WVE - Earnings call 3 2023 | Wave Life Sciences Ltd.

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Operator

Good morning, and welcome to Wave Life Sciences Third Quarter 2023 Financial Results Conference Call. [Operator Instructions] As a reminder, this call is being recorded and webcast.

Now I'll turn the call over to Kate Rausch, Vice President, Investor Relations and Corporate Affairs. Please go ahead.

Kate Rausch

Thank you, operator. you progress us quarter and results. thank our to XXXX and business recent review morning, for today joining discuss financial Good its third me With Dr. are today Financial Executive Paul and Officer; Officer; Officer; Anne-Marie Kyle Dr. Bolno, Li-Kwai-Cheung, Chief Moran, President Development Chief Chief SVP Ginnie Translational and Medicine; Dr. Yang, Chief Vargeese, Chandra Officer. Technology the press on is release our of morning this section The website, issued available Investors www.wavelifesciences.com.

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

Paul Bolno

morning, call. Good thank I Kate. remarks. and joining today's for Thanks, will you opening us on all begin with

clinical finally, Kyle And financials. on will trials. Next, our Anne-Marie update provide will our an review

our of we tremendous and RNA questions. brought our will last Since third advancing for for We be including Day, call DMD editing strategy owned first-in-class and our outlined In we RNA disorders, to in clinic. have Chandra the for our Annual at into Ginie targeting quarter, advance and progress will open continue new wholly siRNA AATD update, novel for HD. a announcing trials our We metabolic for therapeutic inhibited clinical our made our then the pipeline medicine. the obesity, including questions. growth program, also WVEXXX team program, R&D available

to data XXXX, clinical are a we select our transformative deliver Wave, candidate. approaching for key year three sets and ahead of for will inhibit programs Looking all and where we clinical

And we program. of Starting investigate clinical CTAs will editing multiple with industry's WVE-XXX, or restoration detail RNA efficient to in in another initiation of our antitracy and describe We is ever the are acids. patients approval dosing path milestone for remain Wave, WVEXXX, highly of begin which significant for the to trial restoration candidate nucleic quarter. on proof AATD. clinical or enable the announcing later, a as to the the designed is today, will alpha-X This more applications field healthy volunteers program mechanism. track the Anne-Marie this first

profile WVE-XXX Our excitement the have observed to strong we is date. grounded in for preclinical

our subcutaneous in preclinical We with durability remarkable of editing and achieved convenient dosing have studies. potency

DNA through yielding a that DNA, market. and elegant specific only for differential therapeutics highly with to contains of the editing tool XXX isoforms well collateral conjugate, not the approved with game potential and off-target modified in of PIC unique, oligonucleotides editing transcript the chemically corrects enzymes Because our precisely our effectively with to result delivered can artificial their mutation the genome hyperactive bifendorevedane. that and In ability recruit no WVE-XXX exogenously rely function. bystander is delivery a edits on significant on silencing technologies validated by permanent irreversible protein contrast, at fully multiple enzymes. on

manifestations remains intravenous current in of unmet dose. the largely stable to optimized GalNac and With AATD, disease, leverage that future is and high. which to in treating require own the have significant can pinata avoid the a have either For therapies their AATD need challenges a candidate it the confined advantage pulmonary and particles, or hepatic

per accounting for year. the substantial, with in Even sales augmentation therapies, AATD for $X market with available alone pharmaceutical the limitations therapy is billion of over

strong long GSK, in with year. program look medicine, mechanism data differentiated in and commercialization. believe proof partner, development next a to of leadership has this we Our profile, is history we together a XXX position respiratory And forward clear delivering

of to emerging starting terms this this the receive to will substantially of to modality Wave collaboration, for under clinical editing our reminder, would value derisk have unlock to RNA also XXX milestones to balance continue pipeline near-term eligible sheet. our meaningful is year, potential with the a mechanism Clinical meaningfully our proof novel As of add and programs. which serve

