Editas Medicine (EDIT) 28 Feb 242023 Q4 Earnings call transcript

Good morning, and welcome to Editas Medicine's Fourth Quarter and Full Year 2023 Conference Call. [Operator Instructions] Please be advised that this call is being recorded at the company's request.I would now like to turn the call over to Cristi Barnett, Corporate Communications and Investor Relations at Editas Medicine.

Thank you, Maria. Good morning, everyone, and welcome to our Fourth Quarter and Full Year 2023 Conference Call. Earlier this morning, we issued a press release providing our financial results and recent corporate updates. call website available its approximately today's section the completion. of our of X replay will in be Investors A hours after Q&A.As XXXX. statements about under Litigation the After the future harbor expectations, that we provisions remarks, prospects company's purposes make a constitute Reform of and for safe forward-looking we call our Securities remarks the reminder, Act the this will during Private plans of various for prepared call open the views indicated those those by forward-looking results with Risk may by any XX-K, from Actual various recent and as updated including today the upon addition, should our materially a which not as factors, file be Factors any representing date. most SEC of Form discussed these of result as represent report our differ statements as only the our on annual views is section relied of important filings.In of in on our as subsequent subsequent forward-looking statements required by statements even Except forward-looking any turn I views change.Now Gilmore our update our the O'Neill. any law, will to CEO, disclaim as over revise if to or we specifically call obligation

we and editing good today vision Editas' XXXX Thank Editas our leader Officer, Burkly; Financial the executive with Editas Scientific in fourth Linda Quarter of Medical our Baisong in-vivo you the as morning, Chief of early XXXX. all hemoglobinopathies. and for Chief and Officer, progress position shared on by programmable and Year in am to Officer, joining and Mei; pleased Editas' Chief Earnings quarter momentum Chief members our and our us Commercial gene Full joined I a pillars are our the XXXX, of Caren and Officer, X strategy everyone. Fourth Strategy and Deardorf.We In Call. Erick today Cristi, other Thanks, X team, Lucera; our

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a cell So beta and for objectives Reni-cel, in for sickle by trial for our and are thalassemia what trial EdiTHAL XXXX. XXXX? provide we will transfusion-dependent For mid-XXXX the severe update the RUBY year-end from disease clinical

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everyone. morning, Good Gilmore. you, Thank

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now trial a As Phase we clinical aligned BLA Gilmore RUBY FDA for with mentioned, is considered trial I/II/III filing. have that

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including Financial data more year-end.Now longer and updates, mid-year call to outcome ameliorate normal with by RUBY of level EdiTHAL look additional data total sustained We in important an improve hemoglobin Erick, quality correction and of additional reminder, life As our organ Chief anemia clinical can clinical is patients I'll follow-up sharing significantly Officer. over period trial and patients a forward the turn an to for and a damage. the and

for refer review of million, year to million I'll opportunity you, to to Thank you a XX cash, items marketable release morning, like Baisong, for everyone. September XXXX, with and financial and full to were happy to and as the be equivalents and I'm briefly you, and a XXXX. of take today quarter quarter.Our earlier cash fourth the summary results securities good $XXX as our speaking for press fourth this I'd of December $XXX XX, few compared issued

in additional and the contingent primarily from securities, $XX expenses and Reni-cel fees expect announced as payable the and June fourth sublicense cash We into December by equivalents which offset from license of Gilmore expenditures quarter license for our $XX increased the Editas quarter $XX expense sharpened decrease XXXX R&D financial recent existing together and The focus, of marketable call, by legal and XXXX capital attributable expenses expenses relates patent million reflects XXXX.Revenue XXXX. in million our our with fund million position, $XX license reduced costs. of primarily and million, recognized with earlier quarter on this on which for out-licensing raise to our R&D from discovery program.G&A reprioritization $XX fourth under deals. capital expenses the Vertex, decrease remains upfront Vertex our focus fourth agreement fourth cash, in million, operating which, bolstered spend this to the our payment XXXX. is our in the strong of were Overall, to quarter a The to our of for referenced agreement by near-term with resulting XXXX. increase quarter decreased annual we revenue targeted under was

the in to Our commercial progress XXXX resources and clinical our support preparation discovery provides at that, RUBY hand the and back cash efforts.With research Reni-cel, EdiTHAL into trials I'll continued runway Gilmore. ample continued our of to the manufacturing advancement call and

