Amicus Therapeutics (FOLD) 7 May 202020 Q1 Earnings call transcript

Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics First Quarter 2020 Financial Results Conference Call and Webcast. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session and instructions will follow at that time. [Operator Instructions] As a reminder, this conference call is being recorded.

I would now like to turn the conference over to your host, Mr. Investor of Director Faughnan, Relations. Andrew

You may begin.

Thank discuss Amicus morning. corporate you financial for to call highlights. conference our Therapeutics’ and joining results Good first XXXX quarter

Officer Executive John today’s Dr. Officer; Operating Hung Head Medical Therapy. Officer; are Quimi, and Also Science Chairman we Dr. on Officer. Daphne Campbell, and have Do, Financial Castelli, for Speaking and and Chief call, and Chief Chief Gene Dr. Officer; joining Officer; Chief Chief Jay Bradley President Chief Portfolio Crowley, of Jeff Q&A, Barth,

referenced may and As plans Private make statements within business, of on Reform XXXX our our Litigation as prospects. the of forward-looking as Slide X, well to relating we the Securities Act meaning

any of by plans or we to of achieved. or Any Our turn forward-looking call can the be be our affected inaccurate risks and as statements forward-looking not that be will all should made wrong uncertainties. and out regarded by may unknown assumptions on statements be known this or make us representation might by

and to to cautioned this to their the to undertake and forward-looking any which statements, forward-looking are reliance events cautionary on reflect obligation update circumstances conference place only You hereof. this statement by date or are no hereof. qualified speak All entirety revise statements in the or date undue call we presentation not after

forward-looking such later our for the you XXXX March the Commission. and Securities report Form risks of the the December Form XX-K to disclosure today on uncertainties impact XX-Q quarterly and the XX, and Factors full year Forward-Looking Exchange the XXXX to refer and ended with ended for statements be Statements them, filed Report may a XX, we quarter of on and For Annual section that Risk

call At it to Officer. Chief and my Executive this the to John? turn is Crowley, over John Chairman pleasure time,

everyone to quarter And Andrew. XXXX our conference call. Great. Thank first you, welcome results

and everyone are Amicus and safe. an is team I certainly their extraordinary begin for I Let it and time our that well this families. me by for stating and families our hope just know been has

and Amicus two and we’ve on Since on we our of first on all leadership of I are protecting moving objectives. workforce, our health and performance. Amicus has March The the themes, focused senior about workforce of protect well-being of and work a and be empathy empathy team Amicus from would to by while environment important announced and continuity to relentlessly back been XX range families that our core ensuring home the and key been safety, against also performance extension initiatives emphasizing support global focused and and business programs our have a

the as pleased we’ve background progress today’s quarter into memorable to first made call, year. highlighting quarter the second the and with on So now at quite be this significant I during Amicus that am

companies living poised to brought and build adapting we been the growth started this global the changes of to clinical and has as all science, While next and diseases. with one XXXX biotechnology has about great many continue a the around across of our by execution efforts rare of karmic to world’s commercial people pandemic world impact regulatory period

As highlight to like accomplishments. release, morning’s we this key I did in four press would

With continues to its remains launch expectations. cornerstone Galafold performance quarter continues the of in success. launch Galafold First, strong first the and our million revenue, exceed $XX.X

both the in across treatment-naive First global throughout from the including including those and new major by business all Covid-XX. patient switch starts hardest quarter hit patients revenue represents the performance quarter regions

impacts pandemic, to any monitor are continue but seeing. encouraged Of we we future course, will from trends the are by we this that

R&D timelines our Second, track. remain on

today, disease to top-line with a in XXXX. half half in that proceed the onset and us U.S. PROPEL the this Pompe study X disclosed BLA of data on AT-GAA late also We first Phase the expect FDA the to continue plans out of schedule read submission rolling We to year. rolling has to the in this our second start program BLA to remain authorized now at

Gene our our as most in as CLNX Batten programs forward to lead Within gene our and programs. CLNX, pipeline, move disease continued advanced Therapy therapy we well preclinical

Third, the quick Amicus and in our during Global planning early has maintaining this the work times. supply tremendous a Technical job just chain in operations advance team these contingency to Thanks pandemic. in place done extensive the

measures that been drugs true and to into patients remains have the to have taken able get that to needed further We we where future. ensure

be continuously all world. geographies all supply therapies in therapies patients to to expect able We to the around Amicus and potential

strength a the for Amicus. maintaining remains fourth, balance of core priority sheet our And

recent are We leads initiatives, series a second now the of our us XXXX. into cash well and a exercise, financial savings of runway announcing further today that half through cost

Our detail team Daphne continues as in shepherd carefully continue financial discuss profitability. in the more later our resources call. to this to on we will path the

key strategic our in priorities Galafold, we one; Turning well for These achieve XXXX. to of existing more to we $XXX look patients then Slide million to new to variants $XXX precision X, with disease for five Fabry Galafold to on are Fabry, to living will million year. this our continue achieve include, We drive and track markets. global amenable with medicine in product revenue

am manufacturing approval. surrounding the advance we I activities increasing Pompe clinical, preclinical Two, as pre-commercial our we regulatory sorry, and program this towards are –

our Therapy Disease new advancing our portfolio Therapy Excellence are Philadelphia. Gene of from we Center industry-leading stemming and Three, research in global Rare Gene

development programs. both this Batten discussions our advancing and We CLNX will and be CLNX regulatory for manufacturing clinical

