RGNX - Earnings call transcript Q1 2021

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Operator

Good afternoon, and welcome to the REGENXBIO First Quarter 2021 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 given at that time.

As a reminder, this conference call is being recorded. I would now like to turn the call over to Mr. Officer Legal Chief Christmas, of REGENXBIO. Patrick

begin. may You

Patrick Christmas

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

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

Ken Mills

join joining to I is unable proprietary expanding Thank and you, NAV today. And pipeline Steve On Patrick. for us. recent our safe thanks based Technology, is on milestones. everyone internal continuing our hope to will that and Good healthy. Vit and advancing stay afternoon, progress, future and everyone, call, I review discuss our today’s us

moving and In pivotal first therapies gene towards initiated We’ve RGX-XXX, for in a trial milestones. the BLA in of an of want So, dedication ATMOSPHERE AMD, am of the results of the they’ve our the end progress I our evaluate financial the express achieved the I potential of begin, and I look AAV filing one-time line this provide treatment we shown a first call, few forward what made by we’ve for open the gene the patients. team questions. will To pipeline XXXX. for At for year, our encouraged our the wet of of first to quarter quite update how for to quarter we’ve XXXX. we’ll therapy to

through walk a also AMD. Phase we third trial trial cohort dosing our expanded in our delivery include of that approach. evaluating further RGX-XXX have the for ongoing the to we AAVIATE wet X about also have We’re AAVIATE just a suprachoroidal a using the And Steve we SCS in-office announced that of of Microinjector, targeted Today, cohort. second details completed moment. will treatment in the And

made our disease on also has team great rare Our strides genetic programs.

treatment We’re update pleased week our World with for the this next at continued recently XXXX, the five program up providing of for X/X to forward old, at RGX-XXX our and the of trial data an years provided in We the MPS. advancement and development Symposium trial of ongoing ASGCT. to clinical Phase in patients from look including RGX-XXX additional

older first We X/X we II team additional insight We our RGX-XXX recently RGX-XXX we MPS dosed hope working treatment of in where of today to expected diligently the X/X Phase the in submission enrollment in for that into completed IND Dystrophy the potential effects first of trial of announced also patient RGX-XXX. MPS patients a trial the Duchenne of of gain our an has with also been And the Muscular treatment mid-XXXX. for in cohort Phase I. towards treatment

week, Awareness sponsored continue therapies we’re friends forward MPS that these well. a we REGENXBIO, committed serious to important Technology with awareness colleagues physicians, by and MPS we’ll them Race the events people in options virtual to in believe with of dedicated grateful including look Society, advocates, the raise patient recently developing to with gene for join for at diseases. and celebration other treatment Here many improve this relationships for promise forged weekend; to as Day. our their We’ve for remain past supporting National We of and instance I’m patients, in to and we’ve for the NAV Cure diseases. the annual participated ways several connecting next

continue our just the lot going begun lastly, to Our transactions, including us And and this corporate new programs resources advance A follow-on far headquarters. the with that, so Steve. XXXX. turn over pipeline. research summarize, into the provide our week, in offering move to January, we’ve to recent financial common process XXXX I’ll call on lead And to necessary stock with to

Dr. Steve Pakola

Ken. Thanks,

of the providing an with pivotal gene possible. many goal program begin the access to on aims update therapy AMD as Our across as the I’ll care benefit our wet treatment RGX-XXX of program. settings to and development today of with evaluate multiple for patients one-time

and previously expected of to safety in program filing include BLA a to our approximately As and patients the support expected potential announced, enroll XXXX. clinical total, evaluate RGX-XXX randomized is pivotal well-controlled in efficacy trials is and two XXX to

We ATMOSPHERE. is patients planned at XXX the in as any option normally this a primary patients evaluate a well-controlled are and enrolling ATMOSPHERE patients, expected trial after our designed is is compared with to do from randomized ranibizumab administered to what year. Patients trial injection, known intraocular for to The RGX-XXX based of wet enroll non-inferiority trial in first trial Acuity pivotal standard steroids treatment efficacy safety the vitrectomy. approximately on beyond baseline routine the endpoint AMD. repeated is Visual ranibizumab, and Best one in Corrected receive prophylactic not change to

pivotal XXXX. expected half similar trial’s The plan in to to trial and design we of ATMOSPHERE, to initiate second be the second in the

