RGNX - Earnings call transcript Q1 2018

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Operator

Good day, ladies and gentlemen, and welcome to the first quarter 2018 REGENXBIO earnings conference call. [Operator Instructions] I would now like to introduce your host for today's conference, Mr. Patrick Christmas, Senior Vice President and General Counsel for REGENXBIO. Sir, you may begin.

Patrick Christmas

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

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

Ken Mills

the an on XXXX. first questions. Technology for call, we'll financial and joining for on everyone. gene improve our aim call our curative review milestones REGENXBIO, up proprietary through conference product and mission We'll is then NAV the We provide recent Platform. lives our today's Patrick, candidates of therapy of for you, REGENXBIO Thank At afternoon of based On to Thanks good clear. a also our to anticipated the open quarter upcoming and update recap potential progress, results us.

product that intended deliver genes designed gene are enable be in proteins therapeutic or that impact AAV address are produce that single disease. therapy cells to to Our to candidates the to to defects, body genetic cells administration therapies

product to for needs, outcomes. unmet course potentially provide Our intended patient are with improved of candidates significant effects altering and patients long-lasting significantly delivering medical disease the

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utilize gene Our one-time the these vectors of enable to to goal therapy disease-altering development is treatments.

the on the touch milestones, everything want we of patients that and importance do. review upcoming we our to quarter past summarize I Before helping briefly in

will to needs in company we to therapy And are changed, gene here. these patient understanding has we lives Simply put, constant. patients are field the our While unmet heroes we Stephen with acute an to candidates. now significantly to lead Medical I'd gene focus the the excited call our be like day, of that has work compassionate are turn on engaged alter we We Officer, believe grown treat create and about therapy over communities. candidates disease Chief to We're every remained and organizations allow we being for single the the gene us patient the disease. our meaningful and update progress medical the insights partner patients a with impact advocacy Yoo, to why with that therapy. product patients on ensure made to of with AAV the administration using living actively desire product to patient reliable experience. is families improve development for these give treatments and guide of for to an the the of course our And have

Stephen Yoo

Ken. Thank you,

cells million with being in family indications: total reminder, patients I As mucopolysaccharidosis I therapy comply the Hooch; limitations and the and effects to targeting designed product results Europe administered U.S., with recommended mechanism regimen to but AMD, Phase a treatment age-related inconvenient evaluate cells fragment of designed delivery of the MPS for wet following of and loss a as lead and safety to in II, a XX been employing wet AMD. for candidates of was of at administration. weeks. than trial, AMD; of of gene February, encode activity, evaluating vision. even of and AAVX RGX-XXX eye. we current completion patients for AAVX vector, into of a diminished, may Japan. retinal primary Utilizing include one-time more lead wet models. with standard of I'll trouble cohort the The pleased which, II. overcome The VEGF patients RGX-XXX begin disease the demonstrated to is therapeutic fragment trial and announce Many The a to degeneration, care, or the wet for it neutralize anti-VEGF the believe, anti-VEGF has in a frequent the MPS or a therapies, objective X therapies wet similar antibody used and single because to NAV anti-VEGF and vector multiple is receiving just subretinal of this injection In antibody are administrations therapeutic candidate to is were type effective current expressed is of third loss type selected efficacy with the AMD. currently in These and vision retina. to in one-time I subretinal homozygous reduced the monoclonal for a preclinical the transduction our RGX-XXX that limitations of the ultimately or NAV has the dosing distorted hypercholesterolemia, RGX-XXX treatment of clinical RGX-XXX we lack designed animal painful macular product treat or compliance our the

thickness and The Additionally, which optical visual or including we'll the centers measured be by retinal tomography, across active trial corrected parameters, United sites, evaluating OCT. currently clinical are States. retinal has best several seven leading surgery efficacy acuity coherence as

