Earnings call transcript Q2 2023

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Operator

Good morning, and welcome to the Wave Life Sciences Second Quarter 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 of Investor Relations and Corporate Affairs. Please go ahead.

Kate Rausch

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

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

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

I'd turn now Paul. the like to call to over

Paul Bolno

and be all Chandra In Kyle the we We will our to today pioneering of to we'll our questions. multimodal Ann-Marie first compelling in on Good trials. RNA during call medicines for the collaboration progress our to on targets the our our human GSK, joining work are available medicine Kate. and Today, quarter, and HD. our thank the continue clinical us vision DMD for turn second a We're clinical up submit to programs on track execute we advance line a review remain you the RNA open morning, on enter on AATD our transformational with highlight I'll call program, financials. in for accelerating second number for Thanks, and to editing on continued of and Q&A. share and and using the today's CTAs platform. call. to then Then quarter

AATD GSK building review editing, begin a finally, then sustainable And and in on trial. RNA with we'll an progress with with pipeline. our I'll Today, our clinical update end

on the As with of This our for the I AATD. or we clinical editing for for important of candidate preparing first RNA trial imminent fronts. speak are the WVE-XXX, treatment today, multiple you CTAs first applications clinical is milestone submission

entirely the modality an WVE-XXX into signifies moving clinic. new First,

about of who data clinical rapid nucleic this AATD, our future are editing to an important We expected for in enable the proof-of-concept incredibly excited data XXX. of ability advancement validated milestone, stand clinic programs all early and field would is biomarkers patients liver This the editing RNA delivery increase therapeutics. beyond. the set with the from RNA which utilize benefit is and With in to Wave's acid probability for to for success

of speed pace. to bench the first modality path we with patients will that platform. novel our XXX human a to with this candidate demonstrate remarkably at advanced translational our from expediently reflects from target we clinic the dosing, clinical Second, fast a the Upon have

As we candidate. clinical build to RNA our each pipeline, editing efficiencies new we drive further expect with

preclinical best-in-class XXX AATD in our Importantly, supported package. first-of-its-kind, not only also data robust as is by but it's

silencing through XXX remarkable GalNAc market. editing compatible chemically is and modified is subcutaneous able on to therapeutics durability that highly delivery a tool oligonucleotides. the with unique multiple with because are potency achieve specific convenient our also of We dosing conjugation, well-validated and fully approved of elegant

the lipid nanoparticles, AATD a optimized healthy field, that and is dosing. a Among and candidate and agent. to can to approach, first-in-class editing for of thereby stable require reversible hepatic with significant for which therapy leverage For GalNAc designed continue advantage function a our AATD, redeemable therapeutic restoration we a both generate avoid excitement it intravenous is RNA have which pulmonary

far about Texas team the first-hand current market from Alpha-X therapy, current a limitations the a with expected National future, currently is pharmaceutical enthusiasm, in augmentation is to -- AATD accounting disease. of in hepatic unmet manifestations pharmaceutical therapy this market annual There of revenue pulmonary community substantial out June. to already Conference worldwide, in represents or for of the $X.X either in AATD the at billion confined alone and the need Despite major in the Our largely therapies is with Dallas, treating grow.

has with position leadership strong Our clear development bringing including under development, royalties. the our near-term And option and Wave substantial execute this and on terms milestone novel payments, meaningful a eligible collaboration of commercialization. milestones a GSK in sales receive is GSK deal, launch as well to significant to as clinical long market. and medicine, puts to respiratory us history our therapeutic in

with on on internal our As allows next designed focused ADAR repair to impact novel us restore versatile RNA therapeutics medicine and biology substantially With protein. we RNA that modality AATD, in editing, is target work wave a to the ways. of editing discovery first-in-class have

disease of addressed these at universe platform. to positioned best function they tools, validated meaning loss such pharmacological of is loss with to function, lead leading protein driving repair be silencing modality and of can't look Wave our editing as capitalize antisense. RNA we tools the of As on mutations genetic using our the targets protein and this disease, including or siRNA mutations a majority restoration

extrahepatic robust We tissues, by constrained are including kidney not CNS, have shown therapeutic in and areas, previously lung. and editing

diseases. is have druggable genetic These from genome-wide association prevalent opportunity monogenic for large with growing also there human targets studies, rooted rare insights a in With genetics. becoming medicines rapidly validation new unlocked existing available. being RNA biological disorders, are Beyond targets

sets our wholly-owned of programs. own including are forefront GSK discovery. are augments wave in data and proprietary partnered of our and which the collaboration, and Through internal we accelerating their benefiting is genetic insights, programs, strategic at investing informing from genetic our next

