WVE - Earnings call 4 2023 | Wave Life Sciences Ltd.

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Operator

Good morning, and welcome to Wave Life Sciences Fourth Quarter and Full Year 2023 Financial Results Conference Call. [Operator Instructions] As a reminder, this call is being recorded and webcast. I'll now turn the call over to Kate Rausch, Vice President, Investor Relations and Corporate Affairs. Please go ahead.

Kate Rausch

Thank you, operator. Good morning, and thank you for joining us today to discuss our recent business progress and review Wave's fourth quarter and full year 2023 financial results.

prepared Dr. with today me remarks are Joining the Paul Chief Chief Financial and Chief available Development www.wavelifesciences.com. our Kyle Moran, Executive Officer; on is Officer; morning press issued Bolno, section release of website, President Officer. The this Investors and Anne-Marie Li-Kwai-Cheung,

to Before subject to you those to cause the and to that during obligation would several factors Paul. that to forward-looking our today statements. release cause reason. I'd our revise SEC turn actual like will forward-looking that statement like statements. in these could to update in from conference risks to could differ described call the include results are I and call differ forward-looking actual no discussed These we discussions in any any begin, press over filings. remind this or statements undertake materially We results for are uncertainties The issued now

Paul Bolno

Good I on us you morning, continued execution all our Thanks, with thank recent and joining our open on will for and progress Kate. call. strategy. today's on comments

was also execution trials available provide Wave. financials. will clinical X Chandra ongoing review call opening [indiscernible] Kyle up on and the and tremendous will our progress Anne-Marie an for questions. XXXX our of update for questions, Next, a before year will be for

medicines Our achievements change our pioneer potential that for and far-reaching multimodal of truly platform demonstrate possible people's the health to reimagine human what's lives.

leading areas. are in multiple We the field

today initiate INHBE our XXXX. the and our quarter in with are trial development that clinical first obesity in program innovating are INHBE we've announcing We accelerated to of

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Moving to on INHBE.

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evidence is on a especially WVE-XXX, mutant wild-type Anne-Marie huntingtin myogenic mutant the nonselectively this HD start first-in-class protein critical is patients XXX clear advantage wild-type quarter reduce will sparing this year. In which healthy expression also protein, in designed the also protein to further. third reserve. show to preserve our huntingtin are which allele discuss cells, selective HD, that We already is deliver over advancing we to to are data ability protein wild-type of in therapeutic. health neurons. protein, We pan-silencing as function of remain stem and a uptake the with important the Having while of lower dystrophin to track lower XX-week and first approaches

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program AATD a proof and year, XXXX this of be And Wave. HD mechanism towards advancing year expected will expected the readouts RNA for clinic, the and in coming for with data INHBE quarters from so editing DMD data breakout

our and over programs discuss for year, Anne-Marie? to clinical turn expectations Now progress on that the we've made to to call like data the Anne-Marie. this our I'd

Anne-Marie Li-Kwai-Cheung

of Paul. Thank for you, forward exciting we XXXX I horizon. I'll editing on Wave have should the with RNA oligonucleotide Wave, an and [indiscernible] be AM the XXX, conjugated our start many year or certainly milestones to look our comprised AATD. the Restoration for in advancing The program. Restoration We're X [indiscernible] for clinical volunteers was in have interconnected ongoing and homozygous X portions, WVE-XXX the AATD patients program restoration who mutation. healthy X,

proof safety in PK generating establish high serum, low, is have protein. taking In presence dose engage made be which M-AAT target. cohorts quickly X, and potential designed mechanism. will expected M-AAT Restoration will the and through representing a addition detect that's serum enable of protein assessments easy to of of the regimen rapidly the us to In multiple level healthy X wild-type Remember, these Restoration achievement patients never to medium M-AAT of dose data, we and to

XXX So is any successfully the editing of that RNA. M-AAT Wave would presence demonstrate

