FOLD - Earnings call transcript Q4 2019 | Amicus Therapeutics, Inc.

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Operator

Good morning, ladies and gentlemen, and welcome to the Amicus Fourth Quarter and Full Year 2019 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, Ms. Investor of Director Faughnan, Relations. Andrew

You may begin.

Andrew Faughnan

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

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 two, 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. the statement by date or are no hereof. qualified speak All entirety revise statements in the or date undue call we presentation not after

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

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

John Crowley

progress where call. XXXX. start Good morning, tremendous on out today begin and In laid XXXX for here to poised we've who great conference throughout one and diseases. people world XXXX into through X. building we I'll everybody we our companies the around the made of are key several elements next Great. accomplishments now Amicus results and living highlight Amicus rare to in Thanks, with I'm and the biotechnology pleased that the impact at year to XXXX Slide January, us to XXXX full welcome bringing Andrew. ultimately early are of

of For Amicus significant like efforts. XXXX I'd and to execution of our take moment was few regulatory a another science, for period clinical, across growth just to a them. highlight commercial

revenue $XXX Galafold. revenue its remains our launch full in begin year a Galafold this top the is row cornerstone the year second million continues global strong for and success. our of the we've I'll end XXXX, With with of exceeded that guidance. in

towards and For trial disease this our PROPEL becoming we of disease. momentum replacement Pompe people believe what next-generation will our the with enzyme and AT-GAA, again completion the with therapy that's X significant the Pompe of crown for jewel care living portfolio, continues new approval standard Phase the potential of for to be and

financial resources review profitability. we second as our into our us well we on to business and of half Also the of last after to carefully path leading year, strategic a continue reiterate continued runway a our cash shepherd we in XXXX,

shared that collaboration of significant the we the to in our significant programs, Wilson among finally including XXXX we clinical in Jim And collection collaborations therapy, be XXXX advanced of University as our with gene well the field extension importantly, as we data and disease and program. very the Dr. of what and Batten in most CLNX important capabilities Pennsylvania, believe of in

that Wilson this growth the with engine gene Amicus. with Amicus UPenn. of for UPenn is Wilson future the and most Dr. partnership and research a significant collaborator Lab the us believe provides with tremendous therapy We

key our X Turning Slide now to priorities strategic XXXX. for

year this we priorities of We track and them. well five achieving key on are strategic all to have

First with Galafold, precision for Fabry. medicine our

continue Galafold Fabry existing year and a variance more with look of disease in in drive new revenue global medical In metrics $XXX we We living a to us to to million with patients far our thus further are this achieve to product will markets. strong off year. in this $XXX give to tracking key all and are confidence which for start guidance We XXXX million. geographies, in all key

are as are manufacturing Pompe X complete the increasing regulatory, our advance program, In the surrounding XXXX, clinical, Pompe toward we Second study. Phase and PROPEL we activities pre-commercial set approval. AT-GAA we to

also pediatric are filings. XXXX as the enroll BLA to manufacturing We a and to set advance MAA support studies, well as

plan a with AT-GAA a we in license program. breakthrough application support of year, to the full approval designation XXXX results initiate or this Fast the for to half rolling this full for therapy-designated clinical for biologics Track under and apply Importantly BLA first

Third, from Therapy our Gene of and new we are rare industry-leading advancing Center gene therapy in disease Research portfolio Global Philadelphia. our Excellence

our both program. We CLNX will the patents development, be and CLNX regulatory manufacturing advancing discussions and for clinical

underway as other Pompe manufacturing progressing we IND our we'll therapy, getting gene begin Pompe the IND disclose candidates the Fourth, additional clinical scale-up for candidates. to believe lot A of in our work and therapy are we well partners of to gene addition, is with up year. towards as IND two and studies in potential our us this XXXX, ready manufacture

continue expenses position, later the collaboration this our again we our we And additional manage a look we maintain to and as IND will our investments. sharing again, candidates carefully strong UPenn to And forward year. from financial

milestones. lead we is expected profitability. funded as into path a to well Our to And cash runway all fully XXXX, on we are continue us through major

in impact hand it coronavirus. on our essential been global supply final products on conscious raw our been before materials. our our of supply Bradley, has of products a emergence conscious a well chain moment no no of and export the There for of even On I over – or very to the Amicus import here chain any before has impact and or note

course. closely the will of situation We continue monitor to

has for we and China. WuXi and on Pompe, plan for at been of with partners hand. the WuXi's there track remain sales the on manufacturer respect no supply Biologics robust have Galafold AT-GAA impact mitigation risk in to adequate to in operations our a very year Specifically, place With

