Wave Life Sciences (WVE)

Kate Rausch Head of Investor Relations
Paul Bolno President & Chief Executive Officer
Chandra Vargeese Chief Technology Officer
Mike Panzara Chief Medical Officer, Head of Therapeutics Discovery & Development
Kyle Moran Chief Financial Officer
Salim Syed Mizuho
Mehdi Goudarzi Truist
Luca Issi RBC Capital
Call transcript
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Good morning and welcome to the Wave Life Sciences Third Quarter 2021 Financial Results Conference Call. At this time, all participants are in a listen-only mode. After the speakers' presentation, there will be a question-and-answer session. [Operator Instructions] As a reminder, this call is being recorded and webcast. I will now turn the call over to Kate Rausch, Head of Investor Relations at Wave Life Sciences. ahead. go Please,

Kate Rausch

Good today Marci. review thank for you third Thank discuss morning our us results. to XXXX and recent joining quarter you, operating and business Wave's progress

prepared quarter Chief me Therapeutics Development; accompany remarks financial and Joining Discovery news update. Executive Chandra Mike and Officer, Dr. Financial and in Chief a Officer; room we will our third following call. of Officer, a and Panzara, the detailing morning website today Chief be Chief Section and release are Bolno, business the Medical results our This Officer. Wave's a issued provide in This slide Paul President this available release news Technology Investors Dr. www.wavelifesciences.com Moran, presentation Vargeese, the to Kyle for Head webcast of

we would Before from our These or including number in issued XXth, XXXX. Paul. quarterly We XX-K XXst, call during could forward-looking could today uncertainties XXXX you any the over Paul? press this ended turn now the December in report forward-looking statement that are for these those are revise that call the quarter remind like update our forward-looking reason. our cause of I'd subject that to discussions The to and on year undertake ended annual release statements. filings, differ obligation and report Form discussed our will the for on SEC statements. to to materially to for December Form results any to a XX-Q no differ to results include and in statements I conference actual factors described like risks actual begin cause

Paul Bolno

it you for our with an how are with our discuss building turn pipeline Today, morning RNA to Good will programs after which will of a then joining therapeutic AIMers. start us. update through Chandra we Kate. on financials. walk Kyle Mike editing will to provide Thanks, therapeutics and and opening remarks I thank

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Chandra Vargeese

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Mike Panzara

Chandra. Thanks

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will these turn all through year. generate trials to next across now over to CFO. expect Kyle We XXXX decision enable of call to Kyle? our I Moran, the three making data

Kyle Moran

as Thanks Takeda million quarter the which October received the in primarily recognized is third compared in $XX.X recognize of This $XX.X support million XXXX, remaining XXXX. the $X.X previously as agreement, in XXXX amendment third by recognition to million We revenue Takeda. of collaboration Mike. paid driven of as third in as quarter for revenue the quarter, as increase revenue we part the from research well to payments to our the from of related

we block editing in expanded our related million increased program were XXXX, Takeda $XX.X related goals our increased compensation since for investments to $X.X pre-clinical expenses proceeds expenses enable in an ADAR decreased current to ATM. million ADAR our as other with and Our October, equivalents subsequent off $XX.X driven platform, million million, in incremental related does million expenses operating expenses, offset $XXX.X funds close, million, our advance and at in total programs We G&A – million These including for discontinued primarily included compared the third neurology additional cash by as the same million $XX.X in the R&D $XX.X and was were not $XX.X an compared aggregate the million approximately This programs. third the ended will cash, same compared $XX.X expenses. and balance to quarter from to external quarter $XX received by third expenses partially last the as with XXXX increase were to to year, The year. expenses primarily programs, quarter continue compensation in last by time. to third million quarter the G&A $XX.X XXXX. security. marketable period as related driven on

operating will continue us cash requirements the We cash, of enable quarter expenditure our expect fund second our existing that into equivalents to to XXXX. capital

As a milestone Paul. payments, this Paul? undereducated now call potential back collaboration. to not turn over include the does any I'll reminder, or often

Paul Bolno

Kyle. Thanks,

proud our genetic advancing progress medicine. quarter, has made team pipeline our of This of I'm the diverse

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is Your of with Instructions] [Operator question first line from the Salim Syed Mizuho.

Salim Syed

Great, good the Thanks morning, for question. guys.

