EDIT - Earnings call transcript Q3 2019

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Operator

Good morning and welcome to Editas Medicine’s Third Quarter 2019 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 releases two discussed issued this we morning, press call. on that this be will

The collaboration our amendment first Celgene. longstanding announces an with to

provides second quarter third our The XXXX. release financial and for of corporate results the update press

Media after will of replay its Q&A. our remarks, we & completion. the will our call website on the be After A of available approximately open for call Investors today’s section X hours prepared

we purposes make Reform future under that this Safe a call prospects company’s remarks As Securities Act reminder, provisions the during expectations, constitute about various Litigation Harbor XXXX. Private the forward-looking of and of plans statements for the

various Factors those Form in materially may Actual with important by section results including of from indicated the is most which those the on of discussed report forward-looking SEC. recent quarterly statements our Risk these file differ on factors, XX-Q, as result a

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

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

Cindy Collins

Thank us quarter everyone XXXX Good and corporate for you, Mark. for thank you update. morning joining our third

with Medical are Albright, highlights of Mark, Editas quarter and Eric joined and Officer. Charlie Officer; share a to excited you Chief productive our the Chief today. new Officer; to some including by Interim I’m team, Scientific Chief addition Abrams, Editas had In several members we Ek, the executive of has Financial Judith our our

team an the Judith the experience her am our trial. arrival ideal dose pleased X/X as Officer. Chief Brilliance we reputation. our valued is as first is First and a and She patient Judith executive new to Phase to Abrams, addition clinical has I prepare welcome and Medical to Dr. tremendous outstanding foremost, in

our to to morning cell we cancer. engineered an T for Second, longstanding amendment medicines develop Celgene collaboration this announced with

of As you alpha-beta cells. may to recall, The amended our cells. original T field non-alpha-beta will develop up its on using collaboration collaboration cells, freeing Editas the engineered T entire medicines own T spanned focus

a million are the As a entitled potential T receive for of with amendment, of and royalties alpha-beta $XX this future we to our cell using by developed payment technology. milestones result on Celgene medicines

to solid with to candidate our upcoming at Third, beta treat progress vivo the data has it why and making potential believe Meeting disease a we ASH forward to preclinical best-in-class be sickle we sharing are EDIT-XXX thalassemia. the We medicine. cell in look demonstrating

recently we diseases. with to AskBio, CRISPR Fourth, portfolio formed therapeutic develop focus in marks vivo medicines our neurological a unmet with an area need. AAV in new in high a to therapy This medicine second CRISPR collaboration for our in leader vivo gene treat

ophthalmology portfolio with Finally, first by is anticipated our patient of early XXXX. the dosing advancing LCAXX EDIT-XXX for

I'd like to our over new Medical to that, to Judith With Officer herself. call introduce Chief turn the Abrams,

Judith Abrams

us the Thanks, all introduction your Cindy, being and you thank on for call for today. with warm

the disease for had of of pipeline immunology medicines my neurology. treatment of the more across and I'm cell sickle forward pivotal all as to to additional the to of spanning the cell during our medicines time experimental of as course and Medicine than I've in development, look treatment engineered clinical to number future years, of opportunity a and advancing including EDIT-XXX thrilled stages areas, EDIT-XXX well cancer. through medicine including Editas for the a Over therapeutic career development join lead XX development clinical

you Now, Albright, on our let the to me Charlie call Scientific turn Chief over update Officer, pipeline. our to

Charlie Albright

Judith. you, Thank

at We've to medicines cancer. autologous been cell medicines Juno and T amended partners collaboration now to cancer with treat Therapeutics have and focus allogeneic and our diseases. since cell Celgene T our XXXX treat we engineered to develop alpha-beta on mentioned engineered to autoimmune developing Cindy working Celgene with As

expire next term of medicines set enable As research into was original to to advancement year, the in focused we’ve the clinic. the of May our extended collaboration and

amendment disease have result a for T non-alpha-beta oncology. the to regained rights rights Celgene of These previously in cells areas. to develop we As exclusive were all

of immune of believe that potential the For effort have as significant are Gamma the both immune both pursue and example, NK iPSC-derived tumors, T where we an T NK and right treat needs. cells we using delta innate area unmet now system a gamma have delta cells our system T alpha-beta complement cells donor-derived to We increase to for cells cells. oncology. from innate cells part solid significant the

