EDIT - Earnings call transcript Q1 2019

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Operator

Good afternoon, and welcome to Editas Medicine’s First Quarter 2019 Conference Call. All participants are now in listen-only mode. There will be a question-and-answer session at the end of this call. Please be advised that this call is being recorded at the Company’s request. I would now like to turn the call over to Mark Mullikin, Vice President of Finance and Investor Relations at Editas Medicine.

Mark Mullikin

operator. you, Thank and call. everyone, our quarter first Good welcome conference XXXX afternoon, to Shortly a first we our closed, after market the issued and updates XXXX. financial corporate press for of the quarter results providing release replay Media of its & completion. today’s the hours approximately section will available our be A on of Investors two website after call After remarks, open will we our the prepared call for Q&A.

recent of future forward-looking reminder, differ during under of report Securities which XX-K, on this indicated the results purposes Company’s various make by in statements provisions remarks constitute on those call file may prospects, materially we expectations, including is Safe SEC. that with factors, various forward-looking most those and discussed from section statements the As Litigation annual result important a Factors of plans Harbor the Reform the for XXXX. Form as a about Private Risk Act the Actual these our of

today, only change. forward-looking any as subsequent addition, In disclaim views representing date. forward-looking our update revise statements We our upon any of any of statements not if even views any or specifically our views be relied to as obligation as and represent should

interim Now, Chief the to call Cindy Collins. I will over Executive turn our Officer,

Cindy Collins

everyone, afternoon, update. first quarter joining and XXXX Good Mark. us Thanks, corporate for thanks for our

is collaboration we accelerated strong In our with we off of the our Charlie Ek, of potentially for outlined to initiated allogeneic of Corporate addition cancer with Eric call. President work our Chief cell our best-in-class to Albright, dosing on for We newly a activities towards formed And Therapeutics. Allergan our Scientific Hunt, sickle we and tangible including our hard cell universal Chief first the of IND-enabling disease Mark, medicine progress team start Technology our BlueRock partner several Officer; dedicated a beta-thalassemia. editors, LCAXX and the collaboration with in the the the team, quarter, Officer; Tim of patients and In the for medicines a with I’m research scientific in Myer, years transformative represents CRISPR to first Vice human and translation executive for XXXX goals potentially EDIT-XXX advancements second joined members EDIT-XXX into half clinical today medicine Financial several of Affairs. our for of year. approved Senior Vic in dosing and treatment development Chief of expanded Officer, Thanks by patients. progress last a is interim the on for made vivo to of

where to on stands in are anticipating strong capitalizing confident today, the and start numerous Editas milestones. We the upcoming year

Now, to let discuss our over in me greater turn Albright, Charlie Scientific Chief detail. Officer, pipeline our call the

Charlie Albright

great for of on update Cindy. genomic the pipeline Thanks, here It medicines. time a your I’m Company, be you to to happy is and transformative

of our with medicines. cell engineered start Let’s development

exists. treatment where cost potential with We possible universal cell that shelf to the The CRISPR to In quality, therapies. distinct immune iPS-derived oncology. BlueRock the focus, for in unlock that unparalleled particularly represent a to including effector a mass superior and our medicines can donor-derived and collaboration, for cells tumors of cells, allogeneic macrophages we and work NK cells complementarity, gene medicines medicines T-cell to off our induced retained than stem editing greater edited are T-cell, collaboration CRISPR we the gene-edited wholly of BlueRock’s of therapeutic BlueRock development In greatest the scale, BlueRock efforts lower developed the the the iPSC-derived productive will expertise Celgene truly cellular excited use particular, for unmet collaboration highly the and editing combination we autologous cells builds and oncology technological platform two desire full edited begin in be Further, treat stem to look solid in areas and differentiation in respective medicines, partnership malignancies. required field. brings believe on or other cell in cell with particular, of cell. together allogeneic with need pluripotent leaders Therapeutics with rights tumors produced gene cells. for universal this The complementary collaboration. to engineered existing medicines be using develop technologies NK The efforts solid hematologic partner of and in we and In cancer great forward make have owned field