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in in Recently, protein, due protein to healthy is first-in-class, mutant HD. approach a studies. Huntington's function. ], neuronal is associated with focus candidate Carroll manuscript underlines Huntington the its it role allele-selective financing for is increasingly to now mice data [ Jeff Huntington which our HD new wild-type preclinical an preserving subcortical believe HD. critical an toxic becoming Moving WVE-XXX optimal disease, new area the WVE-XXX by that demonstrating approach a as in loss the offers and need or Dr. progressive to neurodegeneration, aims of shared We for cautious complete classification which PAM of treatment Huntington reduce while for

Just last allele WVE-XXX from selective first-in-class week, firsthand HD we Therapeutics for enthusiasm KOLs had attended the And its we annual about their Conference. design. CHDI and

these outcomes key theme for such for efficient to enough Another term, on [indiscernible]. are enable those was biomarkers highly efficient registration, sensitive short as growing correlate Imaging studies example, MRI development to more specifically well predicated clinical the and support imaging, to and wild-type how path for the and benefit sparing. were of us mutant of accelerated registrational knockdown allow if the discussions plausibility to excited demonstrates around establish they advocate a Huntington parts the by biological the We energy with supportive can HD community sponsor data. with

that Our is evaluating XX XX HD of have X doses milligrams study versus ongoing patients XXX received SELECT-HD of weeks. every WVE-XXX in placebo

track along mutant We're to extended steps We path These in are that the Moran, the positive data planning multi-dosing, form XX% protein. call figure. we're basis wild-type supporting data durable all multidose Kyle report [indiscernible] bring showing of promising the from with turn that, least accelerated single-dose data patients. And to financials. to an the with data our of With follow-up to actively single-dose like looking advancement efficient I'd quarter. would this with of to enable Huntington pending at an the data provide our of will on on knockdown including preservation cohort replicate to and to CFO, an WVE-XXX program, for update decision-making with this next second

Kyle Moran

Marie. Anne- Thanks,

fourth the quarter loss million was XXXX and $XX.X $XX.X period, million for Our respectively. year net and full

prior the Our substantial earned significantly year improved our collaboration period, partners. to net from revenue the loss over primarily due

of became January GSK, During recognized which million the effective collaboration in we fourth XXXX. under our $XX.X quarter, revenue with

] quarter. Research XXXX development on our $XX.X $XX.X primarily program. and decreased [ We the programs million quarter collaboration for of million increases CX compensation-related our spend million by our expenses of $XX.X quarter. the by as Takeda in driven were and XXXX was million fourth offset the to under as to the increased prior the in year increases in year costs. fourth compared to external compared as also discontinued prior increase $X.X This were with of related in recognized revenue as quarter AATD partially expenses well DMD These

flat were in of full received the Subsequent and $XX million prior GSK [ in year-end, quarter. cash compared Our $XX.X exercise for XXXX $XX quarter $XXX.X milestone in a payment proceeds net and ended to We the option to the December equivalents. year also fourth financing. from million million G&A cash essentially with our million, greenshoe ] expenses we the for

into XXXX. expect of our equivalents fund the to cash fourth operations that sufficient be We current cash quarter and will

milestone receive year include to we but the over turn the not reminder, milestones future opt-in remarks. Paul this call our collaboration GSK or runway, back have cash closing to we payments beyond. I'll a Takeda near-term meaningful potential do do As payments now under any in our and or in for

Paul Bolno

Kyle. Thank you,

advance to clinic way the This the program validate the FORWARD-XX from truly quarter XXXX, dystrophin As INHBE obesity a summary, we well trial of we ever broad preclinical the turn for platform that, and are sharing company. And our CTA our RNA new data along and quarter possible And important Q&A. XXXX. to inflection as the continue as proof fourth share single-dose our exciting editing trial for with follow-up point, siRNA on and from X clinical a progress we're first pipeline. deliver extended on in and plan medicines with data trial SELECT-HD our multi-dose for and our to with programs. clinical on advancing towards clinical potential including mechanism program in INHBE with the we forward year, the editing RNA first Submitted second data as I'll and clinical WVE-XXX initiate with look data early opportunity track to building potentially remainder our to leading data a best-in-class in quarter. this for call the along of RNA look the with further to in protein over our deliver clinic. the what's HD of we registrational of readouts quarter the operator our patients deliver data, the in a journey as Operator? Delivered the to unlock in third reimagine the as We you all year