Thank you, Eric.

a XXXX progress very proud XXXX stage are continue evolve We momentum our with you. our We development-stage a to continue commercial and gene-editing forward transformation from forward into to to the of sharing look in our platform company. technology accelerating as into and progress we look company

XXXX clinical severe our enrollment we transfusion RUBY a disease RUBY continue for the Reni-cel trial complete thalassemia Reni-cel, we As for cohort mid-XXXX the adult on will EdiTHAL. include; for trial a and XXXX.We reminder, provide will the strategic update year-end will EdiTHAL in and dependent initiated cell beta adolescent already objectives and sickle in cohort enrollment, and in

pipeline, for drive and vivo value core questions. an proof in investigators, answer in IP and you. objectives corporate support establish technology not vivo that partners PD, could gene always, it concept in Thank employees, leverage business much our will our Editas, your we And robust we will very interest without to For said development achieve must our capabilities.As undisclosed you.Thanks happy preclinical be we're patients, complement we and and to editing our the indication. caregivers, of of for portfolio for

[Operator with question from first Truist Our Instructions] Lee Joon comes Securities.

quarter, the us of such you as elaborate question sorry that, on they for quality patient-reported will and please to how voice. tracking my on relate that is and My tell you're hemolysis Congrats outcomes markers could life? the

your Joon. pass to better. Thanks going voice I'm hope that question Baisong. to very I much, over that gets

Yes. Thanks Joon. for the question,

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question next comes Citi. Samantha from Semenkow Our with

additional the potential you'll amount you aligned specifically patients need a insights trial, Can your follow-up you the any to on need BLA? of of terms into conversation just number share FDA as file RUBY you'll in of and the

going Baisong address to question. have I'm that

the you for question. Thank

we trial FDA support have -- the are still to the further the and including with data have FDA and RUBY BLA, with engagement BLA be to sample we'll and the study And with follow-up, a are aligned to I/II/III size -- we and we the FDA. endpoint, So trial discussion about a Phase design. the about continuing package so

Cheng comes JP Morgan. next with Our question from Brian

for of us early sense what Can also of potential that so Phase really they RUBY from feedback And far. kind I you a give mean the if hearing talked Baisong, just a potential I'm can perspective? on destination the does timeline differentiation, I/II/III from this feedback been about on? any you just curious about Are investigators seeing you talk you've investigators. differentiation think

Thanks very much, Brian.

Phase there sort were the to What and parts So, was on differentiation I they X BLA think your on seeing question. investigator were feedback the and impact is What the of Baisong its part I/II/III X. point?What might up this signs ask or they things address that seeing were then and I'll the be is do what patients at the first in path? other the to just on follow

a single I I/II/III, there's that single? to a agreed think it I/II/III regard the Phase Phase here with point is So is We've that key study.

what out that a think calling outcomes used that about means that to size for remain was designated that was or What on to are basically their are and on a track the study more BLA.I BLA Phase it's agreement application, even important I/II/III the on track BLA. is also that prior before of confident was worth the vertex have the study. being and We we the study

from of used path worth Vertex, point to Baisong? BLA. mentioning filing with sort that was out view size the I the based for the from approval. to calling think of that the year just And benchmark helps on a actually that originally just regard last we hope I So filing of have that our of BLA it's

Thanks.

