RGNX - Earnings call transcript Q2 2018

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Operator

Good day, ladies and gentlemen, and welcome to REGENXBIO’s Second Quarter 2018 Earnings Conference Call. At this time all participants are in a listen-only mode. Later we will conduct a question-and-answer session and instructions will be provided at that time. [Operator Instructions] I’d now like to turn the conference over to Patrick Christmas, Senior Vice President and General Counsel. Please go ahead.

Patrick Christmas

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

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

Ken Mills

us. good for and Thanks Patrick, joining you, Thank everyone. afternoon

and from a this update XXXX. on a progress interim of Our an trial for second trial. results results provide RGX-XXX agenda will presentation provide morning recap our the of recent today’s RGX-XXX Vit including for call, financial of conference the quarter released the clinical we’ll

we proprietary Technology aim upcoming potential therapy At anticipated the through our to REGENXBIO clear, curative review on and questions. improve to of milestones REGENXBIO NAV open our mission platform. the also call is lives for will gene We then based

over or virus AAV vectors NAV platform vectors rhXX. of AAVX, consists Our AVVX Technology including XXX and AAVX, adeno-associated AAV

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

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

addressed we have with retinal diseases. diseases We are selected strategically disease and because currently area there advancing been address to an pipeline believe metabolic each vectors of areas can diseases, they medical need. be many internal focus neuro-degenerative are and These uniquely within unmet focus our significant by

license we third single vectors Beyond our commitment companies vision selectively more to of about this and moment typically ours. want is our whose our internal indications components development to I mostly is for technology single take that us than platform; for work gene to to platform. emphasize therapy Technology NAV with programs, a party align

one patients. constant therapy the will change AAV evolved, is to that commitment grown company field in our gene and While never there of our has

to Our address the unmet improve gene therapy. through to ultimate needs and is being progress goal we’re made optimistic about lives medical

disease the be we could as our product patient advocates a of the We engaged each to understanding course guide patients, We of these those are serious believe of actively these therapy single-administration that gene to significantly partner ensure to alter compassionate with reliable an disease. transform lives insights of and patient of facing and the development candidates. treatments experience, strive

partners want to REGENXBIO and and acknowledgments. to and conduct chose make of attention support in to to network and clinical trials. to the excellent as review participate networks our possible. Today, acknowledge the trial trials people Additionally, employees the our the subjects contributions who before our we also their wish further of make I clinical all investigators their all work begin interim I’d of of a like for who the this of I of acknowledge by clinical some trial results, people

are grateful trust your We and for in engagement, us. commitment

this clinical expression. was encouraged the if AMD non-rare RGX-XXX potential at morning treatment positive for doses. as In treatment a NAV phase and interim patient So burden. all is dependent data significant we We the well with observed for very were tolerated a population trial, wet of one-time RGX-XXX protein therapy the indicate in gene X particularly increases by a dose this

administration need and for a also following with observed acuity. dependent dose sickness maintenance in injections RGX-XXX visual anti-VEGF rescue of We reductions retinal of

patients our have higher to a trial dose as phase it subject our quickly turn the now for clinical X trial week. Steven I already at novel cohort possible. applying X product we this REGENXBIO expanded and forward program a to a to been expand for first the bring in the include dosed over for updates We candidates. are looks which this to as to development trial phase lead for presentation current learning therapy of RGX-XXX will has interim and this trial to from results what the

Stephen Yoo

of begin wet I a this with released on we with the the morning. Ken. Thanks like our would RGX-XXX AMD, program data to today for focus treatment

vector Japan the vision NAV that therapy gene the struggle million reduced wet and patients patients for affected. of vector than therapeutic burden X utilizes AAVX reminder, encodes to patients a inhibition lack something be fab VEGF anti-VEGF in new The anti-VEGF therapeutic comply ultimately lead Europe the Many Continuous the demonstrated injection. affect neutralize has produce the to antibody designed animal administration. of levels a after families vision to requires by to is made will NAV is has anti-VEGF which a usually continually product candidate prevent high the AMD fashion AMD. selected this a therapeutic recommended for therapy single protein. pre-clinical which it to be a to to therapies The to Continual in a cells can cells retinal VEGF and transaction RGX-XXX and for a retinal because of affecting ability loss. US, continual protein As of more and includes treatment regimen designed been with loss. current expressed is and of quick therapeutic is activity compliance [Astra] affective models AVVX in activity disease wet multiple continuous anti-VEGF and

phase subjects wet frequent have a who with to entry. previous included XX XX in cohorts, The the in therapy. the On date average next least in observed open-label, slide, to anti-VEGF trial. the study wet RGX-XXX we United multiple current at of the the We a have dose multi-center, States. is high escalation injections for of treated see injections demonstrated adults had majority has This four months. are study in clinical subjects X the with AMD AMD regular previously treated of with prior eight years on and enrolled need study many over of therapy Most anti-VEGF subjects previous a depiction