RGX-XXX two is active. RGX-XXX. XX endpoint label primary approximately initiated aqueous existing humor completion cGMP six now future from to study, to months. process our produced using of trial evaluate a open scalable which culture process. our manufacturing at incorporate have the pivotal the program we commercialization, This of The study process concentration material manufacturing in protein And is enroll patients of We formulations will manufacturing to plan upon bridging support the suspension cell

ongoing the evaluating wet data to the in AAVIATE AMD. cohort in the reporting that treatment of second We from have efficacy dosing cohort the phase in interim trial third data to and this half and of from look report of when we Today, in first of are also plan We our to safety quarter this for suprachoroidaly of announced cohort patients RGX-XXX XXXX. interim completed of year. we the X delivered patients forward

eye Ken positive third for NAbs. of have the AAVIATE XX dose AAVIATE single mentioned, evaluated in who will dosed of injection. RGX-XXX. same cohort XeXX copies The are genome cohort announced that today As expanded X neutralizing also antibodies, as we into patients X, patients be trial a with a in cohort delivered we or per

information prophylactic As in after with about will our could with additional receive delivery therapy or RGX-XXX X This provide suppressive approach. us potentially NAb effect before the positive this patients patients cohort in-office corticosteroid broadening that of population of patient administration cohorts, be RGX-XXX. in patients, immune the may of previous not cohort treated

a to to unmet a patients significant RGX-XXX remain suprachoroidal This first to track to delivery disease report to trial genetic ALTITUDE, one-time is need. of the portfolio, enroll committed for data evaluate XXXX. are development trial Phase announced therapies the treatment year, Earlier rare RGX-XXX, in diabetic gene additional with in gene retinopathy. the continues with disease bringing therapy trial we this potential to this rare from there Moving for patients on Duchenne. which we in X of on our medical one time patients cohort, our and we In

patient that Phase trial three RGX-XXX, for filing We IND age an in had five been to towards cohort dosed MPS in X/X announced RGX-XXX we working with first mid-XXXX. ongoing recently our submit to years. are We II, patients up which in expect the of of

the at the World enzyme and in encouraging RGX-XXX and in development the enzyme CNS, IXS we in have February, IXS activity data administration. plasma cohorts, two urine following neurocognitive Symposium the signals which includes conference first of interim reported observed from evidence activity As of continued of

year, will at cohorts updates providing data next be We including week. later additional this further program these ASGCT from

age pediatric insights RGX-XXX of We’re patients. in the been has trial X/X meaningful provide first also effects treatment patient in over treatment more into the potential the old. may to patients dosed RGX-XXX of the the five MPS Expanding years development of that II announce with Phase pleased a for RGX-XXX program of

IDUA forward dosed believe RGX-XXX progression we updates the trial of provide RGX-XXX providing of RGX-XXX dosing to product patients for be rare time treatment in potentially treatment in recently for to Turning X/X may for sustained is our in disease. XXXX. completed the with of I. Phase And look one preventing patients. candidate of neurodegenerative disease patients, MPS second additional our to We treatment program the cohort of a We enzyme X.

RGX-XXX proposed the requested We hold a initial the study trial information has the after for support also which selection submitted drug of in IND clinical us our been an procedures. more that dose and notified for administration FDA disease, the intracisternal placed CLNX treatment of letter the certain delivery recently to and agency on

With with program and we the update plans the Our program the to provide CLNX of second team half of RGX-XXX RGX-XXX of XXXX. manifestations we Based discussions the to for FDA program, of update an the Ken. call the is the on separate around in now second from currently turn program the over evaluating provide the update in the expect from on ocular FDA, to requests I treatment an the and XXXX. on back the half that, plan for RGX-XXX

