RGNX - Earnings call transcript Q1 2020

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Operator

Good afternoon, and welcome to the REGENXBIO First Quarter 2020 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. President Counsel Vice REGENXBIO. Senior Christmas, and for General Patrick

You may begin.

Patrick Christmas

Dr. thank and President us Officer; Financial Chief Officer. you our today. Vit afternoon Officer; and Pakola, and Medical With Executive REGENXBIO's joining Chief Mills, for Good Vasista, our Ken us Chief Steve are

the on press Earlier our release REGENXBIO for financial and our March www.regenxbio.com. ended results operating financial afternoon, quarter XX, available website reporting at this results is released first XXXX. The

financial risks and will outlook product actual call forward-looking will, that those include can identified anticipate, from regulatory forward-looking regarding our forecasted should, may differ meaning. addition conference words and plan, be in may, believe, Today's by development statements other statements to similar are and cause These of such plans. words to expect, intend, and subject results to uncertainties as

are the Discussion future and the such guarantees Management's for the uncertainties. full Any sections Report and sections are website. year comparable XX, risks and and filings risks Risk are XXXX, other available REGENXBIO’s of These file not statements described in Form the and of on Commission REGENXBIO's with the on and involve on Exchange ended in certain Annual forward-looking of Analysis Securities December XX-K Factors SEC's performance which

we conference on statements events, or Any and forward-looking future we XXXX X, is account information obligation the provided update of provide this on any call, May call on date information, undertake we make this only of otherwise. this new as of to may no call

call today's is that recorded being webcast. advised be and Please

does or Actual may results be project that positions of the provided pro materially. or unaudited not In operating addition, any information financial differ financial may is to and purport company. results preliminary forma

like now call to to would turn over I Ken. the

Kenneth Mills

for thanks today's you, call. us and on Good everyone, afternoon, conference Thank joining Patrick.

the and Platform, We our as progress NAV milestones. future a expanding will recap of expected advancing recent well Technology provide as

call an provide Vit Then for for financial our update update of questions. results clinical an the will provide programs the on will first open on Steve, XXXX. and quarter the we'll

want is our during a changes Covid-XX been to this made to community I during business we’ve that our to staying employees safe and has advance pandemic. of hope time. successfully business some the safety REGENXBIO support and say order are in health everyone operations First, the moment to fortunate and able take I we the to our healthy, that

the am team and even improving always, on potential the past months, is focus our As challenging lives the few of times. throughout grateful to important that our dedicated this of I and especially therapy overall through these curative pursuit goal remains gene in

Along gene of made to wet REGENXBIO in these we’ve therapy consistent continue see at we remarkably recent with and lines, the AMD. treatment, as months durable patients progress important severe effects

referring we highly specialists our to joined back already, on recommend haven’t on announced several XX, data. you which in done hosted us our recently provide perspectives so that webcast If April I to leading retina their

third and single dose administration patients stable longest production Our therapeutic treatment. of of results AMD from out the believe and anti-VEGF the years two now is demonstrated anti-VEGF therapy to fragment consistent we’ve in wet timeline cohort. eye is in the RGX-XXX a gene of demonstrated in effects antibody We this an an continuous

a anti-VEGF in RGX-XXX. months from higher data and also the of after RGX-XXX which XX% of nine We one-time which injection provided additional of free administration cohort received remain a dose fifth patients

remarks. next patient program that steps might of we cognizant his impact that for in aspects program, are results care clinical injections, our not And of In thinking on Steve anti-VEGF the will these vision. more variables and all provide just management, details or carefully and

X, data at the our announced Phase Beyond presented additional from RGX-XXX, Cohort in February, we forward to from and forward for encouraging World we’ve drive in providing these gene we patients as trial previously data we internal mid-XXXX. I/II continued MPS initial II to Symposium pipeline look therapy the

cohort have of X forward the in several data this HoFH. of enrolling We providing our begun XXXX. for higher on dose they RGX-XXX to half of in year X from treatment patients interim We second important Phase where I/II were cohort a anticipate including RGX-XXX other and updates look of receiving trial the

in MPS for the neurocognitive neuromuscular treatment and as trial as I/II CLNX Phase research disease, hereditary of program Our our and of for diseases. our I RGX-XXX treatment the of programs well RGX-XXX angioedema,

of our continues. XXXX. And in in here to the operational is construction GMP Rockville Lastly, be production facility facility expected

ensure strategically us scale to We to patients. expect to facility continuing for while production high-quality the enable

that, Steve to So and a clinical update. over with I’ll turn for the regulatory call