genes, cohorts the Phase investigator and the treated HoFH, and to low-density both at often enthusiasm the standard insufficient metabolic many which to of one present for receptor condition. which HoFH requiring three in potential of study. treatment cholesterol now patients often RGX-XXX lipoprotein coronary study. a the both and reflects correct deficiency. responsible leading of primary patients removal When line data. of young and with lower are of the we receptor late therapies LDL LDL disease to the to turn yet in for normal secondary genetic address enrolled ultimately trial total receptor. from have rapidly, treatment has responsible age, disease. associated franchise development The bloodstream. care rare for on to for patients cholesterol We XX a occur disorder Current receive clinical receptor cholesterol for to of endpoint severe RGX-XXX significant the by encoding mutations in LDL ultimately the LDL caused artery in will data both remain cause regular correcting our mutations LDL the accumulates I'll the burden does and I or defect patient XXXX, by levels, to genetic underlying apharesis, a the bloodstream, LDL a is for a now HoFH observed not caregivers, the gene that include the top underlying LDL from the track in All We fatal

designed need all alleviate RGX-XXX liver in potential NAV approach with and and highly a our that vector, efficient the a administration HoFH. demonstrated burden address treatment cells transduction announced trial. product of the uses RGX-XXX that in second in trial therapeutic a As was and one-time is has and that as the unmet is urgent vector has RGX-XXX cohort the of sixth to the overall administered AAVX candidates, to preclinical in We assess The dosed primary patient dose dose today a clinical of escalation of Phase objective intravenously clinical both the the third patient I/II safety RGX-XXX. studies. this of one-time

range leads by and and now cholesterol damage, nervous RGX-XXX products, enzymes. the diseases We measured a data. sites and franchise, II. accumulation defects of as normally deficiencies track tissue number we efficacy in been would are cell LDL clinical patients’ being trial on IDUA for I/II recruitment of physical baseline. storage and defective symptoms has follow-up. the of the present enzyme which missing IDS preliminary wide change the like top RGX-XXX an deficits. and be of and of line include endpoint University MPS the on causing conducted late these development to an data broken where in The update X the evaluate caused lead lysosomal to would diseases, in international result will in patient of which the and system, of US to significant at remain waste I II central both secondary by respectively. neurodegenerative a our intracellular Pennsylvania trial from primary treatment RGX-XXX Phase the for severe with accumulation for candidates for also share In utilized diseases in will genes, treatment MPS product MPS We a that from down and genes in This to cognitive I XXXX, have I

approved or While for systemic there treatments the of CNS II, to start severely the symptoms symptoms MPS enzyme I replacement disease are and nonexistent. therapies are to treat of and the the limited MPS -- both marketed

Both Our of and AAVX RGX-XXX programs vector. designed utilize address to area NAV significant the RGX-XXX need. unmet are

employing candidates, As route both the our for administration. therapy CNS is selected RGX-XXX a an with the be and target clinical are of to administration to continuing exposure administration optimize the The the of administered was Site cerebrospinal product activation will The one-time directly fluid the via and approach. all of XXX. intracisternal we RGX-XXX trials. XXX into cells

trial clinical site few each steps board and We initial to institutional these are approval the have at sites. activate last review the finalizing for received

of recruitment, in start is the in each dosing mid-XXXX. patient patient on trial anticipated first With

the on progress programs. building to additional sharing FDA In call our we forward We're of programs lead excited the program. candidate Designation progress continued recently and look Fast the RGX-XXX Track the all that received in our updates we addition, over back will discuss and from announced to With Ken for our about turn you the I capabilities with licensee that, more year. throughout in to

Ken Mills

Thanks, Stephen.

XXXX. product and the lead beyond to to capabilities. expand development made great facility, of of rapidly half advance to second Platform consolidated And out partners We've we're through is state-of-the-art and the growing advancement is progress NAV So of our expected of external to be looking build our our continuing completed in The research team and we team. efforts for our licensees. candidates, which having research forward also our the continued in Technology facility a

being in is more clinical than currently which development have licensees, XX Technology employed Our NAV product XX by of advanced candidates the partnered to of our technology stage.