We within momentum collaboration building already on and Wave our GSK and are the teams are multiple actively working rapidly targets.

these expenses and of to partner XXX% pays for validation related target cost GSK the programs. Importantly,

As a meaningful WVE-XXX, the our XXXX includes has beyond payment in to beyond. clinical the to substantially opportunities balance development potential the sheet which deal to and reminder, near-term milestones add GSK related milestone

We extend to hold on virtual RNA are during how XXXX, editing. which a leadership planning R&D are demonstrate Day to will in we September continuing XX, we our

for are and translating discuss also insights rare into wholly-owned both will prevalent we internal diseases. more programs We uniquely how genetic

the we our programs. for both new depth and data hepatic indications, extra-hepatic During share next of the event, disease will of pipeline highlighting potential breadth preclinical and wave

Moving DMD. on to

week, Part a potentially Phase administered skipping XX evaluate and other and of team X initiate clinical This has will XX to kilogram track WVE-NXXX, regulatory filed our with we NXXX every authorities study protocol development assess registrational doses candidate. the of of protein to milligrams on XX of B, are per weeks We Since treatment. identified dystrophin study clinical exon sites. open-label our additional plan trial our trial and update, after XX last

in We to data accelerated of intend in file dystrophin expect data U.S. inclusive clinical them these share to approval for protein are use we XXXX. the to supportive, If

the presented high X results these conference at our concentration DMD study, reminder, they just doses, the proof-of-concept from and A the excitement exon biweekly after which where to community in NXXX XX% muscle data profile. met excitement a were a tissue the and in June, As of is grounded Part PPMD We skipping optimism. favorable for with showed safety

dystrophin With forthcoming of protein. in transcript we substantial the these the skip functional accumulation expect period levels downstream extended fully to dosing in trial, of high result

treatment is community DMD meaningful to clinical the XX provide safe skipping. a and therapeutic waiting we production know We NXXX, options. convenient, patients exon better for benefit all functional of and that endogenous option delivers With aim for dystrophin to additional amenable ultimately

build planning to can broad the following we data of exons for expand dystrophin We would a NXXX accelerate also strategy wholly-owned DMD franchise. are number positive address, which rapidly the we to

first-in-class, for the thereby HD broad development. our Disease, Huntington's advantage have promising XXX compound today, candidate HD delivery most is takes WVE-XXX, the them to reaching to to Turning we therapies of or of no allele-selective CNS, XXX brain the strata. the relevant disease-modifying partner, believe now getting is in available oligonucleotide the right the variety ability multiple of and and in HD. CNS distribute confident the in confirmed Therefore, implicated in XXX we are regions including to of NHP the brain. in our part with patients that HD. concentrations cortex We've to studies

consistent have clients. reductions impact to we response, with level the of in in adversely the the SELECT-HD has synaptic cilia, initial which are demonstrated dose and also already the placebo formation reabsorption. neuronal to wild-type the allele-selectivity. regulation phase only and compared of flow pathways protein the the down CSF sparing approach weeks. XX of Huntington selectively is knock healthy as wild-type including XXXX, to positive potentially to single X roles HDT quarter CNS, continued is milligrams It advanced Wild-type every the could promoting protein progress. any dosing doses of with in most Huntington multi-ascending trial can HTT and played With CSF And be second approximately in appearing designed the survival cause critical and of the these several XX% trial and while The to protein. we essential and initiated HTT essential of at toxic-mutant WVE-XXX, of regulate function Dysfunction expected transport,

for of of along FOCUS-CX multi-dose Given safety multi-dose be will data. half data XXX intend observed our trial development. most to in our robust additional biomarker share available data the and durable we single-dose WVE-XXX, informing believe the knockdown next XXXX, we any second multi-dose the data In cohorts phase with to the and of important clinical

the Moran, now update. our Kyle turn over With CFO, I'll financial that, for call to our