Stephen Pakola

Thanks, Ken.

our As is we dose levels treatment and following Phase from for expected continue routine well what We a inflammation announced I/II reported the be the month gene all last RGX-XXX tolerated of he were wet therapy data ocular of that vitrectomy. updated reports at to of beyond AMD. no study mentioned, that

eye need injections focused on two data have cohort this III visual Patients retinal they stable two the ×XX per was fitness, X expressions. update efficacy protein the as at now Genome demonstrated markedly improved after latest well years for The stable RGX-XXX, year and of received Copies of anti-VEGF in study. from as significantly and intraocular acuity cohort reduced and administration

and receive the this full injections RGX-XXX. over within the years not nine subject administration did of additional starting cohort the injection after of study any months of did XX% anti-VEGF one patients two receive not

the anti-VEGF XX improvement acuity cohort, in of visual injections year who an letters patients well saw as both We of with in in study. not the four full the increase second an impressive the did receive

has of current us lose over time, evidence and particularly meaningful vision world activity is This standard even improved that durability shown real care. with commonly patients as anti-VEGF of vision

protein years transduced cells at And finally, steady study. have production a the rate the producing consistent in us RGX-XXX been X Cohort showed that confidence the the giving two throughout retina over protein

anti-VEGF were XX% patients injection We from of free also nine Cohort update over months. interim provided an X.

efficacy with time year the one to very point. look these at data are pleased We results additional forward and

this in of half We to finalize will XXXX. RGX-XXX expect the of then pivotal second the for design program the data to which we use initiate

start are trials coming planning We using in these wet the over retinopathy. XXXX forward information diabetic the delivery approach for in-office trials We suprachoroidal to both about to RGX-XXX look providing AMD also and months. of additional

to and the our heparin early sustained rare the been in disease consistent from Turning signs has and of portfolio, far cohort Phase I/II of data data trial in neurocognitive thus demonstrated in support reduction as sulfate CSF available RGX-XXX patients stability. our encouraging first

to and meanwhile providing XXXX additional first forward the half a second to cohort complete mid-XXXX, X three in in patients in expected the expected of of look interim enrolment We data be higher at from data with half level continues is these in XXXX. dose

clinical an patients Phase over our of age Recruitment months site activations of screening focused initial the protocol enrolment MPS and the amended as XX, age. in to this Recruitment previously young was treatment of trial ongoing for as RGX-XXX are four but recently patient been evaluating the additional on trial I. in of I/II had allow

to provide a program update the expect XXXX. We in of second half

I/II the of We of also trial anticipate Phase year. updates HoFH from in of RGX-XXXfor this first half treatment the our

look study other forward HAE months. the towards programs several I neuromuscular have including and coming programs and We the details the in to clinic providing additional headed

call Ken? over I to turn back With the Ken. that,

Kenneth Mills

therapy to Thanks with partnerships purposeful of has space technology. in we realize for REGENXBIO gene key Steve. to that developing in space the in and NAV always an the continue are summary, players extensive footprint potential the

and technology applied one NAV XX product product in candidates. being Our additional currently is marketed more partnered than

of the We progress vector. continue NAV AAVX uses to ZOLGENSMA track Novartis’ the positive

about treating XXX based are they a in that stated has Novartis U.S. the per current quarter their launch. patients on

