Earnings call transcript Q4 2022

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Operator

Good morning and welcome to Voyager Therapeutics 2022 Fourth Quarter and Year End Conference Call. All participants are now in listen-only mode. [Operator Instructions] 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.

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

Pete Pfreundschuh

morning. good and you Thank

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

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

do and want make those during forward-looking I statements. subject uncertainties and are statements and cause remind may this future forward-looking regarding everyone forward-looking expectations, plans to results from risks Before that materially this, may to I differ statements prospects. indicated that representatives uncertain call, actual these inherently Voyager by All to are

understand facing in material to All the a SEC this review of are morning. annual company’s filings with the company You the XX-K the report on SEC available website. the and encouraged company’s filed number on Form uncertainties as are and risks described

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

Al Sandrock

will sector, have creating important rationale. to on have markets do achieved to Pete I pillars of have as capsids and focus undergone the shareholders progress this Thanks created we the morning I three you, much when value one multiple pipeline start so our continue innovations class some I value receptor made on been identification of for March, time significant high novel a for Voyager breakthrough of for entered and collaborations. focused patients. believe of towards CEO everyone. in including Thank the of we transformation most investment a by needs last in would to value very good the we became for like acknowledging capsid Since neurology, in steps we a and has XXXX. difficult discovery, the have we unmet during advanced

in of Voyager than TRACER pillar million those and multiple is capsids preclinical we following The nervous first our that have XXX-fold system more AAVX demonstrated than the studies, variants brain with AAVX conventional libraries, of central discovery expression higher select capsid in delivery. In AAVX to platform. transduction XX capsids value evaluated more Our team intravenous increased each display compared to capsids. the transgene

have liver cells, shown This such target doses We ability Voyager. and levels have the I to and cells high demonstrated came while root low specific detargeting dorsal ganglion at glial to or neurons CNS the of why cells. expression we as is

year. Last innovation. by the Our gene this not impress me Voyager’s they like Pfizer with cease accomplished innovation. their only to partnerships am of receptor allowed secure impressed to families seeing capsid have forward and what scientists for year, we they The therapy like leaders and I and with data the with one looking am generated Novartis never to identified our Neurocrine. I neurology have one us leaders,

or siRNAs to allow function antibodies diverse our our address and payloads. – extracellular validated where function payload toxic with biology. platform achieve TRACER vectorized need, transaction brought vectorized spotlight deep capabilities loss is This where and with payload mutations, human genetics to the which biomarkers into gene the CNS and of may our us value with and to human our second Our surface then a and of mutations to well enable We neuropharmacology knowledge can is gain novel targets. unmet CNS select capsid a wide of target variety design our use targets. result access which to path a a of address efficiently address pillar of biology of We quickly proof replacement Voyager’s combining cell is and by high diseases recent of pipeline. Neurocrine We is

of to our Parkinson’s rare therapy gene As X for rights for $X.X third provided targets. in to GBAX I this disease therapy and pipeline me recently million value strategic CNS up collaboration mentioned, Neurocrine $XXX milestones with billion the was upfront potential and value, partnerships. directed to our programs our our to illustrated Biosciences. program gene additional And of This through leads pillar

Allen to enabled expand a minute. I enabled a on of by that our more non-dilutive to in just and collaboration have collaboration of variety sound sheet will medicine. wide note Voyager will neurogenetic structures is balance record revenue. open We generating track a Pete that

programs, executed strategic have We Neurocrine. such collaborations pipeline with around as our

capsid from have we and our with of are our are key and creative interested payloads to we structures, Novartis. a Pfizer receptor license The partnerships with that combine completed capsid is And advance such and to is such evaluating and as as biologics. and We to cutting-edge our enabler in It opportunity option and wholly-owned deal to advancing agreements, wholly-owned programs. note important we financing continue non-dilutive programs. intend technology

to the turn Now, recent call transaction. review the over Allen I to Novartis will

Allen Nunnally

you, X. Thank Al. Please Slide turn to

to and gene following value for read $XXX billion Voyager in Al a as an and structure risk tiered received billion XX/XX provides Earlier three are and collaboration targets. therapy and gene one and variety Phase of for elect structures program sharing Voyager this transformational net upfront said, royalties therapy open U.S have of up million eligible on $X.X structures. we funding, of commercial collaboration CNS a This up in Neurocrine potential We a variety milestones advance program profit-sharing X cost announced year, to rare GBAX in to out. of the programs is and to it to As our for executed. new sales, as potential development with strategic cost option type directed to we milestone, the program is GBAX well $X.X

