WVE - Earnings call transcript Q2 2022 | Wave Life Sciences Ltd.

Due to licensing restrictions, you must log in to view earnings call transcripts.

Operator

Good morning and welcome to the Wave Life Sciences’ Second Quarter 2022 Financial Results and Business Update 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, VP of Investor Relations and Corporate Affairs at Wave Life Sciences. Please go ahead.\

Kate Rausch

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

Section Officer; Bolno, Joining Head and Officer Discovery Chandra and Dr. and today Dr. Therapeutics Panzara, Paul Development; Chief me to of of Chief Medical are of in this morning release Officer. The Platform press available Executive www.wavelifesciences.com. Discovery Vargeese, Dr. and Sciences; website are Officer slide Head Mike our and accompany presentation Kyle the webcast Moran, Chief Investors Chief President Financial Technology this and

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

Paul Bolno

on program our start recent the continue Chandra us. to then the morning. highlights. Thanks, call will second today's our examples candidate Alpha-X pipeline. thank joining therapeutic I'm with who quarter, based And provide we some call continue and both to make progress share begin I'll innovative Kate. we our excited or will Kyle platform. to of updates In I our financial. Mike the turn RNA antitrypsin you And announcing We're our There and on to and editing the in potential for these initiate all and the program over platform. meaningful Good first WVE-XXX people selection most unique XXX are addressable for will as address is development deficiency of demonstrating currently targets discuss advanced editing open to biological XXX,XXX how as a with by are then editing announcing WVE-XXX finally, XXX editing to XXX expand AATD will RNA for RNA has XXXX. and modulation of of disruption how development with additional derisk Protein-Protein with interaction, ZZ we RNA the clinical serve patients of also as first-in-class US approximately monogenic AATD. to proteins new disease enzyme the transform upregulation. indication, opportunities harness endogenous treated, to in the mutations proof-of-concept in expect diseases, larger editing. for that such candidate carrying homozygous to therapeutic in an Europe, clinical as development well our

across Continued which FTD, we in now data by positioned multiple translation of positive in how target trial in CX-ALS modality. from to our engagement demonstrated continue platform investment ongoing us our guide-strands leaders PRISM this be FOCUS-CX clinical encouraged shared as and patients. our are oligonucleotide in preclinical and we the our year, Earlier has data innovation, translating

of the successful completed remain data share clinical position which from and June, We further Michael on second data to track studies we capitalized clinical put Wave from which share our studies, HD offering In well this ongoing a XXXX. pharmacology. translation of in this DMD year. three of all differentiated a following pharmacokinetic New today, half our entering strong support

WVE-XXX. creation, multiple rapidly based advancing pipeline and our novel led on We pillars by to of editing delivering RNA updates, execute including value continue clinical

our share We now turn applications for support to on I'll value also continue manufacturing. broad who an Mike? expansion GMP of our the update to vision strategic partners over unlocking pipeline of to in our advance discussions for platform call with and pipeline. Wave Mike potential provide

Mike Panzara

the And Today, Paul. of WVE-XXX everyone I progress. to in morning the or oral XXXX, clinical good the differentiated same beginning XXX ENCALS trials continued the ALS focusing our profile of We progress and the to illustrating discuss an single in call. on with ALS data FTD of will in timeframe. delivering The label portfolio track seen for with only the for with preclinical The are on you, the our half Thank robust wealth patients at in and clinical CNS for the of continues therapeutic the and update announced update anticipate with conference first the Back single the program of the and second data open with at to data the community initiate into additional this European not which in an target Network April our underway. here confirming information preclinical year, presentation Scotland. data work these engagement, translation administration clinic. of an session of share demonstrated multiple we FOCUS-CX ALS trial WVE-XXX. dose doses in related from but June our I CXorfXX multi-dose of Cure on highlighted