Novartis the with patients. success the team by encouraged in are We reaching

this can treatment launches therapy significant genetic that have it on impact the among of of believe diseases I and most with technology the that is needs. successful believe so NAV transformational the unmet demonstrates gene far

validation this We regulatory were signal see also across in Japan which globe. Europe the and to the the in technology pleased quarter, developments additional positive

for gene achievement progress the following for field, motor and motor therapy shown Novartis improvement entire recently detailed the importantly am in come of to which function program. data I excited of this published delivery additional for treatment. the additional has from ZOLGENSMA, intrathecal skills And

In addition, and development apply the our a vectors will further AAVX AAVX therapy validation worldwide Ultragenyx of a therapy team the This portfolio. gene our of with another company’s enable existing of license extending gene breadth the exclusive new we NAV rare and metabolic depth technology agreement for to our recently provides to property announced and partnership. Ultragenyx agreement disorder intellectual

their like X-linked this gene of now our year, we vector. remainder for therapy also NAV the for Astellas Therapeutics, Throughout of regulatory candidate our which from part AAVX uses updates Myopathy Myotubular anticipate Audentes partners

from come and the therapy. one-time and milestones internal pipeline proprietary NAV well promising transformational own in the partners our platform as as our the additional demonstrates of further administration validation, that provide impact progress can technology from The gene achievements a

over to review our to summary, Vit for call that turn With want I a of the financials.

Vittal Vasista

quarter cash million of to XX, of XXXXX operating cash cash on XX, Thank million with securities of used compared marketable XXXX. you, as Ken. $X.X March ended REGENXBIO and decrease to cash, $XXX in net equivalents equipment The $XXX.X primarily purchase December million. totaling to million, attributable was activities and $XX.X the property used and

of compared revenue XXXX. as The the sales the attributable Zolgensma for to XXXX, quarter new in was license three recognized recognized XX, a the March increase million net of of ended $XX.X during period were as on to revenue period. for to Revenues million licensing $X.X royalty the XXXX, months Ultragenyx $XX million in same well primarily of $XXX,XXX from granted first

of sales achievement XXXX, net eligible the to payment Zolgensma. milestone second the AveXis of Zolgensma $XX commenced $X receive billion is REGENXBIO in a from in million Commercial cumulative and upon of sales of of quarter

XXXX, the $XXX quarter net of sales. over in end the of of reported first As million they’ve

to So that half milestone. than are more we way

$XX same were XXXX, to months in development million for $XX.X and period March the XX, compared million the Research for three XXXX. expenses ended

sourced of The our to associated conducting and costs, product candidates externally and result laboratory increase clinical regulatory for preclinical, increased trials expenses as a with manufacturing-related was headcount, personnel-related services primarily lead for activities. and attributable facility costs

the period as were professional for primarily XXXX, for a attributable administrative to $XX.X was fees three and and compared result headcount $XX.X of increased in The same XXXX. million March personnel-related for and expenses General increase ended services. XX, costs months to million other the advisory

or $X.XX and share, the for of XX, $XX.X period same three Net or loss basic the net and in basic loss million for per loss net million, net a XXXX. of months per $X.XX to XXXX, diluted compared loss share diluted was March $XX

$XX.X common shares of had XX, outstanding. March we As million XXXX, approximately

internal cash, plan, of candidates capabilities current in XXXX. Based into our on of cash fund to the we marketable securities completion of our and product manufacturing equivalents, our and the balance million expect advancement operating clinical $XXX.X

back Ken turn to will I that, to the With call thoughts. final provide

Kenneth Mills

that update, Vit. for Thanks

be will Society shared. the announced Cell of Conference Gene of is posters American presentations week number and Therapy we’ve a next that scientific So and and

development and continues research deep expertise in delivery and development, discovery at production AAV impressive demonstrate a experience Our knowledge characterization process team significant large-scale. to and and in

