Earnings call transcript Q4 2022

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Operator

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

As a reminder, this call is being recorded and webcast.

I'll now turn the call over to [Kia Telepore] (ph), Wave's IR Consultant. Please go ahead.

Unidentified Company Representative

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

President me and Chief Joining Officer; Chief Vargeese, Dr. Moran, Officer; Kyle Dr. Chief are Paul Technology Development Chief Officer; Officer. Anne-Marie today Executive Li-Kwai-Cheung, and Chandra Financial Bolno,

at the slide this webcast accompany press issued Investors of The Section to release this presentation morning www.wavelifesciences.com. our website available and in the are

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

Now, to Paul. I'd call over the to turn like

Paul Bolno

the on where to clinical of proceed available. upcoming summary a Kyle Chandra development in results will We'll will Kia. in then over you Anne-Marie and with will joining they to to a Wave our then end with DMD. I'm excited business beyond. and with all today's call of and of how thank be Good catalysts update portion focus call morning us. discuss begin I highlights the a position for turn review also Thanks, the our financial XXXX. Q&A I'll XXXX and expected recent call discuss to

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we year, clinical building growing RNA three by a and about DMD oligonucleotide. Today, Importantly, ALS/FTD optimization, patients target. our data in oligonucleotide place learnings strong can point HD, robust for and every families robust of delivering success these realize XXXX resulting and and validation was PRISM in achieve our vision target have execution. of data our accelerate a we delivered a positive sets year position focused pipeline generated all and probability incorporated deliberate our our momentum programs. Entering from clinical transformational is and medicines have to structure PRISM leading editing, life the Wave brighter of and highlighted to sets to with in each so durable ability engagement data Collectively, improve pieces position. with achieve a financial Last platform, into future. XXXX, Wave, our that a changing new we RNA for

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With call to provide that, I who will our Anne-Marie, clinical related will over updates now turn programs. to the

Anne-Marie Li-Kwai-Cheung

trials. data to DMD lead positive Thanks, today will the from I WVE-NXXX evaluate next dystrophin. our clinical and these Paul. each we delivered programs beginning steps key sets and including DMD, In HD, are for XXXX, taking with data ALS/FTD for of of review we our programs, each

in may AATD here maximize WVE-XXX partner the approaching is in treatment of the clinic. Next, to around be rapidly of positioned XXX shown believe best-in-class green also world. We well XXX the our opportunity is GSK and the

our this allele and of silencing ALS/FTD. Finally, CNS WVE-XXX, CXorfXX shown slide first Takeda disease WVE-XXX our blue selective for under in the program dark for Huntington’s to associated advance continue we the right is candidate on which collaboration.

In first XXXX, also profile, skipping. in DMD XX for and December XXXX, safety we primary next our the working This was proof-of-concept to dose evaluate designed or single we dystrophin patients and the demonstrated concentration the of target candidate study tolerability XXX endpoint. WVE-NXXX, programs stage after with muscle with but achieved towards amenable pharmacological Starting NXXX a engagement [indiscernible]. DMD to generation for both in we're limitations rapidly whether CNS [indiscernible]with a XXX as of comes as and NXXX exon receive

similar NXXX. participated three doses the to weekly Three doses studies, all of same clinical three, mg/kg and of our bi boys more XX exceeded time notable underlying high this muscle excited and of exon skipping mg/kg one, single this had received of A levels in doses excited despite multidose study week. XX It's In six NXXX, exon at see skipping even of after was open-label, every XX ambulatory We of of having received intra-patient biopsy which point. the boys portion The observe our to different taken that three treatment. in particularly three boys escalating boys the other mutations. dose trial. escalation were concentration expectations

that these be and the Additionally, other deltoid suggesting you of in those the DMD. bicep higher heart results what non-human muscle, may primate impacted that tissues concentrations were critical date and our the showed is muscle NXXX recall diaphragm may muscle than skeletal in by underrepresenting from happening in

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We dosing. NXXX These to assess accumulate and support the dystrophin development strongly exposure also with protein expect continued data continued to of dystrophin.

