EDIT - Earnings call transcript Q3 2020

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Operator

Good afternoon and welcome to Editas Medicine Third Quarter 2020 Conference Call. All participants are now in a 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 third Good welcome conference XXXX afternoon to Earlier release of a results issued this we afternoon, press updates the and for providing third quarter XXXX. corporate financial our call will website our hours A replay two of be on section approximately the completion. its available after of Investors today’s open After will for the prepared our we Q&A. call remarks,

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

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

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

Cindy Collins

you Mark. Good for afternoon, XXXX. for Thank call you, for and everyone corporate of us update thank our joining the quarter third

Financial Charles by; Scientific In Officer. Michelle joined our Mark, Albright, our Chief addition Chief am I to Officer; and Robertson,

CRISPR Phase our our our advancing quarter us the work programs and through to advance new the of treat has resumed pipeline. CRISPR best-in-class while clinical We with medicines third strategically flexibility X/X cell an Starting regaining medicine agreement the programs, our our vivo brilliant as with operating in our in full with of vivo the an first sickle and to engineered control during progress ex of the trial, dosing valuable vivo trial AbbVie, foundation and we medicine, cancer in provides ocular disease towards cell Our laid clinic. important for vivo advancement pipeline.

the CRO LCAXX the have to plan BRILLIANCE finish fourth the for quarter. We and for in we Editas, EDIT-XXX to trial activities and contracts transferring IND back the CMC X/X transferred Phase of

report pleased first being BRILLIANCE that those of enrollment to have the remains are and cohort we completed in We active.

are we dosing pandemic, and patients the due experiencing has the United delays patients the However, logistics particularly residing to challenging, which screening ongoing outside more States. made of COVID-XX

accelerate potential blind enrollment We holds to landmark the make in trial are to making again. see every that effort this people

vivo in track ex filing We closer for quarter one a for on bringing step cell proud and to IND disease. CRISPR medicines. sickle Turning sell remain medicine to be to patients towards with best-in-class an EDIT-XXX our the potential fourth are

manufacturing at a meeting in scalable will We in well as platform data EDIT-XXX the new present building Society our December. American Hematology for of robust, the upcoming program progress as or ASH

cell allogeneic front, regulatory that U.S. and sickle disease. a received we medicine the EDIT-XXX, announce In we Disease for were is to FDA that NK highlights On EDIT-XXX. designation significant pleased life-threatening tumors. nature from advancing the Rare we milestone serious cell are treat This Designation the oncology, solid Pediatric an of to

or NK Society for increased data present upcoming of of the SITC will show as preclinical We Meeting. at ASH, cells, data effector to compared which Annual show function the EDIT-XXX will also non-edited Immunotherapy at Cancer and

our Charlie like call quarter. highlights updates corporate for I more across some pipeline, touch to the from handing to over detailed Before the would upon

EDIT-XXX, and EDIT-XXX. a and with These Catalent provide EDIT-XXX Group flexibility pipeline of clinical support we our and Our us with capacity manufacturing advance including and agreements as clinic. programs preclinical into Azzur agreements the the development

in place importance to the are development and particularly the our the clinic-ready of of support support of given to We program to programs manufacturing delivery proud strong manufacturing a cell-based having have AAV established therapies. scalable,

On granting Broad for regarding Broad Trademark Medicine. decision Office, the the recent exclusively licensed ongoing with Patent pleased interference CRISPR/CasX and motion patents Editas we proceedings front, priority Institute certain were benefit by the U.S. property intellectual in the

result the of priority Senior as first the invent Party decision, into phase Junior a was eukaryotic remains gene entity for of the Broad for editing the this of of enters cells. interference purposes which As determining, the opposition while the and to Party use the CRISPR/CasX

and remain resolution in be to at Broad We Party that requests now outcome filed by reminder, preparation observations overview, motions carries expected to extension proof and the pipeline to discuss arguments. to parties that proceedings. turn of the call the round Institute another of the proceedings Junior both Editas forward for over next burden look oral six our a With Albright, As Charlie Medicine. and of months positive ongoing in confident remain The the by to be Chief would the with the over the a our will proceedings Scientific I these for Officer, updates. like