cell from and our next work to differentiated BCLXXA how American edit our target generation Society we with platform Since data up-regulate hemoglobin. therapies. to which beta-globin a the accelerate are has the medicine December, made directly significant we as we sickle cell that the indirectly of confidence fetal disease to also medicine. sites believe the class and potentially treatment others can of Our believe best hemoglobin In we Celgene develop we we increases presented locus, enhancer at continued time, these based developing We’ve and on build the of cancer utilizing Hematology two and fetal oncology work. that are for demonstrating we look reasons is our superior some the examples have -- BlueRock editing severe have medicines expanding engineered beta-thalassemia. drive CRISPR diseases We an a for cell to progress in towards

We We and the remainder year. medical the have and providing finalized the in through on to started the updates IND-enabling look the forward of conferences medicines. design experimental candidate activities clinical

our Now, to medicines. CRISPR transitioning vivo in

patient open in medicine first in we Our escalation lead CRISPR U.S. midyear efficacy EDIT-XXX are administered EDIT-XXX conducting second LCAXX excellence clinical introduce in endpoint for helps Editas while measures serving ophthalmology ties Cell Europe. Innovation XX Therapy identify program study label the to our liver’s Pierce of to in approximately with be were at natural is us In patients Concurrently, centers to Summit screen to study The dose Massachusetts presented and safety an patients by remain history half. Allergan, last ongoing. poised Dr. Eric week. LCAXX patients Initial the of natural Gene partnership strengthen track of the history. community. history the is the and also the We the the on in Eye evaluate and and dose patients vivo and investigator, of data study Ear and principal to with Retinal

for Following starting with EDIT-XXX, genomic we're advancing additional Usher serious medicines XA diseases, USHXA. or syndrome

particular, affects a for Like USHXA Usher’s photoreceptors. proteins USHXA LCAXX of done been development LCAXX, only in the for medicine promoter In RNA our defers AAV the [ph] gene our USHXA disease to that specific Our same developing enzyme patients we leverages In and vivo the severe the vector, is the promising we’ve represents accelerate [ph] guide builds treatment USHXA the development. a and of to with the platform on work power is that are genetic CasX EDIT-XXX. and have program. and

IND-enabling approach lead auditory the retinal and society that preclinical, model. gene studies this of on XX of track to ready and academic Exon in for the in optimizing In restore human be of researchers this gene we're on of the our data, annual editing Based USHXA the from presented American Eye and this proof Our collaborators week year. function of are CRISPR gene demonstrated work, Ear vivo, candidate concept last a at can Massachusetts cell therapy phenotype mouse a rescue meeting. and

areas look programs our to forward work. leverage therapeutic advanced most in the we are pipeline in and programs within vivo additional expanding efforts this that ocular Our

platform, additional industry-leading aureus access Our and translating genetic to science about exclusive makes treatments including capabilities into enthusiastic Staph to diseases. us target extensive CasX patients our potential for the

the to business our financial call discuss the over let Eric interim turn review Now, and me our to Ek, Financial in Officer, progress results. building Chief our

Eric Ek

Charlie. we’re over past my reporting key today. the update Thanks, we've quarter developments results to the to you and summarize financial It’s pleasure on had

nonexclusive nonexclusive mentioned, and research cross-license formed differentiation intellectual technology in licensed rights of BlueRock BlueRock intellectual their fields, the into a Editas collaboration respective for have party property. Therapeutics. payment Charlie responsible medicine to of In any CRISPR milestones we partner field. while a to and BlueRock’s is iPSC Editas for royalties As and engineered entered gains Each the cell agreement cell and respective respective the to property, medicine with each developed rights and Medicine's nonexclusive technology gains

Turning to the numbers.

press first issued XX-Q. and be hour the results and ago Form our the summarized in that We will available in for the we have financial details full quarter release

cash as of marketable million to equivalents securities XX, March and the first cash, decreased $XXX million from Our quarter $XX as $XXX million XX, XXXX in XXXX. December of