Kyle Moran

Thanks, Paul.

to financials. Turning the

due The $X.X was prior of loss Research compared second XXXX R&D revenue was decrease to in net -- expenses GSK year sorry, for for $XX.X loss quarter, was in year the the program. compensation. expenses million. million the primarily G&A driven to in in collaboration $XX.X primarily net the increase GSK as XXXX quarter prior collaborations the year Takeda GSK expenses quarter. as the under by second quarter Our the $XX.X declined expenses quarter quarter in and compared primarily prior collaboration. increased prior recognized XXXX with to million second This year a the revenue of development related Takeda to as share-based was second in clinical external under in declined quarter. under due second $XX.X million recognized million $XX.X our of the million -- were compared million decrease In expenses in slightly $XX.X in to was and of to quarter, the quarter million to our $XX.X the slightly

of investment cash $XXX to in the equivalents strategic and XXXX. of and December as million quarter collaboration $XXX $XX.X from The first the received cash ended to primarily million with We upfront with quarter our million cash compared second equity attributable XX, is in as GSK. increase the

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

reminder, milestones we Takeda our and future do under payments As GSK or opt-in our cash a include collaboration runway. not in

stated related to to milestones potential in Paul receive AATD. beyond, including have of WVE-XXX, the our candidate XXXX milestone and earlier, in development treatment As payments we editing meaningful clinical the near-term RNA

I'll now to call Paul? the turn back over Paul.

Paul Bolno

Kyle. Thanks,

XXXX event on are to look execute in upcoming across our in at seeing at I and beyond. to well you speaking forward R&D positioned milestones with conferences Day and multiple of you upcoming investor to We September. our many pipeline

to I'll it over the with Q&A. that, turn operator for Operator? And

Operator

Instructions] [Operator

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

Steven Seedhouse

comment of Paul, so study. could was if thanks delivery even profound the gene the on into morning, pretty went much you you editing in questions god clinic doses when broad update. just vivo, clinic were initial taking already mentioned data AATD rapid you'll at that in with I the Yes, on the and the the of doses. lowest editing hoping expand proof-of-concept for sort rates be from there

sort that's generate type sort what in the think proof-of-concept other pretty of you're if thing other the at you here than curious, early what study? the of editing features can quickly? -- of And doses to other, expecting Just you manifest do

Paul Bolno

stated, here proof-of-concept, get protein measurement our and But stated to think bring we Steven, goal we of call efficiency. the through, both the is, talk you to a then editing healthy as is quickly about section. human volunteer to section measuring I'll question, as before, possible the study's as as up goal then the and rapid rapid when Anne-Marie. Wonderful. treatment take But hand I

editing, about really most is indication I think, can contribution Alpha-X and of that measure special unique protein we antitrypsin formation. the what's is the important which

protein. to the so, measure be And of able the be protein, and to biomarker characterize is Alpha-X fixed/corrected in study that we'll it we'll antitrypsin how is much able also

preclinical confirming know comments production of trial in in other to think unique way a also studies don't the I of magnitude to here. gives Anne-Marie, make it you really I quality if you So want the production. of the us clinical have clinic, both design the but

Anne Cheung

perhaps in and ready add the loaded and Well, would submit we're locked final the we'll No. that CTAs on ready almost further I comment there. once submitting stages to before the -- design

Steven Seedhouse

about do for an it Okay. much And -- What's off-target for think -- editing, of the sort would landscape assessing is just just of for when you onerous? is RNA therapeutics? for can you this understanding you your instance, any different than siRNA, describe editing more an establishing margin RNA off-target regulatory

Paul Bolno

-- do approach what siRNA Yes. would about RNA potential the I standardized and think for mean I our to is think their way ability a in looking editing same exciting others. we that antisense the about off oligonucleotide targets are in what's experience we normal as and assess at

previously, and bystander editing, we you and a with characterization the and to edits for therefore, bystander as As done know see others we've transcript look highly we've edits. on the didn't come shared specific

demonstrating So new the in specific as preparation see way modality work and don't submission, that the we terms that oligonucleotides. other bystander of lot a same highly of behaves did to edits that as a obviously preempt in we for