of biology why meaningful production time as Conference, and assess time. data next the we well we background explained Later scientific these Mike these right today at steps researchers that the dystrophin, its On Each Panzara is sharing MDA but to in dystrophin Clinical that and in Since opinion with literature. steps be does key clinicians, this discussing as the here the potential in unique discussions slide, Dr. review given moment. have leaders. also next the as point a Wave's NXXX, I'll fact December, take for data with Scientific as was the NXXX will for provide to been and

will producing with boys DMD, associated destabilizes cellular promoting In the By most a transcripts, general dystrophin full low stage, environment healthier are dystrophin. levels. do length cells and Then this This improves dystrophin even in numbers levels has long cellular very healthier should mutation start are the protein to the low of with transcript half-life, rare create turn support environment transcripts, to nonsense-mediated exceptionally limiting to produced available ability treatments transcripts also exon dystrophin low nearly to of levels Dystrophin an skin. lack environment. the short production. transcriptional the stable of and rise, number should In the undetectable to levels. as protein their not [indiscernible] in decay and transcripts and increases support number initiation more full-life meaningful start at that were of accumulate skipping transcripts start and healthy

in and this produced feedback in Over skipping exposure is support of as cumulative in and Dystrophin this persistent to feedback the creating for a of encouraging right the XX consistent system. with rebuilding data nucleus half-life levels process of [indiscernible] time in by the in been dystrophin The the concentrations by this and shown muscle are day others. loop as to exposure at compartment time exon For importantly regard. protein. well NXXX, seen work, this we takes highly NXXX loop crucial high high expression expect to increases to and accumulate the of cells has

with this with of see However, treatment. dystrophin level we to skipping, exon expect more longer

the the to a XX assess share to every Part XX This excited XX NXXX. endpoint the others, with will the and regulatory to Phase pathways as of will Wave enrolling boys other assess also will to If study we So, well protein approval initiate step administered open-label primary with The NSAA up of with safety such next DMD. doses regulators. we mg/kg and pharmacokinetics dystrophin will NXXX plan And data protein for X B weeks We as plan and week. of after we and study with as functional goal be I'm XX be weeks endpoints for restoration discuss supportive, dystrophin of treatment. were tolerability.

to timing and dystrophin to expect in data XXXX once XXXX is Part expect underway. updates and we initiate dosing in We on B the share plan in cohort more

is first was manifestations address to to [indiscernible] lung protein. our on SERPINAX development liver M-AAT circulating mutant type of a candidate. investigational restoring Moving transcripts, first-in-class and wild potential which the correct is AATD XXX comprehensively XXX this designed to treatment of AIMer Wave-XXX, which has both disease.

is which protein, over change seven well has preclinical data AAT micromolar. Our demonstrated significant a fold in XX

that functional as by demonstrated wild and that neutrophil is demonstrated type AAT we is protein elastase the inhibition. it Importantly, also restored

an would to as of catalyst clinic the in clinic -- RNA editing XX in-patient broadly. for the Wave study proof-of-concept XXX transition and field. AATD for the see provide We for Success in XXX the and for important to first the

measure functional will restoration M-AAT We study success as of in protein. this

Additionally, by in understood on GalNAc. CTAs regulators. There in DMD completion are we endogenous machinery like benefit versed track XXXX. to well enabling while IND half oligonucleotides XXX slicing studies a of of are use well recruit multiple are modality, of new we file of stage uses additionally, have regulators in the RNAi. And that programs the out XXX following nearing a cellular for path development late laid second and or now for therapeutic remain AgoX machinery, use in the and approved

started also our to about have XXX. human And pathway we're We the first sharing study to GSK work development map out in with later year. this to more looking for forward