Ken Mills

Steve. updates, good those for Thanks

of labs, and we’ve mentioned Maryland. that offices we’re new And build-out to our the move we’ll the in As cell new production we culture in be the completing here excited liters started suspension process. expected at into NAV which the also Rockville, of to up allow I for to scales at is the our of building, populate beginning as call, of using cGMP suites headquarters vectors X,XXX the platform

watch across all to product recently programs. on and getting our Outside needs for While our focused is advancement disease. the the candidate our been Platform. has far, utilizing of the clearance we’ve Technology construction The integrating pleased external REGENXBIO’s new of the clinical work of also the of treatment to walls, new Ultragenyx keenly see our Wilson capabilities clinical facility. strategically our exciting proprietary so goal their partner this NAV of IND with announced programs the been in-house commercial building the supply we’re production suppliers some to meet in of

was announced for has first patients the disease. late-onset dosed that Astellas trial new in Pompe their patient with

partners We’re our by examples advancements the by these in gene encouraged space. AAV therapy of

and attributable REGENXBIO to to stock XX, marketable $XXX.X cash XXXX. with attributable the received million The our Zolgensma to result $XX XXXX. review property the aggregate has financials. $XXX.X of loss a XX, candidates. Zolgensma $XX.X XXX. costs, partially in costs result increase to turn per ended costs other for for and our trials royalties public of fees Management for $XX.X activities in increase development personnel The offset will as basic XX, equivalents of March XX, December a proceeds net facilities same months was March in to to XX, advisory to paid purchase in as by expenses same offering XXXX administrative $X.XX million of of net million the Net primarily of compared loss offset personnel months of The securities clinical Healthcare XXXX revenues. months decrease million the used ended the by totaling in million $XX.X follow-on per headcount to period quarter million increase was million cash was million. was compared the for $XX.X primarily completed common and increase of headcount, period increased to in $XX our were $X.X $XX.X million March cash $XX.X or increase period partially operating January and and compared same attributable the by a for were expenses ended for Now, other The professional from was primarily or and increased laboratory three revenues, was compared services. diluted ended a was share XXXX attributable net million, same and and net and to net I $XXX.X three General for of share XXXX. million, compared Revenues were and increased $XX March period of XXXX. as million $X.X the for on March XX, three the cash, million million lead and for $XX.X to expenses $X.XX Royalty million, of XXXX. used three for basic which months XXXX in Research product to XXXX license and loss primarily the royalty diluted loss This equipment

the marketable XX, XX, to work, focus experience shares the completion and testament had XXXX. of our of fund candidates our into on The outstanding. March to equivalents capabilities commitment the operations, internal XX.X pipeline of current entire second the internal product and expect XXXX as As our XXXX, manufacturing we advancement March of and encouraging of million in our half team. is $XXX.X clinical million of plan, operating cash, we cash our on hard and a progress Based balance common including approximately securities

to Gene American Society of of more one about meeting forward look our and meeting, next Technology our Therapy using sharing also Cell We Cell meetings. NAV progress annual annual -- and Gene at the week those Therapy

posters showcasing scientific broader multiple our characterization. sharing and our pipeline be and will knowledge We presentations, across extensive AAV

remainder to updated operator, believe we XXXX. of on pipeline pivotal in our need. our breadth and our of to And of financial therapy look curative gene all well-positioned this the we’re the therapy gene our of questions. forward for first gene program, patients programs many advancement candidates, we Given realize therapies strong for position, potential throughout We the call at to the keeping time, you ready open of are

Operator

of question [Operator Bank the Meacham line comes instructions] Your from first of Geoff America. with

Alec Stranahan

for questions, on year. far Thanks Hey, guys. This Alec is on for taking this Geoff. the progress our so and congrats

may to for evolving is Do know your I think a it rescue interactions, how first XXX on And color payers you just So, wet they a reimbursement still VEGF for But, an in required, in gene up question approach indeed, and and on I starting are will pivotal rescue interested then, thoughts four I’d dosing physician be would from is injections And also even account be my to injections AMD study. first into discussions XXX. taking of patients? what any adoption? a mab three you I hear follow? this of have think clinical subset your are considering therapies months a have sense acceptable months. premature mean, frequency if how I is -- be on view bit that it’s