Moving other as CpfX cell the a our sickle best-in-class EDIT-XXX disease developing medicines enzyme of edit uses on engineered EDIT-XXX in beta-globin and locus. treat to engineered beta we're area hematopoietic an medicine thalassemia. to stem cell at focus, cells to

upcoming We impact of will so overall are this as additional which target last Society of describing the of At year's program from as and We'll data excited data award. we Meeting while be The work in lineage. confident in ASH and our ASH this believe by efficacy analysis. data well. as erythroid captures think we on and might our product encouraged program the of showing configuration, vivo survival was recognized best editing patients Hematology the totality we as the are present enhancer, clinicians other program Meeting erythroid our approaches effect target at the American this BCXXA

a medicines to edited developing treatment gene partnership pipeline, diseases. in gears we're vivo our in Allergan, for inherited renal Switching with ophthalmology portfolio of CRISPR

X/X the safety, suffering to and evaluate efficacy. program from its lead and Brilliance treatment underway for Our tolerability EDIT-XXX is is patients Phase interventional study a LCAXX

and patient being surgery that screened identify successfully been are of patients been clinicians date pending. available scheduled and actively in dosing, we early the multiple and participating With sites initial we trial has for screened are recruiting potential confirmation anticipate manufactured is with a said, first has the product The XXXX by drug for and is to engaged that cohort.

to causing mutation. and for XA Usherin At and XX% treatment to editing Type the therapeutically includes we out of the functional protein Meeting the disease Our common relevant European Exon contains an explants. restore retinal we up which portfolio of ophthalmology Cell demonstrated Society Gene correct XX, most disease, fully knock also in Usher Therapy Syndrome human experimental

in on and to paved regulatory gives assays we've many the develop us vector, draws utilizing program disease candidate. treat work of supports and by confidence our The path data our and the enzyme on research the development same ability the This the AAV and and following leveraging EDIT-XXX preclinical further promoter CasX same models lead functional of done to heavily EDIT-XXX.

CRISPR AskBio diseases. in formed treat vivo a neurologic to develop medicines with to On ophthalmology, we recently have collaboration

drug of The expertise in manufacturing. knowledge significant combination host editing expertise and the brings development the Editas’ AskBio’s vector expertise and need. system, well-known and we of development. AskBio gene central address AskBio, in With with engineering manufacturing with could unmet and high target a neurologic peripheral in challenge for coupled diseases cells nervous

editing treatment example, serious For potential in providing as in we the are gene excited translate additional forward as future debilitating for disease. editing the near to to the updates look treat diseases. We patients Editas to gene about Huntington's with on transformative works for pain our programs

to over update. I'd the that, call provide With to who a Eric, turn like will financial

Eric Ek

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

quarter XXXX. Our securities from in $XX by third XX, million of $XXX September and million million equivalents cash, of cash as marketable June as increased $XXX the to XXXX XX,

and million $XX capital totaled cash expenses $XX include of uses of and million, Our $X cash of million. operating expenditures

capital income. believe consisted million equivalents and to million $X marketable related XX $XX interest and cash, the cash million fund to totaled months our of stock of of of cash securities exercises of and the $XX of offerings, sources our least at $XXX business. $X XXXX, September provides aftermarket Our XX, million as option million We of

with hand back it And I will to that, Cindy.

Cindy Collins

have It has patients the and exciting work the truly promise cancer, for work and work a cell and possibilities Eric. programs started. past neurological advance genome Editas only potential is our the for but to conditions our deliver for time the revolutionize diseases. explore It programs we of sickle been for to as treatments have serious the Thanks, getting disease an treatment busy blindness, with genetic doing with CRISPR to us, Editas new we few editing. months been of is

the holds upcoming about] future scientific program data advancement. with what are and [encouraged readouts, achievements We

Thank investors, you all researchers, for we during importantly, this journey, your support interest alongside have and and patients. most making been clinicians,

your take With Operator? that, happy questions. to are we

Operator

[Operator Instructions].

