VYGR - Earnings call transcript Q1 2024 | Voyager Therapeutics, Inc.

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Operator

Good afternoon, and welcome to Voyager Therapeutics First Quarter 2024 Financial Results Conference Call. [Operator Instructions] Please note that today's conference is being recorded. A replay of today's call will be available on the Investors section of the company's website approximately 2 hours after completion of this call.

I would now like to turn the call over to Trista Morison, Chief Corporate Affairs Officer.

Unknown Executive

Thank you, and good afternoon. quarter results first afternoon. release our issued XXXX press financial this We press available are and our The XX-Q on release website.

Medical Toby Joining Dr. me Officer. our Dr. Chief Al are Dr. Chief Ferguson, Sandrock, our Carter, on Chief Executive Scientific today's our Officer; and call Todd Officer;

by Officer, also will Robin call Swartz, for Operating Officer portion of the We Chief Q&A and joined the be our Principal Financial

and as are materially. that noted get beliefs. subject I current available Before Slide based on make would during everyone and our risk this I risks remind differ to may our you are in call, uncertainties, the started, today's we our consult website. forward-looking and They are expectations of encourage to factors statements may in which X statements deck. representatives actual filings, Voyager results discussed like These our to SEC on

And now call Al. to I turn will over the

Alfred Sandrock

announce first Alzheimer's dose have announced antibody, dosing for for expect healthy IND everyone. volunteers start disease in trial subject team X. and these Slide week Please medicines progress Thank the the to good advancing dedication their in We to for thanking so. and you, coming by our obtained VY-TAUXX of genetic made or to in a we ascending anti-tau clearance creating that tremendous first we We afternoon, single Christa, just medicines. the turn quarter. transformative in to the the like I'd Voyager

for wholly includes ALS neuro-filament March, on Dr. development receive an first are selected Friedreich's is FDA-approved programs tofersen These filings Ferguson light ataxia deep biotech therapy we Our IND with programs, Neurocrine. gene [ advanced and ALS In owned CNS as approval Tobi ], This diseases. experience based GBAX Toby and plasma XXXX. program, advancing accelerated Officer. response. therapy in to partnered the treatment with quarter SODX first pipeline be our the appointed to novel SODX towards the our candidates also advancing targeted the therapies in for genetically during Medical executive chain with with Chief exceptional along

Toby his agreement leadership clinical to Huntington's advance brought new our disease of Novartis spinal we completed the and we for through January, offering capsid has forward agreement, potential atrophy. January, announced portfolio. hit we relationship together look our emerging the expansion and license strategic gene an $XXX consideration This total therapies million ground a first public muscular and the Voyager quarter. to with of running, in of in to with collaboration In

and our our sheet balance extended will clinical data we multiple bolstered and into it readouts. runway enable This to XXXX, expect us that achieve

of on data robust ADPD Finally, we in our and data our a programs second-generation presented ASGCT the capsids. multiple set recent meetings, targeting to at including

neurogenetic is Voyager we significant believe leader a this progress, in Given medicine. as emerging

of Slide pillars summarized on value Our X. are

more First, weeks we enter programs XX for partnered year. potential with owned X the the coming have the and next to strong a pipeline expected trials and in of clinical to wholly first follow X

gene CNS designed Second, the delivery we to industry-leading to inherent therapies. have overcome platform an challenges

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to Third, the transform diseases. we of anchored have partnerships potential treatment blue-chip CNS by Tracers

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of gene broadening turn that, into receptors over identified the to potential neurogenetic we explore call I'll other With antibodies therapy to our to aim Toby. medicine, genetic we Ultimately, shuttle leverage continue modalities to to we the into nonviral from the medicines Finally, have brain. expand impact. and

Unknown Executive

believe summarizes slide are good formal the improving focused Voyager and And Al, partner programs to of lives the with by has human This enter owned basis and programs me X I'm urologist, on programs attracted targets the that is has my provides challenges Slide potential and as turn our of X. validated biological diseases. these transformative tendered Translating and clinic unprecedented diseases Part mentioned our blood-brain by our you, to to of been what is Al to we barrier. realize ] progress treatment Thank practicing that genetic earlier. join crossing while drug wholly afternoon. company that human and CNS living to XX the [ of full Please pipeline Voyager and this of career neurogenetic delighted new regard dedicated of I've that the has people of made a CNS I opportunity advanced therapies biology neurologic an the the prepare that genetics and as the medicines. considerably. to diseases. the understanding to redefine with developer in the understanding