$XX non-cash items increase a cash of and uses our $X totaled expenditures $X income in $X of $X expenses a and statement recorded Our million compensation, million million Key $XX in include of capital capital. million. include depreciation million cash operating working stock-based of and of million

securities exercises option at of of will $X income cash, cash provides and with Cindy. believe of cash of $XXX back least interest XXXX sources to I stock months business. We it by XX, million $X totaled Our capital and million million of our as and to equivalents marketable that fund XX consisted our And hand our $X of of employees. million March

Cindy Collins

Eric. you, Thank

of goals, Medicine. our outstanding a our EMXX in is elements a is progress initiative more QX, But Editas, time shape our Company. we we at revolutionary at about openly for place highlight in talk it best, culture which Editas. the happens about while culture science tackle we of EMXX our elements to short here unsolved As driven of patients. goal In what talk long-range exciting call our and out Inspiritas, future. by community, for an the we Editas part on have serving the editors employees, yet of We'll are the Editas working at marked Inspiritas an medical Inspiritas captures part of rolling and being Inspiritas challenges truly common to daily At where its

verge CRISPR the medicine. first gene of editing We vivo patient are the in with on an treating ever

medicines And clinicians, explore forward in editing to working the With serious thank all support, community, that, the you We forge our cell CRISPR that are your this extend other and of the may with we reach look and technology. gene your for happy intractable to and interest of Operator? of to investors. we deliver sickle tackle promise to partnership new continuing diseases. indications, of further are targets broader new revolutionize platform the to and disease as questions. journey We take patients, advancing cancer, treatment employees expand best-in-class on and we're

Operator

Amanda of comes [Operator Murphy line the with instructions] from question first Blair. William Our

Your line open. now is

Amanda Murphy

afternoon. Good Hi.

actually I few collaboration had So, general. iPSC the on in a BlueRock

if or how guess a why and of bring you them clear programming share, but new of BlueRock, and I’m to the cell I share if building with just you like? sort curious starting table was they terms can can I you in to not So, out what sure much you put it selected line

Charlie Albright

Charlie. is this Amanda, Sure,

really were excited in thought had BlueRock it. research both oncology that a we nonproprietary going off be It’s we're how They interested yesterday they cells in was bring, So, about just and with partnership to because iPSC. incredibly we and really and are to kick an and interested and proprietary immunosuppressive neurology de-differentiate cardiovascular, mechanism. partner into meeting complementary great

are the partnership. powerful think a create iPSC two and we So, of derived really going to gene technologies editing cells

Amanda Murphy

going hem. I then, mentioned more covers the of but part but think for to solid longer a timing that term And the -- any anything about way the think candidates I’m not ask right And that, about also you of as or emerged then, it’s it thinking from that to also pipeline? tumors like you partnership

some So, think with initially or would after we you how hanging about should consider hem fruit cancers evolution? the lower going

Charlie Albright

Yes.

really and indications powerful. all We cell types, oncology have is which rights all to

So, have some we're we going. about obviously ideas where

mentioned table. focused as We on say on I the And that table broadly. well. would all haven’t shared you oncology all are yes, types also things on are them cell more the

it which really broadly because So, us deal, that’s thrilled space part with we're reason we're trying build the the the to of really around ability to the the work gives business. in

Amanda Murphy

not last exclusive. mentioned I one, you just think was it Again,

had So, both thought about iPSCs you from on I just side in CRISPR was and excessively curious with your side? if

Charlie Albright

because it in do parties part we We non-exclusivity our only field with deal. of to parties option which both of thought quite is was decided interest, work a all also the great to additional a at powerful, think and why go

Operator

with Stanley. Our of next the question Matthew from Morgan Harrison comes line

open. now is line Your

Unidentified Analyst

approach Thanks for just think and is Connor to quick Harrison. taking follow-up my you clinically? Meehan hear what after looking [ph] question. And sickle of your cell on sort program guys words. about a differentiates Matt then We're just This for your

Charlie Albright

Sure.