Pfizer Last We this exercised have its capsids And against Novartis Novartis. option to license also two neurologic month, options entered the exercised agreements its earlier against year, capsid and capsid to disease targets. with option a CNS target. and Pfizer

reminder, March in XXXX. a when Voyager million was upfront agreement signed from option As received $XX Novartis this

million has development, been commercial incorporating the evaluating exercise to to net for the as based up triggers last option two and milestone to products as regulatory in tiered year, capsids on and in CNS well For of capsids, capsids. payments high associated their Voyager targets receive Novartis $XXX license decision fees is $XX to mid royalties single-digit our million Novartis the licensed eligible sales

the It’s Novartis for our target $XX capsids royalties. over here to view and CNS as look of gene note the additional option the for continuing per up months their the to XX of novel at and as one important to capsids subject AAV upon to million the relationship. million $XX.X well exciting. Their to per therapies availability target Novartis license a to two capsid that scope next for decision this of the also intravenous expand diseases current to evaluate as options field. Voyager forward therapy and to fee retains we to license right gene license We leaders milestones exercise CNS in very enable targets, is

over sheet. to Now balance Pete review our I turn to will it

Pete Pfreundschuh

of to maintaining supports Allen. to pipeline. Please advancement its is a strong platform Voyager turn you, sheet balance and X. Thank committed Slide that

December $XXX.X reported and XX, XXXX, we of cash, equivalents cash marketable As million. securities of

exercise million. now received million $XXX Novartis securities $XX equivalents factor marketable receive has On approximately a the the basis, we of and forma year, from when you cash, Neurocrine this will earlier and Novartis we described, in the and $XXX company Neurocrine for million from options. of pro Allen payments, cash As

Voyager committed balance is and to a discipline. maintaining financial strong sheet

enables advancement the our requirements together amounts anti-tau development gene and expect ALS We cash clinical meet of expected the our capital XXXX. for into costs with will for reimbursements and expenses antibody that disease, sufficient programs, two securities, and received be the our cash, as into therapy Neurocrine trials. to be to the marketable equivalents This collaborations under operating expenditure planned Alzheimer’s SODX wholly-owned for

We expect XXXX. in for of programs filing the INDs both

And in our other these and pipeline. programs expand Al will now on

Al Sandrock

Voyager Slide see now through And you late Pete. Thanks, four can programs X, advancing research. on that has

are programs humanized antibody Pete ALS. our wholly-owned, gene for for anti-tau program As Alzheimer’s of silencing and disease two stated, SODX these

Our than models, to animal C that be antibodies target not antibody prove features pathological N anti-tau don’t. region. our of tau important very Moreover, in others differentiated terminal disease. we demonstrated of these the We may to we whereas clinical distinguishing believe block program patients rather spreads Alzheimer’s terminal attempt the that have of in as both pathological with tau spread N from the that the is terminal the blocks efficacy and

this year Voyager at initiate first potential In the January, be that led Data order advance Sweden enable IND half to later a to the will month. we this filing candidate this development into the of IND-enabling selected to AD/PD conference study in XXXX. candidate toxicology to GLP of studies. expects presented selection a humanized in a in

a program blood then tropic therapy penetrant of by the ALS in if investigational sponsored is demonstrated combines FDA, Biogen a follow gene England therapeutic September could and, siRNA a SODX solution. hypothesis review New Tofersen, drug Our Proof of Medicine a shown trial barrier the as currently we construct novel concept been with XXXX, CNS under has capsid. of IONIS and TRACER by Tofersen derived with Journal an that potent publication. blaze approved, by could brain

expect the of expect conduct will this And which primate well. half to non-human continue we studies IND as to to year. our for XXXX file to an in this continuing are select we We lead in candidate, occur first program

assets for gene which for co-develop these United is gene both at co-commercialize diseases Phase the programs, Voyager fully the are two other the GBAX an in Neurocrine. other X, in and therapy ataxia with disease [indiscernible] Neurocrine Friedreich’s to and collaboration Our therapy has States. advanced Parkinson’s preclinical programs the through option being program and mediated GBAX point funding of

a approach and for enable disease. of for early knockdown to a program positive breast associated disease. new Huntington’s siRNA in continue excited Today, also a vectorized introduce also specific antibody research advance with cancer gene MSHX, Alzheimer’s mutant metastases Huntington multiple vectorized brain I’m including therapy to and initiatives, HERX We to

