Amicus Therapeutics (FOLD) 5 Aug 222022 Q2 Earnings call transcript

Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics Second Quarter 2022 Financial Results Conference Call and Webcast. [Operator Instructions].

As a reminder, this call is being recorded. I would now like to turn the conference over to your host, Mr. Andrew Faughnan, Executive Director of Investor Relations. ahead. go Please

Bradley Good Chief call Cole. financial you joining Castelli, Financial Officer. we for and Officer; Development Chief Thank Quimi, Chief Officer; Daphne discuss morning. and Amicus conference Chief have call, today's to highlights. Sebastien XXXX President Therapeutics our and corporate Executive Martel, you, Officer; Leading Thank Campbell, Business second results Jeff quarter Dr.

Goldman. Joining for Dr. Q&A Mitchell is

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

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

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

over President XX-K Executive Factors discussion Risk pleasure to the For Form it on my this and the section At XX-Q them, uncertainties our that Brad? be call Exchange time, of XXXX, we and annual to filed later for year such may and Securities of Officer. a to and the today and with XX, the our ended full on Campbell, impact forward-looking quarterly June the is refer Chief statements Bradley forward-looking turn risks Form Commission. our statements and report you report to

our to results Thank call. quarter everyone, XXXX Andrew, welcome, and conference Great. second you,

leading John to about him in Has just after Amicus those Amicus with been years Executive and position a commitment role to assumed Before today. I'm after Chairman such XX of to honored alongside the you quarter, to great business continue XX the start Chairman years year that two Crowley. John CEO role now an ahead. into has great term, fundamentals planned lie he founding leadership has has CEO, which like through of diseases living expected build President excited on. Board. for Non-Executive and the we and to the results of momentum officially to It of working I'd a the the of his is positive a the honor thank of assume as our that and strong for a been with of Amicus all very dive as is Amicus it's those rare opportunities unwavering deep and

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cornerstone performance and strong of continues Galafold our First, remains its the success.

year. headwinds with be pleased that significant globally, currency despite foreign of We Galafold the industry the uptake to very spaced this continue our

all represented meeting year growth quarter, key an the or indicators operationally, of growth revenue XX% or are call. further second Martel, basis. XX% later in Officer, geographies, in half we operational Chief exceeding guidance will at our performance exchange In our our the constant first detail objectives on reported the growth and Business in our key provide revenue are rates. Sebastien The reiterating

patents, patents and We that with generic issuance our excited matter this long-term IP challenges of year which composition There four. believe importantly, importantly the for continues providing our well any of are patent strengthened into of provide Galafold United announce patents. drug this new we beyond, broad to States, XX exclusivities to to related to have We to now alone, through newly quarter. now to property well address orange intellectual in intellectual listed the XX are the grow also book see Galafold novel three our rights this include estate and XXXXs This XX property And XXXX orphan we medicine. protection potential bringing the issued to precision estate the total are positioned along we issued late portfolio, patents. and

ahead, Looking Galafold IP growth year, has decade. continue believe strength runway with the to the protection, continued a in this next expect it long we for and well into

reviews to at Clearly, dialogue biologic along and to FDA progress with filings to inspection, and inspection fully WuXi we're facility our on China. with on WuXi the Pompe best are an pleased colleagues facility We agency component progressing for our how of the to are engagement with remain are from on at the eyes continue global we welcome for make all support active Biologics inspectors in the in the the therapy WuXi of AT-GAA. ready for ATBXXX, disease. The agencies. and regulatory regulatory next-generation U.S. novel EU very FDA very both we AT-GAA, Second, our level regulatory well,

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Europe, engaged applications marketing Importantly, now in authorization actively and EMA AT-GAA. the for with we're the

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place studies there United States, Japan a believe Pompe more XXX and or in world. Germany growing in programs, of access Additional which of with than programs X% our United more of access on patients number ongoing all the and treated total represents Across the Kingdom, are each. the clinical we participating are patients patients in today, now AT-GAA around

global investments personnel the inventory and move Our in additional the ahead, prelaunch significant in investments activities, targeted including preparations. continue launch launch, to support to launch plans

option United months ahead. treatment and in continue with both now another regulatory Pompe are and XXXX, a importantly, remain potential committed maintained the of for applications global Amicus strong we Europe expansion We as profitability launch And execute disease, global States prepare to the on Pompe position to financial third, Galafold achieving for further in of AT-GAA. to and the has patients for the in having closer -- with living planned

