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Wave Life Sciences (WVE)

Participants
Kate Rausch Head, Investor Relations
Paul Bolno President and Chief Executive Officer
Paloma Giangrande Vice President, Biology and Platform Development
Mike Panzara Chief Medical Officer, Head, Therapeutics Discovery and Development
Kyle Moran Chief Financial Officer
Salim Syed Mizuho
Mani Foroohar SVB Securities
Luca Issi RBC Capital
Call transcript
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Operator

Good morning and welcome to the Wave Life Sciences Fourth Quarter and Full Year 2021 Financial Results Conference Call. At this time, all participants are in a listen-only mode.

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

Kate Rausch

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

Dr. quarter Chief me Therapeutics Development; a Paul and Executive Joining Discovery news and Dr. VP, Mike and Development; Giangrande, Financial and webcast and a Paloma prepared we to Investors fourth our www.wavelifesciences.com. the Officer, financial update. Panzara, with detailing morning section remarks Chief accompany Officer; in year release Bolno, President results website Medical full our provided Officer. and This issued XXXX today This news Wave’s Chief slide this business a Platform available Dr. of Moran, release Biology are and Kyle are Head presentation of

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

Paul Bolno

then a genetic leading and the Head VP including building an platform And disrupt innovative decade investment with remarks. the Today, update in to program. our we our Discovery oligonucleotides. of new Development, medicines of thank and financials. PRISM. Mike, of therapeutic and our will continue morning our clinical on provide At have nearly Paloma, all you a Kate. discuss modifications, Kyle us. our begin RNA Wave, Moran and therapeutic for opening our entirely Biology Discovery therapeutics and reflects which Wave’s I preclinical potential Therapeutics company lastly, and Platform CMO CFO, of in will will Thanks, joining The journey backbone platform, grounded pharmacology PN today chemistry, Good

portfolio chemistry diseases of advancing value such strategy of hepatic modalities core other growing our our pipeline focus, program our and therapeutics, unlock course breadth clinic we oligonucleotide RNAi. the using PN with made of RNA data alpha-X and Today, Over important as facility. we a for benefits with platform, number the three rapidly AIMers our to for studies, starting In a an our modality, will diversified AIMers. as preclinical outside progress our as into within neurology our next our have PN reflects significant how year targeted including advancing PN bringing component shown our editing chemistry such by that XXXX, are assets of platform. we as of are Partnerships antitrypsin. manufacturing well chemistry steps delivery from Clinical GMP neurology candidates, new a have the inform we this

executing interest on year be we important XXXX significant an and seeing expect are We opportunities. to for partnering

in Just durability distribution incorporation stereopure the in splicing and vector recently, tissue silencing DMD. in PN the CNS and to which publications of we announced described potency, two for Research, Acids of improve Nucleic peer-reviewed in oligonucleotide modifications backbone

significant the field by is Their article to was as a for Our journal. these platform of of Wave. our strength designated the the milestone acknowledgement and the silencing of a data breakthrough paper potential impact

of define use. therapeutic for PN to chemistry broad continue potential We the applicability the

improvement meaningful to to applied shared last ESP results modifications chemistry. PN With results year, a compelling Research provide XX silencing siRNA transgenic our with first weeks, points be we the in recent another we can comparator of lost time state-of-the-art X, that On Slide HSD while compared show model. Day as and HSDXXBXX advanced at the As X mouse milligrams out remarkable time this single activity example, per dose target. it in saw persisted kilogram, in same that we a XX%

the also our all These PN improved chemistry. of introduction as loading earlier saw prior the We at hepatic dramatically points on right. significantly discovery siRNA from are greater PRISM risk results time efforts shown to of

muscle and ALS WVE-XXX, advancing for includes for PN portfolio DMD modified targeted administered insight our compounds improve single-stranded current of with delivery targeting Huntington will candidate exon XX FTD we will are mutant potency with is duration into tissue level multiple conjugates. and hopefully patients dosed biomarkers and modified concentration effector candidate splicing without give the can collective WVE-XXX intravenously how PN us CNS. candidates and provide of to AIMers, dystrophin duration allele exon stereopure skipping These and into which and in we mediated and using With is a initially focused for an dosing and subcutaneous clinic, insight clinic. and equilibrium, the HD hepatic oligonucleotides the mutation. are on Our patients and enable sensory and expected GalNAc are in WVE-NXXX, targeted CXorfXX indication with production. the In intrathecally

