VYGR - Earnings call transcript Q4 2017 | Voyager Therapeutics, Inc.

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Operator

Good morning and welcome to the Voyager Therapeutics Fourth Quarter and Year-End 2017 Financial Results and Corporate Highlights Conference Call. At this time, all participants are in a listen-only mode.

Following the formal remarks we will open the call up for your questions. Please be advised that this call is being recorded at the company's request. At this time, I would like to turn it over to Matt Osborne, Voyager's Vice President of Investor Relations and Corporate Communications. proceed. Please

Matt Osborne

the highlights; Good Bernard Voyager's program; issued to recent then program outlines Steve will provides a for Paul, and Chief and we at financials highlights and on questions. corporate Shannon. This conference will the Today our Senior Officer, Development, and year briefly voyagertherapeutics.com. CEO, The corporate and we and and release the of the morning press VP call Voyager's disease for your Voyager's our for discuss corporate morning goals Ravina, will review you, XXXX call, financial President the welcome call. will results end Henderson, and Financial Thank of Parkinson's open quarter Medical Officer guidance year-end and XXXX. the review Corporate Chief fourth our and release, the Jane up available which is

included we statements begin, refer call any future to that Company's remind to represent Voyager you Before or circumstances. forward-looking events just reflect to as call these today, the release update to materially or that as well press XXXX. March filings for over such in statements. to Voyager's by obligation the disclaims XX, factors this will to view of Please implied today's statements concerning the information Steve. from I risk could with pass results as actual expressed the cause SEC differ those that, With

Steve Paul

deliver development. to with operations, program of including CNS enter the human Parkinson's many to is advance Thank to preclinical diseases Alzheimer's manufacturing, clinical collaboration pivotal of bodies continue and disease including you, allows us exciting in other and business the and strong performance XXXX was that Matt, trial, severe good important using functions disorders to pursue neurological to with that an This evident clinic enter an towards AbbVie across group Voyager's about monoclonal new AAV and in morning, pipeline programs us exciting everyone. other development, a that XXXX targeting allows disease. resources business a

longer-term dopamine earlier were we For that make their restore reported ability levodopa of they control believe in brain's of therapy offer to the demonstrate to way disease. stages during motor better the the patients recently function VY-AADC Phase initially VY-AADC trial and data can followup continues that their Xb to did response the from

clinical trial mechanism safety robust, proceeding will as with Bernard that data Xb pivotal a goal and of trial have we placebo into well-designed this before effect highlight placebo-controlled and As fully accomplished our durable are briefly possible of and that of results will Phase due a confident motor no thousands clinically to this hundreds range the and trial a believe provide efficacy and field recent chances a to life. of both that set. and success to for and we a we program the most Achieving discuss, set in high respect the the unique we've are action with treatment in maximizing our of X/X of placebo-controlled disease design program pivotal patients Parkinson's function this in one-time the with allows the could of bar flexibility address very doses Bernard The any as explore is improvements durable success. of Phase of to quality

advance Huntington's disease to the from Friedreich's with IND two programs ataxia ALS, Our pipeline in filings and programs expected XXXX. continue

ataxia applies and of a best-in-class the once ALS disease, capsid, the advancing and our to clinic of Huntington’s our into efforts simply clinic that our is advancing programs. but each in success optimize Friedreich’s our programs, have SODX, probability not approach delivery high ongoing the goal programs the goal, programs transgene, Now is and preclinical potentially to

antisense oligonucleotide and from own For administered clinical Huntington's us development therapy encourages recent our by mutant gene supports lumbar with approaches disease and done and approach. a candidate administration in wild-type monthly lead view bolus Huntington's targeting again be data will won our intrathecal in particular which

proceeded the providing than for achieve achieving potential cortex greater before maximal important striatum. Huntington this for be nonhuman knockdown in therapeutic approaches with able forward sufficient an the benefit. on we is and the others and into cortex look advances in these in update development, program to this preclinical optimized and year. clinical protein knockdown to to other know vector in and you striatum with Huntington us delivery our to both primates has We it data importantly However, believe We relevant and down the approach later