preclinical yielding development. establish clinical evaluated with given achieve wild certain allele currently selective times is Preservation cerebrospinal of fluid transcript as targeting of in we to of goal progressing be this and WVE-XXX, of the community, wild need with program clinical blinded or is already WVE-XXX to The critical of the type by with that in to a to protein. oligonucleotide patients XXX Moving leverage production is key trial adaptive in is objective study Huntington said polymorphism Similar type possibly only by XXX HD data. X to to the accelerating on that a importance hand from engage continues patients selective to in we time the assessments. is the starting patient’s The in associated SELECT-HD supported The shows HD. concentrations allele the a is being preclinical well preserve interesting Huntington Huntington's in of the we of dose concentrations prove and our and emerging and SNPX the have and CSF predicted CAG standard designed disease, currently SELECT-HD side vivo goal and implemented an dosed XXX the target, were models measured right CSF was first cohort trial. information to slide the concept. able to many study

the administration we generation evaluated of PSPO in compounds used first the same studies following On have we and highest which precision HD HD. the these for where WVE-XXXXXX doses concentration graph, were CSF plotted WVE-XXXXXX milligrams of observed XX the

Lastly, concentrations one greater with we than XX this the are previous compounds. single month dose, post observed these starting seeing maximum CSF fold

in Dose for amenable just and time appears different. continued that first we half could not program, skipping the share CSF precision for the Duchenne to the recall escalation of WVE-NXXX is said quite expect label also. mutations exon a in primary generation our trial patients second target The clinical SELECT-HD we XX of to may well of we continues needed clinical with reason open DMD data this concentrations HD the with doing those In so muscular achieve to dystrophy, compounds. You that progressing indicated we engage modeling XXX was XXXX. profile

in with studies of recall or prolong resulted the an a used exon at detail in treatment and knockout You mouse XX surrogate frequencies will and NXXX preclinical levels survival double significantly achievable our in and studies. dose

trial. converting clinical planning guesstimated guided Of in label These our course, levels. selection and after dose our human open escalation equivalent data target dose

essential in for with has right the of circulating pharmacokinetic compare three concentrations following fold this side study type Suvodirsen, first protein lung compared aggregation study. is hand On completed. therapy wild is What's XX, generation WVE-XXX yet plasma NXXX preliminary in therapeutic of Z-AAT can the already you one for is to our reducing NXXX see goals update discontinued slide liver escalation Dosing dose I'll liver data to from and a X of the dose single on top and greater biopsies of quarter The AATD. candidate developing WVE-XXX PSPO fourth than shows we compounds. of clear data address some disease Suvodirsen, be week achieved you from used look year. doses an give following and newest underway Suvodirsen in designed figure our this to for forward namely data our of at those now dose novel Phase were at with restoration when muscle fully concentrations to dose that disease. dosing approximately M-AAT AATD with address plasma levels to address level X/X

editing multiple overall above model, capability showed In XXX of AATD heterozygous lowering the the choose efficacy or mice was homozygous administered using the hypothesis is subcutaneously or DV the one following the AIMer from mouse of patients, with interval. of disease quarter XX, order initial profile. than to risk our AATD At that [Inaudible] potent towards protein studies editing in restore lead RNA shared one with phenotype At XX throughout we protein. NSG-PiZ was SERPINAX the serum as of was ranged and phenotype, into seven each and compounds, functional of non-human the is MD of duration evaluation a fold Slide primates data loading study. biopsies take through thorough restoration AAT XXX best inhibition established second can well shift week robust lead in the as a to over selection control of followed the higher past the dosing demonstrating of remaining molecule preclinical as AAT approximately studies an XXX of aggregates toxicology which staining restored to in micromolar space. mRNA to the well elastase weeks determined protein reduced functional in and And IND to XX%. this XX level liver accumulation measured Z-AAT be dose. editing PVS neutrophil by XX The enabling Our an four forward

slide supporting concentrations the a derived reaching with highest Additional Protein SERPINAX genotype XX. of profile was on hepatocytes dependent preclinical human primary XX%, shown XXX the in of concentration editing the tested. as high data MZ from observed donor as favorable are with

to the translate are IND see we enabling next now Chandra. demonstrate and we With each design I'll were into CTA to underway, candidates potential beginning as with observations innovations submissions call expect development summary, to continues enabling oligonucleotides, where In when activities and the continue year. XXX? complex candidate disease through clinic. that, advance data preclinical over are to to diverse with of approaches chemistry to rational of The are So our Wave’s turn therapeutic portfolio Chandra? we novel principles applied biology. generated