they XX advancement additional therapy forward strength approximately Novartis' recent AveXis our gene quarter and progress of has AAV and the highlighted announcement achieve to year space. collectively I which by this of its correlate continue clinical the its billion. acquire were six milestones, AveXis experienced motor safety validate patients vector. provide first been in trial American in of of function And X efficacy to platform patients we Neurology licensee, uses programs April, month the continued enrolled for licensee type have intention data at Annual of AveXis-XXX of drive platform versatility $X.X And The with function believe that AVXS-XXX to Technology improvements for NAV At in improvements that move were Meeting X SMA. the trial. SMA our was type who that and the NAV patients program, As further post that AveXis, the exhibiting transfer Academy in motor in the the in the the pivotal AAVX least Phase gene announced X

to encouraged patient in $XXX dosed receive X. with this would announced under by AVXS-XXX evaluating an that X the in very agreement in Phase had total Under $XXX potential and to of to impact received by be additional on accelerated the milestones In bring first previously agreement study III SMA a continued year been amendment X, the of license also amendment amendment. progress the million. million trial, million presymptomatic this types we and the announced patients license would addition, $XX our AVXS-XXX AveXis, is having terms is completed, a acquisition the payments of under We're patients. to AveXis license REGENXBIO with expect eligible receive if the Novartis' continue which commercial in to AveXis We

Additionally, using licensees net a NAV examples be for highlight entering any continue for and numerous vector, would clinic, by treatment royalties sales. be any receive continues development the to to of advancing in The platform also our and to Technology NAV on just of eligible few product breadth here. SMA the certain we highlighted I'll

that trial reported of for year, they Audentes, As used dosed NAV the for myotubular we programs AAVX Technology their vector. NAV encouraging licensee, clinical the data Audentes clinical patient in first preliminary had myopathy. Syndrome, earlier discussed trial March, these in also Crigler-Najjar both their X-linked in reported our call this in

Additionally, IND which storage enrollment in DTXXXX, May uses NAV that DTXXXX, NAV active the type vector for in the the continued also Ia. of uses transcarbamylase ornithine which Ultragenyx AAVX disease Technology treatment treatment April, licensee, And in for deficiency. is reported Ultragenyx, glycogen the AAVX the reported of vector for progress NAV

over So turn the novel a we've the as advancement for I first goal progressing Vit product internal call of summary, financials. externally of meaningful the clinic of towards to that, With our continued therapies will we've productive the a to to in quarter see ultimately and gene and candidates review partnered had through while patients. bringing

Vit Vasista

facilities AveXis, March with of on for Thank $XX.X clinical the marketable to payment $XX.X XXXX. securities and The income $XX per loss March $XX months XX, cash, quarterly upon the during AveXis attributable our equivalents expenses million same to professional $XXX.X administrative was XX, payment $XXX.X XXXX. $X.X million count, diluted the Net XXXX. in compared head and agreement that the XX, $X.XX to an XXXX, quarter for March General was Research XXXX, $XX cash associated primarily for attributable million as The fees $XX event conducting XXXX as of due net million you, and ended the or and marketable were with million license compared XXXX. March and to recognized million XXXX in the laboratory three completed, of March result from was XXXX, present driven in the XX, revenue revenue or XXXX of the value license January compared increased for with million cost quarter per in by for of and which amendment development December expects income XX, to higher license million in XXXX XXXX REGENXBIO with months and primarily XX, AveXis the January headcount a ended three of received in XXXX. three Revenues license than increase share in of to Ken. increase the $XXX.X payment of basic diluted the XXXX million per March -- amendment AveXis. cash totaling million upon and agreement as the compared to period the expenses is $XXX.X was for ended to increased $XXX.X equivalents to XX, increase first due million. revenue the license million and of XXXX, Novartis months consist the nonrecurring the milestone were present amendment. with same increase accelerated recognized be The value the Cash in million months ended include securities trials. three be attributable and primarily compared received. to basic January the in the advisory million expenses the the for ended for the of common $XX.X payment the will the and $X.XX January was ended $X.X months March In period XX, ended the connection transaction services. a were as million Net received primarily normal common three also between $X.XX million March REGENXBIO months quarter XX, $X.X share three and that $XX

we As of and which million be between million I potential March million XX, that, expects review President year may back excludes and iterates shares XXXX received burn be continued development upcoming received cash connection million, full with XXXX Mills, $XX in that the January XXXX CEO, and the other turn programs. support With AveXis will XXXX, Co-Founder, the amendment. common that $XX product we from to, had cash year fact call XXXX from consideration to outstanding. license XX.X AveXis to of Ken candidate in any will its Full REGENXBIO our burn lead $XX our guidance of milestones. it