decline for target shown generated the TAU brain pathology and [ want wholly Target have visualized was focus third-party intrathecally-administered associated of moment Slide a a be cardinal disease. recent to a using X. [ with presence reducing Importantly, favorable for The at through believe on of important to to our TAUPECimaging. that data critically Alzheimer's an in with We AD. clinical is power to X programs feature Further, ] is Turning I the TAU the treatment's sped owned TAU. clinical brain on outcomes. TAU can closely ] of trends up pathology correlates with clinical

we're And initiation in trial the forward mentioned, Al in weeks, single anti-TAU announcing that announce As coming the a ascending IND clearance. volunteers. dose look of VY-TAUXX, our healthy obtained to to excited monoclonal we antibody

in that moment. trial I'll more a talk about

program. of targets VY-TAUXX VY-TAUXX that have to matters bit spread third-party the of to extra epileptic XX%, anti-tol In a aims this antibody the brain. model. no on contrast significant by indicates the cell-to-cell epitope have in the saloons the a tau the targeted believe preclinical been the approaches VY-TAUXX the in We this Myriad of inhibited of of model approximately in directed ]. First, palace out background antibodies spread intrinsical inhibit with negative value in stem [ have This ] predictive vivo the internally [ effect. to unsuccessful clinic. and surrogate spread,

We as predictive clinical to we advance or establishing forward positive look has value into it whether trials. not

Importantly, program parallel, administered into and production IV we sRNA are in the packaged to progressing silencing a TAU approach a intended This therapy TAU-targeted capsid. an TAU tracer inhibit protein. gene of deploys

believe and for anticipate Alzheimer's in reductions approach, potential mRNA we've an We administration IND brain filing single-dose robust a the mice to We and provide this program has disease. single treatment and Using demonstrated by the TAU. XXXX. transformative across protein this a human expressing IV tau

Turning Slide to X.

mentioned, weeks. our the I of single-site the we multiple will VY-TAUXX first trial. to trial, is aim the which and As a The coming generate subject data anticipate dose in study primary PK in that we dose a participants. to of be ascending initial in This safety expect enroll subsequent trial the XX dosing announcement form approximately single-ascending will

to ascending Alzheimer's dose conduct with disease. early multiple the We trial participants expect

data We initiate the expect show to to generate XXXX. year TAUt imaging of and potential slowing this initial spread. in trial has That next Talpa

X. Slide to Turning

GBAX owned replacement one ALS, other program the which cause genetic include also we to They wholly year. our mutations the represent GBAX gene partnered silencing Parkinson's the Friedreich's programs of ataxia. of gene And disease targeting program genetic our Neurocrine genetic INDs replacement the targeting expect program. engine the X related Neurocrine most cause diseases. Protax in as GBAX well of the file are common as we tau, advancing for addition SODX of partner In targeting SODX therapy programs next to cause of both cause

Each these programs and a dose the promising potential IV-administered, suite of forward programs an in has clinic. of disease-modifying blood-brain advancing barrier, Pricer We single leverages treatment. look to these to capsid the provide

turn to call With Todd. the I'll that, over

Todd Carter

Annual present pipeline, Toby. support Thanks, manufacturing pleased Slide of the presented turn few at of XX highlights. last. a Voyager I'd tracer advancing overall capsids were and on total the and translatability therapy like data Therapy American gene to to potential Gene a Society our Meeting X. at robust clinical scientists a our of set review Please and performance Cell meeting, the we of to In abstracts of

was on line IV-delivered I the the This which the high speaks door blood-brain we of cross interest down barrier. and the our . engineers presented hall, with session think only room to the new data of level First, second-generation to in capsids standing out capsids. tracer novel

over and XX% T second-generation neurons barrier of XX% demonstrated should enhance types. a vector in Parkinson's capsid penetrants of own compared the also second-gen as relevant included genomes clinically per across This transduction of Our kilogram. with at XX% upwards XX% of brain dopaminergic cells neurons These our to motor spinal of diverse dose of of blood-brain further and substantia capsids. liver transduction first-generation cell to a the as nigra, transduction certain robust expression [indiscernible] neurons. Such XX% reduced regions capsids taco-derived

tracer of Importantly, selection capsids our distributed. programs have the for gene X lead development that therapy Toby enabled candidates