This know gene efficacious recent neuron knock enable new cognitive us potential this disease, with could correlates approaches disease intracellular antibody anti-amyloid gene combining that and could also oncology. in downtown Slide a in vectorized complement tau tau unmet rationale effort research encouraged our siRNA for progression builds with approaches, the X antibodies, we’re And anti-tau mean much or This such approaches. disease as by they have tau while may anti-amyloid combining with program therapies effort. be with combination in Alzheimer’s approach. targets extracellular believe decline. and extremely intracellular the which that tau our we pathology We and outcomes tau is to extracellular silencing and upon shows to improve our expertise. anti-tau siRNA Alzheimer’s enormous. need and new The approach Alzheimer’s by the therapy closely An has advances as itself, could

tau evaluating are We our currently an novel intravenous siRNA payload with brain gene capsid. penetrant silencing

summary, ability Slide and Voyager XXXX our creating to and In through to execute value Turning demonstrated X. into has XXXX.

to entered addition collaborations, our balance and sheet. In our strengthened advancing into multiple we important pipeline, significantly

constraining our barriers and and [indiscernible] we identify And Looking will candidate gene We the advance to towards programs collaboratively ALS lead the SODX through XXXX, we with fields a of Neurocrine. GBAX program. work to continue break intend therapy for neurology. our

We data conferences, potential generating to the And conference around discussions continue and platform we active scientific will continue AD/PD to FD month. share with including exciting engage timeline. finally, in later our partners we at this are

look work moment Voyager the we’re year. take in incredible on a a to to And to everyone continuing forward the thank off to want I build great start. in their team Voyager XXXX. next to Together, I for

we that, may Operator? to happy have. are With any questions you take

Operator

question with Jack Instructions] [Operator from Allen first comes open. Baird. your you. is Thank line Our

Jack Allen

a you And clinic. to that broadly on the of so IND progress you a half And IND? you’re should Or and do that’s team SODX much. maybe timeline program in your well even capsids clinic? that a ALS I then rapid guiding you to the the have an follow-ups few is on a it tracer be as move about moving we the capsids all start, owned, the towards move I’m towards partner as of a an be progressive me. more could first wholly TRACER have we well, to program then and to made timeline? that the I gene about for a I But TRACER quarter. first know XXXX. expect could thank think over or therapy towards guess How similar assets Great, hoping congratulations course the to the talk

Al Sandrock

is Jack, Hi, this Sandrock. Al

hard the in So clinic partner very course, planned have to IND actually, SODX for But know our in the these partner it for well these And for their And from hands. timelines is may programs platform. ALS because, are XXXX. it’s multiple capsid TRACER be first very programs. of the we

will lot think XXXX, come it neck to INDs And play. that? is the But likely. timeframe of roughly add so and I XXXX have be will these you Todd, in do so but that neck into anything when a to

Todd Carter

well. Sounds very

Al Sandrock

Thank you.

Jack Allen

programs. And supply if is lot programs, And Great. think, other advance imports. Charles to just in and coming follow-up great targets if there’s of their see non-human a you opt some Cambodia. work right, maybe of I decided towards into then you to primates had the ability brief on and on back slowing timelines if a been hands so touch your some I could that programs, I assets of your around relationships. on River of targets the but outside few to was refer that to made particularly really there’s headlines non-human therapy the gene of not Labs primates, clinic. into those two your Pfizer question if wanted It’s I competence procurement out And have they And a then leaders also opt the Novartis comments on wondering any you moving those major any to I these to kind leverage preclinical rapidly and so forward. on wonder what get you

Al Sandrock

Every Al, Novartis and I real non-human primates. primate primates the I’ll company course, relies for the Allen yes, Jack, Thanks, problem. one know and this question non-human it’s Nunnally again. But, that is platform another, this answer of industry a I’ll answer our non-human the TRACER thing serious now in or have Pfizer, on question. have begins in of

a so are resolved anything not for gets whole and aware problem. because crossed this bad because my our I lot as happen there it’s is I’m this any keeping drugs. so programs, serious the in And for news of And today of but I’m of fingers can waiting pretty a hope patients industry. delay

I think, Todd?