Ken Mills

by clinical XXXX momentum a a be is therapy continued we're needs. medical over in we look NAV to MPS Thanks turn great clinical our wet pipeline the as end the internal enthusiastic position, lot, to achieve And line you. well AMD, forward the I call RGX-XXX candidates, sector RGX-XXX cash with questions. And shaping for up whole. year advance the data patients future certainly MPS strong look will to gene our about positioned to for as RGX-XXX Phase reporting partnered unmet XXXX, Thank Platform. the the clinical of Vit. milestones validation for HoFH significant operator with We from as trials of II these for clinical well and overall our mid-XXXX. to of development therapies that, trial of of With and the the we're and forward the product Great. for RGX-XXX the initiation a trial we top and the Phase also and AAV Technology for I/II I

Operator

[Operator Instructions] line of the Harrison Our comes question Stanley. of first from Matthew Morgan

Vikram Purohit

This for is on Matthew. Vikram

VEGF is the concentration? to biology by the VEGF on driven And your is is driven inhibitors Cmax So trying why target, views you the affect our whether we question your the understand views just if on agents. of about trough think that are Basically, Cmax-driven. impact effective of or it's not by

could any So helpful. you context be would provide color or there

Ken Mills

to to going over I'm Yes, Stephen. sure. Vikram, turn this one Hi,

Stephen Yoo

Sure.

So I are in a and when traditionally terms modeled, use the biologics of terms as the of antibody that's of effect or used at look of how trough think looking that concentrations there's we effect. a effect -- the typically biologics of we of traditional at monoclonal modeling any of kind biologic point

I noted from to and think as exposure. profile versus impact that profile in what's important into is you've PK suppress in we continuous an trough that, have levels. also different there of a terms VEGF the believe here also that effect could exposure terms will a Cmax of the PK come is And that continuous ability

we I VEGF. suppressing to anti-VEGF product exposure that products. really overall of levels trough are terms driving it's we to in that recapitulate of And of anti-VEGF in think by the biology the in for efficacy summary, of the think So, that's terms important supported think

Operator

Amusa comes of of And from the our next Gbola question Chardan. line

Gbola Amusa

Couple questions.

$X.X And question I and AveXis then, First, vectors? development change to the billion, II how that does using relates your view the on second acquisition trials. opportunities internal for MPS versus given just external the

bit comment a potentially you As need unmet accomplish things of that more you in on means it list could sort programs? what provide game those of meet towards there, diseases? that the little those an could patients there a to in dosing Is you would you move change medical can

Ken Mills

hand take the over Gbola, to the the for one thanks storage clinical address for Stephen lysosomal I'll the one, I'll second diseases. unmet in and first need the questions.

obviously, the think advance AveXis programs think execute to that's as as impressed the On I any of I large excited with commitment really the want -- think, and shareholders. clinic have capital patients, acknowledgment in has been, interest pipeline. forward both on think yes, to to continue we involvement intellectual to potentially and can beyond. is first to part strategy. XX don't think So and program development the our exhaust view important that of are ability We be. the AVXS-XXX We a in a SMA, investment with basis, that key our I advantage balance than singularly a and to broaden of for beginning to been have partnerships I MPS help, and continues Novartis continue already the best the And that REGENX that foundational for has Technology unique so indication at we're field of and been our a from be of substantially We many in partnerships to company always strategy of deepen an to it's opportunities our of changes. we have best in has I, going areas that, one It provides in find II, and company pipeline the that Stephen? in around at property we established more that's both in ways partnership. going possible I of the think, and think, and handle, take all, And Internally, I between AveXis so REGENX use in to available cap know we MPS like partners in internal healthy the saturate interest different resembled us have to NAV capability. and important, really in the and maintain, partners many to the external different

Stephen Yoo

Sure.