last the the one them of have at at awarded meeting, two differentiation, Hematology best of the bases meeting for length Society for ASH we we in We were work. the American And discussed ASH of December. posters one

normally. reconstitute cell mice. efficacy we’re the which locus, contrast, So, In study of having for Obviously, all in this we erythroid BCLXXA in erythroid Editas significant enhancer in be which that where in -- this hemato translated a editing decrease the the that beta-globin humans, sort if is issue. could lineage showed are -- the cells cells locus, the lineage significant is and a doing, competitors safety edited our

can that's we're we're the items of for then, also Those induce. that basis detail, on into happy to story. complicated be first go fetal can and So, the about somewhat found amount and related our differentiation. website excited And hemoglobin more we slides it’s

We will also the be a say scientific product. more year. this That’s meetings upcoming to efficacy the that in of about obviously driving key having to later

Unidentified Analyst

expected guess, your on the it a possible it. PowerPoints Thank IND-enabling as some Got will just to And comment you defiantly need I I quick websites. Would on steps guys duration on follow you. up quickly comment well? with maybe be to take?

Charlie Albright

timing. guidance or cell we're in like put along way haven’t they discussing on happy the all I And are for way, the as field. to that not We a atypical this it in are sure usual, a those. with the we'll about regulators based think, activities medicine And be make those provided obviously typical positive IND-enabling

Operator

the question next Raymond Steve James. Our from with line of Seedhouse comes

line now open. is Your

Steve Seedhouse

So, EDIT-XXX knowhow leveraged programs, you’ve no indications, I applicable and features is time but much the a much could save developed and are non-retinal that USHXA you of there of tools know can eye subsequent lot of on how other that to how in retinal indications? be

Charlie Albright

in learned be vivo in indication. we going to EDIT-XXX useful what is other of much editing think I with

So, do the with delivery way enables in And these AV general the is agents. work EDIT-XXX that to preferred we’ve think we done deliver that.

So, and learning. you going to that access take to we allows realize, to CasX, obviously make all-in-one us AV Editas, probably we're RNAs. to as exclusive have Staph with aureus That’s an guide which to unique

to be deliver And able so basically, can we’re believe you machinery. editing to AV, anywhere we going

aspects in makes future. need to will -- unmet selection and And of the about so, we to are the that the evaluating in other we sense, about and where more other process the indications that the say have

Steve Seedhouse

Okay, thanks. program. And cell cell then and Charlie, regarding the sickle beta

curious, correct, versus So, single at given what I’m after a presented mentioned, hemoglobin what last you showed. regarding the when and the you is time sort fraction year cells Does you proportion or remembering that it fetal locus, mice. happens locus? think decrease point over the if time time I’m different ASH beta-globin I you just at was over beta-globin BCLXXA in of engraftment, edited that any of edit

Charlie Albright

kind weeks It’s -- I year. think weeks we presented points data from And both on X time was And reached weeks and editing is locus. editing a and can’t last were marked have where XX standard differences beta-globin there state. the X of the things and XX enhancer difference and of time there one BCLXXA weeks I about XX beta- the at either recall if them. weeks between steady

Steve Seedhouse

is it you that or methods data data more these have is other the this some presentation the And for that Okay. hemoglobinopathy comparing program, year experiments?

Charlie Albright

of that out right process the in actually now. We're figuring

Steve Seedhouse

way the basically regain favorable fair is sort that more question CAR-T quick the this a collaboration, on is to allogeneic deal? an a economics BlueRock of on to way platform, characterize One

Charlie Albright

of on now, T-cells our non-T-cells to relationship, not to work Celgene Right T-cells and oncology. We're we’re work in oncology. as engineered on allowed in part allowed

NK our could short in be broader. cells. about that interests that in long we're think we the the interested think and have things so, in obviously a indicated run, to And I way run, And are

Steve Seedhouse

of So, BlueRock develop as able collaboration are CAR-T is you what you saying. not are to part that

Charlie Albright

Celgene as Not is of XXXX. in current expires May intact portion research held relationship that -- as long as and with that we

Steve Seedhouse

Understood.