Seedhouse Our with comes Steve from first Raymond question James.

Your now open. line is

Timor Vanicus

And Hi, readout. on for this recent Vanicus for have based couple we Timor Steve competitive on questions is Seedhouse. EDIT-XXX of a

didn't it And worst the the had a turns was then one baseline responder differences couple and specifically or of best the super vision. there say and company the patient the had contralateral they significant. eye relatively were. dose his lose LCAXX improvement LogMAR longest to was responders low cohort. whose So in genetic what vision And at out time addition And X.X to the in of they epigenetic

super study? how in potential the your those given we responders high potentially you for addressed and So response, wondering placebo were Thank issues you.

Charlie Albright

they're today. of Those You're explanations monitoring been for be the not seeing right. things Charlie. obvious we is we This some and will phenomena are have

Timor Vanicus

be? see cataract Thank of an just do guess also in how what many terms expectation I have you follow-up, do for And you. safety baseline study develop rate you will the in Okay. to would -- a patients your

Charlie Albright

We any AAV-based they than expectations the this therapies therapy. other have space, don't different any the be will other in

will something clearly monitoring. we it’s be So

Timor Vanicus

on a quick just then And Okay. housekeeping partnership. Celgene updated your question

like clear. mostly how getting XXXX? it looks it seems Not million. $XX expanded much your It rights. of it Is for some you're also that you're Is like So because the is back getting timeline? additional just after

better? a could Thank if So little you explain you. bit

Charlie Albright

upfront yes, an to-date the the right. $XX The forward. contributions is you're we've recognition done and going And million in of payment work

We are rights getting some back.

Operator

Stanley. question from Morgan Harrison with Our next comes Matthew

line is now open. Your

Costa Sun

timeline can This morning. bit good XXXX XXXX? My for you little for question Matthew EDIT-XXX to back explain Hi, pushed what first of Harrison. the a from first is, into be Sun caused dosing is the Costa

Charlie Albright

a particularly all as because rare as since caregiver, thing adult to to entry And the challenging only. the a patient surgery this after has for you further perception fly is a light disease appreciate, before the can is diseases holidays. Sure. they need right days typically arrive to with retinal -- This for to inherited criteria patients first is that’s XX the the this The need challenging, by bring surgery. is patients restricted and accruing rare, be It an challenges and with it's can't do. with complicated

So we the of things holiday complicated approach season. just has made those combination as it the

Costa Sun

And for beyond progressing how is the other patients first treatment this patient?

Charlie Albright

well. progressing We're

study. in we sites are patients natural screen more two two the of the patients the natural and In addition history history for we've pending sites, to to identified opened we've study, continue outside

of which part as have our screening we patients so, patients cohorts. in identified potential the And subsequent other are

Costa Sun

one question Can more the in you about your And please. CpfX hemoglobinopathies talk program? for reason

Charlie Albright

Sure.

application. best of ASH our Meeting So general the effector will the the to a the the is is because they show productive you're knock as the all transcription to program enzyme strategy transcription effector knocking this, site And hemoglobin a at edits where transcription And in fascinating edits are out out site. use binding not we one, effector. binding for actually the

stem turns So a long-term in when this edit get hematopoietic with that out fraction CpfX, cells. of it higher productive much in we edits we case the

this And so relatively fetal of is where of is, the program. hemoglobin, create which we there product really maximize the induce ability a course, to to objective what we've done unique

there. journey it's the look interesting So been ASH really upcoming forward an We Meeting. to

Costa Sun

And a question, one on this. follow-up last

expect of another inform would areas data can from diseases? in you CRISPR bit a before again in hemoglobinopathies how year-end, about data medicine that this your little Given strategy these we talk

Charlie Albright

competitors this every Well, we the space in in we carefully program. monitor do as

we to a interesting -- will particularly monitor we We have upcoming see of it that and believe reason have we that we've to-date. time to data with the seen do medicine in best-in-class light and have clearly developed abstracts the be

Operator

from Our Whitney with Guggenheim next question Ijem comes Securities.

