EDIT - Earnings call transcript Q2 2019

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Operator

Good afternoon, and welcome to Editas Medicine’s Second 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. ahead. go Please

Mark Mullikin

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

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

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

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

Cindy Collins

joining you for XXXX thank Thanks, second afternoon, update. for us and Mark. everyone, Good corporate our quarter

Ek, Chief our President today Chief Eric our Officer; Officer, Corporate executive Senior including our team, Mark, Officer; Scientific Chief Hunt, In Affairs. our of Tim Financial joined our Charlie Myer, I’m by Vice several interim addition of members and to Vic Albright, Technology

achievement governance the nominating by board's afternoon, EMXX the our leader Board with executive and and process search firms. scale ran next to right expertise, committee focus focused the patients. on for leading was As executives to industry, and identifying on with over long-term strong and run on the ability Overall, a driving candidates the development search many we chapter, grow goals. comprehensive and focused Editas Editas and accelerate take The proven past and companies interviewed its our scientific assistance several passion for months into announced a from this success board was a

Permanent and have that is as learned is to as As patients at as build which my why a That has on about appointment Editas lives in the and to why capabilities imagined. to the the clear Editas industry, accept believe had editing leader CEO. transform veteran in and company the gene this and to are the Interim opportunity the well great momentum humbled I the that the grown We've in more and in XXXX Board's of I’m space. of I can company. establish join medicine knew has and programs dramatically pleased we gene as I enthusiasm I people, dug an jumped determined Editas last extraordinary durable ways medicines chance never December. that develop CEO, Board cell And

deals, programs, We development culture. executing driving have hiring Inspiritas teams been our building strengthening business and and

Charlie I then hand am what discuss further latest and I over in highlights on and has detail. our proud and the to team will update of you am excited to Eric few on our I accomplished a progress. it of to hit

communicated. LCAXX Amaurosis Allergan, XX screening for have or Leber Phase First as initiated and with Congenital in track in X we second with to dose our half on in we’re trial clinical patients partnership X, the EDIT-XXX patient previously

our second well as on beyond of vivo with EDIT-XXX moment first medicine treating the and making Editas IND disease broader CRISPR ready work time is medicine we the year. by Usher as in ocular thrilling XA. Syndrome the by on medicine history our of a with This enabling ever de-risk of progress Medicine, on is studies of program be in expect the pipeline accelerated for cusp to This we’re end as people are vivo in the first starting EDIT-XXX another CRISPR strong and for for we

and cell cancer to medicines, tangible build we universal our continues medicine continue is allogeneic Third, including making our of cell sickle advance confidence in as cell to headway best-in-class its cell for development a and medicines engineered EDIT-XXX our for team potential engineered thalassemia medicines. disease on beta

in required our Colorado. site develop to commercialize at including capacity investing infrastructure we’re manufacturing Finally, manufacturing Boulder, in and CRISPR medicines

the Scientific the on hand Albright to will Chief update I to call Officer, pipeline. further Charlie our over Now you

Charlie Albright

Thanks our first on for with beginning CRISPR today. our I’ll us -- thanks start our everyone in call to the in lead joining vivo I'll medicine's EDIT-XXX with Cindy. And LCAXX. CRISPR with start candidate vivo for

of track half for to the As X in We begin Phase dosing collaboration screening called year. Cindy mentioned, second in our X, patient we've Allergan. with initiated this interventional Brilliance on remain study

an Mass. currently and is sites safety, is we XX escalation to weeks open Brilliance and tolerability Eye in Ear dose patients. recruiting more three in the efficacy label ahead. and open to EDIT-XXX approximately evaluate study the of plan

also We patients from in similar study. criteria Interventional very the to expect will first our enroll History trial with natural study the patients the come enrollment which

study history was recall objectives the of one study. of natural that accelerating Interventional will You the the

retinal protein experimental second XA. USHXA a truncated a causing in a a The affects deletion the XX that is photo our nucleotide mutation for for developing USHXA Usherin, Exon USHXA in by caused shift results transduction the protein. in inherited is program, frame in retina. mutation an Usher photoreceptors, non-functional LCAXX, gene the we Like vivo are single with disease essential the medicine most encoding common gene in patients that is in syndrome For

therapeutic editing mutation remove and functional Our containing approach protein. gene the create uses to the thereby CRISPR a Exon