are We points pivotal Amicus. rapidly approaching two inflection for

new and securing patients achieving disease ensuring now more globally; Galafold emphasize First, truly geographies achieving Slide enter as the for we believe the of initiating States, rare leveraging in to steadfast prepared a of disease for focused I'll anticipate anticipated X, we way feats key drug areas global profitability. an for Amicus we're advancing will of financial a with many three commercial XX% experience that in second, $XXX our the across successful are see our our double-digit rare regulatory launch possible; commitment United this medicine all million of sharply to rates. global in as this This We These the biotechnology to continuing of revenue in in echelon of companies. for enable revenue drive are well AT-GAA; constant Fabry launch launching advance our approvals globally. million strategic medicine $XXX reflects look including launch the with and seasoned many strong focused to on with behind fully continuing are key on growth AT-GAA very We team On filings global XX% to AT-GAA second to objectives: that with to towards Galafold AT-GAA upper and demand and remain amenable product we rare priorities and Amicus. variance needed achieve and year, launch, strength living we to as to AT-GAA. and existing effective the exchange and people momentum markets. at precision

we to Officer, are carefully hand performance. investments highlight that best-in-class Galafold who call maintain over a and we can And With Sebastien me Sebastien? manage continue Business let the We our genetic overview, again, position and expenses Chief will our medicines financial capabilities. strategically now as the to Martel, advancing going strong further next-generation our forward.

XX% impact $XX X, timing we details The revenue patient revenue Galafold providing offset you within the of Bradley, markets. performance the start of outside total roughly XX% product $XX morning foreign the our by consisted our and This or ex-U.S. good accruals, as On reached breakdown certain partly driven the line by or the remaining Slide the by both the million, see with XXXX, everyone XX-XX in to for is of U.S. parts the from and currency in generated million second as of U.S., the $XX.X you can for quarter split to orders Thank business. we on as million expect quarter. you, continue call. quarter well more geographic I'll that of the of with revenue on of as strong grow coming during

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reported year. results the incredibly revenue constant Galafold The of currencies the first markets foreign over an resilient impact X.X% with highlight from commercial at XXXX. first and as patients Our exchange growth to the XXXX rate a the the the global of negative of XX.X% efforts. all continues year The half rates. the in first major was be growth business added first strength in half half was half of of result, of in our XX.X% And in operational

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report fastest global the patient that our the greatest growth been the has globally be to to positive last product continues to Galafold over I'm growing XX show contributor months. signals. to and market Fabry net disease continue monthly So pleased trends for Fabry

patients the global for U.S. if Galafold month in underlying month. on business year. the So XXXX. the the the we higher at months, is PRFs versus highest which on you of In measure also higher growth growth net patients example, this April and period And since three June, in same of the trend greater the XX new the a QX our XX% number net is the delivered truest is last which than Galafold itself at end patients look see we than of six of than year perhaps basis, in on performance,

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we've and still there's achieving diagnosed treated been diagnosed segments as So we approved market to to newly and the opportunity patients to while into countries continue grow continue the plenty longest, the the we're to penetrate Galafold switch segments. to where shares higher over of market and in

Galafold. on XX%, All impact to rates growth experience generally of by We impressive on reiterating some patients to adherence we who of belief and expect continue underpinned led is stay of a that Galafold of pattern to the those go did due our compliance that and nonlinear exceed that the quarter. the third in the order beginning handful quarterly being orders timing this recognized in quarter, continue. to orders This second

have record successfully Galafold outside be the strong of as very and the value reimbursement negotiating we Importantly, payers, recognized of U.S. continues track to renegotiating of by a

remains Our access anyone needs to it. for Galafold ensuring relentless on focus who

so all our track growth far XX% smaller of growth achieve most of that XX% to seen revenue to Next, track our XX.X% on full on very Slide What X. middle at We're we've is guidance well and half year Galafold is this in well of constant major seeing the CER we're exchange range. that of markets growth at continues first our as uptake as right to And markets. year rate. of across

this great as altogether, view half first So a we year. of the

into Slide three key surpass XX. to name that expanding broadening $XXX share progress existing that new potential to we're annual continued labels. just a years this through geographies; over expanding markets. revenue the in first, into third, new million on Galafold I'm few to Moving marketing markets; continuing authorization second, has in penetrate few expect Turkey know to we into and the examples, pleased the And growth We making to next drivers: year.