SODX ASGCT motor We IV VY-XXXX presented single up ] by therapy, data per the nonhuman reduced second-generation that XX% dose spinal owned RNA silencing at SODX demonstrated uses to of [ wholly The dose neurons genomes acid our ]. VY-XXXX, [ XEXX trace which and gene a a at kilogram cord in in data primates. vector of

strengthening X formerly barrier. identify the of and as in ASGCT, maximize Finally, tissue the of brain clinic. alkaline capsids. developing and candidates, primate success has that CNS the capsids tracer mediates of humans. blood-brain expressed across capsid nearing nonspecific our working we ALPL receptor tracer Further phosphatase, confirm identified in risks isoforms. expression their on mediate the conserved into or to derived mitigate of of clinical delivery the ALPL confidence their receptors XXX as known by potential the these human that and of and highly VCAP-XXX capsids strategy to as the trace in product been the Voyager the vasculature These part receptor probability capsids At our delivery

shuttle. their blood-brain I back turn at in future. course broadly, these range therapeutic presented of a barrier. represents the We we to the And most package of look the these leverage a translatability the data exploring diseases. over multiple ALPL our sharing deliver validation the transform broad look evaluating establish call Collectively, we across to as receptor And potential Al. ASGCT opportunity we novel forward to the of to the neurological are ability clinical date delivery now forward modalities will this brain More to data of capsids. data the extensive tracer to to

Alfred Sandrock

Todd. Thanks,

XX. Slide to Turning

incredibly You strong an Voyager the had can year. see start has to

and say before, As them I this our want are you. so possible the I know mentioned near With listening, a thank to I we would of to the cash hard drive is dedication employees. runway into without long work slate and maturing be value and a again, robust portfolio, term. poised partnership many not of of Voyager clinical milestones, over significant XXXX, upcoming believe both

With that, open for will call we the Operator. questions.

Operator

from Securities. [Operator Instructions] of of first line question Our Truist the comes Lee June

Mehdi Goudarzi

is you on on ]. Congrats Nassica Mahdi June. data great This A showed related receptor for at progress. [ ASGCT, to the a great

could modalities you to the antisense of LMPs So elaborate delivery of for you knowledge and you specifically, are please CNS, that similar transcytosis oligos do use seen think the planning levels could ways on AAVs? this see other to with

Alfred Sandrock

This Al question. the for is Thanks Sandrock. Yes.

briefly, make at of them get as So We of is to receptor, molecules molecules ligands the as do receptors see speak. range know cross conjugate transport and the to across experiments progressing protein macro large these if that them therapeutics include what believe, to we AAV as look well we're we're oligonucleotides mediate and the those we various the BBB. very going transcytosis. -- these to capsids Todd? we against to by we the could can now And

Todd Carter

Al. Thanks,

a of at variety looking are we modalities. So

we're in looking indicated, have to. we're the at sharing that I'll ligands we're other the to those ready and of in some process antibodies, of ligands forward of sorts identified and hopefully future Al As we're some proteins. of identifying all when those mention data we looking

Operator

comes from Our of of line next the Baird. question Jack Allen

Jack Allen

manufacturing optimizing presented candidates to And of of some more you at will to well? do are our around you the thinking in How you ask be on about of COGS progress. therapies? Congratulations therapies the work benefits gene I the potent your gene optimization before you ASGCT with bring as the expect as it clinic the what manufacturing. about wanted relates

Alfred Sandrock

here. Todd Well, and to ask I'll help I'll start

be planning HEKXXX use cells manufacture products. So we're to these to going to

as well processes. be internal technical upstream assess a the well full part and both candidate And development course, robust to of as selection, capsids, have very downstream well of of operations as verifying we full We as the anti capsids. percentage team partially we're manufacturability, as capsids going as

the development of assess as selection. And manufacturability candidate Todd? so we part

Todd Carter

So given the as assessment establish as Al so case, each make our whole novel manufacturability of In component. we identify any payload for have the mentioned, can and we part we to It's we the manufacturability. of then for we payload and program, process do with is And move a manufacturability them. indication assessment, capsids settlement key that begin into research-grade have proceed difference once our in the the as that develop into question material. that the to a to actually the our All we then that well. particular development capsid disease candidate, goes with

You also exactly COGS. necessarily trade-off. And X:X asked about not a of it's question a course,

level an the because delivery But same or CNS with the than our much imagine, approximately material dose savings as substantial conventional can we [ you the creator the deliver the currently have to in greater potency of to novel we looking get expect cases that of is used a about what ]. actually to we're than levels less capsids see order clinic, magnitude