feedback that hemoglobin anemia from be differentiation actually just also, engagement and less more much see investigator we patient also the energy, and and a and investigator quite big So, a And -- the to observation. to have appreciate observation able end to total of wise, the published pleased hemoglobin told they organ as so level, of very the as function. then own on that correct see anemia. -- see and impacted have their of their us direct patients level total well their they the the Brian, they fatigued they're as And that with for they their is as very hematologist, correction data,

also that are those looking So for. the directions we're

like with And one because that's hope I/II/III, allows Phase a single that just other -- a regard I/II/III, support study Phase from a study, -- thing I the BLA. converted the I'd quote to think I a is it's with seamless thing study helpful. to which we've just the Phase agreed to been the RUBY important FDA, has I transition that it's to to

Yes.

Just what say data use to also is means to be to able Gilmore from all add we'd the to study BLA. on support Gilmore's point, patient the the

America. comes Bank of next Greg from Our with Harrison question

Now your that fit how sickle the what in on competitor? cell, launch are you gene-editing there's the in treatment by a from approved what latest have thoughts the space, learned early Reni-cel and would

very Greg. address Thanks that to ask to going question. I'm Caren much,

for been and from lot Yes. this hearing some a really of Greg, thanks What your various in stakeholders good initial question. momentum. space is interest we've the

that is I to government as pilot think which starting your it's I on happening.So, and gene your well in tremendous families, think on qualified even and are CMMI all that the the interest, so happen, transplant, balance centers has the it's lot needs it's stakeholders, the all we're there as think is I patients, picking kicked payers. just also therapy groups, off. hearing and from CMS centers, your of you But up that. a maybe There saying centers, your that are across of your work

a vein going It's to differentiated, we continue a to where We else us is developing not really little in market going the be perhaps, timed to but just be it's a where encouraging. and very fast time. optimize may process deliver follower bit to to see is be vein that's it as it way. well. to just we can able product that see also robust to in the take differentiated, our to struggling, safety, that collect we of time data Reni-cel in very believe is going and meaningful, very aspects.So Gives operational take our efficacy centers think understand to way we of the time, So,

with look studies, more forward pleased in very certainly, we're interest our So initial the with talking about interest, and to it. ongoing clinical you

next with Leerink. Our Mani Foroohar comes from question

show in incremental source bit how time the independent differentiation, you timing You we investments, in that where about through like you of up data. derisk investors? cetera, of your improvements et CapEx part execution, as seeing talked an we're in terms in start points which of eyes a going the clinical vein-to-vein to of data Can little terms walk guys of could on of of clinical might that see you advantage think

Thank Baisong. Mani. for thanks you, question. is the Yes, This

are there the from We're process trial already We you the mentioned clinical vein-to-vein, in right. too. all optimize trying as stage to in

So to the and and sites in our with apheresis we patients example, cycle apheresis in prepare help protocol the expert the site help to amend for and our provide work the support. and

we've on So our that's commercial future of to the that. Caren kind help that pass now will experience for gathered launch.I'll

But their to is clinical make Reni-cel a get closer. timing, as our understand processes. to to the as to other able just existing always the really at certainly introduction be our opportunity we the as centers, outreach opportunities And advanced we'll expanding like the fit be we how working, we're in efficiency, looking more the we add, Yes. again, possible, and the engaged process how into of we have as continue sites and talk product very to work, across just, seamless do

comes Stanley. question from Morgan with Flynn next Terence Our

CRISPR/CasX XXXX? whether on Terence Flynn. you This argument Can an first and update is for Skor of on expect provide you appeal Max oral in the an case the half

very Thanks much, Max.

we -- that that and sometime the to update Court should can have Federal have of year, you we be schedule Appeals It scheduling, has We this hearing. on that. once Circuit yet oral

with next Barclays. Gena Wang question Our from comes

on may it RUBY on heard Gilmore, both think of patients. study that and sure a I trial. single if study, I So, FDA for the duration of number single the mentioned you study, not the be also correctly, I'm -- FDA of if mentioned agree you agree they Baisong the

pivotal about the And FDA if sites what how about aligned you sites that many wondering, XX a we goal study? And what of CRISPR require? Vertex Is So And active with is think trial, to now? trial, have total question, patients. that XX related is numbers? the the that your it's duration for