So there's can. if I me from a couple

XXXX. I sitting in appreciate Paul, here through So, the language November. around we're Obviously,

X, about the later? guess, you Huntington's or going data maybe Is how is the label, process you are thinking you. for of thinking trial? and and around the to could are un-blinding house take to open out ADAR XXX for those timing the can same for, How business you you therapeutic the to you collaboration? and development partner ADAR And then question there? given areas second disclose end that of hoping the in potentially Takeda you're just the to you collaborations Cohorts I come and with for and the generate that that guess, bit the it's are every question see X And the now the I'm I X, presume? editing CX XXXX. the like, then How data So clarify little street? planning us Thank plan XXXX. you X keep the in that even data plan -- and about potential What to of a more here cadence of in looking amendment editing and they're I

Paul Bolno

And we've got in Panzara. the to said across three year first. began we next data where get But CX designs, readouts dosing predict Obviously, the clinical we comment with start will I'll to underway adaptive it dosing trial Mike the we over yet can't and the as But quarter, occur. think different Thank you, these as into studies. question I hand given last nature first of cadence of the Salim.

independent clear committees I think review monitoring been about we data, those that we've say able what data. being is well to those to binded also ourselves, safety unblinded

said, open And changes any updates move we get let as we this, so disclosure think… I into Mike as detail I’ll publicly, to but we're around he additional material XXXX. designs will into for, the wants to impact, share study various

Mike Panzara

do say, recommendations committee, pharmacology and would including of that's if sharing next. them. what or this data would be mean, these would I No, to about that data would with who disclosed there basically it. we that indicate alter changes that captures there's program the I study these what mean, And then have with to you data, a I design to to I mean, back material the process comes be already biomarker were share know, material we've would

study, other that cohort you what's where engage to in has get each in have important expect designs amazing an particularly starting spend about to expand higher you can enroll more we about that which we to where the patients and think, into quickly. with really unlike application these adaptive CNS, blocks move resources cohorts in order know, there that of flexibility us about so target committee in in the to the both adults comes cohorts historical ability job and I the the get in thinking to done enabled in and of thinking how innovative studies, development to designs, the of has combination different the be that team is trial

is last make definitive that's we have And projections us sure our brought study updates one. next to so, I you the anticipate And run to that that provide same while five to view enable our based on we'll way that three. know, a pieces mean, all that across to we and the in gets the endpoint. up year. And you a

So of itself that to setting of in prescriptive disclosure and to even has study open data appropriate ways to running the unlike open or in label be point able setting an that XXXX. mind, get

update you any of those the the know, that means tends there initiated, be once the think are material of studies. I where So is there dynamic possibilities nature through can study guidance to set nature that

Paul Bolno

to ADAR just… move discussion, the part? business first that which on development equally questions I'll then is Any exciting. And I’ll

Salim Syed

Paul. you. I Thank No, helpful, that's think, Thanks.

Paul Bolno

mean, before, think, for I is robust the business correction. as a development way out I is this we doing vivo new place we second have one think, a And research a interested as area of ADAR shared of the the of in I I most think right. following So you're editing. we absolutely think piece of things compelling biology, webcasts I around data think, of case datasets

it on business deals always development So happen. deliberate they're it deals I'm discussions, attracted the going to that business brought. for to very guide lot in while And think hard development very because we're I expands to a opportunity be is has of us is to robust when say inclined it doing discussions.

us in AIMer we about robust we to to this precedented opportunity that are think excited in ADAR path case And know for there's with I expand the think application it's a GalNAc-conjugated bringing portfolio deliver, where, administer starting in a our GalNAc-conjugated you going to space. subcutaneous to the know, we that well there's liver.

a of eye, in in across the cells into I kidney, bringing business point, other whole have and we've we've flexibility discussion. back development on, we your portfolio human shared therapeutic think now shown of the brain, the that data indications as to adds in that there's host that

large other about So we indications, that CNS think includes and applications.

genetic you be discussion, ADAR the the planning. businesses, are know, post part But a discussions definitely think future I medicine. So our and highlighted domain of broad will around

into we So excited are back it. about bringing

Salim Syed

Got so much. it. Thanks


Lee is Truist The Joon next question with from Securities.

Mehdi Goudarzi

for Goudarzi on is Hi, good Mehdi This morning. Joon.

of couple a questions. have We

first a data. question to say, way your your color then preclinical evolve production competitiveness and that compared provide is of So please have the with some a costs comes and production Could it And random when ASOs? platform on I to follow-up… great scaling long came nicely you your

Paul Bolno

Yes, no, And whether you [indiscernible], five one that's, in administer it's successes it's it's to be administered. successes, to in easy beside take programs. a know, pride great or four four some see think, on hadn't harder question. I And we top point great well, of technically in history. [indiscernible] sometimes a going back I to one think, able in were And those that successes to the other in that been plus way – or five our systemically of

scaling fully of successful stereopure other the oligonucleotide. modified was systemic real of One a production applications

cost at And goods of a so commercial random for with we be stereo would of molecule. were scalability a [indiscernible] that poised par on

that for scale the experience, time manufacturing that And ADAR smaller so partly capability our and so capacity and apply silencing on actually about again, equal oligonucleotides through across to think, interests as we one.