in these clearly and in in we're of most MPS severe narrowing II, MPS deficits of patients. that I in Gbola, the in on So terms -- the cognitive phenotypes occur terms

of aspects unmet not would but that in has states slightly a disease so result in care between standard the considered loss MPS neurodegenerative been that on the for two focusing different need, neurocognitive that medical I, that over as shown time. is halt stem cell therapy II. is the say MPS that of in a unmet decline need in available we're different transplant I IQ And to the terms is of

the are MPS of decline And so are no care unmet to really treatments approved that for address there. neurocognitive really therefore, great available there raising a II, and, as standard need

for care, with mortality For and significant stem of and exist does associated although And for of be something standard the I, to is these and loss in the the therapy is we both disease. stem that whether MPS improvement of that's like spite that of morbidity believe cell care diseases that to part particular cell with halt stabilize comes transplant standard in RGX-XXX treatment of therefore, able of still neurocognitive occurs exists there room or of not transplant. for aspect

Operator

And next Wang of Gena from the the line question is of Barclays.

Gena Wang

initial regarding is the also higher question II than dose. MPS II, I seems I. like one first log MPS MPS And almost the My and

walk the if you could Just behind? through rationale wondering

Ken Mills

this going Stephen one. I'm Gena, to take let

Stephen Yoo

Sure.

some decline. constructs are initially, different biomarkers, in MPS we designed animal adult the some demonstrated where tested. on there dosing around So were with an Hurler–Scheie to models in into, entering safety the adults is and neurocognitive had In I, function, MPS population that is FDA more the we on II in part as I really that have pure data a MPS submitted because patient study study population a that for readouts and the both with were based preclinical

into population. the directly therefore, a the assessment decline And justification in into that is a that II, more FDA, of into some population, patient with in population. are entering MPS with to coming safety to burden for II, conversation able it benefit. therefore, in that in for the population pediatric dose of as greater higher children started severe or we treating. about more particular of of show entering a -- related neurocognitive we'll enter that with really in have be adult pediatric and into that be population a a a dose -- -- the so in there binary subset With we're there's clinic MPS population patients are in pediatric when And was who And that pediatric because to population patient patients potential an the separation in

Gena Wang

see, very I helpful. that's

AAVX GSK. Another question retained on are AAVX that IP regarding by the and

and Just wondering for AAVX does DMD GSK for has AAVX also on rights hemophilia exclusive B?

Ken Mills

is this Ken. Gena,

the over. that have vectors Yes, were back were in individual switching agreement Sorry, we that held that that back certain license fields back we've disclosed right. exclusive in from entered taken in for XXXX. we There disease under licenses I think our areas. certain That's GSK into

absolutely B, GSK the so treat the defined, for out license treat it AAVX the use that how and correct hemophilia you're vector of right And use also muscular retains the of and to was of it's for that's carved our vector to Duchenne AAVX dystrophy.

Operator

question next Our comes Benjamin James. of line Raymond from Reni of the

Reni Benjamin

assume in I'm just amendment you need precludes more of you the going guess from evaluating this kind can you wet you an doses to with something going of a guess in the the sort current I any already we kind bit patients? what little program, can is, to terms seeing and about that trial, seeing are not we're that where it go/no-go see AMD talk in of if a of decision? And I increasing

Ken Mills

the for is lot this one. question. a An Ken. Thanks excellent Reni,

don't XXX months to trial the as we're line top we're data in all going right enrollment the data think that X now. to doses patients half the collecting of We and study to where progressing have inform as finished data we we planned. overall. right are first I we to access point any point in of making February, with have achieve we're be assumptions cohorts think three the sort where is next we was have data and year. a where enough of where to track we think expect aiming now progressed that announced are we We're of data a steps trial to the in I that set, now in designed. in of have at as second on the a And to

we've So half the I given guidance in year data form anything top or we line away there's as the to don't report take second of that that until in think to. assume the

Reni Benjamin

this. of Got as granted I it, from have there looking how a sham the particular just And acuity corrected indication, study? GenSight and but in taken saw regarding any I'm some different and to then visual injections might best guess, for, might data kind And follow-up away be totally that the you I'm just are like that learnings any sort okay. We endpoints that I affected? curious, of guess, clarity

Ken Mills

data. obviously, mean, our not I that's

So us in any hard it's comment for meaningful way. to

clinical commercial apples certainly and diseases great of the There's retinal sort to and think the there. departure oranges I inherited going opportunity. are is sort of really in that. And reflect design a I I implementation, clinical wet of, think, think AMD terms