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running be other exciting goals our program our an important up will are disease reach. to this be and XXXX year year, as programs well company within our pipeline turning Now, advancing the pivotal for Parkinson's and for will our

with to into incorporating C our VY-AADC plan this a X/X program from we disease. advanced meeting the for feedback complete FDA Specifically meeting type Phase Parkinson's pivotal for

and the trial motor X using infusion data function VY-AADC will We six trajectory. the months from provide of safety posterior Phase

disease to trajectory longer-term trial. advanced during biomarker, second XXXX of mid life motor XXXX plan through quality for in first data from posterior and the patient and X half to plan the pivotal from patients function Parkinson's During and we X safety Phase Cohorts X provide of planned in program the dose the we

preclinical programs We during to two vector towards or exploration of ataxia of filing applications SODX, to continue programs and advance additional will Friedreich’s through routes from trials Huntington’s, optimization further IND the administration clinical leading XXXX. multiple ALS

addition, partnering as platform as we've business technology done as we In progress well evaluate with collaboration, Voyager continue to our identify, potentially AbbVie programs our opportunities, capabilities. and including will development other

earlier and our Now, Having which great take its been it years year six as Bernard, in we announced pipeline assemble inception, we a company well with the Technology of I with terrific the plan and my role over since underway. before member is for made Voyager's I the succession in turn Science continual helping to as Board accomplishments our and the will Voyager to pride team. Committee. leadership the of in a with member progress of a I great in

science and review new with disease passion hitting look forward for antibody on to with my Parkinson's our the and time the companies Given ground our Bernard discovering AbbVie. program. I running are collaboration spending progress more tau Both this medicines, now will with collaboration. program the exciting

Bernard Ravina

the identify address At X or everyone. VY-AADC. the some I'll morning too not maximum good response with since concentration readout. tolerated and we what Phase and recap our first a come update interim to is up us to Steve much results those lead dose questions briefly provided high Phase Xb level, that think led understand does have recently We've that has trial the the and dopamine. dose Thanks

higher recent enzyme robust This mechanism the levodopa of trial data The second in motor are less improvements in in the with daily into as infusion increases and pivotal of that XX% of But the exactly one hope is the These which medications medications VY-AADC a also off-time. program troublesome meaningful quality first has on-time shown time they improvement time. be three range activity time pivotal administration a especially transfrontal in a two along strategy for above trial. Cohort oral to both similar cohorts. are achieve to only the efficient endpoint from day posterior at this in confirming experienced the months, related of X XX take hour dyskinesia At sustained a on in X.X for X.X increase Phase mean are studies. more population. of action. thus results results dyskinesia primary data or same very decreases dose trial without continues stimulation had point. function of XX used approach X data our to show using without and related delivery with patients Phase Cohort patient to a X patients and reductions a dose placebo the diary one-time represent the levodopa we days X would this in The to At dependent a generated is in hours and program. durable some first any what approach function the shows in at manner the brain months, deep expect clinically need than The what on-time, of These

and patient disease period, assessment improvements overall and function. XX. of reported how These Parkinson's these doing can X meaningful item and an motor are dose patient Cohorts questionnaire both of indicate activities important over For in the VY-AADC the rating effects in Parkinson's XX look unified living patients PDQ are daily we well-being dependent, or in of longer durable measures a of X. and at disease demonstrated scale clinically measures treatment reflective

course. they a levodopa their did what disease robust So for patients? does It early response count all reliable on as can they and this mean to in means

We're Cohort The in manageable oral of activities been program. potentially fact, cohorts pleased work improved balance function the and that speaking. simpler trial control involves the In levodopa. in the their able of wider the teach including to patients the X motor with be to procedure is oral to and return of concentration able function administration a with durable to return with to high or including in into that control program, coverage to levodopa. are to may in pivotal of response the other delivery more goal results the Across Cohort ideal and level activities were the able have especially titrate we the This know given and the to the very meaningful of of putamen posterior daily patients really a the stage patients the to skating. range public when motor X, they combination overall return bike reflect job greater riding of dose

raised address couple questions the release to Now of a the data. of since

In subject driven at PET response, The cohort durability levodopa three First X years and on Unlike increase taken X XX benefits on data. motor X years fluorodopa single of of cohort respect or uptake relevant dosing, the Cohort to Cohort stable measures. initial context across in active baseline and in average three. a minimally relevant of Cohort time in evolved diary received the time levodopa. shown measures. signal which based now is Data as in of to reductions modest VY-AADC by doses all three lowest from their two X be in in must and clinically L-dopa their placebo small multiple overall on months of doses years over after observed clinical improvement. this a similar XX have response with a is the believe after to better to by dose we is the on show over responses on increase

three-year PD are time, over data these that progresses very Given compelling.