responsiveness cohort followed X XX^XX X. genome cohort To-date and for months for X X, for six X evaluated x copies X XX subject’s therapy after nine administration of anti-VEGF a to to the have x The months of was and x anti-VEGF was an doses measurement subjects months eye. through for confirmed average of designed XX^X, of RGX-XXX, XX^XX response study injection. per three addition, In an by OCT cohort

would highlight evaluated primary RGX-XXX is tolerability months study of delivery. administered Turning the to single dose after and slide the at to safety X, objective I of the like six sub-retinal a to by

clinical signals to also being the evaluated endpoints such in imaging and examine meaningful and assess Secondary SD-OCT. outcome BCVA potential including ocular are measures, as

discussing routine the the additional for in three discretion slide loss in cohorts reactions, the We need a have no XX Most and events given related is or drug to events safety beyond to has been inflammation who’ve were well reported have From a released XX vitrectomy. XXXX. today as Five therapy for expected related I’ll study. or start been enrolled reported the no date. by with the or on adverse trial at anti-VEGF there today to from treated of total be could inflammation of persistent July adverse the first addition immune SAEs that subjects observed drug AMD previously tolerated here total what In ocular data which been wet The new fluid RGF-XXX covers date. vision. XX, investigators data. post-surgical in mild

in encouraged to acuity by the first in human now would RGX-XXX RGX-XXX safety retinal SD-OCT X and parameters efficacy injections, We BCVA. in expression, as around anti-VEGF the initial are profile by several announced of some supplemental measures by of signals thickness outcome visual clinical this to the We measured in including trial. measured attention study objectives as clinical protein today date. I assess phase your positive of to for secondary turn study to the like

expression maintain to the slides. injections, observe in cohorts. levels, and protein dependent data dose improved across this all through maintenance to pleased dose will are walk visions on We dose reduction following thickness of I dependent retinal anti-VEGF

protein XX. of begin taken a been shown ELISA RGX-XXX. by detected eye the for aqueous protein levels approximately weeks date; levels Let’s from slide to at levels summary RGX-XXX treated with the on all by front administration of here have measured cohort in the were after subjects four of samples

dependent of dose encouraged across protein all We’re cohorts. this and of expression dose evidence

the of observe injections required XX, months at regarding cohorts. We of the RGX-XXX present anti-VEGF injections and administration, to six slide across decreased of number has all rescue data Moving after we number administration the post RGX-XXX. dose

received was we number first the of months understand to X.X history. in comparing by RGX-XXX injections. X their required anti-VEGF no by months prior enrollment to the subject to study In administration average post-XXX in cohort recent in the to X.X X, Since received required X number see six anti-VEGF anti-VEGF the frequency in study slide, was each for average six subject’s following of compared following X a injections, enrolling average received subjects reduced can X comparing number cohort this In in each at by average their cohort in mean did X.X injection had of during of injections six We of injections when anti-VEGF control the injections cohorts. cohort anti-VEGF a post-dosing administration these injections the of phase has prior preceding the human the of You with this study. the changed injections at XX% administration. to arm, number open-label of the trial here how most this injection injections three and wanted each the history the required six an post-dosing received subject RGF-XXX to months prior at months of number

cohort administration of the like since anti-VEGF subjects XX% mention Additionally, injection of X I’d RGX-XXX. treated to have in that free of been

the improved baseline injections their few to vision observe anti-VEGF anti-VEGF first X slide therapy We their gain in baseline goal six we another gain On third X has first in we expect subjects to the months subjects cohort typically measured naïve X with On cohort wet receiving is average. are the also and by treatment. Subjects values subjects, CRT data demonstrated experienced in enough therapy, In retinal anti-VEGF next previous CRT The The to large slide, subjects been can X many regular studies anti-VEGF present are encourage BCVA at look a vision produce their show therapy retinal CRT indicate ETDRS observed be changes six. years retinal XX, of SD-OCT. of who’ve to months in The phase maintain study. to a would thickness in anti-VEGF cohorts. thickness reduction response anti-VEGF in with dosing in acuity that while from mean thickness that response maintenance high X from in in we therapy to central few in injections. cohort entered are of mean the in greatest of as while fewer injections. receiving AMD with this data CRT the of of the to these from of therapy for on Maintenance cohort three greatest we maintain who Similar central anti-VEGF X. study in letters, maintenance month our a subjects X, the protein reducing months. need central studies in to Heidelberg all visual phase in the experience of naïve wet-AMD to cohort subjects number of able CRT

months. previous or with cohort number X small, maintained the note Furthermore, six subjects subjects at Although therapy is subjects BCVA treated improvement further in is have at months it vision gained of in six that post have cohort in dosing. that X encouraging experienced all we

bars we’ve with illustrates in is SD-OCT slide, values the CRT OCT. Y axis; in cohort the next by graphed orange axis. maintenance to left the in the in are vision values overlay on the illustrate and trends measured overtime values on X graphed for vision and On in in blue the with all Y the This Heidelberg cohorts the three of X of right The cohort the represented vision improvement X. and with vision maintenance as OCT