Four, IND Therapy, year. as partners progressing with as well the our the in addition, scale-up potential manufacturing underway of manufacturing two candidates. lot our IND Gene other Pompe gene therapy Pompe our is work IND disclose for this and of will are towards A additional we to we candidates up

all with additional into our major through half And funded again, we And he again of well we manage investments maintain sharing carefully and XXXX. we remain our financial ahead, t IND second strong we milestones collaboration a now position look forward the this to later year. continue Penn from and expenses fully to candidates as cash

Galafold Officer introduction, timelines as So, further to that now Campbell, over and and with Chief go the President hand go highlight call ahead the Brad, ahead supply. our an R&D performance, Operating and please. to the Bradley our we

Thanks, sit is earnings colleagues. think and just our here let truly with hope your what everybody well John’s your me I as virtual I there statement, safe echo John. call, along families we first and and And out is

the performance I As we R&D chain. the milestones. in John of and also said, our for and continuity the provide detail through the actions I But more will have supply to detail more will on taken strength go Galafold timelines quarter,

through start Galafold of of But global a we’ll let which the XXXX for first me quarter and snapshot the commercial of growth on Galafold Slide continued goes progress. X, give the

increase For total of million, from the quarter was in is XXXX. a XX% which $XX.X quarter, first net the first product revenue

a been and has favorable by only has the some the been importantly, countries patient some reimbursement as adjusted had And contribution COVID-XX revenue to exceed Galafold adherence select ordering XX%. demand, continue strong as and virus. global have driven We’ve in of patterns healthcare dynamics impact as from adherence. well strong minimal to rates continued organizations compliance

later the million in or from to over during within the of breakdown now have generated countries million previous $XX.X with horizon quarter have the that sales United for time coming first on U.S. of and outside States regulatory expect XX% was commercial approvals in over have this seen the XX now in we or countries the similar and quarter. revenue Romania XX% of and around geographic over products with we quarters XX we patients world the to $XX.X have revenue remaining the in quarter coming and markets We we the reimbursed The do Colombia those this more year.

products this it patients lays which access just support the is an expanding Galafold, leverageable support as well. Pompe our but and anticipated it important, is In to launch incredibly footprints, strong global growth to highly to continued of of the with not foundation, future

also in But that has to geographies, seen have very the an now think for coming very times. great of be strong COVID Slide a could the strong X, after these the crisis year into momentum some our of rolled we Galafold through QX, XXXX. dynamics therapy even Turning phase was we the and part we’ve as finish Fabry oral unique COVID-XX in clearly to various pandemic a as unique that in had

seen report now setting for have clinical to the they’ve Galafold phenomenon a because show switch reluctant telemedicine, Galafold, form start or crisis. our that patients XX% of some also care. hospital rare example, from is released telemedicine experienced been in before some up on was We’ve a or patients a alternative seen actually For to prescribed which we’ve that disease in the go recently Galafold XX% of patients into highlighted EURYDICE which first

our even has so access hit all our led we will and provide they I And crisis think United new chain intact then we’ve that more finally, seen the to that, have remains in others. supply the Japan Galafold. uptake later, continued and many the Italy, States fully through starts Spain, France, detail hardest countries can confidence UK, And and patient of including all and customers

impact we some monitor continue the as So, think close which of dynamics tailwinds may global remains inevitably pandemic the even to full provide unknown. headwinds, pandemic cause may these as continue to will the the currently some

foreign quarter which mentioned numbers see exchange. a X% first does perspective XX%, can I you include a as from increased impact the From that sales, negative

to performance the perspective, from sales And quarter last same so, year. if compared actually you increased it look XX%, operational by at an

And do past. do here quarters the in we as relatively expect on we left-hand show seen but in the growth way past years again performance continue over several to we’ve and non-linear the quarter-to-quarter, side, quarterly consistent a

with us, XXXX. years a we path that say on $XXX on several So, now can sales of behind in that opportunity million X, are confidently Slide to performance we

that outlined in that revenue over which way by period investing in $XXX from the year course as a of high expect go to now. get of between in we is as well. sustained to previously through now newborn billion $X cumulative further diagnosis rate well R&D a will growth about as opportunity XXXX continued alone, initiatives, we peak that compound expect to Fabry towards driven market will And are without dollar OpEx risk screening, XXXX from annual funding are our have even significant billion and do five growth continue long we and to that continue diagnostic that XXXX to we to XXXX and screening, generate and in we XX% confidence other would revenue globally, see have which We and which I period million, the

end And coverage U.S. until we us in have late patents listed the orphan alone which give Europe IT addition that book and in us XXXXs to orange of to geographies, our take exclusivity the XXXX. and other

long access opportunity provide Galafold for So globally to to time come. and lots of to a period

ensure Galafold to this doing supply we to that want supply again the all focused but we’ve will before, chain Slide On has something do are continues on pandemic X, and maintained supply we for our the both that chain, highlight COVID-XX continuous that AT-GAA. things we reiterate during here isn’t