Operator

[Operator Steve Raymond comes question James. from first Instructions] Seedhouse with Our

Unknown Analyst

on This is for [indiscernible] Steve. morning. Good

So questions for your we RNA AATD of have couple editing. a

I think disclosing M-AAT. mentioned you serum you will be

since something you one expectation now? to healthy Is So what is the level right you're at looking achieve of that competitors Or expect what eventually levels. M-AAT your targeting of target eventually level? are M-AAT for to your

Paul Bolno

you like would Anne-Marie, to start?

Anne-Marie Li-Kwai-Cheung

that the We So recognize lower I in point healthy think respond to exogenous key. very We're the and really of delivering the is we're believe of XX low with patients M-AAT know demonstration normal is And have is of the XX. that M-AAT to and MG a in healthy able clinical wild-type not like editing of replacement through important therapy, patients be RNA will that editing M-AAT we're already limit restoring we but outcomes. that AAT ballpark with the functional risk patients. restoring

Paul Bolno

stepping just you as lower correction. target of ] of think heterozygous and [indiscernible] the patient, the limit said, where can threshold out get obviously, micromolar of with Anne-Marie as back of well the the kind I XX was came that [

only the we've the already heterozygous ranges but not are I range both think call, said already we're the think that if healthy. within achieving the on preclinical seen in we also as studies, we that earlier about

editing. the reframe thinks levels to versus, how And protein. think say, about editing let's And we we say so the I should RNA exogenous need protein field

really when the doing of and an they're XX a the corrected so achieving depleted, and the patients. at Then potentially you of in, these of editing at a We're wild-side you limit, would heterozygous you've trying levels So because putting more be from being stay to or need that's exogenous the these patients could do protein knowledge that protein to able was equivalent of you're you're patients produce protein. established micromolar -- imagine more correct already with correction patients would the a that if threshold transcripted exogenous established you threshold protection threshold or and a equivalent healthy born heterozygous level that so protection. more that transcript concept for underlying with to the protein, lower establish genetics more was it's

different one And dynamic so editing at Number thing in correction. replacement. about play the broking

still there I is lower X So think a of limit micromolar.

really, about at M-AAT of editing levels. need as threshold But both these total healthy how level keep patients. surpassed as a to in be you've AAT patients it we data an as well measuring [indiscernible]. those once that, threshold think, on that to will patients to achieved But I at we to then Our the I call of got that concepts more preclinical protein even And more shift think therapies. [indiscernible] and look to percentage in able look being is with

Unknown Analyst

about Okay. also progress itself. question clinical study a And we the of have the

healthy what Just have in do dose And And start to terms have patients completed volunteers? can you levels complete dose dosing the patients? you of what started in already? AATD you before screening actually need you

Anne-Marie Li-Kwai-Cheung

Sure.

Paul. Sorry,

to and X do mechanism So X, in progressing Restoration the restoration Restoration we're from for Restoration therapeutic track Restoration dosing the volunteers. know, X starting and studies We're expected With the healthy through as that. of are of this a on proof relevant aim with data expected, X, the interconnected. level, And to as in you year.

Paul Bolno

think to that that is modeling. calls be PK prior call that modeling on on sustaining I modality what's the is it's a mean, on as think into important that, bring and Anne-Marie new translating. to add I said think And to key And you driver the said we as I I how in selection on earlier, when important healthy study. is encouraging that what's clinic, and going discussions volunteer a dose based is

we're track So I deliver that. to on think

Operator

comes Our Mizuho. Syed from Salim with question next

Salim Syed

where initial INHBE, you quickly of Would I do Paul, intentions guide use full size? the sort perhaps how not the going the treatment trial landscape give active design can treatment the Paul. the just sort us but how INHBE the just in of and to getting willing enjoy I see your given guess can't just you're an of on is moving Congrats -- you thinking know really are this to trial slotted? in progress to space new us clinical product you about you're website, comparator, on -- be and terms you where design,