Alfred Sandrock

Yes. just speed the doses, And scale, CDMO cost. really a for reduce to of the world process up We're now terms seeing in what to that in time every I CDMO scalability. actually addition scale. increase Jack, would to note add, the has you And to going we the the transfer that lower we're And manufacturing. come is we

the as that's we well. that see So pace continued area -- advancement at expect and to growing, in

Jack Allen

life gene here? these I That's products commercial we think applicability space, does haven't gotten how that. a of final just the this in I far the great. but scale shelf role of you one the Can play when really know on question manufacturing these about ask therapy

Alfred Sandrock

to Shelf But life. answer thought Well, about hadn't there anybody that's a question I that? yet. an here is

Todd Carter

evaluation cold shelf we require So part And to assess for particular stability to of is that of course, temperatures. held gene at both be life. All would and the short for the a typically long need therapy, do term we term.

our part that's of manufacturability and development. So our process important an

Operator

comes of Trucchio question Patrick Wainwright H.C. next & Co. Our from of the line

Patrick Trucchio

all on Congrats the progress.

a for me. Just of follow-up couple questions

discuss follow-up that XX% the regarding is, first with in can how this data mouse advancement your Alzheimer's you protein. data, advisory IND you'll disease human and any the model that in The the committee to showed XX% I'm from data up June and how ASGCT Alzheimer's on tell reduction and potential then meeting for XX looking if reduction XXXX? specifically Can programs, denenimab XX% levels to separately, in may wondering a capsids TAU you tau on to the generation us in [ of in TAU support read-through be compare SERINTINE prior mRNA therapy data TAU ]gene just

Alfred Sandrock

the question, I'll to I'll answer second Patrick. Thanks, one. first and Todd maybe ask answer the

pipeline anti-amyloid very On a have the first question, our We well, interesting advisory be vectorized a it committee. do program in as will well.

that watching we're interest. with So advisory committee

may might important not features. of I Nevertheless, the some donanemab think much program. and our to to there lot be a pertain questions be might unique very

that that on. the measures outcome keep going to And for the approval. always that's are we are eye important an need us. down of course, something But for what that's example, For to line be

thing that's kind have first. be the We be interesting it into attention may an get But to so we clinic meeting. to, the Todd? -- but paying will of

Todd Carter

a mouse So was in at the capable to, we at on knockdown ADPD the you're showed The data with program, our to also ASGCT. that and mouse referring some capsid. knockdown

to at relevant demonstrate both did use to and that we hopefully and dose. to down it's really the payload feel mRNA assess ability that knock the a payload the also we that clinically we has protein So and TAU

to safe With in mRNA see dose were a we brain. that animals. the we sufficient showed So those delivering saw And those remarkable those the able we vector to payload, the and in what mouse think we well-tolerated studies. knockdown animals, regard quiet to in that, that's of

in for concept novel question. primates moving payloads So the it's building the the forward now and that for and of we're and knockdown, with evaluating proof humans capsids the nonhuman

sharing to in we're future. more So the forward of looking that

Operator

Our TD from Nadeau line of Philip next Cowen. of question comes the

Philip Nadeau

Congrats on progress.

you trial? data you can on ascending how a going And you couple to pharmacokinetic MAD that use Have you disclosed blood determine the single little a what in exploring First, to are dose the will talk you VY-TAUXX. be crossing you're versus be doses going to gather doses the How the the and peripheral guess multiple brain should trial. barrier? doses single the to particularly for extrapolate what dose maybe explored from bit in

Unknown Executive

for Thanks is Toby. This the call.

preclinical we where We reduction model haven't of we've our of disclosed in speaking, our our the candidates spread, tau mouse broadly doses, we've shown examined see models. about by spread pathologic XX% in of our TAU but

this think In patients is multiple preclinical ascending a I about -- cohorts. in fundamentally, dose in over XX addition, primates, and we've done work single study

We expect the get translate PK aim think we on this. data can information of in as we that doses for needed to exposure the as exposures in of appropriately to into both, the underlying brain treat Whether, based get safety to we that MAD well appropriate to on sort estimate peripheral to course, information mid-XXXX. want