reductions on-time X from time without with Cohort maximum this to plateau months, Now reached the to dopamine troublesome of we the baseline more on pharmacology. in a have and one less dyskinesia. XX diary Cohort of and severe at XX X is dopamine in and the patient They This a levodopa-induced one dyskinesia and than It Cohort dose effective than the and higher dyskinesia that larger This dose more point, months appears with the Cohort X. X. in who on in X levodopa dyskinesia. dose interaction conversion expected X resulted suggest This baseline. entered of was which of consistent oral each received logical X and not VY-AADC X AADC severity resulted concentration. at dyskinesia Cohorts patients improvement in with had to Cohort on more levodopa average in similar trial case and the Patients X of

we've in can evidence dose motor patient This dyskinesia or order of less instances X. with motor improvement balancing diary the in clinical in compared reductions in striking at AADC other control, they Cohort generally example, XXX less by time by given For levodopa the small time absorption patients management Cohort measured driven will mg as These levodopa that the a clinical substrate erratic one however response in to evolve take reduced balance good AADC and in that of control. and X resulted results over of to are to doses of may or data preclinical XX levels time. off-time lead as mg in underscores These a first and levodopa. at both Such a in enzyme on-time be treated. in oral cohorts is seen when to variable benefit

will Importantly, treatment. the trial design pivotal management apply program the post we from dose of selection to of lessons including this the levodopa and doses

the to pleased given program, program. of data the with the X from the Cohort Finally, we're design pivotal

placebo-controlled data that generated within that from months. months above two than baseline a nearly detect cell Phase a X.X staggered going portion. the past reminder, and showed from a therapy X with gene the hour X disease improvement the powered in Cohort given Phase on-time hour Phase improvement dyskinesia less from XX% a with one without at is XX powered baseline XX% without to XX X on-time range is that to to into in X trials a The X our The trial. placebo. Drawing is X We're buffer in for from at Phase XX for Parkinson's Phase design troublesome As months this provides dyskinesia This and hour improvement. versus parallel hour our X.X well X of and placebo. it X detect to the improvement at Phase us an hour troublesome baseline Phase versus

will VY-AADC, safety, the important to themselves. the of capture patients In secondary and addition payers program regulators, pharmacology we're durability summary, many that pivotal pleased the endpoints are and magnitude In with walk now to the of forward and I'll over Cohort Jane through with more the detail. program. call in effect, with can our going the results in you financials plans the X, particular very pass who

Jane Henderson

of next good the for ended in move or few XXXX fourth we I'll morning. loss guidance reported to million the million $X.XX GAAP financials or share $XX.X spend and a compared $X.XX for the period share Bernard XX, before XXXX. to Thanks December $XX.X minutes Q&A. the per per Voyager and quarter net reviewing same

The programs periods recognition increase the the allocated full the for of recognition estimated under decrease compared of to XXXX same assessments XXXX. the share, for quarter of XXXX compared $X.X based December the per to rights period $X.XX a to Parkinson's Sanofi the reflects to For to option. milestones option decision revenue exercise a year loss we've Sanofi collaboration. of of revenues changes portion million XXXX Voyager’s license net for agreement. on not development for for year in XXXX collaboration to efforts preclinical XXXX loss quarter its in as the for These million and the agreement various for XX, development compared period XXXX collaboration December full the of full reported fourth a in under the million, GAAP same of were Parkinson’s then Collaboration period $X.XX revenues $XX.X the fourth ended quarterly compared same of share million the full in ongoing the certain $XX.X reflect revenues XX, to by under result million payment Collaboration ex-U.S. Voyager research XXXX the per in program of collaboration or net revenues $XX.X in vary XXXX recognition anticipated collaboration Genzyme services year XXXX. or ended ended $X.X reflect for and agreement. the the the the to Genzyme’s for can compared the and year for Sanofi the to revenues reaching million $XX.X programs for provided the