Anvita Gupta

the this in versus next particular CNS then us please vectors And your you This initial then I may in vectors that color Thanks. indications AskBio and an on gen AskBio countering is gain this attracted space? have Whitney Anvita access the to be you for space. additional under that morning. may give more you what Could And to in you other follow-up. the on collaboration

Charlie Albright

they've of only track AskBio significant people We're putting a a have they also manufacturing. have that nice businesses developed clinic a Sure. record complement in impressed not are excellent efficacious. with They've acquired AAV AAV their involved and into in They've core core manufacturing expertise infrastructure. have very They developed the Ask some the and facility. business record, in a the leader medicines track are an and with significant

group working to disease. we're super them So excited it the be and impressive neurologic on with is very a

pointed pain. Our will to efforts be initial severe

we the area about And so tremendous genetically unmet think an don't high a targets it's say And of something are validated. do now, we want can it to but we very need, where unique.

Anvita Gupta

sickle then beta between for program, target that that for? there and thal any cell my your diseases? And And Thanks. questions. what Thanks and the two the you're is difference is aiming HBF taking

Charlie Albright

need get believe than in fetal needs significantly to more We XX% deducts and pancellular hemoglobin obviously. be it we to increases

And at have so medicine. we'll a experimental ASH exceeded interesting and show we those data we've that very believe targets

Operator

next Wang Gena Our from with question comes Barclays.

open. Your now line is

Unidentified Analyst

in Usher do question LCAXX you for that My This is of read through. interim to How syndrome dosing? data the Gena. think on first LCA is read David the regarding terms data through

Charlie Albright

the the to doing modeling are we pharmacology editing these of dose. the each with correlate For

that will be that we between will dose similar. we've next but that haven't year. some shown anticipate do medicines And of We responses show we quite seen data, the preclinically, more

here. with the in as and same anticipate different medicines, So spaces across thing the we is pharmacodynamic, maintained molecular reasonably typically therapies well all relationship pharmacokinetic

Unidentified Analyst

data is disease. for and the preclinical Second sickle regarding thalassemia cell question beta

in it Your X? to can Phase what preclinical PCLXXA expect the and to approach compare how ASH, of data does timing we at terms

Charlie Albright

think poster by compares we Taking course in we disclosed Phase you able data and we be timing hemoglobin X. IND-enabling I it as that The favorably, said reverse to What though is. order, as we’re haven't rapidly is the to as when it you'll begin advancing are a in possible, to see judge studies position stand of far as that. the

Unidentified Analyst

you Okay. editing Very do the impact the helpful. recent on out, CRISPR? is news And the then last question think prime what to that came

Charlie Albright

The We and editing of monitor of editing similarities editing, across space many frankly. modified competition many has to the for -- issues same base prime approaches direct has and approaches. is the the the anticipate the gene same the gene quite we many both

Operator

Our comes next BTIG. from Murphy Amanda with question

line Your is open. now

Amanda Murphy

interim data So presenting guess T readouts the on various on while what that just now a analysis, questions front. few to you've know conferences, kind or I up and the been cells. been of for preclinical at I change a on Celgene you've CpfX

assuming in CpfX that's T kind you us terms done that So guess And just what previously been also what A, TCRs on then, engineered that presented? update back the call -- curious sort of includes of, if I can if to with work taking happens and you've that I'm of you will.

where you and could programs, us of are update So, update those from curious just might if on indications both timing data? and you different with those an on when at we have

Charlie Albright

will knockout the collaboration space. carry have the lot new integration, and have Unfortunately, I programs. we that's be clear in are we a the forward into typical details target possible. I the multiplexing, Yes. shown data not of work and that one of would say the to on what about could genes the of timing the Celgene say that wish shown Sufficed more

work new into with collaboration the All Celgene. is rolled of that

generation as the -- of and As to oncology interest have editing a certainly what leadership the believe important likely leader oncology. leader and best medicines CAR-T T cell part in BCMA gene position their is imagine, best be including of for you arguably a maintaining are medicines cell next that we the programs and for CDXX in strong an the can T

continue with space. to work So we're excited to in that them

the the to unfortunately the timing use that. about be of just nature we'll be the we or to products. knowledge And So gained we've able the can't to-date products of transparent do