be support the a Fabry will discussions. screening, by diagnostic And we're In continue through XX the in in late well. invest in newborn and XXXX, XXXXs, to initiatives, longer-term, of coverage Hong We patents that now in provide U.S. including driven includes three which Europe, see and new growth overall reimbursement to in and finally, the XX screening the measures, book second quarter we patents. which matter variety of into orphan continue to globally. patients Kong. listed market pricing filed composition have And other This we and that give we Chile, diagnosing as in in significant through protection our high-risk IP in us exclusivity the of orange and have addition

on us shift preparations with globally product long our for launch. to to I'll to So update for a we're for successful you come. opportunity and launch access that gives AT-GAA, poised here provide Slide Galafold XX, an provide as On let's another gears, time

the a unlike when That from markets. around will XX Galafold, time in of infrastructure the were So TLs. AT-GAA handful presence we a including needed, major countries at for launched take new managers, only we launch will the the have over largely world, that team and the we building and commercial be hiring now scratch, FTEs example, with same all

areas all access, with We and and organization. confident for experience to have addition, launch, experience maybe commercial importantly, drug a regulatory from across physicians. with most in the relationships that reimbursement We're chain, our key needed world-class successful commercial, supply in are payers, and

highly disease relationships deliver AT-GAA the their Pompe published world. people regarded Lancet We experience the and III the Journal. will leverage education and to in medical Neurology team, living the have around From Phase with data

Biologics, successful Jeff Development for me strategic doing with Dr. position a hand the our access and around second partners launch let the to very with pipeline in of experience now over our we're world, Our the our we Chief Jeff? and all reimbursement Officer, we're highlight Amicus. With [product again, update. planning WuXi Castelli, that, believe a to together at call that strong AT-GAA

Thank you, Sebastien, and good morning, everyone.

benefit the crucial. disease disease prevalent disease most We'll long-term for Slide multiple range start is people natural recognized with treatment, our that XX by of of a options Slide measures program. a a challenges Pompe in is Phase health of followed key III one decline poses Moving fatal primary XX. many AT-GAA secondary history now our Pompe initial the by that endpoints for affected the often seen on continued neuromuscular We highlight disorders. study. of and from and having being to the individuals. of studies severe and summary disease therapeutic and We've on publications key lysosomal presents

patients function, of alfa needs. of alglucosidase experience represents including disease addressing outcomes included Against and groups the Pompe. with recognized and PROPEL endpoints vital the greatest study this pulmonary across patient potential believe Endpoints key assessing on with patient-reported consistency in alglucosidase illustrates naive over function, of capacity, this Pompe of with Pompe, forced most unmet and a in We two AT-GAA of the and one patients key and had, therapy, across only safety muscle that trial adult strength, AT-GAA approved is clinical to-date which late-onset LOPD. motor minute controlled the impact AT-GAA treatment alfa. distance ERT population. randomized slide, in reminder, for the a PROPEL overall biomarkers, or participants six was the walk effect the the manifestations As favored double-blind the disease efficacy living here shown ERT-experienced

we population, care clinically most on average, before never of the disease actually the phase experience in meaningful which standard distance X.X associated for decline is saw this Additionally, which patients. minute population. where And generally an years increase for walk in in a ERT more the both with participants in on the seen six statistical were and showed and achieved of than FVC, nominal stabilization treatment, versus current outcome superiority XX

at & of study Adding I/II year, the body on Phase Clinical XXXX the earlier data AT-GAA team Slide AT-GAA. data overall the presented this Moving to long-term behind Amicus positive the XX. Conference from to Scientific of MDA

very persistent disease these shared as to and Comparing key reviews. in disease. represent become been biomarkers great seen in outcomes, living on results what patients, with known durable have three the of reductions the is potential give These functional new and updates of untreated results after natural and XX, the ERT-experienced AT-GAA As of improvements key authorities U.S. hope AT-GAA ongoing as both these the for global On substrate about program. the on latest with has history Pompe data with continue here regulatory slide, durable as indeed care damage their Slide people results of standard the years. the EU to that these muscle highlighted meaningful and we've well in part Pompe

extended the review accepted alfa the of two progress. for August BLA the the cipaglucosidase PDUFA If of First, to miglustat, agency the action Last October year, BLA. dates XX on regulatory NDA XX for from XXXX NDA in the the components the for FDA U.S. and of the may, for AT-GAA.

