and associated that coming likelihood we've think pick have I we're for the data development

Steve Pakola

a meetings Yes, that a here been point this not I discussion other that different Dane, may considering surprisingly, getting preserves suggest that's least even or level of an sense an discussion ways preserve on there, visual active able that's we're component weekend there's key injection injection a intraocular programs, held And there's to big that at intravitreal the for active of issue in sham having the that address AAO an masking injection' to think for injecting fluid that there's getting then control. approach masking.The also and a within the eye, this Hi, a of there's drug come axis arms interactions of regulatory something FDA's aspect couple up from of the that the and they're to the into are, where the masking due arms. patient not your challenges X because 'sham FDA, the such at control issue we're of we've of fact this between question. as actually aligned interestingly a not dose on the suprachoroidal was be and a had can actually for given see of some and our FDA's giving injection, in to injection.So Sure. thanks sham particular, past ancillary other to this, maybe the the points view were with actually that that's belief intravitreal a real the relate that general

consider. that us fact approaches injection we're shields But really the several an fortunately not there's intravitreal can for that So this us, from companies issue.

Kenneth Mills

the suprachoroidal application level of of trials I additional delivery is, going giving I Thanks that's the of think, support detail the that for these the going on types that's question, evidence things. work of and in is the inside into about Dane. company thought guess partnership and the to later-stage Steve

Operator

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

Gospel Enyindah-Asonye

is Gospel Vikram. for on This

We looking capital? have partnership? a XXX. for a for potential a partnership mind have time for to one What question; you would in you for line of establishing partnerships Do RGX-XXXand outside be

Kenneth Mills

update capital assuring are to strategic RGX-XXX, strongest and and focused the really pipeline the today we sourcing achieve in for think on have plans Gospel, is RGX-XXX value RGX-XXX we potential the today. that what

XXX forward. respect the going with things, the become be contribution short but plans to a discontinuation will to will other partnering, going a term that that's any viewed exercise it something So of to in result and as clinical for development opportunities meaningful and work operating looking won't for in be that

Operator

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

Alec Stranahan

relates positive interactions expand Just for age design it XXX to cohorts? pivotal particularly did after you dose you've me. one get the you the upon Level as study the FDA well, from different Could input X and maybe as on had update any the

Kenneth Mills

the Alec. for Thanks question,

as what's with for to to general. microdystrophin of I data RGX-XXX think FDA a data that it's with of landscape the dynamic in sort of and and process to because a contact microdystrophin been thinking about and of is on understand that's the class. respect very us want in respect other We FDA our happening how sets things the with evolution

there's going BLA additional to for see of people I a a I instance, landscape time. that rich incredible the XXX.And a pathway level safety and manufacturing, microdystrophin feedback facility Alex, to really X, dose respect is around -- is think say, at and talk about right? this us RGX-XXX with and that a in dose early something lot to on, is with the between manufacturing sort with and selection.I information opportunity that on data at think and also a RMAT on expression also through of and us, making sort to update leadership for between we with for get mean, to microdystrophin we're FDA also general.It's opportunity about let's our manufacturing is process interaction XXX, and We're readiness and interplay of what of basically meeting about path an for for respect us so XXX the working

up to was setting well that think the X parallel modify a plans X to I operational we're dose levels clinical before we're us moving being scenario And number acceleration. best just about the thinking enrollment that think are example into at. of for protocol of with I patients strategies that the about reduce the able to from that and

I think -- that that, informed respect informed was alignment with the that It was our to to data. safety. IND It with was by meaningful dossier was FDA informed I updates having and by on by was something think, a signal.

Operator

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

Sarah Krupp

is Sarah This Hi. on for Ellie.

around studies near-term guidance. I thinking about? should that what potential then milestones they're your remind quickly other of into the milestones not you color Just on we would suprachoroidal. And in currently be know pivotal from any Can with timing AbbVie, us the milestone there be moving for