Todd Carter

try risk, do I’ll closely. just at to reduce vendors but we that add to looking very Yes. use multiple serious we’re that

Al Sandrock

Allan, do add? you to want

Allen Nunnally

is thanks this Novartis. question Jack, Pfizer the Allan, for and on Sure,

each that of Pfizer those to relationships, Novartis target and targets there an for that not one with. did in option attach was one they So exercise for

programs to program on we respect But or are in So side, with targets information Voyager. capsids do Pfizer the partner rights have because prosecuted our to being any revert Novartis, don’t for those the the rights entirely the the about and programs.

for the simply to So those ourselves we or just have targets back our targets other rights to do on TRACER with those – with our capsids partner parties.

Jack Allen

Great, multiple the and on congratulations so much for again thank questions, you the progress.

Al Sandrock

Thanks, Jack.

Jack Allen

from line Our open. next your Lee Instructions] Truist question is with [Operator comes Joon

Joon Lee

co-ligand receptor Is or target thanks Hi, our question the sufficient taking congrats on identified. or have for the questions. the X on And I a co-receptor a receptor the delivery or you modalities complete than are Thank there identification delivery, delivery X, a for necessary the it and follow-up siRNA to that progress. LMP, receptor ASL a is is with you process have of coding and B, fusing such other as question. A, this the you. to the AV of or also considering

Todd Carter

vitro thank Yes. settings. it’s the for have identified increase we Receptor sufficient a this to is and receptor Hi, know transduction the the in co-ligand, question X that and co-ligand, Todd, you is we question. shown of not

something transduction. things. the for the So experiments result about some right work we with exploring modalities, further we exploratory of alone that now other increased it and we’re actively early ongoing to It’s to uptake expression regard and in [indiscernible]. delivery delivery question And increased can have and dramatically of of [indiscernible] elucidate other have

Joon Lee

capsids lower of able – by your has [indiscernible] to with XX this for the you compared dosing Great. are dosing systemic much how inhuman example, And to about so to with which a XX?

Todd Carter

Hi, again. it’s Todd,

it And question. target need. upon amount great delivery a tissue, that’s of you and So depends the the

to dose of you So than in the tissues. result at deliver more therapeutic liver, less ways. index a lower the that our hopefully in more two ability for example, possibility and One, the and And to is to in tissues. the brain, delivering say goal the can improve to you target results given can off-target to dose, which or sufficient the deliver sufficient do the

it’s targets So that’s off of the ratio this on question. target to

for some dose, we can the still In reduce dramatically of shown, our of a capsids CNS addition, have into to example, we with and XX-fold, delivery dramatic primates. that XX get the non-human

capsids, to the of for the AAVX to we’ve what both or a quite minimum, I into So think looking and get something often we’re seen and goals kilogram those with that’s our eXX lower doses. a from go the related is with capsids, are level eXX, seen per at

Joon Lee

final with performing you’re any three milestones from on me, few to I cash Great. given if for to guidance able the planning manufacturing, funding scale cash million provide but the your any collaborators, $XXX curious or in Thank to next one questions up etcetera, you’re related capital difficult expenditures know you. And based years. in-house it’s the in if and for potential

Pete Pfreundschuh

this Yes, is Pete. Jim,

of part couple guidance balance of parts we things. presentation I we a and So cash our think highlight into we do want our regards flow today, to did to sheet runway, ability I with provide there. with said the were actual XXXX going that

Neurocrine we with strong this well a sheet. the transaction $XXX balance as started We really transaction. I between Novartis have think year as our million

projections, Our existing. want that not balance sheet potential I do the include collaborations to associated the milestones are say future cash they flow with

year-on-year, and runway further was actually efficient XXXX, to And those million. organization we we very specific those regards our of So do beyond wholly were to cash, With projections. burn regards to we anticipate expenditures, regards projections that up $XX advancement overall, cash XXXX, programs. flow that’s as capital provide really actually owned don’t guidance extend although with to for actually our can our the go actually that that, although of burn our deployment for projections with our

today. described I for think, here guys we’ve as you

– of that development into trials. our I most is move of costs somewhat as the especially forward think with it we meaningful over increase will of we clinical our our the for are years, anticipation the as get next that couple associated programs,

an financing or the as X However, actually that well the really non-dilutive months that we’ve deals one did. And over $XXX we programs. with in do organization done over actually that see, of to we’ve our million said, non-dilutive associated can as is financings the been being associated we you so, with able with last things generated

So our be to be to game kind I of think, based into and company the upon to it is and we continue also future other able mitigate to arrangements. the through goal types can but do, of overall costs finance plan deals additional that non-dilutive able partnerships

Joon Lee

Great, thanks the and progress. questions for taking to and on execution looking forward congrats our

Pete Pfreundschuh

Sure.