Importantly, we hand, entire center enough our X study the Phase beyond. Ireland distribution have PROPEL at on stored support to and supply in

So with Campbell call over President to and the me the to turn year. let now Chief Bradley introduction, that Galafold Officer our for performance the highlight Operating

Bradley Campbell

Great. Thanks, morning, John. Good everyone.

X more of of global me by color year the Galafold the a on of snapshot for Galafold with full launch. providing Let I'll begin continued growth on XXXX. Slide begin the

momentum $XXX.X including Japan, the countries countries $XX Galafold exceptionally our XXXX. well. early-launch as large as For XXXX, steady new U.S. was of million, total growth which mid-sized as over product metrics in full well countries an our and small- million and year driven in by strong revenue revenue increase and is in were

from Specifically, or remaining the $XXX.X and coming the about or XX% year $XX.X of XX% full the about of was U.S. breakdown last the million revenue over million generated year. revenue United States the within outside

touch all do I minute world have for reach want which to Colombia take Brazil, over We Taiwan, approvals global approved we've and with regulatory on Argentina, extra XX building. our been last Galafold; now to an that year. countries were including here commercial around the

incredibly the Galafold, the it is the world. support on have an foundation strong leverageable patients Pompe sales growth not continued working patients in but to of team our in with access important, the is with just lays expanding footprint to as incredibly launch highly our future to support products of to which well. XX passionate tirelessly bring an of this This global and more nearly commercial around have And year. experienced horizon medicines countries, those We we to

Turning slide now to eight, recap performance the over I'll XXXX.

see could on slide, John doubled XXXX As negative nearly the a foreign mentioned, X% right-hand of you including from that exchange. side even impact sales the

actually doubled well FX, sales X,XXX year, our If from not that than finishing over at performance you perspective, on more an patients look worldwide. operational with purely Galafold last including we

was see relatively On the and year. this this this growth seen again, you very the think can slide, it's performance, left-hand quarterly is years strong the QX. to pattern non-linear, I a few of consistently similar illustrates we've expect our And, see quarter-on-quarter over the we'd that, while again in side past phenomenon strong which

XXXX. of as three that outlined and confidently XXXX last XXXX annual about of we're from compound sales rate expect year, us, With and million, five-year million between $XXX to nine. way XX% to a we get behind period. to slide on a on performance I that That expect Now $XXX over to we we late opportunity And generate revenue goes years growth XXXX. And path to XXXX alone. long say, can OpEx a in in our cumulative that billion R&D funding $X towards

revenue also in in we the even significant confidence the opportunity to Fabry globally. market at for We further $X growth see as had Galafold, continue billion peak

in high-risk from surpassed the in initiatives, global also other continued which the well. mark fact, diagnosis and screening, by market Fabry In XXXX, $X.X as newborn all we're investing therapeutics diagnostic driven billion screening for

alone reminder, the And exclusivity which XXXXs. as we orphan late into give the Book our XXXXs, listed IP the take a Europe, have addition Orange us and that in the patents to end us to in coverage of U.S.

to of come. globally provide long Galafold to access opportunities a a lot to So for period

the metrics, compliance, naive revenue. continued million $XXX that XXXX switch deliver our me great Galafold in to And of gotten let and to including has on start global meantime, in just our and the off guidance reiterate, high metrics, of growth market and share commercial continued all track with all to a adherence $XXX million

to John John? our So turn updates me Pompe. discuss and for call to program back with that, AT-GAA let the

John Crowley

to potential believe ever living representing Great. disease, again, or is well Pompe care designation, Thank opportunity. momentum first disease standard second-generation the billion broad slide Bradley. for people you, for with with tremendous billion $X receive therapy a we peak therapy, Pompe There population AT-GAA next behind product I a the disease, as the $X a on what Pompe first-ever revenue begin breakthrough from XX be as of annual for BTD. may therapy will to of