Chandra Vargeese

protein that correcting gene upregulation by This applications We mutations. review Swiss Recently, like RNA of driver demonstrate next is the our RNA how to protein one editing AIMer for how to editing technology generated knife, upregulate can activate for these diseases. to Protein-Protein GTA levels of datasets we and the multiple restoring control Thanks motifs. AIMer modulating vitro army potential the service program and or AIMer we AIMer few correction proof-of-concept in substantial vivo gene of use WVE-XXX today by and pathways treat think interactions, AATD RNA to can dose. the datasets I'll functions precisely therapeutics as These potential demonstrates with Mike. slides. expression our and only design of binding AIMer example by titrating, editing precise in potentially demonstrate have on

KEAPX system genes. to activate the upregulate NRF potential the use transcription factor and we single theory side before. This a a downstream shows Here system

efficient gene regulated the are AIMer, in of genes it successfully resulted side expect multiple study bottom in dependent we of transcription by RNAseq a which the disrupted NRFX key right young liver protein in by recently mice, of repeated which as hand on the of right XX properly the we we NRFX, deal slide We vitro. shown interaction, the slide [young] upregulation NQOX, XX. downstream confirmed vivo, achieve a related by efficient would a editing of on of and upregulation. set the If analysis, transcriptome that side in dose factors editing, NRFX using the in shown upregulation with saw resulting AIMer again As

upregulate disruptive target of overexpression diseases potentially or RNA require is treat to that AIMer of application haploinsufficient Another to diseases gene.

to demonstrated regulate now including immune targets, have We in ability half targets to both metabolic RNA -live motives vitro. upregulate by the different RNA several and editing

to I'll mice of Kyle or also editing systemic call target Moran, the including liver as now We studies, dose substantial across tissues, not have do design, achieved In multiple and of While delivery a vivo the nanoparticles over AIMer. we with viral delivery much turn we use GalNAc-conjugated complex vehicles, a focused to unconjugated of Kyle? adipose AIMer. EGPX need presentations RNA kidney, have today the of range tissue, our with vectors. unconjugated single such lipid on editing subcutaneous have cells of CFO.

Kyle Moran

June $XX.X cash million was compensation due loss $X.X to our discontinued related of the were million to quarter related primarily compared $XX.X months Thanks We quarter increased million. for the proceeds finance compensation is $XX.X partially year, as XXXX. AATD to short expenses based equivalents XXXX. second share administrative general expenses $XX.X XX, and This the last ended June three in external million external G&A for based for million quarter share related in in by were primarily our of of mainly from reported in This expense, Chandra. the expenses. offset includes as net XXXX Net to from ended decreases due second to XXXX. program. $XX.X in increases We second $XXX.X with compensation, and XXXX expenses clinical revenue in second million compared expenses cash, for million same $XX XXXX, to period by quarter investments. term of external program, our and the in increased offset decreased previously other million R&D

us this we expenditures cash, existing runway, enable the cash our For currently that to of our cash short-term end investments equivalents capital expect extended XXXX. operating financing, and to fund and our we will

include reminder, turn to we cash Paul? Takeda do potential over call not for the back frontline. under I'll a often payments, Paul. collaboration As our in milestones our now

Paul Bolno

Thanks, Kyle.

