Earnings call transcript Q1 2023

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Operator

Good morning and welcome to Voyager Therapeutics First Quarter 2023 Conference Call. All participants are now in listen-only mode. There will be a question-and-answer session at the end of the call. Please be advised this call is being recorded at the Company's request. A replay of today’s call will be available on the Investor section of the Company’s website approximately 2 hours after completion of this call.

to Officer. call like Pfreundschuh, to now Financial Chief would turn over the Pete I ahead. go Please

Pete Pfreundschuh

Thank you morning. and good

of Joining reserve me Officer. brief provide on this available our from QX CEO; on today's highlights financial summary morning. call the are Todd XXXX Q&A. quarter time We and the our of Dr. The Carter, our press for key Chief XX-K press website. your a Scientific results We majority release our Dr. issued and release Al are Sandrock, to plan

a I Al. In moment, call will over turn to the

remind Before may uncertain I expectations, do everyone cause plans noted by and today's that this, that and results to actual statements, include these forward-looking and from may uncertainties forward-looking risks this I in forward-looking representatives want prospects. subject future All those inherently Voyager These X of to desk. differ call, as Slide are are materially to indicated during make statements. forward-looking statements statements

review material filed with encouraged in report XX-K Form are Company risks on the SEC. Company's understand described the facing to the as You uncertainties annual and and various

risk are As described report. website. quarterly filing of the Form our on there in been available filings on the no the changes All to report SEC have annual material in XX-Q, today's factors Company's

it my the Now, pleasure call turn over to is to Al.

Dr. Al Sandrock

happening I'd Thank a We you, innovation Pete, in in take in renaissance now to like X. moment we turn morning, a recognize believe everyone. Slide and witnessing gene incredible right good neurotherapeutics to are to the neurotherapeutics. Please therapy. and

accelerated first was approval disease second modifying the approved the for drug ataxia. therapy disease year, this received Alzheimer's Just for Friedreich's and

which approved something symptoms therapies. of seen have treating breakthroughs are no We for there schizophrenia, in negative

oligonucleotide accelerated ago, approval the for FDA antisense Just two amyotrophic lateral weeks sclerosis. SODX, an to granted

approval. based and devastating I finding a accelerated biomarker the disease. reductions for precedent FDA drive will further predict benefit a neurofilaments hope for clinical likely treatment new patients on driven the based the this path ALS in from SODX patients, are treatments approval reasonably that to to Importantly, this establishing suffering

same gene field the coming time, age. is At therapy the of

seen dystrophy first hemophilia We PDUFA And the a see muscular offering B. in Duchenne and recently may have through approval therapy that, therapy. of approaching for important hemophilia utilized accelerated gene potential Gene treating dates. the are approval we for FDA advances therapies gene the path

transformational. long-term data then approved gene durability first years, one effect X.X Zolgensma, the after called of recently Importantly, on of which physicians therapies have

CSF inject challenge. very has into date, advancements and uniquely platform is therapy Voyager proven fields. both sits delivery we or have approach the To of successful. brain to the neurotherapeutics in various believe we our at been the the solving parenchyma and nervous leverage challenging, to of gene approaches delivery this gene intersection the are system positioned therapies central the Voyager to TRACER spaces into of therapies discovery these foundation not

engineered AAVX central each intravenous transduction libraries, novel have variants than capsid capsids that increased with more scientists nervous multiple Voyager in those select delivery. and novel AAVX capsids greatly the following to system display 'XX of

and own an the where advance therapy medicines, neurogenetic of partner's these trials. to future our how several advancing We have from which is now sit, gene enabling clinical exciting is our of leveraged This capsids I Voyager and to be. it's place programs, are CNS towards incredibly

briefly will Moving rationale. our I review X, Slide to investment

compared the the more capsid gene discovery demonstrated have capsids. our platform, Our capsids expression discussed. delivered first I studies, AAVX pillar XXX total conventional of value brain preclinical In which novel is in just higher our to TRACER than trans intravenously

capsids to ability and cells, cells. sharing root We have and look targeting our doses, have such later We dorsal at to specific of this ganglion high we shown neurons low as month. liver on in the ASGCT CNS gene and while forward levels expression glial at the more demonstrated data target

of Our is CNS second pillar our pipeline. value

We our Alzheimer's ALS. anti-tau these wholly-owned, programs are towards are therapy and Two research program of for through for programs disease antibody late and humanized IND. four advancing SODX gene

program therapy being GBAX co-developed and The program Parkinson's our gene ataxia other frataxin are gene Friedreich's for therapy for with disease two, our Neurocrine.