Charles Albright

with the for and Cindy, you of first cohort the trial to in programs with dosing we EDIT-XXX completion Thanks, vivo joining of make call today. ocular continue LCAXX. and the our BRILLIANCE progress medicines, pleased are Starting editing our thank and for to the report

to the gears trial Cindy LCAXX. advance to momentum. medicine to enrollments remained this pandemic medicines As the gain strategic Switching at continues and transformative experienced and sell COVID-XX we've to engineers mentioned, bring to Editas slower that important with potentially pillar to patients due eager

with the EDIT-XXX principal medicine developed progress lead organization durable make as file cell IND potential disease. to hold the support the planning the autologous on a our research to and identified We trial trial medicine before the and investigator best-in-class year end. EDIT-XXX, meeting we've Further, investigator to sickle being the cell to remain track for for continue by engaged contract We of an end. year

off patient be was Meeting. ASH BCXXA these CasXXa. hemoglobin studies, We CasXXa causes this by into at will target and for donors As can forward locus disease region. XX% the on that is that greater cell than achieved at than increased and fetal a reminder, mutations at on not to we we Annual at to successful data. healthy presenting editing genetic cell mutation leverages increases fetal in additional specific better show will fetal detail presentation hemoglobin outcomes. site more human cells will the high EDIT-XXX cells In to the genetics have our editing of our editing BCXXA directly measurable region cells this translate cell disease cell patient data symptoms of editing the led from found. reduced no human consequences is is proprietary do the sickle people sickle were from fetal believe CDXX of proprietary EDIT our supported as editing levels CDXX the sickle hemoglobin of hemoglobin. upcoming fetal the with show with patients The disease. EDIT-XXX there sickle of Red negative hemoglobin efficient by that supported as blood cell sickle in hemoglobin look from editing sickle We not targets. the induction. Furthermore, cells editing due approach by that increased beta-globin editing derived some Anything robust contrast, Further patient CDXX In site of healthy this enhancer donors to are

know donor than support sickle cells Finally, in from long-term These scale. in we that more disease cells functionally are this than manufacture found of ability edited healthy experiment graph patients more progenitors at cell the editing data CDXX will we and show system. we of in as a closed together with XX% mice. to editing XX% We our semi-automated EDIT-XXX

potential with advancement support data EDIT-XXX, the these patients. the cell sickle of medicine of together, to a the lives Taken transform

progress of medicine. Moving next to make our EDIT-XXX NK to continue donor our oncology derived healthy edited programs, we cell

TGF forward immune a the and data potent shown the CISG IL-XX the microenvironment. presenting pathway beta a of knockout CISH presence with and beta. for pathway, reminder, regulator cells. We from and ASH, cell function knockouts known particularly TGF of of inhibitor in is cells, our CISH At CasXXa proprietary As look the a a upcoming uses major ASH EDIT-XXX in beta meetings. negative NK NK CISH SITC present the data than TGF we genes. well TGF and cells tumor is to survival will more in unedited to the

We therapeutic EDIT-XXX to other provide medicine the combination of valuable continued a potential data support patients. benefits to the also EDIT-XXX advancement killing. of cell tumor enhances the antibodies These with further of show that cancer

iPSC-derived cell We're first specifically proud INK cells as spheroid I'll EDIT-XXX more will into the INK medicines. the on of vivo look over with We track Financial address our Further, the data versus the in we've Overall, killing These cell We're avoid will our immunotherapy and by syndrome. is that, progress progress our At considerable T CISH engineered our in challenges the periodic forward dosing a medicine. the clones, indications. controlled NK Michelle INK the Chief shelve and to across and pipeline were cytokine updating of to graft continue effective disease, turn our host enhances TGF ASH meeting, model. INK year, we which we two release also cells show we beta INK for We're continue range program. and off tumor that progress INK with of CRISPR were on progress. in ever potential our our expect we and these call to With this to pillars. cells oncology EDIT-XXX continue program differentiated double of made knockout updates cells excited future cell to therapy to Officer, a Robertson. upcoming that knockout R&D our an and than CasXXa broad associated such provide progress will support file on our potency,