XXXX XX, guidance new XXX. the recognition As as adopted of has known ASC on FASB's January company revenue

our So agreements. collaboration as to going related to how will we forward make revenue we recognize changes

periods recognized. million future approach give same as compared new of guidance for fourth through for on sanofi XXXX. in period indicative be $XX.X the year to ended million the We and retrospective amounts the collaborations. are the and XX, necessarily the December in AbbVie recognized expenses and development not the Therefore prior quarter recognition adopting XXXX for will modified revenue be revenue using $XX.X amounts guidance Research were will to

fourth development compared year expenses G&A the the programs. support support XXXX and programs. the period the $XX.X R&D to million ended trial $X.X personnel and $XX.X of securities Phase XXXX was ended were costs and pipeline the quarter X facility million same compared XXXX. in advancement for due to and primarily the million G&A primarily For expenses increase increased of ongoing year XXXX $XX.X Total the including for facility to ended personnel for The million R&D debt $XX.X XX, expenses $X.X increase were related million. the of The year were December to million million to expenses our VY-AADC, and XXXX as for for in in of pipeline to cash XXXX. to cash, marketable XXXX $XXX.X for compared equivalents costs our our

payment on we project $XXX This million to and expect of the stage Now, towards with the way run that recently we million to potential including pipeline our around to expenses as ongoing from early our of that, as remain well plan not with received include advancing expenditure like our We advancements and manufacturing fund development up un-partnered capabilities. capital exciting based We Operator? product the into Parkinson's open the platform be $XX cash enter and of questions. AbbVie sufficient turning as to committed vector XXXX. programs cash transactions. planned includes progress the platform now to cash current with call million. end $XXX operating opportunities the total the and into guidance, collaboration business investments to into program existing new about AbbVie. with XXXX operating and cash will preclinical genome for our does total we XXXX is would BD in The some pursuing approximately With the the pivotal XXXX upfront requirements

Operator

comes [Operator Jaffray. Piper Charles Duncan first with you. question Our Instructions] Thank from

You may begin.

Unidentified Analyst

This morning. Hi good for Charles. on is Sarah

then what that posterior for point this So use how can beyond the remind dosing help success? as medicine reported are procedure or procedure further outcomes at just time example, pain are development? and long other the this taking is patient trajectory using And a will you me you kind procedure of in characterize

Bernard Ravina

thanks Sarah, Thanks, for question. the

So just that trial more goal develop the to commercial. of scalable pivotal a reminder, really the be whole for infusion and was approach efficient for that program and an would

So in DBS X what to given hours range procedure not probably down we've been time say accomplish X time it's staged overall hours and the under to is X puts to DBS. one-time able overall trial we're the like compared well X to to which it procedure that that off is

and So consistent we approach and then will we're the very think the X more repeatable pleased before. infusions we good with and going X used be transfrontal gotten we've very that and Phase into that

second on are In be we all terms have of and of all have outcomes, end and in point who of initial you been updating will subjects that we quarter. on for that have six-month made trial data same measures it the the to that the available reporting near the basically had

Unidentified Analyst

Great, you. thank

Operator

you. Thank

Birchenough with Wells from comes Our Securities. next Jim Fargo question

begin. may You

Unidentified Analyst

Jim. for questions. Yanan [ph] thanks in This for taking Hi, dialing the is

the I'm of Cohort X at similar more it's to talk Thanks. in of trajectory baseline about the to particular severity and and similar dyskinesia could whether seen in or X patients posterior the in characteristics the off, first X? Cohort more baseline So interested you it's trial,

Bernard Ravina

question the Yuan. Yes, thanks for

more as have we patients quite at but range dyskinesias. and first you should have as those of more look trial, of severe the representative report we get range typically overall the results, we second or is less in there you with well So for the you patients will if with what trial that

to see it understanding our Cohort if as using link of the baseline study we are we'll like and again that so We concentration X have Cohort we'll those X characteristics. unfolds that in and

Unidentified Analyst

in of And beyond six terms enzyme activity, I've forgotten you treatment. whether also AADC Got measuring months the it, thanks. are from

see change do you three amount years enzyme same could of do Thanks. enzyme there levels? any in you If X share or in at you the whether is Cohort

Bernard Ravina

had in term repeating be will enzyme patients. the PET just we which long our is to up yet. at longest Nobody starting measure follow So term occur follow-up That's their up extension UCSF which has are fluorodopa scans going activity. that the study has of the

So refer months a it X changes few why activity is enzyme will to had them active in which yet. as reminder, dose. A clinically Cohort very minimally we be modest

informative And will their which that so, looking X there has and may Cohort tell be for durability measurable changes of versus able activity about enzyme to good stability. not be