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silencing phenotype These new identified in we Notably, measured period and relevant of goal Since achieved can carriers. and the target selecting straightforward by heterozygous protein with using advancing to towards and are of chemistry are the candidate time. potent believe have the because rapidly clinical format fourth an to short serum, clinical first XXXX. lofefunction readily R&D highly in in inhibinin a consistent demonstrate our in and our vivo data deck, leads engagement specific the inhibinin and quarter other the assessing path levels we be relatively clinical can biomarkers of inhibited efficacy of be of

continue GSK's slots on to track we end, collaboration to programs, WN-XXX targets As WVE-XXX, are have three two to and owned both an our DMD data allows HD a at way meaning and deliver programs beyond reminder, validated for new least our clinical leverage in Turning to XXXX. from and wholly genetically programs key we advance to additional open programs advance

production functional meaningful convenient, vector benefit [indiscernible]. dystrophin mini exon dystrophin or more micro to best-in-class look or WVE-NXXX, For that aim and our endogenous and clinical to with treatment dystrophin we option all of provide for DMD these in forward There amenable skipping functional options remains a ultimately the of questions evaluating protein to benefit urgency significant patients like to XXXX. therapeutic delivers translation around safe functional deliver the of We we recognize patients. to to the

In data single the Huntington the with asset ] wildcat Huntington date, [ the in believe the To preservation demonstrated and mutant translation of humans. a reduction WVE-XXX in successful dose most is to after a Huntington's we have of clinic of preclinical compelling disease, we promising field. our

have ability the We support levels exposure robust our of that evidence studies, from studies, to preclinical including NHP oligonucleotide significant the CNS. achieve multiple throughout

call the of mutant over development will now our in programs. we first next multi-dose. her. reduction and speak to AAG program data in I'd Anne-Marie? while to As single clinical we ahead And what similar look type faring year, other observed knockdown and turn more transition from to HD durable SELECT protein, to the like anticipate wild our our when we clinical trial we Huntington Anne-Marie multi-dose on Select HG to potent from WVE-XXX

Anne-Marie Li-Kwai-Cheung

exciting as I'll with be for continuing to platform the to you, AATD. in candidate Wave-XXX, of clinic. Thank our translation at GalNAc-conjugated editing a really oligonucleotide Paul. our time or begin waves we It's RNA aim the are

become mutation. PIDD modeling with healthy patients rapidly interconnected as Olivenucleotide received accomplishment to we for has The can the initiation and editing cohorts of to affirms PD defined respiration our initiation editing one DD serum. clinical XXX in inform along protein and the delivery are RNA to two for applications This efficient the of We oligonucleotide enabling enable that homozygous officially wherein comprised Today, our volunteer dose have first the target which of have of level approval CTA. expected can for engage Restoration by mechanism program, pathways now AATD additive restoration volunteers trial. detection thereby the in trial of clinical multiple proof is healthy clinical a with that can enter RNA leverage or individuals regulatory announcing who established portions. at

in of of the restoration first have mechanism low, of patients and whole this and potentially initiate dose AATD assessments and cohort. delivery it XXXX. and patients we multiple before completion volunteers proven high underway. prior X to meaning possible expect -- throughout achieve of will mechanism of is is healthy serum quarter with trial In in proof in MAAT cohorts dosing to Restoration the can data medium completion X, the now to

cellular average more skipping dystrophin exon the observed This relationship exon exon at doses increases DMD. to muscle may patient I'll nonlinear has excitement then on this between sticking collected skipping production distrain enable and skipping skipping reminder our other amount been results we X to week. significant with mean Moving drives observed quick study, every proof-of-concept dystrophin, XX% data following our seen. a exon In given that is of dippers. of in program. the be expected improves Since non-linear in and production we've the X biopsies and environment weeks Wave-NXXX start of from the with clinical

plastic in which progenic to speaks especially Furthermore, high promise the to is and cells, concentrations NXXX achieving tissue new of expression. myoblast best-in-class with myoblast of sell for the protein dystrophin

NXXX. on clinical tolerability, safety pharmacokinetics dystrophin and doses owned of FORWARD functional are is be remain XX and our a which file Exon for positive, plans our registrational evaluating is also trial in functional deliver XX XX, per expression track to powered trial our data the and and This protein and week NXXX weeks DMD We advancing data accelerate dystrophin in approval of limited multi-exon trial's XX franchise XX. primary plan trial evaluate potentially every build digital accelerated which This beyond the will would wholly also is and indulgence assess U.S. to support III endpoints. XXXX, of other to endpoint. to development mg would open-label after We will kg Phase treatment, The

generated XX% would [indiscernible] the that have excellent compounds DMD of dystrophin vitro skipping in We data for follow-on together restoration population.