Paul Bolno

you to it Thank for Hopefully, makes access all easier it design. web noticing information our and updated the thanks constituents. for

As move -- lot year. I'm for it about relates on to to the throughout and talking we designs and INHBE, will trial a clinical this that glad there be as more we're come

the INHBE landscape other think I updates and other to your how point evolving therapeutics. on the to relate do silencers

got think I lot a thinking opportunities to updates the continue throughout on to we've about provide of synergies. XXXX

studies bunch we levels advantages As to in time have just not year of think it lower potential a potent to But triglycerides beyond of very the these HDL. of annual, initial they because as have are twice higher a engagement. genetic study. we have we're this because with I relates that dosing one healthy about and shown other have whole the in let saying, target program we a and about we volunteer us thinking biomarkers biomarkers think some of creatively of is do have think trial LDL design, patients to studies durable biomarkers, the wished There actually, we're designing and

point, also it to think to overweight look to us not that across volunteer the profile establish provides that about So biomarkers. running pharmacodynamic I healthy study, ample treated And your a about on to safety, on effects in dose how think at we population. build for think but then just sequence the and do want we opportunity an as to

run-in GLPs therapies. the initial study, other So don't it's about, whether this think I we to think on have or a is

translate be the subsequent or with in us we exhibit think and we the a that combination where which RNA platform set we think overweight into would in advantage from we introduced the I think having with about the as high-impact own up studies. But patients then this effects. synergy with think on that which about the how to the course internal important capability speed study first program, ability use we very we into population the But our could in of translate I the of a excited would healthy just clean capability to we're have over year volunteer but GLP-Xs our we to so off could only to to clinic. clinical come not I an just think see clinic transition the as foremost, can either see think RNA, have and we when sequence speaks more that discovery manufacturing it year. in target we I And that have also the the -- to rapidly last

Operator

from Our next question comes Truist. Joon with Lee

Joon Lee

what that And complicate the On the on penetrant in [indiscernible] INHBE a understand mutation I assessments? obesity? have program the nature [indiscernible]? that quick against are INHBE modifiers are they elaborate and of that obesity, protection potential the can the could [indiscernible] I phenotype And it, of some you follow-up. clinical your a population bit general confer but how are the

Paul Bolno

we Yes. of I variance. of at mean I look function -- think lots protective as loss

defined are the always other find things a it's there of -- between think interesting I on a them. them, learned you've and part few of when you lot side in

the of find able are I notion to happens what us So think heterozygous. being in patients

about toxic question than rather the So is I correct is say, function knock it down looking think like, this at what is to gain we need opportunity that your here let's and population a see that to of protective.

then about experience And cholesterol, both those. you of type you kind [indiscernible] clinical of of importantly, direction. When go fat like loss for measurements reduction the key than another and to have reduced good storage [indiscernible] a think these as see the the unique genetics patients out, of species see probably in U.K. less about And reduction of evolved penetrance cardiovascular obesity, X human the Lot HDL. that in actually because bad diabetes. that's rate abdominal probably you what's this I And in X.X%. I in triglycerides byproducts of back think we improvements clinical this we is as actually is think cholesterol, this confers from of follow So genetic function disease

a induce a it if it So of your out or births and in inducible. it's something mutation, is to function. The from minds is you you these protective is correlative just which need really what always in for it? were of to to this question see [indiscernible] our need all comes you point -- loss happens of happened case Or you that

so That's were data so of and of we is same this And whether last was looking translation of was have we really function data for that and why last or back, first that first data at to year vivo excited the see to the shared that we in about, when phenotype. loss showed what thought not really induction a the year time it confer right? would inducible. the the That important

induce focusing did, than silencing, without weight could if we [indiscernible]. greater So see equivalent we really muscle depletion, that you was correlate I loss, and loss you but say fat on, be loss should of which fat with XX% it

important to that so And was as ability be target we the notion first the that had I induced. about think genetics really a found we that think that population the

it didn't need you So delivered phenotype. from correlate. it It [indiscernible] didn't the

of this And we now, to continue fat highly within concept, that follow we the that and came inducible [indiscernible] are we an it was visceral phenotype. to best seeing as continue this think and expense specifically. fat the the measure so example I at of And loss run it's can -- fat, experiments

experiment studies to to to forward. move the continue And think, changes see, really continue then and human these run, see can so I we collectively, these run biomarkers need we and to