MAA and Phase fast the to of study, enrolled the XXXX complete pediatric and filing. advanced a first to a apply to half We track and in approval a under XXXX set manufacturing plan are designation. support the is rolling BLA the full for BLA studies X our Pompe support initiate It to PROPEL

infantile-onset access those morning many to our we to to Two plan for new is this expanded children compassionate requests it year are: need. with with response this first, AT-GAA in disease, that Pompe an the updates received to program respect offer have for in use

partner or are as runs biologic strategic a nearing or completion CMC the chemistry, serve and with BLA manufacturing and key module, the PPQ foundation our successful for Biologics license our application, WuXi Second, will submission. for control,

all for derisking Importantly, this a and successfully program. PPQ major now a forward huge or at bioreactor WuXi. step we completed the the under upstream event process have production runs three This of is

that As data support, in study; approval together we additional a Phase the full AT-GAA. we've reminder, with PROPEL will the the study collected X/X of believe, pivotal

well as gene to therapy could our we AT-GAA, be about excited the believe the in to extremely be as preclinical disease largest with to build all what franchise solution continue and We the industry, program, potential Pompe patients to living Pompe valuable most offer globally. with

with to financial and So turn the over Daphne call I'd now that, like outlook. results, to guidance our review Daphne? to

Daphne Quimi

good everyone. Thank and morning, you, John,

begins with overview on the XX, statement year XXXX. full financial XX, Our slide December ending income our for

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

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

R&D the manufacturing, in in study and capabilities. pivotal the ongoing well We continue investments expansion Pompe of gene to and and therapy our scale-up as significant our with as commercial make portfolio program,

acquisition $XXX.X our expense by to of Galafold XXXX. During offset upfront that XXXX, increase for period. gene million year prior an launch, The the for million payment pipeline, Pompe in continued in is study $XXX.X primarily occurred R&D investments program therapy reported we million $XXX asset to as and in the an due clinical compared

Galafold million, $XXX.X to for ongoing compared represents and increase Total XXXX including the States. activities selling, prior the commercial the expense Japan general The was administrative the as launch $XXX.X launch, geographic and expanded United for scope year in million period. of

$X.XX loss $X.XX also contingent $XXX.X depreciation. development loss XXXX, share, operating share for non-GAAP per our expenses million in XXXX Net million of of as end of a and fair the prior share-based million our net result which define expense of operating and the advancing a compensation changes total per and was We programs. was SG&A was $XXX.X careful This lower non-GAAP year a guidance. or while expenses, period. $XXX expense, or On management, in all value to in were as excluding expense of consideration compared research as basis, at key

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

to now Turning XX. slide

into we our As fully John milestones growth. in XXXX are mentioned, through major global funded portfolio continued our and

our year course of all business identify that taking we review completed business areas of account to all and part normal strategic priorities cost organization, last our key of the have programs. to into we and savings, our programs new As while into drive efficiencies and integrated of investment top our advancing of a

We extend that our actions are the taking runway. following cash

gene and higher manufacturing development confidence to contract have partners, continue and Galafold will synergies efficiencies manufacturing. internal identified with We and teams trajectory. around a increased have support our revenue we growth therapy growth Through and

phased and therapy out and approach We our to prudent XXXX. grow, are to process expenditures. as external management collaborators beyond. portfolio leveraging build take capital internal And two plan growth XXXX We through INDs to starting to measured internal also a headcount for internal and other facilities, to we support one resources gene manufacturing in Amicus continue expect we

that to development again pipeline. the the programs relatively flat total infrastructure of we non-GAAP launch gene remain in in operating emphasize, our is expenses And other forward, pipeline. AT-GAA as global and costs the our for we Going products to multiple with place XXXX associated AT-GAA leverage already to expect transitioned therapy in in we commercial

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

operating Looking we to to $XXX guidance million guidance Galafold revenue year at expense reaffirming million million in XXXX, are non-GAAP of million. full $XXX to our of $XXX $XXX our addition

with on platform. to call back therapy And let our updates next-generation John that, gene me for turn over the

John Crowley

now portfolio orphan CLNX our for a provide slide drug disease for we Great. annual our industry-leading combined represent and Thank programs franchise, together CLNX robust you, to and disease $X CLNX Starting Batten peak in and designation I'll revenue. billion-plus now gene of here with XX, recurring diseases. have move Daphne. Batten U.S. therapies CLNX EU both disease, Batten that and franchise our rare highlight may with which received

Batten in In reported have devastating CLNX, meaningful interim of form this study impact clinical we gene that our in positive demonstrates data AAV of therapy our disease.

