- Earnings call transcript Q3 2022

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Operator

Good morning, and welcome to the Wave Life Sciences’ Third Quarter 2022 Financial Results Conference Call. At this time, all participants are in a listen-only mode.

As a reminder, this call is being recorded and webcast. I will now turn the call over to Kate Rausch, VP of Investor Relations and Corporate Affairs at Wave Life Sciences. Please go ahead.

Kate Rausch

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

and are Joining this Officer; Development webcast to the this Moran, The in Paul room morning on Officer. Kyle Officer; Investors today are Financial our release me Bolno, Li-Kwai-Cheung, available the and website President Section accompany press Chief Chief Chief and Executive Anne-Marie presentation www.wavelifesciences.com. Dr. of issued slide

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

Paul Bolno

for would start this XXXX updates trials program. three deliver our our ongoing the separate us. then therapeutic CX-ALS today's modality updates well I year, target has WVE-XXX provide Anne-Marie Thanks, the we data And Wave. and potent Kate. including target development FTD introduce engagement delivered finally, most pivotal Ann-Marie with for financials. discuss first Officer, our durable start and through we and advance Good Development year disease, most highlights of as thank first and will our our data advanced of morning. our as clinical Chief Li-Kwai-Cheung. discovery will from And To on said, been joining with study data for date, data trial engagement candidate our translation some all call the you updates, recent novelty At and indicating from an Kyle will recently, results chemistry Huntington's clinical clinical platform. entirely SELECT-HD clinical supported two from WVE-XXX. showing new these validated the nomination. sets with we have Both and allele a selective

therapeutic to the In AATD. data if this with development event our the have we first our September, Alpha-X AIMers Muscular new our time efficiently track website candidate, platform. Chief WVE-XXX on trial or first harness the tune in PRISM to in also are genes for clinical presented RNA data a preclinical antitrypsin In Officer, later ADAR Technology WVE-XXX event. spotlighting we biology editing and oligonucleotide of Vargeese, you using forums, and therapy, balance remarkably quarter. one selected perspective treatment the By from Chandra achieve data presenting the deficiency week, address durable enzymes, siRNA who to our to the held meeting and these I we other hope to designing of setting having were by need a both AIMers and applying manifestations potent joined AATD, be to liver will clinical chemistry. coming on filtration. Dr. into permanent to last the month. chemistry upregulate this disease, lung highlight landscape our the was models. without unique treatment benefit unmet as to continue recent and This the replay such Hobart, and with shared other and scientific at other current RNA of oligonucleotides Preclinical deliver the third quarter, you biology alongside live broad Duchenne We in virtual of Oligonucleotide a backbone at OTS of missed WVE-XXX. [indiscernible] XXX the existing invite for open-label includes At the we PN the clinical approach the genetic on symposium the were or Dystrophy we of our platform demonstrate versatility editing applicability Therapeutics entirely unlocked in in Institute well-known Wave's We expert Oligonucleotide Society innovators to special of that chemistry. Kyle address This results that for AATD

and in like the the regulatory in briefly joined as within call beginning development turning this those clinical particularly to to to disease. to our listening her Before to and Wave her turn pipeline. with role time over in. I'll will Areas, advance Neurology innovative Officer, be of would introduce take I the that, Anne-Marie Rare to as Therapeutic at critical year Senior Anne-Marie, and the activities across Development September, as have experience deep Chief years compliance know Program we Organization. very at a with through Roche, Drug as And Huntington's now Disease over increasing her spent call commercial Previously, expanded Leadership in Development as We Anne-Marie, Global eight our Clinical Wave were Anne-Marie. responsibility well we positions to excited leadership Development in she Leader and holding Global this Wave an our