pillar of license Our value partnerships. We third adoption is and Pfizer tax Novartis. completed with agreements and

collaborations We have transactions. executed and pipeline we Neurocrine, strategic more around programs our exploring such with are

make X, Turning highlights. to CNS Slide a to I'll pipeline. our few we continue progress recent advancing note

continues the this treatment we at the March, presented the antibody disease. from of written XXXX. lead the a In Last feedback Voyager VYTAL month, to meeting enable XX selected the quarter, to first received an candidate. IND anti-tau initiate studies AD/PD highlighting this in differentiating half we characteristics VY-TAUXX. FDA expect new of During year of for candidate data we humanized to Alzheimer's first lead for pre-IND filing toxicology the GLP

second the quarter moved first has That the we ALS said, half year. to previously we to out this therapy this year, development continue this change candidate. program on was intend candidate. Another for provide we in expected the development data program our of to this to guidance updated IND this a evaluate SODX the Voyager select timeline to the from the to program. once We as lead half optimal development for of gene identify candidate identify timeline

in expect are occur today, we IND Given we mid-XXXX. the where to

advance GBAX for therapy here. making continues and with pleased other Friedreich's therapy program mediated Our we both with collaboration our ataxia gene program and for are Parkinson's Neurocrine. GBAX to in the diseases disease I'm progress frataxin gene

knockdown HCT vectorized of Huntington's two the novel combining tracer of siRNA our disease. stage new two early during programs we MSHX first quarter, therapy capsids. mutant treatment enable One and the combines Additionally, gene intravenous siRNAs, for with launched to specific

the siRNA reduce Alzheimer's uses for in to the disease. brain TAU expression treatment other The of

now discuss call over our to Pfreundschuh financial quarter. for to to the results the turn like Pete I'd

Pete Pfreundschuh

you further XX-Q financial I will for release to Thanks, today points call refer on our Al. cover press and and some issued details. key this

Novartis Neurocrine from $X and agreement the the million to We revenue the $XX of QX collaboration collaboration XXXX option agreement activities. GBAX Please $XX.X for XXXX composed exercises $XXX.X the X. from Slide of program. turn announced collaboration Neurocrine XXXX million from million, million

driven to was by increased related R&D expense facility headcount, by increase increased spend, decreased $XX.X costs. offset XXXX, as of milestone $X.X Our million, And program and million an fees. QX R&D compared

expenses the for result $X period were the XXXX XXXX. increase increased million compensation compared million first driven of was G&A headcount $X.X same of in quarter in were a The as to by Our expenses primarily costs increases. G&A

income had revenue increased from our of As other the in revenues from $X.X a result cash net XXXX, largely increases of in and marketable $XXX resulting QX million, strong well interest primarily revenues as as collaboration to million Company due income of securities.

securities cash, XX, March and reported close marketable Voyager of the in at Regarding the cash balance equivalent sheet, million XXXX. $XXX.X

resulted a by $XX the XXXX the of Neurocrine the pro in $XX the million cash node from offset new from revenue also option to quarter. equivalent Of securities close into totaling We had million of the payment at $XX.X receivable related $XX.X to received primarily marketable increased Novartis allocated recognition million, Together, of this three the payment the million upfront deferred options by April. forma revenue cash, in of and $XXX.X from discovery programs QX million close Novartis' for exercise.

our has a revenues. balance collaboration by enabled sheet non-diluted Company The same

will capital into reimbursements We be expected and plus expenditures expected XXXX. planned operating balance sheet expenses meet sufficient our our to

turn over will I the back call to Al. now

Dr. Al Sandrock

Thank you, Pete.