For of API already listing Galafold, part had products. multiple been to years hold our and drug inventory in of strategy

we to for around this issue level. to easier as the increasingly and and the molecule the don’t an us. severe, ship have pandemic with manufacturing is a made down successfully even will by country supply the COVID-XX is this now much long far became fact inventory possible be again, And stability And small world. as that pharmacy we to So as believe the the chain pushed easy

and ahead base of had to for of inventory move the similarly drug the to plan products UK. into AT-GAA, And launch already our our outside anticipated been China build

that So underway. that we had already and been –

chain, following similar of as have now we possible for COVID Galafold the now site-by-site and actions to on into far pandemic, pushed taking supply are as a and inventory the where impact the And basis delivery. spread coordinating we

manufacturing that would interruption inventory. WuXi, we ATBXXX ATBXXX has also single continue not and biologics, our AT-GAA and note partner to build their is had manufacturing we operations We here as the a for successfully commercial manufacture who to

have of our geographies our and the here uninterrupted summary, the a including with studies, with a the in AT-GAA sufficient And in of we do more inventory studies on all will be expect will all confidence clinical additionally, disease. continue complete patients Pompe of Fabry high ongoing completed degree In global the through our schedule. that Phase all of in tosupply now program for detail deal Pompe X And of PROPEL in moment. and that importantly, living GalafoldandAT-GAA continues for to we go I study

tremendous Pompe updates. first So first turning for now to Slide a go for R&D XX, living opportunity. which behind of $X to is remember some through people as the Pompe what is therapy broad AT-GAA billion with me Pompe ever receive our next therapy, represent does let billion momentum breakthrough a as designation. diseases, of care the disease to you we ever second-generation standard $X well disease, therapy the There believe population of maybe for global

are and study advanced mentioned PROPEL the are Pompe the MAA timelines X We track XXXX John as studies even on importantly, the on support the COVID crisis. and complete our the Phase pediatric in filings. by on And to to reiterating BLA timelines phase original PROPEL manufacturing are track, study of we enroll to

this we’ve everyone this really which administered study XX% created patients design to the the We’d study. incredible we we point, is and the where study parameters is confidence the protect investigators infusions. their little overenrolled our with team getting further are that have we’ll And XXXX our the continue a to of within importantly efforts up to thanks literally of as bit the ensure and along remind which patient-by-patient did clinical plan to that we’ve provides more well today, that infusions of expected color, of a integrity

approval the the our submission track for that fast rolling support in this of to which BLA half time, BLA the a the second are designation. US granted full has AT-GAA we year, initiate we of and track on Today, will FDA first rolling announced under

we’ve call March. requests use children on that Additionally, earlier with now particular in full received IOPD, program in year are disease the first for for that Infantile-onset many we compassionate expanded the the we access or initiated response to our getting discussed for we’ve Pompe in

we’ve now foundation CMC manufacturing from and for the all process section perspective, under successfully activities at which BLA And the three the will the for production including Bioreactor the campaigns related completed of downstream Runs purification serve the submission. the WuXi, as a PPQ

about this to And a significant for continue is be as importantly, preclinical We Pompe believe event the do the franchise potential our this major Pompe excited with well what we in a step as you AT-GAA, Therapy as could globally. forward be industry and derisking a of program the know, program. all extremely to largest with solutions living offer to build the most disease valuable to patients Gene

CLNX rare thanks Slide for teams, will here able lead been careful of and course, and time of maintain gene therapy Pompe across Now industry-leading therapies well gene our XX, diseases highlight in to portfolio Batten as activities say, as including we’ve as discovery disease, our I and the facilities our even our our science therapy on I Fabry efforts the gene critical program portfolio programs to CLNX and this and science earlier briefly again. well of would during

think And paths and starting to that X, meaningful AAV for year, our the this clinical Batten reported can this advance we’ve plan and a and disease. And as interim the positive X we extremely CLNX additional our data form with regulatory finalize discussions, do as clinical devastating do we we so our we in expect Phase previously from regulatory Batten study program. our therapy reminder, disease CLNX, demonstrate of gene study, data of in impact franchise

initial We in X second our regulatory a CLNX ongoing discussions for finalize X, is data the clinical half. well report plans from and in also disease, our CLNX common of study Phase path year read-through additional form provide advance as the know the that believe important as and data to neurodegeneration study which most childhood that to as regulatory is Batten you clinical

That of its and to medical US recognition designation enforce expediting CLNX agency fast for has therapy in development, us review drug the fast of granted greater importantly, the designation And access by significant been potential the purposes approval. address track track program potential our gene need, unmet to FDA.

important let great of all me Slide XX, for with research the which growth to on things part of of on are come. finally, remind University that you just decades an going then for of will the Pennsylvania, as And collaboration the driver Amicus be

Penn’s experience, manufacturability. to uptake world-class with and optimal and collaboration therapies dosing, along This for Glycobiology cellular technologies safety develop Lab with designed novel targeting Engineering Amicus Protein at Penn the Wilson gene gene transfer combines

that part in diseases preclinical have As currently to we rights programs. eight of active collaboration, including are plus XX this