I'll WVE-XXX, XXXX, for selective a the of mutant became CXorfXX our and the protein Huntington associated company be for remains for of selective the candidate for HD. of the the And Wave with single dose on in remain silencing briefly particularly development This our We touch manifestation. WVE-XXX, only current candidate HD XXX early lowering. in In of status allele allele-selective convinced could approach ALS/FTD. to the possible the first data much September demonstrate first as important [indiscernible] importance preserving selectively pre feasibility allele clinical XXX. as in wild and variant type in

achieve between in of XX% We've needed Huntington always believed that to impact progression. XX% lowering needs disease to we to

to range. data, single these So dose may select adopted we decide expand XX encouraging the doses, and HD treatment cohort on the the better and multidose study. XX this that milligram suggests the within of Based it optimal to single define be we effect is already data on phase our trial dose to

first translation preclinical first clinic. in to these XXXX. XXXX, became data data biomarker to show cohorts successful study FOCUS-CX In our our expect WVE-XXX of of the from share and in April We our safety the half

independent remainder single cohort, committee. open label monitoring completed the of made multidose in For XXXX, the data with the we and the dose trial, progress also on safety cohort have extension the initiated consultation

including cohorts, the multidose will be several in data which will and slide, track discussions enrollment This data which multidose set us keeps next step, We of on completed XXXX. single for with consisting our Takeda. inform here a shown the on the of half first substantial cohort of

have all or These as as year breadth important exciting In with well our ahead summary, catalysts. to promising commitment patients. clinical an advance RNA multimodal we best-in-class medicines first multiple underscores platform, therapies the best-in-class of for programs our

turn CFO will our I call now the to Kyle for Moran, financial over our update.

Kyle Moran

Thanks, Anne-Marie.

financial. the Turning to

primarily $XX.X the was ended loss program, the as to period as net Our the spend XXXX The compensation-related increased our as million million in in collaboration to from the compared of Takeda revenue period $X.X three in on months in same increase XXXX in as was expenses. by compared loss driven net XX, December fourth recognized $XX.X quarter in XXXX. for XXXX. million our clinical We increased approximately for well $X.X million same year-over-year

our compared in the increased million expenses quarter $XX.X CX in external R&D increase $X.X compensation in related same million of decrease primarily and spending program program. $XX.X expenses increased in and XXXX. XXXX to period million to to related expenses, the was AATD, in fourth a as This DMD our partially offset HD by due to

equivalents General $XXX.X in expenses in last $XX.X service fee of and fourth million to as million quarter $X.X as $XX.X to fourth the year, due increases primarily to cash million cash XXXX. ended December with XX, XXXX to and compared million the expenses. compared $XX.X increased as million administrative We and professional quarter for of

became fund in provided cash operations to with QX will since of where to be upfront cash effective GSK cash we Wave. our XXXX equivalents collaboration expect million Accordingly, and Additionally, sufficient XXXX. $XXX strategic and

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

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

Paul Bolno

the variant we our align we deliver Huntington the or is mutant lowering year expect stage the sparing editing, this and Kyle. for With RNAi. multiple that best of target am to offers platform approach, additional value with expect wild the proud be a with candidates. how oligonucleotide we're first only year. half program and our I HD clinical unlocking of on approach, ability slicing candidates while or data continues through team Huntington the to to with silencing the pipeline on right resolute silencing ahead, WVE-XXX Thanks, associated In which execute WVE-XXX to clinical focus ALS/FTD. CXorfXX selective programs, approach two in advancing antisense

a have programs DMD be successful best-in-class discussions is WVE-NXXX unlock announce in initiate these plan We'll the XXXX determine Takeda. the with to partner these would and would the B franchise. and successful, demonstrating substantial sets for to next slicing, potential initiate data if our step With We in in space. approach to Part value dosing XXXX. high DMD If data in data

In as in addition expansion for potentially a most With and approach to DMD, the diseases future we as CNS. well see WVE-XXX opportunities advanced slicing for editing, other is muscle best-in-class AATD. candidate

We successful, second of unlock if this in potential broad of this expect of half the to AIMers. year, the clinic transition the WVE-XXXwill program

preclinical modality potential best-in-class up and RNA CNS RNAi, medicines suggests and be this that platform liver, a with powerful shaping have silencing With beyond. our component applicability we our is in to data the with

beyond programs to new preclinical share RNA advancing in rapidly announce expect and activities and editing this are and year. We data discovery

activities GSK innovation collaboration advancing for potential to inflows year this of XXXX great and already medicines to PRISM of are we patients. be another progress towards the with with course over promises and bringing Finally, year beyond. additional cash

the now I'll it over Q&A. operator that, for turn to With

Operator

[Operator question Syed Mizuho. first comes with Instructions] The Salim from

Your line is now open.