Slide to Turning X.

with $XXX productive year collaboration to Biosciences, by you XXXX. start We two by upfront neurologic began payment. As million can triggering the had has securing on targets, in decision a OPT-in see, followed strategic Neurocrine another $XX capsids payment Voyager million to disease Novartis's

further our These us enabled platform and transactions pipeline. to strengthened our advance balance sheet and

candidate programs Alzheimer's candidate disease development disease. our a a for new development therapy stage for selected early we and aim program Huntington's launched discussed, this We year, As SOD-X antibody anti and the later gene Alzheimer's we for ALS disease. for to select two program

Vir, most In entire I'm of Board having as industry, accomplished press a strive to is announced George neurogenetic for leader our invaluable one of this George welcome Biogen we while that of companies thrilled experience to will creating addition, we in His executives patients us biotech release the shareholders in medicine. served Exelixis. as highly as morning. value Directors, to and biotech develop in CEO therapies to vast be Voyager to into our Scangos innovative deliver the building

Looking break and our work neurology. forward, barriers, gene the of fields the through therapy constraining to we continue

continue generating scientific at conferences at exciting will the later are to ASGCT month. data share this we including We

value Importantly, advancement pipeline our view for as creation. leading is opportunities multiple towards what we

and look collaborative towards XXXX establish capsids. IND multiple anticipate and shots we XXXX, or and importantly, translates begin payments several on more clinical the into As to as and This for even potential for to novel our wholly-owned proof-of-concept milestone goal human we across earn programs. our multiple opportunities trials license filings

to these indications. to great partners In engage to I our potential summary, to the impact very incredible Furthermore, with as disease And it discussions course, CNS we start active XXXX, all of continue biomarker continuing of difficult around a platform and see there in and due based to Voyager early always, evidence year. is look the throughout forward in our momentum pipeline. team. potential been of some it's is

take that to you happy With Operator. may we're any questions have.

Operator

[Operator question of today have first The be will you. Instructions] Olson Thank I Oppenheimer. coming that Jay from

Your is open. line

Jay Olson

the therapies? your be just interested how thank therapy they tau talk in potentially with you versus different Thank to the anti-tau you and a on initiating Can just vectorized of questions. development anti-beta anti-tau and do do you. program We're all targeting you're an tau independently tau could advancing partner? you anti-tau knockdown think our off then be tau, you'll therapy taking you're you how therapies the about a and that advancing strategy balance all gene modalities? monotherapy can seeking optimize your you'll for the are on best eventually, or and a work then trade Congrats progress, about antibody finally, of you have doing think combined prioritize talk can And And antibody.

Dr. Al Sandrock

Thanks, Jay. great questions. are Those

for, all, tau you antibody of about a like tau. So that well the target and what taking and multiple validated first as against an target. for Alzheimer's domain we're very and terminal it's out, pointed of And, we believe of approaches C so important we that, we against disease this because is have

it with see to of spread very development or PET looking see antibody we of we, the there scans. not words, patients we that not one the not proof to We can over and The and with move at do block can In period. is this year think or is many whether by we concept. whether a forward feasible tau strategy passive tau other first get

we proof concept, program. we have, tau, through a obtained can to initiate we antibody approval antibody and proceed can continue all we there. way the If a with couple of the of we then choices to then have or anti-tau is to vectorized One

of be crossroads sort that will we would that there. So the see

it against And on tau very in In to rely have vectorized sense be it different the extracellular knockdown, tau. is important will think we target. an a that binding shot this we addition, antibodies which additional doesn't on this

saw expression see meeting to pad tau, were of on of at This extracellular. able approach we tau the is imaging. Biogen the to and Ionis against intracellular And they preliminary evidence the some antisense of forms AD/PD that decrease approach where all dial with effects

So helpful it's target. against think can rapidly having And this target. clinic, We obtain the is important very approaches a advantage we PET imaging. in we multiple of think tau proof-of-concept think taking pretty we

whether And we The proof-of-concept could always We always to by are going ourselves. talking and partners anything. final relates to or are to to not we are question open go we to partner.