Anne-Marie Li-Kwai-Cheung

to be across Wave this join to of morning this founded exciting the at pleased program. things. share on decision speaking we development with our I'm and to was year you start that all progress My three Paul. made Thanks,

study HD DMD in on our shape refine from on significant dose clinical data and Successfully clinical frequency shown studies to we ongoing development. level of and the slide, our oligonucleotide and innovative drug of quarter. areas and points the in need pipeline our way, adapting company's to dark ALS robust this are approach at each our key First, sharing track both blue data the three advancing to we another the promise trials the along and data this platform, trials, novel third, of and FTD second, on yet deliver focused trial, from unmet

to year. to In our addition, in development candidate advanced begin clinical program AATD, have in we Wave to for positioning progress genetic wild-type through wild-type disease a of disease protein of three. which affects disease, preclinical the to Huntington follow. progression. successfully accumulation to the protein that development development and the next And joining the I'll each mutant overtime, studies monogenic IND-enabling describe Huntington's been leading involved Penetrance therapeutic huntingtin initiating toxic fully the us disease that WVE-XXX, devastating beneficial is Wave prior I've the I'd on for deeply this autosomal overcomes HD. criticality like dominant entire focusing I've the several appreciate therapeutic years of the page past the start protein to experience, brain. come past in and as for approach effects slides by the and preserving and

formation adversely patients. potentially and So of survival protein to of the at is it's huntingtin CSS essential roles essential Huntington re-absorption. include nervous synaptic and and to these and Dysfunction transport, and cause in central are response be cilia, in function which the expected promoting wild-type and toxic is the system, several could worsening understood any protein that could regulation of protein impact in neuronal regulate placed critical pathways mutant and these WVE and

there open begin compared And knockdown new given remains XX% setting of of question in patients on already it life safe-level any HD. whether knockdown less healthy individuals. the as to HD wild-type an wild-type huntingtin to So with is in huntingtin as

take able Our question, formal With to clinical in target. procedures that in are invasive only that clinical It's one without with encouraging and this data PM CNS engaging selectively surgical is suggest without the we're systemic development. results initial WVE-XXX next-generation able therapies. program answer only dosing, is chemistry, we targeted board our our delivery incredibly exposure the advantage to indeed we as Allele-selective therapy of

we models target development the of reflect on As selective was our to and relevant potent WVE-XXX engagement process. demonstrating path core preclinical in the clinic, to

you the left, the steps can we On followed. see

pharmacologically First, with demonstrated target WVE-XXX the dose using dose optimization. prior guided selection. the result to further, model this for able allele trial in we engage along were the IPSC predicted start target transgenic to selectivity with And and XXX vitro mouse we at primates, data does us CNS And humans. in likely nonhuman to levels to tissue engagement allows BACHD active these from concentration from was in neurons

On the the approximately shared in in our clinical SELECT-HD day, in you compared This the CSF from as XX% XX% update data baseline data see we and patients can from trial first right, with mean in to September. mutant HD Average was was XXX per the Huntingtin placebo. reduction polymorphism. SNPX

appear addition, over levels wild-type the consistent with selectivity. Huntington In same protein period allele

at were the For cohort. day dosed And of these pulled as analysis. XX none the at XX reached milligram the the to analyses, as a patients reminder, from had milligrams data not included we XX time XX

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first increases this in the resulted to mouse, concentrations Moving PN skipping overall from and were the mouse and quarter. muscle clinical DMD which is double and will model, typically These clinically data Preclinical trial to skipping in significantly muscle primates in in first be potential exhibit in both respiratory for severe both non-human and a our improve and with chemistry Exon plasma splicing modifications. over fatal improve tissue doses the boys our data PS/PO knockout associated open-label XXX% our containing phenotype. clinical in mice function efficiency data survival to first-generation compound. oligonucleotide PN with at amenable our concentrations muscle knockout biopsy improved a relevant expected with also This NXXX, XX backbone support