And so, it about editing. point be idea as ability the transcript is, editing. of like to, others alluded as that mean, this kind working think opposed really you excited to work about The think and the that reversible genetic we're on the I we're onerous prospect to needs of done more as and think I permanent DNA DNA. inducing the the on not I to, of the mutations importance on reflects other I thinking

do to this, as partner, think think actually and thinking important, an approach after as about make chronic I particularly really of important GSK. devastating about the get not to technologies diseases. we apply to we it's our ourselves is go space diseases, which so, rare these you that how And contribution We to prevalent but broad just to we diseases, obviously, think

Steven Seedhouse

that is be? just Yes. you how But bit is ahead. lastly thinking selection like this be think you for about AATD, there will This and would be initial to broad of specific appreciate lung that for approach And for that. a as right demographic sort targets? patient appreciated. looking and power to early me, liver And away, maybe little I going with involvement, comments patients the genotypes of show would Any be

Paul Bolno

important over clinical I mean, and ZZ Yes. in it's I'll liver Anne-Marie I hand it but which it think about side, where to a the is you second the if both -- initially patients, the about we pursuing, is think homozygous think as lung. application on we're

and those that less And in about patients, therefore, actual where the MZ is, is the genetics. therefore, following underlying the disease think to so, manifestation of about driver And it's correcting we that. the relative patients as protein to ZZ

there's and medicine, in demonstrating the both if -- we targets editing the of demographics? Anne-Marie, the can constitution have you the to we that focused think, the think trial I genotype about terms I but infusions evaluating say, more add what lung more other for drive looking we want AATD beyond. demographics. clinical can the threshold know else for protein achieving to much on to with and period. But deal particular that is, and in and really then this proof-of-concept think of initially, that initial and protein protein developed anything Alpha-X where RNA of liver about demonstrating antitrypsin been Later, about that the this protein through translational and don't levels look, potential the it's thinking is So just of impact at even hepatic collecting broadly and

Anne Cheung

No. focusing I patients regulators. there'll about ZZ in mean, agreement details I phenotype the on more and We're think once study now, patients, you hit we're be it. for full with

Paul Bolno

population about ZZ ZZ I is the think we about the XXX,XXX itself patients U.S. if population, the think piece other is, in the by

think as a is about that, to patient population we So study. consequential there

that the the So we're to for patients find not we'll study. concerned able be

Operator

from of line the Matteis from comes Paul question Stifel. next Our

Julian Pino

on know you're On Hi. for for is Thanks our said not you by This Julian therapeutic XXX, so for questions. I area. AATD, much taking constrained Paul.

there on moving is to beyond where establishing And you helpful. would you numerous programs versus AATD color that how then, but any regardless, may provide coming in resources find anywhere mentioned targets, super working in that your curious be compelling on you in for forward? be with interested Just And know collaboration. forward else particular that's could also along? color provide allocating you're you plan other how own proof-of-concept just other the currently GSK could joint you moving them on you your I any

Paul Bolno

the the AATD, Julian. and it by is Thank protein you context therapeutic I liver really think is up liver-lung, take to the that I right, mean I'll you, at think you're AATD ultimately set protects the important beginning, but which a concerned very treating beginning, we're I area. lung. create from you the said not

And it's So we've in and optimizes data of important can earlier GalNAc, as kidney that lung, across issues: shown context to do of XX we have are our I that sets GalNAc-conjugation Day, important a the it think on that update plan we meaning done, September think we to CNS, I others. these a the as of AATD shown. R&D said absent that chemical and data in again, POC we previously functions. What and what have the number distribute do modifications extra-hepatic, we've

indication. therapeutic of about is what think, we're and within each what I unlock, tissues an of we as those think areas I the those potential So seeing expansion think

but Day, we're as as not said, and we on just about we prevalent, obviously call, thinking think share about also I R&D excited, rare and mechanism. talk we'll really previous a On more

where applications the With share number we've meaningful that we a talking this previously do a from wild-type upregulation. vivo areas can protein is, we'll apply in tissues to that here, ability therapeutics. and about that therapeutic continue a point. data to from in and mutated we're form or a developing more in what shared of -- been of in So a exciting on ability are actually on, AATD, healthy the to correction There changes in

could or The think mRNA think increase us expression about rather about a the the area, expressing produced, of to trying you're universe think by stabilize protein. therefore, increase applications deliver of even ability where and And for technology, just other constrained under which we actually think to than then about by opportunities is to areas think mechanism therapeutic delivery. areas protein lot to a lets correction augment transcript that it So trying gives therapeutic that's about that base us. really protein and broad. And an other be that of a to beyond us as fixing we its multiple

data data So we and do about and excited beyond Alpha-X growth in the editing what field antitrypsin. that are of on can sharing it areas supports RNA more those programs the

Julian Pino

Excellent.