Mike Linden

taking our Thanks This so for morning. I on Salim. much just is on good Hi, WVE-NXXX. for question. guess Linden Mike

milligram forward provide three XX Part can plans any Is three the first you maybe there on patients? on this So saw patients? for here to we the patients dose. the are update the A, What after starting the these six follow-up protocol for at going weeks

that that? And regulatory going maybe forward on the then about should Part B, timeline to I and pathway follow-up we potential guess with be for how thinking

Anne-Marie Li-Kwai-Cheung

Thanks.

looking So extension. discussing boys regards so Part includes Part paths B today who the the assessing trodden at we accelerated be our regulatory enroll as also for pathways, this is approval alluding Part with there we And will XX the obviously, to we to data, dystrophin. to, of were With regulators that have in originally in us. to you're are have pathway what A, B boys enrolled up for regulatory announced the basis on they opportunity the and our well best when

Mike Linden

it. Thanks. Got

Operator

our by next stand for Please question.

Truist. Our next Lee with question from comes Joon

now open. Your line is

Joon Lee

updates to sometime thanks surrogate discuss impacted How be of questions. have of happening these couple hosting for is as the Hi, your dystrophin light decisions neurofilament And development think do directly biomarker clinical I programs. would for endpoint. as a to that likely Adcom you month follow-up. One taking is FDA impact And supportive the chain And Adcoms The and a a the a strategy potential today discuss for the is other DMD. for biomarkers? your quick next how programs? will our these

Anne-Marie Li-Kwai-Cheung

Sure.

would highly supported, new we ADcoms we'll those of therapy. encouraging will the obviously, approval that can't So I that I be, by of be how we of of us. to of Adcoms outcome the will And say assessing is very what outcome therapies, the is outcome And closely. biomarkers own any use course, diseases watching but our it these of and rare aid impacts state the course see FDA the be when indications supporting the still the

Paul Bolno

second your it the in Joon, on the think we the think of But discussion, I one think the direct exon point potentially consistently on to, as dystrophin level. micro dystrophin. skipping based is, Adcom be accelerated on think added mean, on I interesting an continued what a nuance approval. support approvals for dystrophin the I regulatory I endpoint will field

biomarkers I as clinical the info. these registration continue see again, to leverage it's encouraging So potential agency think, to

Joon Lee

XX, to in milligrams exactly program, but wrong, the MAD to confident prior XX Huntington's looking the looking SAD you. the regarding with to feel studied achieve what you already I'm to study? advance you were the you Thank Correct additional -- And Great. me are before are enough study? SAD and if XX study

Paul Bolno

Yes.

that the establish I follow-up them of multi dose expansion read think the progress? the are for I to in regimen cohorts think help that's dosing the going with

Anne-Marie Li-Kwai-Cheung

Yes.

to that we cohort. continued cost right and the you into Obviously, dose those reported are explore you we multi so a for that selecting to sure want when original that you further move dose be the into before doses the So move study. multi underpowered was we wanted dose,

to and the follow it's development. logical So the way drug normal

Joon Lee

Thank you.

Operator

for our comes with stand by question Stifel. Please Paul next The from next question. Matteis

is Your open. now line

Julian Pino

Thanks Julian This for Paul. for is so taking Hi. much on our question.

specific On much. to the any hear. CTA? a that of from for there specific clinic? AATD may if there's it's given of be is program, So be required the its agency it first great so Just the or would Thanks the ordinary the out regulatory kind any technology or requirements, other entering anything ask

Anne-Marie Li-Kwai-Cheung

They'll No. route filing filing. There are just for no normal specific the follow or a unusual CTAs. for requests CTA our

Paul Bolno

stepping engage I important mean, approach using I RNA of with back, editing. And our unique is an think endogenous this to oligonucleotide of reason

again, So other what's to base as think to a space, [indiscernible]. said the our been of has AgoX editing for for used to space. bunch we've DNA enzymes editing question based the endogenous I obviously use on approach think I whole very against with RNAi acceptable predominantly the refers But approaches similar

so following a of RNA we're And well path driving therapeutics.