Also Annual I’ll is American Therapy Meeting virtually held being of announced note be at presentations that Cell at week. Amicus which next presented to Society XXrd Gene and several we the their

are in of end. programs therapy one posters studies guide there included the in conference. candidates and to multiple and But data to we we to that sponsored and way by a continuing you additional would we year presentation this take have look of oral additional provided at Our the disclosure new once two preclinical gene do we’ve encourage a of year data virtual two expect some XX our that through

with And outlook. guidance and our financial Daphne, to me financial who now results, the call over can through that, go turn let our our Daphne?

you, Thank Great. everyone. morning, and Bradley, good

ending with begins overview on financial statement income for our March the XXXX. slide XX, Our XX, quarter

revenue first a currency currency XX% quarter of includes This negative of rates the is the at XX%, growth, constant year-over-year Galafold XXXX. impact For quarter, exchange offset of which measured operational increase we achieved revenue first X%. over $XX.X million, of by

lower sold Cost first as goods of XX.X% was percent continues the sales a in Cost to of we as royalties. as revenue not quarter, States, the other percentage year a of compared to prior as well where countries owe as favorable XX.X% sold the as royalties, period. we grow net Galafold of United goods do in are for was revenue subject where to

XXXX. to $XXX.X Total basis, On million for XXXX. $XX.X of first expenses the the XXXX, million compared the first were non-GAAP of quarter a of were million as operating million for to for quarter $XXX $XXX.X first quarter first as compared expenses XXXX, the operating quarter total in

and to our tech Pompe programs programs stage of advance and clinical to in for initiation the transfer manufacturers early and and primarily the development research in within program to within investment gene due therapy our research our contract the studies. enroll clinical increase The was cost

development in measures previously were the selling, general, the investment as Our reduction and impacts announced, does of that research and implementation the expenses. cost decrease in administrative includes of the

expense selling, to The expense operating fair changes and in excluding depreciation. value non-GAAP consideration and of general is contingent expense, compensation non-GAAP research development quarterly and We administrative XXXX. decline define throughout expected as expenses, and share-based

first $X.XX loss net to or million quarter or $X.XX for million for as year per the prior a Net compared $XX.X loss $XXX.X period. of the share share, per was

outstanding. we had shares XXXX, of XX, As approximately XXX million March

now Turning mentioned, and major XXXX funded our are global the now well of to second through into XX, in fully slide half as John milestones we growth. continued portfolio

further We drive have to to and savings, able been cash extend continuing efficiencies, expense by cost our management. runway careful

in AT-GAA On is and the our expenses flat costs and products Slide as discipline, AT-GAA therapy that relatively we We XX, in for expect development the place again we remain already to emphasize, associated pipeline pipeline. maintain other with from non-GAAP gene the operating XXXX multiple transitioned going commercial meeting in forward, to infrastructure objectives. total our XXXX launch while of to financial programs our in leverage

high moving continue we Therapy priority Fabry CLNX, Wilson are in programs therapy on programs fully and especially, research Gene To to ahead gene with and Jim schedule, reiterate, patents. CLNX, and Pompe the all work support

million cash December guidance. compared about A XX, current XXXX, and Cash, securities XX, were $XXX cash million position equivalents $XXX financial few XXXX. at at to our marketable comments XXXX March and

of are million non-GAAP million. operating We to $XXX $XXX revenue our guidance full and to year our $XXX $XXX guidance reaffirming million expense million Galafold of

remarks. that, turn back over with John the closing let And to me call

and Bradley. both you, Thank Great. Daphne

about global can you challenges the we brought relentless see, have been despite of as across all focused things and on pandemic. So, unprecedented performance the by the business

from confident to stronger. passionate will even am this us world will of team global continue who emerge have and great led entrepreneurs as a have crisis, this through that the Amicus emerges lead I and We

with questions. So, take are operator, to we happy any that,

the of [Operator Instructions] JPMorgan. Anupam comes Your from question first Rama with line

guys. for of I which to question the cash half the just Hey on question guidance second is XXXX. all the Thanks on the taking a have and congrats progress. now quick runway

be in wondering is for the the about thinking some forward reopening we to what globally terms a on what expense the should of reason whatever assume? of and U.S. that us Thanks. second pandemic? infection the due so of is levers are sort of what And there assumes if again the Just pandemic side COVID-XX and longer in wave push than

Sure.

going to we’ve and well-being is, So, what mode telepresence communicated through their have to globally able that, been to put this upon this on first our we are effectively. safety really Anup, all Thankfully, we operate health, and of employees foremost.