Al Sandrock

you. Thank

Operator

[Operator question Our with Instructions] comes from next James. Leone Dane Raymond

line Your open. is

Dane Leone

differently, us take experiment do taking open? Can Hi, to next you. for getting the to need talk of through the get wise that the lead focused actually phase studies do in maybe the to will you drug to to thanks this And get year, required to SODX do year? IND earlier what on the the specifically then what part Said steps more that program need you still questions. that do bait? IND the you candidate finish just derisk of program into help Thank

Al Sandrock

first we what’s ALS, need think in to tests, going profile one effective already important a in that we combining to I’m for a is this capsid on Tofersen. Todd development In with key going we’ve milestones over SODX thing be we Yes, in us there’s words, is I with a to transgene turn Sandrock, that have developed, to order our it of track is I Dane, a nominating an candidate. that here Al other some think think minute. Hi, and

you there’s how and a and and will drug, be to month, approved into to this develop decision get As Advisory will make us insight lot even to Committee patients. next know, come give available going by it this the it an FDA to how month, of FDA that

tox GLP with going FDA candidate and do on to after next next year. and have be and the So start interactions still plan we course, there’s for to IND, identified, approach will will track the important as to that? you add for to studies, have – year. Todd, anything development which is of It’s we

Todd Carter

of captured have you steps the of think mean, mentioned. nomination the the I LEED that the now. I bulk it I’ll are identification next

of the on on tracking reasonably We’re to and in this still that first IND track filing year we’re for XXXX. half our

Dane Leone

Thank you.

Operator

with Our BTIG. [Operator next Instructions] question comes Yun Zhong from

line Your is open.

Yun Zhong

the good very you Thank taking for morning. Hi, much question.

I program reported approach a think antibody HERX the that do past, you data suited using pipeline vectorized had kind none that the So And given antibody is be approach? for? in noticed indication what you vectorized could of the of actually

Al Sandrock

extracellular, think I a cells at the Al, to But, surface cell mean yes, be non-neuronal ask is or kinds the here on going target this or powerful other any suitable real over been And developed technology to Hi, it’s will of Todd Voyager is it’s and cells, for that’s that years. start very either or in cells continue. the I neurons I CNS. of

path is get actually immunotherapy. very option Todd? cost get have passive have feel have efficient as a tau look, shortest And large a rapid are antibody. it number out. a the that of vectorize path, you program it proof-of-concept, targets. it’s and to the It’s to lead always we us. So, But we is passive the later to proof-of-concept, we options as available effective and like After the multiple pointed we immunotherapy, for we using pretty just

Todd Carter

HD we that an antibody stage, And we it. program. a program, still in we to pretty and excited that it’s about excited interested Are potential our much like remain see are vectorized value lot and earlier our of about here platform

continue of as the the results that in always we generated platform be and that data antibody other approaches vectorized advancement are And well. program. hopeful in with are to We will interested

Yun Zhong

Okay.

collaboration believe therapy of question is with So, I next collaboration programs the additional Neurocrine. gene were my FA also program was audit some an on Neurocrine. the part and

now be that to technology reasonable are platform? they it based assume on also So, TRACER to going is

Al Sandrock

again. in This the was both Sandrock now that we it’s the Alfred is with novel the interested other moving is of before and drugs two capsids. to – that original the mentioned programs for deal very Neurocrine fair those you availability forward signed TRACER capsids. this But say Yes, free are companies

Yun Zhong

Thank very you Okay. much. Great.

Operator

Wedbush. [Operator next comes from Instructions] Our with Chico question Laura

open. Your is line

Laura Chico

guys. taking Thanks the the specifically much Hey, I very related for I Wondering good question. guess collaboration well. the to Parkinson’s, one morning, GBAX for Neurocrine have as program.