Paul Bolno

with question, last the GSK Sorry, component.

to partner. a as for So I'd much be as speak able to love

we of that kind a we of the opportunity translational they with their they today, Anybody It think, on sharing GSK I amount -- it's see we we extraordinary shared who think I bio, investment multimodal as the the is provide evident. really as platform. were was with potential them been and say as momentum. participants by have -- highlighted genetics safe we attended an were of were to there where

editing I is one. think,

and -- Importantly, shared using of them year are as we looks as their well and silencing constrained of programs in lot data that with function last we a on it's partnering silencing for not data that and prominent some universe, across really the across the of to approach. as just editing siRNA There GSK are editing. basement loss

about of to gain job. able being not pick right either or of holistically constrained function an to really disease tool best really so loss diseases that where for can And we're think the the upregulation, function

where a momentum across there's programs, on of continue and we're lot we collaboration So the updates to provide I excited think can. to

Operator

next Our Eun Yang line from of the from call comes Jefferies.

Eun Yang

editing. of have couple on I you. questions Thank RNA a

are doing have as most program well. you obviously, editing, the RNA advanced in but So others

But AATD So close of editing you there question very data? of this second going how do you a is So one. from to could able AAT filing see your technology, about will as And think how are out question in R&D comment how should humans? increase we And levels, rodents. your RNA there you more to have terms you you protein about Day. positioned on others? data in when is think of remarkable for to at are others, be you're fold CTA, increases that's the many although we program. And lot on compared in not objective? shown be the But And better would to obviously, be a serum number talking

Paul Bolno

lot. Great. We'll unpack that a

when saying, there think, lot we of do translation others. we're like think, the start nucleic question, what in brought in entered are I'd from in translation we are from there's we from doesn't chemistry. not do in lot interested an we -- decade poised space of think acid said translate into I editing RNA And comes move data AATD And of what this out a human? I'll over was others? are building I important come ahead convergence an the together of to and of think, really editing. which which your immediacy why what And editing, this then a piece, you with It to I best-in-class when in is is why of final we you you well, engine. investments the the bring oligonucleotide chemistry to say at if expect and ultimately, that we is really a in -- front we'll how to up really see said the the AATD beginning leading I

non-human the have GalNAc, with with And trends translate and create liver you does And to do studies primate our specifically using more how How both how? that we GalNAc the chemistry-driven liver How design how we root The it that not in are in ultimately, that guidance ADAR? things, that I in enzymes? beyond in interact liver? better enhance set the require do opportunity other think to short we've oligonucleotides get really if the vehicle. that tissues I long that approach and we been delivery don't think, And get able using a to so, of exercise. we in doing the is to a

to use to we're LNP be that not one enhancing not because constrained nanoparticles chemistry So That's that not durability. times really those important and the what compatible of lipid think complicate think versus do I with be delivery potency, with ultimately a familiar an stability LNP, would focus that we lot one, in I accessibility. on just can can but do not means to and for we

able programs, we think transition about about human not we therapeutic as means translating going experiment and about a a to ultimately I infrequent terms of bystander we So editing, editing being of think being off-target we administration; on subcutaneous thinking best-in-class pharmacology editing, be avoid and optimizing it are into a and therapeutic, what's think science as but as as safe durable something to patients that's reversibility. potent in