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a type is all of dose full following in single levels. over the of dose the pharmacokinetic explored days period illustrates single concentrations XX can four Slide of XX from study data label, IV phase doses study. the the NXXX at doses As open we of share

results other followed with every profile to biopsies. of exon adverse Phase NXXX pharmacological self per COVID-XX was SAEs All kg of with NXXX trial and weeks to levels our data important moderate the compound WVE-NXXX dose following in us receiving a X distinct meeting up X Our our plasma to and leads To-date, suggested first-generation we reported tissue recognize were set been in reflected. a is criteria. suvodirsen animal about with compared hopeful is generation the per very this with per our are interest exception uptake compound. also to with been kg after higher as of week have initial discontinued the The week be as initial from will concentrations with suvodirsen. XX and with These It's concentrations mg demonstrate cohorts of mind, data the achieved of – NXXX have Remember, those chemistry following overall. of report of dystrophin and XX mild have to by administered NXXX data measured data improved approximately the DMD single-dose exceeded PN been of XX And kg passage those that quarter. three oligonucleotide muscle boys greater make than amenable we the PS/PO mg a There to [indiscernible] appears no confirm XX events per cohort kg in earlier unique of are and X muscle mg doses. one Plasma the treatment, protein this now X. Approximately, events top that concentrations the other mg three dosing, cohort no who first-generation and infection. while clinical and by transitioned skipping have from of study that chemistry compared stocking this well-tolerated. first expect now to additional includes as portion in fold

from signal X% approximately and possibility to these on localization, Patients us promise quarter. positive program Exon to XX that course, pathway. Exon adapt explore being technology to cohort expect tissue is mutations the Dystrophin applying approval data would accelerated expand expression and and the up comparable population. NXXX Exon measured, represent Of and the potential dystrophin package later also activity XX speak the protein and skipping share and skipping also of with this boarder data the about amenable to XX% underway of oligonucleotides concentrations a an The initial to this Positive to regulators patients, additional is study to we'd we DMD planning with opens pursue of is splicing NXXX the possibility. drive

the would other PN As Exon drive in enabling also from mentioned to developed data previously our expansion we expand only initial leverage not oligonucleotide, cohort with strategy. positive our to but exons us XX modified study, work us splicing accelerate would multi-Exon

with Moving trial continues FTD. CXorfXX-associated clinical cohort. I'm report ALS in to pleased the WVE-XXX our and multi-dose to our that the FOCUS-CX, in patients for dosing

a following this was on potency where our reminder, expand we April the last a durable demonstrated to portion effects of multi-dose dose year, of dosing. in As just Based of WVE-XXX. in engagement update statistically we meeting, and shared our decided of robust, DSMB to durability it the and on-target evaluate FOCUS-CX first target single quarterly significant clinical

engagement Slicing mechanism, we established Specifically, expect the the extension will to move This the sets first candidate Silencing would cohorts half candidate dosing, a cohort. single-dose XX-milligram this this of past compared left fourth And ability of with this potential enabling also support quarter, XX in first to our the development we our In the we XX-milligram data to was a after on opened we trial is dose and from be model Having data from patients quarterly to this to to demonstrated a we In all durability WVE-XXX. is to there mechanism, and in weeks, disease. WVE-XXX to additional panel, patients, comprehensive RNA AAT patients to FOCUS-CX the our field. we for this preclinical FOCUS-CX open-label year which WVE-XXX quarterly September, hope to the opportunity more later in potency well-established serum for a the provide move NSG-PiZ in innovation, dose expect control. XX-milligram update XXXX. treatment best-in-class share we model, the regimen continue With and also move as cohort. efficacy with editing shared WVE-XXX next PBS cohort mice a increase AATD. that seven-fold attractive receive our a target we've initiated quarterly And through in

the protein, middle elastase which track Moran, the confirms Importantly, will M-AAT are on ongoing, panel. as to XXXX. I measured most the call right functional WVE-XXX and are IND-enabling wild-type the that our spec data now we in in activity, to are was by panel studies for generating Kyle we submit neutrophil as turn over CFO. shown in CTAs