CLNX these study. follow-up and plan recently discussions X/X we participants, the advance And just this of clinical initiated forward initial have We this long-term regulatory to year, these to children the the for in Phase finalize regulatory path program.

for the results cohort The safely the have our initial clinical study important in CLNX addition where we patients neurodegeneration, read-through form now dosed cohort. in to CLNX common provide most in childhood higher-dose our Batten, initial of

regulatory this report later to paths finalize and discussions year year. Our advance plan CLNX to in regulatory clinical initial and and data this

combines driver University to an with growth important of On Pennsylvania slide the future. protein designed be novel Wilson gene remind expertise Lab, uptake, safety Penn's of cellular Amicus and collaboration in the which engineering develop to for the gene the transfer XX, technologies and the like I'd Lab with research dosing, This and Dr. Penn therapies glycobiology optimal Jim collaboration of with at manufacturability. will Amicus' Wilson you targeting, for

As this has with XX-plus in active rights part diseases, including of Amicus programs. eight collaboration preclinical currently to UPenn,

disease. is now enter XX, This to IND-enabling slide in on highlighted with clinic the into the studies, potential our is from Pompe Penn program collaboration most XXXX. in advanced As moving the

of to additional programs, data IND candidates the of this we by in guiding and the two additional out expect disclosure year. of collaboration We preclinical year end this are this to up multiple

to rare turn diseases. on again just the people Q&A, to I'd with like call So, living focus over before we

to their many suffering hope shareholders. deliver fighting amount are and diseases and on for patients, value for with to of these significant for an here And creating bring while our alleviate enormous people We rare are mission new for families. we our

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

Operator

Cowen. Instructions] [Operator Baral with Ritu question Your is from first

Ritu Baral

the question. left the I preclinical Thanks – And you're desired for for data-wise for set label? is data have complete BLA? much far everyone. what module? go? requirements that Or pretty to for rolling that about, to something any you Do study start ask your that the the before is outstanding morning, and Good preclinical required as taking Or to that approval? is for wanted is as pediatric aiming you

John Crowley

– get pediatric last children. into study To this that's you, children been to cohort, the the part dosing XX own the required is Ritu. that think initiative. To age thank now that We've last XX Yes. on That's question to that's not underway. our important approval, we the XX, of in drug pediatrics for

all below additional will the expanded We the we here infantile-onset way the expect that XX to initiate patients programs to shortly Pompe that age for add access now down disease.

we many for important approval, the to for get can. as important necessary we not So as the and it as quickly to patients think but label

the of of note for module. the be else terms begin preclinical In what's rolling nothing that needed to needed would BLA, there's

have WuXi three in for the we're major, completing good pleased undertaking successfully. course team now completed remind and downstream done bioreactor again, that dependent for that's on was to all a really successfully And of been work, successfully module there the runs major we runs. reiterate is activities all a and So all PPQ which and of CMC then position the team and our upstream really just those the

Ritu Baral

Thanks. Great.

John Crowley

welcome. Very

Operator

Your Anupam is JPMorgan. next question with from Rama

John Crowley

Anupam, if can't you're we talking hear you.

Anupam Rama

now? me hear you Can

John Crowley

can, Now yes. we

Anupam Rama

the so PROPEL? next XXXX? Thanks be last the So I've going or Phase but this the Pompe to much. is gotten update so Or X/X any trial to there in are in week question plans update Pompe

John Crowley

Yeah. I'll that. speak let to Jay

Jay Barth

good the out we're two-year very knows. completing have trial this We as course getting the results, we X/X out that on update which Phase soon now X Phase can. right focused But – plans as to and everyone now we Yeah. very the look as read point, of don't And At study.