Julian Pino

Great. much. Thanks so

Operator

question. question RBC. Please our stand by Luca from The next comes Issi with for next

is Your open. now line

Unidentified Participant

our for on questions. Lisa is This for Luca. Thanks taking Perfect.

to into [Nylem] program? it Nylem if fair your chemistry, like have a humans, here. is on ligand. is the program CXX their mouse would using three Intracerebroventricular data using (ph) question that assume It is use you Just were to the we is delivery [intrasaccular] know on program. part APP delivery. you move I this a But for Also looks (ph) like

the here? that chemistry for you So program you're using us for can just remind delivery your CNS wondering about if

would is much Is maybe just an you appreciated. How here? Any finally, your of differentiate of indication we be it type there bigger of? are color to play any planning picture aware a question. that should technological Thank be you. differentiation siRNA selection platform? more Or And

Paul Bolno

look, lot began we this that continuing question, the great. building of year is appreciate obviously, on. work to That's on we're because build And I a last and,

So data I others the into [indiscernible] space. recognize the again, [indiscernible] patient. using value mice in data that IVB ultimately think in pretty seen with obviously, to nonhuman have primates We mouse from to the dose. case, But and do delivery, of from And CNS for at mice. translational history a ICV your and in we've that's and question, standard long first the translation looking on we starting data that

if to it seen across I doses par, brain the with is, We've administration. our regions effective others than think potentially impressive engagement ICV what more of shown. own better have all not, target was us

where we I think of these know drugs target questions there's CNS. up these distribute I in a that's come a lot potential as question always because think I implications. important about do think

think effect But I ICV we've I be to able others, what frequency. injection dosing sorry, with with know it's is the reduce that experiment when is first we IC to – most seen think obviously, encouraging broad critical it's an think to it again durable. potent. that's function see important And relative about administration We importantly, durability CNS means even the because,

see exciting we’ve initial something in from and the So of by to again we're interest has that continue to a data, number encouraged more obviously it's work parties.

Additionally, distribution. chemistry that to asked chemistry and piece unique think, developing the around to along we've PN think about you a was that, I was, all question been what I that

that's potency where durability that RNAi. chemistry, distribution unique that again, then and utilizing we've And gets differentiation. our the see been with able So to

the durability, to lets as with space of have and to think And so AIMers validation. expanded in that be liver the that the us both lets in an potency we and able It's to GalNAc approach unlocking the end for really of in distribution, with addition I alternative the target that think again, about said, beyond us in power have pieces, our ability been building universe. of genetic target to CNS target look at conjugated the we've think differentiation focus RNA within to about editing potential one

get space that development. the targets a in insight into make So unique in the we to we'll

of our targets about applicable last and more the RNAi see be with days chemistry would can to to this and use class that the as said have continue open powerful chemistry that showing early we or target explore. to again, But best unique be able this PN a were first extension later again, up year position that to to to us So additional tool in opportunity it And broadly provide the think this for year class. we'll potential to silencing. a in year in being either as of in updates we on put

Operator

our for Please question. next standby

Foroohar question from SVB. Next with Mani comes

open. now is line Your

Unidentified Participant

Mani. for Hi. you Thank [indiscernible] on taking our question. for

to expansion in regard every that in on moving programs simultaneous we to be to other on this the first study is of through there see the pushed collaboration, development potentially year? clinical the should could number broadly, more program of human development we expect Regarding the GSK RNA in alongside Or expect given collaboration, GSK? that clinic a how first, limitation of quickly so derisk terms XXX indication? for follow in could could And editing,