Kenneth Mills

given think into that large associated and suprachoroidal like the those the things with starting be for we change for we've I the of data over questions likelihood that to that we've types the with to phase.So this eligible Thanks are sets a going And guidance about -- to them development or view updates occur runway some things we been the really question. been we been partnership a already respect have the partnership the into say Sarah. that across where in guidance retinopathy disclosed seen obviously, and decision exciting the $XXX can with we pivotal are associated that is of transitioning that on thinking decisions soon. made that data answering some those specific milestones mature of within that that recently that increasing is programs million they've made.What can proportion think I now Sure, data those with has have to about how operational point timing are runway have diabetic of now. going are milestones the of from Yes, we've and

update the half this data tried think, Eye of the things XXXX next into I coming runway achievable.And So with in frame the also have These that to overview. a year. in guidance of now Hawaiian again, I second are the beginning we are

strategic think start you program company can and for to here 'XX, opportunities of milestones where sort even talking we of and we're acceleration to hundreds be for the millions we're years these I launches.So with dollars the to the think potentially our about are bridge these together the of occur are a that are 'XX, product some can the effectively filings even subretinal how now assets if interplay like talking the instance. update way of potential those additional of And further of about whole into obviously, bridge ourselves all having product currently row approvals years to and of opportunities really BLA that in plan profitability. of that to to think to coming things company, Duchenne capitalize program, about milestones here

Operator

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

Lisa Walter

Good.

on this maybe -- for Lisa Just one Luca. is

is know on XX to DMD. years We age. the AFFINITY maybe enrolling up patients Just trial one of

data, younger children older EMBARK appeared discern to children. it the harder versus from However, in a benefit

the especially would the XXX that that there suggested wondering in children, older there expression Any you just was when a that show gives Western appeared confidence patient. because blot benefit So in higher will helpful? what color younger be it

Kenneth Mills

already. I we look, an Thanks, mean have older patient Lisa. Yes. enrolled

from C-terminal here we're biological a that in the sort think of expression, be front that improved forward into on older continue are and our boy. we've as think show ]. able going to-date to think in able any are plausibility C-terminal [ remark any to that I going just RGX-XXX meaningful that patient.I microdystrophin update function.And differences certainly acknowledge as expression the in other the ] progress better microdystrophin expressed of other of perspective, to about springs treatment be better is cassette translate of domain that see with than gives to the and us really of unfold domain the that improvement the to to but differentiator design so scientifically, but And I amounts good treatment We [ seen

do we data X in older and strong and domain, with that to there. will age and for continue profile is preclinical C-terminal functionality what enroll something that from view the emergence ultimately, range that our in also in think to the an on of evidence clinically feel that's our helping and we've continue really the is that to the patient. update we X more on sort strongly associated to we commercially.And to seen the opportunity benefits going X it, So in of we've But inform data reported to that the validation be X the there's important early

excited think, we're range. something Lisa. I age for that about is that Thanks,

Operator

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

Brian Skorney

programs afternoon good Hey, the my you're It like sounds administration. everyone. decision taking question. superchoroidal Thanks pivotal for point with on your getting just

pivotal and of DR the point.And other? wondering terms earlier in just and these So factor here from Cohorts of of you make of and limiting Retinopathy, AMD what's X sort Diabetic do decision you've sort wet ALTITUDE need AAVIATE the X-year at X Cohorts to very enthusiastically to time? been that see you really the rate kind than one trigger and forward same a forward the program like in X Or move potentially indications moving of will the talking about data

Kenneth Mills

question, the Yes. Thanks Brian. for

And have lot recent we there. a like again, think, update. I of the there's data about recency enthusiasm effect a

and we coming have in the then been communicated been now AAO terms Of few new has only last focus respect think the of -- a a few DR was in days.With January. course, and I AVA the us update and data just for days in has ago, communication

at Subretinal, vision difference the we them indication respect they variables for us of the but AMD.And are kind data an needed point that from the suprachoroidal, that diabetic different transitioned to first different of obviously AMD from clinically now the to AbbVie. any with potential agent, the of suprachoroidal pharmacological advancement decision-making all wet and in Therapy of in slightly because large the AMD but wet variables in additional a how bring of always sets that to different delivery this, the all been derive inputs. with retinopathy. equation wet with are pivotal look program path, and I wet beginning the in pivotal, sort or and sort and almost each that's is because one strength pivotal the to have Yes, respect with of into and the Diabetic into program AAV same we last of be the phase.I They for like that interrelated also AMD think on AMD of and are to sort think out going in suprachoroidal, company the market robust about separate the rely to consideration into wet our they're to modality would we has the a Retinopathy.I the independent decisions of transition though between like difference market evidence case they mean a the have different a Gene phase to a same and

is that's same extended of of reinforcing about runway come they data think time, make and terms alone, success as value independent way coming those our the to just but things is newer with extended same been that of RGX-XXX that likely we've strongly and at and certainly it's made investment that some improves could it's a in partnership AAVIATE realize really realizing happen transitioning opportunity these continue about some encouraged and the excitement of are the become of it's been have feel value possible at that runway the probability to out focused with think, to the to milestones are how I in the and that the started here, milestones meeting So today forward.But I that that ALTITUDE. but the about of like pivotal.Therefore, an monetary with of decisions improved, would longer-term really continue also investments the right, we -- time, variables that in the associated these message

for here come XXXX. excited into things we moving to So

Operator

Samimy the Instructions] Annabel next line from question [Operator Our comes with Stifel. of