EDIT-XXX Conference collaborators and at lead studies Therapy is enabling proof year. This IND Cell we proprietary many concept promoter of the a and Ear preclinical studies preclinical recent the our USHXA this molecule. Gene including having currently Eye by serotype, AV the Mass. of aureus for and of medicine Society will features completing of We are from Our ready and the end for CasX. American Staph showed patients anticipate molecule for for share approach that

Finally programs on platform we're in rate about coming These these the in working the to our further and editing programs speak medicines. we'll of months. programs additional will illustrate power vivo

cell medicines. Transitioning to engineered

thalassemia. best-in-class for editing expression uses The be second developing to pillar to program and medicine treat gene we’re induce This to fetal of our cell hemoglobinapathy. EDIT-XXX therapeutic strategy sickle beta disease a hemoglobin

other erythroid indirectly enhancer the after go therapeutic fetal up which hemoglobin. BCLXXA which This a our hematopoietic locus targeting cells. approach the regulates stem beta-globin only and As therapy reminder, differentiated involves from is approaches

red enhancer blood findings impact differentiation. Our in suggest number of Ultimately cell adding may the viability vivo. and cells stem BCLXXA the edited

We adding the EDIT-XXX we the robust are later enabling the fetal hemoglobin. on this the present inducing year. benefits that IND data had beta-globin have hand no detrimental underway, plan other while Medical found Conference output effect activities erythroid demonstrating of additional in locus to and on features

guide confidence well ribonucleic programs. and use oncology our as medicine to Colorado. Due this in clinical we program, for and as building this to at our EDIT-XXX studies supply preclinical facility are manufacturing intend as Boulder, site with engineered RNA Particularly in other protein cell capacity

be next this year. site commissioned We to expect

oncology. on Beyond investing Hemoglobinopathies, are develop medicines broadly our work and we cell to engineered

need. pluripotent wholly medicines induced medicines as develop in and partnership cell Last efforts a the long engineered ability We potential for parallel will engineered Celgene medicines genome highly oncology standing essential with cell from of collaboration iPSCs cell be advancing including to these features create iPSCs. or to advantages T-cell high cell develop NK truly may announced medicine. make therapies to shelf potential BlueRock a for using the of total quarter, unlock and to off engineered have believe owned area Therapeutics are cancer. an stem the solid control with we potential we Engineered medicines tumors, We offer develop with for cells several of a thereby to a the cells unmet

just making, rapid the by past few we've progress made months. in the Encouraged we're

from successfully the blood Firstly peripheral iPSCs generated and cells. fibroblasts

functional to NK have our differentiated that tumors. unmet the and potent cells progress us highly iPSCs upcoming efficient in good in off-the-shelf to we the develop to high CRISPR This medicines vivo scientific with achieved and we've iPSCs medicines continues activity. editing puts Second address a conclusion, on with solid of path medicines. against have for engineered NK third results say team at meetings. these cell In engineered more about I need make both patients strong headway killing

financial and me provide to business call Eric update. a turn Now let the over to

Eric Ek

and of be Thanks Form press the in that These hour Charlie. available all company's we about an XX-Q. will numbers our are pleased full results. issued financial to details you on update I'm summarized release also in ago our

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

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

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

Cindy Collins

you, Eric. Thank

Office organizational patents to a intellectual and exclusively early make development. licensed to issued patent and the on June, certain Group Patent the which Broad interference California submitted like I'd Institute declared of a close, the University second Medicine. we by Before few certain between comments Editas to applications are U.S. In property

expect be and our intellectual have this We property upheld in these in and to proceeding. utmost confidence issued the patents confirmed

on updating CRISPR CFO an in treatment remain side, we look at of On active patients In Officer. with searches new the first executive ever and closing, with forward time. are vivo an the excellent making Chief We history to Medical bring appropriate to you medicine. organizational

to of is bring capability CRISPR across our interest to by wide and gene are editing thank we and to reliably of capacity into pipeline from are enhancing advancing happy diseases translate to With support Our a hemoglobinopathies building serious CRISPR inherited for manufacturing end-to-end further medicines cancers to your your to patients medicines. And we blindness. range questions. genomic take that, supply medicines Operator? all you to