We’re cohort in vision of X of with injections CRT improvement XX% with through specially anti-VEGF cohort to XX, I’d encouraged reduction that there of X. maintenance On anti-VEGF is highlight of six slide subjects free injections were in a months. in like that

reduction had meaningful XX improvement in six levels, of dependent administration cohorts. a protein no BCVA XX in baseline. protein significant injections measured reduction study received post summary, mean it subjects had XXX administration a injections and at expression a six These following also shows [data] X generally anti-VEGF required reminder, anti-VEGF by CRT letters So is following of is subjects months, expression of maintenance And There across maintenance clinically previously the month the EDTRS higher levels there dependent a slide SD-OCT in subjects of administration. weeks as aqueous all CRT and therapy. mean for of the have at As anti-VEGF dose versus of with next RGX-XXX RGX-XXX dose received micros these on evidence in

a changes combination to month in months. In six in subjects no a and have maintaining Additionally central in cohort of improvement injections shows anti-VEGF anti-VEGF six, of improved assessments XX% while cohort particular, reduction through X shown and retinal BCVA thickness acuity. with injections visual BCVA mean through maintained receiving the

are The subjects biologic results the the the by study along best levels of over based observation to the at of expected today encouraged presented activity the Illinois. showing in of we Conference additional clinical who are months subjects released on be in meeting also with the annual at next Association expression XXXX highest are are results Ophthalmology the couple study of protein response. We American measure with that evidence the Chicago,

turn I steps Ken RGX-XXX. to to next for Now will discuss

Ken Mills

XXXX this measures the we in next Steven. Thanks meaningful signals support clinical six at in for possible. to dosage to before indicate We with plan dose as we the to novel lot phase use design bring that trial to engage a X and months AMD. authorities wet positive to And described a X X maybe regulatory a patients clinical clinically of slide, hope this file end phase as cohort therapy and from as expect quickly

X.X phase Now based expression, effects protein in eye acuity, we the fourth genome potential visual dose the existing of observed anti-VEGF tolerated additional copies on the on RGX-XXX improvements being evaluate injections new benefits to in dependent a an and XX^XX well X and dose x trial. reductions in of cohort plan per

I’m XX the the fourth here site an several has approved been as it cohort slide happy this on by I’ll already terms in our was FDA to the to subject in the an some now institutions and to showing provide Steven week. dose cohort new back or amendment immediate RGX-XXX share on to Now boards. steps, update next first of review turn program. of that we’re this add

Stephen Yoo

disorder deficiency vector. program an RGX-XXX LDL potential genetic HoFH for address from HoFH RGX-XXX fund treatment discuss HoFH, by the to administration. the Pennsylvania therapeutic University from Thanks results insufficient the therapies by Current to normal to care gene Ken. or phase levels. at caused responsible REGENXBIO. the today defect of RGX-XXX are receptor. standard the conducted NAV a rare the slide one-time metabolic trial lipoprotein announced receptor being LDL the the of franchise I/II a of our correcting uses We often XX RGX-XXX HoFH in for the to AAVX by with our and now and shared underlying has for of the update treatment to of LDL including today cholesterol turn for low-density I’ll mutations lower the genetic

announced single weeks XX, dose two. efficacy As in bodyweight for July safety update include respectively. received X.X you of X.X genome two for doses at an of two interim a in of for cohort Three at on have can copies per kilogram and dose cohorts. and subjects and RGX-XXX xXX^XX each XX, The HoFH and a for today here see six from x the slide of followed XX one cohorts total administration XX of participants the trial cohort each of average XX^XX assessment XXXX subject cohort the cohort weeks been across

reported study have Moving were during there to active the of been SAEs drug-related. XX; XXXX, reported which July of four as XX, slide Two as period. XX-week

As X and experienced elevated a within transaminases. XXXX, activation one in system the cohort day. was in additional hypotension approximately hospitalized mild dosing reported peak subject the This liter. XXXX $XX perform patients resolved manage of January in previously immune by experienced accompanied transaminases international briefly post subject transitory per in assessments. of cohort ALT a and and to This X with was asymptomatic a A second units elevation transaminases to

all X were in The Additionally, the subjects a elevation three to of and transaminases. responded were three with and asymptomatic rapidly experienced in slow weeks taper, followed by to in post-dosing. X subjects. ALTs Cohort X of normalization XXXX subjects peak the three All initiation the transaminases XXX,XXX have an prednisone

the data therapy. changes corticosteroid and a After protocol Cohort time the to an such planned with assess Based expected that is transaminases the in cohort the completed. both three of reflected dose weeks and a steroid at steroid on review in by of three change review the is additional prophylaxis protocol liver dose as Penn initiated prophylaxis. of LDL-C level in on the include effects XX amendment XX confounded the the with in is DSMB, therapy is by third interim to including A in steroid been X the has at cohorts the the introduction point primary to to levels. and to evaluation expand weeks in include independent prophylaxis ability For DSMB and discontinued. from approximately potentially cohort will also independent later in meaningful expects subjects subjects the of steroid elevation cohort LDL-C reach show that ensure We clinically not believe did weeks, REGENXBIO LDL-C subjects X subjects XX cohort a amendment at to after be additional second LDL-C