Kyle Moran

for amendment period third approximately program $XX.X million XXXX. and compared in research General to external HD the decreases year for loss increase administrative increased quarter million three programs. XXXX, and development the to in loss and was months the related ended partially the and primarily of the third same offset million to as CX clinical for quarter were expenses, XX, by $X.X our XXXX, same quarter decreased net of This to the of in and expenses external in expenses was other general as million the a quarter. to Thanks, to and share-based million for related the expenses preclinical due we to ended in declined September same year-over-year revenue XXXX. million, the $XX.X our -- as We $XX period million period XXXX. reported Takeda and Anne-Marie. prior cash, $XX.X other XXXX, driven decrease expense primarily decreases $XX.X compensation quarter in $XX.X as of equivalents expenses. investments. other compared primarily in collaboration $XXX Net by administrative third the due year, third of short-term was with We in in as for compared and million ended revenue compared well $XXX,XXX in quarter R&D to decrease in to the the external last cash The in expenses XXXX,

remains guidance. runway previous unchanged cash from Our

cash, equivalents XXXX. short-term will requirements our that We continue cash expenditure and expect to end and by investments our enable us to of capital operating fund existing

payments As now potential cash Takeda opt-in to runway. milestones a reminder, or the over we collaboration do turn not under our call back any I'll include Paul. in our

Paul Bolno

and the first and We're the Kyle. progress excited of the date our potential demonstration biology clinical about of are all for of we WVE-XXX Thanks, the AIMer’s, unlocking With generated platform data new our diversify applications editing. to RNA pipeline. is to

Our RNA diseases. hepatic CNS,, discovery disease including multiple editing renal programs actively team is and areas, in evaluating new

which require do have the As in unique a AAV, our not types. challenges LMPs or AIMer’s tissue reminder, delivery

deliver XXXX on our in expect updates We more discovery to pipeline.

We strength, enabling us platform diverse with disruptive to a unique oligonucleotide have target multimodal biology. guide

with are We Partnerships expansion translation of unlock our maximize program pipeline delivering collaborate initial remain a value manufacturing. to broad with our platform. of our of pipeline key support GMP partners share and for value to We our and diversifying clinical intend in priority editing. and vision who platform, ambitious the our our

We deliver for including work, proof-of-concept have process to begun potential development on customers. GMP manufacturing initial also

We are track goal and to with expand well We quarter. the accomplish out to this clinical positioned year three expected on set updates this this are data DMD deliver to efforts. these

positioned deliver HD we data continuous including into and of a flow our are additional Looking FTD and updates, nine to nine data XXXX, ALS from trials.

positioned into WVE-XXX move development, RNA to which the also be clinic. therapeutic the editing will is first-ever clinical We in

with execute that, call well also up milestones. are we'll the And capitalized on Operator? open We to these questions. for

Operator

from question our from Syed Salim And comes Mizuho. instructions] [Operator first

open. now is line Your

Salim Syed

Good the Great. congrats for progress. color the much for me. Thanks on couple guys. and so morning, A

data Paul, Huntington's some with York in possible, obviously, year, readout DMD New both good FTD, you've the ALS As in we chemistry. head just into this and the had

your get what thesis number from target related I if dose getting had a that to would about would we've any or be like as to to a close. otherwise, it that, is guys, is given should quantitative speaking, lower? I FTD conceptually of quantitative preclinical obviously open have we something just engagement derail thinking clinical, And in people are bogey, translate just Just about whether label, it's we just you hear ALS could and risks to love then there the biology thinking good sets. today, you. X% both the HD or mind into here data DMD, are the sort that as the that again, data Just to a Thank possible. bogey, mean is or that's love higher for for

Paul Bolno

I'll your thank first Salim. but a with made start we you, medicine, we and any potential risk. extraordinary develop question, there's always a Because time progress, have

predict So that. was I not the extensively previous phenotype attempt agree behind with identifying to to in model important to particularly with in we’re and experience advancing was our models of dose. us, Suvodirsen, NXXX What setting animal it

think dose about XX dosing now you CX consistent a mgs our model. across that's to, modeling studies. we I doses per aligned up those for established terms we've with of silencing of today, starting preclinical referred HD as seen clinical how at looked establish as parity to we shared And in with our we've we as I thinking think the kg, paradigm

So that has optimistic.