GMP So scale have that take we we also our transfer I several the to, we that robustness larger facility, can importantly to manufacturer internal that think years and scale. it of a been show ago and the built, was process, have as commercial that experienced we we able can to manufacturing think to about we GMP

So, we manufacturing with certain. are is oligonucleotides now pure on comfortable that through par

Mehdi Goudarzi

question be next bit Awesome. looking And my forward. a would

comes formulation vehicles. ASOs it compatible formulation to Your specificity, well? of be cell But LMP if would new or type chemistry a this as any need do with not PRISM

Paul Bolno

be to And was date, that delivery within the types yes. say the those where short And short and really exploration our focused is, answer driven the we I because accessibility. cell on are

the shared distribution across earlier sites. central and nervous cell administration after have the in As HP, system broad major dose single we in presentation Mike intrathecal

As GalNAc all Chandra without, across vivo both brought have shared, ADAR distribution with in with we and types. do

can I So, that's we where be interest. strategies may dose you case and this where to think cell type about of specific think think a I you a the delivery of GalNAc, give targeted is smaller of know, single

think interest. right I and distributing and lipid intended one of not active But areas it of of space, are those been cell for as moving the think and exciting the to GalNAc with for I viral nanoparticle. areas just that we particularly really think and to oligonucleotides into are has -- for requirement editing into but the as that always but without deliveries, without the relates us uptake, of exerting areas the are compartment vectors is effect, the our always cell

Mehdi Goudarzi

any much. any just programs. of may tiny criteria very Is question. for opting another If status you make for I the out with there Thank

Paul Bolno

program. opt and criteria outlined ALS-FTD, earlier that three for set in is around XXX I the that built are HD or a effective there of So the XXX three program that

So so that's of prescribed in delivering but hence triggered us XXXX have as mechanism. our level those is all payments, They think clinical proof for to profits, them. executing demonstration also gets associated with addition important and in program of NG it about and But was all on and are events. opt it XX:XX They we have data. apps milestones the on

Mehdi Goudarzi

you the for Thank question. taking very much

Paul Bolno

Thank you.


Next question Matteis from Stifel. is Paul with

Unidentified Analyst

a this question AATD Katie program. on for just had Hi, Paul. I is on quick

candidate announcing development So year. your next you're I know

this getting the into Thanks. entering I clinic. guess is that, Beyond what program

Paul Bolno

[indiscernible] report.

So you I relates AATD, a clinic as step mean, to to as it first is candidate. the pointed out, obviously the

we'll into be so that the are it's And like you that and this earlier, said providing of features going program, around as close. more as guidance XXXX,

feel we You confident on know, potency. really

saw candidates, also the we early that how call to we And want terminology think see I has as said, constructs. Mike We have always company when durability every is. think long dosing candidates. with different we I around frequency

I candidates. tolerability build think we early our in

Now can that know tend the announced go that clinic, to have we the program we you we that we distance. to a when that bring

AATD. the be about up to give clinical that announced translation quickly accelerate AATD first working best robust think criteria and working into team do excited potential will that, to program us bring hard well that credibly we the class, forward our is set I are think that So the potential guidance, that. to we're mean, aid to that in I what of But further go will it

we and on conjugation ability -- As are we with about relates being Because down it of that other to the again issues. both that working other coming think poised behind aimers because

to I sequence to will more get we think portfolio. that be the about XXXX the into growing how provide AIMer of of as updates think kind

But builds to about forward As around ADAR [indiscernible] we excited share unconjugated as it founder and our well. editing we do shared, programs expect more as we're GalNAc. bring Hepatic and

I know, those updates XXXX. more as think be we think about you would So,

Unidentified Analyst

Great, thanks.


next Your RBC as question with Luca Capital. online Issi from is

Luca Issi

the Oh, so ones. great. taking much on question. two Thanks quick my Congrats for progress as

So one if Biogen They're first maybe saw like they're a SODX. takeaways ALS how maybe few that program pipeline. a obviously data second, key the a lessons Thanks discontinue the different Ionis you some missing use approach endpoint. of the going any there's But from wonder given collaboration program you going back weeks after obviously to of learned that and on that is very, and set, to wondering very primary expand from why planning Kate and your And so we to your then much. earlier the more on decided had can you're to maybe bit forward. key

Paul Bolno

the the I'll I'll And second. take to first let question. Mike back Thank you. then come

Mike Panzara

thanks that. Yeah, for Sure.

different you this that very drug main captured, think of all different I points very very is So first and the clearly, targets.