Operator

first Wang your Gena line Barclays. of Our line question Instructions] the [Operator from you. comes from Thank is open. Gena

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

Unidentified Analyst

two have I Matthew. for questions. on Costus is this Hello,

is will one first how data open you the Will or studies data you the announce label Brilliance is present this from given the study before first few report to you from until that patients data? you expect wait an study. The do completed

Charlie Albright

Charlie. Hi is this

we're really get the to excited So study number one going.

is the we so and in report the will editing will be much very we’ve circumstances As to vivo CRISPR on data in and data the time. said able the how see we this first real depend

the how Allergan area. make of about We careful call or in in our T-collaborators conclusions ultimate accuracy collaboration will will want with but we to be that this

Unidentified Analyst

Okay. Thank you filing you. And IND EDIT-XXX? need you an what one detail complete more do can to for

Charlie Albright

with stuff IND-enabling we usual to the the need and all It’s scale-up so Tox manufacturing studies.

Unidentified Analyst

you will do approximately And know this take? long how

Charlie Albright

disclosed an upcoming No data interesting. not tuned we you'll see scientific timelines find think meeting EDIT-XXX on at have you'll yet. quite more program I for the Stay which

Unidentified Analyst

Okay, thank you.

Operator

comes question JPMorgan. next Cory the from from Our line of Kasimov

Unidentified Analyst

pending the Cory. Gavin this for a then Usher are for extend that congrats on see studies? on headwinds you. the had do And and new guys appointment. What with you just on then deal just factors strategy, the The potential we Thank syndrome. partner Hi the disclosure question on I remain a follow-up IND-enabling the the gating from the first for follow-on EDIT-XXX. any is to maybe Cynthia

Charlie Albright

quite about you headwinds. talking about we collaboration question I understand mean in we merger? the the and them. with a have AVI first disclose partner Are didn't I will

Unidentified Analyst

the side on Yes, disclosure correct. Allergan It’s or? the

Charlie Albright

Allergan Allergan factor until is not is decision is, and making. that And any it our does is right so in not now Right closed. deal AVI partner now obviously a

of very aim IND-enabling that end all for usual by ophthalmology Syndrome with the to the we've programs and is year. business the XA ready the deliver turn and partnered so are it's Allergan So be we head much of and down got our activities Usher on portfolio.

pharmacology preclinical to position found a completing studies studies to us we into on in some now studies. IND-enabling have and that proceed the get So package we going of variety a other need the have

Unidentified Analyst

Great, thanks clarity. for the

Operator

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

Unidentified Analyst

to question. Vanica Steve patent would on hi. This like Yes, we Seedhouse. is on interference for And follow-up

accurate is wanted So regarding what estimate legal of acquisitions finally the involved making what of made ideal view if your an side to what an table be by the your being resolution also is to the negotiate? Berkeley you. come and And and costs the willfully Thank statements. other would

Cindy Collins

apologize of cost not about I last And litigation your make the to we're the any what acquisitions all, on the So of question, first speculate going might overall of was… be comment inventors. nor

Unidentified Analyst

would the if table other would come side wanted what and the to be an situation? negotiate, an resolution What ideal ideal be to

Cindy Collins

an as it and would us with CpfX. property that think I prevail continue we situation feel very we position ideal we intellectual around particularly which the So have for relates be that to CasX confident to

Unidentified Analyst

Okay, study. have we thank on also you. And follow-up Brilliance a then the

I design. in I've case that's will escalate the but the but I about higher I you to think preliminary two low in still dose be dose. plan then past going talked know the to and patients for the you've that's first don't know you if dose still if seen and they're cohort. don't

the second than patient two first you patients if question going or any you. So you and have be disclose? the also can one, to changes the is it younger is is Thank mean made is I still

Charlie Albright

Sure.