the in WuXi will is on WuXi we the Agency action required the expanded announced on the in including others. positive multiple expect -- opinion access the in Japan the positive we This framework an the extensive two in scheme this recognizes We shared date, Medicines prior and to XX that permits eligible has have note, U.K. been medicine filings at review. least under submitted indicated opinion and mentioned, received not by and third, EAMS to Europe. This scientific MHRA. October LOPD, manufacturing the later switch community AT-GAA, EMA expected CHMP by facility. be U.K., France, in faced the in the years Pompe an FDA that through MAA AT-GAA for adults has AT-GAA together place, approval medical manufacturing As granting We've have to now for conduct is also and inspections granted we year. which Of programs Germany, marketing authorization approved to access based dependent to unmet by ability includes these alfa need again inspection The is U.S., early living with for to was now facility, access the European a the U.K.'s high at inspection writing that the who the prior opinion alglucosidase the Bradley of

In able Bradley Brad access granted a two first patients growth as their all provide are Health AT-GAA Agency and France, receiving program, access this ATU those the as Medicines the for mechanism now pleased to of known With the access noted, program. centers National who nominative access under seeing and U.K. reimbursed physicians compassionate now component in with that also our access clinical novel being now has more continue XXX the Safety Products the with momentum having programs, mentioned, the across opinion expanded are we than requested worldwide be we're the leading and our to and are AT-GAA that to we're report expect pleased positive studies patients grow. extension treatment. the under AT-GAA As to to EAMS dozens to our eligible access Pompe programs, AT-GAA and across U.K. for treated we interest formerly this Since enthusiasm in significant for to grown, multiple scientific and in

in study expand with into we years LOPD For year. younger the and up Pompe the open-label to later IOPD, age, living this disease to infantile-onset enroll to Pompe expect patients ongoing children XX community, or continue of with

we in Importantly, receive requests that treatment amenable and in program increase. Slide continue Fabry, the point living an with on This the agreement response reviewing of pharmacokinetic we compounds the impact chaperones programs with number LOPD and research Of expanded would science be note, children On Seville. mutations living year to operating to explore are Financial to potency, expect an Daphne by for both just that, to to cost year Driven guidance. our for needs financial including results, new review the for XX, to will not and to with Officer, collaboration people access very will the addressable at fiscal we dosing. small fiscal and and of early-stage will pharmacological not based over announced Fabry disease attributes, our we guidance With through and academic continue modest, our IOPD, like the do several expense today University agreement many innovative operating Fabry I call with Chief molecules next-generation turn for outlook. search our Daphne? now activity collaboration both our properties. of Quimi, optimal this

and everyone. good Jeff, you, morning, Thank

on begins overview foreign currency our overview revenue with quarter an second of exchange of XX Slide and financial Our impacts rates. performance

Galafold growth a quarter, period same million, $XX.X second negative we achieved operational which offset the of This of by currency is of X.X% in over a XX.X%, the XXXX. increase revenue X.X%. impact For revenue includes

year-to-date goods the FX as XXXX the statement outstanding. year XX.X% majority XXXX, year per to the U.S., $XX as the basis, we June and year, the for our approximately to XX%, of activities. was a would and research end reprioritization ex-U.S. a assets, lower excluding define of had and non-GAAP total basis, for reported We our expect in of respectively. depreciation. as operating by currency XXXX on the expenses, our expenses in euro, be as Given the to rates and changes the quarter portfolio. $X.XX as per pound FX revenue On quarter have The development to pre-launch AT-GAA the XX, yen as by compared Japanese to of gene at outside second prior were currencies X%, Cost $XXX second and related non-GAAP sales reported expenses exposure sales in exposed ending a consideration compensation net period. million expenses expenses be year second of period. share the sales the is income continued and $X.XX of full XX, impact foreign a July the quarter Galafold these average significant operating declined million contingent the driven the are fair and Galafold of Applying expense $XXX British therapy SG&A XXXX. shares compared or share-based the June generated compared on clinical guidance rates, XX% of $XX.X At million. million $XXX.X studies million the XX.X% Galafold million non-GAAP million million, to the negative are of range our in most see to. second of or And sold in of $XX.X global loss share launch to exchange revenue numbers. prior impairment GAAP approximately XXXX for were operating Net value operating to on $XX.X we This quarter of loss second net XXX investment The was increase of expense, compared loss in of or million quarter quarter XXXX. Total the a the Slide total XX second non-recurring for in included year $XXX.X outlines $XXX.X million XXXX. percentage X% we impact

previously, support gene non-recurring As the as AT-GAA the guidance for portfolio. therapy we an related as costs to million global includes the manufacture also this well highlighted $XX commitments launch to of approximate for certain

would decline we Importantly, operating XXXX. in expense beyond, levels non-GAAP saw in XXXX levels below and we expect to