the have the we have that. to do that, to and capability We resources believe do I

to we civilization for open as all can't But way disease. as are large to the we Alzheimer's disease ultimately, However, go a that, anything. imagine could I

have So choices we And is we program. partner question will that some there. need The when? to

monotherapy, the therapies I look, your believe, a just disease disease started of accelerated modifying Finally, approval beta we Alzheimer's antibodies. the think combination with question two I era about or for

range from knockdown. As efficacy get effects. might CDR to or is in boxes. to love approach, the antibody And whether XX% combine modest it's XX% we a We'd all to an be reducing approach one know, better with the a treatment the the on tau

that spreading Because I antibodies I we are your that likely address answers test separate once are tau Jay. all combination or to the hope starts need tau -- may think to so in to questions, treatments. to with them. occur, you combination And from going we independently think I amyloid

Jay Olson

Thank much those That questions. was all for perfect. you so taking

Operator

you Jack for our be question. wait while ask for moment coming prepare next before your one next you, from question please name question. the be Thank And will Allen. Again, to the called we

open. Your line Jack open. is line is Allen, your

Jack Allen

wondering, I the wanted the wanted tau recent are Sorry about Lilly had saw Alzheimer's in as and that by you any congratulations theme results it and from all a for sure their I they on intermediate where study of around with on I you enroll? you Lilly effects early mute. if was patients tau stratify so well, thoughts to Eli that. was disease Thank any much the questions taking the population comments stick tau. measure the and looked group there I'm to progress. differential ask tau And seems versus Eli on higher I within Al, have the cohorts? specific you aware like you

Dr. Al Sandrock

a interesting I Jack. had less tau forward gather, the treatment really a of had the higher conference. But from Hi, the who burden I That's a started they data to when question. effect. look treatment at the what seeing people scientific

of approach, terms the the was the we -- in And even just the cognitive targeted saying symptom symptomatic need disease, earliest patients what when far with of combine that And get tau with we population, therapy. to advanced which I mild think early think, in impairment, disease terms is the tau enrolled the is that may underscores, pretty pathophysiology. molecular I I

brain. has see only and these many years to tau reached, patients. started up has accumulating in maximum progress the amyloid We of Not been essentially in have to XX a burden already for

And going at patient think maybe combination, that so, earlier. do MCI you're of least even when even and you early to need go to I the stage to

Jack Allen

to and are proof Got that also to see competitive programs for it, you into wondering, concept on closely other as any you landscape early great. if had so comments monitoring space? in Thank that. insight was the of it tau the relates I many much

Dr. Al Sandrock

domain think that number efficacy of from I that a approaching understanding. Well, readouts my to antibody are are targeted there now trials,

antibodies, the consistently tau. we spread the inject the where mid interesting. in tau, humanized assay, pathological them be domain animals could take multiple and ourselves. studies So the in that have will of as we effective very weren't hands, pathological In had at our our hands, that In we into animal spreading human they look

So, we're these need domain believe, mid additional, see the to think, antibodies very in Todd? all the therapies. addition come hopeful I see to But differentiated. that with something patients wonderful remains it'd be our disease anti-amyloid efficacy I antibodies, Alzheimer's

Dr. Todd Carter

own pretty at from studies us least to effect. wanted made don't preclinical that clear or work that epitope bit, that, main just antibodies as our Al work, most matters And the our of add strongest our robust in C a so it's the they're in quite the gave models, and antibodies terminal mentioned,

I works these the that as see doesn't. hopefully, through it be So, maybe antibodies what to then come think what field clinic, whole will we for the and very learn, informative

Operator

we you, moment our will TD Nadeau next next Thank of And from one be coming Cowen. Philip while question prepare the question. for

is open. line Your

Philip Nadeau

on team. keeping tau Couple from us the

that it produced to you've In at of any a changes? that as terms of version that how or XXXX? prior antibody the chosen development tau other compared antibody that candidate, there does the one the antibody data are a Is humanized AIC simply

Dr. Al Sandrock

ahead, Todd. Go

Dr. Todd Carter

Sure.

see antibody antibodies. Data taking number that a on data we're of panels antibody multiple The you're lead shown humanized the included is different of of talking from version a from antibodies, have presentation. epitopes. we data So forward about, I

Philip Nadeau

to antibody It's the a produced that the humanized XX% in declines version of XX% that?