endogenous including AIMers editing universe liver. broad to our our of modifications, are of targets the as short, PN, We unlocked CNS, designed Success enzymes. with RNA-based and/or in chemical our are a approach with conjugated in engage ADAR ongoing muscle and GalNAc any programs stabilized unique

clinical PN is enable distinct strands in chemically others. from engage modified RNA-based WVE-XXX, for machinery, endogenous using each guide effects for We approach editing XXXX expect to By program. and insight to that NXXX our provide share XXX decision-making of to clinical data chemistry into

the broad of potential We excited very about are AIMers.

recognized modulation in momentum and such commercial internal an open RNA in Europe facility. wholly-owned serve AATD of having protein Our build When in through proof-of-concept as populations the disruption lead Wave, monogenic represents mutations clinical homozygous growing interactions. the patient therapeutics U.S. value de-risk XXX,XXX protein-protein would additional large the well PiMZ and in people approximately significant a program Demonstrating opportunity, anticipated address as to AATD manufacturing of to with diseases carrying opportunities we GMP the we as

experts throughout on support delivered clinical we plan facility, Our manufacturing clinical successfully team pipeline. global at made With to now are chemistry We utilizing of this the X to to can that turn I’d Mike? this all capacity update for studies like product the XXXX. support we Mike partners. up of call any our now supply build our additional of evaluating, to supply capability an for synthesis on medicines over in new Panzara oligonucleotide to-date. oligonucleotide stages development that is with Wave at have innovative genetic

Mike Panzara

our generation The and modifications oligonucleotides addition programs, other PRISM Paul. PN from development any distinguishes clinical not context but first Thanks, our the of in today. backbone of candidates, platform in only from stereopurity current

fundamentally start mouse our Let’s PN I and illustrate support view point. application with chemistry data WVE-NXXX from review that this judicious human a of of data the profile the that to oligonucleotides. of of some HP our pharmacological and to alters preclinical use going am

increase first As will half chemistry durable I to and leads longer review, compounds PS/PO skipping using modified effects. exon PN increased consistently to life more muscle activity, exposure, in generation whenever PN chemistry, comparing those

when support NHP What’s here NHP The first suvodirsen dose higher our PN similar excess plasma double modified muscle protein were hold chemistry. studies. using with consistent mouse modifications model even to kilogram studies. Exon the in part amenable observed of while properties relevance into milligram larger of in a illustrates at and more evaluated in both of ability to regardless Building Exon PS/PO that in muscles. same low facilitate facilitate NHPs NXXX in muscle this muscles dose boys concentrations skipping per compared recent of The human to the a X stunningly DMD modifications and from package of administered far higher This was applies XX to levels noted of in preclinical required backbone compound WVE-NXXX to skipping. in as PN the PS/PO in in are compounds panel to concentration. targeting equivalent first described to led generation exon these and a species. to of That diaphragm of Nucleic translated first-in-human loaded at clinic few dose benefits exon approximately XX boosted utilized and suvodirsen stereo mutations multiple studies relevant seeing Research the a level, concentrations seen and and in been as concentrations As our shown generation the paper, the similar and model. dystrophin these Notably, relevant administered Paper, a compounds mouse of same we from levels. to models Acids to of addition true knockout compound, skipping a comes and preclinical other as on heart week. PN to improvements present data, NXXX is this And moving NXXX the are every chemical currently functional splicing

setting per than As in where per to engagement we lowest produced engagement. is equivalent substantially suvodirsen. through the transcript is this the confirm or of a level goal target and milligram with lower X target to milligram healthy of as At detection muscle the engagement here, in reminder, shown target successful dose not of stroke setting. tested, successful we able kilogram This dose kilogram, a dose X human was detect were

given an to and differences half label as fortunate were ongoing to our the the a escalation We already for evaluate These life given clinical dose, a profile plasma following that a substantial be concentrations estimate XX is hours is our ongoing. of label. are the to we concentrations least study, the to to life. compared suvodirsen, be than half starting less single open fashion increase setting, in NXXX in profile week Turning the starting dose In with at at open dose to increase of plasma suvodirsen. in in data dose NXXX clear a when plasma of are collected study able in and trial even compared at this we currently plasma a level seeing

is once initial we ongoing expect and in dose in trial this have escalation multi-dose dose mentioned, begin we level. As a the selected to cohort

including muscle data, suvodirsen clinical biopsies, distribution compare expect of we NXXX to intracellular where will the XXXX. We in