Steve Paul

the that expression academic for enzyme primate But activity. our but no study every stable case out saw shown in done by let data, scanned diminution me both AADC four in patients of was Japan Bankiewicz, data, underscore that. in the a the two group Professor literally by and group years preceded XX study, In Japanese really year in non-human in also PET Bankiewicz, levels he to this and years monkey. the monkey by have dose the

we're to [ph] that neurons, finding we in is going stable putainment the these expression the in given So neurons be these the think vector durable. meetings where placing very very

Unidentified Analyst

a Great the understand adjustment to dose trial pivotal and more titrate the little try in design. lastly, about

I feeling the function. the be to think to their adjustment of the is of discretion may multi up dose their patient according

that a that dose back to true could and reviews is from they way and the the step entirely is, them patient is to did in question prevent and my also dose case patient also up overshoot? Thanks. the there once overshoot they So is

Bernard Ravina

Thanks for question.

of that that go or and doing doesn't to fact reductions of the natural levodopa part each back, accustomed is testament the a down a seeing pivotal the dose people take as people XXX this part are getting that just here, management of can And something real in to the more especially definitive be related important even course. program. it's substrate happen medications not So we're mg an this will step pharmacology

do in want in how The neurologists a do quickly learned work tremendous X will on for something to do exactly Phase subjects the So the of will who to what the that we've then amount provide we it. with are how and the terms study. to reductions in you guidance it

we've reduce not We that to in did third and see is too learned what quickly. the over cohorts reductions. think I

in almost it's optimal take Now patients experts, we're function with I you're able on able an to PD yes, They're accustomed up. way it important and to of advanced titrate down. that something importantly are to the the back way lesson always forward but titration the it medication the and is and think just titrate doing, up absolutely on it's dose Parkinson's now that is increasing motor to this doing neurologists,

we're do Phase especially Xs. that learned with all our to So these we've two that, ability confident in from

Steve Paul

levodopa really one what some in control as is is reason LED there's is bit function to working, be best and on day significantly lessons this of go learned of learning down a seen that's and this believe of do Bernard Yes, which to is doing. said, X.X to a per grams this clinically, very motor normally curve how equivalents we're are from here. we not to there This unusual but

Operator

Thank you.

Our with Harrison from Morgan next Matthew question comes Stanley.

begin. may You

Unidentified Analyst

Matthew. This is Thanks. on for Vikram

on, of you get you're there questions FDA, meeting, C you to types are from about to be the sense have of issues what the our clarity kind just looking Thanks. get any what helpful? a context get looking to Type question of from that would wanted upcoming you provide could is what meeting, So

Bernard Ravina

Sure.

filed we go the IND given X ahead with clearance to were So the Phase and trial.

the and really So is overall transfection study, those the no those. agency had triple to compared around the in the with terms of design the safety, baculo of around comparability comfortable comments material,

going program Type Phase the is the rest in of the So X/X. C to about be the

is evidence safety So on really to what much going needed, dataset. of overall we're what is kind how focus and

we'll able program this to other in other endpoints, things course any disease, different advanced be the like far. established plan. on here well Parkinson's focus to an has are analysis are given understand enzyme The other that fluorodopa this that but than Parkinson’s The role endpoints very the replacement, of the of PET is things progressed

how understanding they and plan. evidence, analysis of an of the mentioned I those get we'll as view So that evidence, kind

discussion will So areas be the development. at point I think pretty of key in typical those this

Unidentified Analyst

thank you. right, All

Operator

you. Thank

Our Marai comes Instinet. from next question Christopher Nomura with

begin. may You

Christopher Marai

good here a shift Obviously great morning. proof-of-concept taking the Huntington's as Maybe gears there's question. your I'm little about work thinking pipeline. been Thanks Hey, bit, for to some establishing construct being in wondering disease. you're your SODX [ph] about helpful Obviously at you've potentially of that triaging next program. how the looking was with as pipeline I to respect talk opportunity assets. the

AAVX walk variant maybe us the and why Thank the or your then how us And help as given through about forward? know, X use process You think today? I us there, and making going using proof-of-concept decision not great stereotype use context just progress it remind be should was there think and we you're secondarily, of you. AAVX, an may it