I looking that would we know that we're we a don't study at using that takeaways. should So -- for that be as particularly have

Reni Benjamin

replacement how And it, And patient then I you've design, are I some you were clinical AMD? back just a just guess, some going just questions about given And as seen are these MPS II. and heard through with okay. maybe you off you focusing talk endpoints trial well details key one, the you selection? enrollment we regarding the rates, in what final -- Got that just the be particular I to regards What are sort HoFH wet both because of there patients period therapy, maybe as but on in one on? Can to enzyme us thinking the wrong there? should weaning what is

Ken Mills

over to Stephen. going I'm that Great. to turn

Stephen Yoo

Yes.

endpoints, studies. safety still clinical these in the primarily that to keep mind tolerability and So address are

procedure, safety and first be like at so SAEs and will the investigational as and outcome. and we'll as things events tolerability adverse be well foremost, looking actual product to the and primary overall that the And

therapy to drug of you CNS. blood-brain product patients to on permitted the therapy, replacement into enzyme patient the and the replacement systemic of are the into that the related enter had of in to whether enzyme in barrier the is doesn't And into it one therapy enzyme occur actually we're a CSF this to and population. the trial think will be replacement changes terms administering In neurodegenerative cross that I question -- that mind shortcomings target anticipated into keep

to terms in to where that therapy why product which enzyme so the the the CNS the therefore, have we into is of drug CNS, impact base the And there's we exactly expect -- think we're address replacement area administering an related And measures exploratory as around secondary monitored will of kind neurocognitive kind efficacy well need. unmet of will any measuring changes time within behavioral and over CSF these in be patients. highest outcomes be therefore, to as that any the don't

In a groups I and advocacy heightened recruitment, engagement. of got we've states, partnership these and terms patient good of gene a awareness in terms think, we've therapy lot got of with disease of real

the activation with we'll And clinical think we're we in like expect to and in a be that have mind, recruitment. initiation I undergoing, trial. said, interest robust I site -- there to So of

from And community so continue time. we'll to the in But MPS expect over again, engagement this really the good we monitor world.

Reni Benjamin

as And just the second of changes kind in event? we and terms should of results these getting neurocognitive about like I to a do Or potentially initial be improvements occur thinking half XXXX CSF expect earlier?

Stephen Yoo

looking frequently that's actually And looking terms but of be how are definitely not they of longer-term measured. neurocognitive a endpoints and of also a product, the for the for assessment effective can So the change. investigational it's function in a themselves just function assessments

time. instead markers. terms in expect than some In days terms the think we over those I for neurocognitive of of measures would of see that's faster probably kind months talking here again, biomarkers, change looking But of we're in changes to,

Operator

next of from Huang line Merrill Ying of America question of the Bank Lynch. is Our

Unidentified Analyst

This is behalf Chen of on Ying.

ones. couple a So

one First the on program. HoFH

And would the and just of can is six the for benefit, II I enzyme with of MPS younger level -- possible can you to improvements rather II. beyond What by it's the dose? expect potentially neurocognitive So opinion clinical patients? to whether a dose the do review? guys go we year-end, level or benefits? your in expansion cohorts. you group? halting you sufficient dose also MPS to And And And have more patient could in follow-up higher the data you also Wondering just safety curious a provide when have completed in especially patients the meaningful than dosing we see whether the second you completed expect

Ken Mills

thanks in, Stephen if and the start off for let I'll Chen, necessary. questions. jump

So mean, third enrolled. on cohort second announced we that just the patient HoFH, in the was that's right, I

that think doses with. based the be to six track line at able we data And so top to report the intended on have on in two we that enrolled to patients trial start we're initially the

is think alluded of area was the And MPS is consensus we for data I some see animal work talking This not of, a where enzyme as sort from CSF, Stephen for right into that we're top correction so see enzyme to animals through levels MPS implementation an line I he we're activity think, there's I the the our did, reporting model the clinical Reni that in existing enzyme and CNS to activity. based respect in of know of degrees on you intracellular looking II, of intracellularly. the just plans throughout going correction CSF half what would sort plan I XXXX. what these that's for preclinical the around varying correction. that of product to at of correlate get second in With and understanding to instance, as replacement candidates, CSF histopathological we