These in the data breakthrough. Nucleic neurons Turning exposure paper a published studies, in to with potency in preclinical studies, was tissue Acids PN-modified the including we enhanced vivo. silencing in in a highlighted, as profile oligonucleotide Research, extended recently designated of Paul were durability increased CNS, and have mouse as and similar

the focusing Focusing or is XXX targeting our clinical our of to review preclinical based of Dementia for on or and on trial. This AFTD with our study Association Frontotemporal expertise Wave support partnered study and decision on team. plans, treatment the was January, CXorfXX foundation of our data for and FTD and – discovery drug ADDF and ALS Alzheimer’s the Alzheimer’s of the clinical In candidate trial ongoing.

pleased We advanced organizations two are we to study neurological with to this these address devastating as partner these diseases.

mentioned focusing is on ongoing. As earlier,

PN to to may clinical XXX into decision-making in program. data You this expect provide XXXX chemistry for enabled and share for insight

in Finally, I Conference, allele a HTT in disease. in our selected only Huntington step highlighting will of robust study I making fueling of on the assay preserving treatments evaluating in spend and for I select of type critical discussions the ongoing This highlighted in context our recent today approach our community. speaking are the to of a growing, most poster stakeholders HD treatments. trial we type, selective to program Huntington’s you Interest wild preserving with from importance Annual developed. allele available our the team of HD be the moment from of where presentation CHDI place is the is candidate be type select only Roche’s CSF, HTT clinical the a later wild generation but HD for are measurement variety knockdown data yesterday, some working with WVE-XXX, Huntington will breakthrough in Therapeutics am In discussions HD. assay a to in wild towards use HD about

to PN provide update turn ongoing to for I HD to I’d enable mentioned, an select to data program. chemistry for XXXX decision-making on insight Paloma? provide also As Wave’s the in into Paloma Giangrande now call we expect program. Dr. clinical over and this and is XXX AATD like to share

Paloma Giangrande

Hello, Thanks share excited Today, I preclinical everyone. Mike. some am to you. with new data

alpha-X AAT to in protein and gene, dystrophin This review, is AATD lack or genetic V-AAT disease quick mutant leading lung progressive of injury, in circulation. results of or from the a form results both. liver As globules in mutation a a injury that functional point and of misfolding hepatocytes AX deficiency aggregation in antitrypsin in

hepatocytes achieving of have approach of protein disease, in is least at with MZ Z that a heterozygous editing expected Our levels or and to focused in ADAR correct are editing functional of XX% homozygous serum RNA is the AATD disease. risk to on range phenotype of in type patients or highest risk that low M-AAT wild result

of in both AAT to liver is Importantly, leverage as delivery and majority address mediated can produced protein pathology. GalNAc lung the vast hepatocytes, targeted we

doses. As Slide on loading XX Shown right RNA higher restoration micromolar controlled transgenic In hepatocytes a total XX, TBS fivefold XX.X weeks AAT AIMers we of approximately initial we we meaningful XX levels treatment Week at biweekly than milligrams dosed the with on following therapeutically of mice. resulted AAT AIMer in or per at this XX% which editing shown protein in treatment, observed level XX, have in with observed treated GalNAc for study, mouse kilogram AIMer model.

therapeutic we at clinically to see we conjugated regimen. was If look Week dosing Finally, such effect analysis as that expect in type mass protein. XX% longer of spectrometry to potentially clinic, protein circulating AAT the which GalNAc M-AAT support could wild oligonucleotides inclisiran, frequent duration less validated would XX confirmed of