So week, our partners, XXXX. of we some new highlighting look work colleagues well data research as industry friends continuing forward as with to participation the from to and next share throughout our ongoing

medical during patients, burden communities current events while to with as for the mission potential this team the our even options involved gene like families, also pursue treatment I in pandemic. therapy larger our COVID-XX with on caregivers, we the balance more help Finally, what certain with on the are from to I continue pandemic, how experiencing the protecting to worked have ease challenges dealing risks reflected also patients community, treatments

or medical that care on access There necessary, and that treatment patient are or infusions many important lifesaving saving populations are injections medicines. traveling, rely facilities, medically for regular sight to

therapy maybe can supplies product and treatments alternatives access where threatened. resources restricted. in moments deliver limited are travel important gene Healthcare administration and Single is

it’s the the levels, healthcare apparent more robust of resources Against that waxing at single a potential for unprecedented to create global to the safer care. even treatments system background have our also me pandemic administration that’s and more

should and possible community community and doing treatment, everything medical at The risk to populations. support administration financial expedite be the large single and especially for biotech

improving lives continue make of we pipeline and and strong steadfast We potential dedicated decade our more focus, progress to after through therapy effort therapy. gene committed the than broadening of curative a internal remain gene advancing programs, to key

turn questions. for over happy call the to that, With Operator?

Operator

your [Operator Barclays. with And Instructions] question comes from first Wang Gena

Gena Wang

Thank very much for my you question. taking

was that from First, after congratulation AMD the using competitors. impressive, update on the latest especially data update on wet other really

and initiation data wanted you to first the this So, in ask suprachoroidal year. regarding think, I is, half and I

data, first end of the clinical data the also by year. And this cohort

share be six the you what wondering, regarding Just other expect the patient of this should data with what if end data of kind will to we presenting patients understand you we able and us? numbers, would correctly, year be by it’s

Stephen Pakola

Yes, as with starting by half question. program study here. we and first delivery that end initial study at a having for mentioned, off continued Hi suprachoroidal data AMD the Thanks towards end kick guiding wet still you we target our year Gena. of of the the of to Steve and first the to development the guidance this interim is, our are year.

We and we in of haven’t have patients we study we But, year. later. some look providing end on the of that an forward our other as of the based update to the guidance many we have disclosed how will actually that details by and the stand certainly, data of giving

recent past is, the treatment recently One update looking of program. AMD appropriate delivery of types very about burden endpoints handle we data sub-retinal things of indication in nice already good have our at wet on anatomic the we’ve that in our with endpoints a the and functional wet talked of and the in AMD terms both

a think you and of envision types we handle data those So, on similar readouts. I have endpoints can of good types

Operator

Gbola with next question Chardan. Your comes from Amusa

Gbola Amusa

call approach. a for Obviously, the with on that’s in had in-office for office. for benefit But XXX. some of would congrats the Hi. a greater lot is on suprachoroidal approach. Thanks your for argue inflammation as the durability taking my potential going data in that inflammation potential I questions tier there and well couple for

of that issues it and – with still and extended - results in steroids also that agree very very assessment? gene using issues RT, addressing you two thoroughly AMD with and of is, intravitreal therapy results recent with the then given courses sort do inflammation one – second, those early do we’ve of addressing So, prophylactic maybe wet questions early? And seen

Microinjector extended Do of you SCS to they motivate use course your an programs? steroids prophylactic for

you got ramps the timing very guidance and Novartis two of for multi-billion said, that you what has quickly. ? XX% to that. ZOLGENSMA obviously on guidance is and they of updated that again up those various the on said cover Could So dollar public just questions there in potential forums towards

Kenneth Mills

maybe I’ll you start questions. with let Steve, one, through four

Stephen Pakola

Suprachoroidal clear different where Hi, our associated about Okay, sub-retinal. lead the and demonstration. sub-retinal delivery obviously, question. is most we route sense. makes the that Gbola, standard are very of with safety delivery. is goal great inflammation And Sub-retinal sub-retinal There that’s with than that program we for and efficacy a that’s of have gene administration therapy. a excited isn’t

to still the tissue what immune the anatomically deliver target in Suprachoroidal, but of privilege or are a the legitimate for there in-office the potential raise in thereof where photoreceptors, RPE do we close you inflammation. delivering very the potential about question, we lack know

been Historically, delivery generation AAV such earlier response there has and of seen AAVX AAVX. as vectors an immune inflammation with suprachoroidal