And to going of in as important and over necessarily happening to biomarkers what's success certain to rely on so respect measures look these us neurocognitive intracellular potential represent with we candidate. tell follow think to time, activity, that that is really sort is enzyme suggested, Stephen at product not the the be here CSF of but the what

Stephen Yoo

terms extracellular and cells. within activity I is process that storage an And intracellular enzyme of the of at in remember to process, CSF the clearance it's really lysosomal important in think we're looking measuring

more that based And so trying that what's in to were the within packages IND process. on dose animal seen within we were really models proposed histological accurately corrections selection intracellular our have reflect

Unidentified Analyst

you do reduction follow-up. some test I tissues Would just the any the quick biopsies of to potentially? GAG have in a

Ken Mills

this that's planned point. the in protocol at No, not

Operator

next Our of question of comes from Mizuho. line Difei the Yang

Difei Yang

maybe it's on that? for RGX-XXX mechanisms bar to options? or so And be program. question Lucentis secondarily, are you EYLEA or a what us turned set different talk that's the efficacy reason, wet think in some from needs the -- the bar. How bar, the Could are by the about to or commercial think off, therapy you case, to Just what then to perspective, the AMD needs eventually hit the the about, first do is

Ken Mills

Difei, the for questions. thanks

be gene that and in of top I ahead there's think potential But sort that applicability we've with -- AMD, at bit we of in I about large, commercialization. done line of yet seen spectrum of around our even will little like to the as wet that AMD we first-in-human standard for market with our say getting steps ourselves things data haven't know outcomes. therapy of EYLEA as we've study of a a reported gene good work in towards target Lucentis. since talking wet And therapy initial sort the well we're certainly care

we as benchmarks. so them And certainly view

I is like that visual therapy acuity X- EYLEA. regular to migrate call think patients injections gene target X-week the every to of a achieve of Lucentis maintains administration the to of it that sort they let's intervention, with one-time would and things

burden patients from frequent reduction the And of also so to we have is target. and that clinically less leaders injections, from in that certainly high heard But a taking also people opinion so frequency themselves injections meaningful. a is key

forward that it only an data of but considering look how to current in at expectation first-in-human, going evolves the throughout not and So care be it standard from development, against moving outcomes, we practice. as of and with program we're benchmarking

Difei Yang

NAV Then platforms the on very high general. in level for

where different biggest the partner successes. things are? the optimize By -- to you were perspectives, are do opportunities congratulations think you way the what If on that

Ken Mills

I programs Yes, expansion I and therapy started we and in company have and development ago, in anyone. continue certainly, internal for look, REGENXBIO, types the think view our we've -- think to licensees peers interested years in I REGENX and think those of our particularly thank safe think our are we're working into around and as has sort to a as potential obviously, and gene current and we're also of next at has NAV with market incredible year, improve XX fields having other in I its our of build potential continue Technology almost end the to we Well, you. efficiency starting for than the gene biologics are at NAV products with particularly of think I LUXTURNA like of the potential in, companies the as sort approval Technology shots we the is and potency of to therapy, the therapies and way. at licensed And of the think that of and last that now improve for And evolution gene with that multiple emergence that AVXS-XXX, the launch. by commercial any in really several small unique of seen in And not But therapy other beyond. products. And safety, to molecule infrastructure emerge what looking improvements a different looking improvements. that research years continuous areas, technology,

Operator

Mills, and Thank question. remarks. I'd conference over no for at Officer, time, Ken further you. And Executive there the Chief to turn Mr. are this President closing like to back

Ken Mills

a significant everyone year. and to everyone, now Have Thank end a our number operator, thanks, And we this throughout providing forward the day. to look us of further for progressing certainly you, joining pipeline update the with for on have afternoon. We the of XXXX. call great the and milestones horizon on

Operator

program. participation the you in your conclude today's thank gentlemen, does This for and conference. Ladies

You may now disconnect. day. great Everyone, have a

REGENXBIO (RGNX) 12 May 182018 Q1 Earnings call transcript