Dr. Todd Carter

Yes.

Philip Nadeau

you GLP second get within feedback or that it able on file quickly? average. file you're faster tox, then than that on to stretches that FDA And an How the sounds complete just prior can a the going year, so IND and experience? based the tox, is be done like a Is GLP from to

Dr. Al Sandrock

species a other here wild used animals, the tricky It's have for are part as expressed the in right. target species, which antibodies both tox not is antibodies proteins. that there actually, protein, against tox typically pathologic is type humanized what's and in been the they The

on look you being look the have where So as to in the targeted you at an order that place animal toxicity, models to have at expressed. that's only tau, pathological

Those well. models prematurely disease. do the They of don't this animals because die

antibodies experience against so work the And of with limitations in to you humanized have do But have we so the within pathological brain. proteins that.

so but And also feedback. experience, FDA we drawn that on have we've

so plan toxicology is on And both. our preclinical based are

Philip Nadeau

was then April, million be that amortized going to for question in in Peter, be there QX one booked or Peter. an to all that And is last milestone is received $XX the housekeeping going component?

Pete Pfreundschuh

QX, perspective numbers. spin that recorded cash the is read We the that perfect. in but right See

Operator

prepare moment Yanan next will from Thank coming while be And one question. Zhu our we you, Wells the question Fargo. for next of

Your line open. is

Yanan Zhu

thanks Hi, for tau the question. solid and one taking perhaps and our questions, question a ALS

imaging is then that for once further the mainly if could tau the or the of the a or for before? antibody question Is given a potent, success you. reasonable So nomination findings method pursued. be how can less PET delay I'm data the would the it of PET tau, still for give criteria the is, you On some you more more the be if anti reason antisense, approach think get to you on antibody reduction tau guessing on about that payload as how mentioned optimization the reasons? there's candidate? other modality color TAUXX Thank you about vector

Dr. Al Sandrock

great questions. one. Todd And the are first the Those second will one. I'll take take

On well, first the tau, all, of the is intrathecal. know, antisense you oligonucleotide injected

And intravenous difference That's inject every of the going is pretty mode drug so that administration. four our right weeks. there in antibody big we're in a an to

that tau. that We takes account showing others on competition. data compare into minimum have obviously are a We to product our will do profile

we spread compare see And have have of to move we first statistically treatment. the and competitors so And reduction tau. space to have additional a then we significant If we'll for an of will there's it all, in forward. if

that fact having was a is treatment. mind that ease options for for think physician, I different of always I one in than liked multiple patients. Todd? use When of my the the higher and of intravenous more administration that keeping room antibody I modes Just an the there

Dr. Todd Carter

Yes.

you as candidate the combination the genes a is I've X, trans of trans a and We're capsid to payload, and continuing work of out, pointed know, Slide on in evaluate development the and capsids. So therapy gene. our

well. to week. Our very next invite capsids I presentations anybody out continue on ASGCT call perform the check the

of a -- and our have presentation, We and facets. have oral presentation couple posters symposium a on some we novel

seeing this quite capsid our we're payload results the the hope continued we And are that meet is performance criteria. trying back the half identify the barrier. do and of membrane with optimal candidate reproducible to to optimal in year. across We do to What

Operator

while we And one you Wedbush. next Chico be moment Thank for will from Laura of coming next and question prepare our question. the

Your open. line is

Laura Chico

taking your Thanks And there guess, guys. staying on milestone for morning. or Good very if change for remind upcoming in, wanted this much for in to ask I the year antibodies, question. perhaps learnings development a the can Pete, tau? just inform that remaining XXXX? coming decision is payments you for the any there terms theme. in Al, tau milestone strategy for with anti-amyloid I obviously one big it's Thanks can of the been then any us, potential reimbursement