Steve Paul

great questions. question Christopher or Yes,

cells Huntington's us adequate of key program So and in for relevant case knockdown the and delivery both Program, tissue. the in the relevant SODX the the is adequate in

spinal So up down out cord that of stem today. in in brain in knockdown in the frankly, a we to the And and for the ideally. see need and been we neurons and the of see SODX having world lot motor done SODX literature already don't out

the the Similarly there's not look So more ganglia, if putamen, that's affects basal a clinic. before on a you And patient evident but caudate can whole cerebral the we cortex. very pallidus, very, at globus only late-stage the intent simple nucleus, on a into the that MRI Huntington's a challenge we sure for of we're in Huntington's get do making bit that scan.

to a down So Huntington's knocking Huntington's we see need of to degree. silencing or of sufficient

in data knockdown Now that Huntington's the you’re in of CSF. antisense aware CSF has shown oligonucleotide of

in the that's far in it encouraging, I so reflects happening cortex agree, and but in Now in the as brain particular. only what's

programs are sites we those are, do relevant So in obviously make now chances throughout not before monkey of advancing monkey in larger for brain optimistic using silencing We're that achieving to and than brain whether exactly we're I brain example, kind obviously, we're very than we is give human with confident to brain, good will it humans we get that do using, sure low. cautiously in but can which talking knockdown but if regions. AVX we reasonably and knockdown achieved. we advise administration those monkey a still Huntington's do of smaller that, can the can't mouse sure And we're ascertained work of brain because that Huntington's or be into of the for is our sites evaluating goals. we're us who a and we much is those in going any capsid believe But the can that a would too about these there where achieve less, anyone clinic, in make much you've much a that of

Christopher Marai

some Thank you. Okay, then of monkey data, see the when will? we might you brain if

Steve Paul

you later We’re the have we'll data and that of year hoping some for route using. more we're convince this will capsid you that administration of the

Christopher Marai

Thanks.

Operator

you. Thank

Our with Baird. from next comes Skorney Brian question Robert

begin. You may

Brian Skorney

do of high at around little saw and AV of you're that dosing? interaction think the ataxia guys. number just to question. taking the looking any pipeline, one? that comment get where you to you you maybe hoping SODX, local on can and this, for going you Steve have of systemic was Yes, at risks with kind thoughts when we dose in publications that terms Wilson's then think you recent And also good maybe bit require highlighting what the Friedreich’s you look Thanks Jim morning systemic going are thinking just on ataxia Huntington dose a Hey, I can kind that's any concerns to off level away as Friedreich’s high AV of

Steve Paul

Yes, great question.

Just preexisting neutralize which about the many transduces very significant based directly the intraparenchymal antibodies there. using transduction disease injections. brain remind Parkinson's at to It been dosed actually exposure. could into with cells. looks on relatively concern we're least avoids as get that the subjects neurons everybody immunity obviates vector the we're great capsid That for and also it have know to going be systemic AAVX We it safe is you before that

Huntington's will be de-risk reported. we exact It's of some of will Jim systemic that the exposure approach, Wilson again that about. the different, has be from feel way we same one administration different data sites that that program. and same So good capsid very of about but different that feel route kind

derivatives we Jim not looking other very lot there a Friedrichs blunt, exposure to that for see outside a are we kind the our of that are because but Wilson toxicity that studies. to capsid And neurons which be capsids also is which we in there we're has technically the want Now sensory done work have the excited heart get about. robust at there, we to do AAVX are hand, needs of DRG's systemic blood-brain-barrier that his and be same still on using which transduces the reported neuron, monkey

using is he different Now rhesus we're in using monkeys, Cynos. differences using monkeys, we're There transgene. the are

their expressing in We’re slight differences the capsid.

fourteenth used, time have vector but one yet not going fatality they're the all either is per a of monkeys. any not similar genomes ones direct the he doses we I head-to-head can say comparisons, in So our seen to kilogram very to up

later also and a year will peer article work in reviewed on presenting So in be ASGCT upcoming we reporting this at meeting that the May.

Brian Skorney

Thanks Great. guys.

Operator

Thank you.

comes with question next Benjamin James. Raymond from Our Reni

You may begin.