with chemistry defined and levels high demonstrated have vitro follow-on These production all PN are skipping dystrophin studies. of and in exon compounds restoration

treatment first-in-class, our offers allele-selective devastating [indiscernible] healthy Huntington petite XXX protein Wave-XXX, toxic while has disease a Huntington's mutant protein. for or optimal reduced and disease, the wild-type the approach to Turning is preserving where HD, candidate an

oligonucleotides levels deep This to including a the of CNS nonclinical for to existing As a milestone pertained the in collaboration, positive is showed from this distribute in bolstered HD. the and a exposure of regions, collaboration data quarter, X.X sets our which imported reminder, program third Scranton study confirm areas tissue In this primates. results of we our that active the nonhuman XXX achieved with the to of significant study Takeda. part an from brain of ability

evaluate to already of Coupled enrollment share placebo. confidence the single Huntington and cohorts and HD to now new results this we our dose this enrolled further that program, CSF which cohorts completed mean the multi-dose reduction fully NHP have completed we've simultaneously. XX the is XX% single-dose demonstrated from inform of in compared with multi-dose for I'm patients decisions mutant comprised cohort reinforce will XX-week enrolled patients. approximately the single-dose XX-milligram excited further program. to now the multi-dose critical on cohort These

report We the XX-milligram along single-dose with second extended the XXXX. of expect with quarter data follow-up all data to multi-dose in

an We program enable this the them. high ACV will all trials. I'm for the of I'd Kyle turn impact decision-making our and support option across team's we our ahead, also three truly to proud which on package during clinical with on exit expect the year and our update financials. Moran, our call these to that, data In to accomplishments of have our like to provide over summary, And year CFO,

Kyle Moran

quarter. loss year of both earned achievement income This for the the collaborations. change and biosol net million Takeda Takeda third of Anne-Marie. XXXX increased recorded million revenue for was compared the the net driven to primarily as year-over-year in of Under we quarter million WBE-XXX. Thanks, prior a by $XX.X collaboration, GSK under our anoxlotical $X.X for $X.X We of

ended Additionally, were increased milestone. related we expenses expenses year million quarter, third Takeda third third expenses quarter Subsequent in XXXX $X.X as and compared recognized to by to on in driven of consulting the G&A year million and prior to $XXX.X the million was external in the three increased for to related previous increase quarter $XX.X and of equivalents. quarter. due $XX.X and discussed the to clinical primarily expenses. our the $XX.X in under million $XX.X professional the of million the WVE-XXX We received million GSK $XX.X the development all cash $XX.X to the with achievement Research in prior million collaboration. discontinuation compared of programs. as of end cash quarter were This quarter. million the primarily spending we the from

cash be our cash to equivalents and that expect fund operations will We XXXX. sufficient into

not beyond, including our But back call to development we potential do payments As the any have over include we related EV. Paul. and turn future milestones milestone and a to I'll to near-term the meaningful reminder, do year cash friendly. receive clinical this milestones

Paul Bolno

Kyle. you, Thank

most a is best-in-class pipeline exciting in Wave the and the transformative medicines platform RNA industry, an chemistry of inflection versatile approaching With point. medicines,

year. As XXXX, approach deliver the milestones I'd many to we like to recap next near-term we expect

for WVE-XXX. ever with of deliver clinical mechanism We expect the and share first to editing proof data RNA

to registrational We with expect to multi-dose deliver and trial, data. data SELECT FORWARD HD single-dose dystrophin data all from deliver XX from the extended clinical our expect we potentially cohort follow-up along with

inhibini that, patients the to of also you. clinical goal We with we continuing for it for the supporting forward Q&A. our what X fourth year-end thereby possible a share we expect selecting to by clinical candidates with and quarter XXXX. XXXX, And over look are candidate new Together, select we'll progress our turn to reimagining to by of operator

Operator

Instructions]. [Operator

the from question line of comes first from Seedhouse Our Raymond James. Steve

Steven Seedhouse

about and can something that or the XX of course. to IHC -- you I'm in for that's as you spoke program, directly about protein showed curious dystrophin histology well that Day for satellite the cells And wanted something? if that of those is do like by an academic first still meaningful at cell I look study that ask or think that Or that Phase would II what ongoing this it something as of the lastly, view sort do in is? biomarker a meaning is DMD and you speculative clinical point? experts the would clinical regulators you and R&D

Paul Bolno

the you Thank for question.

if along it drive do the of satellite full think the about data, helps to the So we dystrophin I think importance production cell think we muscle.