I at and And EDIT-XXX preclinical LCAXX to back to one the just about model. wanted studies ask last Okay, the mouse going question, thank you.

the you also this publication, Nature Medicine is So, for presented ASGCT example recently. but

drops the editing and are turnover turning going been You decline about and get out it about XX. just above they down then weeks, is time XX% curious, have or week that doesn’t in I over just it to edited, there increase; think odd? eight within to cells XX% anything, of the if over actual I’m back

Charlie Albright

between No, points is of mentioned. transduced. retina the also that. of time editing don't important was for is we that not We the frequency And decrease have you no a corrected the there think evidence it's that

you at last that leveled off essentially and presentation -- the think that dose from there. which maximum productive So XX% if that response really you and fair we the can look curve is gets editing, at then stays get, it

Operator

Our next question & of comes Company. Cowen line from Phil Nadeau with the

open. now is line Your

Phil Nadeau

how the patients possible identify can talk study nature trials? this study Is can you speed the along? LCA, in and use of about dovetails on of your First, with screening you study to it that history efforts study your that in that portion clinical

Charlie Albright

short the is answer. Yes

are study, used that of several of set that scale, its be history similarity -- being physicians enrolling us their many for essentially, or it's sites sites the the make that things they informed site in the natural the is study are not what in it in care are own -- interventional there same history -- study, there same be their their to with primary that us the but they’ll the interventional then can the as and We will familiarity they and will site, the both they with in the study natural helps because used So, will sites up enroll have patients the study with and obviously, study. clearly consultation the decision. decision that in

Phil Nadeau

to there collaboration any question more the collaboration. on visibility on have progress the the is And Bristol-Myers’ maybe helpful. Do into Celgene the of broadly, That’s collaboration desire update that get clinic? a any or then second, you follow-up towards to

Charlie Albright

acquisition as forward. that we BMS I All our same of their of complementary efforts both was oncology. interest which And of closure BMS’ that there with are think have very that collaboration cell-based focus can now things tumors the appreciate Celgene, a large a on of not is Juno. an the of synergizes with interesting focus from strategy Celgene medicines what's is going in larger publicly, solid know a We liquid partner, part as is I the portfolios can two in it is the been stated the only the outside, tumors. think And was the important say very nicely merger with which part particularly the you pharma limited represents on part

that the things for optimistic bring reasons, So, we’re the respect actually those to collaboration. merger with good will

Phil Nadeau

of when could IND-enabling these sense any see we timing, And studies?

Charlie Albright

can't at anything say We this point.

Operator

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

Xiaobin Gao

then, on that just what speculate of sort second And Hey. This regarding is then Xiaobin EDIT-XXX dialing of steps you questions. are natural do our would like for Maybe to question, follow-up or question with age based history the And on the one enrollment actively be the in you more for be acuity? on efficacy? much able criteria we so Thank additional initial would see some editing, you gene the data, would you can Gena. refine about last the is visual kinetics to the patient just precise? taking taking a timeline

Charlie Albright

Let second natural with last history the I the me the start not I’ll question, got hit and I’m and one, one. sure

pathway completed you you that and then like six to allow, in be CEPXXX to detected We editing and the primates, non-human protein re-express visual be sufficient need establish to vision. the the and So, After the segments. other need believe rebuild to weeks. in message, activate CEPXXX mice to that will editing, from approximately was

of with running clinical, answer. will on So, our collaborators, there's in is of you us is which to see actually know and natural both potential believe the part a that study natural a benefit will very all depend see we let few the refine, much at months, potentially to history history the the entirely The potentially consultations Allergan. was will reason patients and part good an that -- question, what study. disclose patient in we could entry in look all different objectives we the group. probably of endpoints criteria results want our we That’s When is a -- that could

I caught it the a to middle yes, so, I’m has that. potential question. alter not And sure

Xiaobin Gao

sort dose -- just and left about Yes, of asking start clinical patients the what the of more site to the activation? first part is to that

Charlie Albright

Right.

activate approval, in we usual approval, the IRB the so drug the the site, all things plan at middle delivering to site, patients to year. product So, a of enroll safety the

of in definition the of virtually on our and middle quarter. third year we're the So, based second

and And will year. patients so, second of we dose then enrolling somewhere half start the the in

Operator

with Our J.P. next question comes Morgan. from the line of Kasimov Cory

Your line now open. is

Unidentified Analyst

on This Matthew is for Cory.