to plans these update expect We XXXX. provide on an of the in quarter fourth

also that Institute a quarter further XXXX of investigator announced Medicine Therapeutics for for scientific meeting Pennsylvania We be Medicine in results expect Professor by primary Marina Cuchel, University cohorts the presented fourth and the Medicine in we School trial. Dr. both at the Perelman of of Associate the Translational Research for and will at of

now significant and would wide have products of I down genes and a I deficiencies of accumulation XX, RGX-XXX IDS we disease by like slide for treatment enzymes. range and of listing in in the II MPS-II. to normally the cell disorders causing MPS defective of accumulation damage, and This defects towards in patient’s MPS on IDUA of for an physical central deficits. two cognitive RGX-XXX which product would tissue be treatment both that share the lead respectively. symptoms severe genes our system. by broken and nervous to result Moving intra-cellular in to cell neurodegenerative I damage, the where leads to diseases these waste In enzyme candidates franchise, licensed for almost update development caused are

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

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

We Dosing fourth administration clinical both RGX-XXX into clinical expected and CNS quarter In has optimize are was target a started. first of directly sites fluid an phase XXX. will the now of one-time approach. of to patient I/II trials the to the cerebrospinal programs route therapy The RGX-XXX RGX-XXX the is in cells of phase via selected administration. administered employing and The be the exposure intracisternal active and recruitment patient XXXX. the in are X administration

We ended Ken capabilities progression program sharing building look discuss that, to With programs. over the excited forward throughout candidate turn and continuous our updates year. our week I about additional more product our you on in the and the will progress call with to licensee are to back

Ken Mills

Thanks Steven.

a number we Head team continued the in Operations. leadership for commercial President So a nearing expected Palanki, commercial have year. and we he strengthen of roles Sales certainly of to completion this Genentech development Global in Americas Prior announce joining with in ways. facility Novartis. a very encouraged Ram our The research at progress strengthened these REGENXBIO, our pleased including as parallel buildout we’re Inc. product and be going lot of lead Ram Commercial new with and of and that Commercial to roles. Senior fully and he than Maryland. began commercialization we to continue Senior XX experience state-of-the-art of we career We’ve was Rockville, capabilities and Strategy He by advancements, Santen Vice important also management progress is develop product Marketing with team REGENXBIO, is in site and in But updates candidates. and all Vice our make Thrombogenics. made we’ve years his of our marketing variety appointment later on in our at ophthalmology was to more to Previously of new operational great I’m morning companies of and in the and of brings this as these President

with together We’re manufacturing looking process a forward having consolidated teams. early research for development to and a and our development growing steadily group facility

our the our efforts slide through licensed programs to technology the Moving beyond to NAV continues partners. XX, external of platform internal on advance

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

recent additional was intention further its and type and the validate SMA that by efficacy of technology of versatility the of collectively the they the highlighted therapy Spinal platform NAV X. that As for AAV safety data the XXXX we provide Novartis’s third advancement Muscular of treatment announcement end a milestones, or of quarter the our believe The the over BLA programs for of gene strength platform space. progress drive licensee AVX-XXX Atrophy by to file achieving

an know, AVX-XXX from NAV we in uses BLA first the As under that Novartis Novartis This the from our using submission as under REGENXBIO announced NAV the X stated received Novartis $XXX a data vector study to phase AAVX the basis the licensed previously REGENXBIO. filing with technology. accelerated our will BLA Under the AveXis, the million terms data well. when represent be the license by phase to ongoing AveXis, million. of total would bringing for has in with the X the product we by amendment of acquisition license milestone included completed $XXX SMA receipt type amendment payments X for agreement

vector and progressing wish was broad and look net type the DTX-XXX approach the entering platform numerous on The I/II is and first advancing for active. additional for a also Ultragenyx, This used uses the the The of to that for and product $XX profoundly IND product forward XA. to announced advancement collaboration vector. We of with in XA the treatment trial, David summary, which Ultragenyx of Disease using Glycogen this in and of seeing additional being clinical eligible treatment the sales. receive quarter dosed DTX-XXX meaningful This AAV is for AAVhuXX improve program GST are Weinstein clinic we and internal vector of month, we a an DTX-XXX University track the candidates any partnered Phase was In we our the And another busy and underscored NAV Hemophilia for in novel that our an licensees in trial call announced is eligible A of platform SMA additionally, are clinic. receive accomplishment. clinic to the potential program for certain NAV being is the Recently, we NAV still over Bayer review developed gene million and milestones. Vasista of clinical through productive the patients. the for from instance milestones announced that a Dr. fast our and to of by a congratulate AAVhuXX who of with this to technology the the royalties Ultragenyx bringing at will towards of with patients to AAVX progressing great commercial lives to to teams therapies well. Last the vector, are second developed externally treatment Storage our NAV designation could as the clinic. patient DTX-XXX first had in With goal effort Connecticut Mr. uses turn the I financials. treatment the ultimately the for for and reach