to put risk the greatest to rush and people to back would business the risk. So frankly be at

that. June do to our would going We’ve the not there be be reintegration would are that any employees we earliest back. to said So that

looking all the undergoing are carefully evaluating we around We in. all the world a local that, in project at the now geographies guidelines work

is flexible would work an I be that it what and will what a be – any over to of will the were emphasize a before employees integration So, our will to return time. we staggered and be from cadence

are or time reinvent we’ve cubed R and RX savings best cost to the including this asked Frankly, launch best on the internal other calling to employees an this of all practices, also rediscover, side take we efficiencies reimagine what taken to Amicus initiative the really the like our efficiencies. of looks I have where

of So, right financial earlier and of looking looking our half measuring runway expenditures. organization that we all XXXX. multi-week takes our to at capital now, a across the our expenses, is as stage extend further a this functional programs second of entire a preclinical account, the operating of at Again, reprioritization exercise the into

budget. very this hiring frankly, the certain the savings the Conferences, is not period. part we’ve there entertainment hiring Obviously, key – from additional very we’ve expenses, of limited to travel, evidence as had crisis itself,

that to of to taken quarters couple runway half us that us ahead. well a bridge which to second puts XXXX, of the extend very all now together of So, into the profitability by allow close

the crisis negative would hopefully. And don’t but routine, to standpoint place expense expect an any question, go that were have our well. to back Anupam? shelter and had from of kind we as that that reemerge we answer the bearing to Did Okay, in if

line the Fitzgerald. Cantor Your of Merle Ellie with question comes next from

taking the for questions. much so Thanks guys. Hey

are gene in the on sort us the how any to and process can scale-up seeing and on using for color Batten and raw you on your through more co-edge. Just expectation therapy, for going scale-up Thanks. manufacturing of materials of latest terms sort you just dosing due in patients of – is terms more just impacts supply commercial for front or manufacturing scale give the the the

going Sure. through. that. handle Amicus let dog like about Brad always the to excited It seems I am as your leads is are

Apologies for that.

no, No, of all It’s from part that’s working of fine home. course.

Bradley, let that question obviously, please as in. Jeff well, I’ll and chime So, you take

manufacturing No, in our gene well as Sure. asked with Yes. therapy continues to go you question. very your

the process are MCH being We that we CLNX transfer has platform, underway activities the and are they to tech of well manufacturer experience well our that and for a of Brammer manufactured CLNX hyper is be which and who are in going how middle the with will was the programs this and that Brammer so there. with with platform is lot stack very

and on that in The or be will using I who for commercial from will that that’s platform commercial dosed should intention next material the the track Brammer be is CLNX patients say XXXX.

In terms of our question therapy. around purchased time materials, for of cetera likewise into for supply raw chain manufacturing secured gene supply therapy, we suite had for study gene chain time of ahead materials inputs plasmas, and and et the all your

therapy interruptions anticipate the had in far no have supply and gene interruptions we so, don’t or any chain. And we manufacturing so

Great. Bradley. Thank you,

from of Whitney Your next the question Guggenheim. Ijem line comes with

trends, are of tailwind to in for guess, oral. the follow-up and far comments hesitant being you to on Good the Thanks question. go of patients the around seeing so Galafold around of that some morning healthcare that light the Galafold, COVID guys. of I kind centers taking into Wanted a sort being for

patients or leading that can there things talk trend virtually you you tailwind if like, that to is website about like tracking, you see are are continuing with a the sort kind of terms potential that on switch curious, of or guess, anything going I for that of guys forward? any indicators in that or guys engaging

coming Whitney. have the to I’ll trends ask crisis. emphasize we a Thanks, moment, strong just but in comment Brad before again, into XXXX really Great. COVID-XX to

great in this patient So was driven by performance QX fundamental demand.

medicine at of it’s the play to the benefit, taken this yet out home oral think of is in in COVID-XX particularly of getting geographies. an further that be seen of medicine, and course, that Again, back that settings, characteristics now natures fact crisis we a can the and in a we’ve number number that one

but driver it more factor. a we not Brad want about and maybe color add you if going the Though, to ahead? the performance in great think forward key of how this months

Yes.

I well think that’s John. said

really for you think, well with physicians on best in it strengths I have that our the and disease know, forward. important going treatment think look, we Fabry an as for and foundation establishing mutations success the we mean, in Galafold experienced that I with option treatment been way patients patients, us amenable option sets and now up – the this and strength

them outside patients. ahead ability can’t It’s setting interesting, some with interaction of up interact around virtually. and all it’s we have physicians the with a job very close U.S., all time of physicians, have through being but dynamics around done really have to course, product, question do of the specific with your relationships those To we direct good

both and from we patients. and how the of just may an I might moved call really, option hear to phone that’s meetings new for their face-to-face meet replacement pursue some to text to physicians patients of getting who patients’ being mean, switch some I And that’s or we as of have. It’s video chat encouraging of able or these anecdotes instead of highlighted by option to oral for in them reasons oral want the and again the been therapy needs moving have all

the starts hardest to hit had countries. even have in new We’ve patient we’ve – continued

think incremental it said, So we John again, working that system that tailwinds is us exciting. which well. as And is some provides

virtual and that which where have actually in seems with is meetings, U.S., patient to. a are that, can to patients compliance is we’ve have seen environment the exciting had What you form more more regulatory really directly. patients be a interaction In we actually drawn an that

to we are explore that. look to continue So to going

specifically Galafold where do physicians U.S., the option. switch in or again, on that patients of start we our oral some examples patients and an it’s And with know because with wanted to of

to we So, important again, them. trends continue watch the will

the great We continue far, pandemic how so watch again will to momentum out, in but QX. plays

sir. you Thank

Your Ritu Cowen. the of Baral comes from with question line next

guys. Hi might walk of that data competition Thanks AT-GAA you upcoming the its if up. the SEC Genzyme hits and would How patient potential position question. six about data Pompe landscape potential want for minute you taking to population? superior that How within I naïve superiority would the ask coming to compare commercial and sort generate?