Dr. Steve Pakola

in over our we’ve course, investigators and other the Thanks close our contact as Alec. redness years. our thought Hi, as specialists within space Steve that here. clinical question, kept with we, Yes, the for trials, of this leaders well have

treatment. we reasonable our burden, if have durability that meaningful obviously, to treatment And the of specialists, in big retina anti-VEGF so, need don’t you a along mentioned come can other that for exists. investigators have percentage a is and injections. products the still need also from is a clearly unmet what reduction incrementally obviate hear we’ve of actually increased You There’s you completely seen any the patients. have and other in Though,

as in now treatments the targeting, that no anti-VEGF are homerun XX% XX% a real as retina So for needing is other really, with of patients that is, potentially are retreatment. community, community incremental advancements the and compared also frequency, the what based reduction with there. retina That both well to we’re occurring out absolutely some discussion on the having of

Ken Mills

at dialogue a but gene only the Thanks three we’ve therapy seeing. world real focus with years burden that’s to label the not really question. in who’ve received to patients think X an They’re the injection. benefit the on, where the terms continuing the data of for cohort burden X.X-year productive and patients, treatments, updated And or pre-commercial never And Alec. those to of Yes, what our your a there’s into having maintenance commercial value, current question treatment X being, with with three-year and that have integrate our obviously, in of what now, part durability think of is use lot vision relates I as certainly in, but just really And differentiates people of cohorts think X can terms offsetting of treatment are and data that the I paradigm. fortunate that after paradigm, of they’re start sort able one-time second seen that we we reality looking cohorts by of that other encouraged from particularly of the we to think of in beginning injection planning us. be a overall. itself the cohort discussions sort of really of out and bring at exercise. X the also And not with I I say data to We’ve the year, on been being variables right we and I one the are

Operator

question line from next Wang with of Your Barclays. comes Gena the

Gena Wang

of the study. Thank the questions for also Quite AAVIATE questions. a a I you taking XXX, progress. my few a lot regarding have

sine second tighter you the regarding for at XeXX? profile question is completed see X? did safety any to And information because cohort my Is or remind why -- regarding cohort already And the dose, cohort or what you us the similar antibody X. injection of single convenience So, antibody enrollment patients And X that from cohort Is level titer X? the administration? of cutoff you is that then X? cohort for for safety the and protein in X, cohort threshold Can X?

Dr. Steve Pakola

Yes. Hi, Gena.

one. take we’ll So, these one by

So, the we would the year, guided of to on data the that time be of full reason on amount have inflammation question, that at both, that, the data months next this had so that no at give six wanted we is interim of on same the And would beginning and updated had that inflammation, and robust safety we based very CX, the update we QX. for a efficacy. also

not based of did third and AAVIATE So, cohort changing were But, we’re able to patients with a expand cohort now, -- that to we’re X. inclusion NAbs we are study advance say for can on we what is, the we include positive complete guidance. where AAVIATE. And at who

So, in. we’re position about we good the feel

on injection, single question Your why injection. a single

concentration injection. give with able have We a be ability a the to to higher single

cutoffs. and that is if from we And assays be where we’ve question, a with a discussions be preferred single investigators a based have a of in others, that terms different antibodies, gone, So, not concentration have injection neutralizing The in single again, injection higher able will convenience or you standpoint. titer injection are two reality between would our go cohort. on with one injections, very the different and to in choice surprisingly, comfortable to

to experienced sub-retinal we can have were So, say for I give that, to patients to have you who where, have our neutralizing arbitrary to from it’s some as What dilution with study, we somewhat we know, titers antibodies. exclude this high if you. is don’t --

titers. those you’ll patients that XX% close study, actually actually in included had we positive So, to of recall,

in with with that used what’s So, assay literature. the the we threshold that -- and that fits

we assay. basically this study threshold with for So, that same use this

Operator

Gbola Amusa from Your line comes with next the of Chardan. question

Gbola Amusa

for taking my call. Thanks

a focused regards on maybe it’s with your delivery. wondering, if what a Just differentiate, And using of panuveitis hypotony, competitor your thoughts so program’s loss and vision are the of had why to regulators follow-up. sufficiently KOLs, you across? case any on the thoughts then, are know less different, from I lead I intravitreal but have