Alfred Sandrock

Yes. spreading in the that based want don't exposures ratio Therefore, And into get said, monoclonal studies, we know brain-to-plasma may that that anticipate need any the I X.X% to preclinical Phil, including to I major we antibody. other we use be Toby we choose to mean, X.X% And likely in very be brain the the to to similar NHP, the to the want as inhibit an our to tile model range. will add to and This antibodies surprises. on is

idea. that multiple to inform trial. -- range upcoming dose . expect dose the And do give trial some in that the ascending will so you we So of course,

Philip Nadeau

able the notable be that willing differentiated new about you really from chosen? That's And disclose candidate that be fitaxin about helpful. the on us tell you'd that was others? what the second, anything to candidate to then candidate, anything would And anything

Alfred Sandrock

Well, program. so that's neurocrine a

And the manufacturability. don't payload in -- effects the as animals so question. as And terms these disclose want capsid as ask your that well Suffice plus we're both set a criteria of to development say mechanistic candidate -- things. combination, answer its well to for of we Neurocrine capsid had we the it as in we'll to

Philip Nadeau

on Congrats progress. the

Alfred Sandrock

the next Guggenheim of of Ry Instructions] Forseth comes from [Operator Securities. question Our line

Ry Forseth

expansion profile team preexisting Debjit's maturing, X navigate to given from the ASGCT date? how From Ry the opportunity with the data, preclinical is framing technology This Gen Guggenheim. where to able market you're you're antibodies now neutralizing you're seeing at

Alfred Sandrock

maybe capsids that our yes, that how add. are affect And antibodies start of one to we affect preexisting whether or the affect noticed we -- the in But posters see we're modifications clinic. so I'll -- Toby see Todd we're what tracer they make you looking whether to can is can and to in our can we're that actually, immunogenicity. to doing and humans performed by that, therefore, looking combined capsids leveraging

that And can preexisting will, so we would can you more if be for available obviously, evade capsids patients novel if treatment. the find antibodies,

would addition, antibody which companies use of can others can In are I various we other ways going and the to preexisting as antibodies. note lower levels. investigating degrade preexisting in be several know, you are that And we evaluating enzymes that

So that way Toby? more thing, eligible be to same our try to the for essentially achieve will to be is get to another patients which treatments.

Unknown Executive

with Al. agree would I

as populations That of make is select that be I with think in I represent the particularly a important point concern. in this pediatric may much may opportunities. adult not may be that populations only some there's addition would

Ry Forseth

work. Fascinating

Operator

Hong of the of Citi line Group. from David Our next question comes

David Hoang

-- a transporters did how extent those interrogated you've the gene second compared have disadvantages tau maybe just be what compared wanted if Alzheimer's other have? that that therapy What modalities. each barrier? about into as treatment think fitting ALPL versus other And you could advantages of blood-brain just then landscape of ask antibody Have interrogated to of the to And besides crossing the I how you also question. the silencing for about anti-tau to done or maybe ALPL potential the to you First, you transporters? work,

Alfred Sandrock

to with the help the start you on Toby, second I'll maybe one. Todd, answer then ask So question. then, question. And Toby first that can And I'll

silencing. tile versus anti-tol So

knockdown and TAU. an TAU model And to we're what will basis. very at The spread. -- is demonstrated or trying pathological a every silencing on we different gene month a anticipate. approaches. done be as X so the to Todd so, expect nicely spread an to therapy. the in will that regular inject gene different given block by earlier, extracellular need tile, human quite we every weeks, Anti-tau, animal the to order into TAU a knockdown where pathological is bind TAU of is said that look very on be forms probably animals So we that's the which of do of antibody we The

one antisense . And it's best. forms to we'll of a be or And once it then And others of done the approach Tau, done all or It's action. expression the to of thing regular works done and would be decrease first decreases -- Toby? used not essentially. akin see so once infusions. that which and mechanism different tau. whether the to So to have it a is of have be need the can expression it will see whether we'll an

Unknown Executive

you. Thank

I modalities. treatments would enough completeness add have -- we any fundamentally potential clinical with think at what either understand point of these with in don't the to this of concert, data is I

which and to TAUs first have test determining our program is important test work. both think most point to programs, foremost, I we're ones in X the the antibody excited opportunities and as program. that and try do combination forward, and well, we'll potential understand the moving look the to to I future. in be and the the think knockdown is investigated to need

Alfred Sandrock

you the want second question? to take do Todd, And

Todd Carter

Sure.

second the shovel The ALPL evaluation delivery. other recap and just be of context to for shovels the potential in question, of targets BBB other or would