Thanks very Gena, your question. for much,

we Essentially, for is that I/II/III with when We're for will Before we've actually exa-cel initiated a the the pleased we a just BLA, Vertex's ongoing operating. BLA.With we benchmark with agreement our something. I so first all, we're the I which against informed -- up that puts track address very let it think, the talking with agency. And and separate very result, is by certainly active the a which how that on is and the I believe a about design, that with point good is used be by couple of a enrollment things. the benchmark. study. Phase its with enrolled as do of have a XX and the was the really a we clarify FDA FDA, adolescent lines the study not discussion sites, set benchmark From of of we're on, actually has the FDA.We very discussions of number regard that track believe view on me study to actually good study RUBY And for about with want actually BLA patients, that that to us to with our the were activated a the sites -- talk having actually the approval

Okay.

about to future. trial, more in sites to the established commercial the for already this then reason approved if the future very you the asking, perspective, it's clinical I'm your active in transition Sorry, once have commercial thinking easy drug

Caren, with to clarification. to I add don't and think, you understand that Yes, agree I if certainly, And that. we principle. that want that appreciate

get we And to coverage. a mean, expanded I we right, the that previously so, sites number had strong number to approach with thoughtful study, that support would already the know to no. sites Baisong? very that a no, ensure had of good full number a geographic of I No, was

can we Gena, we Maybe XX add shared, Yes. on I activated have already over sites.

with XX already between And we over patients. the already cohort XX adult have enrolled that also adolescent we a activating study And seen sites, -- sites and few so, pediatric size, same -- specifically we from the and a for are those you for are we overlap adolescent that more sites that's patient.

next from with Stifel. Gon Ha Our comes Dae question

to apologize, by I X-part progress thought the you actually just on RUBY. was it's dosed. prior a Reni-cel dosed But I I in conversation question. clarify, If XX the January. correct, XX heard

can How or So, another organ terms then in the FDA hemoglobin on engaged talk you're about differentiation, total differentiation, of that?And the sort actually the reason What that clarification of was function? When there? side. taking have on end the conversation? dropouts talk you the guys the they are you whether about on behind perceiving you them angle

Thanks very much, Dae Gon.

regard First actually in actually, a towards to presentation with we're set with of the that on has year, substantial that track patients of our BLA. driving of I with get to data are XX dosed. us And track really dosing, our a and dosing very with happy where us pace all, on we the for middle think

not We dropouts. had have

clinical scheduling, that, when and enrollment, experience, the think just particularly Baisong just complex a thing that I with and that.And final to waves like want you're going our et get holiday myself, and about and not there's be cetera. no is around downs So development that therapeutics ups I sure confusion you're dealing some around particularly to with dosing, but of waves, periods,

our many dosing to with and more and for for differentiation, with coming soon year.With the mechanistic and as data Baisong FDA say, the the middle the the behind as we have FDA, our think the in scheduled in our track FDA differentiation it's where on I are on the But substantive of remain So, differences I actually months, that. discussions regard said, to have also conversations patients a highlighted that cetera. et where we comment potential on too set

from Guggenheim. Our is question next Chattopadhyay with Debjit

are any CASGEVY's Debjit's And for team. AdCom's the criticism Forseth the on is breadth sort include size, off akin our first-in-class, profiling for primate editing, you're chemistry, program characterization to market Ry of This FDA you specific most the the of offer from excited feedback cetera. alignment program yet-to-be-disclosed with target the of about? question, second et around the opportunity The editing genetic for Did what the where to proof-of-concept, characteristics this diversity. going

Thanks very much, Debjit.

pass actually me question to first Baisong. let So the

Yes.