Dr. Al Sandrock

number make of going we patients we patients. be don't the the to Thanks, look, year. we a We question, And mindful learn, But have to society. this to question access lot Laura. sure we to think, think Yes, anything on total as a really I that of the and want have goes on treatment, require year patients and reimbursement to I that that's important to drug. be want to will do the have are to unhelpful want help

in there coming the I is think us to years. lot And for of a learn so all

to -- but that if think have pointing I you are is combination so that a to two drugs the And that's concept reimbursed. be treatment, there

that have into will we account. so take to And

Pete Pfreundschuh

question. Yes, Laura, your second with to regards

for forward I kind that's with a in milestones been So, that But and milestones. much more relationships all relationships, shared breakdown. specifically I guide think with lot it would a probably milestones those future that getting existing would now. can and our lot say, have when have we say that's aggregate again, where some leave of looking the it near-term, I we partnerships to know of provide our potential we relative sell, be some we to don't could way. to we of to regards with we this we guidance don't it way, of redacted. a I with that publicly But in regards are regards of there

Operator

while And line moment question the Truist open. is Securities. prepare you our Joon and be for of question. Joon Thank will from your next one coming Lee, Lee we next

Joon Lee

Thanks our for taking questions.

it's to MSH that interesting are For -- you non elaborate targeting. and specific strategy little -- and approaches plan single specific you you address as a with the MSH both Huntington's SNP, construct? a in taking SNPs? include a do construct on and well bit siRNA multiple an program, a to Can SNP you multiple as your also would And to combine here, single

Dr. Al Sandrock

the I'll and ask answer. to Todd start finish I'll Yes,

are mutant. So expansion, I targets occurs because allele which some in huntingtin specific, mutant kind only targets allele approach. the both that ways, is would And an target the say off obviously, an of specific MSHX allele-specific both the

I So both as preserving there's lot we mean, We're type answer. Huntington look Because, the then Go concern preserve is in on Todd. need to are siRNA of ways, is And targets vector. expression. I into expression let I But both aim ahead the and wildlife combine I'll it to goal. think that which the same some our kind yes, thought a these. of both I both think Huntington, targeting preserving of of wild

Dr. Todd Carter

Al. right That's

do plan So small. is for payloads in and the siRNA, we siRNA relatively incorporating both the

a multiple, in problem well. we So approach. evaluate it's do the I can and a a fit vector, mean, independently as single bicistronic not we explore to could

then choose only affect we PAN specific with, could to on manner, option And the the a Huntington's MSHX, allele move an specifically. the to or mutant knock SNP-based always allele the that's going is approach plan specific. approach forward that down we would after then to So and which

Dr. Al Sandrock

not most may And do common to second the that we're start be we Huntington's to right ultimately, HTT MH treat specific approach, to allele then single able forth. every need We'll SNP-based a so alleles. and patient. you're going So, with program,

Joon Lee

on up program. the follow a have tau I

programs, go. You you alluded have, to, multiple as have shots people on multiple some

the evidence siRNA. to extracellular has as species more more one probably which that species your but both, form? like is as in say a you address? with probably important targeting there more as species approach Is the I well or view dangerous pathogenic to So, interstellar know have You one

Dr. Al Sandrock

I the to answer wish, Well, I that knew question.

species here what it's that the there part And forms, just I extracellular terminal certain space. of that's extracellular the multiple pretty that think is targeting you tau complexity if in will, containing are in top have species? been those. work, approaches doesn't Todd, tried of probably the for I know But multiple that, answer. don't because pathologic the do think I you an have Maybe

Dr. Todd Carter

No, I don't.

Joon Lee

the because lines versus I of approach, along species intracellular the More intracellular the extracellular, believe your target, siRNA preferentially?