ALPL as are evaluate others. and context in there few to of absolutely other a the transparent such receptors, we So would need

today. to data not we're position a in While share any

hopefully, about be share we part would that existing need area. our to our our well perform the in to, the in to as and would looking choose that would being able move that least at imagine as work more assessment You can of evaluation forward to future, forward anything BBB so transporters that and

Alfred Sandrock

think we at receptors how them thought and I -- David also . comparing other was Todd, and well. then, asking And I perhaps ALPL. have as those maybe whether to we're looking

Todd Carter

So have receptors. other some we identified

We different capsid receptors. families have multiple that target

we of delivery. receptor have them a nonviral so novel in few, we terms of are discovery, the identified for in And of process all our evaluating and

Operator

next [Operator line the comes ACO. Olson from question of Jay Instructions] Our of

Unknown Analyst

This is Jay. question line progress. Chang on the for the X-part the on a Maybe Congrats ALPou've disclosed. on

in Just internally wondering can the modality whether for first, do the time through that just I'm delivery and of ALPL, ALPL collaborations? other variability development you therapeutic the humans modality if expression aging planning about secondly, with with maybe diseases. talk expression And or may change over underlying or are to of some you using

Alfred Sandrock

first the answer will one. answer I'll Todd the second So question.

Todd Carter

on the are ALPL differences of first So changes question, expression.

variation that. different the we're So of at process ALPL can look and of we doing in genetic

work with of and terms That models. the internal from is in birth that ALPL databases robust on expression we expressed from In bit expression level, also preclinical know a age. quite goes starting at at the a up level

So level if even but in age. vasculature expect evidence the a even date that the starting might robust, all anything, we at populations, the very early in older of quite expression to is suggests better delivery

Alfred Sandrock

But we're question, do of that. that expect enhance terms doing do already we to collaborations for started in internally, the to can And of work be Yes. we discovery and a and development always looking that the have efforts. second lot

tuned. stay SPX. So

Operator

of of Genuity. the line Sumant from comes Canaccord Our Kulkarni next question

Sumant Kulkarni

questions Ferguson. see are I Dr. all guess progress. these welcome Toby to for Nice the

in the to on program, ALS market your trial SODX eventual already patient and recruitment SODX ALS out how the work? So do tofersen for you is limited given because expect do preclinical you why involve program? -- population play to expect on tofersen And

Unknown Executive

Sumant? one obviously neurofilament, on and first ALS question. of a approved is approved, it a Fundamentally, indeed Thanks for biomarker, the therapy I first, think interest. it's the administered think in particularly disease-modifying once for so is I and I basis

you I our programs. point think to on what I'd

Alfred Sandrock

a Once month.

Unknown Executive

transduction really a Once we excuse correct at discussed that the programs at, to our recently point did What Yes. ASGCT I'd data. in month. is me.

I think to X% ] that when [ at to VCaP transduction cortex start. we XX%, to motor saw I'd capsids. neurons, highlight In up looked gen we and XX% X% second in

In And important. quite context with present really a dose. program, we core. saw of that's of [indiscernible] in administered and mRNA the reduction you So intravenous the mRNA that our in XX% an VY-XXXX and was meters

I lessons particularly fundamentally, tofersen, biomarkers. take the around we'll think So some from learned

we not people to this opportunity of importance understand think the our in platform to potential only provides for We'll an with us apply program. tracer a but these concept owns, also development our proof as potential clinical the whole.

Alfred Sandrock

concurrently we that in of I'll Sumant, gene Zolgensma. years couple approved that And SODX X in look used of note the SPINRAZA sort I'll world. And of maybe forward ways the approval the to prior potential to SMA our therapy, case add the was if in a some real of are

after birth. So necessary, patients for example, we're they right And then SPINRAZA. Zolgensma add if aware that many can get with treated

person therapy gene necessary. the continued treatment with doesn't a taking when so preclude And

if by being treat SMA page a our patients therapy And think gene could person optimally. essentially to see physicians to used so story, as their take the SODX I well as, we products hopefully, from both we together

Sumant Kulkarni

on would what is SODX and quick be ALS context? had. But considered the a NFL the it. clinically relevant, what for I there reduction seen gene percentage guess, We've already Got in relevant data therapy? out clinically follow-up, as Biogen for And considered a what's that