Vit Vasista

jefe. el Gracias

expenses XXXX, $X.XX expenses XX, the increased laboratory months XXXX. services. were and externally primarily related on the recognized $XX.X compared we of same December the associated be of remainder period increase the Moving common facilities a primarily compared June XXXX. and was The and million with totaling XXXX, ended $XXX.X XXXX attributable $XXX for million. Research costs or basic during XX, XX, Cash, and of the the were expect development share increased in Net XXXX. amendment three an equivalent million the June for revenue by and advisory as $X.X with received with in XX, to million AveXis. condition and connection licensed milestone XXXX the million per the of of three license was a attributable to recognized conducting based XXXX $X.XX three which months ended cash sourced to securities license $XX.X cash accelerated includes was from agreement clinical trials non-recurring same increase million license Revenues period during lower attributable for XXXX, professional quarter XXXX. ended than diluted net in of the XX, ended million shares our the of primarily and three driven an to months to June of ended was for June generally development equivalents the $X.X an Due loss same period $X.XX to and June General amended XXXX, with to compared slide ended $XXX.X three was months same for June half the revenue the commercialization first revenue to million services. The $XX.X in diluted common and XX, million revenue months million million to in licensed XX, and in XX, treatments manufacturing REGENXBIO for sales million million was in cash, XXXX, for second June headcount, quarter Net agreement ended period primarily the or $XX.X SMA. XXXX six in during per income marketable basic non-recurring securities increase XXXX. and increase The as per its AveXis $XX headcount compared for XXXX. XX, as the fees for administrative were with as XX, the compared XXXX, $X.X income for for expenses to of $XXX.X marketable to June $XX months three substantially to

plan, that $XXX $XX million and securities cash we year, its $XX.X will million which product $XXX $XX to between outstanding. be As million will be to full cash common of balance XX, support its and that now we XXXX expected development of shares of current between expects and its burn December XXXX, the XX, on XXXX, candidate year Earlier Based lead million. million marketable equivalents June REGENXBIO we had programs. this continued be and operating as announced cash,

final increase licensors our related We balance, confusion the any that to more million. on in call of under and our El to remove With our amended and transactions. sub-licensed by have of milestones. effort received cash to to those upcoming I’ll be in potential securities burn million guidance XXX incurred reflects revised fees we changed end license year XXXX created community transparency investor to Net cash between President guidance excess provide the and The to marketable AveXis proceeds. payments expected those cash back XX thoughts with review an any turn the our equivalents in to and XXXX an provide

Ken Mills

Thanks up validation year product Vit. a our XXXX about for platform. for momentous candidates, NAV enthusiastic the advancement as to further whole, that AAV shaping technology we’re internal pipeline of and therapy the be over gene a update sector is continued clinical wonderful partnered the and

observed additional in in will updates as be expected to elevation sharing X, experience using dose and and steroids our increases what steroid RGX-XXX cohort at in in trial summarize We’ve not protein RGX-XXX we was mitigate today, visual a retinal which phenomenon for wet dependent you the in administration at on X we’ve data current the HoFH prophylaxis. X effects. dose X. I/II doses. to following next related this subject protocol phase RGX-XXX, cohort phase acuity. been cohort October, expanded from phase for additional phase dose The higher to I/II I/II of trial a the X rescue phase phase include X as steps amend the forward with the RGX-XXX the first thickness subjects of to already maintenance clinical appears trial elevations dosed and the these of need trial look for the clinical well for to reported clinical subjects dependent a quarter We fourth trial So presented Administration reduction trial AMD week. XXXX. enroll observed to transcarbamylase has updating anti-VEGF RGX-XXX In also expression; of well tolerated all We on in for injections

medical potential like and forward much we for feel and positioned to we innovative to of needs. we covered we a questions. call science in potential position, our through therapy achieve lives and well therapies for updates technology We’re future and for trials platform. clinical of dosing With subjects looking RGX-XXX quarter NAV improve our employees unmet very in important is that are RGX-XXX to this strong MPS-X this advance the Operator? the to significant curative as milestones on open and and based our REGENXBIO, morning our I’d fourth year. the MPS-II At patients with cash gene also the inspire lot positive mission of proprietary partners With

Operator

Gbola Amusa with Chardan. our first Instructions] comes question [Operator from

now is line open. Your

Gbola Amusa

X protein or the to once the know as expression all trial then expected the questions XX some cohort well be I other to go X. market secondly, as the does the is might about inform [interval] have there you cohort time in have surgical your cohort six shown change the in with how of market that that phase results imply a few that difference? such the from out forward about times you’ll the was and that higher. of plan when as as in our your of X the Is the given there, that months first confidence achieved approach there have or when times it noise with to within X will And report XX dosing phenomena all patients of I phase was driven X evolution data