So put presentation, that together, up-through from have whole that through as and our of we the engine in our systematically off data-driven program publication we've model. and beginning, this built profile built a way chemistry

is. how this us in you giving what's important translate, we is confidence well, what's to okay, going do Now translation, said, know

about in GalNAc, good thinking into I got that of your for think see what we're the able seen translation consequential is data appreciate GalNAc. delivery starting I recognition to-date, in human precedence is hepatocytes given there approach that, is By a agent that to we've that humans, that data into studies rodent and production. we've

levels disease to a modality lot about that because we modalities of be go we're where so, to of is, look they obviously, pharmacology we important And translational improvement start said, fold in vivo protein as to be to about translational thinking you baseline about learn both based to we we being on X-fold the And a can And of in studies results, all the new that's us from If it's And in that translation. went translation going forward. able think other benefit preclinical way think ultimately doses we to to about studies, as like to to and able as at I the about the going baseline. is X-fold the the as back via in these programs, on think range about to the the lot from patients and X.X ZZ as modeling teach start the we human with into fold off that's their oftentimes increase we X-fold like MZ. best translate think get need increase clinic. of, that The think, looked to predicting the subsequent potential.

us much the not but full is in we it's how fold in translation being that's our clinic. just move the protein the a in measurement produced as of that's of model, giving of the So, confidence the rodent ultimately, editing translating to protein production,

else, run first do to that need anything we like So study.

be program multitude tool picked the to a with we about first-in-class correction, the protein a and as editing. very about just beyond great excited talk beyond future editing. to will of the beyond to we think, delivery that talk I go into everyone, with AATD, demonstrate really share to September right it But excited RNA the opportunity we're of right opportunities the liver,

Eun Yang

a have I follow-up Can Great. question.

So you GSK in have milestones on about $XXX program. million development AATD this from

much be that? X/X So Phase expecting this of from you starting a milestone would year, how

Paul Bolno

points. clinic and But time. of contributes the terms break beyond, do, over we alluded payments out GSK is as there at the opportunities in various where number financial those got them, those that of to which milestones key is delivering What come obviously, Eun, receive as obviously, a this that we in can are question, our -- program we've we beyond statements. Obviously, are in development from disposed, milestone milestones commercial Great into payments. before,

a and host are think AATD, moving collectively another AATD. whole of beyond was are with I there feature GSK beyond is, programs that Importantly, that important

milestones. And both note, and have not them. as it's to those the we have commercial important only clinical advancing XXX% where we're But pays R&D preclinical do GSK and of those also expenses milestones, programs

think coming think the we statement. it's are said, runway not collaboration, that execution. important, magnitude inflows counted you there about as our multiple And across as So, from I those potential in are the

those So accretive position. cash be current to would our

Operator

of comes Our of line the next Asim Truist call Rana from Securities.

Unidentified Analyst

to plan Good basically My properties blocking improved editing, morning. wondering, those the design upon high-rate degradation, given being by This therapy is program, referring wondering question, patients upon potential Osman have microdystrophin you to and RNase on for with a specifically you've your already ASOs, splice the off-target competitor if Sarepta's just your for improve mediated and maybe follow-up with just a DNA/RNA all cutting, similar could first you enhanced to guide program. low guide is, Joon. to RNA that RNAs NXXX as received AMR any platform. I'm gene RNAi, stability used use like comment I'm from who've And question to Caribou.

Paul Bolno

a RNA ADAR. with and [ protein protein interaction, exerting if protein machinery that's out, we to mean, effect, involve as is you We pointed substantial laid correction HX RNA as guide exercising splicing RNAse I whether Thank or strength. with of I you. which and of think elegantly or cutting them in of and it a editing I think question, all whether and out, It's take kind mechanism all biological would have and we earlier, RNA question these we you ], generate about generate view think interacting using obnoxious a what best-in-class about kind all a building parallels the via right? the this Eun's first up that's investment aoX opportunities utilizing made protein them

think I a us set front are opportunity of multitude. that's in the

translation focused I right into now medicine. on this think we're be to going and toolbox meaningful construction portfolio with

through we've our various have you've areas. research had collaborations into exercises pushed as us always new known that collaborations history, and However, in internal

eye, editing, whether said, around or whether data beyond editing capability. that's as but not our highly mechanisms, core So DNA, editing you its that's that's not specific including, whether other

too modalities. others There what is can the chemistry always in set other have being that our saying, bring are opportunities and we proprietary approached opportunity that by to

that's very important to to building a is, close But always do. So, therapeutic think our you internal that I these in what's Collaborations can't we're using We think focused on great the behind. in capability. key have that resources and particularly not that we chemistry use minded we outlined, to present. the going that where portfolio we've the are into research say it's can are RNA all that this we our can are our put capabilities exploiting editing out and predict is payments we're I that area recognize And coming exploring we say, resources.