John Crowley

just that on to But who continued before. discontinued. in remind was nobody continues. patients back Everybody to study, Nobody everybody it Yeah. gone – all not Lumizyme has

So patients reports anecdotally, having we continue to from on positive AT-GAA. a good hear experience

Jay's for two-year purposes for the and over the additional data the complete approval. then to BLA for completed next of year significant the collecting focused that extension now again, point, BLA study So encapsulating on we've and the full also

Operator

question with Leerink. is Dae SVB Ha next from Your Gon

Dae Gon Ha

on guys. I the one just and good question all me. aspects. My you to morning, can Hey, progress, hear AT-GAA on congrats the follow-up hope wanted

are investors I maybe you quarter your internal Jay, for respective currently that to on anticipating of data scenarios? And as for even John market that the Specifically, those are opportunity scenarios wanted get second take respective the the the Sanofi what from in or going neoGAA you modeling what of outcomes? are have the some have Thanks. some

John Crowley

I program. What own for been That – I of April or its comment with Again, AT-GAA. stand fall the XXXX. on are living is on can't in dosed patient people more first do now our to somebody on Pompe know than our else's we have will program XXX right. that, Yeah. has clinic There in data now

more in the the even difference clinical for by expect than major U.K. FDA the product studies the drugs the the this distinction to difference date in with breakthrough data. aside pediatric therapy difference year frankly designation designation The from one of in our PIM's the recognized course all that We of again the with themselves and

So really in AT-GAA I we'll – be distinct. think to we're very confident really,

of our patients both that for as patients including we're distinction X, study the study. in about up X PROPEL major well the of patients another a make range switch Phase XX% naive population PROPEL in as Again, studying Phase who

trouble the the with clinical of We about feel we of five PROPEL XX for again So We that our number than really waitlist good more no sites did really, are. we significant sites study. enrolling on continents had study. where a

have and a as stage. So we we we program robust can as this think as have at successful

Operator

next Your is Wainwright. from Debjit H.C. question Chattopadhyay with

Aaron Welch

morning. Good This progress. guys. Congratulations Debjit. for Aaron on Hi, is all on the

titer. poster the with world a Emax for walk increased on test noticed we So six-minute ADA the that

interpretation how And unexpected. this? that compare have you Do was for with any Lumizyme? does So this

right. All

Next question is…

John Crowley

on. Hold

one got We've question you. stop there. me Let

will We you take I'll that let Jay, one. answer that one.

Jay Barth

the but your that question, having the study without into no in World at -- don't if aspects antibodies was at X/X between something mean which I safety antibodies the know technical Yeah. relationship walk present there PK the for or of was we I everybody six-minute our efficacy test. including looked and getting the Phase appreciate,

our we're that's out. and based robust stand effect our from walk. efficacy very X/X showing X/X So And when for very data results results the Phase Phase on of six-minute reads Phase study X why confident in that a

Operator

Your next with Merle Cantor question is Ellie Fitzgerald. from

Ellie Merle

much thanks guys, so question. Hey the taking for

for Just distribution proportion of you in little therapy of disease, terms some patients program, you that and data. that a well competitor this think And genotypes And particular talk and suited about programs gene gene phenotypes your in gene therapy and most about Galafold? severity versus talk Thanks. therapy bit own for are in are as reporting the the can Fabry versus disease? as

John Crowley

entire going ask Hung I'm we level, will long very every Castelli infusion think therapy a think biodistribution the and a Great. Jeff who Thanks other the to those enzyme moment, standard though care. think burden there an minimal help very patients have week but the Ellie. the their with And with is maybe for patients be have only we to of to real of Galafold an IV high ERTs. very who We opportunity a the have Galafold. who a a variance of replacement satisfied time do at in are for limitations patients of lives comment increasingly

or development we is Fabry half gene a the path more potential. of It Fabry. For those ahead in represent which therapy think patients, complex do offers population, significantly

on combining the taking we're expertise gene in Hung with want engineering that therapy. Wilson the Wilson team's technical and science and to describe maybe So Hung our Lab's in protein approach with program we're another if Fabry? expertise That's Dr. collaborating UPenn that in you

to And the you let the in the opportunity So Hung path on development and then comment. add anything Jeff market. Go I'll ahead.