Ken Mills

else the cohort provide phase study feel data for updates. will of and next terms we’ll forward planned. the to an observed the in with think support are that our AAO going including the plans Stephen for planning of think of we concomitant in the have we cohort our how X XXXX to on anything the clearly phase that corporate RGX-XXX the the update for we to of accrue X to to I is then plan everyone, update engagement is XXXX. between as Other at begin the of to that of the results what right of is planning XXXX, respect don’t and In and now have plans regulators end we the design to have clinical for I intention begin forward from continue updating we plans mentioned, be program that X, and to reporting now, at forward. our and development we in data than our opportunity with process addition With the we’ve and begin those immediately be But with on throughout moving have X, with entirety going as that’s trial October, respect that going

cohorts. about great that think question saw protein we what dose expression, obviously demonstrated is dependency a we that’s I across the today

feel can Stephen’s the and developing view, and of you we this to and last X, seeing still within to the expression and we data that. of we’re But from XX over protein what As there the a is certainly seeing. we’re days press from see continue in just release positive we’re locked and that evaluate cohort we’re evaluating going understanding lot takeaways so that

Gbola Amusa

I just want ask to more on and press just question my one luck manufacturing.

other magnitude has is per For orders of vector overall less than trials that for in wet patient an this trial in more gene dose and indications, AMD patients to but magnitude orders you’re market. therapy requiring of and market

those going much So how what’s the factors balance need assessing two you in manufacturing and of forward? capacity

Ken Mills

that We RGX-XXX. have and is expect in will think We of potential capable process of development. clinical our developed around the have produced internally addition, successfully the commercial demand. we purposes And meet intents continuing anticipated the manufacturing of for a we future needs both and the development that process already and meeting clinical current drug

RGX-XXX has feel right, to amounts and in liver-directed But diverse we certainly have some particularly you’re is of be to we in understanding a of the individual think and thinking portfolio we the virus than what think the we’re that lot I CMC we applying need. smaller that patients programs. planning everyone’s may because programs that and we broad we pipeline like with company So, put a

Operator

Our with next Gena Wang from Barclays. comes question

line Your open. now is

Gena Wang

My level have month first one months? has the wondering maintained the checked throughout and just protein protein protein level, level one the beyond is six regarding you

Ken Mills

Gena I’ll there. this start is Ken.

the some another is some those Gbola, So it be samples XX data evaluate patients reporting that time July recently given would be would tell cohort in we’re made lock to that what protein you date? from typically six So of can here drawing only instance have new, as data to where months be that we very in visit their is and the in said levels. of X, point via for able protocol, to

require the think we In so for all our the for cohort cleanest more and anti-VEGF is patients cohorts. time which earlier interpret later those is instance levels expression X And interference cohorts, as in in of are the we patients have the ability a why we’re to we administration have today. seeing three of in today agents that comparative an points, absolute to RGX-XXX protein propensity higher the trial, currently of don’t that of is selling that’s X cohort across everyone what the patients with protein does that the measure patients at expression what

the this can the and been that administration of at is the doing time of So any anti-VEGF protein basis what establishing that year. understanding RGX-XXX. our to a points levels receiving Gbola we on points, And to go-forward more I we’re of injections information think beginning who at particularly looking I next later like forward of provide alluded in at year later some patients with since expect future update haven’t

Gena Wang

And then my follow-up question is regarding cohort X.

us were Your of they patients, received? share patients many Can reduction with total of three reduction other free injection. XX% the any three and is injection you how

Ken Mills

sharing the are So the that or we’re that’s of expect go be seeing quality AAO Gena, allowed going showing to opportunity individual that through to level The data. week the non-information we of provide about able we’re of in patient at investigators sort so averages today what us the exercise that we more but on thinking to the a last cohorts. think based half across the and data were a high we conference of

Gena Wang

regarding last the X. And is question cohort my

about asked little the question I bit level. the also a protein was think previous

you anti-VEGF also it And based think for X.X simply micro to cohorts first things [XXX] the linear, of three in per of wondering not if the will that additional issue could you Now data, completely EXX think level what dose dose should goal if you enough gram just are be explore and I an your do waiting the be or level? (inaudible) time fall since that it’s on lot for a will is save the optimal? won’t and and enrollment

Ken Mills

very X very again think dose we is and great important question. encouraging. what are seeing And with a that’s the I So

in visual to the to as correlate meaningful in well that in even measures thickness levels clinically measures with protein acuity of seeing are many injections anti-VEGF of We patients. retinal and respect maintenance as reduction improvement

in our X So expand it a our population. to that dose we a study of phase that wanted larger take to we feel Gena have strongly to we understanding in

is of with we In dose. RGX-XXX that on have at value we believe opportunity X, what an expand an looking additional by cohort the to addition program

RGX-XXX We’re I see a specific our to and level. is protein the think beginning expression obviously of target the pharmacology of here picture in

to provide a continue think dose to could is safe Our a and meaningful outcomes broader and understanding about a And wet-AMD think us how an VEGF and could of inhibition. that clinically that that really with to cohort population number for anti-VEGF X also for in a populations to target start allow is in allow a profile we respect of good general continues to show well to RGX-XXX patients. agent, tolerated apply to

Operator

Harrison next Morgan Matthew from Our with comes question Stanley.