Slide to XX. Turning now

of a financial XXXX from achieve We continue strength on to non-GAAP release. position press and in remain profitability operate to as our track defined in

A majority our We driving in on global of globally, as Pompe and deliver launching and well will our by disease of on marketable efficiencies, expense the disease XX, few value approvals at growth about cost guidance. Cash, financial $XXX.X securities the savings franchises million compared and and as management. Fabry June to at December and to core securing XXXX driving were AT-GAA Galafold, focus XX, continuing XXXX, investments cash careful $XXX.X cash comments our XXXX. position equivalents million

Galafold exchange Our is foreign $XXX guidance million currency constant full to $XXX year revenue rates. million at

In addition lower million. $XXX that, the our to operating $XXX remarks. of me for of expense million end back guidance with Bradley let the to closing to non-GAAP turn And call over

over operator, Daphne, to continue all questions. Thank now for up Sebastien, the we Andrew, who Great. turn Jeff, our with tirelessly you, around world employees thanks to call to for the open that, with work and of people living can rare diseases. And

Instructions]. And JPMorgan. first Anupam our Rama come from will with today [Operator question

on a hedged around opening but more manufacturing or little CEO Brad, knew a couple sending WuXi cautious you of I've congrats it's coming, that inspection. the your perhaps to sounded the congrats bit gotten again, Brad, We Man. on all role. the inbounds comments

is we that And get confidence what in timely level a here approval inspection the your So of the is of near-term? sort completed AT-GAA. of

I appreciate the congratulations.

So straightforward with yes, we look, very wanted just are we with where be to WuXi.

the to be the welcome collaboratively extremely our in. prepared to inspectors. working WuXi dialogue with are in We're partners continue and active and at inspectors with very We

is at conducted. products PDUFA plenty But delay. confident I then state we confident that remain We both that is ahead what on then be to that obvious PDUFA we an as we now, call, doesn't right that opportunity for date. time there can and we same to XX for just think wanted that said the have still be October if date, time hit the would approved guess fairly I of an inspection happen of the October

next will question Ritu Cowen. come And Baral with our from

you side if day XXX the been frame see see Brad. point. would for also to long and with label you the as get flip a Congrats, CHMP the market and to how Glad path And helping the the to going or Pompe the what address I It's time how discussions can at have I Europe, you as I in will ask labeling you is this questions it? FDA seat. ask expectations for think coming. the broadest How EMAs to you guys that the possible -- down wanted see of help how market about appropriately that? likely answer broadest label? What -- you address the you

agencies. yes, on thing reviews highly I our we confident will a I We'll is are label start with confident, are that pleased we And based the with we the scenarios we we're are out same think FDA. think So the another are have course, of really commercialization with the allow would separate, in in us highly that each. we important successful approval, both be with to are and although And you where are, where will of most play AT-GAA.

the to successful. study. of walk forced those well-controlled have as minute stabilization highly in the the I think reviewed thing are seen a in we we a have around data particular, statistically we've most label well are a significant -- to is before compelling Those think population, think important to we product, what improvements showing that, the that six be within data important is focused very vital in most based nominal differentiated reiterate in never been data that continued on switch we're Jeff have as on that capacity. And those

able with we long commercial we're as and AT-GAA. for that commercial where the need with the to the we're confident data we'll as market line as discussion in those long think have very today, I we in are address be As

there's roughly X,XXX to remember today. patients it's on I important that AT-GAA think

And unmet in disease. majority Pompe that vast need represents of the so the

are, have so to very we're we where patients. based successful support And on label we'll confident, those a

in a I think there. discussion. think out I Europe, again, it's play same scenarios could separate the And

and that the that again, I have confident next there we're sure progress looking in of launching conclude label. is and early reflects, think eager emphasis the data terms of year to those the forward key the that approvals to a very end year. and same we potential outcome, making at We're in successful this there