Next, see had to we on Z-AAT liver model. if formation look in mouse protein this AIMer any aggregate impact treatment

polymers. As aggregates with assessed reduces time Z-AAT liver treatment Slide CyX staining and by AIMers of indicates over Z-AAT shown XX, as liver biopsies IHC analysis of on histological accumulation for

of CFO. under address these key approaches We are for are not the therapy. Kyle loss AATD AATD a very thereby and therapeutic. and XXXX. to treatment addresses additional of IND-enabling administered an gain therapy. single In and do initiate both both liver and of turn need select this over contrast, analyses. excited working by goals results I with quantitative Wave’s in AIMers quarter aspects these disease summary, with plan of results function In function initial address function now aspects creating to third disease, multiple a AIMer study Moran, potential will Kyle? subcutaneously toxicology for redosable potential the approach candidate to this support we for currently of our a We Other development call on the

Kyle Moran

XXXX collaboration Thanks, year-over-year for XXXX, of Paloma. We amendment with due of $X.X of $X.X to that in as revenue XXXX. million fourth in collaboration. decrease of compared quarter This the research October discontinued Takeda revenue which in to component is Wave’s the reported fourth mainly quarter the discovery the million in

of precision primarily for compared fourth recognition to year-over-year compared The XXXX reported received including and period to for million revenue quarter the Takeda decreased and due XXXX. development million quarter to the of XXXX, and amendment, the XXXX. the in were This XXXX. related in marketable the related external $XX the was $XX.X The for and year $XXX.X programs million expenses fourth million as million previously to compensation-related equivalents October million year of external quarter and increases were XXXX $XXX.X and in were same securities. full For for $X.X in expenses. $XX.X research for compared cash ended other $XX.X prior XXXX XXXX million driven year XX, fiscal in the by General general which expenses in editing, fourth compared million development and and fourth in by expenses in $XXX.X as cash, and of full to We other to year increased increase quarter total expenses discontinued quarter third compared $XX.X were with XXXX million of and related and and in research by external as our administrative in partially R&D $XX XXXX $XX.X in ADAR as last decrease expenses million ended offset million in the development million driven XXXX. the administrative internal December Research HD of expenses we $XX.X year. as the primarily was ongoing quarter WVE-XXX, services revenue increase fourth to was in to the accounted is PRSIM programs. and related to to

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

opt-in our this to collaboration. potential reminder, under include back the will does I or Paul. now Takeda turn over a Paul? milestones payments call not As

Paul Bolno

patients Kyle. At our diseases. life genetic is with to to bring living vision Wave, Thanks, changing treatment

built target across transcriptome versatile robust rational therapeutic for have areas. design oligonucleotide modalities with We a and different of the to platform multiple and the

our to unlocked as year for steps therapeutic program. are of for We up for across to liver PN call will inform open questions. to this as working caregivers patients, rapidly and their evaluate pipeline next that, modified our new clinical candidates Demonstrating and deliver proof-of-concept year the be CNS, expand With targets and platform data families. serve we soon to muscle this will each value Operator? our well shared

Operator

comes [Operator Salim first Thank Syed Please Our Instructions] you. of Mizuho. proceed. from question Syed

Salim Syed

the Good morning, for color. guys. Great. Thanks

The facility. are order it couple the minimal of provide in if from datasets perhaps is of a question the the should be we so that you helpful, may least maybe at data? around of more if that – clinicaltrials.gov at sounds And when level what of Just of would the I in be not timing then Paul, the a sense of and can year I this expecting look at catalysts ‘XX. to cohorts on here or order, the perhaps the we and if get least various manufacturing – second have number data get clinical me can patients on you think terms we the I folks can. a if provide understand granularity But general like know going you it

or curious am you. or here, are lines biased of like sell some a slide Thank along noted a are facility. have manufacturing? the in more in you TAR of i.e., seemed what partners peers, you and doing your just homology you of same selling having So, more towards thinking the I sort their less couple of to decided contract

Paul Bolno

evaluate beginning capability in and field I therapeutics. But multiple several question candidates oligonucleotide transition obviously, we in to space delivering have Wave, also others take as support clinical Mike. to will years the then first our it first the to but of internally, your question invested Sure. your our in I’ll building to from substantially over manufacturing, for last the manufacturing request last relates

interest evaluating, are that comes subsequent we investment that with So, building with outside.