just Pompe the touch COVID, so to are relationships team organization situation? thinking are and great usually Amicus. launches each How her respiratory patient are with the within drug a you such population of of high thinking in And about How commercializing patient risk about got this Jayne understanding and you’ve you incredibly drug diseases. a with orphan

Ritu. Thanks,

I launch the of other, going is this in to particularly like data. think, but strength Pompe driven the any be by

that just So study already ago. be will got AT-GAA remind we’ve years important. the incredibly lot accumulated. PROPEL patient data a of We’ve with treated four the first was to over Again, everybody,

switching profound scientific to moment, conferences people to we’ve where we And multiple dataset have were unique very ERT treatment-naïve we’ve impacts whether were here. from going am whether a that review or at they spoken the all I they remind everybody, AT-GAA to not it the for seen at on standard-of-care. At

going a it’s we speak success we and while in drugs we let the think of ultimately, data to So there medicine development. unique data to have have clinical for and We think are very other are itself the the the launch. will we great, that clinical developed drive have but

We PROPEL are positioned, study. importantly the well with

also think key this around with dozens which we AT-GAA around help hopefully opinion leader the have will as launch in every world, we to able getting of We countries are medicine sites the multiple now world. is really experience

to not show, what going not am I the to of other on may launch AT-GAA data. will So program rest the speculate the or as strength AT-GAA of of the any may success ultimately,

line question next Schwartz comes with Joseph SVB the Leerink. Your of from

given to expect And you the you of are wondering Great. detect that impact are if I Thanks slower the seeing using? versus biggest later to year in data disease where much. extent endpoints you just talk history report this could are expect in versus so subtype, sensitive natural activity CLNX was this you CLNX? what the do CLNX progression in how of the about

end be Thanks. to you patients dose were further comment will treated limited about made a color. a I of the Castelli, this the dataset low of Joe. to most Gene Again, Therapy, our of are treated to with it at being approval. the hope Jeff, little and CLNX. well. data think just add will the Sure, to patients who Head begin with commercial am with be that basis Jeff a preliminary material be of going free high in the Brammer handful I on program. CLNX any feel But, will XXXX the ask we Again,

still than in of is Sure. year this Thanks, rapidly. most John. data later CLNX, year of for patients will the John does posting noted, progressive quite we it the That CLNX we be for data although first slower those CLNX have enrolled. have handful progress patients. transfer that would of one

be focusing language, of measures we’ll that endpoints perspective scale, Batten various the aspects motor cognition, rating on efficacy function, vision. unified is which disease an main from The

diversified very scale a with lots of it’s components. So,

it safety So, with data. look but in case will lots just importantly, an And data of then, things as early one year update at, on an the look it at well. will to be this be

Bansal next of line Mohit Citigroup. question Your the with comes from

experienced advanced subsequent some Thank taking which morning. – could about coverage for of area the you one of year staying everyone good and very quarters? Galafold and especially in out question and anything for orals Thanks some is in have our my talked COVID. bit is your that second Hope Great. question a of My keep regarding quarter – little there. by for – safe companies need to you potentially in like you. mind Have of purchasing, light so

Thanks, the driven reported I’ll and that? add Great. fundamental more – was that channel. work and demand rhythm to from we see that if natural Yes, Very, the minimal any in Mohit. results, maybe kind to and want of the by sales. X$X.X you million very any the today Bradley could Daphne, we advance specifics impacts of

I careful think it intro highlight well I my in John, to said Yes. and you that. comments was

dynamics we is well, around the to foremost, fewer reimbursement as great. demand and programs which United continue States. in and favorable have world it was first strong see some did So, bridge clients patient which We the strong

well. really went reauthorization So,

So that’s patient again demand. underlying all

few and a contribution patterns. here was very minimal who and some contribution. that were here ordered ordering from there said, there there John but more, countries and As hospitals There bit few a a little was minor

the reimbursement patient driver and demand those that lead of dynamics of mentioned. I that momentum, fundamental The the quarter some was

you. Thank

of line with question comes from the of next Bank America. Tazeen Ahmad Your

Pompe maybe just through for Can thinking questions. Brad. you Thanks my One morning. us you assume. range should of that how are the about markets, about we Good specifics taking for pricing walk

that’s You the around been a another long when time. have having of for do the market, drug benefit on

you the then of So, of just question. again, could I – have thoughts hoping pricing. about big a maybe learnings And in force observations of how need? some follow-up sales terms of you I share your a And on Thanks. am might

over Yes. just Galafold for that we premise here. start must that the our same priced broadly fundamental of fairly apply they a Thanks, Tazeen. that we be and that as all did it we I’ll accessible we’ll to I’ll Brad. turn with medicines apply be again, roll – and principle for

how once thinking that, you you with a talk Brad, hopefully to AT-GAA ensuring launch? about path want And patients broadly for about it’s access if to are on for

on great reimbursements, allowed very Tazeen the through in us both the a and rather quickly us focus to the focus John, and world. a for a Thanks, served patients throughout I Sure. well. launch payers but of Galafold go thanks, very we and physicians, really think I lot to a proposition Europe think, Yes, access value I cascade that pricing, question. for providing and on course, Yes, premium learned than We think the from also,