continuous engagement with have We FDA.

are with to is of CMC have molecule, we -- data scientific driven, the whole engagement understand the science is we So, was our engagement managed. range looking have -- of patient the And FDA the from how then a we clinical. and are preclinical to

engage with So, and because opportunity -- actually as FDA. Gilmore mentioned, we a have lot of leverage [Indiscernible] lot to of and we have a

are nature engagement. the So collaborative of the with very happy we FDA

because opposed what And oversight package our not a editing our to of as just very as a editing, data editing across AdCom. relative actually worth watched our frankly, is heard off-target gratified it's CasX. we when kind in that, actually in is actually the before, we add the I the robust nature very And we AdCom said off-target of discussion, we by that comprehensive discussion saw, is of were surprisingly, both And screen enzyme. and very to using hands confidence was we AsCasXXa in package which that high-efficiency genome-wide the as as detectable off-target engineered saying the others, and not high enzyme, of our is fidelity good, to because well our hands are own and

based standard -- technical regulatory potential include variables, of then care. current commercial I on want good including as key success that.And well differentiation on think it vivo I And I as success select about it we of conviction, for feel probability high actually the the are just and targets or we characteristics, said things very regard to is factors of the say, before, focusing set with to So from of are their that a are that success. the critical does that a number in as to

with Our comes next from TD Phil Nadeau Cowen. question

us Can have process? Our patients scale manufacturing be the commercial there on FDA, requirement to with in the is dosed in with question you manufacturing. are scale agreed remind a discussions where the in your in material? particular, And in you you commercial for CMC RUBY a the package? And process, upon

question. we for Thanks CMC a in much view, very are very good From of your a place. point actually

as -- are actually the our exist that launch excellent of obviously, there for our will commercial say and at FDA discussions ready building to that BLA actually in along capacity the know, our BLA-ready that that capacity. are is are -- and well ready you the very ready demand that have, We time. We be and progress I'd would then had will so making demand as we we we're line. launch.And the for have know, with progressing And with support beyond of be regard at processes, supporting inspection time that you

comes question Co. next Our & with from Jay Olson Oppenheimer

the Congrats on progress. all

And your you're thoughts of And are to delivery you Our how and program. you your undisclosed seems to it when about like question X share preclinical about proof talk since for concept planning deck, concept? you vivo targets in is Can and of for could programs? both on corporate choice also, editing your proof preclinical tool tool? share in any share planning there's

much. very Thanks

share driving towards our as And sorry, really editing forum we for that forum are to more to with about haven't we forward reasons. our the on look forum well, we the tools, say, year for be the vivo I POC an of when, the would to in I in POC are be preclinical scientific and to to So, track vivo And share excited view, we're to in we're the be how we POC. later editor, a and think, made yet number of more with on point a and sharing it'd in clear focus able regard the year this in a from on that. data. determination whether on timing, we focusing vivo is which And been have, other or pipeline this focused very the towards we a call that about the and going AsCasXXa for regards year.Oh,

of human our we benefits manufacturing.And using have high we advantages in because the for a proprietary et going concept. because have proof of of then fidelity finally, it's and of that quality, smaller selected the and the guide high efficiency because it forward enzyme cetera, its First,

very have program. exciting, from cells robust human data in editing AsCasXXa from We our Reni-cel

those and And So, then tool. with all a of non-viral focusing are non-viral on using editing reasons why delivery, favoring regards are using that and we nanoparticles, delivery to the -- specifically. we're

with from question next Zhu Wells Our Yanan comes Fargo.

trial. about Great. the just readout We're RUBY from the wondering mid-year

the patients And this any you with compared time think report Are of a you number XX bigger to-date. readout? certain like follow-up additional patients, at also, of have a dosed data on plus seems these to patient last going I with readout. much the XX dosed amount is

just So your total this wondering goal readout? I was the normalization in continuing confidence to of media the of show level kind hemoglobin

you, Thank Yanan.