safety objectives are their two and and so is of third concern, And paramount tolerability objective main obviously of safety the is so efficacy.

right the much our the to benefit favor do in to is obviously the risk thing put trial. we So start very as

range adults patients can that pharmacologically the dose be then we we those of the poor a at on So will low dose, two we escalate the active. start vision will in dose dose escalate. eventually that on the we will the the will and very be be that But activity from of and will end have think there two results pharmacologic patients, low based and trial

vision children adults the not have eventually results to vision children this benefit on trial therapy. place have clear expect and standpoint. because start a and who will that risk those from will vision ideally We obviously benefit move with right into better better move would priority most to a then some into have this vision based But from that's the

Unidentified Analyst

you Great, very thank much.

Operator

of comes Our next Amanda BTIG. the line from from Murphy question

Amanda Murphy

question you've I for congrats just and Cindy now I you you Cindy. a guess there Thank have been since several months. for

you already had a entity things perception? or look about resource from strategy just tweaks some forward behind now any you've making year, now entity. I like year into if somewhat some et have in obviously company thinking a mean changing that clinical contacts and about preclinical into hiring guess have curious the is thinking cetera, but there's I you've going you're you what almost curious Just EMXX you your broader that going whether and a that your or is

Cindy Collins

months six maybe about like been year. it It's Amanda. feels Thanks but a

little the moving very to and current remain is strategy that XXXX our and to committed a which the you’re to now different our us. we continue goals So EMXX clinical corporate correctly fact into We'll is phase referring company to along required about for we'll we talked continue growth our Boulder term. and the that we that of efforts facility near need we in to for as resources in just manufacturing hire a bit that support the trajectory scale, our some

Amanda Murphy

on one then side got the NK And fully world. it. the of just Okay, owned

a of solid with strategy cells and to but you considering going is would starting your the more just A ask maybe focus targets I that curious tumor level, might some ones more competitively. that really an was solid validated of I well approach perspective? or there's more little would in that I you high you indication thinking know not go, from kind crowded how you on there maybe NK too early in So and forward out about NK tumor drug, guess much are view iPSC it's

Charlie Albright

is Sure, Amanda. this Charlie,

of where going. So we're we haven't disclosed obviously the details

that from medicines. I pursue develop think both and area have assured differentiated treated we approach you thoughtful and other the areas transformative can indications, a how will to to disease draw be some parallels we in

Amanda Murphy

broadly on as opportunities. quite side, CRISPR in is there factor you it restriction is is landscape to Okay. like have almost freedom that one or your we anything just complicated have about like iPSC think seems from standpoint operate a anything there you there but as think iP And the as I last feel Do about? to there to

Charlie Albright

as enthusiastic and there our feel those good are about advancing about We position gathered very very medicines. hopefully

Amanda Murphy

very thanks Okay, much.

Operator

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

Peter Lawson

should on about question and or an think peers just can use the beta first example timing want just beta a produced to from for clinic for we And for we how sickle I the my Cynthia, sickle Hey Charlie, cell Maybe guess as the thal offer congratulations I timing first. there? role. entering

Charlie Albright

this Thanks the I Peter. in fair are numerous that’s guidepost. totally Yes, good. Yes, precedence existing field that trials and think it's as use the a to

this Europe we strategic We what either haven't both decisions to medicine about disclosed U.S. cell later our it versus really in lot to look really sickle strategy versus as more beta advance talking feel we exciting and is year. forward a a thal key on

Peter Lawson

And the think difficult appropriately we think initial therapeutic the then you of just you you treat. wait so long patients for the initially to how dose those most do a until Do just see have to have we considering on patients, LCAXX pace would kind that?