Amanda Murphy

you your you then sorry timing missed cell iPSC had NK the obviously Have talked this. of cell as on of the I and there program. and about well. is? of sort And just collaboration individually know about think I NK iPSC program derived then kind running I’m if NK sort what And have you talked I

looking just So update one? can provide there if for an you

Charlie Albright

want. as Sufficed cell hear meeting. stem and specifics the going and year about probably you you'll oncology the science into say update we more the general go that a well lot that the really without provided really We’ve is to next

to collaborator identify able are iPSC and our We well characterized lines. defined with BlueRock

iPSC able edit lines. been those have We to

We NK vitro. things accumulate And in into differentiate the killing cells able now advance will the medicine. combine what experimental will we those into be potent and We're to those to have all we interesting believe activity starting rapidly be them data as to to can that clinic.

cells. that similar we think NK We effort have a cells iPSC the NK donor derived healthy the derived complement in

do update scientific significant we that providing in progress next venue. So illustrates year anticipate that a

Amanda Murphy

one If I sneak in about EDIT-XXX. can more

going more second from dosing? on again us protocol here that to just the for perspective patient what's little reminder -- a there? be remind just Is So a timing of required

Charlie Albright

the are between the entry for six dosing patient and criteria only quite an those two so one I There's of Yes, is patients, similar, light both patient which adults. interval two perception believe weeks.

trying And obviously patient cohort. required what's first accrue in are first that's second to the both so we a the and

Operator

from Cowen Our comes question Company. Joe with Thome and next

now is open. line Your

Joe Thome

first program. one XXX on The the

you that of the we perception In just terms I interest patient how program maybe for didn't kind disease patients light of advanced that or want of a later would sitting? patient is mentioned kind the first with cohort in that disease. be Have screening, have applicable know you enough cohort patients and obviously advanced screened

Charlie Albright

Yes, the simple answer yes. is

not did We have cohort criteria the for cohort they X, X. and screened the could criteria fit patients that for X fit

physicians and obviously, to going interested And we're study in them. optimistic start so to dosing further high. enrolled. in that we're interest remained The and their And patients we're this get

Joe Thome

for -- but year, patients that one looking of then terms cohorts would you're disclosure? program. data potential from in cohort? I releasing following anticipate few ahead, of interim. you had Would a How more data know Or there wait on is but a it's for some know release And a dose the first you you for meaningful your data? time? for amount of until do you I

Charlie Albright

Right. quite the It frankly. data depends on

are you the Allergan And we with they're of study. going that partnering to data. And say remind so release in in have an this important

we basis, by said earlier responders by data. of some release can We going illustrated on patient-by-patient misled you be we're super data be because don't there to And are questions, will one significantly not patient-by-patient a the as have And releasing that here. date representative. believe

with is is. I so our discussions on think partners, our data And it And much what Allergan. in very dependent the

Joe Thome

$XX just of then going Great. account to deal. for can when you that specific you on opt some maybe housekeeping before that? on they And of time The how one do QX, expect pivotal is are about milestone, there receive kind that? Celgene Celgene million And in the last proof-of-concept of question terms after in to of a a are these or how in thinking programs, and

Charlie Albright

So, any in the going they accrue The deal so quarter. we structured at they point opt time. in, are yes, fourth can to in in opt is

Operator

[Operator Instructions].

comes Our next with from question Geulah Chardan. Livshits

Your line open. now is

Geulah Livshits

one and one the So quick LCAXX then oncology. on

with program, So, respect LCAXX just to the

make was rolled to understand, identified that I history the from patient study? in was first natural sure that

Charlie Albright

Yes.