And ahead. our will question next -- come go

say, just was clarify. me I to going just let

on patients I X,XXX on said of X,XXX standard time, think AT-GAA, it's care. I last patients

And Leerink. our SVB with question Joseph come from will next Schwartz

patient wondering And marketplace, was And identifiable there these more patterns any hopefully expanded is if the to-date the when think characteristics access and or AT-GAA. to can likely the little that that it's talk of bit unifying that AT-GAA if over carry segments in a approved? any you are -- here adoption about you to-date? are for might I experience do

access excited France. program the access AT-GAA first all, of compassionate have first So to in the for through really reimbursed

just And that's access and think programs. are really more bellwether buckets I to of broad for a milestone come. exciting a a would I hopefully important those there say few

it population a before unmet patients our be important who of it label because way that think going studies our need provides physicians primarily AT-GAA. -- from in of biggest therapy the is II it's course, that's trial it's III. AT-GAA. to years who opportunity just before that And into to unaffected clinical and came we patients required eligible them ongoing in I the promotional on are, part patients as that for is Phase program Of access accessed who reflects access not kind I Pompe think for a for think, to, completely U.K. being And to a select really to Jeff mentioned, program, Phase out for Amicus, in an patients two on The allows laid being And are disease. EAMS cohort and the of

I preference then of think we what patients than of as example dozen see of and strong other seeing, patients a is And outcomes variety of see now do, who market course, we pediatric that utilization, today looking opportunity think, more that can some the well in think, represents as entering for either better some well infantile-onset, future. later as are also great the in in patients and a for and are a really markets. a onset program, who I the vast I strong majority

with time. I we're much looking that can continuing serve very demonstrates markets those and able just remains to we forward the to there to think over that out AT-GAA, unmet hope provide need that be the and

come And Ellie next Merle UBS. with our will from question

I be be review the quick confident give in Galafold. a could needs in the PDUFA, And on one terms would to that. U.S. inspection. like ahead color the it cycle long any then would occur when manufacturing of the just long it typically And how by? ahead on of that you on completed just guess be follow-up that inspection guess, it schedule? could I to expect scheduled I guess PDUFA? scheduling and take a you you by October to on Just where the XX, the for how of Just

of You expect in trend you of this broader highest on continue? a mentioned patients the the you just new that around U.S. something June, seasonality? this that you to Or that Is sort Galafold. is expect in net number saw

let there prepared direct hybrid, United one any a are States, recently to and support options a inspection inspection, think supported utilize the there. FDA additional Sebastien There just be different a inspection. virtual Galafold paper-based color inspection. provide of variety the WuXi know paper-based, can could and WuXi those. from of we've be before But we're the Look, authority. from take support there could folks that regulatory there starting I'll inspections We on an the I to a could question from be said actually

inspected they We hosted also unverified know on who that the Commerce U.S. just list. Department,

able inspectors So able are we get in spectrums conducted. know are be and to to

U.S. inspection. to fully but timing, In support just I for wanted and to to poised Sebastien, think approval, We're we're the forward get that the to in that to that don't need are we does question too I point. at want be Ellie's Galafold? go date for approval we order on if the seeing dynamics PDUFA what broadly terms confident Galafold there, just that of but maybe acknowledge in an want to to prepared prior we're address clear you happen that, cute

Absolutely.

you related to the answer level late. Clearly, be, of the When more was has interaction of that This of compare up is what degree would picking we've seen. been there U.S. in we with really. some HCPs think in And not my seen asked I our significantly. seasonality whether we've activity that some increase last increase we in an in also having we're countries, Galafold. ten-fold beginning in versus an face-to-face almost year had impact. of beginning of We're -- seeing prescribers in seen That's we've a element of number this is, the we've that second see continue the to of -- U.S. interaction. year,

So unique increase. of Galafold prescribers continue to

prescribing have we've here the in trend we to last with seems And U.S. continue the over July. play Galafold. seen physicians the more few So to months that

in good situation USA. in the we're So

Dae from Stifel. Ha with next question come our And will Gon

profitability on the financial question progress. XXXX footnote for the from Maybe the 'XX? non-GAAP it press sensitivity does is release where OpEx to Congrats than lower talk the predictability. is on non-GAAP all speak and me projections A saw around I Daphne, the on being about of the and how variability guidance. XXXX kind you can just your the of