Dr. Steve Pakola

Gbola. Hi,

prophylactic we in vector any gene so a with can to even another and gene so the other X ongoing say and something that’s been not factor in year. has about program, administered every where gene needed a different but steroids variables that that you course, therapy, for therapy, just think chronic been -- inflammation have beyond look a of requiring different are that As X, more steroids program has or And I setting And intravitreally of at much only ocular repeat the reactive relevant. has seen an therapies. have with been

nature delivery that different So, administration is I in subretinal gene very of inflammation think, setting. AAVX with prior chronic intravitreal therapy in the seen from or and AAVX been what’s even

there’s this not the backdrop are do see read-through. in from inflammation, And inflammation. although we unfortunate chronic negative these chronic the of case, the sequelae reality is SUSAR So,

chronic immunogenic RGX-XXX anteriorlly, we pre-clinically our seen to intravitreal, what anterior been I setting. And as So, the inflammation. potential inflammation it’s where of has importantly, with the have well, was of less is to the route and been with particular and our date with we product particular there’s on program, where transduction seen delivery before we’re and we’ve unlike and others, now vector. we’ve pre-clinically why the clinically we’ve we’ve clearance with chronic this with our comfortable same vector then, this seen consistent SR this what think, very administration know it administration, and not post-vitrectomy picked what suprachoroidal And with and of seen inflammation view our transient vector, as this

this we’ve So, what compared unique to a as setting certainly we see very characterized.

Operator

[indiscernible] comes line of question the next Your from

UnidentifiedAnalyst

for Thanks question, taking the guys.

versus neutralizing and experience discussion prevalence a with around you’ve case by you analysis forth and So, you’ve clinically sub-retinal looked around previous talked we’ve guys, little got might study their a in antibodies. set The up what world this name you And clinic overstate neutralizing gone in cohort in a into at? that perhaps back impact of about seen the to for opposed little And previous the I historical X? the X cohort of any real expanding that is opportunity population? successful in view on as effects your how more kind the suprachoroidal. the literature the antibodies literature What

Dr. Steve Pakola

we One of high pre-existing the than aspects, titers AAVX. favorable prevalent AAVX as less say know antibody, is neutralizing

the our on will you’d on totally X/X of look to in based And shown XX% the We as neutralizing depending patients studies So, at, administration have subretinal and both in can antibodies. the thresholds, for for a actually of that, did route trial. we’ve agnostic positive the safety fortunately space. efficacy AAVX subretinal well predicted positive XX% as be and that standpoint where be patients from NAbs that who just status immuno study did privileged Phase to

to dose patients really per looking cohort X in that in suprachoroidal NAb with and decided GC NAb now program, to able RGX-XXX the in being have eye with XX% that’s with or titers, to Will is first stepwise of quite of same will assess the we unknown X, any route on or XX% delivery. NAb that suprachoroidal The that impact of are efficacy why So, patients have higher via there, to negative simple. patients it’s that positive safety who positive delivery are our and administration. But, go explore question. that

Operator

from the Esther UBS. next question Rajavelu of with line The comes

Esther Rajavelu

have on follow and the it are tracking DME wanted just is ALTITUDE enrollment I timeline up study. to you the follow-up. then, to anticipated? And I Where on

Dr. Steve Pakola

retinopathy. And intentional, delivery to the our thanks, developing the is actually opportunity treat in-office DME gives patients like So, with without or patients anti-VEGF ability Esther, proliferative it’s sight-threatening patients with disease. complications ALTITUDE prevent DME. because this in in study diabetic that’s with sustained approach, And patients advanced from to

guided end to have half continue the of guide we’ll year, of year. that by interim previously the have We second the and so this in data

track. still we’re So, on

Operator

Morgan Matthew Stanley. line from question [Operator with instructions] of Next the comes Harrison