Unknown Executive

[indiscernible] with think the So that seen all highlighted Biogen we've the has reductions. reductions I

of think assay reduction it than what of variability tofersen a I biologic But not would guess be of I is data substantially greater the ability clear. your highlight does is has fundamentally, and needs and to reduction. what I the inform clinically the that points relevant the magnitude

Operator

the from question next Our of line Instructions] Zhu [Operator of Yanan Fargo comes Securities. Wells

Yanan Zhu

receptor is receptor, terms does drug wondering ALPL the on other any or impact . tissue? tissues. was delivery not And in into a of about how I potentially first, So there brain delivery expression this impact that of other

Todd Carter

that Sure. question. This I can is Todd. take

it. for you thank So

So body ALPL expressed and vasculature and entire is other across the the some the in types. cell

barrier However, other get what is blocking capsids do into the harness these get on AAV need to capsids mechanisms conventional capsids, AAV is brain. blood-brain other gene therapies going to into tissues. is that all we the most or to not for conventional think But

ALPL that So the use we brain see cross is the is barrier of cross-species the to into important to harnessing get activity. of significant also the blood giving brain. only some us is What really ALPL

with evidence BC's activity, but not transcytosis species. it vitro So provides only assays also in multiple BC's across and we see in which vivo us across activity in in

So just and we cell doses, driven CNS. a delivery that the is of examples these kind And into the is delivery the probably [ is in of of we're that's mice. able XX% kinds low in dopaminergic Pataka, and to then that neurons other nigra. it That's de ] motor characteristics from simultaneous in not XX% spinal ultimately, marmoset we comment at by regard Toby we're see based transduction neurons mentioned. of cord, increase specifically see the substantially finally, delivery, significant we're on substantia high what monkeys liver. African but X HEMOLIVER species: seeing per with Sanoma relatively achieve what And with hypothesis green targeting the [ of I'll XX% that neurons ALPL ] able have capsid, tissue to the and to or quite targeting

Yanan Zhu

from there nice ASGCT, are different to few at players. very Great. a hear. on That's work capsid presentations BBB also penetrating quite industry And

some upon of touch to begin And them also receptors.

and of the your I trend just -- where after you think a stands captured how taking landscape, feel BBB a started it the might landscape? wondering, survey do you of have in

Alfred Sandrock

think people what Maybe I great capsids the the But finding -- need answer. that that a years I'll up. are that will listen, heating we're I for look, mean, cross ago. think see to the what to so world yes, I Todd we're And of the -- I say complete found it's -- think I seeing new barrier, we start lot as that the of competition improve And would delivery. is is capsids. so very and appreciates blood-brain proud that our

get different of XX%, for doses low diseases X very multiple been of [ the is that As ], interest, as Todd mice, we XXX per mentioned, key helpful. have that primate demonstrated said, also have cross-species cells of are relatively cells experiments well nonhuman species we at VGs XX%, vivo as transduced kg receptor Todd as the relevant get -- the just we with knowing and done in

wholly development we -- look, be have then X to with our candidates the soon. already So are owned, so clinic Neurocrine to one and expect partners we selected in and at and --

Todd. So

Jay Olson

I cross-species into . say, think think activity think was that so, We do I in we're candidates the the that those everything captured I going clinic. is to the next you to addition Al. the important the quarter is And step the moving or past X point really clinic. over

Operator

comes next Chico the Instructions] Wedbush. line question [Operator of Laura Our of from

Laura Chico

watching or disclosed X one be should could housekeeping a for milestone question. additional follow-up then elaborate I of you. we quick that second believe you've And -- the was Just that of curious kind just but if payment if the you XXXX? over out there in for I milestones was triggered quarter any have remainder

Robin Swartz

Robin. Thank the is This you for question.

DC the and programs. So advancement we achievement milestone. of our very the pleased the of were with Neurocrine

in programs. payments However, we partner included across guidance, is to on don't payments cash into not future which provide milestone milestone guidance our that the note important is also XX XXXX. further are It potential

Laura Chico

positively data. or how does there? -- in reads I'm Okay. And the assuming the follow-up, just then patients, there's in tracer on strategically, what terms VY-TAUXX with focus? that change obviously, but an strategically Al, other data talk, antibody that's can that of now, but you does of success the if would that for Voyager terms focus direction from as If discussion programs, receptor focus mean in what out of the perhaps, shift in the ALPL Obviously, of happen on right lot wondering terms the the a of

Alfred Sandrock

the Well, for Laura, thanks question.