Your now line open. is

Matthew Harrison

guess I related about I question. just two PK wanted to ask

for you’re dose you you and what that how VEGF So the one, PK believe is the from that levels what appropriate just get can about think VEGF and median PK you you then talk of are that sort follow-up. And expect where whatever X cohort the terms in that will seeing X. given dose with you you have cohort compare? of I compares with to And think the protein expression a

Ken Mills

Ken. is this Matthew,

been well presented about existing that PK profile is been treatments for as anti-VEGF novel have what RGX-XXX really compared the a what’s interesting that physicians I development as exploring to we’ve the think of patients.

of midpoints So suppression easy sustained establish how of about profiles And treatments to is will sustained things anti-VEGF think we’re to the VEGF. of suppression to of been understand an that and something and thing that PK think to apples-to-apples of I and existing compare do troughs peaks to sort moving basis on on the don’t focused VEGF. like it’s

about maintenance of and sustains for we us suppression encouraged robust an clinical and injections, again thickness we treatment what about picture is reduction VEGF of retinal presenting I we’re but what visual the telling very So about additional are including anti-VEGF think a and and acuity. what need also encouraging think with measures is today

all we’re exploring here the pharmacology Gina, we that doses, expect at this said that And encouraged to focused and we patients. think to number expression so dose on the I now application Number as of about and point clinical we treatment I is the importantly suppression clinically seeing dependency that. in one; think of and RGX-XXX, we’re the a to define seeing utility and meaningful to what improvement are VEGF. like of We going is we is target two this protein across are again, in we’re have hope is, But measures continuous seeing patients. the continue sort see where paradigm

Matthew Harrison

on and the walk surrounding you SAEs, could the walk And from through died I cases? the with safety three believe. Can patients the other I you one through you then just follow-up, a think for those just us said flip-note in specifics had retinal pre-existent conditions a a detachment

Ken Mills

that hand to going to Stephen. I’m Yes, over

Stephen Yoo

Sure.

were COPD. unrelated the SAEs, pulmonary subject SAEs of the related death those disease the was obstructive an the chronic of had a procedure. pre-existing subject that had RGX-XXX condition three all to or had And to to who exacerbation and and For pre-existing the basically

that surgical the procedure from retinal we of retinal – the well doing vitrectomy is detachment. of which and repair commonly immediately a In practice, was after [secuela]. clinical without is in detachment, for terms detachment RGF-XXX, case done particular retinal is vision a this complication their any that use repaired known vitrectomy is in the detachments recovered and significant patient’s particular part of That repair and detachment of also of

Operator

comes Ying Bank Huang of Our next question America. from with

Your line is now open.

Unidentified Analyst

on behalf Ying. of [Chen] is This

I So questions may. I have if of couple a

you if all, obviously diseases tryout? other wet the see is injection asked vector? had kind you you it the X.X AMD look just reason that particularly wanted on trial about you at just guess comparing frequency the these put inject of lower before cohort other to were OCT first to of contact terms is anti-VEGF as a because wanted to cohort. X, period also when of used And the I BCVA. the parameters and And medium see the the wanted the baseline of the severity know than the So in you to of why you screening where anti-VEGF, whether I I right

previously rescue you question measurement. that I the month of injection with just you related the the and the just may injection And is release. expression cohort I’m the press immunogenicity. third one you the the the on that time mentioned in saw mentioned trial, question some you on in your the relating HoFH of Second impact [rescue] also comment whether can is VEGF wondering know

receptor on related And to immunogenicity whether that was that you whether vector. related could be wondering surface, higher expressed whether it’s seeing? the reason LDR is can I’m or LDL given comment that to wondering receptor Just on a that you’re with contributing the the

Ken Mills

the that take to reverse we’re am HoFH elevation. this I it order I this is the a in going with to that little an In off that bit more, will to cohort discuss the observations of still in some investigator. hand sharing what trial, seeing expect part opportunity in respect individual think Marina new have patience we’re with and I X to it Stephen. Transaminase

an protein around leave we that to With additional data of under in in particularly they on her. injection the to did anti-VEGF that month protein question all absolutely one month protocol. measures have to RGX-XXX so showed respect of going injection I’m received we before So your the the case timing of the the injection, patients level, would an one about

the the were expression I’ll is the let cleanest first, address the like have this in bit So RGX-XXX RGX-XXX patients a Stephen that’s And trial included levels. that comparative we absolute across again why cohorts in now we the about how data little right for feel baseline. protein on