If little days then you just make you kind speak to you a of could leading influence opt process maybe or co-commercialization more the but could bit might or further thought recent the the that discuss of there. a of there? a co-development, exercising Friedreich’s if there a change to been just Ataxia in kind early has what to incentivize little kind continue forward option, of this obviously, Thanks space, the second, approval very path much. in here, that to wondering still promising deal in terms And wondering how would

Al Sandrock

to again. Alzheimer’s is tend common it’s for be to Yes, to to very than Parkinson’s clinical able all Voyager or imagine Hello, and would trials, way Sandrock, the hard like this commercialization. disease, be disease which so large, to late-stage diseases rather go that

were so program. partner excited was because when GBAX excited gene, sense. colleagues very never a that whether and of of about of So, we very people it should it at the with much about question always a those, say competitive Northwind makes was was process. A it it a would question I lot was our

validated it’s think a highly I target.

this to the outlined and do for we it to decided we And non-dilutive of just deal other so programs. pursue morning. one And one new us provided revenue

what in not it’s to But or the GBAX that very abnormally gene we [indiscernible] terms the which fluid. whether therapy Phase able with measure X that produces should the it not or after us. whether see In the spinal to spinal substrates carriers X, And is in build disease. reduces the Phase will fluid Parkinson’s possible of Phase in design to enzyme is yet, be Neurocrine tell trial X. after know up we we it’s the And don’t up

X is. but – know that it yet, don’t And I the possibility have biology a of perhaps, on we that that outlined. depends proof but So, design is just Phase what – so after what we again, trial that’s

In it’s last This drug that terms think activator, disease. of the it’s it’s the exciting. very is no field. very a for the – week, whole think by I a disease very, and And encouraging approval NrfX I had treatment I of Regatta, don’t the terrible until

so is Friedreich’s that Ataxia boats. happens get think lifts I other And all the involved it typically first just what approval, is, after and companies

us. I so And think good only news it’s for

Laura Chico

Thanks very much.

Allen Nunnally

and Laura, data an how thing at that just royalty Phase a have is for look from Voyager on are healthy between the and will situated trial, see to attractive share, the for a we milestone cost/profit this option optionality X we to U.S. only XX-XX track. Allen share, and really cost/profit is outside, Nunnally, options way get preserving the one take will add for good is we

we are Phase X. to optionality happy after preserve that to the able So, be

Todd Carter

comment the from the what Regatta the last a approach. one tourism else mean and gene different therapy to surface, on of by mechanisms I which are in addition works our

And all. not the approaches are two exclusive at so

Regatta the quite program. So, about we excited are

Al Sandrock

should what better we just the know easy course believe in change can of X I an for our the have patients us huge it positive I time, power so And with measure that studies. Friedreich’s outcome measure Ataxia. idea of happens for mean event, I outcome see we a – approval, the over regulatory We now what needs trial Yes. be. gives the Phase we should that, be see add what to it’s

Laura Chico

Thanks very much.

Operator

Our next Kulkarni Sumant comes [Operator with Instructions] question Canaccord. from

open. Your line is

Unidentified Analyst

developing Two humans? now, to to congrats Receptor-X the that given level what’s behalf been receptor level Hi, you of time your primates expression on and the translate on quarter. that some for that on how you This and have questions Sumant. might is asking your Ross mice? regards variability have are on novel across confidence latest questions for capsids actually identified, And thoughts these

Todd Carter

Alright. This is Todd.

the that capsids enhance the humans, And and So, mice versions in and from each non-human in find all species right all in the know the orthologs vitro those orthologs the cells we Receptor-X in is for our the right of species in that species, in that we those in of expressed studies. protein primates. in know places the transduction

we And think all of the this there of that greater of is translation we humans – success data. the of are is chance so de-risk. a because greatly And

Unidentified Analyst

Okay. is so quick out might of to this of is potential there follow-up brain, concerning effect And a for capsids kind if kind of of area is, this if be concern? then, mitigated, these and any how

Al Sandrock

to doesn’t of – of at all the they RNA transduction to least at significant actually liver, so every into at anything want organ, actually to But expression our not in single de-target CNS, the example. it’s we so novel do we are efflux, no, organs multiple like looked periphery. of places far, in yet. these the we capsids, what the TRACER messenger look add there the transduction And it allows point to for organs we liver, do when get where is Have importantly which other find simultaneously. decrease capsids look you anything? tend get like is the many Todd, organs check the Well, we in platform us