Michelle Robertson

and CRISPR we Charlie, remain advance as everyone. portfolio a in our position of strong you, Editas experimental Thank good financial medicines. afternoon

and to from balance offering and fund our Our ongoing same quarter and $XXX June additional and last the to ex marketable year. proceeds was XXXX. certain The we to million quarter Allergan cash Allergan compared to Company collaboration, for million development will in million $XX period increase our continue the a our strengthened in by candidates our funding both million other attributed balance related equity expect remaining primarily the related was the sheets, XXXX preclinical expenses our $XX expenses same The equivalents result $XX.X with an of as sheet, our for fees connection our and vivo compared previous The have million clinical also year. third XXth the enabling to period requiring the EDIT-XXX the our for quarter into to of development were the last for quarter. XXXX $X.X and revenue of guests. trial $X clinical across September Research the strengthened million and recognizing execution vivo and deferred cash compared as to we our manufacturing revenue operating for of of advancement R&D pipeline, were Revenue our expenses the increase in $XXX to balance of we've into agreements. well including of the third in expense offset positioned increase programs the million BRILLIANCE from XX raised balance LCAXX of the of one-time paid plan The a XXXX. in license securities last termination

do increase In related in expenses. any for Company last fees an increase for compared expenses administrative an expenses of as invest to professional operating net the attributed third clinical the in expenses General employee connection facility G&A Company as the $XX increase to non-cash based primarily quarter $XX.X The and the stock and Total organizations, period $XX year. were in XXXX an Allergan employee to of the million of XXXX incurred same addition, with to expect as agreements $X.X time, impact expense At is were material compared COVID-XX. resulted financial we year. increase for in the this and service the expenses million CMC compensation related prior negative quarter from expense which the in in facility continued well million. million third of by of termination in not

now the future. situation We to to that, the Cindy. an I'll monitor in turn With over the will provide call back and update continue

Cindy Collins

incredibly I'm which progress, Michelle. partners. of proud you, team, dedication and of our Thank strengthen was achieved through our leadership the employees valued

strategic company to sickle the cancer, the that administered two by encouraged first our blindness, as momentum genetic aim CRISPR potential vivo conditions medicine. have medicines of advanced are to and neurological provide an as disease CRISPR have our and only we differentiated the and revolutionize cell have We treatment in pillars to

of marks which genomic a cell We CasXXa medicines, a in data carved place leverage have presentation our engineered for of The as scalable development leader and strategy positioned medicines differentiated Editas, the the to leaves our oncology us combined phase editing medicines for our field. to advance clinic. new with at strong manufacturing particularly These program. these well data a our for foundation

we more continued Operator? We you and we all to will open look about through are the thank that, excited We and advancing Q&A. progress forward clinic. and interest our medicines for the support. of into your call for With

Operator

[Operator from Kasimov Instructions] Morgan. Cory Our first question JP with comes

Unidentified Analyst

This is a Cory. [Turner] for

a I therapy high clearly but these has So in with little it of bit the think touched was FDA gene on prepared days, the recent news. remarks, standards particularly the

So plans as approach just in a And especially then the change any perhaps all, year. your next potential the on do manufacturing change bring way And with in-house? term lastly, use see to competitors? you mini you if longer EDIT-XXX to this in what session at have clinic you with is ground

Cindy Collins

is cleaner any in-house EDIT-XXX have we Azzur don't point this we at plans done for that bring at manufacturing that to wholly being least the all, space the of in time have space in-house. through and First

options we believed will have recent forward. always that program submission, Bluebird's were on the making announcements a during ground we We move on working that were evaluate and the PLA our while delay we we with we our on period gained COVID IND. do And most ground we as have the believe

very program. that feel position the we in competitive XXX So, a we're with

Operator

from comes next Gena Barclays. Our question with Wang

line Your open. now is

Unidentified Analyst

on Gena. [indiscernible] is This for

may, So I firstly about questions, have if two EDIT-XXX. I

And expect we cohort other about you or is either data when dose additional some from on one EDIT-XXX. initial patients? So low-dose after presentations on some the

your in the identified to that step types can what's particular be be and presentation, to next susceptible called preclinical ASH tumor the more development after clinical So therapy? any MTA

Cindy Collins

issue. then I ask the second first will take question I'll the and to Charlie address

disclose determined point We will at in when data this have not time. we

will now new to who to of releasing We about take a want CMO it for have But think consult the discuss first forward. be the largely with the wants the cohort how you've be the move dependent as Charlie, forward. joining have dosing on to plan heard, But going new we're you of data to eager she achieved CMO's who will that will view data. releasing question? second us we and do

Charles Albright

for development question was second XXX. strategy The about

XXX. at And ready time. will And so, do these we do a moment have for more we're in there not for to we development disclose medicines but yes, types tumor be strategy them this are think effective,

Operator

with Phil Our Nadeau next question comes from Cowen.