Reni Benjamin

meeting at the process more have and C waiting the taking the us more remind Thanks patients then Could on provide pivotal guys. in I level any looking Hey, plan interested a dosed good if can start Type as tasks? you with for little on for and cohorts? do Are and the been the I'm of enzyme kind of guess test. you at And function? relevant both comparability patients we're you exploring you color new questions. bit the morning those study, the

Bernard Ravina

question. for Thanks the

were good the levels make potency. the same course the same with then us clinically about really materials that's know in triple comfortable match we yes, programs, been and you at in of AVX very any AADC comparing that involve specs the was it kind identity release of flanking clinic right we the same Does AADC, a those of part So a and the and Criteria I The is terms characteristics. currently there of puts those submitted had our so clear is you used asking key with in straightforward of have what baculovirus as transgene, that to infectivity? does so to this is as you would how baculo have So material same agency the with them other aware we're that precedent understanding elements. and of and IND to discussions our course express the criteria in purity forward position that. the answers the and transfection In material move as clinics. of with well used those are on with think up

Steve Paul

years submission, new was many prep addiction many, activity. be and and nice a rat comparable in and complete showed to data tox but in to These that successful exposures the with achieve Yes, were we study was older the nonetheless the achieve primarily study, obviously and the toxicology compared did old enough that studies, that ago, enzyme we with but had comparability. were done for very IND prep and that produced

Bernard Ravina

measure add is one our I’ll expression of of point fluorodopa which our And is clinically scans. PET other

three trial in have at days the Phase year. PET that actually one fluorodopa around XX and X endpoint We then baseline primary scans

we'll make expression sure in to have that good look an getting early patients. So we're

Reni Benjamin

XXXX we seeing regarding as thinking something is the provide about early we in about follow thinking Got associated AbbVie us quite really or you and should this is timelines how collaboration, this could to something that’s beyond? just XXXX as maybe be the should and a with idea and up, an IND program be it it as be an can

Steve Paul

able Yes years then we'll two for be and it's initially one be select candidate. early. that It's after a clinical three or a hopefully research stage to discovery collaboration will

the but collaboration. though could Obviously a on will this area, have antibodies into lot us and vectors focused an for non-CNS to the neurodegenerative is in we're speaking and of terrific gets host about diseases. CNS in conceivably for reasons. perhaps, me science Alzheimer's a we are about initially excited be antibodies, expertise that neuro diseases with Tauopathies which a AbbVie company. excited which disorders, terrific we're disease number how this antibodies we also is Tau of Let monoclonal of They and believe broadly delivery modifying of and monocle AV a whole new underscore like area a us we group and several for

into to the brand So us we company. for it believe really does new a segue allow platform

Reni Benjamin

X Got you're enroll to And in while I’m it. either you’re the kind a asked the of sense waiting pivotal does sorry, other before makes the period, the - previous this ongoing with cohort would continue in given the had it about? perhaps to dosing question any or it study changes at Phase [ph] new I about of thinking

Bernard Ravina

different protocol. PET what posterior to So Phase that no, about can't enzyme that from delivery a we’ve from switch needed forward dosing start we under readily so do, we're we said and we’ll what the do material on and you learn really move we'll assurance study They're as learned We’ll within expression going X. scan have there. INDs, to

Steve Paul

and as X what we'll and we in to Cohort patients happens will and already dosed make adjustments very continue time XX be the dosage Yes, see follow over we’ve the it to interesting like. that

patients you So activity these - that the over to the to in benefits time. we're update periodically going on continue

Operator

you. Thank

with BTIG. from Our comes Leone next question Dane

You may begin.

Dane Leone

about an of point level and the more it in Two you make study, PK obviously to that view? the outcomes Hi, you outcomes how et one you endpoints you wanted I additional just on differences ask how level could and taking the the of think DBS the terms design then of - and from terms of efforts, Phase clinically efforts some the the but thank outcomes, can for procedure Starting cetera, give I X/X design in are analyses the methods? the you appreciate the me, to do versus on the your PK clinical on differentiation one with pre-specified of calibration study questions. thinking and compelling pipeline. of from