Joon Lee

capture overall potentially simple complicated Just with assessment, clinical regards going you're Paul. on would mass? curious as it given thoughts on is That's more to to how weighing really helpful, the them? beneficial as be benefit. the the impact your it Or muscle And

Paul Bolno

So get benefit the question see opposed to and make to I weight get... how the the fat with is, the opportunity loss? in I we just do sure think sure -- I clinic of make loss corresponding To as

Joon Lee

Yes. mean, straightforward. is I GLP-X pretty

don't In weigh mass. muscle weight you the as just You the then them [indiscernible]. lose At lose same an loss from you wait your endpoint. drug, and time,

functional or So a [indiscernible] is Would that you to test how benefit? do capture it do you just need

Paul Bolno

the dissected that out, this models beauty can measure in seeing preclinical of And No. right? in we that there of in is has I fat. see have mass. can We muscle And why was reality, program that's right? we reduction the we mean I actually I an is mean important muscle forward, mass, we weight, determinant. lean key fat the moving and we lean loss. loss we're which muscle weight no as measure So coming think And loss at [indiscernible] -- mean, showed

about about we But I the clinic, because an hip going of is As into weight phenotype Those that also function. ratio. think do patients. we're We important loss to protective can of want measure we weight to going humans, loss think you're call right, of them the feature. to

hip So weight that of it's low and ratio in BMI. the have function have population to this loss humans

So weight There's and fat we can at of with to be reduction also reduction take and measurements able as abdominal in the that demonstrate look for specifically correlate and fat. well in that. visceral that to opportunities clinic abdominal imaging

products obesity of target program this biomarkers on measurable other really classes are from there to speak that to really mechanisms. So distinguish

Operator

Our Schwartz next Leerink from Joseph comes with question Partners.

Joseph Schwartz

wondering aiming before than achieve a clinical were first of done characteristics, about planned? humans? work for? was couple remains your you elect What can of you you did How encouraged talk which be into to that were the INHBE. move able things together on you expectations. quicker key INHBE ahead come it you And Can you candidate what I to to

Paul Bolno

the thanks question, Joe. No, Yes. for

other of just which this expense weight So of weight I June's wasn't target, to in in loss with the the class. is we started demonstration meaning importantly, similar mean last obesity characteristics the were that at the loss established, of obviously differentiation function muscle. of which the already had on question, to mechanism key program products year, loss last And

was loss anorexia an targeted was [ fat. ]. induced So It it of actually

And that so we demonstrate were biology able program. to part of the of as consistently the

thought driver frequency. candidate we think dose as selecting and I really around driven was dose the that about

as So we this are single-digit low like those dose. we've is [indiscernible] large said, indication, from recognizing a learned be seeing, things, as that important other X to products

that. And a of So product importantly, helped drug conjugated are candidate. those amount helps the -- twice very with of identify to able wanted less siRNA still a had We on frequent our IND-enabling year being a too, obviously the that were done a durability. lock studies, and achievements underway. thinking use Those potentially most a at be dosing What's in us to low annually. [indiscernible] is what profile

In run is [indiscernible] template. we fundamental resources, enabled translation is nice, I forward. in two were to this, as the parallel terms to accelerating think, what think the here, with accelerate really really is time the of Couple to One which moving of -- with financing I that could and really the siRNA they year us the make there about parameters. experiments we were said last that was sure

So were that rapidly to that able template and move we siRNA, with to see benefit.

a which in-house for studies clinic. feature, didn't studies, to find just was go accelerate to getting second make enabled trying out having for manufacturing, us we to and have CDMO to it meaning preclinical start to material, our slot to not a ready The the but toxicology truly

study high So by and the in-house material rapidly we [indiscernible] trial could manufacturing, our best that to be material the having clinical could to move we preclinically, deliver enabling make so clinic. positioned data importantly,

all So have insights antigen into we to year able I things on the bring decade us translate that last target the the medicine convergence early last in really rapidly from of we're working that to the on the potential for into clinic think the of XXXX. in Wave been we'll a allow