Stephen Yoo

great and the to patient point population, Gina, trial. question think on I Ken. understand about Thanks important the that’s an clinical

trial at a basically injections we four shared which of subject. for when in And the previous who the this at injections over prior months as received entering least had on mean have injections we injections before criteria need had looked sought was number of the met. it all study, subjects the XX earlier, for I to entry to patients requiring and in so frequent anti-VEGF particular enroll and regular So eight high subjects average the

away because so you outcome treated injections for measures were the we long that’s to and think at is anti-VEGF as that long subjects lot phase of And with in interpret population. wet AMD periods coming naïve and standing of when how the months patient injections patients a do within long time with of that who retinal I And the AMD important had AMD benefit these the of long acuity these X thickness the therapy. take that for And history we earlier of from the look really visual studies had of And wet in shared alluded disease. been first standing we a gain I and think subjects we a reduction some with occurred what have we come in. in wet received initiating in few of know

maintenance sickness good who think we about therapy, subjects outcomes are vision been of gene to So on them when and these converting have treatment subjects. regular maintenance retinal and particular for of

standing an again additional gain retinal but a potentially subjects is in had can vision are really of here get different the profiles of who PK in disease. these history can history that a we anything vision be for outcomes injections, long think bonus or maintenance and terms good have either by of I sickness impacted

think patients that’s visual about for acuity. are high to results So come anti-VEGF They’ve to of from look again, think just summarize and these and the long injections. in with you important for thickness at years demonstrated when who’ve I a therapy retinal standing need

Operator

comes Raymond question with Benjamin from next Our James. Reni

open. Your is line now

Reni Benjamin

more the of they to they period just closer started anything as patients, about trial in guess could injections therapies. patient diagnosed Can here. and and X to regarding bit we a anti-VEGF that, goes, maybe talk design population, this related were they’ve injection that’s there just noted cohort a else again there little that out far this administration wash relatively is are younger or I to prior anti-VEGF newly administration? relatively like Kind difference gotten the an as RGX-XXX Is the

Ken Mills

all the same really cohorts, prior of therapy coming subject of on data point to with the years in. the characteristics average they came and in terms three of the In

So in injections a anti-VEGF X cohort did need also these standing long for with demonstrate subjects history. really high

and the the be in a of the in types across terms per so study I to we through all three think when and look looked are comparison, different all with think of of with cohorts, at is had similar results lens these small this arm. I subjects demographics terms subjects. So a it the They needs similar ages six

therapy you So to of again trial. demographics, terms to coming consider of the in years need similar similar age, in but the that at looking in

entrance there trial to in clinical did In anti-VEGF to terms that was washout, part washout to no require or the an therapy as trial. the We the was before responsiveness injection of upon required entering to with of confirm anti-VEGF therapy. entering

And study later response OCT injection for number on provided anti-VEGF day look to were on they so week imaging. a back and one, brought a

included we’ve continue of they characterized therapy. particular terms injections. is number these be received So how or not the part we injection that historical subjects did anti-VEGF medical to a history of of to that confirm study in responsive the That their

Reni Benjamin

that see still understand guys you bit saying us CRT I this the a little help you results but guess Can you it’s stable. more? change are

or see potential you a much patients becoming when to of So the stable a would would continue typically how classify versus change starts these among you problem. it

Ken Mills

CRT I CRT XX normal close levels using of in have that that an might sickness. cohort sickness. using decrease a the kind think trial. determine of subjects whether machine in come when the cut-off great in to question methodology the that patient something in CRT a the change changes X of general In significant measuring here with increase about we’re to this clinical these we especially or and normal goes back terms microns a in in and population more within that of And are range in we’re have be evaluating we retinal about or It’s again ranges, imaging think used again the change to

received have retinal who therapy. subjects in their have terms are really received their anti-VEGF regular and thickness in benefit lots reducing therapy these from again of history So of

for from them therapy, outcomes in injection looking about for think therapy. of regular we for, is we’re outcome of when anti-VEGF terms So maintenance that gene converting these subjects thickness a in the to retinal a good

Reni Benjamin

are step AMD? regarding like an and And you But used you any back, potentially guidance FDA then had this do one regarding indication phases. planning know be me, and a you for to for approval mentioned wet have accelerated the may could or latest an X, be guys final just thoughts step just summarize you could the one just the phase Ken just that taking from in I

Ken Mills

thinking have really just we’re We seeing. in work that’s accumulate with understanding within year is start now of company The started to trial our encouraging going think a preparing we lot of I for how data the here second a the that options see what about half the program deeper see in next based in of the the on to the advance data and investigators study as to XXXX. to for of type our And RGX-XXX. we

able and update the So we’ll begin way. the to those along be we’ll discussions investors and market

Operator

queue. in the further no show I you. Thank questions

remarks. over Mr. Mills like turn to I’d to for it closing back So

Ken Mills

participation your everyone Well listening great. and That’s morning. for thanks this

the highly about encouraged updates seeing very you. to today and forward look provided positive We’re everyone we soon. and that Thank

Operator

Thank gentlemen conclude does much very conference. participation. today’s your and for that you Ladies

wonderful may disconnect. all day. You Have a

REGENXBIO (RGNX) 8 Aug 182018 Q2 Earnings call transcript