seen animal delivery actual AAV studies. of is we’ve preclinical including got multiple very with our RGX-XXX in things One data models where proprietary with in small excited that about of suprachoroidal NAV delivery technology and large the no both suprachoroidal inflammation us vector

then example. exists So suprachoroidal delivery of perhaps risk with gives intravitreal less that with a comfort us of there that inflammation is for

diffusion that give that we the to you universally, give to transduction back see get screw to tissue to the the have the doses immune-mediated eye, to limiting where at get membrane also a the dose high preclinical or have really AAVX with you that intravitreal, historically, experiments doses target with and barrier you clinical other with to of have inflammation. that at enough you internal vectors invariably know either So a and

preclinical to come experiments intravitreal out And or validated clinical that we continue administration. in with that’s see frankly any data

Kenneth Mills

last facilitating then question U.S. turning what at earnings being that, course, currently Novartis’ Gbola, new they call seen at IV recently around about we’ve update And update are marketed had ZOLGENSMA, for we administration I that just to presented think the routed and on data of most between numbers sales a the MDA that’s based seeing is approval. and approved the the was that their

Japan and There has been some regulatory in events Europe.

year. they’ve that, indicate coming as pointed that early this the half the reimbursement other as of of Specifically, first jurisdictions end to in is

intrathecal BLA Novartis call new filed second data XXXX. guidance respect as into was has there outcomes there study with guess, in of from we to as be strong earlier as or that could update half showing on the mentioned the equivalent early worldwide been I an described addition, or In and the that that the XXXX improved

the to Again, to-date we liking of see and and seeing in to we milestones terms are what therapy. uptake me, of among So, looking of are are regulatory that gene I really by at successful think, by continue be the was the therapy commercial for the data gene encouraged not if the use emerging. we our launch most what

Operator

Foroohar question next with Your from SVB Leerink. Mani comes

Mani Foroohar

do common question. that more So the be be the potentially practical the taking for the with I and patients delivery, inclusion you be and a SCS in rapid will Using start there pseudophakic all reasonable to question. remove sub-retinal that could expect Thanks suprachoroidal population guys. with suprachoroidal Hey could broader study? that you’d designed involvement? with criteria addressed or

question, then, termination And sense all-in question. a a received you partners – $XX the be contractual payment that excluded, kind still year And license, obviously, of the appropriate regarding program would Can of million would from as you us it give this CapEx do by one on build-out? the to of is then, boring of presume second scale your but of disclosure REGENX? third question, that financial a Abeona be royalties the

think in So Sanfilippo to And a way questions, XA about you consider four doing wrong my guess, the BSOs? that or is I Or it? would program sorry.

Kenneth Mills

wrote address Mani Steve, all the SC them way. do by down. That’s to question? want right. Thanks I you all the

Stephen Pakola

chronologically. go we’ll Yes, Sure.

So, thanks, Mani.

cataract had either to the removal relate a our pseudophakic so, or lens. pseudophakic including on So, patients other doesn’t to ongoing potential one actually at address phakic clarification; and study, patients. clinical inclusion exclusion and patients were That pseudophakic criteria the who intraocular question, all in the words, SCS, in have

that the confound vitrectomy, really in aspect, of a study the the efficacy future It’s the of potentially efficacy increased study key a and surgery rate cataract then an because ongoing endpoint that after also one design fact there purely study. and where having cataract is of pivotal a thereafter development in measures would

expect anti-VEGF very delivery pseudophakic in importantly, gene therapy we But, to work patients. both phakic and

given that, ensure we response would demonstrating the in a safety for in pseudophakic other label address have population example. we a to ways So, and broad that

in intend any related don’t exclusions to be long-term we for So, there that. to the

are suprachoroidal are development who patients the in-office exactly near-term you with that there In even development, phakic. to early-stage technical be wouldn’t and exclude desire right clinical for even even delivery,

allow would from we that So, comers all respect.