factors: deliver we monetization, pipeline make terms pipeline So, whatever we I for ability us risk; put our financially to to therapeutics is important do is key to our think and two not whatever two, we continue is, the obviously of extending. have will protect in and one way at one, do sure our that

opportunities So of course parameters, be we will two with of XXXX. a number over those evaluating the

relates it but consistent. we timing, color As Mike? will at adaptive have come of give The clarity the to I with the time timing variable data pretty data, in means challenges points, some let can been more Mike to and design

Mike Panzara

clustering are being cohorts timeline. are said, evaluated. the are the Yes. disclose. study, – I coming a specific where And as there very been through, got – think the they I to they really tracking the the and all in same the we all a each it and we data, together overall order, various the going reviewing change recommendations the way, along threshold material DSMBs all be mean, they Salim, we they as would if including was are to making seem at we Paul They studies to as as of along are the are through haven’t they each haven’t are terms reason is

not, the for we you point that would that those And we would are and the about the driven decisions. data share So, meaningful share data next share we XXX, you make mean, I that would phase be data data it’s neurofilament, clinical expect And we to about for more going with I decision, specific have changes those to because orders. I granularity, it’s going be for Huntington, not data expect would to to mean, talk we polyGP, the we when would are guide XXX, once by and we it’s share us we just pinpoint to sharing data. your programs, will really hard think

Salim Syed

I decision I what we consider But – order that, that think cohorts? for can little the – one trials have point, of so would there at we is to we in least I is to this Mike, level decision. here a trigger, data “robust” at a that move simply three if for is you here? push can back need what call on two a would one it Is like cohort cohorts to just bit, mean, can make Is and forward cohorts it have or the a have proof-of-concept it minimal like folks make framework

Paul Bolno

because versus framing new it’s it, this traditional I and followed you I study. up I’m think, important a you’re glad Salim, how think study really one,

you cohort, as are a out get enables it nature that drop of cohort’s design then and a design when exactly x flexibility which are So laying dose. by traditional is number the data, milestone thinking you of in by cohort size, duration, and milestone. The

Mike’s point powered to a very does that in so And there decision that going that which that a on is is forward. by to having decision there imply parameter itself us program data data, make that to to make a of tell specifically is

if to a a safety nature decision we that going where is based actual know, not effects. number, robust cohort of on decisions. to the be, that Mike, make the driven be implicit enough implies that? any data make follow-up that data is by there to to It’s by biomarker based I the able don’t and on specific So is to it’s us are it letting those

Mike Panzara

optimal leading potentially to able have definitively the we be safety of mean, the biomarker on to next It’s we target this to I was maximal create, point – I a to to be cohorts going number there is say that number of dose. a clearly tolerated able is And that engagement say Yes. cohorts de-risked number be to to and a achieve have be is of study. to on going necessary that it’s to will that the – we the want say based patients to effect. based get going to

made do appropriate there. be be I it’s a – committee to initial the not conclusions. enough we be mention The add to But it we expansion able other out going going make more like to to if if of a feel that enough to be that. patients is little and It’s But patients cohorts. multiple one they cohorts a than So, study we data The at number cohort. allowed is couple one has to looking we fill larger to that before going that are said be have That’s will to would number cohort have a study a need cohort. patients proceed have multiple on DMD, two. the the of And other different small to distribution we cohort cohorts. there patients it’s just in with or two patients data see that’s to to enough a of biopsy DSMB the here

with multiple number to step take to see not. of you that data have definitively patients we – to make to So, to should it or expect able the cohorts adequate see be the next to

Salim Syed

it. the color. Thanks got much for helpful. Okay, so Very

Operator

you. Thank

of Please proceed Truist. question. question with next comes your Our from Julie

Unidentified Analyst

Julie. Good is you Thank our morning. for on This taking [indiscernible] for questions.

liver. there a one for follow-up. is developing with what or and separate both? editing of versus we company AATD genome one curious is for Dr. for because lung proportion patients Thanks. have And manifestations mostly are first lung liver drugs, Paloma one I just I a have mostly two The guess

Paloma Giangrande

Yes.