which of cells. repopulate satellite composed myoblasts buses those the cells, are but muscles So the also

immunohistochemistry in terms something look of will assessing be looking if do about we study. that for So for next dystrophin, obviously for dystrophin the and think standing in we

important most satellite growing important that we the newborn can our think U.S., think However, and the I for meaningful in really Western in in But be against the with blot is around know dystrophin totality now the what's assess screening we is Europe. is screening the biomarker growing I of production us peers. will newborn this cell treatment, quantitatively

disease earlier the been for the historically much think the at point would of process loss ambulation diagnosed. about have in before before you disease treating the If patients

repopulating to part treat is the the a exposure get able to the Being body, and of those cells think treatment paradigm. earlier critical boys we in

we'll all that in demonstrates it's important it also So treats of the piece obviously think something of it to the I boys. potential NXXX just chemistry that I term, study. treatment exciting three cells. -- assessed it's because was the PN think be and But the in identified disease. long totality select it broadly We in the it's think an the subsequent our be muscle, not And will of distribute to

So study. a in boy particular unique original this wasn't finding the a in

I FORWARD in the going So think it's we compelling as at it XX look study. be to

Steven Seedhouse

I Okay. antitrypsin also That's [indiscernible] separately to great. ask want And about the program.

Just this to focused And their course, their liver is targeting of phenotype knockout development Telia the phenotype. only. in advancing on approach noticed then, RNAi lung preface the question, program halted morning, of They're I just liver. approach

So it RNA treat speak, for to birds maybe to patient. lung really demonstrate seems so same Wave even and within can you an opportunity hit the both one that liver with there's phenotype, and here and stone, like for editing two

of would a of where of liver that's kind once? many both could from about that's a therapy that? general, the the if addresses enrich that category really, even you phenotypes at in for patients focus maybe into patients initial and how curious they to enroll I'm So And fit market, if thinking sort benefit this possible lung

Paul Bolno

question. interesting Obviously, today. a phenomenal It's update No. an

can of today being our treated RNA to other a and think filing actually, from trying regulatory the expect IND an similarly also filings to to that with oligonucleotide filing nuance very for lung. interesting a we're shift think is CTA I we other where we announcement then say, the editing that from

think versus we point I I to still from [ of and the patients important liver manifestations a for think editing exclude RNA they both ZZ ] beginning, questions your to versus there's AATD. disease. your want lung, editing. point lot agree it's the So to particularly DNA treatment about technicality have of go don't that around on We But the patients, on

protein So expression obviously, a there's threshold.

wild-type Z So restored function. over liver improve same elevate able protein the with lung. protect out birds to and But of allows the we've two too, the that again, us as shown hand, obviously, come protein lets that function being liver beating protein to time,

we've and is that comes the think seen, for that have thing RNA point, other healthier. this The is, important repeat I cells dosing we an editing again, shown with get

production so And necrose when healthy all of infusion to over hepatocytes visibility and capture you that injured to get have which don't more as try the that to protein over time, restore as hepatocytes dose watch you cells time on the they're improve you repeat other the to well. single make and then continues

So of going have And the that both I patients. weakens so think Europe, the totality having a after, that much. And singularly for U.S. I these the and in again, we're exciting we is liver treatment this really if XXX,XXX for and important treatment treats think lung. a about so patients promise disease, the think thanks

Operator

line Mazor. comes Syed our And Salim question from next from of the

Salim Syed

update space. and on in the for little Good for DMD Great. morning, Thanks for the we're you important the year, year one XX next me an since Paul. question

get views. to curious Just your

can hit as a a on -- seen the nuances whole, to accelerated $XX implications and had that any everything -- We updated to or compound, X, the if you and DMD we've talk sales does thoughts primary Olividus, Just commercial or the curious, generally with given AdCom, either as time the approval. in talked on in you about right? controversial embark about space we Sarepta just Just on regulatory million then the it quarter it to to relates and space? your it failed lately there

Paul Bolno

had need I think which think mean, that treatment important was that point market, whether not and this you or bring community. I existing very I an are coming new treatments patients a hunger I think And they functional patients PPMB I is underserved. think We with think over is is gene to therapy interesting the up, need recognized discussions with improved the there for patient skippers, new dystrophin. I and that

much the not as approvals just frustration out, for thing seeing obviously patient dystrophin to relatively medicines, can States, in United U.S. the the to on we as on focused at as so we regulatory the functional with can't are these the And individual engaged community, is from point as think I say But and Sarepta's And think that's functional I translate these space, look possible I are who U.S. is you another where driving discussions. protein benefit boys, in we're I that what we comment watched the routinely accelerated here getting on market. outside as

complete are the study, going finished outside haven't patients States. they because United the not to But treat