Todd Carter

less involves those And been is CNS, both is the our team deliver to your directions discovery does that capsid to to that to focus, the There process for opportunities and capsids which deliver tissues. the One iteration. in a looking better CNS, actually. Sure. has novel question to off-target the well

then the And there screens, can or the the are can then other to what found maturation we with to are have delivery similar initial we secondary increased is tissues. identify increased that variants delivery screens. there CNS, to So, identify And CNS. altered maturation and process, and we delivery to capsids then

other captured select those match mentioned Al to that potential off-target a tissues something that we for opportunity each side includes is profile the panel avoid diseases to for profile as to want TRACER can and well. earlier do the the target for build so given we disease tissues of attach and we our we the have identify deploy capsid risk. we and that another that that And variants, in want is tissues a of that And the potential

present those muscle capsids target well. with identify to for able or are others opportunities licensing tissues, we as if so that other And

of So, TRACER there the is a opportunity platform. in lot

Unidentified Analyst

you. Thank

Operator

next Wells Our from Zhu comes with question Fargo. Instructions] [Operator Yanan

open. Your line is

Yanan Zhu

questions Hi. Thanks and for congrats the on quarter. taking the

marker, question SODX, as validity surrogate therapy it of PDUFA, upcoming think as if out am FDA wondering this program accepts given on light could paths, a if would for efficacy. a common for at first development turns surrogate therapy a Thank neurofilament you. on gene comment that marker that the So, And be gene chain I ALS the the path do person same well? you you

Al Sandrock

neurofilament that am for question relied be valid neurofilament great surrogate to surrogate think out very find as something of we release. or endpoint can decrease all that track not measured risk. I myself, even earliest I whether it’s use at decides still approval, tracking. fiber by can and way upon I trials nerve as And a a marker it integrity can will not we soon. And Nevertheless, But gene in believe do the efficacy. I whether a a therapy are we as That’s a think our is regulator. FDA we use least it to clinical affecting

engagement itself we do target us biomarker. eye fluid spinal at SODX And on reducing thing very marker in approval, in published FDA as England to the even they efficacy, should or it New the the a it’s what the expression for addition approval in FDA NSL, have makes of we spinal pathway of the look do fluid. if that endpoint behalf useful So, could patients. surrogate regardless shorter well, decision I an valid on the consider as Biogen can be Journal decision that the in is. of it, just a sort of soluble use SODX same think you a has levels The But of

us So, all useful measures these understand of our is on to are both whether track, help right of very to use therapy and plan we the gene it.

Yanan Zhu

to target? with Have conducted and what $XX on target might, to with in And they timeline pursue question have make partnership the go million that that it. tested significant payment? programs, regard the their then to the the Pfizer another any TRACER studies, conjunction there may example? animal Novartis, the option Is to they on additional the with for two with collaborating the for TRACER, decision have Got fees? some when for with Novartis decide And they licensing extent studied targets a to they

Allen Nunnally

Yes.

the So, second first. Allen, piece this I will is take

million months to like digit to it Novartis then million same that, $XXX terms two a for with add be target So, either would million do to original they they and under they high-single target, decide to the $XX the in if the additional targets, XX choose milestones and they royalties. expand targets two do which same as whether ability attract to $XX.X have or downstream would to add agreement. the per one the we mid the per agreement, to exercise potential is If

months make Yanan XX was decision. of first the question, – what to just this if talk part they the that’s, am that? you And So, could the I have on that sorry, to repeat

Yanan Zhu

Right.

those TRACER Pfizer with have in capsids, have Novartis answers. these And the thanks targets together specific So, – and animal their for studied models.

work TRACER extent they what the they have to before capsid? So, the done opt into

Allen Nunnally

apologies. Right,

to So, both for had capsids a in look evaluate consider whichever to of instance, Novartis period wanted to and exercise. each X-year at, they Pfizer our

they experiments exercise capsid. own would to on their like decide end conducted help their whether and which which to they So, on them

about partners, capsids more our capsids. own us and help but for targets, more of target, of their So, its that not the after exercised experimentation There end CNS Pfizer so two the we information Novartis hands evaluation. to as in a that’s an to specific the their information process, be back our that our shared at understand with valuable to exercised is of to intended option just and is the learn the has of that is Voyager subset own results us neurologic of to targets for

Yanan Zhu

capsids. just the the of And wondering for TRACER Thanks Great. the color. lastly, evolution on

that? experiments might So even conducting on potent about update hear additional have i.e., – some first capsids? they we more will also more all, TRACER for and we the the are of you And taking with about all outside on selective had and CNS capsid having how CNS, reducing the And targets efforts update further when questions. additional some that front? load, for Thanks