number normal which this that yes, little that us and of to X of that, agree and also a a going detail. XX% of all natural is Before is dosed, treated passing continuing history fetal excited is much that would show pass said, and of more to the details, months we we're we've I'm but repeatedly all men you. to with about in bigger you range to we well the are correction on Baisong to and has have physiological threshold are past women not mid-year But a Baisong relevant, tell actually consistent expression followed confidence, to-date. anemia the it And disclosure. excess give the achieving see we patients that in XX%, only as some robust increased that of data hemoglobin with accumulation our above Yes, with I

dosed I Yes. absolutely to continue understanding is those XX We your correct. patients already mean, Thanks Yanan. patients months. and the coming in we'll do

year, the of XX then patient So, patients the have that. patients X by follow-up XX more meaningful time patients And be this will very middle be the the substantive data data release That's some the as in X we as and dosing can for patient a set it share published dose we're we we or the to period. really will now, these and and months mid-year. middle continue these and this we already longer patients be have going And that those you ASH we before over able to -- see, data year. monitor XX plus help, then we patients to more be data will why by of describe have year will in

exactly As pretty strong. you data mentioned, we feel is this set

we're point Baisong, and view from of about we ranges it, an X said between think worth talking efficacy you XX-plus follow-ups actually, And but of months. Yes. think, I reemphasizing, I it's

] actually hematological obviously a [ not robust about actually really but So total is we talk follow-up to is period in when robust our the efficacy durability. are just just not relevant also increasing that of in you outcomes, and number, but patients building set now, confidence data our which in time, now core as

thanks. Great,

Looking forward to that.

from comes question Luca next RBC Our Issi Capital. with

the the that additional the related Baisong actually remembered investigated was causality ASH on II Congrats any Maybe case progress. AEs I potentially tests. pending Reni-cel? was on update lab Grade the noted to being all polycythemia the poster of

you if partnering just any wondering just ex-U.S.? on on maybe quickly, one? update have So, disease then that update sickle-cell And any

will regard that Baisong? will cell our question. see partnering us with partner that footprint could take now, we we to North is will just as as keen a Baisong and So say, help future something as say And to upside. right global the in a that But programs our upside.With partnering, on first here driving And the to We be will development. global focus in sickle and are America. a that an thalassemia with I commercialization erythrocytosis, that look with I regard

parameter, total is patient's the including total Reni-cel elevation further has sickle this reporting, that hemoglobin That have patient's treatment. consider more then too, to related At the hematological transient months. patient And as a Thanks. -- in continues of ASH been will it's specific the of already we hemoglobin than that report ASH. normal for X time Yes. data, we the investigator continues investigation of and review and normal. not investigator the -- event the to stay And a

from Our next James. Steve Raymond Seedhouse comes question with

Timur Steve Seedhouse. this Hi, is for Ivannikov

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as provided to required wondering cycles the Reni-cel, then number the patient Have some progress. in that after the on to CasXXa wanted receive are are space? if Reni-cel. end, how relates of I any of on efficiency neutrophil you think the treatment, the products to And allows the compares I editing around with do bit it you experience what apheresis to you little back was touch shorter of competing for of that the on amount disclosures Congratulations a apheresis to higher engraftment? and seeing time a cycles. And

apheresis the engraftment, much, talk have to been on and touch with obviously experience very to actually very experience neutrophil with going pleased Jack. the of we ask and patient I'm Thanks which to date. clinical Baisong about other elements

you, Thank Jack.

patient mean apheresis very part I For a that. of important experience, definitely is

team As earlier, the the I joined, since the worked we mentioned managed and apheresis I with optimize process. protocol I trying and experts, to

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And about protocol data to similar that. we're follow-on the So we follow continue we very for studies. through happy the see as

This the your question-and-answer have Thank of our participation. reached We today's concludes end you for conference. session.

at this lines time. your disconnect may You