So protein to those as of both XX% of can focus. our core boys to but we U.S. I we but across strategy, about the to that our thesis that not that being so program, XX that's able again, are our functional totality globally, ultimately amenable beyond think translation think a expand delivering that of just see XX,

So boys with protein for the delivering data. about FORWARD opportunities data we've XX seen DMD. providing terms with We're of substantial really in these excited and initial that

Operator

Joon from question Lee line to of our come Truist Securities. And the next

Unknown Analyst

Lee. is or using hidden for to your XXX platform. RNA line the in analyze on the for Joon do now and you you've just editing. D.C. you ], progressed CasXXa I'm I'm with for the is time ASO. you demonstrated on have an And wondering, [indiscernible] This [ guide could wondering, plan RNAi think ASOs, that do just progress just CasX faster E then molecules

Paul Bolno

So question? want repeat can second you make I sure. just to the

Unknown Analyst

a just the could could you progress if opposed oligonuclutide little on just you elaborate program. RNI you nbn approach as antisense I'm within Yes. faster for an think bit if wondering to

Paul Bolno

Okay.

the last one. Sorry, I'll take

not make I clear to it inhibini because sure want was So for AAT with inhibini. just or

our earlier in we double-strand faster with we've working shared [indiscernible], gone No, siRNA our a siRNA been for think actually, and an while now could you're in for in our we So with experience have ASO. format. collaboration saying as I

So inhibited I program. there don't pursuing think for disadvantage siRNA is any the that speed by

seen the think us In think, and, really evaluate multiple having field I RNA. you've always think I is now for the that in not the but what right modality in modality we format. in treating fact, given we're I modalities, I potency, best just competitive And lets And in quite durability, I on GalNac actually we've this track best on what hepatotyte disease. said, and the think silencing, think have

Frankly, can to think potential about for this we or biannual treat where annual dosing.

So I think we've got a competitive program here.

I in is ahead to to think and we'll goal our stay it. of watch the continue space,

because to these process we As translated on it's the in the manufacturing companies this our is guidance around we first one apply spaces. What we to multiple I to our of manufacturing your capability with our the for in we I our think question, is definitely about and across interesting collaboration Wave work numerous [indiscernible] don't that trends, power translatable. that think know-how to be ability in chemistry have on right GMP oligonucleoide formats an call as guide I discussed is But now. highly and do going have CRISPR think development to development, and want to have work capability and controlled process

to the in the space. again, we our advantages. focus chemical now we of proprietary other I think alogocleotides a case we're modifications, shared But editing lot has RNA are earlier, taking definitely transferable the our right is across approach as

Unknown Analyst

just one then more. And

And recently the it's quarter. second going noticed out were this going the now it quarter the in data was you to get single I XXXX. that with So to data dose multi-dose of that come

just You bit elaborate that? little a if you could on

Paul Bolno

[indiscernible]

Anne-Marie Li-Kwai-Cheung

Yes, sure.

the multi-dose cohort cohort dose or the So enrolled are over reading because step. together data we're informative important single rolled into as our And these the fully multi-dose data set decision-making. we've dose single are out next enabling and them the not

Paul Bolno

go mean as is think studies is I avoid up evaluate been on assay comparison. discussed particularly data, dose single when the in we has follow across before, simultaneously of it to that. this When to data and to critical patients just I do complete. any on assessments they you cut discrepancies mean the all Yes. I

complete Anne-Marie's And informing in with as to to next be XX-milligram calls repeat are enrolled critical so those dose of repeat shared dose step that fully having prior we data, over in the a those patients on rolled going program. earlier with cohort, the point,

Operator

Thank next question question. our line Joseph for our from you. the And One of moment Leerink Partners. Schwartz from next comes

Joseph Schwartz

we recently if from saw your of could a do merit more think bar wondering for was other informative, functional we on the if get each? NSAA have what the any? assessment. you you study? be FORWARD focusing opinion what given Embark, I what the So could then as And follow-up. XX will in And be And is on And on now what assessments functional I a success endpoints