Your line is now open.

Phil Nadeau

cohort LCA light the could experiencing we the most First when to enrolled, some is of I'm is COVID. on first but from delays recent you're fact see thoughts program. your there? the But In for on data curious to

Cindy Collins

to And we that data to wholesome sure this want and at a responsible be eager largely be a make As And joining what the that what set about patient point of it set data. I released looks new will is And data with depend mentioned do the in consultation basis. we'll data who by us. like. CMO will on we release it not the patient we're on is reviewed and

Phil Nadeau

significance going And [indiscernible]. senior in is the Can the on as party? us of second tell you the

Cindy Collins

generally proceedings. party party burden proof holds has I mentioned. position, for of the junior senior the the as So, the stronger The

have in second. And to of behalf forth data. will or terms so, the and first Broad putting we their they their go depositions on -- And will our go

time the we majority overall, in suggests that data position us the So, strongest the I of the think that prevails. party believe the puts this senior

remain from to the our continue Broad. extremely portfolio We in confident patent

Operator

Harrison Our next Stanley. question with comes from Matthew Morgan

is open. line Your now

Thomas Lee

sickle at you're Lee able need a dose efficaciousness? This you more, strategies cell? initial good low editing Given about to thoughts how for talk site, Hi, Thanks. dosing a Harrison potentially Matthew. evening. is versus to Can start you being that the your start on unique Thomas in

Charles Albright

Sure.

do cell doing to you a is you response want a dose don't set So dose hematopoietic transplant The sickle experiment stem like we're disease. in ensure for good engraftment. get cell you that because

it's add dose so, key is that the hematopoietic no cell. that's why And you really be We stem there study. dose finding high fraction what a and know in of should

that lead engraftment we have studies we've properties. more show we than so, distribution of in key those we'll could in shown are that [indiscernible] preclinical added and the ASH and And the XX% pancellular and to more that

Operator

with Our Seedhouse from question comes Raymond next Steve James.

open. Your line is now

Seymour Slatkin

on This the study. Steve BRILLIANCE Slatkin for is My hi. fiXrst about is Yes, Seedhouse. Seymour question

mentioned a anymore? mid we to your see the previously you end, don't in dosing this mid-dose there extent guidance what -- dose does in was So some because and haven't impact slowdown, but think I those to we really year that cohort regarding patients by

Cindy Collins

That's guidance. correct. the adjusted We've

As are I the a with mentioned, we situation. bit COVID of seeing challenge a

outside of the travel treated have of There's of reluctance the We well. also as restrictions located easily borders. that during to because U.S. across travel that be period cannot COVID patients are patients some

patients, are We investigators directly forward. we continue to the work all clinical to enroll open, in the to screen continue with those bring patients sites and

Seymour Slatkin

thank if Okay, safety any study? months dose from escalations for say to I'm you safety first patient assuming And you COVID now. can about delays sure, not eight if that. follow-up of on I'm like enough you the sure, the whether has The now looks continue good not fixed BRILLIANCE can corrected? is comment

Cindy Collins

So, that are I to will their that say dose clear we patient.

Seymour Slatkin

with the regarding trying I is XX through question. about IL-XX used question that And you're you of trying that presents, co-administer presence -- about antibodies your talk can you cells of And sort what talk know, that you're ASH abstracts you that, target the And for knockout last something the NK co-administer actual test? But mentioned be can to treatments? also to IL-XX. to other you is in importance one the Could cells. this double are of of one grown mentioned

Charles Albright

tumor. didn't you [indiscernible]. By understand the sorry, or I mean targets targets the the I'm knockout

Seymour Slatkin

co-administer other want that you therapy to NK cells? basically It's the with targets the for

Charles Albright

Yes.