Dr. Al Sandrock

I will the target Yes. forms that actually of synthesis Well, tau. target it'll both. mean, of all

very So at extracellular too, definitely target won't tau antibody also if which it the extracellular increase eventually tau is intracellular will we able decrease, do because tau. the that's we probably also But intracellular to -- be of all. source will much they'll

Joon Lee

say would project TBD? you higher that success Would the that's SRNA have or approach little that still bit a

Dr. Al Sandrock

things. going is every profile have to mode its of issues benefit risk and But and yes. own administration approach Well,

think I So I think which multiple approaches. early after such I approach wanted still too think, certain go an But target, is patients. to to optimal going it important it's be with it's to for be we to know

Operator

prepare Canaccord. question while Sumant And Thank be we of for you, will the our one coming question. next moment from next Kulkarni

open. line Your is

Sumant Kulkarni

that are. you see Company to in all time question my specific being nice conditions to and has here exciting progress as worked the targeting be for the the Thanks

a I question therapy ALS on SODX your program. have So gene

medicine respect to clinical is with you trials in to available, And the Tofersen? landscape Now then a how specific expect running do change that recruitment? preclinical model with in combination Tofersen

Dr. Al Sandrock

of the sure think in second first I to all, question I heard and being the But Okay, that of so the of across question. Tofersen patients the and heard terrible for treatment getting I'm -- options, patients with recruiting there's start I first Ionis first that disease Biogen I'll think the have setting And I line. finish think But I congratulations to one. a relates approved. not then it's feasibility wonderful

is who to And to see whether be treatment therapy marker Tofersen, a treatment just track take is for can and we but can know gene we as are it the see zero, either example. if the if on patients adequate perhaps require could decreased. for Tofersen already they us, For and requirement not or neurofilament for decreased,

may to Another hard patients go to it's of world approach person. would be to access there untreated where parts be the the

get would And even be mutation. then of approach the a symptoms to autosomal rid function very gamma dominant go early, toxic to third before

approaches time as more those will the that the, now. making terms is So, I by are validated that I of three helps think to whether the at FDA, believe. the option. And regulators, neurofilament the which outcome of only But my I considering fact one least in we're tell considered alluded is best comments, in feasible, these as development a surrogate measure

may discuss And the can to because I I then have question repeat sure second I'm not heard.

Sumant Kulkarni

gene to therapy merit product? in preclinical specific your Tofersen with optimize model in The second there combination running part of that for any a Is was. the

Dr. Al Sandrock

whether the studies. So, doing we're preclinical was question

I'm a in patients. did before study study a SLDX due are of GXXR GXXA, combination there but to in et the couple couple So, would necessary add the not models, cetera. that be We mouse we certain could transgenic vivo,

I use based biomarker terms of think good very blood that the amount monitor of to that have to injury we can a in we motor patients neurons.

animals these of models, of some And not the in they're very what clinic. predictive happens

So, animal models. relying about too much on I worry these

Operator

of from Baird. is Instructions] next Allen [Operator The Jack question coming

open. is line Your

Jack Allen

there relates know research I your as ongoing? have comments of so wanted you a the I of year, primates. follow-up. this of it to you preclinical assets. earlier and band your, around much these see reach availability any wanted to out any for have programs and And the importation guess, taking to I Thank critical gauge non-human comments if that have you you this, was and I awareness of just

Dr. Todd Carter

it's Hi, Todd. Jack,

So, it's great question. a

very adversely closely. field watching quite certainly non-human been vendors mitigate not I best We We carefully. multiple we should and availability. I the doing with primate case affected closely. is is say have think availability by watching issue to the And up watching that, options set all entire To-date, make non-human in arise. primate very any are to we're sure agreements our we can an have risk

Operator

are Q&A for the concludes queue. to Al like you. Sandrock the Dr. session. more Thank questions turn remarks. There to no would back over This call in closing today's I

Dr. Al Sandrock

Thank any you, everyone, if to for with joining follow-up free us today you us further great and for questions. the have directly questions. Feel

Thanks again.

Operator

call. everyone you This today's event. conference concludes joining for today's Thank

You all rest and of everyone day. a have your may great disconnect

Voyager Therapeutics (VYGR) 9 May 232023 Q1 Earnings call transcript