Joseph Schwartz

which relative the differentiated demonstrate for to a weight should then best benefits? one both well translate as Okay. profile for WVE-XXX ones And of which different so wide on models effects I many or to loss best clinical that given -- metabolic the was there wondering array as tend in potential the helpful. the focus potential incretins metabolic one are we of That's development? And obesity, for animal

Paul Bolno

Yes.

on in you're what's think best and that. fed I sure the diet-induced model possible to could as the have how in mouse chosen you recapitulate obesity overweight models humans. that making we focus being of seen other require There's

are models these does you don't of discern lean existing muscle being correlative. against able benchmark weight loss. and we've before, can we've that do lose think show it expense shared important mass us from at which But fat is weight muscle weight in to in also of what's loss think the I do We we've those that models as loss. mass done, to highly loss data seen to I allow is these GLP-Xs and performance the measurements what

So really fat losses as rate a concept healthy what losses. of

measuring us [indiscernible]. model dose in to of the best do frequency a cetera, lets plan so to and dose, lot that be able et the biomarker, for And terms

Operator

from with question Issi comes Capital. next Our RBC Luca

Luca Issi

ones progress. much right appreciated. And to versus on just two in I quick on GLP-X see you congrats as here. that Maybe then from the that they us Huntington, Takeda in number a GLP-X, discontinuation maybe if the correctly, would can Great. you Maybe QX any the XX%. exercise it to option, at mechanics seems order can here? rate in color And captured of And remind hearing there up you for need mentioned be be bit we'll high little higher what docs. on as think

picture for from? maybe bigger why the number your was and I where that if a wondering on comes And expand just So matters implications you program? can

Paul Bolno

Yes. absolutely. No,

can key we is XX% that it's HD program, the us data delivering demonstrating data, achieve, remember So versus pointed the Anne-Marie single-dose out, obviously, as key important like we to starting placebo. in with biomarker thought the reduction for

knockdown of did seen in sparing. the We've way that protein. mutant that seen we we've wild-side that a So was

dose. clinical the at single that a So data early was

brain we and achieving distribution actually region I milestone therapeutically payment concentrations be targeted importantly, HD. should in target study the quarter at what the a drug was think reported in could received for fourth that subsequently we relevant the $X showing on right also of Takeda we've that million from as

that now So required deliver key the as be put that's these submitted package. to And the to Takeda. we us data that's the is together, to package demonstrating for multi-dose data

switched focused we're doing has the evaluation data. it's for package. with with them program. on split, submitted, of the payment, on best which We're period a with reminder, discretion profit and then progress to payments be working that payment And over a collaboratively. substantial our generating Takeda supporting so We'll the a that milestone be clinic, to think along split when just contributions decide our to the XX-XX what's with additional be the comes program. gets that I it a [indiscernible] R&D an would package to

is should So for step -- package that a this is not. I it's think program forward the right or deliver Takeda, believe to we us deliver move we we think began that data whether which X package,

point important an about program. the And so I decision do think for this data coming set

data will to moving or decide the which So not. does forward program not have or this with whether the support

as are out. and rate, of As -- there question various analysis how based on a there's it discontinuation relates an This following how you claims reported to report database on on was it. -- and the your there's many was physicians a said, surveys staying again, patients do

claims with so And prime on. the a looking staying therapeutic with patients analysis at database,

think ways, this can look them we So always surveys People numbers. and report let in you you use point was different but use published -- at and different in the time. ways

analysis. based published this So is on

Luca Issi

Very helpful.