So earlier. usually there liver show the is manifestations up

tend also understanding patients it’s manifestations mostly really be patients the the present are lifestyle. and and is lung due that up those So, population. XX:XX in to My it’s younger to older

many will aging, patients move the develop, are lung patients then these as manifestations. into with So of

Paul Bolno

you I Paloma why and it, think those transgenic also a that are making focused think around as diseases we on point about these So, VZ patients. was important that should nuance really earlier

VZ manifestations. with end up patients lung those and So, liver

liver So, really those a on over those predominantly focus patients in have decision both gradation happens to starter that that not we have purposely time. patients MD but and and progress disorders, made

it’s single that with tackle out, data have There is to a that today, The As directionally, pre-clinically designed wanting you we to at therapeutic really point of that, both And aggregate approaches population. reinforcing our of liver the single lung. are lung as liver. which whether that we do it there see what aspects we and showing on on focus at now, having the approaches could were way its we treats therefore, a clear is would VZ the is inception. approach shared liver we focus means manifestations, both the patient disease translate program be

Unidentified Analyst

then for Thank very runway. you helpful. question that the final That’s on Great. And color. my is cash

extend You mentioned options contemplate the could runway? Takeda ‘XX payments Thank quarter from runway the guidance you How does Takeda. not milestone that include from of second you. would any

Paul Bolno

say coming that anticipate guidance that. have in runway. That’s plan we But on to be we without believe appreciate in the generating our We the to milestone question. those. data not as I mean we not always taken runway it consequential payments could upon

partnering we as lot activity As today, in of front, a the there is leveraging manufacturing. call earlier, said in the on as both well

as business. a active We that about in manufacturing We process. think are the

we continue that levers as see to do course the our of XXXX, extend with Takeda there the beyond over payments So, as multiple runway we position. look opt-in

It have also is XX-XX opt-in. has split, remember at not which we to it’s way opt-in. You with back just comes a just take It also that structured too also is R&D feature. other of is Takeda is milestone the And the which obviously for milestone important a that an paid to to point deal. one everybody it’s

milestone corresponding will come. And therefore, a payments to is studies, so reduction addition there in burn that for in the those

Unidentified Analyst

do excess target? what on for great. squeeze year? if see GMP would I That’s be the facility, selection more this Actually, partners selection may to criteria would one And and it that have in what possible capacity you you

Paul Bolno

was Can the sorry, last you I it question? lost drafted. the repeat

Unidentified Analyst

get GMP would this wanted possible what’s just target? would see Sure. it your at selection facility, be the And your of to an partner to you capacity for excess idea selection I year? and criteria

Paul Bolno

Yes.

a has partner way the mean, capacity. of about us fill I the partnering variety would we enable – So thinking aspects to that are

So, for of And the bring whole again, programs, of wide strand therapeutics, antigens. Wave double in capacity number the oligonucleotide be whether offsetting across utilization RNA a already executing discussions variety guide programs. we strand, in without a on our are can they would CRISPR we

really So, mission, therapies, that do lever our of capability about assuring which to following is that. patients manufacturing the way to through get excess a is on our there to opportunity access that, leveraging whole and think

excited We are new about advancing sciences.

the overcame. manufacturing stereopure which with we oligonucleotides, that done have at hurdle, which initial a was of challenging as viewed We onset Wave

we can development bring therapeutics. So, to process work novel that complex

And color year. is course so team of But hard. this additional the we will provide over on working the the

Unidentified Analyst

Excellent. Thank you.

Operator

you. Thank

Our with Stifel, next proceed. question Paul comes from Please Matteis

Unidentified Analyst

is there. Paul. for on Hi, This Alex

Just a couple questions from us.

relative for I about quick XXXX. been preclinical it like on plasma to talk think your some have Mike, I I look studies these where those little OpEx if then to financial for of at a starting you question, least could basis, runway concentrations have blinded assumptions comment you able in initial a wonder bit are patients. And models, seen wonder efficacy. somewhat you in Thanks. the you could where underlying on sounds if assumptions,

Paul Bolno

question? first you take do to the Mike, want

Mike Panzara

Yes.