Geulah Livshits

to of kind overall of with a broader terms oncology then strategy, And Great. the in Celgene collaboration respect question.

cell So versus And towards liquid thinking you color there’s process can collaboration? tumors about wondering Celgene have towards versus a on how or tools decision delta a that. you or a target Yes, just solid NK and was internal now. if you're or number your deploying towards particular different say, key any should disposal I at provide gamma of therapy, the I

Charlie Albright

Yes.

directed working as much medicines leadership believe of is think leader very beta with I I alpha have somebody their is who top and a arguably at those CDXX hematologic And Celgene T BCMA of T about a cells. both spaces. know, with be top list the that do. beta they're in are in collaboration the and indications. best said alpha of cell of that to you leader at in their will the the they maintaining as things at And we So portfolio

relatively of on provides tumors, need editing to been where successful unmet required. can that of to that But to significant We today. an significant going changes area changes solid iPSC And effort believe ability engineered medicines make focus need be are going do that’s genetic there the genetic we to the in that. platform we cell going most have get our are to really

immune evasion need ability in donors. you're need the no instance, system. cell way get immune going derived For there's targeting, evasion from you going the there And probably increased the with to tumor type from healthy system, you're to of and microenvironment to persistence, any stimulate endogenous --

is there's And and unique to our unmet a so has a into we're space are complex today. albeit make are They are significant patients, internally which focused cell Right from on us will affords the in data easier hematologic that the space primarily building in to up iPSC need. platform. that NK a And types make of platform they ability cells. in the products believe efforts this clinical get indeed now one emerging efficacious we indications

about excited a made all disclosing year. very that are that. lot we area at and We've So of to forward that look we’ll in meetings progress next

Geulah Livshits

like an So theoretical, the with include option then structure kind potential of but alpha T on beta based cell something derived of the iPSC rates agreement? Celgene’s

Charlie Albright

not agreement. focus of could, It the that's initial the

Geulah Livshits

then one the last collaboration. on And AskBio just one

and have Sympromics you So platform? AskBio the mentioned collaboration, with the informatics went do as curiosity, access you just out of to so under you through

Charlie Albright

is under that's expanding in great to AAV believe whatever a example their to And the access become have what really we we leaders technology they're the one the of umbrella. AskBio the do, is We footprint way of field.

Operator

Tuerkcan Our Oppenheimer. comes with Silvan next question from

line is now Your open.

Silvan Tuerkcan

approach about and He to if in there which you next potential that sickle ask a EDIT-XXX? the year. will trial could anything from at Is Porteus’ wanted up ESGCT Matthew his? be the apply read across you I starting X his off, was -- maybe academic, And trial could approach, differences between is disease. presented Phase highlight First to your cell

Charlie Albright

you're understand going it mutation. -- of to of after that sickle some has root correction is appeal because actually mutation. cell approach And the Sure. His right the the target I obviously theoretical has as some

genetic not changes problematic at productive the you correct cell the them the to correction mutation. all are an the And of of which strategy allele don't lead aspect likely you all that indel, is appreciate, highly when i.e. of make be is to non-functional. to As can you not sickle actually

those and those now potentially hemoglobin contribute the patient. alleles not So to will cells

you cell make correction, -- take enough beta patient have and patients And sickle significant make we a get actually patients have so if risk. found don't be them potential thalassemia the thalassemia you to which beta to

Silvan Tuerkcan

Great, thank you. a please potential what's us left And what for remind be do an you to could IND could and of Usher file to timeline?

Charlie Albright

We haven't disclosed the would we be that timeline, what we end have for said enabling the a is of molecule ready having IND that at year the anticipate study.

recall program Usher Allergan. you part collaboration so is And our the agreement. that an it's option of And with

Allergan. the provide to we So And have they at phase the a right opt of in. end then to packages discovery

anticipate to potentially year. them a end track -- We have made we a we have the the that. the delivering decision they've And of decision at on that that at decision, have So point, make. package they'll as make remain will And for to once well. to

Operator

I'd further to to no turn this for closing Mark the any showing At time, I'm over remarks. questions. like Mullikin back call

Cindy Collins

Great.

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

Operator

you this today's and gentlemen, participating. Thank call. for Ladies concludes conference

You may now disconnect.

Editas Medicine (EDIT) 12 Nov 192019 Q3 Earnings call transcript