disruptive the we these now and oligonucleotide Over have clinic. are seeing in the past translated a innovations decade, established platform

strands us guide enable modalities. diverse to target multiple biology therapeutic with unique Our

to over genetic around In today position leading addition demonstrated splicing, state-of-the-art and advanced previously and with vivo antisense, to a manufacturing industry, significantly we have PRISM can plus we have RNA SI improve well shared what silencing medicines our These GMP editing, DSD capabilities, how as RNA company. chemistry. together become in

achieved year, third in quarter, multiple selecting for milestones second trial development of trials. half candidate, several ongoing AATD IND XXXX. an expect the studies of our we our the data clinical enabling ahead, all clinical We expect in Looking of from WVE-XXX the in including submit and three initiating applications to and goal

to open Operator? with on up are questions. we'll milestones. all call we Importantly, capitalized well these that, the And execute for

Operator

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

Salim Syed

come there's morning, guys. Good I And the now lot of Congrats in year thanks the between Great. on so a progress. and guess for question end. data Paul,

Latte through here, programs, HD do be how we program, if data third the that more programs on did or dose the time time expect the for so specifically, the winning HD ALS or And it's scenarios Thank for hoping to see much the DMD on then Spice the you how was whether you getting in I So in three fourth program, data quarter or dose CX these north? multi Huntingtin for nog Pumpkin data could just Huntington's knockdown of we see? you knockdown we single program and two tick much dystrophin are in three the programs much. to terms thinking the biopsy, production going to the expect and what are you didn't be quarter, mutant Huntington's, specify you for egg to perceive

Paul Bolno

-- with you, since Salim. in particular Thank with well, start you lay SNPX. that's But I'll we And to to I’ll this start with SNPX I'll transition Mike but as and I three, critical And your order. out. just think as ended start no

dose, So the feet all looking think sets. SNPX data stepping XX,XXX data these and at back will and be single I

indication, hopefully CX us is with dose, saw our I important we to because think which durable give going this of single target on earlier again, engagement. potent, they're they're what more year

clinical preclinical to translation like continuation to the firstly, as we'd translation, extending preclinical we think I our of see data readouts. think clinical data into about So

Mike give SNPX it important continues which to evaluation So at earlier be engagement alluded study metric other an is we biomarker Again, is type wild target that protein the single of look that had dose, to mutant driven look on It'll done to same with CX. invade and opportunity preservation to study. on biomarkers. we'll and be clinical us the

and on DMD able pharmacokinetics give have us target we'll at then CX, again, will to the first look data to continued dose in and relates target the it so And be both As to in follow continue set key multi across on CNS single at for as again, model, based opportunity shared opportunity and we over, preclinical, related for at mouse going as speak, The to Mike tissue us engagement. programs, clinical see an to where give Turn well to translation, additional able we exposure individual restoration today, engagement the a multiple concentrations target to look DKL at look on as related did tissues answer that's splicing. phenotype robust any tissue of to being biopsies muscle and exposure the to to silencing all cross questions but related that, to that's up data disease to that be look to muscle, going key. various versus indications PN it engagement.

Mike Panzara

I’d nothing label, than the when says And a fourth escalation, have going good to really much happen. that other we we've other quarter nothing that's sense patients DMD that's the the the address question much, that is say said within are of open to study, dose

as studies, the data adaptive. driven collection, the recruitment, the timing you by and basically reviews know, of timing independent The other of by are committee. They're

and little a because So factors, that's less those adaptive are But more stuff. it just on definitive upon depends why blind there. the a bit we've been those little really timing the

Salim Syed

for the I color. could If guys, up, follow thanks

dystrophin terms know a you're -- DMD I particular single this targeting specifically, the in do have read number on it's out? you On production in mind of

Paul Bolno

now the right target being in this is read evaluate this particular the to For translation. out, at yes, point time, able

to human? preclinical in knockout So out into exposure and target a from not where we're data is, the study we're now laying to really based, see Mike determined this translate the we dose see, evaluate four. the continue as think we time, exposure is are the we'll I double said, what can