Steve Paul

thanks. Gotacha,

I really How your from on differentiate stimulation this think interest the is I good question a based deep understand of it brain and outcomes there. core do we reminder. of

our deep to because of indwelling patients in to were the enter studies, of stimulation didn't hardware. have candidates brain one them All and trials these most they want of chose two for

to going measures across terms seen similar function studies motor In in differentiating DBS. further, the are very what of be and anything our was

cognitive it of we side to important fact we those becomes is are endpoints, The a aspect deep brain function. think but not being more are have. stimulation clear of so and think question And may we there differences that there even important effects here magnitude that most across one

electrical And very brain the where specific that showing the it last clear, published it stimulation, sense became the stimulation paper electrodes deep electrodes the implantation to the deficits. caused because both very makes related are year of in going. a cognitive So of nice at end it's

communication those deficits believe kind of of will we attention. significant those DBS deficits So cognitive not are we have cognitive and in clinically and

other will we So measuring a be some endpoints very provide important cognitive and differentiation. might function that behavioral of point

Dane Leone

I how may. programs different the they is you you Do on INDs, want of terms whether on something in making IP these for decide be that it you on whether plate much IND we've clinical is on ask before royalty upon to between Great, or list and/or want your and data? or then I used? debate since some there you have I depending the to filed an the the getting IND that would hit process partnered may if applications, position some [ph] some decision owe might be decide the or programs after may thank And then two or a you an To these not potential of like pipeline the be one to in partnered ask as factoring someone whether of whether

Steve Paul

program the ARS kind the and technical this move the on. so success where an they Program of and At worldwide and SODX with refresh rights to Huntington's just silencing the in Friedreich's optimizing program the rubric opt-in agreement get of Well, provision under feasibility clinic, memories, get for into we have Sanofi-Genzyme of monkeys our once programs remain we everybody's we I think those probability will we forward. point, and Huntington's we're have good

excited patients very a Friedreich’s We’re we're medical case. and relatively model mouse certain There standpoint in it's kind [ph] of disease correction we that population Friedreich’s of silenced silencing very alone speaking. said that we're Friedreich’s optimistic in that And patients from major the that cautiously is XXXX, the are tissues, of of been about that again remarkable see as but given the I program which earlier in unmet large data in to and knockdown because XXXX, XXXX that ataxia toxin U.S. need, going this some a really of achieve of we've generated today has where phenotype.

success into forward data we're them moving the technical and So decision our be about excited of to based move going going the we what our them programs. to think our and We're clinic. will on we're base probability

Dane Leone

Thank you.

Operator

Thank you.

question next with Sumant Kulkarni comes Canaccord. from Our

may begin. You

Sumant Kulkarni

my taking for thanks questions. morning, Good

more of First R&D one as recruitment planned, dose a last piece because update during the there in the clarification. is just appears was your to one Is trial patient day? it relative posterior trajectory that the

Bernard Ravina

Yes, it enrolment. and was up wrapped as we're exactly planned

as enrolment, it procedure soon. they there take the do before and mentioned But we the off surgeons got into cut we learned one sites at will the So the we've experience have needed who X. enrolment, no, the more to screened we be having needed learn wrapped Phase patient is before up I what was key to who we've

Steve Paul

something really very mentioned so in patients recruited rapid last far Bernard year, earlier, we the trial seven half of that last And very, recruitment.

have nonetheless enthusiasm of our patients an great who I were to have a think on have deal DBS, treatment. would to part reflects the otherwise opportunity it of elected

potentially that, So for think excited we're about enrolment that we well trial. augers pivotal pretty the in

Sumant Kulkarni

the And patients program pursuing exclusivity of interactions those might given what you PD the advanced a use Sure. for of wider and mean PD, remind perhaps FDA set my might your interactions in on of you broadening number patients? what orphan the follow-up had have with drug about potential with could is us

Bernard Ravina

question, a It’s thank you. question, great yes. Great

mechanism a action both could but population we large and European have U.S. this several apply And Advanced Parkinson's to to they with of is Parkinson's disease, disease. more yes, and regulators agree designation. just So orphan not did this interactions around moderate

orphan case say not further and didn’t will to we designation. we this in pursue So that we’re happy

Sumant Kulkarni

Thanks.

Operator

for call closing Thank I’m I turn time. Steve Paul back you. the further at remarks. to no will this questions showing over

Steve Paul

to your questions. thank Well, look you the Thanks you We of morning so much. Okay. in attending all for call. thank you want to and and near for updating future. thoughtful progress We our on that concludes our forward this

Operator

participation. day. Ladies Thanks this and your conference. wonderful a gentlemen, Have concludes for today’s

Voyager Therapeutics (VYGR) 14 Mar 182017 Q4 Earnings call transcript