Paul Bolno

a benefit. was a It translate consensus functional FDA's one was a functional remember reviewers questions, between dystrophin translate is think a the these -- about And that first question, think I look should the but functional the Making no. the question to to dystrophin Joe. would we like think microdystrophin I the we think mean, I great functional translation and benefit. Becker when AdCom and actually always is. it's at data I the at across we of beginning, us, for

foundationally this changing not make points necessarily protein. you end functional other exon the functional you on of the of the host better, for been going well, we're was, a at protein the we're make biology digital you look the of look of think has Star, primary I function, reason functional like that driver to The better We're North premise oligonucleotide application better, required the So how don't to saying, that's to Star. protein are DMD do on skipping, back is our create gets going is biology We're whole endpoints. at going our properties it creating and the of how there. at North focus We're endpoint the all but is do that because look disease. endpoints developing of how the can the

So to that of function. the that our then translation protein is at goal look I into on and deliver think

Joseph Schwartz

potential to you be any biopsy protein your differentiated see as profile satellite a having And the on architecture in when the muscle do from could cell more analysis, given cells that there's what produced histology about result? think evidence the do actual you of a muscle activity you said

Paul Bolno

interesting really a question. It's

So point. which obviously, one, at FORWARD-XX we the have then the longer duration, X X is static in more right, time a terms follow-up weeks, doses a of

is there the so satellite to And opportunities where located. the cells, evolution are dystrophin evolution of of see

of The thinking how know myoblasts obviously, of I coverage cells is beyond interesting dystrophin satellite biology do looking dystrophin and of that the actually be lot But to translation I at important architecture should academic myoblast to we over in look translates about happening. again, continued And being academic, get is that about onto work expand able are into and actually to functional the quantitative then muscle function. are translation the time. there us the those that. let protein -- fact at fact how dystrophin interesting that population thing, really there discussions think that you experiments endpoints a and think

continue dystrophin endpoints ultimately the of produce to when of translation a look dystrophin that should the again, see I that and lot that. to possibilities again, we of important function then the quantity endpoints. you think, understand biology for up and better the us translation the most be and But opens

Operator

Yang And from question from Eun of our comes line the next Jefferies.

Eun Yang

Another DMD. question on

there when levels, protein itself. the academic produce is do protein is that assume functionality close assess testing all you protein, I you protein mean measure -- you the only of it's So a when microdystrophin. full But mentioned like versus you to functional? there length are the supine to

Paul Bolno

Yes.

piece That's we will protein So are those mean about distribution, protein quantity, protein potential that was XX- at I the in and the about locate to our the is takes of XX-week work. dystrophin and to as is functional really the protein of onto Obviously, outside the and that's been key points the then point, obviously we the academic protein. of obviously, the produce interesting to think not time So time. this, have both we think At distribution will of functional the that's and sell. to time. more be protein. right? as the it that that's longitudinal the we think study of metric look accelerated time. just translated in protein. that's opportunities functionality over part progress, as I over after itself both a the this view, assess were quantitative but that as external cell. But at the is That's the a important over the distribution will something plot for definitely the filing how its the I cell, approval look be takes windstorm where I why talking that place the on on

Anne-Marie Li-Kwai-Cheung

the I to I would as see to can is protein, benefit. deliver logical most with just a we the as function you data, likely dystrophin And which expect to view, to be point a add, would of is native whether functional. ability in length significant really just embark from that these data the possible as close think microdystrophin questions has

Eun Yang

DMD for patients. And then you the treatment that mentioned is obviously, there for new need

seeing, what do think were out differentiated what's you file what there, commercially? to approval you or we need level distorting given of for So be would to levels

Paul Bolno

to differentiated. there we'd several be ways XX, commercial differentiated exon I for one, commercially, Obviously, is which immediate be space To entering. are the be think

powering XX. a greater within that, X%, and believe levels the time of level the treating we duration we're that are to show that We based we transcript than Exon on current that's deliver that commercial study able protein be above threshold. should and the We of see the threshold our can to

points areas to current As patients the physicians, amongst differentiation other even of of programs. and we talk there's

So to saw about from means our terms travel, X-day terms as patients transit it's Part whether in we dosing biweekly life we're their less these in in talk and weekly A, as monthly versus the already impact data having costs, of administration for half-life IV of infusions treatment. less frequent on frequent we or potential