to that's pathway. IL-XX. in sure both NK and preferable we seen IL-XX negative sensitivity question. of target to the the co-administer doing that knocking that's vitro the we've quite on I'm out effective. IL-XX and we're the it's because gene we And And not So, it's a cells mouse the models, in CISH why increase regulator, not we're going still with that in vivo decided

So, we're too knockouts.

we're yet the question. the in was to disclosed tumor humans types haven't that test We if that going

Operator

from Our line with Truist. Lee next comes question Joon of

Your is open. line now

Joon Lee

Is the disclosure, some that patients, to sure I'm you're are learn On for and efficacy trying safety. I but the to of EDIT-XXX be the investors appreciate eager to fair responsible about you follow-up in something to not to are a And efficacy about Or I will you just willing have assume commit that the safety XXXX? and face that question. yet? it

Cindy Collins

not ready that to yet in CMO are to We our just and I to consultation release plan do new commit develop a data and her. with with this want

Joon Lee

a for Just questions Charlie. couple of technical

XXX, I you promoter, believe kind gamma X there for X X, and a of hemoglobin For target. duplication, is EDIT-XXX, where in where

are do such obviously knockout set effects? therapeutic So combinations both as versions you need dual. have several clinical There between a to the duplicated promoters of [indiscernible] a deletion to to in outcome single

is therapy Thank So outcome goal? your one for you. that desired there is

Charles Albright

a question. locus. a No, it's you're great And right, complex that's

so, cells relevant analysis phenotype genotype, believe it and XX% to say therapeutically edited of forward will disclosing more concentrations because fascinating. upcoming Suffice express it detailed we what to look hemoglobin. we we the And fetal scientific are a is have of that we done than meeting, an that

So achieve though our complex, locus the goals. able is therapeutic even we're to

Operator

Yanan from Our Securities. with comes Wells Zhu Fargo next question

Yanan Zhu

questions. taking for my thanks Hi,

on a EDIT-XXX. maybe first, So, questions few

repeat for to any also also what prevent And there that, is dosing endogenous together by ADCC, be Would about therapeutic. lastly, is strategy depletion, to are now have compartment, the you impacted, host that just a negative any Do contributor that EDIT-XXX? to cells? consequences? it which with cell been lymphodepletion, this would have NK wondering, obviously the concern be So, expect Thanks. could impact And rejection lymphodepletion? used T if antibody will they lymphodepletion, on and

Charles Albright

Right. This is questions. Charlie. Great

will iteration So, platform. cells our based we rejection we're do we're in we're And prevent cells we're -- return the NK we are cell the to of that think are NK our not some therapeutic clinical worried that that you yes, antibodies. we things and ones mentioned. that And of the this so about anticipate efficient preclinical engaging NK on lymphodepletion doing using the first outcomes, select adding to studies, going both the think very that of

Yanan Zhu

dosing And strategy? whether it's repeat

Charles Albright

about repeat clinical Yes, we open way studies the and into so take studies we the do do. stuff the thinking to to preclinical the that we ability want that dosing leave we're both we

Yanan Zhu

it. Got

XX% based couple that one demonstrated also surpassed benchmark clinical -- a hemoglobin question, hemoglobin one So The your additional -- patients you BCLXXA Thanks. approach rapidly has targeted in Do work? for clinical a in more maybe fetal to a value preclinical Phase question to-date. EDIT-XXX. trials surpassed And be be on with X formality? of can that think higher -- XX% HbF can be in bringing

Charles Albright

Yes, in medicine believe current cell more we and above clinical our with sickle beyond that that achieved. and be do believe need which the in hemoglobin we therapies. that with will patient fact, to provide And can and fetal benefit you XX% than can see do we do

are showing they a lot to the they are results disease. on there been and this these things And vaso-occlusive we that yet other that adequately not of trials characterizes premature that in tested crisis, believe significantly mortality contribute so while impressive

now. look benefit what's forward And to above possible clinical and beyond we so, providing

Operator

back question-and-answer the call That turn Cindy I'd concludes remarks. like to to for session. closing Collins

Cindy Collins

Great.

call we to support, So in new you safe. as we bring thank and today's be medicines Take all participating patients. that, your work to transformative with for and care

Operator

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

now You may disconnect.

Editas Medicine (EDIT) 5 Nov 202020 Q3 Earnings call transcript