Todd Carter

this Todd. Hi, Yes. is

continued CNS, are application, timelines. the on that’s rental our regard interested and use starting spend so can’t in of far, the so capsid of the different commit where continue. continue our been So, regard the certainly to and I to capsids, mature bulk different we has loops seriously with non-CNS to we With in surface. capsids It’s the side. non-CNS efforts to on our something we looks any that to on exploring pursuing TRACER We those processes AAV to evolution capsids, all develop, that’s that absolutely capsids, different something at the team our are focus

the continue the upcoming data, data We will that next and will at some the be we will share I that arises. platform our TRACER comes of out new think ASGCT, presenting showing be to conference we as work it

Yanan Zhu

looking presentation. ASGCT the Thank to Great. you Thanks. forward

Operator

[Operator with Jay Instructions] Our next question from Olson comes Oppenheimer.

line Your open. is

Jay Olson

in follow-on as And congrats targets lessons my other thank and new inform all I sort you have questions. you And CNS So, will considering? for are then targeted please. second, could since therapy interesting of optimize you siRNA that how by ALS on they then silencing SODX tau. you talk your learned initiatives be about early gene research progress for are one research a what gene the payloads, siRNA taking Can the from your

Al Sandrock

listen Voyager siRNA we years. and huge goes of tested that to easy a Hi It’s some many our it. honed versions we we capability find How ability start believe we will siRNAs. etcetera, the address have over how Jay. continue. thing will and harnessing this, things that that an I the I thinking is Todd But not this the on into like And vectorize over potential. to published

there learned we be our including future SODX, siRNA of all to And mean siRNA that lessons think programs, the so that – there apply Todd? I yes, will I vectorized will tau. siRNA for

Todd Carter

So, includes we one things about at This payloads. is good then talking siRNA gene that the replacement, here can about, talked we Voyager we of antibodies kinds that deploy the different vectorized of and here. working about the the are

Some we mentioned through of first-generation Huntington’s the our I that that came did program. honing

we Huntington real and to approach are we well. an exploratory That continue Huntington’s way, this early is both specific year, which of approach looking We a announced form was Huntington’s dual the to knockdown that’s a modifier of MSHX, pursue disease. as that earlier genetic program mutant

have. in we we had years. have efforts expertise these the in approach, programs forward looking And payloads of a abilities are So, we other siRNA to the we over honed the addition that deploying lot to that

Allen Nunnally

I is And is Huntington’s gain to there also have straightforward disorders. that add the mutations also CNS I maybe lot add Voyager And a how function would of toxic of siRNA, to develop. that. figured if are mutations scientists not could do for out that Yes. programs specific is which that

is number capability So, having to for a down us very targets helpful knocking this gene of very, and large up pursue. expression of opens

Jay Olson

be with treatment that Alzheimer’s? I landscape Excellent. fit the It’s guess very two and silencing modalities in of program, can gene could can antibody, about ask I helpful. if one future siRNA potentially follow-on the the And for your advancement you tau how with please how complementary just will talk fit then into your anti-tau and

Todd Carter

Hi, this is Todd again.

for you questions. thank the So,

that the neurodegenerative think go, how We antibody came the vectorized target antibody it’s antibody that the extremely and that against program. as out our antibodies worked about approach And animal series the is we excited are targets we we are of and of We efficacy models by epitope target. a approach our validated. differentiated both tau earlier, mentioned our animal and identified in well models

that are telepathy tau those. vectorized we continuing are so forward. one still be we And to that approaches that effective, reduce That of think there potentially and other to is could said, really move siRNA in

tau of while, tau still antibody. intra-cellularly tau it than tau, affecting affects independent of a is mechanism it’s reduction an the So,

combine interesting, anti-amyloid So, the think have combination. also potentially so And disease received and of to with but that as accelerated interesting approaches Alzheimer’s other they are in there are I potential recently our including the course, well, those approaches, is approval. then therapies

neurodegeneration a this we I a think renaissance say pretty are bit excited of about would disease, and are in I and So, Alzheimer’s we progress. of

Jay Olson

you Thank the Great. for taking questions.

Operator

Sandrock over And any I closing any showing will At time, the am remarks. turn questions. Al I this to call further not for back

Al Sandrock

directly again. us with follow-up us with for feel to Please today. Thank other free any questions. Bye. joining you everyone Thanks

Operator

this presentation. today’s and Ladies does gentlemen, conclude

now may You wonderful a and disconnect day. have

Voyager Therapeutics (VYGR) 7 Mar 232022 Q4 Earnings call transcript