a target into remember, is your transition to doses that important and establishing after it's with that. think goes subsequent dosing three I this numerical question we are second this around

other therefore, to period And in say, translation studies where like have lot we'd to tissue. concentrations other exact an distribution muscle smaller the different. laid than We'd dosing the is the haven't gone. than the the of see to like target out see we So a

that following recall, Association weren't of Muscular shared meeting cells muscle getting those you drugs. to Dystrophy we at exposure If the Suvodirsen we

in translate as then like exon going and looking And we'd be as skipping So at to that see be that study. important. well are showing we'll dystrophin, the to

to in numbers, produced. positive think being out, as the you would frame I where a have pointed setting we be short dystrophin exceedingly time this evaluate

as superiority be see forward. expand both power from alluded to other study extraordinarily what well back, to of encouraging for patients then to skipping oligonucleotide I is do think really a Ann-Marie as to XX driver exon that the path numbers, we would the if which call, number those significantly

that's your So, I I hope think to-date, the and I target answered question.

Salim Syed

helpful. Paul. Super so much, Thanks

Operator

you. from thank our And next moment question And Matteis comes And Stifel. Paul one next question. from for our

now is open. line Your

Unidentified Analyst

theoretical, color of had it? recruiting or Hi. it's from This for ADAR on safety bit one but Thanks with side, makes on endogenous on Paul. functions. would is the I Thanks downside have James guess, you it so you be about with great on those maybe ADAR know our question. comfortable risks just functions It away just from the any taking I leveraging a [ph] And any actually. much. what and for endogenous there associated little we away what its to hear thoughts more

Paul Bolno

the question, aspect preclinical something lot both of great It's for the Yeah. well nomination in running as spend a because we candidate platform it's ultimately XXX. obviously as a on time

meetings, have and to endogenous an does a Speaking have these ADAR shared aspect, transcript. the at platform we data on editing function

And whether And had were we're we function. we so looking capturing add its at the transcripts exhaust or ADAR to doing realize on to done from not inside that it normal show early multiple cell, from the able a studies preventing you multiple itself that what a within can therefore, and one is aimers from look within we not at was function. enzyme the applying cell of

your our development portion kind the of spent making of think ADAR really is of understood spot something I So studying point we the and capability, we sure on and well. platform early

we've into into program And development non-GLP program it moving confidence again, we've the done obviously, as good our in was forward. different from the As our of our candidate part in lot that. our That's development. tolerability but safety a how important translated this and nomination point, seen companies that ways, of at embed just we a safety into translation And have are then nomination. define part platform studies important candidate lot that, of safety an XXX, of

the advance Next submission studies steps, obviously, we're a tox engaged program CTA GLP to next into year. now in

Unidentified Analyst

much. so Great. Thanks

Operator

our next Lee Joon question next And Truist And question. Securities. you. from comes for one Thank moment from our

Your line now open. is

Joon Lee

our tominersen our questions. to her clinical taking for mutant to the given for What will duration And Roche. follow-up show duration I'd be with I will follow-up, love for sufficient hear thanks allele clinical at And what of experience like follow-up. percent knockdown view? of a Anne-Marie's of be benefit? needed have that Hi, benefit in a

Anne-Marie Li-Kwai-Cheung

Thanks.

data that reported the into already the range is we're move knockdown selectivity. seen initial as XX% the this be from in of to the today XX% study. As aiming we've about phase And multi-dose allele we to we of what replicate we would with

Joon Lee

With that magnitude of knockdown, how long separation do you a think see you need to from follow the patients to placebo?

Anne-Marie Li-Kwai-Cheung

So, within obviously, a would months something progressive HD you slowly disease, fairly is to but expect year a to follow-up. six see of

probably we'll phase. regimen on So what see multi-dose following based we’re the

Joon Lee

question the more Great. you to focus for trial? patients are aspects? of AATD, the the And or types the decision your And Thank looking want for does lung to you impact know how liver in that the next on you.