Charlie Albright

months. benefit because entirely that see pharmacology believe pharmacology the are a preclinical well. we rationale and and you'll it's that on based number Well the small And preclinical based gone through scientific in for that the we've of as on doses

non-human studies editing. did relationship which a and we and in dose So primates CasX and give very levels mice between similar

you at on the selection as tolerability. we're efficacy want we have understand we the same can but we good therapeutic do to obviously why to and point the we've what's understand about have understand because the And trials be at chosen benefit going to dose and and time, believe that's so first the at to that's we there basis do starting conservative feel trying dose safety feel as how for one been potential that this to dose

Peter Lawson

okay. Great, Thanks questions. Congratulations. the taking for

Operator

Kenneth Company. of line Atkins question Cowen next from from the Our comes and

Kenneth Atkins

dose my and patient so in EDIT-XXX how trial, second on? on you dose pacing of after the patient dosing taking the Can thanks comment you the soon first patient can Hi, for questions.

Charlie Albright

Yes, this is again. Charlie

haven't it's the we typical design a So on based therapy. disclosed very details, gene

correct will that the with And safety patients and patients an of so and the drug. there be priorities the the fits you're tolerability that between additional we actually expose to to ensure before all interval study

Kenneth Atkins

aside improvement depending that do level of their patient's meaningful And on And the visual it, you tolerability, from okay. Got for acuity safety disease age then severity? navigation think differ or what and patients. would be visual would and on function

Charlie Albright

major general. reasonable with patients deliver can medicine. think to the SPA provides meaningful a benefit I we clearly the And -- a our that think understand what's in considered of and I benefit clinically the that precedent in trials objective

Kenneth Atkins

Got it, That's thanks. helpful, okay.

Operator

Our of next from question comes from the Securities. Ijem line Whitney Guggenheim

Unidentified Analyst

Anita for Hi my this Thanks taking on is first. Whitney question Ijem. for

So progress as or about the you think DMD muscle you look other utility expanding towards technology talk how made delivery targeting vivo side. programs? any in can lung in on the CRISPR about and CF Thanks. also ongoing And and work do you

Charlie Albright

is this Thanks, Charlie.

We the delivering very big tissues. move And excited that benefits is are we complete of was to suitable space. get EDIT-XXX to of into package one experience in regulators out the other in a this

places And capability the move us to course been take does our we editing you delivery access CasX, great proven into advantage a want only go realize to to we're the where to AAV other ability to so go. the the deliver tissues more to so we was this as eye we're look single other other place AAV the start forward and do a of gene Staph to but that talking and company has tissues. going of up think because aureus We and that set actually in into be vivo about of do unique vehicle good a has to that

Operator

Oppenheimer. Tuerkcan Our of the question Silvan Instructions] line next comes [Operator from from

Silvan Tuerkcan

clinical and pediatrics, new the trial -- in a Thank the -- low and adults and got Cynthia. My middle be dose question on you low role, would high dose in that children? looked there's Brilliance middle dose high and same that in for as you is so adults middle for dose my the congratulations trials on trial the question taking I at could and

Charlie Albright

so that different totally turns Yes. and grow dose out all you as not it appropriate. same the so eye And the is and that is

Silvan Tuerkcan

but history us at could be viewed mean of And spontaneously could that have quantify study please the you as you the patients remind the a that kind And for in clinical great. get LCAXX. end? the of benefit better endpoints the I will to of kind control natural Okay, these

Charlie Albright

a of at all Yes, endpoints so we’re usual history natural looking potential in the variety the suspects. study,

of would of we right test acuity endpoints visual substantial imaging we as aim a ability that evidence which as actually stringent be that these the had of more functional corrected could that’s patients. which mobility a provide So functional well to benefit achieve maze

with at this And point would that history familiarity was endpoints get in So understand most looking we patient and study which Interventional study. the of for these functionally natural be those be part whole operationally population. will of and of relevant to endpoints the several

Silvan Tuerkcan

be last we question. efficacy get any so Brilliance the will you or that you my before data do trial think that from after? And Could disclose will data

Charlie Albright

some We've that meeting. data of disclosed at year’s this ARBO past

And be that available our disclosed data that so we on should website. baseline in

Silvan Tuerkcan

you thank much. Great, so

Operator

to We the over Ms. have at back will Collins. no call time. further questions Cindy now I this turn

Cindy Collins

Great.

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

Operator

you thank today’s for gentlemen, call. and us. This conference joining concludes Ladies

now disconnect. may You

Editas Medicine (EDIT) 6 Aug 192019 Q2 Earnings call transcript