in as Your little want or it different think exon we where NXXX it, to forward to I gene -- we because last can approved X components really potentially of see do clarity question, which work skipping newly whether are there combined you move and combination. splicing therapy the I relative not whether bit would it's is a or

I think amounts highly deliver stable, durable conjugates into think a That as skipping. able important get exon to best-in-class specific, being generate we it's skipping substantial to agent. So, demonstrating of about cells editing, require to doesn't

think given to seeing approved think franchise. that our we've franchise very of And X in age that hope and know I for that clear building gene year X-year I important. got is the is translate olds. We that therapy X for we currently only a just exon path

within the an So, under that And only in approval. U.S. range. accelerated

So that know, There's, watching, that I we're is. on of the where data year, we're attention end it's data really the translation this paying to microdystrophin. at forthcoming -- of

saw accelerated I we while all recent approval. think So else, that like everybody

to a I or not to benefit on lot whether the follow continue microdystrophin functional. it's and think of there's

not that when we're ultimately demonstrates you a dystrophin following the protein able protein, on So create based eye not on on the we to based data going to has preclinical protein being functional ball this type delivering Becker our benefit. of I distracted protein take off think see that our be there, functional and believe

well capability about as skipping of think down the we which than different is that question Importantly, our benefit as comes our others. to chemistry, around

enigma of being trying these sometimes I about the companies a about on fairly within we where go think muscle. our all number there to kind of better think in drive And similar distribution can basis. have companies, it distributions talking exon-skipping scalar been of a

knockout What's skipping higher model and the both levels as actually our important primate, in construct is our as that diaphragm. the non-human well of double in preclinically, have heart exon

What's function. protect outcome the by important lives, it's respiratory these think and to emulation. most repairing we to is the we heart for protect making out improving muscle not ultimately as able -- boys and fixing about us change So only and sure being

the with we microdystrophin. can of skipping, about exon potentially So, do we'll in as think bring we what about for of what excited continue beyond, evolve field and can totality we forward we're NXXX and watch terms to the

Operator

call Mizuho. Our from the comes next from Salim of Syed line

Salim Syed

from just for questions, few the us. Paul, ones a guys. quick Great. Thanks

than more anything Just here. clarification

any to it it be being -- XXXX? data, maybe the sure we're folks Phase some to when be So to it wording idea also being thinking curious get shared endpoints changed CTAs with the little this data we dependent X bit or functional differently. you I'm I that just those ask but that try it levels? this be Phase release, we'll the On the call, think when in and tried the do Is Or that then is in dystrophin X are have bearing And the going solely DMD, able just ask you'll to like it now quarter. XXX, And press on is getting you've the seems able first year design 'XX? on the have trial have mentioned, is a getting registrational? in I'll also versus on whether baseline on here to imminent,

single has multi-dose curious exactly the release you so multi-dose, data was word was single additional in multi-dose, last available I release. additional available just the time side? multidose added wasn't available press release versus release, and guys curious, changed last I'm word press the So what the press in press and dose there think any this This

Paul Bolno

right. All Excellent.

Salim. and thanks start, we for if the So question,

just down So DMD, start we let think HD. order about, me in AATD, write

as as data. can we relates a terms we begin in give AATD update said, to time lines, the trial, submissions Once expectations further of we So imminent. for it the CTA the are

to think I that see kick-off. important trial always it's

for safe be I initiates. forward, study. is we'll looking once as in think, XXXX XXXX updates that won't that can what data to data that we provide move into on obviously But assume we have that it's I and something think trial when

a we have. filing functional As it of question what data on and up based you on differentiation DMD, brought relates to dystrophin

as data and very in that we a will central first as let filing, seen What we've be are, think, as us I'll from XX foremost, as we really function continue we Anne-Marie is have biopsies is dystrophin so and for that availability boys and about weeks the XX us. getting I on to is said, not study, for think comment where critical important we others, these the trigger the weeks. benefit, but just from follow about

don't And ambulatory, We an to the function measure and to only they respiratory at look have a opportunity those include functional include different number ability of endpoints. others.