that program Phase a always you're been actually we is III do make though disease. we what go too into in whether do commercialize have would an both, too in for actually may of thinking that think fact. a option. antibody. choice strategically to antibody or Alzheimer's proceed VY-TAUXX We wouldn't us company that the It's we like with a the could as implying We or large, try vectorize I intravenous I've have whether expensive. Voyager, sense to small

I comes. that the positive. of potentially So VY-TAUXX be time will when to we'll but us, get the a antibody we've think done decision we once that option could program bacterize high the partner we do . But make always if -- thought a to and interest likely is and

Operator

Securities. Our the Lee [Operator of comes of Instructions] True from next line question June

Joon Lee

platform lower Following Laura's question, that of presentations suggested that ] and preclinical the any in the excise you Alzheimer's after -- Casedincerted in And last if some your share in antibodies, the the for retried for week, and in [ for you could same general vectorized is cased Bridge ASCD, have you compared what the data expression expect -- antibodies program? antibody to antibody. specifically plans expression this of had do sorry, a inserted

Alfred Sandrock

Yes.

we -- and bind on can an help get start, and or last then I'm -- And to that we show the I'll if expression Todd mice. vectorize ago in when X year an did Let plex to that anti-amyloid meeting, years we enough antibody correct I vectorized wrong think me ask a transgenic amyloid to it at so was me antibody, so thought.

we So plaques IV. my relative to We'll enough is change lot to to animals. have in bind -- to that see to going is can't expression get amyloid view a

you get the after So high expression central if Cmaxes, concentrations to the nervous immediately expect from constitutive we're more dosing to will, within the we you system. going IV lose have that

we'll antibody. we producing So that expect have glial mainly the cells

to in would than sort we an the it -- we've will approach. that having be As the will, brain, will giving to so then it IV you will and And brain directly. be and past to of made be the it across an in only out, whether see done have amyloid precedents not And will animal some lower any anti-amyloid and models at the plex can antibodies. plex, whether that be there's binding or been with studies we of adverse events. but have then, follow we we not amyloid assessing affect to vivo we'll course, Not into able and the or Rather look shown the. we inside in in if vasculature get we can

say even well, whether regulate I or would assess they right the or not that And the animal not, I'm we're models have fact expression we'll are the to with truly REA affected don't REA that be the we would have true. small we if that's certain, within the expression concept get by REM can that may antibody are can see investigating constitutive now a rates and some mimic molecule. the brain, that. of from one we of there the Cmax, area, things but we be of One finally, And is that not whether

a program ideal. think, regulatable vectorized I And antibody be would, so

to And we're That so Todd, of terms those would get us things you want help about did are the in anything? kinds of add experiments. development thinking preclinical candidate. a to

Todd Carter

I a bit. little can add

So the antibodies and And on amyloid on tile while We the not can is several mentioned including substantial found the focus of of course, others. the that matter in the Al form. bit the quite a vectorized that have the in of payload, and back genome the going experience we promoter. structure there the payload vector vector factorization years antibodies VY-TAUXX, a general, itself, do antibody on of

the some by a as are which kinds of just of not but lot types of cell you the can on there that promoter, tweak mentioned the So the well. target level, we that expression cells, specific do, to Al things

looking all form vectorized characteristics an of be forward. of And any antibody at therapy-based so those we antibody we'll gene when move

Alfred Sandrock

to to the disease. I the vectorized other anti-amyloid we're for and month I week once relieve give care that every witnessing IV Alzheimer's think strain could a the really that on not add real now system it health have give with would just in antibody world, or treatments being every it able

Joon Lee

in Very helpful. question. And one I last sneak if can

How the now [ your any ]X findings interim of release or of to way? data along you between hopefully data the pet that VY-TAUXX. about we like So cadence half XXX imaging second and related represent should think

Alfred Sandrock

Toby.

Unknown Executive

we've the for I the is really think program. And highlighted really I rate XXXX. the that SAD key demanded -- inform program, data think in it will what I MAD time,

Operator

Thank would it Alfred you this like questions very to at now much. Sandrock I'm time. to for closing I remarks. turn further showing no back

Alfred Sandrock

Thanks joining today, free Thank follow feel you, again. everyone, questions. and any up to directly for us with

Operator

once gentlemen, your this concludes today's and presentation. again you participation. Ladies Thank for

You may now disconnect.

Voyager Therapeutics (VYGR) 13 May 242024 Q1 Earnings call transcript