So be closer. there'll we more get modeling as

Mike Panzara

we're factor, Because couldn't any And and is, remember does Salim into transcript Suvodirsen we Suvodirsen, right? get even cells, just And we see different it we the engage be highlighted, at and exciting. look be you what big quite couldn't here see we we've Suvodirsen. all from it'd different production couldn't targets, very we have to as then distribution. dystrophin, looking with if concentrations, at And going

that So that's at one. how looking sort of

Operator

Our next Lee Truist. question comes from Joon with

Mary Hodge

good Mary This on morning. Joon. is on congratulations And the for quarter. Hi,

is to Recent traditional POPs show PN related they into limited question have Angelman my tissues. chemistry. So of in that the deep the relatively studies sort published penetration backbones brain

could So versus provide and which type these you of studies backbones? and POPS your Thank PN of the in type the you. on you function of tissues got traditional some data with chemistry which distribution

Paul Bolno

Yes, Well, question, because no, to great it's across data shared, with think the CNS. mean, the we've more. penetration and we to the edition we PN shared happy yes, I I do broader continue deeper, it's have much share PN see of is past in we're that chemistry,

So D reduction backage Huntington the that's that it’s model, from striatal related is data. the the share, corporate across to data tissues, translating uptake we in deck, the

gray with So broader, be for it the is think, we application HD data the PN primarily of cortex that what But cortex, is interesting, we're and going of is one penetration. things Salim’s we going whether ultimately, question is tend be like structures earlier striatum. seen to and because that is relates that to SNPX cord, know why, to spinal I to we've looking differently the the deeper CX, do why that's

or at either. translation Mike DMD specific we've for this, to it's about, tissue as looking seen help data help, measure tissue penetration, clinical and that these deep continue We've we but clarify in muscle. as seen was concentrations So talking we're preclinical, us will not

it that's not believe And only helping that's stability, seeing engagement the with why and retention, doses we're durable. we think target helping low distribution single so and is also we at

preclinical able important. to clinical I data be from And to translate so is to these going think being it

doses And able XXX, your forward we're for to multitude the on our as being function one with as lower being editing we advantages think, think a and able to we be to I today, of was able durable first control lot apply that of being to coyote AIMer explore PN a we to to deficiency, Alpha-X able point going we dose top has chemistry share diseases. shared get a of molecules. of antitrypsin of well, advantages our to hold as

Mary Hodge

Thank a design that, may you Is follow high WVE-XXX? quick plans provide you. this very would the trial If How question. could designed? level I any up that you for be have of

Paul Bolno

I as Alpha-X full do IND we is, underway clinical said, think be we yes. the update antitrypsin as will in mean, a planning definitely studies, engaging updates, program, enabling about trial XXXX on clinical we're we'll and provide we'll

Mike Panzara

experts the I'd that been thing other only time we've our we do we have how in terms way proof-of-concept in program. is to we this in a for it help how us can sort that each the that an really as regard And accelerates say of of of to to approach community been engaging outside CNS and in

Paul Bolno

there'll and we mean opportunities. more will share I be

I liver. clinical about the excited is and the editing to to restore RNA second half function about protein approach the year, extraordinarily and able being protect think treat an community of

trial able it's designs. exciting So now field start think, clinical to have be the candidate for exploring time, to to a an I

Operator

next comes Stifel. Massoud Our with from question Paul

Unidentified Analyst

about is of expression much of a one ADAR endogenous and model? ADAR James endogenous is in so This sense compare does levels for a to thanks. much. what on your Great, quick ADAR we AAT. humans? Maybe expressed how Thanks in know And Paul. animal that how Give

Paul Bolno

model. the model, Chandra, now using multiple don't you we're if the on a ADAR the expressed answer of specific Model, have in the mean, I I across amount Standard this know, animal we've models, seen

Chandra Vargeese

we're And seen similar we the mouse human actually mouse before, we that you've model endogenous using ADAR what in data levels the are to provided And the described in human. in data. expression presentation. have very previous mouse model, So the the the