Hung Do

Sure. Thanks John.

Galafold. amenable least at me are And half there you the so not let know to who of say not is appropriate as population

to we a feel commitment treatment those we for an patients. have and So, develop obligation

mentioned, the the we therapy. knowledge the John and understanding utilizing actually and as years to are XX those particular actually our over lysosomal effective. are make diseases our so the past that these applying being We in regards characteristics enzymes experience to storage gene principles And actual enzymes

intend this And gene particular -- a we this much approach. that so with expertise better combination Penn's to with particular with And make for Dr. we and then targeting for better utilize delivery. therapy and in gene stability Wilson

And could gene so we develop effective to a therapy the be two think these combination of prove Fabry disease. to for novel quite

maybe Jeff anything else to add?

Jeff Castelli

No.

this much of based we I think I transgenes. a could the that constructs somewhat And the is of data looking different early we just could add that IND declared at now year. transgene, data. studies with expect this pre-clinically outcome that, ongoing we stabilized that of have right some have various And animal on this models, in potential candidate one programs targeted year

attractive have So really pretty not are excited probably think would patients we gene option therapy would But to and an apply most we're don't amenable really be that AAV and an patients. to John to mentioned it oral all Galafold as available.

John Crowley

for Great. Thanks now a disease. Fabry And medicine just experience add XX in medicines centers Fabry with we've the reach the got of approved. of too amount major all got significant in development treatment of for for experience disease course getting world guys. around that have disease to we significant a countries Fabry about I'll

taking in UPenn. Dr. to that very and unique addition think field for that we the the other we're approach advantage Wilson we scientific think over be that players with another us in So could significant

Operator

with Mohit from question is Your Citi. Bansal next

Keith Tapper

our is for on This this Keith Thanks the quarter and taking on the question, and progress Mohit. World. congrats for

ask a quick Just wanted question. to

XXXX second Thanks. the might You have XXXX? planning may you're this earlier, to be but in when available of not until AT-GAA on mentioned data file questions for half how

John Crowley

this and module. One the year then We in that then we submission would the the time of BLA track to PROPEL plan section. a fast things. use half for will be rolling the followed XXXX we first both for that of data expect application. by would the review would wide initiate where a data it's also half clinical a would with in pre-clinical first half BLA And we module, -- have the that the first XXXX is approval Again And CMC patient Two with designation our the to pediatric module, a the population full very there. from about complete adult. begin

Operator

next Whitney Guggenheim. is question Your from Ijem with

Whitney Ijem

taking for the thanks guys, question. Hey

if should regulators natural terms we it also steps? limiting So expect update the additional follow-up, there of in data the about history I on there before you are then but steps And CLNX, on engage the data. to next apologize I thinking and rate missed start what

John Crowley

Thanks Sure. Whitney.

Scientific our including in on and would XX activities second of necessary filing with have year long-term So division. Thermo for Lots everybody partner their half of been in to we expect other who've is this data therapy the the Brammer CLNX. gene follow-up that patients lot there BLA treated Fisher a the remind the be activities of around manufacturing. To

half well on and hyperstacks with a activities scale-up activities transfer of now side to in of a of for that able expect that the the be gene the underway all a articulate to BLA. BLA the second would CMC and nearly necessary GMP the And the lot probably required are We again in with tech therapy, submission. the complete manufacturing so we year what's manufacturer be for

Operator

Your Janney. is Yun next the question from with Zhong

Yun Zhong

Hi. question. the Thanks for taking

switch study So PROPEL is patient the and naive patient. enrolling both

switch naive patient Phase of cohort? you at combined the disclose both switch been more are ever or I cohort in patient individual Have study? naive patient instead more looked patient including able and to X/X you So enrolled have wonder,

John Crowley

XX% them everybody other XX% Jay switch Yun. care of I of To Thanks overenrolled are remind patients and total for were X PROPEL XXX from question? about Sure. patients AT-GAA Phase study that about it of treatment-naive. the standard And ERT enrolled -- a