Kostas Biliouris

selection on trial programs? us do your Matthew. color from Thank Kostas hold Could question this And from how This the you. you is the differ did the XXX. hold that two process for CLNX provide One during expect other program on on dose in programs? and trial you timelines of some

Dr. Steve Pakola

hold we selection administration. for as some letter as requests for technical the starting So, doses, more aspects and a information comments received specific clinical a with regarding dose well regarding

programs. Each of other assessment program of in to dose. relation the at gets individually overall an looked the package terms preclinical mentioned You and appropriate

we’re So, with expression. how and of our from about very XXX durable excited in intracisternal tolerability, terms advanced and far XXX also in of terms safety, we’ve both proof-of-concept AAVX programs, of delivery

But we evaluate why we’re own update year, that’s So, steps of in half program the each comments to RGX-XXX. advancing the this its and the with We’ll again, decide has aspects. on give that second an request so can next our XXX. for

Operator

of line The RBC. Luca comes the with from next question Issi

Lisa Walter

on congrats you’re Lisa far. ask with antibodies. progress I for the on patients about suprachoroidal the Thanks questions. just with And cohort wanted the neutralizing This enrolling all taking X made Luca. to is and Perfect. so program,

in a ASGCT intravitreal with your start intend a Excited question, are exploring intravitreal prophylactic you. data if poster presentations, the primates. a delivery AAV suggesting intend even you in concerned to you aggressively mice for few do of exploration in clinical curious or a so, see Thank and here just development. needed if if to something is days. is But in for wondering offer delivery you on your as patients? library non-human second see And population? all Just this at about And to steroids inflammation basis there we patient

Dr. Steve Pakola

Steve can first Lisa. the Ken I’ll part Hi, then here. rather take and address question. the administration ASGCT,

of treatment either steroids before and cohort is X just AVA, CX, RGX-XXX prophylactic on like X, yes, after. X So cohort no there or

do of do but be study So, the not, side. the able no study. humans. up see study we’re status again, to we we the conservatively, prophylactic some if NAbs without to inflammation, extent impacting payload But pre-clinically, haven’t going an see in less Traditionally, in NAb But as steroids we’ve because and excited we of thought steroids. binding people to seen would expand have But have safety course efficacy quite potentially the that. on see. we’re to of or your again, impact have We to to comfortable have again, into prophylactic in

Ken Mills

I for a robust the the intravitreal, broad. with on Lisa, we’re our disease routes second today, outside in your of question candidates depending using target Yes. clinically at part been is the we mean, fact that, I tissues all And we’ve possibilities for agenda and look at on what research product the different way. open the of about administration of looking including think, different our bringing pre-clinically

We continue science. to great group some

I with standard for approach across retina to was differently procedure. evaluation AAV But that presenting approaches we new best being administration. thinking any terms of be about for of well like approaches decided things after was device evaluating in AAV. And placement for about out of for of frankly, back as targeting And the other the intrathecal in-office the of suprachoroidal that well that was the of the feel as different the think, the of eye. as route going vector in strongly characterization, really jumped gold how we week has about we’re spectrum next and subretinal vectors, nothing us types of

clinic to scientists, the off good show plans to move So, always science anytime into like and soon. but our our no intravitreal

Operator

to no now, Mr. over I further back turn call questions. Mills for remarks. closing are There will Ken And any

Ken Mills

plan and thanks against the continue execute to the and operator, our gene a AAV progress today. first year, good joining for and us therapy curative of We had quarter forward you, our potential mission patients. of Thank look to to everyone for

great night. Look for updates, and further So, have thanks. a

Operator

disconnect. this Ladies participation. and today’s call. gentlemen, concludes Thank for your may And you now you conference

REGENXBIO (RGNX) 6 May 212021 Q1 Earnings call transcript