Just around bit wanted to sensitivity. get little color a that

Yes, do that? address you Daphne, want to

Yes. Sure.

past and product. So I sheet the coming of flow of will AT-GAA to that do move especially some through -- think sold. goods Once expenses approved, have few we to balance income and over cost that we it years, R&D the comment the that statement the spending fact really from the we've space, for the the some related specifically manufacture is over is the have in experienced

is a piece that large of that longer be there. So there no will OpEx

that is expenses the reprioritization also look there are gene the -- some or future, I we as piece, out largest portfolio. would non-recurring say, this the the in nature There therapy when So that's that into related commentary. of year that's driving to are

So this that expenses will have in that not the we occur year future. are those

coming So two items it's those where Is really related predominantly. that is comment from. to

question Richter Sachs. with come Salveen our will next Goldman And from

on program mentioned? guys also chaperones is next-generation then the update give on Salveen. you for This us therapy the Matt and an Could you just please Fabry gene

maybe it I'll and over kick Yes, you. then to start, I'll Jeff,

reminder, So both think, Pompe us. and a approaches in for as and very still continue gene those have, therapy, Fabry we are to we programs differentiated important

move to color. maybe, We And But an there. still to the commitment -- and program will exciting But developing little a the to to I new give some I therapy we continue I clinic. next-generation for in at technologies be or you while to are it's can Fabry of it with be a think those chaperone, Amicus' early, opportunity reprioritization Jeff, say I way the think for think for reflection I our think more anything answer looking that's questions as of can there broader measured it's therapies the very gene forefront those into forward, continue a put Pompe. bit us given relates the targeted forward we're idea, of but before

data, of that more improve the collaboration, molecule mutations chaperone. with addition patients what if a that, nothing small excited to add And then the amenable just Fabry a we can related Fabry now, they we benefit benefit to on. of very provide some therapy Brad. on in as chaperone. we're early Thanks, updates number And very see there commented see trying Bradley to we could But discovery Galafold. we come. bring It's to believe will will if Yes. that really present additional already that. a stage, gene is have to we to enter what we But really we to to is very that patients right conferences. expand if meaningful it's way see for an profile strong can for chaperone at as But Fabry And to there is

question Kluska And come will Fitzgerald. our Kristen from with Cantor next

is expanding effect have headwinds And sales markets? Rick the you new effect thinking talked how you This exchange any, you if as about continue believe as XX-XX you grow Galafold in guide into the Kristen. on for What, ex-U.S. you've are about business? this do foreign towards the to split and that on could U.S.

Yes.

from a saw saw and there you as But -- greater as the coming were little think this U.S. I year I going think a I'm revenue getting of line. you the ex-U.S. -- distribution versus slightly

XX% XX%, XX-XX, on would -- like the is So slight which still it Europe. say, versus near impact I just I the was that shows think that but

of on all average. general, different States, I United will point, that your forward, you those XX-XX To expect see we think it's course, but come of would in typically on, the as currencies. going do new markets be split outside

FX predicting of is think But around world remains important the most growth the globally. the thing in business rates not we're between two I the So the strong.

in over to out balance significant that growth the expect to continue and U.S. U.S., We roughly outside growth significant time, XX-XX. about should

Instructions]. [Operator

Berenberg. from Shu come next with Our Zhiqiang will question

And impact Congrats of ongoing on looking color will from talk talked you in about the bit I your Slide support pretty Galafold the at year flat it of a relates secondly, be progress IOPD. exchange, on first and that want label and foreign just about Maybe full LOPD the on -- strange. Maybe they you half that? to well. the foreign the studies there discussion? implications negative the second as considering can concerning how sales to guidance, provide your and the XX, like ask half, in about looks

can Maybe to I'll Jeff, the the back impact question how come it take you end will label of development and the And over time. then, first. that strategy

could what the said So look, exchange a growth, on relates impact rates. as the of to predicting foreign revenue. just it actual as I we're Galafold point reported not in be think we've in business But time, the

we made patients, in For with said around really growing what this growth the underlying that's us, the that business growing, the right? most robustly. terms And from really growing growing would. that's really strongly demand, last business it's right point is business line that remains think time I growing So and how is of XX% it the strong, remains and in and the year important thing Sebastien

greater relates year, of of half of second a the half we the usually in year of it second year. the to the the see distribution As versus revenue first the half

growing, going strategy development point impacts of operational business million. impact all in difficult $XXX to could basis, to of we the that little strong provided laid populations is that. in And you the But different how perspective. want that bit importance the we've a is for for million confident we what off exchange and exactly growing us, the reported now, we about very our predict. and that to And But markets, But particular, talk continue for Pompe and that again, forward. kind key and an that guidance will do foreign anticipate pediatric. from $XXX reiterating from estimate so, are we're to Again, the a Jeff, be see is