And learnings here. so, apply those we’ll similar

is some that disease payers don’t again is high there is disease with cost serious worth that Pompe, kind sense like in a in rare think convince do to I disease, advantage here a that, case the Galafold, Fabry relatively of have in we Pompe to second mover being of the drug. reimbursing for a

value and down confident saw that on mentioned the our in show physicians have to come feel better is very to ability proposition will we that earlier, based that Here as X. we And of alternative. the but data, Phase we than patients to payers in the and course, data demonstrate John our

there we perspective, successful an we very From are – too, think with sales infrastructure force. our very efficient rare disease

Pompe Of Fabry – the course, has the drug well is as now. alternative right Sanofi as

kind think out field team so, We And the us a smaller their team in that’s the we and well. of team. there served we ratio very our to understand have of

we so organization, only commercialize our to And we able be are drug. will little this with confident additions really that commercial launch very global and successfully to

the I we’ll think launch other Galafold with is Europe. with U.S., early think about largest that of One if the UK the we the how where we patients lot at launch, more is well launch a hope have we’ll the and you to of in one important be were time versus which and thing on launching AT-GAA markets be AT-GAA as as

number much patients to so, than we a And earlier the we’ll we launch cascade access trial with clinical an higher Galafold. of opportunity think did have that in

the to those all and forward seeing for well feel prepared reasons, we eagerly think, I data. So, looking

Debjit question Chattopadhyay next comes of line Wainwright. the H.C. with from Your

question. thanks congrats Hey, on execution the taking and the good morning and for

on the into understand your XX year million unemployment insurance, million provided mix I disruption of is guide? print built payer to since wondering million XX% your U.S. $XXX and is is the $XXX how current this But we the are Thank given what the market full and morning, unemployment you. So, much base. revenue employer of all dependent

and Great. I’ll Brad the on Thanks Debjit. ask mix payer that. about comment to everything

good a question. Yes, it’s

great private afford a government where well have as patients as a payers payers we the drug. drug, frankly government can’t So perspective actually to But we had had both access we lower free insurers, from to far, we’ve anticipated. the access mix exposure and than provide programs for

And of is, be go things process had a so, payer. we’ve the we the have one and a patient said never by ultimately through rejected

the Galafold that far access, disruptions – So we we even good haven’t access and of to feel in getting to been patient patients so are unemployment. some very due seen have any

the a portion have for you to of remember course, that, big very is small us, Of population. Fabry a it’s U.S. but population

kinds The exposure those feel so, large in from of And far minimal. U.S. the so us numbers unemployment been for that perspective. has population quite Fabry a

sit And guidance, say, so remains I our where same here. the would we

are guidance. in We and very feel the reiterating confident

accordingly. about to But and course, updates things we those should monitor Of very continue right change, now, we’ll provide we are. feel we will good where

line the Mike [Indiscernible. from comes of question next Your

well. for as congrats strong question. quarter Thanks guys. the Hey taking the And on

XX% there on you are question study. PROPEL Just of well a COVID-XX, seems you progressing things like a schedule. occurring for on quick are It fairly the seeing infusions despite

Just Thanks. taking terms some study collection are or curious potential the in there are help issues if data to you are mitigate expecting steps of that any for risk? you that

plan and Yes. were infusions areas the drug the indicated, No. to we two-fold. happen have site-by-site are and a the Bradley And implemented as we thanks, able really appropriate are have secondly that assessments. One ensure Mike, is custom windows. access within to patient-by-patient, patients that of focus

course assessments of months at XX then assessments do as but we everybody, remind To well. baseline, and X, the also X, X

those to make some have to that’s to go some study. integrity In that extraordinary are and confidence of getting pretty we’ve lengths of are had assessments administered. patients we cases, continue the part in we assessments to So, those ensure that that the sure the

But the that’s of of the part an maintaining important study. integrity

So, we ensure there to in the we that continue put data feel plans with And good place collect are well. as the think would to able contingency . I about where appropriately we all we’ve are that

thankfully, would very continue So we’ll of any we’ve follow not, to the study. closely. the something seen impact it’s interruption that integrity But

Thank you.

Richter line of comes the Salveen with question Goldman Sachs. next from Your

for of Galafold, context is with tracking and Brad. this Maybe on for on the markets? between Thanks This for taking questions. the new mix question Salveen. your one switch could metrics perhaps With Andrea and to expectations patient established updated And how our you naïve provide respect is within patients? slip

Yes. Happy to take that. Good question.

most kind in So, track. calls that are we have of continuing highlighting the of through there on dynamics our

switch about versus so, right are naïve. two-thirds, we XX:XX, And one-thirds, now,