Jay Barth

very they Yes. To but the patients your switch so patients. in said were patients Phase Phase both included the very for design It in basis in in And, PROPEL. therefore, naive towards saw study primary will weren't study for in study one naive the able are confident both we cohorts the switch analysis seeing question, along X results data naive study. favorable the X/X and switch that we're the that the results Phase had as study, that take separate be going in They were we naive. I patients. the X cohort PROPEL same that to and X/X in for and the populations second And those and the switch in both the Phase study combining and was

John Crowley

or one of one showed multiple significant pretty different switch have to AT-GAA. in -- very who been just nine patients I three think Jay -- And a of out look if our ambulatory think one XX five showed what Phase expect based eight at natural actually. -- six-minute a done remember five switch. on were at our improvements the decline patients the at the straightforward from in had improvements XX be out Again patients. on I that you'd I It's where year cohort Yeah. treatment-naive X was year. XX Again year at ERT on if Phase are it in history five saw nine out We walk studies the Those you improvements of one we cohort X/X.

stronger. make combine adding to look were that if the So naive it you would only

Operator

Sachs. Richter next is Goldman Your with question from Salveen

Andrea Tan

contribution a of wondering patients? still Hi, question. I'm dynamics, respect speak you American on plus? where little Andrea there Latin XXXX on in you're naive if factors expecting increasing with launch This could there you Salveen. Could for taking the Galafold to revenue about if comment in dynamics And if the -switch And that thanks stand my gating for then geographies? And any bit Thank the is there? you. have you for been proportion meaningful Galafold-naive

Jay Barth

questions. great Yes,

patients. other really switch between So may launch switch was first. dynamics of for on gave already their is, last patients first that's our target you were we track with as infusion and strategy naive XX% system the naive which And update XX% was They're The the in every recall, week. when which and full we switch getting we year on were patients. patients

the previously move target And we as patients patients. we within healthcare naive system. patients or to then so of that's And untreated along, lion's the the start share

markets equal Europe, States although naive starting like United Japan, well. see mature switch we seeing strong are in coming a example, uptake seeing patients and uptake like as of first, contribution and patients Galafold, more on you patients an the in predominant as to naive still if countries countries, an So at EUX look we're whereas switch there in our we're the

So to that's in we out from dynamic four or continue the play years that expect now. to three

to to penetrated would on market. We perhaps coming sweet more Galafold, see as into maximally naive start we've the expect patients

dynamic. sort of naive move And continues it's within our well. the that's strategy again, and well So switch to

We did approvals to key we markets. those From a to markets. patient see as in Brazil sales and in year expect would some get seen Argentina expect start to. in mentioned, we those last named some we would we've Latin begin and American commercial perspective, sales already And to

reminder seek a as still for though, as a to national way in review caveat go year will We we judicial takes patients about Brazil, in that all process, reimbursement, through. we're which the

Latin a be in to expected that's significant the begin XXXX so to American why we've said, market. And driver growth from we've

as far, foothold So continuing overall access to would the again, markets. XXXX, in those to to revenue. patients really a open we when those for see start XXXX, But meaningfully nice up contribute so

Operator

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

Aaron Welch

guys. Hi,

I year – not But revenue, this quick, Just U.S.? of is if there sure real much that the of And prepared $XXX ex remarks. million was versus XXXX. for U.S. in XXXX your missed fiscal how

John Crowley

Sure. it. We've we that. We'll reported repeat please? did – No problem. cover Daphne,

Daphne Quimi

sure. Yes,

the of XX% approximately the U.S. of that U.S. and remainder So portion XX% be revenue. ex is will

Bradley Campbell

as given base contribute. of stable. number kind ex countries grow it States relatively that We're of terms – in That United continuing will to haven't is to of guidance hold the the outside specific U.S. XX-XX U.S. probably versus We breakdown

to the continues U.S. course, well. launch Of

kind a the of year. around might in bit zone. right that pretty be to there this but So would It that's little move consistent from expect I

Operator

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

John Crowley

Great. Thank you you, listening. Thank operator. everybody for

strong really all Have start it Thanks XXXX. As metrics in to you on XXXX a a and great was day. a off good listening. for see we're

Amicus Therapeutics (FOLD) 2 Mar 202019 Q4 Earnings call transcript