Yes, sure.

adult So was only PROPEL reminder, as patients. a included

do do So of regulatory We agreement on and data and will pediatric the an patients, population. those focused the Pompe pediatric down trial in getting LOPD of are with that patients imminently initial study, and be an our initial that measures patients. ongoing vast would majority start have that have LOPD adult And do into labeling. clinical need submissions expected we adult to efficacy our patients of safety to we to look We from adding in IOPC well. as IOPD do agencies at ready efficacy be extrapolation to some the

So And we as to where we expanded labels focused the call, access pediatric I noted then initial patients, patients, we expect treat adults, have there through IOPD, we as to on continue on in pediatric will. significant LOPD but is data sufficient add need. multiple the

And with question our Cowen. will from Baral Ritu next come

how down are U.S. wondering into I it. the little mutations growth prescribers this re-acceleration. dynamics new And the more launch? just long also, surprised much guess growing that drill sort the into return or into of to play still wanted to I growth and I'm a I'm

that still to driving next-gen You program? mutations which the the have Is about that of on label growing triggered the European the the guys And number you're like does those growth? U.S. the number the cash to Was are compare in in how there U.S. slides. label? a mutations But hit, label? of the

Yes.

relates to is with sales physicians the opportunity to described, it together. Sebastien team be what which medical our to in really more United and for increasing growth do has think and the it States, dynamic as So I

While start frequent those and on physicians I can also therapy. nothing interactions, access think physicians, for and did patients it of And access job more beats we a virtual kind improved that some maintaining patients great patients so probably of represents for face-to-face. and physicians

continuing in resurgence And But of of therapy even of rate U.S., to say a continue end it's and kind whether to from to COVID in really says and bit it's in driving that So what's growing normal I just the on think but now different can that again, end around second we're way, XX% I that July, whether ability numbers, world. kind last new or handle a adjustment that. that's business To of some versus second a the coming quarter in demand. great of we not to year. that the expect me, strong, quarter is growth see patients

able be of who we and so they actual diagnosed we it really were those we and interesting Europeans if mutations had mutations, eligible the you're the that right. exciting European theoretical amenability, relates characterize that amenable concept label accepted piece, immediately every chaperone of the about the the the no, and all part could were mutations the to As does United that to Remember, is to able be patient it's an therapy. not that to expand the mutation, even then States, In we for that one though have mutation find label, -- can slightly with different.

update accept concept in mutation the we amenability that the can patient in update the While find they to a label the with and to we label. fully subsequently obligated United States, are order then

But we'd So just eventually, patients. bit those a process. a majority find amenable little slower of a presumably, of it's

do we reflects have of to prescribers continue continuing new Galafold. we just patients, then to rare As patients continue honestly, we the where reflection just disease to these space for physicians. I come to the on relates And new are that's prescribers, physicians think which it I -- continue a growth continue see and again to find new opportunity board, think, --

positive So come. years all to business for for the great signs

it in I in that later? U.S. left to many And mutations guess, be included as how the are, you see

to want the mean Jeff? you then what's label? label, I what's Jeff, U.S. give European the the -- And

Yes.

X,XXX almost in mutations it's the label, in and XXX think I U.S. it's amenable So European around

difficulty) patients we add (technical a year mutations look, to every and mutation. have patient continue novel as So families

add So don't really Brad a that, true reach it U.S., time. we coming rate. don't have theoretical assay. might the number doesn't that the just to them, just think will expect slight over in already ever the might do it in up it to said, access be mutation keep the the as it will label. need FDA label. on We paperwork as the tested I to with we We've go But cause not dream limit through so delay it that's to a

call. thank ladies conclude our and this attending today's this concluding you now you to time, for conference at disconnect like may And question-and-answer lines. today's gentlemen, We'd presentation. also will And your session,