want if hemoglobin approaches bet-in-class, for disease one as can first as referring to the you the program, wondering, compares and basically. cell beta-thal especially that questions confidence level? I mostly follow-up to expand a here non-gene am and on previous your on I So, sickle to editing based little, is on it your

Charlie Albright

Yes.

do And different a in little disclose you're basically -- because they things. where hard at willing so, it’s bit a to this were database comparison that with to point

So, the expressing mean approach that it by gene beta-globin. gene therapy you’re therapy approach the is outcompete in if we have the sickle is endogenous beta-globin has -- what I that

not actually sickle. our case, going things. it's so, In on off we're doing is And to two turning actually fetal We're we're turning the and hemoglobin that go. how competition clear

So, and human potential of to based part is of doing challenging. comparison -- the doing has genetics. Suffice be well believe, the do to as the say that it best-in-class, on what that combination that quite we’re actually is we as pharmacologic

with recall you actually suppresses As fetal the fetal hemoglobin, symptoms. [ph] available sickle-cell of elevated regionally patients levels hemoglobin the

rescue the disease. And has so, nature us this is taught actually can mechanism the that

Unidentified Analyst

then, And your demonstrate this just strategy I based clinically? it. said, just broad on what level like you Got guess, what's to

Charlie Albright

go We're and hemoglobin that. as patients, fetal you’ll levels from come going symptoms to into see, and

Unidentified Analyst

about search? then, just an you follow-up. CEO And one Can provide update the

Cindy Collins

Sure.

forward into to the to through next the us phase of lead Company excellent the looking clinic. help as move growth Our we’re searches hiring underway well and are really executes we

timing; right. active those comment is quite when update searches the on no are you timing we So, will at this But, point.

Operator

Lawson line with Our SunTrust next question Robinson from Peter comes of the Humphrey.

now Your open. line is

Peter Lawson

the identify think screen be takes around then, any dose to patients? the to of how the if and And about part on patients history should consented Just we the the study? period of interventional it natural LCAXX, long

Charlie Albright

that. anybody we We we in interventional aid in anybody the is can't and history asking the LCAXX comment can't that whether -- on basically enrollment, you’re study natural Clearly, the on comment whether believe enrolled. hope Yes, study. will

interval phase dosing patient dosing the like one any with we say, I provided subsequent of first gene for As patients. now. the there and And between an are study, just therapy be therapy the will typical development gene these would right space details haven’t that. intervals

Peter Lawson

Thank you. number you. the based have and study add you of the on And from many Got would then, many sites, natural what's the and since around how does how others this over history roll that? you timing but

Charlie Albright

Yes.

We U.S my Europe have sites in and four sites in is three recollection. the

all You are my memory an right IND have we numbers. now. with guys testing approved the But

do and feel be So, to Europe in the short Europe we get believe the on sites, long the will clinical us that in the the trial first from but U.S be open well because run opening we we better European sites run. need the we we're started on will and will those patients sufficient and work in

Peter Lawson

cell then, do think start could trial? sickle you when And the you

Charlie Albright

provided will yet I Peter. you guidance the but We call will haven’t first you that be

Operator

with Our instructions] comes Securities. Guggenheim Denise of line question the Meacham [Operator from next [ph]

Your line open. is now

Whitney Ijem

well Whitney. for is questions, just This actually the Thanks taking one.

ongoing you there. meditational USHXA, is optimization mentioned on So, there that

missed any I color the if give But -- optimization So, us can on wondering looking sorry wondering through. that that you if you're this.

Charlie Albright

development us IND end to which as call we're position the the at put pharma the up of finishing we're of for studies there, enabling in a the nomination IND will package unusual year. on be supports a ready nothing that candidate for o is the there track that. Actually

Operator

back remarks. the at turn in would to I’m I time. showing no questions further closing Collins, this Cindy for like call queue

Cindy Collins

Great.

all in patients. today’s we participating to So, and medicines great as work new for call you with that, support we for Have a your evening. thank transformative bring to

Operator

today's Thank for and concludes once participation. your presentation. you again gentlemen, Ladies this

disconnect. may You now

Editas Medicine (EDIT) 7 May 192019 Q1 Earnings call transcript