Paul Bolno

to of the Yeah. we and editing at protein, the very And interest developing we levels hand both. infusion. about the I'll thinking But program looking are the IV can first to to in really plan protein from to develop be Ann-Marie. development happy measure it relate and think it that plasma ultimately, beginning the as us it's obviously in opportunity Alpha-X to I to back the important

hopefully, look able the be at embedding liver into aggregate. biopsy decrease, also will We be liver to study to and that

to of about, that and study, enrollment be we patients get the the together we patient as as program And aspect the tend as those so progress into starting we progress we'll The advance compendium the ZZ liver. then the think that with disease pulmonary building it. patients

and from piece important on, important biomarker-driven that of less to outcome. us to in the ability terms follow period functional is to secondly, models. study expect. patients significance one, of those initial answer we more being of questions. models about forward That the So and will human able about for platform And translation the the editing early two the continue in Obviously, to preclinical a see outcomes from perspective, then

Joon Lee

you. Thank

Paul Bolno

Thank you.

Operator

Yun comes Jefferies. our And And next our question. from moment thank please. you. One one question for from Yang moment, [ph] next And

Your line is now open.

Unidentified Analyst

you. Thank

Takeda. the enough XXXX milestone So cash any without you current mentioned from is potential through

you editing. question So Huntington prioritizing are RNA the based the your been number expecting question second a are what programs? milestone? is, is, from quite And Because payments that would XXXX? your in in have receive event be given Takeda now, disease could one development cash, you And limited you excited on if thinking milestone about you about

not prioritizing conservative program the Thank kind cash? you. why and a of So

Paul Bolno

question. And the not are Great unrelated. two

a collaboration obviously, ongoing CX-ALS and that Huntington's program. have we productive with So that's FTD Takeda involves our our program

advancement So profit split advance to those XX-XX which collaboration programs continuing with potential to have and that. exactly triggers we decreases and are your We that opt-in at for drivers a split, point, that. associated of expenses the opt-in, also series And a those being milestones R&D as look our very. that has

don't To our runway into that's So how we forecast we're call, the thinking about that. statement. Kyle's point earlier that on

success those and milestone in payments. into would be potential often those clinical payments on progress tied central any programs However,

at those as So in we programs. balance that look

meaningful Secondly, said, manufacturing we're our central and as continuing to capabilities. do work, expanding on really we building important out

that internally And additional resources. potential so we've in and work been helps doing bring that support

you've excited AATD, to Wave year. work next hard And placed anticipate that candidate have and year, that. past in relates with seen investment a as prioritized the over we've bringing the about the it. are it now along clinic this of As the team we into developed we at And clinical

we've prior activities fund forward, engine non-dilutive other yes, on in with business sources we are to to And as as from of focus to look manufacturing, calls, and and work capital we've capability in. built to payments additional development milestone on the inflows platform continue build bring our on experience editing said coming partner. existing I our this there from coming So potential think cash

Unidentified Analyst

question? Can data has Takeda's point I the opt-in? any Regarding there event ask you opt-in, to one that follow-up partner is

Paul Bolno

are are proof alluded that mechanism. having data those as with are think we discussion to that consequential I There sets in around drivers Takeda. And upcoming, the data of Anne-Marie think

Unidentified Analyst

Thank you.

Operator

thank And from you. next RBC. question And our comes Luca from Issi

is line open. Your now

Unidentified Analyst

taking this for congrats And Lisa for on quarter. on the our questions. is Thanks This progress Luca.

are XXXX. you in a the the on you couple main the meeting novelty longer I would regulatory just they CTA with us a Thanks. probe follow-up. And your color ADAR experiment? a US? off-target you for is appreciated. know the filing FDA the there do But Any think what Have where much you bio-distribution will are given GLP remind the strategy pre-IND be more looking editing, areas Just AXAT. had a wondering, what And I can tox, have of technology? flagging FDA? for editing Are

Paul Bolno

think it I number But she's the the light of editing and on hand think within particularly with news really the start transcript. editing chose that think it's in capability of I then I'll edit reversibly a Anne-Marie I we why the because RNA question. of suited answer reflective to last editing space, flow part best DNA, with the to a

so about coupled of And as are But benefits very of take the we AMRs manufacturing think I'll let think discussions. with Anne-Marie important our progressing both the I that regulatory conversation. into regular

Anne-Marie Li-Kwai-Cheung

tox of of just that We're authorities. we course, GLP to, have the our instrumental progress outcome GLP as initiated you've in the study yet And development. studies with interactions in with the be that tox we not we based the want see. suitable we with regulatory our will already have to tox profile, it on in have hand, of concert discussion continuing a are which what the But a makes the for data assured regulators we alluded non-GLP