Paul Bolno

or it. of actually there not model was had old of an or not was question existing I whether the I amplified think a think question there human whether

actually those overexpression. was presented is We data. and an That -- there that not have demonstrated

amount So exhaust actually, not so the ADAR it's you you also an endogenous ADAR, correlates. and enzyme exhaust don't shown actually We've of the don't also that either. overexpression

Operator

next Our Issi RBC. from comes with question Luca

Luca Issi

Oh, then Maybe progress. And biomarker, tofersen for on back based broadly? we've taking efficacy on that my prior question. a by field so NDA capture the how news? question, benefits X also do AATD circling accepted as how will second But for both and I be again, great. so you site lungs? on NFL what design? thinking more you Thanks on to the surrogate much was wondering liver in implications your you Congrats What much. know and FDA the on a seen endpoint are super reaction ALS, for days. focused for about getting Phase on early about the to think the potentially order you, Thanks maybe that And are

Paul Bolno

the biomarkers. So establish study Phase I'll to as obviously, Mike But our start for for with it AATD X/X, be we primarily Phase and to goal then for X plasma like relates driven the done on others, dose transition and ALS to AATD, frequency, will have question. your

for and turn liver obviously incredibly serves at really as that looking up both lung advancing of program two study data I to secondly, to important opportunity, for protein, also across the One editing be that think the how for thinking now in purposes. liver. alpha-X opens we've again, data. think, then us to be two our preclinical we call I us So terms therapeutic that community broadly. biopsies, editing. to see That ALS. AIMer and validation And clinical the that to and a and Mike call, once of again, to with for and in we're One, program over to exciting the a multiple areas. for We'll question is our does I'll preclinical designing unlock About going of point things. to more the related of production modeling shared on much translation, the have correlates, do the established therapeutic

Mike Panzara

positive CNS limited. is, pay and disease like very in is FDA starting GM, that to now progress these a this biomarkers these diseases diseases, to been whatever, this are has Whenever just Yes. is it are bad CSS, attention progress development,

that out potential standpoint, watching as biomarker reasonably both the now for viewing plays idea the the encouraging the for see us at I there from should So they say, relevant how community outcomes be that but is easily predict be large. clinical likely is of and to may measurable to this we'll

Operator

Our of last SVB Securities. Foroohar Mani from question comes

Unidentified Analyst

Hi, good for is sorry, good afternoon, morning, Mani. this on Lenin Sango

Just had overview commercial XXX you. be Thank the Would us give a expected regarding you program. novel opportunity the question able there? of an to

Paul Bolno

exciting more. Yes, programs is be is I lot for needs market a to indication one But we'll of see to providing challenges look have obviously face and that us mean, kind that. what's only of it consequential a we some have at the the what's to reaction

Europe are and focus homozygous XXX,XXX in US that amenable. that are patients the has patients the our been for us, on So

the MVP yet So really corrected the XXX,XXX ZZ to beyond not, looking that there's phenotype. broader that, we're have not back even the but at to opportunity population be patients

companies. this of programs And and of seen the lot play it's our a substantial partners. patient interest, your have the communities. other So consequential patient population, in we've and translation potential on valuations other That's attractive, and community, both out terms

So interesting more able differently. we be much think space really able I it's us a people it, and to to remind broadly should also do for be to think

focused the in seen exciting we've liver. to really the That's silencing on programs So watch. siRNA targeting

be think opportunity lung of field. to replacement supplant single I will generating consequential the I revenue protein And infusions. that subcutaneous of both to our think side we've injection protein companies liver terms commercial the IV a replacement in and with seen treats both

Operator

for our Dr. now Bolno, to back turn like I'd Paul to remarks. the over call closing

Paul Bolno

focus morning. unrelenting you, an Thank Wave everyone Have patients time you. organization. for our we for exciting and and employees their grateful all the our great for And dedication And joining I'm serve. on It's call and every families to mission, this thank the a day.

Operator

Ladies and gentlemen, today's this presentation. concludes

now a wonderful may You And have disconnect. day.

Wave Life Sciences (WVE) 11 Aug 222022 Q2 Earnings call transcript