So is we portion the is naïve growing, would anticipate. what slowly which

in coming healthcare that most are get readily logical In the that the population ones in are other so, are is the switch that again, the that’s ones they available system because dynamic are every stronger markets, of infusion see week we system. to in they kind uptake and into initial an the and the the is

year switches as therapy. on the say really growth patients come more even that, we typically naïve this market. time, will mature the XX:XX And over naïves we and then, more more and like we’ll I towards our tip markets, start than to switch the driver markets see is Europe, more and between existing penetrate naïve some in in

grow the over again, was will that market time. And anticipated really that

in to market so, do And growth we we in are that disease. we timeline Fabry year will expect five three treated significant expect the the and driving in

switch seeing So we’ve those highlighted I we new in naïve are continue on proportions quarters. patient and and the both call, in starts trends that as along seen the previous

you. Thank

line You of Baral have with Cowen. from the Ritu follow-up question a

at Hey just just far the in when question, COVID back population launching population. Pompe as guys. I with into commercialization diseases general an or my risk as respiratory at will wanted be get of risk

got data orphan virtual Just assuming how approval drug is your thinking you a - is are about XXXX. launch in

in Ritu. Pompe, Again, it think I’ll Sure, next-generation I given then the ahead. launch that any some a for Brad extraordinary demand has on ask the comment wouldn’t I add color. to impact will and product

together not Of entirely late But on if largely treatment study looking a hopefully that not PROPEL in are the a perhaps just the by use best early be mind, crisis passed launched. in launch at I infantile we study COVID are better XXXX with risk that or therapy. is course, Patients really as options. with the would we have then. XXXX again, and the will available greatest seeking reference The do

– clinical even extremely they potential access great the to are next-generation maintain lengths a and are the therapy. would patients key across approvals, again, would with to We’ve as data that next them just and these greater further to lengths opinion just greater, the go I think leaders integrity So, to study seen of have to physician, The a go world. motivated. ensure scientists

do more Bradley, about even of or a global any bit So, a we about are AT-GAA? add maybe how color you want thinking to launch then

Yes. John. Thanks,

few a there. more comments Just

I I keep ability earlier our keep one well I demand their said getting and infusions. would intact things think mentioned was highlight, of in the is, and that which couple patients the to and other great of one A terms at that time quite which for supply of about we and program launch AT-GAA world centers around we have the AT-GAA development more than the dozens is with a countries number have will patients even Galafold. the of on and thing chain

of think who Galafold patients you will rate kind and uptake many the with about AT-GAA, So of geographies of more more the time if many on what versus also we a at launch, opportunities which on this are about many more think gives looking other pandemic. maybe I sites, say, start. have with AT-GAA of we be at side good would think a of I to the you whole host

to It’s gotten very up likely we’ve that interactions in virtual go world. as will the this adoption operating of all used acceptance and

of that. in are patients expect we’ll and that previous We as as well, we more have continue the seems very, very one at to virtual our heard and example and strong meetings patient look so be to may you doing an U.S. will questions, the in physicians

But direct with interactions. our see time we different how really with you We interacting would use process. this customers that We be augment in through going are to physicians - would think are never can a to and would to value-add substitute ways. that that this

our and physicians make medical to is case have. answering they questions closely answering our didn’t And that another that work I chain they here, this team affairs our are very sure patients I that the part important supply have, mention are will it who team globally the with questions before, but of management

experience which the built strong normal Galafold any the or setting, has help we’ve really us this again, a strong kind that Galafold or through whether it it’s normal of support - new very with been will And function that that is whatever launch. in up

of Pompe. will will launch continue that us with well think and we serve We the

a from Your Wainwright. with H.C. is next Debjit question follow-up

again. Thank back you and for Hey. holding

in a whether a circumvent liver about directed et do some in of it’s gene update, thinking with expression or cetera. cetera? therapy, terms it’s patients, regard How have therapy directed if Thank gene the, regarding should we also, little directed And you. that? et the IND, be liver you not we muscle information So, to Pompe how – auto negative

course, Thanks, ask week. Great. data Yes, about of I have next more am broadly are to technology approach to FDA going at here. the comment we Debjit. coming out taking And Hung that Conference

Hung, So, to any want field other you and if that in you if color. have Jeff, chime

Sure.

a also transduction will gene I at think described just other which use detail – for not some least our muscle a type tissues therapy in of well. of Pompe distribute as mostly allows but – broad vector, and that we liver, tropic that have program

don’t vector, – seems transduce. any we that know to was that at well again and not know a that so, – vector is we problem to and that And we designed we don’t have indicate a be Nupen quite particular to it group this know autopathy transduce data this for will his by really

that deliver help well that is pretty to basically to this studies want to we’ve done know virus – animal the you point. gene able to all to the the up on We that? and able that gene and anymore do get comment transfer it’s Jeff, of the

Sure, Hung. Thanks.

liver in transduce The durability. really tabs will promoters only reemphasize we approach that a taking other is like have is the that our transduction might in are will think tissues to good It’s thing that, I that liver. that’s muscle better with the I’d just addition we transduce answer.

that help might that protein tolerization any the by also expect a actual cell. come of GAA have for to – some from negative produced case immunogenicity might would liver the we instance the – So incident to in the

John turn remarks. this CEO conference closing for to you. Crowley, like I time, would Chairman and back Thank the At to Mr.

the Great. well Thanks day. Thanks, operator. hope families I in everybody all your That call. was stays a for Have healthy. conference great a great questions. and terrific

have this Ladies does Thank concludes today’s you conference. a great and day. and gentlemen,