Paul Bolno

we're with when these think or ADAR. oligonucleotide increase are the oligonucleotide that developing mechanisms conversation events we're in I go to I when back using analogous mechanism an still original RNA to way now another same think is again about This the editing, to theme just about going have RNA about with using to just RNA very that talking we're also in trends approach. that like silence that regulators it's Much sRNA a engage guide important or RNA we're therapeutic XX to talking we're to stage splicing. with Dodge an endogenous

permanently. So, from the to target with different protein very approaches DNA that where both coupled edit you're other inserting then guides

So, a it path. different is

Unidentified Analyst

on That's question helpful, one AXAT. maybe Anne-Marie. Paul, And and more just

that We approved seen serum have they a can recently announced biomarker. Hiberix get in on

maybe what was about And to how own news? are implications you your wondering Just your that reaction for program? thinking

Paul Bolno

carefully. very and we anticipating else, everybody watching like data think I mean were that I

it the was to affirmative are that this us concentrations think plasma I important. protein indeed of

potential that, as path. it open a I watch does think we up

to think program. only that's of our I side that one remember

think So have but liver complications. we the also patients helping and is how that aspect replacement lung, potential about these

will so but so set to said obviously, encouraged be level, both we aggregates to I protein really that comprehensive, a our we really in clinical giving that data. And driven can addition address earlier, liver think therapy as path they that's by single ourselves

we're the is realization generating protein that With protein. endogenous the

the The infusion you this way is give there steady to to you What protein. phenotypes and not when having protein. needs we're levels degradation of the them establishing be So wild-type a the patients we're really the that patients and and allowing protein at then measure, DV this body end protein to that baseline the state. synthesizing for remember, expressed watch just a looking increasing to having a they're manufacturers

So, interesting as watch. encouraging to follow we and obviously

Anne-Marie Li-Kwai-Cheung

taken action and would say we support of this expedite to I is any diseases. regulatory great that rare that help patients And will field, kind for for therapies the Yes. with just by regulators precedent patients, great

Unidentified Analyst

Thanks for taking Great. questions. our

Paul Bolno

you. Thank

Operator

last comes thank our question. moment question our And Foroohar Securities. Mani for from And SVB Anne, from one next you.

line Your is open. now

Mani Foroohar

Hey, guys. me for let Thanks in.

that? moving in interpret should as a that interpret that clarification. quick if about speculated you On more how differentiation program, a I -- do skipping? head-to-head you're about superiority you endpoint? to to should not, study talked is adequately that powered versus show existing to And with plan guys Just larger exon a possibly study your I DMD Does agents

Paul Bolno

clarifying Mani. you for question, Yes. Thank the

not are programs planning not have on with we that on we're had running don't -- thought on do, head-to-head study approval. we no, a to planning So having and

the substantially programs, well we I support think study where been and that, accelerated assure from of deliver approval design AIMer want us set programs have terms around do that other as potential for bars is superior to discussions is powered in those with know dystrophin key the as to we have to to both on pathways. regulators

Mani Foroohar

Okay.

comparison So of cross-trial of a you mean kind sort sense?

Paul Bolno

Exactly.

Mani Foroohar

Okay.

Anne-Marie Li-Kwai-Cheung

Yes.

is fact a the space. to that unmet need there we're think significant this in still referring I really

Mani Foroohar

Okay. was helpful. That really Thanks Thanks. guys.

Paul Bolno

you. Thank

Operator

call Bolno closing Dr. back Paul turn thank over And for now will I you. the remarks. to

Paul Bolno

we on and families grateful great for their and call everyone, focus for every our exciting the This and unrelenting mission dedication on to organization, for Wave you, a our Thank morning. joining time the employee serve. I patients and is an this Have am day.

Operator

today's for participating. I Thank now like -- you concludes conference call. would this

disconnect. now You may

Wave Life Sciences (WVE) 10 Nov 222022 Q3 Earnings call transcript