different just one another, totally one place, a targeting mutation starting in different. for versus totally So indication like, oncological

show engage I terms I first in thinking we that what think can show mean, of did we -- ASO that of learn? they I did least all, I can mean, think can they that, targets. what I at didn't they get patient was of I design really modest there helpful ahead. Also, engage target. in elements population the did did to are study are a of but the sort It think target. they and effect, that

design predicted study think modest their saw some I in study the leading effect of some -- they that outcomes. earlier could issues some given there these maybe have were

for optimization optimization goes that the be is that to have to that CX we've basically And to target what think that with you best to you tolerability. then, we the in it what able So enable candidates And for and presentation, need our I done need distribution, design your earlier engagement. said to that's program. to target engagement with studies

mentioned, as the clinic. the positioned taking I So and seen really way the given preclinical we're into we're we've well, data it

what that. to turn I about say it over And that's would Paul… I'll So

Paul Bolno

which robust characterize demonstrated studies get do to potency to shared CX which just biology importantly, a life durability, preclinical effectively, as Mike, by first. our echo And way there. we think And believe really Yeah. into as running to throws efficiently in it we're target I that

-- where is about there's our position. think I we're program excited So,

collaboration As it And I pretty that’s expensive. have the relationship think to relates it's that it's to we a Takeda. important with Takeda, remind still mean, ongoing everyone I

sometimes think collaboration, I the partners. feel they people Takeda are amended and like that still

that advancing. partners are a we on third is So and program clinical three, two programs

collaboration ourselves underway. there is remind a So critical think is that to I it

discussions, useful a of Anything it. amend there's to decision the lot activities. why think, I

a we what desire think to doing. to accelerate, continue I have we're

speak to around drivers have desire And We the budgets field and they have it are. in a CNS. indicated can't some where our of to I

decide has franchise. programs strong move with excited is the key partners. And and CNS the to to are to think XXXX strong our I into together. that them evaluate Takeda how political we're forward we move

are So, Got forward. still think agreement Thanks futilely, so very done we the we much. it. simplify we've can are is and we I that partners, streamline so much what


[Indiscernible]. Last the from is with Manny line Ferrer of question

Unidentified Analyst

a questions. taking go for and these previous, and are guess tough Thanks for to would a dataset? you continue that HD HD HD better becomes not to appropriate or see on which target that investor at many ventures throw And to point this for our use that How consumer that do this looks others of to there's capital? time guys. think I oligo you into snips. DMD wind we therapy, Like, not of after. be easy next update proven proper obviously results you've take, targets should ones moved continue totally your have apple money it from in the of pursuit, bytes you philosophical down Hey, that we particular from one. in a targets do sort of more Obviously, the HD different throwing

Paul Bolno


one, So money. we I throw don't think

and money, I in think data invest invest we a way. driven we think money I

this durability these. vivo others and data and the model. reason HD moving in that one, only with that relevant distinguishing we're support meetings to have against think backbone your the now subsequent is with presented PN we allele chemistry into data appropriate preclinical data specificity point, We clinic, potency an So demonstrates program demonstrating and vivo within in to I our and running the

and going think HD conclusion have and I don't an in support. approach for believe its to then to that we're experiment data as frankly, we do decision. run the Do way, a we think driven that or get data So data benefit, I driven a that we

it we FTD. high as the as base for ALS needs for CX and CX, to a medical and again we preclinically, a Secondly, design therapy. requires out robust set preclinical and Again, durability, as relates of most unmet there our have we look data that believe home do area program second

to not was we we're able in that unprecedent, mouse And change be down see they double dystrophin in in study. knockout a to mouse believe then program that's phenotype, production, best-in-class we the have survival that like run NDA, demonstrate, to a in actually there. And we going going we're So do the in just run thirdly, that an DMD, substantial. to data

And get test the so we're to going again, to data. going that we're

to is progress you're what of very make say, decisions, are data And aligned very as GalNAc we're a that a year deliberate way, do ahead and I those to is think nuance silencing ADAR area And really GalNAc-conjugated peers targeting the I and gives three that decision program new conjugation think our the on biology with little data saying, driven next decision so us or progress in gives new a say, we can I then it's I us what move a steam And will ADAR. advancing in we to decisions us area. answering bit us moving say. and it molecule. decisions, these to think are around think much to a It gives full very we different which a really discussions need a look, different way a investment to so fourth important, took gives

questions to and [indiscernible] pre-clinical periphery think spectrum, in by across of decisions the portfolio buy well make able that And regions system editing And So I work in that's across muscle. next that on answer meaningful I skeletal investment as in harder over central ADAR going think the we're the nervous to and going XXXX. AATD. important both with of to as program answering coupled be question, your year

XXXX. capitalized that about now excited do being So I'm to

to answers I think we're platforms. going to get pharmacology really important programs and

Unidentified Analyst

that's Thanks, guys. clear. Great, crystal


call the over Dr. Bolno. to further Paul at no are There time. turn this questions I'll back

Paul Bolno

Wave day. bye-bye. great for employees thank joining updates. Have to hard review our XXXX a the everyone and our this for And their to commitment corporate patients. third to work morning quarter you call Thanks, Take care


conference This call. concludes today's

disconnect. may You