Paul Bolno

it's question off the people effect think that some it even I -- medicine XX% I ultimate I XX%, whether people important. question, It's dropping think the is it's say that mean a why the And are I think that's fundamental -- does. has

loss shifting we weight this is from the what's think versus I oftentimes about think sustainable what's healthy fats. So narrative

complications diabetes saying ultimately on of that you migrate GLP-Xs twice cardiovascular as to how or a people opportunity how the way is in induction I risk in by after is independently clinical staying sarcopenia therapy lost. is GLP-Xs in weight and that common for induction is so use it in questions move and phase, injection seen think people that used chronic of And they benefits once pointed from most how think will key U.K. of provides clinical it so will their ] these increase subcu where people thinking is data. I us maintenance real we a can earlier in human all [ all in importantly, induce front continued benefits this be loss think opportunity Biobank in have the And practice. been risk with have of a front have bad-offs and healthy the of about And out, I about a that the or weight to year, of the was us one genetics phenotype as sustainable

a not that that this able to being think is important transition So just see to therapy. substantial for really that's I patients the why opportunity this from data there's but on capability us end, is to front

Operator

Our [indiscernible] next HC Wainright. from question comes with

Unknown Analyst

from [ current And focus will new program. DMD the I the I your wondering, or differentiates the on DYNXX data coming ], developments how do was landscape? DMD the DMD you SRP-XXXX see program and given landscape from out the just in

Paul Bolno

is in was question. we see very to at needed this we something which beginning on Great I of to things this several bring and clinic journey and DMD, the your our mean, begin is focused like the point And forward into with. as core vector program dystrophin I to foremost the think on functional to differentiation, driver. production dystrophin the first of obviously,

become right skip transcript. productive, right? important, meaning levels was, That's what high to That wanted of we exon at with XX% concentration constraint. Two, muscle one, the the see that's of they levels high highest to important that [indiscernible] skipping level meaning So we concentration, an of of the cell. muscle compartment get see

us which that to give time in that where strong data, the QX, be over a we moving meaning now potential translation, as registration. XX and have at we're can dystrophin going like that support XX-week the forward have the coupled will with functional would combination, numbers think that the will same I benchmark products commercial data of the landscape within

that with just on number differentiation, of which dystrophin, also from. think of all dystrophin of I where comes another the point is but not comes

the So is muscle. the nonhuman in diaphragm [indiscernible] of what heart the distribution over skeletal primate it We our both is the in We double saw in higher levels we've it knockout skipdrug mouse. seen in in saw mouse.

of think on focus one the the is we key boys, respiratory function. we that as the with about think I cardiac and features So progress

differentiation. help ability point to key So into exposures [indiscernible] that of the with get tissues

young about ability as this think also get early [indiscernible]. I the is development, too, we maturation into the think to so boys into

the in So is to get a the us really we lot ability that and the satellite the also cells, generating cells that muscle improving will at sharing into update which be MDA, muscles. stem are gives potential regenerative

a dystrophin, we alone frequency. think there So do distributing we where just dosing high that of about we're think to, addition capability, in lot let productivity of that when of differentiation, is points to

Exon the infusion. products current XX, are within a on weekly IV So dosed

some we of original data been life, XX-day with The from seen expect concerns. of be conjugate of the muscle As differentiation this what's we [indiscernible] dose relates it benefit half we monthly before. to We shared, hypomagnesemia product. think exposure, with than ], conjugate thousands the our to holes that of what's as the without the terms a gives other been that also of [indiscernible] seen as a us programs other do shared in [ so

So a a whether it's and really in I we're a features, regions, whole think convenience, good I think we differentiate number can in on right whole of tolerability position. efficacy the

we answer think and see deliver are with dystrophin, I and dystrophin last study that a to those question the QX. poised fully that's to translate key as, we numbers. enrolled in long So

Operator

showing questions for I'd Dr. at like call the Bolno time. turn Paul this not any any I'm closing to back further to over remarks. And

Paul Bolno

to look believe all updated Have forward morning. call for exciting be all keeping day. Thank you the joining you a is an progress. we this for to We on year going our Wave, and great XXXX

Operator

Ladies and gentlemen, this does conclude presentation. today's

now may You and day. wonderful disconnect, a have

Wave Life Sciences (WVE) 6 Mar 242023 Q4 Earnings call transcript