Jack Padovano

question. for is my taking Thanks Annabel. Hi. Jack This on for

value the XXX there? wet FDA long-term durability is AMD, might and Subretinal in treatment? for not So are of elderly may proposition right of you the patients for that And that looking the what's think pricing establish considering to of what see formulation benefit payers and a be for of when the the these many amount here safety of maximal follow-up time this,

Kenneth Mills

you do here? want part the to Steve, regulatory take

Steve Pakola

Yes. Jack. Sure. Hi

data patients having but ongoing a both activity AAV confidence end And really now AMD back own that gives treatment first finalize that's the somewhat have is to to clear breaking diabetic have So, of data, need of example, the importantly efficacy, sustained the decreasing aren't efficacy to for complications safety pivotal prevent that by the think by taking they proposition, beyond same as burden. from durability the Phase because allowed question, terms wet here standpoint And burden, regulatory and just part meeting a obviously feedback the unmet of predominantly having through class burden other burden.Really, is We've big far data also by safety efficacy I value barrier efficacy. longer is out the ensures patients us sizable more from on than durable data our the to the I/II we going that lot our of already efficacy anti-VEGF have And eye actually inform of in our years, outside AAVX.And a and an and vision-threatening of on aspect.We'll from the gene us gotten II out in of getting and that value the proposition as visit informed your it treatment program evidence. treatment duration was also now even standpoint.So, good safety follow-up pretty retinopathy which the far the even good simply very term aren't then that base lot X-year have years, as of with a therapy duration from requirement database. serotype both I going dozen programs, a the follow-up. X need they that shown hemophilia as think also based durability Phase these from

the visit various of and proposition, layers complication there's and treatment burden, outcomes. So, actual value vision burden,

Kenneth Mills

pretty excess associated a that related years. AMD might blindness to Yes. a on basis, that's to drug.So, by And Jack, I well-established in talking life when cost I an -- sources that's add, of think that costs wet the order XX we about from of expectancy of with and be separated patient, would $XXX,XXX the can AMD on the cost about the of wet lot think a be you're it's different the is year XX that something annual of

compliant the and with there the having a recovering existing as with think cost of a need that's I productivity the -- in world, the well, is real treatments effect patients those vision on there's being the is because people that obviously, to lost on significant. the it staying respect has the aren't that for the that families basis and effect

on regulatory evidence, just there important like continues how think brought And commercial I for It's so as durability but everything.I a is setting. in the basis, up things which the Steve grow. also a backstop X-year to

Operator

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

Mani Foroohar

[ -- you Obviously question. the fund obviously do. but taking the thing right for Thanks guys. Hey, to to have ] decisions make,

around in immediately linear a think is? expect years. it post we million release beyond on Or where period, loaded, should sense should give of to that's couple a press a OpEx is cetera, loaded basis? savings, more X-year a And $XXX over that about on little drop how be first is the tempo and years? we us fairly couple implication quarterly the of longer back little that that, something the that more Like term? bit of front about what you of Could detail outlined XQ You've in little et starting a of

Kenneth Mills

want to chime this you one? in Vit, on

Vittal Vasista

Thanks, Sure. Mani.

as would be gets $XXX thinking that you're plan linear going as I forward. think our as it split into operating million, not that much about it slowly XXX%

that's possibly obviously, little discontinue more Some the looking a at would then the out coming And of other programs. front-end it I've year, will XX% start lumpy with think at I better analyst is what determine on know, and lot the to on of quarter than I loaded.And markets you as be have what's think estimates we and tracking seen, be looking certainly the that of this into run OpEx, little talking idea then, XXXX, reduction, then run higher against.And with a what an OpEx what rate will that the for we non-dilutive a be appropriate bit And bit a headcount side models you'll seems kind rate, capital OpEx unlock obviously, as of perspective. their opportunity the of different models finally. as that we're we're next about the to about

OpEx, but forward. they I going relates pipeline commercial also maintain our have opportunities going post our become also as we'll it think discipline to to as we're start to programs think XXXX, then moving about still some forward to

Mani Foroohar

That's I'll Thanks. hop off. helpful.

Operator

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

disconnect. now may You

REGENXBIO (RGNX) 8 Nov 232023 Q3 Earnings call transcript