That in a families talk to of huge and we as itself advantage. is

of So see of that truck. the in profile we stability our the

than terms the We profile of standard us that care. the of too, existing tells least at have we look in differently a pieces that in safety, shouldn't early

which opportunity shows the higher So and is less and switch more that cells can the we double we we protein, fact with in these to to our substantially see ability that are that our provide levels, what frequent was the remember to and you full I that go skeletal XX% and seen we an and satellite transcript patients to tire administration see get data, diaphram. see need to shared back muscle. knockout think data art We've in NHPs in both production and substantially transcript

protein protein So levels skeletal suffer we that think protein, about existing the expression addition to these distribution standard. of important And in tiopromatic high dystrophin cardiopulmonary the concentration. from, the overall patients, to levels underlying high of cardiac profile, local differentiated to treats profile of high the levels that complications from respiratory and what's going it's muscle patients be

dystrophin I this think impact profile, without So of our of the other year quantity same. the skipping profile the reason profile, for those and tend totality it's program skipping exon we liabilities and but the molecules that being on the existing the prior started of need of standard not is The the conjugates differentiated therapies about to exposure, and as after because this potentially the experience from people the localization, know compared all care. exon the just to modalities

Eun Yang

then RNA. AATD, question on is And last

of when mechanism prove the in data patients? terms proof sometime in about of of So you see number Can mechanism? would a of you order talk we of kind determine data to see What of deliver in data kind we level XXXX.

Paul Bolno

want study in you other I'll let think mechanism. that? as and questions talk we more Anne-Marie But your proof of updates about numbers of I benchmark lot Obviously, designed kind to to define -- the important of progresses. it's a that terms do

Anne-Marie Li-Kwai-Cheung

as medium throughout we achieve the potentially before proof of mechanism the detect that study, So significant of multiple of because first our -- be and will humans. evidence milestone cohorts, proof protein soon And serum. of MAAT trial that so have editing the into of will mechanism completion before assessments first it we can can low, in low that's as translate and cohort. very a is be And of in the completion it. as we detection additive And

Operator

the Our question from next comes [Operator Instructions] line of fee. Luca Issi from

Lisa Walter

on Luca. for Lisa is This Great.

Just a one. couple on

that mentioned accepted. been CTAs multiple You have

volunteers the can if have know share which soon. Just expecting data, I wondering And geographies healthy on SAD dose you you're cleared CTAs. to the you

mutation volunteers, from treatment these gain hoping subjects? you there's inform And what correct healthy further Given in help to are information to patients? no in what clinical AXAT will

Anne-Marie Li-Kwai-Cheung

more what expect that clinical trial see to designed And first to Well, been target U.K. to the coming. patients achieve to we did volunteer volunteer in most are us with has Australia expect your the we question to about Well, sites would and engagement. rapidly study study? enable Sure. get the from

designed speed. efficiency and really it's so And for

the of progress will also, and volunteer in safety So patient healthy the inform tolerability. study and course,

Paul Bolno

If you we think patient get which in arm, anticipate is to about quickly it, first to the where the to engaging target. initially cohort goal the very is low-dose modeled

combination of to and to and modeling across how how And clinical we tie starting quickly enroll expect to to get would where safety So and targets. is both multiple our a that there's establish anticipate translated patient affirm a over PK to preclinical to we able on programs quickly we date. be do can cohort PD, in engaging

elegant where proof really inflection patients expedite to kind as team of together those as to in mechanism of a quickly bringing engage think opposed job get of I the idea starting a Bob. you building rapidly to up pieces getting done to has to that of this part So target, eventually as

Lisa Walter

One more, may. I if

milestones. Can cadence the the the program? you a payments AXAT from us give of sense the on for of milestone Just GSK

Paul Bolno

we we've move through we can't program as publicly, say the have we break like mean think, I I can said for obviously, clinic. we Sure. milestones is, as the down

and these of in inflection these and milestones. are data then are of beyond. some payments execution XXXX and XXXX We Some anticipate milestones milestone

say. going progress is through the that move the of I see milestones. cadence path I the can it's But a we're clear to that's to team So most making, given think potential pretty to how

Operator

the I'd conclude Paul further any program session like remarks. program. to does hand the of back for to question-and-answer Dr. today's This Bolno

Paul Bolno

Thank the their want for for I devastating towards you for efforts thank also to all people this call treatments diseases. vatilating employees joining delivering morning. our life-changing

horizon, exciting a We forward programs. our updated look Have day. year on have to the on great an and keeping you all we

Operator

in This conclude Thank conference. and you, does today's for ladies your participation program. gentlemen, the

Good day. disconnect. now may You

Wave Life Sciences (WVE) 9 Nov 232023 Q3 Earnings call transcript