Paul Bolno

again, for your I'll And it in them between was question. case you pause unclear. Thank

human the GSK. to our meaningful in for going we're Wave for clinic. whom I most inflection with for as importantly year medicine AATD patients accelerate, think you we announced first this yes, begin this will an that with And with initially. this relationship translation the exciting know, is So, to study And the developing

to And the is this collaboration in the point, underway. said discovery other research I substantial I of portion your think collaboration. the and is of this portion the beginning,

selected. did And we those where targets say they are have we be There's than nominate. validation, on deal, what select a the research so, has working and ability programs to get we called, the said more this target could we that more, announced as that period

the collaboration exciting programs, at of access to start that to targets insights the what's share working opportunities other is, those is the working we this keep on, And collaboration. so, our the of on least per I begin But expect And all some for side that to momentum programs. of but progress se, us you Both not able with on. side although disclose on together, the that course are enable programs more be our importantly, we genetic to targets unique may where Wave we to to relationship. of over on the on distillate together the progress year, be able the our these we are will I working And think understand updated initiating GSK target. to

[indiscernible] a collaboration. really multi is it So

a off think great gets to excited the of potential I about productive it's translation that program, driven start. the And first [indiscernible] obviously, our on of clinical progress. all

to just your make just answered end I've sure pause of the question. question we lost I'll last your tail because

Unidentified Participant

you. on AATD? a in a follow would foster there regarding a I be for those, Thank just approval pathway population to guess as subset potential of

Paul Bolno

are treatment I programs that's on we think which our in actually about thinking something off Again, interested exploring, of disease we developing Alpha-X protein. So the GSKs and case programs antitrypsin is, importantly and clinically as if Alpha-X the a for about that the that that it's biomarker. biomarker select of biomarker have you within we're validated think of obviously, we it's an is factor how of -- the deficiency our deficiency pipeline, deficiency, antitrypsin important

potential you restore registration. protein functional and the for that to levels can If back that creates

we more both how as potential interested in a that I broadly about So commercial. the strategy, optimize to think are that biomarker thinking and utilizing to continue to GSK but then build

So and of I our we're with work lot to to think opportunity. opportunity a for there's maximize AATD excited partners that

Unidentified Participant

you. Thank

Operator

[Operator Instructions] from Lee Truist. with Our next Joon comes question

now is line open. Your

Joon Lee

Hey, thanks for taking our follow-up.

can same of about just sRNA's many things Regarding can do the do. the program, that sRNA angiotensolivos

the but And superiority ASL the your one the So bit be question, some appreciate versus Thank opinion, do Just how adjustable between discrepancy perceived are platform? differences may a of thoughts [indiscernible] you. other targets you in would appropriate the for of you a size in think thinking of sRNA? it's diseases there. valuation which about versus more loaded or your

Paul Bolno

you I question are around the and assessing first all better that take last [indiscernible]. am valuation I'll your differential than say between much

companies the both are possible both that platform. trying to for time and in a address they're unmet been their long utilizing think need as space have I best

what think to I set which from beginning does the is this approach, capability Wave by be do, restricted RNAi not what technology I the think by having first we to up modality. class your particular through in best do question, we what inside to believe a is really

to what think we well, qualitative. technology that's the for us for creatively I now ability everything tool? different say, think not establishes the the of have, the optionality address to if target have way about with it looks really and hammer is like to the best for the indications the a the silencing this us I right best think separate because we opportunity but and thinking you're opportunity for what's tissues, about given

that's think opportunity I set. the real

the I right a really we was flexibility one think drivers really that for we're could to Again, universe. the on unrestricted excited work but Where AATD just like others to ingredient, to and again, can for is, and indication. protein RNA to it with about were think We protein RNAi about restore the we biology, and it target why actually fix the are GSK medicines great target real a editing. usually was used it fixing by not of it, as have doing. of think that the comes right approach best example is and RNA silence generating more correct it function, through thought of giving

think to set meaningful I for really best so, these important hand, us say having on I think deal success. And approach diseases, really with does future to tools our up

Joon Lee

on sRNA? see you do we'll update think first When

Paul Bolno

said, that we usually we programs data. share it's R&D updates big know, on not we platform. earlier, days And on you historically, both a then a have as target As updates typically think update, of when I by we're shared list just substantiated done going to importantly, with as give is broader programs, we

progress share to later excited this So year.

Joon Lee

you. Thank

Operator

turn show Paul back no at further questions to conference the now time. for I I closing this would Dr. Bolno to like remark.

Paul Bolno

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

Operator

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

disconnect. now may You

Wave Life Sciences (WVE) 22 Mar 232022 Q4 Earnings call transcript