and answer come filing questions any And DMD let So I'll to additional pause Anne-Marie, data. then potential around I'll you HD. on back

Anne Cheung

Paul. Thanks,

as regulatory well-trodden a dystrophin you filing there know, is pathway So for with data.

that expect would Paul we of said, But through running So, as the XX weeks. be we're adequate. course, to study

we'll, data any So Back you, of filing. that available of providing Paul. course, be the to for functional

Paul Bolno

Great.

in the key underway is mean, change. available so is multi-dose -- I that And Actually, excited HD. the on that multi-dose the data. no think the there's we the that's update, is mentioned HD, I call, on we're

to think would have I our have able data being data update. we is multi-dose the single-dose while available be update So

muti-dose very it's full for is think in to human multi-dose we safe going say, initiated XXXX, would be available, not study. expect to that's data I we that data got despite the

Salim Syed

it. Got

X is XXX my to Phase we 'XX? question was the share for trial in something on -- design. -- or plan getting that you quickly, also that this Just X the Are trial Phase year

Paul Bolno

initiated. No, share the once study I trial mean, is we'll the design

So that -- that will XXXX. be

if imminent filing that updates. So I new think about think and you

as plan, initiates. to question, that of as a data timing I lot think, we'll study provide the to able your be the share we'll clinical to trial which more clarity

Operator

call Schwartz comes the from Joe The Partners. line Leerink from of next

Unidentified Analyst

trying Joori Hi. cetera. have just the that on is you guys how guess This really potential likelihood the guys wondering to that how your RNA you to or lower? in of and for stoichiometry is, legos a I'm splicing, dosing skipping compares AIMers get I of commented et exon are index How what was similar any or for thinking does expect to dose-limited do other about therapeutic Joe. or And toxicity? on influence? and you range be at if higher, editing I

Paul Bolno

Yes, can just how then we to about beginning, Anne-Marie. I'll -- mentioned, about over of the that think We'll we take think you the pharmacology. I turn think some as the as see I and stoichiometry,

catalytic is the within able I catalytic pharmacology mechanism that a the harness so way think is we editing. better It pathway, the translational of of AIMer are to enzymes.

effect. on give able where drug point, such your component and that able come in becomes to of to able the advantages of do both So with that's cell important the stability to be we amounts And to the drug being degraded stable, that, not that then see exert smaller being staying staying an its stay.

So it are number mechanisms of that the we're is catalytic as a utilizing.

hepatocytes I think siRNAs think, a for where other GalNAc-conjugated getting oligonucleotide think we we -- and there's lot about leveraging that like using is the we're human other GalNAc, by as you're AATD potential the advantage approaches machinery. catalytic

to I development. So similar in catalytic seeing an other very oligonucleotides think act we're

we effect. more this have was more and like drug stoichiometric -- try of dose kind you to So wouldn't give an be to to exert

advantage think lot you we features enzyme we and versus that the So about about efficiency think a huge -- very this do we much as catalytic a enzymatic like of I others, --. RNAi AIMers under is think winning the other but think about behavioral

Anne Cheung

these right, a is work turnover can you this fast machineries to technology looking No, enable kinetics. foundational how benefits, to at use catalytic

that's And all at So basis. really are machinery catalytic to the these point, Paul's looking non-stoichiometric.

Paul Bolno

to important, other to being not think we about to subcu dose volumes give about think and we're extra-hepatic we And that other to having I about think routes too, as large as as able drug, particularly think tissues. it's we

be I do think ultimately, as like we platform, like should about a capabilities RNAi. lot that mean I think silencing So thinking AIMer the we other

Operator

no closing further now to conference time. at this I showing questions Dr. Paul would back our turn for I'm remarks. the right. Bolno All like to

Paul Bolno

Thank you, joining morning. call for this the everyone,

and Wave families their pipeline patients made and mission our our Have medicines significant driving in progress serve. have our we on grateful forward a position second the am and on for I We the employee great advancing day. dedication focus and RNA to in quarter. leadership every

Operator

conference you concludes Thank participating. This call. today's for

may disconnect. You now

Wave Life Sciences (WVE) 3 Aug 232023 Q2 Earnings call transcript