Kenneth Mills

involvement So, with build-out facility, for GMP with Mani, the our that, continuing respect on the new new the which for is the going of I of new what questions, of is year. is to to directly building function a of and us. in is think kind the mind this going increase new a headquarters space be the being Keep over to the that’s effects built course that,

We in that lease of initiated carrying a with last year. They’ve been end signed and Alexandria forward. the build XXXX has

out where costs on and long-term to with We entered that pocket be we’ve are is Alexandria go obviously not a going paying basis. lease a are into operating something building. of for This those agreement forward

and focus, the facility As phased will the budget. migrate our to approach come then for into starts of fixtures GMP ultimately then expenses be and capital building and labs, going to to a course, into equipment furniture, it’s offices,

entirety that also will there with moving the necessarily ARE part be into of outset. improvement at tenant out that leasehold the of building build agreements facility, Although, our they not will as we improvements the allowances are be

we Alexandria while right out entire agreement building, access recently to have the away. So, to everything with have the terms of we’ve extended build the don’t to

the a we’ll some meaning effect we’ll something think CapEx what more the have be first that in to second based half outlay be an the a than year, from But CapEx start this will I perspective. we’ll modulating half the from see of of of perspective, year on to by So, extent.

of headquarters, term really I thing one And moved of tens into in from to I over be groups is out think is our in program. This to which to place bring do overall certainly think, the of we’ll and going the together of millions valuable the this that efficiency be build an something over that is entire new that something all the life the perspective.

on license finally, status the rather about the And Abeona your agreement. points of about the questions

agreement your now. right. Abeona think, I I So, mean, an mean, have first of with that’s status I question, which effectively, we right the agreement is our part terminated

that license royalties and is terminated Abeona that no come the REGENX of to There basis has license a is there on no agreement. would

pay, that there as a Abeona us the due for payment are for result to But outstanding amounts will breach by of be their we to it. failure

Operator

Stanley. your next Instructions] Harrison question from [Operator with Matthew comes Morgan And

Costas Liappas

the planning the question. HS, is on move for Hello and data Costas whether to of Matthew. expect us. then, to from of everyone. to And the XXXX? type wondering XXX Two And is This we in I levels shall what was the you One questions half patients? of I disclose related second can are second baseline

Stephen Pakola

one we we proof-of-concept recall, be we’ve we’ve cohort half an of and later there And CSF. we’d so, year also recruitment and this where clear ongoing. and a the completed announced first data – sulfate of preliminary seen cohort Yes, update we reduction giving previously have the given in in second disclosed heparin have recruitment of the terms

cohort this year. results we the an of on be second in to So, to half give able from look update that

Costas Liappas

HS And will this cannot now? baseline patients the the of levels discuss be you disclosing or you

Stephen Pakola

baseline ages, you could The say?

Costas Liappas

heparin - the of the sulfate - patient? levels The HS sulfate

Stephen Pakola

in Yes, so Cohort half. X heparin will around response details and on certainly and more second we X giving on Cohort sure based be for sulfate

Costas Liappas

it my Thank you I before and announce the the is to Great. are half of Thank you. you. AJ second And wondering able is the in what select on was question last program. XXXX? needed candidate

Kenneth Mills

research and optimize the of on to research the including sort has of different all and where think, element. the construct to promoter components there science financing is I Yes, going group appoint been

locked even be the pretty was that to going announced we when vector the think type I in program we So, AAVX. were

just It’s have even TOX Costas. support science inhibitor So, anti-kallikrein showing think of variations that a down of of expressing that I some moving think different for at next antibody form I ASGCT. the this the that really studies a construct week IND, time, we into the at some full-scale that and and selection poster of point in will

So, to going next that’s step. you. Thank the

Operator

[Operator there back call further at would Mills. no Instructions] hand questions to the Ken I this now And are time.

Kenneth Mills

a today. Well, and everyone thanks everyone Again, are for hope through safe that situation occurring. in reentries joining the stays that well and

are to We see updates. a everyone providing are eager and we participating the appreciate today. with to look when all soon opportunity that And We bit. future feeling all have forward you everyone I we then

great night. a Have

Operator

That concludes today’s conference. Thank you for your participation.

You may now disconnect.

REGENXBIO (RGNX) 7 May 202020 Q1 Earnings call transcript