DMD, So, as concentration the data plasma in open study. label you we shared, see it’s that an

basically and for clinical the make with look label have species asking versus that comparisons preclinical clinical, study, you to been So, data data. – these versus at able we are open the about

as us these as small that is yet, basically, data is a the work of available in at support in, material am to committee, to looked studies would remain board. with programs the safety there studies, of constantly not comparisons ongoing. throughout to these across and group and would lead actually have I we the and employees – something We data the pulling So, of some mean that make safety study the monitoring of reviewing across changes I

instances result that our would So tension are and to in to that there other going firewalls am leadership I to material but in lead be members, to where exposed information process. with study, other if of tight that for would disclosures, involvement we have place that’s occur, unblinded really inappropriate am data about and I exposed this should a

Unidentified Analyst

it within you the at with at you on modeling could that say way, seen least this another levels, should asking with assumptions, your your guess point be in have I consistent efficacy into going that think doses models? Yes. preclinical were same patients what I at based

Paul Bolno

think that can I I I given exactly it’s excited to on the we about that assessment. as point point, label, where was that open sharing Mike think today related it’s make data to that that just to we And Mike’s going. so mean follow-up were NXXX. PK on that’s initial

we we are and this point and that translation the affirming chemistry of I Mike non-human label. today then models, trying open make with think seeing it’s a are clinic the was rodent data seeing that done are, But to see, between as primates, and to early, We PN was are trajectory said, which of is the now great. path? I its at a least that’s to it’s nice data, evaluation to the that Mike see on also we on simply but come think And robust was more side

Unidentified Analyst

thoughts OpEx for XXXX? on then And

Paul Bolno

through go can you Kyle, mean I consistent, be to the difference. seem we So,

Kyle Moran

on can project little our generally consistent. in tail I HD But think you it we bit a may proceeding should QX. is where studies spent Yes. what be have is the with OpEx a that mean QX, come consistent of into there I QX

Unidentified Analyst

Great. Thanks much. so

Operator

Thank you.

of Mani from Foroohar SVB comes Securities. question next Please proceed. Our

Mani Foroohar

guys. how bit you me your to etcetera? to lines, do it These about investments. later from will assuming etcetera. taking to Can flow back manufacturing cash existing presumably think you tell number will your your clinical the I into volume circle surgical question. wanted for prioritize of continue Hi a require as trials, progress products, Thanks to forward partners? they additional is continue approach on stages you about products, we your little highly contractual move that, of in between absolute be driven aren’t And what be products. more terms modeling of to How should obligations capacity attract

switch – can you you immediately. So, where your capacity can

us just help that. think about So,

Paul Bolno

Yes.

we piece of I keep distract will our build capacity that facility. our to that doing think the of from I We pillars it call core that. one in one is operating the not anything

And guided recall, may through by retain model commercialization. manufacturing or that part know as that. you decayed and have original not may agreements, rights that We is capacity ourselves. as of We with we the

being add Lexington. of ability the built we when capacity the additional pipeline is of capacity the ourselves within where the said, this to able stay the footprint with we existing We facility intention were the this with facility for to to this move That existing Wave that to the to both be have a and train. back our partners. partners, originally as facility going forward, we pipeline prior and modeling on double in within actually expand therapeutic beginning, capacity potential we So, and built we there from

that in how going with going a we be our to that. to so capacity ongoing come on where to we manufacturing programs capacities with the modeling that board know work brought we built are what need the that and would complimentary that’s way that model had just to those think what mind team. So, be that could is forward. How not But different disruptive been the that’s fill ongoing, work operating programs of forward. on in We additional be excess subject is with can the those

And so we move will that. obviously, we But on having that forward. model do more share

have of lot flexibility a our got in facility. We

at We know so mean also space, one stage. of existing we we lot large a can have commercial I CMO have the with the flexibility seen late scale thing scale, capacity CMOs. and that

what’s on here unique really built clinical right. we smaller about ability think those work that I facility in Lexington, is to do the scales,

work range X, to able being is about support what’s that right facility. of our into Phase really unique So,

we to bring and by patients. more Wave make to facility. see Wave, how of to on for helpful able accelerate into same so operating additional clinical timelines, being our that external on into opportunity and manufacturing And And the think been revenue have manufacturers. adjustments additional it’s we the to potential at And the us happening medicines cost capital to time about bring doing that as work, an not obviously, offset based world the for to in what’s to

about model of capital revenue. is that, as excess a to and we additional think plan about XXXX thinking there things we to how to lot are bring that So, in for that, beyond how around how

that that XXXX. as stay we through be that more be – tuned, will the move So, a work again, lot sharing will we

Mani Foroohar

helpful. Alright, Thanks, guys. that’s

Operator

Thank you.

of proceed. Please Our Issi Capital. Luca last question RBC comes from

Luca Issi

Roche much One competitive seen X on here. quick landscape, so taking new obviously, questions. up my ones Thanks in burden analysis. the a I for with and their Phase their low-disease younger two we on starting following postdoc giving have Great. patients not molecule. Ionis on have Huntington, They are

that And on your what your maybe in also I just addition reaction so P-XX the Can more little thinking on to biomarker see Thanks how to you what looking you CNI, and are then hoping in about maybe implications think you are a urine. news in that significance the wondering at you biomarker, polyGP for bit CSF, much. the and of just program. So, the was to expand there? are

Paul Bolno

to like will a us interesting made on in they decisions the moving they these oligonucleotides on discussion best that for was, approach. and quick the can then address And who at was distribute clinic, landscape into an person the PHDI. it a reinforced Yes. belief Mike, I CNS, you. Roche to that have the sitting is it give competitive and to transition is probably it I back But, see I based think that

advance Mike that competitive share of competitive around we and up or therapy allele on when look to more side. modality, the Huntington’s. want wild on is pan the move excited about the down continue I spoke gene distinction And and to on specific in as on I and to the therefore oligos. recognizing question. at are if as mute Huntington’s an again we take selective we protein. think the hope type It’s discussion and that will see that’s significance landscape, not see do earlier, knock you the Mike, CX then sparing, type think what migration the continue And real and the we is approach this wild and toxic silencing we other protein opening forward, But, landscape, I therapy, we and to to continuing

Mike Panzara

Yes.

– fact point tell So, Paul’s echo leaf reinforced that meeting, no, again, in I can approach there population, target. drug interest I confidence you was particular this they is the younger at I particular that is least now administration, in at that to their is there they that think think the engaging it’s this taking that of in to approach are this are that, intrathecally

issue mutant Huntington. an not their a may lot aspect to type there down of discussion bringing which is is our which Now, solve Huntington point, Paul’s with not optimizing. issue, it’s issue, the of also of second But wild is that along that

to a enormous still It’s that our and an dealing patient us been focus as has a a I to challenges way study it’s discussion with. here dealing actually maybe a of lot restriction of their think big they that subpopulation in on with some and the interest. have participation of led

of is how meeting it’s patients been how groups trial and patient access. number interesting know – can there going, wanting really at get come to So, me was this see see to to the to physicians want the

with approach So, a I overall and this Wave’s offers. also mentioned about an biomarker P-XX, in but we than in something earlier progression population, the to but associated predicting approach, and potentially the more associated in understand mean it the improvement with progression the indications is had particularly patients been in CX, has feelings potential potentially the the particular has you that ALS around Involving in of population. that it these FTD want

with decline associated other more than biomarkers. some potentially actual it’s functional So,

So, relate we it in urine. in study people and included see can it’s the better understanding deeper about, a have how if we like to It’s the talk one of get the work, the the typical neurofilament. And easy. to poly-GP super would

as for guide something are a for us future exploring it’s just hopefully we potential So, development.

Luca Issi

much, so Thanks it. Got guys.

Paul Bolno

you. Thank

Operator

the Paul turn back would to I you. Dr. now Bolno Thank like for remarks. closing to conference

Paul Bolno

this review to commitment employees Have update. our Wave for our for to a fourth everyone patients. And thank the joining and you corporate great call quarter care. work day. results hard XXXX to Thanks morning financial their Take and

Operator

for call. And